According to a study published in Nature, a journal, researchers have found the reason behind obesity leading to hair thinning.Also Read - Surgery For Weight Loss: What is Bariatric Surgery, How Does it Help in Reducing Weight, And Is it Safe?

They found that stem cells within hair follicles in mice given a high-fat diet behaved differently from those in mice with a standard diet. Inflammatory signals in the stem cells led to these differences, ultimately resulting in hair thinning and loss. These fascinating data shed light on the complicated link between obesity and organ dysfunction. Also Read - Study Suggests Obesity is Not Caused by Overeating, it Depends on Quality of the Food

Its well known that obesity is linked to the development of numerous diseases in humans. Heart disease, diabetes, and other ailments are extremely common in obese individuals. However, its not fully clear how body organs specifically deteriorate and lose functionality from chronic obesity. Also Read - What is a Hormonal Belly? 3 Vital Signs That Your Hormones Are The Reason Behind Your Belly Fat

In a recent study, a group of researchers from Tokyo Medical and Dental University (TMDU) used mouse model experiments to examine how a high-fat diet or genetically induced obesity can affect hair thinning and loss.

The authors found that obesity can lead to depletion of hair follicle stem cells (HFSCs) through the induction of certain inflammatory signals, blocking hair follicle regeneration and ultimately resulting in loss of hair follicles.

Normally, HFSCs self-renew every hair follicle cycle. This is part of the process that allows our hair to continuously grow back. As humans age, HFSCs fail to replenish themselves leading to fewer HFSCs and therefore hair thinning.

Although overweight people have a higher risk of androgenic alopecia, whether obesity accelerates hair thinning, how and the molecular mechanisms have been largely unknown. The TMDU group aimed to address those questions and identified some of the mechanisms.

High-fat diet feeding accelerates hair thinning by depleting HFSCs that replenish mature cells that grow hair, especially in old mice. We compared the gene expression in HFSCs between HFD-fed mice and standard diet-fed mice and traced the fate of those HFSCs after their activation, said the lead author of the study Hironobu Morinaga.

We found that those HFSCs in HFD-fed obese mice change their fate into the skin surface corneocytes or sebocytes that secrete sebum upon their activation. Those mice show the faster hair loss and smaller hair follicles along with depletion of HFSCs. Even with HFD feeding in four consecutive days, HFSCs shows increased oxidative stress and the signs of epidermal differentiation, Morinaga added.

The gene expression in HFSCs from the high-fat-fed mice indicated the activation of inflammatory cytokine signalling within HFSCs. The inflammatory signals in HFSCs strikingly repress Sonic hedgehog signalling that plays a crucial role in hair follicle regeneration in HFSCs, described Emi K. Nishimura, a senior author.

The researchers confirmed the activation of the Sonic hedgehog signalling pathway in this process can rescue the depletion of HFSCs.

This could prevent the hair loss brought on by the high-fat diet, said Nishimura.

This study has provided interesting new insights into the specific cellular fate changes and tissue dysfunction that can occur following a high-fat diet or genetically induced obesity and may open the door for future prevention and treatment of hair thinning as well as for the understanding of obesity-related diseases.

(With inputs from ANI)

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Can Obesity Lead to Hair Fall? Here's What The Research Says - India.com

Recommendation and review posted by Bethany Smith

Facial serums are skin care products that the skin can absorb rapidly. They are formulated to contain high doses of ingredients, including actives such as vitamin C and retinol. They are not moisturizers. Instead, they are an additional step in a skin care routine selected to address specific skin concerns.

While many facial serums make claims about their benefits, limited scientific data is available to support these claims. The potential uses and benefits of a serum will depend on the ingredients and their concentration.

Many serums use active ingredients such as:

Common issues face serums claim to address include acne, rosacea, and signs of aging such as wrinkles.

One study of a facial serum targeting fine lines and wrinkles found that after 12 weeks, women showed statistically significant improvements in:

Face serums are generally considered safe. They are available without a prescription. However, because face serums deliver concentrated doses of active ingredients such as vitamin C or hyaluronic acid, people should exercise caution when trying them for the first time.

Pregnant people should exercise caution when selecting skin care products. Using products made with concentrated forms of vitamin A, such as retinol, can harm a developing fetus. Pregnant people should avoid skin care products that contain retinoids or other harmful ingredients.

People who are already using prescription or high dosage skin care products should check with their healthcare providers before using face serums to ensure they are not taking in unhealthy levels of powerful ingredients, such as retinoids.

When introducing a new active ingredient to the skin, there is a possibility of side effects such as:

If a person experiences these symptoms, they should reduce the frequency and dosage of the serum or stop using it altogether if symptoms do not reduce.

The American Academy of Dermatology recommends trying just one new product, like a facial serum, at a time. Starting several new products all at once, particularly anti-aging products, can irritate the skin and make it difficult to determine which products are beneficial.

It is also helpful to perform a patch test for a week before using a new product on facial skin. A patch test involves applying small amounts of the new product on the inner forearm for a few days to check for reactions.

A skin serum will not be effective if it is not used properly. The best time to apply face serum is after cleaning the skin and before applying moisturizer.

Facial serums are concentrated, so people should only use a small amount to cover the facial skin.

Learn more about skin care routines here.

No single skin care product can address all skin health needs. To pick out the best face serum, a person should first decide what skin concern they want to address, such as wrinkles or acne.

Look for a face serum that is a good fit for individual concerns and needs. For example, a person should choose a face serum that suits their skin type, such as oily, dry, combination, or aging.

Some people may wish to find products that are hypoallergenic and noncomedogenic, which means they are less likely to cause allergic reactions or acne.

Products do not have to be expensive to be effective, and serums are available to suit various budgets.

Please note that the writer of this article has not tried these products. All information presented is purely research-based.

This is an oil-free vitamin C face serum designed for people with oily or blemish-prone skin.

SkinCeuticals claims this serum can reduce skin oiliness and wrinkles, and improve skin texture.

The ingredients include:

This serum costs $166 for a 30ml bottle.

Made with Avene Thermal Spring Water, this water-gel serum is safe for adolescents and adults.

It is suitable for oily skin and should be used after cleansing in the morning and evening.

Avena claims that this product can be used safely alongside other acne treatments and can reduce the appearance of pores.

This serum costs $28.00 for 30ml.

This antioxidant face serum is fragrance- and oil-free, and certified cruelty-free.

Drunk Elephant claims it can reduce the signs of sun damage and aging while hydrating the skin. It stays active on the skin for up to 72 hours and should be applied in the morning.

Customers must mix a powder and liquid to create the fresh activated serum.

This serum costs $78.00 for 28ml.

This face serum contains aloe, vitamin A, niacinamide, vitamin C, vitamin E, tea tree oil, and other vitamin and herbal ingredients.

Klur states that this serum may even out skin texture, helps calms inflammation, and increases skin clarity.

People should use it at night and no more than 3 times a week.

This serum costs $110.00 for 30ml.

This serum uses forms of retinoid that The Ordinary states may reduce the signs of aging better than other retinoid products, without causing irritation.

The company recommends people patch test the serum, before using it on facial skin. People should not use it in combination with other retinoid products.

Use only in the evening after cleansing, and refrigerate after opening.

This serum is vegan and free from oil, silicones, nuts, and gluten.

This low-cost serum sells for $9.80 for 60ml.

This cruelty-free skin serum contains milk protein, seaweed extract, and arnica. Ren claims it may calm and soothe the skin while reducing redness and irritation.

It should be used morning and/or night before moisturizing.

This serum costs $58.00 for 30ml.

True Botanicals claims that this anti-aging serum may reduce the appearance of fine lines and wrinkles.

It uses natural antioxidants such as chebula, elderberry, ginger, and echinacea to help strengthen the skins immunity to signs of aging.

After morning and evening cleansing, users can apply one half or full drop to their faces.

This serum costs $90.00 for 30ml.

Skin Authority claims that this lightweight serum reduces the appearance of fine lines, by plumping skin folds and smoothing skin texture.

This serum is designed for use after morning cleansing.

This serum costs $155.00 for 15ml.

Peach and Lily claims that this serum can brighten the skin and reduce the appearance of dark spots.

It contains various ingredients from botanical sources, including green tea extracts, mushrooms, and arbutin. It also contains niacinamide.

It is suitable for all skin types.

This serum costs $39.00 for 50ml.

SkinCeuticals claims that this serum may reduce the appearance of dark spots and improves skin tone.

Ingredients include:

It is suitable for people with all skin types.

This serum costs $98.00 for 30ml.

Bioderma claims that this face serum may increase the skins ability to retain moisture. Made specifically for people with dry skin, it is noncomedogenic, meaning it will not block pores.

People can use this serum in the morning and evening.

This serum costs $27.99 for 40ml.

This serum is suitable for use by people with all skin types. Eminence claims that this face serum helps reduce the appearance of sun-induced skin damage.

Its active ingredients include vitamin C and other antioxidants, which work together to brighten the skin and improve the appearance of fine lines and wrinkles.

This serum costs $110.00 for 30ml.

The rest is here:
12 of the best face serums 2021 - Medical News Today

Recommendation and review posted by Bethany Smith

A man went to a clinic in Mannheim, Germany, where tests found that the rash on his hands and elbows was in fact leukaemia cutis, where the cancer cells are in the skin tissue

Image: NEJM)

A man suffering from a nasty skin rash on his hands and elbows that looked like psoriasis found that it was actually a form of leukaemia.

The 65-year-old went to a dermatologist in Mannheim, Germany, due to the red patches that had developed on the back of his hands and arms and he later found the worrying diagnosis that it was leukaemia cutis.

Tests done at the hospital showed that he had a high white-cell count along with a low number of platelets, it is reported.

The case was published in the New England Medical Journal where it said that a diagnosis of leukaemia cutis was made.

While extremely rare, the condition means that the leukaemia cells are in the skin tissue.

It is found to happen in around three percent of leukaemia cases where the rash has been found on skin including the arms, back or face.

Image:

Other leukaemia symptoms include fever, tiredness and susceptibility to infections.

But in the case of the 65-year-old in Germany, he only had the rash on his hands and elbow.

The New England Medical Journal stated: "A diagnosis of leukemia cutis was made, and the patient was urgently referred to the oncology clinic.

"The patient received a diagnosis of chronic myelomonocytic leukaemia and underwent stem-cell transplantation.

"Two weeks after the transplantation, the patients skin changes had resolved, and the cancer has been in remission since."

Leukaemia Care UK said that usually patients who have the cancer and find rashes do not have this particular condition.

It stated: Leukaemia cutis is very rare, occurring in only about three percent of total cases of leukaemia.

"With this in mind, it is unsurprising that most lesions seen in leukaemia patients are not leukaemia cutis.

In fact, most lesions (40%) seen in leukaemia patients are caused by other complications of leukaemia.

It continued: In the rare occasion that leukaemia cutis occurs, the patient will normally have already been diagnosed with leukaemia.

"However, in seven percent of cases of leukaemia cutis, the skin lesion is the very first symptom of a blood cancer. This is sometimes referred to as aleukaemic leukaemia cutis.

Link:
Man discovers nasty red rash on his hands and elbows is potentially fatal - The Mirror

Recommendation and review posted by Bethany Smith

Your skin is your largest organ, which means it's well worth whatever investment you decide to put toward maintaining its health. While budget beauty can certainly get the job done, sometimes you can't help but lust after the La Mers and La Prairies of the world. Though these types of pricey productsmany of which you probably recognize from the shelfies of the ultra-rich-and-famousalmost never go on sale, right now you can snag them for seriously discounted prices as a part of the Friends and Family Saks Skin-Care Sale.

Wondering how you can suss out which products are worth the splurge? Generally, serums and oilswhich have the highest concentrations of active ingredientstend to give you the most bang for your buck. Beyond that, though, "its important to evaluate and investigate the active ingredients in each product and not simply choose a product based on its marketing, packaging, or promises," Rachel Nazarian, MD, a board-certified dermatologist based in New York City, previously told Well+Good. Lucky for you, our beauty editors have done all the legwork in sorting through Saks' on-sale skin care to determine which products live up to their hefty (albeit currently discounted) price tags. Keep scrolling to shop our picks.

Photo: Estee Lauder

Este Lauder Advanced Night Repair Serum Duo Synchronized Multi-Recovery Complex $153.00

Originally $180, now $153

Consistently ranked as the number-one anti-aging serum in the U.S., this serum optimizes your skin natural repair process to fend off multiple signs of aging, including fine lines, wrinkles, and discoloration. Though its got a lofty price tag, it serves double duty as a skin-protecting antioxidant serum during the day and a skin-recovery anti-aging serum at night.

La Prairie Skin Caviar Skin Caviar Nighttime Oil $451.00

Originally $530, now $451

Nothing says indulgent quite like caviar, which happens to be the star ingredient in this evening oil. The luxe formula includes both caviar-derived retinol and lipids, which work together to stimulate cellular turnover and strengthen your skin barrier. Youll see stronger, smoother, and firmer skin after a single nightand the results will get even better over time.

Dr. Barbara Sturm Hyaluronic Serum $255.00

Originally $300, now $255

Often considered the holy grail of luxury hyaluronic acid serums, its rare that youll find this stuff on sale (which means that your time to snag it is n-o-w). It contains three different molecular weights of hyaluronic acid, which penetrate and hydrate your skin at different levels, plus purslane, a natural ingredient rich in cell-renewal stimulating vitamin A and moisturizing vitamin E.

Cl de Peau Beaut La Crme $468.00

Originally $550, now $468

Believe it or not, the moisturizer encapsulated in this luxe-looking gold container is even more opulent than the packaging implies. It combines many of skin cares buzziest ingredientswrinkle-fighting retinol, hydrating hyaluronic acid, moisturizing squalane, antioxidant-rich silkwith a proprietary dark-spot-correcting blend, and leaves a luminous finish that only enhances its lavishness.

Augustinus Bader The Rich Cream PPC Cellular Renewal Rich Cream $145.00

Originally $170, now $145

Often referred to as the best skin care in the world the technology used in this moisturizer is truly unparalleled. It was initially developed to help with scarring in burn victims, but was brought to the wider market when developers realized how effective it was in aiding in overall skin health. So what makes it so special? It uses epigentic technology to deliver nutrients to specific parts of the cells to change the expression of their DNA, effectively catalyzing them to repair on their own, which improves the appearance of issues like scarring, discoloration, and wrinkles.

La Mer The Concentrate $178.00

Originally $210, now $178

Made with concentrated amounts of La Mers famed miracle broth (which is made from hand-harvested kelp) plus a stabilizing ferment that combines marine algae and seawater and holds three different types of sea minerals that help rejuvenate skin. When one of our editors tested out the concentrate for a full month, she saw her skin transform into a brighter, more even, and more hydrated version of itself, and deemed the product well worth the hefty chunk of change it will cost you.

Testament Beauty Turkish Coffee 3-in-1 Mask $58.00

Originally $68, now $58

For $58 (which, admittedly, feels like a steal compared to some of the other products on this list), this mask will give you triple beauty benefits in a single application. Finely-ground coffee seeds exfoliate and awaken skin, a combination of niacinamide and anti-inflammatory botanicals (think: safflower and sunflower) help to calm irritation, and ceramides and fatty acids aid in hydration and skin barrier strength. Its a scrub, a mask, and a moisturizer all in one, and can be used in place of multiple steps in your usual routine.

Elemis Pro-Collagen Oxygenating Night Cream $136.00

Originally $160, now $136

Your evening skin routine should be all about regeneration (fun fact: your skin repairs itself while you sleep), a process this moisturizer works to support. Its made with a blend of marine ingredients that infuse hydration, stimulate collagen production, and help ramp up your skins own antioxidant defenses, leaving you with a smoother, more supple complexion that will stay moisturized for 12 full hours after you apply.

MZ Skin Lift & Lustre Golden Elixir Antioxidant Serum $280.00

Originally $329, now $280

I count this as one of the few luxury serums in my own skin-care collection, and can confirm that its among the best that money can buy. Formulated by dermatologist Maryam Zamani, its made with a concentrated blend of stem cells, botanical extracts, and hyaluronic acid. According to clinical studies conducted by the brand, over the course of 30 days, the antioxidant-rich serum increases hydration by 25 percent, improves wrinkle depth by 16 percent, and enhances radiance by 38 percent. Personally, I love to use it as a part of my a.m. routine (or ahead of special occasions), because its gold flecks give my complexion a lit-from-within glow that looks gorgeous on its own or layered under makeup. Whats more? You can also dab it on your cheeks, brow bones, or Cupids bow as a stand-in for your go-to highlighter.

Check out the video below to find out which high-end products are a part of a dermatologist's own routine.

Want even more beauty intel from our editors? Follow our Fine Print Instagram account) for must-know tips and tricks.

Our editors independently select these products. Making a purchase through our links may earn Well+Good a commission.

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The Number 1 Serum in the U.S. Is on Sale at SaksAlong With Other Never-Discounted Buys - Well+Good

Recommendation and review posted by Bethany Smith

Kyoto A drug discovered through a method using induced pluripotent stem cells, or iPS cells, from sufferers of amyotrophic lateral sclerosis (ALS) was effective in stopping the progression of the neurological disease in five out of nine patients in a clinical trial, a research team at Kyoto University said Thursday.

While drugs that slow down the progression of amyotrophic lateral sclerosis, also known as Lou Gehrigs disease, have been used in the past, this is the first time that a chronic myeloid leukemia drug called bosutinib has been found to stop its progression, according to the team.

Haruhisa Inoue, a professor of neurology at Kyoto University leading the team, said that larger clinical trials will be needed to determine if the drug can be put to practical use, but We are now looking at the possibility of being able to control ALS with the power of science.

The team reproduced the disease by culturing iPS cells derived from the skin of patients into motor neurons. They then tested a series of drug compounds on the cells to find that bosutinib was effective in slowing down the progression of ALS.

The drug was administered for around three months to nine patients who were in the early stages of the disease and showing signs of deterioration. Progression of the disease stopped in five patients during the treatment period, while four patients continued to deteriorate at the same pace as before.

A comparison of the blood samples from both sets of patients showed that they had differing amounts of a protein unique to nerve cells before the drug was administered. Researchers plan to conduct clinical trials with larger groups while adjusting the dosage and other conditions in the future.

There are about 9,000 people in Japan who suffer from ALS. The disease causes motor neurons to progressively die, resulting in paralysis, with many patients requiring a ventilator to stay alive. Its exact cause is unknown, and no fundamental treatments have been established.

In a time of both misinformation and too much information, quality journalism is more crucial than ever.By subscribing, you can help us get the story right.

PHOTO GALLERY (CLICK TO ENLARGE)

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Drug that stops ALS progression found using iPS cells from patients - The Japan Times

Recommendation and review posted by Bethany Smith

Photo: Francis Collins, by NIH Image Gallery, via Flickr.

Francis Collins, Director of the National Institutes of Health (NIH),has just announcedhis intention to step down at the end of 2021 after more than 12 years heading the agency. The accolades are already rolling in. Noted evangelical political commentator David French, for example, rushed to praise Collins as a national treasure.

But Collinss real legacy is anything but praiseworthy, and the tendency of figures in the faith community to ignore his real record is far from admirable.

This year of all years should have made the truth about Francis Collins clear. Last month, documents were released suggesting that top National Institutes of Health (NIH) officials may haveliedwhen they denied that the NIH had funded gain of function research in Wuhan, China, that could have resulted in a pathogen that could infect humans.

After reviewing the documents, Rutgers University biologist Richard Ebright had a blistering response: The documents make it clear that assertions by the NIH Director, Francis Collins, and the NIAID Director, Anthony Fauci, that the NIH did not support gain-of-function research or potential pandemic pathogen enhancement are untruthful.

It was another blow to the reputation of Collins in a year when his agency has faced multiple scandals and controversies.

Among evangelical Christians and other people of faith in America, Collins has long been the equivalent of a rock star. But Collinss days of glory as a non-partisan role model, especially for the faith community, may be numbered and its not just because of the latest scandal over the origins of COVID-19.

In recent months, Collinss agency has become embroiled in controversies over its funding of stomach-churning medical experiments involving body parts harvested from aborted babies. The disclosures about the experiments followed Collinssrepealearlier this year of restrictions on the use of aborted fetal tissue in NIH-funded research.

Collins has also stirred controversy with his increasingly shrill attacks on unvaccinated Americans and his support for harsh mandatory vaccination policies that will require the firing of employees who choose not to be vaccinated with a COVID-19 vaccine.

The former head of the Human Genome Project, Collins catapulted to fame (and the cover ofTimeMagazine) in 2000 with the announcement of a working draft of the sequence of the human genome.He then became a hero to many Christians with the publication in 2006 of his bookThe Language of God, which recounted his journey from atheism to Christianity.

In an increasingly polarized national environment, Collins is one of the rare heads of a major federal agency to serve under both Republican and Democratic Presidents. Appointed by President Obama to head the NIH in 2009, Collins continued in that role under President Trump and now President Biden. He has regularly drawn praise from lawmakers on both sides of the aisle. At gatherings of evangelical Christians, he has been known to strum the guitar and sing worship songs and receive the adoration of attendees. At one 2019 eventit was reportedthat conference participants lined up for selfies with him.

In 2020, Collins wasawarded the prestigious Templeton Prize, worth more than $1.3 million, for his work integrating science and faith. Previous recipients of the prize have included Mother Teresa, John Polkinghorne, Charles Colson, and Aleksandr Solzhenitsyn.

Among secular as well as religious journalists, Collins often receives what verges on fawning treatment. A writer forThe New Yorkergushedthat Collins is a model of geek cool. He likes big, noisy motorcycles, and, despite a mild manner, he is famously unself-conscious. At the unlikeliest moments, he will strap on a guitar and accompany himself in song, often a tune he has composed for the occasion.

This year, however, Collinss reputation has taken continued beatings, not just because of evasive answers about the role of the NIH in gain-of-function research in China, but also because of publicity around NIH-funded experiments that many Americans, especially people of faith, would find horrific.

In May, reports surfaced aboutmacabre NIH-funded experimentsthat utilized body parts collected fromaborted human fetuses to createhumanized mouse and rodent models with full-thickness human skin.For the experiments, researchersat the University of Pittsburghcut into tiny pieces human fetal spleen, thymus, and liver organs and then transplanted the tissues and hematopoietic stem cells into irradiated mice. Researchers also sliced off skin from the scalp of the aborted babies and then grafted the fetal skin onto the mice. In the words of the scientists: Full-thickness human fetal skin was processed via removal of excess fat tissues attached to the subcutaneous layer of the skin, then engrafted over the rib cage, where the mouse skin was previously excised.

The body parts used for these experiments were harvested from aborted human fetuses with a gestational age of 18-20 weeks. By that age, an unborn baby hasbrain wavesand abeating heart. He canhear sounds and move his limbs and eyes, and his digestion system has started to work.In other words, the human fetuses whose organs were harvested for this NIH-funded research were well-developed tiny humans, not blobs of undifferentiated cells.

In August, an additional project funded by the NIH came to light thanks to documents obtained in a Freedom of Information Act lawsuit by Judicial Watch and the Center for Medical Progress. The lawsuit was filed after Collinss NIH dragged its feet in responding.According to Judicial Watch, the documents show that theNIH has provided nearly $3 million in tax dollars to support a fetal organ harvesting operation by the University of Pittsburgh in its quest to become a Tissue Hub for human fetal tissue ranging from 6 to 42 [!] weeks gestation.

David Daleiden, president of the Center for Medical Progress,commented: The NIH grant application for just one of Pitts numerous experiments with aborted infants reads like an episode ofAmerican Horror Story. Infants in the womb, some old enough to be viable, are being aborted alive and killed for organ harvesting, in order to bring in millions of dollars in taxpayer funding.

Daleiden furtherallegedthat NIH funding was used to underwrite labor induction abortions, where the baby is pushed out of the mother whole and then killed to obtain the desired tissues. In other words, the NIH was facilitating a process where babies, some of the age of viability, [are] to be delivered alive, and then killing them by cutting their kidneys out.

Francis Collins self-identifies as an evangelical Christian, and most evangelicals as well as faithful Catholics regard abortion as the destruction of innocent human life.

So how has Collins responded to these revelations? With horror? With a pledge to investigate? With a promise to stop taxpayer funding of such research?

Since early August, Ive repeatedly tried to get Collins to answer questions about these NIH funded experiments using aborted fetal organs and tissues.Does Collins support this research funded by his agency? Does he have any ethical objections to it? How does he personally justify the research given his religious convictions? I repeatedly emailed these and other questions to the media contacts for the NIH Office of the Director.

The response? His office has refused to answer.

But its not just NIH research involving humans that has been raising controversy this year. In recent months, Collinss NIH has come under fire for fundingabusive medical experiments on dogsthat critics say were unnecessary as well as barbaric. The experiments were funded by the NIH division headed by Anthony Fauci, but that division is under the ultimate oversight of Collins. In late August, 15 members of Congresssent a letterraising questions about the research.

Again, I tried to get a response from Collins about this latest controversy. Again, he refused to respond to questions.

Ironically, while Collins is AWOL when it comes to answering basic questions about the research his agency is funding, he is more than willing to speak out on other topics, especially COVID-19, where he is now becoming a polarizing figure to many because ofincreasingly shrill advocacy of compulsory vaccination as well as his demonization of those who choose not to take a COVID-19 vaccine.

At the end of April,Collins claimedthat he would not require NIH employees to get vaccinated, and he seemed to argue for a positive approach of selling the benefits of vaccines rather than demonizing the unvaccinated or engaging in finger-wagging. Yet by early August his public posture had changed. He was nowcheerleading for compulsory vaccine requirementsimposed by private businesses as well as enthusiastically overseeing compulsory vaccinations for the very workers at the NIH that he earlier said would not face compulsory vaccination.

After President Bidens speech in September declaringwar on the unvaccinated, Collins ramped up his own rhetoric.In an appearance on MSNBC after Bidens speech, Collins firstsuggestedunvaccinated people were selfish, declaring that this is really an occasion to think about loving your neighbor, not just yourself.

Collins then branded both unvaccinated people and politicians who dont favor vaccine mandates as killers on the wrong side of history. Dismissing their views as merely a philosophical political argument that is part of the culture war, Collins complained that this philosophical political argument is killing people, including, Im sad to say, some children. We have to get past this if we really have a future as a nation.

I would like to say particularly to those leaders who are on the wrong side of this, what Lincoln said one time, Collins declared. Citizens, we will not escape history. Do you want to be looked at in the lens of that backward look ten years from now and defend what you did when in fact, we are losing tens of thousands of lives that didnt have to die?

A question for Collins: Is attacking your fellow citizens (including many fellow Christians) as heartless killers because they disagree with you on either vaccinations or vaccine mandates an example of loving your neighbor?

Whatever one thinks about COVID-19 vaccinations, Collinss over-the-top rhetoric demonizing those he disagrees with as killers is beyond the pale, especially for someone who wears his Christianity on his sleeve. As I have writtenelsewhere, his rhetoric is also based on several falsehoods.

So just how far is Collins willing to go to push coercive medicine? Thats an interesting question.

In my home state of Washington, the governor has issuedan emergency orderthat will compel private religious schools and day care centers as well as other private businesses to fire employees later this month if they wont get vaccinated. While there technically is a route for religious exemptions, it is so narrow and onerous that many religious people may not qualify.

Its now being suggested in some states that discharged employeeswont be able to get unemployment benefits. Perhaps the idea is that if the unvaccinated dont die of COVID they can die of starvation instead.

As if that werent bad enough, the unvaccinated face the denial of medical care. Also in my home state, the University of Washington medical system is now apparently denyingorgan transplantsto patients who are unvaccinated, even if those patients have a credible medical reason for not having the vaccinations.

This is pure, unadulterated social Darwinism: Brand a whole class of people as biologically unfit (in this case, the unvaccinated) and then make sure they cant receive medical care, hold jobs, or basically survive. Heap scorn on them, demonize them as killers, and stir up hatred against them so other people begin to abuse them. If Collins is truly concerned about the judgment of history, he should read a little more widely about the sorry results of demonizing entire classes of people as the enemies of society.

Let me be clear: I am not arguing here about whether people ought to get COVID-19 vaccinations, or whether those vaccinations are helpful. For the record, depending on ones risk profile, I think vaccinations are in a persons best interest. The issue is whether in the name of vaccination people should be stripped of their livelihoods, denied medical care, and demonized as enemies of society. In any morally sane universe, the policies being proposed are as immoral as they are unprecedented.

So does Francis Collins endorse depriving unvaccinated people of their right to work and to support their families? Does he endorse denying them unemployment insurance? Does he go even further and endorse denying medical care to the unvaccinated?

I again asked Collins media relations staff for answers. Again, crickets.

Although Collins likes to tout his personal faith, he appears to have very little concern for any sort of conscience rights of fellows religious believers who disagree with him.After all, he dutifully served in a previous administration that repeatedly weakenedconscience protectionsfor medical workers opposed to abortion and thatviolated federal lawby turning a blind eye when California mandated abortion coverage in all private insurance plans.

It might also be noted that as late as December 2020, Collins was still urging thatmost churches should not meet in person, even implying that they shouldnt do sountil the summer or fall of 2021.

And his current promotion of compulsory vaccinations seemingly has no qualifiers. At least, he isnt talking about any.

Collins hasconcededthat various COVID-19 vaccines used cell lines originally derived from an aborted fetus in either their production or testing, which is one reason some people have moral qualms about the vaccines. Yet you wont find Collins advocating for the conscience rights of these people. In fact, Collins has been silent in the face of attacks on religious exemptions for vaccines.

Reasonable people can disagree about whether the use of abortion-derived cell lines is a moral deal-killer for the vaccines. But thats the point: Reasonable people candisagreeabout what violates their conscience. The test of support for religious liberty is not whether you only support the right of people who agree with you.

Not being willing to stand up for the conscience rights of others to determine their own medical treatment is not morally neutral. It is a moral failure. In the words of theCatholic Bishops of Colorado, A person is morally required to obey his or her conscience, and others should respect the right of others to follow their conscience.

Alas, for those who have followed Francis Collins closely over the years, his current failures of moral leadership come as no surprise. As I will discuss in an upcoming article, Collinss career has been one long testament to moral cowardice and confusion.

Here is the original post:
The Appalling Moral Failure of Francis Collins - Discovery Institute

Recommendation and review posted by Bethany Smith

Many brands manufacture eye creams for people who experience eye bags, dark circles, puffiness, and wrinkles. Some are allergy-safe and are also suitable for those who wear contact lenses.

This article explores what eye creams are and what to consider when buying a skin care product for the eye area. It also reviews a range of products available for purchase online and discusses some safety precautions.

Eye creams are cosmetic products specifically formulated for use on the delicate skin around the eyes. Eye creams can help hydrate and brighten the skin and sometimes improves its firmness.

The skin surrounding the eyes is thinner than the skin on the rest of the face. As a result, it may be more prone to dryness, the negative effects of environmental pollution, and sun damage.

Additionally, when a person blinks or uses facial expressions, the skin around the eyes can develop lines, wrinkles, and crows feet.

Some benefits a person may get from using eye creams may include:

However, there are some risks. For example, they may not be suitable for people who wear contact lenses. They could also irritate the skin,

The following are some factors individuals can look for when choosing an eye cream:

Our writer chose the following eye creams based on online research and customer review sites.

Please also note that the writer has not tested these products. All information in this article is research-based, and we do not intend to recommend certain products over others.

Size: 0.5 ounces (oz)

Skin types: All

Eminences eye cream contains snow mushroom, which reportedly pulls moisture into the skin and enhances its elasticity and barrier function.

Eminence states that the other ingredients promote hydration, reduce puffiness, and the appearance of under-eye bags.

In addition, the company claims it does not test its products on animals, and that this eye cream is soy and gluten-free.

Size: 0.5 oz

Skin types: Dry, normal, and sensitive

This SkinCeuticals product is a hydrating cream reportedly suited to individuals who have signs of aging around the eyes, such as crows feet and under-eye bags.

Its main ingredients include soy isoflavones, vitamin E, and silymarin. SkinCeuticals claims these ingredients help improve the skins firmness and nourish dry skin as they restore essential lipids.

Size: 0.33 oz

Skin type: Very sensitive skin

Avnes lightweight eye cream contains chamomile and dextran sulfate. According to the companys website, these ingredients may help with soothing sensitive skin and reducing puffiness.

This eye cream also includes hyaluronic acid to help hydrate the skin.

Size: 0.5 oz

Skin types: All

This Avne eye cream has four main ingredients, including retinaldehyde, hyaluronic acid, dextran sulfate, and thermal spring water.

The company claims these ingredients smooth fine lines and wrinkles, reduce the appearance of puffiness and dark circles, and soften the skin.

Size: 8.4 oz

Skin type: Sensitive skin

This product is a liquid toning lotion with a lightweight texture. Bioderma claims this cream provides a hydrating and refreshing effect.

The company also states that the ingredients in this cream reduce discomfort from dryness and lessen the appearance of visible redness.

The Bioderma website states that users can apply this lotion to their face and eye area after cleansing, and they can use the cream in the morning or evening.

Size: 0.5 oz

Skin type: Sensitive

According to customer reviews, Skin Authoritys Dramatic Eye Lift is a gel featuring a lightweight consistency. The company claims it is nongreasy, does not clog pores, and moisturizes the skin.

The product description states this cream helps lift sagging eyelids, lighten dark circles, and increase the skins firmness.

Its ingredients include evening primrose seed extract, grapefruit extract, and bergamot oil.

Size: 0.5 oz

Skin type: Dry skin

This eye gel aims to moisturize the skin and contains hyaluronic acid. Hyaluronic acid is naturally present in the skin and helps keep the skin hydrated by binding to water molecules.

Neutrogena claims its formula is noncomedogenic, does not clog pores, and has undergone testing by ophthalmologists.

Size: 0.5 oz

Skin type: Dry

The ingredients present in the Neocutis Lumire Riche eye cream include caffeine, sodium hyaluronate, and glycyrrhetinic acid. Neocutis claims these ingredients reduce puffiness, provide hydration, and brighten the skin.

In addition, the company says this product is suitable for people who wish to reduce the appearance of their wrinkles, crows feet, and dark circles.

The company has designed this cream to be noncomedogenic and states the product has also undergone testing by ophthalmologists and dermatologists.

Size: 0.5 oz

Skin type: Dry and delicate skin

Ormedic has formulated this eye gel to provide hydration to dry and delicate skin.

It contains quinoa extract, which reportedly helps reduce puffiness and the appearance of under-eye bags. The website also states that the product uses fermented black tea and plant stem cells, which feature anti-aging properties.

Size: 0.5 oz

Skin types: All

Revision Skincares DEJ eye cream aims to provide intense hydration and reduce the appearance of wrinkles, redness, and sagging.

According to the website, individuals should apply a pearl-sized amount to the under-eye area and along the orbital bone twice per day.

Size: 0.5 oz

Skin types: All

This natural cream formula uses chamomile, green tea, and ginkgo biloba leaf extract to minimize the appearance of puffiness, fine lines, and dark circles around the eyes.

Skin Authority states this product helps the skin retain moisture, while people can use it on their upper eyelid and brow line.

Size: 1 oz

Skin type: All types

This products main ingredient is hyaluronic acid. Although it is naturally present in the skin, its natural function does not involve hydrating the skin.

As a result, this formula uses three forms of hyaluronic acid, which the manufacturer combines with vitamin B5 to help plump the skin and deliver intense moisture.

Additionally, this product is reportedly lightweight, oil-free, and vegan-friendly.

The Ordinary also states it does not test finished products on animals.

Size: 0.5 oz

Skin types: All

This CeraVe cream contains ceramides to maintain the skins natural barrier and hyaluronic acid to retain the skins existing moisture.

CeraVe also uses niacinamide to calm the skin.

CeraVe uses Multivesicular Emulsion (MVE) technology to provide time-released delivery of particular ingredients in cosmetic creams. In this eye cream, MVE technology helps the skin stay hydrated throughout the day.

According to a 2016 evaluation, MVE techniques in skin care products have links to improved acne.

CeraVe claims this product has undergone testing by ophthalmologists. It is also allergy-tested and noncomedogenic. Additionally, the company states it is suitable for individuals with acne-prone skin.

See the original post here:
13 of the best eye creams 2021 - Medical News Today

Recommendation and review posted by Bethany Smith

FROM NORTH AMERICA SYNDICATE, 300 W 57th STREET, 15th FLOOR, NEW YORK, NY 10019

CUSTOMER SERVICE: (800) 708-7311 EXT. 236

TO YOUR GOOD HEALTH #TFB20211018

FOR RELEASE WEEK OF OCT. 18, 2021 (COL. 1)

BYLINE: By Keith Roach, M.D.

---

DEAR DR. ROACH: I am a 71-year-old female in very good health. I have been taking a thyroid replacement since I was 12 years old. I currently take 125 mcg of thyroxine once daily, as well as 60 mg of Cymbalta. Other than arthritis and obesity, I have no health problems. My question is regarding my lab results. My TSH is 0.04 (the normal range is 0.30-5.5); my T3 and T4 are in the normal range. My physician assures me that as long as my T3 and T4 are normal, the TSH is of no concern. I cannot lose weight, no matter what I try. Would you suggest I see an endocrinologist, or is my primary doctor correct? -- D.A.

ANSWER: The thyroid stimulating hormone is a signal from the pituitary gland to the thyroid to "tell" the thyroid to make more hormone. Since your TSH level is low, it suggests that the dose of replacement thyroid hormone is too high. This is despite the fact that the T3 (the active form of thyroid hormone) and T4 (thyroxine, the major thyroid hormone, which T3 is made from) are normal, and despite the fact that you report no symptoms. The range of normal for T3 and T4 is very broad, and the low TSH is good evidence that those levels are too high for you.

Excess thyroid hormone can cause bone disease and predispose to heart problems, such as atrial fibrillation. I think an endocrinologist is likely to say that your dose of thyroxine should be decreased.

Story continues below video

DEAR DR. ROACH: I have a history of C. diff. I had the shingles vaccine, and developed severe diarrhea, which has lasted eight weeks. Could the vaccine have instigated this? I'm now on vancomycin, and it is helping. -- M.G.

ANSWER: Clostridioides difficile is most commonly associated with antibiotic use, but can be acquired in a hospital or other nursing facility or even out in the community. I have never heard of C. diff as a complication of a vaccine, so I did a search on the VAERS database of vaccine side effects. I found no reported cases of C. diff with the shingles vaccine. I really didn't expect to.

Vaccines are a powerful public health tool, and like all medicines, they have the potential for side effects. It is human nature, when presented with a health change, to ascribe it to any new event, such as a new medicine or vaccine. Sometimes they are linked, but sometimes they are not. In this case, I think they most likely are not.

DEAR DR. ROACH: I had my first pneumonia injection (PCV13) in October 2015 and the second injection (PPSV23) in October 2016. My primary care physician sends me reminders that my pneumonia shot is past due. The head nurse at the same primary care clinic reviewed my medical files and says I do not need additional pneumonia shots for the rest of my life. I have asked my cardiologist, urologist and gastroenterologist if I need to update my pneumonia injections, but they avoid answering me. I am 72 years old and have health problems. Since the COVID virus affects the lungs and many suffer from pneumonia, I am concerned about my protection, although I have both Moderna vaccinations. Should I get a pneumonia shot? -- C.V.

ANSWER: Your nurse is correct, you are not recommended for any additional pneumonia vaccines at this time. These pneumonia vaccines protect against only one bacterium, Streptococcus pneumoniae, with no protection against COVID-19. You should get your third dose of Moderna when it's recommended (expected at the time of this writing to be eight months after your second dose). That is, unless the recommendations have changed by then -- this is a fast-moving area!

* * *

Dr. Roach regrets that he is unable to answer individual letters, but will incorporate them in the column whenever possible. Readers may email questions to ToYourGoodHealth@med.cornell.edu or send mail to 628 Virginia Dr., Orlando, FL 32803.

(c) 2021 North America Syndicate Inc.

All Rights Reserved

More here:
TSH tells thyroid gland to make more hormone - New Castle News

Recommendation and review posted by Bethany Smith

Introduction

Polymyalgia rheumatica (PMR) and remitting seronegative symmetrical synovitis with pitting edema syndrome (RS3PE syndrome) are diseases of unknown etiology that affect elderly persons. They are characterized by pain of sudden onset that continues and elevated erythrocyte sedimentation rate and C-reactive protein levels.1 Once the symptoms develop, they greatly impair patients quality of life. With the aging of the population, an increase in the number of such patients has been reported from the UK.2 The incidence of PMR has been found to be higher in individuals of Scandinavian background, lower in Southern European countries, and unknown in Japan.3,4 Although the symptoms improve markedly with glucocorticoid (GC) treatment, prudent tapering of GC is required.5 Although GC could be decreased from the initial dose in all PMR patients, it is difficult to stop GC treatment, as has been previously reported.6 Past studies reported that relapse has been variably associated with female sex, longer duration of morning stiffness, peripheral arthritis, higher erythrocyte sedimentation rate at diagnosis, persistent elevations of C-reactive protein, interleukin-6 levels, and soluble interleukin-6 receptor levels, larger initial doses of prednisone, and a faster rate of tapering.7 Furthermore, reports of GC continuation and risk factors for relapse in Japanese PMR patients are few.4 To better estimate GC treatment duration, the percentage of patients who could stop GC treatment and the baseline variables associated with inability to withdraw GC after the time when the GC continuation rate stopped decreasing were examined. In particular, sex differences were examined.

A total of 105 patients (64 women) who were started on GC treatment for PMR and/or RS3PE syndrome at Ikeda City Hospital from July 2004 to December 2019 were evaluated. Birds criteria8 were used up to 2014, and the EULAR/ACR polymyalgia rheumatica interim standard of classification9 was used between 2015 and 2019 for PMR diagnosis. Patients who showed pitting edema of both hands and both lower extremities and did not fulfill the diagnostic criteria of rheumatoid arthritis,10 spondyloarthropathy,11 or other diseases were diagnosed as having RS3PE syndrome. Both PMR and RS3PE syndrome were diagnosed in patients with PMR who showed pitting edema of both hands and both lower extremities without other cause.

The GC dose was left to the discretion of the attending physician. The initial dose of prednisolone is generally 10 to 16 mg/day, and the aim is to discontinue it by 24 years. The dose was actually reduced by 2.5 mg/day every 24 weeks in patients treated with >10 mg/day, and by 1 mg/day every 24 weeks in patients treated with 10 mg/day.7 When symptoms returned with GC reduction, the attending physician increased the dose promptly. GC was continued when it was resumed for disease recurrence once it had been stopped.

The days from GC initiation to GC withdrawal were calculated. If GC could not be stopped, the last observation day was used. The GC continuation rate during the observation period was then estimated by the KaplanMeier method, creating KaplanMeier curves by sex.

Furthermore, cases that had stopped GC (withdrawal group) and cases that had continued GC for 7.5 years (continuation group) were identified, and the following were compared between them: Age at time of starting GC treatment; sex; type (PMR and/or RS3PE syndrome); erythrocyte sedimentation rate, C-reactive protein, hemoglobin, ferritin, aspartate aminotransferase, and alanine aminotransferase levels before starting GC; days from onset of symptoms to GC initiation; GC maximum dose; GC dose half a year after it was started; presence of relapse (GC restarting or increasing due to deterioration of symptoms); and the presence of concomitant malignant disease. Cases belonging to neither the withdrawal group nor the continuation group constituted the intermediate group. Patients with malignant diseases were counted when they were diagnosed with them within 6 months since GC was started for PMR or RS3PE syndrome. It was thought that cases in the intermediate group included cases that would be reclassified to the withdrawal group or the continuation group if they were observed for a longer time. It was expected that the parameters associated with GC continuation in the intermediate group would be between those of the withdrawal group and the continuation group and totaled the parameters in the intermediate group.

This survey was based on a chart review, but a telephone poll of patients whose charts could not be reviewed was conducted.

Statistical analyses were performed with IBM SPSS Statistics, version 27. KaplanMeier curves were compared using the Log rank test. Comparisons between the withdrawal group and the continuation group were made with Students t-test for independent continuous variables, and the chi-squared test was used to compare categorical data between groups.

This investigation protocol adopted the ethical guidelines of the 1975 Declaration of Helsinki and was approved by the Ikeda City Hospital Ethics Committee (approval number A20010). The data accessed from the medical records were de-identified in this report. Because this report contains no individual persons data, and this investigation was observational and noninterventional, the Ikeda City Hospital Ethics Committee waived the need for patient consent.

There were 58 cases diagnosed by Birds criteria, 43 cases diagnosed by the EULAR/ACR polymyalgia rheumatica interim standard of classification, and 4 cases with pure RS3PE syndrome. There were 32 cases in the withdrawal group and 17 cases in the continuation group.

The dose of GC could be reduced from the initial dose in all patients.

The duration from the onset of symptoms to GC therapy starting was 79.579.6 days (meanSD) in all patients.

The GC continuation rate 7.5 years after starting GC was 52.5% in all patients, 69.2% in women, and 27.1% in men. The rates remained unchanged for 15 years (Figures 1 and 2). The GC continuation rate was significantly higher in women (Log rank test). No patients were prescribed immunosuppressants.

Figure 1 Glucocorticoid continuation rate of all cases (KaplanMeier method).

Figure 2 Glucocorticoid continuation rate by sex (KaplanMeier method). The glucocorticoid continuation rate is higher in women (P=0.020).

P values by Students t-test or the chi-squared test are shown in the table for the other survey items, including numbers of cases, with data displayed as mean standard deviation (Table 1). Since there was a case with suspected temporal arteritis at the beginning in the withdrawal group that was treated with prednisolone 50 mg/day, this case was excluded as an abnormal value for the GC maximum dose and the GC dose half a year after its initiation. In the withdrawal group, there were few women (P=0.016). In all patients, hemoglobin levels (mean SD) before starting GC were lower (10.51.6 g/dL) in women than in men (11.41.8 g/dL) (P=0.014). In the withdrawal group, they were 10.91.5 g/dL in women and 11.91.6 g/dL in men. In the continuation group, they were 10.41.9 g/dL in women and 10.02.8 g/dL in men. Relapses were fewer in the withdrawal group than in the continuation group (P=0.0003).

Table 1 Clinical Features of the 105 Patients

In this study, the duration of GC treatment for PMR and RS3PE syndrome in Japanese patients was longer than in previous reports in English.12 GC treatment was needed for a longer time in women than in previous reports, and it was difficult to stop GC treatment in cases with severe anemia.

Although it has been reported that female sex is a risk factor for long-term GC treatment by Narvez et al13 and Cimmino et al,14 there has been no English-language report from Japan. Aoki et al reported the GC treatment duration of Japanese PMR patients, and they found no difference between men and women.12 They classified their patients into two groups by whether they had stopped GC therapy as of 24 months. Therefore, their observation period was shorter than in the present study. Two important factors causing sex-based disparities are genetics and sex hormones.15 Estrogen enhances B cell differentiation and immunoglobulin production.16 Several studies reported an immunosuppressive role of testosterone on different components of the immune system.15 Furthermore, the small number of GC receptors or low GC receptor affinity in woman may have an effect.14

In the present study, although the GC continuation rate fell to 55.6% at 7 years and 5 months, it did not fall further. The necessity for continuing GC differed among reports. Aoki et al reported that the median time of remission was 16 months.12 Cimmino et al reported that about 26% of the patients required GC treatment for 6 years or more.14 Shbeeb et al reported that the median GC treatment period was 5.95 years.17 Although the present study found a longer GC treatment period than previous reports, a prudent approach to GC dose reduction may have been one reason, given the report of a certain rate of disease recurrence in the report by Aoki et al.12 In 17 patients in the continuation group, three patients have not relapsed. This may show that our treatment is sometimes passive with respect to reducing the GC dose. Although there may have been a bias among certain institutions for longer GC treatment, the bias among institutions cannot explain the sex difference. There sometimes were newly suspected patients who received no GC treatment while waiting for spontaneous resolution. This study included no spontaneously resolved patients. The duration from the onset of symptoms to the start of GC therapy in all patients was over 2 months. This might be one reason for the long GC continuation in this study, and it might be a characteristic of Japanese/Asian people.

There are many more female than male patients. In Ikeda City, in which our hospital is located, the population aged 60 years and over as of March 31, 2020, included 18,880 women and 14,506 men.18 Therefore, it cannot be said that the incidence is higher among women than men.

Since PMR and the RS3PE syndrome often merged, they were considered together in one group.1,19 Since there are few pure RS3PE syndrome cases, whether there are differences between PMR and RS3PE syndrome in GC continuation and by sex is unknown. Aoki et al observed peripheral edema in 41 of 93 PMR patients.12 Such cases would be considered combined PMR and RS3PE syndrome cases according to the definition used in the present study.

Origuchi et al reported that GC dose and CRP 1 year after starting therapy were high in men with RS3PE syndrome.20 In the report by Origuchi et al, the observation period was 1 year, shorter than in other reports.20 In the present study, the womens continuation rate was also not high until 1.4 years after starting.

The present data showed severe anemia in the GC continuation group. Narvez et al reported that hemoglobin levels were low in women with PMR.13 They considered that anemia reflects intense inflammation. The range of ferritin was large for every case, and the difference between the withdrawal group and the continuation group was not clear. Ferritin levels were high in all groups, reflecting the impaired iron utilization in these diseases.

As a limitation of the present investigation, the number of patients was insufficient for a multivariable statistical analysis of the GC continuation rate and anemia. In addition, because some of the patients were interviewed by telephone, recall bias may have occurred. Because this study was retrospective, sex hormone levels were not measured before treatment; it is a future task to measure them in order to identify causes of the sex difference.

When considering the future treatment strategy for PMR and RS3PE syndrome in Japan, it is important to note that it is difficult to stop GC treatment for women and for those with severe anemia.

It is difficult to stop GC for PMR and/or RS3PE syndrome in women in Japan, especially in cases with severe anemia.

All authors have no conflicts of interest that should be declared. All authors take full responsibility for the content of this paper.

1. Mimori A. Polymyalgia rheumatica/PMR & remitting seronegative symmetrical synovitis with pitting edema/RS3PE (in Japanese). In: Mimori A. Physicians Notes on Rheumatology: The Process of Clinical Consideration. 4th ed. Tokyo, Japan: Japan medical journal; 2019:413426.

2. Partington RJ, Muller S, Helliwell T, Mallen CD, Sultan AA. Incidence, prevalence and treatment burden of polymyalgia rheumatica in the UK over two decades: a population-based study. Ann Rheum Dis. 2018;77(12):17501756. doi:10.1136/annrheumdis-2018-213883

3. Gonzalez-Gay MA, Vazquez-Rodriguez TR, Lopez-Diaz MJ, et al. Epidemiology of giant cell arteritis and polymyalgia rheumatica. Arthritis Care Res. 2009;61(10):14541461. doi:10.1002/art.24459

4. Nishioka K, Tanaka T. [Rheumatology: Progress in diagnosis and treatments. topics: III. Rheumatoid arthritis and allied conditions; 2. Allied conditions. 2) polymyalgia rheumatica]. Nihon Naika Gakkai Zasshi. 2014;103(10):24402448. Japanese. doi:10.2169/naika.103.2440

5. Hernndez-Rodrguez J, Cid MC, Lpez-Soto A, Espigol-Frigol G, Bosch X. Treatment of polymyalgia rheumatica: a systematic review. Arch Intern Med. 2009;169(20):18391850. doi:10.1001/archinternmed.2009.352

6. Muratore F, Pipitone N, Hunder GG, Salvarani C. Discontinuation of therapies in polymyalgia rheumatica and giant cell arteritis. Clin Exp Rheumatol. 2013;31(4 Suppl 78):S86S92.

7. Docken WP. Treatment of polymyalgia rheumatica [homepage on the Internet]. Wolters Kluwer; 2018 [Updated September 7, 2018]. Available from: https://www.uptodate.com/. Accessed February 21, 2020.

8. Bird H, Esselinckx W, Dixon AS, Mowat A, Wood P. An evaluation of criteria for polymyalgia rheumatica. Ann Rheum Dis. 1979;38(5):434439. doi:10.1136/ard.38.5.434

9. Dasgupta B, Cimmino MA, Kremers HM, et al. 2012 provisional classification criteria for polymyalgia rheumatica: a European league against rheumatism/American College of Rheumatology collaborative initiative. Arthritis Rheum. 2012;64(4):943954. doi:10.1002/art.34356

10. Arnett FC, Edworthy SM, Bloch DA, et al. The American Rheumatism Association 1987 revised criteria for the classification of rheumatoid arthritis. Arthritis Rheumatism. 1988;31(3):315324. doi:10.1002/art.1780310302

11. Dougados M, Linden SVD, Juhlin R, et al. The European Spondylarthropathy Study Group preliminary criteria for the classification of spondylarthropathy. Arthritis Rheumatism. 1991;34(10):12181227. doi:10.1002/art.1780341003

12. Aoki A, Kobayashi H, Yamaguchi Y. Predictors of long-term therapy with glucocorticoid in polymyalgia rheumatica. Modern Rheumatol. 2020;31(2):417426. doi:10.1080/14397595.2020.1777680

13. Narvez J, Nolla-Sol JM, Valverde-Garca J, Roig-Escofet D. Sex differences in temporal arteritis and polymyalgia rheumatica. J Rheumatol. 2002;29(2):321325.

14. Cimmino MA, Parodi M, Caporali R, Montecucco C. Is the course of steroidtreated polymyalgia rheumatica more severe in women? Ann N Y Acad Sci. 2006;1069(1):315321. doi:10.1196/annals.1351.030

15. Trigunaite A, Dimo J, Jrgensen TN. Suppressive effects of androgens on the immune system. Cell Immunol. 2015;294(2):8794. doi:10.1016/j.cellimm.2015.02.004

16. Moulton VR. Sex hormones in acquired immunity and autoimmune disease. Front Immunol. 2018;9:2279. doi:10.3389/fimmu.2018.02279

17. Shbeeb I, Challah D, Raheel S, Crowson CS, Matteson EL. Comparable rates of glucocorticoid-associated adverse events in patients with polymyalgia rheumatica and comorbidities in the general population. Arthritis Care Res. 2018;70(4):643647. doi:10.1002/acr.23320

18. Ikeda City Office [homepage on the Internet]. Populations in Ikeda city by age and sex (in Japanese); April 2, 2020. Available from: http://www.city.ikeda.osaka.jp/material/files/group/4/020331_nenreibetu.pdf. Accessed July 25, 2020.

19. Cantini F, Salvarani C, Olivieri I, et al. Remitting seronegative symmetrical synovitis with pitting oedema (RS3PE) syndrome: a prospective follow up and magnetic resonance imaging study. Ann Rheum Dis. 1999;58(4):230236. doi:10.1136/ard.58.4.230

20. Origuchi T, Arima K, Umeda M, et al. Clinical outcomes in the first year of remitting seronegative symmetrical synovitis with pitting edema (RS3PE) syndrome. Modern Rheumatol. 2017;27(1):150154. doi:10.1080/14397595.2016.1192744

Read more:
Glucocorticoid therapy withdrawal in polymyalgia rheumatica | IJGM - Dove Medical Press

Recommendation and review posted by Bethany Smith

Komal Jhaveri, MD, discussed the case of a 63-year-old patient with HER2-positive metastatic breast cancer during a Targeted Oncology Case-Base Roundtable event.

During a Targeted OncologyTM Case-Based Roundtable event, Komal Jhaveri, MD, the section head of the Endocrine Therapy Research Program, clinical director, of Early Drug Development Service, and a medical oncologist at Memorial Sloan Kettering Cancer Center in New York, NY, discussed the case of a 63-year-old patient with HER2-positive metastatic breast cancer.

Targeted OncologyTM: How typical is the presentation of this patient in your clinical practice, and what data are most relevant to you in choosing a treatment approach?

JHAVERI: According to the recently updated NCCN [National Comprehensive Cancer Network] guidelines in systemic therapy options for recurrent stage IV disease, we use a taxane with a dual HER2 blockade, which is trastuzumab and pertuzumab in the first line.1 This is obviously based on the unprecedented progression-free survival [PFS] and overall survival [OS] benefit in the CLEOPATRA trial [NCT00567190].2 The paclitaxel was based out of a phase 2 trial that was conducted by my colleague, Chau Dang, MD, here at Memorial Sloan Kettering Cancer Center, and we did not see any febrile neutropenia with the paclitaxel, so that has really become our go-to regimen in the first line.3

What data support treatment options in the second-line setting?

In the second-line setting, we have data from the EMILIA trial (NCT00829166), which led to T-DM1, or ado-trastuzumab emtansine, an antibody-drug conjugate [ADC], the first one for breast cancer.4 This is despite this trial not having patients progressing on trastuzumab, but in clinic this is our contemporary choice...for our patients. Additionally, we have a plethora of options that patients can receive on the third line and beyond. These include tucatinib [Tukysa] plus trastuzumab plus capecitabine [Xeloda] and fam-trastuzumab deruxtecan-nxki [Enhertu].1 We also have other chemotherapies with trastuzumab, such as capecitabine with trastuzumab and capecitabine with other TKIs [tyrosine kinase inhibitors]. There are targeted therapies, including a recent approval for margetuximab-cmkb [Margenza], an Fc-optimized antibody that was approved for use with chemotherapy in this last year.5

We have these many options, but what we do not know is the optimal sequence for third-line therapy and beyond.

Now, what is interesting here is that the triplet of tucatinib plus trastuzumab plus capecitabine, per the NCCN guidelines, is preferred in patients with both systemic and CNS progression on ado-trastuzumab emtansine.1 However, it may be given even in the second-line setting. Fam-trastuzumab deruxtecan is preferred in patients with visceral metastases [if there is disease] progression on ado-trastuzumab emtansine. [However, it is] contraindicated for patients with known pneumonitis or interstitial lung disease.

What makes this ADC special? Why is it distinct from T-DM1?

I think there are a few key attributes that we might want to think of [in] T-DXd [trastuzumab deruxtecan]. One is that the drug-to-antibody ratio [DAR] is 8 molecules of chemotherapy that can be delivered. Now, to put this into context when we think of T-DM1 therapy, that DAR is 3.5. So we are delivering more chemotherapy to the tumor cell. The payload itself is a topoisomerase I inhibitor, which we do not regularly use for our HER2- positive patients.

It is a highly potent payload, which the patients have not seen...in their regimens, and it has a very short half-life, so the free payload has a very short half-life, which is great. The linker is stable, and it is a tumor selective cleavable linker. Once this is delivered to the tumor, it is the pepsin in the tumor that makes the linker cleave and then deliver the payload. It is very tumor selective, so one can potentially expect less off-target toxicity.

And another important attribute to keep in mind is the bystander effect. It has membrane permeability, and so this payload can be membrane permeable and go to the neighboring cells, which might not necessarily be HER2 overexpressing. But that is why we have also seen activity in what we now call HER2 low, which is HER2 IHC 1+ and 2+ patients who have shortened benefit with T-DXd and other novel inhibitors that are also being developed and have this bystander effect. Of note, T-DM1 does not have that. And as we know, we do not utilize T-DM1 for patients who do not have HER2-amplified or HER2- expressive tumors.

Please discuss how the DESTINY-Breast01 trial [NCT03248492] has affected decisions for this patient.

[These data that] led to the approval of trastuzumab deruxtecan [were from] an open-label, multicenter, phase 2 trial [that enrolled] patients 18 [years] or older with unresectable metastatic breast cancer who had centrally confirmed HER2-positive disease and who had prior T-DM1.6,7 Patients with a history of interstitial lung disease were excluded.

This was because [of what] we had already learned from an adjudication committee that was put in place with the phase 1 experience of this agent. And stable, treated brain metastases were allowed to enroll on this trial. These patients were then enrolled in 2 parts. Part 1 included a PK [pharmacokinetic] stage and a dose-finding stage. In the PK stage, we studied 3 doses of which two, 5.4 mg/kg and the 6.4 mg/kg, moved on to the dose-finding stage. Based on the PK data and the safety data, the 5.4 mg/kg [dose] was selected to move forward and is the current recommended and approved dose.

This was the dose that moved into the continuation stage, where most of the patients had TDM-1 resistant disease. There were 4 patients who had TDM-1 intolerance, for a total of 184 patients [who] were treated with this dose, 5.4 mg/kg, with a primary end point of ORR [overall response rate]. One thing that I would like to highlight is that these patients have received up to 6 prior lines of therapy, and [approximately] two-thirds received prior pertuzumab therapy.

Additional baseline characteristics [also should be considered]. Median age was [approximately] 55 years. These were patients predominantly with a good performance state. [Approximately] 50% of these patients were hormone receptor positive. [Approximately] 84% had HER2 positivity by IHC 3+ expression. The remaining were IHC 2+ or 1+ or ISH [in situ hybridization] positive. Visceral disease was present in 92% of patients, of whom 57% had lung metastases. This is important to remember for the discussion of our case. Liver metastases were present in 30%, and the rest also had bone disease. There were 24 [13%] of these patients enrolled in the trial who also had stable, treated brain metastases.

How do the updated data from the DESTINY-Breast01 trial affect treatment decisions?

In the updated DESTINY-Breast01 trial data from June 2020, all but 4 patients had tumor shrinkage.8 The overall response rate, despite this [population being] heavily treated with a median of 6 prior lines of therapy, was 61.4%, including patients who had a complete response. The median duration of response, which is also very important to understand for our patients who are heavily pretreated, was 21 months in this phase 2 trial. And this response was seen rather early: time to response was 1.6 months. So if you are really worried about somebody with extensive disease or burdened disease, this was a quick response with this drug.

The median PFS in DESTINY-Breast01 was 19.4 months. This is rather impressive for such a heavily pretreated population. Just to put this into context and into perspective, when we think of other trials in the third line and beyond, whether it was the TH3RESA trial [NCT01419197] that studied TDM-1 with physician choice therapy, the NALA study [NCT01808573], the HER2CLIMB study [NCT02614794], or the SOPHIA trial [NCT02492711], the median PFS for the patient population [in the] third line [was approximately] 7 to 8 months.9-12 The PFS [in DESTINY-Breast02] of 19.4 monthsin such a heavily pretreated population, I think it is really unprecedented.8 The median OS data at 21 months was 25 months, but what I would really like to highlight here is that this is just 35% maturity of data, and we really need follow-up maturity now to understand the implications. [Approximately] 119 patients were already censored, and 17 were thought to have events at month 2, so at 18 months we had 74% alive, but [these are] still immature data.

Did these data show progression of disease in other areas?

A subgroup analysis for the 24 patients [13%] in the CNS subgroup was presented at ASCO [American Society of Clinical Oncology Annual Meeting] this year.13 Seventeen patients had brain lesions at baseline, and the data [were] available to evaluate responses in the brain for 15 of the 17. Though this was a small subgroup, it was important that there were responses seen in patients with stable brain metastases; 41% had a partial response and another 41% had disease stabilization in the brain. Again, this subgroup also had median of 6 lines of therapy, the same as the total population. Median follow-up here was 11 months, and...in the CNS subgroup, the ORR, PFS, and duration of response were comparable to those in the total patient population treated at the same dose. The median PFS in this population with brain metastases was 18 months [95% CI, 6.7-18.1]. There was also an additional case report where we saw 55% regression of a metastatic brain lesion.

The most common sites when we looked at progression were the liver, lung, and lymph nodes, which was similar in all patients total and the CNS subgroup. Meaning, once you were in the CNS subgroup or the total patient population and then you progressed, the common types of progression were within the liver, lungs, and lymph nodes. Progression in the brain was not as common. There were only 4 of 48 patients who had progressed in the brain, including 2 out of the 8 patients with baseline CNS metastases.

What is the safety profile of T-DXd?

I think the most common adverse events [AEs] that we see, and that were seen in more than 10% of the patients in the study, were nausea, vomiting, alopecia, fatigue, and neutropenia.14 But an important AE that we want to keep in mind is the drug-related ILD [interstitial lung disease] or pneumonitis. The ILD incidence that was reported initially with the August 2019 data cutoff was 25 patients [13%] who developed ILD or pneumonitis. The majority had grade 1 or grade 2 ILD or pneumonitis; however, there were 4 fatal events. The median time to developing ILD was 4.1 months. At the additional 1-year cutoff, and overall median cutoff, there were 3 additional ILD cases determined by the independent adjudication committee.

ILD events were seen mostly within the first 12 months, and after the 12-month mark, only 1 patient developed ILD, perhaps suggesting that ILD is not a cumulative AE. But this is something that we really must be aware of, and not just us, but also our frontline nursing staff who are fielding the calls when the patient calls in. [If there are symptoms that may indicate ILD or pneumonitis,] whether they have shortness of breath, a new cough, extreme fatigue, [we need] to quickly interrupt therapy, get pulmonology involved, and give patients steroids. [There are] patients who are asymptomatic, [so we have] to keep a very close eye on this to make sure that we are not missing anything.

We have become more trained look for these [potential AEs] given that there are so many classic agents with breast cancer that cause pneumonitis: checkpoint inhibitors and everolimus, and [also] CDK4/6 inhibitors....I think we all have become a little more vigilant about keeping a close eye on symptoms for our patients and for also for keeping an eye on the scans to make sure that we are not missing the so-called ground glass opacities for which we might want to interrupt or discontinue therapy. Fortunately, the heart events or cardiac events were very low, including left ventricular ejection fraction decreases or cardiac failure, as is seen with trastuzumab.

What was the design of the HER2CLIMB phase 2 trial?

[Lets move on] then to the phase 2 HER2CLIMB trial of tucatinib [Tukysa] and capecitabine and trastuzumab, which studied patients with HER2-positive metastatic disease who had prior treatment with trastuzumab, pertuzumab, and T-DM1.15 What was key in the study was that active brain metastases not needing local therapy were allowed, but they were not required. So you could have had treated, stable brain metastases, but you were also allowed to have active brain metastases. What is important to remember here is that these are patients with small tumors, less than 2-cm tumors, who do not have symptoms warranting local therapy. They did not require immediate radiation and they were [still] considered eligible.

[More than] 600 patients were enrolled410 in the tucatinib arm and 202 in the placebo arm.15 And they were well-balanced groups with a median age of [approximately] 55 years, all with predominantly good performance status; 60% were hormone receptor positive; overall, they had received 4 prior lines of therapy. Forty-eight percent had a history of brain metastasis. Of these 48%, 60% [of the brain metastases] were stable and treated. The remaining were what was called active, which could be untreated brain metastases. Untreated [meant] no local therapy or systemic therapy or [that they had been] treated in the past with some kind of local therapy but [were] progressing again. Even though they are progressing again, they are not symptomatic enough to warrant additional local therapy.

REFERENCES:

1. NCCN. Clinical Practice Guidelines in Oncology. Breast cancer, version 7.2021. Accessed August 20, 2021. https://bit.ly/2Y4zXiQ

2. Swain SM, Miles D, Kim SB, et al. Pertuzumab, trastuzumab, and docetaxel for HER2-positive metastatic breast cancer (CLEOPATRA): end-of-study results from a double-blind, randomised, placebo-controlled, phase 3 study. Lancet Oncol. 2020;21(4):519-530. doi:10.1016/S1470-2045(19)30863-0

3. Dang C, Iyengar N, Datko F, et al. Phase II study of paclitaxel given once per week along with trastuzumab and pertuzumab in patients with human epidermal growth factor receptor 2positive metastatic breast cancer. J Clin Oncol. 2014;33(5):442-447. doi:10.1200/JCO.2014.57.1745

4. Diras V, Miles D, Verma S, et al. Trastuzumab emtansine vs capecitabine plus lapatinib in patients with previously treated HER2-positive advanced breast cancer (EMILIA): a descriptive analysis of final overall survival results from a randomised, open-label, phase 3 trial. Lancet Oncol. 2017;18(6):732-742. doi:10.1016/ S1470-2045(17)30312-1

5. FDA approves margetuximab for metastatic HER2-positive breast cancer. News release. FDA. December 17, 2020. Accessed August 20, 2021. https://www.fda.gov/drugs/resources-information-approved-drugs/ fda-approves-margetuximab-metastatic-her2-positive-breast-cancer

6. Krop IE, Saura C, Yamashita T, et al. [Fam-] trastuzumab deruxtecan (T-DXd; DS-8201a) in subjects with HER2-positive metastatic breast cancer previously treated with T-DM1: a phase 2, multicenter, open-label study (DESTINY-Breast01). Abstract presented at: San Antonio Breast Cancer Symposium; December 10-14, 2019; San Antonio, TX. Accessed August 20, 2021. https://www.abstractsonline.com/ pp8/#!/7946/presentation/2039

7. Modi S, Saura C, Yamashita T, et al. Trastuzumab deruxtecan in previously treated HER2-positive breast cancer. N Engl J Med. 2020;382(7):610-621. doi:10.1056/ NEJMoa1914510

8. Modi S, Saura C, Yamashita T, et al. Updated results from DESTINY-breast01, a phase 2 trial of trastuzumab deruxtecan (T-DXd) in HER2 positive metastatic breast cancer. Abstract presented at: San Antonio Breast Cancer Symposium; December 8-11, 2020; virtual. Accessed August 20, 2021. https://www. sabcs.org/Portals/SABCS2016/2020%20SABCS/ALL%20ABSTRACTS%202-9. pdf?ver=2020-12-09-104626-337

9. Krop IE, Kim SB, Martin AG, et al. Trastuzumab emtansine vs treatment of physicians choice in patients with previously treated HER2-positive metastatic breast cancer (TH3RESA): final overall survival results from a randomised open-label phase 3 trial. Lancet Oncol. 2017;18(6):743-754. doi:10.1016/S1470-2045(17)30313-3

10. Saura C, Oliveira M, Feng YH, et al. Neratinib plus capecitabine vs lapatinib plus capecitabine in HER2-Positive metastatic breast cancer previously treated with 2 HER2-directed regimens: phase III NALA trial. J Clin Oncol. 2020;38(27):3138-3149. doi:10.1200/JCO.20.00147

11. Murthy RK, Loi S, Okines A, et al. Tucatinib, trastuzumab, and capecitabine for HER2-positive metastatic breast cancer. N Engl J Med. 2020;382(7):597-609. doi:10.1056/NEJMoa1914609

12. Rugo HS, Im SA, Cardoso F, et al. Efficacy of margetuximab vs trastuzumab in patients with pretreated ERBB2-positive advanced breast cancer: a phase 3 randomized clinical trial. JAMA Oncol. 2021;7(4):573-584. doi:10.1001/jamaoncol.2020.7932

13. Jerusalem GHM, Park YH, Yamashita T, et al. Trastuzumab deruxtecan (T-DXd) in patients with HER2+ metastatic breast cancer with brain metastases: a subgroup analysis of the DESTINY-Breast01 trial. J Clin Oncol. 2021;39(suppl 15):526. doi:10.1200/ JCO.2021.39.15_suppl.526

14. Jerusalem GHM, Park YH, Yamashita T, et al. CNS metastases in HER2-positive metastatic breast cancer treated with trastuzumab deruxtecan: DESTINY-Breast01 subgroup analyses. Ann of Oncol. 2020;31(suppl 2):S63-S64. doi:10.1016/j. annonc.2020.03.239

15. Enhertu. Prescribing information. Daiichi Sankyo, Inc; 2021. Accessed August 20, 2021. https://bit.ly/3hkf3mN

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Focus on These Data When Making Treatment Decisions in Breast Cancer - Targeted Oncology

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Aspirin-induced asthma (AIA) is a condition where asthma symptoms can develop after taking aspirin or other nonsteroidal anti-inflammatory drugs (NSAIDs). Its also known as aspirin-exacerbated respiratory disease (AERD) or Samters Triad.

The American Academy of Allergy, Asthma, and Immunology (AAAAI) estimates that 9 percent of adults have asthma and that 30 percent of adults who have asthma and nasal polyps may also have AERD.

Read on to learn more about the underlying causes and risk factors of AIA as well as how this condition may be treated.

Acetylsalicylic acid (aspirin) is a type of NSAID used to relieve pain, inflammation, and fever. Similar medications include ibuprofen (Advil) and naproxen (Aleve).

Aspirin and other NSAIDs interact with an enzyme known as cyclooxygenase-1 (COX-1). While the exact triggers are unknown, its thought that people with AIA have a sensitivity to the way these medications inhibit this enzyme.

You may be more prone to AIA if you have all three of these conditions:

A doctor may still recommend aspirin for the treatment of other conditions, such as preventing heart attacks or strokes, in cases where a person may have already experienced one of these conditions and when the benefits outweigh the risks of triggering asthma symptoms.

Symptoms of AIA tend to develop shortly after taking aspirin or other NSAIDs often within minutes or hours after exposure.

While its important to address any suspected symptoms of AIA with a doctor, heres a breakdown of mild symptoms as well as more severe symptoms that require immediate medical attention.

Mild symptoms of AIA may include:

More severe symptoms of AIA can make it harder to breathe even if you take a rescue inhaler. Although rare, these acute symptoms can be life threatening.

Seek emergency medical help if you experience the following:

People who develop AIA are usually between ages 20 and 50 years old and likely have a combination of:

You may also be more susceptible if you experience the following on a recurring basis:

Age is another consideration. You more generally can become more vulnerable to side effects from NSAIDs as you age past your 50s.

Its also possible that reactions to aspirin could be induced by drug allergies. Besides NSAIDs, other common drug allergies include:

Symptoms of AIA may also be further exacerbated if you also drink alcohol. The AAAAI estimates that 75 percent of people with the condition may experience symptoms after drinking alcohol along with aspirin use.

AIA is typically diagnosed with the help of an asthma specialist, such as an allergist, pulmonologist, or immunologist.

Theres not just one test that can diagnose AIA. Instead, a diagnosis is made with a combination of the following factors:

A doctor may also recommend ordering a test called an aspirin challenge to rule out drug allergies. This involves taking aspirin either in the doctors office or at the hospital while under medical supervision. Any reactions you have to taking aspirin can then be identified and treated.

Along with avoiding NSAIDs, treatment for AIA involves managing symptoms of asthma, sinusitis, and nasal polyps.

You can also talk with a doctor about the following options.

Home treatments can include:

A doctor may recommend one or more of the following medical treatments:

Besides exacerbated asthma symptoms, complications of AIA may include hives (urticaria). The AAAAI estimates that between 20 and 40 percent of people who have chronic hives may have worsening symptoms if they also experience AIA. A type of swelling called angioedema can also occur.

Its also important to consider long-term side effects of taking aspirin and other NSAIDs, especially when taken for longer than recommended. These include:

Avoid mixing aspirin with the following, too:

Aspirin is a type of NSAID primarily used to relieve pain. But be careful using aspirin if you have a history of asthma, sinusitis, and nasal polyps. These underlying conditions may put you at a higher risk of developing AIA.

Talk with a doctor if youre concerned about the risks or side effects of taking NSAIDs or if you have a history of side effects after taking these types of medications. They can help diagnose and treat potential AIA along with related medical conditions.

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What Is Aspirin-Induced Asthma? Causes, Symptoms & More - Healthline

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Leading clinicians at Allegheny General Hospital support first islet cell transplant to treat chronic pancreatitis.

PITTSBURGH (PRWEB) October 05, 2021

Allegheny Health Network (AHN) today announced a groundbreaking new capability for treating patients who suffer from chronic pancreatitis, an inflammation of the pancreas that occurs over many years and, in severe cases, can be life-threatening. Every year, more than 80,000 people are diagnosed with the disease, according to the National Pancreas Foundation.

Surgeons at Allegheny General Hospital (AGH) have joined a select group around the country performing islet cell transplantation to restore the functions of a diseased pancreas. AGHs Institute of Cellular Therapeutics, is one of just a few in the nation that specializes in islet cell isolation, a highly sophisticated process in which islet cells, such as those that produce insulin, are extracted from the patients removed pancreas and transplanted back into the body. For the past six years, the institutes Islet Cell Isolation Laboratory has extracted and processed these life-saving cells to assist in the care of patients at select islet cell transplant centers around the country. With the launch of its own transplant program, AHN becomes one of just a few medical centers in the nation able to provide the comprehensive therapy from start to finish.

Pancreatic islets are tiny clusters of cells scattered throughout the pancreas. Included among these islets are beta cells, which produce the hormone insulin that helps the body absorb glucose from the bloodstream and use it for energy. Diabetes develops when the pancreas does not make enough insulin, the bodys cells do not effectively use insulin, or a combination of both. Massimo Trucco, MD, Director of AHNs Institute of Cellular Therapeutics and an internationally preeminent diabetes researcher, leads the islet cell extraction team at the hospital.

The islet cell transplant is critical for patients with immense pain and have failed other therapies. Dr. Trucco and his staff collaborate with AHNs gastroenterology, abdominal transplant, endocrinology, psychology and social services teams to complete the procedure and provide wrap-around patient support.

Chronic pancreatitis is a debilitating disease that can lead to frequent hospitalizations, higher use of narcotic pain medication and a lower quality of life, said Abhijit Kulkarni, MD, FASGE, an AHN gastroenterologist who evaluates patients for the islet cell transplant procedure. This treatment really represents the pinnacle of pain management for pancreatitis care and can be a true lifeline for the most critically ill patients.

According to Rita Bottino, PHD, from AHNs Institute of Cellular Therapeutics, the poor condition of the pancreas can make extraction of islet cells challenging. We sometimes have to be a little creative in finding a way to inject enzymes into the organ that will break the matrix that holds the cells together. By injecting enzymes, clusters of cells or single cells will be released from the organ and those are the kind of insulin-producing cells that we ultimately want to transplant back to the patient.

Once the islet cells are extracted and ready for transplant, AHN surgeons Harry Williams, MD, Ngoc Thai, MD, PhD, and Tadahiro Uemura, MD, PhD, transplant the cells into the patients own liver through the portal vein. Called an autologous islet cell transplant (TPAIT), the cells continue to produce insulin to control blood sugar levels in the body, eliminating the risk of becoming diabetic.

So we remove the diseased organ, which is causing debilitating symptoms for the patient, while creating a new pathway for insulin production in the body. And the advantage of a smaller time interval between extraction and transplant of these cells is significant and we believe will result in even better outcomes for our patients, said Dr. Williams.

Thus far, AHN has completed one TPAIT procedure and prepared more than 100 islets for regional hospitals including UPMC and The Cleveland Clinic. In addition to treating patients, AHN supplies research donor islets to Mt. Sinai Hospital, Stanford University Medical Center, Vanderbilt University Medical Center, and the universities of Pennsylvania, Miami and San Francisco, among others.

Our ultimate goal when starting our islet cell isolation lab several years ago was to ultimately develop the transplant capabilities and become one of the few one stop shops for this highly specialized care in the country, said Dr. Thai, Director of AHNs Center for Abdominal Transplantation. Having some of the worlds foremost experts in this field at our institution, like Dr. Trucco and his team, has afforded us with an extraordinary opportunity to build an internationally leading program.

To learn more about AHNs Cellular Therapeutics Institute or Transplant program, please visit http://www.AHN.org.

About the Allegheny Health Network: Allegheny Health Network (AHN.org), a Highmark Health company, is an integrated healthcare delivery system serving the greater Western Pennsylvania region. The Network is composed of 13 hospitals, ambulatory surgery centers, Health + Wellness Pavilions, an employed physician organization, home and community based health services, a research institute, and a group purchasing organization. The Network provides patients with access to a complete spectrum of advanced medical services, including nationally recognized programs for primary and emergency care, trauma care, cardiovascular disease, organ transplantation, cancer care, orthopedic surgery, neurology and neurosurgery, womens health, diabetes, autoimmune disease and more. AHN employs approximately 21,000 people, has more than 2,500 physicians on its medical staff and serves as a clinical campus for Drexel University College of Medicine and the Lake Erie College of Osteopathic Medicine.

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AHN Performs Its First Islet Cell Transplants to Treat Chronic Pancreatitis - PR Web

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Otto Warburg probably would have been sent to a concentration camp if the Nazis werent hoping he could cure cancer.

Warburg was a Jewish gay man living openly in Berlin with his partner as Hitler rose to power. Warburg was also a biochemist, as brilliant as he was arrogant. In the 1920s, he discovered a hallmark of cancer, now called the Warburg effect. Malignant cells are ravenous for glucose, or blood sugar, consuming 10 times more than healthy cells. He dedicated his career to studying this strange metabolic anomaly because he believed it was the root cause of cancer.

He won the 1931 Nobel Prize for his work. But it was mostly forgotten by the 1950s, eclipsed by the molecular genetics revolution that set off a search for mutated, cancer-causing genes. He died in 1970 at age 86.

The rise, fall, and recent resurgence of research into cellular metabolism is the subject of Ravenous: Otto Warburg, the Nazis, and the Search for the Cancer-Diet Connection. Author Sam Apple, a journalist based in the Philadelphia suburb of Wyndmoor, weaves together this complex narrative in a way that makes arcane science accessible and fascinating.

The book is also thought-provoking for anyone interested in avoiding cancer and who isnt?

Its not obvious to me that there would be a breakthrough cancer therapy if Warburgs focus on cellular metabolism had not been shunted aside, Apple said in a recent interview. More likely, there would have been more attention to the relationship between our diets and the metabolism of the whole body and cancer. I think this could have had a dramatic impact on cancer prevention. But the link is still is not widely appreciated.

Otto Warburg was related to a wealthy German Jewish clan of bankers, scholars, and influencers. He was groomed for scientific greatness by his father, Emil Warburg, a leading physicist of the time.

The pinnacle of Otto Warburgs career happened to coincide with the rise of the Third Reich. When Warburg refused to sign a declaration of Aryan descent, the Nazi customs official tasked with asking Warburg to lie endeavored to get him punished by the Kaiser Wilhelm Society, the parent organization of Warburgs scientific institute.

Warburg not only managed to get off scot free, but he asked the Wilhelm Society president to ask the Reich Ministry of Finance to rewrite racial decrees so that non-Aryan institute directors would be treated like Aryan directors.

In 1934, at a moment when Hitler had already begun sending Germans to concentration camps, Otto Warburg, a gay man of Jewish descent, wanted Nazi laws rewritten according to his personal needs, Apple writes in the book.

As the author explains, cancer rates were inexplicably rising in Germany and other developed countries, and the Nazis fear of the disease ran almost as deep as their antisemitism and homophobia They despised Warburg, but needed his scientific genius.

Warburg invented several new tools to study the metabolism of cells, including the manometer, an instrument used to measure the force exerted by a gas or liquid.

He knew that in a healthy cell, blood glucose was normally converted to energy in a process using oxygen. This process, which involves enzymes that Warburg spent years identifying, occurs in the cells power stations, now known as mitochondria.

He discovered, to his amazement, that cancer cells broke all the metabolic norms. In addition to overconsuming glucose, the cells turned it into energy using an inefficient process that did not require oxygen even though plenty of oxygen was available.

The cancer cells were chopping glucose molecules in half and spitting the fragments right back out of the cell, Apple writes in an elegantly simple description of anaerobic glycolysis, or fermentation, the biochemical process that also gives us beer and wine. Cancer cells, Warburg realized, were fermenting glucose just as simple organisms like yeast and bacteria do.

But why? Warburg hypothesized more like proclaimed that cancer cells mitochondria were somehow defective, so the cells had to resort to a backup power generator, namely fermentation.

Today, while the search for answers continues, the evidence suggests that fermentation is not a response to a defect. Rather, it gives malignant cells an advantage as they turn into uncontrollable, immortal renegades.

Glucose molecules are the building blocks that cancer needs to create daughter cells, Apple boiled it down during the interview. The most influential scientists now think its about the cancer cells bioenergetic needs.

Following the 1953 discovery of the structure of DNA the genetic instructions for everything cells do oncology researchers became focused on finding and fixing the defective genes that give rise to cancer.

In that postwar era, Warburgs focus on cell metabolism was seen as outmoded, like studying the fuel line in hopes of understanding a high-tech engine.

Beginning in the 1990s, however, some leading researchers realized that certain cancer-causing genes known for their role in cell division also regulated cells glucose consumption. One of those scientists, Chi Van Dang, former director of the University of Pennsylvanias Abramson Cancer Center and now scientific director at the Ludwig Institute for Cancer Research, showed that MYC, a family of genes regulating cell proliferation, also targets an enzyme that can turn on the Warburg effect.

Metabolism-centered cancer therapies that effectively starve tumors are no longer just a concept. Two drugs, ivosidenib and vorasidenib, have already been approved by the Food and Drug Administration for a form of leukemia and are now being tested in brain cancer patients. Rafael Pharmaceuticals experimental therapy, devimistat, has had impressive early results in bile duct cancer trials.

But as Apple points out, cancer is an incredibly persistent foe. It can mutate to evade chemotherapy, molecularly targeted therapies, and even newer immune-boosting therapies. The same thing may happen with metabolic therapies. Whats more, virtually all cancer treatments come with significant side effects.

Thats why the implications for preventing cancer in the first place are so important.

Population-wide studies have directly linked 13 cancers including breast, bladder, lung, colon, liver, and gynecological cancers to the same metabolic abnormalities that are driving the twin worldwide epidemics of obesity and diabetes. The most striking thing that the cancers, obesity and diabetes have in common is resistance to insulin, the vital hormone that enables cells to absorb blood glucose and turn it into energy. To compensate for this resistance, the pancreas pumps out more and more glucose.

The hypothesis is that patients high blood sugar impacts tumor growth by providing cancer cells with an abundance of the fuel they thrive on.

Apple spends almost half his book taking deep, incisive dives into research on the insulin connection. Refined sugar, which is a combination of glucose and fructose called sucrose, contributes to insulin resistance, So does fructose, or fruit sugar especially when it is concentrated in high-fructose corn syrup, the ubiquitous processed-food additive. Consuming fructose, a carbohydrate, also appears to make people add fat tissue more readily than actual fats, such as such as butter.

Precisely how much sugar is too much may be different for each person, depending on genes and age and exercise habits and capacity to store fat safely, he writes. But the path from refined sugar added to our diets to insulin resistance ... to cancer is now well understood and based on widely accepted science.

He touches only glancingly on a fundamental problem: Even if sugars role as a cancer-promoter becomes an article of faith, cutting back on it is tough. And controversy, not to mention quackery, abounds in the field of nutrition. The ketogenic diet, for example, restricts carbohydrates, which are converted to glucose in the body. The diet has shown promise in weight loss studies, as well as in relieving neurological disorders such as epilepsy. But the esteemed Mayo Clinic says the diets high level of saturated fats, combined with limits on nutrient-rich fruits, veggies and grains, is a concern for long-term heart health.

In the final chapter, Apple weaves in a chilling anecdote:

Sugar, of course, cannot be blamed for Nazism or for turning Hitler into a madman. But as his madness grew, so, too, did his taste for sweets. It wasnt only his cherished Viennese pastries. On any given day, Hitler might consume two full pounds of chocolates. He even added sugar to his wine.

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An old idea from a German Jewish scientist spared by the Nazis is getting new life: Prevent and treat cancer by cutting out sugar - The Philadelphia...

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INTRODUCTION Clustered regularly interspaced short palindromic repeats (CRISPR) are a family of DNA sequences, which constitute a primitive immune system that is responsible for protecting prokaryotic cells from phage infections.

New York, Aug. 24, 2021 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "CRISPR Based Therapeutics Market by Type of Therapy, Therapeutic Approach, Therapeutic Area, and Key Geographical Regions : Industry Trends and Global Forecasts, 2021-2030" - https://www.reportlinker.com/p06130494/?utm_source=GNW It was first described in 1987, however, its potential as a gene editing tool was not realized until 2012. Since then, the CRISPR revolution has not shown any signs of slowing down and has been responsible for significant advances in molecular biology and therapy development. Fundamentally, the CRISPR/Cas system involves specific palindromic DNA sequences which work in tandem with a family of caspase enzymes (Cas9, Cas12), in order to excise gene fragments with high precision. Compared to the other targeted nuclease-based systems, CRISPR is relatively faster, and cost-efficient; as a result, the demand for this gene editing tool is very high. The relatively recent discovery / development of novel accompanying nucleases, namely Cas12a, Cas13, Cas14 and dCas9, has significantly improved the precision of this technology. Presently, there are several companies using different variants of the CRISPR/Cas technology for basic research, and the development of gene editing solutions. However, the therapeutic use of this versatile genetic manipulation tool is only being investigated by a select few stakeholders in the pharmaceutical industry. The aforementioned scenario is attributed to the surrogate licensing model, which has granted exclusive control of the associated intellectual property (IP) to three leading players, namely Editas Medicine, CRISPR Therapeutics and Intellia Therapeutics, in the contemporary market.

Clinical trials of CRISPR based therapeutics are currently focused mainly on oncological and hematological disorders; however, several product candidates against certain neurological disorders and infectious diseases, specifically targeting recurrent conditions, are under investigation. Post 2014, the overall interest in this technology has grown exponentially, with several start-ups entering the market and 6 of the top 10 pharmaceutical companies restructuring their efforts in this direction. Over time, a substantial body of evidence has also been generated validating the therapeutic applications of this technology, which has, in turn, prompted the establishment of numerous strategic partnerships (focused on therapy development and clinical research) and has caused investors to put in significant capital into innovator companies involved in this domain, over the last two years alone. In fact, the three leading companies in this industry segment together have combined market capitalization of more than USD 10 billion, and have raised more than USD 2.8 billion in various funding rounds. Despite the possibly limitless potential of the CRISPR/Cas technology, further investigation, probing its safety and therapeutic efficacy in large diverse populations, is required. Key impediments to approval and other existing challenges that are being addressed by stakeholders, include off-target toxicity-related concerns and complexities related to the delivery of CRISPR components into target cells. Concerning delivery, innovators in this field have reported notable success using different types of platforms for facilitating the intracellular administration of CRISPR components; examples of successful delivery methods include electroporation, AAV vectors and lipid nanoparticles (LNPs). A few companies are also evaluating bacteriophages as a potential delivery system for such products. Promising clinical results, and ongoing technical developments, coupled to the growing interest of biopharmaceutical developers, are anticipated to push pipeline products to higher phases and on to commercialization. We believe that the market is likely to evolve at a commendable pace over the next decade.

SCOPE OF THE REPORT The CRISPR Based Therapeutics Market, 2021-2030 report features an extensive study of the current market landscape and future opportunity for the players involved in the development of CRISPR based therapeutics for the treatment of a variety of disease conditions. The study presents an in-depth analysis, highlighting the capabilities of various stakeholders engaged in this domain, across different geographies. Amongst other elements, the report includes: A review of the CRISPR based therapeutics that are currently in different stages of development. It features a detailed analysis of pipeline molecules, based on several relevant parameters, such as target therapeutic area (autoimmune disorders, cardiovascular disorders, dermatological disorders, genetic disorders, hematological disorders, immunological disorders, infectious diseases, inflammatory disorders, metabolic disorders, muscular diseases, neurological disorders, oncological disorders, ophthalmic diseases and others), phase of development (discovery, preclinical and clinical), approach of therapy (ex vivo and in vivo), cell source (autologous and allogeneic), type of therapy (CAR-T therapy, HSC therapy, T cell therapy, Phage therapy and others), and the type of technology used. It also includes information on the completed, ongoing and planned clinical trials for CRISPR based therapeutics, sponsored by various industry players. Elaborate profiles of key players in this domain. Each company profile features a brief overview of the company, its financial information (if available), a brief description of its therapeutic candidates, recent developments, and an informed future outlook. An in-depth analysis of around 2,000 patents related to CRISPR technology that have been filed / granted, since 2015, highlighting the key trends associated with these patents, across type of patent, publication year and application year, regional applicability, IPCR symbols, emerging focus areas, inventor information, leading patent assignees (in terms of number of patents filed / granted), patent benchmarking and valuation. An analysis of the partnerships that have been inked by various stakeholders engaged in the development of CRISPR based therapeutics, during the period 2014-2020, covering research and licensing agreements, R&D agreements, licensing agreements, licensing and manufacturing agreement, product development and manufacturing agreements, joint ventures and other types of partnership deals. An analysis of the investments made at various stages of development of the companies engaged in this field, covering instances of seed financing, venture capital financing, grants / awards, capital raised from IPOs and subsequent offerings. An analysis of the start-ups (established in the time period between 2013-2020 and have less than 200 employees) engaged in the development of CRISPR based therapeutics, based on several parameters, such as number of candidates in discovery, preclinical and clinical phase of development, therapeutic area, amount raised through funding, number of investors, type of funding, number of deals signed, and number of patents filed.

One of the key objectives of the report was to estimate the future growth potential of CRISPR based therapeutics market, over the coming decade. Based on multiple parameters, such as target patient population, likely adoption rates and expected pricing, we have provided informed estimates on the financial evolution of the market for the period 2021-2030. For this purpose, we have segmented the future opportunity across [A] target therapeutic area (hematological disorders, oncological disorders, ophthalmic diseases, infectious diseases and others) [B] approach of therapy (ex vivo and in vivo), [C] type of therapy (CAR-T cell therapy, HSC therapy, T cell therapy, and TIL), [D] key geographical regions (North America, Europe and Asia-Pacific). To account for uncertainties and to add robustness to our model, we have provided three market forecast scenarios, portraying the conservative, base and optimistic tracks of the anticipated industrys growth.

KEY QUESTIONS ANSWERED Who are the leading players engaged in the development of CRISPR based therapeutics? Which key clinical conditions can be treated by CRISPR based drugs? What are the investment trends in this industry? Which partnership models are commonly adopted by stakeholders engaged in this domain? How has the intellectual property landscape in this market evolved over the years? Which factors are likely to influence the evolution of this market? How is the current and future market opportunity likely to be distributed across key market segments?

RESEARCH METHODOLOGY The data presented in this report has been gathered via secondary and primary research. For all our projects, we conduct interviews with experts in the area (academia, industry and other associations) to solicit their opinions on emerging trends in the market. This information is primarily useful for us to draw out our own opinion on how the market will evolve across different regions and technology segments. Wherever possible, the available data has been validated from multiple sources of information.

The secondary sources of information include: Annual reports Investor presentations SEC filings Industry databases News releases from company websites Government policy documents Industry analysts views

While the focus has been on forecasting the market till 2030, the report also provides our independent views on various non-commercial trends emerging in this industry. This opinion is solely based on our knowledge, research and understanding of the relevant market trends gathered from various secondary and primary sources of information.

CHAPTER OUTLINES Chapter 2 is an executive summary of the key insights captured in our research. It offers a high-level view on the current state of the CRISPR therapeutics market and its likely evolution in the short-mid to long term.

Chapter 3 provides a general introduction to CRISPR/Cas system. In addition, we have briefly described the components of CRISPR/Cas system, its mechanism of action and vehicles to deliver CRISPR/Cas components in to the target cells. The chapter lays emphasis on the applications of CRISPR technology. It also includes a discussion on the challenges associated with the use of CRISPR based therapeutics.

Chapter 4 presents a detailed assessment of the current market landscape of CRISPR based therapeutics, along with information on type of therapy (CAR-T therapy, HSC therapy, T cell therapy, Phage therapy and others), approach of therapy (in vivo and ex vivo), cell source (autologous and allogeneic), phase of development (discovery, preclinical and clinical), type of delivery vehicle used (viral vector, electroporation, LNPs, bacteriophage and others), target disease indication and therapeutic area (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, immunological disorders, infectious diseases, inflammatory disorders, metabolic disorders, muscular diseases, neurological disorders, oncological disorders, ophthalmic diseases and others). In addition, it provides an overview of the CRISPR based therapeutics developer landscape, highlighting the players that are active in this domain. It includes information on their year of establishment, company size (in terms of number of employees) and location of headquarters of the drug developers. We have presented a logo landscape, highlighting the distribution of the drug developers based on company size and location of headquarters. Further, it presents an analysis on the initiatives of the big pharma companies in this domain.

Chapter 5 includes profiles of the key players engaged in the development of CRISPR based therapeutics (shortlisted based on strength of product portfolio). Each profile features a brief overview of the company, its financial information (if available), brief details of gene editing technology, therapeutic pipeline, recent developments and an informed future outlook.

Chapter 6 provides an in-depth analysis of the patents filed / granted for CRISPR technology since 2015. The analysis also highlights the key trends associated with these patents, including type of patent (granted patent, patent application and others), publication year, application year, geographical location / patent jurisdiction (North America, Europe, Asia-Pacific and Rest of the World), IPCR symbols, key inventors and leading industry / non-industry players. In addition, it includes a detailed patent benchmarking analysis of leading players and patent valuation analysis, which evaluates the qualitative and quantitative aspects of these patents.

Chapter 7 features a detailed analysis of the partnerships and collaborations that have been inked in this domain since 2014, covering research and licensing agreements, R&D agreements, licensing agreements, licensing and manufacturing agreement, product development and manufacturing agreements, joint ventures and other types of partnership deals. The chapter includes analysis based on year of partnership, type of partnership model, purpose of licensing deal, and most active player(s) (in terms of number of partnerships inked). In addition, the chapter features a discussion on the surrogate licensing practice in the CRISPR based therapeutics market.

Chapter 8 provides an analysis of the investments made since 2014 at various stages of development of companies engaged in this domain, based on the year of investment, number of funding instances, amount invested and type of funding, highlighting most active players (in terms of number of funding instances and amount raised) and most active investors (in terms of number of funding instances).

Chapter 9 presents an analysis of the start-ups (established after 2012 and having less than 200 employees) engaged in the development of CRISPR based therapeutics based on the parameters, such as number of candidates in discovery, preclinical and clinical phase of development, target therapeutic area, amount raised through funding, number of investors, type of funding, number of deals signed, and number of patents filed.

Chapter 10 features an elaborate discussion on the future market potential of various CRISPR based therapeutics. The chapter provides insights on the likely distribution of the current and forecasted opportunity across [A] target therapeutic area (hematological disorders, oncological disorders, ophthalmic diseases, infectious diseases and others) [B] approach of therapy (ex vivo and in vivo), [C] type of therapy (CAR-T cell therapy, HSC therapy, T cell therapy, and TIL) and [D] key geographical regions (North America, Europe and Asia-Pacific).

Chapter 11 is a summary of the overall report, highlighting the key facts and figures related to the research and analysis presented in the previous chapters.

Chapter 12 is a collection of interview transcripts of discussions held with representatives of renowned organizations engaged in the CRISPR technology domain. In this chapter, we have presented the insights on our conversation with Harrison Wong (Public Relations, Burns McClellan, for eGenesis).

Chapter 13 is an appendix that contains tabulated data and numbers for all the figures provided in the report.

Chapter 14 is an appendix, which consists the list of companies and organizations mentioned in the report.Read the full report: https://www.reportlinker.com/p06130494/?utm_source=GNW

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CRISPR Based Therapeutics Market by Type of Therapy, Therapeutic Approach, Therapeutic Area, and Key Geographical Regions : Industry Trends and Global...

Recommendation and review posted by Bethany Smith

This article was originally published here

Life Sci. 2021 Aug 25:119908. doi: 10.1016/j.lfs.2021.119908. Online ahead of print.

ABSTRACT

Genetic disorders and congenital abnormalities are present in 2-5% of births all over the world and can cause up to 50% of all early childhood deaths. The establishment of sophisticated and controlled techniques for customizing DNA manipulation is significant for the therapeutic role in such disorders and further research on them. One such technique is CRISPR that is significant towards optimizing genome editing and therapies, metabolic fluxes as well as artificial genetic systems. CRISPR-Cas9 is a molecular appliance that is applied in the areas of genetic and protein engineering. The CRISPR-CAS system is an integral element of prokaryotic adaptive immunity that allows prokaryotic cells to identify and kill any foreign DNA. The Gene editing property of CRISPR finds various applications like diagnostics and therapeutics in cancer, neurodegenerative disorders, genetic diseases, blindness, etc. This review discusses applications of CRISPR as a therapeutic in various disorders including several genetic diseases (including sickle cell anemia, blindness, thalassemia, cystic fibrosis, hereditary tyrosinemia type I, duchenne muscular dystrophy, mitochondrial disorders), Cancer, Huntingtons disease and viral infections (like HIV, COVID, etc.) along with the prospects concerning them. CRISPR-based therapy is also being researched and defined for COVID-19. The related mechanism of CRISPR has been discussed alongside highlighting challenges involved in therapeutic applications of CRISPR.

PMID:34453943 | DOI:10.1016/j.lfs.2021.119908

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Applications of CRISPR as a potential therapeutic - DocWire News

Recommendation and review posted by Bethany Smith

CRISPR Clinopathomics is an emerging science with applications in biotechnology and cellular biology. It is a genome-wide, single-gene-based technology to which various pharmacological agents can be added to promote disease resistance. It has great potential to address all life sciences challenges with an approach that allows for the production of multiple proteins that can attack many of lifes common challenges. This technology can potentially address most life-threatening diseases such as cancer, infections, inflammation, inherited disorders, diabetes, aging, neurological and other disorders, and chronic conditions, and possibly more.

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*The Sample only consist ofTable of Content (ToC).Research Frameworkof the actual report.Research Methodologyadopted for it.

Major Company Profiles Covered in This Report:Caribou Biosciences Inc., CRISPR Therapeutics, Mirus Bio LLC, Editas Medicine, Takara Bio Inc., Synthego, Thermo Fisher Scientific, Inc., GenScript, Addgene, Merck KGaA (Sigma-Aldrich), Integrated DNA Technologies, Inc., Transposagen Biopharmaceuticals, Inc., OriGene Technologies, Inc., New England Biolabs, Dharmacon, Cellecta, Inc., Agilent Technologies, and Applied StemCell, Inc.

Technological advancements in the field of genome editing are expected to drive growth of the global CRISPR and CAS gene market during the forecast period. Key biopharmaceutical companies across the globe are focused on research and development activities, in order to innovate novel technologies in genome editing. For instance, in September 2020, Intellia Therapeutics Inc. presented new data demonstrating the efforts of In Vivo CRISPR/CAS9 edits to reduce a disease-causing protein or restore a functional protein.

Furthermore, in December 2020, Editas Medicine Inc., a genome editing company, submitted an investigational New Drug (IND) to the U.S. Food and Drug Administration (FDA), indicated for sickle cell disease. Such advancements and R&D activities have increased the demand for genome editing. Hence, these factors are expected to drive growth of the global CRISPR and CAS gene market during the forecast period. Moreover, increasing research in plant genome editing programs is expected to boost the global CRISPR and CAS gene market growth over the forecast period.

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However, off-target effects associated with CRISPR technology such as deletions, unintended point mutations, and translocations are expected to hamper the global CRISPR and CAS gene market growth over the forecast period. Among regions, North America is expected to witness significant growth in the global CRISPR and CAS gene market during the forecast period. This is owing to various companies involved in gene editing and the development of novel therapeutics across the region. Moreover, Asia Pacific is expected to register a robust growth rate over the forecast period, owing to increasing popularity of CRISPR technology in the region.

Key companies involved in the global CRISPR and CAS gene market are CRISPR Therapeutics, Inscripta, Inc., AstraZeneca, Mammoth Biosciences, Addgene, Synthego, Caribou Biosciences, Inc., Takara Bio, Inc., Cellectis, New England BioLabs, Editas Medicine, Inc., Merck KGaA, F. Hoffmann-La Roche Ltd., Intellia Therapeutics, Inc., and Danaher Corporation.

For instance, in November 2019, Caribou Biosciences Inc., a CRISPR genome editing company, announced the results of a new study demonstrating human genome engineering with TYPE I CRISPR-CAS systems.

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Some Notable Offerings by Coherent Market Insights Report on CRISPR and CAS Gene market:

We will provide you an analysis of the extent to which this CRISPR and CAS Gene market research report acquires commercial characteristics along with examples or instances of information that helps you to understand it better.

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The data provided in the CRISPR and CAS Gene market report offers comprehensive analysis of important industry trends. Industry players can use this data to strategize their potential business moves and gain remarkable revenues in the upcoming period.

The report covers the price trend analysis and value chain analysis along with analysis of diverse offering by market players. The main motive of this report is to assist enterprises to make data-driven decisions and strategize their business moves.

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Recommendation and review posted by Bethany Smith

Shape Therapeutics CEO Francois Vigneault. (Shape Photo)

Shape Therapeutics has signed a deal potentially exceeding $3 billion with pharma giant Roche to support the development of gene therapies for Alzheimers and Parkinsons disease, the Seattle company announced Wednesday.

Shapes RNA editing technologies can change the sequence of RNA, which encodes the bodys protein building blocks. The company will deploy this technology with Roche in preclinical studies against targets relevant for these neurological conditions as well as certain rare diseases.

The collaboration may also leverage the biotech companys technologies for gene delivery. This system is designed to deliver RNA editing technology or other payloads directly to specific areas of the body, such as the nervous system or muscle.

Shape aims to unlock the next breakthrough in RNA technologies in the gene therapy space across a wide range of therapeutic areas, said Francois Vigneault, co-founder and CEO, in a statement.

Shapes RNA editing technology could potentially be used to change the amount of a key regulatory protein in the body or treat genetic diseases. The companys gene delivery technology is based on AAV vectors, a platform currently used to treat several rare conditions.

Under the agreement, Shape is eligible to receive an initial payment as well payments for hitting development, regulatory and sales milestones potentially exceeding $3 billion in aggregate value. Any products from the collaboration will be developed and commercialized by Roche.

The collaboration comes on the heels of a recent $112 million Series B investmentin June for the biotech, founded in 2018 with RNA editing technology spun out of the lab of co-founderPrashant Mali, a bioengineer at the University of California, San Diego.

Vigneault was former VP of research at Juno Therapeutics, a Seattle cell therapy company acquired by Celgene in 2018 for $9 billion. Other Juno veterans include Shapes head of platform technologies Adrian Briggs and vice president and head of research David Huss.

The new collaboration may reflect growing drug company interest in neurosciences, which is on the upswing after a lull several years ago. The recent FDA approval of Biogens Alzheimers drug Aduhelm may also be propelling interest in the field.

This new collaboration is also perfectly aligned with our broader efforts across the Roche Group to unlock the full potential of gene therapy, said James Sabry, Head of Roche Pharma Partnering, in a statement.

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Seattle biotech firm Shape Therapeutics inks gene therapy deal with Roche worth up to $3B - GeekWire

Recommendation and review posted by Bethany Smith

Parkinsons disease is the second most common neurodegenerative disorder in the United States, affecting at least 500,000 people. Its a progressive disease, but can be managed with treatment. For now, the condition is diagnosed by symptoms rather than specific lab tests.

By the time a formal diagnosis is made, the National Institute of Neurological Disorders and Stroke (NINDS) notes that the disease has usually progressed to a point where people have difficulty controlling bodily movements.

Thats why its important to know whether its possible to prevent this disease.

Currently, there is no therapy or treatment that can slow the progression of Parkinsons or effectively relieve advanced symptoms, according to the NINDS.

By the time classic motor symptoms of Parkinsons disease show up, a significant loss of brain cells and function have already occurred. Scientists are investigating ways to detect early signs of the disease, to potentially stop or slow the progression.

Researchers aim to learn more about biomarkers of the early stages of the disease. Finding reliable biological identifiers might help doctors diagnose and treat Parkinsons earlier. Identifying these signs would give them more time to try therapeutic treatments before the disease has progressed.

For example, research indicates that it may be useful to study the activity of a neuronal protein in the brain known as -synuclein, or alpha-synyclein.

A 2019 study noted that measurements of -synuclein have shown encouraging preliminary results with regard to potential early diagnosis. Another 2019 study also examined how -synuclein accumulates in the brains of people suspected of having Parkinsons disease.This information could be used to develop therapies, perhaps antibody therapy, to prevent that accumulation from happening.

Scientists are also working to learn more about environmental factors and genetic factors that might contribute to the risk of developing Parkinsons. One recent genetic research breakthrough is the development of a DNA chip called NeuroX, which could potentially determine a persons risk, but more research is needed.

Parkinsons disease is the result of complicated combination of interconnected events, as one 2016 study described it. Since aging is the most common risk factor, future treatments may need to take degeneration of certain neurons into account.

While its not yet known if there are surefire ways to prevent Parkinsons disease, there are a few things experts recommend.

For example, you might try incorporating physical activity into your routine and eating a healthy and balanced diet for a variety of health reasons. So far, research into nutritional supplements is lacking. However, if you have specific dietary needs, talk to your doctor to see if supplementation is appropriate.

Could CBD oil help? Its possible, but we dont know for sure yet. Some research, including a 2018 review of studies, suggest that cannabidiol (CBD) might help prevent Parkinsons disease. However, the studies are mostly animal studies and there is not yet a body of research involving humans and CBD.

Some experts suggest that you may be able to delay some of the effects of Parkinsons disease through regular physical activity. Ideally this would include a combination of exercise that includes:

The NINDS has funded a number of studies to learn more about the impact of exercise, including whether exercise might help people delay the need for medication.

There are a variety of options for treating and managing Parkinsons symptoms, most of which involve medications that address the brains low levels of dopamine. Dopamine is a chemical in your brain that affects movement, and Parkinsons causes your brain to lose neurons that produce this chemical.

Medications that address this include levodopa, or levodopa combined with carbidopa. Or your doctor might prescribe a dopamine agonist, which mimics the action of dopamine in your brain. Other drugs used to treat Parkinsons include:

Another possible treatment option is deep brain stimulation (DBS). DBS was approved by the U.S. Food and Drug Administration in 1997. Many people have found that this treatment, which involves sending electrical impulses into the brain via tiny electrodes, helps control tremors once treatment with levodopa is no longer effective.

A small 2018 study found that DBS seemed to slow the progression of tremors in people with Parkinsons disease. It also found that DBS could be used effectively in people with an earlier disease stage than previously thought.

Scientists hope that more treatments may become available in the future, as they learn more about which drug may or may not be effective at slowing or halting the progression of the disease.

For example, a randomized, double-blind trial of 62 patients found that people with Parkinsons who took a drug usually used to treat diabetes seemed to stop the progression of the Parkinsons symptoms. They received weekly injections of exenatide for 48 weeks.

It was a relatively small study, and longer-term trials are needed, according to the researchers. A larger study involving more patients is currently ongoing.

If you are already living with Parkinsons disease, here are some tips to manage it:

There are medications that can help treat the symptoms of Parkinsons disease, and scientists are currently conducting research that could result in new treatment and therapies.

For example, you might one day have the option to take a medication used to treat prostate gland enlargement if youre at risk for developing Parkinsons disease.

The results of a study published in early 2021 suggest that certain medications often used to treat enlarged prostates are associated with a decreased risk of developing Parkinsons disease. Specifically, the researchers compared terazosin, doxazosin, and alfuzosin, which enhance energy metabolism, to tamsulosin, which is also used to treat benign enlarged prostates. They found that the latter did not seem to have the same effect.

The findings built on their previous research, which suggested that the use of terazosin, doxazosin, and alfuzosin was associated with slower progression and fewer complications in people with Parkinsons disease.

Researchers are also looking into the potential of stem cells to create new neurons to produce dopamine. They are also researching a protein called glial cell-derived neurotrophic factor, or GDNF, to potentially slow the progression of Parkinsons.

Ongoing research into a gene called LRRK2 or LARK2 and how it may interact with other genes related to Parkinsons disease is also promising, as it may shed light on how the disease progresses and how it might be halted.

For now, the symptoms of Parkinsons disease can be managed with medication and potentially deep brain stimulation. But research is underway to look for earlier methods of detection, as well as better treatments. Eventually, we might even have a way to prevent it from developing in the first place.

See more here:
How to Prevent Parkinson's Disease: Tips, Medications, and Research - Healthline

Recommendation and review posted by Bethany Smith

We sat down for an interview with Professor Botond Roska, MD PhD, one of the worlds leading experts in the study of vision and the retina and last years recipient of the Krber Prize, to discuss the ground-breaking therapy he and his team have developed and already successfully used to return some vision to a handful of blind patients.

Can you tell us about the exact nature of your research for which you received the Krber Award?

It is sometimes difficult to know exactly what you get an award for, but I think I received it for two distinct but related scientific pursuits: firstly, my research on understanding vision, and secondly, for developing a treatment called optogenetic vision restoration therapy, through which a completely blind person can be sensitized to light, allowing them to see again.

FactProfessor Botond Roska, MD PhD, is a biomedical researcher and one of the foremost experts on vision, the retina, and treating diseases that cause blindness. He is a founding director of the Institute for Molecular and Clinical Ophthalmology Basel (IOB) in Switzerland and a Professor at the University of Basel. In 2019, he was awarded the Order of Saint Stephen, the highest national honor bestowed by Hungary, as well as the Louis-Jeantet Prize for Medicine. In 2020, he won the Krber European Science Prize for his research on a gene therapy that could be utilized to restore some vision to the fully blind, which he successfully implemented with his team for the first time this May.

What exactly does this procedure you developed entail?

It is a so-called two-component therapy. We inject a gene therapy vector into the eye, which is a small, virus-like particle with a DNA that encodes a light-sensitive protein. We target this protein at some elements of the blind retina. The vector also has a goggle that records the world, and then projects a picture onto the retina in a very specific way, in a particular color.

How effective is it now, and how effective can it potentially become?

The therapy restores a certain amount of vision, but not full sight. Based on the few patients we have had so far, the first of whom we recently published a paper about, the therapy allows patients to recognize objects, but it does not enable them to read, for instance.

However, there is a lot of room to improve. This is only the very first step, the very first optogenetic therapy.

What kinds of blindness or visual impairment can it help or cure?

It is useful in cases when someone is fully blind as a result of photoreceptor dysfunction, but their retina is still connected to their brain via an intact optic nerve.

How profound an effect do you think being able to heal peoples vision at a large scale might have on society?

Blindness is one of the conditions that people name as the worst affliction to have. In fact, in a recent survey conducted in the U.S., participants named it as the worst condition they could suffer from out of a whole host of common health problems, ranking it above cancer, Alzheimers, and other truly devastating diseases.

Our whole life is spent looking at phones and computers. Particularly during the pandemic, we could have almost no social interactions or opportunities to work without these, so that blind people were essentially cut off from the world apart from what little interaction they could have with it through hearing.

Therefore, we hope that such therapies, once they become widely available since they are in the clinical trial phase at the moment will improve many peoples lives tremendously.

Where do you think your research might lead you in the future?

There are three main directions. The first one is simple; as we discussed, we need to keep researching and improving optogenetic vision therapies, expanding them to target other cell types in the retina.

The second one is to do with the fact that if the optic nerve is missing, we cannot provide any therapies at the moment. This is something we are working on at my institute together with researcher Dniel Hillier, who is leading these efforts. We aim to find ways of restoring vision when there is no optic nerve.

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Hungarian-born researcher Dr. Botond Roska has been awarded the 2019 Louis-Jeantet Prize for Medicine for the discovery of basic principles of visual information processing and the development of therapeutic strategies, such as gene therapy, to restore vision in retinal disorders. Within the last few months, the neurobiologist (who celebrates his 50th birthday this year) received []Continue reading

The third is that most visual impairment is partial blindness, in which the method we developed cannot be used, so we are interested in looking into very large diseases affecting a lot more people. We want to try to slow down the degeneration or restore more vision to those with partial sight loss.

Why did you choose to dedicate your career to understanding human vision at the hardware level?

It was a chain of random events. I did not plan to be a vision researcher. I just wanted to understand things. When I finished my medical school, I was quite sure I did not want to treat patients for a living, I was more interested in understanding the human body. I decided to go into research, and I met someone who was researching the retina. I started my research in this field, and came up against an increasing number of questions that I found fascinating.

I went from topic to topic, physiology, virology, the molecular biology of the eye, then towards the end of my graduate studies, I read a paper that said it was possible to make cells light-sensitive using molecules form other organisms. At that time, I understood the retina quite well, and thought that I could combine my knowledge with these findings to try to design a therapy. My lab and myself are both interested in gaining scientific insight as well as designing therapies, and that is the path I plan to continue on for the rest of my career.

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The findings provide proof-of-concept that using optogenetic therapy to partially restore vision is possible, Botond Roska said.Continue reading

What is the worst and best part of the publicity youve received as a result of your success?

The best part is certainly that blind people can become informed that we are working on a therapy, which can provide them with some hope. It is also important for my institute, which can more easily recruit brilliant scientific minds who will come up with even better therapies down the line.

On the other hand, while there is nothing really bad about publicity per se, it can sometimes be time-consuming. Our paper about our first patient came out in May, and it has been downloaded around 90,000 times, with just about every country in the world reporting on our findings. And of course, all of the press outlets in all these countries bombarded our inboxes with requests. We were basically paralyzed for almost a month.

As someone who is called upon with increasing frequency to explain your cutting edge research, how do you approach the issue of science communication?

Communication is very important. It is part of our lives. In Switzerland specifically, it is taken very seriously, and it is part of our jobs as researchers to explain things to the public. We also have professionals to help us navigate the world of public relations.

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President Jnos der decorated neurobiologist Botond Roska with the Hungarian Saint Stephen Order on Tuesday, Hungarys national holiday. Hungarian-born Researcher Awarded for Helping People Regain Their Sight Each year on the holiday of the founder of our state we celebrate those Hungarians whose outstanding talent and achievements serve the well-being and prosperity of many others, []Continue reading

The difficulty today is finding the balance between our work and its communication to the world. Because our research reaches a tremendous number of people extremely quickly thanks to the global nature of social media and other platforms, we get a lot of requests for interviews and articles. The difficulty for us is balancing our responsibility to inform the public of our progress with actually making some. Sometimes, it feels very much like we are in the eye of the storm.

Many people consider you a likely future Nobel laureate. Do you believe you might receive the award?

I dont think about any of that. It does not occupy any of my time or of my imagination. At this moment, there are still great challenges ahead of us if we want to make the therapies as good as they can be. That is what I concentrate on. We also need to focus on innovating and coming up with new tools to help realize our scientific vision. This is also a costly and time-consuming endeavor.

What would you tell aspiring scientists, how can they best succeed in their scientific endeavors?

I think that the key is to come to science with an incredible desire to solve interesting problems. I never wanted to be successful in the public eye, just accomplished at solving scientific questions. For some reason, some scientists are more present in the media, and some of them win prizes, while others win prizes and choose not to have a public presence. Often, the most brilliant and prolific scientists do not even win prizes.

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Returning Vision to The Fully Blind - Interview with Dr. Botond Roska - Hungary Today

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Ryder Comer was born with SMA, the best treatment option was an infusion that cost $2.1 million. Now, his mom is working to add SMA to Idaho's newborn screening.

MIDDLETON, Idaho When Jake and Haley Comer of Middleton welcomed their new baby boy Ryder into the world, they were completely overjoyed. But, they quickly realized something might not be right.

"The whole pregnancy was completely healthy, there were no signs of anything." said Haley Comer, Ryder's proud mom. "As soon as he was born though, he was diagnosed with low muscle tone. No parent wants to think that something is wrong with their perfect little baby."

The Comers went to their pediatrician to get some answers. The doctor agreed that newborn Ryder was "floppy" and had low muscle tone. He recommended genetic testing to see if there was a reason for that. While waiting for the results in the following weeks, the unthinkable happened. Ryder stopped breathing.

"The day after Ryder turned a month old, I found him unresponsive at home, I had him in a little baby carrier. So, I called 911 and started CPR and luckily was able to revive him. We were rushed to the ER, and that initiated a month-long stay in the hospital," said Haley Comer with tears in her eyes.

While they were at St. Luke's, the genetic testing results came back. The news was not what the Comers were hoping for.

"We got our test results back and Ryder was diagnosed with SMA, Spinal Muscular Atrophy Type 1," said Haley. "It was hard, there were a lot of tears, but at the same time we want to be brave. You have to put on a brave face for your baby."

SMA is a progressive, rare genetic disease that impacts the muscles. Babies with SMA Type 1 face many physical challenges, including muscle weakness and trouble breathing, coughing, and swallowing.

Doctors at St. Luke's in Boise told the Comers about a promising new treatment option. A gene therapy infusion that could save baby Ryder's life.

"They told us that they would be transferring him to Salt Lake City to Primary Children's Hospital, because no hospitals in Idaho performed this infusion," said Haley. "A couple days later, we met with a neurologist in Salt Lake and learned about our treatment options. This gene therapy option seemed like the best one. It's a once in his lifetime, one-hour long, gene therapy infusion. He is missing a very important gene that produces survival motor neuron protein. That helps our body move our muscles. It basically injects this gene into his body, and then his body learns to keep producing this survival neuron protein to keep his muscles alive."

#7sHero Tonight at 10 p.m. on KTVB youll meet this precious baby boy, Ryder Comer of Middleton. His parents Jake &...

It sounded like the best option for Ryder, but the Comers were stunned when they found out just how much the infusion treatment would cost.

"It's called Zolgensma, and it costs $2.1 million for one single infusion," said Haley.

Zolgensma is FDA-approved for patients with all forms and types of SMA who are under 2 years of age. Research shows babies dosed with Zolgensma as soon after diagnosis as possible have had better results than those who waited to begin treatment. It's given through an IV infusion that takes about an hour, and it's a one-time treatment.

The Comers weren't even sure their insurance would cover it, but they moved forward with trying to get the pricey infusion procedure preauthorized.

"It was terrifying when they told us," said Jake Comer, Ryder's dad. "I was like I hope you take monthly payments for the rest of my life."

Incredibly, the approval from their insurance company, United Healthcare, went smoothly and quickly.

"We paid our deductible, and our out-of-pocket, and they took care of the rest," said Jake in disbelief. "That definitely took the stress of our shoulders during such a traumatic time, so we are very grateful."

The Comers say it was all a whirlwind. Ryder received the infusion within 24 hours.

"We feel pretty fortunate because a lot of families had to fight for weeks and months to get their insurance to cover this," said Haley. "For Ryder, his disease had progressed so much by the time he got it, he was at the point where every day mattered."

After the $2.1 million infusion, Ryder started making strides within days. The changes they've seen over the past several months have been so wonderful for the Comers.

"His breathing got stronger, his voice got stronger, his cry got louder, he had a really weak cry at first," said Haley. "Now he can lift his arms above his head and grab toys! He can't quite hold his head up yet, but we are working on it. He is in feeding and physical therapy, and we are doing everything we can to get him stronger."

So far, Ryder is the only baby in Idaho with SMA to receive this gene therapy infusion. Haley has a new passion. She is now dedicated to helping other SMA families like theirs.

"What we are really working toward right now is to have SMA added to Idaho's newborn screening. Idaho is one of the last remaining states that does not include SMA on its newborn screening test. It's just a simple blood test. Before the approval of his gene therapy two years ago, the life expectancy for babies diagnosed with SMA Type 1 was not much past two years old. That is just devastating for families. So having SMA added to Idaho's newborn screening will allow these babies to have access to treatment so much sooner. The sooner you get a diagnosis and have treatment, the better outcomes you will have in life."

Haley has been in contact with the Idaho Department of Health and Welfare, and with the organization CURE SMA, which has been working to add this disease to every state's newborn screening. She's in the early stages, but this mom's goal is to make that happen here in Idaho, too.

"We just think that every baby deserves to be treated as soon as they possibly can," said Haley.

August is SMA Awareness Month. Baby Ryder and his parents are paving the way for other Idaho babies born with SMA.

"He's expected to get stronger and hopefully meet those milestones of walking and talking and crawling and all those things, but we just don't know, we are working hard in therapy to give him the best possible chance. One day a time."

For more information on SMA, click here. If you'd like to help the Comer family with their medical expenses, there is a GO FUND ME set up to do so.

See all of the heartwarming segments in ourYouTube playlisthere:

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Ryder's Story: Middleton baby with rare genetic condition gets a $2.1 million infusion to give him a fighting chance - KTVB.com

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NEW YORK An international team of researchers has used transcriptomic data from ulcerative colitispatients to develop a predicted polygenic transcriptional risk score, or PPTRS,that can identify UC-affected individuals at fivefold elevated risk of progressing to surgical resection of the large bowel.

In a paper published on Thursday in the American Journal of Human Genetics, the Georgia Institute of Technology-led team noted that 5 percent to 10 percent of people with UC require bowel resection, or colectomy, within five years of diagnosis, but that polygenic risk scores based on genome-wide association studies generally don't provide meaningful prediction of progression to surgery. However, studies of Crohn's disease have shown that gene expression profiling of GWAS-significant genes provides some stratification of risk of progression to complicated disease through transcriptional risk scoring, or TRS.

In their paper, the researchers demonstrated that a measured TRS based on bulk rectal gene expression in a cohort of UC patients had a positive predictive value approaching 50 percent for colectomy. Single-cell profiling demonstrated that the disease-associated genes were active in multiple diverse cell types from both the epithelial and immune compartments, and expression quantitative trait locusanalysis identified genes with differential effects at baseline and the one-year follow-up, the researchers said. But for the most part, they found that differential expression associated with colectomy risk was independent of local genetic regulation.

Overall, their data suggested that prediction of gene expression from relatively small transcriptome datasets can be used in conjunction with transcriptome-wide association studies for stratification of risk of disease complications.

The researchers began by performing differential expression analysis between baseline rectal RNA-seq biopsies of individuals in the PROTECT multicenter pediatric inception cohort study of response to standardized colitis therapy. Analyses were done on 21 affected individuals who progressed to colectomy and 310 who did not. They identified downregulation of 783 transcripts in the individuals who underwent colectomy and upregulation of 1,405 transcripts overall.

They also obtained rectal biopsy RNA-seq data for 92 affected individuals at week 52 and observed a marked shift in gene expression at follow-up, prompting them to ask whether local regulation of the gene expression might contribute to this effect. They found that there were 72 SNPs that were significantly regulating 308 genes at both time points.

Further examination of the expression of colectomy-associated genes in a single-cell RNA-seq dataset obtained from rectal biopsies provided strong evidence that both epithelial and immune cells contributed to the risk of disease progression, the researchers said.

The researchers then performed a TWAS to capture the effects of all polymorphisms within 1 Mb of each transcript expressed in the PROTECT rectal biopsies and then used the weights to predict gene expression in a validation cohort from the UK Biobank. They tested for differential predicted gene expression in 70 percent of the validation samples and discovered about 800 genes either upregulated or downregulated in UC-affected individuals relative to non-IBD control individuals. They then derived a PPTRS for UC based on the effect sizes of the minor alleles and applied it to the remaining 30 percent of the validation samples, as well as to the PROTECT genotypes, and found that the PPTRS efficiently discriminated UC-affected individuals from non-IBD control individuals.

Significantly, it also discriminated the individuals who underwent colectomy versus those who didn't in both the UK Biobank and PROTECT.

"More extensive single-cell profiling, combined with cell-type-specific genetic analysis of gene expression, is likely to lead to the development of even better transcriptional risk signatures," the authors concluded. "It is also likely that such focused and personalized analysis may highlight specific pathological mechanisms active in particular affected individuals."

They did note, however, that these results were limited by the relatively small sample size of colectomies in the PROTECT study, and that validation of cross-ancestry assessments and the evaluation of the consistency of gene expression prediction across populations should be a high priority.

In an email, corresponding author and GIT researcher Greg Gibson noted that while the study's multiple layers of replication show that transcriptional profiling of the rectum greatly enhances risk stratification for risk of colectomy, this was not a clinical trial, so the approach is not yet approved for evaluation of patients.

"We hope that it will progress to implementation in the near future," he added."The prediction from genotypes alone is less likely to have clinical utility since the precision is still quite low, so that aspect is more research oriented."

He further noted that the approach he and his colleagues used could also be applied to a wide range of diseases, and that they are pursuing that research.

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Ulcerative Colitis Study Analyzes Gene Expression to Measure Risk of Progression to Surgery - GenomeWeb

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Brain development that occurs after birth is also important. Rebecca Saxe at MIT is working to understand the brain structures and activities responsible for social cognition, which allows us to consider the mental states of other people.

Saxe has discovered a particular brain region that is key; by studying how activity in this region and others changes over the course of childhood, she may be able to understand how social abilities develop. She has also found that these brain activity patterns are altered in people with autism spectrum disorders.

Even though researchers are starting to understand some of the processes that govern development and have identified things that can derail it, were far from being able to intervene when such problems occur. But as we gain insights, we could someday test therapies or other ways to address these developmental issues.

Computational neuroscientists use mathematical models to better understand how networks of brain cells help us interpret what we see and hear, integrate new information, create and store memories, and make decisions.

Understanding how the activity of neurons governs cognition and behavior could lead to ways to improve memory or understand disease processes.

Terry Sejnowski, a computational neurobiologist at the Salk Institute, has built a computer model of the prefrontal cortex and analyzed its performance on a task in which a person (or machine) has to sort cards according to a rule thats always changing. While humans are great at adapting, machines generally struggle. But Sejnowskis computer, which imitates information flow patterns observed in the brain, performed well on this task. This research could help machines think more like humans and adapt more quickly to new conditions.

Aude Oliva, the MIT director of the MIT-IBM Watson AI Lab, uses computational tools to model and predict how brains perceive and remember visual information. Her research shows that different images result in certain patterns of activity both in the monkey cortex and in neural network models, and that these patterns predict how memorable a certain image will be.

Research like Sejnowskis may inspire smarter machines, but it could also help us understand disorders in which the function of the prefrontal cortex is altered, including schizophrenia, dementia, and the effects of head trauma.

Researchers are trying to determine the genetic and environmental risk factors for neurodegenerative diseases, as well as the diseases underlying mechanisms.

NHUNG LE

Improving prevention, early detection, and treatment for diseases like Alzheimers, Parkinsons, Huntingtons, chronic traumatic encephalopathy, and ALS would benefit millions of people around the world.

Yakeel Quiroz, at Massachusetts General Hospital, studies changes in brain structure and function that occur before the onset of Alzheimers symptoms. Shes looking for biomarkers that could be used for early detection of the disease and trying to pinpoint potential targets for therapeutics. One potential biomarker of early-onset Alzheimers that shes founda protein called NfLis elevated in the blood more than two decades before symptoms appear. Quiroz has also identified a woman with a protective genetic mutation that kept her from developing cognitive impairments and brain degeneration even though her brain showed high levels of amyloid, a protein implicated in Alzheimers development. Studying the effects of this beneficial mutation could lead to new therapies.

Researchers at the Early Detection of Neurodegenerative Diseases initiative in the United Kingdom are analyzing whether digital data collected by smartphones or wearables could give early warnings of disease before symptoms develop. One of the initiatives projectsa partnership with Boston Universitywill collect data using apps, activity tracking, and sleep tracking in people with and without dementia to identify possible digital signatures of disease.

As we learn more about the underlying causes of neurodegenerative diseases, researchers are trying to translate this knowledge into effective treatments. Advanced clinical trials targeting newly understood mechanisms of disease are currently under way for many neurodegenerative disorders, including Alzheimers, Parkinsons, and ALS.

Connectomics researchers map and analyze neuronal connections, creating a wiring diagram for the brain.

Understanding these connections will shed light on how the brain functions; many projects are exploring how macro-scale connections are altered during development, aging, or disease.

Mapping these connections isnt easythere may be as many as 100 trillion connections in the human brain, and theyre all tiny. Researchers need to find the best ways to label specific neurons and track the connections they make to other neurons in remote parts of the brain, refine the technology to collect these images, and figure out how to analyze the mountains of data that this process produces.

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Eight ways scientists are unwrapping the mysteries of the human brain - MIT Technology Review

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State College, Pennsylvania., August 30, 2021 / PRNewswire / -NeuExcell Therapeutics (www.neuexcell.com), A gene therapy company focused on neurodegenerative diseases has announced a Series Pre-A funding round of over $ 10 million. The round was led by Co-Win Ventures and was attended by other institutional investors Yuan Bio, Oriza Seed, Tsingyuan and Inno Angel.

We are honored to join this very reputable group of investors, he said. Peter Tombros, Chairman of the Board of Directors of NeuExcell Therapeutics. Investor experience and support will enable us to leverage our unique neuroregenerative gene therapy platform across multiple neurodegenerative indications. This funding strength validates our strategy and biotechnology. Further examine our science in the industry.

Professor Gong Chen, co-founder and chief scientific advisor of the company, said: There is an urgent need for breakthrough therapies like us.

I think this is a great opportunity to invest in experienced leadership, he said. Xin Huang, Managing Partner of Co-Win Ventures. NeuExcells unique technology has the potential to act as a platform for treating many neurodegenerative diseases, providing hope for breakthrough new therapies for patients who do not have the right choices today.

With the end of this successful pre-A round, we welcome him. Xin Huang Jonathan Sun attended the board meeting.

About NeuExcellTherapeutics

NeuExcell is a privately held early stage genetic engineering company headquartered in Pennsylvania, USA When Shanghai, China.. Its mission is to improve the lives of patients suffering from neurodegenerative diseases and damage to the central nervous system. Based on Professor Gong Chens scientific research, we have developed a potentially destructive nerve repair technique through the conversion of astrocytes to neurons. In vivo By introducing neural transcription factors through adeno-associated virus (AAV) -based gene therapy. NeuExcells pipeline covers major neurodegenerative diseases such as stroke, Huntingtons disease, amyotrophic lateral sclerosis (ALS), Alzheimers disease, Parkinsons disease, traumatic brain injury, spinal cord injury, and glioma. increase.

About Co-Wof Venture

Founded in 2009, Co-Win Ventures is an early stage investor in healthcare and TMT with a focus on equality, transparency, sharing and innovation. Co-Wins business network China When USA..Total AUM is about US $ 1 billion, Co-Win aims to be a reliable partner for great entrepreneurs to build breakthrough technologies and businesses. Co-Win Ventures has helped more than 140 portfolio companies, including leading leaders in their respective sub-sectors, including Cytek, Connect, Thrive (acquired by Nasdaq-listed company EXAS), Taimei Technology, Genecast, Sinovation and Augta. ..

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NeuExcell Therapeutics Raises Over $ 10 Million Series Before Round To Keep The Company Growing | Around The Web-Pennsylvania

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SAN FRANCISCO--(BUSINESS WIRE)--AllStripes (Company), a healthcare technology company dedicated to unlocking treatments for people affected by rare diseases, today announced the Company completed a $50 million Series B financing round.

The financing was led by Lux Capital, a current investor, joined by JAZZ Venture Partners, Spark Capital, Medidata Solutions (a Dassault Systmes company), McKesson Ventures, and Maveron, along with angel investors including Arif Nathoo, CEO of Komodo Health, and Leila Zegna, Director of the Kabuki Syndrome Foundation.

The funding will support launching 100 new rare disease research programs while expanding global operational footprint, technology and data automation enhancements to improve research insights, further developing the platform capabilities to enhance the user experience and strengthen the life sciences offerings, and continuing to invest in growing the companys team to support creating the playbook for rare disease research.

Beginning research on a rare condition can feel like being dropped into a new world without a map and we are on a mission to change that with data, said Nancy Yu, CEO and Co-founder, AllStripes. Were proud that so many of our current investors have recognized the progress weve made and are continuing to support our vision to transform rare disease research. This investment will allow us to better support the rare disease community, where each persons experience is essential to understanding disease progression - ultimately leading to new treatments for rare disease patients around the globe.

AllStripes has a successful track record of bringing together patient organizations, families, experts and life science partners to advance research, said Adam Goulburn, Partner, Lux Capital and AllStripes board member. If we are going to improve rare disease treatments, these groups must work together. We believe in AllStripes vision to transform rare disease research and empower patients. If the past year showed us anything, it is that patients want a voice when it comes to their health, and their engagement with research is invaluable.

There are more than 7,000 rare diseases and only 5 percent have treatments. By working with AllStripes, we hope to improve the number of treatments available by accelerating research for rare diseases, said James M. Wilson, M.D., Ph.D., Rose H. Weiss Professor and Director, Orphan Disease Center; Director, Gene Therapy Program at the University of Pennsylvania; Professor in Departments of Medicine and Pediatrics, Perelman School of Medicine.

AllStripes is currently partnering with more than 30 patient advocacy organizations across its 40 conditions, supporting more than 3,000 users to date. As a public benefit corporation (PBC), AllStripes is advocating for the importance of real-world evidence in development of treatments and is continuing to build tools that make research more inclusive for the global rare disease community. The Company is committed to transparent data collection and sharing data across the life sciences continuum to advance clinical research.

AllStripes collaborates with various biopharmaceutical companies and other entities on real-world evidence studies, including: HemoShear Therapeutics, Inc., Orphan Disease Center at the University of Pennsylvania, Taysha Gene Therapies, Inc., UCB Biopharma SRL, and Novartis Pharma AG. These joint research programs aim to enhance clinical understanding of rare disease treatment research. AllStripes and the University of Pennsylvania's Orphan Disease Center are focused on clinical understanding of Lesch-Nyhan disease and Crigler Najjar syndrome type 1, with the goal of facilitating therapeutic discovery for both conditions. One of AllStripes most recent partnerships with HemoShear Therapeutics aims to gather real-world evidence on patients' medical experience with serious metabolic diseases through the Journey to Understand MMA and PA (JUMP) study.

To learn more about AllStripes, its partners and its commitment to the rare disease community, please visit allstripes.com.

About AllStripesAllStripes is a healthcare technology company dedicated to unlocking new treatments for people with rare diseases. AllStripes has developed a technology platform that generates regulatory-ready evidence to accelerate rare disease research and drug development, as well as a patient application that empowers patients and families to securely participate in treatment research online and benefit from their own medical data. AllStripes was founded by CEO Nancy Yu and technology developer Onno Faber, following his diagnosis and journey with the rare disease neurofibromatosis type 2. The company is backed by Lux Capital, JAZZ Venture Partners, Spark Capital, Medidata Solutions, McKesson Ventures, Maveron, and a number of angel investors. For more information, visit http://www.allstripes.com.

About Lux CapitalLux Capital invests in emerging science and technology ventures at the outermost edges of what is possible. We partner with iconoclastic inventors challenging the status quo and the laws of nature to bring their futuristic ideas to life. Over the past two decades, Lux has expanded from its New York City roots to Silicon Valley, and built a $4 billion AUM firm of more than 30 full-time professionals, with the versatility to invest at any stage.

JAZZ Venture PartnersJAZZ Venture Partners invests in companies that extend the boundaries of human performance improving how we live, learn, work, play, and experience the world. JAZZ seeks breakthroughs at the frontiers of technology and science, such as advances in artificial intelligence, neurobiology, augmented reality, and closed-loop human-computer systems. JAZZ portfolio companies are unlocking human potential in health, mind-body wellness, accelerated learning and training, sports, entertainment, and the enterprise. More information about JAZZ can be found at http://www.jazzvp.com.

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AllStripes Announces $50 Million Series B Financing to Advance Global Rare Disease Research - Business Wire

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VANCOUVER, British Columbia & BASEL, Switzerland--(BUSINESS WIRE)--Genevant Sciences, a leading nucleic acid delivery company with world-class platforms and the industrys most robust and expansive lipid nanoparticle (LNP) patent estate, today announced that it has entered into a global collaboration and license agreement with Takeda Pharmaceutical Company Limited (Takeda) for the development and commercialization of novel nonviral gene therapies to treat up to two undisclosed rare liver diseases. This is the second collaboration between Genevant and Takeda, following an earlier 2021 agreement to develop nucleic acid therapeutics directed to specified targets in hepatic stellate cells to treat liver fibrosis.

LNP provides a compelling approach to deliver on the promise of gene therapy, and our leadership position in the LNP space is well established. We have enjoyed working with our Takeda colleagues to develop hepatic stellate cell-directed treatments to treat liver fibrosis and are delighted to expand the relationship further with this second collaboration, said Pete Lutwyche, Ph.D., president and chief executive officer, Genevant Sciences Corporation.

Building on our existing foundation with Genevant in liver fibrosis, were excited to expand our work together to develop life-altering, nonviral gene therapies for specified rare liver diseases, said Bernard Allan, head of liver disease research at Takeda. Genevants expertise in the development of LNPs for clinical applications, coupled with Takedas drug development capabilities and history in gastroenterology, gives us a great opportunity to develop new treatment options for patients with liver disorders.

Under the terms of the agreement, Genevant is eligible to receive up to $303 million in upfront and potential milestone payments, plus royalties on future product sales. Takeda has exclusive rights to utilize Genevants LNP technology in the development and commercialization of specified nonviral gene therapies for up to two undisclosed rare liver diseases.

About Genevant Sciences

Genevant Sciences is a leading nucleic acid delivery company with world-class platforms, the industrys most robust and expansive lipid nanoparticle (LNP) patent estate, and decades of experience and expertise in nucleic acid drug delivery and development. The Companys scientists have pioneered LNP delivery of nucleic acids for over 20 years, and the Companys LNP platform, which has been studied across more than a dozen discrete product candidates and is the delivery technology behind the first and only approved RNAi-LNP (patisiran), enables a wide array of RNA-based applications, including vaccines, therapeutic protein production, and gene editing. For more information, please visit http://www.genevant.com.

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Genevant Sciences Announces Global Collaboration and License Agreement with Takeda to Develop Novel Nonviral Gene Therapies for Up to Two Rare Liver...

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