Sugar detox? Cutting carbs? A doctor explains why you should keep fruit on the menu – WJW FOX 8 News Cleveland
(THE CONVERSATION) One of my patients who had been struggling with obesity, uncontrolled diabetes and the cost of her medications agreed in June 2019 to adopt a more whole-food plant-based diet.
Excited by the challenge, she did a remarkable job. She increased her fresh fruit and vegetable intake, stopped eating candy, cookies and cakes and cut down on foods from animal sources. Over six months, she lost 19 pounds and her HbA1c a measure of her average blood sugar dropped from 11.5% to 7.6%.
She was doing so well, I expected that her HbA1c would continue to drop and she would be one of our plant-based successes who had reversed diabetes.
Her three-month follow-up visit in March 2020 was canceled because of COVID-19 lockdowns. When I eventually saw her again in May 2021, shed regained some of the weight and her HbA1c had climbed to 10.4%. She explained that her diabetes doctor and a diabetes nurse educator had told her that she was eating too much sugar on the plant-based diet.
Shed been advised to limit carbohydrates by cutting back on fruits and starchy vegetables and eating more fish and chicken. Sugar-free candy, cakes, cookies and artificial sweeteners were encouraged. In the face of conflicting medical advice, she fell back on conventional wisdom that sugar is bad and should be avoided whenever possible, especially if you have diabetes.
Im a physician, board certified in preventive medicine with a lifestyle medicine clinic at Morehouse Healthcare in Atlanta. This emerging medical specialty focuses on helping patients make healthy lifestyle behavior modifications. Patients who adopt whole-food plant-based diets increase carbohydrate intake and often see reversal of chronic diseases including diabetes and hypertension. In my clinical experience, myths about sugar and carbohydrates are common among patients and health professionals.
Fruit vs. sugar
Your body runs on glucose. It is the simple sugar that cells use for energy.
Glucose is a molecular building block of carbohydrates, one of the three essential macronutrients. The other two are fat and protein. Starches are long, branching chains of glucose.
Naturally occurring carbohydrates travel in nutrient-dense packages such as fruits, vegetables, whole grains, nuts and seeds.
Humans evolved to crave sweet tastes to get the nutrients needed to survive. A daily supply of vitamins, minerals and fiber is needed because our bodies cannot make them. The best source of these substances for our ancient ancestors was sweet, ripe, delicious fruit. In addition, fruits contain phytonutrients and antioxidants, chemicals produced only by plants. Phytonutrients such ellagic acid in strawberries have cancer-fighting properties and promote heart health.
Refined sugars, on the other hand, are highly processed and stripped of all nutrients except calories. Theyre a concentrated form of carbohydrates. The food industry produces refined sugars in many forms. The most common are sucrose crystals, which youd recognize as table sugar, and high-fructose corn syrup, which is found in many processed foods and sweetened beverages.
If you continually satisfy your taste for sweet with foods that contain refined sugar rather than the nutrient-rich fruits at the core of this craving passed on by evolution you may not get all the nutrients you need.
Over time, this deficit may create a vicious cycle of overeating that leads to obesity and obesity-related health problems. Women who eat the most fruit tend to have lower rates of obesity.
Sugar toxicity
Refined sugars are not directly toxic to cells, but they can combine with proteins and fats in food and in the bloodstream to produce toxic substances such as advanced glycation end products (AGEs). High blood glucose levels may produce glycated low-density lipoproteins. High levels of these and other glucose-related toxic substances are associated with an increased risk of a wide range of chronic health problems, including cardiovascular disease and diabetes.
The disease most commonly associated with sugar is Type 2 diabetes. A surprising number of people, including health professionals, incorrectly believe that eating sugar causes Type 2 diabetes. This myth leads to a focus on lowering blood sugar and counting carbs while ignoring the real cause: progressive loss of pancreatic beta cell function. At diagnosis, a patient may have lost between 40% and 60% of their beta cells, which are responsible for producing insulin.
Insulin is a hormone that controls how much glucose is in the bloodstream by blocking glucose production in the liver and driving it into fat and muscle cells. Loss of beta cell function means not enough insulin gets produced, resulting in the high blood glucose levels characteristic of Type 2 diabetes.
Beta cells have low levels of antioxidants and are susceptible to attack by metabolic and dietary oxidized free radicals and AGEs. Antioxidants in fruit can protect beta cells. Researchers have found that eating whole fruit decreases the risk of Type 2 diabetes, with those who eat the most fruit having the lowest risk.
Detoxing from sugar
People interested in losing weight and improving health often ask if they should do a sugar detox. In my opinion this is a waste of time, because it is not possible to eliminate sugar from the body. For instance, if you ate only baked chicken breasts, your liver would convert protein to glucose in a process called gluconeogenesis.
Low-carb diets may lead to weight loss, but at the expense of health. Diets that significantly reduce carbohydrates are associated with nutrient deficiencies and higher risk of death from any cause. On low-carbohydrate ketogenic diets the body will break down muscles and turn their protein into glucose. The lack of fiber causes constipation.
Eliminating foods sweetened with refined sugar is a worthy goal. But dont think of it as a detox it should be a permanent lifestyle change. The safest way to go on a refined sugar detox is to increase your intake of nutrient-dense fruits and vegetables. Once you eliminate refined sugar, youll likely find that your taste buds become more sensitive to and appreciative of the natural sweetness of fruits.
Article written by Jennifer Rooke,Morehouse School of Medicine via Associated Press.
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Sugar detox? Cutting carbs? A doctor explains why you should keep fruit on the menu - WJW FOX 8 News Cleveland
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Struggling with hormonal acne and how to fix it? A skin expert shares her best advice. – Mamamia
How do you diagnose hormonal acne?
While it's best to see a trusted doctor to determine if you have hormonal acne, there are a few things you can look out for to self-diagnose it. Here's what Dr Joshi said:
Now onto the part that anyone with hormonal acne wants to know about: how TF do you fix it?
"Treatment is similar to that in adolescence," Dr Joshi explained. "However, due to persistence, more aggressive treatments and a combination of treatments may be needed to get it under control even for relatively mild symptoms."
"By the time I see patients with persistent acne, they have usually tried various over-the-counter products, they may have been to salons for facials and other treatments, some of which may have made the acne worse, and may contribute to scarring andpigmentation."
Dr Joshi explained that because acne is a medical disease, treatment options need to be medical.
She suggests a combination of topical prescription medication, oral medication (for a period of time), and a personalised, usually basic, skincare regimen.
Listen to Mamamia's podcast for your face, You Beauty. Post continues after audio.
"You can speed up the process by adding a series of in-clinic treatments if you wish but it will take on average three to six months minimum to begin tosee results," she said.
With that being said, Dr Joshi suggests saving your time and money and going straight for a skin consultation with a trusted doctor to obtain personalised advice and prescription medication, if appropriate.
"Treatment is time-consuming and depending on what is involved, may be costly, but the alternative means that acne continues to persist and cause unwanted side effects such as scarring and pigmentation that will also need to be addressed at some point,with added time and cost," she said.
"See a doctor who understands acne and save yourself time, money and the heartache of complications, and have realistic expectations," Dr Joshi said.
"The longer people leave it, thinking it will go away, or trying a variety of different over-the-counter things, the longer the disease is percolating under the surface and the longer it takes to rein it in and the chances of complications such as scarringand pigmentation.
"Treatment takes time, which is the single biggest thing I tell patients. And relapses are common if life gets in the way and they drop the ball."
Feature Image: Getty/Canva/Mamamia.
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How Bad Is It Really to Let Your Pet Sleep in Your Bed? – Livestrong
There are pros and cons to letting Fluffy or Fido snuggle up with you at night.
Image Credit: LIVESTRONG.com Creative
How Bad Is It Really? sets the record straight on all the habits and behaviors youve heard might be unhealthy.
If you crave peak cozy, welcoming your cat or dog or both onto your bed delivers. After all, it's just so companionable to have a pet nearby thanks to the comfort of touching fur and the soft sounds of purrs and snuffles. But should you let your dog sleep with you?
And after a day spent together cats twining through your legs while you make a morning cup of coffee, or your dog curled up nearby while you watch TV cuddling in bed with your pet seems only natural. Indeed, a slim majority (56 percent) of pet owners invite cats and dogs into their bedroom, according to a December 2015 article in Mayo Clinic Proceedings.
But there are some health implications to this practice. Here's what experts have to say about the pros and cons of bringing pets in the bedroom, and whether your dog (or other furry friend) should sleep with you.
1. Your Allergies and Asthma Might Get Worse
About a third of people in the U.S. are allergic to cats and dogs (with cat allergies more common), according to the Asthma and Allergy Foundation of America.
If you're one of that group, allowing your pet in the bedroom means about eight hours of exposure to allergens, says immunologist Heather Moday, MD, author of The Immunotype Breakthrough.
"Breathing in this level of allergens all night long can cause nasal congestion, sinus inflammation and airway irritation if you have asthma," Dr. Moday says. That makes it harder to get restful sleep, and means you'll wake up stuffy and tired in the morning, she says.
And it's not only pet allergies that you need to consider. Cats and dogs carry pollen and dust on their fur, Dr. Moday says. That means allowing pets in bed puts you in close contact with two more common allergens.
This can also exacerbate asthma, as allergies and asthma often go hand-in-hand. Common triggers (like dander, dust and pollen) that lead to allergic reactions like sniffles or itchy eyes can also set off asthma symptoms, according to the Mayo Clinic.
Regularly washing yourself and your pet is one allergy remedy that can help prevent excess dander, dust and pollen.
2. You Might Get Flea Bites
Some parasites like to live on cats and dogs (think: fleas, ticks and lice). And allowing your cat or dog in the bedroom is placing yourself in very close contact with these itch-causing creatures.
Fleas, for instance, bite humans, which can cause itching and irritation, per the Cleveland Clinic.
3. Your Sleep Could Suffer
When it comes to sleep hygiene, there aren't any benefits to having pets in bed, says sleep psychologist Samina Ahmed Jauregui, PsyD, a Pluto Pillow advisor.
But there are some drawbacks: Pets like to move at night, says Abhinav Singh, MD, facility director of the Indiana Sleep Center and member of the National Sleep Foundation medical advisory board. "That can wake people up," he says.
And it's hard to go back asleep after waking up, especially as we age, Dr. Singh says which could reduce the quantity of sleep you get each night.
You'll know it if your pet wakes you up at 3 a.m. and you can't fall back to sleep right away. But what might be more insidious is when a pet's movement causes you to readjust more frequently than you would otherwise.
"Micro-awakenings are not recallable," Dr. Singh says. In other words, if you briefly wake up to toss and turn due to your cat or dog stirring, it's not long enough for your brain to register as a moment of wakefulness. But those brief bursts of sleeplessness still have an effect.
Dr. Singh compares it to flying: Would you rather fly nonstop or have seven connecting flights en route to your destination? These small, routine disruptions diminish the quality of your shut-eye. That'll leave you feeling unrested in the morning, even if you diligently clocked the Centers for Disease Control and Prevention-recommended seven-plus hours of sleep.
Jauregui sums it up: "Even if you are able to return to sleep quickly, having woken up in the first place is a disturbance in quality of sleep."
4. It Could Improve Your Mood
It's not all doom and gloom. Bringing a pet into your bedroom is associated with some significant mental health benefits.
"Many people claim to really enjoy sleeping with their animals because they feel less lonely and happier with their pets," Dr. Moday says. And there are studies to back that up.
For instance, in a survey of just under 1,000 pet owners assigned female at birth, dogs were "associated with stronger feelings of comfort and security" compared to human bed partners, according to November 2018 research in Anthrozos.
Also among the research-backed benefits of time with pets are an increase in oxytocin (a feel-good hormone) and a decrease in cortisol (a stress hormone), according to Johns Hopkins Medicine.
While Dr. Singh generally thinks bringing a pet to bed is best avoided, he makes an exception for someone who is overcoming loss or trauma and finds comfort in a pet's presence. "The dog or cat can be there to support you [and] help you recover emotionally," he says.
7 Healthy Tips for Sleeping With Your Pet in the Bed
Though there are some solid reasons why your dog shouldn't sleep with you, the joys of companionship likely outweigh these potential downsides if you and your pet are in the habit of sharing a bed.
We get it. So if you're disregarding the experts' take and persist in welcoming Fluffy or Fido onto the mattress, follow these strategies to reduce potential pitfalls:
So, How Bad Is It Really to Let Your Pet Sleep in Bed With You?
When it comes to deciding if you should let your dog sleep with you, truly, it depends.
Dr. Singh notes he has cats, and is an animal-lover but if forced to give a bottom line, he notes it's "generally not advisable" to sleep with your pet. It's also best avoided if you have allergies or allergy-trigged asthma. Instead, he recommends having a cat or dog bed or blanket in your bedroom, beside your bed, but not on it.
Jauregui agrees that it's best to keep the bedroom pet-free. But, she adds: "If you struggle with sleep and are not comfortable sleeping in the bed by yourself, then having a pet in the bed with you (that is large enough for both of you) can be OK." Some sleep even if it's potentially disrupted would be better than none, she says.
And if you just can't quit those nighttime snuggles (guilty!), follow the best practices described above when it comes to laundry, pet care and adjusting your sleep routine.
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5 questions facing gene therapy in 2022 – BioPharma Dive
Four years ago, a small Philadelphia biotech company won U.S. approval for the first gene therapy to treat an inherited disease, a landmark after decades of research aimed at finding ways to correct errors in DNA.
Since then, most of the world's largest pharmaceutical companies have invested in gene therapy, as well as cell therapies that rely on genetic modification. Dozens of new biotech companies have launched, while scientists have taken forward breakthroughs in gene editing science to open up new treatment possibilities.
But the confidence brought on by such advances has also been tempered by safety setbacks and clinical trial results that fell short of expectations. In 2022, the outlook for the field remains bright, but companies face critical questions that could shape whether, and how soon, new genetic medicines reach patients. Here are five:
Food and Drug Administration approval of Spark Therapeutics' blindness treatment Luxturna a first in the U.S. came in 2017. A year and a half later, Novartis' spinal muscular atrophy therapy Zolgensma won a landmark OK.
But none have reached market since, with treatments from BioMarin Pharmaceutical and Bluebird bio unexpectedly derailed or delayed.
That could change in 2022. Two of Bluebird's treatments, for the blood disease beta thalassemia and a rare brain disorder, are now under review by the FDA, with target decision dates in May and June. BioMarin, after obtaining more data for its hemophilia A gene therapy, plans to soon approach the FDA about resubmitting an application for approval.
Others, such as CSL Behring and PTC Therapeutics, are also currently planning to file their experimental gene therapies with the FDA in 2022.
Approvals, should they come, could provide important validation for their makers and expand the number of patients for whom genetic medicines are an option. In biotech, though, approvals aren't the end of the road, but rather the mark of a sometimes challenging transition from research to commercial operations. With price tags expected to be high, and still outstanding questions around safety and long-term benefit, new gene therapies may prove difficult to sell.
A record $20 billion flowed into gene and cell therapy developers in 2020, significantly eclipsing the previous high-water mark set in 2018.
Last year, the bar was set higher still, with a total of $23 billion invested in the sector, according to figures compiled by the Alliance for Regenerative Medicine. About half of that funding went toward gene therapy developers specifically, with a similar share going to cell-based immunotherapy makers.
Driving the jump was a sharp increase in the amount of venture funding, which rose 73% to total nearly $10 billion, per ARM. Initial public offerings also helped, with sixteen new startups raising at least $50 million on U.S. markets.
Entering 2022, the question facing the field is whether those record numbers will continue. Biotech as a whole slumped into the end of last year, with shares of many companies falling amid a broader investment pullback. Gene therapy developers, a number of which had notable safety concerns crop up over 2021, were hit particularly hard.
Moreover, many startups that jumped to public markets hadn't yet begun clinical trials roughly half of the 29 gene and cell therapy companies that IPO'd over the past two years were preclinical, according to data compiled by BioPharma Dive. That can set high expectations companies will be hard pressed to meet.
Generation Bio, for example, raised $200 million in June 2020 with a pipeline of preclinical gene therapies for rare diseases of the liver and eye. Unexpected findings in animal studies, however, sank company shares by nearly 60% last December.
Still, the pace of progress in gene and cell therapy is fast. The potential is vast, too, which could continue to support high levels of investment.
"I think fundamentally, investment in this sector is driven by scientific advances, and clinical events and milestones," said Janet Lambert, ARM's CEO, in an interview. "And I think we see those in 2022."
The potential of replacing or editing faulty genes has been clear for decades. How to do so safely has been much less certain, and concerns on that front have set back the field several times.
"Safety, safety and safety are the first three top-of-mind risks," said Luca Issi, an analyst at RBC Capital Markets, in an interview.
Researchers have spent years making the technology that underpins gene therapy safer and now have a much better understanding of the tools at their disposal. But as dozens of companies push into clinical trials, a number of them have run into safety problems that raise crucial questions for investigators.
In trials run by Audentes Therapeutics and by Pfizer (in separate diseases), study volunteers have tragically died for reasons that aren't fully understood. UniQure, Bluebird bio and, most recently, Allogene Therapeutics have reported cases of cancer or worrisome genetic abnormalities that triggered study halts and investigations.
While the treatments being tested were later cleared in the three latter cases, the FDA was sufficiently alarmed to convene a panel of outside experts to review potential safety risks last fall. (Bluebird recently disclosed a new hold in a study of its sickle cell gene therapy due to a patient developing chronic anemia.)
The meeting was welcomed by some in the industry, who hope to work with the FDA to better detail known risks and how to avoid them in testing.
"[There's] nothing better than getting people together and talking about your struggles, and having FDA participate in that," said Ken Mills, CEO of gene therapy developer Regenxbio, in an interview. "The biggest benefit probably is for the new and emerging teams and people and companies that are coming into this space."
Safety scares and setbacks are likely to happen again, as more companies launch additional clinical trials. The FDA, as the recent meeting and clinical holds have shown, appears to be carefully weighing the potential risks to patients.
But, notably, there hasn't been a pullback from pursuing further research, as has happened in the past. Different technologies and diseases present different risks, which regulators, companies and the patient community are recognizing.
"We're by definition pushing the scientific envelope, and patients that we seek to treat often have few or no other treatment options," said ARM's Lambert.
Last June, Intellia Therapeutics disclosed early results from a study that offered the first clinical evidence CRISPR gene editing could be done safely and effectively inside the body.
The data were a major milestone for a technology that's dramatically expanded the possibility for editing DNA to treat disease. But the first glimpse left many important questions unanswered, not least of which are how long the reported effects might last and whether they'll drive the kind of dramatic clinical benefit gene editing promises.
Intellia is set to give an update on the study this quarter, which will start to give a better sense of how patients are faring. Later in the year the company is expecting to have preliminary data from an early study of another "in vivo" gene editing treatment.
In vivo gene editing is seen as a simpler approach that could work in more diseases than treatments that rely on stem cells extracted from each patient. But it's also potentially riskier, with the editing of DNA taking place inside the body rather than in a laboratory.
Areas like the eye, which is protected from some of the body's immune responses, have been a common first in vivo target by companies like Editas Medicine. But Intellia and others are targeting other tissues like the liver, muscle and lungs.
Later this year, Verve Therapeutics, a company that uses a more precise form of gene editing called base editing, plans to treat the first patient with an in vivo treatment for heart disease (which targets a gene expressed in the liver.)
"The future of gene editing is in vivo," said RBC's Issi. His view seems to be shared by Pfizer, which on Monday announced a $300 million research deal with Beam Therapeutics to pursue in vivo gene editing targets in the liver, muscle and central nervous system.
With more and more cell and gene therapy companies launching, the pipeline of would-be therapies has grown rapidly, as has the number of clinical trials being launched.
Yet, many companies are exploring similar approaches for the same diseases, resulting in drug pipelines that mirror each other. A September 2021 report from investment bank Piper Sandler found 21 gene therapy programs aimed at hemophilia A, 19 targeting Duchenne muscular dystrophy and 18 going after sickle cell disease.
In gene editing, Intellia, Editas, Beam and CRISPR Therapeutics are all developing treatments for sickle cell disease, with CRISPR the furthest along.
As programs advance and begin to deliver more clinical data, companies may be forced into making hard choices.
"[W]e think investors will place greater scrutiny as programs enter the clinic and certain rare diseases are disproportionately pursued," analysts at Stifel wrote in a recent note to investors, citing Fabry disease and hemophilia in particular.
This January, for example, Cambridge, Massachusetts-based Avrobio stopped work on a treatment for Fabry that was, until that point, the company's lead candidate. The decision was triggered by unexpected findings that looked different than earlier study results, but Avrobio also cited "multiple challenging regulatory and market dynamics."
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Cell and Gene Therapy Business Outlook Service – Yahoo Finance
DUBLIN, Jan. 14, 2022 /PRNewswire/ -- The "Cell and Gene Therapy Business Outlook" report has been added to ResearchAndMarkets.com's offering.
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The Twice-Monthly Publication Cell and Gene Therapy Business Outlook will offer the following:
Market Sizing and Forecasting of CAGT Markets: Each issue sizes up the market opportunity and projects the future revenues for a given therapeutic segment.
Keeping an Eye on Financing: With billions of investment dollars announced each year, Cell and Gene Therapy Business Outlook tracks who is getting financed (and the companies behind the financing) each issue. On a regular basis we will analyze trends in that financing.
News Briefs and Analysis of the Science That will Shape Tomorrow's Business: Cell and Gene Therapy Business Outlook is designed to provide the most relevant news. With a focus on what the recent news of the day means for business, our curated news and news analysis means that you and your organization can be confident you won't miss an important development in cell and gene therapy.
Deals Between CAGT Companies Tracked: Each issue's "Recent Deals Table" tracks the important deals between stem cell companies as well as the deals they engage in (tech transfers, partnerships, mergers, distribution and other activities) with companies outside the industry.
Cell and Gene Therapy Tools: This newsletter will also report on developments, product launches and deals relating to the makers of cell and gene therapy manufacturing equipment and supplies.
Target Audience Includes:
Director, Vice President or Manager of Market Research
Director, Vice President or Manager of Marketing
Director, Vice President or Manager of Research and Development
Director of Business Insights
Director of Business Development
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"GT and CT have been a hot topic! We are tasked to know this area and need a resource like this"-Consulting Firm
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"The charts and tables are a good idea to keep up with the amount of new announcements in CGT. We appreciated the scientific discussion in this issue and your recent webinar." -Cell and Gene Therapy Startup
For more information about this report visit https://www.researchandmarkets.com/r/upanl3
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BioMarin plans return to FDA with updated data on hemophilia gene therapy – BioPharma Dive
BioMarin Pharmaceutical plans to return to the Food and Drug Administration later this year with clinical trial results it says prove its gene therapy for hemophilia can prevent bleeding for years after treatment.
The data from BioMarin's study, disclosed Sunday ahead of the J.P. Morgan Healthcare Conference, are meant to meet requirements laid out by the agency when it rejected the company's previous approval application a year and a half ago.
They show the gene therapy restored blood clotting protein levels to a range consistent with mild hemophilia and, while those levels waned over time, that trial participants experienced very few, if any, bleeds across the two years most were studied. A handful of volunteers in the Phase 3 trial, the largest to date of a hemophilia gene therapy, were followed for three years and had similar results.
"I believe that these results will answer, quantitatively, quite a lot of the questions that agencies have had," said Hank Fuchs, BioMarin's head of research and development, on a conference call Sunday.
Regulators in Europe have already begun evaluating an application from BioMarin and are expected to make a decision in the first half of this year. In the U.S., BioMarin aims to quickly review the results with the FDA and, should the agency agree, potentially resubmit the therapy in the second quarter.
BioMarin's gene therapy, called Roctavian, is the product of years of research by the California biotech and builds on more than a decade of work by other scientists to develop a treatment for hemophilia's genetic cause. It is designed to deliver into the body a functional copy of the gene that's mutated in people with the "A" form of hemophilia, who are left with little or no clotting protein to stem bleeding.
People with severe hemophilia A, who make up about half of all those with disease, must take regular, preventive infusions of "replacement" clotting protein, also known as Factor VIII. Roctavian, which is meant for these individuals, would in theory allow them to stop, freeing them from chronic treatment while more effectively preventing bleeding.
Results from an earlier, much smaller trial showed such promise and, by mid-2020, BioMarin had come close to replicating those findings in the first group of volunteers enrolled in its Phase 3 study. But the FDA unexpectedly, according to the company sought more information to prove that benefit could last two years.
Last January, BioMarin revealed one-year results from all participants in the trial and, on Sunday, disclosed data from its two-year analysis. Treatment decreased the number of bleeds per year by 85%, from an average of nearly five among the 112 volunteers who were studied for at least six months before infusion to less than one at year two.
Among 17 participants who were given Roctavian three years before the analysis was conducted, the average annual bleeding rate remained below one as well.
"Our clinical outcome here is unassailably great," said Fuchs in a separate interview. "It almost makes the application, honestly, bulletproof."
But levels of Factor VIII activity, which had risen sharply to an average of 43 international units per deciliter of blood at one year, declined to 23 IU/dL by year two and, for those 17 participants, 17 IU/dL by year three. BioMarin reported these values using a lab test known as a chromogenic assay, which it says is more conservative than another one also used.
People with severe hemophilia typically have less than one IU/dL of Factor VIII in their blood, while mild hemophilia is typically considered to be between 5 IU/dL and 40 IU/dL.
The decline has been a source of doubt, causing concerns that Roctavian's ability to prevent bleeds might wane over time as well. At least for the first few years, Sunday's results show that isn't happening yet. BioMarin also points to data from an earlier study, in which annualized bleeding rates remained below one through five years, despite reduced Factor VIII activity.
"A small amount of Factor VIII is going to go a long way towards hemostatic efficacy," said Fuchs on Sunday's call, "and it gives us confidence that what we've seen so far in the Phase 2 study is gonna read through to the Phase 3 study when we get there."
Extrapolating efficacy puts BioMarin on somewhat uncertain ground, however, as it is the first company to advance this far with a gene therapy for hemophilia A. Jean-Jacques Bienaime, BioMarin's CEO, argues the data so far for Roctavian indicate treatment should result in at least five years of bleeding control and perhaps even eight or longer.
"With the Phase 2, we have demonstrated at least five years already. Predicting eight years, I don't think, is a big stretch," he said in an interview.
How the FDA will view BioMarin's data is unclear, although analysts on Wall Street predicted the latest results would be enough to merit an approval. The agency could convene a panel of outside experts to review a resubmitted application from the company, a possibility Fuchs acknowledged on the conference call.
Also uncertain is how Roctavian would be perceived by hemophilia patients and by insurers, should it eventually secure an approval. BioMarin has previously suggested a price as high as between $2 million and $3 million, but that might be viewed as high if Roctavian's benefit isn't lifelong. (ICER, a looked-to drug cost watchdog, previously found Roctavian could be cost effective at a price of even $2.5 million.)
Fuchs said the company plans to present more data at a medical meeting, likely this year, that should help clarify the relationship between Factor VIII activity and expected durability of benefit.
Importantly for Roctavian's future, Sunday's data, while relatively sparse, indicated no new safety issues had emerged in testing. There were no cases of "inhibitors," or antibodies that work against clotting protein, developing following treatment, nor were there any cases of cancer or blood clot blockages.
The former two are both newly of interest following reports of cancers developing in other gene therapy trials, and data showing higher-than-normal levels of clotting factor in a trial of another hemophilia gene therapy being developed by Pfizer and Sangamo Therapeutics.
Note: This story has been updated to include mention of the assay used by BioMarin to measure Factor VIII activity, and of ICER's analysis.
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Castle Creek Biosciences Acquires Novavita Thera to Expand Innovative Cell and Gene Therapy Platform – PRNewswire
EXTON, Pa., Jan. 10, 2022 /PRNewswire/ -- Castle Creek Biosciences, Inc., a late-clinical stage cell and gene therapy company focused on developing and preparing to commercialize disease-modifying and potentially curative therapies for rare genetic diseases, today announced it has acquired Novavita Thera, Inc., a preclinical gene therapy company focused on rare liver and metabolic diseases. The acquisition expands Castle Creek's technology platform by adding in vivo capabilities to its existing ex vivo approach, and broadens Castle Creek's development pipeline beyond skin and connective tissue disorders to rare liver diseases.
"This acquisition is a significant inflection point for Castle Creek and positions us to expand our research and development efforts using a versatile, dual technology platform that will accelerate the discovery of disease-modifying and potentially curative therapies for people living with rare diseases," said Matthew Gantz, president and chief executive officer of Castle Creek Biosciences. "The ability to leverage both ex vivo and in vivo based approaches is a distinct advantage that few cell and gene therapy companies can offer. We are now in position to pursue new indications for devastating rare diseases, while also advancing our ongoing pivotal clinical trial in recessive dystrophic epidermolysis bullosa (RDEB)."
With the acquisition of Novavita Thera, formerly aCytotheryx, Inc., company, Castle Creek will initially develop a gene therapy for hereditary tyrosinemia type 1 (HT1),a rare inborn error of metabolism caused by a lack of the enzyme fumarylacetoacetate hydrolase (FAH) which leads to accumulation of tyrosine and its metabolites in the liver. HT1 affects approximately 1:100,000 live births and leads to cirrhosis, liver failure, hepatocellular carcinoma, and is ultimately fatal if untreated. Liver transplantation is currently the only curative treatment available for HT1.
Castle Creek will advance the development of LV-FAH, a potential therapy based on a lentiviral vector containing a functional copy of the human FAH gene that is administered directly to the patient through the portal vein. The therapy is designed to transduce hepatocytes and deliver the FAH enzyme that is deficient in these cells.Castle Creek plans to submit an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for LV-FAH in HT1. Castle Creek also continues to progress several additional candidates targeting other rare liver and metabolic diseases and skin and connective tissue disorders.
In connection with the acquisition, Joseph Lillegard, MD, PhD, has joined Castle Creek as chief scientific officer. Dr. Lillegard is a board-certified pediatric and adult general, thoracic and fetal surgeon at the Children's Hospital of Minnesota, and led the cell and gene therapy research lab at Mayo Clinic that discovered LV-FAH. Robert A. Kaiser, PhD, DABT, has also joined the company as vice president of preclinical development. Dr. Kaiser is a board-certified toxicologist with over a decade of experience designing, conducting, and reporting preclinical and IND-enabling studies. Dr. Lillegard and Dr. Kaiser will be the company leads for Castle Creek's recently announced research collaboration with Mayo Clinic to advance discovery and development of investigational gene therapy candidates for the treatment of osteogenesis imperfecta and classical Ehlers-Danlos syndrome.
"It is an exciting time to join Castle Creek, a company that has already established an impressive research and development program in cell and gene therapies with proven clinical development and in-house manufacturing capabilities," said Dr. Lillegard. "I look forward to collaborating with the company's dedicated team on development of novel gene therapies. We believe our work to evaluate the safety of in vivo lentiviral vector administration in HT1 has the potential to be a precedent setting approach that can be applied to a range of new therapeutic areas for underserved patient populations."
About Castle Creek Biosciences, Inc.
Castle Creek Biosciences, Inc. is a late-clinical stage cell and gene therapy company focused on developing and preparing to commercialize disease-modifying and potentially curative therapies for patients living with rare genetic diseases. Castle Creek's most advanced product candidate, dabocemagene autoficel (FCX-007, D-Fi), an ex vivo, autologous gene therapy, is currently being evaluated in a Phase 3 clinical trial for the localized treatment of chronic wounds due to recessive dystrophic epidermolysis bullosa (RDEB). The company is also evaluating FCX-013, an ex vivo, autologous gene therapy, in a Phase 1/2 clinical trial for the treatment of moderate to severe localized scleroderma.In addition, LV-FAH, an in vivo, investigational gene therapy candidate, is being assessed in preclinical studies for the treatment of hereditary tyrosinemia type 1 (HT1).Castle Creek is pursuing discovery and development of early-stage novel product candidates utilizing its dual platform of ex vivo and in vivo technologies to expand its robust pipeline. The company operates an in-house, commercial-scale manufacturing facility in Exton, Pennsylvania. Castle Creek Biosciences, Inc. is a portfolio company of Paragon Biosciences, LLC. For more information, visit https://castlecreekbio.com/or follow Castle Creek on Twitter @CastleCreekBio.
About Paragon Biosciences, LLC
Paragon is a global life science leader that creates, builds and funds innovative biology-based companies in three key areas: cell and gene therapy, adaptive biology and advanced biotechnology. The company's current portfolio includes Castle Creek Biosciences, CiRC Biosciences, Emalex Biosciences, Evozyne, Harmony Biosciences, Qlarity Imaging, Skyline Biosciences, and a consistent flow of incubating companies created and supported by the Paragon Innovation Capital model. Paragon stands at the intersection of human need, life science, and company creation. For more information, please visit https://paragonbiosci.com/.
Media Contacts
Adam DaleyBerry & Company Public Relations212.253.8881[emailprotected]
Karen CaseyCastle Creek Biosciences302.750.4675[emailprotected]
SOURCE Castle Creek Biosciences, Inc.
Recommendation and review posted by Bethany Smith
At 15.0% CAGR, Global Gene Delivery Technologies Market Size Will Reach USD 7.86 Billion By 2028: Polaris Market Research – PRNewswire
NEW YORK, Jan. 12, 2022 /PRNewswire/ -- Polaris Market Research recently published a research report on "Gene Delivery Technologies Market Share, Size, Trends, Industry Analysis Report, By Mode (Biological [Adenovirus, Retrovirus, AAV, Lentivirus, Other Viruses, Non-viral], Chemical, Physical); By Application (Gene Therapy, Cell Therapy, Vaccines, Research); By Method; By Regions; Segment Forecast, 2021 2028" in its online research storage.
According to [127+ Pages] research report published by Polaris Market Research, the global Gene Delivery Technologies Market size & share expected to reach to USD 7.86 Billion by 2028 from USD 2.64 Billion in 2020, at a compound annual growth rate (CAGR) of 15.0% during forecast period 2021 to 2028.
What is Gene Delivery Technology? How big is Gene Delivery Technology Industry?
Gene delivery technology is widely used in gene therapies, which involves transferring of genetic and hereditary disorders. These therapies have also performed an important role in shaping the entire pharma landscape. Around 27 gene therapies were revealed in the marketplace and over 990 companies emphasized the research & development, and commercialization of innovative therapies by 2020. The constantly changing market environment for advanced therapies is reportedly driving the market for gene delivery technologies.
The operating market players are building various business strategies to boost the market for gene delivery technologies, while the developing gene delivery technologies are creating openings for several new players in the market. Different research settings offer market applications for various gene delivery technologies. However, due to technical challenges related to each modal type, the clinical settings produce very few applications. Within the clinical settings, physical technologies require a breakthrough in their use.
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Key Aspects Covered By Report:
Top Market Companies Profiles Covered:
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Industrial Gene Delivery Technology Market: Growth Factors
The adoption of gene therapies and subsequent increase in clinical research activities around the globe has fueled the market growth. Also, the growing acceptance of gene therapy products and services has supported the gene delivery technologies market growth prospects. Other key driving factors of the market involve technological advancements in viral vectors, a rising pipeline of advanced therapies, and a growing number of regulatory approvals for advanced therapy products.
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Global Gene Delivery Technology Market: Key Segmentation
Insight by Mode
In 2020, the biological vectors market segment secured the largest revenue share of the gene delivery technologies industry due to the high success rate of Kymriah and Yescarta. Following the acceptance of vectors-based therapy products, the above-mentioned vectors have experienced greater attention.
The chemical delivery method market segment is expected to secure a lucrative growth rate over the study period. Clinical challenges are observed in viral systems which propelled the chemical methods' use. Chemical delivery systems have replaced viral delivery systems because of their capability in combating challenges.
The physical delivery methods market segment has lower transfection efficiency than biological or chemical modes. One other drawback called low cell viability in electroporation-based physical methods enables other market players to gain more share. It helps them to address a high focus on transfection and cell viability issues.
Also Read, Global Gene Therapy Market Report, 2021-2028
Insight by Method
The ex-vivo market segment of the gene delivery technologies market witnessed the largest share in 2020. Its transduction efficiency is the major factor behind the high share achievement, making it an ideal candidate to be used in research settings.
In this market, the in-vivo delivery method market segment is expected to obtain a lucrative growth rate over the estimation period as it features a high preference for highly targeted gene deliveries. Researchers are extending research & development for the market segment. For example, Oregon Health and Science University built the gene-editing tool "Crispr-Cas9" in 2020, which enables genetic code editing for blind people.
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Gene Delivery Technologies Market: Report Scope
Report Attribute
Details
Market Size 2020 Value
USD 2.64 Billion
Market Outlook for 2028
USD 7.86Billion
Expected CAGR Growth
CAGR 150% from 2021 - 2028
Base Year
2020
Forecast Year
2021 - 2028
Top Market Players
Horizon Discovery Group Co., QIAGEN, Oxford Biomedica, SignaGen Laboratories, Hoffmann-La Roche AG, Vectalys, Sirion-Biotech GmbH and Others
Segments Covered
By Mode, By Application, By Method, By Region
Geographies Covered
North America, Europe, Asia Pacific, Latin America and Middle East & Africa
Customization Options
Customized purchase options are available to meet your research needs. Explore customized purchase options
Geographic Overview: Gene Delivery Technology Market
The North American region registered a significant share of the global gene delivery technology market. Various clinical trials are used to access the efficacy of gene therapies to treat hereditary, cancer, genetic mutations, and rare disorders in the U.S. This factor is the key driver of the gene delivery technologies demand growth in North America.
In addition, the availability of better clinical infrastructure also contributes to market growth. Many companies are marketing gene delivery products and accessories, which will boost the gene delivery technologies industry growth prospects. The U.S. has already announced many research projects combined with other leaders under its Horizon 2020 plans. This project will also cover other vector-based gene delivery trials for rare diseases.
Moreover, the Asia Pacific gene delivery technologies industry is anticipated to account for a profitable gene delivery technology market growth rate over the assessment period. The region is well known for the developed pharmaceutical industry even with its large population size, and low labor costs.
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The market is primarily segmented on the basis of mode, application, method, and geographic region.
Gene Delivery Technology Market: By Mode Outlook
Gene Delivery Technology Market: By Application Outlook
Gene Delivery Technology Market: By Method Outlook
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At 15.0% CAGR, Global Gene Delivery Technologies Market Size Will Reach USD 7.86 Billion By 2028: Polaris Market Research - PRNewswire
Recommendation and review posted by Bethany Smith
Is Beam Therapeutics a Good Stock to Buy Now? – The Motley Fool
Risky biotechnology start-ups that soared in recent years can't catch a break in 2022. Beam Therapeutics (NASDAQ:BEAM) recently signed a major research deal with Pfizer (NYSE:PFE) that could be worth up to $1.35 billion, and hardly anyone seemed to notice.
This was clearly great news for Beam Therapeutics, but a stock market scorned for clinical-stage biotech stocks didn't respond the way anyone familiar with the company would have expected. Instead of surging higher in response to the Pfizer deal, Beam Therapeutics stock actually fell nearly 2% on the day of the announcement.
Nearly all biotech stocks are in the doghouse lately and it looks like the market may have missed something here. Let's look closer to see if Beam Therapeutics is a smart buy at the moment.
Image source: Getty Images.
Over the past few years, Pfizer has watched its peers experiment with CRISPR-based gene editing techniques without making any significant investments. Beam Therapeutics' base-editing technology, though, really got the big pharma company's attention.
Instead of removing and replacing entire sections of genetic material like Intellia, andCRISPR Therapeutics (NASDAQ:CRSP), Beam Therapeutics is pioneering a more precise method called base editing. This involves altering just one letter of genetic material at a time, which is a lot more useful than it might seem. Around half of all known genetic variations associated with diseases are caused by single-point mutations.
Pfizer will give Beam Therapeutics a $300 million payment up front to discover new drug candidates aimed at three undisclosed targets that won't compete with Beam's existing programs. Beam's eligible for up to $1.05 billion in milestone payments if all three go on to become a commercial success.
Beam Therapeutics is eligible to receive royalties at an undisclosed percentage of global sales for each future program. Beam Therapeutics even has an option to co-develop and co-commercialize one of the future candidates for a larger cut of sales.
Beam Therapeutics finished September with $934 million in cash after operations burned through $329 million during the first nine months of the year. Pfizer's cash injection should raise the company's cash balance high enough to get through 2023 before it needs to tap investors for more.
There's no telling whether Pfizer will decide to license a candidate from Beam Therapeutics. If Pfizer drags its feet, the gene-editing start-up has some preclinical-stage programs of its own that might have a chance to impress investors before it's time to raise capital again.
The most advanced candidate in Beam's pipeline at the moment, BEAM-101 is an experimental gene therapy for the treatment of sickle cell disease. The company doesn't expect to begin enrolling patients into the first clinical trial with BEAM-101 until the second half of 2022.
With a recent market cap of $4.5 billion, there's already a lot of success for Beam's pipeline priced into the stock. Unfortunately, the road ahead could be a lot more challenging than investors are anticipating. Last year, Vertex Pharmaceuticals (NASDAQ:VRTX) and collaboration partner CRISPR Therapeutics reported highly encouraging results from a clinical trial with CTX001 that started way back in 2018.
CTX001 is an experimental gene therapy for sickle cell disease that's similar to BEAM-101 in that it encourages the production of fetal hemoglobin. If early interim data that Beam Therapeutics posts a couple of years from now doesn't appear competitive with CTX001, the stock could take a tumble. While this is a top gene-editing stock to watch, it's still a little too risky to buy right now.
This article represents the opinion of the writer, who may disagree with the official recommendation position of a Motley Fool premium advisory service. Were motley! Questioning an investing thesis -- even one of our own -- helps us all think critically about investing and make decisions that help us become smarter, happier, and richer.
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Is Beam Therapeutics a Good Stock to Buy Now? - The Motley Fool
Recommendation and review posted by Bethany Smith
JPM 2022: Amicus axes gene therapy program for type of Batten disease, advances another – FierceBiotech
Amicus Therapeutics is moving on from an early-phase Batten disease program after follow-up data showed the therapy didn't stop the fatal nervous system disease from progressing long-term.
The company disclosed the pipeline trim in a preliminary revenue guidance announcement ahead of its Wednesday presentation at the J.P. Morgan Healthcare Conference.
Amicus was advancing a gene therapy program for CLN6 Batten disease, a type of the rare genetic disorder that causes development regression and typically begins in childhood. Back in 2019, Amicus shared data on AAV-CLN6 that showed the gene therapy stabilized children's motor and language functions.
But the CLN6 program will now be discontinued after the company got a look at long-term extension data from a phase 1/2 trial.
RELATED:Amicus shares early look at Batten disease gene therapy
The company found the disease stabilization that had occurred during the earlier portion of the trial was not sustained at the two-year mark. Amicus plans to review the data with the CLN6 Batten disease community to support continued research efforts to find better treatments and cures which are so desperately and urgently needed, according to the guidance announcement.
During its last earnings report in November 2021, Amicus had said it would be ramping up manufacturing activities and regulatory discussions for the program.
Meanwhile, the company will advance its CLN3 Batten disease program, which is currently in a phase 1/2 trial. A readout from the trial and additional preclinical data are expected in 2022, Amicus said. Once the data are released, the company can begin work on a pivotal trial.
Continued here:
JPM 2022: Amicus axes gene therapy program for type of Batten disease, advances another - FierceBiotech
Recommendation and review posted by Bethany Smith
Leptin: Benefits and risks of the leptin diet – Medical News Today
Leptin is a hormone that comes from fat cells. It helps control food intake by sending signals about hunger to the hypothalamus in the brain. This process regulates appetite.
Leptin regulates energy levels by maintaining a balance between hunger and appetite. The hormone triggers the body to respond by eating more when energy levels are low and eating less when energy levels are stable or high.
People who have high levels of body fat have high circulating levels of leptin.
Research shows that having elevated leptin levels can lead to leptin resistance, making weight loss difficult.
This article looks at what the leptin hormone is, what the leptin diet involves, and the advantages and disadvantages of following the leptin diet.
Scientists discovered leptin, a protein that functions as a hormone, in 1994.
Leptin is one of the main hormones responsible for maintaining body weight. Leptin helps people balance how much food they consume by regulating hunger levels. The hormone also controls how much energy a person uses throughout each day.
Leptin comes from fat cells within the body. It enters the blood supply and travels up to the brain. The hormone must cross the blood-brain barrier, a membrane that protects the brain from harmful toxins, to get to the hypothalamus. The hypothalamus is the area in the middle of the brain that controls hormone regulation, among other important functions.
At the hypothalamus, leptin can function by signaling that the body does not need any more food. This response causes the person to feel full. If leptin levels are low, or leptin does not reach the hypothalamus, a person will continue to feel hungry.
Leptin regulates body weight and is an important marker for energy storage. This means if the body has excess energy stored as fat, leptin signals the hypothalamus to reduce appetite and burn excess body fat for fuel. This response helps a person maintain a moderate body weight.
However, when a person has high amounts of body fat, they can develop a resistance to leptin, which leads to abnormally high leptin levels.
Having leptin levels that are too low is less common. Low leptin levels can occur in severe childhood obesity and delayed puberty.
When leptin levels are below average, the brain thinks no body fat is present. This reduced level can cause symptoms of uncontrollable hunger, resulting in excessive food intake. Leptin injections are a way of reducing this problem.
After scientists discovered the hormone in 1994, Byron J. Richards created a diet named after it: the leptin diet.
The goal of the leptin diet is to return leptin levels to normal and create balance within the body. The leptin diet has five main principles:
The leptin diet permits most types of food, but guidelines suggest avoiding chemical additives and processed sugars and sticking to fresh and organic produce.
The leptin diet encourages other lifestyle changes, such as getting plenty of sleep and participating in regular physical activity.
A 2021 study suggests that diets high in fat, carbohydrates, fructose, and sucrose and low in protein are drivers of leptin resistance. The researchers concluded that leptin resistance might be reversible by reducing calories.
However, this research has some limitations, such as small sample sizes, so further evidence is required to verify these claims.
The leptin diet includes limiting snacking and shortening your daily eating window. If a person reduces how much they snack, this could create a calorie deficit necessary for weight loss.
A leptin diet is a sensible approach to weight loss for some people, as the diet promotes eating healthily without harsh restrictions but encourages a routine.
However, at present, no studies are investigating the effects of the leptin diet on weight loss and leptin levels.
It is important to remember that all bodies are different, and a diet that meets the nutritional demands of one person will not always work for someone else.
For example, limiting the number of meals to three per day and cutting out snacking may be effective for a person with a low activity level. However, it is unlikely to meet the energy demands of a person who leads an active lifestyle, exercises intensely, or has a physically demanding job.
Many factors can impact energy needs, including age, pregnancy, breastfeeding, and certain medical conditions.
A person should consider consulting a healthcare professional like a registered dietitian if they are interested in improving health through dietary changes.
Increasing research suggests that obesity causes people to develop leptin resistance.
When someone carries an excessive amount of body fat, they will have too much leptin circulating in the blood. This excess results in that person becoming leptin-resistant. This resistance means their brain stops responding to the leptin signals traveling up to it. It also means their body continues to produce leptin, contributing to elevated leptin and leptin resistance.
Research suggests that weight loss and energy-restricted diets may help reverse leptin resistance.
Leptin is the hormone that controls appetite. Leptin informs the brain when a person has eaten enough, reducing appetite, and produces hunger signals when a person requires energy.
As with any weight loss plan, a person should approach the leptin diet with caution. The diet may be effective for some people, but it may not meet the nutritional demands of every person. Check with a doctor before starting any significant weight loss diet.
Link:
Leptin: Benefits and risks of the leptin diet - Medical News Today
Recommendation and review posted by Bethany Smith
How The mRNA Vaccines Were Made: Halting Progress and Happy Accidents – The New York Times
I said, I am an RNA scientist. I can do anything with RNA, Dr. Karik recalled telling Dr. Weissman. He asked her: Could you make an H.I.V. vaccine?
Oh yeah, oh yeah, I can do it, Dr. Karik said.
Up to that point, commercial vaccines had carried modified viruses or pieces of them into the body to train the immune system to attack invading microbes. An mRNA vaccine would instead carry instructions encoded in mRNA that would allow the bodys cells to pump out their own viral proteins. This approach, Dr. Weissman thought, would better mimic a real infection and prompt a more robust immune response than traditional vaccines did.
It was a fringe idea that few scientists thought would work. A molecule as fragile as mRNA seemed an unlikely vaccine candidate. Grant reviewers were not impressed, either. His lab had to run on seed money that the university gives new faculty members to get started.
By that time, it was easy to synthesize mRNA in the lab to encode any protein. Drs. Weissman and Karik inserted mRNA molecules into human cells growing in petri dishes and, as expected, the mRNA instructed the cells to make specific proteins. But when they injected mRNA into mice, the animals got sick.
Their fur got ruffled, they hunched up, they stopped eating, they stopped running, Dr. Weissman said. Nobody knew why.
For seven years, the pair studied the workings of mRNA. Countless experiments failed. They wandered down one blind alley after another. Their problem was that the immune system sees mRNA as a piece of an invading pathogen and attacks it, making the animals sick while destroying the mRNA.
Eventually, they solved the mystery. The researchers discovered that cells protect their own mRNA with a specific chemical modification. So the scientists tried making the same change to mRNA made in the lab before injecting it into cells. It worked: The mRNA was taken up by cells without provoking an immune response.
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How The mRNA Vaccines Were Made: Halting Progress and Happy Accidents - The New York Times
Recommendation and review posted by Bethany Smith
STAT’s guide to the next generation of CAR-T therapies – STAT
CAR-T cell therapy has been a boon for treating blood cancers. Since the technology was first brought to the clinic, CAR-T has offered patients months or years of life after they had exhausted all other treatment options and would have died within weeks.
Its been incredible, said Marcela Maus, an immunologist and cell therapist at Mass General Cancer Center. Weve seen patients who had multiple lines of therapies and progressed after all of those, [then] get CAR-T and go into long-term remission.
But CAR-T does have hefty limitations, and scientists like Maus are researching ways to overcome some of its major shortcomings. These issues have prevented CAR-T from being used safely and effectively outside of leukemia and myeloma, and even patients who have responded spectacularly to CAR-T usually see their cancers return. The therapies are also still incredibly costly and carry risks, including a reaction known as a cytokine storm that can be life-threatening.
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Potential solutions to these problems are still in the early stages, but scientists are beginning to get a vision of what the future of CAR-T cell therapy might look like. It could involve synthetic biology to engineer a more advanced cell, or engineering other parts of the T cell to make it work better in the challenging environment around a tumor.
The field is growing tremendously, Maus said. Different people are working on different issues then, ideally, the data kind of decides whats going to be the next big thing.
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Heres a look at what experts see as some of the most promising approaches.
Current CAR-T cells use their CAR, or chimeric antigen receptor, to identify and kill cancer cells. These are synthetic proteins that bind to a specific target, like a protein on a cell surface membrane, and then activate the T cell to kill any cell carrying this target.
Armed with a CAR, T cells become pros at killing cancer cells that have their target, but theyll also kill normal cells that happen to carry the protein, too. Once a CAR-T cell is in the body, there isnt much a clinician can do to rein it in if it starts causing a lot of toxicity.
Once we let the CAR out, theyre like teenage kids, Maus said. You can maybe watch, but you cant really control them. So, theres some desire to be able to turn them on or off at will.
So, researchers are also trying to create CAR-T cells that they can manually activate or deactivate. As a group, these are known as controllable CARs, and most work by engineering an additional genetic circuit in the CAR-T cell. In theory, clinicians should be able to activate a switch on the genetic circuit that induces the CAR-T cell to activate their CAR and express it on the T cells surface membrane, thereby activating the receptor. Then, after a while, the T cell will disarm.
The goal is really getting our hands back on the steering wheel for a bit, Maus said.
There are several ways that synthetic biologists are doing this. In one example, researchers engineered a CAR with a protein switch that activates the receptor in the presence of blue light. In another example, researchers added a gene to CAR-T cells that force it to create its CAR and express it on the cell surface, thereby activating it, only in the presence of ultrasound radiation.
That way, it can be focused into a specific location, said Peter Yingxiao Wang, a synthetic biologist at University of California, San Diego, who works on controllable CARs. When the light or ultrasound is on the tumor locally, they can activate the CAR gene to trigger killing. Anywhere else, the CAR T-cells will be benign.
The idea is that the clinician can focus the light or ultrasound onto the tumor to get CAR-T cells to begin killing there. Once that signal is turned off, the CARs should disarm or slowly degrade and deactivate the CAR-T cells killing function. This way, even if the CAR does kill healthy tissue, the damage will theoretically be limited to the area around the tumor.
But this is an infant field right now, Wang added. A lot of these studies are just proof of concepts to show that theyre technically achievable. If you want to move to clinical trials, all of the components must be optimized.
Scientists also must show that theyre truly safe in humans, and that keeping the damage to a smaller surface area will be enough to outweigh the risks in treating tumors located near vital organs like the heart.
Other researchers are working on developing new CARs that can function like a biomolecular computer, able to make simple logical decisions to target cancer cells. Conventional CARs can cause dangerous toxicity because they only use one protein to identify cancer cells, and it may be impossible to discover the perfect target that exists only on cancer cells and not at all on healthy cells.
You can never uniquely define cancer or any other healthy tissue just by one marker, explained Wilson Wong, a synthetic biologist at Boston University. It just doesnt work. Its like trying to find a person and saying, he has black hair. Its like, oh, my God, youll never find him.
But it might be possible to distinguish cancer cells from healthy ones by looking at multiple proteins on a single cell. So, researchers like Wong have begun building more advanced CAR T-cells that use genetic circuits that only activate a CAR under more complex conditions, like the presence of several specific proteins that arent often seen in combination on healthy cells.
In this sense, the CAR is making a logical decision like basic Boolean computing, and synthetic biologists call this technique logic-gating.
Theres a lot of cool genetic circuits you can build, said Yvonne Chen, a synthetic biologist at UCLA. One can think of conditional systems to obliterate cancer cells. One can build OR-gates, AND-gates, and NOT-gates, and layer them on top of one another.
Although, Chen added, a drawback of logic-gating is that by increasing the complexity of the system, you might also be increasing the chance something goes wrong. Its important not to overcomplicate the design. Sophisticated circuits are exciting, but sometimes the solution itself causes problems. For example, for an AND-gate, you also make it easier for the tumor to escape. If the tumor loses either target A or B, it escapes from therapy, she said.
Another issue with conventional CAR-T therapy is that after a while, T cells can simply stop working. Solid tumors, like lung or pancreatic cancer, often have strategies to defend themselves from immune system attacks, including those from CAR-T cells. That makes it harder for CAR-T cells to treat solid tumors and can provide an opening for the tumor to return or progress.
So, researchers like Chen are working on armoring the CAR T-cell against the hostile signals in the microenvironment around a solid tumor. One of these signals is called TGF-beta, a protein which can help shut down T cell activity and help cancer cells avoid death and detection from the immune system. Chen was able to create a CAR cell that is not only resistant to TGF-beta, but can actually subvert the signal and become more deadly when it encounters TGF-beta.
Instead of being dysfunctional, they become activated, Chen said. That actually converts a tumor defense mechanism into a stimulatory signal for our T cells and tells them, youre in an environment where youre likely to encounter a tumor cell. Get ready.
Other scientists are working to keep CAR-T cells which can lose power over time functional for longer. Even with a good antigen, the T cells rapidly lose function, said Shivani Srivastava, an immunologist at the Fred Hutchinson Cancer Research Center who works on this problem. If you trigger a T cell or CAR over and over again, that causes the cell to become exhausted rather than turning into a memory cell or something else.
In one case, Stanford immunologist Crystal Mackall engineered a CAR-T cell that takes breaks before returning to work. She did this by creating a transient CAR that can be turned on or off. It can enhance [the T cells] function and limit how exhausted they are by giving them periodic rest, Srivastava said. Thats a really interesting strategy in principle.
But most of the tactics that scientists have tried so far in the realm of armored CAR-T cells havent worked in the long term, Srivastava said. You need a strategy that can help the CAR T-cells persist long enough to eradicate the cancer and prevent its return, which might be a lifelong project for the immune system.
Well have to find the right combination that will be durable, she said. Often we can find strategies that enhance function for only a short period of time.
Some future approaches might see T cells abandoned altogether. Scientists are slapping synthetic receptors on new or different cell types, such as natural killer cells. One company, called CoImmune, is putting CARs on a synthetic cell called a CIK cell, or cytokine-induced killer cell.
This is a novel cell type. They dont occur in nature, explained Charles Nicolette, the biotechs chief executive.
Theyre made by taking white blood cells and growing them while exposing them to certain immune molecules called cytokines. The advantage of creating new cell types is that biologists can combine certain useful traits from other immune cells, Nicolette said. For example, CIK cells could have the NK cells natural ability to distinguish normal cells from malignant ones and the CAR T-cells enhanced ability to kill.
One day, UCLAs Chen hopes to take this concept even further. To her, the ideal cancer-killing cell would not be derived from anything biological, but a completely artificial cell.
Instead of taking a cell from a patient, but rather build a completely defined, minimal cell that can do what we want and nothing else. It cannot evolve. Cannot mutate. Then, self-destruct when you dont want it there, she said. But, she added, creating synthetic cells like that would be unimaginably challenging, and it might not be possible to create a cell thats both persistent but also unchangeable.
Still, a scientist can dream.
Read more:
STAT's guide to the next generation of CAR-T therapies - STAT
Recommendation and review posted by Bethany Smith
Cancer Gene Therapy Market 2021 Industry Outlook, Comprehensive Insights, Growth and Forecast 2031 | Celgene, SIBIONO, Anchiano Therapeutic, Achieve…
TheCancer Gene Therapy market report is a useful foundation for people looking out for a comprehensive study and analysis of the Cancer Gene Therapy . This report contains a diverse study and information that will help you understand your niche and concentrate on key market channels in the regional and global space. To understand competition and take actions based on your key strengths you will be presented with the size of the, demand in the current and future years, supply chain information, trading concerns, competitive analysis and the prices along with vendor information. The report also has insights about key players, applications of Cancer Gene Therapy , its type, trends and overall market share.
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To set your business plan into action based on our detailed report, you will also be provided with complete and accurate prediction along with future projected figures. This will provide a broad picture of theindustryand help in devising solutions to leverage the key profitable elements and get clarity of the market to make strategic plans. The data present in the report is curated from different publications in our archive along with numerous reputed paid databases. Additionally, the data is collated with the help of dealers, raw material suppliers, and customers to ensure that the final output covers every minute detail regarding the Cancer Gene Therapy industry, thus making it a perfect tool for serious buyers of this study.
The research covers the current Cancer Gene Therapy market size and its growth rates based on records with company outline of Key players/manufacturers: GlaxoSmithKline plc, Adaptimmune Therapeutics plc, Merck & Co., Inc., bluebird bio, Inc., Shanghai Sunway Biotech Co., Ltd, Celgene, SIBIONO, Anchiano Therapeutic, Achieve Life Sciences, Inc., and Synergene Active Ingredients Pvt. Ltd.
TO UNDERSTAND HOW COVID-19 IMPACT IS COVERED IN THIS REPORT
COVID-19 can affect the global economy in three main ways: by directly affecting production and demand, by creating supply chain and market disruption, and by its financial impact on firms and financial markets. Our analysts monitoring the situation across the globe explains that the market will generate remunerative prospects for producers post COVID-19 crisis. The report aims to provide an additional illustration of the latest scenario, economic slowdown, and COVID-19 impact on the overall industry.
Regional Analysis
All the regional segmentation has been studied based on recent and future trends, and thedomainis forecasted throughout the prediction period. The countries covered in the regional analysis of the Global Cancer Gene Therapy market report are U.S., Canada, and Mexico in North America, Germany, France, U.K., Russia, Italy, Spain, Turkey, Netherlands, Switzerland, Belgium, and Rest of Europe in Europe, Singapore, Malaysia, Australia, Thailand, Indonesia, Philippines, China, Japan, India, South Korea, Rest of Asia-Pacific (APAC) in the Asia-Pacific (APAC), Saudi Arabia, U.A.E, South Africa, Egypt, Israel, Rest of Middle East and Africa (MEA) as a part of Middle East and Africa (MEA), and Argentina, Brazil, and Rest of South America as part of South America.
Key Benefits of the report:
This study presents the analytical depiction of the global Cancer Gene Therapy industry along with the current trends and future estimations to determine the imminent investment pockets.
The report presents information related to key drivers, restraints, and opportunities along with detailed analysis of the global Cancer Gene Therapy market share.
The current market is quantitatively analyzed from2021to2031to highlight the global Cancer Gene Therapy growth scenario.
Porters five forces analysis illustrates the potency of buyers & suppliers in the market.
The report provides a detailed global Cancer Gene Therapy market analysis based on competitive intensity and how the competition will take shape in the coming years.
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Table of Contents
Chapter 1: Cancer Gene Therapy Market Overview
Chapter 2: Global Economic Impact on Industry
Chapter 3: Global Market Competition by Manufacturers
Chapter 4: Global Production, Revenue (Value) by Region
Chapter 5: Global Supply (Production), Consumption, Export, Import by Regions
Chapter 6: Global Production, Revenue (Value), Price Trend by Type
Chapter 7: Global Market Analysis by Application
Chapter 8: Manufacturing Cost Analysis
Chapter 9: Industrial Chain, Sourcing Strategy and Downstream Buyers
Chapter 10: Marketing Strategy Analysis, Distributors/Traders
Chapter 11: Market Effect Factors Analysis
Chapter 12: Global Cancer Gene Therapy Market Forecast
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Cancer Gene Therapy Market 2021 Industry Outlook, Comprehensive Insights, Growth and Forecast 2031 | Celgene, SIBIONO, Anchiano Therapeutic, Achieve...
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JPM 2022: Licensing and research tie-ups trump M&A at conference in ‘reflection of last year’ – FierceBiotech
Just like at last year'sJ.P. Morgan Healthcare Conference,M&A is so 2019.
In the third winter of COVID-19, the drug development world hasnt executed on the type of megamergers we saw in 2019, such as Bristol Myers Squibbs $74 billion Celgene deal,which dropped in the lead-up to the healthcare industry's largest gathering.
Of course, we don't know whats happening in private Zoom rooms at the moment, or who managed to make some company-defining contacts at the second virtual edition of the conference.
Galapagos, with $5.6 billion to spend and a thinned late-stage pipeline, made it known Thursday morning that there are, in fact, private Zoom meetings to hash out potential acquisitions. The biopharmas chief business officer wasnt shy to say Galapagos is speaking with a host of companies as the company works on a rebound for 2022.
RELATED:2022 forecast: Biopharma M&A lags in 2021. Will drugmakers still look for bolt-on deals or large transactions?
But what was made public is that biopharmas stuck to licensing and research partnerships this week at JPM, extending a trend that has been going on for months. Backloaded, billion-dollar biobucks pacts are increasingly standard.
This year, I think, is a reflection of last year, and its much more licensing, much more tie-up, said Stuart Henderson, global life sciences industry leader at Accenture, in an interview with Fierce Biotech.
There are a few key factors driving the focus on licensing and collaborations, according to Henderson.
Premiums on acquisitions have increased, thus limiting affordability; venture capital funding has skyrocketed three times in the past five years, reducing the need for pharmas money; biotechs are going all the way to market on their own with tighter labels and more defined populations, shrinking the need for pharmas help in selling drugs; and the economic value of Big Pharma buying a late-stage biotech has declined, he said.
The virtual conference started with multiple licensing and research handshakes revealed Monday morning. Bayer kicked off the week with a $1 billionbiobucks beton a gene therapy deal with Mammoth, using the CRISPR science out of the lab of Jennifer Doudna, Ph.D.
RELATED:JPM22, Day 3: In short order, Perrigo part of a new landscape; Legend, J&J prep for cilta-cel launch
Next up was Novartis,ponying up$163 million to opt in on a COVID-19 antiviral after its collaborator Molecular Partners showed a 78% risk reduction in early-stage patients. Then, Pfizerdoled out$300 million upfront for Beam Therapeutics base editing technique. The COVID-19 vaccine maker madeanother two dealsMonday morning, including a financials-free agreement with Acuitas Therapeutics for its lipid nanoparticle delivery system for use in mRNA vaccines and therapies. Add to that a $100 million biobucks pact with Codex DNA.
This was allbefore 7 a.m. ET on Monday. Pfizers pandemic rival, Moderna, made some waves with its owndevelopment dealat $45 million upfront for Carisma Therapeutics in vivo engineered chimeric antigen receptor monocyte therapeutics in cancer. BioNTech followed with a $750 million milestone paymentspactwith Crescendo Biologics; Bristol Myers Squibb offered awhopping $3 billionin biobucks for cell therapies from Century Therapeutics; and Acadia was stoked tosigna $60 million upfront tie-up with Stoke Therapeutics.
Thats at least nine collaboration and licensing deals in the first day. You get the pattern. It was medtech that brought the M&A: Medtronicoffered$925 million for cardiac mapper Affera, and Exact Sciences willspend $190 millionon testing lab PreventionGenetics.
Henderson said some of the biotechs attracting licensing interest are those with platform technologies and digital biology companies that are attempting to cut the billion-dollar costs of drug discovery and development.
RELATED:JPM22, Day 4: Vertex aims to be cystic fibrosis leader into the 2030s; Gritstone hopes for new cancer biomarker; Galapagos' rebound year
The licensing announcements have slowed since those first days. But could that meanarms will be linked in coming weeks, as final negotiations are hashed out and due diligence is conducted? We hope so.
We know pharma is looking. Plenty of companiesPfizer, Moderna, BioNTech, Novartis and many morehave cash burning a hole in their pockets and pipelines to fill.
Recommendation and review posted by Bethany Smith
Cell and gene therapies: How to encourage and promote the move to market – Business Weekly
The evolution of advanced therapy medicinal products, or ATMPs, is bringing real world results, write James Fry, partner, and Isabel Teare, senior legal adviser from leading law firm Mills & Reeve.
The number of cell and gene therapy products now on the market around the world is near the 100 mark, according to recent research by McKinsey & Company, with many more in development.
These ground-breaking therapeutics can have a profound impact on previously intractable conditions. Products like Novartiss gene therapy Zolgensma, for example, are costly but have the potential to act as a single-dose cure for young patients with spinal muscular atrophy.
London-based Orchard Therapeutics has had some important wins with its ex vivo autologous gene therapy approach, as it seeks to address multiple therapeutic areas with profound unmet need.
The UKs powerful research base and established life sciences sector means that it is well placed to be part of this story. The Cell and Gene Therapy Catapult reports that, in 2020, the UK was host to around 12 per cent of ongoing ATMP global clinical trials some 154 separate studies. A striking statistic.
As more products are rolled out to patients, the journey for others both in overcoming technical obstacles and gaining public acceptance improves. But in the global race to bring these sophisticated products through from concept to clinic, some specific challenges stand in the way.
One of these is the need for a different style of regulatory engagement. The traditional methods used by regulators in dealing with small molecule pharmaceuticals show major limitations when applied to ATMPs.
Regulators have many years of in-depth experience with small molecule medicines. This can mean that regulatory guidance focuses on this section of the market and tends to be prescriptive in nature.
In contrast, regulation of large molecule, cell and gene therapies should, say those close to the industry, be based more on a philosophy rather than prescriptive rules, and should allow a greater degree of flexibility.
To remain at the forefront of this exciting sector, rapid technological progress needs to be matched by a surefooted regulatory response. With many ATMP projects created in the hands of smaller spin-outs or growing mid-sized companies there is no spare cash or capacity for mistakes.
Developers and regulators need to work together to make sure that safe and effective therapies can reach the patients that so badly need them.
A collaborative approach
Developers of innovative therapies can gain real benefits from early and regular engagement with regulatory agencies. The aim here is to smooth the product development path and, at the same time, avoid wasted effort and expense.
With development costs high, collaboration is a must to drive down complexity and uncertainty. The pressures of COVID-19 pandemic may have helped in strengthening the collaborative approach between regulators and developers, and many hope to see these changes embedded in day-to-day practice.
Think holistically
Regulatory strategy needs to form part of the overall development picture. For example, the shelf-life of ATMPs, and also their starting materials, can be very short.
Building an understanding of the practical issues into the supply chain is essential. Issues around cross-border transfers with possible delays for customs checks can seriously undermine the business model for this generation of products.
Likewise, point of care manufacturing may be a necessary element of treatment something regulators may allow for if they appreciate its importance.
New ways to shape clinical trials
Designing clinical trials of ATMPs to make sure that they are not excessively cumbersome or open-ended requires early planning and engagement.
Regulators are working on tools that can help innovators in this area, such as novel trial structures like basket or umbrella trials.
These can be deployed to evaluate multiple hypotheses with the overall goal of improving the efficiency of trial evaluation.
As clinical development progresses it may become necessary to adapt the trial, and regulators need to recognise this. Long term follow-up may also be necessary.
Regulators learning from each other
With advanced therapies setting new challenges, developers can take heart from seeing regulators learning from each other and sharing lessons internationally.
Many products in this area address rare diseases, so a coordinated approach makes sense in serving a group of patients spread around the world.
Likewise, regulators can benefit from looking to novel examples elsewhere.The Japanese authorities offer a special conditional approval that looks at safety and predicted efficacy, before allowing products a provisional authorisation for the market. This enables treatment availability to patients for a number of years during which evidence to establish efficacy is gathered.
An unexpected benefit
The COVID-19 pandemic, while stretching healthcare provision to its utmost, has had some positive impacts. Widespread recognition of the importance of life science innovation is one.
But accelerating new approaches and ways of working with and between regulators is another less obvious benefit. We have looked at how this can strengthen and support cell and gene therapy development and are optimistic that these changes are here to stay.
And the UK governments recent Life Sciences Vision shows a real commitment to pushing through in many of these areas.
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Cell and gene therapies: How to encourage and promote the move to market - Business Weekly
Recommendation and review posted by Bethany Smith
UHN and U of T receive $24-million federal grant for transplant research – News@UofT
Researchers atUniversity Health Network (UHN) and the University of Toronto have received $24 million to advancetechnology to repair and rebuild organs outside the bodyfor patients in need.
The project, led byShaf Keshavjee, is one of only seven across Canada selected to receive funding in the Government of CanadaNew Frontiers in Research Fund(NFRF) Transformation competition, following an international consultation.
"The Ex Vivo Lung Perfusion (EVLP) system we developed here in Toronto has revolutionized lung transplantation in the past decade. Now, it's been translated around the world to increase lung transplant access and it's being extended to other organs," says Keshavjee, a professor and vice-chair for innovation in thedepartment of surgeryin U of Ts Temerty Faculty of Medicinewho is surgeon-in-chief at UHN and a senior scientist atToronto General Hospital Research Institute.
"With this transformative grant, we now have the opportunity to take ex vivo technology to the next level, where we can repair and rebuild organs for transplant."
Atul Humar, director of the AjmeraTransplant Centre(photo byTim Fraser)
Over 4,500 people in Canada are currently waiting for an organ transplant, and more than 270 die each year as the need for transplant greatly exceeds availability.
Ex vivo perfusion systems use specialized machines to maintain, evaluate and treat organs before transplant. They have a huge impact on increasing the number of organs that can be considered for transplant.
TheToronto Lung Transplant Program,led by Keshavjee, has used this technology to double the number of lung transplants performed and lives saved at UHN.
"The New Frontiers grant will allow us to advance applications for lungs and further develop ex vivo systems for other organs, such as liver, kidney, heart and pancreas," says Atul Humar, a co-principal investigator on the project, professor in thedepartment of medicineat U of T and director of theAjmera Transplant Centre at UHN.
Brad Wouters, UHN's executive vice president, science and research, notes that this major grant will enable multidisciplinary teams to develop new, cutting-edge approaches to extend the time that donated organs can be used, and also enable treatment and repair of unsuitable organs to allow treatment of more patients.
It will also help the teams refine and improve equitable organ allocation guidelines for all patients, he adds.
The advancements that this team has made and their continued success is made possible by support from provincial and federal governments, industry partners, external charitable agencies, generous philanthropy from the UHN Foundation and our incredible patient partners, says Wouters, who is also a professor in thedepartment of radiation oncologyat U of T. This award recognizes the tireless efforts of the team, and this support, which have been key to achieving global impact.
The New Frontiers Research Fund was designed to support large-scale, Canadian-led interdisciplinary research projects with the potential to realize real and lasting change.
The fund falls under the strategic direction of theCanada Research Coordinating Committeeand is administered by the Tri-Agency Institutional Programs Secretariat on behalf of Canada's three research granting agencies: theSocial Sciences and Humanities Research Council, theCanadian Institutes of Health Researchand theNatural Sciences and Engineering Research Council.
Over the course of this project, the team of over 20 researchers at U of T, UHN, national and international partner sites will develop sophisticated ex vivo platforms to:
Longer ex vivo preservation prior to transplant will enable many world-first therapeutic applications that will, ultimately, create more organs for clinical transplant.
One example is to use gene therapy to make an organ more like the recipient's cells and help to address the current hurdle of organ rejection by the immune system. Researchers at UHN are also working on changing an organ's blood type so the sickest people can get access to the next available organ, instead of waiting for one that exactly matches their blood a delay that currently can take several months before a match is found.
Another transformative goal is to use medicines and light therapies in the ex vivo circuit to eliminate viral or bacterial infections that previously prevented an organ to be considered for transplant.
"This grant gives us a unique opportunity to extend personalized medicine to every organ group," saysMarcelo Cypel, a professor in the department of surgery at U of T and surgical director of the Ajmera Transplant Centre, who is also a co-principal investigator on the project.
"Not only will it enable longer preservation, this research will let us treat and improve organs. It has the potential to change the paradigm in the field of transplantation."
The change will include several advanced applications, such as the engineering of new organs using stem cells with the goal to make organs available for all in need. Significant progress has already been made in generating new kidneys, lungs and tracheae (windpipe), and their applications will be tested further during the six-year project term.
With the involvement of a multidisciplinary team housed in a world-class centre at UHN, the project will bring personalized medicine to transplant, and go beyond solid organs.
Siba Haykal, plastic and reconstructive surgeon and project co-principal investigator, will lead research involving vascularized composite allotransplantation the transplant of limbs, face, trachea and composite tissues, such as skin and muscles.
"These are very delicate tissues that can't survive outside the body for very long and are very susceptible to rejection," she explains, adding that the current treatment involves high doses of life-long anti-rejection medication for transplant recipients.
Haykal and the team want to develop a system to preserve limbs and tissues out of the body without blood flow for longer periods. This will enable the application of new cell therapies to adapt these tissues to the recipient prior to surgery.
"Whether they have been disfigured by burns or from trauma or cancer, if they've had an amputation and need prosthetic limbs or if they require a new airway, transplantation provides hope for these patients who currently don't have many options," says Haykal, who is an assistant professor in the department of surgery at U of T.
"If we can use techniques that reduce the amount of anti-rejection medication and maybe one day get to a stage where they don't need it anymore, that would have a huge impact on the patient's quality of life."
Humar adds, "I have seen so many people who have literally been at death's door and have been completely turned around by transplant and live a full and healthy life. If we can offer that to more patients, then for me that would be an incredible achievement.
"This funding will also help us disseminate our knowledge, and facilitate other hospitals across Canada and around the world build upon what we're doing at UHN."
This story wasoriginally postedon the University Health Network website.
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UHN and U of T receive $24-million federal grant for transplant research - News@UofT
Recommendation and review posted by Bethany Smith
Rising Focus on Exploring Potential of Stem Cells as Therapeutic Tools in Drug Targeting and Regenerative Medicine to Fuel Revenue Growth of Stem…
NEW YORK, Jan. 10, 2022 /PRNewswire/ --Reports and Data has published its latest report titled "Stem Cells Market By Product (Adult Stem Cells, Human Embryonic Stem Cells, IPS Cells, and Very Small Embryonic-Like Stem Cells), By Technology (Cell Acquisition, Cell Production, Cryopreservation, and Expansion & Sub-Culture), By Therapies (Allogeneic Stem Cell Therapy and Autologous Stem Cell Therapy), and By Application (Regenerative Medicine and Drug Discovery & Discovery), and By Region Forecast To 2028."
According to the latest report by Reports and Data, the global stem cells market size was USD 10.13 billion in 2020 and is expected to reach USD 19.31 Billion in 2028 and register a revenue CAGR of 8.4% during the forecast period, 2021-2028.
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Drivers, Restraints, & Opportunities
Stem cells are cells that have the potential to differentiate into different types of cells in the body. Stem cells have the ability of self-renewal and differential into specialized adult cell types. Stems cells are being explored for their potential in tissue regeneration and repair and in treatment of chronic diseases. Increasing number of clinical trials are underway to assess and establish safety and efficacy of stem cell therapy for various diseases and disorders. Rapid advancement in stem cell research, rising investment to accelerate stem cell therapy development, and increasing use of stem cells as therapeutic tools for treatment of neurological diseases and malignancies are some key factors expected to drive market revenue growth over the forecast period. in addition, growing incidence of type 1 diabetes, spinal cord injuries, Parkinson's diseases, and Alzheimer's disease, among others have further boosted adoption of stem cell therapies and is expected to fuel revenue growth of the market going ahead.
Stem cells are basic cells in the body from which cells with specialized functions are generated such as heart muscle cells, brain cells, bone cells, or blood cells. Maturation of stem cells into specialized cells have enabled researchers and doctors better understand the pathophysiology of diseases and conditions. Stem cells have great potential to be grown to become new tissues for transplant and in regenerative medicine. Stem cells that are programmed to differentiate into tissue-specific cells are widely being used to test new drugs that target specific diseases, such as nerve cells can be generated to test safety and efficacy of drugs that are being developed for nerve disorders and diseases. Stem cells are of two major types: pluripotent cells that can differentiate into any cells in the adult body and multipotent cells that are restricted to differentiate into limited population of cells. Increasing clinical research is being carried out to advance stem cell therapy to improve cardiac function and to treat muscular dystrophy and heart failure. Recent progress in preclinical and clinical research have expanded application scope of stem cell therapy into treating diseases for which currently available therapies have failed to be effective. This is expected to continue to drive revenue growth of the market going ahead.
However, immunity-related concerns associated with stem cell therapies, increasing incidence of abnormalities in adult stem cells, and rising number of ethical issues associated with stem cell research such as risk of harm during isolation of stem cells, therapeutic misconception, and concerns surrounding safety and efficacy of stem cell therapies are some key factors expected to restrain market growth to a certain extent over the forecast period.
To identify the key trends in the industry, research study at https://www.reportsanddata.com/report-detail/stem-cells-market
COVID-19 Impact Analysis
Rising use of Human Embryonic Stem Cells in Regenerative Medicine to Drive Market Growth:
Human embryonic stem cells (ESCs) segment is expected to register significant revenue growth over the forecast period attributable to increasing use of human embryonic stem cells in regenerative medicine and tissue repair, rising application in drug discovery, and growing importance of embryonic stem cells as in vitro models for drug testing.
Cryopreservation Segment to Account for Largest Revenue Share:
Cryopreservation segment is expected to dominate other technology segments in terms of revenue share over the forecast period. Cryopreservation techniques are widely used in stem cell preservation and transport owing to its ability to provide secure, stable, and extended cell storage for isolated cell preparations. Cryopreservation also provides various benefits to cell banks and have numerous advantages such as secure storage, flexibility and timely delivery, and low cost and low product wastage.
Regenerative Medicine Segment to Lead in Terms of Revenue Growth:
Regenerative medicine segment is expected to register robust revenue CAGR over the forecast period attributable to significant progress in regenerative medicine, increasing research and development activities to expand potential of stem cell therapy in treatment of wide range of diseases such as neurodegenerative diseases, diabetes, and cancers, among others, and rapid advancement in cell-based regenerative medicine.
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North America to Dominate Other Regions in Terms of Revenue Share:
North America is expected to dominate other regional markets in terms of revenue share over the forecast period attributable to increasing adoption of stem cell therapy to treat chronic diseases, rising investment to accelerate stem cell research, approval for clinical trials and research studies, growing R&D activities to develop advanced cell-based therapeutics, and presence of major biotechnology and pharmaceutical companies in the region.
Asia Pacific Market Revenue to Expand Significantly:
Asia Pacific is expected to register fastest revenue CAGR over the forecast period attributable to increasing R&D activities to advance stem cell-based therapies owing to rapidly rising prevalence of chronic diseases such as cancer and diabetes, rising investment to accelerate development of state-of-the-art healthcare and research facilities, establishment of a network of cell banks, increasing approval for regenerative medicine clinical trials, and rising awareness about the importance of stem cell therapies in the region.
Major Companies in the Market Include:
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Market Segmentation:
For the purpose of this report, Reports and Data has segmented the stem cells market based on product, technology, therapies, application, and region:
Product Outlook (Revenue, USD Billion; 2018-2028)
Technology Outlook (Revenue, USD Billion; 2018-2028)
Therapy Outlook (Revenue, USD Billion; 2018-2028)
Application Outlook (Revenue, USD Billion; 2018-2028)
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Regional Outlook (Revenue, USD Billion, 2018-2028)
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Genotyping assay marketsize was USD 17.7 Billion in 2020 and is expected to register a robust CAGR of 22.4% during the forecast period. Key factors driving market revenue growth are increasing prevalence of genetic disorders such as Turner syndrome, Alzheimer's, hemophilia, and Parkinson's, and rising demand for genotyping assays globally.
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Rising Focus on Exploring Potential of Stem Cells as Therapeutic Tools in Drug Targeting and Regenerative Medicine to Fuel Revenue Growth of Stem...
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Robert Russell: We have gone to the dogs – Shreveport Times
Robert Russell| Special to the Times
Recently on CBS Mornings, host Tony Dokoupil, presented a story about dogs. An experiment aimed to see if dogs could distinguish between their owners native tongue and a foreign language. The experiment used brain scan technology, and sure enough, different parts of their brains activated based on whether they were familiar with the spoken language or not.
Dogs are mans best friend, or so they say. According to James Gorman, of the New York Times Magazine of October 4, 2021, all dogs are descended from the first dogs, just as all humans can trace their ancestry to the first Homo sapiens." Thus, none of us, or our dogs, have a more ancient ancestry than any other. Breeding led some dogs to be big and black, some small and white, some are strong, and some run fast.
Dogs were prized early on for their sense of smell, and with proper husbandry, they developed the genetics and physiology to make them sniffing machines.
Dogs have genes that improve olfactory ability, and more olfactory nerve cells than humans do. In addition, for centuries now, the Gentry have taken advantage of this intense sense of smell and sharp vision, to hunt prey and search for specialty items like truffles.
Today law enforcement uses dogs to locate drugs, like marijuana. Dogs have absolutely no interest in drugs, but they do enjoy games with a handler. Their training has led them to associate a toy, with the scent of various drugs. Trainers play a vigorous game of tug-of-war with dogs and a favorite towel. To begin the training, they play with a carefully washed unscented towel.
Later play involves a target substance hidden inside the towel. After playing, the dog starts to recognize that as that of his favorite toy. The trainer hides the towel, with the drugs, in typical places, and with the odor identified, he points, to get at the toy. The reward is a game of tug-of-war. Commercially the wine industry is working with dogs to identify spoiled corks and wines with various chemical compounds. They can even detect flaws in the oak barrels and corks, which lead to faults like TCA, VA, and Brett. In another use, a wine festival recently used dogs to sense attendees infected with COVID-19.
Dogs can detect many human diseases. This learning and evolving are not just improving animal skills; they can save lives.
Through human evolution, male eyes are sensitive visually to small details and moving objects, while women are more aware of color changes, according to a vision study, in The Journal of Biology and Sex Differences, referenced in this column a few years ago, noting that women see things differently. Women seem to have a superior tasting and visual skills, on average, to those of men. Since the genes of retinal cone cells are responsible for color perception, they are located on the X-chromosome, and women have two X-chromosomes-this all makes sense.
Secondly, the literature finds that in most cases females had better sensitivity, and discriminated and categorized odors better than males, as this writer can personally attest. Ancient humans lived as hunter-gatherers. Men predominately were the hunters while women were gatherers. Being a successful hunter does not require being good at smelling and tasting, but being, a successful gatherer of plant-based foods does. If you gather fruits and seeds, you need to be good at discriminating poisonous or spoiled foods, from nonpoisonous or fresh ones. Thus, men that did not hunt well, and women that did not taste and smell well, evolutionarily eliminated from the herd.
According to Mia Rozenbaum, at Understanding Animal Research, Dogs have about 220 million scent receptors humans only have 5 million. Dogs have smell receptors 10,000 times more accurate than humans, she notes their nose is powerful enough to detect substances at concentrations of one part per trillion - a single drop of liquid in 20 Olympic-size swimming pools. Dogs can detect cancer by smell, and sniff out a variety of types including skin cancer, breast cancer and bladder cancer using samples from known cancer patients, and people without cancer.
In a 2006 study, five dogs were trained to detect cancer based on breath samples. Once trained, the dogs were able to detect breast cancer with 88 percent accuracy, and lung cancer with 99 percent accuracy. They could do this regardless of the stage of the disease. More recently, a study has even shown that dogs can use their highly evolved sense of smell to pick out blood samples from people with cancer, with almost 97 percent accuracy.
Besides cancer and malaria, dogs can also detect Parkinson's disease, with a myriad of other studies now underway. Perhaps dogs may soon be the winemakers best friend, or so this writer says.
Stay healthy, and Cheers!
You can contact Robert Russell at rob@rlr-appraisals.com.
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Robert Russell: We have gone to the dogs - Shreveport Times
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UPDATE – Bionano Genomics Hosts Day 1 of 2022 Symposium with Six Presentations Highlighting the Superior Performance of OGM in Variant Detection for…
SAN DIEGO, Jan. 10, 2022 (GLOBE NEWSWIRE) -- Bionano Genomics, Inc. (BNGO), pioneer of optical genome mapping (OGM) solutions on the Saphyr system and provider of NxClinical, the leading software solutions for visualization, interpretation and reporting of genomic data, hosted today the first of four days of 2022 Symposium, the Companys premiere event showcasing OGM research applications across key clinical areas of constitutional genetic disease, hematologic malignancies, solid tumors and OGM combined with next-generation sequencing (NGS).
Six presentations from leading researchers across North America and Europe kicked off Symposium. Todays event featured six informative speakers from leading researchers across North America and Europe. These presentations covered a wide range of constitutional genetic diseases in pre- and postnatal genetics and offered insight into potential applications in infertility and reproductive medicine. The presenters supported OGM as an effective alternative to traditional workflows such as karyotype, fluorescent in situ hybridization (FISH), chromosomal microarray (CMA) and Southern blot. Research presentations have demonstrated greater sensitivity, better resolution and faster results from OGM workflows compared to traditional methods.
Performance of OGM evaluated in pre- and postnatal samples. Both Dr. Iqbal from University of Rochester Medical Center and Dr. Shirley Heggarty compared the performance of OGM in the evaluation of pre- and postnatal samples with known chromosomal aberrations. These studies found a high concordance of OGM results compared to traditional methods. In addition, OGM was able to identify both unbalanced structural chromosome abnormalities and balanced structural variants (SVs), like translocations and inversions, that chromosomal microarray (CMA) could not.
Capabilities of OGM to measure repeat expansions were evaluated. Dr. Alexander Hoischen specifically explored the capabilities of OGM to map repeat expansions, which can be particularly challenging types of SVs, in subjects with Canvas syndrome and myotonic dystrophy types 1 and 2. The OGM workflow was able to immediately call very large insertions with greater precision than the standard cytogenetic techniques and with 100% concordance. OGM was shown to allow researchers the ability to see more of the genome, while being less time-consuming and labor-intensive than other molecular methodologies like Southern blotting, as Dr. Nikhil Sahajpal discovered. Results from his validation study were 100% concordant with traditional methods and demonstrated a streamlined laboratory workflow for different sample types.
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The role of OGM as a discovery tool in reproductive disorders including infertility. In addition to pre- and postnatal applications, two speakers, Dr. Laila El-Khattabi and Chaim Jalas, shared how the OGM workflow plays a significant role in their research in infertility and reproductive disorders. In her study, Dr. El-Khattabi used OGM to characterize apparently balanced SVs related to male infertility and identify new genes involved in reproductive disorders. Chaim Jalas shared how OGM used in preimplantation genetic diagnosis can identify structural rearrangements, including balanced translocations and inversions, in embryos prior to transfer, which they indicated could improve pregnancy and delivery rates in an IVF setting.
Among 37 posters appearing in the virtual exhibition hall the top poster per application area selected to compete for best poster of Symposium. In addition to the oral presentations, 37 posters were received and selected for presentation in the virtual exhibition hall. These posters, in the virtual exhibition hall, were voted on by attendees of Symposium and a winner was named in each of four key application areas based on total number of votes. Below are the four poster winners:
Constitutional cytogenomics: Dr. Catherine A. Brownstein, Boston Childrens Hospital
Hematologic malignancies: Dr. Jonathan L. Lhmann, Hannover Medical School
Solid tumors: Dr. Miriam Bornhorst, Childrens National Hospital
OGM + NGS: Dr. Nikhil Sahajpal, Augusta University
Sincere congratulations to our poster winners and thanks to all poster authors for sharing emerging research on OGM from their laboratories, remarked Alka Chaubey, PhD, FACMG, chief medical officer of Bionano. This event is made possible with the enthusiastic participation of the OGM community.
We are thrilled at this strong kick-off to the 2022 Symposium and we are excited about the data shared today by experts from around the world that continues to demonstrate the utility of OGM workflows in variant detection for constitutional genetic diseases, commented Erik Holmlin, PhD, president and chief executive officer of Bionano. Congratulations to the poster winners. We are impressed by all the discoveries our customers are making with OGM towards the goal of elevating human health.
Dont miss Symposium, register now! Symposium registration is open to all and there is no charge for attending this event. Register today at https://www.labroots.com/ms/virtual-event/bngo2022
About Bionano Genomics
Bionano Genomics is a provider of genome analysis solutions that can enable researchers and clinicians to reveal answers to challenging questions in biology and medicine. The Companys mission is to transform the way the world sees the genome through OGM solutions, diagnostic services and software. The Company offers OGM solutions for applications across basic, translational and clinical research. Through its Lineagen business, the Company also provides diagnostic testing for patients with clinical presentations consistent with autism spectrum disorder and other neurodevelopmental disabilities. Through its BioDiscovery business, the Company also offers an industry-leading, platform-agnostic software solution, which integrates next-generation sequencing and microarray data designed to provide analysis, visualization, interpretation and reporting of copy number variants, single-nucleotide variants and absence of heterozygosity across the genome in one consolidated view. For more information, visit http://www.bionanogenomics.com, http://www.lineagen.com or http://www.biodiscovery.com.
Forward-Looking Statements of Bionano Genomics
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as may, will, expect, plan, anticipate, estimate, intend and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) convey uncertainty of future events or outcomes and are intended to identify these forward-looking statements. Forward-looking statements include statements regarding our intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things, the ability for additional data to support the strength of OGM workflows as compared to traditional workflows, the ability and utility of OGM to analyze genomes and reveal answers in genetic disease and cancer research in less time-consuming and less-labor intensive manners than traditional workflows, and the potential for OGM to become part of the standard of care. Each of these forward-looking statements involves risks and uncertainties. Actual results or developments may differ materially from those projected or implied in these forward-looking statements. Factors that may cause such a difference include the risks and uncertainties associated with: the impact of the COVID-19 pandemic on our business and the global economy; general market conditions; changes in the competitive landscape, including the introduction of competitive technologies or improvements in existing technologies; failure of future study results to support those demonstrated during the presentations referenced in this press release; changes in our strategic and commercial plans; our ability to obtain sufficient financing to fund our strategic plans and commercialization efforts; the ability of medical and research institutions to obtain funding to support adoption or continued use of OGM or our technologies; and the risks and uncertainties associated with our business and financial condition in general, including the risks and uncertainties described in our filings with the Securities and Exchange Commission, including, without limitation, our Annual Report on Form 10-K for the year ended December 31, 2020 and in other filings subsequently made by us with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on managements assumptions and estimates as of such date. We do not undertake any obligation to publicly update any forward-looking statements, whether as a result of the receipt of new information, the occurrence of future events or otherwise.
CONTACTSCompany Contact:Erik Holmlin, CEOBionano Genomics, Inc.+1 (858) 888-7610eholmlin@bionanogenomics.com
Investor Relations:Amy ConradJuniper Point+1 (858) 366-3243amy@juniper-point.com
Media Relations:Michael SullivanSeismic+1 (503) 799-7520michael@teamseismic.com
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UPDATE - Bionano Genomics Hosts Day 1 of 2022 Symposium with Six Presentations Highlighting the Superior Performance of OGM in Variant Detection for...
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A Novel Mutation in the TRPM4 Gene | RRCC – Dove Medical Press
Introduction
Long QT syndrome (LQTS) is defined by a prolonged QT interval accompanied by morphological abnormalities in the T and/or U waves on the electrocardiograph (ECG).1 The primary clinical symptom of LQTS is syncope produced by ventricular arrhythmias.24 The clinical diagnosis of LQTS is based on a combination of the patients medical and family history, as well as the 12-lead ECG.5 According to the guidelines, LQTS diagnosis can be made in case the QTc is more than 460ms, and the patient presents some antecedents, most notably a family history of SCD and unexplained syncope.6
LQTS can be classified into two types based on its etiology: congenital LQTS (cLQTS) and acquired LQTS (aLQTS). While the former is a relatively rare genetic cardiovascular disease with a low incidence rate (1/2000-1/3000),7 the latter is frequently subsequent to electrolyte disorders, cardiomyopathy, cerebrovascular accidents, and autonomic dysfunction.
The pathogenesis of cLQTS is related to the mutation of genes encoding for ion channels, such as KCNH2,3,8 KCNQ1,2,9 TRPM4,1012 and so on, and causing ion channel dysfunction with reduced repolarization ion flow and/or increased delocalization ion flow, which in turn leads to prolonged repolarization. Among ion channel genes, mutations in KCNQ1 and KCNH2, which encode voltage-gated K+ channels involved in cardiac action potential (AP) repolarization are most common,10 followed by mutations in SCN5A which encode voltage-gated Na (1.78%), while mutations in other genes including TRPM4 are rare (below 1% of LQTS).11 Dr. Hof and colleagues were the first to hypothesize that TRPM4 mutations cause long QT syndrome, and they detected four TRPM4 variants, including c.1321 G >A, c.1495 C >T, c.1496 G >C, and c.2531 G >A, with no changes in the key LQTS genes.11
Herein, we reported a Chinese proband with cLQTS with a new mutation (NM_017636: exon4: c.434delC, p. Ala145ValfsTer133) in the TRPM4 with the hope that this report may be helpful in future genetic studies and prospective, genetically informed research.
A 75-year-old male was implanted with a permanent pacemaker 18 years ago due to a local diagnosis of bradycardia characterized by recurrent syncope since the age of 20, yet postoperative syncope continued to occur. Syncope occurred again a day before admission, and then he was taken to our hospital. Electrocardiography (ECG) at disease onset indicated sinus bradycardia, anterior wall T wave changes with visible u waves (Figure 1).
Figure 1 The admission ECG showed sinus bradycardia with QTc interval 432ms and U wave.
On admission, the following vital signs were recorded: blood pressure of 135/88mmHg, pulse rate of 59 beats per minute, the body temperature of 36.4C, and breathing rate of 18 beats per minute. Physical examination revealed no evidence of heart failure or pathological nervous system features.
After admission, repeated electrocardiograms suggested prolonged QT intervals, sinus bradycardia, and T wave changes (Figure 2). Ambulatory ECG also showed sinus bradycardia (mean heart rate 59 beats), prolonged QT interval (540ms), and torsade de pointes (Figure 3). Whats more, the electrodes on the patients pacemaker were discovered to be depleted for nearly five years. Laboratory data showed a slightly elevated level of troponin, as well as N-terminal-pro-brain natriuretic peptide, while other laboratory indexes including hepatic and renal function, electrolytes, coagulation, and inflammatory indexes were normal. We also performed a cranial MRI on this patient, and no neurological lesion was found that could cause syncope. Echocardiography indicated no abnormalities in cardiac structure, and the left ventricular ejection fraction was 61%. Moreover, selective coronary angiography was performed, indicating that the patient has no apparent pathological stenosis in the coronary arteries.
Figure 2 (AC) During the hospitalization, the ECG showed the dynamic changes of T wave; the longest QTc interval was 540ms.
Figure 3 Electrocardiogram monitoring shows torsion de pointes tachycardia.
According to the above results and the diagnostic criteria of LQTS, a highly suspected diagnosis of LQTS was finally made (Rating 6.5 out of 5).1,13,14
Then the etiology of LQTS was further explored. For no acquired LQTS associated risk factors such as electrolyte disorders, cardiomyopathy, cerebrovascular accidents, and autonomic dysfunction were found in the patients previous medical history and related examinations after admission, we are suspicious of the patients Genetics of LQTS.
After obtaining the informed consent, we conducted whole-exome sequencing (WES) on the patient and his family to confirm our diagnosis. Gene testing revealed that the patient carried a TRPM4 heterozygous shift mutation (NM_017636: exon4: c.434delC, p. Ala145ValfsTer133). Moreover, WES analysis of his family members revealed that his sister carried the same TRPM4 mutation as the patient (Figure 4), but his two brothers and son did not. Regrettably, the probands parents have all died, and hence their genes have not been obtained.
Figure 4 The results of genetic testing showed the proband and his sister carried a TRPM4 heterozygous shift mutation (NM_017636: exon4: c.434delC, p. Ala145ValfsTer133) (A), while his two brothers and son did not (B).
Because of the high risk of sudden cardiac death, we recommend implanting a cardioverter defibrillator (ICD) for the patient. Due to economic reasons, the patient refused. Due to the patients strong preference for cautious treatment, we administered Shengsong Yangxin Capsule as a placebo.
cLQTS is a rare cardiac disorder inherited in an autosomal trait, with an estimated incidence of 1:20001:3000.7 It is accepted that cLQTS is a rare ion channelopathy, and a host of genes were described to be responsible for LQTS. So far, 15 genes with more than 7000 mutations have been considered to be associated with cLQTS.15 Among the six genes encode for a pore-forming ion channel, while others encode for regulatory subunits or proteins. Mutations in KCNQ1 (3035%) and KCNH2 (2530%) encoding voltage-gated K+ channels involved in cardiac action potential (AP) repolarization are the most common among ion channel genes,10,14 followed by mutations in SCN5A, which encode voltage-gated Na+ (1.78%).11,14 In comparison, mutations in other genes, including TRPM4 are rare (below 1% of LQTS).11,12,14
As far as the pathology of LQTS, it is generally known that QT duration depends on both ventricular AP duration and AP propagation within the ventricle and ion channel dysfunction with reduced repolarization ion flow and/or increased delocalization ion flow leads to prolonged repolarization. According to a sack of animal experiments on TRPM4, TRPM4 affects cardiac electrophysiological activity through nonselective cation permeability, which leads to cLQTS.11 Unfortunately, additional research is required to decipher the biological mechanism underlying TRPM4-induced loss of function of nonselective cation permeability.
Above all, gene test counts for cLQTS. The importance of gene detection for cLQTS lies in exploring its pathogenic mechanism and its treatment, for the drugs targeted specific ion channels can be used with exerting maximal effects.
In our case, a new mutation site on TRPM4 (NM_017636: exon4: c.434delC, p. Ala145ValfsTer133) was discovered through whole-exon detection, which can provide a brand-new direction for gene screening of patients with cLQTS and further complements its diagnostic criteria. As for the detail of gene tests, we used PolyPhen2 to predict whether a new mutation is damaging to the resultant protein function. And then, according to the relevant literature, we did consider that TRPM4 is as same amino acid change as a previously established pathogenic variant regardless of nucleotide change after searching the OMIM database. But the absence of the literature for molecular pathology makes us failure to achieve the information of damaged protein. At last, combined clinical history, ECG, and the results of gene test, it was suspected that TRPM4 mutation (NM_017636: exon4: c.434delC, p. Ala145ValfsTer133) was the pathogenic variant.
In the treatment of cLQTS, beta-blockers effectively prevent cardiovascular disease and ventricular arrhythmia, and ICD implantation is regarded as the ultimate therapy.16 Because of the high risk of sudden cardiac death, we recommend implanting a cardioverter defibrillator (ICD) for the patient. Due to economic reasons, the patient refused, and we administered a placebo.
The incidence of cLQTS is very low, with the incidence of LQTS caused by TRPM4 being even lower, leading to less research on the gene TRPM4. Therefore, we reported a new mutation in TRPM4 (NM_017636: exon4: c.434delC, p. Ala145ValfsTer133) to provide more evidence for gene screening, to improve the detection rate of healthy gene carriers or patients in the early incubation stage, thereby providing further complements to the clinical data of the study about TRPM4. Notwithstanding its limitation such as lack of this patients past clinical data that can help to compare the symptom before and after the permanent pacemaker implantation, detailed information of the pedigree of this patients parents and so on, this report does hopefully serve as useful feedback information for genetic pathogenesis of cLQTS caused by TRPM4 variants.
cLQTS, congenital long QT syndrome; LQTS, long QT syndrome; ECG, electrocardiograph; AP, action potential; ICD, implanting cardioverter defibrillator; WES, whole-exome sequencing; TRPM4, transient receptor potential melastatin 4; aLQTS, acquired LQTS.
All relevant data supporting the conclusions of this article are included within the article.
The need for institutional ethics approval for this case report was waived. Written informed consent was obtained from the patient for publication of this case report and accompanying images.
The patient has provided informed consent for the publication of the case. A copy of the written consent is available for review by the Editor-in-Chief of this journal.
Dr. Rui Huang and Dr. Yinhua Luo are co-first authors for this study.
All authors made substantial contributions to conception and design, acquisition of data, or analysis and interpretation of data; took part in drafting the article or revising it critically for important intellectual content; agreed to submit to the current journal; gave final approval of the version to be published; and agree to be accountable for all aspects of the work.
This work was supported in part by the National Natural Science Foundation of China (82160072) and the Science and Technology Support Project of Enshi Science and Technology Bureau (D20210024).
The authors declare that they have no conflicts of interest.
1. Vohra J. The long QT syndrome. Heart Lung Circ. 2007;16(Suppl 3):S5S12. doi:10.1016/j.hlc.2007.05.008
2. Beiyin G, Tingliang L, Lei Y, et al. Head-up tilt test induces T-wave alternans in long QT syndrome with KCNQ1 gene mutation: case report CARE-compliant article. Medicine. 2020;99(20):e19818.
3. Henk-Jan B, Lucia B. Orgasm induced torsades de pointes in a patient with a novel mutation with long-QT syndrome type 2: a case report. Eur Heart J Case Rep. 2018;2(2):yty062.
4. Joel G, Kinsley H, Amanda W, et al. Recurrent torsades with refractory QT prolongation in a 54-year-old man. Am J Case Rep. 2018;19:1515.
5. Priori SG, Wilde AA, Horie M, et al. HRS/EHRA/APHRS expert consensus statement on the diagnosis and management of patients with inherited primary arrhythmia syndromes: document endorsed by HRS, EHRA, and APHRS in May 2013 and by ACCF, AHA, PACES, and AEPC in June 2013. Heart Rhythm. 2013;10(12):19321963. doi:10.1016/j.hrthm.2013.05.014
6. Priori SG, Blomstrm-Lundqvist C, Mazzanti A, et al. [2015 ESC guidelines for the management of patients with ventricular arrhythmias and the prevention of sudden cardiac death]. Kardiol Pol. 2015;73(10):795900. Croatian. doi:10.5603/KP.2015.0190
7. Zumhagen S, Stallmeyer B, Friedrich C, et al. Inherited long QT syndrome: clinical manifestation, genetic diagnostics, and therapy. Herzschrittmacherther Elektrophysiol. 2012;23(3):211219. doi:10.1007/s00399-012-0232-8
8. Du F, Wang G, Wang D, et al. Targeted next generation sequencing revealed a novel deletion-frameshift mutation of KCNH2 gene in a Chinese Han family with long QT syndrome: a case report and review of Chinese cases. Medicine. 2020;99(16):e19749. doi:10.1097/MD.0000000000019749
9. Motoi N, Marehiko U, Ryota E, et al. A novel KCNQ1 nonsense variant in the isoform-specific first exon causes both jervell and Lange-Nielsen syndrome 1 and long QT syndrome 1: a case report. BMC Med Genet. 2017;18(1):16.
10. Amin AS, Pinto YM, Wilde AA. Long QT syndrome: beyond the causal mutation. J Physiol. 2013;591(17):41254139. doi:10.1113/jphysiol.2013.254920
11. Hof T, Liu H, Sall L, et al. TRPM4 non-selective cation channel variants in long QT syndrome. BMC Med Genet. 2017;18(1):31. doi:10.1186/s12881-017-0397-4
12. Guinamard R, Bouvagnet P, Hof T, et al. TRPM4 in cardiac electrical activity. Cardiovasc Res. 2015;108(1):2130. doi:10.1093/cvr/cvv213
13. Hayashi K, Konno T, Fujino N, et al. Impact of updated diagnostic criteria for long QT syndrome on clinical detection of diseased patients: results from a study of patients carrying gene mutations. JACC Clin Electrophysiol. 2016;2(3):279287. doi:10.1016/j.jacep.2016.01.003
14. Neira V, Enriquez A, Simpson C, et al. Update on long QT syndrome. J Cardiovasc Electrophysiol. 2019;30(12):30683078. doi:10.1111/jce.14227
15. Tester DJ, Ackerman MJ. Genetics of long QT syndrome. Methodist Debakey Cardiovasc J. 2014;10(1):2933. doi:10.14797/mdcj-10-1-29
16. Betge S, Schulze-Bahr E, Fitzek C, et al. [Long QT syndrome causing grand mal epilepsy: case report, pedigree, therapeutic options, and review of the literature]. Nervenarzt. 2006;77(10):12101217. German. doi:10.1007/s00115-006-2118-7
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A Novel Mutation in the TRPM4 Gene | RRCC - Dove Medical Press
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The Science of Thinning Hair, and The Best Ways to Style it – Men’s Health Magazine Australia – Men’s Health
As with many medical issues, there can be many reasons why you have thinning hair. From stress, to hair-styling products, to your diet and your genes, its important to know the culprit behind your lacklustre locks.
But the one thing thats for sure it that youre not alone:an estimated 35 million men are suffering from hair loss worldwide.
For men, the main cause of hair loss or thinning is genetics, but not all conditions are caused by this. Other factors have been shown to disrupt growth when severe enough, such as diet, stress, hormonal imbalances and some medical treatments. These factors can affect the hair follicles causing them to go dormant.
Heres the thing: everyone loses 50-100 hair strands a day on average, but if youre experience unusual hair loss, understanding the different types and causes of this loss is important in seeking treatment from a doctor. There are many types of hair loss, with these being the most common:
Known as male-pattern baldness in men, this refers to the general thinning of hair growth. Terminal hair growth (normal hair) is converted to vellus hair (thin, short and unpigmented hairs).
Causes: Genetics, age and hormonal changes.
Sudden and unpredictable patchy hair loss. It can progress to total head hair loss (alopecia totalis) and total body loss (alopecia universalis).
Causes: An autoimmune disease causes the bodys immune system to attack hair follicles.
Large numbers of hair follicles are induced into their resting phase (telogen), resulting in increased hair shedding typically over 200 strands a day. This can be a short or long-term condition that develops two to three months after the triggering cause.
Causes: Systemic diseases, weight loss, drugs, illness, stress, iron deficiency and scalp inflammation.
Hair loss along the front and sides of the scalp caused by a persistent, prolonged or repetitive pulling force being applied to the hair.
Causes: Hair weaves, tight pony-tails and braiding techniques.
There are limits to how much you can do to reduce hair thinning and loss, but some approaches do help. Ensuring that you have a balanced diet with enough Vitamin D, Vitamin C & Iron is a great place to start but beware of going overboard with supplements as some of them such as Vitamin A could hinder growth.
Some poorly formulated shampoos can cause irritation to the scalp, so swapping to a milder alternative could help keep your scalp in good condition and a healthy scalp has been linked to hair growth. There is a compound called Minoxadil which is clinically proven to reduce certain types of hair loss. It does this by reducing the time that hair follicles are in their resting phase, telogen, and increasing the time spent growing, anagen.
Finally, it is also important to look after the hair that you are growing, including during your styling routine!
If you are experience thinning hair, there are certain hair styles that can give the appearance of a fuller head.
Consider hair styles with minimal parting, a fringe, or even try a combover thats swept to the side to disguise a receding hairline. Coloured hair sprays can also be an option to add shading to the scalp area.
During styling, its best to be gentler to your hair and scalp. While drying and styling hair, you should minimise excessive heat, and this is easiest with a controlled airflow and temperature. Be kinder to your scalp by using the Gentle air attachment (like the one on theDyson Supersonic hair dryer) as they use a gentle, cooler airflow while still drying hair fast.
Extreme heat can cause chemical changes to both the proteins and fats within your hair, this causes it to become weaker and more likely to break, which can reduce the amount of hair covering your scalp. Colouring and especially bleaching of hair can also cause weaken your hair, especially if done frequently.
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The Science of Thinning Hair, and The Best Ways to Style it - Men's Health Magazine Australia - Men's Health
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Why We Need Gender-Inclusive Terms for ‘Father’ and ‘Mother’ – VICE
A column about being a pregnant trans dad, and all the prejudices, healthcare challenges, personal dilemmas, and joys that come with making a family in 2021.
Merriam-Webster defines parent, primarily, as a person who is a father or mother or a person who has a child. Mother is defined as a female parent and father is a male parent. In my experience, this is exactly how most peopleparents and non-parents alikeuse these words. Thats how its meant to work: As any lexicographer will tell you, mainstream dictionaries are descriptive, not prescriptive. In the words of Simon Winchester, dictionaries tell of the language as it is, not as it should be.
We also have to father and to mother as verbs, which gets a bit more complicated. To father is synonymous with beget and sirethough Merriam-Webster notes, in a circular way, that these words are considered archaic, except in relation to domestic animals. To mother seems simpler on the face of it, maybe less overtly sexist? To mother is defined as to give birth to or to care for or protect like a mother. The first definition is the biologically essentialist one, but at least unlike with beget and sire, there are no caveats or domestic animals to be found.
Its subtle, but already we can see clear signs of a patriarchal system of society embedded in language. Is it less problematic to reduce female parenthood to biology nowadays? Surely, no feminist would argue this (except perhaps the trans-exclusionary kind). To me, and likely to any queer parent, the unqualified foregrounding of a biological role in relation to women seems like a less overt, yet much more insidious example of linguistic sexism.
Thankfully, where dictionaries might oversimplify by describing majorities first and foremost, our laws increasingly do not. With conspicuous exceptions like the UK, democratic states tend towards updating legislation to afford all parent-child relationships equal status and protection under the law, regardless of genetics or gender.
Ive written before about birth certificates, for example. In Canada, Australia, and many US states, gender-neutral birth certificates are available to all families, not just queer ones; in Canada and the US, three or more parents can be recognized on a birth certificate; and in the EU, the European Court of Human Rights recently ruled that all member states must recognize cis-queer parents on their childrens birth certificates.
The unavoidable, pragmatic, progressivehowever you see itconsequence of such laws, and, perhaps the ultimate one, is to deprivilege the traditional nuclear family structure, i.e., a married cis-hetero couple and their biological offspring. Usually, discussions on the topic center on LGBTQ+ familiesand we also tend to be most visible in campaigns to equalize outdated family law. But statistically speaking, cishet parents and their children will always make up the majority of non-traditional, non-nuclear families.
LGBTQ+ family rights, and the discussions they prompt about definitions, were never about special treatmentthey are about equality with other families. We needand need to campaign in a broad coalitionfor new systems, starting from the principle of what children, not their parents, need. This includes complete information about their origins and equal recognition of their parentage. Only by moving past our current legal and social focus on parental rightsor worse, some archaic, ideological notion of real familycan we hope to achieve true equality for all families.
How, for example, does legal motherhood and fatherhood work for the growing numbers of families, LGBTQ+ or not, created with the help of donor gametesthat is, with donated sperm or eggs?
Most of us are still very new to frank, informed conversations about using donor gametes to create family, even if weve actually done it. A few years ago, before conceiving my first child with the help of an open-ID sperm donor, I could be overheard saying, Its just like using donated blood or a kidney, right? Today, having exposed myself to the perspectives of many donor-conceived adults, innumerable donor-assisted families, and the staggering dysfunction of our birth registration system, I understand that all origin information is a persons fundamental birthright. And I believe LGBTQ+ family equality campaigners and campaigners for the rights of donor-conceived people could all benefit from closer alliance, despite being, in some ways, philosophically and linguistically at odds.
In donor-conceived online spaces, Ive noticed an almost fundamentalist approach to language. If its a coping strategyto allay donor-conceived adults completely righteous anger and sense of betrayalit makes sense. I cannot imagine many things more destabilizing than realizing youve been lied to in such a profound way by the people you trust more than anyone else, your parents. The urge to reclaim control and define things strictly according to historically-powerful systems of meaning, e.g., who is ones real or biological father/mother, seems reasonable on the face of it.
Enter the queers. Take my own situation: I am a trans man who became a dad with the help of donated sperm (literally donated, since its illegal to receive payment for gametes in the UK). With this sperm, I became pregnant and gave birth. I have always spoken openly to my kid about being donor-conceived, even before he was born. We have all the books. I will fully support my kid if he wants to contact the donor or his donor siblings in the future, or if he doesnt. Ive already looked into ways of making this possible sooner than him turning 18, which is when hell be able to access the contact details the UK regulator holds. Will I make mistakes? Of course. Am I my childs biological mother? No.
Every parent, as an integral part of becoming one, takes on a responsibility to be proactively honest with their kids about how they were created, from day zero. Also, humans are complicated and flawed, and, given its not their origins but those of their children in question, parents should not be the holders or gatekeepers of this information. A childs genetic origins, how they were conceived and birthed, and who their legal parents are can all be clearly and safely recorded for future reference by them.
But a progressive theory of identity, inclusive definitions of mother, father, parent, and my own queerness force me to stop short of referring to the person who donated the sperm that I used as my kids biological father. Yet this hesitation essentially breaks the rules of donor-conceived community etiquette, as I understand it. In short, it necessarily makes me as a parent selfish and in denial about my choices around family creation. It is seen as taking agency away, yet again, from previously deceived donor-conceived adults.
I hope those who take this position will at least hear me out. Its not the biological that gives me pause so much as the father. In every meaningful, modern sense, I am my childs father. I have this role in his life, it is legally true according to UK gender recognition law, and this is how I understand myself as a transgender man, as trans male. To me, any person who has a parental role and uses the word father for themselves is also a fatherno more, no less. Biology has nothing to do with it.
The yes, but biologically line of argument immediately veers toward the same biological essentialism used to oppress queer people, and trans people in particular, for the whole of human history. If you would not insist that trans men are biological women, and vice versa for trans womenif you recognize these as regressive, exclusionary, and dangerous opinions, as opposed to facts (not feelings!)the same must be true when we talk about family roles and relationships.
I want to clarify that Im not talking about people who meet and develop relationships with their donors. I fully accept that words like mother/father/mum/dad/parent come into play when actual human relationships form. I would never be threatened by the idea of my kid one day having two dads, or a mum and a dad if our donor is a trans woman. Thats exactly the meaning of these words that I see as, well, meaningful.
The conversation around donor siblings is a little different, largely because the idea of sibling carries infinitesimal social power relative to parent. I have seen donor-conceived adults object to the portmanteau dibling (donor sibling) as euphemistic, and yet to me, this is exactly the kind of pragmatic approach that keeps the language of family inclusive, progressive, and, ultimately, as useful as possible. Ones personal relationship to siblings resulting from donor connection is different, broadly speaking, from one's relationship to siblings who grew up as a family. Richer language helps us be clearer and more honest about that. It also de-essentializes and broadens how we understand family more generally.
My hope is that we hold firmly and compassionately to the social and political meanings of mother/father/parent and ditch the biologically essentialist ones completely. As we have begun to do with woman and man, we can let go of our need for strict categories and allow for a complexity that says: Language does not create us, we create it, and this, specifically, is no longer helpful. We no longer need both definitions, biological parent and social parent. Its too easy to subconsciously read that and as a versus, or for the question to follow: Which is more real more important? One automatically sounds like it carries more weight, right?
Is a woman less of a mother for not giving birth, or for using a donor egg? If the answer is no, and I believe wholeheartedly it is, then the best way to make this clear is to dispense with the dichotomous idea of biological mother altogetheralong with to mother meaning to give birth to. Likewise, do we think nonbinary parents are really the biological mother or father if their gametes are involved? If not, then lets not use those terms. If we continue using them for cishet parents, were still implying them for everyone. Or worse, we are drifting yet again towards a hierarchy of realness.
The social and political meanings of these words are the ones that actually matter. So lets use different words when we mean different things, and have zero tolerance for the archaic systems of oppressive power that caused us to settle for just one word, used in rank order, in the first place.
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Why We Need Gender-Inclusive Terms for 'Father' and 'Mother' - VICE
Recommendation and review posted by Bethany Smith
What Is Food Freedom? Getting Started, Weight Loss, and Tips – Healthline
Food freedom its a complex term, with definitions ranging from ditching diet culture and restrictive diets to attaining good health and food security through growing your own foods.
Its marketed as an approach to address eating disorders for some and as a way to promote intentional weight loss for others.
However, in the health and wellness space, its an emerging, revolutionary concept that challenges societal norms of dieting and the thin ideal.
It is championed by passionate health professionals and game-changers, such as Shana Spence (@thenutritiontea). Spence is a registered dietitian who takes a non-diet, weight-inclusive approach to health.
She uses her platform to redefine what health means distinct from the diet industrys often-unattainable standards.
Another powerful and passionate food freedom champion is Dr. Kera Nyemb-Diop (@black.nutritionist), who has created a space that emphasizes body respect, eating without guilt, and reclaiming your cultural food heritage as an integral part of your healthy lifestyle.
In this article, we explore food freedom, explain what intuitive eating and mindful eating are, and discuss what roles if any they may have in the pursuit of intentional weight loss.
The food freedom framework has various definitions and applications, including but not limited to (1, 2):
In other contexts, food freedom refers to ditching dieting culture and restrictive diets by giving yourself permission to enjoy all foods in moderation (unless allergies or medical needs prevent you from eating certain foods).
In that application of food freedom, practitioners see food as more than just fuel. They seek to build a positive and judgment-free relationship with all foods, where guilt is not considered an ingredient in the eating experience.
This view of food freedom encompasses intuitive eating and mindful eating, two philosophies that cultivate self-trust around food choices and reject unnecessary restrictions.
Intuitive eating and mindful eating are often used to support recovery from eating disorders such as anorexia nervosa and bulimia nervosa, chronic mental illnesses that negatively affect nutritional status and your relationship with food (3, 4, 5).
Overall, food freedom can help people overcome diet culture or introduce flexibility for intentional weight loss.
Because the varied and overlapping marketing of the term food freedom may lead to some confusion, context matters. This article will focus on food freedom as a non-diet approach to health and nutrition.
The term food freedom has various definitions, including ditching diet culture and cultivating self-trust around food choices. The food freedom approach has been used to support both eating disorder recovery and some intentional weight loss programs.
Food freedom as a therapeutic approach for eating disorder recovery grew out of the need for non-pharmaceutical treatments that emphasize behavioral changes, such as a positive body image and healthy eating attitudes (3, 6).
A 2017 study demonstrated that dieting accompanied by body dissatisfaction and the pursuit of thinness increases the risk of developing bulimia nervosa, binge eating disorder, and purging disorder (7).
Even dieting among inherently lean individuals increases their risk of developing anorexia nervosa (7).
The multibillion-dollar diet industry promotes the thin ideal with unhealthy weight management behaviors, potentially encouraging disordered eating patterns that can contribute to the development of eating disorders (7, 8).
Theres evidence that dieting doesnt help those who are seeking long-term weight loss, either.
Weight regain within 15 years is common among chronic dieters, and approximately 33% of dieters regain more weight than they initially lost (8).
Dieting restrictions contribute to disordered eating. Food freedom, on the other hand, seeks to combat this (5).
Food freedom as a mindfulness-based practice may address disordered eating, including emotional eating and binge eating disorder. It can also help you avoid eating in response to external cues, such as the sight or smell of foods, when youre not physically hungry (6, 9).
In particular, intuitive eating is associated with improved psychological well-being and physical health and fewer dietary restrictions (5, 10).
Food freedom arose from the need for behavior-change approaches emphasizing positive body image and healthy eating attitudes instead of dieting restrictions. It can support folks in recovery from disordered eating or clinical eating disorders.
Although these three terms are often used interchangeably, you may wonder whether they are essentially the same. There are minor distinctions among their presiding principles.
For instance, mindful eating is rooted in the Buddhist practice of mindfulness and living with awareness and intention (11, 12).
Its a meditative practice that is built on the mind-body connection and fosters a state of nonjudgmental awareness that engages your senses sight, smell, taste, and feel during a meal (11, 12).
Mindful eating is the art of being present while you eat.
Similarly, intuitive eating nurtures a mind-body connection, but its distinctively rooted in a weight-inclusive approach to health and serves as the core of the Health at Every Size paradigm (10).
Intuitive eating is guided by 10 principles, including respecting your body, rejecting diet culture, making peace with food, and honoring health through gentle nutrition.
Food freedom, however, isnt so well defined. It may represent true forms of intuitive eating or mindful eating, or it may attempt to bridge gaps between intentional weight loss, caloric restriction, and increased flexibility with food.
Despite these differences, there is a common thread among the three terms: They all seek to reduce unnecessary dietary restrictions and improve your relationship with food.
They aim to remove prospects of guilt, shame, and negative emotions associated with consuming forbidden or bad foods.
The terms food freedom, intuitive eating, and mindful eating may be used interchangeably, but there are differences among these practices. However, they all seek to reduce dieting restrictions and increase flexibility.
Food freedom, when used as a non-diet approach to health, seeks to liberate you from the thin ideal and diet culture, unsafe weight loss or weight management behaviors, and yo-yo dieting.
Whether you choose to adopt a meditative approach with mindful eating or work through the 10 principles of intuitive eating, freedom from restriction and judgment is possible.
Here are some tips:
Food freedom as a non-diet approach to nutrition includes tuning in to your internal cues of fullness and hunger, removing morality from foods, and focusing on health-promoting behaviors rather than the scale.
Intentional weight loss is the active attempt to change your body weight, with the goal of lowering the number on the scale.
Although studies show that intuitive eating is associated with weight loss and a lower body mass index (BMI), at its core, intuitive eating is not a weight loss method (10).
A true intuitive eating program would not advertise weight loss as an outcome, since some people may lose weight while others may gain or maintain weight.
Intuitive eating allows your body to find its happy weight, or biologically determined set point weight.
Likewise, the fundamental principles of mindful eating are not focused on weight loss though some weight loss programs have co-opted its messages of mindfulness (11).
Other programs work to bridge the gap by focusing on health-promoting habits while instituting small calorie deficits that promote slow-paced weight loss without completely avoiding pleasurable foods that might not be nutrient-dense or low in calories.
The principles of intuitive eating and mindful eating dont focus on intentional weight loss, although weight loss, gain, or maintenance may occur when you adopt them. Instead, they focus on allowing your body to reach its happy, natural weight.
Food freedom is a highly marketed term with various definitions, ranging from overcoming diet culture and restrictive diets to engaging in food sovereignty. Therefore, context matters.
As a non-diet approach to nutrition, food freedom includes tuning in to your internal cues of fullness and hunger, decoupling foods and morality, and focusing on health-promoting behaviors not just the scale.
At their core, intuitive eating and mindful eating principles dont focus on or promote intentional weight loss. Rather, they help you discover and engage in health-promoting habits that may lead to weight loss, gain, or maintenance.
These frameworks help people foster positive relationships with foods and their bodies that are built on self-trust and self-compassion rather than on the thin ideal.
See the rest here:
What Is Food Freedom? Getting Started, Weight Loss, and Tips - Healthline
Recommendation and review posted by Bethany Smith
Genetic testing | healthdirect
On this pageWhat is genetic testing?
Genetic testing can give you important information if you are planning a family or if you, or someone you care for, has a genetic disorder. But before you start, there are plenty of issues for you to think about, including where to turn if you choose to do a test.
You might think about genetic testing if:
Genetic tests all look for variations from what genes should normally look like. There are many variations found in genes, and only some of them are important.
The testing is only the first part. The important part is understanding what the test results mean. For that, you should talk to a doctor or genetic counsellor.
Most genetic tests are blood tests. It is also possible to do tests on a sample taken from the inside of your mouth (known as a buccal smear) or from your saliva. These are easy and safe.
If you're pregnant, prenatal testing may include a blood test, chorionic villus sampling (CVS) or amniocentesis. Visit Pregnancy, Birth and Baby website for more information on prenatal screening.
It's also possible to order tests online without seeing a doctor. This is not a good idea. The National Health and Medical Research Council warns people to be cautious of this.
The cost varies enormously. Before you start, ask your doctor or genetic counsellor how much the tests cost and whether or not you're covered at all by Medicare. Tests ordered online are not covered by Medicare.
The main benefit is knowledge: if you have a genetic test, you can get advice about what that test means. If you have a condition that might have a genetic basis, you will understand it more clearly.
If you're having a baby, it helps you plan how to handle the pregnancy and birth and the life that follows. It can also help if you're wondering about having a termination.
For some people, testing can get rid of some of the uncertainty around their health, for example, fears they may be a genetic carrier of a particular disorder.
If you feel well but are worried about having a condition that runs in your family, genetic testing can prompt you into action to reduce your chances of getting ill, whether that's through diet and exercise or through screening for a certain condition.
Some people face discrimination at work if they are known to have a genetic mutation.
If you have any genetic tests, you will probably need to declare them for any life insurance or income protection. If the tests identify a gene variant that increases your risk of disease, that might make it hard or impossible for you to get insurance.
Some of the companies that you can order tests from online are based overseas. They might be less careful about privacy than Australian companies. Some overseas companies sell information about genetic tests to others. Ordering tests from these companies has a risk that your private information could become available to others.
Genetic testing is not always accurate. If you find that you have a variation to a gene, that gives a clue. But it doesn't tell you how much you will affected by the abnormal gene. Some people will be severely affected by an abnormal gene, while another will not be affected too much at all.
Also, testing for genes is complex and it can be hard to tell what minor changes in a gene mean. It is likely that some of the testing done by companies offering their services online will be inaccurate.
That decision is entirely up to you. There is no right or wrong answer. Talk to family and friends you trust. You can talk to your doctor or to a genetic counsellor. Take your time.
It's best to see your doctor or a genetic counsellor if you're thinking about having a test. That way, you can talk through the implications of having the test. And if you go ahead with it, you can get some good advice about what the results mean.
If you have a genetic test, you might find out things you wanted to find out, but you might also find out things you didn't want to know. Your relatives might want to know everything, or they might want to know nothing.
The best way is to talk to them before you have any test, so you understand their point of view.
Visit our 'Guide to genetics disorders' to learn more about genetic disorders and where to go for help.
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Genetic testing | healthdirect
Recommendation and review posted by Bethany Smith