Stem cells are often in the news. These days its usually about some advance in research. Sometimes the controversy about using embryonic stem cells resurfaces. Despite all the coverage (pro or con) stem cells are not well understood. What are they and why are they important?

In more ways than one, its the potential of stem cells that makes them important. At the moment most of the work with stem cells is still in the laboratory; but thats changing. Within the next few years stem cells, in one form or another, will be at work in medical applications such as repairing a damaged pancreas or a heart. In fact, stem cells will be used to repair or even re-grow tissues all over the body skin, liver, lungs, bone marrow. The production of stem cells, their delivery, and procedures for using them will become the basis of an industry. In the not too distant future stem cells, or the knowledge we gain from working with them, will be used in sophisticated repair of the brain and as part of the development of replacement organs. The potential is enormous.

What are stem cells?

Stem cells are found in most multicellular creatures and come in different varieties; all have an important ability: They can fully reproduce themselves almost indefinitely. For example, in mammals like human beings, blood stem cells (hematopoietic stem cells) are active all our lives in the marrow of bones, where they continually produce the many different kinds of blood cells. Therein is another key property for most stem cells; they can become other kinds of cells. The word for this process is differentiate; blood stem cells can differentiate into red blood cells, white blood cells, blood platelets and so forth. The ability to produce different kinds of cells is why stem cells may be used, for example, to repair or replace damaged heart cells something mature heart cells cannot do on their own.

Stem cell jargon

When you read about stem cells, there are a number of words that jump out jargon, yes, but still descriptive. Stem cells are classified by their potency, that is, what other kinds of cells they can become, or put another way, their ability to differentiate into other cells. There is a rank order from more to less potent:

Totipotent sometimes also called omnipotent stem cells can construct a complete and viable organism. In short, they are the same as a cell created by the fusion of the egg and a sperm (an embryonic cell). Totipotent cells can become any type of cell.

Pluripotent stem cells are derived from totipotent cells and are nearly as versatile. They can become any type of cell, except embryonic.

Multipotent stem cells can become a wide variety of cells, but only those of a close family, for example blood stem cells (hematopoietic cells) can become any of the blood cells, but not other kinds of cells.

Oligopotent stem cells are limited to becoming specific types of cells, such as endoderm, ectoderm, and mesoderm.

Unipotent stem cells can only produce cells of their own type, for example skin cells. They can renew themselves (replicate indefinitely), which distinguishes them from non-stem cells.

To a certain extent the potency of a stem cell relates to its usefulness. In one view of an ideal (lab) world, only totipotent stem cells would be used because they can become any other kind of cell. The real world (lab or otherwise) doesnt work that way. For one thing, stem cells of lesser versatility than totipotent cells are valuable for use in specific applications. Even unipotent stem cells, lowest on the potency poll, are arguably better suited for some targeted uses than more generic stem cells. Most importantly, for many uses, especially for medical purposes, pluripotent stem cells are extremely versatile and less controversial.

Avoiding embryonic stem cells

The true totipotent stem cell is a fertilized egg one embryonic cell. To obtain it means detecting and collecting the cell shortly after fertilization and before it begins to divide. Collecting embryonic stem cells one at a time is very difficult and very expensive. Also, in some parts of the world, using embryonic stem cells is highly controversial, usually on religious grounds. Collecting embryonic stem cells can be considered abortion, since the procedure means the cell(s) will not become an embryo. The label abortion is also applied to collecting embryonic stem cells (by gastrulation) shortly after the first fertilized cell begins to divide. These cells, obviously more numerous, are pluripotent and have been the mainstay of stem cell research.

The history of opposition to the use of embryonic stem cells goes back to the 1990s, when stem cell research was in its own infancy. At that time the only source of viable laboratory stem cells was from in vitro living donors. Most of these were harvested from fertilization clinics. They were so difficult to acquire that only a few stem cell lines (painstakingly cultivated generations of embryonic stem cells) were available. Even those were controversial. The United States banned the taking of embryonic stem cells except for 23 grandfathered lines. (This ban was lifted in 2009.)

The controversy over embryonic stem cells can be avoided primarily in two ways. One way is to use adult stem cells. The word adult is a bit misleading since the cells may be derived from fetuses, newborns, and children, which is why theyre sometimes called somatic stem cells. It means that these stem cells come from relatively mature tissue, cells that are already differentiated to a certain degree. Thats why adult stem cells are almost always classified as multipotent, oligopotent, or unipotent. The other way is to transform adult stem cells into pluripotent stem cells. Many approaches to this transformation are being explored in labs all over the world. Some approaches are derived from fetal/newborn substances such as amniotic fluid and placental or umbilical tissue. Other approaches use mature (differentiated) stem cells, such as those from skin, and genetically modify them until they become pluripotent. Such cells are called induced pluripotent stem cells, often abbreviated as iPSC.

At the moment, it is not possible to say which approaches to stem cell production and application will be the most effective. Even some that seem unlikely (stem cells from skin cells?) may turn out to be the most economical and useful. Still, this is where the payoff for stem cell research lies both in terms of scientific knowledge and in profits for medical applications. Consequently the amount of research work in progress is substantial, and often competitive.

Stem Cell Tourism

Because experimental medical techniques and human desperation can add up to big money, there is a developing market for stem cell applications for a variety of medical disorders. Unfortunately, at least for now, with the exception of blood cell transplants and skin cell treatments, most of these applications are either fraudulent or based on shaky experimental results. In general, most stem cell treatments are at best unethical and often illegal; however, their status around the world is a patchwork quilt of laws and regulations (or their absence). It is a near ideal situation for scam artists to lure desperate people into traveling long distances for stem cell treatment that is illegal in their own country. Hence the name: stem cell tourism.

Tracking the Impact of Stem Cell Research

In relative terms, stem cell research is just getting started. Researchers have been at it since the 1950s; but one of the most important discoveries so far induced pluripotent stem cells dates back to only 2006. This means that stem cells are: a. Not yet well understood and b. Their use in medicine is largely experimental and tentative. Heres a useful listing of what the National Institute of Health (U.S. NIH) considers some of the major open questions about adult stem cells:

How many kinds of adult stem cells exist, and in which tissues do they exist? How do adult stem cells evolve during development and how are they maintained in the adult? Are they leftover embryonic stem cells, or do they arise in some other way? Why do stem cells remain in an undifferentiated state when all the cells around them have differentiated? What are the characteristics of their niche that controls their behavior? Do adult stem cells have the capacity to transdifferentiate, and is it possible to control this process to improve its reliability and efficiency? If the beneficial effect of adult stem cell transplantation is a trophic effect, what are the mechanisms? Is donor cell-recipient cell contact required, secretion of factors by the donor cell, or both? What are the factors that control adult stem cell proliferation and differentiation? What are the factors that stimulate stem cells to relocate to sites of injury or damage, and how can this process be enhanced for better healing? [Source: U.S. National Institute of Health]

SciTechStory Impact Area: Stem Cells

Theres not much debate on the importance of stem cell research. It has already had major impact on our understanding of cell biology, and it will provide more. It is just beginning to have an impact on medicine, with much more to come. In fact, news about stem cell research already occurs once or twice a week (on average) that pace is likely to increase. As a matter of keeping up, its necessary to attempt sorting lab work from practical application, which is to say sorting promise from delivery. Even at that it will be difficult to select which stem cell stories are significant.

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Stem Cells - SciTechStory

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February 8, 2017 Blood stem cells from patients with Diamond-Blackfan anemia dont mature properly (right two columns). Credit: Doulatov et al., Science Translational Medicine (2017)

Researchers at Boston Children's Hospital's Stem Cell Research Program were able, for the first time, to use patients' own cells to create cells similar to those in bone marrow, and then use them to identify potential treatments for a blood disorder. The work was published today by Science Translational Medicine.

The team derived the so-called blood progenitor cells from two patients with Diamond Blackfan anemia (DBA), a rare, severe blood disorder in which the bone marrow cannot make enough oxygen-carrying red blood cells. The researchers first converted some of the patients' skin cells into induced pluripotent stem (iPS) cells. They then got the iPS cells to make blood progenitor cells, which they loaded into a high-throughput drug screening system. Testing a library of 1,440 chemicals, the team found several that showed promise in a dish. One compound, SMER28, was able to get live mice and zebrafish to start churning out red blood cells.

The study marks an important advance in the stem cell field. iPS cells, theoretically capable of making virtually any cell type, were first created in the lab in 2006 from skin cells treated with genetic reprogramming factors. Specialized cells generated by iPS cells have been used to look for drugs for a variety of diseasesexcept for blood disorders, because of technical problems in getting iPS cells to make blood cells.

"iPS cells have been hard to instruct when it comes to making blood," says Sergei Doulatov, PhD, co-first author on the paper with Linda Vo and Elizabeth Macari, PhD. "This is the first time iPS cells have been used to identify a drug to treat a blood disorder."

DBA currently is treated with steroids, but these drugs help only about half of patients, and some of them eventually stop responding. When steroids fail, patients must receive lifelong blood transfusions and quality of life for many patients is poor. The researchers believe SMER28 or a similar compound might offer another option.

"It is very satisfying as physician scientists to find new potential treatments for rare blood diseases such as Diamond Blackfan anemia," says Leonard Zon, MD, director of Boston Children's Stem Cell Research Program and co-corresponding author on the paper with George Q. Daley, MD, PhD. "This work illustrates a wonderful triumph," says Daley, associate director of the Stem Cell Research Program and also dean of Harvard Medical School.

Making red blood cells

As in DBA itself, the patient-derived blood progenitor cells, studied in a dish, failed to generate the precursors of red blood cells, known as erythroid cells. The same was true when the cells were transplanted into mice. But the chemical screen got several "hits": in wells loaded with these chemicals, erythroid cells began appearing.

Because of its especially strong effect, SMER28 was put through additional testing. When used to treat the marrow in zebrafish and mouse models of DBA, the animals made erythroid progenitor cells that in turn made red blood cells, reversing or stabilizing anemia. The same was true in cells from DBA patients transplanted into mice. The higher the dose of SMER28, the more red blood cells were produced, and no ill effects were found. (Formal toxicity studies have not yet been conducted.)

Circumventing a roadblock

Previous researchers have tried for years to isolate blood stem cells from patients. They have sometimes succeeded, but the cells are very rare and cannot create enough copies of themselves to be useful for research. Attempts to get iPS cells to make blood stem cells have also failed.

The Boston Children's researchers were able to circumvent these problems by instead transforming iPS cells into blood progenitor cells using a combination of five reprogramming factors. Blood progenitor cells share many properties with blood stem cells and are readily multiplied in a dish.

"Drug screens are usually done in duplicate, in tens of thousands of wells, so you need a lot of cells," says Doulatov, who now heads a lab at the University of Washington. "Although blood progenitor cells aren't bona fide stem cells, they are multipotent and they made red cells just fine."

SMER28 has been tested preclinically for some neurodegenerative diseases. It activates a so-called autophagy pathway that recycles damaged cellular components. In DBA, SMER28 appears to turn on autophagy in erythroid progenitors. Doulatov plans to further explore how this interferes with red blood cell production.

Explore further: Scientists find that persistent infections in mice exhaust progenitors of all blood cells

More information: "Drug discovery for Diamond-Blackfan anemia using reprogrammed hematopoietic progenitors," Science Translational Medicine stm.sciencemag.org/lookup/doi/10.1126/scitranslmed.aah5645

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Posted on Feb. 8, 2017, 6 a.m. in Gene Therapy Sensory

Harvard Medical School scientists have perfected a form of gene therapy that has enabled genetically deaf mice to hear sounds as quiet as a whisper.

Harvard Medical School scientists have perfected gene therapy to the point that it can restore hearing. Their research and experiments have shown that the hearing of genetically deaf mice can be restored to the point that they hear noises at 25 decibels. This decibel level is equivalent to that of a soft whisper.

The Nuances of Gene Therapy for Improved Hearing

Harvard's gene therapy researchers state the most important aspect of their gene therapy breakthrough is a vector they created known as "Anc80". This vector brings a therapeutic gene to the cells within the cochlea's outer ear that are quite difficult to access. These outer hair cells boost sound, empowering inner hair cells to transmit a much more powerful communication to the brain. Gwenalle Gloc of Boston Children's Hospital's Department of Otolaryngology and F.M. Kirby Neurobiology Center, states the new system functions quite well by rescuing vestibular and auditory function to a degree that was not previously achieved in medical history. Research Details

Harvard's research team includes scientists employed by Massachusetts Eye and Ear. The group tested its gene therapy technique on mice with Usher Syndrome. This is a genetic disease that harms hearing as well as vision. Humans who are saddled with this disease are afflicted with a gene mutation that makes the protein harmonin ineffective. As a result, the hair cells responsible for accepting auditory signals and transmitting them to the brain are rendered useless.

The research team tapped into the power of its new vector to transmit an improved version of the gene, referred to as Ush1c, directly into the ear. It didn't take long for the ear's outer and inner hair cells to generate effective harmonin. Subsequent hearing tests conducted on mice proved that animals born deaf could hear. Some of these mice could even pick up on uber-soft auditory signals just like their normal peers.

The Magic of Gene Therapy

The scientific community is abuzz over gene therapy. Some believe gene therapy will ultimately prove to be the cure for deafness. It was only two years ago when scientists and investigators from Harvard and the University of Michigan's Hearing Research Institute found that the hearing-associated protein, NT3, can be stimulated through gene therapy. Additional approaches are geared toward stimulating the regeneration of hair cells within the ear. As an example, Harvard researchers have found that drugs referred to as Notch inhibitors can spur existing ear cells to transition into hair cells that improve hearing in mice.

The Harvard team reports its latest success with gene therapy made use of a similar technique that heightened hearing in 2015. However, these researchers now believe their newly generated vector will restore an even higher level of auditory ability. They also noted that the Ush1c gene applied to deaf mice served to heighten their balance. Mice with Usher Syndrome typically suffer from such poor balance. The Future of Gene Therapy

The future looks quite bright for those who suffer from hearing deficiencies. The research described above is fantastic news for those who suffer from hearing loss. It is possible that gene therapy will eventually supplant cochlear implants that are currently used to improve hearing in young patients. Though Cochlear implants have served patients quite well, there is still room for improvement.

Patients would like to hear an extended range of frequencies and the direction of a sound's source. They would also like to be able to differentiate between the auditory nuances of background noise, voices, music etc. The added benefit of heightened physical balance will serve to enhance Usher Syndrome patients' balance and mobility.

More:
Gene Therapy to Restore Hearing | Worldhealth.net Anti-Aging News - Anti Aging News

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Medical News Today

Gene therapy: Deaf to hearing a whisper BBC News Deaf mice have been able to hear a tiny whisper after being given a "landmark" gene therapy by US scientists. They say restoring ... The researchers developed a synthetic virus that was able to "infect" the ear with the correct instructions for ... Groundbreaking gene therapy restores hearing, balanceMedical News Today Gene therapy helps deaf mice hear sounds as soft as whispersFierceBiotech Gene therapy restores hearing in deaf mice: Experts say the technique could be used in humans in THREE YEARSDaily Mail ResearchGate (blog) -Wired.co.uk -BioNews all 19 news articles »

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Gene therapy: Deaf to hearing a whisper - BBC News

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A physician at the Parkinsons Institute and Clinical Center in Sunnyvale has been awarded $1.9 million from the California Institute for Regenerative Medicine to advance potential therapies for Parkinsons disease.

Birgitt Schuele MD, director of gene discovery and stem cell modeling at the center, will use the funding to study the effects of lowering levels of a key protein linked to Parkinsons disease as a possible gene therapy approach to haltthe degeneration of nerve cells in patients brains.

Other researchers to receive similar funding are from Stanford University, UC-San Francisco, and UCLA.

We are proud to be recognized by the CIRM among 11 projects from leading centers such as Stanford and UCs for our work, Schuele said. Although we are a small organization, we are at the forefront of scientific developments toward novel and innovative treatments for Parkinsons disease.

Parkinsons disease is a progressive, chronic disorder of the central nervous system that affects the motor system and can impair movement, balance and coordination. Common symptoms include tremors and difficulty moving. While the causes are unknown, genetics and environmental exposures are thought to be contributing factors.

According to the institute, more than 1.5 million Americans are living with the disease, and although most patients diagnosed are over 50, some experience onset much earlier. The Sunnyvale institute does research as well as provides patient care.

According to Schuele, characteristic features in a Parkinsons brain are clumps containing a protein called alpha-synuclein. She says studies have shown that too much of the protein can kill nerve cells. In addition, genetic research has discovered families with early and aggressive Parkinsons have the genetic makeup that causes overproduction of alpha-synuclein.

The funding will be used in additional research to see if the gene that creates the protein can be removed, inhibiting production of it in the brain and possibly stopping the progression of the disease.

In order to study this gene therapy, Schuele says she and institute researchers are transforming skin cells donated from Parkinsons patients into pluripotent cells, which then become neurons that can be used to make an artificial brain model. Using the model, Schuele says she will be able to manipulate the gene producing the potentially harmful protein.

We hypothesize that lowering alpha-synuclein to normal physiological levels should shield the nerve cells and slow down the neurodegenerative disease process, Schuele says.

The project is being done in collaboration with the University of Lund in Sweden and Dr. Deniz Kirik, who will test the gene therapy in pre-clinical models in rodents with the hope of preparing for clinical trials within three years.

Originally posted here:
Sunnyvale: Parkinson's institute awarded $1.9 million for research - Milpitas Post

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Small sheets of healthy skin are being grown from scratch at a Stanford University lab, proof that gene therapy can help heal a rare disease that causes great human suffering.

The precious skin represents growing hope for patients who suffer from the incurable blistering disease epidermolysis bullosa and acceleration of the once-beleaguered field of gene therapy, which strives to cure disease by inserting missing genes into sick cells.

It is pink and healthy. Its tougher. It doesnt blister, said patient and research volunteer Monique Roeder, 33, of Cedar City, Utah, who has received grafts of corrected skin cells, each about the size of an iPhone 5, to cover wounds on her arms.

More than 10,000 human diseases are caused by a single gene defect, and epidermolysis bullosa is among the most devastating. Patients lack a critical protein that binds the layers of skin together. Without this protein, the skin tears apart, causing severe pain, infection, disfigurement and in many cases, early death from an aggressive form of skin cancer.

The corrected skin is part of a pipeline of potential gene therapies at Stanfords new Center for Definitive and Curative Medicine, announced last week.

The center, a new joint initiative of Stanford Healthcare, Stanford Childrens Health and the Stanford School of Medicine, is designed to accelerate cellular therapies at the universitys state-of-the-art manufacturing facility on Palo Altos California Avenue. Simultaneously, it is aiming to bring cures to patients faster than before and boost the financial value of Stanfords discoveries before theyre licensed out to biotech companies.

With trials such as these, we are entering a new era in medicine, said Dr. Lloyd B. Minor, dean of the Stanford University School of Medicine.

Gene therapy was dealt a major setback in 1999 when Jesse Gelsinger, an Arizona teenager with a genetic liver disease, had a fatal reaction to the virus that scientists had used to insert a corrective gene.

But current trials are safer, more precise and build on better basic understanding. Stanford is also using gene therapy to target other diseases, such as sickle cell anemia and beta thalassemia, a blood disorder that reduces the production of hemoglobin.

There are several diseases that are miserable and worthy of gene therapy approaches, said associate professor of dermatology Dr. Jean Tang, who co-led the trial with Dr. Peter Marinkovich. But epidermolysis bullosa, she said, is one of the worst of the worst.

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It took nearly 20 years for Stanford researchers to bring this gene therapy to Roeder and her fellow patients.

It is very satisfying to be able to finally give patients something that can help them, said Marinkovich. In some cases, wounds that had not healed for five years were successfully healed with the gene therapy.

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Gene therapy's latest benefit: New skin - Daily Democrat

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Gene Therapy for Heart Disease Wins Fast-Track Status | P&T ... P&T Community The FDA has granted a fast-track designation for a phase 3 study of Generx (Ad5FGF-4, Angionetics Inc.) cardiovascular angiogenic gene therapy as a one-time ... and more »

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Gene Therapy for Heart Disease Wins Fast-Track Status | P&T ... - P&T Community

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A study group from the Mayo Clinic found that thyroid hormone treatment of subclinical hypothyroidism during pregnancy was associated with a lower risk of pregnancy loss, but a higher risk for adverse pregnancy outcomes such as premature birth and gestational diabetes.

Subclinical hypothyroidism (hypothyroidism proven by laboratory results without clinical symptoms) affects around 15% of pregnant women. The recent regulatory changes in laboratory TSH (thyroid stimulating hormone an indicator of low thyroid hormone levels) threshold may have resulted in an overdiagnosis and overtreatment of hypothyroidism. A lower thyroid hormone level during pregnancy has been associated with adverse pregnancy outcomes such as pregnancy loss, placental abruption, premature rupture of membranes, and neonatal death.

An article was recently published in the British Medical Journal that analyzed data from the OptumLabs Data Warehouse database to assess the prevalence, effectiveness, and safety of thyroid hormone treatment of subclinical hypothyroidism among pregnant women. Researchers included 5405 pregnant women in the study with TSH 2.5-10mIU/L between 2010 and 2014. 15.6% received thyroid hormone treatment and the percentage of treated women increased each year. Comparing the treated and untreated groups, treated patients had a 38% lower risk of pregnancy loss, but higher odds of preterm delivery, gestational diabetes, and pre-eclampsia. However, the risk of pregnancy loss was only lower in treated women with higher TSH levels (meaning lower thyroid hormone levels) but not in women with lower TSH. Furthermore, the risk of gestational hypertension was higher among treated than untreated women with lower TSH levels.

In conclusion, it seems that thyroid hormone therapy of subclinical hypothyroidism lowers the risk of pregnancy loss, especially in patients with higher TSH levels, but is associated with a higher risk of adverse pregnancy outcomes. Further studies are needed to assess the safety of thyroid hormone treatment in pregnancy.

Written By: Dr. Fanni R. Eros

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Thyroid Hormone Therapy for Subclinical Hypothyroidism in Pregnancy - Medical News Bulletin

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A new video campaign is calling on the Alberta government to provide more funding to help transgender youth.

The only specialized, multi-disciplinary clinic in the province catering to transgender youth is at the Alberta Children's Hospital in Calgary and is only open one half-day each month as part of a pilot project.

The waiting list for the clinicis up to three years.

"We're hearing a lot of kids stuck in that long wait, and what we're hearing a lot of is desperation," said Amelia Newbert, who runs the Skipping Stone Foundation, the organization behind the video campaign.

The Metta Clinic at the children's hospital provides everything from mental health and psychiatric support, to hormone therapy and preparation for some surgeries for youth aged seven to 20.

Newbert, who is trans,says timing is key with kids.

"The reality of being forced to be exposed to a puberty that doesn't conform to your gender identity is profoundly damaging," she said.

That damagecan lead to an increase in problems such as self-harm, addiction and suicide, according to Newbert.

In thefirst video of the Skipping Stone campaign,Ace Peace, a 16-year-oldtransgenderboy, describes the desperation he felt watching his body change while he waited nine months to get into the clinic.

"When I came out as transgender everyone supported me. I was supported by family, friends and school. But it was still hell for me," he says.

"I was watching my body change in ways I didn't want to see it change. I felt like it was betraying me."

Speaking with the Calgary Eyeopeneron Tuesday morning,Peace said he was struggling at school andthe clinic provided much needed emotional support.

"They knew what to do, they knew what was going on, they knew the emotional impact that it had on me so they tried to get me on hormones as fast as they could," he said.

Pam Krause, whoruns the Calgary Sexual Health Centre, agrees it's important to offer support early.

"If you have to wait three years for something, for something that you know is your truth, Ithink that causes all kinds of difficulties for people. And the system is paying later on when people are having mental health problems," she said.

Alberta Health Services told CBC News that it continues to examine the pilot project, launched in 2014.

"AHS is currently gathering evidence and exploring best practices to determine the best approach to providing services to youth struggling with gender dysphoria," said an email fromJulie Kerr, the health authority'ssenior operating officer foraddictions andmental health in theCalgary zone.

"Any decisions on whether to expand the clinic will be evidence-based."

Alberta's health minister, Sarah Hoffman, said she understands the long delays are frustrating.

"While there are a number of health services that can support transgender youth in different parts of the province, having a designated service like the Metta Clinic has proven invaluable for many patients and families," she said by email.

Kerr said youth struggling with gender identity can find other programs and services in Alberta, including community mental health programs, crisis mental health, family physicians, edrocrinologyand psychiatry services.

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Transgender youth in Alberta need more than 1 part-time clinic, says new campaign - CBC.ca

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NEW ALBANY Carla Copas, APN, is announced the grand opening of Finding Her Health, a new womens health care clinic located at 2708 Paoli Pike, Suite I, New Albany. A ribbon cutting ceremony will be held Feb. 8, at 10 a.m.

Copas specializes in Bio-Identical Hormone Replacement and has been prescribing this type of treatment to her patients with great success for 15 years. Formerly with OBGYN Associates of Southern Indiana, she is opening Finding Her Health to more fully utilize her skill and passion for a personalized approach to healthcare.

As an advanced practice registered nurse, I treat the whole person, not just the diagnosis or disease, Copas said in a news release. Unlike other healthcare offices where there are sometimes dozens of people in the waiting room and the providers see 30-40 patients a day that is one every 5-10 minutes, I take the time with each patient for education and understanding. Most of my patients have a time slot from 30 minutes to one hour. I provide same-day appointments and am available to my patients via secured email and answer within 24 hours.

Services available at Finding Her Health will include gynecology, STI testing and treatment, birth control options, hormone balance, well woman exams, treatment for UTIs, vaginal problems, painful intercourse, decreased libido, irregular menses, fertility, peri-menopause and menopause balance, prevention and other issues specific to womens health.

Im excited about this new venture. Opening this clinic will provide a unique opportunity for me to help women in all walks of life, Copas said. At Finding Her Health, no matter what a womans age or medical situation, I want to help guide my patients to feeling the best they have ever felt.

The public is invited to attend the ribbon cutting ceremony to meet Copas and tour the new office. For more information, visit http://www.findingherhealth.com or Facebook/findingherhealth.

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Public invited to women's clinic opening in New Albany - Evening News and Tribune

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Endocrinology Advisor

Risk of Pregnancy Loss in Subclinical Hypothyroidism Endocrinology Advisor Rozalina G McCoy, MD, from the Mayo Clinic in Rochester, Minnesota, and colleagues conducted the first national study to evaluate the effectiveness and safety of thyroid hormones for pregnant women with subclinical hypothyroidism. The study included ...

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Risk of Pregnancy Loss in Subclinical Hypothyroidism - Endocrinology Advisor

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A new revolutionary stem cell technique is being used to treat those suffering from damaged muscles without the cancer risk that was previously present. This was the first time that researchers had successfully implanted synthetic stem cardiac cells that managed to repair the muscle that a previous heart attack has weakened. Cancer was previously a risk with traditional stem cell therapy as scientists were unable to stop formertumors as the cells continued to replicate.

This procedure is mostly performed on those suffering from blood or bone marrow cancers such as leukemia. But, researchers are also working on developing effective stem cell treatments for those diagnosed with neurodegenerative diseases such as Parkinsons and heart disease too.

Synthetic stem cells are very handy because unlike natural stem cells, theyre easy to preserve and can be adapted to be used in various parts of the body. Ke Cheng, associate professor of molecular biomedical sciences at North Carolina State University, said, We are hoping that this may be the first step towards a truly off-the-shelf cell product that would enable people to receive beneficial stem cell therapies when theyre needed, without costly delays.

[The study can be found here.]

The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion, and analysis. Read full, original post:Pioneering Stem Cell Technique Promise Muscle Regeneration Without Cancer Risk

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Regrowing heart muscles without cancer risk, using synthetic stem cells - Genetic Literacy Project

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By filling out a form, and swabbing his mouth, Harlee O'Watch could save a life.

"To find a match, because the list of donors is so low, is really unlikely," said the 22-year-old.

O'Watch is one of four young adults from Carry the Kettle First Nation who registered with the OneMatch program, which connects donors with people in need of bone marrow or stem cell transplants.

A problem for the 14 indigenous people currently waiting for a match is that, out of the 17,000 people on the Canadian registry, fewer than one per cent are indigenous.

"It doesn't give me much hope if I ever get sick and need a blood transfusion or bone marrow transplant, said OWatch.

It doesn't give me much hope because, if there's no potential matches, I'm going to die, bottom line, and I don't want to die."

Robyn Henwood works for Canadian Blood Services, which runs OneMatch. She covers Alberta to Northern Ontario and the Northwest Territories, including the Prairies, and visited Carry the Kettle to recruit. A match requires a genetic twin and indigenous people are only in Canada.

"It does get more complicated [with] these different ethnic backgrounds. . . even within First Nations that get brought into it, said Henwood.

The chances of finding a match becomes that much more difficult."

This means someone who is Cree cannot donate to someone who is Mohawk, she said.

In the past year, Canadian Blood Services has visited less than 12 reserves to help find matches for indigenous people. Carry the Kettle is Henwoods third community.

"We have been leaving messages and voicemails, not getting a lot of response back, she said.

I'm hoping a new technique will work. Things like this, this is so important to spread our message."

According to Indigenous and Northern Affairs Canada, more than 50 per cent of indigenous people live in urban centres. And yet, Henwood says finding indigenous donors in cities is also a struggle.

"Trying to get someone to sign up and commit for the next 30 to 40 years, to potentially save a stranger's life is not an easy thing to do," she said.

Henwood says informing indigenous people about one match will empower more to donate. Until then, the chance of survival for those waiting on the registry is low.

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Program seeks to boost bone marrow, stem cell donations from indigenous people - CTV News

Recommendation and review posted by sam

Its been a long and difficult road for West Island resident Kevin Butterfill and his Fiance Natasha Camacho-Gomes.

Kevin had testicular cancer in Oct. 2015, he did chemo, he then had the tumor removed and he was in remission, Gomes said.

Its what happened in July 2016 that is causing a lot of grief for Butterfills loved ones.

He proposed to Camacho-Gomes, his girlfriend since 2009, but a week later more health complications arose.

He was diagnosed with myelodysplastic syndrome (MDS) which is a bone marrow disorder.

And to make matters even worse, Butterfill found out the MDS transformed into acute myeloid leukemia in January.

The past two years have been a roller coaster ride for Kevins mother, Heather Butterfill.

Were staying strong, Heather Butterfill said. Im very faithful that hes going to come out of this stronger than ever.

On Saturday, family and friendsgathered at a Provigo grocery store in Kirkland, encouraging those who know Kevin to sign-up to the bone marrow registry.

READ MORE:Venclexta gets accelerated approval to treat leukemia

Butterfill will need a bone marrow stem cell transplant to recover from the leukemia.

However, according to Hema-Qubec stem cell registry manager Susie Joron, the odds of the perfect match being a friend are slim.

Theres 60,000 transplants every year worldwide, Joron said. The chances of having a friend or a neighbour being matched to that one person that we know is very unlikely.

Gomes said the aim of the event is raise awareness about the stem cell donor registry.

A lot of people were interested in joining and trying to see if they were Kevins match, Camacho-Gomes said.

But a really important thing [to note] is that when you join the stem cell registry youre tested against everyone, so you have the potential to save anyones life.

Regardless of who ends up being Butterfills perfect match, friends like Jonathan Coleman are still hoping they can help.

To hear something like this happen to somebody like that, a good-hearted kid, it sucks and sad to hear, Coleman said. You want do anything you can to help him out.

READ MORE:Id do it again in a heartbeat: debunking myths around stem cell donation as #MenGiveLife kicks off

While Butterfill awaits news of a bone marrow donor for his stem cell transplant, Gomes will hope for a perfect match and move on to planning their wedding.

They set a tentative date for Victoria Day 2018.

For more information on stem cell donation and the registry, visit the Hma-Quebec website.

2017Global News, a division of Corus Entertainment Inc.

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Quebec family hopes to raise awareness for patients in need with stem cell registry drive - Globalnews.ca

Recommendation and review posted by Bethany Smith

The 2017 Australian of the Year award went to Professor Alan Mackay-Sim for his significant career in stem cell science.

The prize was linked to barbeque-stopping headlines equating his achievements to the scientific equivalent of the moon landing and paving the road to recovery for people with spinal cord injuries.

Such claims in the media imply that there is now a scientifically proven stem cell treatment for spinal cord injury. This is not the case.

For now, any clinic or headline claiming miracle cures should be viewed with caution, as they are likely to be trading on peoples hope.

Put simply, injury to the spinal cord causes damage to the nerve cells that transmit information between the brain and the rest of the body.

Depending on which part of the spine is involved, the injury can affect the nerves that control the muscles in our legs and arms; those that control bowel and bladder function and how we regulate body temperature and blood pressure; and those that carry the sensation of being touched. This occurs in part because injury and subsequent scarring affect not just the nerves but also the insulation that surrounds and protects them. The insulation the myelin sheath is damaged and the body cannot usually completely replace or regenerate this covering.

Stem cells can self-reproduce and grow into hundreds of different cell types, including nerves and the cells that make myelin. So the blue-sky vision is that stem cells could restore some nerve function by replacing missing or faulty cells, or prevent further damage caused by scarring.

Studies in animals have applied stem cells derived from sources including brain tissue, the lining of the nasal cavity, tooth pulp, and embryos (known as embryonic stem cells).

Dramatic improvements have been shown on some occasions, such as rats and mice regaining bladder control or the ability to walk after injury. While striking, such improvement often represents only a partial recovery. It holds significant promise, but is not direct evidence that such an approach will work in people, particularly those with more complex injuries.

The translation of findings from basic laboratory stem cell research to effective and safe treatments in the clinic involves many steps and challenges. It needs a firm scientific basis from animal studies and then careful evaluation in humans.

Many clinical studies examining stem cells for spinal repair are currently underway. The approaches fit broadly into two categories:

using stem cells as a source of cells to replace those damaged as a result of injury

applying cells to act on the bodys own cells to accelerate repair or prevent further damage.

One study that has attracted significant interest involves the injection of myelin-producing cells made from human embryonic stem cells. Researchers hoped that these cells, once injected into the spinal cord, would mature and form a new coating on the nerve cells, restoring the ability of signals to cross the spinal cord injury site. Preliminary results seem to show that the cells are safe; studies are ongoing.

Other clinical trials use cells from patients own bone marrow or adipose tissue (fat), or from donated cord blood or nerves from fetal tissue. The scientific rationale is based on the possibility that when transplanted into the injured spinal cord, these cells may provide surrounding tissue with protective factors which help to re-establish some of the connections important for the network of nerves that carry information around the body.

The field as it stands combines years of research, and tens of millions of dollars of investment. However, the development of stem cell therapies for spinal cord injury remains a long way from translating laboratory promise into proven and effective bedside treatments.

Each case is unique in people with spinal cord injury: the level of paralysis, and loss of sensation and function relate to the type of injury and its location. Injuries as a result of stab wounds or infection may result in different outcomes from those incurred as a result of trauma from a car accident or serious fall. The previous health of those injured, the care received at the time of injury, and the type of rehabilitation they access can all impact on subsequent health and mobility.

Such variability means caution needs to accompany claims of man walking again particularly when reports relate to a single individual.

In the case that was linked to the Australian of the Year award, the actual 2013 study focused on whether it was safe to take the patients own nerves and other cells from the nose and place these into the damaged region of the spine. While the researchers themselves recommended caution in interpreting the results, accompanying media reports focused on the outcome from just one of the six participants.

While the outcome was significant for the gentleman involved, we simply do not know whether recovery may have occurred for this individual even without stem cells, given the type of injury (stab wounds), the level of injury, the accompanying rehabilitation that he received or a combination of these factors. It cannot be assumed a similar outcome would be the case for all people with spinal injury.

Finding a way to alleviate the suffering of those with spinal cord injury, and many other conditions, drives the work of thousands of researchers and doctors around the globe. But stem cells are not a silver bullet and should not be immune from careful evaluation in clinical trials.

Failure to proceed with caution could actually cause harm. For example, a paraplegic woman who was also treated with nasal stem cells showed no clinical improvement, and developed a large mucus-secreting tumour in her spine. This case highlights the need for further refinement and assessment in properly conducted clinical trials before nasal stem cells can become part of mainstream medicine.

Its also worth noting that for spinal cord injury, trials for recovery of function are not limited to the use of stem cells but include approaches focused on promoting health of surviving nerves (neuroprotection), surgery following injury, nerve transfers, electrical stimulation, external physical supports known as exoskeletons, nanotechnology and brain-machine interfaces.

Ultimately, determining which of these approaches will improve the lives of people with spinal injury can only be done through rigorous, ethical research.

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Yes there's hope, but treating spinal injuries with stem cells is not a reality yet - The Conversation AU

Recommendation and review posted by Bethany Smith

ANDREW and Judy Kim are still searching the globe for a donor for their two-year-old son after he was diagnosed with a rare genetic condition.

The couple's son Alastair was diagnosed with chronic granulomatous disorder (CGD) in February last year.

Mr and Mrs Kim launched an appeal for help in September but the search is still on for a matching donor and their son still needs hospital treatment.

The life-threatening condition wipes out his immune system, meaning even the most minor infections leave him seriously ill.

A course of genetic therapy treatment to help him fight infections has been launched and Alastair has been treated at Oxford Children's Hospital and Great Ormond Street Hospital in London.

The only hope of a permanent cure lies in a bone marrow stem cell donor but it needs to be a 90 per cent genetic match and the family is calling for more East Asians to sign up as donors.

Mr Kim, 37, a medical research engineer, said: "It is not easy to find a match and we pray every day that it will work out.

"We have to make sure that Alastair does not get a cut because it could get infected and he does not have the ability to fight off bacteria.

"That could cascade down the line to something very dangerous for him.

"If we get ill then we have to stay away from him he loves our dog Choco Pie but he is not allowed to stroke her.

"We are doing our best to stay positive and raise awareness about his condition."

Mr and Mrs Kim, who live near Longworth with their other son Micah, five, have already searched the international register of more than four million donors but without success.

They are both of Korean descent so a matching donor will most likely be of Korean, Japanese or Chinese heritage.

The number of East Asians on international donor registers is very limited of the 617,000 registered donors in the UK just 0.5 per cent are east Asian.

The couple, who moved to Oxfordshire from Chicago nine years ago, are now appealing for people around the world, particularly East Asians, to order a free kit through a website they have set up, and take a two-minute home test to see if they could help.

Alastair has had numerous infections since he was born in September 2014.

He spent the first year-and-a-half of his life in and out of hospital but CGD is so rare, doctors never thought to test him for it but eventually a doctor at the John Radcliffe Hospital in Oxford decided to test Alastair for the condition.

The couple desperately want to find a matching donor, but also want to increase the number of East Asians on the donor register.

The couple have run several blood drives at Mrs Kim's office at Oxford University and at Harwell Oxford.

More than 90 people came forward and of those, five were able to donate blood that helped Alastair to fight infections.

Mr Kim added: "At a couple of blood drives we have found matches for other people and hopefully one day a match will found for Alastair."

To join the register go to allysfight.com

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Search goes on for bone marrow match for little Longworth lad ... - Oxford Mail

Recommendation and review posted by Bethany Smith

Human skin can be morphed into genetically modified, cancer-killing brain stem cells, according to a new study. This latest advance has only been tested in mice but eventually, its possible that it could be translated into a personalized treatment for people with a deadly form of brain cancer.

The study builds on an earlier discovery that brain stem cells have a weird affinity for cancers. So researchers, led by Shawn Hingtgen, a professor at University of North Carolina at Chapel Hill, created genetically engineered brain stem cells out of human skin. Then they armed the stem cells with drugs to squirt directly onto the tumors of mice that had been given a human form of brain cancer. The treatment shrank the tumors and extended survival of the mice, according to results recently published in the journal Science Translational Medicine.

The treatment shrank the tumors and extended survival

Usually we think about stem cell therapy in the context of rebuilding or regrowing a broken body part like a spinal cord. But if they could be modified to become cancer-fighting homing missiles, it would give patients with a deadly and incurable brain cancer called glioblastoma a better chance at survival. Glioblastomas typically affect adults, and are highly fatal because they send out a web of cancerous threads. Even when the main mass is removed, those threads remain despite chemotherapy and radiation treatment. This cancer has caused a number of high-profile deaths including Senator Edward (Ted) Kennedy in 2009, and possibly Beau Biden more recently. Approximately 12,000 new cases of glioblastoma are estimated to be diagnosed in 2017.

We really have no drugs, no new treatment options in years to even decades, Hingtgen says. [We] just really want to create new therapy that can stand a chance against this disease.

But theres a problem: brain stem cells arent exactly easy to get. Brain stem cells, more properly known as neural stem cells, hang out in the walls of the brains irrigation canals areas filled with cerebrospinal fluid, called ventricles. They generate the cells of the nervous system, like neurons and glial cells, throughout our lives.

They could be modified to become cancer-fighting homing missiles

A research group at the City of Hope in California conducted a clinical trial to make sure it was safe to treat glioblastoma patients with genetically engineered neural stem cells. But they used a neural stem cell line that theyd obtained from fetal tissue. Since the stem cells werent the patients own, people who were genetically more likely to reject the cells couldnt receive the treatment at all. For the people who could, treatment with the neural stem cells turned out to be relatively safe although at this phase of clinical trials, it hasnt been particularly effective.

More personalized treatments have been held up by the challenge of getting enough stem cells out of the patients own brains, which is virtually impossible, says stem cell scientist Frank Marini at the Wake Forest School of Medicine, who was not involved in this study. You cant really generate a bank of neural stem cells from anybody because you have to go in and resect the brain.

So instead, Hingtgen and his colleagues figured out a way to generate neural stem cells from skin which in the future, could let them make neural stem cells personalized to each patient. For this study, though, Hingtgen and his colleagues extracted the skin cells from chunks of human flesh leftover as surgical waste. That really is the magic piece here, Marini says. Now, all of a sudden we have a neural stem cell that can be used as a tumor-homing vehicle.

That really is the magic piece here.

Using a disarmed virus to infect the cells with a cocktail of new genes, the researchers morphed the skin cells into something in between a skin cell and a neural stem cell. People have turned skin cells back into a more generalized type of stem cell before. But then turning those basic stem cells into stem cells for a certain organ like the brain takes another couple of steps, which takes more time. Thats something that people with glioblastoma dont have.

The breakthrough here is that Hingtgens team figured out how to go straight from skin cells to something resembling a neural stem cell in just four days. The researchers then genetically engineered these induced neural stem cells to arm them with one of two different weapons: One group was equipped with an enzyme that could convert an anti-fungal drug into chemotherapy, right at the cancers location. The other was armed with a protein that binds to the cancer cells and makes them commit suicide in an orderly process called apoptosis.

The researchers tested these engineered neural stem cells in mice that had been injected with human glioblastoma cells, which multiplied out of control to create a human cancer in a mouse body. Both of the weaponized stem cell groups were able to significantly shrink the tumors and keep the mice alive by about an extra 30 days (for scale, mice usually live an average of two years).

Were working as fast as we can.

But injecting the cells directly into the tumor doesnt really reflect how the therapy would be used in humans. Its more likely that a person with glioblastoma would get the bulk of the tumor surgically removed. Then, the idea is that these neural stem cells, generated from the patients own skin, will be inserted into the hole left in the brain. So, the researchers tried this out in mice, and the tumors that regrew after surgery were more than three times smaller in the mice treated with the neural stem cells.

Its a promising start, but it could take a few years still before its in the clinic, Hingtgen says. He and his colleagues started a company called Falcon Therapeutics to drive this new therapy forward. Were working as fast as we can, Hingtgen says. We probably cant help the patients today. Hopefully in a year or two, well be able to help those patients.

One of the things theyll have to figure out first is whether the neural stem cells can travel the much bigger distances in human brains, and whether theyll be able to eliminate every remaining cancer cell. The caveats on this are that, of course, its a mouse study, and whether or not that directly converts to humans is unclear, Marini says. Still, he adds, Theres a very high probability in this case.

Link:
The next weapon against brain cancer may be human skin - The Verge

Recommendation and review posted by simmons

Mouse and human skin cells can be reprogrammed to hunt down tumors and deliver anticancer therapies.

Imagine cells that can move through your brain, hunting down cancer and destroying it before they themselves disappear without a trace. Scientists have just achieved that in mice, creating personalized tumor-homing cells from adult skin cells that can shrink brain tumors to 2% to 5% of their original size. Althoughthe strategy has yet to be fully tested in people, the new method could one day give doctors a quick way to develop a custom treatment for aggressive cancers like glioblastoma, which kills most human patients in 1215 months. It only took 4 days to create the tumor-homing cells for the mice.

Glioblastomas are nasty: They spread roots and tendrils of cancerous cells through the brain, making them impossible to remove surgically. They, and other cancers, also exude a chemical signal that attracts stem cellsspecialized cells that can produce multiple cell types in the body. Scientists think stem cells might detect tumors as a wound that needs healing and migrate to help fix the damage. But that gives scientists a secret weaponif they can harness stem cells natural ability to home toward tumor cells, the stem cells could be manipulated to deliver cancer-killing drugs precisely where they are needed.

Other research has already exploited this methodusing neural stem cellswhich give rise to neurons and other brain cellsto hunt down brain cancer in mice and deliver tumor-eradicating drugs. But few have tried this in people, in part because getting those neural stem cells is hard, says Shawn Hingtgen, a stem cell biologist at the University of North Carolina inChapel Hill. Right now, there are three main ways. Scientists can either harvest the cells directly from the patient, harvest them from another patient, or they can genetically reprogram adult cells. But harvesting requires invasive surgery, and bestowing stem cell properties on adult cells takes a two-step process that can increase the risk of the final cells becoming cancerous. And using cells from someone other than the cancer patient being treated might trigger an immune response against the foreign cells.

To solve these problems, Hingtgens group wanted to see whetherthey could skip a step in the genetic reprogramming process, which first transforms adult skin cells into standard stem cells and then turns those into neural stem cells. Treating the skin cells with a biochemical cocktail to promote neural stem cell characteristics seemed to do the trick, turning it into a one-step process, he and his colleague report today in Science Translational Medicine.

But the next big question was whether these cells could home in on tumors in lab dishes, and in animals, like neural stem cells. We were really holding our breath, Hingtgen says. The day we saw the cells crawling across the [Petri] dish toward the tumors, we knew we had something special. The tumor-homing cells moved 500 micronsthe same width as five human hairsin 22 hours, and they could burrow into lab-grown glioblastomas. This is a great start, says Frank Marini, a cancer biologist at the Wake Forest Institute forRegenerative Medicine in Winston-Salem, North Carolina,who was not involved with the study. Incredibly quick and relatively efficient.

The team also engineered the cells to deliver common cancer treatments to glioblastomas in mice. Mouse tumors injected directly with the reprogrammed stem cells shrank 20- to 50-fold in 2428 days compared withnontreated mice. In addition, the survival times of treated rodents nearly doubled. In some mice, the scientists removed tumors after they were established, and injected treatment cells into the cavity. Residual tumors, spawned from the remaining cancer cells, were 3.5 times smaller in the treated mice than in untreated mice.

Marini notes that more rigorous testing is needed to demonstrate just how far the tumor-targeting cells can migrate. In a human brain, the cells would need to travel a matter of millimeters or centimeters, up to 20 times farther than the 500 microns tested here, he says. And other researchers question the need to use cells from the patients own skin. An immune response, triggered by foreign neural stem cells, could actually help attack tumors, says Evan Snyder, a stem cell biologist at Sanford Burnham Prebys Medical Discovery Institute in San Diego, California, and one of the early pioneers of the idea of using stem cells to attack tumors.

Hingtgens group is already testing how far their tumor-homing cells can migrate using larger animal models. They are also getting skin cells from glioblastoma patients to make sure the new method works for the people they hope to help, he says. Everything were doing is to get this to the patient as quickly as we can.

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Reprogrammed skin cells shrink brain tumors in mice - Science Magazine

Recommendation and review posted by simmons

Glioblastoma is an aggressive form of brain cancer that kills most patients within two years of diagnosis. In tests on mice last year, a team at the University of North Carolina at Chapel Hill showed that adult skin cells could be transformed into stem cells and used to hunt down the tumors. Building on that, they've now found that the process works with human cells, and can be administered quickly enough to beat the ticking time-bombs.

Treatments for glioblastoma include the usual options of surgery, radiation therapy and chemotherapy, but none of them are particularly effective. The tumors are capable of spreading tendrils out into the brain and it can grow back in a matter of months after being removed. As a result, the median survival rate of sufferers is under 18 months, and there's only a 30 percent chance of living more than two years.

"We desperately need something better," says Shawn Hingtgen, the lead researcher on the study.

To find that something better, last year the scientists took fibroblasts a type of skin cell that generates collagen and connective tissue from mice and reprogrammed them into neural stem cells. These stem cells seek out and latch onto cancer cells in the brain, but alone are powerless to fight the tumor. To give them that ability, the scientists engineered them to express a particular cancer-killing protein. The result was mice that lived between 160 and 220 percent longer.

The next step was to test the process with human cells, and in the year since, the team has found that the results are just as promising. The technique differs slightly when scaled up to humans. The patient would be administered with a substance called a prodrug, which by itself does nothing, until it's triggered. The stem cells are engineered to carry a protein that acts as that trigger, activating the prodrug only in a small halo around itself instead of affecting the entire body. That allows the drug to target only a small desired area, ideally reducing the ill side effects that treatments like chemotherapy can induce.

Importantly, the technique can be administered quickly, to give the patients the best chance at survival.

"Speed is essential," says Hingtgen. "It used to take weeks to convert human skin cells to stem cells. But brain cancer patients don't have weeks and months to wait for us to generate these therapies. The new process we developed to create these stem cells is fast enough and simple enough to be used to treat a patient."

The treatment is an important step, but there's still a long way to go.

"We're one to two years away from clinical trials, but for the first time, we showed that our strategy for treating glioblastoma works with human stem cells and human cancers," says Hingtgen. "This is a big step toward a real treatment and making a real difference."

The research was published in the journal Science Translational Medicine.

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Stem cells beat the clock for brain cancer - New Atlas

Recommendation and review posted by Bethany Smith

Brain cancers can be really tricky to treat. Some, such as glioblastomas, spread roots through the brain tissue, meaning they are often impossible to remove surgically, leading to tragically low survival rates. But researchers are working on a way touse stem cells to track down the cancer, kill it, and then melt it away. By doing this, theyve managed to shrink brain tumors in mice to2 to 5 percent of their original size.

The trick has already been tried before using neural stem cells to hunt down and deliver cancer-killing drugs to tumors in mice. But there is a problem: It's tricky to getneural stem cells from humans. The safest way of doing this would be to take adult cells and then induce them in a two-step process to become neural stem cells. This, however, takes time.

Speed is essential, saysShawn Hingtgen, who led the research published in Science Translational Medicine. It used to take weeks to convert human skin cells to stem cells. But brain cancer patients dont have weeks and months to wait for us to generate these therapies. The new process we developed to create these stem cells is fast enough and simple enough to be used to treat a patient.

The researchers found a way to speed the process up byremoving one of the steps entirely, allowing them to produce the neural stem cells from adult skin cells in just four days. Usually, researchers would need to take the skin cell, induce it to become a generic stem cell, and then push it towards becoming a neural stem cell.

But by treating the skin cells with a cocktail of biochemicals, they were able to get the cells to turn straight into neural stem cells. They then tested these to see if they still had the same properties as original neutral stem cells and home in on tumors both in a petri dish and in animals models. They found they behaved exactly the same.

The final step was to see if they could somehow engineer these newly created cells to deliver drugs that are targeted at the cancer. They therefore got the stem cells to carry a particular protein that activates what is called a prodrug, which the researchers describe as forming a halo of drugs around the stem cell.

Were one to two years away from clinical trials, but for the first time, we showed that our strategy for treating glioblastoma works with human stem cells and human cancers, says Hingtgen. This is a big step toward a real treatment and making a real difference.

Continued here:
Scientists Reprogram Skin Cells To Hunt Down And Shrink Brain Tumors - IFLScience

Recommendation and review posted by Bethany Smith

IRVINE, Calif., Feb. 7, 2017 /PRNewswire/ --AIVITA Biomedical today announced it will present details of its patented skin care technology and commercial line of skin care products at the upcoming South Beach Symposium in Miami Beach, Florida. The conference, taking place February 9-12 at the Loews Hotel Miami Beach, will be attended by physicians and practitioners seeking the latest therapies, technologies and procedures in medical and aesthetic skin care.

The South Beach Symposium is a 4-day conference which offers multiple educational tracks allowing medical professionals from both clinical and aesthetic dermatology practices to participate in focused education. AIVITA's Chief Executive Officer, Hans S. Keirstead, Ph.D., will meet with key opinion leaders to discuss AIVITA's new product lines. AIVITA's Chief Science Officer, Gabriel Nistor, M.D., will lead a Continuing Medical Education course in Thursday's session "Anti-Aging Medicine for the Dermatologist." Dr. Nistor's course, titled Stem Cells and Growth Factors in Skin Rejuvenation, will detail advancements in the understanding and application of human stem cell-derived growth factors for skin rejuvenation. On Friday, AIVITA Biomedical Scientific Advisory Board member Dr. Zoe Draelos, M.D. will chair a special symposium, "The Science of Topical Therapy, RX, OTC and Cosmeceuticals," in which she will present research she conducted on AIVITA's skin care advancements. The company will also have a scientific poster on display highlighting the findings of a clinical study which demonstrated improvements in several key areas of visible skin aging using the company's proprietary formulation.

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AIVITA Biomedical to Present Skin Care Technology and Products at 15th Annual South Beach Symposium - PR Newswire (press release)

Recommendation and review posted by Bethany Smith

The Scientist

Regulators OK Clinical Trials Using Donor Stem Cells The Scientist WIKIPEDIA, TMHLEEResearchers in Japan who have been developing a cell therapy for macular degeneration received support from health authorities this week (February 1) to begin a clinical trial using donor-derived induced pluripotent stem (IPS) cells ...

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Regulators OK Clinical Trials Using Donor Stem Cells - The Scientist

Recommendation and review posted by Bethany Smith

The red shows rat cells in the developing heart of a mouse embryo.

A team of scientists from the Salk Institute in the United States created a stir last week with the announcement that they had created hybrid human-pig foetuses.

The story was widely reported, although some outlets took a more hyperbolic or alarmed tone than others.

One might wonder why scientists are even creating human-animal hybrids often referred to as chimeras after the Greek mythological creature with features of lion, goat and snake.

The intention is not to create new and bizarre creatures. Chimeras are incredibly useful for understanding how animals grow and develop. They might one day be used to grow life-saving organs that can be transplanted into humans.

The chimeric pig foetuses produced by Juan Izpisua Belmonte, Jun Wu and their team at the Salk Institute were not allowed to develop to term, and contained human cells in multiple tissues.

The actual proportion of human cells in the chimeras was quite low and their presence appeared to interfere with development. Even so, the study represents a first step in a new avenue of stem cell research which has great promise. But it also raises serious ethical concerns.

A chimera is an organism containing cells from two or more individuals and they do occur in nature, albeit rarely.

Marmoset monkeys often display chimerism in their blood and other tissues as a result of transfer of cells between twins while still in the womb. Following a successful bone marrow transplantation to treat leukaemia, patients have cells in their bone marrow from the donor as well as themselves.

Chimeras can be generated artificially in the laboratory through combining the cells from early embryos of the same or different species. The creation of chimeric mice has been essential for research in developmental biology, genetics, physiology and pathology.

This has been made possible by advances in gene targeting in mouse embryonic stem cells, allowing scientists to alter the cells to express or silence certain genes. Along with the ability to use those cells in the development of chimeras, this has enabled researchers to produce animals that can be used to study how genes influence health and disease.

The pioneers of this technology are Oliver Smithies, Mario Cappechi and Martin Evans, who received a Nobel Prize in Physiology or Medicine in 2007 for their work.

More recently, researchers have become interested in investigating the ability of human pluripotent stem cells master cells obtained from human embryos or created in the laboratory from body cells, to contribute to the tissues of chimeric animals.

Human pluripotent stem cells can be grown indefinitely in the laboratory, and like their mouse counterparts, they can form all the tissues of the body.

Many researchers have now shown they can make functional human tissues of medical significance from human pluripotent cells, such as nerve, heart, liver and kidney cells.

Indeed, cellular therapeutics derived from human pluripotent stem cells are already in clinical trials for spinal cord injury, diabetes and macular degeneration.

However, since 2007 it has been clear that there is not one type of pluripotent stem cell. Rather, a range of different types of pluripotent stem cells have been generated in mice and humans using different techniques.

These cells appear to correspond to cells at different stages of embryonic development, and therefore are likely to have different properties, raising the question about which source of cells is best.

Creating a chimeras has long been the gold standard used by researchers to determine the potential of pluripotent stem cells. While used extensively in animal stem cell research, chimeric studies using human pluripotent stem cells have proved challenging as few human cells survive in human-animal chimeras.

Although the number of human cells in the chimera was low, the findings by the Salk Institute researchers provide a new avenue to address two important goals. The first is the possibility of creating humanised animals for use in biomedical research.

While it is already possible to produce mice with human blood, providing an invaluable insight into how our blood and immune system functions, these animals rely on the use of human fetal tissue and are difficult to make.

The use of pluripotent stem cells in human-animal chimeras might facilitate the efficient production of mice with human blood cells, or other tissues such as liver or heart, on a larger scale. This could greatly enhance our ability to study the development of diseases and to develop new drugs to treat them.

The second potential application of human-animal chimeras comes from some enticing studies performed in Japan in 2010. These studies were able to generate interspecies chimeras following the introduction of rat pluripotent stem cells into a mouse embryo that lacked a key gene for pancreas development.

As a result, the live born mice had a fully functional pancreas comprised entirely of rat cells. If a similar outcome could be achieved with human stem cells in a pig chimera, this would represent a new source of human organs for transplantation.

While scientifically achieving such goals remains a long way off, it is almost certain that progress in pluripotent stem cell biology will enable successful experimentation along these lines. But how much of this work is ethically acceptable, and where do the boundaries lie?

Many people condone the use of pigs for food or as a source of replacement heart valves. They might also be content to use pig embryos and foetuses as incubators to manufacture human pancreas or hearts for those waiting on the transplant list. But the use of human-monkey chimeras may be more contested.

Studies have shown that early cells of the central nervous system made from human embryonic stem cells can engraft and colonise the brain of a newborn mouse. This provides a proof of concept for possible cellular therapies.

But what if human cells were injected into monkey embryos? What would be the ethical and cognitive status of a newborn rhesus monkey whose brain consists of predominantly human nerves?

It may be possible to genetically engineer the cells so that human cells can effectively grow into replacement parts. But what safeguards do we need to ensure that the human cells dont also contribute to other organs of the host, such as the reproductive organs?

While the announcement of a human-pig chimera may have taken many by surprise, regulators and medical researchers well recognise that chimeric research may raise issues in addition to the those already posed by animal research.

However, rather than call for a blanket ban or restricting funding for this area of medical research, it requires careful case-by-case consideration by independent oversight committees fully aware of animal welfare considerations and recognising existing standards.

For example, The 2016 Guidelines for Clinical Research and Translation from the International Society for Stem Cell Research call for research where human gametes could be generated from human-animal chimeras to be prohibited, but supports research using human-animal chimeras conducted under appropriate review and oversight.

Chimeric research will and needs to continue. But equally scientists involved in this field need to continue to discuss and consider the implications of their research with the broader community. Chimeras can all too readily be dismissed as mythological monsters engendering fear.

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What's the benefit in making human-animal hybrids? - The Conversation AU

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An improved gene therapy vector restores hearing and balance in genetically deaf mice, according to Boston's Children's Hospital researchers. Using therapy developed by Massachusetts Eye and Ear, the mice's levels of hearing are reported to be able to detect sounds as soft as 25 decibels, which is comparable to a whisper.

The idea of gene therapy is to deliver a corrected version of therapeutic DNA into the genomes of cells, which corrects genetic diseases.

Viruses can be altered in a laboratory to provide a "vector" that can carry the corrected therapeutic DNA into the cells. The abnormal gene expression is then altered, and the genetic disease corrected.

Previous studies have used vectors to attempt the restoration of hearing among deaf mice. However, the vectors have only managed to penetrate the inner hair cells of the cochlea.

The cochlea is a spiral-shaped tube that changes sounds to nerve messages and sends the information to the brain. Tiny hairs in the cochlea vibrate to carry information about sound to the brain.

Two new studies, published in Nature Biotechnology, have further explored vectors in mice to determine whether other hair cells of the cochlea, which are harder to reach, could be penetrated and corrected.

The first study was led by Harvard Medical School senior investigators Jeffrey R. Holt, Ph.D., of Boston's Children's Hospital in Massachusetts, Konstantina Stankovic, Ph.D., of Massachusetts Eye and Ear, and Luk H. Vandenberghe, Ph.D. The trio developed a new synthetic vector called Anc80 in 2015 at Massachusetts Eye and Ear's Grousbeck Gene Therapy Center.

The new study found that Anc80 could successfully transfer genes to the harder-to-reach areas of the outer hair cells when introduced into the cochlea. "We have shown that Anc80 works remarkably well in terms of infecting cells of interest in the inner ear," says Stankovic. "With more than 100 genes already known to cause deafness in humans, there are many patients who may eventually benefit from this technology."

Gwenalle Gloc, Ph.D., of the department of otolaryngology and F.M. Kirby Neurobiology Center at Boston's Children's Hospital, led the second study. The study tested Anc80 in a mouse model of Usher syndrome. Usher syndrome is a genetic condition caused by abnormalities of the inner ear. The condition causes partial or total hearing and vision loss that becomes worse over time, eventually impairing balance.

Gloc and colleagues aimed to find out whether delivering a corrected gene using a vector in a mouse model of Usher syndrome would enhance hearing and balance.

"This strategy is the most effective one we've tested," says Gloc. "Outer hair cells amplify sound, allowing inner hair cells to send a stronger signal to the brain. We now have a system that works well and rescues auditory and vestibular function to a level that's never been achieved before," she adds.

Gloc and the Boston's Children's Hospital team studied mice with an Ush1c gene mutation - the mutation that causes Usher type 1c among humans. The gene mutation stops a protein called harmonin from functioning, which causes the hair cells that receive sound and communicate with the brain to deteriorate, leading to hearing loss.

Introducing a corrected version of Ush1c to the inner ear of the mice shortly after birth resulted in the inner and outer hair cells in the cochlea producing normal harmonin. Furthermore, the hair cells responded to sound waves and communicated with the brain, thus enabling hearing.

The team found that 19 out of 25 mice heard sounds below 80 decibels and that some of the mice could hear sounds as quiet as 25-30 decibels. "Now, you can whisper, and they can hear you," says Gloc. The researchers also discovered that the gene therapy restored balance in the mice and eliminated erratic movements.

Hearing and balanced improved in the mice that were treated soon after birth. However, hearing and balance were not restored in the mice that were treated 10-12 days after birth.

"Anything that could stabilize or improve native hearing at an early age would give a huge boost to a child's ability to learn and use spoken language," notes Margaret Kenna, a specialist in genetic hearing loss at Boston's Children's Hospital who conducts research into Usher syndrome.

"This is a landmark study. Here we show, for the first time, that by delivering the correct gene sequence to a large number of sensory cells in the ear, we can restore both hearing and balance to near-normal levels."

Jeffrey R. Holt

Future work for the researchers will involve examining why mice treated 10-12 days after birth did not improve. They also aim to test gene therapy in larger animals and plan to develop treatments for other types of genetic hearing loss. With further work, this research may one day lead to treatments that can benefit patients.

Learn whether there is a link between anemia and hearing loss.

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Groundbreaking gene therapy restores hearing, balance - Medical News Today

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Science Daily

Gene therapy restores hearing in deaf mice, down to a whisper ... Science Daily In the summer of 2015, a team of scientists eported restoring rudimentary hearing in genetically deaf mice using gene therapy. Now the research team reports ... and more »

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Gene therapy restores hearing in deaf mice, down to a whisper ... - Science Daily

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