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21
Nov 2016
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Transplant recipient Dan Muscat

Transplant frees scleroderma patient from excruciating pain, gives him hope for the future

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In the summer of 2014, Dan Muscat’s fingertips started to tingle. It felt like tiny shards of glass were piercing the skin underneath his fingernails.…

In the summer of 2014, Dan Muscat’s fingertips started to tingle. It felt like tiny shards of glass were piercing the skin underneath his fingernails. Over the next weeks and months the pain crept into his hands.

His family doctor thought it might be arthritis and prescribed a steroid to get things under control.  “Within days, the disease sped up,” says Muscat. “It went from the top of my head to my toes.  My ankles and my feet were so inflamed I couldn’t walk 20 feet.”

Muscat, who owns a jewelry business in St. Thomas, Ontario, was referred to a rheumatologist who immediately recognized what was happening to him: “Dan had rapidly progressive early onset diffuse scleroderma,” says Dr. Janet Pope of St. Joseph’s Health Care in London.

Scleroderma — from the Greek words for “hard” (sclera) and “skin” (derma) — is the term used for a group of rare autoimmune diseases that, according to the Mayo Clinic, involve hardening of the skin and connective tissues due to an overproduction of collagen.  Diffuse scleroderma is a systemic type of the disease, meaning it affects not only the skin and muscle but also the digestive tract and internal organs, ultimately leading to death.

The future did not look good for Muscat: “He had not responded to usual immune suppressive treatment,” says Dr. Pope.

By late 2015, Muscat was growing desperate. He had heard that Dr. Richard Burt at Northwestern University in Chicago was offering a treatment using a patient’s own bone marrow stem cells to rebuild their immune system and shut down scleroderma.  Muscat reached out to the Canadian Stem Cell Foundation for guidance.

“I investigated costs of Northwestern University treatment,” says Muscat, “and it was somewhere between $300,000 and $350,000. I thought about selling my business or my home.  If I had to, I would have done that.  Financially, it would have been a huge burden.”

Because the American treatment is similar to the combination chemotherapy/ bone marrow stem cell transplant treatment that The Ottawa Hospital’s Dr. Harry Atkins has used successfully against extreme forms of multiple sclerosis (MS, which is also an autoimmune disorder), the Foundation suggested Muscat reach out to him.  Earlier this year Dr. Atkins agreed to take Muscat on as his first scleroderma patient.

“He fit the profile of the kind of patient best treated with these kinds of stem cell transplants” says Dr. Atkins, who is also an associate professor at the University of Ottawa.  “There was a randomized trial carried out in Europe that was very well done and had very good results.  I know the doctors involved and we’d talked a little bit about it, but their methods had also been published and are available for everybody. That’s why we publish — to spread the knowledge.”

By the time Muscat arrived in Ottawa in May, the disease had taken its toll.  Once an imposing figure who worked out regularly and thought nothing of going for five- or six- miles runs, Muscat had watched 45 pounds fade from his six-foot frame.  His brisk walk had turned into a painful shuffle.  His boundless energy (he built a log home by himself, is an avid fisherman and collects classic sports cars) disappeared, making it difficult to get through a day. His fingers became like stone claws, curving in at the knuckles. His skin was so impenetrable he worried the Ottawa medical team might not be able to run an intravenous line.  And the pain was excruciating.

“On a scale of 1 to 10, about 15,” Muscat says.

Muscat began the therapy in June and spent much of last summer undergoing the treatment designed to destroy the diseased immune system and rebuild it with his own purified stem cells, ideally to produce a new, scleroderma-free one.  Though not as harsh as the chemotherapy MS patients undergo, it is not a treatment to be taken lightly.  The risk did not dissuade Muscat: “If the treatment didn’t kill me, the disease would.”

He made it through chemo with few noticeable side-effects beyond losing his hair and tolerating a couple of infections.  Released from hospital in August, he embraced physiotherapy, doing three to five hours daily to get his flexibility back and rid his body of the collagen buildup.

He is now back in St. Thomas, occasionally working at the store — he has to limit his time in public places because the chemo has left him vulnerable to infections. “My pain is one or two percent compared to what it was before.” His range of motion with his arms and legs is much improved and he can get around on foot again.

He realizes it could take a year before the chemo drugs fade from his system and the full benefits of the treatment kick in. Muscat, grateful for the excellent care provided by The Ottawa Hospital’s bone marrow transplant team, is unswerving in his belief that he can beat this disease. “I feel physically and mentally better.  I just don’t feel that I’m being attacked.”

In an email message, rheumatologist Dr. Pope said that while Muscat “has a long road ahead, we expect more recovery with softening of his skin and improved function. His pain is less and he feels that shortly after the transplant a ‘switch’ was turned back to how it should be and he developed a better sense of well-being. I will follow him carefully and I hope for more improvement over the next year or two. He is in physiotherapy and is getting ongoing intravenous drugs for safety as his immune system continues to reconstitute. I am very hopeful that Dan will be even better over time. This is not a cure, but has given Dan a better chance of recovery.”

In Ottawa, Dr. Atkins also urges caution. “It’s going to take months and months to know if this is a real improvement. The changes with scleroderma don’t happen overnight.  This is not a curative treatment; it doesn’t make scleroderma go away and never come back.  It’s something that helps a population of scleroderma patients: some patients improve, some not.  There are patients that progress or relapse again.”

Muscat is positive about the future: “I still have the remnants of the disease, but I can jump in and out of my truck again and I can walk a couple of kilometres with no problems.  I feel very optimistic, like I’ve been given a second chance.”

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17
Nov 2016
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Harry Atkins capture

Dr. Harry Atkins

Stem cell transplant defeats myasthenia gravis

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We can add another disease to list of conditions that can now be treated with bone marrow stem cell transplants: myasthenia gravis (MG).…

We can add another disease to list of conditions that can now be treated with bone marrow stem cell transplants: myasthenia gravis (MG).

This week, CTV News in Ottawa told the story of Anne Scott, a 58-year-old Kemptville, Ontario woman whose MG refused to respond to conventional therapies.

MG is an autoimmune disorder like multiple sclerosis (MS).  It interrupts communication between the muscles and the nerves, which can make swallowing and breathing difficult.

While most patients can be treated with conventional therapies, some, like Scott, cannot.  She had to be placed on life support a dozen times.

Under the supervision of The Ottawa Hospital’s Dr. Harry Atkins, Scott underwent a chemotherapy/stem cell procedure. Essentially, it involves one using a patient’s own blood stem cells to rebuild their immune system after it has been wiped out by chemo therapy. If all goes  well, the rebuilt immune system is free of the disease.

Dr. Atkins has had success using this approach to treat patients with MS, Stiff Person’s Syndrome, neuromyelitis optica and Crohn’s disease. The procedure has been used to treat leukemia and other forms of blood-based cancer for decades.

Scott, who was able to attend her daughter’s wedding  shortly after undergoing the procedure, was one of seven patients in a study led by Dr. Atkins, the results of which have been reported in in JAMA Neurology  All seven patients no longer need treatment for MG and are symptom-free.

You can find out more about the study here.

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16
Nov 2016
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Dr. Paola Marignani

A two-pronged approach to stopping cancer

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Dr. Paola Marignani is searching for new treatments.  More precisely, the Dalhousie Medical School researcher wants a new combination of drugs to target cancer in a new and different way.…

Dr. Paola Marignani is searching for new treatments.  More precisely, the Dalhousie Medical School researcher wants a new combination of drugs to target cancer in a new and different way.

“Many of the drugs we have in the clinic today block oncogenes that were discovered 15 or 20 years ago,” says Dr. Marignani, pictured at right.  “We can’t just keep using the same drugs over and over again in different combinations.  We need to find new targets and new drugs.”

Her key target is LKB1, a protein that has multiple functions throughout the body, including tumour suppression.  LKB1 is often found to be missing or mutated in breast cancer, lung cancer, pancreatic cancer and other forms of the disease.  “If you lose LKB1 or its mutated, it sets up protein signalling pathways for disaster,” says Dr. Marignani.  “It would be like an intersection that once had stop signs and is now without any.  Eventually an accident is going to happen.”

To understand what happens when LKB1 is lost and mutated, her team used re-engineered mice without the ability to express the protein.  That led them to finding a combination of compounds that shuts down aggressive, metabolically active HER2-positive breast cancers — a common form of the disease — in the rodents.

Dr. Marignani is using a two-pronged approach by testing the new compounds in combination with Herceptin®, which has become the standard of care for HER2-positive breast cancer.  “We know Herceptin® is effective,” says Dr. Marignani.  “We can use the discovery we made in mice to strategically attack the cancers from multiple branches using new drugs in combination with the standard treatment of care, and see what happens.”

So far, she is cautiously optimistic.  “The animals tolerate the drug combination, which is very important, and early data suggests the tumours are not progressing.”

Beyond shutting down tumour growth, Dr. Marignani and her team want to find ways to stop cancer from recurring by killing off the cancer stem cells that resist the original treatment.

“There is always the possibility that there are some cancer stem cells hiding out, just waiting, that have developed resistance to the treatment that killed off the bulk of the cancer,” she explains.  “We have seen that our drug combination reduced the proteins that drive recurrence.  We did not anticipate this would happen because there was no evidence in the literature.  In our current study, we need to consider whether stem cells play a role in cancer recurrence in our model and look for pathways that are active in those cells.  We don’t know yet.  We’re working on it.”

With fine tuning of her animal-model preclinical work done, Dr. Marignani hopes to move into a Phase 1 clinical trial, testing the safety of the drug combination in humans.  But she emphasizes that the work is in the early stages.

“It is important that we look for new treatment possibilities even though the current treatments are reasonably good. Targeting oncogenes has served us well, however it is time we expand our toolbox. We can do better.”

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14
Nov 2016
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McMaster’s research centre marks decade with new site/logo

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After a decade of innovation, McMaster’s Stem Cell and Cancer Research Institute (SCC-RI) has launched a new website to draw attention to research advances and share links to resources for patients, care givers and future scientists. …

After a decade of innovation, McMaster’s Stem Cell and Cancer Research Institute (SCC-RI) has launched a new website to draw attention to research advances and share links to resources for patients, care givers and future scientists.  The SCC-RI’s new logo, a stylized hand, represents the numerous people involved in translating research into new therapeutic options for patients.

The SCC-RI was established in 2006 — the same year Dr. Shinya Yamanaka established his game-changing protocol to turn adult skin stem cells back to an embryonic stem cell-like state of pluripotency — to drive new therapies to the clinic. Since the beginning, SCC-RI has focused its research on improving bone marrow and cord blood transplants, finding cell-based solutions to cancer and identifying and targeting the cells responsible for neural disorders such as autism.

“Our commitment to working with human cells and our established drug discovery capabilities make this the best place for moving forward to patient-specific drug discovery,” said Dr. Karun Singh, Principal Investigator at SCC-RI in a recent blog post.

Dr. Singh recently led a team that discovered a gene mutation that causes autism spectrum disorders (ASD).  Having identified the genetic glitch, researchers can now focus their efforts on finding a way to improve the brain connections that are causing symptoms of ASD.

The SCC-RI team has developed a robotic system to test a library of currently available drugs on a variety of diseased cells before starting human trials. In a landmark study, Dr. Mick Bhatia, SCC-RI Director and Senior Scientist, found the antipsychotic drug, thioridazine, kills cancer stem cells responsible for initiating leukemia without harming normal stem cells. A  Phase I clinical trial is now underway.

 

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08
Nov 2016
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Allen Eaves wins Entrepreneur of the Year Pacific Region

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Dr. Allen Eaves, President and CEO of STEMCELL Technologies Inc., has been awarded Entrepreneur of the Year Pacific Winner by Ernst and Young Canada.…

Dr. Allen Eaves, President and CEO of STEMCELL Technologies Inc., has been awarded Entrepreneur of the Year Pacific Winner by Ernst and Young Canada.

The theme for the 2016 awards is recognizing accelerators — those entrepreneurs who “accelerate all of us by bringing us new products and services and by driving our economy” explains Lui Pettrollini, Entrepreneur of the Year Pacific Program Director, in an article carried in the Globe & Mail’s Report On Business Magazine.

Dr. Eaves founded STEMCELL in 1993 to support his own research as the head of the BC Cancer Agency’s Terry Fox Laboratory in Vancouver. Unable to source what he needed to support his research, Dr. Eaves began making media for growing blood-forming stem cells. Today, STEMCELL Technologies offers more than 2,000 cell biology tools to researchers in over 80 countries. It is Canada’s largest biotech company with 850 employees and $150 million in annual sales.

“I run my company like a graduate training program,” Dr. Eaves said in the ROB Magazine piece. “Scientists have a lot of freedom in deciding what they can work on and that inspires them to be creative and think clever thoughts.”

Dr. Eaves has also developed a group of companies including STEMSOFT Software Inc., that makes software for managing data in bone marrow centres, cord blood banks, cellular therapy companies as well as tumour and tissue repositories. He also founded Malachite Management Inc., which provides association management, conference management, fundraising and strategic planning to medical and scientific organizations.

Dr. Eaves is a major supporter of the Canadian Stem Cell Foundation as both a member of the Board of Directors and a founding Till & McCulloch Leadership Circle member.

Dr. Eaves’ accomplishments will be celebrated along with other regional winners on November 22nd at the EY National Awards Gala in Toronto.

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01
Nov 2016
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Schizophrenia linked to neural stem cells producing too few neurons

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Japanese researchers have found that a genetic mutation linked to schizophrenia changes the way brain cells develop and differentiate.

According to a report in today’s Guardian newspaper, the alteration of cell development in the brain changes the normal balance of neurons (nerve cells) and connective tissue in the brain (glia).…

Japanese researchers have found that a genetic mutation linked to schizophrenia changes the way brain cells develop and differentiate.

According to a report in today’s Guardian newspaper, the alteration of cell development in the brain changes the normal balance of neurons (nerve cells) and connective tissue in the brain (glia).

The researchers’ findings, published in Translational Psychiatry, suggest that abnormal neural differentiation leads to fewer neurons and more non-neuronal cells being produced during early stages of brain development, which could be contributing to the presence of the disease.

Dr. Manabu Toyoshima of Japan’s RIKEN Brain Science Institute extracted skin cells from two female schizophrenic patients and two healthy individuals, then reprogrammed them to generate induced pluripotent stem cells (iPS cells), which are like embryonic stem cells in that they have the capacity to differentiate into any cell type in the body.

In essence, the scientists found that the neurospheres (clusters of neural stem cells) derived from the IPS cells of the schizophrenic patients were smaller and produced fewer neurons but significantly more astrocytes — star shaped glial cells.

The findings could help scientists better understand the origins of schizophrenia, a common form of mental illness that affects about one in 100 people and, as the Guardian points out, “is known to be highly heritable, but is genetically complex.”

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24
Oct 2016
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BC Science World unveils bronze tribute to Till & McCulloch

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Scott Sampson, artist Ruth Abernathy, Allen Eaves and Connie Eaves pose with Till& McCulloch statue at Telus World of Science on Sunday

Fifty-five years after they became the first in the world to identify stem cells, Drs.

Scott Sampson, artist Ruth Abernathy, Allen Eaves and Connie Eaves pose with Till& McCulloch statue at Telus World of Science on Sunday

Scott Sampson, artist Ruth Abernathy, Allen Eaves and Connie Eaves pose with Till& McCulloch statue at Telus World of Science on Sunday

Fifty-five years after they became the first in the world to identify stem cells, Drs. James Till and Ernest McCulloch are now a must-see attraction at the Telus World of Science in Vancouver.

Artist Ruth Abernethy’s “portrait in bronze” of the two Canadian researchers was unveiled yesterday outside the main doors of the science centre. Within minutes, children were climbing the stools of the interactive artwork and clamouring to have their photos taken with the great men.

The work of art was commissioned by Dr. Allen Eaves, founder and CEO of STEMCELL Technologies Inc., Canada’s leading biotech company, to honour Till & McCulloch who are well known within the field as the Fathers of Stem Cell Science but virtually  unknown to most Canadians.  Dr. Eaves and his wife Dr. Connie Eaves, an internationally respected stem cell researcher, worked with Drs. Till and McCulloch decades ago at the Ontario Cancer Institute in Toronto.

As Science World CEO Scott Sampson  said in his address to the crowd that gathered at Science World, the tribute is a reflection of new-found Canadian pride in our country’s outstanding achievements in a number of fields.

A sister statue will be unveiled in Toronto in the spring of 2017.

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21
Oct 2016
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Trainees meet with top researchers at the T&M conference

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Billed as “Canada’s premier stem cell research event” the Till & McCulloch Meetings provide an excellent opportunity for young scientists to discuss their work with sector leaders and talk about how they can play a role in advancing regenerative medicine.…

Billed as “Canada’s premier stem cell research event” the Till & McCulloch Meetings provide an excellent opportunity for young scientists to discuss their work with sector leaders and talk about how they can play a role in advancing regenerative medicine.

Named after Drs. James Till and Ernest McCulloch, who discovered stem cells in 1961, the annual conference attracts hundreds of participants from across Canada and all over the world. The meetings, which begin Monday and run through Wednesday in Whistler, BC, foster new collaborations and nurture Canadian leadership and innovation.

Since the conference was first organized in 2001, much effort has been made to support young scientists via networking sessions, workshops and presentations. Top trainees are acknowledged with awards for their poster submissions, presentations and research efforts

Recognizing the importance of the trainee experience in Canada, a special session will be held to develop programs for young investigators. Participants will be asked to recommend the types of workshops and skills development best suited to support trainees and will participate in organizing them. Previous sessions have included workshops in career development, grant review and journal writing and ethics.

For more information on the Till & McCulloch Meetings 2016 click here. 

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20
Oct 2016
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Celebrating the 55th anniversary of one of Canada’s greatest medical discoveries

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Fifty-five years ago, Drs. James Till and Ernest McCulloch identified stem cells and provided the theoretical underpinning for bone marrow transplant procedures that have saved the lives of countless leukemia patients.…

Fifty-five years ago, Drs. James Till and Ernest McCulloch identified stem cells and provided the theoretical underpinning for bone marrow transplant procedures that have saved the lives of countless leukemia patients. It also opened up the field of stem cell science.

To commemorate the breakthrough, which ranks as one of Canada’s greatest medical discoveries, Science World at TELUS World of Science in Vancouver will unveil a bronze portrait of Drs. Till and McCulloch on Sunday.

“It’s impossible to overstate the impact of Dr. Till and Dr. McCulloch’s discovery and their long-time collaboration,” says, Dr. Allen Eaves, President & CEO of STEMCELL Technologies Inc. that commissioned the work of art. “Their work changed the course of cancer research and paved the way for what we now call regenerative medicine.”

Both Dr. Eaves, who co-founded the Terry Fox Laboratory with the BC Cancer Agency, and his wife, prominent cancer researcher Dr. Connie Eaves, were greatly influenced by Drs. Till and McCulloch during their time at the Ontario Cancer Institute in Toronto. Connie was a post-doctoral fellow who worked closely with them and Allen used their methodology in his own cancer research, which led him to set up the first bone marrow transplantation program in Western Canada.

The sculpture was created by renowned artist Ruth Abernethy, whose public portraits in bronze have celebrated the achievements of several prominent Canadians. She is probably best known for her depiction of Glenn Gould sitting on a bench at CBC in Toronto and Oscar Peterson tinkling a piano outside the National Arts Centre in Ottawa. She will attend the unveiling Sunday to talk about her work and sign copies of Life and Bronze: a Sculptor’s Journal.

The accomplishments of Drs. Till and McCulloch are also celebrated in the book Dreams & Due Diligence: Till and McCulloch’s Stem Cell Discovery and Legacy. Author Joe Sornberger will be at the unveiling Sunday to sign copies of his book.  The book is available for purchase on our website by clicking on this link.

The public is welcome to attend the unveiling at Science World at 1455 Quebec Street on Sunday at 1 p.m. Next spring, a sister bronze portrait will be installed at the MaRS Building in downtown Toronto.

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06
Oct 2016
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Steer clear of clinics offering unproven treatments, experts warn

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Suppose you have a disease for which there currently is no cure.  You go online and find a stem cell clinic in the United States offering to rid you of the condition with their miraculous new treatment. …

Suppose you have a disease for which there currently is no cure.  You go online and find a stem cell clinic in the United States offering to rid you of the condition with their miraculous new treatment.  The clinic looks reputable enough and even has certified doctors on staff.  When you investigate further you find out that it will set you back somewhere between $15,000 and $25,000. What should you do?

“Steer clear of them,” advises Dr. Lawrence Goldstein, director of the Sanford Stem Cell Clinical Center at UC San Diego.  “They’re probably taking advantage of you and it’s probably unproven.”

Dr. Lawrence’s made the comments in an Orange Counter Register news feature this week that warns people “whose lives – or whose loved ones’ lives – have been upended by chronic or incurable conditions such as Parkinson’s” to be cautious about clinics offering unproven stem cell cure-alls.

“If somebody is telling you something that’s too good to be true, it’s too good to be true,” says Dr. Sidney Golub, director of the Sue and Bill Gross Stem Cell Research Center at UC Irvine.

We have frequently used this blog to urge caution to those considering stem cell treatments abroad, especially in countries with less-stringent health regulations and clinics offering to treat a laundry list of diseases and conditions with the same stem cell “cure.”

But as the Register piece indicates, there are now nearly 600 clinics in the United States — just a quick drive or flight away for most Canadians — touting expensive stem cell treatments for everything from breast augmentation to spinal cord injuries.  Many use stem cells drawn from the patient’s own fat, arguing that such treatments don’t require approval from the U.S. Food and Drug Administration (FDA).

Our website has a section on stem cell tourism.  You can find it here.  It links to an excellent guidebook produced by the University of Alberta’s Faculty of Law,  Albany Medical College and the Stem Cell Network.  There is also a great web resource provided by the International Society for Stem Cell Research.  You can find it here.

Beyond the possibility of spending money on a treatment that does not work, there is always a risk that the procedure may have adverse side effects. “The risk is far greater than the potential benefit,” Mary Bass, a director of the patient advocacy group Americans for Cures, told the Register.

The FDA also warns that patients may be “vulnerable to unscrupulous providers of stem cell treatments that are illegal and potentially harmful.”

 

 

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