Advancing stem cell research in many ways

Speakers at the Alpha Stem Cell Clinics Network Symposium: Photo by Marco Sanchez

From Day One CIRM’s goal has been to advance stem cell research in California. We don’t do that just by funding the most promising research -though the 51 clinical trials we have funded to date clearly shows we do that rather well – but also by trying to bring the best minds in the field together to overcome problems.

Over the years we have held conferences, workshops and symposiums on everything from Parkinson’s disease, cerebral palsy and tissue engineering. Each one attracted the key players and stakeholders in the field, brainstorming ideas to get past obstacles and to explore new ways of developing therapies. It’s an attempt to get scientists, who would normally be rivals or competitors, to collaborate and partner together in finding the best way forward.

It’s not easy to do, and the results are not always obvious right away, but it is essential if we hope to live up to our mission of accelerating stem cell therapies to patients with unmet medical needs.

For example. This past week we helped organize two big events and were participants in another.

The first event we pulled together, in partnership with Cedars-Sinai Medical Center, was a workshop called “Brainstorm Neurodegeneration”. It brought together leaders in stem cell research, genomics, big data, patient advocacy and the Food and Drug Administration (FDA) to tackle some of the issues that have hampered progress in finding treatments for things like Parkinson’s, Alzheimer’s, ALS and Huntington’s disease.

We rather ambitiously subtitled the workshop “a cutting-edge meeting to disrupt the field” and while the two days of discussions didn’t resolve all the problems facing us it did produce some fascinating ideas and some tantalizing glimpses at ways to advance the field.

Alpha Stem Cell Clinics Network Symposium: Photo by Marco Sanchez

Two days later we partnered with UC San Francisco to host the Fourth Annual CIRM Alpha Stem Cell Clinics Network Symposium. This brought together the scientists who develop therapies, the doctors and nurses who deliver them, and the patients who are in need of them. The theme was “The Past, Present & Future of Regenerative Medicine” and included both a look at the initial discoveries in gene therapy that led us to where we are now as well as a look to the future when cellular therapies, we believe, will become a routine option for patients. 

Bringing these different groups together is important for us. We feel each has a key role to play in moving these projects and out of the lab and into clinical trials and that it is only by working together that they can succeed in producing the treatments and cures patients so desperately need.

Cierra Jackson: Photo by Marco Sanchez

As always it was the patients who surprised us. One, Cierra Danielle Jackson, talked about what it was like to be cured of her sickle cell disease. I think it’s fair to say that most in the audience expected Cierra to talk about her delight at no longer having the crippling and life-threatening condition. And she did. But she also talked about how hard it was adjusting to this new reality.

Cierra said sickle cell disease had been a part of her life for all her life, it shaped her daily life and her relationships with her family and many others. So, to suddenly have that no longer be a part of her caused a kind of identity crisis. Who was she now that she was no longer someone with sickle cell disease?

She talked about how people with most diseases were normal before they got sick, and will be normal after they are cured. But for people with sickle cell, being sick is all they have known. That was their normal. And now they have to adjust to a new normal.

It was a powerful reminder to everyone that in developing new treatments we have to consider the whole person, their psychological and emotional sides as well as the physical.

CIRM’s Dr. Maria Millan (right) at a panel presentation at the Stanford Drug Discovery Symposium. Panel from left to right are: James Doroshow, NCI; Sandy Weill, former CEO Citigroup; Allan Jones, CEO Allen Institute

And so on to the third event we were part of, the Stanford Drug Discovery Symposium. This was a high level, invitation-only scientific meeting that included some heavy hitters – such as Nobel Prize winners Paul Berg and  Randy Schekman, former FDA Commissioner Robert Califf. Over the course of two days they examined the role that philanthropy plays in advancing research, the increasingly important role of immunotherapy in battling diseases like cancer and how tools such as artificial intelligence and big data are shaping the future.

CIRM’s President and CEO, Dr. Maria Millan, was one of those invited to speak and she talked about how California’s investment in stem cell research is delivering Something Better than Hope – which by a happy coincidence is the title of our 2018 Annual Report. She highlighted some of the 51 clinical trials we have funded, and the lives that have been changed and saved by this research.

The presentations at these conferences and workshops are important, but so too are the conversations that happen outside the auditorium, over lunch or at coffee. Many great collaborations have happened when scientists get a chance to share ideas, or when researchers talk to patients about their ideas for a successful clinical trial.

It’s amazing what happens when you bring people together who might otherwise never have met. The ideas they come up with can change the world.

CIRM-funded therapy helps “bubble babies” lead a normal life

Ja’Ceon Golden; ‘cured” of SCID

At CIRM we are very cautious about using the “c” word. Saying someone has been “cured” is a powerful statement but one that loses its meaning when over used or used inappropriately. However, in the case of a new study from U.C. San Francisco and St. Jude Children’s Research Hospital in Memphis, saying “cure” is not just accurate, it’s a celebration of something that would have seemed impossible just a few years ago.

The research focuses on children with a specific form of Severe Combined Immunodeficiency (SCID) called X-Linked SCID. It’s also known as “bubble baby” disease because children born with this condition lack a functioning immune system, so even a simple infection could be fatal and in the past they were kept inside sterile plastic bubbles to protect them.

In this study, published in the New England Journal of Medicine, researchers took blood stem cells from the child and, in the lab, genetically re-engineered them to correct the defective gene, and then infused them back into the child. Over time they multiplied and created a new blood supply, one free of the defect, which helped repair the immune system.

In a news release Dr. Ewelina Mamcarz, the lead author of the study, announced that ten children have been treated with this method.

“These patients are toddlers now, who are responding to vaccinations and have immune systems to make all immune cells they need for protection from infections as they explore the world and live normal lives. This is a first for patients with SCID-X1.”

The ten children were treated at both St. Jude and at UCSF and CIRM funded the UCSF arm of the clinical trial.

The story, not surprisingly, got a lot of attention in the media including this fine piece by CNN.

Oh, and by the way we are also funding three other clinical trials targeting different forms of SCID. One with UCLA’s Don Kohn,  one with Stanford’s Judy Shizuru, and one with UCSF’s Mort Cowan

Facebook Live: Ask the Stem Cell Team About Clinical Trials

Every day at CIRM we get emails and calls from people looking for a stem cell clinical trial to help them. Some have arthritis in the knee or hip and want to avoid surgery. Some have a child with autism and want something that will ease the symptoms. Some have cancer and conventional therapies no longer work for them. Many have run out of options. Some are running out of time.

It’s hard to tell someone who is desperate that you don’t have anything that can help them, that there are no stem cell clinical trials that would be appropriate for them. Many often push back, saying they’ve seen ads online and visited websites for companies that claim to have stem cell therapies that can help them. When I say those therapies have not been approved by the Food and Drug Administration, or even been shown to be safe let alone effective, I can hear the disappointment in their voice.

I know some will go on to try those therapies anyway, because they have nothing else. I don’t blame them. I might do the same myself.

But before making an informed decision about any therapy it is important for people to have all the facts in front of them.

That’s why we are holding a special Facebook Live “Ask the Stem Cell Team About Clinical Trials” event on Thursday, April 25th from noon till 1pm PDT.

We are bringing together three experts who will help us all understand what’s a good clinical trial, and what’s a bogus one. They will talk about:

  • Red flags that a stem cell “clinic” might be more interested in making money than making you better
  • Key things to look for to choose a bona fide stem cell clinical trial
  • What are the questions you need to ask before signing up for any clinical
  • What are good sources of information to turn to for guidance

The Stem Cell Team will talk about CIRM’s Alpha Stem Cell Clinics Network, contrasting the time and resources they devote to offering patients stem cell clinical trials that are endorsed by the FDA, with clinics that promise people their own fat or blood cells can fix everything from bad knees to multiple sclerosis.

Our experts include a doctor and a nurse from the Alpha Clinics Network with years of experience in running and managing clinical trials, plus our own Geoff Lomax who helps support the entire network.

It will be an eye opening, informative and engaging hour and we want you to be part of it.  You can either join us on the day and post questions for the panel to answer, or you can email them directly to us beforehand at info@cirm.ca.gov.

Also, be sure to “like” our FaceBook page before the event to receive a notification when we’ve gone live for this and future events. If you can’t watch the broadcast “live”, not to worry, we’ll be posting it on our Facebook video page, our website, and YouTube channel shortly afterwards.

In the days leading up to the broadcast we’ll give you the broadcast link that will take you to the event itself.

We look forward to having you join us for this really important Facebook Live event.

If you are in the San Francisco Bay Area this week you can join us at our fourth Annual CIRM Alpha Stem Cell Clinics Network Symposium where the topic of how to choose a clinical trial that’s right for you will be front and center.

The symposium is on Thursday, April 18th from 8.30am to 4.30pm. It’s open to the public and it’s free.

You can find details about the event, including how you can register, HERE.

200 years later, the search for a cure for Parkinson’s continues

On the surface, actor Michael J. Fox, singer Neil Diamond, civil rights activist Jesse Jackson and Scottish comedian Billy Connolly would appear to have little in common. Except for one thing. They all have Parkinson’s Disease (PD).

Their celebrity status has helped raise public awareness about the condition, but studies show that awareness doesn’t amount to an understanding of PD or the extent to which it impacts someone’s life. In fact a study in the UK found that many people still don’t think PD is a serious condition.

To try and help change that people around the world will be holding events today, April 11th, World Parkinson’s Day.

The disease was first described by James Parkinson in 1817 in “An Essay on the Shaking Palsy”. In the essay Parkinson described a pattern of trembling in the hands and fingers, slower movement and loss of balance. Our knowledge about the disease has advanced in the last 200 years and now there are treatments that can help slow down the progression of the disease. But those treatments only last for a while, and so there is a real need for new treatments.  

That’s what Jun Takahashi’s team at Kyoto University in Japan hope to provide. In a first-of-its-kind procedure they took skin cells from a healthy donor and reprogrammed them to become induced pluripotent stem cells (iPSCs), or stem cells that become any type of cell. These iPSCs were then turned into the precursors of dopamine-producing neurons, the cells destroyed by PD, and implanted into 12 brain regions known to be hotspots for dopamine production.

The procedure was carried out in October and the patient, a male in his 50s, is still healthy. If his symptoms continue to improve and he doesn’t experience any bad side effects, he will receive a second dose of dopamine-producing stem cells. Six other patients are scheduled to receive this same treatment.

Earlier tests in monkeys showed that the implanted stem cells improved Parkinson’s symptoms without causing any serious side effects.

Dompaminergic neurons derived from stem cells

Scientists at UC San Francisco are trying a different approach, using gene therapy to tackle one of the most widely recognized symptoms of PD, muscle movement.

In the study, published in the journal Annals of Neurology, the team used an inactive virus to deliver a gene to boost production of dopamine in the brain. In a Phase 1 clinical trial 15 patients, whose medication was no longer able to fully control their movement disorder, were treated with this approach. Not only were they able to reduce their medication – up to 42 percent in some cases – the medication they did take lasted longer before causing dyskinesia, an involuntary muscle movement that is a common side effect of the PD medication.

In a news article Dr. Chad Christine, the first author of the study, says this approach may also help reduce other symptoms.

“Since many patients were able to substantially reduce the amount of Parkinson’s medications, this gene therapy treatment may also help patients by reducing dose-dependent side effects, such as sleepiness and nausea.” 

At CIRM we have a long history of funding research into PD. Over the years we have invested more than $55 million to try and develop new treatments for the disease.

In June 2018, the CIRM Board awarded $5.8 million to UC San Francisco’s Krystof Bankiewicz and Cedars-Sinai’s Clive Svendsen. They are using neural progenitor cells, which have the ability to multiply and turn into other kinds of brain cells, and engineering them to express the growth factor GDNF which is known to protect the cells damaged in PD. The hope is that when transplanted into the brain of someone with PD, it will help slow down, or even halt the progression of the disease. 

The CIRM funding will hopefully help the team do the pre-clinical research needed to get the FDA’s go-ahead to test this approach in a clinical trial. 

David Higgins, CIRM Board member and Patient Advocate for Parkinson’s Disease

At the time of the award David Higgins, PhD, the CIRM Board Patient Advocate for Parkinson’s Disease, said: “One of the big frustrations for people with Parkinson’s, and their families and loved ones, is that existing therapies only address the symptoms and do little to slow down or even reverse the progress of the disease. That’s why it’s important to support any project that has the potential to address Parkinson’s at a much deeper, longer-lasting level.”

But we don’t just fund the research, we try to bring the scientific community together to help identify obstacles and overcome them. In March of 2013, in collaboration with the Center for Regenerative Medicine (CRM) of the National Institutes of Health (NIH), we held a two-day workshop on cell therapies for Parkinson’s Disease. The experts outlined the steps needed to help bring the most promising research to patients.

Around one million Americans are currently living with Parkinson’s Disease. Worldwide the number is more than ten million. Those numbers are only expected to increase as the population ages. There is clearly a huge need to develop new treatments and, hopefully one day, a cure.

Till then days like April 11th will be an opportunity to remind ourselves why this work is so important.

Targeting clinics offering bogus stem cell therapies

For some years now CIRM has been raising the alarm about the growing numbers of clinics offering unproven and unapproved stem cell therapies. But we are not alone. Now a leader of the California state Assembly is taking action, trying to ensure the clinics follow the law and don’t endanger patients.

Kevin Mullin is the Speaker pro Tem in the Assembly. He is championing a bill, AB 617, that will create a Stem Cell Clinic Regulation Advisory Group. In a news release Mullin said the motivation behind the bill is simple:

“As the Chair of the Select Committee on Biotechnology, I have heard from patients who have experienced both sides of the treatment continuum. It is clear that more must be done to ensure the proper regulation of for-profit stem cell clinics.”

Concerns about these clinics are well-founded. The clinics claim the treatments they offer – usually involving the use of the patient’s own fat or blood cells – can help address everything from arthritis to Alzheimer’s but offer little or no proof. Because the “therapies” are not approved by the FDA they are not covered by insurance, so people spend thousands, sometimes tens of thousands of dollars for something that is almost guaranteed to do little to help. In some cases, the “treatments” have had disastrous results, harming patients.

The news release from Speaker pro Tem Mullin’s office says CIRM has helped position California as a leader in stem cell research. 

“Unfortunately, not all stem cell clinics are adhering to the expected high standards of review within the industry and, as a result, patients have been subjected to unscrupulous, sometimes harmful practices. AB 617 will address those entities by creating a Stem Cell Clinic Regulation Advisory Group.”

The Advisory Group will review existing licensing and certification laws for clinics offering stem cell therapies. The Group would then make recommendations to the Legislature about ways to improve the existing rules and ensure greater protection for patients. CIRM has been working with Speaker pro Tem Mullin on AB 617 and, as our President & CEO, Maria Millan, said we will continue to do so.

“We fully support AB 617 and Speaker pro Tem Mullin’s efforts to protect California consumers from unregulated and unproven stem cell treatments.  AB 617 will help patients, their families and the medical community identify legitimate clinics that offer scientifically tested clinical trials and treatments that meet federal regulatory requirements.  The field of regenerative medicine and cell and gene therapy are coming of age and entering the realm of medical practice, so AB 617 would set up an important foundation for ensuring that the highest quality care is provided to patients seeking these treatments.”

Gene therapy gives patient a cure and a new lease on life

Brenden Whittaker (left), of Ohio, is a patient born with a rare genetic immune disease who was treated at the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center in a CIRM funded gene therapy trial. Dr. David Williams (on right) is Brenden’s treating physician.
Photo courtesy of Rose Lincoln – Harvard Staff Photographer

Pursuing an education can be quite the challenge in itself without the added pressure of external factors. For Brenden Whittaker, a 25 year old from Ohio, the constant trips to the hospital and debilitating nature of an inherited genetic disease made this goal particularly challenging and, for most of his life, out of sight.

Brenden was born with chronic granulomatous disease (CGD), a rare genetic disorder that affects the proper function of neutrophils, a type of white blood cell that is an essential part of the body’s immune system. This leads to recurring bacterial and fungal infections and the formation of granulomas, which are clumps of infected tissue that arise as the body attempts to isolate infections it cannot combat. People with CGD are often hospitalized routinely and the granulomas themselves can obstruct digestive pathways and other pathways in the body. Antibiotics are used in an attempt to prevent infections from occurring, but eventually patients stop responding to them. One in two people with CGD do not live past the age of 40.

In Brenden’s case, when the antibiotics he relied on started failing, the doctors had to resort to surgery to cut out an infected lobe of his liver and half his right lung. Although the surgery was successful, it would only be a matter of time before a vital organ was infected and surgery would no longer be an option.

This ultimately lead to Brenden becoming the first patient in a CGD gene therapy trial at the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center.  The trial, lead by UCLA’s Dr. Don Kohn thanks to a CIRM grant, combats the disease by correcting the dysfunctional gene inside a patient’s blood stem cells. The patient’s corrected blood stem cells are then reintroduced, allowing the body to produce properly functioning neutrophils, rebooting the immune system.

It’s been a little over three years since Brenden received this treatment in late 2015, and the results have been remarkable. Dr. David Williams, Brenden’s treating physician, expected Brenden’s body to produce at least 10 percent of the functional neutrophils, enough so that Brenden’s immune system would provide protection similar to somebody without CGD. The results were over 50 percent, greatly exceeding expectations.

Brenden Whittaker mowing the lawn in the backyard of his home in Columbus, Ohio. He is able to do many more things without the fear of infection since participating in the trial. Photo courtesy of Colin McGuire

In an article published by The Harvard Gazette, Becky Whittaker, Brendan’s mother, is quoted as saying, ““Each day that he’s free of infection, he’s able to go to class, he’s able to work at his part-time job, he’s able to mess around playing with the dog or hanging out with friends…[this] is a day I truly don’t believe he would have had beyond 2015 had something not been done.”

In addition to the changes to his immune system, the gene therapy has reinvigorated Brenden’s drive for the future. Living with CGD had caused Brenden to miss out on much of his schooling throughout the years, having to take constant pauses from his academics at a community college. Now, Brenden aims to graduate with an associate’s degree in health sciences in the spring and transfer to Ohio State in the fall for a bachelor’s degree program. In addition to this, Brenden now has dreams of attending medical school.

In The Harvard Gazette article, Brenden elaborates on why he wants to go to medical school saying, ” Just being the patient for so long, I want to give back. There are so many people who’ve been there for me — doctors, nurses who’ve been there for me [and] helped me for so long.”

In a press release dated February 25, 2019, Orchard Therapeutics, a biopharmaceutical company that is continuing the aforementioned approach for CGD, announced that six patients treated have shown adequate neutrophil function 12 months post treatment. Furthermore, these six patients no longer receive antibiotics related to CGD. Orchard Therapeutics also announced that they are in the process of designing a registrational trial for CGD.

Tips on how to be a great Patient Advocate from three of the best Advocates around

No one sets out to be a Patient Advocate. It’s something that you become because of something that happens to you. Usually it’s because you, or  a loved one or a friend, becomes ill and you want to help find a treatment. Whatever the reason, it is the start of a journey that often throws you into a world that you know nothing about: a world of research studies and scientific terminology, of talking to and trying to understand medical professionals, and of watching someone you love struggle.

It’s a tough, demanding, sometimes heart-breaking role. But it’s also one of the most important roles you can ever take on. Patient Advocates not only care for people afflicted with a particular disease or disorder, they help them navigate a new and scary world, they help raise money for research, and push researchers to work harder to find new treatments, maybe even cures. And they remind all of us that in the midst of pain and suffering the human touch, a simple kindness is the most important gift of all.

But what makes a great Patient Advocate, what skills do you need and how can you get them? At CIRM we are blessed to have some of the most amazing Patient Advocates you will ever meet. So we asked three of them to join us for a special Facebook Live “Ask the Stem Cell Team” event to share their knowledge, experience and expertise with you.

The Facebook Live “Ask the Stem Cell Team About Patient Advocacy” event will be on Thursday, March 14th from noon till 1pm PST.

The three experts are:

Gigi McMillan

Gigi McMillan became a Patient Advocate when her 5-year-old son was diagnosed with a brain tumor. That has led her to helping develop support systems for families going through the same ordeal, to help researchers develop appropriate consent processes and to campaign for the rights of children and their families in research.

Adrienne Shapiro

Adrienne Shapiro comes from a family with a long history of Sickle Cell Disease (SCD) and has fought to help people with SCD have access to compassionate care. She is the co-founder of Axis Advocacy, an organization dedicated to raising awareness about SCD and support for those with it. In addition she is now on the FDA’s Patient Engagement Collaborative, a new group helping the FDA ensure the voice of the patient is heard at the highest levels.

David Higgins

David Higgins is a CIRM Board member and a Patient Advocate for Parkinson’s Disease. David has a family history of the disease and in 2011 was diagnosed with Parkinson’s. As a scientist and advocate he has championed research into the disease and strived to raise greater awareness about the needs of people with Parkinson’s.

Please join us for our Facebook Live event on Patient Advocates on Thursday, March 14 from noon till 1pm and feel free to share information about the event with anyone you think would be interested.

Also, make sure to “like” our FaceBook page before the event to receive a notification when we’ve gone live for this and future events. If you miss the broadcast, not to worry. We’ll be posting it on our Facebook video page, our website, and YouTube channel shortly afterwards.

We want to answer your most pressing questions, so please email them directly to us beforehand at info@cirm.ca.gov.

Rare Disease Gets Big Boost from California’s Stem Cell Agency

UC Irvine’s Dr. Leslie Thompson and patient advocate Frances Saldana after the CIRM Board vote to approve funding for Huntington’s disease

If you were looking for a poster child for an unmet medical need Huntington’s disease (HD) would be high on the list. It’s a devastating disease that attacks the brain, steadily destroying the ability to control body movement and speech. It impairs thinking and often leads to dementia. It’s always fatal and there are no treatments that can stop or reverse the course of the disease. Today the Board of the California Institute for Regenerative Medicine (CIRM) voted to support a project that shows promise in changing that.

The Board voted to approve $6 million to enable Dr. Leslie Thompson and her team at the University of California, Irvine to do the late stage testing needed to apply to the US Food and Drug Administration for permission to start a clinical trial in people. The therapy involves transplanting stem cells that have been turned into neural stem cells which secrete a molecule called brain-derived neurotrophic factor (BDNF), which has been shown to promote the growth and improve the function of brain cells. The goal is to slow down the progression of this debilitating disease.

“Huntington’s disease affects around 30,000 people in the US and children born to parents with HD have a 50/50 chance of getting the disease themselves,” says Dr. Maria T. Millan, the President and CEO of CIRM. “We have supported Dr. Thompson’s work for a number of years, reflecting our commitment to helping the best science advance, and are hopeful today’s vote will take it a crucial step closer to a clinical trial.”

Another project supported by CIRM at an earlier stage of research was also given funding for a clinical trial.

The Board approved almost $12 million to support a clinical trial to help people undergoing a kidney transplant. Right now, there are around 100,000 people in the US waiting to get a kidney transplant. Even those fortunate enough to get one face a lifetime on immunosuppressive drugs to stop the body rejecting the new organ, drugs that increase the risk for infection, heart disease and diabetes.  

Dr. Everett Meyer, and his team at Stanford University, will use a combination of healthy donor stem cells and the patient’s own regulatory T cells (Tregs), to train the patient’s immune system to accept the transplanted kidney and eliminate the need for immunosuppressive drugs.

The initial group targeted in this clinical trial are people with what are called HLA-mismatched kidneys. This is where the donor and recipient do not share the same human leukocyte antigens (HLAs), proteins located on the surface of immune cells and other cells in the body. Around 50 percent of patients with HLA-mismatched transplants experience rejection of the organ.

In his application Dr. Meyer said they have a simple goal: “The goal is “one kidney for life” off drugs with safety for all patients. The overall health status of patients off immunosuppressive drugs will improve due to reduction in side effects associated with these drugs, and due to reduced graft loss afforded by tolerance induction that will prevent chronic rejection.”

Media shine a spotlight on dodgy stem cell clinics

A doctor collects fat from a patient’’s back as part of an experimental stem cell procedure in Beverly Hills, Calif. on Dec. 5, 2014. (Raquel Maria Dillon / Associated Press)

For several years now, we have been trying to raise awareness about the risks posed by clinics offering unproven or unapproved stem cell therapies. At times it felt as if we were yelling into the wind, that few people were listening. But that’s slowly changing. A growing number of TV stations and newspapers are picking up the message and warning their readers and viewers. It’s a warning that is getting national exposure.

Why are we concerned about these clinics? Well, they claim their therapies, which usually involve the patient’s own fat or blood cells, can cure everything from arthritis to Alzheimer’s. However, they offer no scientific proof, have no studies to back up their claims and charge patients thousands, sometimes tens of thousands of dollars.

In the LA Times, for example, reporter Usha Lee McFarling, wrote an article headline “California has gone crazy for sketchy stem cell treatments”. In it she writes about the claims made by these clinics and the dangers they pose:

“If it sounds too good to be true, it is. There is no good scientific evidence the pricey treatments work, and there is growing evidence that some are dangerous, causing blindness, tumors and paralysis. Medical associations, the federal government and even Consumer Reports have all issued stern warnings to patients about the clinics.”

In Denver, the ABC TV station recently did an in-depth interview with a local doctor who is trying to get Colorado state legislators to take legal action against stem cell clinics making these kinds of unsupported claims.

Chris Centeno of the Centeno-Schultz Clinic, who’s specialized in regenerative medicine and research for more than a decade, said too many people are simply being scammed.

“It’s really out of control,” he told the station.

ABC7 did a series of reports last year on the problem and that may be prompting this push for a law warning consumers about the dangers posed by these unregulated treatments which are advertised heavily online, on TV and in print.

In California there is already one law on the books attempting to warn consumers about these clinics. CIRM worked with State Senator Ed Hernandez to get that passed (you can read about that here) and we are continuing to support even stronger measures.

And the NBC TV station in San Diego recently reported on the rise of stem cell clinics around the US, a story that was picked up by the networks and run on the NBC Today Show.

One of the critical elements in helping raise awareness about the issue has been the work done by Paul Knoepfler and Leigh Turner in identifying how many of these clinics there are around the US. Their report, published in the journal Cell Stem Cell, was the first to show how big the problem is. It attracted national attention and triggered many of the reports that followed.

It is clear momentum is building and we hope to build on that even further. Obviously, the best solution would be to have the Food and Drug Administration (FDA) crack down on these clinics, and in some cases they have. But the FDA lacks the manpower to tackle all of them.

That’s where the role of the media is so important. By doing stories like these and raising awareness about the risks these clinics pose they can hopefully help many patients avoid treatments that will do little except make a dent in their pocket.

Rare disease gets go-ahead to run clinical trial

crf

A young girl with cystinosis: Photo courtesy CRF

Cystinosis is one of those diseases most people have never heard of and should be very grateful they haven’t. It’s rare – affecting only around 500 children and young adults in the US and just 2,000 people worldwide – but it’s nasty. Up to now the treatments for it have been very limited. But a new clinical trial, just given the go-ahead by the Food and Drug Administration (FDA), could help change that.

Cystinosis usually strikes children before they are two years old and can lead to end stage kidney failure before their tenth birthday. It is caused by a genetic mutation that allows an amino acid, cysteine, to build up in and damage the kidneys, eyes, liver, muscles, pancreas and brain.

There is one approved therapy, cysteamine, but this only delays progression of the disease, has severe side effects and people taking it still require kidney transplants, and develop diabetes, neuromuscular disorders and hypothyroidism.

All those are reasons why, in September 2016, the CIRM Board approved $5.2 million for U.C. San Diego researcher Stephanie Cherqui, Ph.D. and her team to try a different approach. Their goal is to take blood stem cells from people with cystinosis, genetically-modify them to remove the mutation that causes the disease, then return them to the patient. The hope is that the modified blood stem cells will create a new, healthy, blood system free of the disease.

Results from pre-clinical work testing this approach in mice have been so encouraging that the FDA has given the go-ahead for that work to now be tested in people.

In a news release Nancy Stack, the Founder and President of the Cystinosis Research Foundation (CRF), the largest provider of grants for cystinosis research in the world, says this is exciting news for a community that has been waiting for a breakthrough:

“We are thrilled that CRF’s dedication to funding Dr. Cherqui’s work has resulted in FDA approval for the first-ever stem cell and gene therapy treatment for individuals living with cystinosis. This approval from the FDA brings us one step closer to what we believe will be a cure for cystinosis and will be the answer to my daughter Natalie’s wish made fifteen years ago, ‘to have my disease go away forever.’ We are so thankful to our donors and our cystinosis families who had faith and believed this day would come.”

Dr. Cherqui says if this is successful it could help more than just people with cystinosis:

“We were thrilled that the stem cells and gene therapy worked so well to prevent tissue degeneration in the mouse model of cystinosis,. This discovery opened new perspectives in regenerative medicine and in the application to other genetic disorders. Our findings may deliver a completely new paradigm for the treatment of a wide assortment of diseases including kidney and other genetic disorders. If so, CRF, through their years of support will have helped an untold number of patients with untreatable, debilitating diseases.”

Those with questions on the trials can call toll free: 844-317-7836 (STEM) and/or visit www.cystinosisresarch.org