The story behind the book about the Stem Cell Agency

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Don Reed at his book launch: Photo by Todd Dubnicoff

WHY I WROTE “CALIFORNIA CURES”  By Don C. Reed

It was Wednesday, June 13th, 2018, the launch day for my new book, “CALIFORNIA CURES: How the California Stem Cell Research Program is Fighting Your Incurable Disease!”

As I stood in front of the audience of scientists, CIRM staff members, patient advocates, I thought to myself, “these are the kind of people who built the California stem cell program.” Wheelchair warriors Karen Miner and Susan Rotchy, sitting in the front row, typified the determination and resolve typical of those who fought to get the program off the ground. Now I was about to ask them to do it one more time.

My first book about CIRM was “STEM CELL BATTLES: Proposition 71 and Beyond. It told the story of  how we got started: the initial struggles—and a hopeful look into the future.

Imagine being in a boat on the open sea and there was a patch of green on the horizon. You could be reasonably certain those were the tops of coconut trees, and that there was an island attached—but all you could see was a patch of green.

Today we can see the island. We are not on shore yet, but it is real.

“CALIFORNIA CURES” shows what is real and achieved: the progress the scientists have made– and why we absolutely must continue.

For instance, in the third row were three little girls, their parents and grandparents.

One of them was Evangelina “Evie” Vaccaro, age 5. She was alive today because of CIRM, who had funded the research and the doctor who saved her.

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Don Reed, Alysia Vaccaro and daughter Evie: Photo by Yimy Villa

Evie was born with Severe Combined Immunodeficiency (SCID) commonly called the “bubble baby” disease. It meant she could never go outside because her immune system could not protect her.  Her mom and dad had to wear hospital masks to get near her, even just to give her a hug.

But Dr. Donald Kohn of UCLA operated on the tiny girl, taking out some of her bone marrow, repairing the genetic defect that caused SCID, then putting the bone marrow back.

Today, “Evie” glowed with health, and was cheerfully oblivious to the fuss she raised.

I was actually a little intimidated by her, this tiny girl who so embodied the hopes and dreams of millions. What a delight to hear her mother Alysia speak, explaining  how she helped Evie understand her situation:  she had “unicorn blood” which could help other little children feel better too.

This was CIRM in action, fighting to save lives and ease suffering.

If people really knew what is happening at CIRM, they would absolutely have to support it. That’s why I write, to get the message out in bite-size chunks.

You might know the federal statistics—133 million children, women and men with one or more chronic diseases—at a cost of $2.9 trillion dollars last year.

But not enough people know California’s battle to defeat those diseases.

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Adrienne Shapiro at the book launch: Photo by Todd Dubnicoff

Champion patient advocate Adrienne Shapiro was with us, sharing a little of the stress a parent feels if her child has sickle cell anemia, and the science which gives us hope:  the CIRM-funded doctor who cured Evie is working on sickle cell now.

Because of CIRM, newly paralyzed people now have a realistic chance to recover function: a stem cell therapy begun long ago (pride compels me to mention it was started by the Roman Reed Spinal Cord Injury Research Act, named after my son), is using stem cells to re-insulate damaged nerves in the spine.  Six people were recently given the stem cell treatment pioneered by Hans Keirstead, (currently running for Congress!)  and all six experienced some level of recovery, in a few cases regaining some use of their arms hands.

Are you old enough to remember the late Annette Funicello and Richard Pryor?  These great entertainers were stricken by multiple sclerosis, a slow paralysis.  A cure did not come in time for them. But the international cooperation between California’s Craig Wallace and Australia’s Claude Bernard may help others: by  re-insulating MS-damaged nerves like what was done with spinal cord injury.

My brother David shattered his leg in a motorcycle accident. He endured multiple operations, had steel rods and plates inserted into his leg. Tomorrow’s accident recovery may be easier.  At Cedars-Sinai, Drs. Dan Gazit and Hyun Bae are working to use stem cells to regrow the needed bone.

My wife suffers arthritis in her knees. Her pain is so great she tries to make only one trip a day down and up the stairs of our home.  The cushion of cartilage in her knees is worn out, so it is bone on bone—but what if that living cushion could be restored? Dr. Denis Evseenko of UCLA is attempting just that.

As I spoke, on the wall behind me was a picture of a beautiful woman, Rosie Barrero, who had been left blind by retinitis pigmentosa. Rosie lost her sight when her twin children were born—and regained it when they were teenagers—seeing them for the first time, thanks to Dr. Henry Klassen, another scientist funded by CIRM.

What about cancer? That miserable condition has killed several of my family, and I was recently diagnosed with prostate cancer myself. I had everything available– surgery, radiation, hormone shots which felt like harpoons—hopefully I am fine, but who knows for sure?

Irv Weissman, the friendly bear genius of Stanford, may have the answer to cancer.  He recognized there were cancer stem cells involved. Nobody believed him for a while, but it is now increasingly accepted that these cancer stem cells have a coating of protein which makes them invisible to the body’s defenses. The Weissman procedure may peel off that “cloak of invisibility” so the immune system can find and kill them all—and thereby cure their owner.

What will happen when CIRM’s funding runs out next year?

If we do nothing, the greatest source of stem cell research funding will be gone. We need to renew CIRM. Patients all around the world are depending on us.

The California stem cell program was begun and led by Robert N. “Bob” Klein. He not only led the campaign, was its chief writer and number one donor, but he was also the first Chair of the Board, serving without pay for the first six years. It was an incredible burden; he worked beyond exhaustion routinely.

Would he be willing to try it again, this time to renew the funding of a successful program? When I asked him, he said:

“If California polls support the continuing efforts of CIRM—then I am fully committed to a 2020 initiative to renew the California Institute for Regenerative Medicine (CIRM).”

Shakespeare said it best in his famous “to be or not to be” speech, asking if it is “nobler …to endure the slings and arrows of outrageous fortune, or to take arms against a sea of troubles—and by opposing, end them”.

Should we passively endure chronic disease and disability—or fight for cures?

California’s answer was the stem cell program CIRM—and continuing CIRM is the reason I wrote this book.

Don C. Reed is the author of “CALIFORNIA CURES: How the California Stem Cell Program is Fighting Your Incurable Disease!”, from World Scientific Publishing, Inc., publisher of the late Professor Stephen Hawking.

For more information, visit the author’s website: www.stemcellbattles.com

 

SCID kid scores big on TV

Evie at book signing

One of the stories I never tire of telling is about Evie Vaccaro. She’s the little girl who was born with a fatal immune condition called severe combined immunodeficiency or SCID. Children with this condition have no immune system, no protection against infections, and often die in the first two years of life. But thanks to a stem cell therapy Evie was cured.

Evie is now five years old. A happy, healthy and, as we discovered last week, a very energetic kid. That’s because Evie and her family came to CIRM to celebrate the launch of Don Reed’s new book, “California Cures! How the California Stem Cell Program is Fighting Your Incurable Disease”.

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Don Reed with Alysia and Evie Vaccaro – Photo courtesy Yimy Villa

Don’s book is terrific – well, it’s about CIRM so I might be biased – but Evie stole the show, and the hearts of everyone there.

KTVU, the local Fox News TV station, did a couple of stories about Evie. Here’s one of them.

We will have more on Don Reed’s book later this week.

Study highlights the problem patients have in taking part in clinical trials and one simple way to change that

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Photo: courtesy Medical Daily

Let’s face it, when you are feeling crummy all you want to do is be quiet, rest and not have to deal with anyone else. So, it’s not surprising that a new survey of people with primary mitochondrial disease (PMD) found that many were often less than enthusiastic about taking part in a clinical trial.

It’s not surprising because PMD, caused by problems with the mitochondria which provide energy within our cells, can lead to a wide variety of debilitating conditions including muscle weakness, visual problems, hearing problems, heart disease, liver disease, kidney disease, gastrointestinal disorders, breathing problems, neurological problems and dementia. Any one of those is bad enough, but if you combine several you can see why it would be hard for a person with PMD to get to a clinical trial site for an experimental therapy.

That’s unfortunate because right now there are no effective treatments for PMD so it’s vitally important that people take part in clinical trials that might lead to new therapies.

Obstacles and opportunities

Fortunately, this study, published in the journal PLOS One, did more than just identify the barriers to taking part in a clinical trial, it also identified some strategies to overcome those barriers.

The barriers included not just the individual’s state of health but also:

  • Requiring patients to discontinue current medications
  • Daily blood tests
  • Requiring patients to pay for the cost of the clinical trial

Ways to encourage increased participation include:

  • Direct communication with a physician involved in the trial
  • Better education and outreach to people with PMD
  • Working with patient advocacy groups

The study says this last point in particular is extremely important.

“We propose widespread, coordinated efforts that involve PMD patient advocacy groups to organize community education sessions that clarify the components and need for efficacious clinical trial design.”

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CIRM CAP meeting

This is something that CIRM knows a lot about. Whenever we fund a clinical trial – or, in some cases a late stage pre-clinical program – we create a Clinical Advisory Panel (CAP) to support it. Each CAP consists of an independent, outside expert in whatever disease the trial is targeting, a CIRM Science Officer, and a Patient Advocate. The Patient Advocate plays a vital role in making sure this project works.

Researchers know the science, but the Patient Advocate knows what it is like to live with the disease and the limitations it may impose. They can help guide and advise the researchers on how to design a clinical trial that works for the patients and makes it as easy as possible for them to be part of the trial.

In the last few years we have created 68 CAPs, ensuring the voice of the patient, and the needs of the patient, are front and center in everything we do.

The easier it is for the patient, the easier it will be to recruit people for the trial and the more likely it is they will stay with the trial to the end. It won’t guarantee the therapy will succeed, but it gives it the best possible chance.

TELL ME WHAT I NEED TO KNOW: A Patient Advocate’s guide to being a Patient Advocate

A few weeks ago I was at the CIRM Alpha Stem Cell Clinic Network Symposium at UCLA and was fortunate enough to hear Gianna McMillan speak about patient advocacy. It was a powerful, moving, funny, and truly engaging talk. I quickly realized I wanted to blog about her talk and so for the first few minutes I was busy taking notes as fast as I could.  And then I realized that a simple blog could never do justice to what Gianna was saying, that what we needed was to run the whole presentation. So here it is.

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Gianna McMillan at the CIRM Alpha Stem Cell Clinic Symposium: Photo courtesy UCLA

TELL ME WHAT I NEED TO KNOW

Gianna McMillan, MA – Patient/Subject Advocate, Bioethics Institute at Loyola Marymount University

Stem cell research and regenerative medicine are appealing topics because patients, families and society are weary of inelegant medical interventions that inflict, in some cases, as much harm as benefit. We are tired of putting poison in our loved ones to kill their cancer or feeling helpless as other diseases attack our own bodily functions. California, full of dreamers and go-getters, has enthusiastically embraced this new technology—but it is important to remember that all biomedical research— even in a new field as exciting and inspiring as stem cell therapeutics – must adhere to basic premises. It must be valid science and it must be based on an ethical partnership with patients and research subjects.

In the world of research ethics, I wear a lot of hats. I have been a subject, a care-giver, an Institutional Review Board (IRB) member (someone who actually reviews and approves research studies before they are allowed to proceed), and I have worked with the government on regulatory committees. These days I am finishing my doctoral studies in Bioethics, and while I love the interplay of philosophy and ethical principles, I most truly identify as an in-the-trenches Patient/Subject Advocate. I am compelled to champion patients who struggle with new and devastating diagnoses, hoping desperately for a cure, and who might be faced with decisions about participating in research for their own benefit and for the greater good of science.

In the old days, doctors made decisions on behalf of their patients— who, meekly grateful for the guidance, did whatever they were told. It is a little different now. Patients are better informed, often do their own homework, and demand to be an integral part of their treatment plan. The world of research has undergone similar changes. Instead of investigators “doing things to research subjects”, best practices involve patients in the design of clinical trials. Patients and experienced subjects help decide what specific questions should be the focus of the research; they identify endpoints in the research that are meaningful to the patient population being studied; and they assist in devising tools for patient-reported outcomes and delivery of study results.

The investigator and the research subject have come to be seen as partners.

While the evolution of this important relationship is healthy and wonderful, it should not be assumed that this is an equal partnership. Why? Because subjects are always at a disadvantage.  I realize that this might be an uncomfortable concept. Physician-investigators in charge of the study might want to qualify this statement it by insisting “but we do our best to accommodate their needs”. Subjects would also rather not admit this—because it is hard to make a decision with confidence while simultaneously acknowledging, “I am really at a disadvantage here.”

However, I have learned the hard way that an honest partnership requires addressing some uncomfortable realities.

A short personal story illustrates what I am talking about. When my oldest son was five years old, he was diagnosed with malignant brain cancer. Before meeting with our son’s treatment team for the first time, my husband and I decided that my husband, articulate and concise, would take the lead. He had a legal pad, with a list of questions… each question and answer would take us down the page until, at last, we would use all the information to make a decision—a life or death decision – on behalf of our young child.

In the meeting, the neurosurgeon pointed at brain scans and explained a few things. And then radiologist drew pictures of machines and treatment angles. The oncologist described risks and benefits and side effects. Then we all looked expectantly at my husband—because it was his turn. This lovely man opened his mouth. And closed his mouth. And then burst into tears, holding that legal pad over his chest like a shield. He could not speak. After a few seconds of horrified silence, I stammered out what few questions I could remember. The doctors answered, of course. Their mouths moved, and I leaned in and nodded while making eye contact – but I have no idea what they said.  All I heard was a loud white noise that filled my skull and my husband’s raspy breathing, and my own voice crying out in my head – “Oh my God! My child! My child!”

The point of this story is to illustrate that good people, educated and prepared, ready to bring their best selves to make the most important decision they would ever make, one that would affect the life of a beloved child— these people could not function. Despite this, in just a few days’ time, we were introduced to a research study, one that might cure our child while limiting the damage to his growing brain.  No matter how well-intentioned the research team was—no matter how desirous they were of a “partnership” with us, we were at such a distinct disadvantage, that the relationship we had with these investigators could not be categorized as one “among equals”.

Even now, more than twenty years later, it is painful for me to reflect on this. But I have learned, working with hundreds of families whose children went into clinical trials, that if we can be honest about the dysfunctional nature of this situation, we might take some action to improve it. Let me be specific about the ways research subjects are at a disadvantage.

  1. They often don’t speak the language of the disease.
  2. They are unfamiliar with the process of research.
  3. They are wrestling with emotions: despair, denial, anger and hope.
  4. Their life has been disrupted – and there are consequences.

Compare this with the research team, who knows the lingo, designed the research plan, is not personally affected by the scenario and well, this is business as usual: enroll a subject, let’s get going! How is the notion of “partnership” affected by such unequal circumstances?

Is a meaningful “partnership” even possible?

I say, yes! And this notion of “partnership” is especially important as new technologies come to invade intimate qualities of “self” and the building blocks of what makes each of us human. However, we need to be realistic about what this partnership looks like. It is not equal.  I am going to take a stand here and say that the partner who has the advantage (in this case, the researcher/scientist) is morally obligated to meaningfully address the disadvantage of the other party. This bears repeating. The partner who has the advantage is morally obligated to meaningfully address the disadvantage of the other party.

Over the years, families and subjects have told me what they want and need from the doctors and researchers they work with. They say:

  1. Tell me what I need to know.
  2. Tell me in a way I can hear it.
  3. Tell me again and again.

Let me expand on these a bit. First, if I am a patient new to a diagnosis, a treatment or research—I probably do not know what I do not know. Help me learn vocabulary, procedures, and systems. Tell me about the elements of informed consent so that I recognize them when I see them in the documents you want me to sign. Explain the difference between “standard of care” and “experimental treatment”. Help me understand the research question in the context of the disease (in general) and my own ailment (in particular). Give me the words to ask the questions that I should be asking.

Secondly, there are many different ways of sharing this information: print, video, websites, peer mentors, nurse-educators, and research team members. Hit the topic from all sides and in multiple formats. Thirdly, please realize that there is a learning curve for me— and it is closely tied to my emotional journey with my predicament. I may not be able to absorb certain facts at the very beginning, but a few weeks later I might be mentally and cognitively in a different place. And obviously, I might be an inexperienced research subject when I sign the consent form— but a few months later I will be vastly more sophisticated and at that time, I need the opportunity to ask my more considered and context-savvy questions.

I want to point out that researchers have access to a deep well of wisdom – a resource that can advise and support ethical actions that will help their disadvantaged partners: researchers can ask their experienced subjects for advice.

Remember those hundreds of families I worked with, whose children ultimately enrolled in clinical trials? These experienced parents say:

  • Let me tell you what I needed to know.
  • Let me tell you how I needed to hear it.

Getting input from these experienced subjects and caregivers does two things.

First, the research team is leveraging the investment they have already made in the participants of their studies; and secondly — very importantly — they are empowering the previously disadvantaged partner. Experienced subjects can to share what they have learned or give suggestions to the research team. Physicians and researchers might even build a stable of peer mentors who might be willing to help newbies learn about the process.

Everything I have said applies to all avenues of clinical research, but these are especially important considerations in the face of new and exciting science. It took a long time for more traditional research practices to evolve into an investigator/subject partnership model. Stem cell research and regenerative medicine has the opportunity to do this from the very start—and benefit from previous lessons learned.

When I was preparing my remarks for today, someone casually mentioned that I might talk about the “importance of balancing truth-telling in the informed consent process with respect for the hope of the family.” I would like to unequivocally state that the very nature of an “informed consent process” requires 100% truth, as does respect for the family—and that this does not undermine our capacity for hope. We place our hope in this exciting new science and the doctors and researchers who are pioneers. We understand that there are many unknowns in this new field. Please be honest with us so that we might sort out our thoughts and our hopes for ourselves, in our own contexts.

What message would I wish the scientists here, today, to take away with them?      Well, I am putting on my Patient/Subject Advocate hat, and in my Patient/Subject Advocate voice, I am saying: “Tell me what I need to know!”

 

 

A road trip to the Inland Empire highlights a hot bed of stem cell research

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Gillian Wilson, Interim Vice Chancellor, Research, UC Riverside welcomes people to the combined Research Roadshow and Patient Advocate event

It took us longer than it should have to pay a visit to California’s Inland Empire, but it was definitely worth the wait. Yesterday CIRM’s Roadshow went to the University of California at Riverside (UCR) to talk to the community there – both scientific and public – about the work we are funding and the progress being made, and to hear from them about their hopes and plans for the future.

As always when we go on the road, we learn so much and are so impressed by everyone’s passion and commitment to stem cell research and their belief that it’s changing the face of medicine as we know it.

Dr. Deborah Deas, the Dean of the UC Riverside School of Medicine and a CIRM Board member, kicked off the proceedings by saying:

“Since CIRM was created in 2004 the agency has been committed to providing the technology and research to meet the unmet needs of the people of California.

On the Board I have been impressed by the sheer range and number of diseases targeted by the research CIRM is funding. We in the Inland Empire are playing our part. With CIRM’s help we have developed a strong program that is doing some exciting work in discovery, education and translational research.”

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CIRM’s Dr. Maria Millan at the Roadshow Patient Advocate event

CIRM’s President and CEO, Dr. Maria T. Millan, and our Board Chair, Jonathan Thomas then gave a quick potted history of CIRM and the projects we are funding. They highlighted how we are creating a pipeline of products from the Discovery, or basic level of research, through to the 45 clinical trials we are funding.

They also talked about the Alpha Clinic Network, based at six highly specialized medical centers around California, that are delivering stem cell therapies and sharing the experiences and knowledge learned from these trials to improve their ability to help patients and advance the field.

Researchers from both UCR then gave a series of brief snapshots of the innovative work they are doing:

  • Looking at new, more efficient and effective ways of expanding the number of human embryonic stem cells in the laboratory to create the high volume of cells needed for therapies.
  • Using biodegradable materials to help repair and regenerate tissue for things as varied as bone and cartilage repair or nerve restoration.
  • Exploring the use of epigenetic factors, things that switch genes on and off, to try and find ways to make repairs inside the body, rather than taking the cells outside the body, re-engineering them and returning them to the body. In essence, using the body as its own lab to manufacture replacement.

Another CIRM Board member, Linda Malkas, talked about the research being done at City of Hope (COH), where she is the associate chair of the Department of Molecular and Cellular Biology, calling it an “engine for discovery that has created the infrastructure and attracted people with an  amazing set of skills to bring forward new therapeutics for patients.”

She talked about how COH is home to one of the first Alpha Clinics that CIRM funded, and that it now has 27 active clinical trials, with seven more pending and 11 more in the pipeline.

“In my opinion this is one of the crown jewels of the CIRM program. CIRM is leading the nation in showing how to put together a network of specialized clinics to deliver these therapies. The National Institutes of Health (NIH) came to CIRM to learn from them and to talk about how to better move the most promising ideas and trials through the system faster and more efficiently.”

Dr. Malkas also celebrated the partnership between COH and UCR, where they are collaborating on 19 different projects, pooling their experience and expertise to advance this research.

Finally, Christine Brown, PhD, talked about her work using chimeric antigen receptor (CAR) T cells to fight cancer stem cells. In this CIRM-funded clinical trial, Dr. Brown hopes to re-engineer a patient’s T cells – a key cell of the immune system – to recognize a target protein on the surface of brain cancer stem cells and kill the tumors.

It was a packed event, with an overflow group watching on monitors outside the auditorium. The questions asked afterwards didn’t just focus on the research being done, but on research that still needs to be done.

One patient advocate couple asked about clinics offering stem cell therapies for Parkinson’s disease, wondering if the therapies were worth spending more than $10,000 on.

Dr. Millan cautioned against getting any therapy that wasn’t either approved by the Food and Drug Administration (FDA) or wasn’t part of a clinical trial sanctioned by the FDA. She said that in the past, these clinics were mostly outside the US (hence the term “stem cell tourism”) but increasingly they are opening up centers here in the US offering unproven and unapproved therapies.

She said there are lots of questions people need to ask before signing up for a clinical trial. You can find those questions here.

The visit was a strong reminder that there is exciting stem cell research taking place all over California and that the Inland Empire is a key player in that research, working on projects that could one day have a huge impact in changing people’s lives, even saving people’s lives.

 

Stem Cell Agency Heads to Inland Empire for Free Patient Advocate Event

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I am embarrassed to admit that I have never been to the Inland Empire in California, the area that extends from San Bernardino to Riverside counties.  That’s about to change. On Monday, April 16th CIRM is taking a road trip to UC Riverside, and we’re inviting you to join us.

We are holding a special, free, public event at UC Riverside to talk about the work that CIRM does and to highlight the progress being made in stem cell research. We have funded 45 clinical trials in a wide range of conditions from stroke and cancer, leukemia, lymphoma, vision loss, diabetes and sickle cell disease to name just a few. And will talk about how we plan on funding many more clinical trials in the years to come.

We’ll be joined by colleagues from both UC Riverside, and City of Hope, talking about the research they are doing from developing new imaging techniques to see what is happening inside the brain with diseases like Alzheimer’s, to using a patient’s own cells and immune system to attack deadly brain cancers.

It promises to be a fascinating event and of course we want to hear from you, our supporters, friends and patient advocates. We are leaving plenty of time for questions, so we can hear what’s on your mind.

So, join us at UC Riverside on Monday, April 16th from 12.30pm to 2pm. The doors open at 11am so you can enjoy a poster session (highlighting some of the research at UCR) and a light lunch before the event. Parking will be available on site.

Visit the Eventbrite page we have created for all the information you’ll need about the event, including a chance to RSVP and book your place.

The event is free so feel free to share this with anyone and everyone you think might be interested in joining us.

 

 

Lessons Learned & Knowledge Shared: 3rd Annual Alpha Clinics Symposium Celebrates the Delivery of Stem Cell Treatments to Patients

The CIRM Alpha Stem Cell Clinics (ASCC) Network was launched in 2015 to address a compelling unmet medical need for rigorous, FDA regulated, stem cell-related clinical trials for patients with challenging, incurable diseases. Since its inception, the Network has treated more than 200 patients in over 40 clinical trials at six leading California medical centers: UC San Diego, City of Hope, UCLA and UC Irvine, UCSF and UC Davis. That has enabled the Network to accumulate a wealth of experience and insight into how best to deliver treatments to patients, and each year it celebrates and showcases this knowledge at the CIRM Alpha Clinics Annual Symposium.

The Network is celebrating the 3rd anniversary of the ASCC Symposium on April 19th on the campus of the University of California at Los Angeles. This year’s theme is the Delivery of Stem Cell Therapeutics to Patients. Clinical investigators, scientists, patients, patient advocates, and the public will engage in thoughtful discussions on how novel stem cell treatments are now a reality. The symposium will address advancements and accomplishments of the ASCC Network in addition to developments and applications in the field of stem cell-based therapeutics. Treatments for cancer, HIV/AIDS, spinal cord injury and stroke will be featured. In addition, this year’s featured keynote speaker is David Mitchell President and Founder of Patients for Affordable Drugs.

The symposium is open to the public and is free. You can find the full agenda for the symposium here and registration can be found on the UCLA ASCC Eventbrite page. The event is highly interactive allowing participants opportunities to ask questions, network and learn about the latest developments in stem cell treatments.

Researcher and patient advocate panel at a past CIRM Alpha Clinic symposium: L to R: David Higgins, CIRM Board; David Parry, GSK; Catriona Jamieson, UCSD: John Zaia, City of Hope; John Adams, UCLA

Patient advocates speak up at the City of Hope 2nd Annual ASCC Network Symposium. (Image courtesy of the City of Hope)


Related Links:

Video illustrates potential path to stem cell repair for multiple sclerosis

“Can you imagine slowly losing the ability to live life as you know it? To slowly lose the ability to see, to walk, to grab an object, all the while experiencing pain, fatigue and depression?”

These sobering questions are posed at the beginning of a recent video produced by Youreka Science and Americans for Cures about multiple sclerosis (MS), a debilitating neurodegenerative disorder in which a person’s own immune system attacks cells that are critical for sending nerve signals from the brain and spinal cord to our limbs and the rest of our body.

In recognition of Multiple Sclerosis Awareness Week, today’s blog features this video. Using an easy to understand narrative and engaging hand-drawn illustrations, this whiteboard “explainer” video does a terrific job of describing the biological basis of multiple sclerosis. It also highlights promising research out of UC Irvine showing that stem cell-based therapies may one day help repair the damage caused by multiple sclerosis.

But don’t take my word for it, check out the five-minute video below:

Related Links:

Using the courts to protect patients from unapproved stem cell therapies

A recent article in Nature looked at using lawsuits to help rein in the activities of clinics offering “unapproved” therapies. CIRM’s Geoff Lomax explains.

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When public health officials wanted to raise awareness about the dangers of smoking they filed lawsuits against the tobacco companies. They accused Big Tobacco of deceptive marketing and hiding the negative health effects of smoking. Ultimately, they won. Now a new study says a similar tactic could prove effective in combating clinics that offer unproven stem cell therapies.

CIRM works tirelessly to accelerate the delivery of stem cell treatments to patients with unmet medical needs. But, that doesn’t mean we support any treatment that claims to help people. CIRM only partners with projects that have been given the go-ahead by the US Food and Drug Administration (FDA) to be tested in people in a clinical trial.  That’s because FDA approval means the clinical trial will be monitored and evaluated under high scientific and ethical standards.

In contrast, there are numerous examples where “stem-cell treatments” not sanctioned by the FDA are being marketed directly to patients. For years the FDA, CIRM and others have been warning consumers about the risks involved with these untested treatments. For example, just last  November the FDA issued a warning and advice for people considering stem cell treatments.

Legal steps

Last year CIRM also helped author a new California law designed to protect consumers. The law requires health care providers to disclose to patients when using a treatment that is not FDA approved or part of an FDA-sanctioned clinical trial.

At CIRM, we frequently direct patients seeking treatments to our Alpha Stem Cell Clinics Network. The Alpha Clinics only perform clinical trials that have been given the green light by the FDA, and they provide expert consultation and informed consent to patients to help ensure they make the best choice for themselves. Further, the Alpha Clinics follow up with patients after their treatments to evaluate safety and the effectiveness of the treatments.

These are steps that clinics offering unproven and unapproved therapies typically don’t follow. So, the question is how do you let people know about the risks involved in going to one of these clinics and how do you stop clinics offering “therapies” that might endanger the health of patients?

Using the law to hit clinics where it hurts

In a recently published perspective in the journal Nature an international team of policy experts considered whether civil lawsuits may play a role in stemming the tide of unproven treatments. In the article the authors say:

“The threat of financial liability for wrongdoing is the primary means by which civil law governs behavior in the private sector. Despite calls for stepping up enforcement efforts, the US Food and Drug Administration (FDA) is currently restricted in its ability to identify and target clinics operating in apparent violation of regulations. The fear of tort liability {lawsuits} may provide sufficient incentive for compliance and minimize the occurrence of unethical practices.”

The authors identified nine individual and class action lawsuits involving clinics offering what they called “unproven stem cell interventions.” A few of those were dismissed or decided in favor of the clinics, with judges saying the claims lacked merit. Most, however, were settled by the clinics with no ruling on the merits of the issue raised. Even without definitive judgements against the clinics the authors of the article conclude:

“Stem cell lawsuits could intensify publicity and raise awareness of the harms of unproven treatments, set legal precedent, reshape the media narrative from one focused on the right to try or practice to one highlighting the need for adequate safety and efficacy standards, and encourage authorities to turn their attention to policy reform and enforcement.”

The authors suggest the courts may provide a forum where medical experts can inform patients, the legal community and the public about good versus harmful clinical practices. In short, the authors believe the legal process can be an effective forum for to provide education and outreach to those with disease and the public at large.

The better option of course would be for the clinics themselves to reform their practices and engage with the FDA to test their therapies in a clinical trial. Until that happens the courts may offer an alternative approach to curbing the marketing of these unproven and unapproved therapies.

It’s time to vote for the Stem Cell Person of the Year

KnoepflerPaul14263

Paul Knoepfler

Oh well, it’s going to be another year of disappointment for me. Not only did I fail to get any Nobel Prize (I figured my blogs might give me a shot at Literature after they gave it to Bob Dylan last year), but I didn’t get a MacArthur Genius Award. Now I find out I haven’t even made the short list for the Stem Cell Person of the Year.

The Stem Cell Person of the Year award is given by UC Davis researcher, avid blogger and CIRM Grantee Paul Knoepfler. (You can vote for the Stem Cell Person of the Year here). In his blog, The Niche, Paul lists the qualities he looks for:

“The Stem Cell Person of the Year Award is an honor I give out to the person in any given year who in my view has had the most positive impact in outside-the-box ways in the stem cell and regenerative medicine field. I’m looking for creative risk-takers.”

“It’s not about who you know, but what you do to help science, medicine, and other people.”

Paul invites people to nominate worthy individuals – this year there are 20 nominees – people vote on which one of the nominees they think should win, and then Paul makes the final decision. Well, it is his blog and he is putting up the $2,000 prize money himself.

This year’s nominees are nothing if not diverse, including

  • Anthony Atala, a pioneering researcher at Wake Forest Institute for Regenerative Medicine in North Carolina
  • Bao-Ngoc Nguyen, who helped create California’s groundbreaking new law targeting clinics which offer unproven stem cell therapies
  • Judy Roberson, a tireless patient advocate, and supporter of stem cell research for Huntington’s disease

Whoever wins will be following in some big footsteps including patient advocates Ted Harada and Roman Reed, as well as scientists like Jeanne Loring, Masayo Takahashi,  and Elena Cattaneo.

So vote early, vote often.

LINK: Vote for the 2017 Stem Cell Person of the Year