Boosting immune system cells could offer a new approach to treating Lou Gehrig’s disease

ALS

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, is one of those conditions that a lot of people know about but don’t know a lot about. If they are fortunate it will stay that way. ALS is a nasty neurodegenerative disease that attacks motor neurons, the cells in the brain and spinal cord that control muscle movement. As the disease progresses the individual loses their ability to walk, talk, eat, move and eventually to breathe. There are no effective treatments and no cure. But now research out of Texas is offering at least a glimmer of hope.

Dr. Stanley Appel, a neurologist at the Houston Methodist Neurological Institute noticed that many of the ALS patients he was treating had low levels of regulatory T cells, also known as Tregs. Tregs play a key role in our immune system, suppressing the action of molecules that cause inflammation and also helping prevent autoimmune disease.

In an article on Health News Digest Appel said:

Stanley Appel

Dr. Stanley Appel: Photo courtesy Australasian MND Symposium

“We found that many of our ALS patients not only had low levels of Tregs, but also that their Tregs were not functioning properly. We believed that improving the number and function of Tregs in these patients would affect how their disease progressed.”

And so that’s what he and his team did. They worked with M.D. Anderson Cancer Center’s Stem Cell Transplantation and Cellular Therapy program on a first-in-human clinical trial. They took blood from three people with different stages of ALS, separated the red and white blood cells, and returned the red blood cells to the patient. They then separated the Tregs from the white blood cells, increased their number in the lab, and then reinfused them into the patients, in a series of eight injections over the course of several months.

Their study, which appears in the journal Neurology,® Neuroimmunology & Neuroinflammation, found that the therapy appears to be safe without any serious side effects.

Jason Thonhoff, the lead author of the study, says the therapy also appeared to help slow the progression of the disease a little.

“A person has approximately 150 million Tregs circulating in their blood at any given time. Each dose of Tregs given to the patients in this study resulted in about a 30 to 40 percent increase over normal levels. Slowing of disease progression was observed during each round of four Treg infusions.”

Once the infusions stopped the disease progression resumed so clearly this is not a cure, but it does at least suggest that keeping Tregs at a healthy, high-functioning level may help slow down ALS.

CIRM is funding two clinical trials targeting ALS. One is a Phase 1 clinical trial with Clive Svendsen’s team at Cedars-Sinai Medical Center, the other is a Phase 3 project with Brainstorm Cell Therapeutics.

The Mother of Modern Medicine: Henrietta Lacks’ Portrait Unveiled at National Portrait Gallery

Back during my research scientist days, using HeLa cells for my experiments was as commonplace as a carpenter reaching for his hammer at a construction site. What makes these cells so handy is their robustness: they are easy to maintain in the lab where they divide indefinitely in petri dishes.

HeLaV_M

Scanning electron micrograph of just-divided HeLa cells.
Credit: National Center for Microscopy and Imaging Research

Henrietta Lacks and the Story of HeLa Cells
The reason they grow so readily is because they originally came from a patient’s tumor. For the longest time I had been under the impression that “HeLa” stood for Helen Lang, supposedly the patient’s name. It wasn’t until Rebecca Skloot’s award-winning book, “The Immortal Life of Henrietta Lacks”, was published in 2010, that I learned their true identity.

Only 31 years old, Henrietta Lacks died of cervical cancer in 1951. Before she died, cells from her cancer were collected without her permission or knowledge. Noticed for their remarkable ability to continually divide in cell culture, these cells, labelled as “HeLa”, became the first human cell line. Though Henrietta Lacks is long gone, her cells still live on in research labs all over the world and have been instrumental to many important discoveries and over 10,000 patents.

The Mother of Modern Medicine: The Portrait

lacks1

Henrietta Lacks (HeLa): The Mother of Modern Medicine by Kadir Nelson (see full portrait here)

The story of Lacks’ contribution to science can now be appreciated not only in the form of words on a page but also paint on canvas. Last week, the Smithsonian’s National Portrait Gallery, in collaboration with the National Museum of African American History and Culture, installed Kadir Nelson’s 2017 portrait of Lacks on the museum’s first-floor presentation wall.

In a Smithsonian.com article, painting and sculpture curator, Dorothy Moss, explained that Lacks’ portrait will be installed next to portraits of more recognizable Americans like Barack Obama and Susan B. Anthony where she hopes it acts as a, “signal to the kinds of history we want to tell. We want to make sure that people who have not been written into traditional narratives of history are visible immediately when our visitors enter. It will spark a conversation about people who have made a significant impact on science yet have been left out of history.”

When you look at the painting, be sure to notice some subtle details that help tell Lacks’ story, like the two missing buttons in her dress that symbolize her cells that were taken without her permission and the “Flower of Life” wall paper pattern meant to represent immortality.

CIRM’s Commitment to the Patient
It’s the learning from the unethical treatment of patients like Henrietta Lacks that in recent years has driven more focus on protecting patients and given them a voice when it comes to their care and their participation in medical research.

This commitment to patients is at the forefront of everything we do at CIRM. For instance, our 29-member Governing Board is composed of ten patient advocates, our CIRM-funded clinical trials are supported by Clinical Advisory Panels (CAPs) that include a patient advocate at the table and our mission itself is wholly focused on accelerating stem cell treatments to patients with unmet medical needs.

I think it’s very appropriate that Henrietta Lack’s portrait is titled, “The Mother of Modern Medicine” because of her legacy of empowering patients to advocate for the development of life-saving therapies.

New findings about muscle stem cells reveals the potential for growing replacement organs

Chrissa Kioussi’s group at Oregon State University has made exciting advances in further unraveling the scientific mysteries of stem cells. In work detailed in Scientific Reports, this group found that muscle-specific stem cells actually have the ability to make multiple different cell types.

muscle_bicep_FaceBook_shutterstock_162592241

Pumping up our knowledge about muscle stem cells

Initially, this group was interested in understanding how gene expression changes during embryonic development of skeletal muscle. To understand this process, they labeled muscle stem cells with a kind of fluorescent dye, called GFP, which allowed them to isolate these cells at different stages of development.  Once isolated, they determined what genes were being expressed by RNA sequencing. Surprisingly, they found that in addition to genes involved in muscle formation, they also identified activation of genes involved in the blood, nervous, immune and skeletal systems.

This work is particularly exciting, because it suggests the existence of stem cell “pockets,” or stem cells that are capable of not only making a specific cell type, but an entire organ system.

In a press release, Dr. Kioussi said:

chrissa_kioussi

Chrissa Kioussi, PhD

“That cell populations can give rise to so many different cell types, we can use it at the development stage and allow it to become something else over time… We can identify these cells and be able to generate not one but four different organs from them — this is a prelude to making body parts in a lab.” 

This study is particularly exciting because it gives more credence to the idea that entire limbs can be reconstructed from a small group of stem cells. Such advances could have enormous meaning for individuals who have lost body parts due to amputation or disease.

Stem Cell Roundup: Protein shows promise in treating deadliest form of breast cancer: mosquito spit primes our body for disease

Triple negative breast cancerTriple negative breast cancer is more aggressive and difficult to treat than other forms of the disease and, as a result, is more likely to spread throughout the body and to recur after treatment. Now a team at the University of Southern California have identified a protein that could help change that.

The research, published in the journal Nature Communications, showed that a protein called TAK1 allows cancer cells from the tumor to migrate to the lungs and then form new tumors which can spread throughout the body. There is already an FDA-approved drug called OXO that has been shown to block TAK1, but this does not survive in the blood so it’s hard to deliver to the lungs.

The USC team found a way of using nanoparticles, essentially a tiny delivery system, to take OXO and carry it to the lungs to attack the cancer cells and stop them spreading.

triple_negative_breast_cancer_particle_graphic-768x651In a news release Min Yu, the principal investigator on the team, said that although this has only been tested in mice the results are encouraging:

“For patients with triple-negative breast cancer, systemic chemotherapies are largely ineffective and highly toxic. So, nanoparticles are a promising approach for delivering more targeted treatments, such as OXO, to stop the deadly process of metastasis.”

Mosquito spit and your immune system

Mosquito

Mosquito bite: Photo courtesy National Academy of Sciences

Anyone who has ever been bitten by a mosquito knows that it can be itchy and irritable for hours afterwards. But now scientists say the impact of that bite can last for much longer, days in fact, and even help prime your body for disease.

The scientists say that every time a mosquito bites you they inject saliva into the bite to keep the blood flowing freely. But that saliva also has an impact on your immune system, leaving it more vulnerable to diseases like malaria.

OK, so that’s fascinating, and really quite disgusting, but what does it have to do with stem cells? Well, researchers at the National Institute of Health’s (NIH) Malaria and Vector Research Laboratory in Phnom Penh, Cambodia engrafted human stem cells into mice to study the problem.

They found that mice with the human stem cells developed more severe symptoms of dengue fever if they were bitten by a mosquito than if they were just injected with dengue fever.

In an article in Popular Science Jessica Manning, an infectious disease expert at the NIH, said previously we had no idea that mosquito spit had such a big impact on us:

“The virus present in that mosquito’s saliva, it’s like a Trojan horse. Your body is distracted by the saliva [and] having an allergic reaction when really it should be having an antiviral reaction and fighting against the virus. Your body is unwittingly helping the virus establish infection because your immune system is sending in new waves of cells that this virus is able to infect.”

The good news is that if we can develop a vaccine against the saliva we may be able to protect people against malaria, dengue fever, Zika and other mosquito-borne diseases.

A scalable, clinic-friendly recipe for converting skin cells to muscle cells

Way back in 1987, about two decades before Shinya Yamanaka would go on to identify four proteins that can reprogram skin cells into induced pluripotent stem cells (iPSCs), Harold Weintraub’s lab identified the first “master control” protein, MyoD, which can directly convert a skin cell into a muscle cell. Though MyoD opened up new approaches for teasing out the molecular mechanisms of a cell’s identity, it did not produce therapeutic paths for replacing muscle damaged by disease and injury.

That’s because MyoD-generated muscle cells are not amenable to a clinical setting. For a cell therapy to be viable, you need to manufacture large amounts of your product to treat many people. But these MyoD cells do not grow well enough to be effective to serve as a cell replacement therapy. Generating iPSC-derived muscle cells provides the potential of overcoming this limitation but the capacity of the embryonic stem cell-like iPSC for unlimited growth carries a risk of forming tumors after the transplanting iPSC-derived cell therapies into the muscle.

169572_web

This image shows iMPCs stained for markers of muscle stem, progenitor and differentiated cells. iMPCs recapitulate muscle differentiation in a dish. Credit: Ori Bar-Nur and Mattia Gerli

A recent study in Stem Cell Reports, by Konrad Hochedlinger’s lab at Massachusetts General Hospital and the Harvard Stem Cell Institute, may provide a work around. The team came up with a recipe that calls for the temporary activation of MyoD in mouse skin cells, along with the addition of three molecules that boost cell reprogramming. The result? Cells they dubbed induced myogenic progenitor cells, or iMPCs, that can make self-sustaining copies of themselves and can be scaled up for manufacturing purposes. On top of that, they show that these iMPCs carry the hallmarks of muscle stem cells and generate muscle fibers when transplanted into mice with leg injuries without signs of tumor formation.

A lot of work still remains to be done, like confirming that these iMPCs truly have the same characteristics as muscle stem cells. But if everything pans out, the potential applications for people suffering from various muscle disorders and injuries is very exciting, as co-first author Mattia FM Gerli, PhD points out in a press release:

in7czFjH_400x400

Mattia FM Gerli, PhD

“Patient-specific iMPCs could be used for personalized medicine by treating patients with their own genetically matched cells. If disease-causing mutations are known, as is the case in many muscular dystrophies, one could in principle repair the mutation in iMPCs prior to transplantation of the corrected cells back into the patient.”

Using biological “codes” to generate neurons in a dish

BrainWavesInvestigators at the Scripps Research Institute are making brain waves in the field of neuroscience. Until now, neuroscience research has largely relied on a variety of animal models to understand the complexities of various brain or neuronal diseases. While beneficial for many reasons, animal models do not always allow scientists to understand the precise mechanism of neuronal dysfunction, and studies done in animals can often be difficult to translate to humans. The work done by Kristin Baldwin’s group, however, is revolutionizing this field by trying to re-create this complexity in a dish.

One of the primary hurdles that scientists have had to overcome in studying neuronal diseases, is the impressive diversity of neuronal cell types that exist. The exact number of neuronal subtypes is unknown, but scientists estimate the number to be in the hundreds.

While neurons have many similarities, such as the ability to receive and send information via chemical cues, they are also distinctly specialized. For example, some neurons are involved in sensing the external environment, whereas others may be involved in helping our muscles move. Effective medical treatment for neuronal diseases is contingent on scientists being able to understand how and why specific neuronal subtypes do not function properly.

In a study in the journal Nature, partially funded by CIRM, the scientists used pairs of transcription factors (proteins that affect gene expression and cell identity), to turn skin stem cells into neurons. These cells both physically looked like neurons and exhibited characteristic neuronal properties, such as action potential generation (the ability to conduct electrical impulses). Surprisingly, the team also found that they were able to generate neurons that had unique and specialized features based on the transcription factors pairs used.

The ability to create neuronal diversity using this method indicates that this protocol could be used to recapitulate neuronal diversity outside of the body. In a press release, Dr. Baldwin states:

KristinBaldwin

Kristin Baldwin, PhD

“Now we can be better genome detectives. Building up a database of these codes [transcription factors] and the types of neurons they produce can help us directly link genomic studies of human brain disease to a molecular understanding of what goes wrong with neurons, which is the key to finding and targeting treatments.”

These findings provide an exciting and promising tool to more effectively study the complexities of neuronal disease. The investigators of this study have made their results available on a free platform called BioGPS in the hopes that multiple labs will delve into the wealth of information they have opened up. Hopefully, this system will lead to more rapid drug discovery for disease like autism and Alzheimer’s

Breaking down barriers to advance stem cell therapies – the view from the Vatican conference

Perry and the Pope

Pope Francis meets Katy Perry at the Unite to Cure conference at the Vatican

All hands were on deck at the “Unite to Cure” conference, organized by the Cura Foundation and the Vatican Pontifical Council,  and held at the Vatican on April 26-28. Religious leaders, scientists, physicians, philanthropists, industry leaders, government, academic leaders and members of the entertainment industry gathered to discuss how to improve human health and to increase access to relief of suffering for the under-served around the world.

Pope Francis spoke of “the great strides made by scientific research in discovering and making available new cures” but stressed that science also needs to have “an increased awareness of our ethical responsibility towards humanity and the environment in which we live.”

He talked of the importance of addressing the needs of children and young people, of helping the marginalized and those with rare, autoimmune and neurodegenerative diseases. He said:

“The problem of human suffering challenges us to create new means of interaction between individuals and institutions, breaking down barriers and working together to enhance patient care.”

So, it was appropriate that breaking down barriers and improving collaboration was the theme of a panel discussion featuring CIRM’s President and CEO, Maria Millan. She had been invited to attend the conference and participate on a panel focusing on “Public Private Partnerships to Accelerate Discoveries”.

As Dr. Millan put it, “Collaboration, communication, and alignment” is the winning formula for public/private partnerships.

She highlighted how CIRM exemplifies this new approach, how everything we do is focused on accelerating the field and that means partnering with the National Institutes of Health and the Food and Drug Administration to create new regulatory models. It also means working with scientists every step of the way; helping them prepare the best possible application for CIRM funding and, if they are approved, giving them the support they need to help them succeed.

It was a wide ranging, thoughtful, engaging conversation with David J. Mazzo, PhD, President & CEO of Caladrius Biosciences and David  Pearce, PhD, Executive VP for Research at Sanford Health. You can watch the discussion here.

People may find it surprising that government agencies, academic researchers and private companies can all collaborate effectively.  It is absolutely critical to do so in order to rapidly and safely advance transformative stem cell, gene and regenerative medicine to patients with unmet medical needs.  Pope Francis and the Pontifical Council at the Vatican certainly believe that collaboration is essential and the “Unite to Cure” Conference was a powerful demonstration of how important it is to work together for the future of humanity.

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.

Gianna McMillan

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!”

 

 

CIRM applauds FDA crackdown on stem cell clinics that “peddle unapproved treatments.”

FDA

CIRM is commending the US Food and Drug Administration (FDA) for its action against two stem cell clinics offering unapproved therapies.

On Wednesday, the FDA filed two complaints in federal court seeking a permanent injunction against California Stem Cell Treatment Center Inc. and US Stem Cell Clinic LLC. of Sunrise, Florida. The FDA says the clinics are marketing stem cell products without FDA approval and are not complying with current good manufacturing practice requirements.

“We strongly support the FDA’s strong stance to seek judicial action to stop these  clinics from marketing unproven therapies that pose a threat to the safety of patients” says Maria T. Millan, M.D., CIRM’s President and CEO. “We agree with FDA Commissioner Dr. Scott Gottlieb’s statement that these ‘bad actors leverage the scientific promise of this field to peddle unapproved treatments that put patients’ health at risk.’”

In his statement yesterday, Dr. Gottlieb denounced the clinics saying they are exploiting patients and causing some of them “serious and permanent harm.”

“In the two cases filed today, the clinics and their leadership have continued to disregard the law and more importantly, patient safety. We cannot allow unproven products that exploit the hope of patients and their loved ones. We support sound, scientific research and regulation of cell-based regenerative medicine, and the FDA has advanced a comprehensive policy framework to promote the approval of regenerative medicine products. But at the same time, the FDA will continue to take enforcement actions against clinics that abuse the trust of patients and endanger their health.”

At CIRM, we believe it is critically important for participants in stem cell treatments to be fully informed about the nature of the therapy they are receiving, including whether it is approved by the FDA. Last year we partnered with California State Senator Ed Hernandez to pass Senate Bill No. 512, which required all clinics offering unproven stem cell therapies to post notices warning patients they were getting a therapy that was not approved by the FDA.

The Stem Cell Agency has taken several other actions to protect people seeking legitimate stem cell therapies.

  • All the clinical trials we consider for funding must already have an active Investigational New Drug (IND) status with the FDA and go through a rigorous scientific review by leading experts.
  • All CIRM-funded trials must adhere to strict regulatory standards and safety monitoring.
  • We have created the CIRM Alpha Stem Cell Clinics, a network of six top California medical centers that specialize in delivering patient-centered stem cell clinical trials that meet the highest standards of care and research.
  • CIRM provides access to information on all the clinical trials it supports.

“Through its funding mechanism, active partnership and infrastructure programs, CIRM has shepherded 48 FDA regulated, scientifically sound, rigorously reviewed promising stem cell and regenerative medicine projects into clinical trials,” says Dr. Millan. “Some of these treatment protocols have already started to show preliminary signs of benefit for debilitating and life-threatening disorders. We are committed to doing all we can, in partnership with patients, the research community and with the FDA, to develop transformative treatments for patients with unmet medical needs while adhering to the highest standards to protect the health and safety of patients and the public.”

To help people make informed decisions we have created an infographic and video that detail the information people need to know, and the questions they should ask, before they agree to participate in a clinical trial or get a stem cell therapy.

 

 

‘Ask The Expert’ on Facebook Live about the power of stem cells to reverse damage caused by a stroke.

facebook-live-brand-awarenessIt’s not often you get a chance to ask a world class stem cell expert a question about their work, and how it might help you or someone you love. But on Thursday, May 31 you can do just that.

CIRM is hosting a special ‘Ask the Expert’ event on Facebook Live. The topic is Strokes and Stem Cells. Just head over to our Facebook Page on May 31st from noon till 1pm PST to experience it live. You can also re-watch the event any time after the broadcast has ended from our Facebook videos page.

Steinberg

We will be joined by Dr. Gary Steinberg, chair of neurosurgery at Stanford University, who will talk to us about his work in helping reverse the damage caused by a stroke, even for people who experienced a brain attack several years ago.

CIRM Senior Science Officer, Dr. Lila Collins, will talk about other stem cell research targeting stroke, its promise and some of the problems that still need to be overcome.

You will have a chance to ask questions of both our experts, either live on the day or by sending us questions in advance at info@cirm.ca.gov.

We’ll post reminders on Facebook so make sure to follow us. But for now, mark the date and time on your diary and please feel free to share this information with anyone you think might be interested.

It promises to be a fascinating event.