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.

How small talk led to a big break; a summer internship at CIRM

At CIRM, California’s Stem Cell Agency, we are fortunate to work with some amazing people. This summer we added another name that list when Melissa Cairos joined us for an internship. Melissa is now on to the next part of her adventure, as a policy wonk in Washington DC., but before she left we asked her to write about her experiences, and thoughts after her time at the Stem Cell Agency.

Melissa

Melissa Cairos

In January of 2018, I had a casual conversation with a woman, whom I had never met before, at a high school basketball game. Through small talk about my studies in school and my career interests for the future, the woman suggested I may be interested in her work because it seemed to be aligned with what I wanted to do. Her work happened to be at CIRM and she happened to be Maria Millan, the President and CEO.

Interestingly, I had never heard of CIRM (the California Institute for Regenerative Medicine) and had limited knowledge of regenerative medicine. But, I had dedicated a semester in spring of 2015 to analyzing and lobbying for the 21st Century Cures Act. I engaged in that work because I believe in the importance of investing in, and expediting the regulatory process for, lifesaving medical innovations, so that they can be accessed faster by patients and at a lower cost. The 21st Century Cures Act has since become law and has created incredible opportunities for both CIRM and the entire field of regenerative medicine.

Since joining CIRM, I have been able to continue with similar work by analyzing legislation, policies and regulations that affect patients’ abilities to access regenerative medicine therapies and our grantees’ abilities to receive reimbursement for their therapies. Because the stem cell and gene therapies CIRM’s grantees are coming up with are so new and innovative, I quickly realized that the legislative, policy and regulatory solutions for them needed to reflect that innovative spirit.

Working alongside Geoff Lomax, (the Senior Officer for CIRM Strategic Infrastructures)  my manager and mentor, we identified a number of potential barriers to access and reimbursement and tried to come up with policy solutions to address them.

For one project, we looked at the high cost of regenerative medicine therapies. Because high cost affects both patient access and potential reimbursement problems for the companies that develop those therapies we felt it was essential to try and come up with policy solutions to address these issues. To do this, we studied the traditional payment structure for drugs and medical devices and found it inappropriate for regenerative medicine in most cases.

This is because regenerative medicine requires a one-time high cost payment, but the regenerative medicine treatments/cures would eliminate long term costs including: previous treatment cost, complications from that treatment, progression of disease cost, hospitalizations, disability, quality of life, co-morbidities, disease effect on longevity etc. Thus, we proposed that payment models for regenerative medicine should consider their unique value benefits, such as the number of additional years of life the treatment added, and the overall cost-effectiveness of a one-time treatment compared to years of  treatment. With this in mind, we suggested innovative payment models that accounted for these factors and further proposed changes that need to be made so that different manufacturers and payors can engage in innovative financing agreements.

Through my work at CIRM, I found that what makes regenerative medicine unique is that it not only offers new ways of treating previously untreatable diseases, but it has additional benefits or value. Not only the economic value, but also the human value, as regenerative medicine offers patients with life threatening or painful chronic diseases a solution that will change their lives and the lives of their families for the better. Through this understanding, I grew an incredible appreciation for CIRM, for not only being a great place to work with incredibly talented and kind people, but also an incredibly unique government agency that reflected the value and innovative spirit of the research it supports.

I am so grateful that I met Maria at that basketball game and got the opportunity to support CIRM in adding value to California in my role this summer as a Policy Fellow. I plan to return to California in the future and work in the health policy field to further support programs, policies, and/or agencies, like CIRM, that bring so much value to this state.

 

 

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.

Stem-Cell-Clinics-to-Trust

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.

Stem Cell Stories that Caught Our Eye: New law to protect consumers; using skin to monitor blood sugar; and a win for the good guys

Hernendez

State Senator Ed Hernandez

New law targets stem cell clinics that offer therapies not approved by the FDA

For some time now CIRM and others around California have been warning consumers about the risks involved in going to clinics that offer stem cell therapies that have not been tested in a clinical trial or approved by the U.S. Food and Drug Administration (FDA) for use in patients.

Now a new California law, authored by State Senator Ed Hernandez (D-West Covina) attempts to address that issue. It will require medical clinics whose stem cell treatments are not FDA approved, to post notices and provide handouts to patients warning them about the potential risk.

In a news release Sen. Hernandez said he hopes the new law, SB 512, will protect consumers from early-stage, unproven experimental therapies:

“There are currently over 100 medical offices in California providing non-FDA approved stem cell treatments. Patients spend thousands of dollars on these treatments, but are totally unaware of potential risks and dangerous side effects.”

Sen. Hernandez’s staffer Bao-Ngoc Nguyen crafted the bill, with help from CIRM Board Vice Chair Sen. Art Torres, Geoff Lomax and UC Davis researcher Paul Knoepfler, to ensure it targeted only clinics offering non-FDA approved therapies and not those offering FDA-sanctioned clinical trials.

For example the bill would not affect CIRM’s Alpha Stem Cell Clinic Network because all the therapies offered there have been given the green light by the FDA to work with patients.

Blood_Glucose_Testing 

Using your own skin as a blood glucose monitor

One of the many things that people with diabetes hate is the constant need to monitor their blood sugar level. Usually that involves a finger prick to get a drop of blood. It’s simple but not much fun. Attempts to develop non-invasive monitors have been tried but with limited success.

Now researchers at the University of Chicago have come up with another alternative, using the person’s own skin to measure their blood glucose level.

Xiaoyang Wu and his team accomplished this feat in mice by first creating new skin from stem cells. Then, using the gene-editing tool CRISPR, they added in a protein that sticks to sugar molecules and another protein that acts as a fluorescent marker. The hope was that the when the protein sticks to sugar in the blood it would change shape and emit fluorescence which could indicate if blood glucose levels were too high, too low, or just right.

The team then grafted the skin cells back onto the mouse. When those mice were left hungry for a while then given a big dose of sugar, the skin “sensors” reacted within 30 seconds.

The researchers say they are now exploring ways that their findings, published on the website bioRxiv, could be duplicated in people.

While they are doing that, we are supporting ViaCytes attempt to develop a device that doesn’t just monitor blood sugar levels but also delivers insulin when needed. You can read about our recent award to ViaCyte here.

Deepak

Dr. Deepak Srivastava

Stem Cell Champion, CIRM grantee, and all-round-nice guy named President of Gladstone Institutes

I don’t think it would shock anyone to know that there are a few prima donnas in the world of stem cell research. Happily, Dr. Deepak Srivastava is not one of them, which makes it such a delight to hear that he has been appointed as the next President of the Gladstone Institutes in San Francisco.

Deepak is a gifted scientist – which is why we have funded his work – a terrific communicator and a really lovely fella; straight forward and down to earth.

In a news release announcing his appointment – his term starts January 1 next year – Deepak said he is honored to succeed the current President, Sandy Williams:

“I joined Gladstone in 2005 because of its unique ability to leverage diverse basic science approaches through teams of scientists focused on achieving scientific breakthroughs for mankind’s most devastating diseases. I look forward to continue shaping this innovative approach to overcome human disease.”

We wish him great success in his new role.

 

 

 

How do you know if they really know what they’re saying “yes” to?

How can you not love something titled “Money, Mischief and Science.” It just smacks of intrigue and high stakes.

And when the rest of the title is “What Have We Learned About Doing Stem Cell Research?” you have an altogether intriguing topic for a panel discussion.

Sue and Bill Gross Hall: Photo by Hoang Xuan Pham/ UC Irvine

Sue and Bill Gross Hall: Photo by Hoang Xuan Pham/ UC Irvine

That panel – featuring CIRM’s own Dr. Geoff Lomax, a regular contributor to The Stem Cellar – is just one element in a day-long event at the University of California, Irvine this Friday, November 13.

Super Symposium

The 2015 Stem Cell Symposium: “The Challenge of Informed Consent in Times of Controversy” looks at some of the problems researchers, companies, institutions and organizations face when trying to put together a clinical trial.

In many cases the individuals who want to sign up for a clinical trial involving the use of stem cells are facing life-threatening diseases or problems. Often they have tried every other option available and this trial may be their last hope. So how can you ensure that they fully understand the risks involved in signing up for a trial?

Equally important is that many of the trials now underway now are Phase 1 trials. The main goal of this kind of trial is to show that the therapy is safe and so the number of cells they use is often too small to have any obvious benefit to the patient. So how can you explain that to a patient who may chose to ignore your caveats and focus instead on the hope, distant as it may be, that this could help them?

Challenging questions

The symposium will feature experts in the fields of science, law, technology and ethics as they consider:

  • Does informed consent convey different meanings depending on who invokes the term?
  • When do we know that consent is informed?
  • What are human research subjects entitled to know before, during and after agreeing to participate in clinical trials?
  • How might the pushback on fetal tissue research impact the scientific development of vaccines, research on Alzheimer’s disease or other medical advancements?

So if you are looking for something thought provoking and engaging to do this Friday, here you are:

“The Challenge of Informed Consent in Times of Controversy,” Friday, Nov. 13, 9am – 4:30pm, at the Sue & Bill Gross Stem Cell Research Center on the University of California, Irvine campus.

The symposium will be livestreamed, and a video recording will be available on www.law.uci.edu following the event.

REGISTER: The symposium is free to UCI student, staff and faculty. There is a $20 registration fee for non-UCI attendees. Visit the event page to register.