The Critical Role of Patient Advocates in Accelerating Stem Cell Cures

At CIRM, our goal is to bring stem cell therapies to patients with unmet medical needs, and we do that by funding the most promising and innovative research in regenerative medicine. A critical component of this goal is to support our patient advocates and make sure that their voices are heard.

At this year’s World Stem Cell Summit, patient advocates from around the world, representing a breadth of diseases and disorders, came together to share their stories, goals, and needs with the larger scientific community.

One session that particularly stood out, was “Accelerating Cures: The Critical Role of Patient Advocates” on Day 3 of the conference. This panel featured key leaders in patient advocacy:

  • Don Reed, the “Grandfather of Stem Cell Research Advocacy”, Vice President of Public Policy at the Americans for Cures Foundation
  • Frances Saldaña, an advocate for Huntington’s disease (HD) and founder of HD-Care at UC Irvine, which is a support group to advance HD research and clinical care
  • Tory Williams, the Executive Director of the Alabama Institute of Medicine (AIM) which raises funds and awareness for stem cell treatments and cures of disease and injury and the author of “Inevitable Collision

The panel was moderated by our fearless leader and head of communications, Kevin McCormack. Each speaker shared their story about how they became a patient advocate and what they are currently doing to push the pace of stem cell research.

Don Reed, Kevin McCormack, Frances Saldana, Tory Williams.

Don Reed, Kevin McCormack, Frances Saldana, Tory Williams.

Don Reed described the heartbreaking story of his son Roman Reed, who suffered a severe spinal cord injury while playing football. Through Don and Roman’s relentless efforts, “Roman’s Law” was passed in 1999, which raised $17 million in California state funding for spinal cord injury research. Don was also a key instigator for the passage of Proposition 71, which gave $3 billion dollars to our agency to fund stem cell research. He continues to be a passionate advocate for stem cell research and spinal cord injury patients, and recently published a book called “Stem Cell Battles: Proposition 71 and Beyond” which you can read more about in our recent blog.

Next, Frances Saldana told a compelling story of raising a family of three beautiful children with a husband who had Huntington’s disease. Unaware of his condition when they were together, Frances’ world took a devastating turn when he died of HD, leaving her to question whether her children would face the same fate. Sadly, all three of Frances’s kids carried the HD mutation. Having to deal with the passing of her two daughters, and a son who is battling the end stages of this disease, Frances decided to share her experience with others and to create a support organization called HD-Care so that others wouldn’t have to face similar experiences alone. HD-Care is conducting an aggressive campaign to bring visibility to HD and supports cutting-edge research in the field including the work done by CIRM-grantee Dr. Leslie Thompson at UC Irvine.

Frances told the audience that her happiest moment since this all began was when her daughter Margie, already suffering from symptoms of HD, spoke at CIRM in 2007. She saw the Board and the scientists and thought, “somebody cares, and somebody will find a cure.” It was a new chapter for her, she explained, and she knew something good was going to happen.

Lastly, Tory Williams, introduced the Alabama Institute of Medicine, which is a non-profit organization that supports the stem cell community with education and public dialogue. She started the institute following both personal and family experiences with cancer and after TJ Atchinson, a close family friend, suffered a severe spinal cord injury. Along the way, she forged a close relationship with Roman Reed who helped her pass TJ’s law in 2013, which is an Alabama state law that promotes spinal cord injury research.

“The goal [of AIM],” said Williams, “is to make a difference in people’s lives affected by disease and injury by helping to advance medicine to eradicate these debilitating issues.”

Laurel Barchas, Student Society for Stem Cell Research

Laurel Barchas, Student Society for Stem Cell Research

When the session was opened up to questions, the atmosphere in the room turned electric. Patients and scientists stood up to tell their stories and asked hard questions. One question came from Laurel Barchas, one of the founders of the Student Society for Stem Cell Research, who asked how we as a society can advocate for mental illness and similar diseases where the symptoms are not visible and where patients are either embarrassed or hesitant to make their disease public. Another question was how emerging countries like Mexico who don’t have the same benefits and infrastructure as the US can promote and support patient advocacy.

The mood of the advocates was positive but measured. They know that new treatments and cures take time but they also pointed out that many people don’t have much time so we have to work as hard as we can to help them.

The panel ended with the consensus that the voices of patient advocates are invaluable, and that they will be the key to accelerating stem cell therapies into cures. Frances Saldaña urged other patient advocates that the key to progress is to be aggressive, and be unafraid to be out there. Don Reed concluded on a similar note with quote from Shakespeare’s Hamlet:

“Whether ’tis Nobler in the mind to suffer

The Slings and Arrows of outrageous Fortune,

Or to take Arms against a Sea of troubles,

And by opposing end them.”


Related links:

Why “Right to Try” laws are more feel good than do good

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L to R: Don Gibbons, CIRM; Jeanne Loring; Beth Roxland; Aaron Levine

In the last few years some 24 states have approved so-called “Right to Try” laws. These are intended to give terminally ill patients faster and easier access to experimental therapies. But a panel of experts at the World Stem Cell Summit in Atlanta today said they are more symbolic than anything and do little to actually help patients get much-needed therapies.

The Right to Try laws are modeled after a federal law that allows “compassionate use” of experimental medications and lets doctors prescribe investigational medicines being safely used in early stage clinical trials.

Beth Roxland, a bioethicist with Johnson & Johnson, says the name of the law is misleading:

“If you look at the actual text of these laws they only say you have the right to “ask” for these drugs. That right already exists in federal law but neither federal law nor these Right to Try laws say you have the right to access.”

Aaron Levine from Georgia Tech says it’s also misleading to assume that just because a state passes a Right to Try law that it has any legal impact. He says state laws don’t over rule the Food and Drug Administration’s (FDA) regulation of this area and so the federal government would still have the authority to stop this kind of access.

But Levine says these laws are interesting in that they are indicative of the growing determination of patients and patient advocates to work around obstacles to access and have a bigger say in their own care.

One of the audience members, William Decker from Baylor College of Medicine, says that in Texas a law was recently crafted saying that as long as a potential therapy had gone through a Phase 1 safety trial it should be offered to the public and the public should be able to pay for it.

“If you know how clinical trials work you know you can get almost any schlock through a Phase 1 trial and the kinds of things that you can get to the public without any idea if they work often turn out to not be very useful. We saw this as an avenue to promote fraud, and the last thing you should be doing to a dying patient is take their money or divert their attention away from something that might help them.”

Decker and his colleagues argued before the Texas Legislature that potential therapies should at least have to go through a Phase 2 trial to make sure they were not only safe but also showed some benefit for patients. In the end Texas lawmakers rejected the Phase 2 idea but did say patients could not be charged for the therapy, and there could be no compensation from insurers or anyone else for the manufacturer of the therapy.

He says removing the financial benefits and incentives pretty much ensured that no company would offer patients a therapy under this law.

Jeanne Loring, a CIRM grantee from the Scripps Research Institute, says that likely won’t stop other clinics in other states:

“Some stem cell clinics are using adipose (stem cells derived from fat) therapy as an option for every disease imaginable and I’m sure some will take advantage of these laws to say it gives them the right to offer these to patients and the patients will pay for them directly. “

Roxland says that may already be happening:

“I think there is some evidence on the stem cell side that companies have popped up in states that have these laws, to make it easier to offer their therapies to patients.”

The panel agreed that in most cases these laws don’t give patients any rights they don’t already have, but do give the appearance of making access easier. They said it’s feel-good legislation, allowing people to feel they are doing something without actually doing anything.

Aaron Levine said that while some companies may try to take advantage of these laws, the most serious ones won’t:

 “Almost any legitimate company that wants an FDA approved product wouldn’t want to take advantage of these laws. It could put their product at risk. Most companies that need to work with the FDA have no incentive to go this route.”

 

 

To modify, or not to modify: Experts discuss human germline modification at WSCS15

The question of whether human germline modification, or the genetic modification of human reproductive cells, should be allowed or banned was discussed by a panel of experts in the Ethics, Law and Society session during Day 1 of the World Stem Cell Summit.

On the panel were Aubrey de Grey, Chief Science Officer of the SENS Foundation, Paul Knoepfler, Associate Professor at the UC Davis school of medicine (and a CIRM grantee), and Aaron Levine, Associate Professor of Public Policy at Georgia Tech.

Aubrey de Grey, Paul Knoepfler, Aaron Levine

Aubrey de Grey, Paul Knoepfler, Aaron Levine

What Paul Knoepfler said…

On the basic research side, Paul discussed how CRISPR has revolutionized the way germline modification is being done from the older, costly, time-consuming method using homologous recombination to the faster, more efficient, and cheaper gene editing technology that is CRISPR.

In the big picture, he said that, “people will pursue germline modification with a variety of different goals.” He further explained that because this will likely happen in the future, scientists need to consider the risks (off target effects to name one) and the societal and ethical impacts of this technology. Another question he said we should consider is, whether as a society, we support the modification of the germline for health or enhancement reasons.

He concluded with a recap of last week’s International Summit on Human Gene Editing saying that while the organizers didn’t put forth a definitive statement on whether there should be a moratorium on editing the human germline, he himself believes that there should be a temporary moratorium on the clinical use of this technology since the idea is still very controversial and there is no overall consensus within the scientific community.

What Aubrey de Grey said…

Aubrey began by saying that as a gerontologist, he is interested in all potential therapies that could postpone the effects of old age, many of which could involve genetic modification. He went on to say that it might not seem intuitive that editing the human germline would be applicable to fighting aging, but that:

“Even though the medical imperative to engaging genetic germline modification may seem to be less clear in the case of aging than it is for inherited diseases, which people are unequivocally agreed on that is a bad thing, never-the-less, the potential application to aging may actually play a significant role in the debate, because we’ve all got aging.”

He gave an example of the ApoE4 gene. If you have two copies of this form of the gene instead of the normal ApoE3 gene, then you have a very high risk of getting Alzheimer’s disease and atherosclerosis. He posed the question to the audience, asking them whether if they knew that they had this disease causing gene, would they consider genetically altering their fertilized eggs back into the safe ApoE3 version to prevent their offspring from inheriting disease even if the therapy wasn’t approved by the FDA. It’s a hard question to answer and Aubrey further commented that if we begin using genetic modification to prevent one disease, where would we draw the line and where would modification end?

He ended with saying that the real question we need to consider is “whether people will want to do germline modification against aging, even though the modifications may really be more in the way of enhancements than genuine therapies.”

What Aaron Levine said…

Aaron Levine began with saying that the question of human germline modification is an old question with new twists. By new twists he meant the recent advances in gene editing technologies like CRISPR and Zinc Finger Nucleases. He further commented that the baseline question of this debate is whether we should modify the DNA of the germline, and that how we do it isn’t as significant.

He played devil’s advocate by saying that germline editing would greatly benefit single gene disorders, but that we should think of the full spectrum. Many traits that we might want, we don’t know enough about and attempting to add or remove these traits using gene editing would be like shooting in the dark.

On policy side, Aaron commented that international policy harmonization would be nice, but that we should treat it skeptically. He said that not everyone is going to agree or follow the same rules and we need to consider this going forward. As for the FDA, he said that its role and regulations regarding germline editing aren’t clear and that these need to be defined.

One really interesting point he made was the issue of unproven stem cell clinics. They exist and pose a huge risk to human health. The real question, he said, is could this turn into unproven CRISPR clinics around the world? He ended with saying that someone will claim to offer this technology soon and asked what we should do about it.

From the peanut gallery…

One of the questions asked by the audience was whether it’s just a matter of time that one of the world’s governments might go forward with human germline modification because of the huge medical implications.

Paul responded first saying that there was a consensus at the gene editing summit that it’s more of a question of when, rather than if this would happen. Aaron agreed and said that he believed it would happen but wasn’t sure when, and followed with saying that the more important question is how it will be done, overseen, and what reasons the editing will be done for.

Bernie Siegel, who is the co-Chair of the World Stem Cell Summit, spoke at the end and said that the panel delivered exactly what he hoped it would. He emphasized a theme that I didn’t mention in this blog but that was brought up by each of the panelists: the voice of patients.

“One of the things missing from the [International Summit on Gene Editing] meeting was the voice of the patient community. Do they understand the concepts of CRISPR technology? Patients are a major stake holder group, and they have the most influence on creating change in policy. When we talk about a moratorium, the patients see it as a five-alarm fire. All they want is to see a few drips of water, and they can’t get it. From a societal and popular culture standpoint, these are a whole group of people that will be experiencing the sweeping changes of biotech today. When those voices that are receiving these technologies enter the conversation, it will be a full debate.”

The bottom line: stem cell therapies will never be widely available if insurers won’t pay for them

The second session of the World Stem Cell Summit in Atlanta moved past all the promising science and right to the nitty-gritty of making cell-based therapies common. Four panelists reminded the audience that while they too are super excited by the potential for this field, unless folks developing therapies think about reimbursement early those therapies will not become a reality in routine clinical care.

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“Stem cell therapies seem unstoppable with seemingly limitless possibilities, but success requires early planning for reimbursement,” said moderator Michael Levinson, a lawyer and physician with the law firm Hogan Lovells.

Elizabeth Powers of the IMS Consulting group suggested the audience pay close attention to the cancer market.  She said insurers and other payers of health care services are tired of paying for “statistically significant” improvements in survival that only translate to a few weeks on average. She said payers are moving away from just whether a new therapy is different from prior therapies and want to be shown true value.

A further reminder to start the reimbursement process early came from panelist Deborah Dean of MiMedx.  She said the process of just applying for a reimbursement code takes two years and after that it can take months or more to then present your case to insurers to turn that code into actual payments.

During the question period there was a bit of potential good news attached to an industry trend I did not expect. The consolidation of insurers, with two major mergers on deck, could actually extend the average length of time a customer is with an insurer from between two to five years to between five to ten years. This may make insurers more willing to pay for a one-time curative therapy that is expensive but eliminates chronic therapy costs.

Call to Action by FDA at World Stem Cell Summit

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FDA Deputy Commissioner Dr. Robert Califf talking at the World Stem Cell Summit

The World Stem Cell Summit annual conference in Atlanta kicked off today with a clarion call from Dr. Robert Califf, the Deputy Commissioner for the Food and Drug Administration. He told the audience:

“We want you to accelerate translation to produce safe and effective therapies that can be delivered reliably”

It was a message that everyone in the room, scientists and patient advocates, would love to be able to comply with. The question of course is how do you do that in a way that puts the emphasis on both speed, to get the therapies to patients who need them, and safety, so you don’t put those patients at risk.

That’s quite a challenge considering that, as panel moderator Julie Allickson of Wake Forest Institute for Regenerative Medicine said:

“the estimate now is it costs $2.4 billion and up to ten years to take something to the clinic.”

Even if that dollar amount is higher than many think it would take to bring a stem cell therapy to a clinical trial it is an indication of the challenge the field faces.

Califf, who has only been at the FDA for 8 months, says that regenerative medicine is:

“not the only field exploding with scientific knowledge and seeing a future that’s very different from what we see today so it’s exciting but also an enormous challenge for the FDA. One of the real eye openers for me is to be at the FDA and hear about drugs that have been on the market for 45 years and we’re still learning about them.”

He says the first goal of the FDA has to be to protect the public, and that it’s hard to balance safety and innovation. “That’s an issue we struggle with every day.”

Califf was optimistic that the balance can be struck and progress can be made, but said that this can only truly be done if the patient is at the table as an active participant.

“Our national clinical research system is well intention but flawed. We need to have a new system that shares information right across the system and where patients are at the center. Patients should be driving the national research infrastructure. They are an essential part of change. It’s happening in Congress because they are hearing from constituents that this is what they want, a voice in the research being done that affects them.”

For the patients and patient advocates in the audience it was a welcome message. For years they have been calling for a louder voice in the research that affects them and their loved ones. Knowing they have a sympathetic ear in the FDA could be an encouraging sign that their voices are finally being heard.

We will be writing more as the conference unfolds so stay tuned!