Keeping patients safe from unproven cell therapies: an interview with the ISCT Presidential Task Force on the Use of Unproven and/or Unethical Cell and Gene Therapies
In this interview, Bruce Levine, President-Elect of the International Society for Cell and Gene Therapy (ISCT) and member of the Presidential Task Force on the Use of Unproven and/or Unethical Cell and Gene Therapies, discusses the ISCT’s new patient resources.
Hear more from Bruce Levine in our video interview, filmed at ISCT 2018. Watch now>>
The ISCT recently launched their new site on unproven therapies. Why now?
Our interest as a society in helping patients navigate issues around unproven therapy dates back around a decade or so. From the 19th century to the 21st, there has been snake oil and treatments that have piggybacked on terms that are in the scientific literature and percolate into the public through the popular press, such as ‘stem cells’.
...the word out for patients is to be informed and do your research
What has happened now is that there is direct-to-consumer marketing by certain fraudulent groups of so-called ‘stem cell therapies’ that are nothing more than a transfer of adipose tissue spun down and marketed for everything from Alzheimer’s to autism. This has proliferated because the public has been deceived that these are true stem cells due to reports of genuine scientific advances. The efforts of ISCT are to make clear that there is a scientifically validated and ethical pathway and then there is the unvalidated, unethical aspect of what is out there. Our goal is to not only make that distinction but more importantly protect patients from possible harm- both medical and financial.
Why do you think it has fallen to a more academic-industry group such as the ISCT to provide this information?
As scientists and clinicians, we can speak the language of science and cell therapy. I think our goal is to interface with those patient advocacy groups and serve as a resource for them on the science in as many ways as we can. There has already been outreach to some of these groups and we look forward to more.
One of the resources this new site has is the definitive list of all the approved therapies as well as the therapies that have been taken off the market. What were you hoping to achieve by listing them?
To put this in context over the years, there have been many efforts by societies, individual scientists and clinicians and bloggers to identify unproven or rogue clinics. That’s hard for a number of reasons. The clinics are appearing, disappearing and changing their names, and some of them have sued other societies who have tried to do this. It is much cleaner and effective to say what is approved; that was the impetus behind this work and this list will be constantly updated. Any patients looking outside this list can be forewarned and forearmed.
While the FDA has moved against a few clinics...they just don’t have the resources to inspect and shut down all 520 [in the USA]
The current version is a list of therapies that have been approved as marketed therapies, so we have distinguished from so-called hospital exemption which in some countries is a legitimate way of participating in clinical trials or investigator-initiated therapies. The problem with the so-called hospital exemption is it means different things in different jurisdictions and therefore there isn’t the standard of quality that there is with formal approval by a national regulatory authority. I think we will address that in the future.
What you hope to add to this resource in the future?
Beyond that list of marketing medical authority-approved therapies, I think we will come out with some version of therapies that are under hospital exemption. We are going to continue to expand the languages in which this is offered; we already have English, Italian, Spanish, Portuguese, Korean, French, German, Hindi and Chinese. We will also continue the outreach to the patient advocacy organizations and individual patients that can serve as liaisons to the community. We have had at the ISCT annual meeting for many years a session dedicated to these effort and to ethics, which is being publicized with the help of our colleagues and journalists to get this important issue out to patients.
What are legal recourse do patients have against these bogus clinics?
I think this is going to be a continuing issue as technology develops; I imagine we are going to have clinics offering unapproved CRISPR therapies pretty soon. Unfortunately, there always will be those people so we have to stay top of it. The FDA, the Australian Therapeutic Goods Administration (TGA) and other agencies have limited resources. While the FDA has moved against a few clinics and the Federal Trade Commission has moved on at least one, they just don’t have the resources to inspect and shut down all 520 or however many there are currently in the US. Maybe there will be attorneys of good will who will be able to protect patients by engaging in legal action against some of these operators and that will serve as an additional deterrent to not adhering to already existing laws.
How are you working with other societies to further this cause?
We are working with other societies in a coordinated fashion. The ISCT is head of the FDA Cell Therapy Liaison Meeting, during which some of these issues come up, and we also participate in the US National Academies of Science, Engineering and Medicine Forum on Regenerative Medicine. At these meetings, there is participation of anywhere from 5 to 20 other professional societies, so we can coordinate and use as many channels as we have to educate and serve as a resource for patients.
As scientists and clinicians, we can speak the language of science and cell therapy
I will throw the question back to you. Are there other things that you think as a professional society that we could or should be doing to assist patients, informing them on cell and gene therapies, and the distinction between scientifically validated and unvalidated and unethical therapies?
That’s a really good question! Even as a media outlet we get questions from patients about these unproven cell therapies or potential clinical trials that they would like to take part in, so I think a resource for scientists or people like me to make sure we are sticking to the same message and being consistent in the language we are using would be helpful.
I think the word out for patients is to be informed and do your research. These operators are very slick from billboards and advertising in airline magazines to blogs and patient testimonials. Unfortunately, they convince patients to use their own money for something that is unproven and ineffective, and to engage in crowdfunding where the whole community is raising money for these types of unvalidated therapies. It destroys trust in the legitimate investigation of cell and gene therapies so there are many, many reasons why we have pursued this, and why we are continuing and enhancing our efforts.