In this interview, Massimo Dominici, ISCT past president, comments on the FDA’s recent statement on ‘unproven’ cell therapies.
In this interview, Massimo Dominici, International Society for Cellular Therapy (ISCT; BC, Canada) past president and advisory board chair, comments on the FDA Commissioner’s statement on ‘unproven’ cell therapies.
Massimo Dominici is professor of medical oncology at the University Hospital of Modena & Reggio Emilia – Modena Policlinic Modena (Italy), was President of ISCT in 2016 and now chairs the ISCT advisory board. He also heads the ISCT Presidential Task Force (PTF) on the Use of Unproven Cellular Therapies set up after the ISCT’s annual meeting in Auckland, New Zealand (22-25 April, 2013).
What prompted you to get more involved in the area of cell therapy regulation?
I decided to get more involved in this area because, in addition to my medical activities and research in stem cells and gene therapy for cancer, I was involved by the Italian government in the Stamina Foundation affair, where stem cells were injected into patients without any scientific evidence. I was employed by the Italian Minister of Health to analyze the cells which were injected. After that experience, I felt, in the age of social media, it was very important to increase awareness in the scientific community and inform the public about unproven cell therapies. Thus, inside ISCT and in collaboration with others, we started sharing thoughts regarding how we can face the unproven cell therapy problem but without any intention to be a police force or a substitute for a regulatory body.
We need to try and create a global awareness. This is a problem that is not happening in exotic countries or faraway places; it is happening next door. It happened in my own country, in Germany, in China and it has been happening in the United States. The impression is that regulatory bodies have been struggling to identify and remove these clinics. The ISCT is very glad about this action taken by the FDA; it’s about time and is providing a warning message for extra-USA situations too. However, I think more has to be done at the global level, possibly at the World Health Organization level, with the contribution of scientific societies together with patient associations.
Why is this statement important?
There has been a lot of development in the field of cell therapy with product approval over the last year, with the FDA announcing this week the approval of the Novartis CAR-T therapy, which is good news. It is helpful to know that the FDA wants to facilitate the approval of cell therapy products that are effective and at the same time will not tolerate products that are useless or even harmful.
The 21st Century Cures Act in the US was somehow thought to have a more tolerant approach to not completely proven cell therapies so, considering that, it is a bit surprising to have this statement. However, this stance from the FDA is certainly reassuring and we hope that it is a trend that will be maintained in the other agencies in the different regions.
The existence of clinics where cells are delivered to patients in a paid-to-be-treated approach without scientific evidence is really damaging innovation in the field. There is still a need to generate novel therapies for lethal diseases and this may require specific regulatory acceleration paths; however, we should only be delivering cells that are supported by pre-clinical data and pushed forward within first-in-human and/or phase I clinical studies. Challenging brutal medical conditions without science only takes advantage of the patients and their poor clinical situations without any specific medical follow-up. It’s then only about the market and not about the science and innovation any more.
How could this statement affect patients’ ‘right-to-try’ and the rise of unproven clinics on ClinicalTrials.gov?
The statement from the FDA is certainly a clear voice which we did not hear from before and this issue has been never so big. I think both the victories for the ‘right-to-try’ and the rise of pay-to-participate clinical trials on the web, and even on ClinicalTrials.gov, were taking advantage of the silence from the regulatory bodies. In the marketing of these unproven treatments, the clinics were aware that although the regulations were in place, essentially the regulatory bodies were silent.
In the near future we may be seeing the creation of a taskforce within the FDA and other bodies to check more carefully what is going on and have a more stringent way to control these centers. Without controls, there is no way to get rid of those clinics where the combination of no scientific evidence and a “disinformed” consent is also creating psychological and economical damage to the affected individuals and their families. It’s a matter of identifying those places which is not a problem because this information is publically available. We would be better off going there and verifying what’s going on.
What prompted your letter to the FDA?
We wanted to say to the FDA that the scientific community supports regulators, underlining that ISCT and others are also active on this topic. The regulators are generally seen as people who tell us how to do things, so the scientific community felt the need to say we agree with your statement. We think that implementing a more rigid approach to control these centers will certainly be of benefit in the future. It’s a simple message and we’d be very happy to collaborate with the FDA and other bodies in supporting them to addressing the complexities of unproven cell therapies.