“If the only tool you have is a hammer…”
On September 12-13, 2016, the FDA held a public hearing intended to solicit feedback on four FDA draft guidances concerning cells, tissues, and cellular-and-tissue-based products (HCT/Ps). During the second day of the hearing, Professor Jeanne Loring, a stem cell researcher at the Scripps Research Institute, addressed the subject of applying a scientific rationale for cell therapies. She also noted the lack of evidence and scientific rationale behind widespread use of “adipose cell therapy” to treat a range of disorders.
Dr. Loring is an advocate of evidence based cellular therapies and has played an important role in drawing attention to the phenomenon of “stem cell tourism” and clinics marketing unproven and unapproved “stem cell” interventions. With her permission, Professor Loring’s statement at the hearing is reproduced below. Complete video recordings of Day One and Day Two of the hearing are now available on the NIH website. A report by Alexandra Ossola provides insight into some of the issues debated at the hearing.
Statement by Jeanne Loring
My name is Jeanne Loring. I’m a stem cell researcher at the Scripps Research Institute in La Jolla California. But today I am speaking only for myself.
I want to speak to just one issue: applying a scientific rationale for cell therapies.
Most of the speakers have been concerned with arguing that the FDA should have less oversight over the use of adipose tissue and amnion for transplantation. Unfortunately, lack of understanding or deliberate ignorance of regulations has led to increasing exploitation of desperate patients by incompetent clinics. The FDA needs to take action to improve regulation and I favor approval of the guidelines proposed. I do wish it would happen sooner.
- I want to bring up a completely different idea about cell therapy that I want to make sure that people know about. Adipose cell therapy is governed by that much-overused axiom:if the only tool you have is a hammer, you will treat everything as if it were a nail.
It isn’t logical or scientific to assume that all disorders can be treated with a single type of cell therapy.
- There is another approach, the use of pluripotent stem cells, guided by defining the disease and developing the cell therapy to treat it. Pluripotent stem cells can be made by reprogramming any person’s skin cells…they only exist in culture dishes. They can make every cell type in the body, and they are currently being used in clinical trials. They’ve been differentiated into retinal pigment cells to treat macular degeneration, into glial cells to treat spinal cord injury, and insulin-producing pancreatic cells for Type I diabetes. Cell replacement therapy is designed to be a one time treatment. My group is working on a cell therapy for Parkinson’s disease. We’ve made induced pluripotent stem cells from Parkinson’s patients, differentiated them into the precise neuron type that is lost in the disease, and we’re working toward obtaining FDA approval to transplant them back to the same patients.
But this approach, in which we rely on scientific evidence to design the tools to treat each disease, is novel, and currently has a complicated pathway to the clinic. As Randy Mills said yesterday, there must be a way to redirect the FDA’s efforts so that they can efficiently identify the cell therapies that are safest and most effective for patients and make them available as quickly as is reasonable.