Below, Dr. Steven Devine, a renowned blood and marrow transplant physician and the newest member of the Be The Match BioTherapies team, shares his thoughts on the challenges and opportunities of the cell therapy industry. Read part one.
What do you see as the biggest challenges that cell therapy companies are facing at this moment? How is Be The Match BioTherapies positioned to help them?
First and foremost, and particularly for an early stage company without an approved product, the key is to design studies that will efficiently evaluate the safety, feasibility and effectiveness of a particular cellular therapy they have developed. Without well-designed studies, a potentially beneficial therapy may never get to the point where it can help patients. Beyond that, navigating the regulatory hurdles is a critical step, as is handling all the logistics necessary to safely collect and transport cells from one place to the next without any mishaps. This is another way Be The Match BioTherapies can help. We can leverage over 30 years of experience in consistently collecting, transporting and tracking cells destined to be used in real patients, in a timely and safe manner.
Manufacturing is another big hurdle. Doing this well and in a reproducible manner is critical, as is meeting the timelines demanded by our patients and their clinical conditions. Another challenge is the globalization of the marketplace and how we deal with that. There are patients in need all over the globe and companies are stepping up to meet that challenge. However, the U.S. is still in the lead, so how do our companies get their effective therapies out to more patients around the globe who need them? We don’t want to inadvertently create a situation where disparities in access to the best therapies are created.
Where do you see the biggest opportunities for cell therapy? Any emerging disease areas?
Cellular therapies are very different from conventional drugs as they will continue to evolve. They are really living, breathing therapies that are amenable to continued modification and optimization based on data generated not only in the laboratory but from safety and efficacy signals gained from the clinical trials we perform. This adaptability is one of the key differences of cellular therapy and also one of its most interesting characteristics.
As far as opportunities, I think we may see an approval for a CAR-T cell therapy for multiple myeloma in the next year, and we will learn more about the effectiveness of this treatment for patients with chronic leukemia who have expended most of their treatment options. I also see big opportunities in solid tumors and will be very interested in the results of these trials as they come out over the next few years.
There are also great opportunities to treat other serious and debilitating non-cancer related diseases such as multiple sclerosis, scleroderma, and sickle cell disease, just to name a few.
What is most exciting to you about the cell and gene therapy space?
The recent approval of a gene therapy for a form of hereditary blindness was a very exciting milestone and I believe we are on the cusp of being able to effectively treat other serious genetic disorders like sickle cell disease and immune deficiency syndromes through gene therapy. The technology is catching up with the complexity of these diseases, so I think we will see significant advances in gene therapy over the next decade.
There are a lot of opportunities to team up with several pioneering organizations and I hope I can help Be The Match BioTherapies evaluate and communicate the best opportunities to make progress and improve the lives of more patients by leveraging the many assets of our organization.