Also called the Roswell Park GEM, the facility is the largest academic cell and gene therapy research and manufacturing facility in the U.S.
While the center’s focus will be on developing cell and gene therapies to treat solid-tumor cancers, it will be building off of the scientific research and discoveries that brought chimeric antigen receptor T-cell therapy to the forefront of oncology care.
Leading the way is Renier Brentjens, MD, PhD, deputy director, chair in the department of medicine and the Katherine Anne Gioia Endowed Chair in Cancer Medicine at Roswell Park.
One of the pioneers of CAR T-cell therapy, Dr. Brentjens spoke with Becker’s about what the new facility means for cancer innovation, as well as on how leaders across the U.S. can best integrate CAR T therapy at their organizations.
Editor’s note: Responses have been lightly edited for clarity and length.
Question: Can you expand a bit on CAR T-cell therapy, what it means for Roswell Park’s new facility and the future of cancer care?
Dr. Renier Brentjens: We actually came up with the name CAR T-cell back in the early 2000s at Memorial Sloan Kettering, where we developed the second, commercially available CAR T-cell product designed for patients with acute lymphoblastic leukemia. That CAR T-cell product, and facsimiles thereof, are being used to treat different types of leukemia, lymphoma, and more recently, multiple myeloma today.
After 23 years at Sloan Kettering, I came back to Buffalo, where I grew up, to work at Roswell Park. At the time, we had six GMP rooms, rooms that met good manufacturing practice regulation standards. I was kind of brought in to expand the cell therapy program in general, but with an emphasis on what I know best, CAR T-cell therapy. We now have 20 GMP rooms at Roswell Park, the most in the country and probably in the world if I had to bet — but we haven’t verified that.
The obvious question is, “What are you going to do with 20 rooms?” The plan we’ve initiated is to utilize around 30% to 40% of that facility for technology that’s been developed here at Roswell Park, as we have an ever-growing body of researchers that are developing next-generation CAR T cells, especially focused on treating solid tumor malignancies. For the other 60% to 70%, we will collaborate with smaller biotech companies and enable them to run trials.
When I ran the first trial at Sloan Kettering, it took four years to get from when the therapy was finalized to actually treating the first patient. Now we can cut that timeline down to 12 to 18 months. Once we gain momentum, we can probably get that down even more.
Whether we make the products ourselves or whether we’re working with biotechs that make their own products, ultimately the goal is to have a research portfolio where we have some type of cell therapy to offer virtually any patient. That’s an ambitious goal, but given what we’ve accomplished over the first three years it’s eminently realistic.
Q: How can hospital and health system leaders prepare their own organizations for the incoming wave of new cancer therapies like CAR T?
RB: Hospital systems that want to get involved in CAR T-cell infusion need to recruit people who have been trained in cell therapies because it has its own list of side effects and ways to manage these patients. A hospital system would initially have to invest in recruitment of faculty who have expertise in the field of administering CAR T-cells.
I actually would envision, in say, 10 years’ time, that cell therapy will become kind of its own subspecialty, where the expertise isn’t in a particular type of cancer, but in selecting the best cell product, infusing it, and managing patients post-infusion.
This is not just adding another drug to your pharmacy. This type of therapy, as good as it is, requires expertise on multiple levels, and hospital systems need to be prepared to do it very thoughtfully. It does require an investment of time and, as with anything, money as well.
Q: We’ve just reported on the patient who has been cancer-free for 19 years after being treated with engineered CAR-T cells. Do milestones such as this one change the narrative around what it means to be diagnosed with cancer?
RB: I remember when we were getting the first group of patients into remission, no one was allowed to use the cure word. I think by any metric, certainly with leukemias and lymphomas, we can now look back and say that we’ve actually cured people of cancer.
With that in mind, there are a lot of obstacles that we still face. In the lab we are working on translating this work from blood cancers — where CAR T is FDA-approved — to solid tumors, which are much more common, but the biology and the immunology is different.
Would this patient be an outlier? Probably. But if we knew then what we know now, we would probably be able to find out why she did so well.
Her success emphasizes the need to keep doing more cancer research. Innovation doesn’t come from the companies that are selling the CAR T cells, it comes from academic research. For us to continue to move forward, it requires that we continue to get financial support to do this type of innovation. These people are alive today because of NIH funding to academic research institutions, which is where the innovation and novelty come from.
