Big things were happening in the world of cancer research back in 2017.
The Food and Drug Administration had just approved the first gene-engineered CAR T cell therapy, and many more cell therapies would soon follow. Those approvals allowed cell therapies to benefit up to 50,000 more cancer patients a year, a huge leap from the few thousand who had been able to take advantage of similar treatments before.
The pipeline from initial testing to FDA approval had taken a mere 15 years, astonishing considering this was the first therapy of its kind. Now, two of the physician-scientists behind those early milestones – Marco Davila, M.D., Ph.D. and Renier Brentjens, M.D., Ph.D. – are leading efforts at Roswell Park Comprehensive Cancer Center to unveil the next cell therapy breakthrough, and they want it to happen even faster.
“We know this technology is powerful, and we know there’s a huge clinical need for it,” Dr. Davila says. “Roswell Park has developed resources right here that will allow us, we expect, to reduce that approval timeline down to as little as seven or eight years. Every year we shave off that timeline means more lives saved.”
Next step: solid tumor cancers
As Senior Vice President and Associate Director for Translational Research and Vice-Chair for Cellular Therapies, Dr. Davila and his team at Roswell Park are working on the critical next step: discovering a way to successfully apply these groundbreaking strategies to solid-tumor cancers. That capability would transform the way those diseases are treated and give patients an additional cancer-fighting weapon in their arsenal, this one extremely targeted and potentially quick-acting.
In some cases, Dr. Davila says, patients can literally see the difference within weeks or even days once they receive the infusion of their own immune cells, armed with stronger defenses against cancer.
“As a clinical provider, it really is inspiring and amazing to see a patient say, ‘Oh my goodness, my lymph nodes are shrinking,’” he says.
And T cell applications are just the beginning.
“This is the future of cancer treatment. We’re in step one of this immune cell gene engineering revolution,” Dr. Davila says. “It’s my expectation that in 10 years, CAR T is going to be the Ford Pinto of cell therapy; we’re going to look back at it and marvel at how far we’ve come and what better technologies we have for our patients.”
Innovation in the lab and at the bedside
Dr. Davila joined the team at Roswell Park in June 2022, drawn by the comprehensive cancer center's cutting-edge facilities.
As a National Cancer Institute-designated comprehensive cancer center, Roswell Park houses all the facilities, equipment and personnel needed to offer these highly complex treatments to cancer patients and to pioneer new strategies not available elsewhere – reflecting a level of collaboration rarely seen, even at large cancer centers.
What truly sets Roswell Park apart – and what drew Dr. Davila here in June 2022 – is that it is one of only a few cancer centers in the country to also house Good Manufacturing Practice (GMP) facilities, which are equipped to genetically reengineer human cells so they can be used for medical treatment. Stringently regulated by the FDA, they entail specially trained staff and equipment, sterilization and monitoring technology and precise atmospheric and environmental standards, among other requirements.
“If an institution has these facilities on-site, it’s an easy way to spot that they have long believed in the power of immunotherapy,” Dr. Davila says. “It takes a lot of money and years to develop GMP facilities, but Roswell Park had one in the works 15 years ago. We are five to 10 years ahead of most institutions because the leadership at Roswell Park saw the power of the cells and said, ‘We believe this therapy will save lives.’”
Cell therapy 101
What is cell therapy? It’s the use of living cells to destroy and control cancer cells. Through genetic engineering, the genes of cancer cells are edited, replaced or inhibited to stop the cancer from growing, or the genes can be added to immune system cells to help recognize and fight cancer. The first example of this type of therapy, stem cell transplantation, began being used about 50 years ago.
What are CAR T therapies? Chimeric antigen receptor T-cell (CAR T) therapies employ a gene that targets a specific type of cancer cell, inserted into a person’s T-cells (a type of white blood cell that attacks foreign substances). Once a patient’s blood is infused with the altered T-cells, the cells adapt the body’s immune response to destroy the cancer cells.
What types of cancers can CAR T therapies treat? So far, CAR T can treat some leukemias, lymphomas and multiple myelomas, but has not yet shown benefit for patients with solid-tumor cancers such as lung, breast, ovarian or prostate.
A cell therapy success story: CAR T
The groundbreaking work of Dr. Marco Davila, Dr. Renier Brentjens and their colleagues on CAR T therapies two decades ago has resulted in cancer-curing treatments for thousands of patients each year. Here’s what that journey looked like:
2001: Non-human investigations begin
Preclinical studies in the laboratory
2003: Initial results published
First evidence CAR T cells may prove effective
2006: Clinical trials begin
