The U.S. Food and Drug Administration has approved the use of gene therapy, Kymriah, also known as CTL109 or tisagenlecleucel, for the most common form of lymphoma: diffuse large B-cell lymphoma, or DLBCL.
DLBCL is an aggressive disease that affects B-lymph cells that make antibodies to fight infections and are an important part of the body’s lymphatic system. The occurrence of DLBCL generally increases with age; most patients are older than 60 when diagnosed.
The OHSU Knight Cancer Institute is an early adopter and one of a handful of certified treatment centers in the nation to offer this therapy to patients with DLBCL. “The potential for this therapy is huge,” says Richard Maziarz, M.D., medical director of the adult blood and marrow stem cell transplant and cellular therapy program in the OHSU Knight Cancer Institute. “Whether it will be realized, that’s to be determined. But having the opportunity to offer this therapy to our patients, this early in the process, is critical. This therapy has the potential to be applied to many other diseases, including solid organ cancers.”
Kymriah, the first gene transfer therapy available in the U.S., is manufactured and marketed by Novartis. It uses chimeric antigen receptor (CAR) T-cell therapy, a form of immunotherapy in which the patient’s own blood cells are collected, genetically engineered to attack B-cell lymphoma cells, then infused back into the patient.
Maziarz, also a professor of medicine (hematology and medical oncology) in the OHSU School of Medicine, is the senior investigator for the CTL109 clinical trial that led to approval of the drug.
He also served as chair of the steering committee throughout the drug’s clinical development. He says one responsibility of his role included exploring options for how to expand and develop the clinical trials across the world.
“We spent roughly 18 months on weekly phone calls with multiple centers across the world, including: Norway, Australia, France, Italy and others,” he explains. “We were managing patients collaboratively. When someone had a problem, everyone knew about it; everyone contributed to the solution for the participants on this trial.”
Kymriah is the only CAR-T therapy FDA-approved for two distinct indications – patients with DLBCL and patients with B-cell acute lymphoblastic leukemia, or ALL. Last year, OHSU Doernbecher was the first hospital in the Pacific Northwest to offer this treatment to pediatric and young adult patients with ALL.
“One of the potential benefits of CAR-T is that we believe the expected set of risks are more controlled, more contained,” says Maziarz. “Now our focus is to limit these treatments’ associated risks, which will only enhance the clinical outcome.
“When we send people home after the course of treatment, they can anticipate long-term results. We’re at the forefront of something new.”
In the interest of ensuring the integrity of our research and as part of our commitment to public transparency, OHSU actively regulates, tracks and manages relationships that our researchers may hold with entities outside of OHSU. In regards to this research, Maziarz has received payments for his service on the Novartis steering committee. Review details of OHSU's conflict of interest program to find out more about how we manage these business relationships.