New clinical trial research demonstrates ongoing promising results for patients with the most common form of lymphoma, relapsed or refractory (r/r) diffuse large B-cell lymphoma, or DLBCL.
The clinical trial, known as JULIET, focused on long-term results for study participants receiving a single dose of tisagenlecleucel, (Kymriah®), the first cell-based gene transfer therapy available in the U.S.
Kymriah 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.
The results from this international clinical trial show that at a median of 19 months after treatment, the overall response rate was 54 percent among 99 trial participants who were followed for at least three months or discontinued therapy early; 40 percent of those participants achieved a complete response, and 16 percent achieved a partial response.
The Phase II study, co-led by 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, was recently published in The New England Journal of Medicine and updated study results were presented at the American Society of Hematology conference in San Diego.
“This study is the first to outline long-term Kymriah results for these lymphoma patients, and we’re seeing that these responses can be sustained,” says Maziarz. “There are a significant number of patients who are remaining free of disease, and none of those still in remission proceeded on to transplantation. This approach appears to offer a single treatment that can relieve symptoms and save lives for people who had otherwise faced a very poor prognosis.”
This is the first global study to examine a CAR-T therapy exclusively in people with DLBCL. Research was conducted at 27 treatment sites spanning 10 countries across North America, Europe, Australia and Asia. The OHSU Knight Cancer Institute was an early adopter and one of a handful of certified treatment centers in the nation to offer this therapy to patients with DLBCL.
Maziarz, also a professor of medicine (hematology and medical oncology) in the OHSU School of Medicine, is the senior investigator for the clinical trial and served as chair of the scientific steering committee throughout the drug’s clinical development. He says future research efforts could include determining which patients may benefit from additional therapies to maintain or improve patient responses.
“We’re changing the natural history of the disease and, as a next step, it is critical to identify subpopulations of patients who may need other therapies in addition to CAR-T,” Maziarz said. “This research is really the foundation for the next wave of studies to assess combination therapies.”
Kymriah is the only CAR-T therapy that has been FDA-approved for two distinct indications – patients with r/r DLBCL and pediatric and young adult patients with r/r B-cell acute lymphoblastic leukemia, or ALL. Last year, OHSU Doernbecher Children’s Hospital was the first hospital in the Pacific Northwest to offer this treatment to pediatric and young adult patients with ALL.
“When we send people home after the course of treatment, most patients can anticipate long-term results,” Maziarz says. “We’re at the forefront of something new.”
Learn more about the trial from the American Society of Hematology press release.
Funding for the study was provided by Novartis. Kymriah® is a registered trademark of Novartis.
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.