Multiple gene therapy trials report high remission rates for certain lymphoma patients

WASHINGTON, Dec. 10 (Xinhua) -- In three clinical trials conducted in multiple sites, a novel gene therapy that genetically engineers a patient's immune system to attack cancer cells demonstrated long-lasting remissions in certain lymphoma patients, researchers reported Sunday.

In a multi-center trial conducted in 10 countries, 81 patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), the most common form of non-Hodgkin lymphoma, was treated with an investigational chimeric antigen receptor (CAR) T cell therapy called Kymriah.

At three months, 26 patients, or 32 percent, achieved a complete response, while five, or six percent, achieved a partial response, according to results presented at the annual meeting of the American Society of Hematology (ASH) in Atlanta.

About 73 percent of patients who responded remained cancer-free at six months during the Novartis-sponsored trial known as JULIET.

Another single-site trial, also sponsored by Novartis, involved 28 patients who received the therapy after their cancers had come back following standard treatments.

Among these patients, 43 percent of DLBCL patients achieved complete remission, as did 71 percent of patients with follicular lymphoma, the second most common form of the disease.

All patients who were in remission at six months are still in remission, after a median follow-up of 28.6 months.

Results of the single-site trial were published in the New England Journal of Medicine.

"Taken together, our data from both trials show that most patients who are in remission at three months stay in remission," said Professor Stephen Schuster of the University of Pennsylvania, who led both studies, said in a statement.

Kymriah was approved by the U.S. Food and Drug Administration in August 2017 for the treatment of patients up to 25 years of age with acute lymphoblastic leukemia that is refractory or in second or later relapse.

A new application has been recently submitted by Novartis to the FDA for approval of Kymriah for the treatment of adult patients with relapsed or refractory DLBCL who are ineligible for or relapse after autologous stem cell transplant.

A third study involving another CAR T cell therapy called Yescarta showed that 42 percent of patients with aggressive large B-cell lymphoma remained in remission at 15 months following treatment.

The study, named ZUMA-1, began in April 2015 by administering Yescarta to 101 adult patients from 22 medical centers who had certain types of large B-cell lymphoma and had not responded to or had relapsed after undergoing at least two other treatments, including chemotherapy and stem cell transplants.

Overall, 52 percent were still alive at 18 months following therapy, with some remaining cancer free two years post-treatment.

All the patients experienced some adverse effects, with three deaths during treatment.

"We are taking a very measured approach to this new therapy, which is effective, but also potentially toxic," Patrick Stiff, director of Loyola University's Cardinal Bernardin Cancer Center, a co-author of the study, said in a statement.

"The therapy should not be considered a cure-all, since some of the patients did relapse after the therapy."

Based on results of the study, the U.S. Food and Drug Administration recently approved Yescarta, which is manufactured by Kite Pharma.

Results of the trail were also reported in the New England Journal of Medicine, and to be presented Monday at the ASH annual meeting.

CAR T cell therapy involves removing immune system cells known as T cells from each patient and genetically modifying the cells in the laboratory and then infused them back into patients to attack and kill cancer cells. The treatment is sometimes referred by scientists as "a living drug."

An accompanying editorial in the New England Journal of Medicine called these trials "a milestone" for CAR T cell therapy and said this personalized therapeutic approach is "a revolutionary treatment for patients with advanced blood cancers." Enditem