CAR-T for Transformed FL Gets Priority Review from FDA

The FDA has granted a faster-than-usual review for yet another Follicular Lymphoma treatment. This one is for KTE-C19, a type of CAR-T therapy, and it is for transformed Follicular Lymphoma, as well as Diffuse Large B Cell Lymphoma (DLBCL) and Primary Mediastinal B Cell Lymphoma (PMBCL).

(We're taking a quick break from ASCO Previews for this one. The review is based on results that were presented at a different convention -- the AACR last month. The results come from the phase 2 ZUMA-1 trial, though a few other different aspects of that trial will be discussed at ASCO.)

CAR-T therapies, in general, involve removing T cells from the patient. T cells are part of the immune system, and their job is to find invaders (like cancer cells) and get rid of them. The problem is, T cells can't recognize cancer cells. So after the T cells are removed, they are changed so they will recognize the cancer cells, and then they are put back into the patient so they can do their job.

Now, KTE-C19 works the same way. It gets its name from its target: the protein called CD19, which appears on the surface of the cancer cells being targeted (and lots of other cells). When those new, changed T cells find the cancer cells, they destroy them, the way they were supposed to.

The Zuma-1 trial looked at two groups of patients, one of them including transformed FL patients. Out of 101 total patients, 24 of them had transformed FL or another type of lymphoma. In that group, the Overall Response rate was 83%, and the Complete Response rate was 71%. After 8.7 months, the OR rate remained high -- 67%, and the CR stayed at 61%.

Patients in the trial were chemorefractory, meaning chemotherapy was no longer working for them. They had had a median of 3 treatments before the trial.

Side effects included things like anemia, decreased white blood cell counts of different types. Several patients had Cytokine Release Syndrome, which occurs when the body is overwhelmed with an immune response. There were 4 fatalities during the trial, 3 of them related to the treatment. Most of the  non-fatal side effects were reversed within a month.

Based on these results, the FDA is granting Priority Review (as it did with Copanlisib earlier this month).  This means that the FDA plans to take action on approval within 6 months, and that it considers the treatment important enough (and doing something that other treatments aren't doing) to speed it along a few months early.

If this is approved, it will give us another tool for transformed Follicular Lymphoma -- we can never have too many.

CAR-T is definitely hot this year at ASCO, with trials and experiments targeting a bunch of different cancers. I know there are a few folks who read regularly who have some experience with it, and who are very excited about CAR-T. Let's hope this one continues to give us all something else to be excited about.

anemia (43%), neutropenia (39%), decreased neutrophil count (32%), febrile neutropenia (31%), decreased white blood cell count (29%), thrombocytopenia (24%), encephalopathy (21%), and decreased lymphocyte count (20%). - See more at: http://www.targetedonc.com/news/ktec19-granted-priority-review-by-fda-for-nonhodgkin-lymphoma#sthash.schjRozT.dpuf

The 8.7-month ORR rate was 67 percent, with a CR rate of 63 percent. - See more at: http://www.curetoday.com/articles/agent-granted-priority-review-to-treat-nonhodgkin-lymphoma#sthash.V9EN38YB.dpuf