FDA Approves ‘Living Drug’ for Treatment of Aggressive Blood Cancer

Thanks to a new treatment approved by the Food and Drug Administration, adult patients diagnosed with non-Hodgkin’s lymphoma — an aggressive form of cancer of blood and lymph nodes — have new hope.

Sylvester Comprehensive Cancer Center, part of the University of Miami Health System, is one of a relatively small number of institutions nationwide that will initially be certified to administer this breakthrough immunotherapy to recurrent lymphoma patients.

The innovative treatment (axicabtagene ciloleucel) genetically engineers a type of white blood cell (T cell) taken from the patients’ own body. Millions of these cells are removed from the patient and sent to the Kite Pharma lab where they are reprogrammed to become a “living drug” that attacks lymphoma cells.

It will be sold under the brand name Yescarta, but it is only cleared for use on patients whose lymphoma has failed to respond or relapsed after receiving at least two prior different forms of treatment. Standard treatments for the disease include chemotherapy and blood and marrow transplants, which may still be needed in addition to this new therapy for some patients.

Sylvester played an important role in conducting the clinical study that led to FDA approval. According to published interim results, 39 percent of the 111 patients who participated nationwide achieved a complete remission, representing an important demonstration of the efficacy of the newer cellular therapies against previously untreatable cancers.

“The therapy offers an exciting option for patients in whom standard therapy has failed,” said Dr. Lazaros J. Lekakis, an expert in stem cell transplantation and cellular therapy who led the trial at Sylvester.

Promising cure comes with risks

This is an exciting new frontier in cancer therapy that provides hope for patients where there was none but some may experience side effects that can be severe or even fatal, explains Dr. Krishna Komanduri, director of the UM Stem Cell transplant and Cellular Therapy program and president of the American Society for Blood and Marrow Transplantation.

Axicabtagene ciloleucel can cause two potentially dangerous reactions. The most common, Cytokine?release syndrome (CRS), is caused by an activated immune system that goes into overdrive leading to symptoms that can include fever and mimic serious infections. The other more serious reaction is CAR-T-cell-related encephalopathy syndrome (CRES), which is characterized by difficulty speaking and other more serious impacts on the brain and nervous system, which can in rare cases be fatal.

“That is why these emerging therapies should be delivered in academic centers highly experienced in cellular therapies who have experts that understand how to anticipate and manage these side effects,” said Dr. Komanduri, who monitored the participants in the trial and also co-authored a paper on how to assess and mitigate the risks associated with the treatment.

More therapies on the horizon

Sylvester is running several other trials using genetically engineered T cells in hematologic malignancies such as mantle cell lymphoma, follicular lymphoma, and acute lymphoblastic leukemia, as well as in solid tumors, such as ovarian cancer, sarcoma and non-small cell lung cancer. A similar treatment was approved by the FDA earlier this year for children and young adults with B-cell acute lymphoblastic leukemia, and Sylvester expects to be able to offer this therapy sometime in 2018.

“CAR-T and other cellular therapies represent an important step forward in our ability to target cancer cells, which will have broad applicability,” said Dr. Joseph  Rosenblatt, chief of the hematology-oncology at Sylvester.

Tags: axicabtagene ciloleucel, Joseph D. Rosenblatt, Kite Pharma, Krishna Komanduri, Lazaros J. Lekakis, living drug, Miller School of Medicine, Sylvester Comprehensive Cancer Center, University of Miami, Yescarta