Engineering Immune Cells to Treat Cancer — CAR T Cells

Engineering Immune Cells to Treat Cancer — CAR T Cells

Ronald Pratt
September 1, 2017

The Food and Drug Administration (FDA) has called this a historic moment because it is considered to be the first gene therapy hitting the US markets.

The US Food and Drug administration (FDA) has announced the approval of the first cell-based gene therapy, something that is has described as a "historic action".

"Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease", said Peter Marks, M.D., Ph.D., director of the FDA's Center for Biologics Evaluation and Research (CBER).

It has already been used to treat children dying from leukemia, who are now cancer free.

The treatment reprograms a patient's own cells to destroy cancer, and then multiply to continue fighting disease for months or even years. "Novartis should not get credit for bringing a $475,000 drug to market and claiming they could have charged people a lot more". Stat News reported on Wednesday that Novartis' head of oncology, Bruno Strigini, said in a conference call that the $475,000 price for Kymriah was an attempt to "balance patient access to the drug with ensuring a return on the company's investment".

There are other severe side effects that can pop up as well that can require hospitalization. The modified cells are then transfused back. In trials, the therapy achieved remission in 83% of patients.

FDA Approves First 'Living Drug' for Childhood Leukemia
Vague Professor of Immunotherapy , director of the Center for Cellular Immunotherapies in Penn's Perelman School of Medicine. The arrangement would see Novartis provide the drug free of charge if patients did not respond to treatment within a month.

Epstein, the former CEO of Novartis' pharmaceuticals division, left in 2016 and now serves as executive chairman at Rubius Therapeutics, a biotech that's also working with cell therapy to develop treatments like Kymriah that don't have to be as personalized.

"There has been an urgent need for novel treatment options that improve outcomes for patients with relapsed or refractory B-cell precursor ALL", Novartis, the drug company that makes Kymriah, said in a statement.

The approval of Novartis' CAR-T therapy comes just two days after Gilead Sciences Inc. announced an agreement to spend $11.9 billion and buy Kite Pharma Inc., which is developing its own CAR-T therapy scheduled for an FDA ruling by November 29. The treatment carries a boxed warning for cytokine release syndrome (CRS) and for neurological events.

Most patients with ALL recover through other treatments such as radiation, chemotherapy and stem cells. For others, Kymriah serves as a "bridge to transplant", keeping them alive longer enough to undergo this therapy. About 3100 people under age 21 are diagnosed with ALL each year in the United States, though most respond to standard therapy.

Health officials in the United States have approved a revolutionary treatment that uses genetic engineering to combat childhood leukemia. Instead of getting a better, updated therapy for a disease every decade or so, we might begin to see second-generation cell therapies in a few years. CAR-T's side effects can be deadly. And a year ago, another company, Juno Therapeutics, said five people in its clinical trials had died, all from cerebral edema. Penn and Novartis are also investigating the next generation of auto therapies for multiple myeloma, and for solid tumors, through trials in glioblastoma, mesothelioma, and ovarian and pancreatic cancer.