First Gene Therapy For Leukemia Approved By FDA


Until recently, the use of auto T-cell therapy has been restricted to small clinical trials, largely in patients with advanced blood cancers.

The country's first approved gene therapy-approved Wednesday to fight leukemia that resists standard therapies-will cost $475,000 for a one-time treatment, its manufacturer announced. "As a breakthrough immunocellular therapy for children and young adults who desperately need new options, Kymriah truly embodies our mission to discover new ways to improve patient outcomes and the way cancer is treated". The lab will preform genetic engineering using a virus and will have the cells multiply before freezing them and shipping back to the hospital, where they will be given to the patient via an IV.

"This approval will open the floodgates for these kinds of therapy to be used in many different leukemias, lymphomas, solid tumors, myelomas", Dr. Prakash Satwani, a pediatric hematologist-oncologist at Columbia University Medical Center, told Business Insider.

The particular cancer in question is a blood and bone marrow cancer which is most common cancer which occurs in children in the United States.

Kymriah is expected to be available through a network of certified treatment centers throughout the United States.

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The price may be high for auto T-cell therapy, but it is important to weigh the costs against the potential benefits for patients, Gwen Nichols, MD, chief medical officer for the Leukemia & Lymphoma Society, said in an interview with OncLive.

The treatment, called CAR-T, is made by the drug company Novartis.

David Mitchell, president of Patients For Affordable Drugs, said: "While Novartis's decision to set a price at $475,000 per treatment may be seen by some as restraint, we believe it is excessive". Salvage therapies for children with relapsed ALL are highly toxic and only work in about 50 percent of relapsed patients.

CTL019 was first tested at Penn in 2010, in adult patients with advanced chronic lymphocytic leukemia (CLL).

USA health officials on Wednesday approved a breakthrough treatment for children and young adults with leukemia. "There are also over 800 cell therapy clinical trials now underway, and a considerable pool of research and pre-clinical work right across the cell therapy sector", said Bruce Levine, PhD, ISCT Commercialization and Immuno & Gene Therapy Committees, Barbara and Edward Netter Professor in Cancer Gene Therapy, University of Pennsylvania Perelman School of Medicine. Kymriah will have a boxed warning for cytokine release syndrome, a potentially lethal systemic response to the activation and proliferation of CAR-T cells, causing high fever and potential for neurological problems. Eighteen percent of patients experienced grade 3 or grade 4 neurologic events.

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This of course results in a very expensive drug price.

There are other severe side effects that can pop up as well that can require hospitalization.

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. Penn and Novartis are also investigating the next generation of vehicle therapies for multiple myeloma, and for solid tumors, through trials in glioblastoma, mesothelioma, and ovarian and pancreatic cancer.

The Novartis-Penn Center for Advanced Cellular Therapeutics (CACT) opened in 2016 and hosted Vice President Joe Biden at the launch of his Cancer Moonshot initiative, cementing Penn's role as an global innovator in the development and manufacturing of personalized cellular therapies.

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