Cancer Gene Therapy Gets Green Light - And $475000 Price Tag

Novartis won FDA approval for its CAR-T drug Kymriah on Wednesday

The new cells would then continue to multiply to fight disease for months or years. "New technologies such as gene and cell therapies hold out the potential to transform medicine". The Food and Drug Administration called the approval historic, this being the first gene therapy on the American market, ABC News reports.

It's a type of cancer immunotherapy, which harnesses the body's immune system to take on cancer cells.

Those reprogrammed cells are sent back to the patient and administered, at which point the new cells try to kill off the cancer cells.

The FDA's move to approve the auto T-cell therapy, which will be marketed under the name "Kymriah" by Novartis Pharmaceuticals, opens the door to a new era of cancer medicine, and medicine more broadly.

Actually, the gene therapy techniques of the CAR-T treatment procedure is not meant to fix the disease causing genes rather these make the T cells get turbocharged and hence cancer can not evade the immune system.

About 3,100 Americans ages 20 and younger are diagnosed with acute lymphoblastic leukemia each year, and 15 to 20 percent of them don't respond to the initial treatment or have their disease return, according to the National Cancer Institute.

"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 treatment is personalized for every patient and Novartis Pharmaceuticals is planning to charge 475,000$ for it.

Although the price of the therapy is high by any standard, Cooper stated that Novartis is "committed to doing everything we can to ensure that children and young adults who can benefit from [tisagenlecleucel] have access to the therapy and we are working with payers to ensure they fully understand the value of [it] and provide coverage for patients. 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.

There are other severe side effects that can pop up as well that can require hospitalization. Both CRS and neurological events can be life-threatening. And the drug comes with serious side-effects, including a reaction known as cytokine release syndrome, that can prove fatal if not controlled with immunosuppressant drugs. Right now, that's where most of the big successes have come from, but one day that could expand out to solid tumors, and maybe even auto-immune diseases like type 1 diabetes, Epstein said. Since the CD19 antigen is also present on normal B-cells, and Kymriah will also destroy those normal B cells that produce antibodies, there may be an increased risk of infections for a prolonged period of time.

"We've never seen anything like this before", said Stephan Grupp, director of the cancer immunotherapy frontier program at Children's Hospital of Philadelphia, the first medical center to study Kymriah in children.

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