FDA approves first-ever gene therapy treatment for acute lymphoblastic leukemia

The logo of Swiss drugmaker Novartis AG is seen at its headquarters in Basel Switzerland

Some 83 percent of those given the therapy were in remission within three months of treatment, the agency said. "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 newly approved drug, Kymriah, is a CAR-T cell treatment developed by Novartis and the University of Pennsylvania. Tisagenlecleucel (Kymriah) is a personalized genetically-modified autologous T cell immunotherapy created to use a patient's own T cells to target and kill leukemia cells with CD19 antigens on the surface, according to a press release.

In its announcement, Novartis said it plans to make additional filings for Kymriah in the US and EU this year, including applications with the FDA and European Medicines Agency for the treatment of adult patients with r/r diffuse large B-cell lymphoma. It is caused by overactive genetically modified immune cells.

While promising, CAR-T treatments won't be like other drugs that win FDA approval, which can quickly wind up on pharmacy shelves and hospitals. However, there were some severe side effects observed in study.

Kymriah comes with a boxed warning for neurological events and cytokine release syndrome (CRS), which can both be life-threatening.

In this type of therapy, patients' own immune cells are modified ex vivo to include a gene for the vehicle protein, which directs them to attack leukemia cells carrying the CD19 antigen. The T-cells are collected then sent to a manufacturing center where they are genetically modified before being infused back into the patient. Other possible severe side effects could include serious infection, low blood pressure, kidney injury, fever and decreased oxygen.

At the time, American Cancer Society Deputy Chief Medical Officer Dr. Len Lichtenfeld said the real promise of these gene therapies is the potential to completely eradicate specific cancers both for the single patient as well as all their descendants.

A spokeswoman for Switzerland-based Novartis declined to say how much the drug will cost, though analysts have estimated a price of $500,000 or more. Researchers hope that this will lead to further CAR-T treatments that can fight more complicated cancers such as pancreatic, breast, and brain cancer.

For more on childhood acute lymphoblastic leukemia, visit the U.S.

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