FDA Approves First Gene Therapy in US

FDA Approves First Gene Therapy in US

Instead of helping the body's T cells better spot cancer, the CAR-T treatment enables them to fight those cancer cells better. "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".

Treatment with the therapy, developed by Swiss pharma Novartis AG, led to remarkable results in children with an aggressive form of leukemia resistant to other drugs. In the US, only a few dozen specialized hospitals are now qualified to provide CAR-T treatments, which require retrieving, processing and then returning immune cells to the patient.

This first use of CAR-T therapy is aimed at patients desperately ill with acute lymphoblastic leukaemia, which strikes more than 3,000 children and young adults in the U.S. each year.

With the treatments available now, fewer than 10 percent of patients with the relapsed, hard-to-treat leukemia are alive five years after diagnosis.

The disease is the most common type of childhood cancer in the US, making up 25 percent of cancer diagnoses for children under 15 years old, according the National Cancer Institute.

"New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses", said FDA Commissioner Scott Gottlieb. Once modified, the cells are infused back into the patient to kill the cancer cells. Tisagenlecleucel carries a boxed warning for cytokine release syndrome (CRS) and neurological events, which can be life threatening. "Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials". The drug's maker, Novartis, is required as a condition of approval to conduct a longer-term study of Kymriah's use, the agency said.

Manufacturing and delivery are more complex in CAR-T therapies than for a typical drug.

Due to these and other potential risks, the agency said it is requiring hospitals that administer the therapy to be specially certified.

"The theory is they should attack the tumor and continue to grow to become a long-term monitoring and treatment system", Lichtenfeld said.

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

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