First Gene Therapy for Leukemia Approved by FDA

On Wednesday, the U.S. Food and Drug Administration took what the federal agency called historic action with the first-ever approval of cell-based gene therapy in the U.S.

Kymriah was approved by the FDA. Scientists refer to Kymriah as a living drug as it involves using immune cells that are genetically modified from patients to attack the cancer in their bodies.

The drug received approval for treating children as well as young adults to the age of 25 that suffer from a type of acute lymphoblastic leukemia and do not respond to the standard treatment or have had relapses.

The disease is a cancer of bone marrow and blood that is the most common cancer amongst children in the U.S. Nearly 3,100 patients 20 years of age and younger are diagnosed annually with ALL.

A new frontier is being entered in medical innovation as there is now the ability to reprogram the cell of a patient to attack cancer, said Scott Gottieb the Commissioner of the FDA through a prepared written statement.

New technologies like cell and gene therapies hold the potential to transform today’s medicine and to create a new inflection point in the ability of doctors to treat and at times cure many of the intractable illnesses, added the written statement by Gottlieb.

The treatment involves the removal of cells from the immune system known as T cell from patients and then genetically modifying them in a laboratory to attack and kill the leukemia cells.

These cells that are genetically modified are infused back into the cancer patients. The therapy is also referred to as CAR-T cell therapy.

The treatment known as well as CTL019 produced a remission in under three months in 83% of the 63 pediatric and younger adult patients. Those patients had not responded to the standard form of treatment or had gone through relapses.

Based upon those results, an advisory panel of the FDA recommended the therapy’s approval last month.

However, the treatment does have its risks including an overreaction of dangerous proportion by the body’s immune system called cytokine-release syndrome. Because of that strong warnings are being required by the FDA.

In addition, this treatment will be available to start only in 32 clinics and hospitals where staff has been trained to administer the therapy.

Novartis, which is the developer of the drug, said the one-time procedure will have a cost of $475,000 for the patients who respond.

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