First Gene Therapy Passes FDA Approval

On Wednesday, the Food and Drug Administration approved a revolutionary gene therapy that utilizes an individual’s blood cells that have been genetically engineered to attack and eradicate cancer cells.

 

Thirty-two medical institutions, including Johns Hopkins Hospital, will train its physicians to administer Kymria, developed by Novartis Pharmaceuticals, to cancer patients.

The FDA approved the gene therapy for patients up to the age of 25, who have been diagnosed with acute lymphoblastic leukemia. To qualify for the gene therapy, the patient must have exhausted all their treatment options. Between 15 percent and 20 percent of people with acute lymphoblastic leukemia have exhausted all their options, according to the FDA.

Novartis Kymria

Acute lymphoblastic leukemia, a quickly progressing cancer that targets the blood and bone marrow, is the most common childhood cancer in the United States. According to The National Cancer Institute, approximately 3,100 new cases are reported each year, involving children and young adults up to age 20.

The expand the use of CAR-T gene therapy, researchers at the University of Maryland Medical Center’s Marlene and Steward Greenebaum Comprehensive Cancer Center are conducting a clinical trial, focusing primarily on treatments for adults diagnosed with leukemia.

Earlier this year, Lentigen Technology acquired a cancer treatment startup, known as Living Pharma, which originated from the University of Maryland School of Medicine. The cancer treatment utilizes the patient’s T-cells to attack and eradicate malignant tumors.

The CAR-T gene therapy is the first of its kind to be approved in the United States. Kymria, a “living drug” that utilizes genetically modified T-cells from individual patients to attack and eradicate their own cancer.

A multi-site study was conducted to determine the effectiveness of CAR-T therapy in patients who desperately ill. The results were astonishing, as 83 percent of the 63 patients who participated in the study went into remission, but some relapsed only a few months later.

The downside to the treatment is it potentially causes severe and life-threatening side effects, according to the FDA. There is also a risk of developing cytokine release syndrome, an immune reaction that causes hypotension, severe organ damage and high fevers. To avoid the immune overreaction, patients must be monitored very closely.

Customized CAR-T therapy will cost $475,000 each. If the cancer does not respond to the therapy, Novartis said it would not charge the patient for the treatment.

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