FDA Approves First 'Living Drug' for Childhood Leukemia

FDA Approves First 'Living Drug' for Childhood Leukemia

Ronald Pratt
September 1, 2017

Despite these negatives, FDA approval of gene therapy is a big step for the treatment both in the U.S. and around the globe. The U.S. Food and Drug Administration issued a historic action today making the first gene therapy available in the United States, ushering in a new approach to the treatment of cancer and other serious and life-threatening diseases.

The price may be high for auto T-cell therapy, but it is important to weigh the costs against the potential benefits for patients, Gwen Nichols, MD, chief medical officer for the Leukemia & Lymphoma Society, said in an interview with OncLive. Novartis estimates about 600 patients a year would be eligible for the treatment, which belongs to a class of drugs known as auto T-cell therapies. Cells are removed from the patient, modified to target cancer cells and expanded outside the body, and then re-infused into the patient.

Novartis estimates some 600 ALL patients a year would be eligible for Kymriah.

The arrangement would see Novartis provide the drug free of charge if patients did not respond to treatment within a month.

There are other severe side effects that can pop up as well that can require hospitalization. Auto T-cell therapies can damage healthy immune cells, including the cells that produce the antibodies that fight disease.

Those early-stage clinical trials, in which more than 90 percent of patients achieved a complete remission one month after receiving the therapy, led to a global registration trial in 2015, involving 68 children and young adults with advanced ALL treated at 25 centers across the world. This also applies to those patients of this particular leukaemia who have suffered a relapse of the cancer.

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The treatment process, known as auto T-cell therapy, will be used to fight the recurrence of the B-cell where cancer has not responded to treatment or returned after initial treatment, something that occurs in approximately 15-20% of patients.

"This therapy is a significant step forward in individualised cancer treatment that may have a tremendous impact on patients' lives", said Dr Carl June, the Richard W. Vague Professor of Immunotherapy, director of the Center for Cellular Immunotherapies in Penn's Perelman School of Medicine.

Dana-Farber/Boston Children's has recently participated in a similar clinical trial of vehicle T-cells - led by Silverman and pediatric oncologist Steven Margossian, MD, PhD - that evaluated the efficacy of auto T-cell therapy (short for chimeric antigen receptor T-cell therapy) in treating relapsed or treatment-resistant B-cell ALL.

"Patients who get this therapy basically have no chance to survive", he said.

"In the next few years", he said, "I think we're going to see dramatic progress and push the boundaries of what many people thought was possible with these adoptive cell transfer-based treatments".

For one, the treatments would have to treat more types of cancer than the one Kymriah was approved for on Wednesday. "Through our collaboration with Novartis, we are creating the next wave of immunocellular cancer treatments, and are eager to progress CAR-T therapy in a host of haematologic and other cancer types". The patient was a little girl nearly dying and is now five years cancer free.