First Gene Therapy in the U.S. Approved for Childhood Leukemia

For decades, medical researchers have taken small, mostly unsuccessful steps toward making gene therapies a reality. But true progress was made Tuesday when the Food and Drug Administration approved an immunotherapy approach to help children and young adults hardest hit by B-cell acute lymphoblastic leukemia (ALL).

Tisagenlecleucel, from Novartis, has now become the first gene therapy offered in the United States. It will be sold under the brand name Kymriah.

Tisagenlecleucel is called a CAR-T cell immunotherapy. Immune cells known as T-cells are removed from a patient’s blood, the cells’ genes are altered in a laboratory, and the new super cells are placed back inside the patient’s blood to begin killing off leukemia cells. Research for the therapy was led by the University of Pennsylvania.

“This is truly a breakthrough in cancer therapy,” said Dr. Stephen Nimer, director of Sylvester Comprehensive Cancer Center, part of the University of Miami Health System.

According to Dr. Edward D. Ziga, principal investigator for the pediatric Kite Pharma study at Sylvester, such therapies harness a patient’s own immune cells to effectively detect and kill leukemia cells that continue to wreak havoc despite treatment with current cancer-fighting medications.

Europe’s first gene therapy, Glybera, was approved in 2012 for treating an extremely rare blood disorder. But its maker, UniQure, removed it from the European market this April, citing high costs and low demand.

“Immunotherapy approaches like Novartis’s newly approved treatment also have great potential for other cancers,” said Ziga. “Success with more patients and further studies could pave the way for additional gene therapies targeted at other types of cancer.”

B-cell ALL is a blood cancer characterized by the formation of many lymphoblasts (immature white blood cells) in the bone marrow and blood, which then crowd out the healthy cells. The result is that a patient’s ability to fight infection and disease is compromised. While ALL can be highly curable, some patients’ cancers fail to respond to standard treatments such as chemotherapy and others recur even after bone marrow transplant. Some patients relapse after initial treatment success, and then don’t respond to additional treatments.

“This is not a replacement for standard of care therapies for patients whose bodies respond to those,” added Ziga. “It is a remarkable new choice for the patients who previously had no other options, a life-saving advance in the treatment of relapsed and refractory ALL that offers new hope.”

The new gene therapy requires close monitoring of patients to manage side effects. The median duration of cancer remission provided by Tisagenlecleucel is not yet known.

For additional information about leukemia, lymphoma and other cancers, or to schedule a consultation, visit Sylvester Comprehensive Cancer Center online, or call 877-243-1056. For more information on the clinical trial at Sylvester, please call 305-243-7648.

Tags: cancer treatment, CAR-T, Dr. Edward D. Ziga, Dr. Stephen Nimer, gene therapy, immunotherapy, leukemia, Sylvester Comprehensive Cancer Center, Tisagenlecleuce