‘Highly revolutionary’: Newly FDA-approved gene modification has promise in cancer treatment

WASHINGTON — A gene-modification process recently approved by the Food and Drug Administration has trailblazing implications: The process is curing cases of childhood leukemia by altering children’s immune system T cells so they can recognize and kill cancer cells.

“The efficacy rate in this pediatric cancer population with acute leukemia is an unbelievable greater than 90 percent response rate,” said Dr. Catherine Bollard, the director of the Center for Cancer and Immunology Research at Children’s National, of the treatment.

“It’s highly revolutionary,” Bollard said as a member of the FDA committee that recommends cancer drug approvals.

Revolutionary because what Bollard and the panel approved isn’t a drug.

Catherine Bollard, M.D., M.B. Ch.B., leads T-cell therapy research as the director of the Center for Cancer and Immunology Research at Children’s National Health System. (Courtesy Children’s National)

“It’s a living biologic. It’s enhancing your own immune system to do something it’s never been trained to do before,” she said.

The CAR-T cell treatment, developed by Novartis with multiple groups in the U.S. and internationally, is initially available to about 30 children’s hospitals.

Here’s how it works: A patient donates blood that’s shipped to the cell factory that genetically modifies the cells so they can kill cancer, then the cells get shipped back and injected into the patient.

Being able to train a person’s immune cells to attack specific ailments is a game changer.

“This will pave the way for other new exciting therapies … to engineer these immune system cells to not only kill cancer but to kill virus infections such as HIV and also as a way to overcome autoimmune diseases,” Bollard said.

“The opportunities are just limitless as we go forward now.”

Children’s National will join the institutions nationwide where the therapy is available in the near future. It also offers many other types of T-cell treatments including T-cell therapies for children with life threatening viral infections, blood cancers and solid tumors.

“This is really a very exciting time for being able to treat some of our most difficult diseases that afflict mankind,” Bollard said.

Editor’s Note: This story has been updated to clarify when CAR-T cell therapies will be available at Children’s National.

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