An experimental new treatment approach for a rare, deadly leukemia can send the disease into remission even in patients for whom the standard therapy has failed, buying them more time to have a stem cell transplant that could save their lives, a small pilot study has found. These findings were published in Science Translational Medicine (2015; doi:10.1126/scitranslmed.aaa5079).

“It was unbelievable, really, seeing a patient who had already failed alemtuzumab [Campath; the drug typically used to treat the disease] literally going back into remission,” said Thomas P. Loughran Jr., MD, director of the University of Virginia Cancer Center in Charlottesville, VA, and one of the leaders of the study. “We were able to get every single patient back into remission.”

The new approach to battling T-cell prolymphocytic leukemia combines immunotherapy, which boosts the body’s immune system, with epigenetics, which is the manipulation of gene activity. It is a cutting-edge combination that holds great promise not just for treating T-cell prolymphocytic leukemia but, possibly, many other cancers as well.

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“There’s been a revolution in the last few years seeing success with immunotherapy, and people speculated that perhaps if you combined epigenetic and immunotherapy, that might be even more spectacular,” Dr. Loughran said. “This is proof of principle that this might be true.”

The pilot study, led by Dr. Loughran and Elliot Epner, MD, of the Pennsylvania State University College of Medicine in Hershey, PA, looked at eight patients with T-cell prolymphocytic leukemia, an aggressive cancer that is extremely difficult to treat. It is also extremely rare, appearing most commonly in older men.

The experimental approach did not cure the patients, but it did send them all into remission. Also, it worked repeatedly, as patients could be retreated and receive the same benefit, providing vital time as they looked for a suitable bone marrow/stem cell donor. (To receive a transplant for this disease, patients must first be in remission.)

There are limitations to the experimental approach. Mounting toxicity limits how many times the treatment can be administered, and the suppression of the immune system can lead to infections and other complications. But the treatment made a significant difference for all the study participants. One patient was expected to live only 4 months but survived 34 months. Three others were still alive at the time the researchers were compiling the trial results.

The drugs used in the treatment are already commercially available. Dr. Loughran, however, believes there needs to be additional study, hopefully in a larger trial, but the rarity of the disease makes recruiting subjects difficult.

As such, he encourages patients with the disease to consider seeking treatment at the University of Virginia. “We’d be very glad to see them here, if they want to come see us,” he said.