A small pilot study has shown that a new experimental treatment approach for a rare, deadly form of leukemia can put the disease into remission. Remission was even seen in patients for whom the standard therapy has failed, buying them more time to have the stem cell transplant that could save their lives.

Thomas P. Loughran Jr., M.D., director of the University of Virginia Cancer Center and one of the leaders of the study, said:

“It was unbelievable, really, seeing a patient who had already failed Campath [the drug typically used to treat the disease] literally going back into remission. We were able to get every single patient back into remission."

The pilot study involved eight patients with T-cell prolymphocytic leukemia, an aggressive cancer that is extremely difficult to treat. It is also extremely rare, appearing usually in older men.

The new method for battling T-cell prolymphocytic leukemia fuses immunotherapy (boosting the body’s immune system) with epigenetics, the manipulation of gene activity. This cutting-edge combination holds great promise not just for treating T-cell prolymphocytic leukemia but, potentially many other cancers as well.

“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,” Loughran said. “This is proof of principle that this might be true."

Although this experimental approach did not cure the patients, it did send them all into remission.

And it worked repeatedly. Patients could be re-treated and receive the same benefit, providing vital time as they looked for a suitable bone marrow/stem cell donor. This is important, because to receive a transplant for this disease, patients must first be in remission.

The approach does, however, have it’s limitations. Growing toxicity limits the number of times the treatment can be administered, and the suppression of the immune system can lead to infections and other complications.

Still, this treatment made a significant difference for all the study participants. One patient was expected to live only four months but survived 34. Three others were still alive at the time the researchers were compiling the trial results.

Zainul S. Hasanali, Bikramajit Singh Saroya, August Stuart, Sara Shimko, Juanita Evans, Mithun Vinod Shah, Kamal Sharma, Violetta V. Leshchenko, Samir Parekh, Thomas P. Loughran Jr., and Elliot M. Epner9 [Epigenetic therapy overcomes treatment resistance in T cell prolymphocytic leukemia](http://Science Translational Medicine 24 Jun 2015: Vol. 7, Issue 293, pp. 293ra102 DOI: 10.1126/scitranslmed.aaa5079) Science Translational Medicine 24 Jun 2015: Vol. 7, Issue 293, pp. 293ra102 DOI: 10.1126/scitranslmed.aaa5079

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