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 the stem cell transplant that could save their lives, a small pilot study has found. The study results have been published in the journal Science Translational Medicine.
The new approach to battling T-cell prolymphocytic leukemia combines immunotherapy - boosting the body's immune system - with epigenetics, the manipulation of gene activity. It's a cutting-edge combination that holds great promise not just for treating T-cell prolymphocytic leukemia but, possibly, many other cancers as well.
The pilot study looked at eight patients with T-cell prolymphocytic leukemia, an aggressive cancer that is extremely difficult to treat. It's 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. 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. (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 four months but survived 34. 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, meaning doctors could, in theory, administer the treatment without further testing. The researchers, however, believe there needs to be additional study, hopefully in a larger trial, but the rarity of the disease makes recruiting subjects difficult.
Based on material originally posted by University of Virginia Health System.