New hope: how an experimental pill cured aggressive cancer in trial patients

An experimental pill stopped a very aggressive type of leukemia in a third of the patients participating in a groundbreaking trial. The promising treatment is still under investigation but has shown satisfying results.

According to the Memorial Sloan Kettering Cancer Center (MSK), one of the institutions involved, the trial enrolled 68 patients at nine hospitals in the US. All of them had seen their leukemia come back after other treatments or had never responded well to traditional chemotherapy drugs.

All the patients suffered from a very aggressive type of leukemia called acute myeloid leukemia. A mutation or rearrangement of a gene fuels this particular form of cancer. According to the Spanish newspaper El Pais, it is the most common form of adult leukemia.

Acute myeloid leukemia attacks the bone marrow, where blood cells are produced, and causes the uncontrolled production of defective cells via a protein called Menin.

The new pill is not a definitive cure and does not work on every leukemia patient. The medication blocks the protein that spreads cancer cells through the blood in this type of leukemia.

One-third of the patients responded adequately to the new pill, Revumenib. Eighteen people achieved complete cancer remission after the trial.

Revumenib is a less aggressive treatment than traditional chemotherapy. According to the study's authors, patients reported mild or no side effects.

Another benefit of the new medicine is that it can be taken at home so that patients can go through treatment in a more comfortable environment, next to their caregivers and loved ones.

Michael Rosensweig, a patient that participated in the study, showed satisfaction with the mild effect of the pill: "The drug did what it was supposed to do and was much easier on my body," he said in a press release by MSK.

Dr. Eytan Stein, Chief of the Leukemia Service and Director of MSK's Program for Drug Development in Leukemia, remarked on that medication aspect in a press release: "This is everything you want in targeted therapy," he said.

According to MSK pediatric oncologist Dr. Neerav Shukla, pediatric leukemias frequently carry the genetic changes of acute myeloid leukemia. Revumenib can be an option for these children, and some have shown responses in the trial, said Dr. Shulka.

However, there is still a long way to go to find a cure for this disease. The new treatment helps patients reach remission but is not a definitive cure.

According to MSK, twelve of the eighteen patients that reached remission went on to receive bone marrow or stem cell transplants. One requisite to qualify for the procedure is having little to no cancer cells in the body.

Other patients combined the treatment with other traditional cancer medication, like chemotherapy. "We hope to eventually develop combination therapies that will help avoid or get around drug resistance," said Dr. Stein.

The patients who saw a cancer remission during the trial developed resistance to the drug in an average of nine months. The cancer cells developed genetic changes that disabled the treatment.

The researchers published the findings of the clinical trial in Nature magazine. They are also working on a second paper, studying drug resistance to improve the effect of revumenib in the future.