Patients with leukemia terminal not responding to treatment now they have hope for a cure, thanks to an experimental new pill called revumenib.
This drug completely eliminated the cancer in a third of participants in a long-awaited clinical trial in the United States.
While not all patients have shown complete remission, scientists remain hopeful as the findings indicate the pill could pave the way for a cure for leukemia in the future.
“We are incredibly confident by these results from patients who have received this drug. This was their last chance,” said study co-author Dr. Ghayas Issa, a leukemia physician at the University of Texas MD Anderson Cancer Center.
“They have progressed on multiple lines of therapy and a fraction of them, about half, have had their leukemia cells disappear from their bone marrow,” he told Euronews Next.
How does this pill work?
Acute myeloid leukemia (AML) is a type of cancer that attacks the bone marrow, where blood cells are made, and causes the uncontrolled production of defective cells.
Revumenib is a new class of targeted therapy for acute leukemia that inhibits a specific protein called menin. The drug works by reprogramming leukemia cells into normal cells.
Menin is involved in the complex machinery that is hijacked by leukemia cells and causes normal blood cells to turn cancerous.
Using revumenib, Issa explained, the engine is turned off and leukemia cells are transformed into normal cells, resulting in remission.
This formula has already saved 18 lives in clinical trials, the promising results of which were published this month in Nature.
Preliminary results showed that 53% of patients responded to revumenib and 30% had a complete remission without any cancer detectable in blood.
Based on data from this study, the U.S. Food and Drug Administration granted “breakthrough therapy designation” to revumenib in December 2022 to expedite its development and regulatory review.
A cure for leukemia, but not for everyone
“This is definitely a step forward and is the result of years of science. Many groups have been working hard in the lab to figure out what’s causing these leukemias,” Issa said.
However, he explained that the drug doesn’t work for all patients. It is for a specific subset of leukemias which generally have missing or mislabeled genes or a chromosome fusion.
The experimental pill targets the most common mutation in acute myeloid leukemia, a gene called NPM1 and a less common fusion called KMT2A. Together, these mutations are estimated to occur in approximately 30-40% of people with acute myeloid leukemia.
The phase 1 study enrolled 68 patients at nine US hospitals. All of them had seen their leukemia come back after other treatments or had never responded well ai traditional chemotherapy drugs in the first place.
Among them was Algimante Daugeliate, a 23-year-old Lithuanian architect who was diagnosed with leukemia. He had received two bone marrow transplants from his sister, but all other treatments had failed. His doctors had even considered palliative care to ease his suffering.
“I was desperate. It was like living a horrible movie. I felt like death was imminent and I was only 21,” he ha He told El Pais.
However, once she started taking revumenib two years ago, Daugeliate made a full recovery. Since then he has been able to resume his normal life, graduating from college and working in a studio architecture a Copenhagen.
Strong impact for limited side effects
According to Issa, although this drug is quite safe compared to standard leukemia treatments, two major side effects have been identified.
The first affects the electrical system of Heart and can be detected with an electrocardiogram (ECG). However, reducing the dosage or stopping the treatment resolved the problem in all cases, Issa said.
The second side effect is referred to as differentiation syndrome – a group of potentially life-threatening reactions to blood cancer treatments, but it can be managed effectively if it is recognized early and appropriate steps are taken to turn it off. According to Issa, all cases of differentiation syndrome in this study were managed with success without complications for the patients.
The study is still in its early stages and the results remain preliminary. Phase I trials like this one aim to test whether a drug is safe and to find the highest dose that can be administered without causing serious side effects.
A Phase II study looking specifically at the efficacy of revumenib is currently underway.
Twelve patients in the study who responded to the drug went on to receive a stem cell or bone marrow transplant. Such transplants require patients to have no cancer or only very low levels of cancer in their blood, and revumenib has helped them get there.
Although the experimental pill is not a definitive cure, the researchers who worked on the trial are optimistic.
“In the future, we plan to combine this pill with the standard treatments we currently have for acute leukemias,” Issa said.
“This is the strategy most likely to lead us to care where patients don’t have to view leukemia doctors after and don’t need leukemia treatment.”