An experimental pill achieves complete cancer remission in 18 people with aggressive leukemia Science & Technology

An experimental pill has achieved complete remission of the cancer in 18 nearly incurable patients with aggressive tumors that have not responded to treatments. The disease, acute myeloid leukemia, is the most common blood cancer in adults, with 120,000 cases per year. The three-year survival rate is only 25%. The new drug, called Revumenib, has completely eliminated cancer in a third of the participants in a long-awaited clinical trial in the United States. “The results are preliminary and do not indicate a definitive cure, but the authors of the experiment are optimistic. “We believe this drug is extraordinarily effective, and we hope it will be accessible to all who need it,” says Dr. Ghayas Issa of the MD Anderson Cancer Center at the University of Texas.

Acute myeloid leukemia attacks the bone marrow, where blood cells are made, causing the uncontrolled production of defective cells. That’s what happened to 23-year-old Lithuanian architect Algimante Daugeliate. She had received two bone marrow transplants from her sister. All other treatments were unsuccessful. Her doctors had begun to consider palliative care to alleviate her suffering. “I was desperate. It was like living through a terrible movie. I felt like death was imminent and I was only 21 years old,” she recalls. Two years ago she started taking Revumenib pills. She finished college and now works in an architecture firm in Copenhagen.

The drug does not work for all patients. The researchers have focused on two genetic subtypes in which a protein called menin allows the leukemia to progress. Revumenib binds to the protein and inhibits it thanks to its complex chemical formula: 32 carbon atoms, 47 hydrogen, one fluoride, six nitrogen, four oxygen and one silver. This formula, C32H47FN6O4S, saved 18 lives. The promising results were published on Wednesday in Nature.

I felt death was imminent and I was only 21 years old

Algimante Daugelaite, architect

Hematologist Pau Montesinos, coordinator of the Spanish Acute Myeloid Leukemia Group, believes the new information is “quite hopeful” but stresses caveats: Revumenib still needs to be tested on hundreds of people to confirm its safety and effectiveness. The Montesinos team, the leukemia department of the La Fe Hospital in Valencia, will take part in the next international trials of the pill developed by the American company Syndax Pharmaceuticals.

Montesinos adds that the drug alone is not a panacea. “In the vast majority of cases, these targeted therapies alone can reverse the leukemia, but rarely cure it,” explains the haematologist. “The strategy is to combine these new drugs with traditional chemotherapy or other approaches.” Montesinos recalls the case of another pill, quizartinib, an experimental treatment by Japanese pharmaceutical company Daiichi Sankyo that inhibits another protein involved in acute myeloid leukemia is. Adding quizartinib to chemotherapy increased remission from nearly 40% to nearly 50%, according to preliminary results from a study of 500 patients suffering from another subtype. “For us, an increase in the survival rate by 10 percentage points is a lot,” says the Spanish doctor.

Revumenib’s mechanism of action – inhibition of the protein menin – is new. Half a dozen pharmaceutical companies are developing substances using the same tactics. Revumenib’s success means good news for them. Oncologist Ghayas Issa has calculated that the new pills could benefit nearly 400,000 people with acute leukemia that is resistant to other treatments, including myeloid and the most common strain in children, called lymphocytic.

These targeted therapies alone can reverse leukemia but rarely cure it

Pau Montesinos, haematologist

Issa and his colleagues confirm that economic factors will be crucial if the pill is approved. According to a report by Democratic Congresswoman Katie Porter, the latest oral cancer drugs in the US are priced at over $235,000 per patient.

Revumenib has another weakness, as Eytan Stein, a hematologist at Memorial Sloan Kettering Cancer Center in New York who led the studies, pointed out. “The main Achilles’ heel appears to be the development of mutations at the drug fusion site that cause resistance,” explains the researcher. Revumenib had some beneficial effect in half of the 60 participants in the clinical study. However, in some patients, the menin protein changed slightly, resulting in resistance to treatment, much like bacteria mutate to become antibiotic resistant.

“This shows that we are on the right track and that the goal of the drug [the menin protein] is crucial for the development of leukemia with these genetic subtypes,” says Stein. To avoid such resistance mutations, the authors suggest combining drugs with different mechanisms of action. As Ghayas Issa and his colleague Eytan Stein observe, menin inhibitors “are definitely going to be part of a treatment for these leukemias.” Architect Algimante Daugelaite celebrates her participation in the process, saying science has given her “another opportunity to study, work, travel, see the world and, above all, live”.

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