An experimental drug cures advanced leukemia in 18 patients

The myeloid leukemia acute is he blood cancer more frequent in adults and is very aggressive, since the survival of those affected three years after diagnosis does not exceed 25%, but now an experimental drug that is administered orally – it is a pill – could open a new way of hope for these patients because he has achieved the complete remission of the disease in 18 patients who were participating in a clinical trial and who had not responded to other treatments.

The drug is called revumeni and eliminated the signs of cancer in almost one in three patients who took it, although these are preliminary results that do not imply a definitive cure, the authors of the study have been optimistic. “We believe this drug is remarkably effective and we hope it will be available to all patients who need it,” said Ghayas Issa, MD, of the University of Texas MD Anderson Cancer Center. The results of the Phase I AUGMENT-101 trial have been published in Nature.

Acute myeloid leukemia affects the bone marrow, where blood cells are made, and causes defective cells to proliferate. The trial involved 68 patients with a median age of 43 years and aimed to assess the safety and antitumor activity of revumenib in two genetic subtypes in which a protein called menin contributes to leukemia progression. Revumenib is an oral small-molecule inhibitor of the menin-KMT2A interaction in children and adults with acute myeloid or lymphoid leukemias with advanced KMT2A rearrangement or NPM1 mutation, and is therefore indicated in these specific cases.

A breakthrough in the treatment of acute myeloid leukemia

“The responses in this trial show that menin inhibitors may be a promising treatment option that is well tolerated by patients and could be the latest addition to successful targeted therapies for acute leukemia,” Issa said. “I look forward to additional data from this and future trials to inform the potential opportunity to offer this specific treatment to more patients.”

“Menin inhibitors may be a promising treatment option that is well tolerated by patients and could be the newest addition to successful targeted therapies for acute leukemia”

The coordinator of the Spanish myeloblastic leukemia group and head of the Hematology service at Hospital La Fe in Valencia, Pau Montesinos, explained how the treatment works in the Hoy por Hoy program of Cadena Ser. “These are data recently published in Nature and there was no information until now, but they are years of work in the development of new molecules for the acute leukemia treatment and this particular drug is being developed, like five or six others that work in the same way, in different clinical trials for only two or three years. We are really seeing that it is a very novel drug that can contribute to patients globally in the medium-term future”.

Montesinos stressed that “the mechanism is very different from conventional chemotherapy” and that this drug repairs the genetic defect present in the leukemia cell so that it returns to normal, unlike the chemotherapy that has been administered for three decades to patients. acute leukemia patients, which kills both cancer cells and healthy cells, which has many side effects.

According to this expert, revumenib has fewer side effects than current chemotherapy treatments, but believes that “on its own it will hardly be able to cure, to definitively eradicate cancer cells. We will have to combine it either with conventional chemotherapy or with other drugs that are being developed.”


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