An experimental leukemia pill has shown promise in early clinical trials, with one-third of patients with advanced disease achieving complete remission.
Levmenib is also known to induce partial remission in half of acute myeloid leukemia (AML) patients in phase 1 clinical trials.
Dr. Scott Armstrong, an expert in blood cancers at the Dana-Farber Cancer Institute in Boston, Massachusetts, who is conducting the trial, hailed the results as “very encouraging.” said more research is needed to prove the drug is effective.
He said his team could apply for Food and Drug Administration (FDA) approval by the end of this year if more rigorous testing is successful. The first trial did not include a control group. This means that this drug has not been tested against current treatments.
Acute myeloid leukemia is a type of blood cancer that affects white blood cells, which begin to divide uncontrollably and become abnormal, unable to fight infection.
About 59,000 cases are diagnosed in the United States each year, and 23,700 people die from the cancer.
The cells involved are notoriously difficult to treat because they are diverse and can mutate rapidly, making it difficult to target them with new therapies such as immunotherapy.
Also, because they divide rapidly and spread to different areas of the body, doctors struggle to eliminate them without harming healthy tissue.
A study published in the journal this month found that Naturethe researchers focused on leukemia caused by mutations in the NPM1 and KMT2A genes.
It accounts for approximately 40% of leukemia cases in the United States. Up to 80% of people diagnosed with these mutations in leukemia die within her 5 years.
In leukemias with these mutations, a protein called menin binds to the gene, allowing cancer cells to continue growing and dividing.
To break this cycle, levmenib uses a molecule that binds to menin, stopping menin from attaching and thus triggering the gene.
Patients were asked to take the drug in capsules twice daily, 12 hours apart.
They were followed for about a year on average.
Results showed that a total of 18 patients (30%) had complete cancer remission during the study. This means that all signs and symptoms of cancer have disappeared.
Scientists said the average time to reach complete remission was two months.
Another 32 patients (53%) also showed partial remission or reduction in tumor size or extent of cancer in the body.
The patient remained in partial remission for 9 months.
Overall, patients survived approximately seven months after study entry.
Nearly all patients experienced side effects, the most common being arrhythmias and nausea.
Seven had to discontinue the study due to severe reactions to the drug.
However, in some participants there was evidence that cancer cells had developed resistance to the drug.
Dr. Armstrong, who led the study, said: “For patients with acute leukemia who have had several previous treatments, this is a very encouraging result.
“However, after two cycles of treatment, some patients developed resistance to levmenib.”
Phase 1 trials are designed to test the safety and optimal dose of experimental treatments.
Currently, patients with NPM1-mutant leukemia or KMT2A mutations are offered chemotherapy to combat this disease.
Surgery is rarely used to treat acute myeloid leukemia. Radiation therapy may be used when cancer has spread outside the bone marrow or blood.
About two in three of these patients will then go into remission, says the American Cancer Society.
According to doctors, patients with NPM1 leukemia tend to respond well to chemotherapy.
