Patients with terminal leukemia who have not responded to treatment now have hope for a cure, thanks to a new experimental pill called levmenib.
The drug has completely eliminated cancer in a third of participants in a long-awaited clinical trial in the United States.
Not all patients went into complete remission, but scientists remain hopeful as research results show the pill may pave the way for future leukemia treatments.
Ghayas Issa, M.D., a leukemia specialist at the University of Texas MD Anderson Cancer Center and study co-author, said:
“They had progressed on multiple treatments, and about half of them had lost leukemic cells from their bone marrow.
How do these tablets work?
Acute myeloid leukemia (AML) is a type of cancer that attacks the bone marrow where blood cells are produced, causing uncontrolled production of defective cells.
Levmenib is a new class of targeted therapy for acute leukemia that inhibits a specific protein called menin. The drug works by reprogramming leukemic cells into normal cells.
Menin is involved in a complex mechanism that is hijacked by leukemia cells and transforms normal blood cells into cancer cells.
Using levmenib turns off the engine and turns the leukemia cells back into normal cells, bringing remission, explained Issa.
The formula has already saved 18 lives as part of a clinical trial, with promising results announced this month. Nature.
Preliminary results showed that 53% of patients responded to levumenib, 30% had complete remission, and no cancer was detected in their blood.
Based on data from this study, in December 2022, the U.S. Food and Drug Administration granted revumenib Breakthrough Therapy Designation to help expedite its development and regulatory review.
A cure for leukemia, but it’s not for everyone
“This is definitely a breakthrough and scientific achievement over the years. Many groups have been working hard in the lab to understand the causes of these leukemias,” said Issa.
However, he explained that the drug doesn’t work for all patients.
The experimental tablets target the most common mutation in acute myeloid leukemia, a gene called NPM1, and a less common fusion gene called KMT2A. Together, these mutations are estimated to occur in approximately 30-40% of acute myeloid leukemia patients.
The Phase 1 trial enrolled 68 patients at nine US hospitals. They all either had relapsed leukemia after other treatments or had never responded well to conventional chemotherapy drugs in the first place.
Among them was 23-year-old Lithuanian architect Algimante Daugeliate, who was diagnosed with leukemia. She had had her two bone marrow transplants from her older sister, but all her other treatments had failed. Her doctor was considering her palliative care to ease her suffering.
“I was desperate. It was like living a scary movie. I felt like death was imminent. I was just 21,” she said. told El Pais.
But two years ago, when she started taking levumenib, Daugeliate made a full recovery. Since then she has been able to resume her normal life, graduated from university and works in an architectural studio in Copenhagen.
Powerful effect with limited side effects
Issa says the drug is fairly safe compared to standard treatments for leukemia, but two main side effects have been identified.
The first affects the heart’s electrical system and can be detected on an electrocardiogram (ECG). However, reducing the dose or stopping treatment resolved the problem in all cases, Issa said.
The second side effect is called differentiation syndrome. life-threatening reaction Although it affects the treatment of blood cancers, it can be effectively managed if detected early and stopped with appropriate measures. According to Issa, all cases of differentiation syndrome in this study were successfully managed without patient complications.
This research is still in its early stages and the results are preliminary. These Phase I trials aim to test whether a drug is safe and to find the highest dose that can be administered without causing serious side effects.
Phase II trials are currently underway, specifically investigating the efficacy of levmenib.
Twelve patients in the trial who responded to the drug underwent stem cell or bone marrow transplantation.Such transplants require patients to have no or very low levels of cancer in their blood, and levmenib helped them get there.
The experimental pill isn’t a definitive treatment, but the researchers who worked on the trial are optimistic.
“In the future, we plan to combine this tablet with our current standard of care for acute leukemia,” said Issa.
“It’s the most likely strategy to lead us to a cure that doesn’t require the patient to see a leukemia doctor afterwards and not need treatment for leukemia.