Near-Terminal Leukemia Patients In Remission As Experimental Drug Raises Hope
In great news for people afflicted with leukemia, scientists have been able to achieve complete cancer remission with the help of an experimental drug.
Acute myeloid leukemia is the most common blood cancer in adults, with 120,000 cases new cases being diagnosed every year. Unfortunately, the three-year survival rate is just 25% for the disease.
The anti-cancer drug in question, called revumenib, has completely eliminated cancer in 18 near-terminal patients with aggressive tumors that did not respond to treatments, El Pais reported.
"The results are preliminary, and do not suggest a definitive cure, but the authors of the experiment are optimistic," said Dr. Ghayas Issa from the MD Anderson Cancer Center at the University of Texas, according to the outlet and added, "we think this pharmaceutical is extraordinarily effective, and we hope for it to be accessible to everyone who needs it."
Acute myeloid leukemia targets the bone marrow, where blood cells are produced. It is characterized by the unregulated production of defective cells.
However, the drug is not a panacea for all patients. In the study, researchers focused on two genetic subtypes, in which a protein called menin helps leukemia to progress.
The results of the study were published Wednesday in the journal Nature.
According to hematologist Pau Montesinos, coordinator of the Spanish Group of Acute Myeloid Leukemia, “In the vast majority of cases, these targeted therapies, on their own, can revert leukemia, but rarely cure it. The strategy is to combine these new pharmaceuticals with classic chemotherapy or other approaches.”
However, Revumenib does have a concerning effect. Hematologist Eytan Stein of the Memorial Sloan Kettering Cancer Center in New York, who led the trials, said, “The main Achilles heel seems to be the development of mutations at the drug’s fusion site, which causes resistance.”
While the drug had some form of positive effect on 50% of the 60 participants in the study, in some of the patients, the menin protein changed a little, leading to resistance to the treatment.
“This demonstrates that we’re on the right path and that the target of the drug [the menin protein] is critical for the development of leukemia with these genetic subtypes,” Stein said.
The drug works in a novel way by attaching and effectively blocking the menin protein. This mechanism of action is similar to substances being developed by at least six pharmaceutical companies.
Issa opines that almost 400,000 people with acute leukemias could benefit from these new pills.
Another stellar study led by a team of Australian researchers found that by using chimeric antigen receptor therapy on the T cells, which naturally occur in white blood cells and make the body immune to viruses, a new treatment of cancer can be introduced. “This new research is really exciting, it uses COVID-19 immunity, T ‘killer’ cells, to recognize COVID, engineers them to attack breast cancer cells--really clever,” professor Robert Booy, who was associated with the research, said.