A new study suggests that an experimental drug being tested for the treatment of multiple sclerosis and to prevent organ rejection might also help people with certain deadly forms of chronic and acute leukemia.
The laboratory and animal study focused on the drug, called fingolimod. Researchers said it might help patients with advanced chronic myelogenous leukemia (CML) or acute lymphocytic leukemia (ALL), and whose cancer cells show a particular genetic change called the Philadelphia chromosome.
The study found that the drug prevented the development of these cancers in mouse models, as well as killing laboratory-grown human CML and ALL cells.
Although the findings must be verified in humans through a future clinical trial, the new research also suggests that the drug might help patients with these leukemias who are resistant to imatinib (Gleevec) and dasatinib (Sprycel), two important current drugs for treating CML and those cases of ALL with the Philadelphia chromosome.
Presently, fingolimod is in advanced clinical-trials testing for the treatment of relapsing multiple sclerosis, and to prevent organ rejection following kidney transplantation.
The new study, led by researchers with the Ohio State University Comprehensive Cancer Center, is published online Aug. 23 in the Journal of Clinical Investigation.
“This novel agent represents a promising new strategy for treating CML that is resistant to imatinib and related targeted agents,” says coauthor Guido Marcucci, associate professor of internal medicine and an oncologist who specializes in leukemia drug development at Ohio State's James Cancer Hospital and Solove Research Institute.
“These findings also suggest that it will be an important contribution to a new therapeutic approach to CML that considers combinations of molecular targeting compounds.”
In leukemic cells, the drug works by reactivating a protein called PP2A, which normally helps protect cells from becoming cancerous. The protein then causes the cells to self-destruct through a process called apoptosis.
“This was true even in leukemic cells from patients that were resistant to imatinib or to dasatinib,” says principal investigator Danilo Perrotti, assistant professor of molecular virology, immunology and medical genetics.