Ruben Mesa, MD, FACP, executive director of the Mays Cancer Centre at UT Health San Antonio, has seen too many patients and families struggle with deadly bone marrow cancers.
Now, thanks to the success of a new drug in a study he helped lead, Mesa hopes to relieve some of those struggles.
An international expert on myeloproliferative neoplasms (MPNs), a group of bone marrow disorders that often lead to leukemia, Mesa is a 25-year trailblazer in MPN research, leading the development of the first U.S. guidelines on diagnosis and treatment of these disorders.
He has been principal investigator or co-principal investigator of more than 70 clinical trials that have resulted in MPN drug therapies, several of which have gained U.S. Food and Drug Administration approval.
Momelotinib, the latest drug to be studied, is an oral pill taken once a day. It significantly improved outcomes of patients treated for myelofibrosis (MF), a rare but fatal bone marrow cancer.
At a recent annual meeting of the American Society of Clinical Oncology, he presented results of the MOMENTUM phase 3 randomized study, which evaluated momelotinib against a second medication, danazol, in symptomatic and anemic MF patients previously treated with existing drug therapy. The MOMENTUM study enrolled 195 MF patients in 21 countries.
An urgent need
In myelofibrosis, scar tissue forms in the bone marrow and hinders the body’s ability to produce healthy blood cells. Anemia, a lack of red blood cells to carry oxygen throughout the body, is observed in virtually all MF patients and negatively impacts survival, Mesa said.
“A third of the patients have anemia up front, and most patients will develop it over the course of their disease,” Mesa said. “For patients with severe anemia, survival is shortened to about two years. Even with mild anemia, the median survival is 4.9 years. A drug to treat anemia in these patients has been urgently needed.” Without anemia, median survival with MF is closer to eight years, he said.
Myelofibrosis is characterized by abnormal signaling of JAK proteins and excessive activation of another protein, ACVR1. Pioneering MF drugs developed over the last decade, such as ruxolitinib, inhibit the harmful JAK signaling.
Momelotinib, however, is the first drug that inhibits both JAK and ACVR1, Mesa said. “It has JAK1, JAK2 and ACVR1 inhibitors combined in one drug and addresses chronic inflammation due to the hyperactivation of ACVR1 as a consequence of the disease.”
Of the enrolled participants, 130 received momelotinib and 65 got danazol. Participants did not know until after 24 weeks which drug they received, and those in the danazol group were allowed to cross over to momelotinib at that time.
In the momelotinib group, researchers observed:
- Significant improvements in symptoms, spleen size and anemia measures.
- Favorable safety and a trend toward improved overall survival.
- Participants required fewer transfusions to replace red blood cells and evidenced better oxygen-carrying hemoglobin levels.
“The results were compelling,” Mesa said. “The study enrolled participants entirely during the COVID time frame, which was remarkable. Momelotinib met all primary and secondary endpoints and within the short span of six months, there was a trend toward overall survival benefit, which is also remarkable.”
An effective treatment
“Findings support the future use of momelotinib as an effective treatment in MF patients, especially in those with anemia,” Mesa concluded. This represents more hope for patients and families like those he remembers.
Read more about this research and other discoveries aimed at curing cancer.
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