Scientists at Mount Sinai Hospital found the new therapy for myelofibrosis in bone marrow cancer was safe, well-tolerated, and resulted in improvement in patients in phase 1b clinical trials. They shared their findings during an oral presentation at the American Society of Hematology Annual Meeting in December.

Patients receiving AVID200 therapy showed improvement in symptoms of anemia and enlarged spleen. The results confirmed the safety of the therapy and demonstrated some evidence of efficacy – although safety and finding the optimal dose were the main goals of the trials, the researchers concluded that the therapy should be combined with other drugs to optimize the effect on patients.

“This is real evidence of cutting-edge translational research at the Tisch Cancer Institute,” said John Mascarenhas, MD, director of the recently opened Center of Excellence in Blood Cancer and Myeloid Diseases. “Our scientists have tested this therapy in the laboratory, medical scientists have successfully tested the first phases, and now the optimal combination therapy approach is the subject of ongoing laboratory research at Mount Sinai Hospital. The most interesting finding in this study was that a subset of patients showed a sustained improvement in platelet counts, of which three were completely normalized, as supported by preclinical studies. ”

Myelofibrosis is a type of bone marrow cancer that disrupts normal blood cell production, causing an enlarged spleen, extensive scarring in the bone marrow, and low levels of red blood cells and platelets, increasing the risk of bleeding. Patients with myelofibrosis who have not received available first-line therapy have a poor prognosis for survival, so additional therapies are urgently needed to help these patients.

The 21 patients enrolled in this multicenter study received AVID200, and although the main purpose of this study was to test safety, some patients had increased platelets and their enlarged spleens decreased in size. However, despite the clinical benefits observed, bone marrow scarring in patients has not diminished, so doctors believe that AVID200 may need to be combined with other rational therapies in the future.

Mount Sinai Health System is New York City’s largest academic medical system, with eight hospitals, a leading medical school, and an extensive network of outpatient practices throughout the Greater New York region.