Researchers from Mount Sinai present encouraging c

New York, NY (December 11, 2021) – Scientists from Mount Sinai have found that a new therapy for myelofibrosis in bone marrow cancer is safe and well tolerated and is associated with modest improvements in patients in a phase 1b clinical trial. They shared their results in an oral presentation at the American Society of Hematology annual meeting in December.

Patients on treatment, called AVID200, showed improvement in symptom burden, anemia and enlarged spleen. The results confirmed the safety of the therapy and showed evidence of effectiveness – although safety and the search for the optimal dose was the primary goal – and the researchers concluded that the therapy should be combined with other drugs to optimize the impact in patients.

“This is a real testament to cutting edge translational research at the Tisch Cancer Institute,” said John Mascarenhas, MD, director of the recently launched institute. Center of Excellence for Blood Cancers and Myeloid Disorders, which was created to translate science into cures for patients with blood cancers. “Our scientists have tested this therapy in the laboratory, medical researchers have conducted a successful Phase 1 trial, and now the optimal combination therapy approach is the subject of ongoing laboratory studies at Mount Sinai. The most interesting finding from this trial was that a subset of patients had a lasting improvement in their platelet counts, including three with normalized counts, supporting preclinical studies conducted by Mount Sinai researchers Ronald Hoffman, MD, Anna Rita Migliaccio, PhD, and Lillian Varricchio, PhD.

Myelofibrosis is a type of cancer of the bone marrow that disrupts the normal production of blood cells, 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 failed available first-line treatment face a well-documented grim prognosis, so additional therapies are urgently needed to help these patients.

Twenty-one patients included in this multicenter trial received AVID200, and although the primary objective of this trial was to test safety, some patients had increased platelet counts and their enlarged spleen decreased in size. However, despite the clinical benefits observed, the patients’ spinal scars did not decrease, so doctors believe that AVID200 will likely need to be combined with other rational therapies in the future.

In addition to this study, Mount Sinai launched the new Center of Excellence for Blood Cancers and Myeloid Disorders to further deepen its commitment to quality care for patients with blood and bone marrow cancer. A leader in the study and treatment of leukemia, including myeloproliferative neoplasms and myelodysplastic syndromes, the Center is one of the most active and recognized programs in the country. It is part of The Tisch Cancer Institute at Mount Sinai, a cancer center designated by the National Cancer Institute.

The Center is focused on providing multidisciplinary, cutting-edge and highly personalized care to patients with all forms of blood cancer and myeloid disorders. The Center brings together world-renowned physicians to provide exceptional patient care based on their experience, innovation, clinical trials, research and education.

“At the Center of Excellence for Blood Cancers and Myeloid Disorders, we believe everyone deserves personalized and excellent care, which is why we are developing an individualized treatment plan for you, using the latest technology and advancements. in biological understanding, ”said Dr Mascarenhas, also professor of medicine (hematology and medical oncology) at Icahn Mount Sinai. “We use genetic, epigenetic, cell signaling and immunological factors in developing your treatment plan. We are proficient in stem cell transplantation and we are expert in overcoming graft versus host disease, one of the main challenges in stem cell transplantation. Thanks to this center and our ongoing research, we will continue to make progress in the diagnosis and treatment of blood cancers in our patients. “

About the Mount Sinai Health System

The Mount Sinai Health System is New York City’s largest academic medical system, comprising eight hospitals, a leading medical school, and an extensive network of outpatient practices throughout the greater New York City area. Mount Sinai advances medicine and health through unparalleled education, translational research and discovery to provide the safest, highest quality, most accessible and equitable care, and the best value of any health care system. health of the country. The health system includes around 7,300 primary and specialist care physicians; 13 joint venture outpatient surgery centers; more than 415 outpatient offices in the five boroughs of New York, Westchester, Long Island and Florida; and over 30 affiliated community health centers. Mount Sinai Hospital is ranked on US News and World Reports “Honor Roll” of the 20 best American hospitals and is the first in the country by specialty: n ° 1 in geriatrics and top 20 in cardiology / cardiac surgery, diabetes / endocrinology, gastroenterology / gastrointestinal surgery, neurology / neurosurgery, orthopedics, pneumology / Pulmonary surgery, rehabilitation and urology. New York Eye and Ear Infirmary of Mount Sinai is ranked # 12 in Ophthalmology. Mount Sinai Kravis Children’s Hospital is classified under US News and World Reports “Best Children’s Hospitals” among the best in the country in four of the ten pediatric specialties. Icahn School of Medicine is one of three medical schools that have distinguished themselves by several indicators: ranked in the top 20 by US News and World Reports “Best medical schools”, aligned with a American News and World Report Honor Roll Hospital and No. 14 in the country for funding from the National Institutes of Health. Newsweek “World’s Best Smart Hospitals” ranks Mount Sinai Hospital first in New York City and in the top five in the world, and Mount Sinai Morningside in the top 20 in the world.

For more information visit https://www.mountsinai.org or find Mount Sinai on Facebook, Twitter and Youtube.


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