“Supporting early career investigators is an investment in the future,” says CEO Janice Frey-Angel of the Aplastic Anemia and MDS International Foundation. “We’ve watched previous AAMDSIF grant recipients become leading researchers in their fields. We look forward to future breakthroughs in treating rare bone marrow failure diseases.”
2021 Grant Recipients:
Salima Benbarche, Ph.D., Memorial Sloan Kettering Cancer Center (MSK), received the Harold Spielberg Research Fund Award. Her research will focus on “Developing synthetic introns for targeting spliceosomal mutant MDS.”
The purpose of my current research study is to identify and understand novel RNA regulators in leukemogenesis. We discovered a way to express proteins of interest in cells bearing the most common mutations in MDS, but not in healthy normal cells, using synthetic (not occurring in nature) introns. We successfully used these synthetic introns to specifically kill cancer cells carrying a defined, cancer-causing mutation and we aim to develop this technology to better understand and cure MDS and specific blood cancers.
Sushree Sahoo, Ph.D., St. Jude Children's Research Hospital, received the Emily Kass Research Fund Award. She will investigate “Mapping Clonal Ancestries in Pediatric MDS to Define Therapeutic Vulnerabilities.”
Myelodysplastic syndrome (MDS) in children is a rare group of disorders in which blood-making stem cells in the bone marrow fail to work properly. As a result, patients develop low blood counts and have increased risk to develop leukemia with cancerous cells, referred to refractory cytopenia of childhood (RCC). Although RCC is the most common category, we do not fully understand the gene and chromosome changes associated with the disease. Some patients with RCC have empty bone marrow which might be mistaken for aplastic anemia, while others have acquired changes that predispose them to more advanced MDS and leukemia. This study intends to use genetics to differentiate between RCC and other blood disorders such as inherited marrow failure or acquired aplastic anemia.
About The Aplastic Anemia and MDS International Foundation (AAMDSIF)
The AAMDSIF is the world’s leading nonprofit health organization dedicated to supporting patients and families living with aplastic anemia, myelodysplastic syndrome (MDS), paroxysmal nocturnal hemoglobinuria (PNH), and related bone marrow failure diseases. The Foundation provides answers, support, and hope to thousands of patients and their families around the world.
The AAMDSIF is a patient-focused, patient-centered organization, serving patients and families throughout the three phases of bone marrow failure diseases; their life-changing phase of diagnosis, their life-threatening phase of treatment, and their life-long phase of living with a chronic disease.
For more information please visit www.aamds.org. Follow AAMDSIF on social media: @aamdsif on Twitter; @aamds on Facebook
Media Contact: Alice Houk, Senior Director, Health Professional Programs Phone: (301) 279-7202 extension 101
(Photographs and more information are available upon request.)