In 2019, Dana-Farber established the Edward P. Evans Center for Myelodysplastic Syndromes (MDS) with generous funding from the Edward P. Evans Foundation. The foundation recently awarded new grants totaling $1.35 million to three Dana-Farber scientists for novel investigations into MDS, a group of blood disorders caused by abnormal bone marrow stem cells.
Benjamin Ebert, MD, PhD, co-director of the Evans Center, chair of Medical Oncology, and the George P. Canellos, MD, and Jean S. Canellos Professor of Medicine, received a Discovery Research Grant to address critical challenges in MDS research. To develop new therapies for MDS, better preclinical models are needed to guide the design of clinical trials. Ebert will use two different genome editing approaches—CRISPR-Cas9 and CRISPR-Cas12—to build more advanced mouse models of MDS to study the numerous genetic mutations driving the disease.
“We hope that multiplexed editing of genes will provide better models of MDS and that this will ultimately lead to robust preclinical studies to guide effective clinical trials,” says Ebert.
Christopher Reilly, MD, and Mounica Vallurupalli, MD, each received Evans MDS Young Investigator Awards, which are geared toward early career scientists looking to establish their own labs as MDS investigators.
Reilly, director of the Multidisciplinary Telomere Biology Disorder Program, is examining the process by which individuals with short telomeres—the protective ends of chromosomes that prevent DNA damage—develop MDS. His project will determine which types of mutations occur in people with short telomeres and how these mutations lead to MDS.
With her grant, Vallurupalli is studying mutations in the splicing factor gene SF3B1, which impair red blood cell production, a hallmark of MDS. She will develop models that systematically test which genetic factors help to overcome impaired red blood cell production in SF3B1-mutated bone marrow stem cells.
“The Edward P. Evans Foundation considers our support of the highly innovative work in Benjamin Ebert’s laboratory, and our support of early investigators, such as Christopher Reilly and Mounica Vallurupalli, as one of the best investments we can make to discover new knowledge that will enable the development of effective MDS treatments,” says Michael Lewis, president of the Edward P. Evans Foundation. “Dana-Farber has been, and continues to be, one of our most valuable partners to achieve this goal, and we are excited to see what these efforts might yield.”
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