Advances in Hematologic Malignancies
Issue 12, Summer 2020
— David P. Steensma, MD
In July 2019, The Edward P. Evans Foundation awarded Dana-Farber a $5 million grant to set up a new center for myelodysplastic syndromes (MDS). Benjamin Ebert, MD, PhD,
the George P. Canellos, MD, and Jean S. Canellos Professor of Medicine at Harvard Medical School and chair of the Department of Medical Oncology at Dana-Farber, is the scientific director of the Center, and I am serving as the clinical leader and
the inaugural Edward P. Evans Chair in MDS Research at Dana-Farber. This grant provides us with a tremendous opportunity to understand MDS disease biology better and, most importantly, to improve outcomes for patients.
New insights into MDS biology and new treatments are clearly needed. While we have made strides in the last few years in understanding many of the basic mechanisms of mutation acquisition and of MDS cell biology, identifying specific cell vulnerabilities
that could be targeted with precision medications or immunotherapies is still in its infancy. In 2020, the FDA approved two new drugs for MDS-related indications: luspatercept for transfusion-dependent patients with ring sideroblasts, and oral decitabine/cedazuridine for patients with all subtypes of MDS except IPSS low risk disease. These drugs were the first new FDA approvals in MDS since 2006, and can be helpful for some patients, but are not curative. The only potentially curative therapy for MDS remains allogeneic hematopoietic cell transplant, which less than 10% of patients with MDS undergo due to the advanced age or comorbidities of many affected patients.
The Evans MDS Center at Dana-Farber includes four components:
- The first component is an active research fund for immediate investigator needs. We have formed a small committee of MDS experts across the Dana-Farber/Harvard Cancer Center to help select the most promising projects to support with these funds.
- Second, an endowment will allow us to continue to do research into MDS in the years to come.
- The third component of the grant is a visiting speaker series, which will help us to bring in two top-notch researchers per year to Dana-Farber to discuss their latest findings that are relevant to MDS.
- Finally, the Center will fund dedicated early-career investigators for two years as an "Edward P. Evans Fellow in MDS Research," in order to encourage promising young physician-scientists with fresh ideas to work on this challenging clinical problem.
Rahul Vedula, MD, was recently named as the inaugural Evans Fellow
in MDS at Dana-Farber. Dr. Vedula is currently working in the laboratory of R. Coleman Lindsley, MD, PhD, on a project related to the splicing factor U2AF1, and he joined the Dana-Farber Adult Leukemia
program as an Instructor in July 2020, where he will benefit from mentorship from Dana-Farber Adult Leukemia Program leaders Richard Stone, MD, and Daniel DeAngelo, MD, PhD.
Dr. Vedula was an outstanding Brigham and Women's medicine resident and Dana-Farber/Partners Cancer Care hematology-oncology Fellow. He received his medical degree at Tufts University and also worked for several years in a biomedical engineering group
at the Boston University Photonics Center.
Omar Abdel-Wahab, MD PhD, of Memorial Sloan-Kettering Cancer Center in New York was scheduled to serve as the inaugural Evans lecturer at Dana-Farber in May 2020, but this presentation was postponed due to the global pandemic. The Abdel-Wahab Laboratory
focuses on understanding the role of disordered mRNA splicing in myeloid malignancies. In 2019, Dr. Abdel-Wahab was an author on four Nature papers and numerous studies in other high-impact journals. We look forward to rescheduling Dr. Abdel-Wahab's
presentation at a future date; this presentation will be open to the public.
The Edward P. Evans Foundation was established in 1984 by Mr. Evans, who was the CEO of Macmillan Publishing, a thoroughbred racehorse breeder, and one of three sons of financier and philanthropist Thomas Mellon Evans of Pittsburgh. His philanthropy has
supported medical and educational causes. Mr. Evans died in 2010 of acute myeloid leukemia (AML), a disease that a subset of patients with MDS will develop, and one of his bequests was to support transformative research into MDS. Since 2014, Michael
Lewis, PhD, has served as President of the Foundation; Dr. Lewis has decades of experience in the pharmaceutical industry designing small molecules and novel biologics, and has keen insight into project management.
The Evans Foundation has already funded many laboratory-based investigators who have greatly improved understanding of MDS biology, particularly in the area of mRNA splicing, as acquired mutations in components of the spliceosome represent the most common
class of mutation in MDS. In addition, the Foundation supported the MDS Clinical Research Consortium administered by the Aplastic Anemia & MDS International Foundation of Rockville, Maryland; Dana-Farber was one of six participating centers in that
Consortium, which conducted several innovative interventional trials, and pooled databases to publish dozens of retrospective analyses.
We look forward to seeing how this new Center evolves and how investigators work together to improve outcomes for patients with MDS in the years to come.