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A new class of treatments called CAR-T therapy is providing options for patients who have all but lost hope in their fight against cancer. This form of immunotherapy is based on genetically enhancing a patient’s own immune cells to target and kill their cancer. The Food and Drug Administration approved Yescarta for adults with a form of blood cancer called non-Hodgkins lymphoma. This second CAR-T therapy follows closely on the heels of Kymriah, which was approved in September to treat certain lethal blood and bone marrow cancers in children.
The National Academy of Medicine announced the election of 80 new members. Election to the Academy is considered one of the highest honors in the fields of health and medicine and recognizes individuals who have demonstrated outstanding professional achievement and commitment to service. Damon Runyon congratulates the four alumni who were elected this year:
Scott A. Armstrong, MD, PhD (Clinical Investigator ’03-’08), Dana-Farber Cancer Institute
Howard Y. Chang, MD, PhD (Scholar ’06-’08), Stanford University
The National Institutes of Health’s High-Risk, High-Reward Research program, funded 86 awards to exceptionally creative scientists proposing to use highly innovative approaches to tackle major challenges in biomedical research. The program is designed to accelerate scientific discovery by supporting high-risk research proposals. Applicants of the program are encouraged to think outside-the-box and to pursue exciting, trailblazing ideas. Four Damon Runyon scientists are recipients of this year’s awards:
Gordon J. Freeman, PhD (Damon Runyon Fellow ’79-’81), Dana-Farber Cancer Institute, Boston, is one of five scientists honored by the 2017 Warren Alpert Foundation Prize for discoveries focused on “Immune Checkpoint Blockade and the Transformation of Cancer Therapy.” Collectively, their work has elucidated foundational mechanisms in cancer’s ability to evade immune recognition through the CTLA-4 and PD-1 pathways and, in doing so, has profoundly altered the understanding of disease development and treatment.
Liron Bar-Peled, PhD (Damon Runyon Fellow ‘14-‘17) of the Scripps Research Institute, La Jolla, developed a new proteomics-based approach to discover small-molecule inhibitors that could be used as anti-cancer therapies. The approach is based on the fact that certain amino acids on proteins have a special chemical reactivity that allows them to form irreversible covalent bonds with suitably designed probe or "scout" molecules.
Alexandra Zidovska, PhD (Damon Runyon Fellow ‘10-‘12) of New York University, New York, has discovered the “internal clock” of live human cells using state-of-the-art fluorescence microscopy. Previously, the only way to tell the precise point of a cell in its life cycle was by studying dead cells. Alexandra’s lab has found that the nuclear envelope, which separates the nucleus with the DNA from the rest of the cell, has a previously undetected type of motion: it fluctuates in shape every few seconds.
Kristopher R. Bosse, MD (Damon Runyon Physician Scientist '16-'20) of the Children's Hospital of Philadelphia, is engineering a new drug to selectively target neuroblastoma cells and deliver a chemotherapeutic agent into the cells. Neuroblastoma is a cancer of the developing nervous system that usually occurs as a solid tumor in a child's chest or abdomen, and is the most common cancer in infants.
Five researchers have been announced as the recipients of the Albany Medical Center Prize in Medicine and Biomedical Research for 2017. They have made important contributions to the development of CRISPR-Cas9, a gene engineering technology that harnesses a naturally occurring bacterial immune system process. The technology has revolutionized biomedical research and provided new hope for the treatment of genetic diseases and more.
The FDA approved Idhifa for acute myeloid leukemia (AML), the result of important contributions from Hai Yan, MD, PhD (Damon Runyon Scholar ‘05-‘07), of Duke University Medical Center, Durham, and Omar Abdel-Wahab, MD (Damon Runyon Clinical Investigator ‘13-‘16), of Memorial Sloan Kettering Cancer Center, New York. They made independent discoveries about mutations in the IDH2 gene and how these contribute to the development of AML.
Marcela V. Maus, MD, PhD (Damon Runyon-Rachleff Innovator ’17-’18) at Massachusetts General Hospital, Boston, published results from her clinical trial with glioblastoma patients showing for the first time that CAR (chimeric antigen receptor) T cells cross the blood-brain barrier to reach tumors and appeared to be safe. These CAR T cells were targeted to EGFR variant III in glioblastoma patients. Her lab is working on next steps such as combining CAR T cells with other drugs to increase the potency of this treatment to shrink tumor size.