Nobel Prize Awarded for Discovery of How Cells Sense Oxygen, ASH Members Elected to the National Academy of Medicine, and more

William G. Kaelin Jr., MD
Peter J. Ratcliffe, FRS, FMedSci
Gregg L. Semenza, MD, PhD

Nobel Prize Awarded for Discovery of How Cells Sense Oxygen

The Nobel Prize in physiology or medicine was awarded to scientists William G. Kaelin Jr., MD, Peter J. Ratcliffe, FRS, FMedSci, and Gregg L. Semenza, MD, PhD, for discovering how cells sense and respond to oxygen.

Their work began in the 1990s, when Dr. Semenza identified genes that switched on when oxygen levels were low to increase erythropoietin levels, thus producing more oxygen-carrying red blood cells. The process allows the human body to adapt to higher altitudes, but cancer cells also can exploit these molecular switches to thrive. Each scientist worked independently to understand the mechanism that allows cells to adapt to different oxygen levels, staying in contact and occasionally sharing data.

While the discoveries could spur new therapies for conditions related to a lack of oxygen, including stroke, heart attacks, respiratory diseases, and cancer, the researchers’ original incentive was to follow their curiosity and unravel basic biology – they did not necessarily expect their work to have clinical applications, according to Dr. Kaelin, who spoke with The Washington Post after the announcement.

“This kind of research is increasingly under threat. It’s much easier for fundraisers and policymakers to say we will support scientists, but … tell us how it will improve outcomes in five years. When you’re doing real science, you have to be prepared to take the road where it takes you – and if you’re doing science, it’s hard to predict where the road is going to take you,” he said.

The three scientists plan to split the approximately $900,000 award equally.

Source: The Washington Post, October 7, 2019.


Hearn Jay Cho, MD, PhD

MMRF Appoints Hearn Jay Cho as Chief Medical Officer

The Multiple Myeloma Research Foundation (MMRF) has selected Hearn Jay Cho, MD, PhD, as its chief medical officer. In this role, he also will lead the Multiple Myeloma Research Consortium, a group of 25 institutions researching treatment options for patients with multiple myeloma.

Dr. Cho will continue to serve as associate professor of medicine at the Icahn School of Medicine at Mount Sinai and as attending physician within the Multiple Myeloma Service at the Mount Sinai Tisch Cancer Institute. He will also continue to manage his laboratory at Mount Sinai.

“We are excited to have Dr. Cho, with his talent, expertise, and experience as a leading physician in the myeloma field, join the MMRF. Dr. Cho will contribute to the MMRF’s mission to find a cure for each and every multiple myeloma patient,” said Paul Giusti, MMRF president and CEO.

Source: Multiple Myeloma Research Foundation press release, September 10, 2019.


Minsoo Kim, PhD

Researchers Receive $2.8 Million NIH Grant to Improve T-Cell Treatment

Minsoo Kim, PhD, from the Wilmot Cancer Institute in Rochester, New York, has received a $2.8 million award supporting his research to mitigate side effects associated with T-cell immunotherapy.

Dr. Kim will serve as principal investigator for a trial studying the off-target toxicity of bioengineered T cells that are re-infused into patients with cancer. Early research suggested that these T cells may “hide” in noncancerous tissues for several days, causing a natural immune system response that can damage vital organs. The researchers hope to find new ways to control this T-cell migration with less toxicity.

“This is already a very promising treatment, but if we can better understand the homing properties of the cells and steer them more quickly and directly toward the cancer, we may be able to minimize side effects and make the treatment more effective,” said Dr. Kim, who is a Dean’s professor of Microbiology and Immunology and of Pharmacology and Physiology. He is also coleader of Wilmot’s Cancer Microenvironment Research Program.

Alongside Dr. Kim, Richard Waugh, PhD, professor of Biomedical Engineering and Vice Provost for Research at the University of Rochester, will serve as co–principal investigator, and Patrick Reagan, MD, of Wilmot Cancer Institute, will serve as co-investigator. The NIH will fund the project through 2024.

Source: University of Rochester Medical Center news release, September 10, 2019.


Max D. Cooper, MD
Jacques Miller, PhD

Max Cooper and Jacques Miller Receive 2019 Albert Lasker Basic Medical Research Award

The Albert and Mary Lasker Foundation announced the winners of its Basic Medical Research Award: Max D. Cooper, MD, from Emory University School of Medicine in Atlanta, and Jacques Miller, PhD, from the Walter and Eliza Hall Institute of Medical Research in Melbourne.

The scientists were recognized for their discoveries in immunology. They first identified the two distinct types of lymphocytes, B and T cells, providing “the organizing principle of the adaptive immune system,” according to a press release announcing the award.

Their findings launched numerous advances in basic science and medicine, including the development of monoclonal antibodies, generation of antibody diversity, major histocompatibility complex (MHC) restriction for immune defense, antigen processing by dendritic cells, and checkpoint inhibition therapy for cancer.

Dr. Miller began his research in the 1960s, working with mice with lymphocytic leukemia. During that time, Dr. Cooper, a pediatrician, became interested in how certain children were unusually susceptible to infections.

Their research later dovetailed, and they were able to delineate the adaptive immune system’s two major branches, each of which performs distinct functions, “opening a new era of cellular immunology.” Their historic discoveries have enabled strategies using immune cells to treat a range of illnesses, including cancer, autoimmune conditions, immunodeficiency disorders, and more.

Source: Lasker Foundation press release, September 11, 2019.


Yiping Yang, MD, PhD

Ohio State Selects Yiping Yang as Director of the Division of Hematology

The Ohio State University Comprehensive Cancer Center – Arthur G. James Cancer Hospital and Richard J. Solove Research Institute (OSUCCC – James) and The Ohio State University College of Medicine have appointed Yiping Yang, MD, PhD, director of the Division of Hematology.

Previously, Dr. Yang served as codirector of the hematologic malignancies and cell therapies program and professor of medicine/immunology at the Duke Cancer Institute and Duke University School of Medicine, where he was a faculty member since 2002. He specializes in the treatment of lymphoma, leukemia, and virus-associated malignancies, and his research focuses on viral immunity and tumor immunology.

Source: The Ohio State University Comprehensive Cancer Center press release, September 9, 2019.


FDA Awards 12 New Research Grants for Rare Diseases, Including AML

The U.S. Food and Drug Administration (FDA) has awarded 12 new grants totaling more than $15 million for the development of medical devices, drugs, biologics, and medical foods for patients with rare diseases, including acute myeloid leukemia (AML).

Awarded through the congressionally funded Orphan Products Clinical Trials Grants Program, the grants will contribute to marketing approval for products for rare diseases and provide clinical data necessary for their development.

As part of the selection process, more than 100 rare disease experts reviewed 89 clinical trial grant applications. Several grants researching hematologic disorders were funded. Cincinnati Children’s Hospital Medical Center received $1.7 million over four years to support a phase II study of chemoprevention with quercetin, a plant flavonol, for squamous cell carcinoma in patients with Fanconi anemia. The agency also awarded $1 million over four years to MD Anderson Cancer Center to support a phase I/II trial looking at the highest-tolerated dose of an imipridone (a class of drugs that targets G-protein coupled receptors) alone and combined with low-dose cytarabine for patients with relapsed/refractory AML, acute lymphocytic leukemia, or myelodysplastic syndromes.

Institutions also have received funding for studies related to daily vitamin D for patients with SCD-related respiratory complications, gastrointestinal stromal tumor, T-cell immunodeficiency, and more.

Source: FDA news release, October 8, 2019.


ASH Members Elected to the National Academy of Medicine

The National Academy of Medicine (NAM) has elected 100 new members, including six ASH members, one of whom is a past president of ASH, Charles S. Abrams, MD. Election to NAM 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. The six ASH members elected to NAM are:

  • Charles S. Abrams, MD, Francis C. Wood Professor of Medicine, Departments of Medicine, Pathology, and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA
  • Marina Cavazzana, MD, PhD, Professor of Hematology, Paris University Medical School; Head of the Biotherapy Department, Hôpital Necker-Enfants Malades, Assistance Publique – Hôpitaux de Paris; and Director, Clinical Investigation Center for Innovative Therapies, Imagine Institute, Paris, France
  • Margaret Anne Goodell, PhD, Chair, Department of Molecular and Cellular Biology, and Professor, Center for Cell and Gene Therapy, Baylor College of Medicine, Houston, TX
  • Stephan A. Grupp, MD, PhD, Novotny Professor of Pediatrics, Perelman School of Medicine, University of Pennsylvania; Section Chief, Cellular Therapy and Transplant, Division of Oncology, and Director, Cancer Immunotherapy Program, Children’s Hospital of Philadelphia, Philadelphia, PA
  • John Eu-Li Wong, MBBS, Isabel Chan Professor in Medical Sciences and Senior Vice President (health affairs), National University of Singapore; and Chief Executive, National University Health System, Singapore
  • Catherine J. Wu, MD, Professor of Medicine, Harvard Medical School, and Chief, Division of Stem Cell Transplantation and Cellular Therapies, Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA

Source: NAM press release, October 21, 2019.


James Kochenderfer, MD

Foundation for NIH Selects 2019 Trailblazer Prize Recipient

The Foundation for the National Institutes of Health (FNIH) has awarded the second annual Trailblazer Prize for Clinician-Scientists to James Kochenderfer, MD, of the National Cancer Institute. Dr. Kochenderfer is recognized for developing immunotherapies using chimeric antigen receptor (CAR) T cells to treat lymphoma and multiple myeloma.

The Trailblazer Prize recognizes outstanding contributions of early-career clinician-scientists whose work has the potential to or has led to innovations in patient care and seeks to raise awareness of the critical role the clinician-scientist plays in biomedical research and clinical care. As winner of the Trailblazer Prize, Dr. Kochenderfer will receive a $10,000 honorarium.

Source: Foundation for the National Institutes of Health press release, October 24, 2019.


Australian Scientists Win 2019 Prize for Innovation for Venetoclax Development

The Prime Minister’s Prize for Innovation, Australia’s pre-eminent award for scientific research, was presented to four scientists in recognition of their work on the BCL2 inhibitor venetoclax. The awardees include: Associate Editor and incoming Deputy Editor of Blood Andrew Roberts, PhD; David Huang, PhD; Peter Czabotar, PhD; and Guillaume Lessene, PhD.

The recipients, all from Walter and Eliza Hall Institute in Melbourne, noted that work on venetoclax began during the 1980s, when then PhD student David Vaux, PhD, neglected an experiment on cancer cells over a weekend. When he returned to work, the cells were alive when they should have been dead. The scientists worked to uncover the protein that kept the cells alive, BCL2, and to understand how to counteract it, setting off decades of work in cancer treatment.

“I think what we’re doing with basic research is to try to understand these diseases much better, so we can develop much better, more specific, better tolerated, and less toxic therapies,” said Dr. Huang. “Many thousands of researchers globally are making a huge impact in this field. … We need to continue to encourage that.”

Drs. Roberts, Huang, Czabotar, and Lessene will share the $250,000 prize.

Source: ABC Science, October 16, 2019.

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