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ASH Remembers Joseph Bertino, National Academy of Medicine Elects Helen Heslop, and more

December 6, 2021

December 2021

Joseph R. Bertino, MD

Remembering Joseph Bertino (1930 – 2021)

Pioneering oncologist Joseph R. Bertino, MD, died on October 11, 2021, at the age of 91.

In 1961, Dr. Bertino began his career in medicine at Yale University as professor of medicine and pharmacology and the first director of the Yale Cancer Center. He moved on to become chairman of the Molecular Pharmacology and Therapeutics Program at Memorial Sloan Kettering Cancer Center in 1987. In 2002, he joined the Robert Wood Johnson Medical School to help grow the Cancer Institute of New Jersey, holding the positions of associate director, chief scientific officer, and interim director.

Dr. Bertino’s research ultimately led to the first curative combination treatment for patients with lymphoma. He and his colleagues also observed gene amplification through their finding that cells can multiply genes coded for dihydrofolate reductase, the target for methotrexate. Previously, DNA was believed to be stable.

“He was a beloved mentor and friend to hundreds of physicians, pharmacologists, scientists and researchers, many of whom have themselves become leaders in the field,” his colleagues at Rutgers Cancer Institute wrote in his obituary. “He led by example, a kind and compassionate physician, outstanding teacher, and a brilliant investigator. Joe created a ‘translational’ research environment long before it was in vogue, bringing together clinical oncologists and basic scientists to tackle key problems in the diagnosis and treatment of cancer.”

Dr. Bertino is survived by his brother as well as his four children and their families. He is predeceased by his wife, Mary Patricia, and another brother.

FDA and NIH Partner With Private Organizations to Form Consortium to Further Gene Therapies for Rare Diseases

The U.S. Food and Drug Administration (FDA), the National Institutes of Health (NIH), 10 pharmaceutical companies, and five nonprofit organizations have formed a partnership with the goal of developing gene therapies for the treatment of rare diseases. As part of NIH’s Accelerating Medicines Partnership program, the Bespoke Gene Therapy Consortium (BGTC) will be managed by the Foundation for the NIH.

Thirty million Americans are currently diagnosed with about 7,000 rare diseases, but only two heritable rare diseases have FDA-approved gene therapies.

“Rare diseases affect 25 to 30 million Americans, but because any given rare disorder affects so few patients, companies often are reluctant or unable to invest the years of research and millions of dollars necessary to develop, test and bring individualized gene therapy treatments for a single disease to market,” said Joni L. Rutter, PhD, acting director of NIH’s National Center for Advancing Translational Sciences, a public sector partner of the BGTC. “The BGTC aims to make it easier, faster and less expensive to pursue bespoke gene therapies in order to incentivize more companies to invest in this space and bring treatments to patients.”

Other public sector partners include the National Institute for Neurological Disorders and Stroke, the National Human Genome Research Institute, and the National Heart, Lung and Blood Institute.

BGTC researchers will explore the biologic and mechanistic steps involved in adeno-associated virusF gene delivery vector production and vector delivery of genes into human cells, as well as how therapeutic genes are activated in target cells. In addition, they will examine methods for streamlining regulatory processes for FDA approval of gene therapies.

Over five years, the NIH and private partners will contribute approximately $76 million toward BGTC-funded projects.

Source: FDA press release, October 27, 2021.

Helen E. Heslop,
MD, DSc (Hon)

Helen Heslop Elected to the National Academy of Medicine

During its annual meeting in October, the National Academy of Medicine (NAM) announced the election of 100 new members, including hematologist Helen E. Heslop, MD, DSc (Hon).

Dr. Heslop was selected for pioneering work in complex biological therapies, leadership in clinical immunotherapy, and for being the first to employ donor and banked cytotoxic T cells to treat lethal virus-associated malignancies and infections in pivotal trials. She is Dan L. Duncan Chair, professor of pediatrics and medicine, and director of the Center for Cell and Gene Therapy at Baylor College of Medicine in Houston, Texas.

“This is … the [academy’s] most diverse class of new members to date, composed of approximately 50% women and 50% racial and ethnic minorities. This class represents many identities and experiences, all of which are absolutely necessary to address the existential threats facing humanity,” NAM President Victor J. Dzau, MD, said in a press release.

Source: NAM press release, October 18, 2021.

NIH Grants $17M to Washington University in St. Louis for Cancer Disparities Research

The NIH has granted $17 million to the Washington University School of Medicine in St. Louis to help address disparities in cancer research, treatment, and outcomes in populations that are underrepresented in clinical trials.

The NIH’s Cancer Moonshot program will fund the Washington University Participant Engagement and Cancer Genomic Sequencing Center, a research program focused on Black patients with multiple myeloma and colorectal cancer, as well as patients of any race or ethnicity with cholangiocarcinoma.

For each of the three cancer types, the researchers aim to enroll 300 patient volunteers to have their genomes sequenced and compared to healthy genomes.

Additionally, the researchers plan to interview patients who decline to participate. “As we’re learning from those who decline, we can bring those concerns back to the whole team and try to implement strategies to improve on the design of these types of studies in the future,” said cancer epidemiologist Bettina F. Drake, PhD, a professor of surgery in the division of public health sciences at the Washington University School of Medicine.

Source: Washington University in St. Louis press release, November 5, 2021.

Researchers Receive $13M to Track Cancer Risk From Environmental Exposures

Funded through a $13 million grant from the National Cancer Institute, researchers at the University of Michigan School of Public Health and Rogel Cancer Center have launched the Michigan Cancer and Research on the Environment Study (MI-CARES). This program will investigate the impact of known and suspected environmental exposures on cancer risk.

As a result of industrial contamination of lakes and rivers from the 1940s‒2000s, Michigan has the highest known per- and polyfluoroalkyl substances (PFAS) levels of any U.S. state. In addition, the state has a long history of environmental pollution, from animal feed contaminated with polybrominated biphenyls in the 1970s to lead and toxins in the city of Flint’s water supply.

The investigators plan to enroll at least 100,000 people from a variety of racial-ethnic backgrounds living in environmental hotspots throughout the state of Michigan. Using surveys as well as blood and saliva samples, participants in the study will be followed over time to track environmental exposures and cancer biomarkers.

“Many communities experience a disproportionate disease burden because of failed governmental stewardship of local environments and the prioritization of private enterprise over health protection. With growing awareness of the health threats of these decisions, it’s essential to put greater focus on environmental contaminants and public health safety,” said principal investigator Celeste Leigh Pearce, PhD, MPH, professor of epidemiology at the University of Michigan School of Public Health.

“With MI-CARES, we will examine well-established carcinogens such as certain components of air pollution and metals, but also focus on environmental contaminants with less data available to adequately assess risk, including PFAS. We will also study their effects together,” said co-principal investigator Bhramar Mukherjee, PhD, chair and John D. Kalbfleisch Collegiate Professor of Biostatistics at the School of Public Health and associate director of quantitative data sciences at Rogel Cancer Center.

Source: Rogel Cancer Center press release, October 20, 2021.


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