Remembering Patti Massicotte, Nobel Prizes Awarded for Discovery of Hepatitis C and CRISPR, and more

Patti Massicotte, MD, FRCP

Remembering Patti Massicotte (1955 – 2020)

Mary Patricia “Patti” Massicotte, MD, FRCP, died on October 1, 2020 from cancer.

Dr. Massicotte was Professor and Director of Clinical Thrombosis in the Department of Pediatrics at the University of Alberta. An active clinician-researcher, she contributed significantly to the field of pediatric thrombosis and anticoagulation, including her role in the REVIVE and PROTEKT studies, which set the standard for randomized controlled trials of anticoagulation prevention and treatment strategies in children for nearly two decades.

Along with Mary Bauman, NP, Dr. Massicotte also influenced warfarin management for children through her involvement with the EMPOWERMENT studies. She was passionate about mentoring and conducting research to improve clinical outcomes for children.

She is survived by her four children: Kyle, Lauren, Michaela, and Liam.


Alok A. Khorana, MD

NBCA Appoints Alok Khorana as Medical & Scientific Advisory Board Chair

The National Blood Clot Alliance (NBCA) has named Alok A. Khorana, MD, as the new Chair of its Medical & Scientific Advisory Board (MASAB). In addition to his role as chair, Dr. Khorana will join NBCA’s Board of Directors, becoming the MASAB liaison with NBCA’s volunteer leadership and working with senior staff on the design and implementation of NBCA programs and services.

“It’s an honor to serve NBCA at a time when the clotting community is experiencing a spectrum of new challenges that require a sharpened focus on the public health impact of venous thromboembolism and life-threatening blood clots,” Dr. Khorana said. “My goal as MASAB Chair will be to ensure that the work we do as an advisory group aligns effectively with NBCA’s mission and contributes to improved understanding and care of all people affected by clotting and clotting disorders.”

Dr. Khorana is a Professor of Medicine at the Cleveland Clinic Lerner College of Medicine of Case Western University, the Sondra and Stephen Hardis Chair in Oncology Research and Vice Chair for Clinical Services at Cleveland Clinic’s Taussig Cancer Institute, and Director of the Gastrointestinal Malignancies Program at Cleveland Clinic.

Sources: Cleveland Clinic news release, July 15, 2020; NBCA press release, April 28, 2020.


Harvey J. Alter, MD
Emmanuelle Charpentier, PhD
Jennifer A. Doudna, PhD

Nobel Prizes Awarded for Discovery of Hepatitis C, CRISPR Gene Editing

The 2020 Nobel Prize in Physiology or Medicine was awarded jointly to Harvey J. Alter, MD; Michael Houghton, PhD; and Charles M. Rice, PhD, for their contributions to the identification of the hepatitis C virus, which led to life-saving diagnostic tests and antiviral treatments.

More than 40 years ago, the Nobel Prize in Physiology or Medicine was awarded to Baruch Blumberg, MD, DPhil, for his discovery of hepatitis B, which ultimately led to the development of diagnostic tests and a vaccine. At that time, Dr. Alter was studying transfusion-related hepatitis at the National Institutes of Health (NIH) and defined a novel form of chronic viral “non-A, non-B” hepatitis. A decade later, Dr. Houghton and his co-workers at the pharmaceutical firm Chiron began the work of isolating the genetic sequence of the hepatitis C virus. Dr. Rice, at Washington University in St. Louis, then found evidence that the hepatitis C virus alone could cause transfusion-related hepatitis. The three scientists’ work led to important improvements in global health through the development of highly sensitive blood tests and antiviral drugs capable of curing the virus today.

Dr. Alter, a longtime member of the American Society of Hematology (ASH), recently retired from his role as a transfusion medicine investigator at the NIH. Dr Houghton is currently a Canada Excellence Research Chair in Virology as well as the Li Ka Shing Professor of Virology and Director of the Li Ka Shing Applied Virology Institute at the University of Alberta. Dr. Rice has been a professor since 2001 and remains active in the Center for the Study of Hepatitis C at Rockefeller University.

The 2020 Nobel Prize in Chemistry was awarded to microbiologist Emmanuelle Charpentier, PhD, of the Max Planck Institute for Infection Biology, and biochemist Jennifer A. Doudna, PhD, of the University of California, Berkeley, for their 2012 discovery of the CRISPR gene-editing tool.

Dr. Charpentier’s first paper on CRISPR was published in Nature in 2011. She met Dr. Doudna at a scientific conference that same year. Together, they published their seminal paper in 2012, proving that bacterial enzyme Cas9 could cut purified DNA in test tubes and be combined with CRISPR-related RNAs that would lead Cas9 to any site on a DNA molecule.

In the years that followed, scientists built on their work to launch studies testing CRISPR-based gene editing’s ability to cure disorders such as sickle cell disease, beta thalassemia, congenital blindness, Duchenne muscular dystrophy, cystic fibrosis, type 1 diabetes, and hemophilia. The technology is also an indispensable tool for experimental hematologists, giving them an opportunity to identify genes of interest and delineate key coding and noncoding sequences.

The 2020 prize in chemistry is the first science Nobel Prize awarded to two women. “I wish that this will provide a positive message, specifically, to young girls who would like to follow the path of science,” Dr. Charpentier said.

“I think it’s great, especially for younger women, to see this and to see that women’s work can be recognized as much as men’s,” Dr. Doudna added.

Sources: The Nobel Foundation press release, October 5, 2020; The Washington Post, October 7, 2020.


New York Genome Center Awards Grants to Close Racial Gaps in Cancer Research

As part of its Polyethnic-1000 initiative to increase understanding of racial disparities in the prevalence of certain cancers, the New York Genome Center has awarded six research grants to teams at Weill Cornell, Cold Spring Harbor Laboratory, Northwell Health, SUNY Downstate Medical Center, SUNY Downstate Health Sciences University, Kings County Medical Center, Mount Sinai Hospital, and New York-Presbyterian.

The projects will investigate the role of ethnicity in bladder cancer; pancreatic, endometrial, and colorectal cancers in Black Americans; breast and prostate cancers in patients of African descent; and lung cancer in Asian Americans.

The research projects receiving grants include:

  • “Immunogenomic Determinants of Ethnic Disparities in Clinical Outcomes for Urothelial Cancer Patients,” Weill Cornell Medicine
  • “Molecular Determinants of Increased Vulnerability to Pancreatic Cancer Among African Americans,” Cold Spring Harbor Laboratory, Northwell Health, and SUNY Downstate Health Sciences University
  • “Ethnic-based Differences Between East Asian and Caucasian Patients in Genomic, Transcriptomic and Immune Profiles of Pre-invasive and Invasive Adenocarcinoma of the Lung,” Weill Cornell Medicine
  • “Molecular Links between Ancestry and Outcome Disparity in Breast and Prostate Cancer Patients Across the African Diaspora in New York City,” Weill Cornell Medicine, Icahn School of Medicine at Mount Sinai, and NewYork-Presbyterian Hospital
  • “Uncovering the Mechanisms of Colorectal Cancer Disparities in African Americans,” Cold Spring Harbor Laboratory and SUNY Downstate Health Sciences University
  • “Mechanisms of Endometrial Cancer Disparities in African Americans,” Cold Spring Harbor Laboratory, Weill Cornell Medicine, NewYork-Presbyterian Brooklyn Methodist Hospital, and Northwell Health

Source: New York Genome Center news release, September 8, 2020.