In 1972, the National Hemophilia Foundation (NHF) introduced the Judith Graham Pool Postdoctoral Research Fellowship, named after the legendary researcher who discovered a method to extract clotting factor from human plasma to formulate cryoprecipitate.
Over the years, the JGP fellowship has funded the basic science and preclinical research of young scientists, often launching their careers. The projects of the 104 researchers who have received the award have produced vital insights into hemophilia and other bleeding disorders, contributing to the development of safer concentrates and enhanced methods of testing and screening, and laying the groundwork for current advances in gene therapy for hemophilia.
Unlike many grant programs, funding comes from donations from NHF chapters and individuals. The award provides up to $52,000 a year for a maximum of two years, and recipients are expected to spend at least 80% of their time on the funded research project.
Here, five awardees share how the fellowship changed their careers, powering research that has affected countless lives of people with bleeding disorders.
Clotting Factor Pioneer
Like many scientists, Gordon Vehar, Ph.D., got where he is today through a combination of hard work and happenstance. He was finishing his doctorate in biological chemistry from the University of Cincinnati College of Medicine when his Ph.D. supervisor, who grew up in Seattle, recommended he do his postdoctoral work at the University of Washington in a lab focused on coagulation. Not just any lab. The preeminent lab in the field of hemophilia, one whose director, Earl Davie, Ph.D., was among the first in the world to propose the coagulation cascade and isolate factor XIII.
“I knew nothing about coagulation, nothing about Seattle,” Vehar says. “It was totally circumstance.” Circumstance that eventually changed the lives of people with hemophilia.
Vehar’s postdoctoral work introduced him not only to the biochemistry of coagulation, but also to the real people affected by it — those with hemophilia. “I knew hemophilia was a disease of coagulation, but if you’re not a doctor treating patients, it’s just cold, hard chemicals and test tubes,” he says. To counter that, the lab’s director hired students with hemophilia to work there. “We got to meet them and see some of the joint damage that was going on and how it impacted them,” Vehar says. “That made it personal.”
His mentor encouraged him to apply for the Judith Graham Pool fellowship. The topic: “Structure and Function of Factor VIII.” It would be the only grant Vehar would write in his career, and it would end up changing the world of hemophilia. Just nine years after winning the grant, he led the scientific team at Genentech that cloned the gene for factor VIII, enabling the production of an artificial, genetically engineered clotting factor that transformed the lives of people with hemophilia.
Today, Vehar is vice president of external innovation at BioMarin Pharmaceutical Inc., in San Rafael, California, where he is responsible for identifying, evaluating, and overseeing all externally funded early research programs. His name is on more than 40 patents and dozens of scientific publications.
First Time’s the Charm
When Bin Zhang received his Ph.D. from Michigan State University, he wanted to study something directly relevant to human disease. So instead of finding a postdoctoral position in the field he was working in — spore formation in bacteria — he joined the lab of David Ginsburg, M.D.,* one of the country’s premier researchers on blood clotting, at the University of Michigan in Ann Arbor and entered the world of bleeding disorders.
Zhang quickly latched onto a project, which involved finding the gene that causes a rare bleeding disorder called combined deficiency of factor V and factor VIII. When Ginsburg told him about the Judith Graham Pool fellowship, Zhang knew his work was well within the scope of the award. He was also aware of the prestigiousness of the fellowship and what it could mean for his future. So, he put together his first-ever grant proposal and won.
“I was excited to receive the award,” he says. “It was an encouragement and recognition of the significance of my work by experts in the field.” The fact that he could write a grant and get the award “told me that I have potential, that I could go on with an academic career.”
The fellowship was the first domino to fall. Thanks to it, he won NHF’s three-year Career Development Award and funding from the March of Dimes. When it was time to apply for a job, he received several offers and picked the Lerner Research Institute at Cleveland Clinic, where he runs his own laboratory focused on early secretory pathway deficiencies in human diseases, the same thing responsible for combined deficiency of FV and FVIII.
Zhang is also paying forward his good fortune. In 2021, two of his postdoctoral Ph.D.s applied for and won the JGP fellowship. “The award is very important in supporting young scientists,” he says.
Essential Support Early in a Career
Lacramioara Ivanciu, Ph.D., received the Judith Graham Pool fellowship soon after starting her postdoctoral training at Children’s Hospital of Philadelphia in the lab of Rodney Camire, Ph.D., a pioneer in the basic science of coagulation.
She was thrilled to learn of her selection for the award. “It was a starting point for my academic career in basic and translational research in bleeding disorders,” she says. Her goal was to generate recombinant coagulation for factor X variants and evaluate their therapeutic potential for the treatment of hemophilia in acute situations. “This work was significant for the preclinical development of FX variants as bypass agents for treatment of hemophilia A and B,” she says. Ivanciu published the findings from her fellowship work in the journal Nature Biotechnology.
Obtaining the fellowship provided her with the necessary support to acquire skills and knowledge related to her career development. “That had a major contribution to my faculty appointment,” she says. It helped her get other grants as well, and today she is starting her own laboratory at the Perelman School of Medicine at the University of Pennsylvania and Children’s Hospital of Philadelphia, focusing on explaining the regulation of clot formation in vivo.
“The National Hemophilia Foundation and the JGP and their other grants provide excellent support for early career investigators,” she says. “They also provide opportunities for therapeutic development that will have a substantial effect on the quality of life of people living with bleeding disorders.”
Her advice to young scientists is to go for it. “Apply for these grants, because they can really make an impact on your career,” she says. “Trust in yourself. Do what makes you passionate.”
A Happy Change in Direction
Andrew Yee, Ph.D., thought he wanted to be a chemical engineer. At least, that’s what he received his Ph.D. in. Then he spent some time in a bioengineering lab looking at endothelial cells, and suddenly, “I wanted to learn about biology and genetics,” he says. “I wanted to work with animals.” This led him to a postdoctoral position with David Ginsburg, M.D., at the University of Michigan.
Yee immediately launched into a project to identify all of the genetic variants in von Willebrand factor (VWF) that caused bleeding disorders. Then Ginsburg told him about the JGP fellowship. He read about Pool’s discovery, which piqued his interest in factor VIII. Specifically, he wondered, how did it interact with VWF?
So, he applied for the JGP fellowship with a research proposal called “Fine Structure-Function Mapping VWF-FVIII Interaction.” It was one of the first grants he’d ever applied for and the first he won.
“I was elated when I got the news that I won,” says Yee, who is now an assistant professor in the Department of Pediatrics, Division of Hematology-Oncology at Baylor College of Medicine in Houston. Not just because he now had funding to pursue his idea for two years, but because of the input he received from the reviewers. “Their suggestions made quite an impact on how I pursued the experiments,” he says.
Yee used his JGP award to develop reagents required for the complex studies in animal models he conducted to explore VWF and FVIII interactions — work that continues today. An added benefit of the award was being able to work with other JGP awardees. “We’re a small community,” he says. “It’s an incredible honor to be a JGP award recipient.”
Although he completed his medical degree and clinical fellowship in hematology/oncology, Sol Schulman, M.D., Ph.D., says it’s the research that “gets me up in the morning.” So, while completing his hematology/oncology fellowship in 2015, he began looking for independent funding to pursue his research interests, which is when he heard about the Judith Graham Pool grant from a previous awardee. “He’d had a positive experience and highly recommended it,” Schulman says. “It seemed like a perfect fit for my interest.”
“A lot of times when you’re applying for these types of awards, people are trying to fit a square peg into a round hole,” he says. “But I felt, wow, this is the right award for the right stage of my training.”
Schulman says the JGP fellowship, which funded his project looking at the role of protein disulfide isomerase in activating clotting, was critical to where and who he is today.
“Most people interested in pursuing research careers in medicine find themselves in a transition around the end of clinical fellowship; suddenly there’s this great abyss out there,” he says. He knew he could focus on clinical medicine and have the security of a salary and academic position. But he also knew the pressures of full-time clinical work would leave little time for the research he yearned to do. The JGP grant, he says, convinced him he could become an independent researcher and bring in his own money.
“It not only provided me with protected time to do the research I was excited about and gain mentors, but it was also a validation at the national level that I was doing something interesting and important,” he says.
Today, Schulman is a principal investigator in the Division of Hemostasis and Thrombosis at Beth Israel Deaconess Medical Center in Boston. His lab works to integrate functional genomics, human genetics, biochemistry, and cell biology to identify new genes and pathways that influence blood coagulation.
* EDITOR’S NOTE: In the print version of this article in the Fall/Winter 2022-23 issue of HemAware, Dr. David Ginsburg’s name was spelled incorrectly.