AA&MDSIF - News

For the last twenty years, the Aplastic Anemia & MDS International Foundation (AA&MDSIF) has been awarding research grants to new researchers to inspire these scientists to stay in research and encourage them to work on projects relating to bone marrow failure disease. Recently, Mason Dunham, an AA&MDSIF college intern studying chemistry and economics at the University of Maryland, College Park, wrote this story after meeting with one of AA&MDSIF's first research grantees, Dr. Leslie Biesecker. Currently at the National Institutes of Health, Dr. Biesecker won a new researcher grant from AA&MDSIF in 1992. In the interview, Dr. Biesecker told the story of how the grant came to him at a key point in his development as a physician-scientist, and the lasting effect it had on his career.

Dr. Biesecker's project took place while he was at

the University of Michigan. He was always interested in research, and began his research career doing experiments with metabolic diseases. During these experiments, he performed bone marrow transplants on test animals, which led to an interest in bone marrow stem cell transplantation and bone marrow failure. While at Michigan, he came across a paper on the possibility of using embryonic stem cells to regenerate the hematopoietic systems of irradiated mice. Noting that it would be "fantastic to have a way to replace dead or dying bone marrow," Dr. Biesecker worked on the idea, with the intent of someday using the technique to regenerate the hematopoietic systems of humans. Unfortunately, the project turned out to be more ambitious than Dr. Biesecker had realized, and he was unable to make much progress. When he told other researchers of his problems, he was almost universally told that the project was too ambitious, and to simply "forget about it."

Fortunately, at least one person still saw merit in the idea, and took it upon himself to mentor the young researcher and help him with the project. Dr. Biesecker remembers Dr. Stephen Emerson as "a good hematology-oncology faculty member" who noted that the first project was "too big of a leap" while urging Dr. Biesecker to "take a step back and�do something basic." The result was a project that focused on characterizing the signals that caused undifferentiated stem cells to grow and transform into blood cells.

However, even with a more focused project and a good mentor to keep him on course, Dr. Biesecker was still at a critical juncture in his career. Still caught in a world he characterized as "half-research, half-clinical service," he needed funding and support to continue to build his research skills and avoid being completely drawn into clinical work. He began looking for funding and realized that with some minor changes in focus, his project could be used to improve knowledge about the relationship between dysfunctional hematopoietic stem cells and aplastic anemia. Thus, in 1992, he applied for a Post-Doctoral Fellowship award from AA&MDSIF (which was then called the Aplastic Anemia Foundation of America). His project was judged to be the most meritorious application, and he was awarded a $25,000 grant from AA&MDSIF. Today, he remembers how important this event was to his development as a researcher, saying, "The grant was the resource that allowed me to keep moving forward." He also credits the grant with helping him get his first position at NIH and notes that his results went into a successful application for a grant at NIH and led to further work on embryonic stem cells.

Dr. Biesecker's research led to a greater understanding of the receptors and growth factors involved in the growth and maturation of hematopoietic stem cells. However, at the conclusion of his project, he found that his interest in stem cells and genomics was leading his research away from bone marrow failure diseases. The early 1990s were a time of great change in the field of genomics, with the beginning of the human genome project and many leaps forward in technology. Perceiving the need for researchers to work with the new technologies, Dr. Biesecker's research focus changed from bone marrow failure diseases to Mendelian disorders, genetic disorders that come from the mutation of a single gene; he has had much success in this field. "Clinical research is a funny business," said Biesecker looking back on his transition. "It's not purely what you can do and it's not what you should do, it's a curious hybrid of the two�the people who are most successful and solve the most problems and do the most good are the people who optimize that the best." Still, he is extremely grateful for the grant from AA&MDSIF, noting that without the grant it is unlikely he would ever have had the opportunity to work on bone marrow failure research at all.

Today, Dr. Biesecker is a senior investigator at NIH's Genetic Disease Research Branch and head of the Human Development section, positions earned after a long and successful research career. He also stays involved in helping young researchers in their careers, heading the Physician Scientist Development Program. He remembers his experience with the AA&MDSIF as "extremely positive" and credits some of his success to the grant.

Looking into the future, Dr. Biesecker sees a lot of potential in the type of stem cell research he did for the AA&MDSIF, saying that the combination of new technology and expanded facilities makes today the "perfect time for a young scientist who is interested in working on [bone marrow failure] to really jump in the stem cell field." As far as potential solutions go, Biesecker sees exciting possibilities in the directed differentiation of stem cells, as well as induced pluripotent cells, which are stem cells that come from the patients themselves and can develop into any fetal or adult cell type.

The course of Dr. Biesecker's career shows the immense difference that a research grant can give a young researcher, and the great results that may come from such a grant. The grant from the AA&MDSIF allowed Dr. Biesecker to remain in research, rather than become an exclusively clinical doctor. As a result, his research shed new light on the molecular basis of aplastic anemia, which has the potential to lead to many new discoveries, and he went on to have a stellar research career. His insights into the trials facing a new researcher have also inspired him to help many young researchers remain in research. Clearly, the positive effects of research go far beyond two years of funding and a single research project. They can help mold a researcher's entire career and lead to extraordinary results. Over the past twenty years, AA&MDSIF has funded over thirty projects like Dr. Biesecker's, and the results of those projects continue to produce important discoveries.