FEATURING: Christopher Porter, MD, Assistant Professor, Children’s Hospital Colorado, Aurora, Colorado

Debra Newman, PhD, BloodCenter of Wisconsin, Milwaukee, Wisconsin

Seeking Cures for Blood Disorders

Hematology is the study of blood in health and disease, including disorders of the red blood cells, white blood cells, platelets, bone marrow, and proteins involved in bleeding and clotting. Hematologists treat millions of Americans currently living with blood disorders including anemia, hemophilia, and deep-vein thrombosis as well as blood cancers such as leukemia, lymphoma, and multiple myeloma. Many of these disorders are deadly and costly, and federally funded research continues to be a major driver in the ongoing quest to develop new treatments and cures.

Over the past several decades, federally funded hematology research supported by the National Institutes of Health (NIH) has yielded significant advances: chronic myeloid leukemia has become a manageable disease that is easily treated; children are routinely cured of acute lymphocytic leukemia; and new types of blood thinners can effectively treat and prevent heart attacks.

One example of promising hematology research underway is work in the lab of Dr. Christopher Porter, a pediatric hematologist/oncologist at Children’s Hospital Colorado in Aurora. Christopher and his research team are using cutting-edge tools to examine the entire genome and identify genes that, when “turned off,” sensitize leukemia cells to chemotherapy. “The goal of the work in my lab is to identify new strategies for treating children with leukemia that will improve what we’re doing now and change the way that we take care of these kids,” said Christopher.

Bright Research, Bleak Funding

While the research in Christopher’s lab and in hundreds of other labs across the country is incredibly exciting, the funding outlook isn’t as bright. Following a decade of flat funding for NIH, the agency’s budget was slashed by more than 5 percent in 2013 as part of mandated federal budget sequestration. These cuts prevented NIH from funding approximately 700 competitive research project grants or “R01s” that year.i Christopher’s proposal was one such grant denied, despite promising preliminary results. As he explained:

My lab had been able to report exciting preliminary data with startup funding, but our institutional startup funds were starting to wane. We really needed supplemental funds to keep this project moving. While our initial application to NIH was scored high enough to have received funding in previous years, it was not within the current funding range.

Another victim of sequestration is Dr. Debra Newman, an investigator at the BloodCenter of Wisconsin in Milwaukee whose research focuses on the activation of platelets, small fragments of blood cells that help blood form clots. Like Christopher, Debra’s application for a NIH research project grant has also been denied despite its being scored as competitive in the peer review process. Debra weathered periods of scarce NIH funding twice in the 1990s, but finds today’s environment to be much more severe. According to Debra:

Most of the people I know have been affected – their research funding has decreased and, consequently, so has the size of their laboratories because they cannot afford to employ the same number of staff. Talented investigators have started to leave research and go on to other things because they can’t support a research operation without money to run it.

Band-Aids Won’t Stop Bleeding

The American Society of Hematology recently launched a grant program designed to provide critical interim support to hematology researchers like Christopher and Debra who applied for competitive grants from NIH but were denied funding due to austerity. The awards, called “ASH Bridge Grants,” are intended to help “bridge” talented investigators to their next grant, supporting their efforts to gather additional data that will strengthen their federal research applications.

While supplementary grant funding programs such as ASH’s are helpful, they in no way replace critical NIH funding that has been cut for hematology research. If NIH funding continues to be threatened, an alarming number of both new and more experienced hematology researchers will turn away from research careers, leaving precious scientific innovation at a standstill. As Debra explained:

I think a more serious effect of the NIH cuts is on the desire of young people to enter into a career in scientific research. When young people see their mentors struggling to maintain a research operation, it makes it a less desirable career option. This will have long-term consequences for advancement of science in the United States.

Debra has visited with her Members of Congress to discuss the importance of scientific research and NIH funding; however, she has observed a disconnect during her conversations. “Most of the lawmakers I speak with in Congress appear to believe that scientific research and NIH funding really are important,” she said. “But the ability to get that funding approved and available to scientists has been difficult – their actions are oftentimes times not exactly in lockstep with their attitudes,” she said.

American Society of Hematology

i Fact Sheet: Impact of sequestration on The National Institutes of Health. [U.S. National Library of Medicine] U.S. National Library of Medicine, 3 June 2014. Web. 19 Mar 2014.