SAB Funding Recommendations

Scientific Advisory Board Funding Recommendations: General Research

Mark Sands, PhD

Members of the National MPS Society have done an extraordinary job over the years raising millions of dollars to both support the Society and fund research. As of 2008, the Society will have funded over $5 million in research. The money designated for research is critical since it supports scientists in the early stages of their careers and funds projects that are considered either too speculative or too clinical for consideration by the National Institutes of Health (NIH).

Many of the research projects funded by the National MPS Society have led to important discoveries. These discoveries have allowed investigators to secure larger grants from the NIH and have gotten us closer to the development of effective therapies for these disorders. Although it is tempting to direct the proceeds from an individual fund raiser towards the specific disease affecting the members family, this is not necessarily the most efficient means of advancing science. For example, investigators applying for grants through the General Research Mechanism may be using a model or technology that appears unrelated to a specific disease or even to MPS diseases as a group. However, some of the most innovative approaches and technologies that might drive the field forward by leaps-and-bounds or take the field in a new productive direction come from unexpected areas of research. In addition, given the similarities among these disorders, there is a good chance that a new technology or a discovery in one disease model will be applicable to other disorders.

The only way these innovative approaches can be funded is through the General Research Fund. Directing a significant portion of the research funds to disease-specific projects reduces the amount of money available for more generalized and possibly innovative projects. In addition, it limits the ability of the Scientific Advisory Board to reward promising new ideas or investigators. As our knowledge of biological mechanisms increases exponentially, it becomes even more important to make resources available for new approaches and technologies regardless of the source.

Mark Sands, PhD, is a Professor in the Departments of Internal Medicine and Genetics at Washington University School of Medicine in St. Louis. Missouri. Dr. Sands received his PhD in molecular and cellular pharmacology at the State University of New York at Stony Brook. The two main goals in his laboratory are to better understand the underlying pathophysiology of lysosomal storage diseases (LSD) and to develop effective therapies for this class of inherited metabolic diseases. Dr. Sands is a recipient of National MPS Society grants in 2001 for Human Hematopoietic Stem Cell-Directed Gene Therapy in a Murine Xenotransplantation Model of Mucopolysaccharidosis VII?, in 2004 for Characterization of the Systemic Inflammatory Response to Lysosomal Storage? and in 2008 for “Metabolic Adaptations and Phenotypic Consequences of Blocking Lysosomal Recycling”. Dr. Sands is the chair of the National MPS Society Scientific Advisory Board.