Multiple sclerosis (MS) is a disease of the central nervous system in which the immune system attacks the brain and spinal cord, typically resulting in muscle weakness, problems with vision and coordination, pain, and in some patients, cognitive impairments. The disorder affects approximately 400,000 people in the U.S. and more than 2.5 million people worldwide. Patients with relapsing forms of MS are currently treated with one of three FDA-approved interferon beta agents or with glatiramer acetate.

Natural products have formed the basis of traditional medicine systems for thousands of years and have been the single most productive source of leads for the development of cancer drugs. This nomination describes the discovery of halichondrin B, a compound isolated from a species of marine sponge, and the subsequent preclinical and clinical research and development of a related synthetic compound into the novel cancer drug Eribulin. 

The emergence of bioinformatics tools, which integrate molecular biology and genomics with computer based information technology, is bringing about a revolution in our understanding of the molecular mechanisms underlying normal and dysfunctional biological processes. The microarray is one such tool that caused a paradigm shift in the manner in which researchers collect and analyze genetic data.

Representative of the Year

Tom Stackhouse, Ph.D., is Associate Director of the Technology Transfer Center at the National Cancer Institute (NCI). The Center serves as the focal point for the implementation of legislation relating to collaborative agreements and inventions for the NCI, including the Frederick National Laboratory of Cancer Research. Dr. Stackhouse oversees the satellite office located in the Frederick National Laboratory of Cancer Research, which provides a full range of technology transfer services to NCI’s scientists located in Frederick. 

Currently, there are no FDA-approved therapies for Niemann-Pick disease type-C1 (NPC). NPC is a rare lethal genetic lysosomal storage disorder that results in an accumulation of cholesterol in the liver and spleen and eventually leads to neurodegeneration. 2-hydroxypropyl-β-cyclodextrin (HPβCD) is a cyclodextrin typically used by the pharmaceutical industry as an excipient. Studies of NPC in animal models have shown that HPβCD can reduce the biochemical burden associated with NPC, improving neurological pathology, decreasing neurological dysfunction, and increasing lifespan. 

In April 2013, President Obama unveiled the “BRAIN” initiative, which called on the scientific community to better understand the human brain in an effort to treat, prevent, and cure neurological diseases. For example, anxiety, depression, substance abuse, and post-traumatic stress are a few pervasive neuropsychiatric diseases that afflict more than 150 million people in developed countries, and approximately 15 million of those are in the U.S.

The National Cancer Institute (NCI), in partnership with the nonprofit Center for Advancing Innovation (CAI) and the Avon Foundation for Women, launched The Breast Cancer Startup Challenge, a first-of-a-kind, international, university-based competition. Through the creation of startup companies, it represents a new
model to accelerate the transfer of federally funded inventions to the marketplace, specifically focused on increasing the volume of developing emerging breast cancer technologies.

Medical research to combat disease is now a global effort and one that now requires an international technology transfer effort to see that new discoveries from research are effectively developed and commercialized to reach patients. While the United States in general, and the National Institutes of health

Cancer vaccines harness the immune system to identify and destroy cancer cells, and are a promising new approach to fighting cancer. In contrast to preventative vaccines, cancer vaccines identify antigens from cancer cells and immunize cancer patients against those antigens to stimulate the body’s immune cells to attack and kill the cancer cells. The National Cancer Institute (NCI) has developed investigational cancer vaccines that induct a specific, targeted immune response against cancer cells expressing the brachyury protein.

Niemann-Pick disease, type C (NPC) is a lethal, neurodegenerative disorder that affects children. Presently, no therapies for NPC are approved by the Food and Drug Administration (FDA). Several studies have suggested the potential use of 2-hydroxypropyl-β-cyclodextrin (HPBCD) to treat NPC, but the critical studies and data required for an Investigative New Drug (IND) application to evaluate HPBCD were not available.