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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 (NIH) in particular, have long led the way in effective biomedical technology transfer, such is not the case with new or younger transfer programs at research institutions outside the US, especially those in emerging and middle-income countries.

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.

On March 10, 2015, the Food and Drug Administration (FDA) approved Unituxin™ (dinutuximab) as part of first-line therapy for pediatric patients with high-risk neuroblastoma, a rare cancer that most often occurs in young children. This approval was the result of a collaborative effort between the National Cancer Institute (NCI), the Children’s Oncology Group, and United Therapeutics Corporation (UTC) for the first approved therapy for pediatric high-risk neuroblastoma.

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 induce a specific, targeted immune response against cancer cells expressing the brachyury protein.

On March 10, 2015, the U.S. Food and Drug Administration (FDA) approved Unituxin™ (dinutuximab) as part of first-line therapy for pediatric patients with high-risk neuroblastoma, a rare cancer that most often occurs in young children. This approval was the result of a collaborative effort among the National Cancer Institute (NCI), the Children’s Oncology Group, and United Therapeutics Corporation (UTC) for the first approved therapy for pediatric high-risk neuroblastoma.