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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.