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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. 

Adeno-associated viruses (AAVs) are attractive delivery vectors in the field of gene therapy. A team from the National Institutes of Health (NIH) developed AAV5-based vectors for delivering gene therapy products into parts of human bodies. Gene therapy is on the brink of becoming a common medical practice; however, developing safe and effective gene therapy products has been challenging. One major issue has been finding a delivery vector to target the diseased tissues in the body without devastating side effects.

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. 

The 2012 Deals of Distinction™ Award was presented to the National Institutes of Health (NIH) along with the University of Illinois at Chicago (UIC), who jointly own one patent family, and to Gilead Sciences (Gilead) for license agreements granted to the Medicines Patent Pool, a newly established initiative of UNITAID, an international organization established to grant licenses for the generic manufacture and purchase of drugs against HIV/AIDS, malaria, and tuberculosis.

According to the World Health Organization, tuberculosis (TB) causes approximately 1.5 million deaths worldwide each year. More than one-third of the world’s population has tested positive for Mycobacterium tuberculosis (Mtb), the bacterium that causes TB, but are not yet ill (latent infection). Of these, approximately ten percent are expected to progress to active TB infection. Treatment is lengthy, and the drugs often come with serious side effects. This has led to poor treatment compliance and allowed the rise of drug-resistant TB strains. 

On July 20, 2012, a committee of the European Medicines Agency recommended for regulatory approval the first gene therapy using an adeno-associated virus vector to treat lipoprotein lipase deficiency (LPLD), a very rare genetic disease. With a normal diet, patients lacking sufficient levels of lipoprotein lipase have abnormally high serum triglycerides and high levels of very low-density lipoprotein (VLDL), resulting in acute severe pancreatitis and chronic conditions associated with high levels of VLDL, such as cardiovascular diseases. 

Malaria is a life-threatening disease transmitted through the bite of mosquitoes infected with malaria parasites. In 2012, there were an estimated 219 million cases of malaria and an estimated 660,000 deaths, mostly among young children in sub-Saharan Africa. With the effectiveness of current drugs diminishing as resistant strains of malaria have emerged, new drugs are urgently needed. The plasmodial surface anion channel (PSAC) found on the surface of red blood cells infected with malaria parasites offers an opportunity to develop new drugs to treat and prevent malaria.

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.

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.