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Cancer is caused by the improper regulation of cascading signals, or pathways, within the cell. One of the most prevalent means of fighting cancer involves the development of small molecule drugs and biologics that target and bind various proteins to disrupt certain pathways. The Hedgehog pathway is involved in embryonic development and is activated in many different tumor types. Smoothened (SMO) is a protein that plays an integral role in this pathway. 

Most people are aware that anti-cancer treatments often have negative side effects, but patients are willing to tolerate these side effects for the potential life-saving effects of the treatment. However, some patients treated with the anti-cancer drug 5-Fluorouracil (5-FU) will have fatal reactions typically caused by cardiotoxicity. A life-saving diagnostic test to identify cancer patients who may experience 5-FU toxicity has been developed by scientists at the National Cancer Institute (NCI).

Most people are aware that anti-cancer treatments often have negative side effects, but patients are willing to tolerate these side effects for the potential life-saving effects of the treatment. However, some patients treated with the anti-cancer drug 5-Fluorouracil (5-FU) will have fatal reactions typically caused by cardiotoxicity. A life-saving diagnostic test to identify cancer patients who may experience 5-FU toxicity has been developed by scientists at the National Cancer Institute (NCI).

The Mid-Atlantic Region State and Local Economic Development Award recognizes successful initiatives that involve partnership between state or local economic development groups and federal laboratories for economic benefit in the region. 

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.

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.