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Abnormal T lymphocyte (T cell) function, including tumor formation in T cells, can lead to a wide variety of diseases. Over 100 million people worldwide suffer from T cell autoimmune disorders of varying severity, such as lupus and graft-versus-host disease, and the incidence of T cell-mediated cancers, including lymphomas and leukemias, has risen over the past decade. These diseases can be deadly, and not all patients respond to standard therapies.

The term “cancer” is used to describe a collection of several diseases that are caused by the aberrant growth of cells and the invasion of these cells into other tissues, where they disrupt normal organ function. Cancers are commonly treated by surgical removal of the cancerous tissue, followed by a regimen of conventional chemotherapy or radiation therapy. Unfortunately, these conventional therapies lack specificity for cancer cells, leading to undesirable side effects that result from the non-specific killing of essential normal cells.

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. 

The National Institute of Neurological Disorders and Stroke (NINDS) and Lixte Biotechnology Holdings, Inc. (Lixte) are collaborating to identify and develop agents that target the Nuclear Receptor CoRepressor (N-COR) pathway. Dr. Zhengping Zhuang’s laboratory at NINDS has determined that several kinds of tumors, including glioblastomas and medulla blastomas, over-express N-CoR. 

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. 

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.