TRND stimulates therapeutic development research collaborations among NIH and academic scientists, nonprofit organizations, and pharmaceutical and biotechnology companies working on rare and neglected illnesses. The program provides expertise and resources, working with research partners to move therapeutics through pre-clinical testing, including plans for clinical trials and submission of an IND application to the Food and Drug Administration. Read the latest news about these collaborations below.
NCATS’ TRND program is supporting two natural history studies on how specific rare diseases progress over time.
Researchers from NCATS’ Therapeutics for Rare and Neglected Diseases program and Agilis Biotherapeutics, Inc., collaborated to bring a gene therapy for the rare pediatric condition aromatic L-amino acid decarboxylase deficiency closer to market.
An experimental drug appears to slow the progression of Niemann-Pick disease type C1 (NPC1), a fatal neurological disease, according to results of a clinical study led by researchers at Eunice Kennedy Shriver National Institute of Child Health and Human Development, NCATS and their colleagues.
NCATS scientists and their colleagues have demonstrated how an investigational drug works against a rare, fatal genetic disease, Niemann-Pick type C1 (NPC1). They found that a closely related compound will activate an enzyme, AMPK, triggering a cellular “housekeeping” system that helps reduce elevated cholesterol and other accumulated fats that in the brains and livers of NPC1 patients, which are hallmarks associated with severe neurological problems.
Patient-focused translational science collaboration among government and academic scientists, patients, and their advocates advances a potential treatment for the rare disease Niemann-Pick type C1.
A study published online in Science Translational Medicine on Feb. 25, 2015, found that NCATS’ TRND and Bridging Interventional Development Gaps (BrIDGs) programs have led to reduced costs for developing new drugs to treat rare diseases and reduced financial risks for therapies targeting these disorders.
An NIH research team enters into an agreement with biotechnology company Vtesse, Inc., to advance treatments for Niemann-Pick disease type C and other lysosomal storage disorders. NCATS and Eunice Kennedy Shriver National Institute of Child Health and Human Development researchers will conduct studies with funding provided by Vtesse.
TRND researchers begins work on three new pre-clinical drug development projects aimed at finding treatments for rare blood disorders and infectious diseases. The research also is designed to provide insights that will broadly improve and accelerate the translational science process.
Baxter International’s BioScience business acquires a drug candidate developed by TRND researchers and collaborators to treat sickle cell disease. Aes-103 is the first drug candidate specifically developed to target the underlying molecular mechanism of sickle cell disease. Baxter now will advance the clinical development activities required for regulatory approval and commercialization. This is the first time a company has acquired a drug candidate developed with TRND program resources.
On Jan. 23, 2013, NIH initiated a Phase I clinical trial to evaluate the safety and effectiveness of cyclodextrin as a potential therapy for Niemann-Pick type C1. This progress is the result of the collaborative efforts of an award-winning, multidisciplinary team of experts from NCATS, the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), the National Institute of Neurological Disorders and Stroke (NINDS), and the National Human Genome Research Institute; Janssen Research & Development, LLC; Washington University in St. Louis School of Medicine; Albert Einstein School of Medicine, New York City; and University of Pennsylvania, Philadelphia.
TRND researchers working to repurpose a chemical substance as a potential therapeutic for Niemann-Pick disease type C1 advance a promising new treatment.Cyclodextrin, normally used as an inactive ingredient in certain formulated drug products, could be used to treat a rare, inherited disease characterized by progressive impairment of motor and intellectual functions in early childhood. Life expectancy often does not exceed an individual’s teenage years.
A collaborative research team, including nine experts from NCATS, was honored in September 2012 for work on an investigational treatment for Niemann-Pick disease type C1, a rare genetic disease of cholesterol storage that eventually leads to neurodegeneration. Comprising investigators from four NIH Institutes and one pharmaceutical company, the team won the Excellence in Technology Transfer Award for its work with 2-hydroxypropyl-β-cyclodextrin (HPβCD) as a potential treatment for a disease that has no therapy approved by the Food and Drug Administration.