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 preclinical 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’ Preclinical Collaboration Enables Gene Therapy for Rare Muscle Disease to Advance to Clinical Trial
NCATS Therapeutics for Rare and Neglected Diseases (TRND) researchers and scientists at Duke University’s Clinical and Translational Science Institute (CTSI) have helped advance a gene therapy for Pompe disease into clinical testing for the first time.
Hibernating Ground Squirrels Provide Clues to New Stroke Treatments
NCATS and NINDS scientists collaborate to find a molecule that may protect brain cells during a stroke.
FDA Awards Six Grants for Natural History Studies in Rare Diseases
NCATS’ TRND program is supporting two natural history studies on how specific rare diseases progress over time.
Gene Therapy for Rare Pediatric Condition Moves Closer to Reaching U.S. Patients
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.
Experimental Treatment for Niemann-Pick Disease Type C1 Appears Safe, Effective
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.
NIH Scientists Find Rare Disease Clues in Cell's Recycling System
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.
NIH, Academia and Patient Advocate Collaboration Speeds Niemann-Pick Type C1 Research
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.
Study Demonstrates Success of NCATS’ Rare Diseases Therapeutic Development Programs
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.
NIH Teams with Industry to Develop Treatments for Niemann-Pick Type C Disease
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.
NCATS Announces New Research Projects to Improve Treatments for Rare and Neglected Diseases
TRND researchers begins work on three new preclinical 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.
First Drug Candidate From NIH Program Acquired by Biopharmaceutical Company
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.
NCATS Research Team Identifies Possible Treatment for Niemann-Pick Type C1
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 Research Leads to NIH Trial to Test Drug for Niemann-Pick Type C1
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.
NCATS Collaborative Project Wins Award for Excellence in Technology Transfer
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.