Corporate Banner
Satellite Banner
Technology
Networks
Scientific Communities
 
Become a Member | Sign in
Home>News>This Article
  News
Return

NIH Scientists Pursue New Therapies to Improve Rare Disease Drug Development

Published: Friday, September 13, 2013
Last Updated: Friday, September 13, 2013
Bookmark and Share
Projects selected for potential to treat specific rare diseases.

Four new pre-clinical drug development projects at the National Institutes of Health will target a form of blindness and diseases characterized by cardiac problems.

The projects were selected for their potential to treat specific rare diseases and to help scientists uncover new information that can be shared with other researchers.

The studies will be funded through the Therapeutics for Rare and Neglected Diseases (TRND) program at the NIH's National Center for Advancing Translational Sciences (NCATS).

This group of projects also marks the TRND program's first use of stem cells as well as its first collaboration with a large pharmaceutical company, Eli Lilly, to co-develop a treatment for a rare disease.

"TRND is grounded in partnerships with academic, government, pharmaceutical and patient advocacy groups. Working in collaboration, scientists conduct pre-clinical development of new drugs and then advance them to first-in-human clinical trials," said NCATS Director Christopher P. Austin, M.D. "Like all NCATS programs, TRND seeks to develop new technologies and more efficient paradigms for translation, in the context of important unmet medical needs."

Two projects employ therapeutic approaches to developing a treatment for retinitis pigmentosa, a severe form of hereditary blindness.

A third project focuses on a potential treatment for hypoparathyroidism, a hormone-deficiency syndrome that can lead to cardiac problems and convulsions.

The remaining project aims to develop a possible therapeutic that targets a cardiac disorder associated with LEOPARD syndrome, an extremely rare genetic disease that affects many areas of the body.

About 80 percent of patients with LEOPARD syndrome have a cardiac disorder called hypertrophic cardiomyopathy, which is a thickening of the heart muscle that forces the heart to work harder to pump blood, which can lead to early death.

A rare disease is one that affects fewer than 200,000 Americans. NIH estimates that, in total, there are more than 6,000 rare diseases. However, effective pharmacologic treatments exist for only about 200 of these illnesses.

Private companies often do not pursue new therapies for rare diseases due to the low anticipated return on investment. Through TRND, NCATS advances potential treatments for rare and neglected tropical diseases to first-in-human trials, an approach known as "de-risking."

This strategy can make possible new drugs more commercially viable and attractive to outside partners, who can invest in their further development and additional clinical trials. The new projects are:

Long-acting Parathyroid Hormone Analog for the Treatment of Hypoparathyroidism
Henry U. Bryant, Ph.D., distinguished research fellow Lilly Research Laboratories, Eli Lilly and Company, Indianapolis

Use of Rapamycin for the Treatment of Hypertrophic Cardiomyopathy in Patients With Leopard Syndrome
Maria I. Kontaridis, Ph.D., assistant professor of medicine Beth Israel Deaconess Medical Center, Boston

Use of Retinal Progenitor Cells for the Treatment of Retinitis Pigmentosa
Henry J. Klassen, M.D., Ph.D., director, Stem Cell and Retinal Regeneration Program University of California, Irvine School of Medicine

Small Molecule Pharmacological Chaperone for the Treatment of Retinitis Pigmentosa
William F. Brubaker, Ph.D., chief executive officer Bikam Pharmaceuticals, Inc., Cambridge, Mass.

The project descriptions are available at (http://www.ncats.nih.gov/trnd-projects.html).

TRND partners do not receive grants. The collaborative project teams receive in-kind support and gain access to TRND researchers with rare disease drug development capabilities, expertise, and clinical and regulatory resources.

Each project has established data-driven milestones to track progress. TRND staff may choose to discontinue projects that do not achieve milestones in the established timeframe. This allows other more promising candidates to enter the program.

"We are particularly excited about this set of collaborative projects for its potential to produce treatments for underserved patient populations," said John C. McKew, Ph.D., acting director of NCATS Division of Pre-Clinical Innovation and director of the TRND program.

A number of early projects fostered by TRND have reached the stage where partners, such as pharmaceutical, biotechnology or disease groups, are being sought to move the treatments out of TRND and in the next phases of clinical development.

Through the program in the last two years, TRND researchers and collaborators have advanced four projects to human clinical trials, evaluating treatments for sickle cell disease, chronic lymphocytic leukemia, hereditary inclusion body myopathy and Niemann-Pick Type C.

TRND projects are applied for via a solicitation process, and NCATS currently is accepting applications until September 30, 2013.


Further Information

Join For Free

Access to this exclusive content is for Technology Networks Premium members only.

Join Technology Networks Premium for free access to:

  • Exclusive articles
  • Presentations from international conferences
  • Over 3,300+ scientific posters on ePosters
  • More Than 4,900+ scientific videos on LabTube
  • 35 community eNewsletters


Sign In



Forgotten your details? Click Here
If you are not a member you can join here

*Please note: By logging into TechnologyNetworks.com you agree to accept the use of cookies. To find out more about the cookies we use and how to delete them, see our privacy policy.

Related Content

NIH Funds Precision Medicine
NIH have committed roughly $31M to launch a new program for Transdisciplinary Collaborative Centers for health disparities research.
Friday, July 29, 2016
Zika Vaccine Candidates Show Promise
Two experimental vaccines have shown promise against a major viral strain responsible for the Brazilian Zika outbreak.
Friday, July 29, 2016
New Medication Shows Promise Against Liver Fibrosis in Animal Studies
Liver fibrosis is a gradual scarring of the liver that puts people at risk for progressive liver disease and liver failure.
Thursday, July 28, 2016
NIH Begins Yellow Fever Vaccine Trial
NIH has initiated an early-stage clinical trial of a vaccine to protect against yellow fever.
Thursday, July 28, 2016
Targeting Autoimmunity
Researchers have developed a strategy to treat a rare autoimmune disease which could lead to treatments of other autoimmune diseases.
Wednesday, July 27, 2016
Molecule May Affect Gaucher, Parkinson's Disease
Research has identified a molecule that restores activity of a dysfunctional enzyme linked to Gaucher and Parkinson's disease.
Wednesday, July 27, 2016
Uncovering Rhinovirus C Structure
Researchers have determined the structure of rhinovirus C. Their findings may aid the development of antiviral therapies and vaccines.
Wednesday, July 27, 2016
Vaccine Strategy Targets Multiple Influenza Viruses
Scientists have identified vaccine-induced antibodies that can neutralize strains of influenza virus that infect humans.
Monday, July 25, 2016
Connectome Map More Than Doubles Human Cortex’s Known Regions
Researchers at NIH have developed software that automatically detects the “fingerprint” of each of these areas in an individual’s brain scans.
Saturday, July 23, 2016
Uncovering a New Principle in Chemotherapy Resistance in Breast Cancer
The NIH study has revealed an entirely unexpected process for acquiring drug resistance that bypasses the need to re-establish DNA damage repair in breast cancers that have mutant BRCA1 or BRCA2 genes.
Thursday, July 21, 2016
Brain Circuits Helps People Cope With Stress
Researchers at NIH have identified brain patterns in humans that appear to underlie “resilient coping,” to stress that help some people handle stressful situations better than others.
Wednesday, July 20, 2016
NIH Investment Into HIV Research Expands
Funding has been awarded to six research teams to lead collaborative investigations worldwide toward an HIV cure.
Thursday, July 14, 2016
Treatment Advancement for Gaucher and Parkinson's Diseases
NIH scientists identify molecule that may act as a possible treatment of neurological diseases.
Wednesday, July 13, 2016
Use it or Lose it: Visual Activity Regenerates Links Between Eye, Brain
The mouse study is first to show visual stimulation helps re-wire visual system and partially restores sight.
Tuesday, July 12, 2016
NIH Funds Million-Person Medicine Study
NIH announces $55million in awards to build foundations for ambitious Cohort Program that aims to engage 1 million participants in lifestyle, environments and genetics research.
Friday, July 08, 2016
Scientific News
Breakthrough Flu Vaccine Inhibited by Pre-existing Antibodies
Universal truths – how existing antibodies are sabotaging the most promising new human flu vaccines.
Gene Therapy for Metabolic Liver Diseases
Researchers have tested gene therapy in pigs from hereditary tyrosinemia type 1, with corrected liver cells being transplanted into the diseased liver.
Zika Vaccine Candidates Show Promise
Two experimental vaccines have shown promise against a major viral strain responsible for the Brazilian Zika outbreak.
New Medication Shows Promise Against Liver Fibrosis in Animal Studies
Liver fibrosis is a gradual scarring of the liver that puts people at risk for progressive liver disease and liver failure.
Raw Eggs Deemed Safe to Eat
A report published today by the Advisory Committee on the Microbiological Safety of Food (ACMSF) into egg safety has shown a major reduction in the risk from salmonella in UK eggs.
Monitoring TTX Toxin in Shellfish
In a number of small studies, mussels and oysters from the eastern and northern part of the Oosterschelde in Holland were found to contain tetrodotoxin (TTX).
Gene Terapy for Muscle Wasting Developed
New gene therapy could save millions of people suffering from muscle wasting disease.
NIH Begins Yellow Fever Vaccine Trial
NIH has initiated an early-stage clinical trial of a vaccine to protect against yellow fever.
Gene-Editing 'Toolbox' Targets Multiple Genes Simultaneously
Researchers have designed a system that modifies, or edits, multiple genes in a genome at once while minimising unintentional effects.
Detecting Alzheimer's with Smell Test
Odour identification test may offer low-cost alternative for predicting cognitive decline and detecting early-stage Alzheimer’s disease.
Scroll Up
Scroll Down
Skyscraper Banner

Skyscraper Banner
Go to LabTube
Go to eposters
 
Access to the latest scientific news
Exclusive articles
Upload and share your posters on ePosters
Latest presentations and webinars
View a library of 1,800+ scientific and medical posters
3,300+ scientific and medical posters
A library of 2,500+ scientific videos on LabTube
4,900+ scientific videos
Close
Premium CrownJOIN TECHNOLOGY NETWORKS PREMIUM FOR FREE!