Corporate Banner
Satellite Banner
Genotyping & Gene Expression
Scientific Community
 
Become a Member | Sign in
Home>News>This Article
  News
Return

NIH Clinical Trial Begins for Treatment of Rare, Fatal Neurological Disorder

Published: Monday, April 29, 2013
Last Updated: Monday, April 29, 2013
Bookmark and Share
Government, industry, academia, and patient groups collaborate on Niemann-Pick Type C research.

A clinical trial to evaluate a drug candidate called cyclodextrin as a possible treatment for Niemann-Pick disease type C1 (NPC), a rare and fatal genetic disease, will start, researchers announced.

Scientists from the NIH's National Center for Advancing Translational Sciences (NCATS) and the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) will conduct the clinical trial at the NIH Clinical Center. Reaching this trial stage required collaboration among government, industry, patient advocacy groups and academic researchers.

No therapies approved by the U.S Food and Drug Administration are available to treat NPC. The disease is characterized by the inability of cells to metabolize and dispose of cholesterol and lipids.

It causes excessive amounts of cholesterol to accumulate within the liver, spleen and brain. NPC leads to progressive impairment of motor and intellectual function in early childhood. In childhood onset cases, life expectancy does not normally exceed a patient's teenage years.

"A crucial part of the NCATS mission is to collaborate within and beyond the NIH on projects to improve and accelerate the translational research process and deliver tangible improvements in human health," said NCATS Director Christopher P. Austin, M.D.

Austin continued, "The cyclodextrin project is an important step in the development of both a potential treatment for a devastating disease that ravages the bodies and minds of its victims and a more efficient way to do translational projects."

In 2009, the NIH Therapeutics for Rare and Neglected Diseases (TRND) program, which is now led by NCATS, selected NPC cyclodextrin as one of its initial pilot projects to repurpose cyclodextrin from its conventional use as an ingredient in other drugs to a therapeutic for this rare disorder.

TRND researchers work with project collaborators to conduct preclinical studies advancing potential treatments for rare and neglected diseases to human clinical trials.

TRND supported animal toxicology studies to evaluate the safety of cyclodextrin and all necessary regulatory efforts and also supported the development of an NPC biomarker.

The biomarker test detects in the blood a modified cholesterol molecule specific to neuronal cells in the brain that would increase as a result of treatment with cyclodextrin.

TRND researchers and collaborators submitted the data in an Investigational New Drug application, filed Nov. 14, 2012, that the Food and Drug Administration (FDA) has now agreed is sufficient to start a Phase I clinical trial.

"The multidisciplinary nature of this collaboration establishes a generalizable model that can be used in the pursuit of treatment candidates for rare and neglected diseases," said John McKew, Ph.D., acting director of the NCATS Division of Pre-Clinical Innovation, chief of the Therapeutic Development Branch and director of TRND. "In addition, the FDA was instrumental in helping our team move this project into human clinical trials."

The NPC Phase I clinical trial will test multiple doses of cyclodextrin in nine patients. Forbes Porter, M.D., Ph.D., senior investigator and NICHD clinical director, and Nuria Carrillo, M.D., TRND staff physician, will conduct the trial.

Dr. Porter also is conducting a natural history study of NPC to collect health information from patients to understand how the disease develops. The natural history study is critical to understanding the clinical significance of a treatment for NPC patients.

"Initiation of this clinical trial is the culmination of two decades of basic and clinical research to understand and develop therapies for NPC," said Porter. "The efforts of the collaborators who make up the TRND NPC team have greatly accelerated translating cyclodextrin from the laboratory to the clinic."

The goal of the Phase I clinical trial is to determine a safe dose of cyclodextrin that will support an expanded Phase II trial to begin to evaluate the effectiveness of the drug.

The team already is in the initial stages of collaborating with the Network for Excellence in Neuroscience Clinical Trials (NeuroNEXT), which is administered by the NIH's National Institute of Neurological Disorders and Stroke, to plan a Phase II multicenter trial.

The NPC cyclodextrin project was made possible by a collaborative approach that also included - in addition to NCATS, NICHD, National Institute of Neurological Disorders and Stroke (NINDS) and the FDA - the NIH's National Human Genome Research Institute; Johnson and Johnson Pharmaceutical Research & Development; Washington University in St. Louis School of Medicine; Albert Einstein School of Medicine, New York City, and University of Pennsylvania, Philadelphia.

A number of family support groups have made significant contributions as well that have led to the launch of the clinical trial through the funding of NPC research and patient support. They include the Ara Parshegian Medical Research Foundation, the International Niemann Pick Disease Alliance, the National Niemann Pick Disease Foundation, and Support Of Accelerated Research for Niemann -Pick Type C.

The NPC clinical trial is the fourth TRND project to advance to human clinical trials in the last 15 months. The three other clinical trials are evaluating treatments for sickle cell disease, chronic lymphocytic leukemia and hereditary inclusion body myopathy. TRND has a portfolio of 14 projects, which focus on rare and neglected tropical diseases.


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

Public Support for National Study
Survey shows the majority of respondents support or show willingness for national precision medicine study.
Thursday, August 18, 2016
How Parkinson’s Disease Alters Brain Activity Over Time
The NIH study provides a new tool for testing experimental medications aimed at alleviating symptoms and slowing the rate at which the diseases damage the brain.
Tuesday, August 16, 2016
Schizophrenia, Autism Share Genetic Causes
Monkey brain developmental atlas pinpoints when, where genes activate.
Tuesday, August 16, 2016
Depression Genetics Insight from Crowd-Sourced Data
Genome sites liked to depression have been discovered from data shared by people who had purchased their genetic profiles online.
Tuesday, August 02, 2016
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
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
NIH Funds Biobank To Support Precision Medicine Initiative Cohort Program
$142 million over five years will be awarded to the Mayo Clinic to establish the world’s largest research-cohort biobank for the PMI Cohort Program
Friday, May 27, 2016
Tick Genome Reveals Secrets of a Successful Bloodsucker
NIH-funded study could lead to new tick control methods.
Tuesday, February 09, 2016
Genomic Signature Shared by Five Types of Cancer
National Institutes of Health researchers have identified a striking signature in tumor DNA that occurs in five different types of cancer.
Monday, February 08, 2016
Genome-Wide Study Yields Markers of Lithium Response
An international consortium of scientists has identified a stretch of chromosome that is associated with responsiveness to the mood-stabilizing medication lithium among patients with bipolar disorder.
Monday, February 01, 2016
Schizophrenia’s Strongest Known Genetic Risk Deconstructed
Suspect gene may trigger runaway synaptic pruning during adolescence – NIH-funded study.
Thursday, January 28, 2016
NIH Genome Sequencing Program Targets the Genomic Bases of Common, Rare Disease
The National Institutes of Health will fund a set of genome sequencing and analysis centers whose research will focus on understanding the genomic bases of common and rare human diseases.
Friday, January 15, 2016
Three Glaucoma-Related Genes Discovered
NIH-funded genetics analysis of glaucoma is largest to date.
Tuesday, January 12, 2016
Biomarkers Outperform Symptoms in Parsing Psychosis Subgroups
Multiple biological pathways lead to similar symptoms - NIH-funded study.
Thursday, December 10, 2015
NIH Supports New Studies to Find Alzheimer’s Biomarkers in Down Syndrome
Initiative will track dementia onset, progress in Down syndrome volunteers.
Tuesday, December 01, 2015
Scientific News
Breast Cancer Cells Found To Switch Molecular Characteristics
Spontaneous interconversion between HER2-positive and HER2-negative states could contribute to progression, treatment resistance in breast cancer.
Some Breast Cancer Patients With Low Genetic Risk Could Skip Chemotherapy
Genetic test can help predict survival and guide treatment options.
LncRNAs Maintain Immune Health
Long non-coding RNAs are key controllers for maintaining immune health when fighting infection or preventing inflammatory disorders.
Enzyme that Triggers Cell Demise in ALS Identified
Scientists from Harvard have identified a key instigator of nerve cell damage in people with amyotrophic lateral sclerosis (ALS).
Molecular Alarm Clock Wakes Resting Ovules
Study of fruit flies yields discovery of a molecular "alarm clock" that activates resting ovules.
Improved Immunity in Older Age
A study from Oxford and Basel universities may point the way to maintaining our immune systems as we get older.
Neurodvelopmental Disorder Cause Linked to SON Gene
A genetic link has been discovered for a previously unxplained neurodevelopmental disorder.
Russian TB Thrives Within Macrophages
MIPT researchers have identified features of mycobacterium tuberculosis strains that may explain their success.
Gene Linked to Hearing Loss Identified
Researchers have identifed a gene associated with age-related hearing loss.
World's Most In-Depth Study to Detect Alzheimer's Disease
A multisite team will see the most thorough and vigorous testing for Alzheimer's ever performed on volunteers.
Skyscraper Banner

SELECTBIO Market Reports
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!