Corporate Banner
Satellite Banner
Biomolecular Screening
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
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 2,500+ scientific posters on ePosters
  • More than 3,800+ 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

Tell-tale Biomarker Detects Early Breast Cancer in NIH-funded Study
The study published online in the issue of Nature Communications.
Thursday, August 13, 2015
Protein Related to Long Term Traumatic Brain Injury Complications Discovered
NIH-study shows protein found at higher levels in military members who have suffered multiple TBIs.
Tuesday, August 04, 2015
Crystal Clear Images Uncover Secrets of Hormone Receptors
NIH researchers gain better understanding of how neuropeptide hormones trigger chemical reactions in cells.
Monday, August 03, 2015
Vital Protein in Healthy Fertilization Process Identified
Researchers at the National Institutes of Health have discovered a protein that plays a vital role in healthy egg-sperm union in mice.
Monday, July 27, 2015
NIH Joins Public-Private Partnership to Fund Research on Autism Biomarkers
Biomarkers Consortium project to improve tools for measuring and treating social impairment in children with autism.
Tuesday, July 21, 2015
Potential Therapeutic for Blinding Eye Disease
NIH research points to microglia as potential therapeutic target in retinitis pigmentosa.
Thursday, July 02, 2015
Linking Targeted Cancer Drugs to Gene Abnormalities
Investigators at the NIH have announced a series of clinical trials that will study drugs or drug combinations that target specific genetic mutations.
Wednesday, June 03, 2015
Possible Treatment for Lethal Pediatric Brain Cancer
NIH-funded preclinical study suggests epigenetic drugs may be used to treat leading cause of pediatric brain cancer death.
Tuesday, May 05, 2015
HIV can Spread Early, Evolve in Patients' Brains
Findings add urgency to screening, treatment - NIH-funded study.
Saturday, March 28, 2015
Test Reliably Detects Inherited Immune Deficiency in Newborns
NIH-supported study suggests that early diagnosis of severe combined immunodeficiency leads to high survival rates.
Thursday, August 21, 2014
NIH Names New Clinical Sites in Undiagnosed Diseases Network
Four-year, $43 million initiative engages broad expertise in study of mystery conditions.
Wednesday, July 02, 2014
Underlying Genetics and Marker For Stroke Discovered
NIH-funded findings point to new potential strategies for disease prevention, treatment.
Friday, March 21, 2014
Epigenetic Clock Marks Age of Human Tissues and Cells
The age of many human tissues and cells is reflected in chemical changes to DNA. The finding provides insights for cancer, aging, and stem cell research.
Tuesday, November 05, 2013
Brain May Flush Out Toxins During Sleep
NIH-funded study suggests sleep clears brain of damaging molecules associated with neurodegeneration.
Friday, October 18, 2013
NIH Researchers Identify Candidate Drug to Treat Batten Disease
The drug, tested in mice, was found to slow the loss of coordination seen in the disorder extending the animals’ life span.
Tuesday, October 01, 2013
Scientific News
Promising Class of New Cancer Drugs Cause Memory Loss in Mice
New findings from The Rockefeller University suggest that the original version of BET inhibitors causes molecular changes in mouse neurons, and can lead to memory loss in mice that receive it.
Electrical Control of Cancer Cells
Research led by scientists at The University of Texas Health Science Center at Houston (UTHealth) has revealed a new electrical mechanism that can control these switches.
Signature of Microbiomes Linked to Schizophrenia
Studying microbiomes in throat may help identify causes and treatments of brain disorder.
Inflammation Linked to Colon Cancer Metastasis
A new Arizona State University research study led by Biodesign Institute executive director Raymond DuBois has identified for the first time the details of how inflammation triggers colon cancer cells to spread to other organs, or metastasize.
Structural Discoveries Could Aid in Better Drug Design
Scientists have uncovered the structural details of how some proteins interact to turn two different signals into a single integrated output.
Determining the Age of Fingerprints
Watch the imprint of a tire track in soft mud, and it will slowly blur, the ridges of the pattern gradually flowing into the valleys. Researchers have tested the theory that a similar effect could be used to give forensic scientists a way to date fingerprints.
Genetic Overlapping in Multiple Autoimmune Diseases May Suggest Common Therapies
CHOP genomics expert leads analysis of genetic architecture, with eye on repurposing existing drugs.
Surprising Mechanism Behind Antibiotic-Resistant Bacteria Uncovered
Now, scientists at TSRI have discovered that the important human pathogen Staphylococcus aureus, develops resistance to this drug by “switching on” a previously uncharacterized set of genes.
Tissue Bank Pays Dividends for Brain Cancer Research
Checking what’s in the bank – the Brisbane Breast Bank, that is – has paid dividends for UQ cancer researchers.
Researchers Publish Landmark “Basket Study”
Researchers from Memorial Sloan Kettering Cancer Center (MSK) have announced results from the first published basket study, a new form of clinical trial design that explores responses to drugs based on the specific mutations in patients’ tumors rather than where their cancer originated.
SELECTBIO

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
2,500+ scientific and medical posters
A library of 2,500+ scientific videos on LabTube
3,800+ scientific videos
Close
Premium CrownJOIN TECHNOLOGY NETWORKS PREMIUM FREE!