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 2,400+ scientific posters on ePosters
  • More than 3,700+ 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

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
Possible Treatment Target for Type 2 Diabetes Identified
Researchers at the NIH have clarified in rodent and test tube experiments the role that inflammation plays in type 2 diabetes, revealing a possible molecular target for treating the disease.
Tuesday, August 20, 2013
NIH Funds Research to Explore a Cell Communication Process
Researchers will investigate the emerging field of extracellular RNA and its role in human health conditions.
Wednesday, August 14, 2013
Therapy May Curb Kidney Deterioration in Patients with Rare Disorder
Innovation in mouse model helps researchers distinguish disease mechanisms and biomarkers.
Tuesday, July 30, 2013
Scientific News
RNAi Screening Trends
Understand current trends and learn which application areas are expected to gain in popularity over the next few years.
New Tool Uses 'Drug Spillover' to Match Cancer Patients with Treatments
Researchers have developed a new tool that improves the ability to match drugs to disease: the Kinase Addiction Ranker (KAR) predicts what genetics are truly driving the cancer in any population of cells and chooses the best "kinase inhibitor" to silence these dangerous genetic causes of disease.
HIV Susceptibility Linked to Little-Understood Immune Cell Class
High levels of diversity among immune cells called natural killer cells may strongly predispose people to infection by HIV, and may be driven by prior viral exposures, according to a new study.
Sweet Revenge Against Superbugs
A special type of synthetic sugar could be the latest weapon in the fight against superbugs.
Access Denied: Leukemia Thwarted by Cutting Off Link to Environmental Support
A new study reveals a protein’s critical – and previously unknown -- role in the development and progression of acute myeloid leukemia (AML), a fast-growing and extremely difficult-to-treat blood cancer.
Long-sought Discovery Fills in Missing Details of Cell 'Switchboard'
A biomedical breakthrough reveals never-before-seen details of the human body’s cellular switchboard that regulates sensory and hormonal responses.
Tracking Breast Cancer Before it Grows
A team of scientists led by University of Saskatchewan researcher Saroj Kumar is using cutting-edge Canadian Light Source techniques to screen and treat breast cancer at its earliest changes.
Zebrafish Reveal Drugs that may Improve Bone Marrow Transplant
Compounds boost stem cell engraftment; could allow more matches for patients with cancer and blood diseases.
DNA Damage Seen in Patients Undergoing CT Scanning
Along with the burgeoning use of advanced medical imaging tests over the past decade have come rising public health concerns about possible links between low-dose radiation and cancer.
The Light of Fireflies for Medical Diagnostics
EPFL scientists have exploited the light of fireflies in a new method that detects biological molecules without the need for complex devices and high costs.
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
2,400+ scientific and medical posters
A library of 2,500+ scientific videos on LabTube
3,700+ scientific videos
Close
Premium CrownJOIN TECHNOLOGY NETWORKS PREMIUM FREE!