NIH clinical trial launched to treat Niemann-Pick disease

A clinical trial reviewing a drug candidate called cyclodextrin as a potential treatment for Niemann-Pick disease type C1 (NPC) has launched.

Scientists from the National Institutes of Health's (NIH) National Centre for Advancing Translational Sciences (NCATS) and the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) will carry out the work.

No therapies have been approved by the US Food and Drug Administration to treat NPC, which is characterised by the inability of cells to metabolise and dispose of cholesterol and lipids.

The condition causes excessive amounts of cholesterol to accrue within the liver, spleen and brain, leading to the progressive impairment of motor and intellectual function in early childhood.

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 traditional use as an ingredient in other drugs to a therapeutic for this rare disorder.

TRND supported animal toxicology studies to evaluate the safety of cyclodextrin and backed the development of an NPC biomarker.

It then submitted the data in an Investigational New Drug application on November 14th, which the Food and Drug Admistration has now determined sufficient to start a Phase 1 clinical trial.

NCATS director Christopher P  Austin,  said: "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.

"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."

The NPC clinical trial is the fourth TRND project to reach the human clinical trials stage in the last 15 months, with the three others evaluating treatments for sickle cell disease, chronic lymphocytic leukemia and hereditary inclusion body myopathy.

Posted by Ben Evans