Experimental treatment for Niemann-Pick disease type C1 appears safe, effective
![](/46/pdcnewsitem/04/77/73/nih.png)
NIH-led clinical trial suggests that drug slows progression of rare neurological disease.
An experimental drug appears to slow the progression of Niemann-Pick disease type C1 (NPC1), a fatal neurological disease, according to results of a clinical study led by researchers at the National Institutes of Health.
NPC1 is a rare genetic disorder that primarily affects children and adolescents, causing a progressive decline in neurological and cognitive functions. The FDA has not approved any treatments for the condition.
The drug, 2-hydroxypropyl-beta-cyclodextrin (VTS-270), is being tested under a cooperative R&D agreement, or CRADA, between NIH and Sucampo Pharmaceuticals. In April 2017, Sucampo acquired Vtesse, which previously had been developing VTS-270.
“The results are very encouraging and support continued development of VTS-270 for treating NPC1,” said Forbes D. Porter, clinical director at NIH’s Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) and the study’s senior author. “Compared to untreated patients we followed in an earlier study, participants who received VTS-270 scored better on a scale used to evaluate disease severity and progression, including elements such as speech, cognition and mobility.”
The study was a Phase I/IIa clinical trial designed to test the drug’s safety and effectiveness. A group of 14 participants, ranging from ages 4 to 23 years, received the experimental drug once a month at NIH for 12-18 months. Another group of three participants received the drug every 2 weeks for 18 months at Rush University Medical Center in Chicago. Initially, participants were divided into groups receiving different doses of the drug, but after observing that the drug was safe and well-tolerated by those receiving the highest doses, the researchers increased dosing for all participants. Their progress was compared to a previous group of 21 NPC1 participants enrolled in an earlier study that documented disease progression.
The researchers did not observe any serious adverse outcomes related to the drug. However, the participants, most of whom had already experienced hearing loss because of the disease, had additional hearing loss after treatment. Earlier studies had shown that the treatment carries the risk for hearing loss. In the current study, hearing loss was compensated with hearing aids, which enabled participants to go about their daily lives.
Because NPC1 symptoms result from cholesterol buildup in brain cells, the researchers also measured cholesterol metabolism in the participants’ central nervous system. The researchers found that after treatment, a molecule derived from cholesterol metabolism in neurons, 24(S)-hydroxycholesterol, had increased. In addition, most participants had lower levels of two proteins indicative of brain injury, FABP3 and calbindin D, implying that there was less damage in the brain. According to the study authors, these results suggest that VTS-270 can improve cholesterol metabolism in neurons, thereby targeting the root of the problem.
The researchers also evaluated the drug’s effectiveness using a neurological severity score, where higher scores indicate more severe effects from the disease. Compared to an earlier group of patients who had not received the drug, VTS-270-treated participants had lower scores in measures of cognition and speech, with mobility scores also trending lower. The authors believe these differences indicate that treatment with the drug can stabilize or slow disease progression.
NICHD researchers led the design, data collection and analysis of the Phase I/IIa clinical trial. VTS-270 was provided by Janssen Research & Development, a Johnson & Johnson company. Researchers are now working on a randomized, controlled phase 2b/3 clinical trial (NCT02534844) that has been approved by the FDA and the European Medicines Agency. The trial is sponsored by Sucampo, and its results will help determine which symptoms are most responsive to the drug and provide information for refining the dose and dosing frequency. The goal of this trial is to obtain regulatory approval of VTS-270.
Related News
-
News CPHI Podcast Series: the power of digital marketing in pharma
Digital marketing is a valuable tool for many industries, and the pharmaceutical and healthcare industry is no exception. The CPHI Podcast Series covers how marketing can be used by companies to increase their engagement and overcome challenges.&n... -
News Novel approach to creating sustainable packaging from rice husks
Researchers have created a new approach to the designing of eco-friendly nanofibres extracted from rice husks, addressing the critical need for sustainable packaging materials in food and biopharmaceutical products. -
News BioNTech to begin mRNA vaccine manufacturing in Rwanda by 2025
German biotechnology company BioNTech has stated their intentions to begin production at their mRNA vaccine factory in Rwanda by 2025, which will mark the first foreign mRNA vaccine manufacturing site on the continent of Africa. -
News Identifying Alzheimer’s Disease biomarker proteins with whole blood tests
A University of Manchester spin-out pharmaceutical company, PharmaKure, has reported successful study results for the quantification of Alzheimer’s Disease biomarker proteins with a whole blood test. -
News Bill & Melinda Gates Foundation to boost mRNA vaccine initiatives in Africa with USD $40m
To address vaccine inequality and accessibility issues, the Bill & Melinda Gates Foundation aims to deliver USD $40m to various biotech companies and vaccine manufacturers in support of mRNA vaccine development. -
News CPHI Podcast Series: Exploring neurological frontiers in Alzheimer's and beyond
The next episode of the CPHI Podcast Series delves into the science and background behind some recent developments in the field of Alzheimer's disease and neurological disorders. -
News Is patient centricity the future of pharmaceutical manufacturing?
In this interview with Sandra Sánchez y Oldenhage, President of PharmAdvice, she speaks to the importance of considering patients in the manufacturing stages of the pharmaceutical supply chain, and how it can redefine healthcare. -
News CPHI Podcast Series: How to leverage AI for Drug Discovery
Artificial intelligence is the topic of debate in the latest episode from the CPHI Podcast Series, where Digital Editor Lucy Chard speaks with Bill Whitford of DPS Group about the integration of AI in healthcare.
Position your company at the heart of the global Pharma industry with a CPHI Online membership
-
Your products and solutions visible to thousands of visitors within the largest Pharma marketplace
-
Generate high-quality, engaged leads for your business, all year round
-
Promote your business as the industry’s thought-leader by hosting your reports, brochures and videos within your profile
-
Your company’s profile boosted at all participating CPHI events
-
An easy-to-use platform with a detailed dashboard showing your leads and performance