Rare Disease Report

Experimental Neimann-Pick Treatment Appears to Slow Progression

AUGUST 11, 2017
Mathew Shanley
In June 2016, Rare Disease Report provided an update on Vtesse’s drug VTS-270, intended for treatment of Niemann-Pick Type C1 disease (NPC1).
Now, after the April 2017 acquisition of the company developing the drug, Sucampo Pharmaceuticals, Inc. has the therapy being tested under a cooperative research and development agreement (CRADA) in conjunction with the National Institute of Health (NIH). Results from a clinical study were published in The Lancet yesterday.
The results of the phase 1/2a trial show that VTS-270, a modified form of cyclodextrin, appears to slow the progression of NPC1, a progressive and ultimately fatal condition that is the result of mutations in the NPC1 gene.
The NPC1 gene codes for a protein that is used to transport cholesterol and other lipids between cells. In patients, neurological symptoms like seizures, delayed motor development, and cognitive problems typically develop from a cholesterol buildup in the brain and begin during early childhood.
A group of 14 patients at NIH, ranging from ages 4 to 23 years, participated in the study. Each received the drug once a month for 12 to 18 months. Another group of participants at Rush University Medical Center in Chicago received the drug every 2 weeks for 18 months. After it was observed that the drug was safe and well-tolerated, dosage was increased in all participants. The common belief among researchers of the study that differences in dosing can stabilize or slow disease progression.
“The results are very encouraging and support continued development of VTS-270 for treating NPC1,” said the study’s senior author, Forbes D. Porter, M.D., Ph.D., clinical director at NIH’s Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) in a press release on the NIH website. “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.”
No serious adverse outcomes related to the drug were observed in the study, however, it was noticed that any participants who had already begun experienced hearing loss had additional hearing loss after treatment. It was expected after conducting earlier studies that this could be the case.
Additionally, most participants had lower levels of the 2 proteins suggestive of brain injury; FABP3 and calbindin D. This implies that there was overall less damage in the brain.
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