Dr. Porter is the Clinical Director for the National Institute of Child Health and Human Development (NICHD) at the National Institutes of Health (NIH) in Bethesda, MD.
In order to design a successful clinical trial for potential NPC treatments, Dr. Porter's research group first initiated a natural history study to get baseline data from NPC patients.
A number of children in the Phase I trial have had to get hearing aids because of the cyclodextrin treatment they receive, but to date the hearing loss is not severe or untreatable.
The Phase II trial by Vtesse is critical for proving that cyclodextrin slows NPC progression, so that regulatory agencies will approve cyclodextrin treatment for every person affected by NPC.
Cyclodextrin has a lot of potential as a treatment for NPC, yet it is not going to be a cure. Multiple therapies will be needed to treat different aspects of the disease.
Due to the advancement of basic science research funded by NPC family groups such as the Ara Parseghian Medical Research Foundation, NNPDF and SOAR, there are multiple potential therapies under investigation, including HDAC and gene therapy.
