Dr. Forbes Porter
Clinical Director (NIH/NICHD)
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.
Dr. Porter is the Principal Investigator for the important Phase I cyclodextrin trial for NPC at NIH. His combination of research and clinical work allows him an integrated bench-to-bedside and bedside-to-bench approach toward understanding NPC pathology and developing therapeutics for the disease.
Niemann-Pick Type C
Getting Involved with NPC
Because Dr. Porter has expertise in genetic disorders of cholesterol synthesis, he was asked to join a project researching NPC.
Natural History Study
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.
Cyclodextrin Clinical Trial
Phase I (NIH)
Dr. Porter is cautiously optimistic that cyclodextrin may be slowing the rate of NPC progression.
Collaboration at NIH
Multiple research groups at the NIH have worked together to advance cyclodextrin as a potential treatment for NPC.
NPC Drug Development
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.