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Dr. Forbes Porter

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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.

Biography

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.

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Niemann-Pick Type C

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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.

Relating to Parents

While it is very emotional, Dr. Porter finds relating to NPC parents and patients extremely rewarding.

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Cyclodextrin

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Cyclodextrin Clinical Trial

Phase I (NIH)

Dr. Porter is cautiously optimistic that cyclodextrin may be slowing the rate of NPC progression.

Ototoxicity

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.

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Collaboration at NIH

Multiple research groups at the NIH have worked together to advance cyclodextrin as a potential treatment for NPC.

Phase II (Vtesse)

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.

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NPC Drug Development

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Cyclodextrin

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.

Other Potential Therapies

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.