Professor Richard Silverman (Chemistry and Molecular Biology) is not content to rest on his laurels following his extraordinary discovery of pregabalin (Lyrica®). Instead, he has used the translational resources of the Chemistry of Life Processes Institute to develop next generation treatments for a range of orphan diseases. An orphan disease is a condition that affects fewer than 200,000 people nationwide. Collectively, they affect as many as 25 million Americans, according to the National Institutes of Health (NIH).

Silverman has developed a new type of enzyme therapy (CPP-115) forlock infantile seizures. This new drug is 5 times more stable than the current drug of choice, allowing longer intervals between IV infusions. In collaboration with Dmitri Krainc (Neurology), the technology used to develop CPP-115 is being applied to Gaucher’s and other diseases based on lysosomal storage disorders.

CPP-115 has been licensed by Catalyst Pharmaceuticals, which hopes to develop CPP-115 for other central nervous system indications, such as epilepsy, Tourette syndrome and Post Traumatic Stress Disorder. CPP-115 has already received orphan drug designation in both the US and the EU for infantile spasms.