Professors of chemistry and biochemistry, Paul Helquist and Olaf Wiest, together with Frederick Maxfield of Cornell University have received a U.S. patent for potential treatments for Niemann-Pick Type C (NPC) disease. Patent No. 9,333,222, “Histone deacetylase inhibitors as therapeutic agents for Niemann-Pick Type C Disease,” covers the full class of histone deacetylase inhibitors for the genetic, fatal lysosomal storage disorder and related diseases.
One of those inhibitors, identified by Helquist and his collaborators, is in a two-year clinical trial that started in September 2014 to assess its effect on NPC. Of the hundreds of known inhibitors, three have been approved by the FDA in the United States for clinical use.
“The language in the patent is such that what works with this particular disease should be translatable to other diseases that are related,” says Helquist, who has several other patents including another involving NPC. “In our ongoing work here at Notre Dame, we are designing and synthesizing new ones to have better drug properties.”
Notre Dame has focused on fighting rare diseases for years, founding what is now the Boler-Parseghian Center for Rare and Neglected Diseases in 2008. Because the diseases offer very limited markets, they are not typically attractive targets for research by large pharmaceutical companies.