The Hempels are one of the many rare disease families who are racing against time to save their children’s lives. Their twin daughters, Addison and Cassidy, have Niemann-Pick type C, a rare and fatal genetic disease that causes progressive neurological deterioration. A documentary about the family, Here. Us. Now., has been commissioned by the Ewing Marion Kauffman Foundation.
There is no treatment for Niemann-Pick type C, so the Hempels did their own research and discovered a simple compound, cyclodextrin, being used in mice. They petitioned the FDA to get permission to use it on their daughters. While not a cure, the Hempels feel that it has slowed the progression of the disease. In 2011, the couple started their own biotech company, Solution Therapeutics, and is seeking funding to start clinical trials of cyclodextrin. The Hempels’ story reminds me of those of several other empowered patients and families who have taken action and are making a difference.
On Friday, September 28, 2012, the Global Genes Project will hold a free screening of the documentary at 7 p.m. at the Harmony Gold Preview House in Los Angeles, California. A limited number of seats will be made available for this private screening.