Here. Us. Now: Documentary Spotlights Rare Disease

Recently, members of Feinstein Kean’s Cambridge staff gathered for a screening of the documentary Here. Us. Now. The film shares the inspirational story of two parents who receive a crash course in drug development. Hugh and Chris Hempel of Reno, NV were forced into the world of rare diseases when their twin daughters, Addison and Cassidy, were diagnosed with Niemann-Pick Type C (NPC1), a terminal disease affecting only an estimated 1 in 150,000 people worldwide.

NPC1 is caused by a genetic mutation involving lipid metabolism and eventually leads to fatal build-up of lipids in the brain. This causes developmental delays and leads to cognitive declines. NPC is diagnosed in early childhood and patients typically lose their battle with the disease before they enter their 20s. With no FDA-approved treatments for NPC, the Hempels faced an uphill battle in finding therapies to extend and improve their daughters’ lives.


After their daughters’ diagnosis left them desperate for options, the Hempels took it upon themselves to research experimental medications that could help their daughters. After sifting through endless reams of scientific studies and running into many dead ends, their diligence paid off when they learned about and secured access to cyclodextrin, a drug found to be effective in treating NPC1 in mice. While it is not a cure, cyclodextrin helped to begin to reverse Addison’s and Cassidy ‘s neurological impairments.

We at Feinstein Kean feel that the film relates to our work in several ways. Here. Us. Now. highlights the urgent need for improvements in the current drug discovery and development system so that we can get potentially life-saving treatments to patients faster. As patients and their caregivers play a larger role in research, they are able to make significant improvements in the battle against rare diseases. FDA regulations, research institutions and pharmaceutical industry business models need to continue to adapt as well in order to make a positive and lasting impact on the rare disease community.

One of the last reasons why this film resonated so well with Feinstein Kean is because of how many familiar names and faces were featured. Our client roster is filled with many organizations that are leading the charge in rare diseases drug development and are happy to see each get the credit they deserve for taking on this difficult, but extremely important, challenge.