October 3, 2019
(Picture courtesy of Jamie Martin, NCBiotech)
Today, there are around 7,000 known rare diseases that impact more than 25 million people in the United States. The 2019 Invest in Cures and Rare Diseases forum convened Sept. 25-26 to discuss how to find treatments for them.
The forum was a collaboration between LaunchBio, the UNC Catalyst for Rare Diseases, NCBiotech and the Rare Disease InfoHub. The UNC Catalyst is a research group housed in the UNC Eshelman School of Pharmacy that focuses on improving research infrastructure and understanding genetic mutations and rare diseases. The event was hosted at the North Carolina Biotechnology Center in Research Triangle Park, N.C.
Anthony Hickey, Ph.D., director of the UNC Catalyst, said the forum was meant to be a way for industry, venture philanthropists and foundations to collaborate and move rare disease research forward.
Hickey also said the event was an important way to find new sources for funding in the field, and to build connections between clinicians and researchers. Both days set aside time for attendees to network.
“It’s bringing together people from around the region [who] have an interest in rare disease, both on the podium and in the audience,” he said. “You have representatives in almost all aspects of rare diseases that should be able to reflect on all the issues that we’re faced with.”
Hickey said around 70 people registered for the conference. The first day focused on the foundations of rare disease research, including cancer and translational research. Panelists from various rare disease foundations discussed the pace of commercialization and how to invest in new patient treatments.
“I don’t think we’re going to come away from this meeting having solved the world’s problems,” Hickey said. “This is just an initial dialogue.”
The second day featured a rare disease forum, with a panel on the barriers to developing affordable treatments. The panelists included foundation directors and officials from private industry and the U.S. government.
“Rare disease is a public health imperative,” said Sharon King, to the audience. King, president of the nonprofit Taylor’s Tale, started the organization after her daughter was diagnosed with Batten disease, a disorder that affects the nervous system.
Christal Delagrammatikas, co-founder and vice president of the Malan Syndrome Foundation, said she was interested in the forum as someone new to the nonprofit world and as the mother of a child with a rare disease. She said she was excited by the scientific collaborations in the field since being impacted by a rare disease can be isolating. Christal’s husband, George Delagrammatikas, agreed.
“I was inspired by and encouraged by the link between different fields,” he said. “If you sit down and talk, you can actually make a real difference.”