Genetic Alliance recently held an event in Chapel Hill, N.C. that brought together patient advocates, scientists and representatives from the pharmaceutical industry in a unique “speed-dating” format. Disease advocacy organizations were invited to present briefly on their opportunities for scientific collaboration, and the roadblocks to accomplishing their goals. The focus was on rare diseases and how best to approach development of therapies for them. This event was an interesting microcosm of the rare disease community with a variety of perspectives represented. Ample opportunity was provided to speak with advocates representing a broad spectrum of diseases such as Huntington’s Disease, Angioma, Adenoid Cystic Carcinoma, Autism, and others. The diseases may be disparate, but the challenges faced by rare disease advocacy organizations are similar.
Adding to this was the scientific perspective provided by the Structural Genomics Consortium, a forward-thinking research institute based out of Oxford University in the UK, Frankfurt, Germany, and UNC- Chapel Hill, N.C. Scientists at SGC-UNC are building a functional map of the understudied protein kinases in human biology and diseases through open sharing of compounds with the scientific community. They are developing a hub for kinase drug discovery.
Representatives from a number of pharmaceutical companies and patient advocacy organizations such as the LRG explored opportunities for collaborations which may lead to future conversations as the LRG explores avenues to aid in our research.
A number of presentations by other rare disease advocacy organizations were given at the event, with the opportunity for a presentation on how the LRG and its research team are tackling the problem of Gleevec resistance, and the roadblocks we need to overcome to solve this.
While only a one-day event with a specific focus, the concept of creating opportunities for patient advocacy organizations, the scientific community and pharma holds promise for future innovation through collaborative efforts.