The Cure Rare Disease Foundation
The Cure Rare Disease Foundation‘s mission is to increase collaborative efforts between researchers in order to accelerate the development of customized therapies for individuals living with rare diseases. The goal is to create these therapies “in time to save our loved ones.”
The range of diseases they work with include Duchenne muscular dystrophy (DMD), limb-girdle muscular dystrophy, Becker’s muscular dystrophy, sickle cell disease, cystic fibrosis, spinal muscular atrophy, Canavan disease, and Charcot-Marie-Tooth disease among other rare, genetic diseases.
How it Originated
This organization was founded by Rich Horgan, a summa cum laude graduate of Cornell who also has a MBA from Harvard. Horgan’s younger brother was diagnosed with DMD which quickly turned this businessman into an advocate for rare disease. He saw there was a desperate need to accelerate the search for treatments not only for DMD but for all rare conditions and took action. Not only do we need more effective treatments faster, we need these treatments to be personalized to the individual.
For rare diseases especially, the “one size fits all” approach just isn’t good enough.
The organization’s board of directors features both parents of children living with a rare disease, healthcare professionals, and those skilled in business.
How it Works
This organization works by collaboration. By bringing together the wisdom of a multitude of individuals who are luminaries in their field, rapid development of therapies is a much more achievable dream. Cure Rare Disease aligns these leaders with a clear research goal, provides them support, and oversees the projects to ensure accountability. In this way, they are able to take advantage of group wisdom while avoiding some of the common hurdles to clinical development.
Their process has 4 steps. First, they establish a cell line for a patient so that therapies can be tested directly against the individual’s genetic profile. This cell line becomes a foundation where all of the collaborators can take “therapeutic shots on the goal.” Next, customized therapies are developed. These therapies are then tested against the cell line of the patient. The most effective of the therapies then moves to development. The Foundation’s goal is that from the start of the investigation, it will take only 1 to 2 years to enter a FDA approved clinical trial.
At the same time that the therapy is moving into development, the organization begins to generalize the process. They create cohorts of individuals who have the same genetic mutation and could also benefit from the same treatment approach.
Cure Rare Disease supports patients and families throughout the journey. Simultaneously, they strive to educate the global community about rare disease and the need for personalized therapeutics.
Of course, critical to this organization’s success is that rare disease families know of its existence, as do the researchers and clinicians who could make invaluable contributions to their projects. If you are interested in the initiatives of this foundation, you are strongly encouraged to reach out. If you would like to support the work of this organization financially, you can do so here. They also have an upcoming Community Conference in July which is free for rare disease families.