At the BIO International Convention recently held in Philadelphia, PA, we talked with Christina Hartman, Senior Director of Advocacy & Policy for the EveryLife Foundation for Rare Diseases about the origins of this amazing organization.
Hartman said, “The foundation was started in Novato, California by Emil Kakkis, who is now
the CEO of Ultragenyx, a rare disease pharmaceutical company. He founded EveryLife
based on his experience with Mark Dant, the organization’s Board Chair. Mark is the father of Ryan Dant who has MPS I (mucopolysaccharidosis 1).”
“in the early 1990s, Mark was a police officer in Texas. He and his wife first we’re told that Ryan had this terrible disease, MPS 1, and that he would die soon so they should just take him home and love him for as long as they had left . For Mark, this was unacceptable. He hit the streets and started raising money.”
“To make a long story short, Mark raised a lot of money was able to find Emil Kakkis who was a pediatrician doing research in California.”
In 2003, the FDA approved a Aldurazyme (laronidase) for the treatment of MPS 1. Hartman noted, “Ryan has now been on this therapy for 20 years is 30 years old and is now engaged. So, it’s an incredible success story. Unfortunately, it’s very unusual and so what Emil and Mark really want to do is translate that experience to the rest of the rare disease community.”
To that end, EveryLife Foundation is focused largely on determining what are the policies in place (or need to be in place) to make sure other rare disease patients can succeed as well as Ryan.
“Over the past 10 years they have had some major successes. They were instrumental in passing the 21st century cures Act. There’s a lot of talk about what’s going to come next after that and that’s something that the foundation will be very engaged in. We’re also very engaged in figuring out how to work with the FDA to streamline the regulatory process. How do we make changes for these small heterogeneous patient populations? We’ve been using randomized controlled trials as the gold standard for decades for 50 years and with what we know about human biology now, that’s not necessarily the best way especially when you have these very small patient populations with lethal conditions,” stated Hartman.
“ We have a meeting coming up in September that we’ll be hosting in Washington DC and we’re going to be convening the leadership of the FDA, the pharmaceutical industry, and the patients together, and taking a look at what is the science of small clinical trials in this age of biological plausibility. And how can we start to use biomarkers as endpoints? What information does the FDA need to be able to vet these to ensure that the drugs that we’re looking at are still safe and effective but there are no bottlenecks in the process in terms of getting them to patients and allowing patients to access these clinical trials,” concluded Hartman.
For more information, visit everylifefoundation.org/