The FD Scientific Advisory Board are tasked with evaluating the science of FD and crafting a path to advance treatments. The full board had not met in over 3-years, and the forum to talk about new findings was silent. When it comes to discovering new medicines, progress doesn’t happen in a vacuum. the sequence of events is more than one person observing cell behaviors under a microscope, and rushing to the patient with a new treatment in hand. It takes giant steps to translate that idea to the clinic. Often, this involves extracting funding from federal grants and staffing laboratories with bright scientists. Then, ideas start to unravel and new treatments emerge. In order to go from the bench to the bedside, basic science and clinical research have to come together.
At the Center, one hears stories every day from people facing FD. Our patients know what they want to fix, but their specific problem needs to be in the hands of the basic scientists. Only they can take potential promising strategies forward by experiments in cells or genetically engineered mice. When basic science and clinical medicine don’t interact, the science can move in opposite directions, rather than in unison. We know what features of FD progress over time and which specific cells we need to save and support. If you want to save vision, you need a way to target the cells of the retina, you don’t need a drug that is going to stay concentrated in the GI tract and not reach the eye. The key to making this happen is communication.
Recognizing the need to talk, the Foundation brought in scientists from key laboratories in the U.S. together with FD parents to NYU. The labs were each given 30 minutes to present their work. As different approaches where debated and ideas bounced back and forth, action items started emerging. The voices of the scientists brought new concepts to the FD program and scientific rigor. At any given time, 2 people with FD are usually hospitalized with serious medical problems. It seemed fitting that the meeting was held at NYU’s Medical Center, where the patients with FD are often admitted. It brought home the urgent need to move the science along to change the lives of patients with FD.
To align the research, a close network of advisory board members is a key strategy. Following the meeting, the Foundation began re-shuffling its scientific advisory board, bringing in top basic scientist Dr. Frances Lefcort and drug development expert Dr. Adrian Gilbert, who both have personal connections to FD. Since then, the scientific progress has shifted up a notch. With the renewed energy, the scientists are talking again on a regular basis with video calls and face-to-face meetings. The scientific advisory board has again become a resource for scientists looking for input into their work on FD and collaboration.