Faculty Scientist
Biomedical Research Institute of New Jersey
What first drew you to Krabbe disease—and why have you stayed?
It was the first project assigned to me when I started my first postdoc. At the time, my PI was drawn into it because of the simplistic nature of the disease mechanism (monogenetic disorder) and yet the aggressive nature of the disease. We both treated it as a challenge for our careers and set our goal to make a difference. Since then, I have been working on projects related to other neurodegenerative disorders. Somehow, the common theme of the disease mechanism always brought me back to lysosome dysfunction. When I started my own lab, my deep-rooted interest brought me back to studying for Krabbe Disease.
What is one thing you wish every family understood about the work happening right now?
I want families to understand that we are working diligently to experiment with various potential therapeutics that can help to alleviate the disease. In addition to the KD scientific community, breakthroughs coming from other similar diseases can bring insights and opportunities to better understand disease mechanisms and/or drugs that may also benefit KD.
What gives you the most hope when you think about the future of Krabbe disease?
Recent approval of Avalayah for Hunter Syndrome, which is a modified form of therapeutic protein that can be delivered directly to the brain without surgery, has opened up a promising therapeutic opportunity to treat patients with all forms of Krabbe Disease.
What keeps you going on the hard days when progress feels slow?
When a day is filled with negative data and uncontrollable events, I always remind myself that consistency and patience bring results. The key thing is to learn from whatever negative results tell us in terms of hypothesis, experiment design, and approach so that we can build a more solid foundation to move forward.
