As my second month in my new role as FPWR's CEO concludes, I would like to reflect on some of my early observations and experiences with this truly incredible Prader-Willi community.
During the short time I've been in this role, I've met or spoken with many volunteers and caregivers and now have begun to build an understanding of the enormous challenges you face on a daily basis and the incredible investment you're making in FPWR’s research to improve the lives of individuals with PWS.Personally, I'm a father of five and have experienced that magical moment of birth, listened for that cry, counted fingers and toes and asked, “Is our baby okay?” I realize how blessed I am. But through my work experience in oncology and working with parents of children with cancer, I've developed a deep understanding of the journey of having a child with significant medical needs. I understand that the whole family is impacted by a PWS diagnosis and the challenges of the life ahead for you and your family.
As you know, with the exception of growth hormone protocol, no FDA–approved treatment exists for PWS today. But together we can change that. With your engagement and partnership, we can fund cutting-edge research to alter the current paradigm for the life of your loved one with PWS. Together we can invest in research that can unlock the genetic mysteries that today seem daunting. Progress is being made!
Having witnessed firsthand some the remarkable genetic breakthroughs in the successful treatment of cancer and for such maladies as inherited blindness and Battens disease, I'm confident that in the next five to ten years, with sufficient funding, we will begin to see breakthroughs so that PWS might be addressed genetically. The timing of success comes down to three things: the right research, sufficient funds to support it and the taking of some risks or leaps of faith!
Recently, FPWR–funded research developments have been highlighted in two prominent publications. I had the pleasure of visiting one of these teams, led by Stormy Chamberlin and Marc Lalande, at their lab at the University of Connecticut, to learn firsthand of their progress. It’s still early, but they have coaxed stem cells to grow into neurons and by manipulating the gene that controls PWS gene silencing, demonstrated that expression of the PWS genes was rescued, such that the cells had a normal gene expression pattern. While this work is still early and being done on mouse models, it demonstrates what’s possible with sufficient funding.
I look forward to serving as your CEO and will communicate with you from time to time. In the interim, please contact me directly with any questions or feedback!