Jeff Watkins - A Consumer's Perspective
permission of Jeff Watkins.
I recently retired from a 40-year career in the energy industry and I am currently traveling with my dog Coach in a 23’ travel trailer throughout the western half of the US and Canada. My late wife, Lilly, passed away 10 years ago after a brief but valiant fight with a brain tumor and my son Noah passed away last July after a 32 year battle with Duchenne Muscular Dystrophy (DMD). My youngest son, Woodrow, lives and works in Annapolis, MD where our family has resided since Noah was diagnosed with DMD in 1988 at the age of five.
The diagnosis was devastating to the entire family, plain and simple. Both boys were born in Boulder, Colorado and when they were still infants we would take them camping in the foothills of the Rockies. We moved to Annapolis when Noah was four and began taking the boys on overnight sailing adventures on the Chesapeake Bay in our 23’ sloop. My wife and I loved the outdoors but realized after the diagnosis that our lives would be forever different is so many ways.
Living in Annapolis afforded Noah the best possible care as several world class research hospitals were within an hour’s drive of our home, including, but not limited to: Johns Hopkins Hospital, Kennedy Krieger Institute and Children’s National Medical Center.
A significant portion of my story is about Noah and his relationship to his younger brother Woody. Noah had the strength of steel in his determination to fight his disease that was the equal to his brother’s muscular strength. Noah never gave in to his condition, determined to walk as long as possible and play baseball with his brother and the neighborhood kids until he was no longer able to stand up alone without assistance. Woody was his rock, always there to carry him on his shoulders to his next destination, whether it be around the block or navigating the numerous physical and mental challenges Noah faced each day of his life.
permission of Jeff Watkins.
As parents, Lilly and I were determined to raise Noah as a normal child, despite the fact that every muscle in his body became weaker on a daily basis. We realized there are no rules for dealing with this disease, only parental intuition and guidance from organizations such as the Muscular Dystrophy Association (MDA) and the Parent Project for Muscular Dystrophy on how to proceed. Noah was able to graduate from Broadneck High School and Salisbury University with the incredible support of his extended family (aunts, uncles, cousins and close friends) and the above organizations. His best buddies and all of his cousins never viewed Noah as weak, rather they saw only a stubborn, determined guy who would make them laugh with his dry wit and understated humor (as well as his passion for the Ravens and Orioles).
Lilly and I received much needed support from MDA during the first few years following Noah’s diagnosis. In 1992 we became involved with the Parent Project for Muscular Dystrophy (PPMD) during its inception as an advocacy organization with a focus on supporting research that will lead to a cure for DMD. When Lilly lost her battle with a brain tumor in November 2006, her birthday in June became the focal point for raising funds for both MDA and PPMD.
The annual “Lilly Fest” has been instrumental in raising awareness about DMD and contributing considerable funds to MDA and PPMD that will eventually lead to effective treatments and a cure. I have participated in outreach meetings with my congressman and senators with the goal of advancing legislation that supports funding for DMD research as well as streamlining FDA’s approach to approving therapies that benefit the DMD community and the many other rare diseases that currently lack viable treatments.
A good friend who served as a peer reviewer for metastatic breast cancer research introduced me to the Duchenne Muscular Dystrophy Research Program (DMDRP) in 2014. I have completed two stints as a consumer reviewer for DMD research grants seeking funding from DMDRP. My job was to provide guidance to the reviewing panel from the perspective of the patient community. Because Noah had experienced all phases of the disease when I was participating in the review process I was able to communicate to the panel the importance of considering the entire DMD community regardless of age and/or the location of their gene mutation when awarding funding.
I found the scientific reviewers to be extremely competent in their understanding of the nature of DMD and very willing to explain the technical aspects of the proposed research in terms that I could understand as a lay reviewer. There were several occasions during the discussions of the proposals when a scientific reviewer would acknowledge the value of the consumer reviewer input. In several instances this understanding of the value of the research to the patient community triggered a change in the scientific reviewers scoring of the proposals. This demonstrated to me the value of my input to the review process.
By participating in the review process I was able to gain a better understanding of just how difficult it is to design and execute a research project with the potential to lead to a positive outcome for the patient community. I was exposed to numerous independent strategies, some extremely novel, with the potential to lead to viable therapies for treating DMD. Because there are limited funds available for DMD research I felt that many worthy proposals would lack the funding they deserve. Therefore the continued efforts by PPMD and MDA to advocate for increased funding for DMDRP research continues to be of utmost importance to the Duchenne patient community.
Last updated Wednesday, September 7, 2016