Two colorful packets arrived in the mail yesterday, one for Charley and one for his older brother Sam. Sammy's envelope contained a slick 2-pocket folder stuffed with colorful inserts -- promotional meterials from a summer sleepaway camp we're considering for him. Sam ripped through the pages, shouting "They have windsurfing! They have a zip line! I am definitely going there!!"
Charley's envelope was also a glossy folder with colorful inserts, but his came from Mass General Hospital; it was an information packet for new patients. The hospital sent it to us in anticipation of our upcoming appointment with a pediatric endocrinologist. Chronic steroid treatment can stunt growth, and Charley hasn't grown in over a year so are considering treating him with a daily injection of human growth hormone. His growth has stalled so much that he is by far the smallest kid in the class and he shares a shoe size with his four-year-old sister Maisy. Much more alarming as far as Charley is concerned -- he can't go on any of the rides he likes best at amusement parks because he never meets the height requirement.
As the boys grow older, the reality of Charley's existence is hitting me in the gut more often and with considerably more force. After Charley got into bed last night I sat down with Sam to view the camp's promotional DVD. Within minutes, he was 100% sold. All it took was a couple of video clips of kids waterskiing, spinning the pottery wheel, roasting marshmallows and screaming "I love camp!"
When the video wound down, Sam looked at me and asked "Mom, why are you crying?" Luckily it was easy to come back with "I'm gonna miss you so much." That reply was true, but the tears were laden with much more than wistful thoughts about how quickly our kids grow and leave us.
I feel grateful that we can give Sam the opportunity to go to a posh sleepaway camp where he can get away, enjoy the activities he loves, and make new friends. But if I'm being brutally honest, I'm also heartbroken that Charley will not have the chance to enjoy that experience. And I am stressed about finding something he can do that will keep him occupied and happy all summer.
Speaking of occupied -- and here I will break into the good news -- we are now extremely busy making plans to move forward with our research that has resulted in promising findings. To begin with, Marie Payton, our new Senior Clinical Consultant, is reviewing FDA-approved drugs and some nutritional supplements that show promise as a treatment for DMD. She is working with Dr. George Vella and a pharmacologist consultant to determine which of these treatments are ready for human clinical trials. Marie will outline a clear strategic plan for getting trials up and running. Our goal is to generate trial-based evidence that will help doctors determine which existing drugs should be prescribed for DMD boys to help them maintain strength until new therapies are developed.
That day is not very far off into the future. In fact, several new therapies -- such as a drug called Ataluren and a treatment called exon skipping -- are already in clinical trials. In addition, several biotech companies and scientists we are funding are on target to begin clinical trials of exciting new therapies within the next 2-5 years. This is not a pipedream. This is not basic science that may one day in the far off future lead to an exciting discovery. Every project we support has a clear scientific plan with a budget and timeline. The question that guides us is always "When will this be ready for clinical trials?" Of course science is somewhat unpredictable, and along the way plans can change. However we work with our researchers to ensure that they keep their eyes on the prize. And of course we all know that the ultimate prize is an effective treatment for all boys with DMD.
If you were not able to donate to our 2007 annual appeal, please consider making a donation now on our "Donate Now" webpage. Our 2007 appeal was down 44%, so we have a lot of ground to make up. Preparing for and implementing clinical trials is a very expensive prospect. Thank you for your support. Please stay tuned for more news of our progress.