Updates from Charley's Mom

I am elated to finally be installed in our new house for so many reasons. First of all, I can now get back to my main job, which is raising money for Charley's Fund. With almost all of the house-related decisions behind us, a good part of my day is freed up to focus on raising funds. I've even become somewhat adept at getting the word out on Facebook, despite my initial aversion to that platform. Speaking of Facebook, Benjy and I are hosting a contest in honor of our upcoming 40th birthdays to see who can collect more birthday donations for Charley's Fund!

Learn more about this friendly competition.

Check out my Facebook birthday wish...and help me beat out Benjy by making a donation toward my wish!

If you want to join the fray but you don't "do Facebook" (and believe me, I understand that decision), please make a donation via our website in honor of one of our birthdays. On Mar 31 we'll announce the winner for most donations as well as biggest total collected.

Back to the house...It's a huge relief to have more space so the kids (and Benjy and me) aren't in each other's faces 24-7. Sam, Charley and Maisy are thrilled to have their own rooms. I love and adore my extra deep bathtub. But when you live with DMD, it's a fact of life that even the most joyous occasions are tinged with sadness. So while we sit in our new living room looking out the huge windows onto the most beautiful meadow and forest beyond, there is a subdued quality to the experience. We do feel lucky to have this incredible place. But no matter how good it feels to be there, there's always the awareness that we needed this house for Charley because he has DMD and DMD is aggressive and progressive and horrid. While we did a great job of making the house subtly fully accessible, DMD is still very much there with us all the time. A visitor might not notice that the doorways are all extra wide or that the shower in the boys' bathroom has no threshold or that the light switches are all lower than they would be in a typical house. But for us, those details are all stark reminders that one day our son may need those accommodations because he has DMD and then the reality of DMD is on our minds again.

It is highly encouraging that we are moving so quickly to develop new treatments. With our new program Pilot Trials Now, we are pushing forward FDA approved drugs that are ready to be tried in clinical trials for DMD boys. The first trial will begin this year at Johns Hopkins/Kennedy Krieger institute with sildenafil, a Pfizer drug that was developed for hypertension and angina. Animal testing indicates that sildenafil may significantly help the hearts of boys with DMD, so we are hopeful and excited to get this trial off the ground. Clinical trials are not cheap, so we are grateful to have support for this one from the Nash Avery Foundation, Cure Duchenne, and Hope for Javier -- three other foundations run by parents of children with DMD.

Our other research collaborations are moving along too, and we remain confident that a treatment will be developed in time to help Charley. If the next five years are as productive and fruitful as the last five years, we will be in VERY good shape. Of course we need your help to make that happen, so please stick with us as we continue to push forward with every fiber of our being.

Posted by Tracy Kramer Seckler at 12:38 PM | Permalink | Comments (0)

This may not sound right but I have to say it anyway -- thank god the kids are back in school!!!!! Summer was full and fun but those last two weeks were trying, I must admit. Now that Maisy has started to pipe up and add her two cents into the sibling dynamic, all three of the kids can really get on each other's nerves!

I shouldn't let that overshadow the fantastic eight weeks of summer. Charley spent every day at Eisner Day Camp, where he is pleased to be the oldest and -- in his opinion -- coolest kid in the camp. Charley's older brother Sam got to spread his wings a little at sleepaway camp in Northern Berkshire county. Although it's sad to know that Charley won't join him there, we were relieved to give Sam his much-needed space.

In mid-August, we were lucky to have the best weather of the entire summer for our one-week vacation at a friend's house in the Hamptons. All we did was lounge by the pool all day long. Ironically Charley was the antsy one, constantly bugging us to play tennis, "volley-balloon," and card games.

On the foundation front, we spent much of the summer working with Marie Payton, our Clinical Consultant, to map out a plan for implementing pilot trials for FDA-approved drugs. We are excited to have a real roadmap for moving forward. Funds permitting, we aim to have at least one trial start this year and one in the first quarter of 2010. Other collaborations are moving forward on track, including the work at AVI Biopharma and Dr. Jim Ervasti's research at the University of Minnesota. Because these and other novel therapies are so close, it is essential that we identify FDA-approved drugs that can help DMD kids stay strong until new breakthrough treatments are available.

One strategy for coming up with the funding for these exciting projects was to reach out to donors who have not been active for over a year. So far the response has been encouraging. People who helped us get off to a strong start are now coming through once again to help us push the ball even farther down the field. Thank you to everyone who answered our plea for help this summer!

Please get involved this fall --

• come to our Spa Day on Sept 25th (bring a friend on the half-price companion ticket!!)


• watch Benjy and his old buddies play football in the Harrison-Rye reunion game


• do your holiday shopping at Live and Let's Give the weekend of Nov 6-8 in Pittsfield


• make a donation in someone's honor...we'll send a cute card!

  This fall marks many new beginnings: the Jewish new year, Charley's birthday, we move into our new house, and the initiation of Pilot Trials Now. I have a good feeling about all this...
  

  Posted by Tracy Kramer Seckler at 10:23 AM | Permalink | Comments (0)

We've definitely entered a new phase in terms of Charley's awareness of his disease. We are transitioning out of the "honeymoon phase," when the limitations of DMD haven't really set in. Until recently, we could kinda sorta blend in with the crowd. Our evening routine has always been tough: In addition to the usual bedtime routines Charley has to take 14 pills with a powdered mixture and then the "yucky drink", a soy-based food supplement from China that smells and tastes pretty much like sewage. We also have to stretch him for 15 minutes every evening. And in the mornings before school -- amidst the mayhem of frozen waffles and unruly bedheads and socks that don't match -- we throw in 12 pills plus a morning dose (2 oz) of the yucky drink. So while these rituals that start and end the day have always been intense, in between we have been lucky enough to function almost like a typical family. Charley is mainstreamed at school, and for the most part he enjoys the same activities as other kids. He has to limit his sodium and sugar intake and he has to rest when he gets tired, but other than that he has been keeping up great.

Over the past few months, however, Charley and his brother have been developing more of an awareness of Charley’s limitations. Just the other day, they were playing with a soccer ball in our backyard. Sam had to retrieve the ball for the 10th time, and in frustration he yelled “I hate this!” Thankfully he quickly clarified his outburst. “I don’t hate YOU, Charley,” he explained. “I love you. I just hate your condition.” It took a while for Sam to understand his feelings of anger, and in fact it is still a “process.” Sometimes his anger is directed at Charley himself, and that is really hard for everyone.

Charley has always been less of an intense personality than his older brother Sam. If he is angry about having DMD, that anger has yet to surface. He does express sadness, and that is very hard for me to bear. This winter he was devastated that he could not ski. One Saturday, I made the idiotic mistake of bringing him with me to the ski shop to buy a new pair of gloves, as we were on our way to go sledding and he had lost his umpteenth glove of the season. We ran into a family we know, and all three kids were booted up, clearly on their way to the local ski mountain. Then a friendly young salesman said, “Hey buddy, are you ready to hit the slopes?” Charley just stared at him, and when we left the store he burst into tears.

We adapt with the times, though, and as soon as the weather improved we bought Charley a bright red electric Razor scooter so he can keep up on the bike trails. Charley’s new ride has brought back the smiles, at least for now. On a recent trip to the Franklin Park Zoo, he had more fun zooming around the crowded walkways than he did looking at the animals. The highlight of this outing for Charley was practically taking out three toddlers as he zipped past them at top speed. The fact that scooters are not allowed in the park – only he was allowed -- made it all the more exciting.

I’ve got lots of adventures planned for this summer…elderly ladies and toddlers beware…Charley is on the loose!

Posted by Tracy Kramer Seckler at 12:28 PM | Permalink | Comments (0)

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.

Posted by Tracy Kramer Seckler at 9:50 PM | Permalink | Comments (0)