Updates from Charley's Mom

Charley is 9 and three quarters years old. When I was a kid looking forward to turning 10, all I could think about was "double digits" and how cool and mature it seemed to have two digits in my age. I felt the same way last year, when my older son Sam turned 10. To have my little boy hit that double digit number seemed like such an important and exciting leap. But as Charley's birthday looms, it's not numbers in my head but words -- specifically the phrase "wheelchair from 10-12 years old." I have repeated those words -- and continue to repeat them -- over and over and over when I tell my story, to teachers, to new acquaintances, at fundraisers. And now my world is colliding with those words. A phrase that became almost rote as I repeated it so many times is all of a sudden fresh and raw and frightening.

We have moved mountains over the past 5 years. If we continue at this pace, Charley and other boys with DMD will surely have not one but several treatment options to extend and improve their lives. Research we got involved in when it was still being worked on in cells and mice has now moved to the clinic. Prosensa's exon skipping drug is in clinical trials in several European countries. Sildenafil is being tried in 30 DMD boys at Kennedy Krieger Institute in Baltimore. Acceleron and Biomarin are two other companies that have DMD drugs in clinical trials. Remember that when Charley was diagnosed in 2004, there was not one human clinical trial for a promising treatment. The progress is astounding.

I know Charley will have treatment options. But I also know that he is 9 and three quarters, and he has four months to go until he's 10 and then we're in that "wheelchair from 10-12 years old" phase. Not every boy with DMD loses ambulation between the ages of 10 and 12. Chronic steroid treatment has prolonged ambulation for approximately two years, giving boys extra time on their feet. And some boys who don't use steroids stay on their feet longer than average. Others end up using a wheelchair when they are 8 or 9. So I don't know exactly how long Charley has to continue playing backyard soccer and "balloon ball" (a really dumb game in which he and his sister bop a balloon up in the air and then both of them try to get to it first, nearly colliding every time) and getting in and out of the car without help.

Nonetheless, I'm determined to stockpile some incredible experiences now. Starting today, I have officially declared this the Year of Doing Everything. From Summer 2010 through Summer 2011, Charley and I and the rest of our family are going to do all the things we keep saying we should do, but never get around to. I'm going to focus on Charley's choices knowing that soon it may be hard or even impossible for him to do the things he's always wanted to do. We'll surely be hitting a lot of professional sporting events. Top on his list are a game at the new Yankee Stadium, watching the men's finals at the US Open (he'll probably have to settle for 3rd round given our budget!), and any professional basketball game but ideally the Lakers. He has his heart set on a concert featuring either Black Eyed Peas or Jay-Z. And a trip to China or Mexico are both top choices (guess where we're going?!). This is an APB to all my friends and family and acquaintances -- if you have freebies or amazing seats to any of the above that you got through work or through a friend or you can't use them or you have extras, please think of Charley! A huge priority for me this year is to pack it with fun.

Charley is still bopping around in his usual exuberant manner. He's tiny for his age (thanks to chronic steroid use) and his 'fro is way-out (thanks to a stubborn refusal to trim his hair) and he's been practicing his dance moves, so the overall image he projects is a very cool, compact dude on the move. I'm still betting that all the amazing stuff we do this year will be his firsts of many...and that as a teenager he'll be climbing the rafters to his seats in Yankee Stadium and standing on two feet grooving at an Eminem concert. I hope we have a year packed with sporting events and concerts and an incredible vacation, and that it ends up being practice for a long life filled with these unforgettable experiences.

Posted by Tracy Kramer Seckler at 10:48 PM | Permalink | Comments (0)

Sometimes for a few blissful minutes, I forget that Charley has DMD. To you that might seem impossible, since we're constantly faced with daily reminders of Charley's disease. Every evening we follow a rigorous routine of supplements and stretching. Since long walks are challenging, we often have to break out our handicap parking placard. And while Charley's brother and sister juggle soccer games and karate lessons, we always have to make sure Charley occupies himself with ping pong, board games and other less strenuous "substitute" activities.

Yet despite the significant accommodations we make for Charley on a daily basis, we still sometimes get lulled into feeling like he's just a typical kid. He has such a passion for sports and outdoor play, and he's such an energetic little bugger. He is just not the type to sit inside and mope about DMD.

This week, we got a harsh reminder of how fragile Charley is. During a recess soccer game at school, Charley was playing goalie. He almost always plays goalie because running up and down the field is way too exhausting for him. He blocked a kick with his hands and the ball bent his handbackward at the wrist, breaking the bone. After a few minutes of rest and ice, Charley got back in goal to finish the game. But back in the classroom, his teacher could tell Charley wasn't himself. She called to say, "For a kid who doesn't generally complain about aches and pains, I can tell something was wrong."

So to the ER we headed, and sure enough the xray revealed a buckle fracture of the left wrist.

The hardest part is that we just don't know how to be the best possible parents. We want him to play outside; we want to encourage his "nothing can stop me" spirit...but we don't want him to break another bone. Charley's bones are fragile due to chronic steroid treatment, so he is more susceptible to fractures than other kids. At his age, a broken leg could mean the end of ambulation. So it's pretty scary to give him the leeway to play outside with his friends and wrestle with his brother and participate fully in whatever recess activity he wants to do.

Charley has already started arguing with us about how long he has to lay off the sports. I hate to limit him...I want so badly for his exuberant spirit to prevail. But the broken wrist incident knocked the wind out of my sails, I must admit. With these new therapies so close, I know we have to do everything we can to keep Charley on his feet for as long as possible. The responsibilty of keeping him safe and sound has never felt so weighty.

DMD is heavy duty, and I know the thoughts I express here can be pretty depressing. But you know I will always end on a positive note...We're lucky that as far as broken bones go, this was a minor fracture. And it was his left arm, thank goodness (he's a righty)! And two of his 18 cousins also happen to have broken arms this week...so Charley's in good company. He can't wait to compare blue casts with his cousin Izzy to see who has more signatures!

Posted by Tracy Kramer Seckler at 10:08 PM | Permalink | Comments (1)

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)