Updates from Charley's Mom

Just when I think life can't get any busier...ok, let me just spit it out, we got a puppy!!!!!

Almost everyone I know looks at me like I am crazy, but I feel so fantastic about our new arrival. Charley and Maisy absolutely love her, and Sammy is finally getting used to having a dog around. Yes, I am busier than I ever thought humanly possible, but I have been on more walks in the past three weeks than I have been on in the last three years. And the smiles Stella has brought to Charley's face are irreplaceable. It is definitely extremely difficult to balance our drive toward a treatment for DMD with taking the time to enjoy what we have now. Every minute that I am walking the dog or taking the kids for a swim (the proverbial "smelling the flowers"), there is definitely a shadow of guilt that I am not cultivating donors or checking in on scientists or updating the website. How can I not do every single thing possible to make sure we reach our goal in time to help Charley and so many other little boys who have such a real chance at a better future?

One answer to this dilemma is to get some help, and thank goodness we can do that. Friends are constantly calling in with fundraising ideas and hands-on help. This summer Charley's Fund will host the bake sale at Egremont on Parade (July 27 at French Park in Egremont). We will also benefit from a very special concert featuring principal musicians of the NY Philhamronic on August 11 at a historic mansion in Lenox, MA. Both of these events are being handled by friends who stepped forward to offer their assistance. Also thanks the help of several close friends, we are developing a fantastic Charley's Fund gift catalog that will feature hip new merchandise...it'll be ready in time for the holiday season.

Charley is at day camp, in the loving and supportive hands of the Camp Eisner staff. Thanks to a daily regimen of supplements and stretching, he is hanging on very well while we aggressively push forward toward a treatment for DMD. We've begun to sort through our investments in the medical research, bringing a close to projects that do not seem to hold promise at this time and honing in on the therapies in development that are showing positive results in preclinical testing. We are in the process of updating the research page of our website so all of our supporters can keep track of our progress. I will let you know as soon as that is done.

Happy July 4 to everyone out there. May this Independence Day mark another year of independence for our country, and also for Charley and all of his DMD "brothers" who are holding on tight until we reach our goal.

Posted by Tracy Kramer Seckler at 10:49 AM | Permalink | Comments (0)

We’ve just arrived home from a phenomenal two weeks in Israel. Charley’s cousin Xander became a bar-mitzvah at the Western Wall. His generous parents invited us along for a fabulous tour of Jerusalem and its environs. Charley’s favorite highlight was floating around in the Dead Sea and covering himself in its legendary therapeutic mud. A dare-devil jeep ride through the Golan Heights and our visit to a secret underground bullet factory were close seconds. These richly historic adventures were balanced by less intellectual endeavors, like taste-testing falafel from every stand we encountered and horsing around in the back of the tour bus with a gaggle of siblings and cousins.

Throughout the trip, that cruel DMD dichotomy was ever-present. On the one hand, there was the sadness and fear each time we arrived at a site requiring physical agility. How could we ever again experience this camel ride or this cave crawl? If we come back in two years will Charley be able to get around at all? While cousins and friends happily discussed which sites they will see on their next visit, we were left wondering if we could ever again navigate the narrow paths and uncountable stone steps.

On the other hand, I doubt anyone on earth feels as deep a sense of joy and wonder and gratefulness that I felt almost every minute of the trip. Certain mundane moments stand out in my mind as almost impossibly beautiful:

standing with the kids on the street in Tel Aviv devouring a fresh mound of sweet strawberries that we had just purchased (and bargained for) at the Carmel market

tears streaming down our faces from laughing so hard when Maisy transformed “Boker Tov” (Hebrew for Good Morning) into “Booger Tov”

hooting and clapping during the apres-dinner entertainment at a Moroccan restaurant, which was an elaborate bellydancing show performed by all the younger cousins

Thanks to my incredible family who sponsored this trip, we came home with a brainful of beautiful memories that will forever brighten our days.

The two weeks away provided a crucial mental break for Benjy and me. Learning about the complicated history of the Middle East and haggling over shekels were rejuvenating diversions from our usual work. We are now back in the saddle, raising money and expediting research.

The vacation came at a crucial time, as we now need all the energy we can muster to raise the money we need during these challenging economic times. The uncertainty of the economy has caused many of our donors to be extra cautious this year, cutting back on expenses that are not absolutely necessary. It is our job to convince people that keeping the medical research on the fast track is a necessary expense that we cannot live without. In fact our own son Charley and thousands of other little boys literally cannot live without it.

Thankfully, several supporters have recently stepped up to the plate in a very big way to help us during these trying times. This week we received a $100,000 pledge from a donor who learned about us through the film Darius Goes West. Another donor presented us with a challenge grant. She will match up to $100,000 raised by May 11 -- Mother’s Day – in honor of the tireless efforts of all moms of DMD boys. Please consider making a donation toward this goal. To be presented with $200,000 on May 11th would be the most incredible gift I could imagine this year.

The fact that we need money is actually a good sign. All of the research we fund is paid in increments based on milestone achievement. We do not pay out our grants and investments in a single lump sum. Instead we pay out funds when we receive a satisfactory progress report from the scientist doing the research. This enables us to cease funding a project if a researcher hits an insurmountable roadblock.

Upon our return we faced news of milestones reached by several of our key researchers, including AVI Biopharma, Dr. Dean Burkin of University of Nevada at Reno, and Dr. James Ervasti at the University of Minnesota. That brings me to the positive note on which I must always end, a writing technique that has mushroomed into a full-fledged superstition. Happy spring, and please tell everyone you know about our Mother’s Day deadline to maximize the $100,000 matching grant!

Posted by Tracy Kramer Seckler at 6:26 AM | Permalink | Comments (0)

Extraneous and repetitive e-mails annoy everyone, I know. But there's one e-mail that keeps popping into my box every few minutes, and I love it! "You've Got Cash" is the subject, and it is a notification from Paypal that a customer has bought a Darius Goes West dvd (or two or twenty).

While I sit at my desk working on upcoming events, preparing the 2008 Charley's Fund budget, and tracking down the painter who is supposed to be finishing our new downstairs bathroom (just a sampling of my daily activities), every few minutes I hear the "blip-bleep" that indicates a new DGW order. I recognize lots of the names as friends and supporters who have been helping Charley’s Fund since we started this fight against DMD. But I am really struck by the number of people I do not know. Darius and his friends have catapulted our cause into the public eye. Customers from Iowa, Hawaii, Canada, even Australia are purchasing the film.

Although the documentary is a wrap, the 12 crew members are continuing their involvement in our fight for a cure. Know About It!, the program that brings the film and related lesson plans to middle and high schools, is up and running. If you are a student, teacher, or parent, check out www.DGWKnowAboutIt.com to learn more about how to get this program into your school. Crew members are speaking at conferences around the country. Plans are in the works for Darius to record a hip-hop album with some favorite rappers. I literally could not dream of a better gift for Charley’s Fund -- 12 young, handsome, hip guys volunteering to travel from state to state, rallying people all over the country to help raise money for our fight against Duchenne muscular dystrophy. Thank you, DGW crew, for your tireless efforts. We are so proud that you will be honored on May 15 at the Matt’s Promise gala at New York City’s Cipriani!!

Ranging in age from 18-29 years old, the Darius crew seems very young to be so determined to help. But we have even younger supporters who are making big sacrifices to push us closer to a treatment for DMD. Charley’s first cousin Solomon Seckler, age 13, recently generated $30,000 in donations to Charley’s Fund by forfeiting his bar-mitzvah gifts. Even when pressed by people who really wanted to buy him something (like his grandmother and Benjy and me), the only request Solomon made was “a check to Charley’s Fund.” I like to think of myself as having been a good kid, but I still can’t imagine saying no thank you to gifts from hundreds of people in honor of a major milestone that I worked really hard to achieve. Solomon even handed over cash that people gave him on his big day! Thanks to Solomon, whose bar-mtizvah theme was Extreme Sports, we now have the “Extreme Mitzvah” version of the Charley’s Fund t-shirt. If you are 13 years old, perform a major mitzvah for Charley’s Fund and we’ll send you this ultra-cool jersey-style t-shirt designed by Solomon.

The $30,000 Solomon raised will be used to finance research being conducted by CombinatoRx, the biotech company in Cambridge, Massachusetts that is working on a DMD therapy. Next week I am headed to the lab in Cambridge to tour the facility and get an update on the cutting-edge research that this company is doing to develop a treatment for Charley and thousands of other DMD boys. Also in January, we make our second payment to AVI Biopharma, the company in Portland, OR that is preparing for a human clinical trial of its exon-skipping therapy. If this therapy works the way we hope it will, it should transform DMD into a much milder form of the disease called Becker muscular dystrophy. People with Becker MD can have mild symptoms, and some are even asymptomatic! We are very pleased that so far the scientists at AVI Biopharma are sticking to the timeline they outlined in their agreement with Charley’s Fund. Speaking of exon-skipping, another company we have funded called Prosensa was recently covered in the New York Times and other major newspapers for moving ahead with their clinical trial in the Netherlands. Prosensa’s scientists have shown that they can produce dystrophin in a single muscle in a DMD boy. They are getting ready to test delivery of the therapy to all the body’s muscles via under-the-skin injection. Six boys will participate in the trial. This is truly historic, and we are waiting with bated breath for the results.

I hope you now know that when I don’t write in the blog for months at a time, that doesn’t mean nothing is happening. To the contrary, so much is happening that I don’t make the time to update you. My new year’s resolution is to update this blog at least every month (also to get in shape but I’m less optimistic about that one). Please hold me to this by e-mailing me with lots of questions about the research, our fundraising efforts, and Charley. By the way, for those of you following the “hair saga,” he is now sporting a way-out 1970s afro. Depending on how you look at it, this could qualify as ending on a positive note. For me, the positive note is that tomorrow he has an appointment with the barber!

Posted by Tracy Kramer Seckler at 9:34 PM | Permalink | Comments (0)

Anyone who hung out with Charley this summer knows he has a new look. He acquired some key accessories to look “cool,�? including mirrored sunglasses, the silver Believe necklace with black suede cord, and navy crocs with strategically placed jibitz (he even has to accessorize his accessories).

Fashion is not the only arena in which Charley is expressing his independent spirit. This summer he took the training wheels off his bike and learned how to ride a two wheeler. He took a couple of ice skating lessons in Sun Valley. The skating instructor commented that he was just as good as any boy his age because although his muscles are not as strong, his determination pulled through.

I am deeply grateful that Charley is so strong-willed. But sadly, sheer determination is not enough to conquer the notoriously aggressive Duchenne Muscular Dystrophy. I pray that his will and determination holds out long enough for the science to come through. Thankfully, the scientists are speeding up. In the three years since Charley’s diagnosis, major strides toward a treatment have been made.

The list of potential therapies with a real shot at making a difference in DMD is growing. We are currently funding 12 projects that have a shot at helping this generation of boys who won’t hold out much longer without significant help from cutting-edge medical research. Some highlights:

We have just contracted with Biofocus, a UK-based company, to screen thousands of FDA-approved drugs to see which ones can increase the body’s production of utrophin, a naturally occurring protein that can compensate for lack of dystrophin (the missing protein in DMD boys).

Dr. Steve Wilton at the University of Western Australia is investigating a “cocktail approach�? to exon skipping -- currently the lead horse in the race for a treatment. The exon skipping therapy varies for each subset of DMD boys, depending on which genetic mutation the boy has. Dr. Wilton is skipping multiple exons at once, which will expedite the process so more boys can be treated more quickly.

Dr. Kanneboyina Nagaraju at Children’s National Medical Center in Washington, DC is testing four drugs that could theoretically help boys with DMD. After administering the drugs to mice with muscular dystrophy for a six-month period, he will analyze the data to determine if any of the four do indeed mitigate the muscle deterioration brought on by DMD. If the data are positive, the next step could be a human clinical trial in Duchenne boys.

To get one of these products to the clinic may seem like a miracle, and it is. But it happens. I recently received a letter from a donor living with multiple myeloma, a deadly form of cancer. Miraculously, she has far exceeded doctors’ predictions thanks to several experimental treatments that have extended her life and given her a quality of life she was not expected to have. Her letter is living proof that although we are working to bring about a miracle, it is really and truly doable. Thank you for sticking with us as we do everything we can to save Charley, Aidan, Javier, Dylan, Cooper, and so many other boys.

Posted by Tracy Kramer Seckler at 12:45 PM | Permalink | Comments (0)