Where in the World Is Charley Seckler?
Forgive the corniness, but that is the only apt title for this sorely belated blog entry. Over the past six weeks, we have traveled across the country twice, down the East coast, and across the Atlantic Ocean. Our first – and most important – adventure was to The Netherlands, where we met with the scientists who are working on the exon skipping therapy for Duchenne Muscular Dystrophy. Prosensa (www.prosensa.nl) is a Dutch biotech firm that has partnered with Leiden University Medical Center to turn exon skipping into a viable therapy for DMD patients. Charley and his brother Sammy had a ball fiddling with the microscopes at the laboratory. For some reason, eating in the University cafeteria was also a highlight.
A dermatologist performed a skin biopsy on Charley to get a sample of his cells. The procedure was a quick pinch on the arm, and Charley handled it well (only two minutes of rabid screaming). Scientists transformed the skin cells into muscle-like cells to be used in their exon-skipping research. The team had never tried exon skipping in DMD cells with Charley’s specific genetic mutation. We just heard from the CEO of Prosensa last week. Charley’s skin cells were successfully transformed into muscle-like cells. Now the exon-skipping experiments on his cell sample have begun, and all is proceeding smoothly.
Although the trip raised many questions for the boys about why we were there, we still managed to keep it light and have a sense of adventure. In Amsterdam, we visited a zoo, a science museum, and lots of great shops and restaurants. And of course Charley is only five years old, so the facts aren’t 100% clear in his mind. Before we left for Europe, I took the boys to a local bookstore to buy some books about The Netherlands. I was waiting on the checkout line with my books about Van Gogh and the tulip trade, when Charley came running over from the children’s section, excitedly waving a Peter Pan book. “Mommy!� he shouted. “Here it is! Here’s where we’re going…Neverland!�
Two days after our return from Amsterdam/Leiden, the boys and I hopped back on a plane for our quarterly visit to the MDA clinic at Children’s National Medical Center in Washington, DC. There are Muscular Dystrophy Association clinics all over the country, including one close by in Springfield, MA. But the range of quality is astounding. There is very little agreement among physicians about how children with DMD should be cared for. Even basic questions like “Should we increase his steroids dose as he gains weight?� are answered differently by different doctors. Because there is so little scientific data on the disease, we have been seeking out doctors who suggest treatments that they “have a hunch� might help based on anecdotal evidence. Although this is a scary basis for medical decisions, we have no choice but to take a chance on these gut feelings until there is more empirical evidence on which we can rely. We chose the neurologist in Washington D.C. because she recommends a cocktail of nutritional supplements that may –or may not – delay the progression of the disease in Charley’s body. Although it was a long and for the most part extremely boring day at the clinic, we managed to save the trip with a visit to the National Air and Space Museum the following day. All in all, Charley actually enjoyed the trip to “George Washington.�
Next stop: San Francisco, California. This was an amazing journey. Four friends from the Harvard class of ’92 put together an incredible fundraising dinner. They invited their friends and colleagues to cocktails and dinner at the University Club. Benjy and I mingled with the crowd and gave a power point presentation about the fund and the research we are supporting. One of the hosts gave a rousing, motivational appeal speech, and the checks started rolling in. We were lucky to have some very generous “matching donors� for the evening, so any gift above $500 was matched. The guests at the dinner – most of whom we had never met before -- were incredibly interested and supportive. We are still receiving gifts from people who attended the dinner, and from those who could not come but donated anyway, so we do not have a grand total. However, at last count we were flirting with $60,000 from that one night!!! I will update you when we have some firm numbers. In the meantime, a big push for our 2006 fundraising plan is to replicate dinner parties like these all over the country. If you are interested in hosting, or if you know someone who might want to host, please call us at (413) 528-5745.
Our most recent trip was out to Salt Lake City to attend the bar-mitzvah of one of Charley’s 18 first cousins (not all from the same family; with Salt Lake City thrown in there, you never know!). Benjy’s sister recently moved out West to enjoy the mountains and the snow. At last, a trip that had nothing whatsoever to do with Charley’s Fund…just family. We are home now, totally spent, looking forward to weeks and weeks of snowball fights and hot chocolate. Hopefully the farthest we will have to go for a long while is to see a movie or catch a bite to eat in town.
Of course we wouldn’t be comfortable with total quiet, so this weekend we are hosting the director and cameraman from Darius Goes West (www.dariusgoeswest.com), an independent documentary about a boy with DMD who traveled across the country with a group of 10 friends. They will spend some time getting to know our family, and interview us for the movie. Please follow their progress on the Darius Goes West website…with a groundswell of public support, Darius Goes West could be the greatest sleeper indie hit of the century!