Big Money from a Small Person
Big Money from a Small Person
Several months ago, I received my nephew's bar-mitzvah invitation in the mail. Among the usual enclosures, such as a card inviting us to a kids' nighttime party and a response envelope, was a hand written card with the following note:
Instead of gifts for my bar mitzvah I would like people to give money to a charity called Charley's Fund. Charley, my cousin, has Duchenne Muscular Dystrophy and Charley's Fund is raising money to help find a cure. This disease makes children's muscles get weaker and weaker and eventually stop working. I feel very lucky to be able to stand at the bema on my bar mitzvah day. My cousin Charley won't have the same privilege unless a cure is found. I want him to stand at the bema like me. Please help me make this happen. Thanks, Jeremy
This note precipitated an influx of donations. Many of the checks were accompanied by letters expressing pride and admiration that a 13-year-old boy would willingly forfeit what was sure to have been a bonanza of cash and electronic goodies. To date, donations in honor of Jeremy's bar-mitzvah total just over $56,400!!!! Thank you, Jeremy, for purchasing the two machines necessary for our exon skipping project in the Netherlands. You got some electronics after all, although I don't think a spectophotometer and an FPLC (an apparatus to purify and/or desalt antisense oligonucleotides) are the gadgets most kids envision getting as bar-mitzvah gifts.
At the celebration this past weekend, it was an overwhelming sensation to look out at a sea of faces and know that literally every person there has supported Charley's Fund. Many of Jeremy's classmates thoughtfully donated in increments of $18 to imbue the gift with luck (in Hebrew, the number 18 symbolizes life). Cousins and friends gave well beyond what I imagine must be their usual allotment for bar-mitzvah gifts. One couple was so moved by Jeremy's note that they gave $5,000 to become Founding Supporters in honor of this occasion.
This incredible fundraising push was truly a group effort. It took Jeremy's leadership and inspiration, the generosity and enthusiasm of his guests, and the support and guidance of his family to make it happen. Thank you to everyone who helped inculcate Jeremy into the culture of mitzvah. Together, we raised significant funds for medical research that could help Charley stand tall and proud at his own bar-mitzvah when he turns 13.
Speaking of the research, the latest update from the Netherlands is very encouraging. The Dutch research team we are funding was just awarded “orphan drug status� by the FDA for its exon skipping therapy. “Orphan drug status� provides legislative, economic, and regulatory incentives to our research team. This will decrease the time and money required to bring this hopeful therapy to market. We are excited that Prosensa/Leiden University Medical Center is the first research team in the world to earn this coveted designation from the FDA for exon skipping in Duchenne Muscular Dystrophy.
Here is a summary of how the research is progressing: Children with DMD are missing a protein called dystrophin. Our team of scientists has discovered that they can successfully restore dystrophin production in Charley's muscle cell culture. In the next few weeks, they will begin testing exon skipping in boys with DMD. This is the first time that exon skipping has ever been tested in humans. The compound will be injected into the biceps of several teenage boys with DMD. Researchers will monitor the subjects to make sure the treatment is safe and effective.
Once this is established, they can move on to a systemic delivery study, which will involve intravenous injections. Remember, this treatment will not work for Charley and other children with Duchenne unless it can be delivered to all of the muscles of the body. The scientists hope to take advantage of the “highway� of blood vessels throughout the body to accomplish this difficult task. Most of the funds we are contributing to this project will be used to develop a better system for delivering the treatment to all of the body’s muscles. We receive frequent updates from our research team. We will keep you posted as the research moves forward.