Dear Friend,
Less than two years ago, my family and I celebrated the birth of my grandson; JB, named after me. A glorious day...my own heart filled with joy. Time quickly began to pass and I watched JB's little body and face develop into a toddler, I could not help but notice all of the family features he was beginning to take on — reminding me of how our own grandchildren are our ties to days and loved ones gone by.
However, there was something different — over the course of the next several months, it became clear that his genetics were made up of something quite different, through the investigation of some minor delays in his development, revealed the discovery of something that I had never heard of -- JB's body was harboring a secret; one that was unlike anything we knew of, you see all the muscles in his body were deteriorating, my son and daughter in law received the news that JB had a form of muscular dystrophy; Duchenne muscular dystrophy (DMD). The most lethal childhood genetic disorder in the United States. This form of muscular dystrophy did NOT come with a cure, did NOT come with a treatment —instead it came with a very grave prognosis. My grandson's beautiful little body would grow, but his muscles would tear and rip, unable to repair themselves — causing him to most likely be wheelchair bound during his early teens, almost all boys diagnosed succumb to the disorder in their late teens, sometimes in their early twenties as the respiratory and cardiac muscles eventually give out. Currently, the only agreed on approach to DMD involves daily and intermittent corticosteroids, however, this comes with very serious side affects...leading me to believe that there has to be a better way.
As you can well imagine, I began a relentless search of potential cures/treatments for DMD. My exhaustive search led me back to a clinic and physician that my grandson would be visiting; Dr. Brian Tseng at Massachusetts General Hospital for Children and the Jett Program for Pediatric Neuromuscular Disorders (JPPND).
Dr. Brian Tseng is a life long physician and scientist who has worked passionately and solely for Duchenne. He currently is an associate professor at Harvard Medical School and the director of the JPPND at Mass General Hospital for Children.
After speaking often with both Dr. Brian and the President of the Jett Foundation (JF), co-founder of the JPPND; Christine McSherry, I realized that the work they were currently funding would be the most aggressive form of treatment that my grandson would benefit from in the immediate future. While there are many avenues of very promising research currently in the pipeline — and I have throughly investigated all of them, I realized that if JB is not kept as healthy as possible today, preserving as much of his muscle integrity as possible — his chances of being cured by these promising avenues; gene and novel therapy, would be slim.
Dr. Brian Tseng has assembled an aggressive multidisciplinary clinic at MGHfC. I learned that this is a new model for DMD, that this is only the second institution in the country with this approach to care. Dr. Brian recently presented his clinic model to an esteemed group of colleagues at the national neurology conference this past month showing the benefit to patients under this model. Before this clinic, there were no standards of care — care for boys with DMD was fragmented and reactive. Dr. Brian provides proactive care, doing everything he can to prolong muscle integrity and life for these boys. JF is a nonprofit organization that has partnered with Dr. Brian and the clinic. Together, they have structured a level of care that will surely help keep JB healthier.
Not only has this partnership between Dr. Brian and JF created a dynamic center (JPPND) for boys with DMD to grow and foster, they are also busy identifying supplements and approved FDA compounds that might be beneficial to JB. The timeline for a new drug/compound through the pharmaceutical companies can be up to seven years in the making and costs can quickly jump into the millions. Dr. Brian and JF have contracted a company; Myomics, that will quickly screen up to 2,000 compounds per month at a fairly inexpensive cost; about $3,000 per compound. This puts a treatment for JB on the map for the immediate future. On a recent visit to Myomics, I learned that this technology is the only one of its kind. It actually measures the muscle contraction, thereby telling the scientist which compounds might be beneficial in maintaining strength for boys with progressive muscle weakness/wasting. What was even more amazing, I learned that just two weeks ago — this lab had two significant breakthroughs — they were able to synergize two compounds together; causing a muscle contraction of 150% higher than the norm — these results came in less than 30 days! Secondly, they were able to move from using DMD mouse muscle (mdx mouse) to human DMD muscle fibers, giving them the capability to truly reproduce what is actually happening in the muscle fibers of those with DMD. This is quite a breakthrough for the entire community affected with DMD! This project has been named appropriately, Compound Quest (CQ). CQ's goal is identify the best compounds for boys with DMD and move them quickly into a human clinical trial at MGHfC. Dr. Brian weeds out what compounds should be tested — will select the best and provide a "cocktail" at the end of the human clinical trial — giving this generation of boys the chance to be healthy and alive when future therapy is finally available. The compounds that this tri — partnered group; Myomics, Dr. Brian and JF, are testing -- are compounds that are readily available to the anyone — things most commonly purchased at health food stores or by prescription through local pharmacies. Thereby reducing the long wait time of a "treatment" for JB to months VS years.
To that end, I am very motivated and inspired to help Dr. Brian and JF fund Compound Quest in an effort to test as many compounds and combinations of compounds as quickly as possible. I have since learned of an additional financial motivator -- two matching gift programs that could qualify JF for a minimum of an additional $80,000 of funding! Myomics will benefit from a grant given by NSF of $0.50 for compound testing in DMD. The criteria of the grant requires JF to raise at least $100,000 of private, non-federal monies by July 31, 2009. The NSF will match $0.50 on the dollar up to $1 million dollars (i.e., an additional $500,000 from NSF).
The second opportunity comes from a private foundation located in southern California. Ironically, the foundation is named; The Quest Foundation. This foundation has accepted Compound Quest request for a grant, however it comes as a challenge grant. If $30,000 is raised by JF, The Quest Foundation will match it with the same, $30,000.
Essentially it all boils down to this; if JF raises an additional $40,000 or more (currently they have raised $60,000 in private funding), prior to July 31, 2009 -- Myomics will receive a grant from the NSF of $50,000 and the additional challenge grant of $30,000 from Quest Foundation. $80,000 of FREE money for research!
In my opinion, an easy investment in the future of this disorder; excellent proactive care by a world class institution and a lifelong DMD physician and scientist. Identification of a cocktail that will delay the inevitable progressive symptoms of DMD to help TODAY and a $40,000 investment that doubles our money into research.
As I look at this tiny boy's face, I see myself and my family history in him...I pray for this boy to become a father, as wonderful as my son and to someday experience the same joy that I have had as a grandfather...to someday look into his own grandson's eyes and know that this deadly disorder has been ridden never to hurt us again.
Thank you from all of us,
Jeff Harvey