Lab Work Beginning
Dear Friends…
Greetings from the humble home offices of The Mitchell and Friends Foundation in St Louis, MO, where we just enjoyed a refreshing spring morning rain. In addition to penning this monthly newsletter, I am also currently gazing at Mitchell Herndon’s memorial tree, planted in his ashes three years ago outside his former bedroom window. (Perhaps against his wishes, I converted our son’s bedroom into my home office.) The tree is a Scarlett Fire Dogwood and has been genetically engineered to grow in the midwestern climate. Its bright pink flowers are just starting to bloom, as they normally do this time of year.
Speaking of genetics, on to business…
With your support, we are two-thirds of the way toward our goal of raising $150,000 for research into Mitchell Syndrome. While we continue to increase awareness and identify new patients (we know of more than 20 right now), our real goal is to develop medical treatments to save the lives of Mitchell Syndrome kids. And in that battle, there are two important fronts on which we are making progress.
Front #1: Mitchell Syndrome Studied by the Department of Neurology
First—here in St. Louis—we were able to present our second $25,000 donation to Dr. Jin-Moo Lee, Chair of the Neurology Department at Washington University. This donation will be added to the Mitchell Syndrome Fund. While that fund grows, Vice-President Michele has been meeting with the doctor-scientists at Wash U to chart the best course for research. We continue to search for a dedicated scientist to lead the way, but even as we do so, Wash U has already started using donated funds to conduct preliminary research on the possible application of Antisense Oligonucleotide (ASO) therapy, and how one version of it might work on Mitchell Syndrome patients. Michele and I are still learning what ASO therapies are—and how to pronounce the dang word—but our rudimentary understanding is that the process knocks down the excessive amount of ACOX1 activity believed to be the driver of disease in Mitchell syndrome. (Mitchell Syndrome likely results from an abnormal ACOX1 gene.) According to Foundation Scientific Advisory Board member Dr. Robert Bucelli, the use of ASOs in a patient with Mitchell Syndrome is still a couple years away. Nonetheless, the work on developing these treatments has already started at Wash U in the laboratory of Dr. Timothy Miller, in partnership with Dr. Kathleen Schoch.
Front #2: Houston Here We Come
Additionally, later this month, we’ll be traveling to Houston Methodist Research Institute to meet with Dr. Hyung-Lok Chung, another member of our Scientific Advisory Board. Dr. Chung was the first to identify Mitchell Syndrome in fruit-fly experiments while working with the Undiagnosed Diseases Network in 2017. Even though he never met Mitchell personally, Patient #1 has always had a special place in Dr. Chung’s heart. Following the UDN, Dr. Chung moved on to Houston Methodist, where he is setting up his own lab and eager to investigate the promising prospect of riboflavin treatments in Mitchell Syndrome patients. In addition to touring Dr. Chung’s lab and delivering our first $25,000 donation to his work, we’ll also be meeting with several other Mitchell Syndrome parents. Expect an exciting and selfie-filled report in next month’s newsletter.
All exciting stuff. Thanks for your part in helping this organization take root and grow. The Mitchell and Friends Foundation not only helps keep Mitchell’s memory alive, but also—like the tree outside his window—reminds us what good things can grow from such terrible tragedies.
Of course, these good things take time. But with persistence, patience, research—and a little spring rain—we can hopefully defeat the rarest of rare diseases.
Sincerely,
Matt and Michele Herndon
