Issue 4 » Time is Running Out for SCV Teen with Deadly Genetic Disorder
Update on Sebastian Velona Foundation’s Race to Cure Batten Disease CLN8
Story and Photos by Stephen K. Peeples
There’s good news and bad news in our third update on a Santa Clarita Valley family’s race to find a cure for Batten Disease CLN8.
As detailed in our last issue, the neurological disorder strikes children, including 14-year-old Sebastian Velona (nicknamed Sebi). It degenerates their nervous systems and is usually fatal by Sebi’s age.
First, the good news. Through tireless fundraising efforts and a personal contribution of $500,000, Sebi’s mom Teri Hughes Fox and her husband Michael E. Fox (Sebi’s stepdad) have now raised more than $1 million for the nonprofit Sebastian Velona Foundation, which the couple established in early 2017.
More good news is that public awareness of Batten Disease is on the rise, thanks in part to the efforts of one of the SCV’s elected officials and Sebastian’s birth father.
In May, the California Senate approved a joint resolution introduced by Sen. Scott Wilk (R-Santa Clarita) declaring the first weekend of June 2018 and each year to follow as “Batten Disease Awareness Weekend” statewide, after Chris Velona reached out to Wilk. The resolution generated valuable press and social media exposure about the disorder.
But there’s still a long way to go on the quest to find a cure.
Since Batten Disease CLN8 is a genetic disorder, replacing bad genes with healthy ones may provide a cure not only for Sebi, but also for untold other children and adults affected by genetic disorders.
The Foundation’s fundraising goal is $3.5 million, to pay for a full clinical trial of gene replacement therapy.
The clinical trial, to be conducted by a “dream team” of genetic research specialists at The Children’s Hospital in Columbus, Ohio, will include Sebastian and a test group of other American kids fighting Batten Disease CLN8.
The goal is to secure Federal Drug Administration approval of the treatment and get it into general use as soon as possible.
First, though, the initial donations and personal funding have allowed the Velona Foundation to move forward with preliminary efficacy studies, as well as production of the clinical vector, a harmless virus used to transport a replacement gene into the body via an epidural injection.
“We just put up our last $500,000 to pay for developing the clinical vector,” Teri said. “It’s a huge step forward and will buy us some time. But the Foundation still needs $1 million to get to the clinical trial. Once started, that would take about six months. So even if everything was to fall in line, the earliest Sebi could be into treatment is seven months.”
Which brings us to the bad news: Time is running out fast for Sebastian.
The disease that’s attacked his nervous system relentlessly for nearly a decade is now attacking more aggressively every day.
He’s rapidly losing the ability to see, speak and walk, and the debilitating seizures he suffers have become more frequent, severe and damaging.
The harsh reality is that Sebastian may not live to see a cure for Batten Disease CLN8. That’s why donations to the Sebastian Velona Foundation are so urgently needed right now.
“We need to raise the next million dollars like yesterday,” Teri said, adding that she and Michael have now “tapped out” their personal wealth, and would never expect others to donate if the parents weren’t all-in.
The Foxes vow the Foundation will carry on with this quest no matter what, because if the gene replacement therapy indeed proves successful, it will revolutionize treatment of many other genetic disorders.
“We’ve got to get some major people to commit right now so we can fully fund the clinical trial,” Teri said. “That could really be our home run.”
We encourage you and your company to donate to the Sebastian Velona Foundation both now and in the future with recurring donations at CureBattenCLN8.org. Please also contact Teri or Michael to arrange a future fundraising event. Meanwhile, you can send and receive Sebastian’s Healing Hugs on Facebook at HealingHugs4Sebi.