MARK YOUR CALENDARS: March 19, 2022
On Saturday, March 19, a group of people who love Miller are putting together an event to raise funding for the research trial, Cure ATP6, by hosting a golf tournament at the Coleman Country Club followed by dinner and some music. The details are still being ironed out, but please save the date, would ya?
We’ll share more info as we have it, but you’re welcome to contact Josh or I with any interest or questions. In a nutshell, expect fun, food, golf, an auction, music, drinks, and maybe a bit of cold weather based on our delayed winter… but SAVE THE DATE: MARCH 19 IN COLEMAN, TEXAS!
Why We’re Raising Funds
Here’s the deal: a research project specific to Miller’s mutation of Leigh Syndrome is in the works – but we need funding.
Please hear us when we say: we NEED funding. This could be a cure for our son. This could mean healing this side of Heaven. This could mean he could walk independently, run, play, and talk – oh to hear my sweet boy’s voice! You guys have shown up in big ways for our family in his five years, and we are eternally grateful. Humbly, we come again asking for help. We cannot do this alone.
Back in March 2021, we met with scientists from the University of Texas Southwestern Medical Center via Zoom and listened to them present their scientific concept for developing a cure for our children: they will utilize gene therapy to silence and replace the low-functioning protein within the mitochondria with a nucleus-coded gene.
The estimated budget of the project is $600,000 for a period of three years. It will be funded through donations via the Rare Village Foundation out of McKinney, Texas. We – the parents of children with ATP6 mutations – have the responsibility to organize fundraising campaigns for the project. The Rare Village Foundation will allocate those funds to the research team.
Let me also say this is not this researcher’s first project in gene therapy or with Leigh’s. Seriously! The initial project, Cure Surf1, is awaiting FDA approval. THAT MEANS IT WORKED. Their scientific concept for developing a cure for Surf1 worked. And the mama who initiated it all, Kasey Woleben, encouraged the research team to focus on ATP6 next. THIS IS REAL AND IT IS HAPPENING.
Thank you, Jesus. And God bless Kasey.
Kasey has a son, Will, who turned 10 in December. TEN. And he’s pretty cute to boot. He was diagnosed with Surf1, another type of Leigh’s than Miller, at the age of two. He deserves a cure, just like our Miller, and the other 14+ children around the world battling ATP6 right now.
To reiterate, while Leigh’s is rare, including both genetic and nuclear occurrences, dozens of mutations are known to cause Leigh Syndrome. Most of them are located in the nuclear genome; only 10%-30% are genetic mutations. That’s us – Miller and me (Jac) carry the genetic mutation in the ATP6 of the mtDNA in the 8993T>g gene. Whether the mutation is in the nuclear or mitochondrial genome, disruption of any of the complexes of the respiratory chain leads to a significant decrease in the production of adenosine triphosphate (ATP).
Listen, we don’t have to understand all of this because these scientists do. We know they do because their efforts have proven fruitful. This is not in vain. This is a very real chance for a cure for our son.
You can see our boy’s beautiful face (a pic snapped at an American Cancer Society Cattle Baron’s set-up day nonetheless) along with other cuties fighting ATP6 on the website. As you can see, we’re at $100k. Honestly, our goal is to raise a minimum of $250,000 to initiate this project and then pray it gets picked up by big pharma to cover the rest of the trial’s needs. Once we have funds raised, the researcher can get create a mouse model, and see if the theory works. This happened for the Cure Surf1 research project – it was funded, in collaboration with UTSW, by Taysha Gene Therapies.
So, please save the date. Call, text, email or DM us if you have any questions or thoughts or would like to help. We’ll take it all!
As always, much love from our family to yours.
Josh, Jac and Mill