Today we’d like to introduce you to Kasey Woleben.
Kasey, can you briefly walk us through your story – how you started and how you got to where you are today.
Over six years ago, our then two-year-old son, Will, was diagnosed with a rare, terminal disease called Leigh Syndrome. We were told that the disease was caused by a defect in a single gene called SURF1, that is critical in the process of converting food into energy. At the time of diagnosis, doctors told us there was nothing they could do to help… no treatment option, no cure, and no hope. We were told our son would most likely not live past the age of ten.
We were devastated by this news, but decided to do everything in our power to fight for our son. We began contacting researchers across the globe in search for answers. After several years of searching with little progress, we finally found the breakthrough we had been looking for. Incredibly, it was right in our hometown of Dallas. UT Southwestern was in the process of starting a cutting edge gene therapy lab, and they were hiring one of the top researchers in the field, Dr. Steven Gray, to lead the lab. After several meetings with Dr. Gray and his team, they felt that Will’s disease was a good candidate for gene therapy, and agreed to begin developing a custom treatment for SURF1 Leigh Syndrome. This was incredible, life-changing news, but it came with a catch. The development of the treatment would be expensive (millions of dollars) and would have to be funded privately.
Knowing that we were racing against the progression of the disease to get our child a treatment, we put all of our energy into fundraising. We connected with several other families who had children suffering from SURF1 Leigh Syndrome and together, we formed the Cure SURF1 Foundation. Over the next 18 months, we raised over $1 million, which helped move the research forward to the point where the gene therapy treatment had been developed and animal models were treated and showing promising results.
We then received more incredible news. The positive results seen in the animal models had attracted the attention of biotech companies. Startup biotech local to Dallas called Taysha Gene Therapies formed a partnership with UT Southwestern to commercialize a number of their gene therapy programs. As part of that partnership, Tasyha would be funding the remaining work to get the SURF1 gene therapy to human clinical trials.
There are still a number of hurdles that need to be cleared before we can get FDA approval and actually get to a point where our son can receive the gene therapy treatment. And even if we get to a point where he can receive the treatment, there’s no guarantee that it works. We have tremendous faith in the team at UT Southwestern and Taysha, but we know this treatment is experimental. Despite these unknowns, we feel incredibly grateful for the position we now found ourselves. Years ago, we were told there was no chance for Will. Now we have genuine hope for not only our son, but for all kids who suffer from this terrible disease.
Over the course of this journey to find a treatment for our son, we learned quickly that we are far from alone. There are MANY rare diseases, most of the terminal, that currently have no treatments or cures. So many families are going through the same desperate search that we’ve gone through in trying to help their children. We’ve been extremely fortunate to make progress on our son’s disease, but we started to ask what we could do to help other families find their own path forward. With that in mind, I’ve partnered with another rare disease mom, Gina Hann, to form the Rare Village Foundation. We’re living in a period of truly transformative medical breakthroughs (AAV gene therapy, CRSPR gene editing, enzyme replacement therapy, etc.) that are opening the doors for treatments of many formerly un-curable diseases. The goal of Rare Village is to empower rare disease families to create their own cures and share our knowledge that we have learned along the way. Saving precious time that rare disease children do not have.
Great, so let’s dig a little deeper into the story – has it been an easy path overall and if not, what were the challenges you’ve had to overcome?
The road has been very difficult. Rare disease parents are given little hope. Cure SURF1 and Rare Village Foundation want to change the way families look at rare diseases. We want to empower families to find cures. Giving HOPE to families who need it the most. The road to a cure is not an easy one. It costs a lot, it’s time-consuming, at times, even heartbreaking. But we must know that our children are leaving a legacy behind and hopefully will change the future of their particular disease.
Please tell us about Cure SURF1 Foundation and Rare Village Foundation.
We are non-profit empowering rare disease families to create cures.
Contact Info:
- Website: www.curesurf1.org rarevillage.org
- Email: kasey@rarevillage.org
- Facebook: facebook.com/therarevillage facebook.com/cureSURF1foundation
Suggest a story: VoyageDallas is built on recommendations from the community; it’s how we uncover hidden gems, so if you or someone you know deserves recognition please let us know here.
