COLUMBUS, Ohio — As Jaren McChesney once quoted, a grateful heart is a magnet for miracles. And McChesney and her husband, Mike, certainly have two grateful hearts right now.
“We are overwhelmed by the support from the community and friends and family and complete strangers, across Ohio, across the country, even across the world,” she said.
The McChesneys have a 5-year-old son named Landon who has a rare genetic disorder. The TBCD gene mutation affects every aspect of his development.
The condition is so rare the McChesneys say they know of only roughly 15 people in the entire world who have it. And that means finding treatment was a huge challenge.
So they set out to find treatment on their own, eventually connected with Rarebase, a California-based public benefit biotech company.
“Our mission really is, for families like this, let’s try to find a path forward for you, instead of it sort of being a dead end,” Rarebase CEO Onno Faber said.
In order to work with Rarebase, the McChesneys had to raise $50,000 by the end of 2021. There were public pleas, fundraisers and even a trip with a sign to College GameDay.
“It was very exciting and nerve-wracking at the same time because we were raising the money, and then it would kind of come to a standstill,” Jaren McChesney said. “And there’d be another surge, so we weren’t sure whether we would make it in time or not.”
But, with a week to spare, they did. And they could not have been more grateful.
“It’s hard to put into words all the outpour of support, so I think everything is achievable if we just stay positive, motivated and focused,” Mike McChesney said.
Meeting that fundraising goal meant the next step in their journey was in motion. Both Landon and his dad gave blood samples that were immediately shipped off to California.
From there, scientists will work to create neurons on which to test already approved drugs they believe could be effective in treating Landon.
“When there’s no drug associated with the disorder, there’s not much they can do, so this is where families and foundations are trying to step up and sponsor research, and yeah, this is what we want to help with and create a platform, a direct discovery platform, that can potentially benefit many people with rare disorders,” Faber said.
It’s a mission that’s personal for the Rarebase co-founder. He also has a rare disorder that causes tumors to develop on his brain and spinal cord. Not having an effective treatment for his condition put him on the path to helping himself and others.
“I do have a lot of hope for the future, and because of the partners that we have, and families we are working with, yeah, we’ll just do everything that’s technically possible to help bring the latest and greatest direct-discovery technology to these type of problems,” Faber said.
Developing the neurons to test will take some time, along with testing wither any drugs are “hits” with those samples, but the McChesneys already know their next mission. To move forward in this process, they will need to raise another $75,000.
“It’s like landing on the moon for us, right, it’s such a big deal,” Mike McChesney said. “I pray that we have to find another $75,000, that those hits happen because that means that there’s a viable option for Landon and other kids again dealing with the TBCD mutation, so again, I think, one step at a time. If we find those hits, that is beyond exciting. I mean, it’s just other-worldly to me. It’s just so hard to put into words the possibilities.”
In the meantime, the McChesneys report Landon is doing great, loving school and progressing in therapy. And the attention surrounding Landon’s journey and the family’s fundraising efforts has allowed the McChesneys to connect with both other families who have children with the condition and with doctors and specialists who are willing to share their input.
“This has been what I’ve been trying to accomplish for the past four years, and it’s actually happening, just getting the conversation started,” Jaren McChesney said. “Because I feel like we’re at the precipice of something really huge, right, so we just have to stay focused and keep moving forward.”