It’s only been 16 months since Our Town shared the story of Lincoln Medeiros, the little boy from Scio who,
when he was only six months old, was diagnosed with the rare genetic disorder spastic paraplegia 50 (SPG50) – a progressive neurologic condition affecting speech, motor control and muscle tone – but a lot has happened since then.
The family welcomed another child, Lincoln turned four, he learned to run and climb, he started preschool. Then, more recently, his mobility – due to the degenerative nature of SPG50 – took a hit, he began wearing ankle-foot orthosis braces to help stabilize his legs and he started using a walker. But the event that stunned the family the most was the cancellation of the grant funding necessary for Melpida – a gene therapy treatment developed to halt the progression of SPG50 – to move into the next trial phase, the phase Lincoln had hoped
to join.
“The CIRM [California Institute for Regenerative Medicine] grant falling through was awful,” Jordan Medeiros said. “All of the families wrote letters to CIRM… to appeal their decision. One mom from Alaska even flew there to make a statement, to no avail.”
It was an event that threw both the families with children suffering from SPG50 and Terry Pirovolakis – the founder of Elpida, the company focused on getting Melpida approved for medical use – into a tailspin, forcing them all to pivot from scheduling the next medical trial to once more scramble for funding.
“We started this journey fundraising alongside two other families in the US trying to get their children treatment,” Jordan recalled. “There was some nice momentum in Alaska and Colorado, but we weren’t reaching our goal fast enough. Then, a family from Brazil joined in the efforts once their son was diagnosed, and they were able to raise most of the $2.8 million needed to buy the eight doses of the drug.”
Then, with the majority of the funding again in hand, the next step was to find a hospital willing to administer the therapy – a one-time dose of Melpida – for free, a task that initially looked like an easy one, with several facilities showing “strong interest.” But then the current administration began rolling out additional cuts to medical research funding and those options disappeared.
“[O]nce the political climate changed, grants and funding was cut drastically within the rare disease space,” Jordan confirmed. “Massive layoffs happened, and treating only eight children became even less of a priority for said hospitals. The FDA has also issued new fees along the way.”
Thankfully, Elpida eventually located one hospital willing to conduct the phase three clinical trial for a reduced rate and a reduced number of children – UT Southwestern in Dallas, Texas.
“The hospital in Dallas has set a price at $1.15 million to treat four children, and they can start as early as November if the funds can be raised by then,” Jordan said.
Which means that Jordan and the other families are once again scrambling, this time to raise money, not only for the cost of administering the gene therapy, but also transport for each child to Texas six times – once for the initial dose and five times for observation – an estimated total of $35,000 per family.
“Honestly, it has been an exhausting two years of fundraising,” Jordan said. “I find it consuming. It’s all I think about. There’s this medication, this hope, that exists and is out there – but Lincoln can’t have it due to funding. It eats at me every single day…”
Which is why she is asking the community once more for assistance.
“We are desperately asking our community to help,” Jordan confirmed. “As a stay-at-home mom of three under the age of four… it is challenging to plan, implement and host fundraisers. We are asking our local businesses if they would consider hosting a fundraiser or making a donation. We do partner with Jack’s Corner Foundation Network, and they are a 501c3 non-profit. As the year is winding down… please consider making a donation.”
