People tend to throw the word “miracle” around carelessly. Surely, they mean no harm when they abuse the term, but for families like the O’Neill’s, words like “miracle” mean a lot more than you’d expect.
Cara and Glenn O’Neill have quite literally done as much as a parent could physically do in order to provide for their daughter Eliza. Now, after years of fundraising, 726 days of self-imposed isolation and non-stop determination to save 6-year-old Eliza’s life, she has finally become the first patient in the world to receive an experimental gene therapy for her deadly disease.
You see, Eliza suffers from a rare condition known as Sanfilippo syndrome. The radical disorder affects the brain and steadily deprives kids of language and understanding. Many refer to it as “childhood Alzheimer’s” due to similar symptoms between those afflicted with each respective syndrome.
In most cases, victims of the disorder eventually become immobile. Tragically, most don’t live beyond their teenage years.
The corrosive syndrome has different types, each with its own unique characteristics. Type A, for example, is the most severe. Eliza suffers from Type A.
(Source: O’Neill Family)
Eliza was initially diagnosed back in 2013, to much dismay. The good news was that at that time, researchers had developed a gene therapy that appeared to work in mice. The bad news was that this treatment hadn’t ever been tested on humans, and the trials to do so required massive funding.
Luckily, Eliza family was able to raise more than $1.8 million for clinical trials in children through the “Saving Eliza” campaign!
The O’Neill’s were never guaranteed that their daughter would be able to take part in the aforementioned clinical trials. To give her the best chance possible, the family went into seclusion in 2014 to prevent Eliza from building up immunity to the virus that would deliver the gene therapy. If that were to happen–in a worst case scenario–Eliza would have been excluded from the clinical trial, claims her dad. Their self-imposed isolation ended recently after almost two whole years.
In truly distressing fashion, Eliza’s hopes and chances for landing a spot on the clinical trials were declining. In an interview with TODAY, Glenn O’Neill recalled the anxiety and stress felt by the whole family, specifically from young Eliza:
“She went from saying sentences and singing songs, to a word or two of that sentence or song, to not saying those things,” Glenn said. “Even then, it’s a syllable of a word or having trouble putting two syllables together… it’s just absolutely devastating.”
(Source: O’Neill family)
Amazingly enough, a true miracle occurred. Eliza was granted the right to be the very first human patient to receive the therapy. As you can imagine, her being the first ever patient means that the success rate or effectiveness of the procedure are unknown.
Learn more about Eliza, her amazing story, and the groundbreaking procedure she underwent! Visit page two for more!
“The experimental therapy Eliza underwent involved a harmless virus that acts like a delivery truck,” Cara, Eliza’s mother, told TODAY. “It carries a normal copy of the gene that makes the enzyme into the girl’s cells The hope is the girl’s body will translate that gene and fix the problem.”
“Gene therapy is a promising treatment option for some diseases, but it’s still experimental and risky,” the National Institutes of Health claims. “It’s only tested for incurable diseases and studies continue to see if it is safe and effective.”
The procedure, which took roughly 30-minutes, was administered at Nationwide Children’s Hospital in Columbus, Ohio on May 10, 2016. Since then, little Eliza has been showing promising improvement.
(Source: O’Neill family)
“There’s been improved interaction and engagement with her,” Cara, 41, said. “It used to be more she was passing by us, looking through us. But now, she’s engaged and seeking really good eye contact and trying to talk.”
“We’re seeing this renewed light in her eyes. This clarity, focus — whatever is happening, it seems her body is able to relax more,” Glenn added.
Though the condition is rare, there are still hundreds of families that are affected by it. Eliza may have been the first to receive the treatment, but many others wish to follow in her footsteps. “There are hundreds of families waiting for a spot in a clinical trial, but few opportunities available, Glenn said. He wants every parent to feel the hope he feels,” claims Glenn O’Neill.
In an effort to raise awareness and support for those who also suffer from Sanfilippo, the O’Neill’s have released a viral video, which you can watch at the bottom of this page.
“Eliza’s future is uncertain right now and that’s a fabulous thing for a Sanfilippo parent because prior to treatment, Eliza’s future was definite: suffering, degeneration, seizures, feeding tubes, wheelchairs and death,” claims her father. “Now her future is uncertain and we think that’s a great thing because of the potential.”
[H/T] TODAY
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