Some diseases are so rare that it feels like researchers have given up on finding a cure. So was the case with Mucopolysaccharidos 1 (MPS I) and one such young boy who suffers from it.
Ryan Dant was diagnosed with MPS I when he was only three and to the horror of his folks, Jeanne and Mark, there is no cure. Even worse, his life expectancy was cut to 10 years and they learned that throughout those years, he would suffer tremendously. The disease is so rare that there is no profitability in a cure, even if it was found. As a result, researchers had abandoned it and forced people like the Dant family to battle some the hard truths.
They refused to give up.
If there was a way to save Ryan’s life, they had to find it. If nobody else was going to help, they had to put matters into their own hands. But they knew whatever research or treatment was required, would cost a lot of money. That’s when the fundraising began.
It started off small with a bake sale, selling cookies and pies and it soon escalated to high end golf tournaments, fishing tournaments and raffles. What began of as a total of $342 from desert grew to $200,000 from a sole donor. That was how The Ryan Foundation was born.
Their second phase on the journey was research. Not much was known from the disease which is why they needed to uncover any shred of new findings that could potentially lead to treatment. Their tireless journey led them to the UCLA laboratory of Dr. Emil Kakkis who was onto something. But alas, he needed more funding. Nevertheless, he was onto something.
A super team was formed among the Dant family, Dr. Kakkis and his small band of researchers. Gradually, they made momentum toward finding treatment.
In 1997, Dr. Kakkis had finally made enough noise to garner the attention of BioMarin Pharmaceutical which led to their budding partnership. This allowed Kakkis to complete his research and kickstart an exciting new drug therapy into clinical trials. Ryan was among the 10 courageous children to volunteer for these trials at the UCLA Medical Center in Torrance, California. They took weekly IV’s of this up and coming drug called Aldurazyme.
It was working.
Almost right away too. It started reversing the progression of MPS I symptoms for all of the volunteers including Ryan.
The FDA saw this achievement and ordered a second trial with 50 more children from all over the world. The Dants and other families battling MPS I went to the FDA Hearing in D.C. to approve the use of Aldurazyme, in which the panel voted unanimously to approve. The drug began improving children’s symptoms all over the world.
It’s important to note that Aldurazyme is not a permanent cure for MPS I. The intense damage to the nervous system is still something that needs to be addressed. The research and funding continues.
The Ryan Foundation continues to do good work in not just MPS I research but in other MPSs like Hunter Syndrome, Morquio Syndrome, and Maroteaux-Lamy Syndrome. It’s a labor of love with zero paid employees, whose proceeds go straight toward scientists looking to cure rare diseases.
This story is the perfect example of how we should never give up on our loved ones, even if we feel the world has given up on them and how if you make enough noise in a field, the right people will hear you and lend a helping hand. To donate to The Ryan Foundation, click here.
