The fifth annual Dancing With Dominic fundraiser, which benefits the research to find a cure for Hunter Syndrome, was hosted on Saturday, April 1, 2017 at Hughes Elementary School by the Henriquez family, whose 7-year-old son Dominic was diagnosed with the disease in October of 2011, when he was 22 months old.
The event included a dance in the gymnasium, performances by Prosper ISD’s dance teams, face painting by the art students, a catered dinner, a silent auction, a raffle, kids activities and crafts, a photo booth and other activities for all the families.
Dominic’s mother, Jeanette Espinola, said she is incredibly thankful for the amount of community support she’s seen during the planning process, and that the event was made possible all because of the help and support of the community of Prosper. She added that about 15 or 16 families from different parts of the country who have been affected by the disease attended the event.
“We had a family gathering in conjunction with the event,” she said. “Because there’s so few of us, we’re a very close community.”
Hunter Syndrome, or Mucopolysaccharidosis Type II (MPS II), is a rare genetic disorder affecting 1 in 150,000 males that slowly destroys the body’s cells due to a missing enzyme, which results in the accumulation of cellular waste throughout the body. It is a progressive and life-limiting disease that mainly affects young boys, and the prognosis is that the children won’t live past their teenage years. Espinola said it was a devastating diagnosis for her family.
“All of a sudden you lose basically all your dreams that you had for your child,” Espinola said. “You were going to see him grow up, see him become an adult. But he may not make it past his teenage years.”
She said the way she dealt with it was to do what she loves to do and plan to host a big fundraiser party along with her husband, who is a DJ, and with the help of the community in Virginia, where they lived before they moved to Prosper. Dominic loves to dance, so that party in 2012 became the first Dancing With Dominic event.
“It was our way of contributing and helping find a cure, and bringing awareness,” Espinola said. “I think that’s the other huge piece of it, is bringing awareness that there are these disorders, there are these kids, and that there is this potential right now to really help them, and the research is pretty much there, we just need the funds right now.”
Dominic’s parents, Jeanette Espinola and Freddy Henriquez, founded the Hunter Syndrome Foundation in 2013, after having hosted already two Dancing With Dominic events, for the specific purpose of funding potential treatments and research and ultimately finding a cure for the disease.
There is one approved treatment for Hunter Syndrome. It consists of an infusion of the man-made version of the enzyme Dominic is missing, which is administered through a four-hour weekly IV treatment that prevents the disease from progressing fast. The medicine he gets, called Elaprase, costs about $12,000 per week, amounting to around half a million dollars per year, and is the second most expensive medicine in the country.
“He’s been getting that for five years now since he was diagnosed,” Espinola said. “But the issue with that is that it doesn’t cross into his brain. So he could still lose his cognitive skills, he could still begin regressing.”
It’s not a complete treatment, so for the past two years he’s been in a clinical trial in Chicago where he’s getting the enzyme to his brain. It helps in slowing down the progression of the disease in his brain.
Researchers have been searching for a permanent cure, so gene therapy is the next step they are working toward. The gene therapy research for Hunter Syndrome is led by two doctors, Douglas McCarty, Ph.D., and Haiyan Fu, Ph.D. at Nationwide Children’s Hospital in Columbus, Ohio. All of the Hunter Syndrome Foundation funds have benefited their work to find a cure. Dr. McCarty said the gene therapy for MPS II is the result of more than a decade of collaborative research efforts with support from MPS II patient family foundations.
“This gene therapy approach targets the root cause of MPS II by delivering the correct gene using a vector that can cross the blood-brain-barrier,” McCarty said. “Our preclinical data have shown great promise with lifelong benefits. We believe that we are well positioned to move forward towards a phase 1/2 clinical trial in patients with MPS II.”
The vector for the gene therapy will cost $1.4 million to produce, and it will cost another million dollars to begin the clinical trials. The family donates the money raised from the yearly Dancing with Dominic event to the Hunter Syndrome Foundation, and through it, 100 percent of the funding goes toward the doctors’ research at Nationwide Children’s Hospital so they can find a cure.
“I’m not doing this by myself, there are families throughout the country who are also raising funds,” Espinola said. “So all of the families’ efforts put together throughout the country have raised over $500,000 so far to help the doctors in their research.”
Espinola said she hopes the family-led efforts are able to fully fund the clinical trials for gene therapy and for some normalcy for her son, Dominic, in the future.
“I hope that Dominic continues to do well and better treatments are found,” she said. “Maybe one day he can be an adult and lead somewhat of an independent life.”