ABOVE: Pictured here are, from left, Ale Marin, Bri Maierle, Florida Gulf Coast University faculty member Courtney Dwyer Satkowski, Nikki Trimarche, and Tori Guasco, who helped raise money on Giving Tuesday for Project Alive. // SUBMITTED
Generous donors and a match from Facebook and PayPal gave $144,078 to Project Alive on Giving Tuesday, Nov. 27, bringing the non-profit organization within $300,000 of its goal to fund a gene therapy clinical trial that could help save the lives of teenage boys.
Project Alive, a grass-roots organization formed by parents to find a cure for Hunter Syndrome, raised more than $123,000 in six different currencies on Giving Tuesday before the match was added. According to a Facebook spokesperson, the matching challenge was fulfilled quickly on the morning of Giving Tuesday after $7 million in matching funds, available starting at 8 a.m. ET, were gone in minutes.
“We are so grateful for the families and local organizations who donated, as well as for Facebook and PayPal who encouraged participation in Giving Tuesday by offering this match,” Project Alive President Melissa Hogan of Thompson’s Station said. “Specifically,
I want to applaud two local Florida organizations, Hunting for a Cure, led by Courtney Dwyer Satkoski to rally students at Florida Gulf Coast University, as well as the MPS SuperHero Foundation, founded by Hunter Syndrome parents Avram Joseph and Monica Anaya. Our community coming together is what is helping us reach the goal of curing Hunter Syndrome.”
While donors in Florida have strongly supported Project Alive, donors on Giving Tuesday and throughout the year come from across the United States and even circle the globe.
Hunter Syndrome is a rare, genetic condition resulting in the lack of a particular enzyme. Without that enzyme, cellular waste builds up throughout the body and causes progressive loss of physical, and in most cases, mental function. Life expectancy for boys with the most severe form of Hunter Syndrome is early teens. There are 500 boys in the U.S. and 2,000 worldwide with the disease.
Hogan’s son, Case, 11, is one of them.
Project Alive is working to fund a promising clinical gene therapy trial for Hunter Syndrome, clinically known as Mucopolysaccharidosis or MPS II. Project Alive parents from Florida to California have worked closely with researchers to develop an effective gene therapy product to treat individuals with MPS II. In 2013, Project Alive began working with Dr. Haiyan Fu, who worked on preclinical gene therapy research for a similar disease, Sanfilippo Syndrome (MPS III), to develop a gene therapy for Hunter Syndrome.
Project Alive has an agreement with Nationwide Children’s Hospital (NCH) in Columbus, Ohio, and has funded the production of gene vector for a Phase I/II clinical trial in patients.
Researchers have FDA approval for the investigational new drug (IND) application, which allows them to proceed with clinical trials as soon as they have the funding.
A total of $2.5 million was needed to fund the complete project from drug development to the clinical trial. Through grass-roots fundraisers such as a car show in Nolensville, social media, GoFundMe, grants, a 4-state bike ride and donations – the largest of which was a $250,000 donation from the Mark Cuban Foundation in the spring of 2018 and now this boost from Giving Tuesday – Project Alive has raised more than $2.2 million toward costs to fund the clinical trial. The remaining funds needed to complete the funding for the clinical trial to begin is now less than $300,000.