NASHVILLE, TN – The grass-roots organization Project Alive raised close to $300,000 with its #15byMay15 campaign and is now just $1.1 million away from fully funding a gene therapy clinical trial that could start in 2018 at Nationwide Children’s Hospital in Columbus, Ohio as early as July.
“We are so encouraged by the outpouring of support throughout the #15byMay15 campaign,” said Allison Muedder, a member of the Project Alive board and mother of Finn, who has Hunter Syndrome, MPS II. The non-profit foundation Project Alive formed to find and fund a cure for Hunter Syndrome, the rare and fatal disease afflicting approximately 500 boys in the US and 2000 worldwide.
Prior to April, Project Alive raised $1.1 million to fund the development and production of a gene therapy drug. The #15byMay15 campaign was an effort to raise the remaining $1.5 million needed to test the drug in a clinical trial. 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.
Project Alive funded the production of drug with Nationwide Children’s Hospital for a Phase I/II clinical trial in patients affected by the disease. The drug, which is the result of two decades of collaborative efforts with researchers in Ohio and at University of North Carolina at Chapel Hill, will be ready in July.
“The idea of the #15byMay15 campaign was if 100,000 people would donate $15 each the remaining funds for the trial would be raised,” Muedder said, adding that although Project Alive has not yet reached its goal, the campaign created the momentum needed to continue the push. The effort continues now with #15foracure.
“It has really empowered people to make the $15 ask of friends, co-workers, and neighbors. Until we reach 100,000 people or the ultimate goal of $1.5 million we’re not stopping,” she said.
“So now we’ll embark on #15foracure,” Muedder explained regarding the continued efforts. “The ask is simple, just $15, but the impact will be powerful if we can spread the word far enough!”
The #15byMay15 brought together people from coast to coast making donations, sharing Project Alive posts and messages, conducting fundraisers and asking friends to do the same. It even caught the attention of the students at Florida Gulf Coast University who are now working on their own campaign to support Project Alive’s effort. Close to 6,000 people made donations to the campaign over the past month.
Affecting approximately 2,000 people worldwide, Hunter Syndrome is a rare, genetic condition resulting in the lack of the enzyme iduronate-2 sulfatase. Without that enzyme, cellular waste builds up throughout the body and causes progressive loss of physical, and in most cases, mental function. The clinical trial targets the most common, severe form of Hunter Syndrome, causing progressive neurological decline and a lifespan in the early teens. In 2017, the first gene editing clinical trial ever performed, widely reported around the world, was in Hunter Syndrome, but targeted the less common, attenuated form of the disease.
Project Alive families have worked tirelessly to not only raise funds, but also raise awareness of the disease through interviews, including national appearances on “The Doctors” and “Inside the NBA,” speaking at conferences, and meeting with drug companies and doctors. The organization developed a documentary series about Hunter Syndrome which introduces viewers to families fighting the disease.
Project Alive families are available for interviews this week. To set up a time to speak to a family, please contact Barbara Esteves-Moore at 615-631-4383 or at firstname.lastname@example.org.