NASHVILLE, TN – Last month, nonprofit Project Alive began a campaign called #15byMay15 to raise the remaining $1.5 million needed to fund a clinical drug trial for the rare disease Hunter Syndrome / MPS II that could begin as early as this summer. Each night as the grass-roots effort grew, organizers began to thank donors on their Facebook page.
Since the campaign began in April, more than 3,000 donors have contributed over $134,000. Project Alive President Melissa Hogan explained, “Every night I’ve been updating a Facebook post with the total donors and amounts and names for the #15byMay15 campaign. Because it was getting so long, it made me wonder how long a Facebook post could be. That number is 63,206 characters and everything I could find said the longest recognized post once that number was increased in 2012 is one by Nokia with 62,896 characters.”
On May 10, 2018, only 5 days shy of MPS Awareness Day on May 15, Hogan said Project Alive’s #15byMay15 donor acknowledgement post reached that maximum 63,206 characters and had to even continue the list of donors in the comments, which have a maximum of 8,000 characters. It’s a good problem to have.
“We’re really just trying to spread the word about our #15byMay15 campaign, but more than that about Hunter Syndrome and how close we are to a possible cure,” Hogan said of her efforts to thank donors on Facebook.
Project Alive began the campaign as a push toward a deadline looming in July. That is when a gene therapy drug will be out of production and ready for a clinical trial at Nationwide Children’s Hospital in Columbus, Ohio. The non-profit organization formed by families of children who have Hunter Syndrome began collaborating with researchers over five years ago and have funded the program to the tune of over $1.2 million, including a contract to manufacture the gene vector to be used in the clinical trial. They have $1.5 million to go in order for the trial to begin later this year.
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 life
span in the early teens.
“This is a critical step towards our goal to develop an effective gene therapy product to treat individuals with MPS II,” said Dr. Kim McBride, the clinical principal investigator of the project. “This project reflects two decades of collaborative efforts of Dr. Haiyan Fu at Nationwide Children’s Hospital and Dr. Joseph Muenzer at University of North Carolina at Chapel Hill.”
In 2013, Project Alive began working with Dr. Fu to see if her previous preclinical gene therapy research for a similar disease, Sanfilippo Syndrome (MPS III), could be developed in Hunter Syndrome. That call began a symbiotic relationship between a researcher who knew the science and parents most familiar with the disease, which accelerated the then ongoing collaborative research project of Drs. Fu and Muenzer.
“With the generous support from Project Alive and many other family foundations we have made pivotal progress since 2014,” said Dr. Fu. “This has led to significant advancement in our MPS II gene therapy research towards 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.
“Now we need the funding to catch up with the science and our mission,” said Hogan, will be in Chicago May 15 speaking at the American Society of Gene and Cell Therapy 21st Annual Meeting. A healthcare attorney and an expert on Hunter Syndrome, Hogan has consulted on research designs for the disease around the world. To support the clinical trial, Project Alive has been raising funds from public donations, grants and fundraisers across the United States.
Hogan and her husband, Chris, a national best-selling author and TV personality, have worked tirelessly to not only raise funds, but also raise awareness of the disease through interviews, speaking at conferences, meeting with drug companies and doctors. Other families around the country are doing the same. The Muedders in Charlotte, N.C., have developed a documentary series about Hunter Syndrome which introduces viewers to families fighting the disease. The Estevez family in Miami has rallied the South Florida community. Many Broward County public schools and private schools along with local businesses have hosted fundraising events to help Project Alive.
The #15byMay15 campaign aims to move 100,000 people to each donate $15 to complete the funding for the clinical gene therapy trial.
Follow the Facebook page and its pinned thank-you post at https://www.facebook.com/projectalive/.
To set up a time to speak to Mrs. Hogan or other Project Alive families, please contact Barbara Esteves-Moore at 615-631-4383 or at firstname.lastname@example.org.