Project Alive president speaking at World Orphan Drug Congress in DC this week

NASHVILLE, Tenn. - April 23, 2018 - PRLog -- What if your child had a rare and fatal disease for which doctors thought they had developed a cure, but the cure sits in medical research limbo due to funding?

This is the real scenario facing the parents who have formed the non-profit foundation Project Alive. They are coalesced around the single goal of curing Hunter Syndrome, the rare and fatal disease afflicting their sons. This month Project Alive launches its #15byMay15 campaign to raise the remaining $1.5 million needed to test a drug developed by researchers who believe it will cure the disease.

Project Alive President Melissa Hogan, whose son Case has Hunter Syndrome (MPS II), will be in Washington, D.C. April 25-27, speaking at the World Orphan Drug Congress. Melissa, a healthcare attorney, is an expert on Hunter Syndrome and has consulted on research designs for the disease around the world.

The #15byMay15 campaign aims to move 100,000 people to each donate $15 in order to complete the funding of a clinical gene therapy trial at Nationwide Children's Hospital in Columbus, Ohio. This grass-roots foundation has already raised $1.1 million to produce the drug to be used in the clinical trial for MPS II patients, and to support an investigational new drug (IND) application.

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.

Hogan and other Project Alive parents from Florida to California have worked closely with researchers to get to this point. They have an agreement with Nationwide Children's Hospital (NCH) in Columbus, Ohio, to fund the production of gene vector for a Phase I/II clinical trial in patients affected by the disease and after beginning production, the drug will be ready in July 2018. The #15byMay15 campaign seeks the money to fund the next step as soon as the drug comes out of production.

"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.

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. In 2017, the appeared on "The Doctors" and "Inside the NBA" among other shows.

Project Alive has developed a documentary series (https://projectalive.org/stories/) about Hunter Syndrome which introduces viewers to families fighting the disease.