Rare Disease Foundation Signs $1.4 Million Contract To Produce Gene Therapy Vector For Hunter Syndrome Clinical Trial
Vector Production and Active IND Meet Major Milestones in Foundation-Scientists Collaboration to Develop an Experimental Therapy for Young Boys with a Rare Dementia-Like Disease
"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.
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. To date, no gene therapy or gene editing has been performed in a patient with cognitive regression, which represents approximately two-thirds of Hunter patients.
"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."
"Last week, we also received FDA approval of our investigational new drug (IND) application, which will allow us to proceed with clinical trials for this gene therapy product in MPS II patients," added Dr. Kim McBride who will lead the trial at NCH. "We truly appreciate the continuous efforts and philanthropy of Project Alive and other patient foundations to sponsor this trial."
To support the clinical trial, Project Alive has been raising funds from public donations, grants and fundraisers across the United States. They have also released a documentary series about Hunter Syndrome that has received significant media attention including on national television shows such as "The Doctors."
Recently the foundation raised over $100,000 on Giving Tuesday to support the vector production for the clinical trial. Project Alive continues to seek major donors who want to have a lasting impact on this devastating disease, as well as on the healthcare system since current treatments for Hunter Syndrome run approximately $500,000 per patient per year.
"We are excited to move forward in our efforts to save these children's lives," notes Melissa Hogan, president of Project Alive, Hunter Syndrome expert, and parent to a ten-year-old with Hunter Syndrome. "But now we need the funding to catch up with the science and our mission."
To find out more about Hunter Syndrome or to support the mission of Project Alive, visit ProjectAlive.org.