Over the past three years, parent-led efforts have collectively raised half a million dollars to support gene therapy research at Nationwide Childrens Hospital (Columbus, Ohio) to treat the ultra-rare disease Hunter Syndrome (also known as Mucopolysaccharidosis or MPS II).

The research is led by Dr. Douglas McCarty and Dr. Haiyan Fu of Nationwide, and funding is expected to bring the research closer to a human clinical trial in late 2017 or early 2018.

To raise funds, the parent-led foundations organize local fundraisers. In Prosper, the Henriquez family hosts an annual family event called Dancing with Dominic, in honor of their 7-year old son Dominic who suffers from Hunter Syndrome. Dominic is a 1st grader at Baker Elementary in Prosper ISD. Without a cure or emerging treatment, Dominic may not live past his teenage years.Dancing with Dominic 2017 will be held on April 1, at Hughes Elementary in McKinney, Texas. Dr. McCarty and Dr. Fu are among the hundreds that plan to attend the event this year.

Hunter Syndrome is a rare, genetic condition that affects approximately 2,000 patients worldwide, almost exclusively young boys. Patients are missing an enzyme, resulting in the accumulation of cellular waste throughout the body.

Babies develop normally for the first few years, and then begin to experience progressive symptoms like stiff joints, enlarged liver and spleen, behavioral problems, constant ear infections and runny nose, and heart valve complications. The average life span for someone with the most common, severe form of the disease is in the early teens.

There is no cure for the disease, although once diagnosed, patients can begin receiving a weekly 4-hour infusion of an enzyme replacement therapy. This medication, Elaprase, is one of the most expensive in the world, often $500,000 or more per patient, per year, and only stabilizes some of the physical symptoms of the disease. Because it does not cross the blood-brain-barrier, it does nothing to prevent the progressive brain damage that occurs in most children affected by the disease.

This gene therapy for MPS II is the result of more than a decade of collaborative research efforts with support from MPS II patient family foundations, Dr. McCarty said. This gene therapy approach targets the root cause of MPS II by delivering the correct gene using a vector that can cross the blood-brain-barrier. Our preclinical data have shown great promise with lifelong benefits. We believe that we are well positioned to move forward towards a Phase 1/2 clinical trial in patients with MPS II.

About the Hunter Syndrome

Foundation

The Hunter Syndrome Foundation is a 501(c)3 non-profit corporation with a mission to fund potential therapies that will ultimately find a cure for this disorder.

The Foundation is part of a coalition of parent-led organizations that are supporting medical research for hunter syndrome. The Foundation was established in Prosper, Texas by the Henriquez family. For more information, visit http://www.huntersyndromefoundation.org.

Read the original post:
Prosper nonprofit brings gene therapy treatment closer to reality - Star Local Media

Comments are closed.