Almost two years ago, Justin noticed that he was having trouble walking straight and sometimes he would just fall down. So, his parent took him see specialists who have confirmed through DNA testing that he has a very rare genetic mutation called Giant Axonal Neuropathy (GAN). Justin is scheduled to be a part of the GAN Natural History Study at the National Institute of Health and will hopefully be chosen to participate in the clinical trial although he will not be in Phase 1 of the trial. Our mission, in partnership with Hannah’s Hope Fund for Giant Axonal Neuropathy, Inc. is to help raise funds to support the development of a treatment and cure for GAN disease, and to be the resource for doctors, scientists and families world-wide.
1st patient has been injected with the GAN treatment!
We are so very happy to finally be allowed to announce that the first GAN patient was injected at the National Institutes of Health (NIH) on May 27, 2015. Patient # 1, Chrissy, is doing well thus far, but we will know a lot more in the next 8 weeks.
University of North Carolina (UNC) School of Medicine researchers have developed an innovative, experimental gene transfer-based treatment for children with giant axonal neuropathy (GAN).
Researchers led by Steven J. Gray, PhD, assistant professor in the Department of Ophthalmology and a researcher in UNC’s Gene Therapy Center and Carolina Institute for Developmental Disabilities, developed the experimental treatment in studies conducted at UNC. Gray’s work in this area was funded almost entirely by Hannah’s Hope Fund, a charity founded by the parents of Hannah Sames, an 11-year-old girl with giant axonal neuropathy (GAN), to support the development of a treatment and cure. This extremely rare genetic disorder causes children to gradually lose the ability to balance themselves, move their muscles and to feel certain sensations. Most children born with GAN do not survive beyond their early 20s because of progressive impairment of their ability to breathe.
The treatment approach developed at UNC uses a genetically modified virus called AAV to deliver a missing gene, the gigaxonin gene (scAAV9/JeT-GAN), into the cerebrospinal fluid of children with GAN. The therapeutic viral vector to be used in each of these injections is prepared at the UNC Vector Core Human Applications Laboratory.
A clinical trial of this approach is now underway at the National Institute of Neurological Disorders and Stroke (NINDS) of the National Institutes of Health (NIH) in Bethesda, Maryland. The first patient was enrolled in May. This is the first gene delivery approach directly into the spinal fluid in order to treat an inherited neurological disorder, and is expected to pave the way to developing treatments for many other related diseases. Read More…
Justin’s Triumph Over GAN Sponsors