Lori and Matt Sames co-founded HHF following the diagnosis of their youngest daughter, Hannah, in March of 2008. At the time of Hannah’s diagnosis, Lori was only able to find one scientist in the world actively studying GAN. Now, the scientists working with Hannah’s Hope Fund have a GAN gene delivery Investigational New Drug (IND) and we are hopeful a Phase 1 trial will begin 2nd quarter of 2014. The goal of the gene delivery project is to rescue the nerves in the central nervous system by delivering a healthy copy of the GAN gene.
We are working on a drug therapy approach to rescue the nerves of the peripheral nervous system. Intermediate filament aggregates are a hallmark of GAN and many other neurological disorders like, Alexander Disease, Dementia with Lewy Bodies, Alzheimer’s Disease, ALS, Parkinson’s Disease, Neural Intermediate Filament Inclusion Disease, Spinal Muscular Atrophy, diabetic neuropathy and some forms of Charcot-Marie-Tooth Disease. Our hope is that the drug(s) we discover that treat GAN will also impact related diseases. Then, we will be able to excite the pharmaceutical industry and have industry drive the drug(s) through commercialization.
In 2013, Hannah’s Hope Fund launched a Global Registry for Inherited Neuropathy (GRIN) in collaboration with the Hereditary Neuropathy Foundation. The goal of the registry is to identify patients with various forms of inherited neuropathy, assist them in receiving a genetic diagnosis and enable them to engage in research for their disease. GRIN is a valuable too to engage industry, as we are able to tell a drug company how many patients have the disease for their drug indication, how many patients said they would be willing to provide tissue samples to drive translation and how many patients said they would be willing to consider volunteering for experimental trials. Unless industry knows they have a solid, engaged audience, they will not invest in drug development for rare diseases, which every form on neuropathy is, and the vast majority are considered ultra-rare (less than 600 US patients). Through GRIN, we hope to find more GAN patients who have been clinically diagnosed with Charcot-Marie-Tooth Disease type 2 (axonopathy).
Please consider making Hannah’s Hope Fund your charity of choice. Precious and innocent lives are depending on us all.