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 the pre-clinical data indicate our GAN gene delivery to the central nervous system may stop progression of this horrific disease and is paving the way for closely related neurological disorders.
The first patient was injected with the GAN Gene Delivery in May of 2015 and so far 11 patients have been injected in the clinical trial as of October 2018! Dr. Steven Gray Director of the UTSW Viral Vector Facility said it best… “None of this work would have occurred without the entire Hannah’s Hope community… While we have come a long way, we have a long way to go.”
This first trial is to test the safety and efficacy of GAN gene delivery to the central nervous system. Newly diagnosed families around the world continue to reach out to us. The goal of the first GAN 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 tool 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.