I’m aware of two trials and one of them is in patients with Alzheimer’s disease and this is in also mild cognitive impairment, so this is a preamble to Alzheimer’s disease, and this is an approach that Dr. Mark Tuszynski and myself have been working at the University of California system for almost 15 years or so. And this involves delivery of adeno-associated viral vector, which is a gene therapy approach, into part of the brain that is called the entorhinal cortex, and this is part of the brain that degenerates in patients with this type of dementia...
I’m aware of two trials and one of them is in patients with Alzheimer’s disease and this is in also mild cognitive impairment, so this is a preamble to Alzheimer’s disease, and this is an approach that Dr. Mark Tuszynski and myself have been working at the University of California system for almost 15 years or so. And this involves delivery of adeno-associated viral vector, which is a gene therapy approach, into part of the brain that is called the entorhinal cortex, and this is part of the brain that degenerates in patients with this type of dementia. This is meant to deliver a trophic factor that will increase the innervation in this part of the brain, specifically in the hippocampus, and then all the projections from the hippocampus into the cortex. So our hope is that by the delivery locally to that part of the brain using MR-guided gene delivery technology, we’ll be able to jump-start again the activity within the hippocampus. Specifically, we hope for increasing the stem cells that reside in the hippocampus to become more and more functional. The beauty of the gene therapy approach is using the technology that we developed is that we can, with the use of magnetic resonance imaging, we can place the gene therapy specifically only in the part of the brain that we’re targeting without any spillover to other parts of the brain that may cause some adverse events. So that is exactly what’s being done in these patients. We’re treating 12 of those patients right now. We have, I believe as of today, two more patients to go to complete this trial. It is done at the University of California, San Diego, and also at The Ohio State University. So it’s an academic trial. So that is a great promise, we hope, to the community when it comes to Alzheimer’s disease. There’s another trial that is ongoing in Europe and also in the States, and this is meant to target patients in the genetic form of the frontotemporal dementia. Progranulin 1 is where the mutation takes place. So these patients are being identified by genotyping. And the innovation here has to do with some of the work that we’ve done quite some time ago, showing that if we deliver the viral vectors into the part of the brain called the thalamus, it undergoes axonal transport, a very specific axonal transport to the cortex, and depending on which part of the thalamus is being targeted, one can alter this axonal projection to specific cortical regions. So this is exactly how this trial is being conducted by the delivery of the AAV that carries a gene for the progranulin 1 into the thalamus, and that allows the axonal transport to spread the therapeutic gene throughout the cortex. Obviously, the temporal cortex is the main target here, but as you can imagine, we cannot locally deliver to multiple cortical regions because those involve just too many needle passes, and in this case, we just do one and then let the projection do the rest. So a very exciting trial, and we’ll be watching very carefully and with great interest what kind of outcomes are in patients. It’s a fast-progressing disease, of course, so I hope that safety and some of the early efficacy will be identified soon.
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