Why We’re Excited
Our group in the department of chemistry has made a discovery that may lead to a new approach to treat Alzheimer's disease. Over the years we have been interested in finding better ways to make complex molecules that occur in nature – especially those that exhibit useful biological activity. A few years ago we received funding from the National Institutes of Health (NIH) to make collections of molecules related to some of these natural products for biological screening to identify new compounds that might lead to treatments for a variety of diseases.
We discovered one group of novel compounds that may have the power to go after neurodegenerative disorders, such as Alzheimer's disease. This is an exciting finding because no new drug for this disease has been approved since 2003; the only drugs available today merely transiently treat the symptoms of the disease. Bottom line: We are in desperate need of new and more effective therapeutic approaches that lead to cures of this debilitating disease.
Signs That We’re On To Something
Could our molecules that target a receptor (PGRMC1) not previously associated with Alzheimer’s disease offer a promising new therapeutic approach? We aimed to find out. First, we worked with researchers in the department of neuroscience here at UT to screen our compounds in a type of worm called C. elegans. Jim Sahn, on my team, and Luisa Scott in the Pierce-Shimomura lab in neuroscience conducted experiments and found some compounds did in fact reduce neurodegeneration in a novel worm model of Alzheimer's disease. This was an amazing discovery, but it was just the beginning.
Although these worms are useful for screening, we wanted to test our compound in an industry-accepted animal model for the disease. Accordingly, we used a mouse model for Alzheimer's disease and were even more thrilled when the mice treated with the compound had significantly better cognition than the untreated Alzheimer’s disease animals.
Where You Come In
We have something that works in both worms and mice. Will it work some day for people with this devastating disease? It will take a lot more work to find out, but if we’re right about this, we could be on the brink of a completely new way to treat Alzheimer’s disease with something that big pharmaceutical companies haven’t yet tried. If we’re successful, there’s a chance that what we do will lead to the development of more effective drugs that not only treat the symptoms of Alzheimer’s, but that also slow or even stop its progression.
Such a discovery represents the holy grail of Alzheimer’s research and would be game-changing.
Be A Part Of This Moment
We are now at a crossroad. We need additional funding so we can perform the necessary proof-of-principle studies so companies will invest in a valid new approach to tackling Alzheimer’s. Additional results will help us also make a compelling case to granting agencies like the NIH and various foundations that support research in this area.
We are asking for your help and support so that we can continue to develop this promising research into a novel therapy to help those suffering from Alzheimer’s disease. With your help, we believe we can make a difference.
This work started at UT – You can now help us continue research that might change the world by
Raising Your Horns Against Alzheimer's Disease.
Department of Chemistry , The University of Texas at Austin
Professor and M. June and J. Virgil Waggoner Regents Chair in Chemistry