Unmet Need
Alzheimer’s disease (AD) is a neurodegenerative disease that affects over 5 million people in North America. AD leads to nerve cell death and tissue loss in the brain, shrinking the brain and affecting many of its functions. The global AD population is steadily growing by 3-5% annually, with no cure or effective treatment (BCC Research). Current pharmacological treatments include cholinesterase inhibitors (Cis) and N-methyl-D-aspartate (NMDA) antagonists. While these therapies both work to mitigate symptoms, there are no marketed treatments to reverse or prevent the progression of the disease (Yiannopoulou et al.). With the immense and rapidly growing size of the affected AD population, there is a significant need for more effective treatments that work to change the course of the disease progression.
Technology Overview
Researchers from Johns Hopkins and University of North Texas Health Science Center have developed a sigma-1 receptor (S1R) ligand that has potential as a therapeutic for the treatment of age-associated cognitive disorders in patients with neurodegenerative disorders, including AD and other forms of dementia. During research studies, the compound was able to reverse certain cognitive deficits in mouse models in a BDNF-dependent manner. In addition to AD, the compound may also act as a therapeutic agent for other neurological disorders including Parkinson’s disease, amyotrophic lateral sclerosis (ALS), and Huntington’s Disease. This compound’s ability to reverse cognitive deficits indicates its potential as a novel therapeutic for the treatment of AD and other cognitive diseases.
Stage of Development
The researchers have completed in vitro and in vivo testing, including evaluation of metabolic stability, pharmacokinetics, and in vivo cognitive efficacy. They are currently testing the product in a genetic model of Alzheimer’s Disease.
Publication
Run-Duo Gao, Michelle Taylor, Tamara McInnis, Zhenglan Chen, Sadakatali S. Gori, Heather M. LaPorte, Maxime A. Siegler, Janet L. Neisewander, Robert H. Mach, Meharvan Singh, Barbara S. Slusher, Rana Rais, Robert R. Luedtke, and Takashi Tsukamoto. ACS Chemical Neuroscience 2023 14 (5), 947-957; DOI: 10.1021/acschemneuro.2c00791
