Promising Stem Cell Research For Alzheimer's And Hearing Loss

“Alzheimer’s is a very complex disease, so any potential treatment has to be able to target multiple biological pathways,” says the lead author of the study, Dr. Stephanie Cherqui, a professor at UC San Diego School of Medicine. “Our work shows that hematopoietic stem and progenitor cell transplantation has the potential to prevent complications from Alzheimer’s and could be a promising therapeutic avenue for this disease.”

This study describes how transplanting hematopoietic stem and progenitor stem cells was effective at “rescuing” multiple signs and symptoms of Alzheimer’s in mouse models of the disease that displayed preserved memory, cognition, reduced neuroinflammation, and significantly less amyloid build-up compared to the untreated controls due to the enhanced microglia health which in turn protected against multiple levels of the disease pathology. 

“Alzheimer’s disease poses a major emotional and economic burden on our society, yet there is no effective treatment available,” says Cherqui. “We are excited to see such promising preclinical results from hematopoietic stem cell therapy and look forward to developing a new therapeutic approach for this devastating disease.”

In other stem cell research, two new papers suggest that modern medicine may one day be able to develop regenerative hearing therapies that may reverse deafness. The USC stem cell scientists describe why hearing is so delicate and how to possibly create new treatments using stem cells. 

“In the non-sensory supporting cells of the inner ear, key genes required for conversion to sensory cells are shut off through a process known as ‘epigenetic silencing.’ By studying how the genes are shut off, we begin to understand how we might turn them back on to regenerate hearing,” said John Duc Nguyen, the first author of one of the papers. Nguyen now works at the biotech company Genentech.

In the second paper, the researchers explored when and how the progenitor cells of the inner ear gain the ability to form sensory hearing cells, describing two specific genes that might be useful for regenerating hearing in adults. 

“We focused on the genes Sox4 and Sox11 because we found that they are necessary for forming sensory hearing cells during development,” said the paper’s first author Emily Xizi Wang, who also conducted her research as a Ph.D. student in the Segil Lab, and works at the biotech company Atara Biotherapeutics.

Gage Crump, a co-author on both papers and the interim chair of USC’s Department of Stem Cell Biology and Regenerative Medicine at the Keck School of Medicine of USC added: “These two papers are not only great science, but also a clear example of Neil Segil’s enduring legacy as an exceptional mentor to the next generation of stem cell researchers.”

“We’re excited to continue exploring the mechanisms by which cells in the inner ear gain the ability to differentiate as sensory cells during development and how these can be used to promote the recovery of sensory hearing cells in the mature inner ear,” concludes the paper’s corresponding author Ksenia Gnedeva, an assistant professor in the USC Tina and Rick Caruso Department of Otolaryngology–Head and Neck Surgery, and the Department of Stem Cell Biology and Regenerative Medicine.