StemCell Therapy

By Alexandra Marvar | July 9th, 2020

For nearly a century, scientists have been studying Alzheimers a disease that kills hundreds of thousands of people per year but that still has no cure. Currently drugmakers and scientists test preventative Alzheimers drug treatments by identifying participants who are at higher risk of developing the disease, and then observing whether the treatment in question prevents the diseases onset. One in ten people develop Alzheimers after age 65, and one in three after age 85. Thus, this observation may take years or even decades which is part of why progress has moved at a snails pace.

An added challenge is that trial participants must have symptoms of Alzheimers to be eligible but the sheer appearance of symptoms indicates that many brain cells have already died, so by the time symptoms have appeared and candidates join trials, it is generally too late for any treatment to have a significant effect on their symptoms.

In a breakthrough, a research team team at Queen Mary University of London has developed a new system to efficiently screen potential Alzheimers treatments that may be able to greatly expedite the path to a cure.

For this study, published Thursday evening Eastern Daylight Time in the Nature group journal Molecular Psychiatry, the research group consisted of a cohort of people living with Down syndrome, who have as much as a 70 percent higher likelihood of developing Alzheimers during their lifetime. Those with the syndrome carry an extra chromosome 21, which contains a gene that increases Alzheimers risk.Researchers collected hair cells from the participants and reprogrammed the hair cells to become stem cells. Then, in a petri dish environment, they grew those stem cells into brain cells.

In these brain-like cells, the researchers observed a rapidly developing pathology that resembled Alzheimers, down to the hallmark trio of Alzheimers indicators: amyloid plaque-like lesions, progressive neuron death and accumulations of tau protein tangled up inside neurons.

With access to this test environment, the team then experimented with two drugs known to inhibit beta-amyloid production, applying them to the new brain cells. In six weeks, they found the drugs successfully prevented the onset of Alzheimers-pathology.

Although the two drugs the Queen Mary University of London researchers experimented with have failed clinical trials for other reasons and therefore arent suitable treatments for Alzheimers in the end, they were able to demonstrate proof of concept: This system of obtaining and developing cells and creating Alzheimers-prone, brain-like cells as a test environment could be used as an Alzheimers drug testing platform for other preventative drugs in the future.

The hope is that, by creating a lab-controlled cellular environment that replicates the human brain as it develops Alzheimers, the time it takes to test potential treatments could be diminished greatly, and the path to a cure could be significantly shortened. The researchers saw results within six weeks. If that expedited timeline to determine the effectiveness of a drug saves years of research, it could save millions of lives not to mention the suffering on the part of people living with Alzheimers and their loved ones.

This work represents a remarkable achievement, as this is the first cell-based system that has the full trio of Alzheimers pathologies, without any artificial gene over-expression, said Queen Mary University of London Professor Dean Nizetic, lead author on the study. This system opens up the prospect for screening for new drugs aimed at delaying or even preventing Alzheimers before neuronal death starts.

Although its still early days, the system raises a theoretical possibility for further development as a tool to predict who might develop Alzheimers. The same stem cell process could be used on anyones hair follicles, the resulting brain cells of which may or may not then develop Alzheimers-pathology in the dish. The idea would be to catch the people at higher risk of early disease in a cell-based system, before it starts in a persons brain and allow for the possibilities of individualized preventive interventions.

However, he said, they are still a long way from reaching this goal, though study co-author Professor John Hardy added that the potential development of a new, human model of the disease would be a great step forward.

See the article here:
Scientists Have Discovered a Way to Speed Up Alzheimer's Drug Testing - Being Patient

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