A new study by researchers at MIT is grabbing attention for successfully testing out a genetic technique that could possibly help them find a treatment for Alzheimer’s.Looking for treatments to cure neurological diseases in humans using mouse models has conventionally not been a fruitful experience for the pharma industry. Moving from one disease model to another is complicated, especially with neurological diseases, as we deal with two very different systems. It is challenging to expect the same results from human clinical trials.
SIRT1 gene’s connection to longevity might help us slow the progression of Alzheimer’s
As reported by the NYTimes, the MIT researchers explored the SIRT1 gene, also called the “longevity gene”, known to delay aging in humans. The researchers took a breed of mice with a model of Alzheimer’s disease and engineered them in such a way that half the group had the SIRT1 gene while half did not. They observed that the mice with the SIRT1 gene retained both memory and the ability to learn for longer periods compared to the SIRT1 deficient ones.
SIRT1 is also known to delay aging in general. It is now becoming a focal point of research due to the possibility that we could uncover the genetic factors linked to Alzheimer’s disease. This is alongside all of the research already underway based on the statistical correlation between the presence of the Apo-E4 gene and the susceptibility to get Alzheimer’s. According to the report, the link between SIRT1 and Alzheimer’s has really only been proven on a petri-dish rather than on a clinical trial platform. This study might provide the first steps to such a study that scales up our understanding of the underlying mechanisms of the disease.
Crossing the Blood-Brain Barrier is the main challenge for SIRT1 activating drugs
SIRT1 works at the heart of the generation of amyloid plagues. In Alzheimer’s disease, amyloid plagues are formed when proteins in the brain break down into toxic amyloid peptides. It is this toxic substance that eventually causes the death of brain cells. In the mouse models tested, the presence of SIRT1 produces more sirtuin proteins that break the amyloid peptides down to a harmless form.
The researchers feel that the use of drugs that activate SIRT1 could reduce the production of amyloid peptides and slow down the progression of Alzheimer’s disease in humans. The main challenge then is to get these drugs across the blood-brain barrier. This doesn’t seem to be easy and they will need to leverage their partnership with GSK to explore options for drug delivery systems. With the current developments in treatment options and biomarker-style drug carriers, this technology could potentially find itself in human clinical trials.
