Recent research suggests that drugs capable of activating vitamin A in the body may offer new therapeutic options for conditions like motor neurone disease (MND), raising hopes for innovative treatments.
A team of scientists has discovered that these drugs, which specifically target receptors necessary for vitamin A activation, could potentially benefit diseases associated with brain deterioration. Laboratory simulations of disease conditions revealed that the “super-activation” of the vitamin A signaling system could provide protection against neuronal damage commonly seen in MND.
Leading the study, Professor Peter McCaffery from the University of Aberdeen explained, “We discovered that these drugs bind and activate the ‘retinoic acid receptor,’ a critical protein involved in the body’s activation of vitamin A. Our research paves the way for identifying new drug targets that may lead to future therapies.”
Currently, MND affects around 5,000 adults in the UK at any given time, with no available treatments extending beyond a few months. The disease progresses to cause the death of motor neurons crucial for muscle control, ultimately leading to severe muscle weakness and, in many cases, a fatal outcome within a few years.
Azita Kouchmeshky, a neuroscientist at the University of California, San Francisco, and one of the study’s authors, elaborated on the research methodology. “We tested these drugs through a series of experiments on neurons cultured in the lab. We introduced harmful chemicals that mimic the changes seen in diseases like MND or ALS (Amyotrophic lateral sclerosis). Typically, these chemicals result in neuronal death. However, applying drugs that bind to the retinoic acid receptor significantly reduced cell death.”
The effectiveness of these drugs was also validated in animal studies, suggesting potential efficacy in living organisms.
The findings are documented in a research paper published in Frontiers in Neuroscience. The drugs were designed and synthesized by Andy Whiting, chief executive of Nevrargenics Ltd and emeritus professor at Durham University. He noted, “There is a critical shortage of disease-modifying drugs for neurodegenerative conditions, particularly for MND. We are dedicated to changing that and providing hope for those affected by the disease.”
Funding for the research was provided by the Chief Scientist Office, the Sprint-MND/MS PhD program, and the Motor Neurone Disease Association. Dr. Nicholas Cole, head of research at the MND Association, expressed enthusiasm for the study’s potential impact: “We are thrilled to support this important early work, which we hope will contribute to discovering new therapies ready for clinical testing. Our ultimate goal is to unravel the complexities of MND and develop effective treatments through ongoing public support and collaboration.”
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