Prions diseases occur when a protein regularly found in the body is misfolded, causing illness. This misfolding leads to brain damage alongside other symptoms, which take years to develop. Once a person is symptomatic, the disease rapidly progresses in the body, leading to death. Researchers at the Broad Institute of MIT and Harvard and researchers from the Whitehead Institute for Biomedical Research have developed promising new therapies for fatal prion diseases. The collaboration between the two programs led to the development of a new set of molecular tools known as CHARMs (Coupled Histone tail for Autoinhibition Release of Methyltransferase), which silences the gene that causes the production of the disease-causing proteins, alongside stopping the production of more of these genes, CHARMs can also stop already generated prion protein genes. One of the researchers, Sonia Vallabh, pushes the urgency of this project as she suffers from an increased likelihood of developing fatal familial insomnia, another form of prion disease. While initially starting in the law industry, Vallabh and her husband shifted their careers toward biomedical research after discovering a lack of treatments for these conditions. Their work doesn't only focus on treating prion disease, but they continue to research other disorders that cause the loss of neurons in the brain caused by toxic protein accumulation.
The CHARMs technology uses epigenetic editing, which uses an epigenome, a make-up of chemical compounds and proteins that can attach to DNA and allow actions such as turning genes on or off, which specifically targets the prion protein gene, quickly silencing them and preventing any more production of misfolded proteins which may have led to a prions diseases. Tests conducted on mice confirm that these tools can remove up to 80% of prion proteins found in the brain, a significant increase from the 21% removal rate used with past techniques to relieve symptoms. The researchers saw challenges when creating CHARMs, especially in trying to ensure that the components used were non-toxic, alongside being able to target the prion gene without any side effects. Using the machinery built into the cells, the researchers could silence the gene, minimizing the potential toxicity. While the development of CHARMs is still too unsafe for humans, the astonishing rate of development shows hope for those who suffer from prion diseases and other diseases caused by protein misfolding.
While new tools to treat prion diseases are being developed at astonishing rates, it will still be a while before CHARMs can be used to treat humans with prion disease. When the time comes when CHARMs are safe to use, they will revolutionize the medical field, helping prevent the decline in mental capacity due to misfolding. I find this article very reliving as while prion disease doesn't run in my family, Alzheimer's does, and both are similar to where misfolding can lead to a decline in cognitive functions. While not the same, if the development goes well for CHARMs to help treat prion disease, it could be used as a stepping stone to preventing Alzheimer's as well.
https://news.mit.edu/2024/charmed-collaboration-creates-therapy-candidate-fatal-prion-diseases-0627
https://www.usatoday.com/story/news/health/2024/06/27/stop-dementia-delay-brain-disease-medical-research/74190613007/
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