Tempering hope with a dose of reality

A responder in the KDA Forum mentioned a BBC News article that showed promise on the research front. The article titled, “Range of brain diseases could be treated by single drug,” was interesting because it mentioned Huntington’s disease and that is often linked to Kennedy’s Disease in regards to the defective DNA. The report mentioned that the study was preliminary, but everything starts out as preliminary and it could eventually lead to something.

“In a study, published in Nature, they prevented brain cells dying in mice with prion disease. It is hoped the same method for preventing brain cell death could apply in other diseases. The findings are at an early stage, but have been heralded as "fascinating".


Many neuro-degenerative diseases result in the build-up of proteins which are not put together correctly - known as misfolded proteins. This happens in Alzheimer's, Parkinson's and Huntington's as well as in prion diseases, such as the human form of mad cow disease.”


Other interesting comments include:

“The idea, which has not yet been tested, is that if preventing the shutdown protects the brain in prion disease - it might work in all diseases that have misfolded proteins.
Prof Mallucci added: "What it gives you is an appealing concept that one pathway and therefore one treatment could have benefits across a range of disorders. But the idea is in its early stages. We would really need to confirm this concept in other diseases."


“Professor of Molecular Neurobiology at King's College London, Roger Morris, said it was a "breakthrough in understanding what kills neurons". He added: "There are good reasons for believing this response, identified with prion disease, applies also to Alzheimer's and other neuro-degenerative diseases. "And because it is such a general response, we already have some drugs that inhibit this response."


The more I read, the more tempering words I saw: “infancy, early stages, need to confirm, and need to see the same results in other diseases.”

I immediately sent the link to Ed, our resident Biology professor, asking for his thoughts. Ed responded back with the following:

“I have not read the paper (yet), but it has always been hoped that misfolded proteins would be a common thread in the mechanism that causes the cell death.  However, it is VERY premature to assume it actually works on other diseases, so I would be very careful how to explain it.  The implementation of such a treatment is likely many years away.”


Still, the more we understand about what causes these health conditions the better chance there is to find a potential link to the diseases and possibly even a treatment. ‘One small step’ might lead to ‘a giant leap’ along the way.