Before I get everyone’s hopes up high, just wanted to say that the research is very preliminary, and have not gone on to live human trials. A new research has shown significant results in potentially stopping and reversing the development of Multiple Sclerosis. We first saw this news on GEN.
Currently, the research is only being done on mice, and will probably take another decade to be used for “curing” Multiple Sclerosis assuming the direction is correct. Regardless, we remain hopeful and are inspired by the scientists from University of Florida in Gainsville.
Gene immunotherapy could potentially stop and even reverse the development of Multiple Sclerosis
Brad E. Hoffman, Ph.D and Associate Professor at the University of Florida College of Medicine mentioned that they were very impressed with the results as it was somewhat unexpected. Multiple Sclerosis is a neurological condition which attacks the central nervous system, causing pain and distress. You can read more about MS here.
Essentially, what Dr. Hoffman is trying to do is to use gene therapy to leverage on the liver’s ability to spark immune tolerance, instead of the common method of delivering a therapeutic or curative protein. Deeper into the science, the approach uses a vector known as the adeno-associated viral (AAV is a small virus which infects humans and primates but is not known to cause diseases) to deliver the gene for myelin oligodendrocyte glycoprotein (MOG is a protein believed to be crucial in the myelination of nerves in the CNS) to the liver. Thereafter, it is thought that the MOG would then trigger the immune system to produce Tregs (sub-population of T-cells which regulate the immune system) and prevent rogue cells from attacking the proteins in the myelin sheath.
The significance of the research lies in its results
Skipping past all the deep dive into the biological sciences (unless you feel that it is interesting and want to know more, in that case please comment and I will summarize), they administered gene therapy to mice with EAE, a progressive disease in mice which shares very similar characteristics to MS. The results were that the treatment had an immunosuppressive effect, preventing mice from developing EAE.
Encouragingly, even mice which received a dose of the gene therapy 7 months back were protected from the disease subsequently after they were introduced to EAE. Most importantly, the gene therapy reversed clinical symptoms, and eradicated paralysis even after they were already experiencing moderate attacks from the disease.
They then concluded that “These results suggest that AAV gene immunotherapy reverses the clinical symptoms associated with EAE disease through a mechanism that suppresses tissue-specific inflammation,”.
Hoffman is continuing to develop the gene immunotherapy platform into a technology which can hopefully treat MS in humans. Yet the cautionary tale is that humans are very different, and each individual differs in composition and genetic makeup. We are infinitely complex. While there are results from mice, it cannot be logically or expressly extended or extrapolated to suggest possible efficacy in humans.
Regardless, we remain hopeful for the future as this is definitely a step in the right direction. There is a lot going on in the healthcare space, and we hope to cover more of them progressively. In the meantime, take some time to read this article and start introducing Vitamin D to your diet to fight against MS.