An experimental compound originally aimed aimed at tackling cancers, particularly brain and basal cell, has now been found to be effective at treating multiple sclerosis (MS). The drug appears to alleviate symptoms of MS, such as leg weakness and forms of paralysis. There are hopes that this could show promise for human application in the treatment of this debilitating degenerative disease.
Multiple sclerosis is a neurological condition that affects around 100,000 people in the UK. The disease arises as a result of the myelin, the insulating coating of nerves, become damaged because of autoimmune destruction or fault with myelin producing cells. The disease is characterised by lesions, commonly in the central nervous system, that lead to reduced myelination, this reduction in insulation causes aberrant nerve signal transduction and miscommunication in the body.
Within these lesions, a particular signalling molecule, Gli1, is usually upregulated, which is shown to sometimes play a role in cancer growth. This new drug, GANT61, acts by blocking this signal. A research team from NYU Langone Medical Center conducted a study in which mice with damaged myelin were given a dose of GANT61 daily for one month. The results of this study showed a 50% increase in mylelin when compared to untreated mice and also showed an increase in neural stem cells that subsequently turn to myelin repair. The untreated mice suffered repetitive phases of leg weakness as well as bladder weakness, treated mice were able to recover from initial episodes of paralysis.
Professor Salzer, researcher at the institute commented on the research: “Our study results suggest that a potential long-term strategy for treating multiple sclerosis”. He added: “Our findings also make clear that there is a resident population of adult neural stem cells that we can target and recruit to treat the disorder”.
The significance for patients, if this drug proves safe and effective in human systems, would be great. In the least, it may prove to alleviate symptoms of a currently incurable disease. However, as always it is important to realise the limitations of models, a significant amount of work will be required before getting close to a human application. It is unlikely that such a drug would become a singular solution to the problem of MS, the research team hopes to follow up with more refined drugs to inhibit Gli1.