There’s an interesting story doing the rounds in the science news at the minute about the trial of a drug normally used to treat cancer that has shown promise in the treatment of Parkinson’s Disease (PD). Nilotinib is currently on label in the US to treat myeloid leukaemia but a small trial of 12 patients has been done on people suffering from PD at Georgetown University. I find this particularly interesting as I know a little about PD through my work. I’ve spent several years carrying out gene tests for PD and know many clinicians and researchers working on the disease, indeed a significant portion of the building I work in is dedicated to research into PD.
Although the precise mechanism of PD pathology remains elusive, and in actual fact there is a good body of evidence now to suggest that there is probably at least two distinct mechanisms, we do know that a part of it is the build up of toxic proteins that neuronal cells are able to breakdown. These proteins clump together to form plaques known as Lewy bodies and a mass of these neuron killing artefacts are one of the classic hallmarks of PD.
The drug works by boosting the body’s lysosome system, the system that would usually clear out these toxic proteins. One of the exciting parts of this treatment is that there are a host of other diseases out there that are caused by the building up of protein aggregates and so the applications could extend beyond just PD.
Effects on patients were dramatic. People rendered mute were able to talk again. Patients that had been bed bound were able to get up and even make the bed. Another plus was that the doses were quite low compared to those required for cancer treatment, which is just as well as the side effects of larger doses can be quite unpleasant in themselves.
This is not, however, a complete home run. Whenever I write a post about something scientific I always go back to the original article and provide a link to it so you can check it out yourselves if you so desire. The only thing I can link back to in this case, though, is a press release from Georgetown University. As far as I can see the work has been presented at a conference of the Society for Neuroscience but it has not been reported in a peer reviewed journal. Whilst this doesn’t mean that the work is worthless it does mean that we have to be highly sceptical of it at this juncture. This was just a safety trial of 12 patients and without a placebo control and the beneficial effects of potential PD drugs have a nasty habit of disappearing upon closer scrutiny. The researchers are now recruiting people for a properly controlled trial but the results of that are likely to be at least a year away. Let’s wish them every success.