Working together towards a world free from MND

During MND Awareness Month we are highlighting some of the research the MND Association funds in our ‘Project a Day’ series. Today, on global ALS/MND awareness day, we wanted to give you a look at the research into motor neurone disease taking place elsewhere.

Thousands of researchers across the globe are working towards a world free from MND. Rather than tell you each of their stories, we have gone to those that fund and facilitate this research, and asked them how their efforts bring us closer to figuring out the causes of MND, and finding treatments for this disease.

“I find huge inspiration in the knowledge that when I finish my work for the day, the MND researchers in Australia are just beginning theirs.” Prof Martin Turner, University of OxfordRead More »

Understanding more about GM604

20141020_MND Kings College_290The MND Association’s Director of Research, Brian Dickie explains more about ‘GM6’, also known as ‘GM604’, a drug in development by an American pharmaceutical company Genervon.

The Association funds a wide range of research that leads to new understanding and treatments, which may one day, bring us closer to a cure for MND. We are hopeful that the increasing international research effort into the disease will accelerate the development of an effective treatment for MND. However for non scientists I also fully appreciate how the ‘system’ often seems designed to impede rather than assist this process.

There has been much discussion online about the results of a small scale study of a drug called GM604, or GM6, produced by the American pharmaceutical company Genervon. You can read some general comments about the drug on our website. I’ve written this blog to explain in a little more detail why the research community is cautious about the results. Read More »

‘Hothouse’ meeting on drug discovery

Sadie’s recent post on the emerging partnership between Peakdale Molecular and the Sheffield Institute for Translational Neuroscience prompted me to say a little about last month’s Drug Discovery Workshop, held in Washington DC and organised our friends at the ALS Association.

I was fortunate to be invited along to this workshop, which brought together over 100 representatives from industry, academia, drug regulators and government and charitable funding agencies, to share their findings and discuss the future directions and opportunities for MND drug discovery. ‘Hothouse’ meetings like these are vital in giving those working in academic labs an important insight into the complexities of turning new knowledge of disease processes into ‘druggable’ compounds.

Those from industry get to see the new theories that are coming out of the academic labs, while the funders can start to identify where targeted early-stage support may help to encourage industry to follow up with the larger-scale investment needed to take ideas from bench to bedside At a time when some of the biggest drug companies are pulling back from working in neurodegeneration, the mood at the meeting might have been muted, but delegates were positively upbeat. One cause for optimism is that some companies, such as Biogen Idec, have seized the opportunity to fill the gap and increase their investment in this area.

Moreover, universities around the world have benefitted from an influx of new staff with extensive expertise in drug discovery, strengthening one of their historical areas of weaknesses. Universities are generally very good at unpicking the complex biological processes that occur in health and disease, but very poor at turning this knowledge into treatments. Another reason for the optimistic mood at the meeting was the clutch of new gene discoveries that occurred last year, in particular the identification of the chromosome 9 form of MND  which promises to open up many of new secrets of the disease. Researchers have collectively now found about two-thirds of all the causes of familial MND. As we identify more causes, generate better models and home in on the common cellular changes that drive the disease, the opportunities for drug development are going to increase.

Meetings such as this help focus attention on the major challenges – but also the exciting opportunities – that lie ahead.