02 December 2011 The 22nd International Symposium on ALS/MND has drawn to a close with delegates eager to build on the knowledge shared and to develop the international community’s research and care capability.
Progress in MND care and research can only be accelerated through debate and the sharing of knowledge and the symposium provides a vital, fertile environment where important collaborations and partnerships can be forged.
The final day of the world’s largest conference dedicated to MND focused on two areas essential to the vision of a world free of MND: clinical trials and disease models.
Clinical trials
Prof Robert Miller from the Forbes Norris ALS/MDA Centre in San Francisco opened the clinical trials session commenting: “After a time where patients and sponsors of trials alike have become disheartened about the lack of positive clinical trials, it is exciting to see so many positives, including the recently approved Neudexta, and the dexpramipexole study.”
Dr Pierre-Francois Pradat from the Centre for MND in Paris France, presented the newly released results of the GlaxoSmithKline Phase I safety clinical trial which involved UK centres. The drug was shown to be safe and well tolerated by people with MND and the investigators reported trends suggesting the drug may slow the decline of respiratory function. Tentative plans are underway for a larger clinical trial in 2012.
Prof Leonard van den Berg, from the University of Utrecht The Netherlands, presented results from the Netherlands lithium carbonate clinical trial. Unfortunately, although they found the treatment to be safe, no beneficial effects were seen. The results from the UK clinical trial of lithium carbonate will be released in spring 2012. The UK study was designed differently with a larger number of participants providing the opportunity to identify subtle changes. The results from the UK trial will provide UK clinicians with the definitive answer as to whether they should prescribe lithium to patients who request it.
In recent years MND researchers have come to realise that inflammation within the central nervous system may play a key role in driving the progression of MND. While there is not yet any data available from the ongoing Phase II trial of the new NP001 drug developed by Neuraltus Pharmaceuticals, the Phase I trial that finished in 2010 demonstrated that NP001 appears to decrease levels of inflammatory activity in MND.
Prof Miller explained how the Phase I trial also revealed a relationship between the levels of certain substances that are produced during inflammation and the rate of disease progression. Not only does this hint at the potential of NP001 to slow the disease through suppression of inflammation, it also suggests that the inflammation-related substances could potentially be used as a much needed objective marker of disease progression.
Disease models
Disease models of MND are important both to understand the causes of MND and to quickly, efficiently and accurately screen and develop new treatments. Advances in technology and increased understanding of genetics of MND have led to the development of new models. This is good news for the research community because it is vital they have access to a wide range of models that complement each other and allow rapid validation of promising results.
The disease model session opened with a presentation on what is arguably the most exciting of these new models – models using ‘iPS’ (induced pluripotent stem) cells. iPS cells are created by taking a skin cell from an MND patient and coaxing it back into a basic stem-cell-like state so it can then be re-programmed into a motor neurone. This technique has been groundbreaking for MND researchers as it has allowed the creation of living human motor neurones in a laboratory petri-dish.
Dr Kevin Eggan, one of the leading researchers in this field, provided delegates with an overview of his latest research explaining: “In itself, ALS* is an interesting test tube for stem cell research.” Confirming that he and colleagues have grown motor neurones from iPS cells that looked and behaved like motor neurones, he cautioned that a model based on iPS research is not the panacea of disease models for MND but rather it is an arrow in a quiver of techniques. His findings and others from laboratories around the world will complement those from MND Association-funded iPS cell studies which are focusing on using TDP-43 mutations.
Chicago 2012
By pulling together the different strands of MND research, this three-day conference enables delegates to reflect on the current MND research landscape and to look at how in partnership they can explore and develop the next exciting stages of research. The next time the international MND research community will come together, on such a scale, to share their latest news will be when they attend the 23rd International Symposium on ALS/MND in Chicago, USA in December 2012.
You can catch up on the news and highlights from this year’s symposium by reading our research blog.
Contact:
Louise Coxon Communications Manager
01601 611 843 / 07760 765 142
louise.coxon@mndassociation.org Notes to editors
*ALS (amyotrophic lateral sclerosis) is the most common form of MND and the name it is known by in the US.
