Gene therapy
Dr Linda Greensmith described the effects of the neurotrophic factor IGF1 (known commercially as Myotrophin) gene therapy, using of a form of this protein known as ‘MGF’. MGF was injected into the muscles of a mouse model of MND, when they first started to show symptoms. Improvements in muscle strength and motor neuron survival were found in treated animals in comparison to controls (untreated animals).
Dr Brian Kaspar has previously shown that gene therapy of the more common form of IGF1 is beneficial in animal models of MND. In his latest study he has taken the seemingly counter-intuitive step of combining this gene therapy with an exercise regime. (There are reports, both suggesting that high levels of exercise is a risk factor for MND, and also that exercise may be beneficial). Dr Kaspar’s results showed that the combination of IGF1 gene therapy and exercise improves the survival of these mice more than the effects of the gene therapy alone. Further study is required to understand the underlying cellular mechanisms causing these effects.
Stem cells
There were no presentations demonstrating the effects of stem cell therapies at this year’s International Symposium. However, Prof Vincenzo Silani presented some laboratory-based research showing that it is possible to turn human stem cells extracted from the bone marrow into a type of support cell for motor neurones called astrocytes. “These data, combined with the recent hypothesis on the essential role played by astrocytes, may open a new prospect for stem cell therapy in ALS, although further investigations and pre-clinical data are needed” he concluded.
Brain-computer interfaces
The final presentation of the meeting described a new technology that may improve the quality of life for people with MND – looking into the possibility of controlling their surroundings, just by thinking about moving an object on a computer screen. The ‘brain-computer interface’ (BCI) that Dr Donaghue (Brown University and CEO of Biotech company Cyberkinetics) has developed consists of an internal sensor to detect brain cell activity and external processors that convert these brain signals into a computer-based output under the person's own control. The sensor, a tiny silicon chip about the size of an aspirin, is implanted on the surface of the area of the brain responsible for movement, the motor cortex. The ultimate goal of this technology is to create a safe, effective and universal system that will enable those with motor impairment to quickly and reliably control a wide range of devices (computer, television etc), simply by using their thoughts.
He concluded "We are eager to see if the initial proof-of-concept obtained thus far in the pilot.. ..for those with spinal cord injury (SCI) can be extended to persons with ALS and other motor neuron diseases."
Press release from Cyberkinetics on this research
Information for researchers