A drug to treat Motor Neurone Disease (MND) – also known as amyotrophic lateral sclerosis (ALS) or Lou Gehrig's disease – appears to have dramatically slowed the diseases' progress in Phase I clinical trials. The scientists responsible are keen to stress the trials' limitations and say they “don't want to add to the hype”, but the work certainly marks one of the most encouraging developments in the fight against the disease that killed Stephen Hawking we have seen.
For people with MND, respiratory failure is the most common cause of death. So the announcement at the 29th International Symposium on ALS/MND that 32 patients given the drug CuATSM showed significantly less decline in lung capacity than beforehand is very important.
The trial's participants had very mixed rates of progression and time since diagnosis, forming a fairly representative sample. Many were also suffering cognitive decline prior to being put on the drug. This also stabilized significantly. A trial like this isn't designed to quantify effects, but disease progression apparently slowed by more than half.
Dr Kevin Barnham of Melbourne's Howard Florey Institute stressed to IFLScience the trial had no control group, so the comparison could only be with the patient's own rate of decline beforehand. It also lasted for just six months, so we don't yet know if the benefits are sustained.
Considering the best existing MND drugs only extend life by 2-3 months, however, these results are easily good for Collaborative Medicinal Development, the company developing CuATSM, to begin enrolling people in a double-blind Phase II trial to start later this year.
The story of CuATSM is an excellent example of the far from linear way science progresses. Fifteen years ago Barnham was part of a group of scientists were toying with the theory that copper had an important part to play in the development of Alzheimer's disease. Barnham told IFLScience they designed a drug that could cross the blood-brain barrier and alter the movement of copper. For a control, they needed a similar drug.
CuATSM was that control. Having used it in an animal trial, the team wondered if they could find any other applications. Drugs that cross the blood-brain barrier are quite rare, and CuATSM has anti-oxidant properties, which raised the possibility it might protect brain cells from damage. Barnham had worked on MND and, going on what he calls “a hunch” tried giving CuATSM to animals with the disease.
When it came to MND, CuATSM's effects in animals were impressive, although Barnham told IFLScience he and his colleagues are still debating the mechanism. The results for Parkinson's models were also sufficiently encouraging that Collaborative Medicinal Development has a second Phase I trial currently underway. However, animal trials showed no benefits for Alzheimer's, the disease against which CuATSM was originally designed.