Results of arimoclomol trial announced by CytRx
On 26 September 2006, CytRx Corporation announced that their drug Arimoclomol had been shown to be safe and well tolerated in a clinical trial of people with MND.
Clinical trial results just announced
Eighty four people with
ALS participated in the double-blind, placebo-controlled trial in 10 centres across America. Participants received one of a range of doses of Arimoclomol for 3 months and were monitored for a further month after taking the last tablet. The drug was found to be safe and well tolerated at all doses.
The preliminary results showed that the drug also appeared to cross the blood brain barrier. Crossing this barrier, which protects the brain and spinal cord, could be important for the delivery of drugs to motor neurones.
CytRx press release on the Arimoclomol clinical trial
A presentation on this clinical trial will be given at the 17th International Symposium on ALS/MND in November 2006.
Next steps for Arimoclomol
The current trial was primarily conducted to determine the safety of arimoclomol. However, the results showed that at the highest dose, there was a trend showing that the drug may have beneficial effects for people with MND.
CytRx Corporation included plans for the next clinical trial of arimoclomol in their press release of the above results. This larger trial in the USA will investigate the beneficial effects of Arimoclomol. No indication was given of when recruitment would begin for this trial. Arimoclomol is not currently available for people with MND.
How does Arimoclomol work?
Arimoclomol works by enhancing a natural cellular defence mechanism within motor neurones. More specifically it works by triggering an increased production of protective proteins, known as 'heat shock proteins' (HSPs), which help cells cope with stress. HSPs help prevent protein aggregation (clumping), which is known to be a feature of MND, they can also help prevent cell death.
MND Association funding similar work
The interest in Arimoclomol as a possible treatment for MND first arose following a report by Dr Linda Greensmith and colleagues at the Institute of Neurology in London. They showed that the drug delayed disease progression and increased survival in an animal model of MND.
As mentioned above, it is known that Arimoclomol exerts its effects by increasing the levels of protective 'heat shock proteins' within cells. However, why these proteins are protective remains unclear. An MND Association-funded PhD studentship starting this month in Dr Greensmith's laboratory will investigate this further.
Results of Arimoclomol treatment in a model of MND (scientific abstract)
Research Information sheet E - Funding MND research (1088kb)
Research
