Last month I wrote about scientific persistence paying off for Mark Pepys, professor of medicine at University College London, who has worked for more than 30 years on diseases caused by the abnormal build-up of amyloid protein.
He was celebrating then the signing of a deal with GlaxoSmithKline, the giant UK-based pharmaceutical group, to develop a treatment for amyloidosis, a rare fatal disease in which amyloid accumulates in organs throughout the body.
GSK will use a drug called CPHPC, which Prof Pepys developed during the 1990s, as the basis of the treatment. CPHPC works by removing SAP, a blood protein that sticks to amyloid and stops enzymes dissolving it away.
Now Prof Pepys is celebrating again. The Proceedings of the National Academy of Sciences, a leading US research journal, has published promising results from the first clinical trial of CPHPC to treat Alzheimer’s disease.
Prof Pepys and UCL colleagues gave CPHPC to five Alzheimer’s patients for three months. They found that it removed SAP from their brains.
The treatment caused no side-effects and the patients showed no clinical deterioration during the trial, though it did not last long enough for the researchers to assess clinical benefits.
Rececca Wood, chief executive of the Alzheimer’s Research Trust which part-funded the study, said the results “are cause for cautious optimism. New treatments for Alzheimer’s disease are desperately needed, and it is possible that this small molecule could be a future candidate.”
Prof Pepys now plans a larger trial in which 80 to 100 patients will receive either CPHPC or a placebo pill over several months, through he still needs to raise funding for it.
“That should tell whether CPHPC brings a significant clinical benefit,” he says. “It will not need to do very much to be better than the Alzheimer’s drugs currently on the market.”
Prof Pepys started developing CPHPC with support from Roche. But the Swiss pharmaceutical group pulled out of the collaboration, saying it had other research priorities, and last December Roche passed all its rights in CPHPC to Pentraxin Therapeutics, a UCL spin-out company.
Following last month’s deal with GlaxoSmithKline on amyloidosis, Prof Pepys hopes the clinical results with Alzheimer’s might tempt GSK to take on CPHPC as an treatment for that disease too.
While Alzheimer’s is potentially a far larger market than amyloidosis, development costs would be much greater – and there is much more competition from other drug companies.
Although there is much medical controversy about ultimate cause of Alzheimer’s disease, Prof Pepys believes that CPHPC could treat the underlying disorder by enabling enzymes to remove amyloid from the brain.