UCL Queen Square Institute of Neurology


Drug study offers hope for Alzheimer’s treatment

14 April 2009

As reported in the Times by Mark Henderson: "A new approach to treating Alzheimer’s came closer when a drug was shown in tests to clear the brain of a damaging protein
A new approach to treating Alzheimer’s passed its first clinical test when a drug developed by scientists was shown to clear the brain of a damaging protein linked to the disease.

The drug completely removed a protein called SAP from the brains of five Alzheimer’s patients, suggesting that it may be a potential therapy for the incurable degenerative condition.

While the study was not designed to investigate whether the drug had therapeutic benefits, its results were so promising that the scientists behind it are now seeking up to £4 million to test it on a larger group.

“There is a severe need for a treatment for Alzheimer’s, and there is nothing available that works well,” said Mark Pepys, of University College London, who is leading the research. “Nothing else looks promising at the moment, and this is a pretty good, safe option. We can’t guarantee it will work, but it’s got a good shot.”

The drug, known as CPHPC, was first developed by Professor Pepys almost ten years ago as a possible treatment for amyloidosis, a disease in which amyloid proteins accumulate in the body’s organs, often with fatal results. While Professor Pepys is still investigating the drug for this purpose, and has signed a deal with GlaxoSmithKline to develop it, he is also pursuing it as a possible Alzheimer’s therapy.

Alzheimer’s also features the build-up of amyloid plaques, in this case in brain cells, making CPHPC a good candidate for treatment.

In the new study, published in Proceedings of the National Academy of Sciences, CPHPC was given to patients aged between 53 and 67 who had mild to moderate Alzheimer’s disease. After three months the drug cleared all SAP from their brains. The study was too short to show whether this had any clinical effect but none of the patients deteriorated during the research period.

“The complete disappearance of SAP could not have been confidently predicted, and the drug, also to our surprise, entered the brain,” Professor Pepys said. Martin Rossor, of UCL Institute of Neurology, who also worked on the research, said: “The safety of CPHPC, together with the novel action of the drug in removing SAP, is very encouraging.”

read more >> The Times | Proceedings of the National Academy of Sciences

reference >>Molecular dissection of Alzheimer’s disease neuropathology by depletion of serum amyloid P component
Simon E. Kolstoe, Basil H. Ridha, Vittorio Bellottia, Nan Wang, Carol V. Robinson, Sebastian J. Crutch, Geoffrey Keir, Riitta Kukkastenvehmas, J. Ruth Gallimore, Winston L. Hutchinson, Philip N. Hawkins, Stephen P. Wood, Martin N. Rossor, and Mark B. Pepys.