A drug developed by scientists at University College London has shown great potential as a treatment for Alzheimer’s disease, through its ability to cleanse the brain of a protein thought to play a major role in the condition.

Lead by Professor Mark Pepys, UCL spin out company Pentraxin Therapeutics has been working on the compound CPHPC that targets a protein called serum amyloid P component (SAP) - a universal constituent of amyloid deposits and plaques, also present in the brains of patients with AD - and removes it from the blood.

The first study of the drug in patients with AD, in which CPHPC was given to five individuals over a three-month period, showed the expected depletion of SAP from the blood as previously observed in all patients given the treatment in trials for other indications.

However, the findings, published in the Proceedings of the National Academy of Sciences, also showed a “remarkable disappearance” of SAP from the brains of patients given the compound, and it is this novel action, as well as the absence of any side effects associated with the drug, which has fuelled excitement over its potential as a treatment for AD.

Surprise result
“The complete disappearance of SAP from the brain during treatment with CPHPC could not have been confidently predicted” commented Professor Pepys, “and the drug, also to our surprise, entered the brain”.

“Coupled with the absence of any side effects, these new findings strongly support further clinical studies to see whether longer term treatment with CPHPC protects against the inexorable mental decline in patients with Alzheimer’s disease,” he added.

The compound’s potential is also being assessed in other therapeutic areas. Last month, it was announced that UK drug giant GlaxoSmithKline has partnered with Pentraxin to develop a dual treatment for amyloidosis - a potentially fatal disease caused by excess abnormal amyloid proteins in the body tissues – which combines CPHPC with an antibody.