Researchers at University College London (UCL) have identified a protein, known as serum amyloid P component (SAP), which may be a possible therapeutic target in Alzheimer's disease.
Lead researchers Professor Mark Pepys FRS has even developed a new small molecule drug, CPHPC, which specifically targets SAP and removes it from the blood.
Working in collaboration with Professor Martin Rossor from the Dementia Research Centre of UCL's Institute of Neurology, Professor Pepys' team have also shown that the drug also removes SAP from the brains of patients with Alzheimer's disease.
During a study, the researchers gave the drug to 5 Alzheimer's disease patients for 3 months, and found SAP to deplete not only from the subjects' blood but from their brains also.
Lab tests revealed the molecular mechanism responsible for the unique effect, and also disclosed for the first time the way in which SAP accumulates in the brain in Alzheimer's disease.
The researchers revealed that neither the administration of CPHPC nor the removal of SAP had any side effects in the patients.
"The safety of CPHPC, together with the novel action of the drug in removing SAP from the brain, is very encouraging", said Professor Rossor.
Although the 3-month treatment period was too short to show any clinical benefit, there was no obvious deterioration.
The researchers are planning longer and larger scale clinical studies to confirm safety and seek evidence of benefit to the patients.
"The complete disappearance of SAP from the brain during treatment with CPHPC could not have been confidently predicted, 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," said Professor Pepys.
The study has been published in the PNAS.