An entirely new type of pharmaceutical substance for Alzheimer’s disease developed by Uppsala scientists is now starting to be clinically tested in the US. It attacks the early stage of the protein filaments that cause the disorder, so-called protofibrills.
“It would be a giant step forward to have a drug that actually targets the fundamental cause, as opposed to merely alleviating the symptoms, as today’s medicines do,” says Lars Lannfelt, professor of geriatrics at Uppsala University.
Lars Lannfelt is very optimistic. This is the first time a substance that directly attacks protofibrills is being clinically tested. The drug was developed in collaboration with the small Swedish biotech company BioArctic Neuroscience AB and the Japanese drug company Eisai. Just over 80 Alzheimer’s patients are included in the clinical trial.
Alzheimer’s disease is characterized by abnormal protein build-up in the brain, so-called plaques. They consist of long strands of protein, fibrills. The Uppsala researchers have previously shown that the prime danger is the pre-stage of these filaments, and they have therefore focused their research on finding a drug candidate, a monoclonal antibody, that specifically targets the pre-stage, so-called protofibrills.
“One of these monoclonal antibodies, mAb158, has proven to completely inhibit the disease in mice,” says Lars Lannfelt.
The antibody has since been further developed to reduce the risk of triggering a reaction from the human immune defense, and in a few years the scientists will find out whether it has the same positive impact on humans as on mice. If the results are positive, the next step will be to involve considerably more patients.