Initial results from an early phase study investigating a new ‘gene silencing’ therapy for Alzheimer’s disease (AD) suggest the treatment has the potential to alter the course of the disease.
The trial represents the first time a type of gene silencing therapy called RNA interference has been used to target a brain disease.
RNA interference is one of the most promising advances in drug development today. It is thought that this novel mechanism will reduce the production of amyloid proteins, thus leading to a prevention of AD diseases including early-onset AD (EOAD), a debilitating disease that strikes in people below the age of 65.
EOAD is the leading cause of dementia in younger people, severely impacting their lives and resulting in early mortality. There are currently no available treatments to halt or reverse the progression of this disease.
Dr Catherine Mummery, Consultant Neurologist at the Dementia Research Centre, at the National Hospital for Neurology and Neurosurgery (NHNN) and Clinical lead for the NHNN Cognitive Disorders Clinic, is one of eight investigators world-wide and the only UK investigator supporting the trial, which is investigating a treatment called ALN-APP, a therapy based on RNA interference.
20 patients with EOAD were enrolled in the first part of the trial. In the study to date, single doses of ALN-APP, administered by injection into the spinal fluid, were well tolerated.
The results demonstrated rapid and sustained reductions in specific amyloid molecules following a single dose of ALN-APP. Patients recruited at UCLH have been supported and monitored at the NIHR UCLH Clinical Research Facility at Queen Square.
Dr Mummery said: “These interim results suggest that the drug targets the amyloid pathway successfully, causing robust lowering of a marker of amyloid pathology in the spinal fluid; so far it has been well tolerated. These promising results now need to be confirmed and expanded upon in a larger phase 2 trial to show whether it translates into clinical benefit.”
ALN-APP is developed by Alnylam Pharmaceuticals in collaboration with Regeneron Pharmaceuticals, Inc.
To find out more about dementia trials and other research at the Dementia Research Centre, visit https://www.ucl.ac.uk/drc/research/clinical-trials or contact us at drctrialenquiries@ucl.ac.uk.
