The US government has stepped up the battle against Alzheimer’s disease by helping to launch a $100m clinical study on a promising new drug, crenezumab, that doctors hope will help against the debilitating condition. The National Institutes of Health is actively involved, helping Genentech and the Banner Alzheimer’s Institute to fund the research.
“These actions are the cornerstones of a historic effort to fight Alzheimer’s disease,” said Kathleen Sebelius, secretary of the US Department of Health and Human Services, on Tuesday. “This is a national plan – not a federal one, because reducing the burden of Alzheimer’s will require the active engagement of both the public and private sectors.”
Dementia is the significant loss of intellectual abilities, such as memory capacity. In 2010 there was an estimated 35 million people with dementia worldwide, a number which is assumed to nearly double every 20 years as the number of elderly people continues to grow. Alzheimer’s disease is the most common form of dementia; other causes include HIV infection, brain tumours, multiple sclerosis.
Alzheimer’s symptoms begin as lapses of memory, difficulty in finding the right words for everyday objects and mood swings. As the disease progresses, individuals display personality changes, unsettling behaviour such as wandering off and getting lost, extreme mood swings and loss of inhibition and sense of appropriate behaviour. This is an extremely frightening disease for both the patients, who feel confused and afraid, and their loved ones, who witness the progressive deterioration.
Alzheimer’s is characterized by excessive accumulation of the amyloid β protein, that leads to amyloid plaque formation and eventually cell death. Additionally, insoluble twisted fibers form inside cells, called neurofibrillary tangles. Crenezumab is a monoclonal antibody that attacks amyloid plaques in the brain. The aim is to use antibodies to specifically bind to target cells or proteins in order to stimulate the patient’s immune system to attack these. Monoclonal antibodies are ‘monospecific’, i.e. they have affinity for the same antigen. Antigens evoke the production of one or more antibodies by the immune system.
Most participants for the clinical trial will come from the world’s largest family to experience Alzheimer’s, an extended clan of 5,000 people who live in Medellín, Colombia, and remote mountain villages outside that city. Approximately one-third of the 5,000 family members carry an autosomal dominant allele of presenilin-1 (PSEN1) that causes the early onset form of Alzheimer’s disease, with onset usually by the age of 45.
Presenilin-1 is one of the four core proteins of the presenilin complex, which mediates proteolytic events (i.e. the breakdown of proteins). Presenilin-1 is important for the breakdown of amyloid and as such plays an integral role in Alzheimer’s that is characterized by accumulation of amyloid.
The government is getting involved and that is already a step forward, irrespective of the outcome of the clinical trial. The Obama administration also launched the National Alzheimer’s Project Act last year, in the aim of finding preventive and treatment measures by 2025. Given that the elderly population is continuously increasing, it is particularly important to find a preventative measure for Alzheimer’s disease and I am happy to see the government getting involved!