In an effort to treat and identify Alzheimer’s patients early on during the disease, the FDA says it may relax the guidelines on Alzheimer’s early-stage trials. The Agency has issued a proposal designed to help researchers and developers find new treatments for patients before the onset of overt (noticeable) dementia.
It is virtually impossible to enroll patients in experimental drug trials for early-stage Alzheimer’s disease because of the lack of definitive biological markers for this stage of the disease, the FDA (Food and Drug Administration) explained. This impasse makes it extremely difficult to develop new treatments.
Alzheimer’s is an irreversible and progressive brain disease that gradually destroys memory and cognition. Experts say that Alzheimer’s is set to affect three times as many people in the USA by the middle of this century, compared to today. The World Health Organization predicted a tripling of Alzheimer’s cases by 2050 globally.
A person with Alzheimer’s eventually becomes unable to carry out basic everyday tasks and requires round-the-clock care. In the majority of cases, Alzheimer’s signs and symptoms appear after the patient is 60 years old, by which time it has been progressing for several years. It is the most common cause of dementia among seniors.
Russell Katz, M.D., director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research, said:
“The scientific community and the FDA believe that it is critical to identify and study patients with very early Alzheimer’s disease before there is too much irreversible injury to the brain. It is in this population that most researchers believe that new drugs have the best chance of providing meaningful benefit to patients.”
In its draft guidance –
Over the last few years, researchers have attempted to find ways to identify early-stage patients using criteria that are based on biomarkers. A biomarker, in this case, is a molecule, gene, or characteristic that helps doctors or researchers determine whether somebody has a disease, or has a higher risk of developing a disease.
Several small breakthroughs have emerged over the last few years in identifying Alzheimer’s earlier on. Experts have tried to develop clinical measures that can more accurately detect subtle cognitive decline. If a combination of imaging and biomarker tests is used, doctors are better able to predict Alzheimer’s disease.
However, today we can more accurately predict Alzheimer’s disease by measuring people’s changes in cognitive abilities than observing alterations in biomarkers.
Dr. Katz said “This draft guidance is intended to serve as a focus for continued discussions between the FDA and pharmaceutical sponsors, the academic community, advocacy groups, and the public. The FDA is committed to vigorously addressing Alzheimer’s disease and will work with industry to help develop new treatments in this early population as expeditiously as possible.”
For patients with noticeable dementia, treatments need to demonstrate not only a positive effect on abnormal thinking, but also how well the patient functions. Human studies are aimed at ensuring that any beneficial effects on cognition are associated with “a clinically meaningful outcome for the patient”.
The problem with early-stage Alzheimer’s disease is that patients have no impairment of global functioning (or extremely little impairment), making it very hard to determine whether a drug or therapy had any clinically meaningful beneficial effect.
For people in the earliest stages of Alzheimer’s who do not perhaps have any functional limitations, such as taking care of themselves, bathing, or getting dressed, just demonstrating that the experimental drug slows down mental decline might be enough to have it approved, the FDA informed.
After winning regulatory approval, the company would then have to carry out larger and longer studies to compellingly show that the medication improves mental function which translates into better health.
The FDA is asking the public to read the Draft Guidance and to make comments during the next sixty days. You can find instructions on how to submit comments in a related Federal Register notice issued today.
The FDA says it will take all the public comments into account when finalizing the guidance document.
The FDA, along with other health authorities in the USA, is calling for both government and private sector efforts to focus on Alzheimer’s treatment and prevention and to improve care and services.
Spinal fluid test – scientists at Nottingham University, England, devised a spinal fluid test which can identify seven proteins. These proteins could be biomarkers for the early stages of Alzheimer’s disease. They hope their research may result in a test for the disease during its initial stage. The researchers published their findings in the Journal of Alzheimer’s Disease. Co-author, Dr. Kevin Morgan, professor of Human Genomics and Molecular Genetics, explained that an early diagnosis of Alzheimer’s could help patients prepare for the future, as well as enable them to be involved in clinical trials.
Smaller brain cortex – people with no signs or symptoms of Alzheimer’s whose brain cortex regions are smaller than normal have a higher risk of developing the disease, according to an article published in Neurology (December 2011 issue).
The researchers, from the University of Pennsylvania and Massachusetts General Hospital, explained that being able to identify patients with no evident memory problems or other symptoms, but who may have a higher risk of cognitive decline, is a major step towards developing news way for doctors to diagnose the disease and to treat it earlier.
Most illness and conditions can be more easily and effectively treated during their early stages.
Written by Christian Nordqvist