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October 2016

Making sense of the headlines: Potential treatments in the news

Greetings from the Brain Health Registry. Recently, news broke that a promising new Alzheimer’s treatment made it into a Phase III clinical trial. The new drug, Aducanumab, appears to remove beta amyloid plaques, a hallmark of Alzheimer’s, from the brain.  Researchers are cautiously optimistic about the drug’s potential to slow memory loss and treat cognitive decline in Alzheimer’s patients, but what is a Phase III clinical trial and how does it differ from other phases or types of clinical research?

First things first: What are clinical trials?

According to the Food and Drug Administration, clinical trials are voluntary research studies conducted on people. These studies are designed to test whether experimental medications or therapies are safe, as well as how well they work to treat disease. Clinical trials play a critical role in helping scientists learn which treatments are most effective against diseases. For individuals who participate in clinical trials, there are potential risks and benefits. A clinical trial participant may experience side effects or their condition may not be improved by the medication. On the other hand, the participant has the opportunity to take a promising new treatment that is not available to the general public, which may stop, slow down, or reverse their symptoms and disease.

Different types of research studies help scientists zero in on each distinct step in controlling the disease –from diagnosis to treatment.

The types and phases of clinical research

Diagnostic Trials focus on new ways of diagnosing disease. For example,  amyloid PET scans have recently made it possible to spot beta amyloid plaques in the brains of patients living with Alzheimer’s disease, brain plaques that were previously only detectable during an autopsy. Thanks to this research, doctors and dementia care experts now have improved methods to help them identify Alzheimer’s in the living brain.

Prevention Trials help researchers determine whether a certain medication or lifestyle change can stop or slow disease progression. In prevention trials researchers typically study groups of people at high risk of developing the disease.

Treatment Trials test therapies aimed at reducing symptoms or stopping the disease once it has already developed. A treatment trial will usually compare a new therapy with the best-known existing treatment. In cases when no such treatment exists, scientists will compare the new therapy to a placebo. A placebo is an inactive substance (such as a sugar pill) designed to appear the same as the treatment under investigation.  In the United States, a new drug or therapy must go through three phases of testing before it is approved by the FDA:

  • Phase I trials. Researchers test an experimental treatment in a small group of healthy people to determine whether the drug is safe. They also work to identify a safe dosage range and to identify side effects.
  • Phase II trials. The experimental treatment is tested in a larger group of people living with the disease. The continued evaluation of whether the drug is safe is the aim, as well as gathering insights about effectiveness.
  • Phase III trials. In this phase, researchers provide the treatment to even more groups of people with the disease, focusing on its effectiveness and side effects.
  • Phase IV trials. These  post-marketing studies are conducted after the treatment is FDA-approved in order to collect more important information about how to best use the treatment safely.

Observational Studies, on the other hand, do not include an experimental treatment or new diagnostic technique. In this type of research, investigators gather information from groups of people to assess health or behavioral outcomes, without assigning participants to a study group or experimental condition.   The Brain Health Registry is a large observational study, unlike any other. We use the internet to efficiently collect a variety of information from our participants.  Every question you answer and every online test you take in the Brain Health Registry helps scientists better understand brain health.

Clinical research myth debunked

Contrary to what you might believe, all kinds of people may be eligible to participate in research—not just those with an illness. Having a diverse group of participants allows researchers to compare healthy volunteers with those who report health or memory concerns.  This is especially true for Alzheimer’s research, in which it may be possible to one day treat healthy people at risk of Alzheimer’s in order to prevent them from ever getting the disease.

As always, we thank you for your participation in the Brain Health Registry. Your contributions are important and help advance brain health science and research. Together, we can accelerate the development of treatments for neurodegenerative disease.