What should I know about clinical trials?

It’s been over 15 years since a new drug for Alzheimer’s disease hit the market (Memantine, also known by the brand name Namenda, in 2003). The race is on amongst researchers to find the next big discovery and ease the burden of this disease for patients and their families. New research findings are made every day, and hundreds of research studies are taking place across the country – so why is it taking so long for a promising clinical intervention to surface?

Bringing a single drug to the market is a laborious process. For any single drug approved by the FDA, there was at one point thousands of compounds researched in the laboratory. This is defined as Phase 1: Drug Discovery. A mere several hundred successful compounds are then tested for effectiveness and safety with animal models as part of the second phase: Pre-Clinical Development. Each one of these phases can involve many labs across the country, teams of several people, and can last many years.

Photo source: Genome Research Limited

Once biological effectiveness and animal safety is established, a select handful of potential compounds will enter into the third, Clinical Trial, phase where compounds are analyzed for effectiveness, safety, and long term safety on humans. It is also at this point when the trial will move to analyzing larger scale numbers of people and potential risks versus benefits. This phase will ultimately identify the exact dosage given to patients, side effects, and more.

Throughout this lifecycle of drug development, potential compounds are regularly cancelled based on research findings of ineffectiveness – perhaps the compound was effective biologically among mouse models but not in humans, or perhaps the drug was tested on the wrong population or at the wrong stage of the disease. This is what funnels the first phase of thousands of compounds down to one safe, effective drug that enters the market. Generally, if these cancellations take place in the laboratory during the pre-clinical phase or prior, these cancellations go unnoticed. However, once the compounds under consideration enter the clinical phase and are tested on humans, these cancellations can become more disappointing, and even devastating.

Getting involved in a clinical trial is a big step for patients and their families. There are many factors that should be taken into consideration before volunteering. Dr. Judy Heidebrink, the University of Michigan’s principal investigator on Alzheimer’s disease-specific clinical trials, weighs in on what is important for patients and families to know: “It’s important to understand the goal of the study. Are researchers testing for the agent’s safety in humans or are they expecting to see an intervention-worthy benefit?” Understanding this ensures expectations are in the correct place when getting started in the study. “It’s also important to understand that there is a possibility that the study could end for any number of reasons.” But there are things you can do to prepare yourself, should that happen. “Be sure to understand the wrap-up process if the study were to come to a close. Would you be brought back in for a wrap-up visit? Would you receive a phone call? Would you receive any of the data of the trial, or your testing scores (if applicable)? This procedure often varies.”

Dr. Judy Heidebrink

Many patients and their families get involved in a clinical trial in hopes of receiving a potential drug therapy before it hits the market. There are many conditions to take into account with this approach. Clinical trials are typically broken down into groups that receive the active drug compound, and those that receive the placebo. It can be valuable to understand how these groups are determined when getting involved, and maintain reasonable expectations that you may receive the placebo rather than any experimental drug. It’s also important to understand that participating in any clinical trial could impact future eligibility to participate in other trials. Because of the nature of drug development, it’s also often difficult or impossible to be aware of which future studies may have this condition when getting involved in a current study.

Getting involved in a clinical trial is a big decision. While there are many risks, there are also many rewards. Patients that participate in a clinical trial are giving the gift of knowledge to the entire healthcare community. So much information is gathered from a clinical trial. Participants also have the possibility of being part of the discovery of disease intervention for themselves and future generations.

Several clinical trials are taking place in Michigan. If you or someone you know are interested in participating, we hope you’ll visit our website here. Nationwide clinical trials information is also available at clinicaltrials.gov.