Clinical Research Studies: Finding New Hope for Alzheimer’s Disease

Dollarphotoclub_88127006By Samuel Henderson, PhD

Alzheimer’s disease is a progressive neurological disease that primarily affects the elderly. In 2015 it was estimated that 5.3 million Americans were suffering from the disease. As the baby boomers age this number is predicted to more than double, with over 13 million Americans affected by 2050. Without significant new treatments, the rise in the number of Alzheimer’s patients will place a tremendous economic and social burden on the United States. 

Unfortunately, the development of new drugs for Alzheimer’s disease has not gone well. In the last 10 years no new drug substance has been approved. Between 2005 and 2015, there has been a 100% failure rate for new drug substances. The only new drugs that have been approved have been reformulation of existing drugs.

The path to finding new hope for Alzheimer’s disease is dependent on clinical research studies. Drug development in Alzheimer’s has focused largely on the accumulation of a sticky protein called amyloid beta. This sticky protein is thought to be toxic to neurons and to interfere with brain function. Various attempts have been made to either prevent the formation of amyloid beta, or clear it from the brain. Unfortunately, to date these attempts have failed to demonstrate efficacy in clinical research studies. As outlined in previous articles in this series, there are other characteristic changes that occur in Alzheimer’s disease that have attracted the attention of those developing drugs to combat Alzheimer’s.

While researchers identify new targets and develop potential new therapies, there is an opportunity for those with Alzheimer’s and their caregivers to help in the fight against this terrible disease.

By participating in a clinical research study for Alzheimer’s disease you can directly help researchers in their efforts to find a cure.

In this article we will dispel some of the common myths and misconceptions around clinical research studies and discuss what it means to participate in a clinical research trial.

Myth: There are plenty of volunteers
One of the most common misconceptions about joining a clinical trial is that “There are plenty of volunteers and I’m not needed”. This couldn’t be further from the truth. Studies in Alzheimer’s disease often involved hundreds and sometimes thousands of patients and can take years just to recruit enough people for one study. Slow enrollment in Alzheimer’s disease trials is recognized as one of the major hurdles to new drug development. The more people that participate in clinical research studies, the faster progress will be made.

Myth: Participants are guinea pigs that will be exposed to dangerous untested drugs
Some people are afraid to participate in clinical trials because they think they may be dangerous. They say they would rather let someone else be the guinea pig, and prefer to wait until the drug is approved. While it is true that clinical trials do carry some risk because drugs being tested have often not been fully evaluated, it is important to remember that before a new drug can be tested in people, it has to be extensively tested in animals and other models. Prior to starting a clinical research trial the design is reviewed by regulatory authorities, including the Food and Drug Administration and ethics boards. Regulatory authorities review clinical trial design for scientific merit, as well as to ensure that appropriate patient safety and protection measures are in place.

During the clinical trial, the safety of the participants is the primary concern. Any adverse reactions to the drug are carefully monitored, followed, and steps are taken to ensure the safety of the participants.

Myth: I need to stop my current medications to join a clinical research study
Another common myth is that participants in a clinical research study will be required to stop taking current medications in order to test the new one. This is not always the case. In many cases, because the clinical trial drug being tested is unproven, it would be unethical to stop a working medication to test a new one. Generally, participants in a clinical study are asked to be on stable dosing of their current medications, and are asked not to change the dose during the course of the study.

Myth: I will lose my primary care physician if I join a clinical study
Participants in clinical studies for Alzheimer’s will be seen by a clinical trial physician who is an expert in Alzheimer’s disease and has treated many other patients with the same condition. A high standard of care for those who participate in a clinical trial is expected, and there is no requirement for you to stop seeing your regular doctors. Participants are encouraged to continue seeing their regular physician according to their normal schedule.

Myth: If I join, I may not get the drug being tested
Typically, in a clinical trial, participants are randomly assigned to either the active study drug or the placebo, which is an inactive substance that looks like the active study drug. This is called a double-blind study, and it is the best way for researchers to test whether the new drug is safe and effective. While there is a chance that participants will receive placebo, rather than the investigational drug, many trials are designed with what is known as an “open label phase”. During this open label phase all participants receive the active study drug at no cost for a period of time. In some cases, the open label phase can last until the new drug is approved and available to the public.

Myth: It costs too much to participate in a clinical research study
It should not cost participants anything to take part in a clinical research study. The costs of a clinical trial are fully covered by the study sponsor. In fact, many studies provide modest compensation for participating, which may include reimbursement for travel and other expenses incurred during the study.

Myth: I need health insurance to participate
Health insurance is not required to participate in a clinical research study. The costs of the procedures and related medical care received as part of the clinical trial will be covered by the study sponsor.

What to expect when you join a clinical trial
If you are interested in being a part of finding new hope for Alzheimer’s disease by participating in a clinical research study, the first step is to find a clinical study in your area. In many cases, your current doctor may not be well-versed in what clinical research studies are currently underway. There are several online resources that can be used to locate potential studies, including www.clinicaltrials.gov or www.trialreach.com. On both of these websites, you can search for Alzheimer’s disease studies that are enrolling in your area.

Comp_61396007The next step would be to contact the clinical trial site and ask about the study. The study site may be enrolling for more than one study, so if you are interested in a specific study it is important that you relay that information to the study site at the beginning of the conversation. The personnel at the study site may ask you some preliminary questions to gauge your eligibility for participation in a particular study. This may include questions about your medical history or current medications.

Finally, if it appears that you may qualify for the study, the study site personnel will schedule a time for you to come in for a more comprehensive evaluation called a “screening visit”. At this first study visit, the site will explain the study in detail, including any risks or benefits. They will ask you to sign a document stating that you understand the study and the potential risks or benefits. This document is called an “informed consent”. It is very important that you fully understand what is expected of you during the course of the study and that you have all your questions addressed.

You always have the option to take the informed consent home to discuss the study with your family and/or your doctor.
After you have signed the informed consent, a full examination will be performed. This examination will likely consist of a complete review of your medical history, medications you are taking, a physical exam, blood work, and, in the case of studies for Alzheimer’s disease, testing of your memory and thinking, as well as other tests required by the specific research study. This will all be done at no cost to you. Based on the results of these screening tests, the study site will let you know if you qualify for the study. Note that although you may be healthy, it is possible that you may not qualify for the study for other reasons such as specific genetic requirements, or particular lab values that may be out of range for that specific study.

Once you have qualified for the study, you will be scheduled for your “baseline visit”, where additional study procedures are performed, and you will be randomly assigned to either the study drug or the placebo. No one will know if you receive the study drug or placebo – not you, or even the study doctor or his/her staff will know until after the study is complete.

Clinical research studies may last from a few weeks, to a few months, to a few years, depending on the design of the study. During this time, you will be required to attend study visits at regular intervals to check on your health and to measure how the study drug is working.

Joining an Alzheimer’s disease clinical study represents a considerable commitment. By participating in clinical research you will have access to the study drug well before the general public, in addition to the benefit of the study doctor’s expertise. Although there are no guarantees that the study drug will improve Alzheimer’s symptoms, you will have taken an essential step to help advance new drug discovery, and most importantly, you will be a part of the fight against Alzheimer’s disease.


Dr. Samuel Henderson, PhD is the Vice President of Research & Development at Accera Inc. Accera is a clinical stage pharmaceutical company that develops therapies for central nervous system disorders. Dr. Henderson has been working on Alzheimer’s disease and neurodegenerative disease research for more than 10 years. Previously, he investigated the genetics of aging at the University of Colorado and was the Lead Scientist at GenoPlex, Inc. researching anti-aging technologies. Dr. Henderson received his PhD in Molecular Genetics and Cell Biology from the University of Chicago, and has a BA in biology from Washington University. For more information on this topic, Dr. Henderson can be reached at shenderson@accerapharma.com

Speak Your Mind