Clinical Trials For Alzheimer’s Disease

Unsurprisingly, an Alzheimer’s diagnosis brings sadness, fear, and uncertainty. It’s a grueling journey for millions of patients and their families across the world. Upon diagnosis, patients and families search for treatments, answers, and – most of all – hope. Importantly, clinical trials for Alzheimer’s bring scientific advances that bring us closer to a potential cure for this terrible disease. Clinical trials for Alzheimer’s provide access to promising treatments and therapies, while helping future patients.

Note: for ease of reading, when I refer to Alzheimer’s, I am generally referring other types of dementia as well.

Why consider clinical trials for Alzheimer’s?  

By participating in a clinical trial, you can help yourself and others. You can help researchers discover new ways to potentially diagnose, treat, and prevent Alzheimer’s disease and related dementias. Participants help scientists figure out what works, what doesn’t, and what might come next. Without participants, progress would stall. 

Critically, the primary benefits to a participant are two-fold:

• You (or your loved one) may receive medical care and new treatments that are not yet available otherwise.
• You will learn about Alzheimer’s and the progression of your (or your loved one’s) condition.

Additionally, there are many benefits of clinical trials for Alzheimer’s that patients and families often overlook. For instance, many trials:

• Use non-drug interventions. 
• Include advanced imaging or genetic testing that would normally not be part of standard care.
• Provide caregiver support programs.
• Provide resources, including educational materials.
• Help families find counseling, legal advice, and even day-to-day tips for managing routines. 

Simply put, joining a clinical trial for Alzheimer’s helps patients and families in a variety of ways. 

Are clinical trials safe?

Importantly, clinical trials aren’t always invasive or risky. Additionally, clinical trials must follow very tight rules that ensure ethical conduct. However, every treatment, whether standard or as part of a clinical trial, comes with potential risks and side effects. So do your research and ask as many questions as you need to feel comfortable.

Types of clinical trials.

There are two types of clinical research studies: observational studies and clinical trials.

• Observational studies collect information from people and compare that data over time. This helps researchers learn how different behaviors or lifestyles relate to health and disease. Additionally, it helps researchers understand how a disease progresses over time.
• Clinical trials test new drugs, medical devices, surgical procedures, or behavior and lifestyle changes, such as exercise. Additionally, clinical trials may test ways to detect and diagnose diseases, and may look into how to improve care for patients. Generally, researchers compare trial results with a control group who do not receive the intervention under study.

Clinical trial phases.

Generally, trials are comprised of 4 phases: 

• Phase I focuses on patient safety, looking at risks and side effects.
• Phase II tests for effectiveness and side effects, to help determine proper dosages.
• Phase III expands the testing group to further study the effectiveness of the treatment while monitoring side effects.
• Phase IV occurs after FDA approval to monitor long-term results.

What types of clinical trials for Alzheimer’s are currently underway?

Researchers now recognize that Alzheimer’s and dementia are diverse diseases that will need a nuanced approach. Interestingly, researchers are studying several types therapies and drugs, including those described below.

Amyloid-targeting therapies.

Amyloid plaques are sticky clumps in the brain believed to disrupt communication between neurons. Anti-amyloid drugs remain a big focus of research, with several late-stage trials in place, including the following:

Excitingly, remternetug, which is currently undergoing clinical trials, has shown a significant reduction in brain amyloid plaques. This medication may benefit those with early Alzheimer’s.

Interestingly, the FDA recently approved Kisunla for patients with mild cognitive impairment or mild dementia stage of disease

Tau-targeting therapies.

Tau proteins can tangle inside brain cells and trigger degeneration. Accordingly, many clinical trials target tau protein to prevent tangles or promote clearance.

Oral medications.

ALZ-801 may become the first oral disease-modifying treatment for Alzheimer’s, making treatment more convenient for patients.

Multi-target drugs.

Since Alzheimer’s is a complex disease with multiple causes, researchers are evaluating drugs that address various aspects of Alzheimer’s pathology at once rather than one target.

Stem Cell Therapies.

Investigations of stem cell therapies are still in the early research phase. However, the therapies may be able to replace damaged brain cells and promote tissue repair.

Immunotherapies.

Immunotherapies aim to harness the body’s immune system to fight Alzheimer’s disease. These treatments include vaccines and monoclonal antibodies that target amyloid plaques, tau tangles, or other pathological proteins.

Non-medication approaches.

While evaluating medications is the main focus for most trials, researchers also look at non-medication interventions. For example, research may evaluate the impact of cognitive training, exercise, diet, and social engagement, including support groups.

Focus on neuropsychiatric symptoms.

Unfortunately, many symptoms of Alzheimer’s, including agitation, depression, and sleep issues, can make life difficult for patients and their families. Fortunately, trials are underway for treatments for these types of symptoms as part of a comprehensive approach to Alzheimer’s care.

Can you or your loved one enroll in a clinical trial for Alzheimer’s?

Deciding to enroll is a clinical trial is a big decision. Before making a decision, talk to your doctor, research your options, and consider what will work for you (or your loved one). And be realistic about your ability to manage required appointments and testing, including any travel if required.

When is the right time to enroll?

Most people think clinical trials are only for the terminally ill with no options left. But that’s far from the truth. In fact, many Alzheimer’s studies start with people in the earliest stages of cognitive decline – sometimes even before symptoms become disruptive. Experts believe this proactive approach may be the best way to preserve cognitive function and slow disease progression over time. However, Alzheimer’s patients may also enter clinical trials at other phases of the journey.

Additionally, some trials include people who have a high risk of developing Alzheimer’s but currently have no symptoms.

Will you or your loved one qualify for clinical trials for Alzheimer’s?

Not every patient qualifies for a trial. Importantly, eligibility for each trial is usually based on factors like age, stage of the disease, existing conditions, and medications. 

But even if you or your loved one is matched to a clinical trial, it doesn’t mean it’s the right trial for you and/or your loved one. It’s important to ask questions to see if the trial is a good fit.

How can you find clinical trials for Alzheimer’s? 

If you’re considering enrolling a loved one, start with his/her neurologist who likely has access to trial databases or can refer you to a local research center. 

Additionally, you can search online registries by location, diagnosis, and trial phase. Consider these options: the US government sites Alzheimers.gov and ClinicalTrials.gov. You can also find trial information at trialmatch (sponsored by the Alzheimer’s Association and Carebox).

Importantly, each trial has eligibility criteria – a list of what types of patients/conditions they could accept. Once you’ve created a list of possible trials for which you (or your loved one) may qualify, you – or your doctor – should reach out to the trial’s team to learn more. Now is the time to learn if you (or your loved one) meet their eligibility criteria. You can also learn details about the trial, including the number of visits expected, and compensation for out-of-pocket costs (if any).

Importantly, the clinical trial listing should include contact information. Try to contact the study coordinator who can answer your questions and help you decide if the trial is a good fit.

Questions to ask before signing the dotted line.

There is a lot of information to consider when thinking about enrolling in a clinical trial for Alzheimer’s. When discussing trials with your doctor or with a study coordinator, take notes and/or record the conversation (ask first!) so you can clearly remember the details discussed.

Below is a list of questions created by Clara Health to ask your doctor and/or the clinical trial:

1. What’s the study’s goal?
2. Why do researchers believe the study’s treatment might be effective? Have there been other studies on it?
3. Is there a possibility I could receive a placebo (a harmless medication with no therapeutic effect)? If I receive a placebo, will I get the test treatment at the end of the trial?
4. Can I take my regular medications while participating in the study?
5. Can I access the treatment after the study finishes? How?
6. Will I have to travel for the study?
7. How many visits are there? What will happen at each appointment?
8. How long will the trial last? Will overnight stays be required? If so, how often?
9. Who will oversee my medical care while I am participating in the trial?
10. Will I receive the study’s results?
11. Who will pay for the care required during the trial?
12. Are there any other potential costs with participating even if I have insurance?
13. Do I get any form of compensation for participation?
14. Can I receive reimbursements for my expenses, such as travel costs, lodging, food, etc.?

Making a decision regarding enrollment.

The decision process can feel overwhelming. Don’t feel rushed – take your time to get the facts and discuss the pros and cons of your options with your doctor. However, realize your doctor may not know all the details of a given trial and may not have the answers to your questions.

Importantly, a second opinion can help you choose a clinical trial. For more information, read Why are Second Opinions Important?

Finally, discuss the options with your family.

Don’t second guess yourself.

Whether you choose a trial, or decide to go with standard course of treatment, don’t harshly judge yourself and your decisions as time progresses. Realize you are making the best decision with the information you have at the time. No one can predict the future.

And don’t feel guilty if you decide not to enroll in a trial. Every family must make the decision that feels right for them.

One family’s experience with clinical trials for Alzheimer’s disease.

When Joe started forgetting names and wandering off during his daily walks, the family chalked it up to aging. But when Joe couldn’t recognize his own home, the family knew something bigger was happening. Unsurprisingly, when they received the Alzheimer’s diagnosis, they were scared and unsure about next steps.

After researching options and speaking with specialists, the family decided to enroll Joe in a memory study. Although they didn’t expect miracles, they saw real benefits. Not only did Joe become part of something bigger than himself, but the doctors also helped the family understand the expected disease progression.

Joe’s family felt supported by a network of doctors, researchers, and other families who understood the realities of living with Alzheimer’s. Importantly, in addition to medication, Joe’s clinical trial involved memory workshops and diet changes. Additionally, Joe learned new games to help with his cognition.

Joe’s family saw small improvements – not dramatic, but meaningful. He smiled more. He was present. Joe and the family felt they made the right decision – for Joe and for the legions of people who will develop Alzheimer’s in the future.

Learn more…

Living with Alzheimer’s is challenging for patients and families. Learn more:

• Diagnosing Alzheimer’s
• How to Care for a Loved One with Dementia.
• Care Homes for Dementia Patients: When Is It the Right Time?
• Hospice Care for Dementia Patients – Everything You Need to Know
• How to Prepare for a Loved One’s End-of-Life Care.

I wrote this post in collaboration with Anthony Ciabarra, MD, PhD. Dr. Ciabarra has been practicing neurology and supervising neurology clinical trials in Fullerton for over 20 years. As a graduate of Yale University School of Medicine, he participated in the Medical Scientist Training Program receiving an M.D. degree and a Ph.D. degree in the Department of Pharmacology. Dr. Ciabarra has participated in the development of a number of breakthrough treatments including new treatments for Alzheimer’s disease, Parkinson’s disease, migraine headache, and other neurological conditions.