Frontotemporal degeneration (FTD) research is how we move from understanding the disease to developing treatments and ultimately, cures. Two main types of research drive progress: observational (longitudinal) studies and clinical trials. Each plays a different but equally important role.

Observational (Longitudinal) Studies: Understanding the Disease

Observational studies follow participants over time to learn how FTD develops and changes. These studies may include cognitive testing, brain imaging, blood samples and potentially optional spinal fluid testing.

They help researchers:

• Understand how FTD progresses
• Identify early biological changes
• Discover biomarkers needed to design clinical trials
• Connect participants to future research opportunities

Studies like ALLFTD Study help build the scientific foundation that makes treatment trials possible.

Clinical Trials: Testing Potential Treatments

Clinical trials test whether new therapies are safe and effective. They are the step that turns scientific discoveries into real treatments for patients.

The Five Stages of Clinical Trial Research

Preclinical Research

Researchers study disease in the lab and in models to understand how a therapy might work and if it appears safe enough to test in humans.

Phase 1

Small group of participants. Focus: safety, dosing, and side effects.

Phase 2

Larger group. Focus: Does the treatment work? Continued safety monitoring.

Phase 3

Large, multi-site studies. Focus: Confirm effectiveness, monitor side effects, compare to standard care. This phase is often required for regulatory approval.

Phase 4 (Post-Approval)

Occurs after approval. Focus: Long-term safety and real-world effectiveness.

(Source: National Institute of Aging website, an official website of the National Institutes of Health)

What Research Is Not

Research is not guaranteed treatment, and participation is always voluntary. Many studies include people at different stages including at-risk family members and care partners.

Why It’s Important to Consider Research

Participating in research helps:

• Accelerate scientific discovery
• Ensure patient and family experiences shape research priorities
• Build toward future treatments
• Connect you with research opportunities and information

The Bottom Line

Progress in FTD depends on people who are willing to explore research participation. If research might be right for you or your family, consider taking the first step today by learning what options are available.

Trusted Resources to Learn More

The Progranulin Navigator helps GRN-FTD families find research, education, and support resources. Visit here.

FTD Disorders Registry connects people affected by FTD with research opportunities and helps accelerate discovery. Visit here.

Consider exploring research today.