Not all research involves giving participants a pill or some sort of therapy. The medical community can learn just as much, if not more, about a disease by learning how a disease progresses in a population. This type of study – called a natural history study – follows a group of people over time who have, or are at risk of developing, a specific medical condition or disease.
A natural history study collects health information in order to understand how the disease develops and how to treat it. Natural history studies are important for scientific advancement in ALD because the research community and drug makers need to know if potential therapies truly change how the disease progresses. Why do some people develop brain involvement, while others have milder symptoms? Why do some experience rapid deterioration, while others develop symptoms at a much slower rate? We have launched two studies to answer some of these questions.
“But what can I do?” At ALD Connect, we have created a Patient Portal where patients and caregivers can directly participate in a research study to create a secure, anonymous, database of ALD patients by telling us about symptoms and how the ALD/AMN affects them over time. On the Portal, your answers to survey questions about your health inform and direct ALD research, so that together we can be one step closer to our ultimate goal – a cure.
The Portal’s NeuroTracker is a rating system that allows patients and caretakers to track a patient’s abilities and health in certain areas over time. Not only does this information help researchers, but the Portal graphs symptoms’ severity and gives patients and caretakers a way to discuss health concerns and symptom management with a doctor.
What Matters to Me? is a section of the Portal where patients and caretakers can directly communicate with researchers. This feature asks patients and caretakers about what disease experience concerns them most; what symptoms do researchers need to address?
Have you been diagnosed with ALD or AMN? Are you a parent of a child with ALD or even a caregiver of someone with ALD? If so, you have valuable information needed to learn more about the disease. Joining is quick and easy. First, create an account with an e-mail and password. Next, review consent form and provide consent to participate in this research study. You can then decide which surveys you want to complete – each should only take a few minutes. We hope that you will continue to track your health over time in the Portal, but how much and how often you participate is up to you.
Join now and help us learn more about this devastating disease and work towards what we all want – a cure for ALD.
Natural History Study (Doctor-Entered Data)
ALD Connect objective is to create a clinical research network allowing researchers to conduct patient-centered comparative research studies. The study involves physicians at Massachusetts General Hospital, University of Minnesota, Stanford University, Kennedy Krieger Institute, and University of Utah to enter data they collect from the ALD patients they care for into a secure ALD Connect database using the NeuroBANK platform (apart from the Patient Portal database). NeuroBANK is an international web-based clinical data repository that allows clinicians and researchers to collect and share clinical data, while keeping the information secure and anonymous.
The data captured by the platform are standardized and harmonized based on a set of agreed upon forms. Although similar to the Patient Portal, we are operating this separate study to learn more about ALD and how the disease changes over time through data collected by experts who care for many ALD patients. With this database, clinicians will be able to compare outcomes against averages calculated from the entire pooled dataset, enabling the rapid identification of important trends.
The study focuses on the following:
- Determining the natural history and correlations of disease-causing mutations in CCALD and AMN as well as other forms of ALD.
- Allowing physician-entered clinical data to be captured and aggregated in the NeuroBANK platform in order for researchers to measure impairment and perform longitudinal measurements (over time) in patients with multiple forms of ALD over a five year window.