Research & Down Syndrome

Research helps us learn more about Down syndrome and answer questions that are important to people with Down syndrome, their families, caregivers, and professionals who support them. Research can help us better understand health conditions and treatments, as well as topics like education and employment. 

Down syndrome research covers many different kinds of investigation, observation, data collection, work, and study. Here are the different types of research:

• Pre-clinical Research - a lot of research takes place in a lab before involving humans. Often, this type of research uses cells in a dish or sometimes models like mice.
• Scientific Surveys - a method of formally gathering information about a certain condition, behavior, or experience. Scientific surveys can be digital, in-person, or by phone.
• Observational Natural History (longitudinal) Study- An observational study does not involve a drug or therapy, but measures what is naturally occurring over time. Biological, cognitive, and/or behavioral changes are tracked by gathering the same data points at different points of a person's life. ABC-DS and LIFE-DSR are two observational natural history studies conducted in individuals with Down syndrome.
• Interventional Clinical Trials - this type of research is designed to test the safety and effectiveness of a specific drug, therapy, or device by measuring changes in a person's health, behavior, etc.

Observational

• Important data are collected over time to be used to develop future research
• No experimental medicines or treatments are involved
• Individuals already taking a medicine may be tracked to see long-term health outcomes without changing their treatment plan

Interventional

• An experimental medicine (study drug) is given to the study participant to evaluate its effectiveness in treating a specific condition. In some clinical trials, a placebo, or non-medicine, is given to the study participant. 
• A "double-blind placebo" study is considered the gold standard of clinical trials. This is where neither the study team nor the participant knows if they receive the drug.

There are many kinds of research, and throughout a person's life they may be asked to participate in different research activities. They could be asked to participate in opinion and observation or analysis research, like an online poll, a digital survey, or a questionnaire at the doctor's office.  They might qualify to participate in an interventional clinical trial that takes place over many months or even years.

Clinical trials are the engine of medical progress. A clinical trial checks to see if discoveries that worked in a lab actually work in people. The primary goals of a clinical trial are to:

• Determine if a treatment is safe and effective

• Gather data on dosage, side effects, and overall impact on health

Clinical trials are important for advancing medical knowledge and improving patient care. 

Every drug, vaccine, and diagnostic test we take undergoes strict clinical testing for safety, quality, and effectiveness before it can be approved for use in humans by the U.S. Food and Drug Administration (FDA). Every single medical therapy - whether it is for cancer or Alzheimer's disease - has to be tested in humans.  When a doctor says to take a medication once a day or twice a day, the information came from a clinical trial.