Clinical Studies


Clinical studies include both clinical management projects that improve people’s lives in the present and clinical trials that hold promise in the future – for people living with ALS, caregivers and loved ones. Both are extremely important in the fight against ALS.

Clinical management grants

Grants are awarded to researchers that seek to improve the lives of people living with ALS, caregivers and loved ones without directly testing a drug. See below for examples and more information.

Clinical trials

Clinical trials carry out tests on people to see whether a candidate therapy is safe and works to counter the effects of a disease. Before human patients are the subjects, therapy is tested in cells or tissue grown in the lab, and then in animals, to provide the best possible assurance that a drug will be safe for people to take. Motor neurons derived from induced pluripotent stem cells (iPSCs) also serve as model for disease that directly reflects a person’s genetic makeup in a dish. iPSCs are used both in preclinical studies and in clinical trials in parallel to the drug treatment.

Types of clinical trials

  • Observational: Type of a trial in which enrolled participants are observed. Outcome measures, such as measures of strength or function may be part of the observation. No treatment is given. Often used to learn about trends of symptoms or the course of a disease or to find a disease biomarker. These trials include natural history studies.
  • Interventional: A type of trial in which exposure to the potential therapy being tested, such as a drug, is assigned. It is used to determine the effectiveness of a treatment or intervention.

Clinical trial phases

  • Pilot trial: Is a small study that may include testing of an approved drug for another indication that is being tested in ALS patients to determine safety and tolerability or a study to identify relevant biomarkers.
  • Phase I: Testing looks at the safety of a candidate treatment, often in twenty or fewer people. Participants are examined for any adverse reactions or side effects. If any appear that are judged to be too dangerous, testing is halted and the drug will not advance any further in the clinical trials process.
  • Phase II: Testing attempts to determine the optimal dose, route (by mouth, by injection, etc.) and timing of doses of the candidate treatment. Information about a drug’s ability to help in the disease may be obtained in the course of phase II testing, but such findings are not able to reliably predict a candidate’s effect. Usually for ALS less than a hundred patients are involved.
  • Phase III: The therapeutic effect of the candidate is specifically sought in phase III testing. This is the stage of testing that enrolls enough patients to allow a statistical judgment that a treatment is effective. Phase III trials for ALS usually require hundreds of participants. Large numbers are required due to the diversity of the ALS population. No person with ALS is the same and notably, disease progression from one individual to another is highly variable.

Other information

Due to requirements for producing reliable data, patients may have to be of a certain age, gender, stage of disease or even race, to participate. People living with ALS participating in clinical trials are required to fulfill certain criteria of the disease or disease severity (“inclusion criteria”) and might be ineligible if they show certain other characteristics (“exclusion criteria”).

Clinical trials can only test relatively few patients and must make predictions from a few people that will most likely hold for the patient population as a whole. Statistics provide the means to judge if a change induced by drug treatment is likely to be a real, reproducible change and for doctors to understand whether observations in a restricted set of patients is likely to hold true for the entire population of patients with that disease.

The placebo effect is well known in medicine as a temporary improvement in pain or other symptoms of a disease that follows a fake treatment. It is a real effect. But it is not due to the active properties of a drug. So testing of any new treatment must ensure that the effect is due to the drug and not to the power of positive thinking or to the extra attention from the medical staff that comes with participation in a clinical trial. Simple hope that a new treatment will work can color expectations and perceptions. A participant’s speech can appear clearer. A person can gather strength to sit up unassisted or take a few steps unaided. Decline in abilities can slow or plateau. These are real effects of positive thinking. A control group helps to balance effects of the mind against effects of a drug.

Why It Matters

A person with ALS that participates in clinical trials is a hero. They give their precious time and could be exposed to possible hardships, even though it may be unlikely that they will not live to see its potential benefits, but know the trial could potentially help people in the future. In addition, even if a candidate fails to hold promise, the results of any clinical trial give new insight and direction to those working to solve the mysteries of ALS.