What are the benefits of the ALS Functional Scale for research?

The ALS Functional Rating Scale (ALSFRS-R) is of central importance for ALS research. The ALSFRS-R can be used to determine the ALS progression rate (ALSPR), which is an established measure of the speed of disease progression .

The ALSPR is determined by the monthly change in the ALS Functional Rating Scale (ALSFRS-R). The slowing of ALS progression is used in therapy research, particularly in most therapy studies, as the main criterion for the effectiveness of a drug . For this reason, the ALS function scale is recorded for all patients in clinical trials at every study visit. The ALSFRS-R data is also relevant for research in patients who are not taking part in a drug trial. This data is used to form comparison groups in clinical research and to develop ALS disease models and digital placebo groups. In clinical trials, a digital placebo group refers to a control group that is compared with a real drug group. By using a digital placebo group, the proportion of patients taking a dummy drug (placebo) can be reduced or completely eliminated. Such a method can also improve the statistical significance of studies and accelerate the development of new therapies.

The development of a digital placebo (or digital twins) is based on ALSFRS-R data (and other clinical information) from a large number of patients (usually several thousand), so there is a high demand for assessment data on the ALS Functional Scale. Data collected during outpatient visits is of great interest, but also data from self-assessment via the ALS app. By providing data from the ALSFRS-R, every patient can make a concrete contribution to supporting ALS research . The effectiveness of symptomatic medication (e.g. to reduce salivation, a speech disorder or a spastic gait disorder) can also be tested using the functional scale. This scale is therefore also of medical importance in the development of symptom-relieving therapies.

The ALSFRS-R data is also used to analyze non-pharmacological therapy. For example, the daily duration of “mask ventilation” (non-invasive ventilation, NIV) or the presence of “artificial ventilation” (tracheotomy with invasive ventilation, TIV) is documented via the ALSFRS-R. This data can be used to scientifically evaluate at what point in the course of ALS ventilation became necessary and what effects ventilation therapy has on the course of the disease. Comparable data can be obtained on nutritional therapy, which is also recorded via the ALSFRS-R. This makes it possible to evaluate the positive effects that nutritional therapy has on body weight and the course of ALS.