Therapies
Therapies for ALS
Amyotrophic lateral sclerosis (ALS) is a disease of the nervous system in which nerve cells responsible for controlling muscles are progressively damaged. For many patients and their families, the diagnosis initially raises many questions—about prognosis, but especially about available treatment options.
Although ALS is currently not curable, various therapies can influence the course of the disease and relieve symptoms. The main goal of treatment is to preserve quality of life and support everyday life as effectively as possible.
On this page, you will find accessible information on current treatment options and new developments in ALS research.
Two medications are currently approved for the treatment of ALS in Germany.
Riluzole is approved for all forms of ALS, while tofersen (Qalsody®) is specifically approved for the treatment of ALS patients with a mutation in the SOD1 gene.
Riluzole is taken orally and exerts its neuroprotective effect through various molecular mechanisms. It slows the progression of ALS by reducing the loss of motor neuron function. In studies from the 1990s, riluzole prolonged survival by several months. Its effectiveness in ALS cases that are often diagnosed earlier today may be greater.
Tofersen is a treatment for ALS patients whose disease is caused by a mutation in the SOD1 gene. As a result of this mutation, disease-causing SOD1 protein accumulates in affected patients. Tofersen is administered into the cerebrospinal fluid, also known as intrathecal administration, and reduces the production of disease-causing SOD1 protein in the brain and spinal cord.
More detailed information on how these medications work is available during a visit to the specialized outpatient clinic for ALS and motor neuron diseases at Ulm University Hospital.
In addition, various new medications and therapeutic approaches are currently being tested in clinical trials.
Why Do We Need Clinical Studies?
Safety and Effectiveness
New medications must be tested in clinical studies before they can be approved. These studies show whether a new treatment is effective in humans and help identify possible side effects.
Vergleich zu bestehenden Therapien
Clinical studies help determine whether a new therapy is more effective than the current standard treatment. This is done by comparing the new therapy with an existing treatment or with a placebo.
Personalized Medicine
Through clinical research, scientists learn which patient groups respond particularly well or poorly to specific therapies. This knowledge is essential for developing individualized and tailored treatment strategies.
Assessment of Long‑Term Effects
Some benefits or side effects only become apparent after months or years. Long‑term clinical studies are necessary to identify these delayed effects.
Regulatory Approval
Without clinical studies, medications cannot be approved by regulatory authorities such as the German Federal Institute for Drugs and Medical Devices (BfArM) or the European Medicines Agency (EMA).
What Is Currently Being Tested in Ulm?
Information on ongoing clinical studies can be found on the website of the Clinical Studies Center of the Department of Neurology at Ulm University Hospital.
A summary of results from ALS studies will follow here.