In March 2021 the European drug agency granted Orphan status to Ganglioside GM1 for treatment of ALS. It was presented by 3R Pharma Consulting GmbH, a consultancy organization acting as proxy for another organization. Why the European drug agency granted Orphan status is still unknown, but it seems that this agency is much more permissive than it's US counterpart, the FDA.
By coincidence, a new article on June 12, was about how ganglioside GM1 may be the cause of Guillain–Barré Syndrome after infection.
The quality of life after an attack of Guillain–Barré syndrome can be significantly impaired. About a fifth of patients are unable to walk unaided after six months, and many experience chronic pain, fatigue and difficulty with work, education, hobbies and social activities. But usually quality of life improves significantly in the first year. Yet in 5% of cases Guillain–Barré syndrome (GBS) can lead to death as a result of many complications.
While GBS is a disease of the peripheral nervous system, ALS is a disease of the motor tracks in the central nervous system. Yet, many patients with ALS also recount that their disease started with an infection.
Some investigators have suggested that mechanisms resulting from molecular mimicry between viral proteins and human proteins participate in the pathogenesis of GBS.
Ganglioside GM1 has important physiological properties and impacts neuronal plasticity and repair mechanisms, and the release of neurotrophins in the brain. Because of GM1's close role in neuron repair mechanisms, it has been investigated as a possible drug to slow or even reverse the progression of a wide range of neurodegenerative conditions. Controlled phase II studies have indicated that GM1 can ease the symptoms of Parkinson's disease
Weirdly for the complement immune system GM1 is sometimes identified as a virus fragment.
Alas Ganglioside GM1 is not the sole case of autoimmunity. Many autoimmune diseases, some of them ALS mimics, are caused by autoantibodies. An autoantibody is an antibody produced by the immune system that is directed against one or more of the individual's own proteins.
To complicate the matter, some autoantibodies are needed to maintain tissue and protein homeostasis through adaptive debris clearance. For example autoantibodies against TDP-43 are found lacking in Patients With Amyotrophic Lateral Sclerosis.
Autoantibody tests may be ordered as part of an investigation. ANA is a marker of the autoimmune process – it is positive with a variety of different autoimmune diseases but not specific. Consequently, if an ANA test is positive, it is followed up with other tests.
Plasmapheresis and intravenous immunoglobulins (IVIG) are the two main immunotherapy treatments for GBS. Plasmapheresis attempts to reduce the body's attack on the nervous system by filtering antibodies out of the bloodstream. Similarly, administration of IVIG neutralizes harmful antibodies and inflammation.
This book retraces the main achievements of ALS research over the last 30 years, presents the drugs under clinical trial, as well as ongoing research on future treatments likely to be able stop the disease in a few years and to provide a complete cure in a decade or two.