It has been discovered that Multiple System Atrophy and Parkinson's disease are of the same type. If Parkinson's disease treatment research, which is ahead, is applied, the development of therapeutic drugs may rapidly progress.
Immunotherapy to suppress α-synuclein, which is thought to produce neurotoxins, treatments to protect nerves from oxidative stress caused by α-synuclein, and repurposing drugs initially developed for Parkinson's disease are being explored.
ION464 (also known as BIIB101)
This is an antisense drug currently in clinical trials that inhibits the genetic information for α-synuclein production. A clinical trial involving 24 patients with Multiple System Atrophy, where ION464 is administered intrathecally, is ongoing in Europe. Phase I trials have been conducted.
*Antisense drugs: RNA drugs that bind to and inhibit or suppress the function of RNA that conveys protein synthesis.
NBMI
This is an antioxidant drug that is expected to prevent the aggregation of α-synuclein. Currently, Phase II trials are being conducted with 16 patients with Multiple System Atrophy and progressive supranuclear palsy.
Immunotherapy (Lu AF82422)
This is an antibody treatment that may slow or inhibit the progression of MSA by neutralizing and removing α-synuclein. Phase II trials have been conducted in the US and Japan, and the results were announced on February 5, 2024. No significant beneficial effects were observed.
Rituximab
Rituximab is a drug used to treat rheumatoid diseases. MSA is known for causing neuroinflammation due to oxidative stress and is often associated with Sjögren's syndrome. Phase II trials targeting 50 patients with MAS-C are ongoing in China to explore the anti-inflammatory effects of Rituximab.
Mesenchymal Stem Cell Autotransplantation
Mesenchymal stem cells, which can differentiate into glial cells (the cells that degenerate in Multiple System Atrophy), can be extracted from one’s bone marrow or fat. It is unclear whether intravenous administration can reach and engraft in the brain. Intrathecal injections are being tested at the Mayo Clinic, while intra-arterial administration is being tested in Korea.
*Intrathecal injection: Since the brain has a blood-brain barrier that prevents drug delivery, directly injecting drugs into the cerebrospinal fluid may be effective.
Coenzyme Q10
It is assumed that some people have a genetic predisposition to develop Multiple System Atrophy. Among them, an anomaly in the gene that synthesizes coenzyme Q10 was discovered, leading to clinical trials using coenzyme Q10. Compared to the placebo group, approximately a 30% reduction in disease progression was observed.
*Since the intake amount is about 10 times the usual amount, future research needs to focus on efficient absorption to prevent side effects.