Please Tell Me About the Latest Treatments for Multiple System Atrophy.

I’m willing to try any treatment that might help. I often faint due to dizziness and am currently taking medication to raise my blood pressure. What treatments are available for Multiple System Atrophy?

S.K., Tokyo

The pathology of Multiple System Atrophy is becoming clearer, and various clinical trials have begun.

Gene therapy to suppress the production of α-synuclein and treatments using Coenzyme Q10, which has antioxidant effects on nerves, have shown promising results. However, these treatments are still in clinical trial stages, and there is currently no widely available cure that directly addresses the root cause of the disease.

The distressing symptoms of Multiple System Atrophy can be managed to some extent through symptomatic treatment with medications. Acupuncture treatment that activates brain function, combined with rehabilitation immediately after acupuncture, has also proven effective. These treatments can be used alongside medication and often lead to improvements in SARA scores.
※SARA - A globally used evaluation method for Multiple System Atrophy.

Acupuncturist Kana Yoshiike

New Treatments for Multiple System Atrophy

Various clinical and experimental trials have begun worldwide.

As the causes of Multiple System Atrophy are gradually being uncovered, a wide variety of treatments are under development. However, since none of the drugs currently in research are curative, it is crucial to focus on preventing further deterioration until new medications are approved.

Treatments Under Clinical Trial

It has been discovered that Multiple System Atrophy (MSA) and Parkinson’s disease are similar types of diseases. If research from Parkinson’s disease treatments is repurposed, the development of new medications may progress rapidly.

Research includes immunotherapy to suppress α-synuclein, which is believed to produce neurotoxins, treatments to protect nerves from oxidative damage caused by α-synuclein, and repurposing medications originally developed for Parkinson’s disease, which shares the same cause.

ION464 (also known as BIIB101)

An antisense drug under clinical trial that inhibits genetic information responsible for α-synuclein production. A trial administering ION464 intrathecally to 24 MSA patients is ongoing in Europe. Currently, Phase I trials have been conducted.
※ Antisense drugs: RNA that binds to and inhibits the function of RNA that conveys protein synthesis instructions.

NBMI

A medication with antioxidant properties, it is expected to prevent the aggregation of α-synuclein. Currently, a Phase II trial is being conducted on 16 patients with MSA and Progressive Supranuclear Palsy (PSP).

Immunotherapy (Lu AF82422)

An antibody therapy that neutralizes and removes α-synuclein, potentially slowing and suppressing the progression of MSA. Phase II trials have been conducted in the US and Japan, with results announced on February 5, 2024, showing no significant effectiveness.

Rituximab

Rituximab, a drug used to treat rheumatoid diseases, is under trial for MSA due to its potential anti-inflammatory effects. MSA is known to involve oxidative stress-induced neuroinflammation and has a high association with Sjogren’s syndrome. A Phase II trial involving 50 MSA-C patients is ongoing in China.

Autologous Mesenchymal Stem Cell Transplantation

Mesenchymal stem cells, which can differentiate into glial cells (affected in MSA), can be extracted from a patient’s bone marrow or fat. However, it is unclear if they reach the brain and integrate when administered intravenously. Trials are being conducted via intrathecal injection at Mayo Clinic and intra-arterial administration in South Korea.
※ Intrathecal injection: Administering medication directly into cerebrospinal fluid to bypass the blood-brain barrier and achieve effectiveness.

Coenzyme Q10

It has been hypothesized that certain genetic predispositions may increase susceptibility to MSA. Mutations in genes involved in Coenzyme Q10 synthesis have been identified, and clinical trials using Coenzyme Q10 have been conducted. Results showed approximately 30% progression suppression compared to the placebo group.
※ The dosage required is about ten times the usual amount, so future research will focus on improving absorption efficiency to prevent side effects.