Multiple System Atrophy (MSA)
About
MSA is an adult-onset progressive neurodegenerative disease. The core pathological marker is the α-synuclein positive inclusion bodies (GCI) in the cytosol of oligodendrocytes. The cause is unknown, but it is currently believed that it may involve: abnormal uptake and misfolding of α-synuclein by oligodendrocytes after being released from neurons, activation of neuroinflammation, and secondary neuronal degeneration caused by loss of myelin basic protein. Core dilemmas: ①α-synuclein targeted immunotherapy failed in the Parkinson's Disease Phase II trial, and related research and development in the MSA field is seriously lagging behind;② The initial response rate of MSA-P to levodopa is only 30-40%, which quickly fails within 2-3 years.
Features & Symptoms
- Involvement of the autonomic nervous system
- Parkinson's tremor
- Postural hypotension, urinary incontinence
- dysphagia
Treatment Options
| Treatment | Description | Stage | Price Range |
|---|---|---|---|
| α-synuclein targeted immunotherapy (monoclonal antibody) | Monoclonal antibody therapy targeting the core pathology of MSA-α-synuclein inclusion bodies in oligodendrocytes. Prasinezumab and cinpanemab were originally developed in Parkinson's disease but the Phase II trial failed to reach the primary endpoint. The α-syn targeting strategy for MSA is currently being redesigned. | Phase II | $200K - $550K/年 |
| Stem cell nerve repair + autonomic nerve regulation joint program | Pluripotent stem cells differentiate into specific neurons/oligodendrocytes to repair damaged neural circuits, combined with autonomic nerve function regulation: intrathecal injection of bone marrow mesenchymal stem cells; autonomic nerve regulation to manage postural hypotension; bladder function regulation to improve urinary incontinence. | Phase I/II | $400K - $1M/疗程 |
| Exercise symptom management + comprehensive support for quality of life optimization | A comprehensive plan for symptomatic management of MSA: levodopa/carbidopa can be used in patients with MSA-P; physical therapy to maintain balance and gait; speech therapy to improve dysarthria and dysphagia; nutritional support to deal with dysphagia and weight loss. | 临床应用 | $120K - $350K/年 |
Prognosis
Death 5-10 years after onset
Additional Notes
Notes: Management of orthostatic hypotension: One of the most disabling symptoms of MSA, it is recommended to raise the head of the bed by 30°, wear elastic socks, increase salt and water intake. Fludrocortisone and midodrine can improve symptoms; Swallow assessment: Swallow function assessment is required immediately after diagnosis, and PEG nutritional support should be completed before severe dysphagia; Urinary management: Urinary incontinence and dysuria are common, and urological evaluation is required to eliminate neurogenic bladder; Differential diagnosis: MSA is often misdiagnosed as Parkinson's disease, and MSA should be considered when levodopa is ineffective; Sleep disorders: Rapid eye movement sleep behavior disorder (RBD) is common, and attention should be paid to night protection to prevent injuries from falling into the bed. Exclusive service commitment: The platform will allocate a dedicated medical team to each patient to assist in dismantling and properly deploying the following precautions throughout the process. When cross-state and cross-border diagnosis and treatment projects or drugs are involved, the platform will make overall arrangements for the entire docking; for disabled and semi-disabled patient groups, the platform will provide rush-free alternative solutions (including remote consultation, door-to-door sampling, direct drug delivery in the cold chain, etc.), to ensure that patients can get the same high-quality diagnosis and treatment resources without having to travel in person.; Study phase: Phase I/II clinical. Prognosis: Death 5-10 years after onset.
Frequently Asked Questions
What is Multiple System Atrophy (MSA)?
MSA is an adult-onset progressive neurodegenerative disease. The core pathological marker is the α-synuclein positive inclusion bodies (GCI) in the cytosol of oligodendrocytes. The cause is unknown, but it is currently believed that it may involve: abnormal uptake and misfolding of α-synuclein by oligodendrocytes after being released from neurons, activation of neuroinflammation, and secondary neuronal degeneration caused by loss of myelin basic protein. Core dilemmas: ①α-synuclein targeted immunotherapy failed in the Parkinson's Disease Phase II trial, and related research and development in the MSA field is seriously lagging behind;② The initial response rate of MSA-P to levodopa is only 30-40%, which quickly fails within 2-3 years.
What are the symptoms of Multiple System Atrophy (MSA)?
Involvement of the autonomic nervous system,Parkinson's tremor,Postural hypotension, urinary incontinence,dysphagia
How is Multiple System Atrophy (MSA) treated?
α-synuclein targeted immunotherapy (monoclonal antibody): Monoclonal antibody therapy targeting the core pathology of MSA-α-synuclein inclusion bodies in oligodendrocytes. Prasinezumab and cinpanemab were originally developed in Parkinson's disease but the Phase II trial failed to reach the primary endpoint. The α-syn targeting strategy for MSA is currently being redesigned.; Stem cell nerve repair + autonomic nerve regulation joint program: Pluripotent stem cells differentiate into specific neurons/oligodendrocytes to repair damaged neural circuits, combined with autonomic nerve function regulation: intrathecal injection of bone marrow mesenchymal stem cells; autonomic nerve regulation to manage postural hypotension; bladder function regulation to improve urinary incontinence.; Exercise symptom management + comprehensive support for quality of life optimization: A comprehensive plan for symptomatic management of MSA: levodopa/carbidopa can be used in patients with MSA-P; physical therapy to maintain balance and gait; speech therapy to improve dysarthria and dysphagia; nutritional support to deal with dysphagia and weight loss.
What is the prognosis for Multiple System Atrophy (MSA)?
Death 5-10 years after onset