Myrtelle is investigating potential gene therapies for MSA, a neurological condition in which some parts of the brain gradually shrink and deteriorate.
Myrtelle’s program in Multiple System Atrophy (MSA) uses a nonpathogenic recombinant adeno-associated virus (rAAV) to deliver gene silencing microRNA (miRNA) directly to the central nervous system (CNS), targeting oligodendrocytes. The miRNA is intended to reduce expression of alpha-synuclein in oligodendrocytes, which is the main constituent of the glial cytoplasmic inclusions (GCIs) found in oligodendrocytes that are the hallmark of MSA pathology. MSA is characterized by progressive autonomic failure, parkinsonism, cerebellar ataxia and pyramidal features leading to death a few years after symptom onset. There are no effective therapies that can halt or reverse the progression of MSA.
Multiple System Atrophy (MSA) predominantly targets adults, manifesting as a progressive neurological disorder. It impairs essential motor functions, leading to difficulties in movement, balance, and coordination. Additionally, it affects speech clarity and the ability to maintain stable blood pressure. Notably, MSA shares numerous symptoms with Parkinson’s disease, often making early and accurate diagnosis challenging.
While the exact cause of MSA remains elusive, it is closely linked to abnormal accumulations of alpha-synuclein protein. These accumulations form clumps in oligodendrocytes, the brain cells dedicated to producing myelin. Myelin is a critical component that insulates neurons and facilitates efficient communication between them. The disruption of myelin production due to these clumps significantly impacts brain function, contributing to the varied symptoms observed in MSA.
Multiple System Atrophy (MSA) is a progressive neurodegenerative disorder characterized by a combination of symptoms that affect both the autonomic nervous system (which controls involuntary actions like blood pressure and breathing) and movement. There are two types of MSA, defined by their most prominent symptoms: MSA-P, with parkinsonism symptoms, and MSA-C, with cerebellar symptoms. Common signs include poor coordination, speech difficulties, and a decline in motor function.
Diagnosing MSA can be challenging, as its symptoms overlap with those of other neurological disorders. The diagnosis is usually made based on medical history, clinical examination, and the exclusion of other conditions. Neuroimaging tests, like MRI, can provide supportive evidence, and autonomic testing can help assess the function of the autonomic nervous system. It’s important to consult a neurologist experienced in movement disorders for an accurate diagnosis.
Currently, there is no cure for MSA, and treatments focus on managing symptoms and improving quality of life. This can include medications to help with movement problems, speech therapy, physical therapy, and measures to manage autonomic symptoms like blood pressure fluctuations. As the disease progresses, more supportive care may be needed, including assistance with daily activities and possibly the use of mobility aids.
While there is currently no cure for MSA, ongoing research is dedicated to understanding the disease better and finding effective treatments. This includes studies on the underlying mechanisms of the disease, clinical trials testing new medications, and research into stem cell therapy and neuroprotective strategies. Participation in clinical trials may offer access to new therapies and contribute to the advancement of knowledge about MSA. Patients and families are encouraged to consult with their healthcare providers about the possibility of joining clinical trials or other research programs.