The International Congress of Parkinson’s Disease and Movement Disorders took place this year from August 27 to 31. During the Congress, researchers and other stakeholders from across the globe convened to discuss new research, treatments, and care practices. According to a news release, biotechnology company Alterity Therapeutics (“Alterity”) presented during the Congress about a natural history study that the company ran on multiple system atrophy (MSA).

The Facts on Multiple System Atrophy (MSA)

Multiple system atrophy is a rare neurodegenerative disorder that affects the autonomic system. The autonomic system controls things like motor skills, balance and coordination, blood pressure, and heart rate. MSA may be considered parkinsonian or cerebellar depending on its symptoms. It typically affects people aged 50-60 or older; this disorder is often fatal within around 10 years of symptom manifestation. Doctors do not know exactly what causes MSA, although some research suggests a buildup of a protein called alpha-synuclein. There is no cure for this disorder. Treatment options may include pacemakers, blood pressure medication, or a variety of other therapies.

Symptoms of Parkinsonian MSA may include:

• Tremors
• Issues with balance and coordination
• Difficult or slowed movement
• Muscle stiffness
• A soft voice
• Problems with bending the arms or legs

People with the cerebellar form may experience:

• Poor movement and balance
• Slurred or slowed speech
• Dysphagia (difficulty swallowing)
• Visual impairments such as blurred or double vision

Finally, there are a number of more general symptoms related to MSA. These can include:

• Constipation
• Incontinence
• Sexual dysfunction
• Postural orthostatic hypotension
• Heat intolerance
• Reduced sweating
• Abnormal breathing at night
• Heartbeat irregularities
• Difficulty controlling emotions
• Color changes in the hands and feet

Results from the BioMUSE Study

In 2020, Alterity announced that it was launching the BioMUSE study, a natural history study that aimed to track how MSA progresses. The study encompassed people who were newly diagnosed or in early stages of the disease, as well as those in later stages. Data from the BioMUSE study were also used to shape a Phase 2 study from Alterity which evaluated ATH434 as a potential therapeutic option. Learn more about natural history studies and how they contribute to a deeper understanding of disease.

In particular, the BioMUSE study focused on identifying potential biomarkers which could be used to aid earlier disease diagnosis. The two poster presentations given at the Congress focused on these findings. First, the poster on using a multimodal approach to diagnose MSA discussed the need for multiple diagnostic biomarkers. It centered around three patients for whom diagnostic was ineffective; researchers used a variety of MRIs and fluid biomarkers to confirm diagnosis. As such, using a multimodal approach and considering multiple biomarkers can increase the probability of diagnosis in early-stage MSA. The secondary presentation evaluated 13 individuals with early-stage MSA. Research found that myoinositol and N-acetylaspartate were MSA biomarkers; increases in myosinositol levels, and decreases in N-acetylaspartate levels, in the brain correlated with multiple system atrophy development.