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Neuroprogressive and Dementia Network part of global MSA study success as one of four UK sites
The NHS Research Scotland Neuroprogressive and Dementia Network (NDN) has told of its success in studying the effects of a new drug on rare neurological condition Multi System Atrophy (MSA)
The Queen Elizabeth University Hospital (QEUH) in Glasgow was chosen as one of just four UK sites alongside London to take part in the global landmark study looking into a treatment for slowing the rapidly progressive disease with around 250 people living with it in Scotland at any one time.
MSA causes the brain and nervous system to gradually deteriorate. It affects balance, movement, and automatic functions like breathing and digestion.
Symptoms reflect the progressive loss of function and failure of different types of nerve cells in the brain and spinal cord. These include slowness of movement, tremor, or stiffness; lack of coordination; quivering voice; fainting or light-headedness; and bladder control problems.
The two main types of MSA are MSA-P, a Parkinson’s-like subtype; and MSA-C, a cerebellar subtype.
Utilising the trial results, biotechnology company Alterity Therapeutics has been able to announce positive data from its phase two study.
It saw Alterity Therapeutics’ drug ATH434 compared to placebo participants with early-stage MSA including MSA-C.
The data showed that ATH434 “produced significant improvement on a number of clinical scales” and slowing of disease progression compared to the placebo.
The data further showed “robust efficacy” on a functional rating scale that assesses disability related to the activities of those living with MSA.
On this clinical measure, ATH434 demonstrated 48% slowing of clinical progression at a 50 mg twice daily dosage level which was statistically significant, when compared to placebo.
“Trends of improved motor performance” were observed on the Parkinson’s Plus rating scale — a clinical assessment tool used to evaluate the severity of symptoms in individuals affected by conditions that share characteristics with Parkinson's disease but who also exhibit additional, sometimes more prominent, neurological features.
Overall benefit was also shown at the 50 mg dose level on the Clinical Global Impression of Severity (CGI-S) — a scale which requires the clinician to rate the severity of the patient's illness at the time of assessment, relative to the clinician's past experience with patients who have the same diagnosis.
Principal Investigator, Dr Vicky Marshall of NHS Greater Glasgow and Clyde — a Consultant Neurologist with an interest in movement disorders — said: “We are delighted to have been able to play an important role in demonstrating the potentially very positive impact of ATH434 and are grateful to those who attended our clinic.
“The resulting data has shown that the drug can produce a slowing of MSA’s progression — an encouraging development given there are currently no approved treatments, and one which could lead to accelerated development of ATH434 amid a significant unmet need. These results now need to be replicated in larger phase 3 studies.”
David Stamler, CEO of Alterity Therapeutics said: “We are very grateful for the invaluable contributions of the study participants and the clinical sites who contributed to the study.”
The Neuroprogressive and Dementia Network is funded by the Chief Scientist Office to promote a culture of clinical research in dementia across Scotland and improve recruitment to high-quality studies from both urban and rural areas.
The Network has grown from four centres in Grampian, Greater Glasgow and Clyde, Lothian and Tayside to now cover the whole of mainland Scotland.
Publication date: 17th March 2025
