Alterity Therapeutics Presents New Data on Multiple System Atrophy, a Rare Parkinsonian Disorder - Seite 2

Two poster presentations were given at the MDS Congress.

The poster entitled, “A multimodal approach for diagnosis of early Multiple System Atrophy” was presented by Dr. Claassen. The analysis describes three clinically probable MSA patients with divergent MRI and fluid biomarker data, supporting the use of biomarkers to improve diagnostic accuracy in early MSA. The presented cases demonstrate that no single biomarker can be relied upon to aid in the diagnosis of early MSA. In addition, divergent clinical and biomarker findings in this case series suggests a multimodal clinical-biomarker approach is required for accurate diagnosis of clinically probable or early MSA. These examples support application of clinical and quantitative biomarkers in clinical trials evaluating disease-modifying treatments for early MSA.

The poster entitled, “Preliminary evidence for evolution of myoinositol and N-acetylaspartate as biomarkers of disease severity in early-stage Multiple System Atrophy” was presented by Paula Trujillo Diaz, PhD, Research Assistant Professor, Department of Neurology, Vanderbilt University Medical Center. The study assessed 13 early-stage MSA patients (motor symptoms ≤ 3 yrs) with diagnosis based on clinical parameters, fluid biomarkers, and quantitative MRI for iron deposition. The investigators then applied a non-invasive MRI technique known as magnetic resonance spectroscopy (MRS) that allows metabolite quantification in the brain, including myoinositol (mI; a marker of gliosis) and N-acetylaspartate (NAA; a marker of neuronal integrity). The results suggest that an increase in mI/water and decrease in NAA/water decrease over one-year in patients with MSA is consistent with MSA pathology. The findings suggest that metabolite concentration by MRS may be useful biomarkers for assessing clinical measures of disease severity and treatment response in MSA.

The poster presentations can be accessed on the Published Scientific Research section of the Alterity website here.

About bioMUSE

Biomarkers of progression in Multiple System Atrophy (bioMUSE) is a natural history study that aims to track the progression of individuals with MSA, a parkinsonian disorder without approved therapy.  The study is being conducted in collaboration with Vanderbilt University Medical Center in the U.S. under the direction of Daniel Claassen, M.D., M.S., Professor of Neurology and Principal Investigator. Natural history studies are important for characterizing disease progression in selected patient populations. The study has provided rich data for optimizing the design of Alterity’s randomized ATH434-201 Phase 2 clinical trial and enrolled approximately 20 individuals with clinically probable or clinically established MSA. BioMUSE continues to provide vital information on early stage MSA patients, informs the selection of biomarkers suitable to evaluate target engagement and preliminary efficacy, and delivers clinical data to characterize disease progression in a patient population that mirrors those currently enrolling in the Phase 2 clinical trial.