Advancing MSA Research: A Conversation with Dr. Peter Barbuti
As global efforts to accelerate Multiple System Atrophy (MSA) research intensify in 2026, scientific leadership has never been more critical. Dr. Peter Barbuti, Director of Research (Interim) for Defeat MSA Alliance (USA) and the MSA United Research Consortium, brings more than 15 years of expertise in alpha-synuclein research — the protein central to MSA and related neurodegenerative diseases.
In a recent recorded interview, Dr. Barbuti shared insights into his scientific background, the current challenges in MSA research, and the strategic direction guiding the consortium’s eight newly funded research projects.
A Career Focused on Alpha-Synuclein
Dr. Barbuti earned his PhD in neuroscience from the University of Bristol in the United Kingdom and has spent over a decade and a half researching alpha-synuclein — the protein thought to be central to MSA, Parkinson’s disease (PD), Lewy body dementia (LBD/DLB), and related disorders.
His research career has taken him from the University of Luxembourg to Columbia University in New York, where his work helped identify distinct lipid signatures in postmortem brain tissue capable of differentiating Parkinson’s disease from MSA. This discovery represents a meaningful step toward addressing one of the most pressing challenges in MSA: misdiagnosis.
“MSA is often initially misdiagnosed as Parkinson’s disease,” Dr. Barbuti explains. “Reducing that diagnostic delay is critical, because the earlier we understand what we are treating, the better we can support patients.”
The Challenge of MSA: Misdiagnosis and Rarity
MSA remains a rare, rapidly progressive neurodegenerative disease with no FDA-approved disease-modifying therapy. Dr. Barbuti notes that as many as 40% of patients are initially misdiagnosed — often with Parkinson’s disease — due to overlapping symptoms.
Complicating matters further, MSA presents in two primary subtypes:
- MSA-P (Parkinsonian subtype), more common in Western populations
- MSA-C (Cerebellar subtype), more frequently observed in East Asia
These distinctions suggest possible genetic or biological differences that require global collaboration to fully understand.
“The rarity of MSA makes international cooperation essential,” he says. “No single country can solve this alone.”
Translating Science Into Strategy
As Director of Research (Interim), Dr. Barbuti’s role extends beyond laboratory science. He serves as a bridge between researchers, board leadership, patient communities, and industry partners.
His responsibilities include:
- Setting short-, mid-, and long-term research priorities and organizing special projects
- Overseeing and supporting the eight funded international research projects
- Ensuring transparency in how donor funds are invested
- Advocating for focused, high-impact research calls
One area he is particularly passionate about is translational science — moving discoveries from laboratory models toward practical clinical application.
“There has been criticism in rare disease research about funding work that never reaches patients,” he acknowledges. “Our focus must be on translational progress — research that can realistically move toward diagnostics and treatment within meaningful timeframes.”
Biomarkers: The Key to the Future
When asked which area of research gives him the most hope, Dr. Barbuti does not hesitate: biomarkers.
Biomarkers — measurable biological indicators which may be found in blood, skin, or other accessible samples — have the potential to dramatically reduce misdiagnosis rates and enable earlier intervention.
“If we can identify reliable, non-invasive biomarkers, we can distinguish MSA from Parkinson’s disease sooner and more accurately,” he explains. “That changes everything — for clinical care, for trial enrollment, and for patient quality of life.”
Biomarkers are also essential for measuring whether emerging treatments are working during clinical trials.
Understanding the Science: Alpha-Synuclein Dysfunction
At the biological level, MSA is thought to be driven by the dysfunction of alpha-synuclein, a protein that normally plays a role in neuronal function. In MSA, this protein misfolds, aggregates, and accumulates within cells — particularly in oligodendrocytes — forming toxic clumps known as glial cytoplasmic inclusions.
Dr. Barbuti compares the process to “a ball of elastic bands that has lost its structure and usefulness.” Once misfolded, the protein becomes difficult for the brain to break down or recycle.
Research efforts currently focus on:
- Preventing alpha-synuclein misfolding
- Reducing protein accumulation
- Understanding early cellular triggers
- Exploring possible genetic and molecular modifiers
This foundational research supports both therapeutic development and improved diagnostic tools.
The Importance of Clinical Trials
While laboratory models are essential for understanding disease mechanisms, Dr. Barbuti emphasizes that clinical trials remain the ultimate proving ground for treatment development.
“Cell cultures and animal models are informative, but they are not human,” he explains. “Clinical trials are where we determine whether a therapy truly makes a difference.”
MSA trials face unique challenges:
- Small patient populations
- Disease differences and heterogeneity
- Misdiagnosis at enrollment
- Difficulty defining measurable endpoints
Despite these hurdles, Dr. Barbuti remains optimistic.
“We are making progress. Standing still isn’t an option.”
A Global Research Effort
Under Dr. Barbuti’s leadership, the MSA United Research Consortium continues to fund research across multiple countries, strategically investing in projects that align with key priorities: biomarkers, disease mechanisms, and translational therapeutics.
The consortium’s model of pooled international funding allows resources to be allocated efficiently while avoiding duplication of effort.
“Our goal is clear,” he says. “The right diagnosis. The right treatment. At the right time.”
Looking Ahead
As research efforts accelerate in 2026, Defeat MSA Alliance (USA) and the MSA United Research Consortium remain committed to advancing scientific understanding while maintaining transparency and accountability to patients and donors.
Dr. Barbuti’s message to the community is one of measured optimism:
“Research is moving forward. The tools we have today are more advanced than ever. With sustained focus and collaboration, we can make meaningful progress.”
