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A new era of discovery for Parkinson’s Disease

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Alessio Travaglia, director of neuroscience at the Foundation for the National Institutes of Health (FNIH), on the outlook for new Parkinson’s treatments.

Parkinson’s disease (PD) is a neurological disorder that affects a person’s ability to move. When certain cells in the brain start to die, the process causes a decrease in a chemical called dopamine.

As a result, individuals with Parkinson’s disease may experience difficulty with everyday activities, such as walking, talking and writing, along with common symptoms, like tremors, stiffness, slowness of movement and difficulty with balance.

Unfortunately, there is no cure for Parkinson’s disease, and the number of patients is rising. The number of Parkinson’s patients is expected to increase to 1.2 million in the U.S. alone by 2030, posing a growing threat to public health.

The disease costs the US$52 billion per year in direct and indirect costs. Although the hallmarks of Parkinson’s disease are well known, and there are several genetic, epigenetic, and environmental factors associated with PD, the underlying causes are still unknown.

Breakthroughs are needed to improve treatment and quality of life for patients, but no disease-modifying drugs have been approved for Parkinson’s disease despite extensive research.

Drug discovery is a complex and time-consuming process involving the identification of new compounds that can be used to treat a particular disease.

The two main hurdles in developing new drugs are identification of new drug targets (specific molecules or proteins involved in the disease process), and the development of novel biomarkers.

Biomarkers are measurable substances or indicators found in the body, such as proteins or other molecules, that can reveal information about a person’s health or disease status.

They are critical in drug discovery, and they can help doctors and researchers better understand a person’s health and make more informed decisions about their care.

The Accelerating Medicines Partnership Program Parkinson’s Disease (AMP PD) is a public-private partnership managed by the Foundation for the National Institutes of Health, a US-based nonprofit organisation.

The programme seeks to identify and validate biomarkers for diagnosis, disease subtyping, prognosis and monitoring progression. This partnership will provide new tools for use in clinical trials and will speed up the development of novel therapies.

Public-private partnerships are essential in drug discovery and development, combining the strengths and resources of both sectors to accelerate research and development, increase efficiency and reduce costs.

Indeed, to accelerate drug discovery efforts, it is crucial to standardise and combine data generated from various sources.

Before AMP PD, data from ongoing Parkinson’s disease studies were isolated in silos, limiting the ability to conduct the cross-study analyses necessary to gain a deeper understanding the disease.

By standardising data and making it easy to share and analyse across different platforms, researchers can more easily identify potential drug targets, biomarkers and therapeutic approaches.

The AMP PD project uses several approaches to achieve its goals. The first is to collect data on biomarkers from multiple sources and ensure that the data collected are standardized.

The second is to use existing clinical, imaging and genetic data to perform standardised tests on thousands of biological samples.

The project will also pursue large-scale biomarker discovery using new and different techniques, working to identify new targets and disease subtypes, track and predict disease progression, and identify biomarkers of Parkinson’s progression that could potentially be targeted by therapies.

Overall, the AMP PD research plan seeks to better understand Parkinson’s disease by studying the molecular and clinical characteristics of patients, ultimately finding new targets, disease subtypes, and predictive markers for disease progression and prognosis.

This partnership between public and private organisations will allow for large-scale analyses of biomarkers and bring PD biomarker research into a new era. Without such a partnership, individual institutions or companies would find it challenging to manage the funds and resources required for this effort.

Dr Alessio Travaglia is a neuroscientist with more than 15 years of experience in basic and translational neuroscience, in settings including academia, non-profit, management consulting, and venture philanthropy. He currently works as director of neuroscience at the Foundation for the National Institutes of Health (FNIH). In this role, he leads the FNIH’s neuroscience programmes, facilitates the advancement and execution of innovative public-private partnerships, and collaborates with government, industry, academia, patient-advocacy groups, and private sector organizations.

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