Validation of diagnostic and prognostic biomarkers for Parkinson disease

Project Information

Biomarkers for PD

Grant agreement ID: 697790

DOI

10.3030/697790

Project closed

EC signature date 19 August 2015

Start date 1 September 2015

End date 29 February 2016

Funded under

• SOCIETAL CHALLENGES - Health, demographic change and well-being

Total cost

€ 71 429,00

EU contribution

€ 50 000,00

Coordinated by

BIOARCTIC AB
Sweden

Periodic Reporting for period 1 - Biomarkers for PD (Validation of diagnostic and prognostic biomarkers for Parkinson disease)

Reporting period: 2015-09-01 to 2016-02-29

Summary of the context and overall objectives of the project

Parkinson’s disease is one of the most prevalent neurodegenerative disorders and a disease with a significant unmet medical need. World-wide, more than 4.6 million individuals over the age of 50 suffer from Parkinson’s disease and it is estimated that the number will more than double by 2030. Today, there are no reliable biochemical biomarkers for Parkinson’s disease and the disease is often difficult to diagnose. The differential diagnosis of Parkinson’s disease is based on clinical features and the golden standard still remains neuropathological confirmation. Misclassification, especially in early Parkinson’s disease, occurs frequently. High sensitivity and specificity can only be obtained at specialized centers, and after several years of follow-up. To be able to modify or halt the disease progression it is beneficial to initiate a disease modifying treatment at an earlier stage than what is possible today. The clinical validation of a sensitive and specific biomarker that also could mirror the treatment effect would make an enormous advantage and need to be developed in parallel with the development of new disease modifying therapeutics.

In this project we have:
• Acquired Human CSF from patients with Parkinson’s disease and healthy controls
• Conducted Biochemical biomarker assay development for Parkinson’s disease
• Conducted a pilot study on clinical cohorts including patients with Parkinson’s disease and healthy controls
• Evaluated the business opportunities and further development path for Parkinson’s disease biomarkers

Work performed from the beginning of the project to the end of the period covered by the report and main results achieved so far

We have successfully established a sensitive and specific biochemical biomarker assay for cerebrospinal fluid analyses and measurements of soluble oligomer/protofibril forms of alpha-synuclein, the presumed neurotoxic forms of alpha-synuclein. This assay has the potential of becoming the first biomarker assay that can reflect the underlying pathophysiology of disease. In a pilot study on clinical cohorts including patients with Parkinson’s disease and healthy controls, ability to measuring α-synuclein oligomer/protofibril in human cerebrospinal fluid from both Parkinson’s disease patients and healthy control has been demonstrated.
Market research in the form of in depth interviews with KOL’s have confirmed the most important potential customer, business opportunities, barriers and commercial value for a new biochemical biomarker in Parkinson’s disease.

Progress beyond the state of the art and expected potential impact (including the socio-economic impact and the wider societal implications of the project so far)

Parkinson’s disease is a significant burden on society and no effective treatment exists targeting the underlying process of the disease. By enabling early and more specific diagnosis of the disease, patients can be given treatment earlier thereby improving quality of life, symptom free time and the cost for society will also be lower. The development of the biomarker assay would also facilitate the development of effective disease modifying treatments benefiting all patients suffering from Parkinson’s disease.

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