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Gene therapy for dementia based on VIB research (Flanders)

While uncommon, dementia is not just a matter of age. Worldwide, early-onset Alzheimer’s affects an estimated 3.9 million people under the age of 65, according to a 2021 study published in the scientific journal JAMA Neurology. One of the most common forms of this type of dementia is called ‘frontotemporal dementia’. Characterized by progressive loss of nerve cells in the frontal and temporal lobes, it comes with a life expectancy of 6 to 8 years after the first symptoms appear. However, a discovery by Flanders’ strategic research center for biotech VIB could help change the tide.

Prof. Christine Van Broeckhoven

Innovative gene therapy

Led by University of Antwerp Professor and world-renowned Alzheimer’s expert Christine Van Broeckhoven, previous VIB research helped enable the development of PR006. This experimental therapy is currently being evaluated as a potentially disease-modifying, single-dose gene therapy for individuals living with frontotemporal dementia with GRN mutations (FTD-GRN). Sponsored by American gene therapy company Prevail Therapeutics, the PR006 phase 1/2 trials are enrolling patients in clinical centers across different countries.

Based on research dating back to 2006

Together with her team at VIB and University of Antwerp, Professor Christine Van Broeckhoven has built up solid expertise in molecular genetics of neurodegenerative diseases. In 2006, Van Broeckhoven’s team discovered that mutations in the GRN gene are the basis of a common familial form of frontotemporal dementia. Furthermore, they found that these genetic mutations lead to a shortage of the growth factor progranulin in the brain, leading to the disease.

The team unraveled this mechanism in close collaboration with Michael Hutton, formerly of the Mayo Clinic, and Howard Feldman and Ian Mackenzie from the University of British Columbia. Researchers from the University of Manchester were also involved in the collaboration. At the time, their joint work was published in two papers in the prominent scientific journal Nature.

From insights to treatment

The teams’ findings indicated that strategies aimed at increasing progranulin levels could be useful to prevent or even treat dementia. That’s why VIB reached out to an industrial partner to help turn these insights into a possible treatment for patients. In 2019, VIB entered into a non-exclusive licensing agreement with American company Prevail Therapeutics for the development of gene therapy for individuals living with frontotemporal dementia with GRN mutations (FTD-GRN).

A subsidiary of American pharmaceutical firm Eli Lilly and Company, Prevail Therapeutics has now set out to test the developed gene therapy in phase 1/2 clinical trials in the US and Australia. A new treatment is currently being tested as a potentially disease-modifying, single-dose gene therapy for individuals living with this form of dementia. The therapy’s development is based on previous research by, among others, Prof. Christine Van Broeckhoven and her team at VIB and University of Antwerp (Flanders). PR006 is designed to slow or stop disease progression in FTD-GRN patients by increasing progranulin levels via delivery of a healthy GRN gene into the central nervous system (CNS).

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More info

VIB
Reported by
website VIB, website ALZFORUM
27 January 2022

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