CORDIS
EU research results

CORDIS

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Development of new therapy for rare motor neuron diseases

Project information

Grant agreement ID: 825713

Status

Ongoing project

  • Start date

    1 November 2018

  • End date

    30 April 2020

Funded under:

H2020-EU.1.1.

  • Overall budget:

    € 150 000

  • EU contribution

    € 150 000

Hosted by:

INSTITUT DU CERVEAU ET DE LA MOELLE EPINIERE

France

Objective

Hereditary spastic paraplegias are a group of rare neurodegenerative diseases due to the degeneration of the long axons of cortical motor neurons. Some complex forms of the disease are also characterized by cognitive impairment. Currently, the only available treatments are symptomatic, act on the motor symptoms and do not prevent or delay the disease progression. There is therefore a strong demand of clinicians as well as of patients and their families for the development of any treatment that could delay the onset and/or the progression of the disease. We have developed a genetic mouse model of a complex form of hereditary spastic paraplegia, the SPG11 form that is extremely invalidating as the patients need to be taken in charge by families or specialized institutions. During the ERC Starting Grant program ER-HSP (GA – 311149), we have shown that this model presents both motor and cognitive impairments. We also identified accumulation of some lipid species in lysosomes as a putative therapeutic target to prevent neurodegeneration in this model. With the proof of concept grant, we plan to perform a preclinical trial to identify a possible treatment for this pathology. We have identified two different strategies to decrease the accumulation of lipids in neurons. In vitro assays have shown that both strategies can decrease accumulation of lipids in lysosomes. Importantly, both strategies can be translated into clinical trial in collaboration with industrial partners and represent potential new orphan drugs.
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Host institution

INSTITUT DU CERVEAU ET DE LA MOELLE EPINIERE

Address

Boulevard De L'Hopital 47
75013 Paris

France

Activity type

Research Organisations

EU Contribution

€ 150 000

Beneficiaries (1)

INSTITUT DU CERVEAU ET DE LA MOELLE EPINIERE

France

EU Contribution

€ 150 000

Project information

Grant agreement ID: 825713

Status

Ongoing project

  • Start date

    1 November 2018

  • End date

    30 April 2020

Funded under:

H2020-EU.1.1.

  • Overall budget:

    € 150 000

  • EU contribution

    € 150 000

Hosted by:

INSTITUT DU CERVEAU ET DE LA MOELLE EPINIERE

France