MJFF Lysosomal Biomarkers Program

Closing Date: 17/01/2023

Grants to develop, optimise and validate biomarkers related to lysosomal function, protein clearance/autophagy and lipid homeostasis.

Established in 2000, the Michael J Fox Foundation (MJFF) was set up to find a cure for Parkinson’s disease (PD) and ensure the development of improved therapies.

The goal of the MJFF Lysosomal Biomarkers Program is to develop, optimise and validate biomarkers related to lysosomal function, protein clearance/autophagy and lipid homeostasis.

Funding will support projects to:

  • Develop, optimise or validate molecular bioassays (eg mass-spectrometry or immunoassay approaches) for autophagy or lysosomal analytes.
  • Investigate imaging approaches in the brain or other areas (eg lipidated microtubule-associated protein 1A/1B-light chain 3 (LC3), autophagosome number, lipofuscin, lysosomal content, glucocerebrosidase (GCase) tracer etc).
  • Measure functional endpoints, dynamic measures, in vivo activity or lysosomal flux (eg heavy labelling approaches).
  • Analyse existing datasets (including non-PD human data) to identify molecular measures of lysosomal function in normal and disease states towards identification of patient enrichment markers for lysosomal targeted therapies.

This scheme welcomes biomarkers to specifically support lysosomal targeted therapies (eg TMEM175, TRPML1, GBA etc) and also those geared towards quantification of lysosomal dysfunction or lysosomal dysfunction states more broadly.

Applications spanning all stages of the biomarker pipeline, including development, optimisation and validation, will be considered. MJFF encourages applicants to be creative and think outside the box when considering how to address challenges surrounding measurements of this pathway (eg evaluation of lysosomal versus secreted GCase, methods to measure protein turnover, lack of readily available tools/antibodies etc).

Proposals will be funded related to:

  • Markers of autophagy (including measures of upstream signaling cascade and more tractable targets like p62, cathepsins, LAMPs etc).
  • Other lysosomal pathway markers/interactors (including lipid markers and GCase interactors).
  • Due to the lack of available antibodies to measure many relevant analytes, antibody generation may be considered with relevant justification for why new development is necessary for the proposed assay work. If proposing novel antibody generation to support assay development, applicants must consider long-term access for relevant assay work (eg use of monoclonals, path to commercialisation and scalability, any IP considerations etc). Working with MJFF to deposit the resulting antibody in an MJFF-designated repository for community access is a requirement of the scheme.

When considering submissions, MJFF will prioritise those that:

  • Have potential to directly inform ongoing or upcoming clinical trials.
  • Directly inform precision medicine approaches for patient selection and stratification.
  • Include clear indication of how the proposed biomarker will be used to inform PD diagnosis, prognosis, monitoring, prediction, susceptibility and/or pharmacodynamic response.

Clear translational value and path to use of the assay in the clinic should be described if fibroblasts or iPSCs are used.

Applicants may propose studies designed to evaluate biomarkers in human biospecimens. Investigators are encouraged to leverage existing tissue and biosample resources if possible. New biosample collection should be limited to studies where clinical biosamples are unavailable for the intended purpose. Studies requesting access to biosamples available through MJFF-sponsored biospecimen and cell line collections are eligible to apply, and in such cases, access to samples will be reviewed in parallel to funding requests by the committees overseeing the biospecimen collection(s) requested.

Funding body Michael J Fox Foundation for Parkinson’s Research (MJFF)
Maximum value 350,000 USD
Reference ID S24560
Category Medical Research
Biotechnology and Biology
Fund or call Fund