World Pharma Conference - Philadelphia

Progress in Parkinson's disease (PD) research and therapeutic development is hindered by the availability of reagents including quality antibodies, robust assays and viral vectors encoding target genes of interest. The limitations are a result of technical hurdles, production and distribution costs, as well as licensing restrictions that prohibit free distribution and inhibit further scientific progress. We hypothesize that directly sponsoring the generation of reagents including antibodies, assays and viral vectors will enable increased sharing and distribution of these much needed tools to accelerate Parkinson's research.

The Michael J. Fox Foundation for Parkinson's Research (MJFF) generated antibodies against the PD-associated LRRK2 protein that were found to be superior to other available antibodies in a number of applications (http://www.pdonlineresearch.org/discussions/lrrk2-antibodies).  These are now widely available to the research community.  With this success, MJFF is generating and characterizing additional antibodies critical for studies of PD: antibodies against phosphorylated forms of LRRK2, an antibody against the PINK1 protein and antibodies that recognize either phosphorylated or aggregated forms of alpha-synuclein.

MJFF is also developing ELISA methods to quantify levels of LRRK2 protein in biological samples.  This assay will enable investigators to further characterize tissue and other biological samples for changes in LRRK2 protein levels in human tissue and to correlate these changes with disease progression.

Finally, MJFF is generating viral vectors encoding several PD targets of interest (including alpha-synuclein and LRRK2). These tools will help facilitate studies in animal models seeking to understand a biological target's relevance to PD.

MJFF is confident that the availability of these reagents to the entire research community will facilitate understanding of PD and accelerate therapeutic development. Further, MJFF can replicate this strategy for generating additional reagents where decreased barriers to access and limited resources hinder progress in PD research efforts.

Authors: Sonal S. Das, Dario R. Alessi, Mark Cookson,  C. Johannes Gloeckner, Andrew B. West, Jeremy Nichols, Ito Genta, Takeshi Iwatsubo, Paul Davies, Alexandra Beilina, Elizabeth Doggett, Niketa Sheth, Mark Frasier, Kuldip Dave, Audrey Dufour, Alison Urkowitz, Shehan, De Silva, Jamie Eberling, Peggy Taylor, Ashley Carleton, Todd Sherer, Brian Fiske