The Michael J. Fox Foundation for Parkinson's Research (MJFF) and the Center for the Advancement of Science in Space (CASIS) today announce a partnership to send a key Parkinson's protein to the International Space Station for growth under microgravity conditions. Microgravity may allow bigger, more regular LRRK2 protein crystals to grow, which helps solve the protein's structure. That information could help scientists design optimized therapies against LRRK2, a key target in the pursuit of a Parkinson's cure.
"We're thrilled that PD research has been selected to travel to the International Space Station and honored to partner with CASIS on behalf of the PD community here on Earth," said Michael J. Fox in a video message played today at the International Space Station Research and Development Conference in Washington, DC.
LRRK2 protein will be sent to the International Space Station (ISS) as part of the SpaceX CRS-12 cargo resupply mission scheduled for no earlier than August 10, 2017. As manager of the ISS U.S. National Laboratory, CASIS coordinates transfer of scientific materials to and from the ISS and work done in the laboratory. MJFF initiated this project and has supported work to ready the protein for growth in space.
Advancing Understanding of Key Drug Target
LRRK2 is a priority target of Parkinson's research and drug development. Mutations in the LRRK2 gene can cause Parkinson's disease and are linked to heightened levels of LRRK2 protein kinase (a type of protein that modifies other proteins). Therefore, researchers believe inhibiting LRRK2 may prevent or slow Parkinson's progression.
Drug developers have experience and success with other kinase inhibitors, so LRRK2 protein is considered a highly "druggable" target. Also raising interest in this area are findings of shared pathology between LRRK2 mutation carriers and idiopathic Parkinson's patients -- suggesting that LRRK2 inhibitors may treat a broader Parkinson's population.
Hindering this line of drug development, though, is limited understanding of LRRK2's exact structure. Greater understanding of a protein's shape and structure helps scientists design therapies more likely to engage that protein and treat disease. The more you know about the cuts inside a lock, the easier it is to design a key that fits.
Overcoming Limitations Imposed by Gravity
Earth's strong gravitational field produces ground-grown versions of LRRK2 protein with low resolution. The microgravity conditions of the ISS laboratory allow proteins to collect into bigger crystal structures with fewer defects that may allow for higher resolution.
"The unique environment of the International Space Station untethers research from restrictions imposed by gravity," said CASIS President and Executive Director Gregory H. Johnson. "CASIS is glad to partner with The Michael J. Fox Foundation to explore the structure of this important piece of the Parkinson's puzzle."
Furthering the Foundation's LRRK2 Roadmap
MJFF funded Stefan Knapp, PhD, at the University of Frankfurt in Germany and Sebastian Mathea, PhD, at the University of Oxford in the United Kingdom to prepare the LRRK2 protein for transfer and crystallization on the ISS. These investigators -- with Susan Taylor, PhD, of University of California, San Diego -- will analyze the samples when returned from the ISS laboratory. After about a month of growth, the LRRK2 protein crystals will be packaged in the returning SpaceX Dragon spacecraft.
This project is a piece of the MJFF roadmap around LRRK2. The Foundation drives research into the biology of this protein and provides research tools available easily at low cost. The MJFF-led landmark Parkinson's Progression Markers Initiative has built a cohort of LRRK2 mutation carriers contributing data and samples to identify biological markers of Parkinson's for diagnosis and tracking. And the Foundation's LRRK2 Safety Initiative answered fundamental questions about the safety of LRRK2 inhibitors that kept major pharmaceutical companies interested in this area of drug development. Today MJFF is working with regulators and drug developers to plot a clinical testing path for the field
About The Michael J. Fox Foundation for Parkinson's Research
As the world's largest nonprofit funder of Parkinson's research, The Michael J. Fox Foundation is dedicated to accelerating a cure for Parkinson's disease and improved therapies for those living with the condition today. The Foundation pursues its goals through an aggressively funded, highly targeted research program coupled with active global engagement of scientists, Parkinson's patients, business leaders, clinical trial participants, donors and volunteers. In addition to funding more than $700 million in research to date, the Foundation has fundamentally altered the trajectory of progress toward a cure. Operating at the hub of worldwide Parkinson's research, the Foundation forges groundbreaking collaborations with industry leaders, academic scientists and government research funders; increases the flow of participants into Parkinson's disease clinical trials with its online tool, Fox Trial Finder; promotes Parkinson's awareness through high-profile advocacy, events and outreach; and coordinates the grassroots involvement of thousands of Team Fox members around the world. For more information, visit www.michaeljfox.org.