The Michael J. Fox Foundation for Parkinson’s Research has created a drug-discovery and -development program to address gaps in Parkinson’s disease research, and speed therapies to market, the group said last week.
The first initiative will provide $1.5 million in funding for Parkinson’s target validation research, MJFF said.
Although a variety of techniques are used in the target validation process, cell-based and small-animal in vivo assays are high on the list.
“There have been a number of potential therapeutic targets identified for Parkinson’s … and the goal of this program is to really determine the therapeutic value of what some of those identified targets are,” Todd Sherer, a scientific manager at the foundation, told Inside Bioassays last week.
“For this particular program, the mechanism that is suggested is to identify chemical compounds that could affect the biological activity of the target,” he added. “That can be done either through a complete in vitro binding assay, or a cell-based or animal assay.
“Once some candidate chemicals are known to influence the biological activity, the goal is to take those into cell-based or in vivo Parkinson’s models to test things like: ‘Does altering the biological function of that target seem to have a therapeutic effect in a model system?’” Sherer said.
MJFF has funded more than $46 million in research to date aimed at finding a cure for Parkinson’s. But according to Katie Hood, MJFF’s director of research, the organization recently began noticing some gaps in key research areas — particularly in drug development — and decided to take an active role in filling them.
“We hadn’t done any funding initiatives in this area specifically,” she said. “With one of our goals being finding a cure for this disease, we started to ask ourselves, ‘Should we be trying to fund more drug development?’”
Hood said that to better answer that question, the foundation convened in Dallas last July with experts from academia, industry, the US Food and Drug Administration, and other foundations to discuss how it could best contribute to the Parkinson’s drug development pipeline. Target validation eventually emerged as clear area that needed to be addressed.
“There are a couple of reasons for this,” Hood said. “Academics don’t have incentive necessarily to validate targets that they identify — it’s not hypothesis-driven science, it’s applied science. So their glory comes from identifying targets, but not necessarily proving their exact relevance to the disease,” she said.
“Meanwhile, we heard in this meeting that industry really isn’t interested in doing the validating themselves,” added Hood. “They’re completely interested in screening their libraries against validated targets, but they’re not usually going to do the validation work themselves.”
To provide more incentive, MJFF will dole out awards of an unspecified amount, although Hood said that on average, individual foundation grants tend to be for $250,000 over two years.
“That varies by program, but it tends to be the average,” she said. “For this program, I think we’ll have budgets that vary quite a bit, depending on where they’re focusing. We’re open, though. The reason we don’t name a specific grant size is that we want people to apply for the amount that they feel is appropriate for the work they’re submitting.”
The target-validation RFA is one of three that the foundation has launched (the two others are clinical discovery awards for human-pilot studies). Hood also said that the foundation has invested a lot of time and money into biomarker research, an area that “we think it going to be incredibly important to Parkinson’s research,” she said.
As to what type of identified targets researchers should be validating, Sherer said it’s wide open.
“This is our first real attempt in this area, so we’re trying to keep it fairly open for the creativity of the scientists,” Sherer said. “That’s why we haven’t really designated a specific target that we’re interested in.
“The target that’s chosen will be part of how the application is viewed, so the researchers will have to justify their targets,” she said. “But we leave it to the expert scientists who may know that some targets are more druggable or more easily manipulated than others, so they fit this analysis better; and some targets may not be ready for this level of scrutiny yet, and more basic work needs to be done.”
Sherer also said that is important to the foundation to foster collaboration among its grant recipients.
Letters of intent are due by Jan. 17, 2005, and funding is anticipated by June 2005, MJFF said. Interested scientists can find more information at the foundation’s website, http://www.michaeljfox.org/.