MJFF invests $5 million in Vandy-BMS Parkinson’s collaboration

Research targets the neurotransmitter glutamate

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NASHVILLE, Tenn.—A $5 millioninvestment from the Michael J. Fox Foundation (MJFF) has helpedlaunch a major collaboration between the Vanderbilt Center forNeuroscience Drug Discovery (VCNDD) and global biopharmaceuticalcompany Bristol-Myers Squibb Co. (BMS) that aims to develop afirst-in-class symptomatic treatment for Parkinson's disease.

Under the terms of the agreement,announced Sept. 21, Vanderbilt University will receive an upfrontpayment and three-year research funding to continue to discoveradditional compounds, while BMS will have the right to develop andcommercialize products resulting from the collaborative researchprogram. Vanderbilt is also eligible to receive milestones androyalties based on developmental success and worldwide sales of thedrugs emerging from the collaboration.

If all milestones are met, theVanderbilt team, led by VCNDD Director P. Jeffrey Conn, is hopefulthat an experimental drug could enter clinical testing as soon as2013.

"The long-term commitment of, andcollaboration with, MJFF has been critical to advancing this programto the stage where it is now—perfectly positioned to work closelywith Bristol-Myers Squibb for further development," said Conn in anews release. "Partnering with Bristol-Myers Squibb is a real winfor Vanderbilt and for Parkinson's patients."

An estimated 1 million Americans haveParkinson's disease, a progressive brain disorder characterized byresting tremors, rigidity and slowness of movement, according to theU.S. National Institutes of Health (NIH). Parkinson's is caused bythe death of nerve cells in a specific brain region that produce theneurotransmitter dopamine.

Founded and led by popular actorMichael J. Fox, MJFF has partnered with Conn and his team atVanderbilt since 2005 in pursuit of a new class of Parkinson'sdrugs targeting the neurotransmitter glutamate. By bypassing thedopamine system altogether, a glutamate-based treatment could providethe same symptomatic benefits of levodopa while avoiding disablingside effects such as dyskinesia (uncontrollable movement). TheVanderbilt team has focused on a specific glutamate receptor calledmGluR4.

The mGluR4 receptors are highlyexpressed in areas of the brain directly relevant to Parkinson'sdisease, Conn says. mGluR4 PAMs represent an approach to correct thedysregulated signaling observed in Parkinson's disease andpharmacologically mimic a surgical procedure that has been successfulin alleviating symptoms of Parkinson's disease.

To minimize the likelihood of sideeffects, the drugs must take a subtle approach to manipulating themGluR4 receptor.

"You can liken it to a dimmer switchon a light in your home, where you can turn up the gain of thereceptor and its activity, or turn it down without completelyactivating it or shutting it off," Conn said in a 2009 interview.

Even if the mGluR4I receptor theory isshown to be correct, "I would not consider this a possible 'cure,'"Conn tells ddn. "The hope is that mGluR4 PAMs could reducesymptoms of Parkinson's disease. The ultimate outcome cannot bepredicted at this stage, but will only be known after completion ofclinical studies. Our hope is that this will be a major step towardstreating Parkinson's symptoms."

Although MJFF's direct funding of theresearch has ended and the organization will not play a direct rolein the ongoing research, "they are highly supportive and haveexpressed an eagerness to provide insights and help as the programmoves forward," notes Conn.
Unlike the traditional steps ofresearch and development and funding a breakthrough drug, MJFFadheres to an aggressively funded research agenda that ensures thedevelopment of improved therapies for those living with Parkinson'stoday. From its inception, MJFF has invested in high-risk,high-reward research targets—an approach that in the last decadehas transformed the broader approach in the Parkinson's diseaseresearch field, according to MJFF CEO Todd Sherer.

The Vanderbilt and BMS collaborativeagreement is "the latest example of how de-risking speeds promisingtreatments toward clinical testing and patients," Sherer says.De-risking is the foundation's strategy to make researchinvestments that build a compelling scientific case for the mostpromising new approaches to Parkinson's disease, he explains.

"This, in turn, builds the businesscase for further investment by funders with the resources to carrythe work forward through the most expensive stages of clinicaldevelopment and regulatory approval," Sherer says.

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