Need New Approaches to Translate Discoveries into Treatments

Said Michael J Fox in a keynote address on Driving the Development and Availability of Novel Therapies

On May 7, 2007, actor Michael J Fox urged the scientists and investors to aggressively translate scientific research into improved treatments for serious diseases like the Parkinson's disease that he has fought for more than a decade. "Levodopa is the gold-standard treatment for Parkinson's. But it's a little frustrating that the best drug that we have is one that has been around for 40 years."

Fox observed that the grants from the National Institute of Health (NIH) have created a system that encouraged academic scientists to publish papers that resulted in academically interesting answers. "But the system has not translated the discoveries into new treatments and cures," noted Fox.

He said the pharmaceutical and biotechnology companies have not taken up high-risk studies critical in determining whether creative ideas could yield therapies for the 20,000 human diseases that have no cure. The industry is focused on finding new indications for existing drugs and platforms, rather than drug discovery. They are more interested in repackaging old ideas and compounds in an effort to continue reaping the returns, Fox observed.

He, however, appreciated the efforts of the biopharmaceutical industry with good humor during his keynote address at the 2007 BIO International Convention. "I can stand still up here and that's thanks to you."

Fox further observed that in the $100 billion a year drug discovery pipeline, the need is not for more money but for re-purposing the money available. He jokingly pointed out that anti-depressants are available for his dog (and his dog is thankful) and noted that more groups could work together to define different standards for success.

Drug discovery is a high-stakes business and companies need to be successful financially. He called for more investments in the early stages of development--effectively bridging the funding gap left between the early-stage, fundamental research supported by the NIH and the spending made by the private sector on clinical trials. This will spur more drugs to be developed with a lower risk to companies--a strategy to "de-risk" the drug pipeline. Fox said, "Filling this unmet need is not about finding more money. It's about spending money differently. We can all achieve success. It's only a question of finding common ground where stakeholders in the drug development process, including academic and industry researchers, government, and private research funders, can work together towards tangible benefits."

Fox, 45, who starred on "Family Ties" and "Spin City" on the TV, was diagnosed with Parkinson's in 1991 and revealed his condition publicly in 1998. In 2000, he founded the Michael J Fox Foundation for Parkinson's Research. His foundation has funded more than $90 million of research, either directly or through partnerships, supporting more than 300 Parkinson's research projects throughout the world. The foundation's portfolio of research investments reflects its focus on translational and clinical research, including major investments in the pursuit of a Parkinson's biomarker and clinical trials exploring neuroprotective and gene therapy techniques.