Getting a drug through the tough clinical trial process, in a manner that satisfies the FDA, is an enormous chore. Sometimes we assume this process weeds out most of the potential treatments. It turns out that drugs and devices that make it into clinical trials have already overcome the most difficult obstacle—making it out of the laboratory. There are thousands of chemicals and contraptions that never get an audience outside of the laboratory. The process of making it from the lab to clinical trials is called “translational research.” Only about 10 percent of drugs that start the clinical trial process eventually make it all the way through to FDA approval. However, the odds of even getting out of the lab are usually much smaller than that.
Only a few ideas get to make it to clinical trials because of financial limitations.
A good analogy is the choice to purchase automobiles in a family. There are hundreds of automobile choices and each one offers you something a little different. You have to look at the costs involved, your needs, then match them up with your financial abilities.
Drug companies and the federal government fund the lion’s share of the clinical trials that occur in the United States. They evaluate a number of choices and match those choices to their expectations. Drug companies look for the drugs most likely to be approved, and that attract a large number of patients. This strategy translates into large profits.
Earlier this week the Michael J. Fox foundation announced that it will grant $2 million next year to fund the steps that will help potential drug therapies get out of the lab and into clinical trials. The project is called Target Validation.
“The Foundation’s strong focus is providing funding for translational research that can drive PD drug development and the delivery of better treatments to patients that much faster,” said Deborah W. Brooks, president and CEO of The Michael J. Fox Foundation. “Target Validation is key to these efforts because of its potential to accelerate the pace of drug discovery and to encourage industry investment in Parkinson’s.”
Later this month the Foundation will announce a new initiative, Novel Approaches to Drug Discovery for Parkinson’s Disease, which complements the Target Validation program. Similar to Target Validation, this program provides critical resources for under-funded stages of the drug development process. It also reflects the Foundation’s emphasis on bridging early discovery work and late-stage translational research to “de-risk” industry investment in new PD therapeutics.
In other words, if the Fox Foundation can provide money to further test ideas and potential treatments, they may help drug companies have a better idea which Parkinson’s disease treatments are more likely to be approved after the grueling clinical trial process.
Why is this important? Let’s say a large, wealthy drug company is looking for a treatment to invest in. Perhaps it is interested in a diabetes treatment. Well, if it is successful, the drug company will be able to offer that treatment to 20 million American diabetics. It would rather gamble on a payoff to 20 million potential customers, than it would on 1.5 million potential customers with Parkinson’s. The argument is much more complex than this, but the diabetes example introduces some real economics.
The Parkinson’s community needs an edge, so we can attract more attention. What if the extra research the Fox Foundation finances moves the research along to a place where the large company is five times more confident that the Parkinson’s treatment will make it through the clinical trial process? That might put the Parkinson’s research in the running for the research dollars that would have otherwise gone to diabetes.
We work hard to be straightforward about all the issues with Parkinson’s disease. We do not enjoy writing about the economic roulette that goes on with your lives hanging in the balance. Nevertheless, we are not going to ignore it. We actually hope it fuels the efforts of those who advocate Parkinson’s disease funding. From the Californian who sits down with her congressional representative to explain life with PD and encourage her representative to vote for bills that will fund Parkinson’s research, to the grandson in Connecticut who collects donations for the Unity Walk, those funds go to give us the edge. Your dollars may lift that one additional idea out of the laboratory and into clinical trials. That one idea that could create the medication that protects neurons for additional decades, or the infusion that will regenerate dopamine-producing neurons in the sustantia nigra.
