September 18, 2024

Challenges ahead

There are limits to the new science of proteins.

For example, researchers often don’t speak the same scientific “language,” making it difficult for them to share information or collaborate. To solve that problem, the international Human Proteome Organization is developing standards for the reporting of experimental data.

Pharmaceutical companies also may have to shift their focus from the search for the next blockbuster drug to the development of combinations of drugs, tailored for different segments of the patient population.

That’s easier said than done. Currently it can take 15 years and more than $800 million to bring a drug to market – in part because of rigorous testing requirements and the high number of potential drugs that never make it through the testing process.

If drug companies could tell in advance which patients – because of their genetic make-up – are most likely to respond to a new medication and are unlikely to experience serious side effects, “you could reduce the number of people you have to study … and you can greatly reduce your cost,” says Alastair Wood, M.B., Ch.B., professor emeritus of Medicine and Pharmacology at Vanderbilt and an expert on pharmacogenomics. But, he adds, “no one’s worked out how to do that yet.”

Gene- and protein-based technologies may speed drug development, thereby lowering the cost. But intellectual property issues and a regulatory structure designed to approve one drug at a time could stifle efforts to develop “cocktails” of different drugs – made by different companies — that attack a disease at several points simultaneously, experts say.

“One of the things that’s really inhibiting progress is that the FDA (U.S. Food and Drug Administration) has never dealt with approval of multi-use agents for an indication like this,” says Raymond DuBois, M.D., Ph.D., former director of the Vanderbilt-Ingram Cancer Center who helped discover the role of the COX-2 enzyme in colon cancer.

“I think it’s going to be hard to get an individual pharmaceutical company to fund these combination trials using drugs from different companies together,” DuBois says. “One possibility would be for the National Cancer Institute to step up to the plate and put some trials together to look at this.”