Can academia bridge the gap between bench and bedside?
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basic research and industry took over as soon as basic discoveries were patented—is breaking down. Pharma’s massive multi-billion dollar investment in R&D over the last few decades has not yielded as many drugs as once hoped for. With investors losing confidence that early-stage biotechs are the sole answer to filling this pipeline void, attention is turning over to academia as the place to possibly carry discoveries a bit farther down the path to approval. So more and more, the ivory tower is poised to encroach into areas that have tended to be the exclusive turf of industry.
Take, for example, the translational medicine program at Massachusetts General Hospital. It is led by Mason Freeman, a physician who has inhabited both cultures: From 2005 to 2007 Freeman left academia to do a sabbatical as head of translational medicine for cardiovascular, diabetes, and metabolic diseases at the Novartis Institutes for Biomedical Research in Cambridge, MA. He then came back to MGH to run the translational medicine program.
Freeman’s group picked a lead compound in late 2007, performed all the studies needed to gain FDA permission to begin clinical testing, designed the clinical studies, and is now more than halfway through a phase II clinical trial in diabetics. The work was done for a biotechnology company that wanted to outsource it, and Freeman says his group took it on as a proof-of-concept program, partly to show to industry that such work can be done in academia.
Another example is the Broad Institute of MIT and Harvard in Cambridge, MA. Stuart Schreiber, director of the Broad’s Chemical Biology Program, says the institute has a “highly novel chemical substance” that looks like a promising candidate to treat cancer. The compound was discovered at the Broad in 2007, and since then Broad researchers have done all the optimization chemistry, as well as the animal pharmacokinetics and toxicity studies, so the compound is ready to go into the clinic. Schreiber adds that over a dozen clinical trials have also been launched by the Broad focusing on existing drugs that could be repurposed for other indications—the kind of work that usually done in biotech and pharma instead of academia.
Both Freeman and Schreiber said pharma’s prejudice about academics is partly justified. “For the most part, the typical academic health center and academic scientist doesn’t know very much about the rigors of drug development,” Freeman says. Schreiber adds that “overall, there has not been a long and outstanding record” of successful drug development programs in academia, “so industry has a good reason to be cautious in their enthusiasm about what academia can deliver.”
They both said, however, that there are a few groups in the country based out of universities and hospitals that, like their own, are beginning to take things much farther than academia traditionally has. Schreiber brought up Vanderbilt Medical Center in Nashville, TN, as a good example. There, researcher Jeffrey Conn leads a program in drug discovery. In a deal much more characteristic of what would happen in the biotech setting, Conn’s group recently signed a $10 million deal with Johnson & Johnson to develop treatments for schizophrenia.
“We believe academic organizations can perform these functions if they have the right people,” says Freeman of these kinds of drug development efforts. Finding people with industry expertise is crucial, they both said. At the Broad, salaries offered to scientists are on par with what they would earn in industry—a luxury that academic centers can’t usually afford. Schreiber says that at the Broad there are “maybe 60 scientists who’ve been in the pharma industry,” and many of them are among the institute’s senior leadership.
Of his own program, Freeman says: “We have specialized scientific and clinical expertise that can be extremely important to the early decisions about whether a therapeutic compound is going to work in a disease. We have imaging tools, trained human physiologists, laboratories of human investigation—and all of this can be tremendously powerful.”
Of course, as they say, the proof is ultimately in the pudding. Only when some of these promising compounds make it through phase III and into the market will the “new academia” model be validated. Until then, industry’s skepticism will be entirely justified.