It’s not every day that you see one Big Pharma company handing an award to another, but that’s just what happened yesterday at the New York Academy of Sciences. New Brunswick, NJ-based Johnson & Johnson (NYSE: JNJ) gave its Dr. Paul Janssen Award for Biomedical Research to Napoleone Ferrara, the Genentech scientist whose discoveries contributed to the development of the blockbuster cancer drug bevacizumab (Avastin), and a related treatment, ranibizumab (Lucentis), for the blinding disease macular degeneration. Genentech, based in South San Francisco, is a unit of the Swiss drug giant Roche.
In conjunction with the award presentation, J&J convened a top-flight group of scientists for a day-long scientific symposium on the future of treatments such as those that Ferrara spearheaded. Ferrara is most famous for his work with vascular endothelial growth factor (VEGF), a protein that promotes blood-vessel growth. His findings contributed to the birth to a new class of drugs—anti-VEGF compounds that starve tumors by cutting off their blood supply, and preserve sight by drying up the leaky blood vessels that cause macular degeneration.
The success of anti-VEGF drugs has brought a mix of celebration and criticism to Genentech. Sales of the cancer product hit $7.5 billion last year, while the macular degeneration drug brought in about $1.6 billion in sales. But late last year, an advisory panel to the FDA recommended the agency revoke its approval for the cancer product to be used in treating breast cancer, based on clinical trial results that suggested it didn’t work more effectively than other treatments. Genentech appealed the decision. Meanwhile, ranibizumab has been under pressure from eye doctors because of its $2,000-per-dose price tag. Many are choosing to use tiny doses of bevacizumab instead, which they can get for $20 to $50 per dose.
Although the commercial controversies did play into the discussion at the award presentation, the scientists chose to focus more closely on work being done to better define VEGF—and hence to improve treatments designed to block it. Ferrara described work in his lab to understandthe different forms of VEGF and how they influence patients’ responses to anti-VEGF treatments. Additionally, his lab is among those trying to understand how drug-resistant tumors recruit and use cells from the bone marrow.
Working out such complexities of cancer should lead both to improvements in anti-VEGF treatments and to ideas for how to combine them with other drugs, Ferrara said in an interview after his presentation. Current treatments “are on the tip of the iceberg,” he says. “We’re looking actively at combinations, not only with chemotherapy but with other biological compounds.”
Ferrara is optimistic that ongoing research in VEGF will also lead to the development of diagnostic tests that will be able to predict which patients will respond best to which treatments—though he acknowledges it may take some time. “The biggest challenge is finding biomarkers, so we can increase the effectiveness of Avastin to make it tumor-specific,” Ferrara says. “But that will take years.”
UCLA researcher Luisa Iruela-Arispe described work in her lab to define the mechanisms by which tumors become resistant to anti-VEGF treatments. She started her talk by noting that drugs inhibiting VEGF performed much better in animal models of cancer than they did in people; bevacizumab was initially approved in colon cancer based on human studies showing it added about five months of survival. Her comments prompted a friendly but salty response from Ferrara, who dashed from his seat in the audience to a microphone and declared: “I never said we’re going to cure cancer.”
Still, everyone was in agreement about the need to better define the complexities of VEGF, so treatments can be enhanced. The day included a presentation by Anthony Adamis, VP and global head of ophthalmology for Genentech, who noted that 60 percent of macular degeneration patients who take anti-VEGF treatments are still visually impaired. He described research looking at various methods for boosting effectiveness of VEGF blockers, such as combining them with anti-inflammatories or with treatments designed to promote the regression of blood vessels.
As for J&J, it currently isn’t doing any work in anti-angiogenesis treatments, says Paul Stoffels, company group chairman of pharmaceutical R&D. The award—established in 2004 and named for Paul Jannsen, who founded a drug company that J&J bought in 1961—was set up to “recognize good science wherever it happens,” Stoffels says. All the talk of combination approaches to anti-VEGF treatments reminded Stoffels of similar work at J&J in HIV, he says. In May, the FDA approved rilpivirine (Edurant), a once-daily HIV treatment that Paul Janssen started developing in 1989, Stoffels says. J&J teamed up with Foster City, CA-based Gilead to develop a combination pill, which was approved in August. The embracing of combination treatments in cancer “is evolving in a similar way to HIV,” Stoffels says.
Stoffels says he expects to see more such cross-company collaborations. “In the end,” he says, “it’s the progress of science that will make a difference for patients.”