Fulfilling the promise of bioscience

Bioscience is facing a Sputnik moment, according to Michael Milken, founder of FasterCures, as he opened the first session of the Partnering for Cures conference. The 1957 launch of Sputnik spurred the United States to focus on science and technology. Today, dramatic advances in genomics and disease research have brought the scientific community to the threshold of new discoveries, and he challenged the panelists in the Partnering for Cures opening session, “The (Near) Future of Bioscience and Health,” to explore what needs to change in bioscience in order to fulfill the promise.

Increased collaboration among stakeholders who don’t normally work together was seen by all to be a crucial factor in the future success of biomedical science. The U.S. Food and Drug Administration’s (FDA) Vicki Seyfert-Margolis said her agency is not just involved in regulation; rather, its role is to promote and drive innovation. She envisions a triangle composed of basic research, product development, and the FDA. “Information should move seamlessly between them,” she said.

Pfizer has been pursuing new collaborations as well, reported Mikael Dolsten, president of its worldwide research and development. “There has been a climate change in relation to regulatory agencies and academic partners,” he said. “I have been enthusiastic to see how many academic medical institutions were willing to take a new look at how to work together, work like one team to address different ways to treat disease,” he said.

Pfizer collaborated with the Cystic Fibrosis Foundation, Dolsten said, to define together a research plan to identify new molecules that will translate into successful compounds for patients with the disease. His company also has partnered with the Lupus Foundation in a similar way. If companies like Pfizer build on this, and others in the bioscience ecosystem see their success, he said, it will bring new interest into these partnerships.

New stakeholders and new voices must also be brought into the process, the panelists agreed. Risa Lavizzo-Mourey with the Robert Wood Johnson Foundation pointed to research conducted at the University of Miami into whether babies in neonatal units feel pain. Neuroscientists partnered with engineers to identify physical manifestations of pain. “The team brings the right kind of innovation to the table,” she said. “We don’t recognize all of the perspectives that we need.” Along those lines, she also identified the nurse scientist as a stakeholder who can help drive innovation, since these individuals are often closer to the patients and more familiar with their needs.

Funding is a problem at all levels. Milken noted that the FDA continues to be underfunded, even as the rest of the world falls back on the FDA for rulings on new drugs and devices when they run into problems in clinical trials. And with venture capital investments into the “valley of death” decreasing, patient collaboration becomes that much more important.

For Jay Schnitzer of the Defense Advanced Research Projects Agency, embracing risk is another key to success in bioscience. He said the field today is too risk averse. “We have to be willing to fail, to fail often, to know we fail and move on,” he said.

The decoding of the human genome is part of a “sea change” in biosciences, said Kenneth Davis of the Mt. Sinai Medical Center. Now, when some patients respond to a treatment, health records can be superimposed with genomic data to identify responders and find others with the same genomic makeup who can be used to test whether a new treatment will be effective. This precision medicine approach is being used with cancer research now, he said, and if more people contribute to biobanks and get behind genomics, he hopes more can be done with brain diseases.

A basic problem in healthcare today, Milken said, is that we spend more resources treating diseases than preventing them. Davis noted that Mt. Sinai is a fee-for-service medical system where payment is not keyed to prevention. But that is changing, he added: bundled payments, accountable care organizations, and other innovations are moving the U. S. healthcare system away from this fee-for-service approach.

In his conclusion, Milken cited notable medical advances. He noted that science averted a gloomy prediction of how widespread polio would be, and even in just a few decades, mobilized efforts have resulted in great strides in AIDS research and treatment. While the challenges are great in the future of bioscience and healthcare, Milken said that he has “a tremendous sense of optimism about the future.”