The Future of Medical Innovation: Beyond Our Wildest Dreams?
Kicking off the second plenary session at Partnering for Cures, Francis Collins, director of the National Institutes of Health, pointed out that the rate of innovation in the biomedical field is often overlooked. Of course, nothing says that this rate of progress is preordained.
For the trend to continue, “we need resources; we need partnerships; but most of all, we need the talents of investigators willing to think wacky ideas,” said Collins. A quick glance at the numbers shows that the biomedical community cannot rest on its laurels. “We know the molecular sequences of more than 5,000 out of the 7,000 known diseases in the world,” Collins reminded the audience. “We have cures for less than 500.”
With that in mind, Collins turned his attention to the panel, composed of individuals whose innovations and research have indeed given us solutions and cures beyond our wildest dreams. Collins asked them all the same question: “What are you working on right now that is particularly innovative or groundbreaking?”
First up was Atul Butte, chief of the Division Systems Medicine and associate professor of pediatrics, medicine, and computer science at Stanford University. “I hope to convince you that the present day is amazing,” said Butte, whose work on gene chips allows him to map diseases by measuring which genes are active in a cell. Developed more than a decade ago, there are more than a million gene chips (which are no larger than a credit card) publicly available. The data derived from the chips have allowed scientists like Butte to define human diseases more precisely by understanding their genetic makeup.
Rudolph Tanzi, director of the Genetics and Aging Research Unit at MassGeneral and a professor of neurology at Harvard Medical School, is also excited about the possibilities found in the genome, but not without caveats.
“In the early 1980s, we found the first genetic variance in a genome and things really took off,” he told the audience. “There was so much enthusiasm and excitement that cures would be just around the corner. But where are the cures?”
Part of the problem, said Tanzi, is that our disease models have been inadequate. By better mimicking brain functions in the lab environment, Tanzi and his team have successfully created “Alzheimer’s in a dish.”
“For the first time, we’re seeing the pathology,” he said. “We see how amyloids are occurring in the brain 15 years before the first symptoms [of Alzheimer’s].” Tanzi hopes that these breakthroughs will enable his team to build the first 3D model of an Alzheimer’s brain.
Also doing amazing work in disease models is Sangeeta Bhatia, the director of Laboratory for Multiscale Regenerative Technologies and a professor at MIT. “We’ve learned that the whole body is organized around units of 100 microns,” she said.
This insight has allowed Bhatia and her team to treat the body’s microns as engineers once treated the microchip, unleashing a burst of innovative research. For example, Bhatia has been working on implantable liver tissue, which begins in the lab but grows on its own once in the body.
“We can grow a little one and the body takes over,” she said. Building “mini-organs” also means that Bhatia can “model diseases in the micro-environment without exposing people to dangerous toxins,” potentially revolutionizing how drugs are tested.
In a somewhat different vein, Bonin Bough, vice president of global media and consumer engagement at Mondelez International, says he’s “maniacally focused on how we accelerate growth.” By this Bough, whose background is with Kraft Food products, is talking about “the colliding” of different industries and talents coming together to create breakthrough innovations. “Where does food play in this space? We launched the first-ever 3D printed Oreo cookie.” Aside from the novelty aspect of this, Bough said that 3D printing and “personalized food” promises to revolutionize health and nutrition. Naturally, “the only way that type of innovation will ever come to life is the colliding of industries,” said Bough, specifically the food and biomedical industries.
Finally, Dan Wattendorf is a program manager in the Defense Sciences Office at DARPA. His work on gene therapy for the government agency that brought the world the Internet and GPS has transformed the field from one that focuses on fixing bad genes in sick people to delivering better genes in healthy people.
“For the purposes of global security, we want to use gene therapy techniques where the protein isn’t present but the person is otherwise healthy,” he said. For example, Wattendorf’s work can help healthy people develop a tolerance to something as minor as a bee sting or as major as a chemical weapon.
Echoing the other panelists, Wattendorf stressed that for his research to progress to actionable solutions requires the collaboration of multiple sectors and industries. Indeed, in his own struggle to add, rather than replace, proteins in a red cell, Wattendorf said “we needed to create a consortium and collaboration” among engineers, academics, and small and large biotech companies.
In his closing comments, Butte reminded the audience that the future is now in biomedical research. “The data is good enough now,” he said. “Don’t wait to get started.”