Today's visionaries battle cancer with cutting-edge science and the stubborness to make it work
Surviving cancer requires teamwork. On the evening of Monday December 8, 2014, a small army of some of the best “soldiers” in the battle against blood cancer mingled with patients and members of the press in “The Swamp” at the California Academy of Sciences in San Francisco. Just feet away, Claude—an albino alligator—laid still, but deadly, much like an undiagnosed cancer waiting to unleash itself on an unsuspecting victim. The crowd gathered there that night to learn how sophisticated research from dedicated and driven scientists promises to—and in some cases already does—cure cancer.
This Top Medical Innovators forum was organized through a partnership between Scientific American Worldview and The Center for Medicine in the Public Interest (CMPI). The event recognized eight experts for their work in the fight against blood cancer, from diagnosis to treatment (see “Innovator Alley”). Gaining ground in this difficult battle demands crusaders like these to continue the mission of developing innovative new treatments.
As Jeremy A. Abbate—publishing director of Scientific American Worldview and global vice president for global media alliances at Scientific American—said in his introduction, “One of the most defining characteristics of the enterprise of innovating is taking nature and making it a little bit better, using what nature gives us and tweaking it just a little bit.
Indeed, when a researcher’s discovery yields a revolutionary new medicine, that tweaking can change lives. “Innovation is inventiveness put to good use,” Bob Goldberg, one of the cofounders of CMPI, told the audience. “Inventiveness springs from the imagination of our honorees this evening.”
As I took the podium, I started to call up four of the innovators who joined us that night: Patricia Ernst of the University of Colorado Denver–Anschutz Medical Campus; Bob Hariri of Celgene Cellular Therapeutics; Carl June of the University of Pennsylvania’s Perelman School of Medicine; and Diane Wuest, representing Colin Hill of GNS Healthcare. I felt awed to be there as I invited Carl June to the panel.
Changing the Odds
June’s groundbreaking approach to combating leukemia begins with the immune-system cells from a particular patient, genetically re-engineers those cells to kill that person’s specific cancer and then injects them back into that individual. He first used this protocol in 2010 to treat an adult with the disease—which typically kills 80–90% of its victims—and the injected cells destroyed seven pounds of tumor. The man is alive and well today.
In addition, June had recently completed a study in which 39 children with leukemia—for whom several traditional treatments had failed—received their own re-engineered immune cells. Of these children, 35 went into complete remission. That translates into a response rate of 92%! As June said, “It’s a heartwarming story, because you see kids who have gone through all the kinds of things that chemotherapy can do, or radiation, and most of them already relapsed after a bone marrow transplant—but after immunotherapy, these kids have returned to a normal life.”
For decades healthcare experts predicted that computers would transform the field, but until Colin Hill came along we did not know precisely how. Hill is combining computational physics, systems biology and personalized medicine to completely rewrite the way that clinical trials are done. Instead of running them in people, he plans to simulate them in computers.
That night in California, I asked Wuest: “Why test a drug in a computer?”
“You can gain a lot of insights,” she answered, “and there are more opportunities to study disease on a holistic level in ways that are cheaper and in ways that are faster because we can take in data that’s already being collected in the healthcare system.” That information can then be used to find just the right treatment for each individual patient.
Mining New Targets
Genetic solutions to cancer have been anticipated since the sequencing of the human genome more than a decade ago, and some of today’s treatments do target changes in genes. But Patricia Ernst believes that examining other cellular alterations will unveil even more targets. She looks at so-called epigenetics.
“Most people are familiar with the concept that when you get cancer one of your genes has suffered a mutation and that’s why the cells grow uncontrollably,” Ernst explained, “but there are a lot of other things that can happen to a cell.” For example, the molecules that control genes—known as regulators—may also cause or affect the growth of cancer. This is an example of epigenetics, and such changes can impact the onset and severity of leukemia.
“There are many new drugs that are being developed that not only influence heritable changes in gene expression,” she continued, “but also directly target epigenetic regulators that are mutated in particular leukemias.”
As Abbate noted earlier, the “tweaking” of natural processes can lead to innovative discoveries, which was the case for Bob Hariri. “We realized probably 20 years ago that for cellular medicine to have a meaningful impact on patients’ lives we were going to have to identify a reliable, renewable source that could be turned into a product and put into the hands of physicians—sort of the same way we provide them with pharmaceuticals,” he said.
While working as a neurosurgeon, he realized that stem cells would be particularly useful in treating head and spinal cord injuries, but he needed a source of them. So he turned to what he called “the leftovers of full-term healthy pregnancies, namely, the placenta.” He added, “This organ is nature’s stem-cell factory.”
At Celgene Cellular Therapeutics, Hariri and his colleagues convert stem cells into new therapies for diseases including cancer.
Never Give Up!
When I asked the panel how we can be sure to keep innovation moving forward, June replied that scientists must be stubborn. “Basically, I tell people when they start in my lab that you have to expect 90% of the time—at least—you are going to fail, so if you’re playing baseball, that means you bat 100,” he said. “You have to get used to that, but when you do get something that works it’s an amazing thing.”
The things that work change lives (see “Marathon Man” and “Saving Dolphins Despite Disease”). As Hariri said, “I am moved and compelled by each story where the investment and the time of creating a new drug, a new therapeutic, has transformed those individuals’ lives.”
As I thought over what the panel had said that night and looked over the crowd, I felt moved, too, and optimistic about a more powerful today and an even more innovative tomorrow.
Scientific American Worldview and The Center for Medicine in the Public Interest recognized eight “Top Medical Innovators.”
Each of them works on life science or medical research that is already changing lives, and is sure to affect many more in the future.
Iannis Aifantis 
Professor and Chair | Department of Pathology | New York University School of Medicine | New York, New York
Aifantis uses blood stem cells to study cellular development and how cells turn into leukemia and lymphoma. He also studies the immediate environment around a tumor in hopes of creating more targeted drugs.
James Allison 
Professor Department of Immunology | Executive Director of the Immunotherapy Platform | University of Texas
MD Anderson Cancer Center | Houston, Texas
Allison showed that blocking CTLA-4 on T cells can cause them to fight cancer more effectively, and this work led to the drug ipilimumab, which is FDA-approved to treat metastatic melanoma.
Susan Desmond-Hellmann 
CEO | Bill & Melinda Gates Foundation | Seattle, Washington
When Desmond-Hellmann was president of product development at Genentech, she contributed to the development of Avastin and Herceptin, which were two of the first gene-targeted therapies for cancer.
Patricia Ernst 
Professor | University of Colorado | Denver–Anschutz Medical Campus | Aurora, Colorado
Ernst studies the epigenetic—traits that can be inherited but are not in the genes—mechanisms that impact the development of healthy blood and the changes that lead to leukemia. This basic research uncovers a vast collection of new drug targets.
Bob Hariri 
Chairman | Celgene Cellular Therapeutics | Warren, New Jersey
Hariri discovered pluripotent stem cells, which are capable of developing into any kind of cell, within the placenta and pioneered their use in regenerative medicine. He and his colleagues work with these cells to fight inflammatory diseases, cancer and other conditions.
Colin Hill 
CEO and Cofounder | GNS Healthcare | Cambridge, Massachusetts
Hill uses advanced computing, sophisticated algorithms and large datasets to assess the impact of drugs on patients. In this way, he is redesigning the manner in which we test drugs: instead of running clinical trials in humans, he runs clinical trials on computers.
Carl June 
Richard W. Vague Professor in Immunotherapy | Department of Pathology and Laboratory Medicine | Director of the Translational Research Program | University of Pennsylvania’s Perelman School of Medicine | Philadelphia, Pennsylvania
He developed CTL019 immunotherapy for B-cell cancers, including acute lymphoblastic leukemia (ALL), non-Hodgkin lymphoma and chronic lymphocytic leukemia. The US Food and Drug Administration gave CTL019 “breakthrough therapy” status in 2014 for the treatment of relapsed and refractory adult and pediatric ALL. Recently, he reported on a study in which this treatment put 35 out of 39 children with leukemia into complete remission.
Christof von Kalle 
Director of Translational Oncology | National Center for Tumor | Diseases and German Cancer Research Center | Heidelberg, Germany
Von Kalle researches techniques in therapeutic genetics, such as using a virus to deliver a drug. This process could lead to treatments for certain cancers and the prevention of others. He also works with colleagues on new ways to diagnose cancer sooner and more accurately.
PATIENT CASE STUDIES: INNOVATION IN ACTION
72-year-old Don Wright—a multiple myeloma survivor—takes aim at his 100th marathon
In February, several weeks before he planned to run his 86th marathon, Don Wright made time to talk with Scientific American Worldview. A few years earlier, in December 2012, he had achieved his goal of finishing one marathon in each of the 50 states. Now, he wants to raise his marathon count to 100, a feat he hopes to reach by the end of 2016. But without a doubt, his greatest accomplishment is staying healthy despite being diagnosed with multiple myeloma just after his first marathon, in 2003.
Then, instead of giving in to the cancer, he decided to take on the Boston Marathon, followed by one race after another. His wife and daughter often come along and run the half marathon at the events he attends. Wright can keep running in spite of his myeloma because of a once-a-day pill called Pomalyst. He has been on the drug for seven years—starting with a clinical trial five years before it was approved—without any troubling side effects. Well, except for one, he laughs: “The big side effect is that it makes me want to go run marathons!”
Jokes aside, Wright deeply appreciates his treatment as well as his health. And of his current cancer-fighting medication, Pomalyst, he says, “It’s helped me live long enough to meet my grandchildren, and that’s extremely important in my life.”
Saving Dolphins Despite Disease
Hardy Jones fights to protect marine life even as he battles cancer
In 2000, life couldn’t have been better for life-long diver and filmmaker Hardy Jones. Teaming up with actor Ted Danson, Jones founded BlueVoice.org to protect whales and dolphins. But in 2003, he was diagnosed with multiple myeloma. His treatment started with Thalomid and a steroid, dexamethasone. Thalomid stops the growth of cancer cells in the bone marrow. “It reduced my myeloma burden by 97%,” Jones says. “It was very fast.”
He started on high doses of both medications, but his oncologist brought them down over time. Eventually, Jones got 16 months off without any treatment at all. “In that 16 months,” he says, “I was quite stable, but then the myeloma began to creep up.” So he tried another drug and experienced a similar cycle—treatment, quick cancer-killing response, eventual treatment vacation, followed by cancer recurrence, which led to a new treatment.
At 71 years old, Jones continues to receive treatment and pursue his work. When he spoke with Scientific American Worldview, he was deep in a project to save dolphins being killed in Peru for use as shark bait. “We’re making a film on it now,” he says. “If we don’t stop this dreadful practice, they will wipe out the dolphins and sharks.”
While Jones saves the world’s marine life, advanced drugs—with ongoing innovations keeping his options coming—save him. There is no better teamwork than that.
Illustrations by Meen Choi
Enhanced with a new guidebook and region-specific ratings, the 2016 Scorecard ventures deeper than ever to track down the latest in biotech innovation