He watched his brother die from a cancer that no drug could cure. Now one of the world’s most renowned cancer researchers says it’s time for Plan B. ... The answers Bert Vogelstein needed and feared were in the blood sample. ... Vogelstein is among the most highly cited scientists in the world. He was described, in the 1980s, as having broken into “the cockpit of cancer” after he and coworkers at Johns Hopkins University showed for the first time exactly how a series of DNA mutations, adding up silently over decades, turn cells cancerous. Damaged DNA, he helped prove, is the cause of cancer. ... Now imagine you could see these mutations—see cancer itself—in a vial of blood. Nearly every type of cancer sheds DNA into the bloodstream, and Vogelstein’s laboratory at Johns Hopkins has developed a technique, called a “liquid biopsy,” that can find the telltale genetic material. ... The technology is made possible by instruments that speedily sequence DNA in a blood sample so researchers can spot tumor DNA even when it’s present in trace amounts. The Hopkins scientists, working alongside doctors who treat patients in Baltimore’s largest oncology center, have now studied blood from more than a thousand people. They say liquid biopsies can find cancer long before symptoms of the disease arise.
Nothing except a crazy experimental treatment never before given to a child: Blood was taken out of 6-year-old Emily’s body, passed through a machine to remove her white cells and put back in. Then scientists at the University of Pennsylvania used a modified HIV virus to genetically reprogram those white cells so that they would attack her cancer, and reinjected them. ... commercializing June’s cancer-killing cells would be like no drug development program ever. Scientists call them chimeric antigen receptor T-cells, or CARTs. T-cells are the immune system’s most vicious hunters. They use their receptors to feel around in the body for cells with particular proteins on their surface and destroy them, targeting infected cells and cancer. With CARTs scientists add a man-made receptor–the chimeric antigen receptor–assembled from mouse antibodies and receptor fragments. A gene code for the man-made receptor is inserted into the T-cell’s DNA with a virus, usually a modified HIV. If the receptor sees cancer, not only does it kill it, it starts dividing, creating a cancer-killing army inside the body. ... Downsides: “So far, it’s only blood cancer, it’s high technology, it’s customized therapy, it’s going to require major investment,” warns Clifford Hudis, president of the American Society of Clinical Oncology, who is nonetheless excited about the cells. The current CARTs kill not just cancer cells but any B-cell, the type of white blood cell that goes wrong in leukemia. Patients are likely to get injections of a protein that B-cells make, called gamma globulin, for the rest of their lives; if the treatment becomes popular there may not be enough gamma globulin to go around. ... “It’s a little early to know whether or not the remarkable results we’re seeing will show us whether these are the drugs we’ve been looking for or whether these are the first powerful signals that we’re headed in the right direction,” says Louis M. Weiner, the director of Georgetown University’s Lombardi Cancer Center . Though the cells are “amazing,” says Charles Sawyers, the past president of the American Association for Cancer Research and a Novartis board member, “what we don’t know is how broadly does this scale?”
In some ways, everything had changed, for Schadt now had four hundred people working for him, along with nine gene sequencers at his disposal and a supercomputer named Minerva in the basement. In other ways, however, he remained a guy in shorts, a guy whose face was always agleam in the light of his laptop, a guy whose office walls were decorated with a palimpsest of indecipherable equations. Most important, he remained a guy who never said no—who never rejected anything as impossible—and when he learned that a woman from Mississippi whom Esquire had written about eight years earlier had been told she had terminal colon cancer, Schadt looked up and said: "That's exactly the kind of patient we take." … It was, in the end, the reason he had come to New York. He probably didn't really need nine gene sequencers. He probably didn't even really need Minerva, because he could do supercomputing with Google and Amazon. But as both a lapsed molecular biologist and a lapsed Christian looking to establish a new faith, he needed something he had never had before. He needed patients. He needed someone like Stephanie Lee.
Patrick Soon-Shiong wants to turn cancer treatment upside down. On January 12, Soon-Shiong and a consortium of industry, government, and academia announced the launch of the Cancer MoonShot 2020, an ambitious program aiming to replace a long history of blunt trial-and-error treatment with what amounts to a training regimen for the body’s own immune system. That system, Soon-Shiong argues, is perfectly adept at finding and eliminating cancer with exquisite precision—if it can recognize the mutated cells in the first place. Helping it to do so could represent a powerful new treatment for the disease, akin to a flu vaccine. ... Soon-Shiong has hit home runs before. This past July, one of his firms underwent the highest-value biotech IPO in history. A cancer drug he developed, called Abraxane, is approved to fight breast, lung, and pancreatic cancers in more than 40 countries. Soon-Shiong’s path from medical school in South Africa through residency in Canada, to UCLA professor, NASA researcher and corporate CEO has given him the bird’s-eye view necessary to take on a project this ambitious, as well as the resources to marshal the world-class computing and genome-sequencing facilities that it requires.
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- Repeat: Mosaic - What’s wrong with Craig Venter? 5-15min
Samumed is finding it easy to raise huge amounts of cash because it believes it has invented medicines that can reverse aging. Its first drugs are targeted at specific organ systems. One aims to regrow hair in bald men. The same drug may also turn gray hair back to its original color, and a cosmetic version could erase wrinkles. A second drug seeks to regenerate cartilage in arthritic knees. Additional medicines in early human studies aim to repair degenerated discs in the spine, remove scarring in the lungs and treat cancer. After that Samumed will attempt to cure a leading cause of blindness and go after Alzheimer’s. The firm’s focus, disease by disease, symptom by symptom, is to make the cells of aging people regenerate as powerfully as those of a developing fetus. ... Hood, 49, had invented a cancer drug that got his previous company, Targegen, bought by Sanofi for $635 million. He has a distinct take on drug development: He thinks everybody takes too many shortcuts and insists on doing work himself that other companies outsource, including formulating drug chemistry, testing drugs in laboratory animals and running clinical trials. ... The target Hood and Kibar went after was obvious: a gene called Wnt, which stands for “wingless integration site,” because when you knock it out in fruit flies, they never grow wings. It’s a linchpin in a group of genes that control the growth of a developing fetus–whether you’re a fly or a person. Together these genes are known as the Wnt pathway. Trigger the right ones and you might revive old flesh. Some cancers do their dirty work by hijacking Wnt, and blocking it might stop tumors.
The rise of immunotherapy hasn’t shifted that reality overnight, but it has sent a new jolt of energy into an age-old dream: that maybe, just maybe, medical science can turn terminal cancers into survivable conditions. ... In the past two years alone, the FDA has approved three second-generation checkpoint inhibitors, and two other arms of immunotherapy—cancer vaccines and a therapeutic approach known as adoptive T cell transfer, in which a patient’s own T cells are engineered outside the body and reinjected into the bloodstream—are showing ever-more-promising results. ... If immunotherapy leads the way to cancer cures in the coming decade, it’ll be tempting to look back on its development as inevitable, a breakthrough that was merely waiting for technology and biological research to make it possible. This would be true to some extent—scientists have hypothesized for over a century about the potential for the immune system to beat back tumors—but such a view would overlook the human choices and biases that shape the course of science. It would also overlook the power of small groups of individuals to spark major advances by bucking conventional wisdom and seeking out new frontiers. In other words, it would ignore the life of Jim Allison—a shaggy-haired, patchily bearded son of small-town South Texas whose creativity, diligence, and zest for pursuing a seemingly quixotic path far from the front lines of cancer research have added up to a revolution.
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Like a number of up-and-coming researchers in his generation, Nosek was troubled by mounting evidence that science itself—through its systems of publication, funding, and advancement—had become biased toward generating a certain kind of finding: novel, attention grabbing, but ultimately unreliable. The incentives to produce positive results were so great, Nosek and others worried, that some scientists were simply locking their inconvenient data away. ... The problem even had a name: the file drawer effect. ... The aim was to redo about 50 studies from three prominent psychology journals, to establish an estimate of how often modern psychology turns up false positive results. ... He wasn’t promising novel findings, he was promising to question them. So he ran his projects on a shoestring budget, self-financing them with his own earnings from corporate speaking engagements on his research about bias. ... researchers involved in similar rounds of soul-searching and critique in their own fields, who have loosely amounted to a movement to fix science. ... The problem, they claim, isn’t that scientists don’t want to do the right thing. On the contrary, Arnold says he believes that most researchers go into their work with the best of intentions, only to be led astray by a system that rewards the wrong behaviors.
The men and women who are trying to bring down cancer are starting to join forces rather than work alone. Together, they are winning a few of the battles against the world's fiercest disease. ... It's not like you don't have cancer and then one day you just do. Cancer—or, really, cancers, because cancer is not a single disease—happens when glitches in genes cause cells to grow out of control until they overtake the body, like a kudzu plant. Genes develop glitches all the time: There are roughly twenty thousand genes in the human body, any of which can get misspelled or chopped up. Bits can be inserted or deleted. Whole copies of genes can appear and disappear, or combine to form mutants. ... Cancer is not an ordinary disease. Cancer is the disease—a phenomenon that contains the whole of genetics and biology and human life in a single cell. It will take an army of researchers to defeat it.