joi, 2 aprilie 2015

Cold Spring Harbor Lab, Seeking Human Subjects, Teams Up With Hospital System



COLD SPRING HARBOR, N.Y. — Some of the world’s finest scientists live in a former whaling village on the North Shore of Long Island, in a compound reminiscent of an army post.


In this pastoral setting, they have very self-consciously taken themselves out of the real world into a cerebral one, where they are searching for the genetic origins of cancer and the drugs to cure it.


But despite the many breakthroughs that have taken place here, at Cold Spring Harbor Laboratory, there is one thing that has been lacking: human subjects.


To remedy that shortcoming, the lab is about to embark on a new collaboration between pure science and clinical science with North Shore-Long Island Jewish Health System, a nearby hospital system that can offer a wealth of patients to study.


“The hardest part of doing clinical research is enrolling patients in clinical trials,” said Dr. Kevin J. Tracey, chief executive of the Feinstein Institute for Medical Research at North Shore-L.I.J. “You have to have lots and lots of patients.”


North Shore-L.I.J., which has 19 hospitals spread through Long Island, Queens, Manhattan, Staten Island and Westchester County, offers a genetically diverse patient base in the millions with different racial and ethnic backgrounds. It diagnoses and treats cancer in more than 16,000 new patients a year.


Dr. Bruce Stillman, chief executive of Cold Spring Harbor lab, recalled arriving there in 1979 to work as a postdoctoral fellow under James D. Watson, co-discoverer of the double-helix structure of DNA, when “we were still trying to figure out what cancer was.”


Dr. Stillman said he began seriously considering the hospital collaboration about a year ago, because he was frustrated by not being able to move quickly from preclinical studies of cancer in the lab to the next stage of trying out therapies in people.


He approached the idea with some trepidation about whether it would lead the laboratory in the wrong direction.


“I didn’t want to dilute the basic science and become applied research,” he said. “Yet the opportunities were just screaming out.”


From the hospital system’s point of view, an affiliation with an institution known for its Nobel laureates is good branding.


Much cancer research is now focused on matching treatments to the genetic makeup of a tumor or even a patient. Cold Spring Harbor Laboratory is a pioneer in using three-dimensional methods to culture tumor cells, as opposed to the conventional two-dimensional cultures many laboratories use. In these three-dimensional cultures, tumor cells form beach-ball-like structures of hundreds of cells called organoids, which better mimic the way cancer functions in real life. Its scientists also study cancer tumors in mice.


Still, there are limits even to such advanced techniques. When scientists find a genetic target in the lab, and a drug that might work on it, Dr. Stillman said, “we need to understand whether it’s really working in the patient.”


More than $120 million will be invested in the collaboration over the next 10 years. Over time, Dr. Stillman said, the lab will build up a research unit in the hospital that will parallel the one it has for animals at Cold Spring Harbor. The closest comparison, he said, would be with academic medical centers like Massachusetts General, Dana Farber, Memorial Sloan Kettering and Johns Hopkins. But, he added, “It’s very rare to have scientists working with clinicians and suggesting what the clinicians do.”


One advantage of having human subjects is that some of the most interesting advances in medicine have come through serendipity — from discovering that a drug that fails for one disease is actually effective in another.


In a cluster of relatively new buildings designed to echo the old whaling architecture, researchers are working on cures for all kinds of cancers. Lindsey Baker, 31, a postdoctoral fellow in cancer biology, said she was excited by the new collaboration because cancer drug trials were often done in elite institutions where most of the patients were white and affluent. North Shore, she said, offers the opportunity to look at other racial and economic groups.


There are also practical advantages to working with a nearby hospital, she said, since the faster a tissue sample is delivered, the healthier it is and thus the better for research.


Although researchers will not know the identities of the patients who have given tissue samples, if a breakthrough discovery were made, it would be possible to retrace the identity of the donor and perhaps apply the new knowledge to that person’s treatment.


Dr. Chris Vakoc, who is studying acute myeloid leukemia, a blood cancer, is one of the resident scientists. His house, its porch littered with children’s toys, overlooks the harbor. He said clinicians often had a more intuitive sense of what worked in their patients than pure scientists.


“We tend to always think about things in a molecular way,” Dr. Vakoc said. “It’s very different treating a patient. Sometimes it’s kind of humbling to hear them say, ‘I doubt this is going to work.’ ”




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