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The Medical Issue

Boston breakthroughs

Treatment, ideas, and research.

Harvard Bioscience

SYNTHETIC WINDPIPE

It sounds like science fiction: Scientists harvest a man’s stem cells, build him an artificial organ, and save his life. But that’s exactly what happened to a young man who was suffering from advanced tracheal cancer. In June, Andemariam Teklesenbet Beyene received the world’s first tissue-engineered synthetic trachea, thanks to doctors at Karolinska University Hospital in Stockholm who used technology developed by Harvard Bioscience. “He was given two weeks to live,” says David Green, the company’s president. Today “he’s alive and well.”

The trachea was created by seeding Beyene’s own stem cells onto a Y-shaped scaffold inside a bioreactor designed by the Holliston-based biotech. In just two days, the new trachea was ready. Because the organ was grown from Beyene’s own cells, his body is unlikely to reject it – a major risk for patients who receive donor tracheas from cadavers. In addition, he doesn’t need immunosuppressant drugs, which increase the chance of infection. (They also cost at least $10,000 a year and are taken for life.) Green says the technique may someday be suitable for all hollow, tubular organs, including blood vessels and the esophagus. And he hopes regeneration of more complex organs will follow.

Vertex Pharmaceuticals

HEPATITIS C PILL

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Hepatitis C is an insidious little bug. By the time it was discovered in 1989, the virus had already been spreading silently for decades – most patients experience no early symptoms. The Centers for Disease Control and Prevention now estimates that 3.2 million Americans are chronically infected with hep C, which causes liver inflammation and can lead to liver damage, liver failure, and liver cancer. Every year, 8,000 to 10,000 people die from related liver disease.

For years, drug manufacturers have been searching for treatments. Enter Incivek, the culmination of 15 years of research by Vertex Pharmaceuticals in Cambridge. The pill, approved by the US Food and Drug Administration in May, blocks an enzyme that allows the virus to multiply in the blood and is the second in a new class of drugs to enter the hep-C market (Merck’s Victrelis was also approved in May). Until now, cure rates stood at about 40 percent for the most common strain of hep C, says Cami Graham, Vertex’s vice president for global medical affairs. With the new drug, she says, “we can cure about 80 percent of people who’ve never been treated.”

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A CURE FOR THE COMMON COLD?

Todd Rider was in the shower 11½ years ago when the light bulb went off: What if scientists could engineer a compound with two parts – one that would hunt down viruses in cells, the other that would trigger those cells to commit suicide? In a research paper published in July, Rider, a senior staff scientist at MIT’s Lincoln Laboratory, and his colleagues showed that they could destroy 15 different viruses in a petri dish using DRACO, a drug that was developed based on Rider’s idea. The researcher vividly recalls the day he saw DRACO annihilate rhinovirus. “I was calling everybody into the lab and saying, ‘Look at the microscope! I have a cure for the common cold!’ ”

The drug not only has the potential to knock out a wide array of bugs, it also remains effective even if a virus mutates and will not harm cells where no virus is present – an obvious safety concern. He and his team looked for toxicity during testing in human cells and in mice. So far, nothing. “It’s an absolutely fascinating concept,” says Dr. William Schaffner, an infectious disease expert and chairman of preventive medicine at Vanderbilt University’s School of Medicine. “Wouldn’t it be wonderful if it worked in larger animals and eventually in humans?” Stay tuned.

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ER LOCATOR APP

Need to find the nearest ER pronto? It’s a tap away for smartphone users, thanks to the free “findER” app released in 2010 (and updated this month) by the Emergency Medicine Network at Massachusetts General Hospital. There are nearly 5,000 emergency departments nationwide, and every year, 30 to 50 open and another 30 to 50 shut down, says Dr. Carlos Camargo, the network’s director. This app makes finding them easy.

SAFETY SCHOOL

Since its launch 20 years ago, the Cambridge-based Institute for Healthcare Improvement (IHI) has been at the forefront of reducing medical errors and improving patient safety, mostly through hospital-based initiatives. But how could the institute expand its reach? In 2008, it launched the IHI Open School online. The program offers such courses as “Introduction to the Culture of Safety” and “Teamwork and Communication.” In just three years, it has enrolled close to 70,000 students and medical residents, many of whom have started campus IHI chapters. “They get together to try to drive change in their own organizations,” says IHI’s Dr. Donald Goldmann.

AN EARLIER AUTISM TEST

With autism, say experts, the sooner the condition is diagnosed, the better the prognosis. But identifying autism in young babies is difficult because many of the telltale symptoms, such as language delays, don’t show up early on. Now, a new brain test conceived by researchers at Children’s Hospital Boston could help change that.

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Studies show that about 1 in 5 siblings of children with autism will also develop the disorder. William Bosl, a neuroscience researcher at Children’s, and his team studied infants who had an older sibling with autism and measured the electrical activity in their brains. Researchers were able to document patterns in female patients at 6 months that distinguished high-risk infants from controls with 80 percent accuracy. Researchers could tell the boys apart 100 percent of the time by 9 months. The test can’t diagnose the disorder, but knowing so young that children are susceptible can be a huge step forward.

AP/Brigham and Women's Hospital, Lightchaser Photograhy

FULL FACE TRANSPLANTS

Dallas Wiens, of Fort Worth, was painting a church in 2008 when his head hit a live electrical wire. The horrific accident obliterated the features on his face, leaving the 26-year-old blind and seriously injured. After nearly two dozen earlier surgeries, he traveled to Brigham and Women’s Hospital this March, where he became the first patient in the country to receive a full face transplant. Wiens says he was motivated to undergo the operation – it took a team of 30-plus doctors, nurses, and anesthesiologists more than 15 hours – for his 4-year-old daughter, Scarlette. “She calls me handsome,” he says. “She’s very happy with my new face.”

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Since Wiens’s operation, the hospital has completed two more full face transplants. And Wiens says he is doing well. “I can feel my lips and I can feel a kiss. I can smile. I can almost wink with my right eye.”

SURGERY-FREE LIPO

Liposuction requires anesthesia and is an invasive procedure with significant recovery time. Now there’s a new option for patients looking to lose pounds with something more than diet and exercise: chilling the fat away. The treatment, called Cool Sculpting, was invented by Dr. R. Rox Anderson and Dr. Dieter Manstein, dermatologists at Harvard Medical School and Massachusetts General Hospital, and by Zeltiq, a medical device company. It works by pulling skin and fat deposits into a vacuum-like chamber where it’s subjected to cold temperatures for about an hour.About three weeks after the procedure, which costs from $700 to $1,500 per treated area, patients notice less fat, which is reabsorbed by the body, says Anderson.

The procedure, which received FDA approval last year, isn’t for the overweight or obese; it works best on people who are within 10 pounds of their target weight. Dr. Melanie Grossman, a cosmetic surgeon in Manhattan, says she’s observed great results after treating small areas around patients’ tummies and backs. “It really works.”