Monthly Archives: September 2014

REPOST: Biotechnology to the Rescue

Developments in biotechnology have continually transformed medical practice and have changed lives. US News Health lists some of the most innovative and life-changing biotech products in the article below.

Wake Forest University's<br /><br /> regenerative medicine lab uses 3-D printing to produce a prototype of a<br /><br /> kidney.

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The life sciences industry is in the middle of a historic boom, churning out new medical weapons at an unprecedented pace. In 2012, the Food and Drug Administration approved 39 “new molecular entities,” completely novel drugs to treat everything from cancer to tuberculosis to HIV. It topped that off by adding 27 more last year, and there are now more than 4,000 investigational medicines in the pipeline.

Medical device-makers are also in high gear; the 33 products approved in 2013 include replacement hips, new cardiac stents and prosthetic spinal discs. Here’s a sampling of the most innovative developments transforming medical practice and offering patients new hope:

Brain boosters

When the complex communication circuits go awry, the result is a range of neurological disorders, including epilepsy, Parkinson’s disease and paralysis. Now researchers are figuring out how to manipulate neurological signals using tiny implants that, in essence, reset the brain. By targeting very precisely the region where seizures and tremors originate, for example, these minicomputers – sometimes in conjunction with drugs, sometimes on their own – can eliminate debilitating symptoms at their source.

Among the people already benefiting is Janie Norman, 43, of Marietta, Georgia, who was diagnosed with epilepsy at age 11. Norman was able to attend college, get married, and start a family, but she had such frequent seizures that she couldn’t get a driver’s license, go to the movies or grocery store on her own, or play a sport – all were too risky.

Then neurosurgeons at Emory University Hospital in Atlanta placed a tiny implant in her brain that detects the abnormal brain signals that usually precede seizures and responds by delivering short electrical pulses to stop the seizures before they start. Norman has been seizure-free ever since. “It’s a miracle,” she says. “I got my driver’s license. I can take my children to the park. It has really changed my life.”

The NeuroPace RNS System, as the device is called, was approved last year and has been adopted by more than 20 clinics specializing in epilepsy. It also features tiny programmable processors that store and transmit information about what exactly is happening in patients’ brains. By waving a wand over her head, Norman can download the information and transmit it to her doctor. The physicians can then use the information to adjust the device remotely.

An explosion of research that aims to “map” the brain’s function has doctors who treat neurologic disorders predicting a bright future. “The hope is that if we can restore some normal activity, then the natural ability of the brain to use those circuits will keep diseases from getting worse,” says Michael Kilgard, a professor at the University of Texas—Dallas whose research aims to normalize brain activity by sending electrical signals to it via electrodes attached to the vagus nerve in the neck.

Other scientists are developing implants that may someday restore movement in paralyzed patients by repairing damaged connections that instruct the muscles. The brain is great at “reorganizing itself,” Kilgard says. “We’re just guiding it in the right direction.”

Bionic body parts

Whether a patient is an amputee who needs a prosthetic limb or an aging person in need of a new knee or hip joint, the fundamental wish is the same: that the new part will work just as well as the old one did. The device industry is answering the call with myriad new technologies.

In May, the FDA approved the first prosthetic arm that will use special sensors and electrodes to pick up signals transmitted from nearby muscles and translate them to control multiple joints. That will allow users to perform complex tasks, such as grasping small items.

The DEKA Arm System, which goes by the Star Wars-inspired nickname “Luke,” also has sensors in its fingers that can detect how tightly the hand is grasping an object. It communicates that information to the patient through a device touching the skin that vibrates lightly in response to a delicate grip and more intensely as the grasp grows tighter. So a person can shake hands and sense how tight his or her grip is, says Justin Sanchez, program manager in the biological technologies office of the Defense Advanced Research Projects Agency, which funded the arm system’s development.

Retired Chicago police detective John Duffy, who was injured in a cycling accident, wears a prosthetic that flexes in response to signals from his environment.

Prosthetic legs are becoming more lifelike, too. Retired Chicago police detective John Duffy, who lost his leg below the knee in a cycling accident, wears one of the new models that rely on hydraulic cylinders to flex the joints based on signals from a system of sensors and a microprocessor in the leg. As he walks, the sensors continually monitor environmental feedback and send signals to the microprocessor, which controls how much resistance the cylinders apply. The resistance varies according to how fast Duffy walks or whether he is on a flat surface, going up or down stairs, or heading uphill or downhill.

The ability to respond to information from the environment results in a near-natural walking pattern, says Steven Gnatz, medical director of physical medicine and rehabilitation at Loyola University Hospital in Maywood, Illinois.

Next up: Scientists are working on prosthetic limbs that convert brain signals – amputees’ thoughts about how they would like to move their prosthetic arms or legs – directly into movements.

Today’s smarter replacement joints are not quite as dramatic as bionic prosthetics, but with a rapidly aging population, the demand is certainly as strong. In response, the joint-replacement industry has been moving away from a one-size-fits-all approach and is offering artificial knees, in particular, designed according to gender, size and location in the left or right leg. (Hip joints are getting somewhat more customized, though the joint is less complicated, and the need is not as great.)

“The original knee replacements were not great fits,” says Steven Haas, chief of the knee service at the Hospital for Special Surgery in New York. “Now there are hundreds of combinations of sizes that allow us to more closely follow the natural anatomy.”

And with the help of magnetic resonance imaging, surgeons can offer even greater precision. They now obtain detailed pictures of a joint prior to surgery, then perform a dress rehearsal of sorts on a computer that helps ensure the best fit and allows adjustments in advance, “so when we do the implant, it will reproduce the natural motions of the knee,” Haas says. Surgeons also now have access to “smart” surgical instruments embedded with tiny computers that help align the joint.

Virus victories

Drugmakers have been mastering new techniques for battling viruses, in the process curing diseases that were once considered unconquerable, or inventing new vaccines to prevent them. “We’re starting to understand, on a fundamental level, how viruses enter cells, how they start the infectious cycle, and how they produce the genetic material to create progeny viruses,” says Richard Plemper, a professor at the Institute for Biomedical Sciences at Georgia State University.

The latest breakthrough came last December with the arrival of the drug Sovaldi, the first in the new generation of medicines to take a different tack against hepatitis C. The disease, long considered intractable, affects an estimated 185 million people around the world. In clinical trials, Sovaldi wiped out hepatitis C in 90 percent of patients, often in as little as 12 weeks.

The key to the success of these new drugs is that they inhibit the ability of viruses to invade healthy cells and to hijack the cellular machinery they use to make endless copies of themselves. Sovaldi blocks enzymes produced by the virus that control part of the replication cycle; it’s a similar approach to the one behind most of the successful HIV treatments. When used in combination with other drugs, enzyme inhibitors can prevent drug resistance, a common problem with other antivirals.

Sovaldi is just one example of how science’s ability to outmaneuver viruses is producing new solutions. In April, researchers at the University of Maryland, collaborating with Novavax, announced that they had developed a vaccine to prevent infection by the Middle East Respiratory Syndrome (MERS) virus, a new malady that had just broken out around the world. It works by preventing the virus from invading cells in the first place.

And scientists at Georgia State University, Emory University and the Paul-Ehrlich Institute in Germany say they have found a way to control measles outbreaks by disrupting the protein that replicates the viral genome. This approach could both cure the disease and prevent it from spreading to other people.

Gene therapies

The promise of gene therapy was always titillating: The technique offers the chance for patients with genetic diseases to replace disease-causing genes with healthy ones. But in 1999, an 18-year-old patient died when a virus-based mechanism used to transport a therapeutic gene into his body touched off a severe immune response. That single tragedy led to the demise of the field.

Now research into gene therapy is roaring back to life, thanks to a series of discoveries that have made the process of replacing faulty genes significantly safer. Scientists have perfected a number of gene-transport mechanisms, or “viral vectors,” that are benign because of improvements in the way they’re engineered and because they’re derived from viruses that aren’t infectious to people. In 2012, Amsterdam-based uniQure won approval in Europe for a product called Glybera marketed to treat an enzyme disorder that causes dangerous fatty acids to build up in the blood.

Although the therapy isn’t a cure, injecting sufferers with a corrected copy of the responsible gene has greatly reduced episodes of pancreatitis, a painful and dangerous consequence. And some patients who receive the therapy have been able to relax their restrictive diets.

Gene therapy is now being developed to treat a range of disorders, from hemophilia to Parkinson’s to a deadly neurological illness called Sanfilippo syndrome. Jerry Mendell, director of the Center for Gene Therapy at Nationwide Children’s Hospital in Columbus, Ohio, has made headway developing treatments for muscular dystrophy, which occurs when a defective gene fails to make a protein vital to normal muscle functioning or the gene is missing. In one recent trial, 12 patients who received a treatment to correct the defective gene saw measurable improvement in their expected walking ability. “Is it a cure? No. But it’s a notable step forward,” Mendell says. “I really believe we will continue to see important advances in this field.”

Miniaturized medicine

Much of the quest to improve medical treatments has focused on making them smaller – much, much smaller – so they can travel through the body delivering therapies precisely where needed, or so they can gather intelligence on what’s going wrong in organs and tissues.

Nanotechnology is making a particularly strong impact in drug development, where biotech companies and university scientists are devising ways to pack powerful medications into microscopic particles that can zoom through the bloodstream to therapeutic targets without damaging healthy tissues. One of the biggest successes so far is Abraxane, a chemotherapy drug encased in nanosized protein-based packages that is widely used to treat breast, pancreatic and lung cancers.

One goal is to use nanotechnology to reach into the brain. This “secluded organ” is separated from the body by the blood-brain barrier, a series of ultranarrow capillaries that prohibit many drugs from passing through, says Alexander Kabanov, a professor and director of the Center for Nanotechnology in Drug Delivery at the University of North Carolina—Chapel Hill. Kabanov’s group has developed nanosized spherical containers that can cross the barrier and that he believes may prove useful in treating neurologic disorders such as Parkinson’s disease and stroke.

Diagnostic devices, meanwhile, are simultaneously shrinking in size and growing more powerful. A prime example is PillCam, a camera encased in a vitamin-sized capsule that can take a series of high-quality pictures as it travels through the digestive system. Ever since the first PillCam hit the U.S. market in 2002, each new generation has improved with the invention of tiny imaging chips that now can take as many as 35 frames per second and transmit them wirelessly to a recording device the patient wears, says Greg Daevault, vice president of global market development for Dublin, Ireland-based Covidien, which makes PillCam.

The device is now used to diagnose diseases of the colon and bowel. Covidien is currently looking at adding nonimaging sensors to the PillCam that might allow it to detect, say, traces of blood or changes in acid levels that might indicate early signs of disease.

Man-made organs

What if rather than using medicine or genes – or an organ donor – to treat a failing heart or liver, your doctor could simply replace those parts with organs that are nearly identical to the originals, made from your own cells? That dream is not as far-fetched as you might think.

Much of the revolution in regenerative medicine is being driven by 3-D printing, the use of specialized machines that can create tissue and organlike structures from a patient’s cells. Among the body parts now streaming from 3-D printers are blood vessels, livers and skin to heal wounds. At the Wake Forest Institute for Regenerative Medicine in Winston-Salem, North Carolina, researchers are using 3-D printing and other techniques to engineer more than 30 different replacement tissues and organs, including bladders, kidneys and, most recently, vaginas made from the cells of girls suffering from a rare genetic condition that causes reproductive organs to be underdeveloped.

“Our preference is to [use] the patients’ own organ-specific cells because they’re less likely to get rejected,” says Anthony Atala, director of the Wake Forest center.

In New York, Jason Spector, associate professor of surgery at Weill Cornell Medical Center, and Lawrence Bonassar, associate chair of the biomedical engineering department at Cornell University, are now developing ears for patients who have been in accidents or who suffer from disfiguring diseases. “We use 3-D photography to capture a very high-fidelity picture of the [intact] ear, so we can make a perfect match,” Spector explains.

“That photograph is then digitized, and the data is used to create a mold. Then we can take cartilage cells from cow’s ears and inject them into the mold,” resulting in an ear that looks and bends just like the real thing. Spector’s team has also invented a way to print ears from 3-D photos, using a protein-based gel that hardens into an earlike structure. And they’re testing methods of using patients’ own cells to create the ears.

Although most of these technologies are still in clinical trials, some patients have reaped the rewards of the research. Luke Massella, 23, has spina bifida and, after his bladder failed at age 10, received a new one made in Atala’s former lab at Harvard. Massella is healthy today, and is working as a special-education paraprofessional for a middle school in Madison, Connecticut. “The bladder functions as my own,” he says. “I don’t even need yearly checkups.”

Janique Goff is a successful business development manager who supports start-up businesses in the biotechnology industry. Add this Google+ account to your circles to get the latest updates in biotechnology.