But a new type of polymer gel that mimics key traits of human vocal cords may provide a remedy. The material, known as polyethylene glycol (PEG), appears to act like a natural vocal cord that also restores vibration to vocal cords that have stiffened due to scarring. PEG is used in certain other medical devices that FDA has approved.

“Currently, there is no solution to this problem and it is highly unlikely that, at least in the foreseeable future, a drug will be developed that can create a biological response,” notes Robert Langer, PhD, a professor in the Department of Chemical Engineering at MIT. Dr. Langer has worked closely with Steven Zeitels, MD, FACS, a professor of laryngeal surgery at Harvard Medical School, to advance the technology.

Sandeep Karajanagi, PhD, an instructor of surgery at Harvard Medical School and a researcher at the Center for Laryngeal Surgery and Voice Rehabilitation at Massachusetts General Hospital, developed the gel while working as a postdoctoral researcher at MIT. Although it degrades over time—and those with vocal cord problems would require a re-injection every 6 months or so—it could significantly change the way doctors treat vocal cord maladies.

Many who suffer from vocal cord problems are children whose cords were scarred from intubation during surgery or people with laryngeal cancer. However, a significant segment of society, including teachers, actors, clergy, trial attorneys, politicians and singers, wind up with voices strained from overuse. “We live in a society in which 90 percent of jobs require substantial voice use,” Dr. Zeitels says.

Human trials will begin in 2012, and Dr. Zeitels believes that a commercially viable product is 3 to 6 years away. For now, researchers are studying how to fine tune the biomechanical properties of the gel while conducting clinical trials. Concludes Dr. Zeitels: “This would be so valuable to society because every time a person loses his or her voice they are unable to communicate ideas and their contributions are lost.”

CONNECT THE DOTS

For more information about throat, larynx and voice problems visit The Institute of Laryngology and Voice Restoration (ILVR) and the National Institutes of Health site Disorders of Vocal Abuse and Misuse. If you rely on your vocal cords for your livelihood, read these tips from the University of Texas Health Science Center concerning general voice care and vocal hygiene.

Tuberculosis (TB) is an airborne infectious disease that is preventable and curable, yet the bacteria is carried by a third of the world’s population, concentrated in resource poor regions of Africa and Asia. For nine million people each year, the disease goes “active” causing them to become sick, unable to work and at risk of infecting friends and family members. The lung disease is rarely contracted from a single exposure to a stranger who is infected, but from repeated exposure to someone with the untreated infection when they cough, speak, sneeze, spit, laugh or sing –releasing microscopic droplets of water into the air – which increases the chance of contracting the disease. With two weeks of appropriate treatment, however, the widespread disease is no longer contagious.

Yet, one of the major obstacles to reducing TB is completing the full course of drugs. Many of us cannot remember to take all of our antibiotics for a week, yet typical antibiotic regimes for TB last from six to nine months. Once patients feel better, their compliance rate falls. With partial doses of the antibiotic regime in the system, the bacterium become increasingly drug resistant, boosting its ability to survive future drug therapies and passing along its drug resistance to those new patients who become infected.

To boost compliance, the current protocol recommended by the World Health Organization is called DOTS, Directly Observed Treatment Short course, where patients take their medicine under the watchful eye of an appropriate care giver. For many, however, that may be difficult for a variety of logistic, personal, economic and social reasons as well as the physical challenges brought on by side effects of taking chemotherapy drugs.

A new penny-a-day urine test comes to the rescue. A study published in The Lancet, said that a new program called X out TB showed promise. Since more than 1/2 of people in developing countries now own cell phones.  Using the technology people want to incentivize them to take the drugs they need may be a perfect match. The idea sprang from a challenge to MIT students to come up with a possible solution to TB compliance. The team developed X out TB, paper test strips that reveal a unique code when urinated upon if the patient has taken their medication. The patient then texts the code in and receives a small incentive. The plan was field tested in Nicaragua, using the newly developed testing strip, a device that would dispense one strip every 24 hours, and a simple text message reporting system.  The results appeared promising.

The program has moved to Karachi where the Stop TB Partnership will roll out the X out TB program. “Currently, the system is still in the development stage, says Shama Mohammed, Co-Director of Program Development and Interactive Research and Development for the Indus Hospital Research Centre in Karachi. “Testing is being done with patients using a very basic form of the strips without the code to determine and improve on the sensitivity of the strips. The complete system with mobile phones and incentives has not been piloted, so it is too early to comment on its use and impact. Once developed, I believe a system like this should be very beneficial to patients and should improve compliance rates.”

Connect the Dots

Visit here to learn about new paradigms in global TB prevention.  You might also be interested in these related healthymagination articles: “Research Finds Faster Treatment for Latent TB,” “Tuberculosis: New Weapons Against an Old Disease,” and “Social Media Lends Haiti a Hand.”

One of the most common types of blood cancer in the United States is also one of the most curable. But despite the high rate of remission of Hodgkin lymphoma (HL), for the small percentage of people who do not respond to standard treatment there has been little hope – until now.

In August, the Food and Drug Administration (FDA) approved Adcetris (brentuximab vedotin), the first new drug approved to treat Hodgkin lymphoma (HL) in the last 30 years.  For those whose disease has remained resistant to standard treatment, this drug appears to offer remarkable promise for both HL and systemic anaplastic large cell lymphoma (ALCL), a non-Hodgkin lymphoma (NHL). “These two types of cancer typically have a higher incidence in young adults and both tend to be responsive to standard care,” says Hildy Dillon, Senior Vice President of Patient Services for The Leukemia & Lymphoma Society. “Hodgkin lymphoma has an 86 percent survival rate with standard treatment, ALCL has a 54.5 percent five-year survival rate.  But for people who don’t respond well to the standard course of therapy then they become very difficult to cure.”

According to the National Cancer Institute, approximately 8,800 people were diagnosed with HL in 2011 with over 1,300 not responding to treatment; another 66,360 were diagnosed with NHL in 2011 with almost 20,000 failing to respond to treatment. About 1,600 were diagnosed with ALCL.

Cancer is primarily treated through radiation and chemotherapy, but because of the often-debilitating side effects and potential long-term risks, researchers continue to pursue new therapies to attack the disease. Both chemo and radiation kill cancer cells, but they also kill healthy cells in the process resulting in hair loss, weight loss, nausea and fatigue. Because of its positive results on the most difficult patients, along with reduced side effects, the new drug is being studied as an option for those beginning their chemotherapy treatments, but for now, it is only approved for those who have tried standard treatments and not responded. “This is a small population that fits into this category,” says Dillon. “Most are in the prime of their lives, and the new treatment gives hope to these patients who didn’t really have options until now.”

One of the most promising new cancer treatments uses drugs that mimic the body’s natural kamikaze warriors. The immune system puts out protein antibodies that find and destroy infections, tailoring each one to seek out and annihilate a specific target, typically a protein that is attached to the surface of a foreign cell. Scientists copied this process and created man-made monoclonal antibodies that can target cancer cells. Some can kill the cells on their own; others transport radioactive molecules that poison the cell once they burrow inside.

Because they can more sharply target the cancer cells and ignore the healthy cells, these drugs may have fewer side effects.  Monoclonal antibodies can be used alone or with other chemotherapy drugs. The recently approved drug doesn’t directly target cancer cells, instead it carries a cancer-fighting drug into a protein (CD30) that frequents the surface the cancer cells associated with Hodgkin lymphoma, but that are found on less than one percent of healthy cells.

In a clinical trial, Adcetris was used to treat high-risk patients whose Hodgkin lymphoma had returned after other treatments, including stem cell transplantations, had failed. Of the 102 patients who participated in the clinical trial, 75 percent saw their tumors reduced to half their size. Almost a third experienced a complete remission with another forty percent seeing a partial remission. “If they respond to Adcetris, they can go on to receive a stem cell transplant,” Dillon continued, “or for patients who have failed stem cell, this treatment may prolong their life.” Of the 58 ALCL patients who had previously undergone multi-agent chemotherapy regimens, 86 percent responded positively to Adcetris with 53 percent attaining complete remission.

“These are very complicated diseases,” said Dillon, “so it is really exciting to see a win like this.”

CONNECT THE DOTS

Read the FDA’s complete announcement about the approval of Adcetris. Learn more from the American Cancer Society about how monoclonal antibodies are being used in cancer treatment.  You might also be interested in reading these healthymagination posts: “Nanoparticles May Revolutionize Future Cancer Treatment,” “Scientists Make Progress on Ovarian Cancer Treatment,” “Breakthroughs to Beat Cancer,” and “GE Launches $1 Billion Campaign to Speed Fight Against Cancer.”

The journal Science named HPTN 052 the 2011 Breakthrough of the Year and said, “The results have galvanized efforts to end the world’s AIDS epidemic in a way that would have been inconceivable even a year ago.”  The study results, published in The New England Journal of Medicine, were released years ahead of schedule because the results were so compelling: treating HIV-infected people with immediate antiretroviral therapy (ART) reduces the risk of transmission by an astounding 96%.

The treatment-as-prevention strategy has the potential for a one-two punch: keeping infected people healthy and helping keep their partners HIV-free. Dr. Julio Montaner says, “Study HPTN 052 conclusively shows that when an infected person receives ART, the ability of the virus to replicate is shut down immediately. At that point, the virus becomes almost undetectable in the blood, and as immunity recovers, the person leads a near-normal life and both AIDS and death are potentially prevented.” Dr. Montaner is Director of the British Columbia Centre for Excellence in HIV/AIDS and Past-President of the International AIDS Society.

The large-scale randomized clinical trial measured the impact of HIV treatment in 1,763 couples (where one partner was HIV positive) in 13 sites across nine countries including Asia, Africa, and the United States. Originally designed to measure the difference between early and delayed ART, the study was halted years ahead of schedule after results showed that early treatment was cutting the transmission rate so dramatically.  Myron S. Cohen, MD of the University of North Carolina at Chapel Hill, Institute for Global Health and Infectious Diseases led the study, which was funded by the National Institutes of Health and conducted by the HIV Prevention Trials Network.

Right now there are 34 million people around the world who are living with AIDS – 27 million are located in Sub-Saharan Africa and South and South-East Asia. Yet only about 6.65 million are currently receiving treatment.

The tide has turned in the war against HIV/AIDS. The conclusive evidence from HPTN 052 gives the world an extraordinary opportunity to move forward and work on eradicating the disease for good. In his editorial comment in The Lancet, Dr. Montaner stated, “The evidence is in: treatment is prevention. Treatment dramatically prevents morbidity and mortality, HIV transmission, and tuberculosis. The challenge remains to optimize the impact of this valuable intervention. Failure to do so is not an option.”

CONNECT THE DOTS

Read our earlier post about study HPTN 052, “World AIDS Day: Treatment as Prevention to Halt HIV/AIDS Epidemic.” Watch a video where U.S. Secretary of State Hillary Rodham Clinton speaks about “Creating an AIDS-Free Generation.” For more news on HIV/AIDS, read our blog posts: “Gamers Solve Puzzle in HIV/AIDS Research that Stumped Scientists,” “Anti-HIV Gel Promising Prevention Method for Women,” “Rapid Testing Technology Could Slow the Spread of AIDS,” “HIV Prevention: One Penny Saves One Life,” and “New Computer Modeling Analyzes Drug Therapies for HIV Patients.”

In a new report, the FDA cites “expedited approval” pathways and streamlined clinical trial requirements as factors that enabled it to review important new drugs quickly and efficiently, with nearly 70 percent of the new medications approved in the US before any other country in the world. Among the most important breakthroughs are:

* Seven major advances in cancer drugs. Two of the new cancer drugs, Zelboraf (vemurafenib) for late-stage melanoma skin cancer and Xalkori (crizotinib) for late-stage lung cancer, are breakthroughs in personalized medicine, according to the FDA, as each were approved with genetic tests which will allow treatment to be targeted to patients it is most likely to help. Another cancer breakthrough, Zytiga (albiraterone acetate), is the first in a new class of drug to treat late-stage prostate cancer.

* Ten new drugs for rare or “orphan” diseases. These disorders often lack any approved therapy because of the relatively small number of people suffering from these conditions. Among these drugs are Caprelsa (vandetanib), the first drug approved to treat medullary thyroid cancer, a rare form of thyroid cancer estimated to affect 1,300 to 2,200 patients in the US, and Adcetris (brentuximab vedotin) is the first new FDA-approved treatment for Hodgkin’s lymphoma since 1977 and the first drug to treat a rare type of lymphoma called systemic anaplastic large cell lymphoma (ALCL). Both forms of lymphoma are considered orphan diseases.

* Two new drugs for hepatitis C. Globally, an estimated 170 million people are infected with chronic hepatitis C, which can lead to such dangerous complications as liver failure, liver cancer, and cirrhosis. The disease ranks as the leading cause of liver transplants in the US. Victrelis (boceprevir) is the first drug for hepatitis C to be approved in its class. The second drug in this class, Incivek (teleprevir), was approved 10 days after Victrelis. According to the FDA, both drugs offer “a greater chance of cure” for some patients’ hepatitis C infection, compared to previously available therapies.

* New treatments to help prevent heart attack and stroke. Pradaxa (dabigatran etexilate) is the first new oral anticoagulant (blood-thinner) approved since warfarin in the 1950s to reduce risk for stroke and blood clots in people with atrial fibrillation, a common type of abnormal heart rhythm. Brilinta (ticagrelor) has been shown to lower risk for heart attack and cardiovascular death in people with acute coronary syndromes.

* A new weapon against MRSA infections. Teflaro (ceftaroline fosamil) is an injectable antibiotic to treat bacterial skin infections, including those triggered by MRSA (methicillin-resistant Staphylococcus aureus), and to treat community-acquired bacterial pneumonia.  MRSA is a so-called superbug that is resistant to treatment with traditional antibiotics and can be serious or life-threatening, so it’s important to have a new antibiotic to combat it.

* The first treatment for scorpion stings. Severe scorpion stings, which are most likely to occur in babies and kids, can cause a wide range of serious symptoms and can be life-threatening if untreated. Anascorp (Centruroides [Scorpion] Immune F(ab’)2 [Equine]) is the first specific treatment approved in the US for this dangerous condition.

CONNECT THE DOTS

For more news about new drugs, check out our blog posts, “Biotech Firm Wins FDA Approval for C. Difficile Antibiotic,” “First New Lupus Drug in 50 Years: Benlysta Gains FDA Approval” and “FDA Approves Daliresp: New Drug for COPD.” To learn more about molecular testing advances and tools that can help doctors better target therapies for cancer, read, “Is My Cancer Different?” Also take a look at “GE Launches $1 Billion Global Campaign to Speed Fight Against Cancer.”

Millions of Americans who suffer from major depression are prescribed antidepressant medication that doesn’t offer them relief, or results in intolerable or disagreeable side effects.

A new therapy, Transcranial Magnetic Stimulation or TMS, which uses highly focused magnetic pulses to stimulate key neurons in the brain that control mood, has been an effective alternative for patients in whom anti-depressant drugs have been ineffective.

“Some people feel like it’s a miracle in their lives,” says Dr. Alan Manevitz, a clinical psychiatrist who practices in New York City and a clinical associate professor of psychology at Cornell University. He was the first doctor in New York State to administer the treatment that is gaining increasing attention in the United States.

“It’s really quite exciting to be able to offer what almost feels like a Star Trek device. Patients sit in a chair, read the newspaper, watch television or talk to our technicians and they go from these really sad stories of not functioning and feeling overwhelmed by their depression to hope.”

Patients come weekdays for about 20 to 30 daily non-invasive treatments, which last about 40 minutes, Manevitz says. After about six weeks, more than half of his patients report significant improvement in their depressive symptoms, and one-third of patients experience a complete remission.

A study published the October 2010 edition of Brain Stimulation showed that TMS keeps patients with major depressive disorder on antidepressants from relapsing within six months.  Long term persistence of this benefit, however, has not been thoroughly studied.

Without insurance, the magnetic stimulation costs about $10,500, but many insurance plans now cover it.  However, approval may require an appeal, Manevitz says. In general, patients also must have experienced at least one unsuccessful treatment to get insurance approval for TMS.

The TMS has been used to treat other brain conditions for nearly 10 years, but the procedure was cleared by the Food and Drug Administration in December 2008 for Neuronetics to market it for use in treatment of major depression.  It also has recently been included in the American Psychiatric Association’s depression treatment guidelines.

“Because it’s administered by the treating physician, the patient is not responsible for administering the treatment,” Manevitz says. “We observe the treatment, so there is no under dosing, inadequate treatment and there are no systemic side effects such as weight gain, sexual side effects or energy depletion.”

CONNECT THE DOTS

For more information about major depression, visit the World Health Organization, PubMed Health or Wed MD.  You might also want to check out these earlier healthymagination posts: “Transcranial Magnetic Stimulation May Offer New Hope for Severe Depression” and “Deep Brain Stimulation for Depression.”

What would you do if the universe gave you a second chance? Matthew Lehrman is using his second chance to go to college. And while his mother will miss him when he leaves New York in September for Centenary College in Hackettstown, NJ, Roberta Lehrman is encouraging her son’s flight from the nest. “This,” she says, “is his opportunity to feel he’s just like everybody else.”

Matthew, however, is not like everybody else. His mother was afraid he wouldn’t live long enough to have a Bar Mitzvah, let alone attend college.

Shortly after his 7^th birthday, Roberta took Matthew to a local emergency room, where a CT scan revealed what had been causing his escalating nighttime headaches and vomiting: medulloblastoma, a rapidly growing, malignant tumor located in the cerebellum. The 11-hour surgery that followed yielded even more devastating news: The cancer had spread to Matthew’s spine. “I thought he was going to die,” recalls Roberta. “That’s what went through my mind: he’s not going to make it.”

The prescribed course of therapy, which would have involved radiation to the brain and spine followed by several months of standard-dose chemotherapy, offered a gloomy 30% chance of survival. “That just wasn’t good enough,” says Roberta. If treatment failed and Matthew didn’t make it, she didn’t want to look back and wonder whether she could have done something more.

Finally, Roberta was referred to the Stephen D. Hassenfeld Children’s Center for Cancer and Blood Disorders at NYU Langone, considered one of the most innovative outpatient treatment centers in the country.

She decided to transfer Matthew there for treatment. The people at Hassenfeld “are an amazing team,” says Roberta. “They were very positive, very up.”

At the time, pediatric oncologist Sharon Gardner, MD, and her colleagues were pioneering “Head Start,” a revolutionary new treatment protocol for young children with brain tumors that aims to limit damage to the growing brain by avoiding or minimizes the use of radiation.

“Head Start” patients undergo daily, high-dose chemotherapy for one week out of every month for five months, followed by a sixth round of even more intensive chemo and an autologous stem cell transplant. Matthew’s disease was both aggressive and advanced, and doctors decided to pull out all the stops to try to save his life. In Matthew’s case, “we weren’t able to eliminate radiation,” explains Dr. Gardner. “But we were able to postpone it until he was a little older.”

Following each chemotherapy cycle at Hassenfeld, Matthew had to be hospitalized at Langone so doctors could manage his high fevers, low blood-cell counts and transfusions. After his stem cell transplant, Matthew underwent six weeks of highly targeted proton-beam radiation. “It was,” says Dr. Gardner, “a long siege. Without both pieces—very intensive chemotherapy and radiation–there’s a good chance Matthew wouldn’t be alive.”

Despite his grueling ordeal, Roberta says Matthew coped “amazingly well,” thanks largely to the care he received at Hassenfeld and NYU Langone. Now cancer-free, Matthew “is doing great,” says Dr. Gardner, who notes that the Head Start protocol has become standard for young children with brain tumors. “But he is obviously a little different than kids who didn’t go through what he went through.”

Matthew still struggles with balance problems, partial hearing loss and learning disabilities. But he refuses to let them rule. “I’ve learned to push hard and work,” he says. In June, Matthew graduated from Community High School in Teaneck, NJ, a specialized school for children aged 14–18 with learning disabilities “After having cancer, I never thought I would be able to do so well in school,” he says proudly. “The lowest I’ve ever gotten on my report card is a B.”

According to Hassenfeld’s director, Dr. Linda Granowetter, children who have survived cancer, often become “special, more empathic people.” In discussing them, she quotes Ernest Hemingway, who famously said, “The world breaks everyone, and afterward, some are strong at the broken places.”

Dr. Gardner believes that’s true of Matthew. “Matthew is an incredible inspiration for all of us,” she says. “Because of the tumor and the radiation and the chemo, his life isn’t easy. But he’s always looking for new experiences, and he doesn’t let it slow him down!”

CONNECT THE DOTS

Find out more information about pediatric brain tumors, including medulloblastoma, at the National Cancer Institute, the Pediatric Brain Tumor Foundation, and the Mayo Clinic.

Imagine living with an unknown genetic disease that causes excruciating pain just from walking. Consider what it is like to spend years undergoing tests at major institutions – places like Harvard, the Mayo Clinic, and others – and still leave doctors scratching their heads.

That is what happened to nine siblings, most in their forties and fifties, from three families. They were all suffering from ACDC, a newly discovered degenerative disease causing their arteries to harden with calcium deposits as thick as deposits found in water pipes. The disease had been a medical mystery until earlier last year, when researchers at the National Institutes of Health Undiagnosed Diseases Program (UDP) discovered that arterial calcification occurs due to a very rare deficiency in CD73, a protein that produces a molecule that prevents calcification.

The siblings still cannot walk more than short distances without pain, but the UDP has obtained FDA approval to administer drugs that could improve the patients’ conditions. These successes and others in the program were announced in the September 26, 2011 issue of the journal Genetics in Medicine.

The government-funded program, an offshoot of the National Human Genome Research Institute (NHGRI), began three years ago and is free to patients. More than 326 people have been accepted and of these, neurological disorders account for nearly half. Commenting on the high percentage of neurological cases, Dr. Cynthia Tifft, Deputy Clinical Director of NHGRI, says, “Many of the unsolved mysteries in medicine revolve around the brain.”

Most patients spend a week at the UDP clinic in Bethesda, Maryland, where they undergo a battery of diagnostic tests in one week. After the patients leave, the UDP researchers hit the lab and conduct meticulous tests looking at gene sequences in the patients’ DNA. First they must pinpoint the source of a defect and then prove that the mutation is actually causing the patient’s problem. Says Dr. Tifft, “We’re looking for a needle in a haystack. Because these diseases are new or extremely rare, it’s like conducting a research study for one person.”

Another case the researchers solved was that of a woman with a painful buildup of protein in her muscles. This turned out to be amyloidosis, a rare bone marrow disease. Armed with this knowledge, her doctors improved her condition with a stem cell bone marrow transplant.

UDP doctors hope that unlocking the mysteries of rare disorders will help in understanding and treating more common diseases, from osteoporosis to Alzheimer’s. Meanwhile, for the patients in the UDP, often there is no proven remedy. The doctors send patients home to their own physicians with recommendations for symptom relief.

There have been so many applicants for the UDP that it was closed to new patients for a while, but is now open for new applications. The caveats: Not everyone is accepted, a correct diagnosis is not always found, and there is a waiting list to begin the process. Yet for people who have exhausted all other options, the UDP may still offer hope.

CONNECT THE DOTS

To read more, visit NIH News. The New York Times had an excellent article as well. More information on how patients with longstanding medical conditions that elude diagnosis by a referring physician may be considered for UDP studies, go to NIH Office of Rare Diseases Research (ORDR) – Undiagnosed Diseases Program.

“So often we read about medical discoveries,” says Chris Coburn, the CCF’s executive director of innovations, “but reaching a critical mass stage, where the science and need and funding come together to create access for individuals, is what our process really does.  For the items on this list, the technology is no longer a question mark and access is opening up. We’re talking about tens of millions of lives.”

The winning innovations include:

Catheter-based renal denervation to control resistant hypertension. One in three American adults is said to have hypertension, or high blood pressure. For many of them, the condition is “resistant”—it doesn’t respond to medical treatment of fewer than four medications to control it. Renal denervation can lower blood pressure in a new 40-minute procedure using a catheter that delivers low-level radio-frequency energy to the kidneys — a development so momentous that the panel ranked it first among all innovations. “This technology is a game-changer,” Coburn says. “It will allow simple denervation to result in a permanent lowering of blood pressure and permanently remove drug requirements for the patient. It’s a great example of the power of technology.”

Concussion management system for athletes. Nearly 4 million sports and recreation related concussions occur each year, many of which never cause a loss of consciousness. That means athletes often return to their sports too soon, risking more serious injuries if injured a second time. New special management systems measure the athlete’s baseline cognitive and motor skills at the start of the playing season, and a special mouthguard that uses Bluetooth technology measures and reports impact data immediately following a blow to the head.

Active, wearable bionic prostheses for amputees. Prostheses of one kind or another have been around for centuries, but these models actually communicate. “Imagine walking on your hands and knees, with a block in one hand,” Coburn says. “You can’t feel the ground. That’s what prostheses have felt like in the past.” But a consequence of so many soldiers returning from Iraq and Afghanistan with missing limbs, he says, has been a dramatic advance in artificial limb technology. The newest limbs have sensors, he says, “just like skin. It tells you your foot is now touching the ground.”

Harnessing data to improve healthcare. It’s called “big data,” the 2.5 quintillion bytes of data created daily. Managing or even accessing data necessary to provide and coordinate healthcare can be overwhelming, but this year has seen new tools to do just that. “It’s bringing new efficiency to patient care at the individual level,” Coburn says, “and then aggregating it with billions of records. So on a single question that might have taken days to answer in the past, now can be addressed in seconds—or less.”

The remaining Top Innovations for 2012 are:

* CT Scans for detection of lung cancer

* Medical apps for mobile devices

* Next-generation gene sequencing

* Implantable devices to treat brain aneurysms

* SGLT2 inhibitors for diabetes therapy

* Genetically modifying mosquitoes to reduce diseases such as malaria, dengue and yellow fever

CONNECT THE DOTS

Visit the Cleveland Clinic’s website for more information on the Medical Innovation Summit and winning innovations. For more news about medical innovations, check out our blog posts: “GE’s $100 Million Challenge to Find and Fund Breast Cancer Breakthrough Ideas,” “Technology Innovations for Independent Aging,” and “Digital Health Transforms the Doctor-Patient Relationship.”

But it was no ordinary video game. Scientists in charge of the research had created the game specifically to solve a scientific puzzle that had baffled scientists for more than a decade: to determine the structure of a key enzyme that helps cause AIDS in monkeys. If they solved the problem correctly, they knew, it might lead to new drugs to treat HIV/AIDS in people. “The cool thing about this scientific discovery game is that we’re able to pass on a level of expertise to people who have none,” says Firas Khatib, a biochemist at the University of Washington who helped design the game.

Gaming has moved far beyond the world of recreation. Video games and simulations are now used to train surgeons, airline pilots, firefighters, soldiers, CIA agents and city planners—not to mention rehabilitate injured patients and motivate dieters, according to a 2010 story by Adam Penenberg in Fast Company magazine.

Khatib, his research advisor, David Baker, and their colleagues turned to gamers after they’d tried every research tool in their toolbox to determine the detailed three-dimensional shape of retroviral protease, an enzyme in an HIV-like virus that cuts up other proteins. Determining the three-dimensional shape of an enzyme is essential to fully understand what it does in cells and to develop drugs that might block it.

To determine the three-dimensional shape of an enzyme (or any other type of protein), researchers start with its sequence of amino acids. There are 20 different kinds of amino acids that can be mixed and matched, and each protein is composed of a long, unique string of them. A protein’s amino acid sequence somehow signals a protein to fold into a unique three-dimensional shape, one it must assume to function properly inside our cells. But a single string of amino acids can fold into an almost infinite number of three-dimensional shapes, and scientists still struggle to predict a protein’s three-dimensional structure from its sequence, even with modern supercomputers.

To do that job more efficiently, Baker’s team had developed a high-powered algorithm called Rosetta that helps determine a protein’s structure by comparing it to well-understood proteins that it seems to resemble. That worked with some proteins, but not the retroviral protease. “We used all the computing power we had with Rosetta, but it wasn’t enough,” Khatib says. To boost their computing power, in 2005 they built a screensaver that ran Rosetta in the background, then found more than 40,000 ordinary computer users to run the screen saver, which shows the long string of amino acids flopping around and folding into various shapes.

Still no dice with the retroviral protease. But they did learn something. Some of the owners more familiar with biochemicals got in touch with the researchers. “People looked and said your algorithm does some really stupid things,” such as folding proteins in ways that would never work, Khatib says.

So Khatib and Baker decided to enlist people to help the algorithm do the job better. They teamed up with Seth Cooper and Zoran Popovic at the University of Washington’s Center for Game Science, which seeks to use gaming to solve problems that people and computers alone have been unable to solve. As it turns out, people are better than today’s computers at recognizing patterns and solving puzzles. So Cooper and Popovic, working with Baker and Khatib, created a video game called Foldit to tap their strengths.

Foldit players came from all walks of life, and most knew little or no biochemistry. But the researchers had designed Foldit so that players would score points when they folded the protein according to well-known laws of physics and chemistry. This meant that players didn’t need to know the science; they just had to be good at solving difficult three-dimensional puzzles. This, as one top player noted, required a “slightly addictive personality.”

More than 600 players in more than a dozen countries ultimately played Foldit, forming teams and developing strategies that they shared with their teammates. And just three weeks after the researchers turned the problem over to the gamers, they had come up with very promising structures. The researchers then turned to their computer programs, including Rosetta, and “literally three days later, it was solved,” Khatib says. Two teams, the Foldit Contenders and Foldit Void Crushers had contributed enough to solving the structure of the retroviral protease that they’re listed as coauthors of the research paper, which was published online in September in Nature Structural & Molecular Biology.

It was the first time to Khatib’s knowledge that gamers had solved a thorny problem that had stumped scientists. But it probably won’t be the last. The researchers plan to make a game to shape a drug that binds and blocks the retroviral protease and could do the same to a closely related enzyme on HIV. “That’s the really exciting next step,” Khatib says.

CONNECT THE DOTS

To learn more, watch a video with interviews involving Foldit players and lead scientist, David Baker.  Read this article from Fast Company magazine about the many ways video games have infiltrated our lives.  You may also like these healthymagination posts: “Rapid Testing Technology Could Slow the Spread of AIDS,” “New Computer Modeling Analyzes Drug Therapies for HIV Patients,” and “Games Designed to Improve Health.”