Top Ten Medical Innovation

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Top 10 Medical Innovations: 2011

Top 10 Medical Innovations: 2011
The Selection Process Breakthroughs Take Center Stage Top 10 Medical Innovations for 2011 Where Are They Now? 1 3 5 25

Top 10 Medical Innovations for 2011
The Selection Process

Which are the up-and-coming technologies and which will have the biggest impact on health care in 2011? Cleveland Clinic’s culture of innovation naturally fosters a good deal of discussion about new “game changing” technologies and which ones will have the greatest impact each year. The passion of our clinicians and researchers for getting the best care for patients drives a continuous dialogue on what state-of-the-art medical technologies are just over the horizon. This book was developed to share outside Cleveland Clinic what our clinical leaders are saying to each other and what innovations they feel will help shape health care over the next 12 months. We used a rigorous process to gather the opinions of Cleveland Clinic physicians and researchers, create a field of nominated innovative technologies for consideration, and develop a consensus perspective of what will be the Top 10 Medical Innovations for 2011. Our team interviewed more than 60 Cleveland Clinic experts to elicit their nominations. To receive consideration, nominated technologies had to meet the following criteria: • • • • The innovation had to have significant clinical impact and offer significant patient benefit in comparison to current practices. It must also have high user-related functionality that improves health care delivery. (40%) Nominated innovations had to have a high probability of commercial success. (20%) The innovation must be in or exiting clinical trials and be available on the market sometime in 2011. (20%) The innovation must have significant human interest in its application or benefits, and must have the ability to visualize human impact. (20%)

We probed the opinions of a broad cross-section of Cleveland Clinic staff from every major medical field. Our primary question was, “What innovations are “game changers” in your field?” In all, these interviews yielded nearly 90 nominations of emerging technologies. These nominated innovations were screened to confirm threshold criteria and consolidate duplicates. Our team prepared research on each technology and then presented a final list of over 45 up-and-coming technologies and their data profiles to two separate panels of leading Cleveland Clinic physicians. In September, each panel met to discuss, debate, and vote. The two panels then voted on the combined lists and established our Top 10 Medical Innovations for 2011. By enlisting the expertise of an independent firm and developing a rigorous selection process, the Top 10 represents an unbiased list of important emerging technologies, based on the collective leading Cleveland Clinic physicians and researchers. We hope you will find our selections interesting and will use them to stimulate your own thinking on future new trends and technologies and maybe even spur some innovation on your own. For this fifth annual event, Gerald Secor Couzens of MMC Worldwide and experts from PricewaterhouseCoopers (PwC) assisted us in navigating the detailed selection process. PricewaterhouseCoopers is a global network with 3,500 health industries professionals that include physicians, nurses, information systems specialists, health policy analysts, actuaries, financial advisors and data analysts. PwC collaborates with health industries clients to help them manage strategic initiatives, deliver financial performance and operational improvements, manage risk and compliance programs, and align core business processes with health information technology.

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Medical Innovations
Breakthroughs Take Center Stage

“The health of nations is more important than the wealth of nations.” — Will Durant, American historian, writer, and philosopher (1885-1981)

In the point-and-click world of the 21st century, the medical innovation process takes a much more traditional route. The end result of this painstakingly difficult, dynamic, and oftentimes complicated process, which is conceived in the mind and born in the laboratory, is that it ultimately saves lives and improves both health and quality of life for people the world over. Thanks to these extraordinary medical breakthroughs, men, women, and children are living not only longer lives, but better ones, too. Advances in medical imaging have helped define disease states with more precision. We now have new diagnostic tests that uncover diseases earlier than ever before, when they are most treatable; powerful drugs for a host of ailments; and intraoperative MRIs and minimally invasive surgical procedures that save lives while leaving little or no trace whatsoever of surgical intervention. These novel technologies are also revolutionizing the research process, making it possible for American science— and the industry that supports it—to stay steps ahead of the rest of the world. Presently, we have countless treatments in clinical trials that promise to prevent or eliminate some of the world’s deadliest ailments. In the meantime, the numbers of people who suffer from heart disease and cancer—our primary killers—have been declining thanks to our early detection capabilities and new treatment possibilities afforded by innovative drugs, devices, and therapies. Even better yet, with the sequencing of the human genome, we have crossed over into a new realm of our scientific evolution and scientists are now making use of nanoparticles to enhance visualization of tumors and also deliver drugs directly to them. Utilizing nanotechnology, with its man-made particles 1-100 billionths of a meter long, we have the potential to revolutionize medicine by successfully treating a variety of diseases that heretofore had been resistant to such attempts. This quickly increasing body of knowledge will also help scientists as they continue to work on more effective—and less toxic—cancer and heart diagnostics and interventions. In the end, it’s these ongoing efforts, on all fronts, which will lead to lower overall costs of care. As procedures become safer and more cost effective, they’ll have the added benefit of being able to gradually migrate from large teaching and research hospitals to community hospitals and, eventually, to outpatient settings.

“When there is an open mind, there will always be a frontier.” — Charles F. Kettering, American inventor and holder of more than 140 patents (1876-1958)

Although medical breakthroughs typically make front-page news—and in the following pages you will read about ten medical transformers that may just do that—rare is the time when we hear about the many talented and dedicated people who performed the countless experiments, made endless calculations, and performed hundreds, if not thousands, of tests before they were able to bring their medical innovation to the marketplace. Behind every innovative new medicine, therapy, or device are untold stories of researchers who refused to give up, even in the face of dead ends, unpredicted and capricious outcomes, and enormous stress on the road to creating something that had never existed before. Their discoveries combine curiosity, doggedness, cutting-edge science, hard work, and oftentimes a soupçon of serendipity. At various points in the innovation process it became possible to move forward only because one brave individual or enlightened committee stepped up and offered the financial backing to keep the project viable, to help bring it to fruition. To innovate—to create something new or different—we need to first recommit to a culture of innovation. The synergy of the marketplace—the venture community so necessary to the process— must be as complete as that of any laboratory in order for us to move forward with a sustainable innovation economy that we can depend on well into the future. Changing the world is no simple undertaking; the same can be said for medical innovation. As you turn the pages of this booklet, you will read about the medical breakthroughs that Cleveland Clinic experts believe are the “game changers” that will transform the practice of medicine in 2011. From searching for ways to uncover the early traces of Alzheimer’s disease to monitoring heart failure patients at home, we owe thanks to the talented and dedicated medical innovators who were willing to endure all the challenges that it took to see their innovation move from initial idea all the way to FDA approval. Their work is extremely vital to our world and we salute them all for their colossal efforts.

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Capsule Endoscopy for Diagnosis of Pediatric GI Disorders
One of the most challenging problems in pediatric digestive medicine is finding the source of hidden bleeding in the gastrointestinal (GI) tract. Problems in the esophagus and stomach can be diagnosed with traditional gastroduodenoscopy and large intestine problems can be uncovered with colonoscopy. Until recently, however, when traditional endoscopic imaging techniques using a fiber optic scope or x-ray failed to reveal the source of unexplained bleeding in the area between the stomach and colon, surgery was often a next step. First approved by the Food and Drug Administration for use in adults in 2001, official approval of wireless capsule Now there is a safe and painless alternative that can uncover many pediatric GI problems. For these difficultto-diagnose cases, many doctors are turning to a high-tech imaging tool called video capsule endoscopy for its detailed high-resolution images of the entire small intestine, an area that is one of the most difficult parts of the GI tract to view with traditional procedures. It all starts by having the child swallow the pill-sized capsule that has the miniature camera inside. This state-ofthe-art tool that barely weighs 1/7th of an ounce is easy to swallow with a sip of water, cannot be damaged by the powerful digestive enzymes of the stomach, requires no air insufflation or sedation, and is painless and safe. The single-use wireless camera in the tiny capsule takes thousands of color pictures and short video clips of the insides of the digestive tract during its travels as it’s pushed along by the peristaltic activity of the intestine’s involuntary muscles. During the course of the six-to eight-hour journey, more than 50,000 images are eventually transmitted to a small data recorder that is worn around the waist and later downloaded to a computer for careful review. Since the quality of the color images is so high, the technology is better than x-rays for detecting small-bowel ulcerations, polyps, and areas of bleeding. And once the source of the problem is finally detected, follow-up treatment can then be initiated. Capsule endoscopies are now used to accurately and non-invasively diagnose a range of problems in the pediatric digestive tract. In addition to Crohn’s disease, the device can help uncover Celiac disease and other absorption disorders, tumors of the small intestine, and injuries to the bowel. The video capsule is also being used to assess ongoing medical therapy in the GI tract, and as a way to definitively rule out the small intestine as the source of medical problems. By contrast, colonoscopy findings were normal in 15 of the 20 patients suspected of having Crohn’s, and were not conclusive for a Crohn’s diagnosis in the remaining five patients. endoscopy for pediatric patients was later granted based on a 2005 study that reported that the newer capsule examination revealed significantly more gastrointestinal abnormalities than did traditional imaging techniques. In half of the 20 patients with suspected small-bowel Crohn’s disease, the capsule study uncovered multiple smallbowel lesions, confirming the diagnosis. Eight remaining patients were found not to have Crohn’s disease, while the remaining two were found to have a rare disorder of the digestive tract called eosinophilic enteropathy.

Top 10 Medical Innovations: 2011

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Oral Disease Modifying Treatment for Multiple Sclerosis
Multiple sclerosis, more commonly known as MS, is thought to be an autoimmune disease that causes the body’s immune system to mistakenly attack the central nervous system. The central nervous system includes the brain, optic nerves, and spinal cord. It contains the nerves that act as the body’s messenger delivery system. Each nerve is covered by a fatty substance called myelin, which protects the nerves and helps in the transmission of messages between brain and other body parts. MS gets its name from the buildup of scar tissue (sclerosis) located in more than one area of the brain and/or spinal cord. “Sclerotic” plaques form when the myelin sheath is damaged, a process called demyelination. Without the protection afforded by myelin, signals transmitted throughout the central nervous system are then disrupted or halted. The process that damages the myelin also kills nerve fibers, leading to disability in many patients. While not considered a fatal disease, MS affects 400,000 Americans and can ultimately lead to a variety of transient or permanent symptoms, including blurred vision, loss of balance, slurred speech, extreme fatigue, memory problems, paralysis, and blindness. In the most common variant of the ailment, patients have periods of well-being followed by sporadic relapses. MS cannot be cured, although there are seven FDA-approved drugs that can modify the course of the disease, helping to lessen the frequency and severity of MS attacks, and reduce the accumulation of brain lesions. MS drugs are effective, but they must be injected or infused on a regular basis, and this is enough to discourage many people from consistently following through with their therapy, because of the bother and sometimes-painful side effects. In order to increase patient treatment compliance, both patients and doctors alike have been asking for an effective oral alternative to add to the MS drug armamentarium. Although MS still has no cure, fingolimod capsules represent a big step forward in treating the underlying causes of this debilitating disease, slowing the progression of disability, reducing its frequency and severity, and ultimately improving the lives of people with MS. Pivotal phase III clinical trials of the drug with 2,600 patients with relapsing forms of MS published in the New England Journal of Medicine reported that a daily dose of fingolimod reduced relapses by 60% annually when compared with placebo, and by 52% when compared with an injectable MS medication. In addition, MRI testing showed that patients treated with the drug had fewer brain lesions. Some patients experienced side effects with fingolimod and as a condition of its FDA approval, fingolimod will be studied globally for an additional five years to monitor its safety. After years of research and final testing, fingolimod was given a priority review reserved for groundbreaking therapies and was unanimously approved this year by the Food and Drug Administration as the first oral disease-modifying protection against MS attacks. The new capsule works differently from all other first-line MS therapies. By successfully preventing the migration of T cells from the lymph glands to the brain and spinal cord, it effectively keeps these marauding cells from attacking the fatty myelin sheaths that cover the nerve fibers. Many years ago, Japanese researchers noted that a fungus discovered in the intestines of wasps could naturally suppress immune responses. Refined into a chemical that could be used for people undergoing organ transplantation, this novel drug eventually failed in achieving that primary goal. Quite surprisingly, however, it was found that the drug could do something very important: blunt the immune system attacks in patients with MS.

Top 10 Medical Innovations: 2011

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Exhaled Nitric Oxide (NO) Breath Analysis for Monitoring Asthma
Asthma is a chronic inflammatory lung disease that affects more than 23 million Americans. Characterized by prolonged or uncontrollable coughing, wheezing, tightening of the chest, and increased mucus secretion, an asthma episode narrows and blocks the airways and makes breathing extremely difficult. Asthma episodes can be as brief as a few minutes or can last for hours, even days, or weeks, unless properly treated and controlled with medication. Even so, more than 3,400 people die from asthma each year in this country. To date there are no known cures for the disorder, but with new medications and techniques it can be managed to the point were the person with asthma experiences few or no symptoms or complications. In order to know how efficiently a patient’s lungs are functioning, how well they are moving air in and out, a lung function exam is given by a doctor using a spirometer, a device that measures the amount of air a person can expel from their lungs, and the amount of resistance to airflow throughout the respiratory tract when the person exhales. While lung function tests and patient complaints of symptoms are considered the standard method for assessing asthma, its severity, and response to medication, it has been increasingly recognized by pulmonary experts that these in-office exams tell only part of the asthma story. Asthma is actually caused by the underlying inflammation triggered by allergies, infections, and irritants that affect lung function by narrowing the airways. However, traditional testing can’t assess this lung dysfunction, which is the root cause of the ailment. Until recently, the decision to treat someone for asthma, and indeed how to treat them, was based in great part on spirometer testing. However, given the importance of the role that airway inflammation plays in asthma, there is now a new FDA-approved hand-held diagnostic testing device that measures levels of exhaled nitric oxide (NO). This is a gas that is produced by epithelial cells that line the inner walls of the lung’s airways. First discovered in humans in the 1980s, NO was later found to be an asthma biomarker in the 1990s.

When specific asthma triggers such as allergens or chemicals inflame the airways, more NO is released than normal, oftentimes long before asthma symptoms have become obvious. The amount of NO in the exhalation indicates how much inflammation there is in the lungs—and the severity of the asthma. To use the NO monitoring device, a patient breathes through a disposable filter attached to the equipment, then breathes out without taking the filter from the mouth. Following the exhalation, the device’s advanced sensor technology automatically indicates how much NO is present in the patient’s exhaled breath. Regular monitoring of NO levels helps doctors and patients develop a good understanding of the way that airway inflammation is progressing. A drop in NO values indicates that inflammation has been reduced and the asthma is better controlled with medical therapy, while a subsequent rise in the NO value indicates inflammation is worsening and the asthma is becoming more severe. Using this critical NO information, a doctor can then tailor the treatment strategy, changing medication and dosages accordingly. When used in conjunction with other laboratory assessments of asthma, NO testing is destined to play a valuable role in adding precision and accuracy to the diagnosing, monitoring and pharmacological management of people with asthma, helping to avoid both under-and over-treatment of the disease.

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Endoscopic Weight-Loss Procedure: Transoral Gastroplasty (TOGA)
Rates of overweight and obesity, higher than ever in the United States, are both linked with an increased risk of life-threatening conditions, including heart disease, stroke, type 2 diabetes, and cancer. Moreover, obesity can lead to psychological distress as a result of poor self image, social isolation, and job discrimination. Obesity is a life-threatening disease affecting 34% of adults in the U.S. However, bariatric surgery to promote weight loss by restricting oral intake and changing absorption of food is a viable option. Most procedures are done laparoscopically, in which instruments are inserted through a tiny incision in the abdominal wall. What remains after the surgery is a gastric sleeve, so small that when even little amounts of food enter the sleeve and More than 225,000 Americans who are 100 pounds overweight and unable to lose weight through diet, exercise, and weight-loss medication are expected to undergo bariatric surgery this year. The surgery doesn’t remove excess tissue but instead reduces size of the stomach and the body’s ability to absorb nutrients from food. In the two years following a bariatric procedure, many people are able to lose more than 60% of their excess weight, a majority experience resolution of their diabetes and high blood pressure and a reduction in their cholesterol. Bariatric surgery is safe, but as with any surgical procedure there is still a risk of complications. In an effort to reduce or eliminate complications and offer a “scar-less” procedure, there is now a new weight-loss option for obese patients who want to lose weight and improve their health but without undergoing major surgery. Final Phase III results of a pivotal multi-center TOGA study are anticipated in late 2010, with FDA approval to follow The Transoral Gastroplasty, or TOGA, represents a significant improvement in minimally-invasive bariatric surgery. Instead of making incisions in the abdominal wall, this incision-less option combines two flexible endoscopic staplers passed through the mouth that are used to create a small restrictive pouch in the stomach. After eating a small meal, patients are left with a feeling of satiety, thereby decreasing caloric intake, and eventually shedding excess pounds. soon after. Since there are no incisions and minimal anesthesia is used, it’s likely that the TOGA procedure will eventually be performed on an outpatient basis. While traditional laparoscopic bypass surgery requires a recovery time of several days and a clear liquid diet that is moved up to soft solids and then a normal diet over time, TOGA patients are typically discharged a few hours after surgery and resume eating normal foods sooner than their laparoscopic counterparts. Weight loss in early placebo-controlled TOGA clinical trials has ranged from 12 to 28 pounds in the month following surgery. Losses approaching 40% of excess body weight can be expected within a year. push against its wall, there is a sensation of fullness that will encourage the patient to stop eating. During a one-to two-hour TOGA procedure, the patient is first given general anesthesia and then a specialized flexible device is placed in the mouth and guided down the esophagus into the stomach. Using a tiny camera at the end of a thin endoscope, the doctor is able to visualize the stomach anatomy. Once the TOGA device is moved into place, it is opened and creates a vacuum, which brings the folds of the stomach together. The cylindrical TOGA device forms a little tube, which is then finally held together with a series of strategically-placed titanium staples and a ring at the new stomach outlet.

Top 10 Medical Innovations: 2011

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Telehealth Monitoring for Individuals with Heart Failure/ Implanted Wireless Cardiac Device for Monitoring Heart Failure
A weakening of the heart’s ability to pump blood throughout the body causes heart failure. Between 500,000 and 900,000 new cases are diagnosed each year in the United States, making this debilitating ailment the most common diagnosis in Medicare patients. Even though great strides have been made in effective identification and medical therapy, the prognosis remains poor for people with heart failure and it accounts for 200,000 deaths annually. Average life expectancy is less than five years in patients with symptomatic heart failure, while 90% of those with advanced disease have survival rates of a year.

The most common cause of this incapacitating ailment is heart damage due to coronary artery disease or high blood pressure. When a person has heart failure, quality of life is minimized. Fatigue, shortness of breath, fluid retention, and excessive urination at night are common symptoms. As the ailment progresses, even mild physical activity becomes exhausting. As fluid accumulates in the lower extremities, swelling of the feet, ankles, legs, and abdomen occur. Older people with heart failure may also experience lightheadedness or confusion. Despite the dramatic improvements in medical care, heart failure is the leading cause of hospitalizations, with more than one million admissions annually. An even larger problem for the health care system is the re-admission rate. More than 27% of heart failure patients return to the hospital for additional care in the month after first leaving the hospital, while 58% are re-admitted over the course of the next six to 12 months. The estimated direct and indirect costs of this extra medical assistance may be as high as $20 billion. There are many good heart failure medications. What’s been missing, however, has been the critical information to help physicians adjust the medication when necessary to keep the patient healthy—and out of the hospital. But this is all starting to change, thanks to wireless health care and a variety of technology-based remote patient monitoring devices that are now playing a major role in heart failure management. Better and more affordable technology is helping to ensure patient adherence to medication schedules and the early detection of signs of decompensation, particularly in the early period after hospital discharge. Various sophisticated methods of assessing heart failure control are now in use or in final testing phases, including an implantable, miniature, permanent monitor with communication technologies that measures and transmits daily pulmonary artery pressure levels, a key indicator of heart health. A recent Phase III clinical trial of the novel system in patients with New York Heart Association Class III heart failure showed a 30% reduction in rate of hospitalization after six months and a 38% reduction per year. Approval of this device by the Food and Drug Administration is expected in 2011. In addition, a variety of sophisticated at-home telehealth monitoring devices are being used at home by patients with heart failure to check and transmit real-time body weight, heart rate, and blood pressure results to a secure database for doctors to review with a smartphone or computer. By utilizing this important information that’s been gathered remotely at home, hospitalizations can then be avoided by readjusting the patient’s medication. This conveniently improves outcomes, reduces costly re-hospitalizations, significantly improves the quality of life of patients, and lessens the huge financial strain placed on the health care system by heart failure.

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Hepatitis C Protease Inhibiting Drugs
Hepatitis C, a common liver disease that affects an estimated 3.9 million people in the United States, is transmitted through exposure to infected blood (blood was not screened for hepatitis C until 1992) and sexual contact with an infected person. The majority of people with the ailment have no bothersome symptoms, which is why health experts say millions are unaware that they even have it. Hepatitis C is typically diagnosed when abnormal liver enzymes are identified through a routine blood test or if the infection becomes severe. Seventy percent of patients with hepatitis C develop chronic disease and 30% may develop cirrhosis of the liver within 20 years of exposure to the virus, which causes severe scarring of the liver. An additional 20% of these patients eventually develop liver cancer. Hepatitis C virus infection is the leading cause of chronic liver disease, the reason for more than 30% of liver transplantations, and a major contributor to as many as 12,000 deaths annually. Total medical costs for people with hepatitis C are approximately $30 billion. To date, treatments for hepatitis C virus infection have been somewhat disappointing. The best cure rates following a 24-to 48-week course of therapy with the combination of the oral antiviral medicine ribavirin (taken three times a day) and an injectable interferon (once a week) is about 40%. The flulike side effects of these drugs are often debilitating, however, leading many patients to stop treatment. When a second round of treatment is used with this chemotherapy combination, success rates plummet to 10%. There are an estimated 300,000 Americans with hepatitis C who have failed this treatment protocol. When medical care is ineffective and the disease progresses, a liver transplant may be necessary. Unfortunately, there are no proven medicines for patients who don’t respond to traditional hepatitis C therapy—until now, that is. The good news is that cure rates with these new protease inhibitors are higher than those with standard hepatitis C therapy. And so, after decades of research and expected FDA approvals, a new era of antiviral medications developed specifically to target hepatitis C virus is about to begin. Study results with a drug called boceprevir were similar with difficult-to-treat patients with hepatitis C, although cure rates were slightly lower with the three-drug combination. When combined with interferon and ribavirin, boceprevir cured the infections of about 66% of the patients who took the drugs for 48 weeks compared to 38% of those in the control group who received the standard therapy for 48 weeks. Two advanced drugs called hepatitis C protease inhibitors now awaiting approval from the Food and Drug Administration (FDA) have the ability to fundamentally change the treatment for hepatitis C for patients who have not responded to previous therapies. Both new drugs have been very successful in curing test subjects in clinical trials, which represents a big step forward in successfully battling this debilitating disease. The experimental drugs belong to a class of medications called protease inhibitors, which work by blocking a key enzyme that viruses need in order to copy themselves and proliferate. New data from Phase 2 trials report that when the experimental drug telaprevir, developed specifically to target hepatitis C virus, was added to standard treatments for hepatitis C, 72% of the patients achieved sustained viral response (or viral cure) after taking the medication for 24 weeks. This study confirmed that the 24-week course was just as effective as it was for patients taking the drug cocktail for 48 weeks.

Top 10 Medical Innovations: 2011

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JUPITER Study: Statins for Healthy Individuals
Approximately 785,000 Americans will have a heart attack for the first time this year while another 470,000 will have a repeat attack. Heart attacks strike both men and women, young and old, and in almost 20% of cases, these myocardial infarctions are deadly. Therefore, if many of the people suffering serious coronary events have cholesterol levels no worse than those of many In the past, low levels of blood cholesterol were assumed to indicate a reduced risk for heart attack and premature death. However, the JUPITER trial—which stands for Justification for the Use of Statins in Primary Prevention: an Intervention Trial Evaluating Rosuvastatin—has changed conventional wisdom, demonstrating that some individuals with normal cholesterol levels benefit from early intervention with a statin medication to prevent disability and premature death from coronary heart disease. JUPITER is important because it demonstrated that many seemingly healthy people with elevated levels of a biomarker of inflammation called high sensitivity C-reactive protein or hsCRP are actually at relatively high risk for cardiovascular disease and can benefit from statin treatment. In the Jupiter Trial, participants with normal to low levels of LDL cholesterol (so-called “bad” cholesterol) and elevated levels of inflammation were randomized to receive a statin or placebo. Experts estimate that using JUPITER findings routinely and then prescribing statins to people with low LDL but high hsCRP In the group receiving a statin, the risk of cardiovascular disease and death was reduced by 44% compared with placebo-treated patients. JUPITER was an international study enrolling approximately 15,000 patients (average age, 66 years) randomly assigned to receive the statin drug rosuvastatin (20 mg/day) or placebo despite normal cholesterol levels. Current guidelines consider LDL cholesterol levels less than 130 mg/dL healthy and levels less than 100 mg/dl ideal. The average LDL-cholesterol of JUPITER study participants was only 108 mg/dl prior to enrollment. levels, five to six million people treated for five years in this fashion would result in approximately 250,000 fewer heart surgeries, angioplasties, heart attacks, strokes, and deaths attributed to heart disease. The potential savings to the health care system may be enormous. JUPITER study results first published in 2008 in the New England Journal of Medicine reported that the statin used by the study participants cut their risk of cardiovascular disease and death by almost half compared to study individuals treated with placebo. In 2010, acting on the advice of an independent advisory committee, the U.S. Food and Drug Administration added language to the label of the drug rosuvastatin based upon the Jupiter results. Previous studies have shown that levels of hsCRP are strongly predictive of the risk of coronary events including cardiovascular death, stroke, myocardial infarction, hospitalization for unstable angina, or arterial revascularization. The JUPITER study defined a high hsCRP value as levels greater than 2.0 mg/L. asymptomatic people, perhaps there is a need for a much more sensitive marker than LDL cholesterol for predicting a person’s heart-attack risk. The reason the researchers looked at this seemingly healthy group was the observation that only about half of individuals who develop coronary heart disease have levels of cholesterol that are clearly elevated.

Top 10 Medical Innovations: 2011

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First Therapeutic Cancer Vaccine Approved by the FDA
After 15 years of research and a roller-coaster ride at the U.S. Food and Drug Administration (FDA) that went on for several years, sipuleucel-T (Provenge) recently became the first cancer vaccine ever approved by the FDA, ushering in a new era in the ongoing war on cancer. Vaccines have long been the dream of cancer researchers for more than a century. Although sipuleucel-T is not a cure for metastatic prostate cancer, it is the first therapeutic vaccine to win FDA approval, which is certainly reason to cheer. Similar experimental therapeutic vaccines are currently in various testing phases for other cancers, including lymphoma, lung, and breast cancer. Prostate cancer is now the second leading cause of cancer death in American men, behind only lung cancer. Surgery and radiation therapy can cure prostate cancer, but once it spreads to the bones, the disease is often incurable. In the United States, prostate cancer is expected to kill more than 32,000 men each year. Hormonal therapy and chemotherapy are commonly used to slow metastatic growth of hormone-sensitive prostate cancer tumor cells and this strategy generally controls the disease for variable time periods until the hormone-resistant cells proliferate and progress. Once the disease develops resistance to hormonal therapy and chemotherapy, historically there have been limited therapeutic options. Now there is sipuleucel-T. Unlike a traditional preventive vaccine (such as a flu or measles vaccine) that revs up the immune system to help prevent infection, sipuleucel-T is a novel option, a therapeutic vaccine given to men with advanced prostate cancer to get their own immune systems to develop an immune response against the disease, leading to improvement in survival. Sipuleucel-T is the first vaccine to demonstrate a survival benefit. This is certainly encouraging news, especially for a vaccine whose only side effects are mild fever and body aches shortly after vaccination. With this new prostate cancer vaccine, doctors hope that the dendritic cells will trigger a cascade of events that leads to an immune response that eventually destroys all rogue prostate cancer cells. In a pivotal Phase III clinical trial reported in the New England Journal of Medicine, sipuleucel-T was found to extend the lives of patients with advanced, metastatic, androgen-independent prostate cancer by an average of four months when compared to patients given a placebo, reducing the risk of death by 24%. It’s now hoped that men will derive greater benefit when the drug is given earlier in the course of the disease, or when combined with other medications. Dendritic cells are the master controllers of immunity: They alert T-lymphocytes—a type of white blood cell—to the presence of foreign intruders within the body. These cells patrol the body, and when they encounter something that should not be there (such as cancer cells), they can process proteins from these invaders and present them to the T-cells. The T-cells in turn use these foreign proteins as a means of “recognizing” the foreigner and setting in motion the immune recognition and destruction of the invader. The new vaccine works differently from other prostate cancer drugs. Dendritic cells, a type of immune cell, are first removed from the patient and then exposed to prostate cancer cells in a laboratory. The goal is to have these specially treated cells seek and destroy the circulating tumor cells throughout the body when they are finally infused back into the patient several days later. The process is repeated two additional times to complete the entire treatment cycle.

Top 10 Medical Innovations: 2011

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Anti-CTLA-4 Drug (ipilimumab): Targeted T-Cell Antibody for Metastatic Melanoma

Melanoma is a form of skin cancer characterized by the uncontrolled growth of pigment-producing cells located in the skin. While anyone can develop melanoma, it is still not clear how all melanomas develop. Exposure to ultraviolet (UV) radiation clearly plays a big role. A history of sunburns, especially severe sunburns at an early age, have been shown to increase the risk of developing melanoma. In addition, exposure to UV radiation from indoor tanning increases the danger. Melanoma usually occurs in adults, but it is sometimes found in children and adolescents. About 20% of all melanomas will spread. This cancer is not only resistant to most drug therapies, but when it moves to the brain, there is little that can be done to arrest its onslaught. Patients with late-stage melanoma have a 15% survival rate. Although melanoma accounts for barely 3% of skin cancer cases, it causes the most skin cancer deaths. The incidence of metastatic melanoma has increased over the last 30 years, and the death rate has been increasing faster than that of most other cancers. There were approximately 68,000 new cases of melanoma in the United States in 2009, with 8,700 melanoma-related deaths. A recent randomized study in the New England Journal of Medicine reported that about 23% of patients with advanced stage III or IV metastatic melanoma in 125 cancer centers who took an experimental anti-CTLA-4 drug called ipilimumab were still alive two years later, compared to 14% of those who did not receive the drug. On average, those taking ipilimumab survived 10 months, compared to the six months for those who received only a melanoma drug.

What ipilimumab does so well is specifically target the protein molecule, CTLA-4 (or cytoxic T-lymphocyte associated antigen 4), which is found on white blood cells and suppresses the immune system from fighting disease. When ipilimumab is taken, it acts as a T-cell potentiator, however, and blocks the action of CTLA-4, allowing the patient’s immune system to fight the cancer more vigorously. Even though ipilimumab does not cross the blood-brain barrier, the newly activated T-cells do. Small studies with ipilimumab have reported that patients with brain metastases caused by melanoma experienced a 15-month response from the drug, on average. The long-held dream of researchers to find a way to harness the patient’s own natural defense system to destroy their cancer tumor cells is now getting closer to reality. The effectiveness of ipilimumab in melanoma therapy further confirms the role of immunotherapy as an effective therapeutic treatment, and there are now ongoing clinical trials with other promising immunotherapeutic drugs for patients with prostate cancer and neuroblastoma. The FDA grants priority review status to medications that offer major advances in treatment, or that provide therapy where no adequate treatment exists. Based on its extraordinary response in improving the survival rates of patients with previously treated advanced melanoma, ipilimumab has received FDA priority review status and the approval of this life-saving drug is expected soon. If all goes as expected, ipilimumab will be the first new melanoma drug available in decades.

Top 10 Medical Innovations: 2011

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New Molecular Imaging Biomarker for Early Detection, Prevention, and Treatment of Alzheimer’s Disease
Alzheimer’s disease (AD) is like a crafty burglar: It quietly gains entrance and steals all that is precious from the brain, then loiters around the scene of the crime, cleverly avoiding all means of detection while the brain slowly deteriorates from its irreplaceable losses. To date, no one has been able to discover the secret of the criminal’s modus operandi. In 1906, German psychiatrist, Dr. Alois Alzheimer first lectured about the mass of plaques and tangles he noted in a microscopic post-mortem brain tissue sample taken from his 51-year-old patient. More than a century later, however, scientists are still stymied as to what causes this devastating brain disease that was named after Dr. Alzheimer. Sadly, it’s a disease without a cure. AD is the most common cause of dementia and the costs to the American family and economy have been staggering. This will only worsen as the AD population in the U.S. grows from well over five million at present to an anticipated 20 million within four decades. The cost of taking care of Americans aged 65 and older with AD is expected to skyrocket from $172 billion to well over $1 trillion in that same timeframe. Presently, even though clinical diagnosis of AD is quite accurate, a doctor can positively diagnose AD only after the patient has died, when brain tissue can be examined under a microscope for the distinctive plaques and tangles associated with the disease. However, that is about to change very soon. Since the distinctive beta-amyloid plaques and tau tangles of AD are present at least a decade before dementia symptoms become evident in many patients, there is now a global hunt underway to find ways to help identify the initiation of brain damage caused by AD. This new goal of detecting dementia at its very earliest manifestation will then allow steps to be taken to protect the brain before too much damage is done so that people—and their loved ones—never have to experience the ravages of AD. A new brain imaging compound will make this early detection possible. After being injected into the patient, AV-45, the radioactive molecular imaging compound then crosses the blood-brain barrier and binds to any beta-amyloid plaques, the tell-tale “signature” that is so characteristic of AD. Follow-up imaging by positron emission computed tomography (PET) allows doctors to effectively peer into the brain of a patient and assess it for dyed amyloid plaques. It’s thought that AV-45 can be used as a biomarker not only for diagnosing AD but also for monitoring disease progress and drug efficacy. Once this novel compound receives its expected FDA approval in 2011, this dream could become a reality, paving the way for better ways to distinguish AD from Parkinson’s disease and other types of dementia; using it as an effective method of tracking disease progression from mild cognitive impairment to late AD; and utilizing it as a key diagnostic in the development and testing of the more than 150 AD drugs presently in the pipeline. Over time, it’s expected that AV-45 will gradually become the dominant part of the diagnosis of AD, an important innovation that will help change the course of AD research and treatment as we now know it.

Top 10 Medical Innovations: 2011

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Top 10 Medical Innovations for 2010

Where Are They Now?

1. Bone Conduction of Sound for Single-Sided Deafness Single sided deafness (SSD) affects 9 million people in the U.S. The newly developed non-surgical, noninvasive, removable hearing device bypasses this requirement by transmitting sound via the teeth and bones, to both cochleae. This digital audio device is nearly invisible and consists of a small microphone unit worn behind the ear on the deaf side and an easy-to-remove dental retainer-like processor for the conversion of audio signals into vibratory energy. The world’s first non-surgical, non-invasive bone-conduction hearing device has recently completed a pivotal clinical trial, and the study results have been submitted to the FDA. In addition to SSD, the intended future indications also include addressing the needs of patients with conductive, mixed hearing loss, and tinnitus. 2. Low-Volume, Low-Pressure Tracheal Tube Cuff to Reduce Ventilator-Associated Pneumonia Ventilator-associated pneumonia (VAP) is the leading cause of hospital-acquired mortality in the ICU and is estimated to result in $1.5 billion in excess expenditure each year. The newly developed endotracheal tube cuff has a special suction setup that provides continuous effective airway seals at low mucosal pressures. It allows all secretions to drain from the subglottic space just above the tube cuff, which dramatically reduces the risk of VAP and death in the hospital ICU. This device received the 510(k) clearance from FDA in late 2009. More physicians are now able to take advantage of this innovative technology to reduce the incidence of VAP and further improve patients’ safety. 3. Continuous-Flow Ventricular Assist Devices The newest version of continuous-flow ventricular assist devices weighs only 3 ounces. It is attached alongside the native heart and runs on rechargeable batteries connected through the abdomen. The new device can quietly and effectively take over the pumping ability of the heart by generating up to 10 liters of blood flow per minute and providing up to 10 years of circulatory support. This device has earned a CE Mark for both bridge-to-transplant and lifetime use and is currently used in the United States as a bridge-to-heart transplant under FDA-approved clinical investigation. 4. Non-Vitamin K Antagonist Oral Anticoagulants Venous thromboembolism (VTE), which is caused by abnormal blood clot, is a serious life threatening condition that affects more than 1 million Americans annually. Two newly developed non-vitamin K antagonist oral anticoagulants, which target the Factor Xa and thrombin respectively, now offer predictable and well-tolerated alternatives to the oral anticoagulant warfarin and can provide a more convenient-and safe-way for patients to dose themselves and prevent blood-clot formation. Approved for use in Canada and Europe, both drugs have already completed the long-term phase III clinical trials and study results are currently under review by the FDA. Dabigatran received FDA approval in October 2010.

5. Fertility Preservation Through Oocyte Cryopreservation Thanks to the new cryopreservation techniques developed in Italy and elsewhere, this novel reproductive technology is now allowing eggs of a healthy woman to be safely frozen and stored, ready to be thawed and fertilized at a later date. Currently, oocyte cryopreservation is offered in more than 50% of Assisted Reproductive Technology clinics in the United States. There have been more than 1,000 healthy births as a result of this novel egg-banking technology. 6. Forced Exercise to Improve Motor Function in Patient’s With Parkinson’s Parkinson’s disease (PD) is a debilitating brain disorder that affects more than 1 million Americans and has no cure. While current available medicines help ease the PD symptoms for a few hours, a novel exercise-based therapeutic strategy can now bring relief to patients for weeks. By conducting “forced exercise” (tandem riding in which patients are forced to pedal 80-90 RPM), patients’ motor function was dramatically improved by 35% and this improvement lasted for 4 weeks after the training ended. Used either alone or as an adjunct to other therapies, forced exercise has opened up a promising avenue in PD treatment and management. Further studies are now ongoing. 7. Outpatient Diagnosis of Sleep-Related Breathing Disorders Sleep-related breathing disorders have historically been assessed by means of an all-night sleep study in a hospital-based sleep disorder lab. With the increase of public awareness of sleep-related disorders, there are not enough sleep centers to meet the burgeoning demand for diagnosis. That is now changing with the introduction of the patient-friendly self-contained sleep-monitoring devices that allow for the sleep testing to be performed anytime in a patient’s home. Data collected through testing can be accessed by clinical professionals for further analysis. Recent studies have shown that home portable monitor testing is as effective as traditional sleep lab testing in the screening and diagnosis of sleep-related breathing disorders. With improved patient access to care and reduced costs, home testing may soon replace expensive sleep-lab testing. 8. Oral Thrombopoeitin (TPO) Receptor Agonist that Stimulates Platelet Production An estimated 50,000 to 100,000 people in the United States are diagnosed with chronic immune thrombocytopenic purpura (ITP). Patients with ITP have low blood platelet count that may produce bruising or excessive bleeding. While most cases of ITP can be controlled, the response to medication is often disappointing and associated with risk. A recently approved oral thrombopoietin receptor agonist has shown high effectiveness in the treatment of ITP by stimulating production of cells in bone marrow that form platelet cells in the blood. Patients taking this new drug were 10 times more likely to reach the target platelet counts as the control group. Further studies are undergoing to evaluate the safety and efficacy of the drug not only as a long-term treatment for ITP, but as a possible treatment for patients with hepatitis-C-related thrombocytopenia and for patients receiving chemotherapy for leukemia.

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Where Are They Now?

9. Devices for Occluding Left Atrial Appendage to Reduce Stroke Risk Compared to people with normal heart rhythm, patients with atrial fibrillation (AF) have a five-fold increased risk of suffering a clot-related stroke and thus have to take anticoagulant medications, such as warfarin, to manage their AF-related stroke risk. However, warfarin is a problematic medication to use and the drug dosage needs to be monitored regularly. By implanting a left atrial appendage occlusion device, which is specially designed to prevent the formation of blood clots, patients may now discontinue warfarin use. The FDA has recently approved one clip-based occluding device to be implanted into AF patients during openheart surgery. Several other devices are currently under clinical trials and are expected to reach market in the coming years. 10. Whole-Slide Imaging for Management of Digital Data in Pathology Whole-slide imaging is a new technology that converts conventional glass slides into digital pathology slides with excellent image quality that can be viewed, managed, stored and streamed over the Internet, and analyzed on a computer. With this technology, pathologists can now scan a slide within seconds and share it immediately and conveniently with peers anywhere in the world. This technology offers tremendous promise for improving patient care by delivering faster diagnoses, reducing patient morbidity and mortality, and lowering overall medical costs. In addition to supporting primary diagnosis, it also provides valuable information to personalized medicine. The U.S. Patent and Trademark Office has recently issued a patent to a digital pathology company for the techniques involved in the processing and displaying of digital slide images stored on a server. Top 10 Medical Innovations for 2009 1. Use of Circulating Tumor Cell Technology Circulating tumor cells (CTCs) are cancer cells that have detached from an existing tumor cell and have entered the bloodstream. Measuring CTCs in a sample of blood can facilitate early detection of recurrent cancer in patients who are known to have the disease. This technological advance helps in understanding response to therapy much sooner, allowing patients to monitor their progress at any point along their treatment course, and guiding the doctor in adjusting therapy as needed. The FDA has approved the use of CTC detection to monitor treatment effects in breast, colorectal, and prostate cancers, while other applications are under active development. 2. Warm Organ Perfusion Device By slowly pumping warm blood through organs and keeping them in a functioning state, this game-changing innovation offers a much better way to transport a variety of living organs—such as hearts—for transplantation. By extending the time that a donor organ can be maintained outside of the body, this technology not only provides better preservation of an organ’s function prior to transplant, but should also help to ameliorate the effects of the worldwide donor organ shortage, which remains one of the biggest problems in this field. The world’s first warm blood perfusion system for the heart has been approved for use in Europe and it is currently undergoing pivotal multicenter testing in the United States with systems for transporting lungs, livers, and kidneys also under development.

3. Diaphragm Pacing System This novel device enables paralyzed patients to breathe without the assistance of bulky mechanical ventilators. It electronically stimulates the diaphragm, causing it to contract, allowing air to move into and out of the lungs to provide unassisted and more natural breathing. Diaphragm-pacing systems have been approved by the FDA and are commercially available in the United States, and can be implanted using minimally-invasive surgical techniques. These devices can dramatically reduce the rates of ventilator-induced pneumonia in paralyzed patients and help to improve patients’ overall quality of life. 4. Multi-Spectral Imaging Systems Once attached to a standard microscope, this imaging system enables researchers to spectrally resolve up to five or six chromogens (colors) that are used to stain specific elements in a single tissue section and accumulate more information compared to previously existing techniques. The ability to stain for multiple cell markers substantially speeds information acquisition by providing results from a single assay that would normally require multiple tests. In addition, simultaneous staining of multiple markers allows researchers to better understand interrelationships of complicated signaling pathways in cancer cells (or other abnormal tissues), allowing the development of more effective therapies. Several multi-spectral imaging systems have been developed within the past few years that have current clinical applications and will continue to provide important diagnostic advances in the future. A new class of medical endoscopes enhanced by multispectral imaging technology is currently under development to help surgeons collect information about human tissue signatures with speed and resolution surpassing anything currently available on the market. 5. Percutaneous Mitral Valve Regurgitation Repair This innovative procedure has been demonstrated to work well for some people with moderate to severe mitral regurgitation or leakage of the inlet valve for the left ventricle. This backward leakage of blood through the mitral valve can cause congestion in the lungs, increased work for the heart, and heart failure. During the procedure, which is performed in the cardiac catheterization laboratory, a wishbone-shaped clip is threaded through a catheter in blood vessels in the groin to the leaking mitral valve. The clip is then applied to bring the center of the two mitral valve leaflets together, which eliminates the backward leakage of blood through the valve and restores normal blood flow into the left ventricle. While open heart surgical techniques remain the clinical standard of care, the mitral valve clip will likely be an important additional treatment for this disease, particularly in patients for whom surgery is too high a risk. Already approved for use in Europe, the mitral valve clip is expected to receive FDA approval in 2011. 6. New Strategies for Creating Vaccines for Avian Flu With the potential to alter management of a potential worldwide health crisis, the novel vaccine strategy uses a mock version of the bird flu virus called a virus-like particle (VLP). This offers a better solution to protect people against infection from the deadly avian virus. VLP-based vaccines do not require live virus for their development, which results in vaccines that are easier and faster to produce. Significantly shorter development and production times compared to live virus vaccines, allow public health authorities to react more quickly in the event of a potential pandemic. With this technology, an influenza vaccine can be created in just 10–12 weeks from the identification of a new viral strain. Several VLP vaccines targeting H1N1 and H5N1 viruses have been created and are currently being tested in multiple human clinical trials.

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Where Are They Now?

7. LESS and NOTES Applications Laparoendoscopic single-site surgery (LESS) and Natural Orifice Transluminal Endoscopy Surgery (NOTES) allow doctors to perform surgeries with a minimum of cutting and virtually no scars. Pain levels and recovery times are significantly reduced with these scarless surgeries, allowing patients to return home and resume daily activities much more quickly. LESS and NOTES have been successfully utilized and continue to evolve in multiple clinical areas. Urologic, gynecologic, and digestive-tract procedures have already been successfully completed. 8. Integration of Diffusion Tensor Imaging (Tractography) This technology is providing important information for neuroscientists probing the long neglected portion of the brain known as white matter, with its densely packed collection of intertwining fibers that allows neurons in the lobes of the brain to communicate with each other. Prior to the development of this technique, assessment of these critical areas was impossible in the living brain. This approach, which employs a modified version of magnetic resonance imaging, is being evaluated in a number of clinical applications. One particularly important area may be its use in preoperative planning prior to invasive neurologic procedures. Doctors can precisely map out a detailed spatial wiring map of the brain and avoid damage to these sensitive areas during neurosurgical procedures. Several studies published in early 2010 demonstrated the superiority of this technology in detecting various forms of dementias and cognitive impairments. 9. Doppler-Guided Uterine Artery Occlusion This is an innovative method to treat uterine fibroids, which are benign muscular tumors of the uterus that are a common cause of pelvic pain and heavy menstrual bleeding. It offers women who have failed medical therapy a minimally invasive alternative to hysterectomy. Through temporary occlusion of uterine arteries, this technology can significantly reduce fibroid size and related symptoms. The experimental procedure is currently undergoing pivotal clinical trials at centers throughout North America and Europe. 10. Private Sector National Health Information Exchange This is a comprehensive system of electronic health records that connects, stores, and shares clinical data from hospitals, physician offices, pharmacies, labs, and other sources to help improve communication flow between health care providers, patients, and clinicians. It has the potential to revolutionize our health care management by reducing the risk of medical errors, lowering costs, and increasing the overall quality of health care. Although primarily a private sector effort, this computerized system is gaining increased public interest. With the enactment of the American Recovery and Reinvestment Act (ARRA), which places a very strong emphasis on the importance of health information exchange, significant ARRA funds have been spent in support of health information exchange efforts. The recent issuance of two regulations defining and supporting “meaningful use” of electronic health records has laid solid foundation for the ubiquitous and effective use of health information technology in the near future.

Top 10 Medical Innovations for 2008 1. Flexible Intralumenal Robotics A novel catheter-based technology has been developed that allows precise remote manipulations within the intra-luminal space with precision and reproducibility that surpasses human capabilities. This technology is likely to have applications in urology, cardiology, cardiac surgery, and other specialties. Increasing clinical experience has been acquired for this truly cutting-edge device technology, particularly in intervention cardiology. It has been proven especially useful in electrophysiology applications to assist in catheter positioning during ablation procedures. However, this technology holds great promise for wide-ranging catheter-based technologies such as endovascular grafting procedures. Current device development is focused on improving precision, ergonomics, and reducing occupational radiation exposure. In a recently completed preclinical in-vivo study, it was reported that vascular robot technology can provide catheter navigation in a much faster and less traumatic way compared with conventional manual catheterization. 2. Percutaneous Aortic Heart Valves For high-risk patients, a technique has been developed that involves inserting a new expandable wire mesh valve with internal valve leaflets. These valves can be inserted through blood vessels in the groin and threaded into the heart or through a small chest wall incision. The indications, benefits, and pitfalls of this technology are now being explored in numerous international clinical trials. In addition, major valve makers have made multiple strategic acquisitions of new supporting technologies to position their products for this promising market. Development of technologies for percutaneous valve replacement or reconstruction of the mitral and pulmonary valves has progressed as well. The FDA approved the first percutaneous heart valve in early 2010 under the Humanitarian Device Exception program. 3. RNA-based Therapeutics This particular innovation uses RNA antisense technology to treat patients who are unable to reach their targeted cholesterol levels with statins alone or who are statin intolerant. The therapy is intended to reduce the production of ApoB-100, a protein that carries certain forms of cholesterol and triglycerides in the bloodstream. Phase III trials are underway to evaluate the efficacy of RNA antisense-based technology on patients with familial hypercholesterolemia. Other clinical trials are also being conducted to examine efficacy in reducing low-density lipoprotein levels in high-risk hypercholesterolemia patients with encouraging early results. Clinical trials are also underway for this technology for a diverse range of conditions including viral infections, cancer, and macular degeneration. 4. Convergence of Advances in Genome Scanning and Informatics to Support Clinical Applications Genetic testing that can be used for personalized risk assessment and disease management has undergone very rapid development during the past couple of years particularly in cardiology, with the number of commercially available genomic tests growing by 25% annually. Clinicians are trying to corroborate the findings yielded by this technology with clinical events. Most of these tests look at a single gene and are used to diagnose rare genetic disorders; however, whole genome scanning is also being developed in this increasingly competitive field. There is growing interest in the development of tests that look at multiple genes that may increase or decrease an individual’s global risk for diseases such as cancer or diabetes.

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Where Are They Now?

5. Oral Anticoagulant Drugs for Treating and Preventing Thrombosis New anticoagulant treatments are being introduced with the goal of curbing complications such as bleeding and thrombosis. Compared to existing therapies, these agents continue to hold great promise for providing effective anticoagulation for patients with atrial fibrillation or who are at risk for deep venous thrombosis. Early results of clinical trials with these new agents suggest that they may be used with decreased complications and easier monitoring compared with existing treatments. Two non-vitamin K antagonist oral anticoagulants have completed phase III clinical trials with the study results currently being reviewed by the FDA. One, dabigatran, recently received FDA approval. 6. Live Attenuated Influenza Vaccine for Children as Young as Two Years Nasal drops containing live attenuated flu can be used as a vaccine in lieu of needles, and provide effective protection from influenza for this high-risk population. Beginning with the 2008/09 influenza season, annual vaccination for all children aged 6 months to 18 years was recommended. In September 2009, a live attenuated influenza intranasal vaccine for the novel H1N1 influenza virus was approved by FDA. 7. Image Fusion for Diagnostic and Therapeutic Use This technology is used to diagnose medical problems, both anatomic and physiologic in nature, as well as to assist minimally invasive procedures such as stent placement or tumor ablation. Current development is focused on determining which applications of the technology produce the most benefit. SPECT/CT imaging in cardiovascular disease holds promise for combined imaging of the myocardium and coronary arteries in ischemic heart disease and as a tool to determine the inflammatory nature of plaque burden. SPECT/CT can be a useful adjunct in oncologic imaging, especially for neuroendocrine tumors or melanoma and may provide more detail regarding the extent of disease in these cases. SPECT/CT can also be a useful adjunct in determining the extent of disease in cases of infected device implants, such as infected joint prostheses. 8. Implanted Device Allowing Neural Control of Objects by the Severely Disabled Novel communication interfaces are being developed for severely motor-impaired individuals to provide the ability to control devices and to potentially restore limb movements. This interface system is designed for individuals paralyzed by spinal cord injuries, stroke, amyotrophic lateral sclerosis, and other advanced central nervous system diseases. Current technology utilizes multiple electrodes incorporated into a tiny device that is surgically implanted into areas of the brain that control movement. The neuronal signature from these areas then undergoes decoding to direct the movement of a computer cursor and other assistive devices. Pilot clinical trials are currently being conducted and a limited number of patients have demonstrated that they can indeed exert control of external devices with their thoughts and can direct communication technology— such as email—by visualizing the movement of their hand.

9. Engineered Cartilage Products for Joint Repair Biomaterials have been developed to replace joint cartilage damaged from injury or arthritis. The materials are surgically implanted into the joint with the intent to restore the damaged cartilage and avoid joint replacement surgery. Advancements are being made for technologies that employ autologous and synthetic materials used for cartilage substitutes in joint reconstruction. All replacement technologies must reproduce the delicate interplay between cellular, structural support and biomolecular elements that constitute normal cartilage. New materials currently under development have been shown to be stronger and to possess the ability to better withstand pressure than materials used in previous generations of this technology. 10. Dual Energy Source Computed Tomography (CT) Imaging The CT device features two x-ray sources and two radiation detectors, which allow for imaging of patients more quickly and with less radiation. The speed at which the dual-source scanner operates allows physicians to image patients with high or irregular heart rates, which used to be a significant limitation of this technology. Increased speed in image acquisition provided by a dual energy source has greatly expanded the application of CT imaging in cardiac disease. Clinicians are now applying this technology to vascular imaging, evaluation of renal disease, and CT colonography. Imaging protocols for dual energy source CT studies can now be performed with equivalent or even lower radiation exposure than before. Top 10 Medical Innovations for 2007 1. Cancer Vaccines These targeted therapies are being used to prevent cancer and treat patients more specifically according to the type of cancer they have. One example of a cancer vaccine is the HPV vaccine developed to prevent cervical cancer caused by human papilloma viruses. Approximately 25% of American teenage girls were vaccinated with the HPV vaccine in 2007. Currently, researchers are working on developing therapeutic vaccines for a variety of cancers including breast, lung, colon, skin, kidney, and prostate. There are more than 270 therapeutic vaccine candidates currently in the pipeline. The first therapeutic vaccine for advanced prostate cancer was approved by the FDA in early 2010. 2. Designer Therapeutics Using Selective Receptor Antagonists Novel therapeutics have been created to block receptor activation that leads to improved patient outcomes. Examples include therapeutics that block the peripheral side effects—such as constipation and nausea—of opioid medications for pain, which can adversely affect patients and lengthen hospitalizations; and control of the body’s stress response to mediate eating and smoking. Research in this area continues with new trials being conducted in multiple areas, such as treatment of muscle and bone disorders.

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Where Are They Now?

3. Neurostimulation for Psychiatric Disorders Neurostimulation, such as deep brain stimulation (DBS), is emerging as a significant treatment option for millions of Americans who are suffering from treatment-resistant depression and treatment-resistant obsessivecompulsive disorder. Studies of DBS in the treatment of resistant forms of depression continue to show promise. DBS has been approved by the FDA for the treatment of dystonia, essential tremors, and drug-resistant obsessive-compulsive disorder. Current research is focusing on the use of DBS for multiple clinical applications, such as the treatment of Tourette syndrome, drug-resistant epilepsy, and obesity. The total market for neurostimulation products in the United States was about $1.3 billion in 2009 and is expected to double by 2015. 4. Optical Coherence Tomography (OCT) This noninvasive imaging technology is used in the treatment and diagnosis of eye diseases, such as diabetic retinopathy and macular holes. OCT has been approved by the FDA and has since become a highly useful medical imaging technique with a market that may top $800 million by 2012. Ophthalmology is predicted to remain the dominant application, but experts envision OCT applications and products expanding to include cancer detection, glucose monitoring, dermatology, and dentistry. In addition, one OCT is being developed for intravascular imaging to assess unstable plaque in coronary arteries. Regulatory approvals in the United States and Europe are expected in 2011. 5. Bronchial Thermoplasty (BT) BT involves the controlled application of heat in the lungs to improve pulmonary function and reduce asthma symptoms. This therapy is used to ward off asthma attacks. In early 2010, the FDA approved the very first BT device for treatment of severe persistent asthma. 6. Ranibizumab This drug therapy inhibits uncontrolled blood vessel formation in the eye, which is the primary cause of agerelated macular degeneration, and the leading cause of new blindness in older Americans. The drug has since become a clinical and commercial success (more than $2 billion in sales since introduction) and remains the standard of care for age-related macular degeneration. Intravitreal injection of the drug has been approved recently by the FDA for the treatment of macular edema after retinal vein occlusion. Several new trials are currently ongoing to test combination therapies with drugs such as volociximab and dexamethasone, which may allow similar or improved outcomes with fewer treatments. 7. Endografting This minimally invasive repair technique traditionally used in cardiology is now being used to treat vascular disease, including thoracic abdominal aneurysms. Multiple studies have demonstrated the satisfactory performance of endografting for treatment of atherosclerotic vascular disease in a variety of clinical settings. Endografting has become an accepted and important therapy for the emergency and elective treatment of diseases of the aorta and peripheral vasculature.

8. Targeted Cancer Therapies This involves using second generation, small molecule tyrosine kinase inhibitors to block or modulate disease and provide treatments for advanced cancers, such as renal cell carcinoma. This technology has been an important advance with several agents in this genre that are either in clinical trials or that have been approved for a variety of cancers including leukemia, pancreatic, breast, lung, and renal. Research is currently being conducted on new drug compounds as well as ways to improve existing drugs through combination therapies and new ways to administer them. The FDA has granted priority review to a new compound for the treatment of adults with newly diagnosed chronic myeloid leukemia. 9. Left Ventricular Assist System (LVAS) This is the first implanted ventricular assist device that senses when to increase or decrease the rate of blood flow. The device takes over most of the function of the left ventricle, and helps generate the force necessary to propel oxygen rich blood throughout the body. This concept has become a commercial success with many trial successes. The role of LVAS as a bridge to transplantation for end-stage, medically refractory heart failure has been well established. As a potential extension of this therapy, the NIH has recently established funding for clinical investigation into the use of these devices as an alternative to medical management in less advanced cases of heart failure. These newer pumps are smaller and potentially more durable and longer lasting than the current generation of pumps. Currently, these devices can be used in the United States in FDA-approved clinical investigations. 10. Convection-Enhanced Delivery of Drugs This emerging drug delivery method is being used to administer medication directly to the site where it is needed, without exposing the rest of the body to a drug’s effects. The current success of this concept is difficult to gauge as a large number of studies have been conducted with conflicting reports of efficacy. However, development is continuing with the aim of improving the overall drug delivery process as well as the catheters needed to implement it. Additional clinical trials can be expected in the future while the medical industry tries to advance this concept.

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Many thanks to the Top 10 Selection Committee members who were essential in the process and selection of the final Top 10 Medical Innovations for 2011. We would like to especially thank Michael Roizen, MD, who for the third year in a row has chaired the selection committee and panel.

Nancy Albert, PhD Director Nursing Research & Innovation Gregory Borkowski, MD Institute Chair Imaging Institute Thomas Daly, MD Section Head Immunology Scott Endsley, MD Staff Medicine Institute Joseph Foss, MD Director Clinical Research for General Anesthesia Thomas Graham, MD Chairman Innovations Brian Griffin, MD Staff Cardiovascular Medicine Georges-Pascal Haber, MD Associate Staff Glickman Urological & Kidney Institute Richard Lang, MD, MPH Department Chair Preventive Medicine

Steven Mawhorter, MD Staff Infectious Disease Venugopal Menon, MD Staff Cardiovascular Medicine James Merlino, MD Staff Colorectal Surgery William Morris, MD Staff Hospital Medicine Steven Nissen, MD Chairman Cardiovascular Medicine Michael Roizen, MD Institute Chair Chief Wellness Officer Ellen Rome, MD, MPH Section Head Adolescent Medicine Richard Rudick, MD Director Mellen Center Phillip Schauer, MD Staff Director Bariatric Surgery Program

Roy Silverstein, MD Chairman Cell Biology Rishi Singh, MD Staff Ophthalmology John Suh, MD Department Chair Radiation Oncology Michael Vogelbaum, MD, PhD Associate Director Brain Tumor & Neuro-Oncology Center Herbert Wiedemann, MD Institute Chair Pulmonary, Allergy & Critical Care Medicine

Thank You

We would also like to give thanks to the team who effortlessly worked to produce the Top 10 book and video – especially: Susan Bernat Gerald Secor Couzens

Cleveland Clinic Innovations Christopher M. Coburn Executive Director [email protected] 9500 Euclid Avenue GCIC 10 Cleveland, Ohio 44195 Tel. 216.444.5757 www.clevelandclinic.org/innovations Cleveland Clinic Innovations is recognized as one of the world’s top corporate venturing organizations by Global Corporate Venturing.

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