Here is a list of up-and-coming technologies which will have great impact in 2014 and help shape healthcare over the next 12 months. The list was developed by Cleveland Clinic, US and presented at its annual Medical Innovation Summit. In reverse order
B-cell receptor pathway inhibitors
Chemotherapy is a blunt instrument designed to indiscriminately kill rapidly dividing cells in the hope that the cancer cells die more and grow back less than healthy cells. That normal cells are routinely damaged in this destructive procedure accounts for the side effects and toxicity of traditional chemotherapy.
The B-cell receptor pathway tightly controls the growth of the infection-fighting cells that are known as B-cells. When B-cells become cancerous, this pathway becomes unregulated and contributes to the malignant nature of the cells. However, this overactive pathway can be inhibited by novel drugs. In the last year, the results of clinical trials in humans conclusively showed that these new drugs are extremely effective at treating low-grade B-cell lymphomas and leukaemia for a long time and with very few side effects.
Ibrutinib targets Bruton’s tyrosine kinase (BTK), a protein essential for B-cell survival and proliferation. While it kills malignant B cells, ibrutinib has little effect on healthy T cells—unlike other lymphoma therapies. This leaves an important arm of the immune system largely intact, enabling patients to remain healthier during treatment.
Many patients respond well to this novel B-cell receptor pathway inhibitor, which lacks many of the side effects of chemotherapy, and it frequently produces long-lasting remissions even in patients with high-risk genetic lesions.
Results from a study published in The New England Journal of Medicine in 2013 reported an overall response rate of 71 per cent for patients with chronic lymphocytic leukaemia (CLL) who were treated with ibrutinib tablets. At 26 months, the estimated progression-free survival rate was 75 per cent and overall survival was 83 per cent. Ibrutinib has also shown very promising results in the treatment of patients with other B-cell malignancies, including mantle cell lymphoma (MCL), a rare and aggressive B-cell subtype of non-Hodgkin lymphoma. With approval from the U.S. Food and Drug Administration (FDA) expected at the end of 2013, ibrutinib would be the first in a class of oral BTK inhibitors and one of the first medicines to file for FDA approval via the new ‘Breakthrough Therapy Designation’ pathway, which is intended to expedite the development and review of drugs for serious or life threatening conditions.
To follow soon after will be other much anticipated B-cell pathway inhibitors for leukaemia, including spleen tyrosine kinase (Syk) inhibitors, like fostamatinib, and phosphoinositide-3 kinase (PI3K) inhibitors, like idelalisib. The B-cell receptor pathway inhibitors are innovative because they help fulfill the initial promise of imatinib. They will help patients who are no longer responsive to chemotherapy live longer, provide an alternative to chemotherapy in the future, and will stimulate additional research to find similar advances for other cancers.
TMAO ASSAY: Novel biomarker for the Microbiome
There is a global hunt in progress using a variety of cardiovascular fingerprints—scientists call them biomarkers—that have been discovered or created to help identify the initiation, development, and ongoing cascade of damage caused by heart disease.
Everyone is familiar with the most common biomarker for heart disease, the blood test for cholesterol levels. In the search for additional ways to predict the risk of heart disease, considerable evidence indicates that a molecule called C-reactive protein (CRP)—a protein made by the blood in response to inflammation of any kind—tends to be elevated in people who go on to develop heart disease and suffer a heart attack.
Scientists have now discovered what may be an important new biomarker for heart disease that serves as an accurate screening tool for predicting future risks of heart attack, stroke, and death in persons not otherwise identified by traditional risk factors and blood tests. The novel biomarker is called TMAO, or trimethylamine N-oxide, and it’s a microbial byproduct of intestinal bacteria.
TMAO is produced when intestinal bacteria digest the nutrient phosphatidylcholine, which is more commonly known as choline. This metabolite is thought to promote atherosclerosis by contributing to cholesterol metabolism in heart artery walls as well as in the liver and intestines. Major dietary sources of choline include egg yolks, red meat and dairy products.
Recently, tests conducted on more than 4,000 adults undergoing angiography over a three-year period revealed that TMAO levels are good indicators of who is at risk for serious heart disease—and can also serve as a possible future treatment target. As reported in The New England Journal of Medicine (NEJM) in 2013, people with the highest levels of TMAO had double the risk of death, nonfatal heart attack, or stroke compared to those with the lowest levels.
Heart experts now believe that by testing the gut biome for cardiovascular risk, doctors soon will be able to personalise nutritional recommendations for their patients on how to best prevent cardiovascular disease based on the production of TMAO.
This advice could include modifying the diet to limit the intake of choline-rich food, using bacteriotherapy to alter the gut microbiota with the use of probiotics or suppressing TMAO synthesis with medication. A laboratory test for TMAO research studies is now commercially available.
Computer-assisted personalised sedation station
A colonoscopy is an exam that lets a gastroenterologist look closely at the inside of the entire colon and rectum for polyps, the small growths that over time can become cancerous. Using a colonoscope, a thin, flexible, hollow, lighted tube that has a tiny video camera on the end, the doctor sends pictures to a TV screen. The exam itself takes about 30 minutes. Patients are usually given light sedation to help them relax and sleep while the procedure is performed.
In order to deliver minimal-to-moderate sedation during a colonoscopy procedure without using the services of an anaesthesiologist, a new computer-assisted personalised sedation device that delivers the prescription drug propofol for sedation via intravenous (IV) infusion was given premarket approval by the FDA in 2013. The novel technology is expected to empower healthcare facilities to more effectively use their limited resources for delivering greater value in the increasingly resource-constrained US healthcare environment.
The new sedation system is a first-of-a-kind device in the world that allows non-anaesthesia professionals to administer the drug propofol during colonoscopy procedures as long as they have professional training and use the machine where there is immediate availability of an anaesthesia professional.
The device can also be used for an esophagogastro duodenoscopy (EGD) procedure, which is an in-office test to examine the lining of the oesophagus, stomach, and first part of the small intestine. A physician-led team performs the EGD with a small camera that is inserted through the mouth and down the oesophagus while the patient is sedated.
The pivotal study for the computerised sedation device demonstrated that healthy patients who were sedated using the device had lower occurrences of low blood oxygen levels (hypoxemia) compared to similarly healthy patients who were sedated with midazolam or a similar medication during elective colonoscopy and upper endoscopy procedures.
According to the American Society of Gastrointestinal Endoscopy, the innovative computer-assisted sedation system can reshape the world of endoscopic procedures, because it can administer propofol without a trained anaesthesiologist on hand. Having an anaesthe siologist for certain routine endoscopic procedures is expensive and this has limited the use of propofol, which is regarded as a superior sedative because it gets to work quickly and has a short half-life, which leads to faster recovery.
The new sedation system is expected to be introduced into clinical practice on a limited basis beginning in 2014.
Relaxin for acute heart failure
Heart failure is a debilitating and potentially life-threatening condition in which the heart is unable to pump enough blood to supply the body. Heart failure affects both quality of life and life-span. Although it’s a diagnosis that carries a poor prognosis, it’s one that is improved and enhanced by medical and device therapy and, in limited cases, heart transplantation.
Serelaxin, a synthetic version of the naturally occurring hormone human relaxin-2, which is associated with pregnancy, has proven that it can help treat patients with acute, decompensated heart failure episodes after being infused over a 48-hour period in the hospital.
Serelaxin is a drug called a vasodilator. In people with heart failure, the chemically engineered form of relaxin increases blood flow throughout the body, which helps a poorly functioning heart to be more effective. Relaxin is also anti-inflammatory. Inflammation associated with heart failure can damage the kidneys, liver, and heart, but serelaxin appears to help prevent this from occurring.
Currently, 25 per cent of all patients admitted to the hospital with acute heart failure die within a year of their admission.
Phase III study results from an international, double-blind, placebo-controlled trial reported a reduction in death rates by 37 per cent in patients with acute heart failure following six months of treatment for an acute episode compared to those who received standard therapy. The FDA gave the investigational heart drug “breakthrough status” in 2013. This increases its prospect for faster FDA approval.
Once approved, serelaxin, the first in a new class of drugs that acts through a mechanism in the heart and kidneys, will become the first treatment breakthrough for acute heart failure in two decades. In clinical trials of patients hospitalised with acute onset of heart failure, serelaxin significantly improved shortness of breath, one of the most frightening and persistent symptoms of the disease. It also reduced organ damage caused by reduced blood flow, protected the kidneys and liver, and helped resolve fluid build up in the lungs more quickly than other therapies.
Faecal microbiota transplantation
Many hospitalised patients develop hospital-acquired infections, often times due, paradoxically, to broad-spectrum and fluoroquinolone antibiotic therapy used for medical treatment. Antibiotics, which are supposed to kill bacteria, can also increase the odds of some people developing a dangerous and potentially lethal infection from rod-shaped bacteria called Clostridium difficile, or C. diff.
C.diff infections—and the infectious diarrhoea that they cause—are a major reason for sickness and increased healthcare costs for hospitalised patients.
Gut microbiota, previously known as gut flora, is the name given today to the microbe population living in the human intestine. C. diff is just one of the hundreds of different microbial species found there, and, like the other intestinal bacteria, it is typically harmless. However, when antibiotics are used, aggressive and friendly bacteria in the gut are often killed together by the drugs. This can cause a gastrointestinal imbalance, which then allows for abnormal C. diff growth. Once passed through the faeces, C. diff is tough and persistent and can survive on dry surfaces for months.
C.diff spores can inadvertently be transmitted to hands, food, utensils, sheets, counter-tops, and curtains. When transferred and swallowed by another person, these spores multiply and produce toxins that lead to inflammation of the lining of the intestine, diarrhoea, nausea, vomiting, and abdominal pain. Symptoms range from moderate to severe, and some patients are at serious risk of death when they don’t respond to standard therapy. C. diff is typically combated with one of two antibiotics, vancomycin or metronidazole. However, many gastroenterologists are now employing faecal microbiota transplantation—the use of human stool transplants—to battle C.diff in those patients who do not respond to standard drug therapy. This novel bacteriotherapy typically entails a colonoscopy or enema to transfer a liquid suspension made from a healthy person’s faecal matter into a sick person’s colon in order to restore bacterial balance and cure a specific disease.
The clinical study results to date have been extraordinary, with the stool proving to be a safe and effective treatment that alters the faecal microbiota to resemble that of their donors with no more than two transplantations. Some people who have had multiple episodes of C.diff have realised benefits from the therapy hours later, have been cured of their symptoms within 24 hours, and have had no recurrences.
As more research about microbiota and how bacteria can actually make people healthier continues, it’s expected that faecal microbiota transplants could become a primary mode of therapy, not only for patients with multiple recurrences of C.diff but also for treating inflammatory bowel disease and non gastrointestinal medical conditions such as rheumatoid arthritis and Parkinson’s disease.
A recent Dutch pilot study reported that obese patients with symptoms known as metabolic syndrome improved their insulin sensitivity and lowered triglyceride levels six weeks after receiving a faecal transplant from thin, healthy relatives. A 2013 study involving the transfer of gut bacteria from obese and lean human twins to mice reported that mice receiving the bacteria from fat twins grew 15 per cent fatter while those with the bacteria from lean twins remained lean.
Perioperative decision support system
Anaesthesia is given to patients to inhibit pain, sedate the body, and also regulate various bodily functions in surgery. In the hospital surgical suite, there is tremendous activity among the medical personnel in attendance, so it’s critical that the anaesthesiologist appropriately recognise all of the patient’s known medical conditions, the surgical procedure being performed, and the anaesthesia work-flow, which includes knowledge of all the anaesthetic agents, fluids, and therapies that will be administered at specific times during the surgery.
Understandably, the anaesthetic record is now one of the most detailed physiological and pharmacological accounts in medicine. In order to meet the demands of modern surgery and improve the standard of care, a new anaesthesia management system that combines the latest in computer technology and microelectronics is now available.
This innovative system electronically documents the care provided by an anaesthesiologist, from pre-op to intra-op and recovery, producing a complete anaesthesia record of events, drugs and procedures. It’s this real-time patient data acquisition that elevates the quality of clinical decision-making, patient surveillance, and physician oversight.
Designed by anaesthesiologists for anaesthesiologists, this electronic anaesthesia record and clinical guidance system enables anaesthesia best practices to be carried out and helps to reduce the risk of anaesthetic errors. The ability of this anaesthesia information management system to collect data automatically enables anaesthesiologists to reliably and continuously create an accurate record, regardless of other demands during surgery. In addition, it also builds a compliant, billable electronic anaesthesia record while also supporting the clinical team managing the needs of the patient, whether a centenarian with multiple health issues or an infant who is eight weeks premature and struggling to thrive.
Having ready access to physiologic trends is essential, because it allows anaesthesiologists to make proper diagnoses and treatment decisions during surgery. Some complex surgeries last as long as 16 hours and clinicians need to be replaced.
With this anaesthesia management technology system running, however, everything during the surgery has been documented, providing the next clinician with all the critical information needed to allow continuity of care.
New era in hepatitis C treatment
Hepatitis C infection, a common liver disease that affects an estimated four million people in the US, is transmitted through exposure to infected blood (blood was not screened effectively for hepatitis C until 1992) or sexual contact with an infected person. The majority of people with the ailment don’t realise that they have the disease because of a lack of symptoms.
Hepatitis C is typically diagnosed when abnormal liver enzymes are identified through a routine blood test or if the infection becomes severe. 70 per cent of patients with hepatitis C develop chronic disease and 30 per cent may develop scarring of the liver and cirrhosis of the liver within 20 years of exposure to the virus. An additional 20 per cent of these patients eventually develop liver cancer. About 15,000 deaths are linked to the disease in the US each year.
While hepatitis C treatment has improved considerably, especially when compared to the 1990s, the news is even better now. Sofosbuvir, the first all-oral hepatitis C treatment, is moving through the final stages of FDA approval.
What this direct-acting antiviral (DAA) drug has is the potential to improve what for many has been a very difficult treatment regimen that can take up to 48 weeks and requires injections of interferon, a drug that is difficult to tolerate.
More important, the drug can improve treatment response rates to 90 per cent or higher in certain groups of patients. In addition to significantly higher cure rates, sofosbuvir—and others now in the pipeline—has better tolerability, safety,and a 12-week treatment duration.
In the FISSION trial, treatment-naïve genotype 2 patients treated with an oral DAA (1 pill daily) and ribavirin (2-3 pills twice daily) had a 67 per cent cure rate, which was just as good as standard therapy, but without the use of interferon.
In the FUSION trial, a study with patients with genotype 2 disease who did not respond to standard 24-week therapy or who had relapsed, those who completed 12 weeks of therapy with the oral DAA and ribavirin had an 86 per cent cure rate. An additional four weeks of the therapy increased the cure rate to 94 per cent. An oral drug with the highest cure rates ever and few side effects: There is finally real hope on the horizon for people with chronic hepatitis C.
Responsive neurostimulator for intractable epilepsy
Epilepsy is a neurological condition that produces seizures—brief disturbances in the normal electrical activity of the brain—that affect various mental and physical functions. Seizures happen when clusters of nerve cells in the brain signal abnormally, which may briefly alter a person’s consciousness or movements. When a person has two or more unprovoked seizures, he or she is considered to have epilepsy.
Medications and other treatments help many people of all ages who live with epilepsy, but in spite of their medication regimen more than one million people continue to have seizures that can severely limit or curtail many of their life’s experiences. Sudden unexpected death in epilepsy, or SUDEP, is a serious concern. It is a non-accidental death in a person with epilepsy, who was otherwise in a usual state of health.
Epilepsy surgery is a possibility for some with intractable epilepsy, but many cannot have it due to the risks involved or because it’s unlikely to be helpful. There is now another option. An implanted neurological device that can significantly reduce the frequency of epileptic seizures gained the unanimous backing of an FDA neurological device advisory panel in 2013.
Surgically implanted under the skin, the small device records electrocorticographic (ECog) patterns through leads containing electrodes that are placed at the patient’s seizure focus within the brain or else they rest on the brain. surface where seizures are known to start. When detection thresholds are met, the device delivers short electrical pulses to interrupt the triggers before any seizure symptoms can occur.
After reviewing ECoG recordings, physicians can assess the relationship between the detections of the device and reported seizures and then, non-invasively, customise the system’s electrical impulses as needed for that patient.
At long last, there appears to be a workable solution for people who suffer out-of-control bursts of electrical energy in the brain. With more than 15 years of development, testing and three clinical trials, the pivotal clinical study reported a 40 per cent reduction in seizure frequency among the treatment group compared to a 17 per cent reduction in the sham-stimulation control group. Long term results demonstrated sustained improvements in seizure frequency, with average frequency reductions of 44 per cent and 53 per cent at one and two years post-implant, respectively.
Genome-guided solid tumour diagnostics
Genomics is the study of groups of genes, how they interact in cells and the role that they play in health and disease. A variety of genomic-based cancer tests are currently available that, without surgery, can analyse the genes in a person’s tumour and predict both the biology and the aggressiveness of the cancer. What each of these novel genomic tests offers is the potential to reduce unnecessary cancer treatments and avoid their attendant side effects.
For prostate cancer, the new tests can predict whether a patient truly has a low-risk form of the disease and— based on the biology of the tumour—help a man decide if he is a good candidate for active surveillance rather than initial therapy.
Women with oestrogen receptor-positive breast cancer can find out if they need chemotherapy in addition to hormonal therapy in their treatment regimen, or whether they can benefit from hormonal therapy alone.
If a person has early-stage colorectal cancer, there is also a multi-gene expression test that looks at the gene activity with a person’s lymph node-negative tumour sample, assesses the risk and provides valuable information to the patient and doctor that traditional technologies do not capture.
To create their novel prostate test, scientists evaluated more than 700 patients and 288 candidate genes that predict clinical recurrence expressed in common by both the primary and highest Gleason patterns, which are indicators of prostate cancer aggressiveness. They then honed the genes down to 17 and asked whether the expression of these 17 genes could predict the presence of high-grade cancer or cancer outside the prostate in men who underwent prostate surgery.
In these studies there was a substantial increase in the number of patients who could more confidently consider active surveillance. This treatment plan entails monitoring cancer closely with blood tests, digital rectal exams, and biopsies at regular intervals to see if the cancer is growing. The protocol avoids unnecessary treatment and its potential side effects, which can include erectile dysfunction and incontinence.
The genomic test also identified a smaller number of patients who, despite seemingly low-risk clinical factors, had more aggressive disease that might require more immediate treatment.
We are now in a new age of cancer diagnosis. Thanks to the development of a variety of genomic tests for various cancers, what we are now witnessing is the beginning of an era of true precision medicine that has its basis in biology.
Retinal prosthesis
Retinitis pigmentosa, or RP, is a group of rare inherited diseases that damage these light-sensitive cells. People with RP experience a gradual decline in their vision because photoreceptor cells—the rods and cones—of the retina slowly degenerate, causing a gradual loss of side and night vision and, eventually, central vision and colour. Many patients become totally blind in both eyes.
There has been no effective treatment for RP—until now. After two decades of development and testing and more than $200 million in funding, the FDA approved a new technology for severe RP in 2013. It combined a surgically implanted 60-electrode retinal prosthesis that receives signals from a pair of external video camera-enabled glasses with a video processing unit that is worn at the waist or carried. This approval follows European agreement in 2011 and a unanimous recommendation by the FDA’s Ophthalmic Devices Advisory Panel in 2012 that this revolutionary product be made available to treat the RP patient population in the US.
The retinal system works when the video unit transforms images from the miniature camera into electronic data that is wirelessly transmitted to the retinal prosthesis, which contains an antenna and electrodes that replace the degenerated cells in the retina. Here, the data are transformed into small electrical impulses that stimulate the retina’s remaining inner neurons, resulting in the corresponding perceptions of light and images in the brain. People then learn to interpret these patterns of light and thereby regain some visual function. Given the profound vision loss that occurs with late-stage RP, the retinal implant will not restore complete vision, but it does allow people to detect light and dark in the environment as well as identify the location or movement of people and objects.
The retinal prosthesis system is approved for people of 25 years and older and have severe to profound RP with bare light perception—they can perceive light, but not the direction it comes from—or no light perception in both eyes.
What this novel retinal implant system does is allow people to reclaim their independence by being able to perform day-to-day activities. Results from a clinical study of 30 participants who received the ‘bionic eyes’ reported that most were able to perform basic activities better with the prosthesis than without it, including walking on a sidewalk without stepping off the curb; matching black, grey, and white socks; and recognising large letters, words, and sentences.
The retinal prosthesis is certainly a game changer in sight-affecting diseases and it represents a huge step forward for the thousands of people who had been without any available options for treating their blindness.
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