- #1 Next Generation of mRNA Vaccinology
- #2 PSMA-Targeted Therapy in Prostate Cancer
- #3 New Treatment for the Reduction of LDL
- #4 Novel Drug for Treatment of Type 2 Diabetes
- #5 Breakthrough Treatment for Postpartum Depression
- #6 Targeted Medication for Hypertrophic Cardiomyopathy
- #7 Non-Hormonal Alternatives for Menopause
- #8 Implantable for Severe Paralysis
- #9 AI for Early Detection of Sepsis
- #10 Predictive Analytics & Hypertension
#1 Next Generation of mRNA Vaccinology
Traditional vaccines have been a guiding light in medicine for centuries. Developed at the end of the 19th century for several infectious diseases, vaccines became healthcare's gold standard for disease prevention. However, traditional vaccines can take years—even decades—to produce. In today's globalized world, there is an increasing need for a very rapid response to new and emerging infectious diseases.
Over the past two decades, widespread interest in RNA-based technologies for developing prophylactic and therapeutic vaccines has increased. Interest heightened during preclinical and clinical trials, which revealed mRNA vaccines provide a safe and long-lasting immune response in humans.
Furthermore, the emergence of the SARS-COV-2 virus and the subsequent COVID-19 pandemic proved that the world needed rapid development of a vaccine that was easily deployable across the world. Because of the previous research that had laid the groundwork for this technology, an effective COVID-19 vaccine was developed, produced, approved and deployed in less than a year.
Discovered in the 1960s, mRNA technology mimics a viral protein and tricks the immune system into mounting an immune response against the "invader." Powerful antibodies are produced that can provide long-lasting protection against an actual viral infection.
Advancements in the generation, purification and cellular delivery of RNA have enabled the development of RNA therapies across a broad array of applications, like cancer and Zika virus. The technology is cost-effective, relatively simple to manufacture and targets previously undruggable pathways.
This landscape-changing technology has the potential to be used to eliminate some of healthcare's most challenging diseases quickly and efficiently. While the technology is not new, COVID-19 unlocked the power of mRNA vaccines, and we are entering a new frontier in fighting disease.
#2 PSMA-Targeted Therapy in Prostate Cancer
Each year, more than 200,000 American men receive a diagnosis of prostate cancer – making it the most commonly diagnosed cancer among men in the United States. While many patients survive prostate cancer, those who develop metastatic cancer have much poorer outcomes and a higher likelihood of dying from their disease. Currently, prostate cancer is the third leading cause of cancer death in the United States.
Early detection and successful imaging are critical for tumor localization, staging the disease, and detecting recurrences. Conventional imaging methods, like include computed tomography (CT), magnetic resonance imaging (MRI) scans and bones scans, are essential but offer limited accuracy.
Promising new imaging technology has emerged called prostate-specific membrane antigen positron emission tomography (PMSA PET). PSMA is an antigen found in high levels on the surface of prostate cancer cells and is a potential biomarker for the disease. PMSA PET uses a radioactive tracer to locate and attach to PSMA proteins, which are then visible by PET. This targeted approach can be used in conjunction with CT or MRI scans to visualize where prostate cancer cells are residing.
Experts anticipate the PET-tracer procedure will soon become the new standard of care for detecting prostate cancer metastasis. In 2020, this technology received FDA approval based on Phase III clinical trials, which showed a substantially increased accuracy for detecting prostate metastasis compared to conventional imaging (bone and CT scans).
Men initially diagnosed with prostate cancer who are at risk for metastatic disease or previously treated but have developed recurrence evidenced by rising PSA are the best candidates for PSMA PET.
#3 New Treatment for the Reduction of LDL
One of every four deaths in the United States results from heart disease, making it the leading cause of death for men, women, and people of most racial and ethnic groups. High levels of blood cholesterol, particularly low-density lipoproteins (LDL-C), are known to be a major contributor to cardiovascular disease, and this correlation has been studied for decades.
Lipid-lowering therapy is a mainstay therapy for cardiovascular risk reduction, and statins are one of the most common medications used to decrease production and increase the removal of cholesterol by the liver. However, some patients require additional therapy for lowering LDL-C, even on the maximum dose of statins.
In December 2019, the Food & Drug Administration (FDA) reviewed the application for inclisiran in treating primary hyperlipidemia (including hereditary hypercholesterolemia) in adults who have elevated LDL-C while being on a maximally tolerated dose of statin therapy.
Inclisiran is an injectable, chemically synthesized small interfering RNA that targets PCSK9 (proprotein convertase subtilisin-kexin type 9). In contrast to statins, it requires infrequent dosing (twice per year) and provides effective and sustained LDL-C reduction in conjunction with statins. Its prolonged effect may help alleviate medication non-compliance, one of the leading causes of failure to lower LDL-C levels.
Inclisiran was approved by the FDA in December 2021 and is a game-changer for heart disease patients, which is one of our Top10 Medical Innovations for 2022.
#4 Novel Drug for Treatment of Type 2 Diabetes
In the United States, 1 in 10 individuals has diabetes, a chronic condition that affects how the body processes food into energy. In a non-diabetic person, glucose from digested food is released into the bloodstream to fuel bodily processes. When blood glucose elevates, the pancreas releases insulin to reduce the glucose to maintain homeostasis. In diabetic patients, however, the pancreas does not produce enough insulin nor properly manage insulin production.
Type 2 diabetes occurs when blood glucose is chronically elevated, making it difficult for blood sugar levels to be maintained. Currently 90-95% of diabetic patients have type 2 diabetes, making it the most common form of diabetes in America.
While some patients can control their type 2 diabetes with healthy eating and exercise (obesity and inactive lifestyle contribute to about 90-95% of diagnoses), many others rely on medication or insulin to manage their condition.
The first medication that doctors typically prescribe is metformin. However, in 2020, the Food & Drug Administration discovered high amounts of carcinogens in the medication and halted most of its production temporarily. This unfortunately made it difficult for patients to receive their needed care, as many other medications on the market have troublesome side effects. So a need arose for novel treatments for patients with diabetes.
One such potential therapy is a once-weekly injectable dual glucose-dependent insulinotropic polypeptide (GIP) and glucagon-like peptide receptor agonist (GLP-1). This treatment is currently in late phase III clinical trials to control blood sugar. Injected under the skin into the stomach, thigh, or upper arm, GLP-1 and GIP receptors activate in the body and cause the pancreas to release insulin and block the hormone glucagon, thereby limiting the blood sugar spike after meal consumption. Additionally, it slows digestion, resulting in individuals remaining full longer and eating less.
Once FDA approved, this treatment will be a novel drug in a new class called dual GIP and GLP-1 receptor agnostics. Thus far, the clinical trials reveal that the treatment significantly reduces hemoglobin A1C in type 2 diabetes and supports weight loss, making it potentially the most effective therapy for diabetes and obesity yet developed.
Recently, the number of adults diagnosed with diabetes annually has doubled in the U.S, and costs of insulin continue to rise, demonstrating the great need for new treatments and offering patients a non-insulin treatment option. The entrance of a blockbuster drug to improve blood glucose levels and aid weight loss will significantly impact the future of diabetes treatment.
#5 Breakthrough Treatment for Postpartum Depression
Postpartum depression is a complex mix of physical, emotional, and behavioral changes after the delivery of a child. These changes are attributed to a hormonal imbalance as the body returns to its pre-pregnancy state. Postpartum depression can occur at any time from the onset of birth up to 4 months post-delivery.
Postpartum depression is very common. Experts believe the rate could be at least twice as much as what current statistics tell us, as many cases go undiagnosed. Currently, counseling and anti-depressant medications are the primary treatments. Many women have benefited from these treatments, yet there remains a sub-population of mothers whose symptoms are not alleviated through these therapies.
A novel treatment has been designed to specifically treat postpartum depression. In 2019, the Food & Drug Administration approved an intravenous infusion treatment of this neurosteroid, which is a derivative of allopregnanolone. The treatment modulates the hypothalamic-pituitary-adrenal axis, which mediates the body’s response to stress, and is modulated by GABAergic signaling.
This treatment design is groundbreaking as it targets the signaling thought to be deficient in hormone-sensitive postpartum depression and the gabaergic hypothesis of depression. The treatment is administered around the clock for 60 hours, or 2.5 days. Additionally, this treatment appears to show benefits very quickly, while traditional anti-depressants typically take 2 to 4 weeks to have a significant effect.
If untreated, postpartum depression can cause significant suffering in mothers and their families. This fast treatment option would be a much-needed breakthrough for families who are undergoing a stressful time and for women who are afflicted with this often overlooked condition.
#6 Targeted Medication for Hypertrophic Cardiomyopathy
Hypertrophic cardiomyopathy (HCM) is a chronic disease in which excessive heart muscle contraction —often caused by genetic changes—can lead to debilitating symptoms and cardiac dysfunction. The American Heart Association estimates that 1 in every 500 people have HCM, but many of those patients go undiagnosed until the disease has progressed. These patients live with physical limitations and fatigue, and many undergo surgery to relieve the heart muscle thickening.
For decades, clinicians have only been able to treat patients' HCM symptoms—using drugs developed to treat other heart problems—with limited effectiveness. Currently, nonspecific medications are prescribed to treat some of the symptoms that HCM shares with other cardiovascular diseases. These therapies include beta-blockers, anti-arrhythmic drugs, calcium channel blockers, and anticoagulants.
A new therapy, however, works to reduce the root cause of the problem in many patients. A targeted cardiac myosin inhibitor reduces hypercontractility caused by genetic variants that put the heart into overdrive. By acting specifically on this mechanism of HCM, this novel treatment not only slows the progression of the disease but also helps some patients improve their quality of life.
The United States Food & Drug Administration has assigned a target action date for this therapy of April 28, 2022. If approved, this would be the first medication dedicated specifically to treating HCM and providing new hope to patients and physicians.
#7 Non-Hormonal Alternatives for Menopause
Hot flashes are the most common symptom for which individuals experiencing menopause, or the end of their menstrual cycle, seek treatment. Also known as vasomotor symptoms, these sudden feelings of warmth in the upper body, often accompanied by sweating, skin redness and a rapid heartbeat, can become so intense that they interfere with daily activities and disturb sleep. More than 50 percent of all menopausal women experience hot flashes, which can persist for an average of seven years.
Many patients experiencing severe menopausal hot flashes are treated with hormone therapy that replaces the estrogen their bodies no longer produce after menopause. While effective and safe when used appropriately, hormone therapy does involve some risk and not all patients are appropriate candidates or ready to try this treatment option.
Fortunately, a new group of non-hormonal drugs, called NK3R antagonists, have emerged as a viable alternative to hormone therapy. These drugs disrupt a signaling pathway in the brain that has been implicated in the development of hot flashes and have shown great promise for relieving moderate to severe menopausal hot flashes as effectively as hormones in clinical trials.
While additional studies are needed to fully understand the effectiveness and safety profile of these new drugs, it is clear that the next generation of non-hormonal treatments for menopausal hot flashes is on the horizon.
#8 Implantable for Severe Paralysis
Approximately 1 in 50 Americans, or 5.4 million people, have some form of paralysis. The leading cause of paralysis is stroke, followed by spinal cord injury and multiple sclerosis. As the number of people living with paralysis and spinal cord injuries increases, so do the costs of treating them. Spinal cord injuries alone cost roughly $40.5 billion annually.
While the cost of treatment is high, the value does not compare to the detrimental effects on patients. Most patients experience a decline in their overall health. No cure exists for paralysis, and most patients rely on rehabilitation services to retain their independence and enjoy a better quality of life.
Recently, a team has offered new hope for these patients by leveraging implanted brain-computer interface (BCI) technology to recover lost motor control and enable patients to control digital devices. The technology uses implanted electrodes to collect movement signals from the brain and decode them into movement commands. It has been shown to restore voluntary motor impulses in patients with severe paralysis due to brain, spinal cord, peripheral nerve, or muscle dysfunction.
Implanted BCI devices have the potential to benefit people with severe disabilities by increasing their ability to interact with their environment, and consequently, providing new independence in daily life. While in infancy, the Food & Drug Administration has granted the implantable a “breakthrough device,” reinforcing the need to move this novel technology to the bedside of patients who need it most.
#9 AI for Early Detection of Sepsis
Sepsis is a severe inflammatory response to infection and is a leading cause of hospitalization and death worldwide. The World Health Organization estimates that it may cause 1 in 5 deaths globally.
It occurs when the immune system releases cytokines into the bloodstream rather than targeting a specific area. The sudden dysregulated immunity can lead to septic shock, a dangerous complication characterized by very low blood pressure and altered mental state.
Because septic shock has a very high mortality rate, early diagnosis of sepsis is critical. However, diagnosis can be complicated because the early symptoms are common across other conditions, and the current standard for diagnosis is non-specific. It can take up to two days to obtain results.
Artificial intelligence (AI) has surfaced as a new tool that can help rapidly detect sepsis in patients. Using AI algorithms, the tool detects several key sepsis risk factors in real-time by monitoring patients' electronic medical records as physicians input information. Flagging high-risk patients can help facilitate early intervention, which can improve outcomes, lower healthcare costs, and save lives.
#10 Predictive Analytics & Hypertension
Often referred to as the “silent killer,” hypertension, or high blood pressure usually shows no symptoms while increasing a person’s risk for serious health problems, including heart disease, heart failure and stroke. Effective treatment options exist; however, many adults remain unaware that they even have hypertension until they experience a significant health crisis.
Predictive analytic methods, which harness healthcare data to forecast disease risk, prognosis and treatment response, offer providers an opportunity to strip hypertension of its deadly nickname. Using these methods, providers can identify the warning signs of hypertension before it becomes severe and devise the most appropriate treatment strategies.
The early detection of any disease, coupled with targeted clinical interventions, will help reduce the cost of care and improve health outcomes. Predictive analytics equip providers with the key that could open the door to preventing hypertension and many other diseases.