1. Dual-acting osteoporosis drug
Approved by the FDA, romosozumab is the first drug for osteoporosis that has a dual effect on both increase bone formation and decrease bone resorption. In a clinical trial published in the New England Journal of Medicine, women with postmenopausal osteoporosis who received romosozumab had a 50% reduced risk of spine fracture and a 38% reduced risk of hip fracture compared with those who took standard anti-resorption therapy (alendronate).
“Patients are getting back to daily activities without risk. The treatment is providing strength not only in bones but in spirit,” according to comments by the Cleveland Clinic.
2. Expanded use of minimally invasive mitral valve surgery
Minimally invasive surgery for mitral valve repair was introduced in 2013. The procedure involves an innovative transcatheter device that was FDA approved to repair the mitral valve in individuals with primary mitral regurgitation (MR) who aren’t eligible for open-heart surgery.
But, in March, the FDA broadened the device’s approval to also include patients with MR as a result of an enlarged left ventricle, known as secondary MR. These are patients in whom optimal medical therapy failed to relieve their symptoms.
Noted the Cleveland Clinic: “This expanded use of the minimally invasive method is bringing relief to more patients by removing some of the risk, fear, and inconvenience associated with cardiac surgery.”
3. First-ever treatment for transthyretin amyloid cardiomyopathy
Transthyretin amyloid cardiomyopathy is a progressive and potentially fatal disease in which amyloid fibrils, composed of misfolded transthyretin protein, deposit in the walls of the heart’s left ventricle. These deposits stiffen the muscle, which eventually leads to heart failure.
But a newly developed compound, tafamidis, binds to transthyretin to prevent misfolding of the deposited protein. In a high-profile clinical trial, patients on tafamidis had 30% lower all-cause mortality as well as a lower rate of cardiovascular-related hospitalizations compared with similar patients on placebo.
“Following Fast-Track and Breakthrough designations in 2017 and 2018, 2019 marked the FDA approval of tafamidis, the first-ever medication for the treatment of this increasingly recognized condition,” the Cleveland Clinic stated.
4. Immunotherapy for peanut allergies
Peanut allergy can be a nightmare for children and their parents. In September, an FDA expert panel recommended the approval of a first-of-its-kind oral immunotherapy treatment for peanut allergy in children a capsule with a minuscule amount of pharmaceutical-grade peanut protein. The immunotherapy’s dose is increased over time while the child builds up a tolerance to peanuts. In a double-blind phase 3 clinical trial, more than 3 in 4 children (76.6%) reached a daily maintenance dose of 300 mg the equivalent of one peanut.
“Though not a cure,” the Cleveland Clinic noted, “the breakthrough treatment lessens the worry of accidental exposure, easing the minds of parents who live in constant fear.”
5. Closed-loop spinal cord stimulation
Conventional spinal cord stimulation works by sending electric impulses along the spinal column through an implanted spinal cord stimulator, reducing the pain signals that reach the brain. However, each patient is prescribed a fixed dose of stimulation, which doesn’t take the individual’s movement into account, limiting its effectiveness.
Now, researchers have developed closed-loop spinal cord stimulation, which gets feedback from the patient’s spinal cord. The closed-loop system uses a stimulator that’s able to communicate in real-time with spinal cord neurons and modulate the dose of stimulation accordingly. It’s the first such system to measure the spinal cord’s response to stimulation and adjust each pulse according to the patient’s activity.
“With this technology, patients are getting measurable pain relief, sleeping better, and taking less medication,” according to the Cleveland Clinic. “Pending approval, closed-loop stimulation could be a saving grace for chronic pain patients everywhere.”
6. Biologics in orthopedic repair
People who’ve torn their anterior cruciate ligament (ACL) have a 20% chance of re-tearing it again. Now, researchers hope for better long-term results using the patient’s biologics cells, blood components, growth factors, and other natural substances to promote better healing and decrease inflammation in orthopedic injuries.
In conventional ACL repair, the torn ligament is replaced with autologous tissue. The new technique known as bridge-enhanced ACL repair (BEAR) uses a sponge injected with biologic factors in combination with the patient’s blood. This acts as a scaffold to stimulate the healing of the ACL, preserving the tissue instead of cutting it. A multicenter, randomized clinical trial using this procedure is now underway.
Other potential uses of biologics for orthopedic repair include its use in rotator cuff injuries and as an anti-infective coating for implant devices.
“Biologics holds the potential to provide every orthopedic patient a more natural, more effective, speedy recovery,” the Cleveland Clinic predicted.
7. Antibiotic envelope for preventing infection with cardiac implants
Cardiac implant devices such as pacemakers and defibrillators come with a risk of infection. But encasing these devices in an antibiotic “envelope” a mesh sleeve embedded with antibiotics ensures the slow delivery of 2 antibiotics, rifampin, and minocycline, for 7 days after implantation. The week-long release of antibiotics minimizes the risk of infection.
“The absorbable envelope received FDA clearance in 2013, but healthcare professionals were awaiting results from the landmark worldwide randomized WRAP-IT Worldwide Randomized Antibiotic Envelope Infection PrevenTion trial,” according to the Cleveland Clinic. “The findings published in March show a 40% reduction in major infections making cardiac implantable device procedures safer for patients.”
8. Bempedoic acid for patients who can’t take statins
Statins cause muscle pain in approximately 5% to 10% of patients who take them. A new agent, bempedoic acid, provides an alternative approach to lowering LDL cholesterol while avoiding these side effects.
Like statins, bempedoic acid works by blocking a key enzyme used by the body to make cholesterol. But unlike statins, it cannot accumulate in the muscle, reducing the likelihood of muscle pain.
“In clinical trials of the therapy, patients saw their LDL levels drop by an average of about [21%]. If approved by the FDA, bempedoic acid could be another addition to the arsenal of cholesterol-lowering treatments available to patients,” the Cleveland Clinic stated.
9. PARP inhibitors for ovarian cancer maintenance therapy
One of the most important advances in ovarian cancer treatment recently, poly-ADP ribose polymerase (PARP) inhibitors have improved progression-free survival in patients, and are now being approved by the FDA for first-line maintenance therapy in advanced-stage disease.
Until now, maintenance therapy hasn’t been widely explored in ovarian cancer. But in a landmark study published late last year, investigators showed a 70% reduction in the risk of disease progression or death at 3 years in participants using a PARP inhibitor approved for maintenance therapy.
The Cleveland Clinic predicted: “Several additional large-scale trials are underway, with PARP inhibitors set to make great strides in improving outcomes in cancer therapy.”
10. Diabetes drugs for heart failure with a preserved ejection fraction
No treatment is yet available for heart failure with preserved ejection fraction (HFpEF). However, sodium-glucose co-transporter 2 (SGLT2) inhibitors—currently used to lower blood glucose for the treatment of type 2 diabetes—are now being investigated for HFpEF.
Using SGLT2 inhibitors for this purpose became of interest when researchers reported that these drugs reduced the risk of cardiovascular death and heart failure hospitalization in a cohort of patients with type 2 diabetes. Individuals with heart failure and reduced ejection fraction without diabetes on SGLT2 inhibitors showed similar results. For patients with HFpEF, research is ongoing but promising.
“With an FDA decision anticipated in 2020, these drugs, among others, are introducing potential new treatment options for patients with this heart failure subtype,” the Cleveland Clinic noted.