Slideshow
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Mobile stroke treatment unit
To improve stroke outcomes, a few hospitals in Germany and the United States are now using mobile stroke treatment units. Each unit, housed in an ambulance-type vehicle, has an onboard paramedic, critical care nurse, EMT, and CT technologist. A portable CT scanner takes brain images in 2 minutes, which are then sent via wireless link to hospital neurologists and neuroradiologists for diagnosis. Doctors then prescribe and direct treatment via a video connection.
Mobile stroke treatment units can treat strokes in as little as 11 minutes after arrival. Because the timing of stroke treatment is so crucial, these new units are giving patients the chance for a full recovery that they may not have had with traditional emergency care.
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Dengue vaccine
The World Health Organizations estimate that more than 50 to 100 million people in more than 100 countries develop dengue fever each year. Of those, 20,000 people die annually from the disease.
Researchers have previously struggled to create a dengue vaccine, primarily because the vaccine would have to simultaneously prevent four different (but closely related) strains of the virus: serotypes 1, 2, 3, and 4.
The world’s first dengue vaccine (Sanofi Pasteur) has just completed Phase III trials of 6,000 children aged 2 to 14 in five areas across Asia. Approximately 60% of the children were protected against the disease after a three-shot, 2-year period.
The vaccine protects against serotypes 3 and 4 better than serotypes 1 and 2, but the results can still mean a very significant reduction in medical costs, lost productivity, and human suffering. It is expected to reach the market by the end of 2015.
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The new art of blood collection and diagnosis
A new blood testing method offers a painless, more accurate, faster, and significantly less expensive way to take blood samples than traditional methods.
The nanotainer (Theranos) is designed to collect just a small drop of blood, taken from the capillaries at the end of the finger. The samples are shipped to a special, CLIA-certified laboratory that can perform hundreds of different tests using only that small drop of blood, and the results are sent back in just a few hours. Further testing can be done with the same blood sample.
The prices of different blood tests vary, but the price listed by Theranos is drastically less than current costs. The cost of a standard lipid panel currently varies from $10 to $10,000. Theranos’s test will cost $2.99.
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PCSK9 inhibitors for cholesterol reduction
Current statin medications are effective in lowering cholesterol, but in some patients, they do not reduce low-density lipoprotein cholesterol (LDL-C) levels enough to prevent cardiovascular events.
New medications called PCSK9 inhibitors affect the cholesterol receptor in the liver using a mechanism similar to statins, but completely novel. These news drugs reduce cholesterol to low levels never seen before.
The drugs are self-injected once or twice monthly. Currently, there are several PCSK9 inhibitors in various stage of development (including Evolocumab, mechanism pictured). The FDA is expected to approve the first of these in 2015.
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Antibody drug conjugates
A new class of drugs called antibody drug conjugates have been designed to treat certain advanced cancers that have spread to other parts of the body. They combine the antigen-targeting specificity of monoclonal antibodies with the cytotoxic potency of chemotherapeutic medications that specifically target a protein only found in and around tumor cells.
The drug works via antibodies that are specifically engineered, so patients’ immune systems won’t consider them foreign and thus try to destroy them. The antibodies are attached to several molecules of a potent toxin that targets cancer-cell proteins, and special linkers hold them all together. The conjugates are delivered intravenously, and work over the course of a few weeks to make their way through the bloodstream to target cancer cells.
More than two dozen antibody drug conjugates are currently in clinical trials, and several drugs have already been FDA-approved to treat advanced HER2-positive breast cancer and Hodg
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Immune checkpoint inhibitors
A class of drugs called immune checkpoint inhibitors are harnessing the power of the immune system to fight against cancer cells. These drugs block the actions of the proteins that usually allow cancer cells to hide from the immune system. The checkpoint inhibitors allow white T cells to destroy invading cancer cells.
These new drugs are resulting in significant long-term cancer remissions, and they have even better results when combined with standard cancer therapies. Two drugs have already been FDA-approved for metastatic melanoma (including the pictured Yervoy), and there is growing evidence that checkpoint inhibitors are effective against a growing number of cancers.
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Leadless cardiac pacemaker
Traditional cardiac pacemakers have a pulse generator with leads that stretch to the heart. Over time, the leads can break, the insulation around them can crack, and they can cause infections, which happens in 2% of cases. Dislodgement of leads occurs in more than 3% of cases. Repairing leads is not impossible, but it is difficult.
St. Jude Medical has introduced the Nanostim leadless pacemaker, which is wireless and only 10% the size of traditional pacemakers. The battery-controlled device can be implanted directly into the heart without surgery: In a 15- to 30-minute procedure, the pacemaker is steered through a femoral vein and into the heart’s right ventricle.
The FDA has not yet approved the device for use in the United States, but several late-stage clinical trials of leadless pacemakers are currently seeking FDA approval.
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New medications for idiopathic pulmonary fibrosis
Without a lung transplant, idiopathic pulmonary fibrosis (IPF) is fatal. Life expectancy after diagnosis is usually 3-to-5 years.
There had previously been no treatment for IPF, but two new drugs have been proven to significantly slow the progress of IPF in large international studies: pirfenidone (Esbriet, Roche) and nintedanib (Ofev, Boehringer Ingelheim).
Pirfenidone slowed disease progression after 13 weeks and also significantly improved lung function. The drug reduced mortality by 48% compared with placebo. Researchers are unsure what makes the drug effective, but it could be anti-inflammatory properties and its inhibition of a growth factor protein.
Nintedanib reduced patients’ annual rate of lung function decline by 48% and 55% in two Phase III studies, compared with 5% for those taking placebo. The drug works by blocking the effect of tyrosine kinases, which are proteins that alert the lungs to make scar tissue.
The FDA has approved both drugs fo
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Intraoperative radiation therapy for breast cancer
For women with early-stage breast cancer, a lumpectomy is usually followed by external beam radiation to the entire affected breast, given five times a week for 3 to 6 weeks. Many women stop therapy early due to time constraints, and whole-breast radiation can have adverse short- and long-term effects.
Intraoperative radiation therapy (IORT) is a new treatment for early-stage breast cancer. IORT is being used to deliver high doses of radiation during lumpectomies. After the tumor and surrounding tissue are removed from the breast, an applicator delivers radiation directly to the site of the tumor. IORT is delivered in a single, 30-minute dose.
A large clinical trial showed that IORT was as effective as whole breast radiation in preventing recurrence in early-stage breast cancer patients. IORT also decreased the risk of skin toxicity compared with traditional radiation treatment.
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Angiotensin-receptor neprilysin inhibitor for heart failure
Although there are currently no cures for heart failure, the number of management options is increasing. The current best treatment for heart failure is the use of angiotensin-converting enzyme (ACE) inhibitors.
A new drug called an angiotensis-receptor neprilysin inhibitor (ARNI) combines sacubitril, a neprilysin inhibitor, with an angiotensis receptor blocker. In an international study, ARNIs reduced cardiovascular death rates or hospitalization due to heart failure by 20% compared with ACE inhibitors. ARNIs also reduced the risk of death by 16%.
The FDA has given Fast Track status to the ARNI drug, currently known as LCZ696, and it is expected to be available in the United States by 2015.
