Special Report

Eight Medical Breakthroughs of 2015

Baxter B. Allen, M.D.

Published: February 8, 2016 11:16 am

Last Updated: January 13, 2020 1:29 pm

Medical advancement is often a long and slow process — decades can sometimes pass before an amazing idea finally materializes into a practical application.

Developing drugs and other treatments often requires costly investments over the many phases of development and trials. Often, drugs that show promise in the early stages — in lab and animal testing — end up useless and even dangerous in humans. Despite these barriers, a handful of major discoveries are made each year, some of which can potentially lead to paradigm shifts in health care.

Some of these discoveries — such as a new drug for a common condition or a new surgical procedure — have immediate applications and impact the patient experience. Other discoveries change how we understand the human body, how future research is conducted, and may take far longer to have tangible outcomes in the real world.

Click here to see the eight medical breakthroughs of 2015.

In 2015, eight such advances took place. The long-term effect of these advancements can only be speculated on at this point, but in each case, there is at least some potential to change the way medicine is performed.

A few, such as the development of a new platform for antibiotic discovery has already had an immediate impact. Others are incremental, but substantial improvements in existing technologies that are likely to have a significant impact on health outcomes in the future. Finally, even the most substantial discovery is unlikely to have a major effect without a clinical trial first being completed or, more mundanely, being approved for use by the U.S. Food and Drug Administration.

1. Genetic therapy for cancer

For years, cancer treatment fell into three categories: chemotherapy, which refers to treatment with drugs that attack rapidly-dividing cells whether they are cancerous or normal; surgery to remove bulky tumors; and radiation, which uses high energy directed to specific spots to try to kill cancer cells left behind by surgery or chemotherapy.

Over the past decade, a fourth pillar has emerged: targeted therapies, which are customized drugs designed to target molecules involved in the growth and progression of specific cancers, as opposed to killing all cells dividing rapidly, as is the process in chemotherapy. These have had a huge impact in the treatment of previously difficult cancers and include such drugs as imatinib (marketed as Gleevec in the United States) for the treatment of certain blood cancers and trastuzumab (marketed as Herceptin) for breast cancer.

Over the last several years, institutions have been developing drugs and genetic therapies designed to activate a patient’s immune system to directly fight cancer — a field of treatments known as immunotherapy — and potentially a fifth pillar of cancer therapy.

In November, the treatment of a baby girl with incurable leukemia made international headlines due to the novel technique used. The baby received a transplant of immune cells from a healthy donor, which had been genetically modified using TALEN, a DNA-cutting enzyme. This modification prevented the transplanted cells from attacking the baby’s body and it made the new cells invisible to the drugs she was taking to fight the cancer itself — therefore allowing the transplanted immune cells to hunt and attack the cancer cells.

While this treatment made headlines, it has yet to be tested in clinical trials, and such off-the-shelf engineered immune cells are likely years away.

2. New HIV treatment

Treatments for HIV have been steadily improving since the devastating illness was discovered in the 1980s. However, no cure has been found, and the life-long treatments have many significant side effects, including liver damage, drug-induced diabetes, high cholesterol, and bone loss. Additionally, due to the disease’s high rate of mutation, resistance to these treatments have built up over time.

This year, a novel treatment was tested in preclinical studies in primates infected with an HIV-like virus (simian immunodeficiency virus, or SIV) and was shown to be highly effective in both treating and preventing infection. This approach uses gene therapy to cause the body to produce a protein that binds to the virus and effectively deactivates it. The protein, called eCD4-Ig, was also found to neutralize 100% of HIV subtypes known to be resistant to standard therapies. If these results are replicated in human trials, this could very well be a functional cure and immunization to HIV.

3. New Antibiotics and New Development Platform

Bacterial resistance to antibiotics is not a new problem, but it has become a more serious issue over the last few years. Superbugs, resistant to even the strongest antibiotics available, have started to appear. The widespread use of antibiotics in agriculture and farming, the use of household antibacterial soaps and cleaners and the overuse or improper use of antibiotics themselves in medical treatments all promote the survival and growth of highly-resistant superbugs.

Unfortunately, we have been slow to develop new antibiotics. The vast majority of antibiotic types were discovered from the late 1920s through the early 1960s, by culturing bacteria found in the soil and testing the chemicals they extrude against bacteria known to cause disease. Since that time, most new antibiotics have basically been chemically altered versions of those previously found, and we have been minimally successful in finding new ways to combat bacterial infection.

This year, researchers were successful in developing a new method of finding antibiotics. They were able to isolate an antibiotics compound effective against two bacterial species most well known for their ability to develop resistance: Staphylococcus aureus (i.e. MRSA) and Mycobacterium tuberculosis. While the discovery of a single active antibiotic agent is good news, the method of discovery is what makes this so important. Using traditional techniques, researchers are unable to culture roughly 99% of the bacterial species found in soil — and therefore cannot adequately study them. The new technique, called the iChip, allows for these bacteria to basically grow in their natural environment, sidestepping the need to grow them on a standard plate in a lab. In addition to increasing the likelihood of finding new antibiotics, this could be useful in the development of drugs for other conditions, such as cancer or inflammation.

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4. New treatments for Alzheimer’s 

Alzheimer’s disease is a major health care issue that will increasingly afflict many in the population. Already, more than 5 million Americans live with the disease, and with over 80,000 deaths attributed to Alzheimer’s as of 2013, it has become the sixth leading cause of death. The prevalence of the disease is expected to nearly triple by 2050 to roughly 14 million Americans. In addition to the high personal burden, there is a significant economic impact as well. As of 2015, Alzheimer’s disease was expected to cost the economy more than $200 billion. Without significant changes, that figure is expected to rise to over $1 trillion by 2050.

Unfortunately, while research in the field has been promising, the results have been underwhelming and there is no cure or meaningful treatment to date. The difficulty may be partly due to the fact that by the time Alzheimer’s is clinically apparent, it may have caused too much damage to the brain to be stopped or reversed. It is indeed now widely accepted that patients with Alzheimer’s have likely been accumulating damage for at least 10 to 20 years prior to the first clinical signs of deterioration. To detect this damage earlier, scientists have developed imaging and neuropsychological testing. This discovery has allowed new treatment techniques to focus on patients earlier in the course of their disease, when it may be still possible to make a difference. This year, data from a phase 1 trial of aducanumab, under development by Biogen, showed significant promise. For the first time, there was a reduction in amyloid plaques in the brains of patients with prodromal or mild Alzheimer’s who took the drug (the accumulation of amyloid plaque is thought to drive the development of Alzheimer’s). Also, there was a statistically significant slowing of clinical impairment in these patients. This was a very early study, but the findings are very encouraging.

5. New cholesterol lowering medications

Heart disease and stroke accounted for more than 740,000 deaths in 2013, representing the greatest and fifth greatest causes of death in the United States, respectively. Heart disease and stroke carry a significant overlap in risk factors: smoking, diabetes, high blood pressure, cholesterol, and many others. Many of these risk factors are modifiable through lifestyle changes and medication. Unfortunately, current medications are not perfect. In treating high cholesterol, statins have been a major success, but roughly 10%-15% of patients are unable to tolerate them due to side effects, and in 10% of patients, they do not adequately lower cholesterol.

This year, the FDA approved two new drugs, evolocumab and alirocumab, after phase 3 studies showed they were safe and effective in lowering cholesterol and reducing cardiovascular events. These drugs work under a different — and likely complementary — mechanism with statins in reducing cholesterol. Statins inhibit a protein, HMG-CoA reductase, which is central in the production of cholesterol by the liver, while the new drugs inhibit PCSK9, which helps to keep LDL (the “bad” cholesterol) in the bloodstream rather than allow its elimination. In this way, statins decrease the production of cholesterol, while PCSK9-inhibitors increase its removal.

6. New heart failure drug

Heart failure is diagnosed in more than 500,000 people per year and causes more than 50,000 deaths each year. With current medical therapy, roughly half of patients die within five years of diagnosis, and less than one-quarter are alive after 10 years.

In July, the FDA approved a new drug for the treatment of heart failure, Entresto. The medication is a combination of an old drug, valsartan, an angiotensin-receptor blocker, and a new compound, sacubitril, a neprilysin inhibitor. A large clinical trial comparing Entresto to enalapril, the gold-standard in the treatment of heart failure, was stopped early due to an overwhelming positive effect over a median follow-up of 27 months. Over this period, there was a nearly 20% reduction in observed death or first hospitalization due to heart failure in patients on Entresto. This amounted to a 2.8% decrease in death in a period just longer than two years. This is a greater effect than has been seen in the use of statins for the prevention of heart disease over a period twice as long.

7. Dengue vaccine

Dengue fever is a mosquito-borne disease endemic to more than 100 countries in the world, mostly in Africa, the Americas, the Middle East, Southeast Asia, and the Western Pacific. It infects an estimated nearly 400 million people per year with a severe, flu-like illness and can cause death in up to 10% of cases without early recognition and proper treatment. Over the last few decades, as global temperatures have risen, the disease has spread further in the Americas and increased in frequency from roughly 100,000 cases in the 1970s to roughly 900,000 as of 2007.

Like many infectious diseases, children and people with poorly functioning immune systems are at the highest risk of serious illness and death. To some degree, efforts have been made over the past 60 years to develop a vaccine. Recently, Sanofi Pasteur has developed a vaccine effective against this virus. In clinical trials recently performed, the vaccine provided protection to nearly 60% of the children who received the vaccine and reduced the incidence of the most severe forms of the disease by 80%. Finding an effective vaccine is the first step towards eradicating the virus.

8. Advances in the treatment of Acute Stroke

The rate of strokes and death from strokes has declined over the last few decades, likely due to a combination of factors such as lower smoking rates and improvements in medications for modifiable risk factors. However, there are still roughly 800,000 strokes in the United States per year, killing nearly 130,000 Americans. Many of those who survive also have significant disability, costing the economy more than $30 billion annually in lost productivity and health care costs.

Many of the strokes that are most likely to lead to significant disability or death are caused by blood clots that block one of the main arteries supplying blood flow to the brain. This past year, four large trials showed for the first time that when done within a few hours of the onset of strokes, catheters can be used to remove these clots to restore blood flow and halt further damage to the brain. The trials were were so successful that three of the four studies were stopped early due to overwhelming positive evidence.

When used appropriately, there was an absolute increase in the likelihood of functional independence of roughly 20% when compared to those treated with standard therapy alone. The use of clot retrieval devices also had no significant increased risk of any adverse outcomes.

While these new technologies provide those who suffer from a stroke with an even greater chance of recovery, what remains most important in treating acute strokes is the early recognition of symptoms and getting to a stroke center as quickly as possible.