The Important Medical Innovations of 2021 – Healthline

Its almost the end of 2021, and it has certainly been quite the year for stories about public health and healthcare.

Once we reach 2022, it will be 2 full years of the COVID-19 pandemic, which has reoriented how we approach our lives and the world around us, and how we relate to healthcare.

With the pandemic still looming, the past year was also one that marked a range of health innovations both large and small including the application of breakthrough CRISPR gene-editing technology, the World Health Organizations approval of the first malaria vaccine, and new approaches to cardiology.

Healthline spoke with several top experts from a range of medical and public health disciplines about what some of the defining, under-the-radar, and impactful healthcare innovations were of 2021.

Not only do they all shed a spotlight on a year of innovation, but they also point to what we can expect more of in the future.

A retrospective on the major impact health innovation has made on society cannot be done without touching on mRNA vaccines.

Some of the COVID-19 vaccines that are currently being distributed think the Pfizer-BioNTech and Moderna vaccines have been developed as a result of a gene-editing process that modifies RNA (mRNA) to trigger an immune response in the body.

Lior Brimberg, PhD, an assistant professor in the Institute of Molecular Medicine at the Feinstein Institutes for Medical Research in Manhasset, New York, told Healthline that the mRNA-based vaccines being given to millions of people to fight the coronavirus have changed the narrative, not only for dealing with the COVID-19 pandemic, but for future viruses.

In fact, there are ongoing trials to assess the efficacy of potential mRNA-based influenza and HIV vaccines.

This technology will allow developing vaccines rapidly and more efficiently against new and current viruses, Brimberg added.

Eric H. Chang, PhD, another assistant professor at the Bioelectronic Medicine at the Feinstein Institutes for Medical Research, echoed those points.

He told Healthline that while mRNA vaccine technology might have seemed like a 2020 headline, the ramp up of vaccine production and possibility for variant-specific modifications is truly unique.

Chang added that the development of more effective at-home COVID-19 rapid tests that have very high accuracy are an inflection point in the continued fight against the pandemic.

They enable the average citizen to quickly know whether they are positive and change their behavior without having to go to a clinic or a lab, Chang said.

In October 2020, Emmanuelle Charpentier and Jennifer A. Doudna made history by winning the Nobel Prize in chemistry for their landmark development of gene-editing technology, CRISPR/Cas9.

To break down what this is in simple terms, think of it as genetic scissors enzymes that cut away portions of DNA and return them to their normal function.

This genetic technology from Charpentier and Doudna can strategically snip away at any kind of DNA molecule at a specific location.

Last year, the heralding of CRISPR technology signified new horizons in fighting a range of diseases in a new way. Now, experts say we are seeing that theoretical promise in real time.

This year, it was shown the first time that this technology can be used in humans. A CRISPR-based drug was injected to the blood of people born with a disease called transthyretin amyloidosis that causes fatal disease, and shown that it decreased substantially the production of toxic protein by their livers, Brimberg explained. This is a milestone in mRNA-based drugs.

Dr. Donald Lloyd-Jones, ScM, FAHA, the president of the American Heart Association, told Healthline that this application of gene editing has big implications for heart health.

Lloyd-Jones explained some people have a gene that facilitates the production of too much of a toxic protein that builds up in the heart muscle over time.

He said this makes the heart get too thick and gets hard to pump efficiently. People can then develop heart failure.

The CRISPR/Cas9 technology was able to target this gene defect to get it to stop making too much of the protein, showing dramatic reductions in the blood levels of that protein production by as much as 90 percent in the blood, so it wont continue to build up in the heart muscle, Lloyd-Jones said.

This particular demonstration of this technology in this disease shows that we can make a real living person actually get this technology in there to edit the gene and stop their liver from making so much of that bad protein that is causing their heart failure, he said. Its really interesting, and I think an important demonstration that, when taken to a scalable level, could really alter the course of a uniformly fatal disease.

Lloyd-Jones said its remarkable to look at the difference the course of a year has made when it comes to this technology and its application in the real world.

There was promise, but now we are seeing the actual application of it, he said.

Additionally, Lloyd-Jones pointed to the example of this gene editing being used to treat Duchenne muscular dystrophy, a genetic disorder that affects boys that is characterized by a progression of muscle degeneration due to shifts that occur in dystrophin, a protein that helps keep your muscle cells in a normal state.

This year, Lloyd-Jones pointed to scientists in Dallas, Texas, who were able to take cells from some of these boys, modify them back to normal function through gene editing, and return them to potentially improve their heart abnormalities.

Beyond these examples, Lloyd-Jones pointed to gene editing potentially being used in more common conditions.

For instance, he pointed to how it might work with people who have familial hypercholesterolemia, a genetic disorder in one gene that means they have difficulty with clearing cholesterol from their blood because they dont make the receptor needed to clear that cholesterol.

The right protein doesnt work to clear the cholesterol from their blood, but what if we could give them back the opportunity to make the right protein and those receptors could work again? Lloyd-Jones said.

These are people who have heart attacks in their teens and 20s. If we could repair that gene and give it back to them, then thats not a one-time thing. You give them back their cell with a normal gene on multiple occasions. You then have an opportunity to extend these peoples lives immensely, he said.

Essentially, the Nobel Prize-winning breakthrough of 2020 is now being used in a way that shows promise not just for treating rare diseases, but tackling more common problems down the line.

In October 2021, World Health Organization (WHO) officials announced they had officially approved the widespread use of the RTS,S/AS01 (RTS,S) malaria vaccine for children in sub-Saharan Africa as well as other areas with high transmission of the Plasmodium falciparum parasite that causes malaria.

At the time, WHO officials said this recommendation was due to the results of an ongoing pilot program that has been used among 800,000 children since 2019 in the countries of Ghana, Kenya, and Malawi.

This is a historic moment. The long-awaited malaria vaccine for children is a breakthrough for science, child health, and malaria control, said Dr. Tedros Adhanom Ghebreyesus, WHO director-general. Using this vaccine on top of existing tools to prevent malaria could save tens of thousands of young lives each year.

Malaria kills about half a million people each year, nearly half of them are children under the age of 5. The new vaccine, made by GlaxoSmithKline and endorsed by WHO, will help prevent many of those cases, Brimberg explained.

When asked to put into context the medical and public health significance of this vaccine, Philip Welkhoff, director for malaria at the Bill & Melinda Gates Foundation, told Healthline that this is not just the first malaria vaccine; its the first vaccine to target a parasite.

He called it a historic milestone in vaccine development, scientific innovation for malaria, and the potential for long-term public-private partnerships.

When introduced for African children under 5 next year and in a targeted way as a complement to existing malaria interventions in eligible countries it can help save tens of thousands more children from malaria each year, Welkhoff said.

This breakthrough didnt happen in a vacuum.

Welkhoff said it builds on a set of effective innovations that, when used in concert, have been instrumental in achieving progress against this deadly yet preventable and treatable disease.

This first-generation vaccine also has paved the way for a robust pipeline of more effective, durable, and potentially game-changing tools that emerged from the research and development [that] the Gates Foundation supported during late-stage development, he added.

Welkhoff recently wrote a piece about how effective these tools can be in fighting the disease.

While many of these transformative tools are still years away from being available in malaria-affected countries, with increased investments now in malaria R&D [research and development], we can accelerate their development and delivery within the decade. These tools are necessary for achieving eradication and the goal of a malaria-free world, he explained.

Welkhoff added that in recent years, countries have diverged from what he called a one-size-fits-all approach to malaria service delivery.

He said that countries, along with organizations such as the WHO, Global Fund, RBM Partnership to End Malaria, U.S. Presidents Malaria Initiative, and Gates Foundation, need to make strategic decisions about how to optimize use of a limited supply of the vaccine alongside other effective and cheaper lifesaving malaria prevention tools and strategies.

Using real-time, high-quality data, countries can maximize impact by determining and targeting the best mix of prevention tools on a district-by-district basis to rapidly reduce malaria cases and deaths, Welkhoff added.

Moving forward, he said we will need to see more effective vaccines, radical cure drugs, cheaper and more effective vector control, and stronger diagnostics. These efforts will be needed to achieve malaria eradication.

In addition, we need to increase the use of sophisticated surveillance tools, like genomic surveillance, and the use of community case management to track malaria transmission as well as the spread of drug and insecticide resistance, Welkhoff said.

This information enables countries to pursue more strategic, targeted responses, stay ahead of the ever-evolving malaria parasite and its mosquito host, and stop malaria transmission in its tracks.

With more investment in malaria R&D, we can accelerate the development and delivery of innovative technologies that, in turn, can usher us into a new era of rapid decline in malaria cases and deaths towards ending malaria for good, Welkhoff said.

Chang cited several examples of technology outside of gene editing that have made strides in simplifying some common issues in healthcare.

One example is Bluetooth-enabled pacemakers, which he said allow rapid and frequent communication between a body-implanted device and clinicians.

Additionally, he cited another common technology that became particularly popular during the height of precipitated COVID-19 lockdowns: telemedicine.

Large-scale telemedicine, which had to be rolled out due to the pandemic, but the improvements in quality and accessibility this year were significant, Chang added.

Beyond high tech pacemaker technology and the increasingly normalized application of telemedicine as a part of our daily lives, Chang highlighted machine learning powered by artificial intelligence (AI).

Machine learning in digital pathology [is impactful] because we can now leverage AI-based analytics to diagnose tissue biopsies for earlier disease detection, Chang said.

Lloyd-Jones said one of the nice things about working in the cardiovascular health space is the fact that there is so much research happening all the time that really moves the field forward.

He said that 20 years ago there was almost nothing that could help treat heart failure. That has changed.

Weve got drugs that prolong lives, prevent heart failure hospitalizations, the ability to really change the quality of life for our heart failure patients. Its a whole new world, he said. Some of my colleagues have changed from calling it heart failure to heart success.

This year, Lloyd-Jones said, we have witnessed trials and data looking at a newer class of drugs called sodium-glucose cotransporter-2 (SGLT2) inhibitors.

They are designed to be diabetes drugs that are really effective at treating heart failure, at reducing cardiovascular death, reducing heart failure hospitalizations, and really changing the natural history of heart failure and improving heart function, he said. I think it has been the story of the last few years and this year the trials continue to show just how powerful that class of drugs is.

Inhibitors are really very safe and extremely effective, and will be an important part of what we do for our patients moving forward, he added.

Lloyd-Jones pointed to the Salt Substitute and Stroke Study out of China this year as a research breakthrough that constructs a road map for how we can rethink our approach to sodium consumption.

He said this research tackles an age-old question of how significant dietary salt intake is in affecting heart attack and stroke risk.

Debates have raged of does it matter, should we be lowering the sodium content of the food supply, should people be cutting back their salt that they add to their foods? he explained.

Whats happening in our country is there is so much sodium in our processed food supply in America. In places like China, its more about what sodium people are putting in their diets in the cooking process, Lloyd-Jones said.

In this salt study, nearly 21,000 people in China were surveyed who were either over age 60 or had high blood pressure that was not well managed.

Researchers randomized the villages that made up the study population size into different groups. Some used a salt substitute, while others continued to use just regular salt, or sodium chloride, in their food when they cooked.

In short, stroke risk, major cardiovascular events, and death were all reduced in those using the salt substitute compared with those using normal salt.

Lloyd-Jones said this bears significance for what we should be looking at moving forward in the United States.

We consume a lot of salt in common processed food items that we might not even realize. If we can take a cue from this years study and consume less salt, it could provide a public health benefit.

If you can shift the blood pressure of a population the average blood pressure lowered by just 2 or 3 millimeters of mercury sounds like a very little bit but shifting the population average, you are reducing risks for heart attacks, for strokes, for heart failure, in essentially the double digits, Lloyd-Jones explained.

With this reduction in terms of 10 to 20 percent, just an average amount of change in the average blood pressure, then hundreds of thousands to potentially millions of heart events could be prevented a year, he said.

Medical research and attempts to close dangerous public health gaps are ongoing.

Brimberg pointed to research this year where developmental biologists devised a method for growing mouse embryos outside a uterus from pre-gastrulation to advanced stages longer than ever before.

He said this will bring about greater understanding of the developmental processes that lead to the formation of tissues and will allow controlled environment to understand the effect of exposure in utero to various factors that might alter the normal process of development.

Brimberg also mentioned greater strides in making insulin more affordable. He cited the Food and Drug Administration (FDA) approval of Semglee, the first interchangeable biosimilar product for the treatment of diabetes.

Approval of these insulin products can substantially reduce the cost for treating diabetes. This is a milestone for people in the U.S. approximately 7 million people who rely daily on insulin, he said.

Essentially, no list of medical advancements for any year can be comprehensive or complete. There are countless innovations being made to make life better for more people and to address some of the most pressing health issues of our day.

For his part, Welkhoff said events such as the rapid development and manufacturing of the COVID-19 vaccines and the WHOs recommendation for the malaria vaccine reveal this to be a year when a combination of significant investment, private-public partnership, and collective political leadership can speed up and unlock scientific innovation that is having huge, lifesaving impact.

Chang said many of the innovations that resonated for him are technology oriented, reflecting how healthcare is changing.

Healthcare is becoming more digital, partly out of necessity during 2021, but this change has also made it more accessible with the potential to reach more individuals who might otherwise not be able to reach a healthcare provider, Chang said.

As we turn the page on one year and look ahead to another, we can be sure that medicine, science, and public health will continue to move ahead with new innovations that will make their mark.

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The Important Medical Innovations of 2021 - Healthline