Promising developments underway include advancements in gene and cell therapy, better testing for COVID, and a renewed focus on climate change.
Jared Auclair, Technical Supervisor for the Northeastern University's Life Science Testing Center in Burlington, Massachusetts, guides a COVID testing lab that cranks out thousands of coronavirus test results per day. His lab is also focused on monitoring the quality of new cell and gene therapy products coming to the market.
Here are trends Auclair and other experts are watching in 2021.
Better Diagnostic Testing for COVID
Expect improvements in COVID diagnostic testing and the ability to test at home.
There are currently three types of coronavirus tests. The molecular test—also known as the RT-PCR test, detects the virus's genetic material, and is highly accurate, but it can take days to receive results. There are also antibody tests, done through a blood draw, designed to test whether you've had COVID in the past. Finally, there's the quick antigen test that isn't as accurate as the PCR test, but can identify if people are going to infect others.
Last month, Lucira Health secured the U.S. FDA Emergency Use Authorization for the first prescription molecular diagnostic test for COVID-19 that can be performed at home. On December 15th, the Ellume Covid-19 Home Test received authorization as the first over-the-counter COVID-19 diagnostic antigen test that can be done at home without a prescription. The test uses a nasal swab that is connected to a smartphone app and returns results in 15-20 minutes. Similarly, the BinaxNOW COVID-19 Ag Card Home Test received authorization on Dec. 16 for its 15-minute antigen test that can be used within the first seven days of onset of COIVD-19 symptoms.
Home testing has the possibility to impact the pandemic pretty drastically, Auclair says, but there are other considerations: the type and timing of test that is administered, how expensive is the test (and if it is financially feasible for the general public) and the ability of a home test taker to accurately administer the test.
"The vaccine roll-out will not eliminate the need for testing until late 2021 or early 2022."
Ideally, everyone would frequently get tested, but that would mean the cost of a single home test—which is expected to be around $30 or more—would need to be much cheaper, more in the $5 range.
Auclair expects "innovations in the diagnostic space to explode" with the need for more accurate, inexpensive, quicker COVID tests. Auclair foresees innovations to be at first focused on COVID point-of-care testing, but he expects improvements within diagnostic testing for other types of viruses and diseases too.
"We still need more testing to get the pandemic under control, likely over the next 12 months," Auclair says. "The vaccine roll-out will not eliminate the need for testing until late 2021 or early 2022."
Rise of mRNA-based Vaccines and Therapies
A year ago, vaccines weren't being talked about like they are today.
"But clearly vaccines are the talk of the town," Auclair says. "The reason we got a vaccine so fast was there was so much money thrown at it."
A vaccine can take more than 10 years to fully develop, according to the World Economic Forum. Prior to the new COVID vaccines, which were remarkably developed and tested in under a year, the fastest vaccine ever made was for mumps -- and it took four years.
"Normally you have to produce a protein. This is typically done in eggs. It takes forever," says Catherine Dulac, a neuroscientist and developmental biologist at Harvard University who won the 2021 Breakthrough Prize in Life Sciences. "But an mRNA vaccine just enabled [us] to skip all sorts of steps [compared with burdensome conventional manufacturing] and go directly to a product that can be injected into people."
Non-traditional medicines based on genetic research are in their infancy. With mRNA-based vaccines hitting the market for the first time, look for more vaccines to be developed for whatever viruses we don't currently have vaccines for, like dengue virus and Ebola, Auclair says.
"There's a whole bunch of things that could be explored now that haven't been thought about in the past," Auclair says. "It could really be a game changer."
Vaccine Innovation over the last 140 years.
Max Roser/Our World in Data (Creative Commons license)
Advancements in Cell and Gene Therapies
CRISPR, a type of gene editing, is going to be huge in 2021, especially after the Nobel Prize in Chemistry was awarded to Emmanuelle Charpentier and Jennifer Doudna in October for pioneering the technology.
Right now, CRISPR isn't completely precise and can cause deletions or rearrangements of DNA.
"It's definitely not there yet, but over the next year it's going to get a lot closer and you're going to have a lot of momentum in this space," Auclair says. "CRISPR is one of the technologies I'm most excited about and 2021 is the year for it."
Gene therapies are typically used on rare genetic diseases. They work by replacing the faulty dysfunctional genes with corrected DNA codes.
"Cell and gene therapies are really where the field is going," Auclair says. "There is so much opportunity....For the first time in our life, in our existence as a species, we may actually be able to cure disease by using [techniques] like gene editing, where you cut in and out of pieces of DNA that caused a disease and put in healthy DNA," Auclair says.
For example, Spinal Muscular Atrophy is a rare genetic disorder that leads to muscle weakness, paralysis and death in children by age two. As of last year, afflicted children can take a gene therapy drug called Zolgensma that targets the missing or nonworking SMN1 gene with a new copy.
Another recent breakthrough uses gene editing for sickle cell disease. Victoria Gray, a mom from Mississippi who was exclusively followed by NPR, was the first person in the United States to be successfully treated for the genetic disorder with the help of CRISPR. She has continued to improve since her landmark treatment on July 2, 2019 and her once-debilitating pain has greatly eased.
"This is really a life-changer for me," she told NPR. "It's magnificent."
"You are going to see bigger leaps in gene therapies."
Look out also for improvements in cell therapies, but on a much lesser scale.
Cell therapies remove immune cells from a person or use cells from a donor. The cells are modified or cultured in lab, multiplied by the millions and then injected back into patients. These include stem cell therapies as well as CAR-T cell therapies, which are typically therapies of last resort and used in cancers like leukemia, Auclair says.
"You are going to see bigger leaps in gene therapies," Auclair says. "It's being heavily researched and we understand more about how to do gene therapies. Cell therapies will lie behind it a bit because they are so much more difficult to work with right now."
More Monoclonal Antibody Therapies
Look for more customized drugs to personalize medicine even more in the biotechnology space.
In 2019, the FDA anticipated receiving more than 200 Investigational New Drug (IND) applications in 2020. But with COVID, the number of INDs skyrocketed to 6,954 applications for the 2020 fiscal year, which ended September 30, 2020, according to the FDA's online tracker. Look for antibody therapies to play a bigger role.
Monoclonal antibodies are lab-grown proteins that mimic or enhance the immune system's response to fight off pathogens, like viruses, and they've been used to treat cancer. Now they are being used to treat patients with COVID-19.
President Donald Trump received a monoclonal antibody cocktail, called REGEN-COV2, which later received FDA emergency use authorization.
A newer type of monoclonal antibody therapy is Antibody-Drug Conjugates, also called ADCs. It's something we're going to be hearing a lot about in 2021, Auclair says.
"Antibody-Drug Conjugates is a monoclonal antibody with a chemical, we consider it a chemical warhead on it," Auclair says. "The monoclonal antibody binds to a specific antigen in your body or protein and delivers a chemical to that location and kills the infected cell."
Moving Beyond Male-Centric Lab Testing
Scientific testing for biology has, until recently, focused on testing males. Dulac, a Howard Hughes Medical Investigator and professor of molecular and cellular biology at Harvard University, challenged that idea to find brain circuitry behind sex-specific behaviors.
"For the longest time, until now, all the model systems in biology, are male," Dulac says. "The idea is if you do testing on males, you don't need to do testing on females."
Clinical models are done in male animals, as well as fundamental research. Because biological research is always done on male models, Dulac says the outcomes and understanding in biology is geared towards understanding male biology.
"All the drugs currently on the market and diagnoses of diseases are biased towards the understanding of male biology," Dulac says. "The diagnostics of diseases is way weaker in women than men."
That means the treatment isn't necessarily as good for women as men, she says, including what is known and understood about pain medication.
"So pain medication doesn't work well in women," Dulac says. "It works way better in men. It's true for almost all diseases that I know. Why? because you have a science that is dominated by males."
Although some in the scientific community challenge that females are not interesting or too complicated with their hormonal variations, Dulac says that's simply not true.
"There's absolutely no reason to decide 50% of life forms are interesting and the other 50% are not interesting. What about looking at both?" says Dulac, who was awarded the $3 million Breakthrough Prize in Life Sciences in September for connecting specific neural mechanisms to male and female parenting behaviors.
Disease Research on Single Cells
To better understand how diseases manifest in the body's cell and tissues, many researchers are looking at single-cell biology. Cells are the most fundamental building blocks of life. Much still needs to be learned.
"A remarkable development this year is the massive use of analysis of gene expression and chromosomal regulation at the single-cell level," Dulac says.
Much is focused on the Human Cell Atlas (HCA), a global initiative to map all cells in healthy humans and to better identify which genes associated with diseases are active in a person's body. Most estimates put the number of cells around 30 trillion.
Dulac points to work being conducted by the Cell Census Network (BICCN) Brain Initiative, an initiative by the National Institutes of Health to come up with an atlas of cell types in mouse, human and non-human primate brains, and the Chan Zuckerberg Initiative's funding of single-cell biology projects, including those focused on single-cell analysis of inflammation.
"Our body and our brain are made of a large number of cell types," Dulac says. "The ability to explore and identify differences in gene expression and regulation in massively multiplex ways by analyzing millions of cells is extraordinarily important."
Converting Plastics into Food
Yep, you heard it right, plastics may eventually be turned into food. The Defense Advanced Research Projects Agency, better known as DARPA, is funding a project—formally titled "Production of Macronutrients from Thermally Oxo-Degraded Wastes"—and asking researchers how to do this.
"When I first heard about this challenge, I thought it was absolutely absurd," says Dr. Robert Brown, director of the Bioeconomy Institute at Iowa State University and the project's principal investigator, who is working with other research partners at the University of Delaware, Sandia National Laboratories, and the American Institute of Chemical Engineering (AIChE)/RAPID Institute.
But then Brown realized plastics will slowly start oxidizing—taking in oxygen—and microorganisms can then consume it. The oxidation process at room temperature is extremely slow, however, which makes plastics essentially not biodegradable, Brown says.
That changes when heat is applied at brick pizza oven-like temperatures around 900-degrees Fahrenheit. The high temperatures get compounds to oxidize rapidly. Plastics are synthetic polymers made from petroleum—large molecules formed by linking many molecules together in a chain. Heated, these polymers will melt and crack into smaller molecules, causing them to vaporize in a process called devolatilization. Air is then used to cause oxidation in plastics and produce oxygenated compounds—fatty acids and alcohols—that microorganisms will eat and grow into single-cell proteins that can be used as an ingredient or substitute in protein-rich foods.
"The caveat is the microorganisms must be food-safe, something that we can consume," Brown says. "Like supplemental or nutritional yeast, like we use to brew beer and to make bread or is used in Australia to make Vegemite."
What do the microorganisms look like? For any home beer brewers, it's the "gunky looking stuff you'd find at the bottom after the fermentation process," Brown says. "That's cellular biomass. Like corn grown in the field, yeast or other microorganisms like bacteria can be harvested as macro-nutrients."
Brown says DARPA's ReSource program has challenged all the project researchers to find ways for microorganisms to consume any plastics found in the waste stream coming out of a military expeditionary force, including all the packaging of food and supplies. Then the researchers aim to remake the plastic waste into products soldiers can use, including food. The project is in the first of three phases.
"We are talking about polyethylene, polypropylene, like PET plastics used in water bottles and converting that into macronutrients that are food," says Brown.
Renewed Focus on Climate Change
The Union of Concerned Scientists say carbon dioxide levels are higher today than any point in at least 800,000 years.
"Climate science is so important for all of humankind. It is critical because the quality of life of humans on the planet depends on it."
Look for technology to help locate large-scale emitters of carbon dioxide, including sensors on satellites and artificial intelligence to optimize energy usage, especially in data centers.
Other technologies focus on alleviating the root cause of climate change: emissions of heat-trapping gasses that mainly come from burning fossil fuels.
Direct air carbon capture, an emerging effort to capture carbon dioxide directly from ambient air, could play a role.
The technology is in the early stages of development and still highly uncertain, says Peter Frumhoff, director of science and policy at Union of Concerned Scientists. "There are a lot of questions about how to do that at sufficiently low costs...and how to scale it up so you can get carbon dioxide stored in the right way," he says, and it can be very energy intensive.
One of the oldest solutions is planting new forests, or restoring old ones, which can help convert carbon dioxide into oxygen through photosynthesis. Hence the Trillion Trees Initiative launched by the World Economic Forum. Trees are only part of the solution, because planting trees isn't enough on its own, Frumhoff says. That's especially true, since 2020 was the year that human-made, artificial stuff now outweighs all life on earth.
More research is also going into artificial photosynthesis for solar fuels. The U.S. Department of Energy awarded $100 million in 2020 to two entities that are conducting research. Look also for improvements in battery storage capacity to help electric vehicles, as well as back-up power sources for solar and wind power, Frumhoff says.
Another method to combat climate change is solar geoengineering, also called solar radiation management, which reflects sunlight back to space. The idea stems from a volcanic eruption in 1991 that released a tremendous amount of sulfate aerosol particles into the stratosphere, reflecting the sunlight away from Earth. The planet cooled by a half degree for nearly a year, Frumhoff says. However, he acknowledges, "there's a lot of things we don't know about the potential impacts and risks" involved in this controversial approach.
Whatever the approach, scientific solutions to climate change are attracting renewed attention. Under President Trump, the White House Office of Science and Technology Policy didn't have an acting director for almost two years. Expect that to change when President-elect Joe Biden takes office.
"Climate science is so important for all of humankind," Dulac says. "It is critical because the quality of life of humans on the planet depends on it."
In the U.S. and Europe, it is illegal to reuse pacemakers and other implants. Therefore, cardiologists export them to the global South where they save the lives of people of all ages.
Often he observed there were no doctors in the E.R.s, and hte nurses could render only basic first aid. “When somebody fell into a coma, they fell into a coma,“ Israel remembers. “There weren’t even any defibrillators to restart a patient’s heart,” while defibrillators are standard equipment in most clinics in the U.S. and Europe as lifesaving devices. When Israel finally visited the largest and most modern hospital in Nairobi, he found it better equipped but he learned that its services were only available to patients who could afford them. The cardiologist there had a drawer full of petitions from patients with heart ailments who couldn’t afford lifesaving surgery. Even two decades ago, a pacemaker cost $5,000 in Kenya, which made it unaffordable for most Kenyans who earn an average of $600 per month.
Since 2003, Israel and a team of two doctors and two nurses visit Kenya and Zambia once or twice a year to implant German pacemakers for free. Notably, the pacemakers and defibrillators Israel exports to Africa would end up in the landfill in Germany. Clinics have to pay for specialized services to dispose of this medical equipment. “In Germany, I could go to jail if I used a defibrillator that is one day past its expiration date,“ Israel says, “but in Kenya, people don’t have the money for the cheapest model. What nonsense to throw this precious medical equipment away while people in poorer countries die because they desperately need it.“
Israel works at the breakpoint between the laws in a wealthy country like Germany and the reality in the global South. The U.S. and most European countries have strict laws that ban the reuse of medical implants and enforce strict expiration dates for medical equipment. “But if a pacemaker is a few days past its expiration date, it still works perfectly fine,“ Israel says. “And it also happens that we implant a pacemaker and five months later it turns out that the patient needs a different kind. Then we replace it and we’d have to trash the first one in Germany, though it could easily run another 12 years.“
“If we get this right, we have lots of devices we can implant, hips and knees, etcetera. Where this will lead is limitless," says Eva Kline Rogers, the program coordinator for My Heart, Your Heart.
Israel has been collecting donations of pacemakers and defibrillators from manufacturers but also from other doctors and from funeral homes for his nonprofit Pacemakers for East Africa since 2003. Most funeral homes in the U.S. and Europe are legally obliged to remove pacemakers from the dead before cremation. “Most pacemakers survive their owners,“ says Israel. He sterilizes the pacemakers and finds them a new life in East Africa. Studies show that reused pacemakers carry no greater risk for the patients than new ones.
In the U.S., University of Michigan professor Thomas Crawford heads up a similar initiative, My Heart, Your Heart. “Each year 1 to 2 million individuals worldwide die due to a lack of access to pacemakers and defibrillators,” the organization notes on its website. The nonprofit was founded in 2009, but it took four years for the doctors to get permission from the FDA to export pacemakers. Since receiving permission, the organization has sent dozens of devices to the Philippines, Haiti, Venezuela, Kenya, Sierra Leone and Ukraine. “We were the first doctors ever to implant a pacemaker in Sierra Leone in 2018,” says Crawford, who has traveled extensively to most of the recipient countries.
Even individuals can donate their pacemakers; the organization offers a prepaid envelope. “My mother recently passed and she donated her device,” says Tina Alexandris-Souphis, one of the doctors at University of Michigan who collaborates on My Heart, Your Heart. The organization works with World Medical Relief and the U.K. based charity Pace4Life to maintain a registry of the most urgent patients and send devices to where they are needed the most.
My Heart, Your Heart is also conducting a randomized controlled trial to provide further evidence that reused pacemakers pose no additional risk. “Our vision is that we establish this is safe and create a blueprint for organizations around the world to safely reuse these devices instead of them being thrown in the trash,” says Eva Kline Rogers, the program’s coordinator. “If we get this right, we have lots of devices we can implant, hips and knees, etc. Where this will lead is limitless.” She points out that in addition to receiving the donated devices, the doctors in the global South also benefit from the expertise of renowned cardiologists, such as Crawford, who sometimes advise them in complex cases.
And Adrian Baranchuk, a Canadian doctor at the Kingston General Hospital at the Queen’s University, regularly travels through South America with his “cardiology van” to help villagers in remote areas with heart problems.
Israel says that he’s been accused of racism, in thinking that these pacemakers are suitable for those in the global South - many of whom are people of color - even though officials in wealthier countries consider them to be trash. The cardiologist counters such criticism with stories about desperate need of his patients. At his first medical visit to Nairobi that he organized with a local cardiologist, six patients were waiting for him. “In Germany, they would all be considered emergencies,” Israel says. One eighty-year old grandmother had a heartrate of 18. “I’ve never before seen anything like this,” Israel exclaims. “At first I thought I couldn’t find her pulse before I realized that her heart was only beating once every three seconds.” After the surgery, she got up, dressed herself and hurriedly packed her bag, explaining she had a ton of work to accomplish. Her family was in disbelief, Israel says. “They told me she had been bedridden for five years because as soon as she tried to get up she would faint.”
Israel has been accused of racism, in thinking that these pacemakers are suitable for those in the global South even though they're considered to be trash by officials in wealthier countries. The cardiologist counters such criticism with stories about desperate need of his patients.
Carsten Israel
The hospital in Nairobi where Israel conducts the surgeries, charges patients $200 for the use of its facilities. If patients can’t afford that sum, Israel will pay it from the funds of his nonprofit. For some people, $200 far exceeds their resources. Once, a family from the extremely poor Northern region of Kenya told him they couldn’t afford the $3 for the bus ride to Nairobi. Israel suspected this was a pretense because they were afraid of the surgery and agreed to reimburse the $3, “but when they came, they were wearing rags and were so rail-thin, I understood that they really needed every cent they had for food.”
Israel is a renowned cardiologists in Germany. And yet, he considers his work in East Africa to be particularly meaningful. “Generally, most patients in Germany will get the treatment they need,” he says, “and I never before experienced that people have an illness that is easily curable but simply won’t be treated.” He also feels a heavy responsibility. Many patients have his personal cell phone and call him when they have problems or good news about how they’re doing.
Some of those progress reports come much faster than in Israel’s home country. Before he implanted a pacemaker in a tall Massai in Kenya, the man had been picked on by his family because he wouldn’t help much with the hard work on the family peanut farm. “When I examined him, he had a pulse of 40,” Israel says. “It’s a miracle he was even standing upright, let alone hauling heavy bags.” After the surgery, Israel advised his patient to stay the night for observation, but the patient couldn’t wait to leave. Two hours later, he returned, covered in sweat. He’d been running sprints with his brothers to test the new device. Israel shakes his head. In Germany, it would be unthinkable for a patient to engage in athletics immediately after surgery. But the patient was exuberant: “I was the fastest!”
The success stories are notable partly because the challenges remain so steep. In Zambia, for instance, there is a single cardiologist; she determined to become one after losing her younger sister to an easily curable heart disease. Often, the hospitals not only lack pacemakers but also sterile surgery equipment, antibiotics and other essential material. Therefore, Israel and his team import everything they need for the surgeries, including medication. If necessary, they improvise. “I’ve done surgery with a desk lamp hanging from the ceiling by threads,” Israel says. He already knows that he will need to return to Kenya in six months to replace the pacemaker of one of his patients and replace the batteries in others. If he doesn’t travel, lives are at risk.