Biotech Breakthroughs: Some advances of 2021 missed the headlines they deserve

While COVID-19 has dominated the airwaves for nearly 2 years, progress in other areas of health and biotech has continued. Because some of these innovations may not have gotten the attention they deserved, Matt Fuchs of leaps.org assembled a collection of noteworthy breakthroughs.

Attacking Alzheimer's

The FDA’s accelerated approval of aducanumab, the first drug that treats the underlying biology of Alzheimer’s, not just the symptoms, was a major story in 2021. Since Alzheimer’s is a complex disease, it is likely to need multiple treatment strategies. Many of these types of therapies advanced last year.

After the FDA action in June to approve aducanumab, there was excitement about drugs that target beta-amyloid, a protein that accumulates in the brain and leads to brain cell death. Among them are  donanemab from Eli Lilly, lecanemab from Eisai and gantenerumab from Roche, all of which attained Breakthrough Designation by the FDA in 2021.

AlphaFold Action

In 2021, Google made AlphaFold, an artificial intelligence system that opens the door to understanding the three-dimensional structures and functions of proteins, the building blocks that make up almost half of our bodies' dry weight, available for free. Since then, researchers have used it to drive greater understanding of how proteins interact. 

Once scientists understand the 3-D structures of proteins that cause various diseases, it will be much easier to design new drugs that can bind to these proteins and change their activity. Before AlphaFold, scientists had identified the 3-D structure of just 17 percent of about 20,000 proteins in the body. AlphaFold enables scientists to know the protein structures of 98.5 percent of the proteome.

Reimagined Virtual Care

Virtual First Care promises a new paradigm to fundamentally reimagine good healthcare. It differs from the ‘bolt on’ approach of video visits as an add-on to traditional visit-based, episodic care. V1C takes a much more holistic approach; it allows individuals to start care at any time in any place, recognizing that healthcare needs extend beyond regular hours. It matches the care setting with each individual’s clinical needs and personal preferences, advancing a thorough, evidence-based, safe practice. 

V1C puts the promise of digital health into practice. It is said to be the blueprint for what good healthcare looks like in the digital era.

Decentralized Clinical Trials

In 2021, digital- and data-enabled approaches have sustained decentralized clinical trials around the world. Throughout the pandemic, these decentralized trials have allowed patients to continue in studies with a reduced need for site visits, without compromising their safety or data quality.

Risk-based monitoring used data and algorithms to identify quality and safety issues without relying entirely on human monitors visiting research sites. Some trials used digital measures to ensure high quality data on target health outcomes that could be captured in ways that made the participants’ physical location irrelevant. More than three-quarters of research organizations, such as pharma and biotech, have accelerated their decentralized clinical trial strategies. 

Designing Biology

In June, scientists at the Brigham and Women’s Hospital published a paper showing that they had genetically engineered yeast – using CRISPR/Cas9 – to sense and treat inflammation in the body to relieve symptoms of irritable bowel syndrome in mice. This approach could potentially be used to address issues with the microbiome to treat other chronic conditions.

There was groundbreaking research on synthetic biology. Ginkgo Bioworks already claims to engineer cells with assembly-line efficiency. That could mean cleaner, more controllable, and affordable ways to manufacture food, therapeutics, and other materials in a factory-like setting.

Several scientists accelerated research to apply the mRNA technology underlying COVID-19 vaccines to make and replace proteins that cause rare conditions such as cystic fibrosis and multiple sclerosis. These applications of basic biology, coupled with incredible advances in computing, materials and drug delivery, hold the promise of progress.

Brain Biomarkers

There was real hope for identifying biomarkers that can predict neurodegenerative disease. Multiple biomarkers can diagnose disease and correlate with disease progression. Some of these biomarkers were detected in cerebrospinal fluid (CSF), while others were measured directly in blood by examining precursors of protein fibers. 

With the advent of omics-based approaches, coupled with new ultrasensitive analytical methods, researchers are beginning to identify informative brain biomarkers that may be able to detect earlier stages of disease when therapeutic intervention could be effective at halting progression.