From dancing molecules fixing spinal cord injuries to the large-scale production of antibiotics from CRISPR-edited bacteria, 2021 has provided reasons for hope during a depressing pandemic. Here, we describe our top 5 list from 2021 to remind us why we all do what we do.
The world has presented us with enormous healthcare challenges this year. So, it is no wonder that vaccine scientists won Time Magazine’s Heroes of 2021.
Although COVID-19 vaccines have been spectacularly effective and deserve the world’s gratitude in our opinion, we identified other notable breakthroughs worthy of your attention. The Radiators debated and voted on many different possibilities; it was a stimulating process and we finally settled on fivebreakthroughs. These five trailblazing advances exemplify science at its best, with all the attendant patience, persistence and hard work needed to make a difference.
Let us know what you think and what your favorite selection might be, or even better, what we missed.
5. Merck’s & Ridgeback Biotherapeutics, Molnupiravir
Merck has published that their antiviral pill, molnupiravir, can significantly reduce hospitalization and death among people with COVID-19 by half. It is the first oral pill of its kind and has already been approved for use in the United Kingdom, and in the U.S. under emergency-use authorization. Pfizer’s oral protease inhibitor has also just been approved and appears to provide better protection in preliminary experiments.
Merck’s nucleoside analogue introduces errors into the genetic code of coronavirus. Its easy oral administration means that it could become the first effective anti-COVID treatment to be given pre-hospital, thus relieving our over-burdened health services.
Like many great ideas, molnupiravir began its journey here in Atlanta, Georgia, at Emory University’s non-profit company Drug Innovation Ventures at Emory (DRIVE). Radyus Scientific advisory board member Dr. Dennis Liotta was instrumental in creating DRIVE’s new model for drug development almost 10 years ago, and molnupiravir provides unambiguous validation.
4. Gene-editing tech uses old expertise to revolutionize anti-bacterial therapy
The John Innes Centre researchers have created a new strain of Streptomyces formicae bacteria that over-produces antibiotics that target superbugs (such as MRSA).
Researchers created this strain of formicamycin using CRISPR/Cas9 genome editing. Unlike previous forms of Streptomyces formicae, where only small amounts of antibiotics were produced, this yields large quantities of molecules and can be scaled up much more easily. Given that the world is grappling with antibiotic resistance, this advance can revolutionize anti-bacterial therapy.
And why stop at anti-bacterial therapy? Excision BioTherapeutics uses this cutting-edge gene therapy to generate functional cures for HIV. Clinical trials are in the pipeline for this exciting treatment.
3. Perseverance has paid off for RAS mutation targeted therapies
Cancer biologists have known about the importance of RAS mutations in human cancer since 1982 and despite its importance, trials to introduce RAS-targeted treatments in tumor management have failed for several decades. Finally, Amgen’s sotorasib has changed all that, being the first approved targeted therapy for KRAS-mutant cancers this year.
Sotorasib is approved for adults with non-small cell lung cancer (NSCLC) whose tumors have a KRAS G12C mutation (the most common RAS mutation in lung adenocarcinoma) and have received at least one prior systemic therapy.
The sotorasib trial demonstrated an objective response rate of 37.1%. Although it may seem modest, it is a giant step forward in lung cancer treatment. We can expect that RAS targeting drugs will be used in other cancers and furthermore, that better ras targeting drugs will be developed.
“It seems to me that 2021 has indeed been an uplifting year for the biomedical science community. If decades of basic science had not taken place, we would be at the absolute mercy of our little enemy (SARS-CoV-2). While drug companies often get bad press, many people are seeing that biopharma companies provide essential medicines. We hope to do our part by building momentum on the preclinical side of things.” -Andrew Bush, Head of Research at Radyus
2. One blood test that detects 50 types of cancer
Galleri is a new blood test that can detect 50 types of cancer from a single blood draw. Within the US today, only five types of cancer are screened for, and the remaining 45 cancers account for 71% of cancer-related deaths.
This new blood test is more commonly referred to as a ‘liquid biopsy’. Like a solid biopsy, the goal is to detect cellular evidence of cancer (namely DNA). Unlike solid biopsies, a ‘liquid biopsy’ does not require a surgical procedure to obtain this information.
While much work remains to be done to document its utility, some experts in the field have said that Galleri has the opportunity to revolutionize cancer screening and lead to reductions in the human and economic toll of cancer.
1. Injectable ‘dancing molecules’ that can one-day reverse paralysis in humans
Northwestern University researchers have developed an incredible new therapy that harnesses ‘dancing molecules’ to reverse paralysis. Their injectable treatment was used to repair tissue in mice after severe spinal cord injury. Astonishingly, within four weeks, the mice had regained the ability to walk.
The treatment is injected as a liquid and immediately gets into a web of nanofibers that mimic the extracellular matrix of the spinal cord. This mimicking effect is key to the therapy’s success. It allows the molecules to ‘dance’ or even leap, connecting them more effectively with receptors involved in neural repair.
This therapy may pave the way to prevent paralysis after significant trauma, and researchers could even apply this new technology to other neurodegenerative disorders.
ready for 2022?
Great therapies start with great science. And great science is worth following and developing thoughtfully. Radyus’s team of experienced drug developers, committed scientists and resourceful project managers are ready to build on your novel research, too.
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