MARCH 15, 2021 -- LeapsMag, the award-winning online magazine created to encourage public discussion about scientific innovation, re-emerges today as Leaps.org, a non- profit media initiative within the Good Worldwide ecosystem, dedicated to rebuilding public trust in science as a force for good and fostering dialogue about the ethical implications of new breakthroughs. Leaps.org's news and commentary cover a wide range of topics including health and medicine, biotechnology, agriculture, research and development, space exploration, and environmental concerns. Notable contributors and interviewees include neuroscientist Sam Harris, geneticist George Church, Nobel Prize winner Eric Kandel, author Steven Pinker, virologist Angela Rasmussen, and many others.
Science and the media that report on it are facing unprecedented mistrust and suspicion, yet at the same time the COVID-19 pandemic has generated a growing public appetite for accessible information about scientific developments. President Biden has tasked his Surgeon General nominee Vivek Murthy with improving public trust in science as one of his key goals. The Kaiser Family Foundation reported in January that roughly 3 in 10 U.S. health care workers express hesitancy about getting a COVID-19 vaccine. A September 2020 Pew Research Center study found that "majorities across 18 of the 20 publics say that limited public understanding is a problem for coverage of scientific research."
And Edelman Worldwide released global survey results showing that trust in scientists and journalists is down compared with last year, and trust in all information sources is at record lows: "In a world of misinformation and media echo chambers," Edelman stated, "how can we rebuild the trust needed to enable the acceptance of science and innovation to create a brighter future for humanity?"
That's where Leaps.org comes in. The original platform was launched by science writer Kira Peikoff in 2017 as an editorially independent outlet for high-quality journalism and commentary, with financial support from Leaps by Bayer, the impact investment unit of Bayer that invests in paradigm-shifting technologies. Leaps by Bayer holds the conviction that responsible innovation requires broad public engagement on a neutral platform that is free of sponsor bias.
Leaps.org's transition to becoming a non-profit media outlet underscores the publication's guiding principle: total editorial independence. Editor-in-Chief Peikoff ensures that funders have no influence over the content published, including no veto power or advance viewing. She will be expanding key partnerships, special events, and philanthropic projects. "In light of the magnified confusion and suspicion raised by the pandemic, there's never been a more important time for science and media to join together," said Peikoff. "We need to highlight the key role of scientific progress in securing society's future while aggressively countering misinformation and breaking down barriers to inclusive discussion. Leaps.org accomplishes these goals through accessible and accurate storytelling, using the highest caliber sources and rigorous fact-checking." Because Leaps.org is not reliant on a revenue-generating model, its journalism is not tied to conventional performance-driven metrics.
"Scientific progress could change the world for the better, but advances will only have impact if people understand the benefits and feel empowered to ask questions. Stimulating this dialogue has never been more important," said Dr. Jürgen Eckhardt who heads up Leaps by Bayer. "We applaud the evolution of Leaps.org into a non-profit initiative that can realize its mission on a larger scale."
"Right now, a healthy relationship with science is vital to address our biggest challenges – from COVID-19 to climate change. It's an honor to be part of the fast-growing, award-winning Leaps.org platform to help science and society thrive together," said GOOD Worldwide Co- Founder and CEO Max Schorr.
As part of the transition, Leaps.org recently launched a new monthly podcast series, "Making Sense of Science," with the first episode featuring NYU medical bioethicist Dr. Arthur Caplan. On March 11, Leaps.org co-hosted "COVID Vaccines and the Return to Life: Part 1" with the Aspen Institute Science & Society Program and the Sabin–Aspen Vaccine Science & Policy Group, the first of a four-part series that will run throughout 2021. Leaps.org's regular publication schedule features original reporting and commentary from highly sought-after journalists, scientists, academics, and thought leaders. The platform has already achieved significant success in making science compelling to a large audience, achieving close to 6 million page views and 6.2 million engagements on social media in 2020 alone.
Leaps.org is a not-for-profit program within the Good Worldwide ecosystem, which also includes Upworthy — a media platform that reaches over 150 million people monthly — whose mission is to share the best of humanity and inspire others to do the same.
Leaps.org publishes award-winning journalism, popularizes scientific progress on social media, and hosts forums about innovation, ethics, and the future of humanity. Leaps.org's projects and activities are supported by a consortium of like-minded partners including the Aspen Institute Science & Society Program, and supporters Leaps by Bayer, the Rita Allen Foundation, the Gordon and Betty Moore Foundation and the Howard Hughes Medical Institute.
Follow Leaps.org @makingsenseofscience on Instagram, @leaps_org on Twitter, and @leaps.org on Facebook and LinkedIn.
In November 2020, messenger RNA catapulted into the public consciousness when the first COVID-19 vaccines were authorized for emergency use. Around the same time, an equally groundbreaking yet relatively unheralded application of mRNA technology was taking place at a London hospital.
Over the past two decades, there's been increasing interest in harnessing mRNA — molecules present in all of our cells that act like digital tape recorders, copying instructions from DNA in the cell nucleus and carrying them to the protein-making structures — to create a whole new class of therapeutics.
Scientists realized that artificial mRNA, designed in the lab, could be used to instruct our cells to produce certain antibodies, turning our bodies into vaccine-making factories, or to recognize and attack tumors. More recently, researchers recognized that mRNA could also be used to make another groundbreaking technology far more accessible to more patients: gene editing. The gene-editing tool CRISPR has generated plenty of hype for its potential to cure inherited diseases. But delivering CRISPR to the body is complicated and costly.
"Most gene editing involves taking cells out of the patient, treating them and then giving them back, which is an extremely expensive process," explains Drew Weissman, professor of medicine at the University of Pennsylvania, who was involved in developing the mRNA technology behind the COVID-19 vaccines.
But last November, a Massachusetts-based biotech company called Intellia Therapeutics showed it was possible to use mRNA to make the CRISPR system inside the body, eliminating the need to extract cells out of the body and edit them in a lab. Just as mRNA can instruct our cells to produce antibodies against a viral infection, it can also teach them to produce the two molecular components that make up CRISPR — a guide molecule and a cutting protein — to snip out a problem gene.
"The pandemic has really shown that not only are mRNA approaches viable, they could in certain circumstances be vastly superior to more traditional technologies."
In Intellia's London-based clinical trial, the company applied this for the first time in a patient with a rare inherited liver disease known as hereditary transthyretin amyloidosis with polyneuropathy. The disease causes a toxic protein to build up in a person's organs and is typically fatal. In a company press release, Intellia's president and CEO John Leonard swiftly declared that its mRNA-based CRISPR therapy could usher in a "new era of potential genome editing cures."
Weissman predicts that turning CRISPR into an affordable therapy will become the next major frontier for mRNA over the coming decade. His lab is currently working on an mRNA-based CRISPR treatment for sickle cell disease. More than 300,000 babies are born with sickle cell every year, mainly in lower income nations.
"There is a FDA-approved cure, but it involves taking the bone marrow out of the person, and then giving it back which is prohibitively expensive," he says. It also requires a patient to have a matched bone marrow done. "We give an intravenous injection of mRNA lipid nanoparticles that target CRISPR to the bone marrow stem cells in the patient, which is easy, and much less expensive."
Meanwhile, the overwhelming success of the COVID-19 vaccines has focused attention on other ways of using mRNA to bolster the immune system against threats ranging from other infectious diseases to cancer.
The practicality of mRNA vaccines – relatively small quantities are required to induce an antibody response – coupled with their adaptable design, mean companies like Moderna are now targeting pathogens like Zika, chikungunya and cytomegalovirus, or CMV, which previously considered commercially unviable for vaccine developers. This is because outbreaks have been relatively sporadic, and these viruses mainly affect people in low-income nations who can't afford to pay premium prices for a vaccine. But mRNA technology means that jabs could be produced on a flexible basis, when required, at relatively low cost.
Other scientists suggest that mRNA could even provide a means of developing a universal influenza vaccine, a goal that's long been the Holy Grail for vaccinologists around the world.
"The mRNA technology allows you to pick out bits of the virus that you want to induce immunity to," says Michael Mulqueen, vice president of business development at eTheRNA, a Belgium-based biotech that's developing mRNA-based vaccines for malaria and HIV, as well as various forms of cancer. "This means you can get the immune system primed to the bits of the virus that don't vary so much between strains. So you could actually have a single vaccine that protects against a whole raft of different variants of the same virus, offering more universal coverage."
Before mRNA became synonymous with vaccines, its biggest potential was for cancer treatments. BioNTech, the German biotech company that collaborated with Pfizer to develop the first authorized COVID-19 vaccine, was initially founded to utilize mRNA for personalized cancer treatments, and the company remains interested in cancers ranging from melanoma to breast cancer.
One of the major hurdles in treating cancer has been the fact that tumors can look very different from one person to the next. It's why conventional approaches, such as chemotherapy or radiation, don't work for every patient. But weaponizing mRNA against cancer primes the immune cells with the tumor's specific genetic sequence, training the patient's body to attack their own unique type of cancer.
"It means you're able to think about personalizing cancer treatments down to specific subgroups of patients," says Mulqueen. "For example, eTheRNA are developing a renal cell carcinoma treatment which will be targeted at around 20% of these patients, who have specific tumor types. We're hoping to take that to human trials next year, but the challenge is trying to identify the right patients for the treatment at an early stage."
Repairing Damaged mRNA
While hopes are high that mRNA could usher in new cancer treatments and make CRISPR more accessible, a growing number of companies are also exploring an alternative to gene editing, known as RNA editing.
In genetic disorders, the mRNA in certain cells is impaired due to a rogue gene defect, and so the body ceases to produce a particular vital protein. Instead of permanently deleting the problem gene with CRISPR, the idea behind RNA editing is to inject small pieces of synthetic mRNA to repair the existing mRNA. Scientists think this approach will allow normal protein production to resume.
Over the past few years, this approach has gathered momentum, as some researchers have recognized that it holds certain key advantages over CRISPR. Companies from Belgium to Japan are now looking at RNA editing to treat all kinds of disorders, from Huntingdon's disease, to amyotrophic lateral sclerosis, or ALS, and certain types of cancer.
"With RNA editing, you don't need to make any changes to the DNA," explains Daniel de Boer, CEO of Dutch biotech ProQR, which is looking to treat rare genetic disorders that cause blindness. "Changes to the DNA are permanent, so if something goes wrong, that may not be desirable. With RNA editing, it's a temporary change, so we dose patients with our drugs once or twice a year."
Last month, ProQR reported a landmark case study, in which a patient with a rare form of blindness called Leber congenital amaurosis, which affects the retina at the back of the eye, recovered vision after three months of treatment.
"We have seen that this RNA therapy restores vision in people that were completely blind for a year or so," says de Boer. "They were able to see again, to read again. We think there are a large number of other genetic diseases we could go after with this technology. There are thousands of different mutations that can lead to blindness, and we think this technology can target approximately 25% of them."
Ultimately, there's likely to be a role for both RNA editing and CRISPR, depending on the disease. "I think CRISPR is ideally suited for illnesses where you would like to permanently correct a genetic defect," says Joshua Rosenthal of the Marine Biology Laboratory in Chicago. "Whereas RNA editing could be used to treat things like pain, where you might want to reset a neural circuit temporarily over a shorter period of time."
Much of this research has been accelerated by the COVID-19 pandemic, which has played a major role in bringing mRNA to the forefront of people's minds as a therapeutic.
"The pandemic has really shown that not only are mRNA approaches viable, they could in certain circumstances be vastly superior to more traditional technologies," says Mulqueen. "In the future, I would not be surprised if many of the top pharma products are mRNA derived."
"Making Sense of Science" is a monthly podcast that features interviews with leading medical and scientific experts about the latest developments and the big ethical and societal questions they raise. This episode is hosted by science and biotech journalist Emily Mullin, summer editor of the award-winning science outlet Leaps.org.