NEW YORK & SEATTLE--(BUSINESS WIRE)--Emendo Biotherapeutics, a next-generation CRISPR biotech expanding the reach of gene editing therapeutics, and Seattle Childrens Research Institute today announced a research collaboration to investigate how hematopoietic stem cells (HSCs) extracted from patients with severe congenital neutropenia (SCN) respond to priming treatments ahead of administering a CRISPR-based therapeutic.

ELANE-related SCN, also known as SCN1, is a rare, autosomal dominant disease in which a mutation occurs in one allele of the ELANE gene, thereby preventing HSCs from differentiating into white blood cells, specifically neutrophils, which leaves the patient highly susceptible to recurrent bacterial infections, osteoporosis, developmental delays and abnormalities.

Patients with SCN often suffer from reduced quality of life due to the lack of improvements in the standard of care, said Dr. David Rawlings, Division Chief of Immunology at Seattle Children's Hospital and Director of the Center for Immunity and Immunotherapies at Seattle Childrens Research Institute. These children are immunocompromised, and, as a result, we feel a great sense of urgency to ensure were exploring all possible avenues towards a solution.

Seattle Childrens collaboration with Emendo, utilizing its unique approach to edit only the mutated allele with CRISPR, will enable us to address the unmet needs of SCN at the very core, added Rawlings, who also serves as a professor of pediatrics and adjunct professor in the Department of Immunology at the University of Washington School of Medicine. Were excited about this opportunity, and look forward to continuing the collaboration beyond this initial study.

Editing the mutated ELANE gene with CRISPR first requires overcoming a technological hurdle: Only the mutated allele must be targeted, while the healthy allele remains intact. Emendo engineered its roster of next-generation CRISPR nucleases to be biologically active and so specific that they can differentiate between two alleles of the same gene. EMD-101, Emendos lead therapeutic candidate for SCN, was specifically engineered to target the mutant ELANE allele.

HSCs have been widely studied as a treatment for sickle cell anemia and cancer, as well as a potential therapy to treat organ and tissue damage. However, HSCs require initial priming prior to stem cell transplantation, which is typically done by administering G-CSF (granulocyte colony stimulating factor). Yet, the same drug is also a short-term treatment for SCN patients.

To better understand how SCN patients would respond to a priming dose of G-CSF and plerixafor, Emendo will evaluate the mobilization of HSCs excised from a small group of patients with SCN, which would be gene-edited later. Concurrently, Seattle Childrens will evaluate the composition of the HSCs obtained from the same patients. Prior mouse studies conducted by Emendo have shown that human cells edited to excise the disease-causing ELANE allele sufficiently engrafted and replaced existing diseased cells, restoring proper neutrophil differentiation.

By combining our allele-specific genome editing technology with Seattle Childrens renowned expertise in SCN spearheaded by Dr. Rawlings we are laying the foundation for future clinical trials that could lead to potential therapies to treat the disease, said David Baram, Ph.D., CEO of Emendo. Our portfolio of engineered nucleases tailored to any gene or allele gives us the unique opportunity to tackle the inherent challenges of SCN. Through this collaboration well be able to provide stronger evidence and further proof points for the capabilities of our technology.

Based on the outcome of the research, a protocol for a clinical trial could be developed with an expected initiation in late 2022, pending regulatory approval. Seattle Children's has certain preferred rights to serve as a clinical trial site.

About Emendo Biotherapeutics

EmendoBio is a next generation CRISPR gene editing company leveraging dual proprietary technology platforms to enable high precision gene editing throughout the genome. EmendoBios novel nuclease discovery platform broadens the targetable range of the genome while its target-specific optimization platform enables highly precise editing, including allele specific editing, while maintaining high efficiencies. The capabilities of the OMNI technology platforms, along with deep expertise in genomic medicine, protein engineering and therapeutic development, provide EmendoBio with a unique advantage when addressing indications within hematology, oncology, ophthalmology and other disease areas. For more information please visit us at http://www.emendobio.com.

About Seattle Childrens

Seattle Childrens mission is to provide hope, care and cures to help every child live the healthiest and most fulfilling life possible. Together, Seattle Childrens Hospital, Research Institute and Foundation deliver superior patient care, identify new discoveries and treatments through pediatric research, and raise funds to create better futures for patients.

Ranked as one of the top childrens hospitals in the country by U.S. News & World Report, Seattle Childrens serves as the pediatric and adolescent academic medical center for Washington, Alaska, Montana and Idaho the largest region of any childrens hospital in the country. As one of the nations top five pediatric research centers, Seattle Childrens Research Institute is internationally recognized for its work in neurosciences, immunology, cancer, infectious disease, injury prevention and much more. Seattle Childrens Foundation works with the Seattle Childrens Guild Association, the largest all-volunteer fundraising network for any hospital in the country, to gather community support and raise funds for uncompensated care and research. Join Seattle Childrens bold initiative It Starts With Yes: The Campaign for Seattle Childrens to transform childrens health for generations to come.

For more information, visit seattlechildrens.org or follow us on Twitter, Facebook, Instagram or on our On the Pulse blog.

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Emendo Biotherapeutics and Seattle Children's Research Institute Announce Collaboration to Develop CRISPR-based Therapeutic Strategy for Severe...

As 2021 draws to a close, COVID-19 remains a pervasive influence over life at Yale and across the world. And yet, even as a new surge in cases portends a winter of uncertainty, a look back at the past year offers many reminders of just how much more we now know about this global threat, the remarkable importance of vaccines and other public health measures, and the resiliency of humankind.

After reviewing the many hundreds of stories published on Yale News this year, we identified several about Yales response to the pandemic that especially resonated with readers and that best capture how the university and our experts have helped make sense of and respond to this disruptive disease. Youll find a list below.

In a second list below, we highlight several non-COVID stories about the people and projects that inspired us and gave us hope for a healthier and more equitable 2022 and the new initiatives that will position the Yale community to be a leader in tackling the challenges of the future.

As the new year began, Yale News reviewed how the campus community pulled together to do the work of the university in the face of unprecedented challenges, and looked ahead to the spring semester.

As chair of the White Houses COVID-19 Equity Task Force, Dr. Marcella Nunez-Smith, the C.N.H Long Professor of Internal Medicine, Public Health, and Management at Yale, became a national voice on racial inequities in COVID-19 treatment and outcomes. Meanwhile, Abbe Gluck, the Alfred M. Rankin Professor of Law and professor of internal medicine at Yale School of Medicine, was named special counsel to the Biden administrations COVID-19 Response team. She also worked in the Office of White House Counsel on other health care issues, including the Affordable Care Act..

In early January, Yale launched its COVID-19 vaccination program in the Lanman Center at Payne Whitney Gymnasium, as vaccines from Moderna and Pfizer gained final approvals for use in the United States.

After spending nearly a year cataloguing and exploring the SARS-CoV-2 genomes intricate makeup, a team of Yale scientists revealed a map of it with an unprecedented level of detail, including more than 100 identifiable structures.

In February, Yale scientists developed a new class of antiviral agents that showed promise for creating COVID-19 therapeutics exhibiting particular effectiveness when used in tandem with the drug remdesivir, another antiviral medication approved for use against the virus.

For most children, COVID-19 infection results in a relatively mild illness. In a few cases, however, a severe immune reaction occurs. During the spring, Yale research found that such rare, life-threatening reactions may be triggered by high levels of alarmins, molecules that make up part of the innate immune system.

The Lanman Center, which early in the pandemic was converted into a field hospital, and later into Yales primary vaccination center, returned to being simply a gym during the summer, as the vaccination operations were shifted to the Rose Center on Ashmun Street.

In July, a Yale-led study found that the COVID-19 vaccination campaign launched in the United States in late 2020 had, at that point, saved some 279,000 lives and prevented 1.25 million hospitalizations. Researchers warned, however, that these gains could be reversed by the highly transmissible Delta variant.

In September, Yale researchers provided important insights into what were then becoming known as breakthrough COVID-19 cases in which fully vaccinated individuals are infected by SARS-CoV-2 and which populations are particularly vulnerable to serious breakthrough illness.

Since the start of the COVID-19 pandemic, scientists had been unclear about how long immunity lasts after an unvaccinated person is infected. In October, a Yale-led team of researchers found an answer: Strong protection following natural infection is short-lived, lasting as little as three months or less.

In October, a Yale-led study found that two of the commonly used COVID-19 vaccines provide protection against multiple variants of the virus that causes the disease, including the highly infectious Delta variant. Their findings also showed that those infected with the virus prior to vaccination exhibit a more robust immune response to all variants than those who were uninfected and fully vaccinated.

In November, a study by Yale political scientists and public health experts found that, when it comes to persuading people to get vaccinated against COVID-19, its more effective to appeal to community spirit than to self-interest.

Breakthrough SARS-CoV-2 infections tend to be mild, but Yale research published in December showed that more older adults have developed severe breakthrough cases during the Delta variant phase of the pandemic, particularly after a longer period of time had elapsed since their last vaccination. The findings, researchers say, reveal the importance of booster vaccinations.

White evangelical Christians have resisted getting vaccinated against COVID-19 at higher rates than other religious groups in the U.S. In November, a Yale study found that persuading these vaccine holdouts had only become more difficult.

In December, as a new COVID-19 variant, Omicron, began to spread throughout the world, public health leaders scrambled to better understand how contagious the new variant is and whether existing vaccines are effective against it. Yale doctors offered insights into the emerging threat.

In February, Yale announced the establishment of the Wu Tsai Institute, an ambitious new research enterprise that will supercharge Yales neuroscience initiative and position the university to reveal the brain in its full, dynamic complexity, thanks to a historic gift from Joseph C. Tsai 86, 90 J.D. and his wife, Clara Wu Tsai.

As a historic renovation of the Peabody Museum proceeds, conservator Mariana Di Giacomo is charged with keeping a close eye on the iconic mural The Age of Reptiles, by celebrated artist Rudolph Zallinger. The experience has allowed her to appreciate layers of detail. In February, Yale News caught up with her and shared a dazzling gallery of images.

In a promising early trial, researchers from Yale reported in February that patients with spinal cord injuries experienced substantial improvements in motor function such as the ability to walk or to use their hands after an intravenous injection of bone marrow-derived stem cells.

After 30 months of renovations, the redesigned Humanities Quadrangle formerly the Hall of Graduate Studies put a vibrant new face on Yales longstanding excellence in the humanities. The refurbished building includes dynamic spaces that promote connections among departments and programs and the cultivation of new ideas.

The late Jeremy Ayers once known as the gender-bending performance artist Silva Thin may seem like an unlikely namesake for an ant. But thanks to Yale ecologist Douglas B. Booher and rock star Michael Stipe, who shared a decades-long friendship with Ayers, a new species from the forests of Ecuador will honor his legacy and his reverence for the diversity of life.

During the summer, the university announced that present and future students at Yale Universitys drama school will no longer pay tuition, thanks to a landmark $150 million gift from entertainment executive and philanthropist David Geffen.

Psilocybin, a psychedelic drug found in some mushrooms, has long been studied as a potential treatment for depression. Yale research published in July detailed exactly what happens in the brain after a dose of psilocybin, and what makes its medicinal properties so promising.

In August, Yale scientists published a study of atmospheric patterns on Mars and Saturns moon Titan that will help lay the foundation for more accurate forecasts of weather on other worlds. Researchers say such forecasts will be vital to the safety and success of future research missions.

In 1965, Yale scholars created a sensation with the revelation of the Vinland Map, which was thought to be the earliest known European depiction of the New World. This summer, a team of Yale researchers said it proved the map to be an elaborate 20th-century forgery.

In October, a series of performances by the Yale Glee Club, Yale Bands, and the Yale Symphony Orchestra held in each of Yales residential college courtyards marked a return to live music on campus following a year of lockdowns and a response to the Black Lives Matter protests of 2020. (With video.)

In November, Yale and the City of New Haven reconfirmed their historic, three-century partnership for a new generation, announcing a six-year commitment that increases the universitys annual voluntary financial contribution to the city and creates bold opportunities for inclusive economic growth that benefit the entire community.

Tony Reno, now in his ninth season as head coach of the Yale football team, is more focused on creating a culture of responsibility, camaraderie, and integrity than on wins and losses but that hasnt kept the Bulldogs from finding success on the field.

On the long road to Yale College, Obed Gyedu-Larbi labored as a domestic aide and Greyhound baggage handler. He also founded a non-profit to feed and clothe homeless people in New York City. For me, he said, it was important to not only work hard for myself.

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2021: The year in review | YaleNews - Yale News

Sometimes youve got to look for the trees in the forest. The good news of 2021 was like a host of saplings little trees lost in the forest of inflation, the pandemic, and catastrophic weather events. Look closer and you will find numerous reasons to cheer, scientific discoveries and advances that give honest-to-goodness hope for humanity.

Most notably, 2021 saw one of the most-effective vaccines ever created, in record time. But thats just the beginning. We witnessed other monster breakthroughs in biology, astronomy, medicine, engineering, computing, genomics, and many more scientific fields.

With so many astounding advances in 2021, it was tough to pick the most significant but we tried anyway. Here are our favorite 15 moments worth telling the kids about. Prepare for your mind to be blown.

The development, testing, and rollout of COVID vaccines has been called the moonshot of our generation. That might be an understatement. Thanks to devoted medical researchers and tens of thousands of everyday Americans who participated in clinical trials, the Food and Drug Administration (FDA) granted the Pfizer and Moderna COVID vaccines emergency use authorization for adults last December, followed by Johnson & Johnsons single-shot vaccine this February. Since then, the vaccine has become available for children as young as 5. Thats a vaccine rollout available to 94 percent of the population (under 5 are excepted so far) in little over a year. Previously, the fastest vaccine to go from development to deployment was the mumps vaccine in the 1960s which took about four years. Although were still struggling with COVID variants and breakthrough cases, this feat of inoculation has saved countless lives and holds promise for a future where we can keep up with viral outbreaks in real time.

Lots of animals can regrow a torn-off tail or a leg lost to a predator, but sea slugs have the coolest regeneration trick by a long shot. As a Japanese scientist discovered this year, these slimy creatures can behead themselves on purpose and grow a whole new body within weeks. The severed head survives on its own while it regenerates vital organs and limbs, likely due to slugs plant-like ability to photosynthesize because of all the algae they eat. Even more impressive, the discarded body lives for weeks before eventually dying off. Researchers think sea slugs use this cool maneuver to hoodwink predators and escape unharmed, or possibly to survive parasite infestations of their lower body.

Brain-computer interfaces (BCIs) hold major promise for people with paralysis, allowing them to operate robotic limbs, wheelchairs, keypads, and other gadgets just by thinking about moving their bodies. But so far, BCIs have mostly been relegated to research settings, as theyve required bulky cables to connect a persons head to a computer to an external device.

Not anymore. The prestigious BrainGate research team has devised the worlds first high-bandwidth, totally wireless BCI that transmits brain signals as quickly and clearly as cabled systems do. In a recent clinical trial, the new device enabled two people with tetraplegia to point, click, and type on a tablet with precision and speed no wires required. More research is needed, but this is a major step toward taking BCIs out of the lab and into the real world to help people with paralysis regain independence.

Americans boundless fascination with unidentified flying objects was finally indulged in 2021. In January, by way of the Freedom of Information Act, The Black Vault website posted the CIAs recently declassified database of every UFO sighting reported by a military pilot, dating back to the 1980s. Concurrently, the CIA uploaded dozens of records of UFO sightings from the 1940s to the early 1990s.

Then, in June, the Pentagon issued a long-awaited nine-page report summarizing everything it claims to know about unidentified aerial phenomena, or UAPs, its fancy term for UFOs. Shocker: The government doesnt know much. The report does assert that UAPs are not U.S. military craft, but otherwise, it pretty much plays the inconclusive card. But hey, although the dossier may not clear up many mysteries, the massive data dump should keep UFO-obsessed armchair detectives captivated for years to come.

About 90 percent of human brain development happens by age five. And although neuroscientists have recently learned a lot about how and when various developments occur, especially in utero, theres still a ton they dont know, particularly about the impacts of nature versus nurture. These answers are now coming, courtesy of the largest, most comprehensive trial on early brain development ever, which kicked off this fall.

Through the HEALthy Brain and Child Development Study, researchers nationwide will track a diverse group of 7,500 pregnant people and their children throughout the next decade. Using neuroimaging and psychological assessments, they aim to map out the normal arc of brain development and discover how pre- and postnatal environments and exposures (stress, socioeconomic status, parents drug use, COVID, etc.) affect it as well as how kids brains adapt. This historic study has the potential to unlock prevailing mysteries about autism, dyslexia, and other childhood neurodevelopment, emotional, and behavioral concerns.

Long before COVID, malaria was and as of time of publication, still is one of the most lethal infectious diseases on the planet. This mosquito-borne pathogen kills half a million people annually, predominantly in sub-Saharan Africa. Over half of those malaria kills are children under age 5, Now, after a century of effort, scientists have finally developed a safe, effective malaria vaccine (the first vaccine for any parasitic disease, by the way), which the World Health Organization (WHO) greenlighted for all at-risk kids in October. Assuming nations prioritize vaccine distribution, experts estimate this breakthrough could prevent 5.3 million malaria cases and 24,000 deaths among children under 5 every year.

An estimated 1.6 million Americans live with type 1 diabetes (aka juvenile diabetes), including 200,000 kids and adults under age 20. With no known cure, this life-threatening autoimmune disease, in which the pancreas stops producing insulin to control blood sugar, almost always requires intense 24/7 management.

That may be about to change. To the shock and elation of diabetes experts, an experimental treatment delivered in an ongoing clinical trial appeared to cure a 64-year-old man of type 1 diabetes, which would be a world first. After receiving infusions of insulin-producing cells grown from stem cells, the mans body now makes insulin on its own, giving him a whole new life, as he told the New York Times.

Because this discovery is part of a five-year study involving 16 other participants, its still too soon to say with certainty whether the treatment is effective and safe long-term. But its the most promising development the world has seen in regard to a type 1 diabetes cure and likely enough to make a parent or child living with type 1 do cartwheels.

In February, almost seven months after launching from Earth, NASAs highly sophisticated Perseverance rover touched down on Mars. The vehicle will spend nearly two years on the red planet, surveying the landscape, searching for evidence of past Martian life, and collecting geological samples to bring back to Earth. Then, in April, NASAs solar-powered Ingenuity Mars Helicopter became the first-ever aircraft to make acontrolled flight on another planet. By December 8, Ingenuity had logged 17 successful flights.

Mars wasnt the only celestial body to make news in 2021. In November, NASA scientists validated the existence of 301 new exoplanets planets that orbit stars other than the Sun bringing the total exoplanet tally to 4,870. The validation frenzy comes courtesy of NASAs new ExoMiner deep-learning technology, which evaluates data collected by the Kepler spacecraft to distinguish legit exoplanets from convincing fakes.

One of the biggest differences between the COVID pandemic and that of the 1918 Spanish Flu (the last global pandemic) is the way that we track it. Its nearly unimaginable that we once had to follow death and infection rates by local tally and had essentially no way of knowing about new viral variants. Now, led by the WHO, scientists have a colossal global collaboration to monitor the spread and evolution of SARS-CoV-2. Huge amounts of data have been collected and shared across borders in real time, allowing researchers to get quickly gain an idea of how a variant like Delta or Omicron spreads and affects case numbers and hospitalizations. We didnt have this sort of technology available at the beginning of the pandemic. As of April 2021, the online GISAID database containedonly one million SARS-CoV-2 genomic sequences. Eight months later, another five million sequences have been added. In other words, genomic sequencing of SARS-CoV-2 has gotten ten times faster since the spring. This accomplishment highlights that one of the biggest challenges of science isnt discovery, but sharing discoveries, and countries across the world are now doing that in a way theyve never done before.

With the average cost of a solar panel plummeting 90 percent between 2010 and 2020, it keeps getting cheaper and cheaper to generate power directly from the sun. Thats great news, as it helps shift our reliance away from fossil fuels, a key contributor to climate change. Frustratingly, however, solar panels havent gotten much more efficient in recent years, which has hindered widespread adoption of this form of clean energy.

To solve this issue, engineers have been looking for alternative materials that can outperform the standard silicon used in solar panels and still be inexpensive. Theyve had high hopes for perovskites, atomically thin, latticed materials that convert sunlight into energy highly efficiently. The only problem? Ultraviolet rays and moisture destroy perovskites in no time, tanking their usefulness.

But this year, Rice University engineers developed and road-tested solar cells made of two-dimensional perovskites. Their invention works much better than earlier models and withstands the elements. The trick with 2D perovskites, the researchers discovered, is that sunlight contracts the spaces between the atomic layers to boost efficiency by up to 18 percent a huge leap forward in this field. With solar companies worldwide working to commercialize perovskite solar cells, this breakthrough should ultimately accelerate societys conversion to solar energy.

In January, Jean-Michel Dubernard, MD, the same surgeon who performed the first-ever hand, double hand, and partial face transplants, accomplished yet another historic feat: the worlds first double arm and shoulder transplant. The operation, performed in France, was a resounding success. The recipient, 49-year-old Felix Gretarsson of Iceland, whod lost both arms in an electrical accident in 1998, has steadily gained mobility throughout the year, charting his progress on Instagram. He can now flex his biceps, pick up objects, and hug his granddaughter. Experts expect he will make more advancements in the coming years. Sadly, Dubernard died in July, but not before giving Gretarssinan entirely new life.

This year, scientists learned a lot about the massive creatures that inhabited the Earth many millions of years ago. First up, dinosaurs. The fearsome predator Tyrannosaurus rex roamed North America starting nearly 70 million years back, and now biologists have finally estimated how many: 2.5 billion. Terrifying, right? If its any comfort, thats the total T. rex population spread out over 2.4 million years. So, really, there were only about 20,000 adult T. rexes living at one time.

Of course, that last generation of T. rex, along with the entire dinosaur kingdom, got wiped off the planet some 66 million years ago by an asteroid. Or wait, was it a comet? Thats the new theory put forth by Harvard astronomers to explain the so-called Chicxulub Impactor, the astronomical body that created a 93-mile-wide, 12-mile-deep crater off the coast of Mexico and, theoretically, killed the dinosaurs. Countering the prevailing asteroid theory, the Harvard astronomers think a comet from the fringes of our solar system got knocked off-orbit by Jupiters gravitational field and broke into chunks. Then an especially large chunk the eventual Chicxulub Impactor slammed into the Earth, wreaking major havoc and wiping out the dinos.

More than 60 million years after the dinosaurs, mammoths were living large, which researchers know because of the extensive fossil record. This year, such fossils yielded an unprecedented discovery: the oldest ancient animal genome ever recovered. In sequencing DNA from three mammoth teeth extracted from the Siberian permafrost, scientists determined the fossils were more than one million years old, obliterating the previous record held by a 560,000-to-780,000-year-old horse leg bone. The DNA also suggests a separate lineage, possibly a different species, of mammoth that scientists werent aware of before.

An international event thats been decades in the making is finally (hopefully!) happening on December 24*. After multiple delays, the James Webb Space Telescope the largest and most advanced scientific telescope in the history of space exploration is scheduled to blast off from French Guiana aboard the Ariane 5 rocket. It will take 30 days to travel nearly 1 million miles to a stable spot in space and another six months to unfold its instruments, align, and calibrate. As it tracks Earths orbit around the sun for the next several decades, the infrared scope will directly observe parts of the universe previously unseeable, thereby demystifying the origin and evolution of our planet, solar system, and galaxies beyond.

One of the biggest differences between the COVID pandemic and that of the 1918 Spanish Flu is the way that we track it. Its nearly unimaginable that we once had to follow death and infection rates by local tally and had essentially no way of knowing about new viral variants. Now, led by the WHO, scientists have a colossal global collaboration to monitor the spread and evolution of SARS-CoV-2. Huge amounts of data have been collected and shared across borders, allowing researchers to get quickly gain an idea of how a variant like Omicron spreads and affects case numbers and hospitalizations. We didnt have this sort of technology available at the beginning of the pandemic. As of April 2021, the online GISAID database contained only one million SARS-CoV-2 genomic sequences. Thats after about 16 months of pandemic. But in the eight months since, another five million sequences have been added. In other words, genomic sequencing of SARS-CoV-2 has gotten ten times faster since the spring. This accomplishment highlights that one of the biggest challenges of science isnt discovery, but sharing discoveries, and countries across the world are now doing that in a way theyve never done before.

What takes todays best supercomputers several days or weeks to process, quantum computers can knock out within seconds. Thats why quantum computing, which leverages the laws of quantum physics for unprecedented processing capabilities, is already considered among the biggest scientific breakthroughs of the 21st century. Eventually, its supposed to revolutionize manufacturing, meteorology, cybersecurity, national defense, and much more.

Well, 2021 made eventually closer than ever. In November, IBM unveiled its 127-qubit Eagle, the most powerful quantum processor yet. Then earlier this month, the company Quantinuum debuted the worlds first commercial product built from quantum computing: a cloud-based cybersecurity platform called Quantum Origin. With the worlds top tech companies and research institutions racing to advance this next-gen technology, expect quantum computing to make our list again next year, and the next, and the next

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15 Truly Unbelievable Ways Science Changed the World in 2021 - Fatherly

The space station is viewed from the SpaceX Cargo Dragon during its automated approach before docking. Credit: NASA TV

While the International Space Station was traveling more than 260 miles over the South Pacific Ocean, a SpaceX Dragon cargo spacecraft autonomously docked to the space-facing side of the orbiting laboratorys Harmony module at 3:41 a.m. EST, Wednesday, Dec. 22. NASA astronauts Raja Chari and Thomas Marshburn were monitoring docking operations for Dragon.

The Dragon launched on SpaceXs 24th contracted commercial resupply mission at 5:07 a.m. EST, Tuesday, Dec. 21 from Launch Complex 39A at NASAs Kennedy Space Center in Florida. After Dragon spends about one month attached to the space station, the spacecraft will return to Earth with cargo and research.

Among the science experiments Dragon is delivering to the space station are:

Bioprinting bandagesBioprinting uses viable cells and biological molecules to print tissue structures. The German Aerospace Center studyBioprint FirstAiddemonstrates a portable, handheld bioprinter that uses a patients own skin cells to create a tissue-forming patch to cover a wound and accelerate the healing process. On future missions to the Moon and Mars, bioprinting such customized patches could help address changes in wound healing that can occur in space and complicate treatment. Personalized healing patches also have potential benefits on Earth, providing safer and more flexible treatment anywhere needed.

Improving delivery of cancer drugsMonoclonal antibodies, used to treat a wide range of human diseases, do not dissolve easily in liquid and so typically must be given intravenously in a clinical setting.The Center for the Advancement of Science in Space Protein Crystal Growth 20 (CASIS PCG 20) experimentcontinues work on crystallizing a monoclonal antibody, pembrolizumab, that Merck Research Labs developed. It is the active ingredient in Keytruda, a drug that targets multiple cancers. Scientists analyze these crystals to learn more about the structure and behavior of the component to create drug formulations that can be administered at a doctors office or even at home.

Assessing infection riskScientists have observed that spaceflight sometimes increases the virulence of potentially harmful microbes and reduces human immune function, increasing the risk for infectious disease.Host-Pathogenassesses space-induced changes in immune status by culturing cells collected from crew members before, during, and after spaceflight with both normal bacteria and bacteria grown under simulated spaceflight conditions. Results could help assess the potential risk infectious microbes may pose and may support development of countermeasures. This could improve care for those with compromised immune systems on Earth.

Roots, shoots, and leavesMulti Variable Platform (MVP) Plant-01profiles and monitors the development of the shoots and roots of plants in microgravity. Plants could serve as a vital part of human life support systems for long-duration spaceflight and habitation of the Moon and Mars. However, space-grown plants experience stress from various factors and recent studies indicate changes in plant gene expression in response to those stressors. Improved understanding of these changes could enable the design of plants that are better suited for growth in spaceflight environments.

Toward lunar laundromatsAstronauts on the space station wear items of clothing several times, then replace them with new clothes delivered on resupply missions. Limited cargo capacity makes this a challenge, and resupply is not an option for longer missions, such as those to the Moon and Mars. In a collaboration with NASA, Procter & Gamble has developed Tide Infinity, a fully degradable detergent specifically designed for use in space, and theP&G Telescience Investigation of Detergent Experiments (PGTIDE) study the performance of its stain removal ingredients and the formulations stability in microgravity. Once proven in space, Tide plans to use the new cleaning methods and detergent to advance sustainable, low-resource-use laundry solutions on Earth.

Parts made in spaceTurbine Superalloy Casting Module (SCM)tests a commercial manufacturing device that processes heat-resistant alloy parts in microgravity. Alloys are materials made up of at least two different chemical elements, one of which is a metal. Researchers expect more uniform microstructures and improved mechanical properties in superalloy parts processed in microgravity compared to those processed on Earth. These superior materials could improve the performance of turbine engines in industries such as aerospace and power generation on Earth.

Students and citizens as space scientistsStudents enrolled in institutions of higher learning can design and build microgravity experiments as part of NASAs Student Payload Opportunity with Citizen Science (SPOCS). As part of their experiments, selected teams include students in kindergarten through 12th grade as citizen scientists. Citizen science allows individuals who are not professional scientists to contribute to real-world research. TheNASA STEM on Stationproject is funding experiments flying on this SpaceX resupply mission, including a study on antibiotic resistance in microgravity from Columbia University in New York and one on how microgravity affects bacteria-resistant polymers from the University of Idaho in Moscow, Idaho.

These are just a few of the hundreds of investigations currently being conducted aboard the orbiting laboratory in the areas of biology and biotechnology, physical sciences, and Earth and space science. Advances in these areas will help keep astronauts healthy during NASAsArtemismissions to the Moon and long-duration space travel and demonstrate technologies for future human and robotic exploration beyond low-Earth orbit to theMoon and Mars.

Learn more about station activities by following thespace station blog,@space_stationand@ISS_Researchon Twitter, as well as theISS FacebookandISS Instagramaccounts.

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Cargo Dragon Docks to Station with Brand New Science - NASA

Jorge E. Cortes, MD, has been an investigative leader for nearly 30 years in the development of numerous leukemia treatments.

It would be difficult to look at data involving practice-changing agents for patients with leukemia and miss the name Jorge E. Cortes, MD. An investigative leader for nearly 30 years, Cortes has led the development of numerous leukemia treatments, including trials for the second-generation tyrosine kinase inhibitor (TKI) bosutinib (Bosulif), which is widely used for chronic myeloid leukemia (CML); omacetaxine mepesuccinate (Synribo), a drug approved for patients with CML when TKIs have stopped working; the third-generation TKI ponatinib (Iclusig), another CML treatment; and glasdegib (Daurismo), a smoothened inhibitor approved for the treatment of older patients with acute myeloid leukemia (AML) and complications like heart or kidney disease that preclude use of intensive induction chemotherapy.

Today, Cortes is leading something even larger than drug trials: the Georgia Cancer Center at Augusta University, which named him its director in 2019, following a 20-plus year career at The University of Texas MD Anderson Cancer Center in Houston. Working to make Georgia Cancer Center a world-class facility and continuing with as much research as possible keeps Cortes busy, but he has still found time to cochair the 26th Annual International Congress on Hematologic Malignancies hosted by Physicians Education Resource (PER), LLC.

The hybrid interactive conference will be held from Thursday, February 24, 2022, to Sunday, February 27, 2022, at the Eden Roc in Miami Beach, Florida. The 4-day event will focus on leukemias, lymphoma, and myeloma. Its presentations and panels will cover the latest developments in chimeric antigen receptor (CAR) T-cell therapy, the most pivotal new trial results, the use of genomics and molecular testing in hematological cancers, and how to cope with the emerging value basedcare landscape.

At MD Andersonwhere he rose from a fellow to the deputy chair of the Department of Leukemia Cortes established himself as one the worlds leading leukemia researchers and the coauthor of more than 1000 published papers. At Georgia Cancer Center, he has less time for research, but he has still managed to launch another multicenter trial of an experimental CML treatment.

[Cortes] is truly a world expert on all things leukemia, has peerless clinical experience, and is an undisputed leader in the field. He has been instrumental in a very large number of trials that have led to drug approvals, and he ranks among the most published authors in the scientific world, Alexander E. Perl, MD, MS, said. Perl is an associate professor of medicine at Perelman School of Medicine at the University of Pennsylvania and a member of the leukemia program in the Abramson Cancer Center in Philadelphia, who has worked with Cortes on trials of FLT3 inhibitors.

Hes an excellent speaker as well, Perl added, and will make a great chair for the conference.

Courtney D. DiNardo, MD, MSCE, a clinical researcher in the Department of Leukemia at MD Anderson Cancer Center, said the key to Cortes success is a level of drive that is unusual even in a world of highly driven people.

Hes always moving; hes always thinking. Hes kind of like the Energizer Bunny. He just keeps going and going, she said.

The agenda for the International Congress on Hematologic Malignancies features dozens of presentations and panels, and most of them are followed directly by question-and-answer sessions with thought leaders. The other program cochairs are Andre H. Goy, MD, physician in chief at Hackensack Meridian Health Oncology Care Transformation Service, chairman and chief physician officer at John Theurer Cancer Center, Lydia Pfund Chair for Lymphoma, Academic Chairman Oncology at Hackensack Meridian School of Medicine, and professor of medicine at Georgetown University in Hackensack, New Jersey, and Sagar Lonial, MD, FACP, chief officer of Winship Cancer Institute of Emory University in Atlanta, Georgia.

The key topics for discussion will include the following:

A MODERN LANDSCAPE

Cortes recently sat down for an in-depth interview with OncologyLive to preview the conference and share his thoughts about the major trends in leukemia treatment.

When I started, leukemia treatment was easy, Cortes said. [Individuals] with AML got 7 plus 3 [cytarabine continuously for 7 days and an anthracycline on each of the f irst 3 days of a treatment cycle]. Patients with CML would get interferon. And individuals with a model of proliferative neoplasms got hydroxyurea. And that was it. It was very easy. Unfortunately, the results were terrible.

Nowadays, theres a lot more complexity in our understanding of the biology. Theres not one AML, theres not one ALL [acute lymphoblastic leukemia]. There are a lot more challenges in classifying the cancer, and the same is true in treatment. We have a lot more treatment options, but the increase in treatment options means that its a lot harder to pick the right one. How do I select when I have 3 or 4 options? How do I combine them? What is the relative value? The answers to all these questions are evolving very rapidly because there [are] a lot of data coming out.

Among the biggest topics of conversation at the hematology conference will be recent trial results for CAR T-cell therapy. In October, the FDA approved brexucabtagene autoleucel (Tecartus) for adults with relapsed or refractory B-cell precursor ALL. The approval was based on results from the ZUMA-3 trial (NCT02614066), in which 71 patients were enrolled and underwent leukapheresis. The CAR T-cell therapy was then successfully manufactured for 65 of those patients and administered to 55. At the median follow-up of 16.4 months, 31 (56%) patients reached complete remission (CR). The median duration of remission was 12.8 months (95% CI, 8.7 months-not estimable [NE]). Median relapse-free survival was 11.6 months (95% CI, 2.7-15.5), and median overall survival (OS) was 18.2 months (95% CI, 15.9 months-NE). Among responders, median OS was not reached at the time of analysis.1

A few days before that approval, Kite submitted a supplemental biologics license application to the FDA to expand the current indication of the CAR T-cell therapy axicabtagene ciloleucel (Yescarta) to include the second-line treatment of adult patients with relapsed or refractory large B-cell lymphoma. The application was based on findings from the phase 3 ZUMA-7 trial (NCT03391466), which showed improved event-free survival compared with standard of care after a median follow-up of 2 years. Among the 359 patients who were randomized 1:1 to CAR T-cell therapy or standard of care, patients in the experimental group experienced a 60% reduction in events.2

We will present a lot of data on CAR T-cell therapy, Cortes said. This is a rapidly emerging field, with a large number of new trial results, not just in acute lymphoblastic leukemia but, increasingly, in other areas as well, and we dedicate a whole section of the conference to the understanding of CAR T-cells. This is something that was addressed last year, but we will do it again because new information keeps coming, and now theres the new indication in acute lymphoblastic leukemia.

Cortes said that studies with venetoclax (Venclexta) in AML will also be discussed. We will present [findings] from the initial phase 1 and phase 2 trials and then the randomized phase 3 studies that cemented venetoclax as the standard of care in a short period of time.

Results of the phase 3 Viale-A (NCT02993523) trial led to venetoclax being adopted as the standard treatment in older patients with previously untreated AML. The trial randomized 286 patients to receive azacitidine plus venetoclax and 145 patients to receive azacitidine plus placebo. At a median follow-up of 20.5 months, the median OS was 14.7 months in the azacitidine/venetoclax group vs 9.6 months in the control group (HR for death, 0.66; 95% CI, 0.52-0.85; P < .001). Participants were also more likely to experience CR (36.7% vs. 17.9%; P < .001) and composite CR (66.4% vs 28.3%; P < .001). Serious adverse events occurred in 83% of patients in the experimental arm vs 73% of patients in the control arm.3 These data, as well as data from the phase 3 VIALE-C trial (NCT03069352), supported the FDA decision in October 2020 to grant regular approval to venetoclax in combination with azacitidine, decitabine, or low-dose cytarabine for the treatment of adults 75 years and older with newly diagnosed AML.4,5

Another major focus of conversation will be research indicating that many patients with CML who have responded completely to TKI treatment and gone several years with no evidence of disease can safely discontinue treatment.

We have [an] increasing amount of trial data on this issue, Cortes said. We want to present the data from The LAST Study [NCT02269267] and elsewhere and put those trials in context and explore which patients are good candidates for treatment discontinuation and how we can do it right to minimize the risk for patients.

EXPANDING HORIZONS

Cortes credits his career in medicine to his uncle. When I was in high school, I wanted to be a dentist for some reason, but my uncle, whom I was very close with, asked me why I wanted to limit myself to treating the mouth when I was a good enough student to become a doctor and treat the whole body. Eventually he convinced me that medicine was probably a better path for me, he said.

The decision to specialize in leukemia treatment and research also happened more by chance than by design. Cortes attended medical school and did his residency in his native Mexico before arriving in Houston for a hematology fellowship at The University of Texas Health Science Center. A portion of his rotation was held across the street at MD Anderson Cancer Center, where he met the team working on leukemia. Impressed by the investigators in the laboratory and the work they were doing, Cortes switched his program to focus on leukemia. More than 1000 papers later, his focus remains unchanged.

Remember, were talking about almost 30 years ago, so in those days, [treating leukemia] was very, very challenging, Cortes said. There were very few new therapies available in leukemia, but there was a good opportunity to study because access to tissue is readily available. You also got the outcomes very quickly, so the clinical trials could be conducted rapidly.

Location was also important to fueling his research. [Houston] was a very active environment for research, Cortes said. There were lots of clinical trials, lots of academic discussions and interaction, so I thought it was a field that was very ripe for discoveries, and sure enough, a lot of new things have happened since then. Some of them, Ive been a part of, and some of them, Ive been a witness to, but its been a very rapid development.

Cortes interest in and experience with TKIs dates all the way back to the beginning. He was investigating CML with Moshe Talpaz, MD; Hagop M. Kantarjian, MD, a 2014 Giants of Cancer Care award winner in the leukemia category; and others at MD Anderson when the initial phase 1 trials of imatinib (Gleevec) began. He saw the incredible efficacy of the drug in those first patients and realized the great potential of targeted medications, specifically with TKIs.

The potential has been recognized in recent years in the expansion of targeted agents and a growing number of assays. Strategies for selecting the best therapies will be a major topic of conversation at the International Congress on Hematologic Malignancies.

We will have an outstanding presentation on the increasing complexity and the molecular diversity of acute lymphoblastic leukemia, which is a rapidly evolving area. Its become very complex, but also very specific, so this presentation will discuss how to use that information to manage patients, Cortes said. We will have the same sort of presentation for acute myeloid leukemia because again, its become a necessity to assess your patient to understand how to proceed with treatment.

There will also be information on these molecular abnormalities in individuals that do not have leukemia but do [have] predisposing factors, these CHIPsor clonal hematopoiesis of indeterminate potential. We have analyses of what these clonal entities mean, and we need to continue discussing them as we try to understand how they should affect our approach.

SHAPING THE NEXT GENERATION

You could say that Cortes enjoys being in the weeds of drug development, having a hand in the process from start to finish. He enjoys the complexity of running large drug trials, analyzing early-stage data to construct late-stage protocols, assembling research teams, and working with both drug companies and the FDA.

Drug development is a very complex endeavor, he said. Having a drug that works is obviously very important, but you have to design the trials in such a way that you get not only the academic answers and the clinical answers that you want, but also the data you need for regulatory approval. You also need to work with a lot of different groups investigators, sponsors, regulatory authorities, and most importantly, you have to work with patients. You need to recruit and enroll them.

Cortes noted that one of the key challenges is adapting opinions about drugs as new information becomes available and modifying trial design accordingly.

Even when the drugs look good initially, you also have to acknowledge that you know very little, and sometimes you learn things that that you didnt expect, he said, citing his experience with the agent ponatinib. It looked like a wonderful, very effective drug, but we learned that ponatinib had risk of arterial occlusive eventsheart attacks, strokes, and things like thatwhich was completely unexpected. The [challenge] was how to react to that. How do you balance the risk-benef it ratio? How do you [work] with the sponsor, the regulator agencies, and the patients?

Cortes strategy for managing these adverse effects secured ponatinib its 2012 FDA approvalalbeit with a black box warningfor the treatment of adults with CML and Philadelphia chromosomepositive ALL. Last year, the FDA expanded the indication. Both approvals were supported by data from the phase 2 PACE trial (NCT01207440)6; the second indication was also supported by data from the phase 2 OPTIC trial (NCT02467270).7

When we talk about what it takes to run a good trial, it all sounds straightforward, almost to the point of being obvious, but its not, DiNardo said. Doing good clinical research is a challenge, and some people are much better at it than others. I worked with Dr Cortes on several trials when I was new to the leukemia team at MD Anderson, and I am very happy I got a chance to learn from the best.

LOOKING BEYOND THE CURVE

Among the discussion of new trial results and new diagnostic tests, the International Congress on Hematologic Malignancies will also explore a relatively new concern: weighing the relative value of various potential treatments beyond their statistical significance.

Youre looking to maximize value for the patient, Cortes said. In a randomized trial, you [are looking to] get an improvement in survival that has a statistical value. But statistical significance may or may not mean something clinically. If [the survival benefit] is just a few weeks and the toxicity profile is harsh, how much of that extra time is spent in the hospital or suffering because of adverse effects? The survival benefit can be somewhat diluted by what kind of lifestyle you have. Youre alive, but are you living a normal life or at least close-to-normal life? And then, you know, how much are you paying for each week or month of extended survival? These are all things you need to consider, and were seeing more interest in thinking about how to balance them.

Cortes has taken a particular interest in improving quality of life for older patients and those with comorbidity that made traditional treatments hard to tolerate. Age alone doesnt make you less able to tolerate treatment, but it is more common that older patients will not be able to tolerate treatment, he said.

Cortes interest in investigating treatments for older patients helped inspire his work to develop glasdegib. The agent was approved in November 2018 in combination with low-dose cytarabine for patients with newly diagnosed AML who are 75 years or older with comorbidities that preclude intensive induction chemotherapy.8 That approval was supported by data from the BRIGHT AML 1003 trial (NCT01546038), in which 115 patients were randomized to receive low-dose cytarabine with or without glasdegib. After a median follow-up of 20 months, median OS was 8.3 months (95% CI, 4.4-12.2) in the investigative arm vs 4.3 months (95% CI, 1.9-5.7) in the control arm (HR, 0.46; 95% CI, 0.300.71; P = .0002).

[Older patients] have more comorbidities; they frequently take other medications, so you have to consider drug-drug interactions, Cortes said. There is also a tendency to give uppatients give up on themselves, doctors are more likely to give up on [finding treatments]and you need to avoid that. Life expectancy is much longer now than it was 30 years ago. For trial [design] purposes, we used to consider patients over 55 [years] as elderly. We wouldnt even [enroll them to] stem cell trials. Nowadays that sounds ridiculous. We realize that its just as important to combat cancer in these patients as it is in patients of any other age.

CARVING OUT A CORNER OF CARE

To relax and recharge, Cortes naturally enjoys something that keeps him moving full speed ahead: long-distance running. I love to run. I have done 8 marathons so far. Ive run them in Chicago, Boston, New York, and Houston. Ill be doing Houston again [in 2022], and hopefully that will qualify me to return to Boston, said Cortes, whose best marathon time is 3 hours and 30 minutes.

Why running? Part of the allure is the chance to get away from stuffy indoor spaces and spend long periods outside. He even enjoys bad-weather days because he likes making himself endure conditions that would send others to the treadmill.

The challenge is the point of long-distance running. Its demanding. The race is demanding. The training is demanding. But it gives you a feeling of accomplishment, he said. I also like that its an individual sport. If I have a bad day, Im not hurting any teammates. Running lets you run your race, at your own pace. You set your goals, and its fun to meet them. But if you dont, you just try again.

References

More:
Adaptation Is Key to Advancing Care for Adult Patients With Leukemia - OncLive