Category Archives: New York Stem Cells

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

Growing new pancreas cells from unprogrammed stem cells has possibly cured a man of type 1 diabetes.

One doctor told the New York Times that this is the biggest development in treatment for the disease since the discovery of exogenous insulin production 100 years ago.

64-year-old Brian Shelton got an infusion of insulin-producing cells in the pancreasthe kinds which cant function properly in diabetes victims, after his wife signed him up for a trial run by Vertex Pharmaceuticals.

Before, his life had been governed by the levels of his blood sugar. Now, his daily insulin requirements are down 91%, accompanied by robust improvements in glucose control.

Its a whole new life, its a miracle, Shelton told the Times. The trial was organized by a Harvard scientist who had two children join the 1.5 million Americans who suffer from type 1 diabetes.

This is the first of five years in which the trial of 17 patients with severe type 1 will be running. The results of the first stage have not been peer reviewed, and so expectedly, the scientists that are excited about the results are also urging caution because its still early days.

MORE: Patch Inspired by Cactus Eliminates Need for Diabetics to Prick Skin for Blood, Collects Sweat Instead

These results from the first patient treated with [the stem cells] are unprecedented, said Bastiano Sanna, Ph.D., Chief of Cell and Genetic Therapies at Vertex in a statement. What makes these results truly remarkable is that they were achieved with treatment at half the target dose.

Funded by the Howard Hughes Medical Institute, Harvards Dr. Doug Milton took 20 years to convert stem cells into islet cells, the insulin-producing pancreatic denizens.

RELATED: People Whove Tried Psychedelics Have Lower Risk of Heart Disease and Diabetes

In 2014 Milton partnered with Dr. Sanna at his previous job to start a company called Semma for the purpose of bringing to market a potential stem cell treatment, and along with other biologists, was able to demonstrate that for the first time, there was a repeatable, scalable method for growing islet cells and that they could cure diabetes in rodents.

Next, Milton and his partners and colleagues closed a $950 million sale of Semma to Vertex Pharma, who put up the money for the trials as they needed to see if injections of the manufactured islet cells could be done at scale, safely, and if the immunosuppressant drugs, typical of anyone receiving any kind of transplant, did not cause long-term adverse health outcomes.

READ: No More Pricks: Scientists Are Rolling Out First-of-its-Kind Blood Sugar Test for Pain-Free Delivery to Diabetics

The night the trial results came in, Mr. Shelton was taken to dinner by Dr. Milton, who revealed that Shelton was at least for the moment, cured of the disease.

The Times touchingly reports that at that moment Shelton checked his blood sugar levels, which were perfect, and then had dinner, after which they were still perfect.

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A New Stem-Cell Treatment Looks to Have Cured a Man of Type 1 Diabetes - Good News Network

AVENTURA, Fla. & NATICK, Mass.--(BUSINESS WIRE)--Cytovia Therapeutics, Inc., a biopharmaceutical company developing allogeneic off-the-shelf gene-edited iNK cells (NK cells derived from induced pluripotent stem cells, or iPSCs), CAR (Chimeric Antigen Receptor)-iNK cells, and Flex-NK cell engager multifunctional antibodies, announced today that its scientific leadership will present at the following upcoming conferences:

iPSC-Derived Cell Therapies Summit (Virtual Event)

Wednesday, December 8th, 2021, 9.30 AMDemonstrating Safety & Efficacy of iPSC-derived Cells to Maximize Clinical Translation(Industry Leaders Fireside Chat: Paving the Way For the Future of Cell Therapy with iPSCs)Featuring Cytovia Therapeutics CMO Stanley Frankel

Wednesday, December 8th, 2021, 3PMAdvancing Clinical Trial Strategy for Successful Clinical Outcomes(Developing iPSC-Derived NK Cell Therapies Against Solid Tumors)Featuring Cytovia Therapeutics CSO Wei Li

Antibody Engineering & Therapeutics Conference (San Diego, California)

December 12th-15th, 2021Dr. Jean Kadouche, Cytovia Therapeutics scientific co-founder and Global Head of Antibody R&D, will present the Design, Manufacturing, and Activity of FLEX Immune Cell Engager Multi-functional Antibodies poster on behalf of the Cytovia team.

We are proud of the scientific thought leadership of our colleagues Drs. Frankel, Li, and Kadouche, who will present Cytovias latest advances, said Dr. Daniel Teper, CEO of Cytovia Therapeutics. Cytovia is committed to translating disruptive innovation in cell therapy to create therapeutics that advance towards a cancer cure. Cytovia is the first biotech company to develop its own best-in-class gene-edited, iPSC-derived NK cells and NK Engager antibodies, with the potential to combine them for optimal clinical outcomes in both hematological and solid tumors.

About Cytovia Therapeutics

Cytovia Therapeutics aims to accelerate patient access to transformational cell therapies and immunotherapies, addressing several of the most challenging unmet medical needs in cancer. Cytovia focuses on harnessing the innate immune system by developing complementary and disruptive NK-cell and NK-engager antibody platforms. It is developing three types of iPSC-derived (or iNK) cells: unedited iNK cells, TALEN gene-edited iNK cells with improved function and persistence, and TALEN gene-edited iNK cells with chimeric antigen receptors (CAR-iNKs) to improve tumor-specific targeting. The second complementary cornerstone technology is a quadrivalent multifunctional antibody platform designed to engage natural killer cells by targeting NKp46 using its proprietary Flex-NK technology.

These two technology platforms are being used to develop treatment of patients with solid tumors such as hepatocellular carcinoma (HCC) and glioblastoma as well as hematological malignancies such as refractory multiple myeloma.

Cytovias research and development laboratories in Natick, MA and GMP cell manufacturing facility in Puerto Rico are augmented by scientific partnerships with Cellectis, CytoImmune, the Hebrew University of Jerusalem, INSERM, the New York Stem Cell Foundation, and the University of California San Francisco (UCSF).

Cytovia Therapeutics has recently formed CytoLynx Therapeutics, a joint-venture entity focused on research and development, manufacturing, and commercialization activities in Greater China and beyond.

Find out more at http://www.cytoviatx.com and follow us on Facebook, Twitter, LinkedIn, and YouTube.

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Cytovia Therapeutics' Scientific Leadership to Present at Upcoming Conferences - Business Wire

For the 13 patients treated at the recommended phase 2 dose (RP2D) the response rate after one cycle of treatment remains at 100% with a 38.5% complete response (CR) rate; one additional patient completed cycle 1 at the RP2D and was assessed with a partial response (PR)Three of 3 patients treated with two cycles in the dose escalation part of the study at the RP2D remain in CR at 6 months after start of treatmentSide effect profile shows only five instances of transient infusion-related reactions (IRR) in more than 100 AFM13 infusions with no episodes of neurotoxicity, CRS or GvHD

HEIDELBERG, Germany, Dec. 09, 2021 (GLOBE NEWSWIRE) -- Affimed N.V. (Nasdaq: AFMD), a clinical-stage immuno-oncology company committed to giving patients back their innate ability to fight cancer will host today a financial community call to discuss recent findings from the investigator sponsored trial (IST) at The University of Texas MD Anderson Cancer Center investigating the treatment of CD30-positive lymphoma patients with its innate cell engager (ICE) AFM13, pre-complexed with cord blood-derived natural killer (cbNK) cells (AFM13-104).

A treatment cycle consists of lymphodepleting chemotherapy with fludarabine and cyclophosphamide followed two days later by a single infusion of cytokine-preactivated and expanded cbNK cells that are pre-complexed with AFM13, followed by three weekly infusions of AFM13 (200 mg) monotherapy. Responses are assessed on day 28 by FDG-PET and patients can receive up to two cycles. Three patients were treated with 1106, three patients with 1107 and 13 patients with 1108 AFM13-pre-complexed cbNK cells per kg body weight.

Response Assessment

A total of 19 patients with CD30-positive relapsed or refractory Hodgkin and non-Hodgkin lymphomas (17 and 2 patients, respectively) have been treated to date across three dose cohorts. According to investigator assessment, 17 of 19 patients had achieved an objective response (ORR 89.5%) to the treatment, with seven complete responses (CR 36.8%) and ten partial responses (PR 52.6%).

In patients treated at the RP2D level of 1x108 cbNK cells per kg, 12 of 13 had classical Hodgkin lymphoma and 1 patient had CD30-positive NHL. In this cohort, 100% of patients responded after the first cycle of treatment with five CRs (38.5%) and seven PRs (61.5%). All patients treated at the RP2D have now received a second cycle of therapy. Response evaluation after cycle 2 will be reported at a future scientific conference.

Initial Durability of Response Observations

Nine patients treated in the dose escalation phase of the study had follow-up at 6 months. Of note, the three patients treated at the RP2D remain in remission at 6 months after start of treatment, two without additional treatment and one on anti-PD-1 antibody maintenance.

In the four responders out of six treated at the two lower dose levels, one patient, who started treatment in September 2020, remains in remission after consolidation autologous stem cell transplant, and three relapsed at 3.4, 4.8 and 6.3 months after start of therapy.

Safety

Five reported cases of transient infusion related reactions were reported after the monotherapy infusions of AFM13. Of note, there were no instances of serious adverse events such as cytokine release syndrome, immune cell-associated neurotoxicity syndrome or graft-versus-host disease.

Conference Call/Webcast Information

The event today will include a review of Affimeds approach to activating the innate immune system in the fight against cancer, preclinical data supporting the combination of Affimeds ICE molecules with adoptive NK cell transfer, a review of the treatment challenges and clinical opportunities for CD30+ lymphomas, and review of the interim data from AFM13-104 by the studys principal investigator, Yago L. Nieto, M.D., Ph.D., professor of Stem Cell Transplantation and Cellular Therapy at of The University of Texas MD Anderson Cancer Center.

Affimed will host a conference call and webcast today, December 9th, 2021, at 8:30 a.m. EST. To access the call, please dial +1 (409) 220-9054 for U.S. callers, or +44 (0) 8000 323836 for international callers, and reference passcode 3065475 approximately 15 minutes prior to the call.

A live audio webcast of the conference call will be available in the Webcasts section on the Investors page of the Affimed website at https://www.affimed.com/webcasts/investor-day/ or https://edge.media-server.com/mmc/p/zzwismtq. A replay of the webcast will be accessible at the same link for 30 days following the call.

About the Phase 1-2 Study

The University of Texas MD Anderson Cancer Center is studying AFM13 in an investigator-initiated phase 1-2 trial in combination with cord blood-derived allogeneic NK cells in patients with recurrent or refractory CD30-positive lymphomas. The first phase of this study involves dose escalation of pre-complexed NK cells, with patients receiving lymphodepleting chemotherapy followed by 1106 NK cells/kg in Cohort 1; 1107 NK cells/kg in Cohort 2; and 1108 NK cells/kg in Cohort 3. The trial is designed to explore safety and to determine the recommended phase 2 dose and evaluate its activity. The recommended phase 2 dose was determined as 1108 NK cells/kg. In each cohort, the dose of the pre-complexed NK cells with AFM13 is followed by weekly doses of 200 mg AFM13 monotherapy for three weeks, with each patient evaluated for dose-limiting toxicities and responses on day 28. MD Anderson has an institutional financial conflict of interest with Affimed related to this research and has therefore implemented an Institutional Conflict of Interest Management and Monitoring Plan. Additional information about the study can be found at http://www.clinicaltrials.gov (NCT04074746).

About AFM13

AFM13 is a first-in-class innate cell engager (ICE) that uniquely activates the innate immune system to destroy CD30-positive hematologic tumors. AFM13 induces specific and selective killing of CD30-positive tumor cells, leveraging the power of the innate immune system by engaging and activating natural killer (NK) cells and macrophages. AFM13 is Affimeds most advanced ICE clinical program and is currently being evaluated as a monotherapy in a registration-directed trial in patients with relapsed/refractory peripheral T-cell lymphoma or transformed mycosis fungoides (REDIRECT). The study is actively recruiting. Additional details can be found at http://www.clinicaltrials.gov (NCT04101331).

About Affimed N.V.

Affimed (Nasdaq: AFMD) is a clinical-stage immuno-oncology company committed to give patients back their innate ability to fight cancer by actualizing the untapped potential of the innate immune system. The companys proprietary ROCK platform enables a tumor-targeted approach to recognize and kill a range of hematologic and solid tumors, enabling a broad pipeline of wholly owned and partnered single agent and combination therapy programs. The ROCK platform predictably generates customized innate cell engager (ICE) molecules, which use patients immune cells to destroy tumor cells. This innovative approach enabled Affimed to become the first company with a clinical-stage ICE. Headquartered in Heidelberg, Germany, with offices in New York, NY, Affimed is led by an experienced team of biotechnology and pharmaceutical leaders united by a bold vision to stop cancer from ever derailing patients lives. For more about the companys people, pipeline and partners, please visit: http://www.affimed.com.

Forward-Looking Statements

This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as anticipate, believe, could, estimate, expect, goal, intend, look forward to, may, plan, potential, predict, project, should, will, would and similar expressions. Forward-looking statements appear in a number of places throughout this release and include statements regarding our intentions, beliefs, projections, outlook, analyses and current expectations concerning, among other things, the potential of AFM13, AFM24, and our other product candidates, the value of our ROCK platform, our ongoing and planned preclinical development and clinical trials, our collaborations and development of our products in combination with other therapies, the timing of and our ability to make regulatory filings and obtain and maintain regulatory approvals for our product candidates, our intellectual property position, our collaboration activities, our ability to develop commercial functions, clinical trial data, our results of operations, cash needs, financial condition, liquidity, prospects, future transactions, growth and strategies, the industry in which we operate, the trends that may affect the industry or us, impacts of the COVID-19 pandemic, the benefits to Affimed of orphan drug designation and the risks, uncertainties and other factors described under the heading Risk Factors in Affimeds filings with the SEC. Given these risks, uncertainties, and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future.

Investor Relations Contact Alexander Fudukidis Director, Head of Investor Relations E-Mail: a.fudukidis@affimed.com Tel.: +1 (917) 436-8102

Media Relations Contact Mary Beth Sandin Vice President, Marketing and Communications E-Mail: m.sandin@affimed.com Tel.: +1 (484) 888-8195

Continued here:
Affimed to Host Virtual Investor Call Today to Discuss Treatment of CD30-positive Lymphoma ... - The Bakersfield Californian

NEW YORK and SOMERSET, N.J., Dec. 7, 2021 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of cellular therapies for neurodegenerative diseases, and Catalent (NYSE: CTLT), a global leader in enabling biopharma, cell, gene and consumer health partners to optimize development, launch, and supply of better patient treatments across multiple modalities, today announced that the technology transfer for NurOwn manufacturing at Catalent's facility has been finalized. NurOwn is BrainStorm's autologous cellular therapy being developed for the treatment of amyotrophic lateral sclerosis (ALS), progressive multiple sclerosis (PMS) and other neurodegenerative diseases.

Catalent entered into a partnership with Brainstorm in 2020 to provide CGMP clinical supply of NurOwn, in anticipation of the product candidate's potential regulatory approval. NurOwn will be manufactured at Catalent's world-class 32,000 square-foot cell therapy manufacturing facility in Houston, Texas.

"The successful completion of this technology transfer with Catalent is an important step in establishing manufacturing preparedness for NurOwn," said Chaim Lebovits, Chief Executive Officer, Brainstorm Cell Therapeutics. "The manufacturing of cellular therapies such as NurOwn is complex, and requires careful planning and very specific expertise. We are very pleased with the progress we have made with our partner Catalent, which has industry-leading capabilities in this area."

Manja Boerman, Ph.D., President, Catalent Cell & Gene Therapy, said, "Our extensive experience in cell therapy development and scale-up was key to the completion of this technology transfer to our state-of-the-art cell therapy facility in Houston, Texas. We look forward to continuing our partnership with BrainStorm and are committed to enabling the advancement of their autologous stem cell therapy product candidate toward a potential future commercial launch."

About NurOwn

The NurOwn technology platform (autologous MSC-NTF cells) represents a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors (NTFs). Autologous MSC-NTF cells are designed to effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression.

About Catalent

Catalent is the global leader in enabling pharma, biotech, and consumer health partners to optimize product development, launch, and full life-cycle supply for patients around the world.

With broad and deep scale and expertise in development sciences, delivery technologies, and multi-modality manufacturing, Catalent is a preferred industry partner for personalized medicines, consumer health brand extensions, and blockbuster drugs. Catalent helps accelerate over 1,000 partner programs and launch over 150 new products every year. Its flexible manufacturing platforms at over 50 global sites supply over 70 billion doses of more than 7,000 products to over 1,000 customers annually.

Catalent's expert workforce exceeds 17,000, including more than 2,500 scientists and technicians. Headquartered in Somerset, New Jersey, the company generated $4 billion in revenue in its 2021 fiscal year. For more information, visitwww.catalent.com.

About BrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwntechnology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug designation status from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm has completed a Phase 3 pivotal trial in ALS (NCT03280056); this trial investigated the safety and efficacy of repeat-administration of autologous MSC-NTF cells and was supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). BrainStorm completed under an investigational new drug application a Phase 2 open-label multicenter trial (NCT03799718) of autologous MSC-NTF cells in progressive multiple sclerosis (MS) and was supported by a grant from the National MS Society (NMSS).

For more information, visit the company's website atwww.brainstorm-cell.com.

Safe-Harbor Statement

Statements in this announcement other than historical data and information, including statements regarding future NurOwnmanufacturing and clinical development plans, constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may," "should," "would," "could," "will," "expect,""likely," "believe," "plan," "estimate," "predict," "potential," and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorm's need to raise additional capital, BrainStorm's ability to continue as a going concern, the prospects for regulatory approval of BrainStorm's NurOwntreatment candidate, the initiation, completion, and success of BrainStorm's product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorm's NurOwntreatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorm's ability to manufacture, or to use third parties to manufacture, and commercialize the NurOwntreatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorm's ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

Contacts:

Brainstorm Contacts

Investor Relations:John MullalyLifeSci Advisors, LLCPhone: +1 617-429-3548[emailprotected]

Media:Mariesa Kemble[emailprotected]

Catalent Media Contact:Chris HallingPhone: +44 (0)7580 041073 [emailprotected]

SOURCE Brainstorm Cell Therapeutics Inc

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BrainStorm Cell Therapeutics and Catalent Announce Completion of Technology Transfer for NurOwn Manufacturing - PRNewswire

Billions of dollars, hundreds of mice and decades of years are sacrificed to research and development for the average pharmaceutical with no guarantee of success. Nothing about that equation makes sense anymore.

And thats why some of the most impressive minds in science are behind Israeli startup Quris,which is rolling out the worlds first clinical-prediction AI (artificial intelligence) platform to evaluate the safety and efficacy of new drugs.

This newly launched, highly scalable automated platform can test thousands of novel drug candidates at once, on hundreds of miniaturized patients-on-a-chip.

Using AI to predict which drug candidates will safely work in humans is expected to improve efficacy and cut drug development costs dramatically.

Israeli Nobel laureate Dr. Aaron Ciechanover and Moderna cofounder Robert S. Langer are actively involved in guiding the science, technology and strategy of Quris, dual-headquartered in Boston and Tel Aviv.

We are at the tipping point of the modernization of drug discovery. I think the Quris platform could be of significant value to pharma companies and the health of society at large, said Langer, one of 12 Institute Professors at MIT.

Saving money and time is the biggest advantage. Eliminating or at least minimizing the use of rodents in drug testing is also important, and not only for animal welfare reasons.

We are not mice, so what works in animal-based trials is not a proper indicator of what will work for people, explained Ciechanover,a physician and research professor at the Technion Israel Institute of Technology in Haifa.

Using a breakthrough way to test drug candidates on miniaturized patients on chips, Quris can demonstrate their safety and efficacy, or lack thereof, through preliminary chip-based clinical trials. This has never been done before, said Ciechanover.

Quris CEO Isaac Bentwich tells ISRAEL21c that AI-driven drug discovery has become the leading frontier for pharma innovation.

However, this wildly expensive technology does not address clinical safety and efficacy. Therefore, most novel drugs still fail clinical trials costing pharma companies more than $30 billion annually.

Quris is the first AI platform to predict which drug candidates will safely work in humans, filling a critical gap in clinical prediction, Bentwich says.

Part of the Quri team in Tel Aviv, with CEO Isaac Bentwich at right. Photo courtesy of Quris

While there are organ-on-chip technologies available to test the effect of drug candidates on, say, the liver, they cannot achieve high throughput.

To generate large amounts of data, you need a platform to perform thousands of experiments, and the current organ-on-a-chip technology does not allow that to happen. Its usually performed one organ at a time, Bentwich explains.

Our chip-on-chip solution does multiple such experiments to train the AI and let the AI define if the drug is safe or not.

Hundreds of genetically diverse patients

Joining together several organs-on-a-chip to build a patient-on-a-chip has proven cumbersome and unscalable, he adds, citing one such device built at Harvard that took up a big box filled with tubes and pipes.

And that was just a single patient-on-a-chip. Our device is two-by-three inches and simulates a hundred patients on a chip by using different architecture and innovations in nanosensing. Its much more scalable and inexpensive, says Bentwich.

Patient-on-a-chip technology will lower the cost and time of drug testing dramatically. Photo courtesy of Quris

Testing to see if a drug is toxic on a single patient-on-a-chip is better than testing it on a mouse. But its not perfect because different people react differently to the same drug, he adds.

Heres where Quris exclusive partnership with the New York Stem Cell Foundation (NYSCF) Research Institutecomes in.

We generate stem cells, and from them different organs, so you have an entire clinical trial on a chip before testing on humans, Bentwich explains.

By testing thousands of drugs known to be safe or unsafe on male and female patients-on-a-chip from different genomic makeups, we are able to train the AI to create representative samples of populations based on many properties.

First clinical trial for Fragile-X drug

Pharma companies can use Quris platform to develop safer drugs faster. At the same time, Quris is using its platform to develop its own pharmaceuticals.

And it made sense to focus on rare genetic diseases that cannot be modeled in mice and arent common enough to attract development dollars.

The first Quris drug addresses Fragile X Syndrome, the most common inherited cause of autism and intellectual disabilities worldwide. This drug candidate was developed initially at the Hebrew University of Jerusalem.

Our Fragile X Syndrome drug is moving into clinical testing in the first half of 2022, says Bentwich, noting that most AI pharma companies do not have drugs at the human clinical testing phase.

Five years to market

This will be a test case to demonstrate how our system can bring a drug to market in five years with millions of dollars, not 20 years with billions, says Bentwich.

We are focusing generally on central nervous system disorders. Brain and liver diseases and cancer are the three areas where safety and efficacy failures of drugs are especially high, he says.

In addition to 18 granted and pending patents, Quris has partnerships with the Technion for various aspects of development, with Tel Aviv University in nanotechnology, and with Hebrew Universitys NewStem drug-discovery spinoff.

The company is doing all this with a lean team of about 20.

Some of the Quris team in Tel Aviv. Photo courtesy of Quris

Its very moving to see extremely talented young guys fresh out of army, not very experienced, working alongside experienced biologists, chemists, nano scientists, micromechanics and robotics experts, using bioconvergenceto solve big problems, says Bentwich.

Dr. Kobi Richter, founder and chief technology officer of Medinol, explained why he participated in Quris recent $9 million seed round.

Our drug discovery process is broken, and technology darlings across biotech, artificial intelligence, machine learning and big data have not been able to overcome the colossal clinical trial failure rate, said.

With a remarkable team of scientific pioneers at the helm and its extraordinary clinical prediction platform, Quris will be a gamechanger for the industry and lead the next era of drug discovery, Richter said.

For more information, click here

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No animals harmed in new chip drug tests that are faster and cheaper - ISRAEL21c

November 19, 2021 Stem cell therapy helped to reduce the number of heart attacks, strokes and death among people with chronic, high-risk, NYHA class II or III heart failure with reduced ejection fraction (HFrEF), especially among those who have higher levels of inflammation, yet hospitalization was not reduced, according to late-breaking research presented at the American Heart Associations Scientific Sessions 2021.

Heart failure is a condition when the heart is unable to adequately pump blood to meet the bodys need for oxygen and nutrients. In heart failure with reduced ejection fraction (HFrEF), the heart muscle enlarges and weakens, resulting in a decrease in pumping ability and fluid buildup in the bodys tissues. Inflammation plays a significant role in the progression of heart failure over time.

This study set out to examine the effects of using stem cells (mesenchymal precursor cells) injected into the heart to target inflammation and treat chronic heart failure. Researchers hypothesized that a single injection of stem cells from healthy adult donors in addition to guideline-directed medical therapy (GDMT) for heart failure would affect the number of times participants were hospitalized for heart failure events and reduce heart attacks, strokes, and/or death.

Cell therapy has the potential to change how we treat heart failure, said Emerson C. Perin, M.D., Ph.D., the studys lead author, the director of the Center for Clinical Research and medical director of the Texas Heart Institute in Houston. This study addresses the inflammatory aspects of heart failure, which go mostly untreated, despite significant pharmaceutical and device therapy development. Our findings indicate stem cell therapy may be considered for use in addition to standard guideline therapies.

The Randomized Trial of Targeted Transendocardial Delivery of Mesenchymal Precursor Cells in High-Risk Chronic Heart Failure Patients with Reduced Ejection Fraction study also called the DREAM-HF trial, is the largest stem cell therapy study to date among people with heart failure. In this multi-center, randomized, sham-controlled, double-blind trial, researchers enrolled 537 participants (average age 63, 20% female) with heart failure and reduced ejection fraction, which is when the left side of the heart, its main pumping chamber, is significantly weakened.

Heart failure was defined using the New York Heart Association (NYHA) functional classification system. This classification system places patients in one of four categories based on how much they are limited during physical activity. Class I heart failure means no limitation of physical activity, with class IV heart failure meaning an inability to have any physical activity without discomfort.

Participants were randomly divided into two groups: 261 adults received an injection of 150 million mesenchymal precursor cells, commonly known as stem cells, directly into the heart using a catheter. The remaining 276 adults received a scripted, or sham, procedure. Healthy adult donors provided the mesenchymal precursor cells.

The study participants were discharged from the hospital the day after the procedure, and researchers followed these participants for an average of 30 months. The studys focus was to examine if the stem cell treatment affected the likelihood of participants returning to the hospital for treatment of worsening heart failure. They also tracked whether participants had a heart attack or stroke, or died, and measured levels of high-sensitivity C-reactive protein (CRP), a measure in the blood indicating inflammation.

While researchers did not see a decrease in hospitalizations due to the stem cell treatment, they did notice several other significant results. The findings include:

We were impressed to learn that stem cell treatment effects were additive to current standard heart failure treatments, Perin said. For the first time, the known anti-inflammatory mechanism of action of these cells may be linked to a cause-and-effect benefit in heart failure. The stem cells acted locally in the heart, and they also helped in blood vessels throughout the body.

Perin and colleagues believe further research is needed to better understand how these stem cells may affect the course of progression of heart failure and how these therapies may be directed to the patient groups that could see the most benefits.

Limitations to the research include the selection of endpoints commonly used in heart failure studies. The studys results suggest that traditional endpoints associated with recurrent heart failure hospitalization do not fully reveal the benefits or mechanisms of these stem cells on heart attack, stroke and death in patients with chronic heart failure.

Co-authors are Barry H. Greenberg, M.D.; Kenneth M. Borow, M.D.; Timothy D. Henry II, M.D.; Farrell O. Mendelsohn, M.D.; Les R. Miller, M.D.; Elizabeth Swiggum, M.D.; Eric D. Adler, M.D.; Christopher A. James, P.A.; and Silviu Itescu, M.D. Authors disclosures are listed in the abstract.

The study was funded by Mesoblast Inc.

https://www.dicardiology.com/article/late-breaking-science-presentations-aha-2021-meeting

Read more:
Stem Cell Therapy for Heart Failure Reduced Major Cardiac Events and Death - Diagnostic and Interventional Cardiology

Using the Cord Blood Stem Cells market research study, youll have access to in-depth market analysis, statistics, and up-to-the-minute data on the Cord Blood Stem Cells market that youll need to forecast revenue, identify growth drivers and challenges, and identify major players likeBioCell Pty. Ltd., Kaneka Corporation, DomaniCell LLC, Future Health Technologies, Eticur, Advanced Cell Technology Inc., Banco De Celulas Madre, J.P. McCarthy Cord Stem Cell Bank, CyGenics Ltd, AlphaCord, Angel Biotechnology, Becton Dickinson and Company, Cord Blood America Inc., Carolinas CBB, BioCord, Cord Bank, HemaStem Therapeutics, Geron Corporation, Banco De Cordon Umbilical, Family CB Services, Cells for Life, Cytolon AG, Activision Life SA, Golden MediTech Company, Genecell International LLC (GCI), CorCell, Babycord Jordan. The research also focuses on services, the influence of COVID-19 on markets, analytics, billings, management, and the system as significant focus topics.

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According to cutting-edge company improvements and intelligent technology, the Cord Blood Stem Cells study also provides estimates. Any tiny detail, necessity, or data pertaining to current or future needs is included in the Cord Blood Stem Cells market report. As a result of the coronavirus spreading, there have been many ups and downs in market conditions; read the research paper for more information.

The Cord Blood Stem Cells market report includes an overview of the Cord Blood Stem Cells market, as well as definitions and a description of the Cord Blood Stem Cells markets components. For example, market trends, drivers, restrictions and opportunities are all covered in the study. Other topics covered include the economy, the supply chain and finance. There are also technical details like software and communication. There is also a segmentation of the Cord Blood Stem Cells market based on the end-user applicationNeurology, Oncology, Cardiology, Cartilage, Blood Disorders, Diabetes, the technology, the types of products/servicesAllogeneic Procedures, Autologous Proceduresand others as well as the regions North America Europe China Japan South East Asia India and the ROW Additionally, the research includes the Cord Blood Stem Cells markets predicted CAGR, which was calculated using historical data on the Cord Blood Stem Cells market, current market trends, and projected developments. Other market aspects including consumption, asset tracking, and security are also discussed in the research. Overall market summary Growth factors (drivers & restraints); Segmentation; Regional analysis; Revenue; Market players; Latest trends and opportunities.

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Chapter 1.Preface1.1. Report Description and Scope1.2. Research Scope1.3. Research Methodology

Chapter 2.Executive Summary2.1. Cord Blood Stem Cells Market, 20182028(USD Million)2.2. Cord Blood Stem Cells Market: Snapshot

Chapter 3.Global Cord Blood Stem Cells Market Industry Analysis3.1. Market Dynamics3.2. Market Drivers3.3. Porters Five Forces Analysis3.4. Market Attractiveness Analysis

Chapter 4.Global Cord Blood Stem Cells Market Competitive Landscape4.1. Company market share analysis4.2. Strategic development: Acquisitions & mergers, New Service launches, Agreements, partnerships, collaborations, and joint ventures4.3. Research and development and Regional expansion, Price trend analysis

Chapter 5.Global Cord Blood Stem Cells Market Deployment Mode Analysis5.1. Global Cord Blood Stem Cells Market overview, share: By Deployment Mode 20182028(USD Million)5.2. Global Cord Blood Stem Cells Market by Cloud-based, On-premise, 20182028(USD Million)

Chapter 6.Global Cord Blood Stem Cells Market overview: By Component6.1. Global Cord Blood Stem Cells Market share, By Component, 2020 and 20286.2. Global Cord Blood Stem Cells Market by Hardware, Software, Services 20182028(USD Million)

Chapter 7.Company ProfilesOverview, Financials, Service Portfolio, Business Strategy, Recent Developments

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A calculation of the impacting factors based on government regulation and mandates on the market development is also included along with the major players in order to give an inclusive summary of the market.This report also studies Cord Blood Stem Cells market on the basis of its market bifurcation types, applications and major geographical regions United States, Europe, China, Japan, Southeast Asia, India, Central & South America, ROW along with present market trend analysis.

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Global Cord Blood Stem Cells Market With An Annual Growth Rate, The Impact Of Covid-19: FAQ The UK Directory - The UK Directory