Category Archives: Stem Cell Medicine

Research Assistant – Department of Obstetrics and Gynaecology job with NATIONAL UNIVERSITY OF SINGAPORE | 239606 – Times Higher Education (THE)

Roles & Responsibilities

Job description: A Research Assistant (RA) position is available in a leading stem cell research team in the Department of Obstetrics and Gynaecology, NUS Yong Loo Lin School of Medicine, NUHS, National University of Singapore. The job entails work in a laboratory and hospital setting. The successful candidate will work with a team undertaking laboratory research and clinical trials on human umbilical cord Whartons jelly stem cells for the treatment of surgical and diabetic wounds, keloids, bed-sores and blood cancers. The team is currently developing cGMP clinical-grade Whartons jelly stem cells for these applications.

Required skills

Job responsibilities:

Employment type: Medical research

Minimum qualifications: The applicant should possess at least a Bachelors degree in the biological, biomedical, biotechnology, pharmaceutical or allied sciences.

Working hours: 8.30 am to 6 pm on week days. A few additional hours on week-ends or after office hours if experiments require this.

Address of workplace: Department of Obstetrics and Gynaecology Research Laboratories, MD11 Clinical Research Centre, NUS Campus, Kent Ridge, Singapore. Only shortlisted applicants will be notified and interviewed.

Originally posted here:
Research Assistant - Department of Obstetrics and Gynaecology job with NATIONAL UNIVERSITY OF SINGAPORE | 239606 - Times Higher Education (THE)

Getting to the root of why hair goes gray – messenger-inquirer

Marco Kaltofen was 11 when he noticed his first white hairs. As his hair grew whiter, his middle-school friends started calling him the professor. By his mid-30s, it was completely white, as it had been for three of his grandparents. His parents went white in their 40s, so I had no chance of avoiding this, Kaltofen says.

Now 61, he is a civil engineer who lives in Boston. He wears his white hair in a ponytail. White hair is part of my identity, and I am completely at peace with it, he says.

Then there is Joe Rees, 75, a retired customs attache who lives in Washington. He is balding, but the hair that remains on the sides and in the back is the same dark brown it always has been. He jokingly attributes this to clean living and a pure heart, although, like Kaltofen, it probably is genetic. His mothers black hair didnt start to go gray until she was in her 70s, and was 50/50 when she died at 88, he says.

Still, Id rather be gray than bald, he says. That way, I wouldnt have to worry about wearing a hat all the time.

To be sure, Rees and Kaltofen are exceptions, since most people start graying in their 50s and 60s. Nevertheless, their experiences are among the many mysteries of gray, white or silver-looking hair that scientists are exploring to learn more about aging. They want to know why some people turn gray early and others late or not at all and what this might signal about their health. They also want to understand the factors that hasten graying, and even whether gray hair is reversible which could be a boon to those allergic to hair dye, or who hate spending money to keep the gray away.

Most important, studying gray hair could point to new approaches in promoting healthier aging, says Candace Kerr, health scientist administrator in the National Institute on Agings Division of Aging Biology.

While graying is one of the markers of aging aging is the ultimate risk factor for why hair goes gray it highlights the need for better understanding of the mechanisms that drive aging and age-related diseases, she says. To be able to target these pathways will be critically important for our aging population to live longer and happier lives.

Hair that looks gray, white or silver actually is colorless. Hair color comes from melanin, a pigment produced by cells in the hair follicles. Over time, these cells suffer damage and become depleted, losing their ability to make melanin. This results in new hair without pigment meaning, no color.

People use gray, white and silver interchangeably to describe hair that is turning or has turned. Its appearance whether it looks, gray, white or silver depends on how much natural color, or pigment, remains, experts say. Hair that has lost all its color typically appears white.

Studies have identified a number of factors that also may speed up gray hair, including smoking, diet, stress and genetics.

Our hair color depends on a set of specialized stem cells called melanocyte stem cells, and every time a new hair grows, these melanocyte stem cells have to divide in two and make a new melanocyte, [or] pigment cells, explains Melissa Harris, assistant professor of biology at the University of Alabama at Birmingham. These pigment cells stay in the base of your hair and their job is to produce pigment. These melanocytes reach out skinny arms, called dendritic processes, that shuttle the pigment to the hair shaft as it grows. So if all your melanocyte stem cells disappear, so do your melanocytes and so does your hair pigment. Thus gray hair.

Because stem cells directly influence hair color, studying gray hair can provide insights about why stem cells age and ultimately fail, offering important clues about the workings of other stem cells in the body for example, those found in muscles, bones and organs. In turn, these ultimately could point to whether gray hair could be a marker for disease, or the opposite, a longer life. Previous studies have not shown a relationship between life span and gray hair, including whether late onset of gray hair predicts longevity. Some research, however, indicates that gray or white hair can be a sign of early heart disease, regardless of age.

In some people, gray hair could potentially serve as indication of their health for instance when caused by stress, or a signal for those who may be developing cardiovascular disease, Kerr says. We still need to learn more about whether and, if so, how late onset of gray hair can signal better health and longevity in some people under certain circumstances, as well as whether early graying means stem cells might be aging.

There are many different stem cells in our body which may or may not age by different means, she says. How stem cells mark aging overall and how they could interact to promote aging is an important question.

This is why scientists who study gray hair regard it as a valuable research tool.

As gray hair researchers, we often have to defend why we study a cosmetic characteristic, rather than a life-threatening disease, Harris says. But what is very cool about gray hair from a scientific point of view is that we can see it with our own eyes, meaning we dont have to take invasive biopsies, and it doesnt kill you. We have asked a lot of important and interesting questions about stem cells by studying gray hair in mice. And, we are constantly on the lookout for gray-haired mice so we can use our scientific skills to find out what makes them gray.

A 2018 mouse study by Team Hair-Us (Harris nickname for her lab colleagues) found a connection between MITF (microphthalmia), a transcription factor (a protein involved in gene expression) important in managing pigment production, and the innate immune system, suggesting that some peoples hair may turn gray in response to serious illness or chronic stress. They discovered a relationship between genes involved in hair color and those that trigger an immune response to a viral infection, suggesting this interaction could increase the chances of developing gray hair.

MITF, in a sense, shields melanocyte stem cells from our own immune system, she says. Normally our immune system protects our bodies from infection. But for melanocyte stem cells, too much immune response is bad for their health, and this leads to their loss and to gray hair. Why melanocyte stem cells are so sensitive to our own natural means for protection, we still dont know.

Im very curious to see whether we see an uptick in individuals with gray hair due to coronavirus infection, she says. Unfortunately, we probably wont know because gray hair is rarely documented clinically, unless it is very extreme.

Scientists still dont know why some people turn gray early, late, or not at all, although they suspect genes, nutrients and possibly the immune system play a role in depleting melanocyte stem cells.

There is still much to learn about what regulates these stem cells and what may contribute to their loss, says Ya-Chieh Hsu, associate professor of stem cell and regenerative biology at Harvard University and principal faculty member of the Harvard Stem Cell Institute.

Among other things, Hsu studies the effect of stress on graying. Most of us are familiar with those before-and-after photographs of U.S. presidents most recently Barack Obama showing a striking increase in gray hair during their terms, even in relatively young presidents. Its known as the Marie Antoinette Syndrome, after the 18th-century French queen whose hair allegedly turned white overnight before she went to the guillotine and her death at age 38 during the French Revolution.

With the aging process, we gradually lose melanocyte stem cells one-by-one over a very long period of time, Hsu says. What we found in our research was that the stress can accelerate that process.

Hsu and her colleagues found that stress stimulates the same nerves that trigger the fight-or-flight response, which in turn causes permanent damage to the pigment-producing cells in hair follicles. The fight or flight response is thought to be a good thing in stressful situations because it can drive us and other organisms to respond to danger rapidly, Hsu says. This activation causes a spike in the neurotransmitter norepinephrine. Norepinephrine raises our heartbeat and allows us to react quickly to danger without having to think about it.

But norepinephrine also tells melanocyte stem cells to pump up their activity and proliferate, and too much norepinephrine, in this case triggered by stress, causes the melanocyte stem cells to burst into so much activity it leads to rapid depletion of the stem cell reservoir, she says. If all the stem cells are depleted, no more pigment-producing cells can be produced anymore, and the hair turns gray.

Other stress hormones, ACTH (adrenocorticotropic hormone) for example, can cause melanocyte stem cells to migrate away from the hair follicle before they can produce the melanocytes needed for hair and skin color, according to research. Such hormones are known to increase in the body after stress, and may have the potential to promote the loss of these cells, regardless of age, says study author Mayumi Ito, associate professor in the departments of cell biology and dermatology at the New York University Grossman School of Medicine.

Hsu believes the connection between stress and hair color could reveal additional information about how stress affects other biological processes. How stress affects our tissues is still poorly understood, and one of the powerful aspects about the melanocyte is that it provides a visible and highly trackable system to study stress, she says.

Ito also found that certain cell signaling proteins called endothelins (substances known to constrict blood vessels and raise blood pressure) bind to melanocyte stem cells and, in doing so, keep them healthy. Interrupting the process causes cell loss and early graying in mice. They are studying whether the same happens in human hair follicles, hoping to find ways to preserve or regenerate the key stem cells that give hair its color.

All of this raises the intriguing possibility that scientists could discover ways to prevent or reverse gray hair.

Team Hair-Us recently published a paper describing a topical drug combination that increased melanocyte stem cells in gray mice, ridding them of their gray and restoring their original fur color perhaps for good. Because the treatment originally developed to regrow hair replenished pigment-producing stem cells, the effects could be long-lasting, Harris says.

We didnt keep the mice forever so we dont know, says Harris, who plans more studies. This has made us very interested in whether gray hair really is permanent, and if we can do something about it. We really want to know and so does everyone else we talk to is whether and when we can bring this to humans.

Excerpt from:
Getting to the root of why hair goes gray - messenger-inquirer

How the COVID Virus Induces Inflammation, Cytokine Storm and Stress in Infected Lung Cells – SciTechDaily

The coronavirus SARS-CoV-2 docks with its spike protein onto the receptor-ACE2 found on the surface of lung cells. The levels of co-expressed genes with the human receptor-ACE2 were calculated from several publicly available datasets pertaining to lung epithelial cells infected with SARS-CoV-2. The resulting gene-signature consisted of members from the Trans-Membrane-Serine-Protease (TMPRSS) gene family which prime the virus spike protein for cell entry. Other genes from the gene-signature are associated with reduced immune responses and activated inflammation, Reactive Oxygen Species (ROS) and cellular stress. Credit: WruckW /AdjayeJ, HHU Duesseldorf, Germany

The researchers Wasco Wruck and Prof. James Adjaye from the Institute of Stem Cell Research and Regenerative Medicine, Medical Faculty of Heinrich-Heine-University Duesseldorf, Germany, employed a bioinformatic approach on transcriptome data pertaining to human lung epithelial cells infected with SARS-CoV-2. The meta-analysis unveiled several adversely affected biological processes in the lung which no doubt also applies to other infected organs such as the heart and kidney. Their study is published in Scientific Reports.

With a current estimation of more than 60 Mio. infected people and more than 1.4 Mio. recorded deaths worldwide, COVID-19 the disease caused by the coronavirus SARS-CoV-2 and is classified as a pandemic by the WHO poses a threat to public health, national economies, and society as a whole. Although effective vaccines are becoming available, we still need a better understanding of the molecular mechanisms underlying the etiology of COVID-19 to enable more effective therapies in the future.

Wasco Wruck, first author of the study, says: We initially viewed the results of the analysis in an unbiased manner. To our surprise, we noticed that several Transmembrane Serine Proteases besides the already known TMPRSS2 were highly correlated with ACE2 expression. Furthermore, in a more in-depth analysis, the gene-signature could be annotated with numerous biological processes. Among these were regulation of viral life cycle, immune responses, pro-inflammatory responses, several interleukins such as IL6, IL1, IL20 and IL33, IFI16 regulating the interferon response to a virus, chemo-attraction of macrophages, and cellular stress resulting from activated Reactive Oxygen Species.

Prof. James Adjaye, senior author of the study, summarizes: We have shown that not having access or the necessary infrastructure to carry out research with the deadly coronavirus SARS-CoV-2 should not preclude progress in dissecting the etiology underlying COVID-19. There are currently numerous transcriptome-based datasets related to experiments carried out on numerous cell types infected with SARS-CoV-2 which can be retrieved and analyzed to identify adversely affected biological processes with the hope of identifying putative druggable targets that can be used to better manage COVID-19 in the future.

Reference: SARS-CoV-2 receptor ACE2 is co-expressed with genes related to transmembrane serine proteases, viral entry, immunity and cellular stress by Wasco Wruck and James Adjaye, 8 December 2020, Scientific Reports. DOI: 10.1038/s41598-020-78402-2

How the COVID Virus Induces Inflammation, Cytokine Storm and Stress in Infected Lung Cells - SciTechDaily

Medicine by Design symposium highlights importance of convergence in regenerative medicine and human health – News@UofT

Researchersarepoised to makeunprecedentedbreakthroughsinhuman health thanks toadvancesin biomedical and computational sciencesthathave drivencritical tools and technologiessuch as genetic engineering,synthetic biology, andartificial intelligence.

Thats the messageDr. VictorDzau, president oftheU.S.National Academy ofMedicine, delivered to Medicine by Designs fifthannual symposium on Dec. 7 and 8.

Thevirtual event, whichattracted more than 500 registrants from across North America, focused on the theme of better science throughconvergence theintegration of approaches from engineering, science, medicine and other fields to expand knowledge and spark innovation.

I think for younger people, there is really not a more exciting time, in my opinion, to do research than now, because we can really see that some of the initial concepts that people have about health and medicinecan be realizedand truly transform the way we do health andmedicine.

In his talk, Dzau focused on the National Academy of MedicinesHealthy Longevity Global Challenge,an international competition that aims to catalyze transformative ideas and breakthroughs that will extend human healthand lifespan.

That program is one of the inspirations for Medicine by DesignsGrand Questions Program, which seeks to fund bold research that promises dramatically better health outcomes by changing the future of regenerative medicine.

Through our Grand Questions Program, we are thinking about what comes next and how to overcome fundamental problems in regenerative medicine,saidMichael Sefton, executive director of Medicine by Design andUniversity Professorin the department of chemical engineering and applied chemistry and theInstitute of Biomedical Engineeringat the University of Toronto.

We have a broad definition of regenerativemedicine, andpreventing degeneration can be as important as the next cell therapy.

Sefton pointed out that the symposium theme of better science through convergencefocusedon a key aspect of Medicine by Design:That we combine campus and hospital investigators, transformative science and translational elements, junior and senior investigators, and local and international collaborators, to address fundamental problems in regenerative medicine.

Thesymposium also featured a talk byRobert Langer, David H. KochInstitute Professorin the department of chemical engineeringat the Massachusetts Institute of Technology. The most highly cited engineer in history, he spoke about lessons helearnedfromhisscientific and business successes and how he decidedto be his own champion after facing criticism for his novel ideas early in his career.

If you try to do things whether its convergence, or things that a lot of people disagree with you have tohang in there, Langer said.Having good intellectual property has been key toraising the funds to do these things, and medicine is an incredibly expensive thing.

And finally, you really need teams that are super driven, and I think these startup companies have been a wonderful way to do this.

The symposium was organized around four sessions: translation, inflammation, biomaterials andimmunoengineering.Invited speakers from across North AmericaincludedKim Warren(AVROBIO),Kenneth Walsh(University of Virginia),Sarah Heilshorn(Stanford University)andMegan Levings(University of British Columbia).

All speakers fromU of T and its partnerhospitals were lead investigators on Medicine by Designs multi-disciplinary, multi-institution team projects. They included:John Dick,Clinton RobbinsandShaf Keshavjee(University Health Network (UHN));Molly Shoichet(department of chemical engineering and applied chemistry and Institute of Biomedical Engineering);Juan CarlosZiga-Pflcker(Sunnybrook Health Sciences Centre);andAndras Nagy(Sinai Health System).

Ted Sargent, vice-president of research and innovation, and strategic initiatives,and a University Professor in the Edward S. Rogers Sr. department of electrical and computer engineering,opened the symposium by congratulatingMedicine by Design on its successful mid-term review, which was conducted in early 2020 by a panel of international experts and theCanada First Research Excellence Fund(CFREF), which funds Medicine by Design.

Medicine by Design has amplified existing areas ofexcellenceatU of Tandour partner hospitals (Toronto Academic Health Sciences Network),and pushed the boundaries of regenerative medicine to tackle cell-based therapies, strategies for endogenous repair and the use of a stem cell lens to target the triggers of disease,Sargent said. In fact, Medicine by Design is such a compelling collaborative, cross-disciplinary initiative that itis a template fora new class of initiatives at the University ofToronto theInstitutional Strategic Initiativesportfolio whosepurpose is to mobilize ambitious,groundbreaking, collaborative, multi-institutional research networks that tackleimportantresearch problems, buildmajorexternal partnershipsboth with industry and emerging companies as well as with global academic peers;and foster societal impact.

They support the pursuit of grand challenges and bold ideas across disciplinary boundaries,further elevate U of Ts profile in high priority research areas of strategic importance,and enable us to realize transformational impacts on issues of major societal import.

The symposium also offered an opportunity for almost 45trainees to present their research during a poster session.KerstinKaufmann, a post-doctoral fellow in the laboratory ofJohn Dick(Princess Margaret Cancer Centre,UHN), won first place.JonathanLabriola, apost-doctoral fellowinSachdev Sidhuslab(Donnelly Centre for Cellular and Biomolecular Research, U of T), placed second, whileSabihaHacibekiroglu, a post-doctoral fellow in the lab ofAndras Nagy(Lunenfeld-Tanenbaum Research Institute, UHN)placed third.The awards were sponsored by STEMCELL Technologies.

YasamanAghazadeh,a post-doctoral fellow in the labsofCristina Nostro(McEwen Stem Cell Institute, UHN)andSara Nunes Vasconcelos(Toronto General Hospital Research Institute,UHN),won theCCRMTranslation Awardfor the poster with the greatest translational potential.AndAi Tian, a post-doctoral fellow fromJulien Muffatslab (The Hospital for Sick Children), won thePeoples Choice Award, a new award this year that wasdetermined byvotingby symposium attendeesand sponsored byBlueRockTherapeutics.

Funded by a $114-million grant from CFREF, Medicine by Design brings together more than 145principal investigators at the University of Toronto and its affiliated hospitals to work at the convergence of engineering,medicineand science. It builds on decades of made-in-Canada excellence in regenerative medicine dating back to the discovery of stem cells in the early 1960s by Toronto researchers James Till andErnest McCulloch.

Regenerative medicine uses stem cells to replace diseased tissues and organs, creating therapies in which cells are the biological product. Regenerative medicine can also mean triggering stem cells that are already present in the human body to repair damaged tissues or to modulate immune responses. Increasingly, regenerative medicine researchers are using a stem cell lens to identify critical interactions or defects that prepare the ground for disease, paving the way for new approaches to preventing disease before it starts.

(Photo of Robert Langer by Jason Alden)

Medicine by Design symposium highlights importance of convergence in regenerative medicine and human health - News@UofT

2020 in Neuroscience, Longevity, and AIand What’s to Come – Singularity Hub

Covid-19 sucked most of the oxygen out of science this year. But we still had brilliant wins.

The pandemic couldnt bring rockets or humans down: multiple missions blasted off to the red planet in the summer of Mars. Two astronauts launched to the International Space Stationand made it safely backin a game-changer for commercial space travel. NASA released dozens of findings on how space travel changes our bodies, paving the way to keep us healthy in orbitor one day, on Mars and beyond.

Back on Earth, scientists scoured mud ponds and fished out a teeny-tiny CRISPR enzyme that packs a massive punch for genome editing. AI and neuroscience became even more entwinedsometimes literally. Biological neurons got hooked up to two silicon-based artificial neurons, across multiple countries, into a fully-functional biohybrid neural network. Others tapped dopaminethe main messenger for the brains reward systemto unite electricity and chemical computing into a semi-living computer. While still largely a curiosity, these studies take brain-inspired computers to another level by seamlessly incorporating living neurons into AI hardware. Now imagine similar circuits inside the brainNeuralink sure is.

More abstractly, biological and artificial brains further fed into each other in our understandingand craftingof intelligence. This year, scientists found mini-computers in the input tree-like branches of neurons. Like entire neural networks, these cables were capable of performing complex logical calculations, suggesting our brain cells are far brainier than we previously thoughtsomething AI can learn from. On the flip side, a hotshot algorithm inspired by the brain called reinforcement learning pushed neuroscientists to re-examine how we respond to feedback as we learn. AI also helped build the most dynamic brain atlas to date, a living map that can continuously incorporate new data and capture individual differences.

As we leave 2020 behind, two main themes percolate in my mind, not just for what theyve accomplished, but as indicators of what lies ahead. These are the trends Ill be keeping my eyes on in the coming year.

Why we age is extremely complex. So are methods that try to prevent age-related diseases, or slow the aging process itself. This nth-dimensional complexity almost dictates that longevity research needs to self-segregate into lanes.

Take probing the biological mechanisms that drive aging. For example, our cells energy factory spews out bullet-like molecules that damage the cell. The genome becomes unstable. Cells turn zombie-like. Working stem cells vanish. Tissue regeneration suffers. Scientists often spend entire careers understanding one facet of a single hallmark of aging, or hunting for age-related genes. The lucky ones come up with ways to combat that one foefor example, senolytics, a family of drugs that wipe out zombie cells to protect against age-related diseases.

But aging hallmarks dont rear their heads in isolation. They work together. An increasing trend is to unveil the how of their interactions workcrosstalk, in science-speakwith hopes of multiple birds with one stone.

This year, longevity researchers crossed lanes.

One study, for example, took a stem cell playbook to rejuvenate eyesight in aged mice with vision loss. They focused on a prominent aging hallmark: epigenetics. Our DNA is dotted with thousands of chemical marks. As we age, these marks accumulate. Using gene therapy, the team introduced three superstar genes into the eyes of aged mice to revert those marks and reprogram cells to a younger state. Youve probably heard of those genes: theyre three of the four factors used to revert adult skin cells into a stem-cell-like state, or iPSCs (induced pluripotent stem cells). Resetting the epigenetic clock was so powerful it improved visual acuity in old mice, and the team has now licensed the tech to Life Biosciences in Boston to further develop for humans.

Another study combined three main puzzle pieces in agingzombie cells, inflammation, and malfunctioning mitochondriainto a full picture, with the surprise ending that senolytics has multiple anti-aging powers in cells. Talk about killing two birds with one stone. Finally, one team (which I was a part of) combined two promising approaches for brain rejuvenationexercise and young bloodto begin pushing the limits of reigniting faltering memory and cognition due to aging.

Longevity research has long been fragmented, but its starting to coalesce into a multidisciplinary field. These crossovers are just the start of a rising trajectory to combat the multi-headed Hydra thats aging. More will come.

If youre looking for a sign that AI is leaving the digital realm of Atari games and heading into the real world, this year was it.

In biotech, theres no doubt of AIs promise in drug discovery or medical diagnoses. In late 2019, a team used deep learning and generative modelssimilar to AlphaGo, the DeepMind algorithm that trounced humans at Go and wiped the Atari libraryto conjure over 30,000 new drug molecules, a feat chemists could only dream of. This year, the viral hurricane thats Covid-19 further unleashed AI-based drug discovery, such as screening existing drugs for candidates that may work against the virus, or newlydesigned chemicals to fight off SARS-CoV-2 infectionthe virus that causes Covid-19.

For now, we dont yet have an AI-designed drug on the market, an ultimate test for the technologys promise. However, although AI wasnt able to make a splash in our current pandemic battle, the scene is set for tackling the next oneand drug discovery as a whole.

In contrast, AI-based medical diagnosis had a resounding win. This year, the FDA approved a software that uses AI to provide real-time guidance for ultrasound imaging for the heart, essentially allowing those without specialized training to perform the test. The approval brings a total of 29 FDA-approved AI-based medical technologies to date. Even as the debate on trust, ethics, and responsibility for AI doctors cranked up in temperature, the Pandoras box has been opened.

Medicine aside, deep learning further honed its craft in a variety of fields. The neuroscience-AI marriage is one for the ages with no signs of fracture. Outside the brain, AI also gave synthetic biology a leg up by parsing the interactions between genes and genetic networksa mind-bending, enormously complex problem previously only achieved through trial and error. With help from AI, synthetic biologists can predict how changes to one gene in a cell could affect others, and in turn, the cells biochemistry and behavior. Bottom line: it makes designing new biological circuits, such as getting yeast to pump out green fuels or artificially hoppy beer, much easier.

But the coup de grce against AI as an overhyped technology is DeepMinds decimation of a 50-year-long challenge in biology. With a performance that shocked experts, DeepMinds AlphaFold was able to predict a proteins 3D structure from its amino acid sequencethe individual components of a proteinmatching the current gold standard. As the workhorses of our bodies, proteins dictate life. AlphaFold, in a sense, solved a huge chunk of the biology of life, with implications for both drug discovery and synthetic biology.

One more scientific brilliance this year is the use of light in neuroscience and tissue engineering. One study, for example, used lasers to directly print a human ear-like structure under the skin of mice, without a single surgical cut. Another used light to incept smell in mice, artificially programming an entirely new, never-seen-in-nature perception of a scent directly into their brains. Yet another study combined lasers with virtual reality to dissect how our brains process space and navigation, mentally transporting a mouse to a virtual location linked to a reward. To cap it off, scientists found a new way to use light to control the brain through the skull without surgerythough as of now, youll still need gene therapy. Given the implications of unauthorized mind control, thats probably less of a bug and more of a feature.

Were nearing the frustratingly slow, but sure, dying gasp of Covid-19. The pandemic defined 2020, but science kept hustling along. I cant wait to share what might come in the next year with youmay it be revolutionary, potentially terrifying, utterly bizarre* or oddly heart-warming.

* For example, Why wild giant pandas frequently roll in horse manure. Yes thats the actual title of a study. Yes, its a great read. And yes, its hilarious but has a point.

Image Credit: Greyson Joralemon on Unsplash

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2020 in Neuroscience, Longevity, and AIand What's to Come - Singularity Hub

Updated Findings Show Continued Efficacy for CAR T-Cell Therapy in Heavily Pretreated Myeloma – Targeted Oncology

As interest in chimeric antigen receptor (CAR) T-cell therapy continues to grow with more promising data coming out and approvals from the FDA in various hematologic malignancies, the role of this cellular therapy has yet to be defined in multiple myeloma, but recent data have inspired hope for this therapy in the relapsed/refractory population.

The B-cell maturation antigen (BCMA)directed CAR T-cell therapy idecabtagene vicleucel (ide-cel; bb2121) has generated excitement in this population following the submission of a Biologics License Application (BLA) in March 2020, seeking approval of ide-cel in patients with multiple myeloma who have received at least 3 prior therapies, including an immunomodulatory drug (IMiD), a proteasome inhibitor (PI), and an anti-CD38 antibody, and a Priority Review designation granted in September 2020. Following delays in the review due to coronavirus disease 2019, the Prescription Drug User Fee Act action date has been set as March 27, 2021.

Deep and durable responses were observed with ide-cel as treatment of heavily pretreated patients with relapsed/refractory multiple myeloma, according to updated results from the CRB-401 study presented by Yi Lin, MD, PhD, assistant professor of oncology and associate professor of medicine at Mayo Clinic, during the 2020 American Society of Hematology (ASH) Annual Meeting. The efficacy and safety findings were consistent with prior findings and supported a favorable clinical risk-benefit profile at target dose levels 150 x 106.1

The median overall survival with ide-cel was 34.2 months (95% CI, 19.2-not evaluable) among all patients in this triple-classexposed population, and half of the patients who had ongoing responses achieved a duration of response >2 years. The median progression-free survival (PFS) was 8.8 months (95% CI, 5.9-11.9). The objective response rate (ORR) overall was 75.8%, which included complete responses (CRs) in 38.7%.

These results from CRB-401 are comparable to the findings from the pivotal phase 2 KarMMa study (NCT03361748), which were presented earlier this year during the 2020 American Society of Clinical Oncology (ASCO) Virtual Scientific Program and support the Biologics License Application. The median OS for this study was 19.4 months, and the median PFS was 8.8 months. The ORR was 73%, which included a CR rate of 33%, and the median duration of response was 10.7 months.2

Ide-cel is being explored in several ongoing studies as well, including the phase 2 KarMMa-2 (NCT-3601078), phase 3 KarMMa-3 (NCT03651128), and phase 1 KarMMa-4 (NCT04196491) clinical trials. These phase 2 and 3 studies are evaluating ide-cel in patients with triple-classexposed disease, and the phase 1 study will explore the use of this CAR T-cell therapy in patients with high-risk newly diagnosed multiple myeloma.

These data have also set the stage for other BCMA-directed CAR T-cell therapies in development for the treatment of patients with multiple myeloma.

In an interview with Targeted Oncology, Lin discussed the updated findings from the CRB-401 study of ide-cel as treatment of patients with relapsed/refractory multiple myeloma.

TARGETED ONCOLOGY: What historical data have we seen with BCMA-directed CAR T-cell therapy in patients with relapsed/refractory multiple myeloma?

Lin: With the CAR T approach in multiple myeloma, the very first case report was actually with CD19-targeted CAR T because there was already experience with that particular antigen in leukemia and lymphomas. There's some ongoing effort in terms of dual targeting with CD19 and BCMA, but BCMA very quickly emerged as an ideal candidate for the myeloma space. This is an antigen that is more uniformly expressed on plasma cells, including myeloma cells, and maybe a small subset of mature B cells, but otherwise BCMA is not expressed on healthy tissues.

There have been some single-center clinical trials with the BCMA-targeted CAR T approach prior to the CRB-401 study, both with National Cancer Institute and the University of Pennsylvania with slightly different constructs. With those early phase 1 studies, there was a little bit more toxicity seen, although there was certainly some response, but the response wasn't particularly durable. CRB-401 is the first in a series of now industry-sponsored multicenter studies, in which we are now seeing a much more encouraging durable response rate and also a more favorable side effect profile as well. At ASH this year, I presented the longer follow-up on the phase 1 CRB-401 study. There is a pivotal phase 2 KarMMa study using the same CAR T construct that had been presented at ASCO earlier this year.

TARGETED ONCOLOGY: Please describe the design of the trial and what was different about the study.

Lin: The CRB-401 study has 2 parts. The first part is the dose-escalation part, and the second part is the dose expansion. The dose escalation is basically testing the range of a fixed dose of 50 million all the way up to 800 million of ide-cel CAR T cells in a relatively small number of patients, basically looking for signs of severe side effects to identify a safe dose. The dose expansion cohort is where we take the more promising doses in terms of response, and also safety profile, and test them in more patients to get a better safety signal, which is then moved forward for phase 2 testing in the KarMMa study.

In the dose-expansion portion of CRB-401, we required that each patient must have had exposure to an anti-CD38 antibody. That was allowed in a dose escalation but not required for everybody. [To be included in the study,] the patient must have had become refractory to the most recent line of treatment before they came on the study. The other thing that was different was that in the dose-escalation cohort, all patients had their myeloma cells in the bone marrow reviewed centrally by immunohistochemistry staining, and they were required to have at least 50% of these cells having BCMA expression in a dose-expansion cohort, to better understand the clinical efficacy and safety profiles of this treatment. We also included some patients that had BCMA expression below that to even levels that were not detectable by immunohistochemistry.

TARGETED ONCOLOGY: What were the results from this study?

Lin: The study [included] a total of 62 patients. The results from the first 33 patients were already published in the New England Journal of Medicine last year, and this year at ASH, data were presented for outcomes of the entire 62-patient cohort, with a median follow-up of now 18.1 months. What we have seen so far is in this entire treated patient cohort these are patients with very high-risk features of myeloma, and close to a third of these patients had high-risk cytogenetics, 37% of these patients had extra modularity plasma effect, and almost half of these patients needed some type of systemic therapy while their CAR T cells are being made. These patients, on average, had 6 lines of prior therapy, and in close to 70% or higher, these patients are either triple-refractory or were refractory to the most recent line of therapy.

For this group of patients that was treated overall, the safety signal was very tolerable, which is not surprising with CAR T therapy because these patients also do get lymphodepletion chemotherapy as part of the treatment with CAR T. We do see that low blood count is the most common side effect, including the more severe low blood counts, but on average, the recovery of these blood counts can be seen well under the first 3 months after CAR T infusion. The other most common side effects that we need to watch for with CAR T are cytokine release syndrome (CRS) and neurotoxicity. What we have seen in this study is that, on average, about 76% of these patients had some type of CRS. However, those that had grade 3 or higher, that is only [seen] in 6.5% of the patients, so much lower, and that's also reflected in the relative lower use of tocilizumab and steroids, as well, to manage the side effects. About 35% of these patients had some type of neurologic side effect, but again, only 1 patient had a more severe form of neurotoxicity. Compared to what we have seen with the CAR T experience in the lymphoma/leukemia space, this is a very, very encouraging safety profile.

We have also now seen that the ORR is quite high. It's 75.8% with a CR and stringent CR rate of about 38.7%. Many of these patients that had bone marrow that were evaluable for minimal residual disease (MRD) response were MRD-negative. We are seeing, since we tested many doses, that there is a dose-related increase in response with increasing [the] dose, and we have also seen that the duration of response is 10.3 months. When we look at the dose that was tested as well in those expansions [in] the 150 to 450 range, what we have seen is that the duration of response is comparable, so not significantly decreased, for patients with high-risk features like those with extramedullary disease for older patients, as well as patients who needed to get bridging therapy during treatment. The median PFS is 8.8 months, and the median OS is 34.2 months.

So far, the response rate, duration of response, and PFS seem to be comparable to what we also now see in the KarMMa study, which has less follow-up, but we are seeing a very nice median OS for a treatment in which we're just giving a 1 dose infusion and no follow-up maintenance therapy.

TARGETED ONCOLOGY: In terms of CAR T-cell therapy, how do you see this strategy impacting this patient population in the future?

Lin: I think there's definitely a role for this in the practice. The BLA for ide-cel has been submitted to the FDA, so we're anticipating review sometime in early 2021. This is very exciting because this could very well be the first CAR T for multiple myeloma. I think this would definitely be a treatment option for these patients. Based on how KarMMa is designed, we anticipate that the FDA approval will be in the space of patients who [have] had at least 3 lines of prior therapy and have been exposed to the currently approved 3 main backbones of treatmenta PI, IMiD, and the CD38 antibody. The full detail is pending final FDA review and the label. However, in that space, certainly looking at the demographic of the patient that's been treated so far as CRB-401 and KarMMa, that's a wider group of patients. Based on the fact that this is a treatment that is a basically living active cells, I perceive that the earlier that patient could get this therapy in the earliest possible approved indication, there would likely be potentially more benefit for the patients.

TARGETED ONCOLOGY: Do you think there is hope for this treatment in other hematologic malignancies outside of lymphomas and leukemias as well?

Lin: That is actually a very interesting question because what we're seeing in terms of the severity of CRS and neurotoxicity is a reflection of our evolving learning about how to manage the toxicity, as well. There is a component to the CAR design, the disease, the nature of the disease, the kinetics of the CAR T actions, in the manifestation of these symptoms. What we are seeing now, with even the prior CAR and next-generation CAR coming on, we will likely see an ongoing improvement in terms of a reduction of severity of these symptoms and also in the ways that we could manage these symptoms.

The fact that myeloma would be the next disease that has an FDA-approved CAR T also relates to the fact that the BCMA antigen is more restricted on the cell type where the malignancy is involved, similar to CD19 for lymphoid malignancy. We are seeing that there are some challenges, for example with acute myeloid leukemia or myeloid neoplasms where a number of antigens could overlap with stem cells, which we wouldn't want to try to hurt. There are some novel CAR approaches to try to overcome that, and those are in very early phase testing, so we'll need to see how those results evolve.


1. Lin Y, Raje NS, Berdeja JG, et al. Idecabtagene vicleucel (ide-cel, bb2121), a BCMA-directed CAR T cell therapy, in patients with relapsed and refractory multiple myeloma: updated results from phase 1 CRB-401 study. Presented at: 2020 ASH Annual Meeting & Exposition; December 5-8, 2020; Virtual. Abstract 131.

2. Munshi NC, Anderson Jr LD, Jagannath S, et al. Idecabtagene vicleucel (ide-cel; bb2121), a BCMA-targeted CAR T-cell therapy, in patients with relapsed and refractory multiple myeloma (RRMM): Initial KarMMa results.J Clin Oncol. 2020;38(suppl):8503. doi:10.1200/JCO.2020.38.15_suppl.8503

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Updated Findings Show Continued Efficacy for CAR T-Cell Therapy in Heavily Pretreated Myeloma - Targeted Oncology

Cell Isolation/ Separation Market worth $15.0 billion by 2025 – Exclusive Report by MarketsandMarkets – PRNewswire

CHICAGO, Dec. 11, 2020 /PRNewswire/ -- According to the new market research report "Cell Isolation/Cell Separation Market by Product (Reagents, Beads, Centrifuge), Cell Type (Human, Animal), Cell Source (Bone Marrow, Adipose), Technique (Filtration), Application (Cancer, IVD), End-User (Hospitals, Biotechnology) - Global Forecast to 2025", published by MarketsandMarkets, the global Cell Isolation Market size is projected to reach USD 15.0 billion by 2025 from USD 6.9 billion in 2020, at a 16.8% CAGR.

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The increasing government funding for cell-based research, the increasing number of patients suffering from cancer and infectious diseases, technological advancements, and the growing focus on personalized medicine are the major factors driving the cell isolation markets growth. Emerging economies such as China and Japan are providing lucrative opportunities for the players operating in the market.

The consumables segment accounted for the largest share of the market, by product segment, in 2019

Based on product, the Cell Separation Market is segmented into consumables and instruments. In 2019, consumables segment accounted for the largest share. This can be attributed to the increasing investments by companies to develop technologically advanced products as well as the repetitive use of consumables.

Centrifugation- based segment accounted for the largest share of the cell isolation market, by technique segment, in 2019

Based on technique, the Cell Separation Market is segmented into centrifugation-based cell isolation, surface marker-based cell isolation, and filtration-based cell isolation. primarily due to the wide usage of this technique among end users. This technique is used on a large scale by biotech and biopharmaceutical companies as well as on a small scale by clinical research organizations and academia. The cost-effectiveness of this technique is another major reason for the large share of this segment. The growing demand for centrifugation techniques in biotech and biopharmaceutical companies is a major factor that is expected to drive the growth of this market segment in the coming years.

The biotechnology and biopharmaceutical companies segment accounted for the largest share in the market, by end user segment, in 2019

The cell isolation market is segmented into hospitals and diagnostic laboratories, biotechnology and biopharmaceutical companies, research laboratories and institutes, and other end users based on end users. In 2019, the biotechnology and biopharmaceutical companies segment accounted for the largest share. The widespread adoption of advanced instruments in cell-based experiments and cancer research in biotechnology and biopharmaceutical companies, as well as the increasing number of R&D facilities globally can be attributed for the large share of this end-user segment.

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North America is the largest regional market for cell isolation market

The Cell Separation Market is segmented into four regions, namely, North America, Europe, Asia Pacific and Rest of the world. In 2019, North America accounted for the largest share of the market. The large share of North America can be attributed to technological advancements, growth in the biotechnology and pharmaceutical industries, increasing prevalence of chronic and infectious diseases, and higher investments in cell-based research in the region.

The major players operating in this Cell Separation Market are Thermo Fisher Scientific, Inc. (US), Becton, Dickinson and Company Limited (US), Beckman Coulter Inc. (US).Merck KGaA (Germany), Terumo BCT (Japan), GE Healthcare (US), Bio- Rad Laboratories Inc. (US), Corning Inc. (US), Roche Diagnostics (Switzerland, Alfa Laval (Sweden), Miltenyl Biotech (Germany), pluriSelect Life Science (Germany), STEMCELL Technologies Inc. (Canada), Akadeum Life Sciences, Inc (US), Bio- Techne (US), Bio Legend (US) and Invent Biotechnologies (US).

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Single-Cell Analysis Market by Cell Type (Human, Animal, Microbial), Product (Consumables, Instruments), Technique (Flow Cytometry, NGS, PCR, Mass Spectrometry, Microscopy), Application (Research, Medical Application), End User - Global Forecasts to 2025

Cell Expansion Market by Product (Reagent, Media, Flow Cytometer, Centrifuge, Bioreactor), Cell Type (Human, Animal), Application (Regenerative Medicine & Stem Cell Research, Cancer & Cell-based Research), End-User, and Region - Global Forecast to 2025

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Cell Isolation/ Separation Market worth $15.0 billion by 2025 - Exclusive Report by MarketsandMarkets - PRNewswire

Cord blood banks sell parents on promising stem cell research, but with no guarantees – The Arizona Republic

Stem cell treatment promise sells pregnant parents on cord blood banks Arizona Republic

Moments after Linda Buzans son Luca is born, her OB-GYN goes to work. She carefully cuts the white umbilical cord, then clamps it shut before any blood can escape. Once the cord is cleaned, she carefully inserts a needle with a long tube attached and lets the umbilical cord blood pump into a collection bag.

From there, the sample will travel in a labeled collection box to Tucson, where a laboratory for one of the oldest and biggest private cord blood banking companies nationwide is located. There, at the Cord Blood Registry laboratory, or CBR,baby Lucas umbilical cord blood will be frozen in a metal tank at less than minus 320 degrees, joining almost 900,000 other samples in storage,including that of his older sister, Lola.

Umbilical cord blood is full of stem cells, so it can be transplanted into patients to treat certain types of cancers, diseases and blood disorders. Umbilical cord blood works as an alternative for many patients who cant find bone marrow transplants.

Butthe odds that either Lola or Luca will develop a disease like cancer that would require an umbilical cord blood transplant are slim,about one in 1,000 or one in 2,000, according to University of Arizona umbilical cord blood stem cell researcher David Harris.

Its difficult to reconcile saving for yourself because youre afraid of cancer, Harris said. Do it based on facts, not fear.

Stem cells in umbilical cord blood could have another purpose: regenerative medicine. Current clinical trials show promise for the use of umbilical cord blood to treat a host of conditions such as neurological disorders, orthopedic injuriesand even diabetes. These potential usages are a new draw for parents to bank their childs umbilical cord blood.

The odds of use for these regenerative medicine applications is much, much higher, Harris said.

He estimates the odds of developing a disease that could be treated by umbilical cord blood stem cells is about one in ten.

Butthe science is still developing, which meanscompanies are selling parents ona product they may never be able to use.

In the past two decades, Harris has been involved with studies to treat kids with conditions like cerebral palsy, strokes, traumatic brain injuries and diabetes.

When you now start to talk about being able to treat a knee injury or a heart attack, or a stroke the ability to actually do that and then to see that it works is very exciting, he said. And thats really where the use of cord blood is going.

So far, Harris said hes seen the most success with orthopedic injuries and in treating children that have suffered from strokes.

With strokes, Harris said he observed children go from being completely paralyzed on one side to being fully functioning.

DONATIONS:Donated cord blood saved Sophie Lee's life, but most parents throw it away

Though some initial results show promise, Harris acknowledges that to move forward with any of these treatments, researchers need to demonstrate a good success rate.

The big question when it comes to using cord blood for regenerative medicine is when it will be incorporated into actual medical practice. For safety reasons, FDA approval for new treatments can take years,if not decades.

Currently, the only way to use umbilical cord blood stem cells for FDA-approved regenerative medicine is to qualify for and register in clinical trials to treat specific conditions. Harris has banked his owns sons cord blood on his belief that more and more umbilical cord blood treatments will become commercially available in the future. Buzan has done the same.

Brandon Buzan packages up his newborn son's umbilical cord blood to be shipped to the Cord Blood Registry lab for storage.(Photo: Amanda Morris)

Its not something that you want to say I wish I had done this, because you cant do it later.You have this one shot,"Buzan said."Even its like one percent of saving their life or helpingI mean for your child, youd do anything as a parent.

In Arizona, obstetrics health providers are required by law to educateexpectant parents about the options to publicly donate or privately bank cord blood. YetDr. Jaime Shamonki, the Chief Medical Officer of CBR, estimates that less than 5% of the population chooses to bank cord blood. A small percentage donate the blood, but a much larger percentage simply throws it away.

Kelly Helms, a Scottsdale-based OB-GYN, said the most common reasonher patients give for not banking their childs cord blood is the cost.

Buzan, who is one of her patients, said she got a discount to bank her childrens samples with CBR because of a connection her familyhad with the company. She paid a little over $1,000 for the initial processing and storage fee for both samples, and continues to pay an annual storage fee of about $120 for each one.

To bank one sample of cord blood and cord blood tissue, the initial cost is$2,830, according to CBR, with a $360 annual storage fee after that. To bank just the cord blood, not the tissue, the initial cost is $1,680, with a $180annual storage fee. Both cord blood and cord tissue have different types of stem cells that are thought to potentially repair the body in different ways.

The stem cells and potential treatments for both sources arent fully understood yet, so there are no guarantees that parents or children will actually be able to use the samplesthey pay to store.

Of the nearly 900,000 samples CBR keeps in storage, Shamonki estimates about 600 have been released for customers to use,representing a usage rate of less than 1 percent. According to Shamonki, the low sample release rate is due to FDA regulations, which stipulate that umbilical cord blood can only be used in approved treatments or clinical trials.

If we didnt have the FDA, then I would be able to release like thousands of units, she said. Its really a regulatory problem, its not a utility problem.

To boost usage of the samples, CBR maintains a registry to match eligible customers to clinical trials and has partnered with research institutions to sponsor clinical trials.

Despite FDA regulations, which Shamonki acknowledges are important to protect public health, she said CBR is releasing more and more units every year. Of the samples taken out, about 83% are used for regenerative medicine purposes, according to CBR.

What I know is that in the next fiveyears, next 10 years, there will be so many more opportunities, Shamonki said. So just because you dont have 100 different clinical trials you can sign up for tomorrow doesnt mean that these applications wont exist in fiveor 10 years, and your child might need it.

CBR is one of many companies that market cord blood banking to new parentsand is one of the biggest. Helms said she always recommends her patients to do their research and pick one of the larger, more established cord blood banking companies. Such companies might includeCBR, Cryo-cell, or ViaCord.

I've had patients lose their cord blood, privately banked blood, because they went with a small company and now they closed down, Helms said.

Even with some of the larger companies, the process of cord blood banking doesnt always run smoothly.

Although she couldn't have her daughter's cord blood stored, Chelsea Radford says she paid over $1,000 to ViaCord for processing fees.(Photo: Amanda Morris)

The first time Phoenix-resident Chelsea Radford heard about cord blood banking, she was already pregnant and facingmyriaddecisions that were sometimes overwhelming. She had never heard of it before reading some pamphlets from her gynecologist, but she was quickly sold on the idea of banking her daughters cord blood and tissue with ViaCord.

Radford has a history of Alzheimers disease in her family, and said she was initially interested in what potential treatments cord blood and tissue might offer for the disease. In 2015, one study suggested that human umbilical cord blood cells could have therapeutic benefitsin mice with Alzheimers disease.

In addition to researching studies, Radford said she spent hours looking at different cord blood banking companies and asking representatives questions about the process. Of all the companies, she found ViaCord to be the most responsive and willing to answer her questions in-depth.

Having the communication and the availability that is what sold me on ViaCord. But that really quickly ended there with the sale, she said.

On the day of her daughter Brylees birth in July2018,Radford went to a hospital that ViaCord had assured her was familiar with collecting cord blood. Soon after, she got a call from the company saying her sample couldnt be stored because not enough blood was collected. Shestill owed ViaCord over $1,000 for a lab processing fee.

Radford wanted more information before she would agree to pay, and said she spent monthscalling, leaving the company messages, and getting no response.

After the birth, nobody responds to anything, she said.

The company called her three months later to tell her that if she didnt pay her bill, they would send it to collections.

I got pissed! Radford said. The only thing they seem to care about is the moneythey dont care about is having a conversation with me about why and how this sample didnt turn out the way it shouldve. All they want to talk to me about is the money.

Still, Radford said she refused to pay a dime until she got an explanation. She contacted the doctor who delivered her baby and said she learned the doctor had never done a cord blood collection before and had never been trained on how to do one.

Finally, she spoke with a ViaCord representative, who she saidtold her this sometimes happens, butthe company wasnt responsible for the fact that the doctor who took her sample didnt take enough blood.

Frustrated and inundated with other responsibilities that come with caring for a newborn, Radford said she decided to pay the fee so that she could move on.

We paid a company to do nothing for us just to get them to leave us alone and not send a bill to collections that I dont feel like we shouldve had to foot in the first place, she said.

If she had a second child, Radford said she wouldnt choose to cord blood bank again, and doesnt recommend it to other moms.

You can still get stem cell help without using your own banked blood and tissueThis is just a costly option that is not a given that its going to work out 100% in your favor, she said. You could have a newborn and be responsible to pay thousands of dollars for nothing.

ViaCord did not respondto multiple requests for comment.

If parents decide to pay for private banking, Radford said they should be careful about making sure the doctors delivering their children know how to collect the samples. Shesaid blood banking companies should be more responsible in making sure that doctors are trained in blood collection.

While Helms is comfortable doing cord blood collection, she was never formally taught how to do it while studying and training to be an OB-GYN.

It was basically taught by the company, she said. Each kits a little different.

Helms said the procedure is fairly simple, but every once in a while, she comes across a company shes never heard of, and a kit she is unfamiliar with. Sometimes she needs to take on the extra responsibility of making sure she understands the directions for that particular kit.

Each company really should take on the responsibility, she said. I can't surf the Internet and look for every YouTube video on every cord blood banking company, she said.

Another potential complication in banking cord blood orblood tissue is that the blood or tissue can become infected.

Birth is not that clean of a process and ideally when you take that needle and you drain the umbilical cord, youll have cleaned that umbilical cord first and you hope that no bacteria get in to the cord blood unit, but its possible, it does happen on a number of occasions, Shamonki said.

Shamonki says CBR tests for any bacterial contamination before storing the tissue and works with parents who have infected samples to discuss possibilities of being able to use the unit in the future.

A Cord Blood Registry worker processes cord blood for storage in the company's Tucson laboratory.(Photo: Amanda Morris)

Nobody knows when regenerative medicine applications for cord blood will become readily available as FDA-approved mainstreamtreatments. New applications for cord blood are being tested every year and new technologies to expand and utilize stem cells in cord blood are constantly being developed.

We dont really know what the limits are, but there are limits to what (umbilical cord blood stem cells) can do, Harris said.

Because cord blood banking is so new it has only been around since 1989 its unclear how long samples can be stored and remain effective.

According to Harris, cord blood samples can still work after being stored for about two decades.

We recently took one out that was 24, 25 years old, he said.

He speculates that properly stored cord blood samples could probably work throughout a person's lifetime, if not longer.

Buzan is aware that stem cells and cord blood treatments are still a new science with no guarantees, but she also believes in the treatments future potential.

Every month, she and her husband receive email updates from CBR that explain some of the new clinical trials and research discoveries involving cord blood.

To be honest the most exciting thing is the unknown the unknown of what the cord blood could do, what theyre looking into now, thats fascinating, she said. Im just so glad we live in a time that where this is available to use this was not an option for my parents or my grandparents.

Amanda Morris covers all things bioscience, which includeshealth care,technology, new researchand the environment. Send her tips, story ideas, or dog memes at and follow her on Twitter @amandamomorris for the latest bioscience updates.

Independent coverage of bioscience in Arizona is supported by a grant from the Flinn Foundation.

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Cord blood banks sell parents on promising stem cell research, but with no guarantees - The Arizona Republic

Procyon Technologies LLC and Novo Nordisk A/S to Collaborate on the Development of a Stem-Cell Based Therapy for Type 1 Diabetes – PRNewswire

TUCSON, Ariz., Dec. 8, 2020 /PRNewswire/ --Procyon Technologies LLC today announced that it has entered into an exclusive research collaboration and license agreement with Novo Nordisk A/S to develop an implantable cell encapsulation device to be used in Novo Nordisk's development of a novel therapy for Type 1 diabetes.

The collaboration brings together Procyon Technologies' expertise with development of oxygen enabled implantable cell encapsulation devices and Novo Nordisk's expertise in stem cell-derived insulin-secreting cells.

The partners will work together to further optimize the device and cells for clinical testing and accelerate the path to First Human Dose with the joint vision of delivering a functional cure for people living with Type 1 diabetes.

Under the terms of the agreement, Procyon Technologies, a startup founded to commercialize innovations developed at the University of Arizona College of Medicine Tucson, will receive an upfront license fee and will be eligible for further payments relating to preclinical, clinical and regulatory milestones. In addition, Procyon Technologies will receive tiered sales milestones and royalties on the annual net sales of the products resulting from the collaboration.

Novo Nordisk will be responsible for the development, manufacturing and commercialization of the products resulting from the collaboration for Type 1 diabetes.

The right cells and the right device

Type 1 diabetes is an autoimmune disease in which insulin-producing beta cells in the pancreas are mistakenly destroyed by the body's immune system. For people with Type 1 diabetes, life-long daily administration of insulin to control their blood sugar and constant blood glucose monitoring is the burden of reality.

"If we are able to offer a treatment that safely and effectively replaces the insulin-producing cells that people with Type 1 diabetes have lost, we could essentially offer them a functional cure for their disease," said Jacob Sten Petersen,DMSc, corporate vice president and head of stem cell research and development for Novo Nordisk.

Since 2008, Novo Nordisk has invested in human stem cell technology and worked on generating a protocol for stem cell-derived insulin producing islet-like clusters for the treatment of Type 1 diabetes.

But having the right cells is only half the solution; the cells also need to be protected from the recipient's immune system to avoid rejection, as well as from the autoimmunity of Type 1 diabetes.

For the last two decades, Procyon Technologies co-founder Klearchos Papas, PhD, a professor in the Department of Surgery and director of the Institute for Cellular Transplantation at the University of Arizona College of Medicine Tucson, has been working on a solution to the second part of that challenge.

"As a pancreas transplant surgeon, the idea of replacing beta cell function in a diabetic patient to prevent progression of diabetic complications makes perfect sense," said Michael M.I. Abecassis, MD, MBA, dean of the UArizona College of Medicine Tucson and professor of surgery and immunobiology. "Therefore, the notion of doing this without the need for major surgery and without the need for anti-rejection drugs by leveraging the assets of academia with those of industry represents the next frontier in curing Type 1 diabetes and preventing its complications."

With support from JDRF International and the National Institute of Diabetes and Digestive and Kidney Diseases, and utilizing key University of Arizona facilities and infrastructure (such as the BIO5 Institute device prototyping lab), Dr. Papas and his team developed oxygen enabled implantable immuno-isolation device technology with a focus on safety, practicality, and the maintenance of viability and functionality of encapsulated cells.

"We are delighted and excited to join forces with Novo Nordisk and provide the 'implantable encapsulation device' part of the functional cure for people suffering from Type 1 diabetes. Novo Nordisk is a leader in the development of stem cell-derived insulin producing islet-like clusters, has demonstrated strong commitment, and has the capacity, infrastructure and most importantly, the shared vision and interest in seeking to bring this functional cure to patients," said Dr. Papas.

"The combination of the implantable encapsulation device with islet-like clusters provides a unique opportunity to develop a novel cell therapy for diabetes. This announcement reinforces the value of JDRF in supporting science and technologies that can be further advanced in partnerships," said Esther Latres, PhD, assistant vice president of research at JDRF.

"Dr. Papas' work exemplifies our research mission in the Department of Surgery. The collaboration between our investigators and clinicians allows for the development of innovative, cutting-edge solutions to the clinical problems we treat every day," said Taylor S. Riall, MD, PhD, chair of the UArizona Department of Surgery. "The partnership between Procyon Technologies and Novo Nordisk represents the culmination of years of hard work and will revolutionize the care of people with Type 1 diabetes."

A therapeutic implant

The Procyon cell encapsulation device is a small, flat, thin, highly durable, flexible implantable chamber. It mitigates foreign body response, promotes the formation of vascular structures on its surface enabling the rapid diffusion of nutrients to the cells inside and the rapid absorption of insulin (or other therapeutic molecules) secreted by the encapsulated cells while providing a barrier protecting them from attacks by the body's immune system without the need for immunosuppressive drugs. The Procyon technology, designed with practical clinical use as a driver, includes integration of oxygen delivery to the implantable device, which enables tighter packing of cells while maintaining their viability and functionality.

About Procyon Technologies LLC:

Procyon Technologies LLC ( was founded in Arizona in 2016. Klearchos Papas, PhD, Allison F. Corkey, MS, Thomas Loudovaris, PhD, and Robert C. Johnson, PhD, are co-founders and worked with Tech Launch Arizona, the University of Arizona commercialization arm, to protect the intellectual property and license the platform technology suitable for the implantation of a variety of therapeutic cells aimed at treating a number of disorders. In addition to being highly respected researchers in the field of diabetes and encapsulation therapy for decades, Dr. Johnson, a part-time research professor in the Department of Surgery at the University of Arizona, has had Type 1 diabetes for nearly 51 years and Dr. Loudovaris is the father of two children with the disease.

Contact: Allison F. Corkey [emailprotected] 520-329-1425

SOURCE Procyon Technologies LLC


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Procyon Technologies LLC and Novo Nordisk A/S to Collaborate on the Development of a Stem-Cell Based Therapy for Type 1 Diabetes - PRNewswire

Genetic engineering transformed stem cells into working mini-livers that extended the life of mice with liver disease – The Conversation US


Scientists have made progress growing human liver in the lab.

The challenge has been to direct stems cells to grow into a mature, functioning adult organ.

This study shows that stem cells can be programmed, using genetic engineering, to grow from immature cells into mature tissue.

When a tiny lab-grown liver was transplanted into mice with liver disease, it extended the lives of the sick animals.

Imagine if researchers could program stem cells, which have the potential to grow into all cell types in the body, so that they could generate an entire human organ. This would allow scientists to manufacture tissues for testing drugs and reduce the demand for transplant organs by having new ones grown directly from a patients cells.

Im a researcher working in this new field called synthetic biology focused on creating new biological parts and redesigning existing biological systems. In a new paper, my colleagues and I showed progress in one of the key challenges with lab-grown organs figuring out the genes necessary to produce the variety of mature cells needed to construct a functioning liver.

Induced pluripotent stem cells, a subgroup of stem cells, are capable of producing cells that can build entire organs in the human body. But they can do this job only if they receive the right quantity of growth signals at the right time from their environment. If this happens, they eventually give rise to different cell types that can assemble and mature in the form of human organs and tissues.

The tissues researchers generate from pluripotent stem cells can provide a unique source for personalized medicine from transplantation to novel drug discovery.

But unfortunately, synthetic tissues from stem cells are not always suitable for transplant or drug testing because they contain unwanted cells from other tissues, or lack the tissue maturity and a complete network of blood vessels necessary for bringing oxygen and nutrients needed to nurture an organ. That is why having a framework to assess whether these lab-grown cells and tissues are doing their job, and how to make them more like human organs, is critical.

Inspired by this challenge, I was determined to establish a synthetic biology method to read and write, or program, tissue development. I am trying to do this using the genetic language of stem cells, similar to what is used by nature to form human organs.

I am a researcher specializing in synthetic biology and biological engineering at the Pittsburgh Liver Research Center and McGowan Institute for Regenerative Medicine, where the goals are to use engineering approaches to analyze and build novel biological systems and solve human health problems. My lab combines synthetic biology and regenerative medicine in a new field that strives to replace, regrow or repair diseased organs or tissues.

I chose to focus on growing new human livers because this organ is vital for controlling most levels of chemicals like proteins or sugar in the blood. The liver also breaks down harmful chemicals and metabolizes many drugs in our body. But the liver tissue is also vulnerable and can be damaged and destroyed by many diseases, such as hepatitis or fatty liver disease. There is a shortage of donor organs, which limits liver transplantation.

To make synthetic organs and tissues, scientists need to be able to control stem cells so that they can form into different types of cells, such as liver cells and blood vessel cells. The goal is to mature these stem cells into miniorgans, or organoids, containing blood vessels and the correct adult cell types that would be found in a natural organ.

One way to orchestrate maturation of synthetic tissues is to determine the list of genes needed to induce a group of stem cells to grow, mature and evolve into a complete and functioning organ. To derive this list I worked with Patrick Cahan and Samira Kiani to first use computational analysis to identify genes involved in transforming a group of stem cells into a mature functioning liver. Then our team led by two of my students Jeremy Velazquez and Ryan LeGraw used genetic engineering to alter specific genes we had identified and used them to help build and mature human liver tissues from stem cells.

The tissue is grown from a layer of genetically engineered stem cells in a petri dish. The function of genetic programs together with nutrients is to orchestrate formation of liver organoids over the course of 15 to 17 days.

I and my colleagues first compared the active genes in fetal liver organoids we had grown in the lab with those in adult human livers using a computational analysis to get a list of genes needed for driving fetal liver organoids to mature into adult organs.

We then used genetic engineering to tweak genes and the resulting proteins that the stem cells needed to mature further toward an adult liver. In the course of about 17 days we generated tiny several millimeters in width but more mature liver tissues with a range of cells typically found in livers in the third trimester of human pregnancies.

Like a mature human liver, these synthetic livers were able to store, synthesize and metabolize nutrients. Though our lab-grown livers were small, we are hopeful that we can scale them up in the future. While they share many similar features with adult livers, they arent perfect and our team still has work to do. For example, we still need to improve the capacity of the liver tissue to metabolize a variety of drugs. We also need to make it safer and more efficacious for eventual application in humans.

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Our study demonstrates the ability of these lab livers to mature and develop a functional network of blood vessels in just two and a half weeks. We believe this approach can pave the path for the manufacture of other organs with vasculature via genetic programming.

The liver organoids provide several key features of an adult human liver such as production of key blood proteins and regulation of bile a chemical important for digestion of food.

When we implanted the lab-grown liver tissues into mice suffering from liver disease, it increased the life span. We named our organoids designer organoids, as they are generated via a genetic design.

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Genetic engineering transformed stem cells into working mini-livers that extended the life of mice with liver disease - The Conversation US