Category Archives: Stem Cell Doctors


Stem Cell Therapy Market Segmentation, Assessment and Growth Opportunities by Forecast 2025 – Scientect

Global Stem Cell Therapy Market: Overview

Also called regenerative medicine, stem cell therapy encourages the reparative response of damaged, diseased, or dysfunctional tissue via the use of stem cells and their derivatives. Replacing the practice of organ transplantations, stem cell therapies have eliminated the dependence on availability of donors. Bone marrow transplant is perhaps the most commonly employed stem cell therapy.

Osteoarthritis, cerebral palsy, heart failure, multiple sclerosis and even hearing loss could be treated using stem cell therapies. Doctors have successfully performed stem cell transplants that significantly aid patients fight cancers such as leukemia and other blood-related diseases.

Know the Growth Opportunities in Emerging Markets

Global Stem Cell Therapy Market: Key Trends

The key factors influencing the growth of the global stem cell therapy market are increasing funds in the development of new stem lines, the advent of advanced genomic procedures used in stem cell analysis, and greater emphasis on human embryonic stem cells. As the traditional organ transplantations are associated with limitations such as infection, rejection, and immunosuppression along with high reliance on organ donors, the demand for stem cell therapy is likely to soar. The growing deployment of stem cells in the treatment of wounds and damaged skin, scarring, and grafts is another prominent catalyst of the market.

On the contrary, inadequate infrastructural facilities coupled with ethical issues related to embryonic stem cells might impede the growth of the market. However, the ongoing research for the manipulation of stem cells from cord blood cells, bone marrow, and skin for the treatment of ailments including cardiovascular and diabetes will open up new doors for the advancement of the market.

Global Stem Cell Therapy Market: Market Potential

A number of new studies, research projects, and development of novel therapies have come forth in the global market for stem cell therapy. Several of these treatments are in the pipeline, while many others have received approvals by regulatory bodies.

In March 2017, Belgian biotech company TiGenix announced that its cardiac stem cell therapy, AlloCSC-01 has successfully reached its phase I/II with positive results. Subsequently, it has been approved by the U.S. FDA. If this therapy is well- received by the market, nearly 1.9 million AMI patients could be treated through this stem cell therapy.

Another significant development is the granting of a patent to Israel-based Kadimastem Ltd. for its novel stem-cell based technology to be used in the treatment of multiple sclerosis (MS) and other similar conditions of the nervous system. The companys technology used for producing supporting cells in the central nervous system, taken from human stem cells such as myelin-producing cells is also covered in the patent.

The regional analysis covers:

Order this Report TOC for Detailed Statistics

Global Stem Cell Therapy Market: Regional Outlook

The global market for stem cell therapy can be segmented into Asia Pacific, North America, Latin America, Europe, and the Middle East and Africa. North America emerged as the leading regional market, triggered by the rising incidence of chronic health conditions and government support. Europe also displays significant growth potential, as the benefits of this therapy are increasingly acknowledged.

Asia Pacific is slated for maximum growth, thanks to the massive patient pool, bulk of investments in stem cell therapy projects, and the increasing recognition of growth opportunities in countries such as China, Japan, and India by the leading market players.

Global Stem Cell Therapy Market: Competitive Analysis

Several firms are adopting strategies such as mergers and acquisitions, collaborations, and partnerships, apart from product development with a view to attain a strong foothold in the global market for stem cell therapy.

Some of the major companies operating in the global market for stem cell therapy are RTI Surgical, Inc., MEDIPOST Co., Ltd., Osiris Therapeutics, Inc., NuVasive, Inc., Pharmicell Co., Ltd., Anterogen Co., Ltd., JCR Pharmaceuticals Co., Ltd., and Holostem Terapie Avanzate S.r.l.

About TMR Research:

TMR Research is a premier provider of customized market research and consulting services to business entities keen on succeeding in todays supercharged economic climate. Armed with an experienced, dedicated, and dynamic team of analysts, we are redefining the way our clients conduct business by providing them with authoritative and trusted research studies in tune with the latest methodologies and market trends.

See more here:
Stem Cell Therapy Market Segmentation, Assessment and Growth Opportunities by Forecast 2025 - Scientect

How Far Have We Come In Treating COVID-19? – The Dispatch

Six months and counting into our coronavirus era, weve gotten much better at quantifying the fight against the disease. How well a country or state or county is faring is broken down into charts and scatterplots: Cases per capita and tests run per capita, hospital capacity and PPE availability, test positivity rate and test turnaround rate and case fatality rate.

Given that blizzard of data, its strange that one question remains so difficult to examine: How much better are we actually getting at fighting the disease?

Weve certainly gotten better at fighting the diseases spread. Everyone knows the drill by now: Masks, social distancing, washing your hands. Whats less obvious is how far weve progressed in the task of healing the very sick. Doctors and hospitals have tried to defeat the virus with a cornucopia of drugs and treatments. But in part because that effort has been so improvisational and eclectic, tracking its effectiveness over time remains a serious challenge.

The central fact of the current stage of the pandemic is this: Although new cases soared throughout July and August far above what we saw in April and May, daily COVID deaths have not climbed back to their late-April heights. That rate may continue to rise even with new cases descending againdeaths are, after all, a lagging indicatorbut the discrepancy is too sharp to ignore.

But this discrepancy itself does not necessarily indicate improved outcomes in COVID treatment, because other differences between the pandemics spring manifestation and its summer one affect the data too. The first wave of the pandemic was clustered primarily in New York and New Jersey, states that failed terribly at keeping the virus, which is most dangerous to the elderly, out of nursing homes. Further, our national testing operation was then in such a sorry state that it's hard to tell how much viral spread we missed the first time around.

I think most of the reduction in fatality rate across the country is accounted for by protecting vulnerable populations and by overtesting, Dr. Howard Forman, a Yale radiologist and health policy professor, told The Dispatch. So by testing more people who are healthier, we make ourselves look better.

This certainly isnt to say there hasnt been improvement in treating the virus. Clinicians will tell you in no uncertain terms theyve seen genuine progress. Gone is the desperate chaos of the early months, with their frantic smorgasbord of ad-hoc treatments and what-have-you-got-to-lose therapeutics; now doctors share a sort of anti-COVID folklore, wedding what solid clinical data exists with treatments that their compatriots around the country have found effective from hard experience.

Theres kind of two parts to the decision-making, said Dr. Megan Ranney, an emergency physician and professor at Brown University. Part one, and the part that we are all most comfortable with, is relying on peer-reviewed evidence. She rattled off a few examples: The antiviral drug remdesivir, which has been shown to reduce COVIDs recovery time; the anti-inflammatory dexamethasone, which can help soothe a berserk immune response to the virus; placing patients who are struggling to breathe on their stomachs rather than their backs.

The other side is the anecdote, the case series, the shared experiences of groups of physicians, she said. This was particularly prominent early in the pandemic when we really had nothing, and no guidance whatsoever. We saw Facebook and WhatsApp groups spring up, as well as email listservs, to try to allow real-time sharing of best practices and to allow people to compare what worked in one setting to what worked in another.

This loose-cannon treatment style helped doctors begin to catch up to a horribly powerful virus under incredibly bad circumstances, but it had its drawbacks too. Several doctors we spoke to brought up the example of hydroxychloroquine, a drug that showed some early promise in treating COVID patients but was ultimately shown in controlled clinical trials to be unhelpful in treating very sick patients. Ordinarily, that would be the end of the road for a given treatmentbut in this case, there were already a number of small case studies and doctors personal testimonies floating around with positive assessments of the drug and endorsements from President Donald Trump created a political constituency for its success. Such case studies and anecdotes are inherently less reliable than large-scale clinical trials, but their existence helped fan the flames of the bizarre conspiracy theories that cropped up around the drug.

And it isnt true that doctors are comfortable now, exactly. It can be hard to remember that were still in the very early days of the fight against a brand-new disease. Staying up to date on all the research coming out is a constant struggle of its own. Just this week, for instance, the FDA unexpectedly decided not to issue an emergency authorization for convalescent plasma, a promising treatment involving transfusions of antibody-rich blood serum donated by those who have already had COVID-19, suggesting the data was not yet strong enough to support it.

I still feel likemaybe not that Im working in the dark, but that maybe Im working with one eye closed, said Dr. Akino Yamashita, who has spent the year treating COVID patients in New Jersey and New York. Every day, its Oh, theres this new study, theres this new article. But does [one study] really change what I do when I see the next patient with this disease? Probably not.

Of the convalescent plasma story, she added: Certainly this goes along with the theme that although there are more treatment options available now, this is a constantly changing story.

Nor does the constant churn of new research focus only on better understanding the effects of the treatments already in circulation. Possible new treatments are working their way through the early stages of clinical trials all the time. Antibody treatments, both artificially manufactured and derived from animals, and stem cell therapies featuring cells known as MSCs (which stands, somewhat confusingly, for either medicinal signaling cells or mesenchymal stem cells)these are a few of the bleeding-edge projects that could potentially follow hydroxychloroquine and remdesivir into the public consciousness in the months to come.

Of course, this multiplication of possible treatments brings another set of challenges entirely.

The standard of treatment for a severe COVID-19 illness is changing, Dr. David Ingbar, a professor of medicine and pulmonary care expert at the University of Minnesota, told The Dispatch. Dr. Ingbar is currently heading up an early clinical trial into treating COVID with MSCs.

Now we believe dexamethasone works in certain populations at certain levels of illness, he said. We know that remdesivir has some benefit in terms of shortening the disease and maybe improving mortality. There are suggestions that, if given early, convalescent plasma might be beneficial. What we dont know very much about is, how do all of these treatments interact? Is it better to get one from column A, B, C, D, and E? Or are only certain combinations beneficial? So its a complicated field, and I think we all as clinicians want to help our patients as much as possible. But there is potential to do harm as well as good with all these treatments.

Presented like this, all this can seem like a dreary deluge of obstacles and complications. So its worth stating unequivocally: All of this is a pretty good problem to have. It may be a while before we can say for sure how far back our medical researchers and clinicians have beaten the virus already. But that fight gets a little less favorable to the virus, and a little more favorable to the humans, every day.

People forget that the longer you can wait to get COVIDhopefully well get a vaccine quickly, so well be able to protect us all from getting infectedbut if you get infected today, your chance of living through that infection is better than if you got infected in February or March, Dr. Ranney said. And I expect that to continue to improve. Its not a linear process. Its two steps forward, one step back in terms of that knowledge generation. So there are going to continue to be things that change, there are going to continue to be things that we think help and maybe dont. Thats normal in medicine. But overall, the trajectory is absolutely a positive and encouraging trajectory in terms of our ability to effectively treat and save lives of patients who are sick with COVID.

Photograph by Go Nakamura/Getty Images.

View post:
How Far Have We Come In Treating COVID-19? - The Dispatch

Congress must save lives and reauthorize cell transplant funding | Opinion – NorthJersey.com

Ronald Roth, Special to the USA TODAY NETWORK Published 4:02 a.m. ET Aug. 19, 2020

In May of this year, religious Jews celebrated Pesach Sheni. Pesach Sheni literally means Second Passover and marks the day in ancient times when someone who was unable to participate in Passover was given a second chance to observe the holiday.

The concept of second chances is a powerful one, not just in the Jewish faith, but in our society as a whole. Some times a second chance is more than just an opportunity for spiritual or personal redemption some times a second chance is literally a second chance at life itself.

I know this first-hand, because thanks to a bone marrow transplant, I was given a second chance at life.

In May 2015, I was diagnosed with a myelofibrosis, a rare form of blood cancer. For three years, I travelled back and forth to Mount Sinai Hospital for blood tests and medications that treated my illness. For those first three years I was fortunate to have few symptoms of my disease and I was able to continue my very busy schedule as rabbi of Fair Lawn Jewish Center/Congregation Bnai Israel.

Patrick Gustoso registers for the National Marrow Donor Program on March 28, 2017, at the Nutley Health Department. Organizers hope to find a match for resident Anne Rotonda.(Photo: Owen Proctor/NorthJersey.com)

In the spring of 2018, my medical fortunes changed dramatically as the disease began a more rapid progression. Since my diagnosis, I had known that a bone marrow transplant would be my only chance at a cure and in August 2018, I received that bone marrow transplant and a second chance at life.

After my transplant, I became an ambassador advocate for National Marrow Donor Program/Be The Match. I committed myself to using this second chance to help others who are in need of access to life-saving cellular therapy. I am not alone in this, all across the country men and women, doctors and patients, survivors and co-survivors, donors and recipients are working tirelessly to make sure live-saving materials are delivered to those in need.

We cant do it alone, we need help help from our elected officials in Washington.

The CW Bill Young Cell Transplantation program provides access to live-saving bone marrow, peripheral blood stem cell and cord blood transplants for patients living with one of more than 70 blood cancers or disorders for which a transplant is the only curative option.

Opinion: Pope John school must reconsider its plans to reopen

Community: NJ man who battled rare disease, depression and two heart transplants says every day is a miracle

Since its inception, the CW Bill Young Cell Transplantation Program has always enjoyed broad bipartisan support in both chambers of Congress. The program has been reauthorized by Congress every five years. It is up for re-authorization again this year and Congress should act swiftly to ensure the program remains able to fulfill its critical live-saving function.

Over the past 30 years, the National Marrow Donor Program (NMDP)/Be The Match has been entrusted by Congress to operate this critical program. During that time, more than 100,000 life-saving and life-extending transplants have been facilitated through the national registry.Many of these transplants took place here in New Jersey.

The Program is up for reauthorization again this summer and it is absolutely imperative that both chambers act swiftly to ensure that the program is reauthorized and that the critical life-saving work continues.The mission of the national registry to match patients and donors and ensure the timely transportation of lifesaving cellular products through the United States and around the world has been made even more challenging by COVID-19.Despite the pandemic, NMDP/Be The Match completed more transplants in June than in any single month in the programs history.This was only made possible by having the status of a federally authorized program that is recognized by other Federal, state, and local agencies and foreign governments.Should the programs authorization lapse at the end of September, there is concern that the program may not be able to operate as efficiently during the continuing public health emergency.

By reauthorizing the CW Bill Young Cell Transplantation Program, Congress can help give second chances to tens of thousands of men and women in need all across the country and here in New Jersey.

Rabbi Ronald Roth lives in Fair Lawn.

Read or Share this story: https://www.northjersey.com/story/opinion/2020/08/19/congress-must-reauthorize-cell-transplant-funds-opinion/3391146001/

Continue reading here:
Congress must save lives and reauthorize cell transplant funding | Opinion - NorthJersey.com

Each of Us Has the Ability to Save a Life; That Is the Power of Blood. – CSRwire.com

Aug. 21 /CSRwire/ - DKMS #PowerOfBlood

With Black donors currently only making up 7 percent of the registry, DKMS has launched the Power of Blood initiative with an aim to increase awareness of the topic in the Black community and encourage participation in the registry. For Emma Hall, a 10-year-old battling Aplastic Anemia, this initiative is a chance to share her story and change the fortunes of patients like her across the country.

Sitting in her backyard in Olathe, KS, Emma daydreams about going back to school, playing soccer with her friends, and - surprisingly for such a young child - simply not being tired all the time. Diagnosed with Aplastic Anemia (AA) in early 2019, Emma has been forced to give up her usual activities and routines, confronted by the reality that even sitting and reading a book can become an exhausting activity.

With their lives currently a blur of hospital visits and blood transfusions, Emmas doctors have told her family that the only cure for her disease is to receive a stem cell transplant from a matching donor. Though the search began with great hope, now over a year later Emma is still unable to find the match she needs to have a second chance at life. Could you be the difference?

Register now to save a life!

Visit link:
Each of Us Has the Ability to Save a Life; That Is the Power of Blood. - CSRwire.com

Viruses have big impacts on ecology and evolution as well as human health – The Economist

Aug 20th 2020

I The outsiders inside

HUMANS ARE lucky to live a hundred years. Oak trees may live a thousand; mayflies, in their adult form, a single day. But they are all alive in the same way. They are made up of cells which embody flows of energy and stores of information. Their metabolisms make use of that energy, be it from sunlight or food, to build new molecules and break down old ones, using mechanisms described in the genes they inherited and may, or may not, pass on.

It is this endlessly repeated, never quite perfect reproduction which explains why oak trees, humans, and every other plant, fungus or single-celled organism you have ever seen or felt the presence of are all alive in the same way. It is the most fundamental of all family resemblances. Go far enough up any creatures family tree and you will find an ancestor that sits in your family tree, too. Travel further and you will find what scientists call the last universal common ancestor, LUCA. It was not the first living thing. But it was the one which set the template for the life that exists today.

And then there are viruses. In viruses the link between metabolism and genes that binds together all life to which you are related, from bacteria to blue whales, is broken. Viral genes have no cells, no bodies, no metabolism of their own. The tiny particles, virions, in which those genes come packagedthe dot-studded disks of coronaviruses, the sinister, sinuous windings of Ebola, the bacteriophages with their science-fiction landing-legs that prey on microbesare entirely inanimate. An individual animal, or plant, embodies and maintains the restless metabolism that made it. A virion is just an arrangement of matter.

The virus is not the virion. The virus is a process, not a thing. It is truly alive only in the cells of others, a virtual organism running on borrowed hardware to produce more copies of its genome. Some bide their time, letting the cell they share the life of live on. Others immediately set about producing enough virions to split their hosts from stem to stern.

The virus has no plan or desire. The simplest purposes of the simplest lifeto maintain the difference between what is inside the cell and what is outside, to move towards one chemical or away from anotherare entirely beyond it. It copies itself in whatever way it does simply because it has copied itself that way before, in other cells, in other hosts.

That is why, asked whether viruses are alive, Eckard Wimmer, a chemist and biologist who works at the State University of New York, Stony Brook, offers a yes-and-no. Viruses, he says, alternate between nonliving and living phases. He should know. In 2002 he became the first person in the world to take an array of nonliving chemicals and build a virion from scratcha virion which was then able to get itself reproduced by infecting cells.

The fact that viruses have only a tenuous claim to being alive, though, hardly reduces their impact on things which are indubitably so. No other biological entities are as ubiquitous, and few as consequential. The number of copies of their genes to be found on Earth is beyond astronomical. There are hundreds of billions of stars in the Milky Way galaxy and a couple of trillion galaxies in the observable universe. The virions in the surface waters of any smallish sea handily outnumber all the stars in all the skies that science could ever speak of.

Back on Earth, viruses kill more living things than any other type of predator. They shape the balance of species in ecosystems ranging from those of the open ocean to that of the human bowel. They spur evolution, driving natural selection and allowing the swapping of genes.

They may have been responsible for some of the most important events in the history of life, from the appearance of complex multicellular organisms to the emergence of DNA as a preferred genetic material. The legacy they have left in the human genome helps produce placentas and may shape the development of the brain. For scientists seeking to understand lifes origin, they offer a route into the past separate from the one mapped by humans, oak trees and their kin. For scientists wanting to reprogram cells and mend metabolisms they offer inspirationand powerful tools.

II A lifestyle for genes

THE IDEA of a last universal common ancestor provides a plausible and helpful, if incomplete, answer to where humans, oak trees and their ilk come from. There is no such answer for viruses. Being a virus is not something which provides you with a place in a vast, coherent family tree. It is more like a lifestylea way of being which different genes have discovered independently at different times. Some viral lineages seem to have begun quite recently. Others have roots that comfortably predate LUCA itself.

Disparate origins are matched by disparate architectures for information storage and retrieval. In eukaryotescreatures, like humans, mushrooms and kelp, with complex cellsas in their simpler relatives, the bacteria and archaea, the genes that describe proteins are written in double-stranded DNA. When a particular protein is to be made, the DNA sequence of the relevant gene acts as a template for the creation of a complementary molecule made from another nucleic acid, RNA. This messenger RNA (mRNA) is what the cellular machinery tasked with translating genetic information into proteins uses in order to do so.

Because they, too, need to have proteins made to their specifications, viruses also need to produce mRNAs. But they are not restricted to using double-stranded DNA as a template. Viruses store their genes in a number of different ways, all of which require a different mechanism to produce mRNAs. In the early 1970s David Baltimore, one of the great figures of molecular biology, used these different approaches to divide the realm of viruses into seven separate classes (see diagram).

In four of these seven classes the viruses store their genes not in DNA but in RNA. Those of Baltimore group three use double strands of RNA. In Baltimore groups four and five the RNA is single-stranded; in group four the genome can be used directly as an mRNA; in group five it is the template from which mRNA must be made. In group sixthe retroviruses, which include HIVthe viral RNA is copied into DNA, which then provides a template for mRNAs.

Because uninfected cells only ever make RNA on the basis of a DNA template, RNA-based viruses need distinctive molecular mechanisms those cells lack. Those mechanisms provide medicine with targets for antiviral attacks. Many drugs against HIV take aim at the system that makes DNA copies of RNA templates. Remdesivir (Veklury), a drug which stymies the mechanism that the simpler RNA viruses use to recreate their RNA genomes, was originally developed to treat hepatitis C (group four) and subsequently tried against the Ebola virus (group five). It is now being used against SARS-CoV-2 (group four), the covid-19 virus.

Studies of the gene for that RNA-copying mechanism, RdRp, reveal just how confusing virus genealogy can be. Some viruses in groups three, four and five seem, on the basis of their RdRp-gene sequence, more closely related to members of one of the other groups than they are to all the other members of their own group. This may mean that quite closely related viruses can differ in the way they store their genomes; it may mean that the viruses concerned have swapped their RdRp genes. When two viruses infect the same cell at the same time such swaps are more or less compulsory. They are, among other things, one of the mechanisms by which viruses native to one species become able to infect another.

How do genes take on the viral lifestyle in the first place? There are two plausible mechanisms. Previously free-living creatures could give up metabolising and become parasitic, using other creatures cells as their reproductive stage. Alternatively genes allowed a certain amount of independence within one creature could have evolved the means to get into other creatures.

Living creatures contain various apparently independent bits of nucleic acid with an interest in reproducing themselves. The smallest, found exclusively in plants, are tiny rings of RNA called viroids, just a few hundred genetic letters long. Viroids replicate by hijacking a host enzyme that normally makes mRNAs. Once attached to a viroid ring, the enzyme whizzes round and round it, unable to stop, turning out a new copy of the viroid with each lap.

Viroids describe no proteins and do no good. Plasmidssomewhat larger loops of nucleic acid found in bacteriado contain genes, and the proteins they describe can be useful to their hosts. Plasmids are sometimes, therefore, regarded as detached parts of a bacterias genome. But that detachment provides a degree of autonomy. Plasmids can migrate between bacterial cells, not always of the same species. When they do so they can take genetic traits such as antibiotic resistance from their old host to their new one.

Recently, some plasmids have been implicated in what looks like a progression to true virus-hood. A genetic analysis by Mart Krupovic of the Pasteur Institute suggests that the Circular Rep-Encoding Single-Strand-DNA (CRESS-DNA) viruses, which infect bacteria, evolved from plasmids. He thinks that a DNA copy of the genes that another virus uses to create its virions, copied into a plasmid by chance, provided it with a way out of the cell. The analysis strongly suggests that CRESS-DNA viruses, previously seen as a pretty closely related group, have arisen from plasmids this way on three different occasions.

Such jailbreaks have probably been going on since very early on in the history of life. As soon as they began to metabolise, the first proto-organisms would have constituted a niche in which other parasitic creatures could have lived. And biology abhors a vacuum. No niche goes unfilled if it is fillable.

It is widely believed that much of the evolutionary period between the origin of life and the advent of LUCA was spent in an RNA worldone in which that versatile substance both stored information, as DNA now does, and catalysed chemical reactions, as proteins now do. Set alongside the fact that some viruses use RNA as a storage medium today, this strongly suggests that the first to adopt the viral lifestyle did so too. Patrick Forterre, an evolutionary biologist at the Pasteur Institute with a particular interest in viruses (and the man who first popularised the term LUCA) thinks that the RNA world was not just rife with viruses. He also thinks they may have brought about its end.

The difference between DNA and RNA is not large: just a small change to one of the letters used to store genetic information and a minor modification to the backbone to which these letters are stuck. And DNA is a more stable molecule in which to store lots of information. But that is in part because DNA is inert. An RNA-world organism which rewrote its genes into DNA would cripple its metabolism, because to do so would be to lose the catalytic properties its RNA provided.

An RNA-world virus, having no metabolism of its own to undermine, would have had no such constraints if shifting to DNA offered an advantage. Dr Forterre suggests that this advantage may have lain in DNAs imperviousness to attack. Host organisms today have all sorts of mechanisms for cutting up viral nucleic acids they dont like the look ofmechanisms which biotechnologists have been borrowing since the 1970s, most recently in the form of tools based on a bacterial defence called CRISPR. There is no reason to imagine that the RNA-world predecessors of todays cells did not have similar shears at their disposal. And a virus that made the leap to DNA would have been impervious to their blades.

Genes and the mechanisms they describe pass between viruses and hosts, as between viruses and viruses, all the time. Once some viruses had evolved ways of writing and copying DNA, their hosts would have been able to purloin them in order to make back-up copies of their RNA molecules. And so what began as a way of protecting viral genomes would have become the way life stores all its genesexcept for those of some recalcitrant, contrary viruses.

III The scythes of the seas

IT IS A general principle in biology that, although in terms of individual numbers herbivores outnumber carnivores, in terms of the number of species carnivores outnumber herbivores. Viruses, however, outnumber everything else in every way possible.

This makes sense. Though viruses can induce host behaviours that help them spreadsuch as coughingan inert virion boasts no behaviour of its own that helps it stalk its prey. It infects only that which it comes into contact with. This is a clear invitation to flood the zone. In 1999 Roger Hendrix, a virologist, suggested that a good rule of thumb might be ten virions for every living individual creature (the overwhelming majority of which are single-celled bacteria and archaea). Estimates of the number of such creatures on the planet come out in the region of 1029-1030. If the whole Earth were broken up into pebbles, and each of those pebbles smashed into tens of thousands of specks of grit, you would still have fewer pieces of grit than the world has virions. Measurements, as opposed to estimates, produce numbers almost as arresting. A litre of seawater may contain more than 100bn virions; a kilogram of dried soil perhaps a trillion.

Metagenomics, a part of biology that looks at all the nucleic acid in a given sample to get a sense of the range of life forms within it, reveals that these tiny throngs are highly diverse. A metagenomic analysis of two surveys of ocean life, the Tara Oceans and Malaspina missions, by Ahmed Zayed of Ohio State University, found evidence of 200,000 different species of virus. These diverse species play an enormous role in the ecology of the oceans.

A litre of seawater may contain 100bn virions; a kilogram of dried soil perhaps a trillion

On land, most of the photosynthesis which provides the biomass and energy needed for life takes place in plants. In the oceans, it is overwhelmingly the business of various sorts of bacteria and algae collectively known as phytoplankton. These creatures reproduce at a terrific rate, and viruses kill them at a terrific rate, too. According to work by Curtis Suttle of the University of British Columbia, bacterial phytoplankton typically last less than a week before being killed by viruses.

This increases the overall productivity of the oceans by helping bacteria recycle organic matter (it is easier for one cell to use the contents of another if a virus helpfully lets them free). It also goes some way towards explaining what the great mid-20th-century ecologist G. Evelyn Hutchinson called the paradox of the plankton. Given the limited nature of the resources that single-celled plankton need, you would expect a few species particularly well adapted to their use to dominate the ecosystem. Instead, the plankton display great variety. This may well be because whenever a particular form of plankton becomes dominant, its viruses expand with it, gnawing away at its comparative success.

It is also possible that this endless dance of death between viruses and microbes sets the stage for one of evolutions great leaps forward. Many forms of single-celled plankton have molecular mechanisms that allow them to kill themselves. They are presumably used when one cells sacrifice allows its sister cellswhich are genetically identicalto survive. One circumstance in which such sacrifice seems to make sense is when a cell is attacked by a virus. If the infected cell can kill itself quickly (a process called apoptosis) it can limit the number of virions the virus is able to make. This lessens the chances that other related cells nearby will die. Some bacteria have been shown to use this strategy; many other microbes are suspected of it.

There is another situation where self-sacrifice is becoming conduct for a cell: when it is part of a multicellular organism. As such organisms grow, cells that were once useful to them become redundant; they have to be got rid of. Eugene Koonin of Americas National Institutes of Health and his colleagues have explored the idea that virus-thwarting self-sacrifice and complexity-permitting self-sacrifice may be related, with the latter descended from the former. Dr Koonins model also suggests that the closer the cells are clustered together, the more likely this act of self-sacrifice is to have beneficial consequences.

For such profound propinquity, move from the free-flowing oceans to the more structured world of soil, where potential self-sacrificers can nestle next to each other. Its structure makes soil harder to sift for genes than water is. But last year Mary Firestone of the University of California, Berkeley, and her colleagues used metagenomics to count 3,884 new viral species in a patch of Californian grassland. That is undoubtedly an underestimate of the total diversity; their technique could see only viruses with RNA genomes, thus missing, among other things, most bacteriophages.

Metagenomics can also be applied to biological samples, such as bat guano in which it picks up viruses from both the bats and their food. But for the most part the finding of animal viruses requires more specific sampling. Over the course of the 2010s PREDICT, an American-government project aimed at finding animal viruses, gathered over 160,000 animal and human tissue samples from 35 countries and discovered 949 novel viruses.

The people who put together PREDICT now have grander plans. They want a Global Virome Project to track down all the viruses native to the worlds 7,400 species of mammals and waterfowlthe reservoirs most likely to harbour viruses capable of making the leap into human beings. In accordance with the more-predator-species-than-prey rule they expect such an effort would find about 1.5m viruses, of which around 700,000 might be able to infect humans. A planning meeting in 2018 suggested that such an undertaking might take ten years and cost $4bn. It looked like a lot of money then. Today those arguing for a system that can provide advance warning of the next pandemic make it sound pretty cheap.

IV Leaving their mark

THE TOLL which viruses have exacted throughout history suggests that they have left their mark on the human genome: things that kill people off in large numbers are powerful agents of natural selection. In 2016 David Enard, then at Stanford University and now at the University of Arizona, made a stab at showing just how much of the genome had been thus affected.

He and his colleagues started by identifying almost 10,000 proteins that seemed to be produced in all the mammals that had had their genomes sequenced up to that point. They then made a painstaking search of the scientific literature looking for proteins that had been shown to interact with viruses in some way or other. About 1,300 of the 10,000 turned up. About one in five of these proteins was connected to the immune system, and thus could be seen as having a professional interest in viral interaction. The others appeared to be proteins which the virus made use of in its attack on the host. The two cell-surface proteins that SARS-CoV-2 uses to make contact with its target cells and inveigle its way into them would fit into this category.

The researchers then compared the human versions of the genes for their 10,000 proteins with those in other mammals, and applied a statistical technique that distinguishes changes that have no real impact from the sort of changes which natural selection finds helpful and thus tries to keep. Genes for virus-associated proteins turned out to be evolutionary hotspots: 30% of all the adaptive change was seen in the genes for the 13% of the proteins which interacted with viruses. As quickly as viruses learn to recognise and subvert such proteins, hosts must learn to modify them.

A couple of years later, working with Dmitri Petrov at Stanford, Dr Enard showed that modern humans have borrowed some of these evolutionary responses to viruses from their nearest relatives. Around 2-3% of the DNA in an average European genome has Neanderthal origins, a result of interbreeding 50,000 to 30,000 years ago. For these genes to have persisted they must be doing something usefulotherwise natural selection would have removed them. Dr Enard and Dr Petrov found that a disproportionate number described virus-interacting proteins; of the bequests humans received from their now vanished relatives, ways to stay ahead of viruses seem to have been among the most important.

Viruses do not just shape the human genome through natural selection, though. They also insert themselves into it. At least a twelfth of the DNA in the human genome is derived from viruses; by some measures the total could be as high as a quarter.

Retroviruses like HIV are called retro because they do things backwards. Where cellular organisms make their RNA from DNA templates, retroviruses do the reverse, making DNA copies of their RNA genomes. The host cell obligingly makes these copies into double-stranded DNA which can be stitched into its own genome. If this happens in a cell destined to give rise to eggs or sperm, the viral genes are passed from parent to offspring, and on down the generations. Such integrated viral sequences, known as endogenous retroviruses (ERVs), account for 8% of the human genome.

This is another example of the way the same viral trick can be discovered a number of times. Many bacteriophages are also able to stitch copies of their genome into their hosts DNA, staying dormant, or temperate, for generations. If the cell is doing well and reproducing regularly, this quiescence is a good way for the viral genes to make more copies of themselves. When a virus senses that its easy ride may be coming to an end, thoughfor example, if the cell it is in shows signs of stressit will abandon ship. What was latent becomes lytic as the viral genes produce a sufficient number of virions to tear the host apart.

Though some of their genes are associated with cancers, in humans ERVs do not burst back into action in later generations. Instead they have proved useful resources of genetic novelty. In the most celebrated example, at least ten different mammalian lineages make use of a retroviral gene for one of their most distinctively mammalian activities: building a placenta.

The placenta is a unique organ because it requires cells from the mother and the fetus to work together in order to pass oxygen and sustenance in one direction and carbon dioxide and waste in the other. One way this intimacy is achieved safely is through the creation of a tissue in which the membranes between cells are broken down to form a continuous sheet of cellular material.

The protein that allows new cells to merge themselves with this layer, syncytin-1, was originally used by retroviruses to join the external membranes of their virions to the external membranes of cells, thus gaining entry for the viral proteins and nucleic acids. Not only have different sorts of mammals co-opted this membrane-merging trickother creatures have made use of it, too. The mabuya, a long-tailed skink which unusually for a lizard nurtures its young within its body, employs a retroviral syncytin protein to produce a mammalian-looking placenta. The most recent shared ancestor of mabuyas and mammals died out 80m years before the first dinosaur saw the light of day, but both have found the same way to make use of the viral gene.

This is not the only way that animals make use of their ERVs. Evidence has begun to accumulate that genetic sequences derived from ERVs are quite frequently used to regulate the activity of genes of more conventional origin. In particular, RNA molecules transcribed from an ERV called HERV-K play a crucial role in providing the stem cells found in embryos with their pluripotencythe ability to create specialised daughter cells of various different types. Unfortunately, when expressed in adults HERV-K can also be responsible for cancers of the testes.

As well as containing lots of semi-decrepit retroviruses that can be stripped for parts, the human genome also holds a great many copies of a retrotransposon called LINE-1. This a piece of DNA with a surprisingly virus-like way of life; it is thought by some biologists to have, like ERVs, a viral origin. In its full form, LINE-1 is a 6,000-letter sequence of DNA which describes a reverse transcriptase of the sort that retroviruses use to make DNA from their RNA genomes. When LINE-1 is transcribed into an mRNA and that mRNA subsequently translated to make proteins, the reverse transcriptase thus created immediately sets to work on the mRNA used to create it, using it as the template for a new piece of DNA which is then inserted back into the genome. That new piece of DNA is in principle identical to the piece that acted as the mRNAs original template. The LINE-1 element has made a copy of itself.

In the 100m years or so that this has been going on in humans and the species from which they are descended the LINE-1 element has managed to pepper the genome with a staggering 500,000 copies of itself. All told, 17% of the human genome is taken up by these copiestwice as much as by the ERVs.

Most of the copies are severely truncated and incapable of copying themselves further. But some still have the knack, and this capability may be being put to good use. Fred Gage and his colleagues at the Salk Institute for Biological Studies, in San Diego, argue that LINE-1 elements have an important role in the development of the brain. In 2005 Dr Gage discovered that in mouse embryosspecifically, in the brains of those embryosabout 3,000 LINE-1 elements are still able to operate as retrotransposons, putting new copies of themselves into the genome of a cell and thus of all its descendants.

Brains develop through proliferation followed by pruning. First, nerve cells multiply pell-mell; then the cell-suicide process that makes complex life possible prunes them back in a way that looks a lot like natural selection. Dr Gage suspects that the movement of LINE-1 transposons provides the variety in the cell population needed for this selection process. Choosing between cells with LINE-1 in different places, he thinks, could be a key part of the process from which the eventual neural architecture emerges. What is true in mice is, as he showed in 2009, true in humans, too. He is currently developing a technique for looking at the process in detail by comparing, post mortem, the genomes of different brain cells from single individuals to see if their LINE-1 patterns vary in the ways that his theory would predict.

V Promised lands

HUMAN EVOLUTION may have used viral genes to make big-brained live-born life possible; but viral evolution has used them to kill off those big brains on a scale that is easily forgotten. Compare the toll to that of war. In the 20th century, the bloodiest in human history, somewhere between 100m and 200m people died as a result of warfare. The number killed by measles was somewhere in the same range; the number who died of influenza probably towards the top of it; and the number killed by smallpox300m-500mwell beyond it. That is why the eradication of smallpox from the wild, achieved in 1979 by a globally co-ordinated set of vaccination campaigns, stands as one of the all-time-great humanitarian triumphs.

Other eradications should eventually follow. Even in their absence, vaccination has led to a steep decline in viral deaths. But viruses against which there is no vaccine, either because they are very new, like SARS-CoV-2, or peculiarly sneaky, like HIV, can still kill millions.

Reducing those tolls is a vital aim both for research and for public-health policy. Understandably, a far lower priority is put on the benefits that viruses can bring. This is mostly because they are as yet much less dramatic. They are also much less well understood.

The viruses most prevalent in the human body are not those which infect human cells. They are those which infect the bacteria that live on the bodys surfaces, internal and external. The average human microbiome harbours perhaps 100trn of these bacteria. And where there are bacteria, there are bacteriophages shaping their population.

The microbiome is vital for good health; when it goes wrong it can mess up a lot else. Gut bacteria seem to have a role in maintaining, and possibly also causing, obesity in the well-fed and, conversely, in tipping the poorly fed into a form of malnutrition called kwashiorkor. Ill-regulated gut bacteria have also been linked, if not always conclusively, with diabetes, heart disease, cancers, depression and autism. In light of all this, the question who guards the bacterial guardians? is starting to be asked.

The viruses that prey on the bacteria are an obvious answer. Because the health of their hosts hostthe possessor of the gut they find themselves inmatters to these phages, they have an interest in keeping the microbiome balanced. Unbalanced microbiomes allow pathogens to get a foothold. This may explain a curious detail of a therapy now being used as a treatment of last resort against Clostridium difficile, a bacterium that causes life-threatening dysentery. The therapy in question uses a transfusion of faecal matter, with its attendant microbes, from a healthy individual to reboot the patients microbiome. Such transplants, it appears, are more likely to succeed if their phage population is particularly diverse.

Medicine is a very long way from being able to use phages to fine-tune the microbiome. But if a way of doing so is found, it will not in itself be a revolution. Attempts to use phages to promote human health go back to their discovery in 1917, by Flix dHrelle, a French microbiologist, though those early attempts at therapy were not looking to restore balance and harmony. On the basis that the enemy of my enemy is my friend, doctors simply treated bacterial infections with phages thought likely to kill the bacteria.

The arrival of antibiotics saw phage therapy abandoned in most places, though it persisted in the Soviet Union and its satellites. Various biotechnology companies think they may now be able to revive the traditionand make it more effective. One option is to remove the bits of the viral genome that let phages settle down to a temperate life in a bacterial genome, leaving them no option but to keep on killing. Another is to write their genes in ways that avoid the defences with which bacteria slice up foreign DNA.

The hope is that phage therapy will become a backup in difficult cases, such as infection with antibiotic-resistant bugs. There have been a couple of well-publicised one-off successes outside phage therapys post-Soviet homelands. In 2016 Tom Patterson, a researcher at the University of California, San Diego, was successfully treated for an antibiotic-resistant bacterial infection with specially selected (but un-engineered) phages. In 2018 Graham Hatfull of the University of Pittsburgh used a mixture of phages, some engineered so as to be incapable of temperance, to treat a 16-year-old British girl who had a bad bacterial infection after a lung transplant. Clinical trials are now getting under way for phage treatments aimed at urinary-tract infections caused by Escherichia coli, Staphylococcus aureus infections that can lead to sepsis and Pseudomonas aeruginosa infections that cause complications in people who have cystic fibrosis.

Viruses which attack bacteria are not the only ones genetic engineers have their eyes on. Engineered viruses are of increasing interest to vaccine-makers, to cancer researchers and to those who want to treat diseases by either adding new genes to the genome or disabling faulty ones. If you want to get a gene into a specific type of cell, a virion that recognises something about such cells may often prove a good tool.

The vaccine used to contain the Ebola outbreak in the Democratic Republic of Congo over the past two years was made by engineering Indiana vesiculovirus, which infects humans but cannot reproduce in them, so that it expresses a protein found on the surface of the Ebola virus; thus primed, the immune system responds to Ebola much more effectively. The World Health Organisations current list of 29 covid-19 vaccines in clinical trials features six versions of other viruses engineered to look a bit like SARS-CoV-2. One is based on a strain of measles that has long been used as a vaccine against that disease.

Viruses engineered to engender immunity against pathogens, to kill cancer cells or to encourage the immune system to attack them, or to deliver needed genes to faulty cells all seem likely to find their way into health care. Other engineered viruses are more worrying. One way to understand how viruses spread and kill is to try and make particularly virulent ones. In 2005, for example, Terrence Tumpey of Americas Centres for Disease Control and Prevention and his colleagues tried to understand the deadliness of the influenza virus responsible for the pandemic of 1918-20 by taking a more benign strain, adding what seemed to be distinctive about the deadlier one and trying out the result on mice. It was every bit as deadly as the original, wholly natural version had been.

The use of engineered pathogens as weapons of war is of dubious utility, completely illegal and repugnant to almost all

Because such gain of function research could, if ill-conceived or poorly implemented, do terrible damage, it requires careful monitoring. And although the use of engineered pathogens as weapons of war is of dubious utilitysuch weapons are hard to aim and hard to stand down, and it is not easy to know how much damage they have doneas well as being completely illegal and repugnant to almost all, such possibilities will and should remain a matter of global concern.

Information which, for billions of years, has only ever come into its own within infected cells can now be inspected on computer screens and rewritten at will. The power that brings is sobering. It marks a change in the history of both viruses and peoplea change which is perhaps as important as any of those made by modern biology. It is constraining a small part of the viral world in a way which, so far, has been to peoples benefit. It is revealing that worlds further reaches in a way which cannot but engender awe.

Editors note: Some of our covid-19 coverage is free for readers of The Economist Today, our daily newsletter. For more stories and our pandemic tracker, see our hub

This article appeared in the Essay section of the print edition under the headline "The outsiders inside"

See the original post here:
Viruses have big impacts on ecology and evolution as well as human health - The Economist

Uncles incredible tribute to niece who died from the good cancer’ – Yahoo News Australia

An uncles poignant and loving tribute to his niece after she died following a seven-year battle with Hodgkin Lymphoma has led to life-saving stem cell and bone marrow donations.

Dr Melissa Baker, a single mum of two and forensic pathologist from Melbourne, died on January 16 - just two days after her 45th birthday.

In her memory, Melissas beloved uncle Max Tomlinson placed her photo and information about how to become a stem cell donor on his rear window in the hope of carrying on her hard work.

In memory of my beautiful niece Dr Melissa Baker. You can save a life, dont let Melissas be in vain. Order your swab kit now. Ideally men aged 18 to 45 with diverse backgrounds needed urgently. Order your kit now urthecure.com.au, it reads in white marker pen.

Melissas beloved uncle, Max Tomlinson, placed her photo and information about how to become a stem cell donor on his car's rear window. Source: Facebook

Melissas sister, Jenni Baker, recently posted a picture of Mr Tomlinsons car on Facebook while thanking a member of the public who tucked a yellow flower under his windshield wiper.

Melissa, whos kids are 13 and 8, waited for a bone marrow match for years after an initial six-month round of chemotherapy didnt work, Jenni, a Melbourne police officer, told Yahoo News Australia on Friday.

She underwent a bone marrow transplant using her own stem cells but it almost killed her when she developed a lung infection, her sister said.

Doctors told the 45-year-old, who had since developed cancer of the bone marrow as a result of the chemotherapy, she desperately needed a donor and so she began advocating for UR The Cure.

The volunteer-run charity works with the Australian Bone Marrow Donor Registry (ABMDR) to increase the number of donors especially middle-aged people of diverse backgrounds.

Melissa, whos kids are 13 and 8, waited for a bone marrow match for years after an initial six-month round of chemotherapy didnt work. Source: Facebook

Reluctantly, in November 2019, she underwent a more risky half-match stem cell transplant where I was her donor, Jenni said.

The odds werent great but she had no choice.

Tragically, after 58 days in the hospital, most of which she spent on a ventilator, Melissa died on January 16.

Jennis Facebook post about her uncles tribute has garnered more than 2,500 likes and hundreds of comments, many of which are people who said they had since signed up to be a stem cell donor.

I was a bone marrow donor for my dad. Unfortunately he passed just four months after the donation. I would do it again in a heartbeat for anyone who needed it, one woman wrote.

Beautiful! Tell your uncle I just ordered my kit! another said.

A woman named Amanda also commented, revealing she had been one of Melissas nurses.

I dont know if you remember me. I am one of the nurses who took care of your sister in the ICU. I always admired how much support Melissa had from you and your sister. Her life is definitely not in vain and the love she had from you all was so strong, she wrote.

Melissa Baker underwent a bone marrow transplant using her own stem cells but it almost killed her when she developed a lung infection. Source: Facebook

Story continues

Jenni said Melissa never thought in her wildest dreams this would happen and had at one point thought the cancer would be a battle she would have to fight throughout her life.

The 47-year-old police officer told Yahoo News Australia Melissa became upset when she was often told she had the good cancer because of Hodgkins higher success rate.

She was so mad about it she even made a blog called I Got the Good Cancer documenting her struggles and treatments.

And then everything bad that could have happened, happened, Jenni said.

Jenni (right) and Melissa (left) are pictured together in front of Parliament House. Source: Facebook

The mum-of-two spent last Christmas intubated and sedated in hospital but was able to squeeze her childrens hands when they came to visit.

When the tubes came out on Boxing Day, Melissa mumbled to Jenni, Im scared. This is really scary.

They were the last words Melissa said.

Just two days later Melissa was ventilated again until the tubes were removed on January 14 - her birthday - after deciding it was too cruel.

Fifty-two hours later she passed surrounded by her parents, siblings and children.

Do you have a story tip? Email:newsroomau@yahoonews.com.

You can also follow us onFacebook,InstagramandTwitterand download the Yahoo News app from theApp StoreorGoogle Play.

More:
Uncles incredible tribute to niece who died from the good cancer' - Yahoo News Australia

CurePSP Partners With Rainwater to Support Pioneering Neurodegeneration Research – Yahoo Finance

Study will use miniature brains cultivated from stem cells to study protein mutations

CurePSP has awarded a $100,000 grant for a potentially breakthrough study that will employ 3D organoid models that have been shown to produce structures similar to those seen in the human brain to understand the effects of protein mutations that are associated with neurodegeneration.

The grant will assist the Tau Consortium Stem Cell Group, created and supported by the Rainwater Charitable Foundation, in helping to extend the scope of this multidisciplinary and multiyear study. The funding has been provided through generous donations from Frank Semcer, Sr., in honor of his wife Mary Jane Semcer; and from F. Jackson Phillips, in honor of his wife, Linda A. Phillips. Both women have received diagnoses of progressive supranuclear palsy (PSP), a rare form of neurodegeneration that involves pathological accumulation of the tau protein, a type of naturally occurring protein in the brain. CurePSPs grant was made under the auspices of the Prime of Life Brain Initiative, a collaborative venture with Rainwater to advance research into neurodegeneration.

The CurePSP grant will support research conducted by investigators of the Tau Consortium Stem Cell Group including Dr. Sally Temple of the Neural Stem Cell Institute, Dr. Alison Goate of the Ichan School of Medicine at Mount Sinai, Dr. Justin Ichida of the University of Southern California, Dr. Celeste Karch of Washington University, and Dr. Martin Kampmann of the University of California San Francisco.

Sometimes called "disease in a dish," 3D organoids are grown from skin-derived stem cells of patients and healthy individuals. Studies using these patient derived cell cultures aim to avoid some of the challenges and limitations of using animal models in research, including lack of construct validity of data (the degree to which data measures what it claims to be measuring) collected from animal studies when projected to humans. Since the first development of 3D neuronal cell cultures (organoids) less than 10 years ago, efforts have focused on the standardization and reproducibility of organoids protocols. Even more recently, genetic engineering techniques (CRISPR) and sequencing methods such as single-cell RNA sequencing have enabled greater robustness and a deeper understanding of these personalized human-derived disease models.

Dr. Kristophe J. Diaz, Vice President Scientific Affairs for CurePSP, said, "The use of human-derived disease models as opposed to reliance solely on animal models is a crucial step in the study of complex neurodegenerative disorders. We are thrilled to participate in this landmark study with the Tau Consortium and thank the Semcers and Phillips for their support."

Patrick Brannelly, Managing Director of the Tau Consortium, added, "PSP has become a key focus in the study of neurodegeneration for several of the worlds best academic institutions. CurePSPs collaboration in this study is greatly valued. We look forward to our continued partnership through the Prime of Life Brain Initiative."

CurePSP was an early supporter of the work of the Neural Stem Cell Institute with grants to Dr. David Butler for his work in developing therapeutic agents to prevent tau protein accumulation with novel antibody-based reagents called intrabodies and to Dr. Mo Liu for her pioneering work in developing PSP organoids.

About CurePSP

CurePSP is the nonprofit advocacy organization focused on progressive supranuclear palsy, corticobasal degeneration, and other prime of life neurodegenerative diseases, a spectrum of fatal brain disorders that often strike during a persons most productive and rewarding years. Currently, there is no effective treatment or cure for these diseases, which affect more than 150,000 people in the U.S. alone. Since it was founded in 1990, CurePSP has funded nearly 200 research studies and is the leading source of information and support for patients and their families, other caregivers, researchers and doctors, and allied healthcare professionals. CurePSP is based in New York City. Please visit http://www.curepsp.org for more information.

About the Rainwater Charitable Foundation

The Rainwater Charitable Foundation was created in the early 1990s by renowned investor and philanthropist Richard E. Rainwater. The foundation supports a range of different programs in K-12 education, medical research, and other worthy causes. In order to deliver on its mission to accelerate the development of new diagnostics and treatments for tau-related neurodegenerative disorders, the Rainwater Charitable Foundation Medical Research team manages the Tau Consortium and the Rainwater Prize Program for advances in neurodegenerative disease research. With more than $100 million invested to date, Rainwater support has helped to advance eight treatments into human trials. For more information, please visit http://www.tauconsortium.org.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200811005112/en/

Contacts

David Kemp kemp@curepsp.org 802-734-1185

Visit link:
CurePSP Partners With Rainwater to Support Pioneering Neurodegeneration Research - Yahoo Finance

A breakthrough drug saved the lives of every critical coronavirus patient who took it – BGR

We may be wrong to expect a miracle cure for COVID-19. Thats because it might not be just one breakthrough drug that delivers the miracle, which is the prevention of life-threatening symptoms. We may end up with several new drugs for COVID-19 that would work independently or in tandem with others to save the lives of patients who develop complications.

Recent studies showed that several therapies can reduce COVID-19 mortality to some degree. But neither remdesivir nor dexamethasone can prevent deaths completely. This is where narsoplimab may come to the rescue, a monoclonal antibody drug thats not supposed to block the coronavirus from infecting cells like other monoclonal therapies being tested for COVID-19. Instead, this drug protects the integrity of blood vessels and helps prevent clotting. Narsoplimab saved the lives of every one of the critical COVID-19 patients who were included in a limited trial in Bergamo, Italy, one of the early epicenters of the European COVID-19 epidemic.

Made by Omeros, narsoplimab was initially devised for other medical conditions that cause damage to the blood vessels, but doctors involved with previous clinical testing thought the drug might also work on COVID-19 cases. Dr. Alessandro Rambaldi from the Papa Giovanni XXIII hospital in Bergamo was involved in a stem-cell trial for narsoplimab before COVID-19, Omeros explained in a press release. The doctor asked Omeros to offer the drug to six COVID-19 patients in critical condition, who needed mechanical ventilators to breathe.

These patients developed Acute Respiratory Distress Syndrome (ARDS), one of the COVID-19 complications that can lead to death. The patients were aged 47 to 63, all had comorbidities, and five of them were men. The researchers administered narsoplimab 48 hours after mechanical ventilation had started, and then all the patients received the drug twice a week for two to four weeks.

All the patients recovered and survived COVID-19, but two of them were hospitalized for about a month after narsoplimab treatment began. Another spent some three months in the hospital after the first dose.

From the study: Clinical outcome of patients treated with narsoplimab. The bar colors indicate the different oxygen support (CPAP, yellow; mechanical ventilation with intubation, red; non-rebreather oxygen mask, green; low flow oxygen by nasal cannula, light green; room air, blue). Narsoplimab doses are marked by blue arrows. Black circles indicate the beginning of steroid treatment (day +2 in patients #4 and #6, day +4 in patient #5 and day +10 in patient #2 and #3). Black asterisk denotes discharge from hospital. CPAP=continuous positive airway pressure; NRM=non-rebreather oxygen mask; VM=Venturi mask; TEP=pulmonary thromboembolism. Image source: Omeros via ScienceDirect

Endothelial injury appears to be caused by direct viral infection in COVID-19, the researchers wrote in the paper, and that damage can be measured with the help of several parameters. The doctors measured those parameters, which proved the drug reduced the cellular damage inside the blood vessels. Narsoplimab also prevents blood clotting, the researchers say, and it might prevent so-called cytokine storms, the overreaction of a patients immune system that can be fatal.

The temporal improvement of IL-6 and IL-8 with narsoplimab treatment suggests that lectin pathway activation may precede cytokine elevation in COVID-19 and that lectin pathway inhibition has a beneficial effect on the cytokine storm described in patients with COVID-19 infection

The researchers say the drug was well tolerated and there were no side-effects. The study also reveals that five of the six patients received steroids at the hospital, with two of them getting the drug after their condition improved significantly (patients 2 and 3 in the graphic above).

One downside of the study is that it wasnt a double-blind, placebo-controlled, randomized trial. But the researchers compared the six patients with two control groups that met similar criteria and characteristics, which showed mortality rates of 32% and 53%.

The patients that we treated with narsoplimab were critically ill, and the uniformly successful outcomes were truly impressive,Rambaldi said. Also of importance in this terribly sick population studied, the drug was well tolerated, showing no adverse effects. The pathophysiology of COVID-19 appears to be consistent with that of stem cell transplant-associated TMA, and the mechanism of the lectin pathway inhibitor narsoplimab looks to be well suited to treat the often-lethal manifestations of both disorders. The outcomes in these six patients provide further evidence of the potential role of narsoplimab in treating diseases caused by endothelial damage.

The drug is under review for federal approval, The Seattle Times reports, and its considered for federal support under the Operation Warp Speed initiative.

More research will be required before the drug can be widely used to treat severe COVID-19 cases. If the drug can indeed deliver the same response in most patients who develop complications, it could potentially save a lot of lives. That said, its unclear how easy it is to manufacture or how much it will cost.

The Omeros study will be published in the peer-reviewed journal Immunology and is available in pre-print form at this link.

Chris Smith started writing about gadgets as a hobby, and before he knew it he was sharing his views on tech stuff with readers around the world. Whenever he's not writing about gadgets he miserably fails to stay away from them, although he desperately tries. But that's not necessarily a bad thing.

The rest is here:
A breakthrough drug saved the lives of every critical coronavirus patient who took it - BGR

BeyondSpring Initiates Expanded Access Program with Plinabulin for Patients Suffering from CIN in the U.S. – Stockhouse

- NCCN Guideline Updates Highlight Need for Maximum CIN Prevention and Resource Allocation for COVID-19 Patients -

- First Patient Dosed in the U.S. Avoided Grade 4 Neutropenia in Cycle 2 with Plinabulin and Pegfilgrastim, Despite Experiencing Grade 4 Neutropenia in Cycle 1 with Pegfilgrastim Alone -

NEW YORK, Aug. 11, 2020 (GLOBE NEWSWIRE) -- BeyondSpring Inc. (the Company” or BeyondSpring”) (NASDAQ: BYSI), a global biopharmaceutical company focused on the development of innovative immuno-oncology cancer therapies, today announced that the Company has initiated an Expanded Access Program (EAP) to enable doctors across the U.S. to use BeyondSpring’s late-stage asset, Plinabulin, to prevent cancer patients’ chemotherapy-induced neutropenia (CIN), both alone and in combination with G-CSFs (the current standard of care), during the COVID-19 pandemic. Dr. Emad Ibrahim enrolled the first patient at Redlands Community Hospital in California on July 28, 2020.

In response to COVID-19, the National Comprehensive Cancer Network (NCCN) recently updated its treatment guidelines for the prophylaxis of CIN, with the objective of preserving hospital and ER resources for COVID-19 patients and maximizing protection for cancer patients against CIN development. This is designed to help necessitate healthcare interactions, and avoidance of hospital / ER visits will also minimize cancer patients’ risk of contracting COVID-19. In light of these NCCN guideline updates, BeyondSpring initiated an Expanded Access Program to enable the use of Plinabulin by oncologists to better protect cancer patients against CIN with the use of myelosuppressive chemotherapies under the current COVID-19 challenges.

Dr. Emad Ibrahim enrolled the first patient under this EAP at Redlands Community Hospital in California:

The recent updates to the NCCN guidelines aim to protect cancer patients from developing CIN in the most effective way possible and enable the healthcare system to reserve precious resources for COVID-19 patients,” said Ramon Mohanlal, BeyondSpring’s Chief Medical Officer and Executive Vice President, Research and Development. In our CIN studies, Plinabulin, in combination with Pegfilgrastim, provided superior protection against CIN, compared to the standard of care alone. The observation in this first EAP patient who completely avoided Grade 4 CIN when given Plinabulin and Pegfilgrastim is a significant achievement for us. At BeyondSpring, we strive to play our part in serving patients and healthcare providers to the highest degree while working through the many challenges imposed by COVID-19.”

Preventing CIN during chemotherapy is extremely important, as this will enable cancer patients to receive the full regimen of chemotherapy and achieve treatment goals. The onset of CIN is the No. 1 reason for treatment modifications, such as downgrading the strength of chemotherapy or stopping chemotherapy altogether. When a patient develops CIN, the treating physician is required to delay the next round of chemotherapy until a patient’s white blood cell count recovers. These changes can have a profoundly negative impact on patient outcomes.

For more information on BeyondSpring’s Plinabulin Expanded Access Program, please visit http://www.beyondspringpharma.com/EAP/. Supplies may be limited.

If you are a physician in the U.S. who would like to request Plinabulin EAP access for your patient, please email expandedaccess@beyondspringpharma.com.

About BeyondSpring Headquartered in New York, BeyondSpring is a global, clinical-stage biopharmaceutical company focused on developing innovative immuno-oncology cancer therapies to improve clinical outcomes for patients with high unmet medical needs. BeyondSpring’s first-in-class lead immune asset, Plinabulin, is a potent antigen-presenting cell (APC) inducer. It is currently in two Phase 3 clinical trials for two severely unmet medical needs indications: one is for the prevention of chemotherapy-induced neutropenia (CIN), the most frequent cause for a chemotherapy regimen dose’s decrease, delay, downgrade or discontinuation, which can lead to suboptimal clinical outcomes. The other is for non-small cell lung cancer (NSCLC) treatment in EGFR wild-type patients. As a pipeline drug,” Plinabulin is in various I/O combination studies to boost PD-1 / PD-L1 antibody anti-cancer effects. In addition to Plinabulin, BeyondSpring’s extensive pipeline includes three pre-clinical immuno-oncology assets and a drug discovery platform dubbed molecular glue” that uses the protein degradation pathway.

About Plinabulin Plinabulin, BeyondSpring’s lead asset, is a differentiated immune and stem cell modulator. Plinabulin is currently in late-stage clinical development to increase overall survival in cancer patients, as well as to alleviate chemotherapy-induced neutropenia (CIN). The durable anticancer benefits of Plinabulin have been associated with its effect as a potent antigen-presenting cell (APC) inducer (through dendritic cell maturation) and T-cell activation (Chem and Cell Reports, 2019). Plinabulin’s CIN data highlights the ability to boost the number of hematopoietic stem / progenitor cells (HSPCs), or lineage-/cKit+/Sca1+ (LSK) cells in mice. Effects on HSPCs could explain the ability of Plinabulin to not only treat CIN but also to reduce chemotherapy-induced thrombocytopenia and increase circulating CD34+ cells in patients.

Cautionary Note Regarding Forward-Looking Statements This press release includes forward-looking statements that are not historical facts. Words such as "will," "expect," "anticipate," "plan," "believe," "design," "may," "future," "estimate," "predict," "objective," "goal," or variations thereof and variations of such words and similar expressions are intended to identify such forward-looking statements. Forward-looking statements are based on BeyondSpring's current knowledge and its present beliefs and expectations regarding possible future events and are subject to risks, uncertainties and assumptions. Actual results and the timing of events could differ materially from those anticipated in these forward-looking statements as a result of several factors including, but not limited to, difficulties raising the anticipated amount needed to finance the Company's future operations on terms acceptable to the Company, if at all, unexpected results of clinical trials, delays or denial in regulatory approval process, results that do not meet our expectations regarding the potential safety, the ultimate efficacy or clinical utility of our product candidates, increased competition in the market, and other risks described in BeyondSpring’s most recent Form 20-F on file with the U.S. Securities and Exchange Commission. All forward-looking statements made herein speak only as of the date of this release and BeyondSpring undertakes no obligation to update publicly such forward-looking statements to reflect subsequent events or circumstances, except as otherwise required by law.

Media Contacts Caitlin Kasunich / Raquel Cona KCSA Strategic Communications 212.896.1241 / 212.896.1276 ckasunich@kcsa.com / rcona@kcsa.com

More:
BeyondSpring Initiates Expanded Access Program with Plinabulin for Patients Suffering from CIN in the U.S. - Stockhouse

Covid-19 Impact: Patients with aplastic anemia at receiving end – Daily Pioneer

Poverty, Government apathy and Covid-19 induced-lockdown restricting travel proved fatal for little Kishan, a 11-year-old boy suffering from Aplastic anemia, a life-threatening blood disorder condition in which the bone marrow and stem cells do not produce enough blood cells

Facing severe financial constraints and waiting timely medical aid, first at Safdarjung Hospital and then AIIMS, both Government hospitals in Delhi, Kishans life was cut short in March this year amid Covid-19 pandemic.

However, Kishans is not a lone case. Dr Nita Radhakrishnan, paediatric haemato-oncologist at Super Speciality Paediatric Hospital, Noida, Uttar Pradesh says that as the deadly Coronavirus captured the attention of the nation in the most unprecedented manner, the non-Covid patients particularly those with the Aplastic anemia have suffered the most in the crisis.

She gave instances of her two teenage patients who succumbed to blood disorder in the Covid catastrophe. Manish (name change), a 17-year-old was suffering with on-and-off fever, gum bleeding, and melena for three months, he came to us in December last year just when Coronavirus had started spreading its tentacles from China to other parts of the world.

The boy was diagnosed with severe Aplastic anemia and was recommended requisite treatment like regular hospital visit for red cell transfusion before he could be given bone marrow transplant (BMT), a life saving treatment.

However, while the family was not able to visit our hospital in Noida due to the covid-lockdown, no blood products were available at the hospital near to the patients locality. In want of blood, Manish could not survive more days.

13-year-old Suresh (name change) too faced similar fate. While Government funds could not be sanctioned for his BMT in time the boy could not visit the Noida hospital for further follow-up due to travel restrictions. Two weeks later, Suresh died due to hemorrhage at his native place, lamented the doctor.

These are just two reported cases from the NCR hospital located near the countrys capital. Several have gone unreported. The Government has no policy nor any long-term plan for such patients.

The prognosis of severe aplastic anemia in our country is dismal. The incidence of 46 per million population of childhood aplastic anemia in India and other Asian countries is higher than what is observed in the West, explains Dr Radhakrishnan. The scenario is gloomy for the patients afflicted with the disease as they need blood transfusion almost every 20 days.

A significant proportion of patients of aplastic anemia (around 30 per cent) die before any definitive treatment is initiated. A study by AIIMS based on a recent series of patients follow-up showed that out of 1501 patients diagnosed over last seven years, only 303 ie 20 per cent received the definitive treatment modalities through either BMT or IST with ATG and cyclosporine, says Dr Radhakrishnan in her case report Aplastic anemia: Non-COVID casualties in the Covid-19 era, published in the latest edition of Indian Journal of Palliative Care.

The doctors have sought urgent intervention. Dr Radhakrishnan says that as we await the peak of Covid-19 in our country and possibly secondary and tertiary waves thereafter, patients with aplastic anemia who are the sickest among all hematological illnesses would benefit greatly from urgent intervention from the Government to ensure timely treatment.

Those suffering with Aplastic anemia, there is mostly delay in diagnosis, delay in initiation of treatment due to monetary constraints, non-inclusion of the disease under government schemes such as Ayushman Bharat and NHM and delay in sanction of money from other Government schemes such as Rashtriya Arogya Nidhi, Chief Minister and Prime Ministers relief fund often due to lack of proper documents, she added.

Delay means, risk of contracting fungal infections and increase in drug-resistant bacterial infections increase which further hamper the treatment, point out Dr Ravi Shankar and Dr Savitri Singh in the study.

Though the Union Health Ministry, after few days of lockdown period, issued directions for continuing treatment for essential health services including reproductive and maternal health services, newborn care, severe malnutrition, and NCDs including cancer care, palliative care, dialysis, and care of disabled, unfortunately those with Aplastic anemia got ignored.

This despite of the fact that these patients are at the highest risk of death following a break in the treatment of few weeks, notes Dr Radhakrishnan.

Because of the closure of offices and absence of staff, during the lockdown period, there was delay in sanction of usual grants due to the lockdown of offices and inability in generating documents such as income certificate from the tehsils.

For instance, Suresh and Manish, both our patients received the Government grant after around 34 months of applying for the same. But both had died before they could reach the hospital for treatment, lamented the hematologist.

See the original post:
Covid-19 Impact: Patients with aplastic anemia at receiving end - Daily Pioneer