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New stem cell treatment ‘freezes’ multiple sclerosis – Telegraph.co.uk

The disease is caused by the immune system malfunctioning and mistakenly attacking nerve cells in the brain and spinal cord.

It leads to problems with movement, vision, balance and speech.

The treatment, autologous hematopoietic stem cell transplantation (AHSCT), was given to patients with advanced forms of the disease who had failed to respond to other medications.

A similar approach has been trialed on people with certain forms of cancer, with encouraging early results.

Dr Paolo Muraro, the new study’s lead author, said: “We previously knew this treatment reboots or resets the immune system but we didn’t know how long the benefits lasted.

“In this study, which is the largest long-term follow-up study of this procedure, we’ve shown we can ‘freeze’ a patient’s disease – and stop it from becoming worse, for up to five years.”

The researchers noted, however, that the nature of the treatment, which involves aggressive chemotherapy, carried significant risks.

The chemotherapy deactivates the immune system for a short period of time, which can lead to greater risk of infection – of the 281 patients who received AHSCT, eight died in the 100 days after treatment.

The treatment works by destroying the immune cells responsible for attacking the nervous system.

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Jonathan Pitre battles blood, lung infections before second stem cell transplant – Ottawa Sun


Ottawa Sun
Jonathan Pitre battles blood, lung infections before second stem cell transplant
Ottawa Sun
People with RDEB have a fault in the gene responsible for a specific kind of collagen that connects the outer layer of skin, the epidermis, with those below it. The clinical trial seeks a biochemical correction to that fault. If the transplant works

and more »

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Amazing medicine – The News International

If we cut off the tail of a lizard, it grows back. If we cut off the hand of a human being, it does not grow back. Why not? This question has perplexed scientists for a long time. Recently scientists at the Translational Genomics Research Institute (TGen) and Arizona State University (ASU) in the US identified three tiny RNA switches (known as microRNAs) which turn genes on and off and are responsible for the regeneration of tails in the green lizard. Now researchers are hoping that using the next generation genomic DNA and computer analysis will lead to discoveries of new therapeutic approaches to switch on similar regenerative genes in human beings.

Micro RNAs are able to control many genes at the same time. They have been compared to an orchestra conductor controlling and directing many musicians. Hundreds of genes (musicians playing the orchestra of life), controlled by a few micro RNA switches, have been identified that are responsible in the regenerative process. This may well mark the beginning of a new era in which it may be possible to regenerate cartilage in knees, repair spinal cords and amputated limbs.

Tissue regeneration has become an attractive field of science, triggered by exciting advances in stem cell technologies. Stem cells are undifferentiated biological cells that are then converted into various types of cells such as heart, kidney or skin through a process known as differentiation. They can divide into more stem cells and provide a very effective mechanism for repair of damaged tissues in the body. The developing embryo contains stem cells which are then transformed into specialised cells as the embryo develops. They can be obtained by extraction from the bone marrow, adipose tissue or blood, particularly the blood from the umblical cord after birth.

Stem cells are now finding use in a growing number of therapies. For instance leukaemia is a cancer of the white blood cells. To treat leukaemia, one approach is to get rid of the diseased white blood cells and replace them with healthy cells. This may be done by a bone marrow transplant through which the patients bone marrow stem cells are replaced with those from a healthy, matching donor. If the transplant is successful, the stem cells migrate into the patients bone marrow resulting in the production of new, healthy white blood cells that replace the abnormal cells. Stem cells can now be artificially grown and then transformed (differentiated) into the heart, kidney, nerve or other typed of cells.

The field of regenerative medicine is developing at a fast pace. It involves the replacement, engineering or regeneration of human tissues and organs so that their normal function can be restored. Tissues and organs can also be grown in the laboratory if the body cannot heal itself. If the cells of the organ being grown are derived from the patients own cells, the possibility of rejection of the transplanted organ is minimised. Stem cells may also be used to regenerate organs.

Each year about 130,000 organs, mostly kidneys, are transplanted from one human being to another. The process of growing organs artificially has been greatly accelerated by the advent of 3D bioprinting. This involves the use of 3D printing technologies through which a human organ, liver or kidney, is produced by printing it with cells, layer-by-layer. This became possible when it was discovered that human cells can be sprayed through the nozzles of an inkjet printer without destroying or damaging them. Tissues and organs can thus be produced and transplanted into humans. Joints, jaw bones and ligaments can also be produced in this manner.

Initially, the work was confined to animals when ears, bones and muscle tissues were produced by bioprinting and then successfully transplanted into animals. Even prosthetic ovaries of mice were produced and transplanted so that the recipient mice could conceive and give birth later. While gonads have not been produced by bioprinting in humans, blood vessels have already been produced by the printing process and successfully transplanted into monkeys. Considerable work is also going on in the production of human knee cartilage pads through the bioprinting process. Wear and tear of the cartilage results in difficulties in walking, particular in older age groups, and often requires knee replacement through surgeries. The development of technologies to replace the damaged cartilages with new cartilages made by bioprinting could prove to be invaluable.

Another area of active research in this field is the production of human skin by bioprinting which may be used for treating burns and ulcers. Technologies have been developed to spray stem cells derived from the patient directly on the areas of the body where the skin is needed. In this way, stem cells help skin cells regrow under suitable conditions. Similar progress is being made in generating liver, kidney and heart tissues so that the long waiting time for donors can be circumvented.

When will we be able to print entire human organs? It has been estimated that complete human kidneys and livers should become commercially available through the bioprinting process within five to seven years. Hearts will probably take longer because of their more complex internal structure. However, one thing is clear: a huge revolution is now taking place in the field of regenerative medicine, triggered by spectacular advances in stem cell research. This presents a wonderful opportunity for learning and developing expertise in this field for us in our country.

In Pakistan a number of important steps have been taken in this fast evolving field. One of them is the establishment of a first rate facility for stem cell research in the Dr Panjwani Centre for Molecular Medicine and Drug Research (PCMD) in the University of Karachi. This institution has already earned an international reputation because of its outstanding publications in this field.

A second important development is that plans to set up an Institute for Translational Regenerative Medicine at PCMD so that Pakistan remains at the cutting edge in this fast emerging field are now under way.

Such initiatives can however only contribute to the process of socio-economic development if they operate under an ecosystem that is designed to promote the establishment of a strong knowledge economy.

Pakistan spends only about 0.3 percent of its GDP on science and about two percent of its GDP on education, bringing the nations ranking to the lowest 10 countries in the world. This is largely due to the stranglehold of the feudal system over our democracy. It is only by tapping into our real wealth our children that Pakistan can emerge from the quagmire of illiteracy and poverty and stand with dignity in the comity of nations.

The writer is chairman of UN ESCAP Committee on Science Technology & Innovation and former chairman of the HEC. Email: [emailprotected]

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What’s the Catch: The Fountain of Youth – Paste Magazine

Scientist Juan Carlos Izpisua Belmonte from the Salk Institute in La Jolla, California, claims that the aging process may reversible: Our study shows that aging may not have to proceed in one single direction. With careful modulation, aging might be reversed.

Izpisua Belmonte attests that he implemented a new form of gene therapy on mice that were given a genetic disorder called progeria. After six weeks of treatment, the animals looked youngerand not only that, they had straighter spines, better cardiovascular health, healed more quickly when injured and actually lived longer.

How Its Done The rejuvenating treatment performed on the mice manipulates adult cells, such as skin cells, and turns them back into powerful stem cells (similar to what is seen in embryos). These powerhouses are referred to as induced pluripotent stem (iPS) cells and have the ability to multiply and transform into any cell type in the body; in fact, in trial tests, Izpisua Belmonte says iPS cells are being designed to provide organs and limbs for patients. He claims that his latest study is the first to show that the same technique can be used on other cells to rewind the clock and make them look younger. Izpisua Belmonte explains, The treatment involved intermittently switching on the same four genes that are used to turn skin cells into iPS cells. The mice were genetically engineered in such a way that the four genes could be artificially switched on when the mice were exposed to a chemical in their drinking water.

What This Means: This finding at the Salk Institute suggests that aging may not have to proceed in one directionin fact, Izpisua Belmonte states that it may actually be reversible. Although tests have not been conducted on humans yet, he predicts that applications via creams or injections are a decade away.

This rejuvenating treatment may not lead to immortality, but due to a growing body of evidence, scientists at the Salk Institute theorize that aging is driven by an internal genetic clock that actively causes our body to enter a state of decline. In developing this technology, it is hoped that future treatments designed will slow the ticking of this internal clock and ultimately increase life expectancy.

Whats the Catch? Dr. Sidney Chiu, a 5th year resident at the University of Toronto, thinks this information should be taken with a grain of salt: The findings are promising, but nowhere near ready for the front lines of healthcare. These experiments were done in highly controlled settings on genetically modified mice. If this finding were true, it would be worthy of a Nobel Prize because it would be akin to uncovering the Holy Grail. Chiu elaborates, If you can induce iPS cells, you have the basic building blocks to regenerate anything in the body. But this is far beyond any current medical science we have.

There are also numerous issues to address concerning the study: firstly, the mice are bred in labs for these types of tests, so the variables are controlled from the outset to attain desired results. Chiu adds, In the real world, you cannot turn specific genes on and off using treated water on mice in the wild, let alone humans. There isnt one specific gene for aging; I would be cautious about this scientists claims that isolating merely four could unlock the key to anti-aging. Even if we were just talking about reviving skin tissue, if his findings were true, it would be a breakthrough.

Chiu says that while it is technically possible to alter genetic material when humans are in an embryonic state, that wasnt done here (gene editing research in human embryos is currently allowed in Sweden China, and the United Kingdom. The United States doesnt currently have any legal prohibitions against it).

But its not to say that all of this is in the realm of science fiction; Chiu offers knowledge of research being conducted specifically for telomeres and their relationship to aging. Think of telomeres as the plastic caps that protect your shoelaces from fraying. The laces would be our chromosomes, the recipe for making a living thing. In fact, telomeres have an important role; they protect genetic material from damage that could otherwise lead to diseases or cell death. But because the number of cell divisions in telomeres is finite, once they become shorter (in length) and can no longer reproduce, it causes tissues to degenerate and eventually die. It is theorized that this process may contribute to the human aging process. So scientists are trying to find ways to extend the length of telomeres.

Izpisua Belmonte says that chemical approaches (via creams or injections) might be in human clinical trials to rejuvenate skin, bones and muscle within the next decade. However, from his perspective as a frontline healthcare worker, Chiu believes that we may just have to wait a bit longer than that before such innovations are accessible to everyone.

Main Photo by Thomas Rydberg, CC-BY

Tiffany Leigh is a Toronto-based food, travel, and science writer.

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Gene Therapy Saves Puppies From A Fatal DiseaseAnd Maybe Us Next – Vocativ

For decades, some unluckydog lovers have welcomeda bundle of barking joy into their homes, only to see them perish from a mysterious disease mere weeks after their birth. The pups seemingly healthy muscles had literally wasted away in front of their owners eyes until they could no longer stand and breathe.

It wasnt until 2010 that a French research team isolated the genetic cause of this specific muscle-wasting disease in a group of Labrador Retrievers; these dogs were suffering from a single mutation that left them unable to produce an essential protein known asmyotubularin.Whats more, it was the exact kind of mutation and disease also long found in male human babies, too. That made the researchers wonder if these unfortunate puppiescould help us study the disease and even someday find a way to saveboth pets and people.

Now, years down the road, it appearsthey were right, thanks to a cutting-edgegene therapy treatment.

An international group of researchers, including some from the original French team, gathered together 10-week-old puppies with the mutation to take part in a randomized controlled trial. The dogs who were given a treatment that repaired their defectivemyotubularingene avoided the crippling muscle degeneration that killed the placebo-treated dogs by week 17. And by the ninth month of study, the saved puppies muscle and neurological function continued to match readings from healthy dogs, particularly forthose that got the highest doses.

The findings, building on an earlier proof-of-concept study of dogs and mice by the researchers, signal that a scaled-up treatment could save the lives of boys with the same sort of genetic flaw.

I believe that the dog study will be about as close as we will ever get to a human study, senior author Dr. Martin Childers of the University of Washington told Vocativ in an email. Because we found evidence that the gene therapy product spread throughout the entire skeletal musculature of adult dogs after a single infusion, it seems reasonable to expect a similar result in human patients.

Gene therapy has received plenty of attention for its potential to treat otherwise irreparable DNA defects, but according to the researchers, theres been little focus on bone- and muscle-relatedgenetic disorders. The condition treated in the current study, called x-linked myotubular myopathy, affects around one in every 50,000 boys, with most sufferers living no more than a few years. And though theres no true tally of how often it affects dogs, case reports of similar-sounding diseases have been published stretching back decades.

There will undoubtedly be hurdles to climb before the treatment Childers and his team developed, or a similar one, can be tested in people, Childers said. It is always possible that humans might respond differently, thus, clinical trials will be conducted with extraordinary care and oversight, he explained. And though the dogs suffered little adverse effects from the therapy delivered via a harmless virus researchers will still have to watch out for any possible toxicity in people.

That said, the treatment offers hope for both man and mutts. The changes seen after a single treatment have lasted for several years in the small sample of dogs the team has raised. So its possible that people wont need repeated doses or they would be infrequent, Childers said a big positive, given how expensive gene therapy is today.

And its also likely that these treatments, within the larger field of regenerative medicine, will find a place for dogs and other animals sooner than it will for people.

Veterinary medicine is ahead of human medicine in some cases with respect to regenerative technologies, Childers said. Stem cell infusions, for example, have been given to pets and horses for more than a decade.

But people may not have to wait so long for the promise of gene therapy either. Childers is hopeful that Audentes Therapeutics, a San Francisco biomedical company hes collaborating with (and which partially funded the current study), will begin their first human trials of a gene therapy treatment for x-linked myotubular myopathy, based on his teams research, later this year.

The teams findings were published earlier this February in Molecular Therapy.

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50 Years Frozen: Cryonics Today – Paste Magazine

On January 12, 1967, psychology professor James Bedford died due to cancer-related natural causes. Within hours, a team of scientists filled his veins with antifreeze. They packed his body in a container full of dry ice, and in so doing made Bedford the first man ever frozen alive in the name ofwell, if not science, something that aspired to be science one day: cryonics.

On December 23rd, 2009, at 4 a.m., I listened to my neighbors play Forever Young for the fortieth time in a row. Either the partygoers had either left or the DJ had died, and any attendees were either passed out or too blitzed to notice. The song played on repeat:

Forever young, I want to be Forever young.

I aged 10 years that night, while Bedfordtucked away in a fresh liquid nitrogen bath that came complementary with his 1991 inspectionremained immortal.

What is Cryonics, for Crying out Loud? Fifty years have passed since Bedford volunteered to become the first cryogenically frozen man. And while cultural depictions sporadically crop upthink Austin Powers, Futurama and yes, Mel Gibsonin Forever Youngcryonics is often thought to belong more to the realm of science fiction than science, and to put an even finer point on it, an escapist fiction that eludes actionable reality.

Yet cryonics offers grounds just as fertile for ethics as they do the imagination. Just think: people wage fierce wars about when life begins. Cryonics twists, turns and flips that argument around to become a deeper meditation on the moment that life ends.

So when does it?

When a Body Becomes a Patient The Alcor Life Extension Foundation which preserved Bedford describes cryonics as an effort to save lives by using temperatures so cold that a person beyond help by todays medicine can be preserved for decades or centuries until a future medical technology can restore that person to full health. The Foundation tellingly describes its members as patientsnot bodies. The dewars are not coffins, they are the temporary resting place for people who will one day wake up.

Michael Hendrix, neuroscientist and assistant professor of biology at McGill University, describes how the future of cryonics rests upon the promise of new technologies in neuroscience, particularly recent work in connectomicsa field that maps the connections between neurons a detailed map of neural connections could be enough to restore a persons mind, memories and personality by uploading it into a computer simulation.

In other words, cryonics claims that a cryogenically frozen person is not dead. He or she is merely on pause, similar to the way a video game character wont age while the player fiddles through the menu screen. The cycle of life rests upon the ability of scientistsand technologyto catch up to an idea born centuries before its time.

And as far as the science of resuscitation, cryonics does not actually rely upon the preservation of the entire body (as the choice of some people to have just their heads frozen, notably MLB player Ted Williams, testifies to), but upon the ability to map out the neurological connections between the brain, lift that map and recreate it in another bodypossibly a robot, possibly something scientists and dreamers havent yet conceived.

The Grounds for Debate Arguments against cryonics often hinge upon two main points. The first is that at best, the ethical implications of the procedure show a Labradors level of devotion to the promise of science. At worst, they play upon the emotions (and pocketbooks) of the bereaved survivors, who hold out false hope for the resuscitation of their loved one, possibly derailing and even deranging the cycles of the grieving process. The second rawand undeniablefact is that the technology for making a frozen person reenter society as a whole, living human being simply does not exist.

As for arguments for it? The most simple, powerful argument of all: immortality.

In 2014, the total count of cyropreserved bodies reached 250. An estimated 1,500 people total had made arrangements for cryopreservation after their legal death. The New York Times cites nonreligious white males as the main partakers, outdoing females by a ratio of three to one. As the worlds first volunteer, Bedford received a freebie, but most cyropreservation costs at least $80,000. A Russian company, KioRus, boasts the steal at $12,000 a headliterally speaking. But costs all but disappear in the face of a successful experiment. Say someone pays $80,000 now to rejoin the living 200 years later? Forget about calculating inflation differences.

No matter what side of cryonics one comes down uponand science offers arguments for botha central idea remains, both chilling and mesmerizing, depending upon the way its turned. A successful cyropreservation would entail rebirthbut into a world wholly different than the one left behind. If James Bedford came back tomorrow, could he handle the emotionalnot to mention mentaltribulations of adjusting to a world that moved on without him? Would the forever young experience drone on like the song on that December night, an individual sentenced to the eternal return of the same song, Existence?

After my own encounter with Forever YoungI certainly hope not.

Elisia Guerena is a Brooklyn based writer, who writes about tech, travel, feminism, and anything related to inner or outer space.

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Cryonics This Scottish author pays 50 pounds a month to preserve his brain after death – Zee News

New Delhi: Of late, the science of cryonics seems to have captivated the hearts of scientists and the public alike with some people now opting for cryopreservation after their deaths.

Cryonics is the practice or technique of deep-freezing the bodies of those who have died of a disease, in the hope of a future cure.

In a latest, an author from Scotland has started paying a research institute to preserve his brain cryogenically after his death.

As per reports, DJ MacLennan has been paying 50 pounds ( appriximately Rs 4,000) a month for the past decade to Alcor Institute in Arizona, USA, to preserve his brain in the hope that he can one day be brought back to life.

MacLennan, who lives on the Isle of Skye, has told the institute that when he dies he wants the team of volunteers to fill his body with anti-freezing liquid before plunging it into ice water. His body will then be wrapped in a polyethylene, submerged in alcohol and lowered into ice before being shipped to Arizona. The head will then be removed and frozen in liquid nitrogen before being stored.

According to MacLennan, if organs can be donated and aren’t wasted anymore, brains should definitely not be wasted, instead it’s the important part to store.

While the full-body procedure costs 75,000 pounds, the author from Skye, has opted for the 40,000 pounds brain freeze.

In November last year, a 14-year old girl who died of cancer became the first child to be cryogenically frozen after death in the UK.

The procedure was carried out after winning a landmark court case shortly before her death. She had written a heartbreaking letter to a judge explaining that she wanted a chance to live longer after suffering from a rare form of deadly cancer.

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When Screening for Disease, Risk is as Important to Consider as … – University of Virginia

Physicians and patients like to believe that early detection of cancer extends life, and quality of life. If a cancer is present, you want to know early, right?

Not so fast.

An analysis of cancer screenings by a University of Virginia statistician and a researcher at the National Cancer Institute indicates that early diagnosis of a cancer does not necessarily result in a longer life than without an early diagnosis. And screenings such as mammograms for breast cancer and prostate-specific antigen tests for prostate cancer come with built-in risks, such as results mistakenly indicating the presence of cancer (false positives), as well as missed diagnoses (false negatives). Patients may undergo harsh treatments that diminish quality of life while not necessarily extending it.

Yet the benefits of early diagnosis through screening often are touted over the risks.

It is difficult to estimate the effect of over-diagnosis, but the risk of over-diagnosis is a factor that should be considered, said Karen Kafadar, a UVA statistics professor and co-author of a study being presented Sunday at a session of the 2017 meeting of the American Association for the Advancement of Science. How many diagnosed cases would never have materialized in a persons lifetime, and gone successfully untreated? Treatments sometimes can cause harm, and can shorten life or reduce quality of life.

Kafadar is not advocating against screening, but her findings show that frequent screening comes with its own risks.

As a metric for evaluation, reduction in mortality is considered the standard. So if a disease results in 10 deaths per 100,000 people in a year, and screening reduces the deaths to six per 100,000 people, then there seems to be an impressive 40 percent reduction in mortality.

However, a more meaningful metric, Kafadar said, may be: How much longer can a person whose case was screen-detected be expected to live, versus a case that was diagnosed only after clinical symptoms appeared? This issue becomes harder to discern how long a patient survives after a diagnosis versus how long the patient might have lived anyway. Some cancer cases might never become apparent during a persons lifetime without screening, but with screening might be treated unnecessarily, such as for a possibly non-aggressive cancer. And some aggressive forms of disease may shorten life even when caught early through screening.

Kafadar and her collaborator, National Cancer Institute statistician Philip Prorok, gathered long-term data from several study sources, including health insurance plans and the National Cancer Institutes recently completed long-term randomized control trial on prostate, lung, colorectal and ovarian cancer, to consider several factors affecting the value of screening over-diagnosis, lead time on a diagnosis and other statistical distortions to look at not just how many people die, but also life extension.

People die anyway of various causes, Kafadar said, but most individuals likely are more interested in, How much longer will I live? Unfortunately, screening tests are not always accurate, but we like to believe they are.

Because the paper considers together the factors that affect statistical understanding of the effectiveness of screening, rather than looking at each of these factors in isolation as previous studies have done, it offers a new statistical methodology for teasing out the relative effects of cancer screenings benefits and risks.

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Quick fix: Zeman turns last-place Pescara around in 3 days – The Republic

ROME Turnarounds dont get much quicker than this.

Three days after he was hired, Zdenek Zeman coached last-place Pescara to its first outright win of the season by a score of 5-0 the first time the Abruzzo club has won with a five-goal margin in Serie A history.

All I can do is thank the lads because we only had three training sessions together but they showed that if we dedicate ourselves and if we stay focused we can do well, Zeman said after the victory over Genoa on Sunday. We broke the spell.

For its only other victory this season, Pescara was awarded a 3-0 win over Sassuolo in August because Sassuolo used an ineligible player. Sassuolo had won 2-1.

Previous coach Massimo Oddo was fired last week after six consecutive losses in the last two of which Pescara had conceded a total of 11 goals.

So on Thursday, Pescara rehired the 69-year-old Zeman, a chain-smoking Czech coach known as the Bohemian who had guided the club to the Serie B title five years earlier with a record number of goals.

Known for his all-out attacking style and 4-3-3 formation, Zemans impact was immediate.

After an own-goal five minutes in, Gianluca Caprari the only holdout from Zemans first spell at Pescara scored two, English-born Libya international Ahmad Benali added another and 20-year-old Alberto Cerri got his first Serie A goal.

The result prompted the firing of Genoa coach Ivan Juric.

Zeman doesnt say much but when he does speak hes very clear, Cerri said. He told us to clear our heads and give our all.

Pescara is still 11 points from safety with 13 matches to go but its only one point behind penultimate Crotone and two points behind Palermo. The goal is to catch Empoli, which is 10 points ahead.

It doesnt depend only on us, it depends on how the others do, too, Zeman said. We would need the three squads ahead of us to stall and us to leap forward. Plus, we still have to face (Serie A leader) Juventus so we already have one less match available even though well try to win that one.

Zeman, who will turn 70 before the end of the season, has a long history with Juventus. He accused the Turin power of drug abuse nearly 20 years ago when he coached Roma, sparking a years-long trial.

In 2004, Juventus physician Riccardo Agricola was convicted of administering banned substances, including the hormone EPO, to Juventus players from 1994-98, and was handed a suspended sentence of 22 months. He was cleared on appeal a year later.

Zeman left Pescara in 2012 to return to Roma, which was an offer he said he couldnt refuse. But memories of his first spell at Pescara, when the club scored a Serie B-record 90 goals in 42 games with a team that featured current Italy internationals Ciro Immobile, Lorenzo Insigne and Marco Verratti made it easy for him to come back.

Zeman said he inherited a squad in poor physical condition.

Were going to have to work on that aspect a lot, Zeman said. The important thing is that the lads dont think the season is over.

Pescaras hard-core ultra fans stayed away from the Genoa match in protest at the squads poor results.

I would have liked for them to have come out and given us a hand, Zeman said. I hope we can convince them to return.

Andrew Dampf on Twitter: http://www.twitter.com/asdampf

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Family pushes bill expanding medical training in schools – WBIR.com

Currently only a school nurse can administer a shot to a student in the condition, but a bill in the state legislature would allow other school employees to be trained so they could also intervene.

Grant Robinson , WBIR 11:57 PM. EST February 19, 2017

KNOXVILLE – On Wednesday a couple from Knoxville will address the senate education committee in support of a bill that would expand access for training and administering a life-saving drug.

Adrenal insufficiency is a condition where a body doesn’t produce cortisol – a hormone essential for managing stressors.

People with the disease may go into adrenal crisis, a medical state marked by severe pain, low blood pressure and loss of consciousness that if untreated can lead to death.

Adrenal crisis can be treated, but under the current state law school nurses are the only employees at schools who can administer the shot.

Senate Bill 117 amends that law by allowing other school employees to be trained to administer the injection.

Landon Adzima is a junior at Gibbs High School and lives with adrenal insufficiency. During last year’s wresting state tournment, Landon suffered an adrenal crisis.

“There’s nothing I could really compare it to,” Adzima said. “It just felt like my body was shutting down and that I was helpless and there was nothing I could do about it.’

Adzima’s parents were in attendance and were able to administer the drug. They worry that if a student is at school and the nurse is off campus, an adrenal crisis could cause lasting damage.

“We think it’s important that school employees could be properly trained to give this injection in the absence of a school nurse,” Andy Adzima said. “They’re always the nearest person to the student in an emergency that might happen at the school and the time they might have to wait for someone who’s legally allowed to give this injection could make a huge difference.”

The bill would only require schools to train personnel if a parent or guardian notified the school that a student is diagnosed with adrenal insufficiency.

The bill is sponsored by Senator Richard Briggs (R – Knoxville), who is also a physician.

( 2017 WBIR)

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The Secret Behind Trump’s Flowing Locks – eParisExtra.com (blog)

Whether you like the President or not, its hard not to like his full head of hair. As Trump himself said As everybody knows, I do not wear a wig. My hair may not be perfect but its mine. The Presidents hair has been a topic of jokes, intrigue and even magazine covers. Some have even suggested that he has grown more hair over the past few years. When most men his age are going bald, what is Mr. Trumps secret?

According to Trumps personal physician, Bornstein, he credited the drug finasteride for Trumps hair growth and said that Trump has all his hair. Finasteride is better known by the name Propecia.

But what is finasteride, and how does it work? This is an oral drug approved by the Food and Drug Administration (FDA) to treat and reverse male pattern baldness. This type of baldness usually begins at the temples and leaves a bald top with some hair around the sides of the head.

Finasteride blocks the action of an enzyme called 5-alpha-reductase. This enzyme changes testosterone to another hormone (DHT) that causes hair loss in males. Finasteride increases hair growth on the scalp by inhibiting this enzyme and also fuels new hair cell growth ( journal Expert Opinion in Drug Discovery) . The effect of finasteride on the scalp will only last as long as the medicine is taken. When it is stopped, the hair will be lost.

Finasteride is also used to treat BPH (benign prostate enlargement) which is common as men age. Men with BPH usually have difficulty urinating and a need to get up at night to urinate. Finasteride helps make these symptoms less severe and reduces the chance that prostate surgery will be needed. It may be used alone or in combination with other medicines.

Finasteride works by increasing the level of testosterone which in turn decreases prostate size and reduces hair loss. If one were to speculate, it is possible that the President has a low PSA and a relatively high testosterone level due to the effect of finasteride.

According to the Journal of Investigative Dermatology, Finasteride has gone through three placebo-controlled trials to assess its effects on male pattern baldness. Men who had taken finasteride had 107 more hairs per square inch after one year of use and 277 more hairs per square inch after five years of use. It should be noted that the average person may have anywhere from 1,050 to 1,935 hairs per square inch according to the journal. No wonder, Mr. Trumps head of hair looks fuller!

The drug has relatively few side effects. Rarely, men reported erectile dysfunction and decreased libido, and there was a slight association with depression, but it is considered a safe drug.

Irrespective of your political leanings, or whether you personally like the President or not, or whether you think he is going to win a lot, Mr. Trump has certainly won the battle of hair. He has managed to avoid baldness and maybe even grow hair. Whether he has done this with the help of Finasteride, remains a matter of speculation until we know for sure. But finasteride remains an option for those men who are starting to lose hair, and would like to hang on for a little bit longer.

This information is strictly an opinion of Dr Prakash, and is not intended to replace the advice of your doctor. Dr Chris Prakash is a contributing columnist, and author of eParisExtras The Doctor is In column. He is a medical oncologist at Texas Oncology Paris. He is board certified in Internal Medicine, Oncology and Hematology. He can be reached at 9037850031, or Sucharu.prakash@usoncology.com

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The Secret Behind Trump’s Flowing Locks – eParisExtra.com (blog)

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These transgender patients now have a place to change their lives all at once – Miami Herald


Miami Herald
These transgender patients now have a place to change their lives all at once
Miami Herald
It was the final touch on her new, true self. For everything else, the 28-year-old went to Dr. Christopher Salgado at the University of Miami, the lead surgeon at the hospital's new LGBTQ clinic. … In 2014, Medicare began covering hormone therapy and

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These transgender patients now have a place to change their lives all at once – Miami Herald

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Mayo doc’s stem cell experiment blasts into space – Post-Bulletin

JACKSONVILLE, Fla. As a boy growing up in Kano, Nigeria, Dr. Abba Zubair dreamed of going to space.

On Sunday, his work hitched a ride with a private rocket blasting off from NASA’s Kennedy Space Center in Cape Canaveral, Fla., on a trip to the International Space Station.

Dr. Zubair, an associate professor of laboratory medicine and pathology at the Mayo Clinic’s Florida campus, prepared a science package involving stem cells as part of a resupply mission to the ISS aboard a SpaceX Falcon 9 rocket.

“It was my first rocket launch view,” said Dr. Zubair, who was on hand to watch and listen to the deafening sound as his experiment rode into space. “It was incredible.”

The stem cells — specialized cells derived from bone marrow come from Dr. Zubair’s lab. Dr. Zubair, according to a report from the Mayo Clinic, specializes in cellular treatments for disease and regenerative medicine. He hopes to find out how the stem cells hold up in space and if they can be more quickly produced in microgravity.

More specifically, Zubair said, he is hoping the research can help in treatment of patients who have suffered a stroke-related brain injury.

“Stem cells are known to reduce inflammation,” he said in a press release. “We’ve shown that an infusion of stem cells at the site of stroke improves the inflammation and also secretes factors for the regeneration of neurons and blood vessels.”

The problem with such a treatment and studying the treatment is generating enough stem cells for the job. Based on current regenerative medicine studies, patients need at least 100 million stem cells for an effective dose. However, reproducing stem cells can be time consuming since the cells naturally limit their numbers.

“Scalability is a big issue,” Dr. Zubair said. “I’ve been interested in a faster way to make them divide.”

And on earth, everything is impacted by gravity, from how high we grow to our bone size and other physiological traits. “So, how can we use the effect of gravity to impact how the cells divide?” he asked.

Experiments that simulate stem cell growth in microgravity, thus far, have shown cells do grow more quickly than experimental controls, he said. So he began working toward getting an experiment into space. The experiment needed to be designed so the crew onboard the space station could run the experiment with some simple training, and Dr. Zubair will be able to watch the experiment in real time via a video connection. “We’ll get some data as early as next week,” he said.

If all goes well, growing stem cells in space something Dr. Zubair admits sounds like a dream of the distant future might become a reality more quickly than many people think.

“There are some companies interested in floating labs,” he said. “I think the future is bright. There are a lot of possibilities in the area of regenerative medicine.”

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Blast off: Stem cells from Mayo Clinic physician’s lab launch into space – Medical Xpress

February 19, 2017

Consider it one physician’s giant leap for mankind. Today, the latest rocket launch from NASA’s Kennedy Space Center in Cape Canaveral, Florida, included a payload of several samples of donated adult stem cells from a research laboratory at Mayo Clinic’s Florida campus. The launch by SpaceX, an American aerospace manufacturer and space transport services company, is part of NASA’s commercial resupply missions to the International Space Station.

The biological cells come from the laboratory of Abba Zubair, M.D., Ph.D., who says he has eagerly awaited the launch following several delays over the past couple of years. Dr. Zubair, who specializes in cellular treatments for disease and regenerative medicine, hopes to find out how the stem cells hold up in space. He says he’s eager to know whether these special cells, which are derived from the body’s bone marrow, can be more quickly mass-produced in microgravity and used to treat strokes. Microgravity is the condition in which people or objects appear to be weightless. The effects of microgravity can be seen when astronauts and objects float in space. Microgravity refers to the condition where gravity seems to be very small.

“At Mayo Clinic, research drives everything we do for patients,” says Gianrico Farrugia, M.D., vice president, Mayo Clinic, and CEO of Mayo Clinic in Florida. “This space cargo carries important material for research that could hold the key for developing future treatments for strokea debilitating health issue. Research such as this accelerates scientific discoveries into breakthrough therapies and critical advances in patient care.”

Dr. Zubair says he has dreamed of this moment all his life, with a passion for space that goes back to his childhood in the northern city of Kano, Nigeria. There, he says he came across a book about the first moon launch and became instantly enthralled. In high school, he recruited other physics students to build a model rocket prototype using corrugated metal and rudimentary materials from the local blacksmith. When it came time to apply for college, however, the school adviser steered him from becoming an astronaut. “He said it may be a long time before Nigeria sends rockets and astronauts into space, so I should consider something more practical,” Dr. Zubair recalls.

With the goal of being useful to patients and helping cure disease, he headed to medical school in Nigeria. His training took him to the University of Sheffield, in Sheffield, England; the University of Pennsylvania in Philadelphia; and Harvard University in Cambridge, Massachusetts, as he specialized in bone marrow transplants and stem cell research. He came to Mayo Clinic’s Florida campus to treat cancer patients and others whose conditions could be helped by regenerative medicineall the while running a research lab that studies adult stem cells.

Dr. Zubair came across a request for research proposals that involved medicine and outer space four years ago. His mother had died of stroke in 1997, and he had been thinking about stem cells as a treatment for stroke-related brain injury. Collaborating with Mayo Clinic neurologists James Meschia, M.D., and William D. Freeman, M.D., he studied mouse models of stroke.

“Stem cells are known to reduce inflammation,” he explains. “We’ve shown that an infusion of stem cells at the site of stroke improves the inflammation and also secretes factors for the regeneration of neurons and blood vessels.”

One big problem is that it may take as many as 200 million cells to treat a human being, and developing vast numbers of stem cells on Earth can take weeks.

“It’s further complicated, because some patients are unable to donate cells for themselves, and, sometimes, there aren’t enough donors who are a good match, as sometimes occurs for minorities,” he says.

Studies in simulators on Earth have shown that adult stem cellsthe undifferentiated cells that exist in the body to replace damaged or dying cellsreproduce quickly and reliably in microgravity. While it’s not known why microgravity works better than a petri dish, some researchers speculate the conditions may be similar to the floating environment of developing cells in the body. With funding from the Center for the Advancement of Science in Space, a nonprofit organization, Dr. Zubair hopes to find that, in space, stem cells can be reproduced safely in large quantities, providing new opportunities for patients.

He’ll gather real-time information about the cells as astronauts conduct experiments measuring molecular changes.

“We’ll be looking to see if there are genes activated in microgravity and analyzing the stages of the cell cycle,” he says.

“We may discover proteins or compounds that are produced that we can synthesize on Earth to encourage stem cell growth without having to go to microgravity.” Over the last three years of planning, he says he’s been tickled to learn about the challenges of space-based research, such as the need for techniques to handle fluids that don’t mix in microgravity.

Most importantly, experiments will continue after the expanded stem cells return to Earth.

“We’ll study them to make sure they’re normal, functional and safe for patients with stroke,” he says. “My work in regenerative medicine has always been intentionally translationalnot just to study what the cells do and what can be done with them but to make a difference for patients. That’s what makes our project unique.”

For the launch, Mayo Clinic is collaborating with the Center for Applied Space Technology (CAST) in Cape Canaveral, and BioServe Space Technologies in Boulder, Colorado. CAST supported Dr. Zubair’s research by providing strategic mission planning, proposal development, spaceflight technical support and served as an interface between the research team and various space activities and agencies. BioServe provided space flight hardware, on orbit research protocol and scheduling interface.

Explore further: Researcher to grow human cells in space to test treatment for stroke

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Blast off: Stem cells from Mayo Clinic physician’s lab launch into space – Medical Xpress

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Resetting the immune system ‘stops MS in it’s tracks for at least five years’ – The Sun

The condition is caused by the immune system malfunctioning and mistakenly attacking nerves in the brain and spinal cord

A GROUNDBREAKING treatment that resets the immune system could stop the spread of mutliple sclerosis in nearly half of patients, expertssay.

The risky treatment involves wiping the bodies immune system with cancer treatment and rebooting it with a stem cell transplant, but not all patients will be suitable.

Alamy

A stem cell transplant followed by aggressive chemotherapy could reset the immune system and manage the symptoms of MSThe treatment prevents symptoms of the disease worsening for five years in 46 per cent of patients, a study from Imperial College London found.

Multiple sclerosis affects around 100,000 people in the UK, and 2.3 million worldwide.

The condition is caused by the immune system malfunctioning and mistakenly attacking nerves in the brain and spinal cord.

This leads to a range of symptoms including fatigue, problems with arm and leg movement, vision and balance.

There is no cure but certain medications can help slow progression of the disease.

But a stem cell transplant, followed by aggressive chemotherapy, could change that.

Participants in the studyhad advanced forms of the disease and had not responded to any other treatment.

They were given aautologous hematopoietic stem cell transplantation (AHSCT) A process in which removes healthy stem cells from the body to allow medics to kill the remaining ones.

It aims to stop the immune system from attacking the bodys nerve cells.

The results, published in the journal JAMA Neurology, suggested some patients saw an improvement in their symptoms.

http://www.alamy.com

In the treatment, a patient is given a drug that encourages stem cells to move from the bone marrow into the blood stream, and these cells are then removed from the body.

The patient then receives high-dose chemotherapy that kills any remaining immune cells.

The stem cells are then transfused back into their body to re-grow their immune system.

Previous studies have suggested this resets the immune system, and stops it from attacking the nerve cells.

But medics warn that because thetreatment involves aggressive chemotherapy that inactivates the immune system for a short period of time, some patients died from infections.

Out of the 281 patients who received the treatment in the study, eight died in the 100 days following the treatment.

Older patients, and those with the most severe forms of the disease, were found to have a higher risk of death.

MSis a neurological condition that affects your nerves.

Its caused when your immune system isnt working properly and the coating around your nerves, called myelin, is damaged.

The protective coating helps ensure messages travel smoothly from your nerves to your brain, but when it is damaged the messages become disrupted meaning they can slow down, become distorted or not make it at all.

Once diagnosed, MS stays with you for life, but treatments and specialists can help you to manage the condition and its symptoms.

The cause is not know and there isnt yet a cure, but research is progressing fast.

Symptoms:

Treatment: According to the MS Society, more than 100,000 people in the UK have MS and symptoms usually start in your 20s and 30s, affecting more women than men.

While there is no known cure for MS, there are several ways to treats its symptoms including medication, diet, exercise and physiotherapy.

The best course of action depends on what symptoms the sufferer has.

Dr Paolo Muraro, lead author of the study, said: We previously knew this treatment reboots or resets the immune system and that it carried risks but we didnt know how long the benefits lasted.

In this study, which is the largest long-term follow-up study of this procedure, weve shown we can freeze a patients disease and stop it from becoming worse, for up to five years.

However, we must take into account that the treatment carries a small risk of death, and this is a disease that is not immediately life-threatening.

Most patients with multiple sclerosis have a type of the disease that has flare-ups, known as relapses, followed by an improvement in symptoms.

Dr Muraro said the number of years this treatment prevented symptoms from worsening wasfar greater than would be expected in untreated patients with severe forms of relapsing MS.

Dr Muraro added: These findings are very promising but crucially we didnt have a placebo group in this study, of patients who didnt receive the treatment.

We urgently need more effective treatments for this devastating condition, and so a large randomised controlled trial of this treatment should be the next step.

Dr Sorrel Bickley, head of biomedical research at the MS Society, said: This study is one of the largest to date looking at AHSCT as a treatment for MS and the findings offer some encouraging insights.

It shows that AHSCT can slow or stop progression for many years, and the treatment is most effective in people with MS who have active inflammation in their brain and spinal cord.

There are more than 100,000 people with MS in the UK, its a challenging and unpredictable condition to live with and thats why the MS Society is funding research like this to further our knowledge and find treatments for everyone.

If anyone with MS is considering AHSCT they should speak to their neurologist as a referral is needed to access this treatment via a trial or on the NHS.

We pay for your stories! Do you have a story for The Sun Online news team? Email us attips@the-sun.co.ukor call 0207 782 4368

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Resetting the immune system ‘stops MS in it’s tracks for at least five years’ – The Sun

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Giving the Gift of Life ~ National Donor Day – Shoshone News Press

February 18, 2017 at 1:00 am | By Lisa Turpin Special to the News-Press

National Donor Day, observed on Feb. 14, is a great time to register as an organ, eye, and tissue donor or to make an appointment to donate blood or platelets.

What could show more love on Valentines Day than the act of giving ones body to help another?

Whether you are a living donor of blood products, stem cells, kidney or liver, register with your state as an organ donor, or make the decision for your loved one to be a donor, you are truly giving the gift of life.

Nationally, more than 119,000 people are waiting for an organ transplant, including 2,091 children.

That doesnt include the number waiting for a bone marrow (stem cell) matched donor which is much more complicated to find.

Significant progress continues in the advancement of transplantation medicine with goals of lengthening life spans, restoring function, appearance, and quality of life.

But it still takes the generosity of donors and their loved ones to make a transplant possible.

Claudia Swigart of Pinehurst believes the true value of organ donation is the gift of time.

In her case, fifteen years with her husband Wendell that she, their five combined children, thirteen grandchildren, and twelve great-grandchildren may not have had.

Wendell and his three siblings all had Polycystic Kidney Disease (PKD), an inherited condition causing cysts to form in the kidney causing damage and kidney failure.

Wendell worked in the mine here in the Valley, shares Claudia.

He found out he had Polycystic Kidney Disease when he was thirty-four and he was careful, he exercised, ate healthy and never smoked. He didnt have any kidney problems until he was sixty-three and had to have open heart surgery.

The surgery was hard on Wendell and his lungs collapsed, he nearly died and it put his kidneys in distress.

He started dialysis after that and was eventually put on the kidney transplant list to receive a transplant at Sacred Heart Medical Center.

The dialysis center in Pinehurst had not opened, so Claudia drove Wendell to Coeur dAlene two times a week for three-hour treatments.

Claudia shared, I am so thankful they opened a dialysis center here. Its exhausting enough to be on dialysis without the traveling.

But there is more to this story.

We always liked telling everyone we could about what happened because we knew God had His hand in the plan, explains Claudia.

They normally traveled to Arizona in their camper for the winter.

Wendell would arrange to have dialysis at the center in Arizona instead of Coeur dAlene.

Well, in 2001 we were planning on leaving so Wendell called to remove himself from the transplant list while we were gone. But, when he called to arrange dialysis at the center in Arizona, they were full! said Claudia.

Since Wendell couldnt have dialysis in Arizona, they were forced to stay home which meant he remained on the transplant list.

Just a few weeks later we got the call! Claudia exclaimed.

Wendell was told he had a matched kidney on the way from a donor in Alaska.

Wendell was sixty-five at the time and he asked if there were any younger people waiting for transplants, anyone still raising young kids who needed it more than he did. His doctor knew he was that kind of man and firmly told him that it was Wendells kidney and he was taking it!

Wendells kidney was such a good match he never experienced any problems or symptoms of rejection.

The transplant coordinators said that the Swigarts could write a letter to the donors family in Alaska if they wanted to have communication with them or thank them.

We wrote a letter to the family two months later and Wendell told them he would take real good care of the kidney, Claudia said.

Wendell did take great care of himself but unfortunately fought esophageal cancer unrelated to his kidneys and passed away in March of 2016 at the age of 80.

The donors family never wrote back, so they do not know the identity of the donor, but Claudia and Wendell were glad they sent the thank-you letter.

We went back to Arizona the year after the transplant and didnt have to worry about dialysis any more. We may never have gotten to do that and he sure wouldnt have had the life he had without the generosity of the donor and their family.

Wendell Swigart had 15 extra quality years with his bride and they celebrated their forty-sixth wedding anniversary before his passing.

Statistics say that only three out of 1,000 people who die are candidates for organ donation, and thats if their families agree to donation.

Even if you register as a donor, it is still up to your family to make the final decision.

Making your family aware that you want to be a donor is the most important thing you can do. For more information visit http://www.donatelife.net or http://www.Organize.org.

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Giving the Gift of Life ~ National Donor Day – Shoshone News Press

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US Kalytera buys Israel medical cannabis firm Talent for over $10m – The Times of Israel


The Times of Israel
US Kalytera buys Israel medical cannabis firm Talent for over $10m
The Times of Israel
Talent is currently studying the use of cannabidiol (CBD) an antioxidant extracted from the cannabis plant to prevent and treat Graft versus Host Disease (GvHD), a life-threatening condition that can occur after stem cell or bone marrow
Kalytera Therapeutics Acquires Israeli Cannabis DrugmakerThe Jewish Press – JewishPress.com

all 2 news articles »

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US Kalytera buys Israel medical cannabis firm Talent for over $10m – The Times of Israel

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A key ion channel may SLACK off in ALS – ALS Research Forum

Slacking off in ALS? Mutant SOD1 may partially close the SLACK ion channel resulting in increased excitability in some neurons (Zhang et al., 2017).[Image: NIGMS.]

Increased activity in the motor cortex of the brain may occur in most forms of ALS (see September 2015 news). But whether this hyperexcitability contributes to the disease remains an open question.

Now, researchers at Yale University make the case that ALS-linked mutant SOD1 may downregulate a key sodium-gated potassium ion channel, known as SLACK, through an apoptosis signal-regulatingkinase1 (ASK1)-based mechanism (Zhang et al., 2017).

The findings may help explain how motor neuron hyperexcitability occurs in ALS. These changes in excitability may contribute to disease pathogenesis and may underlie fasciculations, one of the earliest clinical manifestations of the disease.

The question is whether this pathway is the primary way that SOD1 mutations cause disease, said Steve Vucic of the University of Sydney, who was not involved in the study. If so, [there] is a tremendous opportunity for developing treatments against these kinase pathways.

The study is published on January 24 in the Journal of Neuroscience.

Excitement builds

Neuronal hyperexcitability emerged in recent years as an early and potentially unifying stepin ALS, due to its detection in a number of sporadic and genetic forms. While the evidence is still not yet conclusive, some studies suggest that this prolonged excitation can lead to toxicity, strengthening the case that these changes in excitability may contribute to the disease (Fritz et al., 2013; Hadzipasic et al., 2014).

How hyperexcitability occurs in ALS remains unclear. But a growing number of studies suggest that mutant SOD1 may be involved, at least in some cases of the disease (Wainger et al., 2014; van Zundert et al., 2008).

Researchers at Yale University, led by Leonard Kaczmarek and Arthur Horwich, wondered whether mutant SOD1 could trigger hyperexcitability in motor neurons by downregulating a key membrane-bound ion channel called SLACK (sequence like a calcium-activated K channel), also known as KCNT1 or KNa1.1.

SLACK is a key regulator of excitability that helps neuronsreturn to the resting state upon firing. Its widely expressed in the CNS and its dysfunction has also been implicated in neurological diseases including Fragile X and epilepsy (Barcia et al., 2012; Heron et al., 2012; Martin et al., 2014).

Hyperexcitability in the bag. Researchers use sea slug bag cell neurons to study underlying hyperexcitability mechanisms. [Image: Kabir et al., 2001 under a CC-BY-NC-SA license.]

To investigate this question, first co-authors Yalan Zhang and Weiming Ni turned to the neuronalmodel system, the sea slug Aplysia. The system gained recognition in the 1960s for its role in providing Eric Kandel Nobel Prize-winning insights into learning and memory formation.

The approachinvolves the manipulation and study of bag cell neurons, very large neuroendocrine cells in the sea slugs abdomen that control egg laying. The really big advantage is that, because of their size, you can inject materials into them and then use a very fine microelectrode to record changes in excitability, all without any disturbance of the cytoplasm, Kaczmarek said.

The researchers compared the activity of potassium channels in bag cell neurons in the presence or absence of wild-type or mutant SOD1, including soluble oligomers of increasing size. They found that SOD1 or mutant SOD1 G85R monomers had no effect. But when they injected SOD1 G85R oligomers, they observed a reduction in outward potassium currents by 20-30%. This drop occured within 10 minutes and increased with larger oligomer size.

Whats more, SOD1 G85R oligomers increased excitability of these neurons. Injection of these soluble 300 kDa protein complexes decreased the neurons resting membrane potential and increased its susceptibility to firing in response to applied stimuli, they found.

Further experiments identified the SLACK channel as the one most likely to have been affected by mutant SOD1, because neurons pretreated with siRNA against SLACK mitigated the effect of these protein complexes in these neurons.

Together, the results suggest that soluble mutant SOD1 oligomericcomplexes may lead to hyperexcitability due to partial closure of SLACK, a key sodium-gated potassium channel that helps neurons return to their resting state upon firing.

ASK1ing for trouble

How could mutant SOD1 downregulateSLACK? The researchers suspected that these effects may be triggered by ASK1, a key kinase that has been previously implicated in the destruction of motor neurons in the disease (Raoul et al., 2002).

ASK1 has been shown to mediate key effects of mutant SOD1 in mouse models of the disease including ER stress and disruption of axonal transport (Lee et al., 2016; Song et al., 2013). In addition, inhibitingthis pathway appears to extend the survival of a SOD1 G93A mouse model of the disease (Fujisawa, et al. 2016).

To investigate this possibility, the researchers blocked ASK1 signaling and determined the impact of SOD1 oligomeric complexes on potassium channel activity. They found that the suppression of outward potassium current could be abolished by pre-treatment with an inhibitor of the apoptosis signaling regulating kinase ASK1. Similar effects were achieved with an inhibitor of one of ASK1s downstream targets, JNK.

The results, Kaczmarek said, suggest that mutant SOD1 oligomericcomplexessuppressSLACK channels in neurons through a ASK1-based mechanism, causing hyperexcitability.

Its an attractive idea, says Massachusetts General Hospitals Brian Wainger, who was not involved in the study. The findings may provide a potentially direct mechanistic connection between mutant SOD1 and motor neuron hyperexcitability in ALS.

Mind your Potassium and KCNQs. Researchers are evaluating Kv 7.2 potassium channel activators including retigabine (orange) in hopes to reduce hyperexcitability in people with the disease. More specific channel modulators are being developed. One such activator, AUT00063, is being evaluated at the phase 2a stage by the London startup Autifony Therapeutics to treat hearing disorders. [Miceli et al., 2011 under CC BY 4.0 license.]

But a change in excitability may not be the only or even the most important consequence of SLACK down regulation, according to Kaczmarek. SLACK may act as an activity sensor, providing a direct link between neuronal firing and protein synthesis.

His teamhas previously shown that SLACK channel activity plays a role in synaptic development, through its ability to regulateactivity-dependent protein synthesis (Brown et al., 2010; Zhang et al., 2012). When you precipitate the channel from mammalian brain, it pulls down several messenger RNAs, he pointed out, and mutations that cause channel overactivity are associated with epilepsy (Barcia et al., 2012; Kim et al., 2015).

In fact, Kaczmarek added, it may not be the hyperexcitability of motor neurons that is toxic in ALS, but rather its proposed (but not yet tested) consequences on protein synthesis. A rapid change in the activity of these channels, as we saw here, is likely going to alter protein synthesis, and that can produce much longer-lasting effects, potentially more consistent with a late-onset disease.

This was an extremely elegant study, and an ingenious way to approach the issue of hyperexcitability, said Steve Vucic, who, in collaboration with University of Sydneys Matthew Kiernan in Australia helped identify these neuronal changes as an early sign of ALS in people with the disease. The goal now will be to see if this same pathway is affected in the mammalian models, or in human ALS iPS cells.

Brian Wainger agrees. The key questions, according to Wainger, are whether these findings hold up in mammalian models, and whether these findings can be generalized to other forms of the disease.

Searching for ALS-linked gene variants in SLACK or related ion channels might also provide insight into its relevance for the human disease, added Vucic.

Approaching the clinic

Hyperexcitability is clearly a clinical feature of many forms of familial and sporadic ALS, explains Wainger. Thats why it is attractive as a convergent mechanism for many forms of ALS. But one of the challenges is to determine to what extent an increase in firing is relevant for disease pathogenesis, rather than, as some argue, being a compensatory mechanism. Directly modulating excitability is one of the clearest ways of answering that question directly, he added.

If motor neuron hyperexcitabilitydoes hold up as a driver of disease, however, it may be a good target for therapy, according to Kaczmarek. I see this as very much a therapeutic possibility.

The reason is because opening up these potassium ion channels may help motor neurons in people with ALS return to their resting state and thereby, reduce hyperexcitability in the disease.

Finding magneto. Researchers are using transcranial magnetic stimulation to evaluate in part whether mexiletine and retigabine reduce hyperexcitability in people with the disease.[Image: NIH].

Kaczmareks team is now hoping to do just that by developing a SLACK activator. The project is ongoing.

In the meantime, clinicians are aiming to reduce hyperexcitability in people with ALS by repurposing existing medicines in hopes to treat the disease. Brian Wainger is leading an effort to determine whether the epilepsy drug retigabine may be helpful in ALS. The drug, identified by Wainger as a potential treatment while in the laboratory of Kevin Eggan, may help normalize the activity of motor neurons by opening up Kv7 potassium channels in people with the disease (see April 2016 news; ; Wainger et al., 2014).

Across the US, the University of Washingtons Michael Weiss is taking a different approach. He is evaluating whether mexiletine, a sodium channel blocker, may reduce hyperexcitability in people with the disease (see March 2016 news). Both strategies are currently at the phase 2 stage.

In a disease that has a selective neuronal vulnerability like ALS, says Wainger, I think it is likely that the electrophysiological properties of the neuron are going to be related to the degenerative nature of the disease. So normalizing those properties may have a good chance of being helpful.

References

Zhang Y, Ni W, Horwich AL, Kaczmarek LK. AnALS-associatedmutantSOD1rapidlysuppressesKCNT1 (Slack) Na+-activated K+ channels in Aplysia neurons. J Neurosci. 2017 Jan 24. pii: 3102-16. [PubMed]

Fritz E, Izaurieta P, Weiss A, Mir FR, Rojas P, Gonzalez D, Rojas F, Brown RH Jr, Madrid R, van Zundert B. MutantSOD1-expressing astrocytes release toxic factors that trigger motoneuron death by inducing hyperexcitability. J Neurophysiol. 2013 Jun;109(11):2803-14. 2013 Mar 13. [PubMed].

Hadzipasic M, Tahvildari B, Nagy M, Bian M, Horwich AL, McCormick DA. Selective degeneration of a physiological subtype of spinal motor neuron in mice with SOD1-linked ALS. Proc Natl Acad Sci U S A. 2014 Nov 25;111(47):16883-8. [PubMed].

Wainger BJ, Kiskinis E, Mellin C, Wiskow O, Han SS, Sandoe J, Perez NP, Williams LA, Lee S, Boulting G, Berry JD, Brown RH Jr, Cudkowicz ME, Bean BP, Eggan K, Woolf CJ.Intrinsic membrane hyperexcitability of amyotrophic lateral sclerosis patient-derived motor neurons. Cell Rep. 2014 Apr 10;7(1):1-11.[PubMed]

van Zundert B, Peuscher MH, Hynynen M, Chen A, Neve RL, Brown RH Jr, Constantine-Paton M, Bellingham MC. Neonatal neuronal circuitry shows hyperexcitable disturbance in a mouse model of the adult-onset neurodegenerative disease amyotrophic lateral sclerosis. J Neurosci.2008 Oct 22;28(43):10864-74. [PubMed].

Barcia G, Fleming MR, Deligniere A, Gazula VR, Brown MR, Langouet M, Chen H, Kronengold J, Abhyankar A, Cilio R, Nitschke P, Kaminska A, Boddaert N, Casanova JL, Desguerre I, Munnich A, Dulac O, Kaczmarek LK, Colleaux L, Nabbout R. De novo gain-of-function KCNT1 channel mutations cause malignant migrating partial seizures of infancy. Nat Genet. 2012 Nov;44(11):1255-9. [PubMed].

Heron SE, Smith KR, Bahlo M, Nobili L, Kahana E, Licchetta L, Oliver KL, Mazarib A, Afawi Z, Korczyn A, Plazzi G, Petrou S, Berkovic SF, Scheffer IE, Dibbens LM. Missense mutations in the sodium-gated potassium channel gene KCNT1 cause severe autosomal dominant nocturnal frontal lobe epilepsy. Nat Genet. 2012 Nov;44(11):1188-90. [PubMed].

Martin HC, Kim GE, Pagnamenta AT, Murakami Y, Carvill GL, Meyer E, Copley RR, Rimmer A, Barcia G, Fleming MR, Kronengold J, Brown MR, Hudspith KA, Broxholme J, Kanapin A, Cazier JB, Kinoshita T, Nabbout R; WGS500 Consortium., Bentley D, McVean G, Heavin S, Zaiwalla Z, McShane T, Mefford HC, Shears D, Stewart H, Kurian MA, Scheffer IE, Blair E, Donnelly P, Kaczmarek LK, Taylor JC. Clinical whole-genome sequencing in severe early-onset epilepsy reveals new genes and improves molecular diagnosis. Hum Mol Genet. 2014 Jun 15;23(12):3200-11. [PubMed].

Raoul C, Estvez AG, Nishimune H, Cleveland DW, deLapeyrire O, Henderson CE, Haase G, Pettmann B. Motoneuron death triggered by a specific pathway downstream of Fas. potentiation by ALS-linked SOD1 mutations. Neuron. 2002 Sep 12;35(6):1067-83. [PubMed].

LeeS, Shang Y, Redmond SA, Urisman A, Tang AA, Li KH, Burlingame AL, Pak RA, Jovii A, Gitler AD, Wang J, Gray NS, Seeley WW, Siddique T, Bigio EH,LeeVM, Trojanowski JQ, Chan JR, Huang EJ. Activation of HIPK2 Promotes ER Stress-Mediated Neurodegeneration in Amyotrophic Lateral Sclerosis. Neuron. 2016 Jul 6;91(1):41-55. [PubMed].

Song Y, Nagy M, Ni W, Tyagi NK, Fenton WA, Lpez-Girldez F, Overton JD, Horwich AL, Brady ST. Molecular chaperone Hsp110 rescues a vesicle transport defect produced by an ALS-associated mutant SOD1 protein in squid axoplasm. Proc Natl Acad Sci U S A. 2013 Apr 2;110(14):5428-33. [PubMed].

Fujisawa T, Takahashi M, Tsukamoto Y, Yamaguchi N, Nakoji M, Endo M, Kodaira H, Hayashi Y, Nishitoh H, Naguro I, Homma K, Ichijo H. The ASK1-specific inhibitors K811 and K812 prolong survival in a mouse model of amyotrophic lateral sclerosis. Hum Mol Genet. 2016 Jan 15;25(2):245-53. [PubMed].

Brown MR, Kronengold J, Gazula VR, Chen Y, Strumbos JG, Sigworth FJ, Navaratnam D, Kaczmarek LK. Fragile X mental retardation protein controls gating of the sodium-activated potassium channel Slack. Nat Neurosci. 2010 Jul;13(7):819-21. [PubMed].

Zhang Y, Brown MR, Hyland C, Chen Y, Kronengold J, Fleming MR, Kohn AB, Moroz LL, Kaczmarek LK. Regulation of neuronal excitability by interaction of fragile X mental retardation protein with slack potassium channels. J Neurosci. 2012 Oct 31;32(44):15318-27. [PubMed].

Kim GE, Kronengold J, Barcia G, Quraishi IH, Martin HC, Blair E, Taylor JC, Dulac O, Colleaux L, Nabbout R, Kaczmarek LK. Human slack potassium channel mutations increase positive cooperativity between individual channels. Cell Rep. 2014 Dec 11;9(5):1661-72. [PubMed].

disease-als hyperexcitability mexiletine retigabine SOD1 topic-preclinical topic-researchmodels

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A key ion channel may SLACK off in ALS – ALS Research Forum

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Stanford scientists create glow-in-the-dark mice, may advance gene therapies – The Indian Express

By: PTI | Updated: February 20, 2017 6:53 pm Not only did mRNA technique make the mouse glow, it also later ran around, completely unaware of the complex series of events that had just taken place within its body, researchers said. ( Image for representation, Source: Youtube)

Stanford scientists have successfully developed glow-in-the-dark mice using compounds that create proteins responsible for lighting up fireflies, an advance that may pave the way for new gene therapies.

Timothy Blake, a postdoctoral fellow at Stanford University in the US refined compounds that carry instructions for assembling the protein that makes fireflies light up and delivered them into the cells of an anaesthetised mouse.

Watch all our videos from Express Technology

Not only did the technique make the mouse glow, it also later woke up and ran around, completely unaware of the complex series of events that had just taken place within its body, researchers said.

This success could mark a significant step forward for gene therapy. It is hard enough getting these protein instructions, called messenger RNA (mRNA), physically into a cell. It is another hurdle altogether for the cell to actually use them to make a protein. If the technique works in people, it could provide a new way of inserting therapeutic proteins into diseased cells.

Its almost a childlike enthusiasm we have for this, said Robert Waymouth, a professor at Stanford. The code for an insect protein is put into an animal and that protein is not only synthesised in the cells but its folded and it becomes fully functional, capable of emitting light, said Waymouth. Although the results are impressive, this technique is remarkably simple and fast. Unlike traditional gene therapy that permanently alters the genetic makeup of the cell, mRNA is short-lived and its effects are temporary.

The transient nature of mRNA transmission opens up special opportunities, such as using these compounds for vaccination or cancer immunotherapy. Gene therapy is a decades-old field of research that usually focuses on modifying DNA, the fundamental genetic code. That modified DNA then produces a modified mRNA, which directs the creation of a modified protein.

Also Read:Gene-editing cell therapy saves two babies from cancer

The current work skips the DNA and instead just delivers the proteins instructions. They used a novel, deceptively straightforward creation, called charge-altering releasable transporters (CARTs). What distinguishes this polycation approach from the others, which often fail, is the others dont change from polycations to anything else, said Paul Wender, professor at Stanford.

Whereas, the ones that were working with will change from polycations to neutral small molecules. That mechanism is really unprecedented, Wender said. As part of their change from polycations to polyneutrals, CARTs biodegrade and are eventually excreted from the body.

One application of this technology is vaccination. At present, vaccines require introducing part of a virus or an inactive virus into the body in order to elicit an immune response. CARTs could potentially cut out the middleman, directly instructing the body to produce its own antigens.

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Stanford scientists create glow-in-the-dark mice, may advance gene therapies – The Indian Express

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Global Gene Therapy Technologies, Markets and Companies 2016-2026 – Research and Markets – PR Newswire (press release)

Gene therapy technologies are described in detail including viral vectors, nonviral vectors and cell therapy with genetically modified vectors. Gene therapy is an excellent method of drug delivery and various routes of administration as well as targeted gene therapy are described. There is an introduction to technologies for gene suppression as well as molecular diagnostics to detect and monitor gene expression.

Clinical applications of gene therapy are extensive and cover most systems and their disorders. Full chapters are devoted to genetic syndromes, cancer, cardiovascular diseases, neurological disorders and viral infections with emphasis on AIDS. Applications of gene therapy in veterinary medicine, particularly for treating cats and dogs, are included.

Research and development is in progress in both the academic and the industrial sectors. The National Institutes of Health (NIH) of the US is playing an important part. As of 2015, over 2050 clinical trials have been completed, are ongoing or have been approved worldwide.A breakdown of these trials is shown according to the geographical areas and applications.

The markets for gene therapy are difficult to estimate as there is only one approved gene therapy product and it is marketed in China since 2004. Gene therapy markets are estimated for the years 2016-2026. The estimates are based on epidemiology of diseases to be treated with gene therapy, the portion of those who will be eligible for these treatments, competing technologies and the technical developments anticipated in the next decades. In spite of some setbacks, the future for gene therapy is bright.The markets for DNA vaccines are calculated separately as only genetically modified vaccines and those using viral vectors are included in the gene therapy markets

Profiles of 188 companies involved in developing gene therapy are presented along with 233 collaborations. There were only 44 companies involved in this area in 1995. In spite of some failures and mergers, the number of companies has increased more than 4-fold within a decade. These companies have been followed up since they were the topic of a book on gene therapy companies by the author of this report.

Key Topics Covered:

Part I: Technologies & Markets

1. Introduction

2. Gene Therapy Technologies

3. Clinical Applications of Gene Therapy

4. Gene Therapy of Genetic Disorders

5. Gene Therapy of Cancer

6. Gene Therapy of Neurological Disorders

7. Gene Therapy of Cardiovascular Disorders

8. Gene therapy of viral infections

9. Research, Development and Future of Gene Therapy

10. Regulatory, Safety and Ethical Issues of Gene Therapy

11. Markets for Gene Therapy

12. References

Part II: Companies

13. Companies involved in Gene Therapy

For more information about this report visit http://www.researchandmarkets.com/research/jtwqds/gene_therapy

Media Contact:

Laura Wood, Senior Manager press@researchandmarkets.com

For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900

U.S. Fax: 646-607-1907 Fax (outside U.S.): +353-1-481-1716

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/global-gene-therapy-technologies-markets-and-companies-2016-2026—research-and-markets-300407996.html

SOURCE Research and Markets

http://www.researchandmarkets.com

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Global Gene Therapy Technologies, Markets and Companies 2016-2026 – Research and Markets – PR Newswire (press release)

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What’s It Like Getting Health Care As A Black Transgender Person? – BuzzFeed News

Your Post Has Been Launched!

Fabulous! Don’t forget to share with your friends on Twitter and Facebook.

We want to hear your stories.

And its not for a lack of trying, either.

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Shoot, maybe you dont even have insurance, which means you cant get the health care you really need.

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Maybe they refused to use your preferred pronoun or made sweeping generalizations without ever actually asking about your lifestyle.

ID: 10528689

Maybe they said snide or even outright mean things to you. And because of this mistreatment, you knew that place wasnt the right one for you even if it was just to get checked up for a nagging cough or an eye exam. Maybe such an instance even forced you to travel two hours now just to see someone whos trans-friendly and knows what theyre doing.

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If so, please share your secrets with the class.

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You can submit your story (anonymously, if you want to) in the form below. You can also view the form here, which might be easier on mobile. Please share as much detail as you can vent to us! The more we know about your experience, the more likely it is that we can include you in a future post. Your story, whether related or unrelated to transition, could help other people with their own medical struggles.

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Bone Marrow – Structure, Function, Disease and More

1 of 2

Use to navigate.

This colored scanning electron micrograph (SEM) is showing the internal structure of a broken finger bone. Here, the periosteum (outer bone membrane, pink), compact bone (yellow) and bone marrow (red), in the medullary cavity, can be seen. Photo Credit: STEVE GSCHMEISSNER/Science Photo Library/Getty Images

Updated July 15, 2016.

Bone marrow is the soft, flexible connective tissue within bone cavities. A component of the lymphatic system, bone marrow functions primarily to produce blood cells and to store fat. Bone marrow is highly vascular, meaning that it is richly supplied with a large number of blood vessels. There are two categories of bone marrow tissue: red marrow and yellow marrow. From birth to early adolescence, the majority of our bone marrow is red marrow.

As we grow and mature, increasing amounts of red marrow is replaced by yellow marrow. On average, bone marrow can generate hundreds of billions of new blood cells every day.

Bone marrow is separated into a vascular section and non-vascular sections. The vascular section contains blood vessels that supply the bone with nutrients and transport blood stem cells and mature blood cells away from the bone and into circulation. The non-vascular sections of the bone marrow are where hematopoiesis or blood cell formation occurs. This area contains immature blood cells, fat cells, white blood cells (macrophages and plasma cells), and thin, branching fibers of reticular connective tissue. While all blood cells are derived from bone marrow, some white blood cells mature in other organs such as the spleen, lymph nodes, and thymus gland.

The major function of bone marrow is to generate blood cells. Bone marrow contains two main types of stem cells. Hematopoietic stem cells, found in red marrow, are responsible for the production of blood cells.

Bone marrow mesenchymal stem cells (multipotent stromal cells) produce the non-blood cell components of marrow, including fat, cartilage, fibrous connective tissue (found in tendons and ligaments), stromal cells that support blood formation, and bone cells.

In adults, red marrow is confined mostly to skeletal system bones of the skull, pelvis, spine, ribs, sternum, shoulder blades, and near the point of attachment of the long bones of the arms and legs. Not only does red marrow produce blood cells, but it also helps to remove old cells from circulation. Other organs, such as the spleen and liver, also filter aged and damaged blood cells from the blood. Red marrow contains hematopoietic stem cells that produce two other types of stem cells: myeloid stem cells and lymphoid stem cells. These cells develop into red blood cells, white blood cells, or platelets. (See, bone marrow stem cells).

Yellow marrow consists primarily of fat cells. It has poor vascular supply and is composed of hematopoietic tissue that has become inactive. Yellow marrow is found in spongy bones and in the shaft of long bones. When blood supply is extremely low, yellow marrow can be converted to red marrow in order to produce more blood cells.

If bone marrow becomes damaged or diseased, it can result in low blood cell production. Bone marrow disease can develop from bone marrow and blood cancers such as leukemia. Radiation exposure, certain kind of infections, and diseases such as aplastic anemia and myelofibrosis can also cause blood and marrow disorders. These diseases compromise the immune system and deprive organs and tissues of the life giving oxygen and nutrients they need. A bone marrow transplant may be done in order to treat blood and marrow diseases. In the process, damaged blood stem cells are replaced by healthy cells obtained form a donor. The healthy stem cells can be obtained from the donor’s blood or bone marrow. Bone marrow is extracted from bones such as the hip or sternum. Stem cells may also be obtained from umbilical cord blood to be used for transplantation.

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Bone Marrow – Structure, Function, Disease and More

Recommendation and review posted by Bethany Smith

‘Exciting’ new therapy shows promising results for hemophilia … – CTV News

CTVNews.ca Staff Published Friday, February 17, 2017 10:00PM EST Last Updated Friday, February 17, 2017 10:28PM EST

A small gene therapy trial involving several Canadian patients is offering new hope to people living with hemophilia, a rare and potentially fatal genetic disorder.

Hemophilia patients can suffer prolonged or uncontrollable bleeding, even after minor injuries. That’s because they lack blood clotting factors, or proteins.

There are two types of hemophilia, A and B, and both are very rare disorders. Hemophilia A affects an estimated 2,500 Canadians, while hemophilia B affects about 600 Canadians, according to the Canadian Hemophilia Society.

A new gene therapy developed at the Children’s Hospital in Philadelphia has produced very encouraging results. Preliminary research suggests that a single dose of the experimental therapy may help patients with hemophilia B, which involves a deficiency of blood clotting factor IX.

The therapy involves using a gene engineered to replace the faulty one in people with hemophilia. The engineered gene is placed into an inactivated virus and then infused into the liver, where it helps the body produce a clotting factor that prevents bleeding.

“It poses the possibility for a one-time treatment that would bepotentially life-altering for the patient,” Dr. Lindsey George, a hematologist at The Children’s Hospital of Philadelphia, told CTV News.

Scientists found that after just one dose, none of the nine patients involved in the trial suffered any bleeding for up to a year. Although more research and larger studies are needed to confirm the benefits of the therapy, researchers are very encouraged.

“It is very exciting, certainly from my standpoint as a clinical investigator,” Dr. George said.

Dr. Jerry Teitel, the medical director of the Hemophilia Treatment Centre at St. Michael’s Hospital in Toronto, who collaborated with the Philadelphia researchers, called it “a revolutionary therapy.”

The results so far are wonderful, in fact even better than what we had dared to hope,” he said.

Of the nine patients who’ve received the treatment, four are Canadian.

Among them is John Konduros, a 52-year-old bakery owner in Cambridge, Ont. As a lifelong hemophiliac, he has always lived in fear of any bumps, cuts or bruises that could cause internal bleeding, disability — and even death.

“It has probably affected every single part of my life, from being a kid to now,” he told CTV News. “If you ever saw me as a kid, I was never in a group. I was always on the sides.”

Konduros said it was common for him to miss two or three weeks of school if another kid happened to kick him in the leg while they were playing. In one class photo, he is seen with a big bruise under his right eye – another side effect of his condition.

But since Konduros received the experimental treatment about eight months ago, he has not had any dangerous bleeds.

“I’m extremely happy in the sense of massive relief. I feel like I don’t have to be as vigilant or worrisome about everything and anything that’s going on around me,” he said.

So far, the immune systems of two trial patients have reacted to the treatment, but scientists say there were no serious side effects. Konduros has had no problems with the therapy.

“If the doctors wanted me to go down every weekend for more tests to accelerate anything I would say ‘sure’ because the improvement it gives anyone who has hemophilia is huge,” he said.

Dr. Teitel said scientists have “a long way to go” in developing a therapy that can help more hemophilia patients.

“We need to show that in large numbers, the results do hold up,” he said. “We need to show that the results last for a long period of time, not necessarily last a lifetime.”

With files from CTV’s medical specialist Avis Favaro and producer Elizabeth St. Philip

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‘Exciting’ new therapy shows promising results for hemophilia … – CTV News

Recommendation and review posted by simmons

Should We Die? – The Atlantic

So, you dont want to die? I asked Zoltan Istvan, then the Transhumanist candidate for president, as we sat in the lobby of the University of Baltimore one day last fall.

No, he said, assuredly. Never.

Istvan, an atheist who physically resembles the pure-hearted hero of a Soviet childrens book, explained that his life is awesome. In the future, it will grow awesomer still, and he wants to be the one to decide when it ends. Defying aging was the point of his presidential campaign, the slogan of which could have been Make Death Optional for Once. To (literally) drive the point home, he circled the nation in the Immortality Bus, a brown bus spray-painted to look like a coffin.

He knew hed lose, of course, but he wanted his candidacy to promote the cause of transhumanismthe idea that technology will allow humans to break free of their physical and mental limitations. His platform included, in part, declaring aging a disease. He implanted a chip in his hand so he could wave himself through his front door, and he wants to get his kids chipped, too. Hed be surprised, he told me, if soon we dont start merging our children with machines. Hed like to replace his limbs with bionics so he can throw perfectly in water polo. Most of all, he wants to stick around for a couple centuries to see it all happen, perhaps joining a band or becoming a professional surfer, a long white beard trailing in his wake.

Istvan made his fortunes in the real-estate business, but in 2003, he was working as a reporter for National Geographic in Vietnam when he almost tripped a landmine. The experience shook him so badly he quit journalism and devoted his life to transhumanism. I thought, death is horrible, he told me. How can we get around it?

But his central goalpushing the human lifespan far beyond the record 122 years and possibly into eternityis one shared by many futurists in Silicon Valley and beyond. Investor Peter Thiel, who sees death as the great enemy of man, is writing checks to researchers like Cynthia Kenyon, who doubled the life-spans of worms through gene-hacking, as the Washington Post reported last April. Oracle founder Larry Ellison has thrown hundreds of millions toward anti-aging research, according to Inc magazine, and Google founders Larry Page and Sergey Brin launched the Google subsidiary Calico specifically with the goal of curing death. Under President Donald Trump, the quest for immortality might pick up steam: Among the candidates he is reportedly considering to head the Food and Drug Administration is Jim ONeill, who sits on the board of the anti-aging SENS Research Foundation.

Some life-extension endeavors are already here. Several companies already offer cryogenic freezing to people who wish to have their dead bodies cooled with liquid nitrogen and stored for centuries, with the hope that new medical technologies will by then be available to re-animate them. A British teenager who sued for the right to be cryogenically frozen after her death from cancer in October now floats in frosty slumber in a Michigan cryostat facility.

Meanwhile, scientists in California are expected to launch a clinical trial in which participants will have their blood cleaned of age-related proteins, the Guardian reported, with the goal of helping them live longer and healthier lives. A drug called rapamycin, which extended the lives of mice by a quarter, is also being tested. The thinking is, if we figure out what chemical event signals to the body that its time to wrap things up, said Sheldon Solomon, a psychology professor at Skidmore College, you could be at a certain age for a long time.

The billionaire technologists obsession with living forever can approach a sort of parody. Oracles Ellison once said, Death makes me very angry”suggesting this pillar of nature is just another consumer pain-point to be relieved with an app.

But lets assume, for the sake of argument, that it can be. Lets say human lives will soon get radically longeror even become unending. The billionaires will get their way, and death will become optional.

If we really are on the doorstep of radical longevity, its worth considering how it will change human society. With no deadline, will we still be motivated to finish things? (As a writer, I assure you this is difficult.) Or will we while away our endless days, amusing ourselves towell, the Process Formerly Known as Deathwhile we overpopulate the planet? Will Earth become a paradise of eternally youthful artists, or a hellish, depleted nursing home? The answers depend on, well, ones opinion about the meaning of life.

I didnt realize how much mainstream support there was for eternal life until I had dinner with a friend who, its worth noting, is even more traditional than I amhes not even on Twitter.

I interviewed this guy who wants to live forever, I said. Isnt that wild?

What do you mean? my friend asked. You dont want to live forever?

If he never died, he explained, he could finally pursue all the hobbies and dreams hes never had time for. Even alternate careers, like architecture. (Hes a lawyer.) Hes never quite understood calculus, but with all the time in the world, he could master it. He would take a sabbatical every four years to travel the world.

Ill admit, his passion for a long life of solving integrals and kayaking through rainforests did drag me closer to the immortality corner. Even if I extended my life by just a few years, I could finally get to the bottom of my Netflix and Pocket queues.

And I had been silently dismissing life-extension enthusiasts spiels about seeing their great-great-grandkids grow up, since I dont have kids and probably never will.

Butbutif I was certain I could stay sharp and energetic well into my 90s, maybe my stance on motherhood would change. I wouldnt worry so much about kids cutting into my productivity if my ability to produce was limitless. Sure, Id probably have a few sleepless nights and groggy days in the early years. (Unless, of course, Silicon Valley really gets cracking on those robot wet-nurses.) But once Olga Jr. was out of the house and working as a Martian News correspondent or whatever, I could more than make up for lost time.

This feeling of abundant possibility is one of the chief motivations of the pro-longevity crowd. Projects and ambitions like mastering every musical instrument in the orchestra, writing a book in each of all the major languages, planting a new garden and seeing it mature, teaching ones great-great-grandchildren how to fish, traveling to Alpha Centauri, or just seeing history unfold over a few hundred years are not realistic: there is simply not enough time to achieve them given current life expectancy, wrote Nick Bostrom, an Oxford philosopher and grand-daddy of life-extension (so to speak), with fellow philosopher Rebecca Roache in 2008. But, they continue, if we could reasonably expect from an early age to live indefinitely, we could embark on projects designed to keep us occupied for hundreds or thousands of years.

Among the many downsides of dying is the prospect of never reaching ones full potential. Right now, Im projected to die when Im about 82. But what if it takes me until I’m 209 to write the great American blog post?

Still, a common fear about life in our brave, new undying world is that it will just be really boring, says S. Matthew Liao, director of the Center for Bioethics at New York University. Life, Liao explained, is like a partyit has a start and end time. We get excited because the partys going on for an hour, and we dont want to miss it. We try to make the most of it while were there.

But imagine theres a party that doesnt end, he continued. It would be bad, because youd think, I could go there tomorrow, or a month from now. Theres no urgency to go to the party anymore.

The Epicureans of ancient Greece thought about it similarly, Solomon said. They saw life as a feast: If you were at a meal, youd be satiated, then stuffed, then repulsed, he said. Part of what makes each of us uniquely valuable is the great story. We have a plot, and ultimately it concludes.

Dan McAdams, a psychology professor at Northwestern University, explains that people make sense of their lives through narrative arcs. Without an ending, there cant be a story. How would we process life events differently, given infinite do-overs? For example, because we have a vague sense that people are supposed to die at roughly 80, we now grieve people who die at 20 more than those who die at 78. But if people began living to 500, that might change, McAdams pointed out. There might be far more tragedy in the world if were mourning the loss of every 90-year-old the way we now would a child. Were just so much trained by evolution and culture to know that our life is going to be relatively short and constrained, he said, and to be somewhat cautious so we dont screw it all up. (Of course, if technology also makes us smarter as it makes us live longer, who knows what types of new arcs well construct for ourselves.)

Bostrom dismisses the thought that theres something about impending death that adds meaning or motivation to our days. It often seems the young are most energetically pursuing different kinds of activities, and the closer you get to death, the more people lean back, he told me. Partly its due to their reduced energy and health.

Which, of course, he hopes we can fix.

Once living longer becomes possible, who will get to do it? Istvan believes life-extension technology should be available to everyone, not just the wealthy. He supports a universal health-care system with life extension as one of its core benefits. (Health-care costs wouldnt spiral out of control, he and some others think, because the longer-living humans would also be healthier. Istvan plans to pay for this universal Zoltancare by selling government land in the western United States.)

Others believe that soon after life-extending technology becomes available, the price will drop rapidly and it will become attainable by mostjust as occurred with personal computers.

But the worry in the short-term, is what happens? The rich could get richer and the poor could get poorer, Liao said. Because the rich could afford to extend their lives first, and life-extenders could amass more resources over the course of their long lives, income inequality could grow even more profound.

Then again, thats how things work now. If someone comes up with a new cancer drug, we dont say lets not use it until every person has access to it, Bostrom told me. By that logic, we should stop kidney transplants.

Even if eternal life gets equitably distributed, theres still the problem of what to do with all the excess centenarians running around. Eventually, were going to run out of room here on Earth. One solution would be to dramatically curtail reproduction, focusing instead on the health and longevity of those already here. As the philosopher Jan Narveson put it, we are in favor of making people happy, but neutral about making happy people. That might mean, though, that you wont have a great-great-great-grandkid to attend the dance-recitals of.

There is a chance that worrying less about death might short-circuit our naturally tribalist natures, easing resource-allocation issues in the process. Solomon, the Skidmore psychologist, researches terror management theory, which suggests the knowledge of our eventual demise makes people psychologically retrench. Being reminded of death causes study subjects to adhere more firmly to their existing worldview, mistrust outsiders more, and even to, ahem, support charismatic leaders who may not be very qualified. So in some ways, eliminating the prospect of death might make us want to ratify all the climate treaties and equitably divvy up the worlds food supply.

… That is, of course, unless immortality has the opposite effect, making us paranoid that well die too soon for no reason. After all, even if we can eliminate aging, we cant eliminate chance. Lets say you expected to live to 5,000 and your heads being frozen, theres a power outage, and it turns into a pile of mush, Solomon said. We might become even more hyper-vigilant.

Liao and others think one answer to the overcrowding problem might be interstellar space travelwhich, they assume, will be invented by then. When Earth turns into an overpopulated dump, Liao says, the immortal can just hop between planets.

I told him an eternity spent on Venus among youthful billionaires does not appeal to me.

What if all your friends go to Venus? he asked. He offered an earthly comparison: Youll be here while everyones in Brooklyn?

(Everyones already in Brooklyn, though, and Im still here in Northern Virginia.)

Space travel is also how Liao envisions us overcoming the boredom problem. Right now, the journey between solar systems is too long for a human to accomplish in a normal lifespan, but with life extension, that wont be a concern anymore. We wont run out of things to do, the thinking goes, because there will always be another planet to explore. Well all cheerfully grow old aboard our interstellar minivan.

And in general, Liao explained, humans engage in lots of pleasures that arent repetitive, like forming new relationships, making music, learning things, and experiencing natural wonders.

If thats what human existence is about, and you can continue to do that, why not be able to live longer? he asked me.

I guess I do like hiking, I said.

You might even enjoy hiking on Mars, he said.

Eh, dont push it.

* * *

The somber side to the debate is whether life extension will cause us to lose our appreciation for natural human vulnerability. In other words, society might begin to preference those who have swallowed anti-aging drugs, making un-enhanced humans a sort of rotting underclass.

Parents who have babies with mild disabilities might be blamed for not doing Gattaca, as Liao puts it. (Istvans platform reads, Develop science and technology to be able to eliminate all disabilities in humans who have them.) Well have to wrestle with whether those who dont take fountain-of-youth pills should be charged more for health insurance. Worse yet, by jetting off to a new planet, the enhanced and immortal could abandon Earth to mere mortals, the cruelest and most extreme form of segregation.

Life-extensionists zeal for perfect cells does, to some, sound like an invective against uniqueness. Thats what Melinda Hall, a philosophy professor at Stetson University and author of a recent book about transhumanism, takes issue with. People with disability are saying, this is a primary part of my identity, she told me, so when youre saying you want to get rid of disability, it sounds genocidal.

Istvan dismisses disability-rights advocates as a fringe minority, saying I would bet my arm that the great majority of disabled people will be very happy when transhumanist technology gives them the opportunity to fulfill their potential. (Betting your arm is, of course, no biggie when you can just get a bionic one.)

In general, Hall said, the transhumanists have the wrong idea about the problems facing humanity. People are going to be starving and dying, but were going to build a colony on Mars? she said, Thats going to cost billions of dollars, and I think that should be spent somewhere else.

Of course, that wont stop the billionaires from following their dreams. Perhaps our best hope is that on the path to immortality, theyll discover something useful to broader swathes of society. Metformin, an old diabetes drug recently shown to extend the life of animals, is now being tested as an anti-aging pill. If it really does allow people to stay healthy in old age, some would regard it a public health revolutioneven if it fails to help Peter Thiel meet his cyborg-descendants in 2450.

In that way, todays life extensionists might follow the proud tradition of other explorers who shot for another galaxy and ended up straddling the moon. The alchemists write about trying to find elixirs of gold and immortality. They never find that, but they discovered chemistry, Solomon said. Ponce de Leon never found the fountain of youth, but he found Florida.

See the rest here:
Should We Die? – The Atlantic

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Chief justice cheers turnout at Longview off-site session – Longview News-Journal

Fifth-grader Sophia Yastrebov craned her neck Thursday to reach the microphone and ask Texas Supreme Court justices a question they probably ask themselves from time to time.

Do you ever have trouble separating your personal preferences from a persons legal rights? the Longview home-school student asked during a question-and-answer session after the courts Longview session in the Belcher Center at LeTourneau University.

Eleven-year court veteran Justice Phil Johnson fielded that one.

The answer to that is, sometimes, he told Sophia and the audience of more than 1,000 high school and college students, hundreds of lawyers, scores of judges and others from across the region who came to see the states highest civil court hear two cases.

In all, some 1,400 people saw a scene Thursday they would have had to travel to Austin to see before 1997, when voters changed the Texas Constitution to allow off-site sessions.

In answering Sophias on-point question, Johnson confessed he might enter a case thinking one way.

But you research the law, he said. You just come out differently than you would prefer to come out. The answer is, we have a system of laws that has to be predictable. And people have to depend on them.

That was just one lesson but a major one that the nine justices said they wanted to leave behind. The judges acknowledged their job is an obscure one to most Texans, hopefully less so after each of their roughly twice-yearly out-of-town sessions.

Theyve been in mystery for too long, retired Longview plumber Peter Saccoccio said after watching the two cases be heard either of which could set legal precedent.

Making all Texas courts transparent is not merely a campaign promise for the justices, all of whom are Republicans.

Weve gone to high schools and universities across the state, Chief Justice Nathan Hecht told a lunch audience after the morning session. But the best I can remember is, this is the best off-site meeting we have held.

Hecht credited Longviews John and Susan Coppedge, whom he met in 1987, the year he campaigned for his first election to the Texas Supreme Court, for sharing his love of making Texans more aware of their judiciary.

Coppedge is recognized statewide as an expert on the Texas judiciary, often shepherding hopefuls for the Supreme Court, Court of Criminal Appeals and local appellate venues through their political campaigns.

Thursdays events, which included all nine justices teaching afternoon continuing education classes for lawyers in the Gregg County Courthouse, climaxed a two-year effort by the retired surgeon.

Hecht and the court are mandating that all Texas courts go online, with electronic filing implemented in stages. Civil courts are wrapping up that change now, and criminal courts are to be added beginning July 1.

By 2019, all of the court records in all of the trial courts other than justices of the peace and municipal courts will be electronic, Hecht said, adding the next step after that will be opening that portal to all residents.

We need to open up the court records, so people can see the cases that are being filed, he said.

The court will issue rulings from the morning sessions by the end of its term this summer. At roughly one hour apiece, the hearings brought a reunion of sorts for the justices.

Former Supreme Court Justice Craig Enoch and former Chief Justice Wallace Jefferson were squaring off in an inheritance dispute that had wended its way up through the trial and appellate courts in Fort Worth, Waco and Austin.

And now were in Longview, Enoch noted as he began his 20-minute allotment to speak.

Enoch represents a family claiming the woman who was married to their grandfather had been unduly influenced to alter her will by a niece and nephew to whom she is related by blood. The Kinsel family is claiming their stepgrandmother, Lesey Kinsel, was unduly influenced by her nephew and niece, Bob Oliver and Jane Lindsey, while living with dementia.

Justice Eva Guzman stopped Enoch at one point, asking whether someone who has lost competence can regain it.

Enoch replied that medical testimony in the trial record says incompetence is incurable despite good days and bad days.

Enoch said the stepgrandmother had amended her will three times through the years.

All of a sudden, she makes a significant change to the trust, he said, adding the woman dropped the Kinsels from her will and sold her ownership in their grandfathers ranch under direction of the niece and nephew and a law firm that also is being sued by the Kinsels. Once you have undue influence, then any change is improper.

Jefferson represents that law firm, Jackson Walker.

He said the niece and nephew brought their aunt, the Kinsels stepgrandmother, to his clients office in 2007.

The first thing he did was separate Lesey from Jane and Bob, Jefferson said. And he brought in a second lawyer, as witness, while he asked questions to see if she was competent.

The Kinsels won a $3 million jury verdict at the trial court level, but it was reversed on appeal because that court ruled a claim of interference with inheritance rights doesnt exist.

A ruling for the Kinsel family would establish such a right.

The second case involves a doctor who asked to be dismissed from a wrongful death suit under a claim of governmental immunity.

Dr. Leah Anne Gonski Marino was in a residency program in 2015 at a University of Texas Physicians Clinic in Houston, but she was paid by the nonprofit UT Health Systems Foundation.

Her claim of governmental immunity led to her dismissal by the trial court, but an appeals court rejected that dismissal on grounds the nonprofit foundation is not a governmental entity and doesnt control the doctors work.

The lawsuit arose from a hormone injection Marino gave the plaintiffs pregnant daughter, when the daughters regular physician was not available.

The pregnant woman later developed breathing problems and died, along with her unborn twins, en route to a hospital.

Liability typically follows control, attorney Joseph Gourrier argued for the mother and grandmother suing the doctor.

The justices peppered Gourrier, and defense counsel John Strawn Jr., with questions trying to pin down whether the UT medical foundation is a governmental entity. Court members drew multiple citations by the attorneys of the bylaws, policies and other documents governing the assignment of residencies to young doctors under the arrangement of the two UT entities.

The case has implications statewide, including in Longview where Christus Good Shepherd Medical Center offers a medical residency program for doctors in partnership with UT Health Northeast near Tyler.

Justices eager to meet students, public Thursday

Texas Supreme Court will gavel-in at Longview’s Belcher Center Thursday

Texas Supreme Court to hear weighty issues in Longview

Security tight for visit by state’s highest court

Texas Supreme Court needed voters’ permission to travel

Texas Supreme Court Longview stop expected to draw thousands

Originally posted here:
Chief justice cheers turnout at Longview off-site session – Longview News-Journal

Recommendation and review posted by sam


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