Researchers at Baylor College of Medicine have discovered a new mechanism that helps maintain and repair bones in adults. Ultimately, this could help develop new therapeutic strategies to improve bone healing.

Knee Bones

Osteoporosis is a skeletal disease characterized by reduced bone density and changes in the microarchitecture of bones. These changes weaken the bone and increase the risk of fracture. This disease develops particularly in older people. Today, a new study could eventually lead to the development of therapeutic strategies to improve bone healing in these patients. According to the results published in the journal Cell Stem Cell on the 5th December, 2019 researchers have discovered a new mechanism that contributes to the maintenance and repair of bones in adults.

Read Also: HGH Is Now A Solid Treatment For Osteoporosis According To Studies

Adult bone repair relies on the activation of bone stem cells, which still remain poorly characterized. Bone stem cells have been found both in the bone marrow inside the bone and also in the periosteum the outer layer of tissue that envelopes the bone. Previous studies have shown that these two populations of stem cells, although they share many characteristics, also have unique functions and specific regulatory mechanisms. said Dr. Dongsu Park, assistant professor of molecular and human genetics, pathology and immunology at Baylor College of Medicine, where the study was conducted.

Of these two populations, periosteal stem cells are the least known. Although the scientists know that this is a heterogeneous population of cells that can contribute to the thickness, formation and repair of bone fractures, no one has yet been able to distinguish between the different subtypes of bone stem cells in order to study the regulation of their different functions.

Here, however, Dongsu Park and colleagues were able to develop a technique in mice to identify different subpopulations of periosteal stem cells, define their contribution to the repair of bone fractures and identify the specific factors that regulate their migration and proliferation under physiological conditions.

In rats, they discovered specific markers for this class of cells. They identified a specific subset of stem cells that contribute to lifelong bone regeneration in adults. They also observed that periosteal stem cells react to inflammatory molecules, chemokines, which are normally produced in bone injuries.

Read Also: The Exciting Future of Joint and Cartilage Repair

In detail, periosteal stem cells have receptors that bind to the CCL5 chemokine. The CCL5 chemokine sends a signal to the cells to migrate to the injured bone and repair it. By suppressing the CCL5 gene in rats, the researchers found defects in bone repair that delayed healing. However, when they gave CCL5 to rats that had lost CCL5, the bones recovered faster.

Our findings contribute to a better understanding of the healing of adult bones. We believe this is one of the first studies to show that bone stem cells are heterogeneous and that different subtypes have unique properties that are regulated by specific mechanisms, said Dongsu Park. We have identified markers that allow us to distinguish between the subtypes of bone stem cells and have investigated what each subtype contributes to bone health. The understanding of how the functions of bone stem cells are regulated offers the possibility of developing new therapeutic strategies for the treatment of bone damage in adults.

Read Also: Implants from Own Stem Cells May Offer Solution to Back Pain, Researchers Say

In the long term, these findings may therefore have potential therapeutic applications, particularly in people with osteoporosis or diabetes.Indeed, people with diabetes may be prone to falls and fractures due to possible neurological, visual or renal complications. In addition, bone fragility in diabetics is likely to be due to changes in bone remodeling and, in particular, an increase in bone resorption.

https://www.cell.com/cell-stem-cell/fulltext/S1934-5909(19)30458-8?

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Researchers at Baylor College of Medicine Discover How to Improve Bone Repair - Gilmore Health News

Bone Marrow Stem Cells Comments Off on Researchers at Baylor College of Medicine Discover How to Improve Bone Repair – Gilmore Health News | January 4th, 2020

McLean (United States) (AFP)

When a person's immune system is impaired by a genetic disease, a bone-marrow transplant can be a powerful therapeutic tool, but with a major downside: during the first few months the recipient's defenses against viruses are severely weakened. The slightest infection can lead to a hospital trip.

A still-experimental type of treatment known as T-cell therapy aims to assist during this vulnerable period -- the months during which the body is rebuilding its natural defenses. After two decades of clinical trials, the technology has been refined, and is being used to treat more and more patients, many of them children.

A boy named Johan is one of them.

Today he is a mischievous, smiling toddler with a thick shock of light-brown hair, who never tires, playfully tormenting the family's puppy, Henry.

There is no sign of the three-year-long medical and emotional roller-coaster ride he and his family, who live in an affluent Washington suburb, have been on.

The first traumatic surprise came with the results of a pregnancy test: Johan was not planned.

"That was a huge shock. I cried," said his mother, 39-year-old Maren Chamorro.

- Risky procedure -

She had known since childhood that she carried a gene that can be fatal in a child's first 10 years, chronic granulomatous disease (CGD).

Her brother died of it at the age of seven. The inexorable laws of genetics meant that Maren had a one in four chance of transmitting it to her child.

For their first children, she and her husband Ricardo had chosen in-vitro fertilization, allowing the embryos to be genetically tested before implantation.

Their twins Thomas and Joanna were born -- both disease-free -- seven and a half years ago.

But in Johan's case, a post-birth genetic test quickly confirmed the worst: he had CGD.

After conferring with experts at Children's National Hospital in Washington, the couple took one of the most important decisions of their lives: Johan would receive a bone-marrow transplant, a risky procedure but one that would give him a chance of a cure.

"Obviously, the fact that Maren had lost a sibling at a young age from the disease played a big role," Ricardo confided.

Bone marrow, the spongy tissue inside bones, serves as the body's "factory" for the production of blood cells -- both red and white.

- His brother's immune system -

Johan's white blood cells were incapable of fighting off bacteria and fungal infections. A simple bacterial infection, of negligible concern in a healthy child, could spread out of control in his young body.

Luckily, Johan's brother Thomas, six years old at the time, was a perfect match. In April 2018, doctors first "cleansed" Johan's marrow using chemotherapy. They then took a small amount of marrow from Thomas's hip bones using a long, thin needle.

From that sample they extracted "supercells," as Thomas calls them -- stem cells, which they reinjected into Johan's veins. Those cells would eventually settle in his bone marrow -- and begin producing normal white blood cells.

The second step was preventive cell therapy, under an experimental program led by immunologist Michael Keller at Children's National Hospital.

The part of the immune system that protects against bacteria can be rebuilt in only a matter of weeks; but for viruses, the natural process takes at least three months.

- Hurdles remain -

From Thomas's blood, doctors extracted specialized white blood cells -- T-cells -- that had already encountered six viruses.

Keller grew them for 10 days in an incubator, creating an army of hundreds of millions of those specialized T-cells. The result: a fluffy white substance contained in a small glass vial.

Those T-cells were then injected into Johan's veins, immediately conferring protection against the six viruses.

"He has his brother's immune system," said Keller, an assistant professor at Children's National.

Johan's mother confirmed as much: today, when Thomas and Johan catch a cold, they have the same symptoms, and for nearly the same amount of time.

"I think it's pretty cool to have immunity from your big brother," Maren Chamorro said.

This therapeutic approach -- boosting the body's immune system using cells from a donor or one's own genetically modified cells -- is known as immunotherapy.

Its main use so far has been against cancer, but Keller hopes it will soon become available against viruses for patients, like Johan, who suffer from depressed immune systems.

The chief obstacles to that happening are the complexity of the process and the costs, which can run to many thousands of dollars. These factors currently restrict the procedure to some 30 medical centers in the United States.

For Johan, a year and a half after his bone marrow transplant, everything points to a complete success.

"It's neat to see him processing things, and especially play outside in the mud," his mother said.

"You know, what a gift!"

Her only concern now is the same as any mother would have -- that when her son does fall ill, others in the family might catch the same bug.

2020 AFP

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The 'supercells' that cured an infant's grave genetic illness - FRANCE 24

Bone Marrow Stem Cells Comments Off on The ‘supercells’ that cured an infant’s grave genetic illness – FRANCE 24 | January 2nd, 2020

Meet the inflammation and immunity researcher studying the fundamental cellular mechanisms behind uncontrolled inflammatory responses to allergens

As the prevalence of allergic disease continues to rise worldwide, the work of immunologist Dr. Adam MacNeil has never been more important. By identifying novel targets in allergic inflammation to enable the development of innovative therapies, MacNeil and his team are pushing toward a healthier future. Were interested in allergic inflammation from two different branches, firstly, how the cells that contribute to inflammation emerge from the bone marrow, and secondly, how mature mast cells contribute to inflammatory mechanisms at the site of exposure, explains MacNeil, associate professor in the interdisciplinary Health Sciences department at Brock University, Canada.

Dr. Adam J. MacNeil, Associate Professor of Immunologyat Brock University's Department of Health Sciences.Pictured from left to rightare;Melissa Rouillard, Aindriu Maguire, Rob Crozier, Adam MacNeil, Jeremia Coish, Katie Hunter, Colton Watson, and Natalie Hicks. Image courtesy of theMacNeil Lab.

The MacNeil Lab investigates mechanisms in hematopoietic stem cells directing the maturation of the most well-known allergic mediator cellsmature mast cellsthat drive allergic inflammation. A key research goal for the team is to identify how an allergen activates a mast cell to create an inflammatory response.

Seeking to understand the signals that stimulate a progenitor cell to become a mast cell in different tissues, this research looks to determine the signaling pathways directing the epigenetic, and ultimately proteomic, profile of these cells1-3. To do this, cells are isolated and matured from bone marrow to create functional, phenotypical mast cells, which are primed with allergen-specific IgE molecules before addition of the allergen to activate the cells. The inflammatory response to the allergen, and the cell signaling processes that contribute to the inflammatory mechanisms, can then be measured through the secretion of histamines in degranulation mechanisms, or release of pro-inflammatory mediators such as cytokines, chemokines, and lipid metabolites.

Brock University

Being able to identify and sort cells with a specific immune profile requires tools capable of precision sorting of heterogeneous populations of cells. MacNeil expands: Were working with a heterogeneous population of cells in the bone marrow and trying to take only the stem cells out. So, it's a very small population within the total population of cells. Many of the assays that we want to do with that small population of cells are very well-suited to being sorted directly onto a 96-well plate where we can then actually conduct the experiment directly, knowing exactly how many cells are in each well and what the particular profile of those cells is. That makes the Sony SH800S a really strong tool for our lab.

When it comes to optimizing and streamlining the lab's work, Sony technology offers advantages over traditional methods. The traditional flow cytometer or cell sorter in any core lab is operated by a technician, and they're the only one allowed to touch it. That doesn't make for great learning opportunities for graduate students, and it's much better if they can actually interface with the instrument themselves, says MacNeil. The software and automation really allow for that to happen, but also adds to the robustness of the instrument. The way in which it has been designed means that it's pretty difficult to break it.

With an epigenetic approach to understanding how mast cells differentiate, and the effect of inhibiting specific signaling pathways in those cells, the MacNeil Lab uses sorted cells in functional assays such as immune cell profiling and cytokine secretion. Also, the cells can be sorted into plate-based assays for ChIP or RNA-Seq to assess their genetic profile. We're not only interested in sorting. We bought the device because it's robustly dynamic, explains MacNeil, referring to the Sony SH800S. You can look at data acquisition and not have to even use the sorting function at all in certain scenarios. There are many times that were simply interested in looking at the phenotype of our cells and not worried about sorting necessarily. Weve found this instrument to be very easy to use and to give us robust data in terms of the immune profile of our cells.

In addition, the SH800S microfluidic sorting chip helps to automate key stages of instrument setup and demonstrates versatility with a wide range of chip sizes, ranging from 70130 m, for sorting a variety of cells. The chip ultimately gets to the robustness of the instrument, explains MacNeil. Because of the chip, we have such peace of mind about how the instrument functions that we don't even worry about clogging of the instrument and all of the problems that the chip ultimately solves. If we do run into a problem, we can just change the chip. I certainly find the chip technology to be really well suited to our type of lab environment.

For MacNeil, the Sony SH800S Cell Sorter is a great fit for the lab, with a seamless software interface and great overall instrument design and modularity for easy plate-based sorting.MacNeil lab logocourtesy of the MacNeil Lab.

Working within the diverse multidisciplinary department at Brock University opens unique and fascinating research avenues not available to all immunologists and has led MacNeil to interesting collaborations and knowledge exchange on transdisciplinary projects.

As part of these broader research avenues, working with sociologist Prof. Terrance Wade and cardiovascular biologist Prof. Deborah OLeary, MacNeil also studies adverse experiences in childhood. The team is investigating whether such events may set the immunological stage for dysregulated inflammation in later life, through mechanisms involving stress-stimulated cortisol release that can shape how the immune system is responding4.

In another stream of collaborative immunological research, MacNeil collaborates with psychologist Prof. Anthony Bogaert to look at the role of the immune system in shaping sexual orientation as part of the fraternal birth order effect. This research looks at how early pregnancies stimulate the immune system to make antibodies against brain proteins in fetal males that may then affect their social behaviors in later life5. Its something I may not have expected to ever work on, says MacNeil. But when you come to a diverse department with a wide lens on health, these kinds of opportunities emerge. Were now interested in using the SH800S to test hypotheses for particular mechanisms underlying this phenomenon.

Looking ahead, MacNeil expects tissue heterogeneity to be a key issue to tackle in the field of immunology. Cell populations simply aren't uniform, he says. Mast cells in different locations in the body don't have exactly the same phenotype, and so, as our research proceeds and we continue to probe the role of the mast cell in allergic inflammation, we're very conscious that tissue heterogeneity is going to be a factor. But with such challenges come opportunities. Were ultimately interested in going into those tissues and trying to pull mast cells out. To do this, we would require an instrument like a cell sorter. Once the cells are sorted, we can interrogate their functional phenotype, including how they degranulate, secrete cytokines and metabolize lipids etc. toward one day potentially modulating their phenotype for the hundreds of millions affected by this inappropriate immune response, MacNeil concludes.

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Innovative therapies: Novel targets in allergic inflammation - SelectScience

Bone Marrow Stem Cells Comments Off on Innovative therapies: Novel targets in allergic inflammation – SelectScience | January 2nd, 2020

After years of ethical debates and breakthroughs in the lab, CRISPR has finally made its way to clinical trials. Researchers are now looking at whether the DNA-editing tool, as well as more conventional gene therapies, can effectively treat a wide array of heritable disorders and even cancers.

Theres been a convergence of the science getting better, the manufacturing getting much better, and money being available for these kinds of studies, says Cynthia Dunbar, a senior investigator at the National Heart, Lung, and Blood Institute. Its truly come of age.

CRISPR formally known as CRISPR-Cas9 has been touted as an improvement over conventional gene therapy because of its potential precision. CRISPR (clustered regularly interspaced short palindromic repeats) is a genetic code that, contained in a strand of RNA and paired with the enzyme Cas9, acts like molecular scissors that can target and snip out specific genes. Add a template for a healthy gene, and CRISPRs cut can allow the cell to replace a defective gene with a healthy one.

In April, scientists at the University of Pennsylvania announced they had begun using CRISPR for cancer treatments. The first two patients one with multiple myeloma, the other with sarcoma had cells from their immune systems removed. Researchers used CRISPR to genetically edit the cells in the lab, and then returned them back into their bodies.

On the other side of the country, Mark Walters, a blood and bone marrow transplant specialist at the University of California, San Francisco, Benioff Childrens Hospital in Oakland, is gearing up for trials that will use CRISPR to repair the defective gene that causes sickle cell disease. With CRISPR, once youve made that type of correction, [that cell] is 100 percent healthy, says Walters.

Another team is tackling the same disease using a type of hemoglobin, a protein in red blood cells, thats normally made only in fetuses and newborn babies. Researchers found that some adults continue to produce these proteins throughout their lives, and when those adults also have sickle cell disease, their symptoms are mild. So the international team used CRISPR to disable the gene that interferes with production of this hemoglobin, resuming its production and protecting the adult patients against sickle cell disease.

Several other CRISPR studies are in the works to treat a range of inherited disorders, including hemophilia and SCID-X1 (also known as X-linked severe combined immunodeficiency, the so-called bubble boy disease in which babies are born without a functioning immune system).

At St. Jude Childrens Research Hospital, a gene therapy trial cured Gael Jesus Pino Alva (pictured with his mother, Giannina) of SCID-X1, the bubble boy disease. (Credit: St. Jude Children's Research Hospital/Peter Barta)

The past year also saw success in a handful of experiments on conventional gene therapy. Instead of using CRISPR to repair disease-causing genes, these treatments use hollowed-out viruses to ferry healthy versions of genes into cells. Millions of these altered cells are released into the bloodstream or bone marrow in hopes that enough will land in the right places. But because scientists cant predict where the circulating genes may end up, this shotgun approach has had unintended, sometimes fatal, consequences including, in an earlier study, inadvertently activating leukemia-causing genes in patients treated for SCID-X1.

But in 2019, researchers learned that using a different type of virus one related to HIV to transport the genes may prevent these side effects. In an April study, researchers at St. Jude Childrens Research Hospital in Memphis, Tennessee, and UCSF Benioff Childrens Hospital in Oakland collected bone marrow from eight newborns with SCID-X1. They loaded corrective genes into the disabled HIV-related virus, which carried them into the patients bone marrow stem cells. The infants also received low doses of busulfan, a chemotherapy that gave the doctored stem cells room to grow. So far, we havent seen anything worrisome, says Ewelina Mamcarz, a pediatric oncologist at St. Jude who led the research team. The study recently added its 12th patient.

Gene therapy does have its momentum [back], says Mamcarz, reflecting on the fields setback after the earlier studys leukemia side effects. Theres so much that still needs to be done, and so many questions, she says. [But] this is how medicine evolves. We always want to be better than we were a week ago.

In the future, the hope is that gene therapy technologies will move beyond mending simple genetic mistakes and be used to combat big killers like diabetes or heart disease. [Those diseases are] more challenging, but a lot of them would benefit from knocking out a bad gene, says Dunbar.

For now, though, researchers are optimistic about the progress thats already been made. All of this has been very encouraging, says Dunbar. [And] for sickle cell in the U.S. and hemophilia in the developed world, these diseases may soon be solved.

[This story originally appeared in print as "Gene Therapy Gets Clinical."]

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Gene Therapies Make it to Clinical Trials - Discover Magazine

Bone Marrow Stem Cells Comments Off on Gene Therapies Make it to Clinical Trials – Discover Magazine | December 31st, 2019

In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering -- by editing her genome. Victoria Gray's recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research -- gene therapy. "I have hoped for a cure since I was about 11," the 34-year-old told AFP in an email.

"Since I received the new cells, I have been able to enjoy more time with my family without worrying about pain or an out-of-the-blue emergency." Over several weeks, Gray's blood was drawn so doctors could get to the cause of her illness -- stem cells from her bone marrow that were making deformed red blood cells. The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 -- pronounced "Crisper" -- a new tool informally known as molecular "scissors." The genetically edited cells were transfused back into Gray's veins and bone marrow. A month later, she was producing normal blood cells.

Medics warn that caution is necessary but, theoretically, she has been cured. "This is one patient. This is early results. We need to see how it works out in other patients," said her doctor, Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville. "But these results are really exciting." In Germany, a 19-year-old woman was treated with a similar method for a different blood disease, beta thalassemia. She had previously needed 16 blood transfusions per year.

Nine months later, she is completely free of that burden. For decades, the DNA of living organisms such as corn and salmon has been modified. But Crispr, invented in 2012, made gene editing more widely accessible. It is much simpler than preceding technology, cheaper and easy to use in small labs. The technique has given new impetus to the perennial debate over the wisdom of humanity manipulating life itself. "It's all developing very quickly," said French geneticist Emmanuelle Charpentier, one of Crispr's inventors and the cofounder of Crispr Therapeutics, the biotech company conducting the clinical trials involving Gray and the German patient.

Cures

Crispr is the latest breakthrough in a year of great strides in gene therapy, a medical adventure started three decades ago, when the first TV telethons were raising money for children with muscular dystrophy. Scientists practising the technique insert a normal gene into cells containing a defective gene. It does the work the original could not -- such as making normal red blood cells, in Victoria's case, or making tumor-killing super white blood cells for a cancer patient. Crispr goes even further: instead of adding a gene, the tool edits the genome itself.

After decades of research and clinical trials on a genetic fix to genetic disorders, 2019 saw a historic milestone: approval to bring to market the first gene therapies for a neuromuscular disease in the US and a blood disease in the European Union. They join several other gene therapies -- bringing the total to eight -- approved in recent years to treat certain cancers and an inherited blindness. Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution. "Twenty-five, 30 years, that's the time it had to take," he told AFP from Paris.

"It took a generation for gene therapy to become a reality. Now, it's only going to go faster." Just outside Washington, at the National Institutes of Health (NIH), researchers are also celebrating a "breakthrough period." "We have hit an inflection point," said Carrie Wolinetz, NIH's associate director for science policy.These therapies are exorbitantly expensive, however, costing up to $2 million -- meaning patients face grueling negotiations with their insurance companies. They also involve a complex regimen of procedures that are only available in wealthy countries.

Gray spent months in hospital getting blood drawn, undergoing chemotherapy, having edited stem cells reintroduced via transfusion -- and fighting a general infection. "You cannot do this in a community hospital close to home," said her doctor. However, the number of approved gene therapies will increase to about 40 by 2022, according to MIT researchers. They will mostly target cancers and diseases that affect muscles, the eyes and the nervous system.

Bioterrorism

Another problem with Crispr is that its relative simplicity has triggered the imaginations of rogue practitioners who don't necessarily share the medical ethics of Western medicine. Last year in China, scientist He Jiankui triggered an international scandal -- and his excommunication from the scientific community -- when he used Crispr to create what he called the first gene-edited humans. The biophysicist said he had altered the DNA of human embryos that became twin girls Lulu and Nana.

His goal was to create a mutation that would prevent the girls from contracting HIV, even though there was no specific reason to put them through the process. "That technology is not safe," said Kiran Musunuru, a genetics professor at the University of Pennsylvania, explaining that the Crispr "scissors" often cut next to the targeted gene, causing unexpected mutations. "It's very easy to do if you don't care about the consequences," Musunuru added. Despite the ethical pitfalls, restraint seems mainly to have prevailed so far.

The community is keeping a close eye on Russia, where biologist Denis Rebrikov has said he wants to use Crispr to help deaf parents have children without the disability. There is also the temptation to genetically edit entire animal species -- malaria-causing mosquitoes in Burkina Faso or mice hosting ticks that carry Lyme disease in the US. The researchers in charge of those projects are advancing carefully, however, fully aware of the unpredictability of chain reactions on the ecosystem.

Charpentier doesn't believe in the more dystopian scenarios predicted for gene therapy, including American "biohackers" injecting themselves with Crispr technology bought online. "Not everyone is a biologist or scientist," she said. And the possibility of military hijacking to create soldier-killing viruses or bacteria that would ravage enemies' crops? Charpentier thinks that technology generally tends to be used for the better. "I'm a bacteriologist -- we've been talking about bioterrorism for years," she said. "Nothing has ever happened."

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Year in Review: Gene Therapy Technology and a Milestone 2019 for Medical Research - News18

Bone Marrow Stem Cells Comments Off on Year in Review: Gene Therapy Technology and a Milestone 2019 for Medical Research – News18 | December 31st, 2019

The 2010s are coming to an end, and looking back there have been some pretty amazing advances and innovations in health and science.

Advances in prosthetic limbs

Prosthetic limbs have been around since ancient times. In Egypt, a prosthetic wooden toe was found on a mummy dating back 3,000 years. By the Dark Ages, inventors could incorporate hinges on prosthetic arms used by knights. In modern times, the field of prosthetics has turned to incorporating more technology into physical stand-ins for limbs. In the last several years, theres been a boom in advances that have led to the best and most useful prosthetics weve ever seen.

Reports from the early 2010s talked about the potential for new technology to allow people to control prosthetics with their minds and to receive sensory information from their devices. It may have been a reach in the early part of the decade, but now it is literally within grasp. There are new prosthetic hands being tested that give the user the ability to grab objects with their thoughts and even to sense the texture of what they are touching. New bionic hands allow the user to feel again by sending signals back to the brain about the things they are touching, like whether its hard or soft. Other research groups have been working on bionic arms that can move based on the users thoughts through a brain-computer interface. While these have demonstrated its possible to accomplish these goals in the lab, theres still more to be done before people can use these devices outside in the real world.

Many of these advanced prosthetics are still prototypes and may not reach the general population for a while. Luckily, cheaper 3D printers have made simple prosthetics more accessible. These are important because a prosthetic device can improve the quality of life for people. For example, this person has been printing prosthetic hands and arms for people in Africa after watching an online tutorial. New materials that go into 3D printers are cheaper than they used to be and are being used in prosthetics to provide a more affordable option for patients.

Although prosthetics have been around for ages in some form or another, they arent always used. One variable to consider is the social acceptance of having a prosthetic. Theres still a lot of stigma around disabilities and many people may reject prosthetics even if they are available. In 2012, an athlete with both feet amputated competed in the mens 400 meter race at the Olympics in London. There was some controversy over whether the runner with a prosthetic foot should be allowed to run in races with people who dont have prosthetics or if they should only be allowed in competitions specifically for people who have them. Prosthetics also need to be comfortable and usable in order to be successfully adopted. In one study, about 4.5 percent of people rejected prosthetics and 13.4 percent stopped using their prosthetics. As the new prosthetics that are more natural and intuitive to use come to market, hopefully more people will benefit, and the social barriers to acceptance will disappear.

CRISPR

The genome modification technique called Clustered Regularly Interspaced Short Palindromic Repeats, aka CRISPR, was a culmination of a few decades of work by scientists, and major studies explaining the method were published in 2013. The version of it called CRISPR-associated protein 9 or CRIPSR-Cas9 is what most researchers are specifically using in most cases. It involves a regular gene editing mechanism that happens in bacteria. The bacteria can take sections of DNA from attacking viruses and essentially use that to remember the viruses if they return. When the virus is back, the bacteria can target the matching sections of DNA in the virus, cut it and disable the virus.

Though 2013 was only six years ago, as far as science goes, CRISPR has been moving at lightning speed towards practical applications. Using CRISPR to edit a gene sequence, researchers can now add, delete or modify DNA segments more quickly and accurately than ever before. Since the technique was developed, researchers have used CRISPR to target diseases caused by a single gene like cystic fibrosis or sickle cell disease.

Probably the most infamous use of CRISPR are the CRISPR babies. In late 2018, a Chinese researcher, He Jiankui, claimed to have used CRISPR to modify the genomes of two babies to include a mutated version of a gene that protects against HIV. This case was and is highly controversial for the ethical concerns with genetically modifying a human genome at the embryo level, or germline, meaning it can be passed down to future generations and has not been done before in humans. Recently, MIT Technology Review obtained excerpts from Hes research, and experts say that the report and data may be untrustworthy. This means it is still unclear if He and collaborators actually successfully modified the babies genomes. The scientific community overall condemns this way of using CRISPR to edit a human germline genome and has called for an international moratorium on it until a framework can be agreed on.The researcher has been sentenced to three years in prison in Shenzhen, China.

As fraught with controversy as the CRISPR babies may be, CRISPR technology still holds a lot of promise and can be used responsibly, supporters say. For example, researchers are using it to target cancer cells by taking a patients immune cells, modifying them using CRISPR and then infusing the patient with the modified cells. For blood diseases, a patient with sickle cell disease is reported to be responding well to a CRISPR treatment that has allowed her body to produce a crucial protein.

Another area that has boomed this decade partly because of CRISPR technology is stem cell therapy, which well get into in the next section.

Stem cell therapy

Technically, the only Federal Drug Administration (FDA)-approved stem cell therapies are blood-forming stem cells derived from umbilical cord blood. Blood-forming stem cells are used to treat patients with cancer after chemotherapy has depleted blood cells, as well as patients with blood disorders like leukemia whose bone marrow tissues are damaged. These types of treatments have been around for about 30 years, but in the 2010s weve seen potential for more uses of stem cells in health care.

The main idea behind stem cell therapy is that because the cells are pluripotent meaning they can become many other types of cells they can be introduced into parts of the body that are damaged and need new cells. On top of that, researchers can now extract some types of stem cells from a persons body, so no need for umbilical cords. This opens up the possibilities for highly personalized treatment where one person can be treated with stem cells from their own body.

Researchers are exploring how stem cells can be used to treat liver disease, cerebral palsy, stroke, brain injury and others. There are many ongoing research-backed clinical trials for stem cell therapy. A quick search for stem cell therapy on the governments clinical trial database turns up 5,638 results. And because of the work necessary to even get to the clinical trial stage, theres likely an order of magnitude more stem cell therapy studies in the pre-clinical trial stages.

Stem cell therapy is also being offered in for-profit clinics around the U.S. In these cases, the clinics are typically taking fat tissue from a patient, isolating the stem cells and then administering the stem cells back to the patient. In some cases, the treatments may lead to health complications, like blindness in a few extreme cases, and the FDA warns that such treatments are unapproved and potentially harmful. The FDA is ramping up regulation of stem cell clinics and earlier this year took a specific clinic in Florida to court.

Although there are many stem cell clinics offering unproven stem cell therapies, its not all hype. Granted that its difficult to pass the clinical trial stage to get FDA approval, stem cell research may lead to new treatments for several health conditions that could completely change the health care landscape.

You can follow Chia-Yi Hou on Twitter.

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The 3 most important health innovations of the past decade - The Hill

Bone Marrow Stem Cells Comments Off on The 3 most important health innovations of the past decade – The Hill | December 31st, 2019

Doctor close-up of a doctor showing a picture of a kidney on a tablet in a hospital

With a float in this years Rose Bowl parade celebrating organ donation, there are a lot of questions many have about the process and why they should donate their organs.

Legacy of Hope, the Alabama organ donation alliance, said over 1,400 Alabama residents are waiting for a life-saving transplant, with 471 lives saved in 2018.

2.9 million residents across the state are on the registry.

Can I become an organ donor?

The federal government organ donation website, Organdonor.gov, says anyone 18 and older can join the national and state organ donor registries and donate as long as they and their organs are in healthy condition.

The Tennessee donor registry also allows anyone between 13 and 17 to join as long as they have a state ID, drivers license, or leaners permit. However, their parents will have the final say on organ and tissue donation if that decision needs to be made.

Even if you have health issues, you could still donate even one organ, which could save or improve a life.

What can be donated?

How do I register to donate?

There are two registries: The National Donor Registry and the state registry.

In Alabama and Tennessee, if you checked yes to organ donation when applying for or renewing your license, youre already on the state list.

If you didnt check yes, you can make your decision when applying for or renewing your drivers license or state ID at your local DMV or visit your states registry online.

In Alabama, Legacy of Hope manages the state registry, and you can sign up here.

In Tennessee, Donate Life Tennessee manages the state registry donation registry, and you can sign up here.

Youll need to check yes every time you renew to stay on the list.

You can join the national registry hereor in the iPhone Health app.

Who will get my organs if I decide to donate?

Its possible anybody could get your organs if you donate. People of different races match frequently, according to organdonor.gov.

The matching process includes many factors such as location, how long a recipient has been on the list, medical need, and determining blood and tissue type.

The Organ Procurement and Transplantation Network handles the matching process and it varies based on the organ being transplanted.

Does my decision to donate affect the care I get in the hospital?

No. The medical teams saving your life will do everything in their power before donation becomes a possibility. A separate team handles organ retrieval should it be necessary.

The donation process only begins once brain death is confirmed. In those cases, a potential donor must have no brain activity and be unable to breathe without a machine.

Legacy of Hope says in Alabama, two doctors have to mutually agree that a patient is brain dead before the process starts.

Where can I find more information?

If youre trying to decide or just want more information, there are multiple resources.

Visit link:
Curious about organ donation? Heres what you need to know - WHNT News 19

Bone Marrow Stem Cells Comments Off on Curious about organ donation? Heres what you need to know – WHNT News 19 | December 31st, 2019

Soft tissue injuries in muscles, tendons and ligaments, andosteoarthritis, can make moving around painful and limit your physical activity. ButDr. James Presley,a Mayo Clinic physical medicine specialist, says two specialized treatments are growing more common and can help you heal faster.

Platelet-rich plasmais a specialized treatment that Dr. Presley says can bring relief for many patients dealing with soft tissue injuries.

Platelet-rich plasma is a way of trying to harness the bodys immune system or the bodys own ability to heal tissues, Dr. Presley says. [We] concentrate it and then spot-shoot it into the area of injury.

Dr. Presley says the process involves taking blood from your arm, processing it to concentrate the platelets, then injecting it directly into the affected area.

These treatments seem to be helpful in helping the healing process move along when it comes to tendon and ligament injuries, and potentially to help decrease pain and improve function in a joint that has some arthritis, Dr. Presley says.

The second treatment is calledbone marrow aspirate concentrateand involves extracting cells, including stem cells, from bone marrow in the pelvis; processing them into a solution; and injecting them into a painful joint.

The studies that have been done with this have shown patients have decreased pain and thereby improved function of a joint with mild to moderate osteoarthritis, Dr. Presley says.

But he says the best thing you can do is protect your muscles, tendons, and ligaments from injury by finding a happy medium between staying active and avoiding overuse.

Go here to see the original:
Advances In Treatment Of Soft Tissue Injuries (Video) - South Florida Reporter

Bone Marrow Stem Cells Comments Off on Advances In Treatment Of Soft Tissue Injuries (Video) – South Florida Reporter | December 31st, 2019

For decades, the DNA of living organisms such as corn and salmon has been modified, but Crispr, invented in 2012, made gene editing more widely accessible. Image: YinYang/IStock.com via AFP Relaxnews

In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering by editing her genome.

Victoria Grays recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research gene therapy.

I have hoped for a cure since I was about 11, the 34-year-old told AFP in an email.

Since I received the new cells, I have been able to enjoy more time with my family without worrying about pain or an out-of-the-blue emergency.

Over several weeks, Grays blood was drawn so doctors could get to the cause of her illness stem cells from her bone marrow that were making deformed red blood cells.

The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 pronounced Crisper a new tool informally known as molecular scissors.

The genetically edited cells were transfused back into Grays veins and bone marrow. A month later, she was producing normal blood cells.

Medics warn that caution is necessary but, theoretically, she has been cured.

This is one patient. This is early results. We need to see how it works out in other patients, said her doctor, Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville.

But these results are really exciting.

In Germany, a 19-year-old woman was treated with a similar method for a different blood disease, beta thalassemia. She had previously needed 16 blood transfusions per year.

Nine months later, she is completely free of that burden.

For decades, the DNA of living organisms such as corn and salmon has been modified.

But Crispr, invented in 2012, made gene editing more widely accessible. It is much simpler than preceding technology, cheaper and easy to use in small labs.

The technique has given new impetus to the perennial debate over the wisdom of humanity manipulating life itself.

Its all developing very quickly, said French geneticist Emmanuelle Charpentier, one of Crisprs inventors and the cofounder of Crispr Therapeutics, the biotech company conducting the clinical trials involving Gray and the German patient.

Cures

Crispr is the latest breakthrough in a year of great strides in gene therapy, a medical adventure started three decades ago, when the first TV telethons were raising money for children with muscular dystrophy.

Scientists practicing the technique insert a normal gene into cells containing a defective gene.

It does the work the original could not such as making normal red blood cells, in Victorias case, or making tumor-killing super white blood cells for a cancer patient.

Crispr goes even further: instead of adding a gene, the tool edits the genome itself.

After decades of research and clinical trials on a genetic fix to genetic disorders, 2019 saw a historic milestone: approval to bring to market the first gene therapies for a neuromuscular disease in the United States and a blood disease in the European Union.

They join several other gene therapies bringing the total to eight approved in recent years to treat certain cancers and an inherited blindness.

Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution.

Twenty-five, 30 years, thats the time it had to take, he told AFP from Paris.

It took a generation for gene therapy to become a reality. Now, its only going to go faster.

Just outside Washington, at the National Institutes of Health (NIH), researchers are also celebrating a breakthrough period.

We have hit an inflection point, said Carrie Wolinetz, NIHs associate director for science policy.

These therapies are exorbitantly expensive, however, costing up to $2 million meaning patients face grueling negotiations with their insurance companies.

They also involve a complex regimen of procedures that are only available in wealthy countries.

Gray spent months in hospital getting blood drawn, undergoing chemotherapy, having edited stem cells reintroduced via transfusion and fighting a general infection.

You cannot do this in a community hospital close to home, said her doctor.

However, the number of approved gene therapies will increase to about 40 by 2022, according to MIT researchers.

They will mostly target cancers and diseases that affect muscles, the eyes and the nervous system.

Bioterrorism

Another problem with Crispr is that its relative simplicity has triggered the imaginations of rogue practitioners who dont necessarily share the medical ethics of Western medicine.

Last year in China, scientist He Jiankui triggered an international scandal and his excommunication from the scientific community when he used Crispr to create what he called the first gene-edited humans.

The biophysicist said he had altered the DNA of human embryos that became twin girls Lulu and Nana.

His goal was to create a mutation that would prevent the girls from contracting HIV, even though there was no specific reason to put them through the process.

That technology is not safe, said Kiran Musunuru, a genetics professor at the University of Pennsylvania, explaining that the Crispr scissors often cut next to the targeted gene, causing unexpected mutations.

Its very easy to do if you dont care about the consequences, Musunuru added.

Despite the ethical pitfalls, restraint seems mainly to have prevailed so far.

The community is keeping a close eye on Russia, where biologist Denis Rebrikov has said he wants to use Crispr to help deaf parents have children without the disability.

There is also the temptation to genetically edit entire animal species malaria-causing mosquitoes in Burkina Faso or mice hosting ticks that carry Lyme disease in the US.

The researchers in charge of those projects are advancing carefully, however, fully aware of the unpredictability of chain reactions on the ecosystem.

Charpentier doesnt believe in the more dystopian scenarios predicted for gene therapy, including American biohackers injecting themselves with Crispr technology bought online.

Not everyone is a biologist or scientist, she said.

And the possibility of military hijacking to create soldier-killing viruses or bacteria that would ravage enemies crops?

Charpentier thinks that technology generally tends to be used for the better.

Im a bacteriologist weve been talking about bioterrorism for years, she said. Nothing has ever happened.IB/JB

RELATED STORIES:

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2019: The year gene therapy came of age - INQUIRER.net

Bone Marrow Stem Cells Comments Off on 2019: The year gene therapy came of age – INQUIRER.net | December 29th, 2019

A mum whose ex bludgeoned her teenage son to death with a hammer before killing himself is making a heartfelt plea to strangers to try and save her own life.

Tania Morris fears this Christmas may be her last after being diagnosed with Hodgkin's Lymphoma shortly after the tragedy in Burslem, Stoke-on-Trent.

The 49-year-old, who was the only survivor of the brutal attack, was later told that her only chance of survival is a stem cell transplant, StokeonTrentLive revealed.

In the most cruel twists of fate, her only hope would have been heronly child, 19-year-old Nathan Bates, who was murdered just weeks before she was diagnosed with the deadly blood disease.

Tania and her loved-ones are now desperately urging people to join the Antony Nolan register in the hope they can get their very own Christmas miracle.

Tests on other family members including Tania's mum Viv, 69, and dad Robert, aged 70, showed they are not suitable as they are too old and one has a pacemaker.

And her brother Darren Morris, 48, was not a match either.

Meanwhile, her younger brother Adam Morris, who could have fitted the bill, died of a heart attack at the age of 41 four years ago.

Brave Tania, of Burlsem. told StokeonTrentLive: "It's heartbreaking. The doctors just keep saying we need a fit-and-healthy 19-year-old and that breaks my heart as that's how old Nathan was when he was murdered.

"My younger brother could have been a match but he died of a heart attack.

"My other brother Darren was devastated when he was tested and wasn't a match. He wanted so much to help me.

"Dad's only a half match. He's too poorly himself to go ahead but if and when it becomes life or death he could be a last roesort. They're worried it would kill both me and him.

"It would really be desperate measures if it comes to that. It would be my last option because it could kill me.

"My mum's not even been tested because she's had a heart bypass.

"We are just hoping that someone comes forward. It's my only chance of beating this."

Tania's ex, Robert Goodwin, attacked Nathan as he slept at his home, knowing Nathans mum would be out.

An inquest heard Goodwin then left the property and hanged himself in nearby woodland at a spot where Tania would routinely walk her dogs.

It is believed he did so with the intentions that his ex would discover not just her sons body, but also his.

At the time of Nathan's murder, Goodwin was on bail after being charged with assaulting Tania and had retained his liberty despite having breached a condition not to contact her.

And it was while she was coping with the stress of organising Nathan's funeral the following December that she first noticed her own health decline.

The official blood cancer diagnosis then came in January this year.

She has since endured endless rounds of chemotherapy - but has just been told the latest treatment is not working.

Tania said: "He's just a coward for what he did to Nathan. He wouldn't accept any responsibility for what he did. He wanted me to drop the charges but I refused because I was scared he would do it to someone else.

"He just couldn't cope with the thought of going to prison so he killed Nathan, killed himself and if I don't find a donor, he may yet kill me. All the stress he's put our family through also led to my mum's heart attack.

"He had no reason to do what he did, he just wanted to upset me in the worst possible way.

"He said he loved me to pieces but then he did this.

"Since the day he attacked me, it's just been one thing after another."

Tania, who is currently too sick to work at Churchill China, added: "I haven't properly grieved for Nathan because of the cancer.

"I'm up at hospital every week and fighting this disease just leaves me so tired. I can't sleep anymore. If I'm not thinking about the cancer, my thoughts turn to Nathan.

"I'm not coping very well. It's been a rollercoaster couple of years.

"I've then got all my money worries. I just don't know what more I can cope with.

"I was planning on having a 50th birthday in the new year but we've put the plans on hold because I don't know when I'll be in hospital. I wanted to something to say thank-you to all my friends and family but everything is still up in the air.

"I can't plan anything. My life has been on hold since the day that man attacked me."

Tania has monthly appointments at Christies in Manchester to check whether there is a match.

Urging people to join the register, she added: "It's just a simple test you do in your own home send off.

"I'm not doing this just for me but for everyone else who needs a donor. You never know, you could save someone's life. It's my last chance to see another Christmas."

Who can join?

Anyone aged 16 to 30 and in good health can join.

Younger donors provide better outcomes for patients, so our recruitment age range means we focus on recruiting the best possible donors.

How to join?

Fill in the online application form and Antony Nolan will send a swab pack in the post to complete and send back which will then be added to the stem cell register.

How long will I be on the register?

Those who join will remain on the register until the age of 61. If you ever come up as a match for a patient, Antony Nolan be in touch straightaway.

What happens if I'm a match?

Antony Nolan will organise everything including travel and accommodation.

How do you make a donation?

There are two ways you might be asked to donate:90% of people donate via their bloodstreamand10% have their stem cells collected via their bone marrowwhile under general anaesthetic.

See the article here:
Cancer-hit mum's plea for donor after her ex murdered son who could have been match - Mirror Online

Bone Marrow Stem Cells Comments Off on Cancer-hit mum’s plea for donor after her ex murdered son who could have been match – Mirror Online | December 29th, 2019

Edward Aschoff, a college football reporter for ESPN, died Tuesday on his 34th birthday, according to ESPN

When ESPN reporter Edward Aschoff died, he had been diagnosed with multifocal pneumonia and a rare disease known as HLH, his fiance tweeted.

Aschoff was first admitted to the hospital and diagnosed with pneumonia in many parts of his lungs but was brought back to the emergency room when antibiotic treatment failed and he got worse, Katy Berteau said.

"After many tests - bone marrow and lung biopsies - treatment was started for a presumed diagnosis of HLH," she tweeted. "Within 3 days of being moved into the ICU, he passed."

HLH, hemophagocytic lymphohistiocytosis, is a rare disease that affects the immune system.

She did not provide any further details about the manner of Aschoff's death, which occurred on his 34th birthday.

Other people, including Aschoff himself, expressed surprise about the seriousness of the illness in a young man in apparently good health.

"Anyone ever had multifocal (bilateral) pneumonia in their early 30s as some who never gets sick and has a very good immune system? Asking for two friends ... my lungs," he tweeted on December 5.

More questions have come up about his second diagnosis, HLH. It is unclear if Aschoff had HLH or pneumonia first, if one came from the other, and exactly how he died so quickly.

Here is what we know about the diseases Aschoff's had:

Pneumonia is when air sacs in the lungs fill with fluid or pus. It can be caused by a virus, bacteria or a fungus, causing a fever and respiratory problems.

It can occur in one or both lungs, and multifocal means the pneumonia occurs in multiple places.

Thousands of people die around the world each year of pneumonia, but most healthy people can fight it off, especially with antibiotics and antiviral medications. The people most at risk are the young, elderly, frail or immune-compromised.

HLH is a rare disease that affects the immune system, making certain white blood cells attack other blood cells and enlarging the spleen and liver, according to Johns Hopkins Medicine.

It can be inherited or acquired, Johns Hopkins said. About a quarter of cases are passed down through families, and the rest come from infections, a weakened immune system and cancer.

Symptoms can include coughing, difficulty breathing, fever, headaches, rashes, swollen lymph nodes, jaundice and digestive problems, according to Johns Hopkins.

There is treatment for HLH, and acquired forms may clear when properly treated, Johns Hopkins said. If familial HLH goes untreated, it is usually fatal.

Treatments include chemotherapy, immunotherapy, steroids, antibiotic drugs and antiviral drugs. Stem cell transplants can cure HLH in most cases if drug treatments don't work, Johns Hopkins said.

There is no way to prevent HLH, the medical center said.

See more here:
ESPN reporter Edward Aschoff was diagnosed with pneumonia and HLH before he died. What is HLH? - Q13 News Seattle

Bone Marrow Stem Cells Comments Off on ESPN reporter Edward Aschoff was diagnosed with pneumonia and HLH before he died. What is HLH? – Q13 News Seattle | December 28th, 2019

Victoria Gray's recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research -- gene therapy.

Picture: Supplied.

WASHINGTON, United States - In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering -- by editing her genome.

Victoria Gray's recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research -- gene therapy.

"I have hoped for a cure since I was about 11," the 34-year-old told AFP in an email.

"Since I received the new cells, I have been able to enjoy more time with my family without worrying about pain or an out-of-the-blue emergency."

Over several weeks, Gray's blood was drawn so doctors could get to the cause of her illness -- stem cells from her bone marrow that were making deformed red blood cells.

The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 -- pronounced "Crisper" -- a new tool informally known as molecular "scissors."

The genetically edited cells were transfused back into Gray's veins and bone marrow. A month later, she was producing normal blood cells.

Medics warn that caution is necessary but, theoretically, she has been cured.

"This is one patient. This is early results. We need to see how it works out in other patients," said her doctor, Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville.

"But these results are really exciting."

In Germany, a 19-year-old woman was treated with a similar method for a different blood disease, beta-thalassemia. She had previously needed 16 blood transfusions per year.

Nine months later, she is completely free of that burden.

For decades, the DNA of living organisms such as corn and salmon has been modified.

But Crispr, invented in 2012, made gene editing more widely accessible. It is much simpler than preceding technology, cheaper and easy to use in small labs.

The technique has given new impetus to the perennial debate over the wisdom of humanity manipulating life itself.

"It's all developing very quickly," said French geneticist Emmanuelle Charpentier, one of Crispr's inventors and the cofounder of Crispr Therapeutics, the biotech company conducting the clinical trials involving Gray and the German patient.

CURES

Crispr is the latest breakthrough in a year of great strides in gene therapy, a medical adventure started three decades ago when the first TV telethons were raising money for children with muscular dystrophy.

Scientists practising the technique insert a normal gene into cells containing a defective gene.

It does the work the original could not -- such as making normal red blood cells, in Victoria's case, or making tumour-killing super white blood cells for a cancer patient.

Crispr goes even further: instead of adding a gene, the tool edits the genome itself.

After decades of research and clinical trials on a genetic fix to genetic disorders, 2019 saw a historic milestone: approval to bring to market the first gene therapies for a neuromuscular disease in the US and a blood disease in the European Union.

They join several other gene therapies -- bringing the total to eight -- approved in recent years to treat certain cancers and inherited blindness.

Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution.

"Twenty-five, 30 years, that's the time it had to take," he told AFP from Paris.

"It took a generation for gene therapy to become a reality. Now, it's only going to go faster."

Just outside Washington, at the National Institutes of Health (NIH), researchers are also celebrating a "breakthrough period."

"We have hit an inflection point," said Carrie Wolinetz, NIH's associate director for science policy.

These therapies are exorbitantly expensive, however, costing up to $2 million -- meaning patients face gruelling negotiations with their insurance companies.

They also involve a complex regimen of procedures that are only available in wealthy countries.

Gray spent months in the hospital getting blood drawn, undergoing chemotherapy, having edited stem cells reintroduced via transfusion -- and fighting a general infection.

"You cannot do this in a community hospital close to home," said her doctor.

However, the number of approved gene therapies will increase to about 40 by 2022, according to MIT researchers.

They will mostly target cancers and diseases that affect muscles, the eyes and the nervous system.

**BIOTERRORISM **

Another problem with Crispr is that its relative simplicity has triggered the imaginations of rogue practitioners who don't necessarily share the medical ethics of Western medicine.

Last year in China, scientist He Jiankui triggered an international scandal -- and his ex-communication from the scientific community -- when he used Crispr to create what he called the first gene-edited humans.

The biophysicist said he had altered the DNA of human embryos that became twin girls Lulu and Nana.

His goal was to create a mutation that would prevent the girls from contracting HIV, even though there was no specific reason to put them through the process.

"That technology is not safe," said Kiran Musunuru, a genetics professor at the University of Pennsylvania, explaining that the Crispr "scissors" often cut next to the targeted gene, causing unexpected mutations.

"It's very easy to do if you don't care about the consequences," Musunuru added.

Despite the ethical pitfalls, restraint seems mainly to have prevailed so far.

The community is keeping a close eye on Russia, where biologist Denis Rebrikov has said he wants to use Crispr to help deaf parents have children without the disability.

There is also the temptation to genetically edit entire animal species -- malaria-causing mosquitoes in Burkina Faso or mice hosting ticks that carry Lyme disease in the US.

The researchers in charge of those projects are advancing carefully, however, fully aware of the unpredictability of chain reactions on the ecosystem.

Charpentier doesn't believe in the more dystopian scenarios predicted for gene therapy, including American "biohackers" injecting themselves with Crispr technology bought online.

"Not everyone is a biologist or scientist," she said.

And the possibility of military hijacking to create soldier-killing viruses or bacteria that would ravage enemies' crops?

Charpentier thinks that technology generally tends to be used for the better.

"I'm a bacteriologist -- we've been talking about bioterrorism for years," she said. "Nothing has ever happened."

Visit link:
2019: The year gene therapy came of age - Eyewitness News

Bone Marrow Stem Cells Comments Off on 2019: The year gene therapy came of age – Eyewitness News | December 28th, 2019

WASHINGTON: In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering - by editing her genome.Victoria Gray's recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research - gene therapy.'; var randomNumber = Math.random(); var isIndia = (window.geoinfo && window.geoinfo.CountryCode === 'IN') && (window.location.href.indexOf('outsideindia') === -1 ); console.log(isIndia && randomNumber "I have hoped for a cure since I was about 11," the 34-year-old told AFP in an email.

"Since I received the new cells, I have been able to enjoy more time with my family without worrying about pain or an out-of-the-blue emergency."

Over several weeks, Gray's blood was drawn so doctors could get to the cause of her illness - stem cells from her bone marrow that were making deformed red blood cells.

The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 - pronounced "Crisper" -- a new tool informally known as molecular "scissors."

The genetically edited cells were transfused back into Gray's veins and bone marrow. A month later, she was producing normal blood cells.

Medics warn that caution is necessary but, theoretically, she has been cured.

"This is one patient. This is early results. We need to see how it works out in other patients," said her doctor, Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville.

"But these results are really exciting."

In Germany, a 19-year-old woman was treated with a similar method for a different blood disease, beta thalassemia. She had previously needed 16 blood transfusions per year.

Nine months later, she is completely free of that burden.

For decades, the DNA of living organisms such as corn and salmon has been modified.

But Crispr, invented in 2012, made gene editing more widely accessible. It is much simpler than preceding technology, cheaper and easy to use in small labs.

The technique has given new impetus to the perennial debate over the wisdom of humanity manipulating life itself.

"It's all developing very quickly," said French geneticist Emmanuelle Charpentier, one of Crispr's inventors and the cofounder of Crispr Therapeutics, the biotech company conducting the clinical trials involving Gray and the German patient.

Crispr is the latest breakthrough in a year of great strides in gene therapy, a medical adventure started three decades ago, when the first TV telethons were raising money for children with muscular dystrophy.

Scientists practising the technique insert a normal gene into cells containing a defective gene.

It does the work the original could not -- such as making normal red blood cells, in Victoria's case, or making tumor-killing super white blood cells for a cancer patient.

Crispr goes even further: instead of adding a gene, the tool edits the genome itself.

After decades of research and clinical trials on a genetic fix to genetic disorders, 2019 saw a historic milestone: approval to bring to market the first gene therapies for a neuromuscular disease in the US and a blood disease in the European Union.

They join several other gene therapies - bringing the total to eight - approved in recent years to treat certain cancers and an inherited blindness.

Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution.

"Twenty-five, 30 years, that's the time it had to take," he told AFP from Paris.

"It took a generation for gene therapy to become a reality. Now, it's only going to go faster."

Just outside Washington, at the National Institutes of Health (NIH), researchers are also celebrating a "breakthrough period."

"We have hit an inflection point," said Carrie Wolinetz, NIH's associate director for science policy.

These therapies are exorbitantly expensive, however, costing up to $2 million - meaning patients face grueling negotiations with their insurance companies.

They also involve a complex regimen of procedures that are only available in wealthy countries.

Gray spent months in hospital getting blood drawn, undergoing chemotherapy, having edited stem cells reintroduced via transfusion - and fighting a general infection.

"You cannot do this in a community hospital close to home," said her doctor.

However, the number of approved gene therapies will increase to about 40 by 2022, according to MIT researchers.

They will mostly target cancers and diseases that affect muscles, the eyes and the nervous system.

Another problem with Crispr is that its relative simplicity has triggered the imaginations of rogue practitioners who don't necessarily share the medical ethics of Western medicine.

Last year in China, scientist He Jiankui triggered an international scandal - and his excommunication from the scientific community - when he used Crispr to create what he called the first gene-edited humans.

The biophysicist said he had altered the DNA of human embryos that became twin girls Lulu and Nana.

His goal was to create a mutation that would prevent the girls from contracting HIV, even though there was no specific reason to put them through the process.

"That technology is not safe," said Kiran Musunuru, a genetics professor at the University of Pennsylvania, explaining that the Crispr "scissors" often cut next to the targeted gene, causing unexpected mutations.

"It's very easy to do if you don't care about the consequences," Musunuru added.

Despite the ethical pitfalls, restraint seems mainly to have prevailed so far.

The community is keeping a close eye on Russia, where biologist Denis Rebrikov has said he wants to use Crispr to help deaf parents have children without the disability.

There is also the temptation to genetically edit entire animal species - malaria-causing mosquitoes in Burkina Faso or mice hosting ticks that carry Lyme disease in the US.

The researchers in charge of those projects are advancing carefully, however, fully aware of the unpredictability of chain reactions on the ecosystem.

Charpentier doesn't believe in the more dystopian scenarios predicted for gene therapy, including American "biohackers" injecting themselves with Crispr technology bought online.

View original post here:
2019: the year gene therapy came of age - Times of India

Bone Marrow Stem Cells Comments Off on 2019: the year gene therapy came of age – Times of India | December 27th, 2019

Freeze thaw chambers are also called refrigerated humidity chambers. Freeze thaw chamber is used for applications which require temperature cycling down below freezing. Principle of freeze thaw cycle is used in cryopreservation technique. Cryopreservation is the process of preserving living cells and tissues at cryogenic temperature, which lead to suspended metabolic activity of cells in liquid nitrogen. Principle of freeze thaw cycle is nowadays used as treatment method for cancer, as freezing temperature is used in cryosurgery for local tissue destruction.

Report Overview @

http://www.transparencymarketresearch.com/freeze-thaw-chambers-market.html

Freezing and thawing cause cell death due to ice crystal formation, osmotic shock, and membrane damage. Advanced freeze thaw chambers are available in the market with new features, such as, epoxy coating to avoid corrosion, better condenser and evaporator, capillary tube system, CFC-free polyurethane foam for insulation, broad temperature range from -20 C to 60 C, and long service life designs.

Rise in understanding about the physical and chemical properties of freeze and thaw cycle, advancement in the field of mechanics, and flexibility to stimulate a broad range of conditions are some of the factors driving the growth of the freeze thaw chambers market. However, expensive maintenance and stringent regulatory standards are anticipated to hamper the growth of the market during the forecast period. Customized freeze thaw chambers are the ones which can be modified based on the requirement of the end-user. Introduction of customized freeze thaw instruments is likely to boost the growth of the freeze thaw chambers market during the forecast period.

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On the basis of application area, the global freeze thaw chambers market can be segmented into biopharmaceutical development, molecular biology and biochemistry, medical application, and others. Medical application can be further divided into cryopreserved products and others. Cryopreserved products can be further classified into blood transfusion, bone marrow transplantation, artificial insemination, in vitro fertilization (IVF), stem cell and organ, and others.

The biopharmaceutical development segment is propelling the growth of the freeze thaw chambers market as freezing is one of the processing steps in drug development that ensures the stability and quality of drugs, while freeze thaw chambers provide stability to drug substances. Freeze thaw chambers are also used for ASTM material testing. The cryopreserved products segment is expected to drive the growth of the freeze thaw chambers market during the forecast period due to increase in awareness about the importance and function of stem cell and tissue engineering. Advancement in organ transplantation technology is also another important factor contributing to the growth of stem cell and organ cryopreservation. Freeze thaw chambers are extensively used to transfer cryopreserved products to the end-user for applications, such as, blood transfusion, bone marrow transplantation, artificial insemination, and in vitro fertilization (IVF).

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Based on end-user, the global freeze thaw chambers market can be segmented into biopharmaceutical companies, pathology and research laboratories, hospitals, and stem cell and blood banks. The pathology and research laboratories segment is boosting the growth of the freeze thaw chambers market as these chambers are required for research applications which require very low freezing temperature. Biopharmaceutical companies is another growing segment of the freeze thaw chambers market. Freeze thaw chambers maximize productivity and reduce production cost by providing flexibility to drug substances.

In terms of region, the global freeze thaw chambers market can be categorized into North America, Europe, Asia Pacific, Lain America, and Middle East & Africa. North America is the leading market for freeze thaw chambers in the world due to extensive funding support from the government for research activities in the region. Europe is another leading market for freeze thaw chambers market owing to extensive research in the field of biochemistry and molecular biology in the region. The freeze thaw chambers market in Asia Pacific is expected to grow significantly during the forecast period due to rise in awareness in the region regarding medical applications involving cryopreservation process, such as, umbilical cord, stem cell, and blood sample.

Key players operating in the global freeze thaw chambers market are Darwin Chambers, Newtronic Lifecare Equipments Pvt. Ltd., Caron Products and Services, Inc, BIONICS SCIENTIFIC TECHNOLOGIES (P) LTD., Santorius, Feutron Klimasimulation GmBH., LR Environmental Equipment Co.Inc., and Dycometal.

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Freeze Thaw Chambers Market Poised to Expand at a Robust Pace by 2026 - Market Research Sheets

Bone Marrow Stem Cells Comments Off on Freeze Thaw Chambers Market Poised to Expand at a Robust Pace by 2026 – Market Research Sheets | December 27th, 2019

Global Hematopoietic Stem Cell Transplantation (HSCT) Market 2019 by key players, regions, type, and application, forecast to 2025. The Report contains a forecast of 2019 and ending 2025 with a host of metrics like supply-demand ratio, Hematopoietic Stem Cell Transplantation (HSCT) market frequency, dominant players of Hematopoietic Stem Cell Transplantation (HSCT) market, driving factors, restraints, and challenges. The report also contains market revenue, sales, Hematopoietic Stem Cell Transplantation (HSCT) production and manufacturing cost that could help you get a better view of the market. The report focuses on the key global Hematopoietic Stem Cell Transplantation (HSCT) manufacturers, to define, describe and analyze the sales volume, value, market competition landscape, market share, SWOT analysis and development plans in future years.

The report provides information on trends and developments and focuses on market capacities, technologies, and the changing structure of the Hematopoietic Stem Cell Transplantation (HSCT) Market. The new entrants in the Hematopoietic Stem Cell Transplantation (HSCT) Market are finding it hard to compete with the international dealer based on quality and reliability.

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Major Players included in this report are as follows Regen Biopharma IncChina Cord Blood CorpCBR Systems IncEscape Therapeutics IncCryo-Save AGLonza Group LtdPluristem Therapeutics IncViaCord I

Hematopoietic Stem Cell Transplantation (HSCT) Market can be segmented into Product Types as AllogeneicAutologous

Hematopoietic Stem Cell Transplantation (HSCT) Market can be segmented into Applications as Peripheral Blood Stem Cells Transplant (PBSCT)Bone Marrow Transplant (BMT)Cord Blood Transplant (CBT)

Hematopoietic Stem Cell Transplantation (HSCT) Market: Regional analysis includes:Asia-Pacific (Vietnam, China, Malaysia, Japan, Philippines, Korea, Thailand, India, Indonesia, and Australia)Europe (Turkey, Germany, Russia UK, Italy, France, etc.)North America (United States, Mexico, and Canada.)South America (Brazil etc.)The Middle East and Africa (GCC Countries and Egypt.)

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Objective of Studies: 1. To provide detailed analysis of the market structure along with forecast of the various segments and sub-segments of the global Hematopoietic Stem Cell Transplantation (HSCT) market. 2. To provide insights about factors affecting the market growth. To analyse the Hematopoietic Stem Cell Transplantation (HSCT) market based on various factors- price analysis, supply chain analysis, Porte five force analysis etc. 3. To provide historical and forecast revenue of the market segments and sub-segments with respect to four main geographies and their countries- North America, Europe, Asia, Latin America and Rest of the World. 4. To provide country level analysis of the market with respect to the current market size and future prospective. 5. To provide country level analysis of the market for segment by application, product type and sub-segments. 6. To provide strategic profiling of key players in the market, comprehensively analysing their core competencies, and drawing a competitive landscape for the market. 7. To track and analyse competitive developments such as joint ventures, strategic alliances, mergers and acquisitions, new product developments, and research and developments in the global Hematopoietic Stem Cell Transplantation (HSCT) market.

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The huge assortment of tables, graphs, diagrams, and charts obtained in this market research report generates a strong niche for an in-depth analysis of the ongoing trends in the Hematopoietic Stem Cell Transplantation (HSCT) market. Further, the report revises the market share held by the key players and forecast their development in the upcoming years. The report also looks at the latest developments and advancement among the key players in the market such as mergers, partnerships, and achievements.

In short, the Global Hematopoietic Stem Cell Transplantation (HSCT) Market report offers a one-stop solution to all the key players covering various aspects of the industry like growth statistics, development history, industry share, Hematopoietic Stem Cell Transplantation (HSCT) market presence, potential buyers, consumption forecast, data sources, and beneficial conclusion.

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Global Hematopoietic Stem Cell Transplantation (HSCT) Market 2019 Industry Key Players, Trends, Sales, Supply, Demand, Analysis & Forecast to 2025...

Bone Marrow Stem Cells Comments Off on Global Hematopoietic Stem Cell Transplantation (HSCT) Market 2019 Industry Key Players, Trends, Sales, Supply, Demand, Analysis & Forecast to 2025… | December 27th, 2019

Sickle cell disease is a genetic condition in which red blood cells, which should be circular, adopt a crescent shape and are sticky and rigidALAMY

The first patients to receive gene-editing treatments for inherited blood diseases will enter the new year free of agonising symptoms.

The experiments suggest that altering DNA could treat sickle cell disease (SCD) and beta thalassemia, conditions both caused by faulty genes that hamper the bloods ability to carry oxygen.

The companies behind the trials said that a patient in the US with SCD had been well since July. A thalassemia patient in Germany had been free of symptoms for nine months. Previously she had 16 blood transfusions a year.

British patients could be offered similar experimental therapies next year. The treatment for both conditions involved a high-precision gene-editing tool called Crispr-Cas9. It was used to alter the DNA of some of the cells of Victoria

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Sickle cell patient is pain free after geneediting trial altered her DNA - The Times

Bone Marrow Stem Cells Comments Off on Sickle cell patient is pain free after geneediting trial altered her DNA – The Times | December 25th, 2019

Last Christmas a student received what he says was the best Christmas present ever the chance to save a strangers life with a stem cell donation.

This Christmas, the student found out the patient who received his donation was successful, and that the patient was recovering well.

In December last year, Will Briant, 23, from Kennington, received an email that informed him he had come up as a potential match for a blood cancer patient in desperate need of a stem cell transplant.

Will said: Just a week before Christmas, I got the best Christmas present ever. I was told that I was the best match for the patient, and I would be donating early in the new year.

I was so excited. When you sign up you know that its such a tiny chance that youll be found as the best match for someone, so to actually be chosen felt really exciting.

Also, because it was just before Christmas, it felt quite exciting to know that the patient would find out that they had a match just in time for Christmas.

At the beginning of this year Will donated his stem cells at The London Clinic.

Will said: For four days before the donation I had a course of G-CSF injections to increase the number of stem cells I was producing.

This caused mild flu-like symptoms. I just felt a bit tired and achy really.

The whole way through I kept thinking about the recipient and how in this context I was absolutely delighted to have mild flu-like symptoms.

It was quite strange to be doing it for real, after talking to so many potential donors when I volunteered with Marrow at university.

Will initially joined the Anthony Nolan stem cell register in 2014.

His girlfriend, who volunteered with Edinburgh Universitys Blood, Bone Marrow and Transplant Society, which is part of blood cancer charity Anthony Nolans student volunteer network, called Marrow, suggested that he sign up.

Will said: My girlfriend, Libby, told me this amazing statistic that a quarter of all stem cell donors sign up through Marrow at university, so I couldnt not join.

If it wasnt for Marrow and for Libby, I wouldnt have become a donor and given someone hope of a second chance of life just before Christmas.

Following his donation Will then went back to his studies and his job, barely giving a second thought to what hed just done.

Will recently received a letter from the hospital to say that the donation had been successful and the donor recipient was recovering well.

Will said: It was honestly the best letter Ive ever received. It was especially powerful because it really hit home that not only had I given him a second chance of life, but also I had given his wife, his children, his grandchildren and his friends more precious time with him.

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Donor Will gives the gift of life at Christmas - London News Online

Bone Marrow Stem Cells Comments Off on Donor Will gives the gift of life at Christmas – London News Online | December 24th, 2019

Of all blood cancers, leukaemia and lymphoma are among the most curable.

However, many people, including doctors, still believe the disease leads to immediate death.

This is no longer true today as they are not fatal.

With optimal treatment, the majority of patients go into remission and are considered cured.

These two cancers have been more extensively studied than other forms of cancer, due to the ease in obtaining samples from blood, bone marrow or lymph nodes, spurring the advent of novel targeted therapies for a cure, says consultant haematologist Dr Ng Soo Chin.

Most blood cancers start in the bone marrow, where blood is produced.

Bone marrow contains stem cells, which mature and develop into red blood cells, white blood cells or platelets.

In most blood cancers, normal cell development is interrupted by the uncontrolled growth of an abnormal type of a particular blood cell.

These abnormal blood cells, which are cancerous, prevent your blood from performing many of its functions, like fighting off infections or preventing serious bleeding.

Leukaemia or white blood is classified into acute and chronic disease, which is then divided further into subtypes: acute lymphocytic leukaemia, acute myeloid leukaemia, chronic lymphocytic leukaemia (CLL) and chronic myeloid leukaemia (CML).

The presentation between acute and chronic leukaemia differs.

The acute person will tell you he was well a week ago and is now down with symptoms such as lethargy, anaemia and recurrent infection.

Suddenly, he may look pale, so we check his blood count for any abnormalities. A bone marrow exam will further confirm whether it is acute.

With chronic leukaemia, the patient can be unwell for a couple of months.

We are increasingly picking up cases early because of blood test availability.

The survival rate has improved tremendously for acute leukaemia, with more than 50% fully cured because bone marrow transplants are easily available in the country.

For CLL and CML, 95% of patients are alive at the 10-year mark, says Dr Ng.

Generally, chronic leukaemia patients belong to the older age group (50 years and above), but acute leukaemia can occur in all ages.

Leukaemia symptoms are often vague and not specific, so its easy to overlook them as they may resemble symptoms of the flu and other common illnesses.

In fact, chronic leukaemia may initially produce no symptoms and can go unnoticed or undiagnosed for years.

Lymphomas, a type of blood cancer that begins in a subset of white blood cells called lymphocytes, can be classified into Hodgkins and non-Hodgkins.

The main difference between Hodgkins and non-Hodgkins lymphoma is the specific lymphocyte each involves.

Lymphocytes are an integral part of your immune system, which protects you from germs.

Five-year survival rates are high with Hodgkins lymphoma at 86% and non-Hodgkins lymphoma at 70%.

You can beat the disease even if it is detected at a late stage.

Multiple myeloma, which is the third kind of blood cancer, forms in a type of white blood cell called a plasma cell.

Patients often complain of bone pain, and unfortunately, this type of cancer has no cure.

Blood cancers typically involve abnormal white blood cells and can affect paople of all ages, depending on the type of cancer. 123rf.com

Fear of treatment

Chemotherapy is a much dreaded word among cancer patients.

But with advances in medicine, newer chemotherapy-free treatments are now available.

Dr Ng says, Traditionally, cancer is treated via surgery or radiation the layman says we fry and poison them, which is not far from the truth!

Radiation means burning the cancerous area, but a lot of times, the cancer can also be present elsewhere, so there is limitation to this treatment.

With chemotherapy, we use cytotoxic (cell-killing) drugs they go in and knock off both cancer and normal cells.

The short-term effects include vomiting, hair loss, appetite loss and weight loss.

But as doctors, we are looking at a different perspective. We are more worried about white cells dropping (neutropenia) because the patient can pick up an infection that can potentially kill him.

Neutropenia is a condition that results when the body does not have enough neutrophils, a type of white blood cell that is an essential first line of defence against infections.

Thats one risk of chemotherapy, although we can now improve neutropenia by giving a growth factor injection.

But for certain cancers, we need to step up the drugs.

He adds: We are scared of neutropenia, but patients are more concerned about bodily changes.

The older ones get upset over losing hair because they cannot take it when others ask them what has happened to their hair.

Young people are not as concerned with hair loss because it can be trendy.

We understand that chemotherapy is less than pleasant and strong doses can impair fertility in young patients, especially women.

Despite current technology, only one-third of patients are successful in freezing their eggs.

What he is concerned about is that chemotherapy can actually increase the patients risk of getting another cancer, especially blood cancer.

It can happen the day after! says Dr Ng.

Most experts believe chemotherapy damages stem cells, so if youre unlucky, you might get acute myeloid leukaemia after undergoing chemotherapy for breast cancer.

Its just like crossing the road there is always a risk of being knocked down.

All our cells have a biological clock and there is an orderly exchange of old and new cells.

But with blood cancers such as leukaemia, there is a clone of abnormal cells.

Cancer cells have an advantage over normal cells because they can survive longer.

Chemotherapy is still needed to treat most acute blood cancers, although if the mutation is known, targeted therapies can be applied.

For chronic blood cancers, there is no need for chemotherapy. Oral drugs are enough to combat the disease.

Eventually, many patients are able to wean off the drugs.

As we may be aware, immunotherapy is the buzzword in cancer treatment today.

Also called biologic therapy, it is a type of cancer treatment that boosts the bodys natural defences to fight cancer.

It uses substances made by the body or in a laboratory to improve or restore immune system function.

One of the latest treatment modalities is the CAR T-cell therapy, a form of immunotherapy that uses specially altered T cells a part of the immune system to fight cancer.

A sample of a patients T cells are collected from the blood, then modified to produce special structures called chimeric antigen receptors (CARs) on their surface.

When these CAR T-cells are reinfused into the patient, the new receptors enable them to latch onto a specific antigen on the patients tumour cells and kill the cells.

At the moment, this intravenous therapy is available in the United States and hasnt reached our shores yet. It has to be properly regulated first, says Dr Ng.

A volunteer is having his head shaved to donate hair to make wigs for cancer patients in this filepic. Hair loss is one of the side effects of chemotherapy that affect patients the most.

Following natural remedies

The consultant haematologist errs on the side of caution when patients ask about natural cancer remedies, or the dos and donts during treatment.

We always believe there should be a scientific approach to the problem.

If patients are doing okay while undergoing treatment and there is no weight loss, I tell them to go ahead and do what they always do.

However, just be particular about food hygiene, as there is a chance you may get food poisoning.

If youre undergoing chemotherapy, then youll land yourself in hospital, and if your luck is bad, you may even land up in the ICU (intensive care unit).

So make sure the food is cooked and not left overnight to reduce your chances of infection.

Eat a balanced diet, he advises.

When it comes to exercise, he says to work out within your limit.

Instead of pushing the body and running marathons or climbing mountains, go for walks.

Dr Ng says, Life should go on, but be sensible.

Dont go to crowded places because you may pick up an infection, but dont be withdrawn either. All humans need social interaction.

With the billion-dollar dietary supplements industry, companies are constantly trying to lure customers into buying their products.

A lot of supplements are just glorified vitamins in different packaging.

The more expensive they are, the more people will buy them, thinking they are good.

There are people with good intentions, but unfortunately, there are also a lot of scammers out there that is life.

For the amount you spend on supplements, why not keep the money aside and go for a trip once your treatment is over? he suggests.

Often, the late diagnosis is due to preference for alternative treatment.

These alternative treatments are like fashion shows, after some time, they go out of trend.

For me, youre wasting valuable time because cancer is not your friend.

Yes, chemotherapy is tough, but with the latest chemo-free regimen, patients are more willing to come forward.

The earlier it is treated, the higher your chances of recovering, he says.

To share his 30-odd years of knowledge and experience in the field, Dr Ng has written his third book titled Understanding Blood Disorders.

Intended for patients, caregivers and healthcare professionals, proceeds from the sales of the 270-page book will go to the newly set-up Faith Hope Love Hospice Care Malaysia in Petaling Jaya, Selangor.

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Leukaemia and lymphoma have a good survival rate - The Star Online

Bone Marrow Stem Cells Comments Off on Leukaemia and lymphoma have a good survival rate – The Star Online | December 24th, 2019

Mila Roncevic (Photo: Private album)

24 December 2019 There has been some great news just before Christmas from America where little Mila Roncevic has been receiving life-saving treatment.

Mila, from Rijeka in Croatia, has been fighting leukaemia (acute myelogenous megakaryocytic leukaemia AML M7) and earlier this year was given just a 1% chance of beating it. A massive fundraising campaign in Croatia and abroad back in April helped raise $5.6 million to get her to the Childrens Hospital of Philadelphia for urgent treatment.

Mila has been at the hospital since 3 April and on Monday her father has shared some incredible news that after a successful bone marrow transplant, the leukemia has receded.

Mila is fine right now. She underwent a bone marrow transplant from a donor on 1/11/2019 and so far its better than okay. It was not a surgery per se, but rather a bone marrow transplant after achieving and maintaining remission for six months. Transplantation is not an operation but a complex procedure which includes preparation of the so-called conditioning, that is, achieving the conditions for receiving donor stem cells and then recovering and waiting for defence cells to be created on their own, in which case she had to remain in complete isolation because there is no immunity. In order to receive the transplant, the disease must recede, which they achieved within a month of arrival and maintained for almost half a year, her father Marin told 24sata.

Marin says that just three weeks after the transplant, Mila was released from hospital and to the amazement and disbelief of all the doctors, she is now running, lifting, eating and drinking alone and does everything that was considered impossible in the early post-transplant period.

She looks and acts like a perfectly healthy baby, even though we know that there are still many potential dangers ahead, Marin said.

Marin says going to Philadelphia was certainly the right decision, which has been confirmed by the successful outcome of the treatment.

In Croatia, the chances for successful treatment were below 1%. It was also the right decision because at that moment we had no alternative, nor enough time to look for it. It is difficult to actually talk about percentages, because as far as we are told, cases with similar diagnoses that have been successfully cured in Croatia and Europe do not exist, he said, before thanking everyone again who helped get Mila to the United States for treatment.

(Photo: Private album)

Thank you people. If it wasnt for your empathy, kindness and intrinsic desire to help, none of this would be possible. Every kuna, every prayer and every good thought saved Mila and will help countless other children for whom funds will be provided for. This action also showed how important it is for all of us to stick together and how much we can do that way. We are incredibly grateful to have genuinely and openly begged for help and received it. Its not just about the money. It is about the love, support and prayers of all people who have felt our affliction and suffering.

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Little Mila on amazing path to recovery - Croatia Week

Bone Marrow Stem Cells Comments Off on Little Mila on amazing path to recovery – Croatia Week | December 24th, 2019

The Galloping Ghosts are looking beyond the scoreboard to help classmate Juwan Adams battle pediatric cancer.

Khalis Whiting and her Abington girls basketball teammates have a different perspective this holiday season. A perspective that changed when through Kisses for Kyle they were put in touch with two area families who have a child battling pediatric cancer.

One George Hamlin is a student in Upper Dublin High School. The other, Juwan Adams, is a senior at Abington. Both families were on the receiving end of gifts and gift cards at the Abington-Upper Moreland game last week. It is the 10th annual giving back game since coach Dan Marsh initiated the project.

It was an amazing experience, said Whiting, the Ghosts sophomore point guard. I was not familiar with the families, but I have seen (Adams) a few times in school, but I never really knew the story until this came up and I researched him.

He has a powerful story, and hes such a strong young man to be going through this. Every time I see him in the hallways, hes always smiling and so positive. So you know give it to his family.

Adams, who has been battling Hodgkins Lymphoma since April of 2016, is in need of a match for an allogeneic stem cell transplant.

Right now, he is out of options, his mother, Andrea Adams, said. We know for sure that an allo stem cell transplant will cure him, but unfortunately, in order to do that, we need a donor.

We have tested everyone in our family, and no one is a full match, so were relying on the Be the Match registry to locate a donor. Sadly, for minorities theres a very slim chance to actually find a full 10-out-of-10 match because we dont have enough minorities on the registry.

Hes had almost every treatment available for Hodgkins, including an auto stem cell transplant, which is where he gave himself his own cells in February of 2017. He was in remission for about a year, and unfortunately, in May of this year, we found out the cancer is back, and its been spreading consistently since May.

Despite missing more than half of the last three-and-a-half years of school, Juwan maintains a 4.25 GPA and is a member of the National Honor Society.

Abington School District has been really great with accommodating him, giving him tutors, and whenever hes an in-patient, he does hospital school, Andrea said.

Juwan is the drum captain and lieutenant of Abingtons marching band, and since September is Pediatric Cancer Awareness month, for the past four years, the marching band has worn gold ribbons on their uniforms.

Since his diagnosis in eighth grade, Juwan has been active in pediatric cancer awareness events, and for the past four years, he has held toy and book drives that he presents to the children at CHOP on his birthday in July.

Last week, Juwan and his family were on the receiving end.

It was great because Juwan never wants gifts, Andrea said. Having a child with cancer, financially its tough, especially now that we have only have one person working fulltime, and that lifted a tremendous burden, and it was so unexpected.

To have him honored by his school and his peers, he was super excited about it, and he really appreciated it because a lot of kids (battling cancer) have to give up school, or their friends tend to abandon them.

I want to thank the team and the Abington students because it is very easy to turn your back on kids that are going through these things. These kids have really rallied around him. This is one of the reasons he fights so hard to be in school. He sometimes sneaks to school because he feels the love from his school and his peers. That was one thing he definitely wanted to do to finish his senior year with his class. They have really rallied around him, and this kind of thing gives him the extra push he needs to keep fighting.

Statistics say Juwan has a 23 percent chance of finding a perfect match, but the Abington senior has been proactive.

When they told him the odds of finding his match, he said he had to do something about it, Andrea said. He set a goal to register a thousand people on the national registry, half of those being minorities.

We have gone around holding bone marrow drives. Hes now an ambassador for Be the Match, and hes been an ambassador for CHOP for about two years.

Juwan is inviting everyone to join his fight.

"People are starting to wake up and realize kids with cancer more and more are dying each day, and if they have a chance to do something, they should take every step they can," Juwan said earlier this fall. It's been hard sometimes, but I have my friends and family to support me.

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Abington working toward big win - Sports - The Intelligencer

Bone Marrow Stem Cells Comments Off on Abington working toward big win – Sports – The Intelligencer | December 24th, 2019