Category Archives: Stem Cell Treatment

Genetic profiling can guide stem cell transplantation for patients with myelodysplastic syndrome – Medical Xpress

February 9, 2017 Credit: NIH

A single blood test and basic information about a patient's medical status can indicate which patients with myelodysplastic syndrome (MDS) are likely to benefit from a stem cell transplant, and the intensity of pre-transplant chemotherapy and/or radiation therapy that is likely to produce the best results, according to new research by scientists at Dana-Farber Cancer Institute and Brigham and Women's Hospital.

In a study published in the New England Journal of Medicine, the investigators report that genetically profiling a patient's blood cells, while factoring in a patient's age and other factors, can predict the patient's response to a stem cell transplant and help doctors select the most effective combination of pre-transplant therapies. The findings are based on an analysis of blood samples from 1,514 patients with MDS, ranging in age from six months to more than 70 years, performed in collaboration with investigators from the Center for International Blood and Marrow Transplant Research.

MDS is a family of diseases in which the bone marrow produces an insufficient supply of healthy blood cells. Treatments vary depending on the specific type of MDS a patient has; donor stem cell transplants are generally used for patients with a high risk of mortality with standard treatments.

"Although donor stem cell transplantation is the only curative therapy for MDS, many patients die after transplantation, largely due to relapse of the disease or complications relating to the transplant itself," said the study's lead author, R. Coleman Lindsley, MD, PhD, of Dana-Farber. "As physicians, one of our major challenges is to be able to predict which patients are most likely to benefit from a transplant. Improving our ability to identify patients who are most likely to have a relapse or to experience life-threatening complications from a transplant could lead to better pre-transplant therapies and strategies for preventing relapse."

Researchers have long known that the specific genetic mutations within MDS patients' blood cells are closely related to the course the disease takes. The current study sought to discover whether mutations also can be used to predict how patients will fare following a donor stem cell transplant.

Analysis of the data showed that the single most important characteristic of a patient's MDS was whether their blood cells carried a mutation in the gene TP53. These patients tended to survive for a shorter time after a transplant, and also relapse more quickly, than patients whose cells lacked that mutation. This was true whether patients received standard "conditioning" therapy (which includes chemo- and/or radiation therapy) prior to transplant or received reduced-intensity conditioning, which uses lower doses of these therapies. Based on these results, doctors at Dana-Farber are now working on new strategies to overcome the challenges posed by TP53 mutations in MDS.

In patients 40 years old and over whose MDS didn't carry TP53 mutations, those with mutations in RAS pathway genes or the JAK2 gene tended to have a shorter survival than those without RAS or JAK2 mutations. In contrast to TP53 mutations, the adverse effect of RAS mutations on survival and risk of relapse was evident only in reduced-intensity conditioning. This suggests that these patients may benefit from higher intensity conditioning regimens, the researchers indicated.

The study also yielded key insights about the biology of MDS in specific groups of patients. Surprisingly, one in 25 patients with MDS between the ages of 18 and 40 were found to have mutations associated with Shwachman-Diamond syndrome (a rare inherited disorder that often affects the bone marrow, pancreas, and skeletal system), but most of them had not previously been diagnosed with it. In each case, the patients' blood cells had acquired a TP53 mutation, suggesting not only how MDS develops in patients with Schwachman-Diamond syndrome but also what underlies their poor prognosis after transplantation.

The researchers also analyzed patients whose MDS arose as a result of previous cancer therapy (therapy-related MDS). They found that TP53 mutations and mutations in PPM1D, a gene that regulates TP53 function, were far more common in these patients than in those whose disease occurred in the absence of previous cancer treatment.

"In deciding whether a stem cell transplant is appropriate for a patient with MDS, it's always necessary to balance the potential benefit with the risk of complications," Lindsley remarked. "Our findings offer physicians a guide - based on the genetic profile of the disease and certain clinical factors - to identifying patients for whom a transplant is appropriate, and the intensity of treatment most likely to be effective."

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New research shows that quickly identifying patients with high-risk acute myeloid leukemia (AML), and speeding the process to find them a stem cell donor and performing the transplant earlier, can significantly improve their ...

Patients with the most lethal form of acute myeloid leukemia (AML) - based on genetic profiles of their cancers - typically survive for only four to six months after diagnosis, even with aggressive chemotherapy. But new research ...

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A large, nationwide study published in the journal JAMA Oncology found that people who received transplants of cells collected from a donor's bone marrow the original source for blood stem cell transplants, developed decades ...

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Genetic profiling can guide stem cell transplantation for patients with myelodysplastic syndrome - Medical Xpress

Stanford scientists describe stem-cell and gene-therapy advances in scientific symposium – Scope (blog)

Using stem cells and gene therapy to treat orcure disease may still sound like science fiction, but a scientific meeting here last week emphasizedall the fronts onwhich it is moving closer and closer to fact.

Were entering a new era in medicine, said Lloyd Minor, MD, dean of the School of Medicine, in his opening remarks at the first annual symposium of the schools new Center for Definitive and Curative Medicine. Stanford researchersare poised to use stem cells and gene therapy to amelioratea wide swath of diseases, from common diagnoses such as diabetes and cancerto rare diseases ofthe brain, blood, skin, immune system and other organs. Ultimately, the goal is to create one-time treatments that can provide lifetime cures; hence the definitive and curative part of the centers name. Stanford is a leader in this branch of medical research, Minor said, addingThis is a vital component of our vision for precision health.

Stanford has a long history of leading basic-science discoveries in stem cell biology, andis now engaged in studyingmany different ways those discoveries couldbenefit patients, saidMaria Grazia Roncarolo, MD, who leads the new center.Our job is to produce clinical data so compelling that industry will pick up the product and take it to the next stage, Roncaraolo told the audience.

Among otherevent highlights:

More coverage of the days events is available in a story from the San Jose Mercury News that describeshowAnthonyOro, MD, PhD, and his colleagues are fighting epidermolysis bullosa, a devastating genetic disease of the skin. Oro closed his talk with a slightly goofy photo of a man getting a spray tan. It got a laugh, but his point was serious: Our goal for the cell therapy of the future is spray-on skin to correct a horrible genetic disease.

Ambitious? Yes. Science fiction? In the future, maybe not.

Previously: One of the most promising minds of his generation: Joseph Wu takes stem cells to heart,Life with epidermolysis bullosa: Pain is my reality, pain is my normaland Rat-grown mouse pancreases reverse diabetes in mice, say researchers Photo of Matthew Porteus courtesy of Stanford Childrens

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Stanford scientists describe stem-cell and gene-therapy advances in scientific symposium - Scope (blog)

Stem Cell Network

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Stem Cell Network

Stem cell treatment effective in anterior cruciate ligament tear – Daijiworld.com

New Delhi, Feb 6 (IANS): With stem cell treatment effective in several health conditions, including spinal problems, doctors say the medical procedure can also help in speedy recovery of anterior cruciate ligament tear - a common chronic sports injury.

The medical procedure has advantage over surgeries because they are less invasive and focus on regeneration and healing of the tissues and ligaments rather than to cut and replace it.

In a case study, 28-year-old Mohan Verma - - a footballer suffering from anterior cruciate ligament was cured through Stem Cell treatment also known as Human Embryonic Stem Cell (HESC) in just eight months time.

Initially he was told to undergo a surgery and was informed that the complete recovery would take at least a year.

According to the doctors, HESC is used in three phases for ACL, so that the stem cells could grow, repair and regenerate the ligaments, tissues in the knee.

"Each treatment phase lasts 4-6 weeks during which 0.05 ml human embryonic stem cells is injected. The physician continuously administers the HESC. No immune-suppressants are given to the patients. In addition to HESC therapy, the patient receives physiotherapy and occupational therapy," said Geeta Shroff, stem cells specialist at Delhi-based Nutech Mediworld Hospital.

Comparing it to the conventional treatment, Shroff said: "In surgical procedure the graft goes in at a steeper angle than the original ACL which causes compression of the cartilage and hence most of the young athletes undergoing surgery end up with arthritis by the age of 30."

"Moreover the position sense and the strength of the knee can never be restored," she added.

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Stem cell treatment effective in anterior cruciate ligament tear - Daijiworld.com

‘Stem cells grew me a new heart’ says Gordon Foster after treatment … – Hull Daily Mail

An East Yorkshire dad who missed out on a heart transplant by just one per cent has become the first patient in Europe to undergo revolutionary treatment on compassionate grounds.

Gordon Foster suffered his first heart attack at 30 and went on to have two major and several smaller heart attacks which caused severe damage to his heart muscle.

Yet, despite his heart working at only 17 per cent of its normal function, he missed out on a heart transplant because it was just above the threshold of 16 per cent.

Now, Gordon has become the first patient to undergo stem cell treatment to regenerate part of his dead heart muscle through the new Compassionate Treatment Programme, the first of its kind in Europe, at the world-famous St Bartholomew's Hospital in London.

Read more: Migraines change Hull mum's accent into Scouse, Geordie and Irish

At his home in the hamlet of Thornholme near Bridlington, Gordon, 59, said: "I thought last year would be my last. Now, I have so much hope for the future.

"I have had the best care I could ever wish for from the NHS. I can't believe how lucky I've been. Someone up there must be looking out for me."

Gordon developed cancer when he was 22 and believes his illness may have been linked to his father's work for the RAF testing British nuclear weapons in Christmas Island in the 1950s. Around 21,000 British servicemen were exposed to explosions wearing no protective clothing and dressed in khaki desert fatigues.

He said: "I remember as a child, the radio used to crackle when my dad walked past and when he died, he was riddled with cancer."

Gordon underwent intensive radiotherapy which may have damaged his heart and suffered the major heart attack eight years later as he renovated his home for him and his bride-to-be Joanne. The couple brought forward their wedding day in case he did not survive.

However, they went on to have two children James, now 25, and Rebekah, 23, but Mr Foster's health declined over the years despite him throwing himself into his job as a welder fabricator for structural steel firm Severfield Reeve.

"I worked in heavy industry, handing upside down off beams welding, and I was putting in 110-hour weeks, often away from home for seven weeks at a time," he said.

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"I just got it into my heard that I had to prove to people that I wasn't on the scrap heap and threw myself into work. With hindsight, it was the wrong thing to do."

After experiencing more heart attacks and being told by a cardiology specialist he needed a heart transplant, Gordon went to Papworth Hospital in Cambridgeshire. Tests showed his heart function rate was 17 per cent, just one per cent too high for a heart transplant.

He said: "It was a very humbling experience. I thought I had it bad but when I saw some of the other patients, it was unbelievable how sick they were.

"I thought they were far more deserving than me and I would have felt guilty if they had put me on the transplant list."

So, Gordon soldiered on, his health deteriorating by the day. And he sank into a deep depression as his life became more restrictive. Despite his employers changing his role several times in an attempt to reduce his workload, he had to give up work on medical grounds about 10 years ago.

At home, a stair lift was fitted and his home was adapted because he ended up with terrible pains in his chest and on his knees by the time he reached the top of the stairs.

Five years ago, Gordon was invited to take part in a Stem Cell Research Trial, funded by the Heart Cells Foundation, to help patients with heart failure. However, he was selected at random as part of the group receiving a placebo so his condition did not improve.

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However, in September, the Stem Cell Research Team invited him back down to St Bart's to see if he was suitable for treatment under its new Compassionate Treatment Programme and treatment began in November.

Jenifer Rosenberg, chairwoman of the Heart Cells Foundation, said the programme's aim was to treat patients with severe heart disease with stem cell therapy on compassionate grounds to give them back their lives.

For five days, Gordon had injections to stimulate the growth of his own stem cells and on the sixth day, the team extracted bone marrow from the bottom of his back.

His bone marrow was sent by express courier across London to a laboratory where scientists extracted stem cells which were then put straight into the dead section of Gordon's heart muscle in an attempt to regenerate it. And it worked.

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"When I got back home, I felt really good," he said. "I could walk up the stairs without getting the pain and it was so marvellous."

Although he still has a lung condition and is about to begin another trial at Castle Hill Hospital in Cottingham to help his breathing, his heart continues to repair itself thanks to the stem cell treatment.

He said: "I will forever be thankful to the Heart Cells Foundation and the team at St Bart's. Without them, I wouldn't be here today and I'm enjoying every moment I spend with my wife and my children.

"Not only has the stem cell treatment I received helped to improve my physical health, but it has also massively improved my mental health and I now live every day with hope for the future."

Consultant cardiologist Professor Anthony Mathur, a director of interventional cardiology at St Bart's, said: "The launch of the programme is a momentous milestone in our research and Gordon's story proves just how important it is to offer cell therapy to those who have no other medical choice.

"With more than one million people suffering with heart disease and failure in the UK, the need for treatment in this field has never been greater.

"We hope to lead the way to the treatment ultimately being available to thousands of other patients through the NHS so we can help people like Gordon to lead near normal lives again."

Visit here to donate to Heart Cells Foundation and the Compassionate Treatment Programme.

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'Stem cells grew me a new heart' says Gordon Foster after treatment ... - Hull Daily Mail

Clinic claims it has used stem cells to treat Down’s syndrome | New … – New Scientist

Downs: an extra chromosome 21

Department of Clinical Cytogenetics, Addenbrookes Hospital/Science Photo Library

By Andy Coghlan

A CLINIC claims it has used stem cells to treat Downs syndrome in up to 14 people. As far as we know, its the first time that stem cells have been used to treat Downs syndrome, says Jyoti Titus, manager at Nutech Mediworld clinic in New Delhi, India.

The announcement has set alarm bells ringing. Its not clear to independent stem cell or Downs experts how stem cells which can form many types of tissue might treat Downs, a genetic disorder caused by having an extra chromosome. The use of these cells does not make biological sense and may place the babies at considerable risk of side effects,says John Rasko of the International Society for Cellular Therapy.

Clinically proven stem cell therapies are only just starting to become available. The first off-the-shelf stem cell treatment to gain regulatory approval was launched in Japan last year, and prevents transplanted organs from attacking their recipients. A number of research teams are putting other experimental stem cell therapies through stringent clinical trials.

But hundreds of clinics worldwide already offer stem cell treatments unvetted by regulatory authorities. A patent held by the clinics medical director, Geeta Shroff, from 2007 suggests that the cells offered by Nutech Mediworld could be helpful for over 70 types of conditions, from Downs syndrome to Alzheimers disease, and even vegetative states.

The use of stem cells doesnt make sense and may place the babies at considerable risk

Most treatments for children with Downs syndrome centre on support including speech and behavioural therapies. But in a study published last year Shroff, reported that a baby with Downs syndrome developed better understanding, improved limb muscle tone, and the ability to recognise his relatives after receiving stem cells (Journal of Medical Cases, doi.org/bx3v).

Theres no comparison to similar individuals with Downs syndrome, and no indication this therapy had any effect whatsoever, so the author has no basis at all for saying the injections were beneficial, says Elizabeth Fisher at University College London.

But since no other treatment was given, it is evident that the childs improvements were due to stem cell treatment, says Titus. He started babbling and crawling, and his facial features underwent a change. The boy, who lives in Singapore, is now 3 years old. He continues to develop age-appropriate skills, says Titus.

Shroffs study says she injected the cells, developed from a donated embryo, into his blood, back muscles and under his skin, as well as giving them as a nasal spray. Stem cells have an innate ability to repair and regenerate, and that is how the babys condition improved, says Titus.

Theres no obvious way in which this treatment would have worked, says Victor Tybulewicz at the Francis Crick Institute in London. To have any effect, neural stem cells would need to be injected into the brain, he says.

The author appears to have no idea of where [the cells] are going, or what theyre doing, says Fisher. Its even worse now we know theyve treated 14 patients, not just one.

Titus says that the way the cells were developed means recipients dont need immunosuppressants. But Tybulewicz disagrees. I expect the most likely outcome of the injections would have been that they were recognised as foreign and eliminated by the immune system, he says. More details of the biological impact of the stem cells will be revealed in a study that has been submitted for publication, says Titus.

Nutech Mediworld isnt the only clinic offering stem cells. An analysis led by Rasko last year identified 417 unique websites advertising stem cell treatments directly to patients. Of these, 187 were linked to 215 clinics in the US. Thirty-five websites were linked to organisations in India.

Although India introduced national guidelines on clinical stem cell research and treatments a decade ago, these are not legally binding.

This article appeared in print under the headline Clinic claims stem cells treat Downs syndrome

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Clinic claims it has used stem cells to treat Down's syndrome | New ... - New Scientist

Stem Cell Therapy Offers Hope to Multiple Sclerosis Patients (VIDEO) – Newsy

ByEthan Weston February 2, 2017

Stem cell research is making medical breakthroughs, and now, it could offer hope to people who have multiple sclerosis.

A newNational Institutes of Healthstudy suggests one-time stem cell transplants might be more effective than long-term medicinal treatment at treating relapsing-remitting MS.

Multiple sclerosis is an autoimmune disease that causes a person's immune system to attack their central nervous system. Common symptoms are impaired motor function, weakness and chronic pain. Relapsing-remitting MS is the most common form of the disease.

Stem cells are cells that haven't decided what they want to be when they grow up. That means they can develop into different types of cells. Because of that, they can be used to heal older damaged cells, like those attacked by the immune system.

The study followed 24 people who weren't having success with the typical MS medications. The experimental treatment suppressed participants' immune systems with chemotherapy. Then, their own stem cells were transplanted back into their bodies to rebuild their immune systems.

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Five years after treatment, most participants' symptoms were in remission. Some of them even showed some improvements.

Larger studies will be needed to confirm these findings. But the head of the study said it's a good first step toward more effective treatment for an incredibly debilitating and deadly disease.

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Stem Cell Therapy Offers Hope to Multiple Sclerosis Patients (VIDEO) - Newsy

Experimental Stem Cell Therapy Stops Multiple Sclerosis In Its … – Vocativ

The prognosis for people affected by multiple sclerosis (MS), a degenerative autoimmune disorder that decimates the central nervous system, is a bleak one. The disease oftenbegins with a sudden burst of neurological symptoms like muscle spasms, vision problems, and trouble walking, then progresses differently, depending on which form of MS someone has. But eventually, nearly everyone with the disease comesto the point of being unable to move, breathe, or live independently. And sufferers on average live anywhere from five to ten years less than the general public.

Currently, the best medications we have available do little more than slow MS down, or tamp down peoples symptoms. But an experimental therapy continues to provide the first glimmers of something ground-breaking an actual way to stop one form of the disease in its tracks, and maybe even reverse some of the damage already done.

In this months Neurology, researchers detailed the final five-year-old results of a small clinical trial called HALT-MS. Twenty-four volunteers with MS who hadnt responded to conventional drugs were first given a powerful form of chemotherapy, high-dose immunosuppressive therapy (HDIT), that wiped out their immune system. Then they were given a transplant of their own stem cells taken out earlier, known as autologous hematopoietic cell transplant (HCT). These purified cells, the researchers theorized, would seed a new generation of uncorrupted white blood cells and reset the immune system, freezing MS in its place.

For the most part thats exactly what the combination HDIT/HCT therapy did. Nearly 70 percent of patients, five years in, have experienced no signs of the disease progressing. They havent had a relapse of symptoms, become more disabled, or had new brain lesions show up in imaging exams. Some have actually improved physically in the years since the treatment. And even those not in complete remission appear to be suffering less than before. Importantly, though the treatment isnt free of side-effects, there havent been severe ones. There were three deaths seen during the trial, all of whom experienced worsening MS, but none were attributed to the treatment.

The volunteers all had relapsing-remitting MS, the most common form, in which symptoms come and go with little rhyme or reason.

The evidence at this time is encouraging, but it isnt definitive, study author Dr. Linda Griffith, a researcher at the National Institute of Allergy and Infectious Diseases (NIAID), which sponsored the study, told Vocativ.

As Vocativ has previously reported, this isnt the first trial to find similar success rates for HDIT/HCT, though it does come with its own dangers. Patients can die from it, and like all kinds of chemotherapy, the deliberate weakening of the immune system often leads to more infections. It also doesnt seem to be as effective for more advanced types of MS, when the disease has stopped causing active inflammation, said Griffith. And while it could be promising for people in the earliest stages of MS, the research needed to promote it as a first-line treatment isnt there yet either, she added.

For now, the only trials of HDIT/HCT have been small and isolated. And though the effects of it when successful seem to extend as far out as 13 years later, its too early to call it a full-on cure. We still dont have a clear grasp of why MS happens in the first place, but its thought that multiple triggers like infections and unlucky genetics combine to increase peoples risk. So even if resetting someones immune system does treat MS completely, its plausible that some percentage of patients could fall victim to it again down the road, Griffith explained. We just dont know enough right now.

But Griffith is hopeful that larger, randomized studies will be underway within the next year or so. And if those prove to be as successful as the HALT-MS trial and others, the therapy could someday soon lead to a light at the end of tunnel for the millions of MS sufferers alive today.

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Experimental Stem Cell Therapy Stops Multiple Sclerosis In Its ... - Vocativ

From Down syndrome to ‘near normal’? New Delhi clinic makes stem cell claims that worry experts – National Post


National Post
From Down syndrome to 'near normal'? New Delhi clinic makes stem cell claims that worry experts
National Post
A New Delhi clinic that has claimed to help paralyzed Canadians walk again by injecting them with stem cells now says it can use the same treatment to make children with Down syndrome almost near normal. Nutech Mediworld says it has treated up to 16 ...

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From Down syndrome to 'near normal'? New Delhi clinic makes stem cell claims that worry experts - National Post

Woman grows a nose on her spine after experimental stem …

A female patient in the US has grown a nose on her back following a failed experimental stem cell treatment that was intended to cure her paralysis. The nose-like growth, which was producing a thick mucus-like material, has recently been removed as it was pressing painfully on herspine. If you ever needed an example of the potential perils of stem cell therapy, and just how little we actually know about the function of stem cells, this is it. Its also notable that this stem cell therapy was carried out in a developed country, as part of an approved trial (apparently unwanted growths are more common in developing nations with less stringent medical safeguards).

Eight years ago, olfactory stem cells were taken from the patients nose and implanted in her spine. The stem cells were meant to turn into nerve cells that would help repair the womans spine, curing her of paralysis. Instead, it seems they decided to do what they were originally meant to do and attempt to build a nose. Over a number of years, the nose-like growth eventually became big enough and nosy enough to cause pain and discomfort to the patient. As reported by New Scientist, surgeons removed a 3-centimetre-long growth, which was found to be mainly nasal tissue, as well as bits of bone and tiny nerve branches that had not connected with the spinal nerves. [DOI: 10.3171/2014.5.SPINE13992 Autograft-derived spinal cord mass following olfactory mucosal cell transplantation in a spinal cord injury patient]

Your olfactory system. 1 is the olfactory bulb (the bit of your brain that processes smells); 6 is the olfactory receptors that bind to specific chemicals (odors). [Image credit: Wikipedia]

What went wrong, then? Basically, at the top of your nasal passages there is the olfactory mucosa. This region contains all of the machinery for picking up odors, and the neurons for sending all of that data off to your brains olfactory bulb for processing. Cells from this region can be easily and safely harvested, and with the correct processing they behave just like pluripotent embryonic stem cells that can develop into many other cell types. These olfactory stem cells could develop into cartilage, or mucus glands, or neurons. The researchers obviously wanted the latter, to cure the patients spinal nerve damage but seemingly they got it wrong, and thus she sprouted a second nose. Moving forward, newer olfactory stem cell treatments have an isolation stage to prevent this kind of thing from happening. [Read:The first 3D-printed human stem cells.]

Its important to note that medicine, despite being carried out primarily on humans, is still ultimately a scientific endeavor that requires a large amount of trial and error. In the western world, its very, very hard to get a stem cell therapy approved for human trials without lots of animal testing. Even then, the therapies are often only used on people who have nothing to lose. Obviously its hard to stomach news like this, and Im sure that stem cell critics will be quick to decry the Frankensteinian abomination created by these scientists. But when you think about the alternative no advanced medicine and significantly reduced lifespans for billions of people then really, such experimental treatments are nothing to sneeze at.

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Woman grows a nose on her spine after experimental stem ...