Malia

March is Multiple Sclerosis Month and Hadassah leads the world in the stunning success and implementation of the research at Hadassah Hospital in Jerusalem. Malia is just one of the many stories of triumph in Prof. Dimitrios Karussis program.

Malia was a top Dallas trial attorney-a senior partner in a law firm and the mother of three children, the youngest, age five. Her energy level was high and her life was full. And then, 18 years ago, she was diagnosed with multiple sclerosis (MS).

In the beginning, her symptoms were mild. For example, she was a little unsteady in her walking. She began taking a weekly injection of Avonex because, as her doctor told her, "You wouldn't want me to treat you for high blood pressure only after you had a heart attack."

Malia took Avonex for about 10 years. Still, her life became a journey of declining energy and mobility. Her biggest fear was that she would lose her vision and not be able to drive her children around. Fortunately, she didn't experience any problems with her vision, but her gait did get worse, as did the fatigue.

Ultimately, she could only walk a very short distance with a walker. Her balance became more unsteady. Her speech was harder to understand. And the fatigue got so bad that even taking a shower was so exhausting that she needed a nap afterward.

Malia tried four or five different medicines, but none seemed to help. As she explained, however, the promise of these medicines is only to slow the progression of the disease, not to improve upon one's symptoms.

At one point on this odyssey of decline, Malia fell and broke her leg and found herself in a wheelchair for six weeks. She began to research other treatment options, surfing the Internet extensively. It was how she came upon the innovative stem-cell work of Prof. Dimitrios Karussis, senior neurologist at Hadassah Hospital Ein Kerem and head of its Multiple Sclerosis Center. She learned that Prof. Karussis was conducting a unique MS clinical trial involving the injection of a patient's own bone marrow-derived stem cells directly into the spinal cord.

When she met Prof. Karussis for the first time, she was walking with a walker, but couldn't walk any real distance. It was December 2014. In the spring of 2015, Malia, who was accepted into one of Prof. Karussis' clinical trials, traveled back to Hadassah to have a bone marrow extraction, which would be the basis for her stem-cell transplant. As she explains, just a syringe of cells is extracted, but then these cells are enhanced in culture for six months. In the fall of 2016, Malia received her first infusion. The actual infusion, itself, Malia relates, took only five minutes!

"I got the infusion at 2 p.m.," Malia recalled, "and eight hours later, I did something I had not done in two to three years." She explained that before the infusion, whenever she got out of bed, to get back in, she would have to lift her right leg with her hands. This time, post infusion, after she got up to go to the bathroom, she was able to get back into bed by just lifting her right leg onto the bedwithout holding it! She called the kids; her husband, David, made a video of her new ability. When her doctors came in that next morning to see how she was doing, she showed them her new skill. "Everyone was sobbing," she recalled; "we were all so excited by how amazing it was."

Malia said: "The most important thing of all was that the level of my fatigue was so improved."

Taking herself back to the night she returned home from that first infusion, Malia recalled: "We arrived back home at midnight. The next morning at 8, I was up writing my blog. Then I unpacked, did laundry, and made a five-course dinner for my husband, after which he went to bed, exhausted from the jet lag. I, however, stayed up to crochet a new blanket until midnight."

That renewed energy has never disappeared since Malia started getting stem-cell infusions at Hadassah. She received two more in 2016 and the most recent in February 2017. The kind of improvements varied with each infusion-sometimes the improvement in her gait was most noticeable; other times, it was the clarity of her speech. With the 2017 infusion, it was her balance. A year or so ago, she could only balance herself on a "whole body vibration machine" for about a minute and a half. Then she worked this up to five minutes. After this fourth infusion, she could stay on the machine for 12 minutes and even let go of the bar with one hand.

In addition, before every infusion, a baseline measurement of her abilities is taken. The most recent baseline, Malia reported, was higher than ever before. By the same token, her overall physical improvements are the best they've been.

Malia's recovery is not without its setbacks. As the stem-cell treatment wears off, she does experience some backsliding in her improvements-perhaps in her speech or the way she walks. Nevertheless, after she has a treatment, the improvements return or surpass the previous ones.

"I had forgotten what it was like to feel like a regular person, like someone with a normal level of energy; I had forgotten what it was like to feel good," Malia said.

When she is out and about with her walker, she sees people looking at her with sympathy. But she doesn't feel sorry for herself, she notes, because she has gotten her life back. Malia feels blessed that she has come this far. "Until you've been there, you can't really understand just how important it is to feel like a regular person."

Malia had stopped practicing law when she was diagnosed with MS. But just recently she reactivated her law license. She took on a case, with a colleague. And in the beginning of April, she will be going to trial.

"When you feel like you can do things you used to do, life is as it should be," Malia said.

Malia no longer takes any medication for MS and being an MS patient is no longer a central focus of her life. But, she is now a stem-cell activist and fundraiser on behalf of Hadassah because "I feel I have been given a gift and I want to ensure that more people with MS can be helped."

While she doesn't know the other participants in the clinical trial, she did meet one man from London last time she was at the hospital for her infusion. As she relates, this man was "giddy with excitement, lifting his cane in the air with one hand." They began to talk and he explained that he just had his first stem-cell infusion and, overnight, he was able to lift his cane up in the air with his right hand when for 15 years he had been unable to even hold anything in that hand.

"What I would like Hadassah supporters around the world to understand," Malia related, "is that yes, I get the wonderful benefits of Prof. Karussis' stem-cell treatments (and, of course, she does not minimize his trailblazing hard work and miraculous success), "but I also was surprised to learn that I have this large Hadassah cheering squad that gives me hope and supports me-making me believe in possibilities for improvement. As Malia recalled, when she sits with a visitor from Hadassah and sees the look of awe on the face of this Hadassah supporter because of the leap in her improvement, it gives her renewed strength. "There are many things I want to do with the rest of my life," Malia said, "and raising money for Hadassah is at the top of the list."

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A stem cell-derived heart muscle cell. Proteins that are important for muscle cell contraction are highlighted in red and green, and cell nuclei are blue. Credit: Joseph C. Wu, M.D., Ph.D., Stanford Cardiovascular Institute

Using human heart cells generated from adult stem cells, researchers have developed an index that may be used to determine how toxic a group of cancer drugs, called tyrosine kinase inhibitors (TKIs), are to human cells. While 26 TKIs are currently used to treat a variety of cancers, some can severely damage patients hearts, causing problems such as an irregular heartbeat or heart failure.

For the study, reported February 15 in Science Translational Medicine, the researchers used stem cell-derived heart cells from 13 volunteers to develop a cardiac safety index that measures the extent to which TKIs kill or alter the function of heart cells. They found that the TKIs' toxicity score on the index was generally consistent with what is known about each drug's heart-related side effects.

This work follows on the heels of an earlier study from the same research team, published in Nature Medicine, in which they assessed the heart cell toxicity of doxorubicin, a chemotherapy drug that also causes heart-related side effects, including heart failure. In that study, the researchers used stem cell-derived heart cells from women with breast cancer to correctly predict how sensitive each womans heart cells were to doxorubicin.

Such tests could ultimately help the pharmaceutical industry identify drugs that cause heart-related side effects earlier in the drug development process and help the Food and Drug Administration (FDA) during the drug review and approval process, said the study's senior author Joseph C. Wu, M.D., Ph.D., director of the Stanford Cardiovascular Institute.

I hope this research will be helpful for individual patients, once we further implement precision medicine approaches, he added.

Ranking Heart Toxicity

To assess the potential risk of heart toxicity for drugs in development, pharmaceutical companies use laboratory tests involving animals (usually rats or mice) or cells from animals or humans that are engineered to artificially express heart-related genes. Drug candidates that appear to have an acceptable balance of benefits and risks typically proceed to testing in human clinical trials.

But there can be biological differences between these existing models and humans, so non-clinical lab tests can have significant limitations, explained Dr. Wu.

Currently, the first time humans are exposed to a new drug is during clinical trials, he said. We think it would be great if you could actually expose patients heart, brain, liver, or kidney cells to a drug in the lab, prior to clinical treatment, allowing researchers to determine whether the drug has any toxic effects.

Dr. Wu, a cardiologist by training, studies toxicities cancer drugs cause in heart cells. Human heart muscle cells (called cardiomyocytes), however, are hard to obtainrequiring risky heart surgery that may be of no direct benefit to the patientand are notoriously difficult to grow in the lab.

As an alternative, researchers have developed a method to produce heart cells from human induced pluripotent stem cells (hiPSCs). hiPSCs are created by genetically engineering normal human skin or blood cells to express four specific genes that induce them to act like stem cells. Chemical treatments can prompt hiPSCs to develop into mature cell types, such as heart muscle cells.

A large body of research has established that human adult stem cell-derived heart cells, which function and grow in cell culture, can be used as an initial model to screen drug compounds for toxic effects on the heart, said Myrtle Davis, Ph.D., chief of the Toxicology and Pharmacology Branch of NCIs Division of Cancer Treatment and Diagnosis, who was not involved in the studies.

For the Science Translational Medicine study, Dr. Wu and his colleagues set out to determine if a panel of human stem cell-derived heart cells could be used to evaluate the heart toxicity of 21 different FDA-approved TKIs.

They generated hiPSC-derived heart endothelial, fibroblast, and muscle cells from 13 volunteers: 11 healthy individuals and 2 people with kidney cancer who were being treated with a TKI. Using drug concentrations equivalent to what patients receive, the investigators next determined how lethal each TKI was to the heart cells.

They found that several TKIs were very lethal to endothelial, fibroblast, and heart muscle cells from all 13 individuals, while others were more benign.

Stem cell-derived heart muscle cells grown in a dish spontaneously contract as a beating heart does, so the researchers also analyzed the effects of TKIs on the cells beat rate, or contractility. They found that several TKIs altered the cells beat rate before they were killed by the drug treatment. If severe enough, an irregular heartbeat (called an arrhythmia), can disrupt normal heart function.

From these lethality and contractility experiments, the team developed a cardiac safety index, a 0-to-1 scale that identifies how toxic a TKI is to heart cells (with 0 being the most toxic). They then used the index to rank the 21 TKIs. The control treatment scored a 1, while a few TKIs that are labeled by the FDA with boxed warnings for severe heart toxicity scored close to 0.

Safety indices like this one can be very useful during drug discovery, said Dr. Davis, and the applicability of the index developed by Dr. Wu and his colleagues will become clear when they evaluate its performance with more compounds.

And for the safety index to be applicable to more patients, the panel of cells used to develop it would need to be gathered from a sufficiently representative population of people reflecting different ages, races/ethnicities, health statuses, and other characteristics, said Lori Minasian, M.D., deputy director of NCIs Division of Cancer Prevention, who was not involved in either study.

For example, the study did not include cells derived from patients with [pre-existing] cardiac disease, said Dr. Davis.

A Personalized Approach

In addition to their potential application during drug development, Dr. Wu believes that stem cell-derived heart cells could potentially be used to predict toxicity risk for individual patients. He and his colleagues explored this possibility in their Nature Medicine study.

Doxorubicin, used on its own or in combination with other drugs, is an effective treatment for breast cancer and several other types of cancer. Like TKIs, however, it is known to cause heart toxicities, such as arrhythmias and heart failure, in a small proportion of patients. But there has been no way to predict which patients will experience these side effects.

The researchers developed stem cell-derived heart cells from eight women with breast cancer who had been treated with doxorubicinhalf of whom experienced cardiotoxicity from the treatment and half who did not.

In several different lab tests, the heart cells from women who had experienced cardiotoxicity were more sensitive to doxorubicin than those from women who had not. More specifically, in heart cells from women who had experienced cardiotoxicity, doxorubicin treatment caused more severe irregularities in cell contractility, and even low concentrations of the drug killed the cells.

An Improved Model

While the stem cell-derived heart cell model may be an improvement over the current [drug testing] system, its not perfect, said Dr. Minasian. For example, the model does not capture contributions of other organs and cells to the toxic effects of a drug, she explained. The drug may be broken down in the liver, for instance, and side products (called metabolites) may also cause toxic effects.

In addition, the lab-grown stem cell-derived version of someones heart cells are not going to be exactly the same as the cells found in that persons heart, Dr. Wu noted. Nevertheless, they reflect the same genetics and they are pretty good at predicting drug response, he said.

Looking forward, Dr. Minasian said, figuring out how to best use this approach is going to take more work, but being able to better predict human response [to cancer drugs] is important.

The research teams next steps include conducting prospective studies to determine whether they can use a patients stem cell-derived heart cells to potentially predict if that person will develop heart toxicity before they actually receive cancer treatment.

This article has been republished frommaterialsprovided byNCI. Note: material may have been edited for length and content. For further information, please contact the cited source.

Reference

Sharma, A., Burridge, P. W., McKeithan, W. L., Serrano, R., Shukla, P., Sayed, N., ... & Matsa, E. (2017). High-throughput screening of tyrosine kinase inhibitor cardiotoxicity with human induced pluripotent stem cells. Science translational medicine, 9(377), eaaf2584.

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Selfless Ray Noble may never meet the stranger whose life he saved.

The 29-year-old registered as a stem cell and bone marrow donor four years ago after a young girl his wife knew was diagnosed with cancer.

If that was my little girl Id want someone to be there for her, he said.

Ive been a blood donor for a while, so I thought why not sign up to the stem cell register as well.

And last year dad-of-one Ray, from Wallsend, made a life-saving donation after being told he was a match for an unknown patient in urgent need of a transplant.

Now blood cancer charity Anthony Nolan have urged more people to follow Rays example after a survey revealed that 50% of young men from the North East could not be encouraged to sign up to a blood stem cell or bone marrow register for any reason.

Every year there are 2,000 people in the UK in need of a bone marrow or stem cell transplant. This is usually their last chance of survival.

For Ray, the path to becoming a blood cancer patients last hope started when a relative of his wifes friend was diagnosed with the disease.

The process was pretty simple, he said.

I followed the instructions on the Anthony Nolan website about how to sign up.

Within a week or two they sent me a spit test, where I basically had to spit into a tube and send it off so it could be analysed.

I then got a card a few weeks later saying I was on the register.

Since signing up, Ray has been identified as a potential match for two patients.

About two years ago Anthony Nolan got in touch to say that I was a potential match for someone and I had to go and give some samples.

On that one they managed to find a closer match - I was eight out of 10 and they found a 10 out of 10, which was obviously better for the patient.

Then around Christmas last year they confirmed that I was a match for someone.

After undergoing several health checks and injections to stimulate the stem cells in his blood, Ray travelled down to Sheffield in April last year to make the donation.

All in all it took about four or five hours, he said. Id been aching a bit before the procedure because of the injections but afterwards I felt totally fine.

Ray, who is dad to two-year-old Ariana, has since convinced several friends and relatives to sign up.

For me its a question of, why not?, he said.

Its not that likely that youre ever going to be asked to donate - its just a case of being on there for someone if they need it.

I always ask people: How would you feel if it was your child or parent or cousin, if they needed a donor and you werent a match - would you want someone to step up and help them?

Every 20 minutes someone in the UK finds out they have a blood cancer.

Around 2,000 people in the UK in need of a bone marrow or stem cell transplant every year. This is usually their last chance of survival.

75% of UK patients wont find a matching donor in their families. So they turn to Anthony Nolan to find them an unrelated donor.

Healthy adults aged between 16 and 30 can sign up for a simple, pain-free test through the Anthony Nolan Trust.

The charity particularly need more young men to sign up. They produce more stem cells than women and are six times more likely to donate, but make up just 15% of the register. They also need more donors from black and minority ethnic backgrounds as they often struggle to find matches for people in these groups.

Check the list of criteria to make sure youre eligible to join and fill in an application form, either online or at an Anthony Nolan recruitment event.

If you come to a recruitment event and your application is OK, you can give your saliva sample there. If you apply online, youll be sent spit kit in the post. All you need to do is spit into a small tube and post it back.

The sample will be tested and the results put in the charitys database. Every time someone needs a transplant, theyll automatically compare their tissue to yours and the 620,000 other individuals on the register.

You can donate your stem cells in two ways.

Nearly 90% of people donate their stem cells quickly and easily in a process similar to giving blood, called peripheral blood stem cell collection.

The other 10% donate through bone marrow, where they give cells from the bone marrow in their pelvis.

If youre on the register, you must be happy to donate stem cells in either way.

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'If that was my little girl I'd want someone to step up': Stem cell donor on lifesaving transplant - ChronicleLive

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A non-surgical treatment that uses a patients own bone marrow stem cells to treat chest pain or angina improved both symptoms and the length of time treated patients could be physically active, according to recent research.

We injected a catalyst molecule that caused bone marrow stem cells to enter the patients blood, then harvested them to re-inject into the patient,said Hadyanto Lim, Ph.D., study senior author.

Thirty minutes after the cell separation procedure finished, the collected stem cells were injected back into the patient through an IV.

Four weeks after receiving the treatment, patients experienced significantly fewer angina-related symptoms, and they were able to exercise at a higher intensity and for a longer period of time.

The studys limitations are the small number of patients and absence of a control group. Because no control group was used, the placebo effect cannot be ruled out, Lim noted.

Although this treatment is currently used to treat some cancers multiple myeloma and lymphoma it will need more investigation before it can be made available to the general public to treat angina, according to Lim.

The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion, and analysis. Read full, original post:Hard to treat chest pain may be improved with a patients own stem cells

For more background on the Genetic Literacy Project, read GLP on Wikipedia.

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This week a very special delivery was made from space that will help further research that could eventually lead to a mind-blowing, futuristic way to cure diseases: shooting unmanned satellite wombs into orbit and then retrieving from them batches of stem cells that can be used to treat patients. Regardless of the outcome, the scientific experiment will still advance our knowledge of these unique cells.

On Thursday Dr. Abba Zubairat the Mayo Clinic in Jacksonville, Florida, received frozen stem cells grown at the International Space Station. The package was part of the 5,400 pounds of scientific samples and equipment that splashed down on Sunday off the coast of California inside a SpaceX Dragon-10 capsule completing a historic round-trip mission.

Up there, one of the astronauts helped us to image the cells, harvest the cells and freeze them in a way that we can use them here on Earth and compare them to cells we grew here in the lab, Zubair, the principal investigator of the stem cell experiment, told Salon.

Zubairs team will look to see if the culture grown in the near-zero gravity of low-space orbit, about 250 miles above the Earths surface, results in healthier cells than onesgrownin aterrestrial lab. If so then it would helpconfirm the theory that microgravity, which resembles the weightless-likebuoyancyof female womb, is best environment for growing stemscells.

Stem cells, from which all other types of cells originate, are the bodys raw materials, and as such offer immense potential to cure many diseases. Doctors already use stem cells forbone-marrow transplants and treating blood-related diseases like leukemia, as well asfor some eye-related disorders. Researchers believe were only in the very early stages of developing revolutionary stem cell therapiesto combat cancer, Alzheimers disease, Parkinsons disease, Type 1 diabetes, heart disease and strokes. In the future, stems cellscience could even lead to growing organs in a lab that can be transplanted into humans.

But stem cells are finicky. As they replicate in a lab, many of them develop imperfections and have to be discarded. It can take a month to grow the roughly 200,000 cells needed to treat one patient, Zubair said. Gravity might be the culprit.

In nature, these cells start their life after an egg is fertilized. Humans, right from conception, develop almost in a microgravity environment, Zubair said. Fetuses develop in amniotic fluid. Theyre buoyant, which cancels the effect of gravity because theyre suspended in a liquid. Thats how three-dimensional growth in a fluid environment is possible. We think gravity does play a role in the shape and development of the cells and how organs develop.

In other words, if the cells are suspended in fluid, they can grow and move in any direction, producing more of them, compared withhow they grow on a flat surface, like in a petri dish.

This is why stem cells are typically grown in a bioreactor, a common bioengineering tool that gently stirswater containing the seed cells and certain nutrients that promote growth. But because of the way gravity affectsfluids, many of the cells become damaged and cant be used for treatment. (In the language of physics, the problem has to do with something called shearing force.) By placing a bioreactor in the microgravity of orbit, the effects of gravity on liquid mechanics is virtually eliminated.

If growing stem cells in spaceproves to be efficient, thats when things get interesting. Growing stem cells at the International Space Station is anexperimental endeavor, so its not really a viable place to begin manufacturing themin great quantities. But theoretically, Zubair says, bioreactor satellites could be put into orbit and left there to grow cells until theyre remotely called back to Earth or sent wherever future interplanetary pilgrims wind up. As the cost of sending small satellites into low orbit falls, this system could be commercially viable.

There are companies that are interested in developing a floating lab in space to grow not only stem cells but also tissues and organs down the road for human use or for use elsewhere as we hopefully colonize other planets, like Mars, Zubair said.

This might seem out of this world, but the technology for growing stem cells remotely already exists. If space is the place to grow human parts and this research will help to determine that then designing systems and deploying these bioreactor space wombs might not be that far off in the future.

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Lacey Miller will probably never forget her sons first birthday, at least not for the usual reasons. She threw him a party at a park near their home in Culver. Throughout the whole thing, she couldnt walk and could barely see, merely moving was a challenge.

I just kind of sat there and everyone cleaned up and they got me back to the house, she said.

Miller has a form of multiple sclerosis, a disease that disrupts communication between the brain and the rest of the body. The symptoms numbness, difficulty concentrating, blurred vision and fatigue come in cycles and tend to worsen over time.

When she was diagnosed in 2012 at age 27, Millers symptoms werent so bad: little things like numbness in her legs and blurry vision. It wasnt until three years later, when she became pregnant with her son, that things went downhill fast. She had to be induced into labor early because she had so much trouble walking. After that, there was vertigo, vision loss and walking became more difficult. Her worsening symptoms culminated in an emergency room trip following her sons first birthday party.

I was going to be in a wheelchair in probably a year, she said.

Internet exploration brought her to a study at Northwestern University in Chicago that is further exploring the idea of, in a sense, resetting the immune systems of multiple sclerosis patients. Multiple sclerosis develops when a persons immune system attacks the protective covering of nerve fibers in the central nervous system, making it difficult for the brain to communicate with the rest of the body.

Miller applied to join the study in June 2016 and was approved in September. She started her testing and treatment in early January, embarking on what would be months of grueling treatments and travel.

Through it all, Miller thought about her now-18-month-old son, Emmerick.

Even on the days when it was like, This sucks. This is really hard, it was like, Im doing this for him so he can have a mom who can run down to the park with him and do things with him, she said.

Thats a win

A handful of prominent studies in recent years have raised hope that a procedure called hematopoietic stem cell transplantation can prevent further disability among patients with multiple sclerosis.

A study on 25 multiple sclerosis patients published last month gave further hope. Three years after undergoing stem cell transplants, disability symptoms in nearly 80 percent of the patients had not worsened, according to the study, published in the in the Journal of the American Medical Association Neurology. At five years, nearly 70 percent of the patients symptoms still had not worsened.

Dr. Linda Griffith, an author on the study and a medical officer at the National Institute of Allergy and Infectious Diseases, which sponsored the study, said being able to halt existing symptoms is a big advance.

To us, thats a win if they dont get worse, she said. We have no notion of being able to cure MS here and make it go away. Its not going to go away. Its a bad disease. But medical investigators are really thrilled and excited when they can find that the disease isnt getting worse.

Dr. Richard Burt, a professor of medicine and chief of immunotherapy at Northwesterns Feinberg School of Medicine, found similar results in a 2015 study he and his team published in the Journal of the American Medical Association. Five years after undergoing stem cell transplants, only 10 percent of multiple sclerosis patients had worsened symptoms.

The majority got better and stayed better, he said. Thats a paradigm change.

Creating hope

The stem cell transplant procedure involves extracting a patients bone marrow stem cells using a long needle and using chemotherapy and other toxic medications to clear out the rest of the cells. In multiple sclerosis patients, the patients own stem cells are then put back into the body.

It is now immature, just like if you were a child again and your immune system was learning all over again whats its supposed to be reacting against versus not, Griffith said.

While it sounds exciting, Griffith cautions the research is far from conclusive. She hearkens back to the hype around early studies that showed the procedure could be helpful for breast cancer patients. It ultimately wasnt.

What needs to happen next, she said, is a randomized study that compares groups of multiple sclerosis patients who either receive the transplant or the medications typically used to treat the condition, following them over time.

Not for everyone

Burt, the first doctor to perform these stem cell transplants for multiple sclerosis patients in the U.S., performed the procedure on Miller.

He cautions that only a specific subset of multiple sclerosis patients will benefit from the procedure. They must have a common form of the condition called relapse-remitting, meaning symptoms come in waves that recur over time. He doesnt perform the procedure on people who have progressed to a later stage, as it would be too late to help them, he said. Imaging on their brain and spine must show new lesions, indicating the disease is active.

Its frustrating because people learn about this on Facebook and want to get it, she said. Theyre upset when we decline them.

Burt is confident, however, that he knows who his procedure can help and who it cant help.

We developed this over many decades, he said.

When Miller, who is now 32, went in for testing with Burt, it was in the middle of an attack. She could barely walk into the office at Northwestern; her dad had to hold her arm for balance.

Although she was accepted into the study, getting her health insurance carrier on board proved to be a struggle and logistical issues delayed her ability to officially participate. Burt is still treating her under the study protocols, however, on whats referred to as a compassionate basis.

Millers first trip to Chicago was a short one for pre-study testing: imaging to check the progression of her multiple sclerosis, among other evaluations. She flew back to Chicago on Jan. 21 for what would be a two-week trip. First, they gave her chemotherapy to kill her stem cells and suppress her immune system. That meant she lost her hair.

Then they put a long needle into her neck to remove the remaining stem cells. She still has a tiny, round scar where the needle went in.

On Feb. 12, she flew back to Chicago again to have her stem cells put back into her body. She was discharged on March 1.

Sometimes, Burt said his patients notice right away the so-called MS fog, the fatigue and inability to think clearly, is lifted once they finish the procedure.

Miller said that was the case for her. She noticed other things right away, too. Walking around downtown Chicago was much easier after the surgery than before. She no longer had to use a cane or wall to guide her and she no longer had to stop and rest.

The other day, her fiance, Chris, asked her what the score of the basketball game was. She told him.

He was like, Can you see that? she said. I was like, Yeah! I didnt realize things are just so crisp and clear.

The near-constant numbness in her legs also isnt as bad as it used to be.

We were kind of joking the other day because my fiance touched my foot, she said. I was like Oh my god, I can feel that! I hadnt been able to feel my feet.

No more drugs

Burt theorizes that in addition to helping peoples quality of life, stem cell transplants could also help them financially, a point he hopes to flesh out in his current research.

A 2015 study in the journal Neurology found multiple sclerosis drugs cost patients roughly $60,000 per year. The price of the drugs increased annually between 1993 and 2013 at rates of five to seven times higher than the rate of prescription drug inflation, according to the study.

Miller, who works as a juvenile parole officer in Jefferson County, estimates she was spending about $500 a month on the drugs after her insurance paid its portion. She had to quit one medication a shot she gave herself three times per week because it gave her flu-like symptoms. Not having to take medications anymore was one of the main reasons Miller said she wanted to enroll in Burts study.

Im kind of one of those people that I dont even like taking Tylenol, she said. Id rather just not take anything.

Miller isnt currently on any multiple sclerosis medications. She said she hasnt felt this good in years. She returned to work part time this week.

Burt routinely travels to medical centers around the world explaining the procedure in hopes other neurologists will perform the stem cell transplants on multiple sclerosis patients. The older ones tend to be more set in their ways, but he believes the technique will catch on among younger doctors.

The procedure is currently being performed in England, Sweden and Brazil, Burt said. A hospital in India recently expressed interest, too.

At the end of the day, we want to help people throughout the world, Burt said, and in fact this is spreading throughout the world.

Reporter: 541-383-0304,

tbannow@bendbulletin.com

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Bone Marrow Stem Cells Comments Off on Stem cell transplant reduces Culver woman’s MS symptoms – Bend Bulletin | March 24th, 2017

A good starting point for any scientist in any field is to acknowledge that theres a lot that we dont know. We dont know, for example, why there is more matter than antimatter in the universe. We dont know quite how the evolution of the dinosaurs panned out. And, perhaps most surprisingly of all, we dont know quite how many organs the human body has or what all their functions are.

This January, researchers have announced that a brand new organ had been discovered in our bodies after it had long been mistaken for something else. Now, writing in the journal Nature, a group of researchers from the University of California, San Francisco (UCSF), have found that the lungs in mice have a hidden feature too they help make blood.

Specifically, it appears the lungs produce over half of the platelets the components that bind blood together to stop us bleeding out when were wounded involved in circulation.

So not only do our breathing bags allow us to respire, but they also help keep our cardiovascular system full to the brim. Well thats rather lovely of them.

Thats not all. The researchers also managed to identify a cache of stem cells the type that can differentiate into almost any cell type with the right biological programming that can transform themselves into blood cells.

Bone marrow is thought to be the primary source of such stem cells, so this new revelation suggests that if our bone marrow is damaged and unable to keep up with its regular blood cell manufacture, our lungs can step in to make up for the shortfall.

This finding definitely suggests a more sophisticated view of the lungs that they're not just for respiration but also a key partner in formation of crucial aspects of the blood, senior author Mark Looney, a professor of medicine at UCSF, said in a statement.

A little caveat worth mentioning at this point is that this hasnt been directly imaged in humans, but mice. Nevertheless, the biological workings of these little critters is surprisingly similar to that of humans, which is part of the reason why theyre used in so many medical-themed studies so theres a good chance human lungs also possess the same hidden features.

Using a remarkable technique allowing the platelets to fluoresce, the team were able to directly trace the paths of the mousey platelets, and found they were coming from within the lungs. The megakaryocytes the platelet-producing cells are also seen moving back and forth between the lungs and the bone marrow, depending on where they are needed the most.

Perhaps studying abroad in different organs is a normal part of stem cell education, Looney added.

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Scientists Have Discovered A Secret Function Of Lungs - IFLScience

Bone Marrow Stem Cells Comments Off on Scientists Have Discovered A Secret Function Of Lungs – IFLScience | March 24th, 2017

Mumbai:A 45-year-old man, who was suffering from 45 per cent burns due to a chemical spill at work, has been healed with stem cell treatment, said the authorities at a hospital here on Friday.

Ram Naik (name changed) was brought to city-based StemRx Bioscience hospital after receiving first aid in another hospital. Nearly 45 per cent of his upper body was burned due to a chemical spill during work.

The impact of the burns led to a charred look on his face and body. Also, joint mobility due to the burns was reduced. The outer layer of the skin was affected, facial burns were of grade II level and in some instances grade III burns were also present, leading to deeper structures like the subcutaneous tissue also being affected.

According to the doctors, burn wound healing involves a series of complex processes, with healing time and scar tissue being the most important parameters that affect treatment outcomes. Burn injuries, especially severe ones, are proving to have devastating effects on the affected patients.

They said that stem cells have been recently applied in burn wounds to promote superior healing of the wounds. Not only have stem cells been shown to promote better and faster healing of the burn wounds, they are also capable of decreasing inflammation and prevent scar progression and fibrosis.

Therefore, the doctors decided to provide Naik stem cell treatment.

Regenerative Medicine researcher at Stemrx Bioscience hospital Pradeep Mahajan said that within two days, a notable improvement in his condition was observed and the swelling and charred appearance started reducing.

"Mild eyelid movements were noticed and on the third day the burns started drying on the face and he could open his mouth and eyes. Growth factors derived from platelets, cells, fibroblasts, collagen-based gel etc. was used during treatment. In addition, in areas with deep burns, sheets of PGLA coated with cells and growth factors were used," said Mahajan, adding that different medication and treatments were imparted and closed dressing was avoided.

"Blood transfusion and supplementary fluids were given intravenously to maintain systemic homeostasis," said Mahajan.

Stating that on 5th and 6th day following treatment, dry scales from the face and body started peeling off, the doctor's team also observed impressive changes such as new skin forming within a week of treatment with cells and growth factors.

By conventional modalities, it takes more than eight weeks for the patient to heal and many additional months for the patient to be able to regain joint and facial movements.

"By the 10th day of the treatment, dry scales completely peeled off and by the 14th day the patient had no tenderness or burning pain. Joint movements became free as well, Steady rate of progression of healthy skin formation is being noticed. Areas with deep burns are also healing at a rapid rate and I am confident that within a month we will accomplish thorough healing and the patient will be back to normal," Mahajan said.

Medical sciences say that such cases are challenging to manage considering the degree of impairment they result in due to prolonged healing period. Also, through conventional therapeutic modalities healing occurs with scar formation and results in contractures. Chances of systemic complications and infection are also high.

However according to the medical team, by using stem cells, the natural healing potential of the body is used, leading to reduction of healing time and promoting regeneration of affected tissues. This also reduces the mental trauma and financial burden that a patient goes through when under conventional management.

"Stem cell-based therapy has offered a novel and powerful strategy in almost every medical specialty including burns and wound management. Stem cells have proven to have tremendous potential in enhancing wound healing and facilitating skin regeneration," Mahajan said.

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Man with 45% burns healed with stem cell treatment - Zee News

Skin Stem Cells Comments Off on Man with 45% burns healed with stem cell treatment – Zee News | March 24th, 2017

Scientists claim that #Stem Cells obtained from skin provide a new weapon against brain tumors. Jedd Wolchok, a cancer immunotherapy expert at the Memorial Sloan Kettering Cancer Center, says that nanoparticles are thinner than a human hair, and help to fight tumors. Previously, doctors used stem cells to target breast cancer tumors. Latest clinical trials show that the new therapy is useful for patients with brain tumors. According to a study published in the journal "Science Translational Medicine," the treatment shrinks the tumors and extends the survival of victims.

Researcher says that it's time to forget about drugs that spur the immune system to fight tumors. Stem cells will be used on a large scale to treat patients. Every year, pharmaceutical companies develop a number of antibodies and proteins that block the overexpressed molecules, enabling the immune system to target tumors. All these medicines are harmful to the nervous system. In contrast, the stem cells directly target a tumor without damaging the neurons. Jedd Wolchok believes that the current anti-cancer drugs work in only 10% to 40% of patients. There is no use of drugs that target only several cells of a tumor and fail to completely destroy it. Stem cells destroy a tumor within a few minutes. However, the process is very complicated and only experienced neurosurgeons should perform an operation. Once a patient receives radiation therapy to shrink a tumor, his immune system mounts an aggressive response that wipes out both the tumors and metastases throughout the body.

Jedd Wolchok will find out whether it is possible to use nontoxic nanoparticles to sensitize the immune system or not. He requires more time and further research before he publishes his findings. He says that it is not easy to pass the nanoparticles through the tumors as the particles are bigger than macrophages. However, specific blood proteins can be used to coat the nanoparticles, facilitating their uptake. Once these particles reach the brain tumor, they act as tumor killers. Jedd and his team will carry out an experiment on mice with breast cancer. Wolchok builds his study on an earlier discovery that brain stem cells have a weird affinity for cancers. #Beat The Clock

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Skin cells provide a new weapon against brain tumors - Blasting News

Skin Stem Cells Comments Off on Skin cells provide a new weapon against brain tumors – Blasting News | March 24th, 2017

Before we get to the therapeutic stuff, here is a reminder of the main problem people with Parkinsons disease face.

Researchers are reasonably sure that the accumulation of a protein called alpha-synuclein is responsible for neurons dying in people with PD. However, there are two competing theories as to how it builds up andspreads,the threshold theoryandthe ascending theory(also called the prion hypothesis). The ascending theory states that alpha-synuclein spreads from cell to cell, infecting cells as the protein moves up through the brain.The threshold theory recently put forward by Dr. Ole Isacson and Dr. Simone Engelender, proposes that alpha-synuclein builds up independently in each affected cell.

Regardless, an improved understanding of exactly how such proteins misfold and clump together is at the heart of the riddle that is Parkinsons as well asa long list of other diseases. Thankfully a number of labs around the world have been working on this sticky problem. Additionally, if anyone wants to help you can do so very easily from any computer, watch this video to learn how.

The ongoing revolution in genetics is playing an increasingly important role in our understanding of the disease while also revealing whyit varies so much from patient to patient. There havebeen dozens of mutations and variants associated so far with the disease. We are just beginning to understand the role our genes play in the development of neurological diseases but an immense amount of progress has been made in the last 15 years since the human genome was sequenced. Now that sequencing costs have plummeted to around a thousand dollars we are on the verge of a new era in medicine that promises to give patients treatments tailored to their specific condition.

Personalized medicine is healthcare based on your unique genetic and molecular blueprint. Each individual has distinct genetic makeup, biomolecule and metabolic profiles, set of gut microbes, and so on. Similarly, there is no one-size-fit-all in healthcare. How you stay healthy or how you are treated for disease should be catered to match your unique profile. Knowledge of your genomics, proteomics, metabolomics, microbiotics, and other bioinformatics allow for the improvement in the quality of life, from disease prevention to therapy best suited to you. (from the Personalized Medicine Initiative in British Columbia.)

A better understanding ofgeneticswill help unlock a cascade of other problems that surround this disease includingmitochondrial dysfunction, lysosomal degradation, neuroinflammation,gut bacteria, andepigenetics, among others. And thankfully there is now a large interconnected global community of researchers working to solve these problems with more resources and better tools than in all of human history combined. This growth in a variety of public and private sector health initiatives across disciplines has lead a growing number of experts to believe that we will make more progress in the next decade than we did in the past century, which is good reason to be hopeful consideringwhat medicine was like a hundred years ago.

This medical revolution will be further bolstered by new and improved imaging techniques.A big part of the problem we still have with this disease is that we cant actually see what is wrong. Every person who has PDhas slightly different symptoms but we dont really know why primarily because we cant accurately see inside patients heads. Soon a new line of imaging techniques will be available that will give surgeons and researchers a much better understanding of what is going on inside the heads of each patient.

In addition, there are some immense ongoing collaborations such as theEuropean human brain projectand theU.S. brain initiativethat are trying to do for the brain what the human genome project did for our understanding of the genome. If successful it will give researchers unprecedented insight into how our minds are pieced together.

Then there are the new therapies themselves.

Levadopa For 50 years now this wonder drug has brought relief to millions. Of course, problems still persist, namely in getting it past that stubborn blood brain barrier and making sure a more steady supply is delivered to reduce on/off fluctuations. To get around some of those problems we now havepatches, slow release and extended release capsules, as well asintestinal pumps that deliver a steady flow of the drug directly into the intestines. Of course this drug is not an ideal solution as there are nasty side effects that come from long term use, predominantly dyskenisia which gives people the motor control of a blob of jelly, but for now, it is still the best stop-gap solution we have.

Deep Brain Stimulation This science-fiction wonder has become the undisputed Queen of modern treatments. It has already proven itself to be a miracle worker, re-animating hundreds of thousands with its electric wizardry. It too is steadily improving, from John Palfermans book,Brain Storms,Instead of implanting devices that simply deliver a continuous electrical stimulation, they are developing technologies that deliver stimulating jolts only when required. ..The idea is to design DBS so that the system can monitor the electrical activity in the basal ganglia, and when it detects an abnormal signal, it can respond automatically with an appropriate stimulation. A smart device

New Drugs There is along list of promising drugs that are already in clinical trial.Some of these drugs have the potential to not only offer symptomatic relief but hit the holy grail that is actual disease modifying therapies.

Neuromodulation techniques A number of novelneuromodulation techniques are being tested for clinical use. The most prevalent is called transcranialmagnetic stimulation in which magnets are attached to the outside of patients headsthat send a focused electric current deep into the target areas of the brain. Already an approved therapy for depression, TMS is now being tried in PD.

Immunotherapies The relatively recent identification of alpha-synuclein as playing a key role in disease formation has lead researchers to believe that we may be able to harness the bodies immune system to stop the protein from clumping while also mitigating the bodies natural inflammatory responses that damages neurons.

Pharmacogenetics The genetic revolutionhas spurred the development of a relatively new field of pharmacology called pharmacogenetics. Eventually, instead of making one drug for everybody, we will be able to tailor drugs to better fit each persons unique condition.

Stem Cell Therapies Though there were a series of trials in the 90s that had mixed results, recently a number of labs around the world have begun reexamining the therapeutic potential of stem cells. This is thanks in part to the 2007 discovery of anew type of stem cell called IPS cells which allow researchers to grow fully functioning stem cells from patients own skin cells. This has opened the door to a new set of therapies while also giving us better disease models. Since those first trials we have also made a series of other advances in our understanding of how to use stem cells which has lead to somestunning results in trials on other apes. Some labsare hoping to push forward with human trials starting at the end of this year.

Gene Modification Therapies As discussed earlier, the field of genetics is blowing up and one of the biggest benefits to society that will come from it is a new set of therapies called gene modification therapies.The most popular one today is called CRISPR, a technique that already allows researchers to cut and paste genetic code, changing the genome of living organisms. A number of articles have come out touting these kind of gene-editing techniques as the future of medicine. This first use ofCRISPRwas in a lung cancer patient in Chinalast fall, but it is also being used to help us understand neurodegenerative disordersincludingParkinsons disease.

Direct Programming In conjunction with gene therapy, direct programming is believed to bethe final solution to the problem of neurodegeneration. It is a subset of the new field of synthetic biologythatwill eventually allow us to change cell types in living organisms. For example, inpeople with Parkinsons disease we will be able toreprogram other healthy cells in the affected area, such as glial cells or astrocytes, and directly turn them into dopamine-producing cells.

When it comes right down to it, the reason why we have not been able to cure a lot of the diseases that are still with us today, such as neurodegeneration or cancer, is that there are an incredible number of factors to consider when trying to treat them, possibly too many for any human, or even any group of humans, to make sense of. But there might be a solution to this problem as we are now figuring out ways to export more and more of our intellectual abilities into computers. Already computers have become as good ashumans at diagnosing certain conditions, and astaggering number of healthcare companieshave now invested heavily in applyingartificial intelligence to the medical industry.This, along with further advances in nanotechnology,has a lot of potentialin helping us understand diseases such as Parkinsons and may reveal novel insights into how to treat them.

As you can see, there is plenty in the pipeline. While there may not be any magic bullet, there is no doubt that we will continue to see improvements in the treatment of Parkinsons disease that will benefit millions. While it is important to remain skeptical of all the promises being made, there is very good reason to believe that afflictions such as Parkinsons disease may one day be a thing of the past.

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We're About to Enter a New Era in Parkinson's Disease Treatments - Futurism

IPS Cell Therapy Comments Off on We’re About to Enter a New Era in Parkinson’s Disease Treatments – Futurism | March 24th, 2017

A small stem cell trial in which patients with severe spinal injuriesappeared to make remarkable progress is still showing excellent results, according to the company conductingthe research.

One of the patients in the trial is 21-year-old Kris Boesen, from Bakersfield, California, whose story we reported on last year.A car crash had left theBakersfield, California native with three crushed vertebrae, almost no feeling below his neck, and a grimprognosis. Doctors believed he would live the rest of his life as a paraplegic.

Enter stem cell therapy. Most treatments for serious spinal injuries concentrate on physical therapy to expand the range of the patients remainingmotor skills and to limit further injury, not to reverse the actual damage. But last April, as part of an experimental phase 2 clinical trial called SCiStar, researchers injected Boesen with 10 million stem cells. By July, hehad recovered use of his hands to the point where he could use a wheelchair, a computer and a cellphone, and could take care of most of his daily living needs.In recent months his progress has continued, says his father.

Boesen is not the only patient to have improved in the trial, according toAsterias Biotherapeutics, which is conducting the research. Boesen is part of a cohort of six patients who were experiencing various levels of paralysis and were injected with the 10 million stem cell dose. In a Jan. 24update, the company saidfive of those patientshad improved either one or twolevels on a widely used scale to measuremotor function in spinal injury patients.

On Tuesday, Asterias issued a newupdate, announcingthat the sixth patient in the cohort has experienced a similar improvement.

While spontaneous recovery for spinal injury patients does occur,the likelihood of all six patients recovering to the degree they haveis less likely, researchers say.

This is as good as you could hope at this point, said Charles Liu, Boesens neurosurgeon and director of the USC Neurorestoration Center. So far all the evidence is pointing in the right direction.

To measure improvement in spinal injury patients, researchers use two yardsticks: the Upper Extremity Motor Scale, or UEMS, and the International Standards for Neurological Classification of Spinal Cord Injury, or ISNCSCI. On the UEMS scale,patients are scored from 0 to 5 on theirability to use five key muscles in the wrists, elbows and fingers. The ISNCSCI scale assesses where damage has occurred along the different levels of the cervical vertebrae, which generally determines the scope of impairment to the body and the level of care needed.

For instance, if a patient has sustained damage at the fourth cervical vertebra down, known as C-4, at the base of the neck, it generally means that person is paralyzed from the neck down, requiring round-the-clock care and a ventilator to breathe.A patient with a C-5 injury may not be able to move her arms or hands, requiring about 6 to 12 hours per day of assisted care; and at the C-6 level, better motor function mayallow a patient to take care of most of herdaily living needs on her own.

Which is all to say that even one level of recovery could substantially improve the daily life ofa spinal injury patient.

According to Asterias, all six patients in the 10million-cell cohort have improved their general UEMS scores, and jumped at least one motor level on the ISNCSCI scale on one or both sides of their body.

Two patients have improvedtwo motor levels on one side; and one patient,Boesen, has improved two motor levels on both sides.

Steve Cartt, president and CEO of Asterias, said anotherpatient, Jake Javier of Danville, California, has gonefrom partial paralysis to being able to use his hands well enough to considerpursuing a computer science career.

Throws Like a Regular Throw

In September, Boesens father, Rod Boesen, told us how excited he wasthat his son had regained some feeling in one of his feet. Last week, at11 months post-injection, the elder Boesensaid Kris has continued to improve.

Now he can move his toe and his knee together at the same time, Boesen said. Theyre about to give him a manual wheelchair now [instead of a motorized one]. He can grip with his hands enough to use a manual one.

Boesen said the movement in his sons arms and hands has greatlyimproved since September.Kris, a formerhigh school pitcher, had beenflinging a ball to his dog like people throw hand grenades, Boesen said. They kind of cradle them and thats how Kris would do it. But now he throws like a regular throw, tosses that ball down the hall, has that release point down, and just wings it.

Asterias is currently recruiting patients for a trial in which theyll receive 20 million stem cells, the optimal dose, according to company researchers. Two patients have already started the 20 million stem cell therapy, and six-month results from those patients will be released in the fall, Cartt said.

Patients who received 2 million stem cells in an earlier phase of the study have not shown much change in their condition, according to the Jan. 24 update.

Guarded Optimism

While Boesens father is impressed with the results, the optimism of researchers inside and outside the studyhas been guarded.The trial is still in its early stages, and the sample size is small, said Paul Knoepfler, a cell biology professor and stem cell researcher at UC Davis, who is not involved in the SCiStar study.

As a scientist, I still would want to wait for more data, Knoepfler said. Its certainly interesting, but its still early. Its a phase 2 trial.

To address the issue of small sample size, Asterias islooking at historical data to determinethe level of improvement for patients in similar circumstances who did not receive stem cell therapy. The company has said it found a meaningful difference in the recovery of its study patients compared to the norm.

Liu said one of the most importantresults is the lack of significant side effects or other negative outcomes resulting from the treatment to date.

Thats very significant to me, Liu said. Thats the first thing you look for, is anyone hurt from this therapy.

There was also a concern, he said, that some patients might regress over time, once the initial injection of stem cells wore off. Thathasyet to occur.

No one has lost anything theyve gained, Liu said. We were very happy to see that. This is all very promising.

The next step for the SCiStartrial will be to establish a control group, Cartt said.

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Strong Progress for Paralyzed Patients After Stem Cell Therapy, Company Says - KQED

Spinal Cord Stem Cells Comments Off on Strong Progress for Paralyzed Patients After Stem Cell Therapy, Company Says – KQED | March 23rd, 2017

Jonathan Pitre readies for his second stem cell transplant, which will take place April 13th at the University of Minnesota Masonic Children's Hospital. Tina Boileau / -

Fully recovered from a series of infections, Jonathan Pitre has received medical clearance to undergo a second stem cell transplant.

Pitre, 16, will check into hospital on the last day of March to begin eight days of high-dose chemotherapy and one day of radiation. His stem cell transplant what doctors call Day Zero is scheduled forApril 13 at the University of Minnesota Masonic Childrens Hospital.

The night before he goes into hospital, Pitre will attend the Ottawa Senators game against the Minnesota Wild at the Xcel Energy Centre in Saint Paul. It will be a good night of fun before it all starts again, said Pitres mother, Tina Boileau.

She shared the latest news on her Facebook page on Wednesday.

After many weeks of tests, procedures and appointments at the hospital, Jonathan got the green light to proceed with the second transplant, she said. He has completely recovered from his infections and his body is as strong as can be This time it will work!

Last September, Pitre suffered nausea, hair loss, fevers and exhaustion in the aftermath of his first transplant, which ultimately failed when his own stem cells recolonized his bone marrow.His second transplant has been delayed because of lung and blood infections.

In an interview earlier this month, Pitre told the Citizen hes staying positive even though he understands the physical test that he faces in hospital.

Its mostly thinking about sticking together with the people you care about, your family, he said . You have to stick to them very, very tightly and tell each other that, Its going to be OK, and that Were stronger than this. Were going through this together, not just alone.

Pitre suffers from a rare, painful and deadly form of epidermolysis bullosa (EB), a blistering skin disease.

Hes the first Canadian to take part in a clinical trial operated by the University of Minnesotas Dr. Jakub Tolar, a pediatric transplant specialist who has adapted stem-cell therapy as a treatment for the most severe forms of EB.Although the procedure comes with the potential for life-threatening complications, it has produced dramatic improvements in two-thirds of those EB patients who have survived the transplant: tougher skin, reduced blistering and better wound healing.

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'Butterfly boy' Jonathan Pitre cleared for second stem cell transplant - Ottawa Citizen

Bone Marrow Stem Cells Comments Off on ‘Butterfly boy’ Jonathan Pitre cleared for second stem cell transplant – Ottawa Citizen | March 23rd, 2017

SEATTLE, WA--(Marketwired - March 23, 2017) - Strategic News Service (SNS) has selected lpisremo as a 2017 FiReStarter company to be featured at the 15th annual Future in Review (FiRe) Conference. FiRe 2017 will take place October 10-13 at the Stein Eriksen Lodge Deer Valley in Park City, Utah.

Described by The Economist as "the best technology conference in the world," FiRe features global speakers and participants in technology and the global economy, including Elon Musk, Craig Venter, Michael Dell, Vint Cerf, Leroy Hood, Elena Polyakova, Mark Hurd, Paul Jacobs, Cory Doctorow, Kamran Elahian, Ken Goldman, Dharmendra Modha, and many others. FiReStarters are selected based on the strength of their potential to bring positive change to the world, and are showcased at the FiRe conference in panels throughout the event and with ongoing relationships introduced and supported by SNS.

lpisremo is a disruptive precision bioengineering regenerative-medicine company whose platform technology harnesses and instructs the body's own tissue-resident cells, to produce nearly any therapeutic and regenerative protein, in place, with on/off precision, without the need to inject small-molecule drugs, mRNA, stem cells, or chemicals.

lpisremo's bleeding-edge platform enables the precision modulation of cell signaling pathways to elicit specific gene and protein expression of tissue-restricted transcription factors that are the fundamental biological mechanisms contributing to cardiogenesis, chondrogenesis, neurogenesis, vasculogenesis, skeletal myogenesis, osteogenesis, hepatogenesis, and organogenesis, which are the key mechanisms to regenerating and restoring tissue and organ function to its optimum healthy state. lpisremo's precision platform can target the key mechanisms that cause biological aging to elicit adult skin stem cells self-renewal, boost their lifespan, replenish, and elicit prevention and reversion of cellular aging ("reversing the aging process"), with the goal of making aging a reversible phenomenon.

"lpisremo's team is honored to be recognized as a 2017 FiReStarter company. We are extremely excited to share the next-generation regenerative technology at such a highly regarded technology conference, and we look forward to showing how we could revolutionize the way we treat devastating degenerative and debilitating diseases for which no cures or treatments are available today, including aging," said James Ryan, CEO of lpisremo Inc.

"We have invested years into looking at, and in some cases helping to launch, new medical discoveries, technologies, and procedures, from Personalized Medicine to Precision Medicine and including our Nutritional Microanalysis and UnDx (Undiagnosed) initiatives. lpisremo fits perfectly into this universe of brilliant and radically creative new approaches to medicine, and we are delighted to announce its selection as a FiReStarter company this year. We look forward to helping the team share their passion and research and to partnering with them in the future in making the world a better -- and healthier -- place," said Mark Anderson, Founding Chair and CEO of Future in Review and the Strategic News Service.

Future in Review is a gathering of world-class thought leaders, convened each year with the goal of providing the most accurate look forward in technology. FiRe is a world leader in exploring how technology drives the world economy and in using technology to solve major social challenges. These goals have been consistently achieved through FiRe's collaboration across technology-driven industries and through active support from the global FiRe community.

To register for FiRe 2017, go to http://www.futureinreview.com.

Strategic News Service was founded by Mark Anderson in 1995 as the first subscription-based online news service. Since its inception, SNS has proven the most accurate predictive report covering technology and the global economy. Its subscribers include top managers at technology and finance companies across the globe, including Oracle, Microsoft, HP, Dell, Cisco, Intel, Google, British Telecom, SpaceX, Amazon, Telstra, and others.

SNS has been operating the annual FiRe conference since 2003. FiRe exposes world experts and participants to new ideas, and conversations about them, producing an accurate portrait of the future; and focuses on creating technology solutions to current local and global problems. FiRe 2017 will take place October 10-13, 2017, at the Stein Eriksen Lodge Deer Valley in Park City, Utah. For more information and to register, go to http://www.futureinreview.com.

Future in Review is a Strategic News Service conference. Future in Review and Strategic News Service (SNS) are registered international trademarks. The "SNS Global Report on Technology and the Economy" is the most accurate publicly ranked predictive newsletter in computing and communications.

Websites: http://www.stratnews.com, http://www.futureinreview.com, https://www.elpiseremo.com/

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SNS FiRe Conference Names ElpisEremo a 2017 FiReStarter Company - Yahoo Finance

Skin Stem Cells Comments Off on SNS FiRe Conference Names ElpisEremo a 2017 FiReStarter Company – Yahoo Finance | March 23rd, 2017

A two-step method to make microglia Nature.com Microglia have been reported in some disease models to have beneficial effects; however, research into their potential as a cell therapy is limited by the lack of means to produce readily grafted, autologous microglial cells. Now, in Nature ...

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A two-step method to make microglia - Nature.com

IPS Cell Therapy Comments Off on A two-step method to make microglia – Nature.com | March 23rd, 2017

For a soldier who suffered a spinal cord injury on the battlefield, the promise of regenerative medicine is to fully repair the resulting limb paralysis. But that hope is still years from reality.

Not only powerful, but efficient. Studying diseases in lab-created tissue may help reduce the price tag now roughly $1.8 billion for bringing a new drug to market, which is one of the reasons Ashton received a National Science Foundation CAREER Award for advancing tissue engineering of the human spinal cord. During the projects five-year funding period, his lab in the Wisconsin Institute for Discovery will fine-tune the technology for growing a neural tube, the developmental predecessor of the spinal cord, from scratch.

As the neural tube matures and diversifies during the development of a human embryo, it gives rise to the two core parts of our central nervous system: the brain and spinal cord. By replicating this process in a dish, Ashton hopes to develop a platform for research that is highly reproducible and can be broadly disseminated. Biologists could simply add their cells to Ashtons starter tissue to build a model of whichever spinal cord disease they desire.

By starting with cells from an individual patient, researchers will be able to target disease therapies to a particular genetic background a concept known as personalized medicine. Drug tests in engineered spinal cords may become an intermediate step between animal models and clinical trials of patients affected by Lou Gehrigs disease, multiple sclerosis or spinal cord injuries, helping to bridge the differences between a human and rodent spinal cord.

Weve cured spinal cord diseases in a lot of rodents over the years, but only a small percentage of those drugs work in humans, Ashton says. If we can make the engineered tissue as close as possible to whats in our body, this will eventually translate to better drugs.

The recipe Ashton and fellow UWMadison engineering professors Lih-Sheng (Tom) Turng and David Beebe plan to follow to coax stem cells into forming a neural tube goes something like this: First, they use water-soluble Lego-type molds to create microscale cavities within a jelly-like substance. Then they add human neural stem cells into these cavities, and let the cells coalesce as they do naturally to form neural tube-resembling tissue.

Next, they add signaling molecules whose variable concentration instructs the stem cells to turn into different types of neurons and neuron-supporting cells. Last comes the greatest challenge: getting these specialized cells to connect to one another and form electric circuits that give the spinal cord its function.

The complexity of the central nervous system exists because specific circuits have to form over very long distances. If any part of a circuit goes awry, you lose function, Ashton explains. The biggest open question is whether the tissue we create in vitro will have the proper wiring of different cell types to yield circuits similar to those in our body.

Ashtons CAREER Award will also fund educational activities that range from expanding outreach programs for underrepresented minority K-12 students, to creating a website and exhibit for the public, to educating Madison-area high school teachers about tissue engineering.

Ashton, who is African-American, comes from a socially active family background his grandfather was a minister and president of the Virginia chapter of the NAACP. He has assisted the nonprofit group 100 Black Men of Madison with its K-12 mentoring programs for years.

He plans to use the new grant to develop virtual interactive lab experiments and matching teaching modules.

I hope to attract more students to the fast-growing field of regenerative medicine, Ashton says, and to motivate people to continue to fund this kind of research so that we can develop therapies to cure diseases, instead of just treating their symptoms.

This article has been republished frommaterialsprovided by University of Wisconsin-Madison. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Growing a Neural Tube in a Dish | Technology Networks - Technology Networks

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Published: March 22 2017

Stanford University School of Medicine researchers grew heart muscle cells and used them, along with CRISPR, to predict whether a patient would benefit or experience bad side effects to specific therapeutic drugs

What would it mean to pathology groups if they could grow heart cells that mimicked a cardiac patients own cells? What if clinical laboratories could determine in vitro, using grown cells, if specific patients would have positive or negative reactions to specific heart drugs before they were prescribed the drug? How would that impact the pathology and medical laboratory industries?

We may soon know. Researchers at Stanford University School of Medicine (Stanford) have begun to answer these questions.

May Be Feasible for Clinical Laboratories to Use Pluripotent Stem Cells for Assays

In a Stanford press release, researchers stated that induced pluripotent stem cells (iPS cells), coupled with CRISPRtechnology, could be used to determine:

1) Whether a patient would benefit from a specific therapeutic drug; and

2) The likelihood that the patient might have a negative reaction or bad side effect from that drug.

Thirty percent of drugs in clinical trials are eventually withdrawn due to safety concerns, which often involve adverse cardiac effects. This study shows that these cells serve as a functional readout to predict how a patients heart might respond to particular drug treatments and identify those who should avoid certain treatments, said Joseph Wu, MD, PhD, in the Stanford press release. Wu is Director of Stanfords Cardiovascular Institute and a Professor of Cardiovascular Medicine and Radiology.

The researchers believe their discovery could become a form of diagnostic and prognostic testing performed by pathologists and clinical laboratories if it passes further clinical trials.

Heart Muscle Made from Stem Cells, Study Advances Precision Medicine

The iPS cells are stem cells created in a lab, usually from a persons skin sample, and then induced into becoming cells from other parts of the body. Heart muscle cells made from iPS cells mirror the expression patterns of key genes in the donors native heart tissue. This means the cells can be leveraged to predict a patients likelihood of experiencing drug-related heart damage, according to the Stanford release.

The Stanford study also advanced precision medicine. It combined genetics, large-scale data research, and individualized testing to determine the best treatments for patients, noted an article in United Press International (UPI).

Researchers were motivated by a need to understand individual susceptibility to drug-induced cardiotoxicity, to improve patient safety, and to prevent drug attrition, according to the Stanford study, which was published in the research journal Cell Stem Cell.

Human iPS cells enable the study of pharmacological and toxicological responses in patient-specific cardiomyocytes and may serve as preclinical platforms for precision medicine, the authors noted in the study summary.

Furthermore, the researchers idea could have implications for medical conditions beyond cardiomyopathy, noted an article in LabRoots.

Cardiomyopathy is a disease of the heart muscle that affects millions of people worldwide each year.

Joseph Wu, MD, PhD (above left), and Elena Matsa, PhD (above right), both with Stanford University School of Medicine, led a team of researchers who published a study involving CRISPR that suggests heart muscle cells made from induced pluripotent stem cells (iPS cells) could be used to identify cardiac patients who could benefit from or who could be damaged by certain cardiac medications. (Photo credits: Stanford University.)

Testing Tissues in the Stanford University Research Lab

Heres how the research progressed, according to the Stanford press release:

Matsa, Wu, and their colleagues created heart muscle cells, or cardiomyocytes, from iPS cells taken from seven people not known to be genetically predisposed to cardiac problems;

They sequenced the RNA molecules made by the heart muscle cells to learn which proteins the cells were making, and by how much;

They then compared the results within individualslooking at the gene expression patterns of cardiomyocytes derived from several batches of iPS cells from each personas well as among all seven study subjects.

They also investigated how the cardiomyocytes from each person responded to increasing amounts of two drugs: Rosiglitazone (marketed as Avandia by GlaxoSmithKline), which is sometimes used to treat Type 2 diabetes; and Tacrolimus (marketed as Prograf by Astellas Pharma), which serves as an immunosuppressant to inhibit the rejection of transplanted organs. Each of the two drugs has been associated with adverse cardiac effects in some people, but it has not been possible to predict which patients will experience heart damage.

Gene expression patterns of the iPS cell-derived cardiomyocytes from each individual patient correlated very well, said Elena Matsa, PhD, Stanford Instructor, Cardiovascular Institute, and the studys lead author. But there was marked variability among the seven people, particularly in genes involved in metabolism and stress responses. In fact, one of our subjects exhibited a very abnormal expression of genes in a key metabolic pathway.

Gene Editing Reveals Drug Response Information

Enter the Clustered Regularly Interspaced Short Palindromic Repeats, or CRISPR (pronounced crisper), gene editing technology. CRISPR technology has advanced the study and practice of genetic medicine.

Researchers could not pinpoint a specific gene mutation responsible for abnormal cardiomyocyte response. But they did identify a metabolic pathway that influenced Rosiglitazones response.

They corrected the abnormality using CRISPR-Cas9 (a simplified version of the CRISPR/Cas system). This genome editing technique enables researchers to edit parts of the genome by removing or changing in some manner the DNAsequence, according to yourgenome, an information website dedicated solely to DNA, genes, and genomes.

The results? The Stanford researchers reported boosting a gene expression in the pathway, restoring normal function, and prompting a response to Rosiglitazone that was consistent to that of the other subjects cardiomyocytes.

Clinical Laboratories Become Even More Integral to Cardiac Diagnosis and Treatment

Can iPS-derived cardiomyocytes reliably replicate human heart tissue? Researchers were not sure. So, they created iPS cells from another three people who had heart biopsies or transplants. They then compared the cells made in the clinical laboratory with the gene native cells and found that they were similar in many significant ways.

In the end, cardiomyocytes derived from human iPS cells correlated with patient participants in the Stanford study. And, most importantly, the study revealed a potential ability to test drugs for adverse reactions and improve treatment for millions of people with cardiomyopathy. Should additional research confirm these findings, it could provide medical laboratories with a new approach to improving diagnosis and therapeutic selection for patients with heart disease.

Donna Marie Pocius

Related Information:

Heart Muscle Grown from Stem Cells May Help Doctors Test Treatments

Heart Muscle Made from Stem Cells Aids Precision Cardiovascular Medicine

Transcriptome Profiling of Patient-Specific Human iPSC-Cardiomyocytes Predicts Individual Drug Safety and Efficacy Responses in Vitro

Heart Stem Cells for Individualized Medicine in Cardiology

Stem Cells Create Faithful Replicas of Native Tissues, According to Stanford Study

CRISPR/Cas9 and Targeted Genome Editing: A New Era in Molecular Biology

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Challenging a long-held model about how blood is formed, a study led by UC San Francisco researchers has found that the lungs play a crucial role in the process, producing half of blood platelets and also storing blood-forming stem cells.

The study, performed in mice, also found that blood stem cells and progenitor cells travel freely between the lungs and bone marrow, long considered the primary source of blood production.

If found to occur in humans, this discovery about the lungs role in blood production could provide new approaches for treating blood diseases, pulmonologist Mark R. Looney, M.D., senior author of the study, said in a statement.

Moreover, the success of lung transplantation might be increased by better understanding this process. Immune reaction between donor blood cells in the lungs and the host could contribute to transplant rejection, the study stated.

The study was published Wednesday in the journal Nature. When placed online, the study can be found at j.mp/lungblood.

"This finding definitely suggests a more sophisticated view of the lungs -- that they're not just for respiration but also a key partner in formation of crucial aspects of the blood," Looney said. "What we've observed here in mice strongly suggests the lung may play a key role in blood formation in humans as well."

"Dr. Looney and his team have disrupted some traditional ideas about the pulmonary role in platelet-related hematopoiesis, paving the way for further scientific exploration of this integrated biology," said Traci Mondoro, of the National Heart, Lung and Blood Institute, in the statement.

While it has been known for decades that platelets can be made in the lungs, the study indicates that lung production is a more important factor than previously thought, said Mondoro, project officer at the Translational Blood Science and Resources Branch of the NHLBI, a division of the National Institutes of Health.

Researchers studied the lungs of mice genetically engineered to make a green fluorescent protein in platelets and platelet-making cells called megakaryocytes. They found a larger than expected number of these cells.

Megakaryocytes that release platelets in the lungs originate from extrapulmonary sites such as the bone marrow; we observed large megakaryocytes migrating out of the bone marrow space, the study said. The contribution of the lungs to platelet biogenesis is substantial,accounting for approximately 50% of total platelet production or 10 million platelets per hour.

After discovering this process, the researchers looked for more signs of blood cells residing in the lungs. They found progenitor cells that turn into megakaryocytes, along with blood-forming, or hematopoietic, stem cells. a total of 1 million per mouse lung.

These cells constitute a reservoir that can replenish the bone marrow, the study said.

Under conditions of thrombocytopenia (platelet deficiency) and relative stem cell deficiency in the bone marrow, these progenitors can migrate out of the lungs, repopulate the bone marrow, completely reconstitute blood platelet counts, and contribute to multiple hematopoietic lineages, the study stated. These results identify the lungs as a primary site of terminal platelet production and an organ with considerable hematopoietic potential.

The studys co-first authors are Emma Lefranais and Guadalupe Ortiz-Muoz, both of UCSF. It was supported by the UCSF Nina Ireland Program in Lung Health; the UCSF Program for Breakthrough Biomedical Research, and the National Heart, Lung, and Blood Institute.

bradley.fikes@sduniontribune.com

(619) 293-1020

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Fresh young cells

Dennis Kunkle Microscopy/Science Photo Library

By Jessica Hamzelou

BLOOD from the young seems to have healing powers, but how can we harness them without relying on donors? The discovery of a protein that keeps blood stem cells youthful might help.

The rejuvenating properties of young blood came to light in macabre experiments that stitched young and old mice together to share a circulatory system. The health of the older mice improved, while that of the younger ones deteriorated. Other animal studies have since shown that injections of young or old blood have similar effects.

This may work in people too. Young blood is being trialled as a treatment for conditions like Alzheimers, and aged mice that received injections of blood from human teenagers showed improved cognition, memory and physical activity levels.

We think the drug will improve signs of ageing and boost the immune systems of older people

But these studies rely on young people donating their blood: if this became the go-to therapy for age-related disease it would be difficult to get enough donations to fulfil demand.

The stem cells in our blood could provide an alternative approach. Our red and white blood cells are made by stem cells that themselves come from mother stem cells in bone marrow. But as we age, the number of these mother stem cells declines. One of the worlds longest-lived women seemed to only have two left in her blood when she died at age 115.

The decline in mother stem cells causes people to have fewer red blood cells, and white blood cells called B and T lymphocytes. These declines can cause anaemia and weaken the immune system. Usually the immune system in the elderly is not prepared to fight infections very hard, says Hartmut Geiger at the University of Ulm in Germany.

When Geigers team examined the bone marrow in mice, they found that older animals have much lower levels of a protein called osteopontin. To see if this protein has an effect on blood stem cells, the team injected stem cells into mice that lacked osteopontin and found that the cells rapidly aged.

But when older stem cells were mixed in a dish with osteopontin and a protein that activates it, they began to produce white blood cells just as young stem cells do. This suggests osteopontin makes stem cells behave more youthfully (EMBO Journal, doi.org/b4jp). If we can translate this into a treatment, we can make old blood young again, Geiger says.

Its exciting, says Hanadie Yousef at Stanford University in California. But longer term studies are needed to see whether this approach can rejuvenate the whole blood system, she says.

Until now, most efforts to use blood as a rejuvenation agent have focused on plasma, the liquid component, as some believe it carries dissolved factors that help maintain youth. But Geiger thinks the cells in blood might play a key role, because they are better able to move into the bodys tissues.

Both soluble factors and blood cells are likely to be important, says Yousef. While injections of young plasma rejuvenate older animals, the treatment doesnt have as strong an effect as when young and old animals share a circulatory system, she says.

Geigers team is developing a drug containing osteopontin and the activating protein to encourage blood stem cells to behave more youthfully. It should boost the immune system of elderly people, he says.

Such a drug might have benefits beyond fighting infection and alleviating anaemia. The team also think the protein will boost levels of mother stem cells. Having only a small number of such cells has been linked to heart disease, so Geiger says there is a chance that boosting them may help prevent this.

Osteopontin might also be useful for treating age-linked blood disorders, such as myelodysplasias that involve dysfunctional cells, says Martin Pera of the Jackson Laboratory in Bar Harbor, Maine. It is possible that rejuvenating bone marrow stem cells could help with these conditions, he says.

This study provides more evidence that cells can be rejuvenated, says Ioakim Spyridopoulos at Newcastle University, UK. They have made old blood look young again, although whether it acts young or not will have to be shown in clinical trials.

This article appeared in print under the headline Old blood made young again

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Plasma and stem cells: The future of regenerative medicine

Blood platelet injections and stem cell treatments may sound like the future, but physicians at the Andrews Institute are already practicing these forms of regenerative medicine.

Weight lifting mixed with normal wear and tear left Howie Webber in constant pain.

"I probably felt it about four months ago," said Howie. "I did some stretching, thinking I could make it go away, but it just continued to get worse."

That's when Howie went to the doctor and found out he had two options: surgery or regenerative medicine; he picked the latter.

"I just added up the amount of time I'd be out of work and the cost of surgery, plus the copay and this whole thing just seemed like it would be a little faster and a little easier, and it ended up being just that," said Howie.

Physicians at the Andrew's Institute currently offer two different types of regenerative medicine, platelet rich plasma, or PRP and bone marrow aspirate concentrate, or BMAC.

With PRP, physicians take the patient's blood, separate the platelets and inject those platelets back into the patient at the site of injury. The idea is that platelets carry growth factors and molecules to stimulate the healing process.

BMAC utilizes platelets too, but also the patient's bone marrow harvested from the pelvis.

Both regenerative medicine methods have benefits, perhaps the biggest according to Dr. Brett Kindle, is avoiding invasive surgeries.

"If we need surgery, we need surgery, and that's what it is, but if we can avoid it, that often times is very beneficial from a financial standpoint, missing less work, etc.," said Dr. Kindle. "Also from a quality of life, to be able to get back to doing activities in a more timely manner."

The main difference between the two is price and neither are covered by insurance. BMAC costs upwards of $3,000, while PRP costs anywhere from $600 to $800. Howie opted for PRP.

"It hurt for about three days, then within a week I was pain free," said Howie. "Maybe a little discomfort that you would expect, but it wasn't near as bad as it was before."

Howie's issue was with his hamstrings, but Dr. Kindle said both PRP and BMAC can be used to treat a variety of aches and pains.

"Anything in the limbs," said Dr. Kindle. "Shoulders, elbows, hands, wrists, hips, knees, foot, ankle, all of those areas."

Recovery for both PRP and BMAC procedures is typically one to two weeks. Full effects of the injections don't usually kick in until six to eight weeks later. For more information about regenerative medicine or to schedule a consultation with an Andrews Institute physician, call (850) 916-8700.

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