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tv   Vital Signs with Dr. Sanjay Gupta  CNN  June 24, 2017 11:30am-12:01pm PDT

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term before, stem cells but what exactly are they? where do they come from? how are they used? the answers might surprise you. this is "vital signs." i'm dr. sanjay gupta. we're at emery university in atlanta, georgia. where back in 2010, stem cells were injected directly into patients' spinal cords. it was the first approved trial of its kind. >> one thing that's critical in my opinion is that the injection be done in a very slow and controlled fashion. >> imagine being told you have an incurable disease. then imagine being a test subject for an experimental procedure, one that is not even designed to necessarily cure your illness. >> i don't honestly think this is going to make you better, which means that the reason that you're doing this is to help other people. >> right. >> this was a phase one clinical trial that was investigating the safety of infusing stem cells into patients with a neurodegenerative disease better
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known as als. als disrupts the connections between the brain and the muscles. it leads to weakness in the arms and legs and often the mouth and the throat causing issues with speech and swallowing. currently there is no cure for als. doctors can only try to manage the symptoms for their patients. the average life expectancy, only two to five years. that's why doctors and a team at emery began looking into stem cells more than seven years ago. we're back today to revisit and see how far the research has come. why was there this idea injecting stem cells into the spinal cord could even help? >> let me ask you something. what was the thinking that this could work? if you go way back, why was there this idea that injecting stem cells into the spinal cord could even help? >> yeah. so actually that's a great question, because the answer is counter intuitive. what most people think when they're talking about stem cell social security that we're going to create a cell and replace a cell in the nervous system.
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we're not repair or replacing motor neuron, we're trying to have them heal and grow back. >> does that mean that if you're protecting them that the patient won't get worse? >> that's exactly right. >> stem cells are located throughout our bodies, our reserve army of regeneration or repair. when we're injured or sick, stem cells will divide and create new cells. depending on where theells are in the body they adapt becoming specialized blood cells, muscle cells or brain cells for example. >> it was almost as if the cells were guided missiles that could actually detect where there's a problem and go there. >> for this trial neurofetal stem cells were transplanted into the patients with the hope they could become functioning cells in t cells spinal cord. he even developed a special device for the procedure, it stabilizes and controls the needles delivering the stem cells directly into the spinal cord. so this is it? >> yeah.
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>> the work in the lab has proven that where you put the cells matters and the spinal cord is not just a homogeneous blob. you need to pump the cells in slowly. and you had to be sure that that needle was going to stay fixed with regard to the patient. because things happen in the operating room. >> there was one patient that was transplanted at one of the other centers who experienced a significant deterioration in neurologic function. i think that was a bit of a wakeup call for us that this is not necessarily entirely safe. but we knew that. and we're dealing with a terminal disease. >> ultimately none of this would even be possible without the brave patients like ed. ed is 71 years old and there's no place he'd rather be than outdoors. a world traveler, a mountain climber, a runner, a biker, and then a diagnosis that stopped him in his tracks. >> i know the day, the time, the hour. we were in bangkok and i was running. i was in the middle of a race and my ankle started to rl t
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the left and my left leg. so that had never happened before. i knew about lou gehrig and i knew what als was and the tests were pretty conclusive. i went from the fast emotional to the practical and what do i do to live with this, what do i do to try to further research and help the science and -- >> to help the science ed asked the team at emery about the stem cell clinical trials. he heard the same speech you saw from 2010. this might not help you, the doctors told him. in fact, it could kill you. fearless as he'd been his whole life, ed was ready to take his chances. >> what are my options? it's 100% fatal disease so i could, you know, get my affairs in order which used to be what doctors would tell you with this diagnosis or i could take a shot at something. >> in 2011 ed had a stem cell transplant.
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1.5 million cells injected directly into his spinal cord. the operation was successful with minimal side effects. >> there are a lot of patients who exceeded expectations and that's a digression, but it left us wondering, wow, they did a lot better than we expected. that's important. that is implications for how we move forward. >> moving forward is a slow process. in the united states, stem cells are partly mired in the controversy regarding fetal stem cells currently hung up in fda regulations and cost prohibitive. tens of millions of dollars to conduct one phase of a trial li this. the team at emery are currently working to raise the funds for the third phase of this trial. but it's risk and reward. even slow progress is still progress. >> now that we understand the limitations of having done this the first time we're developing techniques to try and inject the
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spinal cord in the mri scanner so there's a lot of details, questions that we would have never asked had we not done the first round of trials. >> six years after the transplant, ed's als progression is slower than normal. it hasn't yet reached his respiratory system. whether the stem cells had something to do with it or not, it's too early to tell. he's still outdoors as much as he can be, traveling the world in his wheelchair. even throwing out the first pitch with his son at a chicago cubs baseball game. >> i was happy to be part of something that looked forward, and then there's that part of all of us where you say it may not help me but there's something down the line. everybody makes some decision like that. you know, because it's hope. i mean, hope is a big deal. >> in other areas of the world, stem cell research is sometimes a much more advanced and applied treatment. so next we head over to germany where one doctor is helping people get back on their feet
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using their own cells. "vital signs" is brought to you by dubai health care city. go to cnn.com/vitalsigns for global perspective on stories impacting the world around us. i'm ryan and i quit smoking with chantix.
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♪ all come to look for america life's as big as you make it. the all-new 7-seater volkswagen atlas with america's best bumper-to-bumper limited warranty. bill marlet is long way from his home in sioux falls, south dakota. he's in munich, germany, to meet with dr. ekard alt.
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>> good to see you. >> have a seat. >> and even the simplest of greetings, this handshake would have been nearly impossible just eight months ago. >> day-to-day, shaking hands was very painful. >> range of motion is back. >> putting on my jacket, reaching up, like anything that had to do with bending my wrist down was painful. tucking in my shirt hurt and it -- it just got to be so every once in a while i'd wake up in the middle of the night and it would be aching and it was something needed to happen. >> bill lost his arm in a motorcycle accident as a teenager. but the active kid from south dakota didn't stop playing sports like football and wrestling or running track. over the years it put a strain on his remaining hand and wrist. >> there's no cartilage so it's just bone on bone. then there's holes in the bones, and then that was irritating some of the tendons that go across it, and so it was just constantly inflamed, and very sore.
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>> bill works in finance for a company called sanford health. he saw a sanford doctor about his wrist pain who prescribed anti-inflammatory drugs that managed pain but did not heal. then his doctor saw a presentation by this man whose clinic in munich was partnering with sanford. it was for a treatment using stem cells. >> following a wound or trauma or infectionhere comes a call to the stem cells in the blood vessels that are silent, they're sleeping there and nature activates those cells. these cells are able to form any tissue depending on where you inject them. >> in the united states much of the stem cell conversation is controversial especially surrounding embryonic stem cells. in the meantime though, research continued overseas and clinics like this one were approved by
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the european equivalent of the fda, the e.m.a. >> the initial challenge was the ethics. there are ethical concerns but i think this is overcome. >> that means cells that come from your own body. he was the first to use adipose tissue or fat as a prime source for stem cells. he says stem cells are also particularly good at becoming cartilage and bone. >> the trick is how to release those cells. >> here's how it works. under local anesthesia they remove fat tissue using lipo suction. then a few rounds of centrifuge. an enzyme mixture helps separate the cells from the fat and the remaining stem cells are reinjected into the body where the treatment is required like in the knee instead of a knee replacement or in bill's case, directly into the wrist.
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>> my wrist felt better almost with the next coup wks and through the course of the next seven months, it continued to feel better and better. >> these are bill's mri scans from before and after the injection. >> all that white stuff which was inflammation and cysts and inflammation in the bone and in the metacarpal in these bones in your hand pretty much is gone and we see cartilage has been formed in between those little metacarpal bones. >> since you're using your own cells, there's no need to find a match like you would for a bone marrow transplant. that's the most widely used stem cell therapy. there's also little to no chance of rejection by your immune system. >> with any implant you do you have anninfection risk or risk
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of failure. and there are so many patients that suffer from infections. so with stem cells that's a more natural way to heal your defect. >> for patients like bill who paid his own way to germany for the treatment, there aren't many options back home in the states. but as focus shifts from embryonic stem cells to adult stem cells that could start to change. in the united states, there are numerous clinical trials underway using adult stem cells including the first fda trial for rotator cuff treatments. led by sanford and what they've learned from dr. alt who sees great potential for the future. >> i think it will be exponential. it will be the same thing as with the deciphering the human genome. >> stem cells show promising resultfor a physical injury or an illness, but what about developmental injuries in the brain? we're head back to the united states to see how stem cells could impact autism. [ indistinct chatter ]
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today 7-year-old gracie has a smile a mile wide as she plays with her sister on the playground. swinging, sliding and giggling, the gracie we see here wasn't always this way. >> where's mama? >> right before the age of two we were noticing some things that were peculiar. >> gracie would be off to herself in her own world. the other thing was hand
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flapping. in the very beginning when she was a toddler it was really, really bad. it was kicking, screaming, spitting, hitting, we would have tantrums pretty much daily. >> no cereal. we're going to eat dinner. >> oh, are you mad about that? >> it was very difficult just not being able to communicate with her, her being able to tell us what she wants. the day-to-day life was definitely a struggle. >> autism is a spectrum of disorders typically characterized by abnormalities in communication, social skills and repetitive behaviors. this can be diagnosed by age two but more often is not diagnosed until later. there is no cure but behavioral therapy, medications or a
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combination of the two can help improve daily function. when gracie was five they discovered an experimental treatment that could possibly help her. >> i got an e-mail that said duke university was doing this study with core blood stem cells and infusing it with kids with autism to see if it could help and right away, i clicked respond. >> study at duke university is the first of its kind to look at treating autism in children using their own stem cells from umbilical cord blood banked at birth. >> so the study tested whether the child's umbilical cord blood would be safe in treating autism and what end points or what measures we could look at to see if they could improve. so cord blood left over actually contains lots of different kinds of cells.
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and children with autism, we're relying on the blood that's called a monosite. >> they hope they will reduce inflammation and rebuild connections in the brain. >> cells are smarter than drugs and when we put cells in the body they can take multiple actions and be smarter about where to go. >> happy birthday to you! >> it was the unknown, but we life and be able to do the n thint others can do. we were willing to try anything at that point. >> gracie was one of 25 children accepted into the phase one trial. the main stipulation, the child must have had their umbilical cord blood banked at birth, a more common practice these days. >> they did not tell us what they expected it to do so we didn't know what to expect. >> the study was quote, open label.
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that means that everyone, the doctors, the families, they all knew that the therapy was being administered. >> so these cells are not manipulated. we're just watching them and infusing them to the child. >> each child received 1 to 2 billion cells given through an i.v. at six months and then a year later the children returned for observation. >> generally we saw behaviors improve in the children at six months compared to their baseline studies. >> i wonder if it's anything else we could use. >> 70% did report behavioral improvements. but the doctor is cautious about those results. >> my role is to evaluate the children and to determine, have they actually responded to this treatment. that's actually a complicated task because autism is very variable. >> anything else you see happening in that picture?
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>> let me see. >> so we watched the way the child is interacting with their parents while they're playing with them and we do sort of structured tests where we're eliciting social behaviors. we did see some positive results. some children who were not speaking very much had big increases in their vocabulary and functional speech. many children were able to attend to play and meaningful communication in a way that they weren't before. and some children had less repetitive behaviors. >> cautious optimism. both researchers cannot stress that enough. that is also the sentiment of the advocacy group autism speaks. chief scientific officer thomas fraser said the findings of the initial study were encouraging but more work needs to be done. he hopes it does not spark a movement to bank core blood under unproven pretenses.
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bottom line, there are still many unanswered questions. so a second study is now underway. this time with more than 100 children. it's a randomized double blind placebo controlled trial. that's the gold standard in researching potential new treatments. >> that will allow us to have much more confidence in saying that the treatment is actually working. >> in addition in this second study, children are receiving either their own cells or if they don't have their own cells they're receiving donor cells. and at the end of this trial we'll be able to answer several questions. one, do cells help compared to placebo and two, are donor cells equivalent to or better than or worse than the child's own cells. >> we don't know if this therapy wi be curative for autism but i am hopeful that it could be curative in the long run.
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>> to see where she was and see where she is now, it's amazing. i would just say i mean, it's a miracle. >> before the study, autism affected probably 75% of our day and now autism maybe affects 10% of our day. >> stem cell has a long way to go, but for unique ability could offer hope for presently incurable diseases and beyond. >> "vital signs" is brought to you by dubai health care city. go to cnn.com/vitalsigns for a global perspective on health stories impacting the world around us.
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is why it took you so long to come here. expedia. everything in one place, so you can travel the world better. hello on this saturday. you are in "cnn newsroom." i'm ana cabrera in new york. president trump is now responding to a stunning report in "the washington post" that details how and when president obama learned that russia was leading a major campaign to sway last year's election. president trump tweeting this, just out, the obama administration knew far in advance of november 8th about election meddling by russia. did nothing about it. why? the post report alleges the cia first told president obama last august about moscow's interference. the intelligence even detailed vladimir putin specific instructions

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