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Vital Signs with Dr. Sanjay Gupta

Various Stem Cell Therapies Profiled; Possible Effect of Stem Cell Therapy for ALS and Autism Treatment Examined. Aired 2:30-3P ET

Aired June 24, 2017 - 14:30   ET


[14:30:00] DR. SANJAY GUPTA, CNN CHIEF MEDICAL CORRESPONDENT: You've probably heard the 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 Emory 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.

DR. NICHOLAS BOULIS, EMORY UNIVERSITY: One thing that's critical in my opinion is that the injection be done in a very slow and controlled fashion.

GUPTA: 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.

BOULIS: 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.


GUPTA: This was a phase one clinical trial that was investigating the safety of infusing stem cells into patients with a neurodegenerative disease amyotrophic lateral sclerosis, better 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 Nick Boulis, John Glass, and a team at Emory began looking into stem cells more than seven years ago. We're back today to revisit Dr. Boulis and see how far the research has come.

GUPTA: Why was there this idea injecting stem cells into the spinal cord could even help?

BOULIS: Yes. So actually that's a great question because the answer is counterintuitive. What most people think when they're talking about stem cells is that we're going to create a cell, and we're going to replace a cell in your system. So we're not repairing or replacing motor neurons. We're trying to protect them and induce them to heal and grow back. GUPTA: From the protective side first, so what does that look like?

Does that mean that if you're protecting them that the patient won't get worse?

BOULIS: That's exactly right.

GUPTA: Stem cells are located throughout our bodies, our reserve army of regeneration and repair. When we're injured or sick, stem cells will divide and create new cells. Depending on where the cells are in the body they adapt, becoming specialized blood cells, muscle cells, or brain cells for example.

BOULIS: It was almost as if the cells were guided missiles that actually could detect where there's a problem and go there.

GUPTA: For this trial neuro-fetal stem cells were transplanted into the patients with the hope they could become functioning cells in the spinal cord. Dr. Boulis 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?

BOULIS: Yes. The work in the lab has proven that where you put the cells matters. And the spinal cord is not just a homogeneous blob. So you've got to do the targeting correctly.

GUPTA: Were there any safety problems.

BOULIS: It turns out the surgery proved to be surprisingly safe to the point at which I think a lot of us got cavalier about it. There was one patient that was transplanted at one of the other 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.

GUPTA: Ultimately none of this would even be possible without the brave patients like Ed Tessaro. 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.

ED TESSARO, ALS PATIENT: 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 roll to 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.

GUPTA: To help the science, Ed asked the team at Emory 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. TESSARO: What are my options? It's 100 percent 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.

GUPTA: In 2011 Ed had a stem cell transplant, 1.5 million cells injected directly into his spinal cord. Dr. Boulis performed the operation, which was successful with minimal side effects.

[14:35:00] BOULIS: 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 has implications for how we move forward.

GUPTA: Moving forward is a slow process. In the United States, stem cells are partly mired in the controversy surrounding embryonic and fetal stem cells, partly hung up in stringent FDA regulations, and cost prohibitive. Tens of millions of dollars to conduct just one phase of the trial like this. Dr. Boulis and the team at Emory 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.

BOULIS: Now that we understand the limitations of having done this the first time we're developing techniques to try and inject the 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.

GUPTA: Six years after the transplant, Ed Tessaro'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.

TESSARO: I was happy to be part of something that looked forward, and then there's that part of all of us where you say, well, it may not help me but there's something down the line. Everybody makes some decision like that, you know, because it's something, it's hope. 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 using their own cells.


[14:40:39] GUPTA: Bill Marlette is a long way from his home in Sioux Falls, South Dakota. He's in Munich, Germany, to meet with Dr. Eckhard Alt.

BILL MARLETTE, PATIENT: Dr. Alt. Good to see you.

GUPTA: And even the simplest of greetings, this handshake, would have been nearly impossible just eight months ago.

MARLETTE: 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 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.

GUPTA: 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.

MARLETTE: There's no cartilage, so bone on bone. Then there's because of the stress on the bones, 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.

GUPTA: Bill works in finance for a company in Sioux Falls called Sanford Health. He saw a Sanford doctor about his wrist pain who prescribed a regimen of anti-inflammatory drugs that manage pain but did not heal. Then his doctor saw a presentation by this man, Dr. Echhard Alt, whose clinic in Munich was partnering with Sanford. It was for a treatment using stem cells.

DR. ECKHARD ALT, MUNICH: Following a wound or trauma or infection, there comes a call to the stem cells in the blood vessels which 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.

GUPTA: In the United States much of the stem cell conversation is controversial especially surrounding embryonic stem cells and fetal stem cells. Clinics have popped up across the country, but they lack oversight from the U.S. Food and Drug Administration. In the meantime, though, research continued overseas, and clinics like this one were approved by the European equivalent of the FDA, the EMA.

ALT: The initial challenge was the ethics. When you use embryonic cells there are ethical concerns. But I think this is overcome with autologous cells.

GUPTA: Autologous cells, that means cells that come from your own body. Dr. Alt was the first to use adipose tissue, or fat, as a prime source for stem cells. He says stem cells derived from adipose tissue are also particularly good at becoming cartilage and bone.

ALT: The trick is how to release those cells.

GUPTA: Here's how it works. Under local anesthesia Dr. Alt's team removes some fat tissue using liposuction. The extracted tissue then undergoes a few rounds of centrifuge to dissolve in an oil in connected tissue. An enzyme mixture also helps separate the cells from the fat. Then the remaining stem cells are re-injected 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.

MARLETTE: My wrist felt better almost within the next couple weeks, and through the course of the next seven months, it continued to feel better and better.

GUPTA: These are Bill's MRI scans from before and after the injection.

ALT: 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.

GUPTA: 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.

ALT: With any implant you do you have an infection risk or you have a risk of failure. And there are so many patients essentially in U.S. and in Europe which suffer from infections. So with stem cells that's a more natural way to heal your defect.

[14:45:01] GUPTA: 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 approved trial for rotator cuff treatments led by Sanford and what they've learned from Dr. Alt, who sees great potential for the future.

ALT: I think it will be exponential. It will be the same thing as with the deciphering the human genome.

GUPTA: Stem cells show promising results for a physical injury or an illness, but what about developmental disorders in the brain? We're headed back to the United States to see how stem cells could impact autism.


GUPTA: Today seven-year-old Gracie Gregory 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.


WADE GREGORY, GRACIE'S FATHER: Right before the age of two we were noticing some things that were very peculiar.

GINA GREGORY, GRACIE'S MOTHER: Gracie would be off to herself in her own world. The other thing was hand flapping. In the very beginning when she was a toddler it was really, really bad. [14:50:07] 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.

GUPTA: Autism is a spectrum of disorders typically characterized by abnormalities in communication, social skills, and repetitive behaviors. This developmental disorder can be diagnosed by age two but more often is not diagnosed until later. There is no cure, but behavioral therapy, medications, or a combination of the two can help improve daily function. When Gracie was five the Gregory family discovered an experimental treatment that could possibly help her.

GINA GREGORY: I got an e-mail one day 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."

GUPTA: The study at Duke University at Durham, North Carolina, 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.

DR. JOANNE KURTZBERG, DUKE UNIVERSITY: So the study tested whether the child's umbilical cord blood would be safe in treating autism. And then we defined what end points or what measures we could look at to see if they could improve. So cord blood, which is the babies blood left over actually contains lots of different kinds of cells. And children with autism, we're relying on the blood that's called a monosite (ph).

TRUMP: Dr. Joanne Kurtzberg and her research team hope these cells will reduce inflammation and rebuild connections in the brain.

KURTZBERG: 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.


WADE GREGORY: It was the unknown, but we want our child to succeed in life and be able to do the things that others can do. We were willing to try anything at that point.

GUPTA: 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.

GINA GREGORY: They did not tell us what they expected it to do so we really didn't know what to expect.

GUPTA: The study was, quote, "open label." That means that everyone, the doctors, the families, they all knew that the therapy was being administered. KURTZBERG: So these cells are not manipulated. We're just watching

them and infusing them into the child within a few hours.

GUPTA: Each child received one to two billion cells given through an I.V. At six months and then a year later the children returned for observation.

KURTZBERG: Generally we saw behaviors improve in the children at six months compared to their baseline studies.

UNIDENTIFIED FEMALE: I wonder if it's anything else we could use.

GUPTA: And 70 percent did report behavioral improvements. But Dr. Geraldine Dawson, Dr. Kurtzberg's counterpart, is cautious about those results.

GERALDINE DAWSON, DIRECTOR, DUKE CENTER FOR AUTISM AND BRAIN DEVELOPMENT: 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?


DAWSON: 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.

GUPTA: Cautious optimism -- both researchers cannot stress that enough. That is also the sentiment of the advocacy group Autism Speaks. Chief scientific officer Thomas Frazier 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. 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.

[14:55:07] DAWSON: And that will allow us to have much more confidence in saying that the treatment is actually working.

KURTZBERG: 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?

GINA GREGORY: We don't know if this therapy will be curative for autism, but I am hopeful that it could be curative in the long run. WADE GREGORY: To see where she was and see where she is now, it's

amazing. I would just say, I mean, it's a miracle.

GINA GREGORY: Before the study, autism affected probably 75 percent of our day, and now autism maybe affects 10 percent of our day.

GUPTA: Stem cell therapy has a long way to go within the lab and in the eyes of the public. Research has been slow and progress even slower. But with their unique ability to develop into any type of cell in the body, stem cells could offer hope for presently incurable diseases and beyond.