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With CRISPR-Cas9, Stanford Team Looks for Landslide Victory over Sickle Cell Disease

/ Todd Dubnicoff / Leave a comment

The results are in folks. Though it’s too early to declare a winner, it looks very likely that sickle cell disease is going to be soundly defeated by CRISPR-Cas9.

Reporting in Nature on Monday, Stanford researchers devised a method to efficiently correct the sickle cell mutation in human blood stem cells using the super-popular, user-friendly CRISPR-Cas9 genetic editing technology. Speaking to Reuters, lead scientist Matthew Porteus forecasted that clinical trials are just around the corner:

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Matthew Porteus
Image: Stanford Medicine

We think we have a complete data set to present to the FDA (Food and Drug Administration) to say we’ve done all pre-clinical experiments to show this is ready for a clinical trial

Sickle Cell Disease 101
Sickle cell disease is an inherited blood disorder that causes the generation of abnormal, sickle-shaped red blood cells in people afflicted with the disease. As a result, the misshapen cells become sticky and clump up inside blood vessels which can cause debilitating pain, anemia and organ failure. Besides pain medicine and frequent blood transfusions, the only other treatment available is a blood stem cell transplant which can be curative but carries significant risks including a high mortality rate.

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Sickle cell disease leads to abnormally shaped red blood cells (watch our video for more info)

Because the disease arises from a single DNA mutation, scientists have been working hard to use gene editing techniques as a treatment strategy. In this procedure, the patient’s stem cells are collected from their bone marrow and then the mutation is corrected in the lab. The repaired cells are transplanted back into the patient which avoids risk of immune rejection due to mismatched donor blood.

CRISPR-Cas9: the New Kid in Gene Therapy Town
Other gene therapy techniques have been successful at fixing the sickle cell mutation and are currently in clinical trials. But the more recent CRISPR-Cas9 method is much easier to carry out. Cas9 is an enzyme with an attached piece of RNA, a genetic molecule, that can be engineered to bind specifically to the sickle cell gene and, like molecular scissors, snip the DNA. This break in the DNA activates the cell’s natural DNA repair functions. At the same time, the correct sickle cell gene is delivered to the stem cells with the help of a harmless virus.

The researchers were able to correct 30 to 50% of the mutated cells using this method. That’s well over the 10% threshold that’s thought to be needed to get a clinical benefit in people. And 16 weeks after transplanting the cells into mice, the cells were still intact and healthy in the animal’s bone marrow. Just a few weeks ago, a group at UC Berkeley reported a slightly different CRISPR-Cas9 method to correct the sickle cell gene and found that about 25% of cells were corrected.

According to the Reuters interview, Porteus hopes to begin clinical trials in 2018. Add that to the sickle cell gene therapy trials already underway, including a CIRM-funded trial sponsored by UCLA, and you can’t help feeling optimistic that sickle cell disease will be voted out of the existence in the not so distance future.

Meeting the scientists who are turning their daughter’s cells into a research tool – one that could change her life forever

/ Kevin McCormack / 1 Comment

There’s nothing like a face-to-face meeting to really get to know someone. And when the life of someone you love is in the hands of that person, then it’s a meeting that comes packed with emotion and importance.

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Lilly Grossman

Last week Gay and Steve Grossman got to meet the people who are working with their daughter Lilly’s stem cells. Lilly was born with a rare, debilitating condition called ADCY5-related dyskinesia. It’s an abnormal involuntary movement disorder caused by a genetic mutation that results in muscle weakness and severe pain. Because it is so rare, little research has been done on developing a deeper understanding of it, and even less on developing treatments.

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The Grossmans and Chris Waters meet the Buck team

 

That’s about to change. CIRM’s Induced Pluripotent Stem Cell  iPSC Bank – at the Buck Institute for Research on Aging – is now home to some of Lilly’s cells, and these are being turned into iPS cells for researchers to study the disease, and to hopefully develop and test new drugs or other therapies.

Gay said that meeting the people who are turning Lilly’s tissue sample into a research tool was wonderful:

“I think meeting the people who are doing the actual work at the lab is so imperative, and so important. I want them to see where their work is going and how they are not only affecting our lives and our daughter’s life but also the lives of the other kids who are affected by this rare disease and all rare diseases.”

Joining them for the trip to the Buck was Chris Waters, the driving force behind getting the Bank to accept new cell lines. Chris runs Rare Science a non-profit organization that focuses on children with rare diseases by partnering with patient family communities and foundations.

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Steve and Gay Grossman and Chris Waters

In a news release, Chris says there are currently 7,000 identified rare diseases and 50 percent of those affect children; tragically 30 percent of those children die before their 5th birthday:

“The biggest gap in drug development is that we are not addressing the specific needs of children, especially those with rare diseases.  We need to focus on kids. They are our future. If it takes 14 years and $2 billion to get FDA approval for a new drug, how is that going to address the urgent need for a solution for the millions of children across the world with a rare disease? That’s why we created Rare Science. How do we help kids right now, how do we help the families? How do we make change?”

Jonathan Thomas, the Chair of the CIRM Board, said one way to help these families and drive change is by adding samples of stem cells from rare diseases like ADCY5 to the iPSC Bank:

“Just knowing the gene that causes a particular problem is only the beginning. By having the iPSCs of individuals, we can start to investigate the diseases of these kids in the labs. Deciphering the biology of why there are similarities and dissimilarities between these children could the open the door for life changing therapies.”

When CIRM launched the iPSC Initiative – working with CDI, Coriell, the Buck Institute and researchers around California – the goal was to build the largest iPSC Bank in the world.  Adding new lines, such as the cells from people with ADCY5, means the collection will be even more diverse than originally planned.

Chris hopes this action will serve as a model for other rare diseases, creating stem cell lines from them to help close the gap between discovery research and clinical impact. And she says seeing the people who are turning her idea into reality is just amazing:

“Oh my gosh. It’s just great to be here, to see all these people who are making this happen, they’re great. And I think they benefit too, by being able to put a human face on the diseases they are working on. I think you learn so much by meeting the patients and their families because they are the ones who are living with this every day. And by understanding it through their eyes, you can improve your research exponentially. It just makes so much more sense.”

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RARE Bears for RARE Science

To help raise funds for this work Rare Science is holding a special auction, starting tomorrow, of RARE Bears. These are bears that have been hand made by, and this is a real thing, “celebrity quilters”, so you know the quality is going to be amazing. All proceeds from the auction go to help RARE Science accelerate the search for treatments for the 200 million kids around the world who are undiagnosed or who have a rare disease.

 

Meat the future of stem cells. And I do mean “meat”.

/ Kevin McCormack / Leave a comment

'...And just a pince of stem cells.'

Over the years there have been a lot of interesting, odd ball, even a few really rather crazy stories about stem cell research that have made the news. So in honor of Halloween, we thought we’d look back at a few of them to remind ourselves that not all science is worthy of pursuit.

Celebrity meat:

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Back in 2014 a company called BiteLabs claimed it was going to make  “fine artisanal salami from meat that has been lab-grown from celebrity tissue samples.” You read that right. They were going to make salami from famous people.

Here’s how they described the process. First they would take a small sample of stem cells from the celebrity, the kind of cell that is used to grow and repair damaged muscles. Then they would grow those cells in the lab, increasing their number to millions of muscle cells. Those are then ground up, mixed with regular salami and some spices, fats and oils until you had the desired consistency and texture.

Then they were stuffed into casings, and dried, aged and cured until you end up with celebrity salami.

Not surprisingly it attracted a lot of attention. The Twitterverse was filled with images of celebrities people wanted to “eat” – Jennifer Lawrence, ‘a new kind of Hunger Games’. It was also filled with headlines from magazines like Cosmopolitan asking “Is this the weirdest food of all time”.

Turns out it was more of a joke, or at least a fun way to get people discussing bioethics and pushing the boundaries – or maybe it was the buttons – of tech and society.

Meet the most expensive meat in the world

If that was meant to be a joke then some researchers at Maastricht University in the Netherlands didn’t get it. Because the next year they actually produced a burger that was made out of stem cells.

They took some bovine – aka ‘cow’ – stem cells, grew them in the lab (this took three months so definitely not a “fast food”), then mixed them with salt, breadcrumbs and egg and cooked them in a little butter and sunflower oil.

People who tried it described it as “tough” and “not that juicy”. Harder to stomach than the burger itself was the price tag, more than $300,000.

A mammoth task

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It’s not just meat that is attracting the attention of stem cell researchers. More recently a team of Korean and Russian scientists decided it might be fun to try and use stem cells to “grow” a mammoth. You know, the giant, woolly, elephant-like creatures that went extinct thousands of years ago – except for occasional starring roles in the Ice Age animated movies.

They were going to take some DNA from the remnants of a mammoth found in the frozen tundra in Siberia, decode its genome, then create a functioning cell nucleus and transplant that into an elephant’s embryo. Easy right? What could possible go wrong (for some suggestions see Jurassic Park/World).

Maybe if that doesn’t work out they could just grow the cells into meat and market them. Mammoth burgers. Sounds yummy doesn’t it.

Happy Halloween.

 

Ingenious CIRM-funded stem cell approach to treating ALS gets go-ahead to start clinical trial

/ Kevin McCormack / 1 Comment
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Clive Svendsen

Amyotrophic lateral sclerosis (ALS), better known as Lou Gehrig’s disease, was first identified way back in 1869 but today, more than 150 years later, there are still no effective treatments for it. Now a project, funded by CIRM, has been given approval by the Food and Drug Administration (FDA) to start a clinical trial that could help change that.

Clive Svendsen and his team at Cedars-Sinai are about to start a clinical trial they hope will help slow down the progression of the disease. And they are doing it in a particularly ingenious way. More on that in a minute.

First, let’s start with ALS itself. It’s a particularly nasty, rapidly progressing disease that destroys motor neurons, those are the nerve cells in the brain and spinal cord that control movement. People with ALS lose the ability to speak, eat, move and finally, breathe. The average life expectancy after diagnosis is just 3 – 4 years. It’s considered an orphan disease because it affects only around 30,000 people in the US; but even with those relatively low numbers that means that every 90 minutes someone in the US is diagnosed with ALS, and every 90 minutes someone in the US dies of ALS.

Ingenious approach

In this clinical trial the patients will serve as their own control group. Previous studies have shown that the rate of deterioration of muscle movement in the legs of a person with ALS is the same for both legs. So Svendsen and his team will inject specially engineered stem cells into a portion of the spine that controls movement on just one side of the body. Neither the patient nor the physician will know which side has received the cells. This enables the researchers to determine if the treated leg is deteriorating at a slower rate than the untreated leg.

The stem cells being injected have been engineered to produce a protein called glial cell line derived neurotrophic factor (GDNF) that helps protect motor neurons. Svendsen and the team hope that by providing extra GDNF they’ll be able to protect the motor neurons and keep them alive.

Reaching a milestone

In a news release announcing the start of the trial, Svendsen admitted ALS is a tough disease to tackle:

“Any time you’re trying to treat an incurable disease, it is a long shot, but we believe the rationale behind our new approach is strong.”

Diane Winokur, the CIRM Board patient advocate for ALS, says this is truly a milestone:

“In the last few years, thanks to new technologies, increased interest, and CIRM support, we finally seem to be seeing some encouraging signs in the research into ALS. Dr. Svendsen has been at the forefront of this effort for the 20 years I have followed his work.  I commend him, Cedars-Sinai, and CIRM.  On behalf of those who have suffered through this cruel disease and their families and caregivers, I am filled with hope.”

You can read more about Clive Svendsen’s long journey to this moment here.

 

Know Your Stem Cell History with Gladstone’s Interactive Timeline Tool

/ Todd Dubnicoff / Leave a comment

Stem cell biology is such a young area of research. It was only in 1998 that the first human embryonic stem cell line was generated by Jamie Thomson. A dizzying amount of breakthrough research has occurred in that short span of time, including the Nobel Prize winning work of Shinya Yamanaka for devising a method for reprogramming adult cells into an embryonic stem cell-like state (aka the induced pluripotent stem cell (iPS) cell technique). Because of the compressed time frame of these discoveries, it’s hard to keep track of the key highlights and the order in which they occurred. And there are plenty of fundamental, decades-old studies which our non-scientist stem cell champions may not be aware of.

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The Gladstone’s stem cell timeline tool is fun and informative. Check it out!

That’s where the Gladstone Institutes’ new online stem cell timeline comes to the rescue. Released on October 12th, in celebration of Stem Cell Awareness Day, as well as the tenth anniversary of iPS cells, the timeline has a nifty interactive feature that allows you to swipe through a quick glance of the key milestones over the years. Then, simply tapping on a particular event gives you more detailed information. Check out it on the Gladstone Institutes website. Who knows, it might come in handy at your next pub trivia night or your next crossword puzzle.

 

Creating a “Pitching Machine” to speed up our delivery of stem cell treatments to patients

/ Kevin McCormack / Leave a comment

hitting-machine

When baseball players are trying to improve their hitting they’ll use a pitching machine to help them fine tune their stroke. Having a device that delivers a ball at a consistent speed can help a batter be more consistent and effective in their swing, and hopefully get more hits.

That’s what we are hoping our new Translating and Accelerating Centers will do. We call these our “Pitching Machine”, because we hope they’ll help researchers be better prepared when they apply to the Food and Drug Administration (FDA) for approval to start a clinical trial, and be more efficient and effective in the way they set up and run that clinical trial once they get approval.

The CIRM Board approved the Accelerating Center earlier this summer. The $15 million award went to QuintilesIMS, a leading integrated information and technology-enabled healthcare service provider.

The Accelerating Center will provide key core services for researchers who have been given approval to run a clinical trial, including:

  • Regulatory support and management services
  • Clinical trial operations and management services
  • Data management, biostatistical and analytical services

The reason why these kinds of service are needed is simple, as Randy Mills, our President and CEO explained at the time:

“Many scientists are brilliant researchers but have little experience or expertise in navigating the regulatory process; this Accelerating Center means they don’t have to develop those skills; we provide them for them.”

The Translating Center is the second part of the “Pitching Machine”. That is due to go to our Board for a vote tomorrow. This is an innovative new center that will support the stem cell research, manufacturing, preclinical safety testing, and other activities needed to successfully apply to the FDA for approval to start a clinical trial.

The Translating Center will:

  • Provide consultation and guidance to researchers about the translational process for their stem cell product.
  • Initiate, plan, track, and coordinate activities necessary for preclinical Investigational New Drug (IND)-enabling development projects.
  • Conduct preclinical research activities, including pivotal pharmacology and toxicology studies.
  • Manufacture stem cell and gene modified stem cell products under the highest quality standards for use in preclinical and clinical studies.

The two centers will work together, helping researchers create a comprehensive development plan for every aspect of their project.

For the researchers this is important in giving them the support they need. For the FDA it could also be useful in ensuring that the applications they get from CIRM-funded projects are consistent, high quality and meet all their requirements.

We want to do everything we can to ensure that when a CIRM-funded therapy is ready to start a clinical trial that its application is more likely to be a hit with the FDA, and not to strike out.

Just as batting practice is crucial to improving performance in baseball, we are hoping our “Pitching Machine” will raise our game to the next level, and enable us to deliver some game-changing treatments to patients with unmet medical needs.

 

Trash talking and creating a stem cell community

/ Kevin McCormack / Leave a comment

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Imilce Rodriguez-Fernandez likes to talk trash. No, really, she does. In her case it’s cellular trash, the kind that builds up in our cells and has to be removed to ensure the cells don’t become sick.

Imilce was one of several stem cell researchers who took part in a couple of public events over the weekend, on either side of San Francisco Bay, that served to span both a geographical and generational divide and create a common sense of community.

The first event was at the Buck Institute for Research on Aging in Marin County, near San Francisco. It was titled “Stem Cell Celebration” and that’s pretty much what it was. It featured some extraordinary young scientists from the Buck talking about the work they are doing in uncovering some of the connections between aging and chronic diseases, and coming up with solutions to stop or even reverse some of those changes.

One of those scientists was Imilce. She explained that just as it is important for people to get rid of their trash so they can have a clean, healthy home, so it is important for our cells to do the same. Cells that fail to get rid of their protein trash become sick, unhealthy and ultimately stop working.

Imilce is exploring the cellular janitorial services our bodies have developed to deal with trash, and trying to find ways to enhance them so they are more effective, particularly as we age and those janitorial services aren’t as efficient as they were in our youth.

Unlocking the secrets of premature aging

Chris Wiley, another postdoctoral researcher at the Buck, showed that some medications that are used to treat HIV may be life-saving on one level, preventing the onset of full-blown AIDS, but that those benefits come with a cost, namely premature aging. Chris said the impact of aging doesn’t just affect one cell or one part of the body, but ripples out affecting other cells and other parts of the body. By studying the impact those medications have on our bodies he’s hoping to find ways to maintain the benefits of those drugs, but get rid of the downside.

Creating a Community

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Across the Bay, the U.C. Berkeley Student Society for Stem Cell Research held it’s 4th annual conference and the theme was “Culturing a Stem Cell Community.”

The list of speakers was a Who’s Who of CIRM-funded scientists from U.C. Davis’ Jan Nolta and Paul Knoepfler, to U.C. Irvine’s Henry Klassen and U.C. Berkeley’s David Schaffer. The talks ranged from progress in fighting blindness, to how advances in stem cell gene editing are cause for celebration, and concern.

What struck me most about both meetings was the age divide. At the Buck those presenting were young scientists, millennials; the audience was considerably older, baby boomers. At UC Berkeley it was the reverse; the presenters were experienced scientists of the baby boom generation, and the audience were keen young students representing the next generation of scientists.

Bridging the divide

But regardless of the age differences there was a shared sense of involvement, a feeling that regardless of which side of the audience we are on we all have something in common, we are all part of the stem cell community.

All communities have a story, something that helps bind them together and gives them a sense of common purpose. For the stem cell community there is not one single story, there are many. But while those stories all start from a different place, they end up with a common theme; inspiration, determination and hope.

 

Using skin cells to repair damaged hearts

/ Kevin McCormack / Leave a comment
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Heart muscle  cells derived from skin cells

When someone has a heart attack, getting treatment quickly can mean the difference between life and death. Every minute delay in getting help means more heart cells die, and that can have profound consequences. One study found that heart attack patients who underwent surgery to re-open blocked arteries within 60 minutes of arriving in the emergency room had a six times greater survival rate than people who had to wait more than 90 minutes for the same treatment.

Clearly a quick intervention can be life-saving, which means an approach that uses a patient’s own stem cells to treat a heart attack won’t work. It simply takes too long to harvest the healthy heart cells, grow them in the lab, and re-inject them into the patient. By then the damage is done.

Now a new study shows that an off-the-shelf approach, using donor stem cells, might be the most effective way to go. Scientists at Shinshu University in Japan, used heart muscle stem cells from one monkey, to repair the damaged hearts of five other monkeys.

In the study, published in the journal Nature, the researchers took skin cells from a macaque monkey, turned those cells into induced pluripotent stem cells (iPSCs), and then turned those cells into cardiomyocytes or heart muscle cells. They then transplanted those cardiomyocytes into five other monkeys who had experienced an induced heart attack.

After 3 months the transplanted monkeys showed no signs of rejection and their hearts showed improved ability to contract, meaning they were pumping blood around the body more powerfully and efficiently than before they got the cardiomyocytes.

It’s an encouraging sign but it comes with a few caveats. One is that the monkeys used were all chosen to be as close a genetic match to the donor monkey as possible. This reduced the risk that the animals would reject the transplanted cells. But when it comes to treating people, it may not be feasible to have a wide selection of heart stem cell therapies on hand at every emergency room to make sure they are a good genetic match to the patient.

The second caveat is that all the transplanted monkeys experienced an increase in arrhythmias or irregular heartbeats. However, Yuji Shiba, one of the researchers, told the website ResearchGate that he didn’t think this was a serious issue:

“Ventricular arrhythmia was induced by the transplantation, typically within the first four weeks. However, this post-transplant arrhythmia seems to be transient and non-lethal. All five recipients of [the stem cells] survived without any abnormal behaviour for 12 weeks, even during the arrhythmia. So I think we can manage this side effect in clinic.”

Even with the caveats, this study demonstrates the potential for a donor-based stem cell therapy to treat heart attacks. This supports an approach already being tested by Capricor in a CIRM-funded clinical trial. In this trial the company is using donor cells, derived from heart stem cells, to treat patients who developed heart failure after a heart attack. In early studies the cells appear to reduce scar tissue on the heart, promote blood vessel growth and improve heart function.

The study from Japan shows the possibilities of using a ready-made stem cell approach to helping repair damage caused by a heart attacks. We’re hoping Capricor will take it from a possibility, and turn it into a reality.

If you would like to read some recent blog posts about Capricor go here and here.

Funding stem cell research targeting a rare and life-threatening disease in children

/ Kevin McCormack / Leave a comment
cystinosis

Photo courtesy Cystinosis Research Network

If you have never heard of cystinosis you should consider yourself fortunate. It’s a rare condition caused by an inherited genetic mutation. It hits early and it hits hard. Children with cystinosis are usually diagnosed before age 2 and are in end-stage kidney failure by the time they are 9. If that’s not bad enough they also experience damage to their eyes, liver, muscles, pancreas and brain.

The genetic mutation behind the condition results in an amino acid, cystine, accumulating at toxic levels in the body. There’s no cure. There is one approved treatment but it only delays progression of the disease, has some serious side effects of its own, and doesn’t prevent the need for a  kidney transplant.

Researchers at UC San Diego, led by Stephanie Cherqui, think they might have a better approach, one that could offer a single, life-long treatment for the problem. Yesterday the CIRM Board agreed and approved more than $5.2 million for Cherqui and her team to do the pre-clinical testing and work needed to get this potential treatment ready for a clinical trial.

Their goal is to take blood stem cells from people with cystinosis, genetically-modify them and return them to the patient, effectively delivering a healthy, functional gene to the body. The hope is that these genetically-modified blood stem cells will integrate with various body organs and not only replace diseased cells but also rescue them from the disease, making them healthy once again.

In a news release Randy Mills, CIRM’s President and CEO, said orphan diseases like cystinosis may not affect large numbers of people but are no less deserving of research in finding an effective therapy:

“Current treatments are expensive and limited. We want to push beyond and help find a life-long treatment, one that could prevent kidney failure and the need for kidney transplant. In this case, both the need and the science were compelling.”

The beauty of work like this is that, if successful, a one-time treatment could last a lifetime, eliminating or reducing kidney disease and the need for kidney transplantation. But it doesn’t stop there. The lessons learned through research like this might also apply to other inherited multi-organ degenerative disorders.

Asterias’ stem cell clinical trial shows encouraging results for spinal cord injury patients

/ Kevin McCormack / Leave a comment
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Jake Javier; Asterias spinal cord injury clinical trial participant

When researchers are carrying out a clinical trial they have two goals: first, show that it is safe (the old “do no harm” maxim) and second, show it works. One without the other doesn’t do anyone any good in the long run.

A few weeks ago Asterias Biotherapeutics showed that their CIRM-funded stem cell therapy for spinal cord injuries appeared to be safe. Now their data suggests it’s working. And that is a pretty exciting combination.

Asterias announced the news at the annual scientific meeting of the International Spinal Cord Society in Vienna, Austria. These results cover five people who got a transplant of 10 million cells. While the language is muted, the implications are very encouraging:

“While early in the study, with only 4 of the 5 patients in the cohort having reached 90 days after dosing, all patients have shown at least one motor level of improvement so far and the efficacy target of 2 of 5 patients in the cohort achieving two motor levels of improvement on at least one side of their body has already been achieved.”

What does that mean for the people treated? A lot. Remember these are people who qualified for this clinical trial because of an injury that left them pretty much paralyzed from the chest down. Seeing an improvement of two motor levels means they are regaining some use of their arms, hands and fingers, and that means they are regaining the ability to do things like feeding, dressing and bathing themselves. In effect, it is not only improving their quality of life but it is also giving them a chance to lead an independent life.

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Kris Boesen, Asterias clinical trial participant

One of those patients is Kris Boesen who regained the use of his arms and hands after becoming the first patient in this trial to get a transplant of 10 million cells. We blogged about Kris here

Asterias says of the 5 patients who got 10 million cells, 4 are now 90 days out from their transplant. Of those:

  • All four have improved one motor level on at least one side
  • 2 patients have improved two motor levels on one side
  • One has improved two motor levels on both sides

What’s also encouraging is that none of the people treated experienced any serious side effects or adverse events from the transplant or the temporary use of immunosuppressive drugs.

Steve Cartt, CEO of Asterias, was understandably happy with the news and that it allows them to move to the next phase:

“We are quite encouraged by this first look at efficacy results and look forward to reporting six-month efficacy data as planned in January 2017.  We have also just recently been cleared to begin enrolling a new cohort and administering to these new patients a much higher dose of 20 million cells.  We look forward to begin evaluating efficacy results in this higher-dose cohort in the coming months as well.”

People with spinal cord injuries can regain some function spontaneously so no one is yet leaping to the conclusion that all the progress in this trial is due to the stem cells. But to see all of the patients in the 10 million stem cell group do well is at the very least a positive sign. Now the hope is that these folks will continue to do well, and that the next group of people who get a 20 million cell transplant will also see improvements.

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Roman Reed, spinal cord injury patient advocate

While the team at Asterias were being cautiously optimistic, Roman Reed, whose foundation helped fund the early research that led to this clinical trial, was much less subdued in his response. He was positively giddy:

“If one patient only improves out of the five, it can be an outlier, but with everyone improving out of the five this is legit, this is real. Cures are happening!”