One year later, spinal cord therapy still looks promising

Jake Javier – participant in the SCIStar study

The beginning of a clinical trial, particularly the first time a new therapy is being tested in people, is often a time of equal parts anticipation and nervousness. Anticipation, because you have been working to this point for many years. Nervousness, because you have never tested this in people before and even though you have done years of study to show it is probably safe, until you try it in people you never really know.

That’s why the latest results from the CIRM-funded SCiStar Study, a clinical trial for spinal cord injury, are so encouraging. The results show that, one year after being treated, all the patients are doing well, none have experienced any serious side effects, and most have experienced impressive gains in movement, mobility and strength.

Ed Wirth, Chief Medical Officer at BioTime

In a news release Ed Wirth,  BioTIme’s Chief Medical Officer, said they were encouraged by what they saw:

“We believe the primary goals of the SCiStar Study, which were to observe the safety of OPC1 in cervical spinal cord injury patients as well as other important metrics including related to the optimal timing of OPC1 injection, tolerability of the immunosuppression regimen, engraftment of OPC1 cells, and rates of motor recovery observed among different study subpopulations, have all been successfully achieved.”

The study involved transplanting what the researchers called AST-OPC1 cells into patients who have suffered recent injuries that have left them paralyzed from the neck down.  AST-OPC1 are oligodendrocyte progenitor cells, which develop into cells that support and protect nerve cells in the central nervous system, the area damaged in spinal cord injury. It’s hoped the treatment will restore connections at the injury site, allowing patients to regain some movement and feeling.

Altogether 25 patients were involved. Three, in Cohort 1, were given injections of just two million OPC1 cells. This was to ensure the approach was safe and wouldn’t endanger patients. The remaining 22, in Cohorts 2-5, were given between 10 and 20 million cells. One year after the last patient was treated the results show:

  • MRI scans show no evidence of adverse changes in any of the 25 SCiStar study subjects.
    • No SCiStar study subjects had worsening of neurological function post-injection
    • At 12 months, 95% (21/22) of patients in Cohorts 2-5 recovered at least one motor level on at least one side and 32% (7/22) of these subjects recovered two or more motor levels on at least one side. 
    • No patient saw decreased motor function following administration of OPC1 and all either retained for 12 months the motor function recovery seen through 6 months or experienced further motor function recovery from 6 to 12 months.
    • All three subjects in Cohort 1 and 95% (21/22) of those in Cohorts 2 to 5 have MRI scans at 12 months consistent with the formation of a tissue matrix at the injury site. This is encouraging evidence the OPC1 cells have engrafted at the injury site and helped to prevent cavitation, a destructive process that occurs within the spinal cord following spinal cord injuries, and typically results in permanent loss of motor and sensory function.

“We appreciate the support of the California Institute for Regenerative Medicine, the world’s largest institution dedicated to bringing the future of cellular medicine closer to reality, whose generous grant funding to date of $14.3 million has helped advance the clinical development of our OPC1 program and generate these encouraging clinical results in patients with traumatic spinal cord injuries.”

BioTime is now planning to meet with the Food and Drug Administration (FDA) later this year to discuss next steps for the therapy. Soon as we know the outcome of those talks, we’ll share them with you.

Old cells need not apply: how a stem cell’s age can impact potential treatments

Getting older is a normal, at times existential, part of life. The outward changes are abundant and noticeable: thinning of the hair, greying of the hair, and added lines to the face. There are also changes that happen that are not so abundantly clear in terms of outward appearance: slowing of healing time for bone fractures and a gradual loss of bodily function. The process of aging poses one very fundamental question — Could understanding how stem cells age lead to a greater understanding of how diseases develop? More importantly, could it guide the approach towards developing potential treatments? Two different studies highlight the importance of evaluating and understanding the process of aging in stem cells.

The first study, led by Dr. Michael Fehlings, looked at the impact of donor age in relation to stem cell therapies for spinal cord injuries (SCI). Dr. Fehlings, with a team of investigators from the University of Toronto and Krembil Research Institute, University Health Network, used an adult rat model to look at how cells derived from young vs. old stem cells affected tissue regeneration and recovery after a spinal cord injury.

Some rats with a SCI received cells derived from stem cells in the umbilical cord blood, which are considered “young” stem cells. The other rats with a SCI received cells derived from stem cells in the bone marrow, which are considered “old” stem cells. The results showed, ten weeks after treatment, that rats given the “young” stem cells exhibited a better recovery in comparison to those given the “old” stem cells.

In a press release, Dr. Fehlings stated that,

“Together, this minimally invasive and effective approach to cell therapy has significant implications on the treatment of traumatic cervical SCI and other central nervous system injuries. These results can help to optimize cell treatment strategies for eventual use in humans.”

The full results to this study were published in Stem Cells Translational Medicine.

The second, separate study, conducted by Dr. Stephen Crocker at UConn Health, looks at brain stem cells in people with multiple sclerosis (MS), a neurodegenerative disease caused by the inflammation and destruction of the insulation around the nerves, also known as myelin. Problems with insulation around the nerves can prevent or complicate the electrical signals sent from the brain to the body, which can lead to problems with walking or other bodily movements.

Drawing of a healthy nerve cell with insulation (left) and one damaged by multiple sclerosis (right). Image courtesy of Shutterstock

Dr. Crocker and his team found that brain stem cells in patients with MS look much older when compared to the brain stem cells of a healthy person around the same age. Not only did these brain stem cells look older, but they also acted much older in comparison to their healthy counterparts. It was also discovered that the brain stem cells of MS patients were producing a protein that prevented the development of insulation around the nerves. What is more remarkable is that Dr. Crocker and his team demonstrated that when this protein is blocked, the insulation around the nerves develops normally again.

In a press release, Dr. Valentina Fossati, a neurologist at the New York Stem Cell Foundation who evaluated these brain stem cells, stated that,

“We are excited that the study of human stem cells in a dish led to the discovery of a new disease mechanism that could be targeted in much-needed therapeutics for progressive MS patients.”

The complete study was published in the Proceedings of the National Academy of Sciences (PNAS).

Rats, research and the road to new therapies

Don Reed

Don Reed has been a champion of CIRM even before there was a CIRM. He’s a pioneer in pushing for funding for stem cell research and now he’s working hard to raise awareness about the difference that funding is making.

In a recent article on Daily Kos, Don highlighted one of the less celebrated partners in this research, the humble rat.

A BETTER RAT? Benefit #62 of the California Stem Cell Agency

By Don C. Reed

When I told my wife Gloria I was writing an article about rats, she had several comments, including: “Oo, ugh!” and also “That’s disgusting!”

Obviously, there are problems with rats, such as when they chew through electrical wires, which may cause a short circuit and burn down the house. Also, they are blamed for carrying diseased fleas in their ears and spreading the Black Plague, which in 1340 killed half of China and one-third of Europe—but this is not certain. The plague may in fact have been transmitted by human-carried parasites.

But there are positive aspects to rats as well. For instance: “…a rat paired with  another that has a disability…will be very kind to the other rat. Usually, help is offered with food, cleaning, and general care.”—GUIDE TO THE RAT, by Ginger Cardinal.

Above all, anyone who has ever been sick owes a debt to rats, specifically the Norway rat with that spectacular name, rattus norvegicus domesticus, found in labs around the world.

I first realized its importance on March 1, 2002, when I held in my hand a rat which had been paralyzed, but then recovered the use of its limbs.

The rat’s name was Fighter, and she had been given a derivative of embryonic stem cells, which restored function to her limbs. (This was the famous stem cell therapy begun by Hans Keirstead with a Roman Reed grant, developed by Geron, and later by CIRM and Asterias, which later benefited humans.)

As I felt the tiny muscles struggling to be free, it was like touching tomorrow— while my paralyzed son, Roman Reed, sat in his wheelchair just a few feet away.

Was it different working with rats instead of mice? I had heard that the far smaller lab mice were more “bitey” than rats.  

Wanting to know more about the possibilities of a “better rat”, I went to the CIRM website, (www.cirm.ca.gov) hunted up the “Tools and Technology III” section, and the following complicated sentence::

“Embryonic stem cell- based generation of rat models for assessing human cellular therapies.”

Hmm. With science writing, it always takes me a couple of readings to know what they were talking about. But I recognized some of the words, so that was a start.

“Stemcells… rat models… human therapies….”  

I called up Dr. Qilong Ying, Principle Investigator (PI) of the study.

As he began to talk, I felt a “click” of recognition, as if, like pieces of a puzzle, facts were fitting together.

It reminded me of Jacques Cousteau, the great underwater explorer, when he tried to invent a way to breathe underwater. He had the compressed air tank, and a mouthpiece that would release air—but it came in a rush, not normal breathing.

So he visited his friend, race car mechanic Emil Gagnan, and told him, “I need something that will give me air, but only when I inhale,”– and Gagnan said: “Like that?” and pointed to a metal contraption on a nearby table.

It was something invented for cars. But by adding it to what Cousteau already had, the Cousteau-Gagnan SCUBA (Self Contained Underwater Breathing Apparatus) gear was born—and the ocean could now be explored.

Qi-Long Ying’s contribution to science may also be a piece of the puzzle of cure…

A long-term collaboration with Dr. Austin Smith centered on an attempt to do with rats what had done with mice.

In 2007, the  Nobel Prize in Medicine had been won by Dr. Martin Evans, Mario Capecchi, and Oliver Smithies. Working independently, they developed “knock-out” and “knock-in” mice, meaning to take out a gene, or put one in.  

But could they do the same with rats?

 “We and others worked very, very hard, and got nowhere,” said Dr. Evans.

Why was this important?

Many human diseases cannot be mimicked in the mouse—but might be in the rat. This is for several reasons: the rat is about ten times larger; its internal workings are closer to those of a human; and the rat is considered several million years closer (in evolutionary terms) to humans than the mouse.

In 2008 (“in China, that is the year of the rat,” noted Dr. Ying in our conversation) he received the first of three grants from CIRM.

“We proposed to use the classical embryonic stem cell-based gene-targeting technology to generate rat models mimicking human heart failure, diabetes and neurodegenerative diseases…”

How did he do?

In 2010, Science Magazine honored him with inclusion in their “Top 10 Breakthroughs for using embryonic stem cell-based gene targeting to produce the world’s first knockout rats, modified to lack one or more genes…”

And in 2016, he and Dr. Smith received the McEwen Award for Innovation,  the highest honor bestowed by the International Society for Stem Cell Research (ISSCR).

Using knowledge learned from the new (and more relevant to humans) lab rat, it may be possible to develop methods for the expansion of stem cells directly inside the patient’s own bone marrow. Stem cells derived in this fashion would be far less likely to be rejected by the patient.  To paraphrase Abraham Lincoln, they would be “of the patient, by the patient and for the patient—and shall not perish from the patient”—sorry!

Several of the rats generated in Ying’s lab (to mimic human diseases) were so successful that they have been donated to the Rat Research Resource center so that other scientists can use them for their study.

“Maybe in the future we will develop a cure for some diseases because of knowledge from using rat models,” said Ying. “I think it’s very possible. So we want more researchers from USC and beyond to come and use this technology.”

And it all began with the humble rat…

Using 3D printer to develop treatment for spinal cord injury

3d-printed-device

3D printed device

Spinal cord injuries (SCIs) affect approximately 300,000 Americans, with about 18,000 new cases occurring per year. One of these patients, Jake Javier, who we have written about many times over the past several years, received ten million stem cells as part of a CIRM-funded clinical trial and a video about his first year at Cal Poly depicts how these injuries can impact someone’s life.

Currently, there is nothing that completely reverses SCI damage and most treatment is aimed at rehabilitation and empowering patients to lead as normal a life as possible under the circumstances. Improved treatment options are necessary both to improve patients’ overall quality of life, and to reduce associated healthcare costs.

Scientists at UC San Diego’s School of Medicine and Institute of Engineering in Medicine have made critical progress in providing SCI patients with hope towards a more comprehensive and longer lasting treatment option.

shaochen chen

Prof. Shaochen Chen and his 3D printer

In a study partially funded by CIRM and published in Nature Medicine, Dr. Mark Tuszynski’s and Dr. Shaochen Chen’s groups used a novel 3D printing method to grow a spinal cord in the lab.

Previous studies have seen some success in lab grown neurons or nerve cells, improving SCI in animal models. This new study, however, is innovative both for the speed at which the neurons are printed, and the extent of the neuronal network that is produced.

To achieve this goal, the scientists used a biological scaffold that directs the growth of the neurons so they grow to the correct length and generate a complete neuronal network. Excitingly, their 3D printing technology was so efficient that they were able to grow implants for an animal model in 1.6 seconds, and a human-sized implant in just ten minutes, showing that their technology is scalable for injuries of different sizes.

When they tested the spinal cord implants in rats, they found that not only did the implant repair the damaged spinal cord tissue, but it also provided sustained improvement in motor function up to six months after implantation.

Just as importantly, they also observed that blood vessels had infiltrated the implanted tissue. The absence of vascularized tissue is one of the main reasons engineered implants do not last long in the host, because blood vessels are necessary to provide nutrients and support tissue growth. In this case, the animal’s body solved the problem on its own.

In a press release, one of the co-first authors of the paper, Dr. Kobi Koffler, states the importance and novelty of this work:

“This marks another key step toward conducting clinical trials to repair spinal cord injuries in people. The scaffolding provides a stable, physical structure that supports consistent engraftment and survival of neural stem cells. It seems to shield grafted stem cells from the often toxic, inflammatory environment of a spinal cord injury and helps guide axons through the lesion site completely.”

In order to make this technology viable for human clinical trials, the scientists are testing their technology in larger animal models before moving into humans, as well as investigating how to improve the longevity of the neuronal network by introducing proteins into the scaffolds.

 

 

The most popular Stem Cellar posts of 2018

The blog

You never know when you write something if people are going to read it. Sometimes you wonder if anyone is going to read it. So, it’s always fun, and educational, to look back at the end of the year and see which pieces got the most eyeballs.

It isn’t always the ones you think will draw the biggest audiences. Sometimes it is diseases that are considered “rare” (those affecting fewer than 200,000 people) that get the most attention.

Maybe it’s because those diseases have such a powerful online community which shares news, any news, about their condition of interest with everyone they know. Whatever the reason, we are always delighted to share encouraging news about research we are funding or encouraging research that someone else is funding.

That was certainly the case with the top two stories this year. Both were related to ALS or Lou Gehrig’s disease.  It’s a particularly nasty condition. People diagnosed with ALS have a life expectancy of just 2 to 5 years. So it’s probably not a big surprise that stories suggesting stem cells could expand that life span got a big reception.

Whatever the reason, we’re just happy to share hopeful news with everyone who comes to our blog.

And so, without further ado, here is the list of the most popular Stem Cellar Blog Posts for 2018.

All of us in the Communications team at CIRM consider it an honor and privilege to be able to work here and to meet many of the people behind these stories; the researchers and the patients and patient advocates. They are an extraordinary group of individuals who help remind us why we do this work and why it is important. We love our work and we hope you enjoy it too. We plan to be every bit as active and engaged in 2019.

71 for Proposition 71

Proposition 71 is the state ballot initiative that created California’s Stem Cell Agency. This month, the Agency reached another milestone when the 71st clinical trial was initiated in the CIRM Alpha Stem Cell Clinics (ASCC) Network. The ASCC Network deploys specialized teams of doctors, nurses and laboratory technicians to conduct stem cell clinical trials at leading California Medical Centers.

StateClinics_Image_CMYK

These teams work with academic and industry partners to support patient-centered for over 40 distinct diseases including:

  • Amyotrophic Lateral Sclerosis (ALS)
  • Brain Injury & Stroke
  • Cancer at Multiple Sites
  • Diabetes Type 1
  • Eye Disease / Blindness Heart Failure
  • HIV / AIDS
  • Kidney Failure
  • Severe Combined Immunodeficiency (SCID)
  • Sickle Cell Anemia
  • Spinal Cord Injury

These clinical trials have treated over 400 patients and counting. The Alpha Stem Cell Clinics are part of CIRM’s Strategic Infrastructure. The Strategic Infrastructure program which was developed to support the growth of stem cell / regenerative medicine in California. A comprehensive update of CIRM’s Infrastructure Program was provided to our Board, the ICOC.

CIRM’s infrastructure catalyzes stem cell / regenerative medicine by providing resources to all qualified researchers and organizations requiring specialized expertise. For example, the Alpha Clinics Network is supporting clinical trials from around the world.

Many of these trials are sponsored by commercial companies that have no CIRM funding. To date, the ASCC Network has over $27 million in contracts with outside sponsors. These contracts serve to leverage CIRMs investment and provide the Network’s medical centers with a diverse portfolio of clinical trials to address patients’’ unmet medical needs.

Alpha Clinics – Key Performance Metrics

  • 70+ Clinical Trials
  • 400+ Patients Treated
  • 40+ Disease Indications
  • Over $27 million in contracts with commercial sponsors

The CIRM Alpha Stem Cell Clinics and broader Infrastructure Programs are supporting stem cell research and regenerative medicine at every level, from laboratory research to product manufacturing to delivery to patients. This infrastructure has emerged to make California the world leader in regenerative medicine. It all started because California’s residents supported a ballot measure and today we have 71 clinical trials for 71.

 

 

Stem Cell Agency celebrates 50 clinical trials with patient #1

Yesterday the CIRM Board approved funding for our 50th clinical trial (you can read about that here) It was an historic moment for us and to celebrate we decided to go back in history and hear from the very first person to be treated in a CIRM-funded clinical trial. Rich Lajara was treated in the Geron clinical trial after experiencing a spinal cord injury, thus he became CIRM’s patient #1. It’s a badge he says he is honored to wear. This is the speech Rich made to our Board.

Rich Lajara

Hello and good afternoon everyone. It’s an honor to be here today as the 50th clinical trial has been officially funded by CIRM. It was feels like it was just yesterday that I was enrolled into the first funded clinical trial by CIRM and in turn became California’s’ 1st embryonic stem cell patient.

I became paralyzed from the waist down in September 2011. It was Labor Day and I was at a river with some close friends. There was this natural granite rock slide feature next to a waterfall, it was about 60 feet long; all you had to do was get a bucket of water to get the rocks wet and slide down into a natural pool. I ended up slipping and went down head first backwards but was too far over and I slid off a 15’ ledge where the waterfall was. I was pulled from the water and banged up pretty bad. Someone said “look at that deformity on his back” and tapped my leg and asked if I could feel that. I knew immediately I was paralyzed. I thought this was the end, little did I know this was just the beginning. I call it being in the wrong place at the right time.

So, after a few days in the hospital of course everyone, as well as myself, wanted a cure. We quickly learned one didn’t exist. A close family friend had been making phone calls and was able to connect with the Christopher & Dana Reeve Foundation and learned about a clinical trial with “stem cells”. One of my biggest question was how any people have done this? “Close to none”, I was told.

I was also told I most likely would have no direct benefit as this was a safety trial? So why do it at all? Obviously at that time I was willing to overlook the “most likely” part because I was willing to do anything to try and get my normal life back.

Looking back the big picture was laying the ground work for others like Kris or Jake (two people enrolled in a later version of this trial). At the time I had no clue that what I was doing would be such a big deal. The data collected from me would end up being priceless. It’s stories like Jake’s and Kris’ that make me proud and reinforce my decision to have participated in California’s first stem cell clinical trial funded by prop 71.

Like I said earlier it was just the beginning for me. A couple of years later I became a patient advocate working with Americans for Cures. I have been able to meet many people in the stem cell industry and love to see the glow in their face when they learn I was California’s first embryonic stem cell patient.

I can’t even fathom all the year’s of hard work and countless hours of research that had lead up to my long anticipated surgery, but when I see their glowing smile I know they knew what it took.

I also enjoy sharing my story and bridging the gap between myths and facts about stem cells, or talking to students and helping inspire the next generation that will be in the stem cell industry.  As a matter of fact, I have 13 year old sister, Maddie, dead set on being a neurosurgeon.

Fast forward to today. Life in a wheelchair is not exactly a roll in the park (no pun intended) but I have grown accustomed to the new normal. Aside from some neuropathic pain, life is back on track.

Not once did I feel sorry for myself, I was excited to be alive. Sure I have bad days but don’t we all.

In the last 14 years CIRM has funded 50 human clinical trials, published around 2750 new peer-reviewed scientific discoveries, and they’ve transformed California into the world leader in stem cell research. As I look around the posters on the wall, of the people whose lives have been transformed by the agency, I can’t help but be struck by just how much has been achieved in such a short period of time.

While my journey might not yet be over, Evie and 40 other children like her, (children born with SCID) will never remember what it was like to live with the horrible condition they were born with because they have been cured thanks to CIRM. There are hundreds of others whose lives have been transformed because of work the agency has funded.

CIRM has proven how much can be achieved if we invest in cutting-edge medical research.

As most of you here probably know CIRM’s funding from Proposition 71 is about to run out. If I had just one message I wanted people to leave with today it would be this. Everyone in this room knows how much CIRM has done in a little over a decade and how many lives have been changed because of its existence. We have the responsibility to make sure this work continues. We have a responsibility to make sure that the stories we’ve heard today are just the beginning.

I will do everything I can to make sure the agency gets refunded and I hope that all of you will join me in that fight. I’m excited for the world of stem cells, particularly in California, and can’t wait to see what’s on the horizon.

 

Has Regenerative Medicine Come of Age?

Signals logo

For the past few years the Signals blog site –  which offers an insiders’ perspectives on the world of regenerative medicine and stem cell research – has hosted what it calls a “Blog Carnival”. This is an event where bloggers from across the stem cell field are invited to submit a piece based on a common theme. This year’s theme is “Has Regenerative Medicine Come of Age?” Here’s my take on that question:

Many cultures have different traditions to mark when a child comes of age. A bar mitzvah is a Jewish custom marking a boy reaching his 13th birthday when he is considered accountable for his own actions. Among Latinos in the US a quinceañera is the name given to the coming-of-age celebration on a girl’s 15th birthday.

Regenerative Medicine (RM) doesn’t have anything quite so simple or obvious, and yet the signs are strong that if RM hasn’t quite come of age, it’s not far off.

For example, look at our experience at the California Institute for Regenerative Medicine (CIRM). When we were created by the voters of California in 2004 the world of stem cell research was still at a relatively immature phase. In fact, CIRM was created just six years after scientists first discovered a way to derive stem cells from human embryos and develop those cells in the laboratory. No surprise then that in the first few years of our existence we devoted a lot of funding to building world class research facilities and investing in basic research, to gain a deeper understanding of stem cells, what they could do and how we could use them to develop therapies.

Fast forward 14 years and we now have funded 49 projects in clinical trials – everything from stroke and cancer to spinal cord injury and HIV/AIDS – and our early funding also helped another 11 projects get into clinical trials. Clearly the field has advanced dramatically.

In addition the FDA last year approved the first two CAR-T therapies – Kymriah and Yescarta – another indication that progress is being made at many levels.

But there is still a lot of work to do. Many of the trials we are funding at the Stem Cell Agency are either Phase 1 or 2 trials. We have only a few Phase 3 trials on our books, a pattern reflected in the wider RM field. For some projects the results are very encouraging – Dr. Gary Steinberg’s work at Stanford treating people recovering from a stroke is tremendously promising. For others, the results are disappointing. We have cancelled some projects because it was clear they were not going to meet their goals. That is to be expected. These clinical trials are experiments that are testing, often for the first time ever in people, a whole new way of treating disease. Failure comes with the territory.

As the number of projects moving out of the lab and into clinical trials increases so too are the other signs of progress in RM. We recently held a workshop bringing together researchers and regulators from all over the world to explore the biggest problems in manufacturing, including how you go from making a small batch of stem cells for a few patients in an early phase clinical trial to mass producing them for thousands, if not millions of patients. We are also working with the National Institutes of Health and other stakeholders in discussing the idea of reimbursement, figuring out who pays for these therapies so they are available to the patients who need them.

And as the field advances so too do the issues we have to deal with. The discovery of the gene-editing tool CRISPR has opened up all sorts of possible new ways of developing treatments for deadly diseases. But it has also opened up a Pandora’s box of ethical issues that the field as a whole is working hard to respond to.

These are clear signs of a maturing field. Five years ago, we dreamed of having these kinds of conversations. Now they are a regular feature of any RM conference.

The simple fact that we can pose a question asking if RM has come of age is a sign all by itself that we are on the way.

Like little kids sitting in the back of a car, anxious to get to their destination, we are asking “Are we there yet?” And as every parent in the front seat of their car responds, “Not yet. But soon.”

Stem cell treatment for spinal cord injury offers improved chance of independent life for patients

kris-boesen

Kris Boesen, CIRM spinal cord injury clinical trial patient works to strengthen his upper body. (Photo/Greg Iger)

A spinal cord injury is devastating, changing a person’s life in a heartbeat. In the past there was little that doctors could do other than offer pain relief and physical therapy to try and regain as much muscle function as possible. That’s why the latest results from the CIRM-supported Asterias Biotherapeutics spinal cord injury trial are so encouraging.

Asterias is transplanting what they call AST-OPC1 cells into patients who have suffered injuries that left them paralyzed from the neck down.  AST-OPC1 are oligodendrocyte progenitor cells, which develop into cells that support and protect nerve cells in the central nervous system, the area damaged in spinal cord injury. It’s hoped the treatment will restore connections at the injury site, allowing patients to regain some movement and feeling.

The latest results seem to suggest they are doing just that.

In a news release, Asterias reports that of the 25 patients treated in this clinical trial none have experienced serious side effects. They also reported that magnetic resonance imaging (MRI) tests show that more than 95 percent of the patients have shown evidence of what’s called “tissue matrix” at the injury site. This is encouraging because it suggests the implanted cells are engrafting and helping prevent a cavitation, a serious process that often occurs in spinal cord injuries and can lead to permanent loss of muscle and sensory function plus chronic pain.

The study also shows that after six months:

  • 100 percent of the patients in Group 5 (who received 20 million cells) have recovered at least one motor level (for example increased ability to use their arms) on at least one side
  • Two patients in Group 5 recovered one motor level on both sides
  • Altogether four of the 25 patients have recovered two or more motor levels on at least one side.

Not surprisingly Ed Wirth, the Chief Medical Officer at Asterias, was pleased with the results:

“The results from the study remain encouraging as the six-month follow-up data continued to demonstrate a positive safety profile and show that the AST-OPC1 cells are successfully engrafting in patients.”

While none of the patients are able to walk, just regaining some use of their arms or hands can have a hugely important impact on their quality of life and their ability to lead an independent life. And, because lifetime costs of taking care of someone who is paralyzed from the neck or chest down can run as high as $5 million, anything that increases a patient’s independence can have a big impact on those costs.

The impact of this research is helping change the lives of the patients who received it. One of those patients is Jake Javier. We have blogged about Jake several times over the last two years and recently showed this video about his first year at Cal Poly and how Jake is turning what could have been a life-ending event into a life-affirming one.

 

Stem Cell Roundup: Jake Javier’s amazing spirit; TV report highlights clinic offering unproven stem cell therapies

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Jake Javier: Photo Michael Clemens, Sees the Day

In the Roundup we usually focus on studies that highlight advances in stem cell research but today we’re going to do something a little different. Instead of relying on print for our stories, we’re turning to video.

We begin with a piece about Jake Javier. Regular readers of our blog will remember that Jake is the young man who broke his neck the day before he graduated high school, leaving him paralyzed from the upper chest down.

After enrolling in the CIRM-funded Asterias clinical trial, and receiving a transplant of 10 million stem cells, Jake regained enough use of his arms and hands to be able to go to Cal Poly and start his life over.

This video highlights the struggles and challenges he faced in his first year, and his extraordinary spirit in overcoming them.

(thanks to Matt Yoon and his Creative Services team at Cal Poly for this video)

Going Undercover

The second video is from the NBC7 TV station in San Diego and highlights one of the big problems in regenerative medicine today, clinics offering unproven therapies. The investigative team at NBC7 went undercover at a stem cell clinic seminar where presenters talked about “the most significant breakthrough in natural medicine” for improving mobility and reducing pain. As the reporter discovered, the reality didn’t live up to the promise.

NBC7 Investigative Report