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.

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…

Stories that caught our eye: National Geographic takes a deep dive into iPS cells; Japanese researchers start iPS cell clinical trial for spinal cord injury; and do high fat diets increase your risk of colorectal cancer

Can cell therapy beat the most difficult diseases?

That’s the question posed in a headline in National Geographic. The answer; maybe, but it is going to take time and money.

The article focuses on the use of iPS cells, the man-made equivalent of embryonic stem cells that can be turned into any kind of cell or tissue in the body. The reporter interviews Kemal Malik, the member of the Board of Management for pharmaceutical giant Bayer who is responsible for innovation. When it comes to iPS cells, it’s clear Malik is a true believer in their potential.

“Because every cell in our bodies can be produced from a stem cell, the applicability of cell therapy is vast. iPSC technology has the potential to tackle some of the most challenging diseases on the planet.”

But he also acknowledges that the field faces some daunting challenges, including:

  • How to manufacture the cells on a large scale without sacrificing quality and purity
  • How do you create products that have a stable shelf life and can be stored until needed?
  • How do you handle immune reactions if you are giving these cells to patients?

Nonetheless, Malik remains confident we can overcome those challenges and realize the full potential of these cells.

“I believe human beings are on the cusp of the next big wave of pharmaceutical innovation. The use of living cells to make people better.”

As if to prove Malik right there was also news this week that researchers at Japan’s Keio University have been given permission to start a clinical trial using iPS cells to treat people with spinal cord injuries. This would be the first of its kind anywhere in the world.

Japan launches iPSC clinical trial for spinal cord injury

An article in Biospace says that the researchers plan to treat four patients who have suffered varying degrees of paralysis due to a spinal cord injury.  They will take cells from the patients and, using the iPS method, turn them into the kind of nerve cells found in the spinal cord, and then transplant two million of them back into the patient. The hope is that this will create new connections that restore movement and feeling in the individuals.

This trial is expected to start sometime this summer.

CIRM has already funded a first-of-its-kind clinical trial for spinal cord injury with Asterias Biotherapeutics. That clinical trial used embryonic stem cells turned into oligodendrocyte progenitor cells – which develop into cells that support and protect nerve cells in the central nervous system. We blogged about the encouraging results from that trial here.

High fat diet drives colorectal cancer

Finally today, researchers at Salk have uncovered a possible cause to the rise in colorectal cancer deaths among people under the age of 55; eating too much high fat food.

Our digestive system works hard to break down the foods we eat and one way it does that is by using bile acids. Those acids don’t just break down the food, however, they also break down the lining of our intestines. Fortunately, our gut has a steady supply of stem cells that can repair and replace that lining. Unfortunately, at least according to the team from Salk, mutations in these stem cells can lead to colorectal cancer.

The study, published in the journal Cell, shows that bile acids affect a protein called FXR that is responsible for ensuring that gut stem cells produce a steady supply of new lining for the gut wall. When someone eats a high fat diet it upsets the balance of bile acids, starting a cascade of events that help cancer develop and grow.

In a news release Annette Atkins, a co-author of the study, says there is a strong connection between bile acid and cancer growth:

“We knew that high-fat diets and bile acids were both risk factors for cancer, but we weren’t expecting to find they were both affecting FXR in intestinal stem cells.”

So next time you are thinking about having that double bacon cheese burger for lunch, you might go for the salad instead. Your gut will thank you. And it might just save your life.

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.

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.

 

Stories that caught our eye: Is a Texas law opening up access to stem cell treatments working? Another CIRM-funded company gets good news from the FDA.

TexasCapitol_shutterstock_494317324

Texas Capitol. (Shutterstock)

In 2017 Texas passed a sweeping new law, HB 810, which allowed medical clinics to provide “investigational stem cell treatments to patients with certain severe chronic diseases or terminal illnesses.” Those in favor of the law argued that patients battling life-threatening or life-changing diseases should have the right to try stem cell therapies that were involved in a clinical trial.

Now a new study, published in the journal Stem Cells and Development, looks at the impact of the law. The report says that despite some recent amendments t there are still some concerns about the law including:

  • It allows treatment only if the patient has a “severe, chronic” illness but doesn’t define what that means
  • It doesn’t have clearly defined procedures on tracking and reporting procedures so it’s hard to know how many patients might be treated and what the outcomes are
  • There is no Food and Drug Administration (FDA) oversight of the patients being treated
  • Because the treatments are unproven there are fears this will “open up the state to unsavory and predatory practices by individuals preying on vulnerable patients”

The researchers conclude:

“While HB 810 opens up access to patients, it also increases significant risks for their safety and financial cost for something that might have no positive impact on their disease. Truly understanding the impact of stem cell based interventions (SCBI) requires scientific rigor, and accurate outcome data reporting must be pursued to ensure the safety and efficacy behind such procedures. This information must be readily available so that patients can make informed decisions before electing to pursue such treatments. The creation of the SCBI registry could allow for some level of scientific rigor, provide a centralized data source, and offer the potential for better informed patient choices, and might be the best option for the state to help protect patients.”

Another CIRM-funded company gets RMAT designation

Poseida

When Congress approved the 21st Century Cures Act a few years ago one of the new programs it created was the Regenerative Medicine Advanced Therapy (RMAT) designation. This was given to therapies that are designed to treat a serious or life-threatening condition, where early clinical stage trials show the approach is safe and appears to be effective.

Getting an RMAT designation is a big deal. It means the company or researchers are able to apply for an expedited review by the FDA and could get approval for wider use.

This week Poseida Therapeutics was granted RMAT designation by the Food and drug Administration (FDA) for P-BCMA-101, its CAR-T therapy for relapsed/refractory multiple myeloma. This is currently in a Phase 1 clinical trial that CIRM is funding

In this trial Poseida’s technology takes an immunotherapy approach that uses the patient’s own engineered immune system T cells to seek and destroy cancerous myeloma cells.

In a news release Eric Ostertag, Poseida’s CEO, welcomed the news:

“Initial Phase 1 data presented at the CAR-TCR Summit earlier this year included encouraging response rates and safety data, including meaningful responses in a heavily pretreated population. We expect to have an additional data update by the end of the year and look forward to working closely with the FDA to expedite development of P-BCMA-101.”

This means that five CIRM-funded companies have now been granted RMAT designations:

California’s Stem Cell Agency Accelerates Treatments to Patients

The following article is an Op Ed that appeared in today’s print version of the San Francisco Chronicle

SanFranChronicle_Web

Biotechnology was born in California in the 1970s based on the discovery out of one of its universities and California is responsible for an industry that has impacted the lives of billions of people worldwide. In 2004, the voters of California approved Proposition 71, creating the California Institute for Regenerative Medicine and setting the state on the path to becoming a global leader in stem cell research. Today the therapies resulting from the institute’s work are not just changing lives, they are already saving lives.

Lives like Evie Vaccaro, who is alive today because of a treatment CIRM is funding. Vaccaro was born with SCID, also known as “bubble baby disease,” an immune disorder that often kills babies in their first two years. Vaccaro and dozens of other babies were given stem cell treatments thanks to the institute. All are showing improvement; some are now several years past treatment and considered cured.

An accident left Jake Javier from Danville paralyzed from the chest down on the eve of his high school graduation. Javier was treated in a CIRM-funded clinical trial. Today he has regained the use of his arms and hands, is driving a car and is a sophomore at Cal Poly San Luis Obispo. Five other patients treated at the same time as Javier have all experienced improvements meaning that instead of needing round-the-clock care, they can lead independent lives.

A study by the Tufts Center for the Study of Drug Development estimated it takes at least 10 years and $2.6 billion to develop one successful drug. In 14 years, and with just $3 billion, CIRM has funded 1,000 different projects, enrolled 900 patients, and supported 49 different clinical trials targeting diseases such as cancer, kidney failure and leukemia. Four of these programs have received an expedited designation by the U.S. Food and Drug Administration, meaning they could get faster approval to help more patients

We have created a network of world class medical clinics that have expertise in delivering treatments to patients. The CIRM Alpha Clinics offer treatments based on solid science, unlike the unlicensed clinics sprouting up around California that peddle unproven and potentially harmful therapies that cost patients thousands of dollars.

CIRM has:

  • Supported the creation of 12 stem-cell research facilities in California
  • Attracted hundreds of top-tier researchers to California
  • Trained a new generation of stem-cell scientists
  • Brought clinical trials to California — for example, one targeting ALS or Lou Gehrig’s disease
  • Deployed rigorous scientific standards and support so our programs have a “seal of approval” to attract $2.7 billion in additional investments from industry and other sources.

We recently have partnered with the National Institutes of Health to break down barriers and speed up the approval process to bring curative treatments to patients with Sickle Cell Disease.

Have we achieved all we wanted to? Of course not. The first decade of CIRM’s life was laying the groundwork, developing the knowledge and expertise and refining processes so that we can truly accelerate progress. As a leader in this burgeoning field of regenerative medicine, CIRM needs to continue its mission of accelerating stem-cell treatments to patients with unmet medical needs.

Dr. Maria T. Millan is President and CEO and Jonathan Thomas, JD, PhD, is the Board Chairman of the California Institute of Regenerative Medicine. 

 

 

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.

 

The story behind the book about the Stem Cell Agency

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Don Reed at his book launch: Photo by Todd Dubnicoff

WHY I WROTE “CALIFORNIA CURES”  By Don C. Reed

It was Wednesday, June 13th, 2018, the launch day for my new book, “CALIFORNIA CURES: How the California Stem Cell Research Program is Fighting Your Incurable Disease!”

As I stood in front of the audience of scientists, CIRM staff members, patient advocates, I thought to myself, “these are the kind of people who built the California stem cell program.” Wheelchair warriors Karen Miner and Susan Rotchy, sitting in the front row, typified the determination and resolve typical of those who fought to get the program off the ground. Now I was about to ask them to do it one more time.

My first book about CIRM was “STEM CELL BATTLES: Proposition 71 and Beyond. It told the story of  how we got started: the initial struggles—and a hopeful look into the future.

Imagine being in a boat on the open sea and there was a patch of green on the horizon. You could be reasonably certain those were the tops of coconut trees, and that there was an island attached—but all you could see was a patch of green.

Today we can see the island. We are not on shore yet, but it is real.

“CALIFORNIA CURES” shows what is real and achieved: the progress the scientists have made– and why we absolutely must continue.

For instance, in the third row were three little girls, their parents and grandparents.

One of them was Evangelina “Evie” Vaccaro, age 5. She was alive today because of CIRM, who had funded the research and the doctor who saved her.

Don Reed and Evie and Alysia

Don Reed, Alysia Vaccaro and daughter Evie: Photo by Yimy Villa

Evie was born with Severe Combined Immunodeficiency (SCID) commonly called the “bubble baby” disease. It meant she could never go outside because her immune system could not protect her.  Her mom and dad had to wear hospital masks to get near her, even just to give her a hug.

But Dr. Donald Kohn of UCLA operated on the tiny girl, taking out some of her bone marrow, repairing the genetic defect that caused SCID, then putting the bone marrow back.

Today, “Evie” glowed with health, and was cheerfully oblivious to the fuss she raised.

I was actually a little intimidated by her, this tiny girl who so embodied the hopes and dreams of millions. What a delight to hear her mother Alysia speak, explaining  how she helped Evie understand her situation:  she had “unicorn blood” which could help other little children feel better too.

This was CIRM in action, fighting to save lives and ease suffering.

If people really knew what is happening at CIRM, they would absolutely have to support it. That’s why I write, to get the message out in bite-size chunks.

You might know the federal statistics—133 million children, women and men with one or more chronic diseases—at a cost of $2.9 trillion dollars last year.

But not enough people know California’s battle to defeat those diseases.

DonReed_BookSigning2018-22

Adrienne Shapiro at the book launch: Photo by Todd Dubnicoff

Champion patient advocate Adrienne Shapiro was with us, sharing a little of the stress a parent feels if her child has sickle cell anemia, and the science which gives us hope:  the CIRM-funded doctor who cured Evie is working on sickle cell now.

Because of CIRM, newly paralyzed people now have a realistic chance to recover function: a stem cell therapy begun long ago (pride compels me to mention it was started by the Roman Reed Spinal Cord Injury Research Act, named after my son), is using stem cells to re-insulate damaged nerves in the spine.  Six people were recently given the stem cell treatment pioneered by Hans Keirstead, (currently running for Congress!)  and all six experienced some level of recovery, in a few cases regaining some use of their arms hands.

Are you old enough to remember the late Annette Funicello and Richard Pryor?  These great entertainers were stricken by multiple sclerosis, a slow paralysis.  A cure did not come in time for them. But the international cooperation between California’s Craig Wallace and Australia’s Claude Bernard may help others: by  re-insulating MS-damaged nerves like what was done with spinal cord injury.

My brother David shattered his leg in a motorcycle accident. He endured multiple operations, had steel rods and plates inserted into his leg. Tomorrow’s accident recovery may be easier.  At Cedars-Sinai, Drs. Dan Gazit and Hyun Bae are working to use stem cells to regrow the needed bone.

My wife suffers arthritis in her knees. Her pain is so great she tries to make only one trip a day down and up the stairs of our home.  The cushion of cartilage in her knees is worn out, so it is bone on bone—but what if that living cushion could be restored? Dr. Denis Evseenko of UCLA is attempting just that.

As I spoke, on the wall behind me was a picture of a beautiful woman, Rosie Barrero, who had been left blind by retinitis pigmentosa. Rosie lost her sight when her twin children were born—and regained it when they were teenagers—seeing them for the first time, thanks to Dr. Henry Klassen, another scientist funded by CIRM.

What about cancer? That miserable condition has killed several of my family, and I was recently diagnosed with prostate cancer myself. I had everything available– surgery, radiation, hormone shots which felt like harpoons—hopefully I am fine, but who knows for sure?

Irv Weissman, the friendly bear genius of Stanford, may have the answer to cancer.  He recognized there were cancer stem cells involved. Nobody believed him for a while, but it is now increasingly accepted that these cancer stem cells have a coating of protein which makes them invisible to the body’s defenses. The Weissman procedure may peel off that “cloak of invisibility” so the immune system can find and kill them all—and thereby cure their owner.

What will happen when CIRM’s funding runs out next year?

If we do nothing, the greatest source of stem cell research funding will be gone. We need to renew CIRM. Patients all around the world are depending on us.

The California stem cell program was begun and led by Robert N. “Bob” Klein. He not only led the campaign, was its chief writer and number one donor, but he was also the first Chair of the Board, serving without pay for the first six years. It was an incredible burden; he worked beyond exhaustion routinely.

Would he be willing to try it again, this time to renew the funding of a successful program? When I asked him, he said:

“If California polls support the continuing efforts of CIRM—then I am fully committed to a 2020 initiative to renew the California Institute for Regenerative Medicine (CIRM).”

Shakespeare said it best in his famous “to be or not to be” speech, asking if it is “nobler …to endure the slings and arrows of outrageous fortune, or to take arms against a sea of troubles—and by opposing, end them”.

Should we passively endure chronic disease and disability—or fight for cures?

California’s answer was the stem cell program CIRM—and continuing CIRM is the reason I wrote this book.

Don C. Reed is the author of “CALIFORNIA CURES: How the California Stem Cell Program is Fighting Your Incurable Disease!”, from World Scientific Publishing, Inc., publisher of the late Professor Stephen Hawking.

For more information, visit the author’s website: www.stemcellbattles.com