10 Years/10 Therapies: 10 Years after its Founding CIRM will have 10 Therapies Approved for Clinical Trials

In 2004, when 59 percent of California voters approved the creation of CIRM, our state embarked on an unprecedented experiment: providing concentrated funding to a new, promising area of research. The goal: accelerate the process of getting therapies to patients, especially those with unmet medical needs.

Having 10 potential treatments expected to be approved for clinical trials by the end of this year is no small feat. Indeed, it is viewed by many in the industry as a clear acceleration of the normal pace of discovery. Here are our first 10 treatments to be approved for testing in patients.

HIV/AIDS. The company Calimmune is genetically modifying patients’ own blood-forming stem cells so that they can produce immune cells—the ones normally destroyed by the virus—that cannot be infected by the virus. It is hoped this will allow the patients to clear their systems of the virus, effectively curing the disease.

Spinal cord injury patient advocate Katie Sharify is optimistic about the latest clinical trial led by Asterias Biotherapeutics.

Spinal cord injury patient advocate Katie Sharify is optimistic about the clinical trial led by Asterias Biotherapeutics.

Spinal Cord Injury. The company Asterias Biotherapeutics uses cells derived from embryonic stem cells to heal the spinal cord at the site of injury. They mature the stem cells into cells called oligodendrocyte precursor cells that are injected at the site of injury where it is hoped they can repair the insulating layer, called myelin, that normally protects the nerves in the spinal cord.

Heart Disease. The company Capricor is using donor cells derived from heart stem cells to treat patients developing heart failure after a heart attack. In early studies the cells appear to reduce scar tissue, promote blood vessel growth and improve heart function.

Solid Tumors. A team at the University of California, Los Angeles, has developed a drug that seeks out and destroys cancer stem cells, which are considered by many to be the reason cancers resist treatment and recur. It is believed that eliminating the cancer stem cells may lead to long-term cures.

Leukemia. A team at the University of California, San Diego, is using a protein called an antibody to target cancer stem cells. The antibody senses and attaches to a protein on the surface of cancer stem cells. That disables the protein, which slows the growth of the leukemia and makes it more vulnerable to other anti-cancer drugs.

Sickle Cell Anemia. A team at the University of California, Los Angeles, is genetically modifying a patient’s own blood stem cells so they will produce a correct version of hemoglobin, the oxygen carrying protein that is mutated in these patients, which causes an abnormal sickle-like shape to the red blood cells. These misshapen cells lead to dangerous blood clots and debilitating pain The genetically modified stem cells will be given back to the patient to create a new sickle cell-free blood supply.

Solid Tumors. A team at Stanford University is using a molecule known as an antibody to target cancer stem cells. This antibody can recognize a protein the cancer stem cells carry on their cell surface. The cancer cells use that protein to evade the component of our immune system that routinely destroys tumors. By disabling this protein the team hopes to empower the body’s own immune system to attack and destroy the cancer stem cells.

Diabetes. The company Viacyte is growing cells in a permeable pouch that when implanted under the skin can sense blood sugar and produce the levels of insulin needed to eliminate the symptoms of diabetes. They start with embryonic stem cells, mature them part way to becoming pancreas tissues and insert them into the permeable pouch. When transplanted in the patient, the cells fully develop into the cells needed for proper metabolism of sugar and restore it to a healthy level.

HIV/AIDS. A team at The City of Hope is genetically modifying patients’ own blood-forming stem cells so that they can produce immune cells—the ones normally destroyed by the virus—that cannot be infected by the virus. It is hoped this will allow the patients to clear their systems of the virus, effectively curing the disease

Blindness. A team at the University of Southern California is using cells derived from embryonic stem cell and a scaffold to replace cells damaged in Age-related Macular Degeneration (AMD), the leading cause of blindness in the elderly. The therapy starts with embryonic stem cells that have been matured into a type of cell lost in AMD and places them on a single layer synthetic scaffold. This sheet of cells is inserted surgically into the back of the eye to replace the damaged cells that are needed to maintain healthy photoreceptors in the retina.

UCLA team cures infants of often-fatal “bubble baby” disease by inserting gene in their stem cells; sickle cell disease is next target

Poopy diapers, ear-splitting cries, and sleepless nights: sure, the first few weeks of parenthood are grueling but those other moments of cuddling and kissing your little baby are pure bliss.

The bubble boy.  Born in 1971 with SCID, David Vetter lived in a sterile bubble to avoid outside germs that could kill him. He died in 1984 at 12 due to complications from a bone marrow transplant. [Credit: Baylor College of Medicine Archives]

The bubble boy. Born in 1971 with SCID, David Vetter lived in a sterile bubble to avoid outside germs that could kill him. He died in 1984 at 12 due to complications from a bone marrow transplant. [Credit: Baylor College of Medicine Archives]

That wasn’t the case for Alysia and Christian Padilla-Vacarro of Corona, California. Close contact with their infant daughter Evangelina, born in 2012, was off limits. She was diagnosed with a genetic disease that left her with no immune system and no ability to fight off infections so even a minor cold could kill her.

Evangelina was born with Severe Combined Immunodeficiency (SCID) also called “bubble baby” disease, a term coined in the 1970s when the only way to manage the disease was isolating the child in a super clean environment to avoid exposure to germs. Bone marrow transplants from a matched sibling offer a cure but many kids don’t have a match, which makes a transplant very risky. Sadly, many SCID infants die within the first year of life.

Until now, that is.

Today, a UCLA research team led by Donald Kohn, M.D., announced a stunning breakthrough cure that saved Evangelina’s life and all 18 children who have so far participated in the clinical trial. Kohn—the director of UCLA’s Human Gene Medicine Program—described the treatment strategy in a video interview with CIRM (watch the video below):

“We collect some of the baby’s own bone marrow, isolate the [blood] stem cells, add the gene that they’re missing that their immune system needs and then transplant the cells back to them. “

Inserting the missing gene, called ADA, into the blood stem cells restores the cells’ ability to produce a healthy immune system. And since the cells originally came from the infant, there’s no worry about the possible life-threatening complications from receiving non-matched donor cells.

This breakthrough didn’t occur overnight. Kohn and colleagues have been plugging away for over twenty years carrying out trials, observing their limitations and going back to lab to improve the technology. Their dedication has paid off. As Kohn states in a press release:

“All of the children with SCID that I have treated in these stem cell clinical trials would have died in a year or less without this gene therapy, instead they are all thriving with fully functioning immune systems.”

Alysia Padilla-Vacarro and daughter Evangelina on the day of her gene therapy treatment. Evangelina, now two years old, has had her immune system restored and lives a healthy and normal life. [Credit: UCLA Broad Center of Regenerative Medicine and Stem Cell Research.]

Alysia Padilla-Vacarro and daughter Evangelina on the day of her gene therapy treatment. Evangelina, now two years old, has had her immune system restored and lives a healthy and normal life. [Credit: UCLA Broad Center of Regenerative Medicine and Stem Cell Research.]

For the Padilla-Vacarro family, the dark days after Evangelina’s grave diagnosis have given way to a bright future. Alysia, Evangelina’s mom, poignantly recalled her daughter’s initial recovery:

”It was only around six weeks after the procedure when Dr. Kohn told us Evangelina can finally be taken outside. To finally kiss your child on the lips, to hold her, it’s impossible to describe what a gift that is. I gave birth to my daughter, but Dr. Kohn gave my baby life.”

The team’s next step is to get approval by the Food and Drug Administration (FDA) to provide this treatment to all SCID infants missing the ADA gene.

At the same time, Kohn and colleagues are adapting this treatment approach to cure sickle cell disease, a genetic disease that leads to sickle shaped red blood cells. These misshapen cells are prone to clumping causing debilitating pain, risk of stroke, organ damage and a shortened life span. CIRM is providing over $13 million in funding to support the UCLA team’s clinical trial set to start early next year.

For more information about CIRM-funded sickle cell disease research, visit our fact sheet.

Creating a Genetic Model for Autism, with a Little Help from the Tooth Fairy

One of the most complex aspects of autism is that it is not one disease—but many. Known more accurately as the autism spectrum disorder, or ASD, experts have long been trying to tease apart the various ways in which the condition manifests in children, with limited success.

But now, using the latest stem cell technology, scientists at the University of California, San Diego (UCSD) have identified a gene associated with Rett Syndrome—a rare form of autism almost exclusively seen in girls. And in so doing, the team has made the startling discovery that the many types of autism may be linked by common molecular pathways.

The research team, led by UCSD Professor and CIRM grantee Alysson Muotri, explained in a news release how induced pluripotent stem cell, or iPS cell, technology was used to pinpoint a gene associated with Rett Syndrome:

“One can take advantage of genomics to map all mutant genes in the patient and then use their own iPS cells to measure the impact of mutations in relevant cell types. Moreover, the study of brain cells derived from these iPS cells can reveal potential therapeutic drugs tailored to the individual. It is the rise of personalized medicine for mental and neurological disorder.”

iPS cell technology—a process by which scientists transform adult skin cells back into embryonic-like stem cells, after which they can be coaxed into maturing into virtually any type of cell—is a promising way to model diseases at the cellular level. But in order to truly understand what is happening in the brains of people with autism, Muotri and his team needed more samples from autistic individuals—on the order of hundreds or even thousands.

The Tooth Fairy Project allows scientists to gather large quantities of cells from autistic individuals for genomic analysis—simply asking parents to send in a discarded baby tooth.

The Tooth Fairy Project allows scientists to gather large quantities of cells from autistic individuals for genomic analysis—simply by asking parents to send in a discarded baby tooth.

Luckily, Muotri had a little help from the Tooth Fairy.

Or, more accurately, the Tooth Fairy Project, in which parents register for a “Fairy Tooth Kit” that lets them send a discarded baby tooth of their autistic child to researchers. Housed within each baby tooth are cells that can be transformed—with iPS cell technology—into neurons, thus giving the researchers a massive sample size with which to study.

Interestingly, the findings presented here come from the very first tooth to be sent to Muotri. Specifically, the team identified a mutation in the gene TRPC6 was present in children with autism. Additional experiments in animal models revealed that the TRPC6 mutation was indeed associated with abnormal brain cell development and function.

And for their next trick, the team found a way to reverse the mutation’s damaging effects.

By treating the cells with the chemical hyperforin, they were able to restore some normal function to the neurons—offering up a potential therapeutic strategy for treating ASD patients who harbor the TRPC6 mutation.

Drilling down even further, the team found that mutations in another gene called MeCP2, which causes Rett Syndrome, also set off a genetic domino effect that alters the normal function of the TRPC6 gene. Thus connecting this syndrome with other, non-syndromic types of autism.

“Taken together, these findings suggest that TRPC6 is a novel predisposing gene for ASD that may act in a multiple-hit model,” said Muotri. “This is the first study to use iPS cell-derived human neurons to model non-syndromic ASD and illustrate the potential of modeling genetically complex sporadic diseases using such cells.”

Find out more about how stem cell research could help solve the mysteries behind autism in our Autism Fact Sheet.

CIRM Scientists Discover Key to Blood Cells’ Building Blocks

Our bodies generate new blood cells—both red and white blood cells—each and every day. But reproducing that feat in a petri dish has proven far more difficult.

Pictured: sections from zebrafish embryos. Blood vessels are labeled in red, the protein complex that regulates inflammation green and cell nuclei in blue. The arrowhead indicates a potential HSC. The image at bottom right combines all channels. [Credit: UC San Diego School of Medicine]

Pictured: sections from zebrafish embryos. Blood vessels are labeled in red, the protein complex that regulates inflammation green and cell nuclei in blue. The arrowhead indicates a potential HSC. The image at bottom right combines all channels.
[Credit: UC San Diego School of Medicine]

But now, scientists have identified the missing ingredient to producing hematopoietic stem cells, or HSC’s—the type of stem cell that gives rise to all blood and immune cells in the body. The results, published last week in the journal Cell, describe how a newly discovered protein plays a key role in generating HSC’s in the developing embryo—giving scientists a more complete recipe to reproduce these cells in the lab.

The research, which was led by University of California, San Diego (UCSD) professor David Traver and supported by a grant from CIRM, offers renewed hope for the possibility of generating patient-specific blood or immune cells using induced pluripotent stem cell (iPS cell) technology.

As Traver explained in last week’s news release:

“The development of some mature cell lineages from iPS cells, such as cardiac or neural, has been reasonably straightforward, but not with HSCs. This is likely due, at least in part, to not fully understanding all the factors used by the embryo to generate HSCs.”

Indeed, the ability to generate HSCs has long challenged scientists, as outlined in a CIRM workshop from last year. But now, says Traver, they have found a crucial piece to the puzzle.

Specifically, the researchers investigated a signaling protein called tumor necrosis factor alpha—or TNFα for short— a protein known to be important for regulating inflammation and immunity. Previous research by this study’s first author, Raquel Espin-Palazon, and others also discovered it was related to the healthy function of blood vessels during embryonic development.

In this study, Traver, Espin-Palazon and the UCSD drilled down even further—and found that TNFα was required for the normal development of HSCs in the embryo. This surprised the research team, as the young embryo is generally considered to be sterile—with no need for a protein normally charged with regulating immune response to be switched on. Explained Traver:

“There was no expectation that pro-inflammatory signaling would be active at this time or in the blood-forming regions.”

While preliminary, establishing this relationship between TNFα and HSC formation will be a boon to researchers looking for new ways to generate large quantities of healthy, patient-specific red and white blood cells for those patients who so desperately need them.

Learn more about how stem cell technology could help treat blood diseases in our Thalassemia Fact Sheet.

Stem Cell Stories that Caught our Eye: Skin Cells to Brain Cells in One Fell Swoop, #WeAreResearch Goes Viral, and Genes Helps Stem Cells Fight Disease

Here are some stem cell stories that caught our eye this past week. Some are groundbreaking science, others are of personal interest to us, and still others are just fun.

Building a Better Brain Cell. Thanks to advances in stem cell biology, scientists have found ways to turn adult cells, such as skin cells, back into cells that closely resemble embryonic stem cells. They can then coax them into becoming virtually any cell in the body.

But scientists have more recently begun to devise ways to change cells from one type into another without first having to go back to a stem cell-like state. And now, a team from Washington University in St. Louis has done exactly that.

As reported this week in New Scientist, researcher Andrew Yoo and his team used microRNAs—a type of ‘signaling molecule’—to reprogram adult human skin cells into medium spiny neurons(MSNs), the type of brain cell involved in the deadly neurodegenerative condition, Huntington’s disease.

“Within four weeks the skin cells had changed into MSNs. When put into the brains of mice, the cells survived for at least six months and made connections with the native tissue,” explained New Scientist’s Clare Wilson.

This process, called ‘transdifferentiation,’ has the potential to serve as a faster, potentially safer alternative to creating stem cells.

#WeAreResearch Puts a Face on Science. The latest research breakthroughs often focus on the science itself, and deservedly so. But exactly who performed that research, the close-knit team who spent many hours at the lab bench and together worked to solve a key scientific problem, can sometimes get lost in the shuffle.

#WeAreResearch submission from The Thomson Lab at the University of California, San Francisco. This lab uses optogenetics, and RNAseq to probe cell fate decisions.

#WeAreResearch submission from The Thomson Lab at the University of California, San Francisco. This lab uses optogenetics, and RNAseq to probe cell fate decisions.

Enter #WeAreResearch, a new campaign led by the American Society for Cell Biology (ASCB) that seeks to show off science’s more ‘human side.’

Many California-based stem cell teams have participated—including CIRM grantee Larry Goldstein and his lab!

Check out the entire collection of submissions and, if you’re a member of a lab, submit your own. Prizes await the best submissions—so now’s your chance to get creative.

New Genes Help Stem Cells Fight Infection. Finally, UCLA scientists have discovered how stem cells ‘team up’ with a newly discovered set of genes in order to stave off infection.

Reporting in the latest issue of the journal Current Biology, and summarized in a UCLA news release, Julian Martinez-Agosto and his team describe how two genes—adorably named Yorkie and Scalloped—set in motion a series of events, a molecular Rube Goldberg device, that transforms stem cells into a type of immune system cell.

Importantly, the team found that without these genes, the wrong kind of cell gets made—meaning that these genes play a central role in the body’s healthy immune response.

Mapping out the complex signaling patterns that exist between genes and cells is crucial as researchers try and find ways to, in this case, improve the body’s immune response by manipulating them.

Moving one step closer to a therapy for type 1 diabetes

When I was a medical journalist one word I always shied away from was “breakthrough”. There are few true breakthroughs in medicine. Usually any advance is the result of years and years of work. That’s why good science takes time; it takes hundreds of small steps to make a giant leap forward.

Today we took one of those steps. ViaCyte, a company we have supported for many years, just announced that the first patient has been successfully implanted with a device designed to help treat type 1 diabetes.

It’s an important milestone for the company, for us, and of course for people with type 1 diabetes. As Dr. Paul Laikind, the President and CEO of ViaCyte, said in a news release, this is an exciting moment:

“To our knowledge, this is the first time that an embryonic stem cell-derived cell replacement therapy for diabetes has been studied in human subjects, and it represents the culmination of a decade of effort by the ViaCyte team, our collaborators, and our supporters at the California Institute for Regenerative Medicine and at JDRF.”

The VC-01 device is being tested in a clinical trial at the University of California, San Diego Health System. There are two goals; first to see if it is safe; and secondly to see if it helps patients who have type 1 diabetes. When the device is implanted under the skin the cells inside are able to sense when blood sugar is high and, in response, secrete insulin to restore it to a healthy level.

The beauty of the VC-01 is that while it lets cells secrete insulin out, it prevents the body’s own immune system from getting in and attacking the cells.

The device is about the length and thickness of a credit card but only half as wide which makes it easy to implant under the skin.

Today’s news, that this is now truly out of the lab and being tested in patients is an important step in a long road to showing that it works in patients. The people at ViaCyte, who have been working hard on this project for many years, know that they still have a long way to go but for today at least, this step probably feels a little bit more like a skip for joy.

From Stem Cells to Stomachs: Scientists Generate 3D, Functioning Human Stomach Tissue

The human stomach can be a delicate organ. For example, even the healthiest stomach can be compromised by H. pylori bacteria—a tiny but ruthless pathogen which has shown to be linked to both peptic ulcer disease and stomach cancer.

The best way to study how an H. pylori infection leads to conditions like cancer would be to recreate that exact environment, right down to the stomach itself, in the lab. But that task has proven far more difficult than originally imagined.

Part of a miniature stomach grown in the lab, stained to reveal various cells found in normal human stomachs [Credit: Kyle McCracken]

Part of a miniature stomach grown in the lab, stained to reveal various cells found in normal human stomachs [Credit: Kyle McCracken]

But now, scientists at the Cincinnati Children’s Hospital Medical Center and the University of Cincinnati College of Medicine have successfully grown functional, human stomach tissue in a dish—the first time such a feat has been accomplished.

Further, they were then able to test how human stomach tissue reacts to an invasion by H. pylori—a huge leap forward toward one day developing treatments for potentially deadly stomach disease.

Reporting in today’s issue of the journal Nature, senior author Jim Wells describes his team’s method of turning human pluripotent stem cells into stomach cells, known as gastric cells. Wells explained the importance of their breakthrough in a news release:

“Until this study, no one had generated gastric cells from human pluripotent stem cells. In addition, we discovered how to promote formation of three-dimensional gastric tissue with complex architecture and cellular composition.”

The team called this stomach tissue gastric organoids, a kind of ‘mini-stomach’ that mimicked the major cellular processes of a normal, functioning human stomach. Developing a human model of stomach development—and stomach disease—has long been a goal among scientists and clinicians, as animal models of the stomach did not accurately reflect what would be happening in a human stomach.

In this study, the research team identified the precise series of steps that can turn stem cells into gastric cells. And then they set these steps in motion.

Over the course of a month, the team coaxed the formation of gastric organoids that measured less than 1/10th of one inch in diameter. But even with this small size, the team could view the cellular processes that drive stomach formation—and discover precisely what happens when that process goes awry.

But what most intrigued the researchers, which also included first author University of Cincinnati’s Kyle McCracken, was how quickly an H. pylori infection impacted the health of the stomach tissue.

“Within 24 hours, the bacteria had triggered biochemical changes in the organ,” said McCracken.

According to McCracken, as the H. pylori infection spread from cell to cell, the researchers also recorded the activation of c-Met, a gene known to be linked to stomach cancer—further elucidating the relationship between H. pylori and this form of stomach disease.

Somewhat surprisingly, little was known about how gastric cells play a role in obesity-related diseases, such as type 2 diabetes. But thanks to Wells, McCracken and the entire Cincinnati Children’s research team—we are that much closer to shedding light on this process.

Wells also credits his team’s reliance on years of preliminary data performed in research labs around the world with helping them reach this landmark:

“This milestone would not have been possible if it hadn’t been for previous studies from many other basic researchers on understanding embryonic organ development.”

Scientists Develop Stem Cell ‘Special Forces’ in order to Target, Destroy Brain Tumors

Curing someone of cancer is, in theory, a piece of cake: all you have to do is kill the cancer cells while leaving the healthy cells intact.

But in practice, this solution is far more difficult. In fact, it remains one of the great unsolved problems in modern oncology: how do you find, target and destroy each individual cancer cell in the body—while minimizing damage to the surrounding cells.

Encapsulated toxin-producing stem cells (in blue) help kill brain tumor cells in the tumor resection cavity (in green). [Credit: Khalid Shah, MS, PhD]

Encapsulated toxin-producing stem cells (in blue) help kill brain tumor cells in the tumor resection cavity (in green). [Credit: Khalid Shah, MS, PhD]

But luckily, Harvard Stem Cell Institute scientists at Massachusetts General Hospital may have finally struck gold: they have designed special, toxin-secreting stem cells that can target and destroy brain tumors. Their findings, which were performed in laboratory mice and which appear in the latest issue of the journal STEM CELLS, offer up an entirely unique method for eradicating deadly cancers.

Harvard Neuroscientist Khalid Shah, who led the study, explained in last Friday’s news release that the idea of engineering stem cells to kill cancer cells is not new—but there was a key difference in scientists’ ability to target individual cells vs. difficult-to-reach tumors, which is often the case with brain cancer:

“Cancer-killing toxins have been used with great success in a variety of blood cancers, but they don’t work as well in solid tumors because the cancers aren’t as accessible and the toxins have a short half-life.”

The solution, Shah and his team argued, was stem cells. Previously, Shah and his team discovered that stem cells could be used to circumvent these problems. The fact that stem cells continuously renew meant that they could also be used to continually deliver toxins to brain tumors.

“But first, we needed to genetically engineer stem cells that could resist being killed themselves by the toxins,” said Shah.

In this study, the research team introduced a small genetic change, or mutation, into the stem cells so that they become impervious to the toxin’s harmful effects. They then introduced a second mutation that allowed the stem cells to maintain and produce and secrete toxins throughout the cells’ lifetime—effectively giving it an unlimited supply of ammunition to use once it encountered the brain tumor.

They then employed a common technique whereby the toxins were tagged so that they only sought out and infected cancer cells—leaving healthy cells unscathed.

“We tested these stem cells in a clinically relevant mouse model of brain cancer,” Shah described. “After doing all of the molecular analysis and imaging to track the inhibition of protein synthesis within brain tumors, we do see the toxins kill the cancer cells and eventually prolonging the survival in animal models.”

While preliminary, these results are encouraging. As the team continues to refine their method of development and delivery, they are optimistic that they can bring their methods to clinical trial within the next five years.

Stem Cell Stories that Caught our Eye: Perspective on “Walking” Patient, Blood Stem Cells have a Helper and Three Clinical Trials at One Campus

Here are some stem cell stories that caught our eye this past week. Some are groundbreaking science, others are of personal interest to us, and still others are just fun.

Some perspective on nasal stem cells and ”walking” patient. PZ Meyers writing on ScienceBlogs did a good job of putting some perspective into the hype in many news outlets about the spinal cord injury patient who was treated with nasal stem cells. He starts out admitting he was “incredulous” that there was anything to the study, but after a thorough reading of the actual journal article he was convinced that there was some real, though modest gain in function for the patient. His conclusion:

“Sad to say, the improvements in the man’s motor and sensory ability are more limited and more realistic than most of the accounts would have you think.”

The research team actually reported on three patients. One got barely noticeable improvement; the patient in the news reports regained about 25 percent of function—which is indisputably a major gain in this population—and the third was somewhere in between.

shutterstock_132771389

Meyer speculated about a reason for the improvements that was left out of most press reports. In addition to the stem cell harvested from the patients’ own nasal passages injected on either side of the injury the team also harvested nerve fibers from the patients legs and transplanted them across the site of the injury. They hoped the nerve strands would act as a bridge for the stem cells to grow and close the gap. It is also possible that being nerve cells they could provide the right cell-to-cell signals directing the nasal stem cells to become nerves. Meyers closed with an appropriate summary:

“I think there’s good reason to be optimistic and see some hope for an effective treatment for serious spinal cord injuries, but right now it has to be a realistic hope — progress has been made. A cure does not exist.”

Body’s own helper for blood stem cells found. In a case of the children ordering around the parents, a team at the Stowers Institute in Kansas City found that one of the progeny of blood-forming stem cells in the bone marrow can control the activity of the stem cells. In particular, they were looking at megacarocytes, the relatively rare bone marrow cells that normally produce the blood platelets you need for clotting a wound.

Blood stem cells are the most common stem cell therapy today, but one plagued by our limited ability to control their growth. Knowing this involvement of their offspring gives researcher a new avenue to search for ways to grow the much needed parent stem cells. Genetic Engineering & Biotechnology News wrote up the findings.

(Yes, I may be the only person in World Series-obsessed San Francisco writing something positive about Kansas City this week.)

Three clinical trails launched at just one campus. We have written individually about three clinical trials that began in the last month at the University of California, San Diego. Now, the university has written a good wrap up of the three trials that got posted to ScienceDaily.

Collectively, the three trials show the breadth of stem cell research starting to reach patients. One trial, for diabetes, uses cells derived from embryonic stem cells encased in a pouch to protect them from immune rejection. Another uses cells derived from fetal nerve stem cells to treat spinal cord injury. And the third involves a drug that targets the cancer stem cells that are believed to cause much of the spread of the disease and resistance to chemotherapy in cancer patients.

CIRM is funding two of the three trials and supported much of the basic science that led to the third. We expect to be funding 10 projects with approved clinical trials by the end of the year. The field is moving.

Don Gibbons

CIRM-Funded Scientists Make New Progress Toward Engineering a Human Esophagus

Creating tissues and organs from stem cells—often referred to as ‘tissue engineering’—is hard. But new research has discovered that the process may in fact be a little easier than we once thought, at least in some situations.

Engineered human esophageal tissue [Credit: The Saban Research Institute].

Engineered human esophageal tissue [Credit: The Saban Research Institute].

Last week, scientists at The Saban Research Institute of Children’s Hospital Los Angeles announced that the esophagus—the tube that transports food, liquid and saliva between the mouth and the stomach—can be grown inside animal models after injecting the right mix of early-stage, or ‘progenitor,’ esophageal cells.

These findings, published in the journal Tissue Engineering Part A, are an important step towards generating tissues and organs that have been damaged due to disease or—in some cases—never existed in the first place.

According to stem cell researcher Tracy Grikscheit, who led the CIRM-funded study, the researchers first implanted a biodegradable ‘scaffold’ into laboratory mice. They then injected human progenitor cells into the mice and watched as they first traveled to the correct location—and then began to grow. The ability to both migrate to the right location and differentiate into the right cell type, without the need for any external coaxing, is crucial if scientists are to successfully engineer such a critical type of tissue.

“Different progenitor cells can find the right ‘partner’ in order to grow into specific esophageal cell types—and without the need for [outside] growth factors,” explained Grikscheit in a news release. “This means that successful tissue engineering of the esophagus is simpler than we previously thought.”

Grikscheit, who is also a pediatric surgeon as Children’s Hospital Los Angeles, was particularly hopeful with how their findings might one day be used to treat children born with portions of the esophagus missing—as well as adults suffering from esophageal cancer, the fastest-growing cancer in the U.S.

“We have demonstrated that a simple and versatile, biodegradable polymer is sufficient for the growth of a tissue-engineered esophagus from human cells. This not only serves as a potential source of tissue, but also a source of knowledge—as there are no other robust models available for studying esophageal stem cell dynamics.”

Want to learn more about tissue engineering? Check out these video highlights from a recent CIRM Workshop on the field.