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.

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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 2.0; saving time, saves lives

It’s been almost ten years since the voters of California approved Proposition 71, creating the Stem Cell Agency and giving us $3 billion to fund stem cell research. So this is an appropriate time to look back and see what we have done with the money so far, the progress that’s been made, and where we are heading in the next ten years.

Over the next few weeks we’ll be taking a more detailed look at all these elements – it’s too much to cover in one blog – but let’s start with where we’re heading. At yesterday’s meeting of our governing Board, the Independent Citizens Oversight Committee, President & CEO, C. Randal Mills, Ph.D. charted a course for future funding.

Randy Mills, Stem Cell Agency President & CEO

Randy Mills, Stem Cell Agency President & CEO

Right now it can take up to two years for a project going into clinical trials to apply for and get funding from us. As Randy said in a news release we issued after the meeting, “That’s just unacceptable”:

Under what he is calling CIRM 2.0, Randy is proposing to trim that down dramatically:

“We are going to shorten that to just 120 days. But we’re not just making it faster, we’re also making it easier for companies or institutions with a therapy that is ready to go into clinical trials to be able to get funding for their project when they need it. Under this new system they will be able to apply anytime, and not have to try and shoehorn their needs into our application process.”

The goal is not just to make it easier to apply for funding, but also to get more, high quality applications. Right now there is pressure on companies to apply before they are really ready because they know if they miss a current application deadline it could be another year to 18 months before another award cycle comes around. Under CIRM 2.0 application will be accepted on a monthly basis, so applicants won’t have to worry about missing one deadline – they can just apply the following month. Applying when they are ready will increase the likelihood that the projects will be and of high quality.

And as Randy points out, if it works at the clinical stage of funding, it can work at every stage:

“Speeding up the process, at all stages of research, just makes sense. The faster that researchers can get access to the funds they need to do their work, the faster they are likely to be able to produce something that helps patients.”

The speeding up of the process doesn’t just involve companies and researchers being able to apply anytime, it also means that when they do apply they’ll have to have all the supporting documentation and studies on hand to show they are ready to go as soon as the Board approves funding.

In the past there was often a delay of six months or even more after an application had been approved for funding while research milestones were negotiated and agreements signed. Because CIRM 2.0 will involve identifying milestones much earlier in the application process that delay will disappear.

This new approach involves a complete overhaul of the way we currently work but we think it’s worth it. We plan to start by introducing these changes for the projects that are furthest along, those ready to go into clinical trials, but in time we intend extending this to cover everything we fund.

Making these changes will help us trim a two-year process down to just three months. That means any therapy that proves successful is getting to the patients who need it much sooner than it otherwise would. And with many of the diseases we are targeting, saving time means saving lives.

October ICOC Board Meeting to Begin Soon

The October ICOC Board Meeting begins this morning in Los Angeles, CA.

The complete agenda can be found here, including a special Spotlight on Disease focusing on Retinitis Pigmentosa.

For those not able to attend, you are welcome to dial in!

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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.

Stem cell stories that caught our eye: Some good news got a little overplayed on blindness and Alzheimer’s

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.

Stories on blindness show too much wide-eyed wonder. While our field got some very good news this week when Advanced Cell Technologies (ACT) published data on its first 18 patients treated for two blinding diseases, many of the news stories were a little too positive. The San Diego Union Tribune ran the story from Associated Press writer Maria Cheng who produced an appropriately measured piece. She led with the main point of this early-phase study—the cells implanted seem to be safe—and discussed “improved vision” in half the patients. She did not imply their sight came back to normal. Her third paragraph had a quote from a leading voice in the field Chris Mason of University College London:

“It’s a wonderful first step but it doesn’t prove that (stem cells) work.”

The ACT team implanted a type of cell called RPE cells made from embryonic stem cells. Those cells are damaged in the two forms of blindness tested in this trial, Stargardt’s macular dystrophy and age-related macular degeneration, the leading cause of blindness in the elderly. Some of the patients have been followed for three years after the cell transplants, which provides the best evidence to date that cells derived from embryonic stem cells can be safe. And some of the patients regained useful levels of vision, which with this small study you still have to consider other possible reasons for the improvement, but it is certainly a positive sign.

CIRM funds a team using a different approach to replacing the RPE cells in these patients and they expect to begin a clinical trial late this year

Stem cells create stronger bone with nanoparticles.   Getting a person’s own stem cells to repair bad breaks in their bones certainly seems more humane than hacking out a piece of healthy bone from some place else on their body and moving it to the damaged area. But our own stem cells often can’t mend anything more than minor breaks. So, a team from Keele University and the University of Nottingham in the U.K. laced magnetic nanoparticles with growth factors that stimulate stem cell growth and used external magnets to hold the particles at the site of injury after they were injected.

It worked nicely in laboratory models as reported in the journal Stem Cells Translational Medicine, and reported on the web site benzinga. Now comes the hard step of proving it is safe to test in humans

Stem cells might end chronic shortage of blood platelets. Blood platelets—a staple of cancer therapy because they get depleted by chemotherapy and radiation—too often are in short supply. They can only set on the shelf for five days after a donation. If we could generate them from stem cells, they could be made on demand, but you’d have to make many different versions to match various peoples’ blood type. The latter has been a bit of a moot point since no one has been able to make clinical grade platelets from stem cells.

plateletsA paper published today by Advanced Cell Technologies may have solved the platelet production hurdle and the immune matching all at once. (ACT is having a good week.) They produced platelets in large quantities from reprogrammed iPS type stem cells without using any of the ingredients that make many iPS cells unusable for human therapy. And before they made the platelets, they deleted the gene in the stem cells responsible for the bulk of immune rejection. So, they may have created a so-called “universal” donor.

They published their method in Stem Cell Reports and Reuters picked up their press release. Let’s see if the claims hold up.

Alzheimer’s in a dish—for the second time. My old colleagues at Harvard got a little more credit than they deserved this week. Numerous outlets, including the Boston Globe, picked up a piece by The New York Times’ Gina Kolata crediting them with creating a model of Alzheimer’s in a lab dish for the first time. This was actually done by CIRM-grantee Lawrence Goldstein at the University of California, San Diego, a couple years ago.

But there were some significant differences in what the teams did do. Goldstein’s lab created iPS type stem cells from skin samples of patients who had a genetic form of the disease. They matured those into nerve cells and did see increased secretion of the two proteins, tau and amyloid-beta, found in the nerves of Alzheimer’s patients. But they did not see those proteins turn into the plaques and tangles thought to wreak havoc in the disease. The Harvard team did, which they attributed, in part, to growing the cells in a 3-dimensional gel that let the nerves grow more like they would normally.

The Harvard team, however, started with embryonic stem cells, matured them into nerves, and then artificially introduced the Alzheimer’s-associated gene. They have already begun using the model system to screen existing drugs for candidates that might be able to clear or prevent the plaques and tangles. But they introduced the gene in such a way the nerve cells over express the disease gene, so it is not certain the model will accurately predict successful therapies in patients.

Don Gibbons

UCLA Study Suggests New Way to Mend a Broken Heart

When you suffer a heart attack, your heart-muscle cells become deprived of oxygen. Without oxygen, the cells soon whither and die—and are entombed within scar tissue. And once these cells die, they can’t be brought back to life.

But maybe—just maybe—there is another way to build new heart muscle. And if there is, scientists like Dr. Arjun Deb at the University of California, Los Angeles (UCLA), are hot on the trail to find it.

Scar forming cells (in red) in a region of the injured heart expressing blood vessel cell marker in green and thus appearing yellow (see arrows). This study observed that approximately a third of the scar-forming cells in the injured region of the heart adopted "blood vessel" cell-like characteristics. [Credit: Dr. Arjun Deb/Nature]

Scar forming cells (in red) in a region of the injured heart expressing blood vessel cell marker in green and thus appearing yellow (see arrows). This study observed that approximately a third of the scar-forming cells in the injured region of the heart adopted “blood vessel” cell-like characteristics. [Credit: Dr. Arjun Deb/Nature]

Published yesterday in the journal Nature, Deb and his team at UCLA’s Eli & Edythe Broad Center for Regenerative Medicine and Stem Cell Research have found some scar-forming cells in the heart have the ability to become blood vessel-forming cells—if given the proper chemical ‘boost.’

“It is well known that increasing the number of blood vessels in the injured heart following a heart attack improves its ability to heal,” said Deb. “We know that scar tissue in the heart is associated with poor prognosis. Reversing or preventing scar tissue from forming has been one of the major challenges in cardiovascular medicine.”

Tackling the ever-growing problem in heart disease can seem an almost insurmountable task. While heart disease claims more lives worldwide than any other disease, advances in modern medicine in recent decades mean that more and more people are surviving heart attacks, and living with what’s called ‘heart failure,’ for their hearts can no longer beat at full capacity, and they have trouble taking long walks or even going up a flight of stairs.

Transforming this scar tissue into functioning heart muscle has therefore been the focus of many research teams, including CIRM grantees such as Drs. Deepak Srivastava and Eduardo Marbán, who have each tackled the problem from different angles. Late last year, treatment first designed by Marbán and developed by Capricor Therapeutics got the green light for a Phase 2 Clinical Trial.

In this study, Deb and his team focused on scar-forming cells, called fibroblasts, and blood-vessel forming cells, called endothelial cells. Previously, experiments in mice revealed that many fibroblasts literally transformed into endothelial cells—and helped contribute to blood vessel formation in the injured area of the heart. The team noted this phenomenon has been called the mesenchymal-endothelial transition, or MEndoT.

In this study, the researchers identified the molecular mechanism behind MEndoT—and further identified a small molecule that can enhance this transition, thus boosting the formation of blood vessels in the injured heart. This study bolsters the idea of focusing on the creation of blood vessels as a way to help reverse damage caused by a heart attack. Said Deb:

“Our findings suggest the possibility of coaxing scar-forming cells in the heart to change their identity into blood vessel-forming cells, which could potentially be a useful approach to better heart repair.”

The Nose Knows: Stem Cells are Vital Players in Brain Circuits Responsible for Smell

Ah, the smell of coffee! You can thank your olfactory bulb.

Ah, the smell of coffee! You can thank your olfactory bulb.

Ah, the mouth-watering scent of freshly baked bread and the intense aroma of roasted coffee beans. You can thank nerve cells in the front of your brain — in direct contact with your nasal passages — that convert odor molecules in the air into brain signals and generate your perception of those wonderful smells.

Loss of the sense of smell is often one of the earliest symptoms in people stricken with brain disorders such as Parkinson’s and Alzheimer’s. So the study of this part of the brain called the olfactory bulb, that’s responsible for smell perception, is an attractive area of research that could help provide insights into fundamental brain function and its connection to neurodegenerative diseases. Last week, scientists at the National Institutes of Health (NIH) moved the field a step forward by reporting in the Journal of Neuroscience that brain stem cells play a vital role in sustaining the proper brain cell circuitry in the olfactory bulb.

Studies in adult mice have shown that brain stem cells deep inside the brain have the uncanny ability to travel to the olfactory bulb, transform into nerve cells, and set up appropriate circuits with surrounding nerve cells. The NIH team had previously demonstrated that when a nostril is plugged for 20 days in these mouse studies, depriving the olfactory system of stimulation, the nerve cell connections scatter and become very disorganized. But after removing the plug for 40 days the proper connections and patterns are re-established.

The brain stem cells uncanny ability to migrate through the thin rostral stream, transform in to neurons, and make the right connections with surrounding neurons in the olfactory bulb, the large structure in the upper right. (Image credit:  Belluscio Lab, NINDS).

Newly born nerve cells migrate along a thin path and connect up with surrounding nerve cells in the olfactory bulb, the large structure in the upper right. (Image credit: Belluscio Lab, NINDS).

In the current study, the team used genetic engineering techniques to precisely remove only those brain stem cells in adult mice that transform into the olfactory nerve cells. Again when a nostril was plugged the nerve cell connections were disrupted. But this time when the brain stem cells were eliminated and the nose plug removed, the nerve cell connections remained disorganized. This result reveals that the system relies on a replenishing supply of brain stem cells. As senior author Leonardo Belluscio, Ph.D. states in a NIH press release:

“We found that without the introduction of the new neurons, the system could not recover from its disrupted state.”

Even when the brain stem cells were eliminated in mice that were not given the nose block, a deterioration of the olfactory bulb nerve cell network was still observed by the research team. These results turn scientists’ understanding of brain circuits on its head: rather than being mostly stable structures, in this case the olfactory brain circuits appear unstable by default and must continually receive new neurons (from stem cells) to not only restore disrupted connections but also to preserve stable circuits.

Dr. Belluscio reflected on these intriguing results and its implications for neurologic disease:

“This is an exciting area of science. I believe the olfactory system is very sensitive to changes in neural activity and given its connection to other brain regions, it could lend insight into the relationship between olfactory loss and many brain disorders.”

To hear more from Dr. Belluscio about these results, watch this video interview. And for more about the role of stem cells in adult brain circuitry, watch this seminar by UCSF researcher and CIRM grantee Arturo Alvarez-Buylla, PhD.

Cranking it Up to Eleven: Heightened Growth of Neural Stem Cells Linked to Autism-like Behavior

Autism is not one single disease but a suite of many, which is why researchers have long struggled to understand its underlying causes. Often referred to as the Autism Spectrum Disorders, autism has been linked to multiple genetic and environmental factors—different combinations of which can all result in autism or autistic-like behavior.

Could an unusual boost in neural stem cell growth during pregnancy be linked to autistim-like behavior in children?

Could an unusual boost in neural stem cell growth during pregnancy be linked to autitism-like behavior in children?

But as we first reported in last week’s Weekly Roundup, scientists at the University of California, Los Angeles (UCLA) have identified a new factor that can occur during pregnancy and that may be linked to the development of autism-like behavior. These results shed new light on a notoriously murky condition.

UCLA scientist Dr. Harley Kornblum led the study, which was published last week in the journal Stem Cell Reports.

In it, Kornblum and his team describe how inflammation in pregnant mice, known as ‘maternal inflammation’ caused a spike in the production of neural stem cells—cells that one day develop into mature brain cells, such as neurons and glia cells. This abnormal growth, the team argues, led to enlarged brains in the newborn mice and, importantly, autism-like behavior such as decreased vocalization and social behavior, as well as overall increase in anxiety and repetitive behaviors, such as grooming. As Kornblum explained in a news release:

“We have now shown that one way maternal inflammation could result in larger brains and, ultimately, autistic behavior is through the activation of the neural stem cells that reside in the brain of all developing and adult mammals.”

However, Kornblum notes that many environmental factors may cause inflammation during pregnancy—and the inflammation itself is not thought to directly cause autism.

“Autism is a complex group of disorders, with a variety of causes. Our study shows a potential way that maternal inflammation could be one of those contributing factors, even if it is not solely responsible, through interactions with known risk factors.”

These known risk factors include genetic mutations, such as those to a gene called PTEN, which have been shown to increase one’s risk for autism.

Further research by Kornblum’s team further clarified the connection between inflammation and neural stem cell overgrowth. Specifically, they noticed a series of chemical reactions, known as a molecular pathway, appeared to stimulate the growth of neural stem cells in the developing mice. The identification of pathways such as these are vital when exploring new types of therapies—because once you know the pathway’s role in disease, you can then figure out how to change it.

“The discovery of these mechanisms has identified new therapeutic targets for common autism-associated risk factors,” said Dr. Janel Le Belle, the paper’s lead author. “The molecular pathways that are involved in these processes are ones that can be manipulated and possibly even reversed pharmacologically.”

These findings also support previous clinical findings that the roots of autism likely begin in the womb and continue to develop after birth.

One key difference between this work and previous studies, however, was that most studies point to irregularities in the way that neurons are connected as a key factor that leads to autism. This study points to not just a network ‘dysregulation,’ but also perhaps an overabundance of neurons overall.

“Our hypothesis—that one potential means by which autism may develop is through an overproduction of cells in the brain, which then results in altered connectivity—is a new way of thinking about autism.”

Advances in the fields of stem cell biology and regenerative medicine have given new hope to families caring for autistic loved ones. Read more about one such family in our Stories of Hope series. You can also learn more about how CIRM-funded researchers are building our understanding of autism in our recent video: Reversing Autism in the Lab with help from Stem Cells and the Tooth Fairy.

Stem cell stories that caught our eye: fast track marketing in Japan, a 3D cell tour and autism

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.

Event showed great progress, but Japan nipping at our heals. The San Diego Union Tribune’s Brad Fikes seemed to be enjoying covering the Stem Cell Meeting on the Mesa in his own backyard in La Jolla. He stayed for the full two days of the industry Partnering Forum and when we chatted he said he had more good material than he could use. I was certainly willing to second the sentiment of the opening paragraph of the story he wrote:

“More than ever before, stem cell therapies appear poised to transform medicine — potentially curing heart disease, diabetes and paralyzing injuries, among other ailments.”

But the last portion of his piece was a little unsettling. He noted the frequent discussion at the meeting of Japan’s new fast track path for marketing stem cell therapies. The CEO of Athersys, one of the leading companies in the field, announced at the meeting that his company would be taking their lead product to Japan first for marketing, not the U.S.

Turning cell biology into a 3-D game. The ability to track cells has become one of the most important limiting factors in stem cell biology. We need to know where cells go when they are transplanted in the body, but even before that, we have found that the interaction of cells with their environment often determines if stem cell offspring do their jobs and we need to track cells to understand this.

Now a team at Drexel University that includes an expert in computer software and hardware used in gaming has

Researchers at Drexel touring a group of cells using 3D glasses.

Researchers at Drexel touring a group of cells using 3D glasses.

provided the field with an invaluable tool. They can label various cells with distinctive markers and follow their movements. More important they can use an elaborate software program to integrate individual slices of a tissue into a 3D sample that researchers can “tour” while wearing 3D glasses.

Red Orbit quoted Andrew Cohen the leader of the computer development team:

“It’s like Photoshop for cell biologists. The software outlines cells and blood vessels, keeping track of them as they’re dividing and moving around one another. This provides a wealth of information on the patterns of cell shape, motion and division. Visualization of the 3-D microscopy data together with the analysis results is a key step to measure and ultimately understand what drives these cells.”

Cally Templea, a leading expert from the Neural Stem Cell Institute in Rensselaer, NY, was also quoted about the power of this new tool to help stem cell biologists understand how stem cells interact with their environment:

“LEVER 3-D is amazing, it opens new vistas for understanding the stem cell niche.”

Autism linked to stem cell burst (in mice). The accelerated brain growth seen right after birth in many people with autism spectrum disorder has been linked to a burst of nerve stem cell division triggered by inflammation. The study at the University of California, Los Angeles, could explain why inflammation during pregnancy, either due to an autoimmune reaction or an infection, has been shown to be a risk factor for the disorder.

Health Canal posted the press release from the university that quoted the senior author of a paper in the journal Stem Cell Reports, Harley Kornblum:

“We have now shown that one way maternal inflammation could result in larger brains and, ultimately, autistic behavior, is through the activation of the neural stem cells that reside in the brain of all developing and adult mammals.”

The researchers gave pregnant mice a toxin found in bacteria and discovered that it triggered an excess production of nerve stem cells in their pups. This resulted in enlarged brains and behavior associated with autism, such as a reduced interest in interacting with other mice.

Little guy regrowing his head could help us. While a few species have the ability to regrow a severed body part, the tiny Hydractinia—commonly called snail fur—out does the rest in that it can regrow its head. BBC did a nice job of describing work at the University of Galway trying to explain how it accomplishes the feat and putting the work into perspective with other recent research findings.

After harvesting the creatures off the backs of hermit crabs the Galway team isolated embryonic stem cells from them, to which they attributed the ability to regrow something as complex as a head. The snail fur may be unique in that no other adult animal is believed to harbor embryonic stem cells. The researchers hope to use the tiny creature to learn how we might be able to turn on some ancestral regenerative capacity in humans.

Don Gibbons

Scientists Reach Yet Another Milestone towards Treating Type 1 Diabetes

There was a time when having type 1 diabetes was equivalent to a death sentence. Now, thanks to advances in science and medicine, the disease has shifted from deadly to chronic.

But this shift, doctors argue, is not good enough. The disease still poses significant health risks, such as blindness and loss of limbs, as the patients get older. There has been a renewed effort, therefore, to develop superior therapies—and those based on stem cell technology have shown significant promise.

Human stem cell-derived beta cells that have formed islet like clusters in a mouse. Cells were transplanted to the kidney capsule and photo was taken two weeks later by which time the beta cells are making insulin and have cured the mouse's diabetes. [Credit: Douglas Melton]

Human stem cell-derived beta cells that have formed islet like clusters in a mouse. Cells were transplanted to the kidney capsule and photo was taken two weeks later by which time the beta cells are making insulin and have cured the mouse’s diabetes. [Credit: Douglas Melton]

Indeed, CIRM-funded scientists at San Diego-based Viacyte, Inc. recently received FDA clearance to begin clinical trials of their VC-01 product candidate that delivers insulin via healthy beta cells contained in a permeable, credit card-sized pouch.

And now, scientists at Harvard University have announced a technique for producing mass quantities of mature beta cells from embryonic stem cells in the lab. The findings, published today in the journal Cell, offer additional hope for the millions of patients and their families looking for a better way to treat their condition.

The team’s ability to generate billions of healthy beta cells—cells within the pancreas that produce insulin in order to maintain normal glucose levels—has a particular significance to the study’s senior author and co-scientific director of the Harvard Stem Cell Institute, Dr. Doug Melton. 23 years ago, his infant son Sam was diagnosed with type 1 diabetes and since that time Melton has dedicated his career to finding better therapies for his son and the millions like him. Melton’s daughter, Emma, has also been diagnosed with the disease.

Type 1 diabetes is an autoimmune disorder in which the body’s immune system systematically targets and destroys the pancreas’ insulin-producing beta cells.

In this study, the team took human embryonic stem cells and transformed them into healthy beta cells. They then transplanted them into mice that had been modified to mimic the signs of diabetes. After closely monitoring the mice for several weeks, they found that their diabetes was essentially ‘cured.’ Said Melton:

“You never know for sure that something like this is going to work until you’ve tested it numerous ways. We’ve given these cells three separate challenges with glucose in mice and they’ve responded appropriately; that was really exciting.”

The researchers are undergoing additional pre-clinical studies in animal models, including non-human primates, with the hopes that the 150 million cells required for transplantation are also protected from the body’s immune system, and not destroyed.

Melton’s team is collaborating with Medical Engineer Dr. Daniel G. Anderson at MIT to develop a protective implantation device for transplantation. Said Anderson of Melton’s work:

“There is no question that the ability to generate glucose-responsive, human beta cells through controlled differentiation of stem cells will accelerate the development of new therapeutics. In particular, this advance opens the doors to an essentially limitless supply of tissue for diabetic patients awaiting cell therapy.”