Stem cell stories that caught our eye: a good review at the NY Times, expanding cord blood and leukemia

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.

Review paints picture of the field today.
A writer I have respected for many years, Karen Weintraub, wrote a nice review of the current state of stem cell clinical trials in the Tuesday Science Times in the New York Times. She discusses the steady, methodical progress being made:

“Researchers have been slowly learning how to best use stem cells, what types to use and how to deliver them to the body — findings that are not singularly transformational, but progressive and pragmatic.”

She quotes our senior VP Ellen Feigal about the safety seen so far in clinical trials and notes that CIRM should have 10 clinical trials enrolling patients by the end of the year. She also covers the dangers of clinics offering unproven therapies and the power of using iPS-type stem cells to model diseases in the laboratory. Overall, a nicely balanced piece.

Making mitochondrial disease and 3-parent embryos personal. A little newspaper in Oregon called the Willamet Week has published a story that makes the issues around so-called “three-parent” babies very personal. The controversial procedure aims to allow women with rare mitochondrial diseases to have normal children.

Mitochondria, known as the powerhouses of the cell, have the unusual trait of being the only part of the cell besides the nucleus to have any DNA. It is these few genes in the mitochondria that we inherit solely from our mothers because when the DNA from the egg and sperm fuse, the mother’s mitochondria stay in the fluid outside the nucleus. So, to avoid passing along faulty mitochondrial genes, a team in Oregon devised a way to insert the DNA from the mother’s nucleus into a donor egg that had its nucleus removed, a process called nuclear transfer.

Guided by a microscope researchers insert the nucleus from one woman into the egg of another

Guided by a microscope researchers insert the nucleus from one woman into the egg of another

The paper provides a long read—nearly 4,000 words—that goes into great detail about the procedure, the ethics, the research team’s views on the ethics, and the personal story of a patient living with a disease of exhaustion she calls “mitochondrial crash.” The writer lets the patient have the last word on ethics:

“To me it’s win-win because you’re not messing with God’s child. You’re just taking out the bad parts. I don’t want to pick out a blond-haired, blue-eyed tall kid, picking your child’s traits, but to rule out a potentially lethal chronic illness brings in a whole different story.”


Cord blood might now save more adult cancer patients.
Umbilical cord blood is a literal lifesaver for many pediatric cancer patients allowing them to withstand harsh chemotherapy and be rescued by the stem cells in the cord blood. But the procedure is used in few adults because the vast majority of cord blood samples don’t have enough stem cell for an adult requiring the use of two cord samples and doubling the chance for potentially deadly immune reactions.

A team at the University of Montreal screened more than 5,000 molecules looking for one that would let them expand the number of stem cells from one sample in the lab. They hit upon one that they say could allow a 10-fold increase in the number of single cord samples suitable for adults. They expect to begin clinical trials in December.

Science News ran a brief review of the work and the blog Science 2.0 ran the university’s press release with a bit more detail.

Trial begins with cancer drug named for CIRM
Researchers at the University of California, San Diego, announced this week that they had begun a clinical trial with leukemia patients using a drug named for our agency cirmtuzumab. This molecule, in the class of drugs called antibodies, disables a protein that cancer stem cells use to accelerate the growth of cancer.

This trial, for patients with recurrence of their chronic lymphocytic leukemia, became the third CIRM funded team this month announcing plans to start clinical trials. In addition to our blog post the San Diego Union Tribune wrote about the latest trial, and we issued press releases on the trials for spinal cord injury and diabetes.

Don Gibbons

New formula a more efficient way to reprogram adult cells to become like embryonic stem cells

Shinya Yamanaka won the Nobel Prize for developing a recipe of genetic factors that can turn back the clock of adult cells and make them behave like embryonic stem cells. But he would be the first to tell you his recipe ultimately may not be the best one for making these stem cells called iPS cells.

Virtually from the day he published his groundbreaking work, teams around the world have tried to develop new formulas that get around some problems with the original. One issue is the low efficiency of getting true stem cells. Another is the high rate of genetic aberrations that can be produced in the resulting stem cells.

Now, a team pairing researchers at the Hebrew University in Jerusalem and the Whitehead Institute in Cambridge, Massachusetts, has published a new recipe that seems to yield many more true stem cells, ones that are called pluripotent because they can make all cell types. The new cells also seem to have fewer genetic alterations, which could make them safer for clinical use in people.

They made the improved cells by moving from OSKM to SNEL—from the original genetic factors, Oct4, Sox2, Klf4 and Myc, to Sall4, Nanog, Esrrb and Lin28. An elaborate computer analysis of the function of genes helped them come up with the formula.

This work used mouse cells, so up next on their agenda is coming up with a similar formula that works in human cells. HealthCanal ran the university’s press release and Genetic Engineering & Biotechnology News ran a slightly more technical analysis of the work.

Don Gibbons

Harder, Better, Faster, Stronger: Scientists Work to Create Improved Immune System One Cell at a Time

The human immune system is the body’s best defense against invaders. But even our hardy immune systems can sometimes be outpaced by particularly dangerous bacteria, viruses or other pathogens, or even by cancer.

Salk Institute scientists have developed a new cellular reprogramming technique that could one day boost a weakened immune system.

Salk Institute scientists have developed a new cellular reprogramming technique that could one day boost a weakened immune system.

But what if we could give our immune system a boost when it needs it most? Last week scientists at the Salk Institute for Biological Sciences devised a new method of doing just that.

Reporting in the latest issue of the journal Stem Cells, Dr. Juan Carlos Izpisua Belmonte and his team announce a new method of creating—and then transplanting—white blood cells into laboratory mice. This new and improved method could have significant ramifications for how doctors attack the most relentless disease.

The authors achieved this transformation through the reprogramming of skin cells into white blood cells. This process builds on induced pluripotent stem cell, or iPS cell, technology, in which the introduction of a set of genes can effectively turn one cell type into another.

This Nobel prize-winning approach, while revolutionary, is still a many months’ long process. In this study, the Salk team found a way to shorten the cellular ‘reprogramming’ process from several months to just a few weeks.

“The process is quick and safe in mice,” said Izpisua Belmonte in a news release. “It circumvents long-standing obstacles that have plagued the reprogramming of human cells for therapeutic and regenerative purposes.”

Traditional reprogramming methods change one cell type, such as a skin cell, into a different cell type by first taking them back into a stem cell-like, or ‘pluripotent’ state. But here, the research team didn’t take the cells all the way back to pluripotency. Instead, they simply wiped the cell’s memory—and gave it a new one. As first author Dr. Ignacio Sancho-Martinez explained:

“We tell skin cells to forget what they are and become what we tell them to be—in this case, white blood cells. Only two biological molecules are needed to induce such cellular memory loss and to direct a new cell fate.”

This technique, which they dubbed ‘indirect lineage conversion,’ uses the molecule SOX2 to wipe the skin cell’s memory. They then use another molecule called miRNA 125b to reprogram the cell into a white blood cell.

These newly generated cells appear to engraft far better than cells derived from traditional iPS cell technology, opening the door to therapies that more effectively introduce these immune cells into the human body. As Sanchi-Martinez so eloquently stated:

“It is fair to say that the promise of stem cell transplantation is now closer to realization.”

Stories of Hope: Stroke

Six months after surviving a stroke, Sonia Olea wanted to die. Her right leg was weak, her right arm useless. She had trouble speaking and even small tasks were challenging. Just making a phone call was virtually impossible. One morning, she woke up with her arm pinned in an awkward, painful position. After finally repositioning it, she wanted to call her fiancé, but knew she couldn’t get the words out. That’s when it hit her.

Sonia has seen first hand how a stroke can rob you of even your most basic abilities.

Sonia has seen first hand how a stroke can rob you of even your most basic abilities.

“I thought, I’m only 32,” says Sonia. “How could this be happening to me?”

Nobody really had an answer. A stroke occurs when a blood clot blocks a vessel in the brain and cuts off blood flow. Brain cells begin to die within minutes when they are deprived of oxygen and nutrients. Stroke rates are on the rise for young adults for a variety of reasons but no one could pinpoint specifically what caused hers.

Slowly, Sonia fought back from her depression and realized she could do this. She would find a way to recover. Just one year later, she got a call from Stanford University; asking if she would be willing to participate in a cutting-edge, stem cell-based clinical trial.

Was she ever. The answer, says Sonia, was a no-brainer.

Rescuing Brain Cells
Led by CIRM grantee Gary Steinberg, M.D., Ph.D., chairman of the Department of Neurosurgery at Stanford School of Medicine, the early phase clinical trial tested the safety of transplanting bone marrow stem cells into the brain. It was a revolutionary approach.

“The old notion was that you couldn’t recover from a stroke after around three months,” says Steinberg. “At that point, the circuits were completely dead—and you couldn’t revive them.”

While this was partially true, it was thought that brain cells, or neurons, just outside the stroke damage might be saved. Steinberg and collaborators at the University of Pittsburgh recognized that stem cells taken from bone marrow wouldn’t transform into functioning neurons. However, the transplanted cells could release molecules that might rescue neurons that were impaired, but not yet dead.

Brain Surgery
Sonia had surgery to transplant bone marrow stem cells into her brain in late May 2013. The improvement was almost instantaneous. “When I woke up, my speech was strong, I could lift up my feet and keep them in the air, I even raised my right hand,” says Sonia. Though the trial was primarily designed to study the stem cell therapy’s safety, researchers were also interested in its effectiveness.

“Sonia was one of our two remarkable patients who got better the day after surgery and continued to improve throughout the year,” says Steinberg. 18 patients in total were treated in that study.

Although Sonia’s treatment results are still very preliminary, they bode well for a separate CIRM-funded stroke research project also led by Steinberg. In this study, cells grown from embryonic stem cells will be turned into early-stage neuron, or brain, cells and then transplanted into the area of stroke damage. The team has found that transplanting these neural cells into mice or rats after a stroke helps the animals regain strength in their limbs. The team is busy working out the best conditions for growing these neural cells in order to take them into clinical trials.

In the meantime, Sonia continues to improve. “My leg is about 95 percent better and my arm is around 60 percent there,” says Sonia. “My speech isn’t perfect, but I can talk and that’s something I never could have done before the surgery.”

The added function has made a huge difference in her quality of life. She can walk, run, drive a car, call a restaurant to make a dinner reservation—simple things she took for granted before having a stroke. But most importantly, she has confidence in the future.

“Everything is good,” says Sonia, “and it’s only going to get better.”

To learn about CIRM-funded stroke research, visit our Stroke Fact Sheet. Read more about Sonia’s Story of Hope on our website.

Stem cell stories that caught our eye: first iPS clinical trial, cancer metabolism and magnates helping heal hearts

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.

First clinical trial with reprogrammed stem cells.
Today, a Japanese woman became the first patient to be treated with cells derived from reprogrammed iPS-type stem cells. The patient received cells matured into a type of cell damaged in the most common form of blindness, age-related macular degeneration.

Those cells, a normal part of the eye’s retina, were made from stem cells created from a skin sample donated by the patient several months ago. In the intervening time the resulting retinal cells have been tested in mice and monkeys to make sure they will not cause tumors. Because the cells have the same genes as the patient, researchers believe they may not be rejected by the patient’s immune system in the absence of immune suppressive drugs—the beauty of iPS technology.

Right now, that technology is much too cumbersome and time consuming to result in a broadly applicable therapy. But if this first clinical trial proves the immune system get-out-of-jail-free theory, it should intensify efforts to make iPS technology more efficient.

When Japanese authorities gave permission to treat the first patient earlier this week Popular Science provided an easy read version of the story and Nature News provided a bit more detail.

Cancer cells don’t handle their sugar well. Sugar has a bad rep these days. Now, it looks like manipulating sugar metabolism might lead to ways to better treat leukemia and perhaps, make therapies less toxic to normal cells. It turns out cancer cells are much more sensitive to changes in sugar level than normal blood stem cells or the intermediate cells that give rise the various branches of the blood system.

David Scadden at the Harvard Stem Cell Institute has long studied the role of the stem cell's environment in its function.

David Scadden at the Harvard Stem Cell Institute has long studied the role of the stem cell’s environment in its function.

A team led by old friend and colleague at the Harvard Stem Cell Institute, David Scadden, first looked at sugar metabolism in normal blood forming stem cells and their intermediate cells. They found that the parent stem cell and their direct offspring, those intermediate cells, behave differently when faced with various manipulations in sugar level, which makes sense since the intermediate cells are usually much more actively dividing.

But when they manipulated the genes of both types of cells to make them turn cancerous, the cancer cells from both were much more sensitive to changes in sugar metabolism. In a university press release picked up by ScienceCodex David said he hoped to interest drug companies in developing ways to exploit these differences to create better therapies.

Magnets and nanoparticles steer stem cells.
Getting stem cells to where they are needed to make a repair, and keeping them there is a major challenge. A team at Los Angeles’ Cedars-Sinai hospital that we fund (but not for this study) has taken an approach to this problem that is the equivalent of holding your pants up with a double set of button, a belt and suspenders.

Treating damaged hearts in rats they first loaded iron-containing nanoparticles with two types of antibodies, one that recognizes and homes to injured heart tissue and one that attracts healing stem cells. After infusing them into the animal’s blood stream, they placed a magnet over its heart to hold the iron nanoparticles near by. The iron provided the added benefit of letting the team track the cells via magnetic resonance imaging (MRI) to verify they did get to and stay where they were needed.

In a press release from the hospital picked up by ScienceDaily the lead researcher Eduardo Marban said:

“The result is a kind of molecular matchmaking,”

The study was published in Nature Communications and you can read about other work we fund in Marban’s lab trying to figure out once you get the stem cells to the heart exactly how do they create the repair.

Reprogrammed stem cells turned into white blood cells. We have written often about the difficulties of getting stem cells to create fully mature blood cells. Last week we talked about a Wisconsin team breaking the barrier for red blood cells. Now, a team at the Salk Institute is reporting success for white blood cells.

Starting with iPS-type stem cells they got the mature white cells via a two-step process. First they manipulated one gene called Sox2 to get the stem cells to become the right intermediate cells. Then they used a gene-regulating molecule called a micro-RNA to get the middleman cells to mature into white blood cells.

In a press release from the Salk, lead researcher Juan Carlos Izpisua Belmonte noted the clinical importance of the work:

“In terms of potential clinical applications, the hematopoietic system represents one of the most suitable tissues for stem cell-based therapies. . .”

The team published the research in the journal Stem Cells and the web portal BioSpace picked up the release.

Book on early spinal cord injury clinical trial. The title of a book on the first ever clinical trial using cells from embryonic stem cells kind of says it all: Inevitable Collision: The Inspiring Story that Brought Stem Cell Research to Conservative America.

Katy Sharify's experience in the first embryonic stem cell trial is featured in a new book and she discussed it in a video from a CIRM workshop.

Katy Sharify’s experience in the first embryonic stem cell trial is featured in a new book and she discussed it in a video from a CIRM workshop.


The book details the personal stories of the first and fifth patients in the spinal cord injury trial conducted by Geron. That company made the financial decision to end its stem cell product development in favor of its cancer products. But the spinal cord injury trial is now set to restart, modified to treat neck injuries instead of back injuries and at higher doses, through CIRM funding to the company that bought the Geron stem cell business, Asterias.

In a press release from the publisher, the book’s author explained her goal:

“Through this book I hope to bridge the gap between science and religion and raise awareness of the importance and power of stem cell research.”

The fifth patient in the Geron study, Katie Sharify, is featured in our “Stories of Hope” that have filled The Stem Cellar this week.

Don Gibbons

Stories of Hope: Sickle Cell Disease

This week on The Stem Cellar we feature some of our most inspiring patients and patient advocates as they share, in their own words, their Stories of Hope.

Adrienne Shapiro pledged she would give her daughter Marissa the best possible life she could have—wearing herself out if necessary. Her baby girl had sickle cell disease, an inherited disorder in which the body’s oxygen-carrying red blood cells become crescent shaped, sticky, rigid, and prone to clumping—blocking blood flow. Doctors warned Adrienne that Marissa might not live to see her first birthday. When Marissa achieved that milestone, they moved the grim prognosis back a year, and then another year, and then another.

Adrienne has seen first hand how difficult it is to live with this blood disease.

Adrienne has lived through several generations of the inherited blood disease.

Adrienne worked tirelessly to help Marissa. “I was constantly asking questions,” Shapiro says. And for a long time, it worked.

However, things began to unravel for Marissa as she reached adulthood. A standard treatment for sickle cell disease—and the excruciating pain caused by blocked blood vessels—is regular blood transfusions. A transfusion floods the body with healthy, round red blood cells, lowering the proportion of the deformed, ‘sickle-shaped’ cells. But when she was 20, a poorly matched blood transfusion triggered a cascade of immune problems. Later, surgery to remove her gall bladder set off a string of complications and her kidneys shut down temporarily. After that, her immune system couldn’t take any more insults. Now, at age 36, she’s hypersensitive.

“She can’t be transfused. She can’t even have tape next to her skin without her body reacting,” Adrienne said.

Pain control is the newest and continuing nightmare. Adrienne tells harrowing stories of long waits in hospital emergency rooms while her daughter suffers, followed by maddening arguments with staff reluctant to provide enough drugs to control the intense pain when her daughter is finally admitted.

“When she was a kid, everyone wanted to make her feel good,” Adrienne says. “But when we moved from the pediatric side to the adult side, they treated her as a drug seeker and me as an enabler. It’s such a slap in the face.”

For Adrienne, the story is all too familiar. She is the third generation in her family with a sickle cell child. Another daughter, Casey Gibson, does not have the disease but carries the sickle cell mutation, meaning she could pass it to a child if the father also has the trait. One in 500 African Americans has sickle cell disease, as do 1 in 36,000 Hispanic people.

There is only one sure way to stop this story from repeating for generations to come, Adrienne says, and that’s research. She believes stem cell science will be the answer.

“I’ve been waiting for this science to get to the point where it had a bona fide cure, something that worked. Now we’re actually nearing clinical trials. It’s so close.”

In fact a CIRM-funded project led by Don Kohn, M.D. at UCLA aims to start trials in 2014. Kohn and his team intend to remove bone marrow from the patient and fix the genetic defect in the blood-forming stem cells. Then those cells can be reintroduced into the patient to create a new, healthy blood system.

“Stem cells are our only hope,” Adrienne continues, “It’s my true belief that I’m going to be the last woman in my family to have a child with sickle cell disease. Marissa’s going to be the last child to suffer, and Casey is going to be the last one to fear. Stem cells are going to fix this for us and many other families.”

For more information about CIRM-funded sickle cell disease research, visit our Sickle Cell Disease Fact Sheet. You can read more about Adrienne’s Story of Hope on our website.

Stories of Hope: Spinal Cord Injury

This week on The Stem Cellar we feature some of our most inspiring patients and patient advocates as they share, in their own words, their Stories of Hope.

Katie Sharify had six days to decide: would she let her broken body become experimental territory for a revolutionary new approach—even if it was unlikely to do her any good? The question was barely fathomable. She had only just regained consciousness. A week earlier, she had been in a car crash that damaged her spine, leaving her with no sensation from the chest down. In the confusion and emotion of those first few days, the family thought that the treatment would fix Katie’s mangled spinal cord. But that was never the goal. The objective, in fact, was simply to test the safety of the treatment. The misunderstanding – a cure, and then no cure — plunged the 23-year-old from hope to despair. And yet she couldn’t let the idea of this experimental approach go.

Katie never gave up hope that stem cell-based therapies could help her or others like her living with spinal cord injury.

Katie never gave up hope that stem cell-based therapies could help her or others like her living with spinal cord injury.

Just days after learning that she would never walk again, that she would never know when her bladder was full, that she would not feel it if she broke her ankle, she was thinking about the next girl who might lie in this bed with a spinal injury. If Katie walked away from this experimental approach—what would happen to others that came after her?

Her medical team provided a crash course in stem cell therapy to help Katie think things through. In this case the team had taken stem cells obtained from a five-day old embryo and converted them into cells that support communication between the brain and body. Those cells would be transplanted into the injured spines. Earlier experiments in animal models suggested that, once in place, these cells might help regenerate a patient’s own nerve tissue. But before scientists could do the experiment, they needed to make sure the technique they were using was safe by using a small number of cells, too few to likely have any benefit. And that’s why they wanted Katie’s help in this CIRM-funded trial. They explained the risks. They explained that she was unlikely to derive any benefit. They explained that she was just a step along the way. Even so, Katie agreed. She became the fifth patient in what’s called a Phase I trial: part of the long, arduous process required to bring new therapies to patients. Shortly after she was treated the trial stopped enrolling patients for financial reasons.

That was nearly three years ago. Since then, she has been through an intensive physical therapy program to increase her strength. She went back to college. She tried skiing and surfing. She learned how to make life work in this new body. But as she rebuilt her life she wondered if taking part in the clinical trial had truly made a difference.

“I was frustrated at first. I felt hopeless. Why did I even do this? Why did I even bother?” But soon she began to see how small advances were moving the science forward. She learned the steep challenges that await new therapies. Then this year, she discovered that the research she participated in was deemed to be safe and is about to enter its next phase, thanks to a $14.3 million grant from CIRM to Asterias Biotherapeutics. “This has been my wish from day one,” Katie says.

“It gives me so much hope to know there is an organization that cares and wants to push these therapies forward, that wants to find a cure or a treatment,” she says. “I don’t know what I would do if I thought nobody cared, nobody wanted to take any risks, nobody wanted to put any funding into spinal cord injuries.

“I really have to have some ray of hope to hold onto, and for me, CIRM is that ray of hope.”

For more information about CIRM-funded spinal cord injury research, visit our Spinal Cord Injury Fact Sheet. You can read more about Katie’s Story of Hope on our website.

CIRM 2.0: How to Build a Better Stem Cell Agency and Speed up Treatments to Patients

Change is never easy. We all get used to doing things in a certain way and it can sometimes be difficult to realize that the way we have chosen, while it may have worked well at one time is perhaps not the best way to achieve our goals at this time. Well, change is coming to the stem cell agency.

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It’s not surprising that our new President & CEO, C. Randal Mills, Ph.D., would want to introduce some of his own ideas about how best to run the agency in the current moment of stem cell science. After all, it’s those ideas that landed him the job in the first place. Now Randy wants us to develop a clearer focus, one that is more aligned with his 4-point criteria for assessing everything we do.

  1. Will it speed up treatments to patients
  2. Will it increase the likelihood of successful treatments for patients
  3. Does it target an unmet medical need
  4. Is it efficient.

That new focus begins with re-imagining how we can be most effective in the way we fund research. Right now we put out what’s called an RFA or Request for Application, telling people who have promising projects in a particular area of stem cell research to submit an application and if they are successful they’ll get up to $20 million, depending on the kind of project.

The problem is, we often have long gaps between each round of funding and so a company or institution with a promising therapy will sometimes have to wait as much as a couple of years before they can apply again. If they do wait and are successful in their application it could still be another year or two before they are able to gain actual funding and begin a clinical trial. But when lives are at stake, you can’t afford to wait that long. So we’re looking at ways of speeding things up, making it easier for the best science to get the funds needed when they are needed.

At our Board meeting yesterday Randy outlined some broad concepts about what he wants to do and how it can be done. It’s part of his vision for the agency, a new focus that he is calling CIRM 2.0 (with acknowledgments to Dr. Paul Knoepfler who coined the term earlier this year)

As with any simple idea it’s really complicated. We need to achieve greater speed, to streamline the way we do things, without sacrificing the quality of the review process because we need to ensure that we only fund the best science.

In the months to come, as the precise details about these proposed changes are fine tuned, the Board will hear in greater detail how this will work and, as always, it will be up to them to decide if they think it’s a good idea.

Either way it will start a conversation about how we can become more efficient and more effective at living up to our mission, of accelerating therapies that target patients with unmet medical needs. And that always has to be a good thing.

For more details about the other big events at yesterday’s Board meeting, including awarding $16 million to ViaCyte to help it advance its promising therapy for type 1 diabetes, you can read the news release posted on our website.

Stories of Hope: Leukemia

This week on The Stem Cellar we feature some of our most inspiring patients and patient advocates as they share, in their own words, their Stories of Hope.

Stem cells create life. But if things go wrong, they can also threaten it. Theresa Blanda found that out the hard way. Fortunately for her, CIRM-funded research helped her fight this threat, and get her life back.

Theresa's battle with leukemia took a happier turn after entering into a stem cell-based clinical trial.

Theresa’s battle with leukemia took a happier turn after entering into a stem cell-based clinical trial.

In the first few days of human development embryonic stem cells are a blank slate; they don’t yet have a special, defined role, but have potential. The potential to turn into the cells that make up our kidneys, heart, brain, every other organ and every tissue in our body. Because of this flexibility, stem cells have shown great promise as a way to regenerate dead, diseased or injured tissue to treat many life-threatening or chronic conditions.

But some studies have suggested a secret, darker side to stem cells—so-called cancer stem cells. Like their embryonic cousins, these cells have the ability to both self-renew— to divide and make more copies of themselves – and specialize into other cell types. Many researchers believe they can serve as a reservoir for cancer, constantly reinvigorating tumors, helping them spread throughout the body. To complicate matters, these slow-growing cells are often impervious to cancer therapies, enabling them to survive chemotherapy.

For Theresa Blanda, cancer stem cells were dragging her down a slippery slope towards disease and possibly death. In 2003, she was diagnosed with polycythemia vera (CV), which causes the body to produce too many red blood cells. As sometimes happens with CV patients, her body began producing too many white blood cells as well. Eventually, she developed an even more serious condition, myelofibrosis, a form of bone marrow scarring that results in an enlarged spleen, bone pain, knee swelling and other debilitating symptoms.

“You couldn’t even breathe my way or I’d bruise,” says Theresa. “I didn’t think I was going to make it.”

Her doctors wanted to do a bone marrow transplant, but were having difficulty finding the right donor. “Finally, I just asked if there was some kind of clinical trial that could help me,” says Theresa.

Fortunately, there was.

The Root Cause
At UC San Diego’s Moores Cancer Center, Catriona Jamieson, M.D., Ph.D., had made a discovery that would have a big impact on Theresa’s health. In research funded in part by CIRM, Jamieson found a key mutation in blood-forming stem cells. Specifically, a mutation in a gene called JAK2 was being passed on to Theresa’s entire blood system, causing CV and myelofibrosis. Without effective treatment, her condition could have progressed into acute myeloid leukemia, a blood cancer with a very poor survival rate.

“These malignant stem cells create an inhospitable environment for regular stem cells, suppressing normal blood formation,” says Jamieson. “We needed to get rid of these mutated stem cells so the normal ones could breathe a sigh of relief.”

The answer was a JAK2 inhibitor being developed by San Diego-based TargeGen. Though the trial had already started, they made room for Theresa and the results were amazing. Within weeks, her discomfort had faded, her spleen had returned to normal and she was back at work.

“In a month or two I was feeling pretty good,” says Theresa. “I could climb stairs and the swelling in my knee had gone down.”

She continued on the drug for five years but safety issues forced the trial to be suspended. But the work continues. With continued support from CIRM, Jamieson and others are investigating new JAK2 inhibitors, and other alternatives, to help myelofibrosis patients.

“Because of CIRM funding, we’ve managed to develop a number of agents that have gone into clinical trials,” says Jamieson. “That means patients have lived to hold their grandchildren, attend their mom’s hundredth birthday party and live fruitful lives.”

For more information about CIRM-funded leukemia research, visit our Leukemia Fact Sheet. You can read more about Theresa’s Story of Hope on our website.

September ICOC Boarding Meeting Begins Soon

The September ICOC Boarding Meeting begins this morning in Berkeley, CA.

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

For those not able to attend, feel free to dial in:

Dial in Infomation:

United States: (800) 230-1093
Access Code: 334835

WEB MEETING ACCESS INFORMATION:
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* https://www.webmeeting.att.com
* Meeting Number(s): 5114686455
* PARTICIPANT CODE: 313650

WEBEX LINK:
1. Go to https://cirm.webex.com/cirm/onstage/g.php?MTID=ef6aa60e45eb581e0e24ea4d2…
2. Click “Join Now”.

We will be providing a summary of the meeting’s highlights after the meeting—so stay tuned!