The Stem Cell Bank is open for business

Creating a stem cell bank

Creating a stem cell bank

When you go to a bank and withdraw money you know that the notes you get are all going to look the same and do the same job, namely allow you to buy things. But when you get stem cells for research that’s not necessarily the case. Stem cells bought from different laboratories don’t always look exactly the same or perform the same in research studies.

That’s why CIRM has teamed up with the Coriell Institute and Cellular Dynamics International (CDI) to open what will be the world’s largest publically available stem cell bank. It officially opened today. In September the Bank will have 300 cell lines available for purchase but plans to increase that to 750 by February 2016.

300 lines but no waiting

Now, even if you are not in the market for stem cells this bank could have a big impact on your life because it creates an invaluable resource for researchers looking into the causes of, and potential therapies for, 11 different diseases including autism, epilepsy and other childhood neurological disorders, blinding eye diseases, heart, lung and liver diseases, and Alzheimer’s disease.

The goal of the Bank – which is located at the Buck Institute for Research on Aging in Novato, California – is to collect blood or tissue samples from up to 3,000 volunteer donors. Some of those donors have particular disorders – such as Alzheimer’s – and some are healthy. Those samples will then be turned into high quality iPSCs or induced pluripotent stem cells.

Now, iPSC lines are particularly useful for research because they can be turned into any type of cell in the body such as a brain cell or liver cell. And, because the cells are genetically identical to the people who donated the samples scientists can use the cells to determine how, for example, a brain cell from someone with autism differs from a normal brain cell. That can enable them to study how diseases develop and progress, and also to test new drugs or treatments against defects observed in those cells to see which, if any, might offer some benefits.

Power of iPSCs

In a news release Kaz Hirao, Chairman and CEO of CDI, says these could be game changers:

“iPSCs are proving to be powerful tools for disease modeling, drug discovery and the development of cell therapies, capturing human disease and individual genetic variability in ways that are not possible with other cellular models.”

Equally important is that researchers in different parts of the world will be able to compare their findings because they are using the same cell lines. Right now many researchers use cell lines from different sources so even though they are theoretically the same type of tissue, in practice they often produce very different results.

Improving consistency

CIRM Board Chair, Jonathan Thomas, said he hopes the Bank will lead to greater consistency in results.

“We believe the Bank will be an extraordinarily important resource in helping advance the use of stem cell tools for the study of diseases and finding new ways to treat them. While many stem cell efforts in the past have provided badly needed new tools for studying rare genetic diseases, this Bank represents both rare and common diseases that afflict many Californians. Stem cell technology offers a critical new approach toward developing new treatments and cures for those diseases as well.”

Most banks are focused on enriching your monetary account. This bank hopes to enrich people’s lives, by providing the research tools needed to unlock the secrets of different diseases, and pave the way for new treatments.

For more information on how to buy a cell line go to or email

Sonic Hedgehog provides pathway to fight blood cancers

Dr. Catriona Jamieson: Photo courtesy Moores Cancer Center, UCSD

Dr. Catriona Jamieson:
Photo courtesy Moores Cancer Center, UCSD

For a lot of people Sonic Hedgehog is a video game. But for stem cell researcher Dr. Catriona Jamieson it is a signaling pathway in the body that offers a way to tackle and defeat some deadly blood cancers.

Dr. Jamieson – a researcher at the University of California, San Diego (UCSD) – has a paper published online today in The Lancet Haematology that highlights the safety and dosing levels for a new drug to treat a variety of blood cancers. CIRM funding helped Dr. Jamieson develop this work.

The drug targets cancer stem cells, the kind of cell that is believed to be able to lie dormant and evade anti-cancer therapies before springing back into action, causing a recurrence of the cancer. The drug coaxes the cancer stem cells out of their hiding space in the bone marrow and gets them to move into the blood stream where they can be destroyed by chemotherapy.

In a news release Dr. Jamieson says the drug – known by the catchy name of PF-04449913 – uses the sonic Hedgehog signaling pathway, an important regulator of the way we develop, to attack the cancer:

“This drug gets that unwanted house guest to leave and never come back. It’s a significant step forward in treating people with refractory or resistant myeloid leukemia, myelodysplastic syndrome and myelofibrosis. It’s a bonus that the drug can be administered as easily as an aspirin, in a single, daily oral tablet.”

The goal of this first-in-human study was to test the drug for safety; so 47 adults with blood and marrow cancer were given daily doses of the drug for up to 28 days. Those who were able to tolerate the dosage, without experiencing any serious side effects, were then given a higher dose for the next 28 days. Those who experienced problems were taken off the therapy.

Of the 47 people who started the trial in 2010, 28 experienced side effects. However, only three of those were severe. The drug showed signs of clinical activity – meaning it seemed to have an impact on the disease – in 23 people, almost half of those enrolled in the study.

Because of that initial promise it is now being tested in five different Phase 2 clinical trials. Dr. Jamieson says three of those trials are at UCSD:

“Our hope is that this drug will enable more effective treatment to begin earlier and that with earlier intervention, we can alter the course of disease and remove the need for, or improve the chances of success with, bone marrow transplantation. It’s all about reducing the burden of disease by intervening early.”

Stem Cell Stories that Caught our Eye: What’s the Best Way to Treat Deadly Cancer, Destroying Red Blood Cells’ Barricade, Profile of CIRM Scientist Denis Evseenko

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.

Stem Cells vs. Drugs for Treating Deadly Cancer. When dealing with a potentially deadly form of cancer, choosing the right treatment is critical. But what if that treatment also poses risks, especially for older patients? Could advances in drug development render risky treatments, such as transplants, obsolete?

That was the focus of a pair of studies published this week in the New England Journal of Medicine, where a joint Israeli-Italian research team investigated the comparative benefits of two different treatments for a form of cancer called multiple myeloma.

Multiple myeloma attacks the body’s white blood cells. While rare, it is one of the most deadly forms of cancer—more than half of those diagnosed with the disease do not survive five years after being diagnosed. The standard form of treatment is usually a stem cell transplant, but with newer and better drugs coming on the market, could they render transplants unnecessary?

In the twin studies, the research team divided multiple myeloma patients into two groups. One received a combination of stem cell transplant and chemotherapy, while the other received a combination of drugs including melphalan, prednisone and lenalidmomide. After tracking these patients over a period of four years, the research team saw a clear advantage for those patients that had received the transplant-chemotherapy treatment combination.

To read more about these twin studies check out recent coverage in NewsMaxHealth.

Breaking Blood Cells’ Barricade. The process whereby stem cells mature into red blood cells is, unfortunately, not as fast as scientists would like. In fact, there is a naturally occurring barrier that keeps the production relatively slow. In a healthy person this is not necessarily a problem, but for someone in desperate need of red blood cells—it can prove to be very dangerous.

Luckily, scientists at the University of Wisconsin-Madison have found a way to break through this barrier by switching off two key proteins. Once firmly in the ‘off’ position, the team could boost the production of red blood cells.

These findings, published in the journal Blood, are critical in the context of disease anemia, where the patient’s red blood cell count is low. They also may lead to easier methods of stocking blood banks.

Read more about this exciting discovery at HealthCanal.

CIRM Scientist on the Front Lines of Cancer. Finally, HealthCanal has an enlightening profile of Dr. Denis Evseenko, a stem cell scientist and CIRM grantee from the University of California, Los Angeles (UCLA).

Born in Russia, the profile highlights Evseenko’s passion for studying embryonic stem cells—and their potential for curing currently incurable diseases. As he explains in the article:

“I had a noble vision to develop progressive therapies for the patient. It was a very practical vision too, because I realized how limited therapeutic opportunities could be for the basic scientist, and I had seen many great potential discoveries die out before they ever reached the clinic. Could I help to create the bridge between stem cells, research and actual therapeutics?”

Upon arriving at UCLA, Evseenko knew he wanted to focus this passion into the study of degenerative diseases and diseases related to aging, such as cancer. His bold vision of bridging the gap between basic and translational research has earned him support not only from CIRM, but also the National Institutes of Health and the US Department of Defense, among others. Says Evseenko:

“It’s my hope that we can translate the research we do and discoveries we make here to the clinic to directly impact patient care.”

Creaky Cell Machinery Affects the Aging Immune System, CIRM-Funded Study Finds

Why do our immune systems weaken over time? Why are people over the age of 60 more susceptible to life-threatening infections and many forms of cancer? There’s no one answer to these questions—but scientists at the University of California, San Francisco (UCSF), have uncovered an important mechanism behind this phenomenon.

Reporting in the latest issue of the journal Nature, UCSF’s Dr. Emmanuelle Passegué and her team describe how blood and immune cells must be continually replenished over the lifetime of an organism. As that organism ages the complex cellular machinery that churns out new cells begins to falter. And when that happens, the body can become more susceptible to deadly infections, such as pneumonia.

As Passegué so definitively put it in a UCSF news release:

“We have found the cellular mechanism responsible for the inability of blood-forming cells to maintain blood production over time in an old organism, and have identified molecular defects that could be restored for rejuvenation therapies.”

The research team, which examined this mechanism in old mice, focused their efforts on hematopoetic stem cells—a type of stem cell that is responsible for producing new blood and immune cells. These stem cells are present throughout an organism’s lifetime, regularly dividing to preserve their own numbers.

Molecular tags of DNA damage are highlighted in green in blood-forming stem cells. [Credit: UCSF]

Molecular tags of DNA damage are highlighted in green in blood-forming stem cells. [Credit: UCSF]

But in an aging organism, these cells’ ability to generate new copies is not as good as it used to be. When the research team dug deeper they found a key bit of cellular machinery, called the mini-chromosome maintenance helicase, breaks down. When that happens, the DNA inside the cell can’t replicate itself properly—and the newly generated cell is not running on all cylinders.

One of the first things that these old stem cells lose as a result is their ability to make B cells. B cells, a key component of the immune system, normally make antibodies that fight infection. As B cell numbers dwindle in an aging organism, so too does their ability to fight infection. As a result the organism’s risk for contracting dangerous illnesses skyrockets.

This research, which was funded in part by CIRM, not only informs what goes wrong in an aging organism at the molecular level, but also points to new targets that could keep these stem cells functioning at full capacity, helping promote so-called ‘healthy aging.’ As Passegué added:

“Everybody talks about healthier aging. The decline of stem-cell function is a big part of age-related problems. Achieving longer lives relies in part on achieving a better understanding of why stem cells are not able to maintain optimal functioning.”

Stem cell stories that caught our eye: young blood, cord blood, and blood cancers

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.

Pinning down young blood’s rejuvenating power. A trio of studies in the past week provided more evidence that giving older mice the blood of younger mice can rejuvenate some aspect of their function to a younger state. This has been shown for some years with various tissues, such as CIRM grantee Irina Conboy’s work at UC Berkeley looking at revitalizing older muscle. The recent studies all showed improvement in various aspects of brain function. Most important, the studies started to uncover some reasons for why the young blood could be beneficial when introduced into older animals. Conboy has suggested that one thing it does is provide an environment that lets muscle stem cells do a better job. The three current teams’ work suggests there are probably many factors at play in the young blood. The Boston Globe focused on the work of the Harvard team but puts all three projects in perspective. The San Francisco Business Times focused on the Stanford work and includes an extensive Q&A with the lead researcher.

Expanding cord blood could expand uses. The blood-forming stem cells found in umbilical cord blood have proven extremely valuable as a part of therapy for certain blood cancers. The problem with them is there just are not enough of them in a single cord to treat anyone large than a nine or 10-year-old child. That means when an adult needing a blood stem cell transplant can’t find a matching adult donor and has to resort to cord blood, they receive cells from two cords doubling the chance for severe side effect. Now, a team at Mount Sinai School of Medicine in New York has found a way to get cord blood stem cells to proliferate in the lab in greater numbers than anyone has in the past. They accomplished the trick by resetting the genetic switches that turn genes on and off. Genetic Engineering and Biotechnology News ran a description of the work.

Deciding on banking cord blood. I handle many desperate patient calls here at CIRM, and occasionally get a call from a parent wanting advice about banking their soon-to-be-born child’s cord blood. While I never offer specific advice, I do try to talk through a few factual issues for them to consider, such as the limitation on the number of cells in the cord discussed above. In this Huffington Post blog a mom walks through her family’s decision process for two different pregnancies that came to different, though pretty logical, conclusions for each. She raises many important considerations. However, note that toward the end when she talks about research “advancing” for several diseases, for all those diseases many more years of research will be needed before cord blood therapies become a reality if they ever do.

Blood cancers vs. blood stem cells. One of the difficulties of treating blood cancers is you often end up killing off the vital blood forming stem cells at the same time you destroy the cancer cells. A team at Dartmouth has developed a method to make it easier to distinguish between the stem cells and the cancer cells. Knowing this difference should help researchers find more specific cancer therapies that can destroy the cancer without harming the needed stem cells. Science Codex posted the press release from the medical school. You can read about projects CIRM funds in the field on our leukemia fact sheet.

Don Gibbons