Video describes life with Huntington's disease, hopes for stem cell therapies

The University of California, Irvine has begun posting video interviews with their stem cell institute director Peter Donovan (You can see all of those videos here.). Of the eight interviews, including ones with former CIRM board chair Robert Klein and leading UCI stem cell researchers, one of the most moving is with Huntington's Disease advocate Frances Saldana.


Saldana lost her husband, one daughter and two of her husband's brothers to the disease. Her two remaining kids have advanced Huntington's disease. She now has two grandchildren, both of whom have a 50/50 chance of having inherited the disease. In the UCI video Saldana says:

"This is not an illness that is going to go away once you lose your loved one. It's going to carry on through the generations. When they do inherit Huntington's Disease they will lose the ability to walk, to talk and to swallow and they will start to experience atrophy of the muscles which causes spasticity and a lot of pain. It's very painful."

Saldana spoke to the CIRM governing board about life with Huntington's disease back in 2007. Saldana is one of many of the passionate advocates for Huntington's disease who have spoken with the agency over the years and who have been tireless supporters of stem cell research.

"What I would hope for, if not a cure, is a treatment to alleviate human suffering for our families. I don't think stem cell research will provide a cure for my two other children, but I have two grandchildren and now they are vibrant and beautiful and I hope they have a full life ahead of them, but we need to have something for them if and when they might be carrying that mutant protein."

The agency currently funds five awards with a focus on the disease. You see a list of these awards, learn more about Huntington's disease, and watch a video CIRM produced about the disease on our disease fact sheet.

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Stem Cell Awareness Day introduces high school students to heros

Stem Cell Awareness Day on Wednesday brought scientists to close to 60 classrooms to deliver lectures on stem cell science and inspired events throughout the world, some of which are ongoing throughout October. (You can see a full list of events here.)

The Daily Pilot had a nice story on an event at University of California, Irvine, which brought more than 100 high school students into UCI's Sue and Bill Gross Hall. CIRM contributed $27.2 million to the construction of the building. In addition to stem cell labs, the building houses equipment to help people with spinal cord injuries regain strength. The story quotes one of the students who attended:

"It's nice to see our tax dollars go to something that actually helps people," said Brittany Jackson, a Trabuco Hills High School senior. "There's so much technology. You don't realize there's so much that goes into helping those less fortunate."

The story goes on:

Standing between a computer monitor showing color dyed stem cells under a microscope and a refrigerator growing stem cells, Christina Tu talked about why she and researchers do what they do.

One scientist is working with a bioengineer to generate cells that can help a baby recover from heart failure at birth. Tu, the stem cell core facility coordinator, moved into stem cell research from another field because of the possibilities.

"They must have this passion," she said. "A lot of times, outcome is negative. You have to overcome that to see what you want to accomplish."

Nancy Luke, a lawyer stricken with multiple sclerosis, encouraged the students to move science forward.

"What I have is the hope that [what UCI is studying] can cure MS," she said. "Researchers rock. And you guys will be the heroes."

That's what Stem Cell Awareness Day is all about: Exposing people to those heroes, and encouraging students to become heroes themselves.

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Alzheimer's leader discusses stem cell progress

Tom Vasich at the University of California Irvine did a Q&A with CIRM grantee Frank LaFerla in advance of the September 30 Southern California Alzheimer’s Disease Research Conference. La Ferla and his colleagues have been working on stem cell-based therapies for Alzheimer's disease.

In a question about the public health impact of Alzheimer's disease LaFerla said:

It’ll be enormous, especially locally. California has the nation’s largest baby boom population; Orange County by itself ranks fifth. Alzheimer’s is going to hit us hard, because age is the most significant risk factor for the disease. One of every 20 people over 65 will be affected by dementia, and eventually half of those over 85 will suffer from Alzheimer’s. This is going to put an amazing strain on our healthcare system and on families.

He went on to talk about his own research, funded by CIRM:

We’ve had a lot of success with animal models showing that neural stem cells can reverse Alzheimer’s-like cognitive deficits. We’ve progressed to creating a population of human neural stem cells that will be the basis of clinical trials on patients. We’re still in the early stages, and we’re fortunate to have received a grant from the California Institute for Regenerative Medicine that is supporting our work.

We had the pleasure of talking to LaFerla about this week a few years ago. In this video, he describes the work and the importance of finding a therapy for the disease.



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Stem cells improve brain function after radiation therapy

CIRM grantees at University of California Irvine have used human neural stem cells to help alleviate brain damage that occurs after radiation to treat brain tumors.

Radiation can be an effective way of treating tumors in the brain, but the radiation also kills surrounding healthy tissue in addition to the destroying the tumor. Even if the cancer is eliminated the person can be left with debilitating learning and memory loss. A press release from UCI quotes senior author on the work Charles Limoli, who has a CIRM SEED award to carry out this work:

“In almost every instance, people experience severe cognitive impairment that’s progressive and debilitating,” Limoli said. “Pediatric cancer patients can experience a drop of up to three IQ points per year.”

Limoli and his team wanted to know if the brain's stem cells could repair that damage. They injected human neural stem cells into the brains of rats that had undergone radiation treatment. Those stem cells migrated to the damaged part of the brain and matured into nerves and the brain's support cells. The release quotes Limoli:

“This research suggests that stem cell therapies may one day be implemented in the clinic to provide relief to patients suffering from cognitive impairments incurred as a result of their cancer treatments,” Limoli said. “While much work remains, a clinical trial analyzing the safety of such approaches may be possible within a few years, most likely with patients afflicted with glioblastoma multiforme, a particularly aggressive and deadly form of brain cancer.”

If their work is successful, this technique could help people live normal lives after being treated for brain cancers. That would be good news for individuals, their caregivers and for the state, which loses tax income when people are unable to work or must decrease work to care for family members.

Cancer Research, July 15, 2011
CIRM Funding: Charles Limoli (RS1-00413-1)

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Clinical Trial of Hope at Stanford -- guest blogger Roman Reed

Under the direction of Dr. Gary Steinberg, an advance long considered impossible is moving forward today: Stanford announced yesterday that it will participate in Geron's human clinical safety trials for a novel treatment for spinal cord injury. These are safety trials to be sure and not efficacy trials, more tests will need to be run, but this is already farther along than ever before.

To understand what this means to me, jump back in time…

Eight years ago, I held in my hand a paralyzed rat.

As I sat there in a wheelchair paralyzed from a spinal cord injury, holding this rat in wonderment, awe and jealousy, my mind barely comprehended that this rat was actually moving. His formerly paralyzed legs pushed strongly against my hands, trying to get away from my grasp. (A video of a rat from that test is available here.) His coat full and healthy, meaning he had no pain. This rat was the tangible realization of a seemingly impossible dream: to cure paralysis.

This was the pioneer rat of what would become the world’s first human embryonic stem cell clinical trials: the “Geron Trials”.

That rat walked because of Dr. Hans Keirstead at the University of California, Irvine Reeve-Irvine Research Center, whose brilliance is matched only by his courage and determination. Nearly everyone denied him funding initially, because his experiment was ‘too far reaching”. But he would not quit.

This rat was also walking again, because California’s Roman Reed Spinal Cord Injury Research Act of 1999 provided Keirstead the initial funding for this Pioneering Research! (CIRM has since funded Keirstead's follow-up research with a Comprehensive award.)

The first human clinical trials for hESCR treatment were accomplished because of Geron Corp. Geron is literally footing the bill: risking their financial backbone to advance the field. It is to be hoped they will be richly rewarded for such fiscal courage.

These trials of hope through stem cells are being overseen by Stanford’s brilliant Dr. Gary Steinberg and Dr. Steve McKenna of San Jose Valley Medical Center.

Stanford is committed to the cause of cure; and there too, I can speak from personal experience—because Dr. Steinberg is the founder and director of the Stanford Partnership for Spinal Cord Injury and Repair.

One day, I hope to fulfill the late great Christopher “Superman” Reeve’s prediction, that:

“One day, Roman and I will stand up from our wheelchairs, and walk away from them forever.”

Cure did not come in time for our great champion, but I believe he is smiling down on us this day, as Stanford, California, and the world take one leap toward the fulfillment of his dream.

Be mindful, this is just the beginning…



- Roman Reed is the namesake of the Roman Reed Spinal Cord Injury Research Act passed in California. As a result, more than $12.5 million in state funds has been awarded to scientists conducting research in spinal cord regeneration. An additional $50 million has been leveraged from outside sources.

Here is additional information about CIRM's spinal cord injury funding.

Growing space for California stem cell research

On left and right, Berkeley Stem Cell Center co-directors
David Schaffer and Randy Shekman, and center,
Mary West, manager of the new lab. (Photo by Jan Ambrosini)

Berkeley is the most recent institution to open new stem cell space funded by CIRM. Their CIRM-funded stem cell facility, which had its opening Oct. 5, is also a core facility for QB3, a bay area biotech incubator. David Shaffer, co-director of the Berkeley Stem Cell Center, said of the facility:

“The new facility will serve as a central resource to greatly enhance stem cell research amongst Berkeley and QB3 investigators, as well as collaborators at Lawrence Berkeley National Lab and Children’s Hospital Oakland Research Institute.”

To date, University of California campuses at Irvine and Davis have both opened their new stem call buildings amidst much fanfare. By the end of October, UCLA, University of Southern California, and Stanford will all have cut their respective ribbons.

These buildings are, to a one, beautiful, gleaming, well equipped centers for cutting edge research. But they are more than that. They are also a safe haven for stem cell research, protected from the ups and downs of federal funding. CIRM first dreamed up and approved funding for these stem cell buildings when President Bush was in office and most stem cell research had to occur in isolation from the microscopes, the pipettes, the refrigerators, the reagents, and the latex gloves most labs purchase with their NIH funds. The research had to take place in space and on lab benches supported only through private or state dollars.

That space was hard to come by, making the early days of stem cell research a considerable challenge. Take Susan Fisher at UCSF who lost her stem cell lines to a power outage while working in a converted dentist office in San Francisco in order to put distance between her cells and federal dollars. (Here's a video about Fisher's experience)

In the past year President Obama opened up federal funding for more stem cell research, but now recent events put that funding back in question. During this time of uncertainty, it’s reassuring to know that so many institutions in California have space where their work toward new therapies can continue uninterrupted by political turmoil.




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Stem cell awareness, poetry, and the blues

Stem Cell Awareness Day is underway, which is both true and practically rhymes, making it a fitting opening for a post about poems.

We held the second annual poetry contest this year in conjunction with events and seminars being held worldwide. Entries were judged by Don Reed, sponsor of the Roman Reed Spinal Cord Injury Research Act and stem cell blogger, and Margaret Hermes, MA in poetry from Boston University, a PhD from Indiana University and a practicing poet and teacher.

From among 18 entries hailing from six countries they selected two winners and two finalists. You can read those poems on our official announcement.

But it’s not all serious around here when it comes to stem cells. Among my favorite poems are these two non-entries, which weren’t in the official contest. One is from Tom Vasich, assistant director of communications at UC Irvine. The other is from Chris Stiehl, who works with CIRM to reach out to patient groups throughout California.

Just like those pluripotent blues
By Tom Vasich

I shot some stem cells in my vein
With hopes that they would reach my brain
And trigger many proteins there
To help grow back my missing hair

The stem cell source was highly pure
And guaranteed to spark a cure
Their lineage was unapproved
Which the DOJ so doth moved

“Illegally pluripotent”
Said the letter I was sent
“Are those cells now in your melon
Take them out or you’re a felon”

Too late, the cells had taken hold
To which my mien grew thick and bold
What should have been a source of pride
Under a cap I now must hide

So how I fell into this mix
I place the blame on politics
Should I vote for a Democrat
Who’ll free me to remove my hat?

Or should I move to Mexico
Where I can let my new hair grow?
By choosing how I want to live
I now become a fugitive

Oh, somewhere in this world so bright
People have the legal right
For stem cell usage as they choose
I’ve got those pluripotent blues


Stem Cell Research at the Bat
By Chris Stiehl

An adaptation of “Casey at the Bat”
by Ernest Lawrence Thayer

The Outlook wasn't brilliant for the Stem Cell nine that day:
The score stood four to two, with but one inning more to play.
And then when Cooney died at first, and Barrows did the same,
A sickly silence fell upon the patrons of the game.

A straggling few got up to go in deep despair. The rest
Clung to that hope which springs eternal in the human breast;
They thought, if only Research could get but a whack at that -
We'd put up $3 billion, now, with Research at the bat.

But McCulloch preceded Research, as did also Jimmy Till,
The former was a quondam doctor and the latter was too young still;
So upon that stricken multitude grim melancholy sat,
For there seemed but little chance of Research's getting to the bat.

But McCullough let drive a single, to the wonderment of all,
And Till, the bio-physicist, tore the cover off the ball;
And when their Nature piece was published, and the men saw what had occurred,
There was Jimmy safe at second and McCullough a-hugging third.

Then from 5,000 throats and more there rose a lusty yell;
Prop 71 rumbled through California’s central valley, it rattled in the dell;
It knocked upon the Sierras and recoiled upon the flat,
For Research, mighty Research, was advancing to the bat.

There was ease in Research's manner as he stepped into his place;
There was hope in Research's studies and renewal on Research's face.
And when, responding to the cheers, he lightly doffed his hat,
No stranger in the crowd could doubt 'twas Research at the bat.

Ten thousand eyes were on him as he injected marrow into mice;
Five thousand tongues applauded when stem cells grew like rice.
Then while the litigious pitcher ground the ball into his hip,
Defiance gleamed in Research's eye, a sneer curled Research's lip.

And now the leather-covered sphere came hurtling through the air,
Research stood a-watching it; with scientific curiosity, not despair.
Close by the sturdy batsman the ball unheeded sped-
"That ain't my style," said Research. "Strike one," the judge had said.

From the benches, from hope-filled people, there went up a muffled roar,
People awaiting cures and treatments, like waves upon a distant shore.
"Kill him! Kill the judge!" shouted someone on the stand;
And its likely they'd a-killed him had not Research raised his hand.

With a smile of Christian charity great Research's visage shone;
He stilled the rising tumult; CIRM bade the game go on;
Research signaled to the pitcher, and once more the spheroid flew;
But Research still ignored it, and the judge said, "Strike two."

"Fraud!" cried the labs and scientists, and echo answered fraud;
But one scornful look from Research and the audience was awed.
They saw his face grow stern and cold, they saw his muscles strain,
And they knew that Research wouldn't let that funding ball go by again.

The sneer is gone from Research's lip, to the chucker he shouts “Curtains!”;
His firmness of conviction makes the outcome predictable, certain.
And now the pitcher holds the ball, and now he lets it go,
And now the air is shattered by the force of Research's blow.

Somewhere in this great land of ours it’s cloudy, there is no sun;

And somewhere cytoplasts are no longer studied, investigators have no fun;

And somewhere over silent labs there hangs a heavy pall;
In California, Stem Cell hearts are happy now--for Research hit the ball.

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Update on stem cell treatments for spinal cord injury

A nod to Paul Knoepfler at UC Davis, who has posted a blog entry about the Geron trial for spinal cord injury. It provides a nice summary of the science behind the trial, and a reminder of why patients might be hard to recruit. He refers to Michael Martinez, a jockey who recently sustained a severe spinal cord injury, and who was rejected for participation in Geron’s trial developed from human embryonic stem cells. As Knoepfler points out:

An important element of this treatment, as highlighted by the recent case with Martinez, is that the injury has to be of a certain kind to be treatable using Geron's approach. If too severe, the animal models would suggest the treatment won't help. In addition, the treatment must be given with 1-2 weeks of the injury.

We recently released a video about stem cell therapies for spinal cord injury, featuring CIRM board member Oswald Steward who directs the Reeve-Irvine Research Center at UC Irvine. CIRM funds several spinal cord injury research projects at that center, including some with Hans Keirstead who first developed the therapy being tested by Geron.

While talking with Dr. Steward for that video he discussed hopes for a therapy that would be effective after that first 1-2 week window. His lab recently showed that in rodents, neurons could be nudged to regrow over the site of an older injury (here's the UCI press release). Another group at UC Irvine found that human neural stem cells restored some mobility to mice that were in the chronic stages of spinal cord injury (here's our blog entry on that work).

You can learn more about CIRM work toward a stem cell based therapy for spinal cord injury on our Spinal Cord Injury Stem Cell Fact Sheet.

Neural stem cells help mice with chronic spinal cord injury walk again

Human neural stem cells transplanted
into mice grew into neural tissue
cells, such as oligodendrocytes.
Brian Cummings / UCI

A study published last week by CIRM grantees at UC Irvine gives a big ray of hope to people living with spinal cord injuries. Brian Cummings and Aileen Anderson showed that human neural stem cells could restore some mobility to mice with induced spinal cord injuries. According to a press release from UC Irvine:

The UCI study, led by Aileen Anderson and Brian Cummings of the Sue and Bill Gross Stem Cell Research Center, is significant because the therapy can restore mobility during the later chronic phase, the period after spinal cord injury in which inflammation has stabilized and recovery has reached a plateau. There are no drug treatments to help restore function in such cases.

Other stem cell strategies for treating spinal cord injury, including the trial by Menlo Park, CA-based Geron, focus on the period of time immediately following injury.

In this latest work, three months after the stem cell treatment the mice showed consistent improvements compared to untreated mice.

The release quotes Aileen Anderson as saying:

“This study builds on the extensive work we previously published in the acute phase of injury and offers additional hope to those who are paralyzed or have impaired motor function.”

This seems like a good time to quote Roman Reed, the namesake of the Roman Reed Spinal Cord Injury Research Act and founder of the Roman Reed Foundation: “Turning stem cells into cures.” This paper is one more step toward that goal that we all share.

PLoS ONE, August, 19, 2010
CIRM funding: Desiree Salazar (T1-00008)

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Mysteries of stem cell migration revealed

CIRM-funded Researchers at the University of California, Irvine published an interesting paper this week that helps explain one mystery — how do transplanted stem cells go to the right place? This is an important issue for diseases such as multiple sclerosis, where transplanted stem cells would have to navigate to the damaged nerves.

In a press release, senior author Thomas Lane (shown in photo) said:

"Previously, we've seen that adult neural stem cells injected into the spinal column knew, amazingly, exactly where to go. We wanted to find what directed them to the right injury spots."

What the team found is that in mice with an induced form of MS, transplanted neural stem cells responded to signals being sent by inflammatory cells at the site of the damage. The neural stem cells responded to those signals by migrating to the right place and maturing into a type of nerve cell called an oligodendracyte, which could help heel the disease.

According to the press release, three weeks after the initial treatment, 90 percent of the cells had grown into fully formed oligodendrocytes.

Proceedings of the National Academy of Sciences, Online Edition, June 1
CIRM Funding: Kevin Carbajal and Christopher Shaumburg (T1-00008)

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UC Irvine Opens the Sue & Bill Gross Hall

On Friday, May 14 UC Irvine held the grand opening of their newly constructed Sue &Bill Gross Hall. According to a story on the University's web site:

The $80-million, 100,000-square-foot building was designed to facilitate contact between patients in the first-floor clinic and rehabilitation center and stem cell researchers in first-, second- and third-floor labs.

(Image: Hans Keirstead greets Bill Gross at the opening ceremonies. Courtesy of Daniel A. Anderson / UCI)

This was one of 12 facilities throughout California that received a CIRM Major Facilities Award to construct space for stem cell research. UC Davis opened their facility in March. Although Irvine held the second opening, theirs was the first for a newly constructed building.

The 12 major facilities received $272 million from CIRM, with private donations and institutional investments bringing the project totals to more than $1 billion. Sue and Bill Gross made a $10 million gift to the Irvine facility. An independent review of the impact of this investment for the state economy last year by The Analysis Group suggested that the projects would create 13,000 job years of employment and $100 million in tax revenue.

UC Irvine leveraged their CIRM dollars beyond their original goals -- they managed to construct a fourth floor for the same cost as the original three floor proposal.

An LA Times story quotes UC Irvine Stem Cell Research Center director Peter Donovan as saying:

"Whatever we achieve here, it is your legacy as much as ours," he said. "The use of stem cells can revolutionize the treatment of human diseases and injuries."

Scientists are due to move into the building over the next few weeks.



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Embryonic stem cells repair radiation damage in mice

Radiation can effectively destroy brain tumor cells – but at a cost. While killing the tumor cells the treatment also damages normal cells in portions of the brain involved in learning and memory, leaving people with varying levels of impairment. New work by researchers at the University of California, Irvine suggests that human embryonic stem cells are able to ameliorate radiation-induced normal tissue damage.

The group, led by CIRM SEED grantee Charles Limoli, irradiated the heads of rats then transplanted human embryonic stem cells into the brain. In a memory test four months after the radiation, transplanted rats performed as well as rats that had never been irradiated. Rats that received radiation but no transplanted stem cells showed a significant decline in learning and memory.

The transplanted cells had migrated through the brain and matured into a variety of brain cells. The cells did not form any tumors (at least by 4 months) – something scientists are careful to watch for in transplanted stem cells.

In a press release by UCI, Limoli said:

"With further research, stem cells may one day be used to manage a variety of adverse conditions associated with radiotherapy."

Proceedings of the National Academy of Science: November 10, 2009
CIRM funding: Charles Limoli (RS1-00413-1), Peter Donovan (RC1-00110-1)

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Neural stem cells reverse Alzheimer's symptoms in mice

Researchers at the University of California, Irvine have reversed Alzheimer’s-like symptoms in a mouse model of the disease with injections of neural stem cells. The mice used in this study mimicked the human disease, showing learning and memory defects and accumulating both beta-amyloid plaques and tau protein tangles within the brain, the two hallmark pathologies of the disease.  Mice that received injections of mouse neural stem cells performed significantly better in memory tests than mice that received control injections. The stem cells did not replace cells lost to the disease. Instead, the injected cells secreted a protein known as brain-derived neurotrophic factor (BDNF), that helped nourish the surviving neurons, encouraging those cells to grow more fibers and form more connections. The injected cells did not reduce the plaques or tangles. Current therapies for Alzheimer’s disease can only reduce the severity of symptoms or slow progression. To date, this is only the second potential treatment shown to actually improve memory in mice with advanced plaque and tangle pathology.



Proceedings of the National Academy of Sciences, August 11, 2009
CIRM funding: Frank LaFerla (RS1-00247-1), Matthew Blurton-Jones (T1-00008)

Related Information: UCI Press Release, University of California, Irvine, LaFerla bio

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