Geron trial -- baby steps toward a cure

Today Geron announced that the first patient had been treated in their groundbreaking trial based on human embryonic stem cells.

At first blush: Woo hoo!! This is the first test of technology that holds hope for millions of people living with spinal cord injury and other diseases.

But it’s important to remember that this first trial is a very, very early step. Any potential therapy goes through three rounds of human trials. The first, phase I, trial is intended only to verify that the potential therapy is safe. This trial can establish dosing and begin to monitor side effects of the drug or treatment. In some cases a phase I trial will also begin to reveal whether the drug or therapy has signs of effectiveness, but that’s not the primary role of the trial.

After the small phase I trial comes a slightly larger phase II trial. It confirms safety and also begins to look directly at whether the drug or therapy is effective.

It isn’t until some years later that a large phase III trial confirms whether the drug or therapy is effective compared to standard therapy.

Many potential therapies fail after the phase I trial, even after years of positive results in animals. Humans aren’t mice (or other laboratory animals) and scientists can’t ever know what side effects will turn up in their human patients.

That’s why it is important to temper that initial “woo hoo” with a bit of patience. This could be one of those trials that fails phase I. Or phase II. Or phase III. But as many years of biomedical research have shown, for every failed trial there are also a number of successful trials. Whatever the results of this first phase I trial, it’s the beginning of a new era that will one day result in the first successful therapy based on human embryonic stem cells. That day will have scientists and patients alike whooping for joy.

Here's patient advocate Roman Reed talking about what a stem cell therapy for spinal cord injury means to him:

Patient advocates vital to stem cell research progress

Nature Medicine carried a piece Friday by CIRM governing board member Jeff Sheehy, writing about the importance of having a patient advocate voice in biomedical research. Sheehy, who is living with HIV, is a long-time advocate for HIV/AIDS research. He has been on the CIRM board since the beginning in November 2004, and is a vocal participant in CIRM working groups including the group that makes research funding recommendations to the full board (the Grants Working Group), for which he is vice-chair.

Sheehy writes:

The presence of vocal, engaged patient advocates has added an indispensable dimension to the proceedings. In measuring research quality, advocates tend to focus on a project's ability to benefit people—not just drive scientific curiosity—which keeps even basic biomedical research grounded in its ability to produce concrete health benefits.

CIRM’s governing board includes 12 patient advocates representing HIV/AIDS, MS, diabetes (type 1 and type 2), heart disease, spinal cord injury, cancer, Alzheimer’s disease, Parkinson’s disease and autism. Sheehy goes on to say:

... CIRM is made up of patient advocates from a wide spectrum of diseases and conditions who work together to advance therapies across the board. And contrary to critics' assertions, these advocates have not narrowly focused on their own diseases, but have uniformly advocated for the best approaches for moving basic research towards the clinic. They support each other.

Patient advocates serve a powerful role even if they aren’t directly involved in funding decisions. Don Reed has been a vocal supporter of stem cell research since his son Roman Reed suffered a spinal cord injury. He is sponsor of the Roman Reed Spinal Cord Injury Research Act that funds spinal cord research in California, founder and co-chair of Californians for Cures and blogger on his own site www.stemcellbattles.org and for the Huffington Post.

In a recent Huffington Post blog entry about the World Stem Cell Summit he wrote about the importance of patient advocates staying involved in stem cell research at a political level, in order to maintain the U.S. leadership in stem cell research:

But unless we in the patient advocacy community can encourage Congress to pass a stem cell research protection act the dream will have been stolen.

CIRM came about in part because of the passion and support of patient advocates like Sheehy and Reed, and they continue to be a crucial part of the success of CIRM and of the progress made in stem cell research.

Here's Sheehy advocating for a stem cell therapy for HIV/AIDS. CIRM has funded two disease teams (City of Hope and UCLA) focusing on developing therapies for the disease.

New poll finds widespread support for stem cell research

A new Harris Interactive/HealthDay poll came to the not-so-startling conclusion that most Americans support using embryos left over from in vitro fertilization for research purposes, including human embryonic stem cell research.

According to the report, Humphrey Taylor, chairman of the Harris Poll that conducted to online survey said:

"Even among Catholics and born-again Christians, relatively few people believe that stem cell research should be forbidden because it is unethical or immoral."

The process of in vitro fertilization (IVF) can result in ten or more embryos. If a woman gets pregnant quickly, then the remaining embryos stay in a freezer until the couple decides they no longer want to pay for storage. At that time, the couple can either put the embryos up for adoption (an option rarely chosen), discard the embryos — destroying them — or donate them to research. In order to donate those embryos to research rather than discarding them, a couple must give full informed consent that they understand how the resulting stem cells will be used. The NIH will only approve lines for funding that have clear consent and were derived from excess IVF embryos.

One telling finding from the survey was:

Seventy-three percent (versus 72 percent in 2005) believe that stem cell research should be allowed "as long as the parents of the embryo give their permission, and the embryo would otherwise be destroyed."

A recent post on the Baker Institute blog had a nice analysis of the relationship between IVF and stem cell research. In it, the authors write:

With such wide support for IVF, it is remarkable that there is still such a public outcry in the United States over the use of leftover embryos for scientific research. From a moral standpoint, one can argue that in both cases an embryo is destroyed and the destruction of an embryo is always wrong. But isn't it worse to discarding an embryo than to use the embryo for research and development of treatments for currently incurable diseases? Embryonic stem cell research has the potential to bring relief to people suffering from diseases such as Parkinson's disease, muscular dystrophy and diabetes, as well as catastrophic injuries including those to the spinal cord.

Federal agencies such as the NIH can only fund research with already created embryonic stem cell lines. They cannot fund the creation of new lines from discarded IVF embryos. This work is often funded by private agencies or organizations such as CIRM. Here’s a list of all CIRM-funded grants for the creation of new lines.

A.A.

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.

A.A.

Nobel-winning IVF work laid groundwork for stem cell research

On Monday the Nobel Prize in Physiology or Medicine went to Robert Edwards for his efforts to make in vitro fertilization a reality. The Nobel Prize-winning discovery not only allowed millions of couples to start families, it opened up the field of stem cell research.

According to the Nobel Prize press release:

Approximately four million individuals have so far been born following IVF. Many of them are now adult and some have already become parents. A new field of medicine has emerged, with Robert Edwards leading the process all the way from the fundamental discoveries to the current, successful IVF therapy. His contributions represent a milestone in the development of modern medicine.

Alan Trouson, CIRM president, was in the thick of the early IVF work. He led the team that produced Australia's first IVF baby -- the third in the world. He went on to develop ways of using fertility drugs and freezing embryos to increase the success rate of IVF. He was also among the first to realize the possibilities of taking stem cells from the unused embryos to create cells with the potential to become every cell in the body.

As a sign of how far the IVF field has come, on the same day that Edwards won his Nobel Prize researchers at Stanford University published work showing a technique for selecting which embryos have a 90% chance of resulting in a healthy blastocyst -- a stage of development at about 5 days old, when the embryo is implanted into the mother's womb.

According to a Stanford press release, about 2/3 of embryos created through IVF normally die. Improving those odds could greatly increase the rate of successful IVF pregnancies. The work was led by Renee Reijo Pera, who is also a CIRM grantee (Comprehensive Award and New Cell Lines Award). The release goes on the say:

“It completely surprised me that we could predict embryonic fate so well and so early,” said Reijo Pera. If an embryo’s values fell within certain windows of time for the three predictive parameters, that embryo was more than 90 percent likely to go on to develop successfully into a blastocyst.

Being able to predict which embryos will survive greatly improves a woman's chance of getting pregnant. Riejo Pera is quoted in the release as saying:

"Women, their families and their physicians want to increase the chances of having one healthy baby and avoid high-risk pregnancies, miscarriages or other adverse maternal and fetal outcomes. It’s truly a women’s health issue that affects the broader family.”

Reijo Pera stressed the importance of the work in understanding the earliest stages of human development, where many developmental anomalies are though to originate. It could also be important for the creation of new embryonic stem cell lines, which come from embryos that are discarded from IVF clinics.

Running, cycling and swimming for a cure

Any excuse to link stem cell research and a favorite activity...

In the top 10 list of our most popular entries, spinal cord injury ranked among the topics of most interest to readers. And for good reason. It's the first condition to be approved for an embryonic stem cell trial. It's also a terrible condition in dire need of a therapy.

Those readers interested in helping find a cure for spinal cord injury might want to check out the Team Reeve Marathon season. Short of toiling in the lab, this a great way to move the research forward. The Reeve Foundation will help train runners in return for their fund-raising efforts.

If running for a cure sounds appealing but your heart lies with an ailment other than spinal cord injury, you can check out your favorite disease charity. Many raise money by training volunteers for marathons, triathlons, cycling events and hikes.

The Sanford-Burnham Medical Research Institute also recently posted information about Team Sanford-Burnham in which fund-raisers run with the scientists whose work they are supporting.

As runner Judy Wade, a Team Stanford-Burnham enthusiast, says about marathons she's run:

"You have to remind yourself why you’re doing this, the cause. You feel really good about yourself when you’re finished.”

(She's right!)

While out there raising funds you can also raise awareness of stem cell research. Here are some answers to the most common misconceptions about stem cell research.

A.A.

What's hot at 100?

This blog is turning 100 -- posts that is. On the eve of our centenial, here's a top 10 of what's hot:

1: Geron to begin stem cell trial for spinal cord injury
2: UC Irvine Opens the Sue & Bill Gross Hall
3: iPS cells and embryonic stem cells -- similar but not the same
4: CIRM grantee Joanna Wysocka wins Outstanding Young Investigator Award
5: Stem cells treat Parkinson's disease in rats
6: Update on stem cell treatments for spinal cord injury
7: Neural stem cells help mice with chronic spinal cord injury walk again
8: iPS and embryonic stem cells -- similar but not the same
9: The competition that isn't: Adult vs. embryonic stem cells
10: What the embryonic stem cell research ban means to California researchers

Of these, I'll admit a preference for #4. It's exciting to see enthusiastic young women scientists being recognized for their work. Wysocka, an assistant professor at Stanford University, credits her CIRM SEED grant (summary here) for pulling her into work with human embryonic stem cells. She's since gone on to receive a New Faculty Award (summary here) and was given among the highest scores for a recently submitted NIH grant for human embryonic stem cell work to address a devastating developmental disorder. That grant has been caught up in the recent funding battle (mentioned in this blog entry).

A.A.