ISSCR public symposium: Hope, passion and restraint for stem cell clinical trials

The annual meeting of the International Society for Stem Cell Research is Mecca for the hardcore stem cell research community. The next four days will be filled with intense and detailed discussions of all of the molecular details of how stem cells work--details we need to understand to move stem cells into the clinic effectively and safely.

But last night, as always, the meeting officially started with a symposium designed specifically for the public. For the third year I have had the pleasure of being on the planning committee for this event and this year we decided the field was ready to discuss clinical trials. We chose the title “The Stem Cell Promise: Moving to the Clinic” and decided to alternate brief talks by patient advocates and scientists/clinicians. Here is a list of speakers from the event.

What a rapt full house of attendees heard was an impassioned evening of hope and excitement balanced nicely with restraint and patient persistence. Early on California’s spinal cord injury advocate Roman Reed reminded the audience that the Geron trial, the first using cells derived from embryonic stem cells, is really about safety, not someone getting up out of a wheel chair. But at the same time he urged everyone in the audience to become an active advocate; to write to their legislatures to make sure this work gets funded: “Take a stand for research; take a stand for stem cells, so that one day we all can stand.”

Michael Fehling, a neurosurgeon at Toronto West Hospital, acknowledged that if scientists are going to design the perfect trial there's more they need to know about how neuronal progenitor cells will behave at the site of an injury. But he said, “You don’t have to have all the answers,” adding that most scientists believe our knowledge level is ready to begin clinical work. He admonished those in the field to make sure they learn from clinical trials and any information they can glean from how the cells perform in patients to direct a new wave of basic research to learn more of the answers needed to design a better trail. “We need reverse translational research.”

D.G.

CIRM sends Patient advocates to international stem cell meeting

Today stem cell scientists and patient advocates are descending on Toronto for the annual meeting of the International Society for Stem Cell Research. Among those registering and preparing for three days of science is Don Reed, who is one of 20 patient advocates who received a CIRM stipend to attend. 

On his blog today, Reed writes about his trip to Toronto and the important role of patient advocates in promoting science:

Patient advocates like my son Roman Reed are the emotional muscle behind research for cure. It is not easy for a paralyzed person to travel, but he will be there, listening and learning, and speaking.

Because there will be funding fights ahead, both nationally, and in every state.

When a politician says, “We can’t afford to fund the research”—someone must be there to say: “We cannot afford NOT to fund the research—and here’s why.” That someone is us. If it is just the scientists talking, it is all too easy for politicians to ignore them.

Think of he numbers. There are only a handful of top research scientists. Politically, they are negligible. If they only talk to each other, they might as well pack up their test tubes and go home, because they will never get public funding.

But there are millions of patients and family– patient advocates. Working together, we cannot be ignored.

From its inception, CIRM has recognized the importance of patient advocate voices in scientific decision-making, including ten such advocates on our governing board (bios of all board members are available here). Jeff Sheehy, patient advocate board member for HIV/AIDS blogged about his role on our board:

I serve on the governing board as a patient advocate for HIV/AIDS, and in that role I along with the other patient advocate board members have been able to directly influence the direction of the agency. Our voice has helped shape decisions regarding CIRM policies and funding. As [vice chair Duane] Roth writes, patient advocates can grasp some of the most complex and thorny policy and scientific issues and “tip the scales” in the direction of sound public policy that seeks prudently to accelerate progress towards cures.

ISSCR is kicking off the meeting tonight with a public symposium consisting of patient advocates and scientists discussing progress being made in spinal cord injury (featuring Reed's son Roman Reed), multiple sclerosis and blood disorders. Hopefully those advocate voices will help drive home the importance of continued funding for stem cell research.

A.A.

Guest blogger Roman Reed: spinal cord injury stem cell trials get $25 million boost from CIRM

From the editor: Yesterday the CIRM governing board approved a $25 million loan to help fund a trial testing the use of an embryonic stem cell-derived therapy for spinal cord injury. Here's our press release. Our guest blogger Roman Reed was injured in a football accident and has since fought tirelessly for spinal cord injury research.

Yesterday's announcement that CIRM will help fund the Geron spinal cord injury Human Trials ensures that paralysis cure will have it's day!

It was my honor to be the inspiration of the Roman Reed Spinal Cord Injury Research Act in 1999, which provided the original funding for Hans Keirstead's research which is being tested in these trials, the first in the world.

However, the expense of the trials might conceivably have exhausted Geron's financial resources, narrowing the scope of the trials. This would have been devastating, not only to our hopes for paralysis cure but also for the entire field. The world is watching. Had the trials been too limited for lack of funding, it might have cast a shadow of doubt on its outcome-- instead, the trials go forward, strongly.

Geron's nine year struggle to build a mountain of preliminary data (so the FDA could approve the human trials) was an heroic effort, on which so much depended. With the leadership of Geron, the courageous biomed company came through for California and our country, and the world-- and so did the California stem cell program.

When the oversight committee announced its decision yesterday, it authorized a $25 million dollar loan to Geron to assist in the clinical trials. This was a careful loan, milestone driven, with each installment given only on the successful completion of predetermined goals.

I join the patient advocate community across the globe in expressing absolute full support of CIRM's descision to provide funding for America's first hESC derived Human Clinical Trials.

As a Patient and Patient advocate, I am overjoyed that Prop.71 took a stand for the Geron trials, the most important experiment in the world for the fields of spinal cord injury, neuro-regenerative medicine and stem cell research.

Truly, the Geron Trials are setting the pace of spinal cord injury. Paralysis affects 650,000 Californians and 1.9 million Americans, with an additional 5.6 million Americans who suffer from all forms of paralysis. (Here's information about CIRM's funding for spinal cord injury stem cell research.)

This epic step was accomplished through partnership and collaboration through a new age continum of support beginning with State funding from Roman Reed Spinal Cord Injury Research Act, Education at the University of California, Irvine, private industry support from Geron and voter-approved CIRM/State funds.

In a truly historic partnership for stem cell research and cure, state funding from what has been called “Roman's Law” gave Hans Keirstead the seed money to achieve empirical evidence and proof of principle. Hans then sold his pioneering technique to Geron, and advanced biotechnology farther by founding California Stem Cell with the proceeds.

With admirable courage and determination, Geron pushed this science all the way to FDA approval to become the world's first embryonic stem cell derived human clinical trial.

Now our beloved state agency, the California Institute for Regenerative Medicine will provide funding to bring about a full and complete Human Clinical Trial!!

The Keirstead/Geron/CIRM Trials advances the entire field of stem cell research.

Today's vote of support is a truly historic milestone in medicine.

Ladies and Gentlemen welcome to the beginning of personalized stem cell medicine and cure!

Thank you Prop.71 for Taking A Stand-- So One Day-Everybody Can!

Here's Roman Reed discussing stem cell-based therapies for spinal cord injury:

Full Vision Cure? Full Stem Cell Research Required

By Don C. Reed

The world is delighted that patients with injured eyes in Italy regained their sight, thanks to the use of stem cells transplanted from their own good eyes. This is a wonderful victory for Dr. Grazziella Pellegrini, the University of Modena, and everyone involved. They are doubly to be congratulated because they are working with one hand tied behind their backs. In Italy, it is illegal to fund embryonic stem cell research. So they did what they could with what was allowed.

To give sight to someone who has been blinded in one eye is wonderful indeed.

But to claim this success invalidates the need for embryonic cells, which some have done, would be wrong.

First, the improvement only works on injured eyes, not those damaged by disease or genetic problems. This disqualifies the overwhelming majority of blindness sufferers, such as people with macular degeneration or retinitis pigmentosa. Also, not every injury can be treated. Everything depends on the patient having enough good tissue from the eye to draw cells from. It would be useless to a person born blind.

It has also been reported that another scientist was not able to duplicate Dr. Pellegrini’s success. According to a story in Bloomberg News Dr. Ivan Schwab, an ophthalmology professor and stem cell researcher at UC Davis:

"…has treated patients in clinical trials with a procedure based on Pellegrini’s work. While his patients improved for a time, the benefits didn’t endure."

Adult stem cell work has been studied for more than half a century; it would be shocking indeed if there were no successes. This particular study began in 1998—the same year human embryonic stem cells were first isolated.

As the father of a paralyzed young man (Roman Reed, inspiration for a California law which has funded embryonic stem cell research) I follow the research closely. This fall, we hope, an embryonic stem cell treatment will go to human trials.

Adult stem cell research is a useful tool. But it is only one tool, not the toolbox.

Patient advocates, like the vast majority of scientists, support full stem cell research, not any single kind to the exclusion of all others.

Don C. Reed
Citizen-sponsor, Roman Reed Spinal Cord Injury Research Act
Stemcellbattles.wordpress.com