Stories of hope and scientific progress in the CIRM Annual Report

This past year, CIRM scientists made significant progress toward new therapies. Several disease teams showed that their approach is likely to be effective and they are likely to be reaching clinical trials on target in the next few years. Other groups made progress in understanding how embryonic stem cells form adult cells and tissues and mimicking disease in a laboratory dish. CIRM formed new global partnerships to leverage world-wide stem cell expertise and seven of our major facilities opened their doors to stem cell scientists.

I know all this because it's in the just-published annual report. You can read the stories here, along with letters from Governing Board chair Robert Klein and President Alan Trounson.

My favorite part of every annual report is the focus on patient advocates and their stories. This year, we feature people living with (or caring for) those with amyotrophic lateral sclerosis (Lou Gehrig's disease), Huntington's disease, HIV/AIDS, epidermolysis bullosa and stroke, as well as a follow-up story on a woman living with a form of pre-leukemia who we featured in last year's report. She's doing well on a new therapy that came out of CIRM-funded research.

The patients and their stories keep CIRM scientists' focus where it belongs -- on developing new therapies for chronic disease and injury.

A.A.

Stem cell progress on brain awareness week

This week marks Brain Awareness Week, with events worldwide to bring people up to speed on brain research. I went to the cool search tool on the Dana Foundation web site and found that several CIRM grantees are hosting events this week. That makes sense, given that roughly a quarter of our funding goes to neuronal diseases. (You can see charts of CIRM stem cell research funding allocations here. The charts are slightly out of date — stay tuned for some updates in the next month.)

Brain diseases are seen as a big challenge for stem cell therapies, in part because the brain itself is such a complex web of neurons. Simply replacing a few lost neurons won't necessarily replicate the lost connections. We have a story discussing some of those issues and describint innovative approaches CIRM grantees are taking to developing new cures for brain diseases.

The good news is that some CIRM grantees are learning that stem cells can be coaxed to form the support cells in the brain that nourish neurons. These support cells could be what provide a therapy for diseases such as ALS, MS, stroke and spinal cord injury. Other grantees are using stem cells in the lab to test new drugs for Parkinson's disease.

A group at UC Davis is attempting to use the body's own mesenchymal stem cells to preserve unaffected neurons in people with Huntington's disease. This technique won't bring back lost cells, but saving additional cells from dying off could prevent some of the terrible side effects of the disease.

Another team of CIRM grantees at UC Irvine found that at least in rodents, stem cells were able to repair some memory loss due to Alzheimer's disease. This work is a long way from treating humans, but still provides hope for people who have lost loved ones to this devastating disease. Here's a video we produced about that work:



We've produced several other videos about CIRM's brain related research:

• Fred H. Gage talks about using embryonic stem cells to model neuronal disease
• Spinal Cord Injury: Progress and Promise in Stem Cell Research
• Huntington's Disease: Progress and Promise in Stem Cell Research
• Parkinson's Disease: Progress and Promise in Stem Cell Research
• Spotlight on ALS seminar
• Spotlight on Huntington's disease seminar
• Spotlight on Alzheimer's disease seminar
• Spotlight on Batten disease seminar
• Spotlight on Parkinson's disease seminar

 - A.A.

A salute to caregivers from Leeza Gibbons

At CIRM we want nothing more than to find cures for the diseases that afflict people around the world. Until that day comes, many of the diseases we hope to cure — Alzheimer’s disease, Parkinson’s disease, spinal cord injury, ALS, to name a few — place an incredible burden on the caregivers in addition to the patients themselves.

Caregivers have no greater advocate than Leeza Gibbons, who also serves on the CIRM governing board. Her online caregiver support network, Leeza’s Place, is celebrating National Family Caregiver Month with events throughout the country to both support and educate caregivers. A list of those events is available here.

On her blog, Leeza wrote:

At Leeza’s Place, we know the job of being a caregiver is both frustrating and fantastic; isolating and invigorating; a blessing and a burden. Those husbands and wives, daughters, sons and friends who care for someone they love often find they are stronger than they ever imagined they could be.

As we celebrate those courageous spirits, we offer respect and gratitude to all who are on the front lines of humanity. A month recognizing caregivers is just a starting point for us to create more national awareness, better support, respite and care. At Leeza’s Place, this month (and every month) we offer hugs of support, approval and a blanket of love!

To hear more about Leeza’s Place and Leeza’s caregiving mantra of “Take Your Oxygen First” visit their online media page here.

Here is Dan Desmond, who has ALS, paying a touching tribute to his wife and caregiver, Gloria Desmond.



A.A.

Embryonic Stem Cells Generate Model for ALS

Researchers at the Salk Institute for Biological Sciences have grown embryonic stem cells into the motor neurons and support cells that underlie amyotrophic lateral sclerosis (ALS). Also known as Lou Gherig’s Disease, ALS has no cure and no effective treatment. In this disease, the motor neurons slowly degenerate leaving a person paralyzed. Why the neurons die is not known, however the support cells called astrocytes have long appeared to play a role. Now researchers have coaxed embryonic stem cells to form the motor neurons and astrocytes in a lab dish to better understand their relationship in ALS. What they learned is that astrocytes containing a mutation associated with ALS killed off the neighboring motor neurons. This mutation is in a gene that makes a protein whose normal role is to protect the body from damaging oxygen free radicals. When the group grew these same cells in the presence of a powerful anti-oxidant, the motor neurons survived. In addition to understanding the biology of ALS, the group thinks they could use this system to screen drugs that may be able to treat ALS.



Cell Stem Cell: December 4, 2008
CIRM funding: Fred H. Gage (RC1-00115)

Related Information:Press release, Salk Institute for Biological Sciences, Gage  bio