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.

UC Davis scientist on a quest for cures in the cleanest of labs

Gerhard Bauer in the UC Davis GMP facility

One of the real thrills of working at CIRM is talking to the researchers who are so excited about finding new therapies. As part of our lunchtime talk series, today we heard from Gerhard Bauer of UC Davis. The only thing more exciting to Bauer than new therapies is the thought of having those therapies come out of his beautiful new GMP lab.

A GMP (Good Manufacturing Practice) lab is a clean facility that can be used to process the cells and other products that might one day go in people. When you see photos of scientists in white suits looking through microscopes, they’re likely in a GMP facility. If the lab sparkles so brightly you need sunglasses to look at the photos, it’s probably one of Bauer’s six GMP labs he’s built since moving from Austria to the U.S. in the 1980s to work on HIV/AIDS. He’s like the MacGyver of GMP labs. Nothing already manufactured was quite perfect enough for his facility, so in his spare time, you know, when he wasn’t running a lab and picking paint colors for the building, he also designed better GMP equipment.

Do I sound smitten? My apologies. I do aim for professional disinterest, but I am only human and I also really, really want to see therapies for some of the diseases they are tackling in that facility. It’s inspiring to see such enthusiasm in the people who are working toward those cures. Among the many diseases under investigation in the facility (Huntington’s disease, peripheral artery disease, bone fractures, liver disease) Bauer is part of the Stanford Disease Team working toward a therapy for the horrific childhood skin disorder epidermolysis bullosa. He and other members of that team recently spoke about the work at a governing board meeting. Videos of those talks are available here, but be warned that the disease is awful and the images are graphic. Personally, I can’t look.

One fun thing we learned is that Bauer has been taking on CIRM Bridges interns and training them in GMP procedures (here's a video about the Bridges program if you aren't familiar with it). He’s hired one, and has another working in the lab now. That’s exactly what we were hoping for in the Bridges program. Undergrad or masters students are learning stem cell science and getting trained for jobs in California’s expanding stem cell biology sector.

The lunch talks are a great opportunity for CIRM staff to hear about how those disease teams are progressing and to understand the challenges. Getting to a cure isn’t easy. One thing we all learned from Bauer’s talk is that whatever therapy the team comes up with, it’s going to be absolutely, totally, completely GMP certified. And clean? It’s going to be clean. Because when Bauer wasn’t dreaming up better lab equipment he was also certifying the cleaning protocols.

Here’s a video we made last year about the GMP facility:



- A.A.

Stem cells treat life-threatening skin condition

This week researchers at the University of Minnesota published a paper showing that stem cells from the bone marrow can help kids with a blistering skin condition called epidermolysis bullosa. The disease is horrible. Lacking a protein to anchor skin in place, the children's blister at the slightest touch -- on their skin, in their throat, inside their eyelids, and anywhere else skin forms.

The group gave the kids a bone marrow transplant, replacing their own blood system with cells that make the form of collagen lacking in kids with the disease. It worked. In a press release, John Wagner, M.D., director of pediatric blood and marrow transplantation and clinical director of the Stem Cell Institute, said:

“To understand this achievement, you have to understand how horrible this disease actually is. From the moment of birth, these children develop blisters from the slightest trauma which eventually scar. They live lives of chronic pain, preventing any chance for a normal life. My hope is to do something that might change the natural history of this disease and enhance the quality of life of these kids.”

A Canadian CBS news story quotes Pediatric dermatologist Dr. Elena Pope, medical director of the EB clinic at Toronto's Hospital for Sick Children, as saying:

"It's extremely, extremely exciting for us who are working in this area to actually see some steps forward."

CIRM funds a disease team headed by Alfred Lane at Stanford Univerversity, who is also working toward a stem cell-based therapy for the disease. His team is creating reprogrammed iPS cells from the children's skin, inserting a good copy of the mutated gene, and transplanting the resulting skin cells back onto the children. 

CIRM's epidermolysis bullosa disease team: Anthony Oro,
Gerhard Bauer, Alfred Lane, Marius Wernig

Whichever approach is successful long-term, it's nice to see progress being made for this truly horrible disease. 

A.A.