Funding stem cell research targeting a rare and life-threatening disease in children

If you have never heard of cystinosis you should consider yourself fortunate. It’s a rare condition caused by an inherited genetic mutation. It hits early and it hits hard. Children with cystinosis are usually diagnosed before age 2 and are in end-stage kidney failure by the time they are 9. If that’s not bad enough … Continue reading Funding stem cell research targeting a rare and life-threatening disease in children

Asterias’ stem cell clinical trial shows encouraging results for spinal cord injury patients

When researchers are carrying out a clinical trial they have two goals: first, show that it is safe (the old “do no harm” maxim) and second, show it works. One without the other doesn’t do anyone any good in the long run. A few weeks ago Asterias Biotherapeutics showed that their CIRM-funded stem cell therapy … Continue reading Asterias’ stem cell clinical trial shows encouraging results for spinal cord injury patients

Salk scientists explain why brain cells are genetically diverse

I’ve always wondered why some sets of genetically identical twins become not so identical later in life. Sometimes they differ in appearance. Other times, one twin is healthy while the other is plagued with a serious disease. These differences can be explained by exposure to different environmental factors over time, but there could also be … Continue reading Salk scientists explain why brain cells are genetically diverse

Making a deposit in the Bank: using stem cells from children with rare diseases to find new treatments

Part of The Stem Cellar series on ten years of iPS cells For Chris Waters, the motivation behind her move from big pharmaceutical companies and biotech to starting a non-profit organization focused on rare diseases in children is simple: “What’s most important is empowering patient families and helping them accelerate research to the clinical solutions … Continue reading Making a deposit in the Bank: using stem cells from children with rare diseases to find new treatments

Stem cell stories that caught our eye: improving heart care, fixing sickle cell disease, stem cells & sugar

Here are some stem cell stories that caught our eye this past week. Some are groundbreaking science, others are of personal interest to us, and still others are just fun. Using “disease in a dish” model to improve heart care Medications we take to improve our quality of life might actually be putting our lives … Continue reading Stem cell stories that caught our eye: improving heart care, fixing sickle cell disease, stem cells & sugar

HOPE for patients with Duchenne Muscular Dystrophy-associated heart disease

It’s an exciting week for CIRM-funded clinical trials. Yesterday, we blogged about a young man named Kris Boesen who is responding positively to a stem cell therapy in a Phase 1/2a CIRM-funded clinical trial for spinal cord injury run by Asterias Biotherapeutics. Paralyzed from the chest down after a terrible car accident, Kris now has regained some use … Continue reading HOPE for patients with Duchenne Muscular Dystrophy-associated heart disease

Young man with spinal cord injury regains use of hands and arms after stem cell therapy

Hope is such a fragile thing. We cling to it in bad times. It offers us a sense that we can bear whatever hardships we are facing today, and that tomorrow will be better. Kris Boesen knows all about holding on to hope during bad times. On March 6th of this year he was left … Continue reading Young man with spinal cord injury regains use of hands and arms after stem cell therapy

Stem cell stories that caught our eye: functioning liver tissue, making new bone, stem cells and mental health

Here are some stem cell stories that caught our eye this past week. Some are groundbreaking science, others are of personal interest to us, and still others are just fun. Functioning liver tissue. Scientists are looking to stem cells as a potential alternative treatment to liver transplantation for patients with end-stage liver disease. Efforts are … Continue reading Stem cell stories that caught our eye: functioning liver tissue, making new bone, stem cells and mental health

Seeing is believing: how some scientists – including two funded by CIRM – are working to help the blind see

“A pale hue”. For most of us that is a simple description, an observation about color. For Kristin Macdonald it’s a glimpse of the future. In some ways it’s a miracle. Kristin lost her sight to retinitis pigmentosa (RP). For many years she was virtually blind. But now, thanks to a clinical trial funded by … Continue reading Seeing is believing: how some scientists – including two funded by CIRM – are working to help the blind see

A look back at the last year – but with our eyes firmly on the future

Better. With that single word Randy Mills, our President and CEO, starts and ends his letter in our 2015 Annual Report and lays out the simple principle that guides the way we work at CIRM. Better. But better what? “Better infrastructure to translate early stage ideas into groundbreaking clinical trials. Better regulatory practices to advance … Continue reading A look back at the last year – but with our eyes firmly on the future