While we are here at ISSCR 2019 hearing various scientists talk about their work, we realize that there are various breakthroughs in stem cell research in a wide variety of different fields going on every day. It is wonderful to see how scientists are hard at work in developing the latest science and pushing innovation. … Continue reading Stories of the week – preterm birth and mice with a human immune system
Rare Diseases
“A new awakening”: One patient advocate’s fight for her daughters life
We often talk about the important role that patient advocates play in helping advance research. That was demonstrated in a powerful way last week when the CIRM Board approved almost $12 million to fund a clinical trial targeting a rare childhood disorder called cystinosis. The award, to Stephanie Cherqui and her team at UC San … Continue reading “A new awakening”: One patient advocate’s fight for her daughters life
CIRM-Funded Clinical Trials Targeting Blood and Immune Disorders
This blog is part of our Month of CIRM series, which features our Agency’s progress towards achieving our mission to accelerate stem cell treatments to patients with unmet medical needs. This week, we’re highlighting CIRM-funded clinical trials to address the growing interest in our rapidly expanding clinical portfolio. Today we are featuring trials in our blood and … Continue reading CIRM-Funded Clinical Trials Targeting Blood and Immune Disorders
Bridging the divide: stem cell students helping families with rare diseases become partners in research
Sometimes it’s the simplest things that make the biggest impact. For example, introducing a scientist to a patient can help them drive stem cell research forward faster than either one could do on their own. Want proof? This year, students in CIRM’s Bridges to Stem Cell Research and Therapy program at California State University (CSU) … Continue reading Bridging the divide: stem cell students helping families with rare diseases become partners in research
jCyte starts second phase of stem cell clinical trial targeting vision loss
Studies show that Americans fear losing their vision more than any other sense, such as hearing or speech, and almost as much as they fear cancer, Alzheimer’s and HIV/AIDS. That’s not too surprising. Our eyes are our connection to the world around us. Sever that connection, and the world is a very different place. For … Continue reading jCyte starts second phase of stem cell clinical trial targeting vision loss
CIRM Alpha Clinics Network charts a new course for delivering stem cell treatments
Sometimes it feels like finding a cure is the easy part; getting it past all the hurdles it must overcome to be able to reach patients is just as big a challenge. Fortunately, a lot of rather brilliant minds are hard at work to find the most effective ways of doing just that. Last week, … Continue reading CIRM Alpha Clinics Network charts a new course for delivering stem cell treatments
Raising awareness about Rare Disease Day
One of the goals we set ourselves at CIRM in our 2016 Strategic Plan was to fund 50 new clinical trials over the next five years, including ten rare or orphan diseases. Since then we have funded 13 new clinical trials including four targeting rare diseases (retinitis pigmentosa, severe combined immunodeficiency, ALS or Lou Gehrig’s … Continue reading Raising awareness about Rare Disease Day
Partnering with the best to help find cures for rare diseases
As a state agency we focus most of our efforts and nearly all our money on California. That’s what we were set up to do. But that doesn’t mean we don’t also look outside the borders of California to try and find the best research, and the most promising therapies, to help people in need. … Continue reading Partnering with the best to help find cures for rare diseases
Rare diseases are not so rare
It seems like a contradiction in terms to say that there are nearly 7,000 diseases, affecting 30 million people, that are considered rare in the US. But the definition of a rare disease is one that affects fewer than 200,000 people and the National Institutes of Health’s (NIH) Genetic and Rare Diseases Information Center (GARD) … Continue reading Rare diseases are not so rare
A ‘Call to Action’ for change at the FDA
It’s bad enough to have to battle a debilitating and ultimately deadly disease like Huntington’s disease (HD). But it becomes doubly difficult and frustrating when you feel that the best efforts to develop a therapy for HD are running into a brick wall. That’s how patients and patient advocates working on HD feel as they … Continue reading A ‘Call to Action’ for change at the FDA
The Stem Cellar 