At CIRM we don’t have a disease hierarchy list that we use to guide where our funding goes. We don’t rank a disease by how many people suffer from it, if it affects children or adults, or how painful it is. But if we did have that kind of hierarchy you can be sure that … Continue reading Stem cell agency funds Phase 3 clinical trial for Lou Gehrig’s disease
ALS
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
Avalanches of exciting new stem cell research at the Keystone Symposia near Lake Tahoe
From January 8th to 13th, nearly 300 scientists and trainees from around the world ascended the mountains near Lake Tahoe to attend the joint Keystone Symposia on Neurogenesis and Stem Cells at the Resort at Squaw Creek. With record-high snowfall in the area (almost five feet!), attendees had to stay inside to stay warm and dry, … Continue reading Avalanches of exciting new stem cell research at the Keystone Symposia near Lake Tahoe
Using stem cells to fix bad behavior in the brain
Diseases of the brain have many different names, from Alzheimer’s and Parkinson’s to ALS and Huntington’s, but they often have similar causes. Researchers at the Gladstone Institutes in San Francisco are using that knowledge to try and find an approach that might be effective against all of these diseases. In a new CIRM-funded study, … Continue reading Using stem cells to fix bad behavior in the brain
Ingenious CIRM-funded stem cell approach to treating ALS gets go-ahead to start clinical trial
Amyotrophic lateral sclerosis (ALS), better known as Lou Gehrig’s disease, was first identified way back in 1869 but today, more than 150 years later, there are still no effective treatments for it. Now a project, funded by CIRM, has been given approval by the Food and Drug Administration (FDA) to start a clinical trial that … Continue reading Ingenious CIRM-funded stem cell approach to treating ALS gets go-ahead to start clinical trial
How the Ice Bucket Challenge changed the fight against ALS
200 people in Boston take the Ice Bucket Challenge: Photo courtesy Forbes A couple of years ago millions of people did something they probably never thought they would: they dumped a bucket of ice cold water on their head to raise awareness about a disease most of them had probably never heard of, and almost … Continue reading How the Ice Bucket Challenge changed the fight against ALS
A Dream made me change my mind. Almost.
Dream Alliance: photo courtesy Daily Telegraph, UK On Friday I was faced with the real possibility that a horse had made an ass out of me. Over the years we have written many articles about the risks of unproven stem cell therapies, treatments that have not yet been shown in clinical trials to be safe … Continue reading A Dream made me change my mind. Almost.
Rare disease underdogs come out on top at CIRM Board meeting
It seems like an oxymoron but one in ten Americans has a rare disease. With more than 7,000 known rare diseases it’s easy to see how each one could affect thousands of individuals and still be considered a rare or orphan condition. Only 5% of rare diseases have FDA approved therapies People with rare … Continue reading Rare disease underdogs come out on top at CIRM Board meeting
Rare Disease Day, a chance to raise awareness and hope.
Battling a deadly disease like cancer or Alzheimer’s is difficult; but battling a rare and deadly disease is doubly so. At least with common diseases there is a lot of research seeking to develop new treatments. With rare diseases there is often very little research, and so there are fewer options for treatment. Even just … Continue reading Rare Disease Day, a chance to raise awareness and hope.