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
Author: Kevin McCormack
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
TV’s Dr. Oz takes on clinics offering dubious stem cell treatments
At first glance motor car racing legend A. J. Foyt and TV celebrity heart surgeon Dr. Mehmet Oz would seem to have little in common. But this week they both made news for being at opposite ends of an all too familiar story: for-profit medical clinics offering unproven stem cell therapies. Foyt, who is now … Continue reading TV’s Dr. Oz takes on clinics offering dubious stem cell treatments
The power of the patient’s voice: how advocates shape clinical trials and give hope to those battling deadly diseases
Tennis great Martina Navratilova was once being interviewed about what made her such a great competitor and she said it was all down to commitment. When pressed she said “the difference between involvement and commitment is like ham and eggs; the chicken is involved but the pig is committed.” That’s how I feel about the … Continue reading The power of the patient’s voice: how advocates shape clinical trials and give hope to those battling deadly diseases
How a Soviet space craft proved an inspiration for CIRM’s latest Board member
George Blumenthal’s life changed on October 4, 1957. That’s the day the Soviet Union launched Sputnik, the world’s first artificial earth satellite. The beach ball-sized satellite marked the start of the space race between the US and the USSR. It also marked the start of Blumenthal’s fascination with science and space. Fast forward almost 60 … Continue reading How a Soviet space craft proved an inspiration for CIRM’s latest Board member
Stories that caught our eye: stem cell transplants help put MS in remission; unlocking the cause of autism; and a day to discover what stem cells are all about
Stem cell transplants help put MS in remission: A combination of high dose immunosuppressive therapy and transplant of a person’s own blood stem cells seems to be a powerful tool in helping people with relapsing-remitting multiple sclerosis (RRMS) go into sustained remission. Multiple sclerosis (MS) is an autoimmune disorder where the body’s own immune system … Continue reading Stories that caught our eye: stem cell transplants help put MS in remission; unlocking the cause of autism; and a day to discover what stem cells are all about
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
Stem Cells Profiles in Courage: Frank’s final gift
Not every story has a happy ending. But they do all have something to teach us. In the case of Frank St. Clair the lesson was simple: live life fully and freely, love those around you, and never give up. We were fortunate enough to get to know Frank as one of the people we … Continue reading Stem Cells Profiles in Courage: Frank’s final gift
Stem Cell Profiles in Courage: Karl’s Fight with Cancer
When I think of a pioneer I have an image in my head of people heading west across the Americans plains in the 18th century, riding in a covered wagon pulled by weary oxen. Karl Trede doesn’t fit that image at all. He is a trim, elegant man who has a ready smile and a … Continue reading Stem Cell Profiles in Courage: Karl’s Fight with Cancer
The Stem Cellar 