The recent agreement transferring GSK’s rare disease gene therapies to Orchard Therapeutics was good news for both companies and for the patients who are hoping this research could lead to new treatments, even cures, for some rare diseases. It was also good news for CIRM, which played a key role in helping Orchard grow to … Continue reading Stem Cell Agency’s supporting role in advancing research for rare diseases
Clinical Trials
New CIRM Alpha Stem Cell Clinic offers HOPE for boys with deadly disease
For people battling Duchenne Muscular Dystrophy (DMD), a rare and fatal genetic disorder that slowly destroys muscles, hope has often been in short supply. There is no cure and treatments are limited. But now a new clinical trial at the site of one of the newest CIRM Alpha Stem Cell Clinic Network members could change … Continue reading New CIRM Alpha Stem Cell Clinic offers HOPE for boys with deadly disease
Therapies Targeting Cancer, Deadly Immune Disorder and Life-Threatening Blood Condition Get Almost $32 Million Boost from CIRM Board
An innovative therapy that uses a patient’s own immune system to attack cancer stem cells is one of three new clinical trials approved for funding by CIRM's Governing Board. Researchers at the Stanford University School of Medicine were awarded $11.9 million to test their Chimeric Antigen Receptor (CAR) T Cell Therapy in patients with B … Continue reading Therapies Targeting Cancer, Deadly Immune Disorder and Life-Threatening Blood Condition Get Almost $32 Million Boost from CIRM Board
The Story of a South African Bubble Boy and a Gene Therapy That Gave Him His Life Back
Ayaan Isaacs was born in South Africa on March 4th, 2016 as a seemingly healthy baby. But only a few days in to life, he contracted a life-threatening liver infection. He thankfully survived, only to have the doctors discover a few weeks later that he had something much more troubling – a rare disease that … Continue reading The Story of a South African Bubble Boy and a Gene Therapy That Gave Him His Life Back
Patients at the heart of Alpha Stem Cell Clinics Symposium
I have been to a lot of stem cell conferences over the years and there’s one recent trend I really like: the growing importance and frequency of the role played by patient advocates. There was a time, not so long ago, when having a patient advocate speak at a scientific conference was almost considered a … Continue reading Patients at the heart of Alpha Stem Cell Clinics Symposium
Straight to brain: A better approach to ALS cell therapies?
Getting the go ahead to begin a clinical trial by no means marks an end to a research team’s laboratory studies. A clinical trial is merely one experiment and is designed to answer a specific set of questions about a specific course of treatment. There will inevitably be more questions to pursue back in the … Continue reading Straight to brain: A better approach to ALS cell therapies?
Stem Cell Agency’s Diane Winokur hailed as Visionary
Generally speaking, I am not a huge fan of gala dinners. It’s not that I don’t like seeing people who do remarkable things getting a well-deserved honor. It’s just that the dinners often go on too long and the food is usually not very good (hey, this is San Francisco, those things matter). But last … Continue reading Stem Cell Agency’s Diane Winokur hailed as Visionary
A road trip to the Inland Empire highlights a hot bed of stem cell research
It took us longer than it should have to pay a visit to California’s Inland Empire, but it was definitely worth the wait. Yesterday CIRM’s Roadshow went to the University of California at Riverside (UCR) to talk to the community there – both scientific and public – about the work we are funding and the … Continue reading A road trip to the Inland Empire highlights a hot bed of stem cell research
Stem cell study holds out promise for kidney disease
Kidney failure is the Rodney Dangerfield of diseases, it really doesn’t get the respect it deserves. An estimated 660,000 Americans suffer from kidney failure and around 47,000 people die from it every year. That’s more than die from breast or prostate cancer. But now a new study has identified a promising stem cell candidate that … Continue reading Stem cell study holds out promise for kidney disease
The moment of truth. A video about the stem cell therapy that could help millions of people going blind.
“No matter how much one prepares, the first patient is always something very special.” That’s how Dr. Mark Humayun describes his feelings as he prepared to deliver a CIRM-funded stem cell therapy to help someone going blind from dry age-related macular degeneration (AMD). Humayun, an ophthalmologist and stem cell researcher at USC, spent years developing … Continue reading The moment of truth. A video about the stem cell therapy that could help millions of people going blind.
The Stem Cellar 