Yesterday the governing Board of the California Institute for Regenerative Medicine (CIRM) awarded $8.39 million to the University of California, San Francisco (UCSF) to fund a clinical trial for sickle cell disease (SCD). An additional $51.08 million was awarded to fifteen community colleges and universities across California to fund undergraduate and master’s level programs that … Continue reading Board Funds Fifteen Bridges to Stem Cell Research and Therapy Programs Across California and New Sickle Cell Disease Trial
Clinical Trials
Gene therapy is life-changing for children with a life-threatening brain disorder
If you have never heard of AADC deficiency count yourself lucky. It’s a rare, incurable condition that affects only around 135 children worldwide but it’s impact on those children and their families is devastating. The children can’t speak, can’t feed themselves or hold up their head, they have severe mood swings and often suffer from … Continue reading Gene therapy is life-changing for children with a life-threatening brain disorder
We’ve got cash, here’s how you can get some
When the voters of California approved Proposition 14 last November (thanks folks) they gave us $5.5 billion to continue the work we started way back in 2014. It’s a great honor, and a great responsibility. It’s also a great opportunity to look at what we do and how we do it and try to come … Continue reading We’ve got cash, here’s how you can get some
Physicians and patient advocates on the front lines of the fight for a more equitable health system
Over the last year there has been increasing awareness of the inequalities in the American healthcare system. At every level there is evidence of bias, discrimination and unequal access to the best care. Sometimes unequal access to any care. That is, hopefully, changing but only if the new awareness is matched with action. At the … Continue reading Physicians and patient advocates on the front lines of the fight for a more equitable health system
CIRM Board Approves New Clinical Trial for ALS
This past Friday the governing Board of the California Institute for Regenerative Medicine (CIRM) awarded $11.99 million to Cedars-Sinai to fund a clinical trial for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. ALS is a neurodegenerative disease that results in the death of nerve cells in the brain and spinal cord, causing … Continue reading CIRM Board Approves New Clinical Trial for ALS
An Open Letter to CIRM for World Sickle Cell Day
Nancy M. Rene Dear CIRM, World Sickle Cell Day is this Saturday June 19th. The goal of this day is to increase knowledge of the disease and understanding of the challenges faced. It is a day that I greet with very mixed feelings. I’m of course extremely grateful to CIRM for the time and money … Continue reading An Open Letter to CIRM for World Sickle Cell Day
Sometimes a cold stare is a good thing
A retina of a patient with macular degeneration. (Photo credit: Paul Parker/SPL) Age-related macular degeneration (AMD) is the leading cause of vision loss and blindness in the elderly in the U.S. It’s estimated that some 11 million Americans could have some form of the disease, a number that is growing every year. So if you … Continue reading Sometimes a cold stare is a good thing
CIRM-catalyzed spinout files for IPO to develop therapies for genetic diseases
Graphite Bio, a CIRM-catalyzed spinout from Stanford University that launched just 14 months ago has now filed the official SEC paperwork for an initial public offering (IPO). The company was formed by CIRM-funded researchers Matt Porteus, M.D., Ph.D. and Maria Grazia Roncarolo, M.D. Six years ago, Dr. Porteus and Dr. Roncarolo, in conjunction with Stanford … Continue reading CIRM-catalyzed spinout files for IPO to develop therapies for genetic diseases
Two voices, one message, watch out for predatory stem cell clinics
Last week two new papers came out echoing each other about the dangers of bogus “therapies” being offered by predatory stem cell clinics and the risks they pose to patients. The first was from the Pew Charitable Trusts entitled: ‘Harms Linked to Unapproved Stem Cell Interventions Highlight Need for Greater FDA Enforcement’ with a subtitle: … Continue reading Two voices, one message, watch out for predatory stem cell clinics
CIRM funded trial may pave way for gene therapy to treat different diseases
Image Description: Jordan Janz (left) and Dr. Stephanie Cherqui (right) According to the National Organization for Rare Disorders (NORD), a disease is consider rare if it affects fewer than 200,000 people. If you combine the over 7,000 known rare diseases, about 30 million people in the U.S. are affected by one of these conditions. A … Continue reading CIRM funded trial may pave way for gene therapy to treat different diseases
The Stem Cellar 