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
Rare Diseases
Stem Cells Profile in Courage: Pat Furlong, Patient Advocate
One of the true joys for me in helping put together this year’s Annual Report was getting to know the patients and patient advocates that we profiled in the report. These are some extraordinary individuals and the short profiles we posted only touch the surface of just how extraordinary. So, over the next few weeks … Continue reading Stem Cells Profile in Courage: Pat Furlong, Patient Advocate
Stem cell and gene therapy research gets a good report card from industry leader
The state of the regenerative medicine field is strong and getting stronger. That was the bottom line verdict at the 2017 Cell and Gene Therapies State of the Industry briefing in San Francisco. The briefing, an annual update on the field presented by the Alliance for Regenerative Medicine (ARM), gave a “by the numbers” look … Continue reading Stem cell and gene therapy research gets a good report card from industry leader
Cured by Stem Cells
To get anywhere you need a good map, and you need to check it constantly to make sure you are still on the right path and haven’t strayed off course. A year ago the CIRM Board gave us a map, a Strategic Plan, that laid out our course for the next five years. Our Annual … Continue reading Cured by Stem Cells
Translating great stem cell ideas into effective therapies
In science, there are a lot of terms that could easily mystify people without a research background; “translational” is not one of them. Translational research simply means to take findings from basic research and advance them into something that is ready to be tested in people in a clinical trial. Yesterday our Governing Board approved … Continue reading Translating great stem cell ideas into effective therapies
Failed stem cells may cause deadly lung disease
Breathing is something we take for granted. It’s automatic. We don’t need to think about it. But for people with pulmonary fibrosis, breathing is something that is always on their minds. Pulmonary fibrosis (PF) is a disease where the tissue in your lungs becomes thick and stiff, even scarred, making it difficult to breathe. It … Continue reading Failed stem cells may cause deadly lung disease
Stem cell agency funds clinical trials in three life-threatening conditions
A year ago the CIRM Board unanimously approved a new Strategic Plan for the stem cell agency. In the plan are some rather ambitious goals, including funding ten new clinical trials in 2016. For much of the last year that has looked very ambitious indeed. But today the Board took a big step towards reaching … Continue reading Stem cell agency funds clinical trials in three life-threatening conditions
Meeting the scientists who are turning their daughter’s cells into a research tool – one that could change her life forever
There’s nothing like a face-to-face meeting to really get to know someone. And when the life of someone you love is in the hands of that person, then it’s a meeting that comes packed with emotion and importance. Last week Gay and Steve Grossman got to meet the people who are working with their daughter … Continue reading Meeting the scientists who are turning their daughter’s cells into a research tool – one that could change her life forever
How research on a rare disease turned into a faster way to make stem cells
If Forest Gump were a scientist, I’d like to think he would have said his iconic line a little differently. Dr. Gump would have said, “scientific research is like a box of chocolates - you never know what you’re gonna get.” A new CIRM-funded study coming out of the Gladstone Institutes certainly proves this point. … Continue reading How research on a rare disease turned into a faster way to make stem cells
Funding stem cell research targeting a rare and life-threatening disease in children
If you have never heard of cystinosis you should consider yourself fortunate. It’s a rare condition caused by an inherited genetic mutation. It hits early and it hits hard. Children with cystinosis are usually diagnosed before age 2 and are in end-stage kidney failure by the time they are 9. If that’s not bad enough … Continue reading Funding stem cell research targeting a rare and life-threatening disease in children
The Stem Cellar 