Cool Instagram image of the week: Chemo- and radiation-free blood stem cell transplant showing promise Bubble baby disease, also known as severe combined immunodeficiency (SCID), is an inherited disorder that leaves newborns without an effective immune system. Currently, the only approved treatment for SCID is a blood stem cell transplant, in which the patient’s defective … Continue reading Friday Roundup: A better kind of blood stem cell transplant; Encouraging news from spinal cord injury trial; Finding an “elusive” cell that could help diabetics
SCID
Using the AIDS virus to help children battling a deadly immune disorder
More than 35 million people around the world have been killed by HIV, the virus that causes AIDS. So, it’s hard to think that the same approach the virus uses to infect cells could also be used to help children battling a deadly immune system disorder. But that’s precisely what researchers at UC San Francisco … Continue reading Using the AIDS virus to help children battling a deadly immune disorder
Progress to a Cure for Bubble Baby Disease
Welcome back to our “Throwback Thursday” series on the Stem Cellar. Over the years, we’ve accumulated an arsenal of exciting stem cell stories about advances towards stem cell-based cures for serious diseases. Today we’re featuring stories about the progress of CIRM-funded clinical trials for the treatment of a devastating, usually fatal, primary immune disease that … Continue reading Progress to a Cure for Bubble Baby Disease
From trauma to treatment: a Patient Advocate’s journey from helping her son battle a deadly disease to helping others do the same
For every clinical trial CIRM funds we create a Clinical Advisory Panel or CAP. The purpose of the CAP is to make recommendations and provide guidance and advice to both CIRM and the Project Team running the trial. It’s part of our commitment to doing everything we can to help make the trial a success … Continue reading From trauma to treatment: a Patient Advocate’s journey from helping her son battle a deadly disease to helping others do the same
Stem cell stories that caught our eye: bubble baby therapy a go in UK, in-utero stem cell trial and novel heart disease target
There were lots of CIRM mentions in the news this week. Here are two brief recaps written by Karen Ring to get you up to speed. A third story by Todd Dubnicoff summarizes an promising finding related to heart disease by researchers in Singapore. CIRM-funded “bubble baby” disease therapy gets special designation by UK. … Continue reading Stem cell stories that caught our eye: bubble baby therapy a go in UK, in-utero stem cell trial and novel heart disease target
CIRM-funded life-saving stem cell therapy gets nod of approval from FDA
If you have read our 2016 Annual Report (and if you haven’t you should, it’s brilliant) or just seen the cover you’ll know that it features very prominently a young girl named Evie Padilla Vaccaro. Evie was born with Severe Combined Immunodeficiency or SCID – also known as “bubble baby disease”; we’ve written about it … Continue reading CIRM-funded life-saving stem cell therapy gets nod of approval from FDA
Bye Bye bubble baby disease: promising results from stem cell gene therapy trial for SCID
You don’t need to analyze any data to know for yourself that Evangelina Vaccaro’s experimental stem cell therapy has cured her of a devastating, often fatal disease of the immune system. All you have to do is look at a photo or video of her to see that she’s now a happy, healthy 5-year-old with a … Continue reading Bye Bye bubble baby disease: promising results from stem cell gene therapy trial for SCID
CIRM Alpha Clinics Network charts a new course for delivering stem cell treatments
Sometimes it feels like finding a cure is the easy part; getting it past all the hurdles it must overcome to be able to reach patients is just as big a challenge. Fortunately, a lot of rather brilliant minds are hard at work to find the most effective ways of doing just that. Last week, … Continue reading CIRM Alpha Clinics Network charts a new course for delivering stem cell treatments
Stem Cell Stories that Caught our Eye: stem cell insights into anorexia, Zika infection and bubble baby disease
Here are some stem cell stories that caught our eye this past week. Some are groundbreaking science, others are of personal interest to us, and still others are just fun. Stem cell model identifies new culprit for anorexia. Eating disorders like anorexia nervosa are often thought to be caused by psychological disturbances or societal pressure. … Continue reading Stem Cell Stories that Caught our Eye: stem cell insights into anorexia, Zika infection and bubble baby disease
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
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