A pipette drops stem cell research fluid on a special container. (Photo: CI Photos, via Shutterstock)
The “bubble babies” saga and a California-financed cure for their life-threatening affliction has hit another snag, more than two years after a British company abandoned the effort. It is a story that involves more than $40 million from California’s stem cell agency, federal regulators, the University of California, the agonizingly slow pace of science and 20 children who have been denied care — not to mention a company called Orchard Therapeutics PLC.
Baby Evangelina Padilla-Vaccaro on the day she received a gene therapy stem cell transplant.(Photo: UCLAhealth.org)
Twenty children seeking treatment for a rare affliction called the “bubble baby disease” today have some big-time, good news concerning a life-saving genetic therapy that they were once denied as the result of a tangled affair that included private profit and the public funding of cutting-edge scientific research.