Imagine a child suffering from an incurable disease. Now imagine that the cure is just waiting to be unlocked from inside that child’s own cells. Earlier this year, UCSF launched the Living Therapeutics Initiative to accelerate groundbreaking work into cellular therapies and advance promising new treatments for our most vulnerable patients.
Here’s what the experts have to say:
Michelle Hermiston, MD, PhD
Director, UCSF Pediatric Immunotherapy Program
"Historically, when a child was diagnosed with leukemia, and they weren't responding to chemotherapy, we considered it incurable. It meant that their disease was smarter than our science. Now, with cellular therapies, our science has caught up. We can actually use these new technologies when conventional approaches have failed, and we can offer these kids a second chance to live normal lives and get back to doing the things that kids are supposed to do, like going to school and playing with their friends. It's just awesome. I feel so privileged to do this work."
Susan Lynch, PhD
Director, UCSF Benioff Center for Microbiome Medicine
"The recent discovery that we share our bodies with a dynamic population of microbial cells that shape early development and health has been one of the most exciting advances in biomedical research in decades. Advancing this field through the lens of living microbial therapeutics offers incredible opportunities. We are designing and testing ‘cocktails’ of live microbes to treat a range of conditions, including multiple sclerosis, periodontitis, allergies, and complications of prematurity. I am so excited about what this could mean for our youngest and most vulnerable patients. To be at this stage of discovery and translation is simply wonderful."
Tippi MacKenzie, MD
Co-Director, UCSF Center for Maternal-Fetal Precision Medicine
"My work focuses on fetal therapies – treating children’s genetic diseases in the womb, before birth. We are currently running the world’s first clinical trial using blood stem cells donated by mothers and transfused into the fetus to treat a fatal blood disorder. One day, we may be able to edit a fetus’s own cells to repair the mutation that causes the disease. Right now, UCSF has amazing expertise in the basic science of stem cell biology, and we have world-class clinical capabilities at our hospitals. The Living Therapeutics Initiative is bringing these pieces together. It will be transformational."
Mark Walters, MD
Interim Chief, Division of Hematology
"I have spent my career trying to cure children with sickle cell disease. But this is only possible when there is a matched transplant donor, which happens rarely. We are now trying to modify the sickle DNA in a child’s own blood cells, then use the healthy cells to cure the disease. The challenge is improving the technology so we can modify enough cells for a cure. With the Living Therapeutics Initiative, we can do this work here at UCSF. We can broaden access to this kind of curative therapy and make it available to so many more patients."