Translating Regeneration Science Into Treatments for Kids
Ophir Klein is growing teeth, which is just slightly less odd than what Jeffrey Bush is growing – tissues that make up the face.
Jason Pomerantz is growing muscle; Sarah Knox is growing salivary glands; and Edward Hsiao is printing 3-D bone using a machine that looks about as complex as a clock radio. Together, these UCSF faculty members are cultivating organs of the skull and face – known as the craniofacial complex – which too often go terribly wrong during fetal development.
Deformities of these bones or soft tissues, the most common of birth defects, can cut life short by blocking the airway or circulation. Or they can disfigure a face so profoundly that a child struggles to see, hear, or talk. Most painfully, such deformities can render children physically other, potentially leading to a lifetime of corrective surgeries and social isolation.
As director of the UCSF Program in Craniofacial Biology, Klein orchestrates a multisite research endeavor to translate tissue regeneration science into improved treatments for these kids. Using stem cells from patients with craniofacial deformities, Klein, Bush, Pomerantz, Knox, Hsiao, and colleagues are growing tiny functioning segments of organs, called organoids, to pinpoint when and how in fetal development such design flaws occur.
They are among scientists across UCSF who are cultivating cellular systems, such as miniature brains and breasts, from patient cells. They serve as dioramas of disease – models derived from human cells – either displacing or complementing the mouse models that have served science well, though inexactly, for years.
The effort is one of the most viable payoffs to date from stem cell science. With these organoids, physicians and scientists can not only trace the pathways of normal and abnormal development, but also test treatments for their effectiveness in humans. Organoids are also one tiny step toward the ultimate goal of generating complete organs, as a way to circumvent rejection issues and save the lives of those who now die waiting for transplants.