Working with a team of more than 100 UCSF specialists, Tippi MacKenzie, MD, and
Elliott Vichinsky, MD, are leading work to bring cutting edge therapies to patients with
genetic blood disorders.
For Dr. Vichinsky, this work began more than 30 years ago, when most patients
with these disorders died in infancy. Today, thanks to cutting edge research and revolutions in patient care, patients are living much longer, higher quality lives.
This work took a major leap forward in the fall of 2017, when his colleague Dr. MacKenzie launched the world’s first clinical trial of an in-utero stem cell transplant therapy. Her first patient, baby Elianna, was born just four months later.
Now 18 months old, she is being treated by Dr. Vichinsky. Elianna's birth came after a second-trimester diagnosis of a normally fatal blood disorder, Alpha Thalassemia Major. Her story hints at the lifesaving possibilities of fetal therapy for families who face similarly devastating diagnoses.