Catalyst Program Awardee Executes World’s First In Utero Blood Stem Cell Therapy Clinical trial

Tippi MacKenzie (left), MD, of UCSF Benioff Children’s Hospital San Francisco, visits with newborn Elianna, who received a stem cell transplant before birth
Tippi MacKenzie (left), MD, of UCSF Benioff Children’s Hospital San Francisco, visits with newborn Elianna, who received a stem cell transplant before birth. Photo by Barbara Ries

Accelerating Discoveries to Clinical Impact

 

The Catalyst Program is UC San Francisco CTSI’s early translational accelerator focused on advancing UCSF discoveries with clinical relevance and commercial potential. The centerpiece of the Catalyst Program are Catalyst Awards that provide both industry advisor mentorship and seed funding to UCSF PIs with promising projects.

 

Back in 2015 Catalyst Awardee, Tippi MacKenzie, MD, who won for her work on in utero hematopoetic cell transplantation for treating alpha thalassemia major, said the program’s process gave her the skills needed to move from lab to clinical trials. “Taking the leap to a clinical trial is huge. It involves a body of knowledge I don’t have,” MacKenzie said. “The Catalyst Program has provided that knowledge. Having the Catalyst team sort of hold your hand through the process has been great.”

 

Fast forward to 2018, MacKenzie’s team has safely transplanted a woman’s stem cells into her growing fetus, leading to the live birth of an infant with a normally fatal fetal condition. The infant, who had been critically ill during the second-trimester of pregnancy due to alpha thalassemia, is the first patient enrolled in the world’s first clinical trial using blood stem cells transplanted prior to birth. Although the mother’s stem cells did not engraft, this early stage research trial showed that it is a viable, safe procedure and will continue to be evaluated in other patients in the ongoing clinical trial.

Read News Coverage here:

https://www.ucsf.edu/news/2018/05/410436/ucsf-first-utero-stem-cell-transplant-clinical-trial

https://www.nytimes.com/2018/05/25/health/fetal-bone-marrow-transplant.html

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