Friday, September 27, 2019

In Utero Transplant in First Clinical Trial Successful

At the University of California, pediatric surgeons have treated a fetus, in the second trimester, using stem sells from the mother's bone marrow. After being born in February of 2018, this baby was officially the "first patient enrolled in the world's first clinical trial using stem cells transplanted prior to birth"(Daley 2018). Although the baby is still living with alpha thalassemia, a deadly genetic disease, pediatric surgeon, Tippi Mackenzie states, "her healthy birth suggests that fetal therapy is a viable option to offer to families with this diagnosis”(Daley 2018). In 2016, the trial officially began to investigate stem cell research to treat thalassemia. In this specific case, the baby had a lethal form caused by "abnormalities on the HBA1 and HBA2 genes,"(Daley 2018) called alpha thalassemia major, which typically causes babies to die before birth or are stillborn.

This unfortunate diseaese "is an inherited blood disorder that affects the body's ability to produce hemoglobin,"(Falck 2018) and can cause swelling of the liver or heart. Therefore, after four months in a clinical trial, five blood infusions, and one stem cell transplant, it is amazing that Elianna Constantino was born healthy. Elianna's enlarged heart, a sign of thalassemia, was detected during pregnancy through an ultrasound. According to the UCSF statement, "intrauterine blood transfusions were required to treat the swelling before the stem cell transplant could be performed"(Daley 2018). Even though it was a lengthy process, Elianna is said to be "doing great." Therefore, which once was a universally fatal disease, "can now be managed as a chronic disease," says Elliott Vichinsky, a hematologist and the founder of the Northern California Comprehensive Thalassemia Center. Today, Vichinsky is overseeing the baby's treatment and everything is looking healthy.

1 comment:

  1. Great topic of choosing from Emily! I agree with you this once fatal disease can now be able to control with treatment. This is excellent news because it helps the mother who fetuses diagnosed with alpha thalassemia from terminating the pregnancy.