Cord Blood Registry (CBR) announced the launch of an FDA-regulated clinical trial being conducted at The University of Texas Health Science Center at Houston to investigate two forms of stem cell therapy in children diagnosed with cerebral palsy. The randomized, placebo-controlled study will compare the safety and efficacy of an intravenous infusion of cord blood stem cells to bone marrow stem cells.
There is a critical need for the development of new treatments as the incidence and prevalence of cerebral palsy has increased over time. According to the Centers for Disease Control and Prevention, it is estimated that nearly 1 in every 323 children in the United States has been diagnosed with cerebral palsy — a group of disorders that affects an individual’s ability to move and maintain balance and posture. The condition is caused by abnormal brain development or damage to the motor control centers of the developing brain. Infusion of cord blood stem cells to help trigger the body’s own repair mechanisms could provide a non-invasive therapeutic option that does not exist today.
Recruiting efforts are underway to enroll 30 children between the ages of 2 and 10 diagnosed with cerebral palsy: 15 with a CBR processed and stored cord blood unit who may be administered an autologous stem cell infusion and 15 children who may undergo a bone marrow harvest and autologous stem cell infusion. To evaluate improvement in functional status among participants, five children in each group will be randomized to a placebo control group during the baseline/treatment visit. Parents will not be informed if their child received stem cells or were given a placebo until the 1-year follow-up examination. At that time, parents whose children were administered the placebo may elect to have their child receive the stem cell therapy, either through bone marrow harvest or cord blood. In order to be eligible to participate in the cord blood arm of the trial, families must have a qualified CBR processed unit that was collected at birth.
The principal investigator of the study is Dr. Charles Cox, the Children’s Fund Distinguished Professor of Pediatric Surgery at The University of Texas Medical School at Houston. Dr. Cox noted that “there is preclinical data indicating that the ongoing neuroinflammatory response is a driver of further injury in cerebral palsy so the hope is to reduce this neuroinflammation.”
“Our goal is to break the cycle of inflammation and injury,” he added.
A child’s eligibility will be determined in consultation with the study team at The University of Texas Health Science Center at Houston. Families must be able to travel to Houston for the treatment and follow-up visits at 6, 12, and 24 months. Study collaborators include CBR, Let’s Cure CP Foundation, TIRR Foundation, and Children’s Memorial Hermann Hospital.
Date: April 15, 2014
Source: Cord Blood Registry
Filed Under: Drug Discovery