9 year old stem cell trial pioneer dies in Batten disease
Posted by attilachordash on January 22, 2008
This is exactly the type of clinical trial news that should be taken extremely carefully with all due respect and grief.
A girl enrolled in a stem-cell trial for a fatal disease has died. In January, the nine-year-old received a brain transplant of neural stem cells derived from fetal tissue. She was one of six children in the trial for Batten disease, in which children rarely live into their teens. An independent group monitoring the trial decided that the death was due to the disease not the experimental treatment and said the trial could continue.
The quote is from Monya Baker’s Niche post.
From the press release on StemCells Incorporation’s Phase I clinical trial of its proprietary HuCNS-SC®product candidate (purified human neural stem cells):
The trial is designed to evaluate the safety and preliminary efficacy of HuCNS-SC cells as a potential treatment for infantile and late infantile neuronal ceroid lipofuscinosis (NCL). NCL, which is often referred to as Batten disease, is a rare and fatal neurodegenerative condition afflicting infants and children.
The patient, a nine-year-old girl, was transplanted with HuCNS-SC cells in January 2007 and was due to return this month to the trial site, Oregon Health & Science University’s (OHSU) Doernbecher Children’s Hospital, for her 12 month follow-up. She was hospitalized nearly two weeks ago, suffering from an apparent viral infection, seizures and respiratory distress before succumbing earlier this week.
Posted in Nature Report Stem Cells, The Niche, USA, biotechnology, clinical trial, medicine, regenerative medicine, stem cells | No Comments »






