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First recipient of new Batten's disease stem cell cure well enough to go home

18 December 2006
Appeared in BioNews 389

The first child patient to receive stem cells derived from fetal brain tissue is said to be doing well after the procedure. Daniel Kerner, a six year old from Orange County, California, US, who suffers from the rare brain disorder Batten's disease, underwent the risky and experimental surgery in Portland, Oregon, a month ago. Kerner's parents spoke to the media last week to report that their son seems to be responding well to the treatment.

Batten's disease is a previously incurable brain condition where cells in the brain are unable to produce an enzyme involved in removing build ups of fat and protein in the cell called lipopigments. This build up eventually leads to neuronal death. Sufferers of the disorder gradually lose the ability to walk and talk and are generally blind and paralysed by the time they die in their teens.

The new procedure was the first to take place since the US Food and Drug Administration (FDA) approved human tests in October 2005. Oregon Health and Science University doctors agreed to carry out the experimental treatment in March 2006 and in July Daniel Kerner's parents learnt that their son would be the first to undergo the procedure. Talking to the press last week Marcus Kerner, Daniel's father, said: 'He was a little boy who was basically waiting to die, now he's waiting to get better.'

US biotechnology company Stem Cells Inc. has developed and owns the rights to the new treatment for Batten's disease. It say that the new technique is very different from previous attempts to use fetal brain tissue to treat brain disorders where generally cells have been injected into sufferers with little selectivity or purification. In the Batten's disease experiments cells from donated aborted fetuses were highly purified and selected for their ability to replace the faulty cells in Batten's disease sufferers. It is hoped that the new cells will engraft into the brain and produce the enzyme needed to remove the damaging lipopigments.

The goals of the latest experiment are modest, with researchers mainly trying to ascertain whether the stem cell injections harm the recipient. A further five children are expected to be treated in this trial. Animal experiments using the technique in mice showed promising results but the Portland researchers have been trying to temper expectations for this ethically problematic clinical trial. The researchers have remained silent except for a brief press conference two days after the surgery was conducted on 14 November.

Child stem cell recipient heads home
Yahoo Daily News |  12 December 2006
Hope grows as boy improves
The Oregonian |  13 December 2006
OHSU pioneers stem cell surgery
KTVZ |  12 December 2006
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