Boy has unprecedented surgery
Daniel Kerner, a California boy who received the first known implant of purified brain stem cells last month at Oregon Health & Science University, is about to leave the hospital and head home.
"Daniel is doing exceptionally well," his father, Marcus Kerner, an assistant U.S. attorney in Trabuco Canyon, Calif., said Sunday in a phone interview. "We're very, very excited about what is going on with our son."
The 6-year-old has Batten disease, a deadly, progressive illness in which the body cannot make an enzyme that removes toxins from the brain. That buildup of toxins steadily damages the brain, causing blindness, problems moving and thinking and seizures.
People with Batten disease, which is genetic, usually die in childhood. Daniel already is unable to speak and uses a wheelchair.
This is the first time that the family has confirmed Daniel had the surgery. In March, Kerner said he had applied for the trial.
On Nov. 14, OHSU doctors drilled through the boy's skull and injected a form of nervous-system stem cells purified from human fetal tissue by Palo Alto, Calif.-based StemCells Inc., the company sponsoring the trial. Tests on animals showed the cells may start making the missing enzyme, perhaps slowing or halting the disease.
Doctors think it was the first transplant of pure stem cells into the brain, though pieces of fetal brain tissue have been put into the brains of other sick people before.
The main goal of the OHSU experiment is to see whether implanting the stem cells is safe. Doctors plan to operate on six children, testing two doses of stem cells. Although doctors hope the treatment helps, it has never been studied in people and so has unknown risks and uncertain benefits.
Earlier, doctors said they intended to monitor the child for at least 30 days before performing a second stem-cell transplant.
The fact that Daniel is expected to leave the hospital this week doesn't mean the operation eases the disease. It just shows that he recovered from the surgery, as expected. Doctors will follow him and any others in the trial for years to see whether the implanted stem cells appear to slow the disease or ease symptoms.
The Kerner family understands that the experiment may not help but hopes it will, Marcus Kerner said in March, when doctors were planning the trial. "We don't have blinders on. We know this is research," Kerner said. But "we believe this is going to be one of the most profound medical breakthroughs in history, and we believe it's going to save his life."
Families and friends of children with Batten are watching the research closely and hopefully. A confidentiality agreement between OHSU and StemCells Inc. has limited public discussion of information about the trial.
The Kerners have planned a news conference Tuesday to share their experience and thoughts about the research.
