Gene Therapy May Slow Alzheimer's Disease
Altered Cells Placed in Brain Safe; Seem to Slow Mental Decline
April 27, 2004 -- It's new. It's radical. And now a gene-based therapy for Alzheimer's disease has passed its first human test.
One patient died and another was injured before doctors learned how to do it safely. But now the technique -- brain implants of genetically altered skin cells -- looks safe enough for more advanced human tests.
The early results look promising, according to a report by Mark Tuszynski, MD, PhD, at this week's meeting of the American Academy of Neurology in San Francisco. Tuszynski is professor of neuroscience at the University of California, San Diego, which holds the patent on the new technology.
"These results are intriguing," Tuszynski says in a news release. "If these effects are borne out in larger, controlled trials, this could be a significant advance over existing therapies for Alzheimer's disease."
Here's how the gene therapy works:
It's not a cure. But in the six patients who successfully completed surgery, the gene therapy slowed the relentless progress of Alzheimer's disease. The patients had been losing 7 points a year on a test of mental function. A year and a half after surgery, this decline slowed to a loss of 4 points a year.
Unfortunately, the brain surgery to implant the gene therapy used only a local anesthetic on the first two patients. They moved during surgery, causing bleeding in the brain. One of the patients died five weeks later of a heart attack.
After doctors switched to a general anesthetic (being put under), there were no more injuries.
This safety record -- and the early signs that the treatment might be working -- means a phase II trial can begin. That study will be conducted at Rush University in Chicago. More medical centers may become involved if the study moves into larger-scale trials.
The University of California holds the patent on the treatment and has licensed it to Ceregene Inc. in San Diego, a subsidiary of Cell GeneSys Inc. in South San Francisco, Calif.
Gene Therapy May Slow Alzheimer's Disease
Altered Cells Placed in Brain Safe; Seem to Slow Mental Decline
April 27, 2004 -- It's new. It's radical. And now a gene-based therapy for Alzheimer's disease has passed its first human test.
One patient died and another was injured before doctors learned how to do it safely. But now the technique -- brain implants of genetically altered skin cells -- looks safe enough for more advanced human tests.
The early results look promising, according to a report by Mark Tuszynski, MD, PhD, at this week's meeting of the American Academy of Neurology in San Francisco. Tuszynski is professor of neuroscience at the University of California, San Diego, which holds the patent on the new technology.
"These results are intriguing," Tuszynski says in a news release. "If these effects are borne out in larger, controlled trials, this could be a significant advance over existing therapies for Alzheimer's disease."
Gene Therapy for Alzheimer's Disease
Here's how the gene therapy works:
- An Alzheimer's disease patient donates some skin cells.
- The patient's skin cells are genetically engineered to make a natural brain chemical called nerve growth factor, or NGF.
- The altered cells are grown in the laboratory.
- In a surgical procedure, doctors use needles to implant the altered cells deep in the patient's brain.
- The cells secrete NGF into the brain. This slows the death of brain cells (a hallmark of Alzheimer's) and improves brain-cell function. It also may help restore the connections between brain cells.
It's not a cure. But in the six patients who successfully completed surgery, the gene therapy slowed the relentless progress of Alzheimer's disease. The patients had been losing 7 points a year on a test of mental function. A year and a half after surgery, this decline slowed to a loss of 4 points a year.
Unfortunately, the brain surgery to implant the gene therapy used only a local anesthetic on the first two patients. They moved during surgery, causing bleeding in the brain. One of the patients died five weeks later of a heart attack.
After doctors switched to a general anesthetic (being put under), there were no more injuries.
This safety record -- and the early signs that the treatment might be working -- means a phase II trial can begin. That study will be conducted at Rush University in Chicago. More medical centers may become involved if the study moves into larger-scale trials.
The University of California holds the patent on the treatment and has licensed it to Ceregene Inc. in San Diego, a subsidiary of Cell GeneSys Inc. in South San Francisco, Calif.
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