BEIJING, Aug. 1 (Xinhuanet) -- Scientists have found
a way to change skin cells from patients with Lou Gehrig's disease into motor
neurons that are genetically identical to the patients' own neurons, giving them
an unlimited number of these neurons for study in the laboratory.
The new process should should result in a better
understanding of the disease and lead to new treatments or even the production
of healthy cells that can replace the diseased ones.
"The hope of some scientists is that they might be
able to harness stem cells and program them to generate pluripotent stem cell
lines [capable of differentiating into many different types of cells] which have
the genes of patients," said Kevin Eggan, co-author of a paper appearing July 31
in the online version of Science. "This would open up the possibility of
producing a large supply of immune-matched cells to that patient that could be
used in transplantation methodologies.
"The other hope, and one that's much closer upon us .
. . is if you could produce the cell types that become sick in that person, you
might be able to use them in the laboratory to come to understand basic aspects
of the disease and take the study of disease out of patients, where it's very
difficult, and put it into the Petri dish," added Eggan, who is a principal
faculty member at the Harvard Stem Cell Institute and spoke about the research
at a teleconference Wednesday.
However, the actual therapeutic potential of this
approach is still years away.
Lou Gehrig's disease or ALS (amyotrophic lateral
sclerosis) is caused by the degeneration and death of spinal motor neurons,
which carry messages from the spinal cord to the body's muscles. This leads to
paralysis of muscles and, eventually, death. Some 30,000 people in the United
States suffer from the disease, which has no cure.
"We don't at all fully understand [ALS], and it is
our lack of understanding of that disease process which we believe is preventing
us from developing more effective [treatments]," said Christopher Henderson, a
co-author on the paper and co-director of the Center for Motor Neuron Biology
and Disease at Columbia University. "Because the disease process is happening in
the spinal cord in the central nervous system of patients, we don't at all have
access to living examples of the neurons that are undergoing the disease
process. . . . No way could we go to ALS patients and take samples of their
motor neurons."
The scientists had originally planned to use somatic
cell nuclear transfer (SCNT), or "therapeutic cloning," to try to accomplish
this feat. That process involves removing the genetic material from a donated
human oocyte and replacing it with genetic material from the skin cells of
patients. The approach has been hindered by political, ethical and other
obstacles.
(Agencies)