ALS and US
There is hope!
Harvard-Columbia Team
Creates Neurons from ALS Patient’s Skin Cells
New Key to Understanding
and Treating ALS, and a Step Toward Personalized Regenerative Medicine
NEW YORK (July 31,
2008) – Harvard and Columbia scientists have for the first time used a new
technique to transform an ALS (amyotrophic lateral sclerosis, or Lou
Gehrig’s disease) patient’s skin cells into motor neurons, a process that
may be used in the future to create tailor-made cells to treat the
debilitating disease. The research – led by Kevin Eggan, Ph.D. of the
Harvard Stem Cell Institute and Chief Scientific Officer of the New York
Stem Cell Foundation – will be published July 31 in the online version of
the journal Science.
This is the first time
that skin cells from a chronically-ill patient have been reprogrammed into a
stem cell-like state, and then coaxed into the specific cell types that
would be needed to understand and treat the disease. Though cell replacement
therapies are probably still years away, the new cells will solve a problem
that has hindered ALS research for years: the inability to study a patient’s
motor neurons in the laboratory.
ALS is caused by the
degeneration and death of motor neurons, the nerve cells which convey nerve
impulses from the spinal cord to each of the body’s muscles. The death of
motor neurons leads to paralysis of these muscles, including those involved
in swallowing and breathing, and ultimately leads to death of the patient.
The disease affects about 30,000 people in the
The motor neurons were
created using a new technique that reprograms human adult skin cells into
cells that resemble embryonic stem (ES) cells. The technique used to make
these cells – called induced pluripotent stem (iPS) cells – was a major
advance in the field that was first reported last November by researchers in
Scientists had
originally hoped to create neurons and other adult cells using “therapeutic
cloning,” in which DNA from a patient is inserted into a donated egg to
create embryonic stem cells. That technique, however, has still not been
successful in humans, and is also hindered by a shortage of donated eggs.
If the iPS technique
holds its promise in producing neurons and other cells for research, it will
probably replace the “therapeutic cloning” approach, Dr. Henderson says, but
there are still lots of questions about the iPS-derived neurons.
“We don’t know yet how
similar they are to the motor neurons in ALS patients,” he says. “While the
cells exhibit many properties that are typical of motor neurons, we don’t
yet know whether they will be prone to degeneration that will allow us to
mimic the disease in the culture dish and therefore to screen potential
drugs.”
To listen to a media telebriefing held on the new development, please click
here.
Researchers at
“Project A.L.S. has
always maintained that collaboration between scientists is the answer to
understanding and treating this disease,” said Valerie Estess, founder and
research director, Project A.L.S. “We are thrilled to have catalyzed the
Harvard-Columbia collaboration that led to this discovery.”
“Therapeutic use of the
cells is probably a long way off,” Dr. Henderson says. “Right now there are
safety issues with iPS cells, including a risk of cancer. We also don’t know
how to reintroduce cells into a sick adult in a way that will be beneficial.
All these hurdles need to be overcome first before we can think about using
the cells to treat disease, but we can start immediately to evaluate them as
a tool for drug discovery.”
The Columbia and
Harvard researchers were supported by the Harvard Stem Cell Institute
(HSCI), Project A.L.S, the New York Stem Cell Foundation (NYSCF), the SMA
Foundation, MDA Wings Over Wall Street, the Claire and Leonard Tow
Charitable Foundation, the Spina, Drago and Bowen Families, and Ride for
Life, and the Russell Berrie Foundation.
Investigators at
Columbia's Naomi Berrie Diabetes Center are also collaborating with Dr.
Eggan and others at the Harvard Stem Cell Institute and the
“We are excited to be
able to facilitate and support truly important cross-institutional
collaborations such as this significant research effort by Harvard and
Columbia University
Medical Center is home to the Eleanor and Lou Gehrig MDA/ALS Center, which
cares for over 300 ALS patients each year, the Center for Motor Neuron
Biology and Disease, with more than 40 scientists working to uncover the
cause of ALS and other motor neuron diseases, and the Project A.L.S./Jenifer
Estess/Laboratory for Stem Cell Research. In recent years, CUMC scientists
have discovered that motor neurons may be degenerating in ALS in response to
a toxin released by neighboring cells; developed ways to turn embryonic stem
cells into motor neurons; and uncovered how motor neurons mature and find
their way to their target muscles (most recently in a paper published this
week in Cell by Thomas Jessell, Ph.D., the Claire Tow Professor of
Neuroscience, Biochemistry & Molecular Biophysics and Investigator, Howard
Hughes Medical Institute). This progress and the present article moves
- ### -
