Here is the testimony from the Doctor who is in charge of Adult Stem Cell
Research,
as you can see, he's not ready yet for a "Moon Shot".
just me,
Marjorie

United States SenateTestimony of Darwin J. Prockop, M.D., Ph.D.
Director, Center for Gene Therapy
Tulane University Medical Center
Before the Senate Appropriations Subcommittee
on
Labor, Health and Human Services, Education
United States Senate
September 14, 2000
Good Morning.
My name is Darwin Prockop. I am currently the Director of the Center for Gene
Therapy of Tulane University Health Sciences Center in New Orleans,
Louisiana. I
have an M.D. degree, a Ph.D. degree and two honorary doctoral degrees. In
addition, I am a member of the National Academy of Sciences and the
Institute of
Medicine. I have published over 400 scientific articles. My work has been
continuously supported by the National Institutes of Health. In addition, the
Tulane Center for Gene Therapy that I now head is supported by funds from
Tulane
University, the Columbia Healthcare Association and the State of Louisiana
Consortium for Gene Therapy.
Our Tulane Center for Gene Therapy is carrying out research on a special class
of adult stem cells that we think can potentially be used for the treatment
of a
number of devastating diseases. The adult stem cells we are using were first
discovered 20 years ago, and we now know they have a number of truly
remarkable
features. One remarkable feature is that they can easily be obtained from a
small sample of bone marrow from the same patient who is to be treated.
Therefore if our therapies work, we will not need fetal tissues nor will we
need
to introduce foreign cells into a patient. Another important feature of the
cells is that we can grow them rapidly in the laboratory. A technical
consequence of this fact is that we can introduce new genes without the use
of a
virus. But the cells are of special interest primarily because they can change
into a large number of the many different types of cells found in the human
body. For example, we discovered several years ago that if the cells are
isolated from the bone marrow of an animal, and then injected back into the
blood stream of the same experimental animal from which the cells were
obtained,
they travel to a number of different organs and tissues. Moreover, when they
reach a given organ or tissue they become new cells of the same kind that are
normally found in that organ or tissue. In effect, the cells can repair and
rejuvenate the organ or tissue.
These features of the cells raise some exciting possibilities for using
them in
the therapy of diseases. For example, after infusion into a vein, some of the
cells go to bone and become new bone cells. Therefore, the cells can
potentially
be used to treat diseases of bone such as osteoporosis. In fact, Drs. Edwin
Horowitz, Malcolm Brenner and others at St. Jude's Children's Hospital In
Memphis have recently reported promising results with the cells in treating
children with severe brittle bone disease. Some of the cells go to lung and
become lung cells. Therefore, they can potentially be useful in treating
diseases of the lung such as cystic fibrosis. Some of the cells go to
joints and
cartilage and become cells of joints and cartilage. Therefore, they can
potentially be used to treat diseases of the joint such as arthritis. Some
preliminary data suggest that in the future the cells may also be useful in
treating additional diseases such as diabetes, heart disease and kidney
disease.
One of the most dramatic observations about such cells is that they can become
new cells of the brain. The first observations here were made one and two
years
ago in my laboratory primarily by Dr. Asum Azizi and Dr. Donald Phinney. In
the
past several months these observations have been extended by Dr. Ira Black and
his associates at the Robert Wood Johnson Medical School and by research
groups
at two other medical schools. Because the cells can become some of the
cells of
the central nervous system, my laboratory and others are currently trying
to see
if they can be used to treat disease of the central nervous system such as
Parkinsonism, Alzheimer's disease, stroke, spinal cord injury and multiple
sclerosis. In fact, we have recently published some promising results using
the
cells in a rat model of Parkinsonism.
In closing I would like to make two general statements. One is, yes, it is
true
that if the work that my laboratory and many others are now doing continues to
be successful, it will open the possibility of using stem cells from
adults, or
even from the patient himself or herself, to treat a large number of terrible
diseases. If successful, the therapies will not use any fetal tissues and
probably will not use any viruses.
However, I would like to stress the second very important point: we are not
there yet. We have a long way to go. In my estimation, it will be at least two
years before the first adult stem cells can be tested in patients with
diseases
such as Parkinsonism. It will probably be much longer for testing the cells in
patients with the other diseases I have mentioned. We simply cannot be certain
in advance which therapies will work and which will not. We need several years
of hard work and we need continuing support from sources such as the National
Institutes of Health and other sources such as the Louisiana State Consortium
for Gene Therapy and the Columbia Healthcare Association that are currently
supporting the Tulane Center for Gene Therapy. In my opinion, it would be a
serious mistake to stop all research on human embryonic cells and tissues
because of the exciting discoveries my laboratory and others have recently
made
about adult stem cells. We are simply not ready for a moon shot-like
strategy in
which we place all our bets on adult stem cells. I think it would be a mistake
to tell the millions and millions of people whose lives are being destroyed by
these terrible diseases that they to wait three, four, five years or even
longer
until we see the results obtained with adult stem cells before we even begin
doing research on the other kinds of tissues and cells that may cure their
diseases. I myself would be extremely sorry to see this sub committee or any
other group decide that because of the work we and others have done on marrow
stem cells, the kinds of research called for in the new NIH guidelines
should be
stopped.