Stem Cell Transplant
One Year Update
List of Treatable Diseases
Health Monitoring Equipment
April 2008: These are the latest articles on Stem Cell
Transplant research from Northwestern University
Hematopoietic stem cell transplantation for autoimmune
diseases: What have we learned?
Clinical Applications of Blood-Derived and Marrow-Derived
Stem Cells for Nonmalignant Diseases
October 2008: I am pleased to announce that we have been joined
by a transplantee who had Cancer. Her transplant had to be done
with donor cells, and she is very willing to assist Cancer patients
who can take advantage of a stem cell transplant. Contact me and
I will get you in touch with her.
August 2008: I have past my three year anniversary. I have resumed
most of my activities, and am leading a nearly normal life.The
traveling continues, and the book is nearly ready! More news
on that soon.
April 2008. The ASSIST trial is now primarily being conducted
by Dr. Richard K. Burt at Northwestern University in Chicago
at this time. More exciting news on new facilities will be
forthcoming ,hopefully by the end of the year.
I have sent several patients there, and all have felt that
the experience was rewarding.
I reached my two year anniversary in August, 2007. I am
feeling quite well. I did a fair amount of traveling this
summer, Colorado, South Dakota, Minnesota, Michigan, New
York State, Vermont, Maine, New Hampshire, and Ottawa Canada.
The Colorado trip was a bit daunting, and I did need oxygen
at those altitudes. In fact, I was the only comfortable
one in my party. I am breathing normally and comfortably
at home with no supplemental oxygen. I am beginning to cut
down some medications, and best of all, I am resuming some
of my previous activities, such as quilting and singing.
I attend Pilates class three times a week which greatly
helps my breathing. I will be resuming pulmonary rehab
in the fall, since it only helps my situation. I
continue to speak to any groups who would like to hear
about my success, in an effort to reach others.
A couple of things which may help: If you have
Raynauds, use an oven mitt to get things from the freezer. Also
as far as gloves are concerned, get gloves that are bigger than
your hand. Then, get a thin cotton pair of gloves and use them
as liners. You can take the liners out and wash them.A
thin cotton pair used as liners will help to keep your hands
warm in bitter cold for a comfortable amount of time.
If you have lung problems, such as Pulmonary Fibrosis, etc.,
there are not stem cell trials yet for these diseases, but
there are other clinical trials going on. I plan to have some
information up on the site soon, but in the meantime, take a
look at clinicaltrials.gov to get a feel for what is out there.
The Type II diabetes study is now in trial.
An article in December issue of Scientific American gives
some of the history. More information: clinicaltrials.gov
December 2006: "Arthritis and Rheumatism" published a
comparison of the stem cell trials for Scleroderma,
comparing SCOT, ASSIST and ASTIS trials.
Review of Stem Cell Transplant Trials
*Type I Diabetes SCT coming soon
*Heart Failure SCT in Trial
*Incontinence SCT Available
*Regenerative Medicine (knee replacement) in trial
REGENERATIVE MEDICINE IN THE NEAR FUTURE
Gary S. Friedman, MD
Dr. Friedman is a Kidney Specialist and Cell
Therapies Physician in West Orange, NJ. He is also
Prinicipal Science Administrator of NJ Stem Cell
Research and Education Foundation.
Age invites infirmity. Some body parts"wear out," are
damaged or destroyed by disease, non use, or trauma.
The struggle of those in medicine has been to harness
or redirect the natural healing processes of the body
and alter the course of disease and repair traumatic
injuries. It is ironic that the journey toward longevity
and health has redirected scientists to the beginnings
of life and the regenerative capacity of the stem cell.
In our lives, we will each experience illness and frailty
as the result of decline of cellular functions. It is
now recognized that while some organs have the ability
to heal and even regenerate themselves, our bodies also
rely upon the mobilization of stem cells from our bone
marrow to aid in the healing process. This potential for
cell regeneration is limited and declines as we age.
The search for alternative sources of regenerative stem
cells has converged upon a number of potential sources:
umbilical cord blood and placenta
Extraction of stem cells from bone marrow and peripheral
blood can be invasive and costly procedures which may
carry significant risks. Embryonic stem cells are not
currently a viable option due to patients, product
liability, potential medical malpractice, legislative
and bioethical issues encumbering their use. Liposuction
yields a significant number of stem cells and successful
extraction of them in large, viable quantities is still
Yet, there are 4 million births and nearly 1 million joint
replacement surgeries in the US annually. If routinely
collected, bone marrow from joint replacement surgery and
stem cells from umbilical cord blood and the placenta would
provide unlimited and renewable reserve of stem cells
for all persons in need. These facts underlie the basis
for the recently passed US Federal legislation goals for
Stem Cell banking (US Senate Bill 1317):
Development of an aggregate 150,000 unit bank of
stem cells based in the United States to serve as a
reservoir for stem cell transplantation for patients
with cancer, anemia and metabolic disorders;
Make this reservoir of stem cells units available
for treatment of military personnel or civilians injured
Support research and development of stem cell
therapies to regenerate lost cellular function.
With such a bank, there is nearly a guarantee that each
individual would have multiple tissue-matched stem cells
available whenever needed. Equally important, patients
from ethnic groups or age groups (eg, the elderly,
Asian-Americans, African-Americans, Hispanic-Americans)
which currently have great difficulty finding appropriate
tissue-matches would more easily find the life-saving
stem cell products they need.
Regenerative medicine is a burgeoning branch of medicine
that focuses upon the concept of physiologic "healing"
and restoration of function. Since the first successful
human stem cell transplant in the US in 1968, the abilities
of stem cells to 1. restore a functioning immune system after
chemotherapy; 2. incorporate themselves into functioning
major organs; and 3. "reset" the immune system and reduce
chronic inflammatory states has been cataloged.
Currently, the National Institutes of Health has
established a national database to follow the
immunologic and genomic impact of autoimmune diseases,
pregnancy and stem cell transplant upon patients.
Current uses of stem cells for cancer treatment only
"scratches the surface" of clinical utility. Publications
regarding stem cells and their potential uses for treatment
of stroke, heart disease, liver disease, arthritis,
diabetes,and other common conditions have begun to
permeate medical and scientific literature. Stem cells
represent a potential "treasure chest" for treatment
of a multitude of diseases. Regenerative Medicine
targets reversal of the root cause of disease:
loss of cell function.
Arthritis is one of the leading causes of disability
amongst Americans. There are millions of new cases
of Arthritis diagnosed annually in the US and it
is one of the most frequent causes of personal and
work-related disability. Joint replacement is the
treatment of choice when medical and surgical modalities
fail. The success of joint replacement is tempered
by the realities of perioperative morbidity and mortality
as well as the finite longevity of all joint protheses.
Stem cell-based regeneration of synovial tissue
may delay or eliminate the need for joint replacement
Heart Disease remains one of the leading causes of
morbidity and mortality amongst Americans. There are
500,000 new cases of congestive heart failure (CHF)
diagnosed annually in the US. CHF is the single most
utilized hospital admission diagnosis in the state of NJ
and one of the most expensive. Currently, whole-organ
heart transplantation is the treatment of choice when
medical and interventional cardiology modalities fail.
The success of heart transplantation is tempered by the
realities of organ rejection and the adverse effects
of immunosuppressants. Stem cells transforming into
new, functional myocardiocytes could stave off the
need for heart transplantation and supplement existing
medical treatment for patients with CHF.
Autoimmune diseases such as Scleroderma have always been
intriguing and humbling diseases to understand and
treat--they often have minds of their own, flaring for
inexplicable reasons and quieting down for unapparent reasons.
2006--Where Are We Now?
Because of the courage of some of you, I have been
privileged to be able to witness the benefits of stem
cells in the treatment of this awesome illness. As a
transplant physician, my awakening came in 1998. Faced
with the reality the more and more people needed life-saving
or life-enhancing transplants, the American Society of
Transplantation began to invite some of the most prominent
human stem cell researchers to present to nearly
5,000 transplant physicians.We were left with these
thoughts about stem cells:
Could this be our way through the heart, liver, and pancreas
Could these cells be used to coax the immune system into
accepting a donor's transplanted organ?
Could these cells be used to replace brain, spinal
cord and other as yet untransplantable organs?
Could the expertise of organ transplanter and
oncology/stem cell transplanters bring stem cells safely
to human clinical trials?
So where are we now in 2006? First and foremost, at the
highest levels of government, the stem cell "shot heard
round the world" was fired that last week of December 2005
when the US Senate passed Senate Bill 1317--Bone Marrow and
Cord Blood Cell Transplant Program. The value of stem cells
in the treatment of human diseases is now being realized in
over 100 different diseases. As of 2005, human clinical
treatment of SCLERODERMA with stem cells is a real option
for those who have not sufficiently benefited from medications
used in the treatment of Scleroderma.
Microchimerism: An Investigative Frontier in Autoimmunity and Transplantation
Kristina M. Adams, MD
J. Lee Nelson, MD
Author Affiliations: Program in Human Immunogenetics, Clinical Research
Division, Fred Hutchinson Cancer Research Center (Drs Adams and Nelson),
Department of Medicine, Rheumatology (Dr Nelson), and Department of
Obstetrics and Gynecology (Dr Adams), University of Washington School of
Corresponding Author: J. Lee Nelson, MD, Program in Human Immunogenetics, D2-
100, Fred Hutchinson Cancer Research Center, 1100 Fairview Ave N,
Seattle, WA 98109-1024 (firstname.lastname@example.org).
STEM CELL TRANSPLANTATION FOR SCLERODERMA
Ann E. Traynor, MD
UMASS Medical Center
Dr. Traynor and Dr. Burt participate in the ASSIST trial;
several centers across the US participate in the SCOTT trial.
It is important to know that your center has
experience with HSCT for Scleroderma and to ask about any deaths
among transplant patients.
At Northwestern University and the University of
Massachusetts over the last 3 years, Ann Traynor and
Richard Burt and their colleagues have performed 11 stem
cell transplants for scleroderma. All of the 11 patients are
alive, and all have been able to discontinue using oxygen
and all have markedly improved skin scores. This single
armed trial continues to treat patients at this time. We will
soon open a “randomized trial” comparing transplant to
treatment with cytoxan. Individuals who are randomized
(assigned to one treatment or the other randomly, as by
flipping a coin) to standard cytoxan and fail to respond well
to the cytoxan will be able to proceed with stem cell
While our anecdotal evidence thus far suggests that stem
cell transplant may offer hope for scleroderma and its
progression, the only way to be sure that this treatment is
truly superior to the treatments that are more commonly
available, and that it is acceptably safe, is to compare the
outcomes of similar patients treated by stem cell
transplant or treated by another reasonably aggressive
The Food and Drug Administration encourages us to
perform a “randomized trial” to compare two treatments in
order to determine the relative safety and efficacy of the
stem cell transplant approach. Randomization is like
flipping of a coin, where each patient gets assigned to
either the transplant arm or to the more standard,
aggressive treatment. It goes without saying that, if one
arm begins to show clear superiority and safety, the study
should be terminated and that treatment should become
From this type of comparison the general public, the FDA
and all scleroderma patients can appreciate whether one
treatment is superior and is sufficiently safe. Ethically there
are two major concerns with a trial of this kind: first, that
individuals with scleroderma must not be encouraged to
enter treatments that are unduly risky, because they are
“desperate to find something that works” and two, that, if
stem cell transplant does appear more effective and safe in
such a comparison, that individuals be allowed to move
quickly from the “standard of care” treatment to stem cell
transplant before their disease progresses.
Currently the FDA has approved two randomized,
comparative trials for scleroderma, both of which compare
stem cell transplant to cytoxan alone, in the United States.
Different transplant centers choose to participate in one
trial of the other, largely based on their opinion of the
preparative regimen (the high dose therapy) used in the
transplant arm. The two trials are named the ASSIST trial
and the SCOT trial.
|Both trials compare:
||HSCT vs standard Cytoxan
||HSCT vs standard Cytoxan
|They differ in high dose Therapies given
|| High dose Cytoxan and Campath antibody
|| High dose Cytoxan and Total Body Irradiation
|| Not yet