NIH Launches Study of Hematopoietic Stem Cell Transplantation for Severe, Treatment-Resistant Lupus
Researchers at the Department of Health and Human Services' National
Institutes of Health (NIH) have launched a five-year study to see
whether a therapy using transplantation of hematopoietic stem cells,
blood stem cells found in bone marrow, can produce long-term remission
for patients with severe, treatment-resistant systemic lupus erythematosus
(or lupus), a rheumatic autoimmune disease that can affect the body's
major organs. The study will include a basic research component
to examine the roles of B and T cells, white blood cells in the
immune system, in triggering lupus symptoms.
In this pilot study, 14 patients from ages 15 to 40 will receive
stem cell transplantation therapy, during which their stem cells
will be removed from their bone marrow. These cells, which will
become different kinds of blood and immune system cells in the body,
will then be harvested and cleaned. After the patient's bone marrow
is treated with immunosuppressant drugs to destroy the disease-causing
immune cells, the stem cells will be returned to the bone marrow.
The stem cells will then repopulate the marrow and body to establish
a more properly functioning immune system.
The initial treatment requires several outpatient visits followed
by a two-week hospital stay. Patients then have monthly medical
visits for six months, quarterly visits for two years, and annual
visits for the remainder of the study. Following therapy, researchers
will assess whether this treatment produces sustained, relapse-free
disease remission for 24 months.
"Many patients with severe forms of lupus have limited treatment
options that may offer only temporary relief of symptoms and no
disease regression. For these patients, stem cell transplantation
therapy may offer hope for a normal functioning immune system,"
said Stephen I. Katz, M.D., Ph.D., director of NIH's National Institute
of Arthritis and Musculoskeletal and Skin Diseases (NIAMS).
Severe forms of lupus can devastate patients, causing pain, fatigue,
depression, and in some cases, premature death. Patients who enter
this study must have been treated, to no avail, with high doses
of immunosuppressant drugs, which decrease immune function. Researchers
believe that by combining immunosuppressant treatment with stem
cell transplantation, they can create a new immune system that doesn't
attack the body's healthy cells.
"Advances in cancer therapy can yield advances in treatment
of autoimmune diseases," said Andrew von Eschenbach, M.D.,
director of the NIH's National Cancer Institute. "Cyclophosphamide
used as a cancer therapy suppresses the immune system. This discovery
led to improvements for lupus patients. Similarly, altering the
immune system through progenitor cell transplantation, a cancer
treatment, may also benefit lupus patients."
"Lupus is different from cancer," he added. "However,
we have found that both diseases can benefit from treatment that
modulates the immune system."
In addition to the clinical study, researchers will look at how
B and T cells function in the immune systems of lupus patients.
Previous studies have shown that the dysfunctioning T cells, which
normally regulate the immune response, coupled with overactive B
cells, which target both foreign and healthy cells, are responsible
for the destructive autoimmune process that takes place in lupus.
Researchers will compare the activity of B and T cells extracted
from patients before stem cell transplant therapy with those that
repopulate in the "new" immune system after therapy. They
will be looking for cell properties that may contribute to the faulty
immune response found in lupus. B cell studies will be conducted
by scientists at NIAMS, while T cell studies will be conducted by
scientists at NCI.
Researchers at the National Institute of Neurological Disorders
and Stroke and the National Institute of Diabetes and Digestive
and Kidney Diseases will join this collaborative effort, investigating
the central nervous system and kidney involvement, respectively,
of lupus patients. "This study is an example of what can be
accomplished when scientists from various disciplines combine resources
and experience to create better outcomes for patients," said
Lupus is a chronic, inflammatory, autoimmune disease that mainly
affects women of child-bearing age. Its symptoms range from unexplained
fever, swollen joints and skin rashes to severe damage of the kidneys,
lungs or central nervous system. Lupus is three times more common and
is frequently more severe in African Americans and Hispanics/Latinos.
Studies show these groups also experience more complications of
lupus, including kidney failure for both and neurological problems
for African Americans.
For additional information on this clinical study, please contact:
Patient Recruitment and Public Liaison Office
10 Cloister Court
Bethesda, Maryland 20892-4754
Toll Free: 1-800-411-1222
TTY: (301) 594-9774 (local), 1-866-411-1010 (toll free)
or e-mail at firstname.lastname@example.org
The mission of the National Institute of Arthritis and Musculoskeletal
and Skin Diseases (NIAMS), a part of the Department of Health and
Human Services' National Institutes of Health, is to support research
into the causes, treatment and prevention of arthritis and musculoskeletal
and skin diseases, the training of basic and clinical scientists
to carry out this research, and the dissemination of information
on research progress in these diseases. For more information about
NIAMS, call the information clearinghouse at (301) 495-4484 or (877)
22-NIAMS (free call) or visit the NIAMS Web site at http://www.niams.nih.gov.
The mission of the National Cancer Institute is to coordinate
the National Cancer Program, which conducts and supports research,
training, health information dissemination and other programs with
respect to the cause, diagnosis, prevention and treatment of cancer,
rehabilitation from cancer, and the continuing care of cancer patients
and the families of cancer patients.