Patients have a type of blood cell disorder that is very hard to cure. We are now suggesting
a treatment that might help patients live longer without disease than other treatment plans
would. This treatment is known as a stem cell transplant. We believe this may help patients
as it allows us to give much stronger doses of drugs and radiation to kill the diseased cells
than we could give without the transplant. We also think that the healthy cells may help
fight any diseased cells left after the transplant.
Stem Cells are special "mother" cells that are found in the bone marrow (the spongy tissue
inside bones), although some are also found in the bloodstream (peripheral blood). As they
grow, they become either white blood cells which fight infection, red blood cells which carry
oxygen and remove waste products from the organs and tissues or platelets, which enable the
blood to clot. For the transplant to take place, we will collect these stem cells from a
"donor" (a person who agrees to donate these cells) and give them to recipient. Patients do
not have a sibling that is a perfect match, so the stem cells will come from a donor who is
the best match available. This person may be a close relative or an unrelated person whose
stem cells best "matches" the patients, and who agrees to donate stem cells. Before the
transplant, two very strong drugs plus total body irradiation will be given to the patient
(pre-conditioning). This treatment will kill most of the blood-forming cells in the bone
marrow. We will then give the patient the healthy stem cells. Once these healthy stem cells
are in the bloodstream they will move to the bone marrow (graft) and begin producing blood
cells that will eventually mature into healthy red blood cells, white blood cells and
platelets.
This research study will also use CAMPATH-1H as a pre-treatment. CAMPATH-1H is an antibody
against certain types of blood cells. CAMPATH-1H is important because it stays active in the
body for a long time after infusion, which means it may work longer at preventing GvHD
symptoms.
The stem cell transplant described above is considered to be "standard" treatment. We would
like to collect additional blood as described below in order to evaluate how the immune
system is recovering.
We are asking permission to draw blood from the patient so that we can measure the number of
certain blood cells called T regulatory cells. T regulatory cells are special immune cells
that can control or regulate the body's immune response. We want to determine whether T
regulatory cells are important participants in graft versus host disease (GVHD), infection
and relapse. In GVHD, certain cells from the donated marrow or blood (the graft) attack the
body of the transplant patient (the host). GVHD can affect many different parts of the body.
The skin, eyes, stomach and intestines are affected most often. GVHD can range from mild to
life-threatening. We do not know whether T regulatory cells can modify these conditions. We
want to measure these T regulatory cells and learn if these cells do influence these
conditions. If we learn that T regulatory cells do affect these conditions, then it may be
possible to modify these cells for the benefit of transplant patients.
Phase:
N/A
Details
Lead Sponsor:
Baylor College of Medicine
Collaborators:
Center for Cell and Gene Therapy, Baylor College of Medicine Texas Children's Hospital