Umbilical Cord Blood Infusion to Treat Type 1 Diabetes
Umbilical Cord Blood Infusion to Treat Type 1 Diabetes
While this study is now completely enrolled, we do hope to develop a "next generation" cord
 blood based study sometime in early 2009. Please continue to contact us if you have a child
 with newly diagnosed Type 1 Diabetes (T1D) who alo has their OWN cord blood in storage.
Objective:

 Our goal is to transfuse autologous umbilical cord blood into 23 children with T1D in an
 attempt to re-establish immune tolerance and perhaps regenerate pancreatic islet
 insulin-producing beta cells and improve blood glucose control. As secondary goals, we aim to
 study the potential changes in metabolism/immune function leading to islet regeneration.

 Background/Rationale:

 Stem cells provide an exciting approach towards curing T1D. Autologous bone marrow
 transplants have been used successfully for patients undergoing high dose chemotherapy, and
 for a variety of cancers and autoimmune disorders such as multiple sclerosis, lupus, and
 rheumatic disorders. Recent studies in immunodeficient mice with chemically-induced
 pancreatic damage have shown that bone marrow-derived stem cells preferentially home to the
 pancreas and may have the capacity to initiate pancreatic regeneration, thereby restoring the
 endothelial interactions in the pancreas and correcting the associated elevated blood sugar
 levels Human umbilical cord blood cells transfused into a model of amyotrophic lateral
 sclerosis (ALS) resulted in delayed disease progression of two to three weeks and increased
 lifespan. Umbilical cord blood has shown promise as an excellent source for deriving stem
 cell populations, and has been used successfully in transplantation for a variety of
 diseases, including acute lymphocytic and myeloid leukemia, lymphoma, Fanconi anemia, and
 sickle cell disease. Furthermore, umbilical cord blood-derived stem cells have the capacity
 to differentiate into a variety of non-blood cell types, including hepatocytes, neural cells,
 and endothelial cells. In addition, umbilical cord blood contains a greater proportion of
 hematopoietic stem cells than bone marrow.

 In addition, cord blood contains a large number of immune cells called regulatory T cells,
 These regulatory T cells may be helpful in diminishing autoimmunity. The need to re-establish
 tolerance in patients with established autoimmunity provides another potential mechanism for
 cord blood as a therapy for type 1 diabetes.

 Description of Project:

 23 children > 1 year of age with T1D and stored umbilical cord blood are being be recruited.
 The cord blood will be infused into the children in the GCRC in an attempt to regenerate
 pancreatic islet insulin-producing beta cells and improve blood glucose control. As secondary
 goals, we aim to track the migration of transfused cord blood stem and study the potential
 changes in metabolism/immune function leading to islet regeneration.

 Anticipated Outcome:

 It is hoped that there will be preservation of beta cell function assessed by mixed meal
 stimulated C-peptide secretion. Changes in immunological markers/function may be observed

 Relevance to Type I Diabetes:

 Type 1 diabetes is still associated with tremendous morbidity and premature mortality.
 Patients require multiple daily insulin injections throughout their lives as well as close
 monitoring of their diet and blood sugar levels to prevent complications. Unfortunately,
 there is presently no permanent cure for diabetes. Whole pancreas or islet cell
 transplantation is available only to a very limited number of patients and necessitates
 potential lifelong immunosuppressive therapy. The need to find a cure for T1D cannot be
 overstated -autologous stem cell transfusions either with their potential to differentiate
 into islet cells or provide immune tolerance that leads to islet regeneration appear to be a
 safe and potentially viable option.
Inclusion Criteria:

 1. Must have a diagnosis of T1D and have stored umbilical cord blood in an AABB and/or
 FACT accredited cord bank.

 2. TID diagnosis will be defined as having a clear history of polydipsia, polyphagia,
 polyuria, and weight loss consistent with a clinical diagnosis, diagnosis will mot be
 based solely upon the presence of autoantibodies.

 3. Cord blood meets all selection and testing criteria (see below).

 4. Able to complete mixed meal tolerance / glucagon stimulation test.

 5. Normal screening values for CBC, Renal function and electrolytes (BMP).

 6. Willing to comply with intensive diabetes management

 Exclusion Criteria:

 1. Complicating medical issues that would interfere with blood drawing or monitoring.

 2. Chronic use of steroids or other immunosuppressive agents for other conditions.

 3. Positive infectious disease markers from mothers' blood or cord at time of collection
 (See below for details).

 4. Any evidence of illness on planned infusion date (i.e. fever >38.5 C, vomiting,
 diarrhea, wheezing, or crackles).