Treating diabetes, cerebral palsy and heart disease with cord blood
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cord blood
Cord blood use is being trialed in the treatment of type 1 diabetes, heart disease and cerebral palsy.
Type 1 Diabetes
Type 1 Diabetes is also known as juvenile diabetes. It is an autoimmune disease caused by the body's own immune system attacking and destroying the insulin producing cells in the pancreas. Insulin allows the body to process sugar to create energy, and without insulin, the body literally starves as it cannot process food.
Type 1 Diabetes affects more than 140,000 people in Australia alone and, while it can be managed, at present it cannot be cured. As a result, it is a lifelong and often disabling disease that can severely impact the quality of life of those who are afflicted.
Researchers are looking at a wide range of potential cell-based therapies and the use of autologous umbilical cord blood as a source of immunomodulatory cells for the treatment of autoimmune diseases has become increasingly popular7-10. Umbilical cord blood contains a population of immature but highly functional regulatory T-cells (Tregs)11. These regulatory T-cells could, in theory, limit inflammatory cytokine responses and energize effector T-cells, which are thought to play a key role in autoimmune processes12,13.
In the laboratory infusion of human blood stem cells into diabetic animals has demonstrated a reversal of the disease2,3. The potential of such cells to provide a source of safe and effective immunomodulation may be of the greatest importance in treating type 1 diabetes4-6. As such, umbilical cord blood Tregs have become a major focus in designing cell-based therapies for children with Type 1 Diabetes14
Cord blood and cerebral palsy
Cerebral palsy (CP) is a permanent physical condition that affects movement and muscle coordination. It results from damage to part of the brain, usually before birth. In Australia it is estimated that a child is born with cerebral palsy every 15 hours and despite advances in medical science the incidence of CP has not declined.
Babies most at risk of cerebral palsy are those born prematurely or with low birthweight. While the reasons for this remain unclear, cerebral palsy may occur as a result of problems associated with preterm birth or may indicate an injury has occurred during the pregnancy that has caused the baby to be born early. For most people with cerebral palsy, the cause is unknown.
Except in its mildest forms, it is usually diagnosed within the first 12-18 months of life. The early signs are a lack of muscle coordination when performing voluntary movement, walking with one foot or leg dragging, walking on the toes or muscle tone that is either too stiff or too floppy.
Cerebral palsy cannot be cured; to date, treatment plans for children have focused on improving a child's physical and mental functioning through physical, occupational, speech and behavioural therapy. Additional treatment for CP has included special braces to compensate for muscular imbalances, mechanical and communication aids as well as surgery.
However, there is now some exciting research regarding the use of cord blood cells for people with cerebral palsy taking place around the world. Researchers at Duke University, USA are looking at infants and children diagnosed with cerebral palsy and infusing them with their own cord blood. Parents, therapists and researchers are observing dramatic improvements in the motor and speech skills of the children with cerebral palsy, in some cases within a few days of being treated with their own cord blood extracted at birth. Additional trials are underway in the US, Europe and a trial is expected to commence in Australia mid-2011.
Real life Stories
Videos 1 and 2: These two amazing videos show the dramatic changes that Maia Friedlander, a young New Zealand girl with cerebral palsy, underwent after receiving stem cell therapy using her own cord blood. The cord blood was inserted by Dr Joanna Kurtzberg at Duke University and within two days Maia's parents began to notice significant changes.
Video 3: At 8 months old Dallas Hextell was diagnosed with Cerebral Palsy. Unable to communicate or control his body, conventional therapy had had little impact. Dallas was accepted into the Duke University clinical trial and this video shows the remarkable improvement of Dallas after receiving his own cord blood.
Video 4: Chloe Levine underwent an infusion of her own cord blood stem cells to treat cerebral palsy. Her progress since the infusion has been remarkable and this video shows how cord blood changed her life.
Video 5: This video features two children Emma Jabs and Alyssa Dupuis, who received infusions of their own stem cells to treat cerebral palsy.
Cord blood and heart disease
Cardiovascular disease (CVD) refers to all diseases of the heart and blood vessels. Affecting more than 3.7 million Australians, it is the leading cause of death in Australia and is one of Australia’s largest health problems.
Heart failure occurs when cardiac tissue is deprived of oxygen. If the damage is significant then there is a loss of cardiac muscle cells, which in turn results in a variety of events including formation of scar tissue, thinning of the heart walls, increased blood flow and pressure, heart failure and eventually death.
Although there are a vast array of medications, plus a range of surgical options available to treat heart disease neither approach actually addresses the loss of function in the damaged tissues. Researchers are now exploring potential therapies using various stem cell sources (including cord blood) to repair or replace damaged tissue.
Results to date have shown that in animal models, cord blood stem cells can move to injured cardiac tissue and improve both vascular function and blood flow at the site of injury, with an overall improvement in heart function. In other studies researchers have shown that cord blood stem cells showed strong growth potential for engineered vascular grafts that could be used help treat heart defects. Although more research needs to be done, scientists believe cord blood stem cells may have a future role in treating children born with congenital heart defects.
1. International Diabetes Federation. Facts & figures: diabetes prevalence. http://www.idf.org/home/index.cfm?node=264. Accessed September 15, 2007
2. Beilhack GF et al: Purified allogeneic hematopoietic stem cell transplantation blocks diabetes pathogenesis in NOD mice. Diabetes 2003;52:59–68 .
3. Hess D et al: Bone marrow-derived stem cells initiate pancreatic regeneration. Nat Biotechnol 2003;21:763–770
4. Limbert C et al: Beta-cell replacement and regeneration: Strategies of cell-based therapy for type 1 diabetes mellitus. Diabetes Res Clin Pract 2008;79:389–399.
5. Hussain MA et al: Stem-cell therapy for diabetes mellitus. Lancet 364:203–205.
6. Couri CE et al: Secondary prevention of type 1 diabetes mellitus: stopping immune destruction and promoting beta-cell regeneration. Braz J Med Biol Res 2006;39:1271–1280
7. Ende N et al: Effect of human umbilical cord blood cells on glycemia and insulitis in type 1 diabetic mice. Biochem Biophys Res Commun 2004;325:665–669.
8. Haller M et al: Insulin requirements, HbA1c, and stimulated C-peptide following autologous umbillical cord blood transfusion in children with type 1 diabetes (Abstract). Diabetes 2007;56(Suppl. 1):A82.
9. Viener H et al: Changes in regulatory T cells following autologous umbillical cord blood transfusion in children with type 1 diabetes (Abstract). Diabetes 2007;56(Suppl. 1):A82.
10. Voltarelli JC et al: Autologous nonmyeloablative hematopoietic stem cell transplantation in newly diagnosed type 1 diabetes mellitus. JAMA 2007;297:1568–1576
11. Godfrey WR et al: Cord blood CD4(+)CD25(+)-derived T regulatory cell lines express FoxP3 protein and manifest potent suppressor function. Blood 2005;105:750–758
12. Fruchtman S: Stem cell transplantation. Mt Sinai J Med 2003;70:166–170.
13. Han P et al: Phenotypic analysis of functional T-lymphocyte subtypes and natural killer cells in human cord blood: relevance to umbilical cord blood transplantation. Br J Haematol 1995;89:733–740
14. Haller MJ et al: Autologous umbilical cord blood infusion for type 1 diabetes. Exp Hematol 2008;36:710–715
Type 1 Diabetes
Type 1 Diabetes is also known as juvenile diabetes. It is an autoimmune disease caused by the body's own immune system attacking and destroying the insulin producing cells in the pancreas. Insulin allows the body to process sugar to create energy, and without insulin, the body literally starves as it cannot process food.
Type 1 Diabetes affects more than 140,000 people in Australia alone and, while it can be managed, at present it cannot be cured. As a result, it is a lifelong and often disabling disease that can severely impact the quality of life of those who are afflicted.
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Type 1 diabetes - Fast Facts
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Researchers are looking at a wide range of potential cell-based therapies and the use of autologous umbilical cord blood as a source of immunomodulatory cells for the treatment of autoimmune diseases has become increasingly popular7-10. Umbilical cord blood contains a population of immature but highly functional regulatory T-cells (Tregs)11. These regulatory T-cells could, in theory, limit inflammatory cytokine responses and energize effector T-cells, which are thought to play a key role in autoimmune processes12,13.
In the laboratory infusion of human blood stem cells into diabetic animals has demonstrated a reversal of the disease2,3. The potential of such cells to provide a source of safe and effective immunomodulation may be of the greatest importance in treating type 1 diabetes4-6. As such, umbilical cord blood Tregs have become a major focus in designing cell-based therapies for children with Type 1 Diabetes14
Cord blood and cerebral palsy
Cerebral palsy (CP) is a permanent physical condition that affects movement and muscle coordination. It results from damage to part of the brain, usually before birth. In Australia it is estimated that a child is born with cerebral palsy every 15 hours and despite advances in medical science the incidence of CP has not declined.
Babies most at risk of cerebral palsy are those born prematurely or with low birthweight. While the reasons for this remain unclear, cerebral palsy may occur as a result of problems associated with preterm birth or may indicate an injury has occurred during the pregnancy that has caused the baby to be born early. For most people with cerebral palsy, the cause is unknown.
Except in its mildest forms, it is usually diagnosed within the first 12-18 months of life. The early signs are a lack of muscle coordination when performing voluntary movement, walking with one foot or leg dragging, walking on the toes or muscle tone that is either too stiff or too floppy.
Cerebral palsy cannot be cured; to date, treatment plans for children have focused on improving a child's physical and mental functioning through physical, occupational, speech and behavioural therapy. Additional treatment for CP has included special braces to compensate for muscular imbalances, mechanical and communication aids as well as surgery.
However, there is now some exciting research regarding the use of cord blood cells for people with cerebral palsy taking place around the world. Researchers at Duke University, USA are looking at infants and children diagnosed with cerebral palsy and infusing them with their own cord blood. Parents, therapists and researchers are observing dramatic improvements in the motor and speech skills of the children with cerebral palsy, in some cases within a few days of being treated with their own cord blood extracted at birth. Additional trials are underway in the US, Europe and a trial is expected to commence in Australia mid-2011.
Real life Stories
Videos 1 and 2: These two amazing videos show the dramatic changes that Maia Friedlander, a young New Zealand girl with cerebral palsy, underwent after receiving stem cell therapy using her own cord blood. The cord blood was inserted by Dr Joanna Kurtzberg at Duke University and within two days Maia's parents began to notice significant changes.
Video 3: At 8 months old Dallas Hextell was diagnosed with Cerebral Palsy. Unable to communicate or control his body, conventional therapy had had little impact. Dallas was accepted into the Duke University clinical trial and this video shows the remarkable improvement of Dallas after receiving his own cord blood.
Video 4: Chloe Levine underwent an infusion of her own cord blood stem cells to treat cerebral palsy. Her progress since the infusion has been remarkable and this video shows how cord blood changed her life.
Video 5: This video features two children Emma Jabs and Alyssa Dupuis, who received infusions of their own stem cells to treat cerebral palsy.
Cord blood and heart disease
Cardiovascular disease (CVD) refers to all diseases of the heart and blood vessels. Affecting more than 3.7 million Australians, it is the leading cause of death in Australia and is one of Australia’s largest health problems.
Heart failure occurs when cardiac tissue is deprived of oxygen. If the damage is significant then there is a loss of cardiac muscle cells, which in turn results in a variety of events including formation of scar tissue, thinning of the heart walls, increased blood flow and pressure, heart failure and eventually death.
Although there are a vast array of medications, plus a range of surgical options available to treat heart disease neither approach actually addresses the loss of function in the damaged tissues. Researchers are now exploring potential therapies using various stem cell sources (including cord blood) to repair or replace damaged tissue.
Results to date have shown that in animal models, cord blood stem cells can move to injured cardiac tissue and improve both vascular function and blood flow at the site of injury, with an overall improvement in heart function. In other studies researchers have shown that cord blood stem cells showed strong growth potential for engineered vascular grafts that could be used help treat heart defects. Although more research needs to be done, scientists believe cord blood stem cells may have a future role in treating children born with congenital heart defects.
1. International Diabetes Federation. Facts & figures: diabetes prevalence. http://www.idf.org/home/index.cfm?node=264. Accessed September 15, 2007
2. Beilhack GF et al: Purified allogeneic hematopoietic stem cell transplantation blocks diabetes pathogenesis in NOD mice. Diabetes 2003;52:59–68 .
3. Hess D et al: Bone marrow-derived stem cells initiate pancreatic regeneration. Nat Biotechnol 2003;21:763–770
4. Limbert C et al: Beta-cell replacement and regeneration: Strategies of cell-based therapy for type 1 diabetes mellitus. Diabetes Res Clin Pract 2008;79:389–399.
5. Hussain MA et al: Stem-cell therapy for diabetes mellitus. Lancet 364:203–205.
6. Couri CE et al: Secondary prevention of type 1 diabetes mellitus: stopping immune destruction and promoting beta-cell regeneration. Braz J Med Biol Res 2006;39:1271–1280
7. Ende N et al: Effect of human umbilical cord blood cells on glycemia and insulitis in type 1 diabetic mice. Biochem Biophys Res Commun 2004;325:665–669.
8. Haller M et al: Insulin requirements, HbA1c, and stimulated C-peptide following autologous umbillical cord blood transfusion in children with type 1 diabetes (Abstract). Diabetes 2007;56(Suppl. 1):A82.
9. Viener H et al: Changes in regulatory T cells following autologous umbillical cord blood transfusion in children with type 1 diabetes (Abstract). Diabetes 2007;56(Suppl. 1):A82.
10. Voltarelli JC et al: Autologous nonmyeloablative hematopoietic stem cell transplantation in newly diagnosed type 1 diabetes mellitus. JAMA 2007;297:1568–1576
11. Godfrey WR et al: Cord blood CD4(+)CD25(+)-derived T regulatory cell lines express FoxP3 protein and manifest potent suppressor function. Blood 2005;105:750–758
12. Fruchtman S: Stem cell transplantation. Mt Sinai J Med 2003;70:166–170.
13. Han P et al: Phenotypic analysis of functional T-lymphocyte subtypes and natural killer cells in human cord blood: relevance to umbilical cord blood transplantation. Br J Haematol 1995;89:733–740
14. Haller MJ et al: Autologous umbilical cord blood infusion for type 1 diabetes. Exp Hematol 2008;36:710–715
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