For the child itself (autologous transplantation)

Cerebral Palsy and other brain disorders

Cerebral palsy is a disease of the nervous system which afflicts children up to 2.5 years of age. Children suffer from a range of dysfunctions associated with mobility and balance, and the disease occurs mainly as a result of suffocation and generally poor oxygenation of the brain during birth. Stem cells harvested from umbilical cord blood provide hope for children suffering from this disease. Several autologous hematopoietic stem cell transplantations have been performed on children of up to 2 years of age with great success.

  • An 18-old month child underwent a transplantation of hematopoietic umbilical cord blood stem cells collected at birth. The entire transplantation process lasted only a few hours, while the changes in the behavior of Dallas Hextell were evident immediately after transplantation. The boy began to react to stimuli and make eye contact. Shortly afterwards he was able to say his first word, walk and laugh. His parents now recognize that the stem cells of their child, which they banked “in case they were ever needed”, is giving him a second chance for a happy life.
  • Another little girl with Cerebral Palsy, Chloe Levine, whose right side of the body was severely affected, underwent an autologous umbilical cord blood stem cells  transplantation before she was 1 year old. The positive and immediate changes in her kinetic behavior were impressive. Today she can use her right arm with ease, is able to run rather fast and is attending school normally. Her parents have stated that their decision to bank the stem cells of their newborn daughter was the best decision they ever made!

In parallel with the many transplantations that are currently being conducted around the world, scientists are involved in a number of clinical trials to determine the short and long term results of transplantations of umbilical cord blood stem cells on cerebral palsy and  other brain related issues.  Duke University in the USA has documented significant improvements in the clinical state of 120 children with cerebral palsy and 60 with malformations of the brain after autologous transplantations of hematopoietic stem cells.

Other brain disorders

  • A young girl named Abby Pell was diagnosed with a severe brain injury immediately after birth due to lack of proper oxygenation of the brain during birth. Two months later, Abby had severe lesions in 3 of the 4 lobes of her brain. Her parents had the foresight to have banked her stem cells when she was born. After many attempts to determine whether there was a cure and many contacts with specialist doctors, they were put in contact with Duke University. Duke gave them hope that they could improve Abby’s  condition by using  her own stem cells.  Indeed, Abby was the first child ever who was transplanted her very own umbilical cord blood stem cells to deal with the specific type of brain damage that she had. The transplantation took place in February 2005. Abby’s brain has since shown the best possible improvement, very much in line with what doctors expected and hoped for. Abby is now attending school.

Diabetes

Diabetes mellitus type 1 or insulin dependent diabetes usually occurs during childhood or adolescence. This is an autoimmune disease characterized by the destruction of beta cells in the pancreas that produce insulin, and can result in the progressive loss of beta cells. Several studies report an improvement in the clinical state of patients with diabetes type 1 (> 1 year of age) who have undergone autologous umbilical cord blood transplantations. The improvements are particularly evident in cases when the transplantation was done soon after the diagnosis.

  • The American Diabetes Association (ADA) has reported at their recent annual meeting the successful treatment with stem cells of 15 children suffering from type 1 diabetes. These children are well and alive 1.5 years after having received the pioneer stem cell treatment. The treatment includes transplantation of autologous umbilical cord blood stem cells which are administered after mild chemotherapy.

Retinoblastoma

Retinoblastoma is a cancer that occurs in the eye and grows in the cells of the retina. It usually occurs in  neonatal, infancy or early childhood (up to the age of 5).  An autologous stem cell transplantation was successfully administered to a 7 year old girl who developed retinoblastoma in the left eye with metastases to lymph nodes, bones and bone marrow. The stem cells were harvested from peripheral blood. 

  • Another successful case is that of Jesse Farquharson who was 2-years old when he was administered a transplantation of cord blood stem cells to treat retinoblastoma, which appeared immediately after birth with a metastasis in the cerebrospinal fluid. Although his parents apparently could not find a good reason to bank the stem cells of their newborn child, primarily because when he was born stem cells applications were very limited, they eventually proceeded to bank the stem cells, a decision that saved his life.(I’m not sure I understand this – they banked them at birth or not? Just state whether or not they did it. )Although his vision has deteriorated, the child now lives with no tumors and the state of his health remains stable.

Vision

Successful bone marrow stem cells transplantations for the treatment of blindness are already a fact. In recent years, similar treatments but with umbilical cord blood stem cells have been  carried out on a  specific group of patients with encouraging results so far.

  • Doctors have reported the case of a young girl who was born with congenital blindness (due to malformation of the optic nerve), and who was able to perceive light stimuli after a transplantation of autologous hematopoietic umbilical cord blood stem cells. The treatment was carried out when the girl was 18-months old. The treatment lasted a total of 33 days during which 8 separate stem cells transplants were administered. Nine months later the girl reacted for the very first time to light stimulus, which indicates the optical nerve is beginning to function.

Autism

Autism is a disorder which affects the psychological development of a child and is characterized by difficulties in communication and by a limited, repetitive and stereotypical behavior. Many believe that autism is caused by genetic factors while others argue that the main cause is environmental factors. Early diagnosis of autistic children and prompt therapeutic intervention may improve social and communication skills, but the treatment must be tailor-made to every child depending on the severity and idiosyncrasy of the case. There is no universal approach to the treatment of autistic children.

The treatment with stem cells is geared to create new blood vessels in regions of the brain where the flow of blood is restricted. The administration of autologous hematopoietic stem cells is considered the safest procedure, because the cells are young and more efficient than cells from other sources, and their number is usually sufficient if given immediately after diagnosis. The use of compatible allogeneic stem cells is not indicated because of the risk of acute or chronic rejection and the need to administer immunosuppressive drugs. Moreover, the autologous mesenchymal stem cells can contribute effectively in regulating the immune system in autistic children.

  • The Shandong Jiaotong hospital in China has completed a clinical trial which demonstrates the safety and effectiveness of transplantations with autologous umbilical cord blood stem cells, but also transplantations using a combination of umbilical cord blood with umbilical cord tissue mesenchymal stem cells in children with autism.

Gene Therapy

Gene therapy represents the present and the future in terms of treating a number of genetic diseases. The cure consists firstly of replacing the mutated (abnormal) gene that causes the disease, with the corresponding normal gene, and secondly of transferring the normal gene to the patient's cells. The result is that the normal gene integrates in the genome of the patient.

Recently, the scientific community is exploring various procedures to safely and effectively transfer the genes using stem cells as the “transport vehicle” because of their ability to identify and hone in on  the cells that have suffered damage. The transplantations that have taken place so far are exclusively autologous and have successfully been administered to patients with cancer, thalassemia and other genetic diseases.

The combination of two very promising and revolutionary treatments, gene and stem cell therapies, is expected to provide an alternative solution for the transport of the normal gene, something that is currently accomplished in a sub optimal way with viruses.

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