What Stem Cell Treatments Exist?

Current Treatments

• Are there current uses for cord blood stem cells?

  Banking cord blood can change or even save a life. Cord blood stem cells have certain advantages over bone marrow stem cells in transplant, and have been used for 20 years to treat more than 80 life-threatening diseases and disorders. Today stem cell therapies continue to evolve, bringing new hope to patients and their families.

  Below are just a few diseases and disorders that have been treated with cord blood stem cells. If you have stem cell treatment questions, please click here to request more information.

  Cancer

  • Acute Leukemia
  • Chronic Leukemia
  • High-Risk Solid Tumors
  • Hodgkin & Non-Hodgkin Lymphoma
  • Myelodysplastic Syndromes

  Blood Disorders

  • Aplastic Anemia
  • Beta Thalassemia
  • Diamond-Blackfan Anemia
  • Fanconi Anemia
  • Sickle Cell Disease

  Immune Disorders

  • Chronic Granulomatous Disease
  • Hystiocytic Disorders
  • Leukocyte Adhesion Deficiency
  • Severe Combined Immunodeficiency Diseases
  • Wiskott-Aldrich Syndrome

  Metabolic Disorders

  • Krabbe Disease
  • Hurler Syndrome
  • Metachromatic Leukodystrophy
  • Sanfilippo Syndrome

  Saving or donating cord blood stem cells makes them available to treat diseases like those listed above. For inherited genetic conditions, the child may not be able to use his or her own stem cells. In these cases, a matched sibling’s stem cells would be the first choice. Only family banking also offers access to current regenerative medicine clinical trials in autism, cerebral palsy, and pediatric stroke.

• How many people have used their cord blood sample from CBR?

  CBR has more experience providing cord blood for use in treatment than any other family bank. To date, we have released more than 600 samples for families to use. All of the cord blood units released for client use have been viable — the ultimate validation of our processing and storage methods.

• Is cord blood being used in medical treatments?

  Over the past 20 years, cord blood stem cells have been used in 30,000 transplants to treat many life-threatening diseases, such as leukemia and other cancers. In transplant medicine, a patient generally will undergo chemotherapy and then receive an infusion of cord blood stem cells to create a healthy blood and immune system.

  In addition, a new field, called regenerative medicine, is evaluating cord blood stem cells’ ability to help repair and replace cells that have been damaged by disease or injury. These are conditions that have no cure today, such as autism, cerebral palsy, and pediatric stroke. These therapies are still experimental and there is no guarantee that treatments will be available.

• Does CBR have academic affiliations with research institutions?

  Yes. CBR has established leadership in advancing clinical research with newborn stem cells. We are the exclusive family bank partner in studies at several prestigious medical institutions, including Sutter Neuroscience Institute in Sacramento, CA, Georgia Regents University in Augusta, GA, and The University of Texas Health Science Center, in Houston (UTHealth).

• What is the difference between a pre-clinical research study and a clinical trial?

  Clinical trials are experimental treatments using human subjects. They are FDA-regulated and conducted by physicians and medical institutions. Pre-clinical laboratory research uses animals and in vitro studies and is performed prior to clinical trials.

• How does the acquired hearing loss study work?

  Florida Hospital for Children and Cord Blood Registry^® (CBR) are launching a FDA-regulated, Phase I safety study of the use of cord blood stem cells to treat children with sensorineural hearing loss.

  Approximately 15 percent of children in the U.S. suffer from low or high-frequency hearing loss. The most common type of hearing loss, especially at high frequencies, is sensorineural. Acquired sensorineural hearing loss results from damage to hair cells in the inner ear (cochlea) and can be caused by illness, medication, noise exposure, birth injury, or head trauma.

  “Babies are surviving prematurity in historically high numbers today, and with this advance we are seeing more young children born with acquired hearing loss. To date, there are no treatments to repair a damaged inner ear,” said James Baumgartner, MD, Surgical Director of Florida Hospital for Children’s Comprehensive Pediatric Epilepsy Center, and the study’s principal investigator. “Although hearing aids and cochlear implants provide valuable access to sound, they do not restore the complexity of a fully functioning biological ear. Using cord blood stem cells to trigger the body’s own repair mechanisms could provide a non-invasive avenue to normal hearing.”

  The study will enroll 10 children between the ages of 6 weeks and 6 years old, who meet the inclusion criteria for the study. Children with genetic deafness are ineligible for study participation. To ensure consistency in cord blood stem cell quality, CBR is the only family stem cell bank providing units from clients for the study.

• How does the autism study work?

  The study will enroll 30 children between the ages of two and seven, who meet the inclusion criteria for the study. Enrolled participants will receive two infusions—one of the child’s own cord blood stem cells and one of a placebo—over the course of 13 months. Both the participants and the lead investigators will be blinded from knowing the content of each infusion. To ensure consistency in cord blood stem cell quality, CBR is the only family stem cell bank providing units from clients for the study.

  Sutter Neuroscience Institute in Sacramento, California, and Cord Blood Registry have launched the first FDA-regulated clinical trial to assess the use of a child’s own cord blood stem cells to treat select patients with autism. This first-of-its-kind placebo-controlled study is important because 1 in 88 children in the U.S. are diagnosed with autism spectrum disorders each year. The clinical trial will evaluate the ability of an infusion of cord blood stem cells to help improve language and behavior in children with autism.

  “Autism is a growing public health issue. This study goes beyond treating symptoms to understanding how stem cells may initiate repair or healing in chronic conditions like cerebral palsy or autism,” stated Dr. Chez, director of pediatric neurology at Sutter Medical Center and principal study investigator. “We have evidence to suggest that certain children with autism have dysfunctional immune systems that may be damaging or delaying the development of the nervous system,” continued Dr. Chez. “Cord blood stem cells may offer ways to modulate or repair the immune systems of these patients, which would also improve language and some behavior in children who have no obvious reason to have become autistic.”

• How does the cerebral palsy study work?

  Cerebral palsy, caused by a brain injury or lack of oxygen in the brain before birth or during the first few years of life, can impair movement, learning, hearing, vision, and cognitive skills. According to the Centers for Disease Control and Prevention, it is estimated that roughly 1 in every 323 children in the United States has been diagnosed with cerebral palsy.

  Georgia Regents University

  Researchers at Georgia Regents University, in partnership with Cord Blood Registry, are conducting a landmark FDA-regulated clinical trial to evaluate the use of a cord blood stem cell infusion for the treatment of cerebral palsy in children.

  Dr. James Carroll, the principal investigator of the study, notes that “autologous stem cell transplantation, in which the transplant recipient is also the donor, is the safest form of stem cell transplantation because it carries virtually no threat of immune system rejection.”

  For more information on this clinical trial, sign up here.

  How does the study work?

  Recruiting efforts are underway to enroll 40 children, between the ages of 1 and 12, diagnosed with cerebral palsy. Any level of cerebral palsy severity will be allowed. The subjects will be children whose parents have saved their infant’s cord blood, who have clinical evidence of a non-progressive motor disability, and whose parents intend to have a cord blood infusion.

  To ensure consistency in cord blood stem cell quality, CBR is the only family stem cell bank providing units from clients for the study. Study participants must have been unable to sit independently by 12 months or unable to walk by 18 months and must be seizure-free or have seizures that are adequately controlled.

  University of Texas Health Science Center at Houston

  Researchers at The University of Texas Health Science Center at Houston, in collaboration with Cord Blood Registry, are commencing an innovative FDA-regulated clinical trial to investigate two forms of stem cell therapy in children diagnosed with cerebral palsy. The study aims to compare the safety and efficacy of an intravenous infusion of banked cord blood stem cells to freshly harvested bone marrow stem cells.

  Dr. Charles Cox, the principal investigator of the study, notes that “there is preclinical data indicating that the ongoing neuroinflammatory response is a driver of further injury in CP so the hope is to reduce this neuroinflammation.” “Our goal is to break the cycle of inflammation and injury,” adds Cox.

  For additional information regarding this clinical trial, sign up here.

  How does the study work?

  Recruiting efforts are underway to enroll 30 children, between the ages of 2 and 10, diagnosed with cerebral palsy: 15 with a CBR processed and stored cord blood unit who may be administered an autologous stem cell infusion and 15 children who may undergo a bone marrow harvest and autologous stem cell infusion. To evaluate improvement in functional status among participants, five children in each group will be randomized to a placebo control group during the baseline/treatment visit.

  Parents will not be informed if their child received stem cells or were given a placebo until the 1-year follow-up examination. At that time, parents whose children were administered the placebo may elect to have their child receive the stem cell treatment, either through bone marrow harvest or cord blood. In order to be eligible to participate in the cord blood arm of the trial, families must have a qualified CBR processed unit that was collected at birth.

• How does the pediatric stroke study work?

  The trial will enroll 10 children who have their umbilical cord blood banked with Cord Blood Registry, have experienced a perinatal stroke and who meet the inclusion criteria for the study. Children up to 6 years of age who suffered a perinatal stroke and who have access to their own CBR-processed cord blood cells may be eligible for the study. Please contact us if your child meets these criteria and is suffering from the effects of a stroke.

  Families who have saved their children’s cord blood with Cord Blood Registry^® now have access to a new use for the stem cells. A new clinical trial will investigate using a child’s own cord blood stem cells as treatment after a pediatric stroke. Below are details on how Cord Blood Registry is helping uncover new potential uses for cord blood stem cells.

  While stroke is often thought of as a disease of the older population, the other time in life when risk is highest is from 28 weeks gestation to 4 weeks old. Stroke occurs when blood flow is interrupted to part of the brain, leading to the destruction of brain cells. Stroke is one of the top 10 causes of death for children⁶⁴. This trial is important because 50 to 80 percent of surviving children will have permanent neurological deficits³⁵. If you think your child may be eligible for this FDA-regulated clinical trial, please complete this form.

• Can my children participate in a clinical trial if their stem cells are not banked with CBR?

  Clinical trials are evaluating cord blood stem cells for their ability to help conditions and injuries like autism, pediatric stroke, cerebral palsy, and traumatic brain injury. Currently, all FDA-regulated clinical trials require the use of a child’s banked cord blood stem cells. Several of these trials are only available to children who have their own stem cells banked with CBR.

• How are patients for clinical trials chosen?

  The inclusion criteria for each trial are different. To get connected to future clinical trials that are open to CBR families, please visit CBR’s Center for Regenerative Medicine.

• What is Regenerative Medicine?

  Regenerative medicine is a new approach to treatment. It’s all about establishing normal function in the body through exciting areas such as gene therapies, tissue engineering and cell-based therapies, which stem cells play a vital role in. The U.S. Department of Health and Human Services has identified the value of this cutting-edge approach and stated that “regenerative medicine is a rapidly growing field of biomedicine that will revolutionize health care treatment.” In fact, it’s estimated that 1 in 3 people in the U.S. may benefit from a regenerative therapy in their lifetime.

  1. Mason, Brindley, Culme-Seymour, Davie. Cell therapy industry: billion dollar global business with unlimited potential. Regen Med. 2011:6(3)265-272.
  2. S. Department of Health and Human Services. 2020: A New Vision. A Future for Regenerative Medicine. 2006.
  3. Harris DT. Expert Opin. Biol. Ther. 2007 Sep;7(9):1311-22.