If you are planning to use stem cell treatment for your child, carefully consider each point in the checklist.
Cerebral palsy and stem cell treatments
Cerebral palsy and stem cell treatments
Stem cell treatment for children with cerebral palsy has potential hazards - we do not recommend it unless the treatment is part of a clinical trial approved by an ethics committee and by clinical trial regulators.
Key points to remember
- increased knowledge about stem cells has led scientists to explore whether stem cells can be used to treat a range of conditions including cerebral palsy
- stem cell treatments may have potential for improving function in cerebral palsy
- there are different types of stem cells and different types of stem cell treatments
- we do not yet understand how to use stem cells in cerebral palsy
- currently stem cell treatment for children with cerebral palsy is unproven, although there are case reports of short and longer term improvement from centres in Germany, the Middle East, China and India
- stem cell treatment for children with cerebral palsy has potential hazards - we do not recommend it unless the treatment is part of a clinical trial approved by an ethics committee and by clinical trial regulators
- if parents are planning to use stem cell treatment for their child, they should carefully consider the points in the section below ‘What should parents do?’
Why might you consider stem cell treatment?
Parents who have a child with a disability, especially a severe disability that significantly limits function and/or quality of life, may want to consider any therapy that offers hope of cure or significant improvement.
The discovery of stem cells in adult brains has led scientists to explore whether stem cells can be used to treat a range of conditions affecting the brain, including cerebral palsy.
Stem cell therapy is well established in the treatment of cancer patients with conditions such as leukaemia and lymphoma.
What is a stem cell?
A stem cell is a primitive cell that has the potential to develop into a range of cell types and form different tissues. Stem cells have the ability to renew themselves by dividing.
There are 2 main types of stem cells:
- Embryonic stem cells that are found in the very early embryo. These cells have the potential to develop into all the different tissues and organs that the developing embryo will need as it matures.
- Adult stem cells that are found in the tissues of humans who have matured beyond the embryonic stage (that is, in the tissues of fetuses, infants, children and adults). The function of adult stem cells is to supply new cells for repairing tissues; for example, bone marrow stem cells develop into blood cells.
Cerebral palsy is a permanent physical condition that affects movement and posture and can cause a range of other problems. It is caused by damage to, or lack of development in a part of the brain that controls movement. Cerebral palsy is the most common physical disability in childhood occurring in 1 in every 500 babies. Cerebral palsy is often called ‘CP’ for short. For more information, see the cerebral palsy page on this website.
Could stem cell therapy help children with cerebral palsy?
Scientists are developing stem cell therapies for the replacement or repair of damaged tissues such as nerves, muscle and other parts of the body.
Stem cell therapy is a rapidly developing area of research but is a long way from being a practical reality for cerebral palsy treatment. Since cerebral palsy is complex, it seems unlikely that a single type of cell replacement therapy will be developed for all children with all types of cerebral palsy.
The hope is that in the future, some type of stem cell therapy may help children with cerebral palsy by replacing injured nerve cells that can take over the function of the damaged areas of the brain. As more information becomes available from carefully conducted scientific studies, this therapy may prove useful for children with cerebral palsy.
There are many possible ways to use stem cells to make new tissues for repairing damaged tissue.
You may have heard or read that one source of stem cells for treating cerebral palsy is cord blood from the umbilical cord of a newborn baby. You may have stored your child’s own cord blood in a private cord blood bank. While cord blood is a rich source of adult stem cells and is used in stem cell transplants for leukaemias, there is no evidence to support using any particular type of stem cell in cerebral palsy.
What happens in a stem cell transplant in a child with cerebral palsy?
First, there needs to be a source of stem cells. Sources can be:
- a child’s own cord blood - some parents choose to store their child's cord blood at the time of birth
- donor cord blood from a donor cord blood bank
- bone marrow from the child or from a related or unrelated donor - if the stem cells come from a donor, the cells need to match the child’s tissue type
Then the doctor injects the stem cells into the child. The method used will depend on the centre carrying out the therapy. The injection may be:
- an intravenous infusion into the child's bloodstream, or
- into the space around the spinal cord by a lumbar puncture
There is no certainty that the stem cells will develop into nerve cells (also called neurons). For a stem cell treatment in cerebral palsy to be successful, several things may need to happen:
- the stem cells need to develop into nerve cells
- these nerve cells have to develop in an area where they can form connections with other nerve cells
- the nerve cells have to develop connections in such a way that the cells deliver meaningful messages to the muscles that control movement
- the stem cells may also work by developing into support or helper cells that are important for the nerve cells to function – this is an area of ongoing research
To make the treatment more likely to be effective, most centres offering this treatment also recommend intensive physical therapy programmes following the stem cell treatment. This makes it very difficult to know whether improvements are the result of the stem cell treatment itself, or the intensive physical therapy, or are the result of the child growing and developing.
Can anything go wrong?
Yes, things can go wrong.
- many of these treatments are carried out in countries where standards of health care differ from those in New Zealand
- there is the risk of introducing viral diseases and prion diseases (such as Creutzfeldt–Jakob disease - CJD) from donor cells. It is really important to know where the donor cells came from and whether the person who donated them was a healthy individual – this is not always carefuly regulated in non-approved trials
- bacterial infections can also be introduced, especially if the donor cells have not been properly stored
- because we do not know what factors influence the development of the stem cells into the kind of cells that are needed, there is a possibility that they will develop into cells that are harmful. There are reports of tumours developing after stem cell transplantation
- stem cells that multiply (remember that is the function of a stem cell) without differentiating or developing into mature cells may cause complications, which may be serious depending on where in the body this happens
Does it work?
We do not know the answer to this.
There is a study going on at Duke University in the United States (US). It is ethically approved and supported by the US National Institutes of Health (NIH). But it is not a treatment. It is a study. It plans to follow 70 children who have received intravenous infusions of their own cord blood. There will be no control group, but the children will be followed up over a period of years. It will therefore be some time before any results are available.
The interest in stem cell therapy has led to the development of many commercial clinics claiming to offer stem cell therapies. In many parts of the world these clinics are unregulated, uncontrolled, and the treatment unproven.
In some clinics, the treatment being offered may not be what is claimed and the cells given may not even be true stem cells.
There are many claims on the internet of dramatic improvements following stem cell treatments. But there are no completed controlled studies. Remember people do not like to publicise their failures – so we really do not know how successful this treatment is. There are real concerns about exploiting vulnerable families who only want to do the best for their children.
What should parents do?
Our advice is that stem cell treatment for children with cerebral palsy is unproven and has potential dangers. Therefore we do not recommend it, unless the treatment is part of a clinical trial approved by an ethics committee and by clinical trial regulators.
If parents are planning to use this option for their child, they should carefully consider the following points:
- What information is available about the donor cells?
- Who donated them?
- Was that person healthy?
- Is there a risk that my child could be infected with a serious disease as a result of this treatment?
- How will the treatment be given?
- How much will this treatment cost?
Remember that there is always more than a dollar cost. Think about the disruption to the rest of the family as a result of travelling overseas; the safety of the destination; loss of earnings.
- What qualifications do the people giving the treatment have?
- What follow-up and treatment evaluation will they offer?