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Stem Cell Research and Therapies for the treatment of Cerebral Palsy
Scope Position Paper: November 2009
Background
Scope is a not-for-profit organization, providing disability services throughout Victoria, Australia, to thousands of children and adults with physical and multiple disabilities, many of whom have Cerebral Palsy. Cerebral Palsy is a group of disabilities that affects a person's neurological system. Functionally this means someone with Cerebral Palsy may have difficulties moving, maintaining balance and posture and in some cases it may affect a person’s intellectual functioning. It is a term used for brain damage that occurs before or during birth. Currently there is no medical cure for Cerebral Palsy.
Scope is supportive of and participates in evidence based research and ‘best practice’ therapies that may assist in the achievement of its vision of ‘overcoming the personal, structural and attitudinal barriers that prevent those with a disability from participating in community life’. Scope is particularly interested in research that aligns with its research direction: ‘A better life for people with disabilities’.
Stem Cells
The potential of stem cells to improve ways to treat diseases such as Cerebral Palsy has inspired significant interest in this growing field of medical research. Stem cell science has understandably attracted an unprecedented level of attention both in the scientific community and within the media, due to the very real potential it appears to offer. The potential of these technologies has created hope for many Australians with Cerebral Palsy and their families in their quest to find a cure.
What are Stem Cells?
Stem cells differ from other human cells in 2 ways:
- Stem cells are unspecialized, meaning they have not yet developed into cells that perform a specific function in the body. These unspecialized stem cells are capable of self renewal.
- Stem cells can differentiate, meaning they can divide and produce cells that have the potential to become other more specialized cells or tissues.
The Application of Current Stem Cell Science to the Treatment of Cerebral Palsy
Currently, the range of conditions for which there are scientifically proven treatments using stem cells is small. The Australian Stem Cell Centre, an initiative of the Australian government, and respected authority in the area claim ‘The only established stem cell therapies are those of the blood system involving transplants of blood stem cells’ All other stem cell procedures, including those offered to people with Cerebral Palsy, are currently considered highly experimental. While there is a growing body of scientific research that suggests stem cell treatments may be of benefit to people with Cerebral Palsy, to date, this research is considered inconclusive within the general scientific community.
According to the Australian Stem Cell Centre (ASCC) ‘human adult and embryonic stem cells could be used in the generation of cells and tissues for cell based therapies’ ("Australian Stem Cell Centre," 2009). Cell based therapies are treatments in which stem cells are induced to differentiate into specific cell types that are then used to replace damaged cells within the body. According to the ASCC, ‘due to their ability to replace damaged cells in the body, stem cells could be used to treat a range of conditions or diseases that involve stem cell damage or loss’ ("Australian Stem Cell Centre," 2009), such as Cerebral Palsy. The ASCC also state ‘it is hoped the introduction of undifferentiated or differentiated stem cells into damaged tissue will lead to the regeneration of the cell types within that tissue’ ("Australian Stem Cell Centre," 2009). The ASCC are confident that in the future, neural stem cells will be able to regenerate nerve tissue, providing promise for people with Cerebral Palsy. Additionally, a recent research study, the results of which were published in the British Journal of Hematology, suggests that umbilical cord blood stem cells may be beneficial in the treatment of Cerebral Palsy, not only in animals but in early human trials. (Harris, 2009).
The Path from Research to the Clinic
Most medical advances are based on years of research. The path from research to everyday use is long and often complicated for any medical science. There is a long process that proves first in laboratory studies, and then in clinical research/trials that something is safe and will be effective. A clinical trial is the testing of a new medicine or therapy on people. In Australia, every new medical treatment must undergo detailed clinical testing before it can be registered and made available to the community. By the time a study treatment gets to a stage where it is offered to people in clinical trials, it has been thoroughly researched in the laboratory. Despite many members of the community being understandably eager for science to realize the potential of stem cell research, stem cell therapies for Cerebral Palsy have not yet been through the required rigorous scientific process to prove their safety and efficacy and therefore can only be considered experimental.
Scope’s Concerns
There are an increasing number of Australians with Cerebral Palsy and their families spending large amounts of money seeking treatment at experimental private stem cell clinics outside of Australia. Laws which regulate stem cell research differ markedly from country to country. There is particular variation in the laws governing stem cell use. Many jurisdictions lack the regulatory rigor around the use of stem cell treatments that is present in countries such as Australia. The regulations in Australia are based on sound scientific evidence and are driven by clear ethical standards. This lack of regulation in several countries around the world has given rise to the establishment of clinics which, if in Australia, would currently not be legal. Little is known about the details of the experimental treatments provided in these countries, as the protocols used and the evidence base have not been shared openly. Predominately these clinics rely on self-reporting by patients and their families as evidence of improvement. These reports are usually found on the various clinics’ websites. This form of evidence, although it may be credible, is not subjected to the scientific scrutiny necessary to be accepted by respected scientists across the globe.
Medical researchers warn against engaging in such premature treatments, claiming, without appropriate scientific scrutiny it is impossible to know what the risks may be to the patient (Braude, Minger, & Warwick, 2005). They warn of possible neurological complications, brain tumors, viral infections and even death. It is important to note however, that these warnings are themselves anecdotal in nature and are not well understood. Many experimental treatments are offered without any quantification of such potential risks. Therefore it is impossible for people investigating the treatment to make any kind of informed decision as to benefits and risks.
Additionally, Scope is concerned about the impact unregulated technologies could have on an otherwise very promising area of science. The potential of unwanted outcomes from unregulated stem cell based treatments is currently impacting on the sound reputation of this area of science generally. Any medical science is highly dependent on government and community support. Without this support and associated funding provision, the technologies are unlikely to advance to their potential. This will have an impact on the potential benefit stem cell based technologies will have on people with cerebral palsy in the future.
Scope’s Position
Scope supports research that focuses on overcoming the personal, structural and attitudinal barriers that prevent those with a disability from participating in community life. With this intent, it supports new advancements in the support of people with Cerebral Palsy. This includes a diversity of research in both adult stem cell and embryonic stem cell technologies. However, this research remains immature, and to date, its application in humans is not yet supported by enough sound scientific evidence. Stem cell therapies for people with Cerebral palsy are unproven and highly experimental.
To conclude, Scope urge people with Cerebral Palsy and their families to approach offered stem cell therapies cautiously, as to date, there is little scientific or clinical evidence to establish their efficacy or potential harm. Stem cell science is young and despite its enormous potential, its positive applications to people with cerebral palsy are far from being established scientifically.
References
Australian Stem Cell Centre (2009). Patient Information. Retrieved October 21st, 2009
Braude, P., Minger, S., & Warwick, R. (2005). Stem Cell Therapy: Hope or Hype?: Safety and quality must be assured before this treatment can really benefit patients. British Medical Journal, 330, 1159-1160.
Harris, D. (2009). Non-haematological uses of cord blood stem cells. British Journal of haematology, 147, 177-184.
