Stem cells show promise as a treatment for some neurological injuries, and they are a hot topic for the cerebral palsy community. After 15 years of international laboratory studies related to cerebral palsy, we know that stem cells may prevent injured cells from dying, and may help with some level of repair. Whether this could help individuals with cerebral palsy is unknown.
Researchers at Murdoch Children's Research Institute are working with many different experts to explore the idea of stem cells as a possible therapy for cerebral palsy. Right up front, I would like to say that we are not looking at whether stem cells might be useful for treating cerebral palsy (not yet at least). We are looking at what sort of research it is possible for us to do, and whether there is enough evidence to make more research worthwhile. For the latter I feel comfortable saying that yes, more research is worthwhile.
I am less comfortable about what sort of research is needed next. The preclinical studies don’t tell us which type of stem cell might be most effective or the best way to put the cells into someone’s body, and we don’t really know how the cells might be acting. With so much yet to find out, we clearly need more basic research. The problem with studies in the lab or with animals is that they only go so far in imitating cerebral palsy (which is a pretty complicated developmental disorder). Is it time to test this in humans or not?
Research into stem cell therapies for cerebral palsy is at its infancy and there are currently no available treatments. Various new strategies are being explored, but most of these will not offer a complete cure. Rather, the therapies being investigated may allow us to limit the damage to cells in the brain and reduce the symptoms.
Cerebral palsy can damage many cells in the brain, and these cells can be completely lost. This means it is likely that any new treatment will have to be applied quickly, between the initial injury that has caused damage and the onset of permanent cell loss. Scientists expect that future treatments may protect and repair damaged brain cells before they are entirely lost.
Although so much is unknown, there are private clinics around the world already offering stem cell ‘treatments’. Of course, families want every opportunity for their children, and desperate families are travelling overseas and paying over $40,000 for unregulated, unproven and possibly dangerous procedures. These families are choosing to take a much higher risk than Australian scientists, clinicians and ethicists are. Other families wait patiently (and impatiently) to see what the risks are.
It is hard to understand the risks of new treatment ideas because we don’t know how likely each imagined problem is. And we can’t know the unimagined problems at all. Can we justify unknown side effects and risks? Maybe we can, if there is a benefit, but at the moment we have barely more than a hint that treatment with stem cells could help children with cerebral palsy.
Who gets to decide whether a new treatment justifies the risks? In Australia, we rely on Human Research Ethics Committees to decide if we can justify learning more about the treatment in a clinical trial. We rely on Clinical Ethicists to help doctors and hospitals decide about treatments, and the Therapeutic Goods Administration to decide which drugs and devices should be available.
I imagine the balance between risk and benefit as a seesaw. We can reduce the weight on the ‘risk end’ by using a child’s own cells instead of someone else’s, by delivering into the blood instead of into the spinal cord or brain, or by using a type of stem cell that is already commonly transplanted in humans such as cord blood or bone marrow cells. Also, we reduce the risk by studying safety carefully rather than rushing into a treatment or even into a large trial. To add weight to the ‘benefit end’ of the seesaw, doctors and scientists concentrate research in the most positive directions, and make sure it is well designed and rigorous.
Right now, Australian researchers are trying to balance the seesaw to move research forward while not putting children at too much risk. Just like you, we eventually want to find out if stem cells might help children with cerebral palsy.
The Murdoch Children's Research Institute may publish material submitted to the blog and remove any comments it deems inappropriate or offensive at its sole discretion. The Institute accepts no liability in respect of any material published or the content and accuracy of any material published. If you have any concerns with any of the published material or comments on the blog, please contact us at firstname.lastname@example.org.