Emerging therapy
Stem cell therapy
Umbilical cord blood, cord tissue MSCs, bone marrow mononuclear cells
Several different products share this name. Some are licensed medicines for blood disorders; none is a licensed treatment for cerebral palsy or autism anywhere in the world.
Randomised or controlled trials in children are under way or completed, but the result is not yet settled enough for routine care. Taking part in a registered trial is reasonable; paying for it as an established treatment is not.
Where it stands, condition by condition
The same therapy can be well supported for one problem and completely untested for another. This is the single most common place families are misled.
| Condition | Evidence | What that means here |
|---|---|---|
| Blood and immune disorders | Established care | Cord blood transplantation is a licensed, decades-old treatment — this is where the word “approved” genuinely applies. |
| Cerebral palsy | In clinical trials | Randomised controlled trials have been run and more are under way. Where benefit is reported it is usually small and measured on motor scales, not a return to typical development. |
| Hypoxic-ischaemic encephalopathy (newborn) | In clinical trials | Early-phase trials, usually added to cooling. Feasibility and safety are the main findings so far. |
| Autism | Early research only | A well-conducted randomised trial of cord blood did not meet its main endpoint. A possible signal in a subgroup has not been confirmed in a separate trial. |
| Genetic epilepsies | Early research only | No controlled paediatric evidence. Biological reasoning only. |
What it is
“Stem cell therapy” is not one treatment. The products offered to families differ in what the cells are, where they came from and what they can do — and those differences matter more than the shared label.
The main types you will be offered are umbilical cord blood (the child's own, stored at birth, or a donor's), mesenchymal stromal cells from cord tissue, placenta, fat or bone marrow, and bone marrow mononuclear cells taken on the day of treatment.
Cells may be given into a vein, into the spinal fluid by lumbar puncture, or occasionally directly into muscle. Route, dose, number of sessions and whether cells are expanded in a laboratory all vary between centres, and there is no agreed standard.
How it is meant to work
Most of these cells are not thought to turn into new brain cells in any meaningful number. That is a common misunderstanding, and clinics rarely correct it.
The leading explanation is paracrine signalling: the cells release growth factors and vesicles that briefly dampen inflammation, support surviving neurons and encourage blood vessel growth.
If that is the mechanism, then the effect depends on a short biological window rather than permanent engraftment — which is also why repeat courses are often sold, and why a single dramatic change is biologically unlikely.
What has actually been tested
- Cerebral palsy has the most paediatric data of any indication here. Randomised controlled trials using cord blood or cord-derived cells have been published, and pooled analyses generally report small average gains on gross motor scales, more often in younger children and at higher cell doses.
- “Small average gain” is an honest description. It is not the same as walking independently, and it overlaps with what intensive rehabilitation alone can achieve in the same period.
- In autism, the largest randomised trial of umbilical cord blood did not meet its primary endpoint. The authors described a possible signal in children without intellectual disability; that finding needs its own trial before it can be used to justify treatment.
- In newborn hypoxic-ischaemic encephalopathy, published work is mostly early-phase: small numbers, added on top of therapeutic cooling, reporting that the procedure was feasible and tolerated rather than that it changed outcome.
What we still do not know
- Which cell type, dose and route works best — no head-to-head trial has settled this.
- Whether gains seen at six months persist for years.
- Whether children with severe structural brain injury benefit at all, since most trials enrol milder cases.
- Whether repeated courses add anything over a single course.
Risks and costs
- The procedure itself: sedation or anaesthesia, lumbar puncture headache, infection, bleeding.
- Infusion reactions, fever and, rarely, allergic reactions — more likely with donor cells.
- Poorly characterised products: unlicensed preparations have caused serious infections when manufacturing was not controlled.
- The non-medical risk is real too. Courses are expensive, usually not reimbursed, and families sometimes delay or drop rehabilitation to afford them.
Questions to ask before you agree
A centre that is doing good work will welcome these questions and answer them in writing.
- Exactly which cells am I being offered — the source, the dose, and whether they were expanded in a laboratory?
- Is this a registered clinical trial? If yes, what is its registry number, and am I paying?
- Which published studies support this for my child's condition and severity, not for a different diagnosis?
- What outcome will be measured, with which scale, and at what time point?
- What happens if there is no change — is a second course then recommended, and on what basis?
More in this section
Exosomes
The cell-free next step after stem cells, with genuinely interesting laboratory science — and, in children, almost no controlled clinical evidence at all.
Early research onlyMuse cells
A distinct cell type with an unusual property — it appears to home to damaged tissue on its own — and an unusually small amount of clinical evidence, almost none of it in children.
Early research onlyPhotobiomodulation
Non-invasive, painless and inexpensive compared with cell therapies — with small, mostly short studies behind it and a wide gap between what is claimed and what has been shown.
In clinical trialsMagnetic stimulation
The most clinically established technique on this site — genuinely approved for some uses in older adolescents, and still investigational for most of what it is offered for in children.
Early research onlyPeptide preparations
Two quite different things share this word: prescription neuropeptide preparations used routinely in some countries, and an unregulated wellness trade. Neither has good evidence in children.
Early research onlyMedicinal mushrooms and nootropics
A research band worth watching, and a supplement shelf to approach carefully. Some of these compounds have real laboratory interest; almost none has been tested in children with neurological conditions.
Established careCannabidiol and cannabis-based products
The one product in this group that became a real medicine — and only for three named epilepsy syndromes. The oil sold in a shop and the licensed solution are not the same thing.