What is HIE?
Neonatal hypoxic-ischaemic encephalopathy (HIE) occurs when a newborn's brain is deprived of oxygen and blood flow before, during or shortly after birth — for example after placental abruption, cord prolapse or a prolonged, difficult delivery. The injury unfolds over hours to days: an initial energy failure, a latent phase, then a secondary wave of inflammation, excitotoxicity and cell death.
HIE affects an estimated 1–3 per 1,000 live births in high-income countries and more than a million newborns a year worldwide. It accounts for roughly 22% of neonatal deaths and is the leading cause of cerebral palsy, with lifelong motor, cognitive and seizure disorders in survivors.4
The current standard of care
Therapeutic hypothermia — cooling the baby to 33–34 °C for 72 hours — is the only proven treatment. It must begin within six hours of birth, which excludes many babies who present late or are born far from a cooling centre.
Cooling helps, but modestly: about one in seven babies treated avoids death or disability who otherwise would not have.2 Around 48% of infants with moderate or severe HIE still die or are left with significant disability despite cooling. There is no approved pharmacological add-on and no alternative on the market.
Why the field has struggled
Dozens of neuroprotective drugs have failed to translate from animals to babies. Most target a single mechanism — one receptor, one inflammatory pathway — while HIE injures the brain through several at once: vascular damage, blood–brain barrier breakdown, inflammation, oxidative stress and loss of neurons and their supporting cells.
The newborn brain also has real regenerative potential, which drugs designed only to block damage do not harness. A therapy that could repair the vasculature and support recovery would be a different class of intervention.
New approaches in development
Cell therapies are the most active area. Several academic groups have run early trials of mesenchymal stromal cells (MSCs). Among them, the Utrecht PASSIoN study showed that intranasal MSCs were safe and feasible in newborns with perinatal stroke — an important step for cell therapy in neonatal brain injury.3 Peptide and small-molecule candidates are also in development.
Peak 15 Bio's Can‑Vas 001 is an allogeneic combination of placental endothelial colony-forming cells (ECFCs) and MSCs, delivered intranasally as an add-on to cooling within 24 hours of birth. In a large-animal model of HIE with cooling, the combination produced 100% survival versus 57% for cooling alone, restored blood–brain barrier integrity and increased mature neuron counts by around 30%.1 Read about what ECFCs are and why the intranasal route.
Why HIE comes first
HIE is where the need is most urgent: a newborn, a window of hours, and no medicine to offer. It is also where a regenerative therapy can be tested well. Orphan-drug and rare-paediatric-disease designations provide clear regulatory pathways and Priority Review Voucher eligibility, and validated MRI biomarkers allow early read-outs. Because the neurovascular injury in HIE is shared with cerebral palsy, stroke and neurodegenerative disease, what is learned in treating newborns can later be carried into those conditions.
Frequently asked questions
How common is HIE?
Roughly 1–3 per 1,000 live births in the US and Europe — about 30,000 babies a year — and more than a million a year globally.
Is there a cure for HIE?
No. Therapeutic hypothermia reduces the risk of death and disability but does not cure the injury. No medicine is approved to treat HIE anywhere in the world.
What is the link between HIE and cerebral palsy?
HIE is the leading single cause of cerebral palsy. Around 20% of babies with moderate-to-severe HIE develop CP.
- Chand KK et al. npj Regenerative Medicine 2021.
- Jacobs SE et al. Cooling for newborns with hypoxic ischaemic encephalopathy. Cochrane Database of Systematic Reviews 2013.
- Baak LM et al. Feasibility and safety of intranasally administered mesenchymal stromal cells after perinatal arterial ischaemic stroke (PASSIoN). Lancet Neurology 2022.
- Global Burden of Disease Collaborative Network. GBD neurological disorders results.
Can‑Vas products are investigational and have not been approved by any regulatory authority. This page is for general information and is not medical advice.