Better than a cold blanket
Speeding cell therapies from bench to bedside
Co-authored with Moayed Hamza and Mark O’Neill
Neonatal hypoxic ischemic encephalopathy (HIE) is a type of newborn brain damage that is caused by a lack of oxygen to the brain before or shortly after birth. Where HIE is graded as moderate or severe, it can result in long-term disability and in some cases can result in death. HIE remains a global health problem accounting for a quarter of neonatal deaths worldwide and is the second most common cause of preventable childhood disability.
Therapeutic hypothermia (a cooling blanket) is the standard of care for moderate to severe HIE in high-income settings. Despite cooling, there remains a 30% mortality in treated babies with 22% of survivors suffering major neurodevelopmental disabilities such as cerebral palsy or significant cognitive, language and behavior impairment. There is no treatment available in rural America where the sick neonate is often cared for up to 100 miles from home, and there is no effective treatment in low to middle income countries.
But there is possibly a better solution than just a cold blanket.
HAON Life Sciences is a new biotech developing treatments for serious and debilitating diseases including HIE, from their Can-Vas allogeneic (off-the-shelf) cell therapy platform. Can-Vas isolates therapeutic cells from whole term healthy placentas and creates neuroprotective and regenerative treatments that can be produced at scale. HAON’s first candidate is Can-Vas-001, an intranasal spray being developed to allow early treatment of newborn babies suffering from HIE.
The promise of a global network of MRS imaging machines
One reason new cell therapies take a long time to go from bench to bedside is the lack of timely and adequate data. As an example, a biotech start-up developing breakthrough cell therapies to tackle neonatal HIE might spend years of time and money to discover whether the therapy was effective.
What if you could use imaging in the first weeks of life to predict the long-term outcomes for these vulnerable patients? This would cut years out of the process, reducing cost and improving patient outcomes.
For HIE, magnetic resonance spectroscopy (MRS) imaging acquired within the first 14 days of life provides a solution to predict outcomes in babies with moderate to severe HIE 2 years later. MRS is a type of brain imaging for measuring key metabolites within the living brain such as N-acetyl aspartate (NAA) and lactate (lac). The ratio of these metabolites can be measured using MRS imaging providing early insight into long term survival and developmental outcomes for babies with HIE.
What if we had global network of MRS imaging machines? What if we could provide real-time, privacy-preserving data to cell therapy developers as well as the regulatory bodies? We could use the global privacy-preserving edge cloud service developed for the Pediatric Moonshot to implement this vision.
FDA has created an accelerated approval pathway for just this scenario to allow an expedited option for treatments that provide a meaningful advantage over available therapies for serious conditions. The recent explosion in cancer drug approvals demonstrates this. It takes far less time to see whether a drug shrinks a single tumor than to determine whether the drug improves the survival of a cancer patient.
The approval decision via this pathway is based on surrogate endpoints which are measures of effect for specific treatments or drugs in clinical trials. A surrogate endpoint like MRS imaging in this case may correlate with a real clinical outcome that could take much longer to measure. For serious conditions like HIE, using surrogate endpoints to evaluate the efficacy allows a treatment to reach the market years earlier than waiting to evaluate its effects on irreversible morbidity and mortality.
Bottom line: Cell therapy technology combined with a global network of imaging machines will speed new therapies from the bench to bedside.
To learn more about HAON’s work see www.linkedin.com/company/haon-life-science or reach out to Moayed Hamza or Mark O’Neill
The Pediatric Moonshot mission is to reduce healthcare inequity, lower healthcare costs, and improve outcomes for children — locally, rurally and globally — by creating privacy-preserving, real-time AI applications based on access to data in all 1,000,000 healthcare machines in all 500 children’s hospitals in the world. To learn more, visit https://pediatricmoonshot.com/.
