Two months after his son, Apartim Dey Singha, turned two, Apurba Dey Singha, a chartered accountant based in Kolkata, felt that the boy seemed to be manifesting “developmental challenges”. These included a marked reluctance to maintain eye contact, diminished communication skills and “hyper-aggressiveness”. After visits to child psychologists — a couple of them in New Delhi — he and his wife learnt, in 2016, that young Apartim was “moderately autistic”.
They began a regimen of behavioural therapy and diet control (“gluten-sugar-caffeine free”) and though there appeared to be improvement, Mr. Singha felt it wasn’t enough.
“We were quite anxious and I researched quite a bit about autism until I came upon links to stem cell treatments,” he said in a phone conversation.
Turning to cord blood
Autism is a neurodevelopmental disorder that impairs a person’s social ability to interact. It manifests itself through speech disability and other social behavioural issues. It shows up early and can be diagnosed by the age of three. By virtue of it being related to neuronal development, one strain of hypothesis holds that autism results from impaired connectivity among neurons. Therefore, their proponents say, using cord blood (blood from the umbilical cord and known to be a rich source of stem cells) can potentially help in the birth of new neurons or repair damaged connections and help at least some children. A 2017 estimate suggests that 23 out of every 10,000 children born in India have autism.
Mr. Singha, as part of his research on finding treatments for his son, found that a group of scientists at Duke University, North Carolina, U.S., had begun a five-year clinical trial in 2014 to test this theory.
“Luckily, we had saved Apartim’s cord blood cells through ‘Life Cell’,” he said, in a reference to the Chennai-based company which claims to be one of India’s largest stem cell bankers.
Early results from the Duke University trial, and published last year in the peer-reviewed Stem Cells Translational Medicine , seemed to suggest that infusing children with their stem cells was “safe”. While a few of the 25 children involved reported allergic reactions, the researchers said these were unlikely to be related to the infusion. On the bright side, several of the parents of the children enrolled in the trial reported “significant improvements”, the authors note in their study. However, this could very well have been due to a powerful placebo effect, where optimism about a therapy clouds caregivers’ objective assessment of its effects.
Discounting the placebo effect in the autism trial would only be known in a double-blind controlled study, which means only some of the children enrolled in the trial would actually be given the stem cells and others a fake, harmless injection and nobody, even the doctors themselves, would not know who had got what.
Duke University researchers commenced such a trial last year in which Apartim too was enrolled. “The way I saw it, there was no harm,” said Mr. Singha, who subsequently travelled to the U.S. and had his son participate in the trial last June. While it’s indeterminable if the four-year-old actually got an infusion of stem cells, Mr. Singha said that he has seen “some improvement” in his son. When he was formally diagnosed with autism in 2016, he had scored 30 on a scale of 100-point autism-assessment scale and a recent test showed a “score of 9”, said Singha.
While the design of the trial requires participants to take infusions after six, nine and 12 months, Singha said he hasn’t “yet decided” if he will enrol Apartim in future sessions.
Private cord blood banks are enthused. “It’s heartwarming to see clinical improvement and development milestones in Apartim Dey Singha. There are many ongoing clinical trials and this certainly gives hope to parents who have preserved their baby’s stem cells at birth,” Mayur Abhaya, CEO and MD, LifeCell, said in a statement.
Scepticism abounds
However independent researchers are far from convinced. For one, autism is known to have a significant hereditary component, that is, it is passed on via the genes. So harnessing the body’s own stem cells, which could itself carry aberrant genetic mutations, would be counterproductive, said Sheffali Gulati, Professor of Paediatric Neurology at the All India Institute of Medical Sciences, New Delhi. “It’s a shot in the dark because the cell biology in autism at this time is a mystery. Second, how are stem cells going to cross into the brain?” she queried. The so-called blood-brain barrier (BBB) seals off circulating blood and attendant cells, molecules from the fluids in the brain.
Joanne Kurtzberg, who leads the Duke trial, has argued that stem cells can cross the BBB by transforming into specialised cells called microglia, that send specific anti-inflammatory signals. These chemical reactions work to fix the weakened neuronal connections, which characterise autism. Internationally too, neurologists such as Dr. Gulati are sceptical of this hypothesis but say that the Duke trial is a well-designed investigation involving a large number of children and so is likely to yield important answers.
jacob.koshy@thehindu.co.in
