India’s regulator for medical professionals has declared that using unproven stem cell therapy to treat autistic children will be treated as “professional misconduct” indicating that it makes doctors liable for punitive action in case of violation.
Until now, the ambiguity around the controversial therapy allowed some doctors and companies to subject hundreds of kids to the experimental treatment, which comes at an exorbitant cost.
In a recent order, the Ethics & Medical Registration Board of the National Medical Commission, citing scientific evidence collected by an expert committee, said that stem cell therapy is not recommended as a treatment for autism spectrum disorder in clinical practice.
“In view of the above recommendation, use of stem cells in ASD, its promotion and advertisement will be considered as professional misconduct,” says the NMC order, dated December 6.
The order says that further research needs to be conducted and encouraged in terms of well-designed double-timed, randomised clinical trials to explore the safety and efficacy of therapy for the condition.
“It is a crucial move that will protect vulnerable parents from exploitation in the hands of unscrupulous centres and doctors falsely selling the therapy as a cure for autism,” said Parul Kumtha, a trustee of the parents’ support group Forum for Autism (FFA).
Though there is no database on such centres, estimates suggest there are more than 100 such centres across India offering stem cell therapy for autistic children. Many paediatric neurologists and neurosurgeons have endorsed stem cell treatment as a cure for the disability.
It is estimated that one round of treatment costs parents anywhere between Rs 3-5 lakh and there are often multiple rounds of therapy for autistic children.
Autism in India
Autism spectrum disorder (ASD) belongs to a group of disorders called neurodevelopmental disorders, which are characterised by delays or disturbance in the acquisition of skills in a variety of developmental domains, including motor, social, language and intellectual development.
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The worldwide prevalence of ASD is 0.76 percent, according to the World Health Organization, and the prevalence in lndia is estimated to be around 1-2 per 1,000 people, which in absolute number means that nearly 18 million people in the country may be affected with the condition.
There is no treatment that can cure ASD and for parents and caregivers, education and counselling is the first step, in order to understand, accept and cope with the child’s problem and also learn how to be part of home-based parent-mediated intervention.
The approaches that have worked in improving the condition of autistic children include behavioural interventions such as applied behavioural analysis as well as naturalistic developmental behavioral interventions such as early start Denver model (play based behavioral therapy), speech or language therapy, occupational therapy, educational approaches, and pharmacological management of comorbid problems such as epilepsy, sleep disorders and behavioral or psychiatric problems.
Stem cell for autism
Stem cells are special human cells that are able to develop into many different cell types and are capable of self-renewal. They play an important role in repair and regeneration of various tissues and organs in the body and have been harnessed for research and for therapy.
This therapy has been investigated as a possible treatment option in autism. It is based on the unproven hypothesis that it helps in offering neural cell protection by enhancing neural tissue repair and preventing ongoing neuronal damage, thus reducing the severity of autistic symptoms.
But Dr Uma Ladiwala, a stem cell researcher based in Mumbai, said that no evidence has been generated anywhere in the world so far through double blinded, randomised control trials showing the therapy results in significant improvement for autistic kids compared with standardised behavioural therapy.
The NMC, in its order, noted that five randomised controlled trials or controlled clinical trials have been conducted so far, with the largest being a well-designed double-blind randomised placebo-controlled study in the US. In this study of 180 children with ASD, stem cell therapy was given to 119 children with ASD, with 61 controls.
At the end of 6 months, however, there was no difference between cases and controls in any of the primary outcome measures. Another controlled trial in the country also did not find any difference between SCT and placebo. The other three trials — one in lran and two in China — did find some benefit, but these studies were found to be inadequate in terms of their methodological rigour and their results are therefore not reliable, said the NMC circular.
“Thus, it can be safely concluded that as of now there is no clear evidence that stem cell therapy is efficacious in ASD,” says the order.
Double whammy for parents
Kumtha recalled that she had heard of instances where desperate parents had resorted to mortgaging their houses or selling jewellery to fund the experimental treatment in pursuit of the “cure” promised by some stem cell therapy centres.
Ladiwala pointed out that while the guidelines on stem cell research and therapy by the Indian Council for Medical Research, which were updated last year, say that the therapy can only be offered for autism and some other conditions, this has to be only within clinical trials, with no charges levied on patients.
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“But not only are vulnerable parents lured into opting for a treatment that does not work, there is no proof of its safety either. They are also made to pay through their nose in clear violation of the ICMR guidelines, which state that parents should in fact be compensated in case of side effects,” she said, adding that even the few research projects on this in India have not been carried out in the prescribed manner.
Kumtha said that often, stem cell centres also offered standard behavioural therapies in addition to stem cell therapy and any improvement in a patient was then projected as a proof of the efficacy of the stem cell therapy alone.
This is misleading and unscientific, she said.
Dr Satendra Singh, a disability activist who teaches at the University College of Medical Sciences in New Delhi, said the latest NMC order will now offer stakeholders a chance to challenge the treatment offered by some doctors in state medical councils and courts, if they violate the NMC order.
“There were a lot of grey areas earlier and various centres and doctors made full use of this by trapping desperate parents,” he said.