This is an exciting time in humankind’s quest to seek effective treatments for diseases. Thanks to spectacular advances in science, especially molecular and cellular biology, difficult-to-treat diseases are seeing the emergence of highly effective treatments. For example, we now see cancers melting away with immune or targeted therapy. Gene-based therapies, immunomodulators and transplanting human cells or tissues are breaking barriers as never before.But outside the developed world, there are huge barriers before these therapies reach ordinary people. They have astronomical costs and are subject to patent laws and monopolisation. This is a bigger battle that will have to be fought even in India. But there is another challenge intrinsic to our healthcare ecosystem. Lack of scientific protocols, poor regulation and a large private sector seeking to monetise all care result in many new therapies being pushed without proven scientific efficacy. The best way to understand this is by observing the enormous growth of India’s stem cell therapy industry, which attracts desperate patients from all over the world with fantastic promises of a cure.AdvertisementTherefore, when on January 30, the Supreme Court delivered a judgment banning the use of stem cells as “therapy” for autism, it was a milestone. Though it addresses only autism, it sets a precedent for how any novel treatment should be regulated. It unpacks the worrying state of healthcare regulation and predatory commercialisation. The verdict also provides protection for citizens from fraudulent and harmful misuse of expensive medical advances.Stem cells are unique unspecialised cells, normally present in the embryo, after birth and in adulthood. They self-renew by dividing repeatedly or transform into specialised cells performing different functions. Scientists are trying to harness this property, particularly when the body is unable to self-repair, and no cure exists. They have been tried in neurological problems like Parkinson’s disease, Alzheimer’s and spinal cord injuries. Their use in most diseases is currently confined to research. Bone marrow transplant, used successfully in treating blood disorders, especially cancers, is a form of stem cell therapy and the only one approved by the US FDA and ICMR. It is commonly used in India.Autism is a neuro-developmental condition, characterised by social communication deficits and repetitive behaviours. It can usually be managed with behavioural and supportive therapies. Those mildly affected are socially well-integrated, but those with severe disease face difficulties. Sadly, they are vulnerable to a plethora of unsubstantiated “cures”, including stem cell “therapy”.AdvertisementIn order to determine whether any therapy is effective, well-designed clinical trials are mandatory using a globally accepted method. There have been clinical trials of stem cell therapy in autism, but the results are mostly inconclusive due to poor methodology, inconsistent doses and short follow-ups. High-quality randomised, controlled trials concluded that it was of no benefit for autism. Besides, the procedure is invasive. Despite claims that it’s “safe”, data shows worrying side effects such as epilepsy, headache and infection at the site of injection.India is a hot spot for stem cell centres claiming cures not only for autism but also for many diseases, and “medical tourism” for this therapy is a thriving industry. This is strange because stem cells is one area where India has regulations. However, taking advantage of the regulators’ inaction, practitioners brazenly advertise this therapy for a host of other conditions for which no cure exists. The charges are exorbitant, running into lakhs per injection.In recent years, NGOs working with autistic children were approached by caregivers who had tried stem cell therapy, but without benefit, despite spending large amounts of money. Some, in fact, developed unpleasant side effects. That is when a unique alliance of caregivers, NGOs and doctors filed a petition in the Supreme Court in May 2022 challenging its legal permissibility. They also pointed out that even though a regulatory framework exists, it was not enforced.Subsequently, the Ethics and Medical Registration Board of the National Medical Commission deemed stem cell therapy as not recommended. Further, the use of the therapy, its promotion or advertisement was labelled as professional misconduct. The SC verdict concluded from a perusal of the ICMR and NMC guidelines that the use of stem cells for the treatment of autism is not recommended as routine treatment due to the lack of scientific support for its efficacy. And, that any use must be as an approved and monitored clinical trial. Also, the use of stem cells outside an approved clinical trial is unethical and constitutes medical malpractice. Medical practitioners using it would attract consequences of professional misconduct, including cancellation of registration and a penalty.The judgment also says that patients have no right to demand unproven therapies outside clinical trials. This may be a response to the argument that unproven therapies are offered because of patient demand, and they provide “consent” for it. This is a noteworthy part of the judgment which may have far-reaching consequences beyond stem cells and autism.Will the judgment change anything? Will the exploitation of helpless citizens seeking deliverance from disease, suffering and death stop? We have a long way to go, especially in a country where healthcare’s scientific moorings are tenuous and dominated by commerce. Where a wide spectrum of therapies, traditional and modern, holds sway, implementation will not be easy.you may likeUnderstandably, the ability of ordinary people to distinguish scientific therapies from those being peddled for commerce is limited, especially when faced with the prospect of no cure. They need humane, neutral guidance. There are two gatekeepers — regulatory bodies and the medical profession, through its commitment to provide rational care. When they are weak, unwilling or compromised, the task becomes onerous.India’s challenge in facing difficult diseases needs both — access to newer and proven expensive therapies and protection from dubious ones. This case offers hope and a model. If an alliance can join forces to protect vulnerable patients, they could also lead the upcoming tougher battle for access.Nagral is a surgeon and one of the co petitioners in the case on behalf of the Forum for Medical Ethics. Ladiwala is a Neuroscientist and stem cell researcher