Hope, Hype and the Law: Regulating Stem Cell Therapy for Children with Intellectual Disabilities

This article critically examines the Indian regulatory framework governing stem cell therapy in the context of intellectual disability, with particular attention to statutory regulation, ethical oversight, and judicial standards on informed consent, negligence, and professional care writes Prof. Dr. P. Sree Sudha.

Introduction

Stem cell-based interventions occupy a particularly sensitive position within contemporary bioethics, lying at the intersection of scientific uncertainty, therapeutic hope, and vulnerability of patients. Nowhere is this tension more evident than in the promotion of stem cell therapy for children with intellectual disabilities. While regenerative medicine continues to evolve, claims of therapeutic benefit in this context remain scientifically unsubstantiated, raising profound ethical and legal concerns regarding consent, exploitation, and professional responsibility.

In India, the emergence of private clinics advertising stem cell interventions for neurodevelopmental conditions has triggered regulatory intervention and public debate. Parents of children with intellectual disabilities often navigate a landscape marked by informational asymmetry and emotional distress, making them particularly susceptible to therapeutic misconception. Against this backdrop, law functions not merely as a regulatory instrument but as an ethical safeguard.

This article examines the Indian regulatory framework governing stem cell therapy for intellectual disability through the lens of medical ethics and health law. It analyses statutory regulation, ethical oversight mechanisms, and judicial standards on informed consent, negligence, and standards of care, and situates the Indian approach within a comparative perspective drawn from the United Kingdom and the United States. The article argues that India’s precautionary regulatory posture reflects core bioethical principles respect for autonomy, non-maleficence, and justice and is essential to prevent the exploitation of vulnerable children while permitting ethically defensible scientific research.

Understanding Intellectual Disability and Stem Cell Therapy

Intellectual disability is recognised internationally as a developmental condition rather than a disease amenable to curative treatment. Medical management typically focuses on early intervention, education, behavioural therapy, and social support. Stem cell therapy, by contrast, involves the use of undifferentiated cells capable of self-renewal and differentiation into specialised cell types. While stem cell-based interventions have demonstrated success in certain haematological conditions, their application to neurodevelopmental disorders remains speculative.

The absence of robust clinical evidence supporting stem cell therapy for intellectual disability has significant regulatory implications. In legal terms, such interventions fall within the category of experimental or investigational treatments, thereby triggering heightened ethical and regulatory scrutiny, particularly when children are involved.

Indian Regulatory Framework

I. Institutional Architecture

The regulation of stem cell research and therapy in India operates through a multi-institutional framework. The Indian Council of Medical Research (ICMR), in collaboration with the Department of Biotechnology (DBT), is responsible for framing ethical and scientific guidelines. The Central Drugs Standard Control Organisation (CDSCO), functioning under the Ministry of Health and Family Welfare, regulates clinical trials and approval of new drugs, including stem cell–based products.

This dual structure reflects an integrated approach, combining scientific oversight with statutory drug regulation.

II. National Guidelines for Stem Cell Research, 2017

The National Guidelines for Stem Cell Research (NGSCR), 2017, issued jointly by ICMR and DBT, constitute the central regulatory instrument governing stem cell research and therapy in India. These guidelines replaced earlier versions issued in 2007 and 2013 and represent a significant tightening of regulatory control.

The NGSCR draw a clear distinction between stem cell research and stem cell therapy. While basic and translational research may be permitted under institutional oversight, the clinical application of stem cells is tightly restricted. The guidelines explicitly state that, with the exception of haematopoietic stem cell transplantation for approved indications, all stem cell therapies are considered investigational.

Stem cell interventions involving children and other vulnerable populations are categorised as ‘restricted’ and are subject to enhanced ethical scrutiny. Importantly, the guidelines prohibit the commercialisation and advertisement of unproven stem cell therapies. Stem cell therapy for intellectual disability is not recognised as an approved indication, and its clinical use outside an approved trial framework is therefore impermissible.

III. Drugs and Clinical Trials Regulation

Under the Drugs and Cosmetics Act, 1940, read with the New Drugs and Clinical Trials Rules, 2019 (NDCTR), stem cell–derived products intended for therapeutic use are classified as ‘new drugs’. This classification brings them within the statutory framework governing drug approval and clinical trials.

The NDCTR require prior approval from the CDSCO, phased clinical trials, and the generation of data demonstrating safety, efficacy, and quality. Clinics offering stem cell therapy for intellectual disability without such approval operate in violation of statutory drug regulation, regardless of claims of innovation or compassionate use.

Ethical Oversight and Consent

I. Institutional Ethics Committees

Institutions conducting stem cell research are required to establish an Institutional Ethics Committee (IEC) and an Institutional Committee for Stem Cell Research (IC-SCR). These bodies are responsible for reviewing research protocols, assessing risk–benefit ratios, and ensuring compliance with ethical norms.

For research involving children with intellectual disabilities, ethics committees must ensure that the proposed intervention serves the best interests of the child, minimises risk, and avoids therapeutic misconception.

II. Informed Consent, Negligence and Standards of Care: Indian Case Law

Indian courts have consistently developed jurisprudence emphasising patient autonomy, informed consent, and adherence to accepted medical standards. These principles are directly relevant to stem cell interventions for intellectual disability, which are categorised as experimental.

In Samira Kohli v. Dr. Prabha Manchanda, (2008) 2 SCC 1, the Supreme Court held that consent for medical treatment must be real and informed, requiring disclosure of the nature of the procedure, its risks, available alternatives, and the consequences of refusal. The Court clarified that consent for one procedure cannot be presumed as consent for another, particularly where the latter involves higher risk or experimental elements. Applied to stem cell therapy, this judgment implies that parental consent is invalid if the experimental status, lack of proven efficacy, and potential risks are not clearly disclosed.

The standard of professional care was articulated in Poonam Verma v. Ashwin Patel, (1996) 4 SCC 332, where the Supreme Court held that a medical practitioner who does not possess or exercise the requisite qualifications and skill acts negligently. Offering stem cell therapy for intellectual disability without regulatory approval or scientific validation may amount to negligence per se, as it departs from recognised medical standards.

In Jacob Mathew v. State of Punjab, (2005) 6 SCC 1, the Supreme Court adopted the Bolam test for determining medical negligence, holding that a doctor is negligent if they act in a manner not accepted as proper by a responsible body of medical professionals. Since stem cell therapy for intellectual disability lacks acceptance within the responsible medical community and is not approved by regulatory authorities, its clinical use may fail the Bolam standard.

Further, in Kusum Sharma v. Batra Hospital, (2010) 3 SCC 480, the Court emphasised that medical decisions must be based on current medical knowledge and established practice. Experimental interventions cannot be justified solely on the basis of innovation or patient demand. This principle reinforces the illegality of offering unproven stem cell therapies outside approved clinical trials.

Supreme Court of India on Stem Cell Therapy for Autism Spectrum Disorder

A pivotal judicial development in India’s regulation of experimental biomedical interventions is the Supreme Court’s decision in Yash Charitable Trust v Union of India (2026), which directly addressed the permissibility of stem cell therapy for Autism Spectrum Disorder (ASD). The judgment is of particular relevance to the present inquiry, as autism and intellectual disability frequently intersect in both clinical practice and regulatory discourse, and the affected population consists predominantly of children—a group warranting heightened ethical and legal protection.

The Supreme Court unequivocally held that, in light of the prevailing state of scientific knowledge, the therapeutic use of stem cells for ASD does not constitute recognised or established medical practice. The Court noted the absence of reliable clinical evidence demonstrating safety or efficacy and clarified that such interventions cannot be offered as routine treatment. Stem cell–based interventions for ASD, the Court held, may be undertaken only within the confines of duly approved and closely monitored clinical trials, subject to regulatory oversight.

This determination has direct implications for the legal standard of care in medical negligence. Relying on established precedent, including Jacob Mathew v State of Punjab and Kusum Sharma v Batra Hospital, the Court reaffirmed that medical practitioners are obligated to adhere to practices supported by a responsible body of scientific opinion. Offering unproven stem cell interventions as therapeutic services was treated as a departure from accepted medical standards, thereby exposing practitioners to civil liability and professional disciplinary consequences.

The judgment is equally significant for its treatment of informed consent. The Court observed that consent obtained for an intervention lacking scientific validation cannot be regarded as genuinely informed, particularly where parents are presented with claims of therapeutic benefit unsupported by evidence. Such consent, the Court implied, is vitiated by therapeutic misconception and informational asymmetry. This reasoning reinforces the Court’s earlier articulation of consent in Samira Kohli v Dr Prabha Manchanda, where meaningful consent was held to require disclosure of material risks, alternatives, and the experimental nature of proposed interventions.

Importantly, the Supreme Court embedded its analysis within India’s regulatory framework, holding that the National Guidelines for Stem Cell Research and the New Drugs and Clinical Trials Rules, 2019, are integral to determining the legality and reasonableness of medical conduct. Non-compliance with these instruments was treated not as a procedural lapse but as substantive evidence of breach of professional duty.

From an ethical perspective, the Court expressly acknowledged the heightened vulnerability of children with neurodevelopmental conditions and cautioned against their exploitation through unproven therapies marketed as last-resort cures. By foregrounding principles of non-maleficence and protection of vulnerable populations, the judgment bridges medical negligence doctrine with core bioethical obligations.

For the purposes of this paper, the Supreme Court’s ruling on stem cell therapy for ASD provides authoritative judicial affirmation of India’s precautionary approach to experimental therapies. It strengthens the argument that stem cell interventions for intellectual disability, when offered outside rigorously regulated research settings, undermine valid consent, violate standards of care, and conflict with foundational principles of medical ethics.

Collectively, these decisions establish that deviation from regulatory guidelines, absence of informed consent, and failure to meet accepted standards of care can attract civil and criminal liability. In the context of children with intellectual disabilities, courts are likely to apply these standards with heightened scrutiny due to the vulnerability of the patient population.

Disability Rights and Child Protection

The Rights of Persons with Disabilities Act, 2016 (RPwD Act) provides a statutory framework for protecting persons with disabilities from exploitation and abuse. The Act emphasises dignity, autonomy, and equality in access to healthcare and social services. Subjecting children with intellectual disabilities to high-risk experimental interventions without proven benefit undermines the protective intent of this legislation.

India’s obligations under the United Nations Convention on the Rights of Persons with Disabilities (UNCRPD) further reinforce the duty to safeguard persons with disabilities from unethical medical experimentation and ensure respect for their inherent dignity.

Advertising, Consumer Protection and Medical Ethics

Clinics advertising stem cell therapy as a cure for intellectual disability may violate multiple legal regimes. The Drugs and Magic Remedies (Objectionable Advertisements) Act prohibits misleading claims regarding cures for specified conditions. The Consumer Protection Act, 2019 provides remedies against unfair trade practices and medical negligence.

Professional ethical codes enforced by the National Medical Commission prohibit medical practitioners from endorsing unproven therapies. Regulatory action against such practices reflects the convergence of medical ethics, consumer protection, and public health regulation.

Comparative Perspectives

I. United Kingdom

In the United Kingdom, stem cell research and therapy are regulated through a comprehensive framework involving the Human Fertilisation and Embryology Authority (HFEA) and the Medicines and Healthcare products Regulatory Agency (MHRA). Any stem cell–based medicinal product must receive MHRA approval before clinical use. Experimental therapies for neurodevelopmental disorders are permitted only within approved clinical trials, and commercial use without approval is prohibited.

II. United States

In the United States, the Food and Drug Administration (FDA) regulates stem cell products under the Public Health Service Act and the Federal Food, Drug, and Cosmetic Act. Stem cell therapies intended to treat diseases are classified as biological drugs and require Investigational New Drug (IND) approval and clinical trials. The FDA has taken enforcement action against clinics offering unapproved stem cell treatments, particularly those targeting vulnerable patients.

Comparative Analysis

A comparative analysis demonstrates that India’s regulatory approach is broadly consistent with international best practices. India, the UK, and the US all treat stem cell therapy for unproven indications as experimental and prohibit its commercial use without regulatory approval. The emphasis on informed consent, ethical oversight, and protection of vulnerable populations is a shared regulatory priority. India’s integration of disability rights law into medical regulation adds an explicit rights-based dimension to this framework.

CONCLUSION

Stem cell therapy for intellectual disability lies at the intersection of biomedical innovation, regulatory governance, and the protection of vulnerable populations. Indian law adopts a deliberately cautious approach, recognising both the scientific uncertainty surrounding such interventions and the heightened ethical concerns that arise when children with intellectual disabilities are involved. Through the National Guidelines for Stem Cell Research, statutory drug regulation, judicial doctrines on informed consent and medical negligence, and disability rights legislation, India has constructed a layered regulatory framework that prioritises patient safety, autonomy, and dignity.

Judicial standards articulated by the Supreme Court—particularly in relation to informed consent, accepted standards of care, and professional accountability—reinforce the impermissibility of unregulated or commercial stem cell interventions for intellectual disability. Comparative analysis demonstrates that this restrictive stance is consistent with regulatory approaches in the United Kingdom and the United States, both of which similarly prohibit clinical use of unproven stem cell therapies outside approved trial frameworks.

As regenerative medicine continues to evolve, regulatory systems will inevitably confront pressure to accommodate innovation. However, any relaxation of existing safeguards must remain firmly anchored in scientific evidence, ethical justification, and respect for the rights of persons with disabilities. Until such evidence emerges, the Indian framework appropriately reflects a commitment to preventing medical exploitation while allowing space for ethically sound and legally compliant research.

References

1. Yash Charitable Trust and Others v Union of India and Others, 2026 INSC 96 (Supreme Court of India).

2. Supreme Court of India, Yash Charitable Trust v Union of India (n 1), paras 42-45 (holding that therapeutic use of stem cells for Autism Spectrum Disorder is not recognised as established or sound medical practice based on current scientific evidence).

3. Indian Council of Medical Research and Department of Biotechnology, National Guidelines for Stem Cell Research (2017), paras 1.2, 1.4 and 7.1 (classifying most stem cell therapies as investigational and restricting clinical use outside approved trials).

4. Jacob Mathew v State of Punjab, (2005) 6 SCC 1, paras 19–21 (adopting the Bolam standard for determining medical negligence).

5. Kusum Sharma and Others v Batra Hospital and Medical Research Centre, (2010) 3 SCC 480, paras 89–90 (emphasising adherence to current medical knowledge and established practice).

6. Supreme Court of India, Yash Charitable Trust v Union of India (n 1), paras 48–50 (holding that offering unproven stem cell interventions as therapeutic services constitutes deviation from accepted standards of care).

7. Samira Kohli v Dr Prabha Manchanda, (2008) 2 SCC 1, paras 32–35 (laying down the requirements of real and informed consent).

8. Supreme Court of India, Yash Charitable Trust v Union of India (n 1), paras 52–54 (observing that consent based on exaggerated or misleading claims of benefit is ethically and legally deficient).

9. Ministry of Health and Family Welfare, New Drugs and Clinical Trials Rules (2019), rr 2(w), 21–25 (classifying stem cell–derived products as new drugs requiring regulatory approval).

10. Supreme Court of India, Yash Charitable Trust v Union of India (n 1), paras 56–58 (treating non-compliance with regulatory frameworks as evidence of breach of professional duty).

11. United Nations Committee on the Rights of Persons with Disabilities, General Comment No 3 (2016) on women and girls with disabilities, paras 24–26 (recognising heightened vulnerability of children with disabilities to exploitation in healthcare).