Strengthening Ethical and Professional Practice in Cell and Gene Therapy - Key Highlights from Malaysia’s Updated Guidelines

By Dr. Samuel Ting Chuo Yew

President, Sarawak Society for Cell and Gene Therapy Research (SSCTR)

The Ministry of Health Malaysia and the National Pharmaceutical Regulatory Agency (NPRA) have recently issued two landmark documents that define the national framework governing the use, research, and regulation of advanced cellular and genetic therapies in Malaysia. These are the “Guidance Document and Guidelines for Registration of Cell and Gene Therapy Products (CGTPs)” published by NPRA in 2024, and the “Guidelines on Stem Cell and Cell-Based Therapies (3rd Edition)” released by the Ministry of Health in 2023. Together, these guidelines aim to safeguard public health, ensure scientific integrity, and establish professional accountability in the rapidly evolving field of regenerative medicine.

For healthcare professionals, these documents provide comprehensive regulatory direction on the classification, quality assurance, and governance of all cell and gene therapy products (CGTPs). The NPRA now classifies CGTPs as biological products, thereby subjecting them to the same stringent requirements applied to vaccines, monoclonal antibodies, and other advanced biologics. Both autologous and allogeneic products must be registered before clinical application, except where specific exemptions apply, such as the Clinical Trial Exemption (CTX) or Compassionate Use (CU) pathways. This ensures that all therapies introduced into clinical practice are supported by adequate preclinical, manufacturing, and safety data.

Equally important are the technical standards required to ensure product integrity. Manufacturers and treatment centres must demonstrate compliance with Good Manufacturing Practice (GMP), Good Tissue Practice (GTP), and Good Clinical Practice (GCP). Each product must also have a traceability system to allow post-treatment monitoring, while gene therapy recipients must be enrolled in long-term follow-up programmes to monitor delayed adverse effects such as insertional mutagenesis or oncogenic transformation. These safeguards are critical to maintaining the safety and reproducibility of advanced therapies within Malaysia’s regulatory framework.

The guidelines also emphasise institutional accountability. All facilities offering CGT services must be licensed and inspected by the Ministry of Health, and supported by a recognised Institutional Biosafety Committee (IBC) and an Ethics Review Board (MERC/MREC). These bodies ensure that all procedures are performed under approved protocols, that patient welfare is prioritised, and that any investigational use of cell-based products adheres to ethical principles consistent with the Declaration of Helsinki.

From a professional standpoint, the documents highlight the importance of competency and multidisciplinary collaboration. Personnel involved in cell and gene therapy, ranging from clinicians and pharmacists to biotechnologists and engineers, must undergo appropriate training in aseptic handling, cell culture, biosafety, and clinical risk communication. Pharmacists, in particular, have a crucial role in product storage, transport validation, preparation, and post-marketing pharmacovigilance. Collectively, these measures ensure that cell and gene therapy services in Malaysia are delivered by qualified professionals within a regulated environment.

For the general public, the guidelines serve as a reminder to exercise caution when considering any form of “stem cell” or “gene rejuvenation” therapy. Only treatments conducted in MOH-approved facilities or under NPRA-registered products or clinical trials are considered legitimate. Members of the public are encouraged to verify the credentials of any service provider and to be wary of unverified treatments promoted through social media or overseas centres. Every patient should be provided with comprehensive information regarding the source of cells (autologous or allogeneic), expected benefits and risks, and the need for continuous medical monitoring. Written informed consent remains an essential ethical and legal requirement before any cell- or gene-based procedure is performed.

These national guidelines collectively strengthen Malaysia’s governance of regenerative medicine. They not only protect patients from unproven interventions but also promote scientific excellence and ethical transparency in clinical innovation. As Malaysia advances toward establishing a sustainable ecosystem for cell and gene therapy, these frameworks underscore the shared responsibility of both professionals and the public to ensure safety, accountability, and evidence-based practice.

The Sarawak Society for Cell and Gene Therapy Research (SSCTR) fully supports the implementation of these updated national guidelines. We encourage healthcare professionals to familiarise themselves with the documents and to align their clinical or research activities with the stipulated standards. The public, likewise, is urged to seek care only from accredited facilities and to remain informed about the legitimacy of cell- and gene-based treatments offered in the market.

By strengthening awareness, ethical conduct, and cross-disciplinary collaboration, Sarawak and Malaysia are well-positioned to lead in the responsible advancement of regenerative medicine, from bench to bedside, and from discovery to patient benefit.

References:

1. National Pharmaceutical Regulatory Agency (2024). Guidance Document and Guidelines for Registration of Cell and Gene Therapy Products (CGTPs) in Malaysia.

2. Ministry of Health Malaysia (2023). Guidelines on Stem Cell and Cell-Based Therapies (3rd Edition).

Disclaimer:

This article is intended for public awareness and educational purposes only. It does not constitute medical advice, diagnosis, or endorsement of any specific treatment, product, or provider. Always consult a licensed medical professional before making decisions related to cell- or gene-based therapies.

The accompanying image is an AI-generated artistic representation and does not depict any actual person, product, facility, or medical procedure. It is used solely for illustrative and educational purposes.

The Sarawak Society for Cell and Gene Therapy Research (SSCTR) shall not be held liable for any interpretation, misuse, or action taken based on the contents of this publication. All information provided is accurate to the best of our knowledge at the time of publication and is not intended to substitute guidance from the Ministry of Health Malaysia, the Malaysian Medical Council, or any other regulatory authority.