Expanded Access to Experimental Stem Cell and Gene Therapies in China: A New Regulatory Model

While the recent adoption of a flexible clinical translation model in Hainan Province promises to accelerate biomedical innovation in China, it has also intensified concerns about patient safety, transparency, and misinformation. The success of this new approach is largely dependent on strengthening post-market oversight, access to clinical data, and strategic alignment with international bioethical guidelines.

Boao Lecheng International Medical Tourism Pilot Zone. (Image by Boao Lecheng International Medical Tourism Pilot Zone Administration)

Strategic shift in China’s regulatory regime for regenerative medicine

Over the past year, there have been significant regulatory developments in China's regenerative medicine ecosystem. A significant step was the announcement by Hainan Lecheng government of official pricing for the first batch of three stem cell therapies, which was made during the 2025 Boao Forum for Asia (BFA) Annual Conference. They were among the 12 stem cell and gene therapies approved on February 9, 2025, by the Medical Product Administration of Boao Lecheng International Medical Tourism Pilot Zone (Lecheng Pilot Zone), a step that bypasses the conventional drug approval pathway. Previously, only one stem cell-based therapy – Amimestrocel injection (hUC-MSC) has received a conditional approval nationwide.  

The move towards a more flexible clinical translation model in Hainan Province is not an isolated occurrence. The Hunan Province also passed a new provincial legislation – Regulations on Promoting the Cell and Gene Industry in Hunan Province (Draft) – to facilitate the commercialization of novel stem cell therapies. In attempt to position itself as a leader in regenerative medicine, China has undertaken steps towards reducing barriers for market entry of cutting-edge biomedical treatments. Nonetheless, provincial support for rapid clinical translation and expanded access to stem cell and gene therapies should be balanced against the need to ensure patient safety and efficacy. The Lecheng Pilot Project will thus become a significant litmus test for whether an expedited approval of innovative, experimental treatments is consistent with robust bioethical standards.

Key characteristics of the Hainan Lecheng pilot program

The Hainan Lecheng Pilot program operates under provincial supervision, limiting the administration of these newly approved stem cell therapies to two designated hospitals within the Lecheng Pilot Zone: Shanghai Xiaotong University Ruijin Hospital Hainan Hospital and Sichuan University West Lecheng Hospital. It aims to expedite patient access to low-risk experimental stem cell treatments, thus facilitating their clinical translation. The therapies currently included in the pilot are for knee osteoarthritis, chronic obstructive pulmonary disease (COPD), and complications after bone marrow transplantation.

The model moves away from China’s established regulatory framework, which classified stem cell therapies as drugs subject to extensive preclinical and clinical testing to ensure the safety and efficacy prior to market approval. It also challenges international best practices, particularly the International Society for Stem Cell Research (ISSCR)’s guidelines, which recommend the use of unproven therapies only under tightly controlled, patient-specific investigational protocols. Creating an accelerated pathway for experimental treatments at the provincial level could prove to be a double-edge sword, one that prioritizes fast clinical results, while also raising significant ethical and safety concerns.

Hainan’s approach potentially offers several distinct advantages. First, it provides patients with severe, hard-to-treat conditions with much needed early access to experimental therapies. This could be beneficial to patients with late-stage osteoarthritis, to whom common treatments only offer limited relief. Second, the Pilot facilitates the collection of real-world clinical data that could shape future regulatory choices and optimize therapeutic protocols. Finally, it can help establish Hainan as a center for biomedical innovation and attract international investment and collaboration, ultimately enhancing China’s leadership in the biomedical field.

Ethical issues related to expanded access to stem cell and gene therapies

It is important that the new regulatory model incorporates rigorous safeguards such as strict treatment protocols, adverse event monitoring, and preventing unauthorized use. High ethical standards for experimental treatments should be maintained at all time, and only qualified institutions should be allowed to administer them. This will prevent unauthorized clinics from capitalizing on the pilot program’s flexibility to offer similar or unproven stem cell interventions that can put vulnerable patients at risk. The history of stem cell tourism should serve as a stark warning. In addition to restricting patient access to the two hospitals in the Lecheng Pilot Zone, robust institutional ethics review is needed to ensure compliance with clinical protocols and local regulatory agencies should be more proactive in carrying out regular audits and enforcing penalties for violations.

While Lecheng authorities have emphasized pricing transparency as an objective of the Pilot, this alone is insufficient to ensure patient safety. Transparency requires mandated disclosure of clinical outcomes, particularly adverse events. All aspects of clinical studies should be made openly accessible to allow for independent verification of results, thereby enhancing transparency and accountability. It is also important to establish comprehensive adverse event monitoring systems to detect and record systematically immediate complications and long-term effects. Making such data accessible to researchers and regulators can support evidence-based decisions and build trust with the global medical community. Putting these safeguards in place will deter premature commercialization and ensure that only treatments with verified safety and effectiveness reach the market.

Since the approved therapies do not fall under the category of traditional drugs, proactive steps are necessary to combat the misinformation and hype surrounding stem cell therapies that often exaggerates their effectiveness and prompts vulnerable patients to opt for risky interventions. Authorities at both central and provincial levels can enhance public knowledge by establishing dedicated webpages with accurate information about the status and availability of stem cell therapies, including where patients can receive the treatment, what it comprises, and how it is administered. Furthermore, it is important to flag any misleading content to help reduce the negative impact of misinformation. Medical professionals and researchers can support patients to make informed decisions based on scientific evidence, rather than sensationalized narratives, by leveraging various channels such as news reports and social media to educate the public about the current limitations and risks of emerging therapies.

The way forward

The fast-track approvals of stem cell and gene therapies in Hainan, alongside new legislation facilitating clinical translation in other provinces, have indicated a strong commitment by Chinese local governments to advance biomedical innovation through removing regulatory hurdles to market entry of regenerative medicine therapies. However, the new approach is not without its risks to patient safety. As China continues to strive for global leadership in regenerative medicine, striking a good balance between innovation and patient protection remains crucial.

To ensure long-term success of the proposed model, regulatory authorities need to strengthen post-market oversight, transparency, and alignment with international guidelines. This can help China become a responsible competitor in the global regenerative medicine space and increase public trust in innovative medical treatment it develops. It can further promote international collaborations and harness the potential of stem cell research while minimizing harm to patients.

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Dr. Li Du is an Associate Professor in the  Faculty of Law at the University of Macau in Macau SAR., specializing in international law, food law, biotechnology law and policy, and data protection law. His current research focuses on the legal and ethical implications of novel and emerging biotechnologies, e-health, and generative AI in biomedical research.