The Wall Street Journal recently reported on two trials—one at the University of Pennsylvania (Penn), US and the other at Hangzhou Cancer Hospital, China. Both trials examine the impact of CRISPR-Cas9, a gene editing technology, on cancer.
The Penn trial has been awaiting regulatory clearance for the past two years. In contrast, the Hangzhou trial started administering gene-edited cells to patients within a year. Not a single patient has received CRISPR-Cas9 edited cells in the US, while 86 patients have been trialled in China already. This is surprising as CRISPR-Cas9 was pioneered in the US and Europe. Yet China appears to have taken the lead in its use for patients.
What has fuelled China’s speed in the field?
The simple answer is regulations (or the lack of it). Human clinical trials in the US require approvals from the National Institutes of Health (NIH) health advisory committee, the hospital’s ethics review committee and the US Food and Drug Administration (FDA).
The Penn trial has been cleared by the first two committees but are awaiting approval from FDA. On the other hand, trials in China only require approval from an internal hospital ethics committee. The Hangzhou trial was sanctioned in a single afternoon by a panel of doctors, a lawyer and a former cancer patient. This stark difference in regulation underscores the approach of the two countries to trials.
In the US, safety of trial participants is paramount, which explains the insistence on multiple approval steps. China prefers to accelerate trialling experimental therapies on terminally ill patients who would otherwise die of their diseases.
This cautious versus expeditious approach is also reflected in the consenting protocols of the two trials. The Penn trial patients have to be informed of the trial’s experimental nature, details of the treatment and possible unintended consequences. In contrast, the Hangzhou participants were merely told that the treatment would improve their immune system. They were given details of the procedure “based on their education”.
Gene editing has captured fascination because of its possible use in creating designer babies. That prospect is still many years away, but presently, gene editing can be tested as a palliative for terminal diseases. There are strong safety concerns with trialing a hitherto unused technology on human patients.
However, these concerns have to be contextualized by the number of terminally ill patients dying for want of treatment. India is yet to release a comprehensive gene editing policy. So what should our stance be? Do we go the US way or the China way?
Traditionally, India has aligned with international consensus on healthcare policies. For example, germline gene editing continues to be banned in line with international opinion. The debate on germline editing should not overshadow the more urgent testing of gene editing for healthcare treatments.
China, for example, seems to have decided on ethics and moved head with the trials. Reports of gene editing shrinking tumours in nasal cancer patients will most likely encourage others to take up gene editing trials. However, the technology is still in its nascent stages and clinical trials need to be contingent on robust demonstration of safety and efficacy.
Further, regulation needs to guard against biohackers, who may exploit people by offering inferior or unscientific solutions. Thus, it is essential to formulate robust regulations to promote gene editing research while balancing safety of recipients.
India’s current regulatory architecture for approving novel treatments is ambiguous and assigns overlapping functions to different governmental bodies. This framework needs to be restructured to optimize trial approval time while addressing safety requirements.
A two-step model wherein the government works with industry and research groups to accelerate clinical research is recommended. This model consists of a national apex committee working in collaboration with existing institutional ethics committees and independent accreditation agencies.
The national apex committee, consisting of scientists, clinicians, lawyers, patient and industry representatives, will formulate the guidelines and safety standards for the clinical trials. Given the current scope of gene editing treatments, these guidelines may be based on disease groups.
For example, the apex committee could release safety standards and trial protocol for use of gene editing in cancer or muscle dystrophy. The institutional ethics committee and accreditation agencies will ensure the implementation of these guidelines and be liable if safety standards are not met.
Any therapy which is successfully trialed would be identified for independent verification by the national apex committee to expedite its release. A vital feature of this framework is that there is no absolute moratorium on any technology. Instead, it provides for rigorous safety checks and monitoring mechanisms as the technology/application gets closer to being released to the public.
Its primary goal is to ensure that the regulatory environment is conducive to scientific progress. As such, the framework would allow India to harness the benefits of gene editing while keeping risks in check.
Precision medicine, led by gene-editing research and therapies, is certainly the future of healthcare. India needs to promote indigenous gene editing research to make such treatments available at affordable prices. Ease of doing research—both basic and clinical—stems from streamlined regulations and India now needs to balance safety and “time to approval” to ensure promotion of such research.
If we don’t, other countries which do develop these solutions would be free to charge rates they want. India has the resources and expertise to cultivate a research environment and foster innovation. However, we need to define a regulatory framework to facilitate this growth. This is India’s chance to tailor this cutting edge tool to its own requirements and ensure affordable healthcare to its people.
Shambhavi Naik is a Research Analyst at the Takshashila Institution.