Health ministry eases rules on autoimmune cell therapy, dropping prior trials

South Korea's health ministry said Friday it has issued a notice reclassifying clinical research and treatment using cultured autoimmune cells as low-risk, allowing researchers to apply to provide the therapy on the strength of published evidence rather than completing a preliminary clinical study that takes two to three years.

Autologous autoimmune cell therapy — cells drawn from a patient's own blood and cultured outside the body — was legally classed as medium-risk even though its safety was considered relatively well established, so anyone wanting to offer it first had to run the lengthy preliminary trial. Cancer patients for whom cell treatment was urgent faced waits of several years at home and the expense of seeking care abroad. Japan already treats proven autologous cell therapies as low-risk.

The ministry said its Advanced Regenerative Medicine and Advanced Biopharmaceuticals Review Committee examined the reclassification in June and confirmed the safety of cultured autoimmune cell research and treatment.

Applications must rest on cells that match those used in the cited literature. The origin, characteristics and mechanism of action of the cells have to be the same, and quality control test items are judged against data submitted by the cell-processing facility. The ministry said "the same" means the cells' generally known characteristics and specifications are confirmed and met, not that they are biologically identical.

Safety and efficacy are assessed on the basis of the cited literature or the results of earlier advanced regenerative medicine clinical studies. The treatment plan submitted must match the literature in indication — the target disease and patient group — therapeutic purpose and administration route, while cell dose and frequency may be considered within a similar range for which adequate grounds exist. Evidence must come from peer-reviewed papers at SCIE level or above, and applicants must supply the search strategy used to select them. Clinical results from a sufficient number of patients are required for the efficacy review, and both Korean and foreign literature may be used.

Collection, testing and processing of cells will continue to be carried out at existing cell-processing facilities, reflecting the specialised nature and safety demands of cell culture.

"With this notice, applications for cultured autoimmune cell treatment plans based on the literature are now possible even without conducting clinical research," said Choi Kyung-il, the ministry's advanced medical support officer. "Serious, rare and intractable disease patients will be our priority as we work to give them new treatment options."