Korea Approves Stem Cell Studies for Intractable Diseases

Clinical research on cell therapies aimed at treating intractable diseases with limited treatment alternatives, such as severe pain, has received an "appropriate" ruling from the government.

According to the Ministry of Health and Welfare on Friday, the 7th Advanced Regenerative Medicine and Biopharmaceutical Review Committee of 2026, held the previous day, reviewed six implementation plans submitted by regenerative medicine institutions and two long-term follow-up survey plans submitted by the advanced regenerative medicine safety management agency (the National Institute of Health under the Korea Disease Control and Prevention Agency). Advanced regenerative medicine refers to treatments that use human cells and other materials to regenerate, restore, or form the structure and function of the human body, or to treat or prevent disease. It includes cell therapy, gene therapy, tissue engineering therapy, and convergence therapy.

The three projects ruled "appropriate" in the review are medium-risk, multicenter cell therapy clinical studies. The first project is research targeting patients with knee osteoarthritis. Current treatments for osteoarthritis include exercise therapy, drug treatment, and injection or surgical treatment. However, drug and injection treatments have short-lived effects and require repeated treatment, while surgical treatment carries limitations such as the risk of complications.

This study is a "randomized, double-blind, placebo-controlled" trial in which mesenchymal stem cells derived from the patient's own fat are confirmed by ultrasound and administered into the knee joint cavity (the internal space of the knee joint where the thigh bone and shin bone meet).

A randomized, double-blind, placebo-controlled study randomly assigns patients and blinds both the recipients and the medical staff to whether the real drug is being administered, enhancing the fairness of evaluation. At the same time, by comparing efficacy with a group receiving a placebo (a fake drug containing no active ingredient), it can objectively prove the treatment's effect.

The second project ruled "appropriate" concerns patients with severe chronic neuropathic pain of dental origin. Currently, drug treatments such as gabapentinoids, tricyclic antidepressants, and serotonin-norepinephrine reuptake inhibitors are recommended for chronic neuropathic pain. However, this treatment has the limitation of only temporarily relieving symptoms rather than correcting the underlying cause. This study is also a randomized, double-blind, placebo-controlled, medium-risk, multicenter cell therapy clinical study in which stem cells derived from the patient's own fat are administered.

The third project is research targeting patients with recurrent glioblastoma, in which natural killer (NK) cells derived from the patient's own blood are administered. Glioblastoma is an aggressive brain tumor that proliferates very rapidly and infiltrates extensively. It has a high recurrence rate, and no standard treatment has been established after recurrence.