Next month, patients with severe, rare, and incurable diseases such as arthritis, autoimmune diseases, Parkinson's disease, and cancer will be able to receive stem cell and immune cell therapies currently in the development stage.
Until now, patients in South Korea were unable to use developing cell and gene therapies except to participate in clinical research due to regulations. Consequently, many patients traveled to Japan for treatment, but the law will be amended and come into effect next month.
According to the Ministry of Health and Welfare, the revised 'Advanced Regenerative Bio Law (Law on the Safety and Support of Regenerative Medicine and Advanced Biopharmaceuticals),' which passed the National Assembly in February last year, will come into effect on Feb. 21. Commonly referred to as the 'Regenerative Medicine Law,' this legislation was enacted in August 2020 to allow new advanced regenerative medical technologies like cell and gene therapies for diseases without available treatments or serious, rare, and incurable conditions.
Previously, cell and gene therapies that had confirmed safety and efficacy in the development research stage could only be used for research purposes and not for treatment. The core of the revised law allows this usage and enables hospitals and corporations to partially monetize these therapies. Representative therapies include those using stem cells or immune cells such as T cells and natural killer (NK) cells, as well as gene therapies that utilize gene-editing technologies to remove specific genes that can cause diseases.
Currently, the Office for Government Policy Coordination and the Legislation Research Institute are reviewing relevant subordinate laws ahead of the law's implementation. A government official noted, "We plan to announce the subordinate laws before the Advanced Regenerative Bio Law takes effect on Feb. 21, and the Ministry of Health and Welfare will also hold information sessions."
◇ Hospitals to review treatment plans before starting therapies
Once the law is implemented, patients will not be able to receive regenerative medical procedures such as stem cell injections immediately at hospitals. They must first obtain approval for safety and treatment plans through the Regenerative Medicine Review Committee jointly under the Ministry of Health and Welfare and the Ministry of Food and Drug Safety.
When a hospital designated as a regenerative medicine institution submits a treatment plan, the committee reviews it and decides whether to approve it. Subsequently, medical staff will apply the treatment to the patient. This procedure is in place to ensure patient safety, as these are unapproved drugs.
Jeong Soon-gil, director of the Regenerative Medicine Policy Division at the Ministry of Health and Welfare, explained, "We hold the review committee on the third Thursday of every month to determine the appropriateness of clinical research plans, and starting from the revised law's implementation date of Feb. 21, regenerative medical institutions will begin submitting their treatment plans. Thus, we will start addressing this from the March review meeting."
The review committee is currently chaired by Kim Dong-ik, a professor at Samsung Medical Center, and consists of 20 members, including professors from major universities and university hospitals and heads of patient organizations. The designated regenerative medical institutions following the review process total 112, including major university hospitals, general hospitals, and clinics across the country. Since it is a non-reimbursable market not covered by health insurance, the treatment costs that patients must bear may vary by medical institution.
◇ High demand for treatment of arthritis and autoimmune diseases
There are almost no restrictions on target diseases. Jeong Soon-gil, director of the Regenerative Medicine Policy Division, explained, "This law considers that patients need various treatment options, targeting diseases without treatments or serious, rare, and incurable conditions, allowing for a broad application scope."
However, it will be difficult for healthy individuals to receive developing cell or gene therapies simply for preventive, cosmetic, or aesthetic purposes aimed at anti-aging or rejuvenation. Jeong Soon-gil noted in response to inquiries that "(the Advanced Regenerative Bio Law) legally aims to treat serious, rare, and incurable diseases," adding, "It will not be easy for preventive purposes, such as anti-aging, to be immediately included in treatment plans."
Experts expect that the demand for regenerative medicine will increase in disease groups with many patients but lacking fundamental treatments. Arthritis is often cited as a representative example. The current treatments for arthritis, where cartilage is destroyed and inflammatory changes occur, only include injections to alleviate pain or surgeries to replace joints.
Choi Seung-ho, CEO of Panacell Biotech, which is developing stem cell and exosome treatments, stated, "The demand for stem cell therapies will be greatest in the field of arthritis," explaining that the principle involves injecting stem cells into damaged cartilage tissue to assist the differentiation of the stem cells into cartilage tissue, with various techniques, methods for injecting cells, and ratios depending on the joint scale.
Choi noted, "The areas of autoimmune diseases and respiratory diseases such as lung fibrosis and asthma, which have increased in patients after the COVID-19 pandemic, will also find new alternatives using the regenerative capabilities of stem cells." He explained, "Conditions like polycystic ovary syndrome, where hormonal balance is disrupted with increased male hormones and ovulation disorders, systemic lupus erythematosus (SLE), a chronic inflammatory autoimmune disease, and congenital scoliosis, which causes deformities and growth disorders in the head, chest, and limbs, can also achieve therapeutic effects through injections of stem cells and exosomes derived from stem cells."
Exosomes are small substances derived from cells, ranging from 50 to 200 nm in diameter, that assist in signaling between cells. Choi explained, "Exosomes contain various bioactive substances, providing diverse effects, including cell regeneration and removal of reactive oxygen species," adding that "numerous studies have shown therapeutic effects."
Stem cell treatments may be used to alleviate and improve symptoms in patients with Parkinson's disease, who experience tremors or sluggish movements, and attempts may also be made to enhance the effects of cancer treatment using immune cells such as T cells and natural killer (NK) cells.
◇ "Momentum for developing therapies... expectations for expansion of the regenerative medicine market"
With the law's implementation accumulating patient data, it is forecasted that therapy development will accelerate, and similar to Japan, domestic and foreign demand may increase, opening new markets. As patients can now be billed for treatments, there are expectations that the financial structures of therapy development corporations may improve. Consequently, investors seeking beneficiary corporations are also on the rise. It is crucial to examine whether they are presenting significant clinical research data.
ENCell is developing EN001, a treatment for Charcot-Marie-Tooth disease (CMT), a rare and untreatable disease characterized by muscle atrophy in the hands and feet. This is a mesenchymal stem cell therapy cultured using proprietary technology. It works by traveling to damaged nerves to secrete therapeutic substances and regenerate the nerve-myelin surrounding the nerve cells. The company confirmed the safety and exploratory therapeutic effects of EN001 in a low-dose group clinical study involving patients and initiated a high-dose group trial for patients with CMT type 1A in December last year, planning to complete patient administration by next year.
S.Biomedics is developing TED-A9, a Parkinson's disease treatment utilizing embryonic stem cells. Since 2023, it has been conducting clinical trials of phases 1 and 2a for 12 domestic Parkinson's disease patients, recently drawing interest with research results showing that a patient who received an embryonic stem cell treatment improved to the extent of playing badminton a year later. Kim Dong-wook, CHIEF Technology Officer of S.Biomedics, stated, "Once the Advanced Regenerative Bio Law revision takes effect, treatments for Parkinson's disease will become possible more quickly."
CHA Biotech announced that it plans to conduct clinical studies for its autologous NK cell cancer therapy 'CHANK-101' targeting patients with glioblastoma, a type of brain tumor. GI Innovation is advancing tests evaluating a combination therapy of allogeneic NK cell therapy from its subsidiary GI Cell and next-generation immuno-oncology drug GI-101A among patients with recurrent and refractory solid tumors.
☞ Stem cells
Stem cells are cells that generate the cells that make up the body such as bones, brain, muscles, skin, and heart. They can be obtained from embryos, bone marrow, or umbilical cord blood, and their application range becomes vast depending on how they are grown. There are three main methods for creating stem cells: extracting and culturing cells from the embryonic stage that undergo vigorous division (embryonic stem cells, ESC), applying genetic technology to fully grown cells like skin cells to revert them to a primitive state (induced pluripotent stem cells, IPSC), and extracting and culturing stem cells found in umbilical cord blood, bone marrow, or adipose tissue (adult stem cells).