(Health Korea News / Lee Chung-man) CAR-T treatment, also known as a one-shot treatment for blood cancer, is being explored for its effectiveness as a cure for autoimmune diseases.
CAR-T therapy is a cell therapy that collects T cells from a patient and then recombines them by inserting new genes so that they can actively seek out and destroy specific parts of the cancer cell surface. It is called chimeric antigen receptor-T because it is engineered like a chimera.
This drug was nicknamed a miracle anticancer drug because it showed a dramatic treatment effect in killing all cancer cells in patients with terminal blood cancer with just one dose. Therefore, the price is also very high, with the cost of one dose reaching approximately 500 million won.
Representative products are Novartis’ ▲’Kimria’ (ingredient name: tisagenlecleucel) and Legend Biotech’s ▲’Carvykti’ (ingredient name: ciltacabtagene autoleucel). The Ministry of Food and Drug Safety of Korea approved ‘Kimria’ and ‘Carvykti’ in March 2021 and March 2023, respectively.
The primary target of CAR-T therapy is an antigen on the surface of B cells, as most blood cancers are caused by overexpression of B cells.
B cells are immune cells involved in humoral immunity related to antibodies. When B cells are overexpressed, blood cancer occurs due to lymph node enlargement. Representative diseases include chronic lymphocytic leukemia (CLL) and non-Hodgkin’s lymphoma (NHL).
However, as B cells have recently been identified as a key cause of autoimmune diseases such as lupus and multiple sclerosis, CAR-T treatment is also attracting attention as a cure for autoimmune diseases.
In fact, when the University Hospital Erlangen in Germany administered CAR-T therapy to a patient with systemic lupus erythematosus (SLE) who was refractory to immunosuppressants in 2021, the patient was found to have completely disappeared from all pain.
Erlangen University Hospital has further expanded the study to explore the efficacy of a single dose of CAR-T therapy in 15 patients with B-cell-driven diseases such as SLE, idiopathic inflammatory myositis (IIM), and systemic sclerosis (SSc).
According to the results announced in February of this year, all patients showed improvement in symptoms after 15 months of medication. In particular, eight SLE patients achieved complete remission after medication, showing a treatment effect close to complete cure.
The University Hospital of Erlangen expressed its expectations, saying, “It is usually thought that people with autoimmune diseases have to take immunosuppressants for their entire lives, but CAR-T treatment will provide a new horizon for autoimmune diseases.”
Encouraged by this news, Korean companies are also attempting to develop CAR-T treatment as a cure for autoimmune diseases.
Curocell is a representative example. Curocell is the first company in Korea to receive approval for a CAR-T treatment clinical trial plan (IND). The company has completed a phase 2 clinical trial evaluating its candidate substance, ‘Anbal-cel’, in relapsed or refractory diffuse large B-cell lymphoma (DLBCL).
In April of this year, the next-generation anti-CD19 CAR-T treatment R&D project for refractory SLE treatment was selected as a target for support from the 2024 inter-ministerial regenerative medicine technology development project, and the development of CAR-T treatment for autoimmune diseases is now underway in earnest.
The company plans to study whether its CAR-T treatment ‘Anvalcel’ can expand its indications to SLE. To this end, it plans to receive support of 1.07 billion won and apply for an IND to the Ministry of Food and Drug Safety at the end of 2025.
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Source: www.hkn24.com
