Is a Cure in Sight for the Incurable 'Pediatric Diabetes'?

Diabetes Called Adult Disease
'Type 1 Diabetes' Has Genetic Factors... Can Occur in Childhood
No Cure Except Pancreas Transplant

FDA Approves Immune Suppressant 'Teplizumab' to Delay Onset
Development of Cure Using Stem Cells Underway

'Tegild (active ingredient Teplizumab)', an immunosuppressant approved to delay the onset of type 1 diabetes (Photo by Prevention Bio)

[Asia Economy Reporter Lee Chun-hee] Diabetes is commonly known as an "adult disease" because it usually occurs in adults over 40 years old. However, this term is limited to type 2 diabetes. Type 1 diabetes, caused by genetic factors, often develops in childhood and is also called "pediatric diabetes." Type 1 diabetes has been an intractable disease with no fundamental cure, requiring lifelong insulin injections. However, expectations are rising that a path to treatment may open as the U.S. Food and Drug Administration (FDA) has approved the first-ever treatment for type 1 diabetes.

According to industry sources on the 21st, the FDA recently approved ProventionBio's immunosuppressant "Tegzelt" (generic name: teplizumab), which delays the onset of type 1 diabetes. This is the first time a drug delaying the onset of type 1 diabetes has received regulatory approval. Last month, global big pharma Sanofi acquired U.S. rights, reflecting high market expectations.

Teplizumab is administered once daily over 30 to 60 minutes for 14 consecutive days. Considering the current price of $13,850 per vial, the total treatment cost amounts to $193,900 (approximately 261.92 million KRW). Clinical trials targeting high-risk groups for type 1 diabetes showed an average delay in onset of about two years, with 24.4 months in the control group and 48.4 months in the treatment group. The longest delay reported in clinical patients was 11 years.

Type 1 diabetes is an intractable disease in which immune cells attack and destroy pancreatic beta (β) cells, which produce insulin, due to exposure to environmental factors such as viruses and chemicals in individuals with genetic predisposition. Because insulin secretion is absolutely insufficient, patients must check blood sugar levels more than four times a day and regularly inject insulin.

In South Korea, it is reported that about 3 out of every 100,000 children under 15 years old develop type 1 diabetes. As of 2020, 44,552 people were known to be receiving insulin treatment due to type 1 diabetes.

Unlike type 2 diabetes, which can significantly alleviate symptoms through diet and exercise therapy even if it is difficult to cure completely, type 1 diabetes patients currently have no suitable treatment and must receive insulin injections for life. Pancreas transplantation can cure the disease, but it requires lifelong immunosuppressant medication as a limitation.

Vertex Pharmaceuticals logo (Photo by Vertex Pharmaceuticals)

However, teplizumab also only delays the onset by blocking T cells that attack pancreatic β cells and helping insulin secretion, so it is still not a fundamental cure or preventive drug for type 1 diabetes. Accordingly, some companies are pursuing the development of follow-up treatments that can completely cure type 1 diabetes through stem cells.

Vertex Pharmaceuticals in the U.S. is conducting phase 1 and 2 clinical trials of 'VX-880.' Although the FDA temporarily halted the trial due to dosage escalation issues, it resumed in July. One male participant in his 50s was declared cured as normal insulin secretion resumed, attracting attention. In July, Vertex also acquired regenerative medicine biotech company ViaCyte, which was developing another stem cell treatment for type 1 diabetes called 'VC-02,' for $320 million (approximately 433.8 billion KRW). In phase 1 and 2 trials last year, VC-02 showed results where insulin levels were maintained for a certain period after stem cell administration, reducing the required insulin dosage.

In South Korea, companies such as Tascom and Ensol Biosciences are conducting preclinical development. Tascom is developing a type 1 diabetes treatment based on induced pluripotent stem cells differentiated into pancreatic cells. The company aims to enter human clinical trials within two years. Ensol Biosciences is researching an oral drug called 'Siloa1000 (S1K),' a peptide new drug that suppresses T cell activation and enhances β cell proliferation.

This content was produced with the assistance of AI translation services.