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Children at risk of diabetes type 1. Treatment with acetyl-L-carnitine plus nicotinamide - case reports
Ivana Cristina Fernandez1, María Del Carmen Camberos, Gisel Anabel Passicot
1Hospital de Niños Ricardo Gutierrez, Buenos Aires, Argentina.
Insights
Acetyl-L-carnitine and nicotinamide treatment shows promise for children at risk of type 1 diabetes. This intervention may delay or reverse diabetes development in at-risk pediatric populations.
Area of Science:
- Immunology
- Endocrinology
- Pediatrics
Background:
- Type 1 diabetes (T1D) is an autoimmune disease with increasing incidence in children.
- Early intervention in at-risk individuals is crucial for preventing disease onset.
- Identifying effective and safe therapeutic strategies for T1D prevention is a priority.
Observation:
- A study evaluated acetyl-L-carnitine (ALCAR) and nicotinamide (NIC) in children at risk for T1D.
- Participants were characterized by HLA-DQB1 and positive autoantibodies.
- Children with an initial insulin response peak ≤48 µU were randomized into control and treatment groups.
Findings:
- Treatment with ALCAR and NIC showed positive outcomes in a subset of at-risk children.
- Four patients recovered their metabolic parameters, leading to medication suspension.
- Two patients exhibited favorable evolution while continuing treatment; two others showed slow progression with normal development.
Implications:
- This therapeutic approach may delay or induce remission of T1D in at-risk children.
- ALCAR and NIC represent a potential strategy for T1D prevention or early management.
- Further research is warranted to optimize treatment protocols and confirm long-term efficacy.
Unlabelled:
Abstract Objectives: The aim was to evaluate the treatment with acetyl-L-carnitine (50 mg/kg/day) and nicotinamide (25 mg/kg/day) in children at risk of type 1 diabetes. This treatment was effective and harmless in experimental type 1 diabetes in mice.
Patients:
Nine out of seventy healthy participants of the type 1 diabetes risk study were treated. They were typified for diabetes with HLA-DQB1 and positive autoantibodies. Children with a first peak of insulin response ≤48 µU were randomly distributed in control and treated patients. Children evolution was followed with an intravenous glucose tolerance test. Control children were treated when was another risk parameter was added. During their evolution all children were treated.
Results:
Treatment periods differ (range: 120-16 months) because children began treatment at different times. During the treatment 4 patients recovered their parameters and the medication was suspended; 2 patients continued the treatment with favorable evolution. Two children evolved slowly with normal growth and development. One girl became diabetic because she was treated late.
Conclusions:
In children at risk, this treatment delays the development or remits the evolution of type 1 diabetes.
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