ACTH Therapy for Autism and LKS: Key Findings from Our Recent Study

In our latest study, published in the Epilepsy and Behavior Journal by Elsevier, we explored the efficacy of ACTH therapy on EEG recordings in children with Autism Spectrum Disorder (ASD) and Landau-Kleffner Syndrome (LKS).

Link of the paper: https://doi.org/10.1016/j.yebeh.2025.110308

My journey into this research began when I first met my former principal investigator, Prof. İhsan Kara. He introduced me to the clinical applications of Prof. Atilla Altunel, who had been treating children with neurodevelopmental disorders for many years. Prof. Kara was particularly interested in these treatments due to the rising number of ASD cases in Turkey and worldwide.

As an elder brother of a child with autism, I understand firsthand how challenging life can be for both the affected children and their families. It is crucial to find effective treatments that help integrate these children into society, allowing them to lead normal lives.

Prof. Altunel had been working extensively in the field of neurodevelopmental disorders, particularly focusing on EEG abnormalities in ASD and LKS. Although his clinic has not been open since 2020, his years of experience and previous cases provided valuable insights for our study. He often discussed the presence of Electrical Status Epilepticus during Sleep (ESES) in LKS and various abnormalities in ASD, which we later identified as interictal epileptiform discharges (IEDs). For years, he applied ACTH therapy to children with LKS and ASD, tracking their EEG results.

While his clinical observations suggested significant improvements, the lack of standardized assessment batteries made it difficult to scientifically validate the therapy’s impact, particularly in terms of social and neuropsychiatric outcomes. Due to the absence of structured evaluations in these areas, the full extent of the therapy’s benefits beyond EEG improvements remains uncertain.

From thousands of cases, we analyzed 236 children, eliminating many due to comorbidities and age criteria. Among these 236 children with ASD or LKS who were previously treated by Prof. Altunel, ACTH therapy was administered with varying phases tailored to the abnormalities in each EEG recording. Our findings revealed significant improvements in seizure control and EEG abnormalities:

• In children with LKS and epileptic seizures, the mean ESES index reduction was 49.9% (±17.7), with 50% achieving complete seizure control.

• In children with ASD and epileptic seizures, the mean IED index reduction was 47.2% (±16.7), with 41% achieving complete seizure control.

• Side effects were minimal, transient, and reversible, with no reports of serious adverse events.

Changes in EEG indexes (ESES for LKS and IED for ASD) before and after ACTH treatment in children with and without epileptic seizures. (A) ESES index in children with LKS and epileptic seizures, (B) ESES index in children with LKS without epileptic seizures, (C) IED index in children with ASD and epileptic seizures, and (D) IED index in children with ASD without epileptic seizures. The box plots reveal the distribution of indexes before and after treatment.

For future studies, it is essential to investigate the impact of ACTH therapy for autism and LKS on psychiatric and social aspects. Although not explicitly mentioned in our paper, Prof. Altunel strongly believed in the social benefits of the treatment. We hypothesize that both ESES and IEDs interfere with neurodevelopment, negatively affecting a child’s growth and learning abilities. If addressed with the appropriate treatment, these children may have a better chance at education, social integration, and leading a healthier life.

Further research should incorporate both electrophysiological and neuropsychiatric metrics to improve treatment approaches. Accurate diagnosis is also crucial, as autism exists on a spectrum. A genetically rooted ASD case may not respond to the same treatment as a child with mild autism. Future studies should refine treatment strategies accordingly.

In the coming months, we plan to share our findings on the effects of organic food on children with autism. Our research continues to focus on treating neurodevelopmental disorders using organic molecules. Additionally, we aim to enhance the efficiency of sleep EEG analysis for neurodevelopmental disorders. By reducing the time required for analysis and minimizing false diagnoses, we hope to enable faster diagnosis and more effective treatment. To achieve this, we are actively developing artificial intelligence methods for detecting EEG abnormalities.

There is still much to explore in this field, but we remain hopeful that our research will lead to better outcomes for children with ASD and LKS.