Early-Stage Findings of Ketogenic Agent CER-0001 in Infantile Spasms Warrant Further Investigation


Half of the treated individuals saw a reduction of more than 50% in the number and duration of epileptic spasms following treatment with CER-0001, a ketogenic therapy.

Marc Cantillon, chief medical officer at Cerecin

Marc Cantillon

In a small-scale, open-label study (NCT04727970) of infants with infantile spasms, findings showed that treatment with CER-0001 (Cerecin), an investigational ketogenic agent, may be therapeutically beneficial, as demonstrated through reductions in number and duration of epileptic spasms. Presented at the 35th International Epilepsy Congress, held September 2-6, in Dublin, Ireland, investigators concluded that the data warrant continued investigation.

The study, expected to enroll 10 individuals, included 6 infants aged 3-24 months with infantile spasms who failed treatment with vigabatrin and prednisolone. Using random sample of blood ß-hydroxybutyrate levels, all individuals entering the study were in ketosis by the end of the titration phase, which lasted 5-14 days, and in the maintenance phase where treatment was given for 7 days.

After undergoing repeat vEEG every 24 hours, findings showed that 3 of the 6 treated infants had demonstrated a response to treatment, defined as at least 50% reduction in number and duration of epileptic spasms. Presented by Marc Cantillon, chief medical officer at Cerecin, 1 serious adverse event of aspiration was found in the trial and considered related to CER-0001. Participants in Australia who demonstrated a clinical benefit at the conclusion of the maintenance period entered a 1-year open-label extension where they continued on treatment.

Top Takeaways

  1. CER-0001 Promising for Infantile Spasms: Investigational ketogenic agent CER-0001 exhibits promise in reducing epileptic spasms in infants resistant to previous treatments, with three out of six infants showing significant improvement.
  2. Safety Consideration: However, caution is warranted due to one reported serious adverse event related to CER-0001, emphasizing the importance of safety monitoring.
  3. CER-0001's Broad Neurological Potential: Beyond infantile spasms, CER-0001, also known as tricaprillin, demonstrates versatility as a potential treatment for various neurological conditions, offering potential benefits through its ketone-elevating mechanism.

CER-0001, also known as tricaprillin, is a specific medium chain triglyceride in development for neurologic conditions such as Alzheimer disease, migraine, and infantile spasms. The ketogenic agent is designed to elevated plasma ketone levels to leverage the numerous activities and benefits of ketone bodies.

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Ketogenic diet therapy has been one of the main treatments for drug-resistant epilepsy, with several different types of approaches that have shown efficacy. A recent multicenter retrospective study published in 2022 showed that age, medication, and glucocorticoid use prior to initiation did not impact the efficacy of ketogenic diet therapy for infants with infantile spasms. Conducted from October 2014 to March 2020, the trial featured 481 patients ranging 2 months to 20 years with infantile spasms treated with ketogenic diet.3

Data from that study showed that the rate of effectiveness of this approach increased at 3 months compared with 1 month, and then decreased gradually at 6 and 12 months. At 1, 3, 6, and 12 months after initiating treatment, the rates of seizure freedom were 6.9%, 11.6%, 16.0%, and 16.8%, respectively (X2 = 27.1772; P <.0001), with a significant difference between 3 months and 1 month (X2 = 6.5498; P = .0105) and between 6 months and 3 months (X2 = 3.8478; P = .0498) but not between 12 months and 6 months (X2 = 0.1212; P = .7278).

At the 2023 American Headache Society (AHS) Annual Meeting, held June 15-18, in Austin, Texas, findings from a pilot study (NCT04437199) assessing up to 60 g/day of CER-0001 suggested potential benefit in treating patients with migraine. Otherwise known as the RELIEF study, the trial showed no meaningful difference in the primary end point of change in monthly headache days after 3 months of treatment; however, investigators did observe a mean improvement of –2.75 days favoring the investigational agent at month 2. In the study, similar rates of treatment-emergent adverse events were observed in both the active (90.0%) and placebo (82.9%) arms.4

1. Cerecin to present new infantile spasms data at the 35th International Epilepsy Congress, Dublin 2023. News release. Cerecin. August 29, 2023. Accessed September 5, 2023. https://www.prnewswire.com/news-releases/cerecin-to-present-new-infantile-spasms-data-at-the-35th-international-epilepsy-congress-dublin-2023-301912765.html
2. Cantillon M, Lawson JA, Kossoff E, Chow L, Presanis J, Henderson S. Treatment of drug-resistant infantile spasms with CER-0001, a ketogenic agent: preliminary results. Presented at: 2023 EIC Congress; September 2-6; Dublin, Ireland. Abstract 1067
3. Ye Y, Sun D, Li H, et al. A multicenter retrospective cohort study of ketogenic diet therapy in 481 children with infantile spasms. Acta Epileptologica. 2022;4(11). doi:10.1186/s42494-021-00077-7
4. Chow L, Presanis J, McIntyre N, Henderson S, Cantillon M. Metabolic mechanism in migraine: trciaprilin, a ketogenic agent. Presented at: 2023 AHS Annual Meeting; June 15-18; Austin, TX. P-121.
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