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Tumor growth in patients with tuberous sclerosis complex on the ketogenic diet
Catherine J Chu-Shore1, Elizabeth A Thiele
1Harvard Medical School, Massachusetts General Hospital, 175 Cambridge Street, Suite 340, Boston, MA 02114, USA.
Purpose:
New evidence is emerging that the availability of nutrients plays a key role in regulating the mammalian target of rapamycin complex-1 (mTORC1) signaling pathway in human cancers. Tuberous sclerosis complex (TSC) is a genetic disorder which results in the growth of hamartomatous lesions in multiple organs due to insufficient suppression of the mTORC1 pathway. A minority of patients with TSC who develop epilepsy which is intractable to standard anticonvulsant medical and/or surgical treatments are treated with the ketogenic diet. To provide insight into the effects of nutrient manipulation on tumor growth in this condition, we describe our experience in a unique group of patients with known tuberous sclerosis complex who are on the ketogenic diet for seizure control.
Methods:
A retrospective chart review was performed of patients with TSC treated with the ketogenic diet between January 2002 and May 2007 at Massachusetts General Hospital.
Results:
Five patients with definite TSC underwent serial imaging for tumor growth while on the ketogenic diet or had unchanged imaging prior to the onset of the diet and after termination. Three out of five patients, all children, had progression of a known tumor or tumors or the development of a new tumor while on the ketogenic diet.
Conclusion:
In this limited case series of five TSC patients, the ketogenic diet did not induce tumor regression or suppress the growth of TSC-related tumors.
Insights
The ketogenic diet did not reduce tumor growth in children with tuberous sclerosis complex (TSC). This study observed tumor progression in most pediatric TSC patients on the diet for epilepsy management.
Area of Science:
- Oncology
- Genetics
- Metabolism
Background:
- Nutrient availability influences the mTORC1 signaling pathway, crucial in human cancers.
- Tuberous sclerosis complex (TSC) is a genetic disorder characterized by hamartomatous lesions due to dysregulated mTORC1 signaling.
- The ketogenic diet is used for intractable epilepsy in a subset of TSC patients.
Observation:
- This study reviewed five TSC patients on a ketogenic diet for seizure control.
- Tumor growth was monitored via serial imaging before, during, and after ketogenic diet intervention.
- The study focused on the impact of nutrient manipulation on tumor progression in TSC.
Findings:
- Three out of five pediatric patients with TSC experienced tumor progression or new tumor development while on the ketogenic diet.
- The ketogenic diet did not lead to tumor regression in any of the studied TSC patients.
- No suppression of TSC-related tumor growth was observed during ketogenic diet treatment.
Implications:
- The ketogenic diet may not be an effective strategy for managing TSC-related tumors.
- Further research is needed to understand the complex interplay between diet, mTORC1 signaling, and tumor growth in TSC.
- Findings highlight the need for careful monitoring of tumor development in TSC patients undergoing ketogenic therapy for epilepsy.
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