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Clinical research framework proposal for ketogenic metabolic therapy in glioblastoma

Authors: Tomás Duraj,Miriam Kalamian,Giulio Zuccoli,Joseph C. Maroon,Dominic P. D’Agostino,Adrienne C. Scheck,Angela Poff,Sebastian F. Winter,Jethro Hu,Rainer J. Klement,Alicia Hickson,Derek C. Lee,Isabella Cooper,Barbara Kofler,Kenneth A. Schwartz,Matthew C. L. Phillips,Colin E. Champ,Beth Zupec-Kania,Jocelyn Tan-Shalaby,Fabiano M. Serfaty,Egiroh Omene,Gabriel Arismendi-Morillo,Michael Kiebish,Richard Cheng,Ahmed M. El-Sakka,Axel Pflueger,Edward H. Mathews,Donese Worden,Hanping Shi,Raffaele Ivan Cincione,Jean Pierre Spinosa,Abdul Kadir Slocum,Mehmet Salih Iyikesici,Atsuo Yanagisawa,Geoffrey J. Pilkington,Anthony Chaffee,Wafaa Abdel-Hadi,Amr K. Elsamman,Pavel Klein,Keisuke Hagihara,Zsófia Clemens,George W. Yu,Athanasios E. Evangeliou,Janak K. Nathan,Kris Smith,David Fortin,Jorg Dietrich,Purna Mukherjee,Thomas N. Seyfried
Publisher: Springer Science and Business Media LLC
Publish date: 2024-12-5
ISSN: 1741-7015 DOI: 10.1186/s12916-024-03775-4
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The proposal for ketogenic metabolic therapy as a baseline for glioblastoma treatment is compelling. However, a few points warrant further discussion:

The article emphasizes the utility of the Glucose-Ketone Index (GKI) for monitoring therapeutic efficacy, yet the variability of GKI responses across patient populations with differing metabolic baselines (e.g., insulin resistance or cancer cachexia) remains underexplored. Could the authors elaborate on how GKI targets are adapted for such heterogeneous groups, particularly in relation to individual caloric and macronutrient needs?
While the study highlights KMT’s synergy with chemoradiotherapy, the potential risks of delaying standard-of-care treatments for interim KMT evaluation might raise ethical concerns. How do the authors propose addressing patient safety and long-term outcomes during such delays, especially for aggressive tumor subtypes?
The reliance on self-reported dietary compliance or intermittent biological markers in earlier studies is noted as a limitation. Could the authors suggest specific technologies (e.g., wearable glucose-ketone monitors) or protocols that future studies should adopt to ensure real-time, high-fidelity data collection?

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1 month ago

Thank you for your interest in our work.

The glucose-ketone index (GKI) is less variable than its comprising parameters (glycemia/ketonemia), but is still expected to change dynamically under different patient populations. As we have addressed in the manuscript and in the peer-review reports (which are also available along the article), the GKI is a proxy marker of the underlying biological processes of interest, which are not easily accessible for repeated sampling outside laboratory settings.

The goal of dietary KMT is to mimic the metabolism of fasting, leading to an overall reduction and stability in glycemia and corresponding increase in ketonemia, increased ecological competition for nutrients between non-tumoral and cancer cells, as well as reduced insulin/growth signaling and inflammation, leading to a metabolic state that is inhospitable for tumor growth. Therefore, the GKI would be a suitable marker when following a long-term eucaloric/calorically-restricted ketogenic diet or during fasting, but not as a single measurement, for example, after exogenous ketone supplementation under a high-carbohydrate diet. Naturally, several physiological processes can influence the GKI beyond food selection (e.g., stress responses, sleep quality, physical activity, drug treatments). Accordingly, patients should aim for the lowest, physiologically sustainable GKI over extended periods of time; many factors may increase variability, but the overall trend should be towards a chronic reduction from baseline.

Connecting with your last question: indeed, continuous measurement using both continuous glucose monitors (CGM) and continuous ketone monitors (CKM) would be the ideal monitoring tools to capture the area under the curve (AUC) of glycemia/ketonemia and associations with survival outcomes. This has been emphasized multiple times in the manuscript: “Depending on the trial budget, real-time CGM ± CKM is preferred, delivering more robust data collection and biofeedback”. CGM and CKM sensors are already available for both medical applications and over-the-counter (e.g., Freestyle Libre, SiBio KS1). Integrated, dual CGM/CKM sensors are being developed.

Lastly, we agree that delaying selected standard therapies to evaluate diet-drug KMT (particularly radiotherapy due to potential antagonism) has ethical implications that need to be addressed; changes in SOC timing would be considered for testing combined dietary and pharmacological KMT, not dietary KMT alone. The 5-year survival of patients diagnosed with GBM is less than 5%, which has not improved significantly in the last 50 years. While the contribution of chemoradiotherapy to this long-term survival is very small, it may still provide desirable transient disease control, which may be lower, equal or higher than diet-drug KMT. Beyond GBM, other tumor types and stages have unique risk/benefit considerations. However, we note that it is very difficult to design clinical trials when an established standard therapy is biologically antagonistic with the novel proposed therapy, which may be only applicable to specific timings and contexts (e.g., immunotherapy and corticosteroid use, or, as in this case, KMT and brain radiotherapy). Consequently, we propose that judicious SOC delay and/or dose-reduction with frequent monitoring may be the best compromise. Ultimately, we believe this should be the decision of the informed patient.

1 month ago

Thank you for your thoughtful response and for providing such valuable insights into the complexities of using the Glucose-Ketone Index (GKI) as a marker for ketogenic metabolic therapy (KMT) in glioblastoma treatment. Your explanation of how GKI variability can be influenced by factors such as stress, sleep, and physical activity is much appreciated and helps clarify the challenges of using it as a dynamic marker.

I particularly appreciate your mention of continuous glucose and ketone monitors (CGMs and CKMs) as ideal tools for real-time monitoring, and the development of integrated dual sensors seems like a promising step forward. This could significantly improve data collection, and I agree that incorporating these technologies into clinical trials could enhance the precision and personalization of KMT. It would be interesting to discuss further how these technologies can be practically adopted in clinical practice, especially in terms of patient education and training to ensure optimal use.

Regarding the ethical concerns with delaying standard-of-care treatments, your suggestion of balancing treatment delays with frequent monitoring is thoughtful. However, I wonder if there are ways to further mitigate the risks of such delays. For instance, are there specific biomarkers or imaging techniques that could be monitored during the delay to ensure that the patient’s condition does not worsen? Also, considering the potential for brain radiotherapy to be antagonistic to KMT, it would be helpful to explore how personalized dosing regimens could be adjusted during the overlap between the two therapies.

I look forward to continuing this conversation and exchanging more ideas on how we can refine this approach to optimize patient outcomes in glioblastoma treatment. Your insights into these complex interactions are incredibly valuable, and I’m excited to explore the potential of KMT further.

Thank you again for your time and thoughtful response!

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