First-in-human sonodynamic therapy with ALA for pediatric diffuse intrinsic pontine glioma: a phase 1/2 study using low-intensity focused ultrasound

Syed, Hasan R; Kilburn, Lindsay; Fonseca, Adriana; Nazarian, Javad; Oluigbo, Chima; Myseros, John S.; Packer, Roger J.; Keating, Robert F.

doi:10.1007/s11060-023-04269-8

Cited by 10 publications

(6 citation statements)

References 3 publications

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“…Apart from basic science applications, SDT holds potential for clinical translation 158 , 159 . For example, Zha et al have initiated a phase I clinical trial SDT combined with temozolomide for the treatment of recurrent glioblastoma, a cancer with poor prognosis and ineffective treatments (Trial information: Chinese Clinical Trial Registry ( http://www.chictr.org.cn/ ): ChiCTR2200065992) 159 .…”

Section: Sonotheranosticsmentioning

confidence: 99%

“…However, the number of patients in this study was small and a long-term survival benefit was not demonstrated. In a separate study, Syed et al started both first in human phase I/II study with aminolevulinic acid for pediatric diffuse intrinsic pontine glioma SDT 158 . Preclinical results have shown that SDT through MR-guided focused ultrasound to activate protoporphyrin IX can slow the growth of gliomas and extend survival in animal models.…”

Section: Sonotheranosticsmentioning

confidence: 99%

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Theranostics - a sure cure for cancer after 100 years?

Song,

Zou,

Castellanos

et al. 2024

Theranostics

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Cancer has remained a formidable challenge in medicine and has claimed an enormous number of lives worldwide. Theranostics, combining diagnostic methods with personalized therapeutic approaches, shows huge potential to advance the battle against cancer. This review aims to provide an overview of theranostics in oncology: exploring its history, current advances, challenges, and prospects. We present the fundamental evolution of theranostics from radiotherapeutics, cellular therapeutics, and nanotherapeutics, showcasing critical milestones in the last decade. From the early concept of targeted drug delivery to the emergence of personalized medicine, theranostics has benefited from advances in imaging technologies, molecular biology, and nanomedicine. Furthermore, we emphasize pertinent illustrations showcasing that revolutionary strategies in cancer management enhance diagnostic accuracy and provide targeted therapies customized for individual patients, thereby facilitating the implementation of personalized medicine. Finally, we describe future perspectives on current challenges, emerging topics, and advances in the field.

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Section: Sonotheranosticsmentioning

confidence: 99%

Section: Sonotheranosticsmentioning

confidence: 99%

Theranostics - a sure cure for cancer after 100 years?

Song,

Zou,

Castellanos

et al. 2024

Theranostics

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“…A description of that first patient treatment, which treated the entire right half of the pons in a 5-year-old child after she received a 5 mg/kg body weight i.v. dose of SONALA-001, was published as a Technical Communication [29]. Treatment was staged at that time for half of the pons on Day 1, followed by treatment of the second half of the pons four weeks later, to make certain that complications could be avoided.…”

Section: First-in-child Pediatric Sdt Trial For Diffuse Intrinsic Pon...mentioning

confidence: 99%

“…At this point in time in the development of ALA SDT as a new therapeutic procedure in neuro-oncology, it is important to note that, to date, the safety of SDT appears to be equal to or greater than that of ALA iPDT for the treatment of rGBM [18][19][20]. No procedurerelated serious adverse events have been reported [29], and except for the need to monitor patients overnight for regulatory-grade clinical trial safety monitoring, ALA SDT can be developed as an outpatient procedure carried out within a single day. Increases in levels of biomarkers such as apoptosis and lipoperoxide liberation from the SDT-treated tissue support the hypothesis that a PDT-like mechanism resulting in membrane disruption is a likely contributing mechanism of action [27].…”

Section: Conclusion and Future Directions In The Evolution Of And Res...mentioning

confidence: 99%

“…An additional question to consider for future research is the explanation of the absence of inflammatory processes observed post-ALA iPDT or SDT for malignant gliomas in humans and in animal malignant glioma models. The iPDT process involves actual device insertion and cell death occurring within a few days, yet MRIs taken during the acute post-iPDT or SDT period have shown no signs of inflammation or pseudoprogression [18][19][20]27,29]. Recently, Liu et al [34] used paired single-cell imaging and RNA sequencing of human GBM surgical specimens from patients who had received 5-ALA prior to surgery to demonstrate that cellular labeling, via PpIX fluorescence, was not specific to neoplastic glioma cells.…”

Section: Conclusion and Future Directions In The Evolution Of And Res...mentioning

confidence: 99%

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Theranostic Uses of the Heme Pathway in Neuro-Oncology: Protoporphyrin IX (PpIX) and Its Journey from Photodynamic Therapy (PDT) through Photodynamic Diagnosis (PDD) to Sonodynamic Therapy (SDT)

Marcus,

de Souza

2024

Cancers

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ALA PDT, first approved as a topical therapy to treat precancerous skin lesions in 1999, targets the heme pathway selectively in cancers. When provided with excess ALA, the fluorescent photosensitizer PpIX accumulates primarily in cancer tissue, and ALA PDD is used to identify bladder and brain cancers as a visual aid for surgical resection. ALA PDT has shown promising anecdotal clinical results in recurrent glioblastoma multiforme. ALA SDT represents a noninvasive way to activate ALA PDT and has the potential to achieve clinical success in the treatment of both intracranial and extracranial cancers. This review describes the creation and evolution of ALA PDT, from the treatment of skin cancers to PDD and PDT of malignant brain tumors and, most recently, into a noninvasive form of PDT, ALA SDT. Current clinical trials of ALA SDT for recurrent glioblastoma and high-grade gliomas in adults, and the first pediatric ALA SDT clinical trial for a lethal brainstem cancer, diffuse intrinsic pontine glioma (DIPG), are also described.

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In Vitro Sonodynamic Therapy Using a High Throughput 3D Glioblastoma Spheroid Model with 5‐ALA and TMZ Sonosensitizers

Datta,

Lee,

Moolayadukkam

et al. 2024

Adv Healthcare Materials

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Sonodynamic therapy (SDT) administered using low‐intensity pulsed ultrasound and sonosensitizers is an emerging, minimally invasive, targeted deep‐tissue therapy for solid tumors such as glioblastoma multiforme (GBM). Initial clinical trials show promising outcomes for SDT treatments of GBM. A crucial aspect of SDT is the sonosensitizer that interacts with ultrasound, facilitating energy transfer to the tumor, thus inducing therapeutic efficacy. Current in vitro methods for determining the therapeutic efficacies of sonosensitizers are time‐consuming and expensive. A novel high‐throughput magnetically printed 3D GBM model is used to overcome this challenge. The hypothesis is that the use of two sonosensitizers, one a chemotherapeutic drug, enhances SDT efficacy through their additive chemical interactions. The GBM model is used to evaluate the effectiveness of two sonosensitizer molecules, 5‐aminolevulinic acid (5‐ALA) and theU.S. Food and Drug Administration (FDA)‐approved chemotherapeutic drug Temozolomide (TMZ). It is confirmed that implement high‐throughput GBM models to evaluate sonosensitizer combinations and their efficacies is feasible and, for the first time, show that the combined effect of both sensitizers, 5‐ALA and TMZ, is superior for preventing spheroid growth than employing each molecule separately. This finding is relevant for future clinical trials of GBM treatment with SDT.

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First-in-human sonodynamic therapy with ALA for pediatric diffuse intrinsic pontine glioma: a phase 1/2 study using low-intensity focused ultrasound

Cited by 10 publications

References 3 publications

Theranostics - a sure cure for cancer after 100 years?

Theranostics - a sure cure for cancer after 100 years?

Theranostic Uses of the Heme Pathway in Neuro-Oncology: Protoporphyrin IX (PpIX) and Its Journey from Photodynamic Therapy (PDT) through Photodynamic Diagnosis (PDD) to Sonodynamic Therapy (SDT)

In Vitro Sonodynamic Therapy Using a High Throughput 3D Glioblastoma Spheroid Model with 5‐ALA and TMZ Sonosensitizers

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