A Phase 1/2 Dose Escalation and Expansion Study of Sonodynamic Therapy With SONALA-001 in Combination With Exablate 4000 Type 2.0 MR-Guided Focused Ultrasound in Subjects With Progressive or Recurrent Glioblastoma Multiforme (rGBM)

The primary objectives of this trial are to evaluate the safety, dose-limiting toxicities, maximum tolerated dose (MTD), maximum administered dose (MAD) and recommended Phase 2 dose (RP2D) for future study after a single treatment of SONALA-001 in combination with MRgFUS and to evaluate preliminary efficacy of sonodynamic therapy (SDT) using SONALA-001 and Exablate Type 2.0 device in subjects with progressive or recurrent GBM.

Start Date06 Feb 2023

Sponsor / Collaborator

SonALAsense, Inc.Startup

NCT05123534 / SuspendedPhase 2

A Phase 2 Dose-escalation Study Examining the Safety, Pharmacokinetics, and Preliminary Efficacy of Ascending Drug and Energy Dose Combinations for Sonodynamic Therapy Using SONALA-001 in Combination with Exablate 4000 Type 2.0 MR-Guided Focused Ultrasound in Subjects with Diffuse Intrinsic Pontine Glioma (DIPG) and Diffuse Midline Glioma (DMG)

The primary objectives of this trial are to evaluate the safety and tolerability of sonodynamic therapy (SDT) using SONALA-001 and Exablate Type 2.0 device and to determine the maximum tolerated dose (MTD) or recommended phase 2 dose (RP2D) of MR-Guided Focused Ultrasound (MRgFUS) energy in combination with SONALA-001 in subjects with diffuse intrinsic pontine glioma
Funding Source - FDA OOPD

Start Date12 Aug 2022

Sponsor / Collaborator

SonALAsense, Inc.Startup

NCT04559685 / RecruitingEarly Phase 1IIT

A Phase 0, First in Human, Open-label Study of Intravenous Aminolevulinic Acid HCl (ALA) and MR-Guided Focused Ultrasound Device (MRgFUS) in Participants With Recurrent High Grade Glioma (HGG)

A Phase 0 single center, first in human, open-label study of ascending energy doses of sonodynamic therapy (SDT) utilizing the MRgFUS combined with intravenous ALA to assess safety and efficacy in up to 45 participants with recurrent HGG. Eligible participants who are scheduled for resection will be administered intravenous (IV) aminolevulinic acid HCl (ALA) approximately six to seven (6-7) hours prior to receiving sonodynamic therapy (SDT).

Start Date15 Mar 2021

Sponsor / Collaborator

St. Joseph's Medical Center, Inc.

[+3]

100 Clinical Results associated with SONALA-001

100 Translational Medicine associated with SONALA-001

100 Patents (Medical) associated with SONALA-001

6,783

Literatures (Medical) associated with SONALA-001

01 Jan 2025·Colloids and Surfaces B: Biointerfaces

A hyaluronic acid-based dissolving microneedle patch loaded with 5-aminolevulinic acid for improved oral leukoplakia treatment

Article

Author: Sang, Zhiqin ; Zhang, Zhe ; Hao, Yuanping ; Zhu, Tingting ; Wang, Wanchun ; Qu, Xiaoru

INTRODUCTIONA local microneedle patch loaded with 5-aminolevulinic acid (ALA) was constructed to improve the efficiency of ALA photodynamic treatment of oral leukoplakia, reduce local photosensitivity reactions, and promote the healing of lesions.METHODSThe microneedle patch loaded with ALA was constructed with the hyaluronic acid (HA) solution (ALA-HAMN), and its morphology, strength, mucosal penetration, and biocompatibility were tested.RESULTSIn vivo safety and permeability tests confirmed that ALA-HAMN had good biocompatibility and could penetrate the mucosal barrier and quickly dissolve and release ALA for in situ transdermal administration. The 4-nitroquinoline oxide (NQO) rat model experiment showed that ALA-HAMN can significantly improve photodynamic therapy (PDT) efficiency and has no damage to mucosal tissue compared with the commonly used ALA cotton ball dressing.CONCLUSIONSThe ALA-loaded microneedle patch was successfully constructed for the photodynamic treatment of oral leukoplakia, and the photodynamic efficiency and comfort of oral leukoplakia were improved, which provided an effective delivery mode to improve clinical ALA-PDT treatment of oral leukoplakia (OLK).

01 Jan 2025·Journal of Affective Disorders

Effects of a food supplement containing phosphatidylserine on cognitive function in Chinese older adults with mild cognitive impairment: A randomized double-blind, placebo-controlled trial

Article

Author: Zhang, Chunlai ; Zhang, Meilin ; Zhao, Jiangang ; Chen, Yongjie ; Yan, Jing ; He, Ruikun ; Yue, Zhongbao ; Ma, Fei ; Li, Wen ; Wang, Moyan ; Yang, Tong ; Li, Zhenshu ; Huang, Guowei ; Duan, Huilian ; Xu, Ning

BACKGROUNDPhosphatidylserine (PS) and α-Linolenic acid (ALA), are positively associated with cognitive function, but their combination effects and possible mechanisms remain unclear. We aimed to explore the effects on cognition and potential mechanism of the supplements.METHODSThis randomized, double-blind, placebo-controlled trial recruited 190 MCI patients in Tianjin, China, and randomly assigned in intervention group and placebo group. Each group consumed two capsules every day for 12 months. Each capsule for intervention group contains 144 mg ALA, 31.5 mg PS and 3.6 mg Ginkgo total flavonoids as main functional components, with 0.48 mg Vitamin B₁ (as thiamine hydrochloride), 0.48 mg Vitamin B₆ (as pyridoxine hydrochloride) and 90 μg folic acid as supplement. Capsules for placebo group were identical but contain no active ingredients. Cognitive function, serum n-3 polyunsaturated fatty acids (PUFAs) and neurotransmitters were assessed at baseline and 12 months. Linear mixed effects model and causal mediation analysis were conducted to explore the effects and potential mechanism of the intervention.RESULTSA total of 190 participants (mean [SD] age, 67.95 [5.62] years; 70 (36.8 %) male and 120 (63.2 %) female) were randomized to the placebo group (n = 95) and intervention group (n = 95). Compared with placebo group, the intervention group had statistically significant improvements in arithmetic testing (β, 0.688; 95 % CI, 0.103-1.274), the similarity test (β, 1.070; 95 % CI, 0.472-1.667) and short-term memory (β, 0.600; 95 % CI, 0.399-0.800). Besides, the intervention group had statistically significant increases in serum ALA (β, 1.620; 95 % CI, 0.967-2.265), DHA (β, 2.797; 95 % CI, 1.075-4.532), EPA (β, 1.472; 95 % CI, 0.296-2.643), acetylcholine (β, 0.441; 95 % CI, 0.415-0.468), GABA (β, 0.009; 95 % CI, 0.001-0.016) and 5-HT (β, 0.160; 95 % CI, 0.081-0.238) compared to the placebo group. And the intervention may improve short-term memory by increasing serum ALA levels (average causal mediation effect = 0.132, 95 % CI, 0.053-0.225) with 19.7 % mediation proportion.CONCLUSIONSThis food supplement containing phosphatidylserine could improve different cognitive functions of MCI patients, especially short-term memory, and increase serum n-3 PUFAs and neurotransmitters levels. Serum ALA level might play a mediation role.

31 Dec 2024·Gynecological Endocrinology

Alpha lipoic acid administration improved both peripheral sensitivity to insulin and liver clearance of insulin reducing potential risk of diabetes and nonalcoholic fatty liver disease in overweight/obese PCOS patients

Article

Author: Prampolini, Greta ; Sponzilli, Alessandra ; Petrillo, Tabatha ; Foschi, Martina ; Semprini, Elisa ; Rusce, Laura ; Genazzani, Alessandro D. ; Ricciardiello, Francesco ; Battipaglia, Christian ; Aio, Claudia

OBJECTIVETo evaluate the effects of alpha lipoic acid (ALA) on hormonal and metabolic parameters in a group of overweight/obese Polycystic Ovary Syndrome (PCOS) patients.METHODSThis was a retrospective study in which thirty-two overweight/obese patients with PCOS (n = 32) not requiring hormonal treatment were selected from the database of the ambulatory clinic of the Gynecological Endocrinology Center at the University of Modena and Reggio Emilia, Italy. The hormonal profile, routine exams and insulin and C-peptide response to oral glucose tolerance test (OGTT) were evaluated before and after 12 weeks of complementary treatment with ALA (400 mg/day). Hepatic Insulin Extraction (HIE) index was also calculated.RESULTSALA administration significantly improved insulin sensitivity and decreased ALT and AST plasma levels in all subjects, though no changes were observed on reproductive hormones. When PCOS patients were subdivided according to the presence or absence of familial diabetes background, the higher effects of ALA were observed in the former group that showed AST and ALT reduction and greater HIE index decrease.CONCLUSIONALA administration improved insulin sensitivity in overweight/obese PCOS patients, especially in those with familial predisposition to diabetes. ALA administration improved both peripheral sensitivity to insulin and liver clearance of insulin. Such effects potentially decrease the risk of nonalcoholic fat liver disease and diabetes in PCOS patients.

News (Medical) associated with SONALA-001

03 Jun 2024

·www.prnewswire.com

Leading Cancer Researchers from NYU Langone's Perlmutter Cancer Center Present Latest Clinical Findings & Research at ASCO 2024 Annual Conference

NEW YORK , June 3, 2024 /PRNewswire/ -- Researchers from NYU Langone Health's Perlmutter Cancer Center are presenting their latest findings and research at the 2024 American Society of Clinical Oncology (ASCO) Annual Conference, held May 31 to June 4 at Chicago's McCormick Place. Among these presentations: a three-year update on the long-term efficacy of an mRNA vaccine for use in patients being treated for metastatic melanoma a retrospective look at treatment patterns and outcomes in patients with advanced endometrial cancer a look at the safety and efficacy of a noninvasive combination treatment for patients with recurrent glioblastoma "This year's crop of presentations is a showcase of the Perlmutter Cancer Center's commitment to delivering the highest level of research and clinical care," said Alec Kimmelman, MD, PhD, the center's director. Late-Breaker Rapid Oral Abstract: Three-year update on the long-term efficacy of an mRNA vaccine for use in patients being treated for metastatic melanoma Monday, June 3, 10:15AM CT In a follow-up presentation at ASCO to the recently published clinical trial describing the effectiveness of combining the immunotherapy drug pembrolizumab with V940—an investigational individualized neoantigen mRNA vaccine for people with high-risk, surgically removed melanoma—this three-year update looks at the longer-term recurrence rates of treated patients. The primary analysis of the phase 2 trial, led by Jeffrey S. Weber, MD, PhD, deputy director of Perlmutter Cancer Center, and sponsored by Moderna and Merck, assessed patients who received the vaccine, called mRNA-4157/V940, and the immunotherapy drug pembrolizumab, comparing their risk of recurrence and development of distant metastases to those who had received only pembrolizumab. This three-year follow-up analysis showed a higher percentage of durable and meaningful long-term recurrence-free survival or survival without distant spread of cancer in patients who received both treatments (74.8 percent) compared to those who received only pembrolizumab (55.6 percent). The patients' overall survival also improved with combination treatment, at 96.0 percent compared to 90.2 percent with pembrolizumab alone. "This additional information continues to support the potential of mRNA-4156/V940 in combination with pembrolizumab for patients with metastatic melanoma," said Dr. Weber. "We have followed up this work with a randomized phase 3 trial in more than a thousand patients, which we hope will provide a definitive assessment of the efficacy of the combination of V940 and pembrolizumab compared to pembrolizumab alone." Poster: A retrospective look at treatment patterns and outcomes in patients with advanced endometrial cancer Monday, June 3, 9AM This retrospective study, led by Bhavana Pothuri, MD, director of gynecologic oncology research at Perlmutter Cancer Center and a professor in the Department of Obstetrics and Gynecology, assesses testing for treatment options, treatment patterns, and outcomes among patients with advanced endometrial cancer. In the past decade, newer therapies have been introduced for advanced endometrial cancer. Molecular profiling of mismatch repair/microsatellite instability (MMR/MSI) status has become an important testing tool to determine treatment, but real-world data on the prevalence of its use is limited. Researchers looked at 1,441 deidentified patients with advanced endometrial cancer who started therapy between January 2018 and June 2023, analyzing patient characteristics, treatment patterns, and testing patterns. While MMR/MSI testing was common, less than 50 percent of patients were receiving new therapies, and further study with implementation science may aid in increased adoption. "Advanced endometrial cancer is the fourth most common cancer affecting women in the United States, and it is the only gynecologic malignancy with increasing incidence—and increasing mortality that is soon expected to surpass that of ovarian cancer," said Dr. Pothuri. "Despite increasing trends, there may be outstanding barriers to the adoption of new therapies. We need further studies to assess these changes in outcomes over a longer time frame, along with continued education to increase awareness and access to newer FDA-approved treatments." Poster: The safety and efficacy of a noninvasive combination treatment for patients with recurrent glioblastoma Saturday, June 1, 9AM Dimitris G. Placantonakis, MD, PhD, an associate professor in the Department of Neurosurgery, is presenting an ongoing, multicenter phase1/2 study that explores if sonodynamic therapy is safe and can help patients with recurrent or progressive glioblastoma live longer. Recurrent glioblastoma is a lethal brain tumor that has an extremely poor prognosis and no effective therapies. Sonodynamic therapy is a noninvasive combination treatment that uses a drug, aminolevulinic acid HCL (SONALA-001), and a device, the Exablate 4000 Type 2.0, to deliver focused ultrasound to target glioblastoma cells. This has been shown in previous studies to lead to tumor cell death and improved survival in animal models. People enrolled in this trial are receiving different dosage levels in order to determine the maximum tolerable dose for further study. The phase 2 portion will further characterize safety, along with evaluation of the efficacy of this treatment, with other key endpoints, including overall survival and recurrence rate. "We are very excited about this study because, as a new therapy, it offers hope for people with this disease," said Dr. Placantonakis. "This is also exciting because it offers a noninvasive treatment approach that pulses ultrasound waves from a device that is outside the head, so there is no need to surgically remove the tumor, and it eliminates the risk of side effects that are inherent in conventional brain surgery." Media Inquiries Marlene Naanes Phone: 646-754-5016 [email protected] SOURCE NYU Langone Health

Phase 2ASCOPhase 3Clinical ResultVaccine

14 Nov 2023

·www.biospace.com

FDA Grants Fast Track Designation to SonALAsense’s SONALA-001 in Combination with the INSIGHTEC Transcranial Ultrasound Device for Sonodynamic Therapy of DIPG, a Rare Pediatric Brain Cancer

Encouraging preliminary data from the ongoing DIPG clinical study to be presented at the 2023 Society for Neuro-Oncology Annual Meeting in Vancouver OAKLAND, Calif.--(BUSINESS WIRE)-- Sonalasense, a pioneer in the development of non-invasive Sonodynamic Therapy (SDT) using SONALA-001, today announced that the United States Food and Drug Administration (FDA) has granted Fast Track Designation to the development program of SONALA-001 in combination with the INSIGHTEC Exablate 4000 Type-2 Device for the treatment of patients with diffuse intrinsic pontine glioma (DIPG). This builds upon SONALA-001 also having received orphan drug designation in 2021 for malignant gliomas. The FDA’s Fast Track is a process designed to facilitate the development, and expedited review, of drugs to treat serious conditions and fill an unmet medical need. The program allows eligibility for more frequent meetings with the FDA to discuss the drug's development plan and ensure collection of appropriate data needed to support drug approval, more frequent written communication from FDA about such things as the design of the proposed clinical trials and use of biomarkers, and accelerated approval. “Receiving Fast Track Designation underscores the immediate and urgent need to bring new therapy options for DIPG patients,” said Ely Benaim, M.D., SonALAsense’s Chief Medical Officer and Executive Vice President of Development. “We are committed to working as efficiently as possible to bring SONALA-001 SDT to patients with this universally fatal disease.” DIPG is a rare, aggressive, and deadly pediatric brain tumor that strikes the pons, which links the brain to the spinal cord and helps control breathing, blood pressure, heart rate and other vital functions. It diffuses through the pons in a way that makes surgery impossible. Approximately 300 children are diagnosed with DIPG each year in the US, usually between the ages of 5 and 9. The median survival is 7-11 months after diagnosis with no effective treatments available beyond radiation. The current standard of care with fractionated radiotherapy and supportive care show significant side effects and limited efficacy. SONALA-001 is SonALAsense’s proprietary intravenous aminolevulinic acid HCl (ALA) formulation. SonALAsense’s SDT uses the energy of ultrasound to produce light within the brain to activate protoporphyrin IX, a metabolite of ALA. SONALA-001 SDT is currently being evaluated for safety and preliminary efficacy in clinical study SDT-201, an open label drug and energy dose ranging and expansion study (NCT 05123534). Preliminary data generated from the dose ranging cohort will be presented at a SonALAsense-hosted symposium at the 2023 Society for Neuro-Oncology (SNO) Annual Meeting in Vancouver, Canada, November 16-19. “We are excited for the opportunity to present promising results generated from our DIPG study to date,” said Mark de Souza, SonALAsense CEO. We are fully committed in our efforts to provide hope to this group of patients of the utmost medical need, and to bring this novel treatment to patients as quickly as possible.” About SonALAsense SonALAsense is a clinical-stage company, developing a proprietary form of sonodynamic therapy (SDT) as a first-in-class, noninvasive drug-device combination to treat recurrent glioblastoma, diffuse intrinsic pontine glioma, and other life-threatening cancers. SDT uses SONALA-001 in combination with MR-guided focused ultrasound to selectively target and kill tumor cells. SONALA-001 has received Orphan Drug Designation for Malignant Gliomas. SONALA-001 in combination with the INSIGHTEC Exablate 4000 Type-2 ultrasound device has received Fast Track Designation for the treatment of patients with DIPG. Follow SonALAsense on Twitter and LinkedIn and visit our website at sonalasense.com.

Fast TrackOrphan Drug

09 May 2023

·www.businesswire.com

SonALAsense Welcomes Dr. Ely Benaim as Chief Medical Officer and Executive Vice President of Development

OAKLAND, Calif.--( BUSINESS WIRE )--SonALAsense, a pioneer in the development of non-invasive Sonodynamic Therapy (SDT) using SONALA-001 in combination with Insightec’s MR-guided focused ultrasound (MRgFUS), has appointed Ely Benaim, M.D., as Chief Medical Officer and Executive Vice President of Development. As a new member of the SonALAsense team, Dr. Benaim will play a crucial role in gaining FDA approval and commercializing SDT to treat aggressive brain tumors, such as diffuse intrinsic pontine glioma (DIPG) and recurrent glioblastoma multiforme (rGBM); both of which have ongoing trials. “ Dr. Benaim brings decades of clinical and industry experience and incredible insights to SonALAsense,” said Mark de Souza, PhD, Chief Executive Officer at SonALAsense. “ With his extensive history in oncology practice, bench research, drug and device development, and biopharmaceutical management, he will help us advance sonodynamic therapy through clinical trials and ultimately regulatory approval.” Dr. Benaim began his career as a pediatric oncologist at St. Jude Children’s Research Hospital. Later, he moved into drug development, taking high-profile positions at Amgen, Sangamo BioSciences, Millennium Pharmaceuticals, Rexahn Pharmaceuticals and others. Prior to joining SonALAsense, he was Chief Medical Officer for Novocure, where he managed several pivotal clinical trials. “ I’ve always been attracted to challenging conditions, such as orphan diseases, and driven to accelerate approvals because there were no therapies,” said Dr. Benaim. “ When people say something cannot be done, that’s when I really start getting interested. Both DIPG and glioblastoma are incredibly challenging conditions, and I wanted to be part of the solution.” SonALAsense’s SDT uses SONALA-001, a proprietary formulation of aminolevulinic acid (ALA), to disrupt heme metabolism in tumor cells, increasing production of protoporphyrin, a heme precursor. From there, energy from focused ultrasound excites protoporphyrin molecules, which produce reactive oxygen species that destroy cancer cells. Recently, Dr. Hasan Syed at Children’s National Hospital published a peer-reviewed paper on the first DIPG patient treated with SONALA-001 in the Journal of Neuro-Oncology; a major milestone for this deadly and understudied childhood disease. “ There’s early evidence of activity in patients,” said Dr. Benaim. “ We need to get more patients into clinical trials, gather more data and move as quickly, and as safely, as possible through the regulatory process.” At SonALAsense, Dr. Benaim will work to accelerate this process to get SDT rapidly to patients. He will lead clinical operations, data management, and safety, and play a major role in regulatory filings. He will also be an ambassador for the company, sharing information about this novel therapy with the oncology community. “ We are pleased and honored to have Dr. Benaim join our team,” said company founder and Chief Scientific Officer, Stuart Marcus, M.D., Ph.D. “ He will give us tremendous energy as we advance sonodynamic therapy through clinical trials.” About SonALAsense SonALAsense is a clinical-stage company, developing sonodynamic therapy (SDT) as a first-in-class, noninvasive drug-device combination to treat recurrent glioblastoma, diffuse intrinsic pontine glioma, and other life-threatening cancers. SDT uses SONALA-001 (a drug) in combination with MR-guided focused ultrasound (a device) to selectively target and kill tumor cells. Follow SonALAsense on Twitter and LinkedIn and visit our website at sonalasense.com

Executive Change

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Indication	Highest Phase	Country/Location	Organization	Date
Diffuse Intrinsic Pontine Glioma	Phase 1	US	SonALAsense, Inc.Startup	12 Aug 2022
Diffuse Midline Glioma	Phase 1	US	SonALAsense, Inc.Startup	12 Aug 2022
High grade glioma	Phase 1	US	Insightec Ltd.	15 Mar 2021
High grade glioma	Phase 1	US	SonALAsense, Inc.Startup	15 Mar 2021
Recurrent Malignant Glioma	Phase 1	US	SonALAsense, Inc.Startup	15 Mar 2021
Recurrent Malignant Glioma	Phase 1	US	Insightec Ltd.	15 Mar 2021
Glioblastoma Multiforme	Preclinical	US	SonALAsense, Inc.Startup	06 Feb 2023
Recurrent Glioblastoma	Preclinical	US	SonALAsense, Inc.Startup	06 Feb 2023

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