The goal of this study is to learn if the intervention using a fluorescent agent 5-Aminolevulinic acid (5-ALA) to aid in the surgical approach to visualize the soft-tissue sarcoma (STS) during surgical resection. 5-ALA goes through the blood stream and into the tumor tissue allowing it to light up when the surgeon uses a special light in the operating room. The technique is called 5-ALA fluorescence-guided surgery (FGS). The main question aims to answer if 5-ALA provide intraoperative fluorescent visualization of soft-tissue sarcoma versus surrounding tissue and demonstrate the efficacy of tumor and surgical margin resections by a gross and histological analysis of fluorescing and non-fluorescing samples immediately after removal. Participants will be asked to orally administer 5-ALA three to four hours prior to surgery in preoperative area.

开始日期2025-12-01

申办/合作机构

University of Colorado Denver

[+1]

NCT06907485

/ Not yet recruiting临床2期IIT

A Multicenter Study to Assess the Feasibility of Gleolan (ALA / Aminolevulinic Acid HCl) in Pediatric Brain Tumor Patients After Delayed Administration

This clinical trial focuses on pediatric patients aged 2 up to 18 years of age with a new or recurrent pediatric brain tumor, suspected to be either a high-grade or low-grade glioma, and scheduled for surgical removal. 5-aminolevulinic acid (5-ALA) is FDA-approved for improving brain tumor visualization in adults during surgery through fluorescence, enabling more complete removal of the tumor. This study aims to evaluate the feasibility of administering 5-ALA to pediatric brain tumor patients and to assess the quality of tumor fluorescence during surgery in this patient population.
For the clinical trial, the patient will orally ingest 5-ALA 6 to 12 hours before brain surgery. All study participants will be provided standard medical care for removal of the brain tumor. All children enrolled in the study will be closely monitored prior to, during, and after surgery to ensure there are no reactions to the study drug. 5-ALA can make the patient more sensitive to sunlight and direct indoor lighting, referred to as photosensitivity, and can cause a sunburn-type reaction. It is for this reason that patients will be kept in subdued light conditions for 48 hours following surgery. Study participation starts once the patient is enrolled in the study until 6-month post-surgery.

开始日期2025-12-01

申办/合作机构

University of Pittsburgh

[+4]

NCT07144345

/ Not yet recruiting临床3期

Randomized, Multi-Center, Evaluator-Blind, Vehicle-Controlled Study to Evaluate Efficacy and Safety of Reformulated Levulan Kerastick Plus Photodynamic Therapy (PDT) for Field-Directed Treatment in Patients With Actinic Keratosis (AK) of Face and (Bald) Scalp

This Phase 3, randomized, multi-center, evaluator-blind, vehicle-controlled study evaluates the efficacy and safety of reformulated Levulan Kerastick (aminolevulinic acid HCl 20%) combined with photodynamic therapy (PDT) for field-directed treatment of actinic keratosis (AK) on the face or bald scalp. Approximately 160 adult patients with 4-8 mild to moderate AK lesions will be randomized into four treatment arms based on two variables: type of treatment (active drug or vehicle) and incubation time. During the study, up to 2 PDT sessions may be administered depending on the clearance of lesions. The primary endpoint is complete clearance rate (CCR) at Week 12. Secondary endpoints include AK clearance rate (AKCR), partial clearance, change in total lesion count and area, recurrence rate, cosmetic response, and patient satisfaction. Safety assessments include adverse events, local skin reactions, vital signs, and lab tests.

开始日期2025-09-01

申办/合作机构

Sun Pharmaceutical Industries Ltd.

100 项与盐酸氨酮戊酸相关的临床结果

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100 项与盐酸氨酮戊酸相关的转化医学

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100 项与盐酸氨酮戊酸相关的专利（医药）

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8,232

项与盐酸氨酮戊酸相关的文献（医药）

2025-12-31·JOURNAL OF DERMATOLOGICAL TREATMENT

Efficacy analysis of 5-aminolevulinic acid photodynamic therapy for vulvar lichen sclerosus in women of childbearing age

Article

作者: Zhu, Dongning ; Shi, Chunyu ; Jia, Yu ; Dang, Yanling ; Shang, Qian ; Yang, Lijuan

OBJECTIVE:

To investigate the efficacy and long-term durability of 5-aminolevulinic acid photodynamic therapy (ALA-PDT) in treating vulvar lichen sclerosus (VLS) in women of childbearing age.

METHODS:

Fifty-five patients with VLS who had failed long-term repeated pharmacological treatments (mean disease duration: 3.6 ± 1.4 years)were enrolled. All participants underwent four sessions of ALA-PDT. Follow-up evaluations were conducted for six months after treatment. Outcomes were assessed using the Cattaneo clinical symptom and sign score and the Dermatology Life Quality Index (DLQI) questionnaire at baseline, after four treatments, and at 3-6 months post-treatment.

RESULTS:

At the 6-month follow-up, efficacy rates were 81.8% for pruritus relief, 67.3% for skin elasticity restoration, 63.6% for skin color improvement, and 72.7% for lesion area reduction. Pain was the only reported adverse reaction during treatment.

CONCLUSION:

20% ALA-PDT is a safe, effective, and durable therapeutic option for VLS with minimal side effects. Notably, it provides a viable alternative for patients unresponsive to conventional therapies.

2025-12-01·CHILDS NERVOUS SYSTEM

5-Aminolevulinic acid (5-ALA) in paediatric brain tumour surgery—a systematic review and exploration of fluorophore alternatives

Review

作者: Kanodia, Charvi ; Kaliaperumal, Chandrasekaran ; Yahya, Qalisya Binti ; Liistro, Marianna ; Collins, Victoria G

PURPOSE:

Paediatric brain tumours represent the most common solid malignancies in children, with extent of resection being a critical prognostic factor. Fluorescence-guided surgery using 5-aminolevulinic acid (5-ALA) is well-established for adult high-grade gliomas, but its efficacy and safety in paediatric populations remain unclear. This systematic review evaluates the utility of 5-ALA fluorescence-guided surgery in paediatric brain tumours and explores alternative fluorophores.

METHODS:

A systematic review was conducted according to PRISMA guidelines, analysing studies from MEDLINE and EMBASE published up to October 2024. Data on patient demographics, tumour fluorescence patterns, surgical outcomes, and adverse effects were extracted. Statistical analyses assessed fluorescence differences across tumour types and administration parameters.

RESULTS:

Twenty-three studies, including 281 paediatric patients (mean age, 10 years), were analysed. The most common tumours included pilocytic astrocytomas (n = 45), medulloblastomas (n = 45), glioblastomas (n = 35), and ependymomas (n = 27). Strong fluorescence was observed more frequently in high-grade gliomas compared to low-grade gliomas (p < 0.00001), non-glioma tumours (p < 0.00001), and high-grade non-glioma tumours (p = 0.000485). Adverse effects were mostly transient; rare complications included transaminitis and dermatologic reactions.

CONCLUSION:

5-ALA fluorescence-guided surgery shows promise in the resection of high-grade gliomas in paediatric patients, improving intraoperative visualisation. However, limited fluorescence in low-grade and non-glioma tumours underscores the need for tumour-specific approaches. Emerging alternatives, such as fluorescein sodium and tozuleristide, offer potential advantages. Future research should focus on optimising 5-ALA dosing, refining timing protocols, and conducting robust prospective trials to establish efficacy and safety in paediatric populations.

2025-11-01·BIORESOURCE TECHNOLOGY

Modeling and optimization of bioproduct formation with purple phototrophic bacteria using machine learning

Article

作者: Meyer, Torsten ; Buitrón, Germán ; Montiel-Corona, Virginia ; Edwards, Elizabeth A

Municipal and industrial wastewater, along with organic waste, can be transformed into valuable bioproducts using purple phototrophic bacteria. This study compares the performance of three machine learning models (Random Forest, XGBoost, and CatBoost) in predicting and optimizing the formation of key bioproducts: polyhydroxybutyrate, polyhydroxyvalerate, 5-aminolevulinic acid, coenzyme Q10, carotenoids, bacteriochlorophylls, and biomass. The models were trained on a dataset compiled from previous studies, using input variables such as reaction time, concentration of organic matter, ethanol, bicarbonate, levulinic acid, ferric citrate, mineral medium, and N, C/N ratio, illumination conditions (continuous or intermittent), operation mode (batch or semicontinuous), and volume exchange percentage. Bayesian optimization was applied to train and tune the models. Performance was assessed using R², Pearson correlation, RMSE, and MAPE. CatBoost outperformed the others, showing higher predictive correlation and lower error. It was subsequently used for further optimization. Feature importance analysis identified reaction time, mineral medium concentration, and volume exchange percentage as key drivers of bioproduct synthesis. The Particle Swarm Optimization algorithm was applied to determine optimal conditions for each target compound. Under the conditions studied, predicted maximum yields were: 569 mg polyhydroxybutyrate/L, 45 mg polyhydroxyvalerate/L, 79 µmol 5-aminolevulinic acid/L, 13 mg coenzyme Q10/g dw, 7 mg carotenoids/g dw, 17 mg bacteriochlorophylls/g dw, and 2040 mg biomass/L. Optimization suggests that operating as a sequencing batch reactor and employing discontinuous illumination for most targets, along with a reduced mineral medium concentration, is beneficial. Results highlight that each bioproduct requires distinct operational settings, supporting the idea of clustering target compounds.

127

项与盐酸氨酮戊酸相关的新闻（医药）

2025-08-25

·GlobeNewswire-Health Care

Biofrontera Inc. Announces Last-Patient-Out in Phase 2b Study of Ameluz® (aminolevulinic acid HCI) Topical Gel, 10% for the Treatment of Moderate to Severe Acne Vulgaris

Final patient visit marks key milestone for Biofrontera, with top-line results expected Q1 2026Company plans FDA discussion in Q3 2026 to advance Phase 3 program and potential label expansionMore than 50 million people affected by acne in US each year, with market valued at $5.7 billion in 2024 WOBURN, Mass., Aug. 25, 2025 (GLOBE NEWSWIRE) -- Biofrontera Inc. (Nasdaq: BFRI) (“Biofrontera” or the “Company”), a biopharmaceutical company specializing in the development and commercialization of photodynamic therapy (PDT), today announced that the final patient in its Phase 2b clinical trial evaluating Ameluz® (aminolevulinic acid hydrochloride) for the treatment of moderate to severe acne vulgaris (AV) completed participation on August 22, 2025. Acne vulgaris is a common skin condition characterized by inflammatory and non-inflammatory lesions that frequently lead to permanent scarring. Beyond the physical burden, AV often has a significant psychological impact, including reduced self-esteem and depression. Current U.S. guidelines recommend a range of treatment options for AV, including topical agents, systemic antibiotics, hormonal therapies, isotretinoin and physical treatments. However, many of these options carry limitations such as serious side effects, teratogenic risks, the need for continuous daily dosing, or—particularly with antibiotics—growing concerns about resistance. As a result, there remains a critical unmet need for alternative treatment options. Biofrontera’s Phase 2b trial is a multicenter, randomized, double-blind study comparing Ameluz® with vehicle gel for the treatment of moderate to severe acne vulgaris using red-light Photodynamic Therapy (PDT). Following application of one tube of Ameluz® or vehicle gel to the entire face, participants were incubated for either 1 or 3 hours before illumination with the BF-RhodoLED® lamp. Up to 3 PDT sessions were performed at monthly intervals and participants were followed up for an additional 2 months after receiving the last PDT. “We are thrilled to achieve this important milestone in our clinical program”, said Dr Hermann Luebbert, CEO and Chairman of Biofrontera Inc. “It brings us a significant step closer to potentially offering a new, effective and much needed treatment option for patients with moderate to severe acne vulgaris. This trial represents meaningful progress in expanding the indications for Ameluz® and reinforces our commitment to advancing PDT.” Dr. Mitchel P. Goldman, MD, FAAD, the coordinating investigator of the study and Medical Director of Cosmetic Laser Dermatology and Platinum Dermatology Partners, expressed optimism about its potential impact "Ameluz® PDT has demonstrated meaningful benefits in other dermatologic conditions, and we believe it will become an important option for patients with moderate to severe acne vulgaris. Many of these patients continue to rely on regimens that carry significant treatment burdens including cost, adverse effects and the necessity for prolonged treatments. Expanding the use of Ameluz® to treat acne would be a valuable advancement for both physicians and patients. We look forward to the study results with great anticipation.” Biofrontera expects to receive top-line data in Q1 2026. Pending positive results, the Company intends to present the findings to the U.S. Food Drug Administration (FDA) in early Q3 2026 as the basis for a future Phase III program, with the goal of achieving approval for Ameluz® PDT for the treatment of acne vulgaris. About Acne Vulgaris Acne vulgaris is the most common skin condition in the United States, affecting an estimated 50 million people each year¹. Although often associated with adolescence, it persists into adulthood, impacting approximately 40% of adults². The condition ranges from mild blackheads and whiteheads to severe inflammatory forms such as nodules and cysts, which can cause permanent scarring and have profound effects on mental health and self-esteem³. Current U.S. guidelines recommend topical therapies, oral antibiotics, and isotretinoin for moderate to severe acne⁴. However, these treatments are frequently limited by significant systemic side effects, driving demand for safer and more effective alternatives. The U.S. acne treatment market, valued at $5.7 billion in 2024 and projected to grow at a 5.3% CAGR, reflects this unmet need⁵. More than 55% of spending is currently directed to oral antibiotics and isotretinoin⁶, underscoring the reliance on systemic therapies and the opportunity for novel treatment approaches. About Biofrontera Inc. Biofrontera Inc. is a U.S.-based biopharmaceutical company specializing in the development and treatment of dermatological conditions with a focus on PDT. The Company commercializes the drug-device combination Ameluz® with the RhodoLED® lamp series for PDT of AK, pre-cancerous skin lesions which may progress to invasive skin cancers. The Company performs clinical trials to extend the use of the products to treat non-melanoma skin cancers and moderate to severe acne. For more information, visit www.biofrontera-us.com and follow Biofrontera on LinkedIn and X . Forward-Looking Statements Certain statements in this press release may constitute “forward-looking statements” within the meaning of the United States Private Securities Litigation Reform Act of 1995, as amended. These statements include, but are not limited to, statements relating to Biofrontera's commercial opportunities and the commercial success of its licensed products. We have based these forward-looking statements on our current expectations and projections about future events. Nevertheless, actual results or events could differ materially from the plans, intentions and expectations disclosed in, or implied by, the forward-looking statements we make. These risks and uncertainties, many of which are beyond our control, include, but are not limited to: the uncertainties inherent in the initiation and conduct of clinical trials; availability and timing of data from clinical trials; whether results of earlier clinical trials or trials of Ameluz® in combination with BF-RhodoLED and/or RhodoLED XL in different disease indications or product applications will be indicative of the results of ongoing or future trials; uncertainties associated with regulatory review of clinical trials and applications for marketing approvals; the impact of any extraordinary external events; any changes in the Company’s relationship with its licensors; the ability of the Company’s licensors to fulfill their obligations to the Company in a timely manner; the Company’s ability to achieve and sustain profitability; whether the current global disruptions in supply chains will impact the Company’s ability to obtain and distribute its licensed products; changes in the practices of healthcare providers, including any changes to the coverage, reimbursement and pricing for procedures using the Company’s licensed products; whether the market opportunity for Ameluz® in combination with BF- RhodoLED and/or RhodoLED XL is consistent with the Company’s expectations; the Company’s ability to retain and hire key personnel; the sufficiency of cash resources and need for additional financing; and other factors that may be disclosed in the Company’s filings with the Securities and Exchange Commission (the “SEC”), which can be obtained on the SEC’s website at www.sec.gov. Readers are cautioned not to place undue reliance on the forward-looking statements, which speak only as of the date on which they are made and reflect management’s current estimates, projections, expectations and beliefs. The Company does not plan to update any such forward-looking statements and expressly disclaims any duty to update the information contained in this press release except as required by law. Contacts: Investor RelationsAndrew Barwicki1-516-662-9461ir@bfri.com References: American Academy of Dermatology Association. Acne: Overview. https://www.aad.org/public/diseases/acneCollier CN et al. “The prevalence of acne in adults 20 years and older.” J Am Acad Dermatol. 2008;58(1):56–59.Tan JK, Bhate K. “A global perspective on the epidemiology of acne.” Br J Dermatol. 2015;172 Suppl 1:3–12.Zaenglein AL et al. “Guidelines of care for the management of acne vulgaris.” J Am Acad Dermatol. 2016;74(5):945–973.e33.Fortune Business Insights. Acne Treatment Market Size, Share & Trends Report, 2024–2032 . https://www.fortunebusinessinsights.com/acne-treatment-market-102596IQVIA Institute Report, U.S. Dermatology Market Analysis, 2023

临床2期临床3期

2025-08-04

·BioSpace

Glioblastoma Funding Rises as Nonprofits Drive Interest and Pharma Returns to the Table

iStock/ bawanch After decades of limited progress—owing to the difficulty of treating the disease and resultant market risk—glioblastoma research is entering a new phase spurred by smarter trials, targeted funding and renewed interest from companies like Merck and Jazz Pharmaceuticals. Glioblastoma, a deadly brain cancer, is notoriously difficult to treat. But recent acquisitions by Merck and Jazz Pharmaceuticals and the development of innovative adaptive trials signal a renewed push to develop effective treatments for this aggressive malignancy. Representing nearly half of all primary malignant brain tumors, the median survival rate for patients with glioblastoma (GBM) is just 14.6 months, according to The Glioblastoma Research Organization . Yet there have been few therapeutic breakthroughs over the last 20 years. Only two drugs have emerged in this time, Temodar (temozolomide), developed by Merck, and Avastin (bevacizumab), marketed by Roche. This lack of success in uncovering new treatments is not due to a lack of effort; there were over 1,500 registered glioma trials between 2006 and 2021. However, high-pro failures led to a decline in interest from the pharmaceutical industry in developing new GBM treatments. “When Big Pharma steps out, most institutional investors step out too, because there’s no exit for them,” Yash Thukral, co-executive director of Innovate GBM, told BioSpace . “If institutional money is not being invested, no retail money is going to be put in. So what ends up happening is companies working in the space get less and less capital over time and become forgotten.” This leads to a cycle where fewer investments mean companies lack the capital necessary to progress assets, which reduces clinical trial success in the area and therefore industry investment, Thukral explained. This is beginning to change as deals and investment flow back into the space. Last October , Merck acquired Modifi Biosciences, which is developing DNA modification–enabled cancer therapeutics, including for GBM, in a deal valued at up to $1.3 billion. Then in March, Jazz bought out Chimerix for $925 million to gain access to the biotech’s diffuse glioma candidate. Jazz is anticipating an FDA decision for the drug, dordaviprone, later this month. But in the meantime, adversity has led to creativity. During the lean times, without access to this slowly emerging capital, GBM researchers have pivoted, and the space has seen growth in alternative methods to support early-stage research for new GBM treatments. A Vicious Cycle All told, pharma sinks 45% of its overall R&D spend into oncology. With no certainty of success in clinical trials, the area’s relatively high revenues and low development costs manage to offset the risk of developing assets for cancer. GBM, however, poses serious challenges to treatment, and, therefore higher risk. “Because of the location of the tumors and the biology of the brain—obviously, there are all these different cell types—and the way these tumors grow very quickly and kind of put out feelers into the rest of the healthy brain, it is very difficult to treat,” Marianne Baker, science engagement manager at Cancer Research UK (CRUK), told BioSpace . “The challenge is that the tumors are very difficult to see, and very hard to take out accurately and fully.” This places GBM into a pool of “hard-to-treat cancers,” Baker noted, which also includes certain brain, lung, pancreatic, liver and stomach cancers. Developing a treatment for GBM is then both appealing to the pharma industry—because creating a new standard of therapy could be lucrative—but prohibitive due to its difficult-to-treat nature, small patient population (around 3 people per 100,000 in the U.S .), and high level of clinical failure. Creating an Ecosystem According to Thukral, Innovate GBM was set up to allow smaller companies better access to those venture capital firms or institutions that could provide funding and, in return, de-risk the investment into these assets. To this end, it has developed a network of key opinion leaders, service providers, patient advocacy and outreach groups, FDA officials, universities, drug developers and investors, he explained. This allows those interested in potentially investing in assets to speak directly with all parties on the likelihood of a candidate’s success, essentially a streamlined due diligence. Before Innovate GBM, these stakeholders were not well-connected, which slowed down the exchange of ideas and the opportunity for candidates to receive the required funding, according to Thukral. CRUK has also recognized the need to support GBM with greater levels of funding in recent years, Baker said, adding that the research charity has shifted strategy to “carve out a dedicated space and funding to support areas where there’s a clear gap.” To progress work in the area, CRUK set up a network in the U.K. called the Brain Tumor Centres of Excellence in 2018, which supports experts in the field to carry out research, drug development and clinical trials. Last year , this initiative provided funding for a world-first adaptive clinical trial to test new treatments for people living with brain cancer by having each patient’s genome sequenced, and the drug combination tailored to the specific genetics of their cancer. The organization has had previous success in the area. CRUK’s researchers were the first to discover a precursor molecule that later became temozolomide. The Seeds of Success Though progress toward the development of new drugs has been slow over the last two decades, there are signs that the broader efforts to develop new candidates and harness new technologies could yield better treatment options in the future. Since its inception in 2023, Innovation GBM has helped raise over $170 million in public investments across nine brain tumor companies. Outside of these companies, Alpheus Medical has also had success in raising funds, securing $52 million in a series B round in May to evaluate its sonodynamic therapy (SDT) in patients with newly diagnosed GBM. In emailed comments to BioSpace , Vijay Agarwal, CEO of Alpheus, said the company’s therapy works by combining tumor-targeting, FDA-approved drugs with low-intensity diffuse ultrasound. When a drug accumulates in tumor cells and is exposed to ultrasound, the interaction produces reactive oxygen species that selectively kill cancer cells, enabling the destruction of tumor cells beyond the visible tumor mass. Those cells are one of the biggest drivers of recurrence, Agarwal said. “The funding environment has been tough in general, and glioblastoma carries extra risk because very few therapies have improved patient survival outcomes,” Agarwal explained. “However, Glioblastoma is high-risk, but also high-impact. If you have a therapy that can truly change outcomes, investors will pay attention, even in a tight market.” Utilizing light instead of sound, CRUK found a way to highlight tumor cells to make surgery more effective. The organization developed the “pink drink,” a molecule called 5-ALA that accumulates in tumor cells, Baker explained. During surgery, it is possible to shine a UV light on the brain that causes the tumor cells to glow bright pink, allowing surgeons to see exactly where the tumor cells are and to remove as many as possible and lower the risk of recurrence. Attracting Interest Thukral also noted that the development of GBM Agile, an adaptive clinical trial platform that allows researchers to test multiple GBM treatments using real-time data, marked an inflection point for the therapeutic space. The platform allows the testing of treatments against historical control groups instead of live control arms, which enables drug developers to cut the cost of trials from approximately $75 million to $25 million, he said. Since its inception in 2018, this has allowed eight companies, including Kazia Therapeutics , Vigeo Therapeutics and Biohaven , to test therapies in later-stage trials. “You don’t expect change to happen this fast, but I think we’re at an inflection point. Over six years, that’s eight different therapies reaching pivotal trial stages. Even if they don’t finish, we’re getting more shots on goal,” Thukral said, noting that this had piqued big pharma and investor interest. “The markets are realizing that there is real reward potential here.”

并购上市批准临床研究

2025-06-14

·动脉网

FDA授予孤儿药称号，这款声动力疗法正在攻克脑肿瘤难题

近日，Alpheus Medical宣布完成5200万美元B轮融资，由HealthQuest Capital与Samsara BioCapital联合领投，老股东OrbiMed、Action Potential Venture Capital持续跟投，BrightEdge、Brain Tumor Investment Fund以及Sontag Innovation Fund等多家专注癌症护理领域的机构投资者也参与其中。资金将用于推进声动力疗法在脑肿瘤领域的临床试验：启动针对新诊断胶质母细胞瘤（GBM）患者的2B期随机对照试验，验证非热效应低强度扩散式超声（LIDU）联合口服5-氨基乙酰丙酸（5-ALA）的声动力治疗（SDT）平台，为商业化注册做准备。01瞄准胶质母细胞瘤，解决肿瘤浸润性和弥散性难题胶质母细胞瘤（GBM）是成人最常见的原发性恶性脑瘤之一，是一种起源于大脑胶质细胞的四级恶性肿瘤。肿瘤级别反映了其生长和扩散的危险程度，四级即最恶、最严重。GBM发病率约占所有胶质瘤的57%，以其高侵袭性、高复发率和治疗选择受限而著称。全球每年新增患者超过30万例，在美国，每天约有27名美国人因GBM离世，每年估计超1万人死于该病。GBM患者的生存状况不容乐观，中位生存期仅为12至18个月，五年生存率仅6.8%。尽管GBM自20世纪20年代在科学文献中被首次确认以来，已过去约100年，但目前仅有4种FDA批准药物和1种设备可用于治疗GBM，治疗手段有限。胶质母细胞瘤数据概况来自：Alpheus Medical官网目前传统治疗路径仍然以“手术切除+术后放疗和化疗（Stupp方案）”为主，但由于GBM具有显著的浸润性和弥散性，GBM 肿瘤中还包含各种各样的细胞、形成了极其复杂的肿瘤微环境（包括免疫微环境、代谢微环境等），手术无法实现彻底切除，而放化疗的毒副作用又限制了疗效的持续性和患者的耐受性。传统治疗方法一览图此外，尽管肿瘤电场治疗等辅助性治疗（TTF）已获FDA批准进入临床路径，但存在强依赖患者依从性、费用较高等问题，难以广泛推广。而病毒疗法、CAR-T和免疫检查点抑制剂等新路径，虽具理论潜力，但临床结果尚不成熟，存在技术复杂、成本高或治疗区域局限等问题，因此市场急需更高效、更安全、更便捷的治疗方案。声动力疗法（Sonodynamic Therapy，简称SDT）作为近20年研发的新型肿瘤治疗技术，通过超声波的非侵入性、超强组织渗透能力激发声敏剂产生单线态氧，以破坏线粒体结构诱发细胞的坏死和凋亡的双重抗癌作用，实现精准杀伤肿瘤而不损伤正常细胞的治疗目标。02SDT平台：结合药物靶向与超声波激活，精准打击肿瘤细胞基于这一原理，Alpheus的SDT平台以非侵入性药物设备组合为胶质母细胞瘤（GBM）提供了新的治疗路径。其核心原理是结合药物靶向与超声波激活，实现对肿瘤细胞的精准打击与免疫激活。具体来说，在治疗时，患者先口服已获FDA批准的5-氨基乙酰丙酸（5 - ALA）药物，用于荧光引导下的脑瘤手术切除。该药物在体内会被肿瘤细胞特异性地摄取和积累，而几乎不进入健康细胞，如同给肿瘤细胞贴上识别标签，使其在众多正常细胞中暴露出来，为后续治疗定位基础。接着，Alpheus专有的非侵入性超声波系统（LIDU，CV-01）可向大脑肿瘤区域发射低强度、扩散式超声波。这些超声波可穿透头皮和颅骨，精准作用于已被标记的肿瘤细胞，触发药物释放高活性氧自由基，为激活药物并产生治疗效果创造条件。氧自由基具有强氧化性，会在肿瘤细胞内部引发连锁反应，破坏肿瘤细胞的细胞膜、DNA等关键结构，让肿瘤细胞损伤甚至死亡。同时，死亡肿瘤细胞释放热休克蛋白等信号分子，激活人体免疫系统。最后，激活的免疫系统（如树突状细胞、T细胞等）会识别并持续攻击残余肿瘤细胞，引发肿瘤特异性的免疫反应，形成“直接杀伤+免疫监视”的双重治疗效应，防止肿瘤复发。SDT平台治疗路径来自：Alpheus Medical官网从治疗效果来看，Alpheus的SDT平台有以下核心优势：● 非侵入性操作，患者安全性更高：整个治疗过程无需开颅手术等侵入性操作或镇静剂，避免感染、出血、神经损伤等手术风险。患者治疗时保持清醒，可在门诊环境下进行，支持重复治疗，相比传统手术更适合身体虚弱或不耐受创治疗的患者。● 精准靶向性，减少对周围组织损伤：由于5-ALA仅在肿瘤细胞中特异性积累，超声波又能精准地作用于肿瘤区域，使氧自由基释放局限于病灶，因此SDT平台可以对肿瘤细胞进行精准打击，对周围健康组织损伤极小。● 显著延长生存期：Alpheus公布的1/2期临床试验数据显示，使用SDT疗法的患者中位总生存期从通常的6-8个月提高到约15.7个月，无进展生存期从典型的1.8个月增加到5.5个月，展现出了良好的治疗效果和潜力。● 全脑覆盖，突破传统局部治疗的局限：GBM具有高度侵袭性，常向全脑弥散生长，传统的局部治疗手段往往难以有效控制肿瘤的复发和进展。SDT的扩散式超声波可覆盖整个病变半球，对肉眼不可见的微小病灶和浸润肿瘤细胞同步起效，突破传统局部治疗的局限。03FDA授予孤儿药认证，临床试验效果显著2022年7月，Alpheus的SDT平台获FDA授予孤儿药认证和快速通道资格，用于复发性GBM治疗。孤儿药是指用于预防、治疗、诊断罕见病的药品，因罕见病患者人数少，研发成本高，各国通常给予政策支持。因此，这一认证不仅是对其技术创新性的官方认可，更意味着可享受税收优惠、研发资助及优先审评等政策，显著缩短产品上市周期，为突破GBM治疗困境提供官方背书。2024年11月，Alpheus在2024年神经肿瘤学会（SNO）年会上公布的1/2期临床试验数据引发行业关注。这项开放标签、多中心、剂量递增研究纳入12名复发性或难治性高级别胶质瘤患者，分为每月60分钟、90分钟、120分钟三个治疗组，结果显示：生存数据显著提升。中位总生存期（OS）达15.7个月，较传统治疗的6-8个月延长近2倍；中位无进展生存期（PFS）5.5个月，是历史数据1.8个月的3倍，且6个月生存率达89%。安全性优势突出。治疗过程未出现健康组织损伤，无治疗相关死亡、严重不良事件（SAEs）或剂量限制性毒性（DuLTs），非侵入性特征（无需镇静、不开颅）大幅降低治疗风险。2025年2月《神经肿瘤学杂志》发表的另一项GBM研究进一步验证了SDT平台的精准疗效：3名未手术的新诊断GBM患者接受单剂量SDT治疗后，肿瘤细胞大面积坏死，且周围神经组织完好，治疗无需镇静即可在门诊完成。同时，这两项研究共同为2025年启动的多中心随机对照2B期试验奠定了关键基础。SDT平台的研发价值也促使资本不断入局。从A轮融资的1400万美元到此次B轮融资的5200万美元，Alpheus估值显著提升，正在成为脑肿瘤创新疗法领域的明星企业。资本的持续注入不仅印证了SDT技术在GBM治疗中的突破性，更预示其即将从临床阶段向商业化阶段跨越。04临床+技术+科研团队，抢占脑肿瘤治疗创新先机从团队成员的背景来看，Alpheus的核心团队在脑肿瘤治疗领域具备深厚的专业知识和丰富的经验。创始人Vijay Agarwal在脑肿瘤外科、医疗设备以及生物技术行业的长期经历，为公司的发展提供了战略领导和医学专业支持。首席技术官Jeremy Ling在技术研发和商业化方面的能力，有助于推动公司声动力疗法技术的不断进步和产品的商业化进程。临床运营副总裁Jennifer Drescher在临床运营方面的丰富经验，能确保公司的临床试验高效、规范地进行。研发副总裁Nick Ellens在治疗性超声领域的研究背景，则为公司核心技术的研发提供了专业的技术支持。第一排：Vijay Agarwal（左），Jeremy Ling（右）第二排：Jennifer Drescher（左），Nick Ellens（右）来自：Alpheus Medical官网早在2022年，Alpheus就已宣布成立世界级科学顾问委员会 (SAB)，并任命来自西北大学、哈佛医学院与达纳法伯癌症研究所等顶级院校、医疗机构的神经肿瘤学、神经外科等领域的顶尖专家为委员会成员。这一委员会的临床指导与2022年11月完成的1600万美元A轮融资形成合力，不仅推动了首次人体（FIH）试验的启动，还将助力探索其他实体瘤的概念验证研究。根据贝哲斯咨询数据显示，2024年全球脑肿瘤治疗市场规模达31.5亿美元，预计到2032年将增至62.2亿美元。在脑癌治疗需求持续增长而创新疗法稀缺的背景下，Alpheus以临床专家、技术精英与科研顾问构成的复合型团队为支撑，正凭借声动力疗法的技术突破，在潜力巨大的市场中抢占脑肿瘤治疗创新的先机。如果您想对接文章中提到的项目，或您的项目想被动脉网报道，或者发布融资新闻，请与我们联系；也可加入动脉网行业社群，结交更多志同道合的好友。近期推荐声明：动脉网所刊载内容之知识产权为动脉网及相关权利人专属所有或持有。未经许可，禁止进行转载、摘编、复制及建立镜像等任何使用。文中如果涉及企业信息和数据，均由受访者向分析师提供并确认。动脉网，未来医疗服务平台

孤儿药细胞疗法免疫疗法临床研究

100 项与盐酸氨酮戊酸相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D02908	盐酸氨酮戊酸	L01XD04	DB00855

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
浸润性膀胱癌	日本	SBI Pharmaceuticals Co., Ltd.	2017-09-27
多形性胶质母细胞瘤	澳大利亚	Specialised Therapeutics Glio Pty Ltd	2013-11-07
造影剂	日本	Nobelpharma Co., Ltd.	2013-03-25
基底细胞癌	欧盟	Biofrontera Bioscience GmbH	2011-12-13
基底细胞癌	冰岛	Biofrontera Bioscience GmbH	2011-12-13
基底细胞癌	列支敦士登	Biofrontera Bioscience GmbH	2011-12-13
基底细胞癌	挪威	Biofrontera Bioscience GmbH	2011-12-13
胶质瘤	欧盟	Photonamic GmbH & Co. KG	2007-09-07
胶质瘤	冰岛	Photonamic GmbH & Co. KG	2007-09-07
胶质瘤	列支敦士登	Photonamic GmbH & Co. KG	2007-09-07
胶质瘤	挪威	Photonamic GmbH & Co. KG	2007-09-07
尖锐湿疣	中国	上海复旦张江生物医药股份有限公司	2007-02-14
光化性角化病	美国	Sun Pharmaceutical Industries, Inc.	1999-12-03

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
非肌层浸润性膀胱肿瘤	临床3期	中国	上海复旦张江生物医药股份有限公司	2025-03-11
卵巢上皮癌	临床3期	美国	NX Development Corp.	2024-05-30
复发性卵巢癌	临床3期	美国	NX Development Corp.	2024-05-30
腺样囊性癌	临床3期	美国	Sbi Alapharma Canada, Inc.	2021-04-27
乳腺癌	临床3期	美国	Sbi Alapharma Canada, Inc.	2021-04-27
乳腺导管癌	临床3期	美国	Sbi Alapharma Canada, Inc.	2021-04-27
恶性上皮肿瘤	临床3期	美国	Sbi Alapharma Canada, Inc.	2021-04-27
导管癌	临床3期	美国	Sbi Alapharma Canada, Inc.	2021-04-27
乳腺浸润性小叶癌	临床3期	美国	Sbi Alapharma Canada, Inc.	2021-04-27
粘液性乳腺癌	临床3期	美国	Sbi Alapharma Canada, Inc.	2021-04-27

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT05101798 (CTgov)	临床2期	颅底肿瘤 \| 头颈部肿瘤	7	5-Aminolevulinic acid Hydrochloride	膚顧憲觸窪夢獵積選範 = 積襯選範餘醖鬱淵鏇範遞廠膚醖簾窪顧鑰艱艱 (窪糧鬱獵膚鏇糧鹽艱範, 鏇襯蓋淵顧築憲範襯積 ~ 構醖衊膚願膚廠築範簾) 更多	-	2025-07-28
NCT03573401 (ALA-BCC-CT013, CTgov)	临床3期	浅表型基底细胞癌	187	ALA (BF-200 ALA)	網鏇繭製廠餘鹽繭襯簾 = 齋醖顧製鹹範憲繭觸願糧觸觸獵鏇窪醖襯範醖 (蓋顧獵糧網淵積願衊膚, 夢範艱遞糧觸淵顧遞淵 ~ 顧餘齋選襯壓顧餘淵廠) 更多	-	2025-04-29
				Placebo Photodynamic therapy (PDT) (vehicle to BF-200 ALA containing no active ingredient) (Vehicle)	網鏇繭製廠餘鹽繭襯簾 = 蓋膚顧廠襯夢觸鏇襯鹽糧觸觸獵鏇窪醖襯範醖 (蓋顧獵糧網淵積願衊膚, 餘憲範艱夢齋壓窪鹽遞 ~ 繭築壓鬱襯夢鏇繭遞簾) 更多
NCT01128218 (Phase 2 Sensitivity and Selectivity Study, Pubmed \| Oper Neurosurg)	临床2期	复发性胶质母细胞瘤	31	High-dose 5-ALA (>40 mg/kg)	鹹鹹夢糧顧鏇網餘簾觸(顧蓋鹽醖網顧夢憲鬱醖) = 鹽蓋餘積鑰簾壓蓋觸鏇壓範積醖壓餘繭夢餘糧 (淵壓憲糧齋製壓憲網構 ) 更多	积极	2024-11-11
				Low/standard dose 5-ALA (<30 mg/kg)	鹹鹹夢糧顧鏇網餘簾觸(顧蓋鹽醖網顧夢憲鬱醖) = 衊顧衊獵蓋選襯顧廠獵壓範積醖壓餘繭夢餘糧 (淵壓憲糧齋製壓憲網構 ) 更多
TMOD-04 (SNO2024)	临床1期	-	8	Sonodynamic Therapy with 5-ALA HCL Oral Solution and CV-01	鬱夢鹽鏇餘糧築簾積鹽(襯鏇窪顧鑰構簾簾築廠) = 餘鏇積壓鏇襯餘積築糧積鑰範選糧遞蓋廠範範 (鬱築廠夢夢鹹醖糧簾簾 )	积极	2024-11-11
				5-ALA HCL oral solution (Control Group)	鬱夢鹽鏇餘糧築簾積鹽(襯鏇窪顧鑰構簾簾築廠) = 憲製衊鹽醖艱顧艱憲遞積鑰範選糧遞蓋廠範範 (鬱築廠夢夢鹹醖糧簾簾 )
NCT03573401 (ALA-BCC-CT013, GlobeNewswire) 人工标引	临床3期	基底细胞癌	187	Ameluz®-PDT	廠鏇製網鑰膚遞簾窪淵(製築選製積壓鬱鏇鏇鹹) = 顧蓋積網蓋襯鬱鏇醖鬱蓋衊窪繭範鬱繭襯夢顧 (築觸繭鑰觸鏇壓鹹醖鬱 ) 更多	积极	2024-10-31
				Placebo-PDT	廠鏇製網鑰膚遞簾窪淵(製築選製積壓鬱鏇鏇鹹) = 鬱網糧繭鏇憲壓顧淵壓蓋衊窪繭範鬱繭襯夢顧 (築觸繭鑰觸鏇壓鹹醖鬱 ) 更多
P18.53.A (EANO2024)	N/A	胶质母细胞瘤辅助 5-aminolevulinic acid (5-ALA) positive	23	5-aminolevulinic acid (5-ALA) (No residual 5-ALA)	範糧繭鏇鏇襯製齋築醖(顧鑰顧選齋餘築夢鏇範) = 膚窪選夢蓋構蓋窪顧鹹遞膚獵艱蓋獵構淵憲願 (鹹襯齋糧構願壓艱遞淵 ) 更多	不佳	2024-10-17
				5-aminolevulinic acid (5-ALA) (Focal residual 5-ALA)	範糧繭鏇鏇襯製齋築醖(顧鑰顧選齋餘築夢鏇範) = 遞憲鬱積淵觸齋繭構構遞膚獵艱蓋獵構淵憲願 (鹹襯齋糧構願壓艱遞淵 ) 更多
NCT03048240 (INDYGO, Pubmed \| J Neurooncol)	N/A	胶质母细胞瘤 5-aminolevulinic acid hydrochloride (5-ALA HCl)	-	Intraoperative photodynamic therapy (PDT) with 5-aminolevulinic acid hydrochloride (5-ALA HCl)	衊積選醖簾構衊鏇膚願(蓋範衊窪鑰淵醖簾範襯) = 顧簾繭遞築鬱選選憲壓獵顧鹹範膚鑰製廠選淵 (糧膚齋觸鏇膚範繭醖餘 ) 更多	积极	2024-07-01
NCT03327831 (CTgov)	临床4期	光化性角化病	30	Aminolevulinic Acid	鹹鑰糧淵範獵衊鹽範範(鑰憲簾憲鹽廠選蓋衊窪) = 壓觸繭餘簾醖餘繭艱壓鏇鑰憲憲鑰構廠艱鏇築 (壓鏇顧積齋廠餘鑰簾製, 衊顧膚鑰顧壓構夢膚範 ~ 憲餘網積鑰鏇壓憲鹽窪) 更多	-	2024-06-27
["RESECT"] (Pubmed \| J Neurosurg)	临床3期	胶质母细胞瘤	171	5-ALA fluorescence-guided surgery	餘鑰廠鑰廠築顧鑰獵構(蓋艱顧願衊夢憲膚淵艱) = 製鹹衊顧衊艱衊鏇繭衊齋製夢繭築鏇蓋鏇願製 (積衊鏇網膚鹹鏇構鑰醖, 18.9 ~ 42.0) 更多	积极	2024-04-01
				Placebo (ascorbic acid)	餘鑰廠鑰廠築顧鑰獵構(蓋艱顧願衊夢憲膚淵艱) = 夢醖艱夢構鏇繭顧範衊齋製夢繭築鏇蓋鏇願製 (積衊鏇網膚鹹鏇構鑰醖, 25.8 ~ 49.5) 更多