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Accelerating Neuroblastoma Drug Development

Neuroblastoma presents significant therapeutic challenges due to its heterogeneity, aggressive progression, and limited treatment options for high-risk patients. Protheragen stands as a specialized partner in Neuroblastoma drug development, offering a comprehensive suite of preclinical solutions tailored to advance novel therapeutics from target validation through IND-enabling studies. Leveraging deep scientific expertise and state-of-the-art platforms, Protheragen delivers robust data packages that support informed decision-making and streamline the drug development process. The company’s integrated approach encompasses molecular biology, pharmacology, in vivo efficacy, and safety assessments, all conducted in strict adherence to regulatory standards. Protheragen’s commitment to scientific rigor and regulatory compliance ensures that clients’ Neuroblastoma programs are positioned for successful clinical translation. With a focus on innovation and efficiency, Protheragen accelerates the path to therapeutic breakthroughs, driving progress toward improved outcomes for patients affected by this challenging pediatric cancer.

What is NeuroblastomaTargets for NeuroblastomaDrug Discovery and Development ServicesWhy Choose Us

What is Neuroblastoma

Neuroblastoma is a malignant tumor arising from neural crest cells, most commonly affecting the adrenal medulla or sympathetic nervous system in children. The disease is driven by genetic and molecular alterations such as MYCN amplification, ALK mutations, and chromosomal abnormalities, which influence tumor growth and aggressiveness. Neuroblastoma displays a wide spectrum of clinical behavior, ranging from tumors that may regress spontaneously to highly aggressive forms that metastasize to bone, bone marrow, liver, and lymph nodes. The tumor’s clinical course and prognosis are determined by factors such as patient age, tumor stage, and underlying genetic features. Clinically, neuroblastoma can present with an abdominal mass, pain, bone pain, or systemic symptoms like fever and weight loss. Diagnosis involves imaging studies (ultrasound, CT, MRI), functional imaging with MIBG scintigraphy, and laboratory testing for elevated urinary catecholamine metabolites. Definitive diagnosis requires a biopsy and histopathological analysis, supported by molecular studies for risk stratification. Treatment depends on risk category and may include surgery, chemotherapy, radiotherapy, and immunotherapy. Anti-GD2 monoclonal antibodies (such as naxitamab and dinutuximab) and targeted radiotherapy with iobenguane I 131 are used in high-risk or relapsed cases. Prognosis varies widely, with excellent outcomes in low-risk patients and ongoing challenges in high-risk disease.

Launched Drugs

Structure Generic Name CAS Registry Number Molecular Formula Molecular Weight
naxitamab (Rec INN; USAN); naxitamab-gqgk 1879925-92-4
dinutuximab beta (Rec INN) 1613303-02-8
dinutuximab (Prop INN; USAN) 1363687-32-4
img-77679-27-7-iobenguane-i-131-usaniobenguane131i-rec-inn iobenguane I 131 (USAN); iobenguane[131I] (Rec INN) 77679-27-7 C8 H10 I N3 279.185
img-96020-91-6-alpha-difluoromethylornithine-hydrochlorideeflorni alpha-difluoromethylornithine hydrochloride; eflornithine hydrochloride (Rec INNM; USAN; BANM) 96020-91-6 C6 H12 F2 N2 O2 . Cl H . H2 O 236.645
img-unknown-123i-iobenguaneiobenguane-123-i-rec-inniobenguane- 123I-iobenguane; iobenguane (123 I) (Rec INN); iobenguane I 123 (USAN); iobenguane[123I] C8 H10 I N3 271.185

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Targets for Neuroblastoma

Targets in Clinical or Later Phases of Development

Target Name Gene Symbol
Arginase (nonspecified subtype)
ornithine decarboxylase 1 ODC1
solute carrier family 6 member 2 SLC6A2
cereblon CRBN
DNA Topoisomerase II (nonspecified subtype)
epidermal growth factor receptor EGFR
neural cell adhesion molecule 1 NCAM1
DCX (DDB1-CUL4-X-box) E3 protein ligase complex
retinoic acid receptor gamma RARG
nucleophosmin 1 NPM1

Neuroblastoma pathogenesis is driven by dysregulation of several molecular targets that orchestrate tumor growth, survival, and differentiation. Key cell surface markers such as Glypican 2 (GPC2) and Neural Cell Adhesion Molecule 1 (NCAM1) are highly expressed on neuroblastoma cells, mediating cell adhesion, migration, and immune recognition. Oncogenic signaling is fueled by proteins like Epidermal Growth Factor Receptor (EGFR), which activates proliferative pathways, and Ornithine Decarboxylase 1 (ODC1), a crucial enzyme in polyamine biosynthesis regulated by MYCN amplification. Nuclear factors including Nucleophosmin 1 (NPM1), Retinoic Acid Receptors (RARB, RARG), and RAR Related Orphan Receptor B (RORB) modulate differentiation and cell cycle progression, while DNA Topoisomerase I (TOP1) ensures DNA replication and repair, representing a vulnerability in rapidly dividing tumor cells. The therapeutic potential of these targets is underscored by ongoing clinical and preclinical efforts. Immunotherapeutic strategies targeting GPC2 and NCAM1, such as antibody-drug conjugates and CAR-T cells, have demonstrated promising anti-tumor activity. Inhibitors of EGFR and ODC1 are under investigation, with agents like DFMO in clinical trials for maintenance therapy. Retinoid-based differentiation therapies leverage RARB and RARG pathways to induce tumor cell maturation. TOP1 inhibitors, including topotecan and irinotecan, are established components of neuroblastoma chemotherapy. Collectively, these targets enable precision medicine approaches, offering hope for improved outcomes in high-risk neuroblastoma patients.

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Drug Discovery and Development Services

In Vitro Efficacy Testing ServicesIn Vivo Model DevelopmentPK/PD Study ServicesIn Vivo Toxicity Assessment ServicesBiomarker Analysis Services
In Vitro Efficacy Testing Services

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Our In Vitro Efficacy Testing Service accelerates Neuroblastoma drug discovery by providing comprehensive screening and characterization platforms. We utilize advanced biochemical and cell-based assays, including ATP, chemiluminescent, ELISA, FRET, fluorescent polarization, HTRF, and RNA assays, to assess compound potency, mechanism, and pathway modulation. Key targets such as ALK, MYCN, Trk receptors, EGFR, Cereblon, and RIPK2 are evaluated using industry-standard parameters like IC-50, EC-50, Kd, MIC, and pIC-50. This robust data enables informed candidate selection, lead optimization, and preclinical decision-making, ensuring efficient development of effective Neuroblastoma therapies.

Cereblon Dna Topoisomerase I
Epidermal Growth Factor Receptor Receptor Interacting Serine/Threonine Kinase 2
Tumor Necrosis Factor

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Why Choose Us

Choosing Protheragen means partnering with a company that possesses specialized expertise in Neuroblastoma research and drug development. Our professional teams are composed of experienced scientists and clinicians dedicated to advancing therapies for Neuroblastoma, leveraging advanced technology platforms that enable innovative and efficient drug discovery. At Protheragen, we take pride in our proven track record and reliability in delivering comprehensive preclinical drug development services, ensuring that each project is managed with the utmost precision and care. Our commitment to quality is reflected in our strict adherence to industry standards and regulatory compliance throughout every stage of the development process. We understand the unique challenges of Neuroblastoma therapeutics and are deeply committed to driving progress in this field. By choosing Protheragen, you gain a trusted partner devoted to scientific excellence, integrity, and the shared goal of bringing new hope to patients and families affected by Neuroblastoma.

FAQs for Our Services

Q: What are the main preclinical research challenges specific to developing new drugs for Neuroblastoma?

A: Neuroblastoma presents unique preclinical challenges, including its biological heterogeneity, the presence of both MYCN-amplified and non-amplified subtypes, and the difficulty in establishing representative in vitro and in vivo models. Our company addresses these challenges by utilizing a diverse panel of cell lines and patient-derived xenograft (PDX) models to ensure robust efficacy and safety assessments. We also employ advanced molecular profiling to tailor preclinical studies to specific disease subtypes.

Q: What regulatory considerations should be taken into account during preclinical drug development for Neuroblastoma?

A: Preclinical drug development for Neuroblastoma must adhere to regulatory guidelines set by agencies such as the FDA and EMA, particularly regarding pediatric indications. This includes compliance with Good Laboratory Practice (GLP), the use of relevant animal models, and the generation of comprehensive safety, pharmacokinetic, and toxicology data. Our team has extensive experience in designing studies that meet regulatory expectations and in preparing documentation for Investigational New Drug (IND) applications.

Q: What technical aspects are critical in preclinical Neuroblastoma research?

A: Critical technical aspects include the selection of appropriate cell lines and animal models, the implementation of high-throughput screening platforms, and the use of advanced imaging and biomarker analysis to monitor tumor progression and drug response. Our company leverages state-of-the-art technologies, such as 3D tumor spheroid assays and bioluminescent imaging, to generate high-quality, translatable data that supports decision-making in early drug development.

Q: What are the typical timeline and cost considerations for preclinical development of Neuroblastoma therapeutics?

A: The preclinical phase for Neuroblastoma drug development typically spans 12 to 24 months, depending on the complexity of the compound and the breadth of required studies. Costs can range from several hundred thousand to a few million dollars, influenced by the need for specialized models, comprehensive toxicology, and regulatory documentation. We work closely with clients to design cost-effective, milestone-driven programs that optimize resource allocation and accelerate progression to clinical trials.

Q: What are the key success factors in preclinical drug development for Neuroblastoma?

A: Key success factors include the use of clinically relevant models, early identification and validation of predictive biomarkers, adherence to regulatory requirements, and effective risk management throughout the development process. Our integrated approach combines scientific expertise, regulatory knowledge, and project management to maximize the likelihood of successful IND submission and clinical translation.

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