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Accelerating Amyotrophic Lateral Sclerosis Drug Development

Amyotrophic lateral sclerosis (ALS) presents a significant therapeutic challenge, with limited treatment options and a complex pathophysiology that demands innovative drug development strategies. Protheragen stands as a specialized partner in ALS drug discovery and preclinical development, leveraging deep scientific expertise to address the urgent need for effective therapeutics. Protheragen offers comprehensive preclinical solutions encompassing target validation, lead optimization, pharmacology, toxicology, and IND-enabling studies. Utilizing advanced technology platforms and robust in vitro and in vivo models, Protheragen ensures rigorous data generation and translational relevance throughout the preclinical pipeline. Our teams integrate cutting-edge methodologies with strict adherence to global regulatory standards, supporting the seamless progression of candidate molecules toward clinical evaluation. With a proven track record in neurodegenerative disease research, Protheragen is dedicated to accelerating the development of novel ALS therapies. Through scientific excellence, state-of-the-art infrastructure, and unwavering regulatory compliance, Protheragen empowers partners to overcome the complexities of ALS drug development and advance transformative treatments for patients in need.

What is Amyotrophic Lateral SclerosisTargets for Amyotrophic Lateral SclerosisDrug Discovery and Development ServicesWhy Choose Us

What is Amyotrophic Lateral Sclerosis

Amyotrophic Lateral Sclerosis (ALS) is a progressive neurodegenerative disorder marked by the selective loss of motor neurons in the brain and spinal cord, resulting in muscle weakness, atrophy, and eventual paralysis. ALS affects both upper and lower motor neurons, leading to a combination of spasticity and muscle wasting. The disease arises from a complex interplay of genetic, molecular, and environmental factors. While most cases are sporadic with unclear causes, about 5–10% are familial, often linked to mutations in genes such as SOD1, C9orf72, TARDBP, and FUS. Pathogenic mechanisms include glutamate-induced excitotoxicity, oxidative stress, mitochondrial dysfunction, impaired axonal transport, and neuroinflammation. Clinically, ALS presents with progressive weakness, muscle wasting, fasciculations, and signs of both upper and lower motor neuron dysfunction. Initial symptoms may involve the limbs (limb-onset) or bulbar muscles, affecting speech and swallowing (bulbar-onset). Diagnosis is primarily clinical, supported by electromyography (EMG), nerve conduction studies, and MRI to exclude other conditions. The revised El Escorial and Awaji criteria guide diagnostic confirmation. Treatment options are limited but include riluzole, which inhibits glutamate release, and edaravone, which reduces oxidative stress. Targeted therapies such as tofersen for SOD1 mutations, cell-based therapies like lenzumestrocel, and neurotrophic agents such as mecobalamin may also be used. Despite treatment, ALS remains a progressive and ultimately fatal disease.

Launched Drugs

Structure Generic Name CAS Registry Number Molecular Formula Molecular Weight
tofersen (Rec INN; USAN) 2088232-70-4
lenzumestrocel (Rec INN) 3037017-62-9
img-89-25-8-edaravone-prop-inn-usan edaravone (Prop INN; USAN) 89-25-8 C10 H10 N2 O 174.199
img-1744-22-5-riluzole-rec-inn-usan riluzole (Rec INN; USAN) 1744-22-5 C8 H5 F3 N2 O S 234.198
img-13422-55-4-mecobalaminmethyl-cobalaminemethyl-vitamin-b12meth mecobalamin; methyl cobalamine; methyl vitamin B12; methylcobalamin 13422-55-4 C63 H91 Co N13 O14 P 1344.382

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Targets for Amyotrophic Lateral Sclerosis

Targets in Clinical or Later Phases of Development

Target Name Gene Symbol
5'-nucleotidase ecto NT5E
Glutamate Transporter (nonspecified subtype)
Voltage-Gated Sodium Channel Complex
Sodium channel (nonspecified subtype)
superoxide dismutase 1 SOD1
transient receptor potential cation channel subfamily C member 5 TRPC5
FUS RNA binding protein FUS
5-hydroxytryptamine receptor 1A HTR1A
KIT proto-oncogene, receptor tyrosine kinase KIT
Neurotrophic Factor (nonspecified subtype)

Amyotrophic lateral sclerosis (ALS) is driven by complex molecular mechanisms involving several key therapeutic targets. Notably, Superoxide Dismutase 1 (SOD1) and FUS RNA Binding Protein (FUS) are central to ALS pathogenesis due to their roles in protein misfolding and aggregation, leading to motor neuron toxicity and degeneration. SOD1 mutations result in toxic protein aggregates that disrupt mitochondrial function and cellular homeostasis, while FUS mutations cause its mislocalization and aggregation, impairing RNA metabolism and DNA repair. Additional targets include Poly(ADP-ribose) Polymerase 1 (PARP1), which mediates DNA repair and, when overactivated, contributes to neurodegeneration through energy depletion, and Toll Like Receptor 4 (TLR4), a key mediator of neuroinflammation that exacerbates motor neuron loss. Troponin T3 (TNNT3), although not directly involved in neuronal pathology, reflects neuromuscular junction dysfunction and muscle atrophy in ALS.

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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 for Amyotrophic Lateral Sclerosis (ALS) provides comprehensive, disease-relevant screening of drug candidates using advanced cellular models. Key capabilities include high-content imaging, cytotoxicity, oxidative stress, mitochondrial function, and neuroinflammation assays, enabling robust evaluation of therapeutic efficacy and mechanism of action. We assess critical parameters such as cell viability, protein aggregation, ROS, ATP levels, cytokine release, and neurite outgrowth. This integrated approach accelerates candidate selection, de-risks drug development, and supports translational success by delivering actionable insights into compound safety, potency, and ALS-specific disease modification potential.

5-Hydroxytryptamine Receptor 1A

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Why Choose Us

Choosing Protheragen means partnering with a team that brings deep expertise and unwavering dedication to Amyotrophic lateral sclerosis (ALS) research and drug development. At Protheragen, we have assembled a group of highly skilled professionals who are well-versed in the complexities of ALS, leveraging years of experience to drive innovation in therapeutic solutions. Our advanced technology platforms and state-of-the-art facilities empower us to deliver comprehensive preclinical drug development services that meet the highest industry standards. Protheragen has built a strong track record of reliability, consistently providing clients with robust, reproducible results that accelerate the path from discovery to clinical application. We adhere strictly to rigorous quality standards and regulatory compliance, ensuring that every project is executed with precision and integrity. Above all, Protheragen is committed to making a meaningful difference in the fight against ALS by advancing novel therapeutics and supporting our clients every step of the way. Trust Protheragen to be your reliable partner in pioneering the future of ALS treatment.

FAQs for Our Services

Q: What are the main preclinical research challenges specific to Amyotrophic Lateral Sclerosis (ALS) drug development?

A: One of the primary challenges in preclinical ALS drug development is the lack of fully predictive animal models that accurately recapitulate the complexity and heterogeneity of human ALS. Additionally, the multifactorial nature of ALS pathogenesis, including both genetic and sporadic forms, complicates target validation and efficacy assessment. Our company addresses these challenges by utilizing a combination of in vitro systems (e.g., patient-derived iPSC motor neurons) and multiple in vivo models, along with advanced biomarker analysis to improve translational relevance.

Q: What are the key regulatory considerations for preclinical development of ALS therapeutics?

A: Regulatory agencies such as the FDA and EMA require robust preclinical efficacy and safety data before granting approval to proceed to clinical trials. For ALS, it is critical to demonstrate target engagement, mechanism of action, and safety in relevant models. Our team is experienced in designing and executing studies that meet regulatory expectations, including GLP-compliant safety pharmacology, toxicology, and biodistribution studies, as well as compiling comprehensive IND/IMPD-enabling data packages.

Q: What technical aspects are important to consider when conducting preclinical research for ALS?

A: Technical considerations include the selection of appropriate animal models (e.g., SOD1-G93A mice, TDP-43, or FUS models), the use of advanced behavioral and functional assays to assess motor deficits, and the implementation of sensitive biomarkers for disease progression. Our company leverages cutting-edge imaging, electrophysiology, and molecular biology techniques to provide detailed mechanistic insights and quantitative data, ensuring robust and reproducible results.

Q: What are the typical timeline and cost considerations for preclinical ALS drug development?

A: Preclinical development for ALS therapeutics typically spans 18-36 months, depending on the complexity of the program and regulatory requirements. Costs vary widely, but a comprehensive preclinical package—including efficacy, pharmacokinetics, safety pharmacology, and toxicology—can range from $2 million to $7 million USD. Our company offers flexible, milestone-driven project management and transparent budgeting to help clients optimize both timelines and costs.

Q: What are the critical success factors for preclinical ALS drug development?

A: Success in ALS drug development relies on rigorous target validation, the use of translationally relevant models and biomarkers, and early engagement with regulatory authorities. Additionally, reproducibility of efficacy data across multiple models and endpoints is essential. Our expertise in study design, execution, and data interpretation, combined with a collaborative approach, ensures that our clients' programs are positioned for successful advancement to clinical trials.

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