This page provides a comprehensive overview of therapeutic approaches for neurodegenerative diseases, including Alzheimer's disease (AD), Parkinson's disease (PD), amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and Huntington's disease (HD).
Disease-modifying therapies aim to slow or halt disease progression by targeting underlying pathological mechanisms:
| Target | Approach | Development Stage | Examples |
|--------|----------|-------------------|----------|
| Amyloid-beta | Immunotherapy, secretase inhibitors | Approved/Phase 3 | Lecanemab, Aducanumab |
| Tau | Immunotherapy, kinase inhibitors | Phase 2/3 | Gosuranemab, Tilavonemab |
| Alpha-synuclein | Immunotherapy, aggregation inhibitors | Phase 2 | Prasinezumab, Anle138b |
| TDP-43 | Gene therapy, small molecules | Preclinical | ASO strategies |
| SOD1 | Gene silencing | Approved | Tofersen |
These treatments address symptoms rather than disease progression:
Cognitive Symptoms:
Motor Symptoms:
- Dopaminergic agents (levodopa, dopamine agonists)
- MAO-B inhibitors (selegiline, rasagiline)
- COMT inhibitors (entacapone, tolcapone)
Behavioral Symptoms:
- Antipsychotics (risperidone, olanzapine)
- Antidepressants (SSRIs, SNRIs)
- Mood stabilizers
- Amyloid-targeted: Lecanemab, Donanemab
- Tau-targeted: Semorinemab, Gosuranemab
- Neuroprotective: Memantine, Actovegin
- Symptomatic: Cholinesterase inhibitors
- Lifestyle: Cognitive stimulation, exercise
- Dopamine replacement: Levodopa/carbidopa
- Dopamine agonists: Pramipexole, ropinirole
- MAO-B inhibitors: Selegiline, rasagiline
- COMT inhibitors: Entacapone
- Advanced therapies: DBS, levodopa infusion
- Disease-modifying: Riluzole, edaravone
- Symptomatic: Muscle relaxants, pain management
- Experimental: Gene therapies (SOD1, C9orf72)
- Supportive: Respiratory support, nutritional support
- AAV-vector delivered genes
- CRISPR-based gene editing
- Antisense oligonucleotides (ASOs)
- RNA interference (RNAi)
- Stem cell transplantation
- Exosome therapy
- Cell replacement
- Active immunization (vaccines)
- Passive immunization (monoclonal antibodies)
- Adoptive cell therapy (CAR-T)
| Method |
Advantages |
Limitations |
| Oral |
Convenient, widely used |
Blood-brain barrier |
| Intravenous |
Systemic delivery |
Peripheral effects |
| Intrathecal |
Direct CNS delivery |
Invasive |
| Intranasal |
Non-invasive, targeted |
Limited volume |
| Gene therapy |
Long-lasting |
Irreversible |
¶ Challenges and Future Directions
- Blood-brain barrier: Limiting CNS drug delivery
- Patient heterogeneity: Variable treatment response
- Biomarker development: Need for early detection
- Trial design: Long disease progression, placebo effects
- Combination therapy: Optimizing multi-target approaches
- Precision medicine approaches
- Biomarker-driven patient selection
- Early intervention strategies
- Multi-target combination therapies
- Regenerative medicine
- Treatments Overview — This page
- Treatments Rankings — Ranked list by research activity
- Treatments Dashboard — Data visualizations and metrics
- Total Pages: 538 Treatments pages in this section
- Last Updated: This section is actively maintained
- AADC Gene Therapy for Parkinson's Disease
- AADvac1 - Tau Vaccine
- AAV Gene Therapy Vectors for Neurodegenerative Diseases
- AAV Gene Therapy for Neurodegeneration
- AAV Vectors in Neurodegenerative Disease Gene Therapy
- ACI-35 Liposomal Vaccine
- AD Combination Therapy Matrix - 15x15 Scoring
- AD Therapeutic Approaches Scorecard - 28 Approaches Ranked
- ALS Therapeutics
- ALS Treatment Strategies
- AMPA Receptor Antagonists in Neurodegenerative Disease
- AMPK Activators
- AMPK Activators for Neurodegeneration
- Acupuncture Therapy for Neurodegeneration
- Adenosine A2A Receptor Antagonist Therapy
- Aducanumab (Aduhelm)
- AhR Modulator Therapy for Neurodegenerative Diseases
- Alpha-7 Nicotinic Acetylcholine Receptor Agonist Therapy
- Alpha-Lipoic Acid for Neurodegeneration
- Alpha-Synuclein Aggregation Inhibitors
- Alpha-Synuclein Aggregation Inhibitors
- Alpha-Synuclein Immunotherapy
- Alpha-Synuclein Reduction Therapies for Neurodegenerative Diseases
- Alpha-Synuclein-Targeting Therapies
- Alzheimer's Disease Treatment
- Amantadine
- Ambroxol for Neurodegeneration (GCase Chaperone Strategy)
- Ambroxol for Parkinson's Disease
- Amyloid Immunotherapy Vaccines for Alzheimer's Disease
- Amyotrophic Lateral Sclerosis (ALS) Treatment
See all 538 treatments pages...
- PSP/CBS Treatments: No dedicated treatment overview page exists
- MSA Treatments: Missing comprehensive treatment summary
- Huntington's Disease Treatments: Need expanded coverage beyond basic symptom management
- DLB Treatments: Limited treatment options documented
- Vascular Dementia Treatments: Underrepresented in treatment rankings
- FTD Treatment Landscape: Only one page exists, needs expansion
- Combination Therapies: AD combination therapy matrix exists but needs expansion to other diseases
- Gene Therapy for non-PD diseases: Heavy focus on PD, limited coverage for AD/ALS gene therapy
- Cell Therapy: Need more pages on stem cell approaches across diseases
- Preventive/Prophylactic Therapies: Limited documentation of prevention strategies
- Biomarker-Driven Therapies: Only one page (ATN framework), needs expansion
- Mechanisms: Many treatments lack links to their target mechanism pages
- Clinical Trials: Need more trial status integration
- Genes/Proteins: Incomplete linking to genetic targets
- Create PSP Treatment Overview page
- Create MSA Treatment Overview page
- Expand FTD Therapeutic Landscape page
- Add more gene therapy pages for non-PD targets