Growth Factor Therapies For Neurodegeneration is an important component in the neurobiology of neurodegenerative diseases. This page provides detailed information about its structure, function, and role in disease processes.
Growth factors are proteins that regulate cell survival, proliferation, differentiation, and function. Neurotrophic growth factors are essential for neuronal survival and synaptic plasticity. Delivery of growth factors or their mimetics represents a promising therapeutic approach for neurodegenerative diseases.
- BDNF (Brain-Derived Neurotrophic Factor)
- NGF (Nerve Growth Factor)
- NT-3 (Neurotrophin-3)
- NT-4/5 (Neurotrophin-4/5)
- GDNF (Glial Cell Line-Derived Neurotrophic Factor)
- Neurturin
- Artemin
- Persephin
- FGF-2 (bFGF)
- FGF-20
- FGF-21
- IGF-1 (Insulin-like Growth Factor-1)
- EGF (Epidermal Growth Factor)
- VEGF (Vascular Endothelial Growth Factor)
Growth factors signal through:
- Tyrosine kinase receptors (TrkA/B/C, Ret, FGFR)
- P75NTR (pan-neurotrophin receptor)
- ** downstream pathways**:
- Anti-apoptotic: Activation of survival pathways
- Synaptic plasticity: Enhanced spine formation, LTP
- Neurogenesis: Support of neural stem cells
- Axonal growth: Promotes regeneration
- Glial support: Protects oligodendrocytes
- Anti-inflammatory: Modulates microglia
- BDNF: Synaptic plasticity, memory enhancement
- NGF: Cholinergic neuron survival
- Research: PMID 10341227, PMID 14653450
- GDNF: Dopaminergic neuron protection
- Neurturin: Clinical trials
- BDNF: Nigral neuron survival
- Research: PMID 10400110, PMID 10930470
- BDNF, CNTF, IGF-1: Motor neuron protection
- GDNF: Limited by delivery
- Research: PMID 11592541, PMID 14529974
- BDNF: Reduced in HD, therapeutic target
- CNTF: Neuroprotective effects
- IGF-1: Synaptic function
- Research: PMID 14690788, PMID 15694523
- NGF, BDNF: Post-stroke recovery
- VEGF: Angiogenesis
- FGF: Neurogenesis
- Research: PMID 14744759
¶ Clinical Candidates
¶ BDNF and BDNF Mimetics
| Agent |
Type |
Delivery |
Status |
| BDNF protein |
Native protein |
Intranasal, ICV |
Phase 1/2 |
| 7,8-DHF |
BDNF mimetic |
Oral |
Preclinical |
| TrkB agonists |
Small molecule |
Various |
Preclinical |
| AAV-BDNF |
Gene therapy |
Intraparenchymal |
Phase 1 |
| Agent |
Type |
Delivery |
Status |
| GDNF protein |
Native protein |
Intraputaminal |
Phase 2 |
| Neurturin |
Native protein |
Intraputaminal |
Phase 2 |
| AAV2-Neurturin |
Gene therapy |
Intraputaminal |
Phase 1 |
| Agent |
Target |
Delivery |
Status |
| NGF (NGF) |
TrkA |
Eye drops |
Phase 2 (AD) |
| IGF-1 |
IGF-1R |
Subcutaneous |
Phase 2 (ALS) |
| FGF-20 |
FGFR1 |
Various |
Preclinical |
- Delivery: BBB limits systemic delivery
- Half-life: Short protein half-life
- Side effects: Off-target effects possible
- Dosing: Chronic infusion needed
- Cost: High manufacturing costs
- Intranasal: Bypasses BBB for some proteins
- Intracerebral: Direct to brain (invasive)
- Gene therapy: AAV-mediated expression
- Cell therapy: Genetically engineered cells
- BBB-penetrant small molecules
- TrkB-selective agonists
- Sustained release formulations
- Gene therapy vectors
- Cellular delivery systems
The study of Growth Factor Therapies For Neurodegeneration has evolved significantly over the past decades. Research in this area has revealed important insights into the underlying mechanisms of neurodegeneration and continues to drive therapeutic development.
Historical context and key discoveries in this field have shaped our current understanding and will continue to guide future research directions.
- PMID:10341227 - BDNF in CNS disorders
- PMID:14653450 - NGF and Alzheimer's disease
- PMID:10400110 - GDNF in Parkinson's disease
- PMID:10930470 - Neurturin clinical trials
- PMID:11592541 - Neurotrophic factors in ALS
- PMID:14690788 - BDNF in Huntington's disease
- PMID:14744759 - Growth factors in stroke
- PMID:15761078 - Growth factor delivery challenges