LAMP1

Lysosomal associated membrane protein 1

Score: 0.539 Price: $0.54 Low Druggability Status: active Wiki: LAMP1

🔮 Lysosomal / Autophagy 🧠 Neurodegeneration

HYPOTHESES

11

PAPERS

28

KG EDGES

739

DEBATES

1

3D Protein Structure

🧬 LAMP1 — PDB 5GV0 Click to expand

Experimental structure from RCSB PDB | Powered by Mol*

Druggability & Clinical Context

Druggability

Low

Score: 0.43

Clinical Stage

Phase I

Target Class

Structural Protein

Safety

0.20

Druggability Analysis

Drug Development0.60

Structural Tractability0.70

Target Class0.50

Safety Profile0.20

Key Metrics

PDB Structures:

5

Known Drugs:

4

Approved:

3

In Clinical Trials:

0

Drug Pipeline (4 compounds)

3 Approved

Druggability Rationale: LAMP1 demonstrates moderate druggability (0.55 score) supported by high-resolution structural data (2.37 Å), multiple PDB structures, and existing approved drugs that modulate its pathway indirectly. However, as a structural glycoprotein lacking a canonical enzymatic active site, direct small-molecule targeting is challenging, requiring focus on allosteric modulation or protein-protein interaction interfaces rather than traditional orthosteric binding.

Mechanism: Direct LAMP1 modulators enhance lysosomal membrane trafficking and autophagy flux, while indirect pathway drugs alter lysosomal pH and autophagosome-lysosome fusion dynamics to modulate LAMP1-mediated cellular clearance mechanisms.

Drug Pipeline (4 compounds)

3 Approved

Known Drugs:

Asunaprevir (approved) — Hepatitis C virus infection (indirect LAMP1 pathway involvement)

Chloroquine (approved) — Malaria, lupus (affects lysosomal function and LAMP1-mediated autophagy)

Hydroxychloroquine (approved) — Rheumatoid arthritis, lupus (modulates lysosomal autophagy pathway)

ML-SA1 (research) — Lysosomal storage diseases (investigational TFEB/LAMP1 pathway activator)

Structural Data:

PDB (5) ✓AlphaFold ✓Cryo-EM ✓

5GV0 8ATH 8FY5 8FYF 9C59

UniProt: P11279

🧬 3D Protein Structure

🧬 LAMP1 — PDB 5GV0 Click to expand interactive 3D viewer

Experimental structure from RCSB PDB | Powered by Mol* | Rotate: click+drag | Zoom: scroll

Selectivity & Safety Considerations

LAMP1 selectivity is complicated by its structural role and interaction with multiple autophagy/lysosomal proteins (LAMP2, TFEB, ATG proteins), presenting risk of off-target modulation of the broader autophagy-lysosomal pathway. Isoform selectivity is less critical as LAMP1 is the predominant lysosomal isoform, though distinguishing direct LAMP1 effects from pathway-wide effects remains a key challenge.

3D Protein Structure

PDB: Open in RCSB AlphaFold model

Interactive 3D viewer powered by RCSB PDB / Mol*. Use mouse to rotate, scroll to zoom.

Clinical Trials (8)

Relevant trials from ClinicalTrials.gov

Active

2

Completed

6

Total Enrollment

1,037

By Phase

PHASE1: 1 · PHASE2: 4 · PHASE3: 3

The Effects of Hydroxychloroquine in Patients with Inflammatory Cardiomyopathy Recruiting

PHASE2 NCT05961202 n=200

Inflammatory Cardiomyopathy, Myocarditis

Interventions: Hydroxychloroquine, Prednisolone

Sponsor: Tongji Hospital | Started: 2021-11-01

Erlotinib With or Without Hydroxychloroquine in Chemo-Naive Advanced NSCLC and (EGFR) Mutations Active Not Recruiting

PHASE2 NCT00977470 n=76

Non-small Cell Lung Cancer

Interventions: Erlotinib, Hydroxychloroquine

Sponsor: Massachusetts General Hospital | Started: 2009-10

A Phase 3, Comparative Study of Asunaprevir and Daclatasvir Combination Therapy Versus Telaprevir Therapy in Japanese HC Completed

PHASE3 NCT01718145 n=258

Hepatitis C Virus Infection

Interventions: Daclatasvir, Asunaprevir, Ribavirin

Sponsor: Bristol-Myers Squibb | Started: 2012-11

UNITY 3: A Japanese Phase 3 Study of a Daclatasvir/Asunaprevir/BMS-791325 in Subjects With Genotype 1 Chronic Hepatitis Completed

PHASE3 NCT02123654 n=297

Hepatitis C Virus Infection

Interventions: Daclatasvir, Asunaprevir, DCV 3DAA

Sponsor: Bristol-Myers Squibb | Started: 2014-04

Hydroxychloroquine and Ivermectin for the Treatment of COVID-19 Infection Completed

PHASE3 NCT04391127 n=108

COVID-19

Interventions: Hydroxychloroquine, Ivermectin, Placebo

Sponsor: Centenario Hospital Miguel Hidalgo | Started: 2020-05-04

Safety, Tolerability, and Efficacy of Asunaprevir and Daclatasvir in Subjects Coinfected With HIV-HCV Completed

PHASE2 NCT02124044 n=30

HIV-HCV

Interventions: Asunaprevir and Daclatasvir, Asunaprevir and Daclatasvir with BMS-791

Sponsor: National Institutes of Health Clinical Center (CC) | Started: 2014-02

Hydroxychloroquine and Phlebotomy for Treating Porphyria Cutanea Tarda Completed

PHASE2 NCT01573754 n=48

Porphyria Cutanea Tarda

Interventions: Hydroxychloroquine, Phlebotomy

Sponsor: The University of Texas Medical Branch, Galveston | Started: 2006-03-21

Drug Interaction Study of an OCP (Norethindrone (ND) Acetate and Ethinyl Estradiol (EE))With a Combination of Daclatasvi Completed

PHASE1 NCT02103569 n=20

Hepatitis C

Interventions: FDC of Daclatasvir, Asunaprevir and BMS-, FDC of Norethindrone and Ethinyl Estradi, BMS-791325

Sponsor: Bristol-Myers Squibb | Started: 2014-04

Linked Hypotheses (1)

Lysosomal Positioning Dynamics Modulation0.686

Linked Experiments (0)

No linked experiments

Scoring Dimensions

Knowledge Graph (20)

associated with (1)

LAMP1→neurodegeneration

co discussed (6)

STX17→LAMP1CHMP2B→LAMP1LAMP1→MCOLN1LAMP1→FOXO1LAMP1→IGF2R

▸ Show 1 more

regulates (13)

ACTB→LAMP1PPARA→LAMP1CAT→LAMP1SQSTM1→LAMP1RPS6KB1→LAMP1

▸ Show 8 more

RAB7A→LAMP1SREBF1→LAMP1EIF4EBP1→LAMP1CTSD→LAMP1PPARGC1A→LAMP1LRP2→LAMP1MAP1LC3B→LAMP1MYC→LAMP1

Debate History (1)

Should LAMP1 (Lysosomal associated membrane protein 1) be prioritized as a thera2026-04-22