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 (3)

LAMP1 Overexpression to Enhance Lysosomal Capacity Independent of TFEB in Aged Synapses0.000

Lysosomal Positioning Dynamics Modulation0.686

Lysosomal Membrane Stabilization to Prevent Tau Seeding0.000

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