Table 4.
Interventions targeting lysosomes
| Disease | Intervention | Stage of development | Comment | NCT number |
|---|---|---|---|---|
| Atherosclerosis | Temsirolimus ± Dexamethasone | Phase II | mTOR inhibitor | NCT03942601 |
| Temsirolimus | Phase III | mTOR inhibitor | NCT04433572 | |
| Chloroquine | Not applicable | Autophagy/lysosome inhibitor | NCT00455403 | |
| Hydroxychloroquine | Phase IV | Autophagy/lysosome inhibitor | NCT04161339 | |
| Alzheimer's disease | Trehalose | Phase I | Autophagy inducer | NCT04663854 |
| Hydralazine | Phase III | Autophagy inducer | NCT04842552 | |
| Rapamune | Phase I | Autophagy inducer | NCT04200911 | |
| Rapamycin | Phase II | Autophagy inducer | NCT04629495 | |
| Parkinson’s disease | Exablate BBBD with Cerezyme | Not applicable | Enzyme replacement therapy | NCT04370665 |
| Systemic lupus erythematosus | Hydroxychloroquine or chloroquine | Phase II | Autophagy/lysosome inhibitor | NCT01946880 |
| Sirolimus | Phase II | Autophagy inducer | NCT04582136 | |
| Sirolimus | Phase II | Autophagy inducer | NCT04736953 | |
| Rapamycin | Phase II | Autophagy inducer | NCT00779194 | |
| Crohn’s disease | Rapamycin | Not applicable | Autophagy inducer | NCT02675153 |
| Ciprofloxacin + Doxycycline + Hydroxychloroquine + Budesonide | Phase II | Combination of hydroxychloroquine with others | NCT01783106 | |
| Rheumatoid arthritis | Temsirolimus | Phase II | mTOR inhibitor | NCT00076206 |
| Sirolimus | Phase I/II | Autophagy inducer | NCT00392951 | |
| Infliximab + DMARDs (methotrexate; chloroquine; leflunomidum; cyclosporin A; sulfasalazine; OM 89) | Phase III | Combination of chloroquine with others | NCT00521924 | |
| Multiple sclerosis | Temsirolimus | Phase II | mTOR inhibitor | NCT00228397 |
| Sirolimus | Phase I/II | Autophagy inducer | NCT00095329 | |
| Fabry disease | Agalsidase alfa | Phase III | Enzyme replacement therapy | NCT01298141 |
| Agalsidase beta | Phase IV | Enzyme replacement therapy | NCT00081497 | |
| Tay-Sachs disease | AXO-AAV-GM2 | Phase I | Gene therapy | NCT04669535 |
| Mucopolysaccharidosis diseases | Aldurazyme (for type I) | Phase III | Enzyme replacement therapy | NCT00258011 |
| Idursulfase (for type II) | Phase II/III | Enzyme replacement therapy | NCT00630747 | |
| SAF-301 (for type III) | Phase I/II | Gene therapy | NCT02053064 | |
| ABO-102 (for type III) | Phase I/II | Gene therapy | NCT04088734 | |
| Naglazyme (for type IV) | Phase IV | Enzyme replacement therapy | NCT00299000 | |
| elosulfase alfa (for type IV) | Not applicable | Enzyme replacement therapy | NCT03204370 | |
| AAV2/8.TBG.hARSB (for type IV) | Phase I/II | Gene therapy | NCT03173521 | |
| UX003 (for type VII) | Phase I/II | Enzyme replacement therapy | NCT01856218 | |
| Pompe disease | Alglucosidase alfa | Phase IV | Enzyme replacement therapy | NCT04676373 |
| Gaucher disease | Imiglucerase | Phase IV | Enzyme replacement therapy | NCT04656600 |
| Velaglucerase alfa | Phase IV | Enzyme replacement therapy | NCT04718779 | |
| taliglucerase alfa | Phase IV | Enzyme replacement therapy | NCT04002830 | |
| Non-small cell lung cancer | Binimetinib + Hydroxychloroquine | Phase II | Combination Therapy | NCT04735068 |
| Paclitaxel + Carboplatin ± Bevacizumab ± Hydroxychloroquine | Phase II | Combination Therapy | NCT01649947 | |
| Bevacizumab + Carboplatin + Paclitaxel + Hydroxychloroquine | Phase I/II | Combination Therapy | NCT00728845 | |
| Sunitinib + Rapamycin | Phase I | Combination Therapy | NCT00555256 | |
| Neratinib ± Temsirolimus | Phase II | Combination Therapy | NCT01827267 | |
| Small cell lung cancer | Chloroquine | Phase I | Autophagy/lysosome inhibitor | NCT00969306 |
| Etoposide + Carboplatin + Atezolizumab + BNT411 (TLR7/8 agonist) | Phase I/II | Combination Therapy | NCT04101357 | |
| Colon cancer | Bupivacaine liposome suspension (for pain control) | Phase IV | Bupivacaine slowly released from lysosomes | NCT02052557 |
| FOLFOX/bevacizumab ± Hydroxychloroquine | Phase I/II | Combination Therapy | NCT01206530 | |
| Vorinostat + Hydroxychloroquine | Phase II | Combination Therapy | NCT02316340 | |
| nab-rapamycin + mFOLFOX6 and Bevacizumab | Phase I | Combination Therapy | NCT03439462 | |
| Temsirolimus + Cetuximab | Phase I | Combination Therapy | NCT00593060 | |
| Pembrolizumab + Poly-ICLC (TLR3 agonist) | Phase I/II | Combination Therapy | NCT02834052 | |
| FOLFIRI + Cetuximab + IMO-2055 (TLR9 agonist) | Phase I | Combination Therapy | NCT00719199 | |
| Breast cancer | Hydrochloroquine | Phase II | Autophagy/lysosome inhibitor | NCT01292408 |
| Ixabepilone + Hydroxychloroquine | Phase I/II | Combination Therapy | NCT00765765 | |
| Letrozole + Palbociclib + Hydroxychloroquine | Phase I/II | Combination Therapy | NCT03774472 | |
| Chloroquine | Phase II | Autophagy/lysosome inhibitor | NCT02333890 | |
| Zoledronic acid + Odanacatib (cathepsin K inhibitor) | Phase I/II | Combination Therapy | NCT00399802 | |
| Trastuzumab + Rapamycin | Phase II | Combination Therapy | NCT00411788 | |
| Inetetamab + Rapamycin + Chemotherapy | Phase III | Combination Therapy | NCT04736589 | |
| Rapamycin | Phase II | mTOR inhibitor | NCT02642094 | |
| Radiation + Cyclophosphamide + Imiquimod (TLR7 agonist) | Phase I/II | Combination Therapy | NCT01421017 | |
| Hepatocellular cancer | Sorafenib ± Hydroxychloroquine | Phase II | Combination Therapy | NCT03037437 |
| temsirolimus | Phase II | mTOR inhibitor | NCT01079767 | |
| RO7119929 | Phase I | TLR7 agonist | NCT04338685 | |
| Cholangiocarcinoma | ABC294640 ± Hydroxychloroquine | Phase II | Combination Therapy | NCT03377179 |
| Gastrointestinal cancer | Cobimetinib + Atezolizumab + Hydroxychloroquine | Phase I/II | Combination Therapy | NCT04214418 |
| Prostate cancer | Hydroxychloroquine | Early Phase 1 | Autophagy/lysosome inhibitor | NCT02421575 |
| Docetaxel ± Hydroxychloroquine | Phase II | Combination Therapy | NCT00786682 | |
| Odanacatib | Phase II | Cathepsin K inhibitor | NCT00691899 | |
| Temsirolimus | Phase II | mTOR inhibitor | NCT00919035 | |
| Bevacizumab + Temsirolimus | Phase I/II | Combination Therapy | NCT01083368 | |
| Temsirolimus + Diphenhydramine | Phase II | Combination Therapy | NCT00887640 | |
| Biliary cancer | Trametinib + Hydroxychloroquine | Phase II | Combination Therapy | NCT04566133 |
| Ovarian cancer | Temsirolimus | Phase II | mTOR inhibitor | NCT00926107 |
| Cisplatin + Pembrolizumab + Rintatolimod (TLR3 agonist) | Phase I/II | Combination Therapy | NCT03734692 | |
| OC-L + Ampligen (TLR3 agonist) | Phase I/II | Combination Therapy | NCT01312389 | |
| Pancreatic cancer | LY3214996 ± Hydroxychloroquine sulfate | Phase II | Combination Therapy | NCT04386057 |
| Gemcitabine + Abraxane + Hydroxychloroquine | Phase I/II | Combination Therapy | NCT01506973 | |
| Binimetinib + Hydroxychloroquine | Phase I | Combination Therapy | NCT04132505 | |
| Sirolimus | Phase II | mTOR inhibitor | NCT00499486 | |
| Bevacizumab + Temsirolimus | Phase II | Combination Therapy | NCT01010126 | |
| Radiation Therapy + Nivolumab + SD-101 (TLR9 agonist) | Phase I | Combination Therapy | NCT04050085 | |
| INCAGN01949 + CMP-001 (TLR9 agonist) | Phase I | Combination Therapy | NCT04387071 | |
| Melanoma | Dabrafenib + Trametinib ± Hydroxychloroquine | Phase II | Combination Therapy | NCT04527549 |
| Sorafenib + Temsirolimus | Phase I | Combination Therapy | NCT00349206 | |
| MART-1 Antigen ± GLA-SE (TLR4 agonist) | Early Phase I | Combination Therapy | NCT02320305 | |
| NY-ESO-1 protein + Montanide + Poly ICLC (TLR3 agonist) | Phase I/II | Vaccine | NCT01079741 | |
| Multiple myeloma | Bortezomib + hydroxychloroquine | Phase I | Combination Therapy | NCT00568880 |
| Brain neoplasms | Temsirolimus | Phase I | mTOR inhibitor | NCT00949026 |
| Head and neck cancer | Temsirolimus + Weekly Paclitaxel + Carboplatin | Phase I/II | Combination Therapy | NCT01016769 |
| Cetuximab + EMD 1201081 (TLR9 agonist) | Phase II | Combination Therapy | NCT01040832 | |
| Renal cell cancer | Sunitinib + Temsirolimus | Phase I | Combination Therapy | NCT01122615 |
| Bladder cancer | Temsirolimus | Phase II | mTOR inhibitor | NCT01827943 |
| Sirolimus | Early Phase I | mTOR inhibitor | NCT02753309 | |
| Glioma | Tumor-lysate pulsed DC vaccination + adjuvant poly ICLC (TLR3 agonist) | Phase II | Vaccine | NCT01204684 |
| Esophageal cancer | URLC10-177 + TTK-567 + CpG-7909 (TLR9 agonist) | Phase I/II | Vaccine | NCT00669292 |
| Follicular Lymphoma | Radiation Therapy + Ibrutinib + SD-101 (TLR9 agonist) | Phase I/II | Combination Therapy | NCT02927964 |
| Non-Hodgkin Lymphoma | local irradiation + CPG 7909 (TLR9 agonist) | Phase I/II | Combination Therapy | NCT00185965 |
| Advanced or metastatic tumor combined with COVID-19 | Avdoralimab + Monalizumab + GNS651 (autophagy inhibitor) | Phase II | Combination Therapy | NCT04333914 |
| Nonspecific cancer | GSK1795091 | Phase I | TLR4 agonist | NCT02798978 |
| Anti-Cancer Agent + SHR2150 (TLR7 agonist) | Phase I/II | Combination Therapy | NCT04588324 | |
| Echopulse + PD-1 + Imiquimod (TLR7 agonist) | Phase I | Combination Therapy | NCT04116320 | |
| Durvalumab + MEDI9197 (TLR7/8 agonist) | Phase I | Combination Therapy | NCT02556463 | |
| Ipilimumab + Nivolumab + Tilsotolimod (TLR9 agonist) | Phase I | Combination Therapy | NCT04270864 | |
| NY-ESO-1 protein + Montanide ± Resiquimod (TLR7/8 agonist) | Phase I | Vaccine | NCT00821652 | |
| BMS 986178 + SD-101 (TLR9 agonist) | Phase I | Combination Therapy | NCT03831295 |
Function of several repeatedly mentioned chemical: mTOR inhibitor: Temsirolimus, Sirolimus, Rapamycin, Rapamune; autophagy/lysosome inhibitor: Hydroxychloroquine, Chloroquine. +: combination; +/−: with or without