Table 1: List of promising compounds in clinical trials and FDA-approved for treatment of SCD and/or cancer.
Compound Name | Target | Function | Outcomes | Phase of Clinical Trial | Disease |
Hydroxyurea | b-globin gene | Up-regulates HbF expression | Reduces the frequency of vasoocclusive episodes | FDA-approved | SCD |
SCD-101 | HbS | Anti-sickling | Alleviates Chronic pain and fatigue | Phase 1b | SCD |
Aes-103 | HbS | Anti-sickling | Modified Hb structure | Phase 1 | SCD |
GBT440 | HbS | Anti-sickling | Enhances HbS oxygen affinity | Double-Blind phase 3 | SCD |
Rivipansel(GMI-1070) | Selectins | Pan-selectin inhibitor | Reduces Duration of acute pain crises, and opioid use | Randomized double-Blind phase 3 | SCD |
Crizanlizumab (SEG101) | P selectin | Anti-P selectin monoclonal antibody | Lowers pain crises | Phase 2 | SCD |
RDEA119/BAY 869766 | MEK1/2 | MEK1/2 inhibitor | Well-tolerated with some clinical benefit across a range of tumor types | Phase 1 | Advanced metastatic solid tumor |
Trametinib | MEK1/2 | MEK1/2 inhibitor | Melanoma treatment | FDA-approved | BRAF-mutant melanoma |
CI-1040 | MEK1/2 | MEK1/2 inhibitor | No clinical activity | Phase 2 | NSCLC, colon pancreatic, and breast cancers |
PD0325901 | MEK1/2 | MEK1/2 inhibitor | No clinical activity | Phase 2 (terminated) | Advanced breast and colon cancer, and melanoma |
Selumetinib (AZD6244) | MEK1/2 | MEK1/2 inhibitor | No significant antitum or activity | Phase 2 (terminated) | Breast cancer |
GDC-0973 (XL-518,RG7421) | MEK1/2 | MEK1/2 inhibitor | Partially good responses | Phase 2 | BRAF-mutated both melanoma and pancreatic cancer, and breast cancer |
Pimasertib (MEK162) | MEK1/2 | MEK1/2 inhibitor | Phase 2 | N-Ras-mutated cutaneous melanoma, and ovarian cancer | |
AZD8330 | MEK1/2 | MEK1/2 inhibitor | Phase 1 | Advanced malignancies | |
RO5126766 | MEK1/2 | MEK1/2 inhibitor | Phase 1 | Advanced NSCLC |
Drug's named and their targets, function, clinical benefits, and their phase in clinical trial for SCD and/or cancer treatments are presented.