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.