Table I.
Novel CNS Drugs in Clinical Trials (
Source: Patel & Patel, 2017)
| s/n | Drug candidate | Mechanism | Indication | Findings |
|---|---|---|---|---|
| 1 | Sorafenib + everolimus | Tyrosine kinase inhibitor |
Brain tumor Glioblastoma Anaplastic glioma |
Maximum dose tolerated to be 200 mg twice daily |
| 2 | Sunitinib (high-dose, intermittent) | Tyrosine (multiple) kinase inhibitor | Recurrent GBM | Promising outcomes if intermittent dosage of 300 mg compared to 5 0 mg of previous clinical trial is well tolerated |
| 3 | GnbAC1 | Humanized IgG4 mAb targeting retroviral envelope | Relapsing remitting multiple sclerosis | No clear immunoregulatory effect in MS but showed remyelinating potential. Study showed safety and good tolerance of drug |
| 4 | Vandetanib + temozolomide; vandetanib + carboplatin | EGFR and VEGF receptor 2 inhibitor | GBM | Results unclear; well tolerated but efficacy not ascertained, study terminated |
| 5 | ABT-436 | Vasopressin 1b receptor antagonist | Alcohol dependence | Greater percent days of abstinence than placebo group |
| 6 | Peptide receptor antagonist | Acute migraine | Pain freedom within 2 h of 50 and 25 mg; further research needed to determine long-term safety | |
| 7 | SAGE-217 | Positive allosteric modulator of GABA type A receptor | Major depressive disorder | Administered for 14 days resulted in reduction in depressive symptoms for 15 days but with more adverse events |
| 8 | Rimegepant | Calcitonin gene-related peptide receptor antagonist | Migraine | High percentage of patients free from pain and most bothersome symptoms |
| 9 |
Anlotinib With STUPP Regimen |
Inhibit both tumor angiogenesis and proliferation | Newly diagnosed and recurrent glioblastoma | |
| 10 | Aducanumab (BIIB037) | Human IgG1 monoclonal antibody against a conformational epitope found on Aβ | Alzheimer’s disease | Potentially beneficial being an immune checkpoint inhibitor |
| 11 | Selumetinib | Blocks proteins that allow tumor cells grow without stopping | Astrocytoma low-grade glioma | |
| 12 | Selumetinib | Blocks proteins that allow tumor cells grow without stopping | Neurofibromatosis type 1 and symptomatic inoperable plexiform neurofibromas | |
| 13 | Inotuzumab ozogamicin | Monoclonal antibody, linked to anti-cancer calicheamicin |
Acute lymphoblastic leukemia CNS leukemia |
Improvements in PN-related pain and motor impairment, durable tumor shrinkage |
GBM glioblastoma multiforme; AD Alzheimer’s disease; EGFR epithelia growth factor receptor; VEGF vascular endothelial growth factor, mAb monoclonal antibody