Retinal stroke: research models, targets and experimental drugs
Expert Opin Investig Drugs. 2023 Aug 31. doi: 10.1080/13543784.2023.2254688. Online ahead of print.ABSTRACTINTRODUCTION: Retinal artery occlusion (RAO), often caused by a microembolus and resulting in inner retinal ischemia, could be considered as the retinal analogue to cerebral stroke. Although several therapeutic targets have been suggested in animal models of retinal ischemia and several potential treatments have been evaluated on small series of patients, central retinal artery occlusion (CRAO) is still rarely treatable in clinical practice.AREAS COVERED: Here, we review several animal models of RAO, including increas...
Source: Expert Opinion on Investigational Drugs - August 31, 2023 Category: Drugs & Pharmacology Authors: Alejandra Daruich Matthieu P Robert Marta Zola Alexandre Matet Dominique Bremond-Gignac Source Type: research

CNM-Au8: An experimental agent for the treatment of amyotrophic lateral sclerosis (ALS)
Expert Opin Investig Drugs. 2023 Aug 29. doi: 10.1080/13543784.2023.2252738. Online ahead of print.ABSTRACTINTRODUCTION: The two established disease-specific therapies for the treatment of amyotrophic lateral sclerosis (ALS) are riluzole and edaravone. Limitations of these medications include minimal progression slowing or survival benefit, and effectiveness in only select populations, particularly for edaravone. AMX0035 and tofersen received US FDA approval in September 2022 and April 2023, respectively. However, phase 3 trials further examining both medications' efficacy are ongoing. CNM-Au8 is an efficient catalyst of e...
Source: Expert Opinion on Investigational Drugs - August 29, 2023 Category: Drugs & Pharmacology Authors: J Daniel Bireley John A Morren Source Type: research

Current status and future expectations of nanobodies in oncology trials
Expert Opin Investig Drugs. 2023 Aug 28:1-17. doi: 10.1080/13543784.2023.2249814. Online ahead of print.ABSTRACTINTRODUCTION: Monoclonal antibodies have revolutionized personalized medicine for cancer in recent decades. Despite their broad application in oncology, their large size and complexity may interfere with successful tumor targeting for certain applications of cancer diagnosis and therapy. Nanobodies have unique structural and pharmacological features compared to monoclonal antibodies and have successfully been used as complementary anti-cancer diagnostic and/or therapeutic tools.AREAS COVERED: Here, an overview is...
Source: Expert Opinion on Investigational Drugs - August 28, 2023 Category: Drugs & Pharmacology Authors: Tessa De Pauw Lynn De Mey Jens M Debacker Geert Raes Jo A Van Ginderachter Timo W M De Groof Nick Devoogdt Source Type: research

Ataxia-telangiectasia clinical trial landscape and the obstacles to overcome
Expert Opin Investig Drugs. 2023 Aug 28:1-12. doi: 10.1080/13543784.2023.2249399. Online ahead of print.ABSTRACTINTRODUCTION: Ataxia telangiectasia (A-T) is a life-limiting autosomal recessive disease characterized by cerebellar degeneration, ocular telangiectasias, and sinopulmonary disease. Since there is no cure for A-T, the standard of care is primarily supportive.AREAS COVERED: We review clinical trials available in PubMed from 1990 to 2023 focused on lessening A-T disease burden. These approaches include genetic interventions, such as antisense oligonucleotides, designed to ameliorate disease progression in patients ...
Source: Expert Opinion on Investigational Drugs - August 25, 2023 Category: Drugs & Pharmacology Authors: Katrina Kuhn Howard M Lederman Sharon A McGrath-Morrow Source Type: research

Ataxia-telangiectasia clinical trial landscape and the obstacles to overcome
Expert Opin Investig Drugs. 2023 Aug 25. doi: 10.1080/13543784.2023.2249399. Online ahead of print.ABSTRACTINTRODUCTION: Ataxia telangiectasia (A-T) is a life-limiting autosomal recessive disease characterized by cerebellar degeneration, ocular telangiectasias, and sinopulmonary disease. Since there is no cure for A-T, the standard of care is primarily supportive.AREAS COVERED: We review clinical trials available in PubMed from 1990-2023 focused on lessening A-T disease burden. These approaches include genetic interventions, such as antisense oligonucleotides, designed to ameliorate disease progression in patients with sel...
Source: Expert Opinion on Investigational Drugs - August 25, 2023 Category: Drugs & Pharmacology Authors: Katrina Kuhn Howard M Lederman Sharon A McGrath-Morrow Source Type: research

Experimental drugs for the prevention or treatment of sensorineural hearing loss
Expert Opin Investig Drugs. 2023 Jul-Dec;32(7):643-654. doi: 10.1080/13543784.2023.2242253.ABSTRACTINTRODUCTION: Sensorineural hearing loss results in irreversible loss of inner ear hair cells and spiral ganglion neurons. Reduced sound detection and speech discrimination can span all ages, and sensorineural hearing rehabilitation is limited to amplification with hearing aids or cochlear implants. Recent insights into experimental drug treatments for inner ear regeneration and otoprotection have paved the way for clinical trials in order to restore a more physiological hearing experience. Paired with the development of inno...
Source: Expert Opinion on Investigational Drugs - August 20, 2023 Category: Drugs & Pharmacology Authors: Judith S Kempfle David H Jung Source Type: research