A New Clinical Trial: Squalamine Eye Drops for Wet AMD and Retinal Vein Occlusion
by Maureen Duffy
Ohr Pharmaceutical, Inc. is a company that is dedicated to the clinical development of new drugs for underserved therapeutic needs. One of its two lead drugs in current development is Squalamine Eye Drops for the treatment of wet age-related macular degeneration (AMD).
What is Squalamine?
Squalamine is a water-soluble molecule derived from the internal organs (primarily the liver) of the dogfish shark. It is believed to have great potential for treating some human viruses. Medically, squalamine has been shown to interrupt and reverse the process of angiogenesis.
About Angiogenesis and Anti-Angiogenic Drugs
Angiogenesis is a term that describes the growth of new blood vessels and plays a critical role in the normal development of body organs and tissue. Sometimes, however, excessive and abnormal blood vessel development can occur in diseases such as cancer (tumor growth) and AMD (retinal and macular bleeding).
Substances that stop the growth of these excessive blood vessels are called anti-angiogenic (anti=against; angio=vessel; genic=development), and anti-neovascular (anti=against; neo=new; vascular=blood vessels).
The focus of current anti-angiogenic drug treatments for wet macular degeneration is to reduce the level of a particular protein (vascular endothelial growth factor, or VEGF) that stimulates abnormal blood vessel growth in the retina and macula; thus, these drugs are classified as anti-VEGF treatments.
A History of Squalamine Research
Squalamine Eye Drops for Wet AMD
Initially, the Genaera Corporation developed the squalamine project and administered squalamine as an intravenous formulation for wet AMD in Phase 1 and Phase 2 clinical trials. However, in 2007, Genaera discontinued their clinical trials, due to financial and subject-recruitment issues. The squalamine project was then acquired by Ohr Pharmaceutical, which continued the development of squalamine in a topical eye drop formulation.
Fast Track is a process designed to facilitate the development, and expedite the review, of drugs to treat serious diseases and fill an unmet medical need. Filling an unmet medical need is defined as providing a therapy where none exists or providing a therapy which may be potentially superior to existing therapy.
In August 2012, the FDA commenced a Phase II clinical trial to study the safety and effectiveness of Squalamine Eye Drops. The clinical trial is in the process of enrolling 120 participants at 20 clinical ophthalmology centers across the United States, who will be treated with Squalamine Eye Drops twice daily for a nine-month period. You can learn more about the squalamine clinical trial, including criteria for enrollment and the locations of the 20 clinical trial study centers, at Efficacy and Safety of Squalamine Eye Drops at ClinicalTrials.gov.
The squalamine researchers theorize that the ability to self-administer eye drops to treat wet AMD may be more effective (and less invasive) than the current standard of care, which involves regular injections of Lucentis, Avastin, or Eylea directly into the eye, via a very small needle.
Squalamine Eye Drops for Retinal Vein Occlusion
On August 20, 2013, Ohr announced the initiation of an investigator-sponsored trial to evaluate the safety and effectiveness of Squalamine Eye Drops for the treatment of macular edema (i.e., a swelling of, or accumulation of fluid in, the macula), secondary to retinal vein occlusion. The trial (Study OHR-004) is being conducted by Dr. John Wroblewski at Cumberland Valley Retina Consultants in Maryland and is expected to enroll 20 patients. Dr. Wroblewski recently joined Ohr Pharmaceutical's scientific advisory board.
A retinal vein occlusion is a blockage, or "occlusion," of the small veins that carry blood away from the retina, the light-sensitive tissue that lines the inside surface of the eye. The retina converts incoming light into nerve signals and sends them to the brain, which interprets them as visual images.
Blockage of smaller, "branching," veins (branch retinal vein occlusion or BRVO) in the retina often occurs when retinal arteries that have been thickened or hardened by atherosclerosis "cross over" and place pressure on a retinal vein. A branch retinal vein occlusion is sometimes referred to as "a stroke on the retina."
Patients in this investigator-sponsored trial will receive Squalamine Eye Drops for the first two weeks of treatment. They will then receive two successive injections of Lucentis four weeks apart, while continuing treatment with the eye drops. At week 10 of the study, patients will be randomly assigned to one of two study groups: (a) continue with Squalamine Eye Drops or (b) discontinue the eye drops for the remainder of the 38-week study. "Rescue injections" of Lucentis will be administered as needed though week 38.
About Clinical Trials and Investigator-Sponsored Trials
FDA-Sponsored Clinical Trials
The U.S. Food and Drug Administration (FDA), part of the U.S. Department of Health and Human Services, is responsible for "... helping the public get the accurate, science-based information they need to use medicines and foods to maintain and improve their health." Most FDA-sponsored clinical trials are designated as Phase I, II, or III, based on the questions the study is seeking to answer.
Clinical trials sponsored by pharmaceutical, biotechnology, and medical device companies are called "industry sponsored trials." In a trial in which the principal investigator serves as the sponsor for his or her own study, the investigator becomes the "sponsor-investigator." A sponsor-investigator is anyone who functions as the clinical investigator of a given study and who also holds the investigational marketing application.
You can read more about Investigator initiated trials at the the U.S. National Library of Medicine, National Institutes of Health. VisionAware will provide updates on this important research as they become available.
Branch retinal vein occlusion photo is a Wikimedia Commons file, used in accordance with the Creative Commons Attribution 2.0 Generic license
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