21 May 2015. Food and Drug Administration designated an experimental treatment for uveal melanoma, a rare and aggressive form of cancer in the eye, as an orphan drug that qualifies for incentives to expedite its development. Aura Biosciences also revealed results of preclinical tests showing the ability of its lead biologic therapy code-named AU-011 to treat eye tumors in lab animals.
Uveal melanoma, also called intraocular melanoma, is a disease where cancer cells form in the middle layer of tissue in the wall of the eye known as the uvea. Like the more well-known and prevalent melanoma skin cancer, older and fair-skinned people are more likely to develop uveal melanoma. While the disease occurs infrequently, it is still the most common form of eye cancer among adults, according to National Cancer Institute. Current treatment options for uveal melanoma are radiation therapy, which runs risks of damaging the eye, or surgery to remove the eye.
Aura Biosciences is a biotechnology company in Cambridge, Massachusetts that licenses research conducted at the lab of John Schiller at National Cancer Institute, part of National Institutes of Health. Schiller and colleagues study the ability of virus-like particles that prevent or treat cancers such as cervical cancer caused by human papillomavirus.
Aura Biosciences adapts Schiller’s concept to design synthetic viral protein shells about 55 nanometers in diameter. These shells then become the mechanism for transporting therapeutic molecules, in this case that bind to the uveal melanoma tumor cells. AU-011, says the company, is designed to deliver cancer cell-killing molecules to the tumors, while leaving normal eye tissue untouched. The protein shells with molecules are injected into they eye, then activated by standard ophthalmologic lasers, such as those used in outpatient clinics.
Aura Biosciences reported findings of preclinical tests of AU-011 at McGill University in Montreal with lab rabbits that had uveal melanoma grafted on their eyes, presented last week at a meeting of the Association for Research in Vision and Ophthalmology. The team found after three AU-011 treatments, from 80 to 100 percent of the tumor cells in the animals were killed, without damaging other tissue in their retinas.
Orphan drug designation is granted by FDA for diseases affecting fewer than 200,000 people in the U.S. Drugs and biologics with orphan status qualify for tax credits covering their clinical trials, as well as exemption from normal prescription drug user fees.
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