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Macular degeneration treatments from Europe

Investigational treatments
Investigational treatments for age-related macular degeneration (AMD) are therapies that are in various stages of development but have not yet been approved by the FDA for use in the United States, though they might be available in other countries. In some cases, people in the U.S. can receive these treatments prior to FDA approval, especially if they are enrolled in a clinical trial helps determine safety and effectiveness of the medication, surgery or other therapy.
Eylea and PDGFR-beta combination
In January 2014, Regeneron Pharmaceuticals and Bayer HealthCare announced an agreement to jointly develop an innovative antibody to the Platelet-Derived Growth Factor Receptor Beta (PDGFR-beta) as a potential combination therapy with Regeneron’s FDA-approved Eylea (aflibercept) injections for the treatment of wet age-related macular degeneration (wet AMD).
According to the companies, preclinical data suggest that blocking PDGFR-beta as well as vascular endothelial growth (VEGF) can provide treatment advantages over inhibiting VEGF alone with Eylea or other FDA-approved treatments. Human clinical studies of the combination therapy are planned to begin in early 2014.
Avastin
Some eye doctors are using Avastin (bevacizumab), an FDA-approved cancer medication produced by Genentech, as an “Off Label” treatment for Macular Degeneration. (While Genentech markets Avastin for treatment of colorectal cancer, the company has announced it has no plans to place the drug in clinical trials for treatment of macular degeneration.)
When used off label to treat AMD, Avastin, like FDA-approved macular degeneration drugs Macugen (Eyetech) and Lucentis (Genentech), is injected directly into the vitreous in the back of the eye.
In all treatment groups in the study, at least 60 percent of patients attained visual acuity that met or exceeded the legal standard for driving vision (20/40 or better).
Some eye care practitioners choose to use Avastin off label for AMD treatment, because costs to patients may be significantly lower than when using Lucentis, which received FDA approval as a macular degeneration treatment in 2006.
iSONEP
Currently in early stage clinical trials, this novel treatment for “wet” AMD involves injections of a therapeutic antibody into the eye. Being developed by the biotechnology company Lpath, iSONEP (sphingomab) appears to target abnormal blood vessel growth in AMD as well as inflammation and excessive formation of fibrous tissue.
In December 2010, Lpath announced it had granted Pfizer a global license for commercial development and future sales of the drug pending appropriate studies and regulatory approvals.
“This study provides additional scientific evidence supporting our theory that restoring blood flow in the choroid will have a positive effect on preventing the progression of this terrible disease,” said MacuCLEAR CEO and President Philip G. Ralston Jr.
Evizon
Known as squalamine, the drug has demonstrated ability to prevent development of abnormal blood vessels that damage the eye in wet AMD. Company officials say the drug’s safety profile is a major advantage, and researchers are developing ways to enhance the drug’s properties.
Zybrestat
In June 2010, Oxigene announced encouraging preclinical data regarding development of its investigational Zybrestat treatment for wet AMD. Unlike other macular degeneration drugs, Zybrestat is being designed for topical use in the form of eye drops and could eliminate the need for eye injections. The company said it believes the topical formulation could be ready for clinical development in early 2011.
In December 2011, the company presented encouraging data from a phase 2 study of Zybrestat for the treatment of polypoidal choroidal vasculopathy (PCV) — a potentially blinding eye disease that involves the growth of tiny, abnormal blood vessels under the retina. Oxigene’s CEO said Zybrestat or a similar topical compound might one day become a “major breakthrough in treating retinal diseases,” and that the company is seeking “a partner with strong expertise and experience in ophthalmic drug development to advance this promising clinical program.”

Gene Therapy
Research into gene therapy as a potential treatment for macular degeneration is in early stages. But investigators are searching for ways to introduce specially encoded genes that could alter processes causing macular degeneration.

Stem Cells
In what coul be a milestone for treatment of dry AMD, Advanced Cell Technology (ACT) received approval in the U.S. and Europe in 2011 to begin clinical trials to investigate the safety and effectiveness of using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells for the treatment of dry macular degeneration.

In January 2012, ACT announced the first patient in Europe received the treatment, and the following month the company announced three patient in the U.S. had been treated. Three trials will be conducted in the U.S. and Europe, with 12 patients enrolled in each trial. Preliminary results are expected in early 2013.
In February 2012, another company, StemCells Inc., announced it will begin FDA-approved phase 1/2 clinical trials to evaluate the safety and preliminary effectiveness of the company’s purified human neural stem cell treatment for dry AMD.
The company plans to enroll 16 patients and the stem cells will be administered by a single injection into the space beneath the retina. Patients will be examined at predetermined intervals over 1 year and then will be followed for an additional 4 years in a separate observational study.
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