Many scientists are now interested in studying possible gene therapies and stem cell treatments on the eyes. A few reasons why these researchers love working with the eyes include the facts that testing an eye is minimally invasive, it is easy to get at the eyes, and the structure of the eyeball is well understood by modern science.
Researchers are also keen on using the eye in their tests because they can put their formula in one eye and leave the other eye untouched. This provides a convenient and reliable control for their tests.
The influential Cell and Gene Therapy Catapult, a group organized by Parliament in the UK, told reporters that scientists are increasingly testing out different pre-clinical gene therapies and stem cell treatments using the eyes. This group estimated that 14 out of 60 UK gene or stem cell studies were done in the field of ophthalmology. Most of these tests are about three years away from actually being tested clinically.
Perhaps even more surprising is that ophthalmology is third only to cancer and neurology in terms of clinical research. At least in the UK, six gene or stem cell studies out of 57 trials were done on the eye.
This is not so shocking, however, if you know just how widespread eye diseases are nowadays. With an increasingly aging population, the rates of age-related macular degeneration (AMD), diabetic retinopathy, and other eye issues related to age have been skyrocketing. Doctors simply need a way to cure these people in a more efficient manner.
So how do these therapies actually work? In the case of stem cell therapies, scientists inject the cells into the retina. This is supposed to help rebuild areas of the eye that were affected by a disease. Gene therapy, on the other hand, tries to correct certain genetic deficiencies in the eye.
As of today, all of these ophthalmic tests are in trial phases. The only company that has a product almost on the market for genetic eye issues is called Spark Therapeutics. This Philadelphia-based company is focused on treating a certain form of retinal degeneration. The specific drug Spark Therapeutics is working on still needs more testing, but if the results are good enough for FDA approval, it could be on the market as soon as 2017.
A biotech company in London called NightstaRx is also working on ophthalmological therapies. Their most current research focuses on a certain type of progressive genetic blindness called choroideremia. NightstaRx tested one of their gene therapies for choroideremia, and it was actually shown to be very successful. The six patients who participated in this trial showed either improvement or no negative change in the eyes that received treatment. The eyes that received no testing, however, only got worse.
The American biotech RetroSense Therapeutics showed great promise for helping the degenerative eye disease retinitis pigmentosa. This disease, which affects about 100,000 Americans, occurs when the retina is so damaged that patients experience symptoms like vision loss, tunnel vision, or the inability to see in dark places. The gene therapies produced by this Michigan-based impressed biotech giant Amgen so much that they paid a cool $60 million for RetroSense. Amgen hopes that the RetroSense team will be able to formulate a perfect gene therapy to treat retinitis pigmentosa in the near future.
Many people might be surprised that stem cell treatments have not been mentioned yet. Although stem cell therapy is more newsworthy, perhaps due to its controversy, it has only been used in a few tests.
One noteworthy test using stem cells was done in the UK to cure wet AMD. Wet AMD occurs when blood vessels begin to leak underneath the retina. Researchers in Moorfields Hospital were able to successfully grow new retinal cells using stem cell therapy in two patients in 2015. In addition to the workers at Moorfields Hospital, the pharmaceutical company Pfizer and the UCL Institute of Ophthalmology were a part of this test.
All of these trials show just how prevalent gene therapies and stem cell treatments will be in the future of eye care. In the coming years, biotechs around the world are poised to release revolutionary therapies and trial results that may increase eye doctors' treatment options.