Last year was full of remarkable developments in vision science. Important discoveries in stem cell biology filled in key knowledge gaps, while advancements in gene therapy moved optogenetics from the realm of science fiction to reality (check out our top 10 discoveries from 2016). Here are four key developments to look forward to in 2017.
Developing Fail-Safe Stem Cells
There is still a lot that we don’t know about stem cells, but this lack of knowledge isn’t slowing down the steady march into the clinic. Around the world, experimental stem cell treatments are being tested in people living with a variety of different diseases, including age-related macular degeneration (AMD) and Stargardt disease. FFB-funded scientists, Dr. Andras Nagy, is also working on a stem cell treatment for wet-AMD. His goal is to cure AMD by delivering stem cells to the eye that are genetically engineered to release a sight-saving anti-VEGF drug. Safety is Dr. Nagy’s paramount concern, so he is developing a “fail-safe system” that works by adding a “kill switch” into each stem cell. This kill switch has the ability destroy any rogue stem cells that begin to divide uncontrollably or behave unexpectedly. Dr. Nagy hopes that his fail-safe system will ensure the safety of his new stem cell treatment, in addition to the many other experimental treatments that are currently under development.
Testing Gene Editing to Treat Inherited Blindness
In 2015, the biotech start-up, Editas Medicine, boldly proclaimed that they would begin clinical trials to test gene editing as a treatment for inherited blindness in 2017. While some naysayers scoffed at this quick timeline, the company has been steadily driving CRISPR gene-editing closer to the clinic. I hope the company hits its ambitious clinical target; we’ll be following closely and will keep you updated. Last year, we wrote a lot about CRISPR, which has been hailed as a game-changing tool. I predict that there will be lots of new CRISPR news in 2017, especially because a number of FFB-funded scientists are using CRISPR in their research.
Growth of Regenerative Medicine
In Toronto, the Regenerative Medicine industry finished off with a bang with the launch of BlueRock Therapeutics, which will use innovative stem cell technologies to cure a range of diseases. $225 million was jointly invested by Bayer and the biotech venture-capital firm Versant Ventures to create BlueRock. I was incredibly fortunate to attend the official launch, where the energy was palpable. Looking around the room, I was thrilled to see top FFB-funded vision researchers like Dr. Valerie Wallace and clinical leaders (and past Vision Quest chairs) Dr. David Wong and Dr. Michael Brent. The future of regenerative medicine has never been more bright or so close to home! The creation of BlueRock Therapeutics is a truly pivotal event that is essential for making regenerative medicine a reality for patients. Initially, BlueRock will be focusing on developing therapies for heart disease, but blinding eye diseases are on the horizon.
Making gene therapy mainstream
Gene therapy has been in the works for a long time. This year, it is poised to break into the mainstream. Companies like Spark Therapeutics are in the final regulatory stages of making the first ocular gene therapy an approved treatment. We were fortunate to have some leaders from Spark at our Vision Quest education event this past November and although there was still confidential information that couldn’t yet be shared, we will keep you up-to-date as they continue to pioneer the uncharted path to make gene therapy accessible to the patients who need it. (To learn more about clinical trials, read this.) We will also continue to follow the innovative gene therapy trial that is using optogenetics to restore vision. This experimental treatment for late-stage retinitis pigmentosa (RP) uses genes from light-sensing algae to control nerve cells in the eye to restore sight. Shortly after the clinical trial was initiated by the small start-up company, Retrosense, it was bought by Allergan, which shows that large pharmaceutical companies are interested in making gene therapy a reality for patients. Stay-tuned for updates on these trials and make sure that you are enrolled on the Foundation Fighting Blindness Patient Registry. The FFB Patient Registry is a database that connects people living with blinding eye diseases to new experimental treatments, like Canada’s first ocular gene therapy. As we discover more blindness-causing genes and how they work, I predict that gene-therapy approaches to treating blindness will proliferate.
Predicting the unpredictable?
For me, it is fun to predict some of the exciting developments that might happen in 2017, but I also know that there is an inherent unpredictability to science. At its core, science probes into the unknown with the goal of discovering something new. This is why scientists are often hesitant to predict the next big discovery – they have a deep understanding of the gaps in our knowledge and a respect for the complexity of biology, including vision. With that in mind, our goal is to be both optimistic and realistic about what is possible and probable in both the short-term and the long-term.
Although many vision science mysteries remain, we believe that investing in research is the key to making sight-saving treatments a reality. Thank you to all of the FFB donors for driving vision research forward! We are excited about the next big developments (both predicted and unpredicted) in 2017.
I also spoke about these predictions and more in a recent Facebook Live session: