FDA Approval Brings Long Awaited Hope to Patients With Geographic Atrophy

by Jeff Todd, President + CEO at Prevent Blindness

And just like that, the landscape for vision loss and blindness has shifted dramatically! For years, millions of people around the world who are living with an untreatable and potentially blinding condition, along with their care partners, have been waiting with hopeful anticipation for a treatment that some have coined a “holy grail” of eye research. That wait is over. Late last month, the FDA approved the first ever treatment for geographic atrophy (GA), an advanced form of dry AMD (age-related macular degeneration) that impacts roughly 5 million people worldwide, 1 million of them within the U.S.

AMD is generally thought of in two forms wet (neovascular) and dry (atrophic), though we know there’s a full spectrum of stages across these conditions – including GA. Both forms aggressively attack the retina, ultimately leading to vision loss. Twenty years ago, when I began my career with Prevent Blindness, a U.S. patient advocacy organization with the mission of preventing blindness and preserving sight, there was no pharmacological treatment for either form of AMD. When we received calls from patients or their family members regarding a recent AMD diagnosis, it was heart-breaking for our team to share that there was no treatment for AMD and that they should take steps to prepare for living with vision loss, possibly leading to blindness.

That changed dramatically in 2005 when researchers inadvertently discovered that a therapy developed for the treatment of cancers, also had an unexpected side effect of slowing or stopping the progression of vision loss among some patients living with wet AMD. To say this 2005 discovery was a game changer for the wet AMD patient is an understatement. Yet, while we celebrated with the AMD community, those living with the dry form (the majority of AMD patients, in fact) were left – for 18 years – asking “what about us?”

Dan Roberts, who lives with macular degeneration and is the founder and Director of MD Support, an agency that fosters a support network and resources for those with AMD and other retina diseases, has previously shared, “The field of ophthalmic research has progressed so far in the treatment of wet AMD that we often forget that up to 85% of AMD cases are of the untreatable dry form of the disease.” Upon hearing of this new therapy for GA, he says, “Now the first and only intervention for dry AMD is here, so there’s cause, once again, to celebrate. It is not a cure, but it might allow thousands of people to maintain useable vision for the rest of their lifetimes!”

 The impact of this announcement on care partners and others with a history of GA in their family is also worth noting. As Sherry Williams, a friend and former colleague who cares for a mother with GA and subsequently lives with her own fear of developing the genetically disposed condition, shared with me, “Due to my leadership role with Prevent Blindness, I recall those early days of AMD advancement with excitement, but after seeing many generations of family members experience vision loss due to GA, I was also deeply aware that so many still lived without hope. This first-time treatment for geographic atrophy is an answer to decades of advocacy and prayers; especially grateful is my mom, recently diagnosed with GA, and the millions of others also living with it.”

So, what’s next? Well, for patients, care partners, and those working in patient advocacy, there is little more thrilling than when a first-of-its-kind therapy comes to market.  Almost as exciting is when the second…and third…are approved. Options for the patient and their provider are essential in eye care, as rarely does a single choice in treatment work for everyone. We’re in a wonderfully innovative era of eye research, so while we have waited decades for this advancement, we also know there are scientists at the National Eye Institute (NEI), at universities, and at companies all around the world working tirelessly to bring additional therapies to bear against this still insidious disease, and many others that rob people of their sight.

The pharmaceutical and biotechnology industry is continually under scrutiny, as is the FDA. Sometimes this is rightly so. But it’s important that we also applaud their role in bringing forth life-changing treatments that advance the state of our public health. Even as we ought to be discerning about what works and what doesn’t across the research processes that ultimately bring new discoveries to bear, we must continue to encourage all who are involved in the long journey from bench to bedside to engage the patient population throughout the research process – so that those still waiting for a yet-to-be-discovered treatment can someday feel the hope that those living with GA feel today.

Finally, while research advances for all eye conditions are exciting and sometimes monumental, it remains equally important to remember, as Dan mentioned, that despite current and new treatments, ultimately there remains no cure for AMD or many other eye diseases. This is why continued emphasis on the essential role of vision rehabilitation and resources for those living with vision impairment and blindness is so important. These individuals can continue to thrive and live a high quality of life, regardless of the state of their vision, provided they have access to the proper supports and resources.

This week, Prevent Blindness and dozens of our patient advocates are visiting with their members of Congress to educate them about vision and eye health, as we convene our annual Eyes on Capitol Hill patient advocacy days. As we do, we come in a celebratory spirit. We now have hope for those living with GA, and a promise for more to come. Let us celebrate this accomplishment, work to ensure it is accessible to all, and strive for further patient-centric research advances.