In a groundbreaking development for those affected by macular telangiectasia (MacTel) Type 2, the US Food and Drug Administration (FDA) has approved ENCELTO, the first-ever treatment for this rare and progressive retinal eye disease.
MacTel Type 2, which leads to gradual deterioration of central vision, has had a profound impact on the lives of its sufferers. ENCELTO offers hope as it is designed to slow disease progression and preserve central vision.
The approval of ENCELTO represents nearly two decades of dedication and research spearheaded by Professor Ian Constable AO and the Lions Eye Institute, in collaboration with their global research partners. As a major step toward improved patient outcomes, this achievement underscores the incredible work of Professor Constable and the significant role played by the Institute’s Lions Vision Trials group.
The development of ENCELTO and Lions Vision Trials’ role
Professor Ian Constable, an ophthalmology pioneer, founder and Patron of the Lions Eye Institute, has been deeply involved in advancing treatments for MacTel Type 2 as a Board member of the Lowy Medical Research Institute. His involvement over 20 years, alongside the Lions Eye Institute team, has been significant in the global efforts to develop ENCELTO.
“We have been fortunate to work alongside some of the brightest minds in ophthalmology and clinical trials,” says Professor Constable. “The long-term effort of our Lions Vision Trials team, retinal doctors, and clinic staff has been exceptional in supporting the development of treatments that can change lives.”
The Lions Eye Institute’s contribution to the ENCELTO clinical development has been significant.
Over the course of many years, we provided 20 of our patients with the novel implant. Additionally, we enrolled 143 patients into the MacTel Registry, which will provide crucial data on this disease. The Lions Eye Institute was consistently ranked as one of the highest recruiting sites worldwide. By way of our clinical trials, we are able to continue to support global research collaborations and provide our patients with early access to new, potentially life changing treatments for eye diseases.
The science behind ENCELTO
ENCELTO is an innovative surgical implant designed to release a therapeutic molecule directly into the retina, offering hope to those battling the devastating effects of MacTel. The implant works
by releasing ciliary neurotrophic factor, a molecule known to help slow the degeneration of retinal cells and preserve central vision. As a result, the therapy holds the potential to significantly enhance the quality of life for patients struggling with MacTel Type 2, which leads to loss of central vision – a crucial aspect of daily activities such as reading, driving, and working.
For patients like Kerry, who has been living with MacTel Type 2, the development of ENCELTO has brought hope where there was once uncertainty. Kerry, who participated in the MacTel2 Phase 3 clinical trial, describes her experience with the trial and the life-changing impact it has had.
“I was gutted when I was first diagnosed, as my mother had macular degeneration, and I feared I was facing the same fate,” Kerry recalls. “The trial gave me hope. The implant has been a game changer. It may not have reversed the damage, but it has slowed the progression. I’m incredibly grateful for the opportunity to be part of this clinical trial.”
The future of MacTel treatment and beyond
With the FDA approval of ENCELTO, the next step is to ensure access for patients around the world. While the approval marks a significant milestone, the treatment’s availability in Australia is contingent upon the review and approval by the Australian Therapeutic Goods Administration (TGA). If the TGA approves the therapy, it could soon be available to Australian patients, offering a new hope for those struggling with MacTel Type 2.
“We are only at the beginning of this journey,” says Professor Constable. “The knowledge gained from our research into MacTel will help us better understand other retinal diseases and lead to even more breakthroughs. The collaboration between patients, researchers, and clinicians is vital to the success of our ongoing efforts.
Kerry: A personal story of hope and perseverance
Kerry, a Lions Eye Institute clinical trials participant who enjoys playing sudoku and doing jigsaw puzzles in her spare time
Kerry was in her late 50s when she noticed that she was having trouble reading and doing computer work and she went to her optometrist for a check up. “When I covered my left eye to look at the eye chart, I realised my right eye couldn’t see all the letters on the chart! Parts of my central vision in that eye were completely missing. I could see everything else in the room around me but not what I was directly looking at”.
Kerry was referred on to Professor Chandra Balaratnasingam, an ophthalmologist at the Lions Eye Institute. “I was living in Kalgoorlie at the time and after three to four weeks I had an appointment at the Lions Eye Institute in Perth. Following a series of tests over a few hours I was diagnosed with MacTel Type 2 in both my eyes – a rare disease that has no cure or treatment”, Kerry said. “When Professor Balaratnasingam mentioned there was a clinical trial happening for a new treatment under the leadership of Professor Constable, I jumped at the chance to participate. My mother had macular degeneration and I knew straight away what I was likely to be facing and was concerned for my future”, said Kerry.
Kerry was fearful of the challenges that lay ahead so she started typing on her computer about the things she was grateful for and how she had faced other challenges throughout her life. She ended up writing a biography and had it printed into a book for her family and friends. “Writing about my feelings was very therapeutic and it helped me to accept this unexpected dilemma,” she said.
Half of the participants in the clinical trial received the real test implant in one of their affected eyes and the other half of the participants received a placebo in one of their affected eyes. Kerry now knows that she received the real implant in her right eye (not a placebo) as part of her participation in the trial and is very grateful for the opportunity to preserve her vision in that eye.
In her spare time Kerry likes doing jigsaws, crosswords and sudoku puzzles, so she remains hopeful that approval of the implants in Australia will occur soon so she can have an implant placed in her left eye before the vision in that eye deteriorates further and it’s too late.
“I’m so thankful to the Lions Eye Institute, the Lowy Institute and to Professor Constable for all their support,” Kerry says.
