The FDA has approved a groundbreaking gene therapy for macular telangiectasia type 2 (MacTel), a rare eye disease that leads to progressive vision loss. This approval introduces the first and only implantable therapy utilizing cell technology to deliver a protein that helps slow the disease’s progression. The gene therapy, branded as Encelto, represents a significant advancement in the treatment of MacTel and offers hope for patients struggling with this condition.
MacTel is a slowly progressing eye disease that damages the macula, which is the central part of the retina responsible for sharp vision necessary for everyday tasks like reading, writing, driving, and recognizing faces. Over time, the light-sensitive cells in the macula deteriorate, and the blood vessels supplying this area become swollen and enlarged, leading to gradual vision loss. As the disease progresses, the affected individual’s ability to perform routine activities can be severely impacted, and untreated patients may eventually lose their central vision entirely.
The newly approved treatment, Encelto, involves a tiny implant that contains lab-grown retinal cells. This implant is surgically placed in the eye, where it continuously releases a protein called ciliary neurotrophic factor (CNTF). CNTF plays a key role in promoting the health of the retinal cells and slowing their degeneration. By delivering this protein directly to the macula, Encelto aims to slow the progression of vision loss in patients with MacTel.
This approval marks a significant milestone in the treatment of MacTel, as it is the first therapy to target the disease at the cellular level. The treatment offers a more long-term solution, reducing the need for frequent injections or other interventions. Traditional treatments for MacTel have been limited in their effectiveness, and many patients have been left with few options. Encelto provides a new hope for patients who have been struggling to manage their condition.
The approval of Encelto was based on the results of two phase III clinical trials, which demonstrated that the implant successfully slowed the loss of light-sensitive macular cells in patients over the course of two years. The studies showed that patients who received the implant had better preservation of their central vision compared to those who did not receive the treatment. These findings underscore the potential of Encelto to make a significant difference in the lives of people with MacTel.
The implant is expected to be available in the United States by June, providing patients with a new treatment option. However, like all medical treatments, Encelto comes with potential side effects. Common side effects include red or itchy eyes, eye pain, dry or irritated eyes, trouble seeing in the dark, blurry vision, and eye discharge. Patients may also experience inflammation or swelling inside the eye, as well as issues with stitches, small pupil size, and headaches. In some cases, the implant may cause worsening or new cataracts, or the appearance of floating spots in the vision. These side effects are generally mild, but patients should be aware of them before receiving the implant.
Before undergoing the procedure, patients should inform their doctor about any history of eye infections, as these could increase the risk of complications. After receiving the implant, patients should be vigilant for any signs of discomfort or changes in their vision, such as sudden vision loss or persistent pain. If any of these symptoms occur, patients are advised to seek immediate medical attention.
Encelto’s approval provides a promising new treatment option for individuals with MacTel, offering the potential to slow the progression of the disease and help preserve vision. This therapy represents a major step forward in the treatment of rare eye diseases, and it is expected to have a lasting impact on the way MacTel is managed and treated.
