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1rem

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Professor and Chair of Ophthalmology at Mayo Clinic

ASRS 2025: A post-hoc analysis of the effect of baseline EZ layer loss on treatment outcomes in MacTel

Retina World Congress 2025 will take place May 8-11. As researchers prepare for their, presentation, we are highlighting the upcoming sessions that are scheduled for this meeting, which will be held in Fort Lauderdale, Florida.

Note: The following list of presentations are in alphabetical order by company. Updates will be added to this article as new information becomes available.

The company highlight Phase 2 results for migaldendranib (MGB), Ashvattha’s investigational subcutaneous treatment for wet age-related macular degeneration (wet AMD) and diabetic macular edema (DME). The presentation, entitled, "Emerging Data om Anti-angiogenic Nanomedicine (Migaldendranib MGB/D-4517.2) for AMD and DME" will be given by Michael Singer, MD on Sunday, May 11, 2025; 11:11 – 11:16 am ET.

Phase 2 clinical data demonstrated that MGB administration lowered production of subretinal fluid reducing or eliminating the need for intravitreal anti-VEGF treatment in both the study eye and fellow eye in wet AMD and DME subjects with bilateral disease. New data indicates that MGB may be the first safe systemic therapy for the treatment of wet AMD and DME without ocular adverse events.

Huma Qamar, Chief Medical Officer at Ocugen will represent Ocugen at Retina World Congress to share a presentation and join retinal surgeons and industry peers during a panel discussion.

Inherited and Rare Retinal Diseases Session
Moderated by Rishi P. Singh, MD, FASRS and Kourous A. Rezaei, MD on Thursday, May 8, 2025, 10:35 – 11:12 AM ET

Ocular Therapeutix will present information at the meeting in Fort Lauderdale regarding the axitinib intravitreal hydrogel clinical trials, the use of its candidate OTX-TKI for diabetic retinopathy, and the HELIOS clinical trial for diabetic retinopathy.

Axitinib Intravitreal Hydrogel: Update on Clinical Trials

Presented by Carl C. Awh, MD, FASRS, on Friday, May 9, 2025, 4:33 – 4:38 PM ET.

Presented by Dilsher S. Dhoot, MD, FASRS on Saturday, May 10, 2025, 8:44 – 8:49 AM ET

Volumetric Macular Fluid Analysis of the Impact of a Single Axitinib Intravitreal Injection (OTX-TKI) from the HELIOS Clinical Trial for Diabetic Retinopathy

Presented by Carl J. Danzig, MD on Saturday, May 10, 2025, 8:52 – 8:57 AM ET

The company will present further insights from Stage 1 of the Phase 3 DIAMOND program reinforcing the efficacy and safety profile of OCS-01 eye drops for DME will also be shared. OCS-01 is being developed to be the first non-invasive treatment for DME, addressing key unmet needs for earlier treatment intervention and for inadequate responders to the current standard of care.

Ashvattha Therapeutics to Present at ARVO, Retina World Congress, and AOS Annual Meeting. Ashvattha Therapeutics. April 15, 2025. Accessed April 16, 2025. https://www.globenewswire.com/news-release/2025/04/15/3061682/0/en/Ashvattha-Therapeutics-to-Present-at-ARVO-Retina-World-Congress-and-AOS-Annual-Meeting.html

Ocugen to Present on Modifier Gene Therapy Platform at Association for Research in Vision and Ophthalmology 2025 Annual Meeting and Retina World Congress. Ocugen Inc. April 29, 2025. Accessed April 30, 2025. https://www.globenewswire.com/news-release/2025/04/29/3070074/0/en/Ocugen-to-Present-on-Modifier-Gene-Therapy-Platform-at-Association-for-Research-in-Vision-and-Ophthalmology-2025-Annual-Meeting-and-Retina-World-Congress.html

Ocular Therapeutix™ to Participate in Upcoming Investor and Scientific Conferences. Ocular Therapeutix. April 29, 2025. Accessed April 29, 2025. https://www.globenewswire.com/news-release/2025/04/29/3070022/0/en/Ocular-Therapeutix-to-Participate-in-Upcoming-Investor-and-Scientific-Conferences.html

Oculis to Present its Innovative Late-Stage Pipeline Candidates at Upcoming Ophthalmology Conferences. Oculis Holding AG. May 1, 2025. Accessed May 1, 2025. https://www.biospace.com/press-releases/oculis-to-present-its-innovative-late-stage-pipeline-candidates-at-upcoming-ophthalmology-conferences

Companies working in the retina space have announced that data from clinical trials will be shared at this year’s meeting in Fort Lauderdale, Florida.

RWC 2025: Companies to share data at meeting in Fort Lauderdale

A new study from investigators in Romania examined the incidence rates of retinoblastoma, the need for enhanced screening, treatment options, and long-term prognosis for children with this inherited disease in northeastern Romania. Enhanced screening is critical because of the advanced stage of retinoblastoma at presentation.

 The investigators, led by first author Vlad Constantin Donica, PhD, are from the Department of Ophthalmology, Faculty of Medicine, Grigore T. Popa University of Medicine and Pharmacy, and the Department of Ophthalmology, Sf. Spiridon Emergency County Hospital, both in Iasi, Romania.

Based on the stage of the disease, the clinical signs can range from small (under 2 mm) intraocular tumors, which look like transparent retinal spots, to larger tumors with vessels and white calcified lesions; a large tumor can occupy the entire vitreous cavity and touch the lens, investigators explained.

Leukocoria, which can be present with all forms of retinoblastoma and is the most common sign, can vary from a subtle appearance to a completely white pupil with large tumors. Strabismus is the next most common sign, and more advanced forms can have changes in the iris color and neovascularization, an enlarged cornea and enlarged globe due to increased intraocular pressure, and proptosis with delayed presentation.

The investigators explained that retinoblastoma, the most common pediatric ocular tumor, lacks both proper genetic testing and case registration, resulting in advanced disease stages at patients’ initial presentation.

Donica and colleagues cited results of previous studies that showed that countries in Eastern Europe had the fewest data from patients with retinoblastoma compared with Asia, Africa, Latin America, and North America. Specifically, the Automated Childhood Cancer Information System analyzed 60 pediatric oncology centers between 1978 and 1997 and identified 1995 cases of retinoblastoma from all across Europe. In Eastern Europe, only Belarus, Estonia, Slovakia, and Hungary were among the analyzed countries.

 that was part of the Surveillance of Rare Cancers in Europe assessed the incidence, prevalence, and survival rate of the main embryonal cancers in Europe and found that retinoblastoma was third in incidence and second in survival rate. From 1995 to 2002, analysis of data from the EU showed that during that period, only Poland submitted data. A 2021 report on European incidence of retinoblastoma presented incomplete data from several eastern European countries, including Romania, and reported 8 newly identified cases in 2017.

“The lack of proper data-gathering highlights the importance of reviewing the current status of patients that were diagnosed with retinoblastoma in developing countries,” Donica and colleagues commented.

The study under discussion is a 10-year retrospective analysis of patients diagnosed with retinoblastoma between 2014 and 2024 in Sf. Spiridon Hospital.

The authors identified 10 cases (7 boys, 3 girls; average patient age, 25.9 months), 9 of which had a unilateral tumor and 1 with bilateral tumors. All patients were diagnosed at presentation with clinical stage E, defined as a large tumor touching the lens, with increased intraocular pressure and an enlarged globe 

“Enucleation with successful tumor excision was performed in 6 of 7 cases. Two patients underwent conservative treatment, and one patient was unable to be reached after the diagnosis,” the investigators reported.

The 6 children who underwent successful enucleation had no tumor recurrences. They received a successful ocular prosthesis and, if required, cosmetic reconstruction. The patient with unsuccessful tumor excision had bilateral retinoblastoma that had a delayed initial presentation/diagnosis. Despite aggressive intravenous treatment and enucleation, the tumor recurred with systemic metastasis, and the patient died.

Regarding conservative treatment, 1 patient has had a history of unilateral retinoblastoma for 7 years and underwent 2 cycles of intra-arterial chemotherapy, 13 intravitreal injections, 12 sessions of cryotherapy, 8 sessions of thermotherapy, laser treatment, and cerclage in the first 4 years after the initial diagnosis. An artificial lens was implanted after lensectomy. The patient continues to be followed and treated for amblyopia. The vision currently is 20/25. The tumor remained stable.

The second patient treated conservatively underwent 1 cycle of intra-arterial chemotherapy and intravitreal injections with a favorable response. In this study, based on the American Joint Committee on Cancer, 42.85% of tumors were classified as grade G2, and 57.15% as G3. The optic disc was invaded in 71.42% of cases, with further nerve invasion in 40%. Uveal invasion was present in 57.14% of cases.

Donica and associates commented, “Our 10-year analysis of 10 cases averaged 1 case of newly identified retinoblastoma annually. These data suggest that the incidence of retinoblastoma in our region has a rate of 1:28,554, while cited data report 1:15,000 to 1:20,000 per child birth. Our lower incidence could result from the presentation of these children to other centers across the country. Therefore, the need for a unified registry for following and understanding the different varieties in pediatric cancers has never been greater.”

High-resolution ultrasound can identify lesions exceeding 2 mm and is superior to fetal magnetic resonance imaging, which is more useful for identifying extraocular tumor extension.

 Early prenatal screening can be performed invasively and noninvasively, with the latter using fetal cell-free DNA from a maternal blood sample to search for new/inherited mutations. Invasive methods include amniocentesis or chorionic villi sampling.

Future parents with a history of retinoblastoma can undergo genetic testing during in vitro fertilization, providing them with information on the 

 mutation status before embryonic implantation.

Incipient retinoblastoma detection with early delivery provides a better chance of successful treatment with possible ocular salvage, while term deliveries allow more time for tumoral growth and spread, compromising treatment effectiveness.

 “This more than justifies the necessity of prenatal genetic screening in patients with a risk for developing retinoblastoma,” the investigators said.

Despite universally available ultrasonography and prenatal screening using invasive and noninvasive methods in Romania, no study patients were diagnosed with retinoblastoma using prenatal ultrasound, they pointed out.

Optical coherence tomography (OCT) of the posterior segment is another noninvasive option.

 While OCT is routinely used to diagnose many ophthalmologic pathologies in their clinic, the lack of handheld devices for neonatal screening resulted in delayed diagnosis with advanced disease at presentation, they said.

The authors emphasized the need for registries in developing countries to best manage and monitor the incidence of retinoblastoma. “In our study, all children had an initial presentation with disease stage E, which highlights the need to intensify screening measures in infants to diagnose this disease earlier. Therefore, the lack of proper genetic testing makes us unable to report how many tumors had germline or developed somatic mutations and to provide patients adequate information regarding the risk of their offspring inheriting 

 gene mutations. Unfortunately, enucleation remains the primary treatment method for retinoblastoma in Romania, despite access to conservative eye-saving treatment measures.”

Donica VC, Costea CF, Sandu CA, Pavel IA, 

 CM. Prevalence and management of retinoblastoma. a 10-year retrospective analysis from North-Eastern Romania.

Dimaras H, Corson TW, Cobrinik D, et al. Retinoblastoma. 

MacCarthy A, Draper GJ, Steliarova-Foucher E, Kingston JE. Retinoblastoma incidence and survival in European children (1978–1997). Report from the Automated Childhood Cancer Information System project. 

Gatta G, Ferrari A, Stiller CA, et al. Embryonal cancers in Europe. 

Stacey AW, Bowman R, Foster A, et al. Incidence of retinoblastoma has increased: results from 40 European countries. 

Paquette L, Miller D, Jackson H, et al. In utero detection of retinoblastoma with fetal magnetic resonance and ultrasound: initial experience. 

Rodriguez A, Kelley C, Patel A, Ramasubramanian A. Prenatal diagnosis of retinoblastomas: a scoping review. 

Gerrish A, Bowns B, Mashayamombe-Wolfgarten C, et al. Non-Invasive Prenatal Diagnosis of Retinoblastoma Inheritance by Combined Targeted Sequencing Strategies. 

Soliman SE, Dimaras H, Khetan V, et al. Prenatal versus postnatal screening for familial retinoblastoma. 

Toi A, Sutherland J, Gallie B, Gardiner J, Sermer M. Evaluation of the fetus at risk for retinoblastoma: what is the role of prenatal ultrasound? 

Shields CL, Shields JA. Diagnosis and management of retinoblastoma. 

Shields C, Manalac J, Das C, Saktanasate J, Shields J. Review of spectral domain-enhanced depth imaging optical coherence tomography of tumours of the retina and retinal pigment epithelium in children and adults. 

Enhanced screenings and a unified registry could save vision and lives.

Canonical

clinical

Managing and tracking the worldwide burden of retinoblastoma

 caught my attention with the provocative title 

Impact of Bodyweight Loss on Type 2 Diabetes Remission: A Systematic Review and Meta-Regression Analysis of Randomised Controlled Trials.” The authors define “complete remission of diabetes” as a glycated haemoglobin level (HbA

) less than 6.0% and/or a fasting glucose level less than 100 mg/dL with no use of glucose-lowering drugs at 1 year after weight loss. They define “partial remission” as HbA

 less than 6.5% and/or fasting glucose less than 126 mg/dL with no glucose-lowering drugs, again 1 year after weight loss.

After evaluating 2847 eligible subjects in 22 randomised controlled trials for diabetes remission as a function of weight loss from baseline, the authors found that, on average across all trials, subjects with less than 10% of body weight loss achieved total remission (TR) and partial remission (PR) rates of 0.7% and 5.4%, respectively. For subjects with a 10% to 19% weight loss, PR rates were 48.4% (no studies analysed considered TR for this range of weight loss). TR and PR rates were 49.6% and 69.3%, respectively, for a 20% to 29% weight loss and 79.1% and 81.5% for subjects losing more than 30% of their baseline body weight. Of note, every 1 percentage point decrease in body weight increased the probability of TR by 2.17% and the probability of PR by 2.74%; these effects were not impacted by age, gender, race, diabetes duration, baseline body mass index (BMI), HbA

 level, insulin use or type of weight loss intervention (eg, pharmacotherapy, caloric restriction, bariatric surgery or a combination of interventions). The most interesting finding is the exponentially increased success of weight loss for purposes of diabetes remission with the degree of weight loss achieved, with a threshold weight loss greater than 10% required for meaningful population levels of remission, signalling “a robust dose-
response relationship between bodyweight loss and diabetes remission.”

Remission of diabetes should be of considerable interest to all health care providers, including optometrists, because studies show that even brief periods of remission (ie, 6 months) significantly reduce the risk of vascular diabetes complications by 11% to 35%, including major adverse cardiovascular events (eg, stroke, heart attack, congestive heart failure and cardiovascular death) as well as incidence of diabetic retinopathy (DR) and nephropathy.

 Though some studies have shown that sudden reductions in mean blood glucose levels may exacerbate preexisting retinopathy (eg, with bariatric surgery, initiation of insulin therapy or potent glucagon-like peptide [GLP-1] receptor agonist drugs such as semaglutide),

 this phenomenon, sometimes referred to as diabetic reentry retinopathy or early worsening phenomenon, is typically short-lived, and the long-term benefits of primary DR prevention via diabetes remission will almost certainly yield a massive public health benefit if remission can be sustained.

All this begs the question: How realistic is achieving diabetes remission with currently available weight loss strategies? Specifically, can sufficient weight loss be achieved to induce meaningful population-level reductions in overweight and obesity and thus type 2 diabetes prevalence? Bariatric surgery results in a greater than 10% weight loss in virtually all patients, with mean body weight reductions of 15% to 30%, depending on baseline patient characteristics and the specific procedure chosen. However, it does carry surgical risk as well as increased risk of nutrient malabsorption.

 Bariatric surgery is typically covered by insurance for type 2 diabetes only when the BMI is above 35 kg/m

 Nonetheless, roughly 60% to 70% of patients with obesity with type 2 diabetes experience remission after 5 years, including those with a BMI less than 35 kg/m

GLP-1 or dual GLP/glucose-dependent insulinotropic polypeptide (GIP) therapies typically result in 5% to 10% reductions in body weight, depending on the specific drug, but a majority of patients regain weight after drug cessation.

 The most effective currently available medication is tirzepatide (marketed as Mounjaro for type 2 diabetes and as Zepbound for overweight/obesity; Eli Lilly). Three-year data from the SURMOUNT-1 trial (NCT04184622) of 2359 subjects with obesity (40% with prediabetes) showed 12.3%, 18.7%, and 19.7% reductions in body weight with weekly tirzepatide injections of 5 mg, 10 mg, and 15 mg, respectively, compared with a 1.3% mean weight loss in the placebo group.

 Pooled analysis showed that tirzepatide increases the likelihood of type 2 diabetes remission (defined as HbA

 less than 5.7%) by 16-fold, though there is no evidence that remission is extended without ongoing drug therapy.

GLP-1 therapy is relatively expensive and has been linked to both worsening DR (especially in patients using insulin) and increased risk of nonarteritic anterior ischemic optic neuropathy, particularly with large, rapid reductions in mean blood glucose

; GLP-1s also are associated with excess muscle loss (sarcopenia). One analysis found a 30% or more loss of muscle fibre volume with semaglutide.

 Recently, the SURMOUNT-3 MRI substudy found that tirzepatide did not cause excess sarcopenia compared with that seen with conventional weight loss strategies, such as diet, exercise, and/or bariatric procedures.

What about fasting regimens? Meta-analysis comparing alternate daily fasting (ADF), 5:2 fasting (no food intake 2 days/week with a typical diet 5 days/week), caloric restriction (typically less than 750 kcal/day), and time-restricted eating (usually a “feeding window” of 8 hours) shows that each of these typically results in a 5% to 12% weight loss, with most data favoring ADF to achieve a more than 10% weight loss.

 A Chinese study of the 5:2 diet in type 2 diabetes showed superior reductions in weight loss (4 kg or 8.2 lb difference) and HbA

 level (–0.3% to 0.4% difference) compared with metformin or empagliflozin over 16 weeks, but did not report on diabetes remission.

Meaningful population-wide remission of type 2 diabetes appears to exponentially correlate with the degree of weight loss, with a 15% reduction in body weight required to achieve 1-year partial or total remission rates approaching 50%. Various strategies are available to achieve this end, with bariatric surgery being most effective, followed by potent GLP-1/GIP therapy, followed by caloric restriction/intermittent fasting, based on my review of available evidence. Primary prevention with a focus on environmental contributors to diabetes

 (eg, food insecurity, taxation policies that discourage consumption of sugar-laden foodstuffs, particulate air pollution, neighbourhood walkability, etc) is ideal, but awareness of and education about remission strategies to help patients currently living with type 2 diabetes are within our reach.

1. Kanbour S, Ageeb RA, Malik RA, Abu-Raddad LJ. Impact of bodyweight loss on type 2 diabetes remission: a systematic review and meta-regression analysis of randomised controlled trials. Lancet Diabetes Endocrinol. 2025;13(4):294-306. doi:10.1016/S2213-8587(24)00346-2

2. Dambha-Miller H, Hounkpatin HO, Stuart B, Farmer A, Griffin S. Type 2 diabetes remission trajectories and variation in risk of diabetes complications: a population-based cohort study. PLoS One. 2023;18(8):e0290791. doi:10.1371/journal.pone.0290791

3. Bain SC, Klufas MA, Ho A, Matthews DR. Worsening of diabetic retinopathy with rapid improvement in systemic glucose control: a review. Diabetes Obes Metab. 2019;21(3):454-466. doi:10.1111/dom.13538

4. Lupoli R, Lembo E, Saldalamacchia G, Avola CK, Angrisani L, Capaldo B. Bariatric surgery and long-term nutritional issues. World J Diabetes. 2017;8(11):464-474. doi:10.4239/wjd.v8.i11.464

5. Eisenberg D, Shikora SA, Aarts E, et al. 2022 American Society of Metabolic and Bariatric Surgery (ASMBS) and International Federation for the Surgery of Obesity and Metabolic Disorders (IFSO) indications for metabolic and bariatric surgery. Obes Surg. 2023;33(1):3-14. doi:10.1016/j.soard.2022.08.013

6. Zhou X, Zeng C. Diabetes remission of bariatric surgery and nonsurgical treatments in type 2 diabetes patients who failure to meet the criteria for surgery: a systematic review and meta-analysis. BMC Endocr Disord. 2023;23(1):46. doi:10.1186/s12902-023-01283-9

7. Wilding JPH, Batterham RL, Davies M, et al; STEP 1 Study Group. Weight regain and cardiometabolic effects after withdrawal of semaglutide: the STEP 1 trial extension. Diabetes Obes Metab. 2022;24(8):1553-1564. doi:10.1111/dom.14725

8. Jastreboff AM, le Roux CW, Stefanski A, et al; SURMOUNT-1 Investigators. Tirzepatide for obesity treatment and diabetes prevention. N Engl J Med. 2025;392(10):958-971. doi:10.1056/NEJMoa2410819

9. Fujihara K, Matsubayashi Y, Kitazawa M, et al. Achieving normoglycaemia with tirzepatide: post hoc exploratory analysis of the SURPASS J-mono and J-combo studies. Diabetes Obes Metab. 2024;26(11):5304-5311. doi:10.1111/dom.15887

10. Vilsbøll T, Bain SC, Leiter LA, et al. Semaglutide, reduction in glycated haemoglobin and the risk of diabetic retinopathy. Diabetes Obes Metab. 2018;20(4):889-897. doi:10.1111/dom.13172

11. Amini A, Hamann S, Larsen M. Semaglutide and non-arteritic anterior ischaemic optic neuropathy: review and interpretation of reported association. Acta Ophthalmol. Published online March 8, 2025. doi:10.1111/aos.17473

12. Neeland IJ, Linge J, Birkenfeld AL. Changes in lean body mass with glucagon-like peptide-1-based therapies and mitigation strategies. Diabetes Obes Metab. 2024;26(suppl 4):16-27. doi:10.1111/dom.15728

13. Sattar N, Neeland IJ, Dahlqvist Leinhard O, et al. Tirzepatide and muscle composition changes in people with type 2 diabetes (SURPASS-3 MRI): a post-hoc analysis of a randomised, open-label, parallel-group, phase 3 trial. Lancet Diabetes Endocrinol. 2025;13(6):482-493. doi:10.1016/S2213-8587(25)00027-0

14. Elortegui Pascual P, Rolands MR, Eldridge AL, et al. A meta-analysis comparing the effectiveness of alternate day fasting, the 5:2 diet, and time-restricted eating for weight loss. Obesity (Silver Spring). 2023;31(suppl 1):9-21. doi:10.1002/oby.23568

15. Guo L, Xi Y, Jin W, et al. A 5:2 intermittent fasting meal replacement diet and glycemic control for adults with diabetes: the EARLY randomised clinical trial. JAMA Netw Open. 2024;7(6):e2416786. doi:10.1001/jamanetworkopen.2024.16786

16. Chous AP. 5 diabetes truisms that are (at least partially) false. Optometry Times. March 25, 2022. Accessed May 20, 2025.
https://www.optometrytimes.com/view/5-diabetes-truisms-that-are-at-least-partially-false

A. Paul Chous, MA, OD, FAAO | E: dr.chous@diabeticeyes.com

Chous has a practice emphasising diabetes eye care and education in Tacoma, Washington.

GLP-1 therapies could impact long-term outcomes in diabetic retinopathy.

A realistic outlook on diabetes remission: GLP-1 therapies, diabetic retinopathy’s future

Neurotech Pharmaceuticals recently announced the first commercial manufacturing, shipment, and surgical procedure of the first and only FDA-approved treatment for adults with idiopathic macular telangiectasia type 2 (MacTel), ENCELTO (revakinagene taroretcel-lwey).

“Performing the first ENCELTO surgery for a patient with MacTel outside a clinical trial is an important milestone. For the first time, we can offer a valuable and durable treatment option for patients, one that slows their loss of photoreceptors and maintains more visual function over time,” Charles C. Wykoff, MD, PhD, Retinal Consultants of Texas, Houston, said in a press release.

Angiogenesis 2025: Encapsulated cell therapy for MacTel shows long-term efficacy in phase 3 trials

“Many patients with MacTel experience gradual and progressive functional declines over time, and it is exciting to offer an option to change the course of their disease.”

A bilateral, neurodegenerative disease in adults, MacTel has “characterized local retinal degeneration that causes the gradual loss of cells in the retina, resulting in vision loss and secondary alterations of the retinal vasculature,” a press release said.

Designed to provide long-term, sustained delivery of therapeutic proteins for the treatment of chronic eye disease, ENCELTO is an encapsulated cell-based gene therapy that uses Neurotech’s ECT platform, which is surgically inserted. The ECT platform is made of small, semipermeable capsules containing the proprietary allogeneic retinal pigment epithelium (RPE) cells, which are genetically engineered to produce specific therapeutic proteins for targeted disease treatment.

“This is a huge milestone for people living with MacTel. For the first time, there is an FDA-approved treatment option that could change what is possible for patients,” Richard Small, CEO of Neurotech, said in a press release.

“We have been working toward this for years, and seeing it become a reality is incredibly exciting for the Neurotech team and, most importantly, for the patients who may benefit.”

Increased awareness and diagnosis of MacTel

The availability of an FDA-approved treatment will likely drive greater awareness of MacTel among eye care professionals.

“Historically, MacTel has been underdiagnosed due to its subtle clinical presentation, but the introduction of an intervention will encourage earlier detection and referral patterns among ophthalmologists and optometrists,” said Rishi P. Singh, MD, FASRS, who is vice president/chief medical officer of Martin North and South Hospitals; professor of ophthalmology, Cleveland Clinic Lerner College of Medicine; Cleveland Clinic Martin Health in Stuart, Florida. He is also a member of the editorial advisory board for 

. This could lead to improved patient outcomes as we diagnose and manage the disease at earlier stages, he noted.

“We view the Neurotech device as a platform technology which will be useful for delivering not only neurotrophics but perhaps other molecules, like angiostatics or other neurotrophics other than CNTF to treat other neurovascular glial degenerative diseases like glaucoma, age-related macular degeneration, retinitis pigmentosa,” Martin Friedlander, MD, PhD, who is professor, Department of Molecular Medicine, Scripps Research Institute; chief, Retina Services, Division of Ophthalmology, Scripps Clinic; and president, Lowy Medical Research Institute (LMRI), La Jolla, California, said in a 

A new era for MacTel: Clinicians react to FDA approval of revakinagene taroretcel-lwey

“Approval for this treatment for this disease that is incurable right now...will be a wonderful situation for retina specialists and for the patients. First of all for the patients, and then for the retina specialists who will be able to provide treatment for this incurable disease,” said Anat Loewenstein, MD, professor and head of retina, Division of Ophthalmology, Tel Aviv Medical Center, VP Ambulatory Services; president, EURETINA; Sydney Fox Chair of Ophthalmology, Tel Aviv University; and president, Israeli Ophthalmological Society, Tel Aviv, Israel. She is also a member of the editorial advisory board for 

“By delivering continuous neurotrophic support to the retina, this device offers a novel approach that could slow disease progression and help preserve vision in affected individuals. This shift from passive monitoring to an active treatment strategy is a game changer for both patients and clinicians,” Singh said.

Who should not receive an ENCELTO surgical implant?

ENCELTO has not been tested in pediatric patients or pregnant people.

The outpatient surgical procedure should not be performed if a patient is experiencing an active or suspected eye infection.

Patients should know receive ENCELTO if there is a known patient history of hypersensitivity to Endothelial Serum Free Media (Endo-SFM).

Before receiving ENCELTO, eye surgeons should be aware of all patient medical conditions, including the following

Pregnancy or plans to become pregnant. Although studies have shown that rhCNTF does not enter the bloodstream, its effects on an unborn baby have not been fully studied.

Breastfeeding or plans to breastfeed. It is not known whether rhCNTF passes into breast milk.

Currently taking or have recently taken medicines that lower the chance of blood clots forming in the body such as warfarin, low or regular doses of aspirin, or nonsteroidal anti-inflammatory drugs (NSAIDs).

What should patients avoid while taking ENCELTO?

Wear eye protection during the day and an eye shield at night for 1 week.

Do not drive or use machinery until the eye shield has been removed and the eye surgeon clears the patient that vision has recovered to an acceptable level.

Use a topical antibiotic solution at a frequency of 1 drop 4 times a day for 7 days.

Use a steroid drop taper of prednisolone acetate 1% (or equivalent) starting the day after surgery with the following taper:

1 drop 4 times a day for the first 7 days;

1 drop 3 times a day for the next 7 days;

1 drop 2 times a day for the next 7 days;

Potential adverse effects of ENCELTO and the surgical insertion include but are not limited to:

Retinal tear and detachment (the retina tears and potentially separates from the eye wall, resulting in vision loss)

Vitreous hemorrhage (bleeding within the central cavity of the eye)

Implant extrusion (when the ENCELTO begins to work its way out of the way)

Suture-related issues (such as suture-related eye irritation or exposure of sutures)

Accelerated cataract formation (clouding of the lens of the eye)

Delayed dark adaptation (the eye’s ability to adjust from bright lighting conditions to dark lighting conditions)

Common symptoms to experience following ENCELTO surgery:

First FDA-Approved Treatment for MacTel Performed. August 11, 2025. 

https://www.businesswire.com/news/home/20250811189087/en/First-FDA-Approved-Treatment-for-MacTel-Performed

Friedlander M, Loewenstein A, Rafieetary M, Rishi, Stevenson S. A new era for MacTel: Clinicians react to FDA approval of revakinagene taroretcel-lwey. Ophthalmology Times. Published March 10, 2025. Accessed August 12, 2025. https://www.ophthalmologytimes.com/view/a-new-era-for-mactel-clinicians-react-to-fda-approval-of-revakinagene-taroretcel-lwey

Neurotech Pharmaceuticals performs the first ENCELTO procedure.

First FDA-approved MacTel treatment performed

A study performed by Indian researchers found that children with retinoblastoma were diagnosed after a lag time of under 3 months, which in turn delayed treatment. They cited the need for training and awareness initiatives to improve management of these patients,

 according to the first author, Sima Das, MD. She is from the Oculoplasty and Ocular Oncology Services, Dr Shroff’s Charity Eye Hospital, New Delhi, India.

Retinoblastoma is the most common intraocular malignant cancer in children, and survival rates depend on the geographic location and the economic status of the country. Das and colleagues cited a study that reported that the 3-year survival rate in low- and middle-income countries was 50%, in contrast to almost 100% survival in patients in high-income countries.

The survival rates reported from high-income countries were 88% in the United Kingdom and 93% in the US.

 However, in countries like India, Nepal, Africa, and South American countries, the children with retinoblastoma present much later when the disease is more advanced, and their prognoses are significantly inferior. For example, orbital retinoblastoma has been reported to be 18% in Mexico, 36% in Taiwan, and 40% in Nepal, and ranges from 9% to 45% in India, Das and colleagues related.

“Delay in diagnosis of retinoblastoma has been associated with lower chances of globe salvage and higher mortality.

 Studies from South America have noted prolonged time to diagnosis to be more associated with extraocular disease at presentation.

 Reduced lag time to diagnosis has been associated with less advanced RB, as found in a Swiss study.

Sophie J. Bakri, MD

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