Retinoblastoma
Recent clinical, regulatory, research and industry developments relating to this disease.
Genetics in ophthalmology: molecular blueprints of retinoblastoma.
Global Retinoblastoma Presentation and Analysis by National Income Level.
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
- 3 clinical trials expected to report results, the earliest in Q1 2028.
- Q1 2028Intravitreal Melphalan for Intraocular Retinoblastoma
- Q1 2028Phase 1 Trial of GPC2-Directed Chimeric Antigen Receptor Autologous T Cells (GPC2 CAR T) for Relapsed or Refractory Neuroblastoma and Metastatic Retinoblastoma
- Q1 2035Ocular Conservative Treatment for Retinoblastoma: Efficacy of the New Management Strategies and Visual Outcome - RETINO 2018
Clinical MilestonesViewHide
- 2025-09-04Protocol for the Study and Treatment of Patients With Intraocular RetinoblastomaResults posted
- 2026-02-17Intravitreal Melphalan for Intraocular RetinoblastomaResults expected Q1 2028
- 2025-12-29Phase 1 Trial of GPC2-Directed Chimeric Antigen Receptor Autologous T Cells (GPC2 CAR T) for Relapsed or Refractory Neuroblastoma and Metastatic RetinoblastomaResults expected Q1 2028
- 2025-11-28Ocular Conservative Treatment for Retinoblastoma: Efficacy of the New Management Strategies and Visual Outcome - RETINO 2018Results expected Q1 2035
- 2026-02-17ClinicalIntravitreal Melphalan for Intraocular RetinoblastomaResults expected Q1 2028
- 2025-12-29ClinicalPhase 1 Trial of GPC2-Directed Chimeric Antigen Receptor Autologous T Cells (GPC2 CAR T) for Relapsed or Refractory Neuroblastoma and Metastatic RetinoblastomaResults expected Q1 2028
- 2025-11-28ClinicalOcular Conservative Treatment for Retinoblastoma: Efficacy of the New Management Strategies and Visual Outcome - RETINO 2018Results expected Q1 2035
- 2025-09-04ClinicalProtocol for the Study and Treatment of Patients With Intraocular RetinoblastomaResults posted
Clinical trials
The current development programme across all trial phases.
Research activity
Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.
Major themes3
- Ophthalmology1
- Retinal Neoplasms1
- Retinoblastoma1
Leading journals2
- Human genomics1
- JAMA oncology1
Leading researchers8
- Abdallah E1
- Abdullahi SU1
- Abdulqader RA1
- Adamou Boubacar S1
- Ademola-Popoola DS1
- Adio A1
- Afshar AR1
- Aggarwal P1
Affiliations (unnormalised)6
- School of Medicine2
- Aarhus University Hospital1
- Abii Specialists Hospital1
- Aditya Jyot Eye Hospital1
- Affiliated Hospital of Dali University1
- Africa Institute of Tropical Ophthalmology1
Reference
Authoritative identity, definition & identifiers.
A malignant tumor arising from the nuclear layer of the retina that is the most common primary tumor of the eye in children. The tumor tends to occur in early childhood or infancy and may be present at birth. The majority are sporadic, but the condition may be transmitted as an autosomal dominant trait. Histologic features include dense cellularity, small round polygonal cells, and areas of calcification and necrosis. An abnormal pupil reflex (leukokoria); NYSTAGMUS, PATHOLOGIC; STRABISMUS; and visual loss represent common clinical characteristics of this condition. (From DeVita et al., Cancer: Principles and Practice of Oncology, 5th ed, p2104)
- Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
- Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
- Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
- Related entities are derived from literature co-mention (studied together) — associative, not causal.