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review · Nanoscale Advances

Nanoparticle-based delivery systems as emerging therapy in retinoblastoma: recent advances, challenges and prospects

In plain language

Retinoblastoma is the most common intraocular cancer found in children, and treating this condition remains difficult in developing nations due to late diagnosis. Existing treatments rely primarily on surgery, radiotherapy, and chemotherapy. However, conventional chemotherapy and radiation produce severe systemic side effects by damaging non-cancerous tissues, which contributes to high mortality rates and diminishes patient quality of life. Nanoparticle-based drug delivery systems offer an alternative strategy by enabling targeted delivery, superior tissue penetration and retention, improved bioavailability, and reduced toxicity. These platforms include ligand-conjugated and smart nanoparticles designed to direct therapeutic compounds and imaging agents specifically to tumour sites. Despite substantial evidence supporting the value of nanoparticles for treating retinoblastoma, their clinical translation requires greater focus to address ongoing challenges and help develop safer, more effective paediatric therapies.

Key takeaways

  • Retinoblastoma is the most common paediatric intraocular cancer, with management complicated by delayed diagnosis in developing countries.
  • Conventional chemotherapy and radiation generate serious off-target toxicities that contribute to high mortality and compound failures in clinical trials.
  • Nanoparticulate carriers offer targeted delivery, enhanced penetration and retention, increased bioavailability, and an improved toxicity profile.
  • Ligand-conjugated and smart nanoparticles enable the active targeting of both anticancer drugs and diagnostic imaging agents to tumour cells.
  • Clinical translation of nanoparticle carriers for retinoblastoma remains limited despite widespread evidence of their therapeutic benefits.

Why it matters

Standard retinoblastoma therapies often cause severe side effects in paediatric patients because toxic chemotherapies harm healthy cells. Exploring targeted nanoparticle delivery systems provides a route to direct therapeutics and diagnostic imaging straight to tumour cells. If successfully advanced, this approach could reduce harmful systemic side effects, improve survival rates, and support the development of safer medical treatments for children affected by intraocular cancers.

Commercialisation angle

These delivery platforms could enable targeted therapeutic and diagnostic formulations for use by pharmaceutical developers and oncology clinicians. The research remains in an early stage, as clinical translation of these carriers has not yet received adequate focus. Moving these technologies towards market use will require formulation scientists to resolve existing translation challenges and demonstrate safety and efficacy in clinical settings.

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Abstract

Retinoblastoma is the most common intraocular malignancy in children. The treatment of this rare disease is still challenging in developing countries due to delayed diagnosis. The current therapies comprise mainly surgery, radiotherapy and chemotherapy. The adverse effects of radiation and chemotherapeutic drugs have been reported to contribute to the high mortality rate and affect patients' quality of life. The systemic side effects resulting from the distribution of chemotherapeutic drugs to non-cancerous cells are enormous and have been recognized as one of the reasons why most potent anticancer compounds fail in clinical trials. Nanoparticulate delivery systems have the potential to revolutionize cancer treatment by offering targeted delivery, enhanced penetration and retention effects, increased bioavailability, and an improved toxicity profile. Notwithstanding the plethora of evidence on the beneficial effects of nanoparticles in retinoblastoma, the clinical translation of this carrier is yet to be given the needed attention. This paper reviews the current and emerging treatment options for retinoblastoma, with emphasis on recent investigations on the use of various classes of nanoparticles in diagnosing and treating retinoblastoma. It also presents the use of ligand-conjugated and smart nanoparticles in the active targeting of anticancer and imaging agents to the tumour cells. In addition, this review discusses the prospects and challenges in translating this nanocarrier into clinical use for retinoblastoma therapy. This review may provide new insight for formulation scientists to explore in order to facilitate the development of more effective and safer medicines for children suffering from retinoblastoma.

Research topics

  • Ocular Oncology and Treatments
  • Nanoplatforms for cancer theranostics
  • Cancer-related Molecular Pathways

Read the original research

This page summarises published work. The authoritative version sits with the publisher.

DOI: 10.1039/d3na00462g

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