A decision process for drug discovery in retinoblastoma

Cancela, Maria Belen; Zugbi, Santiago; Winter, Ursula; Martı́nez, Ana; Sampor, Claudia; Sgroi, Mariana; Francis, Jasmine H.; Garippa, Ralph; Abramson, David H.; Chantada, Guillermo; Schaiquevich, Paula

doi:10.1007/s10637-020-01030-0

Cited by 14 publications

(10 citation statements)

References 118 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…Multi‐drug combination chemotherapy, including vincristine, etoposide, carboplatin/cisplatin, cyclophosphamide/ifosfamide, and topotecan, was administered 8 . Intrathecal injection of methotrexate, cytosine arabinoside, and dexamethasone was administered when the CNS was involved 10 …”

Section: Methodsmentioning

confidence: 99%

“…8 Intrathecal injection of methotrexate, cytosine arabinoside, and dexamethasone was administered when the CNS was involved. 10 Abdominal CT and contrast-enhanced MRI of the orbit were reviewed after two or three chemotherapy cycles. Radiotherapy after chemotherapy and autologous peripheral blood stem cell transplantation (APBSCT) were scheduled.…”

Section: Methodsmentioning

confidence: 99%

See 1 more Smart Citation

Clinical features and imaging manifestations of retinoblastoma with hepatic metastasis

Wang

Huang

et al. 2021

Pediatric Blood & Cancer

View full text Add to dashboard Cite

Clinical data of five patients with hepatic metastases of retinoblastoma were analyzed retrospectively (two had bilateral tumors three had unilateral intraocular tumors). On computed tomography, multiple and single low‐density foci were observed. Four patients had tumor remission, and one showed no response after chemotherapy. Three patients who underwent enucleation were at high risk for extensive choroidal invasion. Central nervous system and bone metastases occurred in all five patients. Neuron‐specific enolase and lactate dehydrogenase levels were significantly elevated in all patients. Two patients died (not from hepatic metastasis). Three patients (one with tumor progression and two with shorter courses) are continuing treatment.

show abstract

Section: Methodsmentioning

confidence: 99%

Section: Methodsmentioning

confidence: 99%

Clinical features and imaging manifestations of retinoblastoma with hepatic metastasis

Wang

Huang

et al. 2021

Pediatric Blood & Cancer

View full text Add to dashboard Cite

show abstract

“…A current Latin American GALOP group-led study is testing the use of IT topotecan in the adjuvant setting (96,97); more such studies are necessary to determine the role of IT topotecan in retinoblastoma. Other putative candidates for intrathecal use include romidepsin, etoposide, and teniposide (98).…”

Section: Intrathecalmentioning

confidence: 99%

“…Subsequently, almost 200 compounds were found to be cytotoxic to primary cell lines derived from intraocular and CSF-disseminated retinoblastomas, including drugs already used in pediatric oncology and new hits in preliminary phases of drug development (98). A selection process for prioritizing drugs for subsequent studies before clinical translation based on their pharmacokinetics, biopharmaceutical properties, safety profiles, and the intent of treatment was proposed.…”

Section: Drug Discovery In Retinoblastomamentioning

confidence: 99%

“…Expression of members of the retinoid X receptor family was also found to be altered, supporting vitamin D and retinoic acid, the ligands of these receptor, as candidate retinoblastoma therapeutics. Finally, the NF-kB, IGF1R, and STAT3 pathways were also deregulated; agents that target these pathways have already been proposed (98).…”

Section: Drug Discovery In Retinoblastomamentioning

confidence: 99%

See 1 more Smart Citation

Treatment of Retinoblastoma: What Is the Latest and What Is the Future

et al. 2022

Self Cite

View full text Add to dashboard Cite

The management of retinoblastoma, the most common intraocular malignancy in children, has changed drastically over the last decade. Landmark developments in local drug delivery, namely, safer techniques for intravitreal chemotherapy injection and ophthalmic artery chemosurgery, have resulted in eye globe salvages that were not previously attainable using systemic chemotherapy or external beam irradiation. Novel drugs, oncolytic viruses, and immunotherapy are promising approaches in the treatment of intraocular retinoblastoma. Importantly, emerging studies of the pattern of tumor dissemination and local drug delivery may provide the first steps toward new treatments for metastatic disease. Here, we review recent advances in retinoblastoma treatment, especially with regard to local drug delivery, that have enabled successful conservative management of intraocular retinoblastoma. We also review emerging data from preclinical and clinical studies on innovative approaches that promise to lead to further improvement in outcomes, namely, the mechanisms and potential uses of new and repurposed drugs and non-chemotherapy treatments, and discuss future directions for therapeutic development.

show abstract