Drug which stops tumors’ blood supply could help kids with bone cancer live longer – Frontiers

Report on Pazopanib as a Treatment for Pediatric Multi-Metastatic Ewing’s Sarcoma

Introduction and Alignment with Sustainable Development Goals

This report details the findings of a non-randomized retrospective study on the use of pazopanib for treating multi-metastatic Ewing’s sarcoma in children. This form of bone cancer presents a significant challenge to global health objectives, with historical five-year survival rates below 25%. The research directly addresses the United Nations Sustainable Development Goal 3 (SDG 3), which aims to ensure healthy lives and promote well-being for all at all ages, specifically targeting the reduction of premature mortality from non-communicable diseases.

Methodology of the Retrospective Study

The study, conducted at the Warsaw Mother and Child Institute, evaluated the integration of pazopanib into standard treatment protocols. The primary objective was to assess its impact on survival rates and disease progression in a high-risk pediatric population.

• Participants: 11 patients, aged five to 17, with multi-metastatic Ewing’s sarcoma.
• Intervention: Pazopanib was administered alongside standard first-line treatments, including chemotherapy, surgery, and radiotherapy.
• Duration: The study period was from 2016 to 2024, with patients receiving pazopanib for an average of 1.7 years.
• Mechanism of Action: Pazopanib functions by inhibiting angiogenesis, thereby restricting the tumor’s ability to develop new blood vessels necessary for growth and metastasis.
• Monitoring: Patient progress was tracked through regular imaging, laboratory tests, and monitoring for adverse effects.

Key Findings and Contribution to SDG 3

The study yielded promising results that represent a potential advancement in achieving the targets of SDG 3. The outcomes suggest a significant improvement in patient survival and quality of life, a core component of universal health coverage.

1. Overall Survival: The two-year overall survival rate was 85.7%. Of the 11 patients, 10 were alive at the time of the report.
2. Event-Free Survival: The two-year event-free survival rate, indicating no disease progression, was 68.2%.
3. Treatment Tolerability: Pazopanib was well-tolerated, with minimal and manageable side effects, which did not add severe toxicity to the overall treatment regimen.
4. Quality of Life: The ability for patients to receive pazopanib as a home-based oral treatment after completing intravenous therapy contributed positively to their quality of life.

Implications for Future Research and Global Partnerships (SDG 9 & SDG 17)

While the findings are encouraging, the study’s small scale necessitates further investigation. The report underscores the need for scientific innovation and collaborative efforts to validate these results, aligning with SDG 9 (Industry, Innovation, and Infrastructure) and SDG 17 (Partnerships for the Goals).

• Need for Larger Trials: Researchers strongly advocate for the development of prospective, multicenter clinical trials to confirm the efficacy and safety of pazopanib in a larger patient cohort.
• Innovation in Treatment (SDG 9): This study exemplifies medical innovation by repurposing an existing drug, originally developed for renal cell carcinoma, to address a critical gap in pediatric oncology.
• Call for Partnerships (SDG 17): The successful implementation of larger-scale trials will require significant resources and international cooperation, highlighting the importance of forming public and private partnerships to advance global health research.

Conclusion

The preliminary results from this study suggest that pazopanib, when used as an adjunct to standard therapy, may significantly improve survival outcomes for children with multi-metastatic Ewing’s sarcoma. This contributes directly to the aims of SDG 3 by offering a potential pathway to reduce premature mortality from childhood cancer. To translate these findings into standard clinical practice, it is imperative to pursue further research through larger, controlled trials, leveraging the principles of innovation and partnership outlined in SDGs 9 and 17.

Analysis of Sustainable Development Goals (SDGs) in the Article

SDG 3: Good Health and Well-being

• The article’s entire focus is on improving health outcomes for children with a severe form of bone cancer, Ewing’s sarcoma. It discusses a medical study aimed at increasing survival rates and improving the quality of life for young patients, which is the core mission of SDG 3. The research on the drug pazopanib is a direct effort to “ensure healthy lives and promote well-being for all at all ages.”

Specific SDG Targets Identified

Target 3.4: Reduce premature mortality from non-communicable diseases

• The article directly addresses this target by focusing on a non-communicable disease (cancer) that causes premature death in children. It highlights the “dismal” survival rates, noting that “under a quarter of children with multi-metastatic Ewing sarcoma survived five years after their diagnosis.” The study’s success in increasing the two-year survival rate to 85% is a clear attempt to reduce this premature mortality.

Target 3.8: Achieve universal health coverage, including access to quality essential health-care services and access to safe, effective, quality and affordable essential medicines

• The research aims to establish a new, effective treatment regimen, which is a key component of providing “quality essential health-care services.” The article mentions that pazopanib is an existing drug being repurposed, and that it “was also very well tolerated, with minimal, treatable side-effects,” which speaks to the safety and quality of the medicine. The ability for patients to receive it “as a home treatment” also points towards improving access to care.

Target 3.b: Support the research and development of vaccines and medicines for communicable and non-communicable diseases

• This target is central to the article, which is a report on a clinical study. The text is filled with calls for more research, stating, “Researchers urge the development of larger-scale trials to investigate further” and “Our study could serve as a basis for creating prospective, multicenter clinical trials to confirm these promising results.” This highlights the critical role of R&D in finding treatments for diseases like Ewing’s sarcoma.

Indicators for Measuring Progress

Indicators for Target 3.4 (Reduce premature mortality)

• Survival Rates: The article provides specific quantitative data that can be used as indicators. It mentions the historical “five-year survival rate” of “less than a quarter” (<25%) as a baseline. The study's finding of a "two-year overall survival rate of 85.7%" serves as a direct indicator of progress in reducing mortality.
• Event-Free Survival Rate: The article states that “68.2% of patients made it to the end of the second year without a new ‘event’, meaning their cancer remained stable.” This is a key indicator used in oncology to measure the effectiveness of a treatment in controlling a disease and preventing relapse, directly contributing to long-term survival.

Indicators for Target 3.8 (Access to quality medicines)

• Treatment Tolerability: The article implies a qualitative indicator by noting that “pazopanib was also very well tolerated, with minimal, treatable side-effects.” Measuring and reporting the incidence and severity of side effects is a way to track the quality and safety of a medicine.
• Quality of Life: The article mentions that the “quality of life of treated children was good.” This is a crucial, albeit qualitative, indicator of the overall success of a healthcare intervention beyond just survival statistics.

Indicators for Target 3.b (Support R&D)

• Number and Scale of Clinical Trials: The article is a report on a “small non-randomized retrospective study” involving 11 patients. The call for “larger controlled trials” and “prospective, multicenter clinical trials” implies that the number and scale of such studies are direct indicators of investment and progress in medical research for this disease.

SDGs, Targets, and Indicators Summary

Source: frontiersin.org