DNA strand intertwined with soft fabric, symbolizing soft tissue sarcoma

Soft Tissue Sarcoma: Is Molecular Profiling Worth It?

"Unlocking the potential of NGS in personalized sarcoma treatment."


Over the last few decades, cancer treatments have improved significantly, leading to better outcomes for many patients. Discoveries in chemotherapy and the development of multimodal therapies have dramatically increased cure rates for childhood cancers. Similarly, survival rates for testicular and breast cancer have also seen substantial improvements.

Unfortunately, progress in treating soft-tissue sarcomas (STS) has been slow. These rare tumors, which include over 70 different subtypes, present unique challenges. STS affects both children and adults, with 5% of cases occurring in those under 20, and 30% in those over 75 years old.

Many STS subtypes have unique oncogenesis mechanisms, making them potentially responsive to targeted systemic treatments. Identifying new therapies for STS patients is crucial, as 40% to 50% of patients develop metastatic disease. Once metastasis occurs, treatment options are limited, primarily involving palliative chemotherapy with a median survival of only 12 to 20 months.

Targeting Sarcomas with Next-Generation Sequencing

DNA strand intertwined with soft fabric, symbolizing soft tissue sarcoma

While whole-genome sequencing is valuable for discovery-based research, targeted gene panel sequencing is a cost-effective approach for identifying targetable alterations in cancer patients. By focusing on specific genes or regions, sequencing can be performed at a much higher depth than with whole-genome or whole-exome approaches.

Two extensive studies investigated the role of targeted NGS in identifying actionable mutations in soft-tissue sarcoma patients. These studies classified actionable alterations into four levels based on the strength of evidence indicating drug sensitivity to FDA-approved or investigational agents.

  • Level 1: Alterations associated with approved anti-cancer drugs in sarcomas.
  • Level 2: Standard care biomarker predictive of response to drugs not approved for sarcoma management.
  • Level 3: Alterations associated with compelling clinical evidence of predictive value, but neither biomarker nor drug is standard of care.
  • Level 4: Alterations associated with compelling biological evidence, but neither biomarker nor drug is standard of care.
One study analyzed 584 STS cases using a comprehensive genomic profiling assay based on NGS. The results identified 2,697 alterations across 451 genes, with an average of 4 (1-53) alterations per case. The most frequently altered genes included TP53, MDM2, CDK4, and RB1. At least one targetable genomic alteration was found in 239 cases (41%), with a statistically significant higher number in other and complex genomics sarcomas than in translocation-related sarcomas (p < 0.001).

The Future of Sarcoma Treatment: NGS and Personalized Medicine

The MULTISARC trial, launched in early 2018, aims to demonstrate that NGS implementation can improve overall survival in advanced STS patients. This prospective randomized study randomizes patients with advanced STS to either NGS-informed management or conventional treatment. The trial integrates comprehensive clinical and molecular data, creating a unique resource for the scientific community.

Recent years have seen rapid advancements in sequencing technology, enabling quick and efficient tumor tissue analysis to identify actionable mutations and provide personalized therapies. Studies have shown promise, and it is believed that this technology will become even more beneficial in the future.

High-quality randomized trials and prospective studies are necessary to provide level I evidence supporting NGS implementation in the routine management of advanced STS. As technology advances and more data becomes available, personalized treatment strategies based on molecular profiling hold the potential to significantly improve outcomes for sarcoma patients.

About this Article -

This article was crafted using a human-AI hybrid and collaborative approach. AI assisted our team with initial drafting, research insights, identifying key questions, and image generation. Our human editors guided topic selection, defined the angle, structured the content, ensured factual accuracy and relevance, refined the tone, and conducted thorough editing to deliver helpful, high-quality information.See our About page for more information.

This article is based on research published under:

DOI-LINK: 10.1007/s11864-018-0589-y, Alternate LINK

Title: Is There Value In Molecular Profiling Of Soft-Tissue Sarcoma?

Subject: Pharmacology (medical)

Journal: Current Treatment Options in Oncology

Publisher: Springer Science and Business Media LLC

Authors: Antoine Italiano

Published: 2018-12-01

Everything You Need To Know

1

What makes treating soft-tissue sarcomas (STS) particularly challenging compared to other cancers?

Soft-tissue sarcomas (STS) present a unique challenge due to their rarity and the existence of over 70 different subtypes. This heterogeneity means that each subtype may have distinct oncogenesis mechanisms, making them potentially responsive to different targeted systemic treatments. Progress in treating STS has been slower compared to other cancers, and a significant percentage of patients develop metastatic disease, where treatment options are limited to palliative chemotherapy.

2

What is the difference between targeted gene panel sequencing and whole-genome sequencing, and why is targeted sequencing preferred in some cases?

Targeted gene panel sequencing focuses on specific genes or regions of the genome known to be relevant in cancer, allowing for much higher sequencing depth compared to whole-genome or whole-exome approaches. This makes it a cost-effective method for identifying actionable alterations in cancer patients, including those with soft-tissue sarcoma. Whole-genome sequencing, while valuable for discovery, is more comprehensive and expensive.

3

How are actionable alterations identified through NGS classified based on their clinical relevance in soft-tissue sarcoma?

In the context of soft-tissue sarcoma, actionable alterations are classified into four levels. Level 1 alterations are associated with approved anti-cancer drugs specifically for sarcomas. Level 2 involves standard care biomarkers predictive of response to drugs not approved for sarcoma management. Level 3 includes alterations with compelling clinical evidence of predictive value, but neither the biomarker nor the drug is standard of care. Level 4 alterations are associated with compelling biological evidence, but neither the biomarker nor the drug is standard of care.

4

What is the MULTISARC trial, and how does it aim to advance the treatment of soft-tissue sarcoma?

The MULTISARC trial is a prospective randomized study designed to assess whether implementing NGS can improve overall survival in patients with advanced soft-tissue sarcoma. Patients are randomized to either NGS-informed management or conventional treatment. By integrating comprehensive clinical and molecular data, the trial aims to create a valuable resource for the scientific community, potentially leading to more personalized and effective treatments for STS.

5

What are some of the most frequently altered genes identified through NGS in soft-tissue sarcoma, and what are the implications of these alterations?

In studies using NGS in soft-tissue sarcoma, frequently altered genes include TP53, MDM2, CDK4, and RB1. Identifying these alterations can help in determining potential therapeutic targets. For example, the presence of MDM2 amplification may suggest sensitivity to MDM2 inhibitors in certain STS subtypes. Understanding the specific alterations in each sarcoma case is vital for tailoring treatment strategies.

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