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What’s hot in rare oncology? Our top headlines from ASCO 2024

The buzz at ASCO 2024 was as electric as ever, but making sense of the vast number of abstracts remains an overwhelming task.  

At Bioscript Group, we have a specialist interest in rare disease and oncology, and this year, we wanted to do something different. Harnessing the cutting-edge capabilities of Bioscript Data Insights (BDI), we deployed our team of algorithm-loving data scientists to analyse and summarise the key take-homes across rare oncology. 

Here, we provide an overview of our data-driven insights, highlighting the top headlines across a huge diversity of rare cancers, with links to full abstracts if anything piques your interest. 

Explore our insights into rare cancers across the categories below:

  • Gastrointestinal
  • Genitourinary  
  • Head and neck  
  • Haematology 
  • Lung and respiratory  
  • Skin/melanoma
  • Sarcoma 
  • Advancing personalised medicine

​​​​Want to know more?​ 

We invite you to explore these findings and discover how BDI can help you cut through complexity and uncover valuable insights in your own work. Get in touch with us to learn more about our data-driven approach to medical communications.

Let Bioscript Group be your strategic partner in navigating the rapidly evolving landscape of rare oncology. Contact us now:


Fighting resistance to immunotherapy in alpha-fetoprotein producing gastric cancer (AFPGC): Alpha-fetoprotein-producing gastric cancer (AFPGC) has long posed a challenge due to its resistance to standard PD-L1 immunotherapy. However, research suggests that combining SB265610 (a neutrophil chemokine receptor inhibitor), or apatinib, with PD-L1 immunotherapy could reverse this resistance. This novel approach warrants further investigation and may hold the key to enhancing outcomes for patients with AFPGC. 


In the realm of rare genitourinary cancers, ASCO 2024 was a hotbed of innovation, offering new hope with combination therapies for patients with challenging diagnoses: 

Nivolumab and ipilimumab: A dynamic duo for relapsed extra-renal clear cell cancer: Combining nivolumab and ipilimumab demonstrated significant and lasting activity in relapsed extra-renal clear cell cancer of gynecologic origin, including 4 complete responses, and median progression-free survival of 5.6 months in this chemotherapy-resistant disease. 

Sintilimab and axitinib for treatment of fumarate hydratase-deficient renal cell carcinoma: The combination of sintilimab (an anti-PD-1 immunotherapy) and axitinib (a VEGFR tyrosine kinase inhibitor) demonstrated promising antitumour activity and tolerability in advanced fumarate hydratase-deficient renal cell carcinoma (FH-dRCC), a rare and often difficult-to-treat subtype of RCC.  

Combination therapy for advanced adrenocortical carcinoma (ACC):  
The combination of camrelizumab (an anti-PD-1 immunotherapy) and apatinib (a VEGFR tyrosine kinase inhibitor) showed promising efficacy in a Phase II trial of patients with previously treated advanced adrenocortical carcinoma (ACC), a rare and aggressive cancer with limited treatment options.  

Head and neck 

In the rare head and neck oncology space, ASCO 2024 showcased encouraging progress across diverse tumour types. 

Combining anlotinib and sintilizumab for anaplastic thyroid cancer (ATC): The combination of anlotinib (a multi-kinase inhibitor) and sintilizumab (an anti-PD-1 immunotherapy) demonstrated promising efficacy and manageable toxicity in treating locally advanced or metastatic ATC, a rare and aggressive subtype with poor outcomes.  

Unleashing the immune system’s power against squamous cell carcinoma (SCC): Immunotherapy, either alone or combined with chemotherapy, yielded high response rates in patients with orbital or periocular squamous cell carcinoma (SCC), highlighting its potential in this challenging setting.  

Enhancer of zeste homolog 2: A new target for olfactory neuroblastoma (ONB): Researchers made exciting strides in understanding the role of enhancer of zeste homolog 2 (EZH2) activity in olfactory neuroblastoma (ONB). Their findings suggest that targeting EZH2 could potentially enhance the effectiveness of immune checkpoint blockade therapy, particularly in patients with high EZH2 repression scores. This breakthrough opens up new avenues for personalised treatment approaches in this rare and complex cancer. 


ASCO 2024 was filled with exciting news for patients with hard-to-treat rare blood cancers, as researchers unveiled promising new treatment strategies and innovative approaches. 

A powerful tag team for relapsed/refractory extranodal NK/T-Cell lymphoma (R/R ENKTL): In the battle against relapsed/refractory extranodal NK/T-cell lymphoma (R/R ENKTL), the combination of selinexor and tislelizumab demonstrates both a tolerable safety profile and promising efficacy, with a 72.7% overall response rate. This innovative approach could offer renewed hope for patients who have exhausted other treatment options.  

A beacon of hope for heavily pre-treated mycosis fungoides: Lacutamab, a monoclonal antibody that targets KIR3DL2-expressing cells, is a promising new therapy in heavily pre-treated mycosis fungoides. This novel immunotherapy demonstrated promising clinical activity in patients who had previously seen limited success with other treatments, offering a glimmer of hope in a challenging disease landscape. 

A potential new strategy for primary central nervous system (CNS) lymphoma: Orelabrutinib-containing regimens stepped into the limelight as a potential treatment strategy for primary CNS lymphoma, a rare and aggressive form of lymphoma that affects the brain and spinal cord. This innovative approach could change the game for patients facing this difficult diagnosis. 

Combined therapies for relapsed/refractory primary central nervous system lymphoma (R/R PCNSL): The combination of emavusertib, a novel oral inhibitor of IRAK4 and FLT3, with ibrutinib, showed impressive results in patients with relapsed/refractory primary CNS lymphoma. This exciting new treatment approach achieved a higher complete response rate in patients who had previously been exposed to ibrutinib alone, opening new doors for those who have struggled to find effective therapies. 

Lung and respiratory oncology  

There were significant advancements in the treatment of various rare respiratory and lung cancers presented at ASCO 2024:  

Gene therapy offers hope for recurrent respiratory papillomatosis: In a pivotal Phase I/II study, PRGN-2012, a gorilla adenovirus-based gene therapy, demonstrated remarkable efficacy, with over half of patients achieving a complete response and no serious adverse events reported. 

Combination therapies enhance outcomes for thymic carcinoma: The combination of ramucirumab, a monoclonal antibody targeting the VEGF receptor, with carboplatin-paclitaxel significantly improved response rates compared to chemotherapy alone in patients with thymic carcinoma. 

Novel approaches for advanced lung cancers: While the results were more modest, selinexor, an oral selective inhibitor of nuclear export, demonstrated some anticancer activity and led to prolonged stable disease in some patients with advanced thymic epithelial tumours who had progressive disease after platinum-based chemotherapy.  

Lorlatinib offers hope for ROS1+ non-small cell lung cancer (NSCLC) patients: Lorlatinib, a third-generation tyrosine kinase inhibitor (TKI), showed significant antitumour activity in TKI-naïve patients with advanced ROS1-positive NSCLC.  


ASCO 2024 unveiled promising treatment avenues for various rare skin/melanoma cancers: 

​Isolated hepatic perfusion combined with ipilimumab and nivolumab in ​​​​​​u​veal melanoma: Combining isolated hepatic perfusion (IHP) with immunotherapy appears feasible and effective in managing liver-dominant metastatic uveal melanoma. The sequence of treatment (IHP first vs. immunotherapy first) seems to influence response rates, opening new avenues for optimising treatment plans. 

High response rates to combined penpulimab and chemotherapy for non-melanoma cutaneous carcinoma: Penpulimab, when combined with chemotherapy, has emerged as a potential treatment option for advanced cases, offering hope for improved outcomes. 

Meta-analysis highlights potential of PD-1 inhibitors in acral melanoma: A meta-analysis suggests that adding PD-1 inhibitors to standard adjuvant therapy might benefit patients with resected acral melanoma, a rare subtype. Further research is needed to confirm this promising finding and understand its potential role in early-stage disease management. 


Several studies showcased encouraging results for rare and difficult-to-treat sarcoma subtypes: 

Efficacy and safety of SHR-2554 demonstrated in advanced epithelioid sarcoma: SHR-2554, an oral selective EZH2 inhibitor, demonstrated significant promise in a Phase II trial, achieving its primary endpoint of PFS in patients with refractory epithelioid sarcoma, with an acceptable safety profile.  

Potential clinical benefit of nirogacestat in desmoid tumour: Nirogacestat, a gamma-secretase inhibitor, consistently improved PFS and objective response rates in patients, regardless of prognostic factors. This suggests broader potential benefits for those with this  challenging-to-treat tumour. 

Anti-HER2 therapy showing early potential in desmoplastic small round cell tumour (DSRCT): T-DXd, an anti-HER2 therapy, showed promising efficacy and minimal toxicity in DSRCT, a rare and aggressive tumour often found in young individuals. These findings suggest that targeting HER2 may be a viable treatment strategy for this difficult-to-treat cancer, and warrants further investigation. 

Efficacy of olverembatinib in SDH-deficient gastrointestinal stromal tumours (GIST): Olverembatinib, a novel tyrosine kinase inhibitor, demonstrated promising efficacy in SDH-deficient GIST, a rare subtype primarily found in young adults. This highlights the importance of molecular profiling in tailoring treatment strategies for specific subtypes. 

Phase II clinical trial results of cemiplimab secondary angiosarcomas: A trial of cemiplimab, an immune checkpoint inhibitor, in patients with locally advanced or metastatic secondary angiosarcomas showed promising results, with some patients achieving partial or complete responses.  

Advancing personalised medicine  

ASCO 2024 showcased several exciting advances in personalised medicine: 

Circulating cancer stem cell detection: Researchers developed a groundbreaking platform for detecting circulating cancer stem cells (CSCs) in the blood. These cells are believed to play a crucial role in tumour growth and metastasis. The study also linked high CD47 expression with increased tumour aggressiveness, potentially opening new avenues for targeted therapies. 

Rapid drug screening: Cellentia’s R3CE platform made waves with its impressive 100% concordance with clinical outcomes in breast cancer patients. This rapid drug screening tool holds the potential to revolutionise personalised treatment selection, ensuring patients receive the most effective therapies from the start. 

Enhanced genetic testing: A customised amplicon test system with reduced input biomaterial could enable more accurate identification of rare genetic variants. The amplicon test system offers research costs similar to traditional PCR methods and could allow more precise identification of rare genetic variants, crucial for targeted therapies and personalised treatment plans. 

Improved imaging for neuroendocrine tumours: A new imaging technique, Ga-68 DOTATATE PET/CT, demonstrated significant value in detecting new lesions in patients with gastroenteropancreatic neuroendocrine tumours (GEP-NETs). The results led to a change in management in 41% of patients and represent a new tool to support clinical decision-making. 

Artificial intelligence (AI) for prognostic prediction: AI is making strides in prognostic prediction. A study showcased how AI can assist in predicting overall survival outcomes in upper urinary tract urothelial cancer patients, using clinical factors like performance status, nodal status and grade. 

Want to know more? 

We invite you to explore these findings and discover how BDI can help you cut through complexity and uncover valuable insights in your own work. Get in touch with us to learn more about our data-driven approach to medical communications. 

Let Bioscript Group be your strategic partner in navigating the rapidly evolving landscape of rare oncology.  

Contact us now:

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