Driving research, providing support and improving outcomes for rare disease patients and their families

We attended the 2021 annual Sarcoma Patients EuroNet (SPAEN) conference where patients and academics came together to share their research and experiences. Here we share with you some of the highlights from the conference with a focus on bone sarcoma.

 

Immunotherapy.

In recent years several  immunotherapies which have been licensed for use, particularly in lung and skin cancers. Dr Joanna Szkandera, Medical Oncologist at the Medical University of Graz in Austria, discussed how sarcomas are at the beginning of their immunotherapy journey. A phase 2 clinical trial assessed Pembrolizumab, a PD-1 inhibitor in patients with advanced sarcoma. The majority of patients did not display an objective response with only 1 of 22 patients with osteosarcoma responding(1). However, a further clinical trial combined Axitinib (VEGF inhibitor) with Pembrolizumab, which showed promising results especially in Alveolar soft part sarcoma (2). Nivolumab (PD-1 inhibitor) and Ipilimumab (CTLA-4 inhibitor) have also been trialed in metastatic sarcoma. The phase 2 study found that combining the two therapies showed the most promise and warranted further investigations (3). These initial studies suggest that combination immunotherapy may be effective in some types of sarcoma.

 

Drug repurposing

Sue Burchill, Professor of Cancer Research at the University of Leeds, shared her insight into novel therapy development. She discussed that drug discovery is a long process with the time taken for drugs to go from the lab to the clinic taking between 10-36 years. Drug repurposing can accelerate this process and bring medications to clinic more quickly which is very desirable in diseases such as sarcoma where the outcome is often poor. Professor Burchill is working on repurposing drugs for the treatment of Ewing sarcoma. Using patient derived Ewing sarcoma cells, her lab identify chemo resistant cancer cells, culture them and measure the cells response to existing drugs.  Pre-clinical results show promise however further research including clinical trials is needed.

 

Clinical Trials

Dr Sandra Strauss, Senior Clinical Lecturer and Honorary Consultant Medical Oncologist at University College London Hospital discussed current clinical trials in bone sarcoma.  The OLIE trial is a worldwide phase two clinical trial in osteosarcoma to study the efficacy and safety of Lenvatinib. Lenvatinib is a type 1 tyrosine receptor kinase inhibitor which has antiangiogenic properties. Lenvatinib has already been licensed for use in a number of solid cancers including kidney and thyroid cancer. The rEECur trial is another randomized control trial comparing four chemotherapy agents in the treatment of Ewing sarcoma. There is currently no standardized treatment for refractory Ewing sarcoma, so this trial aims to identify which treatment arm is most effective.  Dr Strauss finished by saying that although there are not enough clinical trials in bone cancer, they are out there, and that clinical oncologists are the most important people to speak to if you are thinking of entering a clinical trial.

 

Patient Public Involvement.

Throughout the conference we heard from patients about their experiences, which was incredibly inspiring. They also discussed the importance of public patient involvement to help inform clinical research.  Often people feel the only way to be involved in clinical trials is through participation, but patients should also be working in partnership with clinicians and academics to ensure the trials are patient centered. The reason for this is patients understand the burden of a disease because they live with it every day. They will also be the ones who bear the risks of clinical trials. Getting patients involved early with the clinical trial process helps to move research forward in a way that prioritises the patient.

The SPAEN conference has provide insight into current sarcoma research and the importance of patient involvement. The Myrovlytis Trust are funding research into osteosarcoma with the aim to rapidly bringing treatments from the lab to the clinic.

 

 

References

  1. Tawbi HA, Burgess M, Bolejack V, Van Tine BA, Schuetze SM, Hu J, et al. Pembrolizumab in advanced soft-tissue sarcoma and bone sarcoma (SARC028): a multicentre, two-cohort, single-arm, open-label, phase 2 trial. Lancet Oncol [Internet]. 2017 Nov 1 [cited 2021 Jun 1];18(11):1493–501. Available from: https://pubmed.ncbi.nlm.nih.gov/28988646/
  2. Wilky BA, Trucco MM, Subhawong TK, Florou V, Park W, Kwon D, et al. Axitinib plus pembrolizumab in patients with advanced sarcomas including alveolar soft-part sarcoma: a single-centre, single-arm, phase 2 trial. Lancet Oncol [Internet]. 2019 Jun 1 [cited 2021 Jun 1];20(6):837–48. Available from: https://pubmed.ncbi.nlm.nih.gov/31078463/
  3. D’Angelo SP, Mahoney MR, Van Tine BA, Atkins J, Milhem MM, Jahagirdar BN, et al. Nivolumab with or without ipilimumab treatment for metastatic sarcoma (Alliance A091401): two open-label, non-comparative, randomised, phase 2 trials. Lancet Oncol [Internet]. 2018 Mar 1 [cited 2021 Jun 1];19(3):416–26. Available from: https://pubmed.ncbi.nlm.nih.gov/29370992/