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A clinical trial has opened in Poland that will test whether regorafenib can be used to treat bone cancers. Regorafenib is a type of drug called a tyrosine kinase inhibitor (TKI). TKIs work by blocking the activation of enzymes known as tyrosine kinases. They are involved in multiple processes in the body. This includes cancer growth. The researchers hope that blocking these enzymes could help to treat osteosarcoma (OS). TKIs have been approved to treat other cancers.

The trial will also look at how the genetic makeup of a cancer affects how a person will respond to treatment. Patients in this trial will have molecular testing to see what genetic changes they have.

We were delighted to interview trial lead Professor Anna Raciborska about REGBONE. Professor Raciborsk is Head of the Department of Oncology and Surgical Oncology for Children and Youth in Warsaw, Poland.

Please could you tell us about the REGBONE trial?

The REGBONE project is a non-commercial clinical trial (it is not funded by a pharmaceutical company). The aim of the project is to improve treatment outcomes in patients with primary malignant bone tumours, which do not respond to standard treatment, by increasing the availability of advanced therapy.

The trial will also develop treatment options using advanced molecular diagnostics for patients who have not responded to the standard therapeutic regimen.

The objectives of the REGBONE project are:

(1) to estimate the nature and frequency of mutations in the tumour tissue.

(2) to compare molecular test results with clinical data. This will allow for the initial assessment of the impact of the mutation status on the clinical condition, course of treatment and prognosis.

(3) to include the new targeted treatment regorafenib in standard therapy.

As part of the REGBONE trial an immortalized cell line (cancer cells grown in a laboratory) will be derived to study the development of the disease, drug sensitivity, and drug resistance mechanisms.

This project is completely financed by the Polish Medical Research Agency (ABM) from state budget funds.

What research led to this trial?

Primary bone tumours are ultra-rare diseases. Approximately 1,100 – 1,200 new cases of malignant tumours are diagnosed in children each year in Poland. Bone tumours account for approximately 6-7% of cases. (This is 6-7 cases in every 100 cases of childhood cancers).

Unfortunately, despite the intensity of therapy in patients with bone cancer, the outcomes have not been improved for over 30 years. For this reason, other treatment options are being investigated throughout the world.

Based on the current research, the inclusion of TKIs appeared to be promising. Also, our preliminary studies showed a significant response rate to this class of drugs in primary refractory bone sarcomas (bone cancer that does not respond to standard treatment).

Why is molecular testing important?

The identification of new mutations in bone tumours has led to a better insight into the molecular basis of tumours. This has enhanced the role of genetic research in everyday practice. Although histopathological examinations are currently the foundation for the diagnosis of bone tumours, new molecular biology techniques allow for the refinement of the diagnosis in many cases. In the near future, they will become the basis for the categorization of these cancers. These methods are also intended to match patients to advanced molecular-targeted therapies.

Who can take part in this clinical trial?

The scope of the project is to cover the entire population of children, adolescents, and young adults from the age of 2 to the age of 21. It is for people who have progressed on first-line treatment or who presented with a recurrence of Ewing’s sarcoma or OS.

The project is conducted at the Institute of Mother and Child (IMID) and the National Research Institute of Oncology (PIB-NIO) as a collaborator. Also, the SARCOMA Association for Helping Patients with Sarcomas and Melanomas (as a patient organization) is a collaborator of the project.

What are the next steps once the trial has finished?

We hope that the results of the research will allow regorafenib to be included as standard therapy in the treatment of primary bone tumours, and this therapy will be reimbursed for all patients in need (the cost of the drug will be covered by the government).  If not, or if necessary, we will continue to look for solutions to improve survival in this group of patients.

What advice would you give to anyone considering taking part in a clinical trial?  

Taking part in a clinical trial gives patients the opportunity to take advantage of the latest therapy, an innovative drug, or a treatment procedure. Although there is no guarantee of success, patients should certainly feel that they are provided with the best possible care. This is especially important if a child has cancer. In the case of cancers in which standard treatment has failed, inclusion in a clinical trial may be a chance to find an effective drug. Patients included in clinical trials do not incur costs related to treatment, are under the complete care of a team of doctors and nurses, and have access to medical procedures and diagnostics at the highest level. It should be remembered that clinical trials are the basis of objective progress in medicine.

So let’s not be afraid of clinical trials. They offer a chance for a better future.

You can find out more about the trial on the trial website. A summary of the trial can also be found on ONTEX.

We understand that clinical trials can be confusing. Visit our clinical trial toolkit for answers to questions about clinical trials and tips for preparing for appointments.

If you are interested in taking part in a clinical trial it is always important to discuss the trial with your doctors first. They will know your case best and be able to advise you on if a trial may be suitable.

We would like to thank Professor Raciborska for taking part in the interview. We would also like to thank study co-ordinator Katarzyna Maleszewska for helping us to organise it.  

Please note that the text in brackets was added by the Osteaosarcoma Now Team.