Soft tissue sarcoma research

All new potential treatments have to be fully researched before they can be adopted as standard treatment for everyone. This is so that

• We can be sure they work
• We can be sure they work better than the treatments that are available at the moment
• We know they are safe

First of all, treatments are developed and tested in laboratories. For ethical and safety reasons, experimental treatments must be tested in the laboratory before they can be tried in patients. If a treatment described here is said to be at the laboratory stage of research, it is not ready for patients and is not available either within or outside the NHS.

Tests of treatments on patients are called clinical trials. There is information about the 4 phases of clinical trials in the trials and research section of CancerHelp UK. In that section you can also visit our searchable database of clinical trials. Choose 'sarcoma - soft tissue' from the dropdown menu box and click 'search'. If there is a trial you are interested in, print it off and take it to your own specialist. If the trial is suitable for you, your doctor will need to refer you to the research team. The database also has information about closed trials and trial results.

Everything covered on this page is the subject of ongoing research. Until studies are completed and new effective treatments are found, these experimental treatments cannot be used as standard therapy for soft tissue sarcomas.

Cancer researchers study both normal and cancer cells so that they can find out more about the disease and what goes wrong within the cell. This research aims to find the causes of soft tissue sarcomas at cell level. They also look at response to treatment and how the genes in the sarcoma cells may affect that. Some genes can predict how the cancer will respond to chemotherapy. These genes are called 'multi-drug resistance' (mdr) genes. Doctors may be able to use this information in the future to choose treatments for people with sarcoma, according to how their cancer is likely to respond.

Doctors around the world are supporting research in hospitals and clinics to improve existing treatments for soft tissue sarcomas. Doctors continue to study different combinations of chemotherapy drugs, different doses, or giving the drugs in different sequences. The aim of this type of research is to find better ways of treating sarcomas with chemotherapy.

The EORTC 62931 trial looked at doxorubicin and ifosfamide chemotherapy after surgery. The researchers found that chemotherapy after surgery did not reduce the chance of sarcoma coming back (apart from a few people at high risk of their cancer coming back). So, chemotherapy is not routinely used after surgery for most types of sarcoma. 

In September 2007, trabectedin (Yondelis) was licensed in Europe for people with advanced soft tissue sarcoma that has not responded to, or has come back after, chemotherapy with ifosfamide and anthracyclines (for example, doxorubicin). Yondelis is a chemotherapy drug that was developed from sea squirts. In December 2009, the National Institute for Health and clinical Excellence (NICE) recommended that people can have trabectedin if their treatment with an anthracycline and ifosfamide is no longer working or they cannot have these chemotherapy drugs . The company that make the drug have agreed to pay for the trabectedin if you need more than 5 cycles. In June 2011, the SMC (Scottish Medicines Consortium) looked at it again and decided not to recommend its use in the NHS in Scotland because they say it is not cost effective.

The PICASSO 3 study is looking at a new drug called palifosfamide-tris alongside doxorubicin for soft tissue sarcoma that has spread. Doctors want to see if this combination works better then doxorubicin alone. Palifosfamide-tris sticks together the cancer cell’s DNA so that the cell can't reproduce itself. So doctors hope this treatment could help to stop the cancer growing.

Brostallicin is a chemotherapy drug that has been looked at for sarcoma, but the results so far have not been as promising as researchers had hoped.

Ewing's sarcomas can occur in bone or in soft tissue. Their treatment and outcomes are different to other types of soft tissue sarcoma. They are also sometimes called primitive neuroectodermal tumours (PNETs). The EURO-EWING 99 trial is looking into different combinations of chemotherapy for Ewing's tumours and PNET.

A trial called OTIS is looking at treosulfan to treat Ewing's sarcoma that has come back after treatment. Treosulfan is a chemotherapy drug similar to one already used to treat people with Ewing’s sarcoma. We know from research that treosulfan can help people with other types of cancer. The researchers think that treosulfan may help people who have already had treatment for their Ewing’s sarcoma.

One trial is looking at treatment for children and young people who have rhabdomyosarcoma. It is using different combinations of chemotherapy before or after surgery (or both), with or without radiotherapy. Another trial is looking at the same treatment options for children and young people with other types of soft tissue sarcoma.

The GeDDIS trial is looking at having either doxorubicin, or a combination of 2 drugs called docetaxel and gemcitabine, as the first treatment for advanced soft tissue sarcoma. It is for people who have sarcoma that has spread into surrounding body tissue, or to other parts of the body. This trial is for children over the age of 13 as well as adults. The aims of the trial are to find out if this combination of drugs works better than the standard treatment for soft tissue sarcoma that has spread and to learn more about the side effects.

One trial is looking at temozolomide for rhabdomyosarcoma. Doctors can treat rhabdomyosarcoma that has not responded to treatment or has come back with the chemotherapy drugs vincristine and irinotecan. Temozolomide is a chemotherapy tablet that doctors use to treat some other types of cancer. Doctors in this trial want to see if adding temozolomide to vincristine and irinotecan helps people with rhabdomyosarcoma.

Another trial is comparing the chemotherapy drugs eribulin and dacarbazine for people with soft tissue sarcoma. Doctors want to see if eribulin can help people with sarcoma that has come back after treatment (recurred). They want to see if it is better than the standard treatment of dacarbazine.

For more information about these trials, visit our clinical trials database. Choose 'sarcoma - soft tissue' from the dropdown menu of cancer types.

Cancers are made up of cells that grow and multiply quickly. They do this because they have a number of mistakes called mutations in their DNA. The gene changes are called a genetic signature. If there is a sarcoma in the body, small numbers of cells with the genetic signature can be found in the blood. Each person has a different genetic signature. The BODIS study is trying to find out if it is possible to use a blood test to monitor people with sarcoma, by looking for the faulty tumour DNA signature. They hope that the test can help them to know how well treatment is working or when a sarcoma has come back.

There is research into

• Radiotherapy for advanced sarcomas (palliative radiotherapy)
• Radiotherapy to help stop sarcomas coming back after surgery (adjuvant radiotherapy)
• Improving the doses we use and the area treated
• Internal radiotherapy

Cancer Research UK is supporting a trial that will look into using different doses of radiotherapy after surgery for soft tissue sarcoma. Doctors want to give enough treatment to control the cancer. But they don't want to give too high a dose as this could cause unnecessary side effects. The trial is called the VORTEX trial and is listed on our clinical trials database.  Another trial has looked into radiotherapy for aggressive fibromatosis (desmoid tumour) that can't be removed with surgery, or has come back.

New ways of giving radiotherapy may be developed for sarcoma. Internal radiotherapy (brachytherapy) is being studied in clinical trials for some types of cancers and has been used to treat sarcoma. Brachytherapy involves having radioactive seeds or wires implanted into the cancer - or after surgery, into the area where the cancer was. The advantage of this type of treatment is that it gives a very high dose of radiation to the site of the tumour, but a much lower dose to the surrounding area than with external radiotherapy.

Brachytherapy could be useful for treating people who have already had radiotherapy and either have some cancer left behind, or have cancer that comes back in the same place some time after their radiotherapy. We are a very long way from knowing how well this will work in sarcoma. But we have mentioned it here because it is something we are asked about.

Biological therapies are treatments that use natural substances from the body, or drugs made from these substances. There are a number of different types of biological therapy, including cancer growth blockers and drugs that stop cancer blood vessel growth (for example bevacizumab).

Some types of biological therapy that block the substances cells use to signal to one another to grow are being tested for some types of sarcoma. They are called tyrosine kinase inhibitors (TKIs) and include imatinib (Glivec) and axitinib. Other newer types of growth blockers include deforolimus and pazopanib.

Imatinib (Glivec)

Glivec is a tyrosine kinase inhibitor. It is licensed in the UK to treat a particular type of sarcoma called GIST. It can also help to treat a slow growing, low grade type of sarcoma called dermatofibrosarcoma protruberans (DFSP). Glivec is also called imatinib. The Americans spell it differently, as Gleevec. One advantage of Glivec is that it comes as a tablet so you can have treatment at home. There is detailed information about Glivec in our cancer drugs section.

GIST stands for gastro intestinal stromal tumour. These most often grow in the stomach, but can be found anywhere along the gut. The National Institute for Health and Clinical Excellence (NICE) recommend Glivec as a first treatment in the UK if you have GIST that cannot be completely removed with surgery.

Before you have Glivec for GIST, you have a test done on the cancer cells. The test checks for a protein called CD117 on the surface of the GIST cells, which Glivec targets. Most GIST cells have a fault in a gene called c-KIT. The gene fault (mutation) makes the cells make too much of the CD117 protein. If the cells test positive for the CD117 protein, Glivec is likely to work very well. But it may work even if your GIST cells are negative for this protein.

The EORTC 62024 trial is looking into using Glivec after surgery. This is called adjuvant therapy. Doctors want to see if it lowers the risk of GIST coming back. This trial is no longer recruiting patients, and we are waiting for the results. An American trial also looked into this and found that in people who took Glivec after surgery, their GIST was kept under control for longer than for people not taking Glivec.

In August 2010 NICE decided not to recommend Glivec after surgery. The recommendations say that the people in the trial have not been monitored for long enough to show it helps people live longer. NICE will look it again when the results from the European trial are available in late 2011. In August 2010, the Scottish Medicines Consortium (SMC) recommended Glivec should be available on the NHS in Scotland for GIST. It is only for people who have a high risk of their tumour coming back after surgery that has completely removed it.

The EORTC 62063 trial is looking at having surgery and imatinib for gastrointestinal stromal tumours (GIST). It for people whose tumours have spread locally or to the liver. Doctors often treat GIST that has spread with imatinib, and it stops the cancer coming back for a while. Doctors are always looking for ways to increase the amount of time before the GIST starts to come back. They think that having surgery, as well as imatinib may help. But they are not sure.The aim of this trial is to see if adding surgery to imatinib can increase the amount of time before GIST starts to come back.

Axitinib

The Axi-STS trial is looking at a new tyrosine kinase inhibitor called axitinib for soft tissue sarcoma that has spread to the lymph nodes (is locally advanced) or to any other organs of the body.

Deforolimus

Deforolimus (also known as AP23573) stops a protein called mTOR from working properly. mTOR is important because it controls other proteins that are needed for cells to grow. So if mTOR can't work, then neither can these other proteins. This could help to slow down or stop the cancer growing. The SUCCEED trial is looking into whether deforolimus can help to control soft tissue sarcoma that has spread, but has been shrunk or stopped from growing with chemotherapy. 

Pazopanib

Pazopanib (also called GW786034) is another TKI known as a multi TKI because it works in two ways. One is to stop cancer cells growing. The other is to stop the cancer being able to make it's own blood supply. This is called anti angiogenesis. Cancers need their own blood vessels to get bigger. Without a blood supply the cancer cannot continue to grow.

The EORTC 62043 trial looked at pazopanib for people with soft tissue sarcoma that continued to grow despite treatment. The trial results are now available and the researchers found that pazopanib was a useful treatment for soft tissue sarcoma and that the side effects were not too bad. A larger trial called PALETTE compared pazopanib tablets with a dummy drug (placebo) in people with soft tissue sarcoma that had spread and grown or come back despite other treatment. The results of this trial showed that people who took pazopanib had on average 3 months longer before their cancer started to grow than those who took the placebo. There is more information about pazopanib and its side effects in the cancer drug section.

Bevacizumab

Bevacizumab (Avastin) is a type of monocloncal antibody. The BERNIE trial is comparing bevacizumab and chemotherapy with chemotherapy alone for children and teenagers with soft tissue sarcoma that has spread.

You can find details of these trials on our clinical trials database. Choose 'sarcoma - soft tissue' from the dropdown menu of cancer types.