Driving research, providing support and improving outcomes for patients and their families affected by rare conditions



We were delighted to attend the MIB Agents FACTOR conference in San Diego in June. The osteosarcoma (OS) community joined forces for an inspirational cause – to make things better for those with OS.

Support for families

MIB agents have several programmes for patients and families to help at a very difficult time. From gaming to prayer, bereavement support to acting as ambassadors, MIB are there every step of the way. A full wellness track was available at FACTOR, including painting, walks, Pilates, flower arranging and more.

The scientific agenda was packed with research updates and promise for the future.

Targeted therapies

Targeted therapy is a type of treatment that targets specific proteins found on cancer cells. These proteins usually control the growth, spread or division of these cells. As these drugs are very specific, they usually produce lesser side effects than chemotherapy.  However, it is important to know who would benefit from each drug. We heard from researchers looking to use the power of targeted therapies to improve outcomes in OS.

Amanda Marinoff discussed how data can be used to determine who will respond to targeted treatment. As OS is a very complex cancer, it has been difficult to work out what genes are driving growth and spread. We now have the ability to look at very large amounts of patient data both at the clinical level and the sequencing level (looking at genetic changes in tumours). Dr Marinoff uses this data to look for changes which would indicate if a patient was at risk of progression.

Alejandro Sweet-Cordero described the use of OS models in the lab to look at responses to treatments. Work in the lab may not reflect what actually happens in the body. His lab are developing OS models in animals, using patient tumour samples to overcome these problems.

The power of liquid biopsies

Pieces of tumours can be found in the blood. These can be used for making decisions about patient treatment. Brian Crompton described his work looking at the DNA found in these fragments, termed circulating-tumour DNA (ctDNA). ctDNA provides information on the tumour and how tumours respond to therapy over time. By looking at these fragments in blood we can detect relapse earlier.

Harnessing the immune system

Immunotherapy is a type of treatment that aims to help the body’s immune system fight cancer. Progress has been limited in OS, as the tumours stop immune cells from working properly. Researchers are looking at new ways to overcome this:

  • Emily Rav described a study combining the use of modified versions of immune cells, CAR and NK (natural killer) cells to enhance the action of NK cells.
  • John Ligon updated us on the use of vaccines containing RNA to treat metastatic OS.
  • Pradeep Shrestha provided information on a new approach to treat lung metastases with a new drug termed WP1066. WP1066 blocks a protein called STAT3 which is involved in cancer growth and spread.

There are many approaches being tested in the lab providing renewed optimism for the future role of immunotherapy in treating OS.

Clinical trials

The ultimate goal of all OS research is to provide effective treatments and improve outcomes. Once a drug has shown promise in the lab and animal studies, it may be suitable to trial in OS patients.

Lara Davis described the SARC038 trial, a phase 2 trial looking at the combination of two drugs, regorafenib and nivolumab in OS. Regorafenib is a targeted therapy that stops cancer cells forming new blood vessels. Tumours need blood to grow so blocking blood vessel formation stops cancer growth. Taking regorafenib on its own showed little promise in OS. In this trial it is combined with nivolumab, a drug that stops cancer cells from turning off the immune system. 12 patients are enrolled in this study, which is ongoing. We await the results of this trial with interest.

Other trials discussed included:

  • CAR T cell therapy (Katie Janeway)
  • A study harnessing the action of NK immune cells for the treatment of OS (Bhuvana Setty)
  • A roundup of OS trials from the Children’s Oncology Group (COG).

Clinical trials represent the final stage of the drug development process. A drug can then become available to patients more widely. They hold great promise and provide treatment options not normally available. However, the outcome of the research is unknown until the results have been assessed. The whole community is immensely grateful to patients taking part in these trials and helping to move the field forwards, towards new treatments and eventually a cure.

‘As a medical research charity it is so important for us to attend conferences like FACTOR, where we can keep abreast of the latest research, meet the community and form vital collaborations with partners worldwide’

Anna Webb, CEO Myrovlytis Trust

We left the FACTOR conference enthused and inspired. We are determined to work with all the wonderful organisations and individuals we met.

Thank you to the Bardo Foundation for funding our trip to FACTOR, enabling us to meet with the osteosarcoma community and help direct our work.

Visit our support group page to find out about organisations near you. We also have a clinical trial tool kit and trial finder.