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 Rigosertib trial: Q&A

Rigosertib trial: Q&A

What is Rigosertib?

Rigosertib is an investigational drug. Laboratory research suggests that it can inhibit tumour growth in EB cancer while leaving non-cancerous skin cells unharmed.

Is Rigosertib available to use?

Rigosertib is not currently available to use. Further clinical trials like this one are needed to make sure it is safe and effective to use. Rigosertib is an investigational drug currently being trialled for a number of cancers and it does not yet have a licence in any therapeutic area.

Who will supply Rigosertib to use in the trial?

The pharmaceutical company Onconova, who manufacture Rigosertib, will supply the drug for the trial.

If you already know that Rigosertib inhibits tumour growth, why are further trials needed?

Laboratory tests suggest that Rigosertib inhibits tumour growth, but it still needs to be tested in humans to check that it’s safe and effective. Clinical trials are needed to make sure that a treatment is safe and works as planned in a suitable number of patients. Once this information can be confirmed, treatments can be made available. This can take quite a long time.

What will this trial investigate?

This exploratory study will:

  • Examine the safey and effectiveness of Rigosertib in treating cancer caused by EB
  • Investigate why Rigosertib affects EB cancer cells but not healthy EB skin cells
  • Collect genetic data from individual participants and look at individual responses to Rigosertib to inform further studies and potential future treatments

Where will the study take place?

The study will take place across three sites: Thomas Jefferson University, Philadelphia (lead by Dr Andrew South), Paracelsus Medical University Salzburg, Austria (lead by Dr Johann Bauer) and St John’s Institute of Dermatology, St Thomas’ Hospital, London, UK (lead by Professor Jemma Mellerio).

When will the trial start?

This study is in its very early stages. Patients will be recruited in the coming months.

Can I take part?

The team aims to recruit six to ten people with Recessive Dystrophic EB who have late stage skin cancer (Squamous Cell Carcinoma) caused by EB that has not responded to standard care.

To take part in any type of clinical trial, the first person to speak with is your own EB doctor. They can talk to you about the trials that they may be taking part in and assess if you may be medically suitable.

To learn more about EB research, please join the DEBRA International Research Involvement Network. 

What’s different about Rigosertib?

This trial will be the first time Rigosertib has been tested in humans suffering from EB.

The therapies currently on offer to treat EB cancer are really limited in effectiveness. The great thing about Rigosertib is that it appears to inhibit tumour growth in EB cancer while leaving non-cancerous skin cells unharmed, and could potentially be effective in treating cancer caused by EB that has not responded to conventional treatments.

Why is this research important?

Aggressive cancer can be a devastating complication for some people with Recessive Dystrophic EB and sometimes other types of EB. An effective treatment for this ultimately fatal cancer may transform the quality of life for people living with the condition who may develop this kind of cancer. It is important to note that not all people suffering from EB will develop skin cancer.

Dr Andrew South, who’s leading the international research team carrying out this project, says: “New medicines are becoming available that target particular features of a given cancer, such as inhibitors that target a certain mutation. This DEBRA funded initiative will be the first clinical trial of a so-called “targeted” cancer therapy that is based on scientific data obtained from studying Squamous Cell Carcinoma in EB. We are extremely excited that this new drug could be offered as a potential treatment.”

Is this a cure for EB?

No. Laboratory research suggests that Rigosertib inhibits tumour growth in EB cancer while leaving non-cancerous skin cells unharmed. This is a potential treatment for EB related cancer, not EB itself.

If successful, this trial could be a first step in finding an effective treatment for the aggressive cancer that can be caused by Recessive Dystrophic EB. An effective treatment may make a real difference to the quality of life for people facing this cancer.

DEBRA is committed to funding pioneering research for symptom alleviation and, ultimately, cures for EB.  We’re also dedicated to providing care and support to the entire EB Community here and now.

Find out about other research projects we’re funding and learn more about how we can help you. 

I want the technical nitty gritty about this DEBRA funded trial – where can I find our more?

Read our detailed summary of the trial here