Transcript
My name is Dr Claire Verschraegen. I'm a professor at The Ohio State Comprehensive Cancer Center. I'm the chief of the medical oncology division. My specialty is really rare tumors and drug development.
I've participated in a lot of studies trying to find new treatment for cancers that don't have any, or for cancers that don't have any curable therapy.
Thank you very much for asking me to discuss a couple of abstracts that were presented at ASCO. ASCO was really a different type of meeting this year, with a video meeting. It's not really the same as what I was used to for the many years that I went to the ASCO meeting.
I was asked to discuss a couple of abstracts on the very rare, kind of quasi-benign condition called tenosynovial giant cell tumor (also known as pigmented villonodular synovitis) which is usually a swelling with an inflammatory component.
The one I have treated were around the knee, but other joints can also be affected. It's painful, it can mimic some cancer conditions at times, and sometimes it's a tumor that's between malignant and benign.
Sometimes, it can metastasize and be fatal in very rare cases. The first abstract was by Maribel Salas, and it's really the evaluation of a new treatment that came up, and it was approved by the FDA for this condition, which is called pexidartinib.
The pexidartinib targets the fusion gene that is known to cause this condition, which is a fusion between the CSF1 receptor and the collagen gene, the collagen being part of what really builds this tumor, and the CSF1 being like the transcription factor that will stimulate the division of, or the production of the collagen.
Pexidartinib specifically blocks the CSF1 receptor, tyrosine kinase receptor, and is a drug that is an oral -inib, like many others that we are now using for various cancer. But it does have some liver toxicity, so it's really only indicated for the very advanced stage of the disease.
On the early stage of the disease, this is still a surgical condition that surgeons will treat, and patients will just get through it most of the time. The main side effect is liver toxicity, which caused the black box on the prescription notice when one wants to use this drug to treat patients.
To really look at how the treatment was tolerated, the group of Maribel Salas looked at the evaluation of patient and healthcare provider when they knew what their attitude and then their behavior was regarding the intake of this medication.
It's a very, very small study; only 12 patients were put in this study and 12 healthcare providers. All were male (I'm not quite sure why, because there's a lot of females with this condition), and the main area for the patients was the complexity of understanding the mechanism of action, the side effects, and the wordiness of consent form and information about drugs.
For their healthcare provider, was it basically almost a similar complaint, that the information about this drug was a little bit too verbose and difficult to follow. I think the company wants to understand that to come up with a better-informed consent or information about the drug.
We'll see if the material changes a little bit once they review this information. It was a qualitative research, and so it's not really something very scientific. It's really to understand the perception of what people think about the condition and the drug.
