Cure Alveolar Soft Part Sarcoma International (iCureASPS)

FYI, I spoke with Beth Fox today, just gathering information. They have just about close the Phase I trial - and nearly decided the dose that is recommended, but there are a few spots left for the pharmacokinetics study on Cediranib and very encouraging there will be a Phase II trial for Cediranib and ASPS (as well as other sarcomas) in kids likely to open in the next 6 to 12 months.

I also found out more about the process of doing trials when you are out-of-state...they will pay for the travel of 1 parent and the child (airfare) and also free stay in Childrens Inn. What is nice is that the Cediranib trial is completely outpatient. Dr. Fox sounded as if they really work hard to make it easier for out-of-state families to do trials there. They need a visit of 7-10 days in the beginning, then day 28, but then if all goes well, just require a flight every 2 months (2 months of med refills). They are fine with managing dose adjustments with the local oncologist.

Another very encouraging thing - she also said Astra Zeneca thinks they will be applying for FDA approval in the Fall. So Cediranib may be commercially available in the coming year. When Afinitor (mTOR) was approved, it took 8 months from submission to FDA approval.

All important to keep in mind as the side effects are not trivial - and the timing of when to start it might be important.

Dr Fox also told me that there have been 2 kids on the Cediranib trial at the NCI - an 11 year old and a 16 year old. The 11 year old is still on it (after 7 cycles) - and clinically has improved (less oxygen requirement) and minor shrinkage (10%). The 16 year old came off after 4 cycles because her large lung tumors (5-6 cm at start) got larger by the 4th cycle (8 cm or so).

In kids there does not seem to be bad fatigue - at the optimal dosing, but she said kids tend to have pretty severe bowel cramps at least once a day followed by diarrhea.

I am wondering what about adult cediranib phase 2 trial in ASPS or they are going to incl. young adult into the kids trial? I think that I have seen that done for other drugs in ASPS since it is obvious that it can facilitate the enrollment greatly. And what disease Astra Zeneca will be seeking the FDA approval for? I have not seen any consistent systematic response in other cancers (or any studies phase 2 being completed for that matter) except ASPS - are they going to apply for cediranib being approved for ASPS indication specifically?

To see when a new drug might make it to market I usually click on the links for shareholders on the company's page. Astra Zeneca will apply for NDA and MMA at the same time (MMA gets you Europe and Canada I think). They anticipate approval in the second half of 2010, which fits with what Dr. Fox told me.

Cediranib will gain approval for glioblastoma multiform (brain) and colon cancer. I have seen some of the data for glio and I think they will get approved. It is something to consider as the availability currently is not so hot.

For our part if we can, we will try less toxic agents first. 'K' is pretty tough with surgery, but we worry it would make her feel sick if she had bad cramps and diarrhea once a day. It is such a complex decision regarding all these meds!