The Colon Club

As I understand it, Mayo Clinic in Rochester MN is one of the clinics participating. If they are, my guess is the other Mayo Clinics probably are too.

is Regorafenib indicated for patients who are refractory to other lines of therapy or can newly diagnosed stage IV patients benefit from this drug as well?

SoConfused wrote:is Regorafenib indicated for patients who are refractory to other lines of therapy or can newly diagnosed stage IV patients benefit from this drug as well?

So far I think the testing has been done on Stage IV who have run out of chemo options. I don't think there is good data on it as a first-line treatment yet.

Kathryn's correct. They were recruiting for a Phase II clinical trial that compared Regorafenib head to head with, and in combo with, both folfox & folfiri, and there was going to be one arm that tested how KRAS-mutant patients responded, too. I don't know if it's still recruiting, but it comes up in clinicaltrials.gov if you search for regorafenib AND colorectal cancer.

The phase III trial that ended early was for patients who'd progressed on all other approved treatments, and it was regorafenib alone. The expanded release programs are often run like a mini phase III+ clinical trial - the docs/clinica have to sign confidentiality agreements, appoint a lead investigator, send regular progress reports to the main Mayo principal investigator, and may even have to do scans/tests ordered on Mayo's frequency. The data will all go toward the final FDA drug approval finding.

This is exciting (I hope). Getting it out to people within six months of unblinding a phase iII study means Bayer has its ducks in a row.

Gaelen, can you expand a bit on what an expanded access program is? When I asked the oncologist about this drug, he said we may have access through one of these programs, but I don't understand what that means. Thanks!

I am on Xeloda and Avastin. I am KRAS Mutant and so I am running out of options. My latest CT Scan showed a slight increase in the size of my mets in both lungs. My oncologist talked about this drug and seems pretty excited about it. I talked to him about the drug maybe extending life by only a month or so. He said that was just an average of a lot of patients. On some patients it worked very well. So I guess I just have to hang in there because he said I would be a perfect candidate for it. So I'm keeping my fingers crossed and will let everyone know how this whole thing goes. I wonder about what kind of side effects there are; I haven't been able to find out any information.

Never give up hope. You never know what's around the corner. Keep Fighting!!

God Bless
Patti

abuttigi wrote:Gaelen, can you expand a bit on what an expanded access program is? When I asked the oncologist about this drug, he said we may have access through one of these programs, but I don't understand what that means. Thanks!

I'm going to default to Wikipedia, here, even though it's not usually one of my preferred sources. However, this article compiles the FDA rules for expanded access and a description of what the process is from the drug company, physician, and insurance angles all into one pretty good summary.

The key thing is that for most expanded access, the patient will have to be Stage IVE, have had to have progressed on all standard therapies, and have some factor which makes the patient ineligible for a standard clinical trial in order to qualify. And the physician administering the Expanded Access program will have to collect data and report it to the drug's principal investigator just as if the patient were participating in a clinical trial. The drug's final approval is conditional upon the proper reporting of this off-study, pre-drug-approval data.

Any clearer?

Thank you all for the information and for deciphering what it all means. You had me at HOPE.

Gaelen wrote:

abuttigi wrote:Gaelen, can you expand a bit on what an expanded access program is? When I asked the oncologist about this drug, he said we may have access through one of these programs, but I don't understand what that means. Thanks!

I'm going to default to Wikipedia, here, even though it's not usually one of my preferred sources. However, this article compiles the FDA rules for expanded access and a description of what the process is from the drug company, physician, and insurance angles all into one pretty good summary.

The key thing is that for most expanded access, the patient will have to be Stage IVE, have had to have progressed on all standard therapies, and have some factor which makes the patient ineligible for a standard clinical trial in order to qualify. And the physician administering the Expanded Access program will have to collect data and report it to the drug's principal investigator just as if the patient were participating in a clinical trial. The drug's final approval is conditional upon the proper reporting of this off-study, pre-drug-approval data.

Any clearer?

Yes, thank you! One last question... if I'm being a pest let me know. As far as I understand, the patient has to be out of options to do this trial. Does that mean that he/she cannot qualify for other trials being offered at the cancer center? For instance, my dad's onc brought up an immunotherapy trial that my dad would qualify for. Do we have to pursue that one before an expanded release Regorafenib trial?
Sorry for all the questions, I just want to make sure I'm working within bounds when I talk to the oncologist and present this to my dad.
Thank you!!

My understaning of expanded access is that usually the drug is only available to stage IV patients who have no other drug options. That's the basic process, but there may be rules specific to regorafenib that aren't clear yet because the program hasn't been formally opened yet.

Thank you Gaelen for your usual concise thorough posts, always count on you for true.
pollo 65

Gaelen wrote:My understaning of expanded access is that usually the drug is only available to stage IV patients who have no other drug options. That's the basic process, but there may be rules specific to regorafenib that aren't clear yet because the program hasn't been formally opened yet.

Okie dokie. I'll be sure to write the question down and ask the onc over Spring Break. Thank you so much!!

I thought I had posted in another thread that the drug was "supposed" to be available in April. Perhaps I forgot. From what I've heard (though the grapevine) is that the first place most likely will be at Mayo in MN. If the old chemo doesn't work for me after a year and a half of being off of it I will try for it unless my health is to much worse. Avastin's life expectancy increase in only a month or two, but some seem to get a remission with it. Avastin for me wasn't good, the aches and pains in the joints and muscles weren't worth it after a while, but a lot of people don't get that. I think it's great not good to get another drug in our arsenal.

I'll still keep praying we get a cure to cancer, but any drug that saves even one more person or gives some more time is good for now:)

I know my ONC will probably want me to start thinking about Avastin again, but Terry, like you, I did not do well on it with the side effects. I'm reading Regorafenib's additional average life was also a few months. But, Avastin works for some...so maybe Regorafenib will work for us and we will be way above average. I'm always so impressed how you keep fighting. Right now I'm "stable", which isn't bad, but isn't good either. If my numbers aren't down this week, I'm pretty much going to be out of options. I was talking to someone today that I don't like this chemo life. All I do is work, doctors appts., and sleep. I was reminded, except for the doctors appts., a lot of people live this kind of life. With the economy what it is, I know many people with two jobs. So, that did help to put a different slant on things - maybe my life isn't so different from the masses. It is certainly different from my life before DX, but it's still a pretty good life. At least I love my job and my friends are awesome. So many people hate what they do and are alone.

Gaelen do you know that if Mayo (for example) has this drug for compassionate use, would we have to go and get it there every couple of weeks or would they make sure our onc. gets it and we just go there possibly monthly or every other?