Clinical Trials: they are not designed only to be a “last resort”

Thank you for posting. So agree! This is why the crossroads of “standard of care” vs trial is a difficult one for all of us.
Dig as deep as you can for measurable data and even if it is very promising, have plan B in place.

And always, get multiple opinions.

I just wanted to add a caveat that, although clinical trials are not "only intended to be a last resort," they should not be taken lightly.

In some trials, you may receive the standard of care or the standard of care plus a trial drug/therapy. For other trials you have to be completely off all other drugs/therapies to test only the trial drug by itself.

I've been Stage-IV PDAC for almost 2 years now, but with pretty good control over everything. After 15 months on SoC therapy (Gem+Abrax+Cis), we started seeing signs and hints (but not definitive evidence) of drug resistance. My neuropathy was getting worse, my bone marrow and bloodwork were taking a beating, I had a break in my work schedule, and an opportunity to participate in a very promising, cutting-edge trial.

Two independent oncologists agreed it was good timing and the right thing to do, so I jumped through all the hoops and was accepted into the trial.

The trial required at least 4 weeks washout from any previous chemo, followed by 7 weeks in the treatment / observation phase. Insurance issues, travel, and other logistics added about 2 weeks, making a 13-week (3-month) period off chemo before I got final results.

Unfortunately, the treatment did no good at all. Cancer grew and spread during that time period instead. We were able to revert to and resume my previous chemo within two weeks of those final results, but the tumor growth didn't just stop on a dime. The primary tumor continued to advance, and within two weeks, landed me in the hospital with an intestinal blockage where I am today, awaiting placement of a stent to open the blockage so I can eat again.

---

In hindsight, a few months before the hints of drug resistance developed, one of my oncologists said I was responding so surprisingly well to the GAC for so long that it would border on unethical to go off it for a trial of unknown efficacy. Although I did reach the point where the trial made sense, the "unknown efficacy" part (which turned out to be zero) was a real disappointment.

Phase-1 trials offer a lot of innovative and promising treatments, but their primary purpose (along with testing effectiveness) is to identify dose-limiting toxicities and recommended dosing for Phase-2 trials. Phase-2 seems to be a sweet spot somewhere between Phase-1 (being a guinea pig) and Phase-3 (risk of placebo).

I thought this was important enough to share. I hope my lesson from this is a benefit to someone!

Che tipo di sperimentazione hai fatto? RMC 6236?
Maria

Thanks very much! I look forward to more news on how your trial is going, as KRAS G12D is next on my targetable mutation list.

I just wanted to add a caveat that, although clinical trials are not "only intended to be a last resort," they should not be taken lightly.

In some trials, you may receive the standard of care or the standard of care plus a trial drug/therapy. For other trials you have to be completely off all other drugs/therapies to test only the trial drug by itself.

I've been Stage-IV PDAC for almost 2 years now, but with pretty good control over everything. After 15 months on SoC therapy (Gem+Abrax+Cis), we started seeing signs and hints (but not definitive evidence) of drug resistance. My neuropathy was getting worse, my bone marrow and bloodwork were taking a beating, I had a break in my work schedule, and an opportunity to participate in a very promising, cutting-edge trial.

Two independent oncologists agreed it was good timing and the right thing to do, so I jumped through all the hoops and was accepted into the trial.

The trial required at least 4 weeks washout from any previous chemo, followed by 7 weeks in the treatment / observation phase. Insurance issues, travel, and other logistics added about 2 weeks, making a 13-week (3-month) period off chemo before I got final results.

Unfortunately, the treatment did no good at all. Cancer grew and spread during that time period instead. We were able to revert to and resume my previous chemo within two weeks of those final results, but the tumor growth didn't just stop on a dime. The primary tumor continued to advance, and within two weeks, landed me in the hospital with an intestinal blockage where I am today, awaiting placement of a stent to open the blockage so I can eat again.

---

In hindsight, a few months before the hints of drug resistance developed, one of my oncologists said I was responding so surprisingly well to the GAC for so long that it would border on unethical to go off it for a trial of unknown efficacy. Although I did reach the point where the trial made sense, the "unknown efficacy" part (which turned out to be zero) was a real disappointment.

Phase-1 trials offer a lot of innovative and promising treatments, but their primary purpose (along with testing effectiveness) is to identify dose-limiting toxicities and recommended dosing for Phase-2 trials. Phase-2 seems to be a sweet spot somewhere between Phase-1 (being a guinea pig) and Phase-3 (risk of placebo).

I thought this was important enough to share. I hope my lesson from this is a benefit to someone!

@aristidina
Yes, I am on the doublet trial of RMC 9805 and RMC 6236.

These target KRAS mutations.

@markymarkfl -was your a BRCA trial? Wishing you the best on your search for next steps

gamaryanne: I had some communications with Markymarkfl back in June and it was Trop2 protein at HD Anderson in Houston; stem cell approach. I know he also has ATM and KRAS12D mutations as I do and CHEK (which I don’t).
I’m also wondering if you will need to go through a phase 3 trial that might include a placebo?

@markymarkfl
I had the great opportunity to talk with Dr Daniel Von Hoff this past weekend. His lab developed Gemcitibine and spoke about other combos that are being tested with it. Since you had such good response for awhile with it, you may want to reach out with your distinctive case. He, or his close colleague Dr Erkut Boranczanci may find this very interesting and have answers that others may not yet be aware of. Just a thought.
Keep fighting!