HELP! I Need Your Opinion. To Try, or Not To Try?


To Try, or Not To Try?

That is my dilemma. I have been invited to participate in a Phase 1 Clinical Trial for a new novel class drug called CPI-613. I don’t know if I should do it or not.

As most of you know, I have been battling this stubborn and aggressive cancer called Synovial Sarcoma. I’ve had multiple surgeries in the leg and lung, radiation, and chemotherapy. The last chemo shrunk the tumours in my lung, but it’s still there. The medical community has labelled me as what I call “PIT”, which stands for Palliative, Incurable, and Terminal. Basically, there is no known cure, and there is no more conventional therapy they can give me. It also means that they (the medical community) expects me to die from this disease.

The only option left for me are clinical trials, if I chose to participate. This is where I am not sure about, particularly this new novel drug CPI-613. CPI-613 works differently than other chemo, as this drug is a targeted therapy that apparently destroys only the enzymes used by cancer cells to multiply and grow. Normal healthy cells does not use the same enzymes as cancer cells. It has been tested on animals with promising results. It has never been tested on humans, and only a handful of participants are undergoing Phase 1 trial at the moment.

Phase 1 trials, in layman terms, means that it will be the first time this drug will be tested on humans, and that the primary objective is to determine what is the tolerable amount humans can take without severe side effects, or simply put, without killing them. The effectiveness of this drug is only a secondary objective in Phase 1 trial. They will start with small dose and gradually increase the dosage until a sever side effect is experienced.

Now I’m a point-form kind of guy and I like to weigh in pros and cons before making a decision:

Pros:

  • Has shown promising results in animals, to shrink and kill cancer tumours;
  • CPI-613 is a novel class targeted drug that it is not expected to have the same toxic side effects as conventional chemo;
  • I’ll be more closely followed and would have more advanced and thorough testing than currently being done to me (frequent PET scan, MUGA scan, ECG, EKG, brain scan, complex bloodwork, etc) ;
  • I’ll be helping the BC Cancer Agency and the medical community in determining and discovering new drugs that may someday help others;
  • If effective on me, I can qualify for further testing and trials of this same drug;

Now the Cons:

  • Animal testing was only on breast, colon, and pancreatic cancer cells. They have not tested it’s effectiveness on Sarcoma in animals;
  • Has never been tested on humans;
  • Toxicity to humans and side effects have not been determined, and could possibly be severe, debilitating, permanent, and deadly;
  • The effectiveness of this drug in humans have not been determined;
  • I’ll be at the BC Cancer Agency facility in Vancouver almost daily (an hour drive from home) during the trial and will be poked and scanned alot;
  • I’ll have less precious, productive time to spend with the family;
  • I will not be able to return to work;
  • If ineffective on me, I will waste few precious months that could have been spent living my life fully;

According to the trial specialist, my last scan showed that the tumours are “relatively unchanged”. I am now stronger and able to do most of the things I used to do. I am feeling normal again, except of occasional anxiety attacks. I honestly do not want to go through the same shit I’ve gone through during the last chemo, but I’m willing to sacrifice again if the benefit outweighs the risk.

Unfortunately I doubt the benefits outweigh the risks. With Phase 1 Clinical Trials, they do not expect to have great results and most of the time, drugs don’t go past Phase 2. The probability of this drug working on me is the same probability of this cancer disappearing if I do nothing.

Do I want to be a guinea pig?

I don’t expect everyone to know what I have gone through physically, emotionally, and mentally...but let me just say that I wish it would never happen to anyone else.

There are still other clinical trials that may soon become available to me, so turning this down doesn’t mean I’ve run out of options or given up.

Please do give your honest opinion whether I should proceed with this Phase 1 trial or not. I’m confused!

Thats a hard one, i think if i were in your situation i would look into other clinical trials that have already been tested on humans. You really need to listen to your gut and if its telling you that this doesn’t sound like something you want to do, i wouldn’t do it. NEVER GIVE UP! Always research new trials and don’t let anyone tell you what to do, do what you feel comfortable with. My thoughts and prayers go out to you and your family.

I would try to get into the following study first before considering the CPI-613:

http://clinicaltrials.gov/ct2/show/NCT01112384?term=SB-939&rank=2

SB-939 is an HDAC inhibitor and there is evidence that HDAC inhibitors suppress synovial sarcoma in vitro and in vivo:

http://www.ncbi.nlm.nih.gov/pubmed/20514024

Plus, the trial is phase II which means the safety has already been assessed. This trial is also in Vancouver.

Thanks Elodie. I did mention SB939 to the trial specialist and BCCA is currently not recruiting for this trial at the moment, but it can become available at any time. I’m on their list so if there was availability they will inform me. There’s also a couple other drugs that they gave me info on and is expected to be available soon.

Wow…that is a difficult one…thinking you would have to weigh the odds…wishing you the best…Kristen

Did the trial specialist give you any explanation as why the SB939 is not recruiting right now? I read from a Torsten Nielsen recent paper that recruitment started in October. Does that mean they already accrued everybody they needed and it can only be available if someone gets out of the trial or they had to stop recruiting for some other reason? I am wondering what it means “it can become available at any time”. Does it also mean “it may never become available”?

By the way, I read that CPI-613 is based on the Warburg effect:

http://en.wikipedia.org/wiki/Warburg_effect

PET scan are an application of that effect. I know that my lung tumors haven't shown up on PET scan. Did yours show up?

Hi Elodie, thank you for the message. Regarding the SB939 phase 2 for sarcoma is active and currently active. The trial specialist told me that, but they are not yet recruiting for it. They are however recruiting for SB939 trial for phase 2 prostate cancer right now.

The other drug available for at any moment is MGCD265, another oral drug. Have you heard of this and know something about it?

Sorry for errors in grammar below, I’m typing from my iPhone and didn’t proofread it.

I had petscan in 2009 and it didnt show up then. I didn’t have petscan last year when my tumours flared up in my lung, just CTs.

I had not heard of MGCD265 before but I just googled it and found it is a VEGFR inhibitor (among other things it inhibits), same class of drugs as pazopanib, cediranib or E7080, which all showed efficiency in a number of synovial sarcoma patients. Therefore, this sounds like an interesting trial…

Khoolhandz,

I can't imagine being in your position, facing the quailty of life decisions you are. Have faith in your judgement, and conviction in what you choose, is all that comes to my mind. I got my pathology results today from a wedge resection biopsy from 2 nodules from my left lung; they came back positive for synovial sarcoma unfortunately, and it seems that I will be facing clinical trial decisions myself in the near future. I don't think I will be faced with the same situaiton you have, I haven't been labeled by anyone yet. I wish you the best, and will be following your story. Good luck, and take it easy!