Now what?

[Greg H]

Originally Posted by cheri

Thanks DanL and Greg
Well, I don't see anything on clinical trials.gov relating to AML without the prior transplant.

I have taken transplant off the table for a variety of reasons....after much thought and research. (no sib donors) And I am not up to being poisoned to near death again with a strong induction regimen......for 2 months more at most....

Cheri,

I remember your earlier posts about how chemo darn near killed you. I've done a lot more reading on MDS than on AML, so I'm not much help. But digging around on clinicaltrials.gov and elsewhere, it does seem like one sort of cytotoxic chemotherapy or another is the weapon of choice. There are some trials out there using Gleevec and even Decitabine, but most seem to involve chemo drugs -- like your Ara C -- as well. As Birgitta notes, Estybon is one exception that might be worth a look.

If your doc is truly a "little guy," it might be a really good idea to get a consult from a "bigger guy" at one of the university or teaching hospitals that's within reach -- though I understand you aren't much interested in Hopkins.

Take care!

Greg
Greg

[Greg H]

Hey Cheri!

Just ran across this article on Estybon in the MDS Beacon. This is an easier-to-read summary of the same study that Birgitta found for us.

Here are some highlights:

Results of a recent analysis confirm that treatment with the investigational drug Estybon may prolong survival in myelodysplastic syndromes patients who can no longer be effectively treated with Vidaza or Dacogen. . . .The median overall survival time for all patients was 36 weeks. The group that received 1,800 mg Estybon every day for three consecutive days every other week had the longest median overall survival time of 49 weeks. The researchers found that 54 percent of patients achieved a complete bone marrow response or a greater than 50 percent reduction in the number of immature bone marrow cells. The median overall survival time for these patients was 44 weeks. Patients who achieved stable disease with Estybon (38 percent) had a median survival time of 40 weeks. Patients who experienced disease progression (8 percent) had a median survival time of 16 weeks. According to the study authors, Estybon was well tolerated. They did not observe any cases of blood-related side effects.

Good luck!

Greg

[Neil Cuadra]

I hope the Estybon trials continue to show good promise.

Estybon is the trade name for rigosertib and there's a forum thread about Estybon here.

[cheri]

Hi everyone~
Sorry for the delay in responding....it's been crazy! With less than 24 hours notice, I went on a whirlwind trip up to New Brunswick NJ to Cancer Institute of NJ--part of Robert Wood Johnson Hosp....entire staff was awesome!
My Dr down here set me up on a STAT visit with the next level institution ( Greg) where they actually do have access to clinical trials...the new Dr squeezed me in as his only patient yesterday!!!!!! Friday of 4th of July weekend! Amazing!

He mentioned that phrase "smouldering" as my counts are low (not raging high WBC) and still almost borderling MDS/AML at 27% blasts....he felt that I might qualify for 2 clinical trials.....don't know that now that I have crossed that line that Estybon would be an option.....I will ask, though....

#1-Long story short, and before reading the fine print, they are doing a clinical trial at CNIJ which would involve a short hospital stay for the first dose; once a week as outpatient for 3 weeks, then 2 weeks off....5 week today cycle for 3 cycles.......steriods and aspirin to combat side effects....24 hrs pre and post treatment.....
This is in the 2nd phase of this trial which has shown promise in China.......I will investigate and give more info later.......

Option #2 was an oral medication that is sponsored by a drug company....I think everyone feels that option #1 is better--hey, it's their study! But I believe that it may be the most realistic for results...... the Dr I was assigned and also my local Dr's mentor are directly involved in this.
I am at my healthiest since my first induction and feel physically up to another type of treatment, as my options are severly limited at this point........and it's better to be treated while before I get really sick. I feel that this a doable treatment I can live with while ever so stubbornly trying to maintain a decent quality of life. (hello, it IS summer after all! ...

This new doctor agreed that since my first course of treatment was so devastating, that more induction chemo or transplant would be risky....I feel like he is treating me as an individual, which I appreciate.....nothing about me has followed a typical path thus far and truthfully, if I can be a help to future generations by participating in a trial to help stamp out these horrid blood diseases, I feel that in itself is a worthy contribution.

I also sing the praises of Marrowforums to health professionals (often getting a raised eyebrow) but once I tell them how professionally run it is and what an awesome source of support and info it is--- I hope they will check it out...I cannot tell you all how glad I am to have been a part of this so that when I got this latest round of "difficult" news, I feel that I have advocates fighting for me that I do not have in my real life! Loved ones care, but they are clueless as to how to find information that you all are so great at getting!

I pray for us all and will continue to keep you posted the more I learn.

Have a great 4th weekend and enjoy every moment!

[Greg H]

Hey Cheri!

Boy, you have made my day today! I am so excited to hear that you have found some options -- and it's fantastic that the folks in the Cancer Institute have a connection to your local doc, because I know that you really get on with him.

This is really outstanding news. I hope you have a great Fourth - and I'm sure it will be a much better one know that you have a good course of action in front of you.

Definitely keep us posted. I'm very interested to find out the names of these new treatments.

Take care!

Greg

[cheri]

Greg! That message made me smile big time--thanks for the awesome encouragement!

In case anyone is interested, I will give you the info in case you want to investigate it....maybe you have relatives nearby...rest assured I will keep you all informed on this new journey....not sure what I am allowed to divulge legally...anyone know about any info restrictions?

Just go on the Cancer Institue of NJ website--www.cinj.org
and type in AML under the clinical trials....2 will pop up...
The first is Phase II study of TPA...etc Protocol #020702....see what you think....(#2 is a pill by a drug co....not at this time...).
Sorry I can't provide more info at the moment--I am on my way out of the door to run my 1st and last campfire program at the park job I was forced to quit since I got this last news....plus, my platelets are not available to me for at least 36 to 48 hours.....I needed them yesterday....literally.......

Well, here I am-- living on the edge, the verge and still standing.....

[Paula W.]

Hi Cheri,

Your story is kind of like my Moms. She is on a trial in NYC w/ Dr. Azra Raza. The drug is called Onconova. She tried vidaza, at first it worked then, it stopped. She is on this trial and her blast is now normal. She is still platelet and HG dependent though. It may be worth talking to Dr. Raza about being part of the trial. Let me know if you need any information. Best of luck to you. Keep the faith.

Paula

[Greg H]

Hey Paula!

I'm so glad that you chimed in here. I have actually been wondering about how your Mom is doing on her trial -- I recall that it was pretty rough sledding at first.

I'm glad to hear her blast count is down. Do the docs expect continued improvement in platelets and/or HgB?

Take care!

Greg

[cheri]

Hi Paula!

I thought we had communicated before and yes we did, back in Jan and Feb~ I still have our PM's!
I never really did well on Vidaza--my numbers were always down and I've always needed platelets and often blood.
Did your Mother start with MDS or AML?
The clinical trial I am considering is specfic to AML, which is why it is so appealing.....Also, my insurance doesn't cover NY, so I wonder if they would cover any parts that the trial didn't cover..I appreciate your input...I am going to gathter that Estybon information for a later date and may contact you....
SO glad to hear your Mom is hanging in there!

[Birgitta-A]

Hi Cheri,
TPA found in croton oil has been used in Asia during a very long time for different diseases for example haematological cancer. The only reference I can find where they use TPA + Dexamethasone + choline magnesium trisalicylate is this Patent Application: http://www.freshpatents.com/-dt20110...0110034425.php

RO5045337 is made by Roche. This drug inhibits the oncoprotein (cancer protein) MDM2. MDM2 is overproduced in many human tumors. MDM2 impairs p53 function. P53 is a very important tumor suppressor gene. http://www.cmod.org/images/CMOD_Pres...017%202010.pdf

Both studies seem to be interesting. Hope you will be able to participate in one of them!
Kind regards
Birgitta-A

[cheri]

Birgitta "A"
"A" is for AMAZING!
How do you find this stuff AND on such short notice?

Yes, the TPA combo clinical trial, mentioned in that patent, is the one I am applying for....75% sure until I talk to my doc and I will give him a copy for his information. The Dr I met with is the first name on the list! (he is a researcher first)
It seems that all signs are pointing to this, so I will give it a try...more info later...thanks!

[Birgitta-A]

Hi Cheri,
Thank you for the kind words ! You know I like to learn more about our disease and treatments so when you tell us about a new trial I just google it. This trial is a mix between a very old drug and two modern components - interesting. Hope you will get the drugs and have a positive response !
Kind regards
Birgitta-A

[cheri]

Hi Birgitta
From what I understand, the steriod (dexamethasone) and aspirin (choline magenesium trisalicylate) are what they found to use to counter the side effects from Phase I....taken on both sides of transfusion for 24 hours of TPA dosage....the fact that the TPA has been around a while is encouraging and especially with the fact that both the steriod and TPA have shown anti Leukemic properties........
All I can do is try...and hope!

[Birgitta-A]

Hi Cheri,
Yes, we hope that dexamethasone and choline magenesium trisalicylate will decrease the adverse effects of TPA at the same time as they increase the counts. Salicylate can decrease platelets - hopefully dexamethasone will increase them.
Kind regards
Birgitta-A

[cheri]

Hi all

Birgitta--When I asked about the aspirin component, the DR said that there was no platelet ramification with this compound....interesting. And yes, the Dexa is supposed to have a positive effect.

I will be meeting this weekend with the Doctor who is in charge of this trial. Spoke with him last nite and get great vibes, and need the lowdown....
I will find out how much I can actually divulge here! (anyone ever have any restrictions on what they can say about their clinical trial? Greg H?)

It seems there have not been a lot that have gone before me in this instance... Do I take the chance to be a pioneer? Do I have a choice?

Weird happening over the weekend....I had a platelet count of 28 last Thursday, so they started the search for my platelets, which would have carried me over the holiday weekend. Friday I had a CBC and they were 16 but I had no way of getting those results. The plan was to go to ER if I started bleeding....As of Tuesday, STILL none could be found...I was scared to death, waiting to have breakout bleeding of some type, as my platelets have been known to drop 10k per day.....not a lot of wiggle room. Had a CBC Tuesday and my platelets were at 30..... Thank GOD!

Thankfully, they held at a time when HLA wasn't available, but just a reminder as to what a scary tightrope walk these blood diseases are!
All I need is one little miracle at a time!

[Greg H]

Originally Posted by cheri

I will find out how much I can actually divulge here! (anyone ever have any restrictions on what they can say about their clinical trial? Greg H?)

Hey Cheri!

They will likely provide you with several pages of legal mumbo-jumbo to sign when you enter the trial, and that should have stuff in it about disclosures -- if there are in fact any restrictions. I'd be surprised if there are any, since they have to register their trial with the FDA and all that sort of stuff.

But my trial is with a branch of the federal government, using a drug developed by a university, which probably makes disclosure less of an issue than a trial that involves a drug company, if yours does. You'll probably want to ask them. Tell them we all want a blow-by-blow account, and we're their best source of potential customers, so they should let you fill us in!

On your platelet experience, I am sure glad that turned out okay. One of the things I have begun to suspect about my platelet ups and downs is that the number depends, to some extent, on how thorough the lab is that day. I often have notations about large platelets and giant platelets -- and often a second note with that indicating the lab did a manual recount of the sample.

But sometimes, like with my last result -- not from my usual lab -- there's no notation -- and often a lower count. My suspicion -- though I don't know this for sure -- is that the automatic platelet counting machine doesn't like large and giant platelets, and so it leaves them out of the count.

If anyone else has any insights into this, speak up.

Good luck Cheri!

Greg