New drug ESTYBON for Vidaza and Dacogen resistent patients
 

Hi all,
Now the new drug ESTYBON (ON 01910.Na) will be in trials for high risk MDS patients have relapsed or become resistant to Vidaza or Dacogen:
http://checkorphan.getreelhealth.com...drome-patients
Kind regards
Birgitta-A

Brigitta,
What do you know about this drug? My mother has high risk MDS 13% blasts in bone marrow. She is currently transfusion dependant and has needed platelets since being on Dacogen. Neither Dacogen nor Vidaza really did anything for her. Her doctors would like to start her on this, but I don't know much about it.
Suzanne

ASH Abstracts on Estybon
 

Hey Suzanne!

There are some presentations on Estybon scheduled for this weekend's American Society of Hematology conference.

One presentation seems to link them all up into a summary, based on research done at St. Vincent's, Mt. Sinai, Stanford, and the NIH.

You can find the abstract here:

http://ash.confex.com/ash/2010/webprogram/Paper27358.html


Out of 48 patients, 13% had what the researchers term a "bone marrow complete response" (blasts go down 50% and stayed below 5% for at least four weeks) and another 27% saw their blasts decreased by half within 4 to 8 weeks. That 40% of patients lived significantly longer than the folks who didn't have such a dramatic response in their marrow.

Median survival among the 27% was 68 weeks. Five of the six who had complete response were still alive at the time the report was prepared.

The ASH abstracts include a couple of reports from the participating research centers:

http://ash.confex.com/ash/2010/webprogram/Paper34755.html

http://ash.confex.com/ash/2010/webpr...aper29141.html

One of these is at Stanford and the other at Mt. Sinai in NYC. Each appears to have treated ten patients and seen a response in five (50% response rate). That includes a couple of folks whose marrow returned completely to normal, as well as other folks who just saw improvements in blood counts.

The patients enrolled in these studies are older (median age 75 in one study and 80 in another), have more aggressive MDS, and have either failed to respond to or relapsed from treatment with Vidaza or Dacogen. In other words, these trials involve some tough cases, and that may lower the response rate to the drug.

A 50% response rate is about what you can expect with Vidaza, so it's nothing to sneeze at.

I gather that the way the drug works is to actually kill cancer cells (the abnormal MDS stem cells in this case), while not killing normal cells. The papers don't report many nasty side effects, though both mention a case of painful or difficult urination, both mention fatigue, one mentions nosebleed, and the other mentions anorexia.

Part of the goal of these trials was to figure out the dosing, so it looks like they tried a variety of schedules and it's not clear whether they've nailed down the best option. It's an IV drug and they were giving it to patients anywhere from 2 to 6 days a week, in some cases every week and in some cases every other week or even monthly.

The Leukemia and Lymphoma Society is helping underwrite research on the drug, so they evidently think it has some promise.

Hope that helps. Good luck to you and your Mom.

Greg

Greg,
Thank you so much for your response! I am hoping that this works for my mom because so far Vidaza and Dacogen have done nothing and her blast counts are up to 13% now. She is now both red blood cell and platelet transfusion dependant, receiving 2 units of RBC every 2 weeks and platelets are needed. Another factor is her lymphoma might be back and I don't know if they will let her in the study if it is. I don't know what alteratives are left at this point. Editing to add my mother is 69 years old.

Estybon
 

Hi Suzanne,
Sorry, I didn't see your post about Estybon but you have got a very good answer from Greg. Hope your mother will have a positive effect if she is getting the drug!
Kind regards
Birgitta-A

I am curious if this drug can be used for MDS in transformation or AML. Dr. Raza (she is a research specialist for MDS) is the doctor that is recommending we move forward with this protocol even with the latest curve ball that has been thrown our way. Her hem/onc is a very big leukemia doctor who also deals with a lot of MDS (both doctors kind of work together consulting each other) he feels we should move forward with tried and true Induction Therapy. Is this drug used for AML? Would you go that route or go with the standard drugs.

I can't help but feel if we would have had the port put in sooner and got moving on this quicker maybe we wouldn't be where we are right now. I can't beleive how fast this disease turns on a dime.

Estybon
 

Hi Suzanne,
Yes, if you look at the abstracts from ASH 2010 that Greg has posted you will find that there are AML patients that have been treated with Estybon. I don't think the results seem to be impressive - perhaps your mother should follow the advice of her hem/onc and try Induction Therapy.

I am not sure that your mother fullfil the criteria for AML yet - she has to have 20 % blast cells or more in the bone marrow but she is near the limit now.

Hope she will have good effect of the therapy she will choose!
Kind regards
Birgitta-A

Suzanne,

Please let us know what your mom decided to do. My Mom is having a port put in tomorrow, 1/13/11 and will start Oconova (ESTYBON) on Monday. Would love to know how your Mom is doing on the drug. I will post as well. God Bless both of them.

Paula

Estybon
 

Paula,
Suzanne's mother suddenly passed away 4th Jan.
Kind regards
Birgitta-A

Oh how sad.... Thank you for the update Birgitta. This disease has a mind of its own it seems. I was hoping to be able to compare Suzanne's mom's progress with my Mom's. Looks like God had another plan.......:(

defective chromosomes returned after bmt and vidaza
 

I'm new to this list. I had a bmt in July and had week long treatments of vidaza in sept. and Oct. but they were then stopped because tests on cells from Oct. 17th biopsy showed that 3 cells of 20 showed abnormalities in chromosomes. My doctor told me this on Dec. 3rd and I was given more of my brother's stem fa cells on that day with the hope that I would acquire enough graft host disease that they would kill of my defective cells. I go back to the doctor tomorrow. So far, I haven't noticed any sign of rash or any other sign of a reaction. My doctor at U.Iowa has told me that if my brother's cells don't kill off the defective cells then my cancer will return.
I've been reading about estybon and would like to know how I could get into one of the clinical trials. I would also appreciate any other info. regarding my condition and what to expect and what to do.

Thank you,
Richard

Richard,

I assume you had an MDS diagnosis. Is that correct?

I hope your brother's cells take over as your new immune system. If not, and you want to look at trials, I suggest you review the general information at the NIH website, look at the trials listed at clinicaltrials.gov, and then contact Leigh Clark, Patient Educator at the AA&MDSIF, for advice about getting into a trial. Her contact information can be found on the Foundation's Clinical Trials: Are They Right For You? page.

Tip: Since Estybon is the trade name for rigosertib, use the word "rigosertib" when you search clinicaltrials.gov.

thanks for info
 

Yes, I have MDS. Thanks for info. regarding clinical trials. What does AA&MDSIF stand for?

It's short for the Aplastic Anemia & MDS International Foundation. They are the primary organization dedicated to helping and advocating for aplastic anemia (AA), myelodysplastic syndromes (MDS), and paroxysmal nocturnal hemoglobinuria (PNH) patients. Marrowforums has the same focus.