Onconova Trial

[Paula W.]

Just wondering if anyone has heard of or has been on a new trial called Onconova. It's a trial for MDS patients whose other chemo (i.e. Vidaza) has failed. It sounds promising, just wondering if anyone had any info on this.

I'm taking my Mom to Mount Sinai in NYC next week to meet up with the trial Doctor.

Thank you for any info.

Paula

[Birgitta-A]

Hi Paula,
Here are results from a small study with Estybon from Onconova. The patients had tried hypomethylating drugs like Vidaza or Dacogen before treatment with Estybon.
http://ash.confex.com/ash/2010/webprogram/Paper34755.html
Kind regards
Birgitta-A
71 yo, MDS Interm-1 dx May 2006, tx dependent from dx, Desferal and Exjade for iron overload, Neupogen for low WBCs, Thalidomide and Prednisone since June 2010 with good effect

[Greg H]

Hey Paula!

A number of medical centers are involved in trials of this drug, tradename "Estybon," technical name "ON01910.Na."

I don't think we've run across anyone on Marrowforums who has enrolled in the trial yet. But you'll find some additional information from the American Society of Hematology Conference in this thread (http://forums.marrowforums.org/showthread.php?t=1499&highlight=estybon).

Good Luck!

Greg

[Paula W.]

Thank you Greg, I will look into it. She should be going to the trial Doctor this week. I'll update progress as we go along. Happy New Year!

Paula

[Paula W.]

Thank you Birgitta,

I will look into the link. Thank you so much for the information.

Paula

[Paula W.]

My Mom will be starting Onconova next Monday the 17th, with Dr. Raza in NYC. It sounds promising and we are optimistic. I will keep you all posted as maybe this drug will help others as well.

Paula

[Birgitta-A]

Hi Paula,
Dr Raza is a very wellknown MDS doctor and I think your mother is in good hands. Hope she will respond!
Kind regards
Birgitta-A

[Paula W.]

Thank you Birgitta, We are very hopeful. We have to be right???

Thank you for your well wishes!

Paula

[Greg H]

Hey Paula!

I echo Birgitta's sentiments. Dr. Raza is one of the best. I hope the Estybon works for your Mom.

I, for one, am grateful to her for participating in a clinical trial that could help many of us MDSers one day. I hope you will pass along to her my thanks.

Do keep us posted on how things are going.

Good luck!

Greg

[Paula W.]

Will do Greg! We are hopeful and optimistic! She had the port put in today and is ready to go on Monday!

Thank you Greg and Birgitta for the encouraging words on Dr. Raza. She has been the most compassionate Dr. I have ever met. I hope all goes the way they are hoping.

Paula

[Paula W.]

My Mom started Onconova yesterday. It's administered through the port and the iv bag needs to be changed everyday for 3 days (72 hour drip). So far no side effects to mention.

We also got the bone marrow results yesterday. Dr. said that blasts are at 2% and she is considered low risk MDS at this point. I'm so confused because her local oncologist said that she has about 6 months to live, so I assumed she was high risk, now Dr. Raza said she is low risk......

Her blood levels are still very low, platelets are about 28,000, wbc 1.8 and hemoglobin is at 8.1.

We met a man at the hospital who is also on Onconova and he has leukemia. After 3 rounds of treatment his numbers have improved. Hope he continues on this path.

Will post as we go along.

Paula

[Greg H]

Paula,

It's hard to know why your local doc might have offered that poor prognosis. Did he (or has Dr. Raza) indicated what your Mom's IPSS score is? It's not that hard to calculate (and there's even a tool on Marrowforums that can help).

Looking at the numbers you just provided, she's below normal in two of three blood counts (and the WBC is borderline). So that would score 0.5 on the IPSS scale.

Her blasts are at under 5%, so she doesn't get any IPSS points for blasts.

0.0 for blasts + 0.5 for blood counts gives us 0.5.

Right now, we are at a score that give us INT-1. A lot of times, docs group the IPSS Low Risk and INT-1 together and refer to those patients as "Lower Risk."

All we need to know to complete the IPSS score is which cytogenetic abnormalities your Mom has. You can check that portion of the BMB report or ask the docs.

Were your Mom's counts or blasts worse when your local Doc gave you the six month prognosis? If you don't mind my asking, how old is she?

Hope that helps. It's nice to hear about the other patient's success with the drug. If you can come up with the cytogenetics we can figure out the rest of the IPSS score.

Good luck!

Greg

[Paula W.]

Hi Greg,

My mom is 77 years old. When she started Vidaza her blast was at a 9%, then went down to normal than increased by 5%, but I don't know what the actual number was. This is when she was told that Vidaza no longer was working and that she needed a trial to survive.

I have the full bone marrow report. (I can email it to you if you would like to see it). This is what I found on cytogenetic.

CYTOGENETIC:
46,XX[20]

This was also in the report. It's all gibberish to me, but maybe it makes sense to you.

DIFFERENTIAL CELL COUNT: 500 cells counted ------------------------------------------- ----------------------------------------------------------------- ----------------- CELL TYPE % NORMAL* CELL TYPE % NORMAL* ----------------------------------------------------------------- ------------------------------------------------------------ MYELOBLAST 2 0 - 1 PRONORMOBLAST 1 0 - 3 PROMYELOCYTE 4 1 -3 BASO NORMOBLAST 1 0 - 3 MYELOCYTE 6 2 - 10 POLYCHROM 6 3 - 20 METAMYELOCYTE 3 5 -15 ORTHOCHROM 3 0 - 8 BAND 6 10 -40 MONOCYTE 2 0 - 3 NEUTROPHIL 29 10 - 30 LYMPHOCYTE 35 5 - 15 EOSINOPHIL 1 0 - 3 PLASMA CELLS 0 0 - 3 BASOPHIL 1 0 - 1 * Adult range

Let me know if this info helps. Thank you for your help and information.

Paula

[Neil Cuadra]

Paula,

The format of your post came out wrong. Is this how the columns line up on your differential report?

DIFFERENTIAL CELL COUNT: 500 cells counted

CELL TYPE % NORMAL* CELL TYPE % NORMAL*

MYELOBLAST 2 0 1
PRONORMOBLAST 1 0 3
PROMYELOCYTE 4 1 3
BASO NORMOBLAST 1 0 3
MYELOCYTE 6 2 10
POLYCHROM 6 3 20
METAMYELOCYTE 3 5 15
ORTHOCHROM 3 0 8
BAND 6 10 40
MONOCYTE 2 0 3
NEUTROPHIL 29 10 30
LYMPHOCYTE 35 5 15
EOSINOPHIL 1 0 3
PLASMA CELLS 0 0 3
BASOPHIL 1 0 1

* Adult range

[Paula W.]

Hi Neil,

Thank you, it makes more sense now. (I still don't know what all the numbers represent LOL). How I copied and pasted is how I received the email report. I have a MAC, so maybe the format isn't working right for my program.

Thanks again for clarifying.

Paula

[Greg H]

Hey Paula!

I'm not too smart about the various cell counts in a BMB, but your Mom's appear to all be in the normal range, with a couple of exceptions. The neutrophils are at 29, which is just 1 away from the high end of normal. The lymphocytes are way high at 35, when the top of normal is 15. So maybe your local doc was worried about transformation to some type of leukemia?

Anyway, the cytogenetics of 46,XX [20} means your Mom is female and has normal chromosomes.

So she doesn't get any IPSS points for cytogenetics. But the 9% blasts would have added 0.5 points, giving her a score of 1, which is still INT-1, lower risk.

I don't put much stock in the IPSS survival rates, since they are all based on data from before there was anything other than supportive care. The official IPSS prognosis for INT-1 is 3.5 years, but, like I say, that's based on data from before Vidaza and Onconova. Plus, every patient is an individual.

Hope that's useful. Glad your Mom's blasts have dropped so far from the 9%.

Take Care.

Greg

[Paula W.]

Thank you Greg for all the information. This disease seems to be such a roller coaster ride. Things change rapidly in both directions, good and bad. It's hard to keep up. Once you think you understand what is going on, it all changes.

My mom completes her first round of Onconova treatment today. Her second round will begin on the 31st. She goes for blood work in between the treatments. I will keep you all informed as we go along. I think it's important for all of us on this site to know about these trials, as they may be an answer to someone prayers!!

God Bless.

Paula

[Paula W.]

It's been 2 1/2 days since my Mom's first round of Onconova ended. She is physically exhausted. It can be from the drug or it can be a sign that her blood dropped. She is exhausted just moving about her home and she didn't go to church today, so I know she isn't feeling all that good. She goes for blood work tomorrow, so we should know if it's the drug or her blood then. If this is a reaction from the drug, so far it's the only one she has experienced thus far.

Paula

[Greg H]

Hey Paula,

Sorry to hear about your Mom's fatigue. In my experience -- and there's research that shows most other folks with MDS have the same experience -- fatigue is the main symptom of this disease. With me it comes and goes, and it's not always related to my blood counts, and, as I understand the research, that's pretty typical.

I'll often have a "bad" day and then, the next, feel great. It can kind of make you nuts.

I hope your Mom's current fatigue is very temporary -- and that you get good blood counts tomorrow!

Take care!

Greg

[Paula W.]

Hi All,

My Mom feel a lot better today, almost back to her old "beautiful" self. I did get her blood work, platelets dropped to 21,000 and Hemoglobin dropped to 8.0. The Doctor said that nothing was to be done, as far as transfusions at this time. Doesn't believe the drug had any part of the drop.

[Paula W.]

Well after days of her exhaustion increasing I brought her back for blood work. Hemoglobin dropped to 6. Blood dropped from 8 to 6 from Monday to Friday. She was sent for a transfusion which helped a lot. Not 100%, but much better. She goes for second round of Onconova on Monday.

[Greg H]

Paula,

Wow! No wonder she was feeling tired. I had one week like that back in December. I burned through two units of red cells in a single week. I was at 6.4 on Tuesday, got two units that day, and then was at 6.2 eight days later.

They decided to give me three units at that point, and it really helped get me back on track. I'm sure they will be watching your Mom weekly until things start to improve.

Good luck to you both!

Greg

[Paula W.]

Wow Greg,

Didn't know that the red cells can drop so quickly. I knew that the platelets only last a couple of days, but I thought the rbc lasted longer. This is the first time in approx 5 years she needed blood. Hope this going to become the norm. She goes for blood work tomorrow. I'll keep you posted.

Thanks for the input, I appreciate it so much.

Stay well!

Paula

[Paula W.]

Hi All,

My Mom finished her 2nd round of Onconova and again needed a blood transfusion. HB dropped to 7.7. 2nd transfusion in 13 days. Platelets rose a little 27,000 to 35,000 (we will take it). She also had blood along with her bowel movement the other day, which scared her a lot. I know the right thing is to take her to a GI dr., but I just don't want to put her through anything else.

She feels good, not great, more scared than anything. Her trial dr. is asking us to be patient and calm. I know this is good advice but hard to do.

She starts third round on Monday. I will keep you posted.

Good health to all.

Paula

[triumphe64]

You don't want to lose the blood at one end that you get transfused on the other. Talk to her trial doctor.

[Greg H]

Hey Paula!

Thanks for the update. I'm sorry to hear that your Mom's counts haven't stabilized yet, but that platelet uptick is a good sign. I think hanging in there is pretty much the prescription at this point -- though I wouldn't let that GI bleeding thing go on very long without talking with the docs about it.

I can't imagine that your Mom would be interested in most of the exciting procedures GI docs are known for -- nor would they be likely to do them, given her platelet level.

Take care!

Greg

[Paula W.]

Greg,

She hasn't had any bleeding since that day. I spoke w/ my brother and my father and we decided to see if it happens again before putting her through any thing else. Just don't want to put more on her at this time. What if it is something serious???? then what??? considering the time line her oncologist gave her, do I want to put her through anymore worry or hell????? Don't know the right answer, just going w/ my gut at this time.

She went to trial dr. yesterday to start her 3rd round. Her hb is at 11.5, which is probably due to the transfusion. Her platelets dropped to 29,000 from 35,000. Dr. suggested a platelet transfusion just to see if it would hold. She will probably do that after this onconova round.

I'm trying to keep positive, but we really don't have any signs that the trial is working yet, maybe to early to tell.

Thanks for your continued support.

Paula

[Greg H]

Hey Paula!

Glad to hear the bleeding stopped. Perhaps it won't return. If the blood was bright red, it could be related to either hemorrhoids or small temporary tears in the anal region, both of which are complicated by constipation or straining during a bowel movement. That sort of thing is usually temporary and not too much to worry about if it doesn't persist.

All any of us can do is go with our gut and hope we made the right decision.

You would, of course, want to mention it to her docs -- particularly the clinical trial doc -- as well as letting them know that you don't think it's good to try to deal with it at the moment.

I'm not sure the reports on Estybon that were presented at ASH had any data on how long it took to respond. Does your trial doc have any info to share on that point?

It can be frustrating to wait for therapy to work -- and hard to keep faith that it will.

Good luck to you and your Mom.

Greg

[Paula W.]

Hi All,

Well, my mom in in between her 3rd and 4th Onconova treatment. She needed a platelet transfusion today as her platelets dropped to 9,000 from 18,000 on Friday. The platelets have dropped dramatically these past couple of weeks. Her HB is holding since her last transfusion at 10.6. They did drop since Friday from 11.6 to the 10.6. WBC are at 1.3.

She kind of as the defeated look on her face but is trying to look strong for us. Seems that everyday is a new challenge. I don't think that the trial is doing what we hoped, but we will continue to give it a fair chance and to continue to give hope to my mother.

She goes for her 4th treatment next Monday.

Paula

[Paula W.]

The day after her transfusion, I picked her up and we went shopping at the mall, she looked and felt great, yesterday we spent the afternoon together w/ family, and she hosted at her house, then last night we went to our weekly poker night. Again, she felt and looked great. All I have to say is "what a ride". One day I feel like I'm losing her and the next it's like nothing is wrong!! Cherish the good days!!!

More blood work today, it can go either way, maybe a transfusion or maybe a dat in AC, who knows!! LOL

Have a great day everyone!!

Paula

[mausmish]

Wonderful news that she's responded so well!

[Paula W.]

Big day for my mom today. She is starting her 5th round of Estybon (Onconova) as well as going for a bmb. I'm kind of nervous about the bmb. She started out on the trial with under 8% blast, but since she started the trial she has pretty much been transfusion dependent with platelets and rbc, although it's been 2 weeks since she needed any. Her platelets are stable between 23,000 and 28,000.

They will check her blood today before doing bmb, in case she needs platelets.

Please say a little prayer. I will keep you all posted. I know we are all hoping for a good turn out as this drug may be the answer to many of our prayers.:)

Paula

[Birgitta-A]

Hi Paula,
Hope your mother was OK during the BMB and that the result will show good effect after treatment with Estybon!
Kind regards
Birgitta-A

[Paula W.]

Well we were very excited on Wednesday 3/16/11, before she went for her BMB she had blood work and for the first time since October, her platelets jumped 10,000 to 35,000 and her RBC to 8.9. So we figure ok, trial starting to kick in right?????

Yesterday, she goes to have the drug disconnected from her port and starts bleeding from the port, so the dr. wanted to check her blood. Her platelets dropped to 17,000 and her RBC to 7. So today she is getting transfusions on both. What a drop in one day!!!! So I don't know where this is going, but I don't have a good feeling. I will know more next week, when we should get some results from BMB. And the roller coaster ride continues. Trying to optimistic but my mom look sooooo defeated yesterday. But she is such a trooper, I couldn't be prouder.

I will keep you all posted.

Paula

[cathybee1]

Thanks for the update.

Many of us have been on that roller coaster ride. I hope this was just a temporary setback for your mom and that today went a little better.

[Paula W.]

Thank you Cathy,

I met her at the hospital yesterday when she was getting the transfusion. Guess what?? Before they did the transfusion they took her blood and her platelets jumped back up to 27,000, so she didn't need platelets, just blood. How it drops from 35,000 to 17,000 then back up to 27,000 in a matter of 3 days, I will never figure it out.

I know all of you have been on this roller coaster ride, it's a wonder how so many of us keep it together.

May a cure be in the near future!!!

Regards,
Paula

[Birgitta-A]

Hi Paula,
Perhaps you have already seen these "warnings" from patients with low platelets. We are all different and many of us tolerate the drugs and food that can give other persons low platelets or increase bleeding risk: http://www.pdsa.org/about-itp/warnings.html

You know the platelet count can change very fast and we don't know the reason but we should always be very careful with all kinds of drugs especially painkillers.
Kind regards
Birgitta-A
72 yo, dx MDS Interm-1 2006. Platelets slowly decreasing from 65 to 22 May 2010. After Thalidomide since June 2010 platelets 109.

[Paula W.]

Thank you Birgitta,

I went to the site and printed out the foods that may do more harm than good. There are a lot of foods on there!!:( I gave my mom the list and I threw out all of her "sweet n low".

Again thank you for your help (and your knowledge).

All the best!

Paula

[Paula W.]

Hi All,

We got my moms bone marrow results today and the results were very good. Her blasts are at a 2% :D. She is still considered low risk MDS and at this point, not at risk for leukemia. Her blood levels have not improved as of yet. The Dr. wants her to remain in the Onconova "estybon" at this time. If her numbers don't improve, she would like to try another drug. I don't know what drug it will be.

She has been blood transfusion & platelet transfusion dependent since on the Onconova. Her doctor insists that it's not from the drug, but from the MDS itself. I will keep you posted...........

Paula

[Birgitta-A]

Hi Paula,
Very good with low blast cells! Do you think it will be possible to combine Estybon with some other drug or is that impossible because Estybon can only be given in trials with a special protocol?
Kind regards
Birgitta-A

[Paula W.]

Birgitta,

I'm thinking they wont mix the drugs. They don't even like changing the dosage on the Estybon. I will find out in a couple of weeks, what their plan is, because if her blood levels don't improve I don't believe they will keep her on the trial. But I am grateful that the blasts are low too!!

Paula