Pretreamtent for aggressive MDS?

[italianburrito]

I really hope I get some sort of response from this post because I am desperate. The bad news keeps coming and I received results that my dad is positive for FLT3-ITD which makes for an aggressive disease. Some websites I have visited said it wasn't a bad progrosis but others say it's a bad prognosis. Maybe the Cuadra's know about this new drug Sorafenib that can be used to suppress this mutation? I have read that taking it before and after transplant can be very beneficial. Has anyone taken this drug before? Please someone out there give me some hope! I love my dad so much and I want him to be around for a long time.

[Neil Cuadra]

Originally Posted by italianburrito

I really hope I get some sort of response from this post because I am desperate. The bad news keeps coming and I received results that my dad is positive for FLT3-ITD which makes for an aggressive disease. Some websites I have visited said it wasn't a bad progrosis but others say it's a bad prognosis. Maybe the Cuadra's know about this new drug Sorafenib that can be used to suppress this mutation? I have read that taking it before and after transplant can be very beneficial. Has anyone taken this drug before? Please someone out there give me some hope! I love my dad so much and I want him to be around for a long time.

Any AML diagnosis of this type carries a poor prognosis, unless one of the new drugs being researched can help. I wish the news were better.

Sorafenib (brand name Nexavar), combined with chemotherapy, attempts to eliminate cancer cells in multiple ways: targeting both internal cell components and surface enzymes, some of which may inhibit the cell's ability to form blood vessels. There are open clinical trials for sorafenib. It's one of many drugs that have been studied for FLT3-ITD, but that's not the only drug of its type.

The overview for a clinical trial for ponatinib (trade name Iclusig) in France describes sorafenib as a "first generation FLT3 inhibitor" while also mentioning "second generation" and "third generation" inhibitors.

A good next step might be to seek advice from an AML expert on specific trials to consider. With your dad's unique history, it'll be hard for you to tell what trials he'd qualify for and which would be most promising for him. I suggest that you not rule anything out until you consult one or more doctors who treat AML.

[italianburrito]

Originally Posted by Neil Cuadra

Any AML diagnosis of this type carries a poor prognosis, unless one of the new drugs being researched can help. I wish the news were better.

Sorafenib (brand name Nexavar), combined with chemotherapy, attempts to eliminate cancer cells in multiple ways: targeting both internal cell components and surface enzymes, some of which may inhibit the cell's ability to form blood vessels. There are open clinical trials for sorafenib. It's one of many drugs that have been studied for FLT3-ITD, but that's not the only drug of its type.

The overview for a clinical trial for ponatinib (trade name Iclusig) in France describes sorafenib as a "first generation FLT3 inhibitor" while also mentioning "second generation" and "third generation" inhibitors.

A good next step might be to seek advice from an AML expert on specific trials to consider. With your dad's unique history, it'll be hard for you to tell what trials he'd qualify for and which would be most promising for him. I suggest that you not rule anything out until you consult one or more doctors who treat AML.

Thank you so much for your quick reply! At the moment my dad does NOT have leukemia. BM biopsy showed less than 1% blast and the cells are moderately dysplastic per the pathologist. He is still transfusion dependent after 3 cycles of Vidaza. I wanted to hear if anyone in this forum had to deal with this mutation and what their treatment entailed. I still have a lot of hope since we caught this early and transplant was discussed from day 1. I will speak to the duke doctor about trials regarding this. Again, thank you for your information and listening ear. My dad is a fighter and will not be brought down easily!!

[KarenE]

I have heard of others on a trial for ASP2215, so you might ask about it. There are several threads anout it on daily strength under the AML forum. Althought most of those taking it do not have MDS the do have the same AML mutation as your father.