International MPN News, Science & Opinion

On trial for her life

Last month, scientists and physicians gathered in Chicago to hear the official results of the so-called COMFORT (controlled myelofibrosis oral JAK inhibitor trial) trials. These were the Phase III double blind trials of a new myelofibrosis drug. The proof was indisputable. In most cases, the drug, Ruxolitinib, dramatically and quickly reduced spleen size and improved the quality of life of myelofibrosis patients, many of whom were condemned to increased suffering before death.

Three and a half years earlier, there was a much more risky undertaking, a Phase I/II clinical trial in which varying dosages of a drug simply labelled “Incyte — Chemotherapy Drug” was given to people desperately ill with MF. One of the participants in that trial, Barbara Beckman, continues to make MPN history. Three and a half years later, still on Ruxo, she is carefully studied to see the long-term effects of the drug on quality of life and life extension. Here is her story.

My Incyte

by Barbara Beckman

It’s July 19, 2011. I looked down at the paper in my hand. For the first time in over 3 ½ years there are no highs or lows by my blood counts. The counts are all in the normal range. My recent bone marrow biopsy was not so optimistic: MF-3 (Persistent).

Under additional findings, I read: Marrow architectural distortion marked; Osteosclerosis: diffuse with bony remodeling;Vascular sinuses dilated; Intrasinusoidal hematopoiesis. I feel better but myelofibrosis is still with me,

In 1993 I had emergency gallbladder surgery. It was then that the doctors discovered I had something wrong with my blood, and they wanted me to see a specialist. The specialist did a bone marrow biopsy and diagnosed essential thrombocythemia. My platelets were over a million, but the rest of my counts were OK. He prescribed Hydrea, which I took for about 10 years.

In 1996 we decided to move back to Idaho fromWashington,in order to be closer to family. (My high school age grandsons were basketball stars and my husband, Don, just had to be there!) No hematologists there but an internist kept track of my blood counts, continued my prescription for Hydrea and I felt okay. However, I was slowly losing weight (hooray!) and through the years I lost my plumpness, and I loved it.

In the meantime I developed a painful ankle ulcer. I belonged to a MPD group online and learned that HU might be the cause, and I stopped taking it . The ulcer healed, but came back when I took HU again. Well, okay, I just didn’t take HU anymore. I buried my head in the sand and tried to think I didn’t have a blood disorder.

We moved back toWashington for my husband’s work. I found another hematologist who let me get by without taking anything. I was getting much more tired, and the weight kept falling off. I had lost 60 pounds. I told my husband that my arms looked like the bone was a curtain rod and my skin was draped over it. It was UGLY!

I insisted that my doctor do a bone marrow biopsy, and the diagnosis came back MF. Luckily I found a MPD group that met in Seattle. This group meets several times a year, mostly to be able to share information and receive support from people who understand. The speaker at the first meeting I attended was someone who was enrolled in a trial in Houston, and was doing well. I talked to her after she spoke. She told me that I needed to try to get into that trial. (Thanks so much, Lois.) http://health.groups.yahoo.com/group/MPDSeattleSupport/

I was able to get an appointment for a consultation with Dr. Verstovsek in Houston. Dr. V told me that there was a trial for a new medication for MF. It was sponsored by Incyte . Some of the criteria for the trial were a large spleen and a white count less than 30. My spleen was large and my white count was 29. He said I could join the trial and I jumped at the chance.

Within the first week I could tell that my spleen was smaller. I seemed to have no side effects, even though I was taking a large dose: two 25mg pills a day. The dry cough that troubled me at night went away. They told me that this medication was not a cure, but was palliative only. That was okay with me—I was desperate to feel better. As time went on I gained weight and felt stronger. (Now I would like to lose a little weight! Dr. V says walk).

I have been on this trial for over 3 ½ years. I did get a little anemic, but it seemed to regulate itself after a while. The first two BMB’s were terribly painful, even though they were done by a beautiful girl who was a student supervised by a teacher. I hope that girl becomes a model and not a medical person! The rest have been done by competent techs and not bad at all. Lidocaine has been the only anesthetic.

Flying to Houston from Seattle isn’t really fun, but Continental has a medical rate, and a through flight. Now I only have to go to Houston every six months. I don’t know what will happen once this drug is approved, but I will cross that bridge when I come to it.

I am a retired teacher, a reader, and a dedicated gardener. My flowers always get lots of compliments and I am able to take care of them. I am also 77 years old. I would like to live to see my great-grandchildren grow up. Did I tell you how wonderful they are?

I have lived longer than I ever thought I would. I have quite a bit of pain in my legs, but I am able to take some painkillers that help. Last time I was inHouston, the lady who cut my hair gave me a cross-stitched cross that she had made, and there was a beautiful encouraging note with it. I didn’t even tell her that I was sick!

And then today a lady gave me a little pewter angel.

On the angel, it said “HOPE”.

© Barbara Beckman and MPNforum.com, 2011. Unauthorized use and/or duplication of this material without express and written permission from this blog’s author and/or owner is strictly prohibited. Excerpts and links may be used, provided that full and clear credit is given to Barbara Beckman and MPNforum.com with appropriate and specific direction to the original content.

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