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How can i decide????????????????????

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Offline Manal

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How can i decide????????????????????
« on: November 12, 2006, 02:41:15 AM »
Dear all

Today, my hematologist suggested trying (he has a an adult patient that is doing this) the butyrate shots . What you need to do is travel twice a year to Washington DC where you take these shots as they are not commercially available, but supposed to be in few time.  They work on the F HB and actually they increase the HB in a very good range. The nice thing is that this substance is not a chemo like hydroxyurea and also not carcenogenic (as hydroxyurea sometimes cause secondary cancers in CERTAIN mutations  of thal ).

All this looks very good. The problem is that this is all under trails and i guess you will be a human lab ,instead of mouse lab.  When i mentioned this to the doctor, he said that it is not that pisimistic as there are ethical limits in trails, but i am not really convienced with this.
He told me that he will be getting the  E-mail of the center for me to contact them next week .

If you were me , shall you take the risk of being under trails or what will you think????????????????????????

How can you decide ?

Manal
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Offline Andy Battaglia

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Re: How can i decide????????????????????
« Reply #1 on: November 12, 2006, 03:16:33 AM »
Thals are always guinea pigs. Every older thal was a guinea pig for desferal. Many were also guinea pigs for L1 and now many are guinea pigs for Exjade. Thals have also been guinea pigs for many other drugs. For most of these choices it has come down to are they willing to risk it if it will keep them alive. I feel like a guinea pig every time I see a TV ad for my asthma med and it says that it causes death in some patients but it is the only med that has ever helped. For now I made the decision to keep taking it. But almost every day I ask myself if the risk is worth it.

It will be interesting to hear the answers to this question.
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Andy

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Offline Christine Mary

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Re: How can i decide????????????????????
« Reply #2 on: November 12, 2006, 06:09:16 AM »
Hi Manal,

I can almost feel your words, and the confusion you are dealing with. Although my husband and I havent had to make any definitave decisions for Lauryn ,YET, I know the day will come shortly. We are juggling the idea of the port. No matter which way we think is best,there is always the "what ifs?".
In my opinion, and especially since hearing the other intermedias expieriences on this site, I would not go through with this potential treatment. I think it's inevitable that Ahmad will eventually need transfusions. No one knows how long any of the meds. will work for intermedias. We do know,though that transfusions work. Yes, it is so scary. With tx's comes chelation, but you ALMOST know what to expect. Like you mentioned before, the meds., have no guarantees as of right now. I am kind of contradictating myself though,b/c my baby cousin is on hydroxyurea (for inter.) and she is doing very well AT THIS MOMENT. My cousin (the mother) figures every day that goes by without having needed the transfusion,the better the baby is. BUT< who knows the long term side effects? AGAIN, anything can happen to anyone of us anytime. We cannot worry about long term,because we are wasting the present. Did you do any research regarding any other thals who have had the treatment you are talking about?Can you get a second opinion? I am confusing myself right now, because there is so much running through my head and I cannot express it through typing. I wish we could talk in person. i'd give you such a big hug. :hug You know whats best for your baby. Go with your maternal instinct. If you feel it's worth a try,then go for it! It can't hurt. Just know whatever you decide, your doing whats best for your child. I will pray for you for your despair.

Love,
Christine :heartred
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Lauryn's Mom
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Kathy11

Re: How can i decide????????????????????
« Reply #3 on: November 12, 2006, 06:27:27 AM »
Hi Manal,
You need to listen to your instinct.If you have doubt you need to proceed with caution.Evaluate all your options and choose the one that feels best for you and your son.
get as much information of the new treatment and its'  side effects.
remember you do have different paths,Dont rush into anything because you may have to deal with consequences  of whatever choice you make,be realistic.That does not mean pessimistic.
Be wise
Kathy
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Kathy11

Re: How can i decide????????????????????
« Reply #4 on: November 12, 2006, 06:31:24 AM »
hi christine
great mind think alike we were typing the same thing at the same time.lol lolo  Kathy
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Offline Christine Mary

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Re: How can i decide????????????????????
« Reply #5 on: November 12, 2006, 03:56:12 PM »
 :rotfl
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Offline Sharmin

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Re: How can i decide????????????????????
« Reply #6 on: March 19, 2009, 03:23:35 AM »
Manal,

I know that you posted this a loooong time ago - but did you ever follow up on it?  Dr. Vichinsky spoke very very highly of butyrate.  He said that it worked extremely well.  In one case it helped a transfusion dependent thal get off of transfusions (this was required because the patient had too many antibodies and was untranfusable). 

Sharmin
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Sharmin
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Offline Manal

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Re: How can i decide????????????????????
« Reply #7 on: March 19, 2009, 11:33:39 AM »
Hi Sharmin

Yes it was so long :biggrin

This trail turned out to be Dr. Suzan Perrine trails in Boston medical center and at that the time when i contacted her the trail was closed (In this trail was the 18 year old intermedia patient that i told you about in another thread and is still receiving this treatment)

At that time she told me that the new oral one will start the following summer ( this is the Hemaquest that is supposed to start in Lebonan in the coming months) but unfortunatly it will be on adults first as they are not able to set a dose for children

Now and after those years with thal, i am more confident to try this. Dr. Suzan had told me once that her drug would get some good results with people who didn't react positivly to hydroxurea

manal
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Offline Sharmin

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Re: How can i decide????????????????????
« Reply #8 on: March 20, 2009, 12:44:26 AM »
Manal,

I wasn't aware that Hemaquest was the oral version of butyrate.  I really hope that it is available soon, especially if it is as promising as it is believed to be.  I hope that Ahmad's hg goes above 10 with it! 

Sharmin
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Sharmin
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Offline Manal

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Re: How can i decide????????????????????
« Reply #9 on: March 20, 2009, 12:56:38 AM »
Thanks Sharmin, this would e a dream, wish i can see it one day

manal
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Offline Sharmin

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Re: How can i decide????????????????????
« Reply #10 on: March 20, 2009, 01:03:05 AM »
Manal,

Have you heard anything new about retro-differentiation?
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Sharmin
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Offline Andy Battaglia

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Re: How can i decide????????????????????
« Reply #11 on: March 20, 2009, 02:23:50 AM »
The drug Dr Perrine has been working on is HQK-1001, a short chain fatty acid. Butyrate is also a short chain fatty acid but not the same drug as HQK-1001. the expectations for HQK-1001 are much higher than what has been experienced with butyrate, and of course will be much easier to administer, being an oral drug.

http://www.hemaquest.com/HTML/technology.html

Quote
Our Founders used a proprietary screening system to identify derivatives of short chain fatty acids (SCFADs) with increased potency, improved therapeutic effects and more favorable pharmacological properties compared to butyric acid. This led to the discovery of a series of compounds that not only induced hemoglobin F induction, but also stimulated the production of a variety of blood cells. These compounds appear to stimulate blood cell production through similar pathways to recombinant proteins, such as G-CSF (Neupogen, Neulasta) and erythropoietin (Epogen, Procrit). However, in contrast to these agents, the compounds can be orally-administered and thus should provide a significant advantage over the current recombinant proteins, which require injections. The first compound, HQK-1001 has now entered clinical development with an initial focus on treating sickle cell anemia and thalassemia.

I also have high expectations for this drug and believe it has the potential to revolutionize treatment for blood disorders.
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Andy

All we are saying is give thals a chance.
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Offline Manal

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Re: How can i decide????????????????????
« Reply #12 on: March 20, 2009, 02:59:41 AM »
Thanks Andy

Sharmin , the last thing i knew about retro-differentiation is that Dr. Abu el Jadayl will start transplanting a HLA matched stem cells to patients without using chemotherapy before transplantation in attemptto curethe disorder. I think that she will be coming to the Cairo conference in May and it will be easy to talk to her and find how things will go


manal
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Offline Sharmin

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Re: How can i decide????????????????????
« Reply #13 on: March 20, 2009, 02:44:15 PM »
Both of these ideas sound so amazing.  I hope that the research moves along quickly!!!!! :pray :pray :pray :pray

For the sake of patients of all ages and their families. 

Sharmin


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Sharmin
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Offline Narendra

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Re: How can i decide????????????????????
« Reply #14 on: March 20, 2009, 04:21:19 PM »
There is more about the Hemaquest traials with HQK-1001 at
http://clinicaltrials.gov/show/NCT00790127

The study starts this month (March 2009) and are currently recruiting patients at
Siriraj Hospital
Bangkok, Bangkoknoin District,
Thailand, 10700

Quote
Primary Outcome Measures:
Safety as assessed by (1) adverse events, (2) laboratory values (3) vital signs (4) physical exam [ Time Frame: 168 days ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
Pharmacokinetics assessed by plasma drug concentration levels [ Time Frame: 140 days ] [ Designated as safety issue: No ]

Pharmacodynamics assessed by red blood cell production and induction of fetal hemoglobin [ Time Frame: 168 days ] [ Designated as safety issue: No ]

Hope for some positive outcome of the trial, which seems to be published by the end of this year.
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