I live in the U.S.A. and i want deferiprone/kelfer/ferripox

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I live in the U.S.A. and i want deferiprone/kelfer/ferripox
« on: March 22, 2009, 06:57:42 AM »
I dont have Thalassemia, I have Friedreich's Ataxia. There is 1 clinical trial for deferiprone in ataxia but it is in France and the problem is I have things going on in the US so I cant really leave for 6 months and I hate these trials where they give half the people placebos.

the FDA in the U.S will probably take 10 years before even reviewing deferiprone/kelfer/ferripox

According to the trial, people given the full drug will be given about 60mgs/kgs a day

Is there anyway I can get this drug. I can fly to Europe or Asia or wtvr to get the drug but only if its available over the counter cause they dont currently give prescriptions to my condition yet, or not that i know off.

My current state right now is I am 22 and can walk, but barely. I dont even like to go to a grocery store cause I either gotta use a walker or risk falling or bumping in to things. The wierd part is I was diagnosed very late in my life (relative to others with friedriechs) when I was 19 and could still run a mile.

Any help is more than welcome but please dont reccomend any trials. I am willing to go any where in the world as long as I dont have to stay, even if this drug has harmfull side effects ill take my chances.
And please dont tell me about
http://www.clinicaltrials.gov/ct2/re...%27s+Ataxia%22

ive asked neurologists and tested some the other drugs myself, i think the chelator is my best bet.
« Last Edit: March 22, 2009, 07:02:56 AM by rkr868 »

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Offline Zaini

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Re: I live in the U.S.A. and i want deferiprone/kelfer/ferripox
« Reply #1 on: March 22, 2009, 08:12:37 AM »
Hello,

Welcome on the forum,i am really sorry to hear about your problems,i don't know much about Friedrich's ataxia apart from that its a condition related to damage of nervous system,but may i ask how did you get iron overloaded,coz defriprone is a chelator ,were you transfused frequently?

Dferiprone is available in my country and i might be able to help you get it,as my daughter is registered with the distributors here,but I'd be grateful if you can explain a bit more ti us as to why do you need it.

And please wait for Andy's reply as he also is in US and must know more about what could be done.

Zaini.
^*^Xaini^*^

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Offline nice friend

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Re: I live in the U.S.A. and i want deferiprone/kelfer/ferripox
« Reply #2 on: March 22, 2009, 08:45:38 AM »
Hi RKR868 ,
 :wekcome2 , i hope you will find this forum very helpful caring, encouraging and a big source of knowledge...i m sorry to hear about wat you are passing through , i hope you will b fine soon .. may GOD blesss you with the best of health and everything ....  stay in-touch with us ...

Best Regards
Take Care
Umair
Sometimes , God breaks our spirit to save our soul.
Sometimes , He breaks our heart to make us whole.
Sometimes , He sends us pain so we can be stronger.
Sometimes , He sends us failure so we can be humble.
Sometimes , He sends us illness so we can take better care of our selves.
Sometimes , He takes everything away from us so we can learn the value of everything we have.

===========
Umair

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Offline Andy Battaglia

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Re: I live in the U.S.A. and i want deferiprone/kelfer/ferripox
« Reply #3 on: March 22, 2009, 03:31:04 PM »
Ferriprox is available in the US under a special program. I know thalassemia patients do take it here. I would suggest contacting Eileen Scott at the Cooley's Anemia Foundation and asking for her help in getting into this program.

Cooley's Anemia Foundation
330 Seventh Avenue, #900
New York, NY 10001
(800)522-7222
Fax: 212-279-5999

Patient Services Manager
Eileen Scott
eileen.s@cooleysanemia.org

Please let me know how this goes. Ferriprox should only be used under a doctor's supervision so please try to do this so that your doctor is aware of the drug and its monitoring requirements. If Eileen is not able to help, please contact me. I will contact the Apo Pharma rep to see what can be done to get you on Ferriprox.

I do believe that Ferriprox will be approved in the US by 2010-2011.
Andy

All we are saying is give thals a chance.

Re: I live in the U.S.A. and i want deferiprone/kelfer/ferripox
« Reply #4 on: March 22, 2009, 08:53:10 PM »
thanx for the responses. My current neurologist is Susan Perlman at UCLA and she speaks highly of the the iron chelator.

As far as what it can do for me, apparently my body has problems with iron staying in my blood but these two sites can tell u much more than i can
http://www.clinicaltrials.gov/ct2/show/NCT00224640
http://www.clinicaltrials.gov/ct2/show/study/NCT00530127

To Andy- U say ferripox will probably be approved by 2010-2011 but i doubt that since in 2006 they told be it would be approved by 2008. Plus call me impatient but i dont want to wait another year or 2. Ive already seen the big changes that a year or 2 can do. IF I do get the drug I will tell my neurologist I got it and i will only use it under her supervision, so im not goin to be reckless with it.
I will contact the the number u asked but I dont think anything will happen (not trying to be negative just from past experiences).

Here is some info on the curefa.org site
Drug- Deferiprone
Sponsor: Apopharma
A phase I/II study of Deferiprone (iron chelator) will be initiated at a number of international sites such as France, Belgium, the UK, Italy and Spain in Spring or Summer 2008.  ApoPharma is working with FA clinicians in Canada and Australia regarding additional sites.  This trial is based on very promising results in a pilot study conducted in France and published last year by Boddaert et al in Blood. If this phase I/II study demonstrates safety and benefit, ApoPharma will submit the trial data to the FDA in hopes of including the United States in a phase III trial of Deferiprone. FARA is working closely with the company to help with patient recruitment through our patient registry.

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Offline Andy Battaglia

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Re: I live in the U.S.A. and i want deferiprone/kelfer/ferripox
« Reply #5 on: March 22, 2009, 09:43:16 PM »
Unlike in the past, Apo Pharma has now submitted Ferriprox to the FDA for approval. By the way, Ferrirpox is already approved in Europe. The US and Canada are among the very few countries that haven't approved it. The climate is right for approval and I fully expect Ferrirpox will be approved by the FDA. This is good news to iron overloaded patients, but yes, you need help now. I will contact the rep from Apo Pharma to find out what the current situation is regarding Friedreich's Ataxia and Ferriprox. Desferal is not useful because it cannot remove mitochondrial iron, so that leaves deferiprone. I would also highly suggest you investigate the use of antioxidants as they have shown to have some value in protecting against damage caused by iron.

I will get back to you after I contact Apo Pharma.

Andy

All we are saying is give thals a chance.

Re: I live in the U.S.A. and i want deferiprone/kelfer/ferripox
« Reply #6 on: March 22, 2009, 09:52:29 PM »
thanx for the quick reply Andy, and actually I am on an antioxidant drug Idebenone my neurologist gave me. Only problem is Ive been on it for a year and 2 months, and Im still getting progressively worse. So it may slow down the progression but it definetly needs help from another drug.

Keep me posted on whats going on, I really appreciate what ur doing

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Offline Andy Battaglia

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Re: I live in the U.S.A. and i want deferiprone/kelfer/ferripox
« Reply #7 on: March 22, 2009, 09:55:56 PM »
There are also many safe antioxidant supplements like vitamin E and IP6 that can be tried. They definitely help protect against the oxidizing effects of iron. They will not eliminate it of course, but they can have a positive effect and slow down the damage. I have emailed my contact at Apo Pharma and will let you know once I receive a response, which should be early in the week.
Andy

All we are saying is give thals a chance.

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Offline Andy Battaglia

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Re: I live in the U.S.A. and i want deferiprone/kelfer/ferripox
« Reply #8 on: March 22, 2009, 11:09:03 PM »
I did receive a response from Apo Pharma. Ferrirprox is not approved for use with Friedreich's ataxia  anywhere in the world, although it is in stage two trials. However,

"the only way to get DFP is actually the same process as it is forThal, which is the compassionate use program. I think we have a number of US FA and NBIA patients

The easiest way to start the process is to have your doctor contact our VP Medical Affairs (ftricta @apotex.com)"

Have your doctor contact them and see if there is a possibility of getting into the compassionate use program.
Andy

All we are saying is give thals a chance.

Re: I live in the U.S.A. and i want deferiprone/kelfer/ferripox
« Reply #9 on: March 22, 2009, 11:31:56 PM »
Ive emailed my neurologist to email ftricta @apotex.com and inquire about the compassionate use program. Thanx for all your help, but Im curious what are the chances i get the drug.

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Offline Andy Battaglia

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Re: I live in the U.S.A. and i want deferiprone/kelfer/ferripox
« Reply #10 on: March 23, 2009, 02:55:48 AM »
I can't predict what the chances are because Ferriprox is not approved for Friedreich's ataxia. However, Ferriprox is not approved by the FDA in the US, yet it is available to patients in the US under the compassionate use program, so there may be some chance this can work.

Because of the experimental nature of Ferriprox use in Friedreich's ataxia, for your own safety you would have to make sure a doctor is monitoring your white blood cell levels regularly should you decide to try to get it from another foreign source. This could prove difficult because of potential liability issues for the doctor. Also, because of the potential that too much iron would be removed, close monitoring would be a must.

You could likely benefit from a supplement program heavy on antioxidants. In addition to Idebenone, IP6, L-carnitine and alpha lipoic acid, vitamin E all help to protect the cells from iron damage. B complex is also an important addition. I would also suggest you contact Osato USA and see if there has been any research into the use of Fermented Papaya Preparation (FPP) in FA patients. FPP has shown some amazing properties in many areas and was the topic of a formal presentation at the International Thalassemia Conference in Singapore in October, 2008.

Osato USA, Inc.
11751 Mississippi Avenue, Suite 110
Los Angeles, CA 90025

TOLL-FREE: 1-877-OSATO-11 (1-877-672-8611)
or Phone: 310-231-1090
Fax: 310-231-1089

Matt Johnson, Manager
email: mj@osatousa.com


Andy

All we are saying is give thals a chance.

Re: I live in the U.S.A. and i want deferiprone/kelfer/ferripox
« Reply #11 on: November 09, 2009, 05:26:17 PM »
my sister also has fa;  i was wondering how you made out with your quest for deferiprone . . .please send me a follow-up . . thanks

 

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