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Inducing Fetal Hemoglobin Production in Beta Thalassemia

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Andy Battaglia:
One of the most intriguing fields of research into a cure for thalassemia is turning on genes so that the body will produce hemoglobin on its own. eliminating the need for transfusions and all the related problems. This is already being done with chemicals such as hydroxyurea and arginine butyrate, in thalassemia intermedia. Research suggests that there may be other chemicals that both work better and  are more tolerable. However, development is being hindered by the costs of drug resreach and development.

I found an excelent analysis of this topic at

http://www.asheducationbook.org/cgi/content/full/2005/1/38


--- Quote ---The ß thalassemias are one of a few medical conditions in which reactivation of a gene product that is expressed during fetal life can functionally replace a deficiency of essential proteins expressed at a later developmental stage...In clinical trials, three classes of therapeutics have demonstrated proof-of-principle of this approach by raising total hemoglobin levels...New oral therapeutic candidates, which stimulate both fetal globin gene expression and erythropoiesis...now make this gene-reactivation approach feasible to produce transfusion independence in many patients. Development of the candidate therapeutics is hindered largely by costs of drug development for an orphan patient population...Major obstacles to development of HbF-inducing agents for thalassemia include: 1) costs of new drug development, including the challenges of pharmaceutical companies weighing the potential market and academic investigators securing financing for drug development; and 2) perceptions regarding the ultimate efficacy (and competitiveness) of this therapeutic approach compared to other approaches. For example, many thalassemia patients are managed with blood transfusions and iron chelators, and the oral chelators are demonstrating therapeutic effects. Therefore, only a fraction of the thalassemia population might initially utilize a HbF-inducing type of therapeutic, even though such drugs would improve the underlying condition...

Summary

The ß thalassemia syndromes are among the few molecularly defined conditions in which an endogenous fetal gene can functionally replace a defective adult gene. Several candidate therapeutics have demonstrated proof-of-principle in these disorders by inducing fetal globin expression and subsequently raising hemoglobin levels in ß thalassemia patients. The magnitude of responses to individual agents is similar to those induced by rhu-EPO in anemic conditions with normal red cell survival, and additive responses occur with drug combinations. New oral candidate therapeutic agents, which both induce fetal globin gene expression and stimulate erythropoiesis, and combinations of therapeutic agents with complementary molecular actions, now make this gene-reactivation approach feasible for many patients. Development of these agents is now hindered largely by costs.
--- End quote ---


I am curious as to whether patients agree with this quote


--- Quote ---... many thalassemia patients are managed with blood transfusions and iron chelators, and the oral chelators are demonstrating therapeutic effects. Therefore, only a fraction of the thalassemia population might initially utilize a HbF-inducing type of therapeutic, even though such drugs would improve the underlying condition
--- End quote ---

Do you feel this way or would you be willing to try a drug that might increase your Hb to the point where transfusions are no longer needed, or do you feel the way your thal is managed through transfusions and chelation is the tried and true method and you wouldn't be willing to take the chance on an alternative approach?

§ãJ¡Ð ساجد:
I'll buy anything as long as it doesn't involve any needles! :biggrin

O.k I can be a little flexible in that. A shot instead of the hours long TransX will do just fine too as long as it is monthly or more apart.

And as a last resort....

ANYTHING no matter how extensive it is (Such as BMT or CBT) as long as it cures me once and for all.

Andy, where do I sign up for being a lab-rat in this process ? :biggrin

Take care, Peace!

Christine Mary:
HI ANDY AND SAJID.

PLEASE DONT LAUGH AT ME, BUT WHAT IS THIS LINK SAYING? THAT THERE CAN/WILL BE A POSSIBLE CURE FOR THAL MAJOR, BY INTRODUCING ORAL MEDS?
I KNOW THEY ARE PROMISING IN INTERMEDIA PTS,BUT WHAT ABOUT MAJORS?
ALSO, IN YOUR OPINIONS,(AND EXPERIENCE) DO YOU THINK THERE WILL BE A CURE IN OUR LIFETIME? :pray

CHRISTINE :hug

Andy Battaglia:
Basically this article says that this is already proven that it will work, but that more than one chemical is needed because no chemical works for all thals. More research needs to be done but as the article hinted quite loudly,
--- Quote ---Development of the candidate therapeutics is hindered largely by costs of drug development for an orphan patient population.
--- End quote ---
The patient population isn't perceived as being large enough to justify the expense of research and development of these drugs. These researchers seem sure that this will work for all beta thals, intermedia or major.

Well, if we go beyond the US, we see that the patient population is quite large enough to justify this research. But how do we get funding for it? How do we get thal the notice that things like Aids get? This is a real challenge and it may take novel approaches to gain the attention needed. Face it. Thal is mostly a problem of the poor in countries like India and Thailand where tens of thousands of majors live...and die. We don't have a world that cares enough about the plight of poor non-whites. It is so sad but also so true. If there were 10,000 babies born in the US with thal every year, it would be getting all the attention it needs. But in India, the resources don't exist so the research doesn't happen. We need a world stage. We need attention if these things within our grasp are ever to be made a reality. Even the gene trials have been slow to come because of money.

I do believe a cure for thal, and maybe more than one approach is on the horizon, but it will take money and that will take thal being noticed. We are doing our part in our global village that is this group (thanks for the term, Miaki  :wink ) but can we honestly say that the international organizations are doing their part? TIF, exactly what is it that you do other than arrange conferences in exotic locales? Where is the day to day help for thals that is found in the Cooley's Anemia Foundation in the US? And TIF, does it bother you that more than one thal active in the orgs has told me that I have single handedly done more for thals than all of TIF??? I would be very embarrassed if I was running TIF. If I, a single person with no financial resources can find ways to help thals all over the world, then why can't TIF do a better job???

We can't sit and wait to be noticed. It won't happen. We have to make noise if thal is going to get on the radar of those who can make things happen. TIF isn't accomplishing this so maybe t's time we look for other ways to make it happen. We live in the media age and savvy people will find ways to be noticed. Let's make it happen.

Christine Mary:
please,please tell me what we can do to raise hell and make ourselves heard! lol i wrote oprah last week (and suprise,suprise,havent heard back).
should we contact our senators,govenors,congressmen? start a global petition?
c'mon people! there are some brilliant minds on this forum, that could make a change, if we just figure out how!
WHAT WILL IT TAKE TO RAISE AWARENESS? HOW CAN WE HELP FUND THE RESEARCH FOR A CURE????? just tell me what I can do to help, and im there!

christine

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