Thalassemia Patients and Friends

Discussion Forums => Working Towards a Cure => Topic started by: Andy Battaglia on April 01, 2006, 08:48:37 PM



Title: Research Priorities for Thalassemia
Post by: Andy Battaglia on April 01, 2006, 08:48:37 PM
What should be the priorities for research about thalassemia and its treatments?

Each member may vote for up to three items in this poll, and you can also change your vote if you change your mind.

If you have issues not included in the poll, please add them as a reply.




Thanks go to Sally for the idea for this poll.


Title: Re: Research Priorities for Thalassemia
Post by: §ãJ¡Ð ساجد on April 01, 2006, 10:50:16 PM
Hi,
    I would have selected BMT instead of this other option, but I have heard that the stem cells taken from the Umbellical Cord (a.k.a Cord Blood) is less prone to rejection than the ones taken from the Bone Marrow. So I would prefer more research to be in Cord Blood department as this process is painless for the Donor than BMT.

Sajid


Title: Re: Research Priorities for Thalassemia
Post by: §ãJ¡Ð ساجد on April 11, 2006, 07:38:08 AM
Well, I can see that cord blood is now in the poll too. I will re cast my vote so that the statistics make more sense and remove my vote from "others"


Title: Re: Research Priorities for Thalassemia
Post by: mustho on April 25, 2006, 01:38:08 AM
I think future of thalassemia research should focus more on to develop iron chelation, as gene therapy and BMT are not that easy things to do. Iron chelation from inside red blood cells may be the future treatment of thalassemia and that may even decline need of regular blood transfusions. Pathology of thalassemia is inbalance of globin chains inside red blood cells, but medical science have not fully explained the iron mediated cell damage due to iron iside cells. I think if we can successfully reach to solve this problem may be the treatment of thalassemia without transfusion.


Title: Re: Research Priorities for Thalassemia
Post by: Poirot on April 27, 2006, 01:32:11 PM
Pathology of thalassemia is inbalance of globin chains inside red blood cells, but medical science have not fully explained the iron mediated cell damage due to iron iside cells. I think if we can successfully reach to solve this problem may be the treatment of thalassemia without transfusion.

Hi,

Could you explain this in a little bit more detail?

I thought, by definition, Beta Thal Majors, either do not produce RBCs, or enough of them to maintain their HB levels. And, RBCs have a natural life, to boot. So, how does "intra-cell" chelation work in maintaining HB or doing away with transfusions? And, how you retain the oxygen carrying capacities of the RBCs if you are going to do away with the iron?

Thanks

Poirot



Title: Re: Research Priorities for Thalassemia
Post by: Andy Battaglia on July 09, 2006, 02:34:46 AM
I would like to remind all members to vote in this poll. Many doctors and researchers visit our site. Let them know what you think are the top priorities in thalassemia research.


Title: Re: Research Priorities for Thalassemia
Post by: alnahrahmea on March 16, 2007, 11:44:54 PM
I am not very sure, but since hemoglobin is blood related, I believe that stem cell research could be a very promising study that will help all Thalassemia patients.


Title: Re: Research Priorities for Thalassemia
Post by: kabir_love on April 01, 2008, 08:52:28 AM
all i can say is so many options......... then we dont have to wait longer

my personal exp.. if the cure will come it would be in gene therepy..  as per my knowledge which 1%..   lol


mikcy


Title: Re: Research Priorities for Thalassemia
Post by: healinglights on June 18, 2008, 06:33:26 PM
I agree that there are many options,  gene therapy on the surface would appear to offer the best possibilities for all types of thals, ( I don't meand to trivialize this) but in my heart I believe that in the end the answer is going to be so simple we will all wonder why we suffered so much for so long.  Something like a shot of anti-malaria vaccine or some other vaccine created for some other purpose.  Its now being established and more widely accepted that the cure for autism is actually PREVENTION, simply not giving vaccines to very young children.  Sometimes the answer is so simiple its unbelievable.


Title: Re: Research Priorities for Thalassemia
Post by: cherieann on June 23, 2008, 04:43:17 AM
That was hard to vote.
All are a priority to us in one form or another.
Quality of life would sum up what we want... don't you think?


Title: Re: Research Priorities for Thalassemia
Post by: nice friend on June 23, 2008, 07:43:31 PM
Hi :
i havevoted 4 three options:

basicaly i want to say that take  hold on the problem from routes  ... u cant achieve targets by handling to the logs ....iron overload and osteoprosis like problem r the logs ( in my view ) route cause is thalassemia so try to prevent thalassemia and then  focus on research and development sector for people who r suffering.....
 
1 ) Before Marriage Testing is the best choice,  bcoze we cant provide medical facilitys 4 all thal all around the world.....

2nd reason prevention is better than cure .

3 reason is how much thals u can manage to treatment .. fine and easy way is to think abt future ...

2 ) Stem cell and cord blood transplantation for Thalassemia

it is for the thals who r living in this era .... so then they could b able to live a healthy  life without transfusion ..... if u can live without transfusion u will never face the problem like iron overload ..

3) Gene therapy for Thalassaemia major

same reasons to vote for this ....  ... research will ring hope for those  people who r suffering to thalassemia .......
--------------------------------------------------------------------------------------------------------------

well as today many of thals r living all around the world u cant look after them .....





Title: Re: Research Priorities for Thalassemia
Post by: suk838 on April 16, 2009, 04:35:30 AM
Vote for

Gene therapy for Thalassemia major

Management of health issues in aging thals

Stem cell and cord blood transplantation for Thalassemia


Good luck for the research for the coming cure for all thalassemia patients


Title: Re: Research Priorities for Thalassemia
Post by: mohamed on October 29, 2009, 08:00:01 AM
vote for
Increasing Hemoglobin levels in thalassemics
Management of iron overload in Thalassaemia major
Management of osteoporosis in Thalassaemia major


Title: Re: Research Priorities for Thalassemia
Post by: Keep Smiling on November 19, 2009, 07:02:31 AM
i think gene therapy is the best option for all thalassemics............


Title: Re: Research Priorities for Thalassemia
Post by: nice friend on November 20, 2009, 07:54:33 AM
@ Keepsmiling ,
Friend , though genetherapy and other treatments has a potential to cure but in the end  these treatments are risky and painfull , so , my 1st choice is Prevention and awareness  campaigns ..  i would like to say here that " try to controle n stop thalassemia to spread bcoze its most important in every aspect , all these treatments come's after that " .... anyways , u r right as welll ..

C ya bro .. dont mind wat i said ,,  if it is something offending u , then plz ignore it ..

Best Regards
Take Care
Umair


Title: Re: Research Priorities for Thalassemia
Post by: Keep Smiling on November 21, 2009, 04:00:49 AM
there is nothing offending in it, the points u mentioned can be taken as  preventive steps...............
but once thalassemia happens to hit u then treatment especially genetharapy is the best option to root out the basic cause of thalassemia..
Hope u doing well....................

Abbas


Title: Re: Research Priorities for Thalassemia
Post by: mohamed on April 06, 2010, 07:13:45 AM
i think hbf inducers are safer, if there is a potent hbf inducer; thalassemia could be a drug based disease as any other disease (other than a transfusion dependent disease which is the major cause of the most serious co-morbidities in thalassemia). antioxidants role in preventing apoptosis and red cell death may help in parallel to HBF induction. but this needs alarge scale of researches to get the best formulation with the best intense dose to achieve both aims.


Title: Re: Research Priorities for Thalassemia
Post by: 7assan on June 11, 2010, 01:03:15 PM
I think gene therapy is the only solution for patients with thalassemia. Because after gene therapy the patient does not need to transplants or medications province the proportion of iron in the blood or other medications. Because after gene therapy patient recover from thalassemia. :rolleyes


Title: Re: Research Priorities for Thalassemia
Post by: Razia Khan on August 19, 2010, 12:56:14 AM
I think cord blood storage is the best option. Dont they say that prevention is better than cure. Especially now that its available in Pakistan, every parent should get it done for every one of their child.

Regards


Title: Re: Research Priorities for Thalassemia
Post by: ankita on September 05, 2010, 01:56:28 PM
cord blood treatment


Title: Re: Research Priorities for Thalassemia
Post by: love and prayers on February 05, 2011, 02:21:25 AM
"Patient recruitment and enrollment in clinical trials is a major challenge. Among people who suffer from severe chronic illness, only 6 percent participate in clinical trials. Almost 50 percent of trial delays result from patient enrollment problems and 86 percent of all U.S. clinical studies fail to recruit the required number of subjects on time. Industry wide there is a 20-30 percent patient dropout rate in Phase II/III studies."

http://www.nhlbi.nih.gov/meetings/workshops/thal_workshop.htm


Title: Re: Research Priorities for Thalassemia
Post by: Parin on March 02, 2011, 09:49:01 AM
Dear All,
Any research or cure coming in near future i mean in 1-2 year.....I read lot many things but no one talked about the time.....any one have any thoughts?


Title: Re: Research Priorities for Thalassemia
Post by: DrRajpal on October 02, 2015, 03:19:21 AM
I would like to remind all members to vote in this poll. Many doctors and researchers visit our site. Let them know what you think are the top priorities in thalassemia research.
ohhh
sir we are 5033 members here,
but participated in it only 127.

sir plz make any system that such polls should come on priority on every visit.
i could see it today , i feel nervous , i am here since long time but participating in it so late.

sir what i know till now,
5033 no. for any research will be one of rare for data collection.
we all should participate in it.

dear friends Dr Andy is a person who is here only for selfless service, we all should respond on his every action.


Title: Re: Research Priorities for Thalassemia
Post by: DrRajpal on October 03, 2015, 03:15:47 AM
That was hard to vote.
All are a priority to us in one form or another.
Quality of life would sum up what we want... don't you think?
Thalassaemia is a lifestyle not a burden.
best quotation i have heard till now,
mem can i use it in my campaigns ,

Qality of life is big concern for thals ,
as all of us know - it would be best if gene therapy gets success , but what till then?
we can work for-
reducing iron overload,
reducing amount of blood getting transfuse,
reducing Endocrine related disturbances,
reducing osteoporosis,
retardation of ageing,
reducing secondary infections,
protecting liver , spleen, heart , kidney , lungs, ears, vision, joints etc.
increasing time between two consecutive blood transfusions.

we all can gets these by simply Yoga , Ayurveda , and Naturopathy.