Is Thalassemia a Big Enough Problem to Justify Research into a Cure?

We are often told that thalassemia is not a big enough problem or not important enough to warrant research dollars being spent on finding better treatment or even a cure. But is this true? Are there really not enough thals in the world to justify money being spent when problems like AIDS seem to command everyone's attention?

The answer may surprise many people. The answer is a most definite YES!

I would like to quote some information regarding this topic. This is an article I have mentioned before and I would like to salute Dr Vichinsky for his efforts to both educate and to find solutions.

From http://www.medicalnewstoday.com/medicalnews.php?newsid=26267

Thalassemia is the world's most common genetic blood disorder and it is rapidly increasing. Seven percent of the world's population are carriers, and 400,000 affected babies are born each year. The World Health Organization (WHO) predicts a doubling of these statistics within the next few decades. Once rare in California, it has become the most frequent disorder detected in the state newborn screening program.

Seven percent of the world's population are carriers! This translates into over 400 million carriers in the world! 400,000 affected babies born annually! These are amazing statistics! How dare anyone say that thal doesn't affect enough people to justify the attention needed? We are on the verge of a major worldwide thal problem and as demographics shift we will see far more cases in countries like the US where previously thal was thought to be a minor problem. It is not!

What is needed is a major continuing program of awareness and prevention education and groups like ours can do their part and each and everyone of us can do as I and Narendra do, by educating every single person we can in our daily lives about thalassemia. We can continue to seek out venues, especially in the media, to help publicize and educate. Shilpa has long suggested the idea of trying to get the attention of Oprah, and I have seen that many others agree that this would be a great way to reach the worldwide public. I have talked to Shilpa many times about this and we do have a basic idea of proposing that a panel be organized to appear on Oprah consisting of doctors, patients and advocates. If we can get our foot in the door, I do believe we can organize a show that would be very effective in bringing the attention that thalassemia needs.

Andy

Yes, it is AWARENESS.

Please lets start to put a plan and see what each of us can do. We should concentrate on that topic and put targets to be achieved.

Concerning my community i would like to tell you that i have been going through many many connections ( you can't imagine how hard this is) in order to reach people who are responsible for a certain program that is very much seen , not only in Egypt but in the whole middle east ( it is as famous as oprah in the middle east). The good news is that tomorrow FINALLY i will be able to arrange a meeting with the producer of the show. I plan to introduce all the subject for him. I had previously talked to the most known hema. in thal in Egypt and by the way she is the president of the Egyptian thal Assosiation, Dr. Amal el Beshlawy and she is wishing to participate. I had a friend who works in Novartis who is investigating if it is permitted for Novartis to have a representitive to talk about the Exjade in a program  or not.  I have three targets from this show, first is to spread awareness ( through three show, but i will start with the most seen )because when you mention the word thal in Egypt, the answer is,'' oh , feed the child properly he needs more iron in his food'' . Second, is to make a fund raising in this program for the thal kids in the govermental hospitals. A couple of months this show collected  $3,000,000 in just one episode for liver transplant patients. Third , arrange for a blood drive.
Anyway, if things went smoothly i will tell you the details and the name of the show.

Another thing i am preparing right now is a memo that i will submit to the health committi in the People's Assembly in Egypt (like the congress in the USA).  Fortunatly my cousin is a syndigate and he will help me in this. My three main concerns in this memo is to make the goverment decrease the price of exjade by enduring part of the price ( i don;t know the exact term in english) cause it is VERY VERY expensive in Egypt. Second,  put a law by which there is compulsery blood analysis to be made before marraige in additional to prenatal diagnosis since thal is a genetic disease that can spread like a bomb. Third, is to try to make the needs of thal patient available because, he is a normal person that can be beneficial to his community if all his needs ( desferal, exjade,blood,.....) are there.

To tell you the truth, i don't count on this memo because things doesn't go that smooth in the Peopl's Assembly, but i will submitt it anyway.

Third thing i will start to work in is to address some of the multinational companies ( like McDonald's, Coca Cola,.... ) because they have activities toward the community in certain areas like cancer in children, hepatits C,....I don't know still how to reach any of these, but if i did i will propose to donate one egyptian piaster for example( one pound is 100 piasters) on each sold meal or can of coke to the Egyptian Thal Assosiation on the 8th of May.

Anyway, i want to make many things for the sake of awarness and Andy's post made me tell you my plans though nothing is concrete right now,but i hope i will receive something positive tomorrow regarding the show.

I will keep you updated in each of these steps and sorry for this long post

Take care
manal

Manal,

Your efforts are remarkable and you deserve a round of applause.      

By the way, the word you are looking for is subsidized. Yes, because of the high cost of exjade it will be necessary for either government or private charitable organizations and even Novartis, to subsidize the cost to make it more affordable to the patients. Your point about thals being contributing members of society if cared for, is well taken. This is so true. Give them a chance and they can be a positive force in our world. All we are saying is give thals a chance.

Please keep us informed about the results of your efforts. It is very inspirational and I hope others will take some of your ideas and use them in their countries.

Manal, this one's for you   

Andy - I love the whole Oprah idea. I think it would be a great show, and would do so much to inform people about thal. A panel would be very effective. Who would you propse to have on it? Would you consider callilng, writing, or e-mailing the Oprah people to sart the ball rolling? You are so knowledgeable and would be a great spokesperson for us. 

Manal - you are so amazing! You are doing so much and have such intelligent ideas. You have chosen three great ways to spread awareness - by contacting the media, politicians, and powerful  businesses. Nice work!

I am always impressed by both of you. Thanks for doing all you can. Love, Jean   

Hi guys,

When Lauryn was first diagnosed in Aug. of this year, I DID write Oprah and her producers a lengthy letter regarding thal,the need for a cure,and spreading awareness....

Im still waiting.........

Ashish has also written Oprah...and is also still waiting. Anyone have any ideas on how to get an "in" with Oprah?

I do have some  ideas for guests and approach but would prefer to keep them private at the moment, as we have been visited frequently lately by people who have no qualms about stealing ideas and claiming them as their own.

Ah, I understand. I'll look into the Oprah thing, also. Merry Christmas to all, Jean

Thanks Andy and Jean for the nice compliment.   :hug But i want to let you know that any thing that you think is positive in these efforts is because of you guys. All the support and faith that one gets in this site inspires me with a lot of things. If any thing good happenes , it is because of you all .

Anyway, here is the last update, i was able to meet the producer of the show. When i started talking aout thal, he told me that this considered an old issue cause they talked about thal before , three years ago when one of their collegues in the studio discovered that his daughter was a thal major. For a moment i was disappointed and felt that the man agreed to meet me because the one who arranged the meeting was the office manager of the network owner. Actually, i started telling him that he can make a new approch in discussing thal like, DNA tests before marraige, subsidizing the exjade, pre natal diagnosis,......etc and these issues will all lead to thal at the end, and then i started to tell him the statistics that Andy spoke about  at the begining of this topic. ONLY, at this point the guy started listening carefully, he was totally shocked from the percentages of carriers either worldwide or locally in Egypt. That was the only thing that grasped his attention and made him very positive and interested to know more information. SO MANY THANKS TO YOU ANDY FOR THESE VALUABLE STATISTICS THAT SAVED ME IN THIS MEETING. I guess that normal people get interested when they know about the possibility of being a carrier as this could be a threat either to them or to their partners without knowing. When you talk about a disease , normally people are not interested as long as they don't suffer from it ( i used to be like this ) ,but when you tell them you could be a carrier and you don't know , they get really very interested. Anyway, the guy promised that he will do an episode about thal this month(hopefully) and he told me to send him any material that can help. I suggested to have a in the episode a hemeatlogist, someone from Novartis, some one from the health ministry, genetic councellor and a patient if any agrees. I will call him again on January 6th to put things in action, but he promised he will do it.
Concerning the Novartis people, they did not agree to come to the show because they say it is against their policy to appear in the media and talk about the exjade as medicine or to discuss subsidizeng the medicine in the media, but they told me that the hematologist is free to talk about the medicne and anything else related to Novartis or the exjade,but they are not allowed which is not conviencing to me.
Anyway i will keep you updated after i call him on Jan 6th.

Love you all
Take care
Manal

Hi Manal.
Thank-you for all your effort ,and care towards the recognition,research and awareness for  all thals patient.good work
I hope all your hopes and dreams eventuate in the new years.
Love Kathy :hug :hug :hug :hug :hug

Hi Manal,

Nice progress! Great going!

Hoping the best for the rest! 

Take care, Peace!

Hi Manal,

These developments are quite exciting. Have you considered asking a representative from Apotex, the maker of Ferriprox to appear on the show? They are often much more willing to cooperate in a venue where they don't have complete control, as Novartis may desire. The contact information for Apotex can be found at http://www.apotex.com/Contacts/General.asp

Exjade is all the rage these days, but the chelator that has been shown to have the greatest effect at removing iron from the heart is deferiprone (L1, Ferriprox, Kelfer). Heart failure is the number one killer of thals and deferiprone has been shown to be superior to desferal in extending the lives of thal patients. While deferiprone is not for every patient because of side effects in a small percentage of patients (there is no chelator that every thal patient can use), for many patients it has worked very well in reducing their cardiac iron loads. As of yet, there has been little information available about the efficacy of exjade in removing iron from the heart. Apotex is often quite willing to present their information, so it may be worth it to  contact them about the show.

Thanks Kathy and Sajid, you are all behind all these efforts, no body else. You are the inspiration.

Andy, i visited Apotex site and they don't have a branch in Egypt, but i emailed them anyway to ask about the possibilty of having someone in the show. I will be waiting for their response and i will let you know.
Thanks a lot Andy

Manal

Manal,
     That is fantastic news! Good job. I am curious to see how it goes. Thanks, Jean