The Latest from Pat Girondi on the Progress of Gene Therapy

Dear friends,

I agree the best thing any thal can do is to take care of themselves to the best of their ability - using all of the resources available to them.  The future is always difficult to predict - and in some cases difficult to even imagine. 

The older thals, who have been hearing claims of a cure for decades are especially likely to be skeptical about developments such as gene therapy. 

It is probably best to live as though there isn't going to be a cure so that everyone can accept their current situation and live full lives WITH thalassemia.  It is very important to be compliant with the treatment and to give oneself the chance to be completely healthy. 

My personal belief about gene therapy is that today there is a much better chance of it becoming a reality than there was in the past.  So much that was only possible in theory a few decades ago is commonplace today - internet, cell phones, robotic surgery, lazers, iphones, etc etc.  The human genome was decoded in 1999 - and then George Bush withdrew funding from these programs brining them to a standstill and poured money into killing people rather than research to improve their quality of life. 

Today with better leadership, more knowledge and experience I think that the possibility is very real - although the timing and funding can sometimes cause problems.  Since the first patient in Paris has been transfusion free for a few years now - we know that with fine tuning gene therapy is very possible and can be successful.  If the two groups working on gene therapy continue to progress it is possible that a cure will come within a few years.  I hope that the trials are on schedule now and that they take place,.

I will talk about myself:

When I posted my last post here, I was talking about myself and nobody else. I said that I do not believe gene therapy will become a reality within my lifetime. I was not talking about younger thals.I trully believe than gene therapy will be a reality for thals like Ahmad or Little A or Zainab or even for older thals.
 In fact, I heard some news about gene therapy yesterday during  my transfusion. The second phase of gene therapy conducted in Greece by the team of Dr Stamatoyannopoulos will possibly begin in January.If it does, this would be very hopeful even for us- older thals, meaning we might catch the train. Although  it is my characteristic not to put my hopes high and lead my life with things I can put my hands on, dreams are always welcome.

Lena

That's great news Lena.  You have set a great example for younger thals whether or not gene therapy becomes a reality.  Your attitude and the way you live - if bottled - would be more useful than gene therapy for all people, not just thals.  You cannot imagine how often I pray that my son grows up to be like you.  I read your posts each day and hope to learn something - not just about thal but about wisdom and strength. I wish you continued - good health, strong will a hopefully a cure for you as well. 

Sharmin 

Sharmin,

Thank you so much for your kind words, you make me blush!
If I, in any way, have succeeded  in managing  my life with thal, I have my parents to thank for it!
They struggled with thal- years back- when no help, no true knowledge were available- on their own, coping with their fears in cruel times.

Lena. 

There are only two phases in gene therapy:
in short, the first is the collection of the sick stem cells from the body and the second one, when the healthy now stem cells are put back after having been corrected with the healthy gene.

Lena.

The first phase was the stem cell mobilization. A major problem is producing enough stem cells in thals to use for inserting the good gene. I have attended lectures twice by Evangelia Yannaki, MD, of George Papanicolaou Hospital, Thessaloniki, Greece, and it is the most complex of lectures to follow. I cannot even begin to explain the process, but the end result is that Sadelain's team, which includes the Greek trial, has had tremendous success in mobilizing stem cells. The results have been far beyond anything seen before, which gives a much better outlook for the next stage, which is actually inserting the good gene into these stem cells.

Seven years ago, it was common to hear older thals say "they've been talking about this new oral chelator for 20 years. I don't believe it will ever be reality." Today, we have Exjade. Yes, these things take a long time to develop and much of this is due to underfunding, but we do see constant progress in treatment of thalassemia. The parents of those born 40 years ago were told that their child would die by age 20. This is no longer believed. Progress is real and in thalassemia treatment, progress is incredible. Compare today to 1970. I agree that for older patients, gene therapy may never be a reality, but it will happen and it will work, but medical progress takes time and money and years of observation to make sure it is both safe and effective, but in the end, we have seen more progress in the field of treating thalassemia than in any other fatal disorder. Thal is no longer classified as a fatal condition, but is now a chronic treatable condition. What other disorder exists where this claim can be made?

I have absolute faith that this incredible worldwide team of researchers will succeed.

Seven years ago, it was common to hear older thals say "they've been talking about this new oral chelator for 20 years. I don't believe it will ever be reality." Today, we have Exjade. Yes, these things take a long time to develop and much of this is due to underfunding, but we do see constant progress in treatment of thalassemia. The parents of those born 40 years ago were told that their child would die by age 20. This is no longer believed. Progress is real and in thalassemia treatment, progress is incredible. Compare today to 1970. I[bgcolor=#ff0900] agree that for older patients, gene therapy may never be a reality, [/bgcolor] but it will happen and it will work, but medical progress takes time and money and years of observation to make sure it is both safe and effective, but in the end, we have seen more progress in the field of treating thalassemia than in any other fatal disorder. Thal is no longer classified as a fatal condition, but is now a chronic treatable condition. What other disorder exists where this claim can be made?

I have absolute faith that this incredible worldwide team of researchers will succeed.

so wht about pat son ...................................?

Pat's son is not an older patient. Pat's son is in his early 20's. By older, I mean >40. And this is simply speaking pragmatically, with some idea of how long it takes between trials and approval. Even then, a patient in good condition who is over 40 and is aggressive in pursuing a participation in trials, might eventually take part in trials.

Au contraire. Andy is seldom bored.