Discussion Forums > Working Towards a Cure
More Good News From Bluebird Bio
Zaini:
Sharmin there was a Pakistani patient in trials ????
Also would hb of 10 be enough to stop bones from expanding ?
Sharmin:
Hi Zaini,
Yes, one of the patients in the study is of Pakistani descent. Click on the link, there's info about all of the patients.
I'm not sure that the hemoglobin will level off at ten, but I was thinking that I would be comfortable with a level near 10. Many thal minors have levels near 10 and they generally do well. Also, if the patients are making normal adult Hg they will have more normal sized Hg so they will actually be better off than thal minors. Obviously it would be nice to achieve higher levels, and possibly that will happen - but reaching a level of 10 will eliminate transfusions, iron overload, chelation etc - and certainly for thal majors it would mean a longer and healthier life for thals. My grandma had a Hg of 9.7 to 10 her entire life. She lived to be 90 years old and was the most active person I know - perhaps that is where my perspective comes from.
This is all quite new - but it seems with each patient they learn more - just like how we've learned from transfusions and chelation - what optimal levels are for effectiveness I think they will learn with this treatment.
Hoping for the best! :hugfriend
sofear:
Are you people using different units? 1, 10, 100 – it's a bit confusing! :huh
Sharmin:
In Canada we do use different units - I sometimes inadvertently use Canadian units. I will correct it in my post.
Btw so fear, I understand that you are a thal minor. Do you have a thal major child or family member who can benefit from gene therapy? I am hopeful that many people will benefit in the near future.
Canadian_Family:
Joining the conversation late. Indeed, this is good news for all of us. Good Luck to all patients and their families.
Although the results are discussed in length here, I think 'cost of treatment'. This is a concern in my mind, how Bluebird intends to make this cure cost effective. There are many patients waiting for the cure, but is it going to be economical for them. Approx $700 thousands to $1Million (I think we discussed this in the past, please correct me if I am wrong) is not something I am willing to take for an answer.
My only hope is very large production of vector to bring the fixed cost down.
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