More Good News From Bluebird Bio

Sharmin there was a Pakistani patient in trials ?

Also would hb of 10 be enough to stop bones from expanding ?

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!  

Are you people using different units? 1, 10, 100 – it's a bit confusing! 

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.

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.

http://wgntv.com/2014/12/30/gene-replacement-could-replace-need-for-life-long-blood-transfusions/

I think the above link and the video will be helpful in answering many questions.  

I also think that the post below - Andy's post from a few months ago will be helpful (the post is old - so please note that we do have more information about sickle cell patients now and the results have been good):

I had a two hour talk with Faraz from Bluebird. I have to say that the more I talk to him, the better feeling I get about Bluebird. It's not often that I hear the Golden Rule brought up in conversation, so I do take note that there is more to Bluebird than money, which is refreshing.

I received answers for most questions that were posed.

Chemotherapy concerns: Chemo brings the same risk of side effects that are present when used with BMT. Loss of fertility is a possibility, but is not predictable, so it is advised the sperm or eggs be collected in advance. Short term side effects from the chemo are a known factor. Reminder: There is no risk of graft vs host disease, removing the biggest concern about BMT. There is nothing to reject, as the patient's own bone marrow is collected and used.

Long term costs: We don't know yet. Patients must continue chelation afterwards until their iron burden is normal, but aside from that, nothing long term is currently known. In theory, there should be no long term issues, but one of the reasons trials are run is to observe over longer periods. As far as long term maintenance, no immunosuppression drugs or other drugs are required.

Are transfusions required after the process? Patients are transfused to a high Hb before the process begins. Only a couple have needed any blood at all after the procedure, and that involved transfusing only small amounts of blood one time. After that, they remain transfusion-free.

Will patients lives eventually become normal? That is the hope and nothing currently known suggests otherwise.

Have there been any setbacks or unforeseen issues? Not so far, and they learn more from each new patient.

Have things changed a lot since the first patient was treated 7 years ago in France? The process continues to become more refined and with each new patient, more is learned. The vector used today is improved and more effective, but there haven't been any major changes. The first patient continues transfusion-free, as have all subsequent patients.

Recruitment for trials: See http://www.northstarstudy.com/resources.html and begin the screening process if interested. The current trials are almost full, but a few more patients will make it into these ongoing trials which are taking place in the US, France, Thailand and Australia. Current patient origins are from France, the US, Pakistan, Australia, Thailand and Syria. More trials will be coming and the age limit will be lifted, allowing younger and older patients to apply. There has been no trouble attracting patients for trials, and with this recent report, we can expect interest to increase, so it is highly recommended that interested patients begin the screening process ASAP, so they can get in the queue and have a chance of being selected for trials. I have seen nothing in any report that should cause any hesitation applying for participation in trials. Bluebird takes care of the cost for the trials, including transportation costs, so do not be deterred by your location.

Re: Sickle Cell: The first patient has been started but it is too early for any reports. With sickle cell, stem cell mobilization has to be carefully done, as the drugs used can mobilize the production of more sickle cells, as well so achieving proper dosing and use of mobilization drugs will take some time and experience. We will eagerly await further reports. It's exciting that the trials have expanded beyond thalassemia.

I have never had any doubts about the eventual success of gene therapy. The waiting has been agonizing, but we are now well on the road to a new era of medicine. We are living in miraculous times and we need to seize the moment and not allow research to be deterred by political buffoons who constantly threaten to de-fund research, as we in the US saw last year with the government shutdown. As world citizens, we should demand better from our leaders. Let's demand that our tax dollars go to helping solve the real problems humanity faces, rather than fund more exploitation of humanity through conflict and wars. There are so many avenues of research taking place to find treatments and cures for a multitude of disorders and diseases, and in most cases, the only thing holding them back is lack of funding for research. We have seen this over the past 10 years regarding gene therapy. We are a couple years behind in the US, due to earlier lack of funding. At every conference, you can find researchers presenting their work, and so much shows real promise, but always the same stumbling block is present and that is lack of support for research. Let's make a conscience decision to support saving humanity, rather than destroying it.

I have attached the latest report as a pdf attachment to this post.

Re: The costs of gene therapy.

In the US, you can expect to have your insurance billed anywhere from $5000-$10,000 per month for Exjade (more for Jadenu), so just one year of chelation is going to be in the $100,000 range. At least for patients in the US, gene therapy will be a cost effective choice. I would think that this would be the case in most developed nations. It's a large cost upfront, but we need to be working on the insurance companies now, so they recognize that in the long term, gene therapy will save them money.

Hi Andy (two thumbs up),

Your point well taken. The cost of treatment in Canada runs about $50000 to $60,000 per year. We have universal health care system, which means Government pays all the bills. Any out of pocket expenses (such as exjade) are covered through insurance.

I am positive that Government will be willing to pay one time for cure and recover the cost in 7-8 years and save that money for unforseeable future.

Brilliant idea...

Andy, Thanks for sharing good news from Bluebird....I pray for Bluebird's continued success !

Zaini, so good to see you back. How is little Z doing? I guess she is not little anymore. My daughter is turning 9 soon...we had joined here when she was not even 1 month old. How time flies