Bluebird Bio Releases Data on Current Gene Therapy Trials

As Sharmin said... Fertility issues and post transplant medication/care.

Thanks Andy

Andy,
Thanks for talking to Bluebird....Few questions in my mind is...

1. which hospitals in US are they partnering with for these trials.
2. what are their main challenges and setbacks so far....not asking about financials.. from patient safety.
3. when can we expect it to be a mainstream cure...covered by insurance.

there are so many questions...but answering these few wiil do I am sure other members and you have additional questions.

Andy,
can we also understand the reason for variance in hb (beta-T87Q) patient to patient post administration of Lenti-globin? How they are trying to achieve a similar hb increase for different patients.
And obviously safety concerns is another must ask.

thanks Andy!

I'm not sure if this is valid question - but I wonder about the interim period - while waiting for the new treated marrow to engraft, how the patient is supported.  I am guessing transfusions of red bloods cells and ivig?

I don't know if I put by question currently: my question was why there is difference in hb increase post gene drug administration between patients

is it because of varying amounts of vector being administered??

There are many variables, including the fact that each patient is unique. However, dosing is still in the testing stage, so the dosing has not been the same for each patient. Time and experience will refine the process.

I think the events of the past week have made it look like it was poor timing for people at Bluebird to not wait another week and make a fortune before selling stock. What I have learned is that it actually is impossible for anyone involved in the company to sell their stock on their own. Each one has a program that has set prices at which X amount of shares will be automatically sold. That is the only way that their own stock can be sold, so they cannot even react to events after they've been made public, so this means they can't do anything even though the stock more than doubled in the past week. I really like their arrangement. The only way they will make big money is by producing a successful product. Even if their only motivation was money, this would be enough incentive to do it right. But I have met people from Bluebird and spoken with others on the phone and there is more to it than money. They really do want for find cures for people. It's about money, but it's also about doing good for others. That's something I can really respect.

The importance to us is that we won't learn anything new from watching their financial picture. The news will come in these periodic announcements at conferences. What is most exciting is that the news has been quite good. Let's hope the trials continue to show success without any unforeseen obstacles. I would describe the people at Bluebird as cautiously optimistic. We're watching the world change right in front of us. After so many years of seeking a road to cures for so many disorders, we have finally found that road and begun our journey.

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.

I think I left out a little bit of information that everyone wants to know. We are hopefully looking at a time frame of 5-10 years before we see FDA approval. Of course, we've all seen how slow things move in the real world, but let's be optimistic about this.

Andy,

Thank you so much for all of this information.  What an incredible source and advocate you are for us.

Incredible, Incredible, Incredible..

Thanks Andy for sharing the information.

Thanks Andy for all the update, it is indeed great & lucky to have you along side

I am joining the party little late, busy with work...

Great news from Bluebird, Andy thanks for clarifying lot of issues. With respect to shares, its good to see financial strength, I like the way 'insiders' dispose their shares (learned something new here).

I continue to hope that Bluebird make progress, however, I still believe MSKCC can bring a lot of value to the table.

PS. I do not hold stock position in Bluebird

Andy,

I agree with your statement about seeing the humanity in this group and the research they are doing.  I have seen a few videos and reviewed some of the companies policies and to be honest I do so a lot of good will on the part of bluebird bio.  

I also see goodwill in Michael Sadalaine, the initial errant gene group and our good friend Pat Girondi.  Let's pray that their research continues and yields strong results in the near future.

Sharmin

Andy, Thanks you so much for the detail sharing. Can't even have enough words to Thank you for all the things you do for this group. Your statement about Bluebird bio's management makes me feel very hopeful that it is not only money...but they do care for the patients. Praying for continued success of Bluebird....