Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials

The timing of the release of this video is encouraging - as of yet however we do not know anything for certain about the progress of the gene therapy trials:

http://www.youtube.com/watch?v=gPPKvouhLvE&feature=youtu.be

That's certainly very encouraging.

Really it was an amazing video. Vey Very encouraging . I just pray to God that the resuts come shortly and it becomes  equally encoraging for all of us.
God bless all of us
Bye

Keeping you in the loop.

Update February 19, 2013

Dr. Boulad and Dr. Sadelain provided answers to your questions. They said the protocol is currently open until July 2014 and patients are still being enrolled. The timing of the results will depend on patient accrual numbers and the levels of gene expression that are seen. They hope these issues will both be solved by July 2014. If it moves forward, this therapy would be for all types of Beta-thalassemia major independent of the Beta-globin gene mutation. In order to start treating patients affected by Beta-thalassemia, all (or at least the majority) of patients would need to engraft with the gene and become transfusion independent without any major side effects. The next steps would be production of the vector in larger scale, FDA approval for the vector, and extending the trial to a Phase II study. Licensing the vector to a biotech company may be necessary to broaden access to this therapy, just as it is with any new medication, chemotherapy, or biologic agent.

As mentioned in the youtube video posted by Sharmin, it takes about $1million to prepare the vector for small number of patients. Licensing to a biotech compnay should bring the cost down.

Best of luck to everyone.

Canadian Family,

Thank you kindly for the update.  It seems like we have taken the first steps in the right direction - and a lot is pending on gene expression in the patients and subsequently financing and manufacturing of the vector.  I hope that more patients will enroll in the study and be cured of thalassemia. 

I hope we begin to see excellent results from the first few patients!

Sharmin

I received this note from Pat G today.

Dearest family,

The 2 treated patients continue to do fantastically. A third patient has already done the stem cell mobilization and will do the 'Gene Transplant' in July. The team will decide whether an increase in chemotherapy is necessary for the next 3 patients.

Thanks for Great News Andy sir, Millions of Thalassemia patient,their family member and their well wishers prayers are finally heard. I am waiting for a day in my life when i will read that now thalassemia is 100% cure.   

I am so glad to hear this.
Zahra

We are all waiting for a day when thalassemia and related hemoglobinopathies are
completely and safely curable.  What this
preliminary news is telling us is that the conditioning and treatment appears
to be safe and there do not appear to be significant side effects
at this time.  This is major, major and wonderful news - because the only
other known cure - BMT - is a grueling procedure plagued with major side effects and
life threatening risks.  To know that this form of gene therapy - were it to be successful - seems
to be safe - is exactly what we need to hear.

I pray that the trials are successful with the low chemo
preparation presently being used - and if additional prep is required
I pray that it won't require high doses and certainly not myeloablation.

Praying for the best

thanks andy for providing the information. I pray that the treatment is successful and all the patients are cured. God bless all

It cannot be any other great than May 08 (World Thal Day) to hear this news.

Thanks Andy and I hope we all shall see the ray of light, the ray of hope, soon.

-P.

Another update from Pat G.

Yesterday it was reported that the first treated patient made it 40 days before transfusing again and that the second made it 60 days. This is not proof that Thalagen works, as chemo therapeutic agents can also stimulate higher hemoglobin.

We do however know that there is 'Gene Expression' and that the patients have tolerated the treatment without incidence.

As always, we have to be patient as this unfolds, but the fact that gene expression is observed is is a major development.

Amazing, thanks for the update

It seems gene therapy really fascinating corporate to invest..... future for Thalassemia is bright, keep faith and patience

http://itsoftware.pharmaceutical-business-review.com/news/sangamo-receives-grant-to-develop-zfp-therapeutic-for-beta-thalassemia-270513

That is fascinating. I am glad to see stem cell research advancing.

wow, this looks great!