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[Sunny2701]

https://www.npr.org/sections/health-shots/2018/04/18/602914728/gene-therapy-for-inherited-blood-disorder-reduced-transfusions


Results of a study published Wednesday show that 15 of 22 patients with beta-thalassemia who got gene therapy were able to stop or sharply reduce the regular blood transfusions they had needed to alleviate their life-threatening anemia. There were no serious side effects.

"We're extraordinarily excited about these early results," says Alexis Thompson, a professor of pediatrics at the Northwestern University's Feinberg School of Medicine, who helped with the study released Wednesday.

"For the first time ever, we have a treatment that we might offer to all our patients," says Mark Walters of the University of California, San Francisco, who also helped conduct the study.

[Andy]

Bluebird Bio of Cambridge, Mass., plans to seek approval of the treatment in Europe by the end of the year, a spokeswoman said in an email.

[Andy]

I have confirmed with bluebird that approval is being sought this year in Europe.

[Sunny2701]

That's wonderful news Andy, thanks for reaching out to them

[dq]

I have confirmed with bluebird that approval is being sought this year in Europe.

Andy, the Luspatercept trials have opened their doors for Thalassaemia Intermedia and from what I understand the trial is running for 46 weeks at minimum.

Two different treatments entirely but;
Would you recommend holding of starting the trial if one has the option of joining it in the hope that Lentiglobin gets approval sooner and is a better treatment option?
Or would you recommend going on the Luspatercept trial if at all possible?

Thanks pal.

[Andy]

I would advise joining a Luspatercept trial, but with the understanding you may be given the placebo.

Luspatercept should be much more financially viable than gene therapy. That will be a big factor.

[dq]

Perfect - thank you. 

I will update you if I get any new news on getting on the trial. Yep unfortunately there is the Placebo possibility

I'm just a little disappointed by the numbers, from what I understand only around 1.5hgb is really achievable in intermedia patients?  Or have I read that wrong and more is possible?

Thanks mate.

[Sunny2701]

Sharing the below excerpt from the link

https://chicagotonight.wttw.com/2018/04/23/study-gene-therapy-blood-disorder-ends-need-transfusions

For 17 years, East Side resident Julissa Perez had to get monthly blood transfusions to treat an inherited blood disorder called thalassemia. The condition left her fatigued and resulted in jaundice, or yellowing, of her eyes. “I was tired and I would take lots of naps,” she said.

But thanks to gene therapy, Perez has not needed a blood transfusion since October 2014. “I feel great,” said Perez, who is a junior at St. Xavier University, where she's studying biology and psychology. She says it’s a “big relief” to no longer need monthly blood transfusions.

Perez was one of 22 patients to participate in gene therapy clinical trials for transfusion-dependent thalassemia. “Of the 22 patients, 15 are now transfusion independent, and what we mean by transfusion independent is they have not received a transfusion in over one year and some for nearly four years,” said Dr. Alexis Thompson, lead author of the study and head of hematology and director of the comprehensive thalassemia program at the Ann & Robert H. Lurie Children’s Hospital of Chicago.

[dq]

I would advise joining a Luspatercept trial, but with the understanding you may be given the placebo.

Luspatercept should be much more financially viable than gene therapy. That will be a big factor.

Perfect - thank you.

I will update you if I get any new news on getting on the trial. Yep unfortunately there is the Placebo possibility

I'm just a little disappointed by the numbers, from what I understand only around 1.5hgb is really achievable in intermedia patients?  Or have I read that wrong and more is possible?

Thanks mate.

[Andy]

dq, I don't think we can draw any conclusions about the long term effect of Luspatercept until the phase 3 trial results are reported. I do know that some patients in the trials are doing very well.

[dq]

Perfect - thanks Andy.

I have a question for you and I'll post it in the thalassaemia intermedia forum - little upset about something..

[Sunny2701]

bluebird bio, Inc. (Nasdaq: BLUE) announced that data from ongoing LentiGlobin clinical studies in transfusion-dependent β-thalassemia (TDT) and severe sickle cell disease (SCD) will be highlighted in oral presentations at the 23rd Congress of the European Hematology Association (EHA).

“As we progress towards our European regulatory filing for LentiGlobin in TDT later this year, we look forward to sharing the updated data from our Phase 3 HGB-207 study of LentiGlobin in people with TDT,” said David Davidson, chief medical officer, bluebird bio. “In addition, we are pleased to be able to share new data from our HGB-206 study of LentiGlobin in people with SCD, which will include data from patients treated under our updated study protocol using plerixafor mobilization of hematopoietic stem cells, and with LentiGlobin gene therapy manufactured using the refined process.”

Oral PresentationsLentiGlobin Gene Therapy for Transfusion-Dependent β-Thalassemia (TDT) in Patients with Non-β0/β0 Genotypes: Updated Results from Northstar-2 (Abstract S833)

Presenter: Franco Locatelli, M.D., Ospedale Pediatrico Bambino Gesù, Rome, Italy

Date & Time: Saturday, June 16, 11:45 a.m. CEST (5:45 a.m. EST)Location: Room A8

Let hope for great results