Thalassemia Patients and Friends

Discussion Forums => Curing Thalassemia. Bone Marrow & Cord Blood Transplant => Topic started by: dq on June 17, 2017, 01:52:36 PM

Title: Newest Treatments
Post by: dq on June 17, 2017, 01:52:36 PM

Hello Andy,

I was wondering if you had any news, documents or sites relating to upcoming drugs.
I've looked into Lentiglobin and Luspatercept however do you know of anything else going on abroad and in the UK.  I understand something in the UK is going through phase 3 trials?

Cheers mate.

Title: Re: Newest Treatments
Post by: Andy Battaglia on June 24, 2017, 05:37:49 PM

It was recently announced that the phase 3 trials for Luspatercept are now full and underway. There is a very good chance it will be approved by 2020, if not before. This is probably the drug that is in trials in the UK, as they are doing trials in multiple countries.

Title: Re: Newest Treatments
Post by: dq on June 24, 2017, 11:54:42 PM

Thanks pal.  What about Lentiglobin? Or is that something completely different?

Cheers

Title: Re: Newest Treatments
Post by: Andy Battaglia on June 27, 2017, 12:08:40 AM

Lentiglobin is the gene therapy product. That is a full cure and trial results have continued to improve as they have improved the product. 3 patients are now completely off transfusion AND chelation. That latter part is huge, as iron load has continued to be an issue for some patients after bone marrow transplants.

Title: Re: Newest Treatments
Post by: Pratik on June 27, 2017, 06:17:29 AM

Andy,

What happened to that Acceleron pharma's drug? Is it still in development?

Title: Re: Newest Treatments
Post by: Andy Battaglia on June 28, 2017, 05:19:57 AM

Acceleron and Celgene decided to work together on Luspatercept, the more promising drug.

Title: Re: Newest Treatments
Post by: Pratik on June 28, 2017, 05:47:20 AM

Quote from: Andy on June 28, 2017, 05:19:57 AM

Acceleron and Celgene decided to work together on Luspatercept, the more promising drug.

That's good to hear.