Thalassemia Patients and Friends

Discussion Forums => Working Towards a Cure => Topic started by: mousumideysamui on June 27, 2009, 03:40:19 PM

Title: gene therapy
Post by: mousumideysamui on June 27, 2009, 03:40:19 PM

I am eagerly waiting for gene therapy.

Title: Re: gene therapy
Post by: suk838 on June 27, 2009, 06:01:27 PM

ya me too can't wait for that to happen where every thalassemia can by that.

Title: Re: gene therapy
Post by: Sharmin on June 27, 2009, 07:00:52 PM

praying for it to happen sooner than later  :pray :pray :pray

Title: Re: gene therapy
Post by: Manal on June 28, 2009, 01:37:32 AM

 :pray :pray :pray :pray :pray :pray :pray

Title: Re: gene therapy
Post by: nice friend on June 28, 2009, 02:11:58 AM

waiting since a long time , so i think that it will b available soon, in soon future :)

Title: Re: gene therapy
Post by: souly on October 11, 2009, 10:11:48 AM

Please see the below message from Pat.
My prayer with the team and the patients.
Souly

Dear family,
 
Things are going wonderfully.
 
We have completed the stem cell mobilisation in the first 3 patients. The per centage of cells that we are entering is in the first batch from the lab is HIGH. This is great news.
 
3 months for our first patient.
 
pat

Title: Re: gene therapy
Post by: Andy Battaglia on October 11, 2009, 03:08:45 PM

What this means?

A major obstacle in using stem cells has been collecting a sufficient quantity of a patient's stem cells from the blood in which to introduce the good genes. This obstacle has now been overcome and a more than sufficient quantity of stem cells can now be "mobilized" from the bone marrow into the blood where they can be collected for later use with the introduced genes. This really is a most important step in the process and I agree with Pat that we are now very close to seeing a human cured with gene therapy. It will be exciting to hear what Dr Sadelain has to say at the upcoming conference in NYC.

Title: Re: gene therapy
Post by: Manal on October 11, 2009, 08:16:48 PM

Thank you for the explaination Andy. I thought that this is an ''easy'' procedure since it is done with the donor in BMT, may be there is a different way

Anyway can not wait to know what Dr. Sadelain will say :pray :pray :pray :pray

manal

Title: Re: gene therapy
Post by: Andy Battaglia on October 12, 2009, 12:16:39 AM

Manal,

After hearing the various presentations at the gene therapy symposium in February, I have to say that it is anything but easy. The road to where they are now has been a long difficult road. Understanding the factors involved in making this happen, just makes this latest development even more encouraging. For gene therapy to work, a sufficient quantity of stem cells must be produced in the bone marrow so that a large enough quantity of the good genes can be implanted, so that the cells with the good beta thal genes can produce enough hemoglobin to free the patient from transfusions. These new good red blood cells must also be able to continue to reproduce in sufficient amounts to maintain the hemoglobin to a range of around 10. I believe we will soon see the fruits of these long years of research and development.

Title: Re: gene therapy
Post by: Sharmin on October 12, 2009, 05:52:25 AM

Quote

These new good red blood cells must also be able to continue to reproduce in sufficient amounts to maintain the hemoglobin to a range of around 10. I believe we will soon see the fruits of these long years of research and development.

Praying for this to happen   :pray :pray :pray :pray :pray :pray :pray

Sharmin

Title: Re: gene therapy
Post by: Zaini on October 12, 2009, 06:36:17 AM

Still a long way to go,but in the end,may be it will be worth waiting for.

Title: Re: gene therapy
Post by: Sharmin on October 12, 2009, 09:42:48 PM

My hope is that the treatment will be available in the next couple of years.  What do you think the likelihood of that is?


Sharmin

Title: Re: gene therapy
Post by: Andy Battaglia on October 13, 2009, 04:14:35 AM

Sharmin,

We should add this to our list for NYC. If possible, we should see if we could talk to Dr Sadelain for a few minutes and see what advice he has as far as what will make a patient a good candidate for gene therapy.

Title: Re: gene therapy
Post by: Lena on October 13, 2009, 05:06:53 AM

Andy,

that would be really interesting. Evangelia Yannaki from Thessaloniki, Greece, will also be at the conference and give a lecture. She is the doctor responsible for gene therapy in Greece, working under Dr. Stamatoyannopoulos and remember that in Thessaloniki the first phase has been completed and in 2010 will proceed to the second and final phase.

Lena.

Title: Re: gene therapy
Post by: Manal on October 13, 2009, 05:08:58 AM

Very interesting Lena.

Has anything been published about the results of the first phase??

manal

Title: Re: gene therapy
Post by: Lena on October 13, 2009, 06:38:02 AM

Manal,

I was not aware of any publication on phase two but I did a little searching and found this "ahead of print" online publishment which concerns phase two trials in Greece:

www.liebertonline.com/doi/abs/10.1089/hum.2009.077

Dr. Evangelia Yannaki is giving a lecture in NY conference, so we shall know more about phase two.

Lena.

Title: Re: gene therapy
Post by: Manal on October 13, 2009, 07:42:32 PM

Thank you Lena for the link, hopefullly we will hear good news

manal