Potential cures

My understanding is that we need to follow the work of the following to know what is happening in terms of a potential cure - or major lifestyle improvement for thalassemia.

Errant Gene - Thalagen

Genetix Pharmaceuticals - Current trials ongoing in Paris

Hemaquest - medication to induce hg F production

Are there any other leads that we may be interested in.  I wish the best to these three companies and hope that their trials are successful and SOON! 

Sharmin

Is it LenrtiGlobin from Genetix pharma that is currently on trial in Paris?

Also, stemcyte (cord blood stem cell bank) had earlier collaborated with various hospitals to treat many thalassemic patients using unrelated umbilical cord blood transplantation therapy; information is available in their website. Not sure about their current status though.

Genetix Pharmaceuticals - Current trials ongoing in Paris

Update on the above trial mentioned by sharmin....

I just read about the clinical trail of gene therapy on a thal patient and is so far doing good. I think there is a lot of positive direction, that we can see will lead to a cure to thalassemia

From:- http://www.cooleysanemia.com/bodies/body419.php

July 3, 2008 -In June, CAF National Executive Director Gina Cioffi had the incredible experience of witnessing history in the making when she met Paul Louis Beauchesne, the first person to successfully undergo a gene therapy trial for thalassemia.

Paul Louis is part of an ongoing gene therapy clinical trial in France led by Philippe Leboulch and supported by Genetix. The trial is taking the gene that causes thalassemia and replacing it with a working gene, with the primary goals of proving that the process is safe and that the basic premise behind the idea is sound.

All has gone well for Paul Louis. He has gone for two 60-day periods with no transfusion, which is thus far substantially longer than before transplant. The therapeutic hemoglobin continues to be made and is circulating in his red blood cells with higher hemoglobin levels in his blood than before. He will continue to be monitored to see possible long-term benefit on his disease. The researchers will repeat this process again in the fall with another patient already recruited for the trial; one patient prior to Paul Louis received back-up cells as a cautionary measure and is thus unlikely to derive clinical benefit .

“This was truly an incredible experience,” Gina says. “I felt so inspired when I visited Paul Louis and had a chance to discuss his thoughts and feelings in deciding to enter the trial. It was truly a privilege to meet him and to report back to the community this success and progress. I think it is vital that our patients, their families and our funders know that their investment in maintaining the good health and support for research is paying off. Gene therapy trials are expensive and difficult to get launched and so we are truly counting on the continued good will of our donors to help accomplish a cure through gene therapy.”

For patients who would like to reach Paul Louis, you may email him your thoughts (in French) at smoky77@hotmail.fr . Babelfish.yahoo.com, a free online translation service will aid you in your correspondence

I hope Paul comes out with flying colors and paves a way for thousands to follow

oh.. this is very very great...  60 days without  bt. is very good. and  i hope they had not hide any side effects from us. as these big company`s  alwayz does to make there profit..

i pray for paul..

This is so so so great!  This is the first good news we have received regarding these trials!  I am so glad that more than one company is working on gene therapy for thalassemia.  I hope that the side effects will be much less than those with conventional thalassemia treatment - and that it will be less toxic and risky than bmt.  It is difficult to know right now.  What are your thoughts Andy?

I also hope for the best for Paul,

Sharmin

hope to hear abt the succes of gene therapy .....

This process seems to be very similar to what Thalagen is also involved with, in conjunction with Dr Michael Sadelain. I do not know if they are utilizing the same vector for delivery of the genes, but hopefully we will be learning more as time passes. Please be aware that this will be a long process involving very few patients at first, as researchers determine if it is safe long term and also effective long term. I hope we can learn more at the Singapore conference about both company's research.

When is the Singapore conference going to take place? Where can we find its proceedings?

I hope we can learn more at the Singapore conference about both company's research.

The Singapore conference is October 8-11, 2008. Our thread about it is at
 http://www.thalassemiapatientsandfriends.com/index.php?topic=1480.msg12086#msg12086

Early registration fee prices have been extended through the middle of August.

The link wth the TIF sponsorship form is at http://www.thalassemiapatientsandfriends.com/index.php?topic=1902.msg16003#msg16003

All forms must be submitted to TIF by July 31 to be considered. Download the form, fill it out and have it signed by the president of your national or local thalassemia association, if you have one, and email or fax it to TIF before July 31.

This article is not longer present on the web.  hmmm.  I hope everything is okay.  I hope that they have started the third patient on the trial. 

Sharmin

Its realy a good news..well Andy what would be the time line to get it by all the patients......I am hoping everything will be all right.....mine as well as all the Thal patiends wishes with them....

Thanks
Ashutosh on the Behalf of my son PARIN

This article is not longer present on the web.  hmmm.  I hope everything is okay.  I hope that they have started the third patient on the trial. 

Sharmin

Sharmin,

If you are referring to cooleys anemia site, that has been down for a while for maintainance.

Has anyone heard any updates on any of this research?

Hemaquest?

Errant gene? (Thalagen)

Genetix Pharmaceuticals?  (Lentoglobin)

Has Hemaquest begun any trials with thal patients yet?

Hopefully Errant gene will begin their trials soon....


There is so much on the horizon, I hope good results start coming out soon! 

As far as Errant Gene, this was Pat G's latest myspace.com blog update. If you are on myspace, add Pat Girondi to your friends list and subscribe to his blog for regular updates.

19 years ago, Michael Sadelain began his voyage that, if successful, will change the world.

There are many people working tirelessly on board his ship: Jason, Hsuan, Norb, Chris, Isabelle, Mike, Angela.... There are investors and benefactors, Jay, Joe, Teddyjimbob, Brendan, Bill, Ron, Angela, Mark, Kosti, George…

There have been the journalists in Europe and the US who have believed.

Most of all, there are the patients and families of the patients whose prayers, sacrifice and confidence have been transformed into positive energy and will to continue.

Thanks to all of them, we are beginning to write the IND. We hope to complete it by Christmas. After 60 days of submission, we should be able to begin Clinical Trials.

We will make the vector, after we have FDA clearance. It will then take a few more months longer but we're almost home

There are no guarantees that we will safely dock but we're ever so close.

Keep the prayers and warm wishes coming. We need them more than ever.

We had nearly 1,000 paid downloads last month. Your kindness and bad taste in music leaves me without words.

Every bit of favorable wind helps.. You have all made me the luckiest (and one of the best looking) men alive.

Thanks again.

Pat Girondi

In addition, he made this announcement.

250,000 dollars donated to Orphan Disease Research from Ron Capano and Cooley's Anemia International.

For roughly 15 years I have been battling Thalassemia and Sickle Cell Anemia side by side with the Capano family of New York.

Thanks in a large part to them and the whole Cooley's Anemia International, we received a check in the amount of 250,000 dollars at the Ristorante Zuppa in Yonkers NY on September 23rd, 2008.

Thank you for your support.

-Pat g

Pat Girondi

I was hoping that Dr Sadelain would be one of the speakers at Singapore, but he is not on the schedule. When I met Dr Sadelain at Dubai, I was impressed by his confidence that they will find a cure for thalassemia with gene therapy. The main impediment is obtaining the adequate funding to get the trials moving and keep them going. This would be much easier if mankind could get their priorities straight, but a great awakening of intelligence will be needed for this to occur. When this happens, perhaps we will see a great shift in funding wars and bank bailouts to funding of projects that would actually be beneficial to mankind.


I'm waiting.