UPDATE ON CLINICAL TRIALS FOR GENETIC TREATMENTS FOR THALASSEMIA AND SICKLE CELL ANEMIACurrent mood: focusedCategory: MySpace19 years ago, Michel 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 homeThere 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
Pat Girondi's latest myspace update on the progress towards starting gene therapy on human subjects to cure thalassemia. It's getting closer and every cd purchased and every download of his music helps to fund this work.
Gene Therapy NewsAs we speak, vector is being made for the first patients. We believe that the vector will be finished and tested by October-November. At that time, the treating doctors will make the decision on when to commence. They may decide to wait until after Christmas.Remember that the first phase is about 'doing no harm'.The second phase is about 'efficacy', does it work?The third phase is about dosaging.We are hopefully confident that we will see efficacy already within 40 days of dosing the first patient.We would like to thank the Thalassemia Foundation and Angela Iacono of Italy. We would like to thank the Cooley's Anemia International and Ron Capano. Both of these groups have helped incredibly to keep things moving forward.I apologize that I have not written an update recently. Things are moving.A special thanks also to Sam Salman, Charles Columbus, Omar Halaby and Christina Slater... They have joined the team 4 months ago and have quickly become crucial to the project.It is July, this year I will do no concerts in Italy. I have no performances scheduled in the US. I am instead scrounging for funds. As you know, we need to stay funded.Anyway... Maybe next summer I will be able to do some concerts in Bangladesh or India or Iran or Pakistan or France or anywhere that you, my family is.Please send your warmest wishes and prayeres... Downloads help and anyone who can afford to donate... we're here.Pat Girondi