Thalassemia Patients and Friends
Discussion Forums => Announcements => Topic started by: Manal on March 06, 2007, 11:27:02 AM
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Hi
A two day conference will be held in Cairo- Egypt on the 8th and 9th of May at the Nile Hilton organized by the Prof of Ped. Hematology Cairo University and Chairman of ETA.
One of the main speakers will be professor Roberto Gambari who is the scientific director of Laboratory for development of Pharmacological & Pharmacogenomic therapy of thalassemia, Ferrara university in Italy. He will be speaking about Plant Extract and Foetal HB.
He wrote an article in the TIF magazine (January issue) about inducing F HB by some plants extracts.
I will be getting more details about the conference and will let you all know
take care
Manal
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More details about the conference
http://www.thalass-eg.com/2007.html
MAIN TOPICS
Gene Therapy
Stem cell Transplantation
Iron overload and iron chelation
Puberty and pregnancy in Thalassemia
Transfusion medicine update
Recent advances in prevention of Thalassemia
Prenatal diagnosis
Plants Extract and Foetal Hemoglobin Production
Foetal Hemoglobin inducers in Thalassemia and sickle cell disease
Sickle cell management
INTERNATIONAL INVITED SPEAKERS
P.S Confirmed international speakers till time of printing.
Prof. Abdallah El Ghafry KSA
Prof. Adlette Inati Lebanon
Prof. Ali Taher Lebanon
Prof. George Atweh USA
Prof. Guido Lucarelli Italy
Prof. Jacques Elion France
Prof. Maria Cappellini Italy
Prof. Marina Kleanthous Cyprus
Prof. Pedro Sodani Italy
Prof. Roberto Gambari Italy
Prof. Salahedin Fattoum Tunisia
Prof. Soad K. Al Jaouni KSA
Prof. Stamatoyannopoulos USA
Prof. Vincenzo De Santos Italy
Manal
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hi manal,
if only i could get on a plane and be there. it sounds great. please let us know of any interesting discussion points to come out of this conference.
regards vic
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Hi vic :hugfriend
You are most welcomed, i wish you could come too. Actually i wish that i can meet all of you. i will be there and will update you with everything
Manal
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Hi
Yesterday, my hema ( by the way she is the president of the conference) told me that one of the speakers will talk about a new technique that is said to cure thal in the transplantation field. They take the blood of the patient and expose his blood to ''something'' that change the nature of the patients stem cells, then his own blood is then re-transplanted back again. This is not Autologus transplant.
I really don't know any details or this ''something'', but i will let you know all the details as soon as i i have them. Can't wait for this conf to start
Manal
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one of the speakers will talk about a new technique that is said to cure thal in the transplantation field. They take the blood of the patient and expose his blood to ''something'' that change the nature of the patients stem cells, then his own blood is then re-transplanted back again.
Is this by any chance retrodifferentiation which is being developed by TriStem in the UK?
http://www.tristemcorp.com/index.htm
We had a thread at our MSN site at
http://groups.msn.com/ThalassemiaPatientsandFriends/general.msnw?action=get_message&mview=0&ID_Message=3478&LastModified=4675558476061856223
Very basically, the technology works by using one's own blood cells which are then retrodifferentiated " a process during which mature, specialised cells can revert back to a more primitive, immature cell stage. These retrodifferentiated stem cells are then capable of redifferentiating into a variety of cell types. Therefore, the process itself has the potential to reconstitute an entire human tissue such as blood, neurones, muscle etc."
This has the potential to greatly reduce or even eliminate the need for blood transfusions, if it works as hoped.
Because it uses the patient's own cells, there is no problem with rejection and it eliminates any ethical issues, sometimes associated with stem cell therapy.
This is a very exciting direction in treating thal!
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Just interested to know,
is this only targetting thal majors or would it be relevant to christian as well who is sickle cell beta thal?????
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Hi Andy
What you explained is exactly what the hema has told me but she didn't mention names. As soon asi know any details i will let you know.
in the the tristem site, i read this attached study
http://www.thescientificworld.com/headeradmin/upload/2006.01.229.pdf
To my understanding this technique is not a cure but it is one of the techniques by which Hb F can be induced in the body and replace the adult HB by taking one of the white blood cells, retrodifferiniate it and then the generated stem cells will target the gamma genes to produce HbF.
So is this considered a cure?
Does this mean that a thal patient can use his own cord blood as it has ready made stem cells???????
Vic
From the article i can understand that Christian's case is applicable too.
Manal
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WOW!
Please let me know if you find out more about this treatment and what the possibility is of its use in the near future.
Sharmin
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Andy,
I have just read about the Tristem research. I am surprised to not have heard about it yet. If this technique works it would be better than transplants and gene therapy because it does not involve chemotherapy or immunosuppression. I am suprised that they are not doing more with this research, I am getting very excited about it - do you think that it is promising in the near future Andy?
Sharmin
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Thanks for the link Manal.
http://www.thescientificworld.com/headeradmin/upload/2006.01.229.pdf
In conclusion, the single-dose infusion of the autologous RSC into patients with beta-thalassemia major has resulted in a reduction in blood transfusion requirement and improved red blood cell morphology, hemoglobin content, and concentration. Furthermore, the biomarker of iron overload, serum ferritin, fell to a statistically significant level at 6 months postinfusion when compared to baseline. This novel regenerative approach towards ameliorating a genetic disorder, though most probably transitory, is profound and may enhance compliance of young thalassemic patients.
At this point I would call this a treatment and not a cure, but a safe treatment that supplies one with his or her own blood, eliminating transfusion reactions of all sorts. (I also think we should not underestimate the positive psychological effect this would have on patients who finally have their own blood running through their veins). It s very promising and I find it very exciting that this will be the subject of a presentation at the Egyptian conference. It is also very nice that the conference coincides with International Thalassemia Day. :smiley
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Hi,
From what I understand, I think it reverts the grown up Stem Cells back to pre mature stage where they produced HbF. This can be helpful to some extent till they grow back and get the nasty switch to HbA production. Remember the genes will remain the same bad ones which will fail after the HbF switch; but nevertheless it will definitely reduce the Transfusion requirement.
However I am still concerned about the quantity of Stem Cells required per session of treatment; after all we were babies when HbF was enough for us while now we need more of that. Will they extract all the stem cells from our bone marrow and convert it every-time ???
Well, I sure hope that it goes well. Anything beats the fortnightly transfusions and daily desferal even if they last 6 months or so.
Take care, Peace!
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Hi
However I am still concerned about the quantity of Stem Cells required per session of treatment; after all we were babies when HbF was enough for us while now we need more of that. Will they extract all the stem cells from our bone marrow and convert it every-time ???
I thought this happenes once and for all. I didn't know it was sessions!!!
Anyway, i think it will be good to email them to ask for details
Manal
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I agree that this procedure would be wonderful, even if it had to be done periodically - thereby reducing transfusion and chelation requirements. However, extracting marrow every 6 months (or whatever time period is required) could also be painful and complicated I would imagine. Hopefully we will all get more information about this soon.
Vic, I also know a young man who has sickle cell, beta thal. He is a handsome 14 year old and doing quite well.
Sharmin
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I remember that I once posted about TriStem since my cousin's collegue is one of the doctors on the panel with the Saudi Doctors.
According to my cousin, this technology failed in Pakistani Trials and patients died thus the experiment was banned. :(
I hope they have more success in Egypt.
Take care, Peace!
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Hi Andy
Is this by any chance retrodifferentiation which is being developed by TriStem in the UK?
I talked to my hematologist Professor El Beshlawy (organizer of the conference) today and yes it is the retrodifferentiation and she told me that she will try to make a seesion about this with the patients ( till now this lecture is for the doctors only) so that we can ask about this technique.
So please if any one has any question about this retrodifferentiation, post it so i can (hopefully) ask in this session
Take care
Manal
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Hi Manal,
I am a bit lost, do you remember i told you about an arab doctor when i emailed you? her name was dr ILHAM ABUL-JADAEEL,what her assistant told us was a lot like retrodifferentiation.Now i am wondering that if we were wrong :doh,,but why did the govt accused her for using poor people for her experimentation :huh ,i wonder what would have been the results if we had started the treatment,, may be she was offering us a help? :sigh i don't know,, please keep us updated..i am feeling STUPID. :coffeestat
ZAINI.
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hi Zaini
You shouldn't feel that way because we should be 100% sure of what we offer to our children. So may be things had developed from the time the study was conducted in Pakistan.
Anyway i am going to attend this lecture ( by the way the speaker will be Doctor Ilham) and will update you with what ever she says and i will ask if things have changed or developed from the time the study was done in your country.
By the way: I read this study and it said that only two patients died from other complications, so whom should we suspect the study or the government??????????????????????
It i really confusing
Manal
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The government of India has also accused Dr Ilham of violating ethical guidelines by only using Indian patients in research into treating aplastic anemia. Under Indian law, parallel studies outside India must also take place. These accusations against the doctor are bureaucratic in nature and have nothing to do with the actual research. I suspect the same thing has happened in Pakistan. I don't think it's fair to say that the doctor is experimenting on the poor, when it is the poor who are most desperately seeking treatment and therefore are most willing to try a new technique, and there is also such a high percentage of poor in Pakistan. Our group has regularly seen desperate pleas for help from people in Pakistan and many have even turned to fake healers out of desperation. Thals in general have continuously volunteered for studies on new medications and techniques and those studying this technology will also find no shortage of volunteers. It should be noted that patients who died during the trials died as a result of complications from their conditions and not from the treatment.
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Hi Andy
I am really confused, could this mean that medical studies could be forged and report incorrect findings to give credits for a treatment. Who should we trust????????/
Manal
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Hi Manal,
I don't think this shows anything negative about the trials of this technology. I think it shows more about the state of bureaucracy in many countries. These laws may have the noble goal of protecting that country's patients from being used as guinea pigs, but it has the unfortunate result of prohibiting research that isn't paralleled elsewhere. There are factors that help decide where trials take place and the cost of doing research in places like India and Pakistan is much less than what the same trial would cost in the west and may be a big factor in the choice of locations.
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Hi Manal,
Thanx for your understanding,really i didn't knew anything about Dr Ilham at that time , and when that accusations came, i had no resources to search whether they were right or wrong,nd her assistant, with whom we met,was not a medical doctor, but a retired banker,he told us himself, i wonder still sometimes that why didn't she hire a medical doctor, anyways she might be a very great researcher and every thing but we didn't know at that time,and like you said we should be 100% sure of what we offer to our children,Andy what ever you said makes a lot of sense, but please do tell me that how do u know that these accusations made by Indian and Pakistani govts were surely of bureaucratic nature, i know health condition here are really bad and everything,i won't deny whatever u said, only i want to be a bit more sure.
thanx
ZAINI.
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Just thought you might wanna read this, :)
KARACHI, July 14 : Pakistani doctors have emerged to be the first to successfully apply Autologous Stem Cell Therapy in treating Thalassemia Major, a debilitating blood disorder that affects hundreds of thousands of people and kills several thousand children before they reach the age of ten.
B-thalassemia is a hereditary disease that impairs haemoglobin synthesis after birth leading to anaemia and dependency on blood transfusions – while repeated blood transfusion causes iron overload that eventually contributes to multi-organ failure.
The above-mentioned therapy, based on research undertaken by Muslim lady scientist of Saudi origin Dr Ilham Abuljadayel, comprises a procedure under which stem cells could be derived from patients’ own blood in very large volumes in a short period of time.
This can be done without any invasive surgery, growth factors, need for HLA matched donor or use of expensive and potentially dangerous immunosuppression,” the principal investigator of the study Dr Tasneem Ahsan, told the agency.
During the study initiated in February 2005 retrodifferential stem cells derived from the blood of 20 recruited patients with B- thalassemia major were infused back into them, after taking all due precautions.
“There is not only a statistically significant drop in blood transfusion requirement but no adverse effect have been seen in any of the patients as a result of the procedure, thus establishing its safety, Dr Tasneem Ahsan said.
She further mentioned that since the infusion of stem cells none of the patients have received any chelation therapy and despite this their serum ferritin, which is a measure of iron overload, dropped significantly in almost all patients.
“Fewer Transfusions and less iron load will lead to better growth and less organ damage thereby increasing the life span and improving the quality of life of patients,” she said maintaining that duration of beneficial effects will become more evident as the study progresses.
The very technique called “Autologous Retrodifferentiated Stem Cell Therapy”, an outcome of research, spread over a span of some 15 years, undertaken by Dr Ilham Abuljadayel has not only been successfully applied on Pakistani thalassemic patients but is also in process of being tried, again in Pakistan, for conditions as Diabetes Type One, Spinal Cord Injury, Muscular Dystrophy, etc.
Meanwhile, answering another question she acknowledged that the stem cells being used in the patients are derived from the patient’s own blood hence they do carry the original gene defect of B-thalassemia.
These stem cells would therefore be expected to be less robust than a normal stem cell and would have a limitation to their life span.
“This does raise the possibility of repeating the autologous retrodifferential stem therapy for continuing benefit in B-thalassemia patients,” Dr Tasneem Ahsan.
Yet, maintaining that the therapy is a major breakthrough, Dr. Tasneem Ahsan reminded that there has been a significantly high incidence of the very ailment in Pakistan, with an estimated 100,000 suffering from the condition.
“70 per cent of the children with B-thalassemia are estimated to die before the age of 10 years in our country, she reminded mentioning that given the prevalent cultural attitudes regarding blood transfusions, the life of a family with one or more thalassemic children is one of utter misery.
It may again not be out of context to remind that to reduce, blood-transfusion-induced iron overload the sufferers require regular chelation therapy which is expensive and most of the local patients are either not receiving any chelation or getting sub optimal chelation in addition to sub-optimal transfusion.
The suffering of the affected individual is compounded by the financial burden of the disease as well as the non-availability of regular blood supply and the prohibitive expense of regular chelation.
ZAINI.
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Countries with large poor populations have been passing laws to try to prevent testing of unproven drugs and treatments on their people, because often those people have been used as disposable by the drug companies (this was the subject of the excellent movie, The Constant Gardener). As a result, laws such as the one in India, requiring parallel testing in other countries, have been passed. The trials that Dr Ilham ran on aplastic anemia patients in India did not have a parallel trial elsewhere, thusly violating the Indian law. The one patient who died during this trial died from complications of aplastic anemia, which is a terrible disorder that is often fatal. The outcome of the trials is not what is the subject of the Indian concerns. It is that no parallel trial took place.
I think the fact that this technology is being presented at the upcoming conference in Egypt gives it even more credibility. Hopefully some new information about trial results will be presented there.
I don't think it can be easy for a parent or patient to agree to take part in a trial for some new drug or treatment without having a good idea of the risks involved. Caution is a good choice until more is known.
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Hi Again,
Just doing a net search my self :),gathering information,tell me what do you guys think ???
For bold investors, their claimed scientific coup, made under the auspices of a company called TriStem, could earn prodigious fortunes for themselves and their backers. But if they are wrong, they will not only lose their shirts: they will have exposed what many scientists believe is fundamentally at fault in the epoch-making scientific field known as stem-cell research.
Stem cells are primitive living cells that can be artificially coaxed to grow into any type of specific cell or tissue in the body. Scientists are confident that stem cells will one day deliver cures for virtually any kind of organ, blood or tissue dysfunction. Stem cells are tantamount to medicine's philosopher's stone – the mythical alchemical process that turned dross to gold – offering astounding new prospects for people afflicted by a huge range of debilitating and life-threatening illnesses – including cancer, heart and brain diseases, spinal-cord injury and diabetes.
Stem-cell therapy promises stunning cuts in hospital and medical-care budgets. Expensive drug courses, postoperative and long-term care of the aged could become a thing of the past. Each individual would have a set of stem cells in storage matching their DNA. Treatment for a wide range of illnesses would involve injections of appropriately enhanced cells to replace damaged tissue or defective blood, or absent bone, cartilage and muscle, or degenerating brain and nerve cells.
But first, scientists must settle on the best way to acquire appropriate stem cells – and they must learn sure ways of differentiating them into the required tissue and cell types for therapy. Stem cells exist at different levels of purity and potency, ranging from pluripotent to multipotent. Pluripotent cells can in theory be turned into every and any kind of cell and tissue type, while multipotent cells are capable of being turned into certain restricted kinds of cells.
And this is where our Hampstead-based husband-and-wife team come in. Ilham Abuljadayel, a postdoctoral biotech scientist originally from Saudi Arabia, claims that she has discovered an infallible and cheap technique for changing white blood cells into perfect, pluripotent stem cells – the gold standard of stem-cell values. Her husband, Ghazi Dhoot, a physicist turned merchant banker, originally from north India, has patented the discovery and seeks to develop it to the point where TriStem (named in honour of their three children) can go public.
Abuljadayel claims that she can take half a pint of your blood and within three hours produce a large quantity of pure stem cells peculiar to your personal DNA. "What we're doing," she tells me, "is taking cells and turning their clocks back to make them young primitive cells, capable of becoming any cell or tissue type in your body." She calls the process "retrodifferentiation". At the same time, and this is the crucial point, she claims that she has already found ways of reprogramming these stem cells into a variety of specialised cells and tissues that could mend a damaged heart, treat Parkinson's disease and spinal-cord injury, and cure diabetes. And that's just the beginning.
So why isn't the world hailing her discovery and seeking to adopt it? The answer is in the fraught connection between stem cells, the complex development of the human organism from the embryo, and the pace at which establishment science moves when faced with new discoveries.
You can read the complete article on,
http://www.timesonline.co.uk/tol/life_and_style/article1002124.ece?token=null&offset=0 (http://www.timesonline.co.uk/tol/life_and_style/article1002124.ece?token=null&offset=0)
ZAINI.
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Hi
This is the detailed program for the conference:
International Thalassemia Day
9 – 10 May 2007
Program of the Conference
1st Day 9/5/2007
09.00 – 10.00 Opening Ceremony.
Chairpersons:
Prof. Amal EL Beshlawy(organizer of the conference)
Prof. Aly. Taher
10.00 – 10.45
Plenary 1
Chairpersons:
Prof. A.S. Khalifa
Prof. M. Nasser
Prof. L. Ragab
• Infusion of Autologous Retrodifferentiated stem cells in patients with Beta Thalassemia (I. Saleh, Pakistan)
10.45 – 12.15
New Era in Stem Cell Transplantation in Thalassemia and SCD
Chairpersons
Prof. H. Kamel
Prof. Y. El Tombari
Prof. A. Kamel
Prof. E. Refky
Prof. A. Hadad
• 10.45 – 11.10 Haplo-identical mother to child transplantation in Thalassemia: Immunological Reconstitution (A. Isgro, Italy)
• 11.10 – 11.35 BMT in SCD (M. Marziali, Italy)
• 11.35 – 11.55 Allogeneic stem cell transplantation (SCT) in patients with B-thalassemia (A. El Jefri )
• 11.55 – 12.15 Bone marrow transplantation in thalassemias (T.Gafaar, Egypt)
12.15 – 12.45 Coffee Break
12.45 – 02.15
Hot Topics in Thalassemia and SCD
Chairpersons
Prof. A. Taher
Prof. S. Sheble
Prof. H. Soed
• 12.45 – 01.10 State of the Art Management of transfusional Iron Overload (M. Cappellini, Italy)
• 01.10 – 01.35 Clinical experience in chelation therapy (A. Taher Lebanon)
• 01.35 – 02.00 Management of thalassemia intermedia :Experience with Hydroxy urea and Butyrates (A.El-Beshlawy Egypt).
• 02.00 – 02.15 Discussion
02.15 – 02.55
Plenary 2
Chairpersons
Prof. F. Hamouda
Prof. N. Kaddah
Prof. A. Moustafa
Prof. G. Mokhtar
• Interactions between red blood cells and the endothelium in normal vascular biology and red blood cells pathology (J. Elion, France)
02.55 – 03.15
Coffee Break
03.15 – 04.45
Updates in Management of SCD
Chairpersons
Prof. A. Inati Khoriaty
Prof. H. Hassab
Prof. M. El Tagui
Prof. H. Gazaly
• 03.15 – 03.35 Sickle Cell Disease-Current Knowledge in the Pathophysiology ,Aassessment and Management of Pain (A. Inati, Lebanon)
• 03.35 – 04.00 Cellular and Molecular Target of Hydroxyurea in Sickle Cell Anemia (J. Elion, France)
• 04.00 – 04.20I Impact of transcranial Doppler immaging in diagnosis and folllw up of CNS vasculopathy in SCD patients under HU (M. Eltagui, Egypt)
• 04.20 – 04.35 Pulmonary Hypertention in SCD: Is there any role to L-Carnitine. (A. Elbeshlawy, Egypt Organizer of the conference)
• 04.35 – 04.45 Discussion
04.45 – 05.25
Plenary 3
Chairpersons
Prof. A. Nazir
Prof. M. El Dafrawi
Prof. S. El Gawhary
• Plant Extracts and Fetal Hb Production (R. Gambari, Italy)
Lunch 05.25
2nd Day 10/5/2007
09 – 10
New Insights in blood Transfusion
Chairpersons
Prof. M. El Ekiabi
Prof. F. Mouftah
Prof. O. Abd El Hakim
• 09 – 09.25 NAT in blood Bank (S. Eissa, Egypt)
• 09.25 – 09.50 The production of transfusable universal donor red cells (F. Goubran, Egypt)
• 09.50 – 10.00 Discussion
10.00 – 10.40
Plenary 4
Chairpersons
Prof. N. Kassem
Prof. N. Fadaly
Prof. M. Khalef
• Abnormal Hbs leading to Thalassemia syndromes (H. Wajcman, France)
10.40 – 12.10
Fetal Medicine in the Management of Thalassemia and Genetic diseasesChairpersons
Prof. M. Abou El Ghaar
Prof. T. Gafaar
Prof. M. El Ansarry
Prof. I. Ramzy
• 10.40 – 11.00 Pre-implantation Genetic Diagnosis: Egyptian Experience (R. Mansour, Egypt)
• 11.00 – 11.20 Fetal Medicine in the new Millennium (M. Momtaz,
• Egypt)
• 11.20 – 11.40 Umbilical cord blood plasticity and ex vivo expansion condition (M. Elansary, Egypt)
• 11.40 – 12.00 Prenatal diagnosis of Thalassemia in Egypt (S. El-Gawhary, Egypt)• 12.00 – 12.10 Discussion
12.10 – 12.30
Coffee Break
12.30 – 01.30
Endocrine Complications in ThalassemiaChairpersons
Prof. I. Ghaly
Prof. S. Jaouni
Prof. N. Nour
• 12.30 – 01.00 State of the art: Growth and endocrine complications in Thalassemia: The Ferrara Experience (V. De Sanctis Italy)
• 01.00 – 01.20 The Pattern of bone disease and its morbidity among Thalassemia patients treated at University Hospital in Saudi Arabia (S. Jaouni, KSA)
• 13.20 – 13.30 Discussion
13.30 – 15.00
Free Communication:
Chairpersons
Prof. N. Sabet
Prof. M. Assem
Prof. A. Gawad
Prof. A. Abou El Enien
Prof. O. Bakr
13.30- 13.45 Intermittent intravenous high dose desferrioxamine therapy in heavily iron –loaded children with B Thalassemia major. (Y.Wal Oman)
13.45-14.00 ;Neurological Disease in Beta Thalassemia (A.Tantawy Egypt)
14.00 – 14.15 :Phenotypic variability in Thalassemia intermedia (E.El-Raoef Egypt.
14.15-14.30 :Rapid screening of B-Globin Gene mutationsby real –time PCR in Egyptian thalassemic children (M.Aziz Egypt)
14.30-14.45 :Immunological evaluation of the early post transplant period in pediatric bone marrow allotransplantation. (H.Abdelrazik Egypt)
14.45-15.0:Visual function evaluation of Egyptian B-thalassemia major patients on long term desferal therapy. (M.Abou El-Ela)
13.30-15.00
Meet the expert: Patient/Doctors Meeting
Chairpersons
Prof. V. Desznctis
Prof. R. Gambarr
Prof. A. Isgro
Prof. A. Taher
Prof. O. Seleem
Prof. S El Gawhary
Prof. A. El Beshlawy
15.00-17:00
Case presentation
Chairpersons
Prof. A. El Beshlawy
Prof. A. Taher
Prof. M. Naser
Prof. E. Abdel Raoef
Prof. F. El Rashedy
Presenters:
Prof. H. Hamed
Prof. K. Salama
Cons. Dr. N. Shaheen
Prof. A. Taher
Lunch