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 APP.
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 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 said.
Yet, maintaining that the therapy is a major breakthrough, Dr Tasneem 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 percent 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.
To a question regarding curative treatment for Thalassemia, she said it lies in a matched allogenic bone marrow transplant, which is being done only in a hand full of centres in the country, costing around Rs 12 to Rs 24 lacs for a single procedure.
During the study being conducted at OMI Hospital Karachi, with specific reference to Thalassemia Major, 20 patients were recruited with the mean of 10 years in accordance with the study protocol and following procurement of informed consent of theirs and/or their respective parents.
"Regular planned clinical and laboratory follow up is being carried out and will continue for a period of two years.
Meanwhile, in a press briefing held at OMI Hospital on Tuesday (July 13) Dr Ilham Abuljadayel told journalists that the retrodifferentiated stem cell technology was also applied in India on four selected patients of aplastic anaemia with considerable success.
In Pakistan, a 24-year-old patient suffering from diabetes type one underwent the autologous retrodifferentiated stem cell therapy only recently and was found to be synthesising her own insulin within four weeks of therapy, she said refusing to giving further details as the study is in its comparatively initial phase.
She, however, commented that Healthcare Solutions International (Pvt) Limited (HSI), a Pakistan based organisation is the first company using the TriStem Corporations pioneering validated Retrodifferentiating technology invented by her.
"Retrodifferentiated stem cells, created from a patients own blood avoid all presently debated and controversial religious, moral and ethical issues associated with embryonic stem cell research in the United States and the West.
With regard to the study conducted in OMI Hospital, Karachi on thalassemic major patients, she said infusion of the autologous retrodifferentiated stem cell has reduced their transfusion by 70 percent and the decline in the iron overload as measured by Serum Ferritin ranges between 15 percent to 50 percent without the use of any form of chelation during four to 12 week.
With all humility, the scientist said the technology invented by her is being currently applied to also treat alzhiemiers, parkinson, multiple sclerosis, motor neuron disease, congestive heart disease and others.
HSI Managing Director, Tariq Mumtaz, said the technology would be attempted to be streamlined for its application in Thalassemia Centers and Units in different parts of the country.
Responding to queries, he said application of technology underway on trial basis, in accordance to set norms and under supervision of principal investigator Dr Tasneem Ahsan is also being monitored by Quintiles, an international body that keeps vigil on medical research taking place across the globe.
Director, Pakistan Medical and Research Council Dr Huma Qureishi, and Incharge Intensive Care Unit, OMI Hospital, Dr Javed Akhter, definite that the technology would open new vistas for treatment in Pakistan, beneficial not only for local patients but also those belonging to different parts of the world.
This is in a scenario when research scientists conducting stem cell research seem to be focusing in vitro results, Tariq Mumtaz said mentioning that it will be several years before those therapies are available to the general public to treat various diseases and indications.
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