Stem cell
transplantation in DMD - first report in China
This is the
translation from the article in chinese about the first case of therapy in DMD
with blood cord stem cell. This news was post in PPMD and send an e-mail to Dr.
Zhang to get more informations about the therapy.
The human stem cell treatment new breakthrough - my courtyard different gene
umbilical cord blood stem cell transplant treats DMD to yield the unprecedented
result
Recently, my courtyard department of neurology Director Professor Zhang Cheng
and Zhongshan two courtyards pediatrics Huang Shaoliang Professor, Professor
Fang Jianpei, Guangzhou blood center Professor Xiao Lulu cooperated, was
contracts the vacation large myo- malnutrition sickness (DMD) the patient to
carry on the different gene navel blood stem cell transplant treatment. After
the patient technique smoothly passed for more than two months, rushed the
chemotherapy pretreatment, the navel blood stem cell transplant, the granular
cell/blood platelet extremely had lacked with the acute repel response pass, is
sick the DMD gene to restore normally. This is the humanity the newest
unprecedented achievement which obtains with the navel blood stem cell
transplant treatment vacation large myo- malnutrition sickness, also is the
humanity carries on the gene diagnosis to treat the disease an new
breakthrough.
DMDpatient30years old on death
According to Professor Zhang Cheng introduced that, the vacation large myo-
malnutrition sickness (Duchenne Muscular Dustrophy, DMD) is a X chromosome
chain-like recessive hereditary disease, take the carrying on four limbs near
end skeletal muscle withers incapably, the calf ëč intestines myo-
ĽŮĐÔ is large as a characteristic, simultaneously implicates the
cardic muscle and the breath muscle. Trouble 3 ~ 4 year old of sickness, about
6 years old appears walks the difficulty, after 12 years old loses walks the
ability, the condition irreversible carrying on worsening, about the usual 20
years old, 30 years old died by the reason respiratory cycle failure. The DMD
disease incidence rate for the live birth baby boy 1/3,500, for the most common
hereditary nerve muscle disease, after its cause of disease is located X
chromosome short arm 2 areas 1 belt (Xp21) the dystrophy gene mutation causes
the anti- ĽˇÎ®Ëő protein lacking, causes the muscle fiber
gradually denaturation, the necrosis. At present in world still not effective
method of treatment.
This example sickness child small tiger (alias), male, 12 years old, come from
the Guangdong continually state, 4 years old appear Louis to throw down, when
nearly 9 years old on the staircase is difficult, gets down squats cannot stand
up, about 10 years old cannot walk, since continuously all has accepted the
treatment in my courtyard.
More than 20years research,more than 3years of animal experimentations end
bringsthe dawnfor the DMD patient
My courtyard department of neurology is the national key discipline, clinical
and the research aspect is at the domestic leading position in nerve hereditary
disease in particular DMD, always is national nerve genetics group's group
leader the unit, separately holds the post by Professor Liu Zhuolin and
Professor Zhang Cheng. They lead the research team had more than 20 years to
the DMD diagnosis and the treatment research, from the clinical epidemiology
investigation, the clinical characteristic analysis, the biochemistry diagnoses
to the gene diagnosis, ĐŻ´řŐß picks out and the pre-natal
diagnosis and the pathogenesis research all walks in the domestic colleague's
front row, in on international has the certain influence, once many times made
the academic report in the international conference, and obtained the medical
department, the State Educational Committee, the provincial science committee
many times two, the third prize. Recently through more than three years of
animal experimentations, they finally has obtained the unprecedented discovery
that, The mouse marrow stem cell transplant may obviously improve the DMD model
mouse's movement function and the biochemistry target, this treated DMD for the
stem cell transplant to provide the powerful experimental basis, has brought
the dawn for the DMD patient's gene treatment.
Sickness childDMDis sick the gene to restore normally
This example sickness child father knew my courtyard department of neurology
has made the unprecedented progress after the DMD stem cell treatment aspect,
strongly requests the hospital to give the attempt treatment, and wrote down
"the life and death shape" to express this desire and the
determination. Thereupon, Professor Zhang Cheng and Zhongshan two courtyards
departments of pediatrics Huang Shaoliang Professor, Professor Fang Jianpei,
Guangzhou blood center Professor Xiao Lulu carried on the different gene navel
blood stem cell transplant to July 26, 2004 in the Zhongshan two courtyards
pediatrics blood ward, the process has been smooth. After the trouble condition
is stable, returned my courtyard department of neurology on September 20 to
continue to accept the treatment. The trouble smoothly rushed the chemotherapy
pretreatment, the navel blood stem cell transplant, the granular cell/blood
platelet extremely had lacked with the acute repel response pass. At present
trouble mental condition good, the blood picture basic is normal; The stem cell
implants the examination proof completely to implant for the donor stem cell;
Before the transplant the trouble DMD gene 19th apparent child flaw,
transplants latter 45 day, 73 days two time examines the DMD gene to restore
normally. Professor Zhang said that, this explained already initially obtained
clinical on to sickness child DMD stem cell treatment the success, this was the
humanity carries on the newest unprecedented achievement with the navel blood
stem cell transplant which the gene treatment vacation large myo- malnutrition
sickness obtained, also was the humanity carries on the gene diagnosis to treat
the disease an new breakthrough.
Treats the disease for the Chinese gene to make the new contribution
The expert pointed out that, this example different gene navel blood stem cell
transplant treats DMD to obtain clinical on the success, is to this kind of
lethal disease clinical treatment unprecedented progress, its significance
certainly not merely limits to should get sick itself, it may radiate to other
diseases, establishes the stem cell transplant treatment hereditary disease and
other nervous system diseases new technical platforms, and so on other nervous
system diseases treatments provides the model for the parkinson sickness,
causes the stem cell transplant treatment the application obtains the
development in the depth and the breadth. Lies in after the international on
significance can completely change our country gene treatment tags along after
the western developed country the image, enhances our country for the whole in
the medical service technical level display good influence.
The expert stressed that, the different gene umbilical cord blood stem cell
transplant to the DMD patient's treatment, is animal experimentation in the
successful foundation in, to the vacation large myo- malnutrition sickness
foundation, the exploration treatment, has the possibility to the present world
in still the not effective method of treatment, 30 year old of before dies of
the breath failure lethal nerve muscle hereditary disease ----- vacation large
myo- malnutrition sickness establishment one effective method of treatment,
treats the disease for the human gene to make the unprecedented contribution.
26/10/04: Stem cell transplantation in
DMD - first report in China
This is the answer for Dr. Cheng Zhang about
this first procedure in the world :
"Dear Dr. David Feder,
Thank you for your email. Today is 92 days that the
DMD boy is transplanted by umbilical cord stem cells. He is twelve years old
and he cannot walk for almost three years. The umbilical cord stem cells was
transfer into his vein 92 days ago. The muscle biopsy was done after 76 days of
the transplantation. We find that the dystrophin staining is weak positive in
some muscle cells. We are conforming that the weak positive muscle cells come
from the donor. The DMD boy has a little improvement in his locomotive
function, for example, he touch his nose a little better than before. Now, the
DMD boy is OK, he like watch TV, and sometimes play cards with nurse. We are
still waiting and observing what happen on him.
Sincerely yours,
Cheng Zhang, MD, PhD - Professor and Chair,
Department of Neurology
First Affiliated Hospital - Sun Yat-sen
University - Guangzhou, Guangdong, 510080
P. R. China"
28/10/04: First cord blood stem cells
transplantation in DMD - more informations
Dr. Cheng Zhang answer my questions in this
e-mail:
"Dear Dr. David
Feder,
Thank you for
your email. Yes, we used a nonrelated cord blood stem cell. Because the HLA
match is very well so the immune reaction is not obvious, but we still use some
immunossupresor drugs now. We are preparing another boys for transplantation.
Best wishes,
Cheng Zhang"