An incredible breakthrough for a young
man treated for sickle-cell disease at
the Dana-Farber Boston Children's Cancer
and Blood Disorder Center.
"If you look at
his blood smear today you you could not
tell that he has sickle cell disease."
"Working out, haven't had a transfusion in
almost six months just about and feel
great. Trying to look great trying to be great."
21-year old Manny Johnson is the first
success story from a new gene therapy
strategy for sickle cell disease.
"I started to see improvement in my skin
you know my body, the blood levels you
know and I was able to be less
restricted.
"It was the like the pinnacle
of many many years of many people's hard
work."
Dr. Erica Esrick is the
co-principal investigator on the
clinical trial which is led by Dr. David
Williams. She's also treated Manny for
several years. Scientists have known that
sickle cell patients with a high ratio
of what's called fetal hemoglobin do
better. Those are red blood cells
typically found in babies. Sickle-cell
disease only affects adult hemoglobin.
For this trial Manny's stem cells were
collected for a new type of genetic
manipulation. Researchers introduced new
genetic material into his blood hoping
to get the body to produce the fetal
hemoglobin and silence the sickle
producing adult hemoglobin.
"The day that
Manny received his cells back was a
pretty emotional day I think for the
whole team."
The team is encouraged by
what they're seeing in Manny. Up until
now the only known cure for sickle cell
disease has been a bone marrow
transplant.
"So while that works
and it's getting better and better there
are a number of patients who don't have
a good match for instance, and they can
in this case if this works, the
technology that we've developed in our
testing, then the person can be their own
donor."
As for Manny getting his health
back is one thing, but he says an even
bigger motivation for going through this
trial is his seven-year-old brother who
also has sickle cell disease.
"Just
telling him this is all for you
and everybody else just like us so that
way he wouldn't have to endure what I
endured for him, you know, make his life a
little easier as he grows up."
Researchers are sharing Manny's a story
at the American Society of Hematology
annual meeting in San Diego.
More research is needed and they're
developing a much bigger multicenter
trial based on this technology. For
Dana-Farber News, I'm Victoria Warren.
