Re: Bubble Babies

[Guest guest]

Morning, Everyone! I thought this article was

interesting because since I saw that bubble movie with

Travolta all those years ago, I have wondered if

they had made any advancements.

New Gene Therapy Protocol: First Successful Treatment

for " Bubble Babies "

Source: Acurian Inc.

by: Wingate, Ph.D.

07/24/2002

Children born without an immune system are often

referred to as " bubble babies " because they must be

kept in complete isolation from the viruses and

bacteria that the rest of us are exposed to every day

without harm. The condition is actually called severe

combined immunodeficiency (SCID), and is caused by the

lack of an essential enzyme, adenosine draminase

(ADA).

A team of Israeli and Italian researchers report that

a new gene therapy protocol has been successful in

completely reversing SCID in one child who is now 22

months old, and appears to be equally successful in

two younger children. Their report appears in the June

28, 2002, issue of the journal Science.

According to Shimon Slavin of Hadassah-Hebrew

University Medical Center in Jerusalem, the

significance of this new development extends beyond

those immediately affected by SCID and has the

potential to be equally effective in curing similar

genetic diseases. " The concept can be applied to all

genetic diseases where there is a need to engineer

stem cells to produce normal products, especially when

patients have no matched donor available for safe bone

marrow transplantation, " Slavin said.

The procedure improves on an existing method to give a

biological advantage to genetically altered stem

cells. Patients with SCID are usually treated with ADA

replacement therapy in an attempt to rescue the immune

system. This replacement therapy, however, is not a

long-term solution; it is only effective for one or

two days at a time and is extremely expensive. Bone

marrow transplantation, preferably from a matching

donor, is the recommended treatment to replace the

abnormal bone marrow stem cells and create a normal

immune system.

Salsabil, 22 months old, is the first child in the

world to be completely cured of ADA-SCID using

genetically altered stem cells. Salsabil is the third

bubble baby born to her family, part of Jerusalem's

Arab community. The first baby died from the

condition. Salsabil's older sister, Tasmin, survived

after receiving an allogeneic cord blood transplant

from a younger brother. After the first child died,

doctors froze umbilical cord blood cells from each

successive birth for possible treatment of subsequent

siblings.

In the early 1990s, scientists had already begun

experimenting with altering T cells and stem cells

through gene therapy. While scientifically successful,

the experiments did not have a notable impact on the

patient's condition because only a minute fraction of

the genetically abnormal bone marrow products were

repaired, Slavin said. The patients still required ADA

replacement therapy to survive because the " good "

cells were overwhelmed by the much larger number of

genetically abnormal cells remaining in the host, he

explained.

By the time Salsabil was born in February, 2000,

significant advances had been made in the field of

gene therapy and stem cell biology. Prof. Slavin

determined that if he could give the genetically

treated cells a biological advantage, they could

prevail over the abnormal cells.

After clearing all the legal issues, the

groundbreaking procedure took place when Salsabil was

seven months old. She received a mild treatment to

suppress her abnormal stem cells, providing a

biological advantage to the genetically corrected

multi-potential stem cells and their products. Then,

the researchers introduced the missing ADA gene into

her purified bone marrow stem cells. No enzyme

replacement therapy was given at any stage. So far,

the new procedure has proved fully successful.

Within a few weeks, the number of lymphocytes (while

blood cells) in Salsabil's blood had risen

dramatically and the toxic products in the blood,

which would normally kill the lymphocytes, had

declined. Within a month, the child was out of

isolation and went home in perfect condition, with no

medications.

Nearly 2 years after the gene therapy, all Salsabil's

T cells and B cells, as well as other products of CD

34 stem cells, are genetically transduced and

functioning normally, meaning that she has a properly

functioning immune system. Interestingly, after

exposure to chicken pox in the family, Salsabil's

antibodies developed spontaneously and she did not

develop any sign of the disease.

Since Salsabil's first-of-a-kind procedure was

performed, two other children have received a similar

protocol from the Italian team, apparently with a

similar success rate.

__________________________________________________

[Guest guest]

Wonderful news. I hate to see children have such a hard time of it.

Stacey in PA