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Giving Thalassemia Patients a Choice
Mario Rossi* is a 27-year old computer
consultant who lives in Torino, Italy. His job forces Mario to travel
often and he recently moved out of the family home, into an apartment
of his own.
That independence was hard-won. Because
Mario suffers from beta-thalassemia, an inherited genetic defect
that damages red blood cells in his body, he needs a blood transfusion
every month to survive. Yet repeated transfusions have a serious
side-effect – a potentially fatal buildup of iron in the body. To
control that iron overload, Mario took a Novartis drug called Desferal
for most of his life.
Launched more than 40 years ago Desferal
remains the gold standard of iron chelation – removing excess iron
and extending the lives of tens of thousands of thalassemia patients
from Torino to Tehran. The treatment is cumbersome, however, and
many patients aren’t able to adhere to the lifelong regimen of painful
infusions via a portable pump for up to 12 hours a day, five to
seven days per week.
Novartis scientists have spent decades
and tens of millions of dollars to develop a more convenient alternative.
Success finally seems to be in sight. A new iron chelator which can
be taken as a dispersible tablet has reached the final, pivotal phase
of clinical testing. If ongoing trials are successful for the new
medicine, still known only by its research number ICL670, regulatory
applications could be submitted as early as next year, followed by
launches in major markets during 2006.
Mario has been taking ICL670 since September
2001, when he joined an early clinical study being conducted at the
Thalassemia Center at Ospedale Regina Margherita in Torino.
“It’s completely changed my life,” he says.
The daily drill of Desferal therapy
made it impossible for Mario to travel as he does today – or to
even consider moving from home where his parents played a crucial
role in his care.
“It’s very difficult to sleep with an infusion
line attached
* Not the patient’s real name. Use of a pseudonym
is required under Italian patient-privacy laws.
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to a Desferal pump,” he says. And there
were frustrating occasions when he’d wake and realize that after
mixing the Desferal solution and finishing elaborate preparations
with the infusion line the preceding evening, he’d forgotten to
switch on the pump, losing a treatment cycle.
“If I had to go back to Desferal,
I’m not sure I could manage,” Mario says, with a grimace.
A Distant Dream
Dr. Antonio Piga, Professor of Medicine at
the Department of Pediatric Hematology, University of Torino, has
watched Mario and hundreds of other patients struggle with Desferal
treatment. Part of the problem is that the benefits of therapy don’t
show immediately. Damage from iron overload to organs such as the
liver or heart takes up to 15 years to become apparent – but by
the time symptoms appear, the consequences are virtually impossible
to reverse. To address the crucial, mental dimension of treatment
compliance, Dr. Piga added a psychologist to his staff a few years
ago.
In all, Dr. Piga has 45 patients currently
participating in ICL670 trials. “Every patient and family who has
experience with Desferal dreams of an oral iron chelator,”
he says. Yet joining a trial wasn’t an easy decision since most
of Dr. Piga’s patients were well-controlled on Desferal.
Mario, for example, pondered for three weeks before finally deciding
to switch to ICL670.
Until recently, it looked like patients might
never have a choice. Desferal was derived from a natural
substance originally discovered in an iron-eating bacterium called
Streptomyces pilosus. But the hunt for a replacement floundered
as scientists repeatedly encountered obstacles in their attempts
to develop a safe and effective oral iron chelator – or even a more
convenient version of Desferal.
Then, in the mid-1990s, as most major pharmaceutical
companies continued to ignore the iron-chelation field, Novartis researchers
made one final push. Applying |
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