Cancer Drug Some Help for Scleroderma
Cytoxan Offers Modest Benefit in Scleroderma Lung Disease
June 21, 2006 - The cancer drug Cytoxan
offers a small benefit to scleroderma patients, a major
clinical trial shows.
In people with scleroderma, the body's immune system attacks connective
tissues throughout the body. This leads to formation of scar tissue in the skin
and organs. Many patients die from scleroderma-related lung disease.
There's no cure for scleroderma, but there's some evidence the
immune-suppressing cancer drug Cytoxan might slow the deadly
scleroderma-related lung disease.
"Our findings indicate a significant, albeit quite modest, treatment
effect of [Cytoxan]," conclude UCLA researcher Donald P. Tashkin, MD, and
However, while Cytoxan is used to treat certain cancers, it can also cause
several different kinds of cancer and is linked to serious side effects.
Therefore, some other researchers say the drug should not be used for routine
care in scleroderma sufferers.
Does Cytoxan really work? A clinical trial at 13 U.S. medical centers tested
the drug against an inactive placebo in 158 patients for one year.
The bottom line: Cytoxan has a small benefit. Compared with those who got
the placebo, patients receiving the drug did 2.5% better on a test of lung
function. They also had somewhat fewer instances of shortness of breath and
less thickening of the skin, as well as somewhat better health-related quality
Those findings appear in the June 22 issue of The New England Journal of
Accompanying the report is an editorial by Fernando J. Martinez, MD, and W.
Joseph McCune, MD, of the University of Michigan, questioning routine use of
Cytoxan for scleroderma patients.
"The modest therapeutic response [to Cytoxan in the Tashkin study] and
the potential for significant toxic effects do not, in our opinion, support the
conclusion that one year of daily [Cytoxan] should be considered routine
therapy for all [patients with scleroderma lung disease]," they write.