Thalidomide: New Expanded Access for Wasting

Thalidomide has already been available through a
compassionate-use program for treatment of certain cases of
aphthous ulcers, and some other conditions. But until now
patients could not obtain the drug through this program for
the treatment of HIV-related wasting syndrome, where
thalidomide might also be effective.

Now the FDA has authorized an expanded-access program for
AIDS-related wasting. This program, conducted by Celgene
Corporation of Warren, New Jersey, will randomize patients to
one of two doses, 50 mg or 200 mg (the most common community
dose so far has been 100 mg); this randomization will allow
researchers to determine if there is a dose response in
either safety and/or effectiveness of the treatment. Response
to therapy will be measured after four weeks, and those
failing the smaller dose can switch to the larger dose at
that time. Patients may continue the therapy indefinitely.

This program will require that physicians get local IRB
(institutional review board) approval to participate -- which
can be a serious obstacle to patients who obtain their
treatment in public hospitals and clinics. Also there will be
cost recovery, meaning that the company, which is small, can
charge for the cost of the program. The cost has not yet been
set, but we have heard that it is likely to be about $1,000
per year -- not a few thousand dollars per year as quoted in
The WALL STREET JOURNAL on August 29.

Entry Criteria

As we go to press on August 30, the protocol is not yet
entirely final. But according to Dr. Sol Barer of Celgene,
the major criteria are likely to be the following:

* Those who are qualified for the separate double-blind
controlled trial of thalidomide which is still recruiting
cannot enter this program, unless they live more than an hour
away from any trial site (by public transportation). Those
who have entered and completed that trial may enter this
program.

* Those ineligible for the controlled trial because of
opportunistic infections, malignancies, IV feeding, diarrhea
or use of other anti-wasting agents -- which includes many
people -- may enter the expanded-access program. In addition,
those who have lost more than 20 percent of body weight can
enter immediately.

* Exclusion criteria are pregnancy, serious peripheral
neuropathy, major abnormalities in blood chemistry, or if the
physician feels the treatment is not appropriate.

* Women of childbearing age can enroll, but must use
specified birth-control methods to prevent any chance of
pregnancy.

For more information about this program, call 800/896-6766,
or after Monday September 4 call 800/253-1596, during
business hours Eastern time.