[Pharm-policy] Canavan article: Gen Patenting run amok?

[James Love]

I don't think I gave the right URL last time:

http://www.bioresearchonline.com/read/sp20001124/368114

Gene patenting run amok?
11/21/2000

Lawsuit alleges patent hinders research

In the first case of its kind, parents of children with a genetic
disorder are suing researchers who developed and patented a test for the
disease. The case involves Canavan disease, a rare disorder that
afflicts children of Ashkenazi Jewish families. Using material donated
by the families of Canavan patients, Reuben Matalon and co-workers at
Miami Children's Hospital developed a genetic test for the disease. The
suit, filed pro bono by Chicago-Kent College of Law in behalf of the
families, alleges that the researchers are trying to profit from the
children's illness, and are hindering access to the test, as well as
future research in this area.

Canavan disease is an autosomal recessive disorder caused by defective
or absent aspartoacylase, which is required for normal brain development
and function. Children afflicted with this disease accumulate
N-acetylaspartate in their brains, leading to destruction of myelin
sheath. The first symptoms appear between 3 and 6 months of age, and
impair walking, talking, and eating. As the disease progresses, it
causes seizures, severe feeding problems, retardation, and blindness,
leading to eventual death between ages 10 and 15.

The search for the gene began in 1981, when a Chicago couple, who had
two children with the disease, convinced Matalon, then at the University
of Illinois at Chicago, to develop a prenatal test, and provided tissue
samples from their two dead children for the project. Using Tay-Sachs as
a model, a disease also afflicting the Jewish community that has been
eradicated due to a widely available and reliable test, the couple
anticipated that a similar test would be developed for Canavan.

However, to their dismay, once the test was developed and the American
College of Obstetricians and Gynecologist recommended that all
Ashkenazai Jewish women undergo testing, Matalon and the Miami
Children's Hospital moved to enforce its patent. They first charged
$25/test, (subsequently it was halved to $12.50), and while seemingly a
small amount of money, the cost for testing for all the mutations can
add up to a considerable sum.

The patent holders also attempted to restrict the number of laboratories
and the number of tests performed. This was done, according to an
account in the Chicago Tribune, so that the hospital could issue an
exclusive license for the test, and facilitate retrieving its royalties.

The suit does not directly challenge the patent, but alleges that the
researchers secretly obtained it using the genetic information and
financial resources that had been donated for the public good and began
charging royalties and limiting the availability of testing.

The families, along with the Canavan Foundation and the National
Tay-Sachs and Allied Disease Association, seek to block Miami Children's
Hospital's commercial use of the Canavan gene and recover damages of
more than $75,000 derived from royalties collected for the gene test.
The Canavan Foundation was forced to stop offering free genetic
screening, according to the complaint, after being advised that it would
have to pay royalties and comply with other licensing terms.

"This case is the ultimate nightmare of how a gene patent can be used
against the very families who made possible the discovery of the gene,"
Judith Tsipis, a professor of biology at Brandeis University and vice
president with the National Tay-Sachs and Allied Diseases Association
Inc., told the Tribune.

Edited by Laura DeFrancesco Managing Editor, Bioresearch Online
ldefrancesco@bioresearchonline.com