Sept. 15--It remains unclear what if any progress San Rafael-based BioMarin
Pharmaceutical has made toward helping a 45-year-old Texas woman with advanced
ovarian cancer gain access to an experimental drug treatment it is developing.
BioMarin spokeswoman Debra Charlesworth said Sept. 6 the company was working
with Congressman Jared Huffman to get the woman into a clinical trial. On
Friday, Charlesworth said, "There has been discussion. I don't know much more
beyond that. There is still ongoing dialogue."
Charlesworth said BioMarin would be providing the drug, but the trial would be
run by another organization, which she declined to name. She said the other
organization has not yet secured approval from the U.S. Food and Drug
Administration to conduct the trial.
Paul Arden, a spokesman for Huffman, said, "Congressman Huffman is working to
get both medical teams together to explore all options."
Time is short for Andrea Sloan, who was diagnosed with stage three ovarian
cancer seven years ago. Her body can no longer tolerate the toxic side effects
of conventional cancer treatments, and her doctors have advised that her best
hope is to begin taking BioMarin's new drug -- poly ADP-ribose polymerase (PARP)
inhibitor BMN 673 -- as soon as possible. The drug has produced promising
results in stage-one trials.
Sloan has requested access to the drug under the FDA's "compassionate use"
policy. BioMarin, however, has rejected her plea, even though -- according to
Sloan -- the FDA has granted its approval. The compassionate use policy permits
the use of unapproved drugs outside of clinical trials under special
circumstances, but it requires the approval of the owner of the drug.
"BioMarin has not told me anything," Sloan said Thursday. "The deal with the
clinical trials is a red herring, and they know it."
Sloan, executive director of the Texas Advocacy Project, an organization that
provides free legal services for low-income victims of domestic violence and
sexual assault, said it is highly unlikely that any researcher would add her to
a trial under these circumstances.
"That is not the way the clinical trial process works. It could skew the
results," she said.
In addition, Sloan said she would not be a good trial candidate because her
tumor is located in an area that makes it impossible to biopsy.
Sloan said since the FDA has not even approved the trial, it could easily be
another three months before it starts.
"I don't know if I'll be here in three months," she said.
Meanwhile, supporters of Sloan have mounted an aggressive social media campaign
to get BioMarin to change its mind. Her story was aired on CBS News on Monday,
and by Friday a petition drive on Change.org had collected more than 119,000
signatures, including those of former speaker of the House Newt Gingrich, singer
Wynonna Judd, basketball star Squeaky Johnson and actresses Constance Zimmer,
Ally Sheedy and Kathy Bates.
Contact Richard Halstead via e-mail at email@example.com
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