The busy scientist was about to leave a conference in Washington for his native Hungary when he was buttonholed by a persistent Main Line mother worried about her son's health.
Amber Salzman told him he absolutely had to meet a researcher in France who was working on the deadly brain disease that could strike her son. He said, sure, someday.
But after boarding the plane to Hungary, he got a surprise message from the flight attendant: Salzman had already set up the meeting, and the French researcher would meet him in Budapest.
That fast-track meeting, and plenty of others arranged by Salzman and her sister Rachel, eventually led to pioneering research published today in the journal Science. The authors report that a novel gene therapy seems to stop progression of ALD, the rare disease featured in the movie Lorenzo's Oil.
The study describes results from just two patients, and the therapy is not without risks. Still, other experts said it looked promising, especially for those lacking a good match for a bone-marrow transplant, the usual treatment.
And the authors agree it would not have occurred without the Salzmans, who cold-called scientists, showed up at meetings, and urged far-flung researchers and companies to collaborate.
"They never quit, the Salzman sisters," said Nathalie Cartier, a scientist at INSERM, a public research institute in Paris.
"They are like fairies," said study leader Patrick Aubourg. "They succeed with problems that we cannot resolve."
It is not uncommon for parents of sick children to set up foundations to raise money and foster research, as the Salzmans did in creating the Stop ALD Foundation. But they had added advantages.
Amber, who has a doctorate in mathematics from Bryn Mawr College and lives in Merion, was a longtime senior executive at GlaxoSmithKline. Rachel, a veterinarian living in Florida, knew her way around a scientific paper.
What they soon learned was not encouraging.
It began in late 2000, when a third sister, Eve Lapin of Houston, learned that her son Oliver had ALD. He had been misdiagnosed as having attention deficit disorder and then Asperger's syndrome.
An MRI finally revealed the cruel truth: There were lesions on his brain. Further tests revealed ALD - adrenoleukodystrophy, which strikes one person in 18,000. The myelin that insulated the nerve cells in his brain was deteriorating, which could lead to seizures and dementia. He had just a few years to live.
Since the disease runs in families, Lapin had her younger son screened, and Amber Salzman had her son tested as well. Their blood revealed early signs of the illness.
The two mothers learned that they were carriers for the disease. They showed no symptoms, as ALD primarily strikes males, but they had passed it on to their sons. Their sister, Rachel, the veterinarian, was not a carrier, so her children would be fine.
Amber Salzman immediately started working her contacts at Glaxo. Her then-boss, Tachi Yamada, urged her to investigate gene therapy.
Just two months after the boys' diagnoses, she and Rachel met in Paris with French scientists Aubourg and Cartier.
The scientists had been studying the disease for more than a decade, and had gotten promising results from gene therapy on lab animals. But to proceed in humans, they needed someone to make a clinical-grade "vector" - a disabled virus that would deliver corrective genes to human bone marrow cells.
That's what led the Salzmans to meet Gabor Veres, the Hungarian scientist at the Washington meeting. He worked for Cell Genesys, which could make the right kind of vector, a lentivirus, from a disabled form of HIV.
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