Although their stories differed in detail, they shared a passion and a purpose no parent would envy: finding a cure for the diseases that afflict — and, in some instances, might one day kill — their children.
At the ribbon-cutting yesterday for Intellimedix, the newest member of the Cambridge biotech cluster, a group of men spoke from personal experience about parent-driven innovation in the high-stakes world of scientific research.
Motivated by his daughter's Dravet syndrome, a severe seizure disorder that doctors told him couldn't be cured, Daniel Fischer co-founded Intellimedix to accelerate the discovery of new drugs, as well as repurpose old ones, to treat neurological diseases. His daughter recently started a new drug and has been seizure-free for a month.
"My advice to parents is don't accept whatever you hear from doctors," he said. "My other message is don't do it alone. You need academia. You need big pharma."
Eleven years ago, Brad Margus learned that two of his sons had a rare, lethal genetic disease that combined a loss of muscle control with cancer and immune deficiency.
While running his company, Margus studied molecular genetics, formed a nonprofit, raised $30 million, set up a clinic at Johns Hopkins Hospital and coordinated clinical trials.
"The good news is the science is not really predictable," he said. "A lot of (the breakthroughs) are based on serendipity."
When Harvey Lodish and seven other MIT professors founded Genzyme in 1981, he never imagined that Cerezyme, a drug he helped develop, would one day be used to treat his grandson for Gaucher disease.
"You never know who's going to benefit from what you do in the lab," said Lodish, chairman of the Massachusetts Life Sciences Center's Scientific Advisory Board.
Parents, he said, play an "enormous" role in raising money to support early-stage research.
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