FOX Business Reports: David, Goliath Team Up in Battle to Cure Rare Diseases

In the world of drug development, collaboration is key.

When Kathy Giusti was diagnosed with a rare and fatal blood cancer nearly 20 years ago, there wasn’t a single drug in the pipeline to help her fight her grim prognosis, and she was expected to die within three years. That is until the then-new mother found determination, launching the Multiple Myeloma Research Foundation, which now is helping to develop 20 treatments at various stages in the pipeline.

Like Giusti, Ilan Ganot is facing a similar tragic circumstance. At just two-years old, his son was diagnosed with Duchenne muscular dystrophy, a muscular degenerative disease that usually kills those it affects by their teen or early adult years. Rather than sit and wait for his son to die, Ganot quit his job as a banker at JPMorgan Chase, devoting his life to finding a cure and starting his own firm called Solid Ventures.

The treatments for more common ailments like asthma, diabetes, and depression are well known and often very lucrative for pharmaceutical companies. But for the more rare diseases like myeloma or DMD, it’s sometimes more difficult to fast-track the development process to get treatments to market.

“It was the worst thing in my life, no question,” Ganot said of his son’s diagnosis. He allowed himself to feel hopeless, like Giusti did, for only a short time before digging in his heels to fight the deadly disease.

“I started reading more online, making phone calls to companies, charities, drug developers. And I started to network my way into understanding what’s going on, who’s doing what, interesting programs, why nothing is working, where the money is missing,” Ganot said. “I tried to figure out what other parents in my situation were doing…and it quickly became clear to me I’m not going back to work at a bank because I’m convinced I can make a big difference.”

Struggle to Survive

Ganot is up against a ticking clock. If no treatment is developed, his son will begin losing the ability to walk by around age 7. For him, it’s all about finding some way to help his son…and fast. After writing the business plan and organizing the right team of scientists and drug developers, the next step was to gather the funds needed to help spur development. For that, Ganot turned to professional investors, and managed to raise $17 million in less than one year.

“We’re looking to get our hands on the science and be active in developing the drugs ourselves. It can be in collaboration: With partners, people in the community, and the industry. But we’re going to be actively involved and lead the effort,” Ganot said.

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