From the moment he was born, Bodhi Bhattarai was charming and inquisitive, talking early and winning over strangers with a big smile. There was no reason for parents Déodonné and Ranjan, MBA ’10, MSHFID ’15, to suspect anything was wrong. By 9 months old, however, Bodhi had missed some milestones.
“He didn’t start to crawl or roll over,” says Déodonné. “He wasn’t cruising on the furniture.”
The couple, who live in Concord, N.H., took Bodhi to see a specialist in Boston who made the diagnosis: spinal muscular atrophy (SMA-Type II).
“It was overwhelming,” says Ranjan. “Almost in passing, the doctor said, ‘He may not ever be able to walk.’ Suddenly we had to come to grips with the fact that our son, who looked perfectly healthy, was not going to have a traditional life.”
A few weeks later, at the annual Cure SMA conference for care providers, researchers and families, Déodonné and Ranjan learned about a treatment being developed by Biogen. Bodhi’s doctors identified him as a perfect candidate for the drug’s clinical trial and the family was eager to have him enrolled.
“We took a leap of faith,” says Déodonné, a lawyer who left her job in health policy to better care for Bodhi. “We essentially agreed to use our infant as a human test subject, not knowing what the side effects could be or what the future might hold.”
In November 2015, the family began making regular 300-mile round trips to Hartford, Conn., for the injections.
Since the trial was blinded, they didn’t know whether Bodhi was receiving the real drug or a placebo. Still, over the next year, they saw his overall strength and energy levels improve. He began rolling over independently and continued to eat and breathe without assistance. He gradually gained strength in his hands, arms, abdomen and leg muscles. When the trial’s first phase ended in November 2016, their hunch was confirmed: Bodhi had been in the dosing group for the drug, which earned FDA approval in late December.
“The wave of relief has been huge,” says Déodonné. “It’s been one of the greatest sources of strength for us, knowing that Bodhi is getting the best treatment out there.”
As the search for a cure continues, the family takes things one day at a time. “It has helped us not worry so much about what his life will be like in one year or three years,” says Ranjan, a user experience architect for a cybersecurity company, whose office is just blocks away from Biogen’s Cambridge headquarters. “I see the building all the time, and it makes me feel good knowing there are people inside literally doing life-changing work.”
Back at home, Déodonné recently found 2.5-year-old Bodhi in the kitchen, opening up cupboards and pulling out pots and pans. “We had never needed to child-proof anything,” she says with a laugh. “Now he has the strength and energy to get into trouble.”
As vice president for the U.S. Rare Diseases Group at biotechnology company Biogen, Lance Colwell '92 connects people like Bodhi suffering from debilitating diseases with potentially life-saving treatments. His current focus is launching Spinraza,™ developed for SMA and one of the fastest drugs ever to be approved by the Food and Drug Administration. Read about his insight on the process.