TURNING ILLNESS INTO ACTIVISM
Resourceful valley entrepreneur fights disease with advocacy
Newly diagnosed with a deadly genetic disease, Palo Alto entrepreneur Ben LeNail walked into a conference of fellow patients and was stunned by what he saw: a tableau of young boys in wheelchairs, profoundly disabled and hopeless.
In shock, he turned and walked back out. Then he stopped, and returned. And vowed to make a difference.
“‘Go back and make friends,’ I told myself. ‘It’s the same disease,’ ” he said. “I realized that we’re not so far apart.”
With limited time but enormous talent and Silicon Valley know-how,
Warrior: “With his background, he is so well-equipped to tackle the ALD world head on. He’s been a guiding light, someone I can look up to and admire, helping me stay hopeful and positive.” — Justin D’Agostino, 33, an ALD sufferer
LeNail has shifted from grief to activism for those less fortunate, mobilizing his 25 years of technology and startup experience to accelerate diagnosis, treatment and awareness of a rare and little-known neurodegenerative disease. It's called adrenoleukodystrophy, or ALD.
ALD is one in a family of genetic illnesses that destroys the myelin, or insulation, that allows brain cells to communicate with one another. Affecting 1 in 16,000 males, there are few treatment options, as dramatized in the 1992 movie “Lorenzo's Oil.”
About half of cases appear in childhood, causing normal healthy boys to quickly go blind and deaf, lose motor control, develop seizures and dementia — dying within three to five years. The only therapy is stem cell transplantation, and it's risky.
The other half of cases typically occur in young adults, ages 20 to 30s, and progress more slowly.
Then there's LeNail.
A problem to solve
Now 51, he didn't experience his first symptoms until age 38 — time enough to build an impressive resume and Rolodex. He exercises, mentors, entertains friends and directs business development at Alta Devices, a Sunnyvale company that manufactures thin flexible solar cells. He was previously a distinguished visiting scholar at Stanford University and senior director of corporate development at Sun Microsystems.
A lifelong athlete, he was puzzled when his legs began to feel leaden while jogging the leafy streets of Palo Alto. He prided himself on summiting Mount Rainier and Mount Shasta, playing squash with friends and skiing with family in Switzerland.
Increasingly, he stumbled. His gait changed.
“You think you're stressed out or tired. Then it progresses and you start catching your feet in rugs,” he said.
A long and frustrating diagnostic odyssey, which included bunion surgery and other failed remedies, ended on Christmas Eve. The news was unexpected — and devastating.
“Our family collapsed for about a year. We were just in shock,” recalled his wife Laurie Yoler, an investor, board member and strategic adviser with the robotics mobility company Zoox.
“You think: ‘What are we going to do?'” she said. “There's no good route forward.”
Then LeNail saw something he recognized: a challenge. Problem-solving was something he was good at, sick or not.
He has no training in medicine. Born and educated in France, he studied constitutional law, with an eye toward becoming a French civil servant until
visiting and falling in love with Seattle, Yoler and technology.
But he's not alone; other Silicon Valley leaders are reaching beyond tech to seek cures. Sergey Brin, the billionaire co-founder of Google, has contributed money and his DNA to a large study to reveal the genetic underpinnings of Parkinson's, a disease for which he has a hereditary predisposition.
Kristen and Matt Wilsey, a Stanford-educated tech entrepreneur and member of San Francisco's prominent Wilsey family, are funding research into their daughter Grace's rare genetic disease. The family of Ray Dolby, the sound engineer and founder of Dolby Labs, donated toward Alzheimer's research after their father's fatal diagnosis.
Innovations expand their reach. Connected through social media, patient communities represent a new paradigm for conducting medical research, linking sufferers and scientists to make an impact. And gene
sequencing, once too expensive for common use, is now readily available, helping reveal a genetic culprit of mystery diseases.
LeNail has immersed himself in medical texts, learning all he can about why it's impossible for him to metabolize certain types of fats, causing a toxic accumulation of fatty acids on nerve cells — and a cascade of neurological problems.
‘My clock is ticking’
He studies genetics and gene therapy, deepening his understanding of ALD's complex inheritance pattern. It's caused by a mutation in a gene called ABCD1, located on the X chromosome. This means that women tend to be just carriers, and have a 50 percent chance of giving it to each child. Men, who transmit it to all of their daughters but none of their sons, are always affected.
LeNail sequenced parts of his genome, and discovered the likely secret to his slow progression: some ”resistance” genes. “Ben's been dealt some good cards,” said Yoler.
There are often roadblocks in diagnosis, LeNail has found, due to doctors' unfamiliarity and often nonspecific symptoms. The research community has been small and fragmented. And patients' families often suffer alone, lacking a way to find each other.
Early progress was made through serendipity and old-fashioned networking, such as coffee chats and friendly references. He found an expert in France, who referred him to a physician in Boston.
Even as his body weakens, LeNail is building momentum.
Seeking West Coast expertise, he detected interest from a rising star at Stanford's Lucile Packard Children's Hospital: Dr. Keith Van Haren, a young assistant professor of neurology. He took Van Haren out to coffee, described the power of partnerships, then outlined the potential of a foundation that could help support Van Haren's research.
“The unique thing about having Ben on your side is you have someone who understands the disease in many ways better than specialists do,” said Van Haren. “He brings with him a whole host of insights, in terms of what matters to patients, what things might be relevant for clinical trials and a lot of wisdom about how to execute these things.
“And then you have the really important component: A deep sense of urgency,” Van Haren said. “It is ‘My clock is ticking' — not ‘Wouldn't it be neat someday?' but ‘Let's get this done now.' “
LeNail helped organize eight patient advocacy groups, then partnered them with seven academic centers across the U.S. to form a consortium called ALD Connect to improve care — and ultimately eradicate — the disease.
“With his background, he is so well-equipped to tackle the ALD world head on,” said Justin D'Agostino, a 33-year-old from Phoenix who is battling the disease. “He's been a guiding light, someone I can look up to and admire, helping me stay hopeful and positive.”
LeNail successfully lobbied for legislative change, getting X-ALD added to newborns' blood screening panel. He produced two videos, one educational and the other showing exercises. He speaks at conferences, such as Stanford's Medicine X.
Now he's visiting new biotech companies, assuring them that there's a well-organized community of patients ready to share valuable clinical information.
New success
There was promising news last month in a gene therapy trial by Bluebird Bio, stabilizing the disease in 15 of 17 young patients during more than two years of follow-up.
The arsenal of therapeutics must expand, he said. “You literally have hundreds of small biotechs that are looking for an illness to cure,” he said. “They're hungry to try something.”
But one of the biggest transformations has been personal.
Before his diagnosis, “he'd go into this room and close the door of his office and read,” said Yoler. “That was who he was on the weekends — an introverted guy. Now our house is full all the time.”
LeNail said he's had to let go of the life he planned to succeed in this new life.
“I'm not the athletic guy any more. But I can't be an ambassador for a disease if I'm just going to cling to that loss,” he said.
“I can take this horrible diagnosis and turn it into something much bigger than me,” he said, “creating something much more powerful.”