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For Dan Doctoroff, funding ALS research is a family matter

Daniel Doctoroff in New York City. Presley Ann/Patrick McMullan via Getty Images

When his father was diagnosed with Lou Gehrig’s disease in 2000, Daniel L. Doctoroff thought it was just a case of horrible bad luck. But when his uncle got the same staggering diagnosis seven years later, Doctoroff realized that he and his family probably had a genetic propensity for the illness.

There are no effective treatments for the disease, formally called amyotrophic lateral sclerosis, or ALS, which killed the two brothers. So Doctoroff has opted not to take a DNA test to see if he inherited a mutation that causes a minority of cases. But he also refused to simply live under a sword of Damocles.

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“Watching someone with ALS is like watching death slowly unfolding,” said Doctoroff, 60, who served as deputy mayor of New York under Mayor Michael Bloomberg and then as CEO of Bloomberg LP. “I said, ‘I have to do something.’ ”

Daniel Doctoroff (second from right) with his father and three brothers taken in 2000, just after his father had been diagnosed with ALS and about a year after he first started showing symptoms. He died about a year and a half later. Doctoroff family

In 2013 he started Target ALS, a nonprofit research foundation trying to accelerate the development of treatments for the usually fatal degenerative neurological disorder. The former investment banker also donated $10 million of his personal fortune to the New York-based foundation.

Target ALS holds its annual meeting Tuesday through Thursday at the Charles Hotel in Cambridge. Executives from more than 90 drug companies and venture capital firms are planning to attend the gathering, where Doctoroff is expected to unveil an ambitious five-year, $100 million strategy to take on the disease.

The plan includes earmarking $30 million to identify validated biological markers that could be used to diagnose ALS early, monitor how it progresses, and gauge whether a drug works. The lack of identifiable biomarkers has been among the many challenges for researchers.

Doctoroff also plans to discuss the progress researchers have made with funding from his group. Within six months, for example, the Cambridge biotech Biogen hopes to start clinical trials of an experimental ALS drug that it bought last year for $10 million upfront from Karyopharm Therapeutics, of Newton.

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Karyopharm originally developed a similar compound as a potential cancer treatment. It was refashioned into a possible ALS therapy after the foundation introduced Karyopharm scientists to three academic researchers from Johns Hopkins University and the University of Florida who collaborated with the drug firm. The foundation contributed $900,000 to the project.

“The whole notion [of the foundation] is to lower barriers to successful collaboration among people who have an academic or commercial interest in ALS,” said Doctoroff, who’s now chief executive of Sidewalk Labs, the urban innovation division of Google’s parent company, Alphabet.

Doctoroff now serves as chief executive officer of Sidewalk Labs, the urban innovation division of Google’s parent company Alphabet.KENA BETANCUR/AFP/Getty Images/AFP/Getty Images

ALS is rare. Approximately 6,000 people in the United States are diagnosed with it each year, according to the Massachusetts chapter of the ALS Association.

The disease was first diagnosed in 1869 by the pioneering French neurologist Jean-Martin Charcot. But it wasn’t until 1939 that the diagnosis of New York Yankees first baseman Lou Gehrig brought national and international attention to the disease. In recent years, ALS has drawn more public attention because of the Ice Bucket Challenge, the fund-raising campaign inspired by the Boston College baseball captain Pete Frates, who has the disorder.

ALS affects the nerve cells in the brain and spinal cord. It gradually robs patients of the ability to speak, eat, and, finally, breathe. Since 1995, only two drugs have been approved to treat it, and neither works very well. Despite the best efforts of doctors and researchers, the disease still kills most patients in three to five years.

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Because the illness is uncommon, funding for research has been modest. In fiscal year 2018, the National Institutes of Health, the single largest source of research financing, spent $83 million on ALS research. As a comparison, research into diabetes, which is common, received more than $1 billion in NIH funding that year.

Target ALS was created to fund promising projects, but the nonprofit insists that multiple researchers, often some in industry and others in academia, jointly apply for funding, said the foundation’s chief executive, Manish Raisinghani.

Target ALS sometimes acts as a matchmaker, bringing together scientists with complementary approaches who might not know about each other.

“By requiring that different constituencies come together and submit proposals, we are breaking down those silos,” said Raisinghani, who has a PhD in pharmacology.

Proposals are judged by a Target ALS independent review committee of 20 members, approximately half of whom are academics and half of whom work for drug companies. To remove potential conflicts of interest, committee members can’t apply for foundation funding.

In another move intended to galvanize research, Target ALS relinquishes any rights to whatever intellectual property comes out of a project that it funds; they belong strictly to the scientists who obtained the funding.

Christopher Henderson, a vice president of neurology at Biogen and chief adviser to Target ALS, says foundation funding for projects like the experimental drug the company bought from Karyopharm was important. But so was Target ALS’s role as a matchmaker between Biogen and the three academic researchers.

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“The money is essential,” Henderson said. “But the real decisive difference is getting the right players in the room who otherwise wouldn’t have met and can do completely new things together.”

Doctoroff, the founder of Target ALS, says the approach has catalyzed interest in ALS research. The foundation’s first annual meeting, in 2013, drew executives from seven biotechs, pharmaceutical companies, and venture capital firms. This year’s meeting is expected to attract 94.

In New York, Doctoroff is best-known for his relentless (and ultimately unsuccessful) pursuit of 2008 and 2012 Summer Olympics bids for the city when he was deputy mayor under Bloomberg. He is also chairman of the board of The Shed, the new $475 million arts center in Manhattan.

But it was his own family’s agonizing experience with ALS that prompted him to start the nonprofit.

His father, Martin Doctoroff, a Michigan state appeals court judge, developed a limp in 1999 and was diagnosed with ALS the following year. He died in 2002 at age 69.

About five years later, Martin’s younger brother, Michael Doctoroff, a Natick businessman, was also diagnosed with the disease. He died in 2010 at 74.

“When my uncle passed away, it was clear it was genetic,” Doctoroff said.

Only 5 to 10 percent of cases appear to stem from a genetic mutation, according to the ALS Association.

In addition to the $10 million that Doctoroff has donated to the foundation, Bloomberg Philanthropies has contributed $20 million, and the financier and philanthropist David M. Rubenstein has donated $10 million.

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The foundation plans to go back to the same donors, and reach out to new ones, to raise the $100 million more for the five-year plan.

Doctoroff said he believes there is a 50-50 chance he has the genetic mutation that caused ALS in his father and uncle, but he’s not undergoing any DNA test.

“There’s nothing I can do about it, so what’s the point in actually knowing?” he said. “I’m going to live my life the way I want to live my life.”


Jonathan Saltzman can be reached at jsaltzman@globe.com.