For 15 years, I sat next to Stephen Kauffman and his dad Scott at Golden State Warriors games. Every National Basketball Association referee knew Stephen because he’d call them out by their first names for bad calls. Sadly, Stephen first started showing signs of neurodegenerative disease at age 27 and was soon diagnosed with amyotrophic lateral sclerosis, ALS. The disease, which causes the brain to stop communicating with the muscles, has no cure. Early on, Stephen would ask me to help him stand for the national anthem and eventually attended games in a wheelchair and a device to help him breathe. He kept his same bright attitude and diligence with the refereeing, but his slow-motion decline was one of the saddest things I’ve ever seen.
Scott and his family deal with ALS 24/7. He is now chairman of the ALS Association. Meeting last week in Palo Alto, Calif., he told me that in the U.S. more than 30,000 people live with ALS, typically only for two to five years after diagnosis. Only 15% of cases are hereditary; the causes of the rest are unknown. There are no diagnostic tests for ALS—doctors eventually rule out everything else.
I’ve noticed that ALS is either fast or slow. In 2012, at age 57, one of my old college roommates told me he noticed he couldn’t hit a golf ball as far and got drunk on half a beer. He soon was diagnosed with ALS and died a year later leaving behind two teenage daughters. On the other hand, Stephen Hawking was diagnosed in his 20s and died at age 76. ALS is often known as Lou Gehrig’s disease. The Yankee great died with ALS at age 37. Here’s a twist: Stephen, now 38, was a huge Lou Gehrig fan as a kid—he had a Gehrig-signed baseball and even wore his No. 4 in Little League.
Only a few drugs slow the disease’s progression, and none provide a cure. Scott told me that the Food and Drug Administration “has pledged to bring ‘regulatory flexibility.’ Of course, patients are willing to risk everything for drugs that might work once they’re deemed safe.” In September, the FDA approved the drug Relyvrio from Amylyx Pharmaceuticals. Patients who take it generally show a slower rate of decline and live longer, but only by a few months.
In July the FDA accepted Biogen’s Tofersen into its accelerated-approval pathway, and it might receive full approval this spring. The drug binds to the SOD1 gene whose mutation is common in ALS patients. A few other drugs are in the pipeline, but little is known about what causes ALS, which makes developing treatments difficult. There is so much research to be done. That takes money.
Remember the ice-bucket challenge in 2014 that took over social media?
