Cosmologist Stephen Hawking, who has ALS, listens to a presentation by… (Eric Reed / Cedars-Sinai )
Clive Svendsen doesn't get rattled easily, but the neurobiologist couldn't help sweating when Stephen Hawking paid a visit to his lab this week.
Hawking is one of the world's foremost theoretical physicists. He pioneered groundbreaking research into how particles behave around black holes and deduced that black holes spit out radiation as they swallow up matter. He's also credited with teaching millions about the mysteries of the cosmos through his books, including the bestseller "A Brief History of Time."
For The Record
Los Angeles Times Wednesday, April 17, 2013 Home Edition Main News Part A Page 4 Local Desk 1 inches; 52 words Type of Material: Correction
Stephen Hawking visit: In the April 13 LATExtra section, the caption for a photo with an article about physicist Stephen Hawking's visit to the Cedars-Sinai Regenerative Medicine Institute reversed the identities of two people in the photo. The photo showed institute Director Clive Svendsen speaking while Dr. Robert Baloh and others watched.
But that's not all. Hawking is perhaps the world's most famous patient with amyotrophic lateral sclerosis, or ALS -- the degenerative nerve disease that Svendsen has been studying for decades.
On Tuesday, Hawking visited the Cedars-Sinai Regenerative Medicine Institute in Los Angeles, which Svendsen heads, to see how researchers there are using stem cells to develop treatments for the disease.
"We were both just -- awestruck is an understatement," said Dr. Robert Baloh, a colleague at the institute who also studies the condition.
ALS becomes devastating for the 5,000 or so people who get it every year. The motor neurons that control their muscles die, leaving them paralyzed and usually killing them within a few years. Hawking, who was diagnosed with the disease in 1963 and has spent most of his adult life in a wheelchair, is an unusual case.
Scientists don't know what causes the nerve death; nor have they been able to devise therapies to slow or halt the disease's course. But they hope that working with stem cells -- flexible, embryonic-like cells that can be trained to develop into various types of mature cells -- will help change that track record.
Hawking had requested a visit to Cedars-Sinai to learn more about the research. During an hourlong tour, his assistants took him through the institute's laboratory, where he viewed stem cells through a microscope and listened to brief "chalk talks" from Svendsen and Baloh.
At Cedars, scientists are using the flexible cells in two ways, they explained.
First, researchers are studying them to learn more about how ALS ravages the body.
In the past, neurologists couldn't look at a patient's nervous system until he or she had died. But today, the team can collect skin cells from a patient with ALS and rewind them to a very early stage of development. Those induced pluripotent stem cells, or iPS cells, can then be coaxed to grow into motor neurons, allowing scientists to observe what happens to those key cells as the disease takes hold.
Svendsen said that Hawking's eyes "lit up" when the team talked about the institute's second focus: using stem cells to create ALS treatments.
These studies employ a different type of stem cell that's derived from fetal brain tissue. Among this collection of cells, scientists isolate the precursor cells that develop into astrocytes -- a type of support cell that helps keep neurons healthy in normal individuals but may malfunction in people with ALS.
The Cedars team hopes to transplant the astrocyte precursor cells into the spinal cords of ALS patients to see whether they'll be able to replace the support cells that are sick and allow patients to maintain their motor function for a longer time.
The researchers are also planning to engineer the fetal stem cells so that they'll make a growth factor called GDNF. In experiments with rats and pigs, transplanting such cells into the spinal cord has slowed the death of neurons.
Such approaches are unlikely to affect Hawking, whose motor neurons died long ago, Svendsen said. But there is a small chance that some dysfunctional connections could reactivate after GDNF injections into the muscle -- perhaps, say, to restore function to a single finger.
"If he could regain function in a finger, that would be huge for him," Svendsen said.
Shortly after the tour, Hawking delivered a speech to 450 people, including Cedars-Sinai employees, ALS patients and their families.
Hawking discussed his childhood, cosmology and what he sees in the universe's future. He drew parallels between the stem cell center's work in discovering the mechanisms of disease and his own quest to understand the fundamental rules of the universe.
"If you understand how the universe operates, you control it in a way," he said.
He concluded by urging the audience to keep asking questions: "Remember to look up at the stars and not at your feet.... Be curious."
Baloh said that experiencing Hawking's curiosity about stem cells was particularly gratifying.
It takes the physicist a long time to converse -- he uses a cheek muscle to control a cursor and spell out words on a computer screen, which are then spoken aloud by a text-to-speech program. But Hawking asked Svendsen and Baloh several questions during the tour and a dinner.
He wanted to know, for example, whether anyone had been cured -- of anything -- using stem cells. (The answer: Yes, but it's still early.)