We seem to be in the middle of an extravaganza of medical research miracles, holding out hope where there wasn’t much before now.

The University of California just shared this amazing story about how UC San Francisco’s researchers are paving the way for a cure for multiple sclerosis in our lifetimes.

It was a discovery that grew out of the revolutionary new theory about MS that they developed – a theory so revolutionary that the main federal funding agency rejected it as ‘biologically implausible’ in the late 1990s when the team sought funding for a clinical trial of a new medicine, rituximab.

UCSF Neurology Professor Stephen Hauser, M.D., tells how they persisted in their theory, finding a pharmaceutical company to fund the trial – in his 2023 memoir, “The Face Laughs While the Brain Cries: The Education of a Doctor’. 

“It’s a story that’s optimistic, that imbues faith in science, and shows that science can improve people’s lives,” he told MedPage Today. “I wanted people to understand the phenomenal progress that we’ve made against MS, a disease that affects millions of people worldwide.”

It’s also a story about how fragile medical research is, he said.It is the story of a lifelong quest that began when Dr. Hauser was a medical resident treating a young woman who was a recent Harvard Law graduate and rising star in President Jimmy Carter’s administration whose brain was being ravaged by an explosive case of MS. 

But in the 1970s, there was no treatment. She lost her ability to speak, swallow and breathe. At her bridal shower just before she was married, she was hooked up to a breathing tube and strapped in a wheelchair. “I decided right there that this common, crippling disease of young adults would be my life’s work,” he writes.

His research has advanced our understanding of the genetic basis, immune mechanisms, and treatment of multiple sclerosis, said the American Brain Foundation in 2022 when it awarded him its Scientific Breakthrough Award. 

“His work led to the development of B cell therapies for MS patients, representing a powerful new approach for progressive forms of the disease,” the Foundation said. “His 40+ year, career-long commitment has changed the landscape of treatment for what has been, until now, a relentlessly progressive form of MS.”

MS is a disease of the brain and immune system. Our brains contain a network of billions of neurons, each nerve cell wrapped in myelin. But with MS, our immune system mistakes myelin for a foreign invader and attacks the myelin. Our bodies are built to protect our brains and nervous system from infections, keeping them safe behind what doctors call the “blood-brain barrier”. 

Dr. Hauser proved that while T-cells disrupted this barrier, it was B-cells and the proteins they produced that attacked nerve cells’ myelin coatings and short-circuited the brain’s network of neurons. The trial, funded by pharmaceutical company Genentech, which was co-founded by UCSF professors, showed that Rituximab stopped the disease virtually in its tracks, changing the face of MS forever. 

That breakthrough led to the 2017 development of a second MS medicine, ocrelizumab and then a third, ofatumumab, in 2020, also pioneered by Dr. Hauser and his UCSF colleagues working closely with industry partners. Today, this trio of drugs is the standard treatment for MS globally, allowing many people to escape the fate that used to befall MS patients.

“Beautifully, this is a UCSF story but it also involves great colleagues at other centers across the globe, courageous industry leaders and fantastic philanthropists who believed in high-risk work at a time when traditional funding sources would never be able to support this research,” Dr. Hauser said. “And finally, without the thousands of patients who trusted in us and were courageous enough to volunteer for these clinical studies, this progress could never have been made.”

“What’s most exciting to me is that these medicines have been associated with dramatic gains for patients,” Dr. Hauser said. “I am confident that people whose MS is just beginning can truly be optimistic about their prospects for a life free from disability — this is completely different from a generation ago.”

In 2022, Dr. Hauser was honoured with the American Brain Foundation’s Scientific Breakthrough Award for his 40+ year commitment to evolving and deepening understanding of multiple sclerosis. His “groundbreaking work on multiple sclerosis has transformed treatment while also leading to discoveries in other areas of brain disease research,” the Foundation said.

Dr. Hauser says he’s more optimistic than ever about the prospects for a cure. “I think we’re well on the way,” he says. “We first need to have complete disease suppression, and we’re not quite there yet.” However, researchers have just completed enrollment of the first study testing if MS can be cured by aggressive treatment at the very dawn of disease. “I think everyone in the field is optimistic that this could happen,” he says.

He’s also confident about science making great strides in combating other devastating neurological diseases. “New technologies plus better disease models are opening the door for a better understanding and realistic prospects for therapies, preventions, repairs, and cures for people with brain diseases,” he told MIT Alumni magazine in February 2024.

Sources:

A cure for multiple sclerosis? Scientists say within our lifetime. University of California, June 13 2024

Stephen L. Hauser, MD: Career-Long Dedication Leads to Breakthrough Research in Multiple Sclerosis. American Brain Foundation, April 13, 2022 

The Quest to Solve MS: ‘It’s a Story That’s Optimistic’ MedPage Today, Sep. 8 2023

On the Path to a Cure for MS. MIT Alumni, Feb 2 2024

The Face Laughs While the Brain Cries: The Education of a Doctor. New Books Network, Jul. 1 2023

Cover image: UC San Francisco