Using Gene "Bridging", He Crossed the "Minefield" of Brain Disease Treatment

Using gene "bridging", he crossed the "minefield" of brain disease treatment

"Sometimes, it strikes me how fragile life is—here one moment, gone the next."

After 30 years in medical research, Lian Qizhou, a professor at the School of Synthetic Biology, Shenzhen University of Technology, couldn’t help but sigh.

This feeling first hit Lian back in the 1990s, when he was a clinical doctor pursuing a PhD, determined to master heart transplantation techniques to save more cardiac patients.

However, fate had other plans. Instead of fulfilling that wish, he stepped into an uncharted "minefield"—conquering Metachromatic Leukodystrophy (MLD), a global medical challenge.

Today, Lian has not only achieved breakthroughs using gene and stem cell technologies but also recently commercialized his 成果，with related contracts exceeding 300 million yuan.

"Medical technology is inherently industrial; its ultimate goal is to make life less fragile," Lian says.

Multiple "career shifts" to save lives

As a rare disease with an incidence of 1 in 50,000, MLD’s pathogenesis is straightforward. "Simply put, genetic defects lead to a lack of enzymes that break down specific fatty substances," Lian told China Science Daily. This causes such fats to accumulate in the nervous system—especially the brain—constantly compressing and eroding neural tissues, leading to demyelination, neuroinflammation, neurodegeneration, and rapidly progressing neurodegenerative symptoms, ultimately threatening life.

Even today, MLD is extremely hard to cure; once symptoms appear, patients rarely survive. Back in the 1990s, when Lian was pursuing his PhD, he never imagined he’d one day confront this global challenge head-on.

In fact, what drove Lian to pursue a PhD was witnessing friends and even family die suddenly from heart issues during his time as a cardiologist. This grief sparked his desire to learn advanced techniques like heart transplantation.

At the time, Lian was studying and practicing at the Affiliated Hospital of Harbin Medical University. But he soon realized heart transplantation faced a major hurdle: a severe shortage of donors—an issue beyond individual effort. So, he shifted his focus to emerging cardiac interventional therapy, saving many patients.

Yet new problems emerged.

"Take myocardial infarction: treatment has drastically reduced acute mortality, but dead heart muscle can’t regenerate, leaving many patients to develop heart failure, with a 50% five-year mortality rate," Lian explained.

Just then, he learned that foreign researchers were using gene and stem cell technologies to grow mouse heart tissue for transplantation, avoiding rejection. This "stimulated" him. "As a clinician, it’s hard to experiment with myocardial regeneration—you must follow medical guidelines strictly, leaving little room for bold innovation."

Lian decided to learn new skills: "Otherwise, I can’t save more lives."

A "绝症" conquered by Chinese scientists

After earning his PhD and working as an attending physician for two years, Lian moved to Singapore for postdoctoral research in 2001. Over the next two decades, he worked at institutions in the Netherlands, the UK, and Hong Kong, China, focusing on gene and stem cell therapy. From a novice struggling with stem cell culture, he grew into a leading expert in cell and gene therapy.

Gradually, his focus shifted from "heart" to "brain."

"Though seemingly different, the underlying logic is similar," Lian told China Science Daily. MLD stems from a lack of fat-breaking enzymes. "So, if we repair genes via stem cells, edit them, and implant enzyme-producing stem cells into the body to spontaneously break down excess fats, wouldn’t that work?"

The theory was simple, but turning it into reality took 15 years. His achievements remained little-known until 2024, when a 2-year-old Swedish girl changed that.

Emma was diagnosed with MLD at 1, and by 2, the disease had damaged her optic nerves, leaving her feet inverted and unable to stand. Her parents sought treatment across Europe and the US, but all efforts failed—Emma seemed doomed.

By chance, Emma’s father, a medical worker, found Lian’s research papers. Hoping for a miracle, the family traveled to the Guangzhou Women and Children’s Medical Center—one of Lian’s collaborative clinical research sites.

Emma was admitted on August 30, 2024. After collecting autologous hematopoietic stem cells, placing a central venous catheter, and undergoing myeloablative conditioning, her genetically repaired stem cells were successfully transfused back on October 9.

On October 25, 2024, Emma—once given "no chance"—was discharged. Media coverage revealed that this "incurable" disease could be overcome, thanks to Chinese scientists.

"Emma wasn’t our first success. We’ve cured several adolescent MLD patients with symptoms, with up to 10 years of safety and efficacy follow-ups," Lian said proudly. "We’ve crossed a 'minefield' in brain disease treatment that others never dared to enter."

Medical industry needs more than "money"

Lian still communicates closely with Emma’s family and local doctors. As attention grew, companies flocked to commercialize his 成果.

Lian had clear plans.

"Commercializing medical technology is natural—otherwise, it betrays the mission to save lives," he said. But due to its life-or-death nature, choosing the right partner was critical.

Lian admitted many firms offered huge sums, but some were outsiders to healthcare. He turned them down.

"When selecting partners, sincerity matters most—whether they see industrialization as a cause, not just a capital game," he said. "Healthcare isn’t about throwing money at problems. I want partners who understand the industry, so we can collaborate to optimize the technology post-transfer."

After careful selection, Lian finally found the right fit, recently transferring full technology for over 300 million yuan.

Lian noted MLD, a representative hereditary white matter rare disease, has a huge unmet market demand. The MLD treatment market is projected to reach $4.92 billion by 2029, with a 6.2% CAGR.

"Amid this huge market, we must stay calm. Beyond market prospects and corporate growth, we must ensure technology maximizes its life-saving value—that’s what a medical scientist should prioritize," Lian said.