【By Observer News, Qi Qian】
"When this pig was only a few months old, its legs began to bend. At first, it just wobbled. A few weeks later, it collapsed completely. By the age of one, it had started to pant heavily." According to a report by Bloomberg on October 29, these moments were both heartbreaking and exciting for Professor Jia Yichang from the School of Basic Medicine at Tsinghua University. He had been waiting for years.
Public information shows that Jia Yichang is currently 53 years old and has long been working hard to research and try to solve the problem of Amyotrophic Lateral Sclerosis (ALS), also known as "Lou Gehrig's disease". Before this, most of his career was spent on mice, by inserting patient gene mutations into the corresponding gene loci in mice, trying to replicate ALS to enhance understanding of the disease mechanism and accelerate the development of treatment methods and programs.
Now, such trials have succeeded in pigs, which proves that ALS can be modeled in larger animals.
This provides Jia Yichang with new research clues and based on this, he has developed the world's first gene therapy drug for ALS, SNUG01.
According to the website of Jia Yichang's company, "Shenjichanghua", in March this year, SNUG01 received approval from the U.S. Food and Drug Administration (FDA) for an Investigational New Drug (IND) application, achieving a key breakthrough from basic research to clinical transformation. This means that SNUG01 has been granted "Orphan Drug" designation (ODD) in the United States, enjoying preferential tax status.
In May this year, the drug officially received a clinical trial implied approval from the National Medical Products Administration (NMPA) of China.
Currently, Shenjichanghua is actively promoting international multi-center clinical trials in China and the United States. If successful, the drug could help 90% of ALS patients.
SNUG01 drug information image
Bloomberg reports that these developments echo China's ambitions in the biotechnology field. This area has been dominated by the United States and European countries so far, and China is committed to becoming a "global technological power," challenging for global leadership. The report believes that full investment in gene editing research may be a key plan for China to develop innovative drugs and reduce reliance on foreign pharmaceutical companies.
Data shows that in 2024, the global gene-modified animal model market is estimated to be $15 billion, doubling within ten years.
In recent years, regulatory authorities in the US and Europe have required strict ethical reviews, making similar studies involving large animals almost impossible. Meanwhile, Western society's animal protection concept has changed, and activists oppose any animal testing. Surveys show that 80% of Americans support gradually phasing out animal experiments.
Compared to this, China's biotechnology sector has made significant progress. The report cites data indicating that China has the largest research monkey population. Since 2010, China has established eight state-owned medical research centers, housing tens of thousands of mice, rabbits, birds, dogs, and primates for research. In 2019, China launched two state-owned gene research centers to develop medical models using pigs and primates.
Currently, the main animal test subjects in the West are mice. Experts generally believe that mice are cheap, reproduce quickly, and are easy to genetically manipulate, but they are imperfect proxies for humans. Their short lifespan means that diseases sometimes develop slowly in mice and may not appear at all, such as cancer.
Bloomberg chart
This obvious gap has raised concerns in Western society. A report released by the German think tank "Mercator Institute for China Studies" warned that the advancement of China's biotechnology industry poses a risk of "over-reliance" on the U.S. and European pharmaceutical supply chains.
At the same time, some Western critics say that large animals are too close to humans, with strong social, intellectual, and emotional bonds, which can easily cross the ethical boundaries of animal testing. Andy Greenfield, a geneticist and reproductive biologist at the University of Oxford in the UK, said, "Many of them are social animals, with consciousness, feeling pain and happiness. These experiments must be very cautious and proven to have real benefits; they cannot be driven solely by curiosity."
However, for Peng Lin, co-founder of Shenjichanghua, the answer is clear - human health comes first.
ALS typically presents as damage to upper and lower motor neurons, as well as progressive muscle weakness and atrophy throughout the body. The incidence rate in China is about 4 per 100,000, with 30,000 to 50,000 new cases each year. The average age of onset is around 55, and the survival period is only 3 to 5 years.
Bloomberg mentions that currently, there are no effective treatment drugs for ALS globally. Thousands of patients die silently every year. From Stephen Hawking, we can see the cruelty of the disease: first muscle weakness, then paralysis, and finally respiratory failure.
Jia Yichang admits that he feels guilty every time he dissects an animal. But this has not changed his mind.
"I personally believe that animal models are still very important for discovering disease mechanisms and developing drugs," Jia Yichang said. "We should value the lives of animals and their contributions, but we should also have compassion for our helpless human comrades. If ALS can be cured in animals, perhaps we can help our human comrades."
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Original: https://www.toutiao.com/article/7566554066084512292/
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