Release date: 2017-11-17
Brian Madeux is 44 years old. In addition to being a little shorter than the average person, this middle-aged man has no place to attract attention.
Today, Brian has become the focus of the world: he is the first person in human history to receive genetic modification therapy.
Brian has become the focus of the world today (Source: Eric Risberg / AP)
“I feel sincere and fearful to be the first patient (to receive GM therapy),†Brian said. “I am willing to accept this risk. I hope it will help me and help other patients.â€
Brian suffers from a rare disease called Hunter syndrome. Around the world, there are fewer than 10,000 patients like him. These patients lack an enzyme called IDS. As a result, toxic metabolites that should have been degraded continue to accumulate in the cells, causing devastating effects on organs such as the heart, bones, and brain.
Many patients will leave the world before they reach adulthood. From this perspective, Brian, 44, is a miracle.
But this does not change the fact that Brian is also a patient. Years of illness have made his body broken. Since he got sick, he has undergone 26 operations to solve a variety of problems. Last year, due to bronchitis and pneumonia, he almost died in his respiratory secretions.
Brian is the first patient to undergo genetic modification therapy (video source: AP)
He doesn't want to live like this anymore. At this time, a California biotechnology company called Sangamo Therapeutics entered his life. The doctor said that the company is developing several gene therapies, one of which is exactly what Hunter syndrome is.
"I have been waiting for this moment in my life." Brian said in bed.
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When it comes to gene therapy, many people are not unfamiliar. In August of this year, the US FDA approved the first gene therapy: scientists can isolate immune T cells from cancer patients, genetically engineer them to recognize cancer cells, and launch attacks. The first girl to receive this therapy has been cancer-free for five years.
Just last week, a study was published in the top academic journal Nature. Using gene therapy, doctors repaired 80% of the body's skin for a rare sick child in just two months, pulling him back from the death line.
However, the gene therapy that Brian wants to accept is quite different. Previously, these therapies were used to separate cells from the patient's body, perform genetic editing in the laboratory, and apply these cells to patients.
Sangamo's gene therapy involves editing the genome directly in the patient.
"We cut your DNA, open a hole, insert a (normal) gene, and sew it up. You can't see the traces of repair," Dr. Sandy Macrae, CEO of Sangamo, said: "It will be you." A part of DNA will accompany you for life."
CEO of Sangamo: The real growth in the field of gene therapy lies in the innovation of drug delivery methods
This method is expected to cure Hunter's syndrome once and for all. Before the birth of gene therapy, patients can only receive enzyme replacement therapy once a week, and enter the IDS enzyme missing from the body. Each year, the cost of enzyme replacement therapy ranges from $100,000 to $400,000. Moreover, intravenous infusion of enzymes cannot break the blood-brain barrier and therefore prevent brain damage.
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On Monday, Brian came to the Benioff Children's Hospital at the University of California, San Francisco (UCSF). There, the nurse wiped his upper arm with a cotton ball and inserted the needle into his vein. Brian is familiar with the process. It seems that this process is no different from enzyme replacement therapy.
However, following the needle into Brian's body, it is not a mature IDS enzyme, but a functional IDS gene and a genetic modification tool that can cut Brian DNA at a fixed position. The number of these molecules is one billion.
Introduction to the principle of gene therapy (video source: AP)
The treatment uses not the current hot CRISPR-Cas9 gene editing method, but a more precise zinc finger nuclease (ZFN) technology. Using a special virus, the researchers shipped the normal IDS gene and the ZFN transformation tool into the liver cells. Then, ZFN cuts a hole in a specific position in the genome, and the normal IDS gene can be seamlessly "pasted".
Brian's genome will be different. For the first time in 44 years, his liver cells will be able to synthesize a healthy IDS enzyme.
Researchers say that as long as 1% of liver cells can be genetically engineered, the enzymes produced are enough to control the disease.
Prof. Carl June, a pioneer in gene therapy, is optimistic about the safety of this study (Source: Penn Medicine)
“How good is the security of this technology? We are observing,†said Professor Carl June, a pioneer in gene therapy at the University of Pennsylvania. He did not participate in the study, but he thought the previous safety test results were great.
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Concerns about security are entirely necessary. As with any treatment, gene therapy has its own unique risks.
Professor Eric Topol believes that "gene therapy is taking care of nature with care" (Source: Scripps Health)
"Gene therapy is taking care of nature with care," said Professor Eric Topol, a well-known scientist at the Scripps Institute for Translational Science. He believes that, limited to the current human cognition, we will never be able to confirm the risk 100%.
In the early stages of the development of gene therapy, viruses as carriers often cause an overreaction of the immune system. In 1999, an 18-year-old boy, Jesse Gelsinger, died and once stopped the gene therapy field.
The star scientist became a sinner overnight and he used himself for 16 years to redeem himself.
In addition, people are worried that genes will not be listened to and inserted into other parts of the genome. A few years ago, it was the abnormal insertion of genes that activated the cancer genes in several children. After receiving gene therapy, they got leukemia.
As a treatment for permanently changing the genome, once Brian chooses to receive treatment, there is no turning back.
Fortunately, we now have a better understanding and control of gene therapy. Brian's viral vector is very safe, ZFN's editing of the genome is also very accurate, and the theoretical risk of off-target is very low. To further increase safety, the researchers limited the scope of the genetic modification system to the liver. Therefore, it does not modify other organs or germ cells.
Brian quietly awaits treatment (Source: Eric Risberg / AP)
“When you randomly insert DNA, sometimes you get good results, sometimes you don’t get any results, and sometimes the results are even harmful to the human body,†says Professor Hank Greely, a bioethicist at Stanford University. “The advantage of genetic editing is that You can put the gene in any position you want."
"At the moment, there is no evidence that this therapy will become dangerous. It is not yet time to worry about fear," said Professor Howard Kaufman of the National Institutes of Health. Previously, it was his advisory panel that approved the trials.
Brian quietly awaits the results of gene therapy. Within a month, he will know if gene therapy is working. The final assessment of efficacy and safety will be performed within 3 months.
Fives
Gene therapy can't reverse the damage that Brian's body has. But once it works, it is expected to let Brian avoid further damage to the body.
Not far from the light, Brian re-imagined life. Many years ago, Brian was a chef who cooked a good dish, and his restaurant hosted many stars. He also loves to ride horses and enjoy the feeling of galloping in the fields. Nowadays, the disease has taken away his hobbies, and gene therapy is expected to get him back to his own life.
For 15 years, Brian's fiancée has never left him (Source: Eric Risberg / AP)
After returning to health, Brian plans to step into the marriage hall. His fiancée is a nurse and the two met in a treatment 15 years ago.
"My disease has the hope of healing for the first time," Brian said. "I feel nervous and excited."
Reference material
[1] AP Exclusive: US scientists try 1st gene editing in the body
[2] Courier | The future has arrived! First patient undergoes genetic modification of the human body – WuXi PharmaTech WeChat
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