Department of Neurology, Huashan Hospital, Haishi.
"Tuk tuk tuk!" Zheng Jiamin calmed down and knocked on the door with her daughter.
"Is it you, have you finished taking Tiantian's medicine?" The doctor inside recognized her and immediately greeted her with a smile.
"Well, hello, Dr. Liu," Zheng Jiamin nodded and handed over the case in her hand with a smile on her face.
"You are on duty today. This is a child's case. Please prescribe another course of medicine."
As she spoke, she sat on a chair and held her daughter on her lap.
This is a girl about 6 years old, with two pigtails, looking around curiously.
While looking at the examination report, Dr. Liu asked, "Has the child's condition improved recently? What is the frequency of attacks? Does he still have a fever?"
Zheng Jiamin recalled the child's condition and said slowly: "It's not bad. The condition has eased in the past few years. Except for when you have a fever, you need to pay extra attention. Other times, it's quite normal."
"Currently, I have attacks once or twice a month. Generally speaking, they are not very serious and can be relieved by taking a shot of diazepam. Compared with the first attack four years ago, it is much better."
Dr. Liu nodded: "This baby has refractory epilepsy. It is really troublesome. You must not relax at all. If the attack is serious, you must send it to the ICU for rescue in time."
"Yeah, I know. I usually pay attention not to feed the child, nor to make her too excited. She must sleep well and not be constipated. She cannot eat chocolate, coffee, or anything else, otherwise she will be ruined. ”
Zheng Jiamin replied seriously: "Besides this, I usually read information on the Internet when I have time. I remember this Dravet syndrome. It is said that there is no specific medicine at present. I can only take some medicine to relieve symptoms and slow down the frequency of attacks. , cannot be cured.”
"Over time, drug resistance will develop with these medicines, so I am careful when giving her medicines. I don't seek merit but seek no faults. I don't seek to completely eliminate attacks. The frequency of one or two attacks a month is acceptable. "
At this point, she suddenly remembered something and asked expectantly: "Has the midazolam nasal spray I asked you to inquire about last time introduced into the country now?"
Dr. Liu adjusted his glasses and muttered: "Midazolam, I remember it was launched abroad 7 years ago. It is the first drug on the market that can stop epileptic states through nasal spray. In the past two years, it seems There was news that it was going to enter the country, and there was a lot of buzz for a while, but recently it is said that it has been given up.”
"Ah? Why?" Zheng Jiamin asked eagerly.
Dr. Liu explained: “The reason is this. This drug has been approved as an orphan drug abroad, but its indication is status epilepticus or multiple consecutive attacks, not Dravet syndrome, so it does not meet the 121 domestic drug requirements. Rare disease.”
"You also know that epilepsy is a common neurological disease and is not a rare disease. Therefore, this drug cannot be introduced into the country in accordance with the standards for rare disease treatment drugs, nor can it enjoy the country's rapid introduction, exemption from clinical launch, etc. preferential policies.”
"If this drug was to be introduced into the country as an epilepsy drug, it would require further clinical trials, and the investment would surge. The company could not afford it, so it gave up."
"How could this happen?" Zheng Jiamin's face was sad and disappointed.
She was originally looking forward to this drug, thinking that its introduction into the country was a sure thing. She thought that after it was launched, it would at least be a symptomatic drug that could greatly alleviate her daughter's condition.
I never expected that it would be such a result.
However, she has no complaints about pharmaceutical companies and can understand their desire to make money.
Since a new drug is developed, it must be registered according to the disease with the most patients, so that more drugs can be sold. It is impossible to register it according to a rare disease such as an orphan drug.
But in this way, there is no way to enjoy the various convenient policies for rare diseases and save the cost of clinical trials.
It can only be said that there are gains and losses, and each has its own difficulties.
"Well, it would be great if there were medicines that could treat the disease in China, so we wouldn't have to look hard for imported medicines."
Such thoughts came to mind, and she couldn't help but feel frustrated: "Rare diseases still don't pay enough attention. Only Sanqing is making generic drugs in this area in China, but Sanqing alone can't do it."
"Let me prescribe two more boxes of Clobazam for you," Dr. Liu said.
Zheng Jiamin came to her senses and nodded.
Dr. Liu considered and asked: "Is it still Sanqing's medicine? Recently, several domestic manufacturers are making clobazam generics. They are very effective, cheaper, and very cost-effective..."
Zheng Jiamin interrupted him and said firmly: "It's okay, just prescribe Sanqing's Clobazam. Its medicine is very effective and my children are used to it. I don't trust other medicines."
Dr. Liu smiled, said no more, and prescribed the medicine.
Zheng Jiamin took her daughter to get the medicine and took a taxi home.
Sitting in the car, looking at the familiar street scene outside the window, she suddenly felt sad, her nose became sore, and two lines of hot tears flowed from the corners of her eyes.
It has been five years since her daughter had her first epileptic seizure. She clearly remembers that it was a Saturday morning. Her husband was holding the child, and the sun was shining in from the window, making the room very warm.
The child may have been too hot, so she raised her hands uncontrollably, her eyes became glazed over, and her eyes began to tilt to one side. She didn't respond even if she shouted loudly.
This seizure lasted for 15 minutes. They were at a loss at first and didn't know what to do.
Seeing that my daughter's pink lips slowly began to turn purple and then gray, with no relief, I panicked and took a taxi to the hospital and was admitted to the ICU.
One week after being discharged from the hospital, her daughter was sent to the hospital again due to fever and convulsions. This time she was diagnosed with Dravet syndrome, a rare childhood disease caused by mutations in the SCN1A gene.
The couple can be considered intellectuals for settling in a first-tier city like Haishi, but they are also confused about such a result.
Dravet syndrome? Never heard of it.
But they understood the intractable epilepsy in children caused by genetic mutations.
Zheng Jiamin is still deeply impressed. That day, when she was leaving get off work, she received a call from the genetic company to officially confirm her daughter's illness. At that time, she felt a tightness in her chest, she couldn't breathe, and she was completely exhausted.
Because she was afraid that the elderly would be worried, she did not dare to tell her family, so she and her husband found an excuse to go downstairs for a walk, and walked around the community again and again, unable to accept the fact that their daughter had a terminal illness.
After walking all night, on my way back, I passed a small garden and saw an iron gate locked from the inside.
The two stood in front of the iron gate. They felt that the whole world was like this garden, completely locked by the iron gate. Their mood collapsed instantly. They couldn't help hugging each other and crying.
Later, it took half a year for the two of them to calmly accept the reality. They began to check a large number of relevant documents, and found that it was not that scary after all.
Many children with Dravet syndrome are in and out of the ICU during infancy and may face paralysis and sudden death at any time.
After the age of 2, it will be relieved a lot and the risk will be greatly reduced.
But the seizures of epilepsy are uncontrollable, and she and her husband don’t know when the next seizure will be or how long it will last. It might be a few minutes, it might be thirty or forty minutes, or even an hour. Every time, they are worried. tremble with fear.
Although their daughter is still a frequent visitor to the ICU now, the two of them have been able to face it calmly and are not so anxious.
The only thing that worries them is that there is no specific medicine and they can only take conventional anti-epileptic drugs. Not only can they not cure the condition, they cannot even control the seizures.
To some extent, it can be regarded as a terminal illness.
Therefore, besides keeping an eye on other drugs and making plans for the future, she didn't know what to do and could only resign herself to fate.
In the past five years, for her small family, life has been like a piece of waste paper, full of wrinkles, but there is nothing to talk about.
Epilepsy was like a big whirlpool, wrapping her up and making the small door in her world tightly closed, not letting in any light.
Huashan Hospital was far away from home, and there were so many thoughts in her mind that she felt tired before she knew it, and she gradually fell into a deep sleep.
Suddenly, a buzzing vibration woke her up.
At first glance, it turned out to be a call from Dr. Liu. When I was hospitalized, I took down his phone number and asked him some questions about his condition.
"Hey, Dr. Liu, I'm sorry, what's going on?" Zheng Jiamin braced herself and asked in a vague tone.
"Sweet mom, right? I have good news for you. There is a new genetic drug for Dravet syndrome." Dr. Liu's voice was very excited on the phone.
"What?" Zheng Jiamin couldn't believe her ears.
Dr. Liu was also very excited and explained nonstop.
"Haha, I just learned that Sanqing Group has newly developed an anti-epileptic gene drug. It has just started clinical trials in our hospital, but some patients in other hospitals have already received the treatment, and the feedback is that the efficacy is quite good."
"Children who have received this gene therapy have had their spontaneous epileptic seizures reduced by about 80% on average, and some of them even no longer have epileptic seizures at all, and this therapy has no negative impact on the child's cognitive ability. Influence."
“Since this drug works by reducing the excitability of abnormal neuron cells and returning them to normal, it is not targeted at specific diseases. Therefore, it is quite universal for epilepsy and can also solve the patient’s tolerance. Troubled by medicine.”
"I have seen the experimental reports of the first and second phases of clinical trials. The data shows that all patients' epileptic seizures have been relieved. The degree of relief is very amazing."
"Although this kind of gene therapy cannot completely solve the problem of SCN1A gene mutation, it should be the only treatment option for Dravet syndrome."
"If your Tiantian wants to participate, please come to the hospital to sign up as soon as possible."
"Great, I'll come right away." Zheng Jiamin's voice was so excited that her voice trembled.
Sanqing Group's genetic medicines are very famous, and they are very popular. My daughter originally suffered from a disease caused by a genetic mutation. Gene therapy from this direction is very promising.
She hung up the phone, wiped the faint tears from the corners of her eyes, and patted the back of the front seat eagerly.
"Master, I want to change my address. Please drive back to me."
"Yes, that's where you get on the bus, Huashan Hospital."
The taxi master also heard the conversation and knew that treatment was important. Without saying a word, he made a U-turn at the intersection ahead and drove quickly down the road.
Zheng Jiamin sat in the back, holding her confused daughter tightly in her arms and looking out the window with a smile.
This time, when she thought about her daughter's condition, she was not depressed or disappointed, but full of hope.
The door in my heart seemed to be no longer closed, but opened a wide crack.
The sunshine pours in, the spring is infinite, and the hope is infinite.
****
Kunming City, Sanqing Headquarters.
Wei Kang leaned back, stretched his limbs, and let out a long breath.
After reading the project information all afternoon, his mind was a little groggy, but overall, the progress of each project was gratifying.
"Huh, there are a lot of drugs entering clinical practice this year. In addition to conventional drugs such as hypoglycemic drugs, anti-inflammatory drugs, and asthma drugs, a trump card drug has been released in the field of urology. All clinical feedback is very optimistic."
"There are also several psychotropic drugs developed last year that have also entered the third clinical phase. Psychotropic drugs have a long clinical cycle. It is quite good to achieve this progress in more than a year."
"The genetic laboratory has produced another breakthrough, a genetic drug for anti-epilepsy. The effect is amazing, enough to fill many gaps in the field, and the market prospect is broad."
"Elepsy is a disease that mainly affects children and elderly patients. There are millions of people across the country. If it can be successfully launched, it will be the greatest gospel for the majority of patients."
"In addition, there are two new improved drugs for antidepressant and antianxiety. The crystal forms and molecular formulas of common first-line drugs have been improved, and some new adjuvants have been added."
"Compared with similar drugs, the efficacy is secondary. The improvement is not too big. The main reason is that the adverse reactions are greatly reduced. From the clinical trials, the response from patients is pretty good."
"Currently, among all new drugs, these three are the most eye-catching. They are drugs to treat chronic renal failure, genetic drugs to treat epilepsy, and drugs to treat social phobia."
"They are all innovative drugs in this field. No new drugs of the same kind have yet been officially launched. We can increase our efforts to seize the market as soon as possible."
"Other manufacturers have related research and development projects, but the results are not as good as Sanqing's, and they are still in the clinical stage. As long as we grab the first batch, we can form a first-mover advantage."
"Haha, Sanqing has once again taken the lead in the field of psychotropic drugs and is at the forefront. It's really gratifying."
Wei Kang couldn't help but smile with relief as he thought about the clinical trial report of the new drug project.
Immediately, he looked at the last new drug. For this new drug, he had some ideas on the development policy, but he was still unable to make up his mind.
"The remaining antioxidant drugs also have great future."
"Anti-oxidation is a very hot concept, and it is closely related to anti-aging. It can be said that it is cooking oil and is widely sought after."
“Anything, whether it’s drugs, cosmetics, or food, as long as it’s linked to anti-aging, there’s nothing that’s not profitable.”
"People's desire to delay aging has not diminished at all for thousands of years."
“This is one of those rare areas where top and bottom take advantage of each other.”
"It's just that for anti-aging, the clinical trial cycle will be longer."
“Generally speaking, drugs that delay aging are most suitable to be made into health care products.
"But medicines are three-part poisons, and any medicine is a double-edged sword. It is difficult to control the degree. It is best to make it a prescription drug and let doctors control it."
"Otherwise, if an elderly person buys it and eats it randomly and develops problems, the company's reputation will be lost."
(End of chapter)
