Genetic Testing: Household Survey of Microorganisms in Food How will artificial intelligence change our medical structure? Code fragments suspended in the blood promote precision medicine in the United States. More than half of the public supports the recruitment of biological databases
With the completion of the Human Genome Project, human medicine is entering a new era. Massive medical breakthroughs and capital investments will usher in a new era of personalized precision medicine.
"Every patient is unique, and doctors have been doing their best to tailor medicines to each person. Just like if you want a blood transfusion, your blood type must match. This is a very important discovery. If We "match" an individual's genetic code to the cancer and use it as a practical standard to determine the dosage of the drug as easily as measuring our body temperature - this is the hope that precision medicine brings us." Obama , January 20, 2015
In his 2015 State of the Union address, U.S. President Obama announced that the United States would invest $215 million to launch a program called "Precision Medicine" (PM). Precision medicine has been brought into the public eye as a national strategy.
The so-called precision medicine is a new "tailor-made" medical model. In this model, medical decisions and implementation are customized based on the patient's individual characteristics. Disease diagnosis and treatment are also based on the patient's personalized genetic information combined with big data such as their individual environment, lifestyle, and personal history. carried out on the basis of.
Medical miracles in the new era
Newborn babies should enjoy the beauty of the world without any worries, but not every baby is so lucky. On July 25, 2015, Carina was born into this world with a loud cry like countless newborns. However, a small lump on her chin destined her fate to be different.
Initially doctors did not diagnose it, but later discovered it was a malignant tumor. Carina, who is only 1 and a half years old, has already undergone 8 rounds of chemotherapy and surgery.
Faced with the stubborn tumor’s continuous growth, Carina’s parents were exhausted both physically and mentally and were at their wits’ end. Radiation therapy might help, but the damage to the brain was prohibitive for the new parents.
The doctor recommended that they try any possible experimental drug.
Genetic testing of the tumor showed that Carina had an abnormal fusion of two gene sequences that caused the tumor. Carina's oncologist Ramamoorthy has found a clinical trial drug that can interfere with the protein synthesized by this type of gene fusion, but the drug is currently only for adults.
Later, Dr. Ramamosi and the drug manufacturer Loxo Oncology applied to the U.S. FDA for a clinical trial in children. Last December, Carina became the first clinical patient.
The tumor, which was initially as big as a walnut, was almost completely gone after 28 days of treatment. Although Carina will continue to take medicine, and the tumor may mutate again and come back, Carina is now a lively and cute two-year-old child.
Karina’s father said gratefully: This treatment has brought happiness back to our family.
The success of Karina and other similar cases is due to precision medicine.
Precision medicine is the intersection and application of biotechnology and information technology in medical clinical practice. It combines big data information from different sources to establish personalized, targeted and effective treatment plans for individuals.
But this is completely different from the existing medical model that selects a universal treatment plan suitable for most patients. For example, Memorial Sloan Kettering Cancer Center in New York City, a leader in this field, has sequenced the genes of 10,000 tumors since 2014 to provide oncologists with the opportunity to develop and design personalized tumor treatment plans.
Booming but slow progress in precision medicine
Any reform and transformation will encounter challenges. The development of precision medicine needs to face challenges from various fields, from the medical industry to the research and development of new drugs.
According to data from Silicon Valley Bank, venture capital firms invested $7 billion in biomedical startups across the United States last year. Most of this enthusiasm was due to breakthroughs in genetic drugs and related technologies.
The success of Loxo Oncology's new drug is a reflection of the role small companies can play. Loxo founder Joshua Bilenker said that the company’s development has benefited from numerous public big data information, such as the National Institutes of Health’s Cancer Genome Atlas.
With the simultaneous support of private equity funds and public funds, Loxo has currently received US$250 million in investment. Bilenkers said that Loxo is mainly targeting relatively simple genotypes of cancer, which means that the company can quickly know whether the treatment plan is effective, which is what investors are happy to see.
Compared with the research and development of new drugs for common diseases by big pharmaceutical companies, these treatment tests can recruit small groups, so the cost is smaller. Blockbuster drugs for common diseases often require large, expensive randomized trials. For cases like Karina's, it is almost impossible to find more than a few thousand patients in the United States for such trials.
This type of targeted genetic medicine is only suitable for a small group of patients and is unlikely to become a best-selling drug and bring large profits to the company.
A company like Loxo will likely recoup the cost of its investment by charging a large sum of money per patient.
Currently, the average cost of cancer treatment is $10,000 per month. But over the past decade, cancer drug prices have skyrocketed, and both insurance companies and patients have begun to worry about prices as the cost of treatment continues to grow for both parties. of).
At the same time, precision medicine certainly has technical risks.
Editas Medicine has been working on a new type of gene editing, CRISPR/Cas9, to treat genomic abnormalities and is expected to begin clinical trials next year.
However, there are still many complex scientific issues that need to be improved before this, such as gene editing itself and how to effectively deliver drugs to the appropriate cells.
Precision medicine has the potential to eventually treat major diseases, but it won’t be easy. Some diseases are very complex and may involve multiple genomes, making it difficult to find relevant corresponding genomic information.
In addition to genomic information, precision medicine also requires the collection of big data information such as patient environment, lifestyle and health history.
Companies such as WellDoc and Omada are trying to understand and record patients' life and health data through mobile communication devices. This data can help patients and doctors find better treatment entry points, especially for chronic diseases such as Patients afflicted by diabetes and hypertension.
Joseph Kvedar, director of the "Connected Health" project advocated by the Medical Alliance Group of the Boston academic medical system, said that despite the success of some projects, there are still A large proportion of patients do not form part of the audience. Precision medicine is nowhere near as deeply embedded as Snapchat, Instagram, or Facebook.
Keweida explained, "Sickness reminders will certainly not be as popular as social media, but we still have great opportunities. Various contemporary social media information and mobile communication information allow everyone to become Unique individuals. If we can track this personalized information, coupled with individual genetic information, we can go further. ”
As Obama said, “We need this information to make it possible. We are healthier ourselves and our families.
”
Pioneer of precision medicine
Herceptin, invented by the biotechnology company Genentech, is the earliest targeted gene drug. It was approved by the US FDA in 1998 and mainly targets HER2. Protein overexpression in metastatic breast cancer.
Since Roche, the Swiss pharmaceutical giant owned by Genentech, introduced Herceptin to markets outside the United States, Herceptin has treated more than two million patients around the world, and global sales have increased More than $64 billion. It is well deserved to define Herceptin as a pioneer in precision medicine.
The following is the development history of Herceptin: 1985 National Institutes of Health research shows that the HER2 gene is frequently overexpressed in breast cancer tumor cells. Genentech scientists, who had cloned the first human HER2 gene in 1990, found a way to humanize mouse-derived antibodies. Through genetic modification, they obtained humanized mouse antibody proteins that target the surface of cancer cells and bind to them. HER2 and does not trigger an immune response, thus Herceptin was invented. Third parties later estimated Gnentech's R&D costs at $150 million to $200 million. 1992-1998 Clinical trials were conducted to verify the safety and effectiveness of Herceptin. Herceptin is used alone or with chemotherapy in patients with HER2-positive metastatic breast cancer. In March 1998 Genentech announced a partnership with diagnostics company Dako to develop a commercial test to diagnose patients who overexpress HER2. In May 1998 Genentech applied to the FDA to place Herceptin on the market. The FDA believes that Herceptin fills a medical gap in the treatment of malignant tumors and has opened a "green channel" for a "priority review". The review will be conducted in the next six months instead of waiting 10 months according to the standard procedure. In September 1998 the FDA approved Herceptin for the treatment of HER2-positive metastatic breast cancer and approved a diagnostic test to help identify patients. August 2000 Europe approves Herceptin. From 2006 to 2008, the FDA approved three different classes of Herceptin-based postoperative treatments for early-stage HER2-positive breast cancer. Then Herceptin was approved for the treatment of gastric cancer. 2014 Herceptin’s first patent expires in Europe. An Indian biotech company approved a highly similar drug in 2013. Under his leadership, a Korean company also obtained approval for a similar class of drugs. Studies of the drug have shown no clinical differences in safety or efficacy compared with the original drug. This was followed by the approval of a number of drugs in Asia. In May 2015, shortly after President Obama announced a $215 million precision medicine research project, the World Health Organization added Herceptin to its list of essential medicines for low- and middle-income countries. In 2019, Herceptin’s first patent is expected to expire in the United States, which is expected to significantly reduce treatment costs. Topics: Personalized medicine, genes, genetic testing, genetic technology, new knowledge in biomedicine, precision medicine