In the past 2020, we have not only been fighting against the COVID-19, but also against other human diseases. In the field of biomedicine, which is closely related to human health, many innovative therapies have matured this year and promoted medical progress. Recently, the authoritative academic journal "Nature Medicine" published a series of articles reviewing ten remarkable advancements in 2020.
1. Early cancer detection: Blood testing identify asymptomatic cancers
Early cancer detection is the key to improving treatment effectiveness and survival rate. A prospective study published in Science in 2020 demonstrated that a screening method combining blood testing and PET-CT scan imaging can detect signs of cancer in asymptomatic patients and identify those who need treatment.
The Johns Hopkins University research team screened more than 10,000 women without a history of cancer, detected tumor DNA and cancer protein markers in their blood samples, and then used PET-CT scans to confirm positive results. In 12 months, 26 different types of cancer were detected early, and there are currently no standard screening methods for some cancer types.
There are 26 cancers (blue dots) in 10 organs were found via blood testing, 12 of them (red circles) are curable with surgery, combined with with PET-CT, 9 cancers of the 12 were identified. (picture source: reference )
Expert comments on the New England Journal of Medicine (NEJM) pointed out that “In the future, sensitive and specific liquid biopsy may become a routine project, and this research is a milestone on the road to the future”.
KRAS is the most frequently mutated oncogene in cancer, encoding an important signal protein in tumors. In one-third of cancers, KRAS is activated. For more than 40 years, scientists have been trying to target KRAS to develop anti-cancer therapies. At the end of 2019 and early 2020, small molecule inhibitors targeting KRAS appeared.
At the end of 2020, clinical trials have brought gratifying results. According to the Phase 1 clinical results published on NEJM, the inhibitor sotorasib on research, which targets the KRASG12C mutation, showed good anti-cancer activity in 129 solid tumor patients.
Recently, the FDA has granted sotorasib breakthrough therapy designation for the treatment of locally advanced or metastatic non-small cell lung cancer (NSCLC) patients with KRASG12C mutations.
3. Medical AI: Breaking the "black box"
The application of artificial intelligence (AI) in medical care, transparency and repeatability are issues of great concern. It is increasingly recognized that interventions involving AI require rigorous forward-looking evaluations to demonstrate the impact on health outcomes.
In 2020, in order to evaluate clinical trials with AI-based interventions, clinicians, scientists, patients, and journal editors have discussed and produced two related guidelines: SPIRIT-AI and CONSORT-AI.
T cells expressing chimeric antigen receptors (CAR) (CAR-T therapy) are one of the major breakthroughs in the field of cancer treatment in recent years, and they have achieved excellent efficacy in the treatment of blood cancers.
In 2020, a study published by scientists at the University of Pennsylvania showed that CARs targeting tumor antigens can not only be expressed on T cells, but also on macrophages, making macrophages a "cellular weapon" against solid tumors. This therapy is called CAR-M. In a mouse model of HER2-positive metastatic ovarian cancer, it successfully reduced tumors in animals and prolonged their lifespan.
Another study published in NEJM modified natural killer (NK) cells to express chimeric antigen receptors against CD19. In clinical trials, 11 cancer patients received this CAR-NK therapy, and none of them had the common toxic effects of CAR-T therapy.
"Nature-Medicine" commented that these advances mean that "cancer treatment ushered in a new era of CAR cells."
5. Diabetes: only need to inject insulin once a week
Many patients with type 2 diabetes who require insulin therapy often need to inject at least once a day, or as many as 2-4 times, and may also need to be combined with oral medications, which means complicated treatment strategies and self-blood glucose management. In the future, insulin that only needs to be injected once a week may greatly simplify the medication regimen and reduce the burden on both doctors and patients.
The insulin analogue icodec developed by Novo Nordisk has shown in the results of phase 2 clinical trials published in 2020 that it can reduce the number of patients' basal insulin injections and reduce the frequency of injections to once a week.
At present, a number of long-acting insulins under research have entered Phase 2 clinical trials. These innovative therapies are expected to bring revolutionary changes to the quality of life of patients with type 2 diabetes.
6. RNA therapy has begun to attract attention
In 2020, a variety of RNA-based therapies will show promise for clinical applications.
Acute hepatic porphyria (AHP) is a rare genetic disease. Due to the lack of specific heme biosynthesis enzymes, porphyrins accumulate and produce neurotoxicity. There is no approved therapy for this disease in the past. New data from a phase 3 clinical study published in June, 2020 showed that an RNAi therapy (givosiran) has long-term therapeutic benefits for AHP patients and has good safety during the treatment period.
Years ago, the ice bucket challenge brought the fatal disease "gradual freezing" (amyotrophic lateral sclerosis, ALS) into the public eye. In July, 2020 NEJM published two important studies at the same time, and the two gene therapies brought hope of cure for ALS patients. One of the test drugs tofersen is an antisense oligonucleotide, which reduces the synthesis of the pathogenic protein SOD1 by regulating the degradation of the messenger RNA of the pathogenic gene. Another experimental drug is to target disease-causing genes and reduce disease-causing proteins by encoding microRNAs. Two patients received the treatment, and one patient showed improvement in clinical indicators.
7. Scientific Community: Call for Equality
On June 10th, 2020, many people in the scientific community participated in the "strike for the lives of blacks", and through a one-day academic shutdown, they supported blacks and other minorities facing racial discrimination in the scientific and medical circles.
8. Poliomyelitis: One step closer to eradication
On August 25, 2020, the World Health Organization's African Regional Committee for Poliomyelitis Eradication Certification announced that the wild polio (polio) virus that causes polio has been eliminated in Nigeria. This historic achievement marked the extinction of wild poliovirus across the African continent.
In 1988, WHO launched a global initiative to eradicate the spread of wild poliovirus. Because poliovirus is highly contagious, to be certified as a "wild polio-free state", a number of criteria must be met, including no cases have been detected for 3 consecutive years. Currently, only the last two countries in the world still have wild poliovirus circulating.
9. Parkinson's disease: personalized therapy
Parkinson's disease is related to the loss of neurons in specific areas of the brain, leading to dopamine deficiency. Therefore, a strategy that scientists have tried for a long time is to use stem cells to regenerate dopaminergic neurons.
In February of 2020, NEJM reported a success case. Researchers at Massachusetts General Hospital and Harvard Medical School reprogrammed cells from a Parkinson's disease patient into induced pluripotent stem cells (iPSC) in vitro and differentiated these cells into dopaminergic progenitor cells. Without the need for immunosuppressants, these cells were implanted into the patient's left and right brains. After 18-24 months of implantation, the patient's Parkinson's disease symptoms improved and there were no adverse reactions.
Researchers believe that this set of strategies can provide patients with personalized treatments for autologous cell therapy for Parkinson's disease.
10. Know about changes in cholesterol in the global population
High blood cholesterol level is an important risk factor for cardiovascular and cerebrovascular diseases, and it used to be considered as a characteristic of wealthy Western countries. This trend has changed a lot in the past 40 years.
A joint team composed of nearly 1,000 researchers from around the world compiled data from 1,127 population studies from 1980 to 2018, and analyzed the cholesterol levels of more than 100 million adults in more than 200 countries and regions. The analysis trend shows that due to the influence of diet, lifestyle, lipid-lowering drugs, etc., cholesterol levels in some European countries have dropped significantly, while in Asians, cholesterol levels are increasing year by year.
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 Early cancer detection. Nature Medicine. Doi: https://doi.org/10.1038/s41591-020-01148-x
 Anne Marie Lennon et al., (2020) Feasibility of blood testing combined with PET-CT to screen for cancer and guide intervention. Science. Doi: 10.1126/science.abb9601
 KRAS inhibitors at last. Nature Medicine. Doi: https://doi.org/10.1038/s41591-020-01149-w
 A shift in cholesterol geography. Nature Medicine. Doi: https://doi.org/10.1038/s41591-020-01150-3
 New CARs in the therapeutic fleet. Nature Medicine. Doi: https://doi.org/10.1038/s41591-020-01151-2
 A personal approach for Parkinson’s disease. Nature Medicine. Doi: https://doi.org/10.1038/s41591-020-01152-1
 Once-a-week insulin. Nature Medicine. Doi: https://doi.org/10.1038/s41591-020-01153-0
 Science strikes for Black lives. Nature Medicine. Doi: https://doi.org/10.1038/s41591-020-01155-y
 Transparency in medical AI. Nature Medicine. Doi: https://doi.org/10.1038/s41591-020-01147-y
 RNA therapies prepare for the spotlight. Nature Medicine. Doi: https://doi.org/10.1038/s41591-020-01154-z
 WHO declares Africa polio-free. Nature Medicine. Doi: https://doi.org/10.1038/s41591-020-01156-x