Check the list of main healthcare innovations in January 2020:
If a person has three or more of the following conditions they are said to have metabolic syndrome; central obesity, high blood pressure, high blood sugar, high blood fat, and low blood high-density lipoprotein (HDL). “Intermittent fasting” (IF) is a popular diet that commonly includes 16-hour fasts every day or fasting for two separate 24-hour periods in a week. A new variant of IF known as “time-restricted eating” (TRE) has recently been demonstrated to benefit people with metabolic syndrome.
The senior author of the study published in the journal Cell Metabolism was Pam R. Taub of the University of California, San Diego, Department of Medicine. Patients with metabolic syndrome were asked to select a 10-hour window in which they were allowed to eat for 12 weeks. They were not allowed to eat outside of the window. TRE led to weight loss, better body composition, lower blood pressure, and less “bad” fat in the blood.
Metabolic syndrome is associated with heart disease and type 2 diabetes, so TRE is a promising way for people to take action and reduce the likelihood of serious disease development. Metabolic syndrome is not a condition of the lazy. According to Mckinsey hard-working employees with chronic work stress have more than double the risk of metabolic syndrome than unstressed colleagues. This suggests that metabolic syndrome patients should not just diet, but also to take measures to relieve their stress. Gaining control over their work situation is one way that Mckinsey recommends. In addition, exercise is well known to improve health, and according to the Mayo Clinic it also releases endorphins that relieve stress. Better control over their work, diets such as TRE and exercise could be just what hard-working people with metabolic syndrome need to improve their health and wellbeing.
The TRE trial only took place for three months, so longer clinical trials are needed to test effectiveness and safety in the long term. Potential risks include dehydration, the heart “skipping a beat”, and the increased risk of gallstones. It may not be suitable for elderly people.
A new method using DNA can allow almost any item to store data. Product information can be stored, for example, in clothes, bottles, and even prescription drugs, without a conventional label. This is particularly powerful for drugs as it could allow the identification of loose pills and prevent counterfeiting within the pharmaceutical supply chain. The expiration date could also be stored within the pills themselves, eliminating any possible confusion.
The innovation called “A DNA-of-things storage architecture to create materials with embedded memory” was published in the journal Nature Biotechnology with senior authors Yaniv Erlich and Robert N. Grass of Erlich Lab LLC, Israel, and ETH Zürich, Switzerland, respectively. Up to 215,000 terabytes of information can be stored in a single gram of DNA. Data is recorded in DNA molecules using the ATCG code of biology, which are then encapsulated in nanoscale silica (glass) beads. These beads can be embedded in materials, potentially including medications.
According to the World Health Organization (WHO), the counterfeit pharmaceuticals trade is worth up to USD $200 billion per year, with 21% of trade in the Americas. Pharmaceutical companies could use the technology to label their medicines directly with an invisible DNA barcode. This has the advantage that accesses to machinery and reagents that can create a DNA barcode is limited, and many pharma companies already have expertise in DNA. This places counterfeiters at a distinct disadvantage. Given that the DNA has so much storage capacity other important information could also be directly wired into drugs such as the expiration date, information on batch quality, and precise labeling of the chemical composition.
Drug counterfeiting is a serious problem. According to accounting firm PwC in developing regions such as Africa, the proportion of fake pharmaceuticals in some countries is as high as 70%. It endangers patient safety and hits a pharmaceutical company’s bottom line, which ultimately reduces their capacity to develop new medicines, so any measures that can reduce counterfeiting are beneficial.
There is currently no safety information on DNA barcodes and it is likely that the regulatory authorities, including the USA’s Food and Drug Administration (FDA), would require clinical trials before approving their addition to medications. This will incur a considerable amount of cost. It is possible that DNA barcodes will only be feasible for new drugs that can incorporate them into clinical trials from the outset. Another disadvantage is that in order to “read” the barcode the DNA would need to be extracted from the drug, which would ruin the medicine.
Checkpoint inhibitor therapy targets proteins such as PD-1 with approved antibody drugs, which release the immune system to attack tumor cells. Although effective in blood cancers checkpoint inhibitors have not been successful for cancer patients with solid tumors. PAK4 kinase is enriched in non-responding tumors. In a mouse model, PAK4 inhibition improved anti-tumor response compared with anti-PD-1 drugs alone.
A study was carried out in mice by senior author Antoni Ribas of the University of California, Los Angeles, that showed that a drug that inhibits PAK4 improved checkpoint immunotherapy of melanoma tumors. The research team demonstrated that activated PAK4 kinase in tumors was associated with a lack of immune cells within the tumor. These immune cells are needed to fight cancer. This effect was reversed by inhibiting PAK4 with a drug improving tumor clearance.
Solid tumors are notoriously difficult to treat with checkpoint immunotherapy, even though it is effective in blood cancers. There is a search for drugs that can cooperate with checkpoint inhibitors to clear solid tumors. The present study shows that PAK4 may be a valuable target for solid tumor drug development for people, which is much needed.
The findings may not be applicable to people, as the study was carried out in mice and results in such studies are not always reproducible in people. Clinical trials with PAK4 inhibitors will be needed to determine whether such drugs are effective in combination with PD-1 checkpoint inhibitors in people with solid tumors. That said the findings are promising.
Efficient and accurate genome modification is needed to create to make gene therapy more widely available to patients. CRISPR is an essential tool of gene therapy, but the technology still needs improvement. A new polymer-stabilized invention has achieved two improvements in genome editing efficiency using CRISPR-Cas9 in clinically relevant primary cells. Combining the two improvements increased gene targeting efficiency and produced two to six times more viable edited cells.
The innovation called “Polymer-stabilized Cas9 nanoparticles and modified repair templates” was published in the journal Nature Biotechnology by senior author Alexander Marson of the University of California, San Francisco.
CRISPR is a powerful gene-editing technique that has recently entered clinical development. Although accurate and precise, efficiency is a problem. That is the number of cells that are altered or fixed by the technique. For example, if the efficiency of a CRISPR therapy that restores sight and is administered to the retina is 10%, only 10% of the eye will have restored sight. This new invention increases the efficiency up to 6 fold, so in our example, 60% of the eye would have restored sight. Clearly this has great benefits for patients receiving CRISPR gene therapies.
Increased efficiency also carries risk. Although every effort is made to ensure therapy is safe before administering to patients for the first time, the point of a phase I clinical trial is to assess safety, because it is unknown. This means that if for some reason the CRISPR had an undesired effect, the greater efficiency of this new technique would mean that a greater proportion of cells would be affected. That said, all patients that enter clinical trials undergo risk/ benefit assessments to determine whether the trial is right for them, and this new efficiency data will be taken into consideration.
On January 13, Adaptimmune Therapeutics presented SPEAR TCR-T at the 38th JP Morgan Congress. It is a therapy based on the T-cell technology platform. They have positive early data for solid tumor patients. This is important as normally T-cell therapy is not effective in solid tumors.
Adaptimmune is a biotech company focused on developing T-cell Receptor (TCR)-T cell therapies for cancer-specific antigens. The SPEAR T-cell technology platform targets melanoma antigen (MAGE) family proteins, MAGE-A4, MAGE-A10, and fetal protein (AFP). Using this platform, one case of liver cancer and one melanoma patient obtained a confirmed partial response (PR). One case of metastatic gastro-esophageal junction cancer and one head and neck cancer had an unconfirmed partial response.
Like checkpoint inhibitors, T-cell therapies are rarely effective against solid tumors. SPEAR TCR-T is showing promising signs that it is one of the first cell therapies that are effective against solid tumors.
The data is very early stage and comprises more than one therapy, so in actual fact, the data is even more stretched. It remains to be seen whether these are rare responders or indicative of a majority. Only time will tell as more patients are recruited and treated in the clinical trials. T-cell therapies have inherent risks, however, the potential benefit of curing a solid tumor often outweigh them.