This Week In Science (February 3-9, 2020)

1.)  CRISPR Gene Editing Takes a Major Step Forward

In a study in Science, researchers announced that they had successfully used CRISPR CAS9 gene editing to edit T-cells in 3 cancer patients. twp gees that encoded for T Cell receptor chains were deleted in order to enhance the expression of a cancer-specific receptor chain. A third gene was removed to improve anti-tumor immunity. CRISPR was successful at editing the genes in all three patients, with the modified T cells persisting for up to 9 months. Most important, the immune response to the gene-editing was minimal. Cytokine release syndrome has been a major impediment to CRISPR technology being deployed, but all 3 patients in the study did not experience any overt side effects related to the therapy. Clinical response to the treatment resulted in two patients showing a stable cancer progression, while the 3rd had a ~50 decrease in cancer mass that was sustained for four months. The improvement was not permanent, and the disease continued to progress in all 3 patients as the modified t-cell count decreased.

It is hoped that CRISPR gene editing will allow for the treatment of diseases like inherited blood disorders, blindness, chromosomal disease, and even cancer. With these results, CRISPR becomes one step closer to reality.

2.) Major Clinical Trial Launched for Parkinson’s Disease

Alkahest Inc, announced that they were launching a new phase 2 clinical trial using AKST4290, This medication inhibits the expression of the C-C chemokine receptor type 3 gene that is thought to be behind the accumulation and activation of inflammatory cells. The trial is funded in part by The Michael J. Fox Foundation for Parkinson’s Research (MJFF). It is hoped that AKST4290 will improve motor function allow for a better quality of life in patients with Parkinson’s disease.

Karoly Nikolich, CEO of Alkahest, stated that “Evidence suggests that the immune system plays an important role in the development and progression of Parkinson’s disease, a disease which impacts nearly one million Americans, AKST4290 suppresses immune-mediated pathways and chronic inflammation and, if effective, could represent an important step forward in the treatment of Parkinson’s disease. We are thrilled to have the support of The Michael J. Fox Foundation, the world’s largest nonprofit funder of Parkinson’s research, in advancing this new program.”

Alkahest is currently in clinical trials for other utilizations of this medication, with promising results being reported for the treatment of neovascular macular degeneration.

3.) Researchers Announce Brain Implant that Allows for Rudimentary Vision

Bernardeta Gómez wearing the glasses that fed information to the implant.

MIT Technology Review announced the results of a 2018 study where a blin patient was given a 100 electrode brain implant that was wired into the visual cortex. Using the brain imp-lant, the patient was able to identify lights, letters, basic shapes, and even able to play a low-resolution Pac-man like game.

Lead researcher Eduardo Fernandez, director of neuro-engineering at the University of Miguel Hernandez, stated that his goal was to “return sight to as many as possible of the 36 million blind people worldwide.” This approach is particularly exciting because it bypasses the eye and optical nerve. Many previous biomechanical approaches to curing blindness were directed at the eye, and the eye’s ability to sense light. However, a large percentage of blind people are not able to benefit from these treatments because of optical nerve damage. This approach of beaming the information straight into the brain allows for a more universal treatment.

While it is still several years away from widespread implementation, this is one more sign of the advancement of treating blindness, and our ability to directly manipulate the brain.

4.) Scientists Discover Virus that has Zero Recognizable Genes

A new virus by the name of Yaravirus was discovered in an artificial lake in Brazil. The researchers were searching for so-called “giant viruses” when they uncovered one that was even more strange. This virus was much smaller than a giant virus but shared some of the same morphology. Scientists were able to isolate the virus as one that infected amoebas in the lake.

The strangest thing about the virus was that it contained no genes that could be identified. Other viruses normally have a gene that while not identical, is still highly related to genes that have already been mapped in other organisms. but this was not the case for Yaravirus.  To find these viruses, the researchers isolated viral particles from dozens of tissue samples from humans and other animals and screened them for circular genomes.

The group confirmed that the DNA belonged to viruses by looking for a gene that codes for a virus’s shell. Viruses remain one of the most unknown of the microscopic world. New genes may provide researchers new approaches and information on how to deal with a variety of human issues.

5.) First clinical trial of an Automated Blood Drawing and Testing Device

A prototype of an automated blood drawing and testing device.
Photo: Unnati Chauhan

An article in the journal Technology announced the results of a robotic blood drawing device that exceeded clinical standards. The robot was able to isolate veins and successfully draw blood 87% of the time. When narrowed down to patients with accessible veins, the robot was able to succeed 97% of the time.

This compares to a normal clinical failure rate of 27%  of patients without visible veins, 40% of patients without palpable veins and 60% of emaciated patients. The robotic device uses ultrasound images to guide a robot to draw the blood and also features an integrated centrifuge-based blood analysis device. Blood draw failures present major medical complications.

Repeated failures dramatically increase the likelihood of thrombosis, infection, and phlebitis. Numerous clinicians are also injured each year when performing a blood draw procedure. As a result, venipuncture is among the leading causes of injury to patients and clinicians. Blood draw failures can also utilize large amounts of staff time and medical supplies, leading to a loss of more than $4 billion dollars each year.

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