Pulse 138: Clinical Trial Validates CRISPR Cancer Immunotherapy

10 February 2020
Giulio Prisco

Doctor Holding CRISPR DNA

The highly anticipated results of a small clinical trial (see below) show that treating cancer patients with a CRISPR-engineered version of their own immune cells is both safe and feasible. While no patient was cured, the feasibility and safety of the approach “hold significant promise for the future of CRISPR-based medicines.”

“Before we did this, no one had ever infused CRISPR-edited cells into patients, and we’re encouraged by the fact that we could do it safely,” says researcher Edward A. Stadtmauer, as reported by Wired. “Now we can move on to a whole new frontier of further engineering these cells and expanding the number of patients treated.”

“Our data from the first three patients enrolled in this clinical trial demonstrate two important things that, to our knowledge, no one has ever shown before,” says research leader Carl June in a Penn Medicine press release. “First, we can successfully perform multiple edits with precision during manufacturing, with the resulting cells surviving longer in the human body than any previously published data have shown. Second, thus far, these cells have shown a sustained ability to attack and kill tumors.”

CRISPR Immune Cells Thrive in Cancer Patients

Researchers at University of Pennsylvania, in cooperation with researchers at Stanford University and Tmunity Therapeutics, have shown that genetically-edited immune cells can persist, thrive, and function months after a cancer patient receives them.

The results of a clinical trial, published in Science, indicate that cells removed from three patients, CRISPR-engineered in the lab, and administered back to the patients, were able to kill cancer months after their original manufacturing and infusion.

Further analysis of these cells confirmed they were successfully edited in three specific ways. This marks the first-ever sanctioned investigational use of multiple edits to the human genome.

Improved Vision in Blind Mice After Gene Therapy

Mice born blind have shown significant improvement in vision after undergoing a new gene therapy developed by scientists at Tohoku University.

In a research paper published in Nature Communications, the scientists describe a gene therapy platform that allows local replacement of mutated genes. The healthy counterparts are delivered by a viral vector (adeno-associated virus, or AAV), which can treat almost any mutation.

The scientists used CRISPR-Cas9 technology, and plan to use the new gene therapy platform to develop a therapy for human patients with retinitis pigmentosa.

Turn Off Inflammation and Age-Related Conditions

Scientists at UC Berkeley have identified a molecular 'switch' that controls the immune machinery responsible for chronic inflammation in the body.

A study published in Cell Metabolism shows that a type of immune proteins, responsible for sensing potential threats to the body and launching an inflammatory response, can be essentially switched off by removing a small bit of molecular matter.

The findings could lead to new ways to halt or even reverse many age-related conditions, from Alzheimer's and Parkinson's to diabetes and cancer.

Biofactories Produce Medicine in Isolated Locations

Chemical engineers at UT Austin and University of Washington have developed a new way to produce medicines and chemicals on demand and preserve them using portable ''biofactories'' embedded in water-based gels called "hydrogels."

The approach is described in a research paper published in Nature Communications.

It could help people in remote villages or on military missions, where the absence of pharmacies, doctor's offices, or even basic refrigeration makes it hard to access critical medicines, daily use chemicals, and other small-molecule compounds.

Handheld Device 3D-Prints to Heal Burn Wounds

Researchers at University of Toronto Engineering and Sunnybrook Hospital have developed a new handheld 3D printer that can deposit sheets of skin to cover large burn wounds and accelerate the healing process. The bio-ink dispensed by the 3D printer is composed of stem cells that differentiate into specialized cell types depending on their environment.

In a research paper published in Biofabrication, the scientists report successful in-vivo trials on full-thickness wounds.

Evolution Explains Why Cancer Isn’t More Common

Scientists at Osnabrück University and Santa Fe Institute have applied evolutionary biology toward a big-picture understanding of cancer. According to the “handicap principle,” evolutionary systems are stabilized against "cheaters" when dishonest signals are costlier to produce (need more energy) than the benefit they provide.

In a research paper published in Scientific Reports, the scientists argue that, by the handicap principle, a cancer cell is a cheater that can't afford to signal for attention.

This could explain how our bodies have evolved to keep us from getting more cancer. If cancer cells could afford to signal, cancer would be much more common.

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