ASH: UPenn's CRISPR-edited immune cells prove safe in small cancer study

This is a photo of the Clinical Research Building of the Perelman School of Medicine at the University of Pennsylvania in Philadelphia, PA
University of Pennsylvania researchers are reporting early results from a trial of CRISPR-edited immune cells in fighting cancer. (Hazmat2)

In 2016, Facebook and Napster billionaire Sean Parker gained a lot of attention for his promises about “hacking cancer” and bringing “disruption” to life sciences research. But it was more than just talk: The internet pioneer put up $250 million to launch the Parker Institute for Cancer Immunotherapy, which then became the first research institution to get a thumbs-up from the National Institutes of Health (NIH) to launch a human trial of an immuno-oncology therapy made from the gene-editing technology CRISPR-Cas9.

Now the researchers running that trial are providing some hints of how it’s going so far. At the American Society of Hematology (ASH) annual meeting in Orlando, the team, led by the Abramson Cancer Center of the University of Pennsylvania, will discuss two multiple myeloma patients and one sarcoma patient who are participating in the trial, which is also supported by Tmunity Therapeutics.

According to an abstract of the study released Wednesday, the CRISPR treatment was well tolerated. The gene-edited cells the patients received expanded and traveled to the tumors they were meant to treat. And the three patients, all of whom had failed previous treatments, are still alive nine months following their cell infusions, though it’s too early to assess their overall response.

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RELATED: Facebook, Napster billionaire Parker to fund first-ever CRISPR trial

The technology at the heart of the trial involves using CRISPR to make three edits in T cells that are collected from patients. One of the edits eliminates PD-1, a “checkpoint” that prevents immune cells from being able to recognize and attack cancer. The other two snips remove parts of T cells that would normally prevent them from attaching to cancer cells.

Then a virus is used to insert an “affinity-enhanced T cell receptor (TCR),” which instructs the T cells to target a particular antigen on cancer cells, according to a statement.

The University of Pennsylvania was a pioneer of CAR-T technology, and research there led to the development and commercialization of Kymriah, Novartis’ blood cancer treatment, which also involves removing immune cells from patients and engineering them to recognize and attack cancer. But the cells used in this trial are different.

CAR-T cells are armed with receptors like CD19, which makes them active in most patients with cancers that are driven by those receptors. CRISPR-edited T cells, by contrast, are only active in the presence of HLA-A201, an antigen that’s overly abundant in some cancer patients. For that reason, patients have to undergo testing for HLA-A201 over-expression before they can receive the treatment.

The ASH presentation is the latest news from the Parker Institute, which has funded immuno-oncology research at a variety of academic institutions and companies. The organization’s other recent cell-therapy endeavors include forming a pact with Xyphos Biosciences to develop next-generation CAR-T therapies and participating in an $85 million funding of ArsenalBio, which also plans to use CRISPR to develop cell therapies.

The Penn trial will include 18 patients with multiple myeloma, synovial sarcoma or myxoid/round cell liposarcoma and is expected to run through 2022. The researchers plan to provide further details about the function of the cells and the clinical outcomes they’ve observed so far on December 7 at the ASH conference.

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