On February 7, 2020 An innovative approach to tackling the challenges of pediatric sarcomas is being pursued by researchers at St. Jude Children’s Research Hospital (Memphis, Tennessee) with a new grant from Alliance for Cancer Gene Therapy (ACGT) (Press release, ACGT Alliance For Cancer Gene Therapy, FEB 7, 2020, View Source [SID1234554033]).

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The $500,000 ACGT grant supports Stephen Gottschalk, MD, chair of Bone Marrow Transplantation and Cellular Therapy at St. Jude’s, in exploring the use of genetically engineered immune cells to attack pediatric sarcoma. Dr. Gottschalk and his team target not only the cancer, but the blood vessels supporting tumor growth. Dr. Gottschalk has conducted more than 25 previous clinical trials and most recently worked on gene therapies for treating infants with the "bubble boy" disease.

"There is an urgent need to develop meaningful treatments for children with solid tumors," says Kevin Honeycutt, CEO and president of ACGT. "Dr. Gottschalk’s research combines state-of-the-art technologies and methodologies to attack two targeted gene proteins found in pediatric sarcomas. The members of the ACGT Scientific Advisory Council are enthusiastic about Dr. Gottschalk’s vision and very promising preliminary data."

"Pediatric sarcoma, which comes back after initial therapy or is present at multiple sites, is difficult to treat even with the most aggressive, currently available therapies," noted Dr. Gottschalk. "Because Sarcomas are a relatively uncommon group of cancers, accounting for about 15 percent of childhood cancers, improving outcomes has been challenging. This ACGT grant enables me to further investigate how we can leverage different proteins to attack not only abnormal sarcoma cells but also the blood vessels that provide nutrients to tumors. We’ve seen this approach work in mice. Given the resources from ACGT, we can move ahead much faster to complete all the preclinical studies that are needed to evaluate our approach in the clinic."

Dr. Savio Woo, chairman emeritus of the ACGT Scientific Advisory Council and recently retired from the Icahn School of Medicine at Mount Sinai (New York, New York), dedicated his career to the pursuit of fundamental science and technology development in gene and cell therapy, and translating those laboratory advances into direct patient benefit. Dr. Gottschalk, who did a post-doctoral session with Dr. Woo from 1992 to 1995, acknowledges that it was Dr. Woo’s commitment to ACGT that drove his quest to secure a grant from the organization.

"Being connected to the organization that Dr. Savio Woo helped direct on the scientific side, is a great honor," said Dr. Gottschalk.

To learn more about Alliance for Cancer Gene Therapy (ACGT), visit acgtfoundation.org, call 203-358-5055, or join the ACGT community on Facebook, Twitter, Instagram and YouTube. To learn more about St. Jude Children’s Research Hospital, visit stjude.org or follow St. Jude on social media at @stjuderesearch.

On February 7, 2020 Eli Lilly and Company (NYSE: LLY) reported that it will participate in the Guggenheim Healthcare Talks Idea Forum on Thursday, February 13, 2020 (Press release, Eli Lilly, FEB 7, 2020, View Source [SID1234554032]). Jacob Van Naarden, chief operating officer for Loxo Oncology at Lilly; Eric Dozier, vice president, Lilly Oncology North America; and Maura Dickler, M.D., vice president, oncology late phase development, will participate in a fireside chat at 1:00 p.m., Eastern Time.

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A live audio webcast will be available on the "Webcasts & Presentations" section of Lilly’s Investor website at View Source A replay of the presentation will be available on this same website for approximately 90 days.

On February 7, 2020 CytomX Therapeutics, Inc. (NASDAQ:CTMX), a clinical-stage oncology-focused biopharmaceutical company pioneering a novel class of investigational antibody therapeutics based on its Probody therapeutic technology platform, reported that on February 3, 2020, the Company granted its recently appointed senior vice president and chief medical officer, Alison L. Hannah, M.D., an option to purchase 250,000 shares of the Company’s common stock at an exercise price per share equal to $7.71, which was the closing trading price on February 3, 2020, the date of the grant (Press release, CytomX Therapeutics, FEB 7, 2020, View Source [SID1234554031]). The Company also granted Dr. Hannah a performance option to purchase 50,000 shares of the Company’s common stock at an exercise price per share equal to $7.71. The performance option vests upon the accomplishment of certain clinical trial related goals established by the Company.

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The stock options were granted pursuant to the Company’s 2019 Employment Inducement Incentive Plan, which was approved by the Company’s board of directors in February 2020 under Rule 5635(c)(4) of The Nasdaq Global Market for equity grants to induce new employees to enter into employment with the Company.

On February 7, 2020 Shanghai Junshi Bio reported that invested $1.4 million in Stemirna Therapeutics, a Shanghai company developing mRNA vaccine therapeutics for cancer (Press release, Shanghai Junshi Bioscience, FEB 7, 2020, View Source [SID1234554030]). Junshi, which made the investment as part of an A+ round, acquired an 3% stake in Stemirna. The two companies will jointly develop novel drugs and combination therapies Formed in 2016, Stemirna identifies tumor-specific antigens, loading them onto a single mRNA strand. Junshi, which has 13 molecules in development, was the first China company approved to market a PD-1 candidate in China, Tuoyi.

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On February 7, 2020 Researchers with the Abramson Cancer Center of the University of Pennsylvania, led by Carl June, reported that results from the first U.S. Phase I trial of CRISPR-Cas9-edited T-cells in humans with advanced cancer. The data was published in the journal Science (Press release, University of Pennsylvania, FEB 7, 2020, View Source [SID1234554028]).

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The trial involved three patients with refractory cancer, two women and one man, all in their 60s. One of the patients had sarcoma and two had multiple myeloma. The approach was similar to that seen in CAR-T therapy, where the patient’s own T-cells are recovered, engineered to express a specific receptor that can detect and kill cancer cells, then reinfused into the patient.

In the case of this trial, instead of engineering the T-cells with a receptor to a protein like CD19, they used CRISPR to remove three genes from the T-cells. Two edits removed the T-cell’s natural receptors, which could then be reprogrammed to express a synthetic T-cell receptor called NY-ESO-1. The third edit eliminated PD-1, a checkpoint receptor that allows cancer cells to hide from T-cells.

The researchers are presenting the data as a positive because it appears to be safe. June told Genetic Engineering & Biotechnology News, "CRISPR technology has proven safe in patients with advanced refractory and metastatic cancer. Our results demonstrate the ability to precisely edit the DNA code at three different genes."

In an accompanying article, Jennifer Hamilton and CRISPR pioneer Jennifer Doudna wrote, "These findings provide a guide for the safe production and non-immunogenic administration of gene-edited somatic cells. The clinically validated long-term safety of CRISPR-Cas9 gene-edited cells reported [here] paves the way for next-generation cell-based therapies."

Before getting overly excited about this, it was also reported that one of the patients has since died and the disease became worse in the other two. June indicated the goal of the study wasn’t to cure cancer, but to show that the CRISPR technique was feasible and safe.

With that goal in mind, it’s safe to say the trial was a success.

"This is a Rubicon that has been decisively crossed," said Fyodor Urnov, a genome editor at the University of California (UC), Berkeley, in a Science article. He noted the trial was the first of its kind in the U.S. and answered "questions that have frankly haunted the field."

The research also suggests what the limitations of the approach are, at least currently.

One of the big concerns in using CRISPR is off-target edits. CRISPR is generally pretty precise, but the human genome is quite larger and even a target of 20 or so specific nucleotides in a gene might be duplicated elsewhere, which could have unintended effects. And, studies of the three patients in the study confirmed that CRISPR had resulted in some off-target edits. There weren’t many and the number of cells affected decreased over time.

There have also been questions on how long gene edits last. In theory, they should last indefinitely, but some research has suggested the body tries to fix the edits and return them to their original state. However, this study showed the CRISPR-edited cells continued at least nine months, which is significant compared to about two months in similar CAR-T therapeutic studies.

So this study, which is significant, is more of a starting point for CRISPR-based therapies, particularly given the modest clinical response.

"It wasn’t like you turned off those genes and those T-cells started doing things that were amazing," Antoni Ribas, a UC Los Angeles oncologist told Science. But it was "a needed start" and going forward, "It’s going to be easier—because they did it first."