Sanford Stem Cell Institute Brings Space Health Emphasis to Precision Medicine World Conference

Both were nominated by Jamieson, who chaired a daylong RNA, stem cell- and gene-based therapies track, bringing a unique space-health perspective to the event. The track featured presentations from three other institute directors: Komor; Gene Yeo, PhD, SSCIC director; and Alysson Muotri, PhD, director of SSCI’s Sanford Integrated Space Stem Cell Orbital Research Center.

Jamieson said she couldn’t be prouder of Komor — one of the most promising researchers in the field of biotechnology — and called both recipients “architects of the future of precision medicine.”

"To say she is a rising star would be a gigantic understatement,” Jamieson said of Komor. “She has base editing under her belt, so to speak, and so much of her career ahead of her. She’s already doing incredible things. I can’t wait to see what’s next for her — and for all of us, who will surely benefit from her brilliance.”

Faulkner’s work has “empowered patients and made their data meaningful,” Jamieson added. “Her work in developing Epic and MyChart have infinitely increased the impact of precision medicine.”

The RNA Revolution: Beyond mRNA

Komor chaired a panel on RNA therapeutics beyond messenger RNA (mRNA), large molecules that carry instructions from DNA to the body’s protein-making machinery. Scientists have used mRNAs to create drugs and vaccines, most notably the COVID-19 vaccine. Additionally, mRNA is used to deliver DNA base editing to treat various diseases.

Panelists included Yeo, who articulated the importance of RNA as a “therapeutic goldmine.” His lab studies RNA biology, identifies RNA targets and devises ways to manipulate RNA. But “RNA is never naked,” as Yeo pointed out. “It’s clothed, so to speak, by RNA binding proteins, which 20% of the human genome encodes for.”

To manipulate RNA to treat diseases, one must understand how to use antisense oligonucleotides (ASOs), single strands of nucleic acid that can change protein production to correct gene expression, potentially treating disease; small interfering RNA (siRNA), double-stranded RNA molecules that can silence specific disease-causing genes; and small nuclear RNAs (snRNAs), which can be engineered to correct faulty, disease-causing gene splicing, Yeo said. He later chaired a session with Mark Kay, MD, PhD, of Stanford Medicine, and David Schaffer, PhD, of the University of California Berkeley, on in-vivo gene therapies, emphasizing delivery as the key feature determining the success of adeno-associated viral (AAV)-based therapies.

‘Twin Crises’ Illuminated by Space

In remarks throughout the day, Jamieson discussed her space-based research on stem cell aging, pre-cancer and cancer, which builds on the landmark NASA Twins study. She focused on the “dual crises” of cancer relapse and stem cell exhaustion, and a shared culprit: the activation of ADAR1p150, an RNA-editing enzyme that can “switch on” more than 20 types of cancer, including brain, breast, small bowel, pancreatic, liver, cervical, lung adenocarcinoma, testicular and uterine.

“Cancer relapse is responsible for nearly 90% of all cancer deaths — and cancer stem cells are the hidden driver of it,” Jamieson said. “Most anti-cancer therapies debulk the tumor’s tip but spare its roots — cancer stem cells — leading to relapse.”

A parallel crisis, according to Jamieson: stem-cell aging. “Stress, inflammation and radiation wear down regenerative cells, accelerating aging, neurodegeneration and immune decline,” she added.

Jamieson also led a session on stem-cell targeted therapeutics, including small-molecule drugs and biologics — complex precision medicines derived from living organisms rather than chemistry. She discussed her discovery of rebecsinib, a first-in-class small-molecule drug that targets ADAR1, the gene that produces ADAR1p150, to stop the growth of therapy-resistant cancer stem cells. The drug — the first ADAR1 inhibitor to receive FDA investigational new drug approval, based partially on data collected from tumor organoids aboard the ISS (International Space Station) — is slated to enter clinical trials later this year.

Along similar lines, Muotri gave a presentation about the study of the human brain in space, which he accomplishes by launching research “payloads,” or projects, to the ISS. Each payload contains hundreds of brain organoids — miniature brains, so to speak, no bigger than the tip of a pen, created from the reprogrammed induced pluripotent stem cells of consenting patients, obtained via skin or blood cells.

“Growing human brain organoids in space can teach us about the impact of astronauts’ cognition,” said Muotri, whose work has elucidated the concept of “space-induced neural senescence" — a term he coined after observing neurotypical brain organoids that spent time in space. His space-based work has also led to the discovery of how Rett syndrome, a rare genetic disorder, might cause symptoms. Because of his discovery, patients with the condition are being treated with an inexpensive repurposed medicine in clinical trials, with the hope that it improves their cognitive abilities.

Jamieson closed the event with the following thought: “We were thrilled to bring a unique perspective on regenerative medicine to such a prominent stage of leaders in the field. This is the conference to be at, if you work in precision therapeutics. “With 18 launches of stem cell-based research projects to the ISS so far, we had much to share.”