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A clinical trial of rebecsinib, a first-in-class investigational drug that inhibits the ADAR1 gene involved in the proliferation of more than 20 cancers, is underway at UC San Diego, offering the rebecsinib clinical trial blood cancer program as a new option for patients with few remaining choices.
Who the Rebecsinib Clinical Trial Blood Cancer Program Currently Serves
The first patient was treated July 6, according to principal investigator James Mangan, MD, PhD, professor of medicine at UC San Diego School of Medicine and a hematologist and oncologist at UC San Diego Health. He called the drug “promising.” “This trial has great science behind it,” Mangan said. “It uses a totally novel mechanism and really is for patients who have a desperate, unmet need.”
Trial Eligibility and Sponsorship
The Phase 1 clinical trial, sponsored by Aspera Biomedicines, is open to adults 18 years of age and older who have secondary acute myeloid leukemia that has either recurred or not responded to treatment. It’s also open to patients with higher-risk myelofibrosis. Both are rare blood cancers for which few treatment options exist initially, and no good options if they return.
The Scientist Behind This Rebecsinib Clinical Trial Blood Cancer Discovery
UC San Diego Sanford Stem Cell Institute Director Catriona Jamieson, MD, PhD, a hematologist and researcher who discovered the drug, said she is “thrilled to take it from bench, and a bench on the International Space Station, no less, to the bedside of patients who need it most.” “Rebecsinib shows all the promise in the world not only to halt the progression of multiple cancers, but to shrink them, as well as prevent their spread to multiple sites in the body,” she added.
A Drug Tested in the Cosmos
The FDA green-lit rebecsinib for clinical trial in March of last year, making it the first and only ADAR1 inhibitor with an investigational new drug application. It’s one of the very first drugs studied in space. Jamieson has sent multiple research payloads to the ISS, testing the drug on various types of highly lethal cancers with ADAR1 involvement, including ovarian cancer, metastatic breast cancer, AML, and glioblastoma multiforme, experiments made possible by millions in grants from NASA’s In-Space Production Applications program.
Why Space Research Accelerated This Rebecsinib Clinical Trial Blood Cancer Timeline
Scientists are still learning about the distinctive properties of space that threaten human health, including microgravity and galactic cosmic radiation. Such conditions create a uniquely stressful environment that mimics an accelerated version of aging and disease progression on Earth. Depending on the experiment, one month in microgravity can give researchers a preview of a few years, if not more than a decade, of maturation on Earth.
What Space Research Revealed About ADAR1
The landmark NASA Twins Study of 2015-2016 found that space can affect the immune system, gut bacteria, body weight, serum metabolites, gene expression and cognition of astronauts, among other health factors. Jamieson’s research found that space also activates ADAR1, which, in turn, produces ADAR1p150, a protein that promotes tumor growth by hiding cancer from the immune system. “Space gives Dr. Jamieson a tremendous chance to see a lot of changes in stem cell DNA in a short period of time,” Mangan said.
Broader Applications of This Rebecsinib Clinical Trial Blood Cancer Research
Rebecsinib’s June clinical trial launch is only the beginning. The trial may eventually expand to other ADAR1-involved cancers, including lymphoma, glioblastoma multiforme, and metastatic breast cancer. Mangan also noted that the drug could serve as “a therapeutic mechanism to restore stem cell function after space travel” for astronauts, with potential applicability to the accumulated cellular stresses humans experience over a lifetime on Earth.
What Patient Advocates Are Saying
Andrew Schorr, 75, who has lived with myelofibrosis and chronic lymphocytic leukemia for decades, said his myelofibrosis is relatively stable at the moment, but if that were to change, rebecsinib “might be another option, and I would be grateful.” “Every patient needs hope for what could be their next treatment, because these drugs peter out,” he said. “Cancer finds a way around them.”
A Patient’s Journey Through the Rebecsinib Clinical Trial Blood Cancer Landscape
Sandra Dillon, 50, was diagnosed with an aggressive form of myelofibrosis in 2006 and spent 13 years taking fedratinib, a drug Jamieson played a prominent role in reviving when the FDA placed it on clinical hold. “The side effects I experienced were so minimal that it was like taking Advil for a headache,” Dillon said. “Fedratinib really fought back the disease in an amazing way.”
When Treatment Options Changed
More recently, however, her disease “broke out of its chains and started getting really, really bad,” Dillon said. She had hoped to participate in the rebecsinib trial but wasn’t able to wait, and underwent a bone marrow transplant last fall before the trial was underway. “When I think about rebecsinib, I think of it as that next stage, if one medication stops working, there are other options that could be effective for this very rare blood disease.”
Could Rebecsinib Represent a New Model for Drug Development?
Schorr is excited to witness rebecsinib’s story arc, having watched other cancer drugs, like Keytruda, rise to greatness over the years. “It meets an unmet need and will hopefully extend lives,” he said, adding that it could play an even more significant role in cancer medicine if it serves as proof of concept for expediting the path to clinical trial by gathering data in outer space.
Rapid Progress From Lab to Clinic
Mangan said cancer centers “across the U.S. are very excited about opening this trial.” “I’m just really enthusiastic about the science behind this drug,” he said. “To take it from a basic science paper published in Cell Stem Cell in 2023 to first-in-human Phase I clinical trial in just three years is a tremendous effort.”
What This Rebecsinib Clinical Trial Blood Cancer Program Means Going Forward
With the trial’s first patient treated in July and cancer centers nationwide expressing interest in opening the study, this rebecsinib clinical trial blood cancer program represents a rare instance of space-based research translating directly into a first-in-human oncology trial within just three years of the underlying basic science publication. Given the drug’s potential relevance to more than 20 ADAR1-involved cancers, its eventual expansion beyond AML and myelofibrosis into cancers like lymphoma, glioblastoma multiforme, and metastatic breast cancer could significantly broaden its impact if early results prove promising.
What to Watch Going Forward
As the Phase 1 trial continues enrolling patients with secondary AML and higher-risk myelofibrosis, researchers, clinicians, and patient advocates will likely watch closely for early safety and efficacy signals that could support expanding the trial to additional cancer types. Given the novel space-based research pathway that helped bring rebecsinib to clinical trial in an unusually short timeframe, this program may also serve as a test case for whether microgravity research can meaningfully accelerate drug development timelines for other hard-to-treat cancers in the future.
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