Uncategorized
STAT+: Finally cracking KRAS as a druggable target
Want to stay on top of the science and politics driving biotech today? Sign up to get our biotech newsletter in your inbox.
Hiya. Today, we see a proliferation of KRAS, as new data validate a once-undruggable space. Also, a double-edged push for PBM transparency, and the Breakthrough Prize was awarded to researchers for advances in gene therapy and ALS genetics.
The need-to-know this morning
- Novo Nordisk said its experimental oral drug, called etavopivat, reduced the risk of pain crises and improved hemoglobin response in patients with sickle cell disease, achieving the goals of a Phase 3 clinical trial.
- Eli Lilly is reportedly nearing a deal to acquire Kelonia Therapeutics for more than $2 billion, according to the Wall Street Journal. Kelonia is a privately held developer of so-called in-vivo CAR-T therapies that make engineered cancer-killing cells inside the body. Its lead therapy is being investigated in an early stage study in multiple myeloma.
- Helus Pharma, developer of psychedelics-based therapies for depression, said CEO Michael Cola has resigned “at the request of the board of directors.” Chairman Eric So was appointed interim CEO.
Finally cracking KRAS as a druggable target
When Revolution Medicines released results for its drug daraxonrasib in pancreatic cancer patients last week, it finally seemed like there was hope in a condition that has long proven intractable for researchers. But as STAT’s Angus Chen reports, it was the product of years of effort — and could be just the start of a wave of new RAS inhibitors.
Want to stay on top of the science and politics driving biotech today? Sign up to get our biotech newsletter in your inbox.
Hiya. Today, we see a proliferation of KRAS, as new data validate a once-undruggable space. Also, a double-edged push for PBM transparency, and the Breakthrough Prize was awarded to researchers for advances in gene therapy and ALS genetics.
The need-to-know this morning
- Novo Nordisk said its experimental oral drug, called etavopivat, reduced the risk of pain crises and improved hemoglobin response in patients with sickle cell disease, achieving the goals of a Phase 3 clinical trial.
- Eli Lilly is reportedly nearing a deal to acquire Kelonia Therapeutics for more than $2 billion, according to the Wall Street Journal. Kelonia is a privately held developer of so-called in-vivo CAR-T therapies that make engineered cancer-killing cells inside the body. Its lead therapy is being investigated in an early stage study in multiple myeloma.
- Helus Pharma, developer of psychedelics-based therapies for depression, said CEO Michael Cola has resigned “at the request of the board of directors.” Chairman Eric So was appointed interim CEO.
Finally cracking KRAS as a druggable target
When Revolution Medicines released results for its drug daraxonrasib in pancreatic cancer patients last week, it finally seemed like there was hope in a condition that has long proven intractable for researchers. But as STAT’s Angus Chen reports, it was the product of years of effort — and could be just the start of a wave of new RAS inhibitors.
Uncategorized
STAT+: Trump order to advance psychedelic treatments generates excitement — and worries
President Trump’s executive order aimed at loosening restrictions on psychedelics as mental health treatments was largely applauded by advocates. But some also quietly worry the White House’s role in trying to bolster the field risks politicizing it and undermining the credibility of research.
The order, which Trump said originated with a text from podcaster Joe Rogan about psychedelics research, directs the Food and Drug Administration to expedite the review of some compounds and calls for the establishment of a new regulatory pathway for experimental psychedelics to be tried by terminally ill patients. It also allocates funding to states developing research programs.
While the order does not actually reschedule any drugs or change legislation, many advocates and researchers welcomed the move, saying it signals the administration’s interest in advancing psychedelics as treatments and could help ease bottlenecks in expanding access.
President Trump’s executive order aimed at loosening restrictions on psychedelics as mental health treatments was largely applauded by advocates. But some also quietly worry the White House’s role in trying to bolster the field risks politicizing it and undermining the credibility of research.
The order, which Trump said originated with a text from podcaster Joe Rogan about psychedelics research, directs the Food and Drug Administration to expedite the review of some compounds and calls for the establishment of a new regulatory pathway for experimental psychedelics to be tried by terminally ill patients. It also allocates funding to states developing research programs.
While the order does not actually reschedule any drugs or change legislation, many advocates and researchers welcomed the move, saying it signals the administration’s interest in advancing psychedelics as treatments and could help ease bottlenecks in expanding access.
Uncategorized
AACR 2026: Cancers of Unknown Primary Identified by DNA Methylation AI Model
SAN DIEGO – Researchers from Kindai University in Japan have developed a machine learning model that accurately predicts the origin of diverse cancer types in patients with cancers of unknown primary (CUP) by analyzing CpG-based DNA methylation. Results showed that the model correctly identified the cancer type in about 95% of cases in the test cohort, and achieved 87% accuracy when applied to an independent validation cohort from 31 cases representing 17 different cancer types. The work was presented at the American Association for Cancer Research (AACR) Annual Meeting.
“Our findings suggest that DNA-based approaches can help identify where a cancer may have started, even when the original tumor is not visible,” said Marco A. De Velasco, PhD, a faculty member in the department of genome biology at Kindai University in Japan.
CUP are metastatic malignancies in which the primary cancer site could not be identified. These cancers are often associated with poorer outcomes, as patients are typically treated with broad, nonspecific chemotherapy regimens rather than therapies targeted to a specific cancer type.
Approximately only 15-20% of patients with CUP show features that allow site-specific therapies. Patients receiving site-directed therapy can survive up to 24 months, compared with six to nine months for those receiving standard treatment.
Patterns in tumor biology, such as gene activity or chemical modifications to DNA, can differ between cancer types and persist even after the cancer has spread and guide development of these therapies. While some methods have shown promise, they have yet to demonstrate clear survival benefits in clinical trials.
The model was developed using methylation data from nearly 7,500 patients with 21 different cancer types obtained from The Cancer Genome Atlas Program and other public datasets. Using machine learning, the researchers identified CpG methylation and built methylation profiles that were associated with different tumor types.
Del Velasco emphasized that the study achieved high accuracy in predicting the origin of diverse cancer types using a small subset of DNA markers, about 1,000 CpG regions selected from hundreds of thousands across the genome. “This is important because it shows that we can simplify complex molecular data while still maintaining strong predictive performance,” he said.
As a limitation, the model was developed using cancers with known origins, rather than true CUP. Testing in CUP patients is important to understand how well the model performs in clinical settings. Additionally, not all tumors are easily accessible for genetic testing, particularly tumors in advanced stage. Looking ahead, the authors aim to adapt and evaluate the model using blood-based biopsy to analyze circulating tumor DNA instead of relying on DNA from tissue samples.
The post AACR 2026: Cancers of Unknown Primary Identified by DNA Methylation AI Model appeared first on GEN – Genetic Engineering and Biotechnology News.
Uncategorized
Viatris recalls extended-release Xanax over dissolution test failure
Viatris, which Pfizer created in 2020, voluntarily withdrew extended-release products made at a plant in Ireland after an analysis revealed an issue that could affect bioavailability.
-
Uncategorized9 years agoThese ’90s fashion trends are making a comeback in 2017
-
Contributors9 years agoThe final 6 ‘Game of Thrones’ episodes might feel like a full season
-
Uncategorized9 years agoAccording to Dior Couture, this taboo fashion accessory is back
-
Uncategorized9 years agoPhillies’ Aaron Altherr makes mind-boggling barehanded play
-
Uncategorized9 years agoUber and Lyft are finally available in all of New York State
-
Uncategorized9 years agoThe old and New Edition cast comes together to perform
-
Uncategorized9 years agoSteph Curry finally got the contract he deserves from the Warriors
-
Uncategorized9 years agoDisney’s live-action Aladdin finally finds its stars