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Earlier HCT improves survival for youth with Shwachman-Diamond Syndrome

A multi-center international study published in NEJM found that performing hematopoietic cell transplants shortly after detecting high-risk features in patients with Shwachman-Diamond Syndrome significantly improves survival rates. The study analyzed 847 cases and found that survival appeared to improve when early HCT treatment occurre...

SourceCincinnati Children's Hospital Medical Center·JournalNew England Journal of Medicine·TypeMeta-analysis·DateSep 23, 2026

New method causes cancer cells to burn out

Researchers developed a new treatment for acute myeloid leukemia (AML) by using AcTor, a molecule that inhibits a signalling protein, in combination with ixazomib. The treatment successfully eliminated cancer cells and leukemic stem cells, showing promising results in mice studies.

SourceUniversity of Gothenburg·JournalMolecular Cancer·TypeExperimental study·DateSep 8, 2026

Mass General Brigham-led trial shows a less-intensive drug combination is more effective in treating AML

A phase 2 clinical trial found that a less-intensive combination of azacitidine and venetoclax was more effective in treating acute myeloid leukemia, with patients tolerating treatment better and spending less time in the hospital. The combination also led to more patients reaching stem cell transplantation.

SourceMass General Brigham·JournalNew England Journal of Medicine·TypeRandomized controlled/clinical trial·DateSep 2, 2026

Immune cells use previously unknown pathway to eliminate AML

Scientists at MD Anderson Cancer Center have identified a previously unknown mechanism by which T cells attack and eliminate acute myeloid leukemia (AML) cells. The discovery reveals that AML cells use a CD64-dependent pathway to evade traditional MHC recognition, potentially explaining why AML is sensitive to immune-based treatments.

SourceUniversity of Texas M. D. Anderson Cancer Center·JournalProceedings of the National Academy of Sciences·DateJul 15, 2026

Epigenetic therapy shows promise against treatment-resistant AML

Researchers discovered a new epigenetic therapy that remained effective in treatment-resistant acute myeloid leukemia (AML) through activating the Hippo pathway, a tumor-suppressing pathway linked to cancer growth and drug resistance. The therapy, NTX-301, consistently reduced leukemia cell survival more effectively than existing hypom...

SourceUniversity of Texas M. D. Anderson Cancer Center·JournalClinical Cancer Research·DateJul 13, 2026

Researchers discover how acute myeloid leukemia invades the lungs and which pathways could halt its infiltration

Acute myeloid leukemia transforms lung tissue into an inflammatory environment that promotes tumor cell expansion and impairs respiratory function. Researchers identified galectin-9 and the IL-33/IL1RL1 axis as potential targets for reducing this serious complication.

SourceInstitut de Recerca Sant Pau (Sant Pau Research Institute)·JournalNature Immunology·TypeExperimental study·DateJun 30, 2026

The University of Colorado Anschutz Gates Institute achieves first-in-U.S. FDA clearance for campus-built CAR T-cell therapy

The University of Colorado Anschutz Gates Institute has achieved first-in-U.S. FDA clearance for a novel CAR T-cell therapy targeting aggressive leukemia cells, representing a potential new treatment approach for patients with hard-to-treat disease. The therapy will be evaluated in a Phase 1 clinical trial starting this summer.

Leukemia study restores silenced gene in mice. Could it point to new treatments for humans?

Researchers created a CRISPR-based tool to pinpoint genes the cancer turns off, restoring a key cancer-fighting gene in leukemia. The study found that blocking KDM4 enzymes can regain expression of the tumor-suppressor gene ZBTB7A, reducing leukemia burden while leaving normal blood formation largely unaffected.

SourceJackson Laboratory·JournalScience Translational Medicine·TypeExperimental study·DateApr 2, 2026

Agent Orange linked to aggressive bone marrow cancer in Vietnam veterans

A new national study highlights the genetic changes that link exposure to Agent Orange to myelodysplastic syndromes, a group of bone marrow cancers that can progress to acute leukemia. The research found that exposed veterans were diagnosed at a younger age and had a higher rate of disease progression compared to unexposed patients.

RELAX study from Dresden: Innovative combination therapy shows promising efficacy in aggressive leukemia

Researchers from Dresden University Medicine developed a new treatment approach for relapsed acute myeloid leukemia (AML), combining standard therapy with the BCL2 inhibitor venetoclax. The innovative combination therapy has shown promising efficacy, increasing remission rates to 75% and improving patients' chances of recovery.

SourceTechnische Universität Dresden·JournalThe Lancet Haematology·DateMar 5, 2026

Black patients diagnosed with aggressive leukemia experience worse outcomes, but differences not linked to key genetic changes

A new study by University of Maryland researchers found that Black patients with aggressive leukemia have lower survival rates and are more likely to die from the disease compared to white patients. The disparity is attributed to younger age at diagnosis rather than genetic differences, according to Dr. Shella Saint Fleur-Lominy.

ASH 2025: Study connects Agent Orange exposure to earlier and more severe cases of myelodysplastic syndrome

A new national study reveals a strong link between Agent Orange exposure and the risk of developing myelodysplastic syndrome, with exposed veterans diagnosed at younger ages and experiencing more aggressive disease. The study found that those with MDS were nearly twice as likely to see their disease progress within two years after diag...

Black patients with acute myeloid leukemia are younger at diagnosis and experience poorer survival outcomes than White patients

Black patients with acute myeloid leukemia are diagnosed at younger ages and have worse outcomes compared to white patients, according to a study analyzing data from 10 clinical trials over 34 years. The study found that Black patients had a higher risk of dying from AML and any cause, even when treated with similar mutations.

Targeted immunotherapy combination offers hope to older adults with leukemia

Researchers found a targeted immunotherapy regimen yielded promising survival outcomes for patients with B-cell ALL, outperforming historical results. The treatment was well-tolerated, with more than half completing the full course of therapy, and responded similarly in patients with complex medical histories.

SourceAlliance for Clinical Trials in Oncology·JournalJournal of Clinical Oncology·TypeRandomized controlled/clinical trial·DateOct 20, 2025

City of Hope Research Spotlight, September 2025

Researchers at City of Hope have identified a potential strategy to overcome treatment resistance in acute myeloid leukemia and uncovered racial disparities in triple-negative breast cancer. By targeting the ALKBH1 protein, which enhances energy production in cancer cells, scientists found that blocking this protein could make CAR T th...

SourceCity of Hope·JournalNature Genetics·DateOct 9, 2025

Study: olutasidenib is highly effective in certain patients with myelodysplastic syndrome

Researchers found olutasidenib to be highly effective in patients with myelodysplastic syndrome (MDS) and IDH1 mutations. The study showed a response rate of 59% and improved blood count improvement, long duration of response, and overall survival rates. This breakthrough offers new treatment options for these patients.

Study shows DNA regulatory switch prompts stem cells to give rise to blood

A new study has found that TAF1 operates as a key molecular switch in adult hematopoietic stem cell maintenance and lineage commitment. This discovery challenges prevailing models of gene regulation and has the potential to lead to new therapeutic strategies targeting the molecule, which could improve blood production and transplantation.

SourceUniversity of Miami Miller School of Medicine·JournalDevelopmental Cell·DateJul 24, 2025