Archive for drug development
Precision chemo-immunotherapy for pancreatic cancer?
Pancreatic cancer is highly lethal and in great need of better treatments. Only about 10 percent of patients remain alive five years after diagnosis. In a new study, researchers in the lab of Marsha Moses, PhD, at Boston Children’s Hospital offer a glimmer of hope. Key takeaway An antibody-drug combination effectively targeted, penetrated, and shrank ... Read More
A diabetes drug with a potential side benefit: Reduced COVID-19 mortality
In 2006, the Food and Drug Administration approved the drug sitagliptin to lower blood sugar in type 2 diabetes. Now, a multicenter observational study in Italy suggests the drug also cuts mortality in diabetic patients hospitalized with COVID-19 — by as much as half. Paolo Fiorina, MD, PhD, of Boston Children’s Hospital led the study, ... Read More
Lung ‘organoids’ capture early-stage lung cancer; could help test treatments
Lung cancer, the leading cancer killer in the U.S., is often missed in its earlier stages. And while recent imaging advances offer earlier detection, early-stage lung cancers still have no targeted treatments. Key takeaways Mini “organoids” made from lung cells offer a rapid platform for tracking early-stage lung cancers and for testing possible treatments. RNA ... Read More
Gene therapy’s future may be all about the bases
Gene therapy offers the possibility of a cure for many genetic disorders, especially those involving a single gene. The first kind of gene therapy used a virus to carry a corrected copy of the gene into people’s cells. When the early viral vectors used in the 1990s were found to have off-target effects, sometimes even ... Read More
A drug treatment for telomere diseases?
For years, Donna Martin carried a piece of scrap paper with the words “dyskeratosis congenita,” which she believed might explain her son Brad’s sudden, mysterious affliction. A routine blood test had revealed Brad’s bone marrow was failing, unable to keep up with his need for healthy blood cells. His condition, Donna knew, would worsen over ... Read More
Targeted small-molecule agent shows early promise against a dangerous infant leukemia
Leukemias involving reshuffling or rearrangement of the mixed lineage leukemia (MLL) gene, known as MLL-rearranged or MLL-r leukemias, account for 70 to 80 percent of acute leukemias in infants under one year old. In these blood cancers, a subset of acute myeloid and acute lymphoid leukemias (AML and ALL), the MLL gene breaks and reattaches ... Read More
Boosting host immune defenses to treat tuberculosis
Current treatment regimens for Mycobacterium tuberculosis (Mtb), the causative agent of tuberculosis, are long, complex, and hard for people to sustain. Moreover, the bacteria often develop drug resistance, and many people harbor multi-drug-resistant strains. In 2018 alone, nearly 1.5 million people died from tuberculosis worldwide. Now, a study in iScience suggests a new approach that ... Read More
Overriding resistance to epigenetic inhibitors in neuroblastoma
Neuroblastoma and other children’s cancers pose unique challenges. They’re not caused by the same kinds of genetic mutations that cause adult cancers, and only a minority of their mutations can be targeted with drugs. In a recent study, a team led by Kimberly Stegmaier, MD, at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center systematically deleted every gene ... Read More
Children wait for new cancer drugs 6.5 years longer than adults
A 20-year analysis finds that FDA-approved cancer drugs took a median of 6.5 years to go from the first clinical trial in adults to the first trial in children. That’s not good enough for researchers at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, who are calling for expanding children’s access to experimental cancer therapies. “It’s ... Read More
‘Nanobodies’ from alpacas could help bring CAR T-cell therapy to solid tumors
In 1989, two undergraduate students at the Free University of Brussels were asked to test frozen blood serum from camels, and stumbled on a previously unknown kind of antibody. It was a miniaturized version of a human antibody, made up only of two heavy protein chains, rather than two light and two heavy chains. As ... Read More