Archive for drug development
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 about Gene therapy’s future may be all about the bases
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 about A drug treatment for telomere diseases?
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 about Targeted small-molecule agent shows early promise against a dangerous infant leukemia
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 about Boosting host immune defenses to treat tuberculosis
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 about Overriding resistance to epigenetic inhibitors in neuroblastoma
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 about Children wait for new cancer drugs 6.5 years longer than adults
‘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 about ‘Nanobodies’ from alpacas could help bring CAR T-cell therapy to solid tumors
In zebrafish, a way to find new cancer therapies, targeting tumor promoters
The lab of Leonard Zon, MD, has long been interested in making blood stem cells in quantity for therapeutic purposes. To test for their presence in zebrafish, their go-to research model, they turned to the MYB gene, a marker of blood stem cells. To spot the cells, Joseph Mandelbaum, a PhD candidate in the lab, attached a fluorescent ... Read More about In zebrafish, a way to find new cancer therapies, targeting tumor promoters
The softer the nanoparticle, the better the drug delivery to tumors
For the first time, scientists have shown that the elasticity of nanoparticles can affect how cells take them up in ways that can significantly improve drug delivery to tumors. A team of Boston Children’s Hospital researchers led by Marsha A. Moses, PhD, who directs the Vascular Biology Program, created a novel nanolipogel-based drug delivery system that allowed ... Read More about The softer the nanoparticle, the better the drug delivery to tumors
Using ultrasound to trigger on-demand, site-specific pain relief
According to the CDC, 91 people die from opioid overdoses every day in the U.S. Here in Massachusetts, the state has an opioid-related death rate that is more than twice the national average. “Opioid abuse is a growing problem in healthcare,” says Daniel Kohane, MD, PhD, a senior associate in critical care medicine at Boston Children’s and professor ... Read More about Using ultrasound to trigger on-demand, site-specific pain relief