Archive for drug development
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
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
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
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
Building a better botox
Aside from reducing wrinkles, botulinum toxins — a.k.a. botox — have a variety of uses in medicine: to treat muscle overactivity in overactive bladder, to correct misalignment of the eyes in strabismus, for a movement disorder called cervical dystonia that causes neck spasms, and more. Two botulinum toxins, types A and B, are FDA-approved and ... Read More
Precision drug delivery systems could ‘trigger’ an age of nanomedicine
What if we could deliver biocompatible nanoparticles into the body and then activate them to release drugs exactly where they are needed, without causing side effects elsewhere? Scientists like Daniel Kohane, MD, PhD, of Boston Children’s Hospital, are developing nanoscale drug delivery systems to do just that, using a variety of materials and triggers that ... Read More
Drug-eluting contact lens offers hope in glaucoma
Daily medicated eye drops are the first line of treatment for glaucoma, the leading cause of irreversible blindness. The drops relieve pressure in the eye, a significant risk factor for glaucoma. But they’re not ideal: their delivery is imprecise, they can cause stinging and burning and patients often struggle to administer them. Adherence is poor: ... Read More