Archive for drug development
The journey to a treatment for hereditary spastic paraplegia
In 2016, Darius Ebrahimi-Fakhari, MD, PhD, a neurology fellow at Boston Children’s Hospital, met two little girls with spasticity and decreased muscle tone in their legs, which affected their walking. Both girls, Robbie Edwards and Molly Duffy, had been diagnosed with hereditary spastic paraplegia (HSP), a group of more than 80 genetic conditions. Untreated, the ... Read More about The journey to a treatment for hereditary spastic paraplegia
Naloxone on demand: Shining a light to reverse opioid overdose
Overdose deaths from fentanyl and other opioids are at record highs in the U.S. Naloxone, if delivered soon after an overdose, is proven to be life-saving. It binds to the same brain receptors that opioids use, thereby blocking opioids’ effects. A naloxone nasal spray (Narcan) is now available over the counter, but there are still problems ... Read More about Naloxone on demand: Shining a light to reverse opioid overdose
Gold particles and light could melt venous malformations away
Venous malformations — tissues made up largely of abnormally shaped veins — are often difficult to treat, especially when located in sensitive areas like the eyes, face, and genitourinary organs. In the worst cases, the lesions are disfiguring and can crush or obstruct surrounding tissues, cause bleeding and clotting, interfere with breathing or vision, or ... Read More about Gold particles and light could melt venous malformations away
Nanobodies from alpacas could steer immune attacks on influenza
While conventional flu vaccines are designed to anticipate the influenza strains projected to dominate in the next flu season, they’re only partially effective. And while antiviral drugs are available to treat active flu cases, the body quickly clears them, requiring high, frequent doses. Coupling one existing flu drug with a special ingredient from alpacas, the ... Read More about Nanobodies from alpacas could steer immune attacks on influenza
Playing the long game: An exciting discovery in telomere disease
Each time our cells divide, the protective caps that keep our chromosomes from fraying, called telomeres, lose a bit of their DNA. Telomeres shorten steadily as we age, but in certain medical conditions like dyskeratosis congenita, the process is accelerated. “Your telomeres determine your lifeline; how long they are determines how old your body is,” ... Read More about Playing the long game: An exciting discovery in telomere disease
Going out of the box to tackle pancreatic cancer
Pancreatic cancers are deadly and hard to treat, in part because they are so often detected at an advanced stage; overall five-year survival rates are about 11 percent. Two separate labs at Boston Children’s Hospital took out-of-the-box approaches to this difficult cancer, and both uncovered some very promising leads. Wiping out pancreatic tumors’ immune defense ... Read More about Going out of the box to tackle pancreatic cancer
Why do so many dementia treatments fail? Questioning mouse models of tau accumulation
To date, the search for effective treatments for dementia has yielded only disappointments. Many recent drug candidates target the tau protein, which aggregates and forms tangles in patients’ brain tissue and is involved in 75 percent of all dementias. While tau-targeting drugs have looked promising in mouse models, they’ve failed in clinical trials. A recent ... Read More about Why do so many dementia treatments fail? Questioning mouse models of tau accumulation
‘On fire’ with sJIA: When arthritis is much more than joint pain
Georgia is finally living her best life. Her toddler years were challenging: At 15 months old, a series of high fevers landed her at Boston Children’s Hospital for two weeks. After many rounds of tests looking for infection and a bone marrow biopsy to rule out cancer, she was diagnosed with systemic juvenile idiopathic arthritis ... Read More about ‘On fire’ with sJIA: When arthritis is much more than joint pain
Tim Springer: Scientist, entrepreneur, and mentor
As an undergraduate in 1966, immunologist, biochemist, and biophysicist Timothy A. Springer, PhD, looked askance at science. The Vietnam War was going on, and he saw science as a means of making Agent Orange and napalm. Questioning his own Ivy League education, he left Yale to spend a year as a VISTA volunteer on a ... Read More about Tim Springer: Scientist, entrepreneur, and mentor
From our labs and clinics: The top 10 COVID-19 science stories of 2021
As COVID-19 waxed, waned, morphed, and waxed again this year, research was taking place throughout Boston Children’s Hospital. Ongoing national studies the hospital is leading or co-leading include Overcoming COVID-19, a CDC-funded study on COVID-19 and multisystem inflammatory syndrome in children (MIS-C); IMPACC, examining immune responses in people hospitalized with COVID-19; and MUSIC, studying MISC’s ... Read More about From our labs and clinics: The top 10 COVID-19 science stories of 2021