Blood stem cell transplants from any donor, without toxicity?
Many blood disorders, immune disorders and metabolic disorders can be cured with a transplant of hematopoietic (blood-forming) stem cells, also known as bone marrow transplant. But patients must first receive high-dose, whole-body chemotherapy and/or radiation to deplete their own defective stem cells, providing space for the donor cells to engraft. These “conditioning” regimens are highly ... Read More about Blood stem cell transplants from any donor, without toxicity?
Dually-targeted liposomes curb triple-negative breast cancer, metastases in mice
Some 15 to 20 percent of all breast cancers are triple-negative, meaning they lack receptors for estrogen, progesterone and human epidermal growth factor type 2. They have the worst prognosis of all breast cancers and very limited treatment options. Finding a treatment that distinguishes between cancer cells and normal cells has been especially challenging. A ... Read More about Dually-targeted liposomes curb triple-negative breast cancer, metastases in mice
How the antidepressant ketamine rapidly awakens the brain, and why its effects vary more in women
In small doses, the anesthetic ketamine is a mildly hallucinogenic party drug known as “Special K.” In even smaller doses, ketamine relieves depression — abruptly and sometimes dramatically, steering some people away from suicidal thoughts. Studies indicate that ketamine works in 60 to 70 percent of people not helped by slower-acting SSRIs, the usual drugs ... Read More about How the antidepressant ketamine rapidly awakens the brain, and why its effects vary more in women
I-ACQUIRE: A new study for young stroke patients
Could I-ACQUIRE help your child?To qualify for this study, your child must meet the following criteria: · is 8 to 36 months old · had a perinatal arterial ischemic stroke confirmed by MRI · has weakness on one side of the body both arm and leg · is in good health · hasn’t had previous ... Read More about I-ACQUIRE: A new study for young stroke patients
Drug repurposing and DNA mining: The hunt for new endometriosis treatments
Endometriosis is a common gynecological condition that may affect more than 1 in 10 reproductive-age women. Yet, there’s very little research into the disease and limited options for treatment. A team in the Vascular Biology Program at Boston Children’s Hospital is trying to change that. In endometriosis, tissue that normally grows inside the uterus grows outside of ... Read More about Drug repurposing and DNA mining: The hunt for new endometriosis treatments
CRISPR-Cas9 screen opens new targets for Ewing sarcoma, other childhood cancers
While the genetic mutations driving adult cancers can sometimes be targeted with drugs, most pediatric cancers lack good targets. That’s because their driving genetic alterations often create fusion proteins that aren’t easy for drugs to attack. “This is one reason why it is notoriously hard to make targeted drugs against childhood cancers — their cancer-promoting ... Read More about CRISPR-Cas9 screen opens new targets for Ewing sarcoma, other childhood cancers
After 80 years, genetic causes of Diamond-Blackfan anemia come into view
In 1938, Louis K. Diamond, MD, and Kenneth Blackfan, MD, at Boston Children’s Hospital described a severe congenital anemia that they termed “hypoplastic” (literally, “underdeveloped”) because of the bone marrow’s inability to produce mature, functioning red blood cells. Eighty years later, the multiple genetic origins of this highly rare disease, now known as Diamond-Blackfan anemia, or ... Read More about After 80 years, genetic causes of Diamond-Blackfan anemia come into view
New angles for blocking Shiga and ricin toxins, and new light on an iconic biological process
Min Dong, PhD, and his lab are world experts in toxins and how to combat them. They’ve figured out how Clostridium difficile’s most potent toxin gets into cells and zeroed in on the first new botulinum toxin identified since 1969. Now, they’ve set their sights on Shiga and ricin toxins, and not only identified new potential lines of defense, ... Read More about New angles for blocking Shiga and ricin toxins, and new light on an iconic biological process
Reviving fetal hemoglobin in sickle cell disease: First patient is symptom-free
Manny Johnson of Boston, 21, previously required monthly blood transfusions to keep his severe sickle cell disease under control. After receiving a new gene therapy treatment, he’s been symptom-free for six months. Researchers at the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center reported Manny’s case Saturday at the American Society of Hematology meeting in San Diego. Manny ... Read More about Reviving fetal hemoglobin in sickle cell disease: First patient is symptom-free
Study sounds another warning about proton pump inhibitors
A new study adds to growing concerns about a class of drugs frequently prescribed to suppress stomach acid in patients with gastroesophageal reflux disease (GERD). Previous research has linked the use of proton pump inhibitors (PPIs) to an increased risk of various pulmonary and gastrointestinal infections in both adults and children. Patients treated with PPIs ... Read More about Study sounds another warning about proton pump inhibitors