Archive for blood disorder
Sickle cell disease, gene therapy, and blood cancers: Mysteries remain
Gene therapy trials for sickle cell disease have been showing great promise, even offering hope of a cure. But in early 2021, the trials ground to a halt after reports of blood malignancies in two people in a trial sponsored by bluebird bio. Investigations later concluded that the gene therapy delivery vectors were likely not ... Read More
Surviving stem cell transplant: New hope when the donor isn’t a full match
To see Tara Daniels today, with a corporate job in marketing and about to close on a house, you’d never know what she’s been through, how thrilled she is to be alive. This month marks five years since she received a high-risk bone marrow transplant for a life-threatening blood cancer. Tara woke up feeling sick ... Read More
Preventing leukemia by preventing rogue blood cells from taking over
As we age, many of us acquire mutations that cause some of our blood stem cells to multiply faster than others, forming their own distinct populations or “clones.” This is known as clonal hematopoiesis. In some cases, a single clone originating from a single genetically altered or mutated stem cell can expand to comprise up ... Read More
How growing up with sickle cell disease is shaping Nancy’s future
Imagine appearing completely healthy while managing a life-threatening condition. Most people who meet Nancy Blankson would never know she has sickle cell disease (SCD). Her symptoms are not easily visible. It’s a challenge she lives with every day along with periodic pain crises, which at times, she says, can be virtually unbearable. Nevertheless, the 20-year-old ... Read More
Teaming up to fast track a novel gene therapy for Diamond-Blackfan anemia
Looking at Lindsay Krieg, it’s impossible to tell she has spent her entire 29 years in an out of hospitals managing a rare blood disorder. “It’s routine for me,” she says. “It’s all I’ve ever known.” Diamond-Blackfan anemia (DBA) prevents Lindsay’s bone marrow from producing the red blood cells essential for carrying oxygen from her ... Read More
Can we prevent leukemia in patients with Shwachman-Diamond syndrome?
Anna Nazarenko doesn’t see herself as sick. The strong-willed, spunky 6-year-old loves to dance and ski, and spent much of April Fool’s day pranking her parents. Aside from the enzymes she takes to help digest her food, you wouldn’t know that she has Shwachman-Diamond syndrome (SDS). The rare, inherited type of bone marrow failure has ... Read More
Transitioning to adult care for sickle cell disease: Ariyanna’s journey
Ariyanna Agnew sits in a waiting room at Beth Israel Deaconess Medical Center (BIDMC). The 22-year-old, who has been a patient of the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center Sickle Cell Disease Program for more than a decade, is seeing an adult hematologist for the first time. It’s a milestone event that would ordinarily ... Read More
Avoiding a lifetime of injections: Can gene editing cure severe congenital neutropenia?
Fionn Mulrooney, a cheerful 11-month-old, in Plymouth, Massachusetts, has no idea he has a life-threatening genetic disease. Nor does he seem fazed by the daily subcutaneous injections his parents have learned how to give him. And little does he know that cells from his bone marrow are helping scientists develop an innovative gene-editing approach that ... Read More
Arthritis drug reduces rates of acute graft-vs-host disease after bone marrow transplant
The immune-suppressing drug abatacept, currently used for rheumatoid arthritis, could make bone marrow transplant safer, report researchers at the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. The phase 2 randomized, multi-center clinical trial, the largest to date, appears in the Journal of Clinical Oncology. Abatacept (brand name ORENCIA) reduced rates of severe, acute graft-versus-host disease ... Read More
Navigating the aplastic anemia journey
Hadas Asgedom is a woman with many titles: daughter, sister of 12, aunt, mother. But, after her own daughter’s journey with aplastic anemia, she may have earned the right to add another — relentless researcher. Hadas and her only child, Asada, live in Tampa, Florida, where Asada had always enjoyed theater rehearsals, soccer, and time ... Read More