Stories about: Alessandra Biffi

Routing gene therapy directly into the brain

Image of mouse brain that received a transplantation of hematopoietic stem cells. The image shows the transplanted cells (green) rapidly engrafted and gave rise to new cells (also green) that have widely distributed throughout the entire brain. 
Image of a mouse brain that received a direct transplantation of hematopoietic stem cells. The image reveals the transplanted cells (green) rapidly engrafted and gave rise to new cells (also green) that have widely distributed throughout the entire brain.

A therapeutic technique to transplant blood-forming (hematopoietic) stem cells directly into the brain could herald a revolution in our approach to treating central nervous system diseases and neurodegenerative disorders.

The technique, which could be used to transplant donor-matched hematopoietic stem cells (HSCs) or a patient’s own genetically-engineered HSCs into the brain, was reported in Science Advances today by researchers from the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and the San Raffaele Telethon Institute for Gene Therapy.

In their study, the team tested the technique in a mouse model to treat lysosomal storage disorders, a group of severe metabolic disorders that affect the central nervous system.

The team’s findings are groundbreaking because, until now, it was thought that HSCs — from a healthy, matched donor or a patient’s own genetically-corrected cells — needed to be transplanted indirectly

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Landmark moment for science as the FDA approves a gene therapy for the first time

Leukemia blast cells, which could now be destroyed using a first-of-its-kind, FDA-approved gene therapy called CAR-T cell therapy
Leukemia blast cells.

Today, the Food and Drug Administration approved a gene therapy known as CAR T-cell therapy that genetically modifies a patient’s own cells to help them combat pediatric acute lymphoblastic leukemia (ALL), the most common childhood cancer. It is the first gene therapy to be approved by the FDA.

“This represents the progression of the field of gene therapy, which has been developing over the last 30 years,” says gene therapy pioneer David A. Williams, MD, who is chief scientific officer of Boston Children’s Hospital and president of the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. “It’s a realization of what we envisioned to be molecular medicine when this research started. The vision — that we could alter cells in a way to cure disease — is now coming true.”

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