ASH: New Approach to Gene Tx Restores Immune Cells in X-SCID

This article originally appeared here.
Share this content:
ASH: New Approach to Gene Tx Restores Immune Cells in X-SCID
ASH: New Approach to Gene Tx Restores Immune Cells in X-SCID

MONDAY, Dec. 11, 2017 (HealthDay News) -- A new approach to gene therapy can restore immune cell types in infants with newly diagnosed X-linked severe combined immunodeficiency (X-SCID), according to a study presented at the annual meeting of the American Society of Hematology, held from Dec. 9 to 12 in Atlanta.

Ewelina Mamcarz, M.D., from the Children's National Medical Center in Memphis, Tenn., and colleagues developed a new approach for X-SCID gene therapy using a safety-modified lentiviral vector together with reduced exposure busulfan (Bu) conditioning for newly diagnosed infants with X-SCID. Six subjects were enrolled over 12 months. Purified bone marrow (BM) CD34+ cells were transduced with the vector generated by a stable producer cell line, which was cryopreserved. Patients were given a median dose of 8.06 × 106 cells/kg transduced CD34+ cells.

The researchers observed no severe adverse events related to BM harvest, Bu exposure, or cell infusion as of July 2017. In the first five evaluable cases, by three to four weeks, complete hematopoietic recovery occurred without any blood product support. In most cases, rapid T cell reconstitution was seen. In addition, there was efficient vector marking in BM CD34+ cells, myeloid cells, and B cells.

"It is very exciting that we observed restoration of all three very important cell types in the immune system," Mamcarz said in a statement. "This is something that's never been done in infants and a huge advantage over prior trials."

Several authors disclosed financial ties to the biopharmaceutical industry.

Abstract
More Information

Share this content:

is free, fast, and customized just for you!

Already a member?

Sign In Now »

Drug Lookup

Browse drugs by: BrandGenericDisease

Trending Activities

All Professions



Sign up for myCME e-newsletters


More in Home

Medicare Requiring Hospitals to Post Prices Online

Medicare Requiring Hospitals to Post Prices Online

Medicare would base part of a hospital's payments on how easy it is for patients to access medical records

Restless Brain Syndrome Tied to Changes in the Brain

Restless Brain Syndrome Tied to Changes in the ...

Morphological differences seen in the primary somatosensory system

People With Opioid Use Disorder Have High Suicide Rate

People With Opioid Use Disorder Have High Suicide ...

Based on data from multiple sources, many opioid-overdose deaths seem to be suicides

is free, fast, and customized just for you!

Already a member?

Sign In Now »