Cell Therapy Highlights: Stem cell-based HIV Trials, Progenitor Cell Transplantation and Germline editing controversy
This week, we highlight some of the more interesting headline-making news and research findings that have appeared over the past week.
FDA Approves Sangamo’s Stem Cell-based HIV Cure Trials
The Food and Drug Administration (FDA) last week approved clinical trials to test a functional cure to treat HIV based on gene editing of blood stem cells. The technique, developed by Sangamo BioSciences, is based on blood-producing stem cells derived from HIV-infected patients whose genes are edited to to create a mutation in the protein CCR5. This mutation eventually prevents HIV from attaching to the body’s blood cells. While HIV remains in the affected patient’s body, it has no ability of infecting T cells.
The trials will be conducted at the City of Hope Medical Center in California by Sangamo BioSciences and researchers from the Keck School of Medicine at the University of Southern California.
Promising pancreatic progenitor cell transplantation findings for Type 2 diabetes published in Stem Cell Journal
The lab of Timothy Kieffer, of the University of British Columbia in Canada, published promising results of an animal study on the use of stem cell transplantation for the treatment of Type 2 diabetes in the journal Stem Cell Reports last week. The authors treated mice with induced type 2 diabetes with encapsulated pancreatic progenitor cells derived from human embryonic stem cells alongside anti-diabetic drugs. After transplantation and alongside treatment with anti-diabetic drugs, the authors found that the cells developed into fully-functional beta cells, and caused improvement in body weight and hyperglycemia in treated patients.
Germline modification approach draws controversy
A lot was written in the past few weeks by various media outlets about germline cell modification. The gene splicing technology that has enabled such modifications to the germline genome and which has received the most attention – and controversy – recently is called CRISPR-Cas9, which stands for clustered regularly interspaced short palindromic repeats and which we wrote about in a previous blog. Jennifer Doudna, a co-inventor of the technology alongside Craig Mello and David Biltmore and five other scientists, co-wrote an opinion piece in Science magazine this week urging caution in using CRISPR and calling for discussion to support further developments and application of the technology. Others have been less subtle. The ISSCR and ARM both issued statements calling for moratoriums on germline editing calling it potentially unsafe, while proponents say it is a way to “cleanse” the genome from deleterious genes and avoid passing on genetic diseases.