Cells Weekly – June 19, 2016

by Alexey Bersenev on June 20, 2016 · 0 comments

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Cells Weekly is a digest of the most interesting news and events in stem cell research, cell therapy and regenerative medicine. Cells Weekly is posted every Sunday night!

1. Cell manufacturing roadmap
US-based National Cell Manufacturing Consortium issued this week the first document – Cell Manufacturing Roadmap. The roadmap is a collaborative project, which outlines the future directions for development of cell therapy manufacturing infrastructure as national priority in US. that can enable large-scale, cost-effective manufacturing of therapeutic cells. From Georgia Tech press release:

… the Georgia Research Alliance (GRA) and the Georgia Institute of Technology (Georgia Tech) have launched the National Cell Manufacturing Consortium (NCMC), an industry-academic-government partnership that recently released the National Roadmap for Advanced Cell Manufacturing. Establishment of the consortium and development of this 10-year national roadmap was sponsored by the National Institute of Standards and Technology (NIST).

2. Gene therapy of metachromatic leukodystrophy – interim report
Researchers from San Raffaele Scientific Institute (Italy) reported results of ad-hoc analysis of gene therapy trial for fatal demyelinating disease – metachromatic leukodystrophy. Preliminary results are great:

At the time of analysis all children had survived, with a median follow-up of 36 months (range 18–54).

A progressive reconstitution of ARSA activity in circulating haemopoietic cells and in the cerebrospinal fluid was documented in all patients in association with a reduction of the storage material in peripheral nerve samples in six of seven patients. Eight patients, seven of whom received treatment when presymptomatic, had prevention of disease onset or halted disease progression as per clinical and instrumental assessment, compared with historical untreated control patients with early-onset disease.

This trial is one more “licensing targets” of GSK.

3. New method for expansion of adult stem cells
New method for long-term expansion of epithelial stem cell was described this week in Cell Stem Cell. Using dual inhibition of SMAD signaling pathways, researchers were able to expand “airway basal stem cells from multiple species”. The senior author said:

“This new methodology opens up new avenues for research in any airway disease, such as asthma or COPD,” says Jayaraj Rajagopal, MD, of the MGH Center for Regenerative Medicine and the Harvard Stem Cell Institute, senior author of the report. “While in the past we could only expand stem cells for a few generations, now we have the ability to generate enough cells to last multiple laboratories for years of experiments. Our system is also very simple, avoiding the complexities of former culture systems and making it more accessible to many labs.”

“We also found that the same methodology works for many tissues of the body — from the skin to the esophagus to mammary glands. Many of these organ tissues cannot currently be cultured, so it remains to be seen whether scientists in these areas will be able to grow stem cells from samples acquired from other minimally invasive procedures, including the collection of secretions. If all this becomes possible, it would represent a big step forward for personalized medical approaches to disease,” he says.

4. A decade of iPS cell discovery
Nature posted nice news feature article on “iPS cell decade” – How iPS cells changed the world. The article emphasizes clinical translation of iPS cell technology:

Developing and testing a therapy in even one person has been educational, says Yamanaka: it took one year and US$1 million. He expects future therapies to use donor-derived iPS cells from a cell bank, rather than making them for each patient.

Takahashi plans to compare banked iPS cells side-by-side with those derived from patients, to observe any differences in immune reaction. She intends to apply to the Japanese government to resume her macular-degeneration trial “very soon”, but when asked, would not specify a timeline.

5. About F-class cells discovery
Jovana Drinjakovic of the Signals blog wrote a nice piece about discovery of F-class cells by Andras Nagy lab.

“Some scientists have a paranoia trying to show that what we are creating is the same as what naturally occurs in our bodies. But what’s natural? Everything that we have created, including embryonic stem cells, is just as artificial as our F-class cells,” says Nagy. Despite scientists’ best efforts to imitate the body’s environment, tweaking the nutrients, growth factors or oxygen until they’re just right for growing cells and tissues in the lab, these conditions are man-made and, according to Nagy, could clearly be considered artificial.

6. New methods and protocols:
Large-Scale production of neurons from human pluripotent stem cells in 3D culture (Stem Cell Rep)
Isolation of murine embryonic hemogenic endothelial cells (JoVE)
Generation of functional podocytes from human iPS cells (Stem Cell Res)
Lentiviral gene transfer into human and murine hematopoietic stem cells: size matters (BMC Res Notes)

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