iPS cell roundup: testing drugs, studying disease, developing therapies

Since Shinya Yamanaka won the Nobel prize last year for reprogramming adult cells to an embryonic-like state–so-called iPS cells–it’s been hard to keep up with the pace of new research using these cells. For those looking for a summary, The Gladstone Institutes has recently posted a nice, accessible explanation of induced pluripotent stem (iPS) cells and how they’re being used in current research. The story focuses on projects by Gladstone Institutes scientists, many of whom are CIRM grantees (there’s a list of awards to Gladstone on our website).

The article lays out Shinya Yamanaka’s initial discovery of iPS cells, coaxing mature cells to revert back to a less differentiated state and reprogramming them to grow into a completely different kind of cell. More recently, scientists like Deepak Srivastava have figured out how to skip that intermediate undifferentiated state and transform cells directly into a new kind of cell.

In the U.S., therapies based on iPS cells haven’t started clinical trials, but researchers are putting them to present-day use as tools to test drugs and better understand disease. Gladstone scientists profiled in the article are using iPS cells to understand ailments as varied as cardiovascular disease, Alzheimer’s disease and HIV and elsewhere scientists are using them to study diseases as diverse as autism, schizophrenia and Huntington’s disease. In this video, Bruce Conklin, a Gladstone Institute scientist, explains how the cells can be used to study disease and also test drugs for toxicity.

CIRM funds iPS cell projects throughout California to scientists like Conklin who are trying to study diseases and test drugs as well as scientists who are developing new therapies. You can learn more about more about those awards on our website.

Rina Shaikh-Lesko

One thought on “iPS cell roundup: testing drugs, studying disease, developing therapies

  1. HIV used to cure 2 genetic diseases: The idea of an Italian scientist proves successful

    After the positive results obtained in the course of many years of study in the laboratory, researchers at the San Raffaele Telethon Institute in Milan tried to correct the genetic defect that causes these diseases with gene therapy. The technique consists in withdrawing hematopoietic stem cells from the bone marrow of the patient and introducing a corrected copy of the gene that is defective using viral vectors derived from HIV (which began to be developed in 1996, thanks to Luigi Naldini's work). Once re-injected into the body, the treated cells are able to restore the missing protein to key organs.

    “In patients with Wiskott-Aldrich syndrome, blood cells are directly affected by the disease and the corrected stem cells replace the diseased cells creating a properly functioning immune system and normal platelets. Thanks to gene therapy, the children no longer have to face severe bleeding and infection. They can run, play and go to school,” explains Alessandro Aiuti, coordinator of the clinical study on these patients and Head of Research of the Pediatric Clinic at TIGET. “In the case of metachromatic leukodystrophy, however,” says Alessandra Biffi, who heads the other study, “the therapeutic mechanism is more sophisticated: the corrected hematopoietic cells reach the brain through the blood and release the correct protein that is 'gathered' there by the surrounding nerve cells. The winning card was to make engineered cells able to produce a quantity of protein much higher than normal, and thus effectively counteract the neurodegenerative process.” Eugenio Montini, who coordinated the molecular analysis of patients' cells, adds, “Until now we have never seen a way to engineer stem cells using gene therapy that is as effective and safe as this one. These results pave the way for new therapies for other more common diseases.”

    Both trials, which involved a team of over 70 people including researchers and clinicians, began in the spring of 2010, and called for the participation of 16 patients in total, 6 suffering from Wiskott-Aldrich syndrome and 10 from metachromatic leukodystrophy. The results published in Science refer only to the first 6 patients (three from each study), i.e. those for whom sufficient time has passed after administration of the therapy to allow scientists to draw the first significant conclusions regarding its safety and efficacy. In total, the Telethon Foundation has invested 19 million Euro for research on these two diseases (11 on metachromatic leukodystrophy and 8 on Wiskott-Aldrich syndrome).


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