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Nobel Laureate Revisiting Lecture – Shinya Yamanaka

Nobel Laureate Revisiting Lecture

Date: Thursday, June 7th at 4 p.m

Wallenbergsalen, Nobel Forum,
Karolinska Institutet, Nobels väg 1

Shinya Yamanaka
Nobel Laureate in Physiology or Medicine 2012

Title: Recent Progress in iPS Cell Research and Application

Induced pluripotent stem cells (iPSCs) can proliferate almost indefinitely
and differentiate into multiple lineages, giving them wide medical
application. As a result, they are being used for new cell-based
therapies, disease models and drug development around the world. In
2014, the world’s first clinical study using iPSCs began for the
treatment of age-related macular degeneration. iPSCs can be used for
regenerative medicine to restore organ function. To push these efforts,
we are proceeding with an iPSC stock project in which clinical-grade
iPSC clones are being established from “super” donors with
homologous HLA haplotypes. Homologous HLA haplotypes are
associated with decreased immune response and therefore less risk of
transplant rejection. The iPSC stock is being designed with the
intention of providing quality-assured cells for medical treatments
around the world. In 2015, we started distributing an iPSC stock clone
to organizations in Japan.

Other applications of iPSCs include drug screening, toxicity studies and
the elucidation of disease mechanisms using disease-specific iPSCs
from patients with intractable diseases. iPSCs established from
patients contain a complete set of the genes that resulted in the
disease and thus represent a new disease model that complements or
in some cases replaces animal models. Indeed, Bosutinib, a drug for
leukemia was revealed to be efficacious for the treatment of
amyotrophic lateral sclerosis (ALS) using a disease model established
from patient-derived iPSC. In Addition, we reported a new drug
screening system using iPSCs derived from fibrodysplasia ossificans
progressiva (FOP) patients, revealing one drug candidate, Rapamycin.
Based on these findings, we have received approval from the Japanese
government to initiate a clinical trial to treat FOP patients. This clinical
trial is the world’s first for a drug that is based on results from an iPS
cell-based model. Over the past decade iPSCs research made a great
progress. However, there are still various hurdles to be overcome,
iPSC-based science is certainly moving forward for delivering
innovative therapeutic options to the patients with intractable diseases.


Professor Thomas Perlmann, Thomas.Perlmann@ki.se
Ann-Mari Dumanski,
Nobel Office, Nobel Forum
08-524 878 00,