Tuesday, November 19, 2013

Breakthrough discoveries: how iPS cells are changing stem cell science

iPS cells
When the Japan-based Alliance Forum Foundation brought its meeting to San Francisco last Friday it had two goals. The non-profit wanted to deepen the long tradition of collaboration between Japan and the U.S. and to honor Shinya Yamanaka, who won the Nobel Prize last year and who bridges the two countries with labs at Kyoto University and the Gladstone Institutes in San Francisco.

Throughout the day Yamanaka’s prize-winning idea for reprogramming adult cells into embryonic-like stem cells was referred to as game changing for all of biology. Those induced Pluripotent Stem cells, or iPS cells, were lauded as valuable research tools today and revolutionary therapies in the not too distant future.

Mahendra Rao, director of the NIH Center for Regenerative Medicine, likened iPS technology to PCR, polymerase chain reaction, a chemical trick invented 30 years ago that lets scientist amplify any desired set of genes for study. This empowered countless opportunities to ask questions about the function of genes. He said biology is viewed as pre-PCR and post-PCR, and he added that iPS is causing a similar paradigm shift. In particular he noted the opportunity to create personalized cells that can have their genes edited or corrected, if they have an inherited error.

Sandy Williams, the president of the Gladstone Institutes, ran through a scenario for how iPS cells can save society billions of dollars. He suggested that the ability to make iPS cells — and in turn nerve cells — from patients with Alzheimer’s Disease will provide such a valuable platform for drug screening that finding a drug to treat the dementia was inevitable. If such a drug was just 50 percent effective, he said it would save the U.S. $540 billion a year in costs associated with the disease by 2050.

Shortly after Sandy spoke, Nancy Stagliano, CEO of biotech company iPierian, provided strong support for his premise. She said that within 12 months the company expects to begin a clinical trail with an antibody therapy identified with an Alzheimer’s patient’s iPS cells.

All of CIRM’s grants can be sorted by the type of cell used and you can read about CIRM grants using iPS cells here.

Leonore and Leonard Herzenberg: courtesy Stanford Medical School
One of the earlier speakers made a fitting tribute to another game-changing technology and the couple who made it happen, Leonard and Leonore Herzenberg, always known on the Stanford campus when I was there as just Len and Lee. Kenichi Arai said that the cell sorting machines they invented and continued to perfect over the years brought drug development to the cellular level and made much of stem cell science possible. Len died the week before the conference, but Lee was able to attend. Stanford's obituary for Len has more about their discovery.

Don Gibbons

1 comment:

  1. Stem cell firm Mesoblast pins hopes on Japanese regulatory change

    Australian biotech firm Mesoblast is excited by this week’s legislative changes in Japan that will allow fast tracked approvals of stem cell products.Mesoblast is the world’s biggest player in the biotechnology sector, with potential therapies made from adult stem cells.This week Japan’s Diet passed bills to ensure the safety of regenerative medicine and to enable swift medical treatment using stem cells.This augers well for Mesoblast, because Japan is the world’s second largest healthcare market.Mesoblast chief executive and founder Professor Silviu Itescu told Inside Business the bills enable Japan’s government to approve new products conditionally, providing their safety is confirmed in clinical trials, even if their efficacy has not yet been verified.”The Japanese legislature has, just for stem cell products, defined them in a unique category as regenerative medicine, providing that in phase-two trials it demonstrates sufficient safety signals,” Mr Itescu said.”And of course safety trials are not usually designed to statistically prove efficacy.”The change means such products might be approved for the Japanese market without having to complete “phase-three” trials.”That’s a very exciting prospect for us, it potentially shaves off several years and very large expenditures in getting our products into the Japanese market which is really the second largest mature market in the world after the United States,” Mr Itescu said.