[Keynote Session] Frontline of Stem Cell Research and Strategies for Success in the Pharmaceutical Industry

[Abstract]
Following embryonic development, most of our tissues and organs are continuously regenerated from tissue/organ specific stem cells. The principal property that distinguishes such stem cells from their daughter cells is self-renewal; when stem cells divide they give rise to stem cells (by self-renewal) and progenitors (by differentiation). In most tissues only the primitive stem cells self-renew. Stem cell isolation and transplantation is the basis for regenerative medicine. Self-renewal is dangerous, and therefore strictly regulated. Poorly regulated self-renewal can lead to the genesis of cancer stem cells, the only self-renewing cells in the cancerous tumor. The Weissman lab has followed the progression from hematopoietic stem cells to myelogenous leukemias. They have found that the developing cancer clones progress at the stage of hematopoietic stem cells, until they become fully malignant. At this point, the ‘leukemia’ stem cell moves to a stage of a downstream oligolineage or multilineage progenitor that has evaded programmed cell death and programmed cell removal, while acquiring or keeping self-renewal. While there are many ways to defeat programmed cell death and senescence, there appears to be one dominant method to avoid programmed cell removal―the expression of the cell surface ‘don’t eat me’ protein CD47, the ligand for macrophage SIRP-alpha. All cancers tested express CD47 to overcome expression of ‘eat me’ signals such as calreticulin and asialogylycoproteins. Antibodies that block the CD47―SIRP-alpha interaction enable phagocytosis and killing of the tumor cells in vitro and in vivo.

[Profile]
Irving L. Weissman received his B.S. from Montana State University and an M.D. from Stanford University. He became Director of the Stanford Cancer/Stem Cell Institute in 2002, which will house the first PhD program devoted to stem cell biology in the nation. He was a founder of three companies, SyStemix, Cellerant, and Stem Cells, Inc., all focused on bringing stem cell therapies into the clinic, and earlier was on the founding SAB’s of Amgen, DNAX, and T Cell Sciences. His main research interests are 1) hematopoietic stem and progenitor cells, 2) central nervous system stem and progenitor cells, 3) lymphocyte differentiation, 4) homing receptors, 5) normal and neoplastic hematolymphoid development, and 6) the phylogeny of stem cells and alloreactivity in protochordates.

[Abstract]
The presentation will predict innovations and new growth opportunities that are expected in the pharmaceutical industry, identify new competitors entering the field and examine the impact of new technologies. It will provide an analysis of the current challenges facing pharmaceutical companies today and some potential business models as solutions to success.

[Profile]
Reenita Das, Senior Vice President Global Healthcare & Life Sciences at Frost & Sullivan, has more than 20 years of healthcare marketing consulting expertise, which include engaging global clients to launch new products in emerging markets. Ms. Das' particular expertise is in competitive intelligence, best practice mapping of core capabilities and understanding of emerging markets and the challenges of the complex business scenario enabling unique strategies to penetrate and operate efficiently. She has a wide experience base covering broad range of sectors, leveraging long-standing working relationships with leading industry participants’ senior executives in the medical devices and medical technology, clinical diagnostics, pharmaceutical, biotechnology and life sciences sector.

[Abstract]
As a result of a significant number of patent expirations of blockbuster drugs since around 2010 and difficulties in developing a next blockbuster drug, a number of mega pharma companies are facing a drastic change in their business models. Recently, their attention to orphan drugs, which have high unmet medical needs, is increasing. In the meanwhile, since a few decades ago, biotech ventures in the US have been developing drugs targeting rare diseases and have established a growing business model. The presentation will introduce Genzyme's business model, which is one of the pioneering orphan drug companies, and issues in rare disease business.