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FDA announces new policy framework for development of regenerative medicine products

Today the U.S. Food and Drug Administration announced a comprehensive policy framework for the development and oversight of regenerative medicine products, including novel cellular therapies.

The framework — outlined in a suite of four guidance documents — builds upon the FDA’s existing risk-based regulatory approach to more clearly describe what products are regulated as drugs, devices, and/or biological products. Further, two of the guidance documents propose an efficient, science-based process for helping to ensure the safety and effectiveness of these therapies, while supporting development in this area. The suite of guidance documents also defines a risk-based framework for how the FDA intends to focus its enforcement actions against those products that raise potential significant safety concerns. This modern framework is intended to balance the agency’s commitment to safety with mechanisms to drive further advances in regenerative medicine so innovators can bring new, effective therapies to patients as quickly and safely as possible. The policy also delivers on important provisions of the 21st Century Cures Act.

We’re at the beginning of a paradigm change in medicine with the promise of being able to facilitate regeneration of parts of the human body, where cells and tissues can be engineered to grow healthy, functional organs to replace diseased ones; new genes can be introduced into the body to combat disease; and adult stem cells can generate replacements for cells that are lost to injury or disease. This is no longer the stuff of science fiction. This is the practical promise of modern applications of regenerative medicine.

What is regenerative capacity of humans?

Aubrey de Grey, Ph.D., Vice President of New Technology Discovery for AgeX Therapeutics, discusses how primitive organisms have better regenerative capacity than more complicated organisms such as humans. In humans, Dr. de Grey notes, our best regenerative abilities are at the embryonic stage. During the Embryonic Fetal Transition, out ability to regenerate plummets and continues to diminish as we age. Dr. de Grey discusses the role evolution plays in this and how scientists may be able to “revive” our regenerative power. This video is part of a series from AgeX on research into aging and human longevity. For more information on Agex Therapeutics, please visit http://www.agexinc.com.

Artificial Intelligence Based Approaches to Identify Molecular Determinants of Exceptional Health and Life Span-An Interdisciplinary Workshop at the National Institute on Aging

Artificial intelligence (AI) has emerged as a powerful approach for integrated analysis of the rapidly growing volume of multi-omics data, including many research and clinical tasks such as prediction of disease risk and identification of potential therapeutic targets. However, the potential for AI to facilitate the identification of factors contributing to human exceptional health and life span and their translation into novel interventions for enhancing health and life span has not yet been realized. As researchers on aging acquire large scale data both in human cohorts and model organisms, emerging opportunities exist for the application of AI approaches to untangle the complex physiologic process(es) that modulate health and life span. It is expected that efficient and novel data mining tools that could unravel molecular mechanisms and causal pathways associated with exceptional health and life span could accelerate the discovery of novel therapeutics for healthy aging. Keeping this in mind, the National Institute on Aging (NIA) convened an interdisciplinary workshop titled “Contributions of Artificial Intelligence to Research on Determinants and Modulation of Health Span and Life Span” in August 2018. The workshop involved experts in the fields of aging, comparative biology, cardiology, cancer, and computational science/AI who brainstormed ideas on how AI can be leveraged for the analyses of large-scale data sets from human epidemiological studies and animal/model organisms to close the current knowledge gaps in processes that drive exceptional life and health span. This report summarizes the discussions and recommendations from the workshop on future application of AI approaches to advance our understanding of human health and life span.

Aging is often described as the outcome of interactions among genetic, environmental and lifestyle factors with wide variation in life and health span between and within species (Newman and Murabito, 2013; Partridge et al., 2018; Singh et al., 2019). Exceptional life and health span represents an extreme phenotype characterized by exceptional survival (well-beyond average life expectancy), delayed onset of age-related diseases (before 80 years of age) (Pignolo, 2019) and/or preservation of good health/function relative to their peers (Perls et al., 2000, 2002; Kaeberlein, 2018). The identification of SNP associations with exceptional life and health span is a starting point for identifying targets for interventions that could potentially promote healthy human aging.

Bioinformatic prediction of critical genes and pathways involved in longevity in Drosophila melanogaster

The pursuit of longevity has been the goal of humanity since ancient times. Genetic alterations have been demonstrated to affect lifespan. As increasing numbers of pro-longevity genes and anti-longevity genes have been discovered in Drosophila, screening for functionally important genes among the large number of genes has become difficult. The aim of the present study was to explore critical genes and pathways affecting longevity in Drosophila melanogaster. In this study, 168 genes associated with longevity in D. melanogaster were collected from the Human Ageing Genomic Resources (HAGR) database. Network clustering analysis, network topological analysis, and pathway analysis were integrated to identify key genes and pathways. Quantitative real-time PCR (qRT-PCR) was applied to verify the expression of genes in representative pathways and of predicted genes derived from the gene–gene sub-network. Our results revealed that six key pathways might be associated with longevity, including the longevity-regulating pathway, the peroxisome pathway, the mTOR-signalling pathway, the FOXO-signalling pathway, the AGE-RAGE-signalling pathway in diabetic complications, and the TGF-beta-signalling pathway. Moreover, the results revealed that six key genes in representative pathways, including Cat, Ry, S6k, Sod, Tor, and Tsc1, and the predicted genes Jra, Kay, and Rheb exhibited significant expression changes in ageing D. melanogaster strain w1118 compared to young ones. Overall, our results revealed that six pathways and six key genes might play pivotal roles in regulating longevity, and three interacting genes might be implicated in longevity. The results will not only provide new insight into the mechanisms of longevity, but also provide novel ideas for network-based approaches for longevity-related research.

An Interview With Sarah Constantin of Daphnia Labs

We recently had the opportunity to catch up with Sarah Constantin, one of the founders of the new biotech startup company Daphnia Labs. The company is developing a new platform for the discovery of geroprotective drugs: in vivo phenotypic screens in model organisms. The company plans to use high-resolution video to track Daphnia during its lifespan in order to screen for drugs that might extend healthspan.

Can you tell us a little about the company, its founders, and what motivated you to start this biotech company?

I’d been working for about a year at the Longevity Research Institute, which is a nonprofit that funds aging research. LRI focuses on trying to replicate studies on interventions that have been reported to extend lifespan in mammals. Basically, of the 50+ compounds out there that have some mammalian evidence for an anti-aging effect, which ones have the best chance of being viable geroprotectors in humans?

Bill Faloon, Director / Co-Founder of Life Extension Foundation — ideaXme Show — Ira Pastor

Has this scientist finally found the fountain of youth?

The black mouse on the screen sprawls on its belly, back hunched, blinking but otherwise motionless. Its organs are failing. It appears to be days away from death. It has progeria, a disease of accelerated aging, caused by a genetic mutation. It is only three months old.

I am in the laboratory of Juan Carlos Izpisúa Belmonte, a Spaniard who works at the Gene Expression Laboratory at San Diego’s Salk Institute for Biological Studies, and who next shows me something hard to believe. It’s the same mouse, lively and active, after being treated with an age-reversal mixture. “It completely rejuvenates,” Izpisúa Belmonte tells me with a mischievous grin. “If you look inside, obviously, all the organs, all the cells are younger.”

Izpisúa Belmonte, a shrewd and soft-spoken scientist, has access to an inconceivable power. These mice, it seems, have sipped from a fountain of youth. Izpisúa Belmonte can rejuvenate aging, dying animals. He can rewind time. But just as quickly as he blows my mind, he puts a damper on the excitement. So potent was the rejuvenating treatment used on the mice that they either died after three or four days from cell malfunction or developed tumors that killed them later. An overdose of youth, you could call it.

Bill Faloon: A Life Long Quest To Reverse Human Aging!

Ira Pastor, ideaXme longevity and aging Ambassador and Founder of Bioquark interviews Bill Faloon, Director and Co-Founder, Life Extension Foundation and Founder of The Church Of Perpetual Life.

Ira Pastor Comments:

On the last several shows we have spent time on different hierarchical levels the biologic-architecture of the life, disease and aging process. We’ve spent some time talking about the genome, the microbiome, tissue engineering, systems biology, and dabbled a bit in the areas of quantum biology, organism hydro-dynamics, and even chronobiology.
As exciting and promising as all these research paths are, at the end of the day, in order for them to yield what many people are looking for, that is radically extended healthspans and lifespans, there needs to be an organized system of human translation build around them, integrating these various products, services and technologies, from supplements, to biologics, to functional foods, to cosmeceuticals, to various physio-therapeutic interventions, and so forth, as well as all the related supporting advocacy and education, as biologic aging is truly a multi-factorial, combinatorial process that is never going to be amenable to big pharma’s traditional “single magic bullet” philosophy that it promoted throughout the last century.

For today’s guest, I could think of no one better to talk with us about this topic and take us into the future on this front, than Bill Faloon, Director and Co-Founder, Life Extension Foundation (LEF), a consumer advocacy organization with over 100,000 members that funds research (investing million per year in researchers around the globe) and disseminates information to consumers about optimal health, and more recently in the area of actionable clinical interventions regarding human biologic age reversal, through a fascinating new project called the Age Reversal Network, defined as an open-source communications channel to exchange scientific information, foster strategic alliances, and support biomedical endeavors aimed at reversing degenerative aging.

Bill is also the Founder of The Church Of Perpetual Life, a nonprofit transhumanist organization aiming to combine discussion integrating spirituality, community, and aging scientific research in a single unified forum.

He’s a board member of the Coalition For Radical Life Extension, which is the organizer of annual RAADfest conference (Revolution Against Aging and Death) which is the world’s largest gathering of radical life extension enthusiasts.

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