Tag Archives: Therapeutics

Organovo Collaborates With Professor Melissa Little for Kidney Tissue Research

transforming treatment_cover

SAN DIEGO and MELBOURNE, Australia and SPRINGFIELD, Va., Jan. 24, 2017 (GLOBE NEWSWIRE) — Organovo Holdings, Inc. (NASDAQ:ONVO) (“Organovo”), a three-dimensional biology company focused on delivering scientific and medical breakthroughs using its 3D bioprinting technology, today announced a collaboration with Professor Melissa Little and the Murdoch Childrens Research Institute, The Royal Children’s Hospital, Melbourne, Australia to develop an architecturally correct kidney for potential therapeutic applications.  The collaboration has been made possible by a generous gift from the Methuselah Foundation (“Methuselah”) as part of its ongoing University 3D Bioprinter Program.

“Partnerships with world-class institutions can accelerate groundbreaking work in finding cures for critical unmet disease needs and the development of implantable therapeutic tissues,” said Keith Murphy, CEO, Organovo.  “This collaboration with Professor Little’s lab is another important step in this direction.  With the devoted and ongoing support of the Methuselah Foundation, leading researchers are able to leverage Organovo’s powerful technology platform to achieve significant breakthroughs.”

“We have developed an approach for recreating human kidney tissue from stem cells,” said Professor Melissa Little, Theme Director of Cell Biology at Murdoch Childrens Research Institute.  “Using Organovo’s bioprinter will give us the opportunity to bioprint these cells into a more accurate model of the kidney.  While initially important for modelling disease and screening drugs, we hope that this is also the first step towards regenerative medicine for kidney disease.  We are very grateful to Organovo and the Methuselah Foundation for this generous support, which will enable us to advance our research with the first Organovo bioprinter in the southern hemisphere.”

Under Methuselah Foundation’s University 3D Bioprinter Program, Methuselah is donating at least $500,000 in direct funding to be divided among several institutions for Organovo bioprinter research projects.  This funding will cover budgeted bioprinter costs and key aspects of project execution.

“We at the Methuselah Foundation have been a long-time supporter of academic and industry research in 3D bioprinting, regenerative medicine, and tissue engineering,” said David Gobel, CEO, Methuselah Foundation. “Our University 3D Bioprinter Program puts Organovo’s breakthrough 3D bioprinting technology in the hands of the brightest scientists at tissue engineering centers of excellence.”

About Organovo Holdings, Inc.

Organovo designs and creates functional, three-dimensional human tissues for use in medical research and therapeutic applications.  The Company develops 3D human tissue models through internal development and in collaboration with pharmaceutical, academic and other partners.  Organovo’s 3D human tissues have the potential to accelerate the drug discovery process, enabling treatments to be developed faster and at lower cost.  The Company’s ExVive Human Liver and Kidney Tissues are used in toxicology and other preclinical drug testing.  The Company also actively conducts early research on specific tissues for therapeutic use in direct surgical applications.  In addition to numerous scientific publications, the Company’s technology has been featured in The Wall Street Journal, Time Magazine, The Economist, Forbes, and numerous other media outlets.  Organovo is changing the shape of life science research and transforming medical care.  Learn more at www.organovo.com.

About Murdoch Childrens Research Institute

Murdoch Childrens undertakes research into infant, child and adolescent health.  As the largest child health research institute in Australia, our 1500 researchers are working hard to translate the knowledge we create from our research into effective prevention, early intervention and treatments for children.  We strive for a healthier community, fewer sick kids visiting hospitals, and the best possible care for children who unfortunately become ill.  The Murdoch Childrens has a proud history of scientific discovery since its inception in 1986, and is currently based at The Royal Children’s Hospital in Melbourne, Australia.  For more information please visit: www.mcri.edu.au.

About Methuselah Foundation

The Methuselah Foundation is a non-profit medical charity working to create a world where 90 year olds can have the health profile of 50 year olds, by 2030.  By opportunistically leveraging resources, enabling partnerships, and awarding prizes and grants, we accelerate disruptive developments in biomedical engineering that will eradicate needless suffering and extend healthy human life.  For more information please visit: www.methuselahfoundation.org and www.neworgan.org.

Scientists Correct Mutated Gene that Causes Sickle Cell Disease in Stem Cells

baby-sickle-vell

For the first time, scientists were able to correct the genetic mutation that causes sickle cell disease in stem cells.

In a collaborative effort, researchers at UC Berkeley, UC San Francisco Benioff Children’s Hospital Oakland Research Institute (CHORI), and the University of Utah School of Medicine fixed the mutation in modified stem cells from patients with the condition using a CRISPR/Cas9 gene editing approach.

The study, “Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells,” was published in the journal Science Translational Medicine.

The scientists hope to re-infuse patients with the modified stem cells and alleviate disease symptoms.

“We’re very excited about the promise of this technology,” Jacob Corn, senior author on the study and scientific director of the Innovative Genomics Initiative at UC Berkeley, said in a news release. “There is still a lot of work to be done before this approach might be used in the clinic, but we’re hopeful that it will pave the way for new kinds of treatment for patients with sickle cell disease.”

The researchers observed in mice tests that after transplants, the modified stem cells stuck around for about four months, an important target of the long-lasting potential of any therapy.

“This is an important advance because for the first time we show a level of correction in stem cells that should be sufficient for a clinical benefit in persons with sickle cell anemia,” said Mark Walters, a pediatric hematologist and oncologist and director of UCSF Benioff Oakland’s Blood and Marrow Transplantation Program and co-author of the study.

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These companies search for a cure to aging– and their discoveries are amazing

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The ideas surrounding life enhancement are not new—in fact, records show an interest in the mysteries surrounding human life for centuries.

Mary Shelley’s Frankenstein explores the idea of creating a life, while Doctor Who achieves life extension through regeneration. Wolverine’s mutations let him heal away his problems, and Captain America froze himself into the 21st century. Just look at almost any Star Trek episode and you’ll see how fascinated people are with the idea of extending life.

These ideas are starting to extend beyond science fiction. What was once seen as fiction is, in fact, highly relevant in today’s scientific community. Life extension research lives in academia at the moment, but it’s also graining traction in nonprofit foundations and national organizations.

This scientific field aims not only to discover the solutions to life’s unanswered aging questions, but also allow humanity to “live long and prosper.”

Why bother with this research?

The “holy grail” of the life extension industry is the cure to aging (obviously) and its discovery would change the course of human history forever.

However, when looking at life extension from the viewpoint of the Average Joe, there are many very real, personal, and emotional reasons which can be tied to the desire for those extra years.

“Seeing friends and family age can be difficult to go through,” said Dr. Chris Barton, assistant professor of biology at Belmont University in Nashville, TN. “As a result, I think that we are becoming more conscious of the aging process and more intentional about trying to find ways to delay it.”

When did life extension research really begin?

 

calico logo

Hopefully Calico doesn’t go the way of Google Glass. (Credit: Calico)

Interest in life extension has existed for decades– one of the largest booms in life extension research began in the 1990s. In 1992, The American Academy of Anti-Aging Medicine was established to explore the mysteries behind our bodies’ aging process.

From then the new millennium began, and with it came companies such as the Methuselah Foundation, co-founded by Dave Gobel and Dr. Aubrey de Grey in 2003, and through its leadership came the “Strategies for

Engineered Negligible Senescence,” or SENS Research Foundation, founded in 2009. In 2013, Google announced its new company Calico, who under the leadership of Arthur D. Levinson would focus on human health in relation to aging and its associated diseases.

“Nothing breeds success better than success,” Dr. Barton explained when reviewing the recent boom in anti-aging research out of these foundations. “While many of these advancements are in basic science research, it is really this foundational understanding of aging that has allowed us to detect and treat numerous aging-related diseases.

“If you look at the life expectancy data from 1960 to today, people are clearly living longer”—life expectancy in the United States alone jumped from age 70 in 1960 to age 79 in 2014, according to The World Bank. “We are currently more effective in treating conditions such as cardiovascular disease, cancer, and other aging-related diseases than we were 30 years ago. I think the recent success we’ve had in these areas is developing an excitement for aging research that can perhaps generate discoveries and technologies that may even further extend our life expectancy,” he continued.

The different areas of life extension research

These companies challenge current researchers and scientists to study the mysteries surrounding aging. The Methuselah Foundation focuses on Organovo and the ability to 3D print functional human tissues with hopes of creating functioning organs, while the SENS Research Foundation focuses on rejuvenation biotechnologies with new therapies which target and repair molecular damage responsible for the body’s aging.

With a Ph. D. in Biochemistry from Vanderbilt University and specializations in physiology, cell biology, and molecular genetics, Dr. Barton was able to provide insight into one of the many areas of research currently being studied among those in the field of life extension and anti-aging.

“Perhaps one of the most popular views behind the aging process is the ‘stem cell theory of aging,’ which states that as we age, our stem cells aren’t able to continue dividing to replenish the cells that are being lost in our tissues and organs,” Dr. Barton explained, believing this to be an area of research holding great promise.

“In addition, every time a cell divides there is the potential for it to accumulate some type of damage to its DNA. Given that stem cells must divide over an entire lifetime, they tend to accumulate quite a bit of damage. It is really the inability of our stem cells to continue growing indefinitely that many believe is the root of the aging process. Without a healthy pool of stem cells, tissues and organs are no longer able to maintain themselves in a way that supports life.”

 

human cell cross section

Learning how to reverse cell damage could be the key to reversing aging. (Credit: Thinkstock)

The hope for researchers is to promote the field and provide the world with a hope for advancement and, one day, a solution.

“Scientific progress, particularly in academia, is most often hindered by the decreases in government funding,” Dr. Barton said. “When large organizations such as these are willing to contribute funds or resources in order to advance research on a specific topic, I think they immediately become relevant to the larger research community.”

And, in the case of anti-aging and life extension research communities, the relevancy of their research extends much further than that in everyday culture, aging treatments, diseases associated with aging, life expectancy, and the overall quality of life every single person will one day encounter with age.

So why haven’t we found the solution to aging yet?

Read more at http://www.redorbit.com/news/technology

Making memories stronger and more precise during aging

Young neurons (pink), responsible for encoding new memories, must compete with mature neurons (green) to survive and integrate into the hippocampal circuit. Photo courtesy of Kathleen McAvoy, Sahay Lab.
Young neurons (pink), responsible for encoding new memories, must compete with mature neurons (green) to survive and integrate into the hippocampal circuit. Photo courtesy of Kathleen McAvoy, Sahay Lab.

HSCI researchers identify new mechanisms by which new neurons sharpen memories

By Hannah L. Robbins, HSCI Communications

When it comes to the billions of neurons in your brain, what you see at birth is what get — except in the hippocampus. Buried deep underneath the folds of the cerebral cortex, neural stem cells in thehippocampus continue to generate new neurons, inciting a struggle between new and old as the new attempts to gain a foothold in the memory-forming center of the brain.

In a study published online today in Neuron, Harvard Stem Cell Institute (HSCI) researchers atMassachusetts General Hospital and the Broad Institute of MIT and Harvard in collaboration with an international team of scientists found they could bias the competition in favor of the newly generated neurons.

“The hippocampus allows us to form new memories of ‘what, when and where’ that help us navigate our lives,” said HSCI Principal Faculty member and the study’s corresponding author, Amar Sahay, PhD, “and neurogenesis—the generation of new neurons from stem cells—is critical for keeping similar memories separate.”

As the human brain matures, the connections between older neurons become stronger, more numerous, and more intertwined, making integration for the newly formed neurons more difficult. Neural stem cells become less productive, leading to a decline in neurogenesis. With fewer new neurons to help sort memories, the aging brain can become less efficient at keeping separate and faithfully retrieving memories.

The research team selectively overexpressed a transcription factor, Klf9, only in older neurons in mice, which eliminated more than one-fifth of their dendritic spines, increased the number of new neurons that integrated into the hippocampus circuitry by two-fold, and activated neural stem cells.   READ MORE

Methuselah Foundation Fellowship Award Winner Tackles Research in Macular Degeneration 

Jennifer Rosa small

Typically, a fellowship and participation in a research study to cure a major disease would occur years after completing undergrad, possibly even after earning a PhD. But Jennifer DeRosa is not a typical student.

As early as high school, DeRosa was already in the lab, conducting research in plant biotechnology at the College of Environmental Science and Forestry (SUNY-ESF) before graduating valedictorian from Skaneateles High School. As a freshman student at Onondaga Community College, she continued to develop skills in molecular biology, analytical chemistry, and cell biology. She logged over 1,600 hours in academic and industry laboratories while maintaining a perfect 4.0 GPA, completing her associate’s degree in Math and Science in only one year.

Although she had planned to continue to a bachelor’s program, DeRosa elected to defer enrollment after being offered a Methuselah Foundation research fellowship. “The fellowship provides distinguished students a year-long stipend to work in any laboratory of their choosing that conducts work on age-associated diseases,” said Methuselah Foundation CEO David Gobel. “We are very pleased that she chose to complete her fellowship at Ichor Therapeutics, where she has been working as a paid intern. Methuselah Foundation has a high degree of confidence in the quality and scope of work being conducted there.”

Her enthusiasm for her work has caught the attention of everyone who works with her. “Jennifer [DeRosa] has distinguished herself at every level since beginning as an intern in January,” stated Ichor’s Quality Assurance Director Scott Campbell. “We are delighted about her decision to stay on and help us drive our age-related macular degeneration program into the next stage of development, including adopting of stringent GMP and GLP regulatory requirements.”

DeRosa is excited about the research that Ichor Therapeutics is currently engaged in, as well as the opportunities to learn in areas beyond the science itself. She said, “I chose to intern at Ichor because as a startup, I knew it would allow me to explore entrepreneurship and take on a greater role than I otherwise could at a large company. Between being able to participate in board meetings, discuss legal and translational strategy with Ichor’s counsel and advisory teams, and meeting the company’s investors to better understand their expectations – Let’s just say it was a simple decision for me to remain here.”

DeRosa’s previous research at Ichor substantially and directly contributed to the company successfully raising $600,000 for its macular degeneration program earlier this summer. DeRosa was a listed author on both the research proposal and business plan, and is also listed on two pending grant applications.

Kelsey Moody, CEO at Ichor Therapeutics, noted, “The most difficult part of having her here is finding sufficient challenges. She has earned complete autonomy since her arrival. Beyond her expansive laboratory skills, she has designed her own studies, written proposals for grants, and led a small team to develop product leads for the macular-degeneration program.”

When her fellowship draws to a close, DeRosa intends to pursue a bachelor’s degree or matriculate directly into a graduate program. However, she plans to remain opportunistic. “The pace, progress, and potential impact of Ichor’s macular degeneration program is addicting. The company’s main focus now is to prepare for series A, after which, who knows what opportunities may present themselves.”