StemCell Therapy

One in 14 Ontarians can expect to be diagnosed with colorectal cancer in their lifetime. COURTESY OF ED UTHMAN/FLICKR

University of Toronto scientists have identified a key protein as a common factor in the growth of many different types of colorectal cancer tumours, according to research published in the Journal of Cell Biology. Colorectal cancer develops in the colon or rectum. In Ontario, it is also the second most fatal cancer, and one in 14 Ontarians can expect to be diagnosed with this form of cancer in their lifetime.

In past research, scientists have linked the excessive accumulation of beta-catenin, a protein with crucial functions in cell development, to the expression of genes that drive tumour proliferation. Research has associated 80 per cent of colorectal cancers with gene mutations that greatly increase the production of beta-catenin.

The co-authors of the study have identified another protein, Importin-11, as the compound that enables beta-catenin transportation to the nucleus of the human cell. Cancer therapies that inhibit this transport could be a promising way to treat colorectal cancer.

Fundamental research provides new knowledge for cancer therapies

The Varsity spoke to Dr. Stephane Angers, a co-author of the study and an associate professor at U of Ts Department of Biochemistry. Angers lab has spent a considerable amount of time studying biological pathways the series of chemical changes during cellular development that give cells their final functions.

Angers noted that Monika Mis, the lead author of the study and a PhD student, uncovered the role of Importin-11 in colorectal cancer in Angers lab. Mis used the gene-editing CRISPR-Cas9 technology to screen genes in colorectal cancer calls to identify a novel gene, IPO11, which encodes for the protein Importin-11.

Current treatment options for colorectal cancer include surgery, chemotherapy, and other radiation therapy. Although this discovery is still in its fundamental stages, blocking the transport of beta-catenin holds great promise for developing new therapies.

As Angers put it, It provides new ammunition, new possibilities, and new knowledge that could lead in the future to new therapies, but it is very much at the discovery level at this point.

More research required to develop therapies

Further research could involve drug discovery and widen the scope of Importin-11 function in various cells. Researchers may also find it valuable to analyze existing data about colorectal cancer. The goal is to understand how the mutations affect tumour formation and develop therapies that harness this knowledge.

Angers lab is also investigating other potential applications of the Wnt pathway, a specific biological pathway associated with beta-catenin. A particularly interesting aspect is its role in regenerative medicine, which is the study of restoring human cells, tissues, and organs.

We think that with new molecules that we have developed we can now activate the pathway in order to promote the regenerative abilities of tissues, noted Angers.

Tags: biology, cancer, medicine, oncology

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New ammunition uncovered by U of T researchers to develop colorectal cancer treatment - Varsity

Aspire Regenerative

SAN DIEGO (PRWEB) February 27, 2020

Dr. Ryan McNally, the medical director of Aspire Regenerative, is the featured guest on Dr. Hanisha Patels natural health podcast, Mahan Health. The episode, New Year, Younger You?, focuses on the latest developments in medicine and technology that promote aging well and gracefully. It is currently available on all major podcast platforms.

During the lively and informative episode, Dr. McNally shares his expertise in the field of regenerative medicine and how it can help rejuvenate healthy tissues. The ultimate goal of these therapies is to slow down the progression of aging and prolong overall wellness. Subjects that Dr. McNally explores during the podcast include stem cell therapy, platelet-rich plasma (PRP) therapy, and products derived from fetal tissue found in cord blood, placental tissue, and perinatal fluid. One of the most promising new areas of regenerative medicine that Dr. McNally discusses is stem cell derived-exosome therapy, which uses the part of a cell that is rich in growth factors.

Dr. McNally is responsible for guiding the vision of Aspire Regenerative, a state-of-the-art facility in San Diego that delivers integrative and technologically advanced medical care. He also oversees the development and implementation of science, research, and technology at Aspire. Dr. McNally is a licensed naturopathic doctor with multiple certifications in regenerative medicine, aesthetics, and injection therapies. In addition, he is a faculty member at the Academy of Integrative Health and Medicine and an adjunct faculty member at Bastyr University California, where he formerly served as chief medical officer. A sought-after speaker at conferences and universities, Dr. McNally also publishes articles in peer-reviewed professional journals and frequently serves as a guest expert for podcasts, magazines, and newspapers.

About Aspire Regenerative: Aspire Regenerative is a state-of-the-art medical practice offering personalized regenerative treatments and integrative therapies to help patients reach their goals and achieve the best possible results. We have reinvented medical care by incorporating the principle of cell and tissue regeneration, which translates to youthful and vibrant function. Our in-depth expertise and collaboration with researchers allow us to integrate innovative technology into individualized patient care with therapies that are safe and effective. For more information, visit https://aspireregenerativehealth.com/.

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Aspire Regenerative Medical Director Featured on New Podcast - PR Web

For many people, the concept of a shorter workweek is enticing. After all, it can be difficult to find enough time for the things we love.

Is it reasonable then, in our quest for happiness, to begin working less? Advocates of a shorter workweek would agree, but these policies have yet to be widely-adopted.

Todays chart plots data from the World Happiness Report 2019 and the OECD to determine if theres any correlation between a countrys happiness and average hours worked per person.

The unhealthy side effects of working long hours are well established. In extreme cases, however, symptoms can extend beyond the usual stress and fatigue.

For example, the American Heart Association found that people under the age of 50 had a higher risk of stroke when working over 10 hours a day for a decade or more. Another study, conducted across 14 countries, concluded that people who worked long hours were 12% more likely to become excessive drinkers.

If working longer days is so harmful to our well-being, what happens if we work fewer hours instead?

The tables below list the happiest countries as well as the unhappiest countries in the OECD; happiness scores range from 0 to 10, with a 10 representing the best life possible.

Based on the data, there appears to be some degree of correlation between a persons happiness and the amount of hours they work.

Heres how the five happiest countries stack up:

The five happiest countries each work over 100 hours less than the OECD average. Compare this to the five least happiest countries:

Coincidentally, all five of the least happiest countries work more hours than the OECD average, up to over 264 hours in the case of Greece.

Happiness is multifaceted, though, and we should avoid drawing conclusions from a single variable. For instance, the World Happiness Report 2019 calculates happiness scores based on eight distinct metrics:

With these in mind, we can make a few additional observations.

Four of the five happiest OECD countries are located in the Nordics, a region known for low corruption rates and robust social safety nets. On the other end of the scale, economic hardship is a recurring theme among the OECDs least happiest countries. The falling Turkish lira and Greeces debt crisis are two significant examples.

To properly measure the happiness-boosting potential of a shortened workweek, it seems we need to isolate its effects.

Employers are now experimenting with shorter work schedules to see if happier employees are in fact better employees.

Perpetual Guardian, a New Zealand-based estate planning firm, trialed a four-day workweek for two months with no changes to compensation.

The trial was hailed as a success. Employee stress levels fell by 7 percentage points while overall life satisfaction rose by 5 percentage points. Perhaps most impressive is the fact that productivity remained the same.

Employees designed a number of innovations and initiatives to work in a more productive and efficient manner.

Helen Delaney, University of Auckland

Following the trial, the firms founder expressed interest in implementing the four-day workweek on a permanent basis.

Filimundus, a Sweden-based software studio, trialed a six-hour workday in 2014. Staff reception was positive, and the company has since adopted it permanently.

There were trade-offs, however. While staff enjoyed more time for their private lives, productivity across different departments saw mixed results.

We did see some decrease in production for some staff, mostly our artists, but an increase in production for our programmers. So money-wise, in costs, it evened out.

Linus Feldt, CEO

Interestingly, the studio also trialed a seven-hour workday, and saw no positive effects.

Public healthcare workers in Gothenburg, Sweden, trialed a six-hour workday for two years. Similar to the first case, compensation was unchanged.

While the trial achieved good resultsstaff experienced lower stress levels and patients received a higher level of carethe policy was unsustainable.

Its far too expensive to carry out a general shortening of working hours within a reasonable time frame.

Daniel Bernmar

17 additional staff were hired to compensate for the shorter workdays, increasing the local governments payroll by $738,000. The city council did note, however, that lower unemployment costs offset this increase by approximately 10%.

These experiments are garnering attention from around the world.

Even Japan, a country known for its overtime culture, is getting in on the action. Microsoft offices in the East Asian country tested a four-day workweek in August 2019, and reported happier staff, as well as an impressive 40% boost in productivity.

While the results of these early experiments are indeed promising, theyve exposed the nuances that exist between industries and job types, and the need for further trials. One thing is certain thoughshorter workweek policies should not be interpreted as a one size fits all solution for happier lives.

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Ranked: The Most Innovative Economies in the World - Visual Capitalist

VALENCIA, Calif. & MELBOURNE, Australia--(BUSINESS WIRE)--AVITA Medical (ASX: AVH) (NASDAQ: RCEL), a global regenerative medicine company, announced that management will present at the Cowen 40th Annual Health Care Conference in Boston. The presentation is being made on 4 March 2020 from 9:20 a.m. to 9:50 a.m. EST at the Boston Marriott Copley Place hotel. Interested parties may access a webcast of the presentation on the Investors section of the companys website at: https://www.avitamedical.com/investors.

Authorised for release by the Chief Financial Officer of Avita Medical Limited.

ABOUT AVITA MEDICAL LIMITED

AVITA Medical is a regenerative medicine company with a technology platform positioned to address unmet medical needs in burns, chronic wounds, and aesthetics indications. AVITA Medicals patented and proprietary collection and application technology provides innovative treatment solutions derived from the regenerative properties of a patients own skin. The medical devices work by preparing a REGENERATIVE EPIDERMAL SUSPENSION (RES), an autologous suspension comprised of the patients skin cells necessary to regenerate natural healthy epidermis. This autologous suspension is then sprayed onto the areas of the patient requiring treatment.

AVITA Medicals first U.S. product, the RECELL System, was approved by the U.S. Food and Drug Administration (FDA) in September 2018. The RECELL System is indicated for use in the treatment of acute thermal burns in patients 18 years and older. The RECELL System is used to prepare Spray-On Skin Cells using a small amount of a patients own skin, providing a new way to treat severe burns, while significantly reducing the amount of donor skin required. The RECELL System is designed to be used at the point of care alone or in combination with autografts depending on the depth of the burn injury. Compelling data from randomized, controlled clinical trials conducted at major U.S. burn centers and real-world use in more than 8,000 patients globally, reinforce that the RECELL System is a significant advancement over the current standard of care for burn patients and offers benefits in clinical outcomes and cost savings. Healthcare professionals should read the INSTRUCTIONS FOR USE - RECELL Autologous Cell Harvesting Device (https://recellsystem.com/) for a full description of indications for use and important safety information including contraindications, warnings and precautions.

In international markets, our products are marketed under the RECELL System brand to promote skin healing in a wide range of applications including burns, chronic wounds and aesthetics. The RECELL System is TGA-registered in Australia and received CE-mark approval in Europe.

To learn more, visit http://www.avitamedical.com.

CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS

This letter includes forward-looking statements. These forward-looking statements generally can be identified by the use of words such as anticipate, expect, intend, could, may, will, believe, estimate, look forward, forecast, goal, target, project, continue, outlook, guidance, future, other words of similar meaning and the use of future dates. Forward-looking statements in this letter include, but are not limited to, statements concerning, among other things, our ongoing clinical trials and product development activities, regulatory approval of our products, the potential for future growth in our business, and our ability to achieve our key strategic, operational and financial goal. Forward-looking statements by their nature address matters that are, to different degrees, uncertain. Each forward- looking statement contained in this letter is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statement. Applicable risks and uncertainties include, among others, the timing of regulatory approvals of our products; physician acceptance, endorsement, and use of our products; failure to achieve the anticipated benefits from approval of our products; the effect of regulatory actions; product liability claims; risks associated with international operations and expansion; and other business effects, including the effects of industry, economic or political conditions outside of the companys control. Investors should not place considerable reliance on the forward-looking statements contained in this letter. Investors are encouraged to read our publicly available filings for a discussion of these and other risks and uncertainties. The forward-looking statements in this letter speak only as of the date of this release, and we undertake no obligation to update or revise any of these statements.

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Research into alternative stem cell sources has identified urine derived renal progenitor cells (UdRPCs) as a possible option for use in regenerative kidney therapies in the future.

Scientists have demonstrated their protocol for the reproducible isolation of kidney stem cells from human urine. These urine derived renal progenitor cells (UdRPCs) could be used to provide easier access to stem cells for regenerative kidney therapies and modelling diseases for R&D.

A shortage of donor organs and the risks and pain associated with bone marrow stem cell extractions and third trimester amniotic fluid collection have encouraged researchers to find alternative sources of stem cells. According to scientists, several laboratories have indicated urine could be an alternative source, at least for kidney stem cells, so the researchers from Heinrich Heine University-Duesseldorf (HHU) Germany,set out to complete a comprehensive molecular and cellular analysis of these cells.

UdRPCs should be considered as the choice of renal stem cells for facilitating the study of nephrogenesis, nephrotoxicity, disease modelling and drug development

Their study, published in Scientific Reports, revealed that UdRPCs isolated from ten individuals express both markers typically seen in bone marrow-derived mesenchymal stem cells (MSCs) and renal stem cells. The renal stem cell markers, according to the paper, allow UdRPCs to be differentiated into cell types present in the kidney, eg, podocytes and the proximal and distal tubules. The study also showed that these progenitor cells have similar properties to amniotic fluid-derived stem cells (AFCs).

Wasco Wruck, bioinformatician and co-author of the study, said: It is amazing that these valuable cells can be isolated from urine and comparing all the genes expressed in UdRPCs with that derived from kidney biopies we could confirm their renal and renal progenitor cell properties and origin.

According to Martina Bohndorf, a study co-author, UdRPCs can also be easily and efficiently reprogrammed into induced pluripotent stem cells using a non-viral integration-free and safe method.

Dr James Adjaye, study senior author and professor at the Institute for Stem Cell Research and Regenerative Medicine (ISRM) in the medical faculty of HHU, revealed that one of the most promising options in the near future is the use of transplantable renal stem cells (UdRPCs) for treatment of kidney diseases as a complementary option to kidney organs. He concluded that human UdRPCs should be considered as the choice of renal stem cells for facilitating the study of nephrogenesis, nephrotoxicity, disease modelling and drug development.

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Kidney stem cells isolated from urine could be regenerative therapies - Drug Target Review

CLEARWATER, Fla., Feb. 4, 2020 /PRNewswire/ --Florida Spine Institute (FSI)is the leading, and one of the most trusted, medical facilities specializing in pain management, neurology, surgery, physical medicine and rehabilitationin Tampa Bay. FSI offers a comprehensive wellness program with a multi-disciplinary spine care team, and board-certified diagnostic, medical, and surgical specialists that provide the most advanced care available. All patient consultations and most treatments are done on a single campus.

The state-of-the-art treatment modalities offered range from physical therapy and a variety of injections to procedures including radiofrequency ablation, spinal cord stimulation implants, toKetamine treatmentsand regenerative medicinesuch as stem cell treatments. Each patient's treatment is customized for the best results.

The Florida Spine Institute has a team of elite spine, neuro, and orthopedic surgeonswho combine surgical skills with experience for the most accurate and effective treatment. Our focus is on minimally invasive spine surgical techniques, motion preservation surgery, cervical and lumbar disc replacement surgery, as well as disc restoration.

FSI offers physical medicine and rehabilitation, a branch of medicine emphasizing the prevention, diagnosis, and treatment of nerve, muscle, bone and brain disorders. The Florida Spine Institute also has a friendly and relaxed in-house MRI imaging center, saving our patients valuable time to access this sophisticated procedure.

Neurologytreats disorders of the nervous system which include the brain and spinal cord, and the peripheral nervous system. Our staff neurologistis board certified by the American Board of Psychiatry & Neurology, the American Board of Electrodiagnostic Medicine, and the American Academy of Balance Medicine. He specializes in the treatment of headache, stroke, and epilepsy.

Botox injections, an FDA-approved treatment, has been safely used for treating various medical conditions since 1989, including muscle spasms, myofascial pain, headache, and back and neck pain. Our physicians can use Botox injections in a safe and effective manner to help ease your pain.

A relatively new cutting-edge treatment, Radiofrequency Ablation (RFA), is often favored over laser spine surgery because it utilizes smaller needles, so it is less invasive and is covered by insurance. RFA is used to treat not only neck and back pain, but also hip and knee pain.

IV Ketamine Infusion Therapy is the latest breakthrough treatment that is producing extraordinary results. Ketamine blocks receptors in the brain that, when overstimulated, are responsible for releasing chemicals that cause inflammation of the nervous system. IV Ketamine treatment has been found to be very effective in treating Depression, Pain, CRPS, PTSD, Fibromyalgia, Lyme Disease and more with excellent results.

Regenerative medicine is a game-changing area of medicine with the potential to heal damaged tissues and organs, offering solutions and hope for people who have conditions that might otherwise be thought to be beyond repair. The Florida Spine Institute offers cutting-edge regenerative medicine therapies that can help you feel better. From stem cell therapy to amniotic tissue treatments, we have a solution that is customized for you.

For more information, please visit http://www.floridaspineinstitute.comor call 727-797-7463

If you have questions regarding treatments with IV Ketamine, please visit http://www.ivketamine.comor call 727-KETAMINE or 727-538-2646.

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With promising results on its first rheumatological protein therapies, Dianomi is strategically expanding upon its core technology to deliver next-generation nucleic acid therapies

MADISON, Wis., Feb. 11, 2020 /PRNewswire/ --Dianomi Therapeutics Inc. today announced that it has licensed a second suite of intellectual property (IP) from the Wisconsin Alumni Research Foundation (WARF), expanding the use of its Mineral Coated Microparticle (MCM) technology into nucleic acid therapy.

Dianomi's core MCM technology mimics the natural, inherent properties of mineralized tissues to stabilize and control the release of active drug molecules and improve their therapeutic function, thus addressing common limitations of artificial polymer-based drug delivery systems. The newly acquired IP covers compositions and methods for delivering nucleic acid-based therapies and has broad utility across nucleic acid fields, including DNA, mRNA and RNAi applications.

Developed at the University of WisconsinMadison by William Murphy, Ph.D., a UW-Madison professor of biomedical engineering and orthopedics and rehabilitation, the MCM technology in combination with nucleic acids has demonstrated favorable results, both in vitro and in vivo. In early animal studies, results of mRNA delivery indicated enhanced transfection and localized sequestration of the gene product, promising a potentially potent and sustained therapeutic effect.

"Dianomi has demonstrated success in developing and optimizing MCM delivery for biologics and other small molecules," said Murphy, co-founder and chief scientific officer of Dianomi. "I look forward to Dianomi's expansion into the area of nucleic acid therapy, building upon the early results of our nucleic acid delivery in regenerative medicine applications."

The newly licensed IP includes issued U.S. patents as well as pending U.S. and international patent applications.Dianomi retains exclusive, global rights to pursue nucleic acid therapeutics independently and to build out its commercialization and development programs with other institutions and therapeutic entities.

"This new suite of intellectual property expands the capability of Dianomi's core technology into new indications and markets having significant commercial and clinical interest," said Martin Ostrowski, chief operations officer and general counsel of Dianomi. "We're thrilled to strengthen our relationship with WARF and continue developing the platform applicability of our technological and clinical programs to improve patient care."

Dianomi's first product is a tailored interleukin-1 receptor antagonist (IL-1Ra) for osteoarthritis, which utilizes Dianomi's MCM technology to provide sustained drug delivery. Dianomi intends to develop its own internal candidates while pursuing collaborative opportunities in a number of clinical indications, including cardiovascular, rheumatological, oncology, vaccines, regenerative medicine, neuromuscular and spinal degeneration, and general health and wellbeing.

About Dianomi

Dianomi Therapeutics is a biopharmaceutical company focused on optimizing the therapeutic profile of biologics, small molecules and nucleic acids to improve patient dosing, safety and efficacy. The company is advancing a pipeline of next-generation treatments for rheumatological disease states, initially targeting osteoarthritis and pain. The company's proprietary Mineral Coated Microparticle (MCM) technology mimics the ability of human bones and teeth to store and protect biologics, and provides greatly improved, sustained delivery of active biologics and other molecules. For more information on the company, please visit http://www.dianomitx.com.

About WARF

The Wisconsin Alumni Research Foundation (WARF) helps steward the cycle of research, discovery, commercialization and investment for the University of WisconsinMadison. Founded in 1925 as an independent, nonprofit foundation, WARF manages more than 2,000 patents and an investment portfolio of $2.7 billion as it funds university research, obtains patents for campus discoveries and licenses inventions to industry. For more information, visitwarf.organd viewWARF's Cycle of Innovation.

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Dianomi Therapeutics Exclusively Licenses Nucleic Acid Technologies from the Wisconsin Alumni Research Foundation (WARF) - Yahoo Finance

The Global Stem Cells Group (GSCG) is set to sponsor the XI Congreso Mundial de Medicina Antienvejecimiento y Longevidad (World Conference of Anti-Aging and Longevity Medicine) to be held in Mexico City, Mexico on February 16-18, 2020.

The medical congress is expected to attract over 450 physicians and researchers from across the world interested in anti-aging and longevity practices and medical innovations. Over 30 speakers are slated to share information with attendees on a wide range of topics on how to lead a long, healthy life and improve longevity.

The GSCG is set to share a number of its latest innovations with congress attendees, including its newly released GCell technology device. This cutting-edge tool utilizes micrograft technology to harness the natural and powerful restorative capabilities of adipose tissues. Because it is FDA compliant, the device allows physicians across the globe to continue practicing adult stem cells-based procedures.

Additional benefits of GCell technology include shorter treatment times, delivering in-office treatments in around 30 minutes with local anesthesia, as well as less fat collection compared to existing treatments (15 mL versus 50 mL). GCell technology holds exciting implications across a range of medical specialties, including orthopedics, dermatology, cosmetic gynecology, aesthetics, and hair loss.

In addition to its GCell technology, the GSCG will also feature its newest line of stem cells products derived from first-tissue exosomes. Cellgenic Flow Exosomes utilizes the latest science and research available in cellular therapies to deliver a non-surgical approach to creating regenerative responses in a broad range of treatments. The product utilizes exosomes, which replicate the signals given out by stem cells, versus actual stem cells. Exosomes play a pivotal role in cell-to-cell communication and are involved in a wide range of physiological processes. These particles transfer critical bioactive molecules such as proteins, mRNA, and miRNA between cells and regulate gene expression in recipient cells.

The XI Congreso Mundial de Medicina Antienvejecimiento y Longevidad is one of the worlds premier events connecting physicians and researchers with todays most innovative treatments and technologies utilizing regenerative medicine, said Benito Novas, CEO of the GSCG. As a worldwide leader in training, education, and innovative products in the field of regenerative medicine, the GSCG is pleased to sponsor this congress and share its exciting new portfolio of products with physicians from across the world.

To learn more about the Global Stem Cells Group and all of the groups latest news and innovations, visit http://www.stemcellsgroup.com/

About Global Stem Cells Group

Global Stem Cells Group (GSCG) is a worldwide network that combines seven major medical corporations, each focused on furthering scientific and technological advancements to lead cutting-edge stem cell development, treatments, and training. The united efforts of GSCGs affiliate companies provide medical practitioners with a one-stop hub for stem cell solutions that adhere to the highest medical standards.

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Mexico City Medical Congress to Showcase the Global Stem Cells Group's Latest Innovations - PRUnderground

Indiana University has appointed Simran Trana as the university's first associate vice president of its Innovation and Commercialization Office.

The ICO identifies, protects and commercializes technology that comes from discoveries and innovations born from IU research. In recent years, under the umbrella of IU's Office of the Vice President for Research, the ICO has restructured to better serve IU inventors and those seeking to partner in bringing IU innovations to the public.

The new position of associate vice president reflects the university's increased focus on facilitating faculty innovation and the translation of innovations into the marketplace to serve the public. Among Trana's responsibilities will be enhancing strategic alliances with private- and public-sector partners and expanding the commercialization of IU discoveries and inventions, ensuring a service-oriented gateway for all members of the IU community seeking assistance with innovation and entrepreneurship.

Trana began her new IU role on Feb. 10.

"Having success in both industry and higher education, Simran brings the right mix of skills and experience to work with IU inventors and industry partners to shorten the time between discovery and the marketplace," said Fred H. Cate, IU vice president for research. "I am delighted that we will have the benefit of Simran's leadership to enhance delivery of the positive outcomes of IU research to Hoosiers and beyond."

Trana has 15 years of experience in product and business development, licensing and venture creation. She spent the past 10 years with Dow AgroSciences, now called Corteva Agriscience, the Agriculture Division of DowDuPont. Trana held multiple roles during this time, managing strategic research collaborations, technology licensing, intellectual property and portfolio development, new product launches, and licensing and corporate development. From 2001 to 2008, she served as director of technology commercialization for the Purdue Research Foundation, which manages and licenses intellectual property for Purdue University.

"Indiana University researchers and entrepreneurs have a long history of driving scientific innovation that impacts global progress and plays a key role in enhancing the well-being of Indiana residents and the Indiana economy," Trana said. "Knowing well that lives have and will be saved, or drastically improved, through access to new IU inventions or treatments, I am eager to get started."

Trana holds a master's degree in plant genetics from Punjab Agricultural University and a master of business administration from University of Ottawa.

She was selected for the new role at IU following a nationwide search involving university leaders:

The committee also included IU faculty inventors:

The Indiana University Innovation and Commercialization Office is tasked with the protection and commercialization of technology emanating from innovations by IU researchers. Since 1997, IU research has generated almost 3,200 inventions resulting in more than 4,800 global patent applications. These discoveries have generated more than $145 million in licensing and royalty income, including more than $115 million in funding for IU departments, labs and inventors.

Indiana University's world-class researchers have driven innovation and creative initiatives that matter for 200 years. From curing testicular cancer to collaborating with NASA to search for life on Mars, IU has earned its reputation as a world-class research institution. Supported by $680 million in 2019 from our partners, IU researchers are building collaborations and uncovering new solutions that improve lives in Indiana and around the globe.

Nicole Wilkins is executive director of research communications in the Office of the Vice President for Research.

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Simran Trana appointed associate vice president of Innovation and Commercialization Office - IU Newsroom

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Newswise A UCLA-led study reveals a new role for a gene thats associated with autism spectrum disorder, intellectual disability and language impairment.

The gene, Foxp1, has previously been studied for its function in the neurons of the developing brain. But the new study reveals that its also important in a group of brain stem cells the precursors to mature neurons.

This discovery really broadens the scope of where we think Foxp1 is important, said Bennett Novitch, a member of theEli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLAand the senior author of the paper. And this gives us an expanded way of thinking about how its mutation affects patients.

Mutations in Foxp1 were first identified in patients with autism and language impairments more than a decade ago. During embryonic development, the protein plays a broad role in controlling the activity of many other genes related to blood, lung, heart, brain and spinal cord development. To study how Foxp1 mutations might cause autism, researchers have typically analyzed its role in the brains neurons.

Almost all of the attention has been placed on the expression of Foxp1 in neurons that are already formed, said Novitch, a UCLA professor of neurobiology who holds the Ethel Scheibel Chair in Neuroscience.

In the new study published in Cell Reports, he and his colleagues monitored levels of Foxp1 in the brains of developing mouse embryos. They found that, in normally developing animals, the gene was active far earlier than previous studies have indicated during the period when neural stem cells known as apical radial glia were just beginning to expand in numbers and generate a subset of brain cells found deep within the developing brain.

When mice lacked Foxp1, however, there were fewer apical radial glia at early stages of brain development, as well as fewer of the deep brain cells they normally produce. When levels of Foxp1 were above normal, the researchers observed more apical radial glia and an excess of those deep brain cells that appear early in development.In addition, continued high levels of Foxp1 at later stages of embryonic development led to unusual patterns of apical radial glia production of deep-layer neurons even after the mice were born.

What we saw was that both too much and too little Foxp1 affects the ability of neural stem cells to replicate and form certain neurons in a specific sequence in mice, Novitch said. And this fits with the structural and behavioral abnormalities that have been seen in human patients.

Some people, he explained, have mutations in the Foxp1 gene that blunt the activity of the Foxp1 protein, while others have mutations that change the proteins structure or make it hyperactive.

The team also found intriguing hints that Foxp1 might be important for a property specific to the developing human brain.The researchers also examined human brain tissue and discovered that Foxp1 is present not only in apical radial glia, as was seen in mice, but also in a second group of neuralstem cells called basal radial glia.

Basal radial glia are abundant in the developinghuman brain, but absent or sparse in the brains of many other animals, including mice.However, when Novitchs team elevated Foxp1 function in the brains of mice, cells resembling basal radial glia were formed. Scientists have hypothesized that basal radial glia also are connected to the size of the human brain cortex: Their presence in large quantities in the human brain may help explain why it is disproportionately larger than those of other animals.

Novitch said that although the new research does not have any immediate implications for the treatment of autism or other diseases associated with Foxp1 mutations, it does help researchers understand the underlying causes of those disorders.

In future research, Novitch and his colleagues are planning to study what genes Foxp1 regulates in apical radial glia and basal radial glia, and what roles those genes play in the developing brain.

The studys first author is Caroline Alayne Pearson, a UCLA assistant project scientist. Other authors are from the University of Texas at Austin, the University of Alabama at Birmingham and the University of Puerto Rico.

The study was funded by the National Institutes of Health, the California Institute for Regenerative Medicine, the Cancer Prevention and Research Institute of Texas, the University of Texas at Austins Marie Betzner Morrow Centennial Endowment and the UCLA Broad Stem Cell Research Centers Research Award Program, including support from the Binder Foundation.

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Gene associated with autism also controls growth of the embryonic brain - Newswise

Paul Mischel (right) is one of six researchers suing the Ludwig Institute for Cancer Research for tapering down its funding.

By Michael PriceFeb. 7, 2020 , 7:10 PM

Alleging that a leading cancer funder is slashing their support in an unethical and reckless way, six prominent cancer researchers at the University of California, San Diego (UCSD), have filed a lawsuit to compel it to continue its current level of support. The suit, filed quietly in November 2019 and amended last week, contends that the Ludwig Institute for Cancer Research(LICR) is gradually drawing down its funding for cancer prevention and treatment research to the six plaintiffs, in order to close its 29-year-old San Diego branch by 2023.

In a statement, LIRC confirmed it is winding down the San Diego branchbut stressed that, [i]n implementing this decision, the Ludwig Institute is honoring its contractual obligations. LICR also said it plans to respond to the lawsuits specific allegations in due course.

The six plaintiffs, Don Cleveland, Arshad Desai, Richard Kolodner, Paul Mischel, Karen Oegema, and Bing Ren, primarily study tumor biology and cancer genomics, though some work more broadly, including Cleveland, who is also known for research on Huntingtondisease. In addition to funding from LICR, they receive substantial support from the National Institutes of Health (NIH), the California Institute for Regenerative Medicine, the Breakthrough Prize, and other sources.

LICR, a nonprofit organization based in New York City and Zurich, nowoversees nine research centers at universities and research hospitals around the world, including seven in the United States. According to figures from the institute, it has committed some $2.5 billion to cancer research since its founding in 1971. Scientists working at its research centers are co-employed as faculty members of LICR and their host institutions, with LICR partially funding the scientists work. In return, LICR earns revenue from patents and licensing agreements related to the scientists work. The San Diego branch is hosted by UCSD. According to figures cited in the lawsuit, between 2013 and 2018, LICR provided the university between $11.5 million and $13.2 million annually, including more than $3 million annually for research activities.

Jeremy Rich, a neuro-oncologist at the UCSD School of Medicine who has collaborated with LICR scientists, says the plaintiffs are the victims of a relationship between UCSD and LICR that has been in a downward spiral for years. The university, he says, doesnt see LICRas one of its own. The deteriorating relationship has fomented doubt about where the scientists loyalties lie, he says. Unfortunately for the investigators, theyre caught between two institutions, he says. It is a tragic thing for cancer research. Our enemies are not one another, but cancer.

The lawsuit, filed by six of the seven principal investigators at the branch, says that the LICR board of directors told the plaintiffs in a May 2018 meeting that it planned to close the branch at the end of 2023, when the researchers contracts end. The complaint saysLICR informed the researchers it would impose a substantially reduced level of funding beginning in 2019 and provide a tapering research budget while the scientists transitioned their research programs elsewhere. Since 2016, LICR has closed branches in Brussels;Melbourne, Australia;So Paulo;Stockholm;and Uppsala, Sweden.

By tapering their funding, the plaintiffs argue, LICR is breaching its agreement to provide future financial support for continuous, active conduct of medical research towards a cure for cancer at UCSD. The plaintiffs ask the court to make LICR continue to fundtheir research programs through 2023 at levels comparable to previous years. They also seek rights to the intellectual property they have generated, which would prevent LICR from filing patents on their work.

In addition, the scientists accuse LICR leadership of damaging their professional reputations. LICR, the lawsuit says, asserted in reckless, unjustified and unsupported public statementsthat the Plaintiffs were not performing cancer research at a level on par with their seniority and the funding.The lawsuit does not detail those statements, however.

Webster Cavenee, a former director of the LICR San Diego branch and current director of an LICR research program at UCSD for central nervous system cancers, declined to discuss the details of the lawsuit, but told ScienceInsider, the San Diego branch was measurably the most recognized and honored branch in the institute.

These scientists are renowned, says David Brenner, UCSD vice chancellor for health sciences, in a statement. They have won numerous awards and garnered significant acclaim from both their peers and the world at large. They have made major contributions in all aspects of cancer science and medicine, from basic research to clinical care, and their work is not yet done.

A UCSD spokesperson confirmed that because each of the researchers is a faculty member, termination of their Ludwig support does not terminate their UC San Diego faculty status, and they will continue to occupy the same space at university faculty. Its unclear how a closing of the branch and tapering of LICR funding might affect funding from NIH or other agencies.

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Major cancer institute sued by its own researchers over 'tapering' funding - Science Magazine

NEW YORK, Feb. 11, 2020 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics, Inc., (BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, today announced that the Company recently held a high level meeting with the U.S. Food and Drug Administration (FDA) to discuss potential NurOwn regulatory pathways for approval in ALS. Repeated intrathecal administration of NurOwn (autologous MSC-NTF cells) is currently being evaluated in a fully enrolled Phase 3 pivotal trial in ALS (NCT03280056).

In the planned meeting with senior Center for Biologics Evaluation and Research (CBER) leadership and several leading U.S. ALS experts, the FDA confirmed that the fully enrolled Phase 3 ALS trial is collecting relevant data critical to the assessment of NurOwn efficacy. The FDA indicated that they will look at the "totality of the evidence" in the expected Phase 3 clinical trial data. Furthermore, based on their detailed data assessment, they are committed to work collaboratively with BrainStorm to identify a regulatory pathway forward, including opportunities to expedite statistical review of data from the Phase 3 trial.

Both the FDA and BrainStorm acknowledged the urgent unmet need and the shared goal of moving much needed therapies for ALS forward as quickly as possible.

This is a key turning point in ourworktowardprovidingALSpatientswith a potential new therapy,said ChaimLebovits, President and CEO ofBrainStorm. We commend the FDA foritscommitmentto the ALS communityandtofacilitating the development, and we ultimately hope, the approvalofNurOwn.The entire BrainStorm team is grateful for the ongoing and conscientious collaboration in the quest to beat ALS.

Ralph Kern, MD, MHSc, Chief Operating Officer and Chief Medical Officer, stated, The entire team at BrainStorm has collectively worked to ensure that we conduct the finest, science-based clinical trials. We had the opportunity to communicate with Senior Leadership at the FDA and discuss how we can work together to navigate the approval process forward along a novel pathway. We appreciate their willingness and receptiveness to consider innovative approaches as we all seek to better serve the urgent unmet medical needs of the ALS community.

Brian Wallach, Co-Founder of I AM ALS stated: There is nothing more important to those living with ALS than having access to therapies that effectively combat this fatal disease. We have been working with BrainStorm for months now because we believe that NurOwn is a potentially transformative therapy in this fight. We were privileged to represent the patient voice at this meeting and are truly grateful to the company and the FDA for this critical agreement. This is a truly important moment of hope and we look forward to seeing both the Phase III data and the hopeful approval of NurOwn as soon as is possible.

About NurOwnNurOwn (autologous MSC-NTF cells) represent a promising investigational approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. NurOwn is currently being evaluated in a Phase 3 ALS randomized placebo-controlled trial and in a Phase 2 open-label multicenter trial in Progressive MS.

About BrainStorm Cell Therapeutics Inc.BrainStorm Cell Therapeutics Inc.is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwnCellular Therapeutic Technology Platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement as well as through its own patents, patent applications and proprietary know-how. Autologous MSC-NTF cells have received Orphan Drug status designation from theU.S. Food and Drug Administration(U.S.FDA) and theEuropean Medicines Agency(EMA) in ALS. BrainStorm has fully enrolled the Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six sites in the U.S., supported by a grant from theCalifornia Institute for Regenerative Medicine(CIRM CLIN2-0989). The pivotal study is intended to support a BLA filing for U.S.FDAapproval of autologous MSC-NTF cells in ALS. BrainStorm received U.S.FDAclearance to initiate a Phase 2 open-label multi-center trial of repeat intrathecal dosing of MSC-NTF cells in Progressive Multiple Sclerosis (NCT03799718) inDecember 2018and has been enrolling clinical trial participants sinceMarch 2019. For more information, visit the company'swebsite.

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Safe-Harbor Statement

Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could causeBrainStorm Cell Therapeutics Inc.'sactual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorms need to raise additional capital, BrainStorms ability to continue as a going concern, regulatory approval of BrainStorms NurOwn treatment candidate, the success of BrainStorms product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorms NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorms ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorms ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

CONTACTS

Corporate:Uri YablonkaChief Business OfficerBrainStorm Cell Therapeutics Inc.Phone: 646-666-3188uri@brainstorm-cell.com

Media:Sean LeousWestwicke/ICR PRPhone: +1.646.677.1839sean.leous@icrinc.com

Or

Katie Gallagher | Account Director, PR and MarketingLaVoieHealthScience Strategic CommunicationsO: 617-374-8800 x109M: 617-792-3937kgallagher@lavoiehealthscience.com

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BrainStorm Cell Therapeutics and FDA Agree to Potential NurOwn Regulatory Pathway for Approval in ALS - Yahoo Finance

10 February 2020

A study, published in Cell Reports, investigating when and how human stem cells develop into egg and sperm cells could one day help generate lab-grown gametes to treat infertility.

Human pluripotent stem cells can evolve into germ cells, which are the precursor cells for gamete development. By growing these human germ cells in vitro, the theory is that gametes engineered in a laboratory setting could someday be used, instead of natural eggs and sperm, in IVF treatment.

The research conducted within the Eli and Edythe Broad Centre of Regenerative Medicine and Stem Cell Research at University of California, Los Angeles (UCLA) provides great hope for those who are unable to produce gametes naturally,including thosewhose fertility has been affected by injury, illness or medical treatment.

'With donated eggs and sperm, the child is not genetically related to one or both parents. To treat patients who want a child who is genetically related, we need to understand how to make germ cells from stem cells, and then how to coax those germ cells into eggs or sperm'Dr Amander Clark, lead author of the study at UCLA, explained.

'Right now, if your body doesn't make germ cells, then there's no option for having a child that's biologically related to you. What we want to do is use stem cells to be able to generate germ cells outside the human body so that this kind of infertility can be overcome.'

In previous studies, scientists have been able to grow similarinduced pluripotent stem calls (iPS cells), and develop them into human skin cells and blood cells. The researchers, in collaboration with Massachusetts Institute of Technology, analysed the hundreds of thousands of genes active when both human embryonic stem cells and iPS cells transition to germ cells.

The data obtained allowed the researchers to firstly formulate when the germ cells are likely to form, which was between 24-48 hours after starting differentiation, and secondly which lineages of the differentiating stem cells give rise to the germ cells.

They also found that the activation and manifestation of germ cells was identical when developed from embryonic stem cells and iPS cells. This information was essential as they needed to ensure that the in vitro environment they had created was mimicking the molecular signals of the testis and ovaries to give hope for successful sperm and egg cell development.

Dr Clark stated: 'This tells us that the approach we're using to begin the process of making germ cells is on the right track. Now we're poised to take the next step of combining these cells with ovary or testis cells.'

Although current research is far from generating gametes, the end goal is that one day scientists are able to use a patient's skin cells to form stem cells, which can be programmed into egg or sperm cells to be used in fertility treatment.

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Lab-grown eggs and sperm a step closer - BioNews

Company signs option agreement with The Rockefeller University to access intellectual property covering compounds targeting key regeneration pathway

Decibel Therapeutics, a development-stage biotechnology company developing novel therapeutics for hearing loss and balance disorders, today announced a new strategic research focus on regenerative medicine approaches for the inner ear. The company is also announcing a collaboration and option agreement that gives Decibel exclusive access to novel compounds targeting proteins in a critical regenerative pathway.

Decibels research focus on regeneration will be powered by the companys research and translation platform. The company has built one of the most sophisticated single cell genomics and bioinformatics platforms in the industry to identify and validate targets. Decibel has also developed unique insights into regulatory pathways and inner ear delivery mechanisms that together enable precise control over gene expression in the inner ear and differentiate its AAV-based gene therapy programs.

"Our deep understanding of the biology of the inner ear and our advanced technological capabilities come together to create a powerful platform for regenerative medicine therapies for hearing and balance disorders," said Laurence Reid, Ph.D., acting CEO of Decibel. "We see an exciting opportunity to leverage this platform to address a broad range of hearing and balance disorders that severely compromise quality of life for hundreds of millions of people around the world."

The first program in Decibels regeneration portfolio aims to restore balance function using an AAV-based gene therapy (DB-201), which utilizes a cell-specific promoter to selectively deliver a regeneration-promoting gene to target cells. In collaboration with Regeneron Pharmaceuticals, Decibel will initially evaluate DB-201 as a treatment for bilateral vestibulopathy, a debilitating condition that significantly impairs balance, mobility, and stability of vision. Ultimately, this program may have applicability in a broad range of age-related balance disorders. There are currently no approved medicines to restore balance. Decibel expects to initiate IND-enabling experiments for this program in the first half of 2020.

Decibel is also pursuing novel targets for the regeneration of critical cells in both the vestibule and cochlea of the inner ear; these targets may be addressable by gene therapy or other therapeutic modalities. As a key component of that program, Decibel today announced an exclusive worldwide option agreement with The Rockefeller University, which has discovered a novel series of small-molecule LATS inhibitors. LATS kinases are a core component of the Hippo signaling pathway, which plays a key role in regulating both tissue regeneration and the proliferation of cells in the inner ear that are crucial to hearing and balance. The agreement gives Decibel an exclusive option to license this series of compounds across all therapeutic areas.

The agreement also establishes a research collaboration between Decibel and A. James Hudspeth, M.D., Ph.D., the F.M. Kirby Professor at The Rockefeller University and the director of the F.M. Kirby Center for Sensory Neuroscience. Dr. Hudspeth is a world-renowned neuroscientist, a member of the National Academy of Sciences and the American Academy of Arts and Sciences, and a Howard Hughes Medical Institute investigator. Dr. Hudspeth has been the recipient of numerous prestigious awards, including the 2018 Kavli Prize in Neuroscience.

"Rockefeller scientists are at the leading edge of discovery, and we are excited to see the work of Dr. Hudspeth move forward in partnership with Decibel," said Jeanne Farrell, Ph.D., associate vice president for technology advancement at The Rockefeller University. "The ambitious pursuit of harnessing the power of regenerative medicine to create a new option for patients with hearing loss could transform how we address this unmet medical need in the future."

In parallel with its new research focus on regenerative strategies, Decibel will continue to advance key priority preclinical and clinical programs. DB-020, the companys clinical-stage candidate designed to prevent hearing damage in people receiving cisplatin chemotherapy, is in an ongoing Phase 1b trial. Decibel will also continue to progress DB-OTO, a gene therapy for the treatment of genetic congenital deafness, which is being developed in partnership with Regeneron Pharmaceuticals. The DB-OTO program aims to restore hearing to people born with profound hearing loss due to a mutation in the otoferlin gene and is expected to progress to clinical trials in 2021.

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To support the new research focus, Decibel is restructuring its employee base and discontinuing some early-stage discovery programs.

About Decibel Therapeutics, Inc.Decibel Therapeutics, a development-stage biotechnology company, has established the worlds first comprehensive drug discovery, development, and translational research platform for hearing loss and balance disorders. Decibel is advancing a portfolio of discovery-stage programs aimed at restoring hearing and balance function to further our vision of a world in which the benefits and joys of hearing are available to all. Decibels lead therapeutic candidate, DB-020, is being investigated for the prevention of ototoxicity associated with cisplatin chemotherapy. For more information about Decibel Therapeutics, please visit decibeltx.com or follow @DecibelTx.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200129005162/en/

Contacts

Matthew Corcoran, Ten Bridge Communicationsmcorcoran@tenbridgecommunications.com (617) 866-7350

Excerpt from:
Decibel Therapeutics Announces Strategic Research Focus on Regenerative Medicine for the Inner Ear - Yahoo Finance

The recent breakthrough of regenerative immunotherapies, also known as CAR-T cell therapy, which beefs up the bodys ability to attack cancer is an example. And at theCenter for Regenerative Medicineat Mayo Clinic, a collective effort of experts involving multiple departments and divisions is driving this rapidly maturing field forward.

It sounds like science fiction.

We are dropping the fiction part, saysDr. Andre Terzic, director of the Center for Regenerative Medicine at Mayo Clinic.

Dr. Terzic underscores innovations in regenerative medicine as transformative in health care from building new tissues and organs to triggering your body to heal itself.

Lets say you cut your skin and the skin will heal on its own, says Dr. Terzic. That ability that is very preeminent with the skin is what wed like to see with other organs.

The present and future of regenerative medicine could be applied to help healheart diseaseand other vital organs, life-threatening cancer, musculoskeletal and neurological diseases and injuries, and even create new organs for transplantation.

For us, its very important to create true hope for patients, true solutions that are both verifiable, validated through many, many of the clinical studies, says Dr. Terzic.

Its a transformative view of medicine from managing patient symptoms to truly going after the root cause of the problem.

The future is remarkable. The word cures will be increasingly real, says Dr. Terzic.

Link:
Regenerative Medicine Is Transforming Health Care - South Florida Reporter

Tissue engineering combines the field of cell biology with material science in order to generate tissues and organs that may be used for regeneration, replacement or reconstruction of human bodies. In the past 10 years, there has been an exponential growth in these therapies, with great optimism and excitement about the potential effects or implications.

Since the end of the 20th century, cultured urethral mucosa cells have been used for repair of hypospadias, a congenital malformation of the urinary tract. In a survey published in 2019, tissue-engineered grafts showed even better results when used in children for primary hypospadias repair than in adults for urethral stricture repair.

Recently, a breakthrough in the surgical treatment of male urethral stricture was reported when a total of 65 patients with urethral strictures successfully were treated with MukoCell, a tissue-engineered oral mucosa transplant. With a mean follow-up of 12.1 months, recurrence was observed in only 12 patients. This corresponds to a success rate of 81.5%.About 1% of the male population suffers from strictures of the urethra.

Patients are chronically ill, with severely diminished quality of life, suffering from low urinary flow, pain, chronic urinary infections, urinary stones, urinary reflux, and damage to and failure of the urinary system. If left untreated, life-threatening urinary retention can occur.

The gold standard for urethral reconstruction is represented by the use of oral mucosa graft, with success rates reported in literature of around 80%. However, due to the complication rate at the mucosa harvest site, only a minority of operative urologists carry out this procedure.

It requires the excision of large segments of mucosa from the mouth of the patients. This severe damage to healthy tissue frequently is accompanied by multiple injuries with a significant impact on patients quality of life intraoral pain, bleeding, swelling, sensory loss and oral numbness which in many cases are persistent.

Other long term consequences include compromised oral health, scarring, chronic ulcers due to repeated bites on scar bulges, impaired lip mobility, permanent salivation, oral stenosis, facial deformities, diminished facial expressions, impaired mouth opening and impaired drinking, eating and speaking, periodontal disease; and loss of teeth and implants. One of the late consequences resulting from chronic irritation and inflammation is the increased risk of oral cancer.

Because of these risks and complications, many doctors and patients refuse this operation. Moreover, in certain situations this operation cannot be performed, such as where the patient only has a small oral cavity or limited mouth opening capacity, meaning access to the oral cavity is limited and excision of larger pieces of oral mucosa is not possible.

A significant proportion of patients are not willing to undergo the excision of oral grafts, including patients with tendency to increased scar formation, where the excision of oral mucosa is associated with risks of parafunctional bites, chronic irritation and inflammation; or patients with dentures, where the excision may lead to poorly fitting dentures or loss of dental implants. This counts even more if there is pre-existing oral mucosal damage, for example after previous removal of oral mucosa.

For other patients, the oral complications cannot be tolerated because impairment of physiognomy, oral anatomy or gustatory sensation impacts their job or social function; such as teachers, singers, politicians, actors, speakers, salespeople, cooks and musicians who play wind or brass instruments.

Tissue-engineered transplants represent the group of advanced tissue-engineered therapies (ATMPs). These are subject to EU regulation; in order to obtain market access, they must receive authorisation from the European Medicines Agency (EMA).

In order to obtain this approval, high standards must be met regarding proof of the quality, safety and efficacy of these products. Although tissue-engineered products may have a high impact on patients health, only a few of them will be approved. Tissue engineering techniques are complex and require a high standard of specialised laboratories.

Regarding quality and safety, MukoCell has already received a certificate from the EMA. MukoCell is manufactured in a state of the art cell culture factory, which has been specifically designed for engineering of tissue especially for medical use and complies with GMP guidelines for the production of pharmaceuticals. The manufacturing process starts with a tiny biopsy from the oral mucosa of the patient.

Oral mucosa is easily accessible in any patient; and biopsy under simple local anaesthesia is easy, non-invasive and painless for patients. The tissue is sent to the tissue factory where the biopsy is explanted in cell culture media. Cells are grown out and undergo a standardised aseptic manufacturing process, at the end of which, before the products are used therapeutically, strict quality and safety tests are conducted. Only if the specified quality criteria are met are the products then released for therapeutic application.

The efficacy of MukoCell has been shown in an open non-interventional study. However, to achieve market authorisation, the EMA requests that efficacy be further confirmed in a pivotal clinical study in direct comparison with native oral mucosa. This study will begin shortly and will involve a total of 200 patients, divided into two therapy groups of 100 patients each. Initial results of the study are expected by the beginning of 2023.

One goal of this clinical study is to show equivalence of the tissue-engineered product with native oral mucosa in urethral stricture treatment; the other goal is to clearly demonstrate the superiority of MukoCell over native oral mucosa as a graft, in terms of the aforementioned frequent and severe intraoral complications and impact on quality of life for patients.

The demonstration of MukoCells superiority is not only important regarding market authorisation, but also with respect to reimbursement by health insurances. The transplantation of native oral mucosa is a procedure developed by hospital surgeons. A critical examination of its safety and effectiveness has never been carried out, and complications are accepted if there is no alternative treatment.

Moreover, besides the surgical procedure which is paid for by the health insurance companies there are no additional costs associated with using native oral mucosa. In contrast, to justify additional costs arising from the use of a cultivated transplant, the efficacy, safety and superiority to native oral mucosa need to be proven.

Therefore, in the clinical trial, it is particularly important that the complications arising from excision of the transplant are recorded and documented as objectively as possible. Since the goal of surgery is to reconstruct the urethra, urologists pay little attention to intraoral complications and commonly play down their severity and importance.

Although the production of MukoCell is very complex and absolute sterility must be maintained during the three-week cultivation period, the costs are acceptable at several thousand euros. What pushes the costs even higher is the need to fulfil the requirements of the EMA in order to obtain marketing authorisation for the product: the planned clinical trial alone will cost around 10m. These costs must also be considered when pricing MukoCell.

The requirements of the regulatory authorities and health insurance companies not only influence the price of the products but also their availability. MukoCell has been on the market since 2013, but its approval is limited to Germany and only applies in a few individual cases due to the issue of reimbursement.

In a 2019 review the opinion was expressed that, due to the specificity of tissue-engineered products and the health benefits they offer, it would be advantageous to reconsider their regulatory requirements. The simplification of these requirements would allow the acceleration of these products into the market, faster availability for the patients and a decrease in the associated costs, making reimbursement less challenging for public health insurances in different countries.

Further, it was stated that the use of MukoCell represents a real, safe and efficient opportunity for patients with urethral stricture diseases. However, at present, regulatory, legal and financial issues represent important factors that restrict and slow down the wider use of MukoCell.

Soeren Liebig, CEOMukoCell GmbHBioMedizinzentrumDortmund+49 (0)23197426370s.liebig@mukocell.comwww.mukocell.com

Please note, this article will appear in issue 12 of Health Europa Quarterly, which will be available to read in February 2020.

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A milestone in the treatment of men's disease with regenerative medicine - Health Europa

The Alliance for Regenerative Medicine Outlines Recommendations on Enabling Cross-border and Regional Access to Advanced Therapy Medicinal Products (ATMPs) in Europe

BRUSSELS, BELGIUM 27 January, 2020

The Alliance for Regenerative Medicine (ARM), the international advocacy organization representing the cell and gene therapy and broader advanced therapies sector, today published a positioning paper outlining recommendations for the timely and effective access to cross-border healthcare for patients.

Todays new position paper focuses, and further elaborates, on the recommendations of ARMs July 2019 report on ensuring timely access to ATMPs in Europe (see the report here). It represents the views of the ARM members and aims to stimulate debate and reach consensus among key stakeholders, including marketing authorisation holders, payers and treatment centres, on solutions to ensure all European patients can secure access to ATMPs, irrespective of their country or region of origin.

Challenges to expanded ATMP access in Europe

ARMs key recommendations

In order to ensure that patients across Europe can access ATMPs, ARM recommends the following:

Additional recommended measures to facilitate industry engagement in existing initiatives could include: improved opportunities for cross-country collaboration, removing duplicative processes at national level, and adopting policy principles to enhance cross-country collaboration.

Janet Lambert, CEO of ARM, commented: Europe has always been a leader in ATMP innovation, both in R&D and getting products to market, however, to ensure that patients have access to these transformative treatments, there are several challenges that need to be overcome at EU, national and regional levels. This paper builds on the EU Market Access Report published in 2019 and the subsequent European stakeholder meeting in Brussels, and outlines the challenges and the recommendations that we, alongside our members, believe will most effectively get these therapies to patients in a sustainable manner.

To read the report in full, please follow this link.

Press inquiriesFor more information about the report or media requests, please contact Consilium Strategic Communications at arm@consilium-comms.com.

About the Alliance for Regenerative Medicine

The Alliance for Regenerative Medicine (ARM) is an international multi-stakeholder advocacy organization that promotes legislative, regulatory, and reimbursement initiatives necessary to facilitate access to life-giving advances in regenerative medicine worldwide. Founded in 2009, ARM works to increase public understanding of the field and its potential to transform human healthcare, providing business development and investor outreach services to support the growth of its 350+ member organizations worldwide. ARM represents the interests of therapeutic developers, academic research institutions, major medical centers, investors, and patient groups that comprise the broader regenerative medicine community and is the prominent international advocacy organization in this field.

ARM has 70+ members across 15 countries in Europe. ARM aims to work closely with European stakeholders, leveraging its membership to create a supportive commercial and regulatory environment to create better conditions for the development and commercialization of ATMPs in Europe; develop strong stakeholder support around proposed solutions to improve patient access to ATMPs; promote clear, predictable and efficient regulatory framework across Europe; and promote international convergence of key regulations and guidance. For more information, visit alliancerm.org.

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The Alliance for Regenerative Medicine Outlines Recommendations on Enabling Cross-border and Regional Access to Advanced Therapy Medicinal Products...

The approach builds on technology developed for regenerative medicine. I work in a lab that investigates ways to engineer tissue for the replacement or repair of human organs. We use some of these methods to grow meat.

The first step involves what I call a high-tech cotton-candy machine. The machine takes in a solution of water and gelatin, spins it at a high rate, and sends out nano- and microfibers that get woven together into a slab. The texture of the slab mimics that of an animals muscular tissuethe part that gives meat its texture. We then immerse the slab into a solution containing stem cells from a cow or a rabbit, where it acts as a scaffolding for the cells to cling to and grow. We use myoblastsstem cells that are already committed to turning into muscle cells. Once the solution has permeated the scaffolding, we turn the stem cells into muscle cells by tweaking the nutrients in the solution. Et voil, we have long, thin threads of muscle, like in real meat.

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Q&A: Growing Steaks in the Lab - Physics

The idea of scientists trying to grow brain tissue in a dish conjures up all sorts of scary mental pictures (cue the horror-movie music). But the reality of the research is quite far from that sci-fi visionand always will be, say researchers in the field. In fact, a leader in this area of research, Arnold Kriegstein of the University of California, San Francisco, says the reality does not measure up to what some scientists make it out to be.

In a paper published on January 29 in Nature, Kriegstein and his colleagues identified which genes were active in 235,000 cells extracted from 37 different organoids and compared them with 189,000 cells from normally developing brains. The organoidsat times called mini brains, to the chagrin of some scientistsare not a fully accurate representation of normal developmental processes, according to the study.

Brain organoids are made from stem cells that are transformed from one cell type to the another until they end up as neurons or other mature cells. But according to the Nature paper, they do not always fully complete this developmental process. Instead the organoids tend to end up with cells that have not fully transformed into new cell typesand they do not re-create the normal brains organizational structure. Psychiatric and neurodevelopmental conditionsincluding schizophrenia and autism, respectivelyand neurodegenerative diseases such as Alzheimers are generally specific to particular cell types and circuits.

Many of the organoid cells showed signs of metabolic stress, the study demonstrated. When the team transplanted organoid cells into mice, their identity became crisper, and they acted more like normal cells, Kriegstein says. This result suggests that the culture conditions under which such cells are grown does not match those of a normally developing brain, he adds. Cellular stress is reversible, Kriegstein says. If we can reverse it, were likely to see the identity of cells improve significantly at the same time.

Brain organoids are getting better at recapitulating the activities of small clusters of neurons, says Kriegstein, who is a professor of neurology and director of the Eli & Edythe Broad Center for Regeneration Medicine and Stem Cell Research at U.C.S.F. Scientists often make organoids from the cells of people with different medical conditions to better understand those conditions. But some scientists may have gone too far in making claims about insights they have derived from patient-specific brain organoids. Id be cautious about that, Kriegstein says. Some of those changes might reflect the abnormal gene expression of the cells and not actually reflect a true disease feature. So thats a problem for scientists to address.

A small ball of cells grown in a dish may be able to re-create some aspects of parts of the brain, but it is not intended to represent the entire brain and its complexity, several researchers have asserted. These organoids are no more sentient than brain tissue removed from a patient during an operation, one scientist has said.

Of course, models are never perfect. Although animal models have led to fundamental insights into brain development, researchers have sought out organoids, or organs-in-a-dish, precisely because of the limitations of extrapolating biological insights from another species to humans. Alzheimers has been cured hundreds of times in mice but never in us, for instance.

That said, the current models are already very useful in addressing some fundamental questions in human brain development, says Hongjun Song, a professor of neuroscience at the Perelman School of Medicine at the University of Pennsylvania, who was not involved in the new research. Using brain organoids, he adds, the Zika virus was recently shown to attack neural stem cells, causing a response that could explain why some babies exposed to Zika in utero develop unusually small brains.

Michael Nestor, a stem cell expert, who did not participate in the new study, says his own organoids are very helpful for identifying unusual activity in brain cells grown from people with autism. And he notes that they will eventually be useful for screening potential drugs.

Even though the models will always be a simplification, the organoid work remains crucial, says PaolaArlotta, chair of the department of stem cell and regenerative biology at Harvard University, who was also not involved in the Nature study. Neuropsychiatric pathologies and neurodevelopmental conditions are generally the result of a large number of genetic changes, which are too complex to be modeled in rodents, she says.

Sergiu Pasca, another leader in the field, says that the cellular stress encountered by Kriegstein and his team might actually be useful in some conditions, helping to create in a dish the kinds of conditions that lead to diseases of neurodegeneration, for instance. What I considerthe most exciting feature remains our ability to derive neural cells and glial cells in vitro, understanding their intrinsic program of maturation in a dish, says Pasca, an assistant professor at Stanford University, who was not part of the new paper.

The ability to improve cell quality when exposed to the environment of the mouse brain suggests that it may be possible to overcome some of the current limitations, Arlotta says. There is not yet a single protocol for making brain organoids in a lab, which may be for the best at this early stage of the field. Eventually, she says, scientists will optimize and standardize the conditions in which these cells are grown.

Arlotta, who is also the Golub Family Professor of Stem Cell and Regenerative Biology at Harvard, published a study last year in Nature showing that she and her colleagues canover a six-month periodmake organoids capable of reliablyincluding a diversity of cell types that are appropriate for the human cerebral cortex. She says it is crucial for organoid work to be done within an ethical framework. Arlotta is part of a federally funded team of bioethicists and scientists working together to ensure that such studies proceed ethically. The scientists educate the bioethicists on the state of the research, she says, and the ethicists inform the scientists about the implications of their work.

Nestor feels so strongly about the importance of linking science, policy and public awareness around stem cell research that he has put his own laboratory at the Hussman Institute for Autism on hold to accept a year-long science-and-technology-policyfellowship with the American Association for the Advancement of Science. He says he took the post to make sure the public and policy makers understand what they need to know about organoids and other cutting-edge science and to learn how to communicate about science with them.

One thing all of the scientists interviewed for this article agree on is that these brain organoids are not actual mini brains, and no one is trying to build a brain in a dish. Even as researchers learn to make more cell types and grow them in more realistic conditions, they will never be able to replicate the brains structure and complexity, Kriegstein says. The exquisite organization of a normal brain is critical to its function, he adds. Brains are still the most complicated structure that nature has ever created.

Excerpt from:
"Mini Brains" Are Not like the Real Thing - Scientific American

DUBLIN, Jan. 31, 2020 /PRNewswire/ -- Allergan plc (NYSE: AGN) today announced it will release fourth quarter and full year 2019 financial results on Monday, February 10, 2020, prior to the open of U.S. financial markets.

For additional materials related to Allergan's fourth quarter and full year 2019 financial results, please visit Allergan's Investor Relations website at https://www.allergan.com/investors.

About Allergan plc

Allergan plc (NYSE: AGN), headquartered in Dublin, Ireland, is a global pharmaceutical leader focused on developing, manufacturing and commercializing branded pharmaceutical, device, biologic, surgical and regenerative medicine products for patients around the world. Allergan markets a portfolio of leading brands and best-in-class products primarily focused on four key therapeutic areas including medical aesthetics, eye care, central nervous system and gastroenterology. As part of its approach to delivering innovation for better patient care, Allergan has built one of the broadest pharmaceutical and device research and development pipelines in the industry.

With colleagues and commercial operations located in approximately 100 countries, Allergan is committed to working with physicians, healthcare providers and patients to deliver innovative and meaningful treatments that help people around the world live longer, healthier lives every day.

For more information, visit Allergan's website atwww.Allergan.com.

Forward-Looking Statement

Statements contained in this press release that refer to future events or other non-historical facts are forward-looking statements that reflect Allergan's current perspective on existing trends and information as of the date of this release. Actual results may differ materially from Allergan's current expectations depending upon a number of factors affecting Allergan's business. These factors include, among others, the difficulty of predicting the timing or outcome of FDA approvals or actions, if any; the impact of competitive products and pricing; market acceptance of and continued demand for Allergan's products; the impact of uncertainty around timing of generic entry related to key products, including RESTASIS, on our financial results; risks associated with divestitures, acquisitions, mergers and joint ventures; risks related to impairments; uncertainty associated with financial projections, projected cost reductions, projected debt reduction, projected synergies, restructurings, increased costs, and adverse tax consequences;difficulties or delays in manufacturing; and other risks and uncertainties detailed in Allergan's periodic public filings with the Securities and Exchange Commission, including but not limited to Allergan's Annual Report on Form 10-K for the year ended December 31, 2018 and Allergan's Quarterly Report on Form 10-Q for the period ended September 30, 2019. Except as expressly required by law, Allergan disclaims any intent or obligation to update these forward-looking statements.

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