UCSD scientists find possibilities for injured brain cells to be repaired – fox5sandiego.com

SAN DIEGO (CNS) Injured adult brain cells revert to an embryonic state and become capable of re-growing new connections, which under the right conditions can help restore lost brain function, according to findings published Wednesday by researchers at UC San Diego School of Medicine.

The findings, published in the academic journal Nature, were part of a collaborative study between UC San Diego, UCLA and the University of Tennessee.

Repairing damage to the brain and spinal cord, until relatively recently, seemed an impossible task. The new study lays out a transcriptional roadmap of regeneration in the adult brain.

Using the incredible tools of modern neuroscience, molecular genetics, virology and computational power, we were able for the first time to identify how the entire set of genes in an adult brain cell resets itself in order to regenerate, said senior author Dr. Mark Tuszynski, professor of neuroscience and director of the Translational Neuroscience Institute at UC San Diego School of Medicine. This gives us fundamental insight into how, at a transcriptional level, regeneration happens.

Using a mouse model, Tuszynski and colleagues discovered that after injury, mature neurons in adult brains revert back to an embryonic state.

Who would have thought, Tuszynski said. Only 20 years ago, we were thinking of the adult brain as static, terminally differentiated, fully established and immutable.

To provide an encouraging environment for regrowth, Tuszynski and colleagues investigated how damaged neurons respond after a spinal cord injury. In recent years, researchers have significantly advanced the possibility of using stem cells to spur spinal cord injury repairs and restore lost function, essentially by inducing neurons to extend nerve fibers through and across an injury site, reconnecting severed nerves.

The latest study produced a second surprise: In promoting neuronal growth and repair, one of the essential genetic pathways involves the gene Huntingtin, which, when mutated, causes Huntington*s disease, a devastating disorder characterized by the progressive breakdown of nerve cells in the brain.

While a lot of work has been done on trying to understand why Huntingtin mutations cause disease, far less is understood about the normal role of Huntingtin, Tuszynski said. Our work shows that Huntingtin is essential for promoting repair of brain neurons. Thus, mutations in this gene would be predicted to result in a loss of the adult neuron to repair itself. This, in turn, might result in the slow neuronal degeneration that results in Huntingtons disease.

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UCSD scientists find possibilities for injured brain cells to be repaired - fox5sandiego.com

Stem cell therapies slowly gain traction as viable treatments for brain disorders – Science Magazine

Jack PriceMIT Press2020288 pp.Purchase this item now

In the late 19th century, the Spanish neuroanatomist Santiago Ramn y Cajal documented, in exquisite detail, the fantastical, uncharted landscapes of the human brain. The ornate cellular structures he drew were, according to Cajal, fragile and irreplaceable. Brain cells, he stated, may die and cannot be regenerated. Cajal then threw down the gauntlet, asserting that it was the job of the science of the future to change, if possible, this harsh decree.

Jack Prices engaging bookThe Future of Brain Repairdetails past, present, and future attempts to address Cajals formidable challenge. In so doing, it provides a vibrant and compelling guide to the important and rapidly evolving fields of stem cellbased therapies and brain repair, which together, he believes, are poised to deliver unprecedented changes to the management of brain diseases.

Unlike the diverse blood cells generated throughout life by specialized stem cells in bone marrow, the two known brain stem cell typestucked into the underside of the dentate gyrus of the hippocampus and surrounding the ventricles of the forebrain in the subependymal zonedifferentiate into a much more restricted set of cells. This intrinsic lack of versatility, coupled with the fact that brain cells are postmitotic and consequently unable to divide, underlies the brains inability to efficiently repair itself.

Two broad strategies for repair are suggested. The first would be to bypass endogenous neural stem cells by introducing non-native brain cells. The second would be to coax native cells into a different set of behaviors.

Parkinsons disease, a progressive movement disorder caused by the incremental destruction of dopaminergic neurons, has provided a fertile testing ground for the first strategy. The first human neural transplantation experiments conducted during the 1980s, which used dopamine-producing cells from human fetal brain cells or from patients own adrenal medullas, failed to meet expectations. It appeared as if not any old cells would do. Although a proof of concept for the feasibility of such approaches was provided, a more precisely defined and renewable source of donor cells was lacking, as was an accompanying robust and affordable commercial-scale manufacturing process.

In the 1990s, researchers showed that neural progenitor cells could be cultured as neurospheresballs of cells that maintain self-replication and multipotency over protracted durations. This discovery led, in 2006, to the first human neural stem cell transplant in a group of children with Batten disease, a rare neurodegenerative disorder. The results, again, were disappointing: the overall survival of treated patients showed no discernible improvement over that of untreated patients. Similar studies in patients with ischemic stroke have proved more promising, but researchers suspect improvements in these cases resulted from the grafts secretion of neuroprotective proteins rather than from cell replacement.

Two other innovations, however, have the potential to address Cajals regenerative vision. The first is our ability to culture embryonic stem cells (ES cells) to produce billions of pluripotent stem cells, which are, in turn, capable of producing every cell type in the human body. Therapies derived from ES cells have been shown to be highly effective in animal models of Parkinsons disease, with evidence suggesting that the transplanted neurons exert their effect by synthesizing dopamine.

The second innovation issues from the work of Shinya Yamanaka, who demonstrated in 2006 that terminally differentiated cells of any type can be reprogrammed into induced pluripotent stem cells (iPSCs) through the administration of just four transcription factors. iPSCs may then be converted into select cell types using different transcription factor cocktails.

Although not without complicationsextended culture of iPSCs has been shown, for example, to result in mutations in P53 and other oncogenesES cells and iPSCs have the potential to transform the science of brain repair and regenerative medicine by enabling the generation and therapeutic deployment of relevant neuronal subtypes in a scalable and low-cost manner. These cells have the additional advantage of retaining the capacity to build new tissues from scratch.

Perhaps most interesting, however, is the recent convergence of pluripotent stem cells with gene editing. Together, these technologies offer the possibility of augmenting natural neural stem cell behavior.

ACKNOWLEDGMENTSThe reviewer is a shareholder of Sangamo Therapeutics.

The reviewer is the executive vice president of research and development at Sangamo Therapeutics, Brisbane, CA, USA.

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Stem cell therapies slowly gain traction as viable treatments for brain disorders - Science Magazine

CareDx Introduces AlloCell: Cell Therapy SurveillanceCareDx Announces Collaborations to Develop Cellular Therapy Diagnostics – BioSpace

SOUTH SAN FRANCISCO, Calif., April 14, 2020 (GLOBE NEWSWIRE) -- CareDx, Inc. (Nasdaq: CDNA), a leading precision medicine company focused on the discovery, development, and commercialization of clinically differentiated, high-value healthcare solutions for transplant patients and caregivers, announced today a biopharma research partnership for AlloCell. AlloCell is a surveillance solution for patients who have received engineered-cell transplants for allogeneic cell therapy.

Allogeneic cell therapy is a rapidly growing area of clinical development with research underway for applications in oncology, cardiovascular, neurological, autoimmune, and infectious disease. In oncology, initial CAR T-cell therapies were created by genetically modifying a patients own immune cells to target specific cancer cells before transplanting them back into the patient. With allogeneic therapies, the CAR T-cells are manufactured from cells of healthy donors for off-the-shelf use in patients, simplifying the manufacturing process, and reducing patients wait time from diagnosis to treatment.

CareDx has over 20 years of expertise in transplant monitoring, enabling the development of AlloCell to monitor allogeneic cell therapies in partnership with cellular therapy companies.

Allogeneic CAR-T is the next stage of immuno-oncology therapy, and we expect it to have a transformative impact on our patients with an increasing range of indications, said Stefan Ciurea, MD, Associate Professor in the Department of Stem Cell Transplantation and Cellular Therapy from The University of Texas MD Anderson Cancer Center. However, with many different cell therapy constructs expected and variability in patients responses to therapy, there is a significant need for a standardized diagnostic measurement of cellular kinetics and persistence to help personalize treatment. AlloCell has the potential to have significant clinical utility to help manage these allogeneic cell therapy patients.

With the goal of improving transplant patient outcomes at the core of what we do, we are glad to begin cellular therapy collaborations to help patients with life-saving immune cell transplants, said Peter Maag, CEO of CareDx.

About CareDxCareDx, Inc., headquartered in South San Francisco, California, is a leading precision medicine solutions company focused on the discovery, development and commercialization of clinically differentiated, high-value healthcare solutions for transplant patients and caregivers. CareDx offers testing services, products, and digital healthcare solutions along the pre- and post-transplant patient journey, and is the leading provider of genomics-based information for transplant patients. For more information, please visit: http://www.CareDx.com.

CONTACTS:

CareDx, Inc.Sasha KingChief Marketing Officer415-287-2393sking@caredx.com

Investor RelationsGreg Chodaczek347-620-7010investor@caredx.com

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CareDx Introduces AlloCell: Cell Therapy SurveillanceCareDx Announces Collaborations to Develop Cellular Therapy Diagnostics - BioSpace

NantKwest and ImmunityBio Announce Therapeutics and Vaccines for Combatting COVID-19; Clinical Trials Anticipated to Begin This Quarter – BioSpace

EL SEGUNDO, Calif.--(BUSINESS WIRE)-- NantKwest, Inc. (NASDAQ: NK) and ImmunityBio, Inc., clinical-stage immunotherapy companies within the NantWorks family of companies, today announced they are in active discussions with the U.S. Food and Drug Administration (FDA) for vaccines and therapeutics to combat COVID-19.

Leveraging ImmunityBios expertise in vaccine development and natural killer cell activation, with a broad platform of immunomodulators currently in clinical trials for cancer and infectious diseases, and NantKwests extensive experience in off-the-shelf, cell-based therapeutics, the companies are combining their resources to design and develop therapeutics and vaccines for COVID-19.

Were in a race against time, but I am confident that, as a result of the incredible hard work the NantKwest, ImmunityBio, and the global scientific communities are undertaking, we will find effective therapeutics and vaccines against this coronavirus, said Patrick Soon-Shiong, M.D., Chairman & CEO of NantKwest and ImmunityBio.

Therapeutics:

The biological, immunological, and physiological status of the patients medical state should inform the treatment strategy to reverse the infectivity and tissue damage caused by this virus. ImmunityBio and NantKwest have developed immunomodulator regimens for COVID-19 based on the biological stage of the patients infection - from the mild, moderate to the severe or critically ill state.

In the mild-to-moderate stage of infection, we believe that the patients infection and viral load could be mitigated with natural killer (NK) and T cell stimulation. Hence, in this early-moderate stage of the disease, we are proposing clinical trials of N-803 alone, and a second trial of haNK alone, or haNK combined with convalescent plasma, said Dr. Soon-Shiong.

Investigational New Drug (IND) applications with the FDA for these trials are pending. ImmunityBios Il-15 superagonist N-803 is currently being used in clinical trials for other indications and has achieved Breakthrough Therapy Designation from the FDA[1] for the treatment of BCG-unresponsive non-muscle invasive bladder carcinoma in situ (NMIBC-CIS) patients. It has also demonstrated encouraging results in lowering the viral load in SHIV-infected monkeys[2], as announced last month at the Annual Conference on Retroviruses and Opportunistic Infections (CROI)[3].

In patients requiring ventilatory support in the severe state of COVID-19 disease, we are exploring the use of bone marrow-derived allogenic mesenchymal stem cells (BM-Allo-MSC) to mitigate the cytopathic storm, said Dr. Soon-Shiong.

NantKwest has proprietary isolation and expansion methods for growing MSCs and is using ImmunityBios automated, closed system (GMP-in-a-Box) to safely and rapidly grow these stem cells from a bone marrow cell bank in approximately 7-9 days. NantKwest has filed an IND with the FDA and anticipates beginning trials in Q2 2020.

Vaccines: Developing a platform for both initial immunizations and subsequent booster injections

First generation Adenovirus platforms (Ad5) currently in use are disadvantaged by inducing adenovirus neutralizing antibodies, thus limiting multiple doses and reducing the immune response to the antigen of interest. ImmunityBio has overcome this obstacle through the development of a second generation Ad5 platform. Through multiple deletions in the adenovirus genome, this next generation platform establishes a vector that is immunologically quiet as it relates to adenovirus protein production in the host dendritic cell and enables this same Ad5 vector to serve both as a prime and a boost treatment, even in patients with pre-existing adenovirus immunity. This second-generation Ad5 [E1-, E2b-, E3- deleted] platform has demonstrated safety in Phase I and Phase II studies in immunosuppressed cancer patients.

Furthermore ImmunityBio has extensive infectious disease experience with this second generation Ad5 platform and has published several peer-reviewed articles on studies demonstrating humoral and cell mediated immunity in H1N1 Influenza[4], HIV[5], SIV[6], Lassa Fever[7], Chikungunya, and Zika virus infections.

While development of therapies is urgently needed in this crisis, as urgent is the need to develop a vaccine with long-lasting cell-mediated immunity. Developing vaccines in the time of pandemics requires novel approaches and the use of modernized genomics, molecular dynamics, and vectors that are proven to induce cell-mediated immunity, with mass scale production capabilities. In 2009, with the H1N1 crisis, the scientific team developing this second generation Ad5 platform demonstrated that such a vaccine for the H1N1 pandemic could be developed in six weeks from identification of the H1N1 sequence. This experience in 2009 allows ImmunityBio to respond as rapidly as possible to the COVID-19 pandemic, continued Dr. Soon-Shiong. I view the spike (S) protein and the nucleocapsid (N) protein as the equivalent of a neoantigen in cancer. A recent study by the National Cancer Institute (NCI) in patients with advanced cancer, published in The Oncologist[8] reported positive evidence that this platform could induce antigen-specific T cell immunity, even in the face of previous adenoviral immunity, said Dr. Soon-Shiong. Together with our scientific collaborators at the NCI, we have recently published evidence[9] that the Ad5 platform can successfully induce cell-mediated immunity following the administration of Ad5-Neoantigens, with total remission of the tumor in pre-clinical models. Based on these findings, we are hopeful that the Ad platform could induce a similar immune response to this novel Coronavirus antigen.

About NantKwest

NantKwest (NASDAQ: NK) is an innovative, clinical-stage immunotherapy company focused on harnessing the power of the innate immune system to treat cancer and virally-induced infectious diseases. NantKwest is the leading producer of clinical dose forms of off-the-shelf natural killer (NK) cell therapies. The activated NK cell platform is designed to destroy cancer and virally-infected cells. The safety of these optimized, activated NK cellsas well as their activity against a broad range of cancershas been tested in phase I clinical trials in Canada and Europe, as well as in multiple phase I and II clinical trials in the United States. By leveraging an integrated and extensive genomics and transcriptomics discovery and development engine, together with a pipeline of multiple, clinical-stage, immuno-oncology programs, NantKwests goal is to transform medicine by delivering living drugs-in-a-bag and bringing novel NK cell-based therapies to routine clinical care. NantKwest is a member of the NantWorks ecosystem of companies. For more information, please visit http://www.nantkwest.com

haNK is a registered trademark of NantKwest, Inc.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include statements concerning or implying that NantKwest will be successful in improving the treatment of cancer. Risks and uncertainties related to this endeavor include, but are not limited to, obtaining FDA approval of NantKwests NK cells as well as other therapeutics as part of the NANT Cancer Vaccine platform as a cancer treatment.

Forward-looking statements are based on managements current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements.

These and other risks regarding NantKwests business are described in detail in its Securities and Exchange Commission filings, including in NantKwests Annual Report on Form 10-K for the year ended December 31, 2019. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

About ImmunityBio

ImmunityBio, Inc. is a privately-held immunotherapy company with a broad portfolio of biological molecules at clinical stages of development. The companys goals are to employ this portfolio to activate endogenous natural killer and CD8+ T cells in the fields of cancer and infectious disease. Specifically, ImmunityBios goal is to develop a memory T-cell cancer vaccine to combat multiple tumor typeswithout the use of high-dose chemotherapy. Regarding infectious disease, ImmunityBio is addressing HIV, influenza, and the coronavirus.

ImmunityBios first-in-human platform of technologies has enabled it to achieve one of the most comprehensive, late-stage clinical pipelines, activating both the innate (natural killer cell) and adaptive immune systems. The product pipeline includes an albumin-linked chemotherapeutic (Aldoxorubicin), a novel IL-15 cytokine superagonist (N-803), checkpoint inhibitors, macrophage polarizing peptides, bi-specific fusion proteins targeting TGFb and IL-12, adenovirus, and yeast vaccine therapies targeting tumor-associated antigens and neoepitopes.

In December 2019, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation to N-803 for BCG-unresponsive CIS non-muscle invasive bladder cancer (NMIBC). Other indications currently at registration-stage trials include BCG-unresponsive papillary bladder cancer, first- and second-line lung cancer, and metastatic pancreatic cancer.

ImmunityBios goal is to develop therapies, including vaccines, for the prevention and treatment of HIV, influenza, and the coronavirus SARS-CoV-2.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include statements concerning or implying that ImmunityBio will be successful in improving the treatment of various diseases, including, but not limited to the novel coronavirus and cancer. Risks and uncertainties related to this endeavor include, but are not limited to, the companys beliefs regarding the success, cost and timing of its development activities and clinical trials.

Forward-looking statements are based on managements current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

[1]: ImmunityBio Granted FDA Breakthrough Therapy Designation for N-803 IL-15 Superagonist in NMIBC December 4, 2019 https://www.businesswire.com/news/home/20191204005300/en/ImmunityBio-Granted-FDA-Breakthrough-Therapy-Designation-N-803

[2]: ImmunityBio Announces Durable Virus Control of SHIV Without Anti-Retroviral Therapy (ART) by Activating NK and Memorty T Cells with N-803, an IL-15 Superagonist March 10, 2020 https://immunitybio.com/immunitybio-announces-durable-virus-control-of-shiv-without-anti-retroviral-therapy-by-activating-nk-and-memory-t-cells-with-n-803-an-il-15-superagonist/

[3]: Combination IL-15 Therapy in a SHIV NHP Model Presented at Conference on Retroviruses and Opportunistic Infections (CROI) March 8-11, 2020 Boston, Massachusetts http://www.croiconference.org/sessions/combination-il-15-therapy-shiv-nhp-model

[4]: Prevention of Influenza Virus Shedding and Protection from Lethal H1N1 Challenge Using a Consensus 2009 H1N1 HA and NA Adenovirus Vector Vaccine. Vaccine. 2011 Sep 16; 29(40): 70207026. Published 2011 Aug 5. doi: 10.1016/j.vaccine.2011.07.073

[5]: Induction and Comparison of SIV Immunity in Ad5 Nave and Ad5 Immne Non-Human Primates Using an Ad5 [E1-, E2b-] Based Vaccine. Vaccine. 2011 Oct 19;29(45):8101-7. doi: 10.1016/j.vaccine.2011.08.038. Epub 2011 Aug 22.

[6]: Control of SIV Infection and Subsequent Induction of Pandemic H1N1 Immunity in Rhesus Macaques Using an Ad5 [E1-, E2b-] Vector Platform.Vaccine. 2012 Nov 26; 30(50): 72657270. Published 2012 Oct 2. doi: 10.1016/j.vaccine.2012.09.058

[7]: Adenoviral Vector-Based Vaccine is Fully Protective Against Lethal Lassa Fever Vhallenge in Hartley Guinea Pigs. Vaccine..2019 Oct 23;37(45):6824-6831. doi: 10.1016/j.vaccine.2019.09.030. Epub 2019 Sep 24.

[8]: A Phase I Trial Using a Multitargeted Recombinant Adenovirus 5 (CEA/MUC1/Brachyury)Based Immunotherapy Vaccine Regimen in Patients with Advanced Cancer. The Oncol. doi:10.1634/theoncologist.2019-0608

[9]: Efficient Tumor Clearance and Diversified Immunity Through Neoepitope Vaccines and Combinatorial Immunotherapy. Cancer Immunology Research July 2019 DOI: 10.1158/2326-6066.CIR-18-0620

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NantKwest and ImmunityBio Announce Therapeutics and Vaccines for Combatting COVID-19; Clinical Trials Anticipated to Begin This Quarter - BioSpace

Leading Urology KOLs Host Live Webinar Entitled The Protect PNS for OAB: A Wireless Uro-Stimulation Injectable Technology on April 29 at 5pm ET -…

Featuring Office Based Neuromodulation Solutions from Micron Medical

BOCA RATON, Fla., April 14, 2020 (GLOBE NEWSWIRE) -- Micron Medical, a privately-held medical device company engaged in the development, manufacture and pre-commercialization of innovative wireless, injectable electroceutical device solutions for urological conditions, today announced that it will host a webinar entitled The Protect PNS for OAB: A Wireless Uro-Stimulation Injectable Technology, on Wednesday, April 29, 2020 at 5pm Eastern Time.

Stephen Deitsch, CEO of Micron Medical, will join Larry Sirls, MD, and Ken Peters, MD, both from Beaumont Health, to discuss Micron Medical's wireless uro-stimulation injectable technology and the current treatment landscape and unmet medical need to treat patients with over-active bladder syndrome (OAB).

The Protect PNS for OAB is an investigational device based on wireless neuromodulation technology and is implanted using a minimally invasive procedure percutaneously through a needle. This technology includes a small injectable device, called a stimulator, and an external transmitter for power that is a fabric antenna placed in clothing. The Protect PNS provides therapy for the relief of OAB symptoms by delivering small controlled electrical pulses to the tibial nerve that travel to the sacral nerve plexus, the group of nerves near the pelvis that control bladder function.

Larry Sirls, MD graduated from the University of Michigan Medical School and completed urology residency training at Henry Ford Hospital. He completed specialty fellowship training in Male and Female Pelvic Reconstructive Surgery, Urodynamics, Female Urology, and Neurourology at Kaiser Permanente Medical Center in Los Angeles. He returned to Detroit in 1993 and has practiced exclusively at Beaumont Hospital Royal Oak since 1996. Dr. Sirls is the Director of Female Pelvic Medicine and Reconstructive Surgery at Beaumont Hospital Royal Oak, is a Professor at the Oakland University William Beaumont Medical School and is the Primary Investigator for the Beaumont Site of the National Institutes of Health Urinary Incontinence Treatment Network, one of only 9 centers in the country selected to perform research on treatment options for urinary incontinence. His areas of interest include reconstructive urology, male and female incontinence and voiding difficulty. He is widely published and is a national thought leader in the evaluation and treatment of male and female pelvic floor disorders. He travels to Sub-Saharan Africa yearly to perform complicated fistula and other reconstructive procedures.

Kenneth Peters, MD, is Chair of Urology at Beaumont Health System in Royal Oak, Michigan, and Medical Director of the Beaumont Womens Urology Center in Royal Oak, a unique multidisciplinary center dedicated to women with chronic pelvic pain and voiding dysfunction. Dr. Peters is also Professor and Chair of Urology at the Oakland University William Beaumont School of Medicine in Rochester, Michigan. He graduated with honors from the University of Michigan in Ann Arbor and earned his medical degree from Case Western Reserve University School of Medicine in Cleveland, Ohio. Dr. Peters subsequently completed his training in surgery and urology at William Beaumont Hospital, where he also completed a fellowship in female urology and urodynamics. A specialist in the treatment of voiding dysfunction and female urology, Dr. Peters is a well-respected clinician, educator, and researcher. He is internationally known for his work on interstitial cystitis, neuromodulation for voiding dysfunction, nerve rerouting surgery to restore voiding in patients with spina bifida, and adult human stem cell injection for stress urinary incontinence. He has published over 100 peer-reviewed articles and book chapters. He has twice won the Society for Urodynamics and Female Urology (SUFU) clinical research award. An active member of many professional organizations, Dr. Peters has served on the Research Council of the American Urological Association. At the 2012 annual meeting of the International Pelvic Pain Society, he was a keynote speaker on the management of chronic pelvic pain. He maintains a private practice in Royal Oak.

About Micron MedicalMicron Medical is a privately held medical device company engaged in the development, manufacture, and pre-commercialization of wirelessly powered, microtechnology neurostimulators, providing patients with convenient, safe, minimally invasive, and highly cost-effective urological solutions that are easily incorporated into their daily lives. Microns goal is to evolve its patented, cutting-edge platform for neuromodulation to standard of care, increasing the accessibility for patients worldwide while lowering the economic impact of urology care management. http://www.micronmed.com

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Leading Urology KOLs Host Live Webinar Entitled The Protect PNS for OAB: A Wireless Uro-Stimulation Injectable Technology on April 29 at 5pm ET -...

Young-onset Parkinson’s may start in the womb – Health24

People who develop Parkinson's disease at a younger age (before age 50) may have malfunctioning brain cells at birth, according to a study that also identified a drug that may help these patients.

At least 500 000 people in the United States are diagnosed with Parkinson's each year. Most are 60 or older at diagnosis, but about 10% are between 21 and 50.

Parkinson's is a neurological disease that occurs when brain neurons that make dopamine become impaired or die. Dopamine helps coordinate muscle movement.

Symptoms get worse over time and include slow gait, rigidity, tremors and loss of balance. There is currently no cure.

"Young-onset Parkinson's is especially heart-breaking because it strikes people at the prime of life," said study co-author Dr Michele Tagliati, director of the Movement Disorders Program at Cedars-Sinai Medical Center in Los Angeles.

"This exciting new research provides hope that one day we may be able to detect and take early action to prevent this disease in at-risk individuals," he said in a hospital news release.

For the study, Tagliati and colleagues generated special stem cells from the cells of patients with young-onset Parkinson's disease. These stem cells can produce any cell type of the human body. Researchers used them to produce dopamine neurons from each patient and analysed those neurons in the lab.

The dopamine neurons showed two key abnormalities: build-up of a protein called alpha-synuclein, which occurs in most forms of Parkinson's disease; and malfunctioning lysosomes, structures that act as "trash cans" for the cell to break down and dispose of proteins. This malfunction could result in a build-up of alpha-synuclein, the researchers said.

"Our technique gave us a window back in time to see how well the dopamine neurons might have functioned from the very start of a patient's life," said senior author Clive Svendsen, director of the Cedars Sinai Board of Governors Regenerative Medicine Institute.

"What we are seeing using this new model are the very first signs of young-onset Parkinson's," Svendsen said in the release. "It appears that dopamine neurons in these individuals may continue to mishandle alpha-synuclein over a period of 20 or 30 years, causing Parkinson's symptoms to emerge."

The study was published in the journal Nature Medicine.

The researchers also tested drugs that might reverse the neuron abnormalities. A drug called PEP005 already approved by the US Food and Drug Administration for treating pre-cancers of the skin reduced elevated levels of alpha-synuclein both in mice and in dopamine neurons in the lab.

The investigators plan to determine how PEP005, which is available in gel form, might be delivered to the brain to potentially treat or prevent young-onset Parkinson's.

They also want to find out whether the abnormalities in neurons of young-onset Parkinson's patients also exist in other forms of Parkinson's.

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Young-onset Parkinson's may start in the womb - Health24