Leading the Movement Towards Direct Cell Conversion: An Interview With Mogrify – Technology Networks

Biotech company Mogrify is deploying its proprietary direct cellular conversion technology to develop cell therapies in a variety of disease areas, including auto-immune, musculoskeletal, respiratory diseases and in cancer immunotherapy.The platform utilizes data from next-generation sequencing and cellular networks to identify transcription factors or small molecules required to directly convert a cell, addressing key challenges that are typically associated with the safety and efficacy of cell therapies.Technology Networks recently spoke with Joe Foster, COO, Mogrify, to learn more about the platform, the challenges encountered in developing cell therapies, and to gain Mogrify's insights on the future of this exciting research space.Molly Campbell (MC): What were some of the major highlights for Mogrify in 2019?Joe Foster (JF): In the past year, Mogrify has solidified its reputation as a pioneer in the expanding field of cell therapy. Using a systematic, data-driven approach, our innovative cell conversion platform addresses many of the challenges impeding systematic discovery, process development and the manufacturing processes.At an operational level, Mogrify has seen unprecedented growth in the last year, with emphasis on world-class science. We have established a leadership team with unparalleled track records, including the appointment of Dr Darrin M. Disley OBE, as CEO and Dr Jane Osbourn OBE, as Chair. Looking forward and with plans to boost our team to 70 individuals working across all disciplines, Mogrify has also moved operations to the new Bio-Innovation Centre in Cambridge, giving our team access to state-of-the-art facilities to continue their work in developing novel approaches to cell therapy.Mogrify received MSDs Innovation Award at the 15th Annual Scrip Awards, in acknowledgement of our potential to transform future cell therapies. Dr Jane Osbourn OBE was also the first female to win the Lifetime Achievement Award, recognizing her significant contributions to the biotech industry. Mogrifys significant fundraising success was also marked at the prestigious European Lifestars Awards, which celebrates excellence in the life science industry. Here, Mogrify was recognized as the Seed Stage Finance Raise of the Year.MC: In Mogrify's opinion, what key trends can we expect to see in the cell therapy space in 2020?JF: Many of the current approaches to cell therapy involve first converting cells back into a stem cell-like stateinduced pluripotent stem cellsbefore then converting them into the cell type required.Mogrify plans to lead the movement towards direct cell conversion, or transdifferentiation, where cells can be transformed from one cell type to another, without having to go through an intermediate pluripotent stage. Direct conversion of cells would enhance the speed of cell therapy development, as cells do not need to use traditional developmental pathways to reach a mature state.

Another bottleneck in the delivery of cell therapies is that most approaches rely on autologous transplants, which are carried out using patient-derived cells. Future innovations are moving towards using allogeneic therapies, where the cells used for therapy are derived from a healthy donor. Such advances are paving the way towards the development of universal donor cells, which would turn cell therapies into off-the-shelf treatments, enhancing the scale and accessibility of the treatments.

Finally, cell therapy methods are likely to move from ex vivo approaches (where cells are isolated from the patient, reprogrammed, and delivered back into the patient), to in vivo approaches, where cell therapies are delivered directly to the recipient, for example, through the use of small molecules present in a reprogramming cocktail or direct gene editing. In vivo technologies would, therefore, be able to reprogram cells directly in living humans, expanding the scope of cell therapy in a clinical setting. Overall, future cell therapies will have the capacity to be more effective, safer, and widely accessible.

MC: What are the key challenges that currently exist when developing and testing cell therapies? How does Mogrify hope to overcome such challenges?JF: The biggest challenges in producing cell therapies surround the efficacy, safety profile, and scalability of clinical treatment regimes. To make treatments safer, delivered cells must bypass the host immune system. This can be achieved with autologous therapy, but comes at the cost of scale and efficiency, as the patients cells need to be extracted, cultured, and reprogrammed before treatment can be delivered. Genetic engineering technologies (such as CRISPR/Cas9) that can be employed to remove the antigenic potential of allogeneic cell therapies (e.g. CAR-T) can be used in conjunction with such treatments, but this brings an additional layer of complication.Another difficulty comes from the technical challenges associated with generating, culturing, and expanding the required cells. In theory, any cell type can be derived from pluripotent cells. However, determining precisely how to generate any cell from pluripotent cells is conceptually and practically complex. Each cell type would require a distinct combination of transcription factors (or small molecules) and optimized culture conditions to ensure robust conversion into the desired phenotype. These technical challenges are associated with slow progress and poor efficiency in deriving reliable therapeutic cells.

Mogrify aims to tackle these hurdles with solutions involving big data, computational predictions, and bioinformatics. Mogrifys proprietary algorithm uses next generation sequencing data to predict the combination of transcription factors necessary to reliably convert any cell type into another cell type. Mogrifys technology provides a framework for direct cell conversion, and can also identify the best culture conditions to ensure that the cell populations remain stable and viable. This greatly improves cell therapy efficiency, as mature cells are created without the often arduous and imprecise process of differentiating cells from pluripotency.

Mogrifys technology is also compatible with in vivo cell therapies, as it can identify a combination of small molecules that will drive the necessary transcriptional networks to create the cells of choice. Therefore, Mogrifys technology can also be applied to overcome safety issues associated with allogeneic ex vivo approaches, and has the potential to greatly enhance the scale at which cell therapies can be delivered.MC: Are you able to tell us more about the latest developments in Mogrify's pipeline?JF: Currently, Mogrify is focused on applying the platform to musculoskeletal disorders, cancer immunotherapy, and auto-immune, ocular and respiratory diseases. Specifically, Mogrify is committed to identifying opportunities in regenerative medicine contexts, where direct cell conversion could have strong therapeutic potential.The current lead musculoskeletal program is in the development of chondrocytes for the treatment of cartilage defects and osteoarthritis. Mogrifys platform identified a cocktail of small molecules that successfully drives the conversion of fibroblast cells to chondrocytes, which has been proven to form functional hyaline cartilage in vitro. This can even be performed using an allogeneic approach without the need for gene editing (as the cartilage is immunopriviliged). Thus, it represents an opportunity for an off-the-shelf therapy that could be a relatively inexpensive and accessible treatment. At present, this treatment is in pre-clinical stages, and has a powerful potential for success in regenerative cartilage therapy. Similarly, an in vivo method of transdifferentiating osteoarthritic chondrocytes to healthy cells is being investigated in ongoing studies using a cocktail of small molecules.

Joe Foster, COO, Mogrify was speaking to Molly Campbell, Science Writer, Technology Networks.

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What exactly are underlying conditions? And why people with them may experience more serious illness from coronavirus – Boston News, Weather, Sports |…

(CNN) Weve heard that elderly people and those with underlying health conditions are most at risk if theyre infected with coronavirus, but those can seem like really general terms. Who does that include? And why can they face more serious illness?

According to the [Centers for Disease Control and Prevention], some of the underlying conditions that may put you at higher risk include: chronic lung disease and asthma, heart disease and undergoing cancer treatment, said CNN Chief Medical Correspondent Dr. Sanjay Gupta in anepisode of CNNs Coronavirus: Fact vs. Fiction podcast. Anyone with diabetes, kidney failure or liver failure may also be at higher risk.

The role of the immune system is to protect against disease or other potentially damaging pathogens. A strong one is needed to help stave off coronavirus infection.

Think of it like this, Dr. Gupta suggested. In your everyday life, youre always fighting off pathogens. Most of the time you dont even realize it. If you have an underlying condition, it makes it more challenging to fight off a virus like this. You may develop a fever, shortness of breath or a cough more easily than someone who doesnt have a preexisting illness.

Additionally, there are more specific reasons why each condition has its own vulnerabilities. Heres a guide to underlying conditions affected by coronavirus and why, and how you can protect yourself or an at-risk loved one.

Eight out of 10 deaths reported in the US have been in adults ages 65 and older, according to theCDC. Older adults have also been more likely to require hospitalization and admission to an intensive care unit.

Older adults are more likely to have long-term health problems that can increase their risk for infection and serious disease. And, our immune systems usually weaken with age, making it more difficult for people to fight off infections, according toJohns Hopkins Medicine.

The quality of our lung tissue also declines over time, becoming more elastic and making respiratory diseases such as Covid-19 of important concern because of the potential for lung damage.

Inflammation in older adults can be more intense, leading to organ damage.

People with chronic airway and lung diseases such as chronic obstructive pulmonary disease (COPD), asthma, pulmonary fibrosis and interstitial lung disease can lay the foundations for more severe infection with coronavirus because of the inflammation, scarring and lung damage those conditions cause,Johns Hopkins Medicine reported.

Covid-19 affects a persons airway and lungs, but those organs work together to provide the body with oxygen. When the lungs are overburdened with an infection, the heart has to work harder, which exacerbates the challenges of people already living with heart disease.

According to the CDC, many conditions can cause a person to be immunocompromised, including cancer treatment, smoking, bone marrow or organ transplantation and immune deficiencies. Poorly controlled HIV or AIDS and prolonged use of man-made steroid hormones or otherimmune-weakening medicationscan also hamper a persons immune function.

Cancer can weaken immunity by spreading into the bone marrow, which makes blood cells that help fight infection, according toCancer Research UK. Cancer prevents bone marrow from making enough blood cells.

Some cancer treatments can temporarily weaken the immune system, too. Because cancer treatments such as chemotherapy, cancer drugs, radiotherapy or steroids are targeted toward cancer cells, they can also diminish the number of white blood cells created in the bone marrow.

A2017 studyfound cigarette smoking can harm the immune system by either causing extreme immune responses to pathogens or rendering the body less effective at fighting disease. This may occur by smoking, negatively altering the cellular and molecular mechanisms responsible for keeping an immune system strong.

When a person undergoes a bone marrow transplant using stem cells from a donor, or they receive an organ, a doctor may prescribe medications to prevent graft-versus-host disease andmitigate the immune systems reactionby suppressing its function. After the operation, it takes time for your immune system to be up and running again.

HIV and AIDS attack the bodys immune system, specifically the bodys T cells, which help the immune system fight off infection. When the diseases are untreated, HIV reduces the number of those cells, making the person more likely to contract other infections or infection-related cancer, according to theCDC.

People with severe obesity, or a body mass index of 40 or higher, are athigher risk of serious disease.

Obesity shares with most chronic diseases the presence of an inflammatory component, a2012 studysaid. Inflammatory responses were linked between the immune system and body fat. Obesity is known to impair immune function by altering white blood cell count as well as the cells that control immune responses.

People with type 1 or type 2 diabetes face an increased risk of getting really sick with Covid-19, as both cause a blood sugar spike. If blood sugar is poorly managed, viral diseases can be more dangerous as high blood sugar may give viruses a place to thrive, according toDiabetes in Control, a news and information resource for medical professionals.

Higherlevels of inflammationhave been discovered in the bodies of people with diabetes, weakening the immune system and making it more difficult for those affected to stave off sickness in general.

The kidneysproduce several hormonesthat affect immune responses. Having kidney disease and failure can weaken your immune system, making it easier for infections to take hold. According to theNational Kidney Foundation, doctors and researchers have found that most infections are worse in people with kidney disease.

The liver is an integral member of the bodysline of defense, helping to regulate the number of white blood cells utilized in immune responses and defend against harmful pathogens. Someone with liver disease is experiencing abnormalities in the function of the immune system, giving rise to more serious illness.

Neurological and neurodevelopmental conditions may also increase the risk of serious Covid-19 for people of any age.

These include disorders of the brain, spinal cord, peripheral nerve and muscle such as cerebral palsy, epilepsy, stroke and intellectual disability, according to theCDC. Those with moderate to severe developmental delay, muscular dystrophy or spinal cord injury are also more at-risk.

People with neurological conditions may not be more at risk due to solely their condition, but because medications they might take to control their condition could hamper their immune system. However, some neurological conditions, such as Parkinsons, have been recognized to haveinflammatory components, which may harm the immune system.

Others including muscular dystrophy, multiple sclerosis or amyotrophic lateral sclerosis (ALS) could cause paralysis to the diaphragm, which leaves those affected very at risk for respiratory failure if they were to be sick with Covid-19.

If you see yourself on the list of those at higher risk for severe illness, there are several things you can do to protect yourself. First, make sure you are contact your doctor or doctors about your risk level. Second, be extra vigilant about the recommendations that most people are being asked to follow.

Stay home whenever possible and avoid close contact with people, theCDC suggests. Wash your hands often to prevent transferring the virus from a surface to your face, and try to clean and disinfect frequently touched surfaces as often as you can.

If you dont have an underlying condition, doing your part by practicing these cautionary measures can help protect not only you, but your loved ones with existing conditions.

Click here for more coronavirus coverage.

The-CNN-Wire & 2020 Cable News Network, Inc., a WarnerMedia Company. All rights reserved.

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Pandemic reveals another shortcoming in preparation: Scattershot, chaotic research for treatments – Anchorage Daily News

In a desperate bid to find treatments for people sickened by coronavirus, doctors and drug companies have launched more than 100 human experiments in the United States, investigating experimental drugs, a decades-old malaria medicine and cutting-edge therapies that have worked for other conditions such as HIV and rheumatoid arthritis.

Development of effective treatments for covid-19 would be one of the most significant milestones in returning the United States to normalcy. But the massive effort is disorganized and scattershot, harming its prospects for success, according to multiple researchers and health experts. Researchers working around-the-clock describe a lack of a centralized national strategy, overlapping efforts, an array of small-scale trials that will not lead to definitive answers and no standards for how to prioritize efforts, what data to collect or how to share it to get to answers faster.

"It's a cacophony; it's not an orchestra. There's no conductor," said Derek Angus, chair of the department of critical care medicine at University of Pittsburgh School of Medicine, who is leading a covid-19 trial that will test multiple therapies. "My heart aches over the complete chaos in the response."

The global biomedical research establishment could be one of most powerful assets in the campaign against the new virus, with experts all over the world - and especially the scientific and medical powerhouse of the United States - in rare alignment in their focus on a single enemy. Some large trials designed to be definitive have launched. But with more than 500 human clinical trials worldwide, the lack of coordination puts the world at risk of ending up with a raft of inconclusive and conflicting studies and little idea of what interventions work for the next wave of illness.

Francis Collins, director of the National Institutes of Health, the nation's largest biomedical research agency, acknowledged researchers' frustrations but said in an interview Wednesday he has been working behind the scenes to launch an unprecedented, public-private partnership to address the problems. He said the framework involves top pharmaceutical companies such as Pfizer and Johnson & Johnson, domestic and international government agencies, including the European Medicines Agency, and academic research centers.

Collins said the month-long discussions have been kept under wraps to ensure buy-in for an approach likely to require sacrifices of personal recognition, scientific credit and profit - a centralized decision, for example, not to proceed with tests of one company's drug to move faster on a competitor's.

"I think we have the necessary clout to steer this whole complicated ecosystem," he said. "When you look at some of the things that are happening sporadically, we may be unlikely to learn what we need to from such disconnected, small trials. The whole point is to replace that with a coherent, evidence-based approach. . . . I want to know what works, and I want to have it answered by June or July."

Agency officials said further details would be released in coming days.

Anthony Fauci, director of the National Institute of Allergy and Infectious Diseases (NIAID), said in an email Tuesday that the partnership led by Collins is the "functional equivalent of a National Strategy."

While Collins was working on developing that strategy, hospitals, drug companies, government labs and individual doctors were flooding the system with proposals for drugs and other interventions to test against the virus - an outpouring that reveals how siloed and fragmented the research enterprise remains. For example, there are 26 separate U.S. trials listed for the anti-malarial drug hydroxychloroquine, all with different designs. Some use the drug as a preventive, others as a treatment. Some use it alone, some with other drugs or vitamins, and some have no comparison group to tell if the drug was responsible for the outcome. That will make it more difficult to conclude whether, or in what circumstances, the drug may work.

Collins said a working group is addressing this problem by sifting through about 100 possible covid-19 treatments to decide which are the six to eight most promising drugs to move forward in large-scale trials. Those will be deployed in large clinical trial networks.

The new federal effort is motivated in part by what happened in China. Clifford Lane, deputy director for clinical research and special projects at NIAID, traveled to the origin of the outbreak in February as part of an international delegation to help the world learn from the Chinese experience. He was troubled by the lack of a strategic plan to prioritize and fast-track the most promising treatments, leading to a mosaic of inconclusive findings.

"We do have to have a bigger strategy than every university, every institute and - to be blunt - every country" working on their own research efforts, Lane said in an interview.

At the heart of the problem is the basic question of whether a drug really works. Typically, drugs and medical interventions are first tested in small clinical trials that establish safety before the most promising ones are funneled into bigger trials, in an iterative and years-long process. These trials, which typically randomly assign patients to receive either a drug or a placebo, prove that medicines, vaccines and medical procedures are effective and safe. But with the urgency of the coronavirus threat, timelines have been squeezed, doctors are doing uncontrolled experiments as they administer regular care and the typical model for research is too slow.

David Boulware, an infectious disease physician and scientist at the University of Minnesota, has gotten at least 50 emails from companies and researchers with treatments they want to test. The urgency to find something - anything - for patients who have nothing other than supportive care has led researchers to pull everything off the shelf: a mix of existing drugs that show promise, stem cell treatments and brand new compounds designed specifically against covid-19.

The energy is remarkable, but it needs to be channeled. Clinical trials, whether for an HIV drug or a brand new medicine, compete for many of the same patients. If there are too many trials at a hospital, none of them may enroll enough patients to get clear results. If there are too many similar small trials running in parallel, their results individually may be inconclusive, and the data could have so many differences they may not be able to be pooled.

"There's all sorts of people wanting to try anything, because people are desperate," Boulware said.

He and others who were unaware of Collins' plans argue that national leadership - whether guidance on how to prioritize trials, a central body coordinating efforts, or a mechanism to play matchmaker among institutions working on similar ideas - could help channel the ubiquitous scientific desire to make a bigger, faster impact.

In this Thursday, April 9, 2020 file photo, a chemist displays hydroxychloroquine tablets in New Delhi, India. Scientists in Brazil have stopped part of a study of the malaria drug touted as a possible coronavirus treatment after heart rhythm problems developed in one-quarter of people given the higher of two doses being tested. Chloroquine and a similar drug, hydroxychloroquine, have been pushed by President Donald Trump after some early tests suggested the drugs might curb coronavirus entering cells. (AP Photo/Manish Swarup, File)

Some large-scale efforts are already underway: The World Health Organization has organized a massive trial in 90 countries of four promising therapies. The National Institutes of Health is conducting a test of the antiviral medication remdesivir at more than 50 institutions and last week launched a large trial for an anti-malarial medication. A $50 million effort at the Duke Clinical Research Institute will test hydroxychloroquine in 15,000 health care workers and create a registry that can be tapped to speed up future trials, such as for a vaccine.

"People should not be fatalistic that we're going to have a paucity of evidence for things that provide benefit," Collins said. "I hope we'll have three to four of those [treatments] by the summer."

But as the federal effort has proceeded largely in secret, individual institutions have scrambled to set up committees of experts who evaluate which trials make sense to move forward. At the University of Pennsylvania, a weeks-old committee gives priority scores to trials based on criteria such as whether they will compete with existing efforts and how likely they are to enroll all the patients needed to get a result. A task force at Duke University does a similar review.

"There's a lot of stuff bubbling up. It would seem like a sensible thing to do would be to align everyone around the same trials, not one trial for each context and not have each institution do its own thing and at the end of the day everyone has done a small trial . . . and we don't know what to make of it," said Steven Joffe, a bioethicist at the University of Pennsylvania. "Let's get to an answer."

Many proposed trials overlap - using the same drug, such as the anti-malaria treatment hydroxychloroquine that has been touted by President Trump, his lawyer Rudolph W. Giuliani and conservative talk show host Laura Ingraham.

Boulware is halfway through one such hydroxychloroquine trial, which examines whether the drug is effective at preventing the disease or in treating people with mild cases. People who participate will receive either the drug or a vitamin in the mail.

He said he is motivated by his experience working on Ebola, when by the time a well-designed trial was up and running, the outbreak was dying down. He plowed ahead with his hydroxychloroquine trial weeks before he heard - last Friday - the National Institutes of Health had declined to fund it. He found international collaborators through chance and social media, when some Canadian researchers emailed to ask if he would share his trial design with them. He ended up connecting the Canadians with one another and is now working on overcoming the complex legal requirements to share data.

But now, his trial is potentially competing with a bunch of others that also test hydroxychloroquine all across the country - and enrollment has slowed in recent days.

Small trials and even anecdotal reports of treatments that appear to have worked on small groups of patients are already being shared, sowing both hope and confusion about the evidence.

A study published Friday in the New England Journal of Medicine, for instance, reported on 53 people who took remdesivir, a failed Ebola drug Trump has praised and many families have already tried to get access to outside of trials. The results were impossible to interpret, though, since some of those patients might have gotten better on their own and there was no comparison group of patients who did not receive the drug. Hydroxychloroquine, the cheap and readily available anti-malarial drug, has also already been widely used in patients, despite only suggestive evidence that it might work.

As more small-scale studies are designed, the risks of inconclusive but suggestive results multiply - and paradoxically, they could make it harder to conduct well-designed clinical trials that get to the bottom of whether a treatment works. Well-designed clinical trials require patients to be willing to be randomly assigned to receive the treatment - or a placebo.

Emma Meagher, chief clinical research officer at the University of Pennsylvania's Perelman School of Medicine, said her institution's study of the malaria drug in severely ill patients does not have a comparison group that receives a placebo because the media around the drug has made it the standard of care despite the lack of evidence. Every meeting, she said, begins with a discussion about how to prevent the next experimental therapy from becoming like hydroxychloroquine.

In some ways, designing research studies when clinicians have an imperative to give their patients the best care possible is an inherently tricky situation. Outside of top-tier research hospitals, patients may not have access to trials, so clinicians may have little option but to give them drugs in what amounts to an uncontrolled experiment. The Infectious Diseases Society of America released guidelines last week that clinicians should give experimental drugs only in trials, but safety-net and small, rural hospitals are less likely to have access to those.

"Do we really want to have [some] people trying new different things and the rest of you sit and wait?" asked Benjamin Linas, an infectious disease physician working on oversight of clinical trial protocols at Boston Medical Center.

Many researchers have said they are hopeful a national strategy will help unify and speed the search, but lament the time already lost. The United States did not have a pandemic clinical trials network ready to be activated, but existing clinical trials networks, such as ones used to test HIV treatments, are now being repurposed. Collins said he had never seen research move faster, but in a pandemic that can still feel slow.

"We have imperfect networks. I mean there are some there, but they've never been tested in this way," said Adrian Hernandez, vice dean for clinical research at Duke University School of Medicine. "Having a common infrastructure that can do rapid cycle trials - that would be beneficial."

Collins said he began partnering with private companies, research institutions and other agencies in mid-March. Had he started sooner, he said, the urgency of the situation might not have been clear to the companies and agencies he had to convince to work together. He disagrees significant time has been lost and noted the efforts were able to piggyback on an existing framework for pursuing drugs with industry collaboration called the Accelerating Medicines Partnership.

Partnerships that previously took a year and a half to build were put together in a week, he said.

"I think it is a world record for anything of this sort; it might have been difficult to get full unanimous agreement to what is clearly unprecedented - for a willingness to give up control," Collins said.

As with other areas of pandemic planning, many hope the United States will learn from this lesson, that it needs a preparedness plan not only for allocating essential supplies and scaling up testing but also in plotting research.

The problem is we need to remember to invest in preparedness, at times when were not affected with a pandemic or epidemic, said Barbara Bierer, director of the regulatory foundations, ethics and the law program of the Harvard Clinical and Translational Science Center. And its hard to repurpose or commit resources to something that doesnt appear immediate on the horizon.

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Pandemic reveals another shortcoming in preparation: Scattershot, chaotic research for treatments - Anchorage Daily News

Stem Cell Therapy Market to 2027 – Global Analysis and Forecasts by Type; Treatment; Application; End User, and Geography – Salamanca Press

NEW YORK, April 15, 2020 /PRNewswire/ -- The stem cell therapy market was valued at US$ 1,534.55 million in 2019 and is estimated to reach US$ 5,129.66 million by 2027; it is expected to grow at a CAGR of 16.7% from 2020 to 2027.

Read the full report: https://www.reportlinker.com/p05882135/?utm_source=PRN

The increasing awareness related to the stem cells therapy in effective disease management and growing demand for regenerative medicines are the key factor driving the stem cell therapy market. However, high cost related of the stem cell therapy limits the growth of the market.Stem cell research has been widely investigated globally for various medical applications, especially for the treatment of humans.This raises the importance of creating public awareness about stem cell research and its clinical potential.

The main role of stem cells is in the replacement of dying cells and reconstruction of damaged tissues. Based on the extensive stem cell research, many scientists have claimed that these cells could probably be used in the treatment of various diseases, including cancer and cardiovascular disease.There is a large number of potential treatment procedures that are undergoing clinical trials, and a notably few stem cell therapies have won FDA (i.e., US Food and Drug Administration) approval for clinical usage. For instance, in 2019, the FDA approved Fedratinib for the first-line treatment for myelofibrosis. Moreover, stem cell therapies are widely used in bone marrow transplantation, and these therapies have benefited thousands of people suffering from leukemia. Hematopoietic stem cells are used for treating more than 80 medical diseases, including immune system disorders, blood disorders, neurological disorders, metabolic disorders, genetic disorders, and several types of cancers, such as leukemia and lymphoma; this is also likely to boost the demand for this treatment procedure during the forecast period. Researchers are further investigating the use of stem cell therapies in the treatment of autoimmune disorders.

The global stem cell therapy market has been segmented on the basis of type, treatment, application type, and end user.Based on type, the market has been segmented into adult stem cell therapy, induced pluripotent stem cell therapy, embryonic stem cell therapy, and others.

The adult stem cell therapy held the largest share of the market in 2019; however, induced pluripotent stem cell therapy is estimated to register the highest CAGR in the market during the forecast period.Based on treatment, the stem cell therapy market has been segmented into allogeneic and autologous.

The allogeneic segment held a larger share of the market in 2019; however, the market for the autologous segment is expected to grow at a higher CAGR during the forecast period.Based on application type, the stem cell therapy market has been segmented into musculoskeletal, dermatology, cardiology, drug discovery and development, and other applications.

The musculoskeletal segment held the largest share of the stem cell therapy market in 2019, whereas the drug discovery and development segment is expected to report the highest CAGR during 20202027. Based on end user, the market has been segmented into academic and research institutes, and hospitals and specialty clinics. The academic & research institutes held the largest share of the market in 2019, and it is also expected to report the highest CAGR during the forecast period.Several essential secondary sources referred to for preparing this report are the FDA, World Health Organization (WHO), Organisation for Economic Co-operation and Development, National Institutes of Health, Spanish Agency for Medicines (AEMPS), Japanese Society for Regenerative Medicine, and Indian Council of Medical Research, among others.

Read the full report: https://www.reportlinker.com/p05882135/?utm_source=PRN

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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Stem Cell Therapy Market to 2027 - Global Analysis and Forecasts by Type; Treatment; Application; End User, and Geography - Salamanca Press

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

View source version on businesswire.com: https://www.businesswire.com/news/home/20200414005353/en/

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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