Researchers Say They Have Clue to Early-Onset Parkinson Disease – AJMC.com Managed Markets Network

Patients with early-onset Parkinson disease may have been born with disordered brain cells that mishandled dopamine for decades, according to a study released Monday.

To perform the study, researchers at Cedars-Sinai Medical Center and UCLA generated special stem cells, known as induced pluripotent stem cells (iPSCs), from cells of 3 patients with young-onset Parkinson disease; the patients were aged 30-39 and hadno known familial history of PD or PD mutations.

The process involved taking adult blood cells back to a primitive embryonic state. These iPSCs can then produce any cell type of the human body, all genetically identical to the patient's own cells. The team used the iPSCs to produce dopamine neurons from each patient and then cultured them in a dish and analyzed the neurons' functions.

The researchers detected 2 key abnormalities in the dopamine neurons:

The investigators also used their model to test a number of drugs that might reverse the abnormalities. One drug, PEP005, or ingenol mebutate gel, used for basal cell carcinoma, reduced the elevated levels of alpha-synuclein in both the dopamine neurons in the dish and in laboratory mice.

The drug also countered another abnormality they found in the patients' dopamine neuronselevated levels of an active version of an enzyme called protein kinase C. However, the role of this enzyme version in Parkinson disease is unknown.

In Parkinson, brain neurons that make dopamine, a substance that helps coordinate muscle movement, become impaired or die. The disease is diagnosed in at least 500,000 people in the United States each year, and the incidence is rising. About 10% are aged 21 to 50 years.

Michele Tagliati, MD, director of the Movement Disorders Program, vice chair and professor in the Department of Neurology at Cedars-Sinai, said the team plans to investigate how PEP005 might be delivered to the brain to potentially treat or prevent young-onset Parkinson. The team also plans more research to determine whether the abnormalities the study found in neurons of young-onset Parkinson's patients also exist in other forms of the disease.

Reference

Laperle AH, Sances S, Yucer N, et al.iPSC modeling of young-onset Parkinsons disease reveals a molecular signature of disease and novel therapeutic candidates [published online January 27, 2020].Nat Med.doi: 10.1038/s41591-019-0739-1.

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Diabetes-related proteins examined for the first time at high resolution – Drug Target Review

A key receptor has been examined for the first time at high resolution which could lead to better treatments for conditions such as type 2 diabetes.

Scientists have examined a key receptor for the first time at high resolution which, they say, broadens understanding of how it might function and opens the door to future improvements in treating conditions such as type 2 diabetes.

The scientists were led by experts at the University of Birmingham, UK and the Max Planck Institute for Medical Research, Germany.

Glucagon-like peptide-1 receptors (GLP1R) are found on insulin-producing beta cells of the pancreas and neurons in the brain. The receptor encourages the pancreas to release more insulin, stops the liver from producing too much glucose and reduces appetite. This combination of effects can help to control blood sugar levels.

Therefore, GLP1R has become a significant target for the treatment of type 2 diabetesand a range of drugs are now available that are based on it. But much remains unknown about GLP1R function because its small size makes it difficult to visualise.

Our research allows us to visualise this key receptor in much more detail than before, David Hodson, Professor of Cellular Metabolism at the University of Birmingham. Think about watching a movie in standard definition versus 4k, thats how big the difference is. We believe this breakthrough will give us a much greater understanding of GLP1R distribution and function. Whilst this will not immediately change treatment for patients, it might influence how we design drugs in the future.

GLP1R visualized in insulin-secreting beta cells at super-resolution (credit: University of Birmingham).

The researchers used a number of techniques to conduct a detailed examination of the receptor in living cells including synthesis of marker compounds, immunostaining, super-resolution microscopy, as well as in vivo examination of mice.

Our experiments, made possible by combining expertise in chemistry and cell biology, will improve our understanding of GLP1R in the pancreas and the brain. Our new tools have been used in stem cells and in the living animal to visualise this important receptor and we provide the first super-resolution characterisation of a class B GPCR. Importantly, our results suggest a degree of complexity not readily appreciated with previous approaches, added Johannes Broichhagen, Departmental Group Leader of the Max-Planck Institute for Medical Research.

The findings were published in Nature Communications.

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Divorce as Seen Through the Eyes of a Child – SWAAY

With so many groundbreaking medical advances being revealed to the world every single day, you would imagine there would be some advancement on the plethora of many female-prevalent diseases (think female cancers, Alzheimer's, depression, heart conditions etc.) that women are fighting every single day.

For Anna Villarreal and her team, there frankly wasn't enough being done. In turn, she developed a method that diagnoses these diseases earlier than traditional methods, using a pretty untraditional method in itself: through your menstrual blood.

Getting from point A to point B wasn't so easy though. Villarreal was battling a disease herself and through that experience. I wondered if there was a way to test menstrual blood for female specific diseases," she says. "Perhaps my situation could have been prevented or at least better managed. This led me to begin researching menstrual blood as a diagnostic source. For reasons the scientific and medical community do not fully understand, certain diseases impact women differently than men. The research shows that clinical trials have a disproportionate focus on male research subjects despite clear evidence that many diseases impact more women than men."

There's also no denying that gap in women's healthcare in clinical research involving female subjects - which is exactly what inspired Villarreal to launch her company, LifeStory Health. She says that, with my personal experience everything was brought full circle."

There is a challenge and a need in the medical community for more sex-specific research. I believe the omission of females as research subjects is putting women's health at risk and we need to fuel a conversation that will improve women's healthcare.,"

-Anna Villarreal

Her brand new biotech company is committed to changing the women's healthcare market through technology, innovation and vocalization and through extensive research and testing. She is working to develop the first ever, non-invasive, menstrual blood diagnostic and has partnered with a top Boston-area University on research and has won awards from The International Society for Pharmaceutical Engineering and Northeastern University's RISE.

How does it work exactly? Proteins are discovered in menstrual blood that can quickly and easily detect, manage and track diseases in women, resulting in diseases that can be earlier detected, treated and even prevented in the first place. The menstrual blood is easy to collect and since it's a relatively unexplored diagnostic it's honestly a really revolutionary concept, too.

So far, the reactions of this innovative research has been nothing but excitement. The reactions have been incredibly positive." she shares with SWAAY. Currently, menstrual blood is discarded as bio waste, but it could carry the potential for new breakthroughs in diagnosis. When I educate women on the lack of female subjects used in research and clinical trials, they are surprised and very excited at the prospect that LifeStory Health may provide a solution and the key to early detection."

To give a doctor's input, and a little bit more of an explanation as to why this really works, Dr. Pat Salber, MD, and Founder of The Doctor Weighs In comments: researchers have been studying stem cells derived from menstrual blood for more than a decade. Stem cells are cells that have the capability of differentiating into different types of tissues. There are two major types of stem cells, embryonic and adult. Adult stem cells have a more limited differentiation potential, but avoid the ethical issues that have surrounded research with embryonic stem cells. Stem cells from menstrual blood are adult stem cells."

These stem cells are so important when it comes to new findings. Stem cells serve as the backbone of research in the field of regenerative medicine the focus which is to grow tissues, such as skin, to repair burn and other types of serious skin wounds.

A certain type of stem cell, known as mesenchymal stem cells (MenSCs) derived from menstrual blood has been found to both grow well in the lab and have the capability to differentiate in various cell types, including skin. In addition to being used to grow tissues, their properties can be studied that will elucidate many different aspects of cell function," Dr. Salber explains.

To show the outpour of support for her efforts and this major girl power research, Villarreal remarks, women are volunteering their samples happily report the arrival of their periods by giving samples to our lab announcing de-identified sample number XXX arrived today!" It's a far cry from the stereotype of when it's that time of the month."

How are these collections being done? Although it might sound odd to collect menstrual blood, plastic cups have been developed to use in the collection process. This is similar to menstrual products, called menstrual cups, that have been on the market for many years," Dr. Salber says.

Equally shocking and innovative, this might be something that becomes more common practice in the future. And according to Dr. Salber, women may be able to not only use the menstrual blood for early detection, but be able to store the stem cells from it to help treat future diseases. Companies are working to commercialize the use of menstrual blood stem cells. One company, for example, is offering a patented service to store menstrual blood stem cells for use in tissue generation if the need arises."

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StemoniX’s microBrain to be Featured in Podium Presentation at SLAS 2020 International Conference & Exhibition – BioSpace

MAPLE GROVE, Minn., Jan. 27, 2020 /PRNewswire/ --StemoniX, a biotech company revolutionizing how new medicines are discovered, announced today that its Director of Applications, Oivin Guichert, Ph.D., will deliver a podium presentation highlighting the company's microBrain technology at the SLAS (Society for Laboratory Automation and Screening) 2020 International Conference & Exhibition at the San Diego Convention Center, Jan. 27-29, 2020. The presentation will be featured as part of the Assay Development and Screening Session during the annual meeting.

During the podium presentation, entitled "New innovation to solve unmet needs: Implementing human induced pluripotent stem cell-derived neural spheroids as a robust screening platform for phenotypic-based central nervous system drug discovery," Dr. Guichert will detail how performing a high-throughput functional screening assay on StemoniX's human induced pluripotent stem cell (iPSC)-derived 3D neural spheroid platform demonstrated the ability to identify a wide range of hits spanning multiple target areas. He will highlight how this model could provide relevant human platforms for disease-specific drug discovery to help overcome traditional hurdles of CNS-targeted drug discovery and development efforts.

Ping Yeh, co-founder and CEO of StemoniX, said: "The SLAS 2020 International Conference & Exhibitionis an ideal event to showcase the value potential of our microOrgan platform and AnalytiX data management and analytical software. As presented by Dr. Guichert and in the six posters, microBrain, microHeart, microPancreas and AnalytiX offer the potential to reshape how drugs are discovered and developed by providing the opportunity to go from model to molecule to validated drug in a fraction of the time and cost required with traditional methods. This includes the near-term potential to identify and advance novel therapeutic targets for Rett syndrome by leveraging our groundbreaking in vitro microBrain model in partnership with AI drug discovery pioneer, Atomwise."

Podium Presentation Details

Title:

New innovation to solve unmet needs: Implementing human induced pluripotent stem cell-derived neural spheroids as a robust screening platform for phenotypic-based central nervous system drug discovery

Session:

Assay Development and Screening

Event

SLAS 2020 International Conference & Exhibition

Date:

Tuesday, January 28, 2020

Time:

4:00 4:30 p.m. PST

Location:

San Diego Convention Center

Room/Location:

6C

Poster Presentations:

About StemoniXStemoniX is accelerating the discovery of new medicines to treat challenging diseases via the world's first ready-to-use assay plates containing living human microOrgans, including electrophysiologically active neural (microBrain) and cardiac (microHeart) cells. Predictive, accurate, and consistent, StemoniX's products combined with its proprietary data management and analytical tools (AnalytiX) are revolutionizing traditional drug discovery and development by radically improving the speed, accuracy and costs required to identify new drugs and conduct initial human cell toxicity and efficacy testing. Through its Discovery as a Service offering, the company partners with organizations to screen compounds as well as to create customized microOrgan models and assays tailored to specific discovery and toxicity needs. Visit http://www.stemonix.com to learn how StemoniX is helping global institutions humanize drug discovery and development to bring the most promising medicines to patients.

Tiberend Strategic Advisors, Inc.

Investor Contact:Maureen McEnroe, CFA+1.212.375.2664mmcenroe@tiberend.com

Media Contact:Ingrid Mezo+1.646.604.5150imezo@tiberend.com

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5 of the biggest medical advances of the past decade – Health24

Every year, medical technology further evolves, and new discoveries are made. This brings hope to those suffering from grave medical conditions. Health24 covers these advances on an ongoing basis, and the following are a few of the biggest breakthroughs of the decade in a nutshell:

The past decade has seen a number of medical headlines involving 3D-organ-printing, up to the point where, recently, researchers managed to create living skin, complete with blood vessels, as well as hearts. While many of these advancements need more research before they can be used in a clinical setting, 3D-printing is set to become more prevalent over the next the next decade, which will make transplanting easier for those in need.

Health24 published several stories about gene therapy over the last ten years. And while there were restrictions placed on gene therapy research in the early 2000s, there's been a strong resurgence, as illustrated by this study focusing on gene therapy in the fight against leukaemia.

Despite a number of setbacks, there were some successes that could translate to treatments in the future. One of the most recent development involves the first clinical trial of its type. Researchers used CRISPR to edit the DNA of peoples immune systems to help treat certain cancers.

While only a small number of patients were involved in the Stage 1 clinical trial, experts believe that this was an important step, in that it proved that the technique is safe to use.

Read more about gene therapy here.

The focus on gut health and our microbiome (the collection of bacteria in the gut) has never been stronger. In the past, researchers didnt pay much attention to the role of the bacteria in our gut, and it's been mainly during the past 15 years that researchers have been studying this concept.

According to the BMJ, the gut microbiota is crucial for essential processes in the body, such as the fermentation of non-digestible dietary fibres. It does more than that, though, and plays a role in many key areas of human health, from our immunity and appetite to the way we digest our food.

This helped researchers to explore the role of gut bacteria in areas like depression. An article in the BMJ reports on changes in the gut microbiota in the case of not only obesity, diabetes, and liver disease, but also cancer and even neurodegenerative diseases.

In fact, a study covered by Health24 links gut microbes to chronic fatigue syndrome, a condition that has been baffling experts for decades.

HIV and Aids remain important public healthcare topics in South Africa. During the past decade, antiretroviral treatment has improved and become more readily available. In fact, the virus is currently controlled so well that the viral load in many patients' blood has become virtually undetectable.

According to Pharmaceutical Technologies, various studies over the past decade found that treatment with antiretroviral therapy has also reduced the risk of spreading the infection to HIV-negative partners in both homosexual and heterosexual couples.

A few months ago, Health24 published a story about a man simply known as the London patient, who became entirely free from HIV following stem cell treatment for Hodgkin's Lymphoma. He was the second patient to demonstrate this phenomenon.

In 2017, a man known as the Berlin patient had two copies of the CCR5-delta32 genetic mutation. The patient stopped his ART 16 months following a bone marrow transplant, and his blood viral load was still undetectable 18 months later.

Canceris one of the leading causes of morbidity and mortality worldwide, with approximately 14 million new cases reported annually, according to the World Health Organization. Experts say immunotherapy is a promising new development, and ongoing research has been conducted over the past decade.

In one of the latest studies, Dr Christopher E. Rudd, a researcher at the Centre de Recherche de l'Hpital Maisonneuve-Rosemont (CR-HMR) and Universit de Montral, discovered a new cell therapy approach that boosts the immune response of T lymphocytes to malignant tumours. The results of the study were recently published in the respected journal Nature.

Image credit: iStock

Compiled by Marelize Wilke

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Scientists Have Grown Snake Venom Glands in The Lab. Here’s Why That’s Awesome – ScienceAlert

For the first time, scientists have produced snake venom toxins in the lab, opening up a much-needed path for developing drugs and venom antidotes that doesn't involve having to breed and milk real-life snakes.

The toxins have been produced through mini glands called organoids, following a process adapted from growing simplified human organs something that is already helping in a wide range of scientific and medical research projects.

In the case of the snakes, researchers were able to blow organoids matching the Cape coral snake (Aspidelaps lubricus cowlesi) and seven other snake species, and they say this new approach is a welcome upgrade on current methods of farming snakes to extract their venom.

"More than 100,000 people die from snake bites every year, mostly in developing countries," says molecular biologist Hans Clevers, from Utrecht University in the Netherlands. "Yet the methods for manufacturing antivenom haven't changed since the 19th century."

By tweaking the recently developed process for growing human organoids including reducing the temperature to match reptiles rather than mammals the researchers were able to find a recipe that supports the indefinite growth of tiny snake venom glands.

Tissue was removed from snake embryos and put into a gel mixed with growth factors, but access to stem cells which is how human and mouse organoids are usually developed wasn't required.

The cells quickly began dividing and forming structures, giving the team hundreds of growing samples in the space of a couple of months, and producing small white blobs from which venom toxins could be harvested.

Al least four distinct types of cell were identified by the researchers within the artificially grown venom glands, and they were also able to confirm that the venom peptides produced were biologically active, closely resembling those in live snake venom.

Snake venom gland organoids. (Ravian van Ineveld/Princess Mxima Center)

"We know from other secretory systems such as the pancreas and intestine that specialised cell types make subsets of hormones," says developmental biologist Joep Beumerfrom Utrecht University.

"Now we saw for the first time that this is also the case for the toxins produced by snake venom gland cells."

The use of snake venom toxins to develop medicines and treatments has been going on since the time of ancient Greece. In the modern age, drugs fighting everything from cancer to haemorrhages have been developed with the help of toxins we find in snake venom.

Having faster and more controlled access to these toxins could mean these treatments can be developed more easily and on a shorter time scale, say the researchers.

Besides drug development, these organoid venom glands should make it easier and faster to develop antivenoms and with so many people suffering deaths, injuries or disabilities because of snake bites, that will make a considerable difference.

"It's a breakthrough," snake venom toxicologist Jos Mara Gutirrez from the University of Costa Rica, told Science.

"This work opens the possibilities for studying the cellular biology of venom-secreting cells at a very fine level, which has not been possible in the past."

The research has been published in Cell.

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Scientists Have Grown Snake Venom Glands in The Lab. Here's Why That's Awesome - ScienceAlert

Alopecia: What causes the hair loss condition? – Home – WSFX

Everyone sheds about 100 hairs each day as part of the normal hair growth cycle, but excess loss is usually a distressing development.(iStock)

Hair loss is typically considered the domain of aging men, but this equal-opportunity condition which has many causes can affect virtually anyone.

Alopecia is the medical term for hair loss, and it doesnt only happen on the scalp. Some illnesses and medications can trigger balding over the entire body, though genetics account for most cases on the head, according to theCleveland Clinic.

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Everyone sheds about 100 hairs each day as part of the normal hair growth cycle, but excess loss is usually a distressing development. Americans spend more than $3.5 billion each year trying to treat it, according to theAmerican Hair Loss Association.

Most peoples hair grows about a half-inch per month, and about 90 percentof your hair is actively growing at any given time, with the other 10 percentin dormant phase. After two or three months, this dormant hair falls out and its follicles begin growing new hair as other follicles begin a dormant phase.

Shedding hair is different from hair loss, when a hair falls out and doesnt grow back. People often shed hair during stressful events, such aschildbirth, a breakup or divorce or during times of grief.

It still doesnt feel good, and it takes the hair [awhile] to reach a certain length where you perceive its presence, said Doris Day, a board-certified dermatologist New York City and an attending physician at Lenox Hill Hospital, also in New York. So it feels like a hair loss, but its not a hair loss.

Aside from heredity, noticeable hair loss can be caused by wide variety of factors, including:

Harsh hairstyles or treatments: Hairstyles that consistently use rubber bands, rollers or barrettes, or pull hair into tight styles such as cornrows, can inflame and scar hair follicles. So can incorrectly used chemical products such as dyes, bleaches, straighteners or permanent wave solutions. Depending on the degree of damage, resulting hair loss can be permanent.

Hormone imbalances: In women, hormonal shifts from birth control pills,pregnancy, childbirth, menopause or hysterectomy can induce more hair follicles than normal to enter the dormant phase.

Illness or surgery: The stress from sickness or surgery may prompt the body to temporarily cease nonessential tasks such as hair production. Specific conditions can also trigger it, including thyroid disorders,syphilis, iron deficiency,lupusor severe infection. An autoimmune condition called alopecia areata, which has no cure, causes rapid body-wide hair loss.

Medications and vitamins: Cancer chemotherapy, which attacks hair follicles in its attempt to kill all fast-growing cells around the body, is a well-known reason for hair loss. Other medications side effects include hair shedding as well, such as some that treat high blood pressure andgout(a painful joint condition caused by a buildup of uric acid). Excessive levels of vitamin A also contribute.

Nutritional deficits: Heavy dieting or eating disorders such asbulimiaandanorexiacan temporarily stun hair follicles to cease growth. This can also occur from insufficient protein, vitamin or mineral intake.

Aging: A natural effect of growing older is slowed hair growth.

Women usually dont go completely bald, but lose hair on the top of the head or the temples. Men tend to lose hair on their temples, and are more likely than women to go completely bald, Day said.

Dermatologists will examine the persons scalp and take a history of medical or stressful events to see whats been going on in their life and their world, Day said.

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The dermatologist may take a biopsy a small patch of skin that includes the hair follicle and send it to a pathologist to determine if an autoimmune disease, such as lupus, is the cause of the hair loss.

Examining the hair and follicle can also determine whether someone has a bacterial or fungal infection, Day said.

Hair loss remedies range from the mild to the extreme and the inexpensive to the costly. Much depends on how much hair is gone and how high a priority it is to mask its absence or replace it.

According to the Cleveland Clinic, treatments include:

Hair weaves or wigs: Typically expensive, wigs and hair weaves either completely cover the head or add to existing hair, restoring the appearance of a full head of hair. They are especially practical for cancer patients and those whose hair loss is temporary.

Topical creams and lotions: Over-the-counter minoxidil (also known as the brand name Rogaine) can restore some hair growth, especially in those with hereditary hair loss. It is applied directly to the scalp. Prescription-strength finasteride (Propecia) comes in pill form and is only for men. According to theAmerican Academy of Family Physicians(AFP), it may take up to six months to tell if these medications are working.

Anti-inflammatory medications: Prescription steroid-based creams or injections can calm follicles damaged or inflamed by harsh chemicals or excessive pulling.

Surgery: Men tend to be better candidates for surgical hair-replacement techniques because their hair loss is often limited to one or two areas of the scalp. Procedures include grafting, which transplants from one to 15 hairs per disc-shaped graft to other locations. Scalp reduction removes bald skin from the scalp so hair-covered scalp can be stretched to fill in the bald areas. Side effects include swelling, bruising and headaches.

Hair-growth laser treatment can also help stimulate hair follicles and improve growth, Day said. People often see results when they combine laser treatment with another intervention, she said. Treatments range in price from $30 and up for Rogaine to about $3,000 for laser treatment, she added.

According to theNational Institute of Arthritis and Musculoskeletal and Skin Diseases(NIAMSD), alternative therapies may not help hair regrow and many are not supported by medical research. However, other treatments that reportedly improve alopecia areata include Chinese herbs, acupuncture, zinc and vitamin supplements, evening primrose oil and aroma therapy.

Viviscal, a natural supplement, has also shownmore hair growthin men compared to those who took fish extract in clinical trials, Day said.

The NIAMSD recommends discussing any alternative treatments with physicians before use.

The drug Tofacitinib is approved to treat adults witharthritis, but a growing number of cases suggest that it can also treat alopecia universalis, a condition in which people lose all of the hair on their body because theirimmune systemattacks hair follicles,Live Science previously reported.

The finding occurred after doctors prescribed a 25-year-old man with alopecia universalis the drug because they had heard it had treated a similar condition in mice,according to a statement from Yale University. After three months of treatment, the man had completely regrown the hair on his scalp, and he had visible eyebrows, eyelashes, facial hair, as well as hair elsewhere on his body.

Its exciting, said Day, who did not treat this particular patient. There seems to be a real effect here.

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Its unclear how Tofacitinib (brand name Xeljanz) works, but researchers hope to determine its mechanism soon. This data may help them learn which biological pathways lead to hair loss.

There are now clinical trials taking place around the country to test the safety and efficacy of the drug for hair loss conditions. One such study lasting 3 months gave Tofacitinib to 66 people with alopecia areata (an immune system condition that causes hair to fall out in patches). Half of the people regrew some hair, and one-third had more than 50 percentof the hair on their scalp grow back, according to the 2016 study, published in the journalJCI Insight.

However, researchers are still working to determine the best dose needed, whether the results are lasting, and whether they can develop a topical form of the drug, Day said. She added that patients should be aware that Tofacitinib has side effects. Its already associated with an increased risk of serious infections, as well as stomach and intestinal tears, according to Pfizer, the manufacturer.

Besides investigating Tofacitinib, researchers are also looking at ways to clone hair or use stem cell therapy to treat alopecia, Day said.

This article first appeared on LiveScience.

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Darzalex EU nod marks first newly diagnosed MM treatment in six years – PharmaTimes

Janssen has announced that the European Commission (EC) has grantedmarketing authorisationfora new Darzalex (daratumumab) combo, for newly diagnosed, transplant eligible patients with multiple myeloma (MM).

On the news, the combination, which consists of the biologic combined with bortezomib, thalidomide and dexamethasone(VTd) is now the first regimen approved in over six years for newly diagnosed patients who are eligible for a stem cell transplant. It also means that the patient population now has their first opportunity to be treated with a monoclonal antibody.

The company says that the approval was based on results from part one of the Phase III CASSIOPEIA (MMY3006) study, which showed that after consolidation, the stringent complete response (sCR) rate was 9% higher in the Darzalex-VTd arm than the VTd alone arm.

Further, at a median follow-up of 18.8 months, PFS was significantly improved in the Darzalex-VTd group, with the addition of the drug resulting in an 18-month PFS rate of 93%, compared to 85% for VTd alone.

The effectiveness of first line treatment is critical to maximise time until relapse, explained Philippe Moreau, principal investigator and Head of the Haematology Department at the University Hospital of Nantes.

He continued, The CASSIOPEIA study answered that question definitively, demonstrating that the addition of Darzalex in combination with VTd can lead to very deep remissions and also prolong PFS. Im pleased to see the European Commission have recognised this as well.

MM is an incurable blood cancer that starts in the bone marrow and is characterised by an excessive proliferation of plasma cells. In Europe, more than 48,200 people were diagnosed with MM in 2018, with more than 30,800 deaths related to the disease.

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Darzalex EU nod marks first newly diagnosed MM treatment in six years - PharmaTimes

2-year-old battles aggressive cancer – Pekin Daily Times

PEORIA For years, the Landwehrs had supported St. Jude through donations and participating in the annual runs.

But it wasn't until their 2-year-old son was a patient at the Midwest Affiliate at OSF HealthCare Children's Hospital of Illinois here in Peoria that it really sunk in how much it means to give to the health network.

"It is a story that you never think is going to be about you," said Seth Landwehr, a detective with the Peoria Police Department and a former member of the Illinois Air National Guard's 182nd Airlift Wing. "You think you can take care of your kids. You think they are healthy. You give them things. We inspect our kids all the time with my wife being a nurse.

"This happened so quickly and it's so aggressive. If it wasn't for St. Jude and everyone down there working so fast ..." he said.

The "this" Landwehr was talking about was the recent discovery that his son, Brigham, has stage 4 neuroblastoma, an aggressive form of cancer. Diagnosed less than a month ago, the little boy is finishing up his first round of chemo, and has already had an operation to remove a softball-sized tumor from his torso, just above one of his kidneys.

"It is really hard to be on this side of the care," said Stacey Landwehr, who used to work as a pediatric intensive care nurse. "You commonly hear that doctors and nurses make the worst patients as they have been on the other side. There's the heartache, the stress and the fact that you can't clock out at the end of the day and leave it.

It's my life now," she said. "But it gives you such a deeper appreciation for everything that (St. Jude) does. I have done the St. Jude runs in the past and donate every year but it now takes on a whole new meaning. It's overwhelming. Everyone's generosity has been so overwhelming."

'Darkest days of my life'

Brigham's parents noticed a dime-sized mark below his ear a few days after Christmas. Dad, who is an optimist, said it'll be fine. Mom, the nurse, worried. They thought it could be a swollen lymph node. They kept an eye on it, and then it kept growing. A sonogram was scheduled for Jan. 9. The results caused their doctor not to like what he saw. They were walked down the hall at OSF HealthCare Saint Francis Medical Center to the St. Jude area at Children's Hospital. Still, Seth was hopeful.

"I was telling my wife that everything is fine. My wife was a peds ICU nurse. She has seen the worst of the worst of the sick babies, and she goes straight to doom and gloom," he said.

The doctors at St. Jude knew right away it was neuroblastoma, which starts in the adrenal glands. An MRI was ordered to check his face, head and neck.

They got the results on a Saturday. They had taken the kids to the car show. It was a way to keep things normal. That was, until Seth saw his son's doctor, who is also a friend, parked in his driveway.

"I knew then it wasn't good," he said.

They told the couple their son had cancer. It was stage 4, and was it was moving through their son's endocrine system. Neuroblastoma is aggressive, and given that Brigham has his blood tested every few months for congenial hyperthyroidism, they figured the cancer was only a few months old.

But in that time, it had spread to his jaw, his ribs, head, spine and lungs.

"I got the news Saturday and for the next four days, I will tell you were the darkest, deepest days of my life. I didn't have a plan, I didn't know what to do," Seth said.

Pay it back

Brigham turned 2 on Jan. 18. That same day, doctors removed the softball sized tumor from his body. And he began his first round of chemotherapy. With their ties to the military Stacey remains in the Illinois Air National Guard and Seth's position as a detective, help was quick in coming. Friends and family donated toys, time, money and many shoulders to lean on.

And St. Jude was right there, the couple says. The Midwest Affiliate Clinic's location at Children's Hospital of Central Illinois has given them cause for hope and joy.

"We have been blessed with amazing surgeons and doctors," he said. "My son turned 2 on Jan. 18, and St. Jude sent up a personalized Paw Patrol cake. It's amazing the amount of love and attention they give to these kids."

And for that, they want people to help St. Jude help others. Ten years ago, Stacey noted, the prognosis for such a diagnosis was grim. Now their son has a 70% to 80% chance of beating it. And that's due to research and care which are funded by donations and events like the St. Jude runs runs, among other things.

They want to pay it back, and one way to is urge people to support St. Jude. But there are other ways.

"Donate blood or platelets when you can. This is all very new for us, and he's (Brigham) has already received a unit of blood. There is never a shortage of need when it comes to blood," Stacey said. Another way is to reduce the risk of infections for people who have their immune systems compromised. Wash your hands. If your child is sick, keep them home so they can't get others sick.

"We have good insurance, so we'll be okay, but there are a lot of people who wouldn't be in this situation," Seth said. "You now see how important the donations are.

"I'm an ambassador for St. Jude forever now."

'Caught it quick'

Brigham has a long road in front of him. There's still plenty of cancer left in his body. He still has several rounds of chemo to undergo here and then a three-month trip up to Chicago for stem cell harvesting and more treatment there. In all, his parents believe it will take a year to finish all the treatments.

But they have hope, as it appears Brigham's cancer was caught early.

"They (the doctors) ask all these questions. 'Have you noticed anything, like verbal activity impairment, balance issues?' And we were like, 'No.' In the last two to three weeks, his vocab has exploded. His imagination is off the charts. He always wants to wrestle with dad, play Star Wars or jump off the bed and into your arms," Seth said.

Normally, kids show up with a light paralysis, can't walk or their motor functions have diminished. And, he said, it's rare to have such a large tumor removed in one fell swoop without any treatment or chemo.

"We caught it quick even though it's stage 4 and it's all over his body, he has his strength and power to fight the chemo," he said.

But boys are boys. A few days after his birthday last week, Brigham got up out of bed and grabbed one of the many toys that people had brought. He fixated on a fire truck. Seth joked it settled the age-old question of what young boys want to be when they grow up. The gift came from Peoria Police Chief Loren Marion.

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2-year-old battles aggressive cancer - Pekin Daily Times

Celavie Biosciences Presented Five-Year Follow-Up Data in Parkinsonian Patients at the World Stem Cell Summit – Financialbuzz.com

Celavie Biosciences, LLC, a company working to improve lives and restore hope by advancing innovations in CNS diseases with regenerative stem cell-based therapies, today announced their presentation of a poster, titled Five year follow-up on the first-in-human transplantation of undifferentiated stem cells into Parkinsonian patients reveals no adverse effects with improvement in motor function or arrest of the disease progression in five out of seven patients, at the Phacilitate Leaders World and World Stem Cell Summit, held January 21-24 in Miami, Florida.

The poster shows five-year follow-up data that expands on the exploratory clinical data in 7 PD patients with four-year follow-up published in Cell Transplantation in 2018. Oleg Kopyov, Executive Vice President and Chief Scientific Officer at Celavie, presented the poster on-site at the Miami Hyatt Regency.

In the results at one year after cell grafting, all but two of the seven patients completing the study showed various degrees of motor improvement, and five of them showed better response to medication. At five-year evaluation, Unified Parkinsons Disease Rating Scale III (UPDRS III) scores remained better than at baseline in 4/7 patients in the OFF condition and in 5/7 patients in the ON condition. None of the patients showed unwanted motor disturbances (dyskinesias), tumor formation, or any detectable immune responses to the grafted cells.

We are excited that the five-year data for our exploratory clinical trial suggest that the neural progenitor cells are able to stop or slow down the motor deterioration in Parkinsons patients that one would expect to see in this timespan, showing continued improvement even compared to the fourth year, said Oleg Kopyov. We anticipate filing an IND with the FDA for a Phase I U.S. trial in patients with moderate to advanced Parkinsons disease this year.

In addition, Sandy Solmon, Celavies CEO, will deliver presentations at two upcoming international industry conferences:

Ms. Solmon will discuss Celavies application of the companys human undifferentiated allogeneic pluripotent stem cells in Parkinsons disease, as well as pre-clinical data in cerebellar ataxia and upcoming milestones. To schedule a meeting with Celavie Biosciences at these conferences, please contact: Mary Beth Cicero at mbcicero@lavoiehealthscience.com.

About the World Stem Cell Summit

Produced by the non-profit Regenerative Medicine Foundation (RMF), and in its 15th year, the World Stem Cell Summit will take place January 21-24, 2020, in Miami, Florida in partnership with Phacilitate Leaders World, as part of Advanced Therapies Week. The Summit is the most inclusive and expansive interdisciplinary, networking, and partnering meeting in the stem cell science and regenerative medicine field. With the overarching purpose of fostering translation of biomedical research, funding, and investments targeting cures, the Summit and co-located conferences serve a diverse ecosystem of stakeholders. For more information about the upcoming World Stem Cell Summit in Miami, please visit: http://www.worldstemcellsummit.com.

About Celavie Biosciences

Celavie Biosciences is a privately-held company whose mission is to improve lives and restore hope by advancing regenerative stem cell therapies for the treatment of Parkinsons disease and other disorders of the central nervous system (CNS). The company develops undifferentiated, unmodified allogeneic pluripotent stem cell-based therapies, holds a strong IP portfolio, including 18 issued patents, and has an experienced management team blending expertise in concept and cell technology, product scalability and entrepreneurship. Celavet, a subsidiary, applies the same proprietary technologies for the treatment and prevention of serious veterinary diseases. More information is available at https://www.celavie.com/.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200122005497/en/

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Celavie Biosciences Presented Five-Year Follow-Up Data in Parkinsonian Patients at the World Stem Cell Summit - Financialbuzz.com