RenovaCare (OTCMKTS:RCAR) and Haemonetics (NYSE:HAE) are both medical companies, but which is the better business? We will compare the two companies based on the strength of their analyst recommendations, risk, earnings, profitability, dividends, valuation and institutional ownership.

Analyst Ratings

This is a breakdown of recent ratings and price targets for RenovaCare and Haemonetics, as reported by MarketBeat.com.

Haemonetics has a consensus price target of $144.40, suggesting a potential upside of 15.46%. Given Haemonetics higher possible upside, analysts clearly believe Haemonetics is more favorable than RenovaCare.

Risk and Volatility

RenovaCare has a beta of 2.42, suggesting that its share price is 142% more volatile than the S&P 500. Comparatively, Haemonetics has a beta of 0.79, suggesting that its share price is 21% less volatile than the S&P 500.

Valuation and Earnings

This table compares RenovaCare and Haemonetics revenue, earnings per share (EPS) and valuation.

Haemonetics has higher revenue and earnings than RenovaCare.

Profitability

This table compares RenovaCare and Haemonetics net margins, return on equity and return on assets.

Institutional and Insider Ownership

99.1% of Haemonetics shares are owned by institutional investors. 0.9% of RenovaCare shares are owned by company insiders. Comparatively, 1.3% of Haemonetics shares are owned by company insiders. Strong institutional ownership is an indication that hedge funds, large money managers and endowments believe a company is poised for long-term growth.

Summary

Haemonetics beats RenovaCare on 9 of the 10 factors compared between the two stocks.

RenovaCare Company Profile

RenovaCare, Inc., a development-stage company, focuses on the acquisition, development, and commercialization of autologous cellular therapies for use in medical and aesthetic applications. It is developing CellMist System for spraying a patient's own skin stem cells onto burns and wounds for self-healing; and SkinGun, a solution sprayer device for delivering the cells to the treatment area. The company was formerly known as Janus Resources, Inc. and changed its name to RenovaCare, Inc. in January 2014. RenovaCare, Inc. is based in Scottsdale, Arizona.

Haemonetics Company Profile

Haemonetics Corporation, a healthcare company, provides hematology products and solutions. The company operates through five segments: North America Plasma; Americas Blood Center and Hospital; Europe, Middle East and Africa; Asia Pacific; and Japan. It offers automated plasma collection devices and related disposables, including NexSys PCS plasmapheresis system and PCS2 equipment and disposables, plasma collection containers, and intravenous solutions, as well as information technology platforms for plasma customers to manage their donors, operations, and supply chain; and NexLynk DMS donor management system. The company also provides automated blood component and manual whole blood collection systems, such as MCS brand apheresis equipment to collect specific blood components from the donor; disposable whole blood collection and component storage sets; SafeTrace Tx and El-Dorado Donor donation and blood unit management systems; Hemasphere software that provides support for blood drive planning; and Donor Doc and e-Donor software to enhance the donor recruitment and retention. In addition, it offers hospital products comprising TEG diagnostic systems that enables clinicians to assess the coagulation status of a patient at the point-of-care or laboratory setting; TEG Manager software, which connects various TEG analyzers throughout the hospital, providing clinicians remote access to active and historical test results that inform treatment decisions; Cell Saver Elite +, a surgical blood salvage system for cardiovascular, orthopedic, trauma, transplant, vascular, obstetrical, and gynecological surgeries; OrthoPAT, a perioperative autotranfusion system for orthopedic procedures; and BloodTrack, a suite of blood management and bedside transfusion solutions that combines software with hardware components, as well as an extension of the hospital's blood bank information system. Haemonetics Corporation was founded in 1971 and is headquartered in Braintree, Massachusetts.

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Contrasting RenovaCare (OTCMKTS:RCAR) and Haemonetics (OTCMKTS:HAE) - Covington Journal

Skin Stem Cells Comments Off on Contrasting RenovaCare (OTCMKTS:RCAR) and Haemonetics (OTCMKTS:HAE) – Covington Journal | October 17th, 2019

A case study published in Leukemia and Lymphoma described a patient with a diagnosis of Waldenstrm Macroglobulinemia (WM) that had subsequently undergone histological transformation to refractory high grade B-cell lymphoma and was successfully treated with CD19-targeted chimeric antigen receptor (CAR)-T cell therapy.1

WMis a type of B-cell non-Hodgkin lymphoma(NHL), typically characterized by overproduction of monoclonal immunoglobulinM, as well as infiltration of malignant lymphoplasmacytic cells into the bonemarrow.

Although considered incurable, WM often follows an indolent course andsome patients can be asymptomatic for long periods. Rarely, the diseasetransforms into a more aggressive form of NHL that has been associated with apoor prognosis.

The patient described in the case studywas a 71-year-old man who was first diagnosed with WM in 1998. The patient wasmonitored without undergoing active treatment for a period of 12 years, atwhich time he developed anemia and splenomegaly. At that time, he underwenttreatment with 6 cycles of fludarabine and rituximab and achieved a partial response totreatment. Following a worsening of symptoms 4 years later, the patient wastreated with 6 cycles of bendamustine and rituximab.

Biopsyof an enlarged cervical lymph node performed at that time revealed high-gradeB-cell lymphoma that was clonally related to the previously seenlymphoplasmacytic infiltrate, consistent with transformation.

Thepatient subsequently achieved a complete response to 6 cycles of rituximab,cyclophosphamide, doxorubicin, vincristine, prednisone (R-CHOP) plus ibrutinibfollowed by 6 months of ibrutinib maintenance therapy that lasted for 18months.

Salvagetherapy included 2 cycles of rituximab, dexamethasone, cytarabine, cisplatin(R-DHAP) followed by 1 cycle of rituximab plus high-dose cytarabine, followedby autologous stem cell transplantation several months later.

Asthe patients disease was considered to be chemorefractory based on subsequent imagingand pathological analyses, he was treated with axicabtagene ciloleucel, aCD19-targeted CAR-T cell therapy currently approved for the treatment of adultpatients with relapsed or refractory large B-cell lymphoma, including high gradeB-cell lymphoma, after 2 or more lines of systemic therapy.2

Althoughthe patient experienced pancytopenia, grade 1 cytokine release syndrome, andgrade 1 neurotoxicity following CAR-T therapy, he achieved a complete response1 month following treatment.

Notably,there was no evidence of either underlying WM or transformed disease at 6 and 12months follow-up.

Thestudy authors noted that longer term follow up in this patient will beinformative, as late relapses have occurred even in patients who achieve a deepresponse after transplant. CAR-T cell therapy may be an effective treatment forrelapsed or refractory WM that has not yet undergone histologicaltransformation, as CD19 is almost universally expressed on lymphoplasmacyticlymphoma cells. The researchers concluded that further analysis of this iswarranted in the context of clinical trials.

References

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Benefit of CD19-Targeted CAR-T Therapy in Patients With Transformed Waldenstrm Macroglobulinemia - Cancer Therapy Advisor

Bone Marrow Stem Cells Comments Off on Benefit of CD19-Targeted CAR-T Therapy in Patients With Transformed Waldenstrm Macroglobulinemia – Cancer Therapy Advisor | October 16th, 2019

BALTIMOREand PHILADELPHIA and IRVINE, Calif., Oct. 15, 2019 (GLOBE NEWSWIRE) -- WindMIL Therapeutics and the University of California, Irvine (UCI) today announced that the first patients have been identified in an investigator-sponsored study for the collection of bone marrow from patients with gliomas. The study will evaluate generating marrow infiltrating lymphocytes (MILs) for these patients through WindMILs proprietary cellular activation and expansion process. The study is being conducted at UCI.

Patients suffering with glioblastoma are in great need of new, promising treatments that might advance the current standard of care, said Daniela A. Bota, MD, PhD, director of theUCI Health Comprehensive Brain Tumor Program,seniorassociate dean for clinical research, UCI School of Medicine and clinical director, UCI Sue & Bill Gross Stem Cell ResearchCenter. The University of California, Irvine is excited toplay a key role in research that may lead to a clinical trial that enlists the immune system in novel ways to fight this terrible disease.

Gliomas are the most common of the malignant brain tumors. Glioblastoma, the most common glioma, has a five-year survival of less than 5 percent. Additional treatment options are urgently needed for these patients. Adoptive immunotherapy is a possible approach for gliomas and the use of MILs, a cell therapy that is naturally tumor-specific, is one such treatment option.

The bone marrow is a unique niche in the immune system to which antigen-experienced memory T cells traffic and are then maintained. WindMIL has developed a proprietary process to select, activate and expand these memory T cells into MILs. Because memory T cells in bone marrow occur as a result of the immune systems recognition of tumor antigens, MILs are specifically suited for adoptive cellular immunotherapy and are able to directly eradicate or facilitate eradication of each patients unique cancer. WindMIL is currently studying MILs in multiple myeloma, non-small cell lung cancer and squamous cell carcinoma of the head and neck, and plans to expand into other solid tumors.

WindMIL is looking forward to working with the University of California, Irvine on this exciting project and is optimistic that MILs may offer the potential to help patients with these hard-to-treat diseases, said Monil Shah, PharmD, MBA, Chief Development Officer at WindMIL.

About WindMIL Therapeutics

WindMIL Therapeutics is a clinical-stage company developing a novel class of autologous cell therapies based on marrow infiltrating lymphocytes (MILs) for cancer immunotherapy. As the leader in cellular therapeutics emanating from bone marrow, WindMIL translates novel insights in bone marrow immunology into potentially life-saving cancer immunotherapeutics for patients. WindMIL believes that Cell Source Matters and the companys proprietary process to extract, activate and expand these cells offers unique immunotherapeutic advantages, including inherent poly-antigen specificity, high cytotoxic potential and long persistence. For more information, please visit: http://www.windmiltx.com.

About UCI Health

UCI Healthcomprises the clinical enterprise of the University of California, Irvine. Patients can access UCI Health at primary and specialty care offices across Orange County and at its main campus, UCI Medical Center in Orange, California. The 417-bed acute care hospital provides tertiary and quaternary care, ambulatory and specialty medical clinics and behavioral health and rehabilitation services. UCI Medical Center features Orange Countys only National Cancer Institute-designated comprehensive cancer center, high-risk perinatal/neonatal program and American College of Surgeons-verified Level I adult and Level II pediatric trauma center and regional burn center. UCI Health serves a region of nearly 4 million people in Orange County, western Riverside County and southeast Los Angeles County. Follow us onFacebookandTwitter.

About the University of California, Irvine

Founded in 1965, UCI is the youngest member of the prestigious Association of American Universities. The campus has produced three Nobel laureates and is known for its academic achievement, premier research, innovation and anteater mascot. Led by Chancellor Howard Gillman, UCI has more than 36,000 students and offers 222 degree programs. Its located in one of the worlds safest and most economically vibrant communities and is Orange Countys second-largest employer, contributing $5 billion annually to the local economy. For more on UCI, visitwww.uci.edu.

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WindMIL Therapeutics and University of California, Irvine Announce Collaboration to Collect Bone Marrow from Patients with Gliomas to Develop Marrow...

Bone Marrow Stem Cells Comments Off on WindMIL Therapeutics and University of California, Irvine Announce Collaboration to Collect Bone Marrow from Patients with Gliomas to Develop Marrow… | October 16th, 2019

Organizers of this years Light the Night, which is held in support of blood cancer research, hope members of the community can join them Oct. 19 as they take to the Rotary Trail to raise awareness and funds for the eradication of various blood cancers.

Leukemia, lymphoma, Hodgkins lymphoma and myeloma can all be beat, but for every inspirational tale of perseverance and survival, there are even more about those who could not hang on long enough and died as a result of the fast moving diseases that affect blood cells, bone marrow and lymph nodes to name a few.

I lost my niece to leukemia 11 years ago, she was 19, said Westlock organizer Joanne Rimmer, with tears in her eyes as fresh as the day she lost her niece.

I had positive thoughts the whole time, I thought she was going to get over it and everything was going to be back to normal and it didnt work out. I didnt want other families to go through that, so I thought it was a good thing to help raise money.

It has been 11 years and Im still crying. Its one way we can do something to say we really miss her.

Rimmer also has a close friend who was able to survive leukemia after a donation of stem cells from her brother saved her life. She also has another friend in Manitoba who is currently fighting off leukemia, with some success. These are her reasons for lighting up the night, so called because participants often hold lanterns of different colours that denote how thatindividual has been affected.

White lanterns are carried by survivors, gold is in remembrance and red is in support, which together makes for quite a sea of colour moving through the town.

Rimmer, who has been taking part in the event in one form or another for 11 years and helping to organize the Westlock event for the last six years is a proponent of having a bare-bones event with little flash or overhead to make sure as much money as possible is donated to the Leukemia and Lymphoma Society of Canada.

Any sponsors or potential contributors are asked to donate directly to the cause instead of providing other supports, which are appreciated, but ultimately unneeded.

I want every dollar that gets raised to actually go towards what were actually raising money for. I dont want to waste it on silly things.

Rimmer is urging those who want to participate to start collecting sponsors, and to register at http://www.lightthenight.ca. Participants should meet at the Rotary Spirit Centre after 6:30 p.m. Oct. 19 for the walk that will begin at 7 p.m., winding it way east on the Rotary Trail to the healthcare centre, then eventually on to the pool. All are welcome to join, from babies in strollers to their great-great parents, said Rimmer, who also noted the event will take place rain or shine.

Link:
Light the Night Oct. 19 - Westlock News

Bone Marrow Stem Cells Comments Off on Light the Night Oct. 19 – Westlock News | October 16th, 2019

This article, written byKatharine Sedivy-Haley, University of British Columbia, originally appeared on The Conversation and is republished here with permission:

When I was applying to graduate school in 2012, it felt like stem cells were about to revolutionize medicine.

Stem cells have the ability to renew themselves, and mature into specialized cells like heart or brain cells. This allows them to multiply and repair damage.

If stem cell genes are edited to fix defects causing diseases like anemia or immune deficiency, healthy cells can theoretically be reintroduced into a patient, thereby eliminating or preventing a disease. If these stem cells are taken or made from the patient themselves, they are a perfect genetic match for that individual, which means their body will not reject the tissue transplant.

Because of this potential, I was excited that my PhD project at the University of British Columbia gave me the opportunity to work with stem cells.

However, stem cell hype has led some to pay thousands of dollars on advertised stem cell treatments that promise to cure ailments from arthritis to Parkinsons disease. These treatments often dont help and may harm patients.

Despite the potential for stem cells to improve medicine, there are many challenges as they move from lab to clinic. In general, stem cell treatment requires we have a good understanding of stem cell types and how they mature. We also need stem cell culturing methods that will reliably produce large quantities of pure cells. And we need to figure out the correct cell dose and deliver it to the right part of the body.

Embryonic, 'induced and pluripotent

Stem cells come in multiple types. Embryonic stem cells come from embryos which makes them controversial to obtain.

A newly discovered stem cell type is the induced pluripotent stem cell. These cells are created by collecting adult cells, such as skin cells, and reprogramming them by inserting control genes which activate or induce a state similar to embryonic stem cells. This embryo-like state of having the versatile potential to turn into any adult cell type, is called being pluripotent.

However, induced pluripotent and embryonic stem cells can form tumours. Induced pluripotent stem cells carry a particularly high risk of harmful mutation and cancer because of their genetic instability and changes introduced during reprogramming.

Genetic damage could be avoided by using younger tissues such as umbilical cord blood, avoiding tissues that might contain pre-existing mutations (like sun-damaged skin cells), and using better methods for reprogramming.

Stem cells used to test drugs

For now, safety concerns mean pluripotent cells have barely made it to the clinic, but they have been used to test drugs.

For drug research, it is valuable yet often difficult to get research samples with specific disease-causing mutations; for example, brain cells from people with amyotrophic lateral sclerosis (ALS).

Researchers can, however, take a skin cell sample from a patient, create an induced pluripotent stem-cell line with their mutation and then make neurons out of those stem cells. This provides a renewable source of cells affected by the disease.

This approach could also be used for personalized medicine, testing how a particular patient will respond to different drugs for conditions like heart disease.

Vision loss from fat stem cells

Stem cells can also be found in adults. While embryonic stem cells can turn into any cell in the body, aside from rare newly discovered exceptions, adult stem cells mostly turn into a subset of mature adult cells.

For example, hematopoietic stem cells in blood and bone marrow can turn into any blood cell and are widely used in treating certain cancers and blood disorders.

A major challenge with adult stem cells is getting the right kind of stem cell in useful quantities. This is particularly difficult with eye and nerve cells. Most research is done with accessible stem cell types, like stem cells from fat.

Fat stem cells are also used in stem cell clinics without proper oversight or safety testing. Three patients experienced severe vision loss after having these cells injected into their eyes. There is little evidence that fat stem cells can turn into retinal cells.

Clinical complications

Currently, stem cell based treatments are still mostly experimental, and while some results are encouraging, several clinical trials have failed.

In the brain, despite progress in developing treatment for genetic disorders and spinal cord injury, treatments for stroke have been unsuccessful. Results might depend on method of stem cell delivery, timing of treatment and age and health of the patient. Frustratingly, older and sicker tissues may be more resistant to treatment.

For eye conditions, a treatment using adult stem cells to treat corneal injuries has recently been approved. A treatment for macular degeneration using cells derived from induced pluripotent stem cells is in progress, though it had to be redesigned due to concerns about cancer-causing mutations.

A path of cautious optimism

While scientists have good reason to be interested in stem cells, miracle cures are not right around the corner. There are many questions about how to implement treatments to provide benefit safely.

In some cases, advertised stem cell treatments may not actually use stem cells. Recent research suggests mesenchymal stem cells, which are commonly isolated from fat, are really a mixture of cells. These cells have regenerative properties, but may or may not include actual stem cells. Calling something a stem cell treatment is great marketing, but without regulation patients dont know what theyre getting.

Members of the public (and grad students) are advised to moderate their excitement in favour of cautious optimism.

Katharine Sedivy-Haley, PhD Candidate in Microbiology and Immunology, University of British Columbia

This article is republished from The Conversation under a Creative Commons license. Read the original article.

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BEYOND LOCAL: Expert recommends 'path of cautious optimism' about the future of stem cell treatment - TimminsToday

Spinal Cord Stem Cells Comments Off on BEYOND LOCAL: Expert recommends ‘path of cautious optimism’ about the future of stem cell treatment – TimminsToday | October 16th, 2019

ZIOPHARM Oncology Inc. (NASDAQ:ZIOP) and Neuralstem Inc. (NASDAQ:CUR) compete against each other in the Biotechnology sector. We will compare them and contrast their analyst recommendations, profitability, institutional ownership, risk, dividends, earnings and valuation.

Earnings and Valuation

In table 1 we can see ZIOPHARM Oncology Inc. and Neuralstem Inc.s top-line revenue, earnings per share and valuation.

Profitability

Table 2 has ZIOPHARM Oncology Inc. and Neuralstem Inc.s return on equity, return on assets and net margins.

Volatility and Risk

ZIOPHARM Oncology Inc. has a 2.55 beta, while its volatility is 155.00% which is more volatile than S&P 500. Neuralstem Inc. on the other hand, has 1.94 beta which makes it 94.00% more volatile compared to S&P 500.

Liquidity

ZIOPHARM Oncology Inc. has a Current Ratio of 8.9 and a Quick Ratio of 8.9. Competitively, Neuralstem Inc.s Current Ratio is 3.8 and has 3.8 Quick Ratio. ZIOPHARM Oncology Inc.s better ability to pay short and long-term obligations than Neuralstem Inc.

Analyst Recommendations

The Recommendations and Ratings for ZIOPHARM Oncology Inc. and Neuralstem Inc. are featured in the next table.

ZIOPHARM Oncology Inc. has an average price target of $6.5, and a 51.52% upside potential.

Institutional and Insider Ownership

Roughly 45.3% of ZIOPHARM Oncology Inc. shares are owned by institutional investors while 4.9% of Neuralstem Inc. are owned by institutional investors. ZIOPHARM Oncology Inc.s share owned by insiders are 0.5%. Competitively, Neuralstem Inc. has 1% of its share owned by insiders.

Performance

In this table we show the Weekly, Monthly, Quarterly, Half Yearly, Yearly and YTD Performance of both pretenders.

For the past year ZIOPHARM Oncology Inc. has 271.12% stronger performance while Neuralstem Inc. has -64.87% weaker performance.

Summary

ZIOPHARM Oncology Inc. beats Neuralstem Inc. on 10 of the 11 factors.

ZIOPHARM Oncology, Inc., a biotechnology company, focuses on acquiring, developing, and commercializing a portfolio of cancer therapies that address unmet medical needs through synthetic immuno-oncology. The company, through its collaboration agreement with Intrexon Corporation, holds certain rights to Intrexons synthetic immuno-oncology platform for use in the field of oncology, which includes a clinical stage product candidate, Ad-RTS-IL-12 evaluated for the treatment of metastatic melanoma, unresectable recurrent or metastatic breast cancer, and Grade III malignant glioma (GBM). Its synthetic immuno-oncology platform employs an inducible gene-delivery system that enables controlled in vivo expression of genes that produce therapeutic proteins to treat cancer. The company, under its license agreement with The University of Texas MD Anderson Cancer Center, along with Intrexon hold license to certain technologies relating to novel chimeric antigen receptor (CAR) T cell therapies, non-viral gene transfer systems, genetic modification and/or propagation of immune cells and other cellular therapy approaches, Natural Killer cells, and T cell receptors. It also has a research and development agreement with the National Cancer Institute utilizing Sleeping Beauty System to generate T cells receptors for the treatment of solid tumors. ZIOPHARM Oncology, Inc. is headquartered in Boston, Massachusetts.

Neuralstem, Inc., a clinical stage biopharmaceutical company, focuses on the research and development of nervous system therapies based on its proprietary human neuronal stem cells and small molecule compounds. The companys stem cell based technology enables the isolation and expansion of human neural stem cells from various areas of the developing human brain and spinal cord enabling the generation of physiologically relevant human neurons of various types. It is developing products include NSI-189, a chemical entity, which is in Phase II clinical trial for the treatment of major depressive disorder, as well as is in preclinical programs for the MCAO stroke, type 1 and 2 diabetes related neuropathy, irradiation-induced cognition, long-term potentiation enhancement, and angelman syndrome. The company is also developing NSI-566, which has completed Phase II clinical trial for treating amyotrophic lateral sclerosis disease, as well as is in Phase I clinical trials for the treatment of chronic spinal cord injury and motor deficits due to ischemic stroke. Neuralstem, Inc. was founded in 1996 and is headquartered in Germantown, Maryland.

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Contrasting of ZIOPHARM Oncology Inc. (ZIOP) and Neuralstem Inc. (NASDAQ:CUR) - MS Wkly

Spinal Cord Stem Cells Comments Off on Contrasting of ZIOPHARM Oncology Inc. (ZIOP) and Neuralstem Inc. (NASDAQ:CUR) – MS Wkly | October 16th, 2019

It hurt Margaret Godfrey to see daughter Rachel Carter in pain, so she painted.

And once the worst pain had passed, Carter needed a way to move forward, so she wrote.

In 2000, at age 24, Carter was diagnosed with multiple sclerosis, a disease where the immune system eats away at the lining of nerves and impairs or disables the brain and central nervous system.

Carter is now a married 43-year-old mother of three who lives in Ridgefield. But when she was diagnosed with MS, it marked a significant shift in her life. The woman who ran marathons, rode a motorcycle to high school and was once a deckhand on a small riverboat cruise ship to prove a point because she was told a woman couldnt hold that job, slowly morphed as her disease altered life.

It started with a fall on vacation during a run. Then falling on runs became more frequent. Gone went the marathons. In came the treadmill. Slowly, over the course of 12 years, the MS progressed and symptoms became debilitating. Running left completely. Neuropathic pain caused her to feel like she was sunburned in the morning, and by the afternoon or evening it felt like third degree burns. Carter left her job in sales in 2013.

I would always end up in tears in the bed, Carter said.

Carter has chronicled her battle with MS, and a stem cell treatment she credits with turning her prognosis around, in a new book called Enduring the Cure: My MS Journey to the Brink of Death and Back. Her mother helped her edit the book, and provided the books cover art. Adrienne van Der Valk also edited the book.

Through pain, Carter and her mother have created art. About five years ago, Carter underwent an experimental stem cell transplant in Seattle to help her rebuild her immune system. Carter lived in Seattle for about three months, while her parents watched two of her children, and the other child lived with an aunt.

As part of the procedure, Carters own stem cells were harvested and then reintroduced to her body after chemotherapy depleted most of her immune system. Its what she described as a rebooting of her whole immune system, in hopes that it would stop attacking her brain and spinal cord. Carter still has her previous brain damage, which impacts her memory and brain power; but so far, the stem cell transplant has improved her condition greatly, she said.

Carter has more energy she used to sometimes not be able to leave bed some days. She also can stand upright and walk easily she used to rely on a walker. And her pain is minimal now, the biggest improvement.

Carters family kept notes during her treatment, and Carter decided to use those, and the memories of her family, to help her write the book with van Der Valk.

Thinking was very fatiguing for Carter, since she still has the brain damage from MS. She could only work about one to two hours on the book at a time. She said writing was frustrating and hard in many ways. Parts of my brain are gone, Carter explained. She said it requires her about 10 times as much brain matter as the average person to process something simple.

Its so much pressure to have something you feel like you have to do, and then you cant do it. I had so little energy, Carter said. I cant explain very well because so many words dont come to me.

In spite of the challenges, Carter feels like sharing her experience will help others because, in life, we all have hard situations, she said.

Godfrey, a 70-year-old painter who lives in Blue River, Ore., found art in the familys experience. She drew inspiration from Gustav Klimt, an Austrian symbolist, who painted in the late 1800s and early 1900s, and created symbolic paintings of her daughter. Godfrey said art makes her an intellectual because of how she has to think about what shes painting. This time she was researching her own child.

Godfrey did a painting of Carter surrounded by old running shoes, a life she had to give up. She did a painting of Carter without her hair, which centered on Carters worries. She did a painting of Carter covered by a quilt with her three kids on it, what kept her going and allowed her to recover. She did a painting of Carter lying in a hospital bed, looking exhausted and resigned, which is the books cover. The final painting in the series, called The Journey Is Not Done, features Carter with hair, months after treatment. It has an unfinished mosaic because Carters journey isnt over.

Creating art is a very meditative thing, Godfrey said. Rather than getting wrapped up in emotions, once I start painting the world is me and paper. All the worries of life disappear. It was a way to enjoy that experience of just being able to throw out my expression, without getting too emotionally tangled up.

More:
Ridgefield woman, her mother write book about battle with MS - The Columbian

Spinal Cord Stem Cells Comments Off on Ridgefield woman, her mother write book about battle with MS – The Columbian | October 16th, 2019

The Duchess of York, affectionately known by British royal fans as Fergie, shared the cover of The Perfect World Magazine, where she can be seen giving Sir David Attenborough an honorary award. Fergie, who turns 60 today, revealed in an Instagram post that moment is one of the best of her life.

She wrote on Instagram: "Thank you all for the birthday messages! This is one of the highlights of my life."

In the cover, Fergie can be seen smiling as she hands the award, shaped like a rhino, toSir Attenborough, the world-famousEnglish broadcaster and natural historian.

Fergie andSir Attenborough attended together last September thePerfect World Foundation gala dinner, where she will present him the award.

The same award has been previously given toMark Shand, the late brother of the Duchess of Cornwall, and primatologist Dr Jane Goodall.

In the early afternoon, the Duchess of York published on Instagram another picture, showing her wearing a colourful sari.

She added in the caption: "Colourful moments"

Fergie's Instagram messages come after her former husband Prince Andrew celebrated her birthday on social media.

Earlier this morning, the Duke of Yorkpublished two identical messages dedicated to Fergie on both Instagram and Twitter.

READ MORE:How Fergie was 'forced to cancel birthday party due to crisis'

The messages read: "Wishing Sarah Ferguson a very Happy 60th Birthday!"

On Instagram, the message was accompanied by three pictures, one depicting Sarah surrounded by members of the York family, including Princess Eugenie's husband Jack Brooksbank and Princess Beatrice's fiancee Edoardo Mapelli Mozzi.

The second picture shows Fergie surrounded by children supported by the charity she is a patron of, Street Child UK.

And the third snap depicts Eugenie, Beatrice and Fergie together while carrying out an engagement.

DON'T MISS

Fergie has previously spoke with enthusiasm about her approaching 60th birthday.

Speaking at a charity event held onbehalf of Street Child and hosted by Hello, the Duchess said:"I think it's really important that my life is beginning at 60, I'm so excited by it."

Outspoken Fergie also revealed she has undertaken laser treatment to her skin to look her best for her milestone birthday.

Similarly, the Duchess had laser also before the wedding of her youngest daughter Princess Eugenie, which helped to make her skin glow.

But these haven't been the only beauty treatments Fergie has undertaken in the past years.

In March this year, Fergie travelled to the Bahamas toundergo stem cell therapy - an alternative to surgery which is yet to be proven fully safe and effective on humans and is therefore not widely available in the UK.

This therapy helped her solving a painful problem related to her feet, which eventually forced her to stop wearing high heels.

I went to the Bahamas in March to have the treatment.

I think my toes were ruined by all the riding I did when I was young.

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Sarah Ferguson reveals highlight of my life as she celebrates 60th birthday - Express

Skin Stem Cells Comments Off on Sarah Ferguson reveals highlight of my life as she celebrates 60th birthday – Express | October 16th, 2019

Medical skin care products are used for beautifying or to address some other skin care problems. The cosmetic industry is booming and skin care forms a very huge part of this industry. The aesthetic appearance is so important that people spend a lot on skin care products and treatment. People being more technologically aware of the various new skin care products trending in the market. In addition to the aesthetic application, the medical skin care products are also used to address issues such as acne, pimples or scars.

Medical Skin Care Products Market: Drivers and Restraints

The medical skin care products is primarily driven by the need of natural based active ingredients products which are now trending in the market. Consumers demand medical skin care products which favor health and environment. Moreover, the consumers are updated with the trends so that various companies end up providing such products to satisfy the customers. For instance, a single product face mask has thousands of different variants.

This offers consumers different options to select the product depending on the skin type. Moreover, the market players catering to the medical skin care products are offering products with advanced technologies. For instance, Santinov launched the CICABEL mask using stem cell material based on advanced technologies. The stem cells used in the skin care product helps to to protect and activate the cells and promote the proliferation of skin epidermal cells and the anagenesis of skin fibrosis.

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Medical Skin Care Products Market: Segmentation

On the basis of product type the medical skin care products market can be segmented as:

On the basis of application, the medical skin care products market can be segment as:

On the basis of distribution channel, the medical skin care products market can be segment as:

Medical Skin Care Products Market: Overview

Medical skin care products are used to address basic skin problems ranging from acne to scars. There are various advancements in the ingredients used to offer skin care products to the consumers. For instance, the use of hyaluronic acid and retinoids is the latest development in the industry. The anti-aging creams are at the forefront as the help treating issues such as wrinkles, scars, acne, and sun damage. Another, product in demand is the probiotic skincare which include lactobacillus and bifidobacterium.

Medical Skin Care Products Market: Region-wise Outlook

In terms of geography, medical skin care products market has been divided into five regions including North- America, Asia- Pacific, Middle-East & Africa, Latin America and Europe. North America dominated the global medical skin care products market as international players are acquiring domestic companies to make their hold strong in the U.S. LOral is accelerating its U.S. market by signing a definitive agreement with Valeant Pharmaceuticals International Inc. to acquire CeraVe, AcneFree and Ambi skin-care brands for US$ 1.3 billion. The acquisition is expected LOreal to get hold of the brands in the price-accessible segment. Asia Pacific is expected to be the fastest growing region owing to the increasing disposable income and rising awareness towards the skin care products.

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Medical Skin Care Products Market: Key Market Participants

Some of the medical skin care products market participants are Avon Products Inc., Beiersdorf AG, Colgate-Palmolive Company, Kao Corporation, LOral S.A., Procter & Gamble, Shiseido Company, The Estee Lauder Companies Inc., Unilever PLC, Revlon, Clinique Laboratories, llc., Murad, LLC., SkinCeuticals, RMS Beauty, J.R. Watkins and 100% PURE.

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Medical Skin Care Products Market Promising Growth Opportunities over 2017 2025 - Lake Shore Gazette

Skin Stem Cells Comments Off on Medical Skin Care Products Market Promising Growth Opportunities over 2017 2025 – Lake Shore Gazette | October 15th, 2019

Ray almost missed it, the message that would change his life. On a Saturday in March 2018, just as he was about to take his dog for an afternoon walk, he pulled his phone from his pocket and discovered a string of voicemails. Eight years had passed since the bomb had blown up underneath him while he was on patrol in Afghanistan, five since hed first met his doctor. Hed been on the waiting list a year. He was getting impatient.

He dialed back. This is it, he thought. It has to be.

A nurse picked up. Ray needed to come to the hospital immediately, she said. They had a donor. He was getting a new penis.

Ray had carried his unseen injury for yearsalways furtive, always anxious, always wondering how anyone who found out might react. Having lost both legs in the blast didnt bother him that much; Ray often left the house in the summertime wearing shorts, his prosthetics shining in the sun. But his other injury? Aside from his parents, hardly anyone knewnot even the guys he went to war with.

For men like Ray who lose their genitals, the usual treatmentif there was anywas phalloplasty: a rolled tube of tissue, blood vessels, and nerves taken from the forearm or thigh and transplanted to the groin, an ersatz penis that needs an external pump to get erect. When he first met with plastic surgeon Richard Redett, an expert in genital reconstruction at Johns Hopkins Hospital in Baltimore, phalloplasty was what he was offered. But soon after, Redett decided Ray could be a candidate for one of the worlds first full penis transplants. Not a crude substitute; the real thing.

This was actually something that could fix me, says Ray. I could go back to being normal again.

Penis transplantation is a radical frontier of modern medicine: extremely rare, expensive, and difficult to perform. Replacing a major organ like a damaged liver is one thing: it contains just one type of tissue. But grafting a penis from a deceased donor onto a living recipient is a chaotic amalgamation that entails stitching millimeters-wide blood vessels and nerves with minuscule sutures.

In 2013, when Ray first went to Johns Hopkins, there was no precedent for such a transplant. Since then, only four patients have had one.

South African urologist Andre Van der Merwe completed the first-ever successful transplant in 2014, sewing a donor penis onto a 21-year-old whose own had turned gangrenous after a grisly circumcision. In 2016, doctors at Massachusetts General Hospital transplanted a donor organ onto 64-year-old Thomas Manning, who had lost his penis to cancer. A year later, Van der Merwe and his team at Tygerberg Academic Hospital in Cape Town repeated their procedure on a 41-year-old victim of another circumcision gone wrong. Ray became patient number four.

After getting off the phone with the nurse that Saturday afternoon, he went into action. With military precision, Ray called his parents, packed the items he would need, boarded his dog, and made his way to the hospital. He checked in, as requested, at 1:30 on Sunday morning. At 2 a.m. Monday, he lay anesthetized on an operating table. And 14 hours after that, Redett and his team had completed the procedure. It was the most extensive penis transplant ever performed, and the first for a military veteran anywhere in the world.

Ray had been a US Navy corpsman trudging through Afghanistan when Taliban fighters ambushed his squad in 2010. As he rushed to give first aid to a downed soldier, he stepped on a roadside bomb. I remember everything froze and I was upside down, he says. I remember thinking a quick thought: This isnt good. And then I was on my back. The butchers bill was steep: both of his legs up to and including the thigh were blasted off, along with his penis, his scrotum, and an upside-down-U-shaped chunk of his abdominal wall. Only a handful of people know the full extent of his injuries.

Two years later, while he was learning to walk on prosthetic legs, his urologist at Walter Reed National Military Medical Center referred him to the reconstructive surgery group at Johns Hopkins.

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At the time, Hopkins was a leader in vascularized composite allotransplantation, more commonly called VCA surgery. Its used in face, hand, armand penis transplants, taking multiple types of tissue from a donor and hooking up blood vessels and nerves so they work for the recipient. In December 2012, Hopkins surgeons completed their first bilateral arm transplant, on an infantryman who had lost both his arms and legs to a roadside bomb. If anyone could help Ray, it was these surgeons.

At their first meeting, Redett talked about phalloplasty, which didnt excite Ray much. He resolved to go through with it, thinking it was the only choice. Yet Redett soon changed course, deciding that Ray was a better candidate for a transplant.

In fact, it was probably the only surgical fix given the extent of the damage. Van der Merwe calls Rays procedure the most complex to date, largely because of the scope of his injury. To repair it, Hopkins doctors didnt just transplant the penis itself. They also transplanted the donors scrotum and extensive amounts of tissue from the thigh and lower abdomen.

When I heard they wanted to do it, I felt this huge sigh of relief, says Ray.

For him, it was almost either you do this transplant, or you live the rest of your life with your defect, Redett says.

Jared Soares

Ray, who is now in his mid-30s, is a thin man of average height, with touches of gray in his beard and a wobbly gait, a result of the prosthetics he now calls his legs. He hasnt discussed his surgery since April 2018, when he gave a short interview to the New York Times. But this March, one year after his surgery, he agreed to talk to me so long as MIT Technology Review protected his identity. (His name has been changed in this article.) He did so, he says, because he wants other veterans to know about their options.

And many others are affected. A total of 1,367 American infantrymen sustained significant genital injuries in Iraq and Afghanistan between 2001 and 2013. Such hidden wounds of war represent a relatively new problem. Bombs from below used to be a death sentence, but better body armor and modern casualty care ensure that more wounded soldiers surviveand more of them with devastating genital-urinary trauma. In a report last year, military urologists wrote that groin injuries have increased to a level never before reported in the history of war.

The US Department of Defense recognized the problem as long ago as 2008, when it set up an institute to research various reconstructive transplants. Eventually, the TOUGH ProjectTrauma Outcomes and Urogenital Healthplaced a figure on it: among infantrymen with genital urinary injuries from Iraq and Afghanistan, 502 were injured so severely that a penis transplant might be their only recourse.

Quantifying the number of such injuries is easy. Outlining the psychological toll they take on guys in their 20s and 30s is much harder.

Even those closest to the trauma, like Timothy Tausch, have to use anecdotes to explain. Hes an Army lieutenant colonel and director of trauma and male reconstructive urology at Walter Reed. As soon as they wake up, theyre not asking about where their legs are, he says. Theyre asking where the testicles and the penis are. You cant put a number on how significantly this affects one of these wounded warriors lives.

Yet some experts wonder if the procedure is really necessary. Kidney and heart transplants save lives, but someone who lost a penis isnt going to die without a new one. Getting one may even be inviting a different set of psychological issues. (It bears mentioning that a poorly documented transplant attempt happened in 2006 in China, but the 44-year-old recipient apparently demanded reversal after his wife panicked, shocked at the idea he had someone elses penis.) In the months following Rays surgery, Hiten Patel, a chief resident at the Johns Hopkins Brady Urological Institute, wrote that a penis transplant lacks both life-saving and life-enhancing properties when compared to a readily available alternative in phalloplasty.

Others argue that for young men devastated by their wounds a transplant is, in fact, both life-saving and life-enhancing. Suicide risk among US veterans is already high: one study found that those deployed between 2001 and 2007 were 41% more likely to take their lives than civilians. Ray himself entertained thoughts of suicide after his injury. The idea gradually faded once he realized he could have gone to war and died; instead he was alive, on the first step of a long climb back.

Even though we do a pretty good job with phalloplasty reconstruction, its still a quantum leap to put on a real penis, says Curtis Cetrulo, one of the surgeons who operated on Thomas Manning in 2016. Phalloplasty recipients, for example, may regain some erotic sensation, but they must use a pump to achieve an erection or have intercourse.

Ray wouldnt say the transplant saved his life, exactly, but it has improved it.

This surgery was a way for me to overcome that little subconscious voice or whatever it was that would always keep me feeling different from everyone else, he says. It was one of those injuries that really stresses you out and you think, Why would I keep going? I guess I always just kept this real hope that theres an answer out there.

Several hours before the hospital contacted Ray, Richard Redett had received a phone call of his own. He had gotten it enough times before to know the words by heart: We may have a donor.

Usually such calls were dead ends: the potential transplant almost never met Redetts strict criteria. For Rays surgery to stand a chance, the donor had to be a young, healthy guy; the organ had to be a good color match and average in size; and, crucially, it had to be no more than two hours away, so that once it had been removed from the donors brain-dead but still living body, it could be brought to Johns Hopkins before it started decaying.

If you do an arm transplant, we know exactly how long that will hold up on ice. But nobody really knows that for a penis, he says.

I remember everything froze and I was upside down.

This particular call on that Saturday in March was more promising. There was a brain-dead patient nearby who was donating his organs, including his penis. Over a rapid string of conversations, Redett evaluated the patients medical history and determined when his team could get there. By the afternoon, Redett knew he had his donor.

Still, no doctor had ever worked with a graft as large as the one Ray required. To transplant a penis, you need the two dorsal arteries and the two dorsal veins from the donor. Fortunately, Rays two penis nerves were intact. But to transplant the abdominal wall and scrotum, even more veins are necessary. Fail to take those, and the new scrotum and abdominal tissue will die, along with much of the skin of the penis.

Over five years, Redett and his team had deciphered the topography of penis transplantation with cadavers and food coloring. It was basically a grand perfusion experiment: inject dye into the blood vessels of a dead man, and watch for blush on the skin to know which vessels are required as part of the transplant. We were injecting every blood vessel we could find down in the region with blue and red food color, he says. We just needed to know which vessels, and we needed to get very quick, very efficient, and very safe. We knew this had the potential to be a very long operation.

On the Sunday afternoon, his team boarded a chartered jet to meet their donor (the donors identity and the state hes from cant be disclosed). At 6 p.m., they entered the procurement room. Other doctors and medical staff, 25 in all, were there grabbing solid organs: lungs, heart, kidneys, liver. Its a bloody choreography, finding your place in an organ procurement. Redett and his team sliced into and isolated the lower abdominal wall, thigh tissue, scrotum, and penis, dissected out the requisite arteries and veins, and let the other doctors take what organs they needed before finishing.

Once they had removed and packed Rays graft, nothing else mattered except speed. Bodily tissue begins to break down the instant its deprived of blood. If enough toxins are released, the tissue can swell so much it asphyxiates. Its why you throw transplants on ice, as Redetts crew did for their Learjet flight back to Baltimoreit delays the breakdown process.

Its also why surgeons train, practice, and visualize their maneuvers. Redetts team had already run dry rehearsals of their procedure. In the operating room, they had set up the table where Ray would lie, figured out where the ice machine went, placed the optical microscope Redett would use, and even tested every power outlet to make sure they wouldnt short a circuit.

As the team ate snacks from their go-bags on the plane back to Hopkins, other surgeons wheeled Ray into the operating theater. By this time it was 11 p.m. on Sunday, almost 24 hours after he had arrived at the hospital. They prepared him by removing all the diseased tissue and exposing the blood vessels, nerves, urethra, and penile stump. At 2 a.m. Monday, Redett and his fellow surgeons took their placessome standing above Ray, the rest tending to the graft at another tableand steeled themselves. The gravity of his mission consumed Redetts thoughts.

We felt very confident we could do it, but we had never done it, he says. If youre not anxious for something like that, youre not thinking hard enough.

In the Johns Hopkins operating room, a surgical microscope with a craned neck like a brachiosaurus magnified the view by up to 20 times, enabling Redett to see the very tip of the needle-point instruments that hold the sutures for stitching together vessels barely two millimeters thick.

The threads are smaller than a human hair, he says. Unless youre under a scope, you cant really even see it.

They began by sewing Rays urethra onto the donors. Then came the arteries and veins that bring blood to the skin of the abdominal wall, scrotum, and penis shaft. Next they sutured Rays penile nerves, which were buried deep underneath his pelvic bone, to the nerves of the donor penis. Finally, Redetts team stitched together the skin.

You know how to do it, but until that last blood vessel is hooked up and you release the clamps and blood flows through itI mean, thats a huge sigh of relief, says Redett.

A kidney transplant usually takes three hours. The first penis transplant surgery in 2014 took nine. Redetts team needed an additional five hours to complete Rays transplant. In a surgery that long, doctors are allowed to take bathroom breaks, and even slug some coffee. Redett did neither.

Ray's first memory after he came out of the anesthesia was the heat. His room was warm to help keep his transplant at body temperature. It wasnt until two days later that Ray looked down and saw his new penis for the first time.

It was swollen and still had a lot of healing to do, he says. In the back of your mind, you know this is a transplant, and you wonder if its going to be too much for you to handle. Once I went through with the surgery, all of those concerns just went away.

The surgery wasnt just technically complex; it also required weighing various ethical questions. For example: if they were giving Ray a scrotum, should they give him testicles too? The answer was no: transferring sperm-generating tissue might have made it possible for Ray to have the donors genetic kids. (In the end, the donor had not given consent to use his sperm.)

Another matter was the prospect of lifelong immunosuppression. In penis transplant surgeries, its critical: Van der Merwe had to cut off half of the penis he transplanted in 2014 because the patient stopped taking his medication and rejection set in.

The world is not designed for a guy like me, says Ray.

The team came up with a novel answer to this problem. In a procedure spearheaded by Gerald Brandacher, scientific director of the reconstructive transplantation program at the Johns Hopkins School of Medicine, bone marrow and stem cells from the donors vertebral bones were isolated in the lab. Two weeks after his transplant, Ray was injected with a large amount of the donors bone marrow cells.

In organ transplants of any type, recipients are typically given a cocktail of immunosuppressant drugs every day. Ray, on the other hand, requires just one pill.

Its kind of like reeducating the immune system, says Brandacher. It allows us to minimize the need for immunosuppression but not completely stop it.

Minimizing the drugs needed after a transplant, in fact, may be what really got the US military interested in surgery like Rays. Immunosuppressants ensure that the body doesnt attack a new organ, but they also weaken the immune system and can lead to toxic complications like kidney failure. For a heart or lung, the trade-off is obvious: immune problems versus death. For a penis, the question is more muddled.

If we can get to a point where we have therapy that doesnt require that level of toxicity, the calculus changes completely, says Lloyd Rose, a former program manager for rehabilitative medicine research in the US Army. Then a transplant can become a surgery for anybody whos missing a hand or a foot or a face or a penisor anything.

If vets with transplants have to take fewer pills, it means fewer complications as they get older, and an easier life. It also saves the government money in the long term. The issue is so important to the military that the $12 million Congress appropriates each year for the Armed Forces Institute of Regenerative Medicine is now spent primarily on immunosuppressive researchnot on paying for things like penis transplants.

On a hot afternoon last April, a year after his surgery, I met Ray for the first time. He balanced his modest frame on his partmetal, part-polymer prosthetic legs, and in his left hand he carried a cane. Even with the support, he picked his way gingerly along the sidewalks until we made our way over to a public bench near a coffee shop.

When I got hurt, one thing I did realize is that the world is not designed for a guy like me, being blown up, he told me matter- of-factly. I knew then I would have to change myself to fit the world.

While he doesnt hide his prosthetics when we met, he wore gym shortshis unseen injury still causes him some consternation. Its not that he hasnt accepted his new penis. On the contrary, Ray doesnt seem to think about it as a donor organ at all. Its just that so few people know what happened to him, and hes not quite ready, and may never be ready, to identify himself.

It may not necessarily be that people are going to say bad things about it, he says. But its just one of those things. Its a private thing.

Jared Soares

Still, those around him recognized a change. A close friend of Rays, one of the few who know, says she noticed a little boost following the procedure. It was such a profound wound, there was a no-light-at-the-end-of-the-tunnel kind of feeling, she says. Now hes much more confident Its this feeling of being whole again.

In some ways, Ray is still figuring out how his transplanted organ will shape the contours of his life. Hes not dating at the moment, and knowing that he cant be a biological father, he wonders if that will deter women who may want to start a family.

In other ways, the surgery has made a huge difference to his daily emotional state. Hes more outgoing, less afraid to meet new people, and more fit, mentally and physically, piecing back together a life interrupted. Important questionssuch as whether hes able to pee standing up (he can), whether he gets erections (he does)already have answers.

He told me, which was the best news I could hear, that it feels normal, says Redett.

It took six months before the nerves of his transplanted penis started firing. Stitching nerves together isnt like splicing a wire; a nerve cells axons, the long threads along which impulses are sent from one cell to another, have to grow all the way out to the organ theyre supplying. Now, more than a year removed from surgery, those nerve signals have grown only stronger. Im still getting sensation back. Its pretty close, Ray says. This is not going to be a quick fix, but Ive seen improvement over time.

Where penis transplant surgery for wounded veterans goes is still up in the air. South Africas Van der Merwe, the originator of the transplant, says the VCA procedure itself is now proven; its future depends on other matters. Theres the problem of who pays, and of finding appropriate donors. And then theres the immunosuppression issue that the military is trying to solve.

The risks of immunosuppression in many peoples minds also outweigh the benefit of doing an arm, or a face, or a genital transplant, Redett says. We disagree, but that will slow down progress.

Ray barely blinked when I asked him some of these questions at our second meeting, in July. Dealing with immunosuppression, he says, is easy: he takes a pill and washes his hands frequently. Guys who need it and can handle it, he says, should get a transplant. He feels no ambivalence about that phone call, when doctors told him they were ready to sew on the donor penis for which he had waited five years.

I dont regret it, Ray says. It was one of the best decisions I ever made.

Andrew Zaleski, a writer based near Washington, DC, covers science, technology, and business.

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New research links a genetic anomaly and some forms of SIDS, or sudden infant death syndrome, which claims the lives of more than 3,000 infants a year.

The research, published in Nature Communications, focuses on mitochondrial tri-functional protein deficiency, a potentially fatal cardiac metabolic disorder caused by a genetic mutation in the gene HADHA.

Newborns with this genetic anomaly cant metabolize the lipids found in milk, and die suddenly of cardiac arrest when they are a couple months old. Lipids are a category of molecules that include fats, cholesterol, and fatty acids.

There are multiple causes for sudden infant death syndrome, says Hannele Ruohola-Baker, professor of biochemistry at the University of Washington School of Medicine, who is also associate director of the Medicine Institute for Stem Cell and Regenerative Medicine.

There are some causes which are environmental. But what were studying here is really a genetic cause of SIDS. In this particular case, it involves defect in the enzyme that breaks down fat.

Lead author Jason Miklas, who earned his PhD at the University of Washington and is now a postdoctoral fellow at Stanford University, says he first came up with the idea while researching heart disease and noticed a small research study that had examined children who couldnt process fats and who had cardiac disease that was not readily explained.

So he and Ruohola-Baker started looking into why heart cells, grown to mimic infant cells, died in the petri dish where they were growing.

If a child has a mutation, depending on the mutation the first few months of life can be very scary as the child may die suddenly, Miklas says. An autopsy wouldnt necessarily pick up why the child passed but we think it might be due to the infants heart stopping to beat.

Were no longer just trying to treat the symptoms of the disease, Miklas says. Were trying to find ways to treat the root problem. Its very gratifying to see that we can make real progress in the lab toward interventions that could one day make their way to the clinic.

In MTP deficiency, the heart cells of affected infants dont convert fats into nutrients properly, resulting in a build-up of unprocessed fatty material that can disrupt heart functions. More technically, the breakdown occurs when enzymes fail to complete a process known as fatty acid oxidation. It is possible to screen for the genetic markers of MTP deficiency; but effective treatments remain a ways off.

Ruohola-Baker says the latest laboratory discovery is a big step towards finding ways to overcome SIDS.

There is no cure for this, she says. But there is now hope, because weve found a new aspect of this disease that will innovate generations of novel small molecules and designed proteins, which might help these patients in the future.

One drug the group is focusing on is Elamipretide, used to stimulate hearts and organs that have oxygen deficiency, but barely considered for helping infant hearts, until now. In addition, prospective parents can undergo screening to see if there is a chance that they could have a child who might carry the mutation.

Ruohola-Baker has a personal interest in the research: one of her friends in Finland, her home country, had a baby who died of SIDS.

It was absolutely devastating for that couple, she says. Since then, Ive been very interested in the causes for sudden infant death syndrome. Its very exciting to think that our work may contribute to future treatments, and help for the heartbreak for the parents who find their children have these mutations.

The National Institutes of Health, the Academy of Finland, Finnish Foundation for Cardiovascular Research. Wellstone Muscular Dystrophy Cooperative Research Center, Natural Sciences and Engineering Research of Canada, an Alexander Graham Bell Graduate Scholarship, and the National Science Foundation funded the work.

Source: University of Washington

Original Study DOI: 10.1038/s41467-019-12482-1

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Some cases of SIDS may have this genetic cause - Futurity: Research News

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In 2006, Shinya Yamanaka shook stem cell research with his discovery that mature cells can be converted into stem cells, relieving a longstanding political-ethical blockage and throwing open medical research on everything from curbing eye degeneration to organ printing.

But that process still has pitfalls, including in risk and scalability, and some researchers are exploring another way first hinted at years ago: new technology to convert mature cells directly into other mature cells without the complex and time-consuming process of first making them into stem cells.

One of those companies, Mogrify, just raised $16 million in Series A financing to bring its overall funding to over $20 million since its February launch. Led by CEO Darrin Disley, the funding will help expand their new base in Cambridge to a 60-strong staff and push forward their direct-conversion approach to cell therapy through research and licensing. Investors include Parkwalk Advisors and Ahren Innovation Capital.

They list potential applications as treatments for musculoskeletal and auto-immune disorders, cancer immunotherapy, and therapies for ocular and respiratory diseases. For example, you could use it regenerate cartilage in arthritis patients.

If you could take a cell from one part of the body and turn it into any other cell at any other stage of development for another part of the body, you effectively have the Holy Grail of regenerative medicine, Disley told Labiotech.eu in April.

Mogrifys advantage over the Yamanaka method called induced pluripotent stem cells (iPS), is that in theory it can be more scalable and avoid the problems associated with iPS. These include instabilities arising from the induced immature state and an increased risk of cancer if any pluripotent cells remain in the body.

The concept behind Mogrify actually predates, by nearly 19 years, Yamanakas discovery, which fast won him the 2012 Nobel Prize in Medicine. A 2017 Nature study on transdifferentiation, as the process is called, of fibroblasts into cardiac tissue traced the idea to a 1987 findingthat a master gene regulator could convert mice fibroblasts into skeletal muscle.

The problem though, according to Mogrify, is that most current efforts rely on an exhausting guess-and-check process. With hundreds of cell types and an even greater number of transcription factors the program that recodes the cell finding the right factor for the right cell can be like a custodian with a jangling, unmarked key ring trying to get into a building with thousands of locks.

Mogrifys key tech is a computer model they say can predict the right combination. The scientists behind the platform published a 2016 study in Nature applying the model to 173 human cell types and 134 tissues.

Before Mogrify, Disley led the Cambridge-based gene-editing company Horizon Discovery.

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Cell therapy startup raises $16 million to fund its quest for the Holy Grail in regenerative medicine - Endpoints News

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Both Tenax Therapeutics Inc. (NASDAQ:TENX) and Neuralstem Inc. (NASDAQ:CUR) are each others competitor in the Biotechnology industry. Thus the contrast of their analyst recommendations, profitability, institutional ownership, risk, dividends, earnings and valuation.

Valuation and Earnings

Table 1 highlights Tenax Therapeutics Inc. and Neuralstem Inc.s gross revenue, earnings per share and valuation.

Profitability

Table 2 shows Tenax Therapeutics Inc. and Neuralstem Inc.s return on assets, net margins and return on equity.

Risk & Volatility

Tenax Therapeutics Inc.s 1.4 beta indicates that its volatility is 40.00% more volatile than that of S&P 500. Neuralstem Inc.s 94.00% more volatile than S&P 500 which is a result of the 1.94 beta.

Liquidity

Tenax Therapeutics Inc.s Current Ratio is 11 while its Quick Ratio is 11. On the competitive side is, Neuralstem Inc. which has a 3.8 Current Ratio and a 3.8 Quick Ratio. Tenax Therapeutics Inc. is better positioned to pay off short and long-term obligations compared to Neuralstem Inc.

Insider and Institutional Ownership

Institutional investors owned 22.2% of Tenax Therapeutics Inc. shares and 4.9% of Neuralstem Inc. shares. Tenax Therapeutics Inc.s share owned by insiders are 4.73%. On the other hand, insiders owned about 1% of Neuralstem Inc.s shares.

Performance

Here are the Weekly, Monthly, Quarterly, Half Yearly, Yearly and YTD Performance of both pretenders.

For the past year Tenax Therapeutics Inc. had bullish trend while Neuralstem Inc. had bearish trend.

Summary

Tenax Therapeutics Inc. beats Neuralstem Inc. on 5 of the 9 factors.

Tenax Therapeutics, Inc., a specialty pharmaceutical company, focused on the identification, development, and commercialization of a portfolio of products for the critical care market in the United States and Canada. It focuses on the development and commercialization of pharmaceutical products containing levosimendan, 2.5 mg/ml concentrate for solution for infusion/5ml vial for use in the reduction of morbidity and mortality in cardiac surgery patients at risk for developing Low Cardiac Output Syndrome. The company was formerly known as Oxygen Biotherapeutics, Inc. and changed its name to Tenax Therapeutics, Inc. in September 2014. Tenax Therapeutics, Inc. was founded in 1967 and is headquartered in Morrisville, North Carolina.

Neuralstem, Inc., a clinical stage biopharmaceutical company, focuses on the research and development of nervous system therapies based on its proprietary human neuronal stem cells and small molecule compounds. The companys stem cell based technology enables the isolation and expansion of human neural stem cells from various areas of the developing human brain and spinal cord enabling the generation of physiologically relevant human neurons of various types. It is developing products include NSI-189, a chemical entity, which is in Phase II clinical trial for the treatment of major depressive disorder, as well as is in preclinical programs for the MCAO stroke, type 1 and 2 diabetes related neuropathy, irradiation-induced cognition, long-term potentiation enhancement, and angelman syndrome. The company is also developing NSI-566, which has completed Phase II clinical trial for treating amyotrophic lateral sclerosis disease, as well as is in Phase I clinical trials for the treatment of chronic spinal cord injury and motor deficits due to ischemic stroke. Neuralstem, Inc. was founded in 1996 and is headquartered in Germantown, Maryland.

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Reviewing Tenax Therapeutics Inc. (TENX)'s and Neuralstem Inc. (NASDAQ:CUR)'s results - MS Wkly

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This is a contrast between VistaGen Therapeutics Inc. (NASDAQ:VTGN) and Gamida Cell Ltd. (NASDAQ:GMDA) based on their institutional ownership, profitability, risk, analyst recommendations, dividends, earnings and valuation. The two companies are Biotechnology and they also compete with each other.

Earnings and Valuation

Table 1 shows gross revenue, earnings per share (EPS) and valuation of the two companies.

Profitability

Table 2 provides us the return on equity, net margins and return on assets of both companies.

Liquidity

The Current Ratio of VistaGen Therapeutics Inc. is 4.9 while its Quick Ratio stands at 4.9. The Current Ratio of rival Gamida Cell Ltd. is 6.5 and its Quick Ratio is has 6.5. Gamida Cell Ltd. is better equipped to clear short and long-term obligations than VistaGen Therapeutics Inc.

Analyst Recommendations

The table shown features the ratings and recommendations for VistaGen Therapeutics Inc. and Gamida Cell Ltd.

VistaGen Therapeutics Inc. has a 1,975.47% upside potential and a consensus price target of $22. Meanwhile, Gamida Cell Ltd.s consensus price target is $16, while its potential upside is 261.17%. The data provided earlier shows that VistaGen Therapeutics Inc. appears more favorable than Gamida Cell Ltd., based on analyst view.

Insider & Institutional Ownership

Roughly 20.4% of VistaGen Therapeutics Inc. shares are owned by institutional investors while 13.1% of Gamida Cell Ltd. are owned by institutional investors. Insiders owned 0.2% of VistaGen Therapeutics Inc. shares. Comparatively, 65.61% are Gamida Cell Ltd.s share owned by insiders.

Performance

Here are the Weekly, Monthly, Quarterly, Half Yearly, Yearly and YTD Performance of both pretenders.

For the past year VistaGen Therapeutics Inc. was more bearish than Gamida Cell Ltd.

Summary

On 6 of the 11 factors VistaGen Therapeutics Inc. beats Gamida Cell Ltd.

VistaGen Therapeutics, Inc., a clinical-stage biopharmaceutical company, engages in developing and commercializing medicines for depression and other central nervous system (CNS) disorders. The company's lead product candidate is AV-101, which is in Phase II development stage, an adjunctive treatment used for major depressive disorder. It also focuses on potential commercial applications of its human pluripotent stem cell (hPSC) technology platform to discover, rescue, develop, and commercialize new chemical entities (NCEs) for CNS and other diseases; and regenerative medicine involving hPSC-derived blood, cartilage, heart, and liver cells. In addition, the company develops CardioSafe 3D, an in vitro cardiac bioassay system for predicting human heart toxicity of small molecule NCEs. VistaGen Therapeutics, Inc. has licensing, sublicensing, and collaboration agreements with BlueRock Therapeutics, LP; U.S. National Institutes of Health; Cato Research Ltd.; and University Health Network. The company was founded in 1998 and is headquartered in South San Francisco, California.

Gamida Cell Ltd., a clinical stage biopharmaceutical company, focuses on developing cell therapies to cure cancer, and rare and serious hematologic diseases in the United States, the European Union, and internationally. The company's lead product candidate is NiCord, a nicotinamide (NAM)-expanded cord blood cell therapy that is in Phase 3 clinical trials for use as a curative stem cell graft for patients in hematopoietic stem cell transplant. It is also developing NAM-NK, an innate immunotherapy of expanded natural killer cells, which is in Phase 1 clinical trials for the treatment of refractory non-Hodgkin lymphoma and multiple myeloma. The company was founded in 1998 and is headquartered in Jerusalem, Israel.

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Reviewing VistaGen Therapeutics Inc. (VTGN)'s and Gamida Cell Ltd. (NASDAQ:GMDA)'s results - MS Wkly

Cardiac Stem Cells Comments Off on Reviewing VistaGen Therapeutics Inc. (VTGN)’s and Gamida Cell Ltd. (NASDAQ:GMDA)’s results – MS Wkly | October 14th, 2019

DUBLIN--(BUSINESS WIRE)--The "Cell Harvesting Market" report has been added to ResearchAndMarkets.com's offering.

This report provides a summary of the cell harvesting market, including a market snapshot and profiles of key players in the market. It provides an exhaustive segmentation analysis of the market with in-depth information about each segment. The overview section of the report provides a description of market trends and market dynamics, including drivers, restraints and opportunities. it provides information about market developments and future trends that can be useful for organizations, including wholesalers and exporters. It provides market positionings of key players using yardsticks of revenue, product portfolio, and recent activities. It further includes strategies adopted by emerging market players with strategic recommendations for new market entrants. Readers will also find historical and current market sizes and a discussion of the market's future potential. The report will help market players and new entrants make informed decisions about the production and exports of goods and services.

The report includes:

Key Topics Covered:

Chapter 1 Introduction

Chapter 2 Summary and Highlights

Chapter 3 Market and Technology Background

Chapter 4 Market Breakdown by Type of Harvesting

Chapter 5 Market Breakdown by End User

Chapter 6 Market Breakdown by Application

Chapter 7 Market Breakdown by Component/Equipment

Chapter 8 Market Breakdown by Region

Chapter 9 New Developments and Patent Review

Chapter 10 Analysis of Market Opportunities

Chapter 11 Company Profiles

For more information about this report visit https://www.researchandmarkets.com/r/lahvpd

About ResearchAndMarkets.com

ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

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Cell Harvesting Market 2019 with Detailed Profiles of Key Companies Including General Electric, Perkinelmer and Thermo Fisher - ResearchAndMarkets.com...

Bone Marrow Stem Cells Comments Off on Cell Harvesting Market 2019 with Detailed Profiles of Key Companies Including General Electric, Perkinelmer and Thermo Fisher – ResearchAndMarkets.com… | October 14th, 2019

The members of the British royal family are always in the public eye. They cant blow their noses without people hearing about it. But with so much attention, comes even more scrutiny.

The royals are under pressure to be perfect at all times. Every move they make is photographed by the paparazzi and the public isunforgiving. Even after they gave birth, people expected both Meghan Markle and Kate Middleton to be back to their normal thin frames in a matter of days.

When celebrities are feeling the pressure to keep up appearances, many of them seek out plastic surgery procedures. Does the royal family do the same?

For many people, the Duchess of Cambridge is the epitome of style and grace. Youd be hardpressed to find a photo of Middleton looking less than perfect. Because of this, people have often wondered if the future Queen Consort has had any work done.

Earlier this year, Dr. Munir Somji of Dr. Medi Spa Clinic in London claimed that Middleton was one of his clients.

He shared before and after pictures of the Duchess on his Instagram.

Our Kate loves a bit of baby Botox, he wrote under the photos, which have since been deleted.

View this post on Instagram

The Duchess of Cambridge, Patron of the @Natural_History_Museum, visited the Angela Marmont Centre for UK Biodiversity to hear how it is championing and helping to protect UK wildlife. The Angela Marmont Centre (AMC) is a unique scientific hub located in the Natural History Museum, and is dedicated to the study of the UKs natural world. The AMC helps public visitors identify their finds, provides vital training in wildlife identification, and leads research and citizen science projects that are helping to transform our understanding of over 80,000 species of wildlife known to exist in the UK today. With populations of many UK species declining, and a significant proportion threatened with local extinction, the AMC brings together a host of individuals and organisations all dedicated to building understanding of the UKs wildlife, so that it can be better protected for future generations. AMC staff work with colleagues from across the Natural History Museum to carry out a range of scientific research to study how and why the UKs wildlife is changing. This ranges from applying ground-breaking scientific techniques, including analysis of environmental DNA through to detecting and identify the miniscule insects and microorganisms that play an important role in healthy environments. At the AMC today The Duchess viewed some of the Museums specimens and saw DNA sequencing live in action.

A post shared by Kensington Palace (@kensingtonroyal) on Oct 9, 2019 at 5:20am PDT

Note the reduction of fine lines on the forehead, he continued. But also note the depression of the medial (middle part) brow but elevation of the lateral tail of the brow.

So many people started believing the rumor that Kensington Palace gave a statement to the New York Post.

A spokesperson for the palace said that the post by Dr. Somji was categorically not true and in addition, The Royal Family never endorse commercial activity.

One royal who has been open about the procedures shes had done is the Duchess of York, Sarah Ferguson.

Ive had a lot of help to look like this at 60! she said in an interview withDaily Mail.

Ive started the laser treatment, but its not finished yet, she continued. The collagen needs to rebuild. I hope it will all be done by my birthday.

She also had this treatment before her daughter, Princess Eugenies, wedding last year. The procedure was done by Dr. Gabriela Mercik in a facility in London.

I dont like the frozen look, Ferguson said of why she chooses more natural procedures. Im so animated and I like to be myself. I dont like the thought of needles and am very glad if I look well and happy Im really happy to be open about what Ive had done.

Originally, the Duchess tried botox but has since switched to less invasive procedures, like her most recent treatment which refines the skins texture.

Shes also had mesotherapy, which is a non-surgical cosmetic treatment.

I need to repair the damage that was done on the beach when I was a child, she continued. Its why I had the mesotherapy, the vitamin cocktail to hydrate and boost the skin.

Not only does Ferguson get work done on her face, but she makes sure her feet are perfect as well!

I think my toes were ruined by all the riding I did when I was young, she said. They shaved the bone here. And implanted stem cells, 20 million of them taken from my midriff, into my feet to make new cartilage. It takes about six months to heal but now I can walk in heels!

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Which Plastic Surgery Procedures Have Members of the Royal Family Had Done? - Showbiz Cheat Sheet

Skin Stem Cells Comments Off on Which Plastic Surgery Procedures Have Members of the Royal Family Had Done? – Showbiz Cheat Sheet | October 14th, 2019

CAMBRIDGE, Mass.--(BUSINESS WIRE)--AVROBIO, Inc. (NASDAQ: AVRO) (the Company) today announced that the first patient has been dosed in the Companys AVR-RD-04 investigational gene therapy program for cystinosis, a devastating lysosomal storage disease, in an ongoing Phase 1/2 clinical trial sponsored by academic collaborators at the University of California San Diego. The gene therapy is derived from the patients own hematopoietic stem cells, which are genetically modified to produce functional cystinosin, a crucial protein that patients with cystinosis lack.

The trial will enroll up to six patients with cystinosis, a rare inherited disease caused by a defect in the gene that encodes for cystinosin. The cystinosin protein enables transport of the amino acid cystine out of lysosomes. When it is absent, cystine accumulates and crystalizes, causing progressive damage to the kidneys, liver, muscles, eyes and other organs and tissues. Cystinosis affects both children and adults; they face shortened life spans and often painful symptoms, including muscle wasting, difficulty breathing, blindness and kidney failure.

Cystinosis is a debilitating and progressive disease, and new treatment options are sorely needed. The current standard of care does not avert deterioration; at best, it can attenuate symptoms. Thats why gene therapy is particularly exciting: It has the potential to change the course of disease -- and the lives of patients -- by addressing the underlying cause of cystinosis, said Birgitte Volck, MD, PhD, President of Research and Development at AVROBIO. We believe we can engineer patients own stem cells so they sustainably produce the functional protein that is needed to prevent a toxic buildup of cystine and halt progression of the disease. We are so pleased that this investigational gene therapy is now in the clinic in collaboration with Dr. Stephanie Cherqui at UC San Diego.

The single-arm trial will enroll four adults and a potential follow-on cohort of two adults or adolescents at least 14 years of age who are currently being treated with cysteamine, the standard of care for cystinosis. If started at an early age and taken on a strict dosing schedule, cysteamine can delay kidney failure. However, the treatment regimen is highly burdensome, with side effects that can be severe and unpleasant, and many patients find it difficult to adhere to this treatment regimen. Even if compliance is high, cysteamine therapy cannot prevent kidney failure or avert other complications.

For people with cystinosis, there are no healthy days. They must take dozens of pills a day, around the clock, just to stay alive. It is a relentless disease and we urgently need new treatments, said Nancy J. Stack, President of the Cystinosis Research Foundation, which supported development of the gene therapy with more than $5.4 million in grants to Dr. Cherquis lab at UC San Diego. We believe that we are now an important step closer to the potential cure that our community has been working toward for many years.

The trials primary endpoints are safety and tolerability, assessed for up to two years after treatment, as well as efficacy, as assessed by cystine levels in white blood cells. Secondary endpoints to assess efficacy include changes in cystine levels in the blood, intestinal mucosa and skin and cystine crystal counts in the eye and skin. Efficacy will also be evaluated through clinical tests of kidney function, vision, muscle strength, pulmonary function and neurological and psychometric function, as well as through assessments of participants quality of life after treatment. The trial is funded by grants to UC San Diego from the California Institute for Regenerative Medicine (CIRM) as well as the Cystinosis Research Foundation.

This investigational gene therapy starts with the patients own stem cells, which are genetically modified so that their daughter cells can produce and deliver functional cystinosin to cells throughout the body. With this approach we aim to prevent the abnormal accumulation of cystine that causes so many devastating complications, said Stephanie Cherqui, PhD, an Associate Professor of Pediatrics at UC San Diego School of Medicine, and consultant to AVROBIO. We have been working toward this trial for years and we are grateful for all the support that brought us to this moment.

About AVR-RD-04

AVR-RD-04 is a lentiviral-based gene therapy designed to potentially halt the progression of cystinosis with a single dose of the patients own hematopoietic stem cells. The stem cells are genetically modified so they can produce functional cystinosin with the aim of substantially reducing levels of cystine in cells throughout the patients body. Before the infusion of the cells, patients undergo personalized conditioning with busulfan to enable the cells to permanently engraft. The Phase 1/2 clinical trial is being conducted under the name CTNS-RD-04 by AVROBIOs academic collaborators at the University of California, San Diego.

About Cystinosis

Cystinosis is a rare, inherited lysosomal storage disorder characterized by the accumulation of cystine in all the cells of the body, resulting in serious and potentially fatal damage to multiple organs and tissues and the shortening of patients life spans. The kidneys and eyes are especially vulnerable; more than 90% of untreated patients require a kidney transplant before age 20. An estimated 1 in 170,000 people are diagnosed with cystinosis.

About AVROBIO, Inc.

AVROBIO, Inc. is a leading, Phase 2 gene therapy company focused on the development of its investigational gene therapy, AVR-RD-01, in Fabry disease, as well as additional gene therapy programs in other lysosomal storage disorders including Gaucher disease, cystinosis and Pompe disease. The Companys plato platform includes a proprietary vector system, automated cell manufacturing solution and a personalized conditioning regimen deploying state-of-the-art precision dosing. AVROBIO is headquartered in Cambridge, MA and has offices in Toronto, ON. For additional information, visit http://www.avrobio.com.

Forward-Looking Statements

This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words such as aims, anticipates, believes, could, estimates, expects, forecasts, goal, intends, may, plans, possible, potential, seeks, will and variations of these words or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding the therapeutic potential of our product candidates, the design, commencement, enrollment and timing of ongoing or planned clinical trials, including the ongoing Phase 1/2 trial of the Companys AVR-RD-04 investigational gene therapy, the anticipated benefits of our gene therapy platform, the expected safety profile of our product candidates, timing and likelihood of success of our current or future product candidates, and the market opportunity for our product candidates. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in preclinical or early stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

Any forward-looking statements in this press release are based on AVROBIOs current expectations, estimates and projections about our industry as well as managements current beliefs and expectations of future events only as of today and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that any one or more of AVROBIOs product candidates will not be successfully developed or commercialized, the risk of cessation or delay of any ongoing or planned clinical trials of AVROBIO or our collaborators, the risk that AVROBIO may not realize the intended benefits of our gene therapy platform, including the features of our plato platform, the risk that our product candidates or procedures in connection with the administration thereof will not have the safety or efficacy profile that we anticipate, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical or clinical trials, will not be replicated or will not continue in ongoing or future studies or trials involving AVROBIOs product candidates, the risk that we will be unable to obtain and maintain regulatory approval for our product candidates, the risk that the size and growth potential of the market for our product candidates will not materialize as expected, risks associated with our dependence on third-party suppliers and manufacturers, risks regarding the accuracy of our estimates of expenses and future revenue, risks relating to our capital requirements and needs for additional financing, and risks relating to our ability to obtain and maintain intellectual property protection for our product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause AVROBIOs actual results to differ materially and adversely from those contained in the forward-looking statements, see the section entitled Risk Factors in AVROBIOs most recent Quarterly Report on Form 10-Q, as well as discussions of potential risks, uncertainties and other important factors in AVROBIOs subsequent filings with the Securities and Exchange Commission. AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

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AVROBIO Announces First Patient Dosed in Phase 1/2 Trial of Gene Therapy for Cystinosis - Business Wire

Skin Stem Cells Comments Off on AVROBIO Announces First Patient Dosed in Phase 1/2 Trial of Gene Therapy for Cystinosis – Business Wire | October 13th, 2019

Jennifer Adair, a senior scientist at Fred Hutch, speaks at the 2019 GeekWire Summit. (GeekWire Photo / Kevin Lisota)

Genetically editing cells using CRISPR could be the answer to curing genetic disorders such as sickle cell anemia. But in order for the technology to be available for people in countries like Nigeria where around a quarter of the population carries the sickle cell trait the technology will need to become substantially cheaper and less invasive.

Thats where gold nanoparticles come in.

Scientists at the Fred Hutchinson Cancer Research Center are devising an approach that vastly simplifies how CRISPR is applied. Their goal is to create a safe process for gene editing that takes place entirely within the body of a patient.

In order to edit human stem cells using CRISPR today, scientists have to follow a process that involves removing the cells from a patients bone marrow, electrocuting those cells, and modifying them with engineered virus particles.

The process gets even more invasive from there. We actually have to treat these patients with chemotherapy, radiation or other agents in order for these cells that were genetically manipulated to be taken up, Jennifer Adair, a senior scientist at Fred Hutch, said during a talk at the 2019 GeekWire Summit.

The researchers think theyve figured out the first step, which is delivering CRISPR to blood stem cells inside the body. Theyre doing that using gold nanoparticles that are about a billionth the size of a grain of table salt and able to smuggle in RNA, DNA and a protein.

Weve been able to show that not only can we make these, but they passively deliver all of those components to blood stem cells, then we do get genetic editing. And weve been able to go on to show that we can correct the sickle cell defect using this approach, said Adair.

The nanoparticles are big enough to carry the CRISPR payload but small enough to infiltrate cell membranes. Gold is a useful medium since it isnt harmful to humans.

The Fred Hutch team published their work with gold nanoparticles earlier this year in the journal Nature Materials. The system safely edited 10 to 20 percent of the target cells, which the researchers hope will increase as the method is refined.

In an ideal world, clinicians would be able to deliver gene therapy through a syringe, a process that might be accomplished in a single office visit. Adair previously published research on agene therapy in a box concept, a table-top device that could provide gene therapy treatments without the need for expensive medical infrastructure.

We need to develop technologies that make gene editing simpler, more affordable and more accessible to patients around the world, Adair said.

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Fred Hutch scientist on how gold nanoparticles could bring CRISPR to the developing world - GeekWire

Bone Marrow Stem Cells Comments Off on Fred Hutch scientist on how gold nanoparticles could bring CRISPR to the developing world – GeekWire | October 13th, 2019

Gaucher disease can predispose patients to rare types of blood cancer, and doctors should be vigilant for the development of these malignancies, a case report suggests.

The study, A case of bony lytic lesions in a patient with Gaucher disease, was published in the journal Clinical Case Reports.

The most common symptoms of Gaucher disease are reduced platelet count, enlarged liver and spleen, and lesions in the bones. This disease also has been associated with an increased risk ofblood disorders.

Researchers in Canada reported the case of a 57-year-old man who developed a rare type of blood cancer cell probably related to Gaucher disease, as he had Gaucher cells cells that accumulate abnormal amounts of a fat molecule (glucocerebroside) characteristic of the condition in his bone marrow.

The man had a scalp lesion that did not heal and progressively increased in size. Doctors performed a biopsy and discovered it was caused by a plasmacytoma, a rare form of blood cancer in which myeloma cells form a tumor in the bones or soft tissues.

At the time of biopsy, the patient had mild anemia, low levels of platelets, and a family of blood proteins called gamma globulins characteristic of myeloma. His kidney function and calcium levels were normal.

Further tests showed that the man had several bony lytic lesions spots of bone damage caused by cancerous myeloma cells and abone marrow biopsy showed infiltration of both plasma cells and Gaucher cells. That led to a diagnosis of plasma cell myeloma probably associated with Gaucher disease.

Gaucher cells infiltrating the bone marrow may mask the extent of abnormal plasma cell infiltrates, and immunohistochemical staining [a method that identifies abnormal cells in biopsies] can be invaluable in identifying the true burden of plasma cells for appropriate classification of suspected plasma cell neoplasia, the researchers said.

The investigators also noted that the man had a history of abnormal spleen size and reduced blood cell count. A bone marrow biopsy performed years earlier showed the presence of possible Gaucher cells.

Gaucher disease should be considered in the differential diagnosis of unexplained hepatomegaly [abnormal liver size], splenomegaly [abnormal spleen size], or cytopenias [reduced blood cell count], the investigators said. They added that further research of the previous symptoms might have allowed diagnosing Gaucher disease before the cancer appeared.

The patient received a combination of chemotherapy, cyclophosphamide, and Velcade (bortezomib), followed by high doses of melphalan and autologous stem cell transplantation. He tolerated the transplant well and was discharged with the recommendation of long-term follow-up.

Alejandra has a PhD in Genetics from So Paulo State University (UNESP) and is currently working as a scientific writer, editor, and translator. As a writer for BioNews, she is fulfilling her passion for making scientific data easily available and understandable to the general public. Aside from her work with BioNews, she also works as a language editor for non-English speaking authors and is an author of science books for kids.

Total Posts: 20

Ins Martins holds a BSc in Cell and Molecular Biology from Universidade Nova de Lisboa and is currently finishing her PhD in Biomedical Sciences at Universidade de Lisboa. Her work has been focused on blood vessels and their role in both hematopoiesis and cancer development.

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Gaucher Might Be LInked to Rare Types of Blood Cancer, Report Suggests - Gaucher Disease News

Bone Marrow Stem Cells Comments Off on Gaucher Might Be LInked to Rare Types of Blood Cancer, Report Suggests – Gaucher Disease News | October 13th, 2019

Blood, of course, plays a crucial role in keeping our bodies alive and functioning.

Red blood cells carry oxygen from our lungs to our muscles. White blood cells are the first responders of our immune systems, detecting infections and foreign agents and triggering the immune response needed to deal with the problem. Plasma, the liquid part of blood, transports not only the cells but also proteins, such as antibodies, and hormones, such as insulin, to every part of the body. It is a beautifully complex system that is the key to our bodies functioning correctly.

Parasites, viruses and bacteria all use the circulatory system to spread around the body. When cancer metastasises and spreads to other parts of the body, it is through tumour cells circulating within the bloodstream. There are also myriad blood cancers, such as leukaemias and lymphomas, and blood disorders, such as sickle cell anaemia, not to mention autoimmune diseases like diabetes or lupus. Sepsis, also known as blood poisoning, is a deadly overreaction to an infection which also has its roots in blood.

Magnetic blood filtration is a tool which enables the physical removal of specific substances from the bloodstream

Most of these diseases are treated with drugs or chemotherapies, some with great success, others, much less. There are also various methods of physically extracting different components from the blood by circulating a patients blood outside of the body through whats known as an extracorporeal circuit. Dialysis, for example, removes excess toxins from the blood, acting as a substitute for kidneys when they fail. Plasmapheresis and leukapheresis are methods of removing harmful antibodies from the plasma or white blood cells from the blood. Similar techniques are also used to harvest stem cells from the blood, which can then be used in cancer therapies (known as stem cell transplantation), if the donor and patient are a match.?

Magnetic blood filtration

Despite these methods, millions of people still die every year from blood-borne diseases. At MediSieve a small, London-based start-up company we are using nanotechnology to develop a new technology which we think can transform our ability to tackle these conditions. Magnetic blood filtration (MBF) is a tool which enables the physical removal of specific substances from the bloodstream. It is similar to other extracorporeal procedures like dialysis, but instead of removing every component of a particular size or weight, MBF removes highly specific targets to address the specific medical issue, thereby removing only the substance that doctors want to remove. Alongside this high specificity, various targets, both big and small, can be removed simultaneously, raising the prospect of removing for example specific cells and harmful antibodies in a single procedure.

To achieve this, we use magnetic nanoparticles coated with binding moieties, such as antibodies, which bind specifically to the desired targets in the blood. These particles are infused into the blood within the extracorporeal circuit, binding to their targets. The blood then flows through a magnetic filter which captures the magnetic particles and the targets bound to them, while the rest of the blood flows back into the patient. Several different particles can be used in the same procedure in order to capture different components. Using this method, anything for which there is a specific antibody or other binding moiety can theoretically be removed directly from the bloodstream. I think that in the long-term the technology could be used to remove specific cells, antibodies, bacteria, viruses, toxins, drug molecules and inflammatory cytokines, the drivers of immune conditions such as sepsis.

The MediSieve Filter is a disposable, single-use device in which the magnetic particles and their targets are captured. It is inserted into the MediSieve Magnet, a reusable medical device which activates the filter. Both can be incorporated into a variety of existing extracorporeal systems and integrate with standard blood pumps and tubing sets.

We are currently developing treatments for malaria, sepsis and leukaemia. The Filter and Magnet have completed pre-clinical testing and are now ready for clinical trials, which we hope to start shortly. Magnetic particles for various clinical targets are currently being developed and validated in the laboratory, with promising results. Animal trials for these are expected to start in 2020.

Malaria

Our potential treatment for malaria is the closest to market because malaria infected cells, uniquely, have naturally occurring magnetic properties is it therefore possible to remove them from the blood using the MediSieve Filter without the infusion of any magnetic particles. The magnetic properties arise from a core aspect of the malaria parasites lifecycle. After infecting a red blood cell, the parasite consumes the protein part of haemoglobin, leaving behind an iron-based waste-product known as haemozoin, which is stored inside the cell. Haemozoin is paramagnetic, thereby giving infected cells their unique magnetic properties.

MBF could be used in highly severe malaria cases in which the patient is hospitalised and at high risk of death. Currently, these patients receive intravenous drugs such as artesunate which can achieve parasite clearance in 36-48 hours; parasite clearance rate is the key indicator of patient recovery, and it can take up to eight doses of IV drugs to achieve complete clearance. Mortality in these cases can be as high as 20%.?

Using MBF alongside the first dose of IV drugs could drastically accelerate parasite clearance rate. We claim that, depending on the patient size and initial level of infection, this approach can remove over 90% of red blood cells containing haemozoin in just two hours. Because they have higher quantities of haemozoin, MBF is better at removing later stage infected cells, whereas drugs are much more effective against earlier stage cells, so they should be complimentary.

MBF has the additional benefit of removing free circulating haemozoin, also known as the malaria toxin, which should also improve the treatment for the patient since drugs can cause the large-scale release of haemozoin as infected cells die.

According to the WHO, in 2017 there were 219 million cases of malaria and 435,000 deaths, mostly children. While overall malaria cases and deaths have been trending downwards in recent years, the number of hospitalised patients is increasing as healthcare infrastructure improves in malaria endemic countries and more patients gain access to hospitals. In the future, MBF could be adapted for use in mobile clinics to reach harder to access areas.

While ourinitial target is severe malaria patients, I also believe MBF could be a valuable tool in the fight against drug-resistant malaria strains, which have been emerging in SE Asia and are causing great concern if drug resistance spreads to Africa, the effect could be catastrophic. It can also be used to treat patients for whom drugs cannot be used, such as pregnant women.

Sepsis

Sepsis is one of the leading causes of death in the developed world with more than 1.9M cases in Europe and the US and published mortality rates of 29% - 50%. Sepsis is a complex syndrome in which bacteria or other pathogens create a dysregulated immune response which can escalate to organ failure and death. The immune response creates an overproduction of pro-inflammatory cytokines, while cell damage over time creates damage-associated molecular patterns (DAMPs) that sustain the syndrome. ?

Our approach to sepsis, which we call SepSieve, uses a cocktail of different particles to remove a number of targets from a patients bloodstream: specific pro-inflammatory cytokines (IL-1, IL-6 and IL-18), DAMPs (HMGB-1), endotoxins (LPS), and gram-negative bacteria. This multi-modal approach tackles the disease from two key angles: Removing the pathogens and endotoxins that trigger the immune response and reducing magnitude of the immune response and preventing the cascade towards septic shock.

Like in malaria, SepSieve would be used alongside existing frontline treatments, specifically antibiotics. While antibiotics are critical for treatment of sepsis, the bacterial cell death they cause releases LPS which accelerates the dysregulated immune response MBF could remove the LPS to prevent the condition from worsening. The main benefit of MBF in sepsis is therefore not so-much the removal of bacteria itself (which is tackled by antibiotics and in any case is not present exclusively in the bloodstream), but rather the removal of all the other components driving the disease.

Gram-negative bacteria such as E. coli account for approximately 50% of sepsis patients, but thanks to the removal of other substances, particularly HMGB-1 and the inflammatory cytokines, I think the combined approach could benefit all sepsis patients. Since magnetic filtration is a purely physical method, it can also target and remove pathogens which are resistant to antibiotics, which again are a huge concern with increasing occurrences of resistant infections in hospitals.

Like in malaria, wwe plan to apply sepsis treatment to hospitalised patients and specifically those in Intensive Care Units. These are the most severe cases and those who stand to benefit the most from the treatment. The idea is to intervene early to prevent the sepsis cascade, in which the disease escalates eventually causing organ failure and death.

In fact, we managed to secure grants worth a total of 1.56M from Innovate UK, the UKs government grant funding body, and the UK National Institute of Health Research to develop and validate our sepsis particles. Currently being tested in human blood models in the companys laboratories, we plan to start animal trials in 2020 which, if successful, will be followed by clinical trials in 2021.

Leukaemia

One of the advantages of the particles we develop to remove pro-inflammatory cytokines for sepsis is that they can also be used in other diseases. This includes auto-immune diseases and cytokine storms such as cytokine release syndrome (CRS), a common side-effect of newer leukaemia treatments known as CAR T-cell therapies.?

In CAR-T therapies, T-cells, a type of white blood cell, are modified to attack cancer cells in a patients bone marrow. Taken either directly from the patient or from a matching donor, the modified cells are infused into the patient in order to directly attack the cancer. Results of clinical trials have been mixed, but these cell therapies are seen as a huge leap forward for leukaemia treatment.?

The problem is that the infused T-cells trigger massive immune reactions within the patient. Indeed, that is the intention the immune reaction is intended to kill the cancer cells but it can easily escalate into the condition called CRS. The result is similar to sepsis an immune over-reaction which attacks the patient and can be fatal. Immune mediators can be used to calm this reaction, but they then prevent the infused CAR-T cells from having their effect, eliminating the therapeutic benefit of the treatment.

Our proposal is to use MBF in CRS patients to remove cytokines from the bloodstream. This should calm the immune reaction, alleviating patient suffering and eliminating the risk of death. But since MBF only removes cytokines from the bloodstream, it shouldnt affect the immune effect of the CAR-T cells in the bone marrow, so the therapeutic benefit should be maintained. In addition, MBF can be stopped at will, so it can be used to control the immune response by maintaining the correct balance of cytokines this is of course not possible with immune mediators which are infused into the patient.

A further benefit that MBF can provide in leukaemia patients is the removal of leukaemia cells from the bloodstream leukaemia patients commonly have very high white blood cell counts due to circulating leukaemia cells. These cause a number of issues such as a reduction in immune function, making patients more vulnerable to infection. They can also prevent certain chemotherapies from working effectively, since they block the drug from targeting cancer cells in the bone marrow. High white blood cell counts also increase the risk of side-effects during treatment, since the sudden death of such a large numbers of cells causes debris to circulate in the blood, putting strain on the body and causing immune reactions like CRS; this is known as Tumour Lysis Syndrome. ?

We are currently focussing development on their sepsis particles, but plan to trial their cytokine particles in CRS at the same time as they are trialled in sepsis, since the pre-clinical validation for each disease is the same. The particles to remove white blood cells, however, are at an earlier stage and will be developed further down the line.

Our ambitions for MBF are certainly large. In the long-term we want to revolutionise the way in which blood-borne diseases are treated. Going far beyond malaria, sepsis and leukaemia, we want to develop treatments for all blood-borne diseases if its in the blood, and doctors want it out, we want to be able to take it out.

My vision is that hospitals all around the world will have Magnetic Blood Filtration Units which will address a huge variety of patients. Only time will tell if this can be achieved, or even if our technology will work at all after all, there have, as of yet, been no clinical trials.

However, the ability to remove specific substances from blood would clearly be of benefit to huge numbers of patients. It is something that we cannot do today, but we certainly should want to be able to do tomorrow. Whether it is MediSieve who gets us there or not remains to be seen.

Author:

Dr George Frodsham is CEO and founder of MediSieve

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It's in the blood - Lab News

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