Archive for the ‘Bone Marrow Stem Cells’ Category

PEORIA, Ill. November is National Bone Marrow Awareness Month.

The Greater Peoria Family YMCA is hoping you take the time to see if youre a match and it comes with a local tie.

Marsha Krone has been a teacher in the Peoria area for over 30 years.

Despite being diagnosed with a rare bone marrow cancer in 2016, shes yet to find a match.

You never know when youre going to be the link to saving someones life and its very simple. You come out, fill out a little paperwork online. You swab the inside of your cheek. You put it back on the cart and its sent back.

You can join the donor registry for bone marrow & blood stem cells this Wednesday, Nov. 6 from 3 p.m. 7 p.m.

It would be awesome to find a match for me, but if I dont, there are other people that their lives can be saved through that, said Krone . Their joy would certainly not be pain for me. I would be happy for them to find their cure and what a gift to be able to give someone.

The YMCAs is located 7000 North Fleming Lane, Peoria, IL 61614. The registry will be held in the upstairs lounge.

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You can be the match, help local people in need Wednesday - CIProud.com

The proposal was nothing fancy. Shawn Russell arrived at the pub, placed a pint on the table in front of Sarah Hodgetts and asked her to marry him.

It was out of the blue, with no ring, nothing. And I thought: Yeah, thats a really good idea, we should just get married, says Sarah. Id been in love with him for ages.

Their story began seven years earlier, in 2009, also in a pub in north London. He was sitting in his flat cap, good Yorkshireman that he was, and was reading the sports pages. I had just got off the Tube after work. From my perspective, it was love at first sight, says Sarah.

But dating wasnt easy. Shawn, a boarding school-educated boy from an Army family, who had lost his mother to leukaemia when he was two, worked as a picture editor at The Telegraph. Although Sarah was down the road in Westminster, where she worked as a civil servant (and still does), their hours were long and their schedules largely incompatible. They managed just six months initially.

It was a disaster trying to date, so we ended up with a firework display of an argument and decided there was no way we could, says Sarah over coffee near her office.

Their split didnt last, however; they had far too much in common. Besides his ridiculous sense of humour, Shawn was so into news and politics, and I worked in politics, so we couldnt not communicate. We realised we were incredibly good friends, says Sarah. Plus, she adds with a smile, he was a very attractive, 6ft 4in blue-eyed man who I was completely smitten with.

Over time, they got back together in a non-committal way, sharing weekends when their busy lives permitted. It was during this period, three-and-a-half years ago, that Shawn, then 44, proposed. The following week, he moved in with Sarah, then 41, and her son Eddy, 10, from a previous relationship, in Kings Cross, and, like all newly engaged couples, they started making plans for their future. A pair of recovering workaholics, they were going to transform their lives.

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I lost my fianc to leukaemia, but in my dreams hes just working the night shift - Telegraph.co.uk

SAN FRANCISCO--(BUSINESS WIRE)--

Imago BioSciences, Inc., a clinical-stage biotechnology company developing innovative treatments for malignant and life-threatening diseases of the bone marrow, today announced the appointment of James D. Watson as Chief Business Officer.

James has a long history of success in biotech financing, business development, and commercial planning. That experience will guide the strategic direction and growth of Imago, said Hugh Young Rienhoff, Jr., M.D., Chief Executive Officer of Imago BioSciences. His experience in corporate development and financings will help ensure the advancement of bomedemstat (IMG-7289) through clinical development in myelofibrosis and other indications.

Mr. Watson most recently served as Chief Business Officer at Sigilon Therapeutics where he closed a $473 million strategic partnership with Eli Lilly for a treatment for Type 1 diabetes. Prior to Sigilon, Mr. Watson was Chief Business Officer for Alvine Pharmaceuticals and led strategy, corporate development, new product planning, and finance during which he closed a transaction giving AbbVie the right to acquire Alvine for $345 million. Previously, Mr. Watson was CEO of a San Francisco-based, boutique investment bank focused on mergers, acquisitions, partnering, and raising capital for life science companies.

I am excited to join Imago BioSciences at this important stage of growth, helping the company realize its full potential. Bomedemstat has great promise for the day-to-day management of myelofibrosis and it offers the additional possibility of altering the course of this disease. Furthermore, its broader myeloproliferative neoplasm platform and expertise in life-threatening diseases of the bone marrow represent an opportunity to address areas of high unmet clinical need and to build a valuable company. said Mr. Watson.

About Bomedemstat (IMG-7289)

Bomedemstat is a small molecule discovered by Imago BioSciences that inhibits lysine-specific demethylase 1 (LSD1 or KDM1A), an enzyme essential for production and normal function of megakaryocytes and for self-renewal of malignant hematopoietic stem or progenitor cells. Megakaryocytes are the primary producer of growth factors and cytokines that drive myelofibrosis pathogenesis.

In non-clinical studies, bomedemstat demonstrated robust in vivo efficacy as a single agent and in combination with other therapeutics across a range of myeloid malignancy models including the myeloproliferative neoplasms encompassing myelofibrosis, essential thrombocythemia and polycythemia vera. The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to bomedemstat for the treatment of myelofibrosis which is currently being studied in an international Phase 2b study. Additional clinical studies in hematologic disorders will begin in 2020.

About Imago BioSciences

Imago BioSciences is a clinical-stage, private therapeutics company focused on malignant and life-threatening diseases of the bone marrow. The initial clinical focus is on myeloproliferative neoplasm (MPN) disorders including myelofibrosis, essential thrombocythemia and polycythemia vera. Investors in Imago include a fund managed by Blackstone Life Sciences, Frazier Healthcare Partners, Omega Funds, Amgen Ventures, MRL Ventures Fund, HighLight Capital, Pharmaron, Greenspring Associates and Xeraya Capital.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191104005265/en/

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Imago BioSciences Further Expands Executive Team with Appointment of James D. Watson as Chief Business Officer - Yahoo Finance

Aplastic anemia is a medical condition that damages stem cells in a person's bone marrow. These cells are responsible for making red blood cells, white blood cells, and platelets, which are vital to human health.

Doctors believe various conditions can cause aplastic anemia, while the disease itself ranges in severity from mild to life threatening.

Medical advancements mean that aplastic anemia is more treatable than ever. In this article, learn more about this rare medical disorder.

When a person has aplastic anemia, their bone marrow does not create the blood cells it needs. This causes them to feel ill and increases their risk of getting infections.

Doctors also call aplastic anemia bone marrow failure.

Doctors do not know exactly how many people in the United States have aplastic anemia.

According to the National Organization for Rare Disorders (NORD), doctors diagnose approximately 500 to 1,000 cases every year. It is most common in older children, teenagers, and young adults.

Researchers believe that most cases of aplastic anemia are due to the immune system attacking healthy bone marrow cells, according to NORD.

Doctors have also identified some of the possible causes of this immune system response, including:

However, doctors usually cannot pinpoint the underlying cause in most aplastic anemia cases.

When the cause is unknown, doctors refer to the condition as idiopathic aplastic anemia.

Symptoms of aplastic anemia include:

These symptoms may be severe. Some people may have heart-related symptoms, such as chest pain.

A doctor will start by asking about a person's symptoms and their medical history.

They will usually use a blood test known as a complete blood count (CBC) to evaluate a person's red blood cells, white blood cells, and platelets. If all three of these components are low, a person has pancytopenia.

A doctor may also recommend taking a sample of bone marrow, which comes from a person's pelvis or hip.

A laboratory technician will examine the bone marrow. If a person has aplastic anemia, the bone marrow will not have typical stem cells.

Aplastic anemia can also have similar symptoms as other medical conditions, such as myelodysplastic syndrome and paroxysmal nocturnal hemoglobinuria. A doctor will want to rule out these conditions.

Sometimes, a person with other medical conditions can develop aplastic anemia. These conditions include:

If a person has these conditions, a doctor will recognize that they are more likely to get aplastic anemia.

Doctors usually have two goals when treating aplastic anemia. The first is to reduce the person's symptoms, and the second is to stimulate the bone marrow to create new blood cells.

People with aplastic anemia can receive blood and platelet transfusions to correct low blood counts.

A doctor may also prescribe antibiotics as a person needs white blood cells to fight infections. Ideally, these drugs will prevent infections until a person can build more new white blood cells.

Doctors usually recommend a bone marrow transplant to stimulate new cell growth in the long term.

For this, a doctor may first prescribe chemotherapy medications to kill off abnormal bone marrow cells that are affecting a person's overall bone marrow function.

Next, a doctor performs a bone marrow transplant by injecting the bone marrow into a patient's body.

Ideally, the individual will receive bone marrow from a close family member. However, even a sibling donor is only a match in 2030% of cases.

People can also receive bone marrow from someone who is not related to them if doctors can find a compatible donor.

Some people cannot tolerate bone marrow transplants, especially older adults, and those having difficulty recovering from chemotherapy. Others may not be able to find a donor that matches their bone marrow. In these instances, a doctor can prescribe immunosuppressive therapy.

Immunosuppressive medicines suppress the immune system, which ideally stops it from attacking healthy bone marrow cells. Examples of these medications include antithymocyte globulin (ATG) and cyclosporine.

According to NORD, an estimated one-third of people with aplastic anemia do not respond to immunosuppressive drugs.

If this is the case, doctors may consider other treatments, such as hematopoietic stem cell transplantation and a medication called eltrombopag (Promacta).

Those with aplastic anemia may face complications due to their disease as well as their treatment.

Sometimes, a person's body rejects a bone marrow transplant. Doctors call this graft-versus-host disease or GVHD.

GVHD can make a person feel extremely ill and can cause symptoms that include:

According to 2015 research, about 15% of aplastic anemia patients who receive immunosuppressive therapy will develop myelodysplastic syndromes or acute myeloid leukemia.

These conditions can develop years after a person's initial diagnosis.

Some people do not respond to aplastic anemia treatments. When this is the case, they are more vulnerable to infections that can be life threatening.

The outlook for a person with aplastic anemia depends on many factors, including:

A doctor will discuss a person's treatment outlook when considering the various therapies.

Aplastic anemia damages stem cells in a person's bone marrow. The bone marrow makes red blood cells, white blood cells, and platelets, which are all essential for the body.

A person with aplastic anemia may experience severe anemia symptoms. Treatment may include chemotherapy, stem cell transplants, and immunotherapy.

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What is aplastic anemia? Symptoms, causes, and treatment - Medical News Today

JenniferDelgado grew up in St. Louis, Missouri. She attended Webster University, whereshe received her Bachelor of Arts in Media Communications. She then went to MississippiState University, where she received a Bachelor of Science in Geosciences witha concentration in Broadcast Meteorology.

In 2006,Jennifer Delgado worked as a morning and noon meteorologist for WTVR-TV inRichmond, Virginia. Then in 2008, she began working at CNN International inAtlanta, Georgia, as their primary meteorologist, as well as a fill-inmeteorologist on all CNN networks. In 2010, she won a Peabody Award for CNNscoverage on the Deepwater Horizon oil spill in the Gulf of Mexico.

In 2013,Delgado was hired as a co-host of AMHQ (Americas Morning Headquarters) at TheWeather Channel. She anchored continuous coverage of breaking news and weatherevents, including live interviews with state and local officials, experts andresidents. She was also their fill-in co-host of Wake-Up with Al.

JenniferDelgado began freelancing as a meteorologist/anchor for WXIA-TV in 2017. Shepresented weathercasts every six minutes during a two-hour morning newscast andproduced weathercasts for radio, web, and the 24-hour weather channel.

Two yearsago, Jennifer Delgado was diagnosed with blood cancer. She underwent treatmentand received a bone marrow/stem cell transplant. Since the transplant, she hasbeen receiving treatment at the Emory Winship Cancer Institute and advocatingfor cancer awareness and more bone marrow donors.

No one is ever prepared tohear the words, you have cancer. It literally blew up my world. I had to stopworking because beating cancer became my full-time job. I knew something waswrong for months based on my symptoms. I was tiredall the time, my bones were aching, had migraines, vertigo andconfusion. Dealing with any illness is stressful, especially if you arent ableto work. Some people say cancer changed their life for the better; however, Idont want to credit cancer for anything positive. It was a wake-up call. Lifeis short, and you have to enjoy every moment.

I immediately went into adeep depression. I hid and only shared the news with my close friends andfamily. I was trying to hide the awful chemo port in my chest and made excuses for my appearanceand fatigue. It was very stressful. I think anyone dealing with a seriousmedical condition should reach out to people going through the same battle. I got some amazing tips from fellow blood cancersurvivors on Instagram and Facebook support groups. I have formed many closebonds and when I am feeling down they completely understand. Cancer patients caneasily go through their savings in a short amount of time. I was lucky to haveamazing health insurance but not everyone is that fortunate. There is a lot of grant money out there forpeople struggling financially. The Leukemia & Lymphoma Society is anamazing organization and helps patients with everything from financial help,information on clinical trials etc.

If you are strong enough, Isay its important to be your own health advocate. You know your body best. Ialso suggest if you have one, reaching out to a friend or family member whoworks in medicine (nurse, PA, doctor) to be your medical advocate. The advocatecan come to your appointments or even join a conference call during yourappointments when you need help understanding your treatment options. I waslucky to have both my mom and one of my best friends to help me interpreteverything. Never be afraid to ask your doctor questions, and dont forgetabout the physicians assistant, who often has more availability.

I was going back and forthto the doctor for nearly a year, and they keep dismissing my symptoms. At onepoint, one doctor told me to take probiotics. I finally decided it was time toget a second opinion when I was having trouble walking. Luckily, I found Dr.Drew Freilich, whom I credit with saving my life. He recognized that mysymptoms were severe and insisted that I needed an MRI. Thats how theydiscovered I had a blood cancer that was attacking my bones. I could havebecome disabled if I had waited longer to get help. If you know something iswrong, you have to be persistent about getting answers.

I know it sounds clich, butmy friends, family, and neighbors. They all took excellent care of me. Theydrove me to the hospital for chemotherapy or bone marrow biopsies. My friends were great and woulddrop by to bring me food or help clean up myhouse.

I know it may sound sillybut my dogs really helped keep my spirits up. Quite often, it was just me and the dogs and duringisolation. I truly believe that pets are healing, and studies show that havingone improves your mental health. There were several weeks when I had to be awayfrom my dogs because my immune system was too weak. I was lucky enough to havegreat friends watch my fur babies. I even tried to convince my friends to driveby Emory Hospital so that I could see them.

I would say you have to bepositive. It seems like its a long way away, and you wonder at times whetheror not everything you did is going to pay off when you finally get toremission. So, I think you have to be positive because you get very paranoid. Ibelieve positive thinking can be healing and improve your health. Keeping inmind that everyones journey is different, I think its also important to see apsychologist or therapist. Sometimes its easier to share your real concernswith a stranger. We always try and put on a brave face for family and friends.

Aftereverything, I felt like I had to give back to the cancer community and EmoryWinship Cancer Center. I got my dogs certified to be Happy Tails therapydogs, and now we visit patients battling cancer while they are getting chemo.Its amazing and emotional all at the same time. Many times, patients will say,your puppy made my day.

Iam also trying to raise awareness for the need of more bone marrow donors.Right now, the majority of donors come from Europe. It would be awesome if morepeople would register to be a bone marrow donor. Its a simple swab test. Ithink its a small price to pay, considering more than 170,000 people arediagnosed with blood cancer every year. Check out Be The Match or The Leukemia& Lymphoma Society.

I am not going to sugarcoatit, staying motivated is extremely challenging and a daily battle. I thinkevery cancer survivor questions, why did this happen to me? Is it gone? How longwill I stay in remission? It can be quite depressing, but you have to live forthe day and stick to a routine. I try to remind myself that there is a reasonwhy I am still alive, and I want to give back to others who are struggling.

Everything. I had months ofchemo to get my cancer level down enough to collect my stem cells for thetransplant. I wondered constantly, will I be in remission? And then once Iwas in remission, how long will I stay in remission before I relapse? Whenyoure dealing with blood cancers, most have no cure. So, theres always thatchance of relapse, and youre always worrying about it.

I did six rounds of chemobefore I was even ready to get a transplant. The stem cell transplant wassomething I was dreading because of the high dose of chemotherapy and losing myhair. That can be a very difficult experience, especially for women. After thosesix rounds, they collected my stem cells, which is not a fun process. Then theyprepped me, and I had the transplant.

After, I was in isolation atthe hospital for three weeks. Then I went home, and I was still under isolationfor another 100+ days. I felt like I was ready to lose my mind. During thistime, your white blood cells are regenerating, which means you dont have animmune system, and you suffer from extreme fatigue and pain. Walking up a shortflight of stairs would wipe me out. I couldnt eat salads, fruits, basicallyanything raw. When I left the house, Id have to wear a mask to protect myimmune system. I really hated that because everyone would stare and pretty muchknew I had cancer.

However, to put a positivespin on it, because of my time in isolation at home, I really felt my creativejuices start to flow. I began brainstorming and thinking of a lot of differentthings because life is short, and the cancer was my wake-up call.

So, my best advice duringthat period is to make a reading list and binge-watch shows on Netflix. I readthe Game of Thrones series. Iliterally had a calendar counting down to 100 days. Thats also the time whenyour hair finally starts to grow back!

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A Discussion With Jennifer Delgado on Life After Cancer and Weathering the Storm - Thrive Global

Doting dad Stephen Armstrong knows all too well what its like to be waiting for a transplant donor.

His son Jacob was diagnosed at two years old with a rare blood disorder and called on the public to donate stem cells to find him a match.

He then set out to raise as much money as he could for the blood cancer charity Anthony Nolan in a bid to save lives.

And now, after raising over 20,000, his efforts have been recognised by the charity as they honour him at an awards ceremony held at the Tower of London in November.

Stephen, 33, of Wallsend, North Tyneside, has been shortlisted for the Individual Fundraiser of the Year Award at the Anthony Nolan Supporter Awards 2019.

The prestigious awards are back for their seventh year and will recognise the outstanding achievements of the volunteers, fundraisers and campaigners who help the pioneering blood cancer charity save lives.

Stephens nomination is in recognition of his incredible fundraising efforts, leading a group of 19 friends and family in a series of physical challenges, all while his son was undergoing treatment.

After Jacob was diagnosed in 2017, Stephen set out to find a matching stem cell donor, as well as raise awareness of the need for more people on the register.

From here Jacobs Journey was born, and through a series of challenges including the Great North Run, the Great North Bike Ride and climbing Ben Nevis, Stephen has helped raise over 20,000 for the charity.

Jacob, who turns four in November, and his family have been told he does not need a transplant, but Stephen and his family want to continue raising awareness for others who arent so lucky.

When Jacob was diagnosed, we were stunned by how few people were on the stem cell donor register. I couldnt believe how a stranger in the street could potentially save our little boys life, said Stephen, an assistant manager for Dixons Carphone.

Anthony Nolan helped us massively while Jacob was ill and provided a great support network. I feel very proud to be nominated for an award, and I hope it can help build even more awareness for the cause.

Stephen and mum Kirsty, 28, received the news in December 2017 that Jacob was suffering from bone marrow failure, which affects between 30 and 40 children each year.

They first became concerned about his health when they went abroad to get married and noticed he was getting bruised easily. The marks would take weeks to disappear, so when the couple returned to the UK they decided to take Jacob to the doctor for a check up.

After tests he was then diagnosed and was treated at the Great North Childrens Hospital in Newcastle, where he received two blood transfusions.

Stephen added: When we were told Jacob did not need the transplant it was the best news in the world, a total relief. He still needs check ups every three months and his consultants is keeping an eye on him. There are so few people on the stem cell donor register so I just wanted to create a ripple effect with awareness and get more people on it.

Stephen, who has raised a further 8,000 for other smaller charities, has also been nominated for our Chronicle Champions Award in the Champion Fundraiser category.

Henny Braund, Chief Executive of Anthony Nolan, said: It is remarkable to see how many people support our work to find a match for those in need of a stem cell transplant. Without them, none of our lifesaving work would be possible.

Stephen has shown tremendous commitment to Anthony Nolan by continually going above and beyond in his fundraising efforts.

Henny added: We want to extend a huge congratulations to Stephen and look forward to celebrating with him at the awards.

The awards take place on Thursday 28 November at the Tower of London, and all winners will be revealed on the night.

Anthony Nolan is the charity that finds matching stem cell donors for people with blood cancer and blood disorders and gives them a second chance at life. It also carries out ground-breaking research to save more lives and provide information and support to patients after a stem cell transplant, through its clinical nurse specialists and psychologists, who help guide patients through their recovery.

To see the full shortlist, and find out more about the charity visit http://www.anthonynolan.org/awards

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Dad who called on the public for stem cells for his son is up for an award - Chronicle Live

MELVILLE, N.Y., Oct. 23, 2019 (GLOBE NEWSWIRE) -- BioRestorative Therapies, Inc. (the Company") (BRTX), a life sciences company focused on stem cell-based therapies, today announced that the Australia Patent Office has issued a Certificate of Grant for the Companys patent application titled Human Brown Adipose Derived Stem Cells and Uses.

This is the second patent issued for the Companys brown fat technology in Australia and adds to three other patents related to BioRestoratives metabolic program (ThermoStem Program) that have previously been issued to the Company in the United States and other countries.

This patent will allow for the protection of a specific isolated human brown adipose tissue stem cell line capable of differentiating into multiple cell types. This particular cell line possesses strong characteristics applicable for potential therapeutic uses for treating a wide range of degenerative and metabolic disorders, including diabetes, hypertension, cardiac deficiency and obesity.

This patent, granted by the Australian Patent Office for our metabolic program, adds to our growing family of IP surrounding and protecting our brown fat metabolic cell program, said Mark Weinreb, CEO of BioRestorative Therapies. In particular, our invention relates to an isolated brown fat stem cell line that we expect to be used in our development of cell-based therapies to treat metabolic disorders.

About BioRestorative Therapies, Inc.

BioRestorative Therapies, Inc. (www.biorestorative.com) develops therapeutic products using cell and tissue protocols, primarily involving adult stem cells. Our two core programs, as described below, relate to the treatment of disc/spine disease and metabolic disorders:

Disc/Spine Program (brtxDISC): Our lead cell therapy candidate, BRTX-100, is a product formulated from autologous (or a persons own) cultured mesenchymal stem cells collected from the patients bone marrow. We intend that the product will be used for the non-surgical treatment of painful lumbosacral disc disorders. The BRTX-100 production process utilizes proprietary technology and involves collecting a patients bone marrow, isolating and culturing stem cells from the bone marrow and cryopreserving the cells. In an outpatient procedure, BRTX-100 is to be injected by a physician into the patients damaged disc. The treatment is intended for patients whose pain has not been alleviated by non-invasive procedures and who potentially face the prospect of surgery. We have received authorization from the Food and Drug Administration to commence a Phase 2 clinical trial using BRTX-100 to treat persistent lower back pain due to painful degenerative discs.

Metabolic Program (ThermoStem): We are developing a cell-based therapy to target obesity and metabolic disorders using brown adipose (fat) derived stem cells to generate brown adipose tissue (BAT). BAT is intended to mimic naturally occurring brown adipose depots that regulate metabolic homeostasis in humans. Initial preclinical research indicates that increased amounts of brown fat in the body may be responsible for additional caloric burning as well as reduced glucose and lipid levels. Researchers have found that people with higher levels of brown fat may have a reduced risk for obesity and diabetes.

Forward-Looking Statements

This press release contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events or results to differ materially from those projected in the forward-looking statements as a result of various factors and other risks, including, without limitation, whether the Company will be able to consummate the private placement and the satisfaction of closing conditions related to the private placement and those set forth in the Company's Form 10-K filed with the Securities and Exchange Commission. You should consider these factors in evaluating the forward-looking statements included herein, and not place undue reliance on such statements. The forward-looking statements in this release are made as of the date hereof and the Company undertakes no obligation to update such statements.

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CONTACT:Email: ir@biorestorative.com

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BioRestorative Therapies Receives A Second Patent in Australia For Its Metabolic Program - Yahoo Finance

United Therapeutics received a building permit Tuesday for a $9.5 million build-out of its cell therapy facility on the second floor of Mayo Clinics Discovery and Innovation Building.

The 21,843-square-foot space will house an automated stem cell manufacturing site, which is one of the first of its kind in the country. The Whiting-Turner Contracting Co. is the project contractor.

The technology, approved by the FDA in 2018, allows the Mayo Clinic Center for Regenerative Medicine to produce cells from the bone marrow of a stem cell donor in large enough quantities to be used as treatments in clinical trials. It allows for the treatment of multiple patients at the same time.

Construction began in 2017 on the $32.4 million building at 14221 Kendall Hench Drive. It held a grand opening in August.

The first floor houses three ex-vivo lung perfusion surgical suites used for lung restoration, another form of regenerative medicine. It turns donor lungs, which previously would have previously been unusable, into viable transplant organs. United Therapeutics also collaborates with Mayo Clinic on lung restoration.

The third floor houses the Life Sciences Incubator for biotech entrepreneurs, which offers coworking space, wet labs, business resources, networking and entrepreneurial training.

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United Therapeutics receives permit for cell therapy facility build-out at Mayo - Jacksonville Daily Record

NEW YORK, Oct. 25, 2019 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leader in the development of innovative autologous cellular therapies for highly debilitating neurodegenerative diseases, today announced that it will be presenting at the Dawson James Securities 5th Annual Small Cap Growth Conference, being held on October 28-29, 2019 at the Wyndham Grand Hotel in Jupiter, Florida.

Preetam Shah, PhD, MBA, Chief Financial Officer is scheduled to present on Tuesday, October 29th at 3:40 p.m. Eastern Time, in Track 2 - Preserve Ballroom B, with one-on-one meetings to be held throughout the conference.

Chaim Lebovits, President and CEO of BrainStorm said, We are pleased to have the opportunity to have Dr. Shah present at the Dawson James Small Cap Growth Conference. Dr. Shah, joined BrainStorm in September 2019, and we look forward to having him present the Companys growth strategy and future to a wide audience of accreditied investors.

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

AboutBrainStorm Cell Therapeutics Inc.BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn Cellular Therapeutic Technology Platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm has fully enrolled the Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six sites in the U.S., supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a BLA filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. BrainStorm received U.S. FDA clearance to initiate a Phase 2 open-label multi-center trial of repeat intrathecal dosing of MSC-NTF cells in Progressive Multiple Sclerosis (NCT03799718) in December 2018 and has been enrolling clinical trial participants since March 2019. For more information, visit the company's website.

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

CONTACTS

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

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

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BrainStorm Cell Therapeutics to Present at the Dawson James Securities 5th Annual Small Cap Growth Conference - GlobeNewswire

The study, published in the Journal of Cannabis Research, looked at a number of studies covering cannabis use and the immune system, noting that little is known on circulating white blood cell counts and cannabis use.

The researchers looked at the National Health and Nutrition Examination Survey (20052016), a survey designed to be nationally representative of United States non-institutionalised population, and found that there was a modest association between heavy cannabis use and higher white blood cell count but that neither former nor occasional cannabis use was associated with total or differential WBC counts.

White blood cells are the cells in our body that function mainly as immune cells originating in the bone marrow.

Today, it is known that cigarette smoking generates several chemicals that are implicated in oxidative stress pathways and systemic inflammation and elevated white blood cell count in tobacco cigarette smokers have been well documented, whereas tobacco abstinence is associated with sustained decrease in white blood cell count.

The study highlights how cannabis is able to mediate its effects through the cannabinoid-1 (CB1) and cannabinoid-2 (CB2) receptors.

CB2 receptors can be found in numerous parts of the body related to the immune system, including bone marrow, thymus, tonsils and spleen. CB1 receptors are present in the central nervous system, and at lower levels in the immune system.

The effects of cannabinoids on hematopoiesis, and immune cell proliferation using animal and cell based models has been widely demonstrated and a number of studies have examined the association of cannabis use and white blood cell counts in human immunodeficiency virus (HIV).

The studies have shown a higher white blood cell count in HIV positive men who used cannabis.

Last year a study discovered certain cannabinoids that enhance the immunogenicity of tumour cells, rendering them more susceptible to recognition by the immune system. This discovery is important because the leading class of new cancer fighting agents, termed checkpoint inhibitors, activates the immune system to destroy cancer cells.

Enhancing recognition of cancer cells with cannabinoids may greatly improve the efficacy of this drug class. The Pascal study was the first to identify a mechanism in which cannabinoids may provide a direct benefit in immunotherapy.

When looking at white blood cell counts the study noted that: Several of the important study limitations merit attention. The observational nature of the study constrained causal inferences. Even though NHANES collects blood and urine specimens, drug testing is not conducted, and cannabis use was self-reported which may lead to non-differential misclassification bias. There was no available information on the route of administration of cannabis (smoking, ingestion, etc.) or cannabis preparation/potency.

In addition, the study is based on fairly recent NHANES surveys (200516) which might be more representative of the increasing cannabis potency compared to NHANES III (19881994) surveys.

A number of laboratory studies have reported suppression of immune responses with cannabinoid administration, and some epidemiological studies found lower levels of inflammatory biomarkers such as fibrinogen, C-reactive protein and interleukin-6 in adult cannabis users.

The study also noted that the reported anti-inflammatory effects of cannabis were greatly attenuated when body weight is controlled for and suggests that the inverse cannabis-body weight association might explain the lower levels of circulating inflammatory biomarkers in adult cannabis users.

The study highlights that these alterations of immune responses by cannabis use might be associated with increased susceptibility to infections and hence the higher white blood cell count, however, it notes that it is possible that the elevated white blood cell count and suboptimal health status contributed to cannabis use rather than cannabis use caused suboptimal health.

The study states: This hypothesis, though, cannot be tested as NHANES does not collect information on cannabis use motives. Another potential mechanism can be through the effect of cannabinoids on stem cells. Pre-clinical studies suggest that cannabinoids stimulate hematopoiesis and hence this stimulation to bone marrow tissues can be associated with increased circulating white blood cell count in cannabis users.

Positive associations between heavy cannabis use, and total white blood cell and neutrophil counts were detected. Clinicians should consider heavy cannabis use in patients presenting with elevated white blood cell count.

Research on cannabis use and the immune system is lacking and the study suggests further research is needed to understand the immune related effects of different modes of cannabis use.

The study noted: Research on heavy cannabis use and cardiovascular health is needed as systemic inflammation, increased cardiovascular risk and increased mortality risk have been all associated with white blood cell elevation within the normal physiologic range.

Studies with repeated measures are needed to study immunomodulatory changes in cannabis users, and whether the mode of cannabis use can differentially affect immune responses.

Additional research is needed to understand the immune related effects of different modes of cannabis use and to elucidate the role of proinflammatory chemicals generated from smoking cannabis.

Originally posted here:
Cannabis use and the immune system: white blood cell count - Health Europa

Nicole Duhaney couldnt believe her luck when she learned she was having identical twins.

I felt like had won the lottery, Duhaney, 21, told TODAY Parents. "It was the happiest surprise."

After being pregnant for what felt like an eternity, Duhaney and her boyfriend, Niles Liburd, finally welcomed sons Emre pronounced Em-ree" and Elijah on Dec. 23, 2018.

Our life seemed perfect, the mom from Huddersfield, England, said.

But just three weeks later, Elijah developed a lump on his cheek, and both babies developed colds they couldnt seem to kick. Suddenly, they were projectile vomiting.

Trending stories,celebrity news and all the best of TODAY.

At just 4 months old, Emre and Elijah were both diagnosed with childhood acute myeloid leukemia. The disease, also known as AML, is a type of cancer in which the bone marrow makes a large number of abnormal white blood cells, according to the National Cancer Institute.

Myeloid leukemia is the second most common pediatric blood cancer, but it's still relatively rare. In the United States there are roughly 500 children a year between the ages of 0 and 14 that are diagnosed with AML, according to Dr. Richard Aplenc, a physician-scientist within the Division of Oncology at Children's Hospital of Philadelphia.

Aplenc said it is not surprising that Emre and Elijah were diagnosed at the same time.

"If the twins are identical, then they share the same placenta and the same blood supply, so that leukemic cell goes to the other twin," Aplenc explained. "We know that if leukemia is diagnosed before a year or so, there is 100 percent chance that the other twin will develop it."

Tragically, 10-month-old Elijah passed away at home in Tuesday. Doctors allowed Emre, who is currently undergoing chemotherapy, to leave the hospital so he could say goodbye to his brother.

The love they had for each other was just unbreakable, Duhaney noted. "They didn't like to be separated."

She recalled how Elijah pulled his brother in for a kiss after a recent stem cell transplant.

Elijah was beautiful. Every person he met, he touched their heart," Duhaney said. There were times when I cried and he rubbed my tears away. I wish God took me instead of him.

As Duhaney and Liburd, 26, make funeral arrangements a GoFundMe has been set up to help the couple with expenses they are finding comfort in knowing Elijah took his final breaths at home.

He spent six months of his life in a hospital, Duhaney told TODAY Parents. His final night he was where he wanted to be, with the people who loved him him the most.

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Baby dies from AML, the same cancer his identical twin has - TODAY

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Children, suffering from DMD, usually die of cardio-respiratory failure. Represtational image

Duchenne Muscular Dystrophy (DMD) is a deadly genetic disorder, 99.9 per cent people suffering from which, die between the age of 13 to 23 years. However, in a first, a 26-year-old patient from Lucknow has survived DMD by regularly taking stem cells for the last five years.

Children, suffering from DMD, usually die of cardio-respiratory failure. But with the stem cell therapy, this patient has not lost muscle power in last five years and heart and lung muscles and the upper half of the body are working well.

Dr. B.S Rajput, the surgeon who is treating this patient, said, "DMD is a type of muscular dystrophy and being a genetic disorder, it is very difficult to treat. Autologous (from your own body) bone marrow cell transplant or stem cell therapy in such cases was started in Mumbai about 10 years back.

Dr Rajput, who was recently appointed as visiting professor at GSVM Medical College, Kanpur, said he has treated several hundred DMD patients and recently this combination protocol was published in the international Journal of Embryology and stem cell research.

The patient's father is elated that his son has maintained well with this treatment and now has even started earning by working on computers.

According to Dr Rajput, this disease is endemic in eastern UP, especially Azamgarh, Jaunpur, Ballia and some of the adjoining districts of Bihar, and one out of every 3,500 male child, suffers from the disease.

Yet the disease is not given as much attention as it should be.

Dr Rajput, who is consultant bone cancer and stem cell transplant surgeon from Mumbai, said though patients in Uttar Pradesh and Bihar get financial support from the Chief Minister's Relief Funds, the treatment of autologous bone marrow cell transplant is not included in the package list of Ayushman Bharat scheme, which deprives many from getting the treatment.

The doctor further informed that efforts are being made to establish the department of regenerative medicine in the medical college, where bone marrow cell transplant and stem cell therapy would be done even for other intractable problems like spinal cord injury, arthritis knee and motor neurone disease.

ALSO READ |Fasting triggers regeneration of stem cells capacity: Study

ALSO READ |UK patient 'free' of HIV after stem cell treatment

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In a first, 26-year-old DMD patient in UP survives with stem cell therapy - India TV News

Whether youve been recently diagnosed with multiple sclerosis (MS) or have been living with the condition for a while, chances are youll sometimes hear terms from your healthcare team that are new to you.

The following is a quick, alphabetical guide to the terminology you may need to know as you manage your condition:

Ankle-Foot Orthosis (AFO) A brace designed to support the position of the foot and motion of the ankle to compensate for nerve damage and muscle weakness in the area caused by MS and other movement disorders. An AFO is typically used to stabilize weak limbs or to reposition a limb with contracted muscles into a more normal position.

Autoimmune Disease Your immune system plays a major part of your bodys defense against bacteria and viruses by sending out cells to attack them once they enter your body. However, if you have an autoimmune disease, your immune system mistakenly attacks healthy cells in your body, causing them to weaken or break down. MS is thought to be just one example of an autoimmune disease. It has been suggested that in MS, your immune system may mistakenly attack the cells in your central nervous system.

Axon Long threadlike structures of nerve cells that send impulses to other cells in your body. Research suggests that damage to or loss of these fibers in progressive MS may be linked to worsening disability and more severe progression.

Central Nervous System (CNS) The group of organs in your body that includes the brain, spinal cord, and optic nerves. If you have MS, your bodys immune system may be working against the CNS, producing neurological symptoms such as muscle weakness and vision problems.

Cerebrospinal Fluid (CSF) A clear, colorless liquid that surrounds the brain and spinal cord to protect the CNS and assist in the circulation of nutrients and removal of waste products. In MS, damage to the myelin sheath of nerve cells causes certain types of proteins to be released into the spinal fluid. The presence of these proteins in the CSF, but not in the blood, may point to a diagnosis of MS.

Clinically Isolated Syndrome (CIS) A first episode of neurologic symptoms that lasts at least 24 hours and is caused by inflammation or demyelination (loss of the myelin that covers the nerve cells) in the CNS. People who experience CIS may or may not go on to develop MS. However, when CIS is accompanied by magnetic resonance imaging (MRI)detected brain lesions similar to those found in MS, you have a 60 to 80 percent chance of a second neurologic event and diagnosis of MS within several years, according to the National MS Society.

Cog Fog A commonly used term that refers to the cognitive changes experienced by many people with MS. According to MS Australia, approximately 50 percent of people with the condition will develop some degree of cog fog, or inhibited ability to think, reason, concentrate, or remember. For some, cognitive problems will become severe enough to interfere in a significant way with daily activities.

Corticosteroids (or Steroids) Prescription medication used to treat relapses in relapsing-remitting MS. Your doctor may prescribe intravenous (IV) corticosteroids if the symptoms of your relapse are causing significant problems, like poor vision or difficulty walking. These drugs work by suppressing the immune system and reducing inflammation in the CNS, and they may help relapse symptoms resolve more quickly. But they wont affect your ultimate level of recovery from a relapse or the long-term course of your MS. Methylprednisolone is a commonly used corticosteroid in MS.

Diplopia (or Double Vision) An eye problem in which you see two images of a single object. It may be present when only one eye is open (monocular) or disappear when either eye is closed (binocular). Diplopia is a common symptom of MS, and it occurs because of damage to the optic nerve.

Disease-Modifying Therapies (DMTs) Drugs designed to reduce new relapses, delay progression of disability, and limit new CNS inflammation in people with MS. Although there are multiple DMTs that have been approved by the U.S. Food and Drug Administration (FDA) for use in MS, these drugs generally work by reducing inflammation in nerve cells in theCNS.

Dysarthria A speech disorder caused by neuromuscular impairment and resulting in disturbances in motor control of the muscles used in speech. Its believed the demyelinating lesions in MS may result in spasticity, weakness, slowness, or ataxic incoordination of the lips, tongue, mandible, soft palate, vocal cords, and diaphragm, causing this speech impairment.

Dysphagia (Difficulty Swallowing) A condition that may occur in people with MS, leading to difficulty in eating solid foods or liquids, frequent throat clearing during eating or drinking, a feeling that food is stuck in the throat, or coughing or a choking sensation when eating or drinking. Its the result of nerve damage within the muscles that control swallowing.

Epstein-Barr Virus (EBV) A virus believed to be a possible cause or trigger for MS. Although the exact cause of MS remains unknown, researchers suggest an infectious agent may be involved in its development. Studies have found that antibodies (immune proteins that indicate a person has been exposed to a given virus) to EBV are significantly higher in people who eventually develop MS than in those who dont. Other research has noted that people with a specific immune-related gene and high levels of antibodies to EBV in their blood are 9 times more likely to develop MS than others.

Evoked Potentials A test that measures the speed of nerve messages along sensory nerves to the brain, which can be detected on your scalp using electrodes attached with sticky pads. Its sometimes used in the diagnosis of MS, because nerve damage can slow down the transmission of nerve signals. Evoked potential tests can indicate nerve pathways that are damaged prior to the onset of MS symptoms.

Exacerbation An occurrence of new symptoms or the worsening of old symptoms that may also be referred to as a relapse, attack, or flare-up. Exacerbations can be very mild, or severe enough to interfere with a person's ability to perform day-to-day activities.

Expanded Disability Status Scale (EDSS) A scale used for measuring MS disability and monitoring changes in the level of disability over time. Developed by neurologist John Kurtzke, MD, in 1983, the EDSS scale ranges from 0 to 10 in 0.5-unit increments (scoring is based on a neurological exam) and relies on walking as its main measure of disability. People with an EDSS of 1 have no disability and minimal loss of function, while those with an EDSS of 9.5 are confined to bed and totally dependent on others for functions of daily living.

Foot Drop (or Drop Foot) A symptom of MS caused by weakness in the ankle or disruption in the nerve pathway between the legs and the brain, making it difficult to lift the front of the foot to the correct angle during walking. If you have foot drop, your foot hangs down and may catch or drag along the ground, resulting in trips and falls. Foot drop can be managed with an AFO or other treatments.

Hematopoietic Stem Cell Transplantation (HSCT) A procedure designed to reboot the immune system, the National MS Society says, using hematopoietic (blood cellproducing) stem cells derived from a persons own bone marrow or blood. If your doctor recommends HSCT, youll undergo a chemotherapy regimen before these cells are reintroduced to the body via IV injection, where they will migrate to your bone marrow to rebuild the immune system.

John Cunningham (JC) Virus A common infection completely unrelated to MS that is found in as many as 90 percent of people, according to the UK's MS Trust. JC virus has no symptoms and is normally controlled by the immune system. However, if your immune system is weakened, the JC virus can reactivate, causing potentially fatal inflammation and damage to the brain known as progressive multifocal leukoencephalopathy (PML). Certain MS disease-modifying therapies have been linked with increased risk for PML.

Lhermittes Sign An electric shock-like sensation experienced by some with MS when the neck is moved in a particular way. The sensation can travel down to the spine, arms, and legs.

Lesion (or Plaque) Refers to an area of damage or scarring (sclerosis) in the CNS caused by inflammation in MS. These lesions can be spotted on an MRI scan, with active lesions appearing as white patches. With regular MRIs, a neurologist can tell how active your MS is.

Lumbar Puncture (or Spinal Tap) A procedure used for the collection of cerebrospinal fluid (CSF), sometimes done to help diagnose MS. For this procedure, your doctor will ask you to lie on your side or bend forward while seated, before cleansing an area of your lower back and injecting a local anesthetic. He will then insert a hollow needle and extract a small amount of spinal fluid using a syringe.

Magnetic Resonance Imaging (MRI) The diagnostic tool that currently offers the most sensitive noninvasive way of imaging the brain, spinal cord, or other areas of the body, according to the National MS Society. Its the preferred imaging method for diagnosis of MS and to monitor the course of the disease. MRI uses magnetic fields and radio waves to measure the relative water content in tissues, which is notable in MS because the layer of myelin that protects nerve cell fibers is fatty and repels water. In areas where myelin has been damaged by MS, fat is stripped away and the tissue holds more water. This shows up on an MRI as a bright white spot or darkened area, depending on how the images are made.

McDonald Criteria A guidance used in the diagnosis of MS, authored by an international panel of experts on the condition, originally in 2010. The guidance was updated in 2017. Among the key changes: advising for the use of brain MRI as part of the diagnostic process.

MS Hug A common symptom of MS. If you experience the MS hug, you may feel like you have a tight band around your chest or ribs, or pressure on one side of your torso. Some people find that it is painful to breathe. The MS hug can last for seconds, minutes, hours, or even longer.

Myelin A substance rich in lipids (fatty substances) and proteins that helps form the myelin sheath. In MS, particularly relapsing-remitting MS, an abnormal immune response produces inflammation in the CNS, effectively attacking the myelin in the cells.

Myelin Sheath An insulating layer of fatty substances and proteins that forms around the nerves in body, including those in the CNS. It allows electrical impulses to transmit quickly and efficiently along the nerve cells, but these impulses can be slowed if the sheath is damaged, causing MS.

Neurodegeneration Refers to the process by which the myelin sheath of cells in the CNS is damaged in MS. Its believed to be a major contributor to neurological disability in the condition, and may be the reason immune modulation treatments (disease-modifying therapy) are generally less effective in the progressive MS than in the relapsing-remitting MS.

Neurologist The point person for monitoring your MS treatment and managing MS symptoms. This specialist typically focuses on conditions affecting the CNS.

Neuropathic Pain A type of pain common in MS that results from changes or damage to the myelin sheath and the axons, or nerve fibers, it normally covers. MS-caused neuropathic pain may be chronic, intermittent, or occur only in response to a stimulus.

Neuropsychologist A specialist you may be referred to who helps you manage the cognitive effects of MS. Neuropsychological testing (or testing of the functioning of your brain) involves identifying memory or learning difficulties associated with MS. Cognitive rehabilitation may improve functioning.

Nociceptive Pain Caused by damage to muscles and joints, it can be either acute or chronic, and may not result from MS itself, but be caused by changes in posture or walking or the overuse of assistive devices in those with the condition.

Nystagmus A common eye abnormality in MS, its characterized by involuntary, rhythmic, back-and-forth motion of the eyeball, either horizontally or vertically. For those with nystagmus, the perception of the rhythmic movement of the surrounding stationary world (oscillopsia) can be disorienting and disabling.

Oligoclonal Bands (OCBs) Immunoglobulins, or proteins, that collect in blood plasma or cerebrospinal fluid (CSF). Although not every person with MS has OCBs, their presence can support a diagnosis of MS. Having OCBs is generally associated with a younger age of MS onset and a poorer prognosis.

Optic Neuritis An inflammatory condition that damages the optic nerve, a bundle of nerve fibers that transmits visual information from your eye to your brain, causing pain and temporary vision loss in one eye. Its been linked with nerve damage resulting from MS, and may be among the first symptoms a person with the condition experiences.

Pseudobulbar Affect (PBA) A neurologic effect experienced by roughly 10 percent of people with MS as well as some with Parkinsons disease or amyotrophic lateral sclerosis (ALS), according to the Multiple Sclerosis Association of America (MSAA). Its characterized by sudden, uncontrollable expressions of laughter or crying without an obvious cause, which can be distressing as well as embarrassing to those who experience it. PBA is believed to be a mood disorder related to the disruption of nerve impulses in the CNS, but its different from depression, which is also common in MS.

Pseudoexacerbation A temporary worsening of symptoms without actual myelin inflammation or damage. It is often triggered by other illnesses or infection, exercise, a warm environment, depression, exhaustion, and stress. Urinary tract infection (UTI) is the most common type of infection to cause a pseudoexacerbation.

Sclerosis A general hardening of the body tissue. The term multiple sclerosis refers to the multiple areas of scar tissue often called lesions that develop along affected nerve fibers and that are visible in MRI scans.

Spasticity A symptom of MS that causes your muscles to feel stiff, heavy, or difficult to move. When a muscle spasms, youll experience a sudden stiffening that may cause a limb to jerk. This may be painful.

Trigeminal Neuralgia (or Tic Douloureux) A type of neuropathic pain that occurs on the face (usually on one side only). Its a known symptom of MS, and you may experience it in your cheek; upper or lower jaw; inside the mouth; or in the area around your eyes, ears, or forehead. In MS, its typically caused by damage to the myelin sheath around the trigeminal nerve, which among other functions controls the muscles used in chewing. The condition is triggered by everyday activities, like tensing facial muscles while shaving or when chewing.

Vertigo An intense sensation of the surrounding environment spinning around one. In MS, vertigo is typically caused by growth of an existing lesion or development of a new lesion on the brain stem or cerebellum, the area in the brain that controls balance. It can also be a symptom of a problem with the inner ear, or it can be side effect of medication used to treat MS or other health conditions you may have.

More:
Speaking Multiple Sclerosis: A Glossary of Common Terms - Everyday Health

The Eastland Companion Animal Hospital in Bloomington is asking dog owners if they want to participate in research on using stem cells to treat dogs with arthritis.

Local dogs wouldjoin a double-blind, placebo-controlled studyto show the effectiveness of stem cells in treating large dogs(70 pounds or more) with arthritis in up to two joints of the knee, hip, elbow, or shoulder. The veterinary clinic has partnered with Animal Cell Therapies, who it's worked with before, to bring this study to Bloomington.

Dr. Kathy Petrucci, founder and CEO of Animal Cell Therapies, explained how dogs will receive the treatment.

The dogs that will receive the stem cells will be sedated, Petrucci said. Depending on what joints are affected, they will receive up to two injections in the joint and they will also receive an IV dose of stem cells.

The FDA oversees the cells that are received from donors for the study. Mothers donating these cells are screened for diseases, and cells are tested for any infections to ensure safety.

Stem cell therapy has been controversial, especially related to humans.

I think a lot of the controversy comes from the misunderstanding of the cell types, Petrucci said. The research in stem cells first started centered around embryonic or fetal tissue use. Its controversial to use embryos and fetal tissues for treatment for anything. The fact that we are using a disposable tissue as our cell sources makes it not controversial at all.

Why Umbilical-Derived Cells

Petrucci explained why umbilical-derived cells are more effective in treating arthritis versus other sources.

We looked at fat, bone marrow, embryonic cells, Petrucci said. The embryonic cells are a lot more unpredictable, and the bone marrow cells are more difficult to work with and less predictable. We didnt think the fat cells are as potent as umbilical-derived cells. Umbilical-derived cells are a lot younger and theyre a little bit more predictable. They are more easy to collect. We obtain cells from donors when the tissue would be normally thrown away. Theres no surgery required, no extra biopsies to obtain fat, no bone marrow from research animals. Its a good, ethical source of stem cells.

Umbilical-derived stem cells have proven successful in past studies on treatment for arthritis, according to Petrucci.

We did a study at the University of Florida on elbows only and we had success with that study, Petrucci said. We had good success with dogs under 70 pounds and (less) success with dogs over 70 pounds, so we changed our dose, which is why were testing dogs 70 pounds and over in this study.

Criteria for eligibility includes dogs weighing 70 pounds or more, being one year of age or older, in general good health, no neurologic issues, arthritis in up to two joints of the knee, hip, elbow, or shoulder, and have all four functioning limbs.

Owners must bring their dogs back to the clinic after 30 days to check for progress and complete a questionnaire. About 50 to 100 dogs are expected to participate in the study.

People like you value experienced, knowledgeable and award-winning journalism that covers meaningful stories in Bloomington-Normal. To support more stories and interviews like this one,please consider making a contribution.

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Bloomington Vet Joins Study For Stem Cell Therapy To Treat Dogs With Arthritis - WGLT News

Ewing sarcoma is a form of bone cancer that usually affects children and adolescents.

Ewing sarcoma can be very aggressive, but the cells tend to respond well to radiation therapy. Ideally, doctors will diagnose the cancer before it has spread.

According to the National Library of Medicine, an estimated 250 children in the United States receive a diagnosis of Ewing sarcoma each year.

In this article, learn more about Ewing sarcoma, including the symptoms, causes, and treatment options.

Ewing sarcoma is a rare type of cancer that usually starts in the bone typically in the pelvis, chest wall, or legs and occurs mostly in children and teenagers.

Dr. James Ewing first described Ewing sarcoma in 1921. He identified cancer cells that looked different than the cells in osteosarcoma, another type of bone tumor.

Doctors may also refer to this cancer type as the Ewing family of tumors. These tumors have distinct cells that usually respond well to radiation treatments.

This rare cancer type accounts for just 1.5% of all childhood cancers and is the second most common bone cancer type in childhood, after osteosarcoma.

Although researchers are unsure why some people develop Ewing sarcoma, they have identified mutations in certain genes in the tumor cells that cause this cancer.

These include the EWSR1 gene on chromosome 22 and the FLI1 gene on chromosome 11.

These genetic mutations occur spontaneously during a person's lifetime. The individual does not inherit them from a family member.

There are no known risk factors for Ewing sarcoma that make one person more likely than another to develop this cancer.

Ewing sarcoma can cause the following symptoms:

An estimated 87% of Ewing sarcomas are sarcoma of the bone. The other types form in the soft tissues, such as cartilage, that surround the bones.

Ewing sarcoma can spread to other areas of the body. Doctors call this process metastasis.

Areas that the cancer can spread to include other bones, bone marrow, and the lungs.

Doctors categorize Ewing sarcoma as one of three types according to its extent:

Before diagnosing Ewing sarcoma, a doctor will take a person's full medical history and ask them what symptoms they are having, when they noticed them, and what makes them better or worse. They will also perform a thorough physical exam, focusing on the area of concern.

A doctor will usually recommend an imaging study to view the bone or bones. These tests include:

If it looks as though a tumor may be present, a doctor will perform a biopsy, which involves taking a sample of bone tissue. They will send this tissue to a laboratory, where a specialist called a pathologist will check it for the presence of cancerous cells.

A doctor may also order blood tests, a bone marrow biopsy, and other scans when necessary. These tests can help determine whether the cancer has spread to other locations.

A doctor will work with a team of cancer specialists and surgeons to recommend and implement particular treatments.

Possible treatments for Ewing sarcoma include:

Doctors may use a combination of treatments depending on how far the cancer has spread and a person's overall health.

Research into new treatments for Ewing sarcoma is ongoing. Some doctors may inform their patients about clinical trials, which help test new treatments.

Possible complications of Ewing sarcoma include:

If Ewing sarcoma has spread to other areas of the body, it can be life threatening. For this reason, it is vital for a doctor to evaluate any symptoms as quickly as possible.

According to the American Academy of Orthopaedic Surgeons, an estimated two-thirds of people in whom cancer has not spread to other areas of the body survive at least 5 years after their diagnosis.

People who are more likely to have positive outcomes include those who have:

The likelihood of successful treatment is different for every individual, so people should speak to a doctor about their or their child's expected outlook.

Ewing sarcoma is a rare type of cancer that mostly affects young people.

When doctors detect it early enough, the condition usually responds well to treatment.

Anyone who notices signs or symptoms of Ewing sarcoma, such as a bone that breaks for no apparent reason or a painful lump or swelling, should speak to a doctor.

Excerpt from:
Ewing sarcoma: Causes, symptoms, and treatment - Medical News Today

NEW YORK, Oct. 24, 2019 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leader in the development of innovative autologous cellular therapies for highly debilitating neurodegenerative diseases, today announced, Chaim Lebovits, President and CEO, will serve as a Keynote Speaker at Cell Series UK.Cell Series UK, will be held October 29-30, 2019, at London Novotel West, London, UK. The Conference, organized by Oxford Global, is one of the foremost events in Europe focused on regenerative medicine and cellular innovation.

Ralph Kern MD, MHSc, Chief Operating and Chief Medical Officer of Brainstorm, who will also participate at Cell Series UK stated, We are very pleased to have Chaim Lebovits presenting at this prestigious conference where global leaders in stem cell and regenerative medicine will have the opportunity to learn more about NurOwn and the critical research being conducted by the Company. Mr. Lebovits Keynote Address, Stem Cell Therapeutic Approaches For ALS, will be presented to leading members of the scientific and business community including potential partners and investors.

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

AboutBrainStorm Cell Therapeutics Inc. BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn Cellular Therapeutic Technology Platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm has fully enrolled the Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six sites in the U.S., supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a BLA filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. BrainStorm received U.S. FDA clearance to initiate a Phase 2 open-label multi-center trial of repeat intrathecal dosing of MSC-NTF cells in Progressive Multiple Sclerosis (NCT03799718) in December 2018 and has been enrolling clinical trial participants since March 2019. For more information, visit the company's website.

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

CONTACTS

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

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

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BrainStorm Cell Therapeutics' President and CEO to be Featured as Keynote Speaker at Cell Series UK 2019 - GlobeNewswire

BOSTON and LONDON, Oct. 22, 2019 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, today announced initial results from a clinical trial with a cryopreserved formulation of OTL-200, a gene therapy in development for the treatment of metachromatic leukodystrophy (MLD) at the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. The initial data show that cellular engraftment with OTL-200 using a cryopreserved formulation is similar to that observed using a fresh formulation with the longest patient having 12 months of follow-up since treatment. The data are being featured this week in a poster session at the European Society of Gene & Cell Therapy (ESGCT) Annual Congress in Barcelona, Spain.

MLD is a devastating and rapidly progressing disease with no standard treatment options. In its most severe forms, patients will not survive beyond their first decade of life.

These data compare the initial results of OTL-200 in the first four MLD patients treated using a cryopreserved formulation to a previously presented integrated analysis of 29 patients treated with a fresh formulation that demonstrated meaningful clinical outcomes. Hematopoietic stem cells are collected, purified and transduced in the same way for both formulations. For the cryopreserved formulation, following transduction, the gene-corrected cells are placed in a specific medium that allows them to be stably frozen. After successful testing and release, the cryopreserved cells are shipped to the site of care where they are thawed and administered to patients who have received conditioning.

Presenting the first supportive data on OTL-200 using a cryopreserved formulation represents a cross-functional effort involving our clinical, CMC and regulatory teams as we prepare for the upcoming European regulatory submission for MLD followed by a BLA in the U.S., said Mark Rothera, president and chief executive officer of Orchard. If approved, a cryopreserved formulation of OTL-200 would more readily facilitate global commercialization and patient access efforts, which are key elements in our mission to deliver potentially curative therapies to patients suffering from often-deadly rare diseases.

Mr. Rothera continued, With over 40 patients now treated using a cryopreserved formulation across our pipeline of six clinical-stage programs, we are confident our approach is supported by a robust set of evidence.

Study Results At the time of the analysis, four early-onset MLD patients (two late infantile and two early juvenile) have been treated with the cryopreserved formulation of OTL-200. All patients are alive and were followed for a minimum of one month, with the longest follow-up out to 12 months in the first patient treated (median follow-up of 0.38 years). The age at the time of treatment ranged from seven months to 42 months.

The initial results in patients receiving the cryopreserved formulation (n=4) demonstrated the following:

Figure 1. Profiles of VCN in bone marrow CD34+ cells: OTL-200 cryopreserved vs. OTL-200 fresh

https://www.globenewswire.com/NewsRoom/AttachmentNg/83f41457-927b-4b1b-9ac2-9d48ac10353a

Figure 2. ARSA activity profile in peripheral blood: OTL-200 cryopreserved vs. OTL-200 fresh

https://www.globenewswire.com/NewsRoom/AttachmentNg/393ca5f0-98ad-47f8-b723-35c5c6c08d8f

c = cryopreserved; f = fresh; Sbj. = subject

We are pleased that these initial data suggest that using gene-corrected cells that have been cryopreserved has a similar impact on clinical biomarkers for early-onset MLD patients as the OTL-200 fresh formulation, said Dr. Valeria Calbi, a hematologist at San Raffaele Scientific Institute and SR-Tiget and an investigator of the study. The four treated patients showed good levels of engraftment of gene-corrected cells and reconstitution of ARSA activity at multiple time points, as well as encouraging early trends in GMFM scores that we look forward to evaluating with additional follow-up. We believe that these data further support the positive benefit / risk profile of OTL-200 as a therapy with potential lifelong benefit for patients with MLD.

Next Steps for OTL-200 Orchard remains on track to submit a marketing authorization application, or MAA, in Europe for MLD in the first half of 2020, as well as a biologics licensing application, or BLA, in the U.S. approximately one year later.

About MLD and OTL-200Metachromatic leukodystrophy (MLD) is a rare and life-threatening inherited disease of the bodys metabolic system occurring in approximately one in every 100,000 live births. MLD is caused by a mutation in the arylsulfatase-A (ARSA) gene that results in the accumulation of sulfatides in the brain and other areas of the body, including the liver, the gallbladder, kidneys, and/or spleen. Over time, the nervous system is damaged and patients with MLD will experience neurological problems such as motor, behavioral and cognitive regression, severe spasticity and seizures, finding it more and more difficult to move, talk, swallow, eat and see. Currently, there are no effective treatments for MLD. In its late infantile form, mortality at 5 years from onset is estimated at 50% and 44% at 10 years for juvenile patients.1 OTL-200 is an ex vivo, autologous, hematopoietic stem cell-based gene therapy being studied for the treatment of MLD. OTL-200 was acquired from GSK in April 2018 and originated from a pioneering collaboration between GSK and the Hospital San Raffaele and Fondazione Telethon, acting through their joint San Raffaele-Telethon Institute for Gene Therapy in Milan, initiated in 2010.

About OrchardOrchard Therapeutics is a fully integrated commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies.

Orchards portfolio of ex vivo, autologous, hematopoietic stem cell (HSC) based gene therapies includes Strimvelis, a gammaretroviral vector-based gene therapy and the first such treatment approved by the European Medicines Agency for severe combined immune deficiency due to adenosine deaminase deficiency (ADA-SCID). Additional programs for neurometabolic disorders, primary immune deficiencies and hemoglobinopathies are all based on lentiviral vector-based gene modification of autologous HSCs and include three advanced registrational studies for metachromatic leukodystrophy (MLD), ADA-SCID and Wiskott-Aldrich syndrome (WAS), clinical programs for X-linked chronic granulomatous disease (X-CGD), transfusion-dependent beta-thalassemia (TDT) and mucopolysaccharidosis type I (MPS-I), as well as an extensive preclinical pipeline. Strimvelis, as well as the programs in MLD, WAS and TDT were acquired by Orchard from GSK in April 2018 and originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy initiated in 2010.

Orchard currently has offices in the U.K. and the U.S., including London, San Francisco and Boston.

Forward-Looking StatementsThis press release contains certain forward-looking statements which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as anticipates, believes, expects, intends, projects, and future or similar expressions that are intended to identify forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, Orchards expectations regarding the timing of regulatory submissions for approval of its product candidates, including OTL-200 for the treatment of metachromatic leukodystrophy, the timing of interactions with regulators and regulatory submissions related to ongoing and new clinical trials for its product candidates, the timing of announcement of clinical data for its product candidates, including OTL-200, and the likelihood that such data will be positive and support further clinical development and regulatory approval of its product candidates, and the likelihood of approval of such product candidates by the applicable regulatory authorities. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, the risks and uncertainties include, without limitation: the risk that any one or more of Orchards product candidates, including OTL-200, will not be successfully developed or commercialized, the risk of cessation or delay of any of Orchards ongoing or planned clinical trials, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates, the delay of any of Orchards regulatory submissions, the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates, the receipt of restricted marketing approvals, and the risk of delays in Orchards ability to commercialize its product candidates, if approved. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading Risk Factors in Orchards annual report on Form 20-F for the year ended December 31, 2018 as filed with the U.S. Securities and Exchange Commission (SEC) on March 22, 2019, as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

1Mahmood et al. Metachromatic Leukodystrophy: A Case of Triplets with the Late Infantile Variant and a Systematic Review of the Literature. Journal of Child Neurology 2010, DOI: http://doi.org/10.1177/0883073809341669

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaMolly CameronManager, Corporate Communications+1 978-339-3378media@orchard-tx.com

Figure 1

Profiles of VCN in bone marrow CD34+ cells: OTL-200 cryopreserved vs. OTL-200 fresh

Figure 2

ARSA activity profile in peripheral blood: OTL-200 cryopreserved vs. OTL-200 fresh; c = cryopreserved; f = fresh; Sbj. = subject

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Orchard Therapeutics Presents Data from OTL-200 in Patients with Metachromatic Leukodystrophy Using Cryopreservation - BioSpace

Across the US,more than 100,000 people are awaiting organ transplants. But there simply arent enough hearts, lungs, livers, and kidneys to meet demand, and 20 people die every day without the organs they need.For decades, scientists have dreamed of using animals to help fill the gap. Theyve been particularly interested in harvesting organs from pigs, whose physiology is similar to our own. Unfortunately, pigs also present some big biological challenges, including the fact that their genomes are chock full of genes that code for what are known as retroviruses, which could pose a serious threat to patients who receive porcine organs.

In 2015, George Church, a geneticist at Harvard University, announced a stunning breakthrough: Working with pig cells, he and his colleagues had managed to disable 62 copies of a retrovirus gene inone fell swoop.This would have been virtually impossible and a logistical nightmare with older forms of genetic modification, writes Nessa Carey in her new book, Hacking the Code of Life: How Gene Editing Will Rewrite Our Futures.But by using the new gene editing technology known as Crispr, the task was a relative cinch.

Its just one example of how gene editing is giving us the power to alter the genome with unprecedented speed and precision. Carey, a biologist with a background in the biotech and pharmaceutical industry, offersa brisk, accessible primer on the fast-moving field, a clear-eyed look at a technology that is already driving major scientific advances and raising complex ethical questions

Its giving every biologist in the world the tools to answer in a few months questions that some scientists have spent half their careers trying to address, Carey writes. Its fueling new ways to tackle problems in fields as diverse as agriculture and cancer treatments. Its a story that began with curiosity, accelerated with ambition, will make some individuals and institutions extraordinarily wealthy, and will touch all our lives.

Though there are several different approaches to gene editing, the most prominent and the one that really supercharged the field is Crispr. The technique, based on an anti-viral defense system thats naturally present in bacteria, requires two pieces of biological material: an enzyme that acts as a pair of minuscule scissors, slicing strands of DNA in two; and a guide molecule that tells the enzyme where to cut.

In bacteria, these guide molecules direct the enzyme to chop up the genomes of invading viruses, preventing them from replicating.

But in 2012 and 2013, two teams of scientists reported that it was possible to hack this system to slice into any strand of DNA, at any complementary location they chose.Researchers could, for instance, create a guide molecule that steered the enzyme to one specific gene in the mouse genome and insert the editing machinery into a mouse cell; the enzyme would then make its cut at that exact spot.

The cell would repair the severed DNA, but it would do so imperfectly, disabling the gene in question.In the years that followed, scientists refined the technique, learning to use it not only to inactivate genes but also to insert new genetic material at specific locations along the genome.

The approach is cheaper, easier, and faster than older methods of genetic engineering, which were first developed in the 1970s. In addition, as Carey explains, it can be used to create smaller modifications to the genome, and leaves fewer extraneous genetic elements. In its most technically exquisite form, gene editing leaves no molecular trace at all. It may just change, in a precisely controlled manner, one letter of the genetic alphabet.

The applications are almost endless.Gene editinghas immense potential for basic research; scientists can learn a lot about what genes do by selectively disabling them. In addition, researchers have used the technology to create a wide variety of organisms that could become valuable agricultural commodities, including mushrooms that dont brown; wheat that produces fewer gluten proteins;drought tolerant, high-yield rice and corn; disease-resistant pigs; and super muscular goats.

How these products will do on the market if they ever reach it remains uncertain.Globally, gene-edited organisms are regulated by a patchwork of conflicting rules. For instance,in 2018, the U.S. Department of Agricultureannouncedthat it would not regulate gene-edited crops that could otherwise have been developed through traditional breeding techniques. A few months later, however, the European Union said that it would subject gene-edited plants to stringent restrictions.

Beyond agriculture, gene editing has enormous potential for medicine. It might, for instance, become a much-needed treatmentfor sickle cell disease. That painful, debilitating disease results from a genetic mutation that causes patients to produce a deformed version of hemoglobin, a protein that helps red blood cells transport oxygen.Ina clinical trial currently underway, scientists are removing stem cells from the bone marrow ofsickle cell patients, using Crispr to edit them, and then infusing the edited cells back into patients.

Even if this trial succeeds, however, gene editing will not be a cure-all.It doesnt always work perfectly and can be challenging to administer directly to living humans (which is why some scientists are instead editing patients cells outside the body). Moreover, many diseases are caused by complex interactions between multiple genes, or genes and the environment. In fact, many of the most common and debilitating conditions arent likely to be good candidates for gene editing any time soon, Carey writes.

And, of course, the ethics of human gene editing can be enormously fraught. Thats especially true when scientists modify sperm cells, egg cells, or early embryos, making tweaks that could be passed down to subsequent generations.This kind of gene editing could theoretically cure some absolutely devastating genetic conditions, but we still have a lot to learn about its safety and effectiveness. It also raises a host of difficult questions about consent (an embryo obviously cannot give it), inequality (who will have access to the technology?), and discrimination(what will the ability to edit a gene related to deafness mean for deaf people, deaf culture, and the disability rights movement more broadly?).

Even in the face of these questions, at least one scientist has already forged ahead. In November 2018, He Jiankui, a researcher then at the Southern University of Science and Technology in China,shocked the worldby announcing that the worlds first gene-edited babies twin girls, who He called Nana and Lulu had already been born. Months earlier, when Nana and Lulu were just embryos, He had edited their CCR5 genes, which code for a protein that allows HIV to infect human cells. By disabling the gene, He hoped to engineer humans who would be protected from HIV infection.

The outcry was swift and harsh. Scientists alleged that Hes science was sloppyand unethical, putting two human beings at unnecessary risk.After all, there are already plenty of ways to prevent HIV transmission, and the CCR5 protein is known to have some benefits, including protecting against the flu.And He had raced ahead of the experts who were still trying to work out careful ethical guidelines for editing human embryos.He Jiankui has shot this measured approach to pieces with his announcement, and now the rest of the scientific community is on the back foot, trying to reassure the public and to create consensus rapidly, Carey writes.

Hacking the Code of Life doesnt break much new ground, and for readers who have been paying attention to Crispr over the past few years, little in the book will come as a surprise. But it does providea broad, even-handed overview of how much has already happened in a field that is less than 10 years old.

Carey swats down the most dystopian dreams about Crispr, like the prospect that criminals might edit their own DNA to evade justice. Shes similarly skeptical that well end up using the technology to create super-beings with enhanced genomes that will make them taller, faster, more attractive.

We actually understand very little about the genetic basis of these traits and what we do know suggests that it will be very difficult to enhance humans in this way, she writes.

But she also acknowledges real risks, including the possibility that the technique could be used to create dangerous bioweapons, that gene-edited organisms could destabilize natural ecosystems,and that our new, hardy crops could prompt us to convert even more of the Earths undeveloped places into farmland.

None of this means that the technology should be abandoned; it hasimmense potential to improve our lives, as the book makes clear.But it does mean we need to proceed with caution. As Carey writes, Ideally, ethics should not be dragged along in the wake of scientific advances; the two should progress together, informing one another.

Emily Anthes, who has written for Undark, The New York Times, The New Yorker, Wired, and Scientific American, among other publications, is the author of the forthcoming book The Great Indoors. Follow her on Twitter @EmilyAnthes

A version of this article was originally published on Undarks website as How Gene Editing Is Changing the World and has been republished here with permission.

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'Hacking the Code of Life': How gene editing will lead to disease cures and nutritionally enhanced food - Genetic Literacy Project

Stem cell therapy for animals has seen breakthroughs

Stem cell therapy is increasingly becoming a more mainstream form of medicine. Usually applied to humans, the use of this regenerative treatment is now also being extended to animals including cats and dogs. Regenerative medicine, particularly stem cell treatment has seen many advancements in recent years with some groundbreaking studies coming to light.

Taking the cells from bone marrow, umbilical cords, blood or fat, stem cells can grow to become any kind of cell and the treatment has seen many successes in animals. The regenerative therapy has been useful particularly for treatment of spinal cord and bone injuries as well as problems with tendons, ligaments and joints.

Expanded Potential Stem Cells (EPSCs) have been obtained from pig embryos for the first time. The cells offer groundbreaking potential for studying embryonic development and producing transnational research in genomics and regenerative medicine, biotechnology and agriculture.

The cells have been efficiently derived from pig preimplantation embryos and a new culture medium developed in Hong Kong and Cambridge enabled researchers from the FLI to establish permanent embryonic stem cell lines. The cells have been discovered in a collaboration between research groups from the Institute of Farm Animal Genetics at the Friedrich-Loeffler-Institut (FLI) in Mariensee, Germany, the Wellcome Trust Sanger Institute in Cambridge, UK and the University of Hong Kong, Li Ka Shing Faculty of Medicine, School of Biomedical Sciences.

Embryonic stem cells (ESC) are derived from the inner cells of very early embryos, the so-called blastocysts. Embryonic stem cells are all-rounders and can develop into various cell types of the body in the culture dish. This characteristic is called pluripotency. Previous attempts to establish pluripotent embryonic stem cell lines from farm animals such as pigs or cattle have resulted in cell lines that have not really fulfilled all properties of pluripotency and were therefore called ES-like.

Dr Monika Nowak-Imialek of the FLI said: Our porcine EPSCs isolated from pig embryos are the first well-characterized cell lines worldwide. EPSCs great potential to develop into any type of cell provides important implications for developmental biology, regenerative medicine, organ transplantation, disease modelling and screening for drugs.

The stem cells can renew themselves meaning they can be kept in culture indefinitely, and also show the typical morphology and gene expression patterns of embryonic stem cells. Somatic cells have a limited lifespan, so these new stem cells are much better suited for long selection processes. It has been shown that these porcine stem cell lines can easily be modified with new genome editing techniques such as CRISPR/Cas, which is particularly interesting for the generation of porcine disease models.

The EPSCs have a high capacity to develop not only into numerous cell types of the organism, but also into extraembryonic tissue, the trophoblasts, making them very unique and lending them their name. This capacity could prove valuable for the future promising organoid technology, where organ-like small cell aggregations are grown in 3D aggregates that can be used for research into early embryo development, various disease models and testing of new drugs in petri dishes. In addition, the authors were able to show that trophoblast stem cells can be generated from their porcine stem cells, offering a unique possibility to investigate functions or diseases of the placenta in vitro.

A major hurdle to using neural stem cells derived from genetically different donors to replace damaged or destroyed tissues, such as in a spinal cord injury, has been the persistent rejection of the introduced material (cells), necessitating the use of complex drugs and techniques to suppress the hosts immune response.

Earlier this year, an international team led by scientists at University of California San Diego School of Medicine successfully grafted induced pluripotent stem cell (iPSC)-derived neural precursor cells back into the spinal cords of genetically identical adult pigs with no immunosuppression efforts. The grafted cells survived long-term, displayed differentiated functionality and caused no tumours.

The researchers also demonstrated that the same cells showed similar long-term survival in adult pigs with different genetic backgrounds after only short course use of immunosuppressive treatment once injected into injured spinal cord.

Senior author of the paper Martin Marsala, MD, professor in the Department of Anesthesiology at UC San Diego School of Medicine said: The promise of iPSCs is huge, but so too have been the challenges. In this study, weve demonstrated an alternate approach.

We took skin cells from an adult pig, an animal species with strong similarities to humans in spinal cord and central nervous system anatomy and function, reprogrammed them back to stem cells, then induced them to become neural precursor cells (NPCs), destined to become nerve cells. Because they are syngeneic genetically identical with the cell-graft recipient pig they are immunologically compatible. They grow and differentiate with no immunosuppression required.

Co-author Samuel Pfaff, PhD, professor and Howard Hughes Medical Institute Investigator at Salk Institute for Biological Studies, said: Using RNA sequencing and innovative bioinformatic methods to deconvolute the RNAs species-of-origin, the research team demonstrated that pig iPSC-derived neural precursors safely acquire the genetic characteristics of mature CNS tissue even after transplantation into rat brains.

NPCs were grafted into the spinal cords of syngeneic non-injured pigs with no immunosuppression finding that the cells survived and differentiated into neurons and supporting glial cells at all observed time points. The grafted neurons were detected functioning seven months after transplantation.

Then researchers grafted NPCs into genetically dissimilar pigs with chronic spinal cord injuries, followed by a transient four-week regimen of immunosuppression drugs again finding long-term cell survival and maturation.

Marsala continued: Our current experiments are focusing on generation and testing of clinical grade human iPSCs, which is the ultimate source of cells to be used in future clinical trials for treatment of spinal cord and central nervous system injuries in a syngeneic or allogeneic setting.

Because long-term post-grafting periods between one and two years are required to achieve a full grafted cells-induced treatment effect, the elimination of immunosuppressive treatment will substantially increase our chances in achieving more robust functional improvement in spinal trauma patients receiving iPSC-derived NPCs.

In our current clinical cell-replacement trials, immunosuppression is required to achieve the survival of allogeneic cell grafts. The elimination of immunosuppression requirement by using syngeneic cell grafts would represent a major step forward said co-author Joseph Ciacci, MD, a neurosurgeon at UC San Diego Health and professor of surgery at UC San Diego School of Medicine.

Other recent advancements include the advancement toward having a long-lasting repair caulk for blood vessels. A new method has been for generating endothelial cells, which make up the lining of blood vessels, from human induced pluripotent stem cells. When endothelial cells are surrounded by a supportive gel and implanted into mice with damaged blood vessels, they become part of the animals blood vessels, surviving for more than 10 months.

The research was carried out by stem cell researchers at Emory University School of Medicine and could form the basis of a treatment for peripheral artery disease, derived from a patients own cells.

Young-sup Yoon, MD, PhD, who led the team, said: We tried several different gels before finding the best one. This is the part that is my dream come true: the endothelial cells are really contributing to endogenous vessels.

When cells are implanted on their own, many of them die quickly, and the main therapeutic benefits are from growth factors they secrete. When these endothelial cells are delivered in a gel, they are protected. It takes several weeks for most of them to migrate to vessels and incorporate into them.

Other groups had done this type of thing before, but the main point is that all of the culture components we used would be compatible with clinical applications.

This research is particularly successful as previous attempts to achieve the same effect elsewhere had implanted cells lasting only a few days to weeks, using mostly adult stem cells, such as mesenchymal stem cells or endothelial progenitor cells. The scientists also designed a gel to mimic the supportive effects of the extracellular matrix. When encapsulated by the gel, cells could survive oxidative stress inflicted by hydrogen peroxide that killed unprotected cells. The gel is biodegradable, disappearing over the course of several weeks.

The scientists tested the effects of the encapsulated cells by injecting them into mice with hindlimb ischemia (restricted blood flow in the leg), a model of peripheral artery disease.

After 4 weeks, the density of blood vessels was highest in mice implanted with gel-encapsulated endothelial cells. The mice were nude, meaning genetically immunodeficient, facilitating acceptance of human cells.

The scientists found that implanted cells produce pro-angiogenic and vasculogenic growth factors. In addition, protection by the gel augmented and prolonged the cells ability to contribute directly to blood vessels. To visualise the implanted cells, they were labelled beforehand with a red dye, while functioning blood vessels were labelled by infusing a green dye into living animals. Implanted cells incorporated into vessels, with the highest degree of incorporation occurring at 10 months.

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Stem cell therapy is for animals too - SciTech Europa

Hematopoietic stem cell transplantation (HSCT), popularly known as bone marrow transplantation (BMT), is a curative modality for a number of benign and malignant blood disorders, said Dr. Murali Krishna Voonna, surgical oncologist and managing director of Mahatma Gandhi Cancer Hospital and Research Institute.

Speaking at an awareness programme on stem cell donation organised here by the hospital, in association with Datri Blood Stem Cell Donor Registry, he said hematopoietic stem cells are immature cells that can develop into all types of blood cellswhite blood cells, red blood cells, and platelets. They are found in the peripheral blood and bone marrow.

A sizeable population are diagnosed to have benign diseases such as thalassemia major, sickle cell anaemia and aplastic anaemia, and the HSCT is among the efficient curative measures. Acute leukaemia and other blood cancers also need this procedure, he said.

Highlighting that stem cell donation and a registry are vital, Dr. Muralikrishna explained for a successful hematopoietic stem cell transplant, the patients genetic typing (HLA typing) needs a close match with that of the donor. Every patient has 25% chance of finding a match within the family, he said.

Dr. Muralikrishna stated that in such cases, finding a donor is a pressing need. There are over 80 donor registries and more than 30 million registered donors across the globe, with a very few Indians being a part of it. This reduces the chances of finding a possible match for patients of Indian origin. Patients are more likely to find a possible match within their ethnicity, which means people sharing the same cultural linguistic and biological traits, he explained.

The problem can be solved if the donors enroll themselves with a registry which will store the stem cell details and the details. Pledging to donate stem cells is easy like swabbing the inner-cheek. The donors are contacted if patients have HLA matching, he said, adding that the stem cell donation was carried out only when a match was found for a patient, not when one pledge to donate.

A blood stem cell collection centre was inaugurated at the hospitals premises on the occasion. Earlier, to avail of such service and for HLA-typing, one has travel to Hyderabad and Chennai, Dr. Muralikrishna said.

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More awareness needed on stem cell donation: expert - The Hindu

Bhubaneswar: The advanced treatment of using stem cells for treating Autism and other neurological ailments have come as a ray of hope for the people living with some of these ailments. Medical experts working in the sector claim that the use of the technology improved the lives of many.

According to experts who practice stem cell therapy, the results have been overwhelming. Many of the patients have either been able to fight a deadly disease with the help of stem cells while many have been able to improve their quality of lives by using it. However, the technology is still not used widely in state hospitals.

Medical experts claim that stem cells could be used to treat neurological disorders like Autism, cerebral palsy, mental retardation, brain stroke, muscular dystrophy, spinal cord injury, head injury, cerebellar ataxia, dementia, motor neurone disease, multiple sclerosis while it has also been used to treat cancers like blood cancer with the help of bone marrow transplant when assisted by stem cell therapy.

However, treatment of Autism with stem cells is a new developing sector where visible changes are said to have been reported among children treated with this technology. However, the advanced technology which is now confined to only private sector is a bit expensive.

Autistic kids are usually treated with drugs for symptomatic relief, special education, occupational speech and behavioural therapies. In Autism, despite the best available medical and rehabilitative treatments satisfactory relief is still a far cry, said Dr Nandini Gokulchandran, Head Medical Services, NeuroGen Brain and Spine Institute, Mumbai.

Dr Gokulchandran claims that she has treated many cases of Autism in kids with stem cells which helped in overcoming their limited abilities. Under the treatment regime, an insertion procedure is undertaken followed by training to improve the skills and abilities of autistic kids.

Another neurologist, Dr Richa Bansod said that in India it has been reported that 1 in every 250 children have Autism and this number in increasing with better recognition and awareness of the condition. On the other hand, stem cells are now been used to fight deadly diseases.

Dr Joydeep Chakaborty, an oncologist and stem cell expert from HCG Cancer Hospital, Kolkata said, Stem cells and bone marrow transplants are now being used to cure blood cancer in many cases. It is also widely used to treat blood disorders like Thalassemia, Sickle Cell Anaemia and others.

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Stem cells treatment gives hope in fighting Autism, blood disorders - OrissaPOST

For anyone who has had hip replacement surgery, Im sure they will agree that it is better to get hit by a bus than to undergo another one. Last year after several years of suffering, I decided to take the leap and go for the hip replacement that my specialist recommended. I was told that it was a common surgery and that it was the best solution for me. Between us; it was probably the most painful thing I have ever gone through. So much so, that at the time, I just wanted to die. Not only did the pain persist for several weeks after the operation, but I was on painkillers for days, which eventually added to my suffering. I had to use a walker for the first 2 weeks and then depended on a cane for over 2 months before I could walk on my own.

My entire demeanor changed, as well as the way I dealt with what once were minor things in life. I feared slipping in the shower, going down the stairs or walking my dogs. No one had prepared me for this. Ive had my share of surgeries including a double mastectomy when I was diagnosed with breast cancer but pain wise; this one was by far the worse. I was hoping after a very long recovery that I would never have to face this situation again. Unfortunately, a year later, I am starting to feel pain on the other side and dread the re-experience of my nightmare.

Although, I heard about Stem Cell, I did not know much about it. So I started to investigate for myself, speak to people, enquire about the procedure and look for a doctor in my area who specialized in Stem Cell. I was willing to do just about anything before considering another hip replacement. After extensive research, I came across Dr. Raj, a Double-Board Certified Orthopedic doctor in Beverly Hills, CA. Going to his website; I learned that he has been in private practice for 10 years. He has been named as one of Americas Top Orthopedists, been featured on the Best of LA and has received numerous other accolades and awards as one of the Top Orthopedic doctors. Providing the ultimate in state-of-the-art orthopedic care, Dr. Rajs practice is always on the cutting-edge of surgical and nonsurgical technologies, such as PRP (Platelet Rich Plasma) injections, stem cell injections for tendinitis and arthritis, minimally invasive surgery and more.

He is Board Certified as a Medical Legal Specialist in America, as well as, Canada and Dubai (Trial, Testimony, Deposition, IME) with a Subspecialty in Hip and Knee Surgery in Los Angeles, including Sports Surgeries.

He is also an Undergraduate from Dalhousie University in Halifax and Canada. He pursued his medical education at Memorial University PGME, before doing his internship and residency in the Department of Orthopedic Surgery. Now that I had found Dr. Raj, all I needed was to get myself educated. So lets start by what are stem cells? This is what I read: Mesenchymal stem cells (MSCs), commonly called stem cells, are precursor cells that havent decided yet what they are going to be in the body. They can differentiate into multiple forms including bone, cartilage, fat and other connective tissues. They play a significant role in the reparative processes throughout the human body.

Where do we find stem cells?

They may be harnessed from fat tissue, bone marrow, synovial tissue or umbilical cord tissue. While stem cell therapy is a promising technology, there is much we are still learning about the causes and pathways that lead to symptomatic osteoarthritis. We have not optimized the factors found in stem cell therapies. To be sure, only the good cells and growth factors are injected into a specific joint. And that is why further research is necessary before being approved by the FDA.

My next move would be to consult with Dr. Raj who would tell me the medical truth, beginning with this question:

What is the current state of Stem Cells and its success rate?

It's relatively new. It's been popular for about 20 years, internationally. In areas like Germany and Korea, it was utilized a lot more. It became popular here when athletes like Kobe Bryant started going to Germany for modified versions of PRP, which led on to regenerative technologies. We have a stigma correlating stem cells with abortions and issues like that. This in itself is completely different. We are not utilizing amniotic stem cells or placenta stem cells. We're utilizing your own stem cells. For issues such as a hip replacement, the most powerful stem cells are the ones in your body. Bone marrow stem cells work well on joints. Joints have zero blood supply. So, if God or the higher power created us where we had blood supply going through our joints, like a cut in our skin - we would constantly replenish or repair. A break in our bone would repair. If you get stem cells and you're in decent enough shape, you will heal no matter what because these stem cells will deposit. Will you heal straight? Probably not - that's where we come into play.

The reason why joints; hips, knees and shoulders degenerate is because there is no blood supply. So, if you have a cut or a loss of cartilage, it stays like that and accumulates overtime. The only way you can control it is externally. You get stronger, you lose weight and you increase your range of motion. But you can't control anything internally.

So regenerative technology is basically utilizing these cells to regenerate cartilage and repair. These are the same cells that flow through our body - and upon signal of an injury will heal skin to skin, bone to bone, tendon to tendon, muscle to muscle. Our joints are just an alcove of joint fluid and no blood supply. The whole concept is - throughout the years, we did steroid injections - they're like band aids. Basically they mask pain. What does masking pain do? It propagates injury. Because we put the band aid on, we don't feel it and we do more. We take this little cut or loss of cartilage and we make it even more over time.

Why is it that specialists do not recommend seeing a surgeon at a certain stage?

There are a lot of people who think one way and everyone is entitled to their own opinions. You can't change opinions.

Are people afraid of stem cells?

Some people are afraid because of stem cells causing cancer. But that's embryonic stem cells.

What is the process?

Bone marrow stem cells are the best because there is a higher chance of live stem cells. Less manipulation, meaning that - in a Mayo Clinic study 4 or 5 years ago, which has a two year follow through on people who are ready to get replacements for joint or knee - they had an 80% success rate where they didn't need it. I do replacements and I do stem cells.

How do you determine what's better for the patient?

My knowledge and years of experience. Also, my knowledge with fitness and being athletic myself. Understanding at a certain point, someone is mechanically compromised. Bone on bone is a term that's been used for years. There are a lot of people who think they are 'bone on bone." Coming from Canada, the US is notorious for doing unnecessary surgeries and replacements. It's the highest rate of replacements in the world. I do not like the term 'bone on bone' because a surgeon will look at an x-ray and say you're bone on bone because that's all they do: replacements. They become a 7-11 or 99 Cents store, lining up 21 people a day. That's not the right way to do things. You don't want to be one of those 21 people getting a replacement because you're not getting that surgeon's full attention. The reality is - you have a PA or an old plastic surgeon who's doing most of your surgery and there is more likelihood of issues. Amongst every specialty there is a lot of ignorance. The whole concept is - you preserve what you have for as long as you can. You have beauty on the outside; you need beauty on the inside too. What's beauty on the inside? Feeling good, you're less inflamed and your joints are healthy.

How does it work with a stem cell procedure?

I extract bone marrow from your pelvis. Take approximately 6 ccs. Under slight sedation, it takes about 5 minutes to take it. Then we separate it via an FDA approved technique. Per FDA, we cannot add anything to it, nor would I want to. We cannot harvest it because the longer it's outside of the body, the better it is. Basically, we then inject those pure cells right away into the joint. It's a four month process for an 80% of regeneration. So, it's not just reduction of inflammation, it's regeneration. It will be a year for a 100% effect. I've had probably about 20% of patients who have taken 6 months+. I've had over a 95% success rate with this technology.

Are you one of the only doctors doing this in LA?

I'm one of them. There are some family and pain management doctors who are doing it. I'm the only Orthopedic surgeon doing it. I'm sure different practitioners are starting to.

Dr. Raj and patient Paula Abdul

How often do you do the stem cell procedure?

You do it one time. It's a powerful injection and there are people Ihave 6 years out who are doing well.

Does it hurt after the fact?

No, not at all. You can walk and move. For example, with your hip - I would combine it with physical therapy to increase your range of motion. Once you have the anti-inflammatory effect, you have to take advantage of it. If you don't increase your range of motion - what happens is - you're walking on one nail vs. 100 nails. You want to dissipate the force over a greater area so that there's a higher chance of external success. Then you strengthen the muscles.

Are there people who are not good candidates for it?

Yes, when it's too far gone. Like I said, people are told they're bone on bone when they're not. They show you different views. It's a marketing gimmick. That person is lined up and ready to sell. Age is relative. There's physiologic age. It really depends on the person. Hypothetically, if you're an inflamed mess, a drinker and abusive to your body, then nothing is going to work. If you take care of yourself and you're motivated with the right protoplasm, then it's going to work.

What about the skeptics or the ones who think it's bad for you?

Don't get me wrong; amniotic stem cells are good for certain situations. Embryonic is bad. It means that it's too far gone. You want live stem cells in an area that does not have blood supply. The data is out there. How can you argue against a Mayo Clinic study with an 80% success rate? How can you argue against the hospitals for special surgery in New York that's doing it, or the Steadman Hawkins Clinic, I'm doing it. Top facilities in the world are doing it and a number of top athletes who are getting it done with success rates. Who's ignorant? Is it that one surgeon or everyone else?

Does insurance cover it?

No, not yet. Insurances are very backwards in terms of their understanding. They would rather cover a replacement.

Is it expensive?

If you break it down par and par and avoid a replacement, not really. On average, you're talking about $7,000, versus hospital, surgeon, facility fees+++,which can be about $25,000.

You're very progressive.

There are a lot of things that I do to try and reduce pain significantly.When I use screws, I use screws that are made out of calcium so they dissolve in your body. Some of my colleagues use tourniquet, I don't use one. I control bleeding and do it in less than an hour. The whole concept is, you don't have atourniquetsqueezing your leg and toxins causing significant pain.

And there you have it. Everything is a risk in life, we do not know if we will wake up tomorrow or if you will get hit by a car and so on so why not try this procedure. I believe that I am lucky enough to have met Dr. Raj. I have taken the decision to undergo the stem cells therapy FDA approved or not, anything before going under the knife one more time. Stay tuned, I will give you a report on the progress.

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Dr. Raj & Stem Cell Therapy Innovation - LATF USA

Eighteen-year-old Aisha Choudhary was just like any other adolescent eyes filled with dreams and a heart brimming with energy. The only difference was she was battling a rare genetic disease, Severe Combined Immune Deficiency (SCID). Diagnosed when she was six months old and undergoing medical treatment for years, she was iron-willed in playing the cards she was dealt.

Since one of the most effective cures for SCID is a stem cell transplant (grafting of the parent cells from which all blood cells develop), Aishas parents, Niren and Aditi, decided to opt for that treatment mode. But their cells were not a complete match with their daughters, and they had to look at external donors. However, due to a low number of voluntary, registered stem cell donors, Aisha could not get a compatible donor whose genetic markers were a close enough match to hers. With no other alternative treatment available, Aisha had a bone marrow transplant. But, it came with a side-effect that cost her life Pulmonary Fibrosis, a disease known to damage the lung tissues.

Aishas Choudhary's role has been played by Zaira Wasim in The Sky is Pink.

Aishas journey has been captured in The Sky is Pink, a recent Bollywood movie starring Priyanka Chopra, Farhan Akhtar, Zaira Wasim, and Rohit Saraf.

The 18-year-olds life story is mirrored in the experiences of many who await stem cell donation as treatment for blood-related illnesses likeleukemia, lymphoma, and sickle cell anemia every year. With very few individuals signing up as donors and the probability of finding a match being a dismal 0.0008 percent in India (against a lean 16 percent abroad), fatalities are mounting year on year.

However, in recent times, there has been one small break in the clouds a number of youngsters, non-governmental organisations, and medical professionals have come forward and are working to spread awareness about stem cell donation and motivate a larger number of people to register as donors.

The stem cells in a human body mainly comprise red blood cells, platelets, and white blood cells. These are found in the umbilical cord of newborns and in the peripheral or circulating blood and bone marrow.

A stem cell donation is as simple and painless as a blood donation.

Certain diseases like blood cancer and leukemia tend to destroy the bone marrow or affect its functioning.For these, treatments like chemotherapy and radiotherapy are tried initially. However, in some cases, they do not prove effective for a cure. The only recourse then is replacing the patients stem cells with those of a healthy person.

One of the main criteria for a successful transplant is a good match between the stem cells of the donor and those of the patient. Therefore, a donor registry will administer a cheek swab test (tissue samples extracted from the cheek) on all potential donors to match cell characteristics. This procedure of pairing generic markers is called Human Leukocyte Antigen (HLA) in medical terms.

A cheek swab test in progress.

Each potential donors tissue is entered in the registry and given an identification number after the test is done. If the registry finds a match at any point in time, the donor is contacted to initiate the transplant.

There are many organisations today that are leading the charge in saving the lives of people suffering from serious blood disorders like cancer, thalassemia, and anaemia.

For instance, Datri, an Ahmedabad-based NGO, is working to create a wide and diverse database of potential stem cell donors by organising donation drives. Founded in 2009 by two doctors and an engineer, the organisation focuses on conducting awareness campaigns and helping individuals sign up on its registry as a committed and voluntary benefactor.

The team of the NGO Datri.

The idea for Datri was initially born in the minds of doctors Nezih Cereb and Soo Young Yang, who run a laboratory, Histogenetics, for determining tissue matches between patients and donors. Since pairing tissue types is imperative for any stem cell transplant, and confronting a severe shortage of donors, the doctor duo would run from pillar to post to meet hospitals requirements. Working with a number of the hospitals in India, they realised just how acute the shortfall was in people willing to donate stem cells. They recognised the immediate need to create a donor registry here.

Soon after, Raghu Rajagopal, an engineer from BITS Pilani and Director of ready-to-eat venture Millets and More, connected with them and they decided to start Datri.

Today, the functioning of the registry, its maintenance, and even the substantial costs involved in conducting the HLA matching are taken care of by the lab. In the last 10 years, Datri has gotten over four lakh people to register as donors and has saved around 600 lives through successful transplantation.

Every day, about 40 people are diagnosed with blood disorders in India. Though these can be cured through a stem cell transplant from a genetically matched donor, there is only a 25 percent chance of finding a match from within the family. Others have no option but to rely on unrelated donors. But the chances of getting a match is anywhere between one in 10,000 and one in two million. There is an urgent need to rope in as many potential donors as possible, which is precisely what Datri is trying to do, Raghu explains.

Another organisation that is dedicated to fighting blood disorders with stem cell treatment is DKMS-BMST. It was formed through a joint venture between two renowned NGOs DKMS, which is one of the largest international blood stem cell donor centres globally, and the Bangalore Medical Services Trust (BMST).

The team of DKMS-BMST.

DKMS was founded in Germany in 1991 by businessman Dr Peter Harf, after he lost his wife to leukemia. BMST was born in 1984 from the vision of Dr Latha Jagannathan, a medical director and managing trustee. Since both organisations had a common goal to find a matching donor for every patient with a blood disorder, they decided to come together to achieve it.

A group of youngsters registering to be stem cell donors.

So far, more than 37,000 people in India have registered as potential donors after attending DKMS-BMSTs donor drives.

In highly populous countries like India, thousands of people are in need of stem cell transplants every year to survive. Though donating stem cells is a painless and non-invasive process, it remains a lesser-known medical concept in India, with only 3.6 lakh people willing to play a part in it. Besides, the chances of stem cells of people of the same ethnicity matching are higher than those of individuals from different ethnic backgrounds. But, it is due to sheer lack of awareness that India lags severely in stem cell donations, say experts.

Students taking a cheek swab test at one of the colleges in Bengaluru.

Dr Govind Eriat, a reputed hematologist and bone marrow transplant specialist, says,

With a major hurdle to stem cell donation in India proving to be the myths surrounding the subject, the youth are coming forward to deconstruct common misconceptions.

For instance, 21-year-old Tejaswini Patel, a student of Information Science at New Horizon College of Engineering, Bengaluru, has been busting the false ideas on stem cell donation, starting among her family and friends. She says,

She adds, with a notable sense of pride, In the last two years alone, around 400 students from my college have registered themselves as donors.

(Edited by Athirupa Geetha Manichandar)

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How Young India is fuelling the future of stem cell therapy and signing up to save lives - YourStory

Stories of using the little blue pill for bone marrow transplants, how pregnancy stress is related to the baby's sex, and a vaccine for breast cancer proliferated on the Internet this week. Here's why you didn't read about them on Medscape.

Researchers at the University of California, Santa Cruz, seem to think Viagra has more to offer in medicine. In a recent study of mice, they tested whether the vasodilator couldspeed up the migration of hematopoietic stem cells and progenitor stem cells from the bone to the blood, where the cells could be harvested noninvasively.

The standard protocol for preparing bone marrow donors for the harvesting procedure, a 5-day regimen of granulocyte-colony stimulating factor (G-CSF),is "complex, costly, unsuccessful in a significant proportion of donors," the study authors write, and typically results in fatigue, nausea, and bone pain. Using a two-drug strategy, oral Viagra and a single injection of the CXCR4 antagonist AMD3100 (plerixafor), elicited the same mobilization of stem cells in 2 hours.

We didn't cover the study because it's still too early to say whether this strategy might be effective in people. After this mouse study, the next step is testing the approach in larger animals before human clinical trials.

A study of 187 healthy pregnant women age 18 to 45 years suggests that preterm mental and physical stress may be related to the baby's sex and increase the risk for preterm birth. In the study, 16% of women were physically stressed, as measured by higher blood pressure and calorie intake; and 17% were mentally stressed with high levels of depressionand anxiety; 66% of the women were in the healthy (nonstressed) group.

Women who were stressed during pregnancy were more likely to give birth to a girl. Typically, 105 males are born for every 100 females, but the study authors found that the male-to-female ratio decreased to 2:3 in psychologically stressed patients and 4:9 in physically stressed patients. Physically stressed mothers also gave birth an average of 1.5 weeks earlier than mothers in the healthy group, with 22% giving birth preterm compared with 5% in the healthy group.

The study authors say the findings demonstrate the importance of maternal mental health. Medscape has covered the consequences of maternal stress extensively, including preterm birth, neurobehavioral risks, and potential links to hyperactivity during the offspring's teen years. However, the sample size in this study was small: the mentally and physically stressed groups combined only included about 60 women. That's not sufficient to inform clinical practice in counseling women who want to get pregnant about how stress may affect the sex of their baby, so we didn't cover it.

News spread this week that Floridian Lee Mercker became the first woman to "beat" breast cancer with the help of a new vaccine. The vaccine, which stimulates the immune system to fight off early-stage breast cancer, was developed and administered by researchers at the Mayo Clinic in Jacksonville, Florida. The vaccine is currently in an early trial.

Reports of Mercker's success raise hopes, but she's reportedly the first participant in the trial. The news report also says she underwent a double mastectomy after her diagnosis in March, so it's unclear what evidence of the vaccine's efficacy the researchers measured. Before this experimental vaccine is relevant to Medscape readers, we need to see additional detailed data from more patients in the clinical trial published in a peer-reviewed journal.

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The Week That Wasn't: Viagra BMTs, Pregnancy Stress, Breast Cancer Vaccine - Medscape

150,000 POTENTIAL STEM CELL DONORS ARE GIVING BLOOD CANCER PATIENTS HOPE THANKS TO ONE UNIVERSITY SOCIETY -1 in 4 stem cell donors are now recruited by Marrow university societies

Students from over 50 universities across the UK have helped blood cancer charity Anthony Nolan recruit an incredible 150,000 students to the Anthony Nolan stem cell register, since the first Marrow group was created 21 years ago.

Marrow is the name given to blood cancer charity Anthony Nolans network of student volunteer groups.

The first Marrow society was created at the University of Nottingham, with the aim of recruiting students to the Anthony Nolan stem cell register. For many people with blood cancers or blood disorders, receiving stem cells from a stranger is their best chance of survival.

Research has found that younger donors are more likely to save the lives of patients, so the work done by Marrow is invaluable. Over a quarter of all stem cell donations that have occurred in the last two years were from donors recruited by Marrow. University students across the country are continually giving people with blood cancer and blood disorders a second chance of life.

Liam Du Ross, 24, from North Wales is a research chemist and signed up to the Anthony Nolan register in September 2014, while at Bangor University.

Liam said: I was at my university freshers fair and stopped to talk to the volunteers running the Marrow stall. I wanted to help someone in need, and I had already signed up to donate blood at this point, so the Anthony Nolan stem cell register seemed like the next step.

Earlier this year Liam received a call to say that he had been found to be a match for someone in desperate need of a stem cell transplant.

When I found out that I was a match for someone, I felt really lucky. I had absolutely no doubts about going through with the donation at all, the whole experience was a pleasure. The nurses involved in the process were exceptional, and they helped to put me at ease. I donated via PBSC (peripheral blood stem cell collection) so I was able to lie there and catch up on podcasts and TV shows!

I thought about my recipient a lot during my donation and how I would feel if I were in their situation. I would love to meet them one day and I hope they feel the same.

To anyone thinking of signing up to the register, I would say that you should absolutely sign up. If someone you knew was that person who needed a transplant, you'd want to doeverythingin your power to help them.

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Shaswath Ganapathi, 21, is a 4th year medical student at Birmingham University and the secretary of Birmingham Marrow. He decided to volunteer with Marrow after his friend, Rohan, sadly died from leukaemia last year. Shaswath and the other committee members hold events across their university, where they encourage students to sign up to the Anthony Nolan stem cell register, any of whom could go on to donate their stem cells in the future.

Shaswath said: The donors I have spoken to have said that its the most life changing thing they have ever done, and they would never have thought that spending a few minutes signing up at a stand and doing a quick cheek swab could lead to potentially saving someones life.

Aisling Cohn, Youth Programmes Manager at Anthony Nolan, said: Marrow really are the unsung heroes helping Anthony Nolan give hope to patients with blood cancer, by signing up an incredible number of potential donors to the stem cell register. Any one of these people could save the life of someone with blood cancer.

It costs 40 to add each new person to the Anthony Nolan register, any money raised by Marrow will directly help save lives. They really are lifesavers!

If a patient has a condition that affects their bone marrow or blood, then a stem cell transplant may be their best chance of survival. Doctors will give new, healthy stem cells to the patient via their bloodstream, where they begin to grow and create healthy red blood cells, white blood cells and platelets.

Marrow

Key statistics

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Students from over 50 universities across the UK help blood cancer charity - FE News