First patient with metastatic triple-negative breast cancer (mTNBC) continues to show no detectable circulating tumor cells (CTC) or putative metastatic tumor cells after 15 weeks of treatment with leronlimab in combination with carboplatin

Second patient with stage 4 HER2+ metastatic breast cancer (MBC) shows 50 percent shrinkage in the primary tumor and no new signs of metastasis in the brain after treatment with leronlimab as a monotherapy

VANCOUVER, Washington, Jan. 22, 2020 (GLOBE NEWSWIRE) -- CytoDyn Inc. (OTC.QB: CYDY), ("CytoDyn" or the "Company"), a late-stage biotechnology company developing leronlimab (PRO 140), a CCR5 antagonist with the potential for multiple therapeutic indications, announced today additional promising results from its clinical trials evaluating leronlimab for the treatment of metastatic triple-negative breast cancer (mTNBC) and metastatic breast cancer (MBC).

New data from the first patient enrolled in the Company's metastatic triple-negative breast (mTNBC) Phase 1b/2 trial continues to show no detectable levels of circulating tumor cells (CTC) or putative metastatic cells in the peripheral blood following 15 weeks of treatment with leronlimab in combination with carboplatin.

The second patient, enrolled through an emergency investigational new drug (IND) with stage 4 HER2+ MBC that has metastasized to the liver, lung and brain, showed a 50 percent shrinkage of the primary tumor and no new metastasis in the brain after treatment with leronlimab as a monotherapy. The patient was previously treated with pertuzumab and trastuzumab for over a year and a half. This patient has been taking leronlimab since November 25, 2019 with one 700 mg dose each week.

"Recent testing of the first patient demonstrated continued absence of CTCs in all blood tubes with only one cancer-associated macrophage like cells (CAMLs) in one of two tubes. In the second patient, the follow up brain scan conducted on January 17, 2020, showed that the largest brain lesion had a greater than 4-fold reduction in size," said Bruce Patterson, M.D., chief executive officer and founder of IncellDx, a diagnostic partner and an advisor to CytoDyn. "Other smaller lesions on the second patient's brain have not changed in size and cerebral edema remains at decreased levels since the last imaging studies. Taken together, these results suggest continued response of both primary and metastatic tumors to treatment with leronlimab for both the first and second patient."

Nader Pourhassan, Ph.D., president and chief executive officer of CytoDyn, added: "We are thrilled to see these additional data that further support leronlimab as a potential treatment option for patients with mTNBC and MBC. As a Company, we look forward to continuing our work in the oncology space and developing a potentially safe and effective treatment option for patients suffering from this deadly disease."

About Triple-Negative Breast CancerTriple-negative breast cancer (TNBC) is a type of breast cancer characterized by the absence of the three most common types of receptors in the cancer tumor known to fuel most breast cancer growthestrogen receptors (ER), progesterone receptors (PR) and the hormone epidermal growth factor receptor 2 (HER-2) gene.1TNBC cancer occurs in about 10 to 20 percent of diagnosed breast cancers and can be more aggressive and more likely to spread and recur.2,3Since the triple negative tumor cells lack these receptors, common treatments for breast cancer such as hormone therapy and drugs that target estrogen, progesterone, and HER-2 are ineffective.4Currently, there are no targeted therapies approved to treat triple negative breast cancer.5

About Leronlimab (PRO 140)The U.S. Food and Drug Administration (FDA) have granted a "Fast Track" designation to CytoDyn for two potential indications of leronlimab for deadly diseases. The first as a combination therapy with HAART for HIV-infected patients and the second is for metastatic triple-negative breast cancer. Leronlimab is an investigational humanized IgG4 mAb that blocks CCR5, a cellular receptor that is important in HIV infection, tumor metastases, and other diseases including NASH. Leronlimab has successfully completed nine clinical trials in over 800 people, including meeting its primary endpoints in a pivotal Phase 3 trial (leronlimab in combination with standard anti-retroviral therapies in HIV-infected treatment-experienced patients).

In the setting of HIV/AIDS, leronlimab is a viral-entry inhibitor; it masks CCR5, thus protecting healthy T cells from viral infection by blocking the predominant HIV (R5) subtype from entering those cells. Leronlimab has been the subject of nine clinical trials, each of which demonstrated that leronlimab can significantly reduce or control HIV viral load in humans. The leronlimab antibody appears to be a powerful antiviral agent leading to potentially fewer side effects and less frequent dosing requirements compared with daily drug therapies currently in use.

In the setting of cancer, research has shown that CCR5 plays an important role in tumor invasion and metastasis. Increased CCR5 expression is an indicator of disease status in several cancers. Published studies have shown that blocking CCR5 can reduce tumor metastases in laboratory and animal models of aggressive breast and prostate cancer. Leronlimab reduced human breast cancer metastasis by more than 98% in a murine xenograft model. CytoDyn is therefore conducting aPhase 2 human clinical trial in metastatic triple-negative breast cancer and was granted Fast Track designation in May 2019. Additional research is being conducted with leronlimab in the setting of cancer and NASH with plans to conduct additionalclinical studies when appropriate.

The CCR5 receptor appears to play a central role in modulating immune cell trafficking to sites of inflammation and may be important in the development of acute graft-versus-host disease (GvHD) and other inflammatory conditions. Clinical studies by others further support the concept that blocking CCR5 using a chemical inhibitor can reduce the clinical impact of acute GvHD without significantly affecting the engraftment of transplanted bone marrow stem cells. CytoDyn is currently conducting a Phase 2 clinical study with leronlimab to further support the concept that the CCR5 receptor on engrafted cells is critical for the development of acute GvHD and that blocking this receptor from recognizing certain immune signaling molecules is a viable approach to mitigating acute GvHD. The FDA has granted "orphan drug" designation to leronlimab for the prevention of GvHD.

About CytoDynCytoDyn is a biotechnology company developing innovative treatments for multiple therapeutic indications based on leronlimab, a novel humanized monoclonal antibody targeting the CCR5 receptor. CCR5 appears to play a key role in the ability of HIV to enter and infect healthy T-cells. The CCR5 receptor also appears to be implicated in tumor metastasis and in immune-mediated illnesses, such as GvHD and NASH. CytoDyn has successfully completed a Phase 3 pivotal trial with leronlimab in combination with standard anti-retroviral therapies in HIV-infected treatment-experienced patients. CytoDyn plans to seek FDA approval for leronlimab in combination therapy and plans to complete the filing of a Biologics License Application (BLA) in 2019 for that indication. CytoDyn is also conducting a Phase 3 investigative trial with leronlimab (PRO 140) as a once-weekly monotherapy for HIV-infected patients and, plans to initiate a registration-directed study of leronlimab monotherapy indication, which if successful, could support a label extension. Clinical results to date from multiple trials have shown that leronlimab (PRO 140) can significantly reduce viral burden in people infected with HIV with no reported drug-related serious adverse events (SAEs). Moreover, results from a Phase 2b clinical trial demonstrated that leronlimab monotherapy can prevent viral escape in HIV-infected patients, with some patients on leronlimab monotherapy remaining virally suppressed for more than four years. CytoDyn is also conducting a Phase 2 trial to evaluate leronlimab for the prevention of GvHD and a Phase 1b/2 clinical trial with leronlimab in metastatic triple-negative breast cancer. More information is at http://www.cytodyn.com.

Forward-Looking StatementsThis press releasecontains certain forward-looking statements that involve risks, uncertainties and assumptions that are difficult to predict. Words and expressions reflecting optimism, satisfaction or disappointment with current prospects, as well as words such as "believes," "hopes," "intends," "estimates," "expects," "projects," "plans," "anticipates" and variations thereof, or the use of future tense, identify forward-looking statements, but their absence does not mean that a statement is not forward-looking. The Company's forward-looking statements are not guarantees of performance, and actual results could vary materially from those contained in or expressed by such statements due to risks and uncertainties including: (i)the sufficiency of the Company's cash position, (ii)the Company's ability to raise additional capital to fund its operations, (iii) the Company's ability to meet its debt obligations, if any, (iv)the Company's ability to enter into partnership or licensing arrangements with third parties, (v)the Company's ability to identify patients to enroll in its clinical trials in a timely fashion, (vi)the Company's ability to achieve approval of a marketable product, (vii)the design, implementation and conduct of the Company's clinical trials, (viii)the results of the Company's clinical trials, including the possibility of unfavorable clinical trial results, (ix)the market for, and marketability of, any product that is approved, (x)the existence or development of vaccines, drugs, or other treatments that are viewed by medical professionals or patients as superior to the Company's products, (xi)regulatory initiatives, compliance with governmental regulations and the regulatory approval process, (xii)general economic and business conditions, (xiii)changes in foreign, political, and social conditions, and (xiv)various other matters, many of which are beyond the Company's control. The Company urges investors to consider specifically the various risk factors identified in its most recent Form10-K, and any risk factors or cautionary statements included in any subsequent Form10-Q or Form8-K, filed with the Securities and Exchange Commission. Except as required by law, the Company does not undertake any responsibility to update any forward-looking statements to take into account events or circumstances that occur after the date of this press release.

CONTACTSMedia:Grace FotiadesLifeSci Communicationsgfotiades@lifescicomms.com (646) 876-5026

Investors: Nader Pourhassan, Ph.D.President & CEOnpourhassan@cytodyn.com

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Impressive Results Continue from CytoDyn's Clinical Trials Evaluating Two Patients with Leronlimab, One in mTNBC and One in MBC - Benzinga

Bone Marrow Stem Cells Comments Off on Impressive Results Continue from CytoDyn’s Clinical Trials Evaluating Two Patients with Leronlimab, One in mTNBC and One in MBC – Benzinga | January 22nd, 2020

The first six months of his young life have been a roller-coaster but Mighty Hudson Cowie is back home in Alberta after an experimental gene therapy procedure in Tennessee was successful.

Its been so long since weve got to see our family, our friends, Hudsons dad Ian said. Even just to be able to show him off to everybody. Weve spent the last six months seeing how amazing of a little guy he is.

Now we finally get to share that with everybody.

Hudson was diagnosed with Severe Combined Immunodeficiency (SCID) within days of his birth on June 23. The condition, known to many as Bubble Boy Disease, prevents his body from fighting illnesses. Essentially, he didnt have an immune system.

READ MORE:Hundreds attend donor drive for Mighty Hudson, Alberta baby with rare immune disease

In August, Hudson was accepted into a gene therapy program at St. Jude Childrens Research Hospital in Memphis. A medical team removed some of his bone marrow cells and replaced the faulty gene with a corrected one. The cells were placed back into his body through an IV.

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They took his own bone marrow out in his bone marrow, hes got hematopoietic stem cells. They were able to take those cells and modify them to create a product, a repaired version of those cells, and then give them back to him, Ian told Global News on Monday.

Medicine is amazing. We took a chance on science and were so glad that we did. Its amazing what theyre able to do.

Ian and his wife Hayley were weighing two options for their little boy: using donor cells or a revamped version of Hudsons own cells. They decided on the second, which meant a trial at St. Judes.

We weighed the pros and cons of both options and for us, [and] personally, we decided that gene therapy seemed like the safer way to go, Ian said.

It didnt rely on a donor, it was the new up-and-coming medicine, the existing results for gene therapy were already incredibly promising and then, as an added bonus, it had substantially less chemotherapy requirements.

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WATCH: (Sept. 9, 2019) A Morinville baby with a rare disorder received a life-changing procedure in the U.S. Hudson Cowies parents give Su-Ling Goh an update on therapy for his immune system.

It honestly sounded too good to be true, Hayley added.

With bone marrow transplant, we heard of graft versus host disease, where its somebody elses cells and they can reject them. With gene therapy, having his own cells, he wasnt going to reject them because his body already knows them.

A few months after the transplant, they started to see results: Hudsons first T-cells, a type of white blood cell thats a key component of the immune system.

From three months, it exploded. At four months, it was even more T-cells. The growth was exponential, Ian said.

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I think thats the thing every doctor hopes to get to say to their patients one day: Hes cured. And the day that we left Memphis, she said that. She said: Hes cured.

We squealed like little schoolgirls. It was very exciting.

Hudsons immune system right now can be compared to that of a newborn baby.

As hes introduced to small bugs, itll just keep growing and growing and be normal, Hayley explained.

There are still unknowns, but the family is very hopeful.

He was Patient 12 on the trial and from what we were informed, everyone on the trial has done tremendously well, Ian said.

I just think its amazing, Hayley added. Its amazing that we were the first ones who got on newborn screening and that this trial was even available for us. It just seems that everything completely lined up.

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I feel like everything has just fallen into place to get him cured.

The treatment is just amazing. Our doctors are brilliant, its just all incredible and hes doing great.

Hudson was one of the first babies to be screened for SCID through a new program. Since SCID was added in May 2019, the Alberta Health Services Newborn Metabolic Screening Program has screened over 34,000 newborns and has diagnosed four cases of SCID.

While very much welcome, being home is still a big change for the Cowies.

Im still processing, even now, Hayley said. Its nice to go see people but we were in isolation for so long that it doesnt just flip all of a sudden. I know were still really protective and we can hear a cough from miles away.

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It was surreal to leave but I feel like Im still dreaming a little bit.

They havent attempted any big outings yet especially given its flu season but they have been able to go to some family dinners.

Its an adjustment for sure, but a good adjustment, Hayley said. He loves people Hes always just smiling and playful hes just happy.

The Cowies will be back in Tennessee for one day for a checkup this weekend. Hudson will have another checkup with the St. Judes team next month. Hell have followups at least once a year until hes 10 years old.

2020 Global News, a division of Corus Entertainment Inc.

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Alberta baby Mighty Hudson home after gene therapy: hes cured - Global News

Bone Marrow Stem Cells Comments Off on Alberta baby Mighty Hudson home after gene therapy: hes cured – Global News | January 20th, 2020

Warsaw (AFP)

Poland has emerged as Europe's leader in stem cell storage, a billion-dollar global industry that is a key part of a therapy that can treat leukaemias but raises excessive hopes.

Submerged in liquid nitrogen vapour at a temperature of minus 175 degrees Celsius, hundreds of thousands of stem cells from all over Europe bide their time in large steel barrels on the outskirts of Warsaw.

Present in blood drawn from the umbilical cord of a newborn baby, stem cells can help cure serious blood-related illnesses like leukaemias and lymphomas, as well as genetic conditions and immune system deficits.

Polish umbilical cord blood bank PBKM/FamiCord became the industry's leader in Europe after Swiss firm Cryo-Save went bankrupt early last year.

It is also the fifth largest in the world, according to its management, after two companies in the United States, a Chinese firm and one based in Singapore.

Since the first cord blood transplant was performed in France in 1988, the sector has significantly progressed, fuelling hopes.

- Health insurance -

Mum-of-two Teresa Przeborowska has firsthand experience.

At five years old, her son Michal was diagnosed with lymphoblastic leukaemia and needed a bone marrow transplant, the entrepreneur from northern Poland said.

The most compatible donor was his younger sister, Magdalena.

When she was born, her parents had a bag of her cord blood stored at PBKM.

More than three years later, doctors injected his sister's stem cells into Michal's bloodstream.

It was not quite enough for Michal's needs but nicely supplemented harvested bone marrow.

As a result, Michal, who is nine, "is now flourishing, both intellectually and physically," his mum told AFP.

A cord blood transplant has become an alternative to a bone marrow transplant when there is no donor available, with a lower risk of complications.

Stem cells taken from umbilical cord blood are like those taken from bone marrow, capable of producing all blood cells: red cells, platelets and immune system cells.

When used, stem cells are first concentrated, then injected into the patient. Once transfused, they produce new cells of every kind.

At the PBKM laboratory, "each container holds up to 10,000 blood bags... Safe and secure, they wait to be used in the future," its head, Krzysztof Machaj, said.

The bank holds around 440,000 samples, not including those from Cryo-Save, he said.

If the need arises, the "blood will be ready to use without the whole process of looking for a compatible donor and running blood tests," the biologist told AFP.

For families who have paid an initial nearly 600 euros ($675) and then an annual 120 euros to have the blood taken from their newborns' umbilical cords preserved for around 20 years, it is a kind of health insurance promising faster and more effective treatment if illness strikes.

But researchers also warn against unrealistic expectations.

- Beauty products -

Haematologist Wieslaw Jedrzejczak, a bone marrow pioneer in Poland, describes promoters of the treatment as "sellers of hope", who "make promises that are either impossible to realise in the near future or downright impossible to realise at all for biological reasons."

He compares them to makers of beauty products who "swear their cream will rejuvenate the client by 20 years."

Various research is being done on the possibility of using the stem cells to treat other diseases, notably nervous disorders. But the EuroStemCell scientist network warns that the research is not yet conclusive.

"There is a list of almost 80 diseases for which stem cells could prove beneficial," US haematologist Roger Mrowiec, who heads the clinical laboratory of the cord blood programme Vitalant in New Jersey, told AFP.

"But given the present state of medicine, they are effective only for around a dozen of them, like leukaemia or cerebral palsy," he said.

"It's not true, as it's written sometimes, that we can already use them to fight Parkinson's disease or Alzheimer's disease or diabetes."

EuroStemCell also cautions against private blood banks that "advertise services to parents suggesting they should pay to freeze their child's cord blood... in case it's needed later in life."

"Studies show it is highly unlikely that the cord blood will ever be used for their child," the network said.

It also pointed out that there could be a risk of the child's cells not being useable anyway without reintroducing the same illness.

Some countries, such as Belgium and France, are cautious and ban the storage of cord blood for private purposes. Most EU countries however permit it while imposing strict controls.

- Rapid growth -

In the early 2000s, Swiss company Cryo-Save enjoyed rapid growth.

Greeks, Hungarians, Italians, Spaniards and Swiss stored blood from their newborns with the company for 20 years on payment of 2,500 euros upfront.

When the firm was forced to close in early 2019, clients were left wondering where their stem cells would end up.

Under a kind of back-up agreement, the samples of some 250,000 European families were transferred for storage at PBKM.

The Polish firm, founded in 2002 with two million zlotys (around 450,000 euros, $525,000), has also grown quickly.

Present under the FamiCord brand in several countries, PBKM has some 35 percent of the European market, excluding Cryo-Save assets.

Over the last 15 months, outside investors have contributed 63 million euros to the firm, PBKM's chief executive Jakub Baran told AFP.

But the company has not escaped controversy: the Polityka weekly recently published a critical investigative report on several private clinics that offer what was described as expensive treatment involving stem cells held by PBKM.

2020 AFP

Read more:
Europe's guardian of stem cells and hopes, real and unrealistic - FRANCE 24

Bone Marrow Stem Cells Comments Off on Europe’s guardian of stem cells and hopes, real and unrealistic – FRANCE 24 | January 20th, 2020

Remember being a teenager in high school and thinking 40 was old? I distinctly recall being 15 or 16 years old and thinking Id have it all figured out by the time I was 25. In my mind, Id be a proper grown-up by then in the career of my dreams and married with a baby25 seemed so far away. For the record, I knew nothing at age 25 and felt anything but grown-up. But oh, how quickly time passes. Here I am, a year from turning 40, Ive been married for 12 years, and our son just turned 11. Even now, I cant say with confidence that I feel like an adult. My husband and I still turn to each other with a quizzical expression wondering how we both got here. Im sure many of you reading this can relate.

Of course, the old adage still rings true, and you are only as old as you feel, but even if I dont feel like it, the mirror tells me a different story. Its a daily reminder that time will have her way and you cant stop her. And dont get me wrongIm all about aging gracefully, but I want to look good while I do. For me, its all about taking care of my skin health. Ive been on a cleanse-tone-moisturize-sunscreen routine since I was 14 years old (thanks, Mom!), and its slowly developed into monthly facials, lasers, and a few injectables.

With my big 4-0 coming up, I wanted to try a few buzzy skincare treatments to help turn back the clock a little and make sure my skin looks 100 when the day finally arrives. Read on to find out what Idid along with my honest thoughts, and feel free to DM me at @skinlexicon if you have any questions!

I have already written about my experience with under-eye fillers and lip filler, and I loved both, but Im definitely very careful about who I see for injectables and what filler I use. Most people have some apprehension about trying fillers because there are a lot of examples where it has gone wrong.

Unfortunately, there are a lot of people walking around with filler that you can see and recognize that it was done badly with an unnatural result, says Nancy Samolitis, MD, FAAD, of Facile Dermatology + Boutique in Los Angeles. Because of this, many people have the misconception that all filler treatments will look this way. When the appropriate filler product is used with the correct technique, injected at the correct depth in the correct amount, it should not be visible or look unnatural. This was the case with my experience having a liquid facial. Samolitis chose different types of filler and Botox to suit specific areas of my face to ensure a natural look that still had movement and didnt look stiff or overdone.

There is still a lot of confusion around when/where/how fillers or Botox are used, so I asked Samolitis to settle the debate once and for all.

Botulinum toxin (Botox, Dysport, Xeomin, and Jeuveau are all different brands): "This is what's known as a neuromodulator," Samolitis explains. "That means it binds to a specific receptor in a nerve ending that prevents the nerve from communicating with a muscle, therefore reducing the muscle activity. These products are used to reduce wrinkles that are associated with certain facial movements/expressions. This is most commonly used for areas such as the frown line between the eyebrows (aka the "elevens"), forehead lines, and crow's-feet lines. It can also be used for upper lip lines, chin dimpling, and lines in the neck.

Newer techniques are using these products to create lift by relaxing muscles that are natural depressors in the face and neck. For example, that muscles that pull down on the brows or the jowls can be relaxed to create a very slight lifted look. Even more exciting and new uses for these products include injecting micro-droplets into the skin (instead of the muscle) to reduce oil production in pores, reduce sweating, shrink blood vessels to reduce redness/flushing, and smooth skin texture. There may even be some benefit for treating acne and rosacea, but this is still being studied.

Fillers: These products replace volume. The most commonly used fillers are made of hyaluronic acid, which is a sugar molecule that is bound into a gel-like injectable and placed into various levels of the skin and deeper tissues to replace tissue that we have lost with aging or enhance tissue that we don't have naturally. Certain thicker fillers are used more appropriately on bone and in the fat compartment in the face to create contour (for example, volumizing and contouring the cheeks, temples, and jawline).

"Fillers that are smooth and [bouncier and more elastic] are used for smoothing and plumping lips and around the mouth where there is a lot of movement," Samolitis explains. "If a thicker filler is used in these areas or injected superficially, it can create an unnatural stiff or overly plumped look. Finally, very thin, almost watery, fillers are used to create a hydrated effect in the top layers in the skin or fill in very fine lines or acne scars."

Samolitis used various types of filler and botox around my face during the treatment, and in most cases, she says we need a combination of the products described above to create a harmonious overall result. Signs of aging in the face are usually a combination of volume loss including bone resorption and decreasing the size of the fat pockets that give our face its characteristic shape and thinning of the skin leading to fine lines and texture change, she explains. Injectables should also always be combined with skin rejuvenation techniques including a good skincare regimen, sun protection, and in-office procedures like micro-needle or laser resurfacing. She tells me any medspa that only provides injectables is doing the patient a disservice by not treating the skin and educating the patient on sun and environmental damage. Hear, hear.

The other bonus of getting filler is that it actually helps to create more collagen. The injection creates a tiny wound and a stretching of the cells called fibroblasts in the dermal layer of the skin. This stimulus initiates a cascade of wound-healing signals that ultimately lead to new collagen production. Obviously, this is on a very small scale, but often repeated filler injection in the same area (most commonly the lips, a fine wrinkle, or acne scars) will lead to longer-lasting results over time.

Of course, injectables arent cheap but the good news is, you dont need to get them that often. Botulinum toxin averages three to four months while filler can be anywhere from three months to several years (it depends on the type of filler, area treated, amount of filler used, and the person's metabolism).

If you havent heard ofThe Beauty Sandwich yet then Id suggest checking it out on Instagramstat. The creator,Ivn Pol, is ringing in a new age of noninvasive and nontoxic beauty treatments with his unique skin-tightening methodand the Hollywood A-list are lining up. As a long-time fashion and celebrity makeup artist, Pol wanted an alternative way to reduce the look of wrinkles without needles and saw an opportunity in the market for a natural alternative to "looking our best" and staying young. So he developed The Beauty Sandwich, a unique and customized combination of advanced radiofrequency including monopolar, bipolar, and infrared RF. The method organically plumps and builds collagen to give a filler-like appearance, and Im obsessed!

So whats the magic sauce? Why does it work? While there are a lot of devices out there that show improvement in resurfacing and remodeling the skin, Pol has found that the secret to delivering lasting results is through combining or stacking one procedure on another, just like a sandwich. Pol stacks two radio frequency (RF) devices on top of each other. The first is a monopolar system called Pellev, which means the RF (radio frequency) can penetrate deeply until the tissue reaches a certain temperature. This deep energy (which feels like heat to the client) tightens the skin while at the same time stimulates the fibroblasts, which are the workforce cells that produce new collagen and elastic fibers in the skin.

The second is the eMatrix system, which is a bipolar RF device that limits its energy to the upper levels of the skin. This too stimulates the fibroblasts as well as causes some superficial exfoliation. The combination of the two creates The Sandwich, which gives an immediate tightening effect with the long-term stimulation of the fibroblasts. Add some great topical skincare, and youll get that radiant glow were all after.

But, of course, the big question is, how much does it cost and how long does it last? While the results are case by case, Pol tells me he typically recommends six to eight treatments with touch-ups before big events, but the results can last up to a year or more. They range in price from $850 to $1400, so its not exactly cheap, but when you think about how long it lasts, its comparable to filler, and its completely natural and safe. This is the natural alternative to have a filler-like appearance to the skin, says Pol. I think of The Beauty Sandwich as weight training. We are strengthening and toning the muscles to help with collagen stimulation which plumps and lifts the skin, contouring the face and defining jawline.

As you can see from the pictures, the method shows visible results after just one session, and that continues two weeks after. In fact, I had the post-treatment glow for up to three days after the procedure, but it is the gift that keeps on giving. Pol says four to 15 days after the treatment, your bodys natural reaction to heal comes into play as fibroblasts become present, and the collagen rebuilding process begins. And it continued to do so days, weeks, even months after treatment.

But aside from the aesthetic results, the biggest transformation Pol has seen is in the way women feel about themselves after a series of treatments. That boost in their self-esteem, the voicemails I receive when they love what they see, that empowerment is why I created The Beauty Sandwich, he tells me. I have always believed that beauty comes from within. It starts from the bed and the sheets you sleep on to what you ingest. Same goes for what you put on your skin.

I have been seeing celebrity estheticianMatthew Miller for monthly facials for at least the past year. He is one of my favorites in the L.A. area and definitely the most reasonably priced that Ive found here, too. So when he told me about his new treatment,DermalStem Regeneration, my ears pricked and I immediately booked an appointment with him. In a nutshell, this treatment is an amazing alternative to micro-needling without any pain or downtime. While I was there for the anti-aging effects, it can also help other concerns like acne, hyperpigmentation, and dry skin.

So what is it? Well, DermalStem Regeneration is another treatment that stimulates the growth of, you guessed it, collagen. Firstly, a Bio-Brasion wet/dry microdermabrasion is used to exfoliate the skin. Then he uses a dermastamp to create microchannels on the skins surface. These microchannels allow a serum derived from bone marrow stem cells to penetrate deeper into the skin. Yes, I said bone marrow stem cells! This game-changing serum is packed with next-level growth factors that trigger the skins natural renewal process and promote the formation of collagen and elastin to provide firmness and elasticity. Read: Sign me up! This is quickly followed with a hyaluronic acid serum and 20 minutes of LED to accelerate healing and reduce redness. Both of these together give you the benefits of physical and chemical exfoliation without the irritation or downtime.

Youll see results instantly with this facial, and it just keeps getting better even up to a month post-treatment as the bone marrow stem cells continue to work. You do need to give your body time to produce the collagen to heal the skin after this one, so theres no need to rush back in straight away or sign up for multiple treatments, but Miller says you will see a noticeable difference after three of these. So how much does it cost? The treatment is pricey at $475, but I really do see the value in this one. You can get discounted rates when you buy in bulka three-pack series is $1210, while a six-pack series is $2280.

As I mentioned earlier, I am very picky about who I allow to inject my face. I have seenJennifer Herrmann, MD, twice now for both under-eye fillers and lip fillers, and her work is very subtle and natural. So when I wanted to try the PiQo4 laser to remove a few dark spots, she was my first port of callshe also turned my own blood into an anti-aging serum.

So what is the PiQo4 laser? According to Herrmann, its a picosecond laser thats used to treat unwanted pigmentation and tattoos. It uses energy to target pigment particles, shatters them, and then your bodys immune system helps clear them away, she says. Because it hits its targets so quicklyat picosecond burstsit converts laser light energy into sound almost instantly as it interacts with targets, generating very little heat in the process. This results in less unwanted damage to surrounding tissues, fewer side effects, increased safety for darker skin, and less pain and downtime for patients. In a nutshell, this game-changing laser can pinpoint dark spots and treat them individually without having to treat the entire face. And it works!

But its not just for people with hyperpigmentation like me. You can also use it to remove unwanted tattoos, sunspots, brown spots. In fact, its one of Herrmanns favorite lasers for diffusing brown spots caused by the sun. She also used a fractionated setting on my skin to treat summer sun damage, and it only took one treatmentmy skin is so much clearer and more even. And it only took a week for the dark spots to completely disappear, too.

How many treatments you need all depends on what is being treated. Discrete brown spots can clear in a single treatment, while tattoos can take more than 10 treatments. Prices begin at $250 per session, which is a small price to pay for clearer, healthier skin.

For my fourth treatment, I went to see the much-adored Camille FieldsTyLynn Nguyen is a regular. When you meet Fields, its easy to see why influencers, models, and celebrities book months in advance to have their faces in her magical hands. Fields has been in the industry for 20 years, and her own glowing skin is proof that her treatments and skincare philosophy works. Shes all about strengthening your skin from the inside outSakara and The Beauty Chef products sit proudly alongside her skincare recommendations

Speaking of which, Fields is an officialBiologique Recherche salonthe beloved French skincare line that has a global cult following. Aside fromits groundbreaking, science-based line, Biologique Recherche offers a series of specialty treatments to enhance the effectiveness ofits products, and the Le Grand Soin is the crown jewel. During this treatment, Fields uses specialty boosters, co-factors, masks, serums, and the world-renowned Remodeling Face Machine (the ultimate for firming and lifting the facial muscles), as well as lymphatic and sculptural massage, LED light therapy, and ultrasound. By the end, your face is taut, clear, bright, and glowy. (I have the pictures and videos to prove it!)

All of this will set you back $550, but if you are looking for a treatment that provides long-lasting, skin healthboosting results, this is itmy skin was transformed. But, of course, total skin health is about so much more than just one treatment. As Fields explains, Our skin is just a reflection of our internal health. To truly address the root cause of any skin concern, we have to first look within. Incredible changes can occur when we respect and understand the physiology of our bodies.

Below, I'm sharing the skincare products I used daily to maintain my glow. Keep scrolling to shop my favorite, ride-or-die formulas.

Cosmedix Salicylic Acid Foaming Cleanser ($39)

I love an exfoliating cleanser to slough off dead skin cells to keep blackheads away and keep my skin feeling smooth and fresh. Both of these are also incredibly gentle on the skin and never irritate or dry my skin out. I'm obsessed with Cosmedix's new foaming cleanser. It's a salicylic acid foaming cleanser, but it's so gentle and doesn't have any parabens, which I love.

Cosmedix Purity Balance Exfoliating Prep Toner ($38)

I'm a big fan of using acids, and this one has both of my favorites,salicylic and lactic acids. I have been using this toner for at least 10 years now, and it is truly one of my hero products. I love how it removes excess oil or residue (including makeup) to reveal smooth, clear skin. It can also be used as a spot treatment for breakouts, and I've even used it in lieu of a cleanser when I forgot to pack it on a work trip once. It's really an amazing product.

IS Clinical White Lightening Complex ($145)

I was gifted with this product about two months ago and have been using it every day since.I do think that my skin overall is brighter and firmer. It's really lightweight (a waterlike essence consistency) too, which I like because I use a lot of serums, and if they're too heavy, it can pill, which I loathe. It is also paraben-free.

The other brightening cream I LOVE isSkinMedica'sLytera 2.0 Pigment Correcting Serum ($154). This product really works to help even skin tone and manage pigmentation.I'm super obsessed with even skin tone, so I have several brightening serums in my regimen, includingCosmedix X-Cell($71), which also has niacinamide and microencapsulated Retinol. I love mixing it withitsvitamin C crystals for an added boost.

IS Clinical Super Serum Advance ($155)

This one is also lightweight, but I love it because it has a really high concentration ofL-ascorbic acid (vitamin C), a whopping 15% to be exact. I also love theCosmedixVitamin C Crystals ($32), which you can combine with any serum or moisturizerVersed also has onefor $20.

Cosmedix Refine RX ($78)

I have been using retinol for years, so I can handle the highest-strength version from Cosmedix, but if you're just starting out or have sensitive skin,the brand hasa range of retinol, so you can work your way up.Serum 16($80) is a good place to start.

SkinMedica HA5 Rejuvenating Hydrator ($178)

There are a lot of hyaluronic acid serums out there. I mean a lot. But after trying almost everything out there (well, not all, but a lot!), I consistently come back to this one. It works, plain and simple. I recently started testing out the IS Clinical Hydracool serum after my friend and celebrity estheticianCandace Marinorecommended it. It's packed with hyaluronic acid and feels incredibly cooling to the skinI'm already a convert.

Cosmedix Cell ID ($110)

My estheticianMatthew Millerrecommends this serum to almost everyone. It has really high levels of niacinamide, a form of vitamin B3 that helps tomanage acne, rosacea, pigmentation issues, and wrinkles. This is a really nice everyday defense serum.

IS Clinical Active Serum ($138)

I mix a few drops of this with the IS ClinicalSuper Serum Advance($155) and the White Lightening Serum ($145) morning and night straight after I've toned my skin. I've been doing this for a few months now, and I can honestly say it instantly tightens and brightens my skin. I have definitely noticed a difference. I really love how fresh it makes my skin feel toothe results are immediate. Not sure how I ever lived without it.

Cosmedix X-Age ($160)

I have been using this moisturizer for about a year now, and I notice the difference in my skin texture and overall appearance when I stop using it. This is worth the $$. It has encapsulated vitamin A and a ton of antioxidants to help improve the texture and elasticity of aging skin. I just really love it. I mix it with my face oil every night for added hydration and dewy skin.Humidify($90) is also lovely when I want something hydrating but lightweight.

DMK Seba-E Oil ($0)

I was first introduced to this face oil through Matthew Miller, and I have been hooked on (actually, I think it's safe to say obsessed with)this face oil ever since. I love to mix it with my moisturizer and even reapply it throughout the day for dewy, glowy skin. It can be mixed with makeup too.Mara BeautyAlgae + Moringa Universal Face Oil ($72) is also really beautiful and great mixed with makeup as is the new face 100% organic oil byKohza Numbers(touted as a daily supplement serum withvitamins A, C, and E). It's super lightweight and looks insane when mixed with foundation. I just love it; I just DEW!

Cosmedix Eye Doctor ($130)

This magical eye cream is hands down one of the best out there. I promise. It is like eye cream and a highlighter in one.TheOpti-Eye Crystal ($95) (which is slightly cheaper) is also great and one ofthe brand'sbest-selling products. But I personally prefer the Eye Doctor because it has retinol and feels more targeted to my anti-aging needs.

IS Clinical Eclipse SPF 50 Plus ($45)

This is almost like a mousse in texture. It's light, not too oily, and mixes into the skin without leaving a white residue but also keeps my skin dewy. I love the size of the bottle, and I use it for my face and body. It's paraben-free and doesn't have any active chemical ingredients. Side note: My husband had a terrible reaction tooxybenzone and avobenzone, so if you're sensitive, please look out for that ingredient and avoid sunscreens that have it.

IS Clinical Copper Firming Mist ($35)

I have triedso many face mists, but this is by far my favorite. It does feel firming and is a great spritz to set makeup. I also really loveCosmedixMystic ($42). It smells so good and is incredibly hydrating.

Up next,18 Reasons Kate Bosworth Is Benjamin Buttoning Right Before Our Eyes

This article originally appeared on Who What Wear

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Bone Marrow Stem Cells Comments Off on I Tried 5 Buzzy Skincare Treatments Before My 40th BirthdayHere Are My Reviews – Yahoo Lifestyle | January 19th, 2020

obal Cell Therapy Technologies Marketwas valued US$ 12 billion in 2018 and is expected to reach US$ 35 billion by 2026, at CAGR of 12.14 %during forecast period.

The objective of the report is to present comprehensive assessment projections with a suitable set of assumptions and methodology. The report helps in understanding Global Cell Therapy Technologies Market dynamics, structure by identifying and analyzing the market segments and projecting the global market size. Further, the report also focuses on the competitive analysis of key players by product, price, financial position, growth strategies, and regional presence. To understand the market dynamics and by region, the report has covered the PEST analysis by region and key economies across the globe, which are supposed to have an impact on market in forecast period. PORTERs analysis, and SVOR analysis of the market as well as detailed SWOT analysis of key players has been done to analyze their strategies. The report will to address all questions of shareholders to prioritize the efforts and investment in the near future to the emerging segment in the Global Cell Therapy Technologies Market.

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Global Cell Therapy Technologies Market: OverviewCell therapy is a transplantation of live human cells to replace or repair damaged tissue and/or cells. With the help of new technologies, limitless imagination, and innovative products, many different types of cells may be used as part of a therapy or treatment for different types of diseases and conditions. Celltherapy technologies plays key role in the practice of medicine such as old fashioned bone marrow transplants is replaced by Hematopoietic stem cell transplantation, capacity of cells in drug discovery. Cell therapy overlap with different therapies like, gene therapy, tissue engineering, cancer vaccines, regenerative medicine, and drug delivery. Establishment of cell banking facilities and production, storage, and characterization of cells are increasing volumetric capabilities of the cell therapy market globally. Initiation of constructive guidelines for cell therapy manufacturing and proven effectiveness of products, these are primary growth stimulants of the market.

Global Cell Therapy Technologies Market: Drivers and RestraintsThe growth of cell therapy technologies market is highly driven by, increasing demand for clinical trials on oncology-oriented cell-based therapy, demand for advanced cell therapy instruments is increasing, owing to its affordability and sustainability, government and private organization , investing more funds in cell-based research therapy for life-style diseases such as diabetes, decrease in prices of stem cell therapies are leading to increased tendency of buyers towards cell therapy, existing companies are collaborating with research institute in order to best fit into regulatory model for cell therapies.Moreover, Healthcare practitioners uses stem cells obtained from bone marrow or blood for treatment of patients with cancer, blood disorders, and immune-related disorders and Development in cell banking facilities and resultant expansion of production, storage, and characterization of cells, these factors will drive the market of cell therapy technologies during forecast period.

On the other hand, the high cost of cell-based research and some ethical issue & legally controversial, are expected to hamper market growth of Cell Therapy Technologies during the forecast period

AJune 2016, there were around 351 companies across the U.S. that were engaged in advertising unauthorized stem cell treatments at their clinics. Such clinics boosted the revenue in this market.in August 2017, the U.S. FDA announced increased enforcement of regulations and oversight of clinics involved in practicing unapproved stem cell therapies. This might hamper the revenue generation during the forecast period; nevertheless, it will allow safe and effective use of stem cell therapies.

Global Cell Therapy Technologies Market: Segmentation AnalysisOn the basis of product, the consumables segment had largest market share in 2018 and is expected to drive the cell therapy instruments market during forecast period at XX % CAGR owing to the huge demand for consumables in cell-based experiments and cancer research and increasing number of new product launches and consumables are essential for every step of cell processing. This is further expected to drive their adoption in the market. These factors will boost the market of Cell Therapy Technologies Market in upcoming years.

On the basis of process, the cell processing had largest market share in 2018 and is expected to grow at the highest CAGR during the forecast period owing to in cell processing stage,a use of cell therapy instruments and media at highest rate, mainly in culture media processing. This is a major factor will drive the market share during forecast period.

Global Cell Therapy Technologies Market: Regional AnalysisNorth America to held largest market share of the cell therapy technologies in 2018 and expected to grow at highest CAGR during forecast period owing to increasing R&D programs in the pharmaceutical and biotechnology industries. North America followed by Europe, Asia Pacific and Rest of the world (Row).

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Scope of Global Cell Therapy Technologies Market

Global Cell Therapy Technologies Market, by Product

Consumables Equipment Systems & SoftwareGlobal Cell Therapy Technologies Market, by Cell Type

Human Cells Animal CellsGlobal Cell Therapy Technologies Market, by Process Stages

Cell Processing Cell Preservation, Distribution, and Handling Process Monitoring and Quality ControlGlobal Cell Therapy Technologies Market, by End Users

Life Science Research Companies Research InstitutesGlobal Cell Therapy Technologies Market, by Region

North America Europe Asia Pacific Middle East & Africa South AmericaKey players operating in the Global Cell Therapy Technologies Market

Beckman Coulter, Inc. Becton Dickinson and Company GE Healthcare Lonza Merck KGaA MiltenyiBiotec STEMCELL Technologies, Inc. Terumo BCT, Inc. Thermo Fisher Scientific, Inc. Sartorius AG

MAJOR TOC OF THE REPORT

Chapter One: Cell Therapy Technologies Market Overview

Chapter Two: Manufacturers Profiles

Chapter Three: Global Cell Therapy Technologies Market Competition, by Players

Chapter Four: Global Cell Therapy Technologies Market Size by Regions

Chapter Five: North America Cell Therapy Technologies Revenue by Countries

Chapter Six: Europe Cell Therapy Technologies Revenue by Countries

Chapter Seven: Asia-Pacific Cell Therapy Technologies Revenue by Countries

Chapter Eight: South America Cell Therapy Technologies Revenue by Countries

Chapter Nine: Middle East and Africa Revenue Cell Therapy Technologies by Countries

Chapter Ten: Global Cell Therapy Technologies Market Segment by Type

Chapter Eleven: Global Cell Therapy Technologies Market Segment by Application

Chapter Twelve: Global Cell Therapy Technologies Market Size Forecast (2019-2026)

Browse Full Report with Facts and Figures of Cell Therapy Technologies Market Report at:https://www.maximizemarketresearch.com/market-report/global-cell-therapy-technologies-market/31531/

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Global Cell Therapy Technologies Market Industry Analysis And Forecast (2018-2026) Dagoretti News - Dagoretti News

Bone Marrow Stem Cells Comments Off on Global Cell Therapy Technologies Market Industry Analysis And Forecast (2018-2026) Dagoretti News – Dagoretti News | January 19th, 2020

Libby Clegg, 29, has represented both Scotland and Great Britain at international sporting events. She won a silver medal at the 2008 Summer Paralympics and won gold at the 2016 Paralympic Games. Clearly up for any challenge, the sprinter is now competing in ITVs Dancing On Ice.

But why exactly is the star registered as blind? The sporting star has a deteriorating eye condition known as Stargardts Macular Dystrophy which gives her only slight peripheral vision in her left eye.

Libby has described her eyesight as being like looking at a pixelated computer screen or a scrunched-up firework, when speaking to The Daily mail.

She added: I have some peripheral sight - barely any - and with what little sight I do have I was able to use to use to follow the lines on the track.

Libby wasnt born blind, and started losing her eyesight at the age of 9.

But theres currently no treatment for Stargardt, and eventually, the mother-of-one will lose her entire sight.

She continued: Im at the age where my sight should be stabilising but its still deteriorating.

Things will never go black, but I dont know yet exactly what I will be able to see.

Speaking to The Radio Times about her learning process on the ice rink, Live explained: Its been a learning process.

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On the track I run with a guide runner and were attached all the time, but basically its like learning a different vocabulary to communicate.

Myself and my partner Mark Hanretty use touch and verbal communication.

Im not as bad as I thought I was going to be, but its not as easy as it looks.

Its a lot harder than I thought itd be, its very technical."

Moorfields Eye Hospital, part of the NHS Foundation Trust, says Stargardt disease is a rare inherited condition affecting one in 8,000 to 10,000 people.

It explains: In Stargardts the light-sensitive layer of cells in the macular region of the eye degenerate.

The macular is the area at the back of the eye which is responsible for the fine detailed vision necessary for activities such as watching TV and reading.

Symptoms of the condition include:

Moorfields Eye Hospital says UV blocking sunglasses can offer some protection for remaining vision, but the condition is currently untreatable.

It adds: A number of novel interventions are currently under investigation, including stem cell therapies. Stem cells are a special type of cell which, when put under the right conditions, can develop into many other types of cell including those found in the macular. It is hoped that new cells derived from stem cells can be grown in a laboratory to be transplanted into the eye to replace areas of dead or non-functioning cells. Stem cells can be sourced from a number of places including blood, bone marrow, umbilical cord and fertilized egg cells.

Researchers are involved in Europes first ongoing stem cell trial for Stargardts. More research will need to be undertaken in the future to determine to what extent stem cell therapy might help improve vision for people with Stargardts.

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Libby Clegg health: Dancing On Ice star is registered blind - what is her condition? - Express

Bone Marrow Stem Cells Comments Off on Libby Clegg health: Dancing On Ice star is registered blind – what is her condition? – Express | January 19th, 2020

Thats something 13-year-old Floyd Korzan learned the hard way when his father, Matt, was diagnosed with acute myeloid leukemia, a highly-deadly form of blood cancer, after he fell ill on a family hiking trip in the Black Hills in 2012.

Matts fight against the disease has since taken the Mitchell family on a journey of frightening lows and celebratory highs and led Floyd to start Relentless Pledge, a non-profit organization that encourages patients to be as relentless as his father as they fight to achieve the goal of overcoming cancer.

We basically have two goals. The first one is to inspire others to overcome obstacles and dream their dreams. And the second part of the goal is to give a Relentless wristband to every cancer patient in Mitchell, our hometown, and eventually, if we get big enough, South Dakota and the United States, Floyd said in an interview with the Daily Republic.

Its a goal borne out of Floyds experience when he sat with his father at the Mayo Clinic in Rochester, Minnesota as he underwent treatments for the disease. Matt beat the leukemia once, but it later returned, a bad sign when associated with this particular kind of cancer. It went into remission again but returned again in 2017, and doctors were not hopeful.

With few options remaining, Matt agreed to a stem cell donation treatment that would use cells provided by his sister, Margaret, who happened to be a 100 percent match, injected into his system following an intense round of chemotherapy. The treatment was difficult, but after 8 million donor cells were circulated through his system, the doctors began to see good results.

Little by little, I came back, Matt said. After 30 days, they did a bone marrow biopsy that showed no evidence of leukemia whatsoever. They did another one at the end of the year, still no evidence of leukemia. They did one at the two-year mark, still no evidence of leukemia.

Visitors to relentlesspledge.org can nominate individuals to receive Relentless wristbands, which remind people to stay the course when attempting to achieve their goals. (Erik Kaufman / Republic)

Floyd recounted the scary days of his dads fight in an essay posted on his website relentlesspledge.org. He asked his father at the time how he could be as strong as he was even as he suffered from the disease and the side effects of his treatment. Matts response was that there may be times in life when the only person left who believes in you is you, and in those times, you must be relentless in order to make it through.

Floyd had found his own inspiration in his fathers fight, and he wanted to spread that inspiration to others who were suffering. Normally a private person, Matt agreed to share his story with the public and helped his son form Relentless Pledge, which encourages people to live life to the limit, to dare, to dream, and be relentless in overcoming challenges.

Visitors to the website can take the pledge, as well as nominate individuals to receive one of the symbols of the organization: a wristband bearing the word Relentless. Visitors can also order wristbands for themselves, family members, friends as well as cancer patients.

The goal is to spread the message of hope to every cancer patient in America, Floyd said. He has shipped orders of wristbands to 26 states around the country and three continents already, and the pair plan to pass out the wristbands to people in the leukemia ward at the Mayo Clinic, where Matt returns periodically for continued treatments. More wristbands will be given out at the Avera Cancer Center in Mitchell.

In total, they estimate theyve given out about 400 of them so far. And the campaign is officially less than two weeks old.

It seemed to strike a chord with people, Matt said.

As the young program grows, Floyd said the organization could expand to include more than just cancer patients in its message. There are others who are trying to accomplish goals, as well, such as public servants or community-minded individuals.

Its not all about cancer. Now were looking at giving them out to local heroes," Floyd said. "So far, weve given them to local firefighters and the librarians at the Mitchell Public Library, and were thinking about the police department."

Floyd hopes Relentless Pledge continues to grow. He is eyeing expanding the Relentless line to include t-shirts that would promote the Relentless Pledge, but for now he is concentrating on fulfilling orders for the wristbands and getting them in the hands of people who need inspiration.

I have big dreams in the future where this organization could expand to have a full line of accessories, Floyd said. Relentless gear.

Matt said he is recovering a little more each day, and hes grateful. Hes been able to throw the ball around in the yard with Floyd again, and he is working to become stronger as he continues his recovery. He said hes grateful for the love and support of Floyd as well as his wife Bam and other children Alexis, Cassidy, Hailey and Jackson. Hes thankful for doctors like Kebede Begna and Lucio Margallo, both of whom Matt described as relentless in helping him fight for his health.

And hes happy to help Floyd spread the message of hope through Relentless Pledge, he said.

I think its great. I think its a noble project, and I think its good for Floyd. Of all the ways a teenager can spend their time, this is pretty solid, Matt said.

Floyd said he plans to continue his work with the organization well into the future. He has a little over four years left before he leaves for college, and with that time he plans to do everything he can to remind those who are struggling to remain relentless.

One Relentless wristband at a time.

The next step is spreading the word, Floyd said.

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Learning to be relentless - The Daily Republic

Bone Marrow Stem Cells Comments Off on Learning to be relentless – The Daily Republic | January 18th, 2020

Research findings show that rare mutations from donor stem cells can be passed onto patients who receive them, potentially causing health concerns.

Researchers from Washington University School of Medicine in St. Louis discovered this while analyzing bone marrow samples from 25 adult patients with acute myeloid leukemia (AML).

Heart damage, graft-versus-host disease and, potentially, new leukemias, are the risks associated with these mutations.

There have been suspicions that genetic errors in donor stem cells may be causing problems in cancer patients, but until now we didnt have a way to identify them because they are so rare, senior author Dr. Todd E. Druley, an associate professor of pediatrics, said in a news release. This study raises concerns that even young, healthy donors blood stem cells may have harmful mutations and provides strong evidence that we need to explore the potential effects of these mutations further.

The harmful mutations were found in surprisingly young donors, explained the researchers. Healthy donors ranged in age from 20 to 58, with an average age of 26 years old. Interestingly, the mutations, because they are so rare, were not detected using usual genome sequencing techniques.

In the study, the researchers sequenced 80 genes that are associated with AML using a technique called error-corrected sequencing. They found at least one harmful genetic mutation in 11 of the 25 donors. Eighty-four percent of the mutations identified in the donor samples were potentially harmful and 100% of the harmful mutations were found in the recipients the most common mutation seen is a gene associated with heart disease.

We didnt expect this many young, healthy donors to have these types of mutations, Druley said. We also didnt expect 100% of the harmful mutations to be engrafted into the recipients. That was striking.

These harmful mutations persisted over time, and many increased in frequency, explained the researchers.

In addition, 75% of patients who received at least one harmful mutation developed chronic graft-versus-host disease. In patients who didnt receive a mutation, 50% developed the condition. Graft-versus-host disease either acute or chronic, can occur in patients who receive an allogeneic transplant, which consists of donor stems cells versus a patients own stem cells.

The researchers plan to examine the mutations in a larger study to answer the questions that this study revealed.

Transplant physicians tend to seek younger donors because we assume this will lead to fewer complications co-author Dr. Sima T. Bhatt, an assistant professor of pediatrics who treats pediatric patients with blood cancers at Siteman Kids at St. Louis Childrens Hospital and Washington University School of Medicine, said in a news release. But we now see evidence that even young and healthy donors can have mutations that will have consequences for our patients. We need to understand what those consequences are if we are to find ways to modify them.

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Mutations in Donor Stem Cells Could Harm the Health of Patients with Cancer, Study Finds - Curetoday.com

Bone Marrow Stem Cells Comments Off on Mutations in Donor Stem Cells Could Harm the Health of Patients with Cancer, Study Finds – Curetoday.com | January 17th, 2020

Doctors use stem cell transplants to treat patients with certain cancers or blood disorders. And donors, whose blood or bone marrow is used for the procedures, are typically young, for a variety of reasons.

But a pilot study released Wednesday raised the possibility that such donors are also passing along mutations in stem cells that could lead to health problems for some recipients.

The study found that nearly 45% of younger donors had mutations in the transplanted stem cells that could raise the risk of conditions that are sometimes seen in recipients, a higher rate than presumed. Researchers also reported that some of these mutations persisted and proliferated in the recipients bone marrow for at least a year.

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What remains unknown is whether those mutations are actually contributing to health problems for recipients.

The study was small, with just 25 donors included and was not large enough and did not last long enough to determine whether people who received cells with these mutations had worse outcomes after a transplant than recipients who got cells without those mutations. Dr. Todd Druley, the senior author of the paper, which was published in the journal Science Translational Medicine, emphasized that patients should continue to receive these stem cells to treat their leukemias or anemias when recommended.

What were trying to say is that now we can provide surveillance before, during, and after a bone marrow transplant so that if theres an increased risk for a particular outcome, treatment for that and surveillance for that can be instituted sooner, said Druley, a pediatric oncologist at Washington University in St. Louis.

Researchers not involved with the study praised its technical prowess, and said it was worth investigating further to see if the transplanted mutations did lead to worse outcomes for recipients projects that Druley and his colleagues have underway. But they agreed it should not yet change clinical practice.

Donors are already screened to make sure they have a clean bill of health and make for a good match for recipients, based on their immune systems. Experts said it would be unrealistic to screen every potential donor for the kinds of mutations Druley and his team found. Those mutations were infrequent, and it wasnt clear they posed health risks to recipients.

We know that younger donors are better than older donors. We know that the better the donor the better the outcomes. We dont know how these ultra-low level mutations affect outcomes at all, said Dr. Corey Cutler, the medical director of the adult stem cell transplantation program at Dana-Farber Cancer Institute.

Hematopoietic stem cells generate blood and immune cells. They are sometimes transplanted into patients with certain blood or immune disorders or cancers whose own cells have been wiped out by chemotherapy, essentially restocking the recipients with healthy cells.

But recipients of these transplants sometimes experience graft versus host disease (when the transplant attacks the recipients tissues), heart or immune conditions, or even secondary cancers. Some experts have suspected these conditions might be caused by mutations in donor stem cells, among other factors. Its in part why they favor younger donors, who are expected to have fewer mutations than older donors. (Donors from 18 to 44 account for 86% of transplants for unrelated patients. Relatives often make for better donors because they are more likely to be matched to recipients based on immune system molecules.)

Most of these mutations are probably benign. But its possible that other mutations not only pose a health risk, but also give their host cells a boost over other cells, helping them proliferate over time. That might mean someone who is 40 could have a bad mutation in one in 5,000 cells, but by the age of 50, it could be in one in 50 cells, Druley explained.

The challenge is detecting those mutations. Standard sequencing technology may pick up mutations if they appear in just a small percentage of cells, but for young donors, it would be like finding the few pebbles in a beach full of sand.

Next-generation sequencing is good if you want to find a mutation thats in 20% of cells youre looking at, or even 5%, Druley said. Were looking at mutations that are one in a thousand, or down to one in ten thousand.

For the new study, Druley and colleagues trained a more powerful tool they call error-corrected sequencing on the cells of the 25 donors, who ranged from 20 to 58 years old. (Fifty-eight would be considered older donors, but the median age of the donors in the study was 26.) They looked for mutations in 80 genes in particular, including genes that, when mutated, are associated with leukemia.

What they found: 11 donors had a collective 19 mutations that were not picked up by standard sequencing technology 16 of which were pathogenic, meaning disease causing.

The researchers also studied the recipients, finding that 14 of the 19 mutations had engrafted, or been taken up by the recipient and started to generate other cells, and were still there a year after the transplant. Thirteen of these mutations were pathogenic.

Researchers said it made sense that younger adults had these types of mutations, even if scientists hadnt previously been able to spot them.

Its known that mutations accumulate over time, said Dr. Ross Levine, a leukemia specialist at Memorial Sloan Kettering Cancer Center, who was not involved in the new study. The question has always been in other scenarios if you can detect these clones at earlier or in different contexts, and if they mean anything.

The new study was not set up to answer that last question.

Thats the next phase of this research, Druley said. Some of these mutations, if theyre going to have an effect, may not have an effect for many, many years. And we had a small population. So we didnt have enough numbers or enough time.

Outside researchers said that if these stem cell mutations do contribute to diseases once transferred to recipients, it might take so long or happen so infrequently that it would be difficult to study. Dana-Farbers Cutler said that, for example, cases of donor-derived leukemia when the recipient develops blood cancer after a transplant occur in less than 1% of transplants from unrelated donors.

If the mutations do increase the likelihood of complications in recipients, then why arent they posing a problem for the donors themselves? That might also be tied to the prevalence of these mutations, the researchers said.

In donors, these mutations might appear in, say, 1% of cells, and there is competition among cells of all different types of genetic variants to multiply. Plus, donors have healthy immune systems that can help suppress bad actors.

But if cells with these mutations make their way into recipients who have had their own cells blasted away with chemotherapy, its a new race. The mutations might give them some advantage to multiply faster than other cells that were transplanted. And as the percentage of cells with these mutations rises, they might be more likely to cause disease.

When you have these mutations in a host, it may take decades for them to expand to the point of causing issues, Levine said. In a recipient, however, its almost like youve reset the playing field.

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Study: Mutations in stem cells of young donors can be passed to recipients - STAT

Bone Marrow Stem Cells Comments Off on Study: Mutations in stem cells of young donors can be passed to recipients – STAT | January 17th, 2020

In November 2018, Chinese biophysics researcher He Jiankuimade a historic announcement.

Two twin girls nicknamed Lulu and Nana had become the worlds first genetically modified human beings.

Using a gene-editing technology known as CRISPR, He had manipulated the DNA of the embryos that would become the girls in an effort to make them immune to the HIV virus.

What first seemed like a historic triumph of science, however, quickly became one of the most infamous scandals in medical history.

The researcher was swiftly fired from his university, put under police investigation, and denounced by experts around the world who said he jumped the gun and carried out an experiment that was unsafe and unethical.

In December, He was sentenced to three years in prison for illegally carrying out human embryo gene-editing intended for reproduction. Its unclear whether the experiment caused any genetic damage to Lulu and Nana or if they are even resistant to the HIV virus.

Kiran Musunuru, one of the worlds foremost genetics researchers, was the first expert to publically condemn Hes experiment.

Nonetheless, Musunuru says the birth of the Chinese twins marks the beginning of a new human era, the possibilities of which are boundless.

Potential future implications of gene-editing technology range from preventing genetic diseases to producing designer babies with custom traits to creating superhumans and controlling our own evolution.

With the release of his new book, The CRISPR Generation: The story of the Worls First Gene-Edited Babies, The Globe Posts Bryan Bowmanspoke to Musunuru about where this technology could go from here and what it could mean for the future of humanity.

The following interview is lightly condensed and edited for length and clarity.

Bowman: Could you explain what CRISPR is broadly and how that technology evolved to where it is today?

Musunuru: CRISPR is one type of gene-editing tool. Gene editing is a technology that allows us to make changes to genes in the DNA and in the cells in the body. If were talking about human beings, typically were talking about changes that are related to health or disease.

There are several types of gene editing tools, but CRISPR is by far the most popular one. CRISPR is interesting because it wasnt invented. It actually exists naturally in all sorts of bacteria. It evolved as a sort of an immune system that can fight off viral infections. Just like we can get viral infections, it turns out bacteria can get viral infections as well. And so bacteria created a system by which they can fight off viruses. So thats where CRISPR came from.

Over the past couple of decades, a variety of very talented scientists identified it, discovered it in bacteria, and then were able to adapt it into a gene-editing tool that can now be used in human cells.

What we can do with CRISPR is either turn off genes and thats easier to do or we can make more precise changes to genes such as correcting a mutation that causes disease.

Bowman: Last year, there was the famous or infamous case where Dr. He Jiankui in China covertly created the first gene-edited babies. And I understand that you were the first expert to publicly condemn the experiment. What exactly did Dr. He do and why did you feel it was so unethical?

Musunuru: What he was trying to do was use CRISPR to turn off a gene called CCR5. By turning off this gene, he was hoping to make the babies that were born resistant to HIV infection, HIV being the virus that causes AIDS.

There are many people who are naturally born with this chain turned off and theyre resistant to HIV. So the rationale was, well, Im going to try to create babies who have the same trait.

What he did was problematic for two reasons. One, it was, to put it lightly, a scientific disaster. Everything you worry about going badly with CRISPR actually did happen. Any technology has a potential for a lot of good with the potential for bad. I compare it to fire. It can be very useful. But if youre not careful, it can cause wildfires and a lot of damage and hurt a lot of people. Its the same with CRISPR. It can do a lot of good. It can help patients who have bad diseases. But if youre irresponsible with it, it could actually cause unintended genetic damage.

Its not clear whether these kids that were born they were twin girls nicknamed Lulu and Nana its not clear whether theyre actually protected against HIV infection. Its not clear whether they might have suffered some genetic damage that might have health consequences for them. Its not clear whether the genetic damage if it did occur could get passed down to their children and affect future generations.

So scientifically, there are a lot of problems with it. The work was very premature. I would say that if we were ever going to do this in a reasonable, rational, safe way, were years away from doing it. But he went ahead and just did it anyway. You can call him a rogue scientist, as clich as it is. And he did it in conditions of secrecy. There was essentially no oversight. And potentially these twins and future generations might suffer the consequences.

The other problem is a problem of ethics. The way in which he did it basically violated every principle of ethical medical research in the textbook. Basically, everything that you could do wrong, he did it wrong.

Whenever we do an experimental procedure, we hope that the benefits greatly outweigh the risks. What he was trying to do was protect these kids from HIV. But the truth is, they were in no particular danger of getting HIV compared to the average person. In China, the prevalence of HIV is about 0.1 percent. So there wasnt really much for them to gain. Even if they did somehow during their lifetime get the HIV infection, we have good treatments to prevent it from proceeding to full-blown AIDS.

So what was the benefit of doing this procedure? You have to balance that against the harms. And the genetic damage thats possible that raises risks of things like cancer and heart disease and other diseases. When you have those risks and very little benefit, then its just not a favorable ratio. And thats intrinsically unethical.

Bowman: Seeing as you said that were years away from doing something like this in a more responsible and ethical way, what are the greatest challenges to getting to a point where parents will have the option to go forth with a gene-editing procedure that might prevent their children from suffering from some kind of genetic disease?

Musunuru: There are really two aspects to this. One is a scientific or medical aspect. Can we get to a place where gene-editing of embryos is well-controlled? Where we know that what were doing is truly safe and appropriate from that perspective?

The second issue is really a decision more for broader society. Is this something that we should be doing, something we want to be doing? This is less about the science and more about ethics and morality and legality and religious values and all sorts of other things. Reasonable people can disagree on whats appropriate and whats not appropriate.What complicates things here is that its not really an all or nothing decision. There are different scenarios where you could see parents using gene-editing on behalf of their unborn children.

I like to break it down is three scenarios. The first scenario is with parents who have medical issues that make it so that theres no way they can have natural biological children or healthy babies if they both have a bad disease and theyre going to pass it on to all of their kids unless you do something like editing. These are unusual situations, but they do exist.

The second scenario is one where parents might want to quite understandably reduce the risk of their child having some serious illness at some point in their lifetime. Im talking about things that are fairly common, like Alzheimers disease or breast cancer or heart disease or whatnot. Theres no guarantee that the editing will eliminate that risk. But you can see how parents might want to stack the odds in their kids favor. Its still medical, but its not perhaps as severe a situation with a kid whos definitely going to get the disease unless you do something.

The third scenario would be cases in which parents want to make changes that are not really medical but are more of what we would think of as enhancements. These could be cosmetic changes like hair color, eye color, things like that.

But it could potentially be much more serious things like intelligence or athletic ability or musical talent. Now, to be fair, thats theoretical. I dont think we are anywhere near knowing enough about how genes influence these things to be able to do it anytime soon. You might actually have to change hundreds of genes in order to make those changes. But you can imagine how certain parents might want to do that, might want to advance their children in the ways that they feel personally are desirable.

Bowman: Can gene editing only be performed on embryos or is it possible to edit genes in later stages of pregnancy or even post-birth?

Musunuru: Theres actually a lot of exciting work going on using gene editing to help patients, whether its adults or children. Right now its been focused mostly on adults who have terrible diseases and its really being used as a treatment to alleviate their suffering or potentially cure the diseases.

Just recently, we got the exciting news that two patients one in the U.S. and one in Europe were participating in a clinical trial. They each had a severe blood disorder. One of them had sickle cell disease. The other had a disease called beta-thalassemia. Earlier this year, they got a CRISPR-based treatment. And whats very exciting is that it looks like not only have their conditions improved significantly, it looks like they might actually be cured.

If that bears out, it would really be historic because these are diseases that affect millions of people around the world and were previously incurable. This treatment is also being explored for things ranging from cancer to liver disease to heart disease.

So theres enormous potential for benefit for living people who have serious diseases. But its a very different situation than editing embryos because youre talking about a person who is in front of you. We are trying to alleviate their suffering. That patient has the ability to freely give consent to the procedure, to weigh the benefits and risks and come up with a decision.

Bowman: How does that work? Is it some kind of cell transplant where the new cells then replicate throughout the rest of the body?

Musunuru: Yeah. It depends on the situation. I mentioned those two patients with the blood disorders. The way it worked there was the medical team used bone marrow stem cells. They basically took bone marrow as if they were going to do a transplant and then edited blood stem cells in a dish outside of the body to fix the genetic problem. And then they took those edited stem cells and put them back into the same patient. Those cells start making the blood cells that are now corrected or repaired. And by doing that, to cure the disease.

Another potential implementation is I work on heart disease. And what wed like to be able to do is turn off cholesterol genes in the liver. So what I envision is that a patient with heart disease would get a single treatment and it would deliver CRISPR into the liver and just the liver. It would turn off genes that produce cholesterol in the liver. The effect of that is permanent reduction of cholesterol levels and lifelong protection against heart disease.

This actually works really well in mice. Ive been working on this in my own laboratory for six, almost seven years now experimenting with it in monkeys. And if looks like it works and Im pretty confident that it will work we could be looking at clinical trials in a few years where were taking patients who have really bad heart disease or a very high risk for heart disease and actually giving them the single treatment within their own bodies that would turn off these cholesterol genes.

Bowman: In terms of more cosmetic applications, theres this popular idea that designer babies will be a reality at some point in the future. But how feasible would it be to use gene-editing for something very basic like choosing eye color or hair color? Are there many genes involved in determining traits like that? Are we close to being able to do that if we choose to?

Musunuru: Well, eye color, hair color, those actually turned out to be fairly simple. Theres only a small number of genes that control those. So in theory, if you wanted to do it, it wouldnt be that difficult.

Personally, my point of view is thats a trivial thing. Like why would you go through all that trouble? Do I care if your kid has blue eyes versus green eyes versus brown eyes? Maybe some parents feel that thats very important. So I think simple things like hair color, like eye color, it could be done fairly readily. I just dont see it as serious enough to warrant doing it.

The more complex things like intelligence, gosh, thats going to be so challenging. I mean, intelligence is just such a complex phenomenon. Theres some genetics involved in it, but there are so many other factors that come into that like upbringing and environment. Were not even getting close to an understanding of how someones intelligence comes about, to be perfectly honest about it.

I will point out that even though some of these things are simpler, in general, the vast majority of people are very, very uncomfortable with the idea of using gene editing of embryos for enhancements.

And I think this reflects a couple of things. I think this reflects the fact that people are more sympathetic if something like this is being used for medical purposes and much less comfortable if its being done to give a child an advantage in a way thats not medical.

It brings to mind the recent scandal where wealthy parents were trying to get their kids into good colleges by actively bribing admissions officers, faking test scores, fabricating resums. That kind of thing makes people very uncomfortable that certain people, particularly wealthy people, might try to use this technology to an extreme to advantage their children.

Theres an economic aspect to that. Wealthy parents might have better access to this technology than those who are not as wealthy. And what does that mean? If wealthy parents are somehow able to make designer babies who somehow are advantaged and other people are not, does that exacerbate socio-economic inequalities in our society?

So I think there are a few reasons why people are uncomfortable with the idea of enhancement, whereas on the whole, the majority seem to be at least somewhat open to the idea that there might be good medical uses.

Bowman: Im really happy that you brought up that socio-economic inequality aspect because I was going to ask you about that. But if we table those concerns for a moment and go way out there, theres this notion you write about that we could ultimately, theoretically, control our own evolution.

Ive heard it suggested that it could be theoretically possible to incorporate traits from other organisms that could be advantageous into our own DNA and essentially enter a new post-human stage of evolution. Is that total science fiction or do you think were entering a period where that is increasingly possible?

Musunuru:Well, with the way things are going with this technology. I mean, weve taken a step towards that. But there are many, many, many, many steps that would need to be taken to actually get to that point. But I think youre right. You see the path. We have the technology. Then its a question of perfecting the technology. A question of learning more about what genes from other species might be advantageous.

The cats out of the bag. The technology is here. Whether its five years from now or 10 years from now or 50 years from now or 100 years from now, these sorts of things will inevitably start to happen. And Im not sure theres much that those who would like to not see that happen will be able to do to stop it in the long run.

China Jails Scientist Who Gene-Edited Babies

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Controlling Our Own Evolution: What is the Future of Gene-Editing? - The Globe Post

Bone Marrow Stem Cells Comments Off on Controlling Our Own Evolution: What is the Future of Gene-Editing? – The Globe Post | January 17th, 2020

Researchers have developed cytochalasin B-induced membrane vesicles which they suggest could be a new form of cell-free therapy in regenerative medicine.

Work on extracellular microvesicles (ECMVs) derived from human mesenchymal stem cells (MSCs) has revealed a potential new form of cell-free therapy.

ECMVs are microstructures surrounded by a cytoplasm membrane; they have proven to be a prospective therapeutic tool in regenerative medicine due to their biocompatibility, miniature size, safety and regenerative properties. These can be used to circumvent the limitations of existing cell therapies without losing any effectiveness.

Cell therapies are grafts or implants of living tissue, such as bone marrow transplants, used to replace and regenerate damaged organ tissue. They currently have limited applications, as they work differently dependent on conditions and the environment they are placed into. They can also be rejected by the immune system.

A study at Kazan Federal University, Russia, has investigated cytochalasin B-induced membrane vesicles (CIMVs) which are also derived from MSCs and are very similar to natural ECMVs.

Proteome analysis of human MSCs and CIMVs-MSCs. Venn diagram of identified proteins MSCs and CIMVs-MSCs (A). Distribution of the identified proteins in organelles, percent of unique identified proteins (B) (credit: Kazan Federal University).

The scientists studied and characterised the biological activity of MSC-derived CIMVs. A number of biologically active molecules were found in CIMVs, such as growth factors, cytokines and chemokines; their immunophenotype was also classified.They also found that CIMVs could stimulate angiogenesis in the same way as stem cells.

The team came to the conclusion that human CIMVs-MSCs can be used for cell-free therapy of degenerative diseases. Induction of therapeutic angiogenesis is necessary for the treatment of ischemic tissue damage (eg, ischemic heart disease, hind limb ischemia, diabetic angiopathies and trophic ulcers) and neurodegenerative diseases (eg, multiple sclerosis and Alzheimers disease), as well as therapies for damage of peripheral nerves and spinal cord injury.

The group say they are continuing to research the therapeutic potential for artificial microvesicles for autoimmune diseases.

The study was published in Cells.

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Novel form of cell-free therapy revealed by researchers - Drug Target Review

Bone Marrow Stem Cells Comments Off on Novel form of cell-free therapy revealed by researchers – Drug Target Review | January 17th, 2020

Phoebe Roskell was just four-years-old when she became very unwell with bone marrow failure.

She became blood and platelet transfusion dependent and was told she needed a bone marrow transplant

Mum Jacquie Roskell, from Catterall, said after many hospital appointments and tests, doctors discovered that Phoebe had a rare and currently incurable disease called Dyskeratosis Congenita (DC).

The telomere biology disorder only affects one in a million people worldwide in which the telomeres - the caps at the end of each strand of DNA - are not present and therefore the DNA becomes damaged and cells cannot do their job.

This can lead to the premature ageing of cells and can cause life-threatening complications as well as shortened life expectency.

Jacquie Roskell, 43, from Catterall, told LancsLive: "In May 2017 Phoebe, then aged just 4-years-old, became very unwell and was found to be in bone marrow failure. She became blood and platelet transfusion dependent and we were told she would need a bone marrow transplant.

"In August of that year and after many tests it was discovered she had the rare and currently incurable disease Dyskeratosis Congenita (DC).

"DC is a telomere biology disorder and only affects one in a million people worldwide. Telomeres are the caps at the end of each strand of DNA that protect our chromosomes, like the plastic tips at the end of shoelaces. Without the coating, shoelaces become frayed until they can no longer do their job.

"Just as without telomeres, DNA strands become damaged and our cells cant do their job. This in turn causes premature ageing of cells such as bone marrow hence why Phoebes failed. Telomeres naturally shorten as we age, but Phoebe was born with exceptionally short telomeres like those of a 100-year-old woman."

Many life threatening complications can be cause by DC, such as liver and lung failure, higher chances of cancer and shortened life expectancy, says Jacquie.

Children with this mutation are unlikely to live longer than 16-year-old.

"There are currently 15 gene mutations identified with this disease and the one Phoebe has is called TINF2," Jacquie explained.

In December 2017, Phoebe's older brother Woody heroically donated his stem cells for her bone marrow transplant at The Royal Manchester Childrens Hospital. He saved her life.

The bone marrow failure was successful, however the disease had not yet been cured.

Jacquie, 43, said: "The treatment was gruelling, and her recovery was long and exhausting. The bone marrow failure was successfully cured but the disease is not.

"Phoebe suffers with episodes of extreme fatigue and exhaustion, joint pains, headaches and migraines. Injuries such as a sprained wrist take longer to heal for Phoebe, and she is still very susceptible to common viruses following her transplant.

"She attends many hospital appointments always in Manchester and is regularly seen by different medical teams including Haematology, Respiratory, ENT, Ophthalmology, Dental and Oral Medicine."

"Phoebe has suffered so much in her very short life. But when she is feeling well she is like a pocket rocket and there is just no stopping her. When she feels unwell she crashes and needs time to recover again,"

"The disease is unpredictable and although much research has been done into Telomere biology disorders there is still a long way to go.

"Much research is being carried out by the incredible charity Team Telomere - that support us as a family. Their aim is to hopefully find a cure for this cruel disease and fingers crossed it is in Phoebes lifetime."

That is why Jacquie is helping to fundraise for Team Telomere.

The charity supports families worldwide in their battle with DC and related Telomere Biology Disorders who often face multiple complex illnesses such as bone marrow failure, lung fibrosis, cancer, and many other challenges.

It also helps to encourage the medical communitys research in finding causes and effective treatments, and to facilitate improved diagnosis by educating medical providers.

To help raise money for Team Telamore, Jacquie is co-organising a pre-loved wedding sale along with her friend Samantha Smith who is the events manager of the Wyrebank in Garstang.

The sale will be held at Wyrebank on the Moorings on Sunday, March 1 from 11pm until 1pm.

Samantha told LancsLive: "The idea is for brides and businesses to sell their wedding items to future wedding couples.

"People can also donate wedding dresses and bridesmaid or we will take a percentage of the sales, and sell them for them."

Bridal stalls cost 15 and business stalls are 40.

All proceeds will be going to Team Telomere.

Alternatively, donations can be made directly via the website here.

You can also find more details on the Wyrebank pre-loved wedding sale here.

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The young girl diagnosed with 'one in a million' genetic disease - LancsLive

Bone Marrow Stem Cells Comments Off on The young girl diagnosed with ‘one in a million’ genetic disease – LancsLive | January 17th, 2020

Autologous stem cell and non-stem cell based therapies involve an individuals cell to be cultured and then re-introduced to the donors body. These therapies do not use foreign organism cells and are therefore free from HLA incompatibility, disease transmission, and immune reactions.Increasing demand for the new therapies in the field of regenerative medicine is directly facilitating the growth of autologous stem cell and non-stem cell based therapies market. Furthermore, since the risk to transplantation surgeries is significantly reduced in these therapies, they are increasingly being preferred for treatment of bone marrow diseases, aplastic anemia, multiple myeloma, non-Hodgkins lymphoma, Hodgkins lymphoma, Parkinsons disease, thalassemia, and diabetes.

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Moreover, rising incidents of cancer, diabetes and cardiovascular diseases along with growing geriatric population is another factor attributed for its high growth. However, side-effects of autologous stem cell and non-stem cell based therapies such as nausea, infection, hair loss, vomiting, diarrhea, etc. are expected to affect the market to an extent. High cost is another factor that can act as challenge to autologous stem cell and non-stem cell based therapies market. In spite of this, less risk post transplantation surgeries and favorable tax reimbursement policies are anticipated to reduce the impact of these limitation during the forecast period.Autologous stem cell and non-stem cell based therapies market can be segmented on the basis of application, end-user, and region.

In terms of application, the autologous stem cell and non-stem cell based therapies market can be segmented into blood pressure (BP) monitoring devices, intracranial pressure (ICP) monitoring devices, and pulmonary pressure monitoring devices. In terms of end-user, the market can be segmented into ambulatory surgical center and hospitals. By region, the market can be segmented into North America, Europe, Asia Pacific, Middle East and Africa and South America. Amongst all, Asia Pacific is anticipated to be the most attractive market owing to favorable reimbursement policies in the region.The players operating in autologous stem cell and non-stem cell based therapies market are limited.

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They are consistently involved in research and development activities for product development to keep up with the growing competition, thereby aiding the growth of autologous stem cell and non-stem cell based therapies market across the world.

The major players operating in autologous stem cell and non-stem cell based therapies market are Regennex, Antria(Cro), Bioheart, Orgenesis Inc., Virxys corporation , Dendreon Corporation, Tigenix, Georgia Health Sciences University, Neostem Inc, Genesis Biopharma, Brainstorm Cell Therapeutics, Tengion Inc., Fibrocell Science Inc., Opexa Therapeutics Inc, Regeneus Ltd, and Cytori Inc., among others.

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Autologous Stem Cell And Non Stem Cell Based Therapies Market Extracts Market, 2018-2026 by Segmentation Based on Product, Application and Region ...

Bone Marrow Stem Cells Comments Off on Autologous Stem Cell And Non Stem Cell Based Therapies Market Extracts Market, 2018-2026 by Segmentation Based on Product, Application and Region … | January 17th, 2020

Stafford Township, NJ Stem cell therapy is an incredible process for healing damaged tissue, so it seems remarkable that it is availablefor petsright here in Manahawkin. Stafford Veterinary Hospital, at 211 North Main St., began offering the advanced treatment in 2019, under the direction of Michael Pride, medical director at the facility.

There, stem cell therapy is most commonly applied to osteoarthritis, but can also be used in dogs suffering from hip dysplasia and ligament and cartilage injuries, as well as mobility ailments and some chronic inflammatory issues such as inflammatory bowel disease and chronic kidney disease, which is common in cats.

Stem cell therapy is actually the only thing that can help to reverse the process of arthritis, Pride said. Everything else is a Band-Aid.

This process can actually help to rebuild cartilage and really reduce inflammation without the need of using aspirin-type medications, Pride said. Its a newer technology that we can use to avoid chronic use of medications, which might actually be detrimental in the long term for the liver or kidneys.

Stem cell therapy treats the source of the problem by offering the ability to replace damaged cells with new ones, instructs the website staffordvet.com.

Stem cells are powerful healing cells in the pets body that can become other types of cells. For example, in the case of arthritis, stem cells can become new cartilage cells and have natural anti-inflammatory properties, thus reducing pain and increasing mobility.

The stem cells are your primary structural cell for all other cells in the body; they can differentiate into almost any other cell, explained Pride. Were processing it down into that primordial stem cell; were activating it, and were injecting it into where it needs to be, and it just starts taking on the characteristics of the cells around it.

Table-top machines from MediVet Biologics are the first Adipose Stem Cell therapy kits for in-clinic use, a major advancement. Stem cell therapy for animals has been commercially available since 2004. MediVet pioneered in-clinic treatment options around 2010.

Pride believes Stafford Veterinary Hospital offers the only such treatment in the immediate area; another is in Egg Harbor Township, Atlantic County.

Were always trying to figure out different ways to help the patient without hurting them, he said while petting a kitten that had been a patient for another type of treatment.

As stem cell therapy is more in the news regarding humans, a pet owners first question might be where the stem cells come from that are used in the process. The answer: from fat tissue of the pet itself, extracted and processed the same day.

As the therapy has been refined in the last decade, it has actually started to become a lot easier, more cost-effective more recently, said Pride, since weve been able to process fat tissue instead of actually getting bone marrow.

Fat tissue actually has a much higher concentration of adult stem cells than bone marrow does, so its less painful for the patient, they heal a lot easier, and we dont have to process it in a different facility.

Everything comes from the animal, and we give it back to the animal. Nothing comes from another animal. We dont have to worry about them rejecting the sample; its their own tissue, and were giving it back to them.

The pet typically goes home the same day after about eight hours. First, X-rays and a consultation with the veterinarian can determine whether the pet is a candidate for the treatment.

A pet owner may not even know that their animal has arthritis.

Cats have a lot of inflammatory issues that they tend to be very good at hiding, said Pride. A lot of people dont realize that they have arthritis. They think, oh, my cats just getting older; hes not jumping as much; hes not as strong; hes just sleeping most of the day, but actually he has arthritis. Its very difficult to diagnose in cats. A lot of times you end up having to do X-rays to find where the arthritic joints happen to be.

An inch-and-a-half incision is the minor surgery that harvests the fat tissue from the belly while the pet is anesthetized. For a cat, about 20 gramsare extracted. For a large dog, about 40 gramsare needed. While the pet is recovering from the incision surgery, the veterinary hospital is processing the sample. When the sample is ready, the pet is sedated because we then have to give them the joint injections. Then we can reverse the sedation, and they go home.

We asked the doctor if the process always works. He gave the example that on average, a dog such as a boxer that was hobbled is now able to walk without seeming like its painful. In an extreme positive case, a dog that had been barely walking might be bouncing all over the place in two months.

It doesnt always work to the extent that we would love it to, but we usually notice that there is a positive effect from it, Pride remarked. Every patient will be different in what they experience.

For the same reason that everyones situation is going to be different, cost of treatment was not given for this story.

It generally takes about 30 to 60 days for relief to show, the veterinarian said, and the animals progress will be monitored.

On average, results last about 18 months to two years before more stem cells might have to be injected. The procedure takes about an hour.

The nice thing is once we collect those stem cells (from the first procedure), we can bank the leftovers they are cryogenically stored at MediVet corporate headquarters in Kentucky and we dont have to go through the initial anesthetic surgery, said Pride.

Stem cell therapy is one of several innovative modalities available at Stafford Veterinary Hospital. Laser therapy, acupuncture and holistic medicine are others. Care for exotic pets is available, as is emergency pet care.

Visit the website staffordvet.com or call 609-597-7571 for more information on general and specialized services, including: vaccinations, microchipping, spayingand neutering, dental care, wellness exams, dermatology, gastrology, oncology, opthalmology, cardiology, soft-tissue surgery, ultrasound, radiography, nutrition, parasite control, boarding, laborand delivery, end-of-life care, and cremation.

Stafford Veterinary Hospital has been in business since 1965, founded by Dr. John Hauge. Today, five highly skilled veterinarians are on staff, and a satellite, Tuckerton Veterinary Clinic, is at 500 North Green St. in Tuckerton.

Pride has been medical director at Stafford Veterinary Hospital since 2008. He attended Rutgers University, then earned his Veterinary of Medicine degree at Oklahoma State University.

The mild-mannered doctor feels a great rewardfrom treating animals that cant speak for themselves when they feel bad.

These guys, theyre always thankful; you can see what they think, he said of treated pets. The turnaround in their attitude, the turnaround in their ability to be more comfortable, you can see it in their faces; you can see it in their actions. You learn to read animals over time.

Its knowing that were helping those who cant help themselves, he added, and you can see it in them; thats the most gratifying.

mariascandale@thesandpaper.net

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Stem Cell Therapy for Dogs and Cats Is Innovative at Stafford Veterinary Hospital - By MARIA SCANDALE - The SandPaper

Bone Marrow Stem Cells Comments Off on Stem Cell Therapy for Dogs and Cats Is Innovative at Stafford Veterinary Hospital – By MARIA SCANDALE – The SandPaper | January 16th, 2020

LONDON, UK / ACCESSWIRE / January 15, 2020 / Hemogenyx Pharmaceuticals plc (HEMO.L) ("Hemogenyx" or the "Company"), the biopharmaceutical group developing new therapies and treatments of blood diseases, is pleased to announce the following update on its activities.

As previously announced, Hemogenyx's CDX product has the potential to treat Acute Myeloid Leukemia (AML) directly as well as providing a benign conditioning regimen for blood stem cell replacement therapy. The Company has now carried out extensive work developing treatments for AML and has to date obtained encouraging results.

Hemogenyx has successfully constructed and in vitro tested Chimeric Antigen Receptor (CAR) programmed T cells (HEMO-CAR-T) for potential treatment of AML. HEMO-CAR was constructed using Hemogenyx's proprietary humanized monoclonal antibody against a target on the surface of AML cells. The Company has demonstrated that HEMO-CAR was able to programme human T cells (converted them into HEMO-CAR-T) to identify and destroy human AML derived cells in vitro.

Following the successful completion of these tests, in vivo tests of the efficacy of HEMO-CAR-T against AML are being conducted utilising a model of AML using Advanced peripheral blood Hematopoietic Chimera (ApbHC) - humanized mice developed by Immugenyx, LLC, a wholly-owned subsidiary of Hemogenyx.

Vladislav Sandler, Chief Executive Officer, commented, "We are encouraged by this new data which demonstrates our continuing progress in the development of novel treatments for blood cancers such as AML. The development of HEMO-CAR-T expands Hemogenyx's pipeline and advances it into a cutting-edge area of cell-based immune therapy. We are excited to have developed another product candidate that should, if successful, provide a new and potentially effective treatment for blood cancers for which survival rates are currently very poor."

About AML and CAR-T

AML, the most common type of acute leukemia in adults, has poor survival rates (a five-year survival rate of less than 25% in adults) and is currently treated using chemotherapy, rather than the potentially more benign and effective form of therapy being developed by Hemogenyx. The successful development of the new therapy for AML would have a major impact on treatment and survival rates for the disease.

CAR-T therapy is a treatment in which a patient's own T cells, a type of immune cell, are modified to recognize and kill the patient's cancer cells. The procedure involves: isolating T cells from the patient, modifying the isolated T cells in a laboratory using a CAR gene construct (which allows the cells to recognize the patient's cancer); amplifying (growing to large numbers) the newly modified cells; and re-introducing the cells back into the patient.

Market Abuse Regulation (MAR) Disclosure

Certain information contained in this announcement would have been deemed inside information for the purposes of Article 7 of Regulation (EU) No 596/2014 until the release of this announcement.

Enquiries:

Hemogenyx Pharmaceuticals plc

http://www.hemogenyx.com

Dr Vladislav Sandler, Chief Executive Officer & Co-Founder

headquarters@hemogenyx.com

Sir Marc Feldmann, Executive Chairman

SP Angel Corporate Finance LLP

Tel: +44 (0)20 3470 0470

Matthew Johnson, Vadim Alexandre, Soltan Tagiev

Peterhouse Corporate Finance Limited

Tel: +44 (0)20 7469 0930

Lucy Williams, Duncan Vasey

US Media enquiries

Tel: +1 (323) 646-3249

Lowell Goodman

lowell@corbomitecomms.com

About Hemogenyx Pharmaceuticals plc

Hemogenyx Pharmaceuticals plc ("Hemogenyx") is a publicly traded company (HEMO.L) headquartered in London, with its wholly-owned US operating subsidiaries, Hemogenyx LLC and Immugenyx LLC, located in New York City at its state-of-the-art research facility and a wholly-owned Belgian operating subsidiary, Hemogenyx-Cell SPRL, located in Lige.

Hemogenyx is a pre-clinical stage biopharmaceutical group developing new medicines and treatments to bring the curative power of bone marrow transplantation to a greater number of patients suffering from otherwise incurable life-threatening diseases. Hemogenyx is developing several distinct and complementary product candidates, as well as a platform technology that it uses as an engine for novel product development.

For more than 50 years, bone marrow transplantation has been used to save the lives of patients suffering from blood diseases. The risks of toxicity and death that are associated with bone marrow transplantation, however, have meant that the procedure is restricted to use only as a last resort. Hemogenyx's technology has the potential to enable many more patients suffering from devastating blood diseases such as leukemia and lymphoma, as well as severe autoimmune diseases such as multiple sclerosis, aplastic anemia and systemic lupus erythematosus (Lupus), to benefit from bone marrow transplantation.

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Hemogenyx's CAR-T Cells are Effective Against AML in vitro - Yahoo Finance

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Cancer enters your body when cells begin to grow out of control. There are various types of cancer and cells in almost every part of the body can become cancer. Leukemia is a type of cancer which starts in the cells, then develops into different types of blood cells. It starts in early forms of white blood cells. There are different types of leukemia which can be divided into acute and chronic. Acute is fast growing and chronic is slow growing.

An Acute Lymphoblastic Leukemia is a type of leukemia which progresses quickly and if not treated, will be fatal in a couple of months. Acute means fast growing and lymphatic means it develops from the early forms of lymphocytes, which is a type of white blood cell. It all starts in the bone marrow and leukemia cells start to invade the body quickly. They can spread to other parts of the body. Some cancers also start in the organs and then spread to the bone marrow, but they are not leukemia.

There are other types of cancer which start in lymphocytes and are known as lymphomas. Leukemias affect blood and bone marrow and lymphomas affect lymph nodes and other organs. It can sometimes be difficult to tell if a cancer of lymphocytes is lymphoma or leukemia. If at least 20% of the bone marrow has cancerous lymphocytes, the disease is considered to be leukemia. Acute Lymphoblastic Leukemia is the most common childhood cancer and children below the age of five are at the highest risk. It can also occur in adults.

RELATED:Kids Born To Obese Mothers Are More Likely To Develop Leukemia

ALL can increase the chances of bleeding and developing infections in the body. Its symptoms include:

In order to diagnose ALL, the doctor must complete a physical exam and also conduct bone marrow tests and blood tests. Doctors are likely to ask about bone pain, since it is the most common symptom of ALL. Here are a few tests doctors carry out.

The doctor might order a blood count, and people who have ALL may have a blood count which shows low platelet count and a low hemoglobin count. The WBC may or may not have increased. A blood smear might show immature cells circulating in the blood, which are usually found in bone marrow.

This process involves taking a sample of the bone marrow from your breastbone or the pelvis. It is an ideal way to test for increased growth in marrow tissue and reduced production of red blood cells.

An X-ray of the chest can allow the doctor to see if the mediastinum, that is the middle partition of the chest is widened. Further, a CT scan can help the doctor estimate whether the cancer has spread to the spinal cord, brain or to any other part of the body.

There are other tests like a spinal tap, which is used to check if cancer cells have spread around the spinal fluid. Tests on the serum urea and liver function might also be done.

The treatment will help bring the count back to normal. When this happens and the bone marrow looks normal, the cancer is in remission. Acute Lymphoblastic Leukemia can be treated through chemotherapy. You might be asked to stay at the hospital for a few weeks in the first treatment. Later, you can continue the treatment as an outpatient.

For those with a low WBC count, you will be asked to spend time in an isolation room. It ensures that you are protected from contagious diseases and other problems. If leukemia does not respond to chemotherapy, a bone marrow or stem cell transplant might be recommended. The transplanted marrow can be taken from a sibling who is a complete match.There are high chances of cancer remission in case of children.

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Criss Angel's Son Has Acute Lymphoblastic Leukemia, But What Is It? - Moms

Bone Marrow Stem Cells Comments Off on Criss Angel’s Son Has Acute Lymphoblastic Leukemia, But What Is It? – Moms | January 16th, 2020

Tacitus Therapeutics exclusively licenses technology for expansion, differentiation and engineering of hematopoietic stem cells for use in therapeutic applications

NEW YORK, Jan. 9, 2020 /PRNewswire/ -- Tacitus Therapeutics, a clinical-stage company, has launched in collaboration with the Mount Sinai Health System to develop stem cell therapies initially targeting blood cancers and related clotting disorders. Their first therapy, HSC100, currently is being investigated in a Phase I clinical trial1.

Tacitus is building upon technology developed by and exclusively licensed from Mount Sinai. Based on research by scientific co-founders Ronald Hoffman, M.D., and Camelia Iancu-Rubin, Ph.D., the technology includes proprietary cell expansion, differentiation and engineering methods. Together, these methods manufacture healthy cells that overcome the limitations of traditional allogeneic, or donor, cell transplantations.

Blood cancers comprise about 10% of new cancer cases in the U.S. each year, and almost 60,000 people die from blood cancer complications annually. Most blood cancers start in the bone marrow, where blood is produced. A common therapy for such blood cancers is a hematopoietic stem cell (HSC) treatment or, as more commonly referred to, bone marrow transplantation. In this process, doctors infuse healthy HSCs into the patient's bloodstream, where they migrate to the bone marrow to grow or engraft.

HSCs for this process can be collected from bone marrow, circulating blood, or umbilical cord blood (CB) of healthy donors. While HSC transplants are common, significant barriers to success exist, including high levels of graft-versus-host disease, low numbers of healthy cells obtained from CB, and increased risk of bleeding due to delayed megakaryocyte, or platelet, engraftment.

Hoffman and Iancu-Rubin are pioneers of bone marrow cell therapy treatments, and development of this technology was enabled by the New York State Stem Cell Science program, NYSTEM. As a New York State Department of Health initiative, NYSTEM awarded a $1 million grant to Hoffman in 2010 that supported the original research underpinning this platform technology. In 2015, NYSTEM awarded Hoffman and Iancu-Rubin an $8 million grant to translate the technology from the laboratory into the clinic, where it is currently in clinical trial1.

Hoffman also serves as Director of the Myeloproliferative Disorders Research Program and Professor of Medicine (Hematology and Medical Oncology) and Iancu-Rubin is Associate Professor of Pathology at the Icahn School of Medicine and Director of the Cellular Therapy Laboratory at Mount Sinai Hospital.

"Promising discoveries by Mount Sinai scientific thought leaders may lead to new, essential cell-based therapies that will broadly benefit patients," said Erik Lium, Executive Vice President and Chief Commercial Innovation Officer, Mount Sinai Innovation Partners. "We're pleased to be collaborating with Tacitus to launch the next stage of development for these technologies."

"Tacitus is committed in its mission to advance next-generation cell therapies with curative potential," said Carter Cliff, CEO of Tacitus. "Based on our founders' solid foundation of research, we are translating these discoveries into broad clinical practice as we look to dramatically improve the standard of care for patients with life-threatening conditions."

About HSC100

HSC100 is an investigational therapy based on allogeneic hematopoietic stem cells (HSC) expanded from umbilical cord blood. HSC100 is being investigated currently in an open-label Phase I clinical trial1 in the United States for treatment of hematological malignancies. The success of unmanipulated cord blood as a source of stem cells has been hampered by the small number of stem cells present in a single cord, leading to delayed engraftment and frequent graft failure. Our proprietary technology includes the use of an epigenetic modifier, valproic acid, to expand the number and the quality of HSCs found in cord blood collections. For more information on HSC100 clinical trials, please visit http://www.clinicaltrials.gov.

1ClinicalTrials.gov identifier NCT03885947.

About Tacitus Therapeutics

Tacitus Therapeutics is a clinical-stage biotechnology company developing advanced medicines for treatment of blood cancers, immune disorders and other intractable disease conditions. Our mission is to pioneer best-in-class therapies using proprietary cell expansion, differentiation and engineering platform technologies that overcome the limitations of traditional cell transplantation. Initial targets include a lead clinical program (HSC100) investigating the treatment of blood cancers, followed by preclinical programs to address clotting disorders and other serious unmet medical needs. For additional information, please visit http://www.tacitustherapeutics.com.

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About Mount Sinai Health System

The Mount Sinai Health System is New York City's largest integrated delivery system, encompassing eight hospitals, a leading medical school, and a vast network of ambulatory practices throughout the greater New York region. Mount Sinai's vision is to produce the safest care, the highest quality, the highest satisfaction, the best access and the best value of any health system in the nation. The Health System includes approximately 7,480 primary and specialty care physicians; 11 joint-venture ambulatory surgery centers; more than 410 ambulatory practices throughout the five boroughs of New York City, Westchester, Long Island, and Florida; and 31 affiliated community health centers. The Icahn School of Medicine is one of three medical schools that have earned distinction by multiple indicators: ranked in the top 20 by U.S. News & World Report's "Best Medical Schools", aligned with a U.S. News & World Report's "Honor Roll" Hospital, No. 12 in the nation for National Institutes of Health funding, and among the top 10 most innovative research institutions as ranked by the journal Nature in its Nature Innovation Index. This reflects a special level of excellence in education, clinical practice, and research. The Mount Sinai Hospital is ranked No. 14 on U.S. News & World Report's "Honor Roll" of top U.S. hospitals; it is one of the nation's top 20 hospitals in Cardiology/Heart Surgery, Diabetes/Endocrinology, Gastroenterology/GI Surgery, Geriatrics, Gynecology, Nephrology, Neurology/Neurosurgery, and Orthopedics in the 2019-2020 "Best Hospitals" issue. Mount Sinai's Kravis Children's Hospital also is ranked nationally in five out of ten pediatric specialties by U.S. News & World Report. The New York Eye and Ear Infirmary of Mount Sinai is ranked 12th nationally for Ophthalmology, Mount Sinai St. Luke's and Mount Sinai West are ranked 23rd nationally for Nephrology and 25th for Diabetes/Endocrinology, and Mount Sinai South Nassau is ranked 35th nationally for Urology. Mount Sinai Beth Israel, Mount Sinai St. Luke's, Mount Sinai West, and Mount Sinai South Nassau are ranked regionally. For more information, visit http://www.mountsinai.org or find Mount Sinai on Facebook, Twitter and YouTube.

About Mount Sinai Innovation Partners (MSIP)

MSIP is responsible for driving the real-world application and commercialization of Mount Sinai discoveries and inventions and the development of research partnerships with industry. Our aim is to translate discoveries and inventions into health care products and services that benefit patients and society. MSIP is accountable for the full spectrum of commercialization activities required to bring Mount Sinai inventions to life. These activities include evaluating, patenting, marketing and licensing new technologies building research, collaborations and partnerships with commercial and nonprofit entities, material transfer and confidentiality, coaching innovators to advance commercially relevant translational discoveries, and actively fostering an ecosystem of entrepreneurship within the Mount Sinai research and health system communities. For more information, please visit http://www.ip.mountsinai.orgor find MSIP onLinkedIn, Twitter, Facebook,Medium, and YouTube.

Media Contacts:

Mount Sinai Cynthia Cleto Mount Sinai Innovation Partners (646) 605-7359 cynthia.cleto@mmsm.edu

Tacitus TherapeuticsJoleen RauRau Communications(608) 209-0792232130@email4pr.com

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Tacitus Therapeutics Launches in Collaboration with Mount Sinai to Develop Stem Cell Therapies for Life-Threatening Diseases - Yahoo Finance

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- Trial will replace currently used chemotherapy conditioning with apamistamab-I-131, Actinium's targeted conditioning ARC, to selectively eliminate lymphoma cancer cells and stem cells to enable engraftment of stem cell gene therapy

- Anti-HIV stem cell gene therapy intended to simultaneously treat patients' HIV-related lymphoma and develop immune cells resistant to HIV

NEW YORK, Jan. 13, 2020 /PRNewswire/ --Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) ("Actinium"), announced today that it has entered into an agreement with the University of California, Davis (UC Davis) to utilize Actinium's Antibody Radiation-Conjugate or ARC apamistamab-I-131 for targeted conditioning and replace the chemotherapy conditioning being used in an ongoing Phase 1/2 stem cell gene therapy clinical trial. In the trial, patients with relapsed or refractory HIV-related lymphoma are being treated with autologous stem cell gene therapy. This is the first gene therapy clinical trial that will utilize ARC based conditioning. The clinical trial will be conducted at UC Davis and may be expanded to additional sites in the future.

(PRNewsfoto/Actinium Pharmaceuticals, Inc.)

Dr. Mehrdad Abedi, Professor, Hematology and Oncology at UC Davis and study lead, said, "This collaboration represents an exciting combination of revolutionary technologies that could further our ability to treat patients with HIV and other life-threatening diseases with gene therapy. Despite the advances made in the field of gene therapy, the reliance on non-targeted chemotherapy and external radiation as conditioning regimens is less than optimal and poses a problem that we hope to reduce or eliminate as part of this collaboration by replacing our conditioning regimen in this study with Actinium's ARC based targeted conditioning. Advances in HIV therapies have dramatically improved patient survival, but current therapies require life-long daily use to keep the HIV virus at bay, can have severe side effects, may be overcome by HIV resistance and do not address the needs of all patients like those in this study with HIV-related lymphomas. We envision a future where a single treatment of our stem cell gene therapy can cure patients of their lymphoma and HIV leaving the patient with a new immune system that can fight, be resistant to and prevent the mutation of HIV. Apamistamab-I-131's demonstrated antitumor effect against lymphoma and ability to condition patients in a targeted manner with a demonstrated tolerable safety profile in the bone marrow transplant setting makes it an ideal conditioning agent for this patient population. Based on these factors and extensive supporting clinical data in the Iomab-B program, we selected this ARC as the conditioning agent for the next phase of our trial as we believe antibody radiation-conjugates are more advanced and hold distinct advantages over novel but unproven conditioning technologies such as Antibody Drug Conjugates and naked antibodies that are beginning to be developed albeit at the preclinical stage."

In the current clinical trial, the anti-HIV stem cell gene therapy is produced by taking a patient's own or autologous, blood forming stem cells and genetically modifying them via gene therapy with a combination of three anti-HIV genes. The intended result is for the gene modified bone marrow stem cells to produce a new immune system and newly arising immune cells that are resistant to HIV via a single treatment. Conditioning is necessary prior to adoptive cell therapies such as gene therapy to eliminate certain cell types such as immune cells and stem cells in the bone marrow so the transplanted cells can engraft. Until now, conditioning in this trial, as is typical, used a multi-drug chemotherapy regimen administered over several days. This approach is non-targeted, associated with toxicities that impairs patients and restricts the use and efficacy of cellular therapy. Apamistamab-I-131, which requires just one therapeutic administration, will displace the non-targeted chemotherapy to condition patients in a targeted manner with the goal of reducing conditioning related toxicities and improving patient outcomes. Actinium and UC David will cross-reference their respective Investigational New Drug applications and will work collaboratively to obtain necessary regulatory and institutional approvals. In this clinical collaboration, Actinium will provide drug product, support for its administration and certain trial costs. UC Davis will be responsible for the production of the anti-HIV stem cell gene therapy and overall conduct of the study and its cost.

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Dr. Dale Ludwig, Actinium's Chief Scientific Officer, said, "We are excited to be working with Dr. Abedi on this clinical study and we appreciate his recognition of the value of our Iomab-ACT targeted conditioning program may provide in support of gene stem cell therapy. This targeted approach using our CD45 ARC, enables both anti-tumor activity and effective conditioning with the potential for reduced toxicity compared to non-targeted chemotherapy and external radiation in the bone marrow transplant setting. Supported by extensive clinical investigation in 12 trials and over 300 patients, a single therapeutic dose of apamistamab-I-131 is sufficient for conditioning and, due to its dual activity, even a patient with active disease could expect to receive therapy within two weeks, which is anticipated to lead to better outcomes compared to chemotherapy, external beam radiation, or exploratory approaches such as naked antibodies or Antibody Drug Conjugates. In addition, CD45, the target of apamistamab-I-131, is ideal for targeted conditioning, as it is not expressed outside of the haemopoietic system and, because it is a poorly internalizing receptor. An ARC approach which does not require internalization of its radionuclide warhead for target cell killing, is anticipated to be more viable and more effective than Antibody Drug Conjugate approaches which need to internalize their payloads. Given the potential of this ARC targeted conditioning technology for bone marrow transplant, we are grateful to Dr. Abedi for the opportunity to advance the Iomab-ACT program into the promising field of gene stem cell therapy."

Sandesh Seth, Actinium's Chairman and Chief Executive Officer, said, "Actinium is thrilled to be working with UC Davis and honored to now be part of this important trial. It has become evident that better conditioning regimens are needed for cell and gene therapies to reach their full potential. Our team is proud to be the first company to establish a clinical stage targeted conditioning portfolio for both cell and gene therapy. We are pleased to extend our ARC technology for targeted conditioning into these rapidly advancing fields and we are committed to establishing a strong leadership position in enabling these adoptive cell therapies fully realize their great potential for improving patients' lives."

Apamistamab-I-131's demonstrated conditioning and antitumor effect in lymphoma1

Actinium's apamistamab-I-131 ARC has been studied as a targeted conditioning agent in over 300 patients in the bone marrow transplant setting in the Iomab-B Program and is currently being studied in a pivotal Phase 3 clinical (SIERRA) trial in patients with relapsed or refractory acute myeloid leukemia. Clinical proof of concept has been established with Iomab-B for targeted conditioning in high-risk, relapsed or refractory lymphoma patients prior to an autologous stem cell transplant where a favorable safety profile with no dose limiting toxicities and minimal non-hematologic toxicities observed and promising efficacy with median overall survival not reached (range: 29 months to infinity) and 31% of patients in prolonged remission at a median of 36 months follow up (range: 25 41 months)1.

1) Cassaday et al. Phase I Study of a CD45-Targeted AntibodyRadionuclide Conjugate for High-Risk Lymphoma. AACR Clin Cancer Res Published OnlineFirst September 3, 2019

About Actinium Pharmaceuticals, Inc.

Actinium Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing ARCs or Antibody Radiation-Conjugates, which combine the targeting ability of antibodies with the cell killing ability of radiation. Actinium's lead application for our ARCs is targeted conditioning, which is intended to selectively deplete a patient's disease or cancer cells and certain immune cells prior to a BMT or Bone Marrow Transplant, Gene Therapy or Adoptive Cell Therapy (ACT) such as CAR-T to enable engraftment of these transplanted cells with minimal toxicities. With our ARC approach, we seek to improve patient outcomes and access to these potentially curative treatments by eliminating or reducing the non-targeted chemotherapy that is used for conditioning in standard practice currently. Our lead product candidate, apamistamab-I-131 (Iomab-B) is being studied in the ongoing pivotal Phase 3 Study of Iomab-B in Elderly Relapsed or Refractory Acute Myeloid Leukemia (SIERRA) trial for BMT conditioning. The SIERRA trial is over fifty percent enrolled and promising single-agent, feasibility and safety data has been highlighted at ASH, TCT, ASCO and SOHO annual meetings. Apatmistamamb-I-131 will also be studied as a targeted conditioning agent in a Phase 1/2 anti-HIV stem cell gene therapy with UC Davis and is expected to be studied with a CAR-T therapy in 2020. In addition, we are developing a multi-disease, multi-target pipeline of clinical-stage ARCs targeting the antigens CD45 and CD33 for targeted conditioning and as a therapeutic either in combination with other therapeutic modalities or as a single agent for patients with a broad range of hematologic malignancies including acute myeloid leukemia, myelodysplastic syndrome and multiple myeloma. Ongoing combination trials include our CD33 alpha ARC, Actimab-A, in combination with the salvage chemotherapy CLAG-M and the Bcl-2 targeted therapy venetoclax. Underpinning our clinical programs is our proprietary AWE (Antibody Warhead Enabling) technology platform. This is where our intellectual property portfolio of over 100 patents, know-how, collective research and expertise in the field are being leveraged to construct and study novel ARCs and ARC combinations to bolster our pipeline for strategic purposes. Our AWE technology platform is currently being utilized in a collaborative research partnership with Astellas Pharma, Inc.

Forward-Looking Statements for Actinium Pharmaceuticals, Inc.

This press release may contain projections or other "forward-looking statements" within the meaning of the "safe-harbor" provisions of the private securities litigation reform act of 1995 regarding future events or the future financial performance of the Company which the Company undertakes no obligation to update. These statements are based on management's current expectations and are subject to risks and uncertainties that may cause actual results to differ materially from the anticipated or estimated future results, including the risks and uncertainties associated with preliminary study results varying from final results, estimates of potential markets for drugs under development, clinical trials, actions by the FDA and other governmental agencies, regulatory clearances, responses to regulatory matters, the market demand for and acceptance of Actinium's products and services, performance of clinical research organizations and other risks detailed from time to time in Actinium's filings with the Securities and Exchange Commission (the "SEC"), including without limitation its most recent annual report on form 10-K, subsequent quarterly reports on Forms 10-Q and Forms 8-K, each as amended and supplemented from time to time.

Contacts:

Investors:Hans VitzthumLifeSci Advisors, LLCHans@LifeSciAdvisors.com(617) 535-7743

Media:Alisa Steinberg, Director, IR & Corp Commsasteinberg@actiniumpharma.com(646) 237-4087

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SOURCE Actinium Pharmaceuticals, Inc.

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Actinium Pharmaceuticals Announces Iomab-ACT Program Gene Therapy Collaboration with UC Davis in Ongoing Clinical Trial for Patients with HIV-Related...

Bone Marrow Stem Cells Comments Off on Actinium Pharmaceuticals Announces Iomab-ACT Program Gene Therapy Collaboration with UC Davis in Ongoing Clinical Trial for Patients with HIV-Related… | January 14th, 2020

Home News Drugs in the Pipeline

The Food and Drug Administration (FDA) has granted Fast Track designation to bomedemstat (IMG-7289; Imago BioSciences) for the treatment of essential thrombocythemia, a myeloproliferative disorder characterized by high platelet counts.

[Essential thrombocythemia] is a quiet bone marrow cancer that can linger for years, said Hugh Young Rienhoff, Jr. MD, CEO, Imago Biosciences. In a subset of patients, the excess of platelets leads to bleeding and clotting including strokes and infractions, each having a significant impact on these patients.

Bomedemstat is believed to inhibit lysine-specific demethylase 1 (LSD1 or KDM1A), an enzyme that plays an important role in the production and function of megakaryocytes and in self-renewal of malignant hematopoietic stem cells. The investigational agent has been shown to be effective in preclinical studies across a range of myeloid malignancy models. The Company plans to initiate a phase 2 trial to assess bomedemstat in patients with essential thrombocythemia.

With only one FDA approved therapy, one that does not increase overall survival, patients are in desperate need of new options. Based on its mechanism and safety data obtained to date, we believe bomedemstat has the promise to be that new treatment, added Rienhoff.

For more information visit imagobio.com.

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Bomedemstat Receives Fast Track Status for Essential Thrombocythemia - Monthly Prescribing Reference

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Sickle cell disease is a painful condition that thousands of kids have to endure. The genetic disease impacts the blood, but it can cause organ damage and other issues, including lots of pain.

Most of the time there is no end in sight, which makes it even harder on families. But the bravery of one teen is helping scientists to develop a potential cure that could change the lives of so many.

Helen Obando recently became the youngest person to ever go through a special gene therapy using stem cells.

The usual treatment for sickle cell therapy is a bone marrow transplant form a healthy sibling, but Helen's older sister Haylee also has sickle cell, so that isn't really an option for the family.

The Obandos were excited to learn about an experimental treatment that has the potential to flip the switch on the genetics and actually cure the disease.

Scientists are hoping that the new treatment could help people with a number of genetic conditions using a technique to manipulate the DNA.

Helen had to spend four weeks in the hospital after her infusion to get strong enough to go home, and they don't know yet if the treatment has worked.

The poor girl has gone through a lot. Her pelvis was harmed before she even turned 1, and at 2, her spleen had to be removed. She's had a lot of painful episodes, and while Haylee was able to match with their younger brother Ryan for a bone marrow transplant, that wasn't an option for Helen.

In the Boston Globe, Helen's mom said that she was scared of the gene therapy option when she first heard of it. But she decided that it was worth the risk to have a chance at being healthy.

Six months since the treatment, it's so far, so good. Helen's hemoglobin levels are at a point that she has never achieved. She actually has no signs of sickle cell right now, and that is just amazing.

What a brave girl to go through a risky procedure.It's a big burden for a teenager to bear, but luckily things have worked out well so far. We hope that Helen continues to find success and health in the new year.

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Helen Obando Is The Youngest To Successfully Undergo Sickle Cell Therapy - Moms

Bone Marrow Stem Cells Comments Off on Helen Obando Is The Youngest To Successfully Undergo Sickle Cell Therapy – Moms | January 14th, 2020