Smart Stem Cells Made From Fat Have the Power to Heal – Freethink

New smart stem cells show a promising power to heal.

Researchers have reprogrammed human fat cells into adaptive smart stem cells that can lie dormant in the body until they are needed to heal various tissues. They demonstrated the cells' effectiveness at healing damaged tissue in a mouse study.

To create the smart stem cells, the team from UNSW Sydney exposed human fat cells to a compound mixture. After about three and a half weeks, the cells lost their original identity and began acting like stem cells, or iMS (induced multipotent stem cells).

"The stem cells acted like chameleons. They followed local cues to blend into the tissue that required healing."

"The stem cells we've developed can adapt to their surroundings and repair a range of damaged tissues," said UNSW hematologist John Pimanda, and co-author of the study, which they published in Science Advances.

"To my knowledge, no one has made an adaptive human multipotent stem cell before. This is uncharted territory."

Next, they injected the experimental iMS cells into healthy mice to see how the cells would respond. The cells remained dormant for some time, but they activated when the mouse was injured. Because of the cells' regenerative ability to act as "smart stem cells," they transformed themselves into whatever tissue was needed to heal the injured mouse --- like bone tissue, heart, or skin.

"The stem cells acted like chameleons," said Avani Yeola, lead author on the study at UNSW Medicine & Health. "They followed local cues to blend into the tissue that required healing."

All cells in a human body contain the same DNA. To differentiate between tissues, like a skin cell versus a bone cell, the cells only use a small portion of their total DNA. The rest of the DNA is shut down naturally by local modifications.

"The idea behind our approach was to reverse these modifications," said Pimanda. "We wanted the cells to have the option of using that part of the DNA if there was a signal from outside the cell."

Tissue-specific stem cells, like those that are restricted to becoming parts of the liver or lung, are limiting. But smart stem cells that can respond to their environment and become any tissue, like multipotent stem cells, will have many uses.

In the future, doctors could take a patient's fat cells, incubate them with the compound, and inject them into the patient to heal heart damage or trauma injuries.

But applications like this could be a long way off. The team needs to do much more research to prove this is safe in humans for different kinds of trauma before it becomes a real therapy.

We'd love to hear from you! If you have a comment about this article or if you have a tip for a future Freethink story, please email us at [emailprotected]

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Smart Stem Cells Made From Fat Have the Power to Heal - Freethink

Are Covid-19 vaccines safe for cancer patients? Here is what cancer experts say – CNN

It's a question that has been on the minds of researchers and oncologists long before the Pfizer and Moderna vaccine rollouts began. Although there is a consensus that the vaccines are safe for most cancer patients, according to the American Cancer Society and others in the medical community, research into whether they will be effective for cancer patients is still a data-free zone. The American Cancer Society recommends that cancer patients talk to their doctors before getting any type of vaccine because all patients and their courses of treatment are different.

There are several factors that may require a cancer patient to delay vaccination, including recent stem cell transplants or other recent use of therapy agents known to reduce vaccine efficacy, according to Dr. Laura Makaroff, the American Cancer Society's Senior Vice President for Prevention and Early Detection.

"As far as safety of the vaccine, every situation for every patient with cancer is a little different. And there's a spectrum of where any one patient might be in their cancer journey," Makaroff told CNN. "The Covid-19 vaccine is definitely safe for people with cancer but it's important that patients have a conversation with their healthcare provider and their cancer care team to determine when is the right time to have the vaccine."

"All the guidance that we're seeing -- the American Cancer society and other leading oncology groups -- is that Covid-19 immunization is recommended for patients in active therapy, but we really understand that there are limited safety and efficacy data on these patients," Makaroff said.

"Despite the lack of safety data specifically in patients with CLL (chronic lymphocytic leukemia), SARS-Cov-2 vaccination is anticipated to be safe."

CLL is characterized by a weakened immune system. The immunocompromization is so severe that CLL patients are advised to avoid live vaccines such as the ones for measles or yellow fever.

"The thing that people don't quite realize is that the impaired immune system in CLL patients -- due to the disease or some of the treatments for the disease -- can actually impact your response to vaccination," Ujjani told CNN.

"We recommend the Covid-19 vaccines for our patients ... but we're not really sure how effective it's going to be," she added. "Patients with blood cancers are typically excluded from the clinical trials evaluating the efficacy of the vaccine."

To remedy the lack of data, Ujjani is launching a research study involving 500 CLL patients across the country -- all of whom are already slated to receive the vaccine from their doctor or pharmacist -- to determine what kind of immune response they will have to the commercially available vaccines.

Advocacy groups for other more prevalent types of cancer are also encouraging cancer patients to get vaccinated as soon as possible. The Lung Cancer Action Network recently asked the US Advisory Committee on Immunization Practices to give lung cancer patients expedited access to the vaccines.

The scientific community agrees that more research is needed to determine the effectiveness of Covid-19 vaccines in cancer patients -- and many doctors are working around the clock to get more trials off the ground.

"We need these data so that we can better inform patients and better prioritize the allocation of the vaccines. If these vulnerable patients form an adequate immune response, we should certainly vaccinate them as early as possible," Dr. Elad Sharon, senior investigator at the National Cancer Institute, told CNN via email.

"But, if research efforts show that these patients fail to form a protective response to these vaccines, then what we will need to do will be to vaccinate everyone around these patients first, so that our medically vulnerable patients are most protected by the people living with them and caring for them."

Ujjani added, "We're all working really hard to answer this question, but it's hard because we've been working kind of against the clock as the vaccines just became available."

"A lot of our patients have suffered in isolation and fear, and they're not sure they're going to get back to a normal life," Ujjani told CNN. "So every oncologist is interested to see how their patients will respond to the vaccine."

Correction: An earlier version of this story misstated the academic journal that published a study conducted by the European Research Initiative. It was published in the journal Leukemia. It also misnamed the CLL Society by including the words of the acronym.

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Are Covid-19 vaccines safe for cancer patients? Here is what cancer experts say - CNN

The race to treat a rare, fatal syndrome may help others with common disorders like diabetes – Science Magazine

Misfolded proteins (orange) in the endoplasmic reticulum may play a role in Wolfram syndromes many symptoms.

By Mitch LeslieFeb. 11, 2021 , 2:00 PM

Maureen Marshall-Doss says the first sign that her vision was deteriorating came when she misidentified the color of a dress. At a backyard get-together about 20 years ago, the Indianapolis resident pointed out an attractive yellow dress another woman was wearing. You see that as yellow? Shes wearing a pink dress, Marshall-Doss recalls her husband responding.

Today, Marshall-Doss is virtually blind. With help from custom made eyeglasses that magnify objects 500 times, I can see shapes, she says. But she can no longer drive and had to quit the job she loved as a school librarian. Along with her dimming vision, she has type 1 diabetes and has lost her sense of taste and smell.

Marshall-Doss is one of 15,000 to 30,000 people around the world with Wolfram syndrome, a genetic disease. For decades, the condition remained enigmatic, untreatable, and fatal. But in the past few years, insights into its mechanism have begun to pay off, leading to the first clinical trials of drugs that might slow the illness and sparking hopes that gene therapy and the CRISPR DNA-editing tool might rectify the underlying genetic flaws. Here is a rare disease that the basic science is telling us how to treat, says physiologist Barbara Ehrlich of the Yale School of Medicine.

The research could also aid more than the relatively few patients with Wolfram syndrome. Driving the diseases many symptoms is a malfunction of the endoplasmic reticulum (ER), the multichambered organelle that serves as a finishing school for many cellular proteins. Known as ER stress, the same problem helps propel far more common illnesses, including type 2 diabetes, amyotrophic lateral sclerosis (ALS), Parkinsons disease, and Alzheimers disease. Wolfram syndrome is the prototype of an endoplasmic reticulum disorder, says medical geneticist Fumihiko Fumi Urano of Washington University School of Medicine in St. Louis. Because Wolfram syndrome is simpler, says Scott Oakes, a cell biologist and pathologist at the University of Chicago, researchers think it could illuminate the mechanisms of other ER-disrupting diseases, which affect hundreds of millions of people worldwide.

In the late 1930s,four children with diabetes were going blind, and doctors were stumped. Like many other people in the United States struggling through the Great Depression, the siblings ate a paltry diet, subsisting on potatoes, bread, oatmeal, and a little milk. But after examining three of the children, Donald Wolfram, a physician at the Mayo Clinic in Rochester, Minnesota, and an ophthalmologist colleague ruled out malnutrition as the cause of their puzzling condition. Lead poisoning and syphilisthough common enoughwerent to blame, either. When Wolfram and his partner wrote up the cases in 1938, they concluded that the symptoms could be manifestations of an hereditary or acquired cerebral lesion.

The physicians were right that the syndrome eventually named for Wolfram is hereditary. Recessive mutations in the gene for a protein called wolframin are responsible for most cases, with glitches in a second gene causing most of the rest. However, the pair was wrong to think the defect lies only in the brain. Instead, the symptoms stem from widespread cell death. Its definitely a disease that affects the whole body, Marshall-Doss says.

The first sign of the illness, appearing when patients are children, is usually diabetes mellitus, or faulty sugar metabolism, sparked by the demise of insulin-secreting beta cells in the pancreas. Most patients also develop the unrelated condition diabetes insipidus, in which the pituitary gland doesnt dole out enough of a hormone that helps control the bodys fluid balance, causing the kidneys to produce huge amounts of urine.

Mutations in the gene for wolframin disrupt the endoplasmic reticulum and lead to cell death throughout the body, causing a range of symptoms.

DeafnessPatients usually beginto lose their hearingin their teens. Balance andcoordination difficultyThe disease attacks the cerebellum, hampering the ability to control movement. Diabetes mellitusInsulin-producing beta cells die, hindering the bodys use of sugar as energy. Difficulty breathingBy damaging thebrain stem, the diseasecan disrupt respiration. Diabetes insipidusBecause of a faultypituitary gland, the kidneys produce too much urine. Loss of visionAs cells in theoptic nerve die, patientsgradually go blind.

V. Altounian/Science

Ellie White, 19, of Centennial, Colorado, who was diagnosed with Wolfram syndrome 12 years ago, says she hasnt had a full night of sleep since she was 3 years old. She gets up again and again to use the bathroom and monitor her blood sugar.

Yet she and other patients say that as disruptive as those problems are, they are not the diseases most dismaying consequence. The biggest symptom of Wolfram syndrome that affects me the most is my vision, White says. Because neurons in the optic nerve perish, patients usually go blind within 10 years of their first visual symptoms.

Other neurons die as well. As the disease progresses, brain cells expire, and walking, breathing, and swallowing become difficult. Most people with Wolfram syndrome die before age 40, often because they can no longer breathe. At 57, Marshall-Doss is one of the oldest patients; one of her mutated genes may yield a partly functional version of wolframin, triggering a milder form of the disease, Urano says.

Two advanceshave made it possible to begin to tackle those symptoms. The first was Uranos discovery nearly 20 years ago that linked Wolfram syndrome to ER stress. The ER is where about one-third of a cells newly made proteins fold into the correct shapes and undergo fine-tuning. Cells can develop ER stress whenever they are under duress, such as when they dont have enough oxygen or when misfolded proteins begin to pile up inside the organelle.

In test tube experiments, Urano and his colleagues were measuring the activity of genes to pinpoint which ones help alleviate ER stress. One gene that popped up encodes wolframin, which scientists had shown in 1998 was mutated in patients with Wolfram syndrome. Following up on that finding, Urano and his team determined that wolframin takes part in whats known as the unfolded protein response, which is a mechanism for coping with ER stress in which cells take steps including dialing back protein production.

Scientists think wolframin plays a key role in the unfolded protein response, though they havent nailed down exactly how. When wolframin is impaired, cells become vulnerable to ER stress. And if they cant relieve that stress, they often self-destruct, which could explain why so many neurons and beta cells die in the disease.

Defective wolframin may harm cells in other ways. The ER tends the cells supply of calcium, continually releasing and absorbing the ion to control the amount in the cytoplasm. Changes in calcium levels promote certain cellular activities, including the contraction of heart muscle cells and the release of neurotransmitters by neurons. And wolframin affects calcium regulation.

Beta cells genetically engineered to lack functional wolframin brim with calcium, Ehrlich and colleagues reported in July 2020 in theProceedings of the National Academy of Sciences. When exposed to lots of sugar, the altered cells release less insulin and are more likely to die than healthy beta cells, the team found. The cells share that vulnerability with beta cells from patients with Wolfram syndrome. We think that excess calcium is leading to excess cell death, Ehrlich says.

ER malfunctions could hamstring other organelles as well. The ER donates calcium to the mitochondria, the cells power plants, helping them generate energy. In 2018, a team led by molecular biologist Ccile Delettre and molecular and cellular biologist Benjamin Delprat, both of the French biomedical research agency INSERM, discovered that in cells from patients with Wolfram syndrome, mitochondria receive less calcium from the ER and produce less energy. Those underpowered mitochondria could spur the death of optic nerve cells, the researchers speculate.

Fumihiko Urano holds dantrolene, a muscle relaxant drug he helped test as a treatment for Wolfram syndrome.

The link between ER stress and Wolfram syndrome has been crucial for identifying potential treatments because otherwise we would have nothing to target, Urano says. But a second development was also key, he says: the advocacy and support of patient organizations, such as the Snow Foundation and the Ellie White Foundation, headed by its namesakes mother. The foundations have stepped up with money for lab research and clinical trials when other sources, including government agencies, didnt come through.

Scientists, patients, and their advocates say Urano also deserves much of the credit. Besides treating patients, he heads the international registry of cases and has taken the lead in organizing clinical trials, screening compounds for possible use as treatments, and devising potential therapies. Fumi is clearly the driving force, says Stephanie Snow Gebel, co-founder of the Snow Foundation, who about 10 years ago helped persuade him to forgo a plum job as department chair at a Japanese university and take over the Wolfram program at Washington University.

Patients could soonstart to reap the benefits. In 2016, Urano and colleagues started the worlds first clinical trial for the disease: a phase 1/2 study of dantrolene, an approved muscle relaxant. The molecule was a top performer when they screened 73 potential treatments for their ability to save cells with terminal ER stress. Dantrolene didnt improve vision in the 22 participants, including White, the scientists reported in an October 2020 preprint. But in some patients, beta cells appeared to be working better and releasing more insulin. The drug is safe, but Urano says it will need to be chemically tweaked to target its effects before future trials are warranted.

Researchers are pursuing other possible treatments targeting ER stress or calcium levels. In 2018, U.K. scientists launched a trial that will include 70 patients to evaluate sodium valproate, a therapy for bipolar disorder and epilepsy that, in the lab, prevents cells with faulty wolframin from dying. Last year, another compound that emerged from Uranos screens, the diabetes drug liraglutide, entered a clinical trial. Also last year, an experimental drug developed by Amylyx Pharmaceuticals for Alzheimers disease and ALS received orphan drug designation from the U.S. Food and Drug Administration for Wolfram syndrome because it curbs ER stress. That designation offers tax breaks and other incentives, and it will get trials started sooner, Urano says.

Ehrlich and her team have a candidate of their own that they have begun to test in rodents: the drug ibudilast, which is approved in Japan to treat asthma. The researchers found it reduces calcium levels in beta cells lacking wolframin and boosts their survival and insulin output. New screening projects may reveal still more candidates.

But Urano knows that even if a treatment receives approval, it would be only a Band-Aid for Wolfram syndrome. Hoping to develop a genetic cure, he and colleagues are introducing replacement genes into cells from patients and from mice engineered to replicate the disease. The researchers are endowing the cells with healthy copies of the gene for wolframin or the gene for a protein that reduces ER stress to determine whether they restore cellular function and reduce cell death. At INSERM, Delettre and colleagues are also evaluating whether directing a working gene into optic nerve cells can curtail vision loss in mice with faulty wolframin. The scientists are still gathering data, but early results suggest the treatment can halt the deterioration.

Urano and his collaborators have also turned to the genome editor CRISPR, deploying it to correct the gene defect in patients stem cells and then growing them into beta cells. When the researchers transplanted the revamped cells into mice with diabetes, the animals blood sugar returned to healthy levels, the team reported in April 2020 inScience Translational Medicine.

Stem cell biologist Catherine Verfaillie of KU Leuven is collaborating on the CRISPR research. But she notes that because the faulty wolframin gene affects so many tissues, researchers will have to figure out how to deliver the CRISPR components to most cells in large organs such as the brain and livera prospect she calls pretty daunting. Urano agrees, predicting that CRISPR-based Wolfram therapies might take 10 to 20 years to develop. The alternative approach, gene therapy, could reach clinical trials more quickly, in 3 to 10 years, he says, because researchers have more experience with gene therapy and have created several treatments that have already been approved for other illnesses.

Because it stems from a single genetic glitch, Wolfram syndrome could also help scientists tease out the role of the ER in more complex diseases, including neurological conditions, type 2 diabetes, and cancer. The ER also falters in those diseases, causing cells to die, but the mechanism is harder to discern because they stem from myriad genetic and environmental factors. In Alzheimers disease, for instance, neurons develop ER stress as misfolded proteins accumulate inside and outside the cells.

Besides deepening researchers understanding of other conditions, the research on Wolfram syndrome might even deliver candidate treatments. Everyone would be very excited if we can make advances in targeting ER stress in Wolfram syndrome, Oakes says. It would open up the whole field to doing this in other degenerative diseases.

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The race to treat a rare, fatal syndrome may help others with common disorders like diabetes - Science Magazine

Arlo’s Army needs stem cell donor as mum begs for help to save three-year-old’s life – Glasgow Live

Gorgeous little Arlo McArthur looks the picture of health and happiness.

Loved and adored by his family this little lively three-year-old from Milngavie is spoiled rotten by his three big sisters and his ultimate day out is playing golf with his daddy.

But behind the cheeky grin lies a devastating truth - he's a "ticking-timebomb" and needs a stem cell transplant to save his life.

So today, we've joined with Arlo's mum Nicole, dad Ian and his three doting sisters Carys, Brooke and Holly in asking Glasgow Live readers to step up and help this brave little boy.

They need young men, between 16 and 30 to volunteer to be tested to see if they are a match for the toddler. There's not much to it, a simple swab test carried out at home is enough for the experts to determine if you're a match.

The more people who register to be tested the better chance there is of finding the ideal candidate willing to donate the bone marrow little Arlo desperately needs.

For this family your help could mean the difference between life and death.

They've lived with the knowledge since he was 10 weeks old that a rare genetic condition could rob their precious little boy of his future.

Diagnosed with Wiskott-Aldrich Syndrome, it means Arlo's immune system doesn't function properly and it's difficult for his bone marrow to produce platelets, making him prone to bleeding.

Its estimated there are between 1 and 10 cases per million males worldwide. Arlo was only the third case at Queen Elizabeth University Hospital.

Doctors say they cant take the risk with an older donor as he was lucky to survive a previous transplant which failed when he was a baby.

His back-up is his dad Ian, 31, but he's only a half-match.

Sadly little Arlo's story isn't unique, across the country 2,300 people a year need a stem cell transplant and charity Anthony Nolan coordinates the search and raises money to support their vital work.

Nicole, 37, dreams of seeing her little boy attend his first day at school next August and believes someone out there can help that dream come true.

She pleaded: "Were asking as many people as possible to register and help give Arlo the life he deserves.

"We want to love and enjoy having our little boy around for a long time. He should be able to live out his life of dreams.

"Put yourself in the shoes of a parent whose child is ill, or someone else who is about to lose a loved one. Youve just been told in a room that they wont make it without stem cells. How does it feel?

"Its not just our Arlo, there are plenty of Arlos out there who need your help."

"People don't realise how easy it is to do. It's not this big operation, just a few injections and a day at an out-patient clinic to save someone's life. I wish it was opt-out, like organ donation.

"We dont have much time but I know in my heart the right match is out there."

To find out how you can can join the register and help the fight to save little Arlo and others just like visit Anthony Nolan's website here.

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Arlo's Army needs stem cell donor as mum begs for help to save three-year-old's life - Glasgow Live

Arlo’s Army: mum opens up on heartbreaking decision to make 3-year-old sick to save his life – Glasgow Live

Little Arlo McArthur has spent his short life fighting.

No one knows better than his mum Nicole just how tough this little warrior is.

Diagnosed with a deadly blood disorder at just 10 weeks old, the three-year-old desperately needs a stem cell transplant to save his life.

And as the campaign to encourage males between 16 and 30 to volunteer to be tested to see if they are a match gathers pace, Nicole has opened up in heartbreaking detail about the harrowing battle to save Arlo.

She told Glasgow Live: "This is every parents worst nightmare. I cannot lose my son. We have such a fight ahead of us.

"He's doing really well at the moment but it's really scary looking at him, knowing he is so vulnerable. It hurts us so much."

It was only two weeks after bringing their baby boy home in 2017 that Nicole and husband Ian discovered something was wrong with him.

"We rushed him to A&E after he vomited a blood clot." Nicole recalls. "Doctors initially diagnosed a severe cows milk allergy and sent us home. Later he vomited again, and we found blood in his poo so we returned.

"It was suggested that it may be leukaemia as his platelets were so low. When they said that, I knew there was something deeply wrong. After that was ruled out, we waited ten more weeks for answers.

This was the point that I realised that doctors were only human, they are not God. They dont know everything: Its about eliminating all of these possibilities until they get to what it is. It was tough.

The Milngavie family spent weeks agonising in the dark and were offered leaflets on living without a diagnosis but kept in touch with medics.

One doctor mentioned that she thought his symptoms matched up with a rare condition, but did not want to define it to Nicole until the test results were confirmed.

After asking another member of staff, the sickened mum discovered they were testing her baby for Wiskott Aldrich Syndrome.

She said: "I Googled it when I got home and I was utterly traumatised to learn that the life expectancy for a child with this condition was five. I was beside myself.

"My friends and family were saying dont be silly and stay positive. I was praying and hoping it wasnt, but I knew in my gut it was what Arlo had."

Nicola steeled herself for the worst, but nothing could have prepared her for the moment she was given the bad news at the hospital. She broke down before the doctor had finished delivering the diagnosis.

Worse still, she was alone as her husband was unable to take any more time off work.

I was complete mess I was in full crazy mode, crying. I had to deliver the news to my husband over the phone. I never imagined it would be like this," she said.

The doctor tried to calm me down and said we could get him a bone marrow transplant. I didnt even know what that was before then.

Wiskott Aldrich Syndrome is a rare genetic immunodeficiency disease that keeps a child's immune system from functioning properly. It also makes it difficult for a child's bone marrow to produce platelets, making them prone to bleeding.

Its estimated there are between 1 and 10 cases per million males worldwide. Arlo was only the third case at Queen Elizabeth University Hospital.

A 10/10 donor match was found for him on December 22 the best Christmas present ever and shipped over from the US. The family battled to have it transported to Scotland from Heathrow in an Uber after flights were grounded during a snow storm.

The baby's transplant was successful, and the cells were engrafting. But that started to change, and his engraftment started to drop with each check in.

He also contracted severe sepsis and began suffering from seizures and it emerged entophilitis, a form of meningitis, had entered his brain. His neck lolled and his eyes danced around. He was taken into intensive care after being discharged.

We thought he might have had brain damage. It sounds terrible but we were accepting of that. Well take anything as long as hes with us. Well deal with what we have to. We just wanted him to live. Thankfully the symptoms resolved themselves.

If he didnt have that within six months, he wouldnt have made it to his first birthday. He was very, very sick.

Arlo urgently needs to find a young, male, donor aged 16-30. His back-up is his dad, who is a half-match.

Nicole explains that the success rate is higher for children under five in a healthier condition. When they find the match, the toddler will have to undergo high-dose chemotherapy to break down his immune system to allow the transplant to take place. He last had this when he was six months old.

One nurse described it to me as taking the body as close to death as they possibly can and rebuilding it. Its really harsh on the body its basically a poison," she said.

He really wasnt well the first time and he ended up on morphine for the pain. He had mucositis all the way from his throat to his gut and had to be fed through a tube to bypass his stomach. It was horrible.

"But if he doesnt do this as soon as he can, his immune system wont be able to withstand it. He could be at risk of leukaemia. I know hes a ticking time-bomb.

While doctors are hopeful that the next transplant will help Arlo, they warned the family to brace themselves for it not working out again. The mortality rate for a transplant is also around 20 per cent but Nicole said its a risk the family is willing to take to give their boy his best chance.

She said: The doctors do speak to you and say You do know if we dont get the result youre looking for things can go wrong. You have to sign paperwork to confirm that you know this could result in death.

So much can happen. You could undergo a transplant to fix one thing, and come out with another.

Once I played devils advocate and asked the doctor what Arlos life would be like if we chose not to take the transplant.

"He told me that Arlo would have a poor quality of life and they could possibly get him to his teenage years, maybe even his late teens. But it 'wouldnt be a nice way to live.' I said thats not an alternative. "

She added: To make him sick and risk his life to make him better all at the same time is heartbreaking. But theres that chance he could live a better life and thats what we are fighting for.

If we lose Arlo in the process, we know that we have done everything in our power to give him a normal life that we possibly can. That's in God's hands. It is our only option.

To find out how you can can join the register and help the fight to save little Arlo and others just like visit Anthony Nolan's website here.

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Arlo's Army: mum opens up on heartbreaking decision to make 3-year-old sick to save his life - Glasgow Live

Bone marrow transplant shows signs of curing brave little boy with one in a million condition – Shields Gazette

One-year-old Max Gardner was diagnosed with aplastic anaemia, in October 2020, a serious condition in which the bone marrow and stem cells do not produce enough blood cells.

After Max developed significant bruises and a rash over his body, parents, Connor Gardner and Rachel Nicholson, from Hebburn, were referred to South Tyneside District Hospital, where their brave little boy underwent tests.

Doctors initially believed that Max had an immune disorder but after he was admitted to the Royal Victoria Infirmary (RVI) further tests helped to diagnose him with aplastic anaemia.

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The family was told that the condition could be fatal if not treated properly.

Doctors said Max needed to have a bone marrow transplant, which has the potential to cure him.

Dad Connor, 29, and mum Rachel, 27, were both tested to see if they would be a bone marrow match and the pair were overjoyed when Rachel was found to be a 9/10 match.

Max started chemotherapy on January 7 at the RVI and mum Rachel donated stem cells on January 13 at Newcastles Freeman Hospital.

The following day, January 14, Max underwent the transplant at the RVI.

The family is now waiting for the results of a Chimerism Test which will tell them for definite whether the stem cells have worked but signs are already looking positive.

Delighted dad, Connor, said: "His neutrophils [a type of white blood cell that protect us from infections] have been more than 0.50 for three days in a row, which means that he is essentially engrafted, which means that his body is accepting the transplant.

"So it is working, but we still have to wait for the test results."

Doctors say there is no doubt that it has worked with the way the numbers have gone up but they have to officially do it like that to make sure, Connor continued.

"But there is no reason why it shouldnt have [doctors] say.

"He has done really well to get to this stage, he has absolutely sailed through it, everyone is surprised with how well he has done.

This the best outcome we could have hoped for.

But it hasnt been plain sailing for the family, who have also had to face additional challenges during the treatment.

Parents Connor and Rachael initially were not allowed to visit Max at the same time due to Covid rules, however the hospital has now eased the restriction in their case.

The family also became sick with Norovirus in the run-up to the transplant, causing concern over whether it would have to be pushed back.

Thankfully, the transplant went ahead as planned and the family made a good recovery, although Max still needs help with his eating.

Max will now have to remain in hospital for a while longer as he recovers from the transplant.

Connor added: We can feel that we are nearly at the end of it.

"His neutrophils are the highest they have ever been since he became poorly so we feel like we are coming to the end.

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Bone marrow transplant shows signs of curing brave little boy with one in a million condition - Shields Gazette

Canada’s blood supply has a diversity problem and people are dying because of it – CBC.ca

Lauren Sano with her father Mark, seen here last year when he was in hospital being treated for a rare form of leukemia. Mark Sano died in October of 2020. He was 52.

Lauren Sanostill wonders,if things were different, whether her father's life couldhave been saved.

"You always wonder if there was someone in the registry who was a bettermatch would have resulted in better outcomes and less transplant complications."

Mark Sanowas a 52-year-old Toronto father of three. He worked in the financial industry as a marketing manager and in his spare time was an avid sportsman who loved tennis, hockey and especially skiing.

In November of 2019 he was diagnosed with a rare form of leukemia. The only thing that couldsave his life, doctors told his family,was a stem cell transplant;a critical treatment for blood cancers and dozens of other diseases.

According to Canadian Blood Services (CBS), stem cells are the body's basic building blocks the raw material from which all cells are made. In blood, stem cells can become red, white blood cells or even blood platelets.

"Without stem cells, the body cannot make the blood cells needed for the immune system to function," CBS says, which runs the national blood bank.

It says a patient must find a match with a donor, and that is usually a person who shares the same ethnic background.

CBS says right now, donors to Canada's blood stem cell registry are more than two-thirds Caucasian, with the other third fracturedin uneven splintersacross race and ethnicity.

It means an Asian patient like Sano, according to the Canadian Blood Services stem cell registry, would have anywhere from seven to less than one per cent chanceof finding a match, depending on hisparticular genetic background.

So when the Sano family sought a match, they found a lack of minority donors who were aclose enough. Sano's daughter Lauren was the closest they could find and even then, she was far from ideal.

"I ended up being a half-match for him and was his donor.It was the most fulfilling and grounding experience."

As fulfilling as it was, it wasn't enough.Sano died at Princess Margaret Hospital in October 2020, 18 months after he was first diagnosed.

Lauren still wonders, whether her dad's life could have been saved, had they found the right donor from a more racially diverse pool of donors.

"I feel very lucky I was able to give him the gift of life.I was at least grateful that I was able to do this for my dad."

The dearth of diversity in Canada's stem cell registry is a problem Canadian Blood Services is familiar with, according toHeidi Elmoazzen, the agency's director of stem cells. Shehas been actively working on increasing the pool of minority donors to give minority patientsa better shot at getting better.

"We find that people tend to find matches within the same ethnic or racial background as them, which is why we're trying to build a registry that reflects the unique diversity we have here in Canada."

Some groups are more diverse than others when it comes to the make up of their stem cells, according to Elmoazzen. For example, she saysBlack people tend to be the most diverse.

A Black person whose ancestors are from the Caribbean might not have the same markers as someone from say, eastern Africa, which makes finding a match challenging.

Adding to the complication is that to harvest stem cells, you literally need young blood.Only young people, between the ages of 17 and 35 can apply.

The ongoing COVID-19 pandemic has also disrupted recruitment efforts, Elmoazzen says.Canadian Blood Services finds 60 to 70 per cent of its potential stem cell donors during its community clinics and with everyone staying home, the number of people visiting is down.

"It's had a heavy impact on our ability to recruit donors this year," she adds.

Still, virtual drives are underway. People interested in donating can still sign up through the Canadian Blood Services website.

There are also volunteers like Lauren Sano, who along with a number of Western University students will be pushing for donations in a virtual blood stem cell drive this month in honour of Black History Month and in April.

The hope is that by reaching out to diverse communities, Sanosays her goal is to help people make donating blood a habit. She says she hopes that willnot only will boost the blood supply, but the supply of blood products, such as stem cells and platelets as well.

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Canada's blood supply has a diversity problem and people are dying because of it - CBC.ca

Dean Tracy Johnson seeks to diversify the pipeline of future scientists and doctors – UCLA Newsroom

When Tracy Johnson was an undergraduate working in a lab at UC San Diego, she found herself suddenly jolted. Conducting research on gene function using fruit flies, she realized she was involved in something deeper and more fulfilling than a traditional classroom experience.

The idea that I was learning things that nobody else knew, that I could make some contribution, says the dean of the division of life sciences in the UCLA College, that was a game-changer.

Johnson, who holds the Keith and Cecilia Terasaki Presidential Endowed Chair in Life Sciences, joined the faculty of UCLAs Department of Molecular, Cell andDevelopmental Biology in 2014. Soon after, she was awarded a $1 million Howard Hughes Medical Institute grant to improve undergraduate science education, which was, in part, used to create the UCLA-HHMI Pathways to Success program.

Pathways gives students from diverse backgrounds an authentic research experience, early on, and in a prolonged way. For years, Johnson said, students of color and those who were the first in their family to attend college pursued science, technology, engineering and math degrees at equal rates as other students but left STEM majors at a higher rate.

It was clear that these statistics had less to do with preparation, she said, and more to do with students not seeing themselves as part of a scientific community. Pathways was designed to rethink that.

The goal was to help students understand they belonged and had important contributions to make.

The Pathways program has honestly opened up the world of research to me. I come from a normal public school in a mainly minority area, so I never knew what research truly entailed, said Venus Hagan, a second-year UCLA student majoring in molecular, cell and developmental biology and minoring in biomedical research.

Hagan noted how getting to do research as an undergraduate helped her discover her passion for it. Without the program, she said, I may have never considered minoring in biomedical research and possibly applying to MD/Ph.D. programs in the future.

In building the program, Johnson looked around the country to find what worked best, and bring it to UCLA. She was interested not just in lab work, but in mentoring as well.

Pathways students participate in a lab course dedicated to Johnsons field, gene expression. The DNA in every cell of a given plant or animal is identical. Expression is the process by which genes, or specific segments of DNA, get turned on. This process allows cells to perform specific functions. For example, this process can tell a cell to become part of a muscle or part of the brain, and so on.

Its a lot for first-year students to dive into, Johnson acknowledged. Theyre freshmen, on campus for barely 10 weeks when they start. Some students have never taken AP biology. It is ambitious, but they rise to the occasion.

Second-year student Nyari Muchaka said enrolling in Pathways was one of the best decisions she has ever made.

The program has provided me with multiple opportunities for summer and during the year research opportunities, and allowed me to find a group of friends I resonate with, said Muchaka, who is majoring in molecular, cell and developmental biology and minoring in biomedical research. Everyone is truly there to help each other which makes it one of the most fulfilling, enriching parts of my college experience. The program helps advance your interest in the biological field but also carry you through some of the best and roughest four years of your life. Pathways is truly an innovative program and provides a foundation for college studies you won't find anywhere else.

Johnson and her co-instructor and research collaborator, Azad Hossain, are preparing to publish some of the student research in an academic journal within the next year. Pathways has enrolled more than 125 students, and these students have taken on more and more responsibility as independent researchers, mentors, tutors and campus leaders as the years have passed. Many have gone on to doctoral programs, medical school, M.D./Ph.D. programs, and a host of other STEM-related careers.

There isnt anything quite like what we do, Johnson said. I think its a model for how to think about student success.

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Dean Tracy Johnson seeks to diversify the pipeline of future scientists and doctors - UCLA Newsroom

State-of-the-Art Treatments Garner University Foot and Ankle Institute Multiple Top Doctor Awards – Yahoo Finance

TipRanks

Lets talk portfolio defense. After last weeks social flash mob market manipulation, thats a topic that should not be ignored. Now, this is not to say that the markets are collapsing. After 2% losses to close out last weeks Friday session, this weeks trading kicked off with a positive tone, as the S&P 500 rose 1.5% and the Nasdaq climbed 2.5%. The underlying bullish factors a more stable political scene, steadily progressing COVID vaccination programs are still in play, even if they are not quite as strong as investors had hoped. While increased volatility could stay with us for a while, its time to consider defensive stocks. And that will bring us to dividends. By providing a steady income stream, no matter what the market conditions, a reliable dividend stock provides a pad for your investment portfolio when the share stop appreciating. With this in mind, weve used the TipRanks database to pull up three dividend stocks yielding 8%. Thats not all they offer, however. Each of these stocks has scored enough praise from the Street to earn a Strong Buy consensus rating. New Residential Investment (NRZ) Well start by looking into the REIT sector, real estate investment trusts. These companies have long been known for dividends that are both high-yield and reliable as a result of company compliance with tax rules, that require REITs to return a certain percentage of profits directly to shareholders. NRZ, a mid-size company with a market cap of $3.9 billion, holds a diverse portfolio of residential mortgages, original loans, and mortgage loan servicing rights. The company is based in New York City. NRZ holds a $20 billion investment portfolio, which has yielded $3.4 billion in dividends since the companys inception. The portfolio has proven resilient in the face of the corona crisis, and after a difficult first quarter last year, NRZ saw rising gains in Q2 and Q3. The third quarter, the last reported, showed GAAP income of $77 million, or 19 cents per share. While down year-over-year, this EPS was a strong turnaround from the 21-cent loss reported in the prior quarter. The rising income has put NRZ in a position to increase the dividend. The Q3 payment was 15 cents per common share; the Q4 dividend was bumped up to 20 cents per common share. At this rate, the dividend annualizes to 80 cents and yields an impressive 8.5%. In another move to return profits to investors, the company announced in November that it had approved $100 million in stock repurchases. BTIG analyst Eric Hagen is impressed with New Residential especially by the companys sound balance sheet and liquidity. [We] like the opportunity to potentially build some capital through retained earnings while maintaining a competitive payout. We think the dividend increase highlights the strengthening liquidity position the company sees itself having right now we expect NRZ has been able to release capital as it's sourced roughly $1 billion of securitized debt for its MSR portfolio through two separate deals since September, Hagen opined. In line with his comments, Hagen rates NRZ a Buy, and his $11 price target implies an upside of 17% for the year ahead. (To watch Hagens track record, click here) Its not often that the analysts all agree on a stock, so when it does happen, take note. NRZs Strong Buy consensus rating is based on a unanimous 7 Buys. The stocks $11.25 average price target suggests ~20% upside from the current share price of $9.44. (See NRZ stock analysis on TipRanks) Saratoga Investment Corporation (SAR) With the next stock, we move to the investment management sector. Saratoga specializes in mid-market debt, appreciation, and equity investments, and holds over $546 million in assets under management. Saratogas portfolio is wide ranging, and includes industrials, software, waste disposal, and home security, among others. Saratoga saw a slow but steady rebound from the corona crisis. The companys revenues fell in 1Q20, and have been slowly increasing since. The fiscal Q3 report, released early in January, showed $14.3 million at the top line. In pre-tax adjusted terms, Saratogas net investment income of 50 cents per share beat the 47-cent forecast by 6%. They say that slow and steady wins the race, and Saratoga has shown investors a generally steady hand over the past year. The stock has rebounded 163% from its post-corona crash low last March. And the dividend, which the company cut back in CYQ2, has been raised twice since then. The current dividend, at 42 cents per common share, was declared last month for payment on February 10. The annualized payment of $1.68 gives a yield of 8.1%. Analyst Mickey Schleien, of Ladenburg Thalmann, takes a bullish view of Saratoga, writing, We believe SAR's portfolio is relatively defensive with a focus on software, IT services, education services, and the CLO... SAR's CLO continues to be current and performing, and the company is seeking to refinance/upsize it which we believe could provide upside to our forecast." The analyst continued, "Our model anticipates SAR employing cash and SBA debentures to fund net portfolio growth. We believe the Board will continue to increase the dividend considering the portfolio's performance, the existence of undistributed taxable income, and the economic benefit of the Covid-19 vaccination program. To this end, Schleien rates SAR a Buy along with a $25 price target. This figure implies a 20% upside from current levels. (To watch Schleiens track record, click here) Wall Streets analysts agree with Schleien on this stock the 3 other reviews on record are Buys, and the analyst consensus rating is a Strong Buy. Saratogas shares are trading for $20.87, and carry an average price target of $25.50, suggesting an upside of 22% for the next 12 months. (See SAR stock analysis on TipRanks) Hercules Capital (HTGC) Last but not least is Hercules Capital, a venture capital company. Hercules offers financing support to small, early-stage client companies with scientific bent; Hercules clients are in life sciences, technology, and financial SaaS. Since getting started in 2003, Hercules has invested over $11 billion in more than 500 companies. The quality of Hercules portfolio is clear from the companys recent performance. The stock has bounced back fully from the corona crisis of last winter, rebounding 140% from its low point reached last April. Earnings have also recovered; for the first nine months of 2020, HTGC posted net investment income of $115 million, or 11% higher than the same period of 2019. For dividend investors, the key point here is that the net investment income covered the distribution in fact, it totaled 106% of the base distribution payout. The company was confident enough to boost the distribution with a 2-cent supplemental payment. The combined payout gives a $1.28 annualized payment per common share, and a yield of 8.7%. In another sign of confidence, Hercules completed a $100 million investment grade bond offering in November, raising capital for debt pay-downs, new investments, and corporate purposes. The bonds were offered in two tranches, each of $50 million, and the notes are due in March of 2026. Covering the stock for Piper Sandler, analyst Crispin Love sees plenty to love in HTGC. We continue to believe that HTGC's focus on fast growing technology and life sciences companies sets the company up well in the current environment. In addition, Hercules is not dependent on a COVID recovery as it does not have investments in "at-risk" sectors. Hercules also has a strong liquidity position, which should allow the company to act quickly when it finds attractive investment opportunities, Love commented. All of the above convinced Love to rate HTGC an Outperform (i.e. Buy). In addition to the call, he set a $16 price target, suggesting 9% upside potential. (To watch Loves track record, click here) Recent share appreciation has pushed Hercules stock right up to the average price target of $15.21, leaving just ~4% upside from the trading price of $14.67. Wall Street doesnt seem to mind, however, as the analyst consensus rating is a unanimous Strong Buy, based on 6 recent Buy-side reviews. (See HTGC stock analysis on TipRanks) To find good ideas for dividend stocks trading at attractive valuations, visit TipRanks Best Stocks to Buy, a newly launched tool that unites all of TipRanks equity insights. Disclaimer: The opinions expressed in this article are solely those of the featured analysts. The content is intended to be used for informational purposes only. It is very important to do your own analysis before making any investment.

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State-of-the-Art Treatments Garner University Foot and Ankle Institute Multiple Top Doctor Awards - Yahoo Finance

Transforming optimism: finding new ways to treat rare cancers – Cancer Research UK – Science Blog

Cancer is an extremely complex disease. There are over 200 different types, some of which are considered common and others which are classified as rare cancers. But what exactly does it mean if a cancer is rare?

Usually, it means it only affects a small handful of people, but doctors might also call a cancer rare if it starts in an uncommon place in the body, or if the cancer is an unusual type and requires special treatment.

For secondary central nervous system (CNS) lymphoma, its an incredibly rare cancer for a combination of these reasons.

Secondary CNS lymphoma is a type of lymphoma thats spread to the brain and spinal cord nervous system after originating elsewhere in the body. And as well as being a rare cancer, secondary CNS lymphoma is an aggressive cancer, which has relatively low survival rates.

However, the latest results from the Stand Up To Cancer-funded MARIETTA clinical trial, which details a new potentially transforming treatment, has shed a glimmer of hope for patients and doctors alike.

We spoke to Dr Kate Cwynarski, who led the study in the UK, about what the latest results could mean for patients with secondary CNS lymphoma.

With a rare cancer such as secondary CNS lymphoma, finding a large enough group of patients can be a real challenge. And in cases like this, researchers have to think on a global scale.

Its a rare disease. So the reality of it is that you would not get this information if we just performed a trial in the UK, says Cwynarski. International collaboration is the only way to do it.

The MARIETTA trial is the largest study focused on patients with secondary CNS lymphoma, involving 24 centres across 4 countries and recruiting a total of 79 patients. It involved the International Extranodal Lymphoma Group (IELSG) lead by Professor Andres Ferreri in Italy and it built on the success of prior research with this group. In the UK, the trial was managed by CRUK Southampton CTU.

In particular, findings from a previous clinical trial partly funded by us, which tested treatments for primary CNS lymphoma, a lymphoma thats only found in the brain, helped inform the design of this clinical trial.

The IELSG-32 trial tested the benefits of an intensive chemotherapy regimen known as MATRIX, followed by either whole brain radiotherapy or a stem cell transplant using the patients own cells.

Cwynarski describes the IELSG-32 trial as practice changing, and its from these impressive results that the MARIETTA trial was developed. So we adapted a strategy that was successful in treating primary CNS lymphoma in the IELSG-32 trial and added another chemotherapy regimen, called R-ICE, to help treat the systemic disease on top of the secondary brain disease.

Cwynarski specialises in lymphoma, so she has treated SCNSL patients both on and off the trial. One of the big benefits of this trial, she describes, is that the inclusion criteria for the cohort more accurately reflected the patients she sees in her clinic and referral practice.

This trial included patients up to 70 years of age. And it wasnt just focused on fit, young people. So I have to say I think it was meaningful, because it included the kind of patients that we actually see.

The trial also included people regardless of when their secondary CNS lymphoma was diagnosed, whether that was when someone was originally diagnosed with lymphoma, during treatment, or after their cancer had come back.

And the results look promising. A total of 49 patients (65%) responded to the treatment in some way, with 37 people going on to have a stem cell transplant. 100% of the patients who had the stem cell transplant had not seen their cancer recur a year after registering onto the trial. We are optimistic many will be cured of this aggressive lymphoma.

But the trial also picked up differences between groups. While the regime was effective to an extent in every sub-group, the most significant results were seen in patients whose CNS disease was discovered at initial lymphoma diagnosis. Within this group, 71% of patients had lived for 2 years without their cancer growing.

A result which has never been seen before.

The results of the trial have completely transformed the teams optimism when meeting new patients. We really have identified a regimen which is intensive, but its potentially curative, concludes Cwynarski, and the word cure is not something weve really used before when talking about this disease.

Recently, Cwynarski has been busy filling out a cohort of her patients DVLA forms, confirming they are fit to drive again after being 2 years treatment free. So thats an amazing success and it was very symbolic as a reminder that these people have been alive and off all treatment for 2 years.

Moments like this are a reflection of the huge impact the MARIETTA trial has had for real people, like Maureen Brewster.

Maureen was diagnosed with lymphatic cancer of the liver in 2011 and was under the watchful eyes of a consultant during her treatment. But in the summer of 2016, I started to have very extreme headaches, says Maureen.

After getting an emergency appointment, she was taken to A&E and admitted to hospital straight away. I was transferred to the National Neurology hospital in Russell Square for a biopsy. They thought I might have had a stroke. But it wasnt. Instead, Maureen was diagnosed with a secondary cancer in her brain.

When Maureen was transferred to UCLH, she was told about the MARIETTA trial. I could have chosen not to go on the trial, but being part of it meant that I would get more examinations and monitoring. So it was more reassuring to be on the trial, she says.

Maureen during treatment.

Maureen went through 8 tough months of chemotherapy before having a stem cell transplant in the summer of 2017. During one round of chemo in the hospital I became ill with an infection and really thought I was going to die. The last chemo prior to me having stem cell transplant was so strong it really had an impact on me and I couldnt eat I felt very poorly for a few weeks.

Maureens stem cell transplant went smoothly and prior to COVID-19, she was having regular check-ups and scans in hospital.

Prior to the first lockdown in March 2020, Maureen was able to do some volunteering and also go back to work, teaching a course on Project Management at a local adult college. In April 2019 I also secured a part-time job as a User Involvement Co-ordinator. It was great to get back to that level.

Dr Cwynarski emphasises that while the trial was a great success for some, it also exposed a group of patients who didnt do so well on the treatment.

The results threw up a real disparity and uncovered an unmet need in a particular group of patients. For the group of patients whose cancer had already failed to respond to a chemotherapy treatment, known as R-CHOP, at the follow up of 2 years, only 20% had not experienced their cancer progressing or getting worse.

We need to target this cohort of patients in a different way, says Cwynarski. So really the challenge is, can we identify experimental agents be it different biological agents or immunotherapies such as CAR T cell therapy in the patients who have relapsed, and maybe bringing these therapies into the frontline.

Lilly

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Transforming optimism: finding new ways to treat rare cancers - Cancer Research UK - Science Blog