Category Archives: Ohio Stem Cells

By Adam Owens, WRAL anchor/reporter

A 74-year-old Raleigh man spends an average of two weeks a month traveling around the world delivering stem cells or bone marrow to patients as part of Be The Match, a volunteer-based donor program.

According to Troy "Davis" Moore, Be The Match uses a database of 135 countries to find life-saving bone marrow or stem cells for patients with leukemia and other blood diseases. The stem cells are delivered from donors to patients around the world by volunteers like Moore.

Moore said, when he retired 17 years ago, gardening and working around the house just wasn't enough. His friend, who was already a volunteer courier with Be The Match, told him about the opportunity.

To date, Moore has logged more than 5,000 hours as a volunteer courier. He has traveled as far as London, Barcelona, Croatia, Portugal, Singapore and Taiwan.

On Thanksgiving week, Moore will travel to South America to pick up blood stem cells and deliver them to a patient in the United States. He had to get a rapid COVID-19 test before his trip.

"It's been a lot more challenging during COVID-19 because the rules have changed so much in these countries," said Moore, explaining he recently ran into some trouble in Croatia when he hadn't had a coronavirus test in 72 hours.

Moore said, although he doesn't get to meet the families he helps due to confidentiality, the job is incredibly special. In an interview with WRAL's Adam Owens, Moore described a trip he made on Christmas Day, 10 years ago, to deliver bone marrow from the United Kingdom to a hospital in Columbus, Ohio.

Moore said a nurse was walking him through the hospital hallways when she tapped on his shoulder and pointed to a patient room. Inside, he saw the parents of a child waiting for a transplant. "I have children and I could only imagine," Moore said. "They were just pacing down the room, waiting for it."

Moore said he plans to keep traveling until he can't anymore. Volunteering works well for him, he said, especially now that his children are grown.

Some people might get caught up in the adventure of travel, Moore said, but he's usually only in another country for one day.

"At some point, you realize that's not what it's all about," he said. "It's about getting [a cure] to someone."

Read more:
Raleigh man delivers stem cells to patients around the world - WRAL.com

Newswise CLEVELAND Three college graduations. Three family weddings. The births of two grandchildren.

Andy Superman Simon has cherished each of these milestones since he was diagnosed five years ago with a glioblastoma multiforme (GBM grade 4), one of the deadliest and most challenging cancers to treat. GBM patients typically survive an average of 12-15 months. Only 6.8 percent of GBM patients survive five years, according to the National Brain Tumor Society.

But the Superman of University Hospitals Seidman Cancer Center who memorably donned a full costume for his final treatment in September 2016 and is free of cancer today with no recurrence is anything but typical.

I feel incredible, says Simon, now 56. I flew through treatment with ease, because I had the best team and the best surgeon. The way I see it, I had cancer, I dont have it.

The crushing headache, similar to a migraine yet inexplicably and mysteriously different, struck early one morning in November 2015. Pulling out of his driveway to head to the ER, Simon was equidistant from two different hospital systems. He and his wife believe that fateful turn to come to UH Ahuja Medical Center, and then UH Seidman Cancer Center, has made all the difference.

If we hadnt gone to UH, I honestly believe in my heart that Andy wouldnt be here today, said Amy, Simons wife.

Neurosurgeon Andrew Sloan, MD, Director of UHs Brain Tumor & Neuro-Oncology Center and the UH Seidman Center for Translational Neuro-Oncology, diagnosed the large mass in Simons brain as a GBM. He performed a craniotomy on Simon using 5-Aminolevulinic Acid (5-ALA), an experimental agent that improves the surgeons ability to identify the tumor. Dr. Sloans own surgical trial assessing this agent was one of only a handful of studies in the United States at the time, though it is now approved for use throughout the US by the FDA. Simon took the 5-ALA prior to surgery, which causes the cancer cells to glow hot pink, for more complete removal of these aggressive, invasive tumors.

Radiation and chemotherapy are the standard of care following a craniotomy for GBM.

Simon also took advantage of a novel phase I clinical trial that involved genetically engineering his own blood cells to express a mutant protein that made them more resistant to chemotherapy enabling him to safely withstand steadily higher doses of toxic chemotherapy through six rounds. While this phase I trial was designed only to show safety and feasibility, the median survival of the participants was 3.3-fold higher than anticipated based on case-matched historical controls with GBM undergoing standard treatment.

A new clinical trial, funded by a $2.3 million grant from the National Cancer Institute and based on the gene therapy Simon participated in, will open at UH Seidman Cancer Center in the next few months.

Andy has been a champion, Dr. Sloan says of the poster-boy for this trial, noting that five-year GBM survivors commonly experience recurrence. Hes a real fighter.

This treatment is really a game-changer. This could be the new standard of care. Its really exciting and very promising.

For the last several years, Simon has celebrated with a big party complete with a photo display of his milestones. He was planning a blowout celebration this year until the pandemic struck.

There is hope, says Simon. I have too many things to fight for, and to live for. Ive gotten too far. Im going to be a statistic for the other side. Every day is a milestone really.

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About University Hospitals / Cleveland, Ohio

Founded in 1866, University Hospitals serves the needs of patients through an integrated network of 19 hospitals, more than 50 health centers and outpatient facilities, and 200 physician offices in 16 counties throughout northern Ohio.The systems flagship academic medical center, University Hospitals Cleveland Medical Center, located in Clevelands University Circle, is affiliated with Case Western Reserve University School of Medicine. The main campus also includes University Hospitals Rainbow Babies & Children's Hospital, ranked among the top childrens hospitals in the nation; University Hospitals MacDonald Women's Hospital, Ohio's only hospital for women; University Hospitals Harrington Heart & Vascular Institute, a high-volume national referral center for complex cardiovascular procedures; and University Hospitals Seidman Cancer Center, part of the NCI-designated Case Comprehensive Cancer Center. UH is home to some of the most prestigious clinical and research programs in the nation, including cancer, pediatrics, women's health, orthopedics, radiology, neuroscience, cardiology and cardiovascular surgery, digestive health, transplantation and urology. UH Cleveland Medical Center is perennially among the highest performers in national ranking surveys, including Americas Best Hospitals from U.S. News & World Report. UH is also home to Harrington Discovery Institute at University Hospitals part of The Harrington Project for Discovery & Development. UH isone of the largest employers in Northeast Ohio with 28,000 physicians and employees.Advancing the Science of Health and the Art of Compassion is UHs vision for benefitting its patients into the future and To Heal. To Teach. To Discover.is the organizations unwavering mission. Follow UH on Facebook @UniversityHospitalsand Twitter @UHhospitals. For more information, visitUHhospitals.org.

About University Hospitals Seidman Cancer Center

UH Seidman Cancer Center is the only freestanding cancer hospital in Northeast Ohio, where all clinicians and staff are dedicated to the prevention, diagnosis and treatment of cancer while researching new and innovative treatment options through clinical trials. Nationally ranked cancer care is also available to patients through the 11-country region at 18 community-based locations. Our UH Seidman specialists make up 14 cancer-specific teams focused on determining integrated care plans tailored to patients needs. UH Seidman Cancer Center is part of the National Cancer Institute (NCI)-designated Case Comprehensive Cancer Center at Case Western Reserve University, one of 50 comprehensive cancer centers in the country. Patients have access to advanced treatment options, ranging from a pioneering stem cell transplant program founded more than 40 years ago and a wide range of immunotherapy to the first and only proton therapy center in northern Ohio for adults and children. Go to UHhospitals.org/Seidman for more information.

Link:
A real life Superman celebrates 5 years of survival from one of the deadliest cancers - Newswise

Famous museums, like the Guggenheim in New York City or the Louvre in Paris, often reside in imposing buildings. Theresa Rayners USS Shenandoah Museum lives in a refurbished camping trailer towed behind a pickup truck. But inside this humble trailer is a priceless collection of memorabilia recalling the U.S. Navys first rigid airship, all lovingly curated and cared for by this Ohio woman with a passion for history and preservation.

The 680-foot-long USS Shenandoah was the pride of the U.S. Navy when christened in 1923. Its mission was to provide airborne surveillance for the fleet; to prove its capability, in 1924 the Shenandoah embarked on test flights across the United States. Soon, mayors, governors, and congressmen were requesting that the Shenandoah fly over their regions. In 1925, the airship was ordered on a tour of Midwest state fairs; it was on this trip, on September 3, that the Shenandoah was destroyed in an early morning squall above southeast Ohio. Of the 43 men on board, 14 were killed, including the captain and four other officers.

Engine parts, food from the galley, and personal effects of the crew were strewn about in fields and forests; shortly after the crash, thousands of looters rushed to the site and scavenged anything that could be torn loose or taken. The Department of Justice and the Ohio National Guard arrived to secure the area in late afternoon, but by then the damage was done. Government officials took a dim view of this rampant theft. In the weeks following the disaster, four truckloads of Shenandoah items were confiscated by federal agents in door-to-door searches throughout the region. Despite these efforts, many of the items became treasured family heirlooms, displayed for decades in parlors and living rooms. These are the artifacts that found their way to Rayners homemade museum on wheels, where visitors can see them up close and hold a bit of history in their hands.

We still get donations after all these years, says Rayner, like when someone is clearing out an older relatives home, and they come across a piece of Shenandoahs framework in the attic. Some families had these things hidden away for decades, fearing the government might come for it someday.

Rayners late husband Bryan grew up near Neiswonger Farm, where the bulk of the wreckage fell. As a boy, Bryan collected anything he could find relating to the wreck of the Shenandoah. His family has a connection to the event: The Rayners, along with other local residents, sold soda pop and water to the thousands who traveled to view the site.

News clippings, photographs, and pieces of the ship continued to accumulate in Rayners Garage in Ava, Ohio, the familys towing business. Bryan had quite a collection when we got married, says Rayner. And the couple showed the collection to anyone who asked to see it.

The idea of a more permanent museum came up after the Rayners were asked to set up a Shenandoah history display in a local store window. A portable museum was proposed, and someone traded the Rayners a used travel-trailer that fit the need. A neighbor built custom glass display cases, and other donated materials soon arrived. The USS Shenandoah Museum was ready for the road in 1995. The museum-on-wheels made the couples collection accessible to a whole new audience. We started visiting schools, scout meetings, church socials, and we gave tours of the museum to people just passing through, says Rayner. The increased exposure resulted in more donations and acquisitions. The Rayners bought a larger trailer when the collection outgrew the original.

Treasures displayed inside the museum include various pieces of the ships duralumin framework, sections of the silver outer cover, fragments of the airtight gas cells, plus assorted ropes and rigging. Items from the ships galley include cups and plates used by the crew, plus a sugar bowl with the sugar still inside. Theres an impressive scale-model of the ship, and original sheet music of the mournful song The Wreck of the Shenandoah by Maggie Andrews.

During the Depression, some people repurposed their Shenandoah relics into household items. We have a lampshade made from Shenandoah canvas, says Rayner. When this particular woman needed to replace a tattered lampshade, she stitched up a new one made from fabric she recovered from the wreckage. Theres also an aluminum wash basin from the ship that had been converted into a hanging planter, eventually discarded by the owner. We found that one behind a house in some weeds, recalls Rayner.

Occasionally, the Rayners would buy pieces at local auctions and rummage sales. Bryan was late for his own surprise 50th birthday party because hed heard that a six-foot-long section of Shenandoah framework was to be sold at an estate auction that day in a nearby town. He won the bid, and that relic is on display in the museum today. (Bryan died in 2013.) Rayner gets frustrated when she hears about items that have been discarded by families unaware of the value. To the untrained eye, what appears to be a worthless fragment of metal or canvas may be a piece of American aviation history.

Rayner gets the most satisfaction out of hearing the personal stories associated with the treasures. She tells visitors how the Shenandoahs captain, Lieutenant Commander Zachary Lansdowne, refused to leave his post during the storm. As the ship began to break up, he realized the control gondola was likely to rip loose from the hull. Lansdowne told the men around him to save themselves, and two men quickly climbed the ladder into the hull to safety. The gondola wrenched loose from the ship and fell to earth, killing Lansdowne and six other men who remained at their posts.

A farmhouse near where the gondola fell is empty today, but still standing. The farmer living there on the morning of the accident set up an aid-station in his kitchen for the airships survivors; his back porch served as a makeshift morgue until the coroners arrived.

Since Bryans death, Rayner shows the museum by appointment only. She guides visitors to the various wreckage sites, pointing out the hand-carved block of sandstone off Shenandoah Road that marks the exact spot where townspeople recovered Lansdownes body. The captains U.S. Naval Academy class ring was allegedly found there 12 years after the accident, grown into the stem of a mustard plant. In 1937, the federal government dedicated an impressive granite and bronze memorial in nearby Ava. The once-isolated Neiswonger Farm is now traversed by Interstate 77. Keen-eyed motorists may catch a glimpse of an American flag flying next to a donated sign in a meadow on the west side of the highway near mile-marker 32, a simple reminder of the history that took place there.

In 1991, the Rayners met Peggy Lansdowne Hunt, the daughter of the Shenandoahs captain, who was visiting Noble County to dedicate a memorial. During Hunts visit, the Rayners were able to share their collection with her. That was a turning point for me, says Rayner, meeting someone who was directly affected by this tragedy. Its one thing to know names, dates, and places. Peggy Lansdowne was just three years old when her father died. That changed her life forever. Thats something you have to factor into history.

Rayner still hopes for mementos from an event that occurred almost 100 years ago. There may be prizes still packed in an attic, awaiting discovery.

Read this article:
The Museum That Fell From the Sky | History - Air & Space Magazine

Detroit Lions Senior VP of business development Kelly Kozole works with her daughter, Morgan, who has a rare neurological disorder called beta-propeller protein-associated neurodegeneration, or BPAN.Michael Rothstein

TROY, Mich. Wearing a white T-shirt with a massive star in sparkling shades of pink, yellow and seafoam green on the front, Morgan Kozole sits in front of a fold-up chalkboard in the living room of her familys Detroit-area home and starts to draw.

Using pink and yellow chalk, she sketches Mickey and Minnie Mouse. The Disney characters are dominant fixtures in the 5-year-olds life and therefore become a soundtrack for the Kozole family: Morgan constantly saying Mickey, with her long, blond ponytail bouncing to whatever song happens to be playing on the Mickey Mouse Club.

These are the two Mickeys, Morgan says, pointing to the chalkboard. Her mother, Detroit Lions senior vice president of business development Kelly Kozole, explains that this is her way of communicating that she would like a visitor to draw Mickey too. If its close, Morgan accepts it. Another Mickey to fawn over.

For Morgans birthday earlier this year, the family went to Disney World. On this trip, the Kozoles saw what they had longed for: the potential of progress.

She knew where we were. She knew Mickey Mouse, Kelly said. Before, she wouldnt go to the characters, and now shes jumping up and down, hugging. She really, along those lines, is also really into birthdays.

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The Happy Birthday song. Before that, she was just kind of looking. Sometimes it was too much for her with everyone singing sometimes loud noises are too much. This year, we had to sing Happy Birthday to her three times.

Birthdays, for children, are happy occasions reasons for grand celebrations of progress toward adulthood. For the rest of Morgans family it is more complicated.

Morgan has a rare neurological disease called beta-propeller protein-associated neurodegeneration, known as BPAN. Its a disorder, more prevalent in girls than boys, that causes delayed development and seizures, communication issues and, sometimes, motor dysfunction. Its unclear exactly how many people are living with BPAN worldwide due to its rarity, although Dr. Sami Barmada, a scientist at the University of Michigan studying BPAN, said a rough estimate is about 500 to 600 people.

Its rare enough that Dr. Henry Paulson, the director of the Michigan Alzheimers Disease Center, said there are experts in neurodegeneration who are unfamiliar with BPAN. While Kelly is trying to advocate for her daughter and others with BPAN through fundraising for research, science only moves so fast.

The Kozoles understand that. So birthdays for the family arent always happy. They are a reminder of what could come.

That ticking time clock, Kelly said. Every birthday isnt exciting for me for her. Because its one year closer to when this bomb is going to go off.

BPANs rarity makes the reality heartbreakingly simple: There are very few effective treatments, little research and no cure. As Morgan learns how to organize her Peppa Pig characters and learns new words on her iPad her future looms.

At some unpredictable point in Morgans teen and adult years the average is around age 25, according to Barmada development will just stop. Progress will decline and, in some cases, disappear. Those afflicted with BPAN begin suffering from progressive dystonia parkinsonism making it difficult to walk, talk or stand.

Any day, Kelly said, it could be like, Oh, your daughters gone.'

WHEN MORGAN WAS born on Jan. 12, 2015, she was, largely, a healthy baby. She was a little jaundiced but nothing worrisome.

When she would go to the doctors office for shots, Morgan didnt cry. It was a little abnormal, but when youre a parent of a young child no crying is viewed as a minor miracle. Kelly and her husband, Kevin, took this as a sign of a tough kid. Nurses even said how great it was.

Looking back, it was a warning sign something was wrong. BPAN causes a high pain tolerance. Before long, more concerns popped up. Morgan wasnt crawling at nine months, wasnt walking at a year. Expected milestones passed without Morgan reaching them. Kevin and Kelly put her in therapy in late 2016 to work up to these childhood progressive traits and began researching potential causes. They wouldnt find an answer for more than two years.

Morgan Kozole suffers from BPAN, a rare neurological disorder, but still loves the same things any 5-year-old would, including the iPad and her favorite character, Peppa Pig.Michael Rothstein

She was diagnosed with cerebral palsy at first. One doctor diagnosed her with that, and then another, our neurologist said she doesnt have that, Kelly said. Then there was speculation but not a full diagnosis she had autism, so we did all the tests for that.

So through this kind of journey of trying to find out what was wrong, it was exciting that she didnt have something that you were going to this test for but you still had so many more questions as you were eliminating all these potential diseases that she could have.

Befuddled, they began genetic testing, and in November 2018 received a letter about a mutation on Morgans WDR45 gene. Kelly Googled it, stumbled upon BPAN and freaked out, calling their neurologist. The neurologist told Kelly not to worry BPAN was very rare, and Morgan didnt have it.

Doctors diagnosed her with epilepsy because of seizures. Morgan took Keppra, which helped accelerate her vocabulary to about 50 words, typical for a 1-year-old, when she was 3. Then doctors said, no, it wasnt epilepsy, either.

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Another meeting with another neurologist led to a different diagnosis. Three days after she and Kevin returned to Michigan from Super Bowl LIII in February 2019, they received a call. Doctors figured out what was wrong.

It was BPAN.

In my mind, its worse than cancer, Kelly said. How is this even possible? That this can even be so painful for kids later on in life. You try so hard to gain all these abilities, and then early adolescence or early adulthood, its just [gone] one day, and Ive seen a lot of these stories.

Theres a BPAN Facebook website, and thats where the doctors sent us. Theres no cure. Theres no therapy. Go to this website. Thats what I was told.

FOR MONTHS KELLY cried, angry and heartbroken. The Kozoles initially told their families and no one else.

In May 2019, Kelly went to her first Neurodegeneration with Brain Iron Accumulation (NBIA) conference. She met other parents, heard their stories and began the new normal.

She used her skills organization, fundraising and business to brainstorm ways to help. Hardly anyone researched BPAN. Without it, there would be no chance for a cure not in Morgans lifetime, which could reach her 40s, and not in the lifetime of those who might come after.

Kevin Kozole, husband of Lions senior VP Kelly Kozole, plays with his daughter, 5-year-old Morgan, who suffers from a rare neurological disorder called BPAN.Michael Rothstein

She shared what was happening with her boss, Detroit Lions president Rod Wood, and his wife, Susan, using a website link to explain BPAN. Wood knew something was wrong because of texts and emails saying they had to take Morgan to this specialist or that appointment.

As that was confirmed and became her reality, she is now able to talk about it in a way, Wood said. Because shes full bore on trying to help generate awareness and financial resources to find a cure for it.

She went from the unknown to the very tragic known to, OK, what are we going to do about it?'

Kelly consulted her aunts, both of whom worked in medicine. Linda Narhi worked in biotechnology for Amgen for more than 30 years; Dr. Diane Narhi was the first female chief of staff at Simi Valley (California) Hospital. From talking with another group of fundraising BPAN parents BPAN Warriors Kelly found a guide.

Any day, it could be like, Oh, your daughters gone.'

Kelly Kozole, Senior VP of Business Ops, Detroit Lions

If her aunts had not been resources, she might have joined BPAN Warriors. But Kelly admittedly needs to be in control, and this was her daughter. She needed to manage this herself. She created a nonprofit called Dont Forget Morgan.

Kellys aunts provided guidance, and Wood offered contacts he had in the finance industry and Silicon Valley. Wood and Lions general counsel Jay Colvin sit on the board. Other Lions coworkers with Woods blessing built the website, designed the logo and created social media plans and the first pitch video for Dont Forget Morgans rollout in 2020.

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Progress started with a $15,000 grant to help with a mouse model study at Sanford Research in South Dakota with another, larger, potential grant to come. In recent months, Kelly has focused largely on fundraising, and another parent of a child with BPAN, Christina Mascarenhas Ftikas, has focused on the medical side of the nonprofit.

This is why Im here, Kelly said. Im supposed to be a vehicle to get all of this awareness and hopefully a cure for BPAN so the child one, two, three, five years from now, there is hope.

There is no, Go to Facebook. There is something where you can actually give a parent, Heres the symptoms to look for.'

ABOUT AN HOUR away in Ann Arbor, Michigan, Kaci Kegler and her husband, Brian, had been in the same Facebook community. Kelly, new to the group and looking for a nearby connection, wrote Kaci a message.

Hey, my daughter was just diagnosed, could we connect?

Kaci understood. She did the same thing, reaching out without success in 2016 after her daughter, Elle, was diagnosed. Kaci wanted to be a resource.

They talked for an hour. There wasnt much Kaci could say to soothe her. Kelly pinged a year later with another message: Im starting a non-profit. Kaci offered to help.Despite suffering from BPAN, Morgan is like any other 5-year-old who enjoys playing with her brother, Connor.Michael Rothstein

Days later, on Feb. 28, Kaci and her husband, Brian, an assistant athletic director for development at the University of Michigan, had their yearly fundraiser for BPAN research on Rare Disease Day at Pizza House in Ann Arbor. They met a doctor who had a connection to researchers at Michigan.

I literally came home and texted [Kelly] and was like, Oh my gosh, we may have inroads, Kaci said. We just started texting. I have never met Kelly face-to-face. We still havent. But weve texted a lot and weve emailed quite a bit.

It just kind of started.

By summer, they went from nothing to putting pieces in place for a full-fledged research project with a two-year, $140,000 grant for Barmada and Dr. Jason Chua to help start to solve BPAN.

Chua was working on the regulation of autophagy, which is the cleaning out of damaged cells, and studying BPAN became a natural extension of the work he had already been putting in. BPAN alters that in neurons. Barmada said Chuas research provided a rare win-win situation to potentially help with BPAN and other diseases, too.

There are a set of questions in BPAN that nobody has the answer to, Barmada said. And Jason and myself, we just seem to be in the right position, the right place to be able to help out.

The goal is to understand what is happening within BPAN itself and how people end up with it while also trying to find therapies for existing patients. Within a year, they are hoping to grow stem cells from people with BPAN in their lab, allowing for the creation of their own stem cells missing the WDR45 gene. Then, they will try to either replace the gene or stimulate autophagy through genetic or pharmacologic means, Barmada said. The hope is this can prevent neurodegeneration.

So far, theyve hired a research assistant to work with Chua, developed tools to manipulate the gene using the genome-editing tool CRISPR and applied for approval from Michigan and the institutional review board to get skin biopsies to obtain stem cells from BPAN patients.

Its a process, but its also a start.

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Since partnering with Michigan and Sanford, Dont Forget Morgan also began working with Dr. Kathrin Meyer, a researcher at the Center for Gene Therapy at Nationwide Childrens Hospital at Ohio State.

Solving this disease is going to require more than Jason and Sami, Paulson said. Its going to be a first shot across the bow, but its going to require more than that. Ill say this being in the field for a long time. Scientists who are coming up the pike say they want to look at Alzheimers, want to look at epilepsy. They dont say, I want to look at a rare disease.

The only way to solve a rare disease is to get someone hooked. Sometimes when you hook a really good one, as I think we have with Jason here, you hook them for life and they make a difference.

MORGAN IS BOUNCING around the Kozoles suburban Detroit home on this late August day. They just returned from northern Michigan, and having two kids, especially one with special needs, makes tidiness unrealistic.

COVID-19 changed things. Morgan hadnt been to many of her therapies for months. Online school barely kept her attention. There was concern she would have regression in her learning. Instead, her speech advanced by being around Kelly, Kevin and her older brother, Connor, all day. She has sung more songs recently to help increase her vocabulary. Sometimes, shell listen 20 times in a row.

Even more than that, Connor said. They arent sure how much shes truly learning versus memorization. But it is something.Morgan Kozole has inspired her mother, Detroit Lions VP Kelly Kozole, to marshal researchers and other advocates to develop a cure for BPAN, and perhaps help future generations of children who live with the disorder.Michael Rothstein

The family gathers inside Morgans bedroom complete with a special Haven Bed with a zipper to keep her safe from wandering around at night, when she could accidentally turn on the stove and hurt herself or others as sleep disorders are another BPAN issue. She sits on the floor and starts playing with her small, yellow dollhouse and a fake ice cream maker. Kelly asks for an ice cream. Morgan makes one for herself instead and pretends to eat it.

Later, outside, Morgan kicks a soccer ball and plays a modified game of catch with a squishy football. Football, no surprise, is big. She says hike a lot. She knows that term, Kevin says, laughing.

In these moments, Morgan seems like any other young child. She attends St. Hugo of the Hills Parish School in Bloomfield Hills, Michigan, but has a one-on-one para nanny to help. She interacts with people, often overly affectionate.

Sitting at the kitchen table after playtime outside, she plays with Starfall, a childrens learning app, on her iPad. They hope it accelerates her word recognition. Morgan is entranced watching Farmer in the Dell and using her hands to eat orange slices and Cheerios. She needs a mirror in front of her to provide her a target for her mouth. She listens to books, another way to try absorbing information.

Morgan can now count to 20 and say three sentences in a row. Kelly and Kevin have tried to give Morgan a normal life in an abnormal situation, but they worry about the future what she wont have and wont be able to experience.

But Morgan has changed some of that outlook, too.

Focus on how she is so loving and has so much pure joy. A lot of parents of special needs [kids] say you can learn so much from these kids, and you really can, Kelly said. She is, every morning, just so happy, and Mama! Hugs and kisses to strangers. She has none of those behaviors you learn as an adult where youre not kind to people or you dont want to talk to someone.

She is just open arms, will give you a hug and is so loving, and its like, Wow, this is really what life is about.'

Read the rest here:
A Detroit Lions VP tries to avoid wasting her daughter from uncommon illness - The Shepherd of the Hills Gazette

U.S. biotechnology company races to get its COVID-19 therapeutic approved for access, which has previously shownpositive results inseverely ill patients

Vancouver, WACytoDyn Inc. (CytoDyn) a late-stage biotechnology companydeveloping leronlimab (PRO 140), a CCR5 antagonist with the potential for multiple therapeutic indications, announcedit is currently accepting only 155 more candidates across the country for its Phase 2b/3 registrational trial in patients with severe-to-critical COVID-19.

CytoDyn is currently enrolling COVID-19 patients in its Phase 2b/3 clinical trial in at least 13 hospitals in nine states (California, Georgia, Massachusetts, New Jersey, New York, North Carolina, Ohio, Oregon and Texas). Please visit the Companys website atwww.cytodyn.com. The sites currently enrolling patients are listed on the home page under Clinical Trial Enrollment, then click on COVID-19 Severe or Critical in the drop-down screen. Please visit the website frequently, as the list of hospitals is updated on a regular basis.

CytoDyn has already enrolled 235 patients in its trial, and the Data Safety Monitoring Committee (DSMC) recently recommended CytoDyn continue its study without modification to achieve its primary endpoint, based on the results the DSMC observed so far.

The DSMC also added that it will look at the unblinded data again, once an additional 58 patients are enrolled.

We are very encouraged the DSMC recommended we continue our trial without modification, said Nader Pourhassan, Ph.D., President and CEO of CytoDyn.We believe this result, combined with the promising data already demonstrated with emergency INDs in over 60 severe and critical COVID-19 patients, is an indicator of positive data.

Its our hope this means our study is not only proving to be safe, but effective. If it wasnt, the DSMC would have stopped our trial or requested modifications.But instead, the DSMC recommended we continue our study without modification, and indicated it would look at the unblinded data once we are 75% enrolled.This, to us, is a very strong signal of positive data, added Pourhassan.

The recovery of a young woman in California provides some hopeful evidence of the effectiveness of leronlimab, Pourhassan said. She had contracted COVID-19 and was in the hospital on a ventilator. Within 24 hours after receiving a single injection of leronlimab, the amount of oxygen she needed started to drop, and 2 days later, she was able to be removed from her ventilator and later, sent home. She believes our drug saved her life.

We are optimistic and look forward to the completion of our study, or possible early evaluation to warrant an Emergency Use Authorization (EUA) from the FDA, Pourhassan concluded.

About Coronavirus Disease 2019

CytoDyn completed its Phase 2 clinical trial (CD10) for COVID-19, a double-blinded, randomized clinical trial for mild-to-moderate patients in the U.S. which produced statistically significant results for NEWS2. Enrollment continues in its Phase 2b/3 randomized clinical trial for the severe-to-critically ill COVID-19 population in several hospitals throughout the U.S.; an interim analysis on the first 195 patients was conducted mid-October.

CytoDyn is currently enrolling patients in its Phase 2b/3 COVID-19 trial for patients with severe-to-critical indications in at least 13 hospitals and clinics across the U.S., which are identified in this press release and on the Companys website under the Clinical Trial Enrollment section of the homepage.

About Leronlimab (PRO 140)

The FDA has granted a Fast Track designation to CytoDyn for two potential indications of leronlimab for critical illnesses. The first indication is 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 completed nine clinical trials in over 800 people and met its primary endpoints in a pivotal Phase 3 trial (leronlimab in combination with standard antiretroviral 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 could 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 may play a role in tumor invasion, metastases, and tumor microenvironment control. 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 a Phase 1b/2 human clinical trial in metastatic triple-negative breast cancer and was granted Fast Track designation in May 2019.

The CCR5 receptor appears to play a central role in modulating immune cell trafficking to sites of inflammation. It may be crucial 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 support further the concept that the CCR5 receptor on engrafted cells is critical for the development of acute GvHD, blocking the CCR5 receptor from recognizing specific 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 CytoDyn

CytoDyn is a late-stage 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 critical 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 immune-mediated illnesses, such as GvHD and NASH.

CytoDyn has successfully completed a Phase 3 pivotal trial with leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients. The FDA met telephonically with Company key personnel and its clinical research organization and provided written responses to the Companys questions concerning its recent Biologics License Application (BLA) for this HIV combination therapy in order to expedite the resubmission of its BLA filing for this indication.

CytoDyn has completed a Phase 3 investigative trial with leronlimab as a once-weekly monotherapy for HIV-infected patients. CytoDyn plans to initiate a registration-directed study of leronlimab monotherapy indication. If successful, it could support a label extension. Clinical results to date from multiple trials have shown that leronlimab can significantly reduce viral burden in people infected with HIV. No drug-related serious site injection reactions reported in about 800 patients treated with leronlimab and no drug-related SAEs reported in patients treated with 700 mg dose of leronlimab. Moreover, a Phase 2b clinical trial demonstrated that leronlimab monotherapy can prevent viral escape in HIV-infected patients; some patients on leronlimab monotherapy have remained virally suppressed for more than six 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 atwww.cytodyn.com.

Forward-Looking Statements

This release contains 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. Forward-looking statements specifically include statements about leronlimab, its ability to have positive health outcomes, including for patients with COVID-19, the possible results of clinical trials, studies or other programs or ability to continue those programs, the ability to obtain emergency use authorization or regulatory approval for leronlimab for commercial sales, and the market for actual commercial sales. The Companys 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 results of the Companys clinical trials, including the possibility of unfavorable clinical trial results,(ii) the existence or development of vaccines, drugs, or other treatments that are viewed by medical professionals or patients as superior to the Companys products, (iii) the market for, and marketability of, any product that is approved, (iv) the sufficiency of the Companys cash position, (v) the Companys ability to raise additional capital to fund its operations, (vi) the Companys ability to meet its debt obligations, if any, (vii) the Companys ability to enter into partnership or licensing arrangements with third parties, (viii) the Companys ability to identify patients to enroll in its clinical trials in a timely fashion, (ix) the Companys ability to achieve approval of a marketable product, (x) the design, implementation and conduct of the Companys clinical trials, ((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 Companys control. The Company urges investors to consider specifically the various risk factors identified in its most recent Form 10-K, and any risk factors or cautionary statements included in any subsequent Form 10-Q or Form 8-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 release.

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US biotechnology company races to get its COVID-19 therapeutic approved for access, which has previously shown positive results in severely ill...

Adult stem cells are the foundation cells for every organ and tissue in our bodies. They are what can replenish damaged areas in virtually any part of our body. They are considered unspecialized cells but are able to transform into any type of cell that we are needing, such as a nerve or heart cell. And they are helping increase patients overall health daily.

Over 1 million patients have been experienced improved health worldwide after being treated with adult stem cells. The National Institutes of Health/FDA-approved database lists that there are nearly 3,500 ongoing or completed clinical trials.

Conditions Treated

Stem cell treatments and their clinical trials have been documented by scientific publications showing therapeutic success in a wide range of conditions that include:

Adult stem cell treatments for multiple myeloma and leukemia have even moved out of their clinical trial stages and are becoming standard medical practices for patients suffering from these conditions. For individuals with multiple myeloma or leukemia, a bone marrow transplant is the most common form of stem cell therapy that they are treated with.

How it Works

If you decide to visit Stem Cell International for stem cell therapy, the process will be comfortable and professional; our physicians have years of expertise working with stem cells treatments.

When you first come in, one of our physicians will meet with you and identify where the source of your bodys damage is present. Once this is discovered, they can begin to offer solutions on the best stem cell therapies that are most relevant to your needs.

We understand this process can be frightening to some because its so new. For this reason, we want to meet potential patients face-to-face so we can have an open and honest conversation about the best route moving forward.

If youre interested in stem cell therapy but arent sure which one is right for you, schedule an appointment with us today!

Follow this link:
STEM CELLS - Regenerative Medicine Institute of Ohio

A novel Bcl-2 inhibitor, APG-2575, has been granted an Orphan Drug designation (ODD) by the FDA for the treatment of patients with chronic lymphocytic leukemia (CLL), announced Ascentage Pharma in a press release.1

This marks the second ODD for APG-2575, after 1 was granted to the drug in July 2020 for the treatment of Waldenstrm Macroglobulinemia (WM).2

At present, CLL still presents considerable unmet medical needs. APG-2575 is a key drug candidate in Ascentage Pharma's pipeline targeting apoptosis. The APG-2575 received this ODD from the FDA shortly after the first ODD in WM, and this designation will be helpful in enhancing our communication with the FDA and expediting our development of APG-2575 in these rare cancer diseases," said Yifan Zhai, MD, PhD, chief medical officer, Ascentage Pharma, in a statement.1 All the policy support and incentives as a result of this ODD will help us accelerate the global clinical development of APG-2575, which we hope will soon offer additional treatment options for patients with CLL."

In hematologic malignancies, APG-2575 may selectively block Bcl-2 as a way to renew the apoptosis process in cancer cells. The first study of APG-2575 in CLL, as well as in small lymphocytic leukemia (SLL), is currently recruiting 35 patients with relapsed or refractory disease. In the phase 1b dose-escalation study (NCT04215809), patients will receive APG-2575 alone or in combination with other therapeutic agents. The primary end point of the study is dose-limiting toxicity, and the secondary end point is the maximum tolerated dose of APG-2575.

The study will follow a non-randomized 3 + 3 design at a starting dose of 200 mg given on day 1 of a 28-day cycle. The dose will be increased to 400 mg, followed by 600 mg, 800 mg, and 1200 mg.

To be included in the trial, patients must be 18 years or older with a histologically confirmed diagnosis of CLL/SLL, and ECOG performance status of 2 or lower, adequate bone marrow function, and a serum creatinine level of 1.5upper limit of normal. In part 1, patients will be eligible for dose escalation if they have received 3 or fewer prior lines of systemic therapy. Female patients are required to be postmenopausal for 2 years or surgically sterile prior to beginning treatment in the study.

Patients are excluded from this study if they have undergone allogeneic stem cell transplant within 90 days of joining the study, have active graft-versus-host-disease or are in need of immunosuppressive therapy, and/or have Richter's syndrome. The study also excludes patients with certain prior therapies and comorbidities that may interfere with APG-2575 treatment.

Multiple cancer centers in the United States are involved in the phase 1b study of APG-2575 including the Mayo Clinic in Scottsdale Arizona, City of Hope in Duarte, California, Dana-Farber Cancer Institute in Boston, Massachusetts, Novant Health in Charlotte, North Carolina, Grabrail Cancer Center in Canton, Ohio, Cleveland Clinic in Cleveland, Ohio, and Swedish Health in Seattle, Washington.

Outside of the realm CLL/SLL, APG-2575 is being investigated in other hematologic malignancies like WM, AML, and T-cell prolymphocytic leukemia. Studies of APG-2575 in these disease states are currently recruiting patients in centers in the United States, Australia, and China.

References:

1. Ascentage Pharma's Bcl-2 inhibitor apg-2575 granted Orphan Drug designation by the FDA for the treatment of chronic lymphocytic leukemia. News release. Ascentage Pharmaceuticals. September 7, 2020. Accessed September 8, 2020. https://prn.to/2ZiOqFJ

2. Ascentage Pharmas Bcl-2 inhibitor apg-2575 granted Orphan Drug Designation by the FDA for the treatment of waldenstrm macroglobulinemia. News release. Ascentage Pharmaceuticals. July 15, 2020. Accessed September 8, 2020. https://bit.ly/329A5gL

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FDA Grants Orphan Drug Designation to Novel Bcl-2 inhibitor in CLL - Targeted Oncology

Mostpeople know breastfeeding is one of the best ways to help a baby thrive. And now it seems a mother's milk has beneficial effects even when her child reaches adulthood.

New University of Toronto research has found that if people genetically at risk of becoming obese are exclusively breastfed as a baby it can help ward off weight gain when they're young adults.

The study is part of a growing body of evidence about the benefits of breastfeeding yet the World Health Organisation says nearly two out of three infants aren't exclusively breastfed for the recommended six months a rate that hasn't improved in 20 years.

READ MORE:Leona O'Neill: I'm very nervous about prospect of sending my children back to school

When asked, 80 per cent of the women who stopped breastfeeding before six months said they would have liked to continue for longer, but often lacked support and guidance.

"Our society is letting mothers down there needs to be much more investment in breastfeeding support and education," says NCT breastfeeding counsellor Cordelia Uys, a breastfeeding expert for the holistic new mums' wellness app Biamother (biamother.com).

"Breastfeeding confers numerous health protections on both mother and child and creates a strong sense of emotional connection. In addition, for a mother to see her baby growing and thriving on her milk can be one of the most satisfying and rewarding experiences of her life."

Here, Uys outlines ten surprising breastfeeding facts...

1. Breast milk is personalised medicine

There are numerous antiviral and antibacterial properties in breast milk that protect a baby from infection. These infection-fighting properties are being continually updated in response to the mother and baby's environment. When a mother's body encounters a new germ, her mature immune system will deploy millions of white blood cells to fight it off and quickly pass them on to her baby via her milk.

2. Breast milk contains stem cells

Every time a mother breastfeeds her baby, stem cells in her breast milk cross the baby's gut and into their blood, and then travel to all the baby's organs, including their brain. These stem cells are capable of becoming functioning cells all over the infant's body. It's believed they can boost and support the infant's optimal development and protect them against infectious diseases.

With antiviral and antibacterial properties, breast milk is personalised medicine, for one thing

3. Breastfeeding has to be learned

Many people think breastfeeding will come naturally to mothers, but in fact, for all female apes, breastfeeding is a learned behaviour. A juvenile female gorilla in Ohio Zoo, having been separated from her mother at a young age, had no idea how to feed her first baby. But during her second pregnancy, zookeepers had the inspired idea of asking human mothers to regularly breastfeed their babies in front of her. When her second baby was born, the gorilla immediately picked it up and put it to the breast.

In the past, human mothers would have learned how to breastfeed by watching relatives and friends. For this reason, it's a good idea for pregnant women who want to breastfeed, to spend some time with a friend who's successfully nursing her baby. The National Breastfeeding Helpline and apps can also offer advice on breastfeeding.

4. Over 95 per cent of women can produce all the milk their baby needs

The vast majority of women can make all the milk their baby needs and, contrary to popular belief, the size of a woman's breasts doesn't impact the volume of milk she can produce.

Milk production depends entirely on supply and demand: in the early months, milk needs to be removed effectively from both her breasts at least eight times in 24 hours for a mother's supply to be established and maintained. By far the most common reason for low milk supply is under-stimulation of a mother's breasts, either because her baby isn't feeding frequently enough or isn't removing milk effectively.

5. Breastfeeding acts as a natural painkiller

Breast milk contains natural painkillers called endocannabinoids. Breastfeeding before and during vaccination injections has been shown to reduce pain in babies.

6. Breastfeeding protects mothers against breast cancer

The Tanka Fisherwomen of Southern China traditionally only breastfeed their babies from their right breast. In the early 1970s, a medical student at a Hong Kong clinic noticed that if Tanka women developed breast cancer, in 79 per cent of cases, it was in their left breast. It was this observation that led to the discovery that breastfeeding is protective against breast cancer.

Breastfeeding expert Cordelia Uys

7. Breastfeeding shouldn't hurt

Pain is there to tell us something is wrong, and this is true for breastfeeding too. Pain and damage happen when a mother's nipple isn't positioned correctly in her baby's mouth. In the majority of cases, when a baby is well-positioning and deeply latched, breastfeeding will be completely comfortable. If breastfeeding hurts, it's important to seek out qualified support as soon as possible.

8. The temperature of a mother's breasts adapts to her baby's needs

A mother's breasts can warm up by 2C if the baby is too cold, and cool down by 2C if the baby is too hot. In fact, it has been shown that when newborn twins are placed in skin-to-skin contact with their mother, each of her breasts will heat up to a different temperature according to each baby's needs. This is called thermal synchrony.

9. Breastfeeding mothers get more sleep

Studies have shown breastfeeding mothers sleep on average 45 minutes more a night than mothers who formula feed. Human milk contains substances that promote sleep and calmness in babies. Mothers release the hormone prolactin into their own blood while breastfeeding, which helps them to fall asleep more easily.

10. Breastfeeding is carbon neutral

When a mother is breastfeeding, there is zero waste and no carbon emissions. Research at Imperial College London has shown breastfeeding for six months saves an estimated 95-153kg CO2 equivalent per baby compared with formula feeding.

:: National Breastfeeding Helpline (nationalbreastfeedinghelpline.org.uk): 0300 100 0212

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Breastfeeding 'even better than previously thought' - The Irish News - The Irish News

Biologic, or regenerative, therapies have altered the way many equine veterinarians treat problematic joints. Some of the most mainstream and popular modalities they currently use to manage osteoarthritis (OA) in horses are autologous conditioned serum, autologous protein solution, platelet-rich plasma, and mesenchymal stem cells.

Most biologic therapies involve collecting and concentrating the horses natural anti-inflammatory and regenerative proteins or cells so they can be injected into an area of pathology (disease or damage) in the same horse.

Autologous conditioned serum is a cell-free extract of whole blood that has been processed to contain high concentrations of interleukin-1 receptor antagonist protein (IRAP), a naturally occurring anti-inflammatory protein within the body. It is marketed under the trade names IRAP and IRAP II.

When preparing ACS, veterinarians collect venous blood in a proprietary syringe system that encourages porous glass beads to bind with white blood cells. During an incubation process the bound white cells release high concentrations of IRAP. The veterinarian then draws the serum off into small portions and freezes it for future injection into arthritic joints. In clinical studies of ACS, researchers have reported improved synovial membrane (joint surface lining) health, stimulation of natural IRAP production, and improved lameness.

Platelet rich plasma is blood plasma thats been centrifuged or filtrated to have a higher concentration of platelets than whole blood. Many horse owners are familiar with PRP and its use in tendon and ligament injuries; however, veterinarians are using it more regularly for treating joint disease.

One of platelets roles in the body is to modulate tissue healing. They do so by releasing growth factors and signaling molecules that initiate repair and promote anabolic (supporting tissue growth) effects. Veterinarians have capitalized on this ability by injecting high concentrations of platelets directly into damaged or inflamed regions. Because many PRP systems allow for stallside preparation, it is a convenient option for immediate treatment without the hassle of incubation or culturing in the lab, as is the case with ACS and stem cell preparation, respectively.

Historically, equine veterinarians have primarily used PRP to help treat soft tissue injuries. More recent work has led to intra-articular (in the joint) use with promising results. Although researchers have demonstrated how platelet-derived products work in vitro (in the lab) and veterinarians have seen promising anecdotal results in vivo (in the live horse), theyve yet to produce evidence-based confirmation of its clinical efficacy.

Mark Revenaugh, DVM, owner of Northwest Equine Performance, in Mulino, Oregon, says the main factors standing between researchers ability to gather objective data and establish a consensus on PRPs efficacy are the high variability among preparation systems, individual patient reactivity to the product, and an unknown ideal concentration of platelets for particular injuries.

Most practitioners cant always check how many platelets are being used, he says. Depending on the system, one veterinarian may be using 100,000 platelets/milliliter and another veterinarian may be using 1 billion platelets/milliliter. These are not the same treatments, even though both are called PRP. I would love to see an industry standard develop.

Overall, PRPs positive anecdotal results and relatively easy preparation make it a useful option for treating osteoarthritis (OA) in horses.

Autologous protein solution (marketed under the trade name Pro-Stride) is essentially a hybrid of ACS and PRP. Its two-step stallside preparation process involves separating whole blood and sequestering white blood cells and platelets in a small fraction of plasma. The veterinarian then concentrates the separate blood components by filtration, leaving a solution of white blood cells, platelets, and serum proteins that provides the anti-inflammatory mediators of IRAP and the platelet-derived growth factors of PRP.

In a 2014 study out of The Ohio State University, researchers revealed that an intra-articular APS injection can significantly improve lameness, weight-bearing symmetry, and range of joint motion in horses that dont have severe lameness or significant compromise to the joint structure.

Mesenchymal stems cells are adult stem cells that can direct regeneration and repair of damaged tissue. Veterinarians have used this type of stem cell as a treatment strategy for equine soft tissue injury for some time; its only recently that veterinarians have begun using them to treat OA, and its not fully clear how they work in this capacity. Researchers working on early stem cell studies hoped to establish evidence that stem cells injected into regions of injury would develop into the respective tissue. While this hypothesis proved to be incorrect, continued research has revealed that these cells might instead have anti-inflammatory effects and the ability to recruit other stem cells to the area that could, in fact, heal damaged tissue.

The two most common forms of mesenchymal stem cells are adipose (fat)-derived and bone-marrow-derived. Some study results have shown that bone marrow sources yield smaller concentrations thanbut are superior toadipose sources in their ability to differentiate into musculoskeletal tissue. Some encouraging data supporting the use of mesenchymal stem cells for treating OA exists, but researchers have only published a small number of studies with promising results. Equine veterinarians have used MSCs to treat intra-articular soft tissue injury (meniscal and cruciate damagecartilaginous tissues and ligaments that support the stifle), with successful anecdotal results. Theyve reported more variable outcomes when using it for primary intra-articular injuries.

Carter Judy, DVM, Dipl. ACVS, staff surgeon at Alamo Pintado Equine Medical Center, in Los Olivos, California, says he currently prefers to use PRP and APS for OA treatment over MSCs. However, he admits there is much to be discovered. What will be interesting to see is how manipulating the cells and providing them with different signals and markers can make their efficacy much more potent and focused, he says.

When weighing treatment options for horses with OA, veterinarians should base their decision to use a certain biologic modality on its cost, availability, and how a horse has responded previously.

Our knowledge base of how the biologics work is improving, but we are in the infancy of understanding, Judy says. Much of the use is based on the clinical response as much as is it on the scientific data.

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Regenerative Therapy Options for Horses With Osteoarthritis - TheHorse.com

This originally appeared in the July/August issue ofDiscovermagazine as "Titans of Immunity." Support our science journalism by becoming asubscriber.

For years, Melanie Mussons friends have marveled at her superpower: staying healthy no matter what germs are making the rounds. Colds and flu felled plenty of Mussons dormmates in college, but the viruses always seemed to pass her by. I never got sick once, she says. I got about five hours of sleep a night, I finished school in three years, and I worked 30 hours a week throughout. My best friends labeled me the machine.

Mussons ironclad immune system also set her apart at her first job. While she was working at an assisted living facility, her co-workers succumbed to a stomach virus that was running rampant. Undaunted, Musson offered to cover their shifts. There I was, the brand-new employee, getting as much overtime as I wanted. I wasnt worried that Id catch [the virus], because it just doesnt happen.

While the rest of us battle seasonal flu, chronic allergies and back-to-back wintertime colds, Musson and other immune masters glide through with scarcely a sniffle something University of Pittsburgh immunologist John Mellors sees all the time. People get exposed to the same virus, the same dose, even the same source. One gets very sick, and the other doesnt.

Its only natural to wonder: Why do some people always seem to fall on the right side of this equation? And could our own immune systems approach the same level with the right tuneup?

Doctors have noted natural variations in the immune response among people since Hippocrates time, but the reasons remained elusive for centuries. New research, however, is starting to illustrate just how your genes, habits and past disease exposures affect the character and strength of your immune response. These discoveries are helping to define the parameters of a race in which people like Musson have a head start and others have much more ground to cover.

The moment a virus, bacterium or other invader breaches your cells walls, your body rolls out a tightly choreographed defense strategy. The main architects of this process are a set of human leukocyte antigen (HLA) genes, which code for molecules that fine-tune the bodys immune response. So when a bacterium gets into one of your cells, your HLA genes churn out proteins that flag the cell as infected so that specialized immune cells will swarm in to destroy it. Other HLA genes activate cells that rein in the immune response, so it doesnt destroy more than necessary.

Like fingerprints, everyones HLA gene assortment is unique. Your HLA genes give you a broad repertoire of immune defense tactics, but that repertoire may be great for some microorganisms and lousy for others, Mellors says. Its not like theres one HLA type thats highly immune to everything. This genetic variation helps explain why you might catch every cold virus going around but havent gotten a stomach bug in decades. A Massachusetts General Hospital study found that some so-called HIV controllers immune stalwarts who dont develop AIDS from the virus HIV have HLA gene variants that prompt specialized cells to swarm in and attack proteins key to the virus function.

But your HLA genes arent the only ones that shape your immune resistance. The Human Genome Project has identified tens of thousands of gene variants that are more common in people who develop specific diseases and less common in people without these conditions.

Flagging these kinds of gene-disease links is a relatively simple matter, says immunologist Pandurangan Vijayanand of the La Jolla Institute for Immunology. After researchers identify a gene sequence thats linked to disease, however, they need to figure out what it is actually doing, says Vijayanand. How is this change in the sequence impacting the cell or causing the susceptibility [to disease]?

To answer this question, Vijayanand and his team are creating what they call an atlas, to catalog which proteins each gene produces and how these proteins change the function of different cell types. For example, he has identified a gene variant that makes people more prone to asthma a condition in which the body attacks its own healthy airway cells by driving high production of proteins that rev up the immune response. Other gene variants appear to help people fight lung tumors by prompting their tissues to produce more T lymphocytes, specialized immune shock troops that kill cancer cells.

While a dizzying number of genetic differences remain to be cataloged, immunologists agree that, in general, these differences help explain why resistance to some pathogens can seem to run in families. People like Melanie Musson probably get a genetic leg up to some degree Musson says her mother, father and siblings rarely get sick. Conversely (and unfairly), you might instead inherit a tendency to develop diabetes, recurrent strep infections or autoimmune diseases.

However anemic or hardy your innate immune arsenal, it supplies only the broad contours of your bodys resistance to threats. Environmental influences fill in the details, from where you live to your sleeping patterns to your history of previous infections.

In a 2015 Cell study, researchers studied more than 100 pairs of identical twins and how their immune systems responded to the flu shot. About three-quarters of the differences they saw were driven by environmental factors rather than genetic ones. The differences in twins immune systems also grew more pronounced the older they got, suggesting that outside influences continue to shape our immune potential over time.

Some of these influences show up in early childhood and may be hard to offset later on. Researchers have long known that children who live on farms are less likely to develop autoimmune diseases like asthma and allergies. An Ohio State University study from July 2019 hints at one reason why: Farm kids have a more diverse array of gut microbes than city kids, and the presence of some of these gut microbes predicts lower frequencies of immune cells that create allergic inflammation. Broad microbial exposure, in short, appears to train the immune system not to overreact to substances like animal dander.

But regardless of where you grew up, if youre unlucky enough to catch certain disease-causing bugs, they can throw your immunity off balance for years. Cytomegalovirus, a relative of the virus that causes chicken pox, stages its attack by reprogramming the human immune system. Some of the virus proteins latch onto certain immune cells, interfering with their ability to fight invaders. Other proteins, according to research from the University Medical Center Utrecht, interfere with the expression of key human HLA genes. And since cytomegalovirus infections are chronic, the resulting immune deficits can go on indefinitely.

Naturally, you cant control where youre raised or what random pathogens you acquire. But you can control your daily routine, what you put into your body and how you shield yourself against germs. In recent years, scientists have begun a full-fledged push to find out which lifestyle habits actually foster a robust immune system and which may be more hype than substance.

While the overall picture of how diet shapes immunity is still blurred, new studies do hint at the immune-strengthening effects of certain types of foods. Garlic, for instance, contains a sulfur compound called allicin, which spurs production of disease-fighting immune cells like macrophages and lymphocytes in response to threats.

(Credit: Lucky_Find/Shutterstock)

Researchers also report that specific bacteria-containing foods such as sauerkraut, kimchi and kefir produce an immunologically active substance called D-phenyllactic acid. This acid appears to signal immune cells, called monocytes, to report for duty by binding to a receptor protein on the cells surfaces. When people eat sauerkraut, very soon afterward, we see in the blood that theres an increase in the level of this substance, says Leipzig University biologist Claudia Stubert. In future studies, she hopes to clarify exactly how the acid affects monocytes activity in the body.

In addition to tweaking their diets, many titans of immunity embark on intense exercise regimens to keep their health robust. I swim and snorkel year-round in the ocean, up to a mile at a clip, from New England to Miami and a few secluded points in between, says Baron Christopher Hanson, a business consultant who claims he almost never gets sick. But so far, scientific proof that exercise improves immunity is limited. While a new study in rats shows that regular exercise changes the prevalence of different types of immune cells, it isnt clear whether these changes make you less likely to get sick.

Getting your daily quota of shut-eye, however, does seem to boost your immunity. Repeated studies show that sleep revs up your immune response, and a recent one from Germanys University of Tbingen reports that it does so in part by preparing disease-fighting T cells to do their jobs more effectively. Thats because your body churns out more integrins proteins that help T cells attach to germ-infected cells and destroy them while youre asleep.

But while getting more sleep could help snap your streak of winter colds, squirting your palms with hand sanitizer may not. In numerous studies, plain old soap and water was shown to kill germs better than sanitizer does. Hand sanitizer is great for alcohol-susceptible bugs, but not all bugs are susceptible, Mellors points out. Whats more, using sanitizer wont have any lasting effects on your immunity. The moment you touch another germy surface, your thin layer of protection will vanish.

Getting plenty of sleep is one way to boost your immune health: The body preps disease-fighting cells while youre asleep. (Credit: Realstock/Shutterstock)

Champions of immunity tend to credit their daily habits with keeping them healthy. But many have also lucked into an ideal balance between effector T cells, the frontline immune soldiers that fend off pathogens, and regulatory T cells, which keep the bodys immune arsenal in check so it wont over-respond to threats. An overactive immune system can be just as troublesome as an underactive one autoimmune conditions like rheumatoid arthritis, multiple sclerosis and allergies all stem from an immune response thats too forceful and sustained.

Last year, scientists at Kyoto University in Japan and elsewhere described one potential way to redress this kind of imbalance: turning effector T cells into regulatory T cells in the lab. Autoimmune episodes are triggered by antigens binding to [a] receptor on effector T cells, says molecular biologist Shuh Narumiya, one of the papers authors. When Narumiya and his colleagues used an inhibitor chemical to block an enzyme that controls cell development, cells that would normally develop into effector T cells turned into regulatory T cells instead a tweak that dialed down harmful autoimmune responses in mice.

While not everyone needs such immune fine-tuning, some people could potentially benefit from a treatment based on this technique, Narumiya says. Filling out the ranks of regulatory T cells could someday help keep a range of disabling autoimmune conditions under control.

Regardless of your T cell balance or your immune track record, theres a hefty dose of serendipity involved each time your immune system faces a threat. You might consider yourself forever prone to the flu or sniffles, but an X-factor a cross-country move, a dietary tweak, a new therapy can unexpectedly realign things and boost your immune potential.

By the same token, no matter how stalwart your HLA gene arsenal, how sound your sleep or how scrupulous your hygiene, you can end up knocked flat with a nasty bug when you least expect it. Immune health is like a gigantic roulette wheel. You throw the ball down and where it lands is a matter of chance, Mellors says. You have an encounter with a pathogen, and at the time you get exposed, your front line is not up to snuff. Even titans of immunity can have Achilles heels and even immune systems that seem licked at the beginning can pull off unlikely victories.

Its a recurring theme of the COVID-19 crisis: Those infected with the virus develop vastly different symptoms. Some barely feel anything a scratchy throat, if that while others spend weeks in the ICU with ravaged lungs, unable to breathe on their own. This wide variation in how people respond to SARS-CoV-2 stems, in part, from each persons unique genetic and lifestyle factors that affect their immune function.

(Credit: Andrii Vodolazhskyi/Shutterstock)

Scientists in Sydney and Hong Kong have found a particular gene variant tied to high rates of severe symptoms of SARS, a coronavirus related to the one that causes COVID-19. Because the novel coronavirus only recently appeared in humans, we dont know exactly which genetic quirks might make us more susceptible to it. Scientists are now investigating whether other specific genes might give some people higher or lower degrees of protection against the virus.

In some older people, or in those who have underlying immune deficits from chronic conditions, regulatory T cells which usually keep immune responses under control do not function normally. When these people get COVID-19, so-called cytokine storms may cause excessive inflammation in the lungs, leading to life-threatening symptoms. A study conducted by researchers in China found that COVID-19 patients with severe illness had lower levels of regulatory T cells in their bloodstream. Children may be less prone to disabling symptoms because their immune systems are better regulated and they have fewer underlying conditions.

SARS-CoV-2 uses a cell surface receptor called ACE2 to enter the cells that line your respiratory tract. New research shows that in smokers, these receptors are more prevalent in the lungs, creating more potential access routes for the virus. If you smoke, says Boston Childrens Hospital immunologist Hani Harb, the virus will be able to enter more cells in higher numbers.

Elizabeth Svoboda is a science writer in San Jose, California. Her most recent book is The Life Heroic: How To Unleash Your Most Amazing Self.

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Why Do Some People Get Sick All the Time, While Others Stay in Freakishly Good Health? - Discover Magazine