Be The Match Presents "Sickle Cell Connect" Virtual Event Series To Raise Awareness Of Treatment Options And Marrow Transplants As A Cure…

MINNEAPOLIS, Sept. 4, 2020 /PRNewswire/ -- Sickle Cell warrior Tahir "StayFresh" Ali of Atlanta, Ga., visits the hospital twice monthly for blood transfusions to reduce complications from sickle cell disease, and manage his potential risk of organ damage or stroke. The 35-year-old music producer and manager estimates he has spent several years of his life in the hospital undergoing treatment for sickle cell disease. Finding a perfectly matched marrow or blood stem cell donor could give StayFresh a chance to live life free of sickle cell and the many complications that the disease presents.

September is Sickle Cell Awareness Month, and Be The Match, the National Marrow Donor Program, is launching several campaigns to help people to learn more about the disease, and take action to help those searching for a matching blood stem cell or marrow donor.

StayFresh is like 100,000 African Americans who battle sickle cell disease, an often-misunderstood genetic disease that is "invisible" because unlike many illnesses, people with sickle cell disease can lead active lives. However, people with severe sickle cell can face debilitating episodes of pain crisis and life-threatening complications. StayFresh is working with Be The Match to rally more Black and African Americans to join the Be The Match Registry.

Be The Match is also hosting a three-part virtual event series to raise awareness about sickle cell disease, provide free resources for patients and families affected by sickle cell, discuss treatment options, and the need for more Black donors on the Be The Match Registry. The event series and more information can be found at: SickleCellConnect.com.

"We are grateful to have a dedicated Patient Services team that is able to produce these informative virtual events to help sickle cell patients and their caregivers learn how to access free resources and advocate for themselves as they navigate their disease," said Erica Jensen, Senior Vice President of Be The Match Member Engagement, Enrollment and Experience. "Nobody with sickle cell disease has to walk alone. Be The Match has a peer connect program, free professional counseling, nurse navigators and strong partnerships with organizations like The Sickle Cell Community Consortium and My Three Sicklers Foundation to ensure we can connect caregivers and sickle cell warriors with the resources they need to best manage this disease."

According to the Centers for Disease Control, sickle cell disease is the most common inherited disease, affecting 1 in 365 Black or African Americans and 1 in 16,300 Hispanic Americans. With sickle cell disease, oxygen-carrying red blood cells are misshapen, hard and sticky, which can result in them getting stuck in blood vessels and clog them. This can cause severe pain crisis, infections, organ damage, low blood counts, stroke and other serious health problems.

Be The Match has also launched an informative online video called "You Are My Match" to raise awareness of the need for more Black or African American donors on the Be The Match Registry to help Black patients battling sickle cell disease find a potentially life-saving match. The video features a 4-year-old girl named Ruby from Lubbock, TX, who has been hospitalized more than 24 times and had three surgeries due to her sickle cell. Ruby's doctors are recommending a blood stem cell or marrow transplant due to the severity of her condition, but she does not have a perfect match on the Be The Match Registry.

Part of the challenge Black patients like Ruby face when searching for a perfect match is low representation of Black or African Americans on the Be The Match Registry. Of the 22 million potential donors on the Be The Match Registry, just 4 percent are Black or African American. Because ethnicity plays a role in finding a match, Black patients will only find a perfect match 23% of the time, which is much lower than other ethnicities, such as White patients, who will find a match 77% of the time.

To learn how to access free resources, read stories about people cured of sickle cell disease following a marrow or stem cell transplant and to join the Be The Match Registry, visit: http://www.sicklecellconnect.com.

Virtual Event Series:Episodes will be streamed via http://www.sicklecellconnect.comwebpage and viewable on Be The Match Social Channels.

Episode 1: What is sickle cell disease?September 10thfrom 12:00 12:30 p.m. CDT We'll chat about sickle cell disease basics with pediatric hematologist Staci Arnold, MD, hear sickle cell warrior Genesis' storyandlearn about barriers and disparities that sickle cell patients face from the Health Equity Manager at Be The Match.

Episode 2: Living with sickle cell diseaseSeptember 17th from 12:00 12:30 p.m. CDT We'll chat with a Be The Match Patient Navigator and Social Worker about the free resources available to sickle cell disease patients and hear from the founder of My Three Sicklers Foundation.

Episode 3:The future of sickle cell diseaseSeptember24th from 12:00 1:00p.m. CDT We'll cover transplant and gene therapy with Staci Arnold, MD and a certified genetic counselor from BeTheMatch. Hear about our policy efforts for sickle cell disease, meet sickle cell warriorDakhiyon, seehow Nia Imani Franklin, former Miss America, is advocating for more diverse donors on the Be The Match Registry, plus watch a live swabbing event!

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Be The Match Presents "Sickle Cell Connect" Virtual Event Series To Raise Awareness Of Treatment Options And Marrow Transplants As A Cure...

Betty Lin-Fisher: Men, take care of your health – Akron Beacon Journal

Tom Fister has been a doting husband to his wife of 41 years, Sue.

Sue has been in and out of the hospital in the last year with complications after her second bout of acute myelogenous leukemia (AML). Last year, she had an umbilical cord stem cell transplant. That was 15 years after she first had the same leukemia.

Tom Fister, 69, was so focused on his wife and her care that the self-described "hard-headed man" ignored his own health to the point where he could have died, his doctor said.

The Copley man was on medication for high-blood pressure, but otherwise did not know he had other health complications.

He was slowly having a harder time walking from the parking deck at University Hospitals to go visit his wife daily before the COVID-19 virus shut down access to the hospitals and the shelter-in-place orders went into effect.

On March 13, Fister got up, showered and went in and checked himself into the emergency room.

"I spent a month in the hospital and three and a half weeks in the ICU," he said. "I pretty much just ran myself down."

When he went to the hospital, doctors discovered he was in acute heart failure and kidney failure and had pulmonary hypertension and severe aortic stenosis, which is a severe narrowing of the aortic valve, said Dr. Joseph Lahorra, a cardiothoracic surgeon and chairman of the Cleveland Clinic Akron General heart, vascular and thoracic department.

Severe aortic stenosis is a chronic and progressive disease that is fatal if untreated, Lahorra said.

"He basically was not following up with anybody because he was taking care of his wife," Lahorra said. Fister also was concerned if he got evaluated for his medical conditions, it would take away from his focus on his wifes care.

Fister is "a very altruistic guy," said Lahorra, and a prime example of men represented in a Cleveland Clinic study released earlier this week.

The national survey by Cleveland Clinic revealed that the COVID-19 pandemic is affecting both the mental and physical health of men. Some men are negatively impacted, while others are making healthier choices.

In an online survey among about 1,000 U.S. males 18 and older, Cleveland Clinic found that 77% of men report their stress level has increased as a result of COVID-19, 59% of men say they have felt isolated during the pandemic and nearly half (45%) of men say their emotional/mental health has worsened during the pandemic.

The survey was issued as part of Cleveland Clinics fifth annual educational campaign, "MENtion It," which aims to address the fact that men often do not talk about health issues or take steps to prevent them.

This year, the Clinic wanted to see what sort of effect the pandemic has had on men over the past six months and what their outlook is.

Here are a few other findings:

Three-in-five men (59%) feel COVID-19 has had a greater negative impact on their mental health than the 2008 recession.

66% of men say they rarely talk about the impact COVID-19 has had on their mental health.

Half of men (48%) have put off seeing a doctor for non-COVID-19 related health issues. That is even higher among men ages 18 to 34 (56%).

While some are struggling, COVID-19 has also inspired healthier habits in some. Nearly half of men (45%) feel healthier now and 22% are exercising more.

While 64% dont see an end to the COVID-19 outbreak in sight, the majority (71%) still remain optimistic about the future as the world continues to battle COVID-19, suggesting the pandemic hasnt dampened spirits completely.

"We couldnt take the temperature on the state of mens health this year without focusing on the one thing that has affected every person in this country and beyond," said Dr. Eric Klein, chairman of Cleveland Clinics Glickman Urological and Kidney Institute. "Many men are finding themselves in new and different roles as a result of this pandemic; for example, they are out of work or are working around the clock at home looking after kids with their partners all while worrying about their familys health and their own health. Its no surprise that mental health rose to the top as a critical issue in this years survey.

"We want to remind men that their health shouldnt take a back seat, especially during a pandemic," Klein said. "If youre experiencing symptoms that indicate a real health issue, dont be afraid to come to the hospital or schedule an appointment with a health care provider, either virtually or in person. Your health is too important not to."

For Fister, once he got to the hospital, doctors had to stabilize his health, including getting him on dialysis three times a week.

Doctors wanted to do whats called a TAVR procedure, a relatively new procedure that has been a paradigm shift for patients and doctors, Lahorra said. The procedure eliminates the need for open-heart surgery in some patients, which would obviously require a much-longer recovery time. In a TAVR procedure, the aortic valve is opened up through the groin and patients can go home the next day.

But for Fister, even proceeding with the TAVR was considered risky, given his initial medical issues. So doctors temporarily opened the valve with a balloon to give him a few months to stabilize his other health issues. He had the TAVR procedure in July, performed by Lahorra and cardiologist Dr. Anmar Kanaan.

Doctors also wanted to keep Fister out of the hospital during the height of the COVID-19 pandemic last spring because he was a severe risk.

"He is the exact patient who if he got COVID, he would not do well. He would do very poorly. We discussed that with him," Lahorra said.

Hospitals have seen patients who were avoiding getting care for fear of contracting COVID-19, to the detriment of their health, he said.

But now, however, data have shown hospitals are safe, even for people at high riks for COVID-19 complications, he said.

"This is a vital message to get out. Continue with your routine health care and certainly for more urgent acute problems, definitely get seen. The environment is very safe," Lahorra said.

Fister is grateful for what he calls his "medical miracle." He feels great now and just got trained to do peritoneal dialysis at home. His wife, Sue, was just moved to a rehab facility and he hopes she will be able to come home soon.

Fister said hes a fairly private person, but agreed to talk about his medical journey because "there are people who might be helped from this," he said. "If I can help some people, thats why were talking today."

Beacon Journal consumer columnist and medical reporter Betty Lin-Fisher can be reached at 330-996-3724 or blinfisher@thebeaconjournal.com. Follow her @blinfisherABJ on Twitter or http://www.facebook.com/BettyLinFisherABJ and see all her stories at http://www.beaconjournal.com/topics/linfisher

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Betty Lin-Fisher: Men, take care of your health - Akron Beacon Journal

Seniors suffer the most from antiquated FDA approval process | TheHill – The Hill

Americans have been heartbroken as report after report shines a light on the COVID-19 pandemic sweeping through nursing homes and decimating some senior community populations. Not only have our seniors been struck in their final years with a hideous virus, but they are also the victims of an antiquated drug approval process that is nearly six decades old and in need of drastic improvement.

The fact is, we already have a model that works, which can be adapted for use in America with minor changes.

While some streamlining of the FDA approval process has taken place, especially in the midst of the HIV/AIDS epidemic, it is still denying our seniors, their families and physicians the opportunity to make informed choices about the level of risk they are willing to take (advised by their doctors) with promising but not officially approved treatments, pharmaceuticalsand therapies. Let seniors, and anyone dealing with a serious medical condition, have the opportunity to make informed decisions.

The biopharmaceutical industry has also seen major changes since 1962, when Congress passed foundational legislation to ensure that consumers would not be the victims of unsafe and ineffective medications. The advent of biotechnology, gene therapy, artificial intelligence, and the internet contributed to improved treatment results for patients and longer life spans. Meanwhile, the FDA has made only incremental improvements (with exceptions) to the rules which shorten the review time required for a drug to receive FDA approval, including Priority Review, Accelerated Approval, Fast Track and Breakthrough Therapy Designation.

These programs reflect the difficulty the FDA and Congress have in balancing the need to protect the American people against the need to make new vaccines and treatments available to those who need them, especially when dealing with life and death and serious illnesses such as the novel coronavirus.

We do not need to reinvent the wheel; We just need to expand an existing program.

In 2014 the Japanese Health Ministry established an accelerated conditional approval pathway for regenerative medicines that enables more patients to access products like tissue grafts and stem cell therapies that have demonstrated initial safety and efficacy, but have not completed the many years of standard clinical testing required under the countrys formal approval process. This program,which is especially beneficial to Japanese seniors,actually builds upon the Parallel Track approval process that was initiated by the FDA in 1992 to allow AIDS/HIV drugs to be accessible immediately following successful safety testing, but before completion of the more rigorous and time-consuming efficacy trials. On Oct. 5, 1992 the first drug to be approved under Parallel Track, stavudine, became available, extending the lives of thousands of sick people before eventually receiving standard FDA approval.

By expanding Parallel Track, all drugs that treat serious and life-threatening illnesses would be conditionally approved once early stage clinical trials suggest that a medicine has a safety profile consistent with the severity of the disease being treated and a likelihood of patient benefit. All physicians would then be able to prescribe such treatments to fully informed patients. As part of the program, the productdeveloper would be requiredto collect relevant data and results from patient use. In addition, a public database would be established where physicians prescribing Parallel Track approved therapies would be required to post anonymized treatment results.

The public database would enable doctors, patients, and the public health community to quickly learn about the successes, failures, benefits, and side effects that patients using Parallel Track approved treatments are experiencing. The FDA already collects real-world data on the side effects associated with approved medicines, which it uses to monitor safety and keep labels updated. Expanding this program would result in additional real time comprehensive patient data.

Now is the time for the United States to take this bold step forward and create an accelerated approval pathway for developmental treatments that give patients and doctors greater control over their health care. ExpandingParallel Track to include a publicly available database of real-world treatment results would accelerate biopharmaceutical innovation, reduce development costs and lower drug prices while expediting new standards of care and cures.

Seniors cant wait 10-12 years to have a drug approved, nor can they pay for drugs that are priced to quickly recoup the $2.6 billion dollars it costs to gain approval. With the expansion of Parallel Track, more seniors will be able to walk out of hospitals and live longer healthier lives.

Diane Abbitt is a Los Angeles attorney who played a leading role in the 1990s campaign to secure early access to not-yet-FDA-approved drugs to treat AIDS. Bartley J. Madden is an independent researcher in Florida. Japans conditional approval pathway was based on a proposal in his book, Free to Choose Medicine: Better Drugs Sooner at Lower Cost.

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Seniors suffer the most from antiquated FDA approval process | TheHill - The Hill

Father of Three with Incurable Cancer is Helping Researchers Get One Step Closer to a Cure – The Suburban Newspaper

In 2016, Danny Wade, a successful marketing professional and an active and doting father to his three young children, aged 11, 8 and 6, checked himself into the emergency department when he began experiencing severe, inexplicable bone pain and unusual fatigue.

Two days later, after undergoing a battery of tests, Danny was diagnosed with multiple myeloma, a little-known and incurable cancer of the plasma cells. He was just 42 years old.

"I was shocked when I got the news, Danny recalls. What upset me most was when the doctor told me that the average life expectancy for myeloma patients was only five to seven years. The thought that I would not see my children grow up was devastating. I knew I had to fight for my life.

Thats exactly what Danny has been doing. Within six months of being diagnosed, Danny went through a difficult high-dose chemotherapy regimen to prepare for an autologous stem cell transplant using his own stem cells. Then in 2017 after further tests, Dannys doctors recommended that his best option for survival was to undergo another transplant with stem cells from a healthy donor. He took his doctors advice and underwent the procedure. Fortunately, he was eligible to participate in a clinical trial at Maisonneuve-Rosemont Hospital where he received a breakthrough therapy involving bi-weekly injections that he will continue to take for a year.

Dannys condition is relatively stable at this time, and he extremely thankful to still be alive. He is thrilled to watch his children grow and to resume being an active part of their lives. He credits his survival to the life-saving treatments that he has access to and the love and support he receives from his partner, Anik. With my beloved Anik by my side, Ive had the courage to get through this nightmare and to have faith that I can get through whatever else the future holds.

Danny is eager to do what he can to help others living with myeloma. I made a promise that once I was doing well, I would do everything in my power to help find a cure so that other patients dont have to live through the horrors I have," says Danny. Danny is a member of the organizing committee of the Montreal Support Group, and recently co-founded the South Shore Myeloma Support Group.

Over the past four years, Danny has seen, first-hand, the life-changing impact that advances in myeloma research are having on the lives of those living with this incurable cancer. Thats why he and his family are more intent than ever to raise as much awareness and funds for myeloma as they can, and will be participating in Myeloma Canadas 12th annual Montreal Multiple Myeloma March on Sunday, September 20, at 10 am.

This years Montreal March has been modified to help stop the spread of COVID-19. In compliance with physical distancing measures, participants are encouraged to hold their own walk in their neighbourhood at the same time as the regularly scheduled March on September 20. Danny and his fellow Montreal Marchers have set their fundraising goal at $60,000 to help further crucial research for this deadly blood cancer that affects nine new Canadians every day.

Myeloma research has produced extremely promising results over the past two decades. In fact, for the first time, theres a cure in sight, says Dr Richard LeBlanc, Medical Hematologist and Oncologist, and holder of the Myeloma Canada Chair in Multiple Myeloma Research at the Universit de Montral. We cant afford to let the current situation stop the progress weve made and put vulnerable people living with myeloma at risk, which is why its more crucial than ever to invest in research and find a cure.

The Multiple Myeloma March, Myeloma Canadas flagship fundraiser is now in its 12th year. The annual five-kilometer event brings Canadian communities together to raise essential funds for research and to help improve the lives of all Canadians impacted by myeloma. Montreal is one of a record 33 communities across the country to be included in this years Multiple Myeloma March. The national fundraising goal is set at $650,000. To learn more about how this event will be working, please click here.

While this years March will undoubtedly be different because of the pandemic, its crucial to stay positive, says Martine Elias, Executive Director of Myeloma Canada. Fundraising has taken a huge hit for many organizations. We need to do all we can to increase awareness and raise essential funds for research that will improve the lives of Canadians impacted by myeloma, and bring us closer to a cure, Martine added. As we mark Myeloma Canadas 15th anniversary, we celebrate the strength of our incredible community. More than ever, were counting on our supporters to help us achieve our goal of $650,000. Canadians impacted by this incurable cancer are depending on us.

This year, a minimum of 50% of funds raised by the Multiple Myeloma March will go directly to support Myeloma Canadas Myeloma Research Priority Setting Partnership (PSP), the first program of its kind in myeloma. The PSP will use input provided by the Canadian myeloma community to identify and define investments in myeloma research over the next 18 months. The balance raised will go toward supporting various myeloma research projects and initiatives that are pivotal for improving quality of life and moving the needle toward a cure.

Multiple myeloma, also known as myeloma, is the second most common form of blood cancer. Myeloma affects a type of immune cell called the plasma cell, found in the bone marrow. Every day, nine Canadians are diagnosed, yet in spite of its growing prevalence, the disease remains relatively unknown. While there is no cure, people with myeloma are living longer and better lives, thanks to recent breakthroughs in treatment. To find the cure, more funding and research are required. To learn more, or to donate, please visit http://www.myeloma.ca

Myeloma Canada

http://www.myeloma.ca

AB

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Father of Three with Incurable Cancer is Helping Researchers Get One Step Closer to a Cure - The Suburban Newspaper

Editorial: UT Healths new teaching hospital will boost region – San Antonio Express-News

A planned 144-bed teaching hospital on the UT Health San Antonio campus slated to open in the spring of 2024 will boost enrollment for many health programs so crucial for the region and state.

The medical school alone could see an increase of 22 students in each enrollment class. The number of medical residents could grow by 100 to 130 slots by 2027. Currently, 1,000 residency slots are offered through UT Health San Antonio.

This is great news for medical and other students training for health professions who want to complete their education in Texas. It will help address the shortage of providers in Bexar County and across the state. Historically, many doctors remain in the communities where they complete their medical training.

The San Antonio hospital would become the fourth operated by the University of Texas System. The others are in Dallas, Galveston and Tyler.

Most of the UT System health science centers have partnerships with independently managed hospitals in their communities.

Most new hospital projects arrive with much fanfare. This project has been quietly moving along over the past year with little public awareness or discussion.

The University of Texas System regents last month placed the nearly $400 million project on its capital improvement program and are expected to appropriate construction funds at their November meeting.

The hospital will be located on 12 acres in the South Texas Medical Center provided by the San Antonio Medical Foundation. The eight-story hospital will be adjacent to the Mays Cancer Center, formerly known as the Cancer Therapy and Research Center. The jointly run UT Health San Antonio-MD Anderson program is a National Cancer Institute-designated research center. The hospital and the cancer center will be connected by a skybridge.

A 530-space parking garage is also included in the project.

Background information provided to the regents describes a high-acuity hospital offering treatment programs in cancer, neurosciences, orthopedics, urology, thoracic surgery and bariatrics.

A distinct, competitive advantage of the hospital will be the unique leading-edge therapies and early-phase clinical trials in the many disciplines in which the university has expertise, including immunologic and stem cell therapies in oncology and diabetes, the backgrounder states.

The construction of a second teaching hospital in San Antonio raises concerns about the multibillion-dollar investment taxpayers have made in the Bexar County Hospital District, which includes University Hospital and some two dozen University Health System clinics.

University Hospital has long been the primary teaching hospital for UT Health San Antonio, and much of its recent expansion including the new 400-bed Womens and Childrens Hospital now under construction alongside University Hospital has been in large part to accommodate the patients seen by UT Health San Antonio doctors.

Will the two be duplicating services and competing for patients?

UT Health San Antonio President William L. Henrich and Bexar County Judge Nelson Wolff said the two hospitals will work in partnership. The hospital district and UT officials are negotiating a memorandum of understanding to that effect, Wolff said.

Henrich said the new hospital will not replicate any services and will complement what is currently offered. He said the new facility will allow for the expansion of the health science centers three primary missions clinical care, biomedical research and education.

Our clinical practices have been growing 8 to 10 percent a year, Henrich said. We have about $300 million in research on campus, and we need more access to clinical trials. Sophisticated clinical trials in cancer, rheumatology, gastroenterology and this hospital will afford us the opportunity to carry out those trials, and that will be a unique feature of this hospital.

In addition, the new hospital is expected to create 750 to 850 new jobs.

This is an important project for San Antonio and Texas. It will expand higher education opportunities, help address the doctor shortage in the state and provide a lift to the local economy by creating skilled jobs.

We look forward to a thriving partnership between UT Health and the University Health System.

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Editorial: UT Healths new teaching hospital will boost region - San Antonio Express-News

There is a dire need for ‘donors of colour’ in the fight against blood disorders – Health24

Compiled by Gabi Zietsman | Health24

04 Sep 2020, 02:45

Two hikers are going to great heights to increase awareness of blood disorders, and the urgent need for bone marrow donors in South Africa.

In a symbolic hike up the country's highest peak in the Drakensberg range, adventurer and bone marrow donor Clayton Coetzee and expert mountaineer and author Gavin Raubenheimer will be tackling Mafadi from 7 to 9 September in honour of those who have helped save the lives of countless people suffering from blood disorders.

Bone marrow donation is close to my heart as one of my dearest friends got sick with Acute Myeloid Leukaemia (AML) several years ago," says Coetzee. "Its then when I heard about the SA Bone Marrow Registry and the life-saving work that they do, and I decided to sign up as a donor."

READ | Could smoking lead to this blood disorder?

Increase in cases

Blood cancer is one of the most common of these disorders and affects children the most, while other blood disorders include non-Hodgkin lymphoma (NHL).

Unfortunately, according to the Search Coordinator for the SA Bone Marrow Registry (SABMR) Alicia Venter, these diseases have increased in the last decade 45% in NHL cases and 26% in leukaemia cases.

"While recommended, prevention efforts, such as lifestyle changes, tend to be less effective for hematologic malignancies than for other cancers, which makes a blood stem cell transplant a patients only hope for survival," says Venter.

In order to be a suitable bone marrow donor, your human leukocyte antigen (HLA) needs to match someone in need. HLAs are genes in a human's DNA that help regulate immunity and affect whether or not a recipient's body will reject a transplant.

READ MORE | Bone marrow transplants less risky now

Need more non-white donors

Finding a match, however, isn't as easy as swiping on a dating app and SAMBR has a serious lack of diversity in the donor database. Currently, there are only about 74 000 local donors on the South African Bone Marrow Registry.

In South Africa, there is a dire need for donors of colour," explains Venter.

"When it comes to matching HLA types, a patients ethnicity plays an important role as HLA markers are inherited. Some ethnic groups have more complex tissue types than others, therefore finding a match is most likely to come from someone of the same ethnic group."

'As easy as giving blood'

The hike is supposed to be symbolic of the uphill battle that faces those suffering from blood disorders, including finding a suitable donor to increase their chances of survival. Besides the gruelling experience, SAMBR will also be doing a cheek swab drive at the same time in Agulhas, Western Cape, and Musina, Limpopo, to help boost donor registrations.

According to Coetzee, it's easy to be registered as a donor all it takes is a cheek swab. If you do match with someone, it's "almost as easy as giving blood".

If a match is found, a donor will undergo a full medical exam to look for any exclusionary factors like obesity, HIV status, other chronic conditions and viral infections. Once cleared, the donor goes on a five-day treatment of injections to increase the number of stem cells in the bloodstream.

On the fifth day, the donor will be admitted to a hospital and connect to a cell separator machine, where the bone marrow donation would be made. The collected samples then has 72 hours to be transplanted to the receiving patient.

In South Africa, doctors will seldom collect bone marrow straight from the source.Possible side effects to receiving the injections may include headache, bone pain and flu-like symptoms.

I want people to know that blood diseases can affect anyone, regardless of ethnicity or gender. Extending beyond our boundaries or comfort zones like climbing Mafadi can be hard, but imagine the indelible difference the act of becoming a donor can have on someone elses life and their loved ones, says Coetzee.

You can follow the hikers' journey onSAMBR's Facebook page.

If you are between the ages of 18 and 45 and want to become a donor, contact the SABMR on021 447 8638 oremail:donors@sabmr.co.za.For more info and how to donate, visit their website.

READ MORE | Leukaemia survivor stories

Image credit: Pixabay

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There is a dire need for 'donors of colour' in the fight against blood disorders - Health24

Stem Cell Trial Tests Treatment for Often-Fatal COVID-19 …

By Jen A. Miller

Researchers are studying whether cells drawn from deep inside our bones may hold hope for the sickest of COVID-19 patients who have severe lung injury called acute respiratory distress syndrome, or ARDS.

The clinical trial, led by Michael Matthay, MD, of UC San Francisco, is testing the effects of infusions of stem cells drawn from bone marrow in the hopes of finding a therapy for the deadly syndrome.

For a patient suffering from ARDS, tiny air spaces in the lungs fill up with fluid and prevent normal oxygen uptake in the lungs, said Michael Matthay, MD, professor of medicine at the UCSF School of Medicine. Thats why the patient has respiratory failure. Usually these patients have to be intubated and treated with a mechanical ventilator.

There is not currently any drug to treat ARDS, which has a mortality rate of 27 percent for mild cases and 45 percent for the most severe cases. An early study on COVID-19 patients with ARDS found that only about 25 percent survive, though that percentage may be as high as 40 percent, according to Matthay.

Patients in a randomized double blind clinical trial are being given mesenchymal stem cells (MSCs) or placebo to test the effectiveness of the therapy. The trial ongoing at UCSF, Zuckerberg San Francisco General Hospital, and soon to expand to UC Davis and four other sites in September hopes to repair the severe and often fatal lung damage in people suffering from ARDS.

Its a very interesting possible therapy, said Matthay. Were really in the early phases of understanding these cell-based therapies.

MSCs are stromal cells found in bone marrow, play a key role in how our bodies make and repair skeletal tissues things like bone and cartilage and support the cells in the bone marrow that produce red and white blood cells. Once researchers discovered that MSCs could modulate inflammation when transplanted into other people, they began exploring them as potential therapies for a range of diseases and traumas.

For example, MSCs are often used to stop graft versus host disease, which is when transplant patients from rejecting new organs or bone marrow. More recent studies have focused on an array of potential disease targets because MSCs have properties that may be useful to treat a variety of human illnesses, said Matthay. Those include autoimmune diseases like Crohns disease and multiple sclerosis, and severe, acute conditions like sepsis and ARDS.

ARDS can be caused by trauma, bacterial infection, or a viral infection like COVID-19. In an earlier trial, Matthay and his team showed that MSCs are be safe to use, and that they have promising benefits in treating patients with ARDS. But they do not know how effective the MSC infusions are, and if results vary depending on what caused ARDS in the first place. Would they work better in a patient whose ARDS was caused by trauma? Or maybe they work better in concert with antibiotics used to treat ARDS that was set off by a bacterial infection.

ARDS is a syndrome of acute respiratory failure that we understand reasonably well, but theres a lot of heterogeneity in terms of the different mechanisms of injury to the lung and also outcomes, he said.

In this Phase 2 double blind clinical trial being funded by the Department of Defense, the National Institutes of Health, and the California Institute of Regenerative Medicine researchers are enrolling 120 ARDS patients. Enrollees are adult ICU patients who are on ventilators with excess fluid in their lungs, and no sign of heart failure as a primary cause of the respiratory failure.

Half of the patients will receive MSCs and half a placebo. As of late August, 28 patients were enrolled in the study, which will run until researchers reach 120 enrollees. The MSCs used in the study have been donated by young, healthy adults via an NIH-supported cell laboratory at the University of Minnesota.

When Matthay launched a new study in early January, he didnt expect it be a COVID trial. Thats because COVID as far as was known then wasnt in the U.S. But thats exactly what it inadvertently became since many patients who met the ARDS study criteria were suffering the condition because of COVID-19.

We started the study in January 2020, and then COVID-19 hit, so we have been enrolling patients over the last eight months, he said. Most of the patients weve enrolled in the trial have ended up having severe viral pneumonia from COVID.

Based on these outcomes and further studies on MSCs, Matthay sees them as a possible route for cell-based therapy. Much like cancer treatment isnt one approach but instead different therapies tailored to the type of cancer cells, use of something like MSCs could be perhaps tailored exactly to whatever specific kind of syndrome of respiratory failure.

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Stem Cell Trial Tests Treatment for Often-Fatal COVID-19 ...

Allogeneic Hematopoietic Stem Cell Transplant Effective for Patients with CLL Previously Treated with Novel Agents – Curetoday.com

Novel agents are becoming an important part of the chronic lymphocytic leukemia landscape as they improve outcomes for patients, but the number of novel agents used prior to allogeneic hematopoietic stem cell transplant (alloHCT) and after do not have an impact on survival outcomes.

AlloHCT was the standard of care for patients with early relapse or refractory chronic lymphocytic leukemia (CLL) and provided an opportunity for long-term disease control in the absence of other treatments. Now, novel agents such as Imbruvica (ibrutinib), Calquence (acalabrutinib) and Venclexta (venetoclax) are improving response rates, progression-free survival (PFS) and overall survival (OS) in patients and paving the way for long-term disease control depending on sequential treatment. Moreover, these novel agents dont come with the same risks as alloHCT such as graft-versus-host-disease (GVHD), organ toxicity and non-relapse mortality (NRM).

However, some patients still face initial barriers with these novel agents and all of the novel agents may not work for them, but researchers have determined that alloHCT remains an option for these patients and is not adversely impacted by novel agents. In a study of 65 patients with CLL who underwent one or more treatments with novel agents, whether prior to alloHCT or after, researchers found a two-year PFS of 63%, OS of 81%, NRM of 13% and relapse incidence of 27%. Moderate to severe GVHD developed in 27% of patients.

The data presented here support consideration of alloHCT for novel agents treated, fit patients, with responding disease, the authors wrote, and should be considered particularly for patients in whom novel agents are not expected to provide long-term disease control.

Ninety-two percent of patients received novel agent immediately prior to alloHCT treatment with a 6% complete response for those receiving Imbruvica, 97% of patients then discontinued it before alloHCT treatment. Patients receiving Venclexta-based therapy prior to alloHCT had a 52% complete response and 95% discontinued treatment prior to alloHCT with intolerance of the therapy in 5%.

After alloHCT treatment, researchers looked at patients in two arms, those who were exposed to one novel agent versus two or more novel agents with similar poor risk features and a similar median age of 60. Both groups had similar PFS and OS outcomes that were not significantly different and that exposure to these novel agents prior to alloHCT were also predictors of PFS after alloHCT treatment with no significant differences between the arms. The predicated outcomes were initially estimated at 66% PFS and 94% OS. Those results suggest that alloHCT is still a meaningful treatment even when patients with CLL are exposed to novel agents, the authors explained.

Taken together, this study suggests that alloHCT is safe and effective in patients with CLL exposed to one or more novel agents prior to alloHCT, they concluded. These data support the use of alloHCT for well-selected patients with high-risk CLL.

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Gamida Cell Announces Two-Part Virtual Miniseries Focused on Omidubicel to Take Place in September – Business Wire

BOSTON--(BUSINESS WIRE)--Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to cures for blood cancers and serious blood diseases, today announced that it will host a two-part virtual miniseries in September focused on key topics related to omidubicel, an advanced cell therapy in Phase 3 clinical development as a potentially life-saving treatment option for patients in need of a bone marrow transplant. The webcasts will take place on Friday, September 11, 2020, and Wednesday, September 23, 2020, both at 1:00 p.m. ET.

The miniseries topics are as follows:

In May, Gamida Cell reported that its Phase 3 study of omidubicel met its primary endpoint, demonstrating a highly statistically significant reduction in time to neutrophil engraftment, a key milestone in recovery from a bone marrow transplant. Gamida Cell expects to present the full data set, including secondary endpoint data, at a medical meeting in the fourth quarter of 2020. The company also expects to begin submitting the biologics license application for omidubicel to the U.S. Food and Drug Administration (FDA) on a rolling basis in the fourth quarter of 2020. Omidubicel is the first bone marrow transplant product to receive Breakthrough Therapy Designation from the FDA and has also received Orphan Drug Designation in the U.S. and EU.

Each webcast will be available on the Investors & Media section of the Gamida Cell website at http://www.gamida-cell.com. A replay of the webcast will be available about two hours after the event, for approximately 90 days.

About Omidubicel

Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers). In both Phase 1/2 and Phase 3 clinical studies (NCT01816230 and NCT02730299), omidubicel demonstrated rapid and durable time to engraftment and was generally well tolerated.1,2 Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia (NCT03173937). The aplastic anemia investigational new drug application is currently filed with the FDA under the brand name CordIn, which is the same investigational development candidate as omidubicel. For more information on clinical trials of omidubicel, please visit http://www.clinicaltrials.gov.

Omidubicel is an investigational therapy, and its safety and efficacy has not been evaluated by the U.S. Food and Drug Administration or any other health authority.

About Gamida Cell

Gamida Cell is an advanced cell therapy company committed to cures for patients with blood cancers and serious blood diseases. We harness our cell expansion platform to create therapies with the potential to redefine standards of care in areas of serious medical need. For additional information, please visit http://www.gamida-cell.com or follow Gamida Cell on LinkedIn or Twitter at @GamidaCellTx.

Cautionary Note Regarding Forward Looking Statements

This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including with respect to the anticipated timing of data disclosures and regulatory filing submissions, which statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to the ongoing global COVID-19 pandemic and clinical, scientific, regulatory and technical developments. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section and other sections of Gamida Cells Annual Report on Form 20-F, filed with the Securities and Exchange Commission (SEC) on February 26, 2020, and other filings that Gamida Cell makes with the SEC from time to time (which are available at http://www.sec.gov), the events and circumstances discussed in such forward-looking statements may not occur, and Gamida Cells actual results could differ materially and adversely from those anticipated or implied thereby. Any forward-looking statements speak only as of the date of this press release and are based on information available to Gamida Cell as of the date of this release.

References

1Horwitz M.E., Wease S., Blackwell B., Valcarcel D. et al. Phase I/II study of stem-cell transplantation using a single cord blood unit expanded ex vivo with nicotinamide. J Clin Oncol. 2019 Feb 10;37(5):367-374. 2Gamida Cell press release, Gamida Cell Announces Positive Topline Data from Phase 3 Clinical Study of Omidubicel in Patients with High-Risk Hematologic Malignancies, issued May 12, 2020. Last accessed August 31, 2020.

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Gamida Cell Announces Two-Part Virtual Miniseries Focused on Omidubicel to Take Place in September - Business Wire

COVID-19 Impacts: Autologous Cell Therapy Market Will Accelerate at a CAGR of Over 22% Through 2020-2024 | Use of Biomass as a Fuel in Boiler to Boost…

LONDON--(BUSINESS WIRE)--Technavio has been monitoring the autologous cell therapy market and it is poised to grow by USD 1.97 billion during 2020-2024, progressing at a CAGR of 22% during the forecast period. The report offers an up-to-date analysis regarding the current market scenario, latest trends and drivers, and the overall market environment.

Technavio suggests three forecast scenarios (optimistic, probable, and pessimistic) considering the impact of COVID-19. Please Request Free Sample Report on COVID-19 Impact

Frequently Asked Questions-

The market is fragmented, and the degree of fragmentation will accelerate during the forecast period. Bayer AG, Brainstorm Cell Therapeutics Inc., Daiichi Sankyo Co. Ltd., FUJIFILM Holdings Corp., Holostem Terapie Avanzate Srl, Osiris Therapeutics Inc., Takeda Pharmaceutical Co. Ltd., Teva Pharmaceutical Industries Ltd., Sumitomo Chemical Co. Ltd., and Vericel Corp. are some of the major market participants. The use of biomass as a fuel in boiler will offer immense growth opportunities. To make the most of the opportunities, market vendors should focus more on the growth prospects in the fast-growing segments, while maintaining their positions in the slow-growing segments.

Use of biomass as a fuel in boiler has been instrumental in driving the growth of the market.

Autologous Cell Therapy Market 2020-2024: Segmentation

Autologous Cell Therapy Market is segmented as below:

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Autologous Cell Therapy Market 2020-2024: Scope

Technavio presents a detailed picture of the market by the way of study, synthesis, and summation of data from multiple sources. Our autologous cell therapy market report covers the following areas:

This study identifies increasing demand for effective drugs for cardiac and degenerative disorders as one of the prime reasons driving the autologous cell therapy market growth during the next few years.

Autologous Cell Therapy Market 2020-2024: Vendor Analysis

We provide a detailed analysis of vendors operating in the autologous cell therapy market, including some of the vendors such as Bayer AG, Brainstorm Cell Therapeutics Inc., Daiichi Sankyo Co. Ltd., FUJIFILM Holdings Corp., Holostem Terapie Avanzate Srl, Osiris Therapeutics Inc., Takeda Pharmaceutical Co. Ltd., Teva Pharmaceutical Industries Ltd., Sumitomo Chemical Co. Ltd., and Vericel Corp. Backed with competitive intelligence and benchmarking, our research reports on the autologous cell therapy market are designed to provide entry support, customer profile and M&As as well as go-to-market strategy support.

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Autologous Cell Therapy Market 2020-2024: Key Highlights

Table of Contents:

Executive Summary

Market Landscape

Market Sizing

Five Forces Analysis

Market Segmentation by Therapy

Customer landscape

Geographic Landscape

Drivers, Challenges, and Trends

Vendor Landscape

Vendors covered

Appendix

About Us

Technavio is a leading global technology research and advisory company. Their research and analysis focuses on emerging market trends and provides actionable insights to help businesses identify market opportunities and develop effective strategies to optimize their market positions. With over 500 specialized analysts, Technavios report library consists of more than 17,000 reports and counting, covering 800 technologies, spanning across 50 countries. Their client base consists of enterprises of all sizes, including more than 100 Fortune 500 companies. This growing client base relies on Technavios comprehensive coverage, extensive research, and actionable market insights to identify opportunities in existing and potential markets and assess their competitive positions within changing market scenarios.

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COVID-19 Impacts: Autologous Cell Therapy Market Will Accelerate at a CAGR of Over 22% Through 2020-2024 | Use of Biomass as a Fuel in Boiler to Boost...