Targeted News Service Published 8:39 p.m. ET Oct. 2, 2020

WASHINGTON - Here's a look at how area members of Congress voted Sept. 25 to Oct. 1.

Along with its roll call votes, the House also passed these measures: the Cyber Sense Act (H.R. 360), to require the secretary of energy to establish a voluntary Cyber Sense program to test the cybersecurity of products and technologies intended for use in the bulk-power system; the Consumer Product Safety Inspection Enhancement Act (H.R. 8134), to support the Consumer Product Safety Commission's capability to protect consumers from unsafe consumer products; the School-Based Allergies and Asthma Management Program Act (H.R. 2468), to increase the preference given, in awarding certain allergies and asthma-related grants, to states that require certain public schools to have allergies and asthma management programs; and the Effective Suicide Screening and Assessment in the Emergency Department Act (H.R. 4861), to establish a program to improve the identification, assessment, and treatment of patients in the emergency department who are at risk of suicide.

House Vote 1:

PRESIDENTIAL ELECTION: The House has passed a resolution (H. Res. 1155), sponsored by Rep. Eric Swalwell, D-Calif., reaffirming the House's commitment to an orderly and peaceful transfer of presidential power after the November election. Swalwell said: "The peaceful transition of power is not only a bedrock principle of America's founding; it is a living ideal that we must exercise and pass down to our children." An opponent, Rep. Matt Gaetz, R-Fla., called the resolution "a way for Democrats to attack the president and disguise the fact that they will refuse to accept the election results unless they win." The vote, on Sept. 29, was 397 yeas to 5 nays.

YEAS: Bob Gibbs R-OH (7th), Troy Balderson R-OH (12th)

NOT VOTING: Jim Jordan R-OH (4th)

House Vote 2:

DISCLOSING TIES TO UYGHUR LABOR: The House has passed the Uyghur Forced Labor Disclosure Act (H.R. 6270), sponsored by Rep. Jennifer Wexton, D-Va., to require publicly traded companies to disclose whether they have business ties to China's Uyghur Autonomous Region in Xinjiang province. Wexton said the requirement would let investors know of a given company's "passive complicity or active exploitation of one of the most pressing and ongoing human rights violations of our lifetime." A bill opponent, Rep. Anthony Gonzalez, R-Ohio, said it wrongly tried to have the Securities and Exchange Commission police human rights violations, a role that would be better handled by the Treasury Department. The vote, on Sept. 30, was 253 yeas to 163 nays.

NAYS: Gibbs R-OH (7th), Balderson R-OH (12th),Jordan R-OH (4th)

House Vote 3:

DISEASE THERAPIES: The House has passed the Timely ReAuthorization of Necessary Stem-cell Programs Lends Access to Needed Therapies Act (H.R. 4764), sponsored by Rep. Doris O. Matsui, D-Calif. The bill would reauthorize a program for transplanting umbilical cord blood, stem cellsand bone marrow to adults and children suffering from various diseases. The vote, on Sept. 30, was unanimous with 414 yeas.

YEAS: Gibbs R-OH (7th), Balderson R-OH (12th),Jordan R-OH (4th)

House Vote 4:

FURTHER COVID-19 SPENDING: The House has approved an amendment to the America's Conservation Enhancement Act (H.R. 925). The amendment would spend $2.2 trillion on new COVID-19 measures, including testing and treatment efforts and unemployment benefits. A supporter, Rep. James P. McGovern, D-Mass., said the spending was needed "for families to pay for necessities like food, utilitiesand rent during this pandemic." An opponent, Rep. Tom Cole, R-Okla., said the amendment had been hurriedly brought to the floor without minority input or adequate time for review, and that it would not pass the Senate. The vote, on Oct. 1, was 214 yeas to 207 nays.

NAYS: Gibbs R-OH (7th), Balderson R-OH (12th),Jordan R-OH (4th)

Senate Vote 1:

CONTINUING APPROPRIATIONS: The Senate has passed the Continuing Appropriations Act and Other Extensions Act (H.R. 8337), sponsored by Rep. Nita M. Lowey, D-N.Y., to extend through Dec.11 funding for health programs, including Medicare, surface transportationand many other government programs. The vote, on Sept. 30, was 84 yeas to 10 nays.

YEAS: Sherrod Brown D-OH, Rob Portman R-OH

Senate Vote 2:

OBAMACARE LITIGATION: The Senate has rejected a cloture motion to end debate on a motion to consider a bill (S. 4653), sponsored by Senate Minority Leader Chuck Schumer, D-N.Y., that would block the Justice Department from making arguments in court for cancelling any provision of the 2010 health care reform law (Obamacare). The vote to end debate, on Oct. 1, was 51 yeas to 43 nays, with a three-fifths majority needed for approval.

YEAS: Brown D-OH

NAYS: Portman R-OH

Read or Share this story: https://www.mansfieldnewsjournal.com/story/news/2020/10/02/heres-how-area-members-congress-voted-sept-25-oct-1/3593470001/

Excerpt from:
Here's how area members of Congress voted - Mansfield News Journal

A baby whose nose bleed turned out to be a one-in-a-million disease has had his life saved by a complete stranger.

Tiny Daniel McAvoy's parents were told he would die unless he received the stem cell transplant before he turned two.

The infant was diagnosed with Wiskott-Aldrich syndrome, a rare blood disorder, when he was just ten weeks old.

Mum Georgie, 30, and dad Andrew, 37, noticed something wasnt right with their newborn when he suffered chronic nosebleeds and passed blood in his nappies.

But after desperately searching for the right donor for more than a year, Daniel has now had the transplant and is a smiley, happy tot back at home.

"Hes eating and playing and I really hope it continues that way. Im just so proud of him, hes done amazing," Georgie, of Benwick, Cambridgeshire, said today.

"There were plenty of times we thought he would die - we even had him christened in hospital because things werent looking great.

"He has the most severe level of the syndrome so from day one we were told he would need a transplant to replace his faulty immune system and defective cells and the procedure took place.

Doctors warned that he would go down quickly after the transplant but he hasnt and they're shocked."

When Daniel was first referred to hospital at three weeks old, medics first thought he had leukaemia, but after recovering from sepsis, he was discharged while they continued to look into the worrying case.

He was then rushed back to hospital at ten weeks old in July last year.

Georgie, who is a stay-at-home-mum, said: The nosebleeds went from nothing to doctors then telling us that he needed a bone marrow transplant to save his life.

He was diagnosed with Wiskott-Aldrich Syndrome in July 2019, which affects only one in a million boys and was the cause of the horrendous bleeds.

I never knew I was a carrier of the gene that causes it, so as a mum I felt pretty guilty that Id given him it."

The newborn woke up in May 2019 with a crust of blood on his nostril, which his parents thought was strange but a health visitor suggested it could be from a scratch.

But the tot began passing blood in vomit and nappies, and a GP then referred him to the hospital, where tests found he had a low platelet count that caused the bleeding.

Following a platelet transfusion to help stabilise him, Daniels parents were told he contracted sepsis but could also have meningitis and a rare type of leukaemia - so he was sent to a cancer ward.

Various procedures ruled out leukemia and out of nowhere the tiny tot began to get well again, so doctors discharged him.

Georgie added: Something still didnt sit right with me but some time later he had an unrelated hernia which needed an operation, and began having nose bleeds again.

I suggested that a doctor needed to look at the bigger picture because I knew there was something wrong with my baby.

The paediatrician looked back at all of Daniels notes and thought it was Wiskott-Aldrich Syndrome, which Id never heard of before, then after the operation to remove the hernia the haematologist agreed.

Within 24 hours of him being tested for this he was pretty much diagnosed and then doctors were speaking to us about bone marrow transplants - it all went very quickly.

In Daniels case, hes had a lot of bleeding problems - he bleeds from his gut, nose, eyes and mouth - so doctors were concerned about a catastrophic bleed that would kill him.

Wiskott-Aldrich Syndrome is a primary immunodeficiency which caused Daniels bleeding and left him prone to infections such as sepsis and also cancer.

He began having antibody infusions once a week as charities such as DKMS ran their global register to find a life-saving stem cell donor for Daniel, and a match was found in September 2019.

Georgie said: We had a terrifying 12 week wait but we were really fortunate that it was quick before a match was found.

His transplant then got delayed three times - the first and second times were because he wasnt well enough, and the third time was due to coronavirus.

I just went into panic mode because I thought he was going to die from this.

But Daniels transplant finally took place after a week-long chemo and immunotherapy course.

Were taking it day by day - if its successful, within two years he should be a healthy little boy.

Its all been a mixture of feelings - its really nice how hes so young that he wont remember how awful this all is but at the same time, sitting there watching your child have chemotherapy is the most heartbreaking thing in the world.

I would do anything to switch places with him.

Now that baby Daniel has been given another shot at life, his family are now calling out for others to join the transplant registers, as four out of ten patients arent lucky enough to get a match.

Georgie added: Its incredible how an anonymous donor saved Daniels life - without that person there would be no Daniel, they are all we needed to give him a second chance at life.

Its so simple and easy to sign up, and by doing so, you could save a babys life.

If you are aged between 17 and 55 and in general good health take the first step to register as a blood stem cell donor by registering for your home swab kit at http://www.dkms.org.uk.

Read more:
Baby whose nosebleeds turned out to be deadly condition saved by stranger - Mirror Online

NEW YORK--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces two presentations at the European Society for Immunodeficiencies (ESID) 2020 Meeting to be held virtually October 14-17, 2020. An oral presentation will provide an update on data from the Phase 1/2 clinical trial of RP-L201 for Leukocyte Adhesion Deficiency-I (LAD-I). An e-poster will highlight preclinical study data on RP-L401 for Infantile Malignant Osteopetrosis (IMO).

Additional presentation details can be found below:

Oral Presentation

Title: A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Results from Phase 1 Session Title: TreatmentPresenter: Donald B. Kohn, M.D., Professor of Microbiology, Immunology and Molecular Genetics, Pediatrics (Hematology/Oncology), Molecular and Medical Pharmacology, and member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at the University of California, Los AngelesSession Date: Friday, October 16, 2020Session Time: 10:45 a.m. 12:01 p.m. CESTLecture Time: 11:45 a.m. CESTLocation: Hall D

This session will be followed by a Q&A from 12:01 p.m. to 12:30 p.m. CEST

E-Poster

Title: Preclinical Efficacy and Safety of EFS.HTCIRG1-LV Supports IMO Gene Therapy Clinical Trial InitiationPresenter: Ilana Moscatelli, Ph.D., Associate Researcher, Division of Molecular Medicine and Gene Therapy, Lund University, Sweden

About Leukocyte Adhesion Deficiency-I

Severe Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18. CD18 is a key protein that facilitates leukocyte adhesion and extravasation from blood vessels to combat infections. As a result, children with severe LAD-I (less than 2% normal expression) are often affected immediately after birth. During infancy, they suffer from recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics and require frequent hospitalizations. Children who survive infancy experience recurrent severe infections including pneumonia, gingival ulcers, necrotic skin ulcers, and septicemia. Without a successful bone marrow transplant, mortality in patients with severe LAD-I is 60-75% prior to the age of 2 and survival beyond the age of 5 is uncommon. There is a high unmet medical need for patients with severe LAD-I.

Rockets LAD-I research is made possible by a grant from the California Institute for Regenerative Medicine (Grant Number CLIN2-11480). The contents of this press release are solely the responsibility of Rocket and do not necessarily represent the official views of CIRM or any other Agency of the State of California.

About Infantile Malignant Osteopetrosis

Infantile Malignant Osteopetrosis (IMO) is a rare, severe autosomal recessive disorder caused by mutations in the TCIRG1 gene, which is critical for the process of bone resorption. Mutations in TCIRG1 interfere with the function of osteoclasts, cells which are essential for normal bone remodeling and growth, leading to skeletal malformations, including fractures and cranial deformities which cause neurologic abnormalities including vision and hearing loss. Patients often have endocrine abnormalities and progressive, frequently fatal bone marrow failure. As a result, death is common within the first decade of life. IMO has an estimated incidence of 1 in 200,000. The only treatment option currently available for IMO is an allogenic bone marrow transplant (HSCT), which allows for the restoration of bone resorption by donor-derived osteoclasts which originate from hematopoietic cells. Long-term survival rates are lower in IMO than those associated with HSCT for many other non-malignant hematologic disorders; severe HSCT-related complications are frequent. There is an urgent need for additional treatment options.

RP-L401 was in-licensed from Lund University and Medizinische Hochschule Hannover.

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket) is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. The companys platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia and Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. For more information about Rocket, please visit http://www.rocketpharma.com.

Rocket Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding its guidance for 2020 in light of COVID-19, the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rockets ongoing trials, our expectations regarding when clinical trial sites will resume normal business operations, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Annual Report on Form 10-Q for the quarter ended June 30, 2020, filed August 5, 2020 with the SEC. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

Go here to see the original:
Rocket Pharmaceuticals Announces Two Presentations at the European Society for Immunodeficiencies 2020 Meeting - Business Wire

Smiths 2005 stem cell law to be reauthorized by House

Rep. Chris Smith (R-NJ)statement submitted during debate in the House of Representatives

on the Stem Cell Therapeutic and Research ActSeptember 29, 2020

Margaret Hahnmy mother-in-lawpassed away on Friday and a Mass of Christian burial will be held today at St. Mary Church in South Amboy, New Jersey. She was 96 and was deeply loved and will be deeply missed.

MargaretPegwas a great womanwife, mother, grandmother, and great-grandmother. She selflessly devoted her life to public service including her amazing work as Sayreville Borough Clerk for twenty years. She had an incredible reputation for getting things done for the people. No matter who served as mayor or on Council, everyone knew she was the power.

My wife Marie and I will join family and friends today at her funeral and internment making it impossible for me to speak today during the debate on the reauthorization of a law I originally authored fifteen years agothe Stem Cell Therapeutic and Research Act of 2005and the Stem Cell Therapeutic and Research Act of 2015.

So, I submit these comments for the Congressional Record.

Madam Speaker, today the House of Representatives will vote to reauthorize the Stem Cell Therapeutic and Research Act.

This was an original idea of mine 20 years ago. Joined by 70 cosponsors, I introduced it in 2001 and again in 2003.

After five long years of hard work and numerous setbacks, my bill was finally enacted into law in 2005.

Beginning in 2001, Dr. Joanne Kurtzberg, who is President of the Cord Blood Association, helped draft my original law.Dr. Kurtzberg has said, Cord blood transplantation is now an established field with enormous potential. In the future, it may emerge as a source of cells for cellular therapies focused on tissue repair and regeneration.

The new law created a nationwide umbilical cord blood stem cell program, designed to collect, derive, type, and freeze cord blood units for transplantation into patients to mitigate and to even cure serious disease. Pursuant to the law, it also provided stem cells for research. The new cord blood program was combined in our 2005 law with an expanded bone marrow initiative, which was crafted over several years by our distinguished colleague, CongressmanBill Young.

I was the prime sponsor again when it was reauthorized in 2015.

Umbilical cord blood stem cells, obtained after the birth of a child, have proved highly efficacious in treating 70 diseases, including sickle-cell disease, lymphoma, and leukemia. And scientists are continuing to study and better understand the regenerative effects of cord blood cell therapies for other diseases and conditions. Bone marrow donations provide lifesaving transplants to treat diseases like blood cancer, sickle cell anemia, or inherited metabolic or immune system disorders.

The National Cord Blood Inventory (NCBI) provides funding to public cord blood banks participating in the program to allow them to expand the national inventory of cord blood units available for transplant. These units are then listed on the registry by the Be the Match Program. The funds appropriated thus far have led to an important increase in the overall number of high-quality cord blood units available through the national registry, including 150,000 NCBI units. Within the Be the Match registry, there are more than 783,000 NCBI units worldwide.

The Program registry allows patients and physicians to locate matching cord blood units, as well as adult donors for marrow and peripheral blood stem cells, when a family donor is not available. The Program is the worlds largest, most diverse donor registry, with more than 22 million volunteers and more than 300,000 public cord blood units. To date, the National Marrow Donor Program/Be The Match (NMDP), through its operation of the Program, has facilitated more than 100,000 transplants. More than 45,000 patients have receivedcord bloodtransplants, according Dr. Joanne Kurtzberg.

The reauthorization before us authorizes $23 million to be appropriated for fiscal year 2021 through fiscal year 2025. It also authorizes $30 million to be appropriated for fiscal years 2021 through 2025 for the bone marrow transplant program. This continues funding at the same levels authorized in the 2015 authorization bill.

Madam Speaker, each year nearly 4 million babies are born in America. In the past, virtually every placenta and umbilical cord was tossed as medical waste. Today, doctors have turned this medical waste into medical miracles.

Not only has God in His wisdom and goodness created a placenta and umbilical cord to nurture and protect the precious life of an unborn child, but now we know that another gift awaits us immediately after birth. Something very special is left behindcord blood that is teeming with lifesaving stem cells. Indeed, it remains one of the best kept secrets in America that umbilical cord blood stem cells and adult stem cells in general are curing people of a myriad of terrible conditions and diseasesover 70 diseases in adults as well as in children.

The legislation that is before us will enable even more patients to receive the treatments that they so desperately need.

(Visited 76 times, 76 visits today)

Follow this link:
Smith's 2005 stem cell law to be reauthorized by House - InsiderNJ

Next, to accommodate the donors immune cells, they had to wipe out Mr. Browns own immune system by bombarding him with chemotherapy and radiation. Next came the transplant procedure itself. On that same February day, Mr. Brown stopped taking his antiretroviral medication. Three months later, after a grueling recovery in which he almost died, he was H.I.V.-free.

For Mr. Brown, the epiphany came one day in the gym, when he found that he was developing muscles again after years of wasting away. That was kind of my proof that it was gone, he said.

Many hurdles remained. A recurrence of leukemia required a second transplant a year later. A brain biopsy left Mr. Brown temporarily paralyzed and nearly blind. He had to be taught how to walk and talk again. His recovery, complicated by injuries from a 2009 mugging in Berlin, left him with a stiff shoulder, limited vision and neurological damage, which prevented him from resuming his work as a translator.

My life is far from perfect, he said in 2015, but it is still my life.

He was living in Nevada in 2013 when he met Mr. Hoeffgen on the Scruff dating app. They moved to Southern California in 2015. In April, Mr. Brown was admitted to a cancer hospital; his leukemia, unrelated to H.I.V., had returned. Covid-19 restrictions kept the couple together on the medical campus for weeks.

This month, Mr. Hoeffgen told Mark S. King, a blogger and AIDS activist, that Mr. Brown had terminal cancer and had been receiving home hospice care. Mr. Brown was aware that he was dying.

I have asked him what he wants me to tell people when we make his situation public, Mr. Hoeffgen said. He said: Tell people to keep fighting. Fight for a cure for H.I.V. that works for everyone. I never wanted to be the only one.

In addition to Mr. Hoeffgen, Mr. Brown is survived by his mother.

One researcher asked whether the couple would consider donating Mr. Browns body to science.

I said, Thank you, but no, Mr. Hoeffgen said. I think hes done enough.

Read the original:
Timothy Ray Brown, First Patient Cured of H.I.V., Dies at 54 - The New York Times

Newswise In a world first, a young man suffering from severe aplastic anemia who could not be helped by standard treatments has been given a life-saving blood transplant with the made-in-Canada UM171 molecule.

The procedure was done by a medical team at the Institute of Hemato-oncology and Cellular Therapy (iHOTC) of Maisonneuve-Rosemont Hospital and the Institute for Research in Immunology and Cancer, both affiliated with Universit de Montral.

The young mans case history, including the lifesaving transplant, was recently published in the scientific journal European Journal of Haematology, highlighting the unique and revolutionary properties of the UM171 molecule.

An autoimmune disease, severe aplastic anemia destroys stem cells in bone marrow and leads to a halt in the production of red blood cells, white blood cells and platelets. For allografting (grafting between individuals) for this disease, the donor's stem cells must be as compatible as possible with those of the recipient to avoid the risk of immunological complications.

No donor option

If no compatible family or unrelated donor can be found, stem cells from a semi-identical family donor, also known as a haplo-identical donor, may be considered, under certain conditions, as an alternative source of cells. However, a family member must be healthy and available for such a procedure; the young man in this case did not have that option.

Cord blood transplantation, which is less demanding in terms of compatibility, is a good option for many patients requiring a stem cell transplant. On the other hand, cord blood generally does not contain enough stem cells for an adult patient weighing more than 70 kg; it produces a slow rise in white blood cells with an increased risk of often fatal infections.

In addition, the rate of graft rejection the destruction of infused cord cells by the recipient's immune system is very high in patients with severe aplastic anemia who have received multiple blood transfusions.

It was after having exhausted all our treatment options that UM171, which had already proven itself in a clinical trial in blood cancer patients, came into play, said UdeM medical professor Jean Roy, a hematologist and clinical researcher at the MRH.

35-fold increase

As well as increasing the number of stem cells in a unit of umbilical cord blood by an average of 35 times, it greatly reduces the risk of a frequent long-term immunological complication (graft-versus-host disease) requiring years of use of toxic immunosuppressive drugs.

The researchers success confirms the excellent performance of UM171, which has already been demonstrated in two other studies conducted by iHOTC research teams with very encouraging results. A third study is now underway.

This young man's story and the other studies with UM171 clearly demonstrate how innovative clinical research, set up by local investigators, can create a culture of excellence and improve care to save more lives, said IHOTC director Denis Claude Roy.

The future will certainly bring us more such accomplishments, and thats very encouraging.

Maisonneuve-Rosemont Hospital is one of 26 institutions that are part of the Centre intgr universitaire de sant et de services sociaux de l'Est-de-l'le-de-Montral, serving over half a million Montrealers.

Read the original post:
UM171 saves another life - Newswise

When 5-year-old Sawyer was diagnosed with sickle cell at a young age, his fraternal twin, Saxton, turned out to be a perfect match.

ATLANTA This is not just a birthday party, its a celebration of second chances.

We are so blessed, said OShea Guillory. We want to share this.

It is also a commitment to help others, too.

Mikari Tarpley is 16. She is at the small birthday celebration and sang her heart out for 5-year-old Sawyer and his brother Saxton. She sings from a place of understanding.

She and Sawyer, her neighbor, have both been through a lot this year.

We found out about Sawyers sickle cell disease at a very early age, Guillory said. He was about three weeks old.

Sawyers mom said she was absolutely devastated. She refused to accept there was nothing she could do to help her son.

I did a ton of research," she said. I found an organization called Be The Match.

She discovered information about transplants that could be a potential cure. Doctors take stem cells from the bone marrow of a donor and transplant it into the recipient.

They just needed a donor.

It turns out Sawyer was born with his cure, Guillory said. His twin, Saxton, was a perfect sibling match.

Across the cul-de-sac, Mikari Tarpley, an actress, was fighting Hodgkins Lymphoma and finishing chemotherapy.

She knew Sawyer has been battling sickle cell. Theyd both lost their hair from treatment. She decided she wanted to use her Sweet 16th birthday to raise money for children like Sawyer.

We couldnt do much for my sixteenth because of COVID, so we thought it would be a great idea to do a fundraiser to help others, Mikari said.

She reached her goal of $16,000 to help children being treated for sickle cell disease at the Aflac Cancer and Blood Disorders Center.

Five-year-old Sawyer received a bone marrow transplant from his fraternal twin, Saxton. It was a success. Before long, Sawyer was riding a tricycle around Childrens Healthcare of Atlanta, racing his nurses and giggling along the way.

I truly have little warriors, Guillory said. My son, who helped save his brothers life - and my son - who was able to go through all of that and come out even stronger.

Now Sawyer, Saxton and their parents are spending their 5th birthday following Mikaris lead by paying it forward.

Sawyers mom OShea is starting a nonprofit, Sawyers Sickle Circle, to spread awareness and knowledge about sickle cell, Beads of Courage and Be the Match program. They launched a fundraiser on Sawyer and Saxtons birthday.

The Guillory family said the best present they could wish for is for other kids to have the chance to celebrate many more birthdays to come.

Continued here:
A perfect match: Bone marrow transplant saves twin brother's life. Now their family pays it forward - 11Alive.com WXIA

By Claudia Paba Prada, MD

For more than 30 years, oncologists have used immunotherapy to treat cancer, harnessing the strength of the patients own immune system to fight the disease. For many, it has been a welcome alternative or supplement to more traditional chemotherapy, radiation, and surgical options.

The disease I specialize in, multiple myeloma, has no cure, but patients can maintain quality of life with treatment combinations. These individuals have cancer cells form in plasma cells within bone marrow, crowding out healthy (red and white) blood cells and damaging bones, the immune system, and kidneys. We use immunotherapy in combination with chemotherapy to treat cancerous plasma cells, transitioning to different drugs when the cancer mutates and becomes resistant to the previously prescribed treatment. Our goal is to get patients to a stem cell transplant or, if they arent an appropriate candidate, to utilize a combination of drugs to kill myeloma cells. We then continue maintenance therapy to keep cancer cells dormant and preserve their existing lifestyle.

Its critical that we never stop studying the biology of the diseases we see, since each patient is different and there is no one-size-fits-all treatment. What approaches have been used previously, their toxicity, and the patients comorbidities (diabetes, heart issues, etc.) all factor into what may or may not be the appropriate next step. With younger, newly diagnosed patients, a more aggressive approach to get to transplantation may be pursued. In older patients, the goal is usually to get to some level of remission, even if that isnt a permanent solution.

Some myeloma patients, however, dont respond to any of the available chemotherapy drugs or may have a cancer relapse after their transplant. Thats why there is excitement within the cancer community about clinical trials were participating in at the

Moffitt Malignant Hematology and Cellular Therapy Program at Memorial Hospital West that have increased what were able to accomplish through immunotherapy.

The new approach is called CAR-T cell therapy and its administered like a blood transfusion after the patients own T cells are reprogrammed to attack the cancer cells. This is done by genetically altering T cells so they produce synthetic molecules called chimeric antigen receptors, or CARs, which enable T cells to recognize and attach to a certain protein in tumor cells and kill them.

We see 70-80 new multiple myeloma cases each year and more than 300 with relapse disease so, while not every patient will be a CAR-T candidate, were hoping many more will be as the trial progresses. Were using drugs under research that are unavailable anywhere else in Florida for myeloma and expect to expand to include leukemia and lymphoma patients in the coming year.

All this work is being done as we establish a myeloma-specific institute at Moffitt/Memorial that will be the only one of its type in Broward and Palm Beach counties. Bringing specialists together and providing South Floridians access to clinical trials is part of what were planning, but its also important to address the whole person and not just the disease. Thats why were already collaborating with the Leukemia and Lymphoma Society of Broward County and have established a support group for myeloma patients and their caregivers. The group will address issues related to a cancer diagnosis and provide opportunities for attendees to discuss concerns, anxieties, feelings related to their illness, treatment, and connected issues. Meetings, even the virtual ones were having during COVID-19, are designed to offer mutual support and information to members by connecting them to others whose situations are similar to their own.

My own journey has taken me from my home country of Colombia to an internal medicine residency in Philadelphia, hematology/oncology fellowship in Memphis, and an advanced fellowship in hematologic malignancies at Dana Farber Cancer Institute in Boston. I was at Dana Farber for seven years before relocating to South Florida in 2017. I joined the Moffitt team at Memorial Hospital West in July and am anxious to further the research and treatment of multiple myeloma at one of the nations leading cancer centers. CAR-T cellular therapy is one of the ways we can get there together.

See original here:
New Weapons in the Battle Against Blood Cancers: Current Articles - South Florida Hospital News

LONDON (Reuters) - Timothy Ray Brown, the first person known to be cured of HIV when he had a unique type of bone marrow transplant, has died in California after relapsing with cancer, his partner said.

FILE PHOTO: Timothy Ray Brown, also known as "The Berlin Patient," smiles during a news conference held by the World AIDS Institute in Washington July 24, 2012. Brown is thought to be the first patient ever to be cured of HIV infection. REUTERS/Kevin Lamarque

It is with great sadness that I announce that Timothy passed away ... this afternoon surrounded by myself and friends, after a 5 month battle with leukaemia, his partner, Tim Hoeffgen, said in a post on Facebook. He said Brown was his hero and the sweetest person in the world.

Brown, born on March 11, 1966, became known as the Berlin Patient after his HIV was cleared by treatment there in 2007.

The Americans case fascinated and inspired a generation of HIV doctors as well as patients infected with the virus that causes AIDS, offering a glimmer of hope that one day a cure will be found that eventually ends the AIDS pandemic.

We owe Timothy and his doctor, Gero Huetter, a great deal of gratitude for opening the door for scientists to explore the concept that a cure for HIV is possible, said Adeeba Kamarulzaman, president of the International AIDS Society (IAS).

Brown was diagnosed with HIV in 1995 while living in the German capital, and in 2006 was also diagnosed with a type of blood cancer known as acute myeloid leukaemia.

While Brown remained clear of HIV for more than a decade after being treated, he had suffered a relapse of the leukaemia in the past year. His doctors said the blood cancer had spread to his spine and brain, and he had recently been in hospice care in his home town of Palm Springs, California.

Im heartbroken that my hero is now gone. Tim was truly the sweetest person in the world, Hoeffgen said in the Facebook post. He said Brown had made it his lifes work to tell the story of his HIV cure and became an ambassador of hope.

For Huetter, the German doctor caring for him in 2007, Browns case was a shot in the dark. The treatment involved the destruction of Browns immune system and the transplanting of stem cells with a gene mutation called CCR5 that resists HIV.

Only a tiny proportion of people most of them of northern European descent have the CCR5 mutation that makes them resistant to the AIDS-causing virus.

This and other factors made the treatment Brown had expensive, complex and highly risky. Most experts say it could never become a way to cure all HIV patients, since many of them would risk death from the procedure itself.

More than 37 million people worldwide are currently infected with HIV, and the AIDS pandemic has killed about 35 million people since it began in the 1980s.

Medical advances over the past three decades have led to the development of drug combinations known as antiretroviral therapies that can keep the virus in check, allowing many HIV positive people to live with the virus for years.

A second HIV patient, Adam Castillejo, who was known as the London patient until he revealed his identity this year, is also thought to be in remission from HIV after having a transplant in 2016 similar to the one Brown had.

Sharon Lewin, president-elect of the IAS and an HIV specialist at Australias Doherty Institute, said Brown was a champion and advocate for keeping an HIV cure on the agenda.

It is the hope of the scientific community that one day we can honour his legacy with a safe, cost-effective and widely accessible strategy to achieve HIV remission and cure.

Reporting by Kate Kelland, editing by Gareth Jones and Nick Macfie

Read the original post:
World's first patient cured of HIV dies after cancer returns - Reuters India

CLR 131, a phospholipid ether molecule, is showing promising disease control in an ongoing phase 2 study (CLOVER-1, NCT02952508) as treatment of patients with relapsed or refractory lymphoplasmacytic lymphoma (LPL) and Waldenstrm macroglobulinemia (WM), according to initial study results.

The findings were presented in a poster during the American Association for Cancer Research (AACR) Virtual Meeting: Advances in Malignant Lymphoma by Jarrod Longcor, chief business officer of Cellectar Biosciences, Inc, the developer of CLR 131.

Prior to the initiation of the phase 2 study, the potential of CLR 131 was demonstrated throughin vitro, in vivo, and preclinical studies. The agent works by binding to and entering the tumor via lipid rafts, which stabilize tumor cells. This mechanism of action is particularly effective in hematologic malignancies.

Read earlier data on CLR 131 here.

Four patients with LPL/WM were enrolled to assess interim efficacy and safety in part A of the trial. The median age of the population was 70 years (range, 54-81). Three of the patients had an ECOG performance status of 0. The mean prior number of therapies received was 2.5 with a median of 2 (range, 1-5). All patients had received prior rituximab (Rituxan); ibrutinib (Imbruvica) and autologous stem cell transplant were received by 1 patient each. In addition, 75% of patients received other agents.

Efficacy in the Overall Population

The objective response rate (ORR) observed with CLR 131 in the LPL/WM population treated in part A of CLOVER-1 was 100% with a major response to treatment observed in 3 patients after 2 to 4 doses of the drug. Despite the characteristics of these heavily pretreated patients, CLR 131 is the only monotherapy that has achieved responses in the relapsed or refractory population.

In the overall population of 4 patients, survival and duration of response (DOR) were also assessed. Two patients in the study received 1 cycle of CLR 131 (2 doses total) and the other 2 received 2 cycles (4 doses total).

Of the 2 patients receiving 1 cycles of therapy, patient 1 had an overall survival (OS) duration 16.4 month, a progression-free survival (PFS) of 16.4 months, and a DOR of 15 months. The same patient had a major response to therapy. The second patients had an OS and PFS of 13.8 months and a DOR of 13.1 months. The response observed in this patient was a partial response (PR).

Of the 2 patients receiving 2 cycles of therapy, patient 3 had the longest OS and PFS at 33.2 with a DOR of 31.7 months and a complete response to treatment. Finally, patient 4 had an OS and PFS of 10.1 months with a DOR of 8.4 months. The type of response shown with patient 4 was a PR.

In all patients, the median DOR was not reached and the ongoing mean was calculated as 17.1 months. Evaluation of all patient responses is ongoing.

Longer-Term Response in Elderly Patient

One patient in the study was a 66-year-old female who, at baseline, presented with pleural effusion and 5 large extra-medullary nodules in the third-line setting. She was refractory to all of the prior treatment she received. CLR 131 in this patient, however, led to 100% overall tumor burden reduction as well as complete resolution of all tumors. The tumor locations resolved included a subdiaphragmatic mass, left epicardial mass, aortic bifurcation, right ovary, and left ovary. This result occurred by day 187. In terms of the duration of complete response, CT and bone marrow biopsy performed at day 406 revealed the patient had a duration of response of over 31 months.

Safety in the Overall Population

The safety analysis in all patients with non-Hodgkin lymphoma treated on the trial (n = 19) revealed treatment-emergent adverse events (TEAEs) that occurred in 15% or more included thrombocytopenia (83%), lymphocyte count decreased (25%), decreased white blood cell count (58%), anemia (58%), and neutropenia (50%). In the LPL population alone, the most common events observed were neutropenia (100%), thrombocytopenia (100%), and fatigue (75%). The analysis demonstrated that patients with extramedullary disease have lower rates of cytopenias at lower grades. The drug was overall well-tolerated in patients.

CLOVER-1 is an open-label, multicenter, 2-part study. In part A, which is now completed, patients with multiple myeloma, diffuse large B-cell lymphoma, LPL/WM, mantle cell lymphoma, chronic lymphocytic leukemia/small lymphocytic leukemia, and marginal zone lymphoma were assessed for interim CLR 131 efficacy. Patients in part A received a starting dose of less than 50 mCi total body dose of CLR131, following by 50 mCi then 75 mCi. Part B is actively enrolling patients with multiple myeloma and LPL/WM to assess the clinical benefit rate, ORR, time to response, time to progression, and OS. Patients in part B will receive either 1 or 2 cycles of CLR 131 100 mCi total body dose.

Read more about CLR 131 for the treatment of hematologic malignancies.

Reference

Ailwadhi S, Longcor, J Oliver K, and Grachev I. CLR 131 demonstrates 100% overall response rate in relapsed or refractory lymphoplasmacytic lymphoma (LPL)/Waldenstroms macroglobulinemia (WM): initial results from ongoing phase 2 trial, CLOVER-1 study. Presented at: American Association for Cancer Research Virtual Meeting: Advances in Malignant Lymphoma; Aug 1719, 2020. Abstract PO-25

Continued here:
Encouraging ORR Observed With CLR 131 as CLOVER-1 Heads To Pivotal Stage - Targeted Oncology

The global stem cell banking market was valued at $1,986 million in 2016, and is estimated to reach $6,956 million by 2023, registering a CAGR of 19.5% from 2017 to 2023. Stem cell banking is a process where the stem cell care isolated from different sources such as umbilical cord and bone marrow that is stored and preserved for future use. These cells can be cryo-frozen and stored for decades. Private and public banks are different types of banks available to store stem cells.

Top Companies Covered in this Report: Cord Blood Registry,ViaCord,Cryo-Cell, China Cord Blood Corporation, Cryo-Save, New York Cord Blood Program, CordVida, Americord, CryoHoldco, Vita34

Get sample copy of Report at:

https://www.premiummarketinsights.com/sample/AMR00013812

Increase in R&D activities in regards with applications of stem cells and increase in prevalence of fatal chronic diseases majorly drive the growth of the global stem cell banking market. Moreover, the large number of births occurring globally and growth in GDP & disposable income help increase the number of stem cell units stored, which would help fuel the market growth. However, legal and ethical issues related to stem cell collections and high processing & storage cost are projected to hamper the market growth. The initiative taken by organizations and companies to spread awareness in regards with the benefits of stem cells and untapped market in the developing regions help to open new avenues for the growth of stem cell banking market in the near future.

The global stem cell banking market is segmented based on cell type, bank type, service type, utilization, and region. Based on cell type, the market is classified into umbilical cord stem cells, adult stem cells, and embryonic stem cells. Depending on bank type, it is bifurcated into public and private. By service type, it is categorized into collection & transportation, processing, analysis, and storage. By utilization, it is classified into used and unused. Based on region, it is analyzed across North America, Europe, Asia-Pacific, and LAMEA.

Get Discount for This Report https://www.premiummarketinsights.com/discount/AMR00013812

Table Of Content

CHAPTER 1: INTRODUCTION

CHAPTER 2: EXECUTIVE SUMMARY

CHAPTER 3: MARKET OVERVIEW

CHAPTER 4: STEM CELL BANKING MARKET, BY CELL TYPE

CHAPTER 5: STEM CELL BANKING MARKET, BY BANK TYPE

CHAPTER 6: STEM CELL BANKING MARKET, BY SERVICE TYPE

CHAPTER 7: STEM CELL BANKING MARKET, BY UTILIZATION

CHAPTER 8: STEM CELL BANKING MARKET, BY REGION

CHAPTER 9: COMPANY PROFILES

Enquire about report at: https://www.premiummarketinsights.com/buy/AMR00013812

About Premium Market Insights:

Premiummarketinsights.com is a one stop shop of market research reports and solutions to various companies across the globe. We help our clients in their decision support system by helping them choose most relevant and cost effective research reports and solutions from various publishers. We provide best in class customer service and our customer support team is always available to help you on your research queries.

Contact Us:

Sameer Joshi

Call: +912067274191

Email: [emailprotected]

Pune

Read more:
Stem Cell Banking Market is forecast to reach $6,956 million by 2023 | ViaCord,Cryo-Cell, China Cord Blood Corporation, Cryo-Save - The Daily...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced that the European Commission (EC) has granted orphan drug designation for AVR-RD-02, the companys investigational gene therapy for the treatment of Gaucher disease. AVR-RD-02 consists of the patients own hematopoietic stem cells, genetically modified to express glucocerebrosidase (GCase), the enzyme that is deficient in Gaucher disease. AVROBIO recently dosed the first patient in the GuardOne Phase 1/2 clinical trial to evaluate the safety and efficacy of AVR-RD-02.

Like many lysosomal disorders, Gaucher disease can lead to debilitating complications throughout the body and brain. The standard of care does not address all these symptoms and may not be able to halt progression of the disease, said Geoff MacKay, AVROBIOs president and CEO. Our investigational gene therapy is designed to address the head-to-toe manifestations of Gaucher disease with a single dose. Were pleased to receive orphan drug designation, which recognizes the potential of our approach to transform the standard of care and, we hope, the quality of life for people living with this rare genetic disorder.

The EC grants orphan drug designation to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions that impact fewer than 5 in 10,000 patients in the European Union. Orphan drug designation gives companies certain benefits, including reduced regulatory fees, clinical protocol assistance, research grants and 10 years of market exclusivity following regulatory approval.

AVR-RD-02 has also received orphan drug designation from the U.S. Food and Drug Administration.

About Gaucher Disease

Gaucher disease is a rare, inherited lysosomal storage disorder characterized by the toxic accumulation of glucosylceramide (GlcCer) and glucosylsphingosine (GlcSph) in macrophages. Macrophages bloated with these fatty substances are called Gaucher cells which amass primarily in the spleen, liver and bone marrow. This results in a variety of potential symptoms, including grossly enlarged liver and spleen, bone issues, fatigue, low hemoglobin levels and platelet counts and an adjusted lifetime relative risk of developing Parkinson's disease that may be more than 20 times greater than the general population. Even on enzyme replacement therapy (ERT) the current standard of care people with Gaucher disease type 1 typically have a shortened life expectancy and may experience debilitating symptoms that significantly reduce their quality of life. An estimated 1 in 44,000 people are diagnosed with Gaucher disease.

About AVR-RD-02

AVR-RD-02 is an investigational lentiviral gene therapy designed to provide a durable therapeutic benefit for people living with Gaucher disease. The therapy starts with the patients own hematopoietic stem cells, which are genetically modified to express functional glucocerebrosidase (GCase). Functional GCase reduces levels of glucosylceramide and glucosylsphingosine, the accumulated substances which cause the symptoms of Gaucher disease. AVROBIO is currently evaluating AVR-RD-02 in GuardOne, a Phase 1/2 clinical trial.

About lentiviral gene therapy

Lentiviral vectors are differentiated from other delivery mechanisms because of their large cargo capacity and their ability to integrate the therapeutic gene directly into the patients chromosomes. This integration is designed to maintain the therapeutic genes presence as the patients cells divide, which potentially enables dosing of pediatric patients, whose cells divide rapidly as they grow. Because the therapeutic gene is integrated into the patients own stem cells, patients are not excluded from receiving the investigational therapy due to pre-existing antibodies to the vector.

About AVROBIO

Our vision is to bring personalized gene therapy to the world. We aim to halt or reverse disease throughout the body by driving durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle and bone. Our clinical-stage programs include Fabry disease, Gaucher disease and cystinosis and we also are advancing a preclinical program in Pompe disease. AVROBIO is powered by the plato gene therapy platform, our foundation designed to scale gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario. For additional information, visit avrobio.com, and follow us on Twitter and LinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words and phrases such as aims, anticipates, believes, could, designed to, estimates, expects, forecasts, goal, intends, may, plans, possible, potential, seeks, will, and variations of these words and phrases or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding our business strategy for and the potential therapeutic benefits of our prospective product candidates, including AVR-RD-02 for the treatment of Gaucher disease; the anticipated benefits of the European Commissions grant of orphan drug designation for AVR-RD-02; the design, commencement, enrollment and timing of ongoing or planned clinical trials and regulatory pathways; the timing of patient recruitment and enrollment activities, clinical trial results, and product approvals; the anticipated benefits of our gene therapy platform including the potential impact on our commercialization activities, timing and likelihood of success; the expected benefits and results of our implementation of the plato platform in our clinical trials and gene therapy programs; and the expected safety profile of our investigational gene therapies. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in pre-clinical or early-stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

Any forward-looking statements in this press release are based on AVROBIOs current expectations, estimates and projections about our industry as well as managements current beliefs and expectations of future events only as of today and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that any one or more of AVROBIOs product candidates will not be successfully developed or commercialized; the risk of cessation or delay of any ongoing or planned clinical trials of AVROBIO or our collaborators; the risk that AVROBIO may not successfully recruit or enroll a sufficient number of patients for our clinical trials; the risk that AVROBIO may not realize the intended benefits of our gene therapy platform, including the features of our plato platform; the risk that our product candidates or procedures in connection with the administration thereof will not have the safety or efficacy profile that we anticipate; the risk that prior results, such as signals of safety, activity or durability of effect, observed from pre-clinical or clinical trials, will not be replicated or will not continue in ongoing or future studies or trials involving AVROBIOs product candidates; the risk that we will be unable to obtain and maintain regulatory approval for our product candidates; the risk that the size and growth potential of the market for our product candidates will not materialize as expected; risks associated with our dependence on third-party suppliers and manufacturers; risks regarding the accuracy of our estimates of expenses and future revenue; risks relating to our capital requirements and needs for additional financing; risks relating to clinical trial and business interruptions resulting from the COVID-19 outbreak or similar public health crises, including that such interruptions may materially delay our development timeline and/or increase our development costs or that data collection efforts may be impaired or otherwise impacted by such crises; and risks relating to our ability to obtain and maintain intellectual property protection for our product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause AVROBIOs actual results to differ materially and adversely from those contained in the forward-looking statements, see the section entitled Risk Factors in AVROBIOs most recent Quarterly Report, as well as discussions of potential risks, uncertainties and other important factors in AVROBIOs subsequent filings with the Securities and Exchange Commission. AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

More:
AVROBIO Receives Orphan Drug Designation from the European Medicines Agency for AVR-RD-02, an Investigational Gene Therapy for Gaucher Disease -...

Hematopoietic stem cells are young or immature blood cells found to be living in bone marrow. These blood cells on mature in bone marrow and only a small number of these cells get to enter blood stream. These cells that enter blood stream are called as peripheral blood stems cells. Hematopoietic stem cells transplantation is replacement of absent, diseased or damaged hematopoietic stem cells due to chemotherapy or radiation, with healthy hematopoietic stem cells. Over last 30 years hematopoietic stem cells transplantation market seen rapid expansion and constant expansion with lifesaving technological advances. Hematopoietic stem cells transplantation is also known blood and marrow transplantation which brings about reestablishment of the patients immune and medullary function while treating varied range of about 70 hematological and non-hematological disorders. In general hematopoietic stem cells transplantation is used in treatment of hereditary, oncological, immunological and malignant and non-malignant hematological diseases.

There are two types of peripheral blood stem cell transplants mainly autologous and allogeneic transplantation. In autologous transplants patients own hematopoietic stem cells are harvested or removed before the high-dose treatment that might destroy the patients hematopoietic stem cells. While in allogeneic transplants stem cells are obtained from a tissue type of matched or mismatched donor. Hematopoietic stem cells are harvested from blood or bone marrow and is then frozen to use later. Depending upon the source of hematopoietic stem cells, worldwide there are three types of hematopoietic stem cells transplants namely bone marrow transplant (BMT), peripheral blood stem cell transplant and cord blood transplant. Major drivers in the hematopoietic stem cells transplantation market are establishment of strong and well developed network of hematopoietic stem cells transplantation organizations having global reach and presence has recognized NGO named Worldwide Network for Blood and Marrow Transplantation Group (WBMT) in official relation with World Health Organization (WHO) and rapid increase in number of transplants. Major restraints in hematopoietic stem cells transplantation market is high cost of transplantation and lack of funding for WBMT and other organizations such as regional, national and donor.

To remain ahead of your competitors, request for a sample[emailprotected]

https://www.persistencemarketresearch.com/samples/14563

The global market for Hematopoietic stem cells transplantation market is segmented on basis of transplant type, application, disease indication, end user and geography:

To receive extensive list of important regions, Request Methodology here @

https://www.persistencemarketresearch.com/methodology/14563

Based on transplantation type, hematopoietic stem cells transplantation market is segmented into allogeneic and autologous. Hematopoietic stem cells transplantation market is also segmented by application type into bone marrow transplant (BMT), peripheral blood stem cell transplant and cord blood transplant. The market for hematopoietic stem cells transplantation is majorly driven by bone marrow transplant (BMT) segment. Based on end user hematopoietic stem cells transplantation market is segmented into hospitals and specialty centers. Peripheral blood stem cell transplant type holds the largest market for hematopoietic stem cells transplantation. Hematopoietic stem cells transplantation market is further segmented by disease indication into three main categories i.e. lymphoproliferative disorders, leukemia, and non-malignant disorders. Segment lymphoproliferative disorder holds largest share amongst the three in Hematopoietic stem cells transplantation market. On the basis of regional presence, global hematopoietic stem cells transplantation market is segmented into five key regions viz. North America, Latin America, Europe, Asia Pacific, and Middle East & Africa. Europe leads the global hematopoietic stem cells transplantation market followed by U.S. due to easy technological applications, funding and high income populations. Other reasons for rise in hematopoietic stem cells transplantation market is high prevalence of lymphoproliferative disorders and leukemia; demand for better treatment options; and easy accessibility and acceptance of population to new technological advances. Transplantation rates in high income countries are increasing at a greater extent but continued rise is also seen in low income countries and expected to rise more. Hematopoietic stem cells transplantation market will have its potential in near future as being a perfect alternative to traditional system in many congenital and acquired hematopoietic disorders management. While India, China and Japan will be emerging as potential markets. An excellent and long term alternative to relief by side effects of chemotherapy, radiotherapy and immune-sensitive malignancies is another driver for hematopoietic stem cells transplantation market. The key players in global hematopoietic stem cells transplantation market are Lonza, Escape Therapeutics, Cesca Therapeutics Inc., Regen BioPharma, Inc., Invitrx Inc, StemGenex, Lion Biotechnologies, Inc., CellGenix GmbH, Actinium Pharmaceuticals, Inc., Pluristem, Kite Pharma, Novartis AG.You Can Request for TOC Here @https://www.persistencemarketresearch.com/toc/14563

Explore Extensive Coverage of PMR`s

Life Sciences & Transformational HealthLandscape

About us:

Persistence Market Research (PMR) is a third-platform research firm. Our research model is a unique collaboration of data analytics andmarket research methodologyto help businesses achieve optimal performance.

To support companies in overcoming complex business challenges, we follow a multi-disciplinary approach. At PMR, we unite various data streams from multi-dimensional sources. By deploying real-time data collection, big data, and customer experience analytics, we deliver business intelligence for organizations of all sizes.

Our client success stories feature a range of clients from Fortune 500 companies to fast-growing startups. PMRs collaborative environment is committed to building industry-specific solutions by transforming data from multiple streams into a strategic asset.

Contact us:

Naved BegPersistence Market ResearchAddress 305 Broadway, 7th Floor, New York City,NY 10007 United StatesU.S. Ph. +1-646-568-7751USA-Canada Toll-free +1 800-961-0353Sales[emailprotected]Websitehttps://www.persistencemarketresearch.com

Go here to read the rest:
The Hematopoietic Stem Cells Transplantation market to Undergo positive Transformation between 2017 and 2025 - Crypto Daily

Fort Collins, Colorado The Stem Cell Therapy Market is growing at a rapid pace and contributes significantly to the global economy in terms of turnover, growth rate, sales, market share and size. The Stem Cell Therapy Market Report is a comprehensive research paper that provides readers with valuable information to understand the basics of the Stem Cell Therapy Report. The report describes business strategies, market needs, dominant market players and a futuristic view of the market.

The report has been updated to reflect the most recent economic scenario and market size regarding the ongoing COVID-19 pandemic. The report looks at the growth outlook as well as current and futuristic earnings expectations in a post-COVID scenario. The report also covers changing market trends and dynamics as a result of the pandemic and provides an accurate analysis of the impact of the crisis on the market as a whole.

Global Stem Cell TherapyMarketwas valued at 117.66 million in 2019 and is projected to reach USD255.37 million by 2027, growing at a CAGR of 10.97% from 2020 to 2027.

Industry Stem Cell Therapy Study provides an in-depth analysis of key market drivers, opportunities, challenges and their impact on market performance. The report also highlights technological advancements and product developments that drive market needs.

The report contains a detailed analysis of the major players in the market, as well as their business overview, expansion plans and strategies. Key players explored in the report include:

The report provides comprehensive analysis in an organized manner in the form of tables, graphs, charts, pictures and diagrams. Organized data paves the way for research and exploration of current and future market outlooks.

Request a Discount on the report @ https://reportsglobe.com/ask-for-discount/?rid=33553

The report provides comprehensive data on the Stem Cell Therapy market and its trends to help the reader formulate solutions to accelerate business growth. The report provides a comprehensive overview of the economic scenario of the market, as well as its benefits and limitations.

The Stem Cell Therapy Market Report includes production chain analysis and value chain analysis to provide a comprehensive picture of the Stem Cell Therapy market. The research consists of market analysis and detailed analysis of application segments, product types, market size, growth rates, and current and emerging industry trends.

1.Stem Cell Therapy Market, By Cell Source:

Adipose Tissue-Derived Mesenchymal Stem Cells Bone Marrow-Derived Mesenchymal Stem Cells Cord Blood/Embryonic Stem Cells Other Cell Sources

2.Stem Cell Therapy Market, By Therapeutic Application:

Musculoskeletal Disorders Wounds and Injuries Cardiovascular Diseases Surgeries Gastrointestinal Diseases Other Applications

3.Stem Cell Therapy Market, By Type:

Allogeneic Stem Cell Therapy Market, By Application Musculoskeletal Disorders Wounds and Injuries Surgeries Acute Graft-Versus-Host Disease (AGVHD) Other Applications Autologous Stem Cell Therapy Market, By Application Cardiovascular Diseases Wounds and Injuries Gastrointestinal Diseases Other Applications

Request customization of the report @https://reportsglobe.com/need-customization/?rid=33553

The market is geographically spread across several key geographic regions and the report includes regional analysis as well as production, consumption, revenue and market share in these regions for the 2020-2027 forecast period. Regions include North America, Latin America, Europe, Asia Pacific, the Middle East, and Africa.

Radical Coverage of the Stem Cell Therapy Market:

Key Questions Addressed in the Report:

To learn more about the report, visit @ https://reportsglobe.com/product/global-stem-cell-therapy-market/

Thanks for reading our report. It is possible to adapt this report to the wishes of the customer. Contact us to learn more about the report and our team will make sure you create a report based on your needs.

How Reports Globe is different than other Market Research Providers

The inception of Reports Globe has been backed by providing clients with a holistic view of market conditions and future possibilities/opportunities to reap maximum profits out of their businesses and assist in decision making. Our team of in-house analysts and consultants works tirelessly to understand your needs and suggest the best possible solutions to fulfill your research requirements.

Our team at Reports Globe follows a rigorous process of data validation, which allows us to publish reports from publishers with minimum or no deviations. Reports Globe collects, segregates, and publishes more than 500 reports annually that cater to products and services across numerous domains.

Contact us:

Mr. Mark Willams

Account Manager

US: +1-970-672-0390

Email:[emailprotected]

Web:reportsglobe.com

Read the original:
Stem Cell Therapy Market Potential Growth, Size, Share, Demand and Analysis of Key Players Research Forecasts to 2027 - The Daily Chronicle

Watch here: https://www.youtube.com/watch?feature=youtu.be&v=VVkQU91KbEs

press release: The UW has a long history of pioneering medical advancements that have transformed the world. From performing the first bone marrow transplant in the United States to cultivating the first laboratory-derived human embryonic stem cells. Now, where will UW medical research go next?

On the next Wisconsin Medicine Livestream, meet trailblazing doctors, researchers, and medical leaders who are charting a bold course to completely alter the health care landscape. During this insightful panel discussion, well explore how gene therapy and cell replacements could hold the keys to treating inherited and acquired blindness. Youll also discover the remarkable potential in xenotransplantation where nonhuman animal source organs are transplanted into human recipients. In addition, you will learn about UW Healths journey to build a multidisciplinary program to serve the community. These, and other, fascinating developments in treatment and care are happening right now at the UW and are the future of medicine. The presentation will be moderated by Robert Golden, the dean of the University of WisconsinMadisons School of Medicine and Public Health.

Our Guests:

David Gamm, professor, Department of Ophthalmology and Visual Sciences; Emmett A. Humble Distinguished Director, McPherson Eye Research Institute; Sandra Lemke Trout Chair in Eye Research

Dr. Gamms lab is at the forefront in developing cell-based therapies to combat retinal degenerative diseases (RDDs). As the director of the McPherson Eye Research Institute and a member of the Waisman Center Stem Cell Research Program, the UW Stem Cell and Regenerative Medicine Center, and the American Society for Clinical Investigation, his efforts are directed toward basic and translational retinal stem cell research. The Gamm Lab uses induced pluripotent stem cells to create retinal tissues composed of authentic human photoreceptor cells rods and cones that can detect light and initiate visual signals in a dish. The aims of his laboratory are to investigate the cellular and molecular events that occur during human retinal development and to generate cells for use in retinal disease modeling and cell replacement therapies. In collaboration with other researchers at UWMadison and around the world, the lab is developing methods to produce and transplant photoreceptors and/or retinal pigment epithelium (RPE) in preparation for future clinical trials. At the same time, the Gamm Lab uses lab-grown photoreceptor and RPE cells to test and advance a host of other experimental treatments, including gene therapies. In so doing, the lab seeks to delay or reverse the effects of blinding disorders, such as retinitis pigmentosa and age-related macular degeneration, and to develop or codevelop effective interventions for these RDDs at all stages of disease.

Dhanansayan Shanmuganayagam, assistant professor, Department of Surgery, School of Medicine and Public Health; Department of Animal and Dairy Sciences, UWMadison; director, Biomedical, and Genomic Research Group

Dr. Shanmuganayagams research focuses on the development and utilization of pigs as homologous models to close the translational gap in human disease research, taking advantage of the overwhelming similarities between pigs and humans in terms of genetics, anatomy, physiology, and immunology. He and his colleagues created the human-sized Wisconsin Miniature Swine breed that is unique to the university. The breed exhibits greater physiological similarity to humans, particularly in vascular biology and in modeling metabolic disorders and obesity. He currently leads genetic engineering of swine at the UW. His team has created more than 15 genetic porcine models including several of pediatric genetic cancer-predisposition disorders such as neurofibromatosis type 1 (NF1). In the context of NF1, his lab is studying the role of alternative splicing of the nf1 gene on the tissue-specific function of neurofibromin and whether gene therapy to modulate the regulation of this splicing can be used as a viable treatment strategy for children with the disorder.

Dr. Shanmuganayagam is also currently leading the efforts to establish the University of Wisconsin Center for Biomedical Swine Research and Innovation (CBSRI) that will leverage the translatability of research in pig models and UWMadisons unique swine and biomedical research infrastructure, resources, and expertise to conduct innovative basic and translational research on human diseases. The central mission of CBSRI is to innovate and accelerate the discovery and development of clinically relevant therapies and technologies. The center will also serve to innovate graduate and medical training. As the only center of its kind in the United States, CBSRI will make UWMadison a hub of translational research and industry-partnered biomedical innovation.

Petros Anagnostopoulos, surgeon in chief, American Family Childrens Hospital; chief, Section of Pediatric Cardiothoracic Surgery; professor, Department of Surgery, Division of Cardiothoracic Surgery

Dr. Anagnostopoulos is certified by the American Board of Thoracic Surgery and the American Board of Surgery. He completed two fellowships, one in cardiothoracic surgery at the University of Pittsburgh School of Medicine and a second in pediatric cardiac surgery at the University of California, San Francisco School of Medicine. He completed his general surgery residency at Henry Ford Hospital in Detroit. Dr. Anagnostopoulos received his MD from the University of Athens Medical School, Greece. His clinical interests include pediatric congenital heart surgery and minimally invasive heart surgery.

Dr. Anagnostopoulos specializes in complex neonatal and infant cardiac reconstructive surgery, pediatric heart surgery, adult congenital cardiac surgery, single ventricle palliation, extracorporeal life support, extracorporeal membrane oxygenation, ventricular assist devices, minimally invasive cardiac surgery, hybrid surgical-catheterization cardiac surgery, off-pump cardiac surgery, complex mitral and tricuspid valve repair, aortic root surgery, tetralogy of Fallot, coronary artery anomalies, Ross operations, obstructive cardiomyopathy, and heart transplantation.

When: Tuesday, Sept. 29, at 7 p.m. CDT

Where: Wisconsin Medicine Livestream: wiscmedicine.org/programs/ending-alzheimers

More:
ONLINE: The Future of Medicine - Isthmus

COMEDIAN Al Murray told Lorraine Kelly today his seven-year-old nephew is "very ill" but is "hanging in there" amid his battle with leukemia.

The Pub Landlord star, 52, appeared on the ITV lunchtime show to urge viewers to donate the price of a round of drinks to cancer charity DKMS, and help them boost their stem cell register.

3

Finley Relf has a rare and aggressive form ofleukaemiaand his only hope of survival is a bone marrow transplant.

Al's partner Eleanor Relf is the sister of Finley's dad Ben. Finley's family had been tested to be a donor, but are sadly not a match.

Speaking to Lorraine, 60, Al said: "Hes an incredibly brave little lad.

"I cant tell you hes well, hes very, very ill, but hes hanging in there.

"They load him up with treatments and he takes it. But he's seven, he wants to be out there playing footballwith his friends."

3

Al went on: "People can get on this register and then be a life saver in waiting. That's what we're trying to do.

"Theres been a decline in donors and contributions. The thing about blood stem cell donations is its dead easy.

"They send you a pack with swabs and you swab some cheek tissue and send it back to them."

And Lorraine joked: "It's just like picking your nose which we all do!"

It takes just a few minutes to become a donorbut Al explained that processing the swabs to add people to the register costs DKMS 40 per person.

3

Finley, who lives in Haywards Heath, West Sussex, with four-year-old brother Jacob, is having chemotherapy at Londons Great Ormond Street Hospital.

Finleys family are hoping another stranger will be identified as his genetic twin with similar tissue. Al said Finley has tried two donors but their stem cells didn't work.

He said: Its not like blood types. Its much more fundamental to your DNA."

bump-dateAmy Huberman shows off growing baby bump revealing she needs 'loo 453 times day'

PUSSYCAT KITTEN?Nicole Scherzinger deletes pic after fans say she's showing a baby bump

HOLL FOR LEATHERHolly Willoughby sets pulses racing in sexy leather skirt on This Morning

DONAL'S BACKFans say it's 'great to see' Donal Maher back on Fair City as Paul helps Ruth

Cute familyKian Egan and wife Jodi Albert 'proud' as they celebrate son's Holy Communion

Baby prepGlenda Gilson reveals she's 'packing hospital bag' as due date fast approaches

"Its not quite fingerprints but its on that level, so thats why we need more people to join the register."

Finleys ordeal started last year when his parents Ben and Nicky noticed he was losing weight.

Doctors discovered his spleen was enlarged and he underwent numerous tests, which led them to diagnosing him with blood cancer.

Visit link:
Al Murray says his seven-year-old nephew is very ill but hanging on in there amid battle with rare form of L - The Irish Sun

AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced that the European Commission (EC) has granted orphan drug designation for AVR-RD-02, the companys investigational gene therapy for the treatment of Gaucher disease. AVR-RD-02 consists of the patients own hematopoietic stem cells, genetically modified to express glucocerebrosidase (GCase), the enzyme that is deficient in Gaucher disease. AVROBIO recently dosed the first patient in the GuardOne Phase 1/2 clinical trial to evaluate the safety and efficacy of AVR-RD-02.

"Like many lysosomal disorders, Gaucher disease can lead to debilitating complications throughout the body and brain. The standard of care does not address all these symptoms and may not be able to halt progression of the disease," said Geoff MacKay, AVROBIOs president and CEO. "Our investigational gene therapy is designed to address the head-to-toe manifestations of Gaucher disease with a single dose. Were pleased to receive orphan drug designation, which recognizes the potential of our approach to transform the standard of care and, we hope, the quality of life for people living with this rare genetic disorder."

The EC grants orphan drug designation to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions that impact fewer than 5 in 10,000 patients in the European Union. Orphan drug designation gives companies certain benefits, including reduced regulatory fees, clinical protocol assistance, research grants and 10 years of market exclusivity following regulatory approval.

AVR-RD-02 has also received orphan drug designation from the U.S. Food and Drug Administration.

About Gaucher Disease

Gaucher disease is a rare, inherited lysosomal storage disorder characterized by the toxic accumulation of glucosylceramide (GlcCer) and glucosylsphingosine (GlcSph) in macrophages. Macrophages bloated with these fatty substances are called Gaucher cells which amass primarily in the spleen, liver and bone marrow. This results in a variety of potential symptoms, including grossly enlarged liver and spleen, bone issues, fatigue, low hemoglobin levels and platelet counts and an adjusted lifetime relative risk of developing Parkinson's disease that may be more than 20 times greater than the general population. Even on enzyme replacement therapy (ERT) the current standard of care people with Gaucher disease type 1 typically have a shortened life expectancy and may experience debilitating symptoms that significantly reduce their quality of life. An estimated 1 in 44,000 people are diagnosed with Gaucher disease.

Story continues

About AVR-RD-02

AVR-RD-02 is an investigational lentiviral gene therapy designed to provide a durable therapeutic benefit for people living with Gaucher disease. The therapy starts with the patients own hematopoietic stem cells, which are genetically modified to express functional glucocerebrosidase (GCase). Functional GCase reduces levels of glucosylceramide and glucosylsphingosine, the accumulated substances which cause the symptoms of Gaucher disease. AVROBIO is currently evaluating AVR-RD-02 in GuardOne, a Phase 1/2 clinical trial.

About lentiviral gene therapy

Lentiviral vectors are differentiated from other delivery mechanisms because of their large cargo capacity and their ability to integrate the therapeutic gene directly into the patients chromosomes. This integration is designed to maintain the therapeutic genes presence as the patients cells divide, which potentially enables dosing of pediatric patients, whose cells divide rapidly as they grow. Because the therapeutic gene is integrated into the patients own stem cells, patients are not excluded from receiving the investigational therapy due to pre-existing antibodies to the vector.

About AVROBIO

Our vision is to bring personalized gene therapy to the world. We aim to halt or reverse disease throughout the body by driving durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle and bone. Our clinical-stage programs include Fabry disease, Gaucher disease and cystinosis and we also are advancing a preclinical program in Pompe disease. AVROBIO is powered by the plato gene therapy platform, our foundation designed to scale gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario. For additional information, visit avrobio.com, and follow us on Twitter and LinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words and phrases such as "aims," "anticipates," "believes," "could," "designed to," "estimates," "expects," "forecasts," "goal," "intends," "may," "plans," "possible," "potential," "seeks," "will," and variations of these words and phrases or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding our business strategy for and the potential therapeutic benefits of our prospective product candidates, including AVR-RD-02 for the treatment of Gaucher disease; the anticipated benefits of the European Commissions grant of orphan drug designation for AVR-RD-02; the design, commencement, enrollment and timing of ongoing or planned clinical trials and regulatory pathways; the timing of patient recruitment and enrollment activities, clinical trial results, and product approvals; the anticipated benefits of our gene therapy platform including the potential impact on our commercialization activities, timing and likelihood of success; the expected benefits and results of our implementation of the plato platform in our clinical trials and gene therapy programs; and the expected safety profile of our investigational gene therapies. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in pre-clinical or early-stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

Any forward-looking statements in this press release are based on AVROBIOs current expectations, estimates and projections about our industry as well as managements current beliefs and expectations of future events only as of today and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that any one or more of AVROBIOs product candidates will not be successfully developed or commercialized; the risk of cessation or delay of any ongoing or planned clinical trials of AVROBIO or our collaborators; the risk that AVROBIO may not successfully recruit or enroll a sufficient number of patients for our clinical trials; the risk that AVROBIO may not realize the intended benefits of our gene therapy platform, including the features of our plato platform; the risk that our product candidates or procedures in connection with the administration thereof will not have the safety or efficacy profile that we anticipate; the risk that prior results, such as signals of safety, activity or durability of effect, observed from pre-clinical or clinical trials, will not be replicated or will not continue in ongoing or future studies or trials involving AVROBIOs product candidates; the risk that we will be unable to obtain and maintain regulatory approval for our product candidates; the risk that the size and growth potential of the market for our product candidates will not materialize as expected; risks associated with our dependence on third-party suppliers and manufacturers; risks regarding the accuracy of our estimates of expenses and future revenue; risks relating to our capital requirements and needs for additional financing; risks relating to clinical trial and business interruptions resulting from the COVID-19 outbreak or similar public health crises, including that such interruptions may materially delay our development timeline and/or increase our development costs or that data collection efforts may be impaired or otherwise impacted by such crises; and risks relating to our ability to obtain and maintain intellectual property protection for our product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause AVROBIOs actual results to differ materially and adversely from those contained in the forward-looking statements, see the section entitled "Risk Factors" in AVROBIOs most recent Quarterly Report, as well as discussions of potential risks, uncertainties and other important factors in AVROBIOs subsequent filings with the Securities and Exchange Commission. AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200928005176/en/

Contacts

Investor Contact: Christopher F. BrinzeyWestwicke, an ICR Company339-970-2843chris.brinzey@westwicke.com

Media Contact: Tom DonovanTen Bridge Communications857-559-3397tom@tenbridgecommunications.com

See the article here:
AVROBIO Receives Orphan Drug Designation from the European Medicines Agency for AVR-RD-02, an Investigational Gene Therapy for Gaucher Disease - Yahoo...

MINNEAPOLIS, Sept. 24, 2020 /PRNewswire/ --Today, Senator Jack Reed (D-RI) introduced the TRANSPLANT Act (S. 4762), which will provide for the timely reauthorization of the C.W. Bill Young Transplantation Program (Program). The National Marrow Donor Program(NMDP)/Be The Match applauds the leadership of Senator Reed and urges Congress to act quickly to reauthorize the Program, which expires on September 30th.

"We appreciate the leadership of the bill's sponsor Sen. Jack Reed (D-RI), and the bipartisan support of the bill's lead cosponsors Sens. Richard Burr (R-NC), Tina Smith (D-MN), and Tim Scott (R-SC)," said NMDP/Be The Match Chief Policy Officer Brian Lindberg.

The Program provides access to live-saving bone marrow, peripheral blood stem cell (PBSC) and cord blood transplants to those living with one of the more than 70 blood cancers or blood disorders for which a transplant is the only curative option.

Since its inception in the mid-1980s, NMDP/Be The Match has been entrusted by Congress to operate the Program and has facilitated more than 100,000 life-saving transplants to give patients with otherwise fatal blood cancers or blood disorders a second chance at life.

The COVID-19 pandemic with increasingly restrictive travel bans, border closings, and the declining availability of scheduled commercial passenger aviation service on international and domestic routes - has created unprecedented obstacles for NMDP/ Be The Match in delivering life-saving cells to the patients who need them.

This new reality creates a web of increasingly complex challenges in facilitating the timely collection and delivery of bone marrow products to patients who are in the midst of treatment protocols or whose conditions have deteriorated to the point that a bone marrow transplant is the only course of treatment that will save their life.

"During the pandemic, we have been able to mitigate any disruption to our mission and complete more than 3,000 lifesaving therapies without missing a single delivery," continued Lindberg. "We have been able to do that, in large part, because of our designation in Federal law as the nation's bone marrow registry. Unfortunately, that designation will lapse with the expiration of our Federal authorization on September 30, 2020."

This designation has helped Be The Match to care for our patients during the pandemic by:

"Our ability to call upon our Federal partners in these extreme circumstances, and our standing in the eyes of foreign governments where we must operate, could be compromised after September 30th if our authorization expires and we are no longer technically designated as the Nation's registry," said Lindberg. "This could put at risk our ability to continue to ensure the timely delivery of life-saving cellular products to otherwise terminally ill patients here and throughout the world.

"Ensuring access to cellular therapy is critical for the patients we serve," continued Lindberg. "Congress must move immediately to reauthorize these programs to expand the number of adult volunteer donors and cord blood units on the national registry so that every American who needs a transplant can find a match."

About Be The MatchFor people with life-threatening blood cancerslike leukemia and lymphomaor other diseases, a cure exists. Be The Match connects patients with their donor match for a life-saving marrow or umbilical cord blood transplant. People can contribute to the cure as a member of the Be The Match Registry, financial contributor or volunteer. Be The Match provides patients and their families one-on-one support, education, and guidancebefore, during and after transplant.

Be The Match is operated by the National Marrow Donor Program (NMDP), a nonprofit organization that matches patients with donors, educates health care professionals and conducts research through its research program, CIBMTR (Center for International Blood and Marrow Transplant Research), so more lives can be saved. To learn more about the cure, visit BeTheMatch.org or call 1 (800) MARROW-2.

SOURCE Be The Match

https://bethematch.org

View post:
National Marrow Donor Program/Be The Match Urges Congress to Quickly Pass Reauthorization Bill to Protect Access to Life Saving Bone Marrow and Cord...

Latest Research Study on Stem Cell Therapy Market published by The Insight Partners, offers a detailed overview of the factors influencing the global business scope. The research report provides deep insights into the global market revenue, parent market trends, macro-economic indicators, and governing factors, along with market attractiveness per market segment. The report provides an overview of the growth rate of the Stem Cell Therapy market during the forecast period, i.e., 20202027. Most importantly, the report further identifies the qualitative impact of various market factors on market segments and geographies. The research segments the market on the basis of product type, application, technology, and region. To offer more clarity regarding the industry, the report takes a closer look at the current status of various factors including but not limited to supply chain management, niche markets, distribution channel, trade, supply, and demand and production capability across different countries. Some of the key players profiled in the study are MEDIPOST, Pharmicell Co., Inc., RichSource, BioTime Inc. (Lineage Cell Therapeutics, Inc.), Mesoblast Limited, Holostem Terapie Avanzate Srl, U.S. Stem Cell, Inc., Caladrius Biosciences, Inc., TiGenix NV, AlloSource, etc.

The stem cell therapy marketwas valued at US$ 1,534.55 million in 2019 and is expected to grow at a CAGR of 16.7% from 2020to 2027 to reach US$ 5,129.66 million by 2027.

Request Sample Copy of Stem Cell Therapy Market at: https://www.theinsightpartners.com/sample/TIPHE100000991/

Stem Cell Therapy Market In-Depth Analysis

Stem cells are preliminary body cells from which all other cells with specialized functions are generated. Under controlled environment in the body or a clinical laboratory, these cells divide to form more cells called daughter cells. Due to the advent of modern health science, these cells play a major role in understanding the occurrence of diseases, generation of advanced regenerative medicines, and drug discovery. There are certain sources such as embryo, bone marrow, body fats, and umbilical cord blood amongst others, where stem cells are generated. The global stem cell therapy market is driven by factors such asincreasing awareness related to the stem cells therapy in effective disease management and growing demand for regenerative medicines. However, high cost related with stem cell therapy is likely to obstruct the growth of the stem cell therapymarket during the forecast period. The growing research and development activities in Asia Pacific region is expected to offer huge growth opportunity for stem cell therapy market.

End User Insights

Based on end user, the Stem Cell Therapy market is segmented into academic and research institutes, hospitals and specialty clinics. The academic and research institutes held the largest share of end user segment in the global market and is expected to grow at the fastest rate during the forecast period.

Key Benefits

The report profiles the key players in the industry, along with a detailed analysis of their individual positions against the global landscape. The study conducts SWOT analysis to evaluate strengths and weaknesses of the key players in the Stem Cell Therapy market. The researcher provides an extensive analysis of the Stem Cell Therapy market size, share, trends, overall earnings, gross revenue, and profit margin to accurately draw a forecast and provide expert insights to investors to keep them updated with the trends in the market.

Competitive scenario:

The study assesses factors such as segmentation, description, and applications of Stem Cell Therapy industries. It derives accurate insights to give a holistic view of the dynamic features of the business, including shares, profit generation, thereby directing focus on the critical aspects of the business.

Scope of the Report

The research on the Stem Cell Therapy market focuses on mining out valuable data on investment pockets, growth opportunities, and major market vendors to help clients understand their competitors methodologies. The research also segments the Stem Cell Therapy market on the basis of end user, product type, application, and demography for the forecast period 20212027. Comprehensive analysis of critical aspects such as impacting factors and competitive landscape are showcased with the help of vital resources, such as charts, tables, and infographics.

Global Stem Cell Therapy Market By Type

Global Stem Cell Therapy Market By Treatment

Global Stem Cell Therapy Market ByApplication

Global Stem Cell Therapy Market By End User

Promising Regions & Countries Mentioned in TheStem Cell Therapy Market Report:

Major highlights of the report:

All-inclusive evaluation of the parent market

Evolution of significant market aspects

Industry-wide investigation of market segments

Assessment of market value and volume in past, present, and forecast years

Evaluation of market share

Study of niche industrial sectors

Tactical approaches of market leaders

Lucrative strategies to help companies strengthen their position in the market

Interested in purchasing this Report? Click here @ https://www.theinsightpartners.com/buy/TIPHE100000991/

Thanks for reading this article; you can also customize this report to get select chapters or region-wise coverage with regions such as Asia, North America, and Europe.

About Us:

The Insight Partners is a one stop industry research provider of actionable intelligence. We help our clients in getting solutions to their research requirements through our syndicated and consulting research services. We are committed to provide highest quality research and consulting services to our customers. We help our clients understand the key market trends, identify opportunities, and make informed decisions with our market research offerings at an affordable cost.

We understand syndicated reports may not meet precise research requirements of all our clients. We offer our clients multiple ways to customize research as per their specific needs and budget

Contact Us:

The Insight Partners,

Phone: +1-646-491-9876

Email: [emailprotected]

Read more here:
Massive Growth Seen in Stem Cell Therapy Market 2020-2027 | In Depth Analysis with Top Key Players RichSource, Mesoblast Limited, TiGenix NV,...

Alyssa Mulvey in the Great North Children's Hospital at the RVI.

The total brought in by the man who walked 21 laps around Elizabethan walls earlier this month reached 2,000 earlier this week.

He was accompanied by Stewart Brown for the whole 26.2-mile marathon distance and family, friends and other supporters did some of the laps during the event in aid of wards in the Great North Children's Hospital (GNCH) at the RVI in Newcastle.

On his fund-raising page, Derek who lives in Highcliffe, Spittal specifically mentioned two local girls from Berwick (Alyssa Mulvey and Kelis Bloomfield) who are currently receiving treatment at the GNCH.

Alyssa has been poorly since birth and was diagnosed with A20 haploinsufficiency, a very rare auto-immune condition, when she was 10.

She has been on ward 3, a bone marrow transplant ward that deals with children who were born with zero immune systems or severe immune conditions. She had recovered well from a stem cell transplant last year, using stem cells donated by her dad Shane, but then contracted Covid-19.

Fortunately, Alyssa made an excellent recovery. However, a check-up weeks later revealed her blood pressure was extremely high and tests found that she had a serious kidney disease called thrombotic microangiopathy (TMA).

Although the condition was not caused by Covid-19, medics believe the virus ignited it in her system and that it had been dormant beforehand. She has now been at the GNCH for 21 consecutive weeks.

Kelis receives treatment on ward 2a. She has eosinophilic esophagitis (EoE), which is a chronic allergic inflammatory disease of the esophagus. She is allergic to things such as grass and pollen, which can cause respiratory failure.

Ward 2a specialises in respiratory and long-term ventilation, and diabetes.

Derek has previously supported the GNCH by doing the Great North Run half marathon his way of giving back to the team who saved the life of his daughter, Saskia, in 2018.

As the run was not held this year due to the coronavirus pandemic, he still wanted to do something in 2020 and so came up with the local challenge, which took place earlier this month before the extra local and national restrictions were announced.

He said: We set off just before 6am and after 8am, Deva (his wife) and Saskia joined us and walked the opposite way. Friends and supporters did some of the laps with us.

"We kept a brisk pace and when we had a break, we had a bacon roll and coffee courtesy of The Mule on Rouge.

"Many of the visitors on the day asked us why were doing the walk and a combination of visitors, friends and supporters saw a lot of people put money into the charity bucket that we had with us when we counted it later, the total in it was 255.

In the afternoon, Deva and Saskia completed half marathon distance and with six laps to go we were joined by the Plundering Pirates of the North East, dressed in costume, who raised money for their own charities. The last lap was done with Stewarts wife Amanda and their children, Mathew and Mia. Me and Stewart were pleased to complete the marathon distance in 10 hours.

Also thanks to all those who have donated online gf.me/u/yny5p2 its great that Ill be giving more than 1,000 to the two wards.

Alyssas mum Kirstie said that her daughter, 14, has infusions meds going in through a central line every day and has the dialysis procedure four to five times a week.

In relation to Dereks fund-raising, she said: He is amazing and we think he is an absolute superhero for helping to raise funds for and awareness of these wards.

The money will be vital for them because they are not as well known as others such as the cancer wards.

Other support for Alyssa and her family includes Kirsties best friend Julie Newton organising a raffle (1,000) and piping events on Thursday evenings at Mordington Avenue until last week (about 700 in total from the bucket donations each week).

Denise Lody from Facebook group Isolation Berwick upon Tweed and Surrounding Area set-up a GoFundMe page that raised 2,651.

Sean Ryan and Dave Smith of 2SPT raised 2,623.55 by running private physical training sessions through the lockdown period.

Further funds came from a beautiful cake raffled by Ashley McKnight and Berwick landscape postcards sold by Julie.

Kirstie said: The response has been unbelievable. Alyssa said to me why are they doing this for me, but I reminded her of how brave she is and told her how much people love her and they wanted to show just how much they care.

We were unable to speak in depth with Kelis mum Stacey, but she did send us a message that said: What Derek has done for these wards is absolutely amazing.

"Kelis has helped out by doing an online raffle, which raised 70 to the cause.

Originally posted here:
Marathon effort in aid of hospital wards - Berwick Advertiser