President Joe Biden: A Proclamation on National Sickle Cell Awareness Month, 2022

Sickle cell disease (SCD) presents grave health challenges for an estimated 100,000 Americans.  For some, it triggers intermittent episodes of pain, difficulty with vision, and serious fatigue.  Other survivors experience this disease more acutely — SCD can cause infections, strokes, and even organ failure.  For almost everyone impacted, coping with inherited red blood cell disorders means putting plans on pause, living with excruciating pain, paying for expensive treatments, and hoping for a day when medications and doctor visits no longer interrupt life.  During National Sickle Cell Awareness Month, we recognize the perseverance of SCD patients, and we recommit to working with our partners in State and local government, the nonprofit space, and the private sector to develop treatments and cures for this debilitating disease.

     Like many rare diseases, SCD affects our population unevenly.  Black and Brown Americans are disproportionately affected.  About 1 in 13 Black children tests positive for the sickle cell trait, and about 1 in 365 Black Americans develops the disease over the course of their lifetime.  Due to persistent systemic inequities in our health care system, these same patients are also often the last to get help.  Few specialty clinics are available for SCD treatments, information about detecting this disease is not always widely shared, and pain management can be a challenge due to the intermittent nature of sickle cell crises and persistent racial disparities in pain assessment and treatment.  Moreover, there exists no universally effective cure; bone marrow and stem cell transplants have allowed some people to overcome SCD, but low donor availability and treatment-related complications render these procedures unviable for many patients.

     Medical professionals and scientists in my Administration and across our Nation are working to put an end to SCD.  The Food and Drug Administration recently approved new drug therapies to help patients manage their pain.  Through its “Cure Sickle Cell Initiative,” the National Institutes of Health (NIH) is striving to develop safe and effective genetic therapies and exploring applications for machine learning to predict organ function decline in SCD patients.  Additionally, the NIH has invited researchers to apply for funding to support large-scale clinical trials on treating SCD pain symptoms.  We are closer than ever to finding a cure today for all patients, and I am optimistic about our progress.

     Even so, it is still important for Americans to understand the signs of this disease, the risks of inheriting this condition, as well as the various resources available to those who test positive.  Most people with the sickle cell trait do not exhibit symptoms, and many are unaware of their potential to carry on this gene.  Experts agree that it is important to get tested, especially if you have family members who have been diagnosed with SCD.  There are also helpful resources online to learn more about this disease, like the Centers for Disease Control and Prevention’s sickle cell information page at cdc.gov/ncbddd/sicklecell/index.html.

     As we continue our quest to cure sickle cell disease, let us celebrate the strides our health experts have made in understanding and treating this condition.  Let us offer strength to those Americans fighting its effects today and unite in our mission to enhance the quality of life for those diagnosed with SCD.

     NOW, THEREFORE, I, JOSEPH R. BIDEN JR., President of the United States of America, by virtue of the authority vested in me by the Constitution and the laws of the United States, do hereby proclaim September 2022 as National Sickle Cell Awareness Month.  I call upon the people of the United States to learn more about the progress we are making to reduce the burden of this disease on our fellow Americans.

     IN WITNESS WHEREOF, I have hereunto set my hand this thirty-first day of August, in the year of our Lord two thousand twenty-two, and of the Independence of the United States of America the two hundred and forty-seventh. 

                             JOSEPH R. BIDEN JR.

American Red Cross urges more African Americans to donate blood to sickle cell disease patients

Although this story is part of a push to get Philadelphia residents to attend a blood drive, its message about a lack of African American donors is relevant to both potential donors and those with sickle cell disease.

The American Red Cross is in great need of more African Americans to donate blood, which is a critical need for many patients with sickle cell anemia.

Sickle cell disease affects 100,000 Americans. According to the Centers for Disease Control, it affects one out of every 365 African American births, which is 73% of all cases.

More Black blood donors urgently needed to help sickle cell patients

The American Red Cross is emphasizing the unique role Black blood donors play in the medical treatment of those living with sickle cell disease during Sickle Cell Awareness Month this September. Right now, more Black blood donors are critically needed to help patients battling sickle cell disease as blood drives – especially those at schools, colleges and universities – continue to be canceled at alarming rates.

Last spring, more Black blood donors gave at Red Cross blood drives held at educational institutions than at any other blood drive location type. As drives across the country canceled this spring due to coronavirus concerns, the number of Black blood donors giving at these schools decreased from over 15,000 in 2019 to about 2,700 this year. Drives at educational institutions make up the largest percentage of fall blood drive cancellations, so the need for more Black blood donors for sickle cell patients is expected to remain urgent.

“Sickle cell disease profoundly impacts the quality of life of those living with this inherited blood disorder, and your blood donation could be the donation that helps a patient keep fighting,” said Dr. Yvette Miller, executive medical director, Red Cross Blood Services. “The pandemic hasn’t stopped the need for transfusions for sickle cell patients. The Red Cross encourages eligible donors to roll up a sleeve and share their strength with patients during Sickle Cell Awareness Month.”

More Black blood donors are urged to make a blood donation appointment by downloading the Red Cross Blood Donor App, visiting RedCrossBlood.org, calling 1-800-RED CROSS (1-800-733-2767) or enabling the Blood Donor Skill on any Alexa Echo device.

How donations from Black blood donors help sickle cell patients

About 100,000 people in the U.S., most of whom are of African or Latino descent, are living with sickle cell disease, making it the most common genetic blood disease in the country. Sickle cell disease causes red blood cells to be sticky, hard and crescent-shaped instead of soft and round. This makes it difficult for blood to flow smoothly and carry oxygen to the rest of the body, which may lead to severe pain, tissue and organ damage, acute anemia and even strokes.

Blood transfusion helps sickle cell disease patients by increasing the number of normal red blood cells in the body, helping to deliver oxygen and unblock blood vessels. Patients with sickle cell disease depend on blood that must be matched very closely – beyond the A, B, O and AB blood types – to reduce the risk of complications. Some of these rare blood types are unique to specific racial and ethnic groups, and because of this, sickle cell disease patients are more likely to find a compatible blood match from a blood donor who is Black.

Get more information about blood and diversity.

Important COVID-19 information for donors

The Red Cross is testing blood, platelet and plasma donations for COVID-19 antibodies. The test may indicate if the donor’s immune system has produced antibodies to this coronavirus, regardless of whether they developed symptoms. Red Cross antibody tests will be helpful to identify individuals who have COVID-19 antibodies and may qualify to be convalescent plasma donors. Convalescent plasma is a type of blood donation collected from COVID-19 survivors that have antibodies that may help patients who are actively fighting the virus. Donors can expect to receive the results of their antibody test within 7 to 10 days through the Red Cross Blood Donor App or the donor portal at RedCrossBlood.org.

The Red Cross is not testing donors to diagnose illness, referred to as a diagnostic test. To protect the health and safety of Red Cross staff and donors, it is important that individuals who do not feel well or believe they may be ill with COVID-19 postpone donation.

Each Red Cross blood drive and donation center follows the highest standards of safety and infection control, and additional precautions – including temperature checks, social distancing and face coverings for donors and staff – have been implemented to help protect the health of all those in attendance. Donors are asked to schedule an appointment prior to arriving at the drive and are required to wear a face covering or mask while at the drive, in alignment with Centers for Disease Control and Prevention public guidance.  

How to donate blood

A blood donor card or driver’s license or two other forms of identification are required at check-in. Individuals who are 17 years of age in most states (16 with parental consent where allowed by state law), weigh at least 110 pounds and are in generally good health may be eligible to donate blood. High school students and other donors 18 years of age and younger also have to meet certain height and weight requirements.

Blood and platelet donors can save time at their next donation by using RapidPass® to complete their pre-donation reading and health history questionnaire online, on the day of their donation, before arriving at the blood drive. To get started, follow the instructions at RedCrossBlood.org/RapidPass or use the Blood Donor App.

About the American Red Cross

The American Red Cross shelters, feeds and provides emotional support to victims of disasters; supplies about 40% of the nation’s blood; teaches skills that save lives; provides international humanitarian aid; and supports military members and their families. The Red Cross is a not-for-profit organization that depends on volunteers and the generosity of the American public to perform its mission. For more information, please visit redcross.org or cruzrojaamericana.org, or visit us on Twitter at @RedCross.

Sickle Cell Disease Association celebrates National Sickle Cell Awareness Month

Sickle Cell Disease Association celebrates National Sickle Cell Awareness Month

Conferences, advocacy, blood donations and other events held throughout September

HANOVER, Md.—Sickle Cell Disease Association of America will participate in National Sickle Cell Awareness Month in September by holding a series of events and supporting the events of member organizations. National Sickle Cell Awareness Month was designated by Congress to focus attention on the need for research and treatment of sickle cell disease.

“Individuals and organizations can join our efforts to bring attention to sickle cell disease by engaging elected officials for proclamations, hosting awareness events, distributing educational information to dispel the myths about sickle cell disease and lighting public spaces, buildings and landmarks red,” said Beverley Francis-Gibson, president and CEO of Sickle Cell Disease Association.

• The 2020 Sickle Cell Disease Therapeutics Conference will take place virtually Sept. 15 at 9 a.m. Hosted by Global Blood Therapeutics Inc. in partnership with the Sickle Cell Disease Association, the conference provides a forum to discuss the latest advancements and future trends for treating patients with sickle cell disease. Health care companies, opinion leaders, patients, policymakers and others will present at the conference. Learn more and register online.

• A Sickle Cell Awareness Month Twitter Party, an online Twitter conversation hosted by the Sickle Cell Disease Association where participants interact, share, learn and celebrate by posting tweets using the hashtag #SickleCellMatters, will take place Sept. 21, 23 and 25 from 12:30 to 1:30 p.m. Register online.

• Fight sickle cell disease by donating blood to the American Red Cross. Donors can make an appointment by downloading the American Red Cross Blood Donor app, visiting RedCrossBlood.org or calling 1-800-733-2767.

• Sickle cell groups across the country will hold National Sickle Cell Awareness Month events throughout September, including town halls, webcasts, walks and races, summits and seminars and fundraisers. Learn more online.

Sickle Cell Disease Association invites advocacy organizations, corporate and federal partners and supporters to use the hashtags #SickleCellMatters, #SickleCellAwarenessMonth, #SCDAA2020AwarenessMonth and #SCDSCTMatters in social media posts about sickle cell disease throughout September. More information, including a flyer and fact sheet, is available online. Sickle cell disease is an inherited blood disease causing red blood cells to take a sickle shape, which leads to blockages that prevent blood from reaching parts of the body. As a result, people with sickle cell complications can experience anemia, jaundice, gallstones, stroke, chronic pain, organ damage and premature death. No universal cure exists. (sicklecelldisease.net)

Sickle Cell Disease Association of America advocates for people affected by sickle cell conditions and empowers community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure. The association and more than 50 member organizations support sickle cell research, public and professional health education and patient and community services. (sicklecelldisease.org)

National Institutes of Health launches initiative to accelerate therapies to cure sickle cell disease

The National Institutes of Health (NIH) today announced the launch of a new initiative to help speed the development of a cure for sickle cell disease, a group of inherited blood disorders affecting at least 100,000 people in the United States and 20 million worldwide. The Cure Sickle Cell Initiative will take advantage of the latest genetic discoveries and technological advances to move the most promising genetic-based curative therapies safely into clinical trials within five to 10 years.

“Our scientific investments have brought us to a point where we have many tools available to correct or compensate for the defective gene that causes sickle cell disease. We are now ready to use these tools to speed up our quest for a cure,” said Gary H. Gibbons, M.D., director of NIH’s National Heart, Lung, and Blood Institute (NHLBI), which is leading the effort.

Sickle cell disease results from a single genetic mutation that causes a person’s red blood cells to form an abnormal, sickle shape. These sickled cells can clog the blood vessels and deprive cells of oxygen. In turn, this lack of oxygen wreaks havoc on the body, damaging organs, causing severe pain, and potentially leading to premature death.

Decades of basic research on sickle cell disease have laid the groundwork for novel genetic approaches to cures, such as the genetic editing of bone marrow cells, which have shown great promise in animal models and in some small-scale human studies. In addition, the NHLBI Production Assistance for Cellular Therapies (PACT) program has been working with researchers to manufacture cellular therapeutic products, including genetically modified cells, that can be used safely in clinical trials with patients.

NIH spends approximately $100 million on sickle cell disease research each year. Through this initiative, NHLBI seeks to support the development of cell and genetic therapies resources, clinical trials, comparator analyses of different management strategies, data repositories and resources, and patient and advocate engagement activities related to curative therapies for this condition. Already in 2018, NHLBI committed an additional $7 million to jumpstart the initiative’s research and engagement infrastructure.

NHLBI has named hematologist Edward J. Benz Jr., M.D., President and CEO Emeritus of Dana-Farber Cancer Institute, as the Initiative’s executive director, and the Emmes Corporation, a contract research organization with specialized expertise in clinical trials, gene, and cell therapy development in preclinical studies, as its coordinating center.

The initiative and other research partners will establish a national data warehouse of genetic therapies for sickle cell disease and conduct comparative analyses of therapeutic approaches to assess both clinical and cost-effectiveness. They will also establish national networks to make it easier for patients and providers to learn and engage with the research, clinical trials, and other activities happening across the country.

“The engagement of patients will be a cornerstone of this effort,” said Benz. “Patients will work alongside researchers in developing and recruiting for clinical trials.”

Currently, the only cure for sickle cell disease is a bone marrow transplant, a procedure in which a sick patient receives bone marrow from a healthy, genetically-compatible sibling donor. However, transplants are too risky for many adults, and only about 18 percent of children with the sickle cell disease have a healthy, matched sibling donor.

The Cure Sickle Cell Initiative seeks to develop cures for a far broader group of individuals with the disease, and it is initially focusing on gene therapies that modify the patient’s own hematopoietic stem cells (HSCs), which make red and other blood cells. These modified HSCs can then be given back to the patient via a bone marrow transplant, making a cure available to more patients who lack a matched donor.

“This initiative is giving patients, families, and communities a reason for hope. I’m particularly pleased that we are able to make this announcement during Sickle Cell Awareness month when we are shining a spotlight on the toll of this devastating disease,” said ADM Brett Giroir, M.D. Assistant Secretary for Health at the U.S. Department of Health and Human Services (HHS).

This patient-focused Initiative builds on the legacy of NHLBI-supported research that has contributed to improving clinical care for patients who have sickle cell disease. It also complements the Institute’s broader sickle cell disease research investment, which includes basic, clinical, translational, and implementation science research.

Part of the National Institutes of Health, the National Heart, Lung, and Blood Institute (NHLBI) plans, conducts, and supports research related to the causes, prevention, diagnosis, and treatment of heart, blood vessel, lung, and blood diseases; and sleep disorders. The Institute also administers national health education campaigns on women and heart disease, healthy weight for children, and other topics. NHLBI press releases and other materials are available online at http://www.nhlbi.nih.gov.

About the National Institutes of Health (NIH): NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit www.nih.gov.

NIH...Turning Discovery Into Health

St. Jude Receives $1Million Sickle Cell Grant

Dr. Glenda Newell-Harris, national president of The Links Foundation Inc. and The Links Inc. tours St. Jude Children's Research Hospital Thursday, May 3. The Links awarded a $1 million grant to St. Jude for sickle cell disease initiatives.

The Links Foundation, one of the nation’s oldest and largest African-American women’s volunteer service organizations, awarded a $1 million Legacy Grant to St. Jude Children’s Research Hospital Thursday with a goal of jumpstarting three critical sickle cell disease initiatives.

St. Jude has researched and treated children with sickle cell disease since opening in 1962, and today touts one of the largest programs taking care of sickle cell patients, with more than 900 children from across the region. The disease is the most commonly inherited blood disorder in the United States, affecting about 100,000 Americans. It more commonly manifests in people of African descent; Hispanics; and people of Middle Eastern, Asian, Indian and Mediterranean descent.

“We have made great progress,” said St. Jude president and CEO Dr. James R. Downing. “These children now are making it through childhood and into adulthood. Back when I was in medical school, most of them died before the age of 5. Their pain crises are less, and we’re able to prevent many of the major complications – much fewer strokes, much fewer renal failures, much fewer problems with their vision.”

The average life span today for children diagnosed with sickle cell disease is 45 years old.
In addition to eventually finding a cure, Downing hopes to see a future health care system that is more receptive to taking care of sickle cell patients once they leave St. Jude.

The executive board of The Links Foundation Inc., the philanthropic arm for The Links Inc., visited St. Jude ’s campus Thursday for the grant award.

“It’s wonderful to hear Dr. Downing say that he wants to be able to do more so that the average life expectancy can be just like everyone else,” said Dr. Glenda Newell-Harris, national president of The Links Foundation and The Links. “And it’s wonderful to be a part of an organization that wants to be a part of that revolutionary work that’s going to need to get done.”

The grant will support expansion of three St. Jude clinical efforts – studies designed to increase knowledge of cognitive deficits in children with sickle cell disease; development of a community health worker education program to counsel parents of infants with sickle cell disease in Nigeria; and an age-appropriate mobile app to help patients develop adequate self-care and disease literacy.

Read more: St. Jude Receives $1M Sickle Cell Grant.

The U.S. Food & Drug Administration Approves Endari, A New Treatment for Sickle Cell Disease

Endari has shown to decrease the number of sickle cell crises and hospitalizations in patients with sickle cell disease. SCD is a rare, debilitating and lifelong hereditary blood disorder that affects approximately 100,000 patients in the U.S. and up to 25 million patients worldwide, the majority of which are of African descent.

Emmaus Life Sciences Inc. announced today that the U.S. Food and Drug Administration (FDA) approved Endari™ (L-glutamine oral powder) to reduce the severe complications of sickle cell disease (SCD) in adult and pediatric patients age 5 and older. Endari reduces oxidant damage to red blood cells by improving the redox potential of nicotinamide adenine dinucleotide (NAD), a coenzyme that has been identified as the primary regulator of oxidation.

"The approval of Endari is a significant milestone for the sickle cell patient community who has not had an advancement in treatment for nearly 20 years and which now, for the first time ever, has a treatment option for children," said Yutaka Niihara, MD, MPH, Chairman and Chief Executive Officer of Emmaus Life Sciences. "Endari reinforces our commitment to discovering innovative therapies that help to improve the lives of people with rare diseases. We thank the FDA for its prompt review and look forward to making treatment available to patients as early as this fourth quarter."

SCD is a rare, debilitating and lifelong hereditary blood disorder that affects approximately 100,000 patients in the U.S. and up to 25 million patients worldwide, the majority of which are of African descent as well as Latinos and other minority groups. Approximately one in every 365 African American children is born with SCD and children between the ages of 2 and 7 are 400 times more likely to suffer from stroke.

Caused by a genetic mutation in the beta-chain of hemoglobin that distorts red blood cells into crescent shapes, SCD lowers oxygen levels in the blood and has an extensive impact on morbidity, mortality and quality of life. Patients often suffer from debilitating episodes of sickle cell crises, which occur when the rigid, adhesive and inflexible red blood cells block the blood vessels, resulting in excruciating pain. Sickle cell crises can lead to organ damage, stroke, pulmonary complications, and other adverse outcomes, including acute chest syndrome (ACS), which may be potentially fatal and is the leading cause of death among people with SCD.

"A sickle cell crisis is the most common acute complication for patients and the number one cause of emergency room visits," said Wally Smith, MD, Florence Neal Cooper Smith Professor of Sickle Cell Disease, Division of General Internal Medicine, Virginia Commonwealth University. "Endari has clinically shown to reduce sickle cell crises and hospitalizations, representing a significant medical advancement for patients with limited therapeutic options that have many side effects."

FDA approval was supported by efficacy data from a 48-week randomized, double-blind, placebo-controlled, multicenter Phase 3 clinical trial evaluating the effects of Endari, prescription grade L-glutamine, as compared to placebo on 230 adults and children with SCD. The results demonstrated that Endari reduced the frequency of sickle cell crises by 25 percent and hospitalizations by 33 percent. Additional findings showed a decrease in cumulative hospital days by 41 percent and lower incidence of ACS by more than 60 percent.

Safety was based on data from 298 patients treated with L-glutamine and 111 patients treated with placebo in the Phase 2 and Phase 3 studies. Endari's safety profile was similar to placebo and well-tolerated in pediatric and adult patients. The most common adverse reactions occurring in greater than 10 percent of patients treated with Endari were constipation, nausea, headache, abdominal pain, cough, pain in extremity, back pain, and chest pain (non-cardiac).

About Emmaus Life Sciences

Emmaus Life Sciences is engaged in the discovery, development and commercialization of innovative treatments and therapies for rare diseases. The company's research on sickle cell disease was initiated by Yutaka Niihara, MD, MPH, Chairman and CEO of Emmaus, at the Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center. For more information, please visit http://www.emmauslifesciences.com.

Forward-Looking Statements

This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, regarding the research, development and potential commercialization of pharmaceutical products. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could delay, divert or change any of them, and could cause actual outcomes and results to differ materially from current expectations. Additional risks and uncertainties are described in reports filed by Emmaus Life Sciences, Inc. with the U.S. Securities and Exchange Commission, including its Annual Report on Form 10-K and Quarterly Reports on Form 10-Q. Emmaus is providing this information as of the date of this press release and does not undertake any obligation to update any forward-looking statements as a result of new information, future events or otherwise.

SOURCE Emmaus Life Sciences Inc.

Related Links: http://www.emmauslifesciences.com