The American Society of Hematology (ASH) is launching a sickle cell disease (SCD) clinical trials network in order to accelerate the development of new therapies for a patient community that has very few treatments and curative options.

The core activities of the network will include matching clinical trial sponsors with research sites, facilitating patient recruitment, and improving the efficiency of clinical trials by advising on optimal trial design and ensuring a coordinated approach across the network. The first study is expected to be initiated in the fall of 2019.

The network will be led by Charles "Chuck" Chesson, PhD, who was named as director in July of 2018. Dr. Chesson comes to ASH after nearly two decades at the public health research organization Social and Scientific Systems, where he most recently served as Group Vice President, Clinical Research and Bioscience.

One of the most common complications of sickle-cell disease occurs when deformed red blood cells clump together, blocking tiny blood vessels and causing severe pain and swelling in the affected body parts.

A new study from MIT sheds light on how these events, known as vaso-occlusive pain crises, arise. The findings also represent a step toward being able to predict when such a crisis might occur.

“These painful crises are very much unpredictable. In a sense, we understand why they happen, but we don’t have a good way to predict them yet,” says Ming Dao, a principal research scientist in MIT’s Department of Materials Science and Engineering and one of the senior authors of the study.

The researchers found that these painful events are most likely to be produced by immature red blood cells, called reticulocytes, which are more prone to stick to blood vessel walls.

National Sickle Cell Disease Awareness Month is a time to acknowledge the challenges faced by Americans with sickle cell disease (SCD) and to reaffirm our commitment to increasing the quality, affordability, and accessibility of different treatment options.

SCD is an inherited red blood cell disorder that is most prevalent among those who have ancestors from sub-Saharan Africa and certain regions of South and Central America. In the United States, SCD occurs in 1 out of every 365 African-American births and 1 out of every 16,300 Hispanic-American births.

Beginning in early childhood, people with SCD experience a lifetime of crippling pain, challenging disabilities, and often premature death. The Centers for Disease Control and Prevention estimates medical expenses of children with SCD average between $11,000 and $14,000, with nearly 40 percent of these patients requiring at least one hospital stay.

In a continuing effort to spur advancements in treating and curing sickle cell anemia, the Doris Duke Charitable Foundation today announced a grant to the Critical Path Institute (C-Path) to lay the groundwork for forming a collaboration of multiple stakeholder organizations to accelerate the development of therapies for the disease.

With the funding, C-Path will conduct the planning necessary to bring together scientists, clinicians and representatives from the biopharmaceutical industry, governmental regulatory agencies, academic institutions and patient advocacy organizations to identify and work collaboratively toward solving significant challenges affecting the entire research and development community.

The anticipated outcome is a consortium model engaging potential partners to work on jointly beneficial drug development tools and novel methodologies, submit them to the regulatory agencies for endorsement, and then make them available to the broader research community.

Medunik USA announced today that Siklos® (hydroxyurea), the first and only hydroxyurea-based treatment FDA-approved for pediatric patients with sickle cell anemia, is now available through the Siklos At Home™ program.

"The recent launch of Siklos® represents an important milestone in Medunik USA's mission to support patients with rare diseases - in this case, sickle cell anemia," said Dean Hopkins, the company's General Manager. "We are looking forward to introducing this treatment to pediatric patients with sickle cell anemia and committed to providing Siklos® at the lowest possible cost to all patients, whether they are cash-paying or have commercial or Medicaid healthcare coverage, through the Siklos At Home™ program, offered through our specialty pharmacy, ProCare Pharmacy Care."

Through Siklos At Home™, commercially insured patients can receive Siklos® for as little as $0 copay per prescription. Patients who are not insured or whose insurance does not cover Siklos® can pay as little as $99 for 60 tablets or $149 for 90 tablets.

Hemoglobinopathies (Sickle Cell Disease (SCD) & Sickle Cell Trait) have the highest overall birth prevalence of genetic conditions screened for during universal newborn screening. There are 80 babies born with SCD each year in Illinois (IL). There are about 5,000 individuals with SCD in IL yet access to quality care is not universal. Let's Talk!

The schedule of event is as follows:

When:

Wednesday; September 19, 2018 from 6:30 PM to 8:30 PM CDT

Where:

South Holland Community Center 

501 E. 170th Street

South Holland, IL, USA

This event has ended.

Sickle cell trait may not be an independent genetic risk factor for ischemic stroke among African Americans, a new meta-analysis suggests.

In a review of four studies with more than 19,000 African-American participants, Hyacinth I. Hyacinth, MD, PhD, MPH, of Emory University School of Medicine in Atlanta, and colleagues reported no association between heterozygosity for the sickle cell mutation or sickle cell trait and incidence of ischemic stroke in JAMA Neurology.

"Sickle cell trait was not associated with a higher risk of ischemic stroke among African Americans," Hyacinth told MedPage Today. "This negative study and its findings are important as we attempt to develop more targeted approaches for stroke prevention."

"Significant stroke disparity exists between African Americans and non-Hispanic whites," he said. "Because this disparity is not explained by traditional risk factors such as access to healthcare, identifying biological factors that account for the differences is an important strategy for stroke prevention."

Six months of hydroxyurea therapy detrimentally effected spermatogenesis in men with sickle cell anemia, according to a small prospective study published in Blood.

“Hydroxyurea, at current doses, causes significant, rapid and unpredictable impairment of spermatogenesis in treated men,” Isabelle Berthaut, PhD, professor of histology and biology of reproduction at Tenon Hospital in Paris, and colleagues wrote.

Hydroxyurea — approved by the FDA in 1998 for the treatment of sickle cell anemia — helps reduce the production of sickle hemoglobin. Its side effects include low blood counts, gastrointestinal symptoms and loss of appetite.

Three previous retrospective studies showed an association between hydroxyurea and potentially detrimental effects on sperm parameters in men with sickle cell anemia. However, those observations occurred on studies involving as few as eight men, seven of whom did not have any comparative semen assessment prior to hydroxyurea initiation.

The Sickle Cell Disease Association of Illinois (SCDAI’s) Annual Management of Sickle Cell Disease Conference is designed for nurses, health care professionals, providers, educators, social workers, students, individuals with sickle cell disease and their families and the community at large. The purpose of this educational activity is to strengthen participant’s knowledge base about sickle cell disease and sickle cell trait through lecture, discussion and sharing of current and future treatment regimen. 

Upon completion of this activity, participants should be able to:

• identify current evidence-based practices to improve care of individuals with Sickle Cell Disease

• discuss complications related to Sickle Cell Disease

• explain resources available to individuals with Sickle Cell

When:

Friday, November 9, 2018 from 7:00 AM to 4:00 PM CDT

Location:

The Loretto Hospital 

645 S Central Ave, Chicago, IL, USA

This year’s event has ended. Stay tuned for more information about next year’s event.

Emmaus Life Sciences, Inc.  announces it has entered into a distribution agreement with Cardinal Health (Cardinal). The agreement makes Endari™ (L-glutamine oral powder), indicated to reduce the acute complications of sickle cell disease in adult and pediatric patients 5 years of age and older, available nationwide to pharmacies that utilize Cardinal as their distributor. Cardinal Health connects patients, providers, payers, pharmacists and manufacturers for integrated care coordination and better patient management. Cardinal Health ranks among the top 25 on the Fortune 500.

Since launching Endari, the first U.S. FDA approved sickle cell treatment in 20 years, Emmaus has brought this therapy to patients in 36 states, Washington DC and Puerto Rico. In addition to the distributors already in place, this agreement with Cardinal will further broaden Endari’s distribution, with the potential to reach many more patients suffering from the acute complications associated with sickle cell disease, such as pain and frequent hospitalizations.