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.

Emmy Award-winning NFL broadcaster Solomon Wilcots and Emmaus Life Sciences, Inc. today announced the launch of Sideline Sickle Cell, an educational campaign designed to raise awareness and drive discussion about recent and future treatment innovations for sickle cell disease (SCD) among physicians, patients and their family members as well as other members of the SCD community in the United States.

The campaign launch, which coincides with the start of National Minority Health Month, will include a national media tour led by Wilcots followed by a series of local market media relations activities and town hall-type meetings that bring together members of the disease community. Sideline Sickle Cell event markets will include Los Angeles, New York, Miami, Atlanta and others as the campaign rolls out in 2019. People can follow the campaign and join the conversation by connecting with @EmmausLifeSci on Twitter and using the hashtag #SidelineSickleCell.

Arianne Clark had a layperson’s knowledge of weight cutting early last summer. She knew it was an unhealthy practice, one her sons — both MMA fighters — took part in.

“I knew that it was dangerous to cut weight,” Clark told MMA Fighting. “I think a lot of people know it’s dangerous to cut weight. You’re not supposed to lose so much weight in a short amount of time. I did not know it was deadly, no.”

Rondel Clark, a Sutton, Mass., native, competed in his second amateur MMA fight on Aug. 12, 2017. He cut weight leading into that bout to make weight. Three days after he left the cage, Rondel was dead at the age of 26.

Later, Arianne found out that Rondel’s kidneys failed due to severe dehydration, leading to his entire system shutting down. Rondel had the sickle cell trait, which Arianne learned accelerated his deterioration. An autopsy concluded that Rondel officially died from extreme rhabdomyolysis, the rapid breaking down of muscle tissue sometimes caused by physical overexertion combined with dehydration.

Modus Therapeutics is going to launch a Phase 1 clinical study in the United States to evaluate the potential of its lead candidate sevuparin for the treatment of sickle cell disease (SCD).

In the trial, the company will broaden the clinical scope and usefulness of sevuparin to treat sickle cell disease by testing its safety when administrated subcutaneously (under the skin).

This follows the acceptance by the U.S Food and Drug Administration (FDA) of an investigational new drug (IND) application for sevuparin.

Modus Therapeutics is currently evaluating the therapeutic activity of sevuparin to manage painful crisis, also known as acute vaso-occlusive crisis (VOC), in patients with sickle cell disease in an ongoing Phase 2 study (NCT02515838).

While parents are often the primary caregivers for their children, especially when he or she lives with a chronic condition, grandparents can also provide a leading role to help the grandchild once he or she has been diagnosed with an incurable disease. For Nancy Rene, whose grandson has sickle cell disease, she quickly learned the benefits her family could experience through her advocacy and encouragement. Join us for this empowering webinar for grandparents to discover how they can engage in their grandchild's pain journey. 

About the Presenter: Nancy Rene spent her career in education. She has served as a teacher of mathematics, school administrator, and math consultant for 36 years. In retirement, she works with various advocacy organizations, including Family Voices of California and Chronic Care Coalition of California. Nancy has served as a member of the Board of the Sickle Cell Disease Association of America. She regularly attends conferences on Sickle Cell Disease and is a Patient Advocate for the California Institute for Regenerative Medicine.

When:

Monday, November 19, 2018

This event has ended.

Technology takes center stage in a new epidemiologic study of acute vaso-occlusive pain associated with menstruation in women with sickle cell disease (SCD). Deva Sharma, MD - who completed her hematology-oncology fellowship at Vanderbilt University School of Medicine and is currently a transfusion medicine fellow at Vanderbilt University Medical Center - is spearheading an initiative to understand the biology behind the severe, debilitating pain experienced by women with sickle cell disease during menstruation.

And she’s doing it in real-time with a little help from the iPod Touch!

Sharma is a 2017 recipient of the Foundation for Women & Girls with Blood Disorders Research Fellowship Award “Promoting XXcellence in Women’s Health: Optimal Management of Women and Girls with Blood Disorders.”

A young Matteson girl who is battling sickle cell disease held a party Saturday for children just like her and an opportunity for people to give blood.

The second annual Sickle Cell Anemia Blood Drive and Celebration celebrated Olivia Shorter's 8th birthday. She was was born with the disease.

Last year, Olivia asked her parents about having a party for other children like her.

"I'm proud, so proud. And shocked she would come up with something so big and she didn't even know it," said mom Danielle Shorter. "It's just something that came from her heart."

In addition to having fun and enjoying the party, guests also donated blood to the American Red Cross.

Medical cannabis use is relatively common among patients with sickle cell disease, according to survey data published in the journal Cannabis and Cannabinoid Research.

Investigators at Yale University conducted an anonymous questionnaire among 58 patients with the disease. Forty-two percent of respondents reported having used cannabis within the past two years, and 31 percent reported having used it within the past month.

Most respondents said that they did so for therapeutic purposes. Common symptoms of sickle cell disease include pain, fatigue, and swelling of the hands and feet.

Many respondents acknowledged that the use of cannabis “allowed for less use of pain medications” – a finding that is consistent with reports from other cohorts of chronic pain patients.

To date, the use of medicinal cannabis has not been evaluated in controlled settings for patients with sickle cell disease.

Approved by the US Food and Drug Administration (FDA) in July 2017 to reduce the acute vaso-occlusive pain complications of sickle cell disease, L-glutamine oral powder (Endari) was the first drug approved for the rare blood disease in the 20 years since the approval of hydroxyurea.

Now, the New England Journal of Medicine (NEJM) has published the 48-week phase 3 clinical trial results that supported the FDA approval. From the data acquired in the phase 3 trial, investigators concluded that the median number of pain crises in children and adults with sickle cell disease was lower among those who received oral therapy with l-glutamine, administered alone, or with hydroxyurea, compared with those who received placebo, with or without hydroxyurea.

“The idea that this [publication] comes a year after [approval] really reflects the vigor of the review process and shows NEJM really did its job of assuring the public interest that L-glutamine, along with the findings of the study, are real and significant,” Darrell W. Harrington, MD, MACP, chief medical officer at Emmaus Life Sciences, told Rare Disease Report®.