The California Institute for Regenerative Medicine (CIRM) board awarded $5.74 million to City of Hope to fund a phase 1 clinical trial testing a novel blood stem cell transplantation procedure for adult patients with severe sickle cell disease (SCD).

The treatment involves induction of mixed chimerism. This refers to transplanting blood-forming stem cells from a healthy half-matched family member donor into a patient who has received a milder, less toxic chemotherapy treatment that removes some but not all of the patient’s diseased bone marrow stem cells. The donor blood stem cell transplants are depleted of some immune cells called CD4+ T cells prior to transplantation. This approach allows the donor stem cells to engraft and create a healthy supply of non-diseased blood cells without causing graft-versus-host disease, a side effect of bone marrow transplantation.

The US Food and Drug Administration (FDA) has approved hydroxyurea tablets (Siklos, ADDMEDICA) for the treatment of sickle cell anemia in pediatric patients aged 2 years and older.

Previously, the hydroxyurea tablets were granted priority review and orphan drug designations from the FDA. Sickle cell anemia, by recent estimates, impacts roughly 100,000 Americans.

The approval was granted based on efficacy and safety data from the Escort-HU study, which included 1047 patients with sickle cell anemia (495 children). The mean hydroxycarbamide levels in the pediatric patients at initiation was 18 mg/kg and 17.3 mg/kg.

This one day 2018 Indiana Sickle Cell Disease Conference was held on April 20, 2018 to provide information about current trends in the field and available resources for healthcare providers, patients, family members, care givers, and social services providers. This activity was planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of Indiana University School of Medicine and Martin Center Sickle Cell Initiative. The Indiana University School of Medicine is accredited by the ACCME to provide continuing medical education for physicians.

This year also, Sickle Cell Disease conference 2019 will be organized by The Martin Center on April 26, 2019.

At the conclusion of this activity, participants should be able to:

• Recognize recommended protocols for treating adult Sickle Cell patients in pain crisis episodes and be able to apply them for improved patient outcomes
• Identify proven methods for effectively transitioning adolescent patients to adult care and apply them for improved patient outcomes
• Describe indications for stem cell transplantation and the benefits for improved patient outcomes

Event start time: 8:30 am
Event end time: 4:30 pm
Phone: 317-927-5158

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Sickle Cell Disease Association, Philadelphia/Delaware Valley Chapter is organizing sickle cell awareness forum on Saturday (April 7 at 10 AM - 2 PM EDT).

Keynote Speakers:

Dr. Roy Gay Hematologist/Oncologist/Internal Medicine Dr. Marjorie Dejoie-Brewer, Medical Director (SCDAA/PDVC)

Sickle cell panel presentation:

Patient stories Community Resources / Health Information,and much more!

Date & Venue:

Saturday, April 7 at 10 AM - 2 PM EDT

125 S 52nd St, Philadelphia, PA 19139-3408, United States

We hope you will join us for this exciting, free and informative event.

Please RSVP to: Dr. Vera Tolbert 215-680-0722, Zemoria Brandon 267-241-5338, Aminata Sidibe 267-210-4555 or bareitu@gmail.com

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This biennial educational conference focuses on the management of adults with sickle cell disease. Experts will review the latest research in sickle cell disease and will discuss management of the most common complications in adolescents and adults with sickle cell disease. Speakers will also discuss emergency room management, transition from pediatric to adult care, new therapies for pain management, gene therapy, and the major treatment modalities for adults with sickle cell disease.

Date & Time: Saturday, May 19, 2018 8:00 AM - 6:00 PM

Location: Marlo Thomas Center Auditorium, St. Jude Children's Research Hospital, Memphis, TN

Target Audience: Hematology, Nursing, Physician Assistant, Social Work
Additional Target Audience: This conference is open to all healthcare providers, social workers, counselors, and others who provide care for those with sickle cell disease as well as consumers and caregivers.

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Red blood cells are on a wild ride. As they race through the body to deliver oxygen, they must maintain a distinct dimpled shape—and bounce back into form even after squishing through narrow capillaries. Red blood cells that can’t keep their shape are associated with diseases like sickle cell anemia.

In a new study, Velia Fowler, PhD, and her lab at The Scripps Research Institute report that a protein called myosin IIA contracts to give red blood cells their distinctive shape. The findings, published this week in the journal Proceedings of the National Academy of Sciences, could shed light on sickle cell diseases and other disorders where red blood cells are deformed.

CIRM – California's Stem Cell Agency – is hosting a special ‘Ask the Expert’ event on Facebook Live. The topic is Stem Cells and Stroke.

Just head over to our Facebook Page (California Institute for Regenerative Medicine) on May 31st from noon till 1pm (PDT) to experience it live. 

We will be joined by Dr. Gary Steinberg, chair of neurosurgery at Stanford University, who will talk to us about his work in helping reverse the damage caused by a stroke, even for people who experienced a brain attack several years ago.

CIRM Senior Science Officer, Dr. Lila Collins, will talk about other stem cell research targeting stroke, its promise and some of the problems that still need to be overcome.

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A Britain's Got Talent contestant whose choir moved judges to tears during their audition has revealed how she turned to singing following the death of her six-year-old son.

Simonne Kerr, 31, of London, joined NHS choir B Positive after losing her son Kavele to complications arising from sickle cell disease, an inherited blood disorder, in 2015.

The choir was created by NHS Blood and Transplant to raise awareness of blood donation and to bring together people whose lives are affected by the 'lifesaving power of blood', including medics, donors and patients with blood conditions.