Global Blood Therapeutics, Inc. today announced the completion of a planned review of Part A of the Phase 3 HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization) Study, which is evaluating voxelotor for the treatment of sickle cell disease (SCD). On the primary endpoint (the proportion of patients with greater than 1 g/dL increase in hemoglobin versus baseline), a statistically significant increase was demonstrated with voxelotor at both the 1500 mg and 900 mg doses after 12 weeks of treatment versus placebo.

The independent Data and Safety Monitoring Board (DSMB) completed its most recent clinical safety review in May 2018 and did not identify safety concerns with voxelotor at either dose level across all patients dosed in the ongoing SCD clinical program, including adult patients in the Phase 3 HOPE Study and adolescent patients in the HOPE-KIDS 1 (GBT440-007) Study. The DSMB supported the initiation of dosing in children as young as age 4.

Crispr Therapeutics AG plunged after the gene-editing company said a planned trial of its treatment to help people with sickle-cell disease was being put on hold by U.S. regulators.

Crispr is developing the therapy with Vertex Pharmaceuticals Inc., and the trial had yet to begin. The companies said in a statement Wednesday that the Food and Drug Administration placed a halt on the experiment until the agency could get more information from the companies.

There are unexpected things that happen that you really can’t prepare for.

Away from home for the first time attending a two-week summer camp, Johanne Konan was expecting a big hug from her two daughters – Elisa and Ines -- on their return.

“When the bus pulled up, I thought they would have come running to me, saying ‘We missed you mom and we love you’,” said the Ivory Coast immigrant. “Instead, the first words out of their mouths were, ‘Can we go back’. I was stunned.”

After the initial shock wore off, Konan and George Ofori were relieved to know that their girls – they were born with sickle cell – relished their time at Camp Jumoke.

Domino's has lost its O's. No A’s or O’s for the New York Yankees. No B in IBM?

Major brands are losing the letters A, B and O in their logos to support the Red Cross’s new blood drive starting Monday.

“Without A, B and O, we can’t save anybody,” the Red Cross says. “Each year, fewer and fewer people are donating blood.”

The Red Cross is always short of blood and looking for new ways to get people to donate. Only about 38 percent of the population is even eligible to give blood, and only about 10 percent of those people — that’s 3 percent of the population — give blood yearly.

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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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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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.

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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