Home2020January 2020 -

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FDA-approved therapies for sickle cell disease (SCD) may not be cost-effective at their current prices, according to a draft report issued by the Institute for Clinical and Economic Review (ICER). The report focused on the clinical benefits and cost-effectiveness of Novartis’ Adakveo (crizanlizumab), Global Blood Therapeutics’ Oxbryta (voxelotor) — both approved in the U.S. last year — and Emmaus Life Sciences’ Endari (L-glutamine). At their current estimated costs, all three medications exceed $1 million per...

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Jasper Therapeutics has raised an additional $14.1 million to finance the development of its investigational conditioning therapy, JSP191, as a safer and more effective treatment to prepare patients for stem cell transplant. The investment, led by Roche Venture Fund and sponsored by other investors, increases the company financing to more than $50 million, the company stated in a press release. JSP191 (formerly AMG191) is designed to replace toxic chemotherapy and radiation therapy as conditioning regimens...

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A potential gene therapy for sickle cell disease (SCD) and beta-thalassemia, known as ARU-1801, has been named an  orphan drug by the U.S. Food and Drug Administration (FDA), a status that helps to advance and support its development. The FDA recently also designated ARU-1801 a potential rare pediatric disease treatment for these same two blood disorders, allowing for priority review. “We are excited to build on the momentum afforded by both today’s announcement of Orphan Drug status as...

Sickle cell disease is a condition that mostly affects people from an African or African-Caribbean origin. Nevertheless, the majority of those who have treated me for this condition have been healthcare professionals of other races. I don’t think they would have typically interacted with sickle cell patients prior to studying in their field. I can count the number of black doctors who have consulted me on one hand, and none of them were specifically hematologists....

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FT-4202, FORMA Therapeutics’ experimental disease-modifying therapy for sickle cell disease (SCD), shows a favorable safety and pharmacokinetic profile in healthy volunteers, according to data from a Phase 1 clinical trial. Patient recruitment is open for the second part of the trial (NCT03815695). Information about trial locations, all in the U.S., is available here. Trial findings were presented in an oral presentation, “Phase 1 Single (SAD) and Multiple Ascending Dose (MAD) Studies of the Safety, Tolerability,...

For most of his 39 years, Charles Hough lived with extreme pain caused by sickle cell disease, which dramatically reduced blood flow throughout his body. But thanks to the gene therapy he received in 2017, Hough is now symptom-free. “I feel like I have a new chance at life, a healthy, full life without any complications,” Hough said in a recent webinar organized by the National Organization for Rare Disorders (NORD) and the American Society for Cancer and...

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I previously shared some tips for managing sickle cell. If you found those useful, I have a few more suggestions that also may help. Deep Heat I find that when I am in pain, balms such as Mentholatum Deep Heat help. Balms that contain ingredients such as menthol and methyl salicylate are counterirritants and have pain-relieving properties. Once applied, the change in temperature triggers the dilation of blood vessels and promotes blood flow. Balms can...

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To help empower individuals living with sickle cell disease (SCD), biopharmaceutical company Emmaus Life Sciences has teamed up with national specialty pharmacy US Bioservices on a treatment adherence program for patients. Through a team of clinicians, the new opt-in enhanced nursing program offers patients information and real-time assistance with their specific SCD therapy. In general, a treatment adherence program is a care system in which pharmacies have a more consultative role in helping patients to...

The U.S. Food and Drug Administration (FDA) granted rare pediatric disease designation to ARU-1801, Aruvant’s experimental gene therapy for the treatment of sickle cell disease (SCD) and beta-thalassemia. This designation means the company will be eligible to receive a priority review voucher once the FDA approves a biologics license application for ARU-1801. ARU-1801 uses a proprietary technology that is intended to increase the number of functional red blood cells by inserting a modified version of...