Conference Coverage

Researchers seek more sickle cell drug research


 

REPORTING FROM SICKLE CELL IN FOCUS

Financial interests

Other drugs in testing for SCD include rivipansel from GlycoMimetics, for which Pfizer is leading development. The sponsors expect to complete the so-called RESET trial by 2019, according to the clinicaltrials.gov. In this study, which is intended to enroll 350 participants, patients who have vaso-occlusive crises are randomly selected for treatment with either rivipansel or placebo.

Speaking during a question-and-answer session at the NIH conference, Robert Swift, PhD, said there’s a need for inexpensive oral drugs to treat SCD. Many other options will remain beyond the finances of people living in poor countries, he said.

“We need to focus not only on the root cause, but on something that is oral and inexpensive to solve the greater sickle cell problem,” Dr. Swift said.

Large drugmakers already have hospital-based sales forces, making SCD drugs administered in this setting attractive to them, he said.

“This is partly about where money is. The drug companies are going where the money is. It’s not oral drugs to treat everybody, it’s something else,” Dr. Swift said. “So someone else is going to have to fund the basic research” into treatments that could be more broadly used.

Dr. Swift said in an interview that he has received NIH funding for developing SCD-101, an oral drug, for which a placebo-controlled crossover study is underway.

Presenters at the NIH conference, including Dr. Saunthararajah, expressed frustration about what they see as relatively little work being done on SCD despite decades of knowledge about the root causes. Like Dr. Swift, he criticized the approach taken in selecting which treatments advance in this field.

“It’s not being driven by what is the most cost effective, what the patients need the most,” Dr. Saunthararajah said. “It’s driven by what will make the most money, not just for [the] drug company, but also for the hospital and also for the physicians.”

Dr. Saunthararajah reported having patents and patent applications around decitabine/tetrahydrouridine, 5-azacytidine/tetrahydrouridine, and differentiation therapy for oncology. He has also been a consultant for EpiDestiny, Novo Nordisk, and Takeda Oncology. Dr. Williams reported having no relevant financial disclosures. Dr. Swift is a managing member of Invenux and reported equity in Mast Therapeutics and SCD Development.

This article was updated on 11/9/2018.

Pages

Recommended Reading

Guidelines cut acute chest syndrome hospital returns in pediatric sickle cell
MDedge Pediatrics
Emerging sickle cell agents target new pathways
MDedge Pediatrics
Clinic eases pediatric-adult transition in sickle cell disease
MDedge Pediatrics
FDA approves hydroxyurea for pediatric patients with sickle cell anemia
MDedge Pediatrics
FDA grants breakthrough therapy designation for severe aplastic anemia drug
MDedge Pediatrics
Children with sickle cell anemia fall short on antibiotic adherence
MDedge Pediatrics
Too few Michigan children with SCD receive pneumococcal, meningococcal vaccines
MDedge Pediatrics
FDA approves long-acting ESA for dialysis-related anemia in children, adolescents
MDedge Pediatrics
Integrated pain program reduced LOS for sickle cell patients
MDedge Pediatrics
Few clinical outcomes convincingly linked to sickle cell trait
MDedge Pediatrics