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Feb 17, 2014
 |  BioCentury  |  Regulation

Sickle cell suffering

Sickle cell patients ask FDA for drugs that change course of the disease

There are two key takeaways from FDA's fifth Patient-Focused Drug Development workshop. The first is that there are opportunities for drugs that treat debilitating chronic effects of the disease, which are not the primary focus of current development programs. The second is that patients want better information about potential risks and benefits of participating in trials.

Sickle cell disease is a hereditary disorder in which red blood cells make abnormal hemoglobin that can distort them into a sickle shape. Symptoms and severity vary widely, even for patients with similar genotypes.

Sickled cells have shortened lifespans, leading to anemia, and can clump in small blood vessels, causing pain, hypoxia and tissue damage. Vessel blockage can lead to an acute pain crisis (vaso-occlusive crisis) that requires hospitalization and narcotics, and can take days to resolve.

Common treatments include hydroxyurea, transfusions and bone marrow transplant.

Only bone marrow transplant is potentially curative. It requires a suitable donor and can lead to graft-versus-host disease (GvHD) or infections.

At FDA's meeting on Feb. 7, some panelists said hydroxyurea reduced their frequency of hospitalization, while others were non-responders or thought the risks outweighed the benefits for their symptom severity.

Hydroxyurea, a generic antineoplastic agent also used to treat cancers, causes side effects including neutropenia, nausea, skin ulcers and blackening of finger- and toenails.

Transfusions were common among the patients, but some had bad reactions to them after developing antibodies, which also made it difficult to find suitable blood.

Transfusions also necessitated use of iron chelators, such as Novartis AG's Exjade deferasirox. Some patients found the chelators helpful, but others discontinued them because of hallucinations or hearing loss.

Patients also reported use of over-the-counter pain medicines, extra fluids, heat or massage therapy, vitamins, dietary supplements or herbal remedies.

In an interview to be broadcast on BioCentury This Week television on Feb. 23, Kim Smith-Whitley, CMO of the Sickle Cell Disease Association of America Inc., said available treatments may reduce pain but don't alter the course of an acute crisis.

"It would be better if we had treatments that were more focused on treating the underlying...

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