For Patients With Rare Blood Cancer, New Drug Offers Relief

For patients with a rare type of blood cancer, treatment might finally be coming out of the Dark Ages.

People with the chronic condition polycythemia vera make too many red blood cells, thickening their blood and increasing the risk for clots, heart attacks or strokes. The main treatment consists of regular blood draws—essentially bloodletting—to keep the disease in check. But a new drug from Protagonist Therapeutics and Takeda can slow down a patient’s production of red blood cells and nearly eliminates the need for phlebotomies, new data show, potentially reducing patients’ fatigue and other consequences of routine blood draws.

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Some 50 per 100,000 people in the U.S. live with polycythemia vera, according to the Cleveland Clinic. Most patients are diagnosed after age 60 and harbor a genetic mutation in a gene called JAK2. The biggest health risk is life-threatening blood clots. But it can progress into aggressive blood cancers in rare cases, and patients tend to have high rates of secondary cancers including skin cancer, data show.

Quarterly, monthly or sometimes weekly blood draws are the most common treatment, along with a daily low dose of aspirin. There is a handful of medications including chemotherapy and a drug called ruxolitinib for patients with higher-risk disease, and some patients still need phlebotomies in addition to those medicines. The blood draws can exacerbate iron deficiency, starving the bone marrow of an essential ingredient it needs to make red blood cells. That can also create a host of complications including debilitating fatigue and the regular hassle of going to the clinic.

The new drug rusfertide uses a novel strategy, mimicking a hormone called hepcidin that regulates how a person’s body uses iron. The drug helps block the iron from reaching the bone marrow where red blood cells are made, slowing down their production. It also allows the patient to build up their iron and start to feel better, the companies said.

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Between weeks 20 and 32 of the trial, some 77% of patients taking rusfertide had a clinical response and weren’t eligible for a phlebotomy, compared with 33% of patients in the control group. In the entire 32-week span, the average number of phlebotomies needed among those getting the drug was 0.5, compared with an average of 1.8 blood draws for those not taking it.

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After the 32 weeks, patients in the placebo arm were able to switch and start taking the drug. The trial will continue to follow the patients for up to three years, to track rusfertide’s longer-term efficacy and safety. The 52-week data is expected later this year, and Takeda plans to file for Food and Drug Administration approval after that, said Dr. Phuong Khanh Morrow, head of the oncology therapeutic area at Takeda.

https://www.wsj.com/health/healthcare/polycythemia-vera-red-blood-cells-treatment-bed50266?st=ZuNFnM&reflink=desktopwebshare_permalink

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