100 Years But Only One Drug: Sickle Cell Patients Wait For Help

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the state Medicaid programs will no doubt weigh the savings of a cure against what the drug companies decide to charge.

What will those savings be? It’s not entirely clear. But a 2009 study tabbed the costs sickle cell treatments–culled from 2001-2005 Florida Medicaid data—at $892 a month for young children up to $2,562 per month for people from 50 to 64 years old. The average was $1,389 per month. Three years earlier, this study estimated that in 2004, 113,000 hospitalizations for sickle cell disease in the U.S. cost nearly half a billion dollars.

The health economics disparities within the U.S. pale next to those between the U.S. and regions like Africa and India, where sickle cell disease takes a huge toll.

For example, there are roughly 100,000 people with the disease in the U.S.; more than 100,000 babies are born with the disease every year in Nigeria alone, the highest burden in the world, according to the U.S. Centers for Disease Control.  (Having sickle cell trait helps protect against malaria, which explains why so many people in areas with endemic malaria survive to pass on the gene.)

A biopharma veteran says another hurdle could be difficulty getting people to join trials. For good reason, African Americans have historically been suspicious of the medical community. Checkmate Pharmaceuticals CEO Art Krieg, who has had tours of duty with several companies, remembers looking at sickle cell while at Rana Therapeutics and at Pfizer (NASDAQ: PFE). “From a scientific point of view we liked it,” he says, but clinical and commercial questions blunted the companies’ enthusiasm. “We didn’t get too far.”

(Krieg has worked for years on the problem of delivering RNA-based drugs into cells, which CRISPR/Cas9 companies will need to solve. He is a scientific advisor to CRISPR/Cas9 developer Intellia.)

Krieg also notes that hydroxyurea, while it doesn’t work for everyone, has gone generic. Insurers will have to be convinced that new, pricey therapies will not just be marginally better than existing treatments, including bone marrow transplant.

Banks stresses that sickle cell disease (sometimes still called “sickle cell anemia”) is complicated. A patient’s genetic variant doesn’t always line up with the severity of his or her disease. There’s no easy way to say, in advance, who might be eligible for a future gene therapy. Jacob Corn says if the IGI program, using CRISPR/Cas9 to edit blood stem cells, gets to humans, the plan is to start with those who’ve already had a stroke or who’ve had a lot of pain crises or acute chest syndrome. “We’d like to start with people who are already ill and reverse their disease,” he says.

If he’s still saying that in two months, it’ll be a good step forward.

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