Bluebird could be on the way to a cure for sickle cell disease

  • Bluebird Bio presented promising results at the American Society of Hematology Annual Meeting.
  • Its sickle cell treatment essentially restores a patient's cells and allows the person's body to produce the appropriate protein for life, CEO Nick Leschly told CNBC, referring to early stage testing.
  • Because gene therapy is an emerging treatment option, Leschly says an appropriate payment system hasn't been developed.

One pharmaceutical company may be heading toward a cure for sickle cell disease.

Bluebird Bio presented promising results for its experimental gene therapy drug at the American Society of Hematology Annual Meeting. Its LentiGlobin treatment is still in the early stages of a clinical study, but the data showed encouraging signs to fix a debilitating disease.

Sickle cell disease is a genetic disorder that slows or stops blood flow. Bluebird's treatment essentially restores a patient's cells and allows the person's body to produce the appropriate protein for life, CEO Nick Leschly explained to CNBC on Monday.

One patient apparently went for a three-mile run after taking the treatment, Leschly said, which people with sickle cell disease normally can't do. Despite the early indications, Bluebird is cautious in calling the drug a cure so it doesn't promise too much "because patients are sitting there really looking for options," he said.

"So we're a little bit careful with that, but that's absolutely within the reach. It is definitely a potential," he said.

Because gene therapy is an emerging treatment option, Leschly says an appropriate payment system hasn't been established. Today, patients typically pay for multiple drug doses over a long period of time, Leschly said. Bluebird's sickle cell disease drug would differ because it's a one-time treatment. That could produce a sense of sticker shock.

To combat that, Leschly suggests moving toward a value-based system where multiple parties share the risk over time. If the treatment works, they pay. If it doesn't, they don't.

"We're talking about doing it one time now, so it's perhaps less about that one-time, up-front payment and more about how we do this over time and sharing that risk over time, then I think you can get out of that sticker shock," he said. "But it is a challenge for the system, and the system's not ready, we'll freely admit that."

Politicians, including President Donald Trump, have assailed the pharmaceutical industry for charging high prices.

Bluebird will consider cost and access when pricing the drug, Leschly said, noting that a large population of sickle cell patients are insured through Medicaid. As for timing, he said part of it depends on regulators, though Bluebird will be as aggressive as possible in getting it cleared.

The company also presented positive results from a clinical study for a multiple myeloma treatment. Celgene has partnered with Bluebird to license the drug.

Shares of Bluebird soared 24 percent on Monday.