Sickle Cell Trials Halted After Two Participants Diagnosed With Cancer
Bluebird Bio said a patient treated more than five years with its Lentiglobin gene therapy was diagnosed with acute myeloid leukemia, and a second patient has myelodysplastic syndrome.
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Bluebird Suspends Sickle Cell Gene Therapy Studies After Cancer Diagnoses
Bluebird Bio said Tuesday that it has suspended clinical trials involving its gene therapy for sickle cell disease after receiving reports that two patients treated with the one-time therapy were diagnosed with cancer. The trials were placed on “temporary suspension” so that Bluebird can investigate the cancer cases to determine if they were caused by the re-engineered HIV virus used to deliver its gene therapy. No such link has been established yet, the company said. (Feuerstein, 2/16)
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Novartis, Gates Foundation Pursue A Simpler Gene Therapy For Sickle Cell
Novartis and the Bill and Melinda Gates Foundation are joining forces to discover and develop a gene therapy to cure sickle cell disease with a one-step, one-time treatment that is affordable and simple enough to treat patients anywhere in the world, especially in sub-Saharan Africa where resources may be scarce but disease prevalence is high. The three-year collaboration, announced Wednesday, has initial funding of $7.28 million. (Cooney, 2/17)
In updates on the opioid crisis —
Stateline:
Opioid Deaths Spark Push To Ease Buprenorphine Rules
With drug overdose deaths soaring during the pandemic to the highest levels ever recorded, a growing chorus of medical experts is calling on the federal government to deregulate the addiction treatment medication buprenorphine. They argue that a requirement that doctors take an 8-hour course and submit to Drug Enforcement Administration (DEA) oversight has stymied the drug’s availability. But opioid addiction treatment providers and a major patient group argue that, in fact, more training is needed to protect patients. Some also worry that looser rules will result in the pills being resold illegally. (Vestal, 2/16)
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FDA Warns AcelRx Of Misleading Ads For Its Pain Drug Dsuvia
The manufacturer of Dsuvia, a pain drug approved by the Food and Drug Administration amid substantial controversy, was taken to task by the agency for creating misleading ads that minimized the risks associated with the opioid medicine. (Silverman, 2/16)
In other pharmaceutical industry news —
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FDA's Woodcock Rejects Proposal To 'Firewall' Agency And Drug Companies
In response to criticism, a top Food and Drug Administration official maintained that creating a firewall between agency staff during the process for reviewing medicines “would cause significant negative repercussions for public health.” (Silverman, 2/16)
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Moncef Slaoui To Test His R&D Philosophy In A New Company
Moncef Slaoui, who headed the Operation Warp Speed vaccine-development effort under the Trump administration, is returning to his previous role as a venture capitalist and announced Tuesday the launch of a new company, Centessa Pharmaceuticals. (Herper, 2/16)
