The results of the small trial have not been published or peer reviewed, but the gene therapy company uniQure is looking to seek approval for the experimental treatment early next year. Plus, news on junk genes, anti-malaria baby wraps, the rollout of cheaper HIV drugs, and more.
The Washington Post: New Gene Therapy Slows Huntington’s Disease Progression
An experimental treatment for the first time slowed the devastating progression of Huntington’s disease, gene therapy company uniQure announced Wednesday, a rare hopeful advance against a cruel genetic disease that robs people of control of their bodies and minds in the prime of life. … About 40,000 people in the United States have symptomatic Huntington’s, which is caused by a mutated gene. (Johnson, 9/24)
In other innovations —
The Washington Post: For Decades, Scientists Puzzled Over A Genetic Anomaly. They Just Solved It
When couples have trouble conceiving a baby or lose a pregnancy, they often undergo routine tests, which can turn up a shock: One of the prospective parents may be missing a chromosome. The most common chromosomal abnormality — carried by about 1 in 800 people — is a “Robertsonian translocation,” when two chromosomes get fused together. People are often healthy, but one short of the typical 46 chromosomes for a human. Most don’t learn they carry this genetic anomaly unless they experience reproductive problems and seek testing. (Johnson, 9/24)
MedPage Today: Insecticide-Treated Baby Wraps Ward Off Malaria
Toting around young children in baby wraps treated with insect repellent cut their malaria infection rate by two-thirds, according to findings of a randomized trial in Uganda. Among 400 pairs of moms and children who all used insecticide-treated bed nets at night, the clinical malaria incidence rate for infants fell by 66% for those carried in permethrin-treated wraps rather than sham-treated wraps … said researchers led by Ross Boyce, MD, of the University of North Carolina at Chapel Hill. (Rudd, 9/24)
On the high costs of prescription drugs —
Bloomberg: Gates Foundation Accelerates Rollout Of Cheaper HIV Drug Lenacapavir Injection
A twice-yearly injection described as the most promising HIV prevention tool in decades is poised to reach millions more people, with new generic versions priced at about $40 per patient per year. The Gates Foundation and Indian drugmaker Hetero Labs Ltd. are among the groups moving to produce the medication, lenacapavir, which Gilead Sciences Inc. sells in the US for a list price of more than $28,000 annually under the brand name Yeztugo. (Kew and Furlong, 9/24)
Bloomberg: Cancer Drug Makers Push $200,000 Cure Even As Cheaper Options May Work
Three hours inland from Chennai, India, traffic crawls on a half-finished road past rice fields and cow crossings until it reaches a newer complex of neat white buildings. Among them is the cancer wing of a hospital founded over a century ago by American missionaries. … A single infusion might cost at least $7,000 at the standard dose in the US, and a year’s treatment more than $200,000. Here, by financial necessity, most of the patients are getting as little as one-sixth of that. (Langreth, 9/24)
Bloomberg: Pharma Middlemen Seek To Head Off New Rules With Voluntary Changes
Pharmacy middlemen are working on a proposal to voluntarily change some of their business practices in an effort to avoid new regulation from the Trump administration, according to people familiar with the discussions. The main lobbying group that represents so-called pharmacy benefit managers, the Pharmaceutical Care Management Association, has drafted proposals to bring to the Centers for Medicare and Medicaid Services, according to a document viewed by Bloomberg News. (Tozzi and Cohrs Zhang, 9/24)
ScienceDaily: Breakthrough Method Could Dramatically Cut Prescription Drug Prices
University of Maine researchers developed a new process to make HBL, a key ingredient in many medicines, from renewable glucose instead of petroleum. The approach not only lowers drug production costs but also reduces emissions. (University of Maine, 9/21)
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