Nov 7, 2019

Doctors safely use CRISPR on cancer patients

A researcher performs a CRISPR/Cas9 process at the Max-Delbrueck-Centre for Molecular Medicine. Photo: Gregor Fischer/picture alliance via Getty Images

Three cancer patients in the U.S. were the first to be safely injected with the gene editing tool CRISPR as a form of immunotherapy to fight their illness, AP reports.

Reality check: It's too soon to know whether the treatment will help, doctors say. The patients, who all failed multiple standard treatments, had varying results after two to three months.

Two of the patients have multiple myeloma, a blood cancer, and the third has a sarcoma, cancer that forms in connective or soft tissue.
The treatment deletes three genes that may be stopping the patients' cells from attacking the disease, and then adds ones to help them fight their cancer.
15 more patients will receive the treatment and doctors will gauge its safety and efficacy.

The bottom line: Researchers are hoping to use CRISPR to treat some genetic diseases, including its progress in treating sickle-cell anemia.

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Caitlin Owens

CRISPR-based gene editing treatments benefit patients with blood disorders

A researcher observes a CRISPR/Cas9 process through a stereomicroscope. Photo: Gregor Fischer/picture alliance via Getty Images

The first two patients to receive CRISPR-based treatments for their blood disorders benefited from the therapy, the two companies developing the treatment announced yesterday — a sign that gene editing may be a safe and effective way to cure blood diseases, STAT reports.

Why it matters: The two blood disorders, sickle cell disease and beta thalassemia, can have a drastic impact on patients' lives. A cure would be transformative.

Go deeperNov 20, 2019

Axios

FDA approves drug with annual cost of $442,000 for rare genetic disorder

Photo: Al Drago/CQ Roll Call

The FDA has approved the drug Givlaari, made by Alnylam Pharmaceuticals, for adults who have acute hepatic porphyria, a genetic disorder that leads to severe abdominal pain and affects just 1 in 25,000 people.

The big picture: Alnylam set Givlaari's net price, after discounts to insurers, at $442,000 per year, or about $39,000 per vial. It's a lofty price tag, but those prices are common among drugs that use new scientific techniques to treat very rare diseases.

Go deeperNov 21, 2019

Marisa Fernandez

HIV-positive babies could benefit from treatment days after birth

Antiretroviral drugs, administered within days of birth, appeared to bring HIV in newborns down to undetectable levels.

Why it matters: About 500 babies in sub-Saharan Africa are born with HIV every day, NPR reports. Though these early-treated children are not cured yet, researchers' recent study published in Science Translational Medicine shows encouraging results from a clinical trial in Botswana.

Go deeperDec 5, 2019