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.

Go deeper

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 deeperArrowNov 20, 2019

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 deeperArrowNov 21, 2019

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 deeperArrowDec 5, 2019