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Health · Gene therapy

One-off gene fix frees patient from weekly alpha-1 infusions

Doctors at Hannover Medical School (MHH) and Shanghai's Renji Hospital have used base editing to correct the genetic error behind alpha-1 antitrypsin deficiency, an inherited disease that damages the lungs and liver. Katrin H., 55, is making the missing protein again and no longer needs her infusions. The treatment is experimental and not yet approved: the first published case covers a single patient and the trial is ongoing.

By The Newsroom · VerifiedSource: Hannover Medical School (MHH) · Journal of Hepatology
Health · Cancer

Nine in ten patients with advanced medullary thyroid cancer alive at three years on selpercatinib

In the international LIBRETTO-531 trial, presented at the ESMO 2026 congress by oncologist Jaume Capdevila of Vall d'Hebron Hospital and VHIO in Barcelona, 92% of patients with advanced medullary thyroid cancer and a RET gene mutation were alive at three years on selpercatinib, compared with 75% on standard drugs. The treatment is only for tumours with that mutation, and the trial is funded by drugmaker Eli Lilly.

Health · Cancer

Barcelona scientists find a weak spot in liver cancer cells

Some liver cancer cells rely on two proteins, CDK16 and CDK18, which cover for each other to repair their DNA. Remove both and the damage builds up, tumour cells stop multiplying and, in mice, tumours shrink. It is a preclinical result from Marcos Malumbres's group at the Vall d'Hebron Institute of Oncology (VHIO), published in PNAS: there is no drug or patient trial yet.

The numbers

92% Patients alive at three years on selpercatinib in LIBRETTO-531, versus 75% on cabozantinib or vandetanib (ESMO 2026). Read the story → 2 Proteins, CDK16 and CDK18, that must be blocked together: removing just one barely affects liver cancer cells, according to VHIO. Read the story → 1 Infusion of the new gene treatment. Before, Katrin H. needed a protein infusion every week for life. Read the story →