New therapies for a range of conditions are coming in 2022
Haemophilia, brain disorders and malaria are being targeted
By Natasha Loder: Health-policy editor, The Economist
PUBLIC INTEREST in medical research has lately been dominated by treatments and vaccines for covid-19. But research in other areas has continued, and is about to bear fruit. In 2022 giant strides will be made in treating haemophilia, and there will be a resurgence of interest in drugs for neurological disorders—and progress on malaria.
Haemophilia, a disorder in which blood fails to clot in the usual way, has been with humanity throughout history, and now affects more than 1.1m men around the world. The earliest mention of it is in the Talmud, which exempts a woman’s third son from circumcision if his two elder brothers have died of bleeding after the procedure. These days haemophilia can be treated by replacing the missing blood-clotting factors through therapies delivered by injection. These have alleviated some of the worst outcomes of a genetic disorder that can disable as well as kill. But for decades researchers have hoped to correct these inborn errors by providing the correct gene via gene therapy. At least one such treatment is likely to reach the market in 2022.
The correct genes for clotting factors are introduced into the body by a harmless virus, a similar trick to that employed by some covid-19 vaccines. Instead of carrying the genetic recipe for a spike protein, however, the virus carries the instructions for making the missing proteins. And unlike a covid-19 vaccine, the virus is engineered so that the genetic material it carries takes up permanent residence inside the cells it infects, granting them, in theory, life-long ability to manufacture the clotting factors.
In 2022 giant strides will be made in treating haemophilia
The two firms closest to having the first licensed product have targeted different variants of the disease. Biomarin, an American firm, has developed a treatment for haemophilia A called Roctavian; uniQure, based in the Netherlands, has devised a treatment for haemophilia B called EtranaDez. Either or both may gain approval from drug regulators in America or Europe in 2022. And coming up behind them is a joint effort by Pfizer, a drugs giant, and Spark Therapeutics, a biotech firm, to treat haemophilia B.
These therapies promise to be life-changing, but are expected to have multi-million-dollar price tags. Their success will therefore depend not only on the science, but on whether those who pay for them can agree on pricing. Some drug firms have struggled to convince European countries that making large upfront payments for medicines can cut costs down the line.
A gene-therapy drug for beta-thalassemia, another blood disorder, was pulled from the German market by its maker, Bluebird Bio, when it was unable to reach an agreement with the government on pricing. This suggests that scientists’ technical ability to develop treatments is starting to push up against the boundaries of what society is willing to fund.
New therapeutics for diseases of the brain are also worth watching in 2022. The granting—by American regulators—of approval for Aduhelm, an Alzheimer’s drug made by Biogen, a biotech firm, in 2021 has reinvigorated interest in therapies for dementia and other neurological disorders—even though some question the efficacy of the new treatment. Eli Lilly, a drugs giant, hopes to follow in 2022 with its own Alzheimer’s drug, donanemab, which also targets the amyloid proteins in the brain.
Alector, a biotech firm, is betting that neurodegeneration can be tackled by harnessing the body’s immune system. In 2022 it will be dosing patients with a drug to treat frontotemporal dementia, a rapidly progressing and severe form of the condition that affects about 170,000 people in America and Europe. In addition, interest is growing in using psychedelic drugs to treat brain disorders.
A new malaria vaccine will be rolled out in sub-Saharan Africa towards the end of 2022. It is a historic step forward, even though it is only 30% effective and requires four doses. Better vaccines will follow. Meanwhile, Pfizer and BioNTech will start producing their mRNA-based covid-19 jab in Africa by the end of the year. That will give efforts to make new vaccines in Africa, for Africa, a welcome shot in the arm.
Natasha Loder: Health-policy editor, The Economist■
This article appeared in the Science and Technology section of the print edition of The World Ahead 2022 under the headline “Blood and treasure”
