Vast Inequities Prevent Access to Life-Saving Treatment
Gautam Dongre’s two children in India and Pascazia Mazeze’s son in Tanzania live with an inherited blood disorder that turns blood cells into instruments of pain. New gene therapies promise a cure for sickle cell disease, and Dongre says he’s “praying the treatment should come to us.” But experts say the one-time treatment is out of reach in India and Africa — places where the disease is most common.
Access to Gene Therapies Limited in Developing Countries
Vast inequities cut much of the world off from gene therapy in general. While access to all sorts of medicine is limited in developing countries, the problem is especially acute with these therapies, some of the most expensive treatments in the world. Beyond their sky-high prices, these therapies are extremely complex to give patients because they require long hospitalizations, sophisticated medical equipment and specially trained doctors. So far, the two gene therapies for sickle cell have only been approved in wealthier countries: both in the U.S., and one in Britain and Bahrain as well.
Widening Healthcare Inequalities
“The vast, vast majority of patients live in an area where they have no access to this kind of therapy,” said Dr. Benjamin Watkins, who treats sickle cell at Children’s Hospital New Orleans. “We as medical professionals, and as a society, have to think about that.” Access to gene therapies was a major focus of this year’s international summit on human genome editing in London. A subsequent editorial in the journal Nature said high prices leave low- and middle-income countries “entirely in the lurch” and could stymie progress across the field.
Struggling for Basic Treatment
For gene therapy to even be an option, people in developing nations must stay alive long enough to get it. There, sickle cell disease is more likely to disable or kill than in wealthy regions. Late diagnosis is common and basic care can be hard to come by. Available treatments can reduce the bouts of pain known as “crises.” However, many patients in rural areas are dying at very young ages without getting the right treatments.
Overcoming Cost and Complexity
Such stark realities make the cost of gene therapies an insurmountable obstacle, experts say. The price tags for the two sickle cell therapies in the U.S. are $3.1 million and $2.2 million, although costs can vary by country. The process of giving the therapies is just as big a hurdle. Patients must go to the hospital, where stem cells are removed from their blood. The process can stretch on for months. Scientists are working to make easier-to-administer versions of the new therapies, but experts say simpler cures will likely still be unaffordable to many.
A Call for Global Accessibility
If gene therapy eventually makes it to developing countries, families like Dongre and Mazeze hope to have access to it for their children. Both agreed it should be an option in all countries — rich or poor. Dongre said, “We all are part of one single planet.”
