Three-year-old Ava was constantly sick. Her gums were inflamed, and every time she got a scraped knee, it turned into a dangerous infection.
Her parents, Alicia and Jon Langenhop, were months pregnant with their third child when they learned that Ava’s constellation of symptoms added up to an extremely rare, inherited disorder of the white blood cells, called leukocyte adhesion deficiency-1. Although antibiotics and antivirals could prolong her life, the disease was considered fatal, usually before kindergarten.
Ava’s primary hope, doctors told the Langenhops, was a bone marrow transplant from someone who was a good match, probably a brother or a sister.
Two-year-old Olivia had inherited the same disease as her big sister. She had been hospitalized with infections, too.
The baby in Alicia’s belly would be the girls’ best hope. Since both parents were carriers of the rare genetic mutation, the new baby, a boy, had a 25% chance of inheriting it, too.
Alicia was still in the hospital last October when they found out baby Landon had the mutation. Around the same time, the couple learned of a research trial in California.
Doctors would take each child’s blood cells, fix the mutation and return them. It should be a permanent fix, with less risk than a bone marrow transplant because the healthy cells would be their own, so their bodies wouldn’t reject them as foreign.
The approach had been tried in only one child, though.
This is the type of research reaching patients nearly two decades after President George W. Bush banned federal funding of stem cell research and 16 years after California residents approved a tax increase on themselves to support research.
Proposition 14 on Tuesday’s ballot asks whether Californians want to continue this work, providing $5.5 billion for stem cell research over the next three decades.
In the early 2000s, stem cell research was controversial because it often required the destruction of human embryos. Though embryonic stem cells remain essential for some therapies, in cases such as the Langenhops’, treatment focuses on manipulating a person’s own cells.
Stem cell science has made tremendous progress, but as in most new fields, the pace remains painstakingly slow. Every treatment has to be the subject of years of trial-and-error research, and many scientific hurdles linger.
Stem cells have been used to treat rare diseases, such as severe combined immunodeficiency, also known as “bubble boy disease,” and they are being tested in more common conditions such as Parkinson’s disease, macular degeneration, Type 1 diabetes and even heart disease.
“Even if a subset of stuff in the pipeline goes all the way, it will change the world for patients who currently don’t have other good options,” said Sean Morrison, a stem cell biologist in Dallas.
“It’s a pivotal time in the field,” said