Early encouraging UC Davis trial data on cell therapy for spina bifida

A Lancet paper is sparking excitement about an investigational cell therapy for spina bifida.

Here’s the study: Feasibility and safety of cellular therapy for in-utero repair of myelomeningocele (CuRe Trial): a first-in-human, phase 1, single-arm study. Myelomeningocele is the most severe manifestation of spina bifida

The work was led by my UC Davis colleagues Diana Farmer and Aijun Wang. The trial is called Cellular Therapy for In Utero Repair of Myelomeningocele. I was not involved in the study, but I’ve followed it closely for years.

Congrats to the team for these early, promising data.

 

Cell therapy for spina bifida

The cells used for repair were placental MSCs. Here’s the key finding:

“Between June 21, 2021, and Dec 5, 2022, six women with fetuses with gestational ages from 24⁺⁵ weeks to 25⁺⁵ weeks were enrolled in the study. Newborns were delivered at a median gestational age of 34⁺⁵ weeks (range 33⁺² weeks to 36⁺⁶ weeks) by caesarean delivery. At birth, all infants had an intact repair site with no evidence of cerebrospinal fluid leak, infection, or abnormal tissue growth. After treatment, MRIs showed reversal of hindbrain herniation and no evidence of tumour formation. No cell-mediated adverse events occurred.”

This is encouraging. It provides early possible signals of both safety and efficacy.

This is a complex cell therapy procedure involving intrauterine surgery on fetuses. Imagine how challenging that is compare to other approaches with MSCs for various health conditions such as simple IV administration.

Putting MSCs right where they might do some good as in this trial also seems more likely to be effective than injecting them into the bloodstream.

Interestingly, and highlighting the importance of animal models, years ago the UC Davis team tested the cell therapy in dogs that spontaneously get spina bifida including one named Arthur (see below).

Hoping for consistent, longer-term results too

Circling back to the new trial results, the paper got some rightfully upbeat coverage including from Nature: World-first stem-cell therapy shows promise for treating spina bifida in the womb.  From the Nature piece:

“Farmer says the children who participated in the trial will need to be at least two years old before any benefits can be observed, because they will have learnt how to walk by then. Her team has been approved to conduct a larger trial in another 29 people, which will give the researchers the statistical power to show whether the stem cells lead to improved outcomes.”

I’m hopeful that the longer-term data will also be encouraging. The field needs options for prevention and treatment of spina bifida.

As a side note, coverage of this story has shown the right balance between enthusiasm about the positive results so far and stressing the need for longer-term follow-up to collect more data. This is how you do it.