26 Dec COVID vaccines made mRNA a household name. How can it help in rare diseases?

A handful of companies have mRNA therapies in human trials for rare diseases.

By Kristina Fiore, MedPage Today

One of the hardest things about managing Jordan Franks’ propionic acidemia (PA) was feeding him.

Jordan never wanted to eat. His body seemed to know that food could poison him, so like many kids with PA, he was food-averse.

“My son didn’t want to eat anything by mouth,” his mother, Jill Chertow, told MedPage Today. “I think the most he would ever eat was like, two pieces of mac ‘n’ cheese. Or he would lick the salt off of a French fry.”

Kids with this rare disease can’t metabolize certain amino acids. So Jordan had a regular — and intense — schedule of formula feedings that he took in through his gastrostomy tube, or g-tube.

“There was a lot of stress in managing his nutrition,” said Chertow, who is president of the Propionic Acidemia Foundationopens in a new tab or window. “He had three different powdered formulas. One was a baby formula, like a Similac. Then there was a second formula that had protein but didn’t have the amino acids that were bad for him. And there was a third formula for calories.”

The formulas had to be mixed and weighed to the tenth of a gram to deliver precise amounts of nutrients. Depending on the time of day, Chertow also had to mix in medications.

Jordan had six of these g-tube feedings each day, and because fasting is dangerous in children with PA, Chertow turned on a pump every night to make sure he didn’t go too long without nourishment.

“It was challenging to manage all the feeds and within a timely manner,” said Chertow. “You can’t be too far off schedule, or it messes up the rest of the feeds for the day. Everything needs to be done pretty close to on time.”

Jordan died in 2016 when he was only 16 years old. There was no treatment for PA during his lifetime, and there still isn’t one. But families and researchers are hoping that the technology honed during the COVID-19 pandemic can help change that.

PA is one of a handful of rare diseases being targeted with mRNA therapeutics. Read more …