A mom's quest: Saving her twin daughters' lives

Chris Hempel, other: You look back and that's when it really hits you, you know.  Then you go, wow, they're deteriorating. 

Because of a rare and fatal genetic disorder called Niemann-Pick type C, the girls have stopped developing. And in fact, they're regressing. 

Hugh: I find myself once in a while going back and looking at some of the tape and it's hard because they're singing their ABC's and they're running around the house and they're talking in full sentences. It's a reminder of their progression and that's not easy.

Hoda Kotb, Dateline correspondent: What do you miss?

Hugh: Oh, that's easy. Just the words, you know. It's their ability to express themselves. Let's see… sorry (cries)

In an attempt to stop the downward slide, Chris and Hugh have developed a strict regimen of drugs and supplements.

Chris (showing the drugs): We've had to create our own virtual pharmacy because with a rare disease, there's just nothing in the pipeline that's going to help us. This probably looks, to a lot of people, scary, like what is this mom doing? She's putting all this stuff in her kids?  But no, I've really done the research. I've talked to the scientists. I make sure it's not going to be harmful. I look at the amounts.

After being told repeatedly that there was nothing she could do to keep her little girls from dying before their teens, Chris, at last, found a glimmer of hope buried in an obscure scientific article.

Chris: This is the Cyclodextrin…

Not even a drug, Cyclodextrin is an organic sugar compound found in butters and salad dressings on grocery shelves across America. And it appeared to have the ability to break down cholesterol, the very thing that was causing Addi and Cassi's systems to malfunction.

Chris: It tastes kind of like Sweet and Low. I did a lot of research in terms of how much could you put in the sippy cup, in terms of ingesting it, so we started doing that. Then the mice data started coming out showing it doesn't look like it's gonna work if they're eating it.

In order to see results, Cyclodextrin had to be injected directly into the bloodstream. Because Cyclodextrin had never been used in this way on humans, the U.S. Food and Drug Administration had to sign off. The Hempels had to file a lengthy application with the FDA. It was a laborious and intimidating bureaucratic hurdle that would stop most people in their tracks.

Hastings: It's almost unheard of that this would be motivated by a parent or a family.

Next, to back up their application, they had to convince a giant pharmaceutical company to give them the scientific data they collected showing that Cyclodextrin was safe.

Chris: It was pretty disheartening really. They just said: "You know look, we don't provide this information and good luck with getting the FDA to approve it." I kinda hit a real brick wall. The response was not what you would expect when you have two  dying children and you're looking for information on a sugar compound.

The tenacious and quick thinking PR exec wouldn't take “no” for an answer.  She decided to go over their heads and directly to their parent company. 

Chris: It seemed logical to me that here we have Addi and Cassi, two beautiful kids, fighting for their lives. I mean Johnson & Johnson cares so much about kids that surely they would help. As soon as they found out about it, they acted immediately.

Chris got the data, but the FDA still needed more.

Hastings: We were gonna have to develop some type of protocol and monitor the safety of giving this drug and also come up with a way that we were actually going to specifically measure if it made some difference.

After six months of back and forth, Chris won her battle.  She got the green light from the FDA to give the girls IV infusions of Cyclodextrin.

On a clear morning in late April, Chris and Hugh prepared their daughters and themselves for a four-day stay at Renown Children's Hospital in downtown Reno. They put on a brave face, but their nerves were palpable. In a few hours, Cyclodextrin was going to flow through an IV drip into Addi and Cassi's veins.

Adam Porath, lead pharmacist: This protocol was different than a typical drug study because for this procedure, we didn't really have a blueprint for what we were supposed to do.

Finally, the two-year quest to find something, anything that could stop the mental and physical decline of Addi and Cassi Hempel begins. Overwhelmed, excited, exhausted, and hopeful, the immensity of this event squeezes out everything else in the room.

Addi and Cassi sit quietly, sharing a bed, staring blankly, as the Cyclodextrin drips into their IV’s. Chris and Hugh Hempel did the impossible—battling the government on behalf of Addi and Cassi—and came out on top.

Now they begin another long wait, to see if their hard won victory pays off in the only way that counts.

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