A Tale of Two Frogs: a rare disease story

Posted Tuesday July 30, 2013 by Melissa

“It’s a miracle!” she said, as she watched her son hop, ever so gently, for the first time. Having never had legs before, even hopping was quite a new endeavor for the young frog. But he hopped nonetheless, and even cracked a smile about it.

Squinting his eyes, Bullfrog said, “Let me measure the hop.” He bent forward with his ruler, monocle dangling from his eye over the lilypad splashed with a minute amount of pond water from the hop.

“Are you sure it was a hop?” he questioned.

The mother scoffed. “A hop? You’re measuring the hop? Why, the boy never had legs before and you think it’s the hop that’s important? And how does measuring the splash tell you about the hop?”

bufo“But we must measure,” said Bullfrog, “or we don’t know if the legs work. We have to measure or we can’t give new legs to other young frogs. You see, we’re not sure how their bodies would like the new legs. The legs might hurt them. They might jump, then fall into the water and drown. Drowning is dangerous. And deadly.”

The mother was perplexed.

Did they witness the same thing? She wasn’t sure why the Bullfrog was so worried about them drowning when he knew full well that without legs, the frogs could hardly catch flies. Without catching flies, they’d slowly wither away and die.

And that was a certainty.

The possibility of drowning from having legs was surely worth the risk.

Bullfrog continued. “You see, I report to the Black Toads, and the Black Toads report to the Goliath Frogs at the Goliath & Frog Administration. The GFA has procedures about these kinds of things.”

The little boy frog hopped to the next lilypad. And the next.

“There! You saw it! What big hops those were! Now, obviously you see the need for all of these little boy frogs to have legs.” The mother frog felt confident Bullfrog would see her point now.

“Yes, I do actually. Those were great hops. Let me measure again….” His ruler did show quite a splash. But, something about it was still off. “Hmmm, he’s not really hopping like a regular frog.”

“Seriously?” she said. “I don’t really care if he’s hopping like a regular frog! The boy’s got legs. He can move. He can eat. He will live!”

Bullfrog remained calm, even in the face of the mother’s insistence. He now watched the young frog hop once, twice, three times, getting further away as his hopping became more part of him and less about the legs.

“You see,” the mother continued, “I know many other mother frogs whose boys have no legs. They are over there, watching my son hop along, from lilypad to lilypad. Do you see? Maybe you can’t see that those mothers are crying? They want to see their boys hop too.”

Bullfrog sat in silence. He himself then jumped from lilypad to lilypad, somewhat uncomfortable at what the mother was saying. Yes, he could see those other mother frogs. And yes, he could see them crying.

He paused at a new and dry lilypad to make his argument.

“But the GFA…. The GFA is here to protect them. To protect them from… drowning. Legs are not for everyone, you know.”

Bullfrog seemed to be growing more uncomfortable at his defenses, his own legs rocking back and forth on the lilypad, as if reminding him of how nice and comfortable it was to have legs.

As if on queue, up jumped an even larger frog.

“Ma’am, I am with the Goliath & Frog Administration and I am here to protect you. Um, them. Um, the Black Toads. Oh, bother! Um, what again am I protecting you from?”

“I don’t precisely know,” she muttered.

“Ma’am, our procedures for these procedures have been tested by tests and tried by trials. We may try out some legs on a couple of other frogs, but those few can’t be too old, too young, too sick, too healthy, too bumpy, too smooth, too green, or too brown either. So, just a few, you see.”

“And then we’ll need to measure and compare and see if those legs are really worth the risk. The risk of drowning, you know. It may take a few years before we are really ready to give those legs to most of those frogs over there.”

Hearing the lengthy list of procedures and criteria, measurements and risks, the mother’s heart broke. “And I’m just supposed to sit here, watching my frog jump, and watching their frogs…?”

“I’m sorry ma’am. In about three years, if all is a success, they’ll be able to get legs also.”

She scrutinized his slimy face, even for one hint of compassion, understanding that his procedures at the GFA were sentencing many of those other little boy frogs to a slow death. Fortunately for him, the slime hid his emotions.

Ztip!

The motion was so quick that no one saw where it came from. The heavens, maybe? A swift blade descended and in one fell swoop, chopped off the legs of Mr. GFA, and carried them off, with the likely destination being someone’s dinner plate.

He lay there, shell-shocked. A stunned silence lay over the pond like a humid fog, but for the falling tears of the nearby mothers.

Once he gathered himself, the slime no longer hid his emotions. Utter despair poured forth at the realization that his legs were no longer.

No one understood more than the mother frogs. A tear slowly rolled down her cheek.TaleofTwoFrogs

“Bullfrog! Bullfrog, sir! Please, someone help me. I’ve lost my legs and now I’m destined to die a slow and agonizing death here alone on this single lilypad. I need my legs. I need legs. Please….”

Bullfrog answered, tentatively. “But Goliath, sir, your procedures? You said that new legs were not ready for other frogs, just a few boys. And you, sir, are probably too old, too healthy, too smooth, and too green.”

Bullfrog continued, more sure now that he remembered correctly, “And the legs, you’re worried about their jumping, the risk of drowning…. You said new legs weren’t worth the risk of drowning, right sir?”

Hadn’t Goliath been explaining this himself? Bullfrog thought, still a little confused.

Goliath sputtered his reply, “But now it’s happened to me!”

—————————

 

This allegory is titled “A Tale of Two Frogs” to express the alternate perspectives of Goliath as Mr. GFA versus being the patient himself. An alternative title would be:

An open letter to Pharma and the FDA about supporting early and rapid compassionate access to an unapproved drug proposing to treat a deadly, degenerative disease where the drug has shown promise in slowing or halting the degenerative nature of the disease and preliminary safety has been established such that the risk profile is acceptable to the community of patients proposed to be treated.

I have a hard time believing that I would be watching my friends’ children continue to suffer if every person at the pharmaceutical company and the FDA involved in the drug evaluation process either themselves suffered from Hunter Syndrome or loved a child at home suffering from it.

It’s now been 3 1/2 years since the Phase I/II started with IT Idursulfase. It now could easily be another 3-4 years before the Phase III trial finally begins, is enrolled, is completed, and the drug finally becomes FDA approved. A child could be born, diagnosed with Hunter Syndrome, and die from it, all within that period of time, although we now know its safety profile and that it works.

I expect compassionate access to IT Idursulfase to happen as quickly as possible, not because I think it is the norm, but because it is the right thing to do, procedures or not.

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One Response to “A Tale of Two Frogs: a rare disease story”

  1. […] is that dichotomy that caused me to write the previous post, A Tale of Two Frogs: a rare disease story. While I’ve been able to celebrate the stability of my son and his new learnings, I am faced […]

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