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-   -   Dwarfism Drug Brings Mixed Emotions for Little People, Parents (http://message.snopes.com/showthread.php?t=92107)

A Turtle Named Mack 03 July 2015 04:42 PM

Dwarfism Drug Brings Mixed Emotions for Little People, Parents
 
An experimental drug to treat dwarfism by lengthening childrenís bones is meeting skepticism from the leading U.S. group that represents little people, which questions giving kids a medication that might make them less little.

http://www.bloomberg.com/news/articl...people-parents

Mr. Billion 07 July 2015 08:23 AM

This seems sort of similar to the deaf community's reactions to the cochlear implant.

It can allow formerly deaf people to hear, but in so doing it diminishes something that makes them unique and drew them together into a vibrant subculture with their own language. (The fact that the deaf have their own languages is one of the most important sources of deaf cultural identity; without that, the little people community is probably much less coherent.) There was a great documentary about the clash over the cochlear implant called "Sound And Fury".

This is pretty interesting. Thanks for sharing it, ATNM.

jimmy101_again 07 July 2015 09:27 PM

The same basic push-back happened decades ago when human growth hormone came out. Treatment of short stature in children is one of the few approved use in children.

I would remind the little people groups that they don't get to say what is best for someone else. If there or no (or minimal) side effects, which hasn't yet been established, then there is no adequate reason that would justify one group deciding what another group is allowed to do.

The ethics of the medical treatment of short stature has been debated and largely resolved in favor of patients get to choose if the treatment is right for themselves.

GenYus234 07 July 2015 09:38 PM

Quote:

Originally Posted by jimmy101_again (Post 1878532)
I would remind the little people groups that they don't get to say what is best for someone else.

Except most of the time it is going to be useful only for minors whose parents would be the ones deciding.

jimmy101_again 07 July 2015 10:39 PM

Quote:

Originally Posted by GenYus234 (Post 1878535)
Except most of the time it is going to be useful only for minors whose parents would be the ones deciding.

Touche, though I would prefer parents make the call.

Jahungo 07 July 2015 11:15 PM

Two facts that may have some implications here, although exactly what those are I am not sure.

1. Achondroplasia is genetically dominant, meaning affected individuals have one "wild type" copy and one affected copy. This means that a parent with achondroplasia will have a 50% chance of having an affected child, 50% unaffected. Even two parents with achondroplasia are likely to have unaffected children. So it is very common for parents with achondroplasia to raise unaffected, "normal height" children.

2. More than 90% of cases of achondroplasia are due to spontaneous mutation. That is, a child born with achondroplasia to two parents without it. So most children with achondroplasia are not born into families with it.

Little Pink Pill 07 July 2015 11:23 PM

Quote:

“My daughter is incredibly smart, she’s funny, she is the most loving person I’ve ever met,” said Martinka, who doesn’t have achondroplasia. “I can’t ask for anything else."
(Bolding mine.)

Maybe you can't, but what about her? Is this about what the parent wants from the kid, or what the kid wants from life?

Loving your child the way they are doesn't mean pretending untreated genetic issues won't affect them Every. Single. Day.

geminilee 07 July 2015 11:58 PM

I think the major distinction between this and cochlear implants, for me, is that deafness does not cause other health problems, and dwarfism does. If it were just a matter of shortness then I would say it is just a choice. The fact of serious health complications takes it out of that realm for me.

St. Alia 08 July 2015 01:40 AM

Quote:

Originally Posted by jimmy101_again (Post 1878542)
Touche, though I would prefer parents make the call.

Who else would?? :confused:

thorny locust 08 July 2015 02:07 AM

Quote:

Originally Posted by geminilee (Post 1878547)
I think the major distinction between this and cochlear implants, for me, is that deafness does not cause other health problems, and dwarfism does.

It doesn't seem clear at this point whether the drug in question itself causes other health problems; or whether it would prevent health problems associated with achondroplasia.

From the article:

Quote:

The study involved just 26 children with an average age of 7.8 years, with the 10 getting the highest dose seeing the most benefit. There was no placebo comparison, so the trial didnít examine how they fared compared to children getting no treatment.

Thereís no evidence so far that the therapy made the proportions of the childrenís bodies more like an average personís. And thereís no way to know if the accelerated growth would stop, a potential problem since other drugs tested to treat the condition have been difficult to control.

A final study of BMN 111 will probably involve 50 to 100 patients treated for six to 12 months, according to BioMarin. That may not be enough time to show if it helps children avoid serious health complications or if long-term use comes with side effects or potential harm.
So to the extent that people don't want to use a mostly untested drug that may cause more problems than it prevents, such reluctance makes considerable sense.

If the drug were proven to work long-term (at least well into adulthood) with rare or minimal negative effects, and with benefits to overall health, that would of course be different. But by the time that's known, most children currently living with the condition may be too old to use it.

I wouldn't criticize anyone for wanting their child to be part of the trials. But I also wouldn't think less of anyone for not leaping to be among the first to try it.

geminilee 08 July 2015 02:14 AM

Most children now would, yes. And I wouldn't criticize someone for not joining the trials. But if it were proved safe, parents should be strongly encouraged to go with it. Dwarfism can cause so many very serious health problems that I think it would be wrong to risk them (assuming of course that the risks from the drug were less, and there wasn't a specific medical reason a child wasn't a good candidate for it.)

A Turtle Named Mack 08 July 2015 01:03 PM

Quote:

Originally Posted by Little Pink Pill (Post 1878546)
(Bolding mine.)

Maybe you can't, but what about her? Is this about what the parent wants from the kid, or what the kid wants from life?

Loving your child the way they are doesn't mean pretending untreated genetic issues won't affect them Every. Single. Day.

The attitude of "she's such a wonderful child" reminds me of the parents of Down Syndrome people - they almost all say what cheerful, delightful people their DS children are. I don't doubt that, but I also have little doubt that nobody goes into pregnancy hoping for a DS case, and very very few would refuse a treatment with modest side effects that would allow their child to develop and progress without the DS effects.

jimmy101_again 08 July 2015 06:59 PM

Quote:

Originally Posted by St. Alia (Post 1878551)
Who else would?? :confused:

Have you read the posts in this thread? The association of little people appears they may consider arguing to ban the treatment. From the cite:
Quote:

An experimental drug to treat dwarfism by lengthening childrenís bones is meeting skepticism from the leading U.S. group that represents little people, which questions giving kids a medication that might make them less little.

GenYus234 08 July 2015 07:17 PM

How about an actual quote from the president of that group?

Quote:

This drug might be right for some people, but we also think it’s important to get across the message that it’s not necessary to live a healthy and productive life. [bolding mine]
Hard to imagine they want to ban the treatment if the president is saying that it might be right for some.

St. Alia 08 July 2015 07:22 PM

In reply to Jimmy101_Again:
Yes, I've read the posts. And I've read the article.

I see nothing that implies anyone trying to ban the drug or trials, and definitely nothing that implies that parents of young children wouldn't be the ones who would make the choice of whether to use the drug or not.

jimmy101_again 08 July 2015 10:22 PM

Quote:

Originally Posted by GenYus234 (Post 1878643)
How about an actual quote from the president of that group?

Hard to imagine they want to ban the treatment if the president is saying that it might be right for some.

Or a different bolding:
Quote:

This drug might be right for some people, but we also think itís important to get across the message that itís not necessary to live a healthy and productive life. [bolding mine]
Which is a direct argument against using the treatment. An argument against a treatment for a particular indication is generally used as an argument against FDA approval for a particular indication.

jimmy101_again 08 July 2015 10:26 PM

Quote:

Originally Posted by St. Alia (Post 1878644)
In reply to Jimmy101_Again:
Yes, I've read the posts. And I've read the article.

I see nothing that implies anyone trying to ban the drug or trials, and definitely nothing that implies that parents of young children wouldn't be the ones who would make the choice of whether to use the drug or not.

Posts 2 and 3 (at least) directly speak to previous resistance to a new treatment by a group associated with the affected individuals. Both cochlear implant and HGH (used to treat short stature) have previously met resistance to FDA approval from support groups for those indications.

thorny locust 09 July 2015 12:18 AM

Quote:

Originally Posted by jimmy101_again (Post 1878680)
Or a different bolding:

Quote:

This drug might be right for some people, but we also think itís important to get across the message that itís not necessary to live a healthy and productive life. [bolding [jimmy101_again's]]
Which is a direct argument against using the treatment. .

If some people with achondroplasia do live healthy and productive lives, then it's just plain a statement of fact.

http://www.medicinenet.com/achondrop...achondroplasia

Quote:

Children and adults with achondroplasia can lead normal lives provided they receive attentive, informed care by their physicians and parents.
https://www.genome.gov/19517823

Quote:

The intelligence and life span in individuals with achondroplasia is usually normal. [bolding mine]

As the drug is currently almost untested and may carry as many or more long-term risks as untreated achondroplasia, then I don't see the sense in vehemently arguing for everyone affected to use it as soon as possible; especially as the article says there's no evidence that the drug in question helps with any of the issues with achondroplasia other than height.

The 12-month "final trial" the company intends to run isn't nearly long enough to check for possible long-term problems.

I don't see any problem with an argument stating that testing should continue but that, in the meantime, people should remember that those with the condition can often lead full and productive lives without the drug.

jimmy101_again 09 July 2015 05:16 PM

Quote:

Originally Posted by thorny locust (Post 1878700)
As the drug is currently almost untested and may carry as many or more long-term risks as untreated achondroplasia, then I don't see the sense in vehemently arguing for everyone affected to use it as soon as possible;

Sorry, I missed where anyone suggested vehemently that everyone affected should use it as soon as possible.
Quote:

I don't see any problem with an argument stating that testing should continue but that, in the meantime, people should remember that those with the condition can often lead full and productive lives without the drug.
And people should also realize that it isn't their call as to whether someone else should use the treatment. And as you said "often lead full and productive lives without the drug" so there are people that might be helped.

thorny locust 09 July 2015 06:51 PM

Quote:

Originally Posted by jimmy101_again (Post 1878799)
Sorry, I missed where anyone suggested vehemently that everyone affected should use it as soon as possible.
And people should also realize that it isn't their call as to whether someone else should use the treatment. And as you said "often lead full and productive lives without the drug" so there are people that might be helped.

1) Some people seem to be saying that it's wrong for anyone to say it might not be the right thing for everyone with the condition to take it. Possibly "vehement" is the wrong word.

2) Nobody appears to have suggested otherwise.

3) We have no idea at this point whether there are people who might be helped by this particular drug. Again, at this point it's unknown both whether it does anything to help actual medical difficulties (as opposed to height) and whether it causes more problems than it helps.


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