My friend is about to become blind, please help if you can

[SariesNightly]

My friend in WA is about to become blind. Please, If you live in DC or know about cystamine eyedrops please help,

http://tinyurl.com/656ss


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Okay heres the story. My eyes are very messed up from my illness. I used to get eyedrops to help them but the FDA doesnt make a lot of money from them so they stopped producing them. Without them my eyes hurt like hell ( I can barley stand to look at my computer screen) an d Im gradually going blind. The thing is, I CAN get them. If I fly all the way to DC. From WA that is VERY expensive. My grandma CAN afford if she cuts back on spending, but I hate that she would have to do that. Shes done far more than enough for me already.
I also know that there are other people who need these eyedrops who will never be able to afford it in a million years and the eyedrops wont be certifed for atleast another ten years. Theyll all lose thier eyesight if they cant get enough money for the trip.

SNIP

What is DU? Thax very much, that should be a bighelp no matter who they are. Im not sure how good of a case it is. Like I said they ARE available, but it costs an arm and a leg. To be more specific, we have to fly to DC to take part in some research study At the NHI. ( The eye drops are 'experimental, however they have been for years and so far theyve done nothing but good) it would be a big help even to get it so that we can some how take part in that study locally and get the eydrops shipped to us..something like that.

SNIP

Thier called Cystamine eyedrops. What they do is deterioriate cystine crystals which up build in the eyes and damage the cornias and eventually lead to blindness.

[renate]

1. have you tried a compounding pharmacy?

I would think Washington state would be full of them. Oregon's got 'em. There's one in Lloyd Center right across the border in Portland.
281-4161, area code 503. Very nice people.
Good luck.

[HysteryDiagnosis]

2. If you know why they are building up... or if it is a genetic issue,

please post...

[HysteryDiagnosis]

3. From what I am getting.... there is kidney involvement? n/t

[SariesNightly]

7. I found some info on the disease

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WHAT ARE THE SYMPTOMS?

There are three clinical forms of cystinosis. Infantile (or nephropathic) cystinosis; late-onset cystinosis; and benign cystinosis. The latter form does not produce kidney damage. Infantile and late-onset cystinosis differ in the age of appearance of the first symptoms and in the rapidity of the clinical course. Infantile cystinosis is usually diagnosed between 6 and 18 months of age with symptoms of excessive thirst and urination, failure to thrive, rickets, and episodes of dehydration. These findings are caused by a disorder called renal tubular reabsorb nutrients and minerals. As a consequence, these important molecules are lost in the urine. Children with cystinosis also have crystals in their eyes (after one year of age) and an increased level of cystine in their white blood cells. Without specific treatment, children with cystinosis develop end-stage renal failure, i.e., lose their kidney function, at approximately 9 years of age.

If cystinosis patients receive a kidney transplant and reach adulthood, their new kidney will not be affected by the disease. However, without cysteamine treatment (see below), they can develop complications in other organs due to the continued accumulation of cystine throughout the body. These complications can include muscle wasting, difficulty swallowing, diabetes, heypthroidism, and blindness. Not all older patients develop these problems, however.

CAN CYSTINOSIS BE TREATED?

Kidney transplantation has proven very helpful in patients with cystinosis, and cysteamine therapy should be considered to try to prevent the late complications of the disease (see above)..

For both young children with cystinosis and older patients with a kidney transplant, cysteamine eyedrops may be available to remove the corneal cystine crystals. However, these are not yet approved by the FDA.
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Cysteamine eyedrops works better than oral because the oral type doesn't reach the eye area, but for some reason they are still not approved, I'm not sure about this but testing has been done since 1986.. and they are still testing!?

[Spinzonner]

4. Has he tried contacting these people for advce/info

http://www.cystinosis.org

[pk_du]

5. Will try and see if these guys are still in business for this stuff

http://archive.cystinosis.org/Cysteamine%20Eye%20Drops.pdf

[pk_du]

6. Gotta be treament refs on here too...

http://www.cystinosis.com/

[pk_du]

8. Two great info resources from WebMD

http://my.webmd.com/hw/health_guide_atoz/nord59.asp

http://my.webmd.com/hw/health_guide_atoz/shc29cys.asp

[riverwalker]

9. this fellow has a grant for research

he can perhaps help with getting the drops for you.

Title: Cysteamine Hydrochloride Eye Drops - New Formulation
Principal Investigator: Dr. Edward Lemanowicz
Institution: Sigma-Tau Pharmaceuticals, Inc.
City / State: Gaithersburg, MD

[HysteryDiagnosis]

10. FYI, NAC is something you may want to learn about, it is

available everywhere, and is explained below. NAC has many potential beneficial properties... some of which you can read about here:

http://www.wholehealthmd.com/refshelf/substances_view/1,1525,809,00.html

http://www.lef.org/magazine/mag2003/may2003_report_pet_02.html

NAC is a form of the amino acid, cysteine, with multiple kidney-protective actions. The research on what NAC can do for the kidney (and the liver) is scientifically well-documented. In one recent study, researchers in Italy demonstrated that NAC normalizes creatinine levels in rodents with kidney failure. It reverses inflammation and restores filtration. Similar findings are reported by French researchers when NAC is given as a pretreatment before experimental kidney damage in rats. Without NAC, there is a 68% reduction in the ability of the kidneys to do their job. With it, the loss of kidney function is only 29%. A third study confirms that an infusion of NAC before and after experimental kidney damage doubles the kidney filtration rate. NAC is strong medicine for the kidneys. It has no adverse side effects, even when small animals like cats are given a human dose.

http://dnrc.niddk.nih.gov/dnrc/Program_93-95/NEI.htm

Age-related cataract and macular degeneration are the major causes of visual impairment and blindness in the aging U.S. population. The NEI supports research on the role of general nutrition and of certain micronutrients-in particular, vitamins and trace minerals with antioxidant capabilities-on the development and worsening of these two eye disorders. NEI-supported scientists also are evaluating the effect of nutritional factors on other visual disorders, such as retinopathy of prematurity, cystinosis, retinitis pigmentosa, and diabetic retinopathy.


http://www.bloodjournal.org/cgi/content/full/91/11/4373

Next, we wanted to test the efficacy of reducing agents, or antioxidants, which are in clinical use for other purposes. Cysteamine bitartrate converts cystines to cysteines and cysteine-cysteamine mixed disulfides. It has been approved by the Food and Drug Administration (FDA) for the treatment of nephropathic cystinosis and appears to maintain normal renal functions in patients receiving the drug.20 Cysteamine was ineffective in blocking ISC formation (at 1 to 50 mmol/L) and actually caused increases in the number of ISCs at higher concentrations (150 mmol/L and 200 mmol/L) (data not shown).

Succimer (meso 2,3-dimercaptosuccinic acid) and dimercaprol are used clinically as chelators of heavy metals, such as lead.21 Succimer was ineffective at blocking ISC formation in vitro (at 1 to 30 mmol/L) and again caused increases in ISCs at concentrations of 100 to 200 mmol/L (data not shown). Dimercaprol caused a statistically significant decrease in ISCs at 10 µm concentration (-20.5%), but at higher concentrations (0.1 to 1.0 mmol/L) caused increases in percent ISCs (data not shown).

NAC is an antioxidant and is converted intracellularly to L-cysteine, which is a precursor to reduced glutathione. We felt that the combined activities of this drug might make it particularly efficacious in blocking ISC formation in vitro. As shown in Table 2, NAC caused statistically significant decreases in percent ISCs at concentrations of 250 µmol/L to 20 mmol/L NAC. At 20 mmol/L NAC, there was a reduction of 35.7% ISCs. NAC (20 mmol/L) had no effect on cell lysis during the 16 hours of cycling, as evidenced by identical concentrations of hemoglobin (measured by absorbance at 430 nm) in the incubation mixtures ± NAC (data not shown). Figure 1 shows a typical smear of ISCs formed in vitro in the presence (Fig 1C) or absence (Fig 1B) of 20 mmol/L NAC.


http://www.naturalopinion.com/nmp/nmp5/Nacecyst.htm
Toxicity and Side Effects:

* Genetic disorder cystinosis, or Fanconi's, causes the body to build up harmful levels of L-cystine (not cysteine)

Copyright 1998 - 2005 by L. Vicky Crouse, ND and James S. Reiley, ND. All rights reserved (ISSN 1527-0661).