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Cystagon

Cystinosis is a hereditary disorder of the renal tubules characterized by the presence of carbohydrates and amino acids in the urine, excessive urination, and low blood levels of potassium ions and phosphates. The body accumulates the amino acid cystine within cells. Excess cystine forms crystals that can build up and damage cells. These crystals negatively affect many systems in the body, especially the kidneys and eyes. more...

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Cause

It is caused by abnormal transport of the amino acid cystine from lysosomes of all tissues, resulting in a massive intra-lysosomal cystine accumulation. Via an as yet unknown mechanism, lysosomal cystine appears to amplify apoptosis such that cells die inappropriately, leading to loss of renal epithelial cells, accounting for the renal Fanconi syndrome, and simlar loss in other tissues can account for the short stature, retinopathy, and other features of the disease.

Symptoms

There are three distinct types of cystinosis each with slightly different symptoms: nephropathic cystinosis, intermediate cystinosis, and non-nephropathic or ocular cystinosis. Infants affected by nephropathic cystinosis initially exhibit poor growth and particular kidney problems (sometimes called renal Fanconi syndrome). The kidney problems lead to the loss of important minerals, salts, fluids, and other nutrients. The loss of nutrients not only impairs growth, but may result in soft, bowed bones (hypophosphatemic rickets), especially in the legs. The nutrient imbalances in the body lead to increased urination, thirst, dehydration, and abnormally acidic blood (acidosis). By about age two years, cystine crystals may be present in the cornea. The buildup of these crystals in the eye causes an increased sensitivity to light (photophobia). Untreated children will experience complete kidney failure by about age 10 years. Other signs and symptoms that may occur in untreated patients include muscle deterioration, blindness, inability to swallow, diabetes, and thyroid and nervous system problems.

The signs and symptoms of intermediate cystinosis are the same as nephropathic cystinosis, but they occur at a later age. Intermediate cystinosis typically begins to affect individuals from age 12 years to age 15 years. Malfunctioning kidneys and corneal crystals are the main initial features of this disorder. If intermediate cystinosis is left untreated, complete kidney failure will occur, but usually not until the late teens to mid twenties.

People with non-nephropathic or ocular cystinosis do not usually experience growth impairment or kidney malfunction. The only symptom is photophobia due to cystine crystals in the cornea.

It is currently being researched at UC San Diego, Tulane University School of Medicine, and at the National Institutes of Health in Bethesda, Maryland.

Genetics

The cause of cystinosis is due to a mutation in the gene CTNS which codes for cystinosin, the lysosomal cystine transporter. Symptoms are seen about 6-18 months of age with profound polyuria ( excessive urination), followed by poor growth, photophobia, and ultimately kidney failure by age 10 years in the nephropathic form. It is important for the child to see a biochemical geneticist and pediatric nephrologist to begin treatment with cysteamine as early as possible. Cysteamine decreases the amount of cystine stored in lysosomes and correlates with conservation of renal function and improved growth. Cysteamine eyedrops remove the cystine crystals in the cornea that can cause photophobia if left unchecked.

Read more at Wikipedia.org


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1994 Ad
From American Family Physician, 3/1/95 by Peter H. Rheinstein

The U.S. Food and Drug Administration approved 85 new drugs and licensed biological products last year, while setting new records for review time and the number of approvals of supplemental applications. The significantly improved performance of the Centers for Drugs and Biologics reflect the effects of the user fee program.

The user fee program, authorized by the Prescription Drug User Fee Act of 1992, enables the FDA to collect fees from pharmaceutical companies and use the proceeds to accelerate the review process at the Center for Drug Evaluation and Research and the Center for Biologics Evaluation and Research. In its recent report to Congress on user fees, the FDA reported that it has met all interim user fee goals to date, including the elimination of overdue drug and biologic submissions. All submissions overdue as of October 1, 1992, are scheduled to be cleared through the FDA by July 2, 1995, and the agency reported to Congress that only nine of 696 overdue submissions remain, and these will be cleared by the July 2nd deadline.

In terms of speeding up the approval process, the most important gains for both drugs and biologics were in median approval times. The median time for the 23 approved vaccines and other biological products in 1994 was 12.2 months, compared with a median time of 23.4 months for similar approvals in 1993. The median time for the 62 new drug approvals in 1994 was 19.0 months, a period 21 percent shorter than the drug approval time required in 1993.

More than one-third (22) of the approved drugs were new molecular entities--products that contain an active substance never before marketed in any form in the United States. The median review time for approval of new molecular entities in 1994 was 17.5 months, compared with 23.0 months in 1993.

Fourteen of the new molecular entities were applications submitted after the start of the user fee program. These applications were approved in a median time of 12.1 months.

Seventeen of the approved new drug applications, including 13 new molecular entities, were in the "priority" classification, which is granted to medications that are expected to have important new therapeutic value. The median time for approval of these products was 15.0 months. The median approval time was 10.4 months for the 10 priority products filed under the user fee program. For seven of the 13 priority new molecular entities, the approval in the United States represents the first approval anywhere in the world. Two of the 13 priority drugs were from foreign manufacturers and were first approved in their countries of origin. Approval in the United States represents their second approval.

The 22 new molecular entities approved in 1994 are listed in Table 1. Two of these products (cysteamine bitartrate [Cystagon] and imiglucerase [Cerezyme]) have been designated "orphan drugs." (Orphan drug designation is available for products used to treat conditions for which the patient population in the United States would be less than 200,000 per year. The FDA usually provides assistance and special incentives to manufacturers of these products.

[TABULAR DATA 1 OMITTED]

Cysteamine is used to treat nephropathic cystinosis, a rare disease characterized by the build-up of the amino acid cystine in the kidneys. The disease affects about 200 people in the United States. During the past 15 to 20 years, most children with the condition had to undergo kidney transplants. Before the availability of renal transplantation, most children with nephropathic cystinosis died in the first decade of life. Studies of cysteamine showed that the drug can stop or slow the progression of renal failure.

The FDA Office of Orphan Products Development has supported research into and development of cysteamine since 1987. Under the orphan grants program, the agency has provided more than $900,000 to researchers at the University of California in San Diego, the National Institutes of Health and the University of Michigan to study drug efficacy in clinical trials.

Vinorelbine (Navelbine) was approved for treatment of non-small cell lung cancer. It is indicated for use alone or in combination with cisplatin for ambulatory patients with advanced non-small cell lung cancer who are not surgical candidates because of the extent of the disease. Before approval of vinorelbine, more than 390 patients had received the drug under a treatment IND (investigational new drug) authorized by the FDA. The agency's treatment IND regulations allow drug developers to provide earlier and wider access to promising investigational therapies for patients with serious or immediately life-threatening conditions for which there are no comparable or satisfactory alternative treatments.

Four new biological products are listed in Table 2. Two of these, pegaspargase (Oncaspar) and abciximab (ReoPro), represent completely new entities. Also included are the first single-dose typhoid vaccine and the first home-collection device to obtain oral fluid to test for human immunodeficiency virus type 1.

[TABULAR DATA 2 OMITTED]

The new drug abciximab is approved for use in the prevention of acute ischemic complications following angioplasty, when blood clots cause abrupt closure of the treated coronary artery. When injected, abciximab binds to platelets and helps prevent the formation of clots. Abciximab is the second therapeutic monoclonal antibody to be licensed. The first, Muromonab CD3, was licensed in 1986 and is indicated for the treatment of acute kidney transplant rejection, and heart and liver transplant rejection in patients who are resistant to the standard steroid therapy

Also in 1994, a nonprescription-strength of naproxen was cleared for over-the-counter marketing as an analgesic and antipyretic. The new preparation, available as a 200-mg tablet, is sold under the trade name Aleve. Naproxen has been sold as a prescription drug under the trade names of Anaprox and Naprosyn since 1980 and 1976, respectively.

Dr. Rheinstein, a family physician, is director of medicine staff at the U.S. Food and Drug Administration.

COPYRIGHT 1995 American Academy of Family Physicians
COPYRIGHT 2004 Gale Group

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