Advances in the Treatment of DISEASES OF THE DEVELOPING WORLD
Some diseases considered rare in the United States are actually very common around the world. For these diseases, new treatments being developed in the United States are helping millions of men, women, and children in the developing nations. Even before the horrors of Hurricane Katrina spotlighted waterborne hazards, here in the world's wealthiest nation, handfuls of Americans have been affected every year by outbreaks of illnesses that are leading causes of death in developing countries.
Cryptosporidiosis and Giardiasis
Neurocysticercosis and Hydatid
Tuberculosis
Cryptosporidiosis and giardiasis, parasitic infections that cause diarrhea, mainly affect people living in developing countries around the world, but even in the United States there are cases every year.65
The parasite cryptosporidium can be found in drinking or recreational water and contaminated food. Once it enters the body it lives in the intestinal tract, but outside the body it can survive for long periods because of a tough outer shell. The parasite causes vomiting and nausea, dehydration, stomach pain, and weight loss.66 Giardiasis is an infection caused by a single-celled parasite called Giardia lamblia or, simply, Giardia. If left untreated in children, complications from these parasites include malnutrition, impaired growth, and death.67
PHARMACEUTICAL ADVANCES
Killing Parasites That Cause Dangerous Waterborne Illnesses
Nitazoxanide, an antiprotozoal approved in 2002, is used to treat children up to 11 years of age with diarrhea caused by cryptosporidiosis and giardiasis. It is the first new drug approved for giardiasis in over 40 years.68 Nitazoxanide is the only drug approved for treating cryptosporidiosis and the only drug approved for giardiasis for children one to 11 years of age. Nitazoxanide works by stopping the growth of the infecting organisms.69 The FDA estimates that nitazoxanide could help 2,500 people in the United States.70
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Both neurocysticercosis and hydatid disease are parasitic diseases from worms and affect approximately 300 Americans annually.71 Neurocysticercosis, caused by pork tapeworm larvae (eggs), is usually spread through contaminated water or food. It causes cysts to develop throughout the body and can cause headaches and seizures if they develop in the brain tissue.72 It is the most common infectious cause of seizures worldwide.73
People can contract hydatid disease, also known as Echinoccocus, by eating contaminated food containing Echinococcus granulosus worm eggs. The infection causes cysts in the liver first but they can also develop in the lungs, brain, bones, and other organs. If the cysts rupture, severe illness results, including fever, low blood pressure, and shock.74,75
PHARMACEUTICAL ADVANCES
First Treatment for Two Kinds of Cyst-Causing Worms
In 1996, albendazole became the first treatment approved to treat both neurocysticercosis and hydatid.76 Albendazole kills the worm by preventing it from absorbing sugar (glucose), which it needs to survive.77 This drug was found to destroy cysts in 40 to 70 percent of patients with neurocysticercosis. Among patients with hydatid disease, albendazole eliminates the cysts in about 30 percent of patients and reduces their size in an additional 40 percent of patients.78
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Tuberculosis (TB) is still the most common cause of death and morbidity in the world, killing 30 million people worldwide in the 1990s, according to the WHO.79 In the United States, fortunately, it is a rare condition affecting just 15,000 people in 2003.80
TB is a contagious bacterial infection that primarily affects the lungs but can attack any part of the body. It is caused by Mycobacterium tuberculosis and is typically spread through the air from a cough or sneeze of someone who is infected with the bacteria. If not treated properly, TB can be fatal.
Patient Perspective: A Disease from Antiquity
PHARMACEUTICAL ADVANCES
Modern Treatment for Ancient "Consumption"
In 1998, the first new drug to treat TB in 25 years, rifapentine, was approved for treatment of pulmonary tuberculosis. It works by stopping the cell multiplication process of the bacteria and is used in combination with other anti-tuberculosis drugs to treat pulmonary tuberculosis.81 Before rifapentine was approved, the health care community was worried about resistance to older drugs that was developing over time. This treatment offers another tool to fight tuberculosis. Also, the dosing regimen for this drug is simpler than other drugs, so patients are more likely to take it.82
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In the last decade (1995-2005), over 160 orphan drugs were approved, compared with 108 from 1984 to 1994 and only 10 in the decade prior
to the passage of the Orphan Drug Act in 1983.83 |
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Endnotes:
65 U.S. Department of Health and Human Services, Centers for Disease Control and Prevention, http://www.cdc.gov/mmwr/preview/mmwrhtml/ss4907a1.htm.
66 Centers for Disease Control and Prevention, What Is Cryptosporidium?, http://www.cdc.gov/ncidod/dpd/parasites/cryptosporidiosis/factsht_cryptosporidiosis.htm# (accessed 14 July 2005).
67 Centers for Disease Control and Prevention, http://www.cdc.gov/ncidod/dpd/parasites/giardiasis/factsht_giardiasis.htm (accessed 14 July 2005)
68 National Organization for Rare Disorders, Giardiasis, http://www.rarediseases.org/search/rdbdetail_abstract.html?disname=Giardiasis (accessed 1 August 2005).
69 National Library of Medicine, MedlinePlus Drug Information, Nitazoxanide, http://www.nlm.nih.gov/medlineplus/druginfo/medmaster/a603017.html (accessed 14 July 2005).
70 Food and Drug Administration, "FDA Approves New Treatment for Parasitic Infections in Pediatric Patients," 2 December 2002, http://www.fda.gov/bbs/topics/ANSWERS/2002/ANS01178.html (accessed 3 October 2005).
71 Food and Drug Administration, "First Treatment Approved for Rare Parasitic Infections," 13 June 1996, http://www.fda.gov/bbs/topics/ANSWERS/ANS00738.html (accessed 1 August 2005).
72 Centers for Disease Control and Prevention, Fact Sheet: Cysticercosis, http://www.cdc.gov/ncidod/dpd/parasites/cysticercosis/factsht_cysticercosis.htm (accessed 3 October 2005).
73 Food and Drug Administration, "First Treatment Approved for Rare Parasitic Infections," op. cit.
74 National Library of Medicine, MedlinePlus Medical Encyclopedia, Echinococcus, http://www.nlm.nih.gov/medlineplus/print/ency/article/000676.htm (accessed 3 October 2005).
75 Food and Drug Administration, "First Treatment Approved for Rare Parasitic Infections," op. cit.
76 Ibid.
77 National Library of Medicine, MedlinePlus Drug Information, Albendazole (Systemic), http://www.nlm.nih.gov/medlineplus/druginfo/uspdi/202668.html (accessed 3 October 2005).
78 Food and Drug Administration, "First Treatment Approved for Rare Parasitic Infections," op. cit.
79 Food and Drug Administration, "FDA Clears Tuberculosis Drug for Marketing," 23 June 1998, http://www.fda.gov/bbs/topics/ANSWERS/ANS00880.html (accessed 15 September 2005).
80 American Lung Association, "Tuberculosis Fact Sheet," American Lung Association, http://www.lungusa.org/site/pp.asp?c=dvLUK9O0E&b=35804 (accessed 15 September 2005).
81 S. Reents et al., Clinical Pharmacology, Gold Standard Multi-media, Inc., Rifapentine, http://cp.gsm.com (accessed 3 August 2005).
82 Food and Drug Administration, "FDA Clears Tuberculosis Drug for Marketing," 23 June 1998, http://www.fda.gov/bbs/topics/ANSWERS/ANS00880.html (accessed 15 September 2005).
83 Food and Drug Administration, Office of Orphan Products Development, "List of All Approved Orphan Products Through the Year 2005," 13 May 2005, http://www.fda.gov/orphan/designat/allap.rtf (accessed 7 September 2005).
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