| |
| |
|
Medicine Information |
|
|
|
Orphan Drugs: Hope Where There Is Little or No Hope
NEW YORK, N.Y., February 18, 2004 - On a visit to his doctor, Gary Jacob received distressing news - not about himself, but a friend of the doctor's. While playing with one of his children, the doctor's friend fell and broke a rib. That was bad enough, but during the examination at the hospital, the father was hit with a startling and totally unexpected diagnosis - he had a disease known as multiple myeloma, a bone marrow blood cancer. The diagnosis was nothing less than a death sentence. Jacob knew of the anguish of multiple myeloma patients. The disease is incurable and nearly always fatal, one of the rare diseases that have few, if any, available treatments. They are known as "orphan" diseases, shunned by most drug-makers because the patient populations are small and commercial development of a drug is seen as economically unattractive. Mr. Jacob was aware because, as Chief Executive Officer of Callisto Pharmaceuticals, Inc., a small Manhattan-based biopharmaceutical company, he is leading a scientific effort to develop a new orphan drug called "Atiprimod" for multiple myeloma patients. "The father's disease brought home to me that what we are doing is really important," says Mr. Jacob. "Everyone agrees we need more drugs to treat multiple myeloma. There are people out there dying without real hope because of a lack of effective treatment for all patients." In steadily increasing numbers, orphan drugs are providing new doses of hope where little or none at all existed. In the decade before the inception of the federal Food and Drug Administration's orphan drug program, 10 drugs were developed by pharmaceutical companies for orphan diseases. In the decades since, the FDA says nearly 250 new drugs were developed and approved, and hundreds more are in the pipeline. Atiprimod is one of those wending its way toward the marketplace. Callisto recently obtained orphan drug designation from the FDA, providing the company with financial incentives to continue the costly development process. The program covers drugs for orphan diseases with patient populations under 200,000. The National Organization for Rare Disorders reports about 25 million people in the United States suffer from an estimated 6,000 orphan diseases. Diseases such as cystic fibrosis, complications affecting HIV-infected people, Gaucher's disease, hemophilia and rare forms of cancer were among the orphans without effective medicines until the FDA program went into effect in 1983 and paved the way for new drugs for patients with these diseases. Large drug-makers have been largely missing from the efforts. According to the orphan drug program's deputy director, Dr. John McCormick, only 15% of applications for orphan drug designation have come from the larger pharmaceutical companies. The reason: expectations of unfavorable investment returns. The FDA orphan drug incentives - grants, seven years of marketing exclusivity and tax breaks - have drawn small pharmaceutical companies with promising drug candidates into the breach. While the future is brighter, the task is still daunting to develop drugs for orphan diseases. Amyotrophic lateral sclerosis (ALS), or Lou Gehrig's disease, affects 30,000 Americans with 8,000 new cases diagnosed annually; Huntington's disease also affects about 30,000 patients. Some diseases affect fewer than 100 patients, according to the National Institutes of Health. An estimated 50,000 patients have multiple myeloma with 15,000 new patients diagnosed each year. Last year, the FDA approved a new drug Velcade for patients with the disease. However, there are still a number of multiple myeloma patients with no treatment available. Dr. Kenneth C. Anderson, who played a major role in the preclinical development and clinical trials of Velcade and is now a member of Callisto's Medical Advisory Board, is among the experts who see a need for more drugs to treat multiple myeloma. "He is excited to see Atiprimod enter clinical trials for evaluation in multiple myeloma patients," Jacob said of Anderson. "He believes it has an opportunity to help patients who have not responded to other drugs. " Dr. Anderson is director of the Jerome Lipper Multiple Myeloma Center of the Dana-Farber Cancer Institute in Boston, MA, and Professor of Medicine at Harvard Medical School. The Phase I/IIa trials for Atiprimod are slated to begin later this month. Dr. Donald Picker, Callisto's Senior Vice President of Drug Development, said studies of Atiprimod in collaboration with scientists at the National Cancer Institute have been very promising. "In essence, we've shown in these early studies that Atiprimod has the potential to intervene with cancer cells and tumors in three ways - by inhibiting their formation, by programming their death and by limiting their ability to grow blood vessels necessary for their survival. Taken together, these findings suggest that Atiprimod could potentially represent a novel class of compounds for development for therapeutic intervention in human cancers," said Dr. Picker. About The Author Alex Michelini is a former award-winning reporter/editor at the New York Daily News. His credits include nomination for a Pulitzer Prize for a series on medical costs. Among his honors, Mr. Michelini received the Deadline Club Award, the Page One Award, the Associated Press Award and the New York Press Club Award (twice). He is the founder of Alex Michelini Public Relations, and has developed and collaborated on articles appearing in the New York Times, the Wall Street Journal, the London Times, the New York Post, the New York Daily News, Bloomberg Radio & TV, CNNfn, WCBS Radio & TV. WINS radio, the Christian Science Monitor, Fox TV, Reuters, Newsday and other media outlets.
MORE RESOURCES:
Google News |
|
|
|
RELATED ARTICLES
Molluscum Contagiosum Virus (MCV) Treatment Information - But First, What Is Molluscum? Molluscum contagiosum treatment is certainly possible with the correct treatment information.Many people are told that molluscum contagiosum must be left to run its course (even by dermatologists). 22 Inside Tips on How You Can Make Your Arthritis Medicines Work Twice as Effectively in Half the.. This report will give you 22 important tips to make sure that you're getting the very best out of your arthritis treatment program.1. Lunesta Withdrawal Lunesta is a newly released sleeping pill. Marketed as a revolutionary sleep aid, Lunesta is the only medication approved by the FDA for long term treatment of insomnia symptoms. VIOXX Lawsuits Vioxx is a prescription medication that has been used primarily in the treatment of signs and symptoms of arthritis, but has also been prescribed to treat acute pain in adults and menstrual pain in women. It is a Cox-2 selective nonsteroidal anti-inflammatory drug or NSAID. Lunesta Memory Problems Lunesta may be the only sleeping pill that is FDA approved for long-term use, but it still has some of the same effects as other sleep aids. Ambien, Sonata and others have all been known to cause this problem. Whats a Good Arthritis Medicine for Me, Doctor? Pain is the body's warning signal that something is wrong. If the pain is due to overuse or arthritis there are many things to consider. New Company Helps Another Medical Condition Become: "Acceptable" Dinner Conversation Recently once taboo subjects like plastic surgery have become acceptable dinner conversation. Medical conditions are no longer whispered to friends, but have instead become part of the dinner table conversation with full blown discussions on Botox, erectile dysfunction and the feared colonoscopy. Treating Neuromas - Ten Tips A neuroma is an inflamed nerve. In the foot, the most common place for a neuroma is between the third and fourth toes. Irritable Bowel Syndrome - Cybill Shepherd Reveals All Cybill Shepherd has revealed that she suffers from irritable bowel syndrome (IBS). The star of Moonlighting and Taxi Driver says that she has struggled with her symptoms for many years, and is now hoping to raise awareness of IBS and encourage sufferers to talk more openly with their doctors. Physicians, Chiropractors and Physical Therapist Agree on a New Treatment for Low Back Pain One of the most prevalent and difficult health conditions to treat in the physical medicine is low back pain. The difficulty in treating low back pain comes from the fact that there are numerous causes including herniated discs, bulging discs, degenerative discs, muscle spasms, facet arthritis, and sciatica. Medication Compliance Kit ... A Life Saver Pill Proof LLC introduces the first, easy to use medication compliance kit for consumers and medical professionals. Owner, Jane Langdon, has a sister in law with Parkinson's Disease. How to Remove the Psychological Addiction to Smoking - and Start to ENJOY Giving Up Smoking! Consider this:Already since 1972, over 60 million people in the USA alone have successfully quit smoking!At one stage over 60% of the adult population was addicted to this drug. Today it is 28% and dropping. Medical Billing Software Takes the Stage Medical Billing SoftwareThe practice is running smoothly, with patients coming in and out with smiles on their faces because of the great service your practice is providing. At the end of the day, you review the office secretary's billing records and realize you are behind, again. After Viagra, Its Now a Premature Orgasm Pill for Women Pfizer's exciting drug history traces back to the most popular Viagra drug, which treats erectile dysfunction in men. Now, they are gearing up to hit the bull's eye with a new premature orgasm pill for women. "Get Rid of Tendonitis ? Now!" Advice From An Expert? Tendons are ropes of fibrous tissue that connect muscles to bones. It is this connection that permits joint motion. The Trouble with Tranqs There's a whole group of drugs out there called benzodiazepines.They are tranquillisers, or sedatives. Treatment for High Blood Pressure The treatment of high blood pressure is one of the most important areas of modern medicine. Good quality care and blood pressure treatment can reduce the risk of death, the risk of stroke and the risk of heart attack or coronary disease. Advanced Surface Ablation In patients with corneal thickness below a certain minimum, performing LASIK would be unwise, because the "foundation" may not go back to its original stability. Our colleagues from around the world, agree on when not to do LASIK, like when the cornea is very steep or very flat. Check Out The Wonders Of Tea Tree Oil Tea tree oil is the essential oil comes from the Australian plant Melaleuca alternifolia. This species is unique to Australia and native to Northern New South Wales. Common Drugs and Their Uses The five drugs that are discussed in this article are Ibuprofen which is a non-steroidal anti-inflammatory drug (NSAID) that is commonly used for relief of Arthritis, Warfarin which is an anticoagulant drug commonly prescribed by doctors to treat venous thrombosis, pulmonary embolism, to treat or prevent dangerous blood clotting in people with arterial fibrillation and to prevent strokes, a benzodiazepine drug called Diazepam which is commonly prescribed by doctors for relieve nervousness, anxiety, and anxiety disorders, a sulfonylureas drug called Glipizide which is used to help control blood sugar levels and used to treat type 2 diabetes and a thyroid medication called Armour Thyroid which is prescribed for the treatment of hypothyroidism.Arthritis is a common illness in today's society; doctors prescribe drugs called non-steroidal anti-inflammatory drugs (NSAIDs) for this condition.
|
| home | site map | Dr. Thad Thomas |
| © 2006 |
