Gary Jacob, CEO of Callisto Pharmaceuticals, Inc., is leading a scientific effort to develop a new orphan drug called Atiprimod for patients with multiple myeloma. The FDA recently granted Callisto orphan drug designation, providing financial incentives to continue the costly development process. Orphan drugs are providing new doses of hope where there was previously little or none at all. According to the National Organization for Rare Disorders, an estimated 6,000 orphan diseases affect approximately 25 million people in the United States. The FDA program went into effect in 1983, paving the way for new drugs for orphan diseases, but large pharmaceutical companies have been largely absent.
Small pharmaceutical companies with promising drug candidates have been drawn into the breach by the FDA's orphan drug incentives, which include grants, seven years of marketing exclusivity, and tax breaks. Amyotrophic lateral sclerosis (ALS), multiple myeloma, and Huntington's disease all affect fewer than 100 people. Dr. Kenneth C. Anderson, a member of Callisto's Medical Advisory Board, is excited to see Atiprimod enter clinical trials for evaluation in patients with multiple myeloma, as it has the potential to help patients who have not responded to other medications. Phase I/IIa trials are set to begin later this month.
Gary Jacob received distressing news during a visit to his doctor - not about himself, but about a doctor's friend.
The doctor's friend fell and broke a rib while playing with one of his children. That was bad enough, but during the hospital examination, the father was given a shocking and completely unexpected diagnosis: he had multiple myeloma, a bone marrow blood cancer.
The diagnosis was equivalent to a death sentence.
Jacob was aware of the anguish experienced by multiple myeloma patients. The disease is incurable and nearly always fatal, making it one of the rare diseases with few, if any, treatments available. They are referred to as "orphan" diseases because the patient populations are small and commercial development of a drug is deemed economically unattractive.
Mr. Jacob was aware because, as CEO 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 patients with multiple myeloma.
"My father's illness drove home to me the importance of what we're doing," Mr. Jacob says. "Everyone agrees that more drugs are needed to treat multiple myeloma. There are people dying with no real hope because there is no effective treatment for all patients."
Orphan drugs are providing new doses of hope where there was previously little or none at all. Pharmaceutical companies developed ten drugs for orphan diseases in the decade preceding the start of the federal Food and Drug Administration's orphan drug program. The FDA reports that nearly 250 new drugs have been developed and approved in the decades since, with hundreds more in the works.
Atiprimod is one of the drugs making its way to the market. The FDA recently granted Callisto orphan drug designation, providing the company with financial incentives to continue the costly development process.
The program covers drugs for orphan diseases with fewer than 200,000 patients.
According to the National Organization for Rare Disorders, an estimated 6,000 orphan diseases affect approximately 25 million people in the United States.
Cystic fibrosis, HIV-related complications, Gaucher's disease, hemophilia, and rare cancers were among the orphan diseases without effective treatments until the FDA program went into effect in 1983, paving the way for new drugs for patients with these diseases.
Large pharmaceutical companies have been largely absent from the efforts.
According to Dr. John McCormick, deputy director of the orphan drug program, only 15% of applications for orphan drug designation have come from larger pharmaceutical companies.
The reason is that investors are expecting negative investment returns.
Small pharmaceutical companies with promising drug candidates have been drawn into the breach by the FDA's orphan drug incentives, which include grants, seven years of marketing exclusivity, and tax breaks.
While the future looks brighter, developing drugs for orphan diseases remains a daunting task.
Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, affects 30,000 Americans each year, with 8,000 new cases diagnosed; Huntington's disease also affects 30,000 people.
According to the National Institutes of Health, some diseases affect fewer than 100 people.
Multiple myeloma affects an estimated 50,000 people, with 15,000 new cases diagnosed each year. The FDA approved Velcade, a new drug for patients with the disease, last year. However, there are still many patients with multiple myeloma who do not have access to treatment.
Dr. Kenneth C. Anderson, who was involved in the preclinical development and clinical trials of Velcade and is now a member of Callisto's Medical Advisory Board, is one of the experts who believes that more drugs to treat multiple myeloma are needed.
"He is very excited to see Atiprimod enter clinical trials for evaluation in patients with multiple myeloma," Jacob said of Anderson. "He believes it has the potential to help patients who have not responded to other medications."
Dr. Anderson is the director of the Dana-Farber Cancer Institute's Jerome Lipper Multiple Myeloma Center in Boston, MA, and a Professor of Medicine at Harvard Medical School.
Atiprimod Phase I/IIa trials are set to begin later this month.
Dr. Donald Picker, Senior Vice President of Drug Development at Callisto, stated that studies of Atiprimod conducted in collaboration with scientists from the National Cancer Institute have been very promising.
"In essence, we've demonstrated in these preliminary studies that Atiprimod has the potential to intervene with cancer cells and tumors in three ways: by inhibiting their formation, programming their death, and limiting their ability to grow blood vessels required for survival. These findings suggest that Atiprimod may represent a novel class of compounds for development as a therapeutic intervention in human cancers "Dr. Picker stated.
No comments:
Post a Comment