The children of a 62-year-old Indiana woman are trying to pressure a Maryland biotech company into treating their mother's pancreatic cancer with an experimental drug not yet approved by the U.S. Food and Drug Administration.
The three daughters of Connie Loughman held a news conference at their parents' Indianapolis home yesterday, pleading for access to the drug. They have launched a video on YouTube devoted to their mother's plight and set up a Web site asking people to e-mail executives at Gaithersburg's GenVec Inc., which is testing the promising cancer treatment.
Company executives say they cannot help until they get more clinical trial information -- maybe not even then. GenVec's entire drug supply is committed to trial participants.
"We are very sympathetic to this family. We know how devastating this diagnosis is, and we're doing what we can to move the drug forward," GenVec Chief Financial Officer Douglas Swirsky said yesterday. "But as a company, we've elected to defer establishing a compassionate-use program until additional data comes out."
The Loughmans are asking for what is known as "compassionate use" treatments -- allowable under a provision within federal regulations -- of GenVec's TNFerade, which is in late-stage clinical testing.
Over the years, more than 100,000 people in life-threatening situations have been allowed access to developing drugs, according to the FDA, which gets about 660 requests for compassionate-use treatments each year. But the companies that make the drugs are often reluctant to get involved in those programs, worried about increasing already high development costs, doing unnecessary damage to patients or falsely raising people's hopes.
Columbia's Osiris Therapeutics offers one of its developing stem cell treatments to children. But the company no longer offers it to adults because it is too expensive.
Navdeep S. Jaikaria, a biotechnology analyst with Rodman & Renshaw in New York, points out that GenVec's TN Ferade is nearing the end of its testing, and he worries that changing the trials could interfere with the drug's commercialization.
"The thing is, you don't know," he said.
While Loughman's daughters understand the business logistics involved, it does little to mitigate their concerns. They've offered to pay for the drug and release GenVec from all liability, and now they've taken their campaign public.
"We're just trying to get some public awareness about our efforts to get Mom access to this drug, I feel like we were out of options, and the only way to get results is to get this out there," Jackie Loughman, the youngest of the three women, said yesterday in a telephone interview.
According to the Mayo Clinic, pancreatic cancer is one of the most serious cancers. It spreads quickly and is rarely detected early, making it a leading cause of cancer death. The National Cancer Institute estimates that 34,000 people will die of the disease this year, and about 38,000 will be diagnosed with it.
Connie Loughman was diagnosed in August after she went to see her doctor, complaining of abdominal pain. She participated in one unrelated clinical trial but had to stop treatment after it put her in the hospital. She also has undergone chemotherapy to treat a tumor on her pancreas.
Because of those treatments, she is not eligible for GenVec's trial, Jackie Loughman said.
Her dad, John, took on the caregiver role for his wife of 44 years and the three daughters rotate in as needed. Connie Loughman had to leave her investment-adviser position, a job she loved. She is now on disability and struggles daily with the disease.
The daughters decided to launch the campaign after reading about TNFerade in a January news article. They linked their mother's situation with that of actor Patrick Swayze, who confirmed he had pancreatic cancer this month. Their hope is Swayze's celebrity would draw more attention, though they have not heard from his camp yet, Jackie Loughman said. The Web site is www.cureconnieandpatrick.com.
The YouTube video (www.youtube.com/watch? v=09gFnSYUjQQ) shows images of Connie Loughman and Swayze. The family's Web site calls TNFerade a "revolutionary" and "life-saving" treatment. A news release issued by the family calls the drug a "cure."
That is troubling to GenVec, which is subject to strict rules when it comes to evaluating its drugs.
"We've never used the word 'cure.' That is a word for this diagnosis that is rarely used," Swirsky said. While TNFerade has shown some results, its real feat is extending median survival about four months, he said.
"We're talking about months, and not years," Swirsky said. "We know how tough this disease is."
The 15-year-old company has about 120 employees and no products on the market. Its main focus is moving TN Ferade forward. But the earliest the drug could be approved and brought to market is 2010, which is not good enough for Loughman's daughters.
"She's one of those people who's always there for you," Jackie Loughman said of her mother. "It's my turn now. "
tricia.bishop@baltsun.com