Entries in Clinical Trials (9)


MS Patients Might Benefit From New Oral Drug

Jupiterimages/Thinkstock(NEW YORK) -- Thousands of Americans who suffer from multiple sclerosis (MS) might one day be able to take advantage of a drug that new research suggests is both safe and effective.

Multiple sclerosis is a chronic and often disabling disease that affects nearly 400,000 people in the United States. It attacks the protective substance called myelin that covers the nerve fibers of the brain and spinal cord.

Myelin is similar to the insulation of a wire and ensures proper nerve function. Once the myelin is damaged, the disease can also damage the nerve fibers themselves, leading to scars in these delicate tissues.

This damage leads to symptoms as mild as tingling in your feet and fingers, or as severe as paralysis or blindness. Eighty-five percent of MS patients are diagnosed with what is called relapsing-remitting MS, which means they experience flare-ups of symptoms, followed by partial or complete recovery.

A team of researchers looked at nearly 1,500 patients in 28 countries taking the experimental oral drug, known for now by the name BG-12, to see whether such flare-ups decreased, as well as whether they experienced any side effects from the treatment. The patients were studied for two years.

The results were encouraging. Overall, the patients receiving the experimental drug did better than the placebo group. Those who took the drug twice daily cut their chances of experiencing a flare-up by 44 percent, while those taking it three times a day reduced their chances by 51 percent. By comparison, the researchers wrote, a drug currently used for MS only cuts flare-up rates by 29 percent. Additionally, an MRI scan revealed that patients taking BG-12 had fewer scars on their nerves and brain.

If it is more widely used, the drug might offer hope to a large number of patients. If approved by the U.S. Food and Drug Administration, BG-12 would join two other oral medications on the market for MS.

"There is a great need for effective oral agents with acceptable safety profiles for MS patients with mild disease," said Dr. Lawrence Samkoff, associate professor of neurology at the University of Rochester in Rochester, N.Y., who was not involved with the study. "These newer oral medications will inaugurate a new era in the treatment of relapsing MS, giving patients and their physicians more choices."

Samkoff added that while BG-12 has some mild side effects, including flushing and an upset stomach, such symptoms tended to decrease within one month of taking BG-12.

Still, more research might be needed before BG-12 becomes more widely available, even after this two-year study.

"[Two years] is the standard these days, but the question is: is it adequate?" said Dr. John Corboy, professor and director of the department of neurology at the University of Colorado. "[It] is quite long in general, but very short when you consider the typical duration someone has MS."

Dr. Timothy Coetzee, chief research officer of the National MS Society, agreed. "As with any new therapy, it's important to do follow-up studies to understand the impact of the treatment over the long term," he said. "This will help us gain more specific knowledge of the long-term impact of this treatment on the immune system as well as the nervous system."

Copyright 2012 ABC News Radio


Drinking Pig Worms to Fight Crohn's Disease

iStockphoto/Thinkstock(NEW YORK) -- Eight years ago, New Yorker Herbert Smith did the unthinkable -- he swallowed thousands of pig whipworm eggs in a desperate bid to quell his advancing Crohn's disease.

The microscopic eggs, invisible to the naked eye, were suspended in a liquid solution.

"There was nothing to it," said the 33-year-old financial analyst, who uses a pseudonym to talk about his worm-drinking ways. "It was drinking half a cup of salty water."

At that moment, he said, "I felt excitement and definitely hope."

For Smith, something incredible happened. After swallowing 2,500 worm eggs every two weeks for three months, most of his Crohn's symptoms vanished.

"I was definitely ecstatic," he said. "The symptom reduction was pretty drastic."

"I did have blood tests before and after," he said, and "the markers of inflammation went down significantly."

As for his physician's reaction, Smith said "he was cautiously optimistic."

Smith has been battling Crohn's disease since he was diagnosed as a teenager.

"The worst day of your life is to find out there's no known cure," he said. "It affects your quality of life in a significant way, and most treatments are subpar."

As many as 1.4 million Americans live with Crohn's disease or its cousin, ulcerative colitis, according to the U.S. Centers for Disease Control and Prevention. In Crohn's disease, the body's immune system mistakenly attacks the lining of the intestine, causing diarrhea, abdominal pain, fatigue, bleeding and infections. For Smith, the disease has been difficult to control.

Despite undergoing multiple operations to remove part of his gut, his symptoms always returned, he said. He calculated that at his current rate of required surgeries, he would eventually run out of small intestine. If this occurred, he would require liquid feedings through an intravenous line, with potentially fatal consequences.

"That realization was pretty hard to take in," said Smith. "I had to do my own research."

Smith started studying the medical literature on how parasites might be useful in the treatment of inflammatory bowel disorders such as Crohn's disease. He also learned that one could order a three-month supply of pig whipworm eggs from Europe for 3,500 euros.

But he was realistic about his treatment goals.

"I knew that the most I could hope for was a remission for Crohn's, not a cure," he said.

Despite his promising response to pig whipworms in 2004, Smith had to stop because "it was very expensive and hard to get," he said.

Today, researchers are eagerly studying the experimental therapy. After finding that pig whipworm treatment was effective and safe in a small number of Crohn's patients, scientists are now conducting multicenter studies across the United States and Europe. The goal is to determine if this treatment will relieve symptoms and be tolerated across a larger group of patients.

Dr. Joel V. Weinstock, a parasitologist and chief of the Division of Gastroenterology/Hepatology at Tufts Medical Center in Boston, said there was a scientific basis behind drinking pig whipworms to help reduce symptoms in autoimmune disorders, such as Crohn's.

"Parasites are known to dampen the immune systems of their hosts," Weinstock explained. More specifically, drawing from animal studies from his laboratory, pig whipworms appear to activate cells that regulate the immune system so that it doesn't overreact.

One advantage of pig whipworms is that they don't cause disease in humans. Weinstock emphasizes that this parasite doesn't migrate outside your gut, can't reproduce in humans and dies off after two months. People who have pig whipworms can't spread them to others, and there are several medications that can be used to rid the body of whipworms, Weinstock said.

According to Weinstock, there have been no major side effects or complications of pig whipworm treatment reported in studies on Crohn's patients. One study on whipworm treatment in those with hayfever documented mild side effects (gas, diarrhea and cramping) that were usually resolved after two weeks. According to Dr. P'ng Loke, an assistant professor in the department of microbiology at New York University, citing the studies available so far, "pig whipworms look to be extremely safe for the time being."

Stories of other patients with autoimmune diseases, like Smith, who are deliberately infecting themselves with worms have surfaced. Dr. Weinstock cautions that patients shouldn't treat themselves with worms. He encourages Crohn's patients to remain on standard, proven treatments and to talk to their physicians.

Smith noted that he kept his physician informed about his parasite experiments. One reason, he said, is that "I didn't want him to do a colonoscopy and be horrified."

For patients who want to explore whipworm therapy, Weinstock encourages patients to talk to their doctors about enrolling in clinical studies. Other pig whipworm clinical trials are planned or under way for more autoimmune diseases, including ulcerative colitis (an inflammatory bowel disease), multiple sclerosis (an autoimmune disorder that affects the brain and spinal cord), psoriasis (an autoimmune skin disorder) and type 1 diabetes.

Smith has declined to participate.

Heather Gelabert, a 32 year-old Florida homemaker, was forced to drop out of college because her Crohn's disease proved so difficult to treat. She was initially taken aback when her physician described pig whipworm treatment, but after a long discussion, she decided to enroll in a clinical trial.

For Gelabert, the promise of this upcoming clinical trial for Crohn's patients brings new optimism. "I am excited and very hopeful," she said with enthusiasm. "I'd like to start now!"

Copyright 2012 ABC News Radio


How Do Docs Prescribe Kids’ Meds? Guess 

Comstock/Jupiterimages/Thinkstock(BOSTON) -- The last time your doctor gave you a prescription, it likely came with specific information on the correct and safe dosage to take, determined from years of clinical trials. But when kids need certain prescription drugs, such as statins, morphine, anesthetic or the asthma drug prednisone, doctors sometimes have to guess how much to give them based on the child’s weight.

The reason, described in a report published Tuesday in the Journal of the American Medical Association, is that about half the prescription drugs commonly given to children have no information on appropriate pediatric doses on their labels.

Researchers from the U.S. Food and Drug Administration studied drugs listed in the Physicians’ Desk Reference, the bible of FDA-approved drug labeling, and counted the number of drugs that came with guidance for use in children, including the appropriate dosage.  Of 461 drugs in the PDR that were for children, 231 had the adequate information doctors would need to prescribe the drug to a child.

For the 105 brand-new drugs approved by FDA between 2002 and 2008 that could potentially be used in children, 43 had pediatric information on the labels.

“You can’t get a product approved in adults without studying how it affects them first,” said Dr. Dianne Murphy, one of the study’s authors. “But children are routinely being given products that are not studied in them.”

The study is part of an effort to increase awareness that prescription drugs are often not tailored to kids’ unique biology. In April, Harvard researchers reported that four out of every five children hospitalized in the U.S. are treated with drugs that have never been tested in children and are FDA-approved only for adults.

For newborns, the picture is even grimmer.  About 90 percent of drugs have never been tested for use in infants.

Dr. Florence Bourgeois, the author of that study and an assistant professor of pediatrics at Harvard, said that about 60 percent of common conditions such as asthma and lower respiratory infections occur in children, but only 12 percent of the clinical trials currently testing drugs look at how they work in children specifically.

So when kids need these drugs, their doctors usually just have to guess how much to give them, usually gauging it against their weight. But kids are not just little adults. The way the liver, kidneys and other organs work to metabolize drugs is far different in early life.

“In some instances, extrapolating adult drug dosage to children might be appropriate, but again, without specific trials to assess that, we simply don’t know,” Bourgeois said.

Experts said testing drugs in children is simply more complicated and costly for drug companies. Clinical trials of children have their own ethical issues and often the science used to study adults doesn’t apply to children. For example, it’s difficult or impossible to get infants and children to breathe into a spirometer to study their asthma.

But alarming as it may seem to parents with sick kids, the situation used to be a lot worse. Dr. Daniel Frattarelli, chairman of the American Academy of Pediatrics’ Committee on Drugs, said even though half of drugs have dosage information for children, it used to be that only about 20 percent did.

The improvements are largely the result of two laws, passed in the late 1990s and early 2000s. The Pediatric Research Equity Act allows the FDA to require drug companies to do additional testing of their products on children if the drug is likely to be used widely in pediatrics. The Best Pharmaceuticals for Children Act gives incentives for drug companies to test their products on children, such as giving them an additional six months of marketing exclusivity.

Both laws are up for congressional reauthorization in 2012, and experts say they are essential to getting more information about appropriate dosage of drugs in children.

Copyright 2012 ABC News Radio


Bias Can Exaggerate Drugs’ Effectiveness

Stockbyte/Thinkstock(PORTLAND, Ore.) -- Doctors -- and patients -- might not be getting all the information they need about the safety and effectiveness of certain drugs because of “publication bias,” the tendency of researchers and medical journals to favor positive results over negative ones.

Researchers running drug trials are required to submit detailed results to the U.S. Food and Drug Administration. But when it comes to reporting trial results publicly in medical journals, “it’s an entirely different ballgame,” according to Dr. Erick Turner, lead author of a study published Tuesday in PLoS Medicine.

“Doctors are trained to regard medical journals as the gospel truth,” said Turner, assistant professor of psychiatry and pharmacology at Oregon Health and Science University in Portland. “But what we’re learning here is it’s not necessarily the whole truth and nothing but the truth.”

Turner and colleagues reviewed the results submitted to the FDA for eight antipsychotic drugs used to treat schizophrenia. They then compared the results to those published in medical journals. Four trials submitted to the FDA, all of which had unflattering results for the drug under study, were unpublished.

“It’s kind of like grade inflation,” said Turner. “Say you’ve got a class full of kids. Some are excellent students and some should be failing. If you give everyone an A, an outside person is not going to be able to appreciate there’s a difference between these two sets of kids.”

Even when the studies were published, the journal articles often overemphasized the drugs’ effectiveness.

“Some of what we found could constitute spin, some would fall into the category of shenanigans,” said Turner. “The take-home message is there are loopholes in the publication process by which doctors may be relying on information that’s incomplete or somehow skewed. The drug’s effects may be exaggerated or its safety concerns may be downplayed.”

Turner said researchers working for a drug company might be inclined to withhold data that’s seen as damaging, adding “there’s no law says they have to publish.” But certain medical journals are also less likely to accept negative trials for publication. Bias, Turner said, could be mitigated by leaving the results out of the decision to publish.

“The person reviewing the study for the journal should be thinking, ‘Is this good science?’ rather than basing a decision on the results,” he said.

Copyright 2012 ABC News Radio


Herpes Vaccine Disappoints in Large Clinical Trial

iStockphoto/Thinkstock(CHAPEL HILL, N.C.) -- The search for a vaccine against genital herpes has hit a snag, researchers say.

Despite earlier success, new results from a larger clinical trial, reported in the Jan. 5 issue of the New England Journal of Medicine, showed that the experimental herpes vaccine provided only moderate protection against herpes type 1, which causes cold sores and occasional genital herpes cases.  The vaccine was not effective at all for the most common cause for genital herpes (type 2).

"We're really disappointed by the results," study co-author Dr. Peter Leone of the University of North Carolina-Chapel Hill said, according to MedPage Today.  

Leone said, MedPage reports, that since the virus types 1 and 2 are too difficult to tell apart, the vaccine's value is rendered inadequate.

Going forward, Dr. Leone said "a different type of vaccine approach" will be necessary to fight both viruses.

Copyright 2012 ABC News Radio


Growing Number of Mothers Making Money on Clinical Trials

Siri Stafford/Photodisc/Thinkstock(SAN ANTONIO) -- Yvette Santana, a 37-year-old mother of four who was diagnosed with diabetes, is one of a growing number of mothers who participate in clinical trials to make extra money.

Before she started participating in studies five years ago, despite working two jobs, Santana could not always afford to buy an $80-box of glucose test strips to monitor her diabetes. She would sacrifice her health to pay bills and buy groceries for her family.

"It gets very hard. You have to see what's more important, your kids and their needs or your own. And as a mother, it's always your kids," said Santana.

But participating in clinical trials provide her with insulin, health check-ups, and free strips. Santana says now she's stable and healthier than she's ever been.

Jennifer Martinez, a 33-year-old mother of four, is not struggling as much to make ends meet. She lives in a two-story brick home in an upscale community. Both she and her husband participate in studies to make extra money, which they use to go to Hawaii once a year.

Martinez started participating in studies when she was 23 so she wouldn't have to put her children in day care.

"It was really just to be able to stay at home with my kids," Martinez said. "I did one study and thought, 'Okay, that's like a lot of money in a short amount of time.' So I started to kind of pick it up and do a little bit more."

Martinez surfs the Web several times a day to find study announcements. She looks for "not crazy studies" -- taking medications that don't affect her heart or her brain and have only minor "over-the-counter symptoms" like nausea. She says she's never had a side effect.

Martinez makes an average of $7,000 a year participating in studies, but has made up to $13,000 in one year. She does about three studies a year, but if she has a big bill to pay or Christmas is coming up, she'll do an extra one.

Her advice to other moms? It's tough to get accepted for studies. You have to be diligent and respond to announcements quickly.

One company Martinez works for is Clinical Trials Texas. Kay Scroggins, the president and CEO of CTT, founded the company out of her home in 2001 with one study.

The company pays all volunteers a $40-to-$100 stipend per visit for their travel and time. The stipend depends on what kind of procedure the study entails.

Scroggins says these studies have "changed a lot" over the last 10-15 years.

"The qualification criteria to be in the study are much more complex and so in the industry we talk about the squeezing the funnel so to speak because you start with a large population and as you go through the criteria one after the other you start eliminating people. And so you come down with a very small group of people that would actually qualify for this study."

About 10 percent of the patients involved in the studies are healthy, according to Scroggins, and all others have pre-existing conditions.

When doing preliminary testing on volunteers, the company has found several cases of breast cancer, hepatitis and HIV.

"They would not have found that if they would not have been in a study," said Scroggins. "A lot of our patients will come in and be very depressed and be put on a medication that they may not have been able to have access to otherwise and seen a big improvement where they're back working. They can interact with their families again."

Dr. Arthur Caplan, the director of the Penn Center for Bioethics at the University of Pennsylvania, says that clinical trials are usually safe, but there are some risks you should be aware of.

"There are risks that something could go wrong, but it depends on what you're doing and what degree of research is involved. The more they're paying you, the more your radar should go up. They're paying you more because there's more risk or more pain involved."

Caplan also warns that if something goes wrong, your insurance company often will not cover it.

"You have to read the fine print. The company may say that those costs will be covered by your insurance company. I'm here to tell you they won't. Your insurance company will not pay for any injuries you get serving as a subject in a trial you signed up for."

Caplan says he can see why participating in studies is attractive, especially to people who don't have medical insurance, but he says you can't kid yourself. "Don't think that being seen by clinical trial technicians is a substitute for health care. At the end of the day, these companies are trying to deliver data to the pharmaceutical companies. They are not your doctors."

Copyright 2011 ABC News Radio


Sisters Thrive on Experimental Cystic Fibrosis Drug

iStockphoto/Thinkstock(BOSTON) -- An experimental drug called ivacaftor has transformed life for two Massachusetts sisters born with cystic fibrosis, a genetic disease that frequently interrupted their schoolwork and extracurricular activities by turning simple colds and viruses into potentially life-threatening lung infections that slowly reduced their ability to breathe freely.

Laura Cheevers, now 13, and her sister Cate, 10, were born with a gene defect that clogged their lungs with thick, sticky mucus, leaving them vulnerable to bacterial growth, infection and inflammation. Cystic fibrosis affects mucus membranes elsewhere in their bodies, including their digestive systems, where it interferes with absorption of nutrients, forcing them to consume thousands of daily calories to keep from losing weight. Both were in and out of the hospital and tired easily from an incurable, progressive disorder, which their parents knew could cut short their lives.

But today both are thriving, thanks to an international team of scientists and the Cystic Fibrosis Foundation, which spent years jointly developing what is shaping up to be the first treatment successfully targeting the basic defect underlying their disease. Although the twice-daily pill only helps CF patients with one particular genetic mutation -- about five percent -- experts expect the research to help them target the other mutations responsible for the disease. Both girls participated in double-blind clinical trials comparing ivacaftor pills to dummy pills among young adults and children. Neither the patients, nor their doctors, knew for sure for 48 weeks if they were getting the medication -- or the placebo.

However, the family and Cate had their suspicions. They noticed that Cate began improving within a month of beginning the study of ivacaftor in children 6 to 11. She stopped coughing, began growing like a weed, and her first lung tests showed "almost a 30-percent bump, which blew all of us away," said her mother, Kim Cheevers. Laura, who was in a similar study of the drug in children and adults ages 12 and older, stayed "pretty much the same as before. She ended up worsening over the winter."

Then, on April 1, everyone in the two trials began getting the medication for sure, and the results have been nothing short of stunning for the sisters from North Andover, Mass.

Laura, who typically "strains to gain a pound a year," has gained about 8 pounds, said Kim Cheevers, a pediatric intensive care nurse at Massachusetts General Hospital in Boston. "She is not coughing at night anymore. When she does get sick -- and this week we all have this horrendous cold and cough -- her mucus isn't that thick, thick sticky mucus. Everything is watery; it's easier for her to clear." More remarkable still, "she hasn't been on a course of antibiotics since she's been on the drug. In the past, every other month she'd be on something."

Laura and Cate are now six months into extensions of the ivacaftor clinical studies, which will provide them with the medication for eight years, their mother said. "We're on it until it gets FDA approval."

Vertex Pharmaceuticals, of Cambridge, Mass., developed the drug in collaboration with a non-profit drug research affiliate of the Cystic Fibrosis Foundation. On Oct. 19, Vertex asked the FDA for a priority review of the drug, which can be granted to medications considered major treatment advances. Vertex also requested similar expedited consideration from the European Medicines Agency, the regulatory body for the European Union.

The trial in which Laura enrolled found that ivacaftor improved lung function, helped with normal growth and weight gain, and reduced infections with few side effects, according to a report in Wednesday's issue of the New England Journal of Medicine. Improvements for those getting the drug began as early as two weeks and lasted for the duration of the study. Furthermore, the rate of serious side effects was worse among placebo recipients than those getting ivacaftor, suggesting it's safe as well.

Up until now, most drugs given for cystic fibrosis treated only its complications, such as chronic lung inflammation and frequent infections, and helped patients clear the clogged mucus better. These drugs helped push median survival in the last four decades from 11 years to what the Cystic Fibrosis Foundation says is now 37 years.

Results from a study of ivacaftor in children ages 6 to 11 (in which Cate participated), as well as use of the drug in combination with VX-809, another drug in the CF pipeline, are being presented this week at the 25th Annual North American Cystic Fibrosis Conference in Anaheim, Calif.

Copyright 2011 ABC News Radio


Duke Sued Over Cancer Trials

Jim R. Bounds/Bloomberg via Getty Images(DURHAM, N.C.) -- A lawsuit filed in North Carolina’s Durham County Superior Court accuses Duke University, Duke University Heath System and five doctors of exposing patients to unnecessary chemotherapy during fraudulent clinical trials.

The trials, which began in 2007 and 2008, were based on work by Dr. Anil Potti -- a former Duke cancer researcher who claimed to have developed a test that could predict which lung cancer patients would benefit from chemotherapy.

The trials were temporarily halted in 2009 when other researchers couldn’t replicate Potti’s results. But the trials resumed after an internal investigation. The lawsuit alleges the findings were withheld.

In 2010, it was discovered that Potti had falsely claimed he was a Rhodes Scholar on his resume and on a grant application. He resigned from Duke. And medical journals including Nature Medicine, the New England Journal of Medicine and the Lancet Oncology pulled his work. The clinical trials were also stopped.

The 90-page lawsuit alleges that Duke ignored warnings about flaws in Potti’s research and even tried to cover them up. The lawsuit  makes two dozen claims, which range from to negligence to fraud.

“Duke conducted clinical trials on cancer patients that should never have occurred. The trials were based on bad science,” plaintiff attorney Thomas Henson told ABC11. “Researchers across the country had been telling Duke and warning Duke about the bad science.”

Of eight patients named as plaintiffs, only two are alive today. One of the lawsuit claims, “loss of chance,” suggests patients who participated in Potti’s trials missed out on other treatment opportunities.

Duke spokeswoman Sarah Avery told ABC11 that  the university was actively investigating Potti’s research and possible misconduct.

In the meantime, ABC11 reports that Potti is still practicing medicine at the Coastal Cancer Center in Myrtle Beach, S.C.

Copyright 2011 ABC News Radio


The Impact of a Government Shutdown on NIH Clinical Trials

Comstock/Thinkstock(BETHESDA, Md.) -- Clinical trials involving new drugs to help cure diseases such as cancer, including cancer in children, will be stopped or slowed by any government shutdown, a spokesman for the National Institutes of Health said on Wednesday.

A shutdown would mean no new studies will be started at NIH, where everyone is a federal employee.

At the NIH Clinical Center there are currently seven new procedures, or protocols, scheduled to start next week that will not begin if the government shuts down over the weekend.

Ongoing studies at the NIH Clinical Center will not admit new patients, according to John Burklow, associate director for communications and public liaison at NIH, which “will delay the completion of all studies currently active at the Clinical Center.”

Burklow says there are approximately 640 clinical trials (and 1,443 variations, or protocols, within those clinical trials) at the Clinical Center that will stop admissions of new patients. Of the 640 clinical trials that will stop admitting new patients, 285 are for patients with cancer and 60 involve children with cancer.  

One new patient -- a child from a poor family with a rare disease -- was supposed to visit NIH on Monday to be added to a clinical trial, and had made special arrangements including traveling to NIH on a Miles for Kids program on Sunday. But none of this will happen if there’s a shutdown: no new patients, after all.

It’s unclear how a shutdown would impact other ongoing clinical trials outside the NIH campus, with NIH grants, since the doctors and other assistants are not federal employees.

But new trials will be stopped, including the phase III study of a promising new cancer drug -- Anti-CTLA4 -- at the Philadelphia’s Eastern Cooperative Oncology Group. That study will not be able to start on schedule if the government runs out of money, which will happen Friday night unless a deal is struck.

Copyright 2011 ABC News Radio 

ABC News Radio