ALS patient denied access to drug/GREAT ARTICLE !!!!!!

ALS patient denied access to drug
Pearl man unable to get medication he thinks could combat disease
Shanderia K. Posey • sposey@jackson.gannett.com • September 9, 2008


What could be more disheartening than living with a terminal illness that daily diminishes your quality of life?
Answer: Being denied access to the one drug you think could improve your condition.
And when you've been diagnosed with amyotrophic lateral sclerosis, also known as Lou Gehrig's disease, time is not on your side. Most ALS patients die within years of the diagnosis. Something Vicki and Ricky Muirhead of Pearl are well aware of.
"ALS takes everything away from you, the only thing you're left with is your mind," Ricky said. "You can't walk, talk or breathe on your own. You're dependent on your caregiver."
For four years, Ricky, 48, lived with involuntary muscle twitches local neurologists diagnosed as benign fasciculation. Then in August 2007, Dr. Y. Harati at Baylor College of Medicine in Houston, Texas, diagnosed him with ALS. Vicki said Harati noticed the muscle between Ricky's thumb and index finger was sunken in - a sign to Harati that ALS was present.
Mayo Clinic defines ALS as a neurological disease that attacks nerve cells that control voluntary muscles. It may begin with muscle twitching or weakness in an arm or leg or with slurring of speech. Eventually, it affects the ability to control muscles needed to move, speak, eat and breathe.
Eighty percent of ALS patients die within three to five years of diagnosis, according to Leigh Langford, an instructor of neurology at the University of Mississippi Medical Center who has worked with ALS patients for 10 years. She said 20 percent die within six to 10 years and some patients' conditions may plateau.
The only drug available for treatment of ALS is Rilutek. Langford estimates the drug costs between $800 and $1,000 a month. It's main purpose is to prolong the life of ALS patients about three to six months.
But a few weeks ago, Ricky joined an ALS online forum and became aware of a drug called Iplex made by Insmed Inc. of Richmond, Va. The Food and Drug Administration approved Iplex Dec. 12, 2005 to treat growth failure in children. It is a man-made form of insulinlike growth factor-1 (IGF-1) normally produced in the body.
Ricky learned ALS patients in Italy were using the drug and reporting amazing results, so he wanted to try it, too.
"Testimonies online of those in Italy showed people who couldn't walk are now walking with a cane. People that couldn't talk are now talking or (people who) couldn't be on the phone are now picking up the phone," Ricky said.
However, as a result of a settlement agreement between Insmed and two biopharmaceutical companies called Tercica and Genentech, American ALS patients can not have access to the drug. Tercica and Genentech hold patent rights for the manufacture and use of IGF-1 products in the treatment of ALS. They sued Insmed for patent infringement, and the case was settled in March 2007. Before the settlement, an expanded access program was created for ALS patients in Italy to have access to Iplex. Tercica and Genentech agreed to allow the program to continue. Tercica and Genentech offer an alternative to Iplex called Increlex.
"As part of the agreement, Insmed is allowed a nonexclusive license with Tercica/Genentech to develop Iplex for the following named indications: MMD (myotonic muscular dystrophy), HARS (HIV-associated adipose redistribution syndrome), ROP (retinopathy of prematurity), recovery from burns and trauma, and recovery from hip fracture," said John Procter, a spokesman for Insmed Inc., in an e-mail to The Clarion-Ledger.
Vicki and Ricky Muirhead are now on a mission to raise awareness of the condition and get access of Iplex to all ALS patients.
"We are trying to get enough people to sign the petition (online) for ALS patients and burn patients to rebuild muscles," Ricky said. They also want senators and congressmen to propose legislative measures for their cause.
Some ALS experts point out a lack of documented evidence of Iplex's effectiveness.
"I don't know how much scientific proof there is," Langford said. She is focused on a year-long study that began in June using lithium to treat patients. Ten centers across the nation are involved.
"It's the biggest promise we've seen," Langford said, noting it's the first time in 25 years another drug could be approved for treatment.
Sharon Matland, vice president of patient services for the ALS Association national office in Calabasas Hills, Calif., isn't ready to give her stamp of approval to Iplex either.
"Right now it has not (been) tested in this country," Matland said, explaining her organization promotes and supports the drug safety processes in the United States, including research on lithium. "We are monitoring how that work is going on over there (in Italy)."
The Muirheads are doing the same and plan to continue to spread the word about their discovery. They attend a monthly ALS support group that meets at the Methodist Rehabilitation Center in Jackson.
Vicki, who is an insulin-dependent diabetic, quit her job as a travel agent to become the primary caregiver of her husband. He's been fortunate to continue working as an electrical engineer in Chicago and flies home weekly, but ALS eventually may make those trips unbearable.
Ricky's speech is now slow and slurred. Two fingers on his right hand feel like they are asleep 24/7, he said. However, he maintains a positive, even comical, attitude. He jokes about hoping others can understand his "ALS accent."
Langford said the disease is more common in men and strikes at the average age of 55. It is mostly considered sporadic and not genetic. Data indicate about 5,600 people are diagnosed with ALS yearly. There is no known cause though some environmental factors have been suggested. For example, the Muirheads wonder if chemicals Ricky used to spray and treat their lawn could have attributed to the onset of ALS.
UMC's ALS clinic takes a multidisciplinary approach to treatment including physical therapy and pulmonary monitoring, Langford said. Diagnosis is one of exclusion, meaning other diseases or conditions must be ruled out. Langford suggests those experiencing symptoms such as weakness of any extremity; tightness in the leg or arm; muscle twitching of the leg, arm or tongue; slurred speech; or difficulty swallowing see their primary physician and get a referral to the Muscular Dystrophy Clinic at the Jackson Medical Mall, which works in conjunction with UMC.
For now, the Muirheads treasure what time they have left. They will celebrate their first wedding anniversary Sept. 16.
"All we are asking for is a chance," Ricky said.
"A chance to live a normal life as long as you can," Vicki said. "In this situation, it's all about quality of life. And if this drug can give you more quality in the amount of time you have, why not?"
To comment on this story, call Shanderia K. Posey at (601) 961-7264.