RILUTEK USERS GET COST BREAK
Since Jan. 10, when Rilutek became available by prescription, phones across the country have been buzzing with questions and answers about its cost and availability.
Rilutek, a drug that affects the nervous system chemical glutamate, was shown in human trials to prolong life expectancy for ALS patients an average of three months.
Pharmacies are charging anywhere from $620 to $900 for a month's supply. Realizing that cost would be prohibitive for many people with ALS, Rhone-Poulenc Rorer (RPR), the company that developed and markets Rilutek, put a financial assistance program in place as soon as the drug was available. Its toll-free Rilutek financial assistance hotline received about 2,600 calls in its first six weeks of operation.
Quantum Health Resources is contracted by RPR to handle those calls at its Fort Worth headquarters. In its Rilutek Continuity Program, Quantum pharmacists intervene with individual callers' insurance companies to help callers get the maximum insurance coverage of the drug's cost.
Quantum pharmacist Mark McSweeny, one of 40 people on the program staff, phones the insurance company and verifies the individual's benefits. He urges insurers to cover Rilutek.
"It normally takes six to 12 months to get a new drug on a company's formulary," McSweeny said. By informing the company about Rilutek and the urgency with which patients need it, "We help facilitate adding Rilutek to the list.
" Acting as a patient advocate, Quantum discusses all options for insurance coverage, including out-of-plan benefits. McSweeny estimated that by March 1, about 80 percent of insurance companies provided some coverage of the Rilutek purchase price.
When Quantum is unable to get insurance coverage for Rilutek, it refers callers to the National Organization for Rare Disorders, a nonprofit organization based in New Fairfield, Conn.
NORD administers RPR's Patient Assistance Program. Anyone interested in qualifying to receive Rilutek for free must complete an application that requests detailed financial information and supporting documents. In January, NORD received 50 Rilutek applications and approved 35 of them, said Lynn Klein, NORD executive vice president.
RPR's wholesale cost for Rilutek is $590 a month, for two 50- milligram pills a day. Pharmacies mark up this price to an average of $707, according to an informal MDA survey. Quantum can help callers find lower-cost pharmacies in their communities or supply the drug through the mail for about $720.
To reach Quantum, call (800) 790-RTEC; NORD's number is (203) 746-6518.
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 GABAPENTIN (NEURONTIN) SHOWS PROMISE IN ALS
The drug gabapentin (brand name Neurontin) has shown encouraging results in a 152-patient trial to test its effects on loss of strength in ALS. Seven medical centers, six of them MDA clinics, took part in the trial. Dr. Robert Miller, director of the Forbes Norris MDA/ALS Center at California Pacific Medical Center in San Francisco and co-director of the MDA clinic there, was the lead investigator.
The drug was tested against a placebo, or sham drug, and neither patients nor investigators knew who was getting the drug and who was getting the placebo. The rate of arm muscle weakening in patients treated with gabapentin was 24 percent to 37 percent slower than in patients taking the placebo.
Q. What is gabapentin (Neurontin)?
A. Gabapentin is a drug manufactured by Parke-Davis that's on the market for epilepsy (seizures). Experimental evidence shows that it may decrease the production of glutamate, a natural central nervous system chemical. Some people with ALS have higher than normal levels of glutamate, and glutamate has been implicated as a possible cause of motor neuron death in ALS even at normal levels (possibly because of other damage to motor neurons in this disease).
Q. Is gabapentin like any other drugs that are being tested or marketed for ALS?
A. Gabapentin is similar to riluzole (Rilutek), approved by the Food and Drug Administration in December for the treatment of ALS, in that it's also a partial blocker of glutamate. The two drugs probably work in different ways.
Q. What measures did the study use to determine the drug's effectiveness?
A. As noted earlier, the study measured arm strength.
Q. How significant is the study?
A. The study, which started with 152 patients and ended with 117 (35 people didn't complete it), showed that patients treated with gabapentin declined more slowly than those treated with a placebo. However, the results didn't quite reach the standard set for statistical significance — a 5 percent or less probability that the results are due to chance and that there isn't any real difference between the drug and placebo groups. In this study, the chance that there was no drug effect is between 6 and 8 percent. This kind of result warrants further investigation to confirm or refute the findings.
Q. Did gabapentin extend survival?
A. The effect of gabapentin on survival in ALS wasn't measured.
Q. Did gabapentin help patients feel better?
A. Positive effects noted by some patients included a decrease in muscle spasms or twitches and improved sleep quality.
Q. Were there adverse effects with gabapentin?
A. Gabapentin was generally well tolerated. The most common adverse effects were dizziness, light-headedness, drowsiness, fatigue and swelling of the feet. These were considered temporary and mild.
Q. How can I get gabapentin?
A. Since gabapentin is already on the market, doctors can prescribe it. However, it isn't yet approved by the FDA for ALS, which puts physicians at risk if they prescribe it for a non-indicated use. (For example, if an ALS patient took legal action against a doctor because of a perceived gabapentin-related problem, the doctor would have a hard time defending such unorthodox prescription of the drug.)
For this reason, many doctors may hesitate to prescribe gabapentin before it receives FDA approval for ALS.
If you're interested in taking gabapentin by prescription or participating in future testing of the drug, see your MDA clinic director.
Q. How is gabapentin taken?
A. Gabapentin comes in tablets to be taken by mouth. It can be crushed to be taken through a feeding tube.
Q. What is the recommended dose of gabapentin for ALS?
A. The recommended dose is 2,400 milligrams a day, divided into three doses of 800 milligrams each.
Q. How much does gabapentin cost?
A. Prices vary, but the average is $150 to $250 per month for a dose of 2,400 milligrams per day.
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EXPERTS MEET ON COMBINATION TRIALS
MDA and its Canadian counterpart have impaneled an impartial body of experts to persuade drug companies and academic researchers to join forces to test in combination the individual drugs that have shown marginal results in ALS treatment trials.
It's hoped that, just as combinations of drugs relatively ineffective on their own have become weapons against cancer and AIDS, this approach may prove successful in ALS as well.
The big challenge to the Working Group will be to overcome the proprietary interest of drug makers for the sake of patients. It will also serve as an independent authority for assessment of clinical trial data, and for coordination of trials to avoid duplication and prevent exclusion of promising treatments.
The group will meet May 3 in Chicago to establish methodology for combination trials.
Director of Science Technology Donald Wood and Medical Advisory Committee Chairman Dr. Leon Charash will represent MDA.
Other experts playing roles are Dr. Jeffrey Rothstein of Johns Hopkins University, Dr. Hiroshi Mitsumoto of the Cleveland Clinic Foundation, Dr. Steven Ringel of the University of Colorado Health Sciences Center, Dr. Robert Brown of Massachusetts General Hospital and Dr. Robert Griggs of the University of Rochester School of Medicine.
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IN ALS COUNTRY
by Anthony J. Vitale
Early in 1993, a close friend of mine was dying from pancreatic cancer at age 51. His wife said to me that being ill is like traveling to a foreign country.
At the time of my diagnosis with ALS, I recalled her statement and it gave me hope since I had lived in many foreign countries. I arrived in East Africa in late 1966 at the tender age of 21. It was my first time away from home. I was trained by the U.S. government in East African language and culture, but I was still in for a bit of a shock. I saw my first grass hut, my first elephant in the middle of the road. The nights were pitch black and as silent as death. Only the occasional glow of village fires illuminated the eternal darkness.
Serious illness is like that darkness. One is nearly helpless to influence or change it. The only defense is self-confidence, optimism and innovation. You have to ignore the danger from wild animals and focus on your goal: to arrive safely and in one piece.
An individual who is able-bodied and then suddenly is severely disabled is like a person being let off a plane in an airport in a foreign country and wished good luck. And there you are, tired, confused, searching for a kind face, and God willing, at least one friend at your side.
You examine your surroundings. You see a strange place with a strange language. And, occasionally, someone even asks you to be a translator. The clothes are all wrong, they're driving on the wrong side of the road, the houses have mud walls and thatched roofs, and the colors and the smells and the sounds are different and strange.
But you soon find yourself adapting to these new colors, styles, smells and sounds simply because they work better in this new environment. The food is different but you've adapted to different cuisine before and you'll have to do it again. The people look different as well.
In any case, you're here to stay. This is not a vacation. The quicker you adjust, the better it will be. You'll be more productive. You'll make new friends. You'll hear and see the real news that doesn't get on TV. It's a new adventure, the safari you never thought you'd get around to taking. The next mountain you wanted to climb.
How you wish you could describe what it's really like to friends back in your home country. A postcard just doesn't convey more than a shadow of an idea. The people back home would have to come to see you in your new place in order to understand.
And what's the matter with some of them? Do they think you've gone off to Mars? It's only another country. Everyone here is human. Don't they know how long you've been away? A month feels like a year; a year is a lifetime. Hurry, because I'm changing so fast, you won't recognize me. Hurry, because when you casually ask "How are you?" and I answer "Not bad," you'll never, never know what "not bad" really means.
Here you are in this new land. You become aware that the sun still shines and birds still sing. Life is good and you're still alive.
And you notice that the people here are smiling more sincerely. They look at you as if you are someone special. They care. The only values here are family and friends. All the rest is unimportant.
From past experience, I knew in my heart that I had enough skill and knowledge to survive some of the most inhospitable regions of the world. Knowing that has made me confident that I could survive this "new country."
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Vitale, a linguist and engineer with Digital Equipment Corporation, has worked for over 20 years developing voice synthesis technology to help people with disabilities communicate using computers. After his ALS was diagnosed in 1993, Vitale continued to press forward with his work, and acknowledges that now he is one of those who may benefit from this technology.
Vitale has participated in MDA fund-raising, made speeches on behalf of MDA, and appeared on the local broadcast of MDA's Telethon, copyright 1995 Anthony J. Vitale.
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UPDATE ON GROWTH FACTORS
Neurotrophic, or nerve growth, factors are being tested as possible therapies for ALS.
CNTF -- A clinical trial of CNTF (ciliary neurotrophic factor) by the drug company Regeneron ended in 1994, and Syntex/Synergen ended a similar CNTF trial in 1995. Toxicity and lack of effectiveness were found in these trials, in which the drug was given by subcutaneous (just under the skin) injection.
But CytoTherapeutics of Providence, R.I., has a new idea — testing CNTF given directly into the fluid surrounding the spinal cord, known as intrathecal delivery. The company has put the gene for CNTF into hamster kidney cells, encapsulated these cells in a membrane that protects them from the patient's immune system and allows retrieval of the capsule, and implanted the cell- containing capsule at the base of the spinal cord.
Ten patients in Switzerland have received the implants in a pilot trial. So far, none have had serious side effects.
As part of the study, three patients had their CNTF implants removed 13 to 17 weeks after they were inserted. In these three, the cells were still alive and still making CNTF. All three have elected to receive a second implant to be left in place for up to six months.
The company says implants may maximize effectiveness, since the CNTF will get directly to the cells where it's needed, and minimize toxicity, since about 500 times less CNTF will be used than in previous trials.
IGF-1 -- As we reported in January (Vol. 1, No. 2), the Food and Drug Administration didn't grant treatment IND (investigational new drug) status to Cephalon and its partner, Chiron, for Myotrophin, which is IGF-1 (insulin-like growth factor 1). Treatment IND status would have allowed Cephalon and Chiron to start an early access program for IGF-1.
Cephalon says it has met with the FDA about its concerns and expects to hear word on its application in the next few weeks.
BDNF -- Amgen and its partner, Regeneron, are conducting phase three (large-scale) trials of BDNF (brain-derived neurotrophic factor) with 1,135 patients at 38 sites in the United States and Canada. Twenty-nine of these are MDA clinics. Patients are giving themselves subcutaneous injections of the drug or a placebo.
Amgen hopes to start a small trial of intrathecally delivered BDNF within the year.
GDNF -- Amgen is doing laboratory toxicity testing of GDNF (glial-derived neurotrophic factor) and will start clinical trials this year or in 1997, if the studies suggest the drug is safe.
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BRITISH LEADER VISITS MDA
George Levvy, chief executive of the Motor Neurone Disease Association (MNDA) headquartered in Northhampton, England, conferred with senior MDA staff in Tucson, Ariz., on March 29.
"Motor neurone disease" is another way of referring to amyotrophic lateral sclerosis (ALS). The MNDA, which was founded in the late 70s and now has 100 volunteer-run branches, offers help to people with ALS in England, Wales, Northern Ireland, the Isle of Man and the Channel Islands, and funds research into the disease.
The MNDA estimates that 1,200 people are found to have ALS every year in Great Britain. About a quarter of the MNDA's income is from gifts that people have left through wills.
"Gifts in the form of legacies left to our Association," Levvy said, "have had a significant impact on our ability to provide care to people with motor neurone disease."
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UCLA NEWEST MDA/ALS CENTER
MDA has established an MDA/ALS Center at the University of California-Los Angeles. The designation brings to eight the number of MDA-funded centers dedicated to ALS research and medical care at major U.S. medical facilities.
The center is directed by Dr. Michael C. Graves, co-director of MDA's UCLA clinic offering medical services to anyone affected by any of the 40 neuromuscular diseases in MDA's program. Some 300 ALS patients in the Greater Los Angeles area are treated at the MDA clinic.
Graves is associate professor of neurology and director of UCLA's ALS Clinic and its Electromyography Laboratory.
The MDA/ALS center conducts MDA-funded research into the causes of ALS and participates in trials of experimental ALS drugs, including BDNF, a neurotrophic growth factor. Much of the research draws on donations made to UCLA's Brain and Spinal Cord Tissue Bank.
The center can be reached at: MDA/ALS Center (310) 825-7266 University of California-Los Angeles Department of Neurology 710 Westwood Plaza Reed Neurological Research Center P.O. Box 951769 Los Angeles, CA 90095-1769
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THE ALS NEWSLETTER
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