FEBRUARY 3 AND 4, 2019

NEWS AND VIEWS

PRICE CONTROLS, YES. THAT IS ONE OF THE MAIN THINGS WRONG WITH OUR COST OF LIVING. THERE ARE NONE. WE CAN'T UPSET THE BIG BUSINESSES BY LIMITING THEIR PRIVILEGES. THERE ARE LIMITS TO WHAT THINGS ARE ACTUALLY WORTH, AND HOW MUCH IT COSTS TO MANUFACTURE THEM IS ONE OF THE FACTORS THAT DETERMINES THE VALUE. IT’S AN INTRINSIC VALUE. THE DESIRABILITY OF IT OR THE NEED FOR IT, THE MARKET VALUE, SHOULD NOT CAUSE THE PRICE TO RISE, ESPECIALLY IF HAVING IT IS CRUCIAL TO SOMEONE’S LIFE. IF THE PRODUCT RUNS OUT, APOLOGIZE TO THE PUBLIC FOR THAT AND THEN OFFER A SECOND PRODUCT IF THERE IS ONE. THIS IS THE KIND OF THING THAT MAKES ME FEEL THAT THE WORLD OF BUSINESS HAS SOME -- OR A GREAT -- TENDENCY TO BE CROOKED. HOW COULD PEOPLE REALIZE THIS AND YET GIVE THE MANUFACTURER THE HIGH PRAISE THAT SO MANY AMERICANS HAVE FOR THE WEALTHY? WE SHOULD PRAISE AND REWARD THE VIRTUOUS INSTEAD. AS FOR $375,000 A YEAR, SUCH PRICING USED TO BE CALLED "PRICE GOUGING," AND IT WAS ILLEGAL. IF EVERY TIME A COMPANY DID THAT, THEY WERE FINED HEAVILY THEY WOULD STOP THE PRACTICE. OF COURSE A POINTED LETTER FROM BERNIE SANDERS WILL HELP.

https://www.usatoday.com/story/news/health/2019/02/04/bernie-sanders-drug-prices-catalyst-firdapse-lems-375-000/2768449002/
Bernie Sanders asks why Catalyst Pharmaceutical is pricing a once-free drug at $375,000
Aki Soga, Burlington Free Press Published 12:41 p.m. ET Feb. 4, 2019 | Updated 4:15 p.m. ET Feb. 4, 2019

Sen. Bernie Sanders, I-Vt., is calling a company's decision to price at $375,000 a year a drug that was previously offered free to those with a rare autoimmune disorder “an immoral exploitation of patients who need this medication.”

In a letter addressed to Catalyst Pharmaceuticals CEO Patrick J. McEneny dated Feb. 4, 2019, Sanders called the change, “a blatant fleecing of American taxpayers” adding "I am profoundly concerned that Catalyst's actions will cause patients to suffer or die."

In a statement posted on his Senate website, Sanders wrote that Catalyst announced the pricing to investors in December.

Shortly after noon, the company said there was no one immediately available to respond to Sanders' letter.

More: Democrats examine drug prices, a first step in Congress' path to cut prescription costs

Sen. Bernie Sanders is asking Catalyst Pharmaceuticals why a drug to treat a rare disease that was once available free has been priced at $375,000.
(Photo: Jason Bean/RGJ)

The drug in question is called Firdapse, which was approved by the U.S. Food and Drug Administration in November last year for the treatment of Lambert-Eaton myasthenic syndrome, or LEMS, in adults.

The FDA describes LEMS as "a rare autoimmune disorder that affects the connection between nerves and muscles and causes weakness and other symptoms in affected patients."

Sanders writes that the active ingredient in Firdapse, 3,4-diaminopyridine, has been used to treat LEMS* for nearly 30 years and that "the clinical effectiveness had been established over decades in Europe and the United States."

Sanders said the cost of the drug ranges from $1,600 to $6,000 per year.

"Until now, patients have been able to access an unapproved version of this drug for free through a Food and Drug Administration compassionate use program," Sanders wrote to Catalyst, adding, "Simply put, this is corporate greed.

Sanders is asking Catalyst for information behind the cost and pricing of Firdapse, as well as other questions such as, "How many patients will suffer or die due to Catalyst's decision to set the annual list price of Fridapse [sic] at $375,000?"

The high cost of prescription medicine has been blamed as one of the drivers of rising U.S. health care costs. In January, the Trump administration announced a plan to eliminate some rebates paid by drugmakers in government programs such as Medicare to lower the prescription costs.

More: VT Insights: 4 challenges Bernie Sanders faces in a 2020 presidential bid

Sanders, who was a candidate for the Democratic presidential nomination in 2016, is contemplating another run for president in 2020.



THIS IS JUST ANOTHER STORY BY REUTERS ON THE SUBJECT OF FIRDAPSE. THIS ADDITIONAL PIECE OF INFORMATION IS WORTH HIGHLIGHTING. YA CAN’T SAY NOBODY LISTENS TO BERNIE. “CATALYST SHARES FELL NEARLY 8 PERCENT TO $2.31.”

https://www.reuters.com/article/us-usa-healthcare-catalyst/senator-sanders-to-ask-why-drug-once-free-now-costs-375k-idUSKCN1PT0ZJ
POLITICS FEBRUARY 4, 2019 / 6:06 AM / UPDATED 2 HOURS AGO
Senator Sanders asks why drug, once free, now costs $375k
Yasmeen Abutaleb
4 MIN READ

WASHINGTON (Reuters) - U.S. Senator Bernie Sanders sent a letter to Catalyst Pharmaceuticals (CPRX.O) on Monday asking it to justify its decision to charge $375,000 annually for a medication that for years has been available to patients for free.

PHOTOGRAPH -- U.S. Senator Bernie Sanders speaks during a news conference on Yemen resolution on Capitol Hill in Washington, U.S., January 30, 2019. REUTERS/Yuri Gripas.

The drug, Firdapse, is used to treat Lambert-Eaton Myasthenic Syndrome (LEMS), a rare neuromuscular disorder, according to the letter, made available to Reuters by the senator’s office. The disorder affects about one in 100,000 people in the United States.

The government is intensifying its scrutiny of the pharmaceutical industry and rising prescription drug prices, a top voter concern and a priority of President Donald Trump’s administration.

Both the Democratic-led U.S. House of Representatives and the Senate, controlled by Republicans, have begun holding hearings this year on the rising costs of medicines. Sanders is an independent who usually votes with Democrats.

In the letter dated Feb. 4, Sanders asked Catalyst to lay out the financial and non-financial factors that led the company to set the list price at $375,000, and say how many patients would suffer or die as a result of the price and how much it was paying to purchase or produce the drug.

Catalyst declined to comment on Sanders’ letter.

Catalyst shares fell nearly 8 percent to $2.31

For years, patients have been able to get the same drug for free from Jacobus Pharmaceuticals, a small New Jersey-based drug company, which offered it through a U.S. Food and Drug Administration (FDA) program called “compassionate use.”

The program allows patients with rare diseases and conditions access to experimental drugs outside of a clinical trial when there is no viable alternative.

Florida-based Catalyst received FDA approval of Firdapse in November, along with exclusive rights to market the medication for several years. The company, which bought rights to the drug from a company called BioMarin in 2012, develops and commercializes drugs for rare diseases.

BioMarin and FDA did not immediately respond to requests for comment.

In December, Catalyst announced it would price Firdapse at $375,000 a year.

“Catalyst’s decision to set the annual list price at $375,000 is not only a blatant fleecing of American taxpayers, but is also an immoral exploitation of patients who need this medication,” Sanders wrote in his letter.

Sanders joins other U.S. lawmakers in investigating the pricing practices of pharmaceutical companies this year.

Democratic Representative Elijah Cummings, chairman of the House Oversight Committee, in January wrote to 12 pharmaceutical firms asking for detailed information on how they set drug prices.

Democratic Representatives Frank Pallone and Diana DeGette wrote to the heads of Eli Lilly and Co (LLY.N), Novo Nordisk (NOVOb.CO) and Sanofi SA (SASY.PA), the long-time leading manufacturers of insulin, requesting information on why the drug’s price has skyrocketed in recent years.

Reporting by Yasmeen Abutaleb; Editing by Sonya Hepinstall and Susan Thomas

Our Standards:The Thomson Reuters Trust Principles.


BOILING THIS DOWN, LEMS IS A MUSCLE WEAKNESS CONDITION USUALLY ASSOCIATED WITH SMALL CELL LUNG CANCER, A LONG HISTORY OF SMOKING, AND HAVING A LATE LIFE ONSET. HOWEVER, THERE CAN BE OTHER CAUSES, IN WHICH CASES IT CAN START EARLIER.

SEE THE LIST OF RELATED CONDITIONS BELOW. ONE OF THESE WHICH I HAVE SEEN, COMMONLY CALLED BELLS PALSY IS “MYASTHENIA GRAVIS,” AND IT TENDS TO GO AWAY WITHOUT TREATMENT, BUT ANY MUSCLE WEAKNESS CAN LIMIT LIFE. IN THIS LEMS IT ALSO AFFECTS MUSCLES IN THE TRUNK OF THE BODY AND THIGHS, SO THAT GOING UP AND DOWN STAIRS BECOMES HARDER. THERE IS MORE ABOUT THESE CONDITIONS AND THEIR CAUSES IN THE “RAREDISEASES” WEB SITE BELOW, SHOULD YOU BE INTERESTED.

https://rarediseases.org/rare-diseases/lambert-eaton-myasthenic-syndrome/

Lambert-Eaton Myasthenic Syndrome

NORD gratefully acknowledges Paul Gozzard, MRCP, Nuffield Department of Clinical Neurosciences, John Radcliffe Hospital, Oxford, UK, for assistance in the preparation of this report.

Synonyms of Lambert-Eaton Myasthenic Syndrome
Eaton-Lambert syndrome
Lambert-Eaton syndrome
LEMS
myasthenic syndrome of Lambert-Eaton

General Discussion

Lambert-Eaton myasthenic syndrome (LEMS) is a rare autoimmune disorder characterized by the gradual onset of muscle weakness, especially of the pelvic and thigh muscles. Approximately 60 percent of LEMS cases are associated with a small cell lung cancer (SCLC), and the onset of LEMS symptoms often precedes the detection of the cancer. The LEMS patients with cancer tend to be older and nearly always have a long history of smoking. In cases in which there is no associated cancer, disease onset can be at any age.

Signs & Symptoms

LEMS is characterized by weakness and fatigue especially of the pelvic and thigh muscles. The disease may affect the patient’s ability to engage in strenuous exercise and may make such activities as climbing stairs or walking up a steep walkway difficult. Onset is gradual, typically taking place over several weeks to many months. There is often a progression of symptoms whereby the shoulder muscles, muscles of the feet & hands, speech & swallowing muscles and eye muscles are affected in a stepwise fashion. The symptoms progress more quickly when LEMS is associated with cancer. Most LEMS patients also exhibit the following symptoms (sometimes called autonomic symptoms): dry mouth, constipation, impotence and, decreased sweating. LEMS patients with or without cancer may also undergo significant weight loss. The tendon reflexes are diminished or absent on examination. Hence, in summary, LEMS is often described as a clinical “triad” of proximal muscle weakness, autonomic symptoms and reduced tendon reflexes.

Causes

LEMS is an autoimmune disorder. Autoimmune disorders are caused when the body’s natural defenses against “foreign” or invading organisms (e.g., antibodies) begin to attack healthy tissue for unknown reasons. LEMS occurs because autoantibodies damage the “voltage-gated calcium channels (VGCC)” on the motor nerve membrane at the neuromuscular junction. These channels normally conduct calcium into the nerve resulting in release of a chemical known as acetylcholine. Acetylcholine helps in the communication between nerve cells and muscles and is one of a group of chemicals known as neurotransmitters, which help to transmit nerve impulses. The autoantibodies attack the VGCC resulting in less acetylcholine release.

In LEMS cases associated with cancer, it is believed that autoantibodies created against the VGCC on the small-cell lung tumor damage the VGCC on the nerve. It is unknown what causes autoantibody production in cases not associated with cancer.

In LEMS cases associated with cancer, affected individuals nearly always have a long smoking history.

Affected Populations

There are approximately 400 known cases of Lambert-Eaton myasthenic syndrome in the United States. These include both males and females, but when LEMS is associated with SCLC, the patients tend to be older and are more likely to be men than women. The average age of onset of SCLC is around 60 years. Around 3% of SCLC patients develop LEMS. Clinical symptoms of LEMS usually precede the SCLC diagnosis (sometimes by many years).When LEMS is not related to cancer, the syndrome may occur at any age.

Related Disorders

Symptoms of the following disorders can be similar to those of Lambert-Eaton myasthenic syndrome. Comparisons may be useful for a differential diagnosis:

MYASTHENIA GRAVIS (MG) is a chronic neuromuscular disease characterized by weakness and abnormally rapid fatigue of the voluntary muscles, with improvement following rest. Any group of muscles may be affected, but those around the eyes and the muscles used for swallowing are the most commonly involved. In LEMS, eye muscle weakness, when present, tends to be mild and, unlike with MG, is almost never the only symptom of the disease. Severe respiratory muscle weakness, which can be fatal in MG is rare in LEMS. On the other hand, the autonomic symptoms which affect most LEMS patients, are not present in MG. For more information on this disorder, choose “myasthenia” as your search term in the Rare Disease Database.)

GUILLAIN-BARRE SYNDROME is an autoimmune disease which occurs when the body’s defense system attacks the nerves, damaging the nerve’s myelin and axon. Nerve signals are delayed and altered, causing weakness and paralysis of the muscles of the legs, arms, and other parts of the body. Abnormal sensations such as numbness or tingling also occur. If muscle nerves are damaged, the patient experiences aching and weak muscles, shortness of breath, and difficulty in swallowing. If the autonomic nervous system is damaged, the patient may experience alterations of blood pressure, heart rate, vision, body temperature, bladder function, and blood chemistries. (For more information on this disorder, choose “Guillain” as your search term in the Rare Disease Database.)

Diagnosis

Diagnosis of LEMS is based on clinical symptoms and signs, electrophysiological studies, and antibody testing.

Electromyography initially shows a small amount of electrical activity in the muscle. After high frequency repetitive stimulation or exercise, there is increased activity in the muscle.

Anti-VGCC antibodies are detectable in around 85% of LEMS patients and when detected they are highly specific for the condition, or by measuring VGCC* antibodies in the blood.

Screening for SCLC* is a very important part of the diagnostic workup for LEMS. A chest CT (and sometimes FDG-PET) scan will usually form the basis of this screening. Depending on the risk profile, a negative initial screen will be repeated at suitable time intervals. A recently discovered tumor marker antibody directed against SOX, which is found in 65% of SCLC LEMS patients as opposed to only 5% of non-tumor LEMS patients may help guide clinical practice in the future.

Standard Therapies
Treatment

Treatment of LEMS may vary depending upon the individual’s age, general health, and the presence of associated cancer to autoimmune disorder. If cancer is present, treatment of Lambert-Eaton myasthenic syndrome first involves treatment directed at the cancer. This may result in relief of LEMS symptoms.

In addition to symptomatic treatment, drugs that suppress the activity of the immune system (immunosuppressive drugs) are used in more severe cases of LEMS, e.g. prednisone (alone or in conjunction with azathioprine or cyclosporin).


VGCC – VOLTAGE GATED CALCIUM CHANNEL (AND ANTIBODIES TO THEM)

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4360501/
Logo of wjcc
World J Clin Cases. 2015 Mar 16; 3(3): 293–300.
Published online 2015 Mar 16. doi: 10.12998/wjcc.v3.i3.293

PMCID: PMC4360501
PMID: 25789302

Voltage gated calcium channel antibody-related neurological diseases

Can Ebru Bekircan-Kurt, Eda Derle Çiftçi, Aslı Tuncer Kurne, and Banu Anlar

ABSTRACT

VOLTAGE GATED CALCIUM CHANNEL (VGCC) antibodies are generally associated with Lambert-Eaton myasthenic syndrome. However the presence of this antibody has been associated with paraneoplastic as well as non-paraneoplastic cerebellar degeneration. Most patients with VGCC-antibody-positivity have small cell lung cancer (SCLC). Lambert-Eaton myasthenic syndrome (LEMS) is an autoimmune disease of the presynaptic part of the neuromuscular junction. Its classical clinical triad is proximal muscle weakness, areflexia and autonomic dysfunction. Fifty to sixty percent of LEMS patients have a neoplasia, usually SCLC. The co-occurrence of SCLC and LEMS causes more severe and progressive disease and shorter survival than non-paraneoplastic LEMS. Treatment includes 3,4 diaminopyridine for symptomatic purposes and immunotherapy with prednisolone, azathioprine or intravenous immunoglobulin in patients unresponsive to 3,4 diaminopyridine. Paraneoplastic cerebellar degeneration (PCD) is a syndrome characterized with severe, subacute pancerebellar dysfunction. Serum is positive for VGCC antibody in 41%-44% of patients, usually with the co-occurrence of SCLC. Clinical and electrophysiological features of LEMS are also present in 20%-40% of these patients. Unfortunately, PCD symptoms do not improve with immunotherapy. The role of VGCC antibody in the immunopathogenesis of LEMS is well known whereas its role in PCD is still unclear. All patients presenting with LEMS or PCD must be investigated for SCLC.

Keywords: Voltage gated calcium channel antibody, Lambert-Eaton myasthenic syndrome, Paraneoplastic cerebellar degeneration, Onconeural antibodies, Small cell lung cancer