New Treatment Offers Hope for Lupus Patients

By Dr. Eric Morand, Monash University, Australia

When real patients have unprecedented positive outcomes to a new treatment, it’s tempting to talk about it as “breakthrough” for medical science. This describes the excitement around a new report from researchers in Germany of a radical new treatment for lupus.

The patients in the study – five people with severe lupus – went into remission following pioneering CAR T-cell treatment, which uses genetically altered cells.

What is lupus, why is this such big news, and what could it mean for other patients and diseases?

Around 5 million people are affected by some form of lupus worldwide. The most common form of lupus is technically known as systemic lupus erythematosus. Though not widespread, it is more common than multiple sclerosis (MS). Both are “autoimmune” diseases where the immune system attacks its owner instead of the germs it is supposed to fight.

MS is an autoimmune disease where the immune system attacks nerve tissue. In contrast, lupus can affect any organ in the body. Treatments for lupus have been so poor for so long that even wealthy and famous people with the disease – like pop star and actor Selena Gomez – have had organ failure resulting in the need for a kidney transplant. A lot of complicating factors have made it hard to improve outcomes for people with the disease.

Firstly, the variety of tissues lupus can affect means no two patients are exactly alike. Diagnosis is hard and often delayed. This also means we researchers have to deal with a lot of complexity as we try to work out what is causing the disease. This clinical variability makes measuring improvement in response to treatment difficult, and many clinical trials have likely failed due to measurement issues.

Second, there is variation between patients in which part of the immune system goes wrong. This means different patients will need different treatments – and we still do not know with certainty how to get this right. But progress is happening fast.

Innate and Adaptive Immunity

The immune system is in two parts, innate and adaptive.

The “innate” immune system responds fast but non-specifically to viruses and other germs that hit the body with a slug of germ-killing inflammatory proteins. The “adaptive” immune system is slower but more precise. It swings into action after the innate immune system and provides long lasting defense against the invading germ.

When you are vaccinated against a disease (such as COVID), the fever and aches you might get in the first day or two is your innate immune system at work. But the long-lasting protection from antibodies is provided by a part of your adaptive immune system, a key part of which is delivered by cells called “B cells”.

In lupus, both parts of the immune system are involved, and both have been successfully used to develop medicines. Earlier this year, the Therapeutic Goods Administration approved anifrolumab, a drug which blocks “interferon”, a crucial protein made by the innate immune system.

Another drug which works on B cells of the adaptive immune system, called belimumab, was approved a few years ago. Unfortunately, neither drug is on Australia’s Pharmaceutical Benefits Scheme yet, so access is extremely limited.

However, we now know that interferon and B cells are both important, and so very strong treatments that almost completely eradicate either could be useful. That is where this potential new treatment comes in.

Already Used to Treat Cancer

Treatments to destroy B cells are used in cancers like lymphoma. The most powerful of these uses CAR-T cells, which train a type of natural cell to be an assassin of the B cell.

CAR-T medicines are highly complex to make, and extremely expensive – but they work.

T cells are collected from the blood, then re-engineered in a special laboratory.

Now, this new report shows targeting B cells using this approach could be effective in lupus too. Building on a first-ever patient treated in this way by the same group a year ago, doctors in Germany created a “homemade” CAR-T treatment and used it in five patients with severe lupus.

Remarkably, all five patients had near complete eradication of disease, allowing them to stop conventional medicines, like steroids, with potentially harmful side effects.

What This Means for Other Patients

So what does it mean for patients in Australia? Well, most centres aren’t able to make their own CAR-T treatments, so delivering this potential treatment will require a commercial approach.

However, it might be quicker to market than other treatments in development as it takes a proven approach into a new disease, rather than being new from the ground up.

One day we might even be able to extend such treatments to other autoimmune diseases, like MS, where B cell-directed treatments have been helpful, as well as in lupus.

This would need to be balanced against risk. Importantly, short term side effects of CAR-T treatment (which include brain and bone marrow problems) can be severe. For this reason, such a treatment would only be used for the most severe cases in which standard treatments have failed, like the patients in the German trial.

Long-term side effects are also unknown at this time, and of course suppressing the immune system so profoundly in the setting of a pandemic is not without major risks.

Formal trials of a commercial CAR-T medicine for lupus are in the advanced planning stages already, and Australia is likely to be front and centre of these due to our lupus expertise and trial-friendly regulatory environment. With all these advances, we can at last tell our patients, and our friends and family with lupus, that there is light at the end of what has been a very long tunnel.

Eric Morand, MD, is a clinical rheumatologist and Head of the School of Clinical Sciences at Monash Health, Monash University in Australia.  Dr. Morand consults with companies involved in lupus drug development, including Novartis and AstraZeneca. He receives funding from Australia’s National Health and Medical Research Council and Lupus Research Alliance US, and is a Director of Rare Voices Australia.

This article originally appeared in The Conversation and is republished with permission.

Survey Finds Over Half of MS Patients Abused by Caregivers

By Pat Anson, PNN Editor

People with multiple sclerosis already have enough problems living with a progressive disease and sky-high medical bills. But now there’s word that many of them are being abused by their own caregivers.   

Researchers at the University of California, Riverside surveyed 206 patients with advanced MS and found that over half of them were being abused in some way by an unpaid caregiver who was often a member of their own family. The mistreatment ranged from psychological (44%) and financial abuse (25%) to neglect (16.5%) and physical abuse (11%). Over 8 percent of patients said they were abused sexually.   

"We knew we would find some level of abuse and neglect, but we were surprised by how prevalent it is," said Elizabeth Morrison-Banks, MD, a health sciences clinical professor at the UC Riverside School of Medicine, who led the study. "The findings of this study represent a collective cry for help from so many families affected by multiple sclerosis across the United States."

MS is a chronic and disabling autoimmune disease that attacks the body’s central nervous system, causing pain, numbness, difficulty walking, paralysis, loss of vision and fatigue.

"Some people live with MS for many years but with milder symptoms, and they may remain completely self-sufficient and never require a caregiver,” Morrison-Banks explained. “Others are less fortunate and develop neurological disability that can make them vulnerable to abuse and neglect if they are unable to move around independently, take care of their own finances, or get away from the situation when family conflict escalates. These problems are compounded if the person with MS and family lack financial resources."

A recent study found over 75% of American adults with MS face financial hardship that has forced them to cut spending on food, clothing and housing. Many have gone into debt or filed for bankruptcy, and over a third have delayed or stopped filling prescriptions because they can’t afford them. The average cost of disease-modifying MS drugs is about $76,000 a year.

For MS patients who are struggling financially, hiring a professional caregiver is not an option, so many have to rely on family caregivers.

"Some family caregivers are also working full time, caring for children or other family members, and sometimes dealing with health issues of their own," explained Morrison-Banks. "I want to emphasize that the majority of family caregivers do not mistreat those they care for, even in situations that can be very challenging. Nonetheless, it is important to recognize the risk factors for mistreatment of people with disabilities, and do what we can to identify, mitigate, and prevent abuse and neglect."

Other risk factors for mistreatment include MS patients with higher levels of cognitive impairment, caregivers having a mental health problem, alcohol use by the caregiver or patient, and low levels of social support within the family.

"Being a full-time family caregiver for someone with substantial neurological disability often presents significant challenges," Morrison-Banks said. "Many families take these challenges in stride, but others end up in situations of abuse and/or neglect."

The survey findings have been published in the journal Multiple Sclerosis and Related Disorders. The research paper is believed to be the first to document the nature and extent of caregiver mistreatment of MS patients in the United States.

The survey did not include patients who had paid caregivers or trained clinicians. A study of paid caregivers is an important next step for the research team. The National Multiple Sclerosis Society funded the UC Riverside study.

Over 75% of MS Patients Face Financial Hardship

By Pat Anson, PNN Editor

Over 75% of American adults with multiple sclerosis face financial toxicity or hardship that has forced them to cut spending on food, clothing and housing. Many have gone into debt or filed for bankruptcy, and over a third have delayed or stopped filling prescriptions because they can’t afford them.

The findings come from a survey of 243 multiple sclerosis (MS) patients conducted by the Harvey L. Neiman Health Policy Institute. The study is the first of its kind to evaluate how financial hardship is forcing MS patients to forego treatment and make drastic changes in their lifestyles and spending.

In recent years, the cost of prescriptions for many disease-modifying MS drugs has nearly tripled to about $76,000 a year. While insurance pays for most of it, many patients are overwhelmed by deductibles and other out-of-pocket expenses. The lifetime cost of treating MS in the United States is estimated at over $4 million per patient.

“Over the last 20 years, higher out-of-pocket costs for advanced imaging tests and increased cost sharing have caused the financial burdens on MS patients to escalate. Among medically bankrupt families, MS is associated with the highest total out of-pocket expenditures exceeding those of cancer patients,” said lead author Gelareh Sadigh, MD, an assistant radiology professor at Emory University School of Medicine.

“Our study results demonstrate the high prevalence of financial toxicity for MS patients and the resulting decisions patients make that impact their health care and lifestyle.”

More Debt, Less Spending

The findings, published in the Multiple Sclerosis Journal, show that over half of MS patients (56%) reported decreases in their income due to disability, unemployment or retirement. To make ends meet, many cut spending on food and clothing (35%) and leisure activities (50%) or withdrew money from their savings (40%) and retirement accounts (15%). Others went into debt by borrowing money (19%) or charging their credit cards (30%).

Over a third of MS patients decided to forego some type of medical care or treatment, such as not filling a prescription (16%), skipping doses (13.5%) or stopped taking medication (13%).

“These data underscore the need for shared decision-making and an awareness of patient financial strain when planning treatment strategies,” said co-author and Neiman Institute researcher Richard Duszak, MD, a professor and vice chair for health policy at Emory University. “In addition to the impact on adherence, financial toxicity was associated with significantly lower physical health-related quality of life, demonstrating the broad consequences of treatment costs for many MS patients.”

MS is a chronic and progressive disease that attacks the body’s central nervous system, causing pain, numbness, difficulty walking, paralysis, loss of vision and fatigue. Disease modifying therapy (DMT) reduces the frequency and severity of MS flare-ups, but many patients can’t afford the drugs.

A 2019 survey by the National Multiple Sclerosis Society found that 40% of MS patients who take a DMT drug altered or stopped taking their medication due to the high cost. According to Healthcare Bluebook, a 30-day supply of a brand name DMT like Gilenya costs about $8,845, or over $106,000 a year.

Criticism of the high cost of MS drugs is growing. Last year when the FDA approved a new MS medication called Vumerity, drug maker Biogen set its wholesale price at $88,000 a year. That brought a rebuke from the National MS Society, which released a statement that accused Biogen of price gouging.

Cost of MS Drugs Nearly Tripled

By Pat Anson, PNN Editor

The cost of prescriptions for disease-modifying multiple sclerosis (MS) drugs nearly tripled in the last decade, even with the introduction of cheaper generic versions, according to a new study funded by the National Multiple Sclerosis Society.

Researchers found spending on 15 MS drugs in the Medicaid program increased from $453 million in 2011 to $1.32 billion in 2017.

“Most of these drugs cost more than $70,000 per year on average and costs for these drugs are among the highest drug cost areas for private insurers as well as Medicare and Medicaid,” said Daniel Hartung, PharmD, of Oregon State University in Portland. “Unfortunately for people with MS, the introduction of a generic drug had a minimal effect on prices overall.”

Hartung and his colleagues found that when a generic version of the drug glatiramer acetate (Copaxone) was introduced in 2015, it was only 15 percent cheaper than the brand name drug made by Teva Pharmaceuticals.

Teva also worked to maintain its market share by encouraging doctors and patients to switch from a 20 mg dose of Copaxone to a 40 mg dose, which was not interchangeable with the new generic.

A second company introduced a generic version of glatiramer acetate in October 2017. Only then did the cost start to come down and generic versions started to get a greater share of the MS market.  

“After our study was complete, the company that introduced the second generic drug dropped its costs significantly, making it the lowest cost disease-modifying drug for MS on the market,” Hartung said. “Despite this, there is an urgent need for more robust competition from generics within these MS drugs.”

A similar study published last year found that Medicare paid nearly $76,000 annually per patient for disease modifying therapy (DMT), which reduces the frequency and severity of MS flare-ups. MS is a chronic and progressive disease that attacks the body’s central nervous system, causing pain, numbness, difficulty walking, paralysis, loss of vision, and fatigue.

Many Patients Can’t Afford DMT Meds

A recent survey by the National Multiple Sclerosis Society found that 40% of MS patients who take a DMT drug altered or stopped taking their medication due to the high cost. Only 11% said they could afford the medication without financial assistance.

“People with MS are paying the price, not only financially, but also physically and emotionally,” Bari Talente, executive vice president of advocacy for the National MS Society said in a statement.

“When someone alters or stops the use of their DMT, it can lead to increased symptoms, relapses, stress and anxiety. We need to make these medications affordable and accessible so people already facing a chronic illness don’t have to deal with deciding between buying groceries for their families or paying for their medication.”

The FDA recently approved the first generic versions of Gilenya (fingolimod) for the treatment of relapsing forms of multiple sclerosis (MS). A 30-day supply of brand name Gilenya 0.5mg capsules currently costs about $8,482, according to Healthcare Bluebook, or nearly $102,000 a year.

Few people actually pay the full amount for a DMT drug. About 45% of MS patients do not pay anything out-of-pocket for their DMT. The average annual cost among those who do pay is about $2,300.

Research Explores Cannabis as Treatment for MS, Alzheimer’s and Huntington’s Disease

By Pat Anson, PNN Editor

The University of Alberta is partnering with a Canadian cannabis company in three research projects exploring the use of medical cannabis for the treatment of multiple sclerosis, Alzheimer’s and Huntington’s diseases. 

Atlas Biotechnologies is investing nearly $300,000 over the next two years to fund the studies. Atlas operates a 38,000 square foot cannabis production facility near Edmonton and will supply customized blends of cannabis products to U of A researchers.

“People are touting (cannabis) for all kinds of things, but without solid scientific evidence,” said Ross Tsuyuki, PharmD, chair of the Department of Pharmacology at U of A. “But there likely are benefits for some conditions.”

The most well-known chemical compounds in cannabis are tetrahydrocannabinol (THC) and cannabidiol (CBD), but the plant has hundreds of other active biological chemicals, each with the potential of having therapeutic benefits. The goal of the research is to identify what specific compounds or combinations of compounds are effective.

“We've got to figure out the best combination of those compounds and how they're actually working in people,” Jeffrey Gossain, Atlas’ chief operating officer, told Folio, the University of Alberta's news site.  “A lot of people will tell you, 'My mom had cancer’ or, ‘My friend had an illness, and they took cannabis and it helped.’ But then for other people they don't have as effective results. 

“Part of the problem is that you don't really know what product they took, how they dosed it or the combinations of chemicals in the product that helped. It's not as simple as just saying, ‘The plant's got THC and CBD.’ You've got to get a lot more detailed than that.”

The research will examine whether CBD and other cannabinoids can relieve pain in patients with multiple sclerosis; if cannabis can reduce neuroinflammation and degeneration of the brain caused by Huntington's disease; and if cannabinoids have neuroprotective activity in models of Alzheimer’s disease.

“Alzheimer's disease, chronic pain, multiple sclerosis and Huntington's disease are all devastating conditions that don't have a lot of effective treatments,” said Tsuyuki. “If we find something, even if it works just a little, that could be an enormous advance for patients. But we have to do our homework first, and that is where we're starting.”

In addition to its partnership with the U of A, Atlas is collaborating with Harvard Medical School in developing cannabis products for pain and other neurological conditions.

A recent study found that medical cannabis is mildly effective in relieving pain and other symptoms in patients with multiple sclerosis (MS). Spanish researchers analyzed 17 clinical trials involving different combinations of THC and CBD, and found cannabis had limited effectiveness in relieving pain, muscle spasticity and bladder dysfunction.

MS is a chronic and incurable disease which attacks the body’s central nervous system, causing numbness in the limbs, difficulty walking, paralysis, loss of vision, fatigue and pain.

FDA Approves Generic Gilenya for Treatment of MS

By Pat Anson, PNN Editor

The U.S. Food and Drug Administration has approved the first generic versions of Gilenya (fingolimod) for the treatment of relapsing forms of multiple sclerosis (MS). That’s welcome news for patients who have long struggled with the exorbitant cost of many MS medications.

A 30-day supply of Gilenya 0.5mg capsules currently costs about $8,130, according to Healthcare Bluebook, or about $97,560 a year.

“Approving safe and effective generics so patients have more treatment options continues to be a priority for the FDA,” Janet Woodcock, MD, director of the FDA’s Center for Drug Evaluation and Research, said in a statement. “Having access to affordable treatments is important for patients with conditions that require ongoing care. The FDA has a longstanding commitment to increasing patient access to lower-cost, high-quality generic medicines.”

The FDA approved generic fingolimod applications from HEC Pharm Co. Limited, Biocon Limited and Sun Pharmaceutical Industries Limited.

Until now, Novartis held the exclusive patent rights to fingolimod, which is sold under the brand name Gilenya. Nearly 300,000 people worldwide have taken Gilenya since it was approved by the FDA in 2010, according to Novartis.

MS is a chronic and progressive disease that attacks the body’s central nervous system, causing numbness in the limbs, difficulty walking, paralysis, loss of vision, fatigue and pain. For most people with MS, there are periods of remission followed by relapses or flareups as the disease progresses. Gilenya is a widely used treatment for relapsing MS.

Generic formulations are often significantly cheaper than brand name drugs. In July, the FDA approved the first generic forms of Lyrica. Today a two-month supply of Lyrica costs about $472, while the same amount of generic pregabalin costs just $21.

Maximizing Profit

Criticism about the high cost of branded MS drugs is growing. Last month when the FDA approved a new MS medication called Vumerity, drug maker Biogen set its wholesale price at $88,000 a year. That brought a rare rebuke from the National Multiple Sclerosis Society, which released a statement that accused Biogen of price gouging.

It can take years for a new drug to get FDA approval and the pharmaceutical industry has long claimed that it needs to set prices high to recover the cost of research, clinical studies and drug development.

That claim is discounted in a recent article published in the journal Neurology, in which researchers asked four drug industry executives about the high cost of MS drugs. The executives were given anonymity to encourage them to speak freely.  

"I would say the rationales for the price increases are purely what can maximize profit," one executive said. "There's no other rationale for it.”

In setting the price for a new drug, executives said they primarily look at what their competitors are charging for similar medications. Companies fear that undercutting competitors with a lower price would undermine the attractiveness of their product.

"We can't come in at less," one of the executives said. "That would mean we're less effective, we think less of our product, so we have to go more."

The problem is unique to the United States, the only developed country that doesn’t have a universal healthcare system that regulates prices.

"And it is only in the United States, really, that you can take price increases. You can't do it in the rest of the world. In the rest of the world, prices decline with duration in the marketplace," another executive said.

Researchers say their study provides new insight into the economics behind pharmaceutical pricing.

"The frank information provided by these executives pulls back the curtain of secrecy on how drug price decisions are made," said co-author Dennis Bourdette, MD, chair of neurology at the Oregon Health & Science University School of Medicine. "We see that it is indeed the race to make more money that is driving up drug prices and nothing more."

In the meantime, MS patients like Jennifer Hochgesang struggle to make their co-payments and deductibles. She gets injections of glatiramer acetate, a generic version of Copaxone.

“It costs over $5,500 a month. When you add my migraine medication and other medications, it adds up to over $13,000 a month. I reach catastrophic level by March in my insurance and that’s the only way I can pay for it,” Hochgesang said. “It doesn’t cost that much in Europe though and it shouldn’t cost that much here.

“It seems sick to me that companies use a drug like this to profit highly from, when people can’t reasonably afford five thousand dollars a month. I believe an MS drug should be accessible to anyone. Otherwise we are only creating medicine for the rich and those able to get insurance or be on disability, leaving the rest out in the cold. That just isn’t right.” 

Biogen Accused of Price Gouging for New MS Drug

By Pat Anson, PNN Editor

FDA approval of a new multiple sclerosis (MS) drug has resulted in a big payoff for one company and sharp criticism from a patient advocacy group.

Last month the FDA approved Vumerity (diroximel fumarate) for the treatment of relapsing-remitting and secondary-progressive MS, as well as management of clinically isolated syndrome (CIS), neurologic symptoms that can be an early sign of MS — a chronic and progressive disease that attacks the body’s central nervous system.

Vumerity was jointly developed by Biogen and Alkermes. Under the terms of their operating agreement, FDA approval triggered a clause in which Biogen paid Alkermes $150 million for the worldwide commercial rights to Vulmerity, along with a share of future royalties.

Biogen said it would account for the Alkermes payment by amortizing its cost “over the expected useful life of the product.” It then announced the price of Vumerity – at a wholesale acquisition cost (WAC) in the U.S. of $88,000 per year. Biogen claimed that was “the lowest annual WAC price for oral MS disease-modifying therapies.”

MS drugs are notoriously expensive, but the $88,000 price tag for Vumerity brought a rare rebuke from the National Multiple Sclerosis Society, which released a statement that basically accused Biogen of price gouging.

“Vumerity is an efficacious and tolerable treatment option for people with relapsing MS, but being priced only $500 lower than the least expensive oral disease modifying treatment, does not show the commitment to affordable access that we had hoped,” said Bari Talente, executive vice president of advocacy for the National MS Society.

“We know that high wholesale acquisition cost (WAC) prices for MS disease modifying treatments put a heavy burden on people with MS. Too many are forced to take on high out-of-pocket costs, navigate through complex systems, and face varied and unpredictable decisions by public and private payers and pharmacy benefit managers.”

The statement points out Biogen has steadily escalated the price of another MS product, Tecfidera, by $40,000 since its launch in 2013. A year’s worth of treatment with Tecfidera now costs nearly $95,000.

“We urge Biogen to publicly commit to keeping price increases lower than the rate of inflation,” Talente said.

A recent study found that prices of several MS drugs have soared over the past decade, to an average of nearly $76,000 per patient annually.

“The pharmaceutical and biotechnology industries claim that the high prices reflect the expense of research and development and need to incentivize continued innovation. These claims are never backed up with transparent data,” said Daniel Hartung, PharmD, and Dennis Bourdette, MD, in an editorial in JAMA Neurology. “These drugs have long since recouped any cost of drug development, yet their prices have continued to rise.

“What is driving this increase is uncertain. However, the simplest explanation is that pharmaceutical and biotechnology companies increase prices because they can, they do it to increase their profit margins, and there are few limits on what they can charge.”

Biogen Involved in Illegal Co-Pay Charity

Biogen is one of three companies accused by federal prosecutors of paying a Florida-based charity to operate an illegal co-pay assistance program that helped Medicare patients buy high-priced MS drugs. The payments are considered kickbacks under a federal law that prohibits companies from subsidizing Medicare patients.

In a settlement announced Wednesday, The Assistance Fund (TAF) agreed to pay $4 million to resolve claims that it acted as a conduit for kickbacks from Biogen, Novartis and Teva Pharmaceuticals.

“Pharmaceutical companies and foundations cannot undermine the Medicare program through the use of kickbacks disguised as routine charitable donations. TAF operated as a vehicle for specific pharmaceutical companies to pay kickbacks at the ultimate expense of the American taxpayers who support the Medicare program,” said U.S. Attorney Andrew Lelling.

The DOJ has been cracking down on co-pay charities and the companies that fund them. Over $840 million in fines and penalties have been collected from eight pharmaceutical companies (United Therapeutics, Pfizer, Actelion, Jazz, Lundbeck, Alexion, Astellas and Amgen) to resolve allegations that they used third-party foundations to funnel kickbacks to patients.

Cost of MS Drugs Soars Despite Competition

By Pat Anson, PNN Editor

The cost of multiple sclerosis drugs has soared over the past decade for Medicare patients to nearly $76,000 per patient annually, according to a new study published in JAMA Neurology.

"We're not talking about patients without health insurance here," said senior author Inmaculada Hernandez, PharmD, assistant professor of pharmacy at the University of Pittsburgh. "We're talking about insured patients, under Medicare. Still, they are paying much more for multiple sclerosis drugs than they were 10 years ago."

Hernandez and her colleagues looked at Medicare Part D claims data from 2006 to 2016 for disease modifying therapies (DMTs) that reduce the frequency and severity of multiple sclerosis (MS) flare-ups. MS is a chronic and progressive disease that attacks the body’s central nervous system, causing pain, numbness, difficulty walking, paralysis, loss of vision, and fatigue.

Some of the most widely used DMTs for treating MS are Copaxone, Tecfidera and Avonex. Although there’s a fair amount of competition between the drugs -- the FDA has approved 19 DMTs for MS – prices have risen in tandem for nearly all of them.

The annual list prices of the drugs more than quadrupled over the 2006-2016 study period, far outpacing inflation.

Not only did the researchers find steep increases in list prices -- the starting point before rebates, coupons or insurance kicks in -- but also in the ultimate costs to both Medicare and its beneficiaries.

"We wanted to see how increases in list prices translated to increases in out-of-pocket spending, and we discovered that actual price increases do get passed down to patients, and that can negatively affect access," said Hernandez.

Alvaro San-Juan-Rodriguez

When it was first introduced by Biogen in 1996, Avonex had an annual list price of about $8,700. Two decades later, Avonex costs nearly $76,000 per patient per year.

“The pharmaceutical and biotechnology industries claim that the high prices reflect the expense of research and development and need to incentivize continued innovation. These claims are never backed up with transparent data,” said Daniel Hartung, PharmD, and Dennis Bourdette, MD, in an editorial in JAMA Neurology. “These drugs have long since recouped any cost of drug development, yet their prices have continued to rise.

“What is driving this increase is uncertain. However, the simplest explanation is that pharmaceutical and biotechnology companies increase prices because they can, they do it to increase their profit margins, and there are few limits on what they can charge.”

Hartung and Bourdette say neurologists who prescribe DMTs should be more aware of their cost. A generic DMT made by Mylan, for example, sells for about $2,000 a month, compared to a branded version that sells for about $6,000.  

Out-of-Pocket Costs for Neurology Drugs Rise Sharply

By Pat Anson, PNN Editor

Out-of-pockets costs for medications to treat multiple sclerosis, peripheral neuropathy and other neurologic conditions rose sharply over 12 years, according to a new study that found the average monthly cost to patients for MS drugs rose nearly 2,000 percent.

One in six people lives with a neurologic disease or disorder, according to the American Academy of Neurology. The annual cost of treating neurologic disorders in the United States is more than $500 billion.

“With many new, high-priced neurologic drugs coming to market and a recent rise in use of high-deductible insurance plans, which shift costs to patients, it is likely out-of-pocket costs will continue to increase,” said lead author Brian Callaghan, MD, of the University of Michigan in Ann Arbor.

The study was published online in the journal Neurology.

Callaghan and his colleagues examined out-of-pocket costs for over 912,000 people with MS, neuropathy, epilepsy, dementia or Parkinson’s disease who were privately insured from 2004 to 2016.

Researchers found that out-of-pocket costs for MS drugs showed the steepest monthly increase. Patients paid an average of $309 a month in 2016, compared to just $15 in 2004. Costs for MS patients in high-deductible health plans were even higher, averaging $661 per month or nearly $8,000 a year.

Co-pays and deductibles for brand name medications for neuropathy, dementia and Parkinson’s disease also rose considerably.

“Everyone deserves affordable access to the medications that will be most beneficial, but if the drugs are too expensive, people may simply not take them, possibly leading to medical complications and higher costs later,” said Ralph Sacco, MD, President of the American Academy of Neurology.

Researchers said neurologists and other physicians usually do not know the cost of drugs they prescribe, so they don’t discuss alternative medications based on a patient’s disease, insurance plan, pharmacy and deductible.

“Out-of-pocket costs have risen to the point where neurologists should be able to consider the potential financial burden for the patient when prescribing medication, but they do not have this information available to them,” Callaghan said. “Neurologists need access to precise cost information for these drugs in the clinic so when they meet with patients to make treatment decisions, they can help minimize the financial burden.”

Even when a generic version of a drug becomes available, it can take years for out-of-pocket costs to drop substantially. It took five years for out-of-pocket costs for gabapentin, for example, to drop to those of other tricyclic anti-depressants after gabapentin went generic in 2004.

A 2015 study found an “alarming” increase in costs for MS drugs and suggested the price increases were coordinated by drug companies.

1 in 5 Multiple Sclerosis Patients Misdiagnosed

By Pat Anson, PNN Editor

Nearly one in five patients who are told they have multiple sclerosis are misdiagnosed with the autoimmune disease, according to a new study of patients referred to two MS treatment centers in Los Angeles. The patients spent an average of four years being treated for MS before receiving a correct diagnosis.

MS is a chronic disease that attacks the body’s central nervous system, causing pain, numbness, difficulty walking, paralysis, loss of vision, and fatigue. The symptoms are similar to those of several other chronic conditions – including neuropathy, migraine and fibromyalgia – which often leads to a misdiagnosis.

Researchers at the Cedars-Sinai Multiple Sclerosis and Neuroimmunology Center analyzed the cases of 241 patients who had been diagnosed by other physicians and then referred to the Cedars-Sinai or UCLA MS clinics.

Their findings, published in the journal Multiple Sclerosis and Related Disorders, indicate that 43 of the 241 patients (18%) with a previous diagnosis of MS did not meet the criteria for the disease.

"The diagnosis of MS is tricky. Both the symptoms and MRI testing results can look like other conditions, such as stroke, migraines and vitamin B12 deficiency," said lead author Marwa Kaisey, MD. "You have to rule out any other diagnoses, and it's not a perfect science."

The most common correct diagnoses was migraine (16%), radiologically isolated syndrome (RIS) (9%), spondylopathy (7%), and neuropathy (7%). RIS is a condition in which patients do not experience symptoms of MS even though their imaging tests look similar to those of MS patients.

The misdiagnosed patients received approximately 110 patient-years of unnecessary MS disease modifying drugs. Nearly half received medications that carry a known risk of developing progressive multifocal leukoencephalopathy, a potentially fatal brain infection.

"I've seen patients suffering side effects from the medication they were taking for a disease they didn't have," Kaisey said. "Meanwhile, they weren't getting treatment for what they did have. The cost to the patient is huge — medically, psychologically, financially."

The cost of disease modifying medications for an MS patient in the U.S. exceeds $50,000 a year. Investigators estimated that the unnecessary treatments identified in this study alone cost almost $10 million. 

Researchers hope the results of the study will lead to new biomarkers and improved imaging techniques to help prevent future MS misdiagnoses.

A similar study in 2016 also found that MS patients were often misdiagnosed. One third of the patients were misdiagnosed for a decade or longer, most took unnecessary and potentially harmful medication to treat a disease they didn't have, and some even participated in clinical trials for experimental MS therapies. About a third suffered from morbid thoughts of death.

New Therapy Helps Improve MS Symptoms

By Pat Anson, PNN Editor

An experimental stem cell therapy developed by Australian researchers is showing promise in treating patients with progressive multiple sclerosis (MS), the most difficult-to-treat form of the autoimmune disease.

MS is a chronic and incurable disease which attacks the body’s central nervous system, causing numbness, difficulty walking, paralysis, loss of vision, fatigue and pain. Most patients go through periods of remission before the condition worsens and turns into secondary progressive MS. In primary progressive MS, the disease steadily gets worse from the start, with no periods of remission.

Scientists at the University of Queensland extracted immune cells from patients who had either primary or secondary progressive MS. The cells – known as T-cells – were then “trained” in a laboratory to target and kill cells infected with the Epstein Barr virus.

When the altered T-cells were injected back into the bloodstream of 10 patients, seven said their symptoms improved. They had more energy, improved concentration, slept better, and had improved vision and balance. There were no serious side effects.

The Epstein Barr virus (EBV) has long been associated with MS, which is why the researchers targeted it. The virus also causes infectious mononucleosis, a glandular fever known as “mono.”

“Although this was an uncontrolled study, our finding of a substantial relationship between clinical response and EBV reactivity and polyfunctionality of the T-cell product, of which both the patients and examining neurologists were unaware, suggests that the clinical benefit might be due to the T cell therapy,” researchers reported in the journal JCI Insight.

“Our data add to the mounting evidence for a pathogenic role of EBV infection in MS. Because T-cells access all CNS (central nervous system) compartments, T-cell therapy targeting only EBV-infected B cells is a treatment modality that could offer favorable safety and durable efficacy.”

This was a Phase I trial, where the primary goal of researchers is to make sure a treatment is safe to use. More advanced studies with a larger number of patients are needed to see how well altered T-cells actually work on MS.

Cannabis Somewhat Effective in Treating MS

By Pat Anson, PNN Editor

Medical cannabis is mildly effective in relieving pain and other symptoms in patients with multiple sclerosis (MS), according to a new study published in JAMA Network Open.

Spanish researchers analyzed 17 clinical trials involving over 3,100 patients – one of the largest reviews to date on the efficacy of cannabinoids in treating MS. Overall, they found that cannabis was safe, but had limited effectiveness in relieving pain, muscle spasticity and bladder dysfunction.

“Small but statistically significant differences were found in favor of cannabinoids for all 3 symptoms,” Marissa Slaven, MD, and Oren Levine, MD, of Ontario’s McMaster University said in a JAMA commentary. “The authors conclude that cannabinoids provide a mild reduction in subjective outcome assessment of uncertain clinical significance and that they are safe.”

MS is a chronic and incurable disease which attacks the body’s central nervous system, causing numbness in the limbs, difficulty walking, paralysis, loss of vision, fatigue and pain.

Medications and disease modifying drugs currently used to treat MS can cost tens of thousands of dollars a year – so a low-cost alternative treatment would be welcomed by many patients.

Four different medical cannabinoids were used in the 17 trials that were evaluated. They contained different levels of cannabidiol (CBD) and tetrahydrocannabinol (THC), the active ingredient in marijuana that makes people high. A lot of uncertainty remains about whether CBD or THC are more effective in relieving MS symptoms – something the JAMA study failed to resolve.

“It is critical that researchers gain a deeper understanding of both of the major (THC and CBD) and minor components of this therapy to unlock its full potential,” said Slaven.

“Given the relative safety of these agents, lack of strong evidence of other effective treatment options, and increasing access in some jurisdictions, it may seem appealing to include cannabinoids in the armamentarium of therapies for MS. But carefully conducted, high-quality studies with thought given to the biologic activity of different cannabis components are still required to inform on the benefits of cannabinoids for patients with MS.  

"The bottom line is there is certainly something happening with cannabinoids in regard to symptoms," Nicholas LaRocca, vice president of healthcare delivery and policy research at the National Multiple Sclerosis Society, told HealthDay. "In spite of very strong interest in cannabinoid therapy, we really have relatively little in terms of good research to guide us in terms of what does and what doesn't work, what works for which types of individuals, and so forth."

A small study was recently launched in Australia that might answer some of those questions. Emerald Health Pharmaceuticals of San Diego is using a synthetic version of CBD – called EHP-101 -- to treat about 100 people who suffer from MS or scleroderma, another autoimmune disease. The placebo controlled Phase I trial is meant to determine whether EHP-101 is safe and has any side effects. Results are expected next year.

Vitamin D Levels May Help Predict Risk of MS

By Pat Anson, Editor

Vitamin D levels in the blood may help predict whether a person is at risk of developing multiple sclerosis, according to a large new study published online in the journal Neurology.

The findings provide the best evidence to date that low levels of Vitamin D may be a contributing factor to multiple sclerosis (MS), a chronic and incurable disease which attacks the central nervous system.

“There have only been a few small studies suggesting that levels of vitamin D in the blood can predict risk,” said study author Kassandra Munger, ScD, of the Harvard T.H. Chan School of Public Health in Boston. “Our study, involving a large number of women, suggests that correcting vitamin D deficiency in young and middle-age women may reduce their future risk of MS.”

Munger and her colleagues analyzed a database derived from blood samples taken during prenatal testing of over 800,000 Finnish women. Using hospital and prescription records, they were able to identify 1,092 of those women who were later diagnosed with MS. They were compared to a control group of 2,123 women who did not develop the disease.

Of the women who developed MS, 58% had deficient blood levels of vitamin D, compared to 52% of the women who did not develop the disease.

Deficient blood levels of vitamin D were defined as fewer than 30 nanomoles per liter (nmol/L). Insufficient levels were 30 to 49 nmol/L and adequate levels were 50 nmol/L or higher.

Researchers found that with each 50 nmol/L increase in vitamin D in the blood, the risk of developing MS later in life decreased by 39 percent. In addition, women who had deficient levels had a 43% higher risk of developing MS than women who had adequate levels.

“More research is needed on the optimal dose of vitamin D for reducing risk of MS,” said Munger. “But striving to achieve vitamin D sufficiency over the course of a person’s life will likely have multiple health benefits.

"Our results further support and extend those of previous prospective studies of (Vitamin D) levels in
young adults and risk of MS, and suggests that many individuals are exposed to an increased MS risk that
could be reduced by broad population-based programs to prevent vitamin D deficiency."

Participants in the study were primarily white women, so the findings may not be the same for other racial groups or men. Also, while the blood samples were taken an average of nine years before MS diagnosis, it is possible some women may have already had MS when their blood was drawn and were not yet showing symptoms of the disease.

MS causes numbness in the limbs, difficulty walking, paralysis, loss of vision, fatigue and pain. Symptoms begin with a series of irregular relapses, and after about 20 years MS worsens into a secondary progressive stage of the disease.

Low blood levels of vitamin D – known as the “sunshine vitamin”-- have previously been linked to an increased risk of developing MS. Danish researchers found that MS patients who spent time in the sun every day during the summer as teenagers developed the disease later in life than those who spent their summers indoors.

Ultraviolet rays in sunlight are a principal source of Vitamin D, which has a wide range of positive health effects, such as strengthening bones and inhibiting the growth of some cancers.

Vitamin D Lowers Inflammation from MS

By Pat Anson, Editor

A new study is adding to the growing body of evidence that Vitamin D supplements can be used to treat multiple sclerosis (MS) and other inflammatory chronic pain conditions.

The pilot study published by Johns Hopkins physicians in the journal Neurology found that taking a high dose of vitamin D3 is safe for people with MS and may help regulate the body’s hyperactive immune response.

“These results are exciting, as vitamin D has the potential to be an inexpensive, safe and convenient treatment for people with MS,” says study author Peter Calabresi, MD, director of the Johns Hopkins Multiple Sclerosis Center and professor of neurology at the Johns Hopkins University School of Medicine. “More research is needed to confirm these findings with larger groups of people and to help us understand the mechanisms for these effects, but the results are promising.”

MS is a chronic and incurable disease which attacks the body’s central nervous system, causing numbness in the limbs, difficulty walking, paralysis, loss of vision, fatigue and pain.

Low blood levels of vitamin D – known as the “sunshine vitamin”-- have been linked to an increased risk of developing MS.

People who have MS and low levels of vitamin D are also more likely to have greater disability and more disease activity.

In the Johns Hopkins study, 40 people with relapsing-remitting MS received either 10,400 international units or 800 international units (IU) of vitamin D3 supplements every day for six months. Patients with severe vitamin D deficiency were not included in the study. The current recommended daily allowance of vitamin D3 is 600 IU.

Blood tests at the start of the study, and after three and six months, measured the amount of vitamin D in the blood and the response in the immune system’s T cells, which play a key role in MS.

Participants taking the high dose of vitamin D reached optimal levels of Vitamin D in the blood (40 to 60 ng/ml), while the group taking the low dose did not reach that target. The people taking the high dose also had a reduction in the percentage of inflammatory T cells related to MS severity. The people taking the low dose did not have any noticeable changes in the percentages of their T cell subsets.

“We hope that these changes in inflammatory T cell responses translate to a reduced severity of disease,” says Calabresi. “Other clinical trials are underway to determine if that is the case.”

Another recent study in Neurology by Danish researchers found that MS patients who spent time in the sun every day during the summer as teenagers developed the disease later in life than those who spent their summers indoors. Ultraviolet rays in sunlight are a principal source of Vitamin D, which has a wide range of positive health effects, such as strengthening bones and inhibiting the growth of some cancers.

Low levels of serum vitamin D have also been linked to fibromyalgia. In a study of over 1,800 fibromyalgia patients published in the journal Pain Physician, researchers at National Taiwan University Hospital found a “positive crude association” between chronic widespread pain and hypovitaminosis D, which is caused by poor nutritional intake of Vitamin D, inadequate sunlight or conditions that limit Vitamin D absorption.

Pain News Network columnist Crystal Lindell began taking Vitamin D supplements when her blood levels were found to be very low. Within a few months she was feeling better, exercising more, and losing weight. You can read Crystal’s story by clicking here.

Sunlight May Delay Onset of Multiple Sclerosis

By Pat Anson, Editor

Exposure to sunlight may elevate your risk of sunburn, skin cancer and other health problems, but it appears to have a beneficial effect in delaying the onset of multiple sclerosis (MS).

Danish researchers found that MS patients who spent time in the sun every day during the summer as teenagers developed the disease later in life than those who spent their summers indoors. Their study, which was published in the online issue of Neurology, also found that people who were overweight at age 20 developed MS earlier.

"The factors that lead to developing MS are complex and we are still working to understand them all, but several studies have shown that vitamin D and sun exposure may have a protective effect on developing the disease," said study author Julie Hejgaard Laursen, MD, of Copenhagen University Hospital in Denmark. "This study suggests that sun exposure during the teenage years may even affect the age at onset of the disease."

MS is a chronic and incurable disease which attacks the body’s central nervous system, causing numbness in the limbs, difficulty walking, paralysis, loss of vision, fatigue and pain.

Ultraviolet rays (UVB) in sunlight are a principal source of Vitamin D, which has a wide range of positive health effects, such as strengthening bones and inhibiting the growth of some cancers.

In the Danish study, over 1,100 people with MS filled out questionnaires and gave blood samples. They were put into two groups based on their sun habits during their teenage years: those who spent time in the sun every day and those who did not. They were also asked about their use of vitamin D supplements during their teenage years and how much fatty fish they ate at age 20.

The people who spent time in the sun every day had an average onset of MS that was nearly two years later than those who did not spend time in the sun. On average, they developed MS at age 33, compared to 31 for those who were not in the sun every day.

"It appears that both UVB rays from sunlight and vitamin D could be associated with a delayed onset of MS," Laursen said. "However, it's possible that other outdoor factors play a role, and these still have to be identified."

Those who were overweight at age 20 developed MS about 1.6 years earlier than those of average weight and 3.1 years earlier than those who were underweight.

Previous studies have shown a relationship between MS and childhood obesity. Obese people are also known to have lower blood levels of vitamin D.

"The relationship between weight and MS might be explained by a vitamin D deficiency, but there's not enough direct evidence to establish this yet," Laursen said.

"A limitation of the study is the risk of recall bias because participants were asked to remember their sun, eating and supplement habits from years before," Laursen said. "In particular, someone with a long history of MS and onset of the disease at an early age, may wrongly recall a poor sun exposure. Additionally, only Danish patients were included into the study, so there should be caution when extending the results to different ethnic groups living in different geographic locations."