Gilenya is the first FDA-approved oral medication for Multiple Sclerosis (MS), a chronic, disabling illness with a significant
but largely undocumented impact on society. Gilenya is a broad pathfinder drug, with the potential to raise the profile of
MS as a public health priority, stimulate new science, and motivate clinicians and patients to seek a wider range of approaches
to ease the burden of disease. And there is the more dynamic element of market uncertainty that will make for an interesting
debut. High cost—and the ramifications of a controversial co-pay program—side effects, and patient compliance are issues for
Gilenya that cannot simply be washed down with a swig of water.
Dr. Jack Burks
On September 23, 2010—161 years after the disease was initially diagnosed —The Wall Street Journal called the FDA approval of Gilenya "another sign of the improving picture for MS treatment." Last month, The European Medicines
Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for Gilenya in patients with
highly active recurring-remitting MS (RRMS). John Golding, president of the European Multiple Sclerosis Platform (EMSP) called
Gilenya a "welcome alternative" for the more than 500,000 people in the EU who live with the disease. Switzerland and Australia
have also approved the drug. And several new drugs with a similar oral indication are on the way.
Plaudits aside, Gilenya may indeed signify the beginning of long-awaited change in the MS community: It treats the disease
in a new way, boasting an ease of use and efficacy profile that many competitors cannot hold a candle to. "It's an area of
intense fascination for people," says Trevor Mundel, global head of development for Novartis. "I take my hat off to the patients
for participating and for bearing with us ... It was a roller coaster of a program to develop this drug."
First diagnosed in 1849, Multiple Sclerosis currently affects between 250,000 and 350,000 people in the United States, with
as many as 200 new cases of MS diagnosed in the US every week, according to the National Institute of Neurological Disorders
and Stroke (NINDS). MS strikes women twice as often as men, and usually appears between the ages of 20 and 40, making it the
leading cause of disability among young adults in the nation.
MS is a disease of the central nervous system (CNS), affecting the brain and spinal cord. In individuals with MS, the body's
own immune system attacks the insulating layer of fatty tissue called the myelin sheath that surrounds and protects the nerves
of the CNS. This attack erodes the myelin sheath over time, producing scar tissue (sclerosis), which eventually disrupts the
communication between the CNS and the rest of the body. These communication disruptions can lead to mobility and vision impairments,
in serious cases resulting in an inability to walk and/or perform many activities of daily living (ADL) skills including cooking,
cleaning, exercise, or other activities involving strength, mobility, or endurance. Other possible symptoms of MS include
weakness, numbness, loss of muscle coordination, and problems with speech and bladder control.
The impact extends even further into the social interaction space. According to an article by Nicole M. Sabapathy and Clare
L. Minahan published in Clinical Rehabilitation and data from the "Sonya Slifka Longitudinal Multiple Sclerosis Study" in 2007, such reduced ability to undertake ADL activities
is also associated with higher levels of depression and decreased quality of life in those with MS.
Unfortunately, in addition to being diagnosed with a CNS disease and facing a reduced capacity for mobility-related activities,
potential treatments of MS thus far have done little to improve the quality of life and instances of depression among this
population. MS is commonly a progressive and relapsing disease; therefore, treatments can only aim to slow the progression
of physical disability and increase the time between relapses.
Dr. Jack Burks
"It is extremely dangerous to use improved 'quality of life' verbiage around these drugs," says Julie Stachowiak—MS patient,
PhD, and author of The MS Manifesto. "Many people assume that taking medicine if you have a condition makes you feel better. The MS disease-modifying therapies
(DMTs) are different. Often they make us actually feel worse in the immediate than if we hadn't taken them."
Dr. Julie Stachowiak
Some of the most well known MS medications currently on the market are Rebif (Merck Serono), Copaxone (Teva Pharmaceuticals),
and Avonex and Tysabri (Biogen Idec). Rebif must be self-injected three times a week at roughly the same time on each injection
day and may have side effects including flu-like symptoms, depression, abdominal pain, increased liver enzymes, and blood
cell count decreases; Copaxone does not have many of these negative side effects, but can leave itchy, painful welts and must
be injected every day; and Avonex is injected only once a week but has many of the same side effects as Rebif. Treatment options
with inconveniences and side effects like these can be a breeding ground for patient noncompliance. "It's scary and it took
me a long time to get used to. And it's inconvenient to take the injections because when you're in your twenties, you don't
really want a set schedule to take meds," says MS patient L. Mello.
"DMTs do not fix people with MS; they slow down the rate at which we get worse," adds Stachowiak.