Competition can be a good thing, especially for the consumer. Recently, I reported that Merck submitted a request to the European Medicines Agency requesting approval for the use of oral Cladribine for the treatment of Multiple Sclerosis.
Since all the major treatment options for MS are currently injection or infusion based, a safe, effective oral medication for MS would be a breakthrough. Merck is not the only pharmaceutical company in the race to be first-to-market with an oral treatment for Multiple Sclerosis. Novartis AG is giving Merck a run for their money. They are actively pursuing approval for their own version of an oral MS medication, FTY720 (Fingolimod). Formal requests for approval are expected to be submitted later this year in both the US and Europe.
FTY720 is an “immune-modulating” drug. It works by binding to the receptor site on immune cells and then trapping them in the lymph nodes. Since the immune cells are trapped in the lymph nodes, the theory is that they are unable to go scampering about in our brains and cause the damage which results in MS symptoms. Phase III (code named TRANSFORMS) of the FTY720 trials has been completed and Novartis presented the results earlier this year at the American Academy of Neurology convention in mid-April, 2009. The results were promising with 80-83% of all patients on FTY720 relapse free! No matter how you look at it, those are quite impressive results.
During the course of the trial, participants were given one pill, administered once a day. The dosage consisted of either 0.5 mg or 1.25 mg. It was reported that the 0.5 mg worked better than 1.25 mg. During the course of the trial, Novartis specifically compared FTY720’s performance to Avonex. Avonex is currently in the number one spot in the MS medication marketplace. Novartis reported that the 1-year relapse rate was reduced by 52% in low-dose patients and 38% in high dose patients compared to Avonex. The long-term trial results were also excellent reporting as follows:
• 67% remained relapse free after three years
• 89% free of inflammatory disease activity (confirmed via MRI scans)
• 75% free of new or enlarged lesions
• Relapse rates remained low after 4 years
All of the MS medications have side effects and FTY720 is no exception. The most common reported side effects were headache, fatigue (not good since MS patients already have abnormal fatigue), flu and other cold symptoms. While annoying, these are no worse than side effects of other currently approved MS medications.
However, in addition to the commonly reported side effects, there are some safety concerns about FTY720, including:
• Increased risk of heart and liver problems
• Elevated liver enzymes (possible sign of liver damage)
• Lowered heart rate
• Increased blood pressure
• Skin cancer (Seven cases of skin cancer were reported. All were successfully treated.)
• Fatal herpes infections (Two patients died of fatal herpes infections. Novartis reported that it was unclear whether or not FTY720 played a role in the development of the infections and the subsequent deaths. However, since FTY720 suppresses the immune system, FTY720 could not be excluded as a contributing factor in the deaths.)
• Haemorhaging focal encephalitis (i.e. inflammation of the brain with bleeding. As with the herpes infections, FTY720’s role could not be confirmed or excluded as a cause.)
Whether it’s because the pharmaceutical companies have listened to our “voice” as we expressed the need for a non-injection based medicine, or simply that they recognize the business opportunity that an oral medication presents, it looks as if MS patients will finally have choices in treatment options in the near future. Regardless of the reason, I’m glad that we are finally going to have some treatment options and am looking forward to the day when shots are not a part of my life.
However, I’m also not going to rush in blindly. As with any new treatment option, the first question to be asked is “Is this effective?” The second question must be “Is it as safe as what I take now?” If either FTY720 or Merck’s oral Cladribine can prove a resounding “yes” to both questions, then we just may have a real breakthrough in MS treatments on the horizon.
© 2009 Mary Kyle.
Resources:
FTY720 (Fingolimod), The Multiple Sclerosis Resource Centre, http://www.msrc.co.uk/index.cfm?fuseaction=show&pageid=1309
80-83 percent of patients taking multiple sclerosis drug FTY720 are relapse-free, Reuters.com © Thomson Reuters 2009 (29/04/09)
Multiple Sclerosis drug FTY720 reduces relapses, The Street.com © 1996-2008 TheStreet.com, Inc. (12/12/08)
FTY720 Multiple Sclerosis therapy exerts differential effects on T cell subsets, The Street.com © 1996-2008 TheStreet.com, Inc. (12/12/08)
FTY720 trial continues despite death, Source: Reuters © Reuters 2008 (09/06/08)
Oral drug, FTY720, reduces disease activity in Multiple Sclerosis, Source: American Academy of Neurology (16/04/08)