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FTY720 (Fingolimod) Oral Medication for Multiple Sclerosis–Results of TRANSFORM Trial

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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)

Add a Comment7 Comments

Blogger

Please check back with us from time to time and let us know how the trials are going and (more importantly) how you are doing. It will be so exciting to get actual first hand feedback from someone involved with the study.

Here's wishing you good luck and sending positive thoughts for a good trial result for you.

December 21, 2009 - 10:17pm
EmpowHER Guest
Anonymous

i wish the best of luck to everyone. i am on the new trials of pills that will be 2 and 1/2 years and looking forward to participating. i was a researcher when able to work so it will be nice to help as a subject now. especially luck to the man who is experiencing all the side effects and getting worse.

December 11, 2009 - 5:45pm
Blogger

I wish your husband the best of luck in these trials. I know that we are on the verge of an alternative to the injection based treatments that are currently available. I'm hopeful that one of these new pills currently being tested will be a home run in terms of safety and effectiveness. Ultimately, I'm still praying for the cure. Best wishes to your husband.

November 16, 2009 - 10:58am
EmpowHER Guest
Anonymous

My husband has PPMS and is on the clinical trial, het started january 14th 2009.
Till now there is no improvement but he is getting worse.
But it is a 50% placebo trial, so we don't know if he is on the real drugs.
But he does has all kind of (possible)side effects.
We hope for the best, this trail for the PPMS patients takes 3 years.
If you want to share your experience: [email protected]

November 8, 2009 - 9:35am
EmpowHER Guest
Anonymous

I like to think that the high price I am paying for my current medication helps keep research moving on future MS drugs as there is a large potential market. In almost all cases, where there is a profit motive, there is investment.

August 24, 2009 - 6:28am

Outstanding news! This article shows the best of the pharmaceutical industry. Sometimes there is a real breakthrough.

August 11, 2009 - 7:30am
Blogger (reply to Linda Fugate PhD)

Yes! This is an exciting time in the research for MS and the preliminaries look good. A third company is also working on an oral medication as well but they are not as far down the pipeline as Merck and Novartis. I'm hoping that one of the drugs turns out to be as effective and as safe as the current therapies. If we get a "yes" on both of those issues, we'll have a real winner. I'll be watching the progress!!!

August 11, 2009 - 8:37am
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We value and respect our HERWriters' experiences, but everyone is different. Many of our writers are speaking from personal experience, and what's worked for them may not work for you. Their articles are not a substitute for medical advice, although we hope you can gain knowledge from their insight.

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