We are getting closer the release of the first oral medicines in the treatment of Relapsing-Remitting MS (RRMS) with the goal of preventing relapses (and hopefully progression and disability). Altruistic patients worldwide are taking part in multiple clinical trials testing the safety and efficacy (effectiveness) of these new forms of medicines.
These newer medicines are giving the MS Community hope because they do not involve needles, but also because they may be found to work even better (higher efficacy) than our older medicines. Unfortunately, however, two patients in one of the trials passed away recently from infections.
MS is an autoimmune disease, and so the body attacks itself (the myelin covering the nerves and the nerves themselves), much like in Diabetes mellitus type 1 (childhood diabetes) where the body attacks the pancreas (the organ which releases insulin). Because MS involves the immune system being hyperactive, many of the medicines to treat MS are immunosuppressants to try to push the immune system down. Immunosupression also makes it harder for the body to fight off infections.
Fingolimod (also called FTY720) is a novel oral medicine being tested in three Phase 3 trials both in the United States and worldwide). Phase 3 trials are large trials where patients either receive the medicine being tested (in this case, Fingolimod) or another standard medicine (or placebo). Fingolimod is being tested in two placebo-controlled Phase 3 trials (one in the United States and the other in the rest of the world) and in one Avonex®-controlled Phase 3 trial) where patients receive a daily pill and weekly injections into the muscle and they do not know if they are receiving the real pill and a fake injection or a fake pill (placebo) and a real injection (Avonx®). The idea of this trial is to compare the safety and efficacy of the new medicine Fingolimod with an older well-established medicine (Avonex®).
The patients volunteering for these trials are true heroes – they are pioneers leading the way for the development of new medications and making it possible for the entire MS community to have greater access to new medicines. Just as the patients who were in the original trails of Avonex®, Betaseron®, Copaxone® and Rebif® blazed the way for hundreds of thousands of MS patients to lead better lives, these patients in the newer clinical trials should be applauded and thanked for their commitment to helping all MS patients and families.
Being in a clinical trial, however, carries with it risk. If we knew everything about the safety and efficacy of the medicine being tested, then we wouldn’t need these trials. Therefore, there may be risks hich are not discovered until something bad happens.
This summer, Novartis (the makers of Fingolimod) announced that a patient in Italy receiving FTY720 in the clinical trial, developed disseminated Varicella Zoster (the infection of chickenpox and shingles) and passed away. Another patient in South Korea developed herpes encephalitis (an infection of the brain by the herpes virus) and also passed away.
The hearts and thoughts of the entire MS community go out to the families and friends of those heroes.
The MS research community is remaining vigilant in the earlier detection and diagnosis of potentially fatal infections while patients are being exposed to immunosuppressants. Patients in the Unites States are still enrolling in the placebo-controlled trial of FTY720 for the treatment of relapsing MS. Patients and doctors are keeping an eye out for any further infections, and the earlier they can be found, the better we can treat them with antibiotics and antiviral medications.
The recent deaths reemphasize the need to have an open dialogue with your neurologist, whether you are in a clinical trial or not, and to retiain and claim your true position as the center of the MS Team in M*STAR (the Multiple Sclerosis Team Approach Rule).
Published On: August 13, 2008