Future Medications and Trials
To translate pharmacology research into patient care, potential drugs ultimately have to be tested in people. This multistage process is known as clinical trials, and it has led researchers to validate life-saving treatments for many diseases, such as childhood leukemia and Hodgkin's disease. Clinical trials, though costly and very time-consuming, are the only way researchers can know for sure whether experimental treatments work in humans.

Researchers conduct clinical trials in three phases (I, II, and III), each providing the answer to a different fundamental question about a potential new drug: Is it safe? Does it work? Is it better than the standard treatment? Typically, researchers do years of basic work in the lab and in animal models before they can even consider testing an experimental treatment in people.

To test drugs in people, strict rules must followed that are designed to protect those who volunteer to participate in clinical trials. Special groups called Institutional Review Boards (IRBs) evaluate all proposed research involving humans to determine the potential risks and anticipated benefits. The goal of an IRB is to make sure that the risks are minimized and that they are reasonable compared to the knowledge expected to be gained by performing the study. Clinical studies can't go forward without IRB approval. In addition, people in clinical studies must agree to the terms of a trial by participating in a process called informed consent and signing a form, required by law, that says they understand the risks and benefits involved in the study.

Phase I trials
Studies test a drug's safety in a few dozen to a hundred people and are designed to figure out what happens to a drug in the body — how it's absorbed, metabolized, and excreted and usually take several months.
Phase II trials
Tests whether or not a drug produces a desired effect and take longer (from several months to a few years) and can involve up to several hundred patients.
Phase III trials
Studies further examines the effectiveness of a drug as well as whether the drug is better than current treatments. Phase III studies involve hundreds to thousands of patients, and these advanced trials typically last several years.

Many phase II and phase III studies are randomized, meaning that one group of patients gets the experimental drug being tested while a second, control group gets either a standard treatment or placebo. Usually phase II and phase III studies are "blinded" in that the patients and the researchers don't know who is getting the experimental drug. Finally, once a new drug has completed phase III testing, a pharmaceutical company can request approval from the Food and Drug Administration (FDA) to market the drug.

Following FDA approval, post-marketing studies (phase IV) might be conducted to assess long-term safety and effectiveness.

Currently there are a number of multiple sclerosis (MS) clinical trials looking into potential medications. These trials are not only being held in the United States, but all around the world as well. The fact that the research is global is very important since it provides a greater diversity of knowledge that is focused on this problem. The more diverse the research, studies, and trials are provides a greater chance that another drug will be approved and someday maybe even a cure found.

Some of these trials are further studies of Avonex, Betaseron, Copaxone, and Rebif but with the difference of them being administered orally. The largest problem with the interferons being taken orally is that it's a live protein and in that form it can't survive in the stomach. The stomach immediately breaks down the proteins in the medications before they can be absorbed into the body unlike aspirin and other non-protein medications.

There are also several other drugs being tested that fall in the oral category. One of these oral drugs as of early 2009 entered Phase 3 clinical trials which means it could be on the market as early as late 2009 or 2010 if all were to go well. This would be a huge milestone if any or all of these oral drugs proved to work. Up till now, all of the medications have been administered through an injection or an infusion. Just like anyone having to take a medication, a pill is almost always preferred over an injection or infusion. Most people, in there right mind, would rather take a pill than get a shot in their butt, arm, leg, stomach, etc...
Medications in Trials
Laquinimod is in the Phase 3 portion of trials after showing promising results with minimal side effects. This oral medication is yet another oral alternative to injections or infusions. The study currently currently beyond its original completion date of March 2011 and is designated as a Fast Track product by the FDA. As of April 2015, it remains in study.

The makers of Laquinimod are Teva Pharmaceutical Industries and Active Biotech.

Laquinimod is a once-daily oral, investigational, CNS-active immunomodulator with a novel mechanism of action being developed for the treatment of relapsing-remitting MS (RRMS), primary progressive MS (PPMS) and Huntington’s disease. The global, Phase III, clinical development program evaluating laquinimod in MS includes two completed pivotal studies, ALLEGRO and BRAVO (both 0.6mg/day). A third Phase III trial, CONCERTO, is currently ongoing and evaluating two doses of laquinimod (0.6mg and 1.2mg/day) in approximately 2,100 patients for up to 24 months. The primary outcome measure is time to three-month confirmed-disability progression as measured by the Expanded Disability Status Scale (EDSS).


Anti-Lingo-1 completed the Phase 2 RENEW study in acute optic neuritis (AON) on April 14, 2015. It's the first clinical study to demonstrate remyelination (the formation of new myelin on axons) following an inflammatory injury in humans.

The new data demonstrates a statistically significant improvement in recovery of optic nerve conduction latency (time for a signal to travel from the retina to the brain’s visual cortex), as measured by the primary endpoint full-field visual evoked potential (FF-VEP), among anti-LINGO-1-treated participants at the last study visit (week 32), as compared to placebo. Results from a sub study of multifocal VEP (mfVEP) are consistent with the FF-VEP findings. The data supplement the top-line, 24-week RENEW results reported by Biogen in January 2015.

RENEW was a randomized, double-blind, placebo controlled, Phase 2 study to evaluate the effect of anti-LINGO-1 in patients treated following a first episode of AON. The study, which was the first to combine functional, structural and clinical efficacy endpoints in AON, enrolled 82 patients across 33 sites in Europe, Canada and Australia. Patients received a total of 6 intravenous infusions of 100 mg/kg anti-LINGO-1 or placebo every four weeks.

A separate Phase 2 dose-range finding, double-blind, placebo-controlled randomized 22 month study (SYNERGY) investigating anti-LINGO-1 in people with relapsing forms of MS (both RRMS and SPMS) is ongoing. SYNERGY is fully enrolled, and results are anticipated in 2016.

"RENEW is the first study to show repair of the human central nervous system (CNS) through remyelination, and the results support our ongoing development of this molecule,” said Alfred Sandrock, M.D., Ph.D., group senior vice president and chief medical officer at Biogen. “We believe the anti-LINGO-1 data point toward a potential new approach to treating demyelinating diseases, and we look forward to the ongoing Phase 2 SYNERGY study results to further clarify the potential of this investigational therapy in MS."


Zenapax (daclizumab) as of August of 2013 is in Phase 3 MS trials of the "Choice" trial. Daclizumab is an interleukin 2 receptor chain specific humanized monoclonal antibody that has shown promising therapeutic effects in MS. It's basically an antibody infusion mainly used for kidney transplant patients to prevent rejection of the new organ.


There are a few other drugs getting ready to enter Phase 1 trials at this time. One can only hope that all of this will come up more and better alternatives for treatment of MS.

Another study is that of a vaccine, with the hope of stopping any further exacerbations. The biggest fear of any illness is the uncertainty of what is next and where one is in the future. Anything that could stop MS where it currently is would be greatly welcomed by most everyone.

Tomorrow also gives us hope because every drug that is developed brings us one step closer to that point finally reaching the end of MS. When that point comes, it could have a tremendous impact on all other diseases. Just look at where past medications had us 5 years ago, and then 10 years ago, and 50 years ago. What medications have done for the world is amazing if you can stand back and look at the big picture. It's possible to make the same progress over the next 5, 10, or 50 years ahead.
Clinical Trials & FDA Rejections
The FDA rejected the new MS drug Lemtrada at the end of 2013, saying the drugmaker didn’t show the drug's benefits outweigh some serious risks. The agency asked the maker, Genzyme, to test the drug in more clinical trials. In two clinical trials, Lemtrada reduced relapse rates over 2 years by about half compared to patients on another MS drug, Rebif (interferon beta-1a).

Genzyme said in a news release it plans to appeal the FDA’s decision on Lemtrada (alemtuzumab). The drug has been approved for MS in Canada, the European Union, and Australia. In the U.S. it received a mixed review from an FDA advisory committee in November. At that time, several members raised concerns about its safety. One potential side effect is called idiopathic or immune thrombocytopenic purpura, or ITP. This causes low platelet counts in the blood and can lead to dangerous bleeding. In the clinical trials, one patient died of ITP. Genzyme put a special patient safety monitoring program in place after the death.

The FDA had earlier approved the drug to treat leukemia, marketed as Campath.

Lemtrada is given as an infusion. It was expected to be used in MS patients who did not respond to other treatments.
Clinical Trials
ClinicalTrials.gov provides patients, family members, and members of the public easy and free access to information on clinical studies for a wide range of diseases and conditions.

ClinicalTrials.gov offers up-to-date information for locating federally and privately supported clinical trials for a wide range of diseases and conditions. A clinical trial (also clinical research) is a research study in human volunteers to answer specific health questions. Interventional trials determine whether experimental treatments or new ways of using known therapies are safe and effective under controlled environments. Observational trials address health issues in large groups of people or populations in natural settings.

The following are links relating to more specific MS trials: