Biogen Idec Testing Regenerative Medicine Drug to Reverse the Path of Multiple Sclerosis

8/27/08Follow @xconomy

Biogen Idec has made a lot of its money on Avonex and Tysabri, drugs that slow down the rate of flare-ups for people with multiple sclerosis. Now the Cambridge, MA-based biotech company (NASDAQ: BIIB) is pursuing a loftier goal. It is working on the first experimental drug that may reverse the symptoms of the neurodegenerative disease.

The drug, being tested in animals and prepped for its first human trial, is designed to block a protein called Lingo-1 that interferes with body’s production of myelin, the fatty protective coating around nerve fibers. People with multiple sclerosis have an overactive immune system that eats away at the myelin layer, and they have no ability to regenerate myelin in the brain or spinal cord, says Sha Mi, a Biogen researcher. That means nerve impulses that control speech, vision, and movement get short-circuited, sort of like when an electrical wire is stripped of its insulation. Biogen thinks it now has engineered a drug that can stop Lingo-1 from doing its dirty work, allowing the body to regenerate myelin coating around nerves. That could restore normal functions, like walking.

“People around the company are very excited about this,” says Kenneth Rhodes, Biogen’s vice president of discovery neurobiology. “It’s potentially a transformational therapy.” Sha Mi, the Biogen scientist who discovered the molecular switch that paved the way for the program, put it this way, “As a scientist, I came all the way from China. If we can create a new medicine to affect patients, that is my dream.”

The drug hasn’t even entered clinical trials yet, and it’s already been an eight-year odyssey. Sha Mi (who goes by the name Misha) joined the company in 2000 from Wyeth’s Genetics Institute unit in Cambridge, MA. Not long after joining Biogen, she found the Lingo-1 protein in a database and learned it was expressed solely in the central nervous system and, then, only in neurons. Later experiments showed that when scientists delete the gene that makes Lingo-1 in mice, those altered mice would recover from a disease in which the immune system eats away at myelin, called autoimmune encephalomyelitis. The same recovery was seen in mice when they were given an antibody drug designed to block the Lingo-1 protein. There were no side effects or dangers seen from producing too much myelin, because the body will only produce the amount needed to cover nerves, Sha Mi says. The combination of experiments, conducted by Biogen scientists and collaborators in China, made the cover of Nature Medicine last October.

Other researchers are working on myelin repair, such as a group led by Bruce Trapp at the Cleveland Clinic, says Rhodes, the Biogen vice president. Madison, NJ-based Wyeth (NYSE: WYE) has attempted to develop conventional small molecule drugs against Lingo, but hasn’t been successful, he says.

Biogen is developing a genetically engineered antibody against Lingo because that approach should do a better job of binding with the Lingo protein target on the surface of cells, Rhodes says. The first version, however, wasn’t quite “optimal,” and a newer one is being engineered with better properties, he says. The latest version is made with fully human DNA, instead of partial mouse DNA, because researchers want to be confident that the drug won’t spark the immune system to reject it, especially if it needs to be given chronically. The company is planning to ask the FDA for permission to start its first human clinical trials, although he wouldn’t say when.

No details are available yet on how the trials will be crafted, but Rhodes made clear that the company’s vision is for Lingo to be used in combination with Avonex or Tysabri. The idea is that those drugs can reduce the immune system’s assault on neurons, quieting the storm. That would give an opportunity for the anti-Lingo-1 drug to step in and regenerate myelin around the nerves.

Since 400,000 people in the U.S. suffer from MS, and there’s nothing else quite like this program poised for clinical trials, it seems unlikely that Biogen will have much trouble recruiting patients in the first study. If they show they can regenerate myelin in even a few people, Biogen will be a few steps closer to fulfilling Sha Mi’s dream.

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  • Dianne G.

    I hope this works as well as it sounds.
    Regards, Dianne-NY

  • Betty L.

    PLEASE try to move this trial as quickly as possible! There are so many of us who would sign up today since our MS is progressing very quickly.

    Thank you so much for working on this very difficult illness.

  • Pam L.

    DITTO to what the above posters wrote. I too would sign up in a heartbeat!
    Thanks so much.

  • Stephen P. Stanko

    I have primary progressive multiple sclerosis. DX 11 years ago and progressing rapidly now. I would sing up in a minute…put not OPEN LABEL only.

  • AJ L

    Oh my God, just show me where to sign, I have gotten to the point where I am looking at a stem cell transplant.

  • Charles Kuhn

    Sign me up. I have had M.S. for 14 years. It has claimed my legs. I am luckier than most, but wait for the day to walk again. Running and dancing wouldn’t be bad either!

  • Trine Skogsholm

    This was exactly the light in the narrow tunnel we needed. Pls sign me up.

  • GLEN WILSON

    I HAVE HAD DR. WADEN EMERY IN FT LAUDERDALE FLA (954)771-8300. FOR OVER 9 YEARS HE BEEN THE BEST DOCTOR. MY FIRST DAY I WAS IN HIS OFFICE I WAS IN A WHEELCHAIR. I HAVE BEEN ON Tysabri FOR 10 MONTHS. WHEN I FIRST SEEN HIM THERE WAS NOT MUCH HOPE I WOULD EVER WALK ANYMORE BUT HE TRY EVERYTHING POSSIBLE AND MANY MEDICATIONS TODAY I CAN RIDE A MOTORCYCLE.I STILL HAVE BAD DAYS.BUT THANKS TO HIM I HAVE MANY GREAT DAYS. PS SIGN ME UP

  • http://embarq MARIA POLICASTRO

    I have nad M S for over 50 years this is great news too bad my husband didn’t live to see this I wish you God speed.

  • Karen H

    Of curse, I’d fight my way to the front of the line – have been dealing with the MonSter for about 30 years but wasn’t officially diagnosed til Nov ’95 about my 50th birthday.

  • Wayne Stillerman

    Where do I sign up? My bags are packed and I’m ready to be part of the human clinical trials. I have been contemplating going overseas for a stem cell transplant so maybe I’ll hold off a little to see what comes of this. This is great news!!!!!

  • Joan Brock

    How wonderful, but what if we are not on any other Biogen drugs? ARE WE ELIMINATED???

  • Lynn Williams

    I think most anyone who has MS and has had one or two relapses would be willing to be part of the trial. I have been dxed for 8 years now and the last 2 years have really headed down hill. I had one infusioin of Tysabri right before it was pulled off the market but am currently going thru their approval process to get back on it. I was on IVIG and had great results with it but my doctor feels that I may have better results with Tysabri. I just want my life back!

  • Edward Hom

    This is the trial every MS patient and family member/friend has been waiting for. I’m sure my sister would be willing to sign up. ONE PRAYER – PLEASE MAKE THE TRIAL OPEN TO ALL MS PATIENTS, NOT JUST THE RELAPSING-REMITTING ONES. The PPMS – SPMS patients are literally at death’s door.

    God, I hope this works. Phase I …..

  • Troy Cummings

    Sign me up I cant Take this MS Thing anymore and the tysabri was doing good for me 14th time i had to try solumedrol to help me out. They need to hurry up on this but its the usa it will be about 10years before they approve it..lol

  • Irely

    Just remember me.
    I’ll been living with R-R MS for 37 years (I’m 49), still able to move and walk, but going down the drain too fast. I’m using Avonex, which have been great for 8 years since my diagnosis. Can we be included in this tryal?
    PLEASE! GIVE ME A CHANCE!

  • Stephen P. Stanko

    What is the hold-up?? Why does everything regarding MS take so long to get moving??

    Who do we have to contact? Name,number,and address!

    MS does not wait!!

  • RAÚL BRAVO

    Estimados Señores

    Mi nombre es Raúl Bravo, tengo 41 años y sufro de Paraparesia Espástica, instancia por a que me ofrezco para experimentar con el nuevo medicamento propuesto. Lo anterioir porque tengo dos niños pequeños que necesito aun cuidar, por lo menmos hasta que se valgan más por si mismos.

    Raúl Bravo Chile

  • Danny Rexrode

    I would like to be a participant in this trial. I have no mobility in both of my legs and am in a wheelchair full time. I also suffer cognitive problems. I am currently using Tysabri and it is helping me from getting any worse, but, I really want to participate in this new trial. Please consider me for this trial.

  • Stephen P. Stanko

    I will ask again WHAT IS THE HOLD UP??
    I have seen the same message before,and no word of progress!
    I thought GWB lost he election.
    Time is not on anyone with MS’s side. Light a candle under someones A..

  • Jenni

    This is by far some of the best news I have ever heard. I am going to be 40 in October and have been living with this (what I call) a total nuisance and pain in the behind disease. Just like alot have said ” Why does it take so long to approve a medicine that is going to turn around the lives of many people.} There should be a cure anyway!!!! Hoping that by the time I am 45 hopefully sooner there will be a cure. In 3-5 yrs they are coming out with the cure for diabetes, Lupus and Chrone’s disease. All same types of this MS. It should be on that list. Don’t ya think…………..

  • john dery

    i,like many ,have tried everything to keep my ms in check ,but what i would’nt give for the old carefree romps down montain trails

  • Kathy Olson

    Today’s date is 6/27/2011

    JUST found this site. My last visit to my Dr; he mentioned “anti-lingo” is a possible new drug coming, that could really help me. So, I googled it tonight and found this post.
    ANYTHING on the internet to tell me “what’s the latest” regarding anti-lingo ?
    I sure want to take this or at least be apart of a trial group. Have had PP MS for 20 years so have LOTS of needed remyelination !!!!!!!!!