There is finally some progress in the hunt for a treatment for the most difficult form of multiple sclerosis — the form that has stubbornly resisted every attempt to find a therapy.
The catch, experts say, is that people with this form of the disease should keep their expectations low, because even the new drug that’s now in the last stages of development doesn’t appear to have a big clinical impact.
The reason that the new drug is generating so much interest, though, is that it’s the first time a therapy for primary progressive MS has had any impact at all.
And it’s the first MS drug to ever receive a “breakthrough” designation from the Food and Drug Administration, which puts the experimental medicine on a fast track to get through the review process as quickly as possible. The agency granted the designation earlier this week.
Primary progressive MS is a relatively rare form of the neurological disease in which a person doesn’t have severe attacks, the way people do with the more common, relapsing-remitting kind. Instead, they suffer a slow, steady physical deterioration with no hope of recovery.
The FDA has approved 13 drugs for treating relapsing-remitting MS, but none have been shown to work for people with primary progressive disease. The new “breakthrough” drug, called ocrelizumab, is one of six that are in various stages of development for this recalcitrant form, one that affects 10 to 15 percent of the estimated 400,000 Americans living with MS.
On Friday, the company behind ocrelizumab, the Roche subsidiary Genentech, will release new Phase 3 trial data at a conference of the Americas Committee for Treatment and Research in Multiple Sclerosis, which is being held in New Orleans.
Genentech has already published a first round of the results, which showed some impact on a wide range of measures for people with primary progressive MS. The new data will include more detail on how ocrelizumab affected subgroups of patients, as well as new information on how it worked in people with relapsing-remitting MS, to show whether it can also work on the more common variety of the disease.
These new results will demonstrate that the drug “appears to have efficacy across the patient population” of people with primary progressive MS, including people who had active inflammation and those who didn’t, according to Dr. Peter Chin, principal medical director for neuroscience at Genentech. It will also show a “significant reduction” in the major markers of the disease for people with relapsing-remitting MS, he said.
Even before those data are released, the fact that the FDA has given the coveted “breakthrough” designation to the drug has given new hope to people who have been waiting for a treatment for years.
“When I found out about this this morning, it was like every cell in my body smiled,” said Gigi Ranno, an outreach coordinator for the Massachusetts Department of Conservation and Recreation, who has primary progressive MS.
“I’ve had MS for over 25 years. It’s the first time that I have heard that there’s a drug that’s this close to being submitted to the FDA for approval for my kind of MS,” she said in an interview Thursday.
The FDA announcement has also generated excitement among advocates like Tim Coetzee, the chief advocacy, services, and research officer at the National Multiple Sclerosis Society. “We’ve had so many failures with treatments for people with primary progressive MS that this really represents a source of hope,” he said.
Experts do warn, however, that even if the drug is approved after the fast-track review, people with primary progressive MS shouldn’t expect spectacular results.
“It is truly a landmark, and really provides hope to people who are living with primary progressive MS. That being said, the results are on the modest side,” said Dr. Tanuja Chitnis, a neurologist at the Ann Romney Center for Neurologic Diseases at Brigham and Women’s Hospital in Boston, who was not involved in the drug trials.
Ocrelizumab works by targeting a certain kind of immune cell that can contribute to the damage to the nervous system, including the nerve cells and the protective covering around nerve fibers. It is taken by intravenous infusion twice a year.
In the clinical trial data released last fall, which covered 732 people with primary progressive MS, people who took the drug had a 24 percent reduction in the progression of disability after 12 weeks compared to those who received a placebo. Study participants on the experimental medicine also experienced slower declines in walking ability and brain volume than people in the control group.
These differences, while undramatic, were statistically significant, which is the main threshold the FDA will be looking for when it decides whether to approve the medication. “It’s a positive result when you’ve had 20 years of failure,” said Coetzee.
Based on the study results, Dr. Dennis Bourdette, executive director of the Oregon Health and Science University’s Multiple Sclerosis Center, said it was “very appropriate” for the FDA to grant breakthrough status to the drug and that he was “hopeful that it will be approved for the treatment of primary progressive MS.”
But Bourdette, who was not involved in the Genentech-funded trials, also noted that the patients in the study were younger than average for people with primary progressive MS, which usually begins about 10 years later than relapsing-remitting MS. Younger patients could be more responsive to treatment, and “it will be uncertain how effective ocrelizumab will be for older and more disabled patients,” he said.
For Michael Bunting, 66, it’s more of a sign of distant hope than something that could give him immediate relief.
Bunting, who talked to STAT last year about his battles with progressive MS, has a different variation of the disease: secondary progressive MS. That’s a kind that often begins with relapsing-remitting MS and then turns into a steady deterioration. So even if the new drug is approved, it’s not something that would necessarily help him because it’s not yet been evaluated for people with his form of the disease.
Still, Bunting sees it as a big step for all MS patients, and one that could lead to more research that could eventually find a drug that would help him.
“If this thing helps just in the primary progressive, that’s great, because it will help us in the long run [to] finally find something that will help the secondary,” Bunting said.
“I just hope they don’t wait too long.”
My friend has Progressive MS and this could just help her. When she looked into it, they said it would be three treatments in a year the first year at $65,000 per treatment x 3. How many people are even able to afford that? That’s a bit more than I make all year! Please tell me that this soon will be affordable and that the insurance companies will kick in, because it doesn’t look likely.
Mark has severe m.so. He cannot talk and cannot move his body. He is totally bedridden.I doubt if this would help him.He is on a feeding tube and he chokes alot.
I would love to know more about this drug! I was told in 2010 that I had p.p.ms and first I was put on Rebif shots that hurt me more than helping. I switched drs, then was put on gelynia which seem to do nothing! So I stopped taking it. I’m 39 turning 40 this summer. I haven’t lost any use of limbs or seeing yet thank God, but I constantly hurt! I have speractic pains throughout my body and limbs. I can’t hold down a job and still fighting for disability! Which I’ve been denied 3times, and waiting on a court date. They just say I’m not disabled enough and can find work. But by being in constant pain I can’t! And I don’t have any lawyer or help with my case. I’m taking (2) 200mg morphine pills and was taking (3) 30 mg oxycodeine a day till last month when my dr told me he told me he can now only give me (2) oxys now… and I really need that extra one to help! I really need something better then these cause I’ve been on them for so long my body is immune! This next appt I’m going to see if my dr can switch the morphine to something else cause they don’t seem to be helping at all. I truly wish medical marijuana was legal here in VA! I rather have it than take pills every day! But if this drug helps I hopefully won’t need so much pain meds!! I’m sick and tired of being in pain every day, and not being able to provide the life my daughter deserves!! I live off of $464 a month cause I get that from Social services for the death of my daughters mother. I live on a church camp for housing. Which I do have to help around to be able to live here but it hurts me to even do the little bit of work that I do, do! But it puts a roof over my daughters and my head! So I will try Anything that may help!!
I have progressive MS. I am also on medicare. Does my Neorogist prescribe this when available and does medicare cover. As most people I can’t afford 68,000.00
How do I go about getting this drug for my mother who is 67 severely handicapped in a wheelchair from MS? She is in a nursing home and has Medicare /Medicaid
How would people go about getting this treatment?
Just wondering when this new is going to come available, does any one know my spouse and I are running out of time , also is it approved in the usa or eroupe.
My son is 49 years old and has had MS since he was 32.NEVER give up.My son is on a feeding tube and he cannot talk nor move his body but one never knows from one day to the next what will happen. Miracles do happen just keep believing as everything is up to God not us. The MS doctors will provide you with the information about the new pill for MS.
When will this be released ?
i am writing on behave of my wife Dr Dina. she had MS for about 9 years. she take Gilinea for about 3 years. unfortunately she did not improve in spite, she deteriorated and now she has secondary progressive MS. i am asking if this new drug will be of hope for her. Thanks a lot.
Does this new drug help secondary progressive / progressive. My partner is getting so bad that she may not have time for an new procedure in drugs. Please give me an answer so we know where we stand. Time is running out.
Respectfully yours ,
I was diagnosed with PPMS when I was 62. The MRIs showed the problem in my spine. My brain was fine. Anyone else have it in their sound. Hopefully the new med will work for me. I’m deteriorating.
Mine is in the spine. No brain lesions as of 3 yrs ago. Been totally wc bound for last 6. From ’99 till 11 I could still hold weight to transfer or stand long enough for husband to help me dress. Then the lungs started acting up. Just the buttons. Turned into. Cesspools breeding fungi and bacteria. I’m seeing neuro wed. Hopefully he will let me try.
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