Medical research and new medications dominated the top multiple sclerosis (MS) news stories of 2016. From the 21st Century Cures Act to new drug approvals and increased research funding, people living with MS are encouraged that answers to some of the most vexing disease questions are right around the corner and treatment options continue to expand.
People with MS are also at the center of research with projects that take patient engagement to a new level. For example, iConquerMS, a patient-governed initiative of the Accelerated Cure Project for Multiple Sclerosis, launched the REAL MS (Research Engagement About Life with Multiple Sclerosis) longitudinal study, which intends to answer important questions about the diversity of the experiences of people living with MS and to identify ways to personalize clinical care by identifying factors that affect progression and treatment outcomes. Members of the iConquerMS community can participate by answering online questionnaires about their disease experience and by providing biosamples for molecular analysis. Members can also suggest topics for future research projects.
21st Century Cures Act becomes law
With significant majority vote, Congress passed the bipartisan 21st Century Cures Act, which was signed into law by President Barack Obama on December 13, 2016. The law will enable medical breakthroughs to address the health challenges faced by Americans. It includes several priorities that MS activists had been working toward during multiple sessions of Congress.
Examples from the bill include:
• Establishment of a data collection system to track the incidence and prevalence of neurological conditions, otherwise known as the Neuro Data bill;
• $4.8 billion in new funding for the National Institutes of Health and $500 million for the Food and Drug Administration (FDA) to bring new treatments to patients;
• A six-month extension protecting access to power complex rehabilitation technology (CRT) wheelchair accessories, which are fundamental components of the chair, such as tilt-and-recline systems and specialized seat cushions.
“I applaud Congress for creating a pathway for promising innovation through the establishment of a data collection system for neurological diseases; providing new funding for the NIH and the FDA; protecting access to CRT wheelchair accessories and more. This groundbreaking legislation truly brings us one step closer to ending MS,” said Cyndi Zagieboylo, president and CEO of the National MS Society, in a press release from the society.
PCORI awards $19.6M to fund MS research
The Patient-Centered Outcomes Research Institute (PCORI) Board of Governors approved $19.6 millionto fund four comparative clinical effectiveness studies that will assess several therapies used to treat multiple sclerosis or its symptoms. Two studies will compare the benefits and harms of disease-modifying therapies (DMTs) that aim to reduce MS attacks or slow the disease’s progression. Another will evaluate the effectiveness of medications used to treat MS fatigue. The fourth will assess whether people with MS in rural and low-income areas receive similar benefits from rehabilitation therapies if they are provided via telehealth versus in a clinic.
The four projects approved include:
• An $8.5 million study comparing the effectiveness of rituximab (Rituxan) to other commonly used DMTs in individuals with relapsing MS. The study will be based at Karolinska Institute in Sweden and conducted in collaboration with Kaiser Permanente Southern California. In particular, it will assess the comparative safety outcomes of the medications.
• A $5.8 million study exploring whether patients get as much benefit from an exercise-based rehabilitation program delivered via internet or telephone compared to when the therapy is provided in a clinic setting. Results from this study may improve treatment options for patients living in rural or low-income areas where resources are often scarce. The trial will be conducted in Alabama and Mississippi, led by a research team based at the University of Alabama at Birmingham.
• A $1.9 million study comparing the effectiveness of three medications — amantadine, modafinil, and methylphenidate — commonly used to relieve fatigue in people with MS. The trial will be led by a team based at the University of California at San Francisco.
• A $3.3 million award for the first pragmatic trial comparing the benefits and harms of the fingolimod (Gilenya) and dimethyl-fumarate (Tecfidera) — two oral DMTs commonly used to treat MS that are believed to be similarly effective, but with different side effect profiles. Led by a research team from the Foundation of the Carlo Besta Neurological Institute in Milano, Italy, the trial will be conducted at sites in the United States, Europe, and Israel.
PCORI has opened another funding announcement for Treatment of MS and will be awarding up to an addition $30 million to support MS research over the next five years.
New disease-modifying therapies for MS
Zinbryta: In May 2016, the FDA approved Zinbryta (daclizumab high-yield process, or DAC-HYP), the 14th disease-modifying therapy for people diagnosed with relapsing MS in the U.S. It was also approved in July 2016 for use in Europe. Daclizumab is a humanized IgG1 monoclonal antibody that binds to the αlpha subunit (CD25) of IL-2R expressed on activated T lymphocytes. Due to potential risk of serious liver problems (including autoimmune-related liver problems) and other immune system problems, the FDA suggests that use of Zinbryta should generally be reserved for patients who have had an inadequate response to two or more drugs indicated for the treatment of MS. Zinbryta is given once monthly by subcutaneous injection.
Ocrelizumab: The FDA granted ”breakthrough therapy designation” and priority review to Ocrevus (ocrelizumab) for the treatment of primary progressive MS (PPMS). As Ocrevus was shown to be effective in both relapsing MS and PPMS, Genentech/Roche is seeking FDA approval for both indications. The FDA is scheduled to deliver a decision by March 28, 2017. Although not yet approved, Ocrevus is due to be a game-changer as the first drug approved to treat PPMS. Ocrelizumab is a humanized monoclonal antibody that binds to a molecule (CD20) on mature B-cell lymphocytes. It works in the same way as rituximab to deplete CD20+ B-cells. Once approved, Ocrevus will be the first official DMT approved for use in primary progressive MS. Ocrevus is given once every six months by intravenous infusion.
Additional therapies for progressive MS are in the pipeline, including siponimod, for which positive trial results were announced at the 32nd Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS), and ibudilast, which was fast-tracked by the FDA. Results from the Phase 2 clinical trial of the neuroreparative therapy anti-LINGO (opicinumab) were disappointing; however, Biogen plans to continue development of the monoclonal antibody therapy.
Progressive MS research focus at ACTRIMS 2016
The meeting of the Americas Committee for Treatment and Research in MS (ACTRIMS) focused on progressive MS: what is currently known about the cause of MS, and new research directions aimed at finding solutions for people living with progressive forms of the disease. Highlights of the meeting included therapies aimed at stopping MS; stem cells and MS; interest in diet and MS, exercise and cognition, and mindfulness; inflammation and progressive MS; and nervous system repair. Access to presentation summaries (abstracts), slide presentations, and posters is available on the ACTRIMS 2016 Website.
NMSS awards $1 million to fund dietary trial to treat fatigue in MS
The National MS Society (NMSS) awarded over $1 million to support a clinical trial at the University of Iowa led by Terry Wahls, M.D., to compare the effect of two popular diets — the Wahls Protocol and the Swank Diet — on MS-related fatigue. This trial is one of more than 380 new and ongoing studies from around the world that the NMSS has invested $50 million in funding in 2016 alone.
• The Wahls Protocol features a modified Paleolithic diet that excludes grains, eggs, dairy products, legumes, and nightshade vegetables, but instead focuses primarily on consumption of vegetables, fruit, meat, and fish. It was created by Dr. Wahls, who was diagnosed with MS.
• The Swank diet is a low-fat diet created in 1950 by Roy Swank, M.D., Ph.D. It limits consumption of processed foods, saturated and unsaturated fats, and red meat. The diet focuses on fruits and vegetables, whole grain cereal and pasta, white fish and shellfish, and skinned trimmed poultry meat.
Researchers are recruiting 100 people with relapsing MS, who experience fatigue and who live within a 500-mile radius of Iowa City, Iowa, to enroll in the 36-week clinical trial. Participants will follow their usual diet for 12 weeks and then be randomly assigned to follow a low saturated fat diet or a modified paleolithic diet for 24 weeks. Interested individuals may complete screening questionnaires (use code: JMJPYEJHP), email MSDietStudy@healthcare.uiowa.edu, or call 319-384-5053.