Full List
I first created a multiple sclerosis treatment pipeline list in August 2005 and posted it in a forum at the website ThisisMS.com. I’ve been maintaining the list ever since, with lots of help from the thisisms community. For the sake of trying something new, I’ve decided to give up on my long-standing determination to be the last person in the world without a blog — so here’s another blog.
In the separate phase 1, phase 2 and phase 3 posts, I’ve put links to more information about each drug / trial. I do updates whenever I find new information, so the list is always as up to date as I can make it.
MS Disease-modifying Treatment Pipeline
February 7, 2010
Approved
- Avonex (intramuscular interferon beta 1a) (Biogen Idec)
- Betaseron / Extavia (subcutaneous interferon beta 1b) (Bayer / Novartis)
- Copaxone (glatiramir acetate) (Teva)
- Novantrone (mitoxantrone) (Merck Serono / OSI)
- Rebif (new formulation: fetal bovine serum-free/human serum albumin-free) (subcutaneous interferon beta 1a) (Merck Serono)
- Tysabri (natalizumab) (Biogen Idec / Elan)
Treatments used “off label”
- Antibiotics: Doxycycline, Amoxicillin, Rifampin, Metronidazole — OR — Doxycycline, Roxithromycin, Metronidazole — OR — Rifampin, Azithromycin, Flagyl
- Azathioprine (aka Imuran)
- Corticosteroids (especially methylprednisolone)
- Cyclophosphamide (aka Cytoxan)
- Intravenous Immune Globulin (IVIg)
- Low-Dose Naltrexone (LDN)
- Methotrexate
- Mycophenolate mofetil (aka Cellcept)
- Plasma exchange (aka plasmapheresis)
Phase III Trials
- Autologous hematopoietic stem cell transplantation (aka Multiple Sclerosis International Stem Cell Transplant Trial)
- BG12 (Biogen)
- Campath (aka alemtuzumab) (Genzyme)
- Cannabinoids – oral (PPMS) (Clinical Neurology Research Group – Plymouth)
- Daclizumab (aka Zenapax) (also in a phase 2 trial) (Facet Biotech, Biogen-Idec)
- FTY720 (aka fingolimod) (2 trials: RRMS and PPMS) (Novartis)
- Interferon beta 1a – PEGylated (bi-weekly or monthly injection) (Biogen Idec)
- Laquinimod (aka ABR-215062) (Teva)
- Minocycline (MS Society of Canada, MS Scientific Research Foundation)
- MN-166 (aka ibudilast and Ketas) (Medicinova)
- Mycophenolate mofetil (aka Cellcept) (with Avonex)
- Mylinax (oral cladribine) (Merck Serono)
- Progestin and Estradiol (Hospices Civils de Lyon)
- Revimmune (aka high-dose cyclophosphamide or cytoxan) (trial for refractory MS) (Accentia Biopharmaceuticals)
- T-Cell Vaccination (aka TCV-01-002) (Sheba Medical Center)
- Teriflunomide (aka HMR 1726) (Sanofi-Aventis)
- Trimesta (oral) (estriol) (with copaxone) (Adeona Pharmaceuticals)
Phase II Trials
- ACT-128800 (Actelion)
- AIN457 (Novartis)
- Alferon N Injection (Hemispherx Biopharma)
- ATL/TV 1102 (Antisense Therapeutics / Teva)
- ATX-MS1467 (Apitope Technology / Merck Serono)
- Autologous hematopoietic stem cell transplantation (2 trials: ASTIMS and HALT MS)
- Bacille Calmette-Guèrin (BCG) Vaccine
- BAF312 (Novartis)
- BHT-3009-01 (aka DNA vaccine) (Bayhill Therapeutics)
- Chaperonin 10 (aka XToll and Cpn10) (C-Bio)
- chronic cerebrospinal venous insufficiency (CCSVI) treatment (University of Ferrera and Bellaria Hospital, Bologna)
- Doxycycline (with Avonex)
- EGCg (Epigallocatechin-Gallate) (Charite University, Berlin)
- Enkorten (aka FAR404) (Farmacija)
- Erythropoietin (aka EPO) (Institut fuer anwendungsorientierte Forschung und klinische Studien gGmbH)
- ESP (an analogue of a metabolite of azathioprine) (Biomolecular Pharma / Mount Allison University)
- Estroprogestins (with IFN beta 1a) (S. Andrea Hospital)
- Firategrast (aka SB-683699 and T-0047) (oral) (Glaxosmithkline / Tanabe)
- Fluoxetine (aka Prozac) (SP, PP & RRMS) (University Medical Center Groningen)
- Flupirtine (Bayer / Schering)
- GEM-SP (SPMS) (Gemacbio)
- Helminth-induced immunomodulation therapy (HINT) (2 studies: University of Wisconsin, Rigshospitalet, Denmark)
- Hookworm (WIRMS – Worms for Immune Regulation in MS) (UK) (RRMS)
- HP184 (aka nerispirdine) (Sanofi-Aventis)
- Idebenone (National Institute of Neurological Disorders and Stroke)
- Interferon alpha (oral) (University of Texas-Houston)
- Kynurenine (aka Tranilast, Rizoben, 3,4-DAA) (Nuon Therapeutics)
- Lipitor (aka atorvastatin) (Pfizer)
- Low-dose naltrexone (aka LDN)
- LY2127399 (Anti-BAFF Human Antibody) (Eli Lilly)
- LymphoStat-B (belimumab) (aka Benlysta) (Human Genome Sciences, Glaxosmithkline)
- Masitinib (SPMS and PPMS) (aka Kinavet or AB1010) (AB Science)
- Mesenchymal Stem Cells (3 trials: University of Cambridge, Hadassah Medical Organization, Cleveland Clinic)
- Methotrexate – intrathecal
- Minocycline (2 trials 1 with copaxone, 1 with rebif)
- Ocrelizumab (R1594) (Roche/Genentech/Biogen)
- Ofatumumab (HuMax-CD20) (Genmab and Glaxosmithkline)
- Omega-3 fatty acids (University of Washington)
- PI-2301 (Peptimmune)
- Pixantrone (aka BBR 2778 ) (Cell Therapeutics)
- Pleneva (aka BGC20-0134) (BTG)
- RG2077 (aka CTLA4-IgG4m) (Immune Tolerance Network and Repligen)
- Riluzole (with Avonex) (UCSF)
- RPI-78M (Nutra Pharma / ReceptoPharm)
- RTL1000 (Artielle ImmunoTherapeutics)
- Stem cell transplant from donor (allogeneic) (2 studies: University of Louisville, Duke University)
- Testosterone (UCLA)
- Tovaxin (Opexa)
- Treosulfan (SPMS) (University of Wuerzburg, Germany)
- Vitamin D3 (with calcium) (St. Michael’s Hospital – Toronto)
- Zocor (aka simvastatin) (RRMS and SPMS) (Merck Serono)
Phase I Trials
- Amiloride (Oxford University)
- AZD5904 (AstraZeneca)
- BaroFeron (BaroFold)
- BIIB033 (aka Anti-Lingo) (Biogen)
- CCX915 (CCR2 antagonist) (ChemoCentryx)
- CS-0777 (Daiichi-Sankyo)
- Extracorporeal photochemotherapy (University of Milano-Bicocca)
- Far Infrared Irradiation (GAAD Medical Research Institute, Canada)
- GRC 4039 (Glenmark Pharmaceuticals)
- INCB8696 (Incyte)
- Linoleic Acid (omega-6) (University of Rochester)
- Lipoic acid (Oregon Health and Science University)
- Low Fat Diet (Oregon Health and Science University)
- Mesenchymal stem cell transplantation (autologous) (Cleveland Clinic)
- MM-093 (Recombinant Human Alpha-Fetoprotein) (Merrimack)
- NI-0401 (NovImmune)
- ONO-4641 (oral S1P receptor agonist) (Ono Pharmaceutical)
- Prolactin (Stem Cell Therapeutics / U of Calgary)
- TK54 (LTKfarma)
- Valomaciclovir (EPB-348) (Epiphany Biosciences)
Pre-clinical
- AEG35156 (XIAP antagonist) (Aegera)
- Aequorin (Quincy Bioscience)
- Arvanil (synthetic capsaicin-anandamide hybrid)
- AT-008 (Androclus)
- AZD 8797 (AstraZeneca)
- Bowmann-Birk Inhibitor Concentrate (BBIC) (Jefferson Medical College)
- Calpeptin (calpain-specific inhibitor) (Medical U of S Carolina)
- Cathepsin S (Medivir)
- CD3 antibodies (Harvard)
- CD83 (Argos Therapeutics)
- CEP-701 (Johns Hopkins)
- CF101 (Can Fite BioPharma)
- CF402 (Can Fite BioPharma)
- CGEN-54 (Compugen)
- CHR-1103 (Chromos)
- Ciliary neurotrophic factor (CNTF)
- CRA-028129 (cathepsin S inhibitor) (Schering)
- CT301 (University of Western Ontario)
- CYLA (calpain inhibitor) (SUNY Downstate Medical Center)
- Dilantin (phenytoin) (Yale University)
- DP IV and APN inhibition (Otto-von-Guericke University, Germany)
- DZ2002 (Shanghai Institute of Materia Medica)
- Epratuzumab (IMMU-103) (Immunomedics)
- ESPRIT (Exon Skipping Pre-RNA Interference) (AVI BioPharma)
- Estrogen receptor alpha (ERalpha) ligand (UCLA)
- Fasudil (Kyushu University)
- Gemfibrozil (aka Lopid) (Rush University)
- GEMS-001 (Gemacbio)
- Glial Growth Factor 2 (aka GGF2) (Acorda)
- Glucosamine (Thomas Jefferson University)
- hBCMA-Fc (La Trobe University – Australia)
- HE3204 (Hollis-Eden)
- IL-1ra gene therapy (San Raffaele Scientific Institute, Milan)
- Interferon-beta – electroporation (EP)-mediated intramuscular IFN-beta gene transfer (Cleveland Clinic)
- LF 15-0195 (Universite Paul Sabatier, France)
- LJP 1207 (La Jolla Pharmaceuticals)
- LLDT-8 (Chinese Academy of Sciences)
- LX2931 (Lexicon Genetics)
- MCT-175 (MultiCell)
- Mikbeta1 (National Institutes of Health)
- MTA (methylthioadenosine) (University of Navarra – Spain)
- MT203 (human anti-GM-CSF IgG1 antibody) (Micromet AG)
- NAPVSIPQ (NAP) (Allon)
- Neurodur (Ceptor)
- Neutrazumab (C5a Receptor antagonist) (G2 Therapies)
- Nicotinamide (a form of vitamin B3) (Children’s Hospital – Boston)
- ONO-2506 (arundic acid) (Takizawa et al)
- OPN-101 (Opsona)
- Oral Copaxone (Teva)
- Peptidomimetics of apoE protein (apolipoprotein), COG133 (Duke University)
- PLP-BPI (University of Kansas)
- PXS25 / PXS64 (Pharmaxis)
- PXS2076 (Pharmaxis)
- Q-Cells (Q Therapeutics)
- QR-442 (Quigley)
- R348 (Rigel)
- RheoSwitch Therapeutic System + TriGrid Delivery System (RheoGene, Ichor Medical Systems)
- ROB 895 (Oxypharma)
- ShK(L5) (UC Irvine)
- STA-5326 (Synta Pharmaceuticals)
- sTNFR:Fc/p80 (Medical University of Lodz, Poland)
- Symadex (imidazoacridinone) (Xanthus)
- Syntonix Transceptor/SynFusion (inhaled interferon-beta:Fc) (Syntonix/Serono)
- Tetracycline (non-antibacterial) (Paratek)
- Timcodar (Recovexx)
- Vasoactive Intestinal Peptide (aka VIP)
- Vitamin B12 (Hospital for Sick Children – Toronto)
- VX15 (Vaccinex and Teva)
- 13-cis-retinoic acid (Kinemed)
- 15d-PGJ(2) (University of Arkansas)
- 5-aminoimidazole-4-carboxamide-1-beta-4-ribofuranoside (AICAR) (Medical University of South Carolina)
Pre-clinical substances
Scoobyjude said
Dignan, just wanted to say that when I was first dxed and was as low as I could get, your pipeline brought me out of it. It made me feel like there was really hope. For that I can’t thank you enough. Keep up the good work.
Lee said
I will read anything you write, Dignan…I’m in awe.
superman said
It sounds like some people are working for our recovery
We ought to them for believing in their coming final success.
anon said
You sir, have given me incredible hope that this is no longer an orphan disease. Thanks you so much for tracking this information, and keep up the good work!!!
Maz said
Anyone has any news on Rituxan for PPMS? Is it a good sign that they’re still running the clinical trial?
Geoff said
Maz, it’s my understanding that the Rituxan for PPMS trial is due to finish in November. The companies expect to report results from the trial some time in the first half of 2008. I’m not entirely sure if that will mean Rituxan would be approved for use in PPMS, or whether another phase 3 trial might be required.
Daniel Drumm said
Your website is very well produced. Good work! I have RRMS.
Rita Masse said
I am absolutely fascinated by how much is in various stages of research. Can you tell me, or do you know how I could find out what exactly is meant by each phase of research? How close or how far are we? Thanks much. I am a person with MS. Rita Masse
100 Merrimack Ave. Unit 63
Dracut, MA 01826
(978) 454-3731
ritaamasse@aol.com
Geoff said
Rita, good question. Here are a couple of good places for info on clinical trial phases:
Wikipedia: http://en.wikipedia.org/wiki/Clinical_trial
Multiple Sclerosis International Federation: http://www.msif.org/en/research/clinical_trials/ct_phases.html
How close are we? If the results of the rituxan PPMS trial are good, they might apply for approval later in 2008. The first of the oral drugs in the final phase (phase 3) before approval could be approved and available in 2009. I can’t predict what drugs will be approved, but I’m pretty sure that starting in 2009, there will be at least one new drug approved to treat MS every single year until they find a cure. The current stage of MS research (that started with the approval of betaseron in the early 1990s) where we wait for years while the next candidate works it way through the trial process is going to end some time in the next 18 months.
Susie said
What’s the news (if any) on Fampridine-SR? I want some!!!
ddietrich said
The FDA is supposed to respond by April to Acorda’s submission for approval for Fampridine SR by October 2009. Another drug with a similar mechanism of action is in clinical trials for MS presently. Look at nerispirdine, under identifier NCT00811902 at clinicaltrials.gov.
Geoff said
ddietrich, thanks for the feedback. I really hope Fampridine and nerispirdine can be of use to people with MS.
I decided not to track the symptomatic treatments in the pipeline. Tracking all of the symptomatic treatments that could be used for MS would be a big job in itself and I decided not to take it on.
Geoff said
Susie,
I haven’t really followed Fampridine very closely as I’m fixated on the disease-modifying treatments and not so much on the symptomatic treatments. It looks to me like Acorda expects to be finished their current phase 3 trial in 2008, but there are still several issues to clear up. See here (starting on p. 41):
http://sec.edgar-online.com/2008/03/14/0001047469-08-002781/Section7.asp
Sally Adams said
I have been taking 17.5 mg of 4-AP (3 times daily) for 12 years. I have it compounded at Medicine Shoppe. It is one of 4 drugs that I cannot live without. Even though I am in a wheelchair, I know I would be unable to care for myself without this drug. I’ll be glad when it is approved, so my insurance company will cover it.
Meander said
Wow…this is some list. I will definitely have to come and read all this thoroughly. Thanks for putting out this information.
http://www.healthcentral.com/multiple-sclerosis/c/73302/profile
Karen said
Do you think that there will be a cure for this terrible disease one day? I try to be hopeful, but at 36, wonder is it too late to reap the benefits of any of these therapies?
Geoff said
Karen,
I do think there will be a cure, and I don’t think it is too late for you to reap the benefits of the therapies in the pipeline.
With all the drugs being investigated, and all the research on MS that is under way, and all the research into the immune system and the central nervous system that isn’t specific to MS, and all the research into genetics that isn’t specific to MS, and all the research into stem cells that isn’t specific to MS, and all the new imaging and testing technologies being developed, the scientific community will figure out MS. I obviously don’t know when, but I fully expect a cure in the next 20 years. I think people, even researchers in a specific area, often underestimate the impact that technological progress can have on our understanding.
Geoff
Karen said
Thanks Geoff. I had a Dr. tell me recently that I would be in a wheelchair by Christmas and there was nothing else they could do for me. I appreciate your encouraging view. K.
Geoff said
Karen,
I’m sorry to read what your doctor had to say. Have you ever checked out the ThisIsMS forums? I know there are a lot of MS patient forums online, but there are a lot of resourceful people who post there and who try all kinds of treatments. Maybe nothing miraculous, but if you want to leave no stone unturned…
http://www.thisisms.com/forums.html
fran said
Have you heard anything of the work done at Henry Ford/Mount Alison? They have done a trial on 7 people (is that a phase I ? ), sounded incredibly promising.
All I can find is the press release. Most frustrating!
Geoff said
Fran, I had seen the press release too, but didn’t find anything else, except this medical hypothesis article: http://www.biomolecularpharma.com/MSMV_Paper.pdf
Since they are talking about the same thing (the MS associated measles virus), I assume the drug they discuss in the article is the same one the press release refers to: ESP, an analogue of a metabolite of azathioprine. This is also interesting because azathioprine has been used off-label as an MS treatment at least since the 1970s.
I guess based on this news I will add the drug to the list in phase 2.
Geoff
carlos hansen said
Your site is very nice, very informative, it’s hopeful – I likey!
Your last update was Jan 4, how often do you update? will we have a cure in our life time?
Geoff said
Carlos, thanks for the comment. I update the site any time I learn of new developments. Sometimes it’s updated multiple times in a week, sometimes not so much.
Regarding the cure, obviously I’m just making a wild guess here, but I think we will have one within 20 years. Saying “have a cure” could mean a lot of things — e.g. discover the cause and feel confident we know what it will take to cure MS, or have a treatment that cures everybody in a phase 2 trial, so we think it’s a cure, but it’s not approved yet, or have a cure that is actually widely available to PwMS. When I say within 20 years, I’m hoping we will have the cure widely available within 20 years. Considering the clinical development of drugs is averaging around 8 years (varies widely though), that would mean I’m saying we’ll figure out MS within 10 years or so. I’m probably an optimist, but I think all the tools (imaging technology, computer simulations, large scale projects to define the proteome, connectome etc etc) are improving at such a rapid pace that we’re going to learn a surprising amount in the next 10 years. It won’t be like the last 10 years, we will acquire knowledge at a vastly great rate, and will have a better idea of how it all fits together.
But keep in mind, I’m not a researcher, so it’s all just wild speculation.
Geoff
Simon said
Hi
I do work in research, in cancer research at a large pharma.
My wife has MS, but unfortunately I think its unrealistic to think of a “cure” in our lifetime. I’m 36.
However, I do think that over the next 30-40 years we will see that MS becomes increasingly well managed both in terms of reducing disease severity in RRMS and in slowing disease progression in SPMS that it will become a manageable condition in the same way that people live and manage with diabetes, and more recently HIV.
It is very rare for scientists to find an actual cure for a condition. But instead more common for a disease to become managed, both symptomatically and in terms of slowing progression.
As our understanding of the physiology and molecular pathways that cause MS increases newer drugs will eventually become available which manage disease so well that in practical terms it will be as good as a cure because it will become more likely that someone just diagnosed with MS will die of heart disease or a stroke before their MS becomes too severe.
Geoff said
Scott, thanks for your thoughts. As Yogi Berra said, it’s tough to make predictions, especially about the future.
Having lots of smart people researching diseases with the technology we’ve had available for the last 40 or 50 years clearly hasn’t been enough to find cures for many diseases. If it was going to be more of the same for the next 40-50 years, I’d agree with you completely. But I am more optimistic about the amount of technological progress we will see over the next few decades and the influence that will have on medical research.
Simon said
Hi Geoff
yes that’s a good point about the technological tools available now.
I think considering the huge investment put into drug research its probably true to say that science research has under-delivered over the last 25 years in terms of cures for diseases. You only have to look at cancer theraoy to see that it still largely depends on 1. chemotherapy and 2. surgery.
When the human genome code was completed it was generally assumed this would lead to a big breakthrough in treatments, but it hasnt really happened yet. Part of the reason for this is to do with the lack of effort that was previously put into effective delivery systems for drugs. So the drug might have been very effecive once it reached its target, but in humans the delivery to that target was poor. But this is now being addressed by looking at ability of using viral and/or lipid delivery systems to effectively deliver drugs to their targets.
So yes I think in many ways a big advance is really due. And perhaps we will see tihis happen now over the next decade.
Geoff said
I haven’t seen any stats on it, but I assume cancer research gets by far largest portion of the research dollar pie. I’m hoping that some of your cancer discoveries will also benefit other diseases, like MS, that have some immune system involvement, not to mention research into cancers of the central nervous system.
How to Get Six Pack Fast said
My friend on Facebook shared this link and I’m not dissapointed that I came to your blog.
Fernando said
Hi,
Have been reading your post at thisisms (I’m in ms limbo right now).
I don’t know if this drug fits here but it is somewhat related:
Cethrin
Cethrin for acute spinal cord injury:
Miami Project neurosurgeon scientists are in negotiations with Alseres Pharmaceuticals, Inc. for the University of Miami to become an investigator site for a Phase 2 multicenter trial of a neuroprotective drug Cethrin. In preclinical studies, the drug has been shown to block an inhibitory injury mechanism that prevents axons from regrowing. A Phase 1 trial involving 27 patients with acute SCI has already been completed and suggests Cethrin is safe and well-tolerated. The data from this trial also suggests the treatment may improve a participant’s motor and sensory function. When the Phase 2 trial is approved by the FDA, The Miami Project will proceed with the trial once it receives University of Miami Institutional Review Board (IRB) approval.
http://www.themiamiproject.org/Page.aspx?pid=339
Sounds impressive but for SCI, why not for MS?
I know, you’d have to have a separate list for “possible” drugs, but anyway, maybe we can keep an eye on this one.
Thank you for your efforts keeping this site up to date, they are very much appreciated.
Fernando
Fernando said
There is a drug called BVA-101 that could fit in the pre-clinical section:
http://www.thisisms.com/ftopict-7035-bva101.html
Is there a reason for it not being in that section?
If it is so, please forget this comment.
Fernando
Geoff said
Fernando,
Thanks for the information. I’m always glad to get tips. For a couple of reasons, I’m not going to add either drug. On BVA-101, it is preclinical, which I’ve given up tracking. I have some stats on the approval rate for drugs (in general, not MS-specific) that indicates that around 10-12% of all drugs that enter phase 1 go on to be approved one day. But of all the compounds tested in the preclinical stage, only around 10% ever even make it into clinical trials. So the odds are that around 1% of the drugs on the preclinical list will one day be treatments, and there aren’t even 100 drugs on the list…so after realizing the futility of it, I decided to give up. But I kept the preclinical section there, in case anybody is interested.
On Cethrin, it does sound interesting, and it sounds like the kind of thing that could one day be used in MS, but in trying to decide what I would include on the list and what I wouldn’t, I arbitrarily decided to include drugs that aren’t in trials for MS at the moment, but where there is a recent statement from the drug sponsor saying they intend to begin MS trials soon (or something to that effect). For Cethrin, I don’t see any reference to one day testing on people with MS. If there is a statement like that out there, let me know and I’ll add it to the list right away.
Thanks again for the info.
Geoff
Fernando said
Ok. You’re right.
How do you keep track of everything? Do you use google alerts or something similar? Goggle alerts is a fine tool.
Fernando
Geoff said
I don’t use Google alerts. I do use Google News, clinicaltrials.gov, the US MS Society’s list of clinical trials, Pubmed, conference abstracts from the major neurology/MS conferences and last but not least, tips I get, especially at ThisIsMS.com.
Geoff
AL RPH said
I believe this article will be of interest to all.
http://www.newswise.com/articles/view/555107/
Kreso said
Thank you for this list !!
You have put an optimitisc smile on my face !
Janet Summers said
Thanks for keeping us up to date, but do you have any news as to whether we should take the new vaccine for Swine flu? I am on avonex, and can’t decide what to do.
Geoff said
Janet,
I take rebif and I just got my H1N1 and seasonal flu vaccines today. I based it on this info from the Canadian MS society:
http://www.mssociety.ca/en/flu_h1n1_vacc.htm
I got the version without adjuvant.
Geoff