View Full Version : possible new drug

14th February 2012, 07:10

ALSTDI are proposing to do a human trial on this in America. They don't have a lot of money and are a not for profit organisation who usually do trials on mice so this is a big step for them. Let's hope it shows promise.


14th February 2012, 09:57
Here are the threads on ALSTDI:



14th February 2012, 10:20
Morning All,

Yes guys this one seems promising , just noted the costs around $50k for the treatment ,. cant see my PCT going hysterical about that one after the remarks about rilutec ,still heres hopeing , and John hows our Irene doing , hope alls well up there and the trial is still helping her cope ,

cheers all


14th February 2012, 11:55
Hi Pete,
Irene remains fairly stable, still standing for transfers and I still feel little bits of movement in her fingers. Fasciculations are still strong in biceps which we hope are a good sign. Updated on PLM the other day and she has lost a point on her FRS score but this is down to saliva issues which seem to get ramped up with the Sodium Chlorite and settle back down again when she is in her non dosing period - which started yesterday for 12 days. Basically I think she is a lot better than she would have been without the sodium Chlorite. Up to starting taking it in October she had lost 34 FRS points in 20months, an average of 1.7 per month. Since then in 4 months she has lost 1 point. It may be a coincidental plateau , placebo or it might be the Sodium Chlorite. My money is on the latter. The man is coming to day to check over the hoist which has been acquired for her transfers and was delivered in August /September last year. It has still not been used.

Take care.


14th February 2012, 22:46
Hi John

I think your best placed to know what is or is not working for Irene, despite what the scores might say ,as long as you both know ,i think pretty much thats all that matters, and have no doubts about the saliva issues, pretty much anything i take affects me ,in that way , still give our love to irene and wish you both well .

Best wishes

Pete and Lynne

15th February 2012, 09:18
Here's an eshot I received from ALSTDI about the clinical trial:

Today, ALS TDI announced that it will launch a Phase II clinical trial in ALS patients. I am honored to be able to share this news with you first, and personally thank you for your support in making the work leading up to this step possible. Thank you!

You can read more about this announcement in a Wall Street Journal article today by Amy Marcus by clicking here (http://forum.mndassociation.org/online.wsj.com/article/SB10001424052970204062704577221220332840952.html). This article raises many important issues related to moving this project forward, as well as general thoughts on the clinical trial process for a disease such as ALS.

In 2010, we published our findings in Nature Genetics that outlined for the first time the potential to treat ALS by focusing on changes in the activity of the immune system. Less than a year later, our research team began testing TDI 132 (Gilenya®) in 2011 for its ability to block certain immune cells from entering the central nervous system (CNS) where they can cause activities that result in damage to motor neurons. Gilenya® is currently marketed by Novartis as a treatment for certain forms of Multiple Sclerosis. Experiments at ALS TDI showed treatment with TDI 132 (Gilenya®) resulted in positive outcome on several disease measures in preclinical studies by blocking that cell activity. It is with this information in hand, and the fact this is an FDA-approved compound, that we are moving into clinical trials. This urgent approach to drug development is exactly what ALS TDI was created to do.

ALS TDI will announce more information about this trial soon, including the final design and enrollment information. We have chosen to announce this today because we believe that it’s important to be as transparent as possible with you and the entire ALS community on our research efforts. To complete the trial will take significant new fundraising, so we are focused on that now and need your help.

I are grateful to have your support and privileged to be able to share this exciting news with you today. I will be hosting a webinar with one of the principal investigators, Merit Cudkowicz, M.D., M.Sc., later this month. If you are able to join us for that, please register in advance online at www.als.net/webinars (http://www.als.net/ALS-Events/ALS-Webinars/) or if you have questions in advance, please just reply to this message.


Steve Perrin, Ph.D.
ALS Therapy Development Institute

15th February 2012, 14:02
Article on ALS News Magazine about the new trial:

Article Highlights

The nonprofit biotech ALS Therapy Development Institute plans to launch a phase 2 trial to determine the safety and tolerability of TDI 132 — also known as fingolimod — in ALS.
Fingolimod (brand name Gilenya) is approved by the U.S. Food and Drug Administration for the treatment of multiple sclerosis. In humans, Gilenya inhibits certain immune system cells from entering the central nervous system, where they can result in damage to motor neurons.
ALS TDI reports that mice treated with TDI 132 showed positive outcomes on several disease measures.


17th February 2012, 16:46
Another article on ALSTDI / Gilenya:

Researchers announced on 14 February 2012 that they are planning to start a clinical trial this year to treat amyotrophic lateral sclerosis with Gilenya, an immunomodulator already approved to treat multiple sclerosis. The Amyotrophic Lateral Sclerosis Therapy Development Institute (ALS-TDI) in Cambridge, Massachusetts, started pursuing this idea in mice in late 2010, after discovering that immune pathways are key to the disease. With only 18 months between the idea’s inception and the start of a clinical study, “this is the definition of bench to bedside,” said Steven Perrin of the ALS-TDI.


Gilenya is one of two treatments with which the ALS-TDI hopes to start trials this year. The Institute initially got into the immune system field when they discovered that blocking the interaction between CD40 on T cells and its ligand CD40L on antigen-presenting cells extended lifetime in ALS model mice (see ARF related news story (http://www.alzforum.org/new/detail.asp?id=2407) on Lincecum et al., 2010 (http://www.alzforum.org/pap/annotation.asp?powID=100973)). The ALS-TDI partnered with Bioden Idec of Cambridge, Massachusetts, to develop an antibody that interferes with CD40L. However, the team expects this project to take a while, needing time to make a human version of the antibody and run a Phase 1 trial, Perrin said, so the researchers in parallel tested a handful of medicines they hoped would have similar immunomodulatory effects but a shorter development time

Like the CD40 antibody, Gilenya reduced the number of circulating lymphocytes and diminished macrophage attacks on peripheral nerves in ALS model mice overexpressing mutant human superoxide dismutase 1, which causes familial ALS. The single Gilenya dose that the ALS-TDI tested in those mice extended lifespan by a week. The ALS-TDI has not yet published these preclinical data. Given that Gilenya is already FDA approved, Institute scientists decided to go straight to a human study, Perrin said.

The researchers have not finalized their study design, but it will likely include two phases. The first, Perrin said, will be a Phase 2a trial with perhaps 30-50 volunteers to make sure the drug is safe for people with ALS. Following that, he envisions a larger, Phase 2b of approximately 250 people.http://www.alzforum.org/new/detail.asp?id=3065

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