Subcommittee on Neurological Disease Committee on June 8th, 2010

[Dr. Denise Figlewicz]

I would like to say two things.

First of all, it must be stated that neurological diseases share some points in common. Every researcher goes about his or her work, but constantly follows the evolution of the research work being done on other diseases such as Huntington's, Parkinson's and Alzheimer's. Indeed, certain drugs that are effective in the case of certain diseases can also be effective in the treatment of ALS. This is why we are not asking for resources for ALS specifically, but for research. The effective treatment might be found a little bit outside of our own field of research.

Secondly, Alex talked about the creation of model systems for the in-lab discovery of treatments. What is also lacking is a budget to transition effective in-lab treatments all the way to the treatment of human patients. Between the two, there is a big black hole.

It is very difficult, because a researcher is not specialized in the application of research to humans. We are specialized in critical research, but there are not many resources available for the development of a treatment between the laboratory and the clinics. The situation is the same for all diseases.

[Dr. Alex Parker]

I previously worked in the United States on Huntington's, for which a system exists. Let us say that we are successful in finding new drugs that are effective in the model system, be it with a fly, a worm or a mouse. We then move on to clinical trials. There is a system in place for Huntington's disease, but there is still nothing for ALS.

In fact, the present problem is that, even if I discover something that works very well with another model, what am I going to do if I do not have the money to carry out a test on a mouse? Clinical trials are assuredly too costly. I cannot pay for everything.

[The Chair, Conservative, Joy Smith]

Thank you, Dr. Parker.

We're going to go on. With the committee's permission, may I ask a question? Is that okay?

[Some hon. members]

Agreed.

[The Chair, Conservative, Joy Smith]

There was one thing you said, that two or three genes could potentially lead to the cause of ALS. Then, Ms. Figlewicz, you said there could be a linkage between the neurological diseases.

Dr. Parker, I think you could answer this: do you think if research was done with these genes there could be a possible linkage, say, to Parkinson's? And why do you think that?

[Dr. Alex Parker]

Yes, I think so. From my lab, I know the recent genes we've identified in ALS affect toxicity in our models for Huntington's disease and also our model for Alzheimer's disease. There's a definite crossover. I don't know about Parkinson's because we don't have that in our lab.

It's somewhat surprising, but maybe not. The point is well taken that anything we find in one of these diseases, for the most part, will have a good chance in functioning across other late onset neurogenetic diseases as well.

[The Chair, Conservative, Joy Smith]

That's very interesting. Thank you, Dr. Parker, for your answer.

We'll now go to Ms. Hughes.

[Carol Hughes, NDP, Algoma—Manitoulin—Kapuskasing, ON]

Thank you.

I greatly appreciate your time here today. I think it's imperative that we hear from as many groups as we can on the neurological illnesses.

I do not know if it is Ms. York or Ms. Travis Valo who made the comment about drug trials. I am wondering how many clinical trials are being done at present in Canada and where. Is it a Baycrest Hospital in Toronto, or elsewhere?

[Felicia Travis Valo]

It's Sunnybrook Hospital. They've had one clinical trial, and that entailed the use of lithium. I think it was going to be—and please correct me if I'm wrong—an 18-month study. It was stopped midway through because it was found to be totally inefficacious.

I think there is a trial under way shortly...

[Melanie York]

Ceftriaxone.

[Felicia Travis Valo]

Ceftriaxone, yes.

[Melanie York]

To follow up on this point, I was on the lithium trial. This was based on a study in Italy. Unfortunately, I think it had a lot of holes in it.

I think everybody is desperate to grab on to something hopeful. I began the study around February 2008. They stopped it in September after six months, and then it took me six months to find out whether I was on the lithium or the placebo. In the end they found that people who had been on the lithium had trended a little more downward. That put so much fear into me. You put your life in the way of...you don't know.

I'm sorry, I need a drink.

[Carol Hughes, NDP, Algoma—Manitoulin—Kapuskasing, ON]

Thank you very much.

I can understand the difficulties for the family members, for those who are around us to support us. I have a sister who has Alzheimer's. She's 57 years old and she was diagnosed at age 50. I understand the frustration with regard to the supports out there.

You did talk about support networks, and I think we need to dwell on that a little more. It's obvious you do get the runaround, that it's extremely difficult to obtain any information or sometimes the direct information as to where you actually need to go for the support. Obviously there is some indication that we need better documents out there. It seems that whether it's research or this documentation or support networks, one of the key factors is the funding.

I'm trying to get some sense as to the main agency where you get all that information, because obviously the ALS Society is very limited as well.

[Melanie York]

I get bits and pieces from the hospital, from the integrated clinic; otherwise I would say I make myself knowledgeable.

[Carol Hughes, NDP, Algoma—Manitoulin—Kapuskasing, ON]

Is there anyone in particular who is able to assist you with respect to direction as to programming, for example, where you can get the programming for your house--the March of Dimes or anything like that? Is it frustrating? I'm sure it must be frustrating to have to go to a variety of different agencies for different assistance.

[Melanie York]

I would think there is not a collective reality around services. I don't want to say there are no services--there are--but I don't feel they are coordinated and presented in a thorough and needed way.

I don't know if that was your experience.

[Felicia Travis Valo]

Definitely.

[Melanie York]

You have to be smart and you have to ask questions. That's true in life anywhere. You have to be your own advocate, even with ALS, and there is only so much energy and so much room you have, but the reality is, I would say, you have to go after your own treatment and your own sense of taking care of yourself. You really have to manage and find your way through that system.

There is a lot of stuff online, and there are doctors in the States, but I just feel that we haven't coordinated everything. The communication, I feel, is not strong. Most of the communication is so depressing--seriously, not that I'm a bundle of joy here. It is so depressing that you don't get a sense that there is a forward movement of advocacy for real change. You feel lost in the system and you don't feel there is a real forward-thinking movement and approach to shift this to a new place.

[Carol Hughes, NDP, Algoma—Manitoulin—Kapuskasing, ON]

Could I just ask, Mr. Parker, are there a certain number of universities in Canada looking at trying to do some research on that? How many dollars are we talking about that you would consider a boost right now?

[Dr. Alex Parker]

Not for awareness, obviously, but for the actual research itself...? How much? Any increase is great; I'm not going to say anything bad about that. We are asking for an across-the-board increase of 1%, and that would benefit all research itself. How much would it be just for the neurological diseases? I suppose an extra....

With an average CIHR grant, for example, if you're lucky, you can get about $300,000 over five years. That's a lot; that would set up a new lab like the one I have for a long time. But those are so hard to come by.

On the amount of funding you get, it's kind of the same. It's just that the number awarded is very low. So if it could be bumped up even a percentage point, that would make a big difference. What happens now is you have an idea and you have to go around to smaller, different agencies and hopefully package enough research to go for the big one, and then if you get it you can finish a project and hopefully find something interesting. That is hard to do.

Right now I have a small amount of funding from the CIHR. I'm very grateful for the support from ALS Canada, and I have funding from Switzerland. As a new person, it has been hard to get into the big grants from the CIHR.

I don't know. Just an increase...I can't give you a dollar amount because I don't have a budget.

[The Chair, Conservative, Joy Smith]

Thank you, Dr. Parker.

We'll now go to Mr. Brown.

[Patrick Brown, Conservative, Barrie, ON]

Thank you, Madam Chair.

Thank you for all the testimony. It has certainly been very interesting.

Felicia, you mentioned that here we are in ALS Awareness Month and there doesn't seem to be that much focus on ALS. Just as a silver lining, we have our annual walk in Barrie this Saturday, and we're expecting a huge turnout.

The last time I met you was with Derek Walton, whom we call Braveheart in Barrie. He is in a wheelchair but still manages to skydive to raise money for ALS and the work that's being done at Sunnybrook. Our community certainly embraced him, and there has been tremendous exposure and awareness for ALS, I believe, in Barrie, and I'm sure in lots of small towns across Canada there are similar people like Derek who are raising awareness. I can only hope that is going to continue to grow.

I want to ask a few questions with regard to opportunities that we may not be engaging. It has been clear what the need is in terms of care giving, and I appreciate how that was referenced. That is certainly good advice that we should put in our report. But concerning research and clinical trials, what are we missing the boat on by having, as you mentioned, a very low amount? What clinical research, what research, are we not doing?

Alex or Denise, is there any light you can shed for the committee? If there were greater investments in research, what would we be doing right now?

[Dr. Denise Figlewicz]

Our clinical trials network is running a trial on ceftriaxone, and a few of the centres are running a second trial. Some of our clinicians work as part of American groups also, so there's an advantage there. We could carry out more trials, because there are things on the launch pad waiting to be tried. For example, I was at the American Academy of Neurology conference in April, and in the session they were talking about the progression of clinical trials from phase one, which is just safety, through phase three, which is the full-blown proper clinical trial. And there are a number of candidates sort of waiting their turn.

[Patrick Brown, Conservative, Barrie, ON]

Do you have any information on those candidates and what's waiting on the launch pad right now?

[Dr. Denise Figlewicz]

There are drugs that act on different mechanisms. One of them is supposedly going to act on intercellular protein aggregates, which has something in common with a number of neurodegenerative disorders. So it's a compound that works on that. That's from a small biotech company. There is something from another small biotech company that is working on the main system of cell death in motor neurons, which has been identified in the lab.

There are a few problems. Small biotech companies can only go so far because of their commercial reality. If something isn't snapped up by a large biotech company and individual clinical trial centres don't have funding--which they don't--this is the stuff that sort of sits by the wayside.