Research for Familial ALS

marash (May 20, 2009 1:46:15 PM)
(This user has entered Research for Familial ALS)

marash (May 20, 2009 1:46:25 PM)
hello?

dr-benatar (May 20, 2009 1:46:51 PM)
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ChatMaster (May 20, 2009 1:47:40 PM)
(This user has entered Research for Familial ALS)

marash (May 20, 2009 1:48:28 PM)
Hello Chatmaster

ChatMaster (May 20, 2009 1:48:33 PM)
Hello marash! Welcome back to our 3rd ALS Experts Chat - Research for Familial ALS!!

marash (May 20, 2009 1:48:49 PM)
Chatmaster, may I ask the first question?

dr-miller (May 20, 2009 1:49:02 PM)
(This user has entered Research for Familial ALS)

ChatMaster (May 20, 2009 1:49:29 PM)
Welcome dr-miller!

ChatMaster (May 20, 2009 1:49:49 PM)
Sure Marash you can have the first question, I will put you on the list!

scott (May 20, 2009 1:49:54 PM)
(This user has entered Research for Familial ALS)

dr-miller (May 20, 2009 1:49:54 PM)
Great to be here.

ChatMaster (May 20, 2009 1:50:03 PM)
Hello everyone! Welcome to the Research for Familial ALS Chat.

ChatMaster (May 20, 2009 1:50:16 PM)
The Research for Familial ALS chat will begin in 9 minutes

Doug (May 20, 2009 1:52:24 PM)
(This user has entered Research for Familial ALS)

ChatMaster (May 20, 2009 1:52:32 PM)
Welcome Doug!

ChatMaster (May 20, 2009 1:52:46 PM)
The Research for Familial ALS chat will begin in 7 minutes

ChatMaster (May 20, 2009 1:53:03 PM)
We are going to hold this chat on a 'first asked, first answered' basis. Please type the following "ChatMaster, I have a question" and I will add you to the list of chat names and call on people to ask their questions one at time.

ChatMaster (May 20, 2009 1:54:18 PM)
When you are directing a question or response to someone within the chat, please begin your question/response with the chatters name (Example: "dr-miller, please tell me about the latest developments in ...")

ChatMaster (May 20, 2009 1:55:08 PM)
You can see all the people participating in this chat by clicking on the Users tab to your right

dr-benatar (May 20, 2009 1:55:50 PM)
(This user has entered Research for Familial ALS)

ChatMaster (May 20, 2009 1:55:52 PM)
The Research for Familial ALS chat will begin in 4 minutes. We are waiting for Dr. Benatar and a few others to arrive

ChatMaster (May 20, 2009 1:56:05 PM)
Welcome dr-benatar!

dr-benatar (May 20, 2009 1:56:14 PM)
Thanks. Pleased to be here

ChatMaster (May 20, 2009 1:56:26 PM)
The Research for Familial ALS chat will begin in 3 minutes

ChatMaster (May 20, 2009 1:57:08 PM)
We are going to hold this chat on a 'first asked, first answered' basis. Please type the following "ChatMaster, I have a question" and I will add you to the list of chat names and call on people to ask their questions one at time.

ChatMaster (May 20, 2009 1:57:28 PM)
When you are directing a question or response to someone within the chat, please begin your question/response with the chatters name (Example: "dr-benatar, please tell me about the latest developments in ...")

gregory (May 20, 2009 1:57:49 PM)
(This user has entered Research for Familial ALS)

ChatMaster (May 20, 2009 1:58:03 PM)
Welcome gregory!

ChatMaster (May 20, 2009 1:58:20 PM)
The Research for Familial ALS chat will begin in 1 minute

ChatMaster (May 20, 2009 1:59:40 PM)
Hello everyone! Welcome to the Research for Familial ALS Chat.

marash (May 20, 2009 1:59:48 PM)
Hello!

ChatMaster (May 20, 2009 1:59:59 PM)
Two of the world's leading experts in Research for Familial ALS will be hosting the 3rd installment of MDA's ALS Awareness Month "Ask the Expert" online chat series. Since the underlying biology of the disease is similar to those of familial and sporadic ALS, everyone should take part in this chat.

dr-benatar (May 20, 2009 1:59:59 PM)
Good afternoon

dr-miller (May 20, 2009 2:00:18 PM)
Hello

ChatMaster (May 20, 2009 2:00:35 PM)
Michael Benatar, MBChB, MS, DPhil is Associate Professor of Neurology, Associate Professor of Epidemiology, Director of the Emory Electromyography Laboratory and Co-Director of the MDA Clinic at Emory University in Atlanta. Dr. Benatar is highly involved in research for familial ALS, currently working on the evaluation of pre-symptomatic carriers of a mutation in the SOD1 gene as well as a Phase II/III study of Arimoclomol

ChatMaster (May 20, 2009 2:01:06 PM)
Timothy Miller, MD, PhD is an Assistant Professor of Neurology at Washington University in St. Louis. Dr. Miller's research focuses on methods to turn off harmful genes in the brain and spinal cord as a way to bring novel treatments to neurodegenerative diseases, in particular ALS. His other research interests include understanding how dysfunction of mitochondria, the power generators for cells, may be causing ALS. Dr. Miller has become a national leader in translational neuroscience and new therapeutic approaches for ALS.

michaelW (May 20, 2009 2:01:36 PM)
(This user has entered Research for Familial ALS)

ChatMaster (May 20, 2009 2:01:39 PM)
We are going to hold this chat on a 'first asked, first answered' basis. Please type the following "ChatMaster, I have a question" and I will add you to the list of chat names and call on people to ask their questions one at time.

ChatMaster (May 20, 2009 2:01:48 PM)
Welcome michaelW

ChatMaster (May 20, 2009 2:01:59 PM)
When you are directing a question or response to someone within the chat, please begin your question/response with the chatters name (Example: "dr-benatar, please tell me about the latest developments in ...")

ChatMaster (May 20, 2009 2:02:31 PM)
Marash, you are up first, please ask your question!!

marash (May 20, 2009 2:02:57 PM)
To Either Dr, Realistically, How effective will SOD1 antisense treatments be for fALS patients with the SOD1 mutation?

dr-benatar (May 20, 2009 2:03:24 PM)
I think we should let Dr Miller answer this first

dr-miller (May 20, 2009 2:04:22 PM)
We do not yet know how effective they will be. We will be testing this in clinical trial. However, based on animal models of SOD1 ALS, it is very likely that decreasing SOD1 levels will be beneficial for the disease. We think that our antisense therapy can do this.

ChatMaster (May 20, 2009 2:05:02 PM)
dr-benatar, would you like to add to this?

dr-benatar (May 20, 2009 2:05:33 PM)
I agree that this is a strategy that makes tremendous sense. Whether or not it is a strategy that works will have to await cilnical trials. That's how we investigate the potential efficacy (and safety) of any new therapy.

ChatMaster (May 20, 2009 2:06:19 PM)
marash, does that answer your question and do you have any follow-up questions to the doctors' responses?

marash (May 20, 2009 2:06:37 PM)
Thank You, i'd like to ask a question after everyone else

ChatMaster (May 20, 2009 2:06:58 PM)
ok, thanks

scott (May 20, 2009 2:06:59 PM)
ChatMaster, I have a question

ChatMaster (May 20, 2009 2:07:18 PM)
okay scott, please go ahead with your question please

scott (May 20, 2009 2:07:33 PM)
Drs., could you please share information about Arimoclomol?

dr-miller (May 20, 2009 2:08:10 PM)
I will let Dr. Benatar answer this first.

dr-benatar (May 20, 2009 2:08:53 PM)
We have recently initiated a clinical trial of arimoclomol in patients with SOD1 positive familial ALS. Our goals are to determine whether the drug is safe and well tolerated in this population, and also whether or not the drug is effective in slowing disease progression. Based on its mechanism of action and the preclinical data that has been published, as well as the fact that it target a very relevant pathological mechanism of the disease, we think that it is a very encouraging agent.

dr-benatar (May 20, 2009 2:09:17 PM)
We are currently enrolling people for this study.

dr-benatar (May 20, 2009 2:10:02 PM)
Is there something else about arimoclomol that you would like to know, Scott?

scott (May 20, 2009 2:10:07 PM)
ChatMaster, I have a follow-up question - Dr. Benatar, you're currently enrolling, but how long is the study?

ellen (May 20, 2009 2:10:24 PM)
(This user has entered Research for Familial ALS)

dr-benatar (May 20, 2009 2:10:38 PM)
Each person who participates in the study will be randomized to receive either arimoclomol or placebo for a total of 12 months

ChatMaster (May 20, 2009 2:10:39 PM)
welcome ellen!

dr-benatar (May 20, 2009 2:11:05 PM)
After the 12 months of the study are completed, we hope to make the drug available in an open-label fashion, but we're still working on this.

scott (May 20, 2009 2:11:16 PM)
Thank you Dr. Benatar

ChatMaster (May 20, 2009 2:11:51 PM)
ellen, do you have a question for either dr-miller or dr-benatar?

michaelW (May 20, 2009 2:12:06 PM)
To either Dr.: What information do either of you have on the efficasy and/or positive effects of IPLEX, Knopp Drug or Lithium for treatment? IPLEX has been approved for compationate use but I cannot find any studies indicating that it has meaningful positive effects. Knopp and Lithium are both in trials and sucess and benefits are yet to be reported with any meaningful results- at least for patient consumption. I do not know if IPLEX is also in study trials.

ellen (May 20, 2009 2:12:09 PM)
Yes Chatmaster

ChatMaster (May 20, 2009 2:12:10 PM)
We are going to hold this chat on a 'first asked, first answered' basis. Please type the following "ChatMaster, I have a question" and I will add you to the list of chat names and call on people to ask their questions one at time.

michaelW (May 20, 2009 2:12:27 PM)
sorry. did not know the protocol.

ChatMaster (May 20, 2009 2:12:38 PM)
ellen I will put you on the list as next up after michaelW's question in answered!! Thankk

ellen (May 20, 2009 2:12:43 PM)
ok

ChatMaster (May 20, 2009 2:13:08 PM)
That's fine michaelW

ChatMaster (May 20, 2009 2:13:44 PM)
Drs. please proceed with answering michaelW's question.

dr-benatar (May 20, 2009 2:13:50 PM)
IPLEX has not really been studied. I think that it would be wisest for the drug to be evaluated in a formal clinical trial before we can recommend it as treatment for ALS. Lithium as you know, is currently under investigation in a clincial trial. Clinical trials really are the very best way for us to establish the balance between the efficacy and safety/tolerability of a new drug.

dr-miller (May 20, 2009 2:14:51 PM)
I do not have any information regarding how Knopp and Lithium are doing in clinical trial. Anecdotal evidence thus far suggests that Lithium will not be quite as effective in the U.S. as was reported in the initial trial, but again, we need the results of the clinical trial to determine this.

dr-benatar (May 20, 2009 2:15:18 PM)
I similarly do not have any information about the Knopp drug I am afraid

ChatMaster (May 20, 2009 2:15:41 PM)
michaelW do you have any follow-up questions?

dr-miller (May 20, 2009 2:15:51 PM)
IPLEX is an interesting candidate for a trial. There is relatively little data available on its efficacy thus far, but agree we need to test it.

michaelW (May 20, 2009 2:16:00 PM)
not for the moment.

ChatMaster (May 20, 2009 2:16:07 PM)
ok, thank you

ChatMaster (May 20, 2009 2:16:23 PM)
ellen, please proceed with your question!

ellen (May 20, 2009 2:16:28 PM)
Dr.'s, can any one enter a trial, or do make the judgement according to individuals health conditions? Do you exclude for any reasons?

dr-benatar (May 20, 2009 2:17:23 PM)
Clinical trials always have formal inclusion and exclusion critiera. The researchers conducting a trial will always need to make sure that anyone who would like to participate in a trial, meets these criteria. But anyone can enquire about a trial to find out if they might be eligible to participate.

vickiC (May 20, 2009 2:17:29 PM)
(This user has entered Research for Familial ALS)

ChatMaster (May 20, 2009 2:17:37 PM)
welcome vickiC!

dr-miller (May 20, 2009 2:17:49 PM)
Each trial will have specific entry criteria depending on what is being tested. Typically, for thos who are interested in participating there is likely to be a research study available, even if not a clinical trial of a new drug.

ChatMaster (May 20, 2009 2:17:56 PM)
We are going to hold this chat on a 'first asked, first answered' basis. Please type the following "ChatMaster, I have a question" and I will add you to the list of chat names and call on people to ask their questions one at time.

ellen (May 20, 2009 2:18:11 PM)
Thank you Dr.'s

ChatMaster (May 20, 2009 2:18:17 PM)
Thank you ellen

ChatMaster (May 20, 2009 2:18:39 PM)
marash, please proceed with your second question,

marash (May 20, 2009 2:19:17 PM)
To Dr.Miller, I have been following fALS antisense drug for over two years now. When will the drug be released for phase 1 testing? Similarly, what about phase II testing? What will be the criteria for eligibility for the clinical trial?

maryR (May 20, 2009 2:19:26 PM)
(This user has entered Research for Familial ALS)

ChatMaster (May 20, 2009 2:19:38 PM)
welcome maryR

ChatMaster (May 20, 2009 2:19:54 PM)
Welcome to the Research for Familial ALS Chat.

ChatMaster (May 20, 2009 2:20:09 PM)
We are going to hold this chat on a 'first asked, first answered' basis. Please type the following "ChatMaster, I have a question" and I will add you to the list of chat names and call on people to ask their questions one at time.

ChatMaster (May 20, 2009 2:20:37 PM)
Currently the doctors are answering a question from marash

dr-miller (May 20, 2009 2:20:37 PM)
Thank you for your continued interest. We are in the final phase of our application to the FDA. I anticipate early June for this application to go through. If all goes well, we hope to start enrolling this fall for Phase I

dr-miller (May 20, 2009 2:21:17 PM)
Phase II will depend on results of Phase I, but will probably follow about 2 years later, but again will depend on the Phase I trial findings.

gregory (May 20, 2009 2:21:24 PM)
CM, may I ask a ?

ChatMaster (May 20, 2009 2:21:50 PM)
sure gregory, you will be up next to ask your question. I will call on you

marash (May 20, 2009 2:22:02 PM)
may I ask a follow up question?

dr-miller (May 20, 2009 2:22:07 PM)
Criteria for the SOD1 Antisense trial will be positive SOD1 Genetic test and early in the course of the disease. There will, of course, be other criteria, but those ar teh major two.

gregory (May 20, 2009 2:22:21 PM)
Dr's: So is SOD-1 a toxic gain of function? Second ?: do you know any families with sensory or pain features.

dr-benatar (May 20, 2009 2:22:47 PM)
Perhaps Dr Miller could address the question of toxic gain of function and I'll tackle the second

dr-miller (May 20, 2009 2:23:05 PM)
I belive that SOD1 is a toxic gain of function. We are working hard on figuring what is the toxicity.

dr-benatar (May 20, 2009 2:23:33 PM)
Sensory involvement probably does occur in ALS, but it is not a major feature of the disease. At least from pathological studies, there is some evidence that sensory involvement may be more common in familial ALS than it is in sporadic ALS.

gregory (May 20, 2009 2:23:49 PM)
cheers

dr-miller (May 20, 2009 2:23:59 PM)
... and is seen in animal models of ALS if look hard for it.

gregory (May 20, 2009 2:24:22 PM)
CM: I'll ask another when you are ready.

marash (May 20, 2009 2:24:32 PM)
CM: I will also like to ask another question

ChatMaster (May 20, 2009 2:25:26 PM)
okay, gregory please proceed with your next question and then Marash you can go after his second question is answered

gregory (May 20, 2009 2:25:31 PM)
Where should I be sending my patients blood for genetic testing (if SOD-1 is negative) and who pays for it?

dr-benatar (May 20, 2009 2:26:25 PM)
We are doing genetic testing for SOD1 as well as other genes, but as part of a research study. If we do the testing as part of the research then we pay for it. I don't believe that clinical testing is available for any genes other than SOD1.

dr-miller (May 20, 2009 2:26:42 PM)
As far as I know there is not a commercial lab doing genetic tests other than SOD1. Research labs are doing this and, if so, the research lab would then pay for it.

gregory (May 20, 2009 2:27:13 PM)
Thanks. Is MDA dialed in with your labs?

dr-benatar (May 20, 2009 2:27:33 PM)
Gregory, you can always contact us at fals@emory.edu for more information about this and other familial ALS research studies.

dr-miller (May 20, 2009 2:27:34 PM)
For example, we are testing all familial ALS patients here (Washington University) for TDP-43 and are gearing up to test for FUS/TLS, the latest gene discovered.

gregory (May 20, 2009 2:28:05 PM)
You guys rock

dr-miller (May 20, 2009 2:28:05 PM)
If you want info on genetic testing through us, email allredp@neuro.wustl.edu

ChatMaster (May 20, 2009 2:28:18 PM)
Thank you doctors, Marash please proceed with your next question

dr-miller (May 20, 2009 2:28:36 PM)
Yes, MDA is supporting our Phase I clinical trial for antisense oligos for SOD1 ALS

marash (May 20, 2009 2:28:55 PM)
Dr-Miller, When you say early onset, are you referring to less than two years?

melissa (May 20, 2009 2:29:01 PM)
(This user has entered Research for Familial ALS)

melissa (May 20, 2009 2:29:13 PM)
Hello

ChatMaster (May 20, 2009 2:29:21 PM)
welcome melissa to the Research for Familial ALS chat!!

melissa (May 20, 2009 2:29:32 PM)
Thank you, has it already started?

ChatMaster (May 20, 2009 2:29:34 PM)
We are going to hold this chat on a 'first asked, first answered' basis. Please type the following "ChatMaster, I have a question" and I will add you to the list of chat names and call on people to ask their questions one at time.

ChatMaster (May 20, 2009 2:29:48 PM)
Yes, we have been chatting for about 30 mins

ChatMaster (May 20, 2009 2:30:00 PM)
please let me know if you have a question for the doctors

melissa (May 20, 2009 2:30:02 PM)
thank you, I apologize for being late, I'm at work

melissa (May 20, 2009 2:30:15 PM)
Dr. Benatar is my doctor :) Here to support him

dr-miller (May 20, 2009 2:30:16 PM)
To deliver antisense oligos we need to put in a small pump with a catheter near the spinal cord. To make sure the surgery will be well tolerated, we are including patients with a forced vital capacity greater than 65%. That is what I mean by early in the disease.

dr-benatar (May 20, 2009 2:30:28 PM)
I also wanted to add that we are enrolling people in the arimoclomol clinical trial in the early stages of the disease - by which we mean that the diagnosis was made less than 9 months prior to enrollment. But other people may use different definitions.

dr-benatar (May 20, 2009 2:31:01 PM)
Thanks for joinnig Melissa. Nice to have you online.

marash (May 20, 2009 2:31:24 PM)
Thank You Dr.Miller

marash (May 20, 2009 2:31:35 PM)
Chatmaster I have a question (after everyone else)

ChatMaster (May 20, 2009 2:31:39 PM)
We have plenty of time left for more questions and plenty of chatters in the room. Who would like to ask the next question of dr miller or dr benatar

ChatMaster (May 20, 2009 2:32:07 PM)
Marash, please go ahead with your next question

marash (May 20, 2009 2:33:00 PM)
To Either Dr, Do you think VEGF is a good target for clinical trials? I know SB-509 is a VEGF drug that is currently being used.

melissa (May 20, 2009 2:33:50 PM)
Chatmaster I have a question for either Dr. (taking number for my turn :) )

dr-benatar (May 20, 2009 2:34:11 PM)
I don't have a well-formed opinion on this subject. As you know, other growth factors have been tested in clincial trials, but have not been found to be effective.

dr-miller (May 20, 2009 2:34:26 PM)
VEGF via viral delivery or given directly in the fluid that surrounds the brain and the spinal cord delayed disease in the animal models. I think it is a good target for delaying disease. I wonder about the safety of VEGF for in terms of its effect on the blood vessels. This will need to be worked out.

ChatMaster (May 20, 2009 2:35:03 PM)
melissa, I will put you on the list

marash (May 20, 2009 2:35:08 PM)
Thank You Dr

dr-benatar (May 20, 2009 2:35:11 PM)
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ChatMaster (May 20, 2009 2:35:27 PM)
melissa, please proceed with your question

dr-benatar (May 20, 2009 2:35:35 PM)
(This user has entered Research for Familial ALS)

dr-miller (May 20, 2009 2:35:44 PM)
I think one of the major issues with previous growth factors trials has been delivery to the brain and spinal cord. This may be improved upon in upcoming trials.

ChatMaster (May 20, 2009 2:35:47 PM)
welcome back dr-b!!

dr-benatar (May 20, 2009 2:35:58 PM)
Sorry, I seem to have been logged off the system, but am now back

marash (May 20, 2009 2:36:33 PM)
Thank You Dr's for your hard work and diligence. You have answered all my questions thoroughly and given me a lot of hope. Thank you and God Bless.

dr-benatar (May 20, 2009 2:36:48 PM)
You are very welcome. Thanks for joining us today.

melissa (May 20, 2009 2:37:00 PM)
My question is about IPlex, I have heard so much about it, especially when I was in DC. If it can truly help PALS, and they administer it to children, why can't we access it? Do you think it helps fALS? Is the patent problem the reason we can't have this drug? I have lost 8 family members and test positive for the SOD1 A4V gene myself, am involved in study at Emory with Dr. Benatar

dr-benatar (May 20, 2009 2:38:01 PM)
Melissa, we discussed IPLEX a little before you joined the chat. The problem with IPLEX, in my mind, is that it has not been studied in ALS. We really don't know whether it would be effecive, harmful or neutral. I think that a clinical trial will be needed to answer the questions you have.

dr-benatar (May 20, 2009 2:38:28 PM)
Chat master, I just typed an answer to this question, but it didn't seem to post on the chat page.

dr-miller (May 20, 2009 2:39:10 PM)
IPLEX is an interesting potential therapy. True that it has made headlines recently (New York Times recently). Currently, however, there is little data. It definitely needs to be tested.

melissa (May 20, 2009 2:39:52 PM)
Why do the block pals from being part of trials? We are going to die anyway, why not let us volunteer for these studies? "compassionate" use makes me laugh, I am angry there is nothing compassionate about letting people die and over 100 years with no medication or cure

ChatMaster (May 20, 2009 2:39:54 PM)
dr-benatar, did your answer finally go through?

dr-benatar (May 20, 2009 2:39:59 PM)
yes.

ChatMaster (May 20, 2009 2:40:01 PM)
thanks

melissa (May 20, 2009 2:40:26 PM)
I would happily volunteer for ANY study to keep this from my children or any other member of my family

scott (May 20, 2009 2:41:25 PM)
Chatmaster, I have a follow-up question to melissa's

ChatMaster (May 20, 2009 2:41:43 PM)
okay scott I will put you on the list

dr-miller (May 20, 2009 2:41:43 PM)
Melissa, it is through brave volunteers like you that we will find new therapies for this disease. I can understand your frustration with our lack of progress on finding a cure. I believe that there are groups trying to open up IPLEX for clinica trial.

dr-benatar (May 20, 2009 2:41:53 PM)
I am not sure that I understand your question Melissa. If you're asking why we do clinical trials rather than simply prescribe all unproven therapies, then I think the answer is that we only learn whether or not a drug is beneficial (or harmful) when we test it in a trial. If we simply prescribe it and don't adequately study it then we won't know whether it works for the individual - or whether it would help individuals who are affected in the future (e.g. future generations of people with familial ALS)

dr-benatar (May 20, 2009 2:42:19 PM)
Did we answer your question Melissa?

ellen (May 20, 2009 2:42:53 PM)
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melissa (May 20, 2009 2:43:27 PM)
yes thank you

melissa (May 20, 2009 2:43:37 PM)
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ChatMaster (May 20, 2009 2:43:44 PM)
okay, scott please proceed with your next question

scott (May 20, 2009 2:43:58 PM)
Mine is a follow-up to Melissa's question. Drs., isn't it true that some drugs have actually shown to accelerate the disease process, and that's another reason why clinical trials are so important?

gregory (May 20, 2009 2:44:05 PM)
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maryR (May 20, 2009 2:44:10 PM)
Chatmaster, I have a question.

dr-benatar (May 20, 2009 2:44:15 PM)
Absolutely. This was true of minocycline, for example

marash (May 20, 2009 2:44:23 PM)
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ChatMaster (May 20, 2009 2:44:27 PM)
i will put you on the list maryR!

dr-miller (May 20, 2009 2:44:29 PM)
Agree

maryR (May 20, 2009 2:44:39 PM)
thank you

ChatMaster (May 20, 2009 2:45:01 PM)
maryR, please proceed with your question!!

maryR (May 20, 2009 2:45:49 PM)
Dr. Miller and Dr. Benatar, if money were no object, what would be the limiting factors in ALS research?

dr-benatar (May 20, 2009 2:47:42 PM)
This is an excellent and complex question. I think that one of the major limitations is that we don't know the cause of the disease (or the risk factors for disease) except in the small number of people with famiilal ALS. Also, I suspect that the disease process begins well before symptoms appear and this means that we begin treatment too late in the course of the disease. We need to find ways to recognize/diagnose the disease at an earlier stage so that we can intervene with potential therapies at an earlier stage. Finally, a better understanding of the disease biology would help us to develop rational therapies.

dr-miller (May 20, 2009 2:48:02 PM)
Figuring out why motor neurons die has been a tremendous difficult question that has occupied many laboratories for many years. Also, many drugs have been tried that haven't worked. In other words, this has been a tough question that has occupied lots of resources, money, and time. More money would make the process move faster (more people working on it), but I think we would have many of the same hurdles.

ChatMaster (May 20, 2009 2:48:44 PM)
maryR, do you have any follow-up questions to the doctors' responses?

maryR (May 20, 2009 2:48:51 PM)
Thank you. I would like to know your approach to patients who present with atypical ALS.

dr-benatar (May 20, 2009 2:49:02 PM)
A few additional thoughts. We need better tools for preclinical testing of drugs (i.e. how do we decide which drugs are worthy of testing in humans). Also, a difficulty that is quite independent of money is the need for patients with ALS to be willing and able to participate in research studies.

dr-miller (May 20, 2009 2:49:46 PM)
As well as a good biomarkers. For example, a good test that predicts ALS or not as well as a test that we can use to follow the disease.

dr-benatar (May 20, 2009 2:49:55 PM)
MaryR, I think that this is a clinical question you're asking. If the disease preesnts atypically, then we need to be extra careful that the diagnosis is correct. This means going to extra lengths to be sure that we're not missing some other disease process (that looks like ALS) but which might be better treated.

dr-benatar (May 20, 2009 2:50:20 PM)
I agree with Dr Miller. New biomarkers are tremendously important. They might help us with earlier diagnosis and also with testing of potential new therapies.

scott (May 20, 2009 2:50:24 PM)
ChatMaster, I have a question.

maryR (May 20, 2009 2:51:04 PM)
Thank you. I have another question after Scott has his turn.

dr-miller (May 20, 2009 2:51:14 PM)
Agree with approach to aytypical ALS. Might depend how atypical. I have seen several cases of atypical ALS that were not ALS at all and required different diagnostic tests than would typically be performed for ALS.

ChatMaster (May 20, 2009 2:51:24 PM)
scott please proceed with your question

scott (May 20, 2009 2:51:35 PM)
To both Drs. - Could you please share your thoughts on how research in familial ALS can affect people with Sporadic ALS?

melissa (May 20, 2009 2:51:46 PM)
(This user has entered Research for Familial ALS)

melissa (May 20, 2009 2:52:02 PM)
I got booted and missed the answer to my questions :( Is there a copy of this chat?

ChatMaster (May 20, 2009 2:52:06 PM)
welcome back melissa!

melissa (May 20, 2009 2:52:25 PM)
Thanks, very disappointing, trying to get back into the room

ChatMaster (May 20, 2009 2:52:35 PM)
Yes there will be a transcript of this chat will be posted at www.mda.org/chat/transcripts.html in the next week

dr-miller (May 20, 2009 2:52:47 PM)
One of the striking findigs in studying familial ALS (SOD1, TDP-43, FUS/TLS) is that there is great overlap clinically. Overall, we believe that there are many common mechanisms that migh lead to new therapies for both.

dr-benatar (May 20, 2009 2:53:01 PM)
Another excellent question. I think that studying familial ALS may shed light on the sporadic form of the disease. Our Pre-fALS study of asymptomatic people who harbor a mtuation in the SOD1 gene is partially designed to identify environmental factors that might be responsible for early vs late onset of disease. Such factors might be relevant to the risk of sporadic als.

dr-benatar (May 20, 2009 2:53:31 PM)
Also, studying people presymptomatically (in our Pre-fALS) study offers an opportunity to identify early biomarkers of the disease process. Once developed, these might also be relevant to sporadic ALS.

dr-miller (May 20, 2009 2:53:53 PM)
If one looks at other neurodegenerative diseases, the proteins involved in familial disease are often linked to sporadic disease. This is trure for SOD1, with recent studies showing involvement of this protein in sporadic ALS.

ChatMaster (May 20, 2009 2:54:08 PM)
scott, does that answer your question?

scott (May 20, 2009 2:54:28 PM)
yes, thank you very much doctors.

melissa (May 20, 2009 2:54:30 PM)
:) I am in that study!

ChatMaster (May 20, 2009 2:54:54 PM)
maryR, please proceed with your question!

dr-benatar (May 20, 2009 2:54:58 PM)
And we're delighted that you are. Thank you (and to others who are participating)

maryR (May 20, 2009 2:56:22 PM)
I have met several PALS who were initially diagnosed with Multifocal Motor Neuropathy without a conduction block, and who received IVIG for a period of months, with some initial improvement. Have you had any patients like those?

dr-benatar (May 20, 2009 2:57:23 PM)
This is always a difficult clinical problem when the presentation is one of purely lower motor neuron disease. When there are upper motor neuron findings, then multifocal motor neuropathy isn't really a diagnostic consideration. When the presentation is purely lower motor neuron, we often have a tendency to want to give a trial of IVIg in the hope that it will be effective.

ChatMaster (May 20, 2009 2:58:20 PM)
The Research for Familial ALS chat will end in 1 minute

dr-miller (May 20, 2009 2:58:22 PM)
I have treated a number of patients with clear multifocal motor neuropathy with excellent improvement with IVIG. We also consider IVIG in some lower motor neuron patients with ALS, though this has been less successful.

ChatMaster (May 20, 2009 2:58:38 PM)
maryR, has your question been fully answered?

ChatMaster (May 20, 2009 2:58:59 PM)
Transcripts of this chat will be posted at www.mda.org/chat/transcripts.html in the next week

dr-benatar (May 20, 2009 2:59:03 PM)
I want to thank everyone for joining this chat session.

ChatMaster (May 20, 2009 2:59:14 PM)
MDA has 4 Living with ALS chat groups that meet weekly. Please see our chat calendar at www.mda.org/chat/calendar.html for a listing of MDA Chat groups.

dr-miller (May 20, 2009 2:59:15 PM)
Thank you very much for the questions and interest

maryR (May 20, 2009 2:59:17 PM)
Thank you.

melissa (May 20, 2009 2:59:20 PM)
Bye Dr. Benatar! :))))

vickiC (May 20, 2009 2:59:20 PM)
Thanks Dr. Benatar and Dr. Miller

ChatMaster (May 20, 2009 2:59:27 PM)
Thank you Dr. Benatar and Dr. Miller for your participation in this experts chat. We look forward to having you again at an Experts chat!

dr-benatar (May 20, 2009 2:59:31 PM)
Bye all.

vickiC (May 20, 2009 2:59:39 PM)
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ChatMaster (May 20, 2009 2:59:39 PM)
Thank you all for your participation!

maryR (May 20, 2009 2:59:42 PM)
Goodbye.

ChatMaster (May 20, 2009 2:59:47 PM)
Chatters can click on the Rooms tab and double-click on MDA Foyer if you want to keep chatting.

scott (May 20, 2009 2:59:52 PM)
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dr-miller (May 20, 2009 2:59:56 PM)
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dr-benatar (May 20, 2009 2:59:58 PM)
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melissa (May 20, 2009 3:00:01 PM)
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ChatMaster (May 20, 2009 3:00:01 PM)
I am going to close the Research for Familial ALS chat now. Goodbye!

maryR (May 20, 2009 3:00:02 PM)
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Doug (May 20, 2009 3:00:24 PM)
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ChatMaster (May 20, 2009 3:00:47 PM)
The Research for Familial ALS chat is now coming to an end.