Guest guest Posted February 1, 2006 Report Share Posted February 1, 2006 Hi, I went to the Trophos web site to read more. If this drug makes it to the actual treatment stage, will it make us stronger? Or will it just keep us from losing any more strength/function? I think I have an idea, but I'd like more info. Thanks for passing this along to us! Blessings, Holly > > From: Senior <jsenior@...> > Date: 2006/02/02 Thu AM 12:14:27 GST > > Subject: Drug research news > > Quote Link to comment Share on other sites More sharing options...
Guest guest Posted February 1, 2006 Report Share Posted February 1, 2006 Their website isn't updated very often, but the big PDF file on Tro19622 is very informative. Here's a quote: ---- Q: What will be TRO19622's potential benefit to patients? A: In patients suffering from a neurodegenrative disease, specific classes of neurons become dysfunctional, progressively lose their capacity to maintain their synapses and axons, and finally undergo cell death. The stresses tha trigger this slow degenerative process are multiple, and differ from disease to disease. For the patient suffering from a motor neuon disease, ALS or SMA, TRO19622 is expected to preserve existing neuronal function, by delaying or even stopping further progression of the disease. Furthermore, by improving neuronal survival and promoting regeneration, TRO19622 may even lead to recovery in function. ---- So, basically the answer is that it should prevent further loss of function, which would be a huge step forward, though not a cure. But there's also that bit about possible recovery of function. That would be a sensational breakthrough. They don't know if it will happen, but I find it encouraging that they even mentioned that possibility at all. Let's assume the drug " only " prevents further deterioration. As Mitch always says, a really effective treatment for SMA is likely to involve multiple drugs. TRO19622: Does not increase SMN protein, but protects neurons from death. Valproic acid: Increases SMN protein, but does not prevent neuronal death. I think the most exciting mid-term prospect is what happens when you put a neuroprotective drug like TRO19622 together with a drug like VPA (or phenylbutyrate, or small molecules). Tokyo, Japan 2006/2/2, hollyrp@... <hollyrp@...>: > Hi, > I went to the Trophos web site to read more. If this drug makes it to the > actual treatment stage, will it make us stronger? Or will it just keep us > from losing any more strength/function? I think I have an idea, but I'd > like more info. Thanks for passing this along to us! > Blessings, > Holly > Quote Link to comment Share on other sites More sharing options...
Guest guest Posted February 1, 2006 Report Share Posted February 1, 2006 Greetings! This is refreshing news that surely lifts our spirits. We are praying hard day by day, that a cure for this motor neuron disease be discovered the soonest time possible. Bless you for sharing with us this piece of good news! Hugs, ming2sy > > Anyone subscribed to Google news alerts for SMA will have got this > already, but I think it's worth posting. In all the excitement over > valproic acid etc., not many people have noticed this little company > in France, which has been quietly developing a drug that protects > motor neurons from neurodegenerative disorders. I have been waiting > for them to announce their Phase I trial results, and here they are > (below). Phase I measures safety and tolerability of course, not > effectiveness, but this is a big step forward. This drug does not have > anything to do with the SMN gene or protein, AFAIK. It works on the > neurons directly, which is why it has potential for ALS as well as > SMA. > > I notice the Phase II/III trial is for ALS only. My guess is that > that's because (1) there are more ALS patients, and (2) neuronal loss > is much quicker in ALS, and therefore the effects of the drug will be > easier to measure. Perhaps an SMA trial will be announced later. > > ----- > http://www.bio-itworld.com/newsitems/2006/february/02-01-06-news- briefs > > Trophos has announced the successful completion of Phase I clinical > trials for its lead compound TRO19622. TRO19622 is representative of a > class of novel compounds identified using the proprietary neuronal > cell screening platform developed at Trophos. In preclinical studies, > these compounds have been demonstrated to promote the survival of a > wide range of neurons in vitro, as well as in several in vivo models > of neurodegenerative diseases. The clinical trials were conducted in > France and involved single and multiple dose studies on healthy adult > subjects. TRO19622 was demonstrated to: i) be well tolerated; ii) have > achieved the effective clinical dose via the oral route and, iii) have > an excellent safety profile.¥Ï The successful completion of Phase I > means that Trophos now expects to initiate a pivotal Phase II/III > clinical trial for the ALS indication in Q4 2006. The trial will be > conducted in the USA and Europe. TRO19622 has already been granted > orphan drug designation status for the treatment of Amyotrophic > Lateral Sclerosis (ALS) in the USA, and for the treatment of Spinal > Muscular Atrophy (SMA) in the EU. Website: www.trophos.com. > > , > Tokyo, Japan > Quote Link to comment Share on other sites More sharing options...
Guest guest Posted February 2, 2006 Report Share Posted February 2, 2006 It's too bad they aren't doing phase I trials with SMA as well. Knowing that the FDA approves drugs for use on a per disease (or condition) basis, waiting for results with ALS will likely delay the use in SMA should positive results occur. They will have to go through all the clinical trials with SMA as well, but they won't occur until after ALS, or so it appears. Also, ...what do you mean by " small molecules " ? I don't think I've ever heard that term. Jay Re: Drug research news Their website isn't updated very often, but the big PDF file on Tro19622 is very informative. Here's a quote: ---- Q: What will be TRO19622's potential benefit to patients? A: In patients suffering from a neurodegenrative disease, specific classes of neurons become dysfunctional, progressively lose their capacity to maintain their synapses and axons, and finally undergo cell death. The stresses tha trigger this slow degenerative process are multiple, and differ from disease to disease. For the patient suffering from a motor neuon disease, ALS or SMA, TRO19622 is expected to preserve existing neuronal function, by delaying or even stopping further progression of the disease. Furthermore, by improving neuronal survival and promoting regeneration, TRO19622 may even lead to recovery in function. ---- So, basically the answer is that it should prevent further loss of function, which would be a huge step forward, though not a cure. But there's also that bit about possible recovery of function. That would be a sensational breakthrough. They don't know if it will happen, but I find it encouraging that they even mentioned that possibility at all. Let's assume the drug " only " prevents further deterioration. As Mitch always says, a really effective treatment for SMA is likely to involve multiple drugs. TRO19622: Does not increase SMN protein, but protects neurons from death. Valproic acid: Increases SMN protein, but does not prevent neuronal death. I think the most exciting mid-term prospect is what happens when you put a neuroprotective drug like TRO19622 together with a drug like VPA (or phenylbutyrate, or small molecules). Tokyo, Japan 2006/2/2, hollyrp@... <hollyrp@...>: > Hi, > I went to the Trophos web site to read more. If this drug makes it to the > actual treatment stage, will it make us stronger? Or will it just keep us > from losing any more strength/function? I think I have an idea, but I'd > like more info. Thanks for passing this along to us! > Blessings, > Holly > A FEW RULES * The list members come from many backgrounds, ages and beliefs So all members most be tolerant and respectful to all members. * Some adult language and topics (like sexual health, swearing..) may occur occasionally in emails. Over use of inappropriate language will not be allowed. If your under 16 ask your parents/gaurdian before you join the list. * No SPAMMING or sending numerous emails unrelated to the topics of spinal muscular atrophy, health, and the daily issues of the disabled. Post message: Subscribe: -subscribe Unsubscribe: -unsubscribe List manager: (Sexy Mature Artist) Email: Esma1999@... oogroups.com List manager: (Sexy Mature Artist) Email: Esma1999@... Quote Link to comment Share on other sites More sharing options...
Guest guest Posted February 2, 2006 Report Share Posted February 2, 2006 Fantastic. The more avenues of attack researched the better. Mitch --- Senior <jsenior@...> wrote: > Anyone subscribed to Google news alerts for SMA will > have got this > already, but I think it's worth posting. In all the > excitement over > valproic acid etc., not many people have noticed > this little company > in France, which has been quietly developing a drug > that protects > motor neurons from neurodegenerative disorders. I > have been waiting > for them to announce their Phase I trial results, > and here they are > (below). Phase I measures safety and tolerability of > course, not > effectiveness, but this is a big step forward. This > drug does not have > anything to do with the SMN gene or protein, AFAIK. > It works on the > neurons directly, which is why it has potential for > ALS as well as > SMA. > > I notice the Phase II/III trial is for ALS only. My > guess is that > that's because (1) there are more ALS patients, and > (2) neuronal loss > is much quicker in ALS, and therefore the effects of > the drug will be > easier to measure. Perhaps an SMA trial will be > announced later. > > ----- > http://www.bio-itworld.com/newsitems/2006/february/02-01-06-news-briefs > > Trophos has announced the successful completion of > Phase I clinical > trials for its lead compound TRO19622. TRO19622 is > representative of a > class of novel compounds identified using the > proprietary neuronal > cell screening platform developed at Trophos. In > preclinical studies, > these compounds have been demonstrated to promote > the survival of a > wide range of neurons in vitro, as well as in > several in vivo models > of neurodegenerative diseases. The clinical trials > were conducted in > France and involved single and multiple dose studies > on healthy adult > subjects. TRO19622 was demonstrated to: i) be well > tolerated; ii) have > achieved the effective clinical dose via the oral > route and, iii) have > an excellent safety profile.¥Ï The successful > completion of Phase I > means that Trophos now expects to initiate a pivotal > Phase II/III > clinical trial for the ALS indication in Q4 2006. > The trial will be > conducted in the USA and Europe. TRO19622 has > already been granted > orphan drug designation status for the treatment of > Amyotrophic > Lateral Sclerosis (ALS) in the USA, and for the > treatment of Spinal > Muscular Atrophy (SMA) in the EU. Website: > www.trophos.com. > > , > Tokyo, Japan > __________________________________________________ Quote Link to comment Share on other sites More sharing options...
Guest guest Posted February 2, 2006 Report Share Posted February 2, 2006 The Phase I trial is already complete. The participants were adults with neither ALS nor SMA, because it was just to check human safety and tolerability. I don't think they need to repeat the Phase I trial now that tolerability and safety have been established. I did some serious searching, and I read on the forum of the French SMA organization that Trophos is planning to undertake a trial in adults with SMA " in parallel " with the ALS trial. That information was posted by an employee of the organization, who had contacted Trophos to check. I don't know if " in parallel " means they will start at exactly the same time, but it certainly does not mean that they will wait for the ALS trial to end before starting the SMA one. It's also interesting that they chose SMA adults for the trial, assuming the information I read was correct. TRO19622 is now designated as an orphan drug for ALS in the US and for SMA in Europe, which seems strange. Perhaps if FSMA or one of the other groups takes an interest, they could set up an SMA trial in the US. I don't entirely understand what " small molecules " are myself, apart from, well, molecules that are small. From what I've read, they are synthetic molecules that are being developed to increase levels of functional SMN protein, using something call " anti-sense " technology. The most recent abstract showed they were capable of increasing SMN levels significantly in He-La cells. Here's the link: http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve & db=pubmed & dopt=Abstra\ ct & list_uids=16226920 & query_hl=1 Here's another article on small molecules: http://www.fightsma.com/news/lay_research.html#adrian The deCode/FSMA project is also working on small molecules in an effort to create a brand new drug that increases functional SMN protein. HTH, 2006/2/2, jondus@... <jondus@...>: > It's too bad they aren't doing phase I trials with SMA as well. Knowing > that the FDA approves drugs for use on a per disease (or condition) > basis, waiting for results with ALS will likely delay the use in SMA > should positive results occur. They will have to go through all the > clinical trials with SMA as well, but they won't occur until after ALS, > or so it appears. > > Also, ...what do you mean by " small molecules " ? I don't think I've > ever heard that term. > > Jay > > Re: Drug research news > > > Their website isn't updated very often, but the big PDF file on > Tro19622 is very informative. Here's a quote: > ---- > Q: What will be TRO19622's potential benefit to patients? > A: In patients suffering from a neurodegenrative disease, specific > classes of neurons become dysfunctional, progressively lose their > capacity to maintain their synapses and axons, and finally undergo > cell death. The stresses tha trigger this slow degenerative process > are multiple, and differ from disease to disease. > For the patient suffering from a motor neuon disease, ALS or SMA, > TRO19622 is expected to preserve existing neuronal function, by > delaying or even stopping further progression of the disease. > Furthermore, by improving neuronal survival and promoting > regeneration, TRO19622 may even lead to recovery in function. > ---- > > So, basically the answer is that it should prevent further loss of > function, which would be a huge step forward, though not a cure. But > there's also that bit about possible recovery of function. That would > be a sensational breakthrough. They don't know if it will happen, but > I find it encouraging that they even mentioned that possibility at > all. > > Let's assume the drug " only " prevents further deterioration. As Mitch > always says, a really effective treatment for SMA is likely to involve > multiple drugs. > TRO19622: Does not increase SMN protein, but protects neurons from > death. > Valproic acid: Increases SMN protein, but does not prevent neuronal > death. > > I think the most exciting mid-term prospect is what happens when you > put a neuroprotective drug like TRO19622 together with a drug like VPA > (or phenylbutyrate, or small molecules). > > > Tokyo, Japan > > > 2006/2/2, hollyrp@... <hollyrp@...>: > > Hi, > > I went to the Trophos web site to read more. If this drug makes it > to the > > actual treatment stage, will it make us stronger? Or will it just > keep us > > from losing any more strength/function? I think I have an idea, but > I'd > > like more info. Thanks for passing this along to us! > > Blessings, > > Holly > > > > > A FEW RULES > > * The list members come from many backgrounds, ages and beliefs So all > members most be tolerant and respectful to all members. > > * Some adult language and topics (like sexual health, swearing..) may > occur occasionally in emails. Over use of inappropriate language will > not be allowed. If your under 16 ask your parents/gaurdian before you > join the list. > > * No SPAMMING or sending numerous emails unrelated to the topics of > spinal muscular atrophy, health, and the daily issues of the disabled. > > Post message: > Subscribe: -subscribe > Unsubscribe: -unsubscribe > > List manager: (Sexy Mature Artist) Email: Esma1999@... > > > > > oogroups.com > > List manager: (Sexy Mature Artist) Email: Esma1999@... > > > > > > Quote Link to comment Share on other sites More sharing options...
Guest guest Posted February 2, 2006 Report Share Posted February 2, 2006 From all I've read - " small molecule " therapy is interchanged with drug therapy & vice versa. Small molecules being able to cross the blood/brain barrier, etc. --- Senior <jsenior@...> wrote: > The Phase I trial is already complete. The > participants were adults > with neither ALS nor SMA, because it was just to > check human safety > and tolerability. I don't think they need to repeat > the Phase I trial > now that tolerability and safety have been > established. > > I did some serious searching, and I read on the > forum of the French > SMA organization that Trophos is planning to > undertake a trial in > adults with SMA " in parallel " with the ALS trial. > That information was > posted by an employee of the organization, who had > contacted Trophos > to check. I don't know if " in parallel " means they > will start at > exactly the same time, but it certainly does not > mean that they will > wait for the ALS trial to end before starting the > SMA one. It's also > interesting that they chose SMA adults for the > trial, assuming the > information I read was correct. > > TRO19622 is now designated as an orphan drug for ALS > in the US and for > SMA in Europe, which seems strange. Perhaps if FSMA > or one of the > other groups takes an interest, they could set up an > SMA trial in the > US. > > I don't entirely understand what " small molecules " > are myself, apart > from, well, molecules that are small. From what I've > read, they are > synthetic molecules that are being developed to > increase levels of > functional SMN protein, using something call > " anti-sense " technology. > The most recent abstract showed they were capable of > increasing SMN > levels significantly in He-La cells. > Here's the link: > http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve & db=pubmed & dopt=Abstra\ ct & list_uids=16226920 & query_hl=1 > > Here's another article on small molecules: > http://www.fightsma.com/news/lay_research.html#adrian > > The deCode/FSMA project is also working on small > molecules in an > effort to create a brand new drug that increases > functional SMN > protein. > > HTH, > > > > > 2006/2/2, jondus@... <jondus@...>: > > It's too bad they aren't doing phase I trials > with SMA as well. Knowing > > that the FDA approves drugs for use on a per > disease (or condition) > > basis, waiting for results with ALS will likely > delay the use in SMA > > should positive results occur. They will have to > go through all the > > clinical trials with SMA as well, but they won't > occur until after ALS, > > or so it appears. > > > > Also, ...what do you mean by " small > molecules " ? I don't think I've > > ever heard that term. > > > > Jay > > > > Re: Drug research news > > > > > > Their website isn't updated very often, but the > big PDF file on > > Tro19622 is very informative. Here's a quote: > > ---- > > Q: What will be TRO19622's potential benefit to > patients? > > A: In patients suffering from a neurodegenrative > disease, specific > > classes of neurons become dysfunctional, > progressively lose their > > capacity to maintain their synapses and axons, > and finally undergo > > cell death. The stresses tha trigger this slow > degenerative process > > are multiple, and differ from disease to disease. > > For the patient suffering from a motor neuon > disease, ALS or SMA, > > TRO19622 is expected to preserve existing > neuronal function, by > > delaying or even stopping further progression of > the disease. > > Furthermore, by improving neuronal survival and > promoting > > regeneration, TRO19622 may even lead to recovery > in function. > > ---- > > > > So, basically the answer is that it should > prevent further loss of > > function, which would be a huge step forward, > though not a cure. But > > there's also that bit about possible recovery of > function. That would > > be a sensational breakthrough. They don't know if > it will happen, but > > I find it encouraging that they even mentioned > that possibility at > > all. > > > > Let's assume the drug " only " prevents further > deterioration. As Mitch > > always says, a really effective treatment for SMA > is likely to involve > > multiple drugs. > > TRO19622: Does not increase SMN protein, but > protects neurons from > > death. > > Valproic acid: Increases SMN protein, but does > not prevent neuronal > > death. > > > > I think the most exciting mid-term prospect is > what happens when you > > put a neuroprotective drug like TRO19622 together > with a drug like VPA > > (or phenylbutyrate, or small molecules). > > > > > > Tokyo, Japan > > > > > > 2006/2/2, hollyrp@... > <hollyrp@...>: > > > Hi, > > > I went to the Trophos web site to read more. > If this drug makes it > > to the > > > actual treatment stage, will it make us > stronger? Or will it just > > keep us > > > from losing any more strength/function? I > think I have an idea, but > > I'd > > > like more info. Thanks for passing this along > to us! > > > Blessings, > > > Holly > > > > > > > > > A FEW RULES > > > > * The list members come from many backgrounds, > ages and beliefs So all > > members most be tolerant and respectful to all > members. > > > > * Some adult language and topics (like sexual > health, swearing..) may > > occur occasionally in emails. Over use of > inappropriate language will > > not be allowed. If your under 16 ask your > parents/gaurdian before you > > join the list. > > > > * No SPAMMING or sending numerous emails > unrelated to the topics of > > spinal muscular atrophy, health, and the daily > issues of the disabled. > > > > Post message: > > Subscribe: > -subscribe > > Unsubscribe: > -unsubscribe > > > > List manager: (Sexy Mature Artist) Email: > Esma1999@... > > > > > === message truncated === __________________________________________________ Quote Link to comment Share on other sites More sharing options...
Guest guest Posted February 2, 2006 Report Share Posted February 2, 2006 In checking the Trophos web site - some really interesting additional info... Under " Trophos and TRO19622: FAQ's " " For the patient suffering from a motor neuron disease, ALS or SMA, TRO19622 is expected to preserver existing neuronal function, by delaying or even stopping further progressin of the disease. Furthermore, by improving neuronal survival and promoting regeneration, TRO19622 may even lead to a recovery in function. " Under " When will TRO10622 be available to patients? " " Trophos is actively devloping TRO19622 to treat patients with the motor neuron diseases ALS and SMA. Pharmaceutical development of a new chemical entity requires sufficent regulatory preclinical safety and toxicity studies to justify its use to treat patients. Today TRO19622 has completed precinical studies and has received authorisation for Phase 1 clinical trials which are underway to study the compound's safety and tolerance in healthy voulnteers. These studies will pave the way to selecting the dose to be used for long-term treatments of ALS patients in Phase 2 clinical trials. In the meantime, additional preclinical studies willb e performed to assure safety over the long term in children as well as in adults. Trophos is working actively with pediatric neruologists and the AFM to develop protocls for clinical trials in this SMA. Trophos is also considering clinical trials of TRO19622 in Huntington's Disease. " For more check out this website: www.trophos.com Pretty exciting stuff - Mitch --- <mongomustgolf@...> wrote: > From all I've read - " small molecule " therapy is > interchanged with drug therapy & vice versa. Small > molecules being able to cross the blood/brain > barrier, > etc. > > --- Senior <jsenior@...> wrote: > > > The Phase I trial is already complete. The > > participants were adults > > with neither ALS nor SMA, because it was just to > > check human safety > > and tolerability. I don't think they need to > repeat > > the Phase I trial > > now that tolerability and safety have been > > established. > > > > I did some serious searching, and I read on the > > forum of the French > > SMA organization that Trophos is planning to > > undertake a trial in > > adults with SMA " in parallel " with the ALS trial. > > That information was > > posted by an employee of the organization, who had > > contacted Trophos > > to check. I don't know if " in parallel " means they > > will start at > > exactly the same time, but it certainly does not > > mean that they will > > wait for the ALS trial to end before starting the > > SMA one. It's also > > interesting that they chose SMA adults for the > > trial, assuming the > > information I read was correct. > > > > TRO19622 is now designated as an orphan drug for > ALS > > in the US and for > > SMA in Europe, which seems strange. Perhaps if > FSMA > > or one of the > > other groups takes an interest, they could set up > an > > SMA trial in the > > US. > > > > I don't entirely understand what " small molecules " > > are myself, apart > > from, well, molecules that are small. From what > I've > > read, they are > > synthetic molecules that are being developed to > > increase levels of > > functional SMN protein, using something call > > " anti-sense " technology. > > The most recent abstract showed they were capable > of > > increasing SMN > > levels significantly in He-La cells. > > Here's the link: > > > http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve & db=pubmed & dopt=Abstra\ ct & list_uids=16226920 & query_hl=1 > > > > Here's another article on small molecules: > > > http://www.fightsma.com/news/lay_research.html#adrian > > > > The deCode/FSMA project is also working on small > > molecules in an > > effort to create a brand new drug that increases > > functional SMN > > protein. > > > > HTH, > > > > > > > > > > 2006/2/2, jondus@... <jondus@...>: > > > It's too bad they aren't doing phase I trials > > with SMA as well. Knowing > > > that the FDA approves drugs for use on a per > > disease (or condition) > > > basis, waiting for results with ALS will likely > > delay the use in SMA > > > should positive results occur. They will have > to > > go through all the > > > clinical trials with SMA as well, but they > won't > > occur until after ALS, > > > or so it appears. > > > > > > Also, ...what do you mean by " small > > molecules " ? I don't think I've > > > ever heard that term. > > > > > > Jay > > > > > > Re: Drug research news > > > > > > > > > Their website isn't updated very often, but the > > big PDF file on > > > Tro19622 is very informative. Here's a quote: > > > ---- > > > Q: What will be TRO19622's potential benefit to > > patients? > > > A: In patients suffering from a > neurodegenrative > > disease, specific > > > classes of neurons become dysfunctional, > > progressively lose their > > > capacity to maintain their synapses and axons, > > and finally undergo > > > cell death. The stresses tha trigger this slow > > degenerative process > > > are multiple, and differ from disease to > disease. > > > For the patient suffering from a motor neuon > > disease, ALS or SMA, > > > TRO19622 is expected to preserve existing > > neuronal function, by > > > delaying or even stopping further progression > of > > the disease. > > > Furthermore, by improving neuronal survival and > > promoting > > > regeneration, TRO19622 may even lead to > recovery > > in function. > > > ---- > > > > > > So, basically the answer is that it should > > prevent further loss of > > > function, which would be a huge step forward, > > though not a cure. But > > > there's also that bit about possible recovery > of > > function. That would > > > be a sensational breakthrough. They don't know > if > > it will happen, but > > > I find it encouraging that they even mentioned > > that possibility at > > > all. > > > > > > Let's assume the drug " only " prevents further > > deterioration. As Mitch > > > always says, a really effective treatment for > SMA > > is likely to involve > > > multiple drugs. > > > TRO19622: Does not increase SMN protein, but > > protects neurons from > > > death. > > > Valproic acid: Increases SMN protein, but does > > not prevent neuronal > > > death. > > > > > > I think the most exciting mid-term prospect is > > what happens when you > > > put a neuroprotective drug like TRO19622 > together > > with a drug like VPA > > > (or phenylbutyrate, or small molecules). > > > > > > > > > Tokyo, Japan > > > > > > > > > 2006/2/2, hollyrp@... > > <hollyrp@...>: > > > > Hi, > > > > I went to the Trophos web site to read more. > > > If this drug makes it > > > to the > > > > actual treatment stage, will it make us > > stronger? Or will it just > > > keep us > > > > from losing any more strength/function? I > > think I have an idea, but > > > I'd > > > > like more info. Thanks for passing this > along > > to us! > > > > Blessings, > > > > Holly > > > > > > > > > > > > > A FEW RULES > > > > === message truncated === __________________________________________________ Quote Link to comment Share on other sites More sharing options...
Guest guest Posted February 2, 2006 Report Share Posted February 2, 2006 2006/2/3, <mongomustgolf@...>: > " Trophos is actively devloping TRO19622 to treat > patients with the motor neuron diseases ALS and SMA. > Pharmaceutical development of a new chemical entity > requires sufficent regulatory preclinical safety and > toxicity studies to justify its use to treat patients. > Today TRO19622 has completed precinical studies and > has received authorisation for Phase 1 clinical trials > which are underway to study the compound's safety and > tolerance in healthy voulnteers. These studies will > pave the way to selecting the dose to be used for > long-term treatments of ALS patients in Phase 2 > clinical trials. In the meantime, additional > preclinical studies willb e performed to assure safety > over the long term in children as well as in adults. > Trophos is working actively with pediatric > neruologists and the AFM to develop protocls for > clinical trials in this SMA. Trophos is also > considering clinical trials of TRO19622 in > Huntington's Disease. " Just to reiterate: This FAQ is over a year old. The Phase 1 trials mentioned above have been successfully completed, and since a Phase 2 SMA trial appears to be in the pipeline, I guess they must have made some progress in developing protocols for SMA too. TRO19622 is the closest brand-new drug to approval, by my reckoning, because the deCode/FSMA project is still at the preclinical stage. Here's hoping the Phase II/III trials are completed successfully and speedily so that it gets into clinics around the world ASAP. With so many teams of dedicated researchers working on this from so many angles, the next few years are going to be very interesting indeed. Tokyo, Japan Quote Link to comment Share on other sites More sharing options...
Guest guest Posted February 3, 2006 Report Share Posted February 3, 2006 Holly: Based on what I read, they (the Trophos people) stated that the neuroprotective qualities of Trophos would 1) stop any additional strength loss (by affecting the mitochondria's ability to emit apoptic elements to cause cell death by interferring with the chemicals that signal it to do so - note the mitochondria is an organelle in every cell of our bodies - generally it acts as the power plant of each cell) and 2) due to the regenerative qualities seen with the drug, may provide for regaining lost strength/function. Of course, that's the hope and aim of all of the different approaches I'd assume. As per the Life of , always look on the bright side of life <whistling>...wouldn't that be fantastic, though!?! I kind of think of such possibilities along the lines of when I place one of my 2 or 3 $1 wagers on the lottery every year (whenever it gets to some ridiculous sum of money) - for a moment I think - wow, that would be nice - and then move on back to getting on with getting on. But, similar to the movie Dumb & Dumber - when Jim Carey's character is told bluntly that his chances of getting together with the woman of his dreams were 1 in a million, he excitedly replied " so your'e TELLING me there's a CHANCE! " This goes back to an old thread from over a year ago - glasses half full versus half empty I suppose. But damn would that be such a wonderful thing for so many people. Mitch --- hollyrp@... wrote: > Hi, > I went to the Trophos web site to read more. If > this drug makes it to the actual treatment stage, > will it make us stronger? Or will it just keep us > from losing any more strength/function? I think I > have an idea, but I'd like more info. Thanks for > passing this along to us! > Blessings, > Holly > > > > > > From: Senior <jsenior@...> > > Date: 2006/02/02 Thu AM 12:14:27 GST > > > > Subject: Drug research news > > > > > > > > [Non-text portions of this message have been > removed] > > __________________________________________________ Quote Link to comment Share on other sites More sharing options...
Guest guest Posted February 3, 2006 Report Share Posted February 3, 2006 Hey Mitch, as a 41yr old woman (shhh don't tell everyone my age, LOL) who has SMA type 2 and toasts with a " glass is half full with a 50% chance of brimming over " , I welcome any such medical trial info that may benefit adults living with SMA. I would luv to be on the list of SMA'ers who get to try this drug. Sure I have weakened alot since the age of 35; from 10 to 35 I've been steady ability-wise, but I still have a nerve response in the parts that have " let me down " . I have only two small areas that have ceased to function aka unresponsive aka dead ... my right thumb cannot extend out/upward from its 2nd joint; it moves away/back at the palm and at the tip. My left index finger has the same failure; the joint at the palm cannot raise my finger upward. The weakened areas; like my left elbow being unable to bend inward when fighting gravity vs in water, and neck, all have me feeling like its there, but when called upon to act it just flexes nothing ey. In my mind it says those MN's are struggling and not yet dead so any medicines that may aid in repairing them would be a huge benefit. I don't want to be stronger than I was, just put me back the way I was. Anything more would be gravy, LOL. Angie On 2006.02.03 09:02, wrote: > Holly: > > Based on what I read, they (the Trophos people) stated > that the neuroprotective qualities of Trophos would 1) > stop any additional strength loss (by affecting the > mitochondria's ability to emit apoptic elements to > cause cell death by interferring with the chemicals > that signal it to do so - note the mitochondria is an > organelle in every cell of our bodies - generally it > acts as the power plant of each cell) and 2) due to > the regenerative qualities seen with the drug, may > provide for regaining lost strength/function. Of > course, that's the hope and aim of all of the > different approaches I'd assume. As per the Life of > , always look on the bright side of life > <whistling>...wouldn't that be fantastic, though!?! > > I kind of think of such possibilities along the lines > of when I place one of my 2 or 3 $1 wagers on the > lottery every year (whenever it gets to some > ridiculous sum of money) - for a moment I think - wow, > that would be nice - and then move on back to getting > on with getting on. But, similar to the movie Dumb & > Dumber - when Jim Carey's character is told bluntly > that his chances of getting together with the woman of > his dreams were 1 in a million, he excitedly replied > " so your'e TELLING me there's a CHANCE! " This goes > back to an old thread from over a year ago - glasses > half full versus half empty I suppose. But damn would > that be such a wonderful thing for so many people. > > Mitch > > --- hollyrp@... wrote: > > Hi, > > I went to the Trophos web site to read more. If > > this drug makes it to the actual treatment stage, > > will it make us stronger? Or will it just keep us > > from losing any more strength/function? I think I > > have an idea, but I'd like more info. Thanks for > > passing this along to us! > > Blessings, > > Holly > > > > > From: Senior <jsenior@...> > > > Date: 2006/02/02 Thu AM 12:14:27 GST > > > > > > Subject: Drug research news > > > > [Non-text portions of this message have been > > removed] > > __________________________________________________ > Quote Link to comment Share on other sites More sharing options...
Guest guest Posted February 4, 2006 Report Share Posted February 4, 2006 What drug are you guys talking about? Valproic acid? Eternal Blessings, Meg. * (My Blog: http://360./pink-tulipameritech (DOT) net) Drug research news > > > > [Non-text portions of this message have been > > removed] > > __________________________________________________ > Quote Link to comment Share on other sites More sharing options...
Guest guest Posted February 5, 2006 Report Share Posted February 5, 2006 Not valproic acid. A new drug called TRO19622 (nice snappy name, huh?). This news release came out a few days ago: http://www.bio-itworld.com/newsitems/2006/february/02-01-06-news-briefs Trophos has announced the successful completion of Phase I clinical trials for its lead compound TRO19622. TRO19622 is representative of a class of novel compounds identified using the proprietary neuronal cell screening platform developed at Trophos. In preclinical studies, these compounds have been demonstrated to promote the survival of a wide range of neurons in vitro, as well as in several in vivo models of neurodegenerative diseases. The clinical trials were conducted in France and involved single and multiple dose studies on healthy adult subjects. TRO19622 was demonstrated to: i) be well tolerated; ii) have achieved the effective clinical dose via the oral route and, iii) have an excellent safety profile. The successful completion of Phase I means that Trophos now expects to initiate a pivotal Phase II/III clinical trial for the ALS indication in Q4 2006. The trial will be conducted in the USA and Europe. TRO19622 has already been granted orphan drug designation status for the treatment of Amyotrophic Lateral Sclerosis (ALS) in the USA, and for the treatment of Spinal Muscular Atrophy (SMA) in the EU. Website: www.trophos.com. , Tokyo, Japan 2006/2/5, Meg W. <pink-tulip@...>: > What drug are you guys talking about? Valproic > acid? > > Eternal Blessings, > > Meg. > > Quote Link to comment Share on other sites More sharing options...
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