I wrote this in response to an email I received from an NB Mom who's child is NED (no evidience of disease):
Shirley - I don't know who you are but I'm so thankful to have received this Happy New Year email from you. My son, Max 5yo, just recurred with NB and we are facing the horrible reality of how few kids survive this damn thing. I can't tell you how happy I am to read that your son is alive and well. I wish I could hear from more parents in your shoes! Thank you so much for the great news.
She responded to me:
Dear Melissa, it was good to hear back from you. You don't know me because I don't post that often any more -- the details of Simon's treatment (98-01) are a bit out of date for those now in treatment. However, I decided to send my holiday e-mail to the newer members of the listserv in addition to the old-timers I'm close to. I was a bit uncertain about doing that, but I do want people like you to know I am available as a resource if needed.
There are many parents like us whose children are NED -- but most such parents choose to depart the nb world and try to live a normal life (who can blame them). I think there are only a few who have stayed on the listserv whose children are ahead of Simon. I know there is at least one parent of a long-time relapse survivor on the list. I feel it is important for those in the battle to know that there are many of us out here who so far are staying one step ahead of this beast.
I took a look at your website and enjoyed seeing the beautiful pictures of Max. I see you are a pal of neil and sam's! I have spoken several times by phone with neil, what a good guy. The picture of Sam and Max is priceless!
Neil would no doubt tell you with a chuckle that I am a biased fan of the mskcc nb team -- Simon did his entire treatment at mskcc, so you can understand we probably are a bit biased!! We do believe that the 3F8 antibodies and the hot antibodies are the main reason simon is still walking around almost 9 years from dx. We are still very close to the nb team and try stay abreast of what is going on there. In fact, I met up with Dr. Cheung about a month ago and was incredibly uplifted by his firm conviction that he IS going to save some relapsed children. We have seen tremendous progress in the past few years and I do feel a breakthrough is right on the horizon. When Dr. C says there is hope, I believe him.
We live about 30 mins away from mskcc by subway. SO, if you ever come this way, or if you ever have any questions about mskcc, please just drop me a line. Alternatively, maybe our paths will cross at the NB conference this summer. I've never been before, but I hear it's an incredible gathering, so I'm going to try to make it.
Mainly I want you to know that Max and your family are in my prayers. I hope this current treatment will do what is needed!! SENDING HOPE!! Very best regards, Shirley Staples
Sunday, January 7, 2007
Tuesday, January 2, 2007
NB as a Business
A reply on ACOR from fellow parent, Neil Hutchinson, who's boy Sam is in treatment with Max here in San Diego for recurred NB. This guy is really on the ball.
Well, I think we should treat NB as a business and put together a business presentation for:
- the myth of competing trials (to overcome the thought that "we can't have too many trials open or we'll never fill our trial" - good trials accrue patients :)
- why it makes good business sense to put drugs into trial for neuroblastoma (Bayer, the manufacturer of nifurtimox may get a 2nd indication for this drug which could translate into a worldwide market; how many kids would this be, what's the money involved)
- trial ratings (I think parents should develop a rating system for trial design; positive aspects should be intra-patient dose escalation, allowing kids who have had the drug before to get it again since they are often precluded, finding a way for kids that have limitations w/respect to platelets to have access to drugs). I think we can also use this to change the mindsets of the current NB doctors to design their trials in a more patient friendly manner.
- then we contact the drug companies that have drugs we would like to trial. One company is Hoffman-LaRoche which has a drug called nutlin. If you search the ANR abstracts (and it's probably on Pubmed), nutlin is something that reverses the off- switch on the p53 tumor-suppressor. John London and I found out about this when talking to some oncs the other day. The company probably is just looking at bigger cancer markets but what if we call them and persuade them to get the drug into trial since I think much of the pre-clinical work is done?
The whole tone would be one of collaboration and positivity (I live a mile from Sea World and it's the old "how we train Shamu"). I think so much of what we have to overcome is simply awareness: if a company knew that parents would support a trial, they may just me interested in accelerating things.
How Niturtimox Became an NB Study Drug
A Post and Reply on ACOR:
Hey Shannon,
Good to see you on the list!
A couple of us got emergency INDs from the FDA to get the drug for compassionate use. I was the first to get it for Penelope and I have to say I was frowned at and not helped by the "experts" at all. They said their was not enough proof, not enough testing, not enough blah blah blah. All I knew was that my daughter was going to die very shortly and I could no longer listen to their excuses for inaction.
I had to get approval from the FDA, the Center for Disease Control and Bayer (the manufacturer) with virtually no help from the medical community. In fact, all the "experts" did was tell me Penelope would die very shortly, that I should "let her die with dignity", and that she would be very symptomatic of her disease in "weeks if not days". I lobbied Washington (Nancy Pelosi's office) and Congressmen and women to put pressure on the regulators to release the drug on an expedited basis. I managed to get the drug within 10 days!!!
When the "experts" told me Penelope would die shortly and I should stop treatment that was in August. Well now we are in December and Penelope is here and battling. Over these past four months we have got to see her laugh and play and enjoy things like her family, Halloween, Christmas and many other wonderful moments. She is actually today feeling very well as she sits next to me bathing her toy dogs and cats!
And Nifurtimox helped save Penelope's life as she had a huge response when combined with topo/cytox. It lasted a good 2 1/2 months when it stopped working for Penelope but it let us bridge to high dose chemo and antibody therapy.
The only doctor who helped at all was Dr. Sholler. She is terrific in every way. She is the type of doctor that is the future for beating this disease.....young, aggressive, operating with a sense of urgency, wanting to get things into clinic before years of perfecting research are complete because kids today have no real options for cure. From the time Neil Hutchison and I discussed the abstract on Nifurtimox that was part of the ANR conference until the time it actually became a clinical trial was about THREE MONTHS! So the next time the docs complain about how hard it is to get something into a trial, how many roadblocks there are and how many years it might take, just remind them that a couple of parents and a great doctor in Vermont got this trial going in three months. My guess is you will hear stunned silence in response.
Anyway, I just think it is so important for our voices to be heard by the "experts". Lets face it, their track record is horrible when after so many years hardly any kid ever survives relapse. We have to do better for these kids, give them more and better options ASAP and part of it is insisting these "experts" move with more urgency to save the kids alive today....they need to operate at a speed and aggressiveness they would move at if it was their own kid who had relapsed NB. And they are not doing that today despite what they say.
John
-----Original Message-----
From: The Neuroblastoma (Cancer) Online Support Group [mailto:N-BLASTOMA@LISTSERV.ACOR.ORG] On Behalf Of Shannon Snow
Sent: Saturday, December 30, 2006 11:13 PM
To: N-BLASTOMA@LISTSERV.ACOR.ORG
Subject: [N-BLAST] Med: Nifurtimox and clinical trials
Hey gang.
I don't have to constantly study any more. I don't have to have a plan B, or even a plan A. Though my relief from studying, worry, and fear is no consolation, Nick's relief from pain is my comfort.
I've been seeing a lot of talk here about this drug: Nifurtimox, and it has my curiosity tickled, so I thought I would check it out. The first thing I read about it was this:
"Nifurtimox (nye-FER-tim-oks) belongs to a group of medicines called antiprotozoals. It is used to treat an infection called American trypanosomiasis (Chagas' disease). Nifurtimox works by killing the protozoa (tiny, one-celled animals) that cause the infection."
It amazes me that anyone would think of trying this drug on Neuroblastoma. It appears to be treatment for infection causing bugs.
Then I learned that a girl with refractory NB got the Chagas' disease and was treated with the Nifurtimox, and as a by-the-way, went into clinical remission. !? It reminds me of the heart medication that ended up growing hair on bald men as a by-the-way. As Tevia's gramma would say, "GO DR. SHOLLER GO."
My hat is off to all the parents and children, and adults with this disease, willing to enter clinical trials. Standard therapy for NB just doesn't work well enough! Clinical trials are where we are going to find better treatments and even possibly the cure. I hate that trials seem so slow, and the rules are so persnickety, but for now, it's the only way to go forward finding the cure.
Shannon: mom to Nick Snow www.nicksnow.com
Hey Shannon,
Good to see you on the list!
A couple of us got emergency INDs from the FDA to get the drug for compassionate use. I was the first to get it for Penelope and I have to say I was frowned at and not helped by the "experts" at all. They said their was not enough proof, not enough testing, not enough blah blah blah. All I knew was that my daughter was going to die very shortly and I could no longer listen to their excuses for inaction.
I had to get approval from the FDA, the Center for Disease Control and Bayer (the manufacturer) with virtually no help from the medical community. In fact, all the "experts" did was tell me Penelope would die very shortly, that I should "let her die with dignity", and that she would be very symptomatic of her disease in "weeks if not days". I lobbied Washington (Nancy Pelosi's office) and Congressmen and women to put pressure on the regulators to release the drug on an expedited basis. I managed to get the drug within 10 days!!!
When the "experts" told me Penelope would die shortly and I should stop treatment that was in August. Well now we are in December and Penelope is here and battling. Over these past four months we have got to see her laugh and play and enjoy things like her family, Halloween, Christmas and many other wonderful moments. She is actually today feeling very well as she sits next to me bathing her toy dogs and cats!
And Nifurtimox helped save Penelope's life as she had a huge response when combined with topo/cytox. It lasted a good 2 1/2 months when it stopped working for Penelope but it let us bridge to high dose chemo and antibody therapy.
The only doctor who helped at all was Dr. Sholler. She is terrific in every way. She is the type of doctor that is the future for beating this disease.....young, aggressive, operating with a sense of urgency, wanting to get things into clinic before years of perfecting research are complete because kids today have no real options for cure. From the time Neil Hutchison and I discussed the abstract on Nifurtimox that was part of the ANR conference until the time it actually became a clinical trial was about THREE MONTHS! So the next time the docs complain about how hard it is to get something into a trial, how many roadblocks there are and how many years it might take, just remind them that a couple of parents and a great doctor in Vermont got this trial going in three months. My guess is you will hear stunned silence in response.
Anyway, I just think it is so important for our voices to be heard by the "experts". Lets face it, their track record is horrible when after so many years hardly any kid ever survives relapse. We have to do better for these kids, give them more and better options ASAP and part of it is insisting these "experts" move with more urgency to save the kids alive today....they need to operate at a speed and aggressiveness they would move at if it was their own kid who had relapsed NB. And they are not doing that today despite what they say.
John
-----Original Message-----
From: The Neuroblastoma (Cancer) Online Support Group [mailto:N-BLASTOMA@LISTSERV.ACOR.ORG] On Behalf Of Shannon Snow
Sent: Saturday, December 30, 2006 11:13 PM
To: N-BLASTOMA@LISTSERV.ACOR.ORG
Subject: [N-BLAST] Med: Nifurtimox and clinical trials
Hey gang.
I don't have to constantly study any more. I don't have to have a plan B, or even a plan A. Though my relief from studying, worry, and fear is no consolation, Nick's relief from pain is my comfort.
I've been seeing a lot of talk here about this drug: Nifurtimox, and it has my curiosity tickled, so I thought I would check it out. The first thing I read about it was this:
"Nifurtimox (nye-FER-tim-oks) belongs to a group of medicines called antiprotozoals. It is used to treat an infection called American trypanosomiasis (Chagas' disease). Nifurtimox works by killing the protozoa (tiny, one-celled animals) that cause the infection."
It amazes me that anyone would think of trying this drug on Neuroblastoma. It appears to be treatment for infection causing bugs.
Then I learned that a girl with refractory NB got the Chagas' disease and was treated with the Nifurtimox, and as a by-the-way, went into clinical remission. !? It reminds me of the heart medication that ended up growing hair on bald men as a by-the-way. As Tevia's gramma would say, "GO DR. SHOLLER GO."
My hat is off to all the parents and children, and adults with this disease, willing to enter clinical trials. Standard therapy for NB just doesn't work well enough! Clinical trials are where we are going to find better treatments and even possibly the cure. I hate that trials seem so slow, and the rules are so persnickety, but for now, it's the only way to go forward finding the cure.
Shannon: mom to Nick Snow www.nicksnow.com
Cell Line Established
Another post to ACOR:
Just wanted to share great news! While we knew that the chance of establishing a new cell line was very small, we did not learn until this week from Dr. Reynolds that he only has about a 3% chance of getting neuroblastoma cells to grow! Dr. Maris told us that Los Angeles has better luck which is why it was done in LA instead of at CHOP. Now I believe it was nothing short of miraculous and I can't help but think our "mad scientist - little Christi" had a hand in this to actually work!
This morning's local newspaper has a huge article with many pictures about FU-NB2006. Although on the web there is only one pic (of Dr. Maris) below is the link to the story. We wish you all a Happy New Year and may the cure be discovered in 2007! Best wishes and much love to you all!!
http://www.advertiser-tribune.com/News/articles.asp?articleID=5702
Warmly,
the Thomas team Mom (Angela)
Angel Christi 5/1997 - 9/2006, forever 9
www.ChristiThomas.com
Just wanted to share great news! While we knew that the chance of establishing a new cell line was very small, we did not learn until this week from Dr. Reynolds that he only has about a 3% chance of getting neuroblastoma cells to grow! Dr. Maris told us that Los Angeles has better luck which is why it was done in LA instead of at CHOP. Now I believe it was nothing short of miraculous and I can't help but think our "mad scientist - little Christi" had a hand in this to actually work!
This morning's local newspaper has a huge article with many pictures about FU-NB2006. Although on the web there is only one pic (of Dr. Maris) below is the link to the story. We wish you all a Happy New Year and may the cure be discovered in 2007! Best wishes and much love to you all!!
http://www.advertiser-tribune.com/News/articles.asp?articleID=5702
Warmly,
the Thomas team Mom (Angela)
Angel Christi 5/1997 - 9/2006, forever 9
www.ChristiThomas.com
Parent Advocacy for NB
A post to www.ACOR.ogr from a fellow NB parent:
I think the idea of a parent advocacy group, well coordinated so that no one is working at cross purposes, is a fabulous idea. 99% of the leading nb docs just will not step outside their own self-made nb boxes. And our children are dying because of it.
Years ago I spoke with Professor Ronald Breslow, the developer of SAHA, and he was convinced that it would cure Bryan. Maybe he was right, maybe he wasn't, but we didn't get the chance to find out because of the archaic trial process. Adults first for several years, then MAYBE pediatrics. It's an outdated system and it needs to be changed. With the communication support of the internet and the research capabilities of parents like you & John I'm sure that a group of parents on a mission, well armed with facts & data, can have an impact.
I think the idea of a parent advocacy group, well coordinated so that no one is working at cross purposes, is a fabulous idea. 99% of the leading nb docs just will not step outside their own self-made nb boxes. And our children are dying because of it.
Years ago I spoke with Professor Ronald Breslow, the developer of SAHA, and he was convinced that it would cure Bryan. Maybe he was right, maybe he wasn't, but we didn't get the chance to find out because of the archaic trial process. Adults first for several years, then MAYBE pediatrics. It's an outdated system and it needs to be changed. With the communication support of the internet and the research capabilities of parents like you & John I'm sure that a group of parents on a mission, well armed with facts & data, can have an impact.
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