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