It's happening thanks to some formidable people with ALS and caregivers. They aren't waiting for a system that doesn't understand urgency.
Here is a posting from a gentleman with ALS on the forum (it requires registration to view) at als.net:
Rich Casey, the CEO of Neuraltus, mentioned that ALSA is considering funding an Expanded Access (Compassionate Use) program for NP001, in addition to its $1.5M grant for a new clinical trial. As you know, Phase II clinical trial of NP001 yielded promising results in 2012, though falling short of its effectiveness goals. However, post hoc analysis identified "responder" biomarkers. Neuraltus has been trying to secure more clinical-trial funding ever since. Now ALSA expressed interest in underwriting a Compassionate Use program for NP001, along with the trial. Neuraltus believes there is an immense interest in the ALS community for this idea, but would like to have concrete evidence. Having a ready list of potential participants would help this cause, demonstrating the unmet need to ALSA as the sponsor. If you are interested, send a letter to Neuraltus requesting compassionate use access to NP001. This would strengthen Neuraltus's hand in dealing with the FDA as well as ALSA. We also should emphasize that the drug should not be withdrawn from those it helps, to prevent " crashes", like those experienced by Persevering and others during the wash-out period of the 2012 trial.
To: Rich Casey email@example.com
Below is a bare-bones sample letter to sign and send, but it would be good include your personal information and why you need NP001.
Dear Mr. Rich Casey,
As a person with ALS I want to join the Compassionate Use program for NP001. I am aware that the upcoming clinical trial would have only a limited enrollment. A large Expanded Access arm would benefit ALS research and help PALS. It is also important that long-term access to the drug should be ensured for those participants that benefit from it. Sincerely,
There are a number of threads on the forum at www.patientslikeme.com that discuss NP001. I urge any PALS, CALS interested to read them. It definitely did something in some PALS in the Phase 2 trial. Some also crashed terribly when the drug was taken away.
Here is an article from the WSJ regarding one of the PALS who was in the last Phase 2 trial in 2012:
And finally, here is a powerful letter that one of the Phase 2 volunteers just wrote to Mr. Casey:
Dear Mr. Rich Casey,Certainly an expanded access program isn't ideal, but it is something. This potential therapy for some PALS (elevated IL-18 was cited as being characteristic in responders) has sat on the shelf for almost three years while the drug developer has not been able to raise the capital needed for a Phase 3 trial. The system is broken. Why scientists have not been more curious about what was happening with this drug is beyond me.
As a person with ALS I want to join the Compassionate Use program for NP001. I was in the NP001 Phase II trial and received the high dose. NP001 stopped my ALS dead in it's tracks during the time I was on it. I know first hand that NP001 is a safe and effective treatment for ALS patients. I am aware that the upcoming clinical trial would have only a limited enrollment. A large Expanded Access arm would benefit ALS research and help PALS. It is also important that long-term access to the drug should be ensured for those participants that benefit from it. Thank you for the opportunity to provide NP001 to the broader ALS community, I know it will save many lives.
People with ALS and their caregivers are taking matters into their own hands and trying to open up some options. They are demanding and getting attention.
I encourage all PALS, CALS who are at all interested in trying to email Mr. Casey now at firstname.lastname@example.org .