Frequently Asked Questions
Q. How effective is Neuralzeta?
A. On average, historically, Neuralzeta at least doubles life expectancy (or much more) for 84% of PALS who take it. No other drug has ever done this.
For 16%, the drug has no effect. It is generally accepted that 12% to 18% of cases are misdiagnosed (i.e., they may have Lyme disease, MS, MG etc. which have similar symptoms) - this may explain the 16% non-responders as they may have a different disease, not ALS.
Q. Why is Neuralzeta so effective compared to other ALS drugs?
A. Neuralzeta was developed following a breakthrough in understanding how ALS develops. Neuralzeta is the first and only drug specifically developed to treat ALS.
Unlike other drugs used for ALS, it is not a “repurposed” drug originally developed for another disease, or a combination of two existing drugs to make a drug that might work in ALS. Neuralzeta’s Mode of Action is based upon Lipid Raft Receptors. This is completely new, never before researched or trialled and a totally different modality to the Mode of Action of all other ALS drugs.
Q. In what way is this different to any other drug?
A. All existing drugs are based on the premise that ALS starts in the neurons or spinal cord or neuronal damage by oxidative stress or other factors, i.e. the problem lies between the brain and the muscle. None of these treatments have ever extended life more than a few months at best - if at all.
Some believe that stem cell transplant is the way forward, but this has never shown any notable success either.
Neuralzeta treats the neuromuscular junction, which is at the functional end of the neuronal system, i.e., the opposite way round to all the existing treatments.
Q. You say that Neuralzeta is the first and only drug specifically developed for ALS. What about Tofersen?
Toferson (Biogen`s Qalsody) is an antisense oligonucleotide, a small string of DNA bases that bind to RNA, i.e., it is a gene therapy, not a drug.
Only approved to treat SOD1 genetic cases (2% of PALS) - and at $14,000 per vial it not effective after a year of treatment.
Neuralzeta is a peptide specifically engineered to recognise a target on lipid raft receptors, i.e., it is a drug.
Effective in Sporadic, Familial, early, mid and late stage disease and all ages 29 to 85. When non responders (16%) are excluded, extends life threefold, and in some cases, indefinitely - and it is provided for free.
Q. Is it safe for humans?
A. As of Spring 2024, PALS have received more than 11,000 doses, 120 years of accumulated patient-treatment, there are more than 25,000 datum points. Treatment is ongoing and continues. No drug related adverse events have ever been reported.
Q. Who supplies it?
A. RC ALS Charity Group.
Q. What does it cost?
A. Nothing. Provided for free by the charity.
Q. This has been running since 2010 and developed through research, animal tests, then a safety study and now 10 years of treating PALS for free. Where does the money come from?
A. It has been financed by a single person as a charitable effort who gets nothing at all in return. Everyone connected with the charitable group is voluntary. Nobody has ever been paid. In fact, they pay their own expenses.
Q. What is the RC ALS Charity Group?
A. A small group of retired scientists / medical research doctors in the UK.
Q. What is the objective?
A. To get it approved by the FDA and made available to all PALS at cost price as a humanitarian endeavor.
Approval means that Neuralzeta will be covered by health insurance and / or National Health Service (NHS) in countries where an NHS is provided.
Q. Currently, how does a PALS get this drug for free?
A. He or she must provide a credible case history to us in writing, a confirmed diagnosis, a valid prescription, sign an informed patient consent, recent blood test and some other paperwork.
Q. Is there any cost at all for the PALS?
We harvest more monthly information on our PALS condition and their family situation than any clinic. This requires some clinical measuring instruments (e.g. blood pressure, oxygen saturation, glycaemic status, breath maximum flow, etc.) to complete the monthly monitoring report form. The cost of these items is approximately $200, and they last forever.
If the person cannot afford this, we arrange delivery to the PALS home at our cost.
Q. How is the monthly monitoring report form sent to the charity?
A. Attached to an email.
Q. How can a PALS be sure that their identity cannot become known?
A. The charity complies with all ethical and legal requirements by maintaining a locked database where every PALS is allocated a number.
Obviously, if a PALS wishes to speak publicly about their Neuralzeta treatment, they are free to do so.
Q. Is it a registered charity?
A. No. That is costly; we focus our very limited funds on providing Neuralzeta to PALS.
Charities cost money to register and maintain, e.g., auditing, annual reports, board of trustees etc.
As nobody has ever been asked for money there is no requirement to register a formal charity.
Q. Who discovered this?
A. One of our group. Irrelevant at this point; the FDA reviews drug safety and efficacy, not who made a discovery.
Q. Is Neuralzeta patented?
A. Yes.
Q What is the patent number?
A. That is confidential as Pharmaceutical companies would reverse engineer it and charge huge amounts of money – just as they do with all the other recently approved ALS drugs typically $12,000 per month. It is not a question that concerns the FDA regulators.
Q. What animal studies were conducted before it was used in man?
A. Independent FDA accredited contractor, Charles River Laboratories, conducted the approved safety study protocol in both Lagomorph and Murine species along with a complete histology investigation and report. No organs showed any anomaly, and no FD (fatal dose) could be demonstrated.
No animal suffered pain and could eat, socialise and roam as much as they wished. As it happens, everyone in the Charity Group are animal lovers. Compassion is not reserved for Humans.
Q. Was a Phase I (safety in man) study conducted?
A. Yes, in 2012.
Q. You say that Neuralzeta on average at least doubles life expectancy (or much more) for 84% of PALS who take it. What is the basis of your clinical data? i.e., how do you measure efficacy?
A. The physical decline rate in a patient from onset of symptoms before starting the drug compared to the rate of decline after starting the drug, observed by their neurologists. Everyone submits a comprehensive monitoring report every month. Accordingly, the Charitable Group maintains a far more accurate, and in more depth, patient clinical file than any ALS clinic.
Q. You say that Neuralzeta on average at least doubles life expectancy (or much more). How much more and how can you be sure?
When non responders are omitted from the data, efficacy statistics show a slowing of the progression by 68%, i.e., trebling of life expectancy (P=0.0001).
For some, life expectancy is infinite.
Q. What does P=0.0001 mean?
A. In medical science, the certainty (to be precise, the word `Probability` is used) of data or information is is measured by the "P Factor".
For example, if the question is who will win the 2032 US Presidential election, that is completely unknown, so the P Factor will be P=1.0000.
If the question is Abraham Lincoln dead - that is absolutely certain, so the P Factor will be P=0.0000.
Neuralzeta clinical efficacy has P=0.0001.
Q. What Efficacy end point are you using?
A. ALSFRS-R (ALS Functional Rating Scale – Revised).
Q. What is the ALSFRS-R? i.e., how is it scored?
A. A fit athlete would score 48 points maximum. A totally paralysed person would score 0 (Zero).
There are 12 physical parameters each with a score ranging from 4 to 0 (Zero).
12 multiplied by 4 = 48 which is the maximum score.
Q. Clinical trials often use other end points, such as grip strength or Forced Vital capacity. Why do you use ALSFRS-R?
A. We use the only end point measurement that matters - the overall physical ability status. If that does not change, life would continue indefinitely. In fact, we harvest more information every month from every patient than the standard 12 question ALS Functional Rating Scale - Revised.
We have an additional 7 parameters (making a total of 19) recorded every month. The charity has the most comprehensive, technically-advanced ALS data-harvesting tool ever developed. Compare this to ALS clinics which see their ALS patients on average once every four months while they are still capable of travelling to the clinic; after that, their data collection usually stops.
Surely self reporting cannot be trusted as a clinic report?
As the ALSFRS-R score is a subjective observation, it will likely be scored differently by any two different people. It is said that the PALS must be scored by a specialist medical professional - but this is not the case.
See the EVALUATION STUDY in the accuracy of on-line self reporting (Journal Amyotrophic Lateral Sclerosis Volume 13, 2012 - Issue 2)
In fact the precise ALSFRS-R score is not particularly relevant. What matters is the CHANGE in the score over exactly the same time periods.
Provided that the SAME person (e.g., the patient or a care giver) does the scoring every month over a long time, the submitted change in data is very accurate. Further, scoring by the same person over extended time ensures elimination of patient or carer bias as the submitted data would rapidly exceed the envelope of credibility.
As scoring the ALSFRS-R is observational and subjective, different people will score differently. In ALS clinics, frequently different people will be assessing the patient on different visits, accordingly, this is problematic, undermining the reliability of clinic assessments of disease progression.
Q. Do you only select the PALS that are more likely to do well – as is done when recruiting for clinical trials?
A. No. Anyone who asks for help is taken on a first come first served basis. There are no exclusion criteria at all. Therefore, the data is truly real-world experience. The only time we decline PALS is when we are short of funds. Before we accept a person, we need to be sure that we have sufficient resources to treat them for a long time.
Q. Clinical trials normally exclude PALS with ALSFRS-R scores below 33, which helps them get better results. Do you?
A. Our average patient STARTING Neuralzeta has an ALSFRS-R score of 33. This means the patient is already at a more advanced stage of the disease than in the typical clinical trial.
Persons with a score as low as 12 have been accepted by the charity
We accept anyone - repeat, anyone - if funding to treat them for a long time is available.
In fact we accept virtually all co-morbidities.
Q. What is the lowest ALSFRS-R starting score among those treated with Neuralzeta?
A. Two patients started on Neuralzeta when their ALSFRS-R score was already down to 12
Q. Generally, clinical trials exclude familial cases. Is Neuralzeta effective in familial cases?
A. Yes. We have a number of patients who have four PALS in the family.
Up to 9 years still being treated after all the other family members had passed from ALS.
Q. What is the longest time you have treated a PALS?
A. Nine years as at 2024.
Q. How do you know that a Neuralzeta patient really has ALS and not Lymes disease etc.?
A. We require a written confirmation of ALS from a recognized neurology clinic.
Q. Is Neuralzeta complicated to administer? Dose frequency?
A. Intra-muscular injection. Same as diabetics who inject insulin daily except that Neuralzeta is injected twice per week.
Q. Does the patient need to go to their doctor’s clinic to receive the dose?
A. No.
Q. Can care givers deliver the doses?
A. Care givers normally administer the dose.
Q. Can PALS inject themselves?
A. Yes. Also, optionally making it even easier, an automatic injector may be used if desired, where the device is just placed on the skin and triggered.
Q. Is an injection painful?
A. Momentary discomfort is a better description. However, if the patient wishes, Emla 5% cream (a topical analgesic available over the counter from a Pharmacy) can be spread on the site of the injection one hour prior to injecting - then the area becomes numb (unfeeling) for about one hour.
Q. Is it manufactured in a qualified facility?
A. It is manufactured in an FDA-accredited cGMP facility by a major producer of prescription drugs
Q. Does it need to be frozen at all times, like some costly Covid medications?
A. It must be kept in fridge at about +5 Centigrade.
Q. Does it have a reasonable shelf life?
A. A few days at ambient temperature (keep it is a cool place) is acceptable while travelling away from home but put in a fridge immediately on arrival.
At +5 C 6-7 weeks.
At -18 C many years.
Q. How do you get it to patients?
A. Usually by FedEx courier.
Q. Is it costly to manufacture?
A. Approximately $800 per month treatment. (Compare this to the recently FDA approved ALS drugs which cost $12,000 per month - and they do not extend life).
Q. Why hasn’t this been presented at ALS symposia or conferences?
A. We submitted scientific papers on the novel drug together with complete clinical efficacy data to the organizers of the 2017, 2018, and the 2019 International ALS Symposia. Every year, we were refused. After 3 years, we gave up submitting papers.
Q. How does Neuralzeta really compare to the other ALS drugs?
A. The charts HERE each show the clinical trial results from all the FDA approved ALS drugs.
Each chart has two lines, one for untreated decline (the placebo group) and the other representing treated decline.
Note that there are medications which relieve some of the symptoms (example: drooling), however they do not affect the progress of the condition.