Tag Archives: survival

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Cyborg Is As Cyborg Does – Reply All Interview

World’s First Fully-Functional Cyborg

Reply All Cyborg

I am the world’s first fully-functional cyborg! Need proof? My part in this Reply All podcast starts at 16:35.

This interview took place over about 3 weeks including one live telephone call and approximately 40 questions over email to which I replied both with text and individual MP3 files of the audio of my computer speaking each answer. It was a rather interesting experience and one that would certainly come in handy for any future interviews. Sruthi Pinnamaneni and Rick Kwan did a great job of stitching all of the questions and answers together to make a single coherent interview.

My desire was to demonstrate that life goes on after diagnosis and that there is still PLENTY that someone can still do despite full paralysis and being dependent on a ventilator. Hopefully other more newly-diagnosed PALS listening to the podcast can take a little inspiration to keep living and contributing your individual wonderful gifts to the world. Together, our voices are amplified and we can create the change we want to happen in the world.

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Neuraltus News!

Phase 2B Enrollment Open Now!

On Thursday, September 22, 2016, Neuraltus Pharmaceuticals announced the commencement of their long-anticipated Phase 2B for their lead candidate NP001. NP001 is a molecule that reverts macrophages (white blood cells) from an activated state where they hunt down and destroy pathogens and injured tissue to a calmer state where they nurture and protect other cells. I have blogged about NP001 extensively in the past. This trial follows up their Phase 2A trial which completed a few years ago. Unfortunately many of the participants in that trial are no longer with us, including my friends Rob Tison and Ben Harris with whom I launched the concurrent Oral Sodium Chlorite Project.

What It Is

This Phase 2B trial is to confirm the results of the post-hoc analysis of the responder class found in the Phase 2A. In that analysis, Neuraltus discovered that patients who were given the highest dose (2mg/kg body weight) and had elevated levels of pro-inflammatory proteins called IL-18 and C-reactive protein responded quite favorably to the drug. If this Phase 2B returns the expected results, NP001 would have a strong case for the same accelerated approval that FDA just granted for the Sarepta DMD drug eteplirsen. We could have the first new treatment since riluzole and the first truly effective one.

Sign Up Now!

I encourage all PALS to use the Clinical Trials tool on my website, provided by our friends at Antidote. It is very important that this trial is fully enrolled as soon as possible so that it is quickly completed and NP001 gets a shot at getting on the market. That is the best chance for it to get to ALL the PALS whose lives could be extended. We did it for the Phase 2A and can do it again for the Phase 2B.

This is a very exciting moment in the history of ALS.

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Guest Blog: Me!

SRG Research News – First A Little Background

As most of you know, I started SciOpen Research Group as a way for me to be able to fire actual bullets in the battle against ALS (well, actually metaphorical, but you get the idea). Our first project failed to extend life in the classic ALS mouse model so we retained the money raised to conduct the planned second part of that experiment. We had another project already in the research pipeline waiting to take the next step in development. For two years SRG was working on creating a novel molecule which would treat the desired pathway without becoming toxic like the reference molecule does at therapeutic doses. Suddenly we had the opportunity to collaborate with researchers already investigating the same pathway, albeit in different conditions (watch the video announcement), with their own library of candidate molecules.

Our collaboration’s first phase is to create a novel transgenic mouse species which represents a 100% drug efficacy in order to be a proof of concept. The project should run through the last half of 2016. As you will see below, a study was recently published which shows that SRG is definitely onto something. Our target protein is significantly elevated in human patients, and that targeting it brings positive results. The study is great indirect support of our project’s goal.

And now, the guest blog featuring myself!

Good News For Our Latest Project!

A recent report published in Science magazine strongly suggests that SciOpen Research Group is onto something with its currently ongoing study of necroptosis in ALS. Necroptosis is a “cousin” of apoptosis. In contrast to apoptosis, which happens regularly in the body, necroptosis is a form of programmed cell death which happens under inflammatory conditions and in which the components of the dead cell spill into the extracellular space. The spilling of the cellular components trigger a response in which immune cells are recruited to the area. Necroptosis is known to be a driver of both genetic ALS and sporadic ALS.

The subject study is not a direct support, in that it was looking at how the optineurin protein contributes to ALS. However, the results showed significant increase of the MLKL protein in human patients and that elimination of the RIPK3 protein or inhibition of RIPK1 had modest but nevertheless positive effects on survival of the SOD1 mice (along with positive biological evidence). This suggests that SRG is on the right track with its MLKL study. We believe that acting on MLKL will have a stronger effect without disrupting other cellular functions which depend on RIPK3 and/or RIPK31 (MLKL is involved only in necroptosis).

This study is YOUR study. It would not be position without your support. SciOpen Research Group is the world’s first fully functional “guerilla biotech”. We function only with your support and study pathways other research organizations either miss or ignore. And we can do it for much less because we are purely volunteer and have no overhead. 100% of your donations go directly to research. To support us you can make a tax-deductible donation (USA residents only) by going to our Donations page, purchase some SRG Gear, and/or go shopping on Amazon Smile and name SciOpen Research Group as your charity of choice (we are a registered and approved nonprofit under IRS 501c3). We work on ALS for you, the ALS Community, because we are part of the ALS Community. Help us continue our novel research into eradicating ALS.

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On Masitinib

I may have to revise my opinion of masitinib (which would not upset me in the slightest). Some of my readers may know that I have not been very optimistic about the probability of the drug being an effective treatment option for ALS. It’s been around for some time as a veterinary drug. But the company AB Science is developing it for ALS and other conditions.

Masitinib:

Preclinical information appears encouraging, although the study has a few issues. The rat model is not like the mouse model and is not very suitable for a survival study. The survival data are also very difficult to interpret due to the curious use of different numbers of animals in each cohort. I will defer to the opinions of my more statistics-inclined members (please feel free to comment!). The cellular data have a similar issue because they were taken in vitro rather than vivo. Nevertheless, it’s encouraging and we can hope for quick human trials.

The press release.

The study (open access!).

And the first USA patient gets approval for Compassionate Use!

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A Boost For Joost

“There are two things people take for granted every day: Time and health. When you lose those, then you wake up.”
— Joost van der Westhuizen

In 1994, Nelson Mandela became the first black President of South Africa. That same year, South Africa also hosted the Rugby World Cup. In that tournament, the home team, the Springboks, overcame considerably unfavorable odds and decades of international isolation (due to the government’s policy of apartheid) to win the Rugby World Cup. This is widely considered one of the greatest moments of South African sporting history and was the basis for the 2009 film Invictus. On that team was a young scrum-half named Joost van der Westhuizen.

In 2003 Joost retired from rugby. By then he was a superstar of South African rugby, having more caps than any other South African player. In 2011 the rugby world suffered a blow with the news that Joost had been diagnosed with ALS. Rather than retreat from the world, Joost decided to make a difference in the lives of people also coping with this dread diagnosis. He formed the J9 Foundation to educate the general public and medical practitioners about ALS, grow ALS research in South Africa, and to aid other South African PALS.

Joost’s story has been made into a documentary called “Glory Game“. In addition to the trailer, you can read about the movie here. The film has done well in South Africa and is now going to be shown first in Vancouver, British Columbia, on April 10, 2016 and in Los Angeles, California, on April 15, 2016. I urge all my friends in those areas to go see it. I have seen it and it’s simultaneously hilarious, upsetting, and uplifting. It shows the courage and determination which made Joost van der Westhuizen such a force on the rugby pitch. I am proud to call him and the Director of the film, Odette Schwegler, my friends.

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Liquid Hope

Usually a new hole in your stomach is bad news, often being either an ulcer or the result of some sort of violence. But for some, properly done, it’s a way to keep fed if the more normal method is no longer available. The question then is what to put through the hole. Obviously it would need to be in liquid form, but one can’t live just on beer alone (and I have tried…). Thankfully, there is a much better alternative.

Amyotrophic Lateral Sclerosis (ALS or Lou Gehrig’s Disease) is a disease outwardly characterized by loss of muscular strength. People coping with diagnosis (PALS – Person(s) with ALS) experience a progressive loss of muscular control as the nerves communicating brain commands to those muscles die. Eventually a specific muscle, the diaphragm, becomes weakened and breathing capacity is diminished. After progressive weakening of the diaphragm, breathing capacity diminishes to the point that blood carbon dioxide levels rise and the person dies of respiratory failure.

Although no two PALS experience the same progression pattern (I call us “Snowflakes From Hell”), usually another important – yet overlooked – muscle group is impacted before the diaphragm. This muscle group is commonly known as the tongue. When the back of the tongue loses strength, it can no longer efficiently create the pre-swallow bolus made up of the food being chewed and it can also no longer guard the airway against intrusion of food below the mouth before the epiglottis closes the trachea and opens the esophagus. This creates a choking situation with the increased possibility of aspiration pneumonia. Obviously both the choking and pneumonia represent substantial threats to life, especially for those with compromised respiratory function. Not only are choking and aspiration both hazards, but the lack of proper nutrition from not being able to eat is a dire handicap in the battle against ALS.

Fortunately for people in such a situation of lingual weakness, such as PALS in mid and late stages, medical science has created the PEG tube. This is a silicone rubber tube a little larger around than your typical drinking straw. It provides a direct route to the stomach and can dramatically lower one’s bar bill (because you don’t taste, you can switch from top-shelf to well brands…). PEG tubes are actually essential tools in “treatment” of ALS by keeping up optimum nutritional (including caloric) content.

Unfortunately, the “medical formulas” many patients are told to exclusively use – such as Nestle COMPLEAT – are based almost entirely on corn syrup for calories, which is the glucose base version of high fructose corn syrup (HFCS – the difference between the two is that HFCS is much sweeter, thus being attractive to processed food manufacturers). Basically, each can is a candy bar with a multivitamin in the middle. We have all heard the news about the perils of excessive sugar intake and how it, in the form of HFCS, is pervasive in processed foods. Eliminating HFCS and still eating just as much glucose sugar, especially as a sole source of calories, is equally harmful.

As I have previously blogged, using these medical formulas for any prolonged period is very risky in terms of your pancreas. I am an otherwise extremely healthy [formerly] athletic man with zero endocrine or any other confounding health issues. Nevertheless, using the traditional “medical formula” every day for two years put me in the ICU for a few days with a severe diabetic and hepatic crisis. I took control of my treatment plan and eliminated the corn syrup by switching from formula to real food (something which hospital dieticians tell patients to NOT do).

Clearly, the traditional enteral nutrition sources are not meant for long-term use. Until recently, most PALS died relatively shortly after diagnosis. This meant a few months of solely enteral nutrition weren’t going to pose a problem. But now, with better care and with adaptive technology better able to restore lost abilities, PALS are living longer post-diagnosis. I am one of those, going past 10 years post-diagnosis. Obviously better nutritional products are required. After taking personal control of my feeding, choosing fresh food blended together with a combination of healthy sources of fat, my blood glucose, liver, and kidney functions all normalized.

Not all PALS have either the ability to make their own blenderized food (is that really a word?) or have people who can make food for them which meets their nutritional and caloric needs. Just opening a can of soup is insufficient, as almost all processed food contains unacceptable levels of sodium, HFCS, etc. Further, PALS have certain requirements such as higher fat and calories. Getting those from improper sources can be hazardous. So what can we do?

Liquid Hope is here! This is a product created as a reaction to the terrible content of the traditional formula and the negative effect on health they can have. It is basically fresh food in a pouch that meets the needs of those with special dietary concerns (dairy free, gluten free, non-GMO, etc.). It’s a full meal replacement suitable for PALS as-is, but can be mixed with avocado, coconut oil, or other healthy fat source to boost calories for those PALS experiencing dramatic weight loss. My readers can learn more about the development of Liquid Hope here.

Even though I was getting mostly fresh food, I was interested in trying out Liquid Hope. The good people at Functional Formularies agreed to supply me a 7 day supply. From the very first meal I felt great! I was fully satisfied as if I had just had a good meal at our local vegetarian restaurant (I really miss their vegetarian lasagna). After 48 hours, I had more than my usual energy, I felt clear, and I was much more regular (constipation is a frequent issue for PALS). I only added a couple tablespoons of coconut oil along with some protein and vitamin additives, like I do all my meals. I was really sad to see the last pouch go down.

In my semi-expert opinion, Liquid Hope is a fantastic enteral nutrition solution and far superior to the usual cans of “medical formula”. I am greatly looking forward to switching fully to Liquid Hope for my nutritional needs. It’s now covered by Medicare*, Functional Formularies can help with the paperwork, and my first regular shipment is on its way!

I have been watching and talking about Liquid Hope on social media for a while. Frequent readers and friends know that I am extremely anti-“medical formula” and push patients to make fresh food for their enteral nutritional needs. Now that Liquid Hope is covered by Medicare* and is provided by a growing network of enteral nutrition providers, I call on all PALS to try it and use it. Let Nestle make snacks, not food staples. PALS have a serious medical condition requiring real nutrition. Take care of yourselves. Either blend fresh (not freshly-opened) food or use an organic and healthy product such as Liquid Hope.

* [So long as you aren’t in what’s known as a competitive bid area. The problem with being in one, in my opinion, is that the reimbursement to providers is based purely on lowest-price, keeping the better products from being available. I can explain the political aspects but that’s an entirely different subject not appropriate for this blog.]

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Scuttle Rebuttal

I am a little peeved at a new attack on the push for FDA’s Accelerated Approval Program for the ALS treatment called GM6 (known as GM604 in clinical trials). This attack basically follows the same line as the one from the ALS Association (and in fact incorporated it by reference). Both are sloppy and disingenuous in style and content. They attempt false comparison to other previously-failed trials without including the reasons for those previous failures and they also try a very clumsy smear by comparison to an act of intentional clinical fraud. I am disappointed that such comparisons are presented to a public looking for facts as well as hope.

First let me discuss the false comparison to previous trial:

  1. The comparisons are false primarily because the old trials exclusively used the ALS Functional Rating Scale (ALSFRS) which is a horrible metric by even the most generous interpretation. This is the only way a call for large trials can be justified, because the numb insensitivity of the ALSFRS (even the Revised version) requires such to make any kind of meaningful conclusion in a heterogeneous disease like ALS. However, the GM6 trial used several biomarker candidates and other objective clinical measurements as surrogate end-points (keep that phrase in mind) which correlated which the subjective end-points such as the ALSFRS-R. The biomarkers used were suggested and evaluated by Dr. Robert Bowser, a 2015 winner of the Sheila Essey Award for significant research contributions to fighting ALS.
  2. Brain-Derived Neurotrophic Factor (BDNF), one of the neurotrophic factors listed in Dr. Dickie’s post, doesn’t cross the Blood Brain Barrier (BBB). It’s almost impossible to get a therapeutic dose into the patient without an intrathecal infusion (directly into spine) using a pump over time. This has numerous obvious drawbacks. It’s also very unclear whether a single neurotrophic factor is useful in ALS, which encompasses a host of deficiencies.
  3. Cilliary Neurotrophic Factor (CNTF) does cross the BBB and early animal model tests indicated efficacy. However, two human trials in 1996 using subcutaneous delivery and intrathecal delivery as well as a review in 2004 revealed no efficacy in lower doses and serious side-effects at high doses. It’s also important to note that the animal data came well before the excellent work ALSTDI did characterizing the extreme difficulties in using that model. Any ALS mouse data released prior to 2009 (and any subsequent found to not strictly follow those guidelines) should be considered suspect. I have personal knowledge of the difficulty in using this model and the false-positive data which can result from improper use.
  4. Next, Insulin-like Growth Factor 1 (IGF-1) also crosses the BBB but has a very very short biological half-life, meaning it is broken down and excreted in a matter of hours. That makes therapeutic levels almost impossible to maintain. A form was created with a buffering agent attached to IGF-1 which roughly doubled the half-life but even that was woefully inadequate. Anyone who remembers the IPLEX debacle of a few years ago knows the story.

The comparisons to such single-target neurotrophic factors as BDNF, CNTF, and IGF-1 are therefore flawed in logic and fact. It is very disingenuous for Dr. Dickie to compare GM6 to them as GM6 is a master regulator and acts in 12 relevant pathways simultaneously. This information is already in the public domain freely available for anyone to look up.

Next, Dr. Dickie compares the GM6 results with those of the initial results of NP001 (actually he links to his own blog post where he addresses the anecdotal reports which came before the official results were published). What he failed to mention was the updated post-hoc analysis which showed a halt of disease progression in 27% of patients in the trial. Further, the analysis showed statistically significant evidence of two biomarkers which identified responding sub-groups. This is a tremendous achievement in ALS clinical trial history. Unfortunately the biomarkers aren’t the same in the GM6 trial so the comparison of the two is incomplete at best. The only real similarity is that both trials used biomarkers as secondary end-points. However, the GM6 trial used them in a way that didn’t require post-hoc analysis.

The comparison with lithium is especially troubling. First, it was far from “recent”, with patient excitement starting in 2007. You can find the data collected in the first PALS-led and created clinical trial which coincidentally also involved lithium. What both ALSA and MNDA failed to report about the study which started the excitement (“Fornai, et al., 2008”) was that the study essentially “cooked the books” by assigning PALS with slow progression of disease to the treatment group while putting the more standard PALS in the placebo group. This was revealed only after the paper was published. In the GM6 trial, run by two leading and internationally well-respected ALS researchers and clinicians, all participants were randomly assigned to receive either drug or placebo. The only way the comparison to the lithium study would be accurate is if the researchers deliberately placed certain patients in each cohort. Genervon merely supplied GM6. The trial was run and data collected by the two principle investigators (fancy name for doctors who run clinical trials). The analysis was then also done by a contract research facility. So any implication that Genervon somehow fabricated the data is false and besmirches the reputations of two prominent ALS doctors.

It must be noted and repeated that the standard FDA clinical trial practice is indeed extremely important in terms of protecting the public from the unscrupulous. The collection of objective scientific data is the foundation of good medical care. Nobody calling for the Accelerated Approval of GM6 disputes this. However, because ALS is so rapidly and uniformly fatal, we are calling for FDA to utilize the discretion it was granted in the face of an earlier similar crisis (the AIDS epidemic). Further, we call on everyone to realize that the GM6 trial used much more than the ALSFRS as a metric and thus smaller trial populations are much more statistically significant than before. The other end-points used in the Phase 2 are objective and not subject to placebo effect. Therefore, the indication of efficacy observed in the trial should be considered stronger than in previous trials which relied almost solely on the ALSFRS.

The Accelerated Approval Program was created to bridge the gap between the need for data and the urgent unmet needs of patients with rapidly-fatal diseases. The GM6 trial was unique in the strength of the preliminary efficacy signal, largely due to the objective biomarker end-points used. Just like the early days AIDS crisis, PALS have no meaningful treatment options and thus no hope. We want to use the very FDA program created to deal with that situation. We admit the need for more scientific data. But we don’t want to die while it’s collected.

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Condition Green

As many of you might already know, I was the late-stage PALS mentioned in the recent Genervon press release. I got interested in this drug some time ago, did some research on it and wrote a blog post about it. I had contacted the company, Genervon, to get information for my post. Thereafter, a dialogue was maintained regarding clinical trial status and future development plans. Being that I am a late stage PALS and still extremely active in awareness, advocacy, and science, they agreed to my request for compassionate use. It was another 9 months going through the process of authorization (mostly because my local hospital had never done anything like this before and together we created a new protocol).

During that time the Phase 2A results came out and I was given access to some of the data. Those, combined with my own experience, gave me the satisfaction that this drug was safe and quite likely effective. I share the concerns about trial size, but like all PALS am concerned for the time required to go through the usual phases of clinical trials. The clinical trial program actually has four parts:

  • Phase 1 – single dose usually in healthy subjects for gauging safety
  • Phase 2 – use in actual patients looking at safety and initial efficacy
  • Phase 3 – larger patient population with different doses, efficacy and SAEs
  • Phase 4 – market surveillance for adverse events

Not only does it take time to fully enroll and execute a large clinical trial but it takes even more time to secure the funding necessary to begin each phase. This is especially true in this current era of venture capital avoiding biotech investment.

I have helped launch other initiatives to get PALS access to experimental treatments. It is critical that patients get more than one or perhaps two chances at early access to treatment while they are newly diagnosed. Drugs that are possibly effective must be made broadly available to patients who are facing otherwise-certain death. Based on the safety and the indication of efficacy in GM6 (mainly borne of my personal experience), I got behind the effort to seek what FDA calls Accelerated Approval so that many more PALS can try it and see where it takes us. Accelerated Approval requires full data surveillance for efficacy, not just serious adverse events (SAEs). The efficacy data determines whether final approval is made. Basically, Accelerated Approval is like a Phase 3 where patients/insurance pay for participation. I believe all PALS would gladly participate in such a program.

If the wider data don’t support the continued use of GM6 I will be the first to admit it. But right now I believe GM6 has the capability to effectively treat ALS in a way no previous drug ever has. And I want to get that opportunity as quickly as possible to as many PALS as possible.

After publishing the press release and posting it on social media and online forums, another PALS started a petition to the FDA to demonstrate the support in the ALS Community for this Accelerated Approval. I would like to urge all who are concerned about ALS – PALS/CALS/Friends – to sign this petition and share it among your social circles. At that link you can sign the petition and post comments to be included with your name. You can also find links to email Senators who oversee FDA and proposed text for those messages.

It is imperative that the comments left on the petition signatures be respectful. FDA isn’t the enemy. They really would like nothing better than to approve a treatment for ALS but need the data to support it. I think we have the data because even though the population was small, the slope of decline as measured by the ALSFRS-R was reduced significantly during the short treatment window. Also, certain biomarker candidates were tracked and correlated with progression. Nevertheless, FDA has to be very careful with the precedent it sets so we as patients must be partners with them in these decisions.

My own experience with GM6 has been positive. The worst part of the entire project was getting the PICC line and the lumbar punctures for CSF samples to make biomarker measurements. I experienced absolutely no adverse events related to the drug. Insofar as benefits, I must admit that the small gains in function noted in the press release are most likely due to surviving neurons branching out new axon terminals to cover the neuromuscular junctions (NMJs) abandoned by the dying motor neurons affected by ALS. GM6 will NOT regrow dead motor neurons. However, it does induce healing in injured ones. In my case, I probably don’t have many injured motor neurons – most of mine are gone. But people who are more recently diagnosed have a higher chance of regaining some lost function in addition to stopping progression.

Based on the information I have seen and my own positive experience, along with the considerable (at best) delay in commencing a larger Phase 2 or 3 trial, I think GM6 deserves Accelerated Approval. I also think this could set a beneficial precedent for future drugs which show similar safety and efficacy signals in early trials. Hence my hope for GM6 getting into the larger population of PALS.

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The ALSETF

As my readers may know, my posts this year have been a little farther and further between. I have been working on some projects. Hopefully one or more can make a real difference for PALS. It’s time to talk about one of these projects: The ALS Emergency Treatment Fund.

The ALSETF is about bringing treatments in late development (post-Phase 2) to ALS patients. Right now there is no hope for the majority of living patients because over 50% do not qualify for clinical trials. For a newly diagnosed patient, the odds of living to see a drug approved which is starting trials at the same time is about 10% (actually much less considering the historical approval rate of ALS treatments). However, with recent advances into the nature of ALS, certain drugs have been developed which show real promise for treating at least a subset of PALS. More are planned to enter trials in the United States very soon. We at ALSETF mean to get that hope ASAP to PALS who are currently living.

ALSETF is a 501C3 non-profit organization with the mission to partner with government and industry agencies to enable Expanded Access Programs. Expanded Access Programs (EAPs) are FDA authorized programs that permit the use of yet-unapproved drugs under medical supervision, in specific cases where those drugs are in late stages of development and have shown preliminary evidence of safety and efficacy. EAPs are only for immediately life threatening conditions for which no effective approved therapies exist. We have open communication with the FDA’s Office of Neurology products for guidance on EAPs involving investigational drugs for ALS. We also maintain open discussion with clinical leaders on the best practices for EAPs, as well as with certain pharmaceutical companies with drugs in trial and in the pipeline.

Our focus right now is to raise up to $5M to help fund certain costs of an EAP such as upgrading manufacturing to clinical-grade, production quantity necessary for fulfilling EAP demand, etc. These are all issues which can currently prevent a pharmaceutical company (especially the small start-ups likely to take a chance on ALS) from accommodating a large EAP. We believe the financial issues can be solved and that the drugs being talked about with excitement in the ALS community can be brought to those who don’t qualify for trials now, while they are still living.

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Wide Open

Tonight I found a double-helping of tasty news: “Regulatory T-lymphocytes mediate amyotrophic lateral sclerosis progression and survival” (PDF). So what’s the big deal? Let me explain in two parts – the scientific then the soapbox.

It has been known for a while that Regulatory T-Cells (Tregs) were helpful in moderating the damage noted in ALS. In fact, one of the authors on this paper, Dr. Stanley Appel, did pioneering work in determining this. What makes this paper exciting is that numbers of Tregs and levels of their associated regulator protein FOXP3 were positively linked to rate of progression. This not only points directly to powerful therapeutic targets but also can be used to separate patient populations between fast and slow progression for more accurate (and shorter) clinical trials, all from a simple blood analysis at diagnosis.

The other thing I found very encouraging about this paper was how it was published. Part of the funding for the research came from NIH. Therefore it would be assumed to be taxpayer-funded and therefore public domain. I have seen open articles published by the paywalled giant Elsevier in such circumstances. However, the chosen journal was an expressly Open Access journal published by the European Molecular Biology Organization called EMBO Molecular Medicine. This is a paper published by top researchers who deliberately chose an Open Access journal. This behavior deserves commendation and support. Research needs to be shared, especially where catastrophic disease is concerned.