Monday, January 18, 2016

An Interview with Dr. Saud Sadiq – FDA Approves Phase II MS Stem Cell Study Conducted by Tisch Research Center of New York

In some dramatic news regarding stem cell treatments for Multiple Sclerosis, the FDA has given its first ever approval for a Phase II Multiple Sclerosis regenerative stem cell trial to the Tisch MS Research Center of New York.

Most previous attempts at using stem cells to repair MS damage have involved intravenously infusing a basic type of stem cell (called mesenchymal stem cells) back into the patient from which they were taken. The Tisch Center (click here) takes this approach several steps further, using proprietary methods to transform raw mesenchymal stem cells into a type of stem cell known as neural progenitors, which are specific to the central nervous system. The cells are then injected directly into the spinal fluid of trial subjects, where, in theory, they should be more effective than raw mesenchymal stem cells at effecting repairs and combating the disease. The Phase I results of the Tisch center’s trial thus far have been quite impressive, offering MS patients worldwide a glimpse into the future of MS treatments and some tangible reasons for hope.

A press release put out by the Tisch Center provides details on what has already been accomplished in the Phase I trial, and what is being planned for the upcoming Phase II study:

"Our unprecedented Phase I results have propelled us into the next phase of research," said Dr. Saud A. Sadiq, Chief Research Scientist at Tisch MSRCNY and the study's principal investigator. He added, "No treatment has shown reversal of established disability until now. The objective improvement experienced in bladder function, vision and walking speed in both secondary and primary progressive MS is remarkable. We now plan to establish efficacy of stem cells as a reparative therapy in Phase II."

The Phase II trial will be a double-blind, placebo-controlled, randomized trial with forty patients in a crossover design. Stem cells are taken from the patient’s bone marrow and manipulated to become brain-like neural cells, a process created and found only at Tisch MSRCNY.  All treatments and research will be conducted at the Tisch MS Research Center of New York and will expand to include an additional leading MS center.

The main obstacle in intiating the study is the need for critical funding. Once funding is obtained, patients will be recruited and the study is anticipated to commence in the summer of 2016.

I had the chance to talk with Dr. Sadiq (who just so happens to be my MS neurologist) about this ongoing and groundbreaking MS stem cell trial, as well as other stem cell related topics. The following interview has been lightly edited for clarity…

How long did it take the Tisch Center laboratories to develop the proprietary process to produce neural progenitor cells from raw mesenchymal stem cells?

Dr. Sadiq: It took about 2 ½ years of intensive lab work, and our lab is the only lab in the world currently using this process.

Is this type of stem cell treatment considered a cure, or can patients expect to require repeated treatments in order to maintain whatever benefits they achieve?

Dr. Sadiq: All of this is still in the experimental phases, so we really don’t know how long the treatments will need to be continued in order to maintain benefit. It’s likely there will be an initial induction period where we will do a number of frequent treatments to induce repair and then to maintain the benefits I think it will require less frequent treatments. But I anticipate that some treatments will need to be given for the duration of the illness, until we find the cure.

What was the treatment protocol during the Phase I trial, and what will the treatment protocol be during the Phase II trial?

Dr. Sadiq: In Phase I we did three treatments over the course of one year, which seemed necessary in order to get some benefit. We don’t know whether the Phase I patients will continue to show benefit or not without further treatment. In the Phase II trial, we are proposing six treatments over a year followed by no treatments over a year to see if benefits are sustained. There will be a control group as well, who will get no treatments the first year and then get six treatments the second year. I anticipate less frequent treatments will be needed to maintain benefit for the duration of the disease until we find a cure.

And would this hold true for any type of regenerative stem cell treatment, such as those being offered by some of the for-profit clinics at which many patients are seeking a single treatment or a single round of treatments over a relatively short period of time?

Dr. Sadiq: Most likely yes, it’s been shown that one treatment probably would be insufficient to even start regeneration. You’ll need multiple treatments…

Are the effects of the regenerative stem cell treatments cumulative?

Dr. Sadiq: You know, we don’t know that yet, in the Phase II trial we intend to collect data on this question…

How much more effective are the neural progenitor cells than the raw mesenchymal stem cells (MSCs) that have been used in other trials?

Dr. Sadiq: Well, as far as I’m aware, the way MSCs have been used in trials so far, there’s not been a single trial that has shown real substantial benefit with MSCs. There’s been some very mild improvements seen in the Cambridge trials, which used one dose given intravenously. It could be that they didn’t use fresh cells or they didn’t use the proper route of administration. Our trial administers the cells intrathecally, directly into the spinal canal. It could be that raw MSCs are in fact effective, but the trials I’ve seen didn’t use multiple dosing. So it’s very difficult to compare the two. But in our hands we found that neural progenitor cells given intrathecally are more effective than raw MSCs.

How much funding is needed for the Phase II trial?

Dr. Sadiq: For the Phase II trial alone we need about $3 million, but for the actual support that we need to build out and expand the labs I’m looking to get $10 million. We need expanded lab facilities and an animal testing facility in place to properly conduct the Phase II trials.

And how much money have you already raised?

Dr. Sadiq: Planning for Phase II we just started, so we haven’t raised anything yet. We have talked to the National Multiple Sclerosis Society, we’ve talked to the National Institutes of Health. We are submitting a grant proposal to the NIH, and the MS Society Is in consultation with me currently to try to arrange funding. We’ll will have to apply for funding grant from the NMSS. We do have a pledge of about $3 million from one donor.

Wow, you have a pledge of $3 million from one donor?

Dr. Sadiq: Yes.

Is the Phase II trial on hold until all of the funding is in place?

Dr. Sadiq: The trial is not on hold, we are really just starting to think about implementation. We just received FDA approval, and we are going to get another MS center involved, I’m in talks with another MS center here in New York, and then after they’re on board we will get things going. So even if we got all the funding right now, we wouldn’t be able to start the trial until late summer 2016. Right now were planning on starting the trial in July or August 2016.

Assuming everything goes as planned, and there aren’t any unforeseen problems, how long do you think it would be until the type of treatment you are trialing would reach common clinical practice?

Dr. Sadiq: It will probably be by the end of the decade, by 2020 I hope if everything goes well.

Do you think there’s any difference in effectiveness between bone marrow derived and adipose (fat cell) derived stem cells?

Dr. Sadiq: Well, we’re not sure. We started working with bone marrow derived cells from the beginning, but adipose derived may turn out to be better because adipose tissues contain a greater number of MSCs, so you may be able to get a better harvest. But because the FDA oversees every project from start to finish, and our trials began using bone marrow derived cells, if we started using adipose cells now we have to go back to the drawing board with the FDA. So in order to continue our ongoing trial protocols, since we started with bone marrow derived cells, we will continue using bone marrow derived cells.

Do you have any thoughts on the clinics that are currently doing mini liposuctions and then re-injecting the cells intravenously the same day or the day after?

Dr. Sadiq: These clinics are just using the stromal fraction, and nobody really knows what that achieves. This process is really not a stem cell therapy, since the stromal fraction only includes a small number of MSCs. It seems like these clinics just want to make money, there’s no evidence that this method would be effective, at least when dealing with neurodegeneration.

What other avenues of research are being pursued in the Tisch MS center’s labs that you consider most promising?

Dr. Sadiq: My goal in life is to find the cause of MS, and that’s really where I concentrate my efforts. I think we have a number of discoveries that are coming together now and I hope that one day soon we will be able to declare that we’ve found the cause of MS.

We do a lot of work with biomarkers as well. In this Phase II trial we are going to look at all the biomarkers that are associated with clinical response, to be able to identify a marker that will be able to predict which patients have the best chance of responding and seeing benefit, and through what mechanisms they are getting better. This is very important work but it’s not the work that drives me to come into the clinic every day at 4 AM.

What does drive you to come to work every day at four in the morning?

Dr. Sadiq: That’s to find the cure to this damned disease.



I can personally attest to Dr. Sadiq’s obsession with finding the cause of and cure for MS. Through the years, Dr. Sadiq and I have developed quite a strong bond, and I know from experience that he can be found hard at work in his clinic and laboratory nearly round the clock, very often at least six days a week. I’ve spoken to Dr. Sadiq via telephone at all hours of the day and night, and have even had office visits with him on the day after Christmas. He once showed up at my bedside at 3 AM when I was hospitalized and suffering miserably from MS related symptoms, to simply be there with me and literally hold my hand. Not your typical doctor, to be sure.

As Dr. Sadiq makes clear, funding is of the utmost importance in order to get this trial up and running in as timely a fashion as possible. There is a tremendous amount of work to be done, and all donations, no matter how small, will make a difference.

As a long-suffering MS patient myself, I know that friends and family are often eager for ways to help fight the disease. While there are many worthy MS charities, in my opinion the biggest bang for the MS donation buck can be had by donating to the Tisch MS Research Center of New York. Donations to the Tisch Center will directly serve to hasten the completion of these stem cell trials and hopefully bring this exciting technology to MS patients worldwide. Please pass the word along, and (click here) to donate.

A big thank you to all who participate in and contribute to this project, and to all the Wheelchair Kamikaze readers that have made their voices heard at the NMSS in regard to funding the Tisch Center’s efforts. It appears that the MS Society may finally be ready to listen, and hopefully the NMSS will this time come through with funding for this vital research.

I’ll keep you posted…

44 comments:

  1. Thank you, Marc. I'm happy you are keeping up with this particular research. Your bond with this doctor increases my faith and hopes with MS research, which often disappoints me. I'm interested in how patients are prepared in order to receive these new stem cells?

    I hope for you also. Faith

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    1. Hi Faith, I too am often disappointed with the state of MS research, but Dr. Sadiq is the real deal. He is obsessively committed to finding a cure for the disease, and he can be quite the maverick. This is made him quite a controversial figure among other, more traditional MS neurologists. Some seem to be so stuck in the status quo that they can't accept that there may be other approaches…

      Thanks for your faith, here's to hoping it will be rewarded soon for all of us…

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  2. thank you for update... great work! I have same solitary sclerosis as you and losing mobility fast. need help asap

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    1. I hear you, the creeping paralysis is relentless. And it's especially frustrating to have a flavor of the disease about which not much is known. And again, most of what is supposedly known about MS may turn out to be completely wrong, but that doesn't make our situations any less frustrating. Hang in there…

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  3. Thanks for sharing this development, Marc. New year, new hope.

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    1. Hey Carmen, thanks for your comment. Yes, we all do need hope, just a little something to hang onto…

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  4. Thank you Marc and Tisch MS center. I was a little sad today and this made my day much better! All the best Dr. Sadiq!

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    1. How does anyone become a participant in this Phase II trial?

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    2. Glad that my blog was able to make you a little less sad today. I know how tough it can be…

      I believe the only people who will be involved in the phase 2 trial are patients who are currently under the care of the Tisch MS center, or of the second center that will be taking part in the trial, which hasn't yet been named.

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  5. I'm deeply grateful for Dr. Sadiq's commitment to discovering the cause of this horrifying disease.

    It seems that as each year passes, the public perception that MS only makes people a little "tired" intensifies. Of course, this puts an even heavier burden on patients.

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    1. I completely agree with you about the public perception of MS. It's really over-the-top ridiculous. Seems like the MS Society in conjunction with the mass media want to paint MS as a disease that's little more than a minor hindrance in people's lives. All of us living with severe disability courtesy of the disease seem to be invisible. Makes me really, really angry…

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    2. This drives me nuts too, the big pharma ads that depict MS'ers living an active lifestyle with only minor disability, managed by whatever drug they're taking. While those of us with more severe cases, not mobile and housebound, are completely ignored.

      Thanks for this interview article. Your doctor gives me hope and I'll be keeping an eye out for the opportunity to participate in the trials. I've been peripherally watching Dr. Burt's HCST work happening in Chicago but the efficacy for SPMS (and PPMS) isn't as clear for such a radical, long-term treatment. The expense to do it outright, if not trial qualified, is astronomical.

      Two CCSVI treatments (Albany, 2010 and Sclafani in Brooklyn, 2013) haven't had significant effect for me. The first time had marginal benefits sustained for about a year while the second was altogether ineffective. But, I know others for whom a first treatment was all that was needed and they continue to do well. I think sustained positive outcomes from CCSVI is very much dependent on the disease state and stage of the individual.

      So, while I wait for the "next big thing" I'm working on smaller problems like ditching some of the narcotics I take in favor of medical marijuana. Cuomo threw a curveball by moving up the availability date and only one of the the two dispensaries on my end of the state is online. It was super disappointing to learn my excellent neuro and her whole practice at the Jacobs Neurological Institute didn't get the ability to prescribe, while another large neurological practice across town (where I had been a patient originally) had 8 docs who could write scripts on day one. JNI will need to get it together asap or I'll need to jump ship.

      Best wishes to you in 2016, Marc.

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    3. Lazy dog, yes, we are in complete agreement on the subject of the "public image" of MS. It's really quite deceiving, as those of us who are more severely afflicted with the disease are given no attention whatsoever by the mass media.

      I've done a lot of research on HSCT, and I've written a few very extensive blog post on the subject if you care to search for them using the search box in the upper left corner of this page. All of my research indicates that unless a patient has enhancing lesions, signifying active inflammatory disease, the treatment won't work for them. That means it won't work for the vast majority of people with progressive MS. For people with RRMS, though, the results are startling in many cases. If I were in RRMS patient, I'd be clawing my way to the top of any HSCT treatment list I could find. Well, not any list, since there are a few unscrupulous centers out there offering the treatment, and it's been shown that the more experienced the treatment center is, the more effective the therapy will be.

      Sorry that your CCSVI treatments didn't stick. Unfortunately, I know far more people who had experiences similar to yours than those who saw permanent benefits from the procedures. Unfortunately, in the case of CCSVI, the hype got ahead of the science. I still think that there is much to be learned by studying the vascular connection to not only MS but a host of neurologic diseases, and the only hope the baby hasn't been thrown out with the bathwater.

      My doctors aren't licensed to prescribe medical marijuana yet, either. I'm waiting…

      Best to you…

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  6. Hi Marc,

    Are you, or will you be part of this trial or any other trial involving the regenerative stem cell research being conducted at the Tisch Research Center?

    BTW, I read every post and appreciate every post. Thank you :)

    Dave - A TM sufferer

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    1. Hey Dave, thanks for the kind words about my blog. You're very welcome. I don't think I'm going to be part of this trial because my disease is so atypical, but I definitely hope to benefit from some of the other research being done at the Tisch center. And who knows, I may get access to the stem cell treatment at some point…

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  7. Forgot a few more questions. Have you asked the doc if this type of treatment would work for spinal cord injuries? How does severed spinal cords or scarred spinal cords play a role in this? Sorry for more questions, but this is what my brain thinks of when I read this stuff!

    Thanks!
    Dave

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    1. Dave, I'm not sure anybody knows the answers to the questions you pose. In theory, the stem cells should repair any type of nervous system damage. In fact, one would think they might even be more effective in treating spinal cords and brains that suffered traumatic injury, since there is in an ongoing disease process that will continue to attack the nervous system tissues even after the stem cells have done their thing. How the stem cells effect regeneration is a question that still be explored, and there's even some recent evidence that it's not the stem cells themselves but some proteins they excrete that actually stimulate the body to start repairing damaged tissues. There have even been some experiments just using the agent that the stem cells are grown in, which becomes rich in these proteins, and has been shown to restore function in paralyzed rodents. The future is definitely bright, we just have to hope that we can hang on long enough to take advantage of these coming developments…

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  8. Hi Marc, thank you for another great post. My husband is the patient of Dr. Williams of Tisch Research Center in New York. We have tried quite a few treatments there, but so far, no luck, but the care that he receives there is great, front desk, nurses, doctors, social services, MRI staff are awesome. My husband, (Roman) is very skeptical about the cure, as most of what he has tried did not work for him. After I read your post to him yesterday, with all the detailed explanations, he became more happy and hopeful that maybe there will be a light at the end of the tunnel for him. I mean at 40 years old, he can barely walk and his vision is 20/400. And he had so much going for him. He lost so freaking much because of this beast MS. Any way I am not sure how they will be selecting candidates for Phase II trial, but hopefully Roman is one of them. :) Even if not, there is hope in the future that this damned disease will be cured and people will get their functions back. Let's hope for the best! Thank you again for all the posts. They are great!
    By the way, did you hear or read anything on Rhodila Rosea. It’s a great adaptogen herb and it helps with mood, stamina energy and all the other good stuff for the body. We have been taking it for a few days and it already made a difference.
    Good luck and keep strong!
    Tatyana

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    1. Hi Tatyana, I'm so sorry to hear about your husband's condition, but it sounds like he has a great wife to help him deal with this damned disease. Believe me, I know just how important having a loving partner can be. Lots of patients with MS aren't so lucky, as I think the divorce rate for MS patients is above 80%.

      I'm glad my post gave your husband some hope. I'm not sure how the patients are being chosen for the phase 2 trial, but I'm sure some inclusion/exclusion criteria will have to be agreed upon by all involved.

      I haven't heard about the herb you mention, but I'll have to check it out. Has your husband seen Dr. Bates, the naturopath who works at the clinic? If not, I would highly recommend it. She's really terrific.

      As you say, we can only hope for the best, and find some solace in knowing that Dr. Sadiq and his staff are hard at work looking for a cure. He's hinted that they are getting closer, if only…

      BTW, I've known Dr. Williams since he first started at the center, and I think he's a terrific guy. I often go to him with questions about research, and he's always happy to answer. I've heard from many patients that he's really a heckuva Dr.…

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  9. Good Interview – Dr. Sadiq sounded like a very knowledgeable and caring . Actually, he is one of the only doctors who researched and discovered a treatment :)
    SEO pakistan

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    1. Dr. Sadiq is a very unique Dr., in that he divides his time between research and clinical practice. He's extremely dedicated, and if anyone is going to find a cure, I really believe it will be him.

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  10. In the process of stem cell therapy that Dr. Sadiq is testing is chemotherapy involved?

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    1. No, Dr. Sadiq's therapy does not involve chemotherapy at all. You're thinking of HSCT, the type of stem cell therapy that uses intense chemotherapy to wipe out a patient's immune system, and then stem cells to help rebooted. It's a completely different approach, like comparing apples to oranges. HSCT can be remarkably effective in patients with active inflammatory disease, but it does nothing to directly address the damage done to the central nervous system by MS. Dr. Sadiq's therapy, on the other hand, doesn't really address the aberrant immune response, but instead concentrates on fixing the damage that the disease does. Like I said, two entirely different approaches.

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  11. Note this Phase II stem cell trial for ALS: http://www.umassmed.edu/news/news-archives/2014/04/FDA-approves-Phase-II-clinical-trial-for-ALS-at-UMMS/

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  12. HSCT is intended for RRMS as well. This is extremely encouraging. Dr Sadiq's sounds genuinely enthusiastic to come up with a substantial treatment for all types of MS. Please keep us informed of his progress

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  13. M
    If this and other similar therapies don't address the "aberrant immune response", how can they b effective?

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    1. This type of therapy isn't concerned with the ongoing "aberrant response", but rather with attempting to fix the damage that response does. As such, treatments like this would need to be given indefinitely, as Dr. Sadiq mentions in the interview above.

      Patients with active inflammatory disease who are in the trial stay on their DMDs, but there is some evidence that stem cells infused intravenously can moderate the immune response.

      Clearly, this type of therapy is only one leg in a multi-leg approach to attacking Multiple Sclerosis. As the underlying etiology of the disease is still a big unknown, there's no way to currently go after the MS "culprit", and immunosuppression is the best the powers that be have to offer as far as dampening disease activity. Regenerative stem cell treatments seek to fix the damage done, but as you note, they don't turn disease off.

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  14. Marc, I am very interested in this program. I have PPMS and am in a wheelchair. I cannot walk or stand alone. I desperately would like to participate in this trial. I feel like it would be an excellent candidate. Who can I contact regarding this trial? Susan

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    1. Susan, because this is a smaller phase 2 trial, I believe the trial subjects will be chosen from the patient population within Dr. Sadiq's clinic, and whatever other clinic is enlisted in the study. Since patients will be getting regular injections of stem cells, and need to be tracked carefully, I believe all trial subjects will already have been known to the physicians.

      If this does change, though, I will let everybody know.

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  15. I am excited about this research. Do you know how long the trial will take to finish?

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    1. I share your excitement about this research. The phase 2 trial is supposed to last two years, and won't get started until this summer at the earliest, so final results could be expected in 2018. Yes, I know, seems like a long way off. Especially when the disease just keeps chugging along…

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  16. What I find so exciting about this procedure is that it does not require that the immune system first needs to be destroyed with chemotherapy.

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    1. Please keep in mind that the type of stem cell therapy that destroys the immune system, commonly called HSCT, has an entirely different goal than the stem cell therapy that Dr. Sadiq is experimenting with. HSCT seeks to reboot the immune system, and has been shown to shut down the MS disease process in properly selected patients for many years at a time. Something like 80% of patients getting this treatment have absolutely no relapses or new lesions for at least seven years afterwards, and many for much longer than that.

      Dr. Sadiq's stem cell trial seeks to regenerate the nervous system damage done by the MS disease process, without addressing that underlying process. This is why, in the above interview, Dr. Sadiq says that repeated treatments of this stem cell therapy will probably be needed for the life of the patient, as long as the cure for MS isn't found.

      These two types of stem cell treatments are completely different approaches, and comparing them is very much an apples and oranges situation. It's very important that patients be clear on this. It's not that one of these therapies is better than the other; they are two completely different ways to attack MS. In fact, for patients with active relapsing remitting disease, a combination of HSCT followed by regenerative stem cell therapy (the type that Dr. Sadiq is trialing) may be the most effective approach available. Of course, this is conjecture, as regenerative stem cell therapy has yet to really prove itself, whereas HSCT has proven itself in many, many patients. Unfortunately, it's not much use in patients with progressive disease or don't have enhancing lesions.

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  17. I became a patient of Dr. Sadiq's last summer - what an inspiring and caring man. How are people NOT pouring money into the Tisch Research Center is beyond me.

    Thank you for this interesting interview!

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    1. Hey, I'm not a fan of MS either, what a coincidence!

      Glad to hear that you are a patient of Dr. Sadiq's. You are in good hands.

      Thanks for commenting…

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  18. You have probably seen this, but in case you haven't , Google Dr. Coimbra and high doses of vitamin D. Trust me,

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  19. Just a quick question I see that they hope to roll this out if all goes to plan in 2020? Is that for mass use if the trail goes well that this would be available to the public. As time is precious thing with this disease.

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    1. Well, the hope is that by 2020, if all goes right, this therapy would be "ready for prime time". Of course, a lot of this would be out of Dr. Sadiq's hands, as its acceptance in the MS treatment universe would rely on a number of factors, including the perception of other doctors, how powerful the results turn out to be, and resistance, if any, from the pharmaceutical industry. Lots of moving parts…

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  20. I was interviewed and accepted into Dr. Burt's (Northwestern U in Chicago) stem cell research project in 2009. I was accepted, but my insurance denied me. I had small kids as well and 9 weeks seemed unbearably long to be away from them. I hope this works. I want my dignity back, please.

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  21. Just found out that this research will be presented at AAN. Very excited! Keeping fingers crossed for good results:)

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  22. I am wondering how can i get medical marijuana in New York.

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  23. Fascinating research and reporting, Marc. Thank you!

    Centenniel

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