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  • The Rogue Stem Cell Clinic Blacklist

    Irving Wesissman, M.D., is a physician scientist who directs Standord's Institute of Stem Cell Biology and Regenerative Medicine. This is a major stem cell program and Weissman is therefore a major figure in stem cell research. He recently became president of the International Society for Stem Cell Research (ISSCR) an independent, nonprofit that promotes the field and offers professional and public education in all areas of stem cell research and application.

    Weissman promises an activist term as president, urging vigorous research but tempered with reality. One area he plans to tackle is the spread of stem cell clinics overseas. At his urging, ISSCR formed a new committee, Task Force on Unproven Stem Cell Therapies.

    Here's what he told the journal Nature recently: "I can't tell you how many phone calls and letters I get every week that start of with "I have blank untreatable condition and I'm going to blank for stem cell therapies. Tell me it's okay.' And of course I can't tell them it's okay. It's time to have a professional body do it.

    Weissman urges that ISSCR publish a 'blacklist' of rogue stem cell treatments " ... we need to list unproven therapies. To do so we need to get from the practitioners of potentially unproven therapies a statement that would allow anyone to look at the peer-reviewed papers from independent investigators that give the evidence that the putative stem cell therapy should work for the disease specified. If they lack peer-reviewed demonstrations of the discovery behind the therapies and IRB and FDA-type approvals, I would recommend that they be put on a list of unproven therapies."

    Herewith the requirements, once again: (1) The provider or clinic must be able to send peer-reviewed papers from independent practitioners that establish the therapy as possible. (2) The provider must send evidence of approval from an independent committee, such as an institutional review board (a document from the clinical entity where the therapy is being practiced that shows the steps taken to protect the patient and justifying the experimental or approved therapy for their specific disease). (3) The provider must send a document from an agency equivalent to the Food and Drug Administration showing the approval of the experiment or the therapy for human subjects with their disease.

    Says Weissman, without all three, patients "must assume they are receiving an unproven therapy and that [he or she] is in danger of risking their life or health, of losing their money, of being away needlessly from home, family, and friends, and of course, of not being therapeutically improved."

    An international task force of physicians, lawyers and advocates has been formed at ISSCR to deal with the blacklist idea: Chair: Patrick Taylor, JD Children's Hospital Boston; Roger A. Barker, PhD, MRCP Cambridge Centre for Brain Repair; Elena Cattaneo, PhD Univ of Milano Dept of Pharmacological Sciences; Alan Colman, PhD Singapore Stem Cell Consortium; Hongkui Deng, PhD Peking University; Harold S.H. Edgar, JD Columbia Law School; Claude Gerstle, MD Ophthalmologist and Advocate; Lawrence S.B. Goldstein, PhD University of California San Diego; Andrew Lyall, Stem Cell Network; Fernando Pitossi, PhD Leloir Institute; Douglas A. Sipp, Riken Center for Developmental Biology; Alok Srivastava, MD, FRACP, FRCPA Christian Medical College; Susan Stayn, JD Stanford University; Gary Steinberg, MD, PhD Stanford School of Medicine; Amy Wagers, PhD, Joslin Diabetes Center;  Irving L. Weissman, MD - Ex Officio Member, Stanford University.

    Mad
  • This Just In: Respiratory Guidelines for Consumers

    Respiratory Management Following Spinal Cord Injury: What You Should Know has just been published by the Consortium of Spinal Cord Medicine. It's a must-read for people with any sort of respiratory complication. This consumer-friendly guide is the companion book to the clinical practice guideline Respiratory Management Following SCI.

    The guide is written and reviewed by medical experts and covers treatments and management of respiratory function for caregivers and family members. You can download it free. It is also available in printed form.

    From the intro: "This guide will answer many of the questions about respiratory health that  may arise after a spinal cord injury.  It will describe a variety of specialized treatments that  people who have sustained a spinal cord injury may require to accommodate their respiratory needs.

    "Additionally, it is hoped that this guide will reassure people who are newly injured or have ongoing difficulties with breathing post-injury that there are safe and reliable methods available for dealing with these difficulties. It is always important for a person with SCI and their caregivers to talk with their doctors, nurses, and therapists to make sure all questions are answered satisfactorily."

    The Consortium, funded by the Paralyzed Veterans of America, comprises members from the leading professional associations that serve the SCI community. Included on the Steering Committee panel are doctors, nurses, psychologists, occupational and physical therapists and others groups.

    The Reeve Foundation is a member of the Consortium.

    The Consortium has produced 10 clinical practice guidelines, widely accepted in the medical professions as the definitive guide to medical care for SCI. Seven of the CPGs are accompanied by a consumer version, including these:
    Autonomic Dysreflexia: What You Should Know
    Expected Outcomes: What You Should Know
    Depression: What You Should Know
    Neurogenic Bowel: What You Should Know
    Pressure Ulcers: What You Should Know
    Preservation of Upper Limb Function Following SCI: What You Should Know

    Mad

  • Woebegone Stem Cell Tourist Report

    This came in the other day, text and punctuation (cringe) unchanged:
    i have been a patient of geeta shroff and i know she is a fraud and a manipulator of desprate people she is just trying to make as much money as possible with no regard as to the dangers of this kind of experimental therapy.

    i hope anybody who is thinking of going to that shithole thinks and thinks again because YOU WILL REGRET IT ,because when you get home from the treatment you will be financially, mentally, emotionally and physically broken DO NOT BELEIVE HER LIES SHE IS A CON !!!!!
    That's from the Internet fringes of Care Cure. The quote was posted last week by a scribe called Regretfull. If you want to see the original, go here, it's on page 45 of "Reports of Patients Treated by Dr. Geeta Shroff, India (Human Embryonic Stem Cells)."

    As you will learn if you slog your way in to the discussion, Dr. Shroff runs a cure clinic in India. She takes on people with a long list of ailments including spinal cord injury, extracts $30,000 a visit, and then injects them with an embryonic stem cell line of her own cooking. There are those among us who are positively charmed by her. See Amanda Boxtel's cure travelogue.

    But Western rationalists take off on Shroff because she hasn't shared the recipe. Nor has she shared data and outcome results in any way close to the manner of the scientific or medical professional. All we get is patient testimony on the Internet. Your job, then, is to take Amanda and Regretfull and sift out what sounds right. And ask yourself, do you really want to experiment with your own coin, and your own body, in Delhi?

    That isn't the only active and amusing crack in the stem cell tourism façade. Another Care Cure writer has challenged the veracity of Ricci Kilgore's testimony about her embryonic stem cell recovery in the Dominican Republic.

    From Page 18 of a thread called "Ricci Kilgore (found a cure)":
    "Medra is a Scam and Ricci is a liar, by Lisa French, My friend and I have been trying to expose Dr Rader. Our goal is to shut down Medra. National attention would ruin Rader."
    Ricci, who is no stranger here and can be seen on p. 186 of the Paralysis Resource Guide, and in two Reeve PRC videos (scroll down to skiing and horseback riding), is an incomplete para who walks with canes. She's from Reno but living in the San Diego area now; she is on the US Olympic adaptive ski team development squad, married to a Navy guy. She's a nice kid, sincere as heck and from the photo on Facebook, she's a newly minted mom. Congratulations Ricci, now saddle up a horse called acrimony.

    Ricci tells the story of getting immediate recovery after getting hooked up with Dr. William Rader's stem cells at the Medra clinic, operating in the Dominican. Now come her challengers who discovered some old news clips from the time of her injury leading them to conclude Ricci may have already gotten tons of recovery back - long before she became the perfect poster girl for the perfect grift.

    Rader, whose company is based in a house overlooking the Pacific in Malibu, California, is a psychiatrist and former owner of a chain of eating disorder clinics in LA. He was once married to All In the Family's Sally Struthers, which is meaningless except perhaps to boost his Hollywood bona fides. He's not real warm to the glow of the media (go here) but aggressively sticks to his story about the healing power of stem cells. He apparently got into the stem cell biz even before 1998, when human embryonic cells were identified. He had a clinic in the Bahamas that got shut down in 2000, thus the move to DR. He claims to have cured Alzheimer's and AIDS.

    For the sake of fairness, here's Ricci's response to some of this recent hoopla.

    But wait, there's more. This also came out last week, accusing Medra of fraud, threats and intimidation. The scam is international (send money to an offshore bank before Rader will bring you to the Dominican for stem cells harvested from the Ukraine). Even Ricci's husband and her new-age uncle back in Reno get drawn in. See this unfold on a website called RipOff Report. Bodie, from Parkville, Maryland, writes:
    The web of interconnected fraud that has been uncovered is beyond belief. Dr Rader probably found Ricci through her so called uncle Dr James Forsythe, a homeopathic aka oncologist , who works with her mother Valerie Kilgore at the century Wellness Clinic in Reno Nevada. Dr Forsythe worked with Dr. Albert Scheller, Rader's chief scientist.

    This is how I believe Medra found the perfect spinal cord patient that miraculously walked.
    We're not talking about the accuracy and fairness of the New Yorker magazine here but all this noise might make you think, twice, about doing business with Shroff or Rader. The least you could do is ask them to clear the record for you: Are all those cure touts really on the payroll?

    One more item to make the point that the wheels may fall off the stem cell tourism racket: Police in Hungary last week arrested four people they suspect of running an illegal stem cell clinic in Budapest. Ye gods! The cops say the treatments were unproven, based on stem cells taken from embryos or aborted fetuses, and cost as much as $25,000 per person. Read it here.

    Mad

  • Canadian Californians Golf for Reeve Foundation

    On Friday August 7 a group of ex-pat Canadians living in Southern California will host a charity golf tournament. The newly formed Canada California Business Council first planned to raise money to help one of their own, Andrew Trevitt, who was paralyzed in a motorcycle accident last year. Andrew said no thanks but suggested the money go to the Reeve Foundation, which helped him and his wife in the chaotic early days after the injury.

    After a long, hard road through rehab Trevitt just spent his first week home since the crash last November. At the time of what he later called "my little whoopsie," Trevitt was testing tires, part of his job as a senior editor at LA-based Sport Rider magazine. A car made an illegal u-turn in front of him on the twisty Angeles Crest highway.

    Two months later, dictating a blog by way of his wife Deborah, he wrote: "I have a broken elbow, wrist, pelvis and leg, along with too many screws and plates to count. Worse, I fractured my spine and I'm not sure if I'll ever ride or walk again."

    In 10 years of motojournalism Trev only crashed this one time. Are bikes dangerous? "The weird thing," he says, "is that in rehab there were people with spinal cord injuries from all kinds of things. People fall off ladders. A guy broke his neck getting into bed, by hitting the bed board. Another guy broke is neck between the first green and the second tee. He tripped and fell. Maybe motorcycles are dangerous. But so is everyday life."

    As a professional motorcyclist and former Canadian National champ, Trevitt, who answers to Trev, knew and accepted the risks. "There was always the risk....I always tried to ride as safely as I can. It was in the back of my mind. After my tipover, I said, well, OK, it's happened."

    In other words, OK, this is what it is, let's deal with it. Trevitt says he worked hard at every aspect of rehab in order to gain independence. He's not yet driving a car but will soon. And he's eager to get back to work at the magazine. He won't ride again, and in fact has no interest in riding. But his skills as a writer are solid, and much missed.

    He writes a column for Sport Rider. It used to be called Full Pin, "because that's how I always approach life and individual tasks-with 100 percent effort." The column has been renamed Stop Watch. "I feel this title will probably be more representative of my new outlook."

    From the blog: "I've got loads of other fond riding memories, but for whatever reason I'm okay with the fact that I probably won't be able to ride again. That ship has sailed, and in the last couple of months I've realized there are loads of other things that I'm still able to do, and I'm looking forward to trying new things that I probably never would have thought of."

    Unlike most newly paralyzed, Trevitt got an extended rehab experience. Partly this was due to complications (other injuries, infections and surgery for a skin sore) but also because he was covered by state workers comp insurance. "For me it's been really great. A lot of people I saw who had to deal with regular health insurance got cut off. As for work, a lot of people find that the job they had, they can't do now. So they give up. It's really disheartening to see people give up."

    No surrender in this family. From Deb, who has been along for the whole bumpy ride: "I wouldn't lie to you, this is no cakewalk, but Andrew's optimistic nature sure helps me and everybody else. Like they say attitude is everything."

    Mad

  • Reeve Grant to Rancho Wheelers

    We visited the Rancho Los Amigos National Rehabilitation Hospital wheelchair sports program this week in Downey, California (for reference, it's right in the middle of the Los Angeles metroplex, one town over from Compton, framed by the 105, 605 and 5 freeways).

    Rancho is a venerable place of healing and hope, going back 100 years ago as the site of the LA County poor farm. Fifty years ago this place was famous for taking care of a large population of people affected by polio. More recently, "the Ranch," as it is sometimes called, has been a Model Systems Center for spinal cord injury, famous for taking care of a large population of uninsured local youth transitioning from the many multicultural knife and gun clubs of LA.

    We were there to deliver a check, a Reeve Foundation Quality of Life grant, to the Rancho sports program. In fact, the check was made out to Las Floristas, a 65-year-old charity comprised of women who fund programs (over $7 million so far) for younger folks at the Ranch. Las Floristas wrote the grant on behalf of the wheelchair sports program, which will use the money to buy specialized sports chairs for peewee basketball players.

    Deborah Veady, a volunteer grant writer for Las Floristas, accepted the check. (As an aside, Ms. Veady's father, an amputee, was a beneficiary of Rancho expertise over 50 years ago.) On hand to bear witness and to give us some perspective were two members of the fourth best in the U.S. Rancho Renegades wheelchair basketball team, Daniel Nong and Danny Maravilla, along with their coach and long-time Rancho sports maven Lisa Hilborn. The team motto is "push hard or go home."

    Herewith are a few quotations from the group. You get the message that around here sports isn't only about purposeful exercise or being part of a team effort, or even about putting the whoop on an opponent. For some, including Daniel and Danny, sports provides a ticket, good for whatever you want it to be for.

    Daniel Nong, 16, hometown Long Beach. Shooting guard, makes 20 percent from the three point line, number 55, and co-captain of the Renegades: "Before I started playing wheelchair basketball I was a quiet kid, I didn't go out, didn't make friends. I was passive about everything. But basketball has given me intensity - life is my oyster, is that the saying? Basketball has given me a goal, to get a scholarship, to go to college. My advice to young people who might be in a wheelchair is that sports will lead the way. Never give up on life. Sports has changed my life so much."

    Danny Maravilla, 16, Long Beach. Point guard, playmaker, number 00, co-captain. Once played wheelchair basketball on the TV show "Suite Life of Zack and Cody." "Basketball has kept me out of trouble. Where I live, there's a lot of not good stuff going on - like gangbangers and drugs. If not for being in sports I'd probably not still be in high school. I can go to college now. The way I look at it being in a wheelchair, you can take it as a bad thing or can make it into a positive thing. For me, my social life is better. Others look at me not as a kid in a wheelchair but as a basketball player."

    Lisa Hilborn, Rancho wheelchair sports director for 20 years. "Wheelchair sports is a powerful vehicle to pull athletes into a positive environment. We have a strong program here but our emphasis is on grades: we are involved with the schools and monitor their progress. We push the kids until they are intrinsically motivated."

    As for the grant money, the program will help younger ball players get the right gear to get to the next level. "We sincerely thank Las Floristas...without them the wheelchair sports program would not exist."

    Deborah Veady: "Thank you Reeve Foundation."

    Mad

  • NY Ups Ante in Egg Business

    Qualified women have been selling their eggs on the open fertility market for many years. There are some who would call this a form of reproductive prostitution but egg donation is quite common: The going rate nationally is between $5,000 and $8,000 per cycle, more for attractive, educated women.

    Getting paid for donating eggs for stem cell research, however, has never been kosher. Until now.

    Going against the bioethical grain, the Empire State Stem Cell board - which will award $50 million a year for stem cell research in New York  - agreed in June that there is no real difference between a woman donating oocytes (eggs) to a fertility clinic that hopes to make a baby than there would be to donating eggs to a lab researching stem cells. In other words, to help meet an ever increasing demand for eggs, it's ok now to get paid for giving them up for somatic cell nuclear transfer research (a.k.a. cloning).

    Said the board, "The risks associated with donating oocytes to stem cell research are no greater than those associated with reproductive donations.  Moreover, donating oocytes to stem cell research arguably confers a greater benefit to society than does oocyte donation for private reproductive use."

    Said David Hohn of the Roswell Park Cancer Institute in Buffalo, "We were unable to come up with an ethical reason for why we should pay for reproductive donations, but not for research."

    The New York policy jumps ahead of the National Academies' guidelines, which strongly discourage any form of payment for non-reproductive egg donation. New York has also gone beyond California's guidelines on paying for eggs: The California Institute for Regenerative Medicine's rules limit payment to egg donors to just "expenses." No one has decided just what constitutes expenses, so you can bet there is some flexibility there.

    New York's policy is in line with the guidelines developed by the American Society for Reproductive Medicine: $5,000 is about right, no one should get more than $10,000 for egg donation. Pretty good compared to say, sperm donation at about $100 a pop.

    Before anyone starts to think that's easy money, consider what an egg donor must endure. Hormone manipulation (one injection per day for 10 days of Follicle Stimulating Hormones) may include side effects such as headaches, mood swings, bloating, nausea and bloating as the ovaries to swell and produce many eggs. PMS cubed, that's how one woman described it. Permanent damage to the ovaries is a risk, albeit small (one or two percent). Extracting the eggs requires twilight sedation (outpatient) by way of IV.  Using a vaginal ultrasound probe, a small needle is guided through the vaginal wall to the ovaries in order to vacuum out the mature eggs.

    I don't have a problem with women making deals for their eggs; the notion of comodification of body parts doesn't concern me (OK, I do not want to see kidneys or other organs listed on Craigslist). I see the ethical issues this way: Donors don’t get paid for their eggs, per se; they should be compensated for the arduous process of egg retrieval. Because of the time involved, plus discomfort and risk, it would be wrong not to pay women.

    What must accompany the new New York policy is a wide-open informed consent process. Women have to know what they're in for, and unfortunately, the human egg industry is largely unregulated and full of brokers and third party outfits that may not feel bound by medical ethics to assure full consent.

    If the market for research oocytes remains robust, there will be advertisements that may entice economically vulnerable women to go for the money, without fully discussing risks. Let's make sure all women have all the facts.

    New York's health department produced a guidebook on egg donation. It lays out the facts, and cautions against aggressive brokers who line up eggs but who don't do medicine.

    See also the National Academies report, mentioned above.

    Mad

  • One + One May Be Less Than Two

    The red pill makes you better. So does the blue one, even though its action is different. So, doesn't it figure that taking red and blue together will make you all the more better? If two or three, or even six or a dozen therapies showed some promise on their own, why not just take everything. Toss in the proverbial kitchen sink.

    Of course therapeutic synergy doesn't work that way. Drugs and treatments are by no means additive or complementary. They may interact in unpredictable ways or create new side effects. Drugs in combination may cancel each other out. Polypharmacy could make you worse.

    Combination treatment has not yet become an issue in spinal cord injury, but it could as new treatments emerge. An injury to the spinal cord isn't a fast and neat process. After the primary damage to the cord a biochemical process continues for hours, days and even months. This secondary wave includes blood loss, swelling, inflammation and tissue degradation due to a range of toxic cellular events.

    While there's not much that can be done about the primary cell loss, at least not in today's clinic, there are strategies to intervene in the secondary cascade to preserve nerve tissue and therefore function. These strategies will require combinations of therapies, timed over months, maybe years, to deal with specific cellular events, long axon growth and guidance, scar reduction, remyelination, and so on. The array of treatments must be carefully sequenced for optimal recovery.

    Scientists are already thinking about such combo therapies. Here's a look at a recent study wherein it was postulated that two treatments together would result in a better outcome than either would have alone. The paper was written by a team including Reggie Edgerton of UCLA, and Martin Schwab from the University of Zurich. Both the Edgerton lab and the Schwab lab are part of the Reeve Foundation International Research Consortium on Spinal Cord Injury. The work was funded in part by the Foundation.

    One arm measured the effect of Schwab's anti-NOGO-A antibody, the other looked at the effect of treadmill training (underpinned in large part by Edgerton's basic science). Both the antibody and treadmill have been shown separately to enhance recovery after spinal trauma. The antibody does this by neutralizing NOGO, a chemical barrier to nerve growth. Treadmill training is believed to work by using movement to refresh nerve plasticity and thereby unmask latent stepping patterns within the spinal cord.

    The anti-NOGO treatment is now in clinical trials in Europe and Canada; it is hoped that the trial will expand next year in the U.S.

    The treadmill work is the basis of the Reeve Foundation NeuroRecovery Network.

    One group of rats got the anti-NOGO treatment for two weeks following incomplete thoracic spinal cord injuries. Seven days after injury these animals then began training on treadmills five days a week for eight weeks. The combined treatment group was compared to rats that got just the anti-NOGO and to animals that just got treadmill training.

    Are the treatments synergistic?

    The short answer is no. Animals that had both the antibody and the physical therapy actually showed a diminished functional recovery.

    The scientists suggest that as the antibody promoted nerve fiber growth, these fibers may have reestablished pre-injury connections. Meanwhile, however, the treadmill training may have interfered with this process. The two therapies may be competitive.

    It may be a matter of timing; the scientists observed that it was better to allow two weeks longer for the regenerative process to rewire the cord before introducing activity-based therapies.

    What's next? Says the paper, "The optimal arrangement of combinatorial treatments, e.g. a growth and regeneration enhancing treatment followed by appropriate rehabilitative training remains an exciting challenge for future studies."

    In other words, more work to do, hold the sink. Future treatments most certainly will be done in some sort of combination or sequence. It will take a while to sort it out so the whole really is greater than the sum of the parts.

    Mad

  • The Spine at 21

    The 4th edition of Spinal Network arrived the other day. It’s big and bold and for the first time, colorful. This book is so full of life it literally has a pulse; it’s so rich in character and dripping with personality that you just want to hug it. It’s so nourishing, in areas of medical, lifestyle, community, etc., that if you could fit it in the Cuisinart, you could make soup that would sustain you across the lifespan.

    Those around the wheelchair world in the late 1980s ago might have gotten a copy of the first Spinal Network…back when it was subtitled the Total Resource for the Wheelchair Community by its creator, yours Madly. That was in Boulder, Colorado, 1988. The following year, the big book begat a periodical called Spinal Network Extra. Not much schwing to that so it became New Mobility.  

    The Spine, as we used to call the big book, is now produced and edited in North Carolina by Jean Dobbs, who got caught up in its web not too long after the first edition came out. Jean came to Boulder to get her masters in journalism. She showed up at the office one day, wasn’t repelled by our aggressive spontaneity so I put her to work. When the enterprise was headed for the rocks after a second edition of Spinal Network drained the treasury, we got a rescue from Malibu, California. I was part of the deal, and Jean came along too; she has had a big hand in guiding both the magazine and Spinal book ever since. Kudos to you, Jean. Thanks for keeping this thing alive.
     
    As for the new edition, as usual, it’s awesomely complete (to quote the LA Times from 21 years ago). I scribed the medical and cure sections again, so expect a balanced diet of advice, humor, truth, pathos and not much bathos. The rest of the book emerges from the collective voice of the spinal community – it you’re new to this paralysis thing, you need to hear it. If you’ve been around, you’ll enjoy the chorus and appreciate the validation.

    Find more here.

    Mad

  • More Reasons to Avoid Experimental Stem Cells

    Stem cell treatment overseas anyone? You may still be tempted by Internet testimonials as people who spend thousands of dollars report uniformly modest results. One more plea for rational thought may not make much difference but there are some very good reasons not to become a medical experimental tourist. A group of prominent scientists and doctors has just penned a cautionary letter to the medical journal Spinal Cord. It's entitled "Position statement on the sale of unproven cellular therapies
    for spinal cord injury," from The International Campaign for Cures of Spinal Cord Injury Paralysis.

    It is printed in whole below. If you want the distillation, here are several take home sentences:

    People with SCI, as with any serious medical conditions, are highly susceptible to advertisements promising recovery, even when the costs are high and potential risks are unknown. It is morally unacceptable to prey on and profit from their hope for a cure.

    Patient-reported anecdotes are not a substitute for medical evidence.

    First, there is a significant placebo effect, especially in neurological diseases which functions such as sensation, spasms or residual movements can vary daily.


    Herewith the letter:

    Over the past few years, it has become possible for people with spinal cord injury (SCI) to purchase experimental treatments, often involving transplantation of 'stem cells' or other cells or tissues. These cell-based therapies are advertised as having beneficial effects, leading to some recovery of function, even though there is little or no evidence supporting such claims. Generally, these interventions are provided in countries where government regulation for consumer protection is less comprehensive or effective than in many developed countries.

    This trend is of concern for several reasons. People with SCI, as with any serious medical conditions, are highly susceptible to advertisements promising recovery, even when the costs are high and potential risks are unknown. It is morally unacceptable to prey on and profit from their hope for a cure. We believe that it is unethical to charge these patients for experimental interventions that are not yet proven safe and effective by properly conducted clinical trials.  Frequently, providers of these treatments attempt to establish a veneer of credibility by citing experimental studies, where they have no direct association. Most often the critical scientific data to support the safety and efficacy of the new treatments are lacking, so they rely on testimonials from patients or their family members. Patient-reported anecdotes are not a substitute for medical evidence. First, there is a significant placebo effect, especially in neurological diseases which functions such as sensation, spasms or residual movements can vary daily. The only accurate way to determine that a treatment is beneficial is to carry out a properly designed study with a placebo-treated 'control' group. Individuals or institutions selling therapy to-date have not carried out controlled trials with valid methods and outcome measures recorded by blinded observers. Second, because of a clear conflict of interest, it is not acceptable that those who profit from providing the treatment should also carry out the evaluation of efficacy and safety.

    Most cell-based treatments carry safety risks, many of which are common to surgery and transplantation in general. These risks can be significantly higher in people living with SCI and are currently not balanced by any reliable assurance of benefit. Thus, it is essential that anyone offering non-standard treatments for SCI provide rigorous long-term clinical follow-up at no charge to assess fully the risks versus benefits. Although highly experimental and potentially dangerous therapies may be more readily justified in people who have terminal conditions, the risk of using these therapies should be tempered by the fact that patients with SCI who receive standard medical care can anticipate a near-normal life span.

    The risks and costs of untested therapies are not limited to the individuals who pay for such treatments. These interventions undermine objective scientific studies that would help other people with SCI. First, these individuals are likely to be excluded from subsequent involvement in scientifically valid clinical trials because of potential interference from the first intervention. Second, unsuccessful treatment of SCI with poorly characterized or unvalidated 'stem cells' in uncontrolled trials can undermine enthusiasm for future developments of valid stem-cell technology. Third, it may not be possible to recruit an adequate number of participants for valid clinical trials if potential participants have instead chosen to undergo uncontrolled, for-profit treatments. Thus, there is a cost to society, as well as for people with SCI when 'for-profit' therapies are offered and purchased.

    We do not rule out the possibility that cellular therapies may improve function and quality of life for recipients and justify the risks, but insist that the onus is on the providers to deliver such proof from a valid clinical-trial program. It is unethical to sell unproven therapies, we do not advise patients to volunteer for such treatment procedures. Unfortunately, in the context of an entrepreneurial enterprise, it is unlikely that accurate, reliable or useful medical evidence will ever be generated.

    More information on questions that should be asked of someone offering a treatment for SCI is available in the free document, 'Experimental Treatments for Spinal Cord  Injury: What you should know'. This is available in several languages at the International Campaign for Cures of Paralysis (www.campaignforcure.org).

    A Blight, A Curt, JF Ditunno, B Dobkin, P Ellaway, J Fawcett, M Fehlings, RG Grossman, DP Lammertse, A Privat, J Steeves, M Tuszynski, M Kalichman and JD Guest

    Mad

  • MS Treatment Pipeline: a New Dawn?

    It’s being heralded as a ‘new dawn’ for MS: Three new drugs have shown well in clinical trials; these would be the first significant medications that can be taken orally. A small trial using blood cord stem cells seems to be on to something. And the role of Vitamin D in MS is becoming clearer: high dose D answers the question why people in Canada get MS at so much a higher rate than those in Spain.

    First the drugs: Fingolimod is an oral immune system modulator that in just-released Phase III trial data kept 80 to 83 percent of multiple sclerosis patients relapse-free after a year of daily treatment. Phase III trials are ongoing. Because fingolimod is a potent immune system dampener, patients will be carefully monitored; some cases of skin cancer have occurred in the early trials.

    Another new oral med called laquinimod, now in Phase III trials, has already gotten a “fast track” designation from the FDA; the drug reduced MRI disease activity by a median of 60 percent compared with placebo. The drug does not turn down immunity; it therefore does not appear to have the side-effect worries the immune drugs have. Laquinimod appears to have a neuroprotective role. MSers are hoping this comes on the market in 2011.

    A third drug, cladribine, is taken orally just a few times a year yet can significantly reduce the chances of a relapse, with very few side effects. A recent British study noted that those taking cladribine tablets were over 55 per cent less likely to suffer a relapse and 30 per cent less likely to suffer worsening in their disability due to MS (compared to those taking placebo). Says lead researcher Gavin Giovannoni of Barts and The London School of Medicine and Dentistry, “These results are really exciting. Our study shows that cladribine tablets prevent relapses and slows down the progression of the disease making patients feel better. Importantly, it does so without the need for constant injections that are associated with unpleasant side effects.”

    Stem cells: just out in the Summer 2009 edition of Multiple Sclerosis Quarterly Report, three patients improved neurologic function after recharging their immune systems by way of blood stem cell transplants. The study involves wiping out the immune system through chemotherapy or radiation, thus destroying most blood cells and bone marrow. Blood stem cells, from the patient or a matched donor, are then transplanted into the patient. These cells are believed to repopulate the bone marrow and remake all the cell types found in the blood. These are early results but show promise for treating the disease.

    Vitamin D may be a major piece of the multiple sclerosis puzzle. It’s long been known that MS correlates to geography; the further from the equator, the higher the risk for MS. Why do people in Scotland, Canada and other northern countries have the highest incidence of multiple sclerosis in the world? According to several papers presented two weeks ago at the American Academy of Neurology annual meeting in Seattle, it’s all about the sun and the sunshine vitamin D. Here are several conclusions reached by clinical experiments: Vitamin D deficiency is a risk factor for MS. Vitamin D has a protective effect against MS development. High doses of vitamin D reduce relapse rates in people who have multiple sclerosis. Vitamin D is cheap and easy to get.

    Mad

  • Stem Cell Trial Gets Long-Awaited OK

    Geron gets nod for stem cell trial for acute SCI.

    It apparently took 10 months to wade through the 21,000-page application, and perhaps a regime change at the top of the United States government, but the U.S. Food and Drug Administration finally gave the go-ahead to the nation’s first embryonic stem cell clinical trial.

    The Geron Corp. has had data for several years showing that paralyzed rats got much better (they walked) after getting injected with a cell type generated from human embryonic stem cells. The company filed papers last March to move from lab animals to humans. The FDA, worried about safety, took its time with the process.

    Timing? What timing? President Barack Obama said in his inaugural address on Tuesday that he’d “restore science to its rightful place,” George Bush is off to Texas and the very next day Geron gets a phone call from the FDA with the good news. But as Geron announced the decision today both the company and the FDA said the timing was purely coincidental. Here’s an FDA source: "The FDA looks to the science on these types of issues, and we approve [such applications] based on a showing of safety," said Karen Riley. "Political considerations have no role in this process."

    Really? Science is now free from political pressure...now that is good news.

    At any rate, this will be the first embryonic stem cell trial coming out of the regulated and scientifically vetted mainstream (which does not include stem cell soloists in India and the Dominican Republic). Geron will enroll 10 patients, that is, 10 unsuspecting people who will experience spinal cord trauma in the coming months. The goal of this Phase I trial is to establish that these cells, already fated to become a neural support cell, won’t grow out of control and won’t cause an immune response in the cord that exacerbates the trauma.

    The work emerges from the lab of Hans Keirstead at the Reeve-Irvine Center in California. Read more here.

    This is terrific news for the Reeve Foundation. See the ABC news coverage, featuring Board Chair Peter Kiernan, who describes the FDA action as a comet in our sky.  Here’s Reeve Foundation CEO Peter Wilderotter on the news and its connection to the legacy of Chris Reeve.

    Interestingly, there was another big stem cell story earlier in the week. A British company called ReNeuron got approved by U.K. regulators to proceed with a stem cell treatment for people with ischemic stroke. The cells are not derived from embryos so there’s no political drama. Read more about that one here.
     
    Here’s the official Geron press release.

    Coverage, of course, is extensive. Wall Street Journal.
     
     And New Scientist.
     
     Mad

  • Reeve Act: Hope Comes Through the Backdoor

    On January 15 the U.S. Senate passed a big public lands bill emerging from the Senate Energy and Natural Resources Committee that places the Bill Clinton birthplace in Hope, Arkansas, on the National Historic Register.

    The omnibus bill also protects 290 million-year old fossilized animal tracks in the Robledo Mountains in Doña Ana County, New Mexico, and authorizes $40 million annually for landscaping and forest restoration projects that cover 50,000 acres.

    Oh, among more than 160 bills in this public lands package, the bill also authorizes the Christopher Reeve Paralysis Act, which has nothing to do with energy or natural resources but a lot to do with hope and human resources.

    Now it might seem that the Reeve bill is being sneaked through the back door, tacked on to a feel-good environmental bill, and that’s not entirely wrong. How and why this happens illustrates how business gets done in Washington, D.C. Nothing sneaky about it but there is an indirectness that leaves you to wonder: Would the Reeve bill have had the juice to make it on its own? Well, maybe so, with the new House and Senate coming to town, but this bill, in similar form, has been percolating in the nation’s capital for 10 years; kudos to Sens. Tom Harkin and Edward Kennedy, so for now let's skip the lessons in civics and legislative strategy and celebrate the fact it’s finally close to being real.

    See this link for more on the CRPA, and since something similar is brewing in the House, expect soon to see a bill on the desk of the new president to improve the health and well-being of people living with paralysis. It will save some fossil tracks, forests, waterways - and keep Hope afloat

    Mad.
  • Coming to Inauguration? Be Prepared

    The historic inauguration of Barack Obama as the new President of the United States is Tuesday, January 20. The day rings in a new administration, a new era in politics and a fresh outlook for the American people.

    Most of us will see this event unfold on television; some will keep up-to-the-second on Facebook or Twitter. Of course thousands of people coming in to Washington, D.C. have tickets, assuring some sort of seat away from the hoi polloi. For the masses of unconnected non-celebrities coming to town, the Mall is open to anyone, free. But expect a red alert in the area of inconvenience: it will be cold, crowded and lacking in creature comforts.

    Be prepared. If you have a ticket, see this: for ticket holders.

    The District has a good website set up on navigating the events of the day.

    The Washington Post has run a series of articles on getting to and from the inauguration. See Inauguration central.

    If you are old or have a disability, expect to camp out for a long day. In other words, be a scout and know your limitations. The D.C. Office on Aging has a handout (below) you must read if you want to take your chances seeing the inauguration. Bring your own food, medications, water and toilet paper. And don’t forget the camera.

    Mad

    Plan for Persons with Disabilities:
    The organizers and transportation officials say people with disabilities and the elderly should expect long delays in getting to the National Mall, parade route and swearing-in ceremony. They are advising everyone to plan ahead and have a backup plan. Here's a highlight of what to expect on Jan. 20:
    • No cars with disability tags or license plates will be allowed to park around the Capitol on Inauguration Day.
    • People with disabilities attending the swearing-in ceremony may be dropped off at South Capitol and E Street and North Capitol and E Street, where golf carts will be available to transport them to security check points.
    • Canes, including those with a fold-down seat, walkers and scooters are allowed at the swearing-in ceremony.
    • There will be raised platforms for wheelchair users in the seating areas at the Capitol, but space is limited. Similar platforms will be on the National Mall, too.
    • Americans with Disabilities Act-compliant bleachers will be available along the parade route for people with disabilities who have parade tickets and those who don't. 
    • Sign language interpreters will be in different sections at the swearing-in and along the parade route. Open captioning for the deaf and hard of hearing will be available on large TV screens on the Mall and parade route. 
    • Audio description services will be available for the blind and those with limited vision at the swearing-in ceremony and the parade. 
    • Thirteen entry points for the parade will open at 7 a.m. Jan. 20. All will accommodate people with disabilities. 
    • MetroAccess, the Metro transit system's subscription service for people with disabilities, will operate its regular schedule on Jan. 20, but there will not be service to inauguration venues or limited or cancelled service to downtown areas. Door-to-door service will be limited. Customers should expect major delays and to maneuver long distances on their own.
    • Access to elevators in Metro's train stations will probably be limited because of crowds. Escalators won't be operating in certain stations. 
    • Wheelchair-accessible port-a-potties will be available along the parade route, National Mall and Capitol grounds.
  • Stem Cell Advisory: Buyer Beware

    Cautionary stem cell tourism guidelines came out earlier this month, taking aim at overseas clinics characterized by unsupported expectations, overhyped therapies and uninformed risk.

    The new guidelines originated from the International Society for Stem Cell Research. (Mission statement: “an independent, nonprofit organization … to promote professional and public education in all areas of stem cell research and application.”)

    From a press release: “The ISSCR is deeply concerned about the potential physical, psychological, and financial harm to patients who pursue unproven stem cell-based ‘therapies’ and the general lack of scientific transparency and professional accountability of those engaged in these activities.”

    In other words, the stem cell field is worried about people getting ripped off or getting harmed. They are also worried about the bigger picture. This is from a piece on Bloomberg:

    “We’re worried about patients and we are worried about the field,” said George Daley, a researcher at the Harvard Stem Cell Institute and member of the task force that wrote the guidelines, in a telephone interview. “The field is at risk from renegade, illegitimate practitioners. If there is a public perception that the science isn’t being done carefully then we’re at risk for losing public support.”Of course anyone considering a trip to India or China or to any of the countries that offer stem cells should read this material and take it seriously.

    Of course even though this represents the considerable opinion of the world’s cell biology brain trust, the guidelines may do little to deter those who see no hope in the choices of approved therapies available at home, and who have little patience that the U.S. stem cell promise will be realized any time soon.

    I didn’t find much buzz about the latest stem cell caveat emptor at the Internet cure hangouts. The news was pretty well disseminated by the mainstream media, with some pithy headlines, like these:
    Beware of Bogus Clinics Offering Stem Cell Cures
    "Rogue" stem cell clinics exploit hope: report

    An ISSCR team from 13 countries drafted the guidelines over the past year and honed them into two documents, Guidelines for the Clinical Translation of Stem Cells, which looks at the scientific, clinical, regulatory, ethical and social justice issues that must be ironed out to move basic stem cell research toward clinical applications.

    Simultaneously, the group released a consumer-oriented Patient Handbook on Stem Cell Therapies. This is the one to read if you’re thinking about getting shot up with stem cells – it sets forth a primer on stem cells and their limited use, on how clinical trials work, and asks the questions that must be answered to know whether you are getting an expensive and exploitative experiment or a real therapy.

    The ISSCR suggests this checklist for people considering a trip abroad for stem cells. Ask for:
    • Evidence of published pre-clinical studies that have been reviewed -- and repeated -- by experts in the field (need it be said, these peer reviewers aren’t allowed to work for the clinic).
    • Evidence that the provider has ethical approval from an independent committee (Good luck with this in China or India).
    • Evidence that the provider has national or regional regulatory approval. In the UK, this is the European Medicines Agency (EMEA).

    The ISSCR warns that certain claims made by providers of stem cell therapies should sound alarm bells:

    • Claims based on patient testimonials (this is the basis of all Internet hopefulness; self-reported effects of any treatment are notoriously inconsistent and unreliable).
    • Claims that multiple diseases or conditions are treatable with the same cells (there is no cell line or cell cocktail that is the universal treatment for say, Parkinson’s and heart disease).
    • Claims regarding the source of cells or treatment details (you might want to know how well screened the cells are for viruses and pathogens – also, where exactly did they come from).
    • Claims that there is no risk. Note: there is risk rolling into a foreign clinic; there is risk with any surgery, with any injection.
    • High cost treatments or those where the true cost is hidden.
    Until the overseas stem cell clinics answer basic questions, they must be considered human laboratories. If the treatments do indeed work, the medical technology must be shared. It’s up to the clinics to make their unsavory reputations go away. Kudos to ISSCR for taking a strong stand.

    Mad

  • A drug to fire-up your patterns?

    The recent Society for Neuroscience meeting in Washington, DC, showcased a huge range of work that, in the whole, leaves little doubt that regenerative medicine and functional recovery are moving forward.

    (A tip of the MadWire hat to Steven Edwards, a C3 quad from South Carolina and a moderator for the CareCure community who compiled a long list of the SCI related papers.)

    Even if you have little tolerance for the academic nature of the writing, it’s possible to tease out quite a bit of optimism from Edwards’ list. Here is a paper that caught my eye, a) because it’s aggressively intended to go to clinic soon; b) it was funded in part by the Reeve Foundation; and c) because it sounds like a pill to make you walk.

    The formal title is “Pre-clinical and clinical development of a drug treatment for Central Pattern Generator (CPG) activation and 'reflex' stepping induction.” A group from Quebec led by Pierre Guertin began with the body of evidence that stepping motions in paralyzed animals are controlled not by brain input but in the spinal cord by a central pattern generator.

    The role of the CPG is central to the field of activity based rehabilitation (e.g. the Reeve Foundation NeuroRecovery Network, which has in many people been able to activate the CPG with repetitive stepping patterns using weight-supported treadmill therapy).

    Guertin, who has already set up a company in Quebec called NeuroSpina Therapeutics Inc. to take his early lab results to the clinic, went fishing for the molecular basis of CPG activation. Using a mouse model his group tried what he calls “a plethora of serotonergic, glutamatergic, adrenergic and dopaminergic ligands over the years.” Some of the drugs worked to induce rhythmic movements, flexion and extension, but when it came time to put weight on the hindlimbs, they had no success with true stepping.

    So, using the skills of a bartender, Guertin mixed a drug cocktail (based on role of serotonin receptors) he has since patented and named Spinalon. He describes this drug as “First-in class activator of the Central Pattern Generator for locomotion. It acutely evokes, upon systemic delivery, repetitive walking movements for one hour in chronic and complete SCI subjects.” OK then, for the record, a drug to make your legs move in the pattern of walking.

    Says Guertin, it appears to work. Weight bearing stepping has occurred in animals, and he’s gone on to try the drug in at least one human: “The first pilot test in patients was done recently. A monoplegic man who received Spinalon every 2 days for 2 weeks showed no atypical side effects providing first preliminary evidence of safety in men (Spinal Cord, in press).”

    You might join me in wondering how a scientist can go from yet-to-be published data on humans to a full-on clinical trial, but Guertin is undeterred. People are already being recruited to test Spinalon, he says. Here's to everyone's patterns and to their continued generation. 

    Mad
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