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לארשי םירירש תולחמ-י.ש.מ םורופ

תועדוה
(דומעה שארב תועיפומ םורופב תונורחאה תועדוהה)

תועדוהה לכ תא רוגס

תועדוהה לכ תא חתפ

(11:54 28/04/11) ומ הרימ הלוח לש םידליל הכימת תצובק

(12:07 04/05/11) קחציו הליא םילוח לש םידליל הכימת תוצובק

(18:07 24/04/11) יודצק מבל POSTIVE RESULTS IN DMD PROGRAMME: • SMT C1100 increases utro

Summit Corporation plc
(“Summit” or “the Company”)
POSTIVE RESULTS IN DMD PROGRAMME:
• SMT C1100 increases utrophin in DMD patient muscle cells to
therapeutically beneficial levels
Oxford, UK, 14 March 2011, Summit (AIM: SUMM), a UK drug discovery company, today
announces positive non-clinical efficacy results for SMT C1100, an orally available drug that
has the potential to be a disease modifying treatment for the fatal disorder Duchenne
muscular dystrophy (‘DMD’).
DMD is caused by the loss of a protein called dystrophin which results in the degeneration of
all skeletal muscles and causes damage to the heart. Currently there is no cure for this
disease. Scientists have previously shown that by increasing (upregulating) production in the
body of a similar, naturally occurring protein called utrophin, it can compensate for the
missing dystrophin and can restore and maintain healthy muscle function.
Summit is pleased to report that new results from studies evaluating the effect of SMT C1100
on dystrophin deficient muscle cells taken from DMD patients have been positive. Dosing of
these human myoblast cells with only low concentrations of SMT C1100 resulted in increased
utrophin protein levels, which if translated into DMD patients, are anticipated to be of
significant therapeutic benefit.
The studies were conducted at Oxford University by Professor Dame Kay Davies FRS, a
world-leading expert who pioneered utrophin upregulation as a therapeutic approach for
DMD. Commenting on the results, Professor Davies said “The use of utrophin protein to
substitute for the missing dystrophin is an approach that has the potential to benefit all DMD
patients, regardless of their genetic mutation. It is also expected to be complementary to the
other therapeutic approaches in development. These significant results provide further
encouragement about the potential of SMT C1100 as a utrophin upregulator drug.”
A leading DMD clinician, Francesco Muntoni, Professor of Paediatric Neurology and Head of
the Dubowitz Neuromuscular Centre at the UCL Institute of Child Health in London added:
“These results in DMD muscle cells demonstrate that SMT C1100 can increase utrophin to
levels, which if replicated in DMD patients, would undoubtedly make it a disease modifying
therapy.”
The results will be presented at the Muscular Dystrophy Association’s National Scientific
Conference being held in Las Vegas, US on March 13-16 2011.
Enhancing a compelling data package
These new data supplement a compelling data package generated from a range of earlier
studies, including those conducted in the ‘gold standard’ in vivo model. These showed that
SMT C1100:
• Increases the amount of utrophin and reduces muscle degeneration, fibrosis and chronic
inflammation.
• Significantly improves resistance to muscle fatigue in a forced exercise model. This is a
surrogate for the six minute distance walk test which is the accepted primary efficacy
end-point in human clinical trials.
In a healthy volunteer clinical trial, SMT C1100 was shown to be safe and well tolerated with
no adverse events reported. In addition, blood plasma levels of SMT C1100 did exceed
levels that are anticipated to produce a therapeutic effect in some individuals, although there
was variability in the results with lower exposure levels reported for others. Summit believe
alternative formulations of SMT C1100 can produce consistently higher results and is seeking
to evaluate these in future clinical studies.
Commenting on the results, Dr Barry Price, Executive Chairman of Summit said: “These
results are important as they show treatment of DMD patient muscle cells with SMT C1100
increases utrophin protein to levels that we anticipate will have significant therapeutic benefit
if replicated in patients. These data reinforce Summit’s belief about the potential of SMT
C1100 as a first-in-class disease modifying treatment for all DMD patients.”
- END -
For more information, please contact:
Summit
Barry Price, PhD,
Richard Pye, PhD Tel: +44 (0)1235 443 939
Singer Capital Markets (Nominated Adviser)
Shaun Dobson / Claes Spång Tel: +44 (0)20 3205 7500
Peckwater PR
Tarquin Edwards Tel: +44 (0)7879 458 364
tarquin.edwards@peckwaterpr.co.uk
Notes to Editors
About Duchenne Muscular Dystrophy and Utrophin Upregulation
Duchenne muscular dystrophy is a fatal neuromuscular disorder that affects 1 in 3,500 boys
with an estimated patient population of over 40,000 in the developed world.
DMD is caused by a genetic defect meaning DMD patients lack an important protein called
dystrophin, which is crucial to maintaining muscle integrity and function. The absence of
dystrophin results in extensive muscle wasting in all voluntary muscles as well as the heart
and breathing muscles and causes severe restriction in the mobility of DMD patients by their
early teens and is ultimately fatal, generally in their twenties. Currently there is no cure for
DMD; corticosteroid treatment is the only frontline therapy and acts to only delay the
progression of the disease.
Summit has identified SMT C1100, a proprietary, orally available small molecule with a novel
mechanism of action for DMD. SMT C1100 acts to modify the progression of DMD by
replacing dystrophin with an endogenous, functionally similar protein called utrophin. Summit
believes the primary advantage of SMT C1100 is that it offers the potential to treat the entire
DMD patient population.
Due to the low patient population and high unmet medical need, DMD is designated as an
orphan indication by the regulatory agencies. Orphan products can expect to receive
additional regulatory support and accelerated approval in addition to seven and ten years of
market exclusivity in the US and EU respectively upon designation by the FDA and the
EMEA.
About Summit
Summit is an Oxford, UK based drug discovery company with an innovative technology
platform called Seglins™ for the discovery of new medicines, a portfolio of drug programme
assets and a commercial strategy of signing multiple early-stage deals.
Seglin™ technology is using new chemistry to access biological drug targets that cannot be
exploited by conventional drug discovery approaches. Summit’s internal research is currently
focussed in the high-value therapy areas and the Company will further exploit the
technology’s wider potential through strategic alliances. Summit’s programme portfolio
consists of a number of drug programmes targeting high-value areas of unmet medical need
including Duchenne muscular dystrophy and C. difficile infections.
Summit’s commercial strategy focuses on signing multiple early-stage drug programme and
technology platform deals that generate upfront cash, remove development costs from the
Company, and retain valuable upside potential.
Summit is listed on the AIM market of the London Stock Exchange and trades under the ticker
symbol SUMM. Further information is available at www.summitplc.com.
Forward Looking Statements
This document contains "forward-looking statements" within the meaning of the U.S. Private
Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by
words such as "anticipates", "intends", "plans", "seeks", "believes", "estimates", "expects" and
similar references to future periods, or by the inclusion of forecasts or projections.
Forward-looking statements are based on the Company's current expectations and
assumptions regarding our business, the economy and other future conditions. Because
forward-looking statements relate to the future, by their nature, they are subject to inherent
uncertainties, risks and changes in circumstances that are difficult to predict. The Company's
actual results may differ materially from those contemplated by the forward-looking
statements. The Company cautions you therefore that you should not rely on any of these
forward-looking statements as statements of historical fact or as guarantees or assurances of
future performance. Important factors that could cause actual results to differ materially from
those in the forward-looking statements and regional, national, global political, economic,
business, competitive, market and regulatory conditions.

רשק רוצ

העדוה ספדה הכנת ההודעה להדפסה

רבחל חלש

הבוגת

:תויורשפא

(08:39 11/04/11) איבל תילגו ןתנ לחר םינשה ךורא וננויסנמ

תלבוסש יתיב תילגו ינא רשאכ .םישוע םתאש רבד לכ לע וכרובת
וליא ....וליאה הלוע דימת ,תוחחושמ 31 תב םויה םירירש ןווינמ
עדומ היה תורדרדהה בצממ הברה םויה םיעדויש המ זא םיעדוי ויה
,םכלוכ לש לבה תמושתל איבהל תשקבמ ינאו .םאתהב הנעמ לבקמו
תושעל תוילוח רושייל חותינב ךרוצ שי םא המהמתהל אל בגב תמקע
חתנל וצר אל םיאפורש תילג ומכ ןיתמהל אל .דעומ דועבמ תאז
26ליגל עיגה איהשכ ףידע המ .יאדכ אלו ןכוסמ הזש וריבסהו
היה וניצרש רבעבש יתמישנ דוקפית 25% .יטירק היה בצמה זאו
תורציה בקע תיתאר המיסח הרצונ תילג רובע ןכוסמ היה בצמה .60%
.ובבותסה תוילוחה ,תמקעה ללגב תונופמיסה
תילגל ,תרמשמ היפרטויזיפ ילופיטל תועדומ התייה אל ףסונ רבד
םישוע ויה וליא .אסיכב תכשוממ הבישי בקע תובר תויורצקתה
רפסמ תכלל הלכיש ןכתי תימוקישו תענומ היפרטויזיפ םירשאמ
בלשב דחא לכ ףקות יתמישנ דוקפיתב ישוקה רתויב בושחהו .םידעצ
היפרתויזיפ לע תטאיצומ ונא ףסכ הברה יל ונימאה .םייוסמ
םירושיאו הלק אל המחלמ וז האפרמהמ לבקל .יטרפ ןפואב תיתמחשנ
הבטהל רושיא לע םחלהל וליחתה השקבב .לבגומ רפסמב םהש
שי םנמוא .סיזורביפ קיטסיסס ילוח ומכ תיתמישנ היפרטויזיפב
םדקתמ בצמב יל ונימאה ,יתמישנ ישוקו החיל םע תידימת היעב םהל
העפות התוא תא שי ,רירש וא בצע הנשמ אלו םירירש ןווינ לש
רישכמל תרבוחמ תונריעב תועש רפסמו הנשי תילג םינש 6 הזמו
היהש םייחה דוקפת תא ליבגמ הז לכ .המישנב הל רזועש פפייב
פפייבה לעשמה םע תוכלוה ונא םוקמ לכל רתויו רתוי רבעב
ץיפהל רשפא הדות .הז יאנת תוושהל הז לע ומחליה ןכל ...ןשקסה
.ןמזומ רוזעל לוכיש ימ לכו הז בתכמ
ירעצלו 31 תב תילג םויה.
ירפ תואשונ םויהש רבעב יתישעש תובר תומחלממ הפייע השיגרמ ינא
לע הדות .רשוא אל רבעבש דועו םיאתמ בכרל רושכימל םירושיאב
ינאש המ רבד שי םא ,םכלוכ לש היישעה לכ לע!!!חוכ רשי לכה
חמשא ןויסינהמ רוזעל הלוכי
תילגו לחר ,ישפוחו חמש גח תכרבב

רשק רוצ

העדוה ספדה הכנת ההודעה להדפסה

רבחל חלש

הבוגת

:תויורשפא

(21:37 09/03/11) ןהכ הרעי התומעה ןעמל המרתה םוי

(08:59 11/03/11) קחציו הליא עבש ראב 'א ףיקמ ןוכית המרתה םוי

(07:39 01/03/11) קחציו הליא השדח הצובק חתופ זכרמב םילגלגה תיב

2011 ראוני

!םולש םידלילו םירוהל

םירגבתמו םידליל רישעמו חמש יתרבח תיב אוה "םילגלגה תיב"
םילבוסה
םייחב ףייכו החמש םידליל קינעמ תיבה .תודלומ תויזיפ תויוכנמ
תויונמוימו
הביבסה לוהינל םילכ םידליל תתל איה הרטמה .תוישיאו תויתרבח
םהלש
תונווגמ תויתרבח תויוליעפ ידכ ךות תישיא המצוע ךותמ
.תויעוצקמו

םידלי לש תועובק תוצובקב ץיק תונחמו עובש יפוסמ םינהנ םידליה
.םיכירדמו
בולישל תוינכות ,עובשה ךלהמב םיגוח לש תכרעמ םהל שי ,ףסונב
,הליהקב
םיישק םתוא תא םיווחה םידלי שוגפל תונמדזהו םיעוריא ,תוביסמ
םידליהש
."םייניעה הבוגב" םילועמ םיכירדמו םתיא םידדומתמ םכלש

תונמדזה "םילגלגה תיב" הווהמ םיליגר רפס יתבב םיבלושמה םידליל
תווחל
הרבחב - םוי לכ םיווח םהש םישק םיצחלו תורחת אלל םתוכנ תא
,החמש
.תיתרבחו תישיא תכמותו הליעפ
,םירוהה ,םכרובע םיווהמ "םילגלגה תיב"ב תונטייקהו עובשה יפוס
תונמדזה
הלבמ םכדליש ןמזב החפשמה ינב ראשלו םכמצעל חוכו ןמז שידקהל
.החמשו תרכומ הרבחב
השימחב םינש 30 מ רתוי הזמ תלעופה התומע הניה "םילגלגה תיב"
ינב ,םידלי 350 מ רתויל תורש םינתונ ונחנא .ץראה יבחרב םיפינס
רעונ
.הייסולכואה ירזגמ לכמ םירגובו

םלוכ ,םיבדנתמ 400 מ רתוי לע ותוליעפ תא ססבמ "םילגלגה תיב"
ירחא
םידליה תא םישגופ םיבדנתמה .תוחפל םייתנשל םיבדנתמ םלוכו אבצ
תיתרבח תרגסמב ,דובכו םוח הבהא םהל םיקינעמו םיווש םע םיוושכ
;#8211&
.תיכוניח

13 דע 10 יאליגב םידלי לש השדח הצובק םיחתופ ונחנא בורקה ץיקב
.םינש
.זכרמה רוזאמ םידלי טולקתו הילצרהב זכרמ ףינסב חתפת הצובקה
ונחנא .דבלב םידלי 15 הנומו םייתנשל תחא תחתפנ תאזכ הצובק
םילבקמ
םיבלושמה םידליל תופידע ןתנית .ןיקת יביטינגוק דוקפת םע םידלי

.םיליגר רפס יתבב

:ב ונילע םיפסונ םיטרפ לבקל ןתינ
http://www.beitgalgalim.org.il/

תא דחא ריכהל לכונש תנמ לע רשק ונתיא רוציל םכתא ןימזמ ינא
.ינשה

:ליימב וא 0528384666 :דיינב רשק יתיא רוציל ןתינ
eli@beitgalgalim.org.il

החמשבו דובכב
ןילביר ילא
זכרמ - םילגלגה תיב להנמ

http://052-8384666 :הוולנ רושיק

רשק רוצ

העדוה ספדה הכנת ההודעה להדפסה

רבחל חלש

הבוגת

:תויורשפא

(01:34 09/02/11) yudchak mabel Tivorsan Pharmaceuticals’ Biglycan Protein Opens Unique Path

Study published in PNAS shows recombinant human biglycan reduces muscle damage and improves function in mouse model of DMD

Providence, RI—December 28, 2010—A novel therapy under development by Tivorsan Pharmaceuticals demonstrated reduction of muscle pathology and improved muscle function in a mouse model of Duchenne Muscular Dystrophy (DMD). The company is actively working towards human clinical studies with a proprietary form of the natural biglycan protein, which appears to have a unique mechanism of action that may apply to all genetic forms of DMD.

In this study, injections of recombinant human biglycan (rhBGN) reduced dystrophic pathology and improved muscle function in mice with the same mutation that affects DMD boys. The study was conducted by Tivorsan’s founding scientist, Dr. Justin Fallon of Brown University, and colleagues. These findings are reported in the December 27 online version of the Proceeds of the National Academy of Sciences.

Duchenne Muscular Dystrophy (DMD) is an unrelenting muscle wasting disorder that leads to death in early adulthood. Affecting approximately 1 in 3,500 newborn boys, this most common of X-linked diseases is triggered by mutations of the gene encoding the dystrophin protein. In normal adults, dystrophin keeps muscles strong, but DMD mutations switch off dystrophin production so that affected boys gradually and permanently lose muscle strength. No current treatments successfully address the biologic course of the disease.

“Dr. Fallon and his colleagues have shown us that we have a biologically active molecule with an apparently unique mechanism of action that may be applicable to all genetic forms of DMD.” said Dr. Joel Braunstein, co-founding partner at LifeTech Research, member of the board of directors and acting CEO at Tivorsan. “Most importantly, biglycan shows potential to slow muscle wasting and modify the progressive course of the disease. The study also suggests that simple injections may have a durable effect on muscle strength.”

“Multiple lines of evidence make biglycan a compelling candidate for moving into human testing as soon as possible. Our team recognizes a special opportunity to help many children and their families suffering from this disease, and therefore we are working hard to advance an optimized form of the biglycan protein through production and preclinical testing into the clinic.”
About biglycan

Biglycan induces the expression in the muscle cell membrane (sarcolemma) of a fetal protein called utrophin that is typically replaced in adults by dystrophin. Thus, utrophin offers an alternative pathway to maintaining the integrity of the muscle cell membrane in DMD boys. Biglycan acts through an apparently unique mechanism of action, distinct from other treatments in development, yet potentially suitable for concurrent use with such treatments. In this study, systemically administered biglycan was well-tolerated and remained effective for approximately three weeks following a one-time dosing. Therapeutic effects persisted out to three months following dosing once every three weeks.
About Tivorsan

Tivorsan Pharmaceuticals http://www.tivorsan.com is a protein therapeutics company pioneering a unique approach to treating serious neuromuscular disorders, including DMD and Becker Muscular Dystrophy (BMD). This method, using recombinant human biglycan (rhBGN), is based on 24 years of basic science work in the Fallon laboratory at Brown University (http://neuroscience.brown.edu/fallon). Tivorsan was formed by Dr. Fallon in collaboration with colleagues from Old Forge Holdings of Greenwich, CT and LifeTech Research, a Baltimore-based technology research and development firm (http://lifetechresearch.com). Early support for the Tivorsan program originated, in part, from Federal sources, as well as Charley’s Fund (http://charleysfund.com) and the Nash Avery Foundation (http://nashaveryfoundation.org), two philanthropies seeking to accelerate the development of cures for DMD.
Contact

Ellen M Martin
Feldman Stakeholder Relations
emm4@pacbell.net
510 832 2044

Dr Joel Braunstein,
Tivorsan Pharmaceuticals
jbraun@tivorsan.com
410-410-4191

You may also read this on Business Wire.

http://http://www.tivorsan.com/2010/12/tivorsan-pharmaceuticals%E2%80%99-biglycan-protein-opens-unique-path-for-preserving-muscle-function-in-duchenne-muscular-dystrophy/ :הוולנ רושיק

רשק רוצ

העדוה ספדה הכנת ההודעה להדפסה

רבחל חלש

הבוגת

:תויורשפא

(12:14 02/12/10) א תור בוטה ינויסינמ

(15:32 20/12/10) ירמד הקבר קבדיפויב

(20:30 22/12/10) קחציו הליא םיטרפ רתוי תעדל וניצר

(10:58 24/12/10) א תור הבושת

(14:35 27/12/10) קחציו הליא הבוגת

(10:09 28/12/10) א תור ףסונ ןויסינ

(09:18 06/01/11) א תור לופיטה יטרפל םינופה תשקבל

(07:44 28/12/10) yudchak mabel Alternative therapy for Duchenne and Becker - should we beli

(20:25 22/12/10) שורב לכימ ופרל הטלוקפה ,יאופר ךוניחל גוחה - החפשמה טקיורפ - הרזעל השקב

,בר םולש החפשמל

טקיורפל ונילא ףרטצהל תונוכנהו תוניינעתהה לע הבר הדות
,"תוחפשממ םידמול האופרל םיטנדוטס ;#8211& ילוח םע תודדומתה"
םע תודדומתה לש תועמשמה המ דיתעל םיאפורה תא דמלל ותרטמש
לע אלא הלוחה לע קר אל ,ןמז ךרואל תוקל/הלבגמ/תינורכ הלחמ
.הלוכ החפשמה

'ב הנשב האופרל םיטנדוטסל הבוח הניה הז טקיורפב תופתתשהה
.ביבא-לת תטיסרבינואב
טבמה תדוקנ תא ןיבמה ,ישונאו בוט םיאפור רוד ךנחל םישקבמ ונא
.תויאופר תויעב םע םידדומתמה תוחפשמהו םילוחה לש

םיטנדוטסה תא דמלל םילוכי םתאש םושמ םכתרזעל םיקוקז ונחנא
.דיתעב אפורה תומדל םורתלו

?הז טקיורפב תופתתשהה תועמשמ המ
דעו רבמצד שדוחמ לחה םכתיבל עיגי האופרל תי/טנדוטס ;#8226&
3 לעו םומינימ תושיגפ 5) תושיגפ 8 ;#8211& 5 ל ינוי שדוח
.(םינושארה םישגפמה רחאל בחרויו רבסוי תופסונ תוירשפא תושיגפ

םע דדומתמה החפשמה תב/ןב תא שוגפת/שוגפי תי/טנדוטסה ;#8226&
,הברק תגרד לכמ - 2 תוחפל - םיפסונ החפשמ ינבו תיאופרה היעבה
.החפשמה תב/ןב לש ילוחה םע םהלש תודדומתהב ותוא ופתשיש
םילוכי םתאו םכתא םדקומ םואת רחאל ומייקתי םישגפמה ;#8226&
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ביבא-לת תטיסרבינוא

רשק רוצ

העדוה ספדה הכנת ההודעה להדפסה

רבחל חלש

הבוגת

:תויורשפא

(08:25 21/12/10) א תור םיטרפ ירמד הקברל

(16:20 15/12/10) בודיוד המענ עגמ ילופיט

(09:27 04/12/10) yudchak mabel Video Testimonials

(08:47 04/12/10) yudchak mabel South Texas Innovative Medicine

November 30, 2010 Thanksgiving: A Day of Unbelievable Gratitude

Four months ago, little did our family have an inkling of what was about to happen in our lives. What started out as a typical, boring afternoon (for Todd Harrison who lives with Becker Muscular Dystrophy) ended up being a breakthrough toward a treatment for M.D, one of which we (parents and uncle) had thought it just wasn’t going to happen anytime soon. Thank God for the NEW generation. Thank God that Todd had not resigned himself to “accepting” his condition as the final chapter in his life. As Todd searched through Youtube videos on the topic of Muscular Dystrophy, he came across a single video that depicted two boys who had been receiving treatment. He viewed it with amazement as he witnessed “before” and “after” treatment. In that instant, Todd KNEW this was something he needed. He was quite simply: Compelled. Immediate. No reservation. He alerted his parents whom viewed the video with typical apathy and skepticm. Never actually verbalizing, it but feeling it inward.

So, a couple of weeks passed and Todd continued to refer to the video. He continued to research and correspond with the doctor by email. Finally, one day, he simply pronounced he was going to travel to Texas. Period. There is something that is awe inspiring that occurs when one makes such a pronouncement. To deny him the opportunity seemed blatantly wrong. How could anyone tell him, “no.” It was at this point, Todd himself, called Dr. Donald Rhodes in Texas and asked when the next available appointment was. Bam, he made the appointment. Here’s a little background on Todd before treatment: Quiet. Shy. Reserved. It was the first time in his young adult life, that he made a decision, planned for execution of the decision, and pronounced it. (As parents, we were proud, yet hoping this would not be setting him up for disaster as previous M.D. treatment opportunities has proved). Soon after, Todd phoned his Uncle Dave and relayed that he was going to “Texas.” Uncle Dave thought, “okay, good deal. Check it out, Todd.” He certainly didn’t think it was appropriate or possible to travel with Todd at this time. He has a printing business and is the sole designer. No way, he could leave town. And then of course, there was the financial reality.

However, something transpired over the next few weeks that was nothing short of miraculous. Within days, David felt compelled that he also had to make this trip. Again, once a decision is made, something larger kicks in…. faith. With no immediate plan in place for funding, David also made his appointment with Dr. Rhodes. Our family had 3 weeks to come up with funding. Crazy? Yes! Absolutely! Within days, fundraising plans were created and travel arrangements booked. Defying Muscular Dystrophy Facebook page was created, a website with paypal donation button enacted, and the donations rolled in! To date, over $14,000.00 was raised and together with personal financial resources, Todd and David traveled to Texas. And the rest is facebook history.

The gains that Todd and David have and are continuing to make simply ‘DEFY’ logic. How long will this last? The story is yet to be written. But for now, for today on this day of THANKSGIVING…. We are most thankful for the HOPE that was ignited in Todd. THANK God for the younger generations who still possess the hope for a better tomorrow. And the belief in seeing a single video that spoke to his soul. HAPPY THANKSGIVING!

Follow Defying Muscular Dystrophy on Facebook.

October 24, 2010

I recently received the newsletter from PPMD regarding the treatment utilized in our clinic. Several questions were posed and we welcome the opportunity to address these valid concerns.

1. What experience does the investigator have with Duchenne?

Before my first patient with DMD nearly 3 years ago, I had no experience with this disease. However, I have dedicated most of my career to the research and treatment of other chronic diseases exacerbated by oxidative stress. Initially my research and treatment focused on patients with Reflex Sympathetic Dystrophy Syndrome (also known as CRPS). Eventually my work led me to diabetes, Parkinson’s Disease, and now with Muscular Dystrophy. I have come to the opinion that oxidative stress is a common link between these diseases.

All of these diseases share pathological changes in the body which can be traced directly or indirectly to a lack of oxygen at the cellular level throughout the body. As a Podiatrist, I have been trained to understand pathologies in the feet. In all of these diseases, oxidative stress shows up first and worst in the feet due to the distance from the heart and central nervous system.

Oxidative stress in Muscular Dystrophy patients is apparent by:

* Diminished skin temperature in the hands and feet, particularly in the fingers and toes
* Increased capillary filling time in the toes
* Diminished circulation in the digital pulp of the toes in the presence of adequate circulation into the foot
* Changes in the bones shown by X-rays
* Diminished nervous system responses

The children with Muscular Dystrophy, who have been examined in our clinic, have all shown decreased foot skin temperatures, increased toe capillary filling times, decreased circulation in the toes while having normal circulation into the feet, subchondral cystic degeneration, and increased Vibration Perception Thresholds.

Of further interest, our clinic previously participated in small study with Diabetes Type 2. These patients showed the same circulation, nerve, and bone changes seen in these children with DMD. At the completion of the study, many of these patients demonstrated improvement and in some cases, complete resolution of these problems.

2. What empirical (observed or experimental) and objective evidence supports this treatment in Duchenne?

As we have stated before on our website, we have had a limited number of children utilizing this treatment but many are experiencing good results from the treatment. Subjectively, patients and their caregivers have reported improvements in strength, movement, balance, sleep, breathing and other areas affecting their quality of life. Objectively, we are getting reports from physical therapists of these patients reporting increases in strength, flexibility and range of motion. We have also had reports of decreased calf measurements.

In addition, with VECTTOR treatment, the parents of the children with DMD uniformly note that their children’s hands and feet are warmer. This is verified and quantified by Temp-Touch evaluation and is certainly suggestive of decreased oxidative stress.

3. What clinical (strength testing, gait pattern, Pulmonary Function Tests (PFT), walk endurance if ambulatory, etc.), imaging (MRI etc), and laboratory (e.g. CPK levels) outcomes are used to document improvement?

Since this is very new, we do not yet have a standard set of tests. We would welcome any assistance from PPMD or any other organization as far as their recommendations or suggestions of what tests might help us better assess the patients' progress.

So far, only two blood tests of children with DMD before and immediately after the initial VECTTOR treatment have been obtained. Both tests demonstrated a significant decrease of TNF alpha. We have had two patients who have had CPK levels before and after several months of treatment. Both of these children have shown significant decreases. Since the exact parameters of these tests are so variable, we are reluctant to say anything other than that, while the results are encouraging, like everything else, they need further evaluation.

An MRI was performed on the first patient with DMD following 6 months of VECTTOR treatment and then again 1 year after that. While the follow-up MRI showed encouraging results, further studies are needed.

We have been informally assessing the patients’ progress in the clinic with video documentation. We realize that this is not scientific and we welcome the opportunity to improve our assessment procedures.

Finally, it is our understanding that some of these children have been receiving standardized independent examinations. We will certainly share the results of these evaluations as we are able.

4. How many Duchenne patients have used this treatment?

35

5. Was it tested in animal models?

No

6. What is the longest course of treatment to date without adverse effects?

As of today the longest course of treatment has been 2.5 years. We have had no adverse effects reported.

7. Have safety trials been conducted and what were the outcomes? What are the potential dangers and or side effects?

Electro stim has been used in various forms for thousands of years starting with the Romans who used electrical stimulation for headaches (electric eels). Interferential stimulation has been utilized in the United States, since the 1950’s. There are no known side effects to interferential treatment and only a few contraindications.

8. What percentage of patients demonstrates improvement and what percentage does not?

We are still working on collecting data and I don't want to misspeak. I can safely say that a large percentage of the boys have noticed at least some improvement in the areas mentioned above. However, a small percentage of boys have reported no noticeable improvement. We hope to have the results from our informal data on the website within the next few weeks.

9. Is there data from independent sources, not just anecdotal reports from a single practitioner or clinic that uses comparisons with control groups to rule out placebo and coaching effects?

Because this treatment is so new, there hasn't been time enough to compile the evidence that is needed to validate the efficacy of the treatment.

We would again like to reiterate our willingness for the opportunity to cooperate in a study with PPMD or any other organization that would like to work with us. We realize that there is some frustration with the fact that the units can only be distributed from our office. There is just no way around that. Until final FDA approval, we can only distribute these beta models from our clinic.

10. Have other investigators used this therapy and confirmed its effectiveness? Has there been any independent review of data?

Not yet, however, as stated above, we look forward to the opportunity to work in cooperation with pro-active organizations such as PPMD who are willing to evaluate and review the effectiveness of the treatment.

One important general comment is that we do not believe it is advisable for patients to discontinue or interrupt existing treatments or medications such as corticosteroids, ACE inhibitors or beta blockers, or assistive breathing technology while trying alternative treatments, because ALL of these “traditional” therapies have been shown through controlled and long-term studies to have benefit.

It may not always be obvious to non-specialists and parents how dangerous it can be to stop existing treatments, because the boys’ bodies may not show immediately an obvious bad reaction, even though there may be potentially dangerous effects internally. Treatments such as steroids, cardiac medicines (ACE inhibitors, beta blockers, AR Bs?) and/or assisted ventilation (bipap) should never be discontinued unless recommended by the neuromuscular physicians or subspecialists.

Steroids, when used chronically, suppress the body’s own steroid production and as a result, they should NEVER be stopped abruptly or without a carefully monitored course by a physician trained in their use. Again, we recommend consultation with the patient’s physician before any changes are attempted.

The same general cautions apply to cardiovascular medications such as ACE inhibitors, ARB's, and Beta-blockers since the risk of serious or fatal cardiac events is high in Duchenne patients and this risk can be heightened when these drugs are abruptly stopped.

As previously stated on our website, our clinic would never recommend any changes in medication, or assistive devices. Any changes should only be handled with your private practitioner in conjunction with the wishes of the patient and their parents.

As all of us are painfully aware, there is no ‘magic bullet’ at this point to help our sons, which is why many different medical and alternative therapies have been and are continually being explored. There are more potential therapies under investigation now than at any other time. It will be even more important to understand how pursuing any potential treatments or therapies might impact inclusion or exclusion from various clinical trials that are under way currently or expected in the near future. We need to keep an open mind about all possible therapies, and likewise, remember to ask the appropriate questions to ensure the best and safest opportunities for our sons.

Parents who cannot afford alternative therapies should be reassured that, to the best of our knowledge, the best therapies for boys with Duchenne are the therapies that are widely accepted by the medical community and available in most clinics.

On a personal note, I would like to state that I have been overwhelmingly impressed with the knowledge and dedication I have witnessed in the parents of these children with DMD. I welcome the opportunity to speak with you and answer any questions that you might have. We have created a Facebook page "South Texas Innovative Medicine" or you are welcome to contact me at the office 361-992-9432. I return all phone calls, but please be patient, it sometimes takes me a few days.

The issue of the cost of treatment has been mentioned. We are doing everything we can to keep the cost of the treatment affordable. I will state here what I have told each of the caregivers of my patients with DMD. If you are not satisfied with your sons’ improvement on VECTTOR, we will buy the machine back from you. No treatment is 100% effective and we would never want to profit from someone who wasn’t able to benefit from the treatment.

Finally, please remember, I've never claimed to be an expert on DMD, nor do I claim to have the “magic bullet”. However, I am the expert on my treatment, and I won't give up as long as I feel that I am helping these boys. I want nothing more than an opportunity to work in cooperation with those who are interested in further investigating the promising results we have seen thus far.

Respectfully,

Donald A. Rhodes, D.P.M., F.A.C.F.A.S

************************************

July, 2010

To DMD Parents:

I would like to take this opportunity to clear up a few misconceptions. I must state from the beginning that my staff and I have been overwhelmed with requests from parents of DMD children in the last few weeks. I am doing everything I can to return phone calls and answer questions, while still running my practice. If you have called the clinic and have not received a return phone call, please be patient with us. Also, please be patient with our web site as well. We are in the process of upgrading our server as our traffic has increased a great deal.

I realize that there is a strong community of DMD parents and from what I understand there has been much discussion about the treatments we have been utilizing in our clinic. I am working on putting together an online forum where I can host a question and answer session within the next couple of weeks. I had hoped to have this session sooner but I am traveling abroad and won't be back until next week.

In the meantime I feel that there are some issues that need to be addressed right away:

First, there seems to be some confusion regarding the Dynatron STS which was originally used in my clinic and VECTTOR which is the new method of treatment. I am the inventor of the STS and stand behind it as an excellent treatment for chronic pain. While it is possible to purchase the STS from a Dynatronics dealer (with a physicians prescription), this is not something I would recommend. Allow me to give you a brief history to explain.

As you all are aware, I am a podiatrist. I opened my practice in 1973 and for years performed complex foot surgery in addition to typical podiatric care. In 1992, I had a post surgical patient who developed a painful nerve condition called Reflex Sympathetic Dystrophy Syndrome (RSDS or also known as CRPS Chronic Regional Pain Syndrome).

This patient was my first experience with RSDS and I very quickly learned that there were little to no treatments available for this debilitating condition. Feeling a sense of obligation to my patient, I was determined to help her to the best of my ability.

I had always been interested in electrical stimulation and its healing effects on the body. I began to investigate this mode of treatment for my patient. Specifically, my research focused on the medical literature that demonstrated the ability of electro-stim to produce certain neuropeptides. It was well established in medical literature that healthy production of these neuropeptides was essential to proper functioning of all bodily functions. After extensive research studying the effects of these neuropeptides and many trials and errors utilizing electro-stim to augment their production, I was able to effectively use electro-stim to achieve remission for my patient.

Because RSDS is a disease with very little to offer in the way of treatment, word began to spread of my patient's remission. Slowly, my podiatric practice became increasingly devoted to patients with chronic pain, such as RSDS. Because I am a podiatrist, my scope of licensure limits me to treating pathologies of the foot. RSDS often, but not always, manifests in the foot. However, even patients who had only upper body symptoms still presented with pathologies in their feet. In my opinion, this is due to the systemic oxidative stress caused by the lack of the above mentioned neuropeptides. This oxidative stress can have devastating effects throughout the body.

During the early years of my work with electro-stim I was utilizing as many as 8 different types of machines to treat my patients. I knew that, in order to ever have my treatment widely available we had to simplify the process. In 1999 I worked in cooperation with Dynatronics Corporation to create and develop the Dynatron STS. The STS was launched in 2000. It was a vast improvement over all of the machines I had been utilizing in my clinic. However, although the STS was released to all physicians and physical therapists, it became apparent that without my direct guidance in changing beat frequencies and pad placements, results were only somewhat successful.

One relatively simple way of measuring autonomic dysfunction in the body is by measuring the temperatures of the extremities. Invariably, persons with autonomic dysfunction will have toes and fingers that are either too cold or too hot. They might also show a significant difference in the temperature of one extremity when compared to the opposite (left vs. right). Through trial and error I was able to utilize these clues to determine which particular pad placement protocol and specific beat frequency a patient needed based on his or her temperature response to the treatment.

Until very recently we would use the STS to run the treatments, but the STS was guided by information that was obtained from the patient's skin temperatures at various points during the testing or treatment cycle. Decisions as to which protocols to utilize were made based on the patient's individual response.

While this method proved to be very successful, the problem was obvious. This temperature testing and guidance could only be accomplished in our clinic. Once a patient was sent home it was impossible to gauge how well he or she was responding to the treatments. This often necessitated a return visit to the clinic for further testing in order to get back on the right track.

It was obvious that we had to figure out some way to automate the testing process so as to eliminate the need for the patients to be seen in the clinic so frequently. This is why the next generation of this technology, called VECTTOR, was created. Unlike the STS, which simply provides the electro-stim, the VECTTOR treatment unit is equipped with special temperature sensors that are constantly monitoring the fluctuations in the body. The internal computer program is designed to calculate this information in order to determine the proper beat frequency and to ensure that the patient is on the right treatment protocol. When a new protocol is needed, the patient needs only to contact the clinic and we send a new protocol (drawing which indicates proper pad placement) via fax or email.

Please understand that the VECTTOR is only in the prototype stage right now. At this time, we are not even able to keep up with the demand of our current patient population. We are working as hard as we can to decrease production time but it is a long process with many delays. As it stands right now we hope to submit VECTTOR to FDA for approval in early September. While we have the eventual hope that this unit can be prescribed by any physician, at this time, it can only be distributed in our clinic.

Just to reiterate; the DMD children who have been under our care who were using STS were doing so with a manual temperature sensing device. Their parents were then reporting the data to me to analyze and alter their treatment course as needed. Anyone attempting to treat with the STS alone will not have the ability to temperature test and, therefore, will have no way of knowing the correct beat frequency for their child. Neither will they have any way of knowing which pad placement protocol to use.

There also seems to be some misconceptions regarding our position on steroids. It's widely accepted that steroids have undesirable side effects. However, as with anything in medicine, one must always consider the risk vs. benefit. Just as we would never tell a chronic pain patient to abruptly stop taking narcotics, we would never tell a DMD parent to stop their child's steroids. Any reduction of medications (steroids or narcotics) would only be considered after noting a considerable improvement in the patient's condition and then only with the cooperation of your regular physician.

The same holds true for respiratory assistive or any other device upon which your child is dependant. We would never recommend discontinuing anything prescribed by your regular doctor, without first consulting with them. We are always happy to discuss our observations with your physician, but ultimately the decision is not in our hands.

On a final note, I would also like it to be known that I would welcome into our clinic any DMD representative, physician, physical therapist or any others who might be interested in learning more about the work that we are doing.

I hope this helps to clarify some of the concerns that have come to my attention. I look forward to speaking with many of you in the future and hope to address further questions at that point.

Respectfully,

Donald A. Rhodes, D.P.M., F.A.C.F.A.S.
http://www.paindefeat.com/wiki/PDFT/HomePage

רשק רוצ

העדוה ספדה הכנת ההודעה להדפסה

רבחל חלש

הבוגת

:תויורשפא

(10:09 24/11/10) וקנ'זור לאקס ברימ

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