Sunday, December 28, 2008
Wednesday, December 24, 2008
Sunday, December 21, 2008
I ASK MY BELOVED ONES NOT TO SUFFDER FOR ME, I WANT YOU TO UNDERSTAND I AM IN A BETTER PLACE, DIFFERT FOR SURE, A BEAUTIFUL GARDEN FULL OF FLOWERS, WITH WONDERFUL PEOPLE WAITING FOR ME, A PLACE WHERE PASSAPORT OR VISAS ARE NOT NECESSARY,FULL OF SUSHIS, SASHIMIS, TEMPURAS, KONIS AND SAQUES (AND WITH NO HANG OVER - PERFECT!).MAYBE THERE WILL BE THAT HOUSE, WITH WHITE CURTAINS AT THE WINDOW, A WHITE SHORT PICKED FENCE, OR MAYBE THAT BEACH WITH WHITE SAND, WITH BLUE AND ESMERALD GREEN WATER, WITH THE ETERNAL SUNSHINE, I DON'T KNOW, THE ONLY THING I KNOW IS THAT IT IS MY TURN TO TAKE CARE OF YOU AGAIN, TO WATCH AND PROTECT YOU FROM THE PLACE I AM, AND WHEN YOU FELL THAT COOL BREEZE OR THAT GOOD WARM...ITS ME HERE WITH YOU. BUT I WANT EVERY ONE TO KEEP SMILING, SO THEN I WILL BE HAPPY AND KEEP DOING HERE WHAT I ALWAYS WANTED TO DO... HELP YOU ALL.
I KNOW I AM STRONG ENOUGH FOR THIS, BECAUSE THEY GAVE IT TO ME WHEN I WAS THERE WITH YOU.
19 DECEMBER 2008. TIME OF PASS: 17:00 HS.
Friday, December 19, 2008
47 mins ago
FRIDAY, Dec. 19 (HealthDay News) -- The Genzyme Corp. drug Mozobil (plerixafor) has been approved by the U.S. Food and Drug Administration to boost a person's blood stem cell count before a bone marrow transplant, the agency said in a news release.
A bone marrow transplant is often performed in people with certain forms of cancer -- multiple myeloma and non-Hodgkin's lymphoma. Before getting high doses of chemotherapy or radiation, people with these forms of cancer may be advised to have blood stem cells collected so the cells can be re-infused after the therapy. Mozobil helps increase the number of stem cells before collection.
The most frequent side effects of Mozobil reported during clinical testing included diarrhea, nausea, fatigue, reactions near the injection site, headache, joint pain and dizziness.
The U.S. Food and Drug Administration has more about this drug's approval history.
Please review/double-click, the hyperlink above.
It contains some very interesting/informative information, to say the least.
The technology is coming. . . . .
ROCKVILLE, Md., Dec 18, 2008 /PRNewswire-FirstCall via COMTEX/ -- Neuralstem, Inc. (NYSE Alternext US: CUR) announced this morning that it has filed an Investigational New Drug (IND) application with the U.SO. Food and Drug Administration (FDA) to begin a clinical trial to treat amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease).
The Company is planning to treat ALS patients through spinal injections of its stem cells via its patented Human Neural Stem Cell technology."Like all first human trials, this proposed trial is primarily designed to test the safety and feasibility of both our stem cells and our method of delivering the cells to the spinal cord in ALS patients," said Neuralstem CEO and President, Richard Garr. "We are also proposing secondary endpoints which we hope will be able to measure a slowing down of the degenerative process."Neuralstem expects to conduct the trial at Emory University with Dr. Johnathan Glass, M.D., Director of the Emory Neuromuscular Laboratory and Director of the Emory ALS Center, as site Principal Investigator (PI). Dr. Eva Feldman, M.DO., Ph.D., Head of the A. Alfred Taubman Medical Research Institute and the De Jong Professor of Neurology at the University of Michigan Medical School, will be the overall PI for the ALS trial program. Formal approvals from these institutions to conduct the trial can come only after FDA approval of the trial protocol.."The filing of this IND is an important event for Neuralstem," said Garr, "but it marks only the beginning of a process which includes working together with the FDA to approve the first human ALS stem cell trial; refining our understanding of how to optimize delivery of our cells into patients; and ultimately delivering a new treatment for patients with this currently incurable disease."About NeuralstemNeuralstem's patented technology enables, for the first time, the ability to produce neural stem cells of the human brain and spinal cord in commercial quantities, and the ability to control the differentiation of these cells into mature, physiologically relevant human neurons and glia. Major Central Nervous System diseases targeted by the Company with research programs currently underway include: Ischemic Spastic Paraplegia, Traumatic Spinal Cord Injury, Huntington's disease and ALS. The Company filed an IND (Investigational New Drug) application with the FDA for ALS clinical trials in December, 2008, and has entered into a collaborative agreement with Albert-Ludwigs-University, in Freiburg, Germany, to develop clinical trials for Huntington's disease..In pre-clinical work, the company's cells have extended the life of rats with ALS (Lou Gehrig's disease) as reported the journal TRANSPLANTATION, in collaboration with Johns Hopkins University researchers, and also reversed paralysis in rats with Ischemic Spastic Paraplegia, as reported in NEUROSCIENCE on June 29, 2007, in collaboration with researchers at University of California San Diego.Cautionary Statement Regarding Forward Looking Information
Thursday, December 18, 2008
Wed Dec 17, 5:25 pm ET
SAN FRANCISCO – Billionaire philanthropists are donating $25 million to the University of California, San Francisco toward building a new stem cell research center.
University officials announced the donation Wednesday from the Eli and Edythe Broad Foundation.
The money will help fund a $123 million laboratory building to bring all the university's stem cell research under one roof.
The Broads, who live in Los Angeles, made their wealth in housing and financial services. They have donated hundreds of millions of dollars to disease research.
Last year, the couple pledged $20 million to the stem cell institute at the University of California, Los Angeles. In 2006, they gave $25 million to establish a stem cell center at the University of Southern California.
MDA/ALS Center Director Recommends Better Planning
Using a noninvasive ventilation device in ALS may actually weaken respiratory muscles faster than not using one, a preliminary study in the March issue of Muscle & Nerve suggests. On the other hand, the use of such devices was found by the researchers to relieve fatigue, to promote a sense of mastery over the illness, and even to prolong survival. Noninvasive ventilation is a term for air delivered through natural openings in the body, such as the nose or mouth, while invasive ventilation means air delivered through a surgical hole in the neck directly into the trachea (windpipe) through a tracheostomy tube. As ALS weakens respiratory muscles, those affected often need a form of assisted ventilation.
A BiPAP machine is a form of noninvasive ventilation that can be used part-time.
A common noninvasive ventilation device is the BiPAP, which stands for "bi-level positive airway pressure." One air pressure is delivered for inhalation, another for expiration. (The name is actually a registered trademark of the Respironics Company of Pittsburgh but is widely used in a generic sense.) BiPAP machines are generally portable and convenient compared with the type of machine usually used to deliver air with a tracheostomy tube. (For more on ventilation, see "What Everyone With ALS Should Know About Breathing," ALS Newsletter, vol. 5, no. 4, 2000; and two articles on breathing and ventilation in Quest, vol. 5, nos. 5 and 6, 1998.)
COULD BiPAP BE HARMFUL?
At least two recent journal publications - a letter in the Feb. 15 New England Journal of Medicine as well as the Muscle & Nerve article - have raised questions about whether noninvasive ventilation is helpful for people with ALS, whether it's dangerous to rely on it instead of moving to tracheostomy ventilation, and what effect noninvasive ventilation has on respiratory function.
The New England Journal letter recounts the story of a man with ALS who died after a mechanical failure turned off his BiPAP system. The Muscle & Nerve article reports a small study that found that measures of pulmonary (lung) function deteriorated in people with ALS who were using noninvasive ventilation while also finding that people felt better and lived longer when they were using it.
People who were able to sleep while using a noninvasive ventilation device lived an average of 20 months from the time of the study's beginning, while those who couldn't sleep with it on lived an average of five months. The study found that measures of pulmonary muscle function and blood oxygen and carbon dioxide levels, taken when the person was off the assistive device, declined over time despite the use of noninvasive ventilation for varying periods during the night and day.
IMPROVED QUALITY OF LIFE
Hiroshi Mitsumoto, a neurologist and neuromuscular disease specialist who directs the Eleanor and Lou Gehrig MDA/ALS Center at Columbia Presbyterian Medical Center in New York, was part of the Muscle &Nerve study. "It sounds contradictory," Mitsumoto said of the study. Noninvasive ventilation, he says, "may make pulmonary function worse, but the patient lives longer and his quality of life is better. So the question is, 'Is it good to have BiPAP or other noninvasive ventilation, or bad to have it?'"
Mitsumoto says the situation can be compared to that of someone with weak leg muscles who wears a brace. The leg muscles may weaken faster through disuse than they would if the person were trying to use them, but his function - walking - actually improves, because the leg is supported by the brace. "I think the [noninvasive] ventilator is doing the same thing," he says. "It's providing better ventilation but deconditioning the muscles at the same time." Mitsumoto noted that more studies need to be done to verify the results of these limited observations.
BiPAP WON'T WORK INDEFINITELY
The important thing for people with ALS and their caregivers to keep in mind, Mitsumoto said, is that BiPAP or other noninvasive ventilation won't be effective indefinitely in ALS. There will come a time when respiratory failure, even with a noninvasive vent system, forces the patient or caregiver to make an emergency choice - whether or not a tracheostomy should be performed and invasive ventilation started. This kind of ventilation can achieve more control and offer better assistance to failing respiratory muscles.
When to begin thinking about moving to invasive ventilation can be a "tough question," Mitsumoto says. "Increasing dependency on BiPAP during the night and even during the day is clearly one sign," he notes. "Increasing discomfort, despite using the BiPAP, is clearly another. Patients must be followed closely by a pulmonary doctor who has extensive experience with ALS. There's no simple, cookbook answer here."
Other experts have noted that morning headaches, increasing sleepiness, a sense of mental fuzziness, and increasing anxiety, particularly when lying down, are also signs that a person may not be getting adequate ventilatory support. Shortness of breath may or may not be perceived.
After a tracheostomy, air is delivered directly to the windpipe.
It's best, Mitsumoto said, to have one's thoughts in writing in an advance directive before an emergency, such as a respiratory crisis or equipment failure, arises.
It's also important, he noted, to keep emergency resuscitation bags (often called Ambu bags) close by and to make sure someone can give temporary ventilation, if that's needed, while waiting for professional assistance.
Many people with ALS, Mitsumoto said, avoid discussing end-of-life issues with their doctors and families.
"How they want to deal with their life is not fully discussed," he said, "but they keep using [noninvasive] BiPAP. Meanwhile, their condition deteriorates, and serious problems can occur."
BiPAP and other noninvasive ventilation systems, he said, are "not an answer" for long-term survival.
"If they want to live [for a long time], they have to tell the doctor, and a prophylactic trach should be done," Mitsumoto said. "There is a lot of misunderstanding and miscommunication. BiPAP is not a machine to be used for long-term survival. Its primary effect is symptomatic relief. More and more people have started depending on BiPAP to live longer, but that's really the wrong usage. The BiPAP machine was not developed for that purpose.
"When they use BiPAP, they should have a good idea of what they want to do. Advance directives should be discussed early. When a BiPAP is utilized without discussing those issues, it's problematic." While I understand the obvious limitations of NIV, it would be useful to have current and clear clinical advice to lean on given the seriousness of respiratory muscle weakness in ALS/MND and its role in sustaining life and giving impoved quality of life.
Wednesday, December 17, 2008
Tuesday, December 16, 2008
by Martine Nouaille Martine Nouaille
VATICAN CITY (AFP) – The Vatican on Friday reopened ethical questions surrounding stem cell research and techniques such as cloning with a document affirming the "dignity of the human embryo."
"Dignitas Personae" (Dignity of the Person), the first "instruction" on reproductive technology in more than 20 years, comes as countries including the United States and France prepare to review policies in the controversial field.
The sweeping instruction lists biomedical techniques considered "illicit" by the Roman Catholic Church such as in vitro fertilisation, cloning, the therapeutic use of stem cells, producing vaccines from embryo cells and the use of the "morning-after" contraceptive pill.
Such practices go against the "fundamental principle" that the dignity of the person must be recognised from conception until natural death, it says.
Issued by the Congregation for the Doctrine of the Faith, the Vatican's doctrinal watchdog, the 33-page instruction updates a 1987 document, "Donum Vitae" (The Gift of Life), which asserted the integrity of the human embryo.
The new instruction virtually enshrines the embryo not only as a human being but also as a whole "person" with all the philosophical and legal consequences that such recognition might entail, according to Bishop Rino Fisichella, secretary of the Congregation for the Doctrine of the Faith.
"The recognition is implicit, but we don't get involved in the philosophical debate," Fisichella said as he presented the document.
The document, approved by Pope Benedict XVI, also reprises the Church's condemnation of in vitro fertilisation, while decrying methods that prevent implantation of the embryo or cause its elimination as "falling within the sin of abortion".
"The blithe acceptance of the enormous number of abortions involved in the process of in vitro fertilisation vividly illustrates how the replacement of the conjugal act by a technical procedure ... leads to a weakening of the respect owed to every human being," the document says.
The text also warns against a "eugenic mentality" arising from advances in genetic engineering, saying: "In the attempt to create a new type of human being, one can recognise an ideological element in which man tries to take the place of his Creator."
Catholics are called to abide by such "instructions," which have had practical consequences across the centuries.
The 1987 instruction, focussing on in vitro fertilisation, was signed by the pope, then Cardinal Joseph Ratzinger, during his 24-year tenure at the head of the Vatican's highest rule-making authority.
It had important consequences for Catholic hospitals around the world as they scrapped programmes to help infertile couples, and it affected funding for certain medical research.
While the techniques condemned by the Church are legal in many countries and widely practised, the new document says Catholic researchers have the duty to distance themselves from a "gravely unjust legal situation and to affirm with clarity the value of human life".
US president-elect Barack Obama, who is to take office on January 20, is expected to act quickly to reverse an executive order by President George W. Bush banning embryonic stem cell research.
Also, French bioethics law is set for review next year.
The Holy See is aware that it is challenging cutting-edge technology, led notably by British embryo researchers, and expects "a variety of reactions," Fisichella said.
"Some will prefer to ignore (the instruction), others will take the easier route of deriding it, and still others will file these pages away as a manifestation of obscurantism blocking progress and free research, but many others will share our concern and our analysis," he said.
Saturday, December 13, 2008
By Philip Pullella Philip Pullella Fri Dec 12, 10:39 am ET
VATICAN CITY (Reuters) – A Vatican bioethics document Friday condemned artificial fertilization and other techniques used by many couples and also said human cloning, "designer babies" and embryonic stem-cell research were immoral.
The long awaited document from the Vatican's doctrinal body marked a big step by the Vatican into the brave new world of biotechnology, an area in which governments around the world are struggling to formulate legislation.
The document also condemned new drugs that block pregnancy from taking hold, such as the so-called "morning-after pill" and the drug RU-486, which blocks the action of hormones needed to keep a fertilized egg implanted in the uterus.
These drugs, as well as the IUD (intrauterine device), which has been in use for decades, were deemed to fall "within the sin of abortion" and are gravely immoral.
"Dignitas Personae (dignity of a person), an Instruction of Certain Bioethical Questions," is an attempt to bring the Church up to date with recent advances in science and medicine.
The document, the most authoritative of its kind from the Vatican in 20 years, said human life deserved respect "from the very first stages of its existence (and) can never be reduced merely to a group of cells."
"The human embryo has, therefore, from the very beginning, the dignity proper to a person," said the Congregation of the Doctrine of the Faith's document, approved by Pope Benedict who headed the same office before his election in 2005.
It said most forms of artificial fertilization "are to be excluded" because "they substitute for the conjugal act ... which alone is truly worthy of responsible procreation."
Condemning in-vitro fertilization, it said the techniques "proceed as if the human embryo were simply a mass of cells to be used, selected and discarded."
The highly technical document said only adult stem cell research was moral because embryonic stem cell research involved the destruction of embryos. It also condemned freezing embryos.
Governments in countries including the United States are grappling with legislation on embryonic stem cell research.
The outgoing administration of President George W. Bush has placed restrictions on federal funds for embryonic stem cell research but President-elect Barack Obama has promised to lift them.
The 35-page document also attacked the concept of "designer babies," either by pre-implantation diagnosis during in vitro fertilization where embryos are selected before being transferred to a woman's womb, or in attempts at human cloning in the future.
It branded as "shameful and utterly reprehensible" diagnosis aimed at ensuring that only embryos free from defects or having the desired sex or other particular qualities are transferred into a woman's womb.
It condemned the concept of human cloning "to satisfy certain specific desires, for example, control over human evolution, selection of human beings with superior qualities, pre-selection of the sex of a child to be born, production of a child who is the "copy" of another, or production of a child for a couple whose infertility cannot be treated in another way."
Saying life was sacred from the moment of conception to the moment of natural death, the document also defended the Roman Catholic Church's right to intervene on such matters.
"There are those who say that the moral teaching of the Church contains too many prohibitions. In reality, however, her teaching is based on the recognition and promotion of all the gifts which the Creator has bestowed on man: such as life, knowledge, freedom and love," it said.
(Editing by Janet Lawrence)41
Friday, December 12, 2008
By Richard Robinson, Science Writer
Subcutaneous (under the skin) delivery of insulin-like growth factor 1 (IGF-1), known as the drug Myotrophin, does not benefit people with ALS at a dose of 0.5 milligrams per kilogram of body weight, according to a large clinical trial whose results were announced.
IGF-1 is a substance the body produces to sustain motor neurons, the nerve cells that die in ALS. Experiments in animal models of the disease suggested IGF-1 treatment may delay death of motor neurons. IGF-1 was tested in ALS a decade ago in two trials, but the results of the two were inconsistent, with one suggesting treatment was beneficial, and the other showing no benefit.
“These results are deeply disappointing to all of us in the ALS community,” said Lucie Bruijn, Ph.D., senior vice president, research and development, The ALS Association. “The subcutaneous delivery route may be the key problem, or it may be that IGF-1 alone is not sufficient to rescue motor neurons.”
The current trial involved 330 people with ALS from 20 ALS treatment centers across the United States. Patients were randomly assigned to receive either IGF-1 or a placebo, injected under the skin twice a day, for two years. The dose used was the highest tolerated dose from previous studies. Neither doctors nor patients knew which treatment the patient had received until the end of the study.
At the end of the two-year treatment period, there were no differences between people with ALS who received IGF-1 and those who received placebo in muscle strength, the need for a tracheostomy for breathing, or survival, indicating that IGF-1 provided patients no benefit.
The ALS Association is the only national, not-for-profit voluntary health organization devoted solely to fighting ALS through research, patient services, advocacy and public education and information. The Association is currently exploring multiple new avenues for treatment through its TREAT-ALS (Translational Research Advancing Therapies for ALS) drug discovery program and clinical trials process.
For more information about The ALS Association’s research program, visit www.alsa.org/research.
Thursday, December 11, 2008
One evening an old Cherokee told his grandson about a battle that goes on inside people. He said, "My son, the battle is between two 'wolves' inside us all.One is Evil. It is anger, envy, jealousy, sorrow, regret, greed, arrogance, self-pity, guilt, resentment, inferiority, lies, false pride, superiority, and ego.The other is Good. It is joy, peace, love, hope, serenity, humility, kindness, benevolence, empathy, generosity, truth, compassion and faith."The grandson thought about it for a minute and then asked his grandfather, "Which wolf will win?"The old Cherokee simply replied, "The one you feed."
Tuesday, December 9, 2008
HIS POWERFUL VIDEO
Craig Ewert, 59, a retired university professor, opted for assisted suicide rather than spend the rest of his life locked in a "living tomb."
Monday, December 8, 2008
Harvard Stem Cell Institute team replicates Lou Gehrig's disease process in lab dish
December 5, 2008
B. D. ColenHarvard News Office
A Harvard Stem Cell Institute research team has succeeded in deriving spinal motor neurons from human embryonic stem cells, and has then used them to replicate the Amyotrophic Lateral Sclerosis (ALS) disease process in a laboratory dish.
The researchers, lead by HSCI Principal Faculty member Kevin Eggan, found that human motor neurons exposed to glial cells carrying a known genetic mutation associated with ALS died, while other types of neurons exposed to the disease-carrying glial cells were unaffected.
"The logical next step is to ask what the glial cells are doing to kill the motor neurons," said Eggan, an assistant professor in Harvard's new inter-school Department of Stem Cell and Regenerative Biology.
Eggan said that the new findings are particularly important for ALS research because they answer the field's long-standing "murder or suicide" question. That is, do motor neurons in patients with ALS - an always-fatal, neurodegeneratative condition known as Lou Gehrig's Disease - die because of something inherent to the motor neurons, do they "commit suicide?" Or, are they "murdered" - is their something external killing them? This latest research strong suggests that the motor neurons are being "murdered" by something in the glial cells carrying a mutation of the SOD1 gene.
Additionally, Eggan said, this latest study is "important for stem cell science because one of the things we've been promising is that these stem cells would be important for drug discovery. We've produced industrial quantities of these motor neurons; we've shown that this does work, that you can overcome the technical limitations. This is a disease process in a petri dish," and that's what we've been promising.
Finally, he said, this experiment once again proves the utility of human embryonic stem cells, and confirms the value of continuing to use them to study both normal development and disease process, particularly as there have yet to be studies demonstrating that alternative types of cells are identical in all respects to human embryonic stem cells.
In addition to replicating the disease process, Eggan, post doctoral fellow Paolo DiGiorgio, and colleagues Gabriella L. Boulting and Samuel Bobrowicz, demonstrated that an inflammatory pathway plays a role in the disease process, and they found a small molecule that has at least some protective effect. Eggan noted, however, than when that same compound has been test on ALS patients it has slowed the disease process. "We don't know," he said, "whether there may be other issues there, whether there are problems involving the blood-brain barrier, or dosing levels."
Less than six months ago, using induced pluripotent stem cell (iPS) technology, a team lead by Eggan, who in addition to his Harvard titles is a Stowers Medical Institute Investigator, produced patient-specific stem cell lines from the skin cells of ALS patients.
In the spring of 2007. Eggan and colleagues created an ALS model using mouse stem cells. But research findings in animal models always beg the question of whether the results will translate directly to humans. In this case, the answer to that question is a resounding Yes!
The publication of this latest paper in the journal Cell Stem Cell is only the second report in the literature of human stem cell work resulting in the replication of a disease process in a laboratory dise. That work, involving Fragile X syndrome, did not involve the derivation of a specific cell type.
The Eggan lab's work was funded by Project ALS, with additional support from the Stowers Medical Institute and the New York Stem Cell Foundation.
Saturday, December 6, 2008
Hi Team Iplex,
I have attempted to explain the process by which Iplex will be distributed in the USA. I am still unclear about out of country delivery of Iplex, and will follow up with Christine O’Neal first thing Monday to try to establish what the protocol for members of Team Iplex outside of the USA will be.I have set first to explain some of the language, which may be unfamiliar to some of us, with links and definitions. For those of you in the pharmaceutical industry, please jump in and fill in any missing links, and please feel free to try to simplify my definitions if you see a way to explain this more clearly. In brief, as many of us are aware, Iplex will be available in the USA by a process called a single patient IND, also commonly called Compassionate or Emergency use. This is a process which sounds very complex, but in reality is nothing more than a lot of paperwork, don’t be alarmed or frightened by it, it’s just paperwork…One of the first questions I have had is cost. When a drug is accessed by a single patient IND the FDA allows the drug manufacturer to recoup reasonable costs only. When I spoke to Christine Friday, she indicated that cost had not yet been set. I have no idea at this time what the monthly costs will be. Many also want to know about insurance reimbursement. At this point, I feel that insurance reimbursement is highly unlikely. I do not know of any insurance company that will pay for any of the costs associated with a single patient IND – that includes associated MD visits, labs, etc. I wish I had better news to report, but this is the unfortunate facts of the matter. A Single patient IND (again, AKA compassionate/emergency use) requires the approval of an IRB. Most of us aren’t familiar with what an IRB is. Here is a brief definition, as supplied by a Wikipedia link and my definition.Institutional Review Boards (IRB's): http://en.wikipedia.org/wiki/Institutional_review_boardEvery hospital/teaching facility that does research involving human subjects must have an IRB that initially reviews/approves proposed research. An IRB protects the ethical interests of humans involved in trial studies. Before it will approve a single patient IND, the IRB will determine that a our risks are reasonable in relation to anticipated benefits – as Iplex is already FDA approved, and has passed rigorous safety testing in other diseases, this shouldn’t be a very complex process. The FDA has already approved this process for Insmed, so their role should be simply shuffling of papers, I don't expect lengthy reviews on their end.Now, many of you may have physicians who are not affiliated with a teaching hospital, or do have a physician who conducts research and has association with an IRB, but who won’t write for Iplex, and are curious about what you can do to access an IRB. Fortunately for those of you, there are independent IRBs. One of the larger independent Institutional Review Boards that may be accessed by physicians not within the realm of academia is Western, their website is here http://www.wirb.com/. There are fees associated with this service, I cannot comment on the pricing, but would suggest you contact Western, or do a web search to locate any independent IRB. Your private physician may also be familiar with an independent IRB, and already have a established relationship with one?So, in summary, here is the process for a Single Patient IND, there are several forms which will be filled out, submitted to the FDA by your doctor, once all of the t’s are crossed and the I’s dotted, the drug will be shipped to your doctor. In my experience with government bureaucracy – it is important the forms be filled out completely and accurately. This is what you should expect your doctor to submit to the FDA.Request for a single patient IND for Compassionate or Emergency Use should be stated at the top of the correspondence. Brief Clinical History of the patient including the diagnosis, the disease status, prior therapy, response to prior therapy and the rationale for requesting the proposed treatment. Proposed Treatment Plan including the dose, route, planned duration, monitoring procedures and modifications (e.g. dose reduction or treatment delay) for toxicity. Reference a published protocol or journal article if appropriate. Drug Supply Reference Statement which would name the supplier or manufacturer and a statement that a Letter of Authorization to cross reference an appropriate IND of the supplier or Drug Master File (DMF) of the manufacturer is included. The treating physician must contact the supplier or manufacturer for such a statement. Informed Consent Statement that states that informed consent and approval of an appropriate Institutional Review Board (IRB) will be obtained prior to initiating treatment. There are some IRBs that have specific procedures for approving emergency requests. Investigator Qualification Statement that specifies the training, experience, and licensure of the treating physician. The first two pages of a Curriculum Vitae (a fancy name for resume) typically contain this information and are usually sufficient. FDA Form 1571 completed with the treating physician listed as the sponsor. Form 1571 and other forms can be downloaded from the Internet. Contact telephone number and facsimile number. If the request is approved, an IND number will be issued by the FDA and the treating physician will be contacted by phone or fax with a letter to follow. The IND is considered active upon issuance of the number. The IND sponsor (treating physician) will then contact the drug supplier and provide the IND number. The supplier may then ship the drug directly to the treating physician.Please feel free to email or call me if you want me to try to explain better. I have never been accused of being a good teacher, I apologize if this isn't as clear as it could/should be - but will be happy to answer any questions to the best of my ability.Andrea--"Being deeply loved by someone gives you strength, while loving someone deeply gives you courage." Lao Tzu Taoist Philosopher,600 BC-531 BC
Friday, December 5, 2008
Here is the briefest recap of my conversation with Dr Allan late yesterday:
1. Insmed is proceeding immediately with a Personal Physician IND (Investigational New Drug), or Compassionate Usage, program for Iplex availability by ALS patients world-wide.
2. This program is being offered after the consideration, and rejection for reasons of timeliness or other hindrances, of either "an approved drug being returned to the workplace" or a "Treatment IND". It is felt by Insmed that the prescribed method, a "Personal Physician IND", will accommodate the needs of the ALS community faster and easier for them.
3. Details of the program are being emailed directly to all patients and/or caregivers immediately. This includes both those who have contacted Insmed directly, usually to the attention of Christy O'Neal, and those whose contact information has previously been submitted by ALS WORLDWIDE or others.
4. Further information will be available on the www.Insmed.com website as of Monday, December 8, 2008.
5. The details of a physician letter that must be provided to the US FDA (Food and Drug Administration) will be linked on the Insmed website and in the individual letters being sent to all patients and caregivers whose names have been provided directly or through other channels. I am not including what I believe to be the accurate section(s) of the FDA.com website because I do not want to misstate or inaccurately misdirect anyone to inappropriate sections of a very large and complex website.
6. ALS WORLDWIDE will continue to provide either contact or organizational support to anyone in the ALS community who needs or requests that support. This can, and will, include suggested dosage information and the assistance or support of a physician who can oversee the use of Iplex in case the patient is unable to procure such assistance from their local family physician or neurologist. Please understand this assistance is in no way as an agent or representative of Insmed-it is strictly as volunteer support to the ALS community and is provided as a courtesy to an underserved patient community. There is no charge or compensation in any form.
7. A single-page letter from the attending physician will be required by FDA and its details will also be either directly stated in the Insmed website or linked to the FDA website as of Monday, December 8, 2008.
8. The results of the Italian Observational Study are not yet available. The observed patients' information is now being compared to historical placebo history for comparative understanding. This is a common technique when double-blind study comparisons are unavailable.
9. Patients from countries other than the US are advised to provide their individual country equivalent of US FDA with the same information as required by US FDA. Alternatively, their US physician representative can submit their request to US FDA if a US address is provided.
10. The final price charged for Iplex, and the method by which such charge is collected, have yet to be determined. My understanding is that "collection of money for the initial shipment(s) of Iplex will not stand in the way of its distribution to the ALS patients." I don't know how long this view will remain in effect, and because the likelihood of private, state or VA insurance coverage is always in question or even improbable, ALS Worldwide/Team IPLEX will be continuing our efforts to secure some form of corporate or foundational assistance for those not covered by insurance. We have not yet received any commitment of such participation and, as stated, we do not yet know what the eventual charge for Iplex will be or when it will be implemented.
Other points were discussed-they are either not relevant or less specific than the above information. We are traveling today through Wednesday, December 10 but are always reachable by email or cell phone. Please feel free to extract any or all parts of the above synopsis in your own individual communications to others within the ALS community.
Best Wishes,Stephen Byer
ALS WORLDWIDE is a non-profit organization that provides support to ALS families internationally
through scientific research interpretation, individual patient advocacy and community activism.
Thursday, December 4, 2008
By MALCOLM RITTER, AP Science Writer Malcolm Ritter, Ap Science Writer Wed Dec 3, 3:10 pm ET
NEW YORK – Consumers should be wary of Web sites from clinics that offer stem cell treatments, says a study that found a lack of firm medical evidence to back up their claims. The Web sites in the study generally portrayed their therapies as safe, effective and ready for routine use, but published research doesn't support that "overoptimistic" picture, the study authors said.
The analysis is presented in the December issue of the journal Cell Stem Cell by scientists at the University of Alberta in Canada. They cautioned that their overall findings can't be applied to the claims of any individual clinic.
The study is "a very important wake-up call," said Dr. George Daley, past president of the International Society for Stem Cell Research, who had no role in the new report.
"I think these Web sites are dangerous," said Daley, a Boston stem cell researcher. "They overpromise effectiveness and safety of the therapy and they completely underestimate and underinform about risks. ... (Such) overhyped marketing directly to the patient is putting patients at risk of financial exploitation at the very least, and physical danger at the worst."
In recent years, desperate patients with few options have traveled to China and other countries where doctors offer stem cell or other cell treatments for such things as spinal cord injuries, Parkinson's disease and blindness.
The new study did not assess the Web site claims directly by checking on how well patients actually fared at the clinics in the study.
Instead, researchers began with the 19 Web sites they found through Google in 2007. Treatments were promoted in several countries, including China, Mexico and Russia. None promoted treatments within the United States; one didn't give a location for treatments.
Last July, the researchers looked for published studies in human patients about using stem cells to treat the medical conditions mentioned most often by the Web sites: multiple sclerosis, Parkinson's and Alzheimer's diseases, spinal cord injury, stroke and heart attack.
They reported finding some encouraging hints but no clear evidence of benefit.
In the same issue of the journal, a report from the international stem cell society describes new research guidelines that condemn the marketing of unproven therapies. The society has posted a patient handbook on its Web site to help people who are considering stem cell therapy.
The guidelines say that in limited cases, doctors may be justified in trying an experimental treatment outside of a formal study for small numbers of seriously ill patients. The guidelines recommend standards for that situation, such as approval from a group of experts with no vested interest in the treatment and a commitment by those offering it to proceed to a formal study.
On the Net:
Cell Stem Cell: http://www.cell.com/cell-stem-cell/home
Stem Cell society: http://www.isscr.org
Patient handbook: http://www.isscr.org/clinical_trans/pdfs/ISSCRPatientHandbook.pdf
Wednesday, December 3, 2008
Treatment of amyotrophic lateral sclerosis patients by autologous bone marrow-derived hematopoietic stem cell transplantation: a 1-year follow-up.
Deda H, Inci M, Kurekci A, Sav A, Kayıhan K, Ozgun E, Ustunsoy G, Kocabay S.
Department of Neurosurgery and Neurology, Akay Hospital, Ankara, Turkey.Background Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder characterized by progressive loss of spinal cord and cortical motoneurons. Despite improved understanding of the mechanisms underlying ALS, in clinical practice the management of ALS remains essentially supportive and focused on symptom relief. However, over the past few years stem cell research has expanded greatly as a tool for developing potential new therapies for treating incurable neurodegenerative diseases. Methods Thirteen patients with sporadic amyotrophic lateral sclerosis (SALS) were included in this study, and bone marrow (BM)-derived hematopoietic progenitor stem cells were used. We selected patients with bulbar involvement and severe loss of movement. Our aim was to put the stem cells into the end of the brain stem and at the beginning of the spinal cord because the blood-brain barrier is intact in ALS and this region was the most affected part in our patients. Under general anesthesia, a total laminectomy was performed at the C1-C2 level. Stem cells were injected to the anterior part of the spinal cord. Results During the follow-up of 1 year after stem cell implantation, nine patients became much better compared with their pre-operative status, confirmed by electro neuro myography (ENMG). One patient was stable without any decline or improvement in his status. Three patients died 1.5, 2 and 9 months, respectively, after stem cell therapy as a result of lung infection and myocardial infarction (MI). Discussion These results show that stem cell therapy is a safe, effective and promising treatment for ALS patients.
Tuesday, December 2, 2008
Leuven (Belgium), Stockholm (Sweden) - Permission has been granted to start the first safety and tolerability trial on patients for a remedy for ALS. ALS is an incurable, paralyzing neurodegenerative disorder that strikes 5 persons in every 100,000. The disease commonly affects healthy people in the most active period of their lives - without warning. Researchers from VIB at the K.U.Leuven have previously shown the possibilities for the use of VEGF in the treatment of ALS through work in animal models. The Swedish Biopharmaceutical company NeuroNova has already built upon this research. Together with UZ Leuven they'll start the first evaluation of safety and tolerability of the drug in patients by the end of this year. This is an important step in the development of a new treatment. It will take several years before the protein can be made available as a medicine.
An incurable disease of the muscles
Amyotrophic Lateral Sclerosis (ALS) can strike anyone. The Chinese leader Mao Tse Tung, Russian composer Dimitri Shostakovich, the legendary New York Yankee baseball player Lou Gehrig, and astro-physicist Stephen Hawking have all been afflicted with ALS. About half of the patients dies within three years - some even in the first year - usually as a consequence of suffocation.
In ALS, the patient's nerve bundles that extend to the muscles deteriorate. As a result the patient loses control of the muscles, and progressively becomes paralyzed. The originating mechanism of this deadly disease of deterioration - which has an enormous medical and social impact - remains obscure. At present, the disease is totally untreatable.
VEGF: a promising candidate drug
VEGF is a substance that controls the growth of blood vessels. Unexpectedly, VEGF also helps neurons survive under stressful conditions. In 2001 Peter Carmeliet's team showed that too little VEGF causes ALS-like symptoms in mice. Later the group of Diether Lambrechts, Wim Robberecht and Peter Carmeliet showed that persons who produce too little VEGF - due to certain variations in the gene that codes for VEGF - have a higher risk of developing ALS. This was the starting point of a search for a possible treatment with the VEGF protein.
Testing the treatment on rats with a severe form of ALS and on rats with a milder form, the researchers found that, in both groups, the VEGF-treated rats manifested the disease later than the untreated animals, and they lived considerably longer.
Using a pump
The researchers also investigated what the optimal technique would be for administering VEGF. An ordinary injection proved to be ineffective. But continuous administration of the VEGF protein directly into the cerebrospinal fluid (the fluid that circulates around the brain and the spinal cord) was quite effective. This was possible by means of a small pump that continuously pumps the VEGF protein in the brain. Furthermore, this technique permits a patient-oriented approach by enabling the administered dose of the VEGF protein to be easily controlled.
A story with several players
These encouraging and promising results were only the first steps on the way to a new remedy. Anders Haegerstrand and his team of the Swedish company NeuroNova have taken the development of the treatment further. After additional studies this research has reached the stage of starting the first trial in patients. Wim Robberecht (UZ Leuven) and Markus Jerling (NeuroNova) will co-ordinate this first trial which is intended to evaluate the safety and tolerability of the drug and the infusion system. It is planned to start at the end of this year, and the investigator Dr Robberecht is currently looking for patients who are eligible for participation. These regulated studies on ALS patients will have to demonstrate the safety of the VEGF administration, and in a later stage the efficacy of VEGF as ALS therapy, before the protein can be made available as a medicine. Such procedures can easily last several years.
Given that this research can raise a lot of questions for patients, we ask you to please refer questions in your report or article to the email address that VIB makes available for this purpose: email@example.com . Everyone can submit questions concerning this and other medically-oriented research directly to VIB via this address.
For practical information concerning the clinical trials you can contact Petra Tilkin (firstname.lastname@example.org).
For more information on this project, please contact
the VIB Communication Service: +32 9244 66 11
Peter Carmeliet: +32 16 34 61 42 of +32 475 87 13 79
Wim Robberecht: +32 16 33 07 70 (0486 09 85 69)
Anders Haegerstrand, CSO, NeuroNova: +46 8786 0900, email@example.com
Markus Jerling, NeuroNova: + 46 8786 0900, firstname.lastname@example.org
For more information on NeuroNova, please contact
Ulf Ljungberg, NeuroNova: +46 8786 0900, email@example.com
More info on
Relevant scientific publications
Lambrechts et al., Meta-analysis of VEGF variations in ALS: increased susceptibility in male carriers of the -2578AA genotype (J. Med. Genet., epub, July 2008)
Zacchigna et al., Neurovascular signalling defects in neurodegeneration (Nature reviews Neuroscience, Vol 9, March 2008, 169-181)
Lambrechts and Carmeliet, VEGF at the neurovascular interface: Therapeutic implications for motor neuron disease (Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease, Vol 1762, Issues 11-12, November-December 2006, 1109-1121)
Storkebaum et al., Treatment of motoneuron degeneration by intracerebroventricular delivery of VEGF in a rat model of ALS (Nature Neuroscience, Vol 8(1), January 2005, 85-92)
Azzouz et al., Deletion of the hypoxia-response element in the vascular endothelial growth factor promoter causes motor neuron degeneration (Nature, vol 429,27 May 2004, 413-417)
Lambrechts et al., VEGF is a modifiere of amyotrophic lateral sclerosis in mice and humans and protects motoneurons against ischemic death (Nature Genetics, Vol 34(4), August 2003, 383-94)
Skene & Cleveland, Hypoxia and Lou Gehrig (Nature Genetics, vol 28, June 2001, 107-108)
Oosthuyse et al., Deletion of the hypoxia-response element in the vascular endothelial growth factor promoter causes motor neuron degeneration (Nature Genetics, Vol 28, June 2001, 131-138)
Note for the editor
The fundamental preclinical VEGF research was done by Diether Lambrechts, Wim Robberecht and Peter Carmeliet of the VIB Vesalius Research Center, K.U.Leuven, under direction of Peter Carmeliet (www.vib.be/Research/EN/Research+Departments/Vesalius+Research+Center, www.vib.be/Research/EN/Research+Departments/Vesalius+Research+Center/Peter+Carmeliet )
The further development of the research is done by NeuroNova (www.neuronova.com). The clinical trial is done in collaboration with Wim Robberecht, linked to UZ Leuven (www.neurology.kuleuven.be) and the research group ‘Neurobiology' of the VIB Vesalius Research Center, K.U.Leuven (More info: www.vib.be/Research/EN/Research+Departments/Vesalius+Research+Center/Wim+Robberecht).
VIB, the Flanders Institute for Biotechnology, is a non-profit research institute in the life sciences. Some 1100 scientists and technicians conduct strategic basic research on the molecular mechanisms that control the functioning of the human body, plants, and micro-organisms. Through a close partnership with four Flemish universities - Ghent University, the Katholieke Universiteit Leuven, the University of Antwerp, and the Vrije Universiteit Brussel - and a solid investment program, VIB unites the forces of 65 research groups in a single institute. Their research aims at fundamentally extending the frontiers of our knowledge. Through its technology transfer activities, VIB strives to convert the research results into products for the benefit of consumers and patients. VIB also develops and distributes a broad range of scientifically substantiated information about all aspects of biotechnology. More info at: www.vib.be.
The University of Leuven is Belgium's largest university and one of the oldest universities in Europe, founded in 1425. It is a comprehensive university with 14 faculties, with a long tradition of high-quality interdisciplinary research and teaching. The University of Leuven has over 33,000 students (12 percent international) and over 17,000 staff members (8,600 in the various university departments and 8,700 at UZ Leuven, the university hospital). More info at: www.kuleuven.be
NeuroNova (www.neuronova.com) is a Swedish bio-pharmaceutical company based in Stockholm, Sweden. NeuroNova has two drug candidates nearing clinical development for Parkinson's disease and ALS. NeuroNova works with neurogenesis and neuroprotection for the treatment of several currently incurable neurodegenerative diseases, including Parkinson's disease, Alzheimer's disease, amyotrophic lateral sclerosis (ALS) and Huntington's disease.
UZ Leuven is a university hospital with a reputation throughout Europe as a centre of medical excellence. High-quality, customised patient care, multidisciplinary cooperation, innovation and continuous training all go hand in hand at UZ Leuven. Thanks to the dedication and drive of our motivated staff, we are able to achieve our mission day in, day out.
Mention both VIB and the university
When reporting this research, please always mention VIB as well as the university concerned.