Sunday, November 30, 2008

Read this post on a diferent forum and thought I should post it here. This letter was sent by Ron Gunn at INSMED who controll the IPLEX trial in Italy. INSMED Company Statement Regarding IPLEX™ (rhIGF-I/rhIGFBP-3) ALS Expanded Access Treatment Program in Italy This Company Statement is intended to communicate the activities of our ongoing IPLEXTM Expanded Access Treatment Program (EAP) for patients with Amyotrophic Lateral Sclerosis (ALS) in Italy. IPLEX (rhIGF-I/rhIGFBP-3) IPLEX is the trademark for the drug known as rhIGF-I/rhIGFBP-3. IPLEX is a synthetic complex of the growth factor, insulin like growth factor-I also known as IGF-I and the major protein in the bloodstream, IGFBP-3, which regulates the actions of IGF-I. IPLEX was approved by the US Food and Drug Administration in 2005 for a rare growth disorder unrelated to ALS. Until the initiation of the EAP, IPLEX had never been investigated for the treatment of ALS. ALS, often referred to as Lou Gehrig’s disease, is a progressive neurodegenerative disorder that affects nerve cells in the brain and spinal cord. Motor neurons reach from the brain to the spinal cord and from the spinal cord to the muscles throughout the body. The progressive degeneration of the motor neurons in ALS eventually leads to their death. When the motor neurons die, the ability of the brain to initiate and control muscle movement is lost. With voluntary muscle movement action progressively affected patients in the later stage of the disease may become totally paralyzed. IGF-I is a neurotrophic factor which has been shown to be essential for normal development of the nervous system. In animal models, IGF-I has been shown to protect motor neurons from injury and promote muscle and nerve regeneration. IGF-I has been studied as a potential treatment for patients with ALS over the last decade. Two randomized controlled trials have been conducted with a product known as MyotrophinTM which is a synthetic version of IGF-I. The larger trial showed slowing of the progression of functional impairment and improvements in quality of life. The second, smaller trial, showed similar trends but these trends did not reach statistical significance. A third trial is currently underway in the United States with Myotrophin, the results of which may be reported sometime this year. Therefore at this point in time, it is unclear as to whether IGF-I is effective in slowing disease progression in patients with ALS. There is evidence that IPLEX is different from the synthetic forms of IGF-I. The binding protein IGFBP-3, which is a component of IPLEX, modulates the activity of IGF-I. One of the roles of IGFBP-3 is to prolong the length of time that IGF-I circulates in the bloodstream. Incorporating IGFBP3 also blocks the hypoglycemic action (blood glucose lowering action) of IGF-I which allows higher doses of IGF-I to be administered. In order to be an effective treatment of ALS it is important for IGF-I to cross the blood brain barrier. It is possible that by having higher amounts of IGF-I circulating in the bloodstream for a longer period of time, greater amounts of IGF-I may penetrate into the brain. In addition, it could be possible that the IGFBP-3 contained in IPLEX helps to transport IGF-I directly into the brain. For these reasons it is important to study the effects of IPLEX as a treatment for ALS. Expanded Access Program in Italy The Agenzia Italiana del farmaco (AIFA) first contacted Insmed in the summer of 2003 about providing IPLEX to patients in Italy. AIFA stated that the Italian Court had examined the clinical documents of an adult patient believed to be suffering from ALS and had decided that he must be treated with IGF-I. We agreed to provide IPLEX at a price to cover our cost to the first patient in Italy in the summer of 2004. In the fall of 2006, we began receiving several new requests from physicians of patients in Italy diagnosed with ALS who received court orders stipulating IPLEX treatment. At that time we, with AIFA’s guidance, established a formal Expanded Access Program to provide IPLEX to physicians for their patients. As part of this program, physicians are required to collect and provide safety information as well as information pertaining to the progression or improvement in the functional manifestations of the disease prior to beginning treatment and every 3 months thereafter. The functional manifestations of the disease are being evaluated using an ALS Functional Rating Scale which is designed to evaluate speech, salivation, swallowing, handwriting, cutting foods and handling utensils, dressing and hygiene, turning in bed and adjusting bed cloths, walking, climbing stairs and respiratory function. The purpose for collecting this information is to help ensure the safety of patients and to gain an understanding as to whether or not IPLEX treatment provides benefit to these patients. There are approximately 30 patients who are currently enrolled in the EAP with additional patients being added periodically. These patients are located throughout Italy from the Calabria region in the south to the Lombardy region in the north and in Sicily and Sardinia as well. Most patients have been receiving 1 injection of 1 vial of IPLEX per day. For a 60 kg person this is equal to 0.5 mg/kg of IPLEX which contains approximately 0.10 mg/kg of rhIGF-I. Patients in the Myotrophin studies received 2 injections of 0.05 mg/kg of rhIGF-I a day for a total daily dose of 0.10 mg/kg. Therefore, to date, most of the patients in the EAP have received the same total daily amount of rhIGF-I as the patients in the Myotrophin studies. So far this dose has been well tolerated and we have not had any reports of serious side effects related to the treatment. We are in the process of having physicians increase the dose to 1 mg/kg and maintain the patients at this dose in order to evaluate the effectiveness of the drug. For most of these patients, the process for obtaining IPLEX begins with patients petitioning the Italian Court for treatment. Following a court decision stipulating IPLEX treatment, Insmed is contacted by the treating physician or the patient’s pharmacist. We in turn, provide instructions to the physician and/or pharmacist along with the relevant documentation for the physician and pharmacist to fill out. Once all of the required documents and import licenses are in place we ship IPLEX to the patient’s pharmacy. Insmed recognizes that ALS is a devastating disease and that there are patients throughout the world who are in need of an effective treatment. However, the Expanded Access Program is restricted exclusively to Italy through license agreements with companies who hold the patent rights for the manufacture and use of IGF-I products in the treatment of ALS. Insmed does not currently have the rights to manufacture or distribute IPLEX for the treatment of ALS outside of Italy. Therefore we are unable to provide IPLEX to physicians for the treatment of their patients anywhere else in the world.

Wednesday, November 26, 2008

when you make the "THE PRAYER LIST" you know you're sick.....

To all: Please pray for the following:
1-Jenna-Thanksgiving-successful surgery and clean MRI
2-Brian Moran, KM-repose of soul
3-Melody Garges-now in hospice-Melody has been battling cancer and side efects for so many years. Her parents, Tom and Sarah Flood, are two of the greatest people/parents in the world-and great examples of faith within the Order of Malta. Please pray and storm the heavens for special healing for Melody and God's Peace as well!
4-Baby Presley-special healing
5-Drew S.-special healing
6-Kirsten Lawley-38 year old mother with breast cancer and 4 children under 9.
7-JB-cataract surgery on Tuesday
8-A D'A--Alzheimer's disease
9-Seton Fell-special healing
10-Kim Renzi-special healing
11-Jim O'S-special healing
12-Kevin H-special intentions
13-Kennedy S.-special healing
14-Devon Lam-Thanksgiving- Devon has stabilized and may be returning home this week. Continue to storm the heavens for wee Devon!
15-Mike P.-guidance for right treatment of lung cancer
16-Kari-special intentions
17-Jonathan R.-special intentions
18-Keith Cassidy-ruptured appendix
19-Joanne P-Thanksgiving-prayers answered, but still 5 more days for novena!

Tuesday, November 25, 2008

Drug used by ALS patients gets closer to distribution
By Elizabeth SimpsonThe Virginian-Pilot© November 26, 2008
A drug that some patients with a degenerative nerve disease have been clamoring to get for more than a year has moved a step closer to being freed for distribution.
Iplex, made by Richmond-based company Insmed, had been used in an off-label fashion by some patients with amyotrophic lateral sclerosis, commonly known as Lou Gehrig's disease.
But it was taken off the U.S. market in 2007 when California-based company Genentech sued Insmed, saying it used a component licensed exclusively to another drug company, Tercica.
That resulted in an outcry from patients who were using Iplex and others who wanted to. People with ALS progressively lose control of their voluntary muscles, such as swallowing and breathing, and eventually become paralyzed. Life expectancy after diagnosis is usually two to seven years.
In response to patients' concerns, Genentech, Tercica and Insmed signed a letter of intent earlier this month freeing Insmed to supply the drug if it receives regulatory permission from the U.S. Food and Drug Administration.
The drug still has not been through rigorous testing, nor has it been approved by the FDA for use by ALS patients. People who want the drug are hopeful that will be the next step. A phone call to Insmed for comment on such progress was not returned Tuesday.
Attorneys at the local office of the Richmond-based Williams Mullen recently got involved with the effort to free the drug for distribution after hearing that Josh Thompson, a Virginia Beach resident and son of prominent developer Bruce Thompson, wanted access to the drug.
Elizabeth Simpson, (757) 446-2635,

the rebuttal

IPLEX is way more than just a growth hormone, better said it's -- IGF-I with a synthetic that binding protein BP3. -- I won't even go into the under dosing aspects of clinical trials .
I totally understand that most people are caught in the middle of uncertainty a doubt created by the so called "Scientific Community" which have as a job to burst any bubble of hope. No doctor is willing to support anything unless they see absolute & categoric "evidence" because lawyers are their biggest concern. I wonder if Dr. Sorensen from the Mayo Clinic or any other neurologists at any of the major neuromuscular centers in the United States (or the entire world for that matter) are willing to share their justification (or evidence) to support prescribing Rilutek, which again almost every major neurologist in the world and least officially supports.
Like I said, doctors are paralized by the fear of getting sue. Most are not willing to go outside their little comfort zone, in the name of "evidence" and are absolutely not willing to stick out their necks for what is right, concerning their patients.
Our medical model (just like the rest of our country's systems and processes) are being held hostage by lawyers, big pharma and special interests. It is a system that practices a profession based on fear, arrogance , cash, and absolutely no guts.
Very few doctors are brave enough to stand up to the official way of thinking, at the expense of being labeled quacks, getting sued or losing credibility with colleagues.
But they're are out there. There is an organization which lists many innovative doctors across the country, I believe that once they're brought up to speed on IPLEX they will be more than willing to prescribe it
But that's just one man's opinion .
Eddie Spaghetti) Esparza
"Si Se Puede"(YES WE CAN!!)
Cesar Chávez

Disappointing Results from IGF-1 Clinical Trial

Disappointing Results from IGF-1 Clinical Trial
By Richard Robinson, Science Writer
Subcutaneous (under the skin) delivery of insulin-like growth factor 1 (IGF-1) 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 released today.
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

Monday, November 24, 2008

Foes of stem cell research now face tough battle

Foes of stem cell research now face tough battle
By KEVIN FREKING, Associated Press Writer Kevin Freking, Associated Press Writer Sun Nov 23, 12:05 pm ET

WASHINGTON – When the Bush presidency ends, opponents of embryonic stem cell research will face a new political reality that many feel powerless to stop.
President-elect Barack Obama is expected to lift restrictions on federal money for such research. House Speaker Nancy Pelosi, D-Calif., also has expressed interest in going ahead with legislation in the first 100 days of the new Congress if it still is necessary to set up a regulatory framework.
"We may lose it, but we're going to continually fight it and offer the ethical alternative," said Rep. Joe Pitts, R-Pa. "I don't know what the votes will be in the new Congress ... but it's very possible we could lose this thing."
Stem cells are the building blocks that turn into different kinds of tissue. Embryonic stem cells,e unlike more mature versions, are blank slates. If scientists could control them, they could direct regenerative therapy, perhaps allowing a diabetic's pancreas to begin produce insulin, for example.
Harvesting stem cells from four- or five-day-old embryos kills the embryo, which outrages opponents of this type of research. But supporters say hundreds of thousands of embryos stored in fertility clinics eventually will be destroyed anyway and that people should be allowed to donate them for research that could help others.
"I believe that it is ethical to use these extra embryos for research that could save lives when they are freely donated for that express purpose," Obama wrote during the campaign in response to 14 questions from scientists, doctors and engineers.
Under President George W. Bush, federal money for research on human embryonic stems cells was limited to those stem cell lines, or families of constantly dividing cells, that were created before Aug. 9, 2001. No federal dollars could be used on research with cell lines from embryos destroyed from that point forward. Federal regulations do not restrict embryonic stem cell research using state or private funds.
John Podesta, head of Obama's transition team, strongly hinted that the president-elect would deal with stem cell research soon after taking office Jan. 20. "As you know, he has said something specific about stem cell research, so I think you can expect that what he said in the campaign will be fulfilled once in office," Podesta said.
Obama made it clear during the campaign he would overturn Bush's directive.
"As president, I will lift the current administration's ban on federal funding of research on embryonic stem cell lines created after August 9, 2001, through executive order, and I will ensure that all research on stem cells is conducted ethically and with rigorous oversight," he said.
Opponents of such research say they will press their case on several fronts.
The main argument is that life begins at conception — that once fertilization occurred in the lab, so did a human being.
Secondly, they will argue that scientists are having success using other methods — adult stem cells that form specific tissues, or reprogramming skin cells to act like stem cells — so money should be directed where the biggest scientific breakthroughs have occurred. For example, this past week, doctors gave a woman a new windpipe with tissue grown from her own stem cells, eliminating the need for anti-rejection drugs.
"We still intend to try and talk about the real facts that it's the adult stem cells providing the actual treatments," said David Prentice, senior fellow at the Family Research Council.
Added Wendy Wright, president of Concerned Women for America: "There's a lot that's happened over the seven years that includes some remarkable scientific discoveries, which really should have made the issue of federal funding of embryonic stem cell research moot."
But Sean Tipton, director of public affairs at the American Society for Reproductive Medicine, took aim at those arguments.
"It's a little disingenuous for opponents who have effectively blocked federal funding of the work to then cite a lack of progress," Tipton said. "You hold someone at the starting line then you criticize them for not getting very far."
Dr. Chi Dang, professor of medicine at the Johns Hopkins University School of Medicine, agreed there have been tremendous advances with adult stem cells. But he said it is not yet clear that they have enough flexibility to be used in all the ways that an embryonic stem cell could be.
"From a scientific viewpoint, we would be cornering ourselves into generalizing things that may not be true," Dang said.
Dang also said these embryos would otherwise be discarded.
"The question is: Is it ethically more acceptable to destroy these embryos by pouring acid on them, or do you deploy these clusters of cells to create new cell lines that could benefit us in the future?"
Samuel Pfaff, a professor at the Salk Institute for Biologic Studies, said he also supports greater embryonic stem cell research to understand what makes them so special that scientists can endow other cells with similar properties.
"I think it's very fair to say that the long-term trajectory for this area of science is to understand embryonic stem cells so well that we don't have to use them anymore." Pfaff said.
On the Net:
Stem cell information at the National Institutes of Health:
Candidates answers on embryonic stem cell research:

iplex update

Dear friends,
Wanted to provide all concerned, a brief update on our current status with Iplex. Insmed has been in contact with, and assured the group leaders of Team IPLEX, that they have begun the process of clearing regulatory hurdles both in the USA and other countries. Insmed has also assured us that the production demands of those currently expressing an interest in Iplex are no barrier at all. While we are all anxious and ready to proceed at once with Iplex, alas, the wheels of the machine must turn and all regulatory obilgations must be met in order for Insmed to legally provide the drug to PALS. This process should be relatively quick, (all things considered).

I regret that there is little more to report at this time. There is little that we, Team Iplex, could, and probably should, do at this time to encourage the momentum of this process. Our continued efforts as a group have acheived what no one thought possible. Any further collective efforts, such as writing/calling, etc, may only serve to distract from Insmeds mandatory compliance with regulatory processes. We (Team IPLEX) have been assured that the second that the regulatory process is in place, it will be updated on Insmed's Corporate Webpages. We have been also assured that we would be notified/get a call outlining the details. However, I would still encourage everyone concerned, to check Insmeds web site on a regular basis.
I just can't tell youi how much it has meant to me and my family, to have been on this journey with all that have helped to make this possible.

To think that a small group of patients and caregivers fighting with all of our might, with a never say die attitude - could make such an incredible difference and have influence on multi-billion dollar companies is pretty incredible, at least that's the way I feel.

Friday, November 21, 2008

Neuralstem enters stem cell collaboration for Huntington's disease in Germany

The collaboration with Professor Nikkah will focus on Huntington's disease.
"We are pleased to have established this collaboration in Germany" said Richard Garr, Neuralstem President & CEO. "The goal of our work with Professor Nikkah will be to qualify our existing cGMP spinal cord cells into Professor Nikkah's human trial program to treat Huntington's disease. As we prepare to submit an IND to treat ALS with our stem cells in the U.S., we continue to look for strategic relationships in both Europe and Asia which will allow us to move the cells into humans. We believe that Professor Nikkah's program is the most advanced of its kind in Western Europe, and we are excited about his working with our cells."
Huntington's disease (HD) results from genetically programmed degeneration of brain cells, called neurons, in certain areas of the brain. This degeneration causes uncontrolled movements, loss of intellectual faculties, and emotional disturbance. HD is a familial disease, passed from parent to child through a mutation in the normal gene. Each child of an HD parent has a 50-50 chance of inheriting the HD gene.
Neuralstem'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 and ALS. 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. The Company expects to file its first IND (Investigational New Drug) application with the FDA for ALS in the fall.

Tuesday, November 18, 2008

pics of my travels to hangzhou, china for stem cells

i broke the bed w/ my leg

angie and my sis

they threw me a going away
they're awesome people

that is my PALS/friend Lenny-he has my challenge
that is Robin to my left-she planned the party-great lady

me miserable w/ needles in face and hands