Spheramine presentation

[frank_ger]

Hi,

Dr. Elke Reissig from BayerSchering AG will provide a presentation on the 1st World Congress on Controversies in Neurology to be held 6-9 September in Berlin.
http://www.comtecmed.com/cony/

Title of the presentation: Spheramine cell therapy.
As all patients of the phase IIb study in the meantime got the surgery maybe we will get some preliminary results whether the positive results from the phase I study will be confirmed and as a result this approach can provide a promising alterantive for all people suffering advanced PD.

Information regarding the risks and the safety profile would also be helpful.

frank

[aftermathman]

you at least seem to be one of the guys who remembers there is a human element to drug research as well as a "pump and dump" perspective.

Why not share your conversations with Titan/Bayer Investor Relations staff, as you know Spheramine is a hot topic here.

Good luck to "Titan longs", you win we all win.

Neil.

[frank_ger]

Neil,

of course I would like to share information.
I am in regular contact with Schering and they told me that the immediate risk of surgery is thought to be the same as for deep brain stimulation, but the cumulative risk, including that caused by hardware remaining
in the brain and repeated surgery in the case of electric stimulator implantation, may be lower.

Preliminary safety results from the ongoing double-blind, placebo-controlled
(STEPS) study in 68 patients are encouraging, while efficacy data from this trial will be presented later.

Promising efficacy results from a pilot study in six patients followed over more than 36 months have been observed with no safety complications.

Will keep you informed.

frank

[jllanguille]

My mother was one of the phase IIb study participants. At the time of enrollment, she was scoring 89-90% on the activities of daily living scale while medicated. Within months after Spheramine she went into a nursing home unable to walk, bath, dress, toilet or feed herself. She was one of 2 patients enrolled through UCLA. The other patient died.

She suffers from hallucinations and has to be restrained because of them.
My mother has spent almost 2 years in a nursing home, in the dementia ward, in restraints due to spheramine and yet is aware of her situation. I wouldn't wish that experience on any living soul.

The records attribute her decline and hallucinations as certainly due to the procedure and probably due to the spheramine.

The informed consent states if you are injured care will be provided at no cost to you. This is not the case. To date none of the cost of care has been paid.

[paula_w]

jilanguille,

If you would like to discuss what happened in your mother's trial would you please send me a private message? Was this ever made public?

paula_w

[jeanb]

My mother was one of the phase IIb study participants. At the time of enrollment, she was scoring 89-90% on the activities of daily living scale while medicated. Within months after Spheramine she went into a nursing home unable to walk, bath, dress, toilet or feed herself. She was one of 2 patients enrolled through UCLA. The other patient died.

She suffers from hallucinations and has to be restrained because of them.
My mother has spent almost 2 years in a nursing home, in the dementia ward, in restraints due to spheramine and yet is aware of her situation. I wouldn't wish that experience on any living soul.

The records attribute her decline and hallucinations as certainly due to the procedure and probably due to the spheramine.

The informed consent states if you are injured care will be provided at no cost to you. This is not the case. To date none of the cost of care has been paid.

jllanguille --

I am so sorry - this is terrible. I was confused because I knew that there is a Spheramine phase IIb trial that has just finished recuriting in the past month or so. I did not know there was an earlier phase II trial - which had begun in 2002.

I'm assuming it was in this study (below) that your mother was in? And also BobT - whose post from a different thread I have included as well.

Why aren't we hearing more about this?

Jean

2002 phase II spheramine study:

TITAN LAUNCHES RANDOMIZED STUDY OF
SPHERAMINEÒ IN PARKINSON’S DISEASE


South San Francisco, CA – December 19, 2002 – Titan Pharmaceuticals, Inc. (ASE:TTP) today announced that it has initiated a multicenter, randomized, blinded, controlled study of SpheramineÒ in Parkinson’s disease. The newly launched Phase II clinical study will enroll 68 patients with later-stage Parkinson’s disease (Hoehn and Yahr Stages III and IV) to further evaluate the efficacy, safety, and tolerability of Spheramine, a novel cell therapy under development by Titan and Schering AG, Germany (FSE:SCH, NYSE:SHR), Titan’s corporate partner for worldwide development and commercialization of Spheramine. Schering AG, Germany is fully funding the clinical development program of Spheramine.

Spheramine is a unique cell therapy product utilizing normal human cells attached to microcarriers that enable long-term survival and function of the cells. The particular cells used, called retinal pigment epithelial cells or RPE cells, produce L-DOPA and directly enhance brain levels of dopamine, a neurotransmitter that is deficient in certain brain regions in Parkinson’s patients, leading to movement disorders. RPE cells can be grown in large numbers using cell culture manufacturing methods to produce many thousands of doses of Spheramine from a single starting tissue sample. Spheramine is injected into the brain regions lacking dopamine, using a surgical technique called stereotactic injection, which does not require general anesthesia.


i am a spheramine patient. While results are not complete periodic updates are sent to FDA. At the time of my surgery the results of 55 had been reported with 13 still in process. According to my documents dated August 2006, the first surgery was April 2003, the last June 2007.
Attached is results of 55 in phase 2 directly from my consent form
What follows is only 2 of 17 pages related to spheramine. whie you may see severe reactions by a few, procedure changes resulted. As in anything some will only see the negatives, others a future. there are no edits.........................
A total of 10 sobjects experienced serious side affects possibly related to the study treatment (surgery or spheramine)
Two subjects died. On subject died from cancer of the esophagas, which was considered entirly unrelated to sphermine treatment. The subject also had bleeding in the brain with temporary weakness of one body side, speech problems and seizures, which was thought to be related to study surgery.. Before he died from cancer he had recovered from symptoms of bleeding from the brain. One subject had a fall, broke a rib and acquired pulmonary infection and empyema and died. This subject also had transient confusion after surgery for one day. He then improved and then had markedly better UPDRS scores (by 23%), but later experienced mental and physical deterioration. Brain autoposy from this subject is available, it demonstrated vital hRPE cells on microcarriers in the brain.

One more subject had bleeding in the brain with temporary weakness of one body side, speech problems, and seizure. He also had a low blood pressure (hypotension) and required artificial respiration. These symptoms have meanwhile resolved, but required prolonged rehabilitation. After surgery and changes in the medication, another subject had dyskinesia (violent involentary movement) and difficulty breathing (asthma attack) taht required artificial respiration for a short period. This subject also had minor bleeding on the brain surface which was not thought to cause any symptoms. Two weeks later, this subject had a dilusional episode (period of confusion) accompanied by suicidal thoughts. these events resolved after adjustment of the subject's antiparkinsonian medication.

Another subject had transient (1day) weakness of one arm and an epileptic seizure resulting from the temporary occlusion of a brain vein. The subject recovered completely after one week.

One subject had experienced brief periods of confusion (lasting up to 5 days), which prelonged her post-surgical hospital stay. The confusion occured at the same time as thinking disorders and dystonia. She had additional temporary weakness lasting up to 7 days. She recovered from these symptoms and improved in her PD symptoms, however later in that course of the trial, the neurological status of this subject worsened including mental deterioration. She is living in a nursing home and needs a wheelchair and feeding by a tube going through her nose to the stomach. The causal relationship of this deterioration with Spheramine treatment is unclear.

One subject experienced worsening of dyskinesia one day after study surgery, which prolonged the post-surgical hospital stay. The symptoms resolved.

One subject experienced paranoid delusions and agitation during the night after surgery. Several weeks later, this subject developed depression. His symptoms are completely resolved.

Two subjects required a hospital stay due to paranoid thoughts and delusion that occured several months after surgery, one 14 months and one 5 months after study treatment. It is uncertain if these problems were related to study drug or would also have occured in the natural course of their disease. One of these two subjects was recovered after 3 weeks, one is recovering.

Another subject experienced neck pain since surgery. It was found that he had a broken neck vertebra, however it was unclear if this was related to surgery since he was known to have had neck surgery in 1995, and the fracture was not fresh when it was detected 6 months after surgery.

Based on the findings of an Independent Data Monitoring Committee (IDMC) comprised of internationally recognized Parkinson's Disease experts, the severity and frequency of these serious side effects are in line with the complications that could normally be expected after this type of intervention. In particular, it is not to be expected that such events will occur in association with sham "placebo" operations. Following recommendation of the Chairman of the IDMC, a number of additonal safety measures have been implemented in the study protocol.

In addition, the following non-serious events seen in study subjects were considered to be possibly, probably, or definitely related to surgery and/or to study drug (Spheramine or Placebo). Common events that occured in 5 or more (or >10%) of 52 subjects were: nausea, violent involuntary movement, altered muscle tone, hallucination and headache. The common AEs occuring in 3 or more (or >5%) of subjects were: slow heart beat, weakness, pain, impaired healing, blood pressure increased, balance disorder, disturbance in attention, slow reflexes, confusional state, depression, insomnia, high blood pressure and low blood pressure.

[BobT]

Both the patient who passed and a patient ( i'll now assume is your mother) with a long nursing home stay, are outined in my previous post. As far as after care goes, the doctors who do our followup testing i consider score keepers. If your mother hasnt been seen by her surgeon, thats where I would start making some noise. My surgeon told me of some followups he had done including a DBS on one of his spheramine (evidently placebo) patients. So if he's doing DBS he must be available.

Other avenues include, Call the office of the Institutuional Review Board at the Hospital the surgery was performed. Speak to the Investigator for your study. Formally report conditions or injuries.

If you have no luck with study related personnel, next I'd go to the hospital review board. The last thing they want out there is this type of thing.

Good luck, God Bless

[paula_w]

Thanks to those involved with this trial for sharing information. I'm very sorry for your mother and understand how upset you must be that people are not helping you. I think its important to tell your stories, because we are trying to improve the conditions for participants in PD trials, as are patients of other diseases. Although we have a healthy resentment for Amgen and for good reason about halting GDNF, it doesn't mean we hate all drug companies. However, they have been allowed to cover up far too much information, and communications about trials must increase among all participants, including patients.

We need for people to participate in these trials to survive, but at the same time should not have to tolerate conditions which can be avoided or prevented. It seems reasonable to expect a thorough understanding of the risks and a clear understanding of what is covered post trial at the very least. Just writing it in the consent is not enough. It should be gone over and repeated pre-trial and patients should be checked for comprehension when possible. Any other ideas for inclusion in a Bill of Rights are welcome. You trial participants are the expert witnesses.

Again, please keep us informed and thank you.
Paula

[Thelma]

jllanguille

Thank you for returning with this information. I am so sorry it turned out to be your Mother. We will ask a million questions but you take your time and answer when you can and if you want to.

This has to be a horrendous happening for you and our thoughts are with you.

Thank you again hon

With much respect and appreciation

Thelma

[aftermathman]

I sympathise with your experience and respect your Mother's bravery in participating in the trial.

I remain positive about Spheramine and return to the comment in BobT's post :

"Based on the findings of an Independent Data Monitoring Committee (IDMC) comprised of internationally recognized Parkinson's Disease experts, the severity and frequency of these serious side effects are in line with the complications that could normally be expected after this type of intervention".

Compare Spheramine's safety profile with that of DBS for some perspective. The safety profile for DBS considered by the UK's National Institute for Clinical Excellence DBS consultation paper:

http://guidance.nice.org.uk/page.aspx?o=IP089

quotes DBS related side effects of :

Severe Dyskinesia, Haematoma, Transient Confusion, Stroke, Brachial plexus injury, Pulmonary embolism, Speech difficulties, depression, gait disorder, and death (amongst others) !!

jllanguille, I can only imagine what you are going through and do not wish to trivialise your experience. On balance I hear more good than bad re. Spheramine and continue to be hopeful that it will provide an exciting new option for us PWP.

Neil.

[vlhperry]

Dear jllanguille,

I am so sorry for all you have been through. I hope you were consulted by the doctor, as well as your mother, about the risks. I hope you realize because you may have agreed with your mother, to take the risks, you are not left feeling the guilt.

I don't know if you are feeling guilty, you should have no feelings of guilt. You might allow your anger to work to provide a positive change for future patients involved in research. As Jean said, "How come no one heard about this?"

Reachers have professional pyschologists to keep the family of the patient feel like they are heard and offer symphathy and convince the patient that no one was at fault and how much worse your mother would have been even without participating in the trial.

Remember, you are not alone. Paula is the best person who can offer you help. Take up her offer and Private Message her. She knows who to contact to bring your mother's experience to the light of people directly involved with Research Ethics with the power to make the Research
hospital enforce their contract with the patient. Your mother's participation in the research is heroic. The people involved in holding up their end of the contract are despicable.

Will pray that a higher power helps you make a decision that is right for you.

Vicky

[jllanguille]

The language they use to characterize what happened minimizes it to the greatest degree possible while remaining truthful.

For example "experienced brief periods of confusion (lasting up to 5 days)", means is she has constant, ongoing and frequently terrifying hallucinations that cause her to try to flee, and these began immediately after the surgery and persist until now, 2 1/2 years later. Because of the hallucinations, she is restrained every moment of the waking day.

[Thelma]

You need a good lawyer!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!! !!

[aftermathman]

if you are in regular contact with Schering, (which I believe you are), I am sure we would all be interested in their position regarding jllanguille's Mother and their standard of post operative care.

Can you assist us ?

Neil.

[frank_ger]

aftermathman,

I have already addressed this subject in general to BayerSchering by email.
If I get an answer I will post it here.

I can understand all the people who experienced trouble after the surgery.
I am wondering that obviously nobody can say something positiv about the Spheramine approach given the positive phase I results. What is wrong?

frank

[aftermathman]

any response is sure to be interesting.

Currently there is a lot of frustration in the PD community re. the conduct of clinical trials. This covers, amongst other things, the role of sham surgery, whether trial participants are given the information to allow them to make an informed consent and the standard of post operative care should complications arise, as is seen in this case.

Clinical trials for PD often tread new ground (e.g. gene therapy), and may involve major brain surgery. In addition participants are sometimes told to freeze or reduce their meds and cannot seek alternative meds for the duration of the trial. After all this the procedure may end up being a sham.

Everyone on this board (I imagine), wants Spheramine to succeed, Titan investors moan about the duration of the STEPS trial, question marks over Titan / Schering welfare of trial participants don't help either community.

You're getting some insight into the human investment in drug research, its a difficult place to be.

Neil.

[paula_w]

frank_ger,

This is the first time we've heard about the adverse events. Jllanguille has not much choice but to react negatively, but for others this treatment may hold real hope.

These trials are very risky but then so is living with Parkinson Disease. I think what bothers us often about these adverse events is the lack of compassion and caring for people who do experience these events.

Thanks for sharing your information.

Paula

[BobT]

Who thinks of all the right questions at the right time. We dont realize whats missing until it's needed. As I read Jllanguilles agony I reread the consent form. It is very thorough so I thought I'll quote 2 paragraphs/

"Your doctor may also be an investgator of this researcch study. As an investigator, your doctor is interested both in your critical welfare and in the conduct of this study. Before entering this study or at any time during the research, you may want to ask for second opinion about your care from another doctor who is not an investigator
in this study."

Does the first sentence say "may also be" and the last say
"ask for second opinion about your care from another doctor who is not"

Then paragraph 2
In the event that an injury occurs as a direct result of the effects of your study participation, BERLEX labs agrees to pay for all reasonable and necessary medical expenses not covered by insurance or a third party payer. The sponsor will not pay for medical expenses that are unrelated to the study or attributable to the natural course of any underlying disease or treatment process. The sponsor will not pay for any expenses caused by the institutes negligence or willful misconduct."

I believe herein lies the root of jllanguilles problem.
1- What insurance carrier covers clinical trials?
2-Who determines if the injury is unrelated to the study?
3-Wouldnt negligence have to be determined by a third party.
4-Who else would be qualified to make that diagnosis?
5-Without getting paid?

OK paula, you asked......Patients Bill of Rights
In addition to those questions
When joining the study, a nice option would be for my neurologist to be certified to do my followup testing. No testing happens at the institute he isnt already doing.

A formal outlined (STEPS if you would) process is to be outlined for patient issues that arise. This must require the patient immediate gets seen for care. If thiscare isnt immediately avail. (Im thinking 72 hours) Emergecy procedures for the hospital or state are provided.

Lists of Qualified Doctors not affiliated with the study should be available.

I read somewhere each study patient costs in the vicinity of 150,000 Sponsors could have the option of joining a pool that covers costs such as jllanguilles or post their own bond. It would certainly show who believes in their product Thats enough for now thats the bad thing about this forum is its hard to shut me up

Paula mails coming
Bob
PS Id still do it again

[paula_w]

Bob T,

That is very true, you can't think of everything and don't know what to ask till you need it or something happens. But that is changing....not as quickly as we'd like it to but then we aren't exactly fully staffed and paid employees either,lol. Your comments are what we need because we are trying to create a tool for participants to use. It needs to be in objective terms that are measurable that all involved can check off as done. Don't want to be too picky or unreasonable but anything has to be better than the one sided documents that have been used thus far.

They need to spend more than the 150,000 if that is what it takes now. It shouldn't cost the patients any expenses if they ever expect to get random sampling of all cultures.

ok stopping for now and thank you! Glad I asked! Glad you answered!

paula

[jllanguille]

Bob T,

It is really interesting to me that they changed that clause in the informed consent. I wonder if they did it as a result of what happened to my mother. The clause in the informed consent she signed says:

"If you are injured as a direct result of research procedures your care will be provided at no cost"

When did you sign your contract? I wonder if they changed it because I called them on it for my mom. I contacted Titan/Berlex/Schering in 03/2006. The study was unblinded for my mother and her neurologist told me her precipitous decline was directly attributable to the Spheramine. He said he didn't know what to do for her, because her decline was not characteristic of PD.

[BobT]

Now I am beginning to think I'm risking my continued participation

It says version 3.27.06
Last amendment 6/5/06

Also under a section I thought was unrelated due to heading

Compensation for research Related Injury
If you think that you have been injured by being in ths study, please let the investigator know right away. If your part in the study takes place at XXXXXXXX you can get treatment there.If you part in the study is not at XXXXXXX
ask the investigator where treatment for the injury would be available locally. You and your insurance company will be billed for this treatment. Some research sponsors may offer a program to cover some of the treatment costs which are not covered by insurance. You should ask the research team if such a program exists

I think it time to take a cue from Tena. My apologies for missing this important paragraph It answers alot of my concerns. Glad I said I'd still do it

Shame on me
Bob

[paula_w]

Bob,

The financial factor and post care is popping up with many clinical trials. You certainly don't have to share any more than you want to, but some people have diaries online and I am fairly certain that doctors tell you about confidentiality requirements.

But you inadvertently may have confirmed what we need to improve - not just the wording of the informed consent - but through safeguards and checklists, the responsibility on the part of investigators and sponsors to make sure participants are familiar with it and understand every word of it. You can't memorize it.

goodnight
paula

[BobT]

I/m not aware of anything I am required to keep confidential. Also, as far as this study goes, all the info is provided by a study nurse, then taken home for a week, then return to have Dr go page by page. Next a independent investigator follows up on their thoroughness, abiity to answer questions and their bedside manner so to speak. Short of giving me a test on the material Idont know what else they could do

Bob

[paula_w]

Then for you Bob, it sounds like it's going well and I'm glad to hear it. We are all waiting for these treatments.


paula

[jeanb]

My mother was one of the phase IIb study participants. At the time of enrollment, she was scoring 89-90% on the activities of daily living scale while medicated. Within months after Spheramine she went into a nursing home unable to walk, bath, dress, toilet or feed herself. She was one of 2 patients enrolled through UCLA. The other patient died.

She suffers from hallucinations and has to be restrained because of them.
My mother has spent almost 2 years in a nursing home, in the dementia ward, in restraints due to spheramine and yet is aware of her situation. I wouldn't wish that experience on any living soul.

The records attribute her decline and hallucinations as certainly due to the procedure and probably due to the spheramine.

The informed consent states if you are injured care will be provided at no cost to you. This is not the case. To date none of the cost of care has been paid.


jllanguille -

Your mother suffered a severe adverse event. She now needs full time care - the informed consent states that "care will be provided at no cost to you" but your family is bearing the entire cost of her care. (I don't have long term care available to me. Does anyone on this board? This would begger my loved ones.)

This is outrageous. Maybe it is time to declare a halt to all trial participation until we are treated with dignity and given the protections we deserve. No one should suffer financial hardship for participating in a clinical trial.

Trial participants receive no compensation, but they should have the right to receive medical care -- at no cost to their loved ones -- in the event that things go wrong with the experimental therapy.

Maybe one of the things the participants bill of rights should call for is legal counsel to review the Informed Consent with the Trial Participant. And also have spelled out in legal terms that Trial Participants WILL receive medical care at no cost to them or their families, in the case of an adverse event from the experimental therapy.


Enough is enough.

[tonyd92236]

Adverse experiences like this can be reported directly to FDA, either by the patient, an advocate, or the patient's physician.

Please check out this page: http://www.fda.gov/medwatch/index.html.

The process is pretty simple, and will usually result in oversight by reviewers at FDA.

Tony

[chasmo]

how can you prove that spheramine was even the problem??

An example is it could have been a stroke caused by the implantation process. So is the implanting doctor responsible??

Do we hold Titan responsible for any neurological event???

Just wondering......

Charlie

[paula_w]

Charlie the issue in this case is not who is responsible; it's about the sponsor not holding to the commitment that if a participant is injured, care will be provided at no cost. They are not honoring that commitment and then changed the consent form after these events happened. Their reasons for not holding to it are unacceptable. I have talked with Jllanguille and will let her tell more if or when she wants to. The company is not fighting who is responsible.

I don't want to discourage anyone who is in the Spheramine trial. The participants should be front and center, being treated fairly and with utmost compassion and caring. They shouldn't have to worry about going into financial ruin.

I am not directing today's tirade at you Charlie, just get so angry when I think about it. These patients risk so much.

paula

[chasmo]

hypothetically.

Does Jllangjuille have any legal recourse??

It seems to me that Titan should pick up what her insurance doesn't, at least.

Charlie

[pegleg]

OK, here are your comments from the second participant in the successful Phase I Spheramine trial. Let me begin by saying how very sorry I am for the lady involved in this terrible situation. I will be praying for the entire family.

It has been 7 years since surgery for me - I believe the first was about 6 months before me. There is collective data after 48 months that show that on the average, Spheramine improved the Phase I participants by about 47% the first year, and maintained 43% average improvement after 4 years (note that the surgery was only done for the worst side symptoms). Here is a link to that report: http://www.titanpharm.com/pdf/SpheramineProtocol101_48monthsResults.pdf
This was Phase I/IIa. I am not an expert in trial phases, but I believe due to the fact that the trial was open label (the participants were aware that they were receiving the drug or treatment), it moves the trial phases a bit forward into Phase II. Phase IIb (also known as the STEPS trial) has this report from a news release as of June, 2007:

South San Francisco, CA – June 28, 2007 – Titan Pharmaceuticals, Inc.
(AMEX: TTP) today announced completion of enrollment in the randomized, double-blind Phase IIb clinical study of Spheramine in the treatment of advanced Parkinson’s disease. A total of 71 patients were treated in the study. Spheramine is being developed by Titan in collaboration with Bayer Schering Pharma AG, Titan’s partner for worldwide development and commercialization of Spheramine. Results from the study are expected to be available in the third quarter of 2008.

Results from a previous open label study of Spheramine in six patients, published in the Archives of Neurology1, demonstrated an average 48 percent improvement in motor function over baseline at

1 year after treatment. Additional follow up data, presented at the International Congress on Parkinson's disease in June 2005, demonstrated continued average improvement in motor function

4 years after treatment of 43 percent. Data from this study also demonstrated significant improvement in quality of life for all patients treated, with no significant adverse events.

Parkinson’s disease results from reduced levels of dopamine production and associated loss of function in specific regions of the brain. Spheramine is a novel cell therapy that utilizes normal human retinal pigment epithelial cells (RPE cells) delivered by stereotactic injection into specific areas of the brain affected by Parkinson’s disease.

Parkinson’s disease affects more than one million people in the United States and an estimated four million people worldwide.

Guidance on sales potential and timelines for Spheramine development, including projected launch of Phase III clinical testing in the second half of 2008, were recently presented by Bayer Schering Pharma as part of its Healthcare Investor Day, and can be viewed at www.investor.bayer.com.
Source: http://www.titanpharm.com/press/Spheramine%20press%20release-June%2028%202007.htm

There were only 6 participants in Phase I, and all had their surgeries at Emory in Atlanta by the same neurosurgeon, Dr. Roy Bakay. There was 1 of these 6 who had to drop out of the trial - she was misdiagnosed (not through anyone's fault) and was determined to have PSP (or one of the Parkinson's Plus syndromes). I believe she is the one on a feeding tube in a nursing home, but this is purely speculation. The FDA granted the Spheramine trials Fast Track designation, which is explained below:

South San Francisco, CA – July 12, 2004 – Titan Pharmaceuticals, Inc. (ASE:TTP) today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for Spheramine for the treatment of advanced Parkinson’s disease. The Fast Track Program is designed by FDA to facilitate the development and expedite the review of drug candidates that demonstrate the potential to treat serious or life-threatening diseases and address unmet medical needs. Spheramine is a novel cell therapy product initially being developed for the treatment of advanced Parkinson’s patients who are not satisfactorily controlled with current medications
Source: http://www.titanpharm.com/press/Spheraminefasttrack.htm

As for my experience with the Spheramine trial, it has been very positive. Only now (7 years post-op) have I started having signs of disease advancement (freezing, dyskinesia when just kicking on, dystonia when off, balance and swallowing difficulty when off). I was in pretty bad shape before the surgery. And to further validate that my improvement and slowed progression is most likely due to Spheramine, the side which was not operated on is definitely weaker and more symptomatic (for example, when I first awaken in the morning or when I go off severely, the toes of the unoperated side curl under and I cannot undo them! Also, there is more rigidity on that side.)

I was unaware of all of the adverse events Bob T. mentioned. I did know aboout the gentleman who passed away, and heard through a couple of sources that although he had a brain bleed and was severely disabled by it, he completely recovered in about 3 months. I talked face-to-face with him and his wife. They were OK with their treatment - which I am sure included financial help. He later developed (or it was later discovered) that he had cancer (I believe of the throat) and his death was determined to not be attributable to the Spheramine or the procedure to implant it.

I say all of this not to weaken the case that this thread addresses, but to give you my positive experience. The events Bob lists seem a bit excessive, but I don't know how the Spheramine trial compares to similar trials utilizing stereotactic surgery and/or transplanting cells from a source other than autogolous (from the patient himself). (Recall that the dopamine-producing retinal cells were acquired from a donor eye).

In my opinion, potential and existing participants in ANY trial should have full disclosure of such events as have been mentioned in this thread. I hope this clears up any misunderstandings about the trial phases and is the single "positive" comment in this thread. :)

Peggy

[paula_w]

Decided that this was too personal of an opinion and perhaps upsetting to others.

Paula

[BobT]

I sense frustration Gee really. I thought this thread was on how to improve the consent form and this womans plight was introduced as the vehicle.
A few problems are that all the issues got put in one bucket. Injury reporting, Immediate care, long term care, financial responsibiilty all must be treated seperately. While some of the results seemed less than steller I saw far more good than bad .

Just a reminder those results were postedi in response to someone slamming the study. My heart to goes to jlangulle, but her case was described in the post. As far as I can see she just raised alot of questions for me. We joked as to whether I read the consent form

jllanuille said"The records attribute her decline and hallucinations as certainly due to the procedure and probably due to the spheramine"
The study said " The causal relationship of this deterioration with Spheramine treatment is unclear."
Two completely different diagnoses
Also before jumping to conclusions I read of moms condition but nothimg os the injuy notifiction process and their response.

If I appear to sound negative I am clearly a flag waver for Titan. I just want it to fail or succeed on its merits not on whos louder

My very first post SPHERAMINE "I BELIEVE"
and today more than ever
Bob

[paula_w]

I need to not speak for Jllanguille. So I'm not - just for myself. I'm talking about telling your true stories. All of the areas in a list of rights and responsibilities will be categorized into sections. And talking about your experiences confirms the issues needed to be included. It's that simple. We don't have anything hidden up our sleeves. We are working on the same thing so many of us are lacking in this process and that is trust.

Sleepy,
Paula

[paula_w]

After a night's sleep and an inspiring sunrise, I reread the thread and want to make sure it's understood that I want Spheramine to work. I knew Peg before she got Spheramine - she used a walker. I don't think I've seen her use it since and that was 7 years ago.

But if a life is destroyed, and that is the risk participants actually take, financial burdens or even ruin should not follow. This is asking too much, and will not attract more participants.

We are all on the same side...and wish there didn't have to be sides.

Bob you can believe in spheramine all you want and I hope you are right. But as one person wondered to me yesterday - is capitalism the way to go where drug companies are involved? That's a sobering thought.

Paula

[jllanguille]

I too want Spheramine to work. It is preferable to me that my mother's sacrifice be for something that was of benefit to mankind.

I do want people to really understand the risks involved in research. I think my mother thought the worst thing that could happen was death. I think many would consider death preferable to spending the last years of your life strapped in a wheelchair, in a diaper, hallucinating horribly in the dementia ward while cognizant of your situation.

I think informed consents should clearly convey the risks, sponsors should honor their commitments to provide care, and there should be no financial burden on the patient or their family to participate in research.

[Thelma]

Not enough for me to push an icon and thanks pop up.

So this thanks is directed at you lady and for the help you are giving that goes beyond any thanks.

Your mother must be proud that you have taken her heartache and turned itaround to help others which was for her a part I assume of her particiation in the first place.

Children and in particular daughters don't stray too far from the Mothers who have raised them in love.

Thank you again for want of a better word.

[paula_w]

Come in come in...what are you thinking? Nobody wants to bite although we are pretty good at verbal debate and self defense,but if you can't take a knock, you shouldn't enter in I guess..Jaye, what's the subject? Did you say something about a timer...hee hee.

Bob, I think we all would like to know more about where you are in all this. Many here have it too...advanced. If there is to be anything currently possible emerging neuroregeneratively or even neuro protectively, we haven't found it yet.

We have two choices....we each are given all of the facts and choose whether or not to take the risk

We can be lab rats

We can not participate for many reasons

Make that 3 choices....my math ..:confused:

paula

[BobT]

I'm just the son of a navy man, with 5 sisters, 2 brothers , 4 daughters and a foster son, i have 36 immediate family members and weve been traced back to 1600s with no PD i'm a manager for a major retailer. I live on Cape Cod, vote republican, think Gods greatest gift to man was women and believe life is what you make of it. i was diagnosed in 98 and im 50 in a few months. I believe if your waiting for life to get better, get a blanket cause your done. Wait for nothing. I seek nothing but good and realize some cant, some shouldnt and some wont. Its not my place to judge. I joined this study because theres no magic pill. I realized whatever the solution surgery was invovled. so for me that eliminated my choice. There was none. I'm convinced I have placebo but then again i know Ive got spheramine. I believe in corporate america because people who take risks deserve it when they hit the motherload. I also believe the same for people on the team, the players for whom the team invested. Thats us. Its a different risk. Whetheryou risk your existence or risk happiness by dedicating your time at the risk of family we need to share in the motherload of protection, prevention and cure. God forbid the drug company post profits. Well without Bayer Titan goes bye and with it my light. So heres to quarterly earnings. i have no drug company affiliation, own no stock. MY immediate goal in life is to WALK 4 daughters own the aisle. At 16. 19, 21 and 22 there isnt even a wedding in sight. Did I miss anything?
Bob

[pegleg]

Thanks Bob for sharing your reality check. I could have almost written somee of thhat myself. Those who sit and wait often end up waiting (how's that for providence?)

Peg ;)