Crohn's Disease Relief

Now I Understand my Fruit Allergies

Since I was a kid I have gotten an itchy painful mouth if I ate Pineapples.  Over time I have figured out I get a similar allergic reaction from various fruits.  Mangos are the worst, pineapple, kiwi, papaya, and cherries.  When my baby son Ben got a itchy diaper rash and red around his mouth after eating pinapple, I knew he had it too. I keep both my kids away from the fruits that I am allergic to although I have a feeling they would probably be fine if they had some.  Well, it will give them something to rebel against when they are older.  Funny story about my mom and artichokes - she is allergic and would never let my brother have them.  Turns out he is allerigic to them too.  Not me though.  But my Dad sneezes if he touches or eats peach fuzz. 

Anyway, this great CNN.com article about Fruit Allergies and Pollen Allergies discusses the mechanisms of a fruit protein oral allergy and was really interesting.  Apparently the proteins in the fruits look very similar to those in pollens and this can explain how the allergy works.  It also explains why canned or cooked fruit don't effect me as much, as the cooking process alters the proteins.

Did patients like Gideon Sofer kill Prochymal's Phase III Trial?

Osiris Therapeutics has halted registration for the Phase III trial evaluating Prochymal for Crohn's disease due to flaws in the study methodology.  Apparently, there was an overly large percentage of people taking the placebo who reported positive results. There has been some speculation that patients may have underreported their symptoms if they felt they were getting the real medication possibly because if they were on the medication in the Phase II trials and were experiencing good results they would be given the medication in the Phase III trials.

My opinion - being that Crohn's disease is totally effected by stress and emotions (at least in my experience it is) is is also possible that people in the trial who started to feel better assumed they were on the real drug and then the euphoria of having a potential miracle cure helped them to feel even better. 

Osiris stops enrollment in Crohn's disease trial

This news is very disappointing not only for investors in Osiris, but for Crohn's patients like myself.  This will push back the approval for the drug to treat Crohn's and a whole new set of study parameters will be devised to try to make sure they have a true result with no problems with an overly large placebo effect.

I find this news very ironic given the squabble that some Crohn's patients had with Osiris regarding the ability for people who got the placebo in the study to get the real drug in a follow up study.

Wall Street Journal Reports: The FDA is Killing Crohn's Patients

I think that this is a lesson for us all in terms of letting the scientific double blind study methodology take its course and try to keep human emotions out of the process as much as possible. 

We want to know if the drug is effective and if it is safe.  There were no problems with adverse reactions or safety issues in the studies - they simply need to be redone to be able to prove the drug works better that no treatment.

I am very sorry to chide Gideon Sofer, who I vastly admire.  I sincerely hope that he is doing better, as I know his Crohn's is very severe.  I think he would understand my *shock value* headline, which is an homage (and a criticism) to his piece in the Wall Street Journal.  I do think it is possible to both admire and criticize someone.

Is Osiris a Growth Stock Waiting to Happen?

People are talking about Osiris Therapeutics (OSIR) as a potential biotech stock for investment.  Here is a blog post I did about the company and its estimated market for the drug in 2010.  By the way, as a company with a high R and D costs and the big product in the future, it has an astoundingly high PE ration - PE (forward) 94.74.

235 Million Market for Prochymal estimated for 2017

Here is another post about some controversy over how they are running the double blind studies.  Patients who assume they got the placebo are asking for the actual drug.

Is the FDA Killing Crohn's Patients?

Here is a quote from Zacks.com.

Here Come the Stem Cells

Finding the next big thing is certainly something biotech investors are always in searching for. Above we discussed our enthusiasm for antisense technology as powerful drug discovery engine. Perhaps the only other discovery platform that provides as much enthusiasm as antisense is stem cells. The leading stem cell company is Osiris Therapeutics (OSIR).

Osiris is developing Prochymal, an adult stem cell product currently in 3 phase III trials for steroid refractory graft-vs-host disease (GvHD), acute GvHD and Crohn’s disease. Prochymal is also in a slew of earlier-stage clinical programs for diabetes, myocardial infarction, chronic obstructive pulmonary disease and acute radiation syndrome. It’s a potential wonder-drug built on a the premise that viable adult mesenchymal stem cells (MSCs) can engraft and selectively differentiate, based on the tissue environment, to such lineages as muscle, bone, cartilage, marrow stroma, tendon and fat.

Research shows that MSCs offer anti-inflammatory, anti-fibrotic and regenerative properties that make this platform an exciting disease modulator treatment engine for discovering new drugs. Investors can pick up one share of Osiris for just over $17 per share.

is the company that is doing those adult stem cell trials for Crohn's diseaseI have heard that many people who participated in the trials reported excellent results.  The people who had no improvement assumed they were on the placebo.  All the Crohnie's are looking forward to furthing testing and availability of this treatment.  The alternative for serious Crohn's is biologic treatments like Humira, and they appear to have a cancer risk associated with them.

Obama Orders Complete review of FDA Operations

In a White House interview which aired Monday, President Obama announced he will order a complete review of FDA operations.  Coming after the salmonella outbreak linked to tainted peanut butter which sickened over 500 people and is linked to 8 deaths, this overhaul will surely also include the drug related half of the agency. 

Obama said that there were numerous incidents which should have been caught by the FDA but were not found in time.  Critics have accused the Bush Administration of cutting the FDA budget to the point making it no longer able to properly respond to threats to public safety.  Obamas announcement of an overhaul of the FDA will be well received as food safety is a hot button issue for Americans.  The question remains: How will this overview effect the drug regulatory agencies attached to the FDA? Obama has already announced the lifting of Bush's ban on new lines for human embryonic stem cell trials.

The economic stimulus package awaiting vote by the Senate, includes $1.1 billion  funding to study the effectiveness of healthcare treatments.  The FDA has also requested access to more records during routine food inspections in response to the peanut butter recalls.

The position of FDA head is currently needing to be filled.  Dr. Joshua Sharfstein, the Baltimore health commissioner, has been floated as one posible choice.  Obama has been advised to select an agency outsider to the position of FDA head.  Sharfstein is current Baltimore health commissioner.  Congressional Representative Bart Stupak of Michigan, a member of the panel that oversees the FDA, has said that he is opposed to the appointment of any current FDA employee to the position of FDA head.  Stupak has previously said that the agency needs a top-to-bottom overhaul. Obama's news conference this weekend shows that he is in agreement.

   

Staying Up To Date on Crohn's News

I am testing out the following widget from Topix that will show current news articles referencing Crohn's Disease. 


I also learned about a wonderful Twitter stream from a guy who "tweets" news and links about Crohn's. It's partly in German, partly in English but a lot of the links are to sites in English so its very useful to me.

SMCCV's Crohn's Twitter Stream

Crohn's Stem Cell Roundup

CNN has a very nicely laid out "Stem Cell Primer" section that explains in layman's terms some of the science behind stem cells - CNN Stem Cell Primer.

This article titles Could Stem Cells Be Used To Cure Crohn's Disease? From Science Daily focuses on the idea of using the patient's own stem cells to "reboot" the immune system.

Last week I wrote about the new FDA aproval of the first embryonic stem cell test, which will attempt to bring back movement to people made paraplegic due to an accident.  Somewhere, I think Christopher Reeve is smiling.

And in December I wrote about a controversy surrounding the FDA and Osiris and how they are managing the trials for Prochymal. 

Christopher Reeve's Dream Comes to Fruition: Embryonic Stem Cell Test Approved

Geron Corp., of Menlo Park, Calif., announced today that the Food and Drug Administration has approved their study of a stem-cell based treatment for spinal cord injuries.  Geron will be able to test the stem cell therapy in up to 10 patients who are within 14 days of a spinal cord injury leaving them paraplegic, able to use their arms but not their legs. Study participants will receive one injection within 14 days of their injury and will then be monitored for one year afterward.

Geron and the FDA both state that that the approval for the study coming on Obama's second day in office is a coincidence this approval seems an obvious signal of a sea change in the US government's position on stem cell research. George W. Bush had imposed such strict limits on embryonic stem cell research that this promising branch of medical therapeutics was practically stifled.  The Obama administration seems to be cautiously reversing these limits as promised in the campaign.  Cautious being an operative word.  On Jan. 18 on CNN, Obama said "I like the idea of the American people's representatives expressing their views on an issue like this."  

It was the dream of Christopher Reeve, and his wife Dana that advances in stem cell research could allow paralyzed victims of injury to regain their mobility.  Their foundation, The Christopher and Dana Reeve Foundation has championed stem cell research, however, the Bush administration had hampered the ability for companies and universities to use stem cells due to strict limits imposed by George W. Bush and Congress. Although there certainly are moral and ethical considerations revolving around the use embryonic stem cells, the fact is that there are embryos which remain frozen after the process of in vitro fertilization.  As a parent, many feel that they would gladly donate these to allow medical science to potentially save lives and help the paralyzed walk again.   

The FDA has been reviewing the application for the Geron study for almost a year.  It will be the first US study to  use embryonic stem cells.  The focus of the study is on differentiating stem cells roles in regenerating the spinal cord.  Other studies using adult stem cells have shown great promise in the treatment of diseases such as Crohn's Disease and Graft Vs. Host disease.  What is exciting about embryonic stem cells is that they can evolve into any cell in the body.  

Wall Street Journal Reports: The FDA is Killing Crohn's Patients

In a shocking article, Crohn's patient accuses the FDA of killing him.  Gideon J. Sofer is a Crohn's sufferer who has taken part in the Osiris Therapeutics Phase II trials of their new adult stem cell therapy Prochymal.  Because Osiris has stated that all of the patients that received the actual drug in the trials reported positive results, Mr, Sofer understands that he must have received the placebo. He has been in and out of the hospital and his disease is progressing.  His doctors have asked Osiris to give them information on whether he received the real drug or the placebo in the double blind tests, but they will not tell them.

Gideon Sofer wants the real drug, but even though it is on Fast Track for approval by the FDA, it might be years before it is available.  Read this quote for the most shocking part of the issue:

Typical approval protocols almost always guarantee patients taking the placebo access to the actual drug -- at the very least -- after the study has ended. But in what appears to me a deliberate act of cruelty, Osiris hung its patients out to dry without any recourse, refusing to confirm which patient got what. The FDA has endorsed Osiris's decision by enabling it to proceed with the study.

I am of two minds on this issue.  One the one hand, I do understand the value of Double-Blind scientific testing of new medications.  Otherwise we will not properly know if the product really works.  Also, they need to study the long term effects of the drug.  Only look at the new black box warnings on Humira and Remicade to understand that what seems like a miracle drug can sometimes have unintended longer term side effects that need to be studied as well as the immdiate results.

On the other hand, if it is within the scientific protocols to allow a patient who received the placebo in a trial to receive the actual medication after the trial period is over, why will Osiris not allow this to occur in this case.  In a case where the disease can lead to a huge reduction in quality of life, extreme pain, and even the possibility of death, can't they bend?

I am personally really looking forward to see the long term effects of Prochymal.  I beleive that stem cell therapies are a beacon on hope for myself and others with a wide range of diseases.  However, I am also a bit concerned about long term effects and would like to know that a product is safe.  I am avoiding taking Humira, although it is the planned next step in my treatment plan, because of two reasons.  One, I want to save the "big guns" of biologic treatments as long as possible so as not to run out of options.  Two, I am afraid of the possible increased cancer risk that may be associated with the biologic treatments.

Although my "mild" Crohn's causes me pain, fatigue, and has hospitalized me, it is not threatening my life at this time.  In fact, Crohn's is not generally considered to be a killer disease, which may be part of why Osiris doesn't want to bend their study rules and provide the medication to their placebo subjects.  However, Crohn's can cause death, usually in terms of complications from surgery. 

I really feel for Gideon Sofer's plight, and I hope that Osiris changes it's mind.  I realize that they have a potential multi-million dollar market for Prochymal and they want the trials to be scientifically sound.  But please, people at Osiris/Genzyme - reconsider and help your study subjects. 

And two other requests: to the FDA I plead that you are able to properly balance the need for testing and study with the need for fast tracking drugs that have the potential to save lives for people that simply cannot wait.  And to the President, please create funding opportunities to allow all appropriate uses for stem cells to save lives.   G.W. Bush and his opposition of stem cell reaseach are about to be out the door.  Please let's have a government environment that allows scientific research to thrive.  We need it!

Anal Fissures hit the Ophrah Show

Oprah has a way of finding interesting topics and bringing them to light.  Recently she did a show on embarrassing problems that people have a hard time sharing with their doctors. As a Crohn's patient, I sure have had those!  When I first went into the hospital (later to be diagnosed with Crohn's) I had a yeast type infection of the groin area.  That was not fun to have to show the doctor.  I guess my immune system was so depleted by the Crohn's inflammation, that the yeast infection took over.

Back to Oprah, one of the problems she and her favorite guest, Dr. Oz covered was anal fissures.  Since this is a common Crohn's symptom I was glad to see it get some national coverage.

I found a very thorough and informative article about anal fissures on UpToDate.com and was very impressed by how they pretty much tell you all you need to know. 

I bet my mom might be mad at me for writing that she had a fissure that she had treated with a botox injection.  We were joking at the time that she had the youngest looking butt anywhere! And if they had some extra they could take out a couple wrinkles on her face while they were at it.

Veterinary college develops vaccine for Johne's disease

In medical news this week I read that scientists at Cornell Veterinary college have developed a vaccine for Johne's disease.  Johne's disease is an animal disease that effects cattle, and other animals and has similar symptoms to Crohn's disease in people.

Here is a quote from Yung-Fu Chang, Cornell professor of microbiology and the paper's principal investigator, via physorg.com.

"Equally important, though, this organism has been suspected to be one of several possible contributing agents to Crohn's disease that has similar pathologic lesions in people. The results of our research may offer useful information to those working with Crohn's disease."

One of the frequently cited "conspiracy theories" about Crohn's disease is that there is a connection between the bacteria shown to be the cause of Johne's disease in cattle and Crohn's disease in humans.  This link has not been proven in scientific tests, but their remains speculation that there may be some link in the diseases.  It may be that the Mycobacterium avium subspecies paratuberculosis bacteria (MAP) that causes Johne's disease in cattle and other animals is a trigger factor for Crohn's disease in humans. MAP bacteria is not killed by the current methods of pasteurization of cow's milk.

I am intrigued by the idea that if there is a vaccine for the bateria for use in cattle, if that means there might be some further study that  could be done for a similar vaccine for humans.  There are already certain genes that have been identified in people that seem to show a highter pre-dispsition to the Crohn's.  I wonder if people that are shown to have these gene's could get a vaccine against MAP bacteria.