Red Hill RHB 104 Clinical Update and Why The FDA Should Approve It

Recently Redhill announced results in July 2018 for a Phase III trial for their drug RHB-104. RHB-104 is an oral pill (no infusions or shots) that uses a combination of antibiotics to treat Crohn's disease. The thinking behind the drug is that Crohn's is a bacterial infection caused by mycobacterium avium subspecies paraturberculosis (known as MAP for short) which is an infection often found in animals. This infection is believed to contribute or cause Crohn's disease. The theory if you have a medication of anti-MAP it can get rid of the infection and put the Crohn's into remission.

I documented in this post of a woman named Julie Doyle who had surgeries and had tried Humira, Remicade, and every other therapy for Crohn's and then finally tried the anti-MAP therapy (similar RHB-104) and within 6 weeks Julie added 10 pounds and within one year she had a colonoscopy that showed the Crohn's was in remission. This presentation shows patients that have taken RHB-104 with the before and after photos of the colonoscopy. This article discusses a man in Australia who took 45 minutes to get out of bed from his Crohn's and unable to function until he took an anti-MAP therapy and has been off medications for over a decade and has an "unrestricted life".  This presentation discusses a man named David who has been in remission for anti-MAP therapy since 1996. Although, these are only anecdotal stories it is important to know that this therapy has literally changed the lives of individuals and families.

The results of the most recent RHB 104 study showed that after 26 weeks the percentage of patients that had remission was 37% vs 23% for the placebo group. The percentage of people achieving remission from weeks 16 to week 52 taking the medication was 18% vs a 9% for the placebo. If someone who had been flaring for years had almost a 1/5 chance of finally being in remission I would ask my doctor how to get that medication. At the 1 year mark 27% of individuals were in remission vs 20% (this result was not statistically significant)

Studies on anti-MAP therapy have been occurring since 1997.  Even before this most recent trial occurred over 400 patients had been in clinical trials. The most recent trial for RHB-104 had 331 patients involved which would put the total number of patients over 700.  Also it important to note that there hasn't been serious side effects reported with anti-MAP treatment as opposed to the placebo.

Redhill will be meeting with the FDA to discuss a potential approval. I honestly hope the FDA approves this drug as many people suffer from Crohn's: are in misery, have no alternatives, and are open to try this drug. It is important to note that this drug has a pretty good safety profile. The drug has been used in clinical trials and on patients for over 20 years. How much longer do patients suffering have to wait? Another drug to treat Crohn's should always be welcomed as another tool in the tool belt for doctors and patients.

My view as someone who has suffered with Crohn's I have had times when I didn't know when a flare would go away and before I took Humira I can still remember the shooting pain in my stomach that felt like a blunt object stabbing away and having to run to the bathroom urgently. The data shows that RHB-104 and anti-MAP therapy can offer remission to some patients who have tried other medications that have failed. Also given these studies have occurred since 1997 provides  years of patient data and to  my knowledge there haven't been serious side effects over the years the drug should be approved for patients to see if it can help them. Life is short and we deserve to live the best life possible.

Low Dose Naltrexone for Crohn's: The No-Brainer Case For The FDA

         
           (This picture is of a colonscopy of a before and after of a Crohn's patient who took LDN)

So in a prior post I discussed low dose naltrexone (LDN). LDN was first introduced in 1984 for people with alcohol dependence. The drug has been shown to reduce the relapse rates. What inspired me to write this post was I saw this recent story that talked about how drug company TNI BioTech met with the FDA and Phase III trials may start as early as first quarter in 2014.

The evidence for low dose naltrexone is pretty convincing in terms of being both effective and safer than the current alternativies of Humira, Remicade, and other drugs. This land mark study from Dr. Jill Smith at Penn State University showed that 67% of people went into remission. 89% of patients showed a clinical response which is similar to the rates of Humira and Remicade (however the only real side effects with LDN is vivid dreams and fatigue). In 2011, this study was done by Smith and her colleagues which found that 78% of patients showed an endoscopic response. 88% of patients who took the LDN showed a 70 point decline in their CDAI score (which is a significant decrease). The only side effect was fatigue.

A recent study from April 2013 in children showed that low dose naltrexone was safe and showed reduced activity for Crohn’s patients. However, only 25% of children (between 8-17 years of age) went into remission, however 67% showed improvement. Here is a case study of a 14 year old girl who had Crohn's and pain for 3 years. After a month of taking 4.5 mg of low dose naltrexone she improved. An EGD (esophagogastroduodenoscopy) showed complete muscoal healing and normal biopsies which is quite impressive.

LDN has been shown to be useful in other treatments like multiple sclerosis, prolonging the live of pancreatic cancer patients, and may even help people with HIV and AIDS. There is no question some more studies have to be done on what LDN can exactly help.

Why on earth is this drug still not approved for Crohn's? I would suggest the FDA talk to patients with Crohn's and see how they currently feel given the current state of options in the Crohn's world. Low dose naltrexone seems to show help patients with minimal side effects compared to Humira, Remicade, Enbrel, 6-MP and any other drugs you want to compare it to. The initial study for LDN on Crohn's was done in 2007. So why is it taking 7 years to now just get around to a Phase III trial? 

Fecal Transplants Come To Houston

This evening I received an e-mail from the University of Texas-Houston stating they were beginning to start fecal transplants for c difficile and for patients that failed conventional IBD therapy. The study being performed will see whether it is better to have fresh stools or frozen stools when doing the fecal transplants. Patients for c difficile have to have had 3 or more recurrent infections while IBD patients have to have had conventional therapy fail.

Dr. Herbert DuPont is heading a large fecal transplant program in Houston. He is Director of the Center for Infectious Diseases at The University of Texas Health Science Center at Houston. According to PubMed as I write this he has over 500 publications. He really seems to be an expert in c difficile and diarrhea.  His curriculum vitae is 71 pages long (he is a busy guy).

This is great news at now it looks like the FDA will be backing off major regulations which will allow more people to get this relatively effective and safe procedure. It will be interesting to see how many IBD patients are treated and the results. Dr. Borody in Houston has had success treating IBD patients (more on the ulcerative colitis side). However from the studies I have seen Borody did enemas multiple times instead of doing the fecal transplant via colonscopy or through the nose. At any rate this is great news if more people now can be exposed to fecal transplant and get rid of the debilitating and crippling effects of c difficile.

If you are interested in this procedure you can contact Dr. Dupont with his info here 

FDA Meeting Workshop Transcripts With Fecal Transplant Doctors: More Regulation = Less Fecal Transplants

I found the transcripts from the FDA meetings in Bethesda, Maryland from May 2-3, 2013. Day 1 is here while Day 2 is here. If you add both documents it is over 600 pages. The first day wasn’t too interesting as it just the biology of fecal transplants and to be honest much of it I did not understand. The second day was more worthwhile because doctors who are actually performing fecal transplant presented what they have seen.  The second day included Dr. David Rubin, Dr. Lawrence Brandt, Dr. Colleen Kelly, Dr. Alexander Khoruts, and Dr. Hebert DuPont.

On the first day Dr. Allen-Vercoe creator of RePOOPulate from Canada only had two patients have done RePOOPulate and were saved by it as shown in this story. Canada is also much more friendly in terms in comparison to the FDA in regulating it as well. Actually RePOOPulate II is now being created and she is looking at it to treat IBD, obesity, and regressive autism.

Dr. Lawrence Brandt pointed out that the mortality rate of c difficile is 4% and jumps to 12% with a second infection of c difficile. The longest case he has had of c difficile has been 9 years.  For fecal transplants Dr. Brandt stops antibiotics (Dificid and Vancomycin) 2-3 days before the fecal transplant. Also Brandt reported the cure rate on fecal transplants is about 93% throughout the world.

Dr. Colleen Kelly reported that there are over 400 reported cases of fecal transplants in the literature however probably thousands performed (Dr. Borody as I pointed out here has done over 1,000 fecal transplants). She reports the success rate for fecal transplants after looking at 11 studies was 90%. To date Dr. Kelly has done 101 fecal transplants (ages 19-92). Of all these transplants 9 of them had inflammatory bowel disease. Kelly reports a 95% success rate. One patient that Kelly had who had ulcerative colitis (who I would point out was in his 70s) got a flare up after being off all his medications for 20 years. Kelly also pointed out that the labor required for one fecal transplant is equal to eight regular colonoscopies. On getting an Investigational New Drug (IND) (that the FDA grants) which she says was like learning a new language.  Dr. Kelly’s journey for the IND started in the fall of 2010 and wasn’t granted until 2012. In this process Kelly had to cancel 2 weeks of seeing patients and spend hundreds of hours working 12 hour days just to comply with the FDA to meet the IND.

Dr. David Rubin of University of Chicago mentions the FDA has been easy to work with yet challenging to work with at the same time. Rubin has been trying to study fecal transplants in ulcerative colitis patients.  He proposed it to his IRB (Institutional Review Board) who said they would give conditional approval but he still would need an IND.  Rubin had to submit a 206 page IND. The FDA had some suggestions and a new 168 page review was submitted.

Dr. Sachin Kunde who works on the pediatric side said that Mass General conducted a Phase I trial of looking at fecal transplants in IBD for children which lead to this study. Kunde discussed a study that looked at using enemas in pediatric patients with ulcerative colitis. What is interesting is close to 70% of patients had a clinical response by the end of the first month to the fecal transplant. Kunde in dealing with the FDA recommends hiring a study coordinator. Kunde’s IRB board also told her to stop what she was doing and get a IND from the FDA. She mentioned that the guidelines have been a challenge even after studying them for a year and a half.

Dr. Alexander Khoruts I learned is an advisor to CIPAC which is trying to commercialize full-spectrum microbiota for fecal transplants. Khourts’s first fecal transplant patient was a 61 year old woman who lost 40 pounds and having bowel movements every 15 minutes. Khoruts even had a patient who had c difficile for 12 years (recurrent infections). He also noted that IBD patients got better. In screening donors Khoruts uses a more rigorous approach then even the FDA (testing for more things, asking more questions, and is trying to standardize the donor process). Khourts himself spends an hour and half every day answering e-mails from c difficile patients.

Dr. Herbert DuPont who is prepared to set up a large fecal transplant program in Houston in association with the  University of Texas-Houston talked about the first fecal transplant he performed in 1970 (he is not a GI by training but an infectious disease doctor). DuPont who has been around a while talked about how things were done much more efficiently in the 1970’s when bureaucratic IRB boards were not around. No screening was done in those days. DuPont had a patent with severe antibiotic associated colitis and renal failure after a surgery. Vancomycin was used but didn’t work. DuPont then got immediate approval to do a fecal transplant. He used a blender and it was administered via retention enema to the patient. What is interesting is the donor was a typhoid carrier. However the patient who received the transplant did not get typhoid fever (it is hard to get typhoid fever from the rectum). In his transplant program DuPont wants to freeze the donor stool in order to standardize the procedure.

CEO Lee Jones of Rebiotix which plans to commercialize the fecal matter that can be used by doctors in fecal transplants. Jones has an IND for manufacturing, creating, and delivering the fecal material. The IND ran 1,500 pages. The company plans to have their product out by 2015 (assuming the FDA doesn’t delay this a few years).

Dr. Jay Slater who works for the FDA says the IND is hard yet very doable (too bad he doesn’t have to fill one out).  Doctors have told Slater how painful the experience of IND is and his response is “it’s definitely something that’s hard, it’s hard for a reason, and it’s very doable”. Also for an IND three phases are required just like for regular drugs.

The FDA is being overzealous as usual when it comes to fecal transplants. Fecal transplants have a cure rate of 90% with no serious adverse side effects (thousands have been performed). Filling out endless paperwork, constantly having to send the FDA data, and having meeting after meeting with the FDA doesn't help the patients truly in need of fecal transplants. With the new IND regulation fewer people will be able to get this life saving treatment and may possibly get worse and or die (which is pretty tragic). Dr. Kelly mentioned she spent hundreds of hours just simply trying to comply with the paperwork which is crazy. How on earth is the FDA going to regulate something they are not experts in? Having to go through three phases of clinical trials for fecal transplants will take many years (meanwhile people will be suffering). The doctors that have performed fecal transplants are the most knowledgeable on the whole procedure. A better way would be to have doctors who are performing them share their protocols and methods with each other in order to develop best practices. 

FDA Proves Utter Incompetence in Regulating Fecal Transplants: 2,000 People Will Die As A Result

Recently the FDA issued vague and unclear ruling on regulating fecal transplants. When I first saw this I was quite enraged, upset, however not too surprised. On May 2-3, 2013 the FDA held a conference which was preceded by this letter in Bethesda, MD to talk to doctors who were performing fecal transplants.  The FDA is now requiring an IND and treating fecal transplant as a biologic which has pushed back fecal transplant for nearly everyone by 2 months. In reality it will take 2 months for the FDA just to get around to this subject let alone do anything about it.  If 14,000 people die from c difficile every year that would say that the FDA will contribute to the death of over 2,000 people.

What is quite interesting is that fecal transplants have been performed since 1958 yet I have never heard of any serious adverse affects or deaths. According to a 2011 ACG meeting about 5% of patients who received fecal transplants also contracted an autoimmune disease after they got the fecal transplant. However it is hard to say if it was from the fecal transplant or something else. It is important to remember that people do actually die from c difficile. In fact the New England Journal article from this January showed that 94% of patients were cured of c difficile compared to the only 31% who received vancyomycin. Even the people running the study said it was unethical to not give the patients fecal transplants.

Dr. Mike Edmond might be the only doctor with some sense on this matter. Basically from the way he explains it doctors will have to apply for an IND number (doctors have to submit their protocol). Then maybe 30 days after the FDA gets the information they will let the doctors know if they can proceed. The FDA won’t even let doctors know what they are looking for.

Just how incompetent is the FDA? Here we have a treatment that cures 80%-90% who have a life threatening illnesses with the only existing therapies being antibiotics such as Dificid and Vancyomycin which can costs thousands of dollars while the fecal transplant is much cheaper. Thousands of fecal transplants have been performed as well.  However there never seems to be enough data for the FDA. Perhaps the FDA is incapable of performing a cost/benefit analysis. Actually the most expensive part of the fecal transplant is testing the donor’s stool. However, since the FDA has not approved fecal transplants insurance companies won’t cover it.

The result of the new FDA ruling on fecal transplant will cause more paperwork for doctors, more bureaucracy, more patients having to suffer, and more people dying because the FDA doesn’t seem to think anything is safe enough. 

C.Difficle and Dificid To the Rescue (Humira Working 8 Months and Counting!)

Apologies for not blogging in a while. I have been working and studying for a board exam which has been stressing me out (however not my stomach). What is strange is that while I did a post on c difficile I actually had it and didn’t even know it! The only real changes that have taken place was all the diarrhea I mentioned in my previous post was caused my c difficle. At first I wasn’t too surprised because I didn’t have any pain in my stomach and it felt like I had some type of bug however couldn’t put my finger on it. I took Dificid which was just released in 2011 and seems to have a high cure rate and ran its course on me. Dificid was $115 for 10 days (20 pills). My insurance company actually saved me over $3,000. Of course this is because the FDA allows so few drugs on the market because they require drug companies to spend a decade and hundreds of billions of dollars developing a drug that may or may not get approved. I am all for patients experimenting with drugs that the FDA deems safe and let patients decide if the drugs are effective.

In other news I went to Walgreens to because I thought I was paying a bunch for vitamin D and wanted to get my medicine through Medco because it would be cheaper. At any rate, I asked for my prescription history and actually now have all the medicines I took since I had Crohn’s in 2011. It would be interesting to gather all the prescriptions of Crohn’s patients to see if there are any similarities.

Lately I have also been wondering about diet and Crohn’s. I went to the doctor today to get some blood work (for vitamin D and a booster shot) and was up to 156 which is high for me. Last week I was in training course for 8 hours a day and was eating well at a local diner with grilled cheese, brownie sundae, and an cheddar omelet. Also I haven’t been working out because I have been so busy studying and working. Look forward to working out more.

Humira still seems to be working! I forgot it has now been over 8 months since I started that drug and it has continued to make me well. Every day I seem to worry about the future. I personally would like to see more creative destruction in the Crohn’s research field. A no brainer is to collect data on willing patients with GI Monitor and see if that tells any patterns.  I also have no idea why the FDA bans naltrexone which could be another tool for doctors to use. Fecal transplants I believe could be a game changer for ulcerative colitis. As I mentioned in this post it seemed to put people in remission for over a decade which I would deem a cure. People that suffer with Crohn’s have to take one day at a time wondering what the next day, week, month, even years will be like. We should get people not from just medicine but from engineering, business, and other fields to see if we can improve what we know about Crohn’s in order to take away the pain.

Fecal Transplant Cures C Difficile! Fecal Transplant for Crohn's and Ulcerative Colitis

Recently, this New England Journal article came out showing that 100% of patients who had a fecal transplant for clostridium difficile (c difficile) saw improvement. Usually the treatment for c difficile is taking vancomycin which can cost $55 per ill (ending up costing $2,000 or more over the course of treatment).  This is great news for people with c difficile. Upon doing further research I learned that this procedure now is being experimented on (not formally though for patients with ulcerative colitis and Crohn’s disease). One great piece of literature I found was this meta-analysis which essentially looked at every single study done on fecal transplants for inflammatory bowel disease. Nearly all the studies so far have only been for c diff and not for inflammatory bowel disease.  One conclusion from the meta-analysis was there was a reduction or complete resolution of symptoms in 76% of patients and prolonged remission in 63% of patients.

Dr. Thomas J. Borody seems to be an expert in fecal transplants. He is based in Australia and has done some good research in the area. He did his first transplant according to this article in the 1980’s on a woman who had incurable colitis. It seems after the transplant her colitis never came back. What we need is more experimentation like this in order to get medical advancements. The problem today is the red tape involved with the FDA or federal government that prevents people like us from getting the treatments we need. Borody has performed over 1,500 transplants and currently does 5-6 fecal transplants a week (most are for irritable bowel syndrome which I actually was diagnosed with in February of 2012 in the same year I had Crohn’s). He has even use fecal transplant for non-stomach related issues like acne, multiple sclerosis, and even people with Parkinson’s disease.

In this study of 6 people that Borody conducted with ulcerative colitis all 6 had no signs of ulcerative colitis after 13 years! I looked on Dr. Borody’s website and it seems you can get a fecal transplant for $12,000-$15,000 (in Austrilian dollars which is about the exact same as American dollars) which is just for the transplant and doesn’t include travel or even nursing care. If this could really could help “cure” Crohn’s I really would consider it. However, there are too many unknowns like a) will it work b) how long may it work c) possible side effects and complications. One major problem in the United States is that the FDA has not approved fecal transplants which makes it hard not only to help people but increases the costs of fecal transplants. Insurance companies will not often pay for something that isn’t approved by the FDA or lacks evidence. As more data comes it showing that fecal transplants can help c diff patients that will change. However, we need experimentation with other conditions like Crohn’s, ulcerative colitis, and other autoimmune diseases to really get the ball rolling.

Other doctors in the United States have performed fecal transplants as well. Dr. Alexander Khoruts of University of Minnesota has performed over 130 procedures according to this article. Dr. Colleen Kelly has performed 45 procedures according to this article. Dr. Lawrence Brandt has performed 17 of these procedures (he has been doing it since 1999), while Dr. Christina Surawicz of the University of Washington has performed 16 procedures. Dr. James Versalovic of Baylor College of Medicine in Houston plans to start a intestinal microbiome transplantation program.

Due to the fact that fecal transplants are not approved yet many people are doing it themselves at home. I found and interesting website where a guy who had ulcerative colitis for 12 years did at home fecal transplants and now is in complete remission (he started feeling much better only after 2 days). This sounds good to some people but I personally worry about whether people are doing it correctly, the risks involved, and these people are also not screening the poo that they use to transplant which can present risks.  Right now donors are usually family members or relatives since their medical condition is usually known. The largest cost of the whole procedure is screening the donor which can cost $1,000 according to this article.

This article discusses how the fools at the FDA seem confused since “feces” doesn’t fit into anyone of their main categories. We need many doctors and patients experimenting with fecal transplant in order to spread knowledge of the best way of doing the procedure. Knowledge is power. It looks as if fecal transplants may have a role in ulcerative colitis but time will tell if it can help for Crohn’s. I would also wouldn’t mind seeing a market for feces where people could give it and collect money for it like they do for sperm or blood. Companies could pop up that would screen the feces and could grade it and let people decide which kind they wanted to supply. I have a feeling drug companies might be interest as well because they pay be able to find a mechanism that works similarly like feces to give the same result. Given there are 500,000 cases of c diff every year we will need a lot crap. Also it would be interesting to see if fecal transplants can help other autoimmune diseases like psoriasis, rheumatoid arthritis or other things like acne, Parkinson’s. Every day that goes by is another day a patient suffers. People need to get over the gross factor and we need to start doing things that are proven to work. 

Xelijanz (Tofacitinib) for Crohn's?

In this FDA press release a drug called Xelijanz (tofacitinib) was approved for rheumatoid arthritis (autoimmune disease like Crohns). This trial showed that patients improved when taking the drug twice a day (at 3 mg) over a 6 month period. Here is a great article that includes tofacitinib along with other possible upcoming treatments for Crohns. Pfizer (the drug company who made this drug) has every incentive to test this drug for other autoimmune diseases. What I have noticed is that one drug can treat many different things (Remicade and Humira treat multiple autoimmune diseases).

One a drug is approved by the FDA it can be used for what is known as “off-label” use. The FDA can’t regulate the practice of medicine so in theory a doctor could prescribe tofacitinib for Crohns however he may have to have the patient consent to some things before hand. Crohns patients who are chronically ill need all the help they can get. We want as many possible tools as possible to fight something that is awful, painful, and makes us worry all the time. Allowing the FDA to get out of the way an let informed patients and doctors experiment could work wonders.

Breaking News: Humira Approved for Ulcerative Colitis!

Today, it was announced that Humira can be used for people with ulcerative colitis (inflammatory bowel disease). Although, I do not suffer from colitis but from Crohn’s I am happy now more patients will have access to a drug that has really helped me (no pain since June!). Perhaps the post I did back in August maybe persuaded the FDA! The panel decision of 15-2 made it pretty clear that the drug should be approved. As I said in that post the FDA really has no business telling patients with chronic diseases what drugs they should or should not use. Often these patients have more and better knowledge than bureaucrats.

 Humira is an $8 billlion drug that is already used for things like Crohn’s, psoriatic arthritis, anklosing spondylitis, plaque psoriasis, and juvenile idiopathic arthritis. I hope the drug is approved for more uses (I have a feeling Humira could help other autoimmune diseases not sure which ones though). Humira is pretty simple to use. You can go onto the forums and other blogs that describe how scary it is but it is a piece of cake (especially if you have gone through a horrible disease like Crohn’s or ulcerative colitis). The injection stings a little bit and really just feels like you banged your skin against something hard. I put band aids over where I inject it to be safe and ripping off the band-aid hurts more than the Humira injection! Today makes a day we have improved the world just a little bit more.

Tofacitinib Improves Ulcerative Colitis While FDA Delays

In this most recent WSJ article the drug tofacitbin helped improved symptoms for people who had inflammatory bowel disease. The article is published in the New England Journal of Medicine which can be found here.  The doses ranged from .5 mg to 15 mg and were taken for 2 months with a 194 patient population.  The largest response was seen in patients who took the largest dosage (15 mg). In the group that took 15 mg 78% of patients saw a response (response was also statistically significant) and was much higher than the placebo rate.  The only side effect was an increase in both good and bad cholesterol (LDL and HDL). However, I would imagine this could be controlled with exercise, eating right, and perhaps a statin (Crestor or Lipitor).

The drug is being studied to be used in patients with ulcerative colitis however patients with ulcerative colitis take similar drugs to Crohn’s patients. Pfizer (company that makes tofacitibin) is still waiting for the drug to be approved for rheumatoid arthritis and the FDA was suppose to make a decision by August 21 however pushed that back three months to November as seen here.  What is interesting is that tofacitinib would be the first drug approved for rheumatoid arthritis in over a decade. What is even more interesting is that tofacitinib has one of the largest clinical databases for any rheumatoid arthritis drug ever submitted with over 5,000 patients taking it in 44 different countries yet the FDA still needs more time to analyze the drug.

What is encouraging however is that in May a panel of FDA advisors in an 8-2 decision should be approved (FDA doesn’t have to follow panel advice but usually does).  I wonder what would happen if you had a panel of 10 family members of people who either suffered from both rheumatoid arthritis and ulcerative colitis. I would be willing to bet the rent money that that decision would be 10-0 to approve. The FDA panel has no personal experience with these illnesses nor do they see the daily pain that people suffer as the result of these diseases. While I agree the FDA should be in charge of safety of drugs they should in no way shape or form be in charge of how effective the drugs are. By pushing back the possible approval date patients will suffer and be harmed in the process which hardly anyone ever talks about. Also tofacitnib seems much safer than Remicade, Humira, or other drugs. The FDA acts like a cartel deciding what drugs go on and off the market. As a Crohn’s patient I want as many options as possible. Let me as a Crohn’s patient decide what I ingest into my body. After all I do care more about my body than any bureaucrat! 

Gideon Softer: FDA Killing Crohn’s Patients (And Others)

I came across this op-ed in the Wall-Street Journal recently in a case about how a young man who was diagnosed with Crohn’s was fighting for his life. What is even worse is that the author (young man who has Crohn’s passed away last year). The author Gideon Sofer enrolled in a clinical trial after having half of his intestine removed. Sofer was enrolled in a clinical trial for stem cell therapy made by a company called Osiris Therapeutics. The drug called Prochymal had already shown promising results in Phase II and Sofer was entering the Phase III trial. However, clinical trials are randomized and double blinded so the patient and doctor has no idea what drug they are getting. In a short period Sofer became worse yet had no idea if he was getting the treatment or placebo. He and his doctors tried to get Osiris to get more information but the FDA doesn’t allow that. Even though Prochymal had “fast track” status from the FDA in 2007 the drug is still not on the market meanwhile Crohn’s patients and patients with other illnesses can benefit from the drug.

The problem with the FDA is that no drug is too safe for them. Every drug does contain risks and side effects the question is what tradeoffs are people willing to make. I would be willing to bet that a typical Crohn’s patients has much more knowledge about Crohn’s disease than anyone on the FDA panel that approves the drug. Real people are suffering and the FDA controls what patients can use or not use. I would rather see the FDA first check to see if the drugs were safe and then allow patients to determine if they worked. Patients are not uninformed fools. When people make decisions they often go to a doctor (sometimes even two or three), do research, and talk to loved ones. What is even more interesting is that the FDA has made it harder today for a drug to become approved even though we more information than we have ever had about the human body. Everybody has different genetics, biology, and chemistry within their body and to make a blanket statement about a group of people who suffer from the same thing is ridiculous. Even people with rare diseases often don’t have the same exact symptoms which makes the case for how every individual is different and when the FDA claims a drug isn’t effective they forget that some people have benefitted.

Also have you ever wondered why Remicade and Humira are not cheap? Since the FDA limits what drugs enter the market they have a monopoly on what drug companies can offer. Drug companies invest billions of dollars into each drug they research and develop yet only a very tiny percent get approved. So in essence when you pay for the cost of a blockbuster drug you are also paying for the drugs that didn’t work since the drug company is trying to try to recoup some of their investment. If more drugs were approved it would create more competition which would lower the prices of all drugs and in the long run help patients with their illnesses. Also because drugs are not approved information is suppressed about what drugs work or don’t work. This helps to retard progresses in our understanding in how drugs interact with the body.

The tragic case of Gideon Sofer teaches us that the FDA does more harm than good and it’s power should be limited and given to the millions of patients that suffer from not only Crohn’s but other chronic and deadly diseases. The FDA needs to realize knowledge is power and they do an incredible job of suppressing progress.