We hope that after reading our blog, you have become more informed on the history and overall process of experimental/clinical trials. There are many current issues such as the Abigail Alliance and the H1N1 experimental drug that is currently being tested. These issues have helped to gain awareness and allow the public to see the practical applications of experimental drug trials. It has lead to potential candidates to research experimental drug trials a bit more before they decide to become a participant. It is a sad that only 5% of eligible candidates actually participate in drug trials.

We hope that after reading this blog you will become a better informed consumer or a better advocate for your patient that may be a possible candidate for a drug trial. The more people who participate in drug trials, the better the chances future patients have of finding a suitable treatment for their ailment.

Also, thank you to our readers for posting such great comments!!!

When I first heard about the swine flu I wrote it off as a fad disease like Mad Cow or Bird flu, but it is clear now that this is real and it is really affecting the general population. A coworker of mine is pregnant and she is going as far as to wearing a mask when working.

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Currently, there is a big debate whether or not patients with terminal illnesses should or should not have access to experimental drugs. However, there are several possible considerations to keep in mind before allowing people access unproven drugs.

One of the problems with allowing such wide access to not yet approved experimental drugs is that it makes the process of clinical trials much more difficult. Currently, drugs must undergo three phases of clinical testing to address safety & efficacy. According to Dr. Susan Okie, making it a “right” for people to have access to experimental drugs, would “fundamentally challenge the government’s system for evaluating drugs.” Furthermore, why bother participating in the clinical trial, when you can get the treatment directly?

In addition, according to the Society for Clinical Trials, legislation to open access to experimental drugs would severely “undermine the system of scientifically valid testing of new drugs that has been a bulwark of health care for several decades.”

When I first started thinking about experimental drug therapies my initial thoughts went to patients with life-threatening diseases. A family friend of mine has been diagnosed with Multiple Sclerosis (MS). Briefly speaking, MS is a chronic neurological disease that affects the central nervous system. The CNS is made up of brain, spinal cord and optic nerves, and the disease causes symptoms that vary from person to person. My friend has had the disease for several years and is now so debilitated that he is now on disability. His main symptoms include things like severe migraines and dizziness. Some days he feels very active and others he can’t even get out of bed. He is taking a drug called Tysabri that had a very short clinical trial time and has been removed from the market once already.

Tysabri works as a monoclonal antibody and it has been made to attach to lymphocytes (white blood cells) and then prevent them from entering the brain. Typically with MS the lymphocytes cause damage to the brain and thus creates the symptoms of MS. The problem with this drug is that is has had several cases where it developes a degenerative brain infection called progressive multifocal leukoencephalopathy (PML) and it is often fatal. This is why it was taken off the market the first time.

When talking to my friend about this, he told me that having good quality of life, even for a short period of time was more important to him than having a pro-longed miserable life. That is why the risk is worth it for him. He also said that when he began the drug his chances for getting the brain infection was 1:24,000 and now after 39 infusions (one infusion every month), not only does he have the record for greatest amount of infusions, but also his chances are now 1: 1,000.

Obviously, the FDA had problems with the drug the first round, so why was it allowed to be put back on the market? The FDA’s idea is that the consumer should decide if the risks outweigh the benefits and whether or not they want to be involved with it. The problem with this thinking is that the whole purpose of the FDA is to make sure that it is safe for the general population. That is why we have experimental drug trails and why the FDA exists at all. If a drug isn’t safe it shouldn’t be let out of trials…right? For my friend, his actions say no. He wants to try anything. I guess it is up to the rest of us to decide for ourselves.

By Stacie Guinn

Additional Resources:

National MS Society

FDA - Tysabri

Sources:

Edelson, E., (2005). FDA Approves Tysabri for Multiple Sclerosis Treatment. Retrieved from http://www.medicalnewstoday.com/articles/16942.php

Medical News Today. (2004). With Tysabri decision, the FDA decalares no drug is too dangerous to be FDA approved. Retrieved from http://www.naturalnews.com/019331.html

National Multiple Sclerosis Society. (2009). What is Multiple Sclerosis?. Retrieved from

http://www.nationalmssociety.org/about-multiple-sclerosis/what-is-ms/index.aspx

According to recent studies, about 120 million people will be affected by Alzheimer's by 2050 worldwide. This seems like a striking number of people who will not have short term memory.

Recent studies have yielded a potentially promising result in the use of a particular cancer drug. This particular drug is from compounds called HDAC inhibitors which allows the DNA to allow neurons to make new proteins. (Dotinga). This drug could potentially stop the progression of Alzheimer's Disease as well as reverse the effects.

This information has been proven in labs among mice but clinical trials aren't expected to commence for another 3-4 years. If the number of Alzheimer patients is steadily increasing then it would seem more prudent to begin clinical trials as quickly as possible. Now that researchers have a better understanding of how Alzheimer's Disease works the potential success of this treatment is very promising. The effects of Alzheimer's Disease is debilitating and is increasing the need for individual care for these patients. If the clinical trials are successful then the number of Alzheimer's patients could drastically be reduced and hopefully in the future the disease can be completely treatable.

It is very sad to see someone with Alzheimer's Disease because the effects of the disease affect those around them as well. Family members are nothing more than strangers to patients with Alzheimer's Disease and it is a very emotional time. It is hard to see someone live the rest of their life without being able to remember where they are or what is going on. It is a sad way to live. Hopefully these clinical trials begin very soon so that people will not have to be affected by this disease.

It is hard to make any judgments about this subject without thinking about the people who have and are participating in the investigation drug programs. With this in mind I thought it would be good to interview someone who had participated in a drug program and get a first hand look at this subject. I interviewed Sue Guinn who had been involved with several drug programs to treat her rheumatoid arthritis.

Stacie: I understand that you were a participant of an investigational drug program.
Sue: That is right. Right after the birth of my second child my arthritis really flared up. It seemed that all my joints were hurting and it was kinda scary. I had been treated with “Gold injection” before and had terrible reactions to it. It is kind funny because that was a drug that WAS approved by the FDA and yet the side effect from that drug is more severe than any I have ever experience. I was somewhat afraid even to try any other medication because of that experience, but I was going to a doctor that I trusted and who had been working with me to find something that would give me relief without causing me such pain as the Gold injections had.

This doctor approached me about going on a study for oral medication that was being investigated. The preliminary studies had been promising and I was a good candidate for this study.

Stacie: What do mean when you say you were a good candidate?

Sue: Well, I was young, OK relatively young. I was 32. I didn’t have any other medical problems. I was not obese. I was a nonsmoker, nondrinker and my symptoms were not so severe as to skew the study and I had the time to come into the office for thefollow-up studies.

Stacie: What do you mean by follow-up studies?

Sue: You know, blood work, EKG’s, chest x-rays… that kinda stuff.

Stacie: Tell me about the study.
Sue: OK. It was a double blind study, which means, that neither the doctor nor patient knew whether it was actual medication or a placebo.

Stacie: Could you take other medication while you were on investigation drugs?
Sue: I couldn’t be on any medication for my arthritis. I had to keep a record of anything I took.

Stacie: Why did you decide to participate?

Sue: I thought if they could find safer medication that would help other people with arthritis it would be worth the inconvenience. I didn’t get paid to participate. The only things I got out of it were free lab work, x-rays and EKG’s. I also would be allowed to stay on the medication, if it helped, while it was being approved.

Stacie: So how did it go?
Sue: Well, at first it went really well. I could tell right away that I was on “real medication”. I felt better. My symptoms started improving about the second week. By the forth or fifth week I was pretty much symptom free. All my labs were coming back good and I was looking forward to staying on the medication.

Stacie: So what happened?
Sue: I had been on the medication for several months and I was working in my yard. I woke-up the next day and I had a rash all over my face, hands, arms and some on my legs. I called the doctor and told him that I thought it was poison ivy. He asked my to come in. He examined me and said he couldn’t be sure but I would have to be taken off the study. I was really upset because I was doing so well and hated to start on any other medication. I had no choice. He started me on Methotrexate, which is a medication that lowers your immune system and in fact is used to treat cancer in higher doses. I did well on it but had more side effects from it than I had experienced when on the investigational drug.

Stacie: Was the investigational drug you had been on ever approved?

Sue: No it never was. While it was in the final stages of approval they found that it caused a life threatening blood disorder. I kinda looked at it as a “God thing” that I got into the poison ivy.

Stacie: Would you ever do another study again?

Sue: Yes. I still believe it is worthwhile to find treatments for illness. If I could help find a drug that might treat an illness that you or your children might have. It would definitely be worth it.

Stacie: Is there anything else you would like to say about investigational drug programs or the way that the FDA goes about approving drugs?

Sue: Yes. I think the FDA takes too long to approve some drugs, especially the ones that treat catastrophic illnesses. If people would be willing to go through the risks of taking the medication and it improved their quality of life while they are alive I think they should be allowed to take them even if it might shorten their lives. That should be their decision.

The Abigail Alliance’s mission is to expand the availability of experimental drug treatments for “patients that have run out of conventional treatment options.” The Abigail Alliance was founded in 2001, after the death of Abigail Burroughs who died of cancer at the age of 21. An experimental drug named Erbitux showed promising results, but unfortunately she was ineligible to participate in the clinical trial.

According to the Abigail Alliance:

- many people die while waiting for experimental drug treatments to receive approval.

- the FDA imposes many restrictive participant requirements for drug clinical trials, leaving out the most ill patients.

- many clinical studies involve the use of placebos to treat life-threatening diseases.

What the Abigail Alliances seeks to do:

- allow seriously ill patients to receive experimental drug therapies, if they have run out of all other options.

- ban the use of placebos in clinical studies.

- give patients and doctors more control over their treatment options and eliminate policies that place decisions on the FDA.

- make it more affordable for smaller biotech companies to conduct clinical studies.

The fight for earlier access to effective, new treatments is a slow but steady one. The FDA is very resistant to change, but the Abigail Alliance has had many accomplishments. Some of these include introducing the ACCESS Act (Access, Compassion, Care, and Ethics for Seriously Ill Patients) to Congress and expanding certain drugs to many seriously ill patients. Some of these drugs include Abraxane (breast & prostate cancer), Alimta (lung cancer), Avastin and many others. Despite these many accomplishments, however, their work is far from over.

By Max Flores

References:
Kovach, S. (2007). The Abigail Alliance: Motivated by the tragic circumstances, families battle an uncaring bureaucracy. Life Extension. 25-30.

Links:

http://abigail-alliance.org/ - The Abigail Alliance for Better Access to Developmental Drugs

http://www.clinicaltrials.gov/ - Registry of Federal and private clinical trials

How a Clinical Trial Works

The process of getting a drug on the market is an extensive process. Before clinical trials, the new treatment has to have laboratory findings (first in cells then animals) and then it has to be approved by the Health Ethics Committee. If the study is approved it will go into three phases of clinical trials:

Phase I (20 - 80 patients) Tests for safety using healthy individuals, and can lasts about two years.

Phase II (100 - 300 patients) Tests safety, dosing, and efficacy. This is administered to a target population.

Phase III (1,000 - 3,000 patients) Tests safety, efficacy, and side effects.

Typically there are multiple trials for each drug. In the 1980s a drug had an average of 30 clinical trials and involved about 1500 patients. By the 1990s these numbers more than doubled to an average of 60 trials per drug and involving nearly 5,000 patients.

After the phase III the drug manufacturer files a New Drug Application (NDA) and the FDA reviews the trials case. This process takes about two years.

Types of Trials

Diagnostic Trials: Used to find better testing procedures for a diagnosing disease.

Prevention Trials: Finds ways to prevent disease in healthy individuals or prevent the disease from returning. This includes vaccines, medicines, vitamins, minerals, or changes in lifestyle.

Quality of Life Trials: Look for ways to help improve the quality and comfort of life inpeople with chronic illnesses.

Screening Trials: Test the most effective way for detecting a certain disease.

Treatment Trials: Test experimental treatments, new approaches to surgery or radiation, or new drug combination therapies.

That’s all great, but why should I care?

Every drug that is on the market has to go through these procedures. It is a process that averages about nine years from start to finish. A lot of people don’t have that kind of time to wait for drugs to enter the market. It is also important because if a drug therapy isn’t ethical and somehow passes FDA regulations, then it is you the consumer that suffers the consequences.

It is sometimes easy to think that drugs that have not been approved by the FDA are better. Individuals who may participate in an experimental drug therapy need to be aware that there are a lot of risks, such as: (1) Suffering from adverse long-term or short-term side effects that could possibly be life-threatening, (2) the drug therapy may not be effective, (3) patient may be taking the placebo and not be receiving any medication, (4) it may require a lot more time and energy than it would to be taking a drug that is already on the market. Before going into a study make sure that you have enough information going into it.

References:

Photo: 2007- CNN Money

Daniel B. Klein, Alexander Tabarrok 2009 fdareview.org

U.S. National Institutes of Health 2007 clinicaltrials.gov

Experimental drug therapy is something that has been apart of life, really ever since the beginning of time. Every culture has their history of medicine. For the Egyptians their extensive knowledge about the human anatomy from their mummification treatments gave them a great understanding of the body. They had a range of remedies, from using thyme as a pain reliever to using camphor for epilepsy. In Greece they used the stars for guides to how individual people will respond to different treatments. Today, at least in the US, the history and regulation of experimental drug therapy lies closely with that of the FDA’s (Food and Drug Administration) history.

The FDA was created as a way to protect consumers from the potential dangers in drugs and food. The FDA can trace its roots through Charles M. Wetherill of the Department of Agriculture who did studies on food, soils, and fertilizers. FDA got its start under the 1906 Pure Foods and Drug Act which prohibited interstate commerce in contaminated and misbranded food and drugs. In the 1930s FDA was given its current name and really started enforcing the regulation of food and drugs. In 1938, President Roosevelt signed the Federal Food, Drugs, and Cosmetic Act. This act gave new meaning for safe consumer goods and made it so that drug manufacturers had to provide scientific proof that their drugs and products were safe before they were allowed on the market.

President Kennedy signed the Drug Amendments of 1962, which stated that drug effectiveness had to be established prior to marketing or else it would not be truly safe. This required drug firms to send the adverse affects reports to the FDA. Also, any advertising in medical journals had to provide the doctor with all the possible risks and benefits.

The laws that were made in the past have shaped where we are today. These laws are beneficial to consumers, because they can have more assurance about taking a drug. But there are also problems with the laws. They often require so much time for an experimental drug to be tested that a lot of people are hurt because they have no way of accessing it.

By Stacie Guinn

Sources:

Fowler, R. (2007) Egytian Medical Treatments. Retrieved from http://egyptian-history.suite101.com/article.cfm/egyptian_medical_treatments

Jansen, W. (1981). The Story Behind the Labels. Retrieved from http://www.fda.gov/AboutFDA/WhatWeDo/History/Overviews/ucm056044.htm

This is an important issue to consumers because every drug that is created and approved by the FDA has to go through experimental drug testing. The integrity of the drug testing directly effects the public. This is an important issue to health care providers because it is important to know the data behind the drugs we administer and their possible side effects.

Experimental drug therapies can provide some hope to some who are ill with terminal illnesses, but it faces many problems:

1. Many may try to exploit the ill, who are already vulnerable, with "promises" of a cure.
2. Some may try and "buy" their spot in clinical trials.
3. Safety/efficacy may not be well known.