There is nothing worse than watching a dear friend wait for the inevitable; the slow process toward terminality. I have such a friend. A dear friend. Once active and energetic, he is now at the mercy of Amyotrophic Lateral Sclerosis; better known as Lou Gehrig’s disease or ALS. The disease that creeps through one’s body insidiously and painfully. Like thousands of ALS patients, my friend is patiently waiting for reprieve. He and others like him are at the mercy of the FDA.
Until recently, I had never met anyone with ALS or knew much about it. But watching my friend’s silent endurance compelled me to educate myself, and hopefully others. ALS raises its head slowly and often undetected until too late. Twitching, slurring of speech, and weakness in the muscles are often the first signs of the deadly disease. Eventually, all muscle movement, speech, breathing, and food intake becomes difficult, painful, and ultimately fatal. The domino effect of nerve cell deterioration gradually breaks down motor neurons in the body. These are the cells that control voluntary muscle movements like walking and talking. Because motor neuron cells extend from our brain to our spinal cord, when damaged, they stop sending “messages” to our muscles that give movement commands. There is no medical magical formula that determines the rate of deterioration.
A few years ago we all remember the “ice bucket” challenge that was supposed to raise money and awareness for ALS patients. $115 million were raised for alleged research. ALS patients have yet to realize any benefit from the fund raiser. They have been waiting in earnest for the promised research to materialize. Time is not on any ALS patient’s side. Unlike other diseases, ALS can morph into terminal at a blink of an eye. ALS patients do not have the luxury of waiting. The frustration is not with drug or research firms, but with the FDA.
For a long time, drugs and treatments slowed the deterioration down rather than reversing it; until now. A small Israeli firm, Brainstorm Cell Therapeutics has developed and is testing a new drug and process called NurOwn. What is different and giving hope to the millions afflicted by ALS, are NurOwn’s results. Unlike other drugs and treatments that only slowed down or stopped (often just temporarily) the deteriorating process, NurOwn has the capability of complete reversal. The process is in its third phase, which means it has already been tested on humans. If the NurOwn induced ALS process is successful, it can also treat other neurodegenerative disease like Parkinson’s and multiple sclerosis.
NurOwn uses cell stem technology. Bone marrow cells are extracted from the patient, matured, and injected back to rebuild deteriorated brain cell blocks. During maturation, the bone marrow cells are manipulated into behaving like normal brain cells. Once injected back, they circulate in the spinal fluid and back into the brain repairing it. A reverse process to the initial onset of the disease.
The Right to Try Act was passed in 2018, allegedly giving terminally ill patients access to experimental treatments. ALS patients have been asking the FDA to fast track the Brainstorm treatment the same way they had done with AIDS. Phase three of testing is in progress and awaiting conclusion if it weren’t for COVID. We have stopped research and treatments for terminal diseases in lieu of finding a vaccine for a virus which people are recovering from. Despite the number of COVID cases reported, the majority of them recover. You don’t hear that on cable news networks. But like AIDS in the 80’s, COVID is now an election campaign issue elevated to unprecedented political heights at the expense of terminal diseases.
We are into the 11th year of NurOwn’s research, but the FDA drags its feet holding bio-pharma companies responsible to outdated strict regulations while at the same time asking them to foot the bill. It takes approximately 12 years and billions of dollars between the creation of a drug in the lab and approval by the FDA. The FDA requires continual testing; often on terminal ill patients waiting for a cure. Some patients fight the US Government for the right to be tested. But in many cases and unbeknownst to them, they are given placebos. Small companies like Brainstorm cannot afford to make the drug available to the general public and insurers unless the FDA approves it. Right now, NurOwn’s one-time treatment is a five figure amount. The treatment needs to be repeated. The average life expectancy of an ALS patient is three to four years. Do the math.
When a government organization like the FDA plays politics, people die. When the FDA picks and chooses who has life priority, it becomes irrelevant. Why should approval of a much needed drug or treatment take so long, especially when it has already been tested? The FDA claims that there are already drugs dealing with ALS. Unfortunately for ALS patients, their disease does not have biomarkers. Cancer research looks for a tumor, a measurable end point which gives researchers a reference toward a cure. ALS does not have a rating scale. One either gets better or not. No two individuals can be measured equally across the board. That is why NurOwn is different. It is individualistic through the patient’s own stem cell recovery. It is unique to each patient.
Some politicians did step up to the plate and written to FDA commissioner asking for parallel research track to be given to ALS as it was given to the AIDS epidemic in the 80’s. Senators Ted Cruz, Marco Rubio, Mike Braun, and Mike Lee are putting pressure on the FDA to give ALS patients access to NurOwn research under the Right to Try Act. In the meantime, my friend is among the 5,000 who are annually diagnosed with ALS. He is also one of the many waiting for the FDA to get its finger out and help him. According to the FDA, the reason that ALS testing takes so long is because the number of people afflicted with the disease is not large enough to get a good sample. How encouraging. Tell that to the 20,000 or so Americans known to have the disease, the 5,000 more who will be afflicted by it, and the thousands that have already died.
Like any degenerative disease, the pain and suffering does not belong only to the patient, but also to his family. Spouses, children, and friends stand by and watch with agony as their loved ones recede into another world of immobility. Their lives stand still between doing all they can to ease discomfort and pain, and a distant hope of recovery through a new drug discovery. In this case, there is a new drug discovery that could bring relief to my friend, his family, and the thousands waiting patiently in immobilizing pain.
There are the Naysayers who want more tests and research into NurOwn, but they are not afflicted by the disease. As one spouse of an ALS patient so aptly put it:” Quite frankly, when I hear people express that is might be risky or dangerous, an ALS patient such as my wife could care less! How many ALS patients will die in the next year waiting for a possible effective treatment?”
The FDA has three objectives; reputation, lobbying, and politics. It protects its reputation first, then aligns itself to profitable lobbyists, and finally turns its attention to the politics of the moment. COVID is the politics of the current moment, just as AIDS was in the 80’s. Media coverage does not help either. The media sways toward the political agenda of their choice. The FDA follows suit.
The FDA weighs reputation, politics, and lobbying against its decision to approve a drug. Its reputation as guardian of public health, has done very little for ALS patients. It’s politics have done even less. The FDA’s strong bureaucratic autonomy operates at the expense of terminal patients and drug availability that can save lives. NurOwn is caught in this vicious bureaucratic triangle at the expense of my friend. Wrong does not start to describe it.
“The FDA weighs reputation, politics, and lobbying against its decision to approve a drug.”
This makes sense, but just because COVID is “hot” right now, how/why does that prevent the FDA from taking action on ALS? I mean… I understand why it would follow the politics of the moment, as you write, but what does it stand to lose by taking action on medications for other diseases? I don’t quite understand.
Ben, they defer approval because they don’t want to go against the grain. In my research I found out some interesting weighing in that they have done in the past. The arthritis foundation has a strong lobby even though asthma is several times more serious and fatal than arthritis but it does not get the publicity that arthritis does, that includes the media. They deferred approval on asthma treatment and drugs in favor of arthritis. They did the same during the AIDS epidemic. I have it on good authority from Brainstorm, that they are only waiting for FDA approval to move ahead with NurOwn, but COVID has put it on the back burner. Phase III has been completed, yet FDA approval is still not forthwith. In the meantime, my friend is dying a slow death. Thank you for your feedback.
so “against the grain” is approving a treatment for an illness without a strong lobby? do I understand that correctly?
The ability to correct the behavior of any institution is by the controlling element that allows that organization to operate. Who, specifically, controls the operating charter of the FDA, to have the effect to bring forth change. Who is that person; as there is always the one that if addressed, can remove the block. If left unchallenged, the block remains. Your article makes me want to do more.