One ALS Patient's Fight For The 'Right To Try'

This is KPBS Midday Edition and I am Tom fudge. If you are diagnosed with a terminal illness, would you want to try and unproven drug which just might help you. If the alternative is certain death, most everybody would say yes. By current law in California, actually says no. A bill introduced in the California legislature would change that, making it possible for doctors to prescribe experimental drugs to their terminal patients. Of the drug company would then choose whether to make the drug available. Several states have passed these so-called right to try lives, but some in the medical community are concerned about right to try might have a negative effect on drug testing. We are going to begin the conversation about right to try with a recorded interview. Maureen Cavanaugh, the host of the day, spoke with David Huntley. Is a former San Diego State professor and a triathlete and he was diagnosed with ALS, also called the Lou Gehrig's disease, in 2013. He wants the right to take a drug called GM604 which is aimed at fighting the progression of the disease. The company called general that makes the drug, which is not yet been approved by the FDA. Here is that interview. Dave, why do want the chance to take GM604? I have had ALS for 2 1/2 years, and based on my progression, will pass in the next 6 to 12 months. There are no drugs in the pipeline for approval in that window and most of the drugs under investigation at best create the symptom and not the disease. The early clinical trial of GM604 resulted in a significant slowing or even cessation of progression in 7 of 8 treated patients. When GM604 was given to another patient outside the clinical trial, he saw modest but measurable improvements and function. If Genevieve is forced to go through a phase 3 trial before approval, it will take three years and I, together with 15,000 ALS patients in the US and 300,000 worldwide, will die. This drug has not passed clinical trials. Doesn't concern you that it could be harmful to you? GM604 has been given to 32 healthy volunteers and non-ALS patient, 26 stroke victims and four Parkinson's disease patients with no adverse events. The safety has been demonstrated. But even if there was not such a strong body of evidence for safety, I would want all ALS patients to have access to GM604 because the disease is 100% fatal. And the tolerance of potential risk versus reward should be a decision left to the patient and consultation with his or her doctor. What do you think GM604 could do for you? I believe that that GM604 has the potential to stop my progression and perhaps restore some of the functionality I have lost. Dave, what is your condition now? For getting ALS, I was a university professor and a distinguished lecturer for the groundwater resources Association of California. In the last year, what happened entirely lost the ability to speak, four years ago I completed both -- the newly -- New Zealand and Boston Marathon and now I'm restricted to a wheelchair, unable to run, walk, or even stand. I cannot swallow more than thin liquids. So I take my nutrition through a feeding tube into my stomach. I have lost the use of my lips and most of the use of my arms. So I can no longer hug or kiss my wife daughter her grandson. And finally, how is it affecting you, knowing that there is a drug out there that may help you, but you are not allowed to take it? It tears me up. Largely, because of my family. Can you imagine that the impact on them if two years after I die, results of phase 3 testing confirm the early results that GM604 would have saved my life? It also frustrates me because I cannot see the logic of denying access to a drug to a population afflicted with a rapidly progressing disease that is 100% fatal. David Huntley. Thank you. Thank you for inviting me. David Huntley, again, suffers from ALS. Joining me now the studio is Linda Clark. She is David Huntley's wife. And advocate of Ray to try legislation. Linda, thank you for coming in. Thank you for having me. ALS is a horrible disease and it sounds like your husband has managed to communicate and as he typed messages which are read by computer? Yes. Is only method of communication nowadays is an iPad and he is typing slowly, a few words per minute so it is increasingly difficult. What has it been like for you and for David's daughter to see him in this condition? It has been devastating. When David contracted this illness, he was in the healthiest condition of his life. He was competing actively in triathlons. We were both involved in skiing and sailing and swimming and cycling adventures. All of that has been taken away. At the worst, I think, has been the loss of communication. When he is lying in bed, he cannot tell me how to alleviate his discomfort. There is no way to communicate what the problem is. I assume that you have been told about ALS, what the rest of us have, that it is a deadly disease, an incurable disease. Absolutely. And there is no treatment and there has not been in 75 years. Now, how did you hear about the drug GM604? My husband, David, the consummate researcher, is always looking at clinical trials around the world for this disease. He actually participated in one clinical trial. So it is on his radar when this drug GM604 released results of their phase 2 study, back in October when this drug GM604 released results of their phase 2 study, back in October 2014, we immediately contacted the manufacturer which is a small biotech in Pasadena and asked what the next steps were and how we could possibly gain access to this drug. What did your doctor tell you about David's chance of being able to try the drug? Our doctors said that he would support us if we could gain access through compassionate use. However, that is a very lengthy process. We do not know that the manufacturer will make it available. So that is one alternative, I guess, called compassionate use. When it comes to experiment with drugs but you said is very difficult. It takes a very long period of time and this company did it once already and we are in contact with a patient that received the drug. His name is Eric Ballard and he personally believes that he benefited from taking the drug and he is with us on this campaign to get the FDA to grant accelerated approval, which is the fastest way to make it available to patients. If your husband were able to take this experimental drug, I guess, what would be your hopes at that point? This is a disease modifying drug. It is a multi-targeted drug, unlike a lot of other ALS drugs, it is being shown to slow or even stop the progression of ALS. That is monumental in the field of ALS research. No other drug has been able to do that. So in other words, David probably would not get better but he would not get worse? It would buy him time and hopefully that time will allow this drug to be further refined or other drugs to become available in hopes of neurogenesis in which you regain some function. Well, you are listening to midday edition and I am Tom Fudge speaking with Linda Clark who is an advocate of right to try legislation and once again right to try refers to the ability of people who are terminally ill to try experiment with drugs that have not been approved by the FDA and Linda, why do you feel so strongly that this legislation is something that we should accept? The legislation is really the voice of the American people saying that we reject this idea that the FDA continues to withhold drugs that show promise of efficacy, especially in the case of terminally ill patients. If a drug shows that it is safe, and it has real potential for improving a patient's quality of life, extending their life, or possibly even saving their life, it makes no sense, it seems immoral and unethical to us to withhold that drug. Once again, GM604, the drug that we are talking about is now in phase 2 trials? There was a meeting between the manufacturer and FDA at the conclusion of the phase 2 trial in which a -- at which time, the manufacturer apply for the accelerated approval program. The FDA could for gay -- forgo phase 3 and make it available and to set conditions. They have a lot of latitude to decide how it should be distributed and what the post marketing surveillance requirements are. So that we gain valuable information while these patients are trying this drug. We are going to talk a little bit more about the legislation. In fact, midday edition producer Megan Burke spoke with California assemblyman, Ian Calderon, who introduced the right to try bill in the legislature and he explains why he thinks terminally ill patient should have access to experiment with drugs. You know, I think what is difficult as legislators is that there is your political hat and your public service had but then there's also, you know, your human hat. You know? I am an individual and people think that because I'm a legislator that the decisions I make, I am separate from all of those decisions when in fact, I suffer the same consequences from the decisions I make as anybody else and if it came to one of my family members or even myself, I feel like I should have the right to live in a state that allows me to -- my greatest, my greatest time of need, to swing to the fences and try and save my own life and I think that that is what the root of this legislation as. Who would be eligible to take experimental medication? Should the right to try bill pass? What does basically, what this bill does is it does not require anybody to do anything. It just affords people the right to try and experimental drug without the state coming after you, whether you are the patient, drug manufacturer, health plan or a doctor. So those that are going to be eligible for this bill, are those that are determined to be terminally ill by the doctor and that is something that is between the doctor and the patient, not something that we are defining. But essentially, somebody who has their doctor tell them, you have an illness and this illness is going to result in your death. That is who is going to qualify for this bill. Why is it the current FDA process good enough for terminally ill patients? You know, the compassionate use a process, I think, that is great and I think that it is better to have that then nothing. But it also takes time. To be approved for the program. If you go to the doctor and the doctor says, I'm sorry but you have six months to live, three of this month you're waiting for the FDA to approve you for compassionate use program, that is your life. And you know, I think that the moment that the doctor tells you have a terminal illness and your illness is going to result in your death, you should have the right right then and there to try and experimental drug to try and save your life and it is not like you can try it straight up. You have to exhaust all other options before you can use this option. So what other legal drugs are out there? You have to exhaust that. But also the same time, it does not preclude you from trying these experimental drugs. I mean, it is a last resort, but at this point in time, right now, it is not something that the state allows you to do. What is your response to people you say, if right to try bills are not near enough that they could potentially undermine the clinical trial process? I do not think that they are wrong. I think that the point of the bill is -- should be brought and we're not requiring drug manufacturers to offer their drugs to any patients. That is a choice that they are going to have to make on their own and we are nothing that they have to but we are just allowed people that live in the state as well as doctors to prescribe it and allow this patients to use it. And if the manufacturer does not want to do it, that is their choice. That is not really what we are after in this bill. That is California some women Ian Calderon who introduced the right to try bill in the legislature which would allow terminal -- terminally ill patients to use experimental drugs and joining me, in the city, Linda Clark, her husband is a suffer from ALS and join me also now is Doctor Gary Fierstein who is the director of the clinical and translational research Institute at UC San Diego. Gary, thank you very much for coming in. Thank you. It is my pleasure. Doctor Fierstein, I think most people would look at David Huntley situation where he has ALS, and say, why not? Why not let him try to stick -- experimental drug? But I think that you have concerns about right to try. Why don't you tell us what they are? Well, it really does represent a larger societal question. Of the rights of the individual compared to the needs of the population at large of society. It is very difficult to argue at the extremes with a clearly terminal disease. That there should not be some opportunities such as this. But on the other hand, I think that there is very little question that this will have an impact on the ability to run and recruit patients into formal clinical trials. Could go into that a little bit further. Why do feel that it would compromise that process? The way the clinical trials generally operate is that there is a test to drug or medicine and then there are typically two groups of patients, so what patient receives the test drug -- it whether it is unknown or does whether it works or not and the other receives the current standard of care that is available or in some cases a placebo, depending upon the disease. If there is an alternative mechanism to bypass that, then it has the potential for undermining the ability to recruit patients into clinical trials. Because most people actually want to receive the test article. The thing to remember, though, is that the vast majority of agents are actually ineffective is a student studies -- phase 2 studies or even in phase 3 you can demonstrate -- the initial efficacy is actually not seen and the side effects typically are not necessarily seen in six patients or 12 patients but requires sometimes hundreds or even thousands of patients in order to really get a full appreciation. So, in essence, if you provide this bypass pathway, it doesn't potential downstream effects in terms of more traditional clinical trials. So, are you saying that if this drugs available to all people who are terminally ill, you might not be able to get enough people to volunteer for these trials? Well, one of the things that we want to be certain of is that if we given it experiment agent to people, that we learn something and I think what you have already heard is that one of the proposals would be to collect information on anybody that receives the agents. But still, the quality of information, really be ability for most diseases to be able to make an accurate risk-benefit assessment on a drug that should be widely distributed across the population does typically require larger scale studies. Well, Linda Clark, I am sure that you have heard some of these arguments opposed to write to try. What is your response to them? Well, I think it right to drive is mostly important because it sends a message that we have to look at a new way of improving drugs and accelerating that approval. A right to try can be improved by requiring post marketing surveillance, where you collect data, so that we advance the science, but also for protection. Consumer protection. We want to know what is working and if it has safety concerns but with respect to very rapidly progressing fatal illness, with no treatment, we think it deserves a protocol of its own and not the 50-year-old standard, which is a double blinded randomized control trial. Which takes 10 years to 15 years, millions of dollars, that absolutely makes no sense when you have people that will die within a few months to a few years. Doctor Fierstein, I have been told that you are really not in a position to say whether you are for or against the right to try, but four people who are -- for people who are terminally ill, are there other options? Is there way to make the compassionate use exception broader? Well, I think that that would be a very reasonable approach. As I mentioned earlier, some of the right to try types of mechanisms would make a lot of sense from the perspective of the individual rights. I think, not only would having compassionate use abroad it but also potentially coming up with more creative trial designs, that allow the movement away from traditional placebo-controlled long-term studies is important. I think the other thing to keep in mind is that, again, at the extremes, it seems pretty clear. But doctors do not actually have a crystal ball that allows them to say, you have a fatal disease. In most cases. In most cases. In most cases. You know, what I worry about sometimes is what the actual criteria would be for deciding when hopelessness begins. In some cases, it is very clear. In other cases, I think, and perhaps the vast majority of cases, I think the line is not as clear-cut as most people would like to believe. Linda Clark, how many states -- I'm afraid that I do not know how many states have a right to try laws? But I believe there is about five with maybe 26 other states that are currently proposing legislation. That is just a guess. Okay. So this is something that is being considered and lots of states in the United States. It is definitely gaining momentum. Linda, what is next for you and David? David, your husband, again who suffers from ALS? We are going to continue proposing accelerated approval. We are working nationally with other people. We have a quarter of 1 million signatures on a petition to the FDA. To grant this drug -- GM604 sits squarely within their own definition of what should be granted accelerated approval. A past safety and efficacy, demonstrated efficacy in a phased to trial. So we are going to continue until we get approval. Well, Linda Clark is an advocate for right to try legislation which allows a terminally ill patients to use experimental drugs with the approval of the drug company. Linda, thank you very much for coming up. Thank you for having us. Once again, Linda's husband David suffers from ALS and my other guest has been doctored Gary Fierstein was director of the clinical and translational research Institute at UCSD. And Gary, thank you very much.