Were racing to find a treatment for COVID-19. Why cant we do the same for ALS? | Opinion – NJ.com

By Robert Hebron

About five years ago, my then 27-year-old daughter Beth was featured in a Vice news documentary on ALS, entitled Die Trying. The program centered on patients desperate attempts to accessing promising investigational drugs to treat a disease that was always terminal and offered no effective treatment or cure other than a pill that on average extends life by three months.

In the ensuing five years, nothing has changed.

Now as I watch the lightening-like speed employed in the development and approval of drugs for COVID-19; the scale-up of expanded access for the anti-COVID-19 drug remdesivir, the promise to donate 1.5 million doses of the drug by its maker Gilead Sciences, and the Emergency Use Authorization by the FDA all occurring in a matter of weeks, I wonder why my daughters life, doesnt merit such an all-hands-on-deck response.

In a recent op-ed piece published in the New York Times about COVID-19, a doctor was quoted as saying, Were almost observers in this. We can put patients on oxygen. We can intubate them in the ICU. But were mainly trying to allow the body to heal on its own. Dealing with COVID largely means dealing with a feeling of helplessness. It immediately struck me that the same could be said about a neurologist treating an ALS patient except that ALS is fatal 100% of the time, there are no mild or asymptomatic cases, people dont get better and heal without treatment.

Approximately 30,000 people in the U.S. suffer from ALS. There is one new diagnosis and one death in this population every 90 minutes. 90% of ALS patients die within five years of diagnosis. On diagnosis, typically the doctor recommends that the patient should get their affairs in order and try to do anything on their bucket list they can still do given the current state of their disease. The progressive loss of limb function along with speech deterioration, deterioration in the ability to chew and swallow food, and finally weakening of the chest muscles leading to an inability to breathe, independent of a ventilator, is what my daughter was told to expect; it is what thousands of Americans hear from their physicians each year: there is nothing we can do for you.

The state of access to investigational new drugs for ALS is abysmal. Most trials exclude patients who are more than two years post-diagnosis, which encompasses roughly two-thirds of the entire patient population. Researchers fear that these patients may not survive the length of the trial or will progress so far into the disease that further disease progression is not easily measured. Once a trial is concluded, the patients involved typically lose access to the drug even if it appears to be working for them.

Dr. Janet Woodcock head of the FDAs Center for Drug Evaluation and Research suggests that ALS patients like my daughter can apply to get new experimental drugs under Expanded Access if they dont qualify for clinical trials or live near a clinical trial site. But companies have to play ball and most object because of cost concerns or fear that an adverse event involving a patient on Expanded Access will adversely affect the enrollment in their clinical trials or prospects for approval by the FDA.

These excuses dont stand up to scrutiny. Under Expanded Access regulations, companies can charge the patient and recover the direct cost of producing the drug plus the cost of administering the Expanded Access. And in the past 10 years, across all diseases, an adverse event involving expanded access patients has only caused the termination of one single trial. Healthcare providers and hospitals can also charge for their participation.

In other diseases, like HIV/AIDS and cancer, expanded access is normal and routine. In ALS, weve seen only 25 successful expanded access applications. That means less than 0.1% of patients, who cant otherwise get access to the drugs through clinical trials have obtained access to investigational drugs and treatments for ALS. We are unlikely to see drugs currently in clinical trials approved within the next few years for ALS. Most patients today, without expanded access to new drugs, wont get the chance to see if these agents offer any benefit to them. They are left to die.

The system is working for patients with COVID-19. I want it to work for my daughter.

First, every ALS drug in clinical trials must have in place an expanded access program that runs alongside the studies, at least once the safety of the drug has been demonstrated in phase 1 and activity has been demonstrated in phase 2. Second, open-label access to new agents for trial participants once the study is over, should be required. This is what happens in other diseases, for HIV/AIDS, for cancer, and it should be the same for ALS. Furthermore, expanded access allows companies to see how their drugs work in broader patient populations rather than the study subjects alone, who are generally a narrowly defined population satisfying a set of rigorous inclusion and exclusion criteria for participation.

The status quo is simply unacceptable. When we want to ramp up drug development, access to new medications, we can do it. COVID-19 has shown us all that, as have diseases like cancer and HIV/AIDS. Making decisions to leave ALS patients without access to experimental drugs is criminal. Its not that we cant do it as a nation; we dont want to.

We can no longer let drug companies and the government hide behind their current woeful response to ALS. COVID-19 patients deserve a shot at surviving this pandemic, a chance to survive. So does my daughter. So do all patients with ALS.

Robert Hebron lives in Maplewood with his daughter who was diagnosed with ALS at the age of 25 in 2013. He also serves on the Clinical Trials Committee of IamALS.org.

Our journalism needs your support. Please subscribe today to NJ.com.

The Star-Ledger/NJ.com encourages submissions of opinion. Bookmark NJ.com/Opinion. Follow us on Twitter @NJ_Opinion and on Facebook at NJ.com Opinion. Get the latest news updates right in your inbox. Subscribe to NJ.coms newsletters.

View original post here:
Were racing to find a treatment for COVID-19. Why cant we do the same for ALS? | Opinion - NJ.com