CLINICAL TRIALS: THE NEED FOR SPEED
Everyone in the ALS community wishes that clinical trials could be quicker to allow pALS to receive treatments. The biggest obstacles to this goal are the FDA requirements that are necessary to ensure medical treatments are safe and effective for people to use. What is being done to speed up getting the treatments to pALS?
Discovering biomarkers can speed up diagnosis, improve screening for clinical trials, and target in clinical trials those with the biomarker/specific version of ALS. In trials of the general ALS public, often results are not positive enough to continue to the next trial phase possibly because some/most of the people in the trial have a version of the disease that is different than that being targeted. Using a targeted subset means the results of trials will be more accurate and will allow more!
RESEARCH
NOTE: CURE ALS has funded centers that have been supporting or working on the TDP43 protein!!!
RESEARCH FOCUSING ON “LONG TIMERS” (survival longer than the typical 2-5 years)
Everyone in the ALS community wishes that clinical trials could be quicker to allow pALS to receive treatments. The biggest obstacles to this goal are the FDA requirements that are necessary to ensure medical treatments are safe and effective for people to use. What is being done to speed up getting the treatments to pALS?
- Expanded access, sometimes called "compassionate use," makes a new, unapproved drug available outside of clinical trials to treat patients with serious or immediately life-threatening diseases or conditions when there are no comparable or satisfactory alternative treatment options and/or for people who don’t qualify for trials. This access also helps researchers further assess the drug.
- The use of “platform” trials to test multiple potential treatments in one trial. The benefits of a platform trial are that there is only one placebo group for the whole trial (instead of one for each treatment), one effort for recruiting, and one effort for the trial process.
Discovering biomarkers can speed up diagnosis, improve screening for clinical trials, and target in clinical trials those with the biomarker/specific version of ALS. In trials of the general ALS public, often results are not positive enough to continue to the next trial phase possibly because some/most of the people in the trial have a version of the disease that is different than that being targeted. Using a targeted subset means the results of trials will be more accurate and will allow more!
- A better, more effective pre-screening process of pALS will be beneficial to both the pALS and the research team. myTomorrows is a company focusing on creating and improving this process. (myTomorrows)
RESEARCH
NOTE: CURE ALS has funded centers that have been supporting or working on the TDP43 protein!!!
- QurAlis: The loss of normal protein TDP43 function leads to a highly significant decrease in the functionality of STMN2, which contributes to motor neuron loss, cytoskeletal weakness, and an impairment in neuronal repair which could be rescued by restoring STMN2 levels. QRL201 is designed to restore the function of STMN2. This study is now in the Phase I stage of clinical trial.
- USC: The protein TDP43 normally operates in and around the DNA and RNA of a cell. When it becomes toxic, it ends up outside of that region, unable to perform its normal function. Using cell/blood samples from people with ALS that have been changed into motor neurons, researchers have found that PIKFYVE reduced neurodegeneration, improved motor function, and lengthened life by stimulating motor neurons to envelope and dispose of the TDP43 toxic proteins. Studies are continuing.
- Packard Center at Johns Hopkins: Nuclear pores are a part of the nuclear envelope that regulates the transportation of molecules between the nucleus and the cytoplasm. Researchers discovered that when the CHIM7 protein is found inside the nucleus (instead of in the cytoplasm where it belongs), the protein TDP43 is in the cytoplasm (instead of in the nucleus where it belongs) and some of the nuclear pores are damaged and/or missing. Researchers have found that when CHIM7 is removed from the nucleus, the nuclear pores are completely repaired and defects in TDP43 still in the nucleus are repaired. This process has been studied using cells/blood in samples of those living with ALS and from autopsies of those whose lives were taken because of this disease. This study is not ready yet for clinical trials. EXCITING SIDE BENEFIT: This study can be used to identify an ALS biomarker that can be used to create a subset of people that have this specific version of ALS. This means that a clinical trial for CHM7/TDP43 can be done using the specific subset of people that have this biomarker, hopefully resulting in a trial that will produce a more accurate (and positive!) endpoint and will allow the study to move to the next trial phase!
- Toferson was approved by the FDA in 2023 for the treatment of the SOD1 gene under the Accelerated Approval pathway. It is hoped that the mechanics of Toferson can be used for other genetic forms of ALS.
- The University of Florida Center for Neurogenics is recruiting participants for a study associated with the C9orf72 genetic version of ALS.
RESEARCH FOCUSING ON “LONG TIMERS” (survival longer than the typical 2-5 years)
- Panelists at the seminar stressed the importance of those living longer than the typical 2-5 years taking part in biomarker, natural history, and genetic studies to allow researchers to learn what is different about them that allows their longevity.
- There is a blossoming field on how to treat people who have had ALS for a longer time. Research on regeneration and repair is taking place in the labs right now, hoping to be moved forward to clinical trials.
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