Clinical trials give patients access to new medications and potential treatments, and many home health and hospice patients may benefit from participation. Here’s a look at some extraordinary trials happening in 2023.
1. Psilocybin Therapy for Demoralization in Terminal Illness
Patients living with a terminal illness may experience demoralization, or severe emotional distress and hopelessness. This condition is different from depression and is considered a normal reaction to overwhelming circumstances, such as a terminal diagnosis.
A clinical trial out of the Dana-Farber Cancer Institute is researching whether psilocybin therapy is safe and useful in hospice care and whether it relieves symptoms of distress. Patients will receive a dose of psilocybin, a naturally occurring psychedelic drug, along with supportive psychotherapy sessions.
The trial is open to patients aged 21 years or older enrolled in hospice care for any terminal illness and experiencing moderate to severe demoralization. Psilocybin isn’t an approved drug, but the FDA has approved the study for investigation of use in hospice care.
2. Gene Therapy for Muscular Dystrophy
Limb-girdle muscular dystrophy (LGMD) is a group of diseases that cause muscle wasting and weakness in the arms and legs. Symptoms vary, but it can cause a weak heart, severe breathing problems, and disability.
A new study expected to start in the summer of 2023 will research the safety of gene-edited muscle stem cell therapy in LGMD patients. The study will also examine whether the treatment encourages healthy muscle development.
The study is open to patients aged 14 years or older who have a confirmed LGMD diagnosis. This will be the first time gene-edited muscle stem cells are tested in humans.
3. HIV Drug for Amyotrophic Lateral Sclerosis or Frontotemporal Dementia
TPN-101 is a reverse transcriptase inhibitor originally developed to treat HIV. Current studies aim to repurpose this drug for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. The rationale for this new research is based on recent work that shows these conditions involve the activation of virus-derived pieces of DNA.
A study from the University of California San Francisco is researching the safety and tolerability of TPN-101 for ALS and frontotemporal dementia (FTD). It will also study whether the drug affects neurodegeneration and disease symptoms.
The study is open to adult patients with ALS or FTD who have a reliable caregiver who can attend appointments. ALS patients must have an onset of weakness within three years before the screening. FTD patients must have a gradual, progressive decline in motor function, behavior, and language.
Qualifying for a Clinical Trial
Clinical trials often have many eligibility requirements. Patients can volunteer for trials on their own, but it’s important to carefully review clinical trial requirements.
Patients should also contact the trial team and ask questions before consenting to treatment. Confirm the goals and length of the trial, the tests and treatments involved, and why the treatment is thought to be better than what is already available.
Ultimately, participation is voluntary. While it can provide new treatment, it’s important to understand both the benefits and the risks. If a trial seems like a good fit, talk to a physician and get in touch with the trial coordinator.
