Long-term hope on stem cells for deafness

A reader of The Niche recently asked me, “What about stem cells for deafness?” It’s a great question and, as they pointed out, stem cells for vision loss gets much more attention.

I’ve covered the translational progress of stem cells to treat many diseases but there has been less progress for deafness in the regenerative medicine arena.

It was exciting recently to hear about success with one type of regenerative medicine, a gene therapy, for a common form of deafness.  Gene Therapy Allows an 11-Year-Old Boy to Hear for the First Time, NYT.  It also got me thinking more again about the reader’s question and stem cells for hearing loss.

Where do things stand in this area?

Before we jump into this, it’s important to note that some people with hearing loss do not see it as a disability or negative condition.

stem cells for deafness
Research on stem cells for deafness.  ANP1 cells (red) delivered to the cochlea in an animal model of hearing loss.  Image courtesy of Lineage Cell Therapeutics. Note that ANPs are auditory neuronal progenitor cells.

Cell therapies and stem cells for deafness

Unfortunately, at present I could only find 8 trial listings on Clinicaltrials.gov for a search for stem cells for deafness.

Of those, only three are recruiting and none are interventional trials of cell therapies for hearing loss.

This trial search could have missed some relevant interventional cell therapy trials. So I searched again, this time using the broader “cell therapy” term with deafness. I got more trials but none of the interventional trials used stem cells. One gene therapy trial listed looks interesting but it seems to be early days there. This was not the same one mentioned earlier.

Given the multiple causes of hearing loss, it is likely that a variety of cell and gene therapies will be needed to address them. In some cases the hair cells of the ear will need to be replaced rather than gene edited. To make new therapies here a reality we need biotech companies.

Biotechs working on cell therapy for hearing loss including Lineage Cell

Cell therapy biotechs working in this area include Lineage Cell Therapeutics and Rinri.

I asked Brian Culley, CEO of Lineage Cell Therapeutics, for his thoughts on where things stand:

“Recent reports of gene therapy being used to treat a patient with otoferlin-mediated hearing loss are incredibly exciting. This is a condition with no FDA-approved therapeutic interventions and it’s exciting to finally see progress made in this area.”

“A challenge for gene therapy is that it can only address one gene. But >150 different genes can cause hearing loss and approximately 95% of kids don’t have an otoferlin mutation, so while it’s a wonderful breakthrough, it’s irrelevant to most of the patient population. There also are countless people with hearing loss from non-genetic sources, such as aging and degeneration. One of the possible advantages of using an auditory cell transplant is that replacing the entire cell might address a much wider array of underlying cellular dysfunction, all in one treatment.”

As he rightly pointed out cell therapies may address a broader range of causes of hearing loss than any one given gene therapy. Brian also provided the beautiful image above from their preclinical research. They are getting some nice ANP engraftment there in the cochlea of an animal model.

Looking ahead

There is real hope for the future related to both gene and cell therapies for hearing loss.  Realistically, it’s going to take time though given where we are now. Hopefully, we’ll see more active, interventional cell therapy clinical trials soon.

Note that Lineage Cell is one of the more exciting cell therapy biotechs. I’m rooting for their other work to succeed too including on OPC1 for spinal cord injury.

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