Grünenthal Partners with Max Planck Society to Develop CMT Type 1A Treatments

Grünenthal, the Lead Discovery Center (LDC) and the Max-Planck Society have signed a research collaboration agreement to develop new therapies for Charcot-Marie-Tooth type 1A (CMT1A).

Two branches of Max-Planck are involved: Max-Planck Innovation and the Max Planck Institute of Experimental Medicine (MPI-EM). These two will work with LDC — an independent company established by MPI in 2008 —  to develop a new screening platform to identify small molecules that might be used in developing treatment candidates.

These efforts will be funded equally by Grünenthal, a German company with a core expertise in pain management, and the Planck Society, also based in Germany.

Grünenthal will be responsible for developing, starting with preclinical or lab research, any treatment candidates stemming from that basic research.

“It’s our aspiration to provide patients with disease modifying treatments. We therefore team up with academia and foster research collaborations leveraging basic research,” Gabriel Baertschi, Grünenthal’s chief executive officer, said in a press release.

This is particularly important, he added, as there now is “no curative treatment available for patients affected by CMT1A” and the burden that places on patients and caregivers.  “We’re looking forward to developing potential treatment options with our partners.”

“We are enthusiastic to collaborate with … our MPI-EM colleagues on this early-stage drug discovery project,” said Bert Klebl, managing director and chief scientific officer of Grünenthal, and “hopefully make a difference in the lives of CMT1A patients.”

CMT1 is the most common type of Charcot-Marie-Tooth disease, accounting for about two-thirds of all recorded cases.

Typical symptoms include muscle weakness and atrophy, as well as reduced sensitivity to touch, heat and cold, particularly in the upper and lower extremities.

CMT1 is caused by genetic mutations that damage the myelin sheath, a protective layer that allows the transmission of nerve signals from the brain to the muscles. The loss of myelin results in a slow transmission of these nerve signals, resulting in decreased sensitivity.

Specifically, CMT1A patients have an extra copy of the PMP22 gene, which codes for the peripheral myelin protein 22. This protein is a key component of the myelin sheath; its duplication disrupts the sheath’s structure and function.

Like CMT1, CMT1A begins with symptoms like leg muscle weakness and atrophy during teenage years, followed by hand weakness and decreased sensations later in life. Most people with this disease subtype remain ambulatory.