Editors' (re)View: Industry dedication to rare disease; Musk brain implants ready for humans?

June 9, 2023
Pharma Manufacturing editors Karen Langhauser and Andrea Corona comment on the notable happenings in the pharma industry from the week of June 5th.

Editor’s note: Welcome to Editors' (re)View, our editors’ takes on things going on in the pharma world that deserve some extra consideration.

Dedication to rare disease patient perspectives

This week we reported that Astellas Pharma is licensing a novel gene therapy from Kate Therapeutics for a rare muscle disorder called X-linked myotubular myopathy (XLMTM).

The move was notable because it was the same indication in which the company had failed in the worst way — four patient deaths during a clinical trial — while testing a gene therapy Astellas had acquired through the $3 B purchase of Audentes Therapeutics back in 2019.

XLMTM is a debilitating disorder that tends to affect boys more severely than girls. Mortality rates are estimated to be 50% in the first 18 months of life, and for patients who survive past infancy, approximately 75% will live to the age of 10. More than 80% of XLMTM patients require ventilator support and most  need a gastrostomy tube for nutritional support.

I applaud Astellas' commitment to this disease. Further digging on their site lead me to a red link on the top of the page labeled "patients and families," where the drugmaker is encouraging those affected by XLMTM to share their experiences so that Astellas can integrate the patient and family perspective into their drug development process.

Awhile back, I wrote a cover story about the rare disease space, which began as literal mom-and-pop industry — where everyday people become advocates, fundraisers, lobbyists and researchers. The niche space has been painstakingly built on the backs of patients and caregivers, with many treatments either compounded for individual patients in labs run by academic institutions or manufactured by small, newly formed biotechs.

Notably absent in the scrappy history of orphan drugs are multinational pharma giants. But over time their involvement has grown. As the industry has become more adept at developing drugs for small patient populations, bigger pharma companies are becoming more visible in what is now a swelling rare disease space.

The key to success in rare disease drugs for the pharma industry appears to be a willingness to stay true to the core values of the once niche space. Pharma companies that figure this out can realize countless benefits — for both their bottom lines and the 300 million patients worldwide suffering from rare diseases.

From the looks of it, Astellas seems to understand this, and hopefully that will lead to much-needed treatments for those suffering from XLMTM.

—Karen Langhauser

FDA says Neuralink can test in humans

This week, in true Elon Musk fashion, the billionaire’s medical device company Neuralink announced in a vague tweet that the FDA had given it the approval to launch its first-in-human clinical study.

The news was surprising, if not scary, for a number of reasons. But mainly, because most Neuralink headlines have been anything but promising.

In December of 2022, Reuters reported that the company was under federal investigation by the Office of the Inspector General for potential animal-welfare violations amid internal staff complaints that its animal testing is being rushed, causing needless suffering and deaths.

As reported by Reuters, records examined and conversations with individuals with firsthand information about the company's animal-testing activities confirmed that since 2018, the company has conducted experiments that resulted in the deaths of approximately 1,500 animals. One of the monkeys mentioned in the complaint to the DOT, labeled "Animal 11" by UC Davis, was implanted with electrodes by Neuralink. The surgery resulted in infections and erosion of the monkey's skin. After three months of persistent infections, the monkey was euthanized. A postmortem examination revealed acute brain bleeding and damage to the cerebral cortex caused by Neuralink's devices.

Despite these allegations and concerning data, in a total of 68 words, Neuralink excitedly revealed nothing more than the fact that it had received approval to launch its first human trials and that recruitment is not open yet. “We’ll announce more information on this soon!” Neuralink tweeted.

Much like any other of Musk’s endeavors, Neuralink’s latest update sounds like the beginning of a dystopian movie I’m not sure I want to watch yet. 

— Andrea Corona