Cell Therapies vs. Neurodegeneration

A personal story and where I see breakthroughs making the leap from labs to lives.

Jun 10, 2025

Hi! I’m Cristina. With roots in science and a track record leading strategy and partnerships across biotech, I focus on one thing: identifying breakthroughs that will make cell therapies mainstream—and invest before the world catches on.

And the writing experiment continues! The “21/90” rule is the idea that it takes 21 days to build a habit and 90 days to turn it into a lifestyle. I’m not sure how many posts it takes to start calling yourself a writer, but I guess I’m finding out in real time (Feel free to share your experiences! or just moral support!!)

This one’s a bit more personal. I hope you’ll find it interesting to see where I believe cell therapies could make a real difference for patients facing neurodegenerative diseases.

Before She Forgot

Martina Gomez was born on November 11, 1923, in the rural town of San Miguel on Isla del Rey, the largest of Panama’s Pearl Islands, a remote archipelago scattered across the Pacific Ocean. The islands earned their name from the tiny, precious pearls that once lay hidden in their waters.

Martina, like most of the 20th-century residents of the Pearl Islands, was a descendant of African slaves brought by Spanish conquistadores and forced into the perilous labor of pearl diving. The conquistadores were relentless not just in their exploitation of labor but in their efforts to convert both the native populations and the enslaved Africans to Catholicism. As a result, Martina grew up a devout Catholic, shaped by both faith and a deep-rooted fear of everything. Despite living on an island, Martina never learned to swim. She carried, unsurprisingly, a fear of la tintorera (Blue Sharks), which, as I later learned, were said to have “taken the lives” of pearl divers, possibly including her own ancestors. [Fun fact: Blue sharks are generally considered nonaggressive but, in modern days, there have been documented cases of unprovoked bite incidents]

Martina made it all the way through the 5th grade in her tiny island and though she didn’t have the chance to pursue higher education, she became a fierce believer that education was the only path out of poverty. Which is why, as an adult, she worked 12-hour days - ironing, cooking, and cleaning for others - to ensure her seven children had the opportunity to study as far as their means would allow.

Despite her tough upbringing, Martina was full of joy and humor. She loved sneaking up on children just to make them laugh, and her cooking was legendary, especially her rich fish soup and her signature coconut rice, which the family joked could “raise the dead.” Martina was known for her sharp mind. She never forgot a birthday and had an uncanny ability to remember every important date, address, and phone number, reminding everyone of them whether they asked or not. She remembered every story of her youth, particularly those that served as warnings or had a lifelong lesson embedded in them. It was as if memory itself was her secret superpower. Martina was deeply loved.

So, when after a lifetime of hardships, she finally owned her own home, had the means to travel, and could watch her grandchildren grow up with opportunities she never had, it was devastating to learn that Martina had Alzheimer’s Disease. She lived with the disease for over a decade, a slow and painful erasure of everything and everyone she once carried so vividly in her mind. I know this story so well because Martina was my Abuela. And not a day goes by that I don’t think of her and how much of her still lives on in me.

Restoring the Mind: The Potential of Cell Therapies

In the early 2000s, Shinya Yamanaka, a Japanese physician and researcher, became curious: Could adult cells be rewound to an earlier, more powerful, embryonic-like state? As it turns out, yes. Through a mix of meticulous experiments and what we imagine was a fair amount of scientific determination, Yamanaka and his team pinpointed the four transcription factors that are needed to reprogram ordinary adult skin cells into a pluripotent state, capable of becoming nearly every cell in the body. The reprogrammed cells, dubbed iPSCs (induced pluripotent stem cells), looked and behaved a lot like Embryonic Stem Cells (minus the ethical baggage, and without all the controversial headlines).

By 2012, Yamanaka had won the Nobel Prize in Physiology, and those once-obscure transcription factors had reached celebrity status in stem cell circles, now fondly known as the “Yamanaka factors”. Today, pluripotent cells like iPSCs are no longer just lab curiosities, they’re the engine behind next-gen therapies aiming to do what no pill or protein has done before: restore the neurons and connections lost to neurodegenerative conditions. Thanks to precise, differentiation protocols, pluripotent cells can turn into cell in the body, including neurons, astrocytes, microglia, and neural precursor cells.

Neurodegenerative conditions like Alzheimer's, Parkinson's, Huntington's, and ALS are a growing global crisis. And despite billions poured into research, most treatments today are still just buying time, not restoring function. The need for truly disease-modifying therapies is urgent. Let’s dive into the progress the field of cell therapies have achieved to date.

Parkinson’s Disease (PD)

Parkinson’s Disease starts with a silent, gradual loss of dopamine-producing neurons in a brain region called the substantia nigra. Dopamine is the neurotransmitter behind our movements, posture, and coordination. Without it, even simple tasks like buttoning a shirt or tying a shoe become uphill battles. While the exact cause remains unknown, aging remains the strongest risk factor, as it is with many other neurodegenerative conditions. Parkinson’s is chronic, progressive, and so far, incurable.

“Parkinson’s disease is a gift. It’s the gift that keeps on taking” - Michael J. Fox

In 2016, Dr. Lorenz Studer along with Dr. Gordon Keller, world-renowned scientists and pioneers in stem cell biology, co-founded BlueRock Therapeutics to translate decades of stem cell research into therapies for patients with PD.

BlueRock is developing bemdaneprocel, an off-the-shelf (yes, the kind you don’t need to custom-make, see my previous post for more on that) investigational therapy made from pluripotent stem cells. The goal is to replace the dopamine-producing neurons lost in Parkinson’s Disease, and for the first time, move from managing symptoms to actually restoring function. Phase 1 clinical trials for bemdaneprocel were a big deal because the study showed signs that the therapy is helping patients control symptoms. In 2019, Bayer acquired BlueRock, a move that signaled Big Pharma’s confidence in the platform and the potential of regenerative medicines (more on Big Pharma’s moves in a previous post). In 2024, the FDA granted the Regenerative Medicine Advanced Therapy (RMAT) designation to bemdaneprocel, a fast-track path reserved for the most promising therapies. So promising, in fact, that BlueRock skipped the typical Phase 2 and jumped straight into Phase 3 trials earlier this year, a move that signals real confidence in the data and the platform.

Chronic Focal Epilepsy

Drug-Resistant Mesial Temporal Lobe Epilepsy (MTLE) is the most common form of focal epilepsy in adults, and one of the toughest to treat. Seizures begin in the mesial temporal lobe and don’t play nicely with standard anti-seizure drugs. For many, it’s a frustrating cycle of trial-and-error with little relief.

Neurona is taking a bold, regenerative swing at the problem. The company is developing an off-the-shelf (everyone is moving in this direction!), one-time cell therapy made from GABAergic interneurons derived from pluripotent stem cells. The goal is elegant in its simplicity: restore inhibitory signaling in hyperactive brain circuits by releasing GABA, the brain’s natural calming neurotransmitter. In Phase 1/2, the results were more than promising, and some patients saw a >95% reduction in seizures that held steady for at least two years. In 2024, the FDA granted Neurona RMAT designation, clearing the runway and enabling the company to head straight into Phase 3. Neurona is expanding its pipeline, not just for epilepsy, but also exploring Alzheimer’s!

Amyotrophic Lateral Sclerosis (ALS)

In the summer of 2014, the Ice Bucket Challenge took over the internet. What started as a quirky dare to dump ice water on your head turned into a viral phenomenon that raised millions globally for ALS research in just a few weeks. Everyone from your next-door neighbor to Oprah and Bill Gates took the plunge.

ALS, or Amyotrophic Lateral Sclerosis, is a devastating neurodegenerative disease that gradually robs people of their ability to move, speak, eat, and eventually breath. It targets the motor neurons in the brain and spinal cord. You may also know it as Lou Gehrig’s disease, named after the legendary baseball player who brought attention to the illness in the 1930s.

“Be passionate, be genuine, be hardworking, and don't ever be afraid to be great” - Pete Frates, baseball player, ALS patient, and creator of the Ice Bucket Challenge

Earlier in 2025, the FDA gave XellSmart the green light to launch a Phase 1 clinical trial testing their motor neuron precursor derived from pluripotent stem cells. What’s really encouraging is the studies from China, which showed that the therapy was safe and slowed disease progression better than current standard treatments. While it’s early days, this represents a hopeful step toward tackling a disease that’s long been stubbornly resistant to effective therapies.

Where the Opportunities Lie

I know there are lots of exciting directions in cell therapy right now, but the progress in Alzheimer’s therapies feels especially urgent and personal.

Alzheimer’s Diseases (AD) is the most common form of dementia worldwide, affecting over 24 million people, a number that’s projected to rise dramatically as populations age. AD is characterized by the aggregation of amyloid plaques and tau tangles in the brain, reflecting a broader pattern of toxic protein aggregation seen across many neurodegenerative diseases.

Most AD cases are late-onset and sporadic, with age as the biggest risk factor. But there’s also a surprising and well-documented link with low educational attainment [So maybe education isn’t just a path out of poverty, it’s also cognitive insurance. Good thing I went to school for nearly 30 years!].

I recently read a study that really stuck with me: researchers used microglia derived from pluripotent stem cells to deliver therapeutic enzymes throughout the brain. The same team had previously developed a reliable protocol to differentiate iPSCs into microglia and then improved it further by adding genetic engineering using (of course) CRISPR/Cas.

In a preclinical model of Alzheimer’s, mice treated with these CRISPR-edited microglia showed a significant reduction in amyloid plaques, even in tough to reach areas like the subiculum. But what really blew me away wasn’t the reduction in plaques (some other modalities have achieved that); it was that the therapy also prevented neuronal loss and preserved synaptic integrity. If anything close to this translates in humans, it could be a total game-changer.

Of course, part of me is always a little scared that I’ll face this disease myself one day, like my abuela did. But knowing that this kind of progress is happening and seeing how far we’ve come in just a few years, gives me real comfort. And it fuels my conviction to keep investing in this space.

If this sparked any ideas or nudge your thinking, I’d love to hear where you think the field heading. And if you’re looking for deeper takes or warm intros to operators and founders building in this space, just say the word!