Multiple System Atrophy: A case study in hope for rare diseases

By Jan Shultis

During the last few weeks of 2025, Houston-area clinical research organization Hope Biosciences Research Foundation (HBRF) received Food and Drug Administration (FDA) authorization to administer Hope Biosciences’ allogeneic (donor) adipose-derived mesenchymal (adult) stem cells (HB-adMSCs) to seven men and women living with Multiple System Atrophy (MSA). As HBRF shared in their announcement, the occasion is noteworthy both for authorization of research in a rare condition and for the first-of-kind protocol design. (As of this writing, the trial is pre-screening participants, moving soon to enrollment. For more information, visit hopebio.org or find the MSA protocol listed on clinicaltrials.gov, NCT07238062.)

As manufacturers of the investigational therapeutic at work in this protocol, Hope Biosciences is proud to advance research in rare diseases, including MSA. Hope’s stance is often considered unusual in the broader pharmaceutical and healthcare industry, which can be perceived as laser-focused on large population areas of most promising financial gain. Sometimes, we are asked why we are willing to go to the lengths that we do, to press our products into service for small groups of people with complex, highly individualized conditions. How does manufacturing cells for a handful advance the vision of one day making cells available to all?

https://www.youtube.com/watch?v=mfJdgwuMedM

The MSA protocol is an excellent and timely jumping point from which to frame an answer about how and why Hope Biosciences believes work in rare diseases is a service both to people and science, and a key to advancing access. Rare disease research matters because:

Results from research in rare diseases can be reasonably extrapolated to other conditions with larger patient populations, because rare diseases fall within disease families or clusters of symptoms. MSA, for example, is a parkinsonian-type condition. Positive safety and efficacy outcomes in individuals suffering from other nervous system condition played a significant part in MSA protocol design. Hope Biosciences has previously supplied billions of cells to hundreds of patients for six successfully completed FDA-authorized studies in Parkinson’s Disease, including a recently concluded Phase II clinical trial with positive topline results. When designing future protocols, Hope Biosciences can look across the swath of all research in parkinsonian-type conditions to identify trends, including data and insights gained from the MSA protocol.

MSA is also a severe, rapidly progressing disease, placing it in the company of other conditions for which Hope Bio has manufactured cells, including but not limited to amyotrophic lateral sclerosis (ALS), certain types of cancer, ataxia, acute Parkinson’s and Multiple Sclerosis, and in palliative care. Again, these cases can be assessed together for patterns in periodicity, volume, and method(s) of administration.

Hope Bio's cells administered to patients during an FDA-authorized clinical trial conducted at an independent clinical research organization.

The complexity of rare diseases can open pathways to administer Hope Bio’s therapeutics in newly refined ways. The MSA protocol, for instance, is the first of its kind to dictate both intravenous and intrathecal administration of HB-adMSCs (over the course of twelve months, patients will receive monthly intravenous infusions of 200 million cells per treatment, and bi-monthly intrathecal injections at a dose of 100 million cells, for a total of twelve intravenous and six intrathecal injections.) Though Hope Biosciences has recorded safe mixed or alternating administration in hundreds of individual patients through Right to Try, an intermediate size Expanded Access protocol like this one, in MSA, represents an important next step in codifying institutional knowledge in increasingly large patient populations. Research organizations and manufacturers find similar value here – rare disease studies represent an opportunity to learn more about how a drug works and patients respond, while illuminating ways to become increasingly efficient in future research with larger participant populations.

Click image to read more about Hope Bio's RMAT designation.

Rare diseases are areas of high need, which means regulatory pathways exist to speed the access pipeline for qualifying therapeutics. Hope Biosciences recently received Regenerative Medicine Advanced Therapy (RMAT) designation for HB-adMSCs for treatment of patients living with relapsing-remitting multiple sclerosis (RRMS). To qualify for RMAT, a therapy must demonstrate preliminary clinical evidence of addressing a serious or life-threatening condition with significant unmet medical needs, as MSA does. The process requires compelling proof that the therapeutic has potential to substantially improve patient outcomes where existing treatments fall short, as Hope Bio has reason to believe the MSA protocol will reveal in this disease condition. Ultimately, RMAT designation grants eligibility for priority review or accelerated approval, including enhanced FDA collaboration. Though the MS trial was significantly larger than the current MSA protocol, continued work in MSA may open regulatory doors in a comparable manner.

Rare diseases are areas of high need, which means real people need real hope, now. MSA is a rare, progressive disease of the nervous system. The majority of MSA epidemiological studies to date have been conducted in Europe, with additional studies readily found from Brazil, Japan and the United States; estimated global prevalence ranges between 2-3 people/100,000. In MSA, parts of the brain atrophy, resulting in loss of motor control typically affecting mobility, speech, bladder control, ability to swallow, and other functions with dramatic implications for quality of life. Generally manifesting in the 50s and 60s, an MSA diagnosis is clinically expected to conclude in death in a projected 5-10 years after diagnosis. MSA is of unknown cause and conventionally considered “incurable,” with no treatment options to reverse or slow disease progression.

The study underway using Hope Bio’s stem cells is a direct answer to a community-driven cry for treatment options. If Hope Bio philosophically believes that technology, including ours, exists to serve people, and if it can be credibly demonstrated that small population rare disease research is a viable pathway toward widespread access, as has been articulated in the preceding points, then the only morally and ethically consistent response to that cry for hope is, “yes!”

And so, Hope Biosciences says “yes,” with hope, to continued rare disease research, including today in MSA.