SyStemix Inc.'s first clinical trial of its approach to reconstitutingchemotherapy-destroyed bone marrow with autologous stem celltransplants not only will test the procedure as a treatment for multiplemyeloma patients, but also will attempt to establish the technologyfor the company's other programs, such as gene therapy against HIV.

SyStemix developed a method of isolating hematopoietic stem cells(HSC), called CD34Thylin, and received a patent on both the processand the substantially pure population of stem cells being selected.

Because the targeted HSCs are responsible for renewing all bloodand immune system cells, SyStemix also hopes to use its technologywith gene therapy to attack disorders by enabling the body toregenerate cells that resist disease or correct genetic deficiencies.

The potential wide-range of uses of the HSC isolation technology andthe patent protection for the stem cells are what attracted Switzerlandpharmaceutical giant, Sandoz AG, to pay nearly $400 million in 1991to acquire a 60 percent stake in SyStemix. Earlier this year Sandozmade another $80 million equity investment, giving it 71.6 percentownership of the Palo Alto, Calif. company.

Sandoz also is collaborating with SyStemix on developing an HIVgene therapy employing the patented HSCs. The idea is to isolate thestem cells and transduce them with a gene that resists HIV in thehopes that the gene will be transferred to T cells and protect themagainst the infection.

John Schwartz, SyStemix's president and CEO, said the company isconducting the first clinical trial of its stem cell therapy on its own.The study is aimed at restoring multiple myeloma patients' bonemarrow and its ability to regenerate white blood cells and plateletsafter they have been depleted or destroyed by chemotherapy. Thecompany was expected to announce today the launch of the trials.

Robert Faulkner, an analyst with S.G. Warburg & Co. Inc., in NewYork, said the myeloma studies are "viewed as the proof of principle"for SyStemix's technology.

"This is what we've been waiting for," he said, "to see if thetechnology lives up to its promise and we expect it to do so."

The Phase I/II trial will involve 20 multiple myeloma patients andwill be conducted by Guido Tricot and Bart Barlogie at the ArkansasCancer Research Center in Little Rock.

The HSCs are isolated using SyStemix's high speed cell sorter andpurified at the company's cell processing center to generate anautologous transplant, 90 percent of which is made up of theCD34Thylin stem cells. The treatment is being developed as asubstitute for conventional bone marrow transplants.

Schwartz said earlier research showed the HSC population isolatedby SyStemix is so pure, it also is free of myeloma tumor cells, whichmay help prevent relapses of the disease.

Christopher Juttner, Systemix's vice president of medical affairs, saidthe patients will receive two infusions of the stem cell transplants.The trials will assess the safety of the procedure and the ability of theprocess to rehabilitate the bone marrow.

In addition to testing the stem cell transplants for myeloma, Juttnersaid clinical trials are targeted for breast cancer and non-Hodgkin'slymphoma.

Juttner said Systemix's approach differs from other stem cell therapyefforts in that the transplants generated are more pure and containmany more HSCs than those of companies isolating CD34+progenitor cell populations. The CD34+ populations contain a varietyof cells, some of which are not self-renewing and which may containcancer cells.

Said Schwartz, "As far as we know, no one is using pure HSC eitherfor isolation or as a target for gene therapy. It is an ideal target forgene therapy. The cells self-renew and will provide a life-longtreatment."

Faulkner said what differentiates SyStemix's stem cell therapy fromcompetitors is a process of selecting each cell individually andidentifying its makeup.

The technology, he added, could have an impact on how the FDAregulates procedures and devices used in stem cell therapy.

"If the FDA requires that dosage be defined - that is, determining thenumber of cells of a particular type - and if SyStemix can establishthe elimination of tumor cells, that may be the standard," Faulknersaid.

SyStemix's stock (NASDAQ:STMX) closed Wednesday up 25 centsto $13.75. n

-- Charles Craig

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